69 results on '"Ashwin K Lal"'
Search Results
2. Genetic Causes of Cardiomyopathy in Children: First Results From the Pediatric Cardiomyopathy Genes Study
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Stephanie M. Ware, James D. Wilkinson, Muhammad Tariq, Jeffrey A. Schubert, Arthi Sridhar, Steven D. Colan, Ling Shi, Charles E. Canter, Daphne T. Hsu, Steven A. Webber, Debra A. Dodd, Melanie D. Everitt, Paul F. Kantor, Linda J. Addonizio, John L. Jefferies, Joseph W. Rossano, Elfriede Pahl, Paolo Rusconi, Wendy K. Chung, Teresa Lee, Jeffrey A. Towbin, Ashwin K. Lal, Surbhi Bhatnagar, Bruce Aronow, Phillip J. Dexheimer, Lisa J. Martin, Erin M. Miller, Lynn A. Sleeper, Hiedy Razoky, Jason Czachor, and Steven E. Lipshultz
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exome ,heart failure ,infant ,molecular ,mutation ,Diseases of the circulatory (Cardiovascular) system ,RC666-701 - Abstract
Background Pediatric cardiomyopathy is a genetically heterogeneous disease with substantial morbidity and mortality. Current guidelines recommend genetic testing in children with hypertrophic, dilated, or restrictive cardiomyopathy, but practice variations exist. Robust data on clinical testing practices and diagnostic yield in children are lacking. This study aimed to identify the genetic causes of cardiomyopathy in children and to investigate clinical genetic testing practices. Methods and Results Children with familial or idiopathic cardiomyopathy were enrolled from 14 institutions in North America. Probands underwent exome sequencing. Rare sequence variants in 37 known cardiomyopathy genes were assessed for pathogenicity using consensus clinical interpretation guidelines. Of the 152 enrolled probands, 41% had a family history of cardiomyopathy. Of 81 (53%) who had undergone clinical genetic testing for cardiomyopathy before enrollment, 39 (48%) had a positive result. Genetic testing rates varied from 0% to 97% between sites. A positive family history and hypertrophic cardiomyopathy subtype were associated with increased likelihood of genetic testing (P=0.005 and P=0.03, respectively). A molecular cause was identified in an additional 21% of the 63 children who did not undergo clinical testing, with positive results identified in both familial and idiopathic cases and across all phenotypic subtypes. Conclusions A definitive molecular genetic diagnosis can be made in a substantial proportion of children for whom the cause and heritable nature of their cardiomyopathy was previously unknown. Practice variations in genetic testing are great and should be reduced. Improvements can be made in comprehensive cardiac screening and predictive genetic testing in first‐degree relatives. Overall, our results support use of routine genetic testing in cases of both familial and idiopathic cardiomyopathy. Registration URL: https://www.clinicaltrials.gov; Unique identifier: NCT01873963.
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- 2021
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3. Risk of Sudden Death in Patients With RASopathy Hypertrophic Cardiomyopathy
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Aine Lynch, Mark Tatangelo, Sachin Ahuja, Chun-Po Steve Fan, Sandar Min, Myriam Lafreniere-Roula, Tanya Papaz, Vivian Zhou, Kathryn Armstrong, Peter F. Aziz, Lee N. Benson, Ryan Butts, Andreea Dragulescu, Letizia Gardin, Justin Godown, Aamir Jeewa, Paul F. Kantor, Beth D. Kaufman, Ashwin K. Lal, John J. Parent, Marc Richmond, Mark W. Russell, Seshadri Balaji, Elizabeth A. Stephenson, Chet Villa, John L. Jefferies, Robert Whitehill, Jennifer Conway, Taylor S. Howard, Stephanie J. Nakano, Joseph Rossano, Robert G. Weintraub, and Seema Mital
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Cardiology and Cardiovascular Medicine - Published
- 2023
4. Attitudes & practices surrounding pregnancy post heart transplant among pediatric providers
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Megan M. Collins, Zhining Ou, Morgan M. Millar, Michelle M. Kittleson, Lindsay J. May, Michelle S. Ploutz, Kimberly M. Molina, Katherine G. Hayes, and Ashwin K. Lal
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Adult ,Pulmonary and Respiratory Medicine ,Transplantation ,United States ,Attitude ,Pregnancy ,Surveys and Questionnaires ,Humans ,Heart Transplantation ,Female ,Surgery ,Child ,Cardiology and Cardiovascular Medicine ,Lung Transplantation - Abstract
Many pediatric heart transplant (HT) recipients reach adulthood and may be interested in family planning; there is little data regarding safety of pregnancy post HT and clinicians' opinions differ. Pediatric HT clinicians are instrumental in early counseling. Thus, a better understanding of pediatric HT clinicians' practices regarding family planning and how well aligned these practices are with adult transplant centers is essential.We conducted a confidential, web-based survey of pediatric HT clinicians in fall 2021. We summarized and compared answers using Fisher's exact test.The survey was sent to 53 United States-based HT directors and to the International Society for Heart and Lung Transplantation and Pediatric Heart Transplant Society list serves. There were 69 respondents. The majority (77%) of respondents felt pregnancy was feasible in selected or all female HT recipients. Ten respondents reported that their institution had an established policy regarding pregnancy post HT. A majority (77%) of HT clinicians would either use a shared care model or recommend transition to their adult institution if pregnancy occurred, though 74% of respondents were either unaware of their corresponding adult institution's policy (62%) or had a counterpart adult program with a policy against pregnancy post HT (12%).While many clinicians feel pregnancy is feasible in pediatric HT recipients, there remains significant practice variation. Few pediatric programs have a policy regarding pregnancy post HT. Future efforts to provide consistent messaging between adult and pediatric HT programs regarding the feasibility and care of post HT pregnancy are warranted.
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- 2022
5. A multi-site survey of providers on the management of heart failure with dilated cardiomyopathy in children
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Yuk M. Law, Elizabeth Jacobs-Files, Scott Auerbach, Ashwin K. Lal, Marc Richmond, Kurt Schumacher, Rakesh Singh, and Arti Desai
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Pediatrics, Perinatology and Child Health ,General Medicine ,Cardiology and Cardiovascular Medicine - Abstract
We conducted a scientific survey of paediatric practitioners who manage heart failure with dilated cardiomyopathy in children. The survey covered management from diagnosis to treatment to monitoring, totalling 63 questions. There were 54 respondents from 40 institutions and 3 countries. There were diverse selections of management options by the respondents in general, but also unanimity in some management options. Variation in practice is likely due to the relative paucity of scientific data in this field and lack of strong evidence-based recommendations from guidelines, which presents an opportunity for future research and quality improvement efforts as the evidence base continues to grow.
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- 2022
6. The genetic architecture of pediatric cardiomyopathy
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Stephanie M. Ware, Surbhi Bhatnagar, Phillip J. Dexheimer, James D. Wilkinson, Arthi Sridhar, Xiao Fan, Yufeng Shen, Muhammad Tariq, Jeffrey A. Schubert, Steven D. Colan, Ling Shi, Charles E. Canter, Daphne T. Hsu, Neha Bansal, Steven A. Webber, Melanie D. Everitt, Paul F. Kantor, Joseph W. Rossano, Elfriede Pahl, Paolo Rusconi, Teresa M. Lee, Jeffrey A. Towbin, Ashwin K. Lal, Wendy K. Chung, Erin M. Miller, Bruce Aronow, Lisa J. Martin, and Steven E. Lipshultz
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Cardiomyopathy, Dilated ,Male ,Genotype ,Gene Expression Profiling ,Inheritance Patterns ,Genetic Variation ,Article ,Cohort Studies ,Phenotype ,Gene Expression Regulation ,Case-Control Studies ,Practice Guidelines as Topic ,Exome Sequencing ,Genetics ,Humans ,Exome ,Female ,Genetic Predisposition to Disease ,Genetic Testing ,Age of Onset ,Child ,Genetics (clinical) - Abstract
To understand the genetic contribution to primary pediatric cardiomyopathy, we performed exome sequencing in a large cohort of 528 children with cardiomyopathy. Using clinical interpretation guidelines and targeting genes implicated in cardiomyopathy, we identified a genetic cause in 32% of affected individuals. Cardiomyopathy sub-phenotypes differed by ancestry, age at diagnosis, and family history. Infants < 1 year were less likely to have a molecular diagnosis (p < 0.001). Using a discovery set of 1,703 candidate genes and informatic tools, we identified rare and damaging variants in 56% of affected individuals. We see an excess burden of damaging variants in affected individuals as compared to two independent control sets, 1000 Genomes Project (p < 0.001) and SPARK parental controls (p < 1 × 10(−16)). Cardiomyopathy variant burden remained enriched when stratified by ancestry, variant type, and sub-phenotype, emphasizing the importance of understanding the contribution of these factors to genetic architecture. Enrichment in this discovery candidate gene set suggests multigenic mechanisms underlie sub-phenotype-specific causes and presentations of cardiomyopathy. These results identify important information about the genetic architecture of pediatric cardiomyopathy and support recommendations for clinical genetic testing in children while illustrating differences in genetic architecture by age, ancestry, and sub-phenotype and providing rationale for larger studies to investigate multigenic contributions.
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- 2022
7. Diversity of Dystrophin Gene Mutations and Disease Progression in a Contemporary Cohort of Duchenne Muscular Dystrophy
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Katheryn E. Gambetta, Michael A. McCulloch, Ashwin K. Lal, Kenneth Knecht, Ryan J. Butts, Chet R. Villa, Jonathan N. Johnson, Jennifer Conway, Matthew J. Bock, Kurt R. Schumacher, Sabrina P. Law, Joshua M. Friedland-Little, Shriprasad R. Deshpande, Shawn C. West, Irene D. Lytrivi, and Carol A. Wittlieb-Weber
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Pediatrics, Perinatology and Child Health ,Cardiology and Cardiovascular Medicine - Published
- 2022
8. Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION
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Chet Villa, Scott R. Auerbach, Neha Bansal, Brian F. Birnbaum, Jennifer Conway, Paul Esteso, Katheryn Gambetta, E. Kevin Hall, Beth D. Kaufman, Sonya Kirmani, Ashwin K. Lal, Hugo R. Martinez, Deipanjan Nandi, Matthew J. O’Connor, John J. Parent, Frank J. Raucci, Renata Shih, Svetlana Shugh, Jonathan H. Soslow, Hari Tunuguntla, Carol A. Wittlieb-Weber, Kathi Kinnett, and Linda Cripe
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Heart Failure ,Muscular Dystrophy, Duchenne ,Duchenne muscular dystrophy ,Cardiomyopathy ,Pediatrics, Perinatology and Child Health ,Humans ,Angiotensin-Converting Enzyme Inhibitors ,Heart ,Original Article ,Cardiomyopathies ,Child ,Cardiology and Cardiovascular Medicine - Abstract
Cardiac disease has emerged as a leading cause of mortality in Duchenne muscular dystrophy in the current era. This survey sought to identify the diagnostic and therapeutic approach to DMD among pediatric cardiologists in Advanced Cardiac Therapies Improving Outcomes Network. Pediatric cardiology providers within ACTION (a multi-center pediatric heart failure learning network) were surveyed regarding their approaches to cardiac care in DMD. Thirty-one providers from 23 centers responded. Cardiac MRI and Holter monitoring are routinely obtained, but the frequency of use and indications for ordering these tests varied widely. Angiotensin converting enzyme inhibitor and aldosterone antagonist are generally initiated prior to onset of systolic dysfunction, while the indications for initiating beta-blocker therapy vary more widely. Seventeen (55%) providers report their center has placed an implantable cardioverter defibrillator in at least 1 DMD patient, while 11 providers (35%) would not place an ICD for primary prevention in a DMD patient. Twenty-three providers (74%) would consider placement of a ventricular assist device (VAD) as destination therapy (n = 23, 74%) and three providers (10%) would consider a VAD only as bridge to transplant. Five providers (16%) would not consider VAD at their institution. Cardiac diagnostic and therapeutic approaches vary among ACTION centers, with notable variation present regarding the use of advanced therapies (ICD and VAD). The network is currently working to harmonize medical practices and optimize clinical care in an era of rapidly evolving outcomes and cardiac/skeletal muscle therapies.
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- 2022
9. ISHLT consensus statement on donor organ acceptability and management in pediatric heart transplantation
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Renata Shih, Karen Lord, Manuela Camino, Jonathan Smith, Angie Scales, Josef Thul, Dimpna C. Albert, Sanjeev Kumar Khulbey, László Ablonczy, Anna Joong, Sharon Chen, Jacqueline M. Smits, Steven J. Kindel, Oliver Miera, Zdenka Reinhardt, Jens Böhmer, Robert G. Weintraub, Matthew Fenton, Jennifer Conway, Anne I. Dipchand, Michael A. McCulloch, Mariska Kemna, Kenneth R. Knecht, Ryan R. Davies, Javier Castro, Richard Kirk, Melanie D. Everitt, Claire Irving, Jonathan N. Johnson, Deipanjan Nandi, Lara Danziger-Isakov, Peta M. A. Alexander, Maryanne R.K. Chrisant, Dipankar Gupta, Luis Garcia-Guereta, Ashwin K. Lal, Gary Beasley, Gretchen B. Chapman, Janet Scheel, Justin Godown, Steve Zangwill, Susan W. Denfield, Antonio Amodeo, Warren A. Zuckerman, Shahnawaz Amdani, Jeffrey G. Gossett, Estela Azeka, Brian Feingold, David N. Rosenthal, Urs Christen, Iki Adachi, Oliver Niesse, Thomas Möller, Jean A Ballweg, Alicia Pérez-Blanco, Martin Schweiger, Ann Punnoose, Bibhuti B. Das, David M. Peng, Daniel Zimpfer, Alison Butler, and Kimberly Y. Lin
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Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,Consensus ,Tissue and Organ Procurement ,Scoring system ,Waiting Lists ,Statement (logic) ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,030230 surgery ,Risk Assessment ,Donor Selection ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Humans ,Cardiopulmonary resuscitation ,Primary graft failure ,Child ,Intensive care medicine ,Transplantation ,business.industry ,Graft Survival ,Tissue Donors ,Donor heart ,Heart Transplantation ,Surgery ,Pediatric heart transplantation ,Waitlist mortality ,Cardiology and Cardiovascular Medicine ,business - Abstract
The number of potential pediatric heart transplant recipients continues to exceed the number of donors, and consequently the waitlist mortality remains significant. Despite this, around 40% of all donated organs are not used and are discarded. This document (62 authors from 53 institutions in 17 countries) evaluates factors responsible for discarding donor hearts and makes recommendations regarding donor heart acceptance. The aim of this statement is to ensure that no usable donor heart is discarded, waitlist mortality is reduced, and post-transplant survival is not adversely impacted.
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- 2020
10. A Validated Model for Sudden Cardiac Death Risk Prediction in Pediatric Hypertrophic Cardiomyopathy
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Andreea Dragulescu, Cedric Manlhiot, Chet R. Villa, Tanya Papaz, Carolyn Y. Ho, Iacopo Olivotto, John J. Parent, Robert G. Weintraub, Alexandre C. Pereira, Seema Mital, Lee N. Benson, Pete F. Aziz, John L. Jefferies, Michelle Michels, Taylor S. Howard, Stephanie J. Nakano, Virginie Beauséjour Ladouceur, Samuel G. Wittekind, Joseph W. Rossano, Letizia Gardin, Elizabeth A. Stephenson, Katey R. Armstrong, Myriam Lafreniere-Roula, Steven D. Colan, Chun-Po Steve Fan, Paul F. Kantor, Seshadri Balaji, Mark W. Russell, Jacob Mathew, Marc E. Richmond, Robert Whitehill, Emilie Jean-St-Michel, Ashwin K. Lal, Beth D. Kaufman, Christopher Semsarian, Jodie Ingles, Jennifer Conway, Heather T. Henderson, Justin Godown, Aamir Jeewa, Anastasia Miron, Ryan J. Butts, and Cardiology
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cardiomyopathies ,Male ,medicine.medical_specialty ,pediatrics ,Adolescent ,Cardiomyopathy ,Risk Assessment ,Sudden cardiac death ,Risk Factors ,Original Research Articles ,Physiology (medical) ,Primary prevention ,Internal medicine ,medicine ,Humans ,Public Health Surveillance ,Young adult ,Child ,Retrospective Studies ,hypertrophic cardiomyopathy ,Models, Statistical ,business.industry ,Age Factors ,Hypertrophic cardiomyopathy ,Reproducibility of Results ,Cardiomyopathy, Hypertrophic ,medicine.disease ,death, sudden, heart ,defibrillators, implantable ,Death, Sudden, Cardiac ,ComputingMethodologies_DOCUMENTANDTEXTPROCESSING ,Cardiology ,Female ,hypertrophy ,Cardiology and Cardiovascular Medicine ,business ,Algorithms - Abstract
Supplemental Digital Content is available in the text., Background: Hypertrophic cardiomyopathy is the leading cause of sudden cardiac death (SCD) in children and young adults. Our objective was to develop and validate a SCD risk prediction model in pediatric hypertrophic cardiomyopathy to guide SCD prevention strategies. Methods: In an international multicenter observational cohort study, phenotype-positive patients with isolated hypertrophic cardiomyopathy 70% prediction accuracy and incorporates risk factors that are unique to pediatric hypertrophic cardiomyopathy. An individualized risk prediction model has the potential to improve the application of clinical practice guidelines and shared decision making for implantable cardioverter defibrillator insertion. Registration: URL: https://www.clinicaltrials.gov; Unique identifier: NCT0403679.
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- 2020
11. Initial multicenter experience with ventricular assist devices in children and young adults with muscular dystrophy: An ACTION registry analysis
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Deipanjan Nandi, Scott R. Auerbach, Neha Bansal, Holger Buchholz, Jennifer Conway, Paul Esteso, Beth D. Kaufman, Ashwin K. Lal, Sabrina P. Law, Angela Lorts, Lindsay J. May, Mary Mehegan, Deepa Mokshagundam, David L.S. Morales, Matthew J. O'Connor, David N. Rosenthal, Muhammad F. Shezad, Kathleen E. Simpson, David L. Sutcliffe, Christina Vanderpluym, Carol A. Wittlieb-Weber, Farhan Zafar, Linda Cripe, and Chet R. Villa
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Pulmonary and Respiratory Medicine ,Transplantation ,Surgery ,Cardiology and Cardiovascular Medicine - Abstract
Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs and in limited numbers. This study sought to describe the outcomes associated with VAD therapy in an unselected population across multiple centers.We examined outcomes of patients with MD and dilated cardiomyopathy implanted with a VAD at Advanced Cardiac Therapies Improving Outcomes Network (ACTION) centers from 9/2012 to 9/2020.A total of 19 VADs were implanted in 18 patients across 12 sites. The majority of patients had dystrophinopathy (66%) and the median age at implant was 17.2 years (range 11.7-29.5). Eleven patients were non-ambulatory (61%) and 6 (33%) were on respiratory support pre-VAD. Five (28%) patients were implanted as a bridge to transplant, 4 of whom survived to transplant. Of 13 patients implanted as bridge to decision or destination therapy, 77% were alive at 1 year and 69% at 2 years. The overall frequencies of positive outcome (transplanted or alive on device) at 1 year and 2 years were 84% and 78%, respectively. Two patients suffered a stroke, 2 developed sepsis, 1 required tracheostomy, and 1 experienced severe right heart failure requiring right-sided VAD.This study demonstrates the potential utility of VAD therapies in patients with muscular dystrophy. Further research is needed to further improve outcomes and better determine which patients may benefit most from VAD therapy in terms of survival and quality of life.
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- 2022
12. Abstract 9544: Physical Activity in Individuals With Hypertrophic Cardiomyopathy: Baseline Data From the Prospective 'Lifestyle and Exercise in Hcm' (live-hcm) Study
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Rachel Lampert, Michael J Ackerman, Bradley S Marino, Matthew Burg, Barbara Ainsworth, Lisa Salberg, Maite Tome, Seshadri Balaji, Carolyn Ho, Richard J Czosek, Cynthia James, Michael Emery, Robert Cooper, Martijn Bos, Jeffrey B Geske, Lubna Choudhury, Susan P Etheridge, Ashwin K Lal, James S Ware, Anne M Dubin, Belinda Gray, Silvana M Molossi, Martin S Maron, Sara Saberi, Daniel L Jacoby, Benjamin W Eidem, Kimberly Harmon, Kevin Hall, Brian Olshansky, Mark S Link, Ian Law, Elizabeth V Saarel, N A Estes, Barry Maron, David S Cannom, Fangyong Li, Kaylie Briske, Laura Simone, Cheryl Barth, Maryann Concannon, and Sharlene Day
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Physiology (medical) ,Cardiology and Cardiovascular Medicine - Abstract
Introduction: Safety of vigorous exercise for individuals with appropriately-treated HCM remains debated. Physical activity practices in this population have not been described. The objective of this report is to describe physical activity pattern in HCM patients. Long term follow up which is ongoing will determine safety of vigorous exercise. Methods: The NIH-funded LIVE-HCM study prospectively enrolled individuals age 8-60 years with clinically overt HCM or patients with a positive genetic test for sarcomeric HCM. Participants (or parents, for children) answered surveys describing activity patterns. Vigorous exercise was defined as > 6 METS for > 60 hours per year. Clinical and demographic data were derived from medical record review. Results: Among 1798 participants, 745 (41%) reported exercising vigorously, including 296 competitive athletes, with 59 at high-school or college varsity level, 770 (43%) were exercising at moderate, and 283 (16%) at low levels. Of those working, 6% described jobs more active than walking, including 16 in protective services, and 11 in sports/coaching. Clinical/demographic data including imaging characteristics and treatments, are shown in Table, and were mostly similar amongst the exercise-groups. Conclusions: Many patients with HCM are engaged in vigorous exercise including competitive athletics. Prospective follow-up of this cohort is ongoing. Comparison of arrhythmic outcomes outcomes in vigorous exercisers vs moderate/low level exercisers will help determine the safety of exercise in HCM patients.
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- 2021
13. Abstract 10725: Prevalence and Impact of Recurrent Rejection on Graft Loss in Pediatric Heart Transplant Recipients: A Pediatric Heart Transplant Society Multi-Institutional Analysis
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Shahnawaz Amdani, James K Kirklin, Ryan Cantor, Devin Koehl, Ashwin K Lal, Peter Chau, Valerie Curren, Jonathan Edelson, John J Parent, Hannah Bostdorff, Ali L BURNETTE, and Jacqueline M Lamour
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Physiology (medical) ,Cardiology and Cardiovascular Medicine - Abstract
Introduction: Rejection remains a significant cause of graft loss after heart transplantation. The current prevalence of recurrent rejection and its impact on graft loss after pediatric heart transplantation is unknown. Methods: All pediatric heart transplants between 1/2000-6/2020 in the Pediatric Heart Transplant Society database were included. Rejection was defined as a clinical event determined by biopsy, echocardiogram or clinical findings that resulted in augmentation of immunosuppression. Recurrent rejection was defined as ≥ 2 episodes of rejection after heart transplant. Freedom from recurrent rejection in the early (2000-2009) and current (2010-2020) era were compared. Multiphase parametric hazard modeling was utilized to understand the effects of each additional episode of rejection on graft loss in addition to identifying other risk factors. Results: Of 6342 heart transplants during the study period, 3921 (62%) had 0, 1386 (21%) had 1 and 1035 (17%) had ≥2 rejection episodes. In the current era, recurrent rejection is less frequent [468/4206 (11.1%) vs. 567/2136 (26.5%); p Conclusions: Currently, recurrent rejection is experienced by 1 in 10 pediatric HT recipients. Each additional episode of rejection post-HT confers an increasing risk for graft loss. Identifying risk factors for recurrent rejection is paramount to improving graft longevity.
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- 2021
14. Significant Variation in Exercise Recommendations for Youth With Cardiomyopathies or Fontan Circulation
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Michael Khoury, Jennifer Conway, Angela Lorts, Ashwin K. Lal, Neha Bansal, Samuel G. Wittekind, David N. Rosenthal, and Danielle S. Burstein
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Heart Defects, Congenital ,Heart Failure ,medicine.medical_specialty ,Exercise Tolerance ,business.industry ,Fontan Procedure ,medicine.disease ,Fontan circulation ,Variation (linguistics) ,Internal medicine ,Heart failure ,Exercise Test ,Cardiology ,medicine ,Humans ,Cardiomyopathies ,Cardiology and Cardiovascular Medicine ,business ,Exercise - Published
- 2021
15. Diagnosis and Management of Myocarditis in Children
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Justin Godown, Yuk M. Law, Sharon Chen, Ashwin K. Lal, Lars Grosse-Wortmann, Leslie T. Cooper, Shriprasad R. Deshpande, Daniela Cihakova, Jeffrey A. Towbin, and Joshua D Robinson
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medicine.medical_specialty ,Myocarditis ,Heart disease ,Statement (logic) ,Biopsy ,medicine.medical_treatment ,Clinical Decision-Making ,Autopsy ,030204 cardiovascular system & hematology ,Multimodal Imaging ,030218 nuclear medicine & medical imaging ,03 medical and health sciences ,0302 clinical medicine ,Cardiac magnetic resonance imaging ,Physiology (medical) ,medicine ,Animals ,Humans ,Child ,Intensive care medicine ,Heart transplantation ,medicine.diagnostic_test ,business.industry ,Cardiogenic shock ,Disease Management ,Prognosis ,medicine.disease ,Combined Modality Therapy ,Disease Models, Animal ,Treatment Outcome ,Heart failure ,Disease Susceptibility ,Symptom Assessment ,Cardiology and Cardiovascular Medicine ,business - Abstract
Myocarditis remains a clinical challenge in pediatrics. Originally, it was recognized at autopsy before the application of endomyocardial biopsy, which led to a histopathology-based diagnosis such as in the Dallas criteria. Given the invasive and low-sensitivity nature of endomyocardial biopsy, its diagnostic focus shifted to a reliance on clinical suspicion. With the advances of cardiac magnetic resonance, an examination of the whole heart in vivo has gained acceptance in the pursuit of a diagnosis of myocarditis. The presentation may vary from minimal symptoms to heart failure, life-threatening arrhythmias, or cardiogenic shock. Outcomes span full resolution to chronic heart failure and the need for heart transplantation with inadequate clues to predict the disease trajectory. The American Heart Association commissioned this writing group to explore the current knowledge and management within the field of pediatric myocarditis. This statement highlights advances in our understanding of the immunopathogenesis, new and shifting dominant pathogeneses, modern laboratory testing, and use of mechanical circulatory support, with a special emphasis on innovations in cardiac magnetic resonance imaging. Despite these strides forward, we struggle without a universally accepted definition of myocarditis, which impedes progress in disease-targeted therapy.
- Published
- 2021
16. Impact of Genetic Testing for Cardiomyopathy on Emotional Well-Being and Family Dynamics: A Study of Parents and Adolescents
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Aijin Wang, Maya Sabatello, Paul F. Kantor, Stephanie M. Ware, Julia Wynn, Min Qian, Priyanka Ahimaz, Joseph W. Rossano, John J. Parent, Paul S. Appelbaum, Ashwin K. Lal, Ashley Parrott, Wendy K. Chung, Linda J. Addonizio, Kathryn C. Chatfield, Teresa M. Lee, Erin M. Miller, and Lisa Yue
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Male ,Parents ,Adolescent ,medicine.diagnostic_test ,Emotions ,Cardiomyopathy ,General Medicine ,medicine.disease ,Article ,Emotional well-being ,Family dynamics ,Surveys and Questionnaires ,Risk stratification ,medicine ,Humans ,Female ,Genetic Testing ,Family history ,Cardiomyopathies ,Child ,Psychology ,Clinical psychology ,Genetic testing - Abstract
Background: Genetic testing is indicated for children with a personal or family history of hereditary cardiomyopathy to determine appropriate management and inform risk stratification for family members. The implications of a positive genetic result for children can potentially impact emotional well-being. Given the nuances of cardiomyopathy genetic testing for minors, this study aimed to understand how parents involve their children in the testing process and investigate the impact of genetic results on family dynamics. Methods: A survey was distributed to participants recruited from the Children’s Cardiomyopathy Foundation and 7 North American sites in the Pediatric Cardiomyopathy Registry. The survey explored adolescent and parent participants’ emotions upon receiving their/their child’s genetic results, parent-child result communication and its impact on family functionality, using the McMaster Family Assessment Device. Results: One hundred sixty-two parents of minors and 48 adolescents who were offered genetic testing for a personal or family history of cardiomyopathy completed the survey. Parents whose child had cardiomyopathy were more likely to disclose positive diagnostic genetic results to their child ( P =0.014). Parents with unaffected children and positive predictive testing results were more likely to experience negative emotions about the result ( P ≤0.001) but also had better family functioning scores than those with negative predictive results ( P =0.019). Most adolescents preferred results communicated directly to the child, but parents were divided about whether their child’s result should first be released to them or their child. Conclusions: These findings have important considerations for how providers structure genetic services for adolescents and facilitate discussion between parents and their children about results.
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- 2021
17. Genetic Causes of Cardiomyopathy in Children: First Results From the Pediatric Cardiomyopathy Genes Study
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Charles E. Canter, Lynn A. Sleeper, Lisa J. Martin, Erin M. Miller, Joseph W. Rossano, Stephanie M. Ware, Debra A. Dodd, Daphne T. Hsu, James D. Wilkinson, Teresa Lee, Ling Shi, Jason D. Czachor, Linda J. Addonizio, Steven D. Colan, Steven A. Webber, Elfriede Pahl, Arthi Sridhar, Paolo Rusconi, Wendy K. Chung, John L. Jefferies, Steven E. Lipshultz, Bruce J. Aronow, Muhammad Tariq, Phillip J. Dexheimer, Jeffrey A. Schubert, Ashwin K. Lal, Melanie D. Everitt, Hiedy Razoky, Paul F. Kantor, Jeffrey A. Towbin, and Surbhi Bhatnagar
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Male ,Pediatrics ,medicine.medical_specialty ,Adolescent ,Cardiomyopathy ,heart failure ,030204 cardiovascular system & hematology ,03 medical and health sciences ,0302 clinical medicine ,Exome Sequencing ,Genetics ,medicine ,Humans ,Genetic Predisposition to Disease ,Genetic Testing ,Registries ,molecular ,Family history ,Child ,Exome sequencing ,Idiopathic Cardiomyopathy ,Retrospective Studies ,Original Research ,030304 developmental biology ,Genetic testing ,0303 health sciences ,medicine.diagnostic_test ,business.industry ,Genetic heterogeneity ,Hypertrophic cardiomyopathy ,Restrictive cardiomyopathy ,Correction ,medicine.disease ,infant ,United States ,Survival Rate ,Child, Preschool ,Female ,Morbidity ,mutation ,Cardiomyopathies ,Cardiology and Cardiovascular Medicine ,business ,exome - Abstract
Background Pediatric cardiomyopathy is a genetically heterogeneous disease with substantial morbidity and mortality. Current guidelines recommend genetic testing in children with hypertrophic, dilated, or restrictive cardiomyopathy, but practice variations exist. Robust data on clinical testing practices and diagnostic yield in children are lacking. This study aimed to identify the genetic causes of cardiomyopathy in children and to investigate clinical genetic testing practices. Methods and Results Children with familial or idiopathic cardiomyopathy were enrolled from 14 institutions in North America. Probands underwent exome sequencing. Rare sequence variants in 37 known cardiomyopathy genes were assessed for pathogenicity using consensus clinical interpretation guidelines. Of the 152 enrolled probands, 41% had a family history of cardiomyopathy. Of 81 (53%) who had undergone clinical genetic testing for cardiomyopathy before enrollment, 39 (48%) had a positive result. Genetic testing rates varied from 0% to 97% between sites. A positive family history and hypertrophic cardiomyopathy subtype were associated with increased likelihood of genetic testing ( P =0.005 and P =0.03, respectively). A molecular cause was identified in an additional 21% of the 63 children who did not undergo clinical testing, with positive results identified in both familial and idiopathic cases and across all phenotypic subtypes. Conclusions A definitive molecular genetic diagnosis can be made in a substantial proportion of children for whom the cause and heritable nature of their cardiomyopathy was previously unknown. Practice variations in genetic testing are great and should be reduced. Improvements can be made in comprehensive cardiac screening and predictive genetic testing in first‐degree relatives. Overall, our results support use of routine genetic testing in cases of both familial and idiopathic cardiomyopathy. Registration URL: https://www.clinicaltrials.gov ; Unique identifier: NCT01873963.
- Published
- 2021
18. Outcomes of Myocarditis in Patients with Normal Left Ventricular Systolic Function on Admission
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Spencer B. Barfuss, Kenneth R. Knecht, Adriana Prada-Ruiz, Ashwin K. Lal, and Ryan J. Butts
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Male ,Chest Pain ,medicine.medical_specialty ,Cardiotonic Agents ,Myocarditis ,Adolescent ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,Chest pain ,Ventricular Function, Left ,Electrocardiography ,03 medical and health sciences ,Extracorporeal Membrane Oxygenation ,0302 clinical medicine ,Interquartile range ,Internal medicine ,medicine ,Extracorporeal membrane oxygenation ,Humans ,Retrospective Studies ,Ejection fraction ,business.industry ,ST elevation ,Troponin I ,medicine.disease ,Respiration, Artificial ,Treatment Outcome ,030228 respiratory system ,Heart failure ,Pediatrics, Perinatology and Child Health ,Cardiology ,Female ,medicine.symptom ,Cardiology and Cardiovascular Medicine ,business ,Anti-Arrhythmia Agents ,Myopericarditis - Abstract
The objective of this study was to describe a cohort of patients with clinical myocarditis and normal left ventricular (LV) systolic function on admission. A retrospective chart review at seven tertiary pediatric hospitals identified patients aged
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- 2019
19. Use of advanced heart failure therapies in Duchenne muscular dystrophy
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Joshua M. Friedland-Little, M.J. Bock, Jennifer Conway, Chet R. Villa, Ryan J. Butts, Kenneth R. Knecht, Kurt R. Schumacher, Michael A. McCulloch, Shawn C. West, Shriprasad R. Deshpande, S. Law, Ashwin K. Lal, Jonathan N. Johnson, K. Gambetta, I.D. Lytrivi, Carol A. Wittlieb-Weber, and David R. Weber
- Subjects
Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,medicine.medical_treatment ,Duchenne muscular dystrophy ,Population ,030204 cardiovascular system & hematology ,Ventricular tachycardia ,Article ,03 medical and health sciences ,0302 clinical medicine ,030225 pediatrics ,Internal medicine ,medicine ,education ,Heart transplantation ,Transplantation ,education.field_of_study ,business.industry ,Retrospective cohort study ,medicine.disease ,Implantable cardioverter-defibrillator ,Heart failure ,Ventricular assist device ,Pediatrics, Perinatology and Child Health ,Cardiology ,Surgery ,Cardiology and Cardiovascular Medicine ,business - Abstract
Background As survival and neuromuscular function in Duchenne Muscular Dystrophy (DMD) improve with glucocorticoid therapy and respiratory advances, the proportion of cardiac deaths is increasing. Little is known about the use and outcomes of advanced heart failure (HF) therapies in this population. Methods A retrospective cohort study of 436 males with DMD was performed, from January 1, 2005-January 1, 2018, with the primary outcome being use of advanced HF therapies including: implantable cardioverter defibrillator (ICD), left ventricular assist device (LVAD), and heart transplantation (HTX). Results Nine subjects had an ICD placed, 2 of whom (22.2%) had appropriate shocks for ventricular tachycardia; 1 and 968 days after implant, and all of whom were alive at last follow-up; median 18 (IQR: 12.5–25.5) months from implant. Four subjects had a LVAD implanted with post-LVAD survival of 75% at 1 year; 2 remaining on support and 1 undergoing HTX. One subject was bridged to HTX with ICD and LVAD and was alive at last follow-up, 53 months after HTX. Conclusion Advanced HF therapies may be used effectively in select subjects with DMD. Further studies are needed to better understand risk stratification for ICD use and optimal candidacy for LVAD implantation and HTX, with hopes of improving cardiac outcomes.
- Published
- 2019
20. Usefulness of Left Ventricular Myocardial Deformation in Children Hospitalized for Acute Myocarditis who Develop Arrhythmias
- Author
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Angela P. Presson, Aaron W. Eckhauser, Cathleen R. Pruitt, Zhining Ou, Mary Niu, Thomas A. Miller, Shaji C. Menon, and Ashwin K. Lal
- Subjects
Male ,medicine.medical_specialty ,Myocarditis ,Longitudinal strain ,Adolescent ,Heart block ,Speckle tracking echocardiography ,030204 cardiovascular system & hematology ,030218 nuclear medicine & medical imaging ,03 medical and health sciences ,Basal (phylogenetics) ,Ventricular Dysfunction, Left ,0302 clinical medicine ,Risk Factors ,Internal medicine ,medicine ,Tachycardia, Supraventricular ,Circumferential strain ,Humans ,cardiovascular diseases ,Child ,Retrospective Studies ,business.industry ,Infant ,Arrhythmias, Cardiac ,medicine.disease ,Hospitalization ,Acute myocarditis ,Heart Block ,Child, Preschool ,Ventricular fibrillation ,Ventricular Fibrillation ,cardiovascular system ,Cardiology ,Tachycardia, Ventricular ,Female ,Cardiology and Cardiovascular Medicine ,business ,Anti-Arrhythmia Agents - Abstract
Cardiac arrhythmias occur in 3-40% of patients with acute myocarditis and cause significant morbidity and mortality. Myocardial injury also results in abnormal myocardial deformation. The relationship between left ventricular (LV) deformation, measured by two-dimensional speckle tracking echocardiography (2D-STE), and arrhythmia in pediatric myocarditis is unknown. We evaluated the association between 2D-STE and arrhythmias in children hospitalized with acute myocarditis. We reviewed patients ≤ 18 years hospitalized for acute myocarditis from 2008 to 2018. Arrhythmias were defined as 1) non-sustained or sustained ventricular tachycardia or ventricular fibrillation, 2) sustained supraventricular tachycardia (SVT), 3) high-grade or complete heart block, and 4) any arrhythmia treated with an antiarrhythmic medication. Systolic LV strain values (including LV global longitudinal strain (GLS), global circumferential strain (GCS), and six segments of LV regional long axis strain) were obtained from initial echocardiograms during hospitalization. Of 66 patients hospitalized, 23 (35%) had arrhythmias. SVT was the predominant arrhythmia (74%). Global and regional strain indices were reduced in the arrhythmia group: LV GLS [-8.9 (IQR -13.6, -6.1) vs. -13.7 (IQR -16.9, -9.7), p = 0.038]; basal inferior/septal [-10.7 (IQR -15.5, -7.8) vs. -16.4 (IQR -18, -11.8), p = 0.009]; basal anterior/lateral [-7.1 (IQR -12.8, -4.7) vs. -9.4 (IQR -16.7, -7.4), p = 0.025]; and mid inferior/septal segments [-9 (IQR -13, -7.7) vs. -14.1 (IQR -22.5, -10.7), p = 0.007]. After controlling for age, reductions in GLS and segmental strain in the two basal and two mid-segments were associated with increased arrhythmia occurrence (p0.05). Our findings suggest that echocardiographic LV deformation by 2D-STE may be useful in identifying pediatric patients with acute myocarditis at risk for arrhythmias.
- Published
- 2021
21. Diversity of Dystrophin Gene Mutations and Disease Progression in a Contemporary Cohort of Duchenne Muscular Dystrophy
- Author
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Katheryn E, Gambetta, Michael A, McCulloch, Ashwin K, Lal, Kenneth, Knecht, Ryan J, Butts, Chet R, Villa, Jonathan N, Johnson, Jennifer, Conway, Matthew J, Bock, Kurt R, Schumacher, Sabrina P, Law, Joshua M, Friedland-Little, Shriprasad R, Deshpande, Shawn C, West, Irene D, Lytrivi, and Carol A, Wittlieb-Weber
- Subjects
Adult ,Cohort Studies ,Dystrophin ,Male ,Muscular Dystrophy, Duchenne ,Adolescent ,Mutation ,Disease Progression ,Humans ,Retrospective Studies - Abstract
Abnormal dystrophin production due to mutations in the dystrophin gene causes Duchenne Muscular Dystrophy (DMD). Cases demonstrate considerable genetic and disease progression variability. It is unclear if specific gene mutations are prognostic of outcomes in this population. We conducted a retrospective cohort study of DMD patients followed at 17 centers across the USA and Canada from 2005 to 2015 with goal of understanding the genetic variability of DMD and its impact on clinical outcomes. Cumulative incidence of clinically relevant outcomes was stratified by genetic mutation type, exon mutation location, and extent of exon deletion. Of 436 males with DMD, 324 (74.3%) underwent genetic testing. Deletions were the most common mutation type (256, 79%), followed by point mutations (45, 13.9%) and duplications (23, 7.1%). There were 131 combinations of mutations with most mutations located along exons 45 to 52. The number of exons deleted varied between 1 and 52 with a median of 3 exons deleted (IQR 1-6). Subjects with mutations starting at exon positions 40-54 had a later onset of arrhythmias occurring at median age 25 years (95% CI 18-∞), p = 0.01. Loss of ambulation occurred later at median age of 13 years (95% CI 12-15) in subjects with mutations that started between exons 55-79, p = 0.01. There was no association between mutation type or location and onset of cardiac dysfunction. We report the genetic variability in DMD and its association with timing of clinical outcomes. Genetic modifiers may explain some phenotypic variability.
- Published
- 2021
22. Review of the discard and/or refusal rate of offered donor hearts to pediatric waitlisted candidates
- Author
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László Ablonczy, Janet Scheel, Anne I. Dipchand, Ryan R. Davies, Richard Kirk, Dipankar Gupta, Deipanjan Nandi, Melanie D. Everitt, Sharon Chen, Ashwin K. Lal, Oliver Miera, Javier Castro, and Martin Schweiger
- Subjects
medicine.medical_specialty ,Adolescent ,Waiting Lists ,Pediatric transplantation ,030232 urology & nephrology ,Geographic variation ,030230 surgery ,Donor Selection ,Refusal rate ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Humans ,Child ,Intensive care medicine ,Transplantation ,Pediatric donor ,business.industry ,Infant, Newborn ,Infant ,Child, Preschool ,Health Care Surveys ,Pediatrics, Perinatology and Child Health ,Heart Transplantation ,Pediatric heart transplantation ,Heart donor ,business - Abstract
We aimed to review current literature on the discard rate of donor hearts offered to pediatric recipients and assess geographical differences. Consequences and ways to reduce the discard rate are discussed. A systemic review on published literature on pediatric transplantation published in English since 2010 was undertaken. Additionally, a survey was sent to international OPOs with the goal of incorporating responses from around the world providing a more global picture. Based on the literature review and survey, there is a remarkably wide range of discard and/or refusal for pediatric hearts offered for transplant, ranging between 18% and 57% with great geographic variation. The data suggest that that the overall refusal rate may have decreased over the last decade. Reasons for organ discard were difficult to identify from the available data. Although the refusal rate of pediatric donor hearts seems to be lower compared to that reported in adults, it is still as high as 57% with geographic variation.
- Published
- 2020
23. Implications of Left Ventricular Dysfunction at Presentation for Infants with Coarctation of the Aorta
- Author
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Kimberly M. Molina, Nelangi M. Pinto, Ashwin K. Lal, L. LuAnn Minich, Aaron W. Eckhauser, Carol Mcfarland, Lindsay J. May, Zhining Ou, Angela P. Presson, Dongngan T. Truong, and Phillip T. Burch
- Subjects
Moderate to severe ,Male ,medicine.medical_specialty ,Time Factors ,medicine.medical_treatment ,Coarctation of the aorta ,Aortic Coarctation ,Ventricular Function, Left ,Ventricular Dysfunction, Left ,Postoperative Complications ,Internal medicine ,Lv dysfunction ,medicine ,Prevalence ,Humans ,Retrospective Studies ,Mechanical ventilation ,business.industry ,Infant ,Retrospective cohort study ,Stroke Volume ,Tertiary care hospital ,medicine.disease ,Respiration, Artificial ,Hospitalization ,Intensive Care Units ,Pediatrics, Perinatology and Child Health ,Cardiology ,Female ,Presentation (obstetrics) ,Cardiology and Cardiovascular Medicine ,Index hospitalization ,business ,Vascular Surgical Procedures - Abstract
Infants with aortic coarctation may present with left ventricular (LV) dysfunction which may complicate the postoperative course and lead to increased healthcare costs. We aimed to define the prevalence of moderate to severe left ventricular (LV) systolic dysfunction, evaluate time to recovery, and compare health care costs. Single-center retrospective cohort study at a tertiary care hospital was conducted. Infants 6 months old at diagnosis with aortic coarctation were identified using surgical codes for coarctation repair between January 2010 and May 2018. Moderate to severe dysfunction was defined as ejection fraction (EF) 40%. Of 160 infants studied, 18 (11%) had moderate to severe LV dysfunction at presentation. Compared to those with better LV function, infants with moderate to severe LV dysfunction were older at presentation (12 vs. 6 days, p = 0.004), had more postoperative cardiac intensive care unit (ICU) days (5 vs. 3, p 0.001), and more ventilator days (3.5 vs. 1, p 0.001). The median time to normal LV EF (≥ 55%) was 6 days postoperatively (range 1-230 days). Infants presenting with moderate to severe LV dysfunction had higher index hospitalization costs ($90,560 vs. $59,968, p = 0.02), but no difference in cost of medical follow-up for the first year following discharge ($3,078 vs. $2,568, p = 0.46). In the current era, 10% of infants with coarctation present with moderate to severe LV dysfunction that typically recovers. Those with moderate to severe dysfunction had longer duration of mechanical ventilation and postoperative cardiac ICU stays, likely driving higher costs of index hospitalization.
- Published
- 2020
24. Risk Factors for Cardiac and Non‑cardiac Causes of Death in Males with Duchenne Muscular Dystrophy
- Author
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Chet R. Villa, Kenneth R. Knecht, Shriprasad R. Deshpande, K. Gambetta, Kurt R. Schumacher, David R. Weber, M.J. Bock, Shawn C. West, Chentel Cunningham, Jennifer Conway, Sabrina P. Law, Carol A. Wittlieb-Weber, Michael A. McCulloch, Irene D. Lytrivi, Jonathan N. Johnson, Joshua M. Friedland-Little, Ashwin K. Lal, and Ryan J. Butts
- Subjects
Adult ,Male ,medicine.medical_specialty ,Adolescent ,Duchenne muscular dystrophy ,030204 cardiovascular system & hematology ,Article ,Sudden cardiac death ,03 medical and health sciences ,Young Adult ,0302 clinical medicine ,Risk Factors ,030225 pediatrics ,Internal medicine ,Cause of Death ,medicine ,Humans ,Retrospective Studies ,Ejection fraction ,business.industry ,Retrospective cohort study ,Vascular surgery ,medicine.disease ,Cardiac surgery ,Muscular Dystrophy, Duchenne ,Heart failure ,Pediatrics, Perinatology and Child Health ,Cohort ,Cardiology and Cardiovascular Medicine ,business ,Cardiomyopathies - Abstract
As survival and neuromuscular function in Duchenne muscular dystrophy (DMD) have improved with glucocorticoid (GC) therapy and ventilatory support, cardiac deaths are increasing. Little is known about risk factors for cardiac and non-cardiac causes of death in DMD. A multi-center retrospective cohort study of 408 males with DMD, followed from January 1, 2005 to December 31, 2015, was conducted to identify risk factors for death. Those dying of cardiac causes were compared to those dying of non-cardiac causes and to those alive at study end. There were 29 (7.1%) deaths at a median age of 19.5 (IQR: 16.9–24.6) years; 8 (27.6%) cardiac, and 21 non-cardiac. Those living were younger [14.9 (IQR: 11.0–19.1) years] than those dying of cardiac [18 (IQR 15.5–24) years, p = 0.03] and non-cardiac [19 (IQR: 16.5–23) years, p = 0.002] causes. GC use was lower for those dying of cardiac causes compared to those living [2/8 (25%) vs. 304/378 (80.4%), p = 0.001]. Last ejection fraction prior to death/study end was lower for those dying of cardiac causes compared to those living (37.5% ± 12.8 vs. 54.5% ± 10.8, p = 0.01) but not compared to those dying of non-cardiac causes (37.5% ± 12.8 vs. 41.2% ± 19.3, p = 0.58). In a large DMD cohort, approximately 30% of deaths were cardiac. Lack of GC use was associated with cardiac causes of death, while systolic dysfunction was associated with death from any cause. Further work is needed to ensure guideline adherence and to define optimal management of systolic dysfunction in males with DMD with hopes of extending survival.
- Published
- 2020
25. Thrombotic microangiopathy following heart transplant in pediatric Danon disease
- Author
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Kristi Glotzbach, Ashwin K. Lal, Shaji C. Menon, Eric R. Griffiths, and Kevin Hummel
- Subjects
Hemolytic anemia ,medicine.medical_specialty ,Thrombotic microangiopathy ,030232 urology & nephrology ,030230 surgery ,Brain herniation ,Cerebral edema ,Angiopathy ,03 medical and health sciences ,0302 clinical medicine ,Fatal Outcome ,Postoperative Complications ,Internal medicine ,medicine ,Humans ,Danon disease ,Child ,Heart Failure ,Transplantation ,business.industry ,Thrombotic Microangiopathies ,medicine.disease ,Tacrolimus ,Glycogen Storage Disease Type IIb ,Schistocyte ,Pediatrics, Perinatology and Child Health ,Cardiology ,Heart Transplantation ,Female ,business - Abstract
This case describes an uncommon acute complication of diffuse thrombotic angiopathy and associated aHUS/TTP in an 11-year-old girl with Danon disease who underwent orthotopic heart transplant. Shortly after transplant, despite an uncomplicated operative course, the patient developed severe kidney injury and progressive altered mental status, culminating in cerebral edema, brain herniation, and death. She had received a single dose of tacrolimus (FK506) and a single dose of antithymocyte globulin. Sources of progressive somnolence, including oversedation from impaired renal clearance of opiates, and severe myopathy as has been previously described in Danon disease, were ruled out, and the patient continued to decline. Initial brain CT scan early after transplant showed no signs of cerebral edema, but repeat CT indicated severe cerebral edema. Based on autopsy, diffuse thrombotic angiopathy, with signs of hemolytic anemia with schistocytes, was likely responsible for her deterioration in the broader condition of aHUS/TTP.
- Published
- 2020
26. Development and validation of a major adverse transplant event (MATE) score to predict late graft loss in pediatric heart transplantation
- Author
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Christopher S. Almond, Lingyao Yang, Seth A. Hollander, Matthew Fenton, Chesney Castleberry, Joseph W. Rossano, Manisha Desai, Doff B. McElhinney, Elfriede Pahl, Melanie D. Everitt, Scott R. Auerbach, Helena Hoen, Kevin P. Daly, Ashwin K. Lal, Elizabeth Pruitt, and David N. Rosenthal
- Subjects
Graft Rejection ,Male ,Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,030230 surgery ,Cohort Studies ,03 medical and health sciences ,Postoperative Complications ,0302 clinical medicine ,Predictive Value of Tests ,Internal medicine ,medicine ,Humans ,Child ,Proportional Hazards Models ,Randomized Controlled Trials as Topic ,Immunosuppression Therapy ,Heart transplantation ,Transplantation ,Proportional hazards model ,business.industry ,Surrogate endpoint ,Graft Survival ,Hazard ratio ,Infant ,Reproducibility of Results ,Immunosuppression ,medicine.disease ,Surgery ,Child, Preschool ,Sample Size ,Heart failure ,Cohort ,Heart Transplantation ,Female ,Cardiology and Cardiovascular Medicine ,business ,Biomarkers ,Kidney disease - Abstract
Background There is inadequate power to perform a valid clinical trial in pediatric heart transplantation (HT) using a conventional end-point, because the disease is rare and hard end-points, such as death or graft loss, are infrequent. We sought to develop and validate a surrogate end-point involving the cumulative burden of post-transplant complications to predict death/graft loss to power a randomized clinical trial of maintenance immunosuppression in pediatric HT. Methods Pediatric Heart Transplant Study (PHTS) data were used to identify all children who underwent an isolated orthotopic HT between 2005 and 2014 who survived to 6 months post-HT. A time-varying Cox model was used to develop and evaluate a surrogate end-point comprised of 6 major adverse transplant events (MATEs) (acute cellular rejection [ACR], antibody-mediated rejection [AMR], infection, cardiac allograft vasculopathy [CAV], post-transplant lymphoproliferative disease [PTLD] and chronic kidney disease [CKD]) occurring between 6 and 36 months, where individual events were defined according to international guidelines. Two thirds of the study cohort was used for score development, and one third of the cohort was used to test the score. Results Among 2,118 children, 6.4% underwent graft loss between 6 and 36 months post-HT, whereas 39% developed CKD, 34% ACR, 34% infection, 9% AMR, 4% CAV and 2% PTLD. The best predictive score involved a simple MATE score sum, yielding a concordance probability estimate (CPE) statistic of 0.74. Whereas the power to detect non-inferiority (NI), assuming the NI hazard ratio of 1.45 in graft survival was 10% (assuming 200 subjects and 6% graft loss rate), the power to detect NI assuming a 2-point non-inferiority margin was >85% using the MATE score. Conclusion The MATE score reflects the cumulative burden of MATEs and has acceptable prediction characteristics for death/graft loss post-HT. The MATE score may be useful as a surrogate end-point to power a clinical trial in pediatric HT.
- Published
- 2018
27. Impact of Insurance and Race on Pediatric Heart Transplant Outcomes- An Analysis of the Pediatric Heart Transplant Society (PHTS) Registry
- Author
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Devin Koehl, Jennifer Conway, Waldemar F. Carlo, James K. Kirklin, Alfred Asante-Korang, Simon Urschel, N. Deal, Neha Bansal, J.A. Kleinmahon, R.S. Cantor, Ashwin K. Lal, and Melanie D. Everitt
- Subjects
Pulmonary and Respiratory Medicine ,Heart transplantation ,Transplantation ,medicine.medical_specialty ,business.industry ,medicine.medical_treatment ,Hemodynamics ,Malignancy ,medicine.disease ,Log-rank test ,Race (biology) ,Internal medicine ,Insurance status ,medicine ,Surgery ,Racial bias ,Cardiology and Cardiovascular Medicine ,business ,Socioeconomic status - Abstract
Purpose Socioeconomic disparities negatively impacts outcomes in heart transplantation (HTx). However, the impact of insurance status and the social construct of race in pediatric patients has not been well studied. Methods The Pediatric Heart Transplant Society registry was queried between January 1, 2000- December 31, 2019. Patients were stratified by insurance status: US government (USG), US private (USP) and Single Payer (SP, UK & Canada) and race (black, white, other). Patients with a selection of more than one race were classified as other. Outcomes after HTx were assessed by Kaplan Meier log rank test including graft survival, time to first cardiac allograft vasculopathy (CAV), rejection, hemodynamic compromised (HC) rejection, and malignancy. Results 5879 children underwent HTx with insurance status as follows: 2800 USG (48%), 2431 USP (41%) and 648 SP (11%). Race breakdown included: 4086 white, 1052 black and 936 other. At 10 years post-transplant, graft survival (65.8% USG, 72.6% USP, 77% SP, p= .0005), time to first CAV (73.5% USG, 77.7% USP, 94.6% SP, p Conclusion In this multinational analysis of pediatric HTx patients, USG was associated with worse post-HTx outcomes. When stratified by race, graft survival was worse in black patients with USG and USP insurance. Both insurance and the social construct of race contribute to worse outcomes post heart transplant and may represent disparity in access to care and other racial bias. These results highlight the importance of further work in this area.
- Published
- 2021
28. Sudden Cardiac Death and ICD Use in Rasopathy-Associated Hypertrophic Cardiomyopathy
- Author
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Joseph W. Rossano, Stephanie J. Nakano, Paul F. Kantor, Katey R. Armstrong, Jennifer Conway, Pete F. Aziz, Ann M. Lynch, B. Seshadri, Robert Whitehill, R. Weintraub, Heather T. Henderson, L. Gardin, S. Ahuja, A. Miron, Marc E. Richmond, Seema Mital, Beth D. Kaufman, Ashwin K. Lal, Taylor S. Howard, John J. Parent, Steve Colan, Chet R. Villa, Justin Godown, Ryan J. Butts, and Mark W. Russell
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,medicine.medical_specialty ,business.industry ,Incidence (epidemiology) ,Hypertrophic cardiomyopathy ,Retrospective cohort study ,Sudden cardiac arrest ,macromolecular substances ,RASopathy ,medicine.disease ,Sudden death ,Sudden cardiac death ,Internal medicine ,cardiovascular system ,Cardiology ,Medicine ,Surgery ,Cumulative incidence ,cardiovascular diseases ,medicine.symptom ,Cardiology and Cardiovascular Medicine ,business - Abstract
Purpose The incidence and risk factors for sudden cardiac death in Rasopathy patients with hypertrophic cardiomyopathy (Ras-HCM) are not well understood. We compared the cumulative incidence of sudden death and ICD therapy in Ras-HCM compared to primary HCM. Methods Phenotype-positive pediatric patients with Ras-HCM (n=157) and primary HCM (n=720) were included in this 22-center retrospective cohort study. The primary outcome was 5-year freedom from sudden death events, defined as a composite of sudden death, resuscitated sudden cardiac arrest, and aborted events i.e. appropriate shock from a primary prevention ICD, estimated using the Kaplan Meier method. Results There were 17 deaths in Ras-HCM patients - 8 sudden death, 9 from noncardiac causes. The 5-year freedom from sudden death events was higher in Ras-HCM than in primary HCM (97.2% vs 93.8% respectively, p=0.039) (Figure 1A). The median age at diagnosis was lower in Ras-HCM (1.1 yr) versus primary HCM (10 yrs) (p Conclusion The incidence of sudden cardiac death is lower in pediatric Rasopathy patients with HCM compared to primary HCM with most events occurring in infancy. It is less likely for Rasopathy patients to receive an ICD than a primary HCM patient even for secondary prevention, possibly due to younger patient age at the time of the event. Further research on risk factors for and mechanisms of sudden death in Ras-HCM is warranted.
- Published
- 2021
29. The TEAMMATE Trial: Study Design and Rationale of the First Pediatric Heart Transplant Randomized Clinical Trial
- Author
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Seth A. Hollander, Kevin P. Daly, Joseph W. Rossano, Matthew Fenton, J. Lee, Ashwin K. Lal, M.J. Bock, Christopher S. Almond, Gloria L. Klein, Elfriede Pahl, L. Barkoff, Lynn A. Sleeper, and Chesney Castleberry
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,medicine.medical_specialty ,Everolimus ,business.industry ,Trial study ,Fda approval ,030230 surgery ,Coronary disease ,Tacrolimus ,law.invention ,03 medical and health sciences ,0302 clinical medicine ,Randomized controlled trial ,law ,Internal medicine ,medicine ,030211 gastroenterology & hepatology ,Surgery ,Pediatric heart transplantation ,Cardiology and Cardiovascular Medicine ,business ,medicine.drug ,Rare disease - Abstract
Purpose Currently there are no-FDA approved immunosuppressants specific to pediatric heart transplantation (HT). In recent years, everolimus (EVL) has emerged as an alternative to tacrolimus (TAC) as a primary immunosuppressant to prevent rejection that may also prevent kidney and coronary disease. However, the two regimens have never been evaluated systematically. Methods The TEAMMATE Trial (IND 127980) is designed to evaluate the safety and efficacy of EVL and low-dose (LD-TAC) compared to standard-therapy TAC and mycophenolate mofetil (MMF). The study design and rationale are reviewed in light of challenges inherent in rare disease research. Results The TEAMMATE trial is the first multicenter randomized clinical trial (RCT) in pediatric HT. The primary purpose is to evaluate the risk-benefit profile of the two regimens to prevent major adverse transplant events (MATE), and to support FDA approval of 1 or both regimens for pediatric HT. Children Conclusion The TEAMMATE trial is the first RCT in pediatric HT. It is anticipated that the study will provide important information about the safety and effectiveness of EVL and TAC and provide valuable lessons into the design and conduct of future trials in pediatric HT.
- Published
- 2020
30. Marijuana in pediatric and adult congenital heart disease heart transplant listing: A survey of provider practices and attitudes
- Author
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Chesney Castleberry, Steven J. Kindel, Kari A. Phillips, Kevin P. Daly, Jonathan N. Johnson, Ashwin K. Lal, Joshua Sparks, and Philip T. Thrush
- Subjects
Adult ,Heart Defects, Congenital ,Male ,medicine.medical_specialty ,Heart disease ,Attitude of Health Personnel ,Best practice ,medicine.medical_treatment ,030232 urology & nephrology ,Medical Marijuana ,030230 surgery ,03 medical and health sciences ,0302 clinical medicine ,Surveys and Questionnaires ,Medicine ,Humans ,In patient ,Practice Patterns, Physicians' ,Child ,Contraindication ,Legalization ,Aged ,Heart transplantation ,Transplantation ,biology ,business.industry ,Middle Aged ,biology.organism_classification ,medicine.disease ,Organizational Policy ,United States ,Family medicine ,Pediatrics, Perinatology and Child Health ,Heart Transplantation ,Female ,Cannabis ,Listing (finance) ,business ,Phytotherapy - Abstract
Despite increasing legalization and use of marijuana, there is no consensus among pediatric heart transplant institutions or providers regarding users' eligibility for cardiac transplant. We sent a survey to pediatric and ACHD transplant providers (physicians, surgeons, transplant coordinators, and pharmacists) assessing their current institution's policies and their personal opinions about marijuana use in patients being considered for heart transplantation. Of the respondents, 84% practice in the United States and Canada. Most providers (80%) care for both pediatric and ACHD patients. Respondents included cardiologists (77%) and surgeons (11%), with the remaining being coordinators and pharmacists. Most providers (73%) reported their institution had no policy regarding marijuana use in heart transplant candidates. Only 20% of respondents' institutions consider mode of consumption, with 87% and 53% approving of oral and transdermal routes, respectively, and only 7% approving of vaporized or smoked routes. While 73% of providers would consider illegal marijuana use an absolute/relative contraindication to heart transplant listing, the number decreases to 57% for legal recreational users and 21% for legal medical users. Most providers personally believe marijuana to be physically and mentally/emotionally harmful to pediatric patients (67% and 72%, respectively). Many institutions lack a policy regarding marijuana use in pediatric and ACHD heart transplant candidates, and there is considerable disagreement among providers on the best practice. With increasing legalization and use of marijuana, each institution will have to address this issue thoughtfully to continue to provide high-quality, consistent, and equitable care for pediatric and ACHD heart transplant candidates.
- Published
- 2019
31. Variability in donor selection among pediatric heart transplant providers: Results from an international survey
- Author
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Anne I. Dipchand, Ryan R. Davies, Justin Godown, Richard Kirk, Anna Joong, Ashwin K. Lal, Michael A. McCulloch, Jeffrey G. Gossett, Janet Scheel, Oliver Miera, and David M. Peng
- Subjects
Adult ,Male ,Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,Adolescent ,Decision Making ,030232 urology & nephrology ,030230 surgery ,Donor age ,Lower limit ,Donor Selection ,03 medical and health sciences ,0302 clinical medicine ,Highly sensitized ,Surveys and Questionnaires ,Humans ,Medicine ,Practice Patterns, Physicians' ,Child ,Aged ,Transplantation ,Deceased donor ,Ejection fraction ,business.industry ,Donor selection ,Age Factors ,International survey ,Middle Aged ,Donor heart ,Family medicine ,Pediatrics, Perinatology and Child Health ,Emergency medicine ,Heart Transplantation ,Female ,Surgery ,Waitlist mortality ,Cardiology and Cardiovascular Medicine ,business ,Consensus guideline - Abstract
There is considerable variability in donor acceptance practices among adult heart transplant providers; however, pediatric data are lacking. The aim of this study was to assess donor acceptance practices among pediatric heart transplant professionals. The authors generated a survey to investigate clinicians' donor acceptance practices. This survey was distributed to all members of the ISHLT Pediatric Council in April 2018. A total of 130 providers responded from 17 different countries. There was a wide range of acceptable criteria for potential donors. These included optimal donor-to-recipient weight ratio (lower limit: 50%-150%, upper limit: 120%-350%), maximum donor age (25-75 years), and minimum acceptable left ventricular EF (30%-60%). Non-US centers demonstrated less restrictive donor selection criteria and were willing to accept older donors (50 vs 35 years, P < 0.001), greater size discrepancy (upper limit weight ratio 250% vs 200%, P = 0.009), and donors with a lower EF (45% vs 50%, P < 0.001). Recipient factors were most influential in the decision to accept marginal donors including recipients requiring ECMO support, ventilator support, and highly sensitized patients with a negative XM. However, programmatic factors impacted the decision to decline marginal donors including recent programmatic mortalities and concerns for programmatic restrictions from regulatory bodies. There is significant variation in donor acceptance practices among pediatric heart transplant professionals. Standardization of donor acceptance practices through the development of a consensus statement may help to improve donor utilization and reduce waitlist mortality.
- Published
- 2019
32. Impact of Race on Listing and Waitlist Mortality in Pediatric Cardiac Transplantation
- Author
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Ashwin K. Lal, Simon Urschel, James K. Kirklin, William Ravekes, Scott R. Auerbach, A. Cabrera, Neha Bansal, Shahnawaz Amdani, Devin Koehl, C. Baker-Smith, and R.S. Cantor
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,Race (biology) ,medicine.medical_specialty ,business.industry ,Family medicine ,medicine ,Surgery ,Listing (computer) ,Waitlist mortality ,Cardiology and Cardiovascular Medicine ,business - Published
- 2021
33. Ventricular Assist Device Outcomes in Children and Young Adults with Muscular Dystrophy: An ACTION Analysis
- Author
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S. Law, Ashwin K. Lal, Neha Bansal, Linda H. Cripe, Deipanjan Nandi, Matthew J. O'Connor, A. Lorts, Mary Mehegan, Kathleen E Simpson, Beth D. Kaufman, Chet R. Villa, Farhan Zafar, M.F. Shezad, David L Sutcliffe, Christina VanderPluym, David N. Rosenthal, Scott R. Auerbach, D. Mokshagundam, Lindsay J. May, and Carol A. Wittlieb-Weber
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,education.field_of_study ,Pediatrics ,medicine.medical_specialty ,business.industry ,medicine.medical_treatment ,Population ,Dilated cardiomyopathy ,medicine.disease ,Respiratory failure ,Quality of life ,Ventricular assist device ,medicine ,Surgery ,Young adult ,Cardiology and Cardiovascular Medicine ,business ,Adverse effect ,education ,Stroke - Abstract
Purpose Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have described their ventricular assist device (VAD) experience in this population, however broader applicability and outcomes remain unknown. Methods We examined outcomes of all patients with MD and dilated cardiomyopathy implanted with a VAD at Advanced Cardiac Therapies Improving Outcomes Network (ACTION) centers 9/2012-9/2020. Results A total of 19 VADs were implanted in 18 patients across 10 ACTION sites. The median duration of support was 252 days (range 11-1681 days). The majority of pts had dystrophinopathy (66%) and the median age at implant was 17.2 years (Table). Device strategy was bridge to transplant in 5 (27.8%), 4 of whom survived to transplant. The frequency of a positive outcome (transplanted or alive on device) at 6 months and 1 year were 89% and 84%, respectively (Figure). A single patient suffered a stroke (hemorrhagic), 2 patients developed sepsis, 1 patient developed respiratory failure requiring tracheostomy and 4 patients patient developed right heart failure (1 of whom required temporary right VAD). Conclusion In selected MD patients, VADs can serve as effective bridges to HT and chronic therapy. Right heart failure and infectious complications were relatively common, while stroke and respiratory failure were rare. Further research is needed to understand the patients who may benefit most from VAD therapy in terms of survival and quality of life and how to mitigate the risks the of the adverse events identified in the current report.
- Published
- 2021
34. A Novel Hybrid Single Ventricular Assist Device (SVAD) Strategy for a Neonate with Ventricular Septal Defect, Aortic Arch Hypoplasia, and Biventricular Dysfunction
- Author
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Ashwin K. Lal, Kimberly M. Molina, Lindsay J. May, Dana Boucek, Eric R. Griffiths, and Michelle Ploutz
- Subjects
Pulmonary and Respiratory Medicine ,Aortic arch ,Transplantation ,medicine.medical_specialty ,education.field_of_study ,Heart disease ,business.industry ,medicine.medical_treatment ,Population ,Cardiomyopathy ,Stent ,medicine.disease ,Cannula ,Hypoplasia ,Ventricular assist device ,Internal medicine ,medicine.artery ,medicine ,Cardiology ,Surgery ,Cardiology and Cardiovascular Medicine ,education ,business - Abstract
Introduction Neonates requiring biventricular VAD (BiVAD) support remain a high-risk population. Common challenges include limited space for multiple cannulae, poor wound healing, and difficulties establishing adequate VAD fill and ejection. Given these concerns, we present a unique case using a hybrid procedure and SVAD implant in a neonate with two-ventricle(2V) circulation. Case Report Female term neonate (birth weight 3.2 kg; BSA 0.2 kg/m2) born with a moderate-sized ventricular septal defect (VSD), aortic arch hypoplasia, and severe biventricular dysfunction due to a MHY7 mutation. In light of her weight, likely need for biventricular support given her underlying cardiomyopathy and need for extensive arch repair, we elected to proceed with a hybrid SVAD support strategy. We hypothesized that this approach would decrease the duration of bypass and minimize the number of cannula over the traditional approach of VSD and arch repair with BiVAD support. We also hoped this strategy would provide improved LV decompression, thus improving pulmonary mechanics and nutritional support. On day 6 of life, she underwent atrial septectomy, bilateral PA band placement, and implant of a 10 ml Berlin EXCOR using a 6 mm EXCOR cannula in the right atrium and a 5 mm EXCOR cannula in the main pulmonary artery. A PDA stent was placed using a perventricular approach. She progressed well post-operatively with discontinuation of inotropic support on post-operative day (POD) 3 and extubation on POD 4. VAD fill and ejection have been appropriate. The clinical course is noteworthy for diaphragm paralysis requiring plication and ongoing BiPAP support. Additionally, she developed NEC on POD 23 and has only tolerated ∼30% of her nutritional needs enterally. To date, she has remained on VAD support > 100 days while awaiting transplant. Summary We present a unique case of a neonate with 2V congenital heart disease who has been supported with a hybrid SVAD strategy using a Berlin EXCOR. While this approach has been advantageous in avoidance of VSD and arch repair and requiring fewer cannulae, it also has had challenges; namely, the need for ongoing respiratory and feeding support. We speculate that this may reflect the challenges of optimal PA band placement as well as difficulties providing balanced systemic and pulmonary flow on SVAD support.
- Published
- 2021
35. Model for End-Stage Liver Disease Excluding INR (MELD-XI) Score Predicts Outcomes in Pediatric Patients Supported with Ventricular Assist Device: An Analysis of the PediMACS Registry
- Author
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Jennifer Conway, James K. Kirklin, Yuk M. Law, Devin Koehl, Justin Godown, Ashwin K. Lal, David N. Rosenthal, Gerard J. Boyle, Shahnawaz Amdani, Angela Lorts, and Ryan S. Cantor
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,medicine.medical_specialty ,Creatinine ,business.industry ,medicine.medical_treatment ,Hemodynamics ,Risk management tools ,medicine.disease ,body regions ,chemistry.chemical_compound ,Model for End-Stage Liver Disease ,chemistry ,Internal medicine ,Heart failure ,Ventricular assist device ,Cohort ,medicine ,Surgery ,Implant ,Cardiology and Cardiovascular Medicine ,business - Abstract
Purpose Our study aims to identify the utility of pre-implant MELD-XI score in identifying pediatric patients at higher risk for morbidity and mortality post-VAD implant. Methods We reviewed all patients listed in the PediMACS database from 9/19/12 to 3/31/19. Patients missing bilirubin or creatinine that precluded calculation of MELD-XI score (n=89) were excluded. Patients were divided into three cohorts based on MELD-XI score pre-implant - low (minimum score 9.4, 13.6, 75-100%-tile). Baseline clinical and hemodynamic data were compared; outcomes data on morbidity and mortality were compared across groups. Results Of 596 patients included, 42.5% were in the low, 29.2% in the intermediate and 28.3% in the high MELD-XI cohort. Compared to the low and intermediate cohorts, patients in the high MELD-XI cohort were significantly older (p Conclusion Pediatric patients with an elevated MELD-XI score have higher acuity of illness pre-implant. This easily calculated score based on routinely collected lab values serves as a promising risk assessment tool in identifying pediatric VAD recipients at increased risk for mortality post VAD implant. Trending the MELD-XI score in patients with advanced heart failure may aid clinicians in timing of VAD implantation.
- Published
- 2020
36. DOES ALTERATION IN MYOCARDIAL DEFORMATION PREDICT ARRHYTHMIA RISK IN CHILDREN HOSPITALIZED FOR ACUTE MYOCARDITIS?
- Author
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Zhining Ou, Shaji C. Menon, Ashwin K. Lal, Cathleen R. Pruitt, and Mary Niu
- Subjects
medicine.medical_specialty ,Acute myocarditis ,business.industry ,Internal medicine ,medicine ,Cardiology ,Deformation (meteorology) ,Cardiology and Cardiovascular Medicine ,business - Published
- 2020
37. Implantable Cardioverter Defibrillator Use in Males with Duchenne Muscular Dystrophy and Severe Left Ventricular Dysfunction
- Author
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S. Law, Jonathan N. Johnson, Joshua M. Friedland-Little, Jennifer Conway, Shriprasad R. Deshpande, Kurt R. Schumacher, M.J. Bock, Kenneth R. Knecht, Chet R. Villa, Irene D. Lytrivi, Ashwin K. Lal, Shawn C. West, Carol A. Wittlieb-Weber, K. Gambetta, Ryan J. Butts, and Michael A. McCulloch
- Subjects
Adult ,Male ,Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,Adolescent ,Duchenne muscular dystrophy ,medicine.medical_treatment ,Population ,Cardiomyopathy ,030204 cardiovascular system & hematology ,Ventricular tachycardia ,03 medical and health sciences ,Ventricular Dysfunction, Left ,Young Adult ,0302 clinical medicine ,Internal medicine ,Humans ,Medicine ,In patient ,education ,Retrospective Studies ,Transplantation ,education.field_of_study ,Ejection fraction ,biology ,business.industry ,Angiotensin-converting enzyme ,Retrospective cohort study ,Fractional shortening ,medicine.disease ,Implantable cardioverter-defibrillator ,Defibrillators, Implantable ,Muscular Dystrophy, Duchenne ,030228 respiratory system ,Echocardiography ,Pediatrics, Perinatology and Child Health ,Cardiology ,biology.protein ,Female ,Surgery ,Myocardial fibrosis ,Cardiology and Cardiovascular Medicine ,business - Abstract
Duchenne muscular dystrophy (DMD) is characterized by myocardial fibrosis and left ventricular (LV) dysfunction. Implantable cardioverter defibrillator (ICD) use has not been characterized in this population but is considered for symptomatic patients with severe LV dysfunction (SLVD) receiving guideline-directed medical therapy (GDMT). We evaluated ICD utilization and efficacy in patients with DMD. Retrospective cohort study of DMD patients from 17 centers across North America between January 2, 2005 and December 31, 2015. ICD use and its effect on survival were evaluated in patients with SLVD defined as ejection fraction (EF)
- Published
- 2019
38. Risk Factors for Cardiac Causes of Death in Males with Duchenne Muscular Dystrophy
- Author
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David R. Weber, Jonathan N. Johnson, K. Gambetta, K.R. Schumacher, Joshua M. Friedland-Little, Irene D. Lytrivi, Chet R. Villa, Shawn C. West, Shriprasad R. Deshpande, Ryan J. Butts, K.K. Knecht, Chentel Cunningham, Ashwin K. Lal, Michael A. McCulloch, M.J. Bock, S. Law, and Carol A. Wittlieb-Weber
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,medicine.medical_specialty ,education.field_of_study ,Ejection fraction ,business.industry ,Duchenne muscular dystrophy ,Population ,Retrospective cohort study ,medicine.disease ,Sudden cardiac death ,Respiratory failure ,Internal medicine ,Heart failure ,medicine ,Cardiology ,Surgery ,Cardiology and Cardiovascular Medicine ,business ,education ,Stroke - Abstract
Purpose As survival and neuromuscular function in Duchenne Muscular Dystrophy (DMD) have improved with glucocorticoid therapy and advances in respiratory care, the proportion of cardiac deaths is increasing. Little is known about risk factors for cardiac causes of death in this population. Methods We conducted a retrospective cohort study of 436 males with DMD followed at 17 centers across North America, from January 1, 2005-December 31, 2015, and sought to identify risk factors for cardiac causes of death, including sudden cardiac death and progressive heart failure. Those dying of cardiac causes were compared to those who died of non-cardiac causes and to those alive at study end. Results There were 29 (6.8%) deaths during the study period at a median age of 19 (IQR: 15-31) years; 8 (27.6%) cardiac (SCD, n=5 and progressive HF, n=3) and 21 non-cardiac (respiratory failure, n=8, multi-organ failure, n=3, stroke, n=1, and other/unknown, n=9). Age at death did not differ based on cause; cardiac 18 (IQR: 15.5-24) years vs. non-cardiac 19 (IQR: 16.5-23) years, p=0.68, but those living were significantly younger (14.9 (IQR: 11.0-19.1) years vs. those who died of cardiac (p=0.03) and non-cardiac (p=0.002) causes). Genetic testing was available for 15/29 deceased (52%) and 292/379 (77%) of those living with all deceased having a deletion, however this did not differ from those living (228/292 (78%), p=0.13). Current or past steroid use was significantly lower in those dying of cardiac causes compared to those living (2/8 (25%) vs. 304/378 (80.4%), p=0.001). Ejection fraction (EF) at study end/death was significantly lower for those dying of cardiac causes compared to those living (mean 37.5% ±12.8 vs. 54.5% ±10.8, p=0.01) but not compared to those dying of non-cardiac causes (37.5% ±12.8 vs. 41.2% ± 19.3, p=0.58). Fractional shortening (FS) was also lower in those dying of cardiac causes compared to those living (mean 18.5% ±10.2 vs. 29.6% ±7.7, p=0.03) but not compared to those dying of non-cardiac causes (18.5% ±10.2 vs. 23.4 % ±9.2, p=0.3). Conclusion In a large cohort of males with DMD, approximately 1/3rd of deaths were from a cardiac cause. Lack of steroid use was associated with cardiac causes of death, while systolic dysfunction was associated with death from any cause. Future studies are needed to define optimal medical management of systolic dysfunction in males with DMD with hopes of extending survival.
- Published
- 2019
39. The Role of Prophylactic Heart Failure Therapy in Duchenne Muscular Dystrophy
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S. Law, Jennifer Conway, K.R. Schumacher, M.A. McCulloch, Zhining Ou, Shawn C. West, Kenneth R. Knecht, Jonathan N. Johnson, Joshua M. Friedland-Little, Shriprasad R. Deshpande, Ashwin K. Lal, K. Gambetta, Carol A. Wittlieb-Weber, Angela P. Presson, Irene D. Lytrivi, Ryan J. Butts, M.J. Bock, and Chet R. Villa
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,medicine.medical_specialty ,Aldosterone ,Ejection fraction ,Digoxin ,business.industry ,medicine.drug_class ,Duchenne muscular dystrophy ,Retrospective cohort study ,medicine.disease ,chemistry.chemical_compound ,chemistry ,Internal medicine ,Heart failure ,Cohort ,Cardiology ,Medicine ,Surgery ,Cardiology and Cardiovascular Medicine ,business ,Beta blocker ,medicine.drug - Abstract
Purpose There is variable practice and debate surrounding prophylactic heart failure (HF) therapy in Duchenne Muscular Dystrophy (DMD) patients with normal systolic function . We sought to determine the impact of HF medications including ACE inhibitors and ARB on the development of moderate systolic dysfunction or death in a cohort of DMD patients. Methods We performed a retrospective cohort study of DMD patients followed at 17 centers across North America from 1/1/2005-12/31/2015. Inclusion criteria were normal systolic function on baseline echocardiogram (ejection fraction (EF) ≥ 55%) and age ≥ 9 years during the study. Moderate systolic dysfunction was defined as an EF≤40% or shortening fraction ≤21% if EF was not available. We compared the composite outcome of moderate systolic dysfunction or death in patients who started HF therapies before evidence of systolic dysfunction (PPX) vs those who did not (non-PPX) using a Cox proportional hazards regression model adjusting for baseline age. We also compared prophylaxis with ACE/ARB (ACE/ARB PPX) to non-PPX. Results 277 males with DMD and normal systolic function were included. Prophylactic HF medications were used in 70 patients (25%) which included ACE/ARB (44, 63%), beta blocker (26, 37%), aldosterone blockade (6, 9%) and digoxin (5, 7%). Medications began at a mean (SD) age of 13.6 (4) years. Mean baseline ages were 10.7 (4.2) and 9.9 (3.9) years in the PPX and non-PPX groups (p=0.16), while mean age to develop moderate systolic dysfunction was 18.6 (5.4) and 16.8 (4.0) in the PPX and non-PPX groups (p=.005). The risk of developing moderate systolic dysfunction or death was 50% lower in the PPX group compared to the non-PPX group (HR=0.50; p=.044) and 79% lower in the ACE/ARB PPX group (N=38) compared to the non-PPX group (HR=0.21; p=0.035) (Figure 1). Conclusion There is variability in the use and choice of prophylactic HF medications in boys with DMD. This cohort showed a benefit to introduction of ACE/ARBs prior to evidence of systolic dysfunction.
- Published
- 2019
40. Transition from brand to generic tacrolimus is associated with a decrease in trough blood concentration in pediatric heart transplant recipients
- Author
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Brian Feingold, Raman Venkataramanan, Rujuta Joshi, Son Q. Duong, and Ashwin K. Lal
- Subjects
Graft Rejection ,Male ,medicine.medical_specialty ,Pediatrics ,Adolescent ,Bioequivalence ,Trough (economics) ,Tacrolimus ,Organ transplantation ,Young Adult ,Blood concentration ,medicine ,Drugs, Generic ,Humans ,Trough Concentration ,Child ,Retrospective Studies ,Transplantation ,Drug Substitution ,business.industry ,Treatment Outcome ,Therapeutic Equivalency ,Child, Preschool ,Pediatrics, Perinatology and Child Health ,Linear Models ,Heart Transplantation ,Female ,Transplant patient ,Liver function ,business ,Immunosuppressive Agents - Abstract
There are limited data available on the bioequivalence of generic and brand-name tacrolimus in pediatric and heart transplant patients. We characterized changes in 12-hour trough concentrations and clinical outcomes after transition from brand to generic tacrolimus in pediatric thoracic organ transplant recipients. Patients with a pharmacy-confirmed date of switch between generic and brand tacrolimus were identified, as well as a matched control group that did not switch for comparison. We identified 18 patients with a confirmed date of switch, and in 12 patients that remained on the same dose, trough concentrations were 14% less than when they were on brand (p = 0.037). The average change was -1.15 ± 1.76 ng/mL (p = 0.045). The control group did not experience a change in trough concentration and was different than the switched group (p = 0.005). There were no differences in dosage changes or kidney or liver function. In the year after switch, 24% of patients who were switched to generic experienced a rejection event vs. 18% in the patients on brand. We suggest a strategy of monitoring around the time of transition, and education of the patient/family to notify the care team when changes from brand to generic or between generics occur.
- Published
- 2015
41. Left ventricular non-compaction cardiomyopathy in children listed for heart transplant: Analysis from the Pediatric Heart Transplant Study Group
- Author
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Brian Feingold, Elizabeth Pruitt, Borah J. Hong, Ashwin K. Lal, and Kimberly Y. Lin
- Subjects
Cardiomyopathy, Dilated ,Male ,Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,Percutaneous ,Waiting Lists ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,Left Ventricular Non-Compaction Cardiomyopathy ,Balloon ,Ventricular Dysfunction, Left ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,medicine.artery ,Angioplasty ,Humans ,Medicine ,030212 general & internal medicine ,Child ,Retrospective Studies ,Transplantation ,Adult patients ,business.industry ,Catheter ,Child, Preschool ,Pulmonary artery ,Cardiology ,Heart Transplantation ,Female ,Surgery ,Chronic thromboembolic pulmonary hypertension ,Cardiology and Cardiovascular Medicine ,business - Abstract
1. Kataoka M, Inami T, Hayashida K, et al. Percutaneous transluminal pulmonary angioplasty for the treatment of chronic thromboembolic pulmonary hypertension. Circ Cardiovasc Interv 2012;5:756-62. 2. Inami T, Kataoka M, Shimura N, et al. Pressure-wire-guided percutaneous transluminal pulmonary angioplasty: a breakthrough in the catheter-interventional therapy for chronic thromboembolic pulmonary hypertension. JACC Cardiovasc Interv 2014;7:1297-306. 3. Kreutzer J, Landzberg MJ, Preminger TJ, et al. Isolated peripheral pulmonary artery stenoses in the adult. Circulation 1996;93:1417-23. 4. Rothman A, Levy DJ, Sklansky MS, et al. Balloon angioplasty and stenting of multiple intralobar pulmonary arterial stenoses in adult patients. Catheter Cardiovasc Interv 2003;58:252-60.
- Published
- 2016
42. Heart failure after the Norwood procedure: An analysis of the Single Ventricle Reconstruction Trial
- Author
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William T. Mahle, Kristin M. Burns, Felicia Trachtenberg, Jondavid Menteer, Andrew M. Atz, Jane W. Newburger, Anne I. Dipchand, Kevin P. Daly, Chet R. Villa, Kimberly Y. Lin, Kurt R. Schumacher, Chenwei Hu, Steven J. Kindel, Jeffrey P. Jacobs, Heather T. Henderson, Marc E. Richmond, Ashwin K. Lal, and Michael A. McCulloch
- Subjects
Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,Time Factors ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,Norwood Procedures ,Article ,Hypoplastic left heart syndrome ,03 medical and health sciences ,0302 clinical medicine ,Postoperative Complications ,Interquartile range ,Internal medicine ,Hypoplastic Left Heart Syndrome ,Extracorporeal membrane oxygenation ,Medicine ,Humans ,030212 general & internal medicine ,Prospective Studies ,Child ,Heart Failure ,Transplantation ,business.industry ,Proportional hazards model ,Infant ,medicine.disease ,Norwood Operation ,Cardiac surgery ,Heart failure ,Child, Preschool ,Cardiology ,Surgery ,Norwood procedure ,Cardiology and Cardiovascular Medicine ,business ,Follow-Up Studies - Abstract
BACKGROUND: Heart failure results in significant morbidity and mortality for young children with hypoplastic left heart syndrome (HLHS) following the Norwood procedure. METHODS: We studied subjects enrolled in the prospective Single Ventricle Reconstruction (SVR) trial who survived to hospital discharge after the Norwood operation and were followed up to age 6 years. The primary outcome was heart failure, defined as heart transplant listing after Norwood hospitalization, death attributable to heart failure or symptomatic heart failure (NYHA class IV). Multivariate modeling was undertaken using Cox regression methodology to determine variables associated with heart failure. RESULTS: Of the 461 subjects discharged to home following Norwood procedure, 66 (14.3%) met criteria for heart failure. Among these, 15 died from heart failure, 39 were listed for transplant (22 had a transplant, 12 died after listing and 5 were alive and not yet transplanted), and 12 had NYHA Class IV heart failure but were never listed. The median age at heart failure identification was 1.28 yrs (interquartile range 0.30 - 4.69 years). Factors associated with heart failure included post-Norwood lower fractional area change, need for ECMO, non-Hispanic ethnicity, Norwood perfusion type and total support time (p
- Published
- 2017
43. Pediatric Cardiomyopathies
- Author
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Teresa M. Lee, Daphne T. Hsu, Paul Kantor, Jeffrey A. Towbin, Stephanie M. Ware, Steven D. Colan, Wendy K. Chung, John L. Jefferies, Joseph W. Rossano, Chesney D. Castleberry, Linda J. Addonizio, Ashwin K. Lal, Jacqueline M. Lamour, Erin M. Miller, Philip T. Thrush, Jason D. Czachor, Hiedy Razoky, Ashley Hill, and Steven E. Lipshultz
- Subjects
Genetic Markers ,Physiology ,Incidence ,Myocardium ,030204 cardiovascular system & hematology ,Prognosis ,Article ,03 medical and health sciences ,Cardiac Imaging Techniques ,0302 clinical medicine ,Phenotype ,Molecular Diagnostic Techniques ,Risk Factors ,Mutation ,Humans ,Ventricular Function ,Genetic Predisposition to Disease ,030212 general & internal medicine ,Age of Onset ,Cardiology and Cardiovascular Medicine ,Cardiomyopathies - Abstract
Pediatric cardiomyopathies are rare diseases with an annual incidence of 1.1 to 1.5 per 100 000. Dilated and hypertrophic cardiomyopathies are the most common; restrictive, noncompaction, and mixed cardiomyopathies occur infrequently; and arrhythmogenic right ventricular cardiomyopathy is rare. Pediatric cardiomyopathies can result from coronary artery abnormalities, tachyarrhythmias, exposure to infection or toxins, or secondary to other underlying disorders. Increasingly, the importance of genetic mutations in the pathogenesis of isolated or syndromic pediatric cardiomyopathies is becoming apparent. Pediatric cardiomyopathies often occur in the absence of comorbidities, such as atherosclerosis, hypertension, renal dysfunction, and diabetes mellitus; as a result, they offer insights into the primary pathogenesis of myocardial dysfunction. Large international registries have characterized the epidemiology, cause, and outcomes of pediatric cardiomyopathies. Although adult and pediatric cardiomyopathies have similar morphological and clinical manifestations, their outcomes differ significantly. Within 2 years of presentation, normalization of function occurs in 20% of children with dilated cardiomyopathy, and 40% die or undergo transplantation. Infants with hypertrophic cardiomyopathy have a 2-year mortality of 30%, whereas death is rare in older children. Sudden death is rare. Molecular evidence indicates that gene expression differs between adult and pediatric cardiomyopathies, suggesting that treatment response may differ as well. Clinical trials to support evidence-based treatments and the development of disease-specific therapies for pediatric cardiomyopathies are in their infancy. This compendium summarizes current knowledge of the genetic and molecular origins, clinical course, and outcomes of the most common phenotypic presentations of pediatric cardiomyopathies and highlights key areas where additional research is required. Clinical Trial Registration: URL: http://www.clinicaltrials.gov . Unique identifiers: NCT02549664 and NCT01912534.
- Published
- 2017
44. Resource Utilization for Initial Hospitalization in Pediatric Heart Transplantation in the United States
- Author
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Aaron W. Eckhauser, Jacob Wilkes, Xiaoming Sheng, Ashwin K. Lal, Nelangi M. Pinto, Dana Boucek, Hsin Yi Cindy Weng, and Shaji C. Menon
- Subjects
Heart Defects, Congenital ,Male ,medicine.medical_specialty ,Hospitals, Low-Volume ,Adolescent ,medicine.medical_treatment ,Comorbidity ,030204 cardiovascular system & hematology ,law.invention ,03 medical and health sciences ,0302 clinical medicine ,Interquartile range ,law ,Internal medicine ,medicine ,Humans ,030212 general & internal medicine ,Hospital Costs ,Child ,Retrospective Studies ,Heart transplantation ,Heart Failure ,business.industry ,Age Factors ,Infant, Newborn ,Infant ,Retrospective cohort study ,Length of Stay ,medicine.disease ,Hospitals, Pediatric ,Intensive care unit ,Hospital Charges ,United States ,Hospitalization ,Intensive Care Units ,Heart failure ,Child, Preschool ,Emergency medicine ,Cardiology ,Health Resources ,Heart Transplantation ,Female ,Pediatric heart transplantation ,Cardiology and Cardiovascular Medicine ,business ,Resource utilization ,Hospitals, High-Volume - Abstract
Pediatric heart transplantation (HT) is resource intensive. Event-driven pediatric databases do not capture data on resource use. The objective of this study was to evaluate resource utilization and identify associated factors during initial hospitalization for pediatric HT. This multicenter retrospective cohort study utilized the Pediatric Health Information Systems database (43 children's hospitals in the United States) of children ≤19 years of age who underwent transplant between January 2007 and July 2013. Demographic variables including site, payer, distance and time to center, clinical pre- and post-transplant variables, mortality, cost, and charge were the data collected. Total length of stay (LOS) and charge for the initial hospitalization were used as surrogates for resource use. Charges were inflation adjusted to 2013 dollars. Of 1,629 subjects, 54% were male, and the median age at HT was 5 years (IQR [interquartile range] 0 to 13). The median total and intensive care unit LOS were 51 (IQR 23 to 98) and 23 (IQR 9 to 58) days, respectively. Total charge and cost for hospitalization were $852,713 ($464,900 to $1,609,300) and $383,600 ($214,900 to $681,000) respectively. Younger age, lower volume center, southern region, and co-morbidities before transplant were associated with higher resource use. In later years, charges increased despite shorter LOS. In conclusion, this large multicenter study provides novel insight into factors associated with resource use in pediatric patients having HT. Peritransplant morbidities are associated with increased cost and LOS. Reducing costs in line with LOS will improve health-care value. Regional and center volume differences need further investigation for optimizing value-based care and efficient use of scarce resources.
- Published
- 2017
45. Management of Cardiac Involvement Associated With Neuromuscular Diseases: A Scientific Statement From the American Heart Association
- Author
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Larry W Markham, Shi-Joon Yoo, William T. Mahle, Brian Feingold, W. James Parks, Scott R. Auerbach, Takeshi Tsuda, Ashwin K. Lal, Paul J. Wang, Andrea A. Domenighetti, John L. Jefferies, Paula Clemens, and Daniel P. Judge
- Subjects
medicine.medical_specialty ,Neuromuscular disease ,Cardiovascular health ,Population ,Cardiomyopathy ,Psychological intervention ,Disease ,030204 cardiovascular system & hematology ,03 medical and health sciences ,0302 clinical medicine ,Muscular Diseases ,Risk Factors ,Physiology (medical) ,medicine ,Humans ,Myotonic Dystrophy ,Intensive care medicine ,education ,Peripheral muscle ,education.field_of_study ,business.industry ,American Heart Association ,Neuromuscular Diseases ,medicine.disease ,Muscular Dystrophy, Emery-Dreifuss ,United States ,Muscular Dystrophy, Duchenne ,Muscular Dystrophies, Limb-Girdle ,Friedreich Ataxia ,Heart failure ,Barth Syndrome ,Physical therapy ,Cardiology and Cardiovascular Medicine ,business ,Cardiomyopathies ,030217 neurology & neurosurgery ,Myopathies, Structural, Congenital - Abstract
For many neuromuscular diseases (NMDs), cardiac disease represents a major cause of morbidity and mortality. The management of cardiac disease in NMDs is made challenging by the broad clinical heterogeneity that exists among many NMDs and by limited knowledge about disease-specific cardiovascular pathogenesis and course-modifying interventions. The overlay of compromise in peripheral muscle function and other organ systems, such as the lungs, also makes the simple application of endorsed adult or pediatric heart failure guidelines to the NMD population problematic. In this statement, we provide background on several NMDs in which there is cardiac involvement, highlighting unique features of NMD-associated myocardial disease that require clinicians to tailor their approach to prevention and treatment of heart failure. Undoubtedly, further investigations are required to best inform future guidelines on NMD-specific cardiovascular health risks, treatments, and outcomes.
- Published
- 2017
46. Ventricular assist device use in single ventricle congenital heart disease
- Author
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Ashwin K. Lal, David L.S. Morales, Chet R. Villa, and Waldemar F. Carlo
- Subjects
Heart Defects, Congenital ,medicine.medical_specialty ,Heart disease ,medicine.medical_treatment ,Heart Ventricles ,Adult population ,030204 cardiovascular system & hematology ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,medicine ,Humans ,Intensive care medicine ,Heart Failure ,Transplantation ,business.industry ,medicine.disease ,medicine.anatomical_structure ,Treatment Outcome ,030228 respiratory system ,Ventricle ,Ventricular assist device ,Heart failure ,Pediatrics, Perinatology and Child Health ,Cardiology ,Implant ,Heart-Assist Devices ,business - Abstract
As VAD have become an effective therapy for end-stage heart failure, their application in congenital heart disease has increased. Single ventricle congenital heart disease introduces unique physiologic challenges for VAD use. However, with regard to the mixed clinical results presented within this review, we suggest that patient selection, timing of implant, and center experience are all important contributors to outcome. This review focuses on the published experience of VAD use in single ventricle patients and details physiologic challenges and novel approaches in this growing pediatric and adult population.
- Published
- 2017
47. Clinical practice patterns are relatively uniform between pediatric heart transplant centers: A survey-based assessment
- Author
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Melanie D. Everitt, Scott R. Auerbach, Seth A. Hollander, Ashwin K. Lal, Kevin P. Daly, Elfriede Pahl, Joseph W. Rossano, Christopher S. Almond, Matthew Fenton, Chesney Castleberry, and Sonja I. Ziniel
- Subjects
Graft Rejection ,medicine.medical_specialty ,Canada ,Attitude of Health Personnel ,medicine.medical_treatment ,Azathioprine ,030204 cardiovascular system & hematology ,030230 surgery ,Perioperative Care ,law.invention ,03 medical and health sciences ,0302 clinical medicine ,Randomized controlled trial ,law ,Internal medicine ,medicine ,Humans ,Healthcare Disparities ,Practice Patterns, Physicians' ,Intensive care medicine ,Child ,Heart transplantation ,Response rate (survey) ,Transplantation ,Everolimus ,business.industry ,Immunosuppression ,Hospitals, Pediatric ,Tacrolimus ,United Kingdom ,United States ,Sirolimus ,Health Care Surveys ,Pediatrics, Perinatology and Child Health ,Heart Transplantation ,business ,Brazil ,Immunosuppressive Agents ,medicine.drug - Abstract
Clinical practice variations are a barrier to the study of pediatric heart transplants and coordination of multicenter RCTs in this patient population. We surveyed centers to describe practice patterns, understand areas of variation, and willingness to modify protocol. Pediatric heart transplant centers were identified, and one survey was completed per center. Simple descriptive statistics were used. The response rate was 77% (40 responses from 52 contacted centers, 37 with complete responses). Median center volume of respondents was eight transplants/year (IQR 3-19). Most centers reported tacrolimus (36/38, 95%) and mycophenolate mofetil (36/38, 95%) as maintenance immunosuppression. Other immunosuppression agents reported were cyclosporine (7/38, 18%), everolimus or sirolimus (3/38, 8%), and azathioprine (2/38, 5%). Overall, respondents answered similarly for questions regarding clinical practices including induction therapy, maintenance immunosuppression, and rejection treatment threshold (>85% agreement for all). Additionally, willingness to change clinical practices was over 70% for all practices surveyed (35 total respondents), and 97% of centers (36/37) were willing to participate in a RCT of maintenance immunosuppression. In conclusion, we found many similar clinical practice protocols. Most centers are willing to collaborate on a common protocol in order to participate in a RCT and support a trial investigating maintenance immunosuppression.
- Published
- 2017
48. When Lightning Strikes Twice in Pediatrics: Case Report and Review of Recurrent Myocarditis
- Author
-
Dylan V. Miller, Kimberly M. Molina, Lindsay J. May, Alisha Floyd, Michael D. Puchalski, and Ashwin K. Lal
- Subjects
Male ,Pediatrics ,medicine.medical_specialty ,Myocarditis ,Adolescent ,Fulminant ,030204 cardiovascular system & hematology ,Polymerase Chain Reaction ,Parvoviridae Infections ,03 medical and health sciences ,0302 clinical medicine ,Clinical history ,Recurrence ,030225 pediatrics ,Biopsy ,medicine ,Humans ,Limited evidence ,medicine.diagnostic_test ,Ventricular function ,business.industry ,Magnetic resonance imaging ,medicine.disease ,Magnetic Resonance Imaging ,Patient management ,Pediatrics, Perinatology and Child Health ,business - Abstract
Myocarditis is an important but incompletely understood cause of cardiac dysfunction. Children with fulminant myocarditis often require inotropic or mechanical circulatory support, and researchers in some studies suggest that up to 42% of children who die suddenly have evidence of myocarditis. Recurrent myocarditis is extremely rare, and the vast majority of reported cases involve adult patients. Pediatric providers who suspect a recurrence of myocarditis have limited evidence to guide patient management because the literature in this domain is sparse. Here we present a unique, illustrative pediatric case of recurrent myocarditis. A 14-year-old boy presented for the second time in 2 years with a clinical history strongly suggestive of myocarditis. Although myocarditis was suggested in the results of cardiac MRI, no pathogen was identified during his first presentation. During his second episode of myocarditis, parvovirus was confirmed by polymerase chain reaction testing of an endomyocardial specimen that also met Dallas criteria for myocarditis. With each presentation, he had decreased ventricular function that subsequently normalized. To the best of our knowledge, there are no reports of recurrent myocarditis in children in whom the diagnosis was confirmed by using MRI and/or biopsy data. Reviewing this distinctive case and the existing literature may help characterize this entity and raise awareness among care providers.
- Published
- 2017
49. Characteristics of Clinically Diagnosed Pediatric Myocarditis in a Contemporary Multi-Center Cohort
- Author
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Kenneth R. Knecht, Gerard J. Boyle, Shriprasad R. Deshpande, Ashwin K. Lal, Shawn C. West, Ryan J. Butts, Marc E. Richmond, Carolina A. Prada-Ruiz, and K. Gambetta
- Subjects
Male ,medicine.medical_specialty ,Pediatrics ,Myocarditis ,Adolescent ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,03 medical and health sciences ,0302 clinical medicine ,Risk Factors ,030225 pediatrics ,medicine ,Ventricular Dysfunction ,Humans ,Child ,Retrospective Studies ,Heart transplantation ,business.industry ,Medical record ,Infant ,Vascular surgery ,medicine.disease ,Cardiac surgery ,Transplantation ,Treatment Outcome ,Heart failure ,Child, Preschool ,Pediatrics, Perinatology and Child Health ,Cohort ,Female ,Cardiology and Cardiovascular Medicine ,business - Abstract
The objective of this study was to describe a contemporary cohort of pediatric patients hospitalized for clinically suspected myocarditis. A retrospective chart review was performed at seven tertiary pediatric hospitals. Electronic medical records were searched between 2008 and 2012 for patients ≤18 years admitted with an ICD-9 code consistent with myocarditis. Patients were excluded if the admitting or consulting cardiologist did not suspect myocarditis during the admission or an alternative diagnosis was determined. One hundred seventy-one patients were discharged or died with a primary diagnosis of myocarditis. Median age was 13.1 years (IQR 2.1, 15.9), with a bimodal distribution; 24%
- Published
- 2017
50. Outcomes of Myocarditis in Patients with Normal Left Ventricular Systolic Function on Admission
- Author
-
Shriprasad R. Deshpande, Spencer B. Barfuss, Ashwin K. Lal, Kenneth R. Knecht, Marc E. Richmond, K. Gambetta, and Ryan J. Butts
- Subjects
Pulmonary and Respiratory Medicine ,Transplantation ,medicine.medical_specialty ,Myocarditis ,business.industry ,medicine.disease ,Internal medicine ,medicine ,Cardiology ,Surgery ,In patient ,Normal left ventricular systolic function ,Cardiology and Cardiovascular Medicine ,business - Published
- 2018
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