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3. ACTION-ARC Pediatric and Adult Congenital Heart Disease Ventricular Assist Device Adverse Event Definitions-2023

Author

Lorts, Angela, VanderPluym, Christina, Adachi, Iki, Perry, Tanya, Alexander, Peta M. A., Almond, Christopher S., Auerbach, Scott R., Barbaro, Ryan P., Bhavsar, Sonya, Bourque, Kevin, Conway, Jennifer, Danziger-Isakov, Lara A., Davies, Ryan R., Eghtesady, Pirooz, Hirata, Yasutaka, Ichord, Rebecca N., Kormos, Robert L., Kroslowitz, Robert, Krucoff, Mitchell, Lantz, Jodie, Mehegan, Mary, Mehran, Roxana, Morales, David L. S., Murray, Jenna, Niebler, Robert A., O’Connor, Matthew J., Pagani, Francis D., Peng, David M., Rossano, Joseph W., Spitzer, Ernest, Steiner, Marie E., Sutcliffe, David L., Taylor, J. Michael, Villa, Chet R., Wearden, Peter D., and Rosenthal, David

Published
2024
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7. A prospective multicenter feasibility study of a miniaturized implantable continuous flow ventricular assist device in smaller children with heart failure

Author

Almond, Christopher S., Davies, Ryan, Adachi, Iki, Richmond, Marc, Law, Sabrina, Tunuguntla, Hari, Mao, Chad, Shaw, Fawwaz, Lantz, Jodie, Wearden, Peter D., Jordan, Lori C., Ichord, Rebecca N., Burns, Kristin, Zak, Victor, Magnavita, Ashley, Gonzales, Selena, Conway, Jennifer, Jeewa, Aamir, Freemon, D.’Andrea, Stylianou, Mario, Sleeper, Lynn, Dykes, John C., Ma, Michael, Fynn-Thompson, Francis, Lorts, Angela, Morales, David, Vanderpluym, Christina, Dasse, Kurt, Patricia Massicotte, M., Jaquiss, Robert, and Mahle, William T.

Published
2024
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9. Impact of HeartWare ventricular assist device discontinuation on the pediatric population: An Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry analysis

Author

Robert A. Niebler, MD, Muhammad Shezad, Mohammed Absi, MD, Othman A. Aljohani, MD, Shahnawaz Amdani, MD, Scott Auerbach, MD, David W. Bearl, MD, Katerina Boucek, MD, Holger Buchholz, MD, Chesney Castleberry, MD, John Dykes, MD, Kevin Engelhardt, MD, Joshua Friedland-Little, MD, Kyle Hope, MD MS, Mehreen Iqbal, MD, Aamir Jeewa, MD, Anna Joong, MD, Angela Lorts, MD, Lindsay May, MD, Deepa Mokshagundam, MD, Deipanjan Nandi, MD MSc, Matthew O'Connor, John J. Parent, MD, David M. Peng, MD, Natalie Shwaish, MD, Svetlana B. Shugh, MD, Christina VanderPluym, MD, Gabrielle Vaughn, MD, and Mattew Zinn, DO

Subjects
pediatric, mechanical circulatory support, ventricular assist device, complications, heart failure, Surgery, RD1-811, Specialties of internal medicine, RC581-951
Abstract

Background: The HeartWare ventricular assist device (HVAD) was discontinued in July 2021. The study aims to describe the impact the discontinuation the HVAD had on pediatric ventricular assist device (VAD) utilization and outcomes. Methods: The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry was queried for type of VAD utilization and outcomes/adverse event rates in patients with a body surface area of 0.7 to 1.4 m2. Results were compared from before and after July 2021. All patients in the registry implanted with an HVAD were reported to determine overall outcomes of these patients and define who remains on the device. Results: The HeartMate 3 (HM3) primarily replaced the HVAD in pediatric patients increasing from 29 of 258 (11%) of implants before July 2021 to 31 of 109 (29%) of implants after. A small increase in the use of the Berlin Heart EXCOR (40 of 258, 16% before to 20 of 109, 18% after) and paracorporeal continuous flow devices (116 of 258, 45% before to 58 of 109, 53% after) was also observed. The rate of ischemic stroke increased in the overall population and a decrease in bleeding complications in the EXCOR group was observed. Of the 187 pediatric patients implanted with an HVAD in the registry, 7 patients remain supported, 1 patient transitioned from the HVAD to an HM3, and 6 patients were lost to follow-up. Conclusions: The HM3 has been the primary replacement for the HVAD in the medium-sized pediatric population. The rate of ischemic stroke was higher after July 2021.

Published
2024
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10. CRISPR/Cas9-Mediated Mutagenesis of Carotenoid Cleavage Dioxygenase (CCD) Genes in Sorghum Alters Strigolactone Biosynthesis and Plant Biotic Interactions

Author

Jingjie Hao, Ying Yang, Stephanie Futrell, Elizabeth A. Kelly, Claire M. Lorts, Baloua Nebie, Steven Runo, Jinliang Yang, Sophie Alvarez, Jesse R. Lasky, and Daniel P. Schachtman

Subjects
CCD, microbiome, sorghum, Strigolactone, Plant culture, SB1-1110, Microbial ecology, QR100-130, Plant ecology, QK900-989
Abstract

Strigolactones are a group of small molecules that play critical roles in plant developmental processes and root biotic interactions. Strigolactones are agronomically important due to their role as a signal for the germination of a parasitic weed (Striga spp.) that reduces yields of cereal crops worldwide. To identify the genes encoding strigolactones in sorghum and their function, we characterized two CRISPR/Cas9-mediated gene knockouts of carotenoid cleavage dioxygenase 8 (CCD8) genes (SbCCD8a and SbCCD8b), which have been shown in other plant species to be involved in strigolactone biosynthesis. Although strigolactones are important for the parasitization of sorghum in Africa, the functions of members of the CCD8 family have not been characterized. The impact of the knockouts on strigolactone production, plant growth and development, resistance to the parasitic weed Striga, and the root-associated microbiomes were investigated in this study. The results revealed that knockout of SbCCD8 genes in sorghum significantly reduced orobanchol production and Striga germination. Strigolactone deficiency altered the shoot and root architecture and reduced grain yield of sorghum. The knockout of the SbCCD8b gene significantly affected the rhizosphere bacterial diversity and community composition at sorghum plant grain-fill stage due to the abolition of orobanchol exudation from roots. Reduced amounts of orobanchol in root exudates also influenced root-associated fungal taxa abundance. Our findings provide new insights into potentially sustainable approaches for the recruitment of beneficial microbes and for parasitic weed control through manipulation of strigolactone production in sorghum.

Published
2023
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11. Impact of HeartWare ventricular assist device discontinuation on the pediatric population: An Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry analysis

Author

Niebler, Robert A., Shezad, Muhammad, Absi, Mohammed, Aljohani, Othman A., Amdani, Shahnawaz, Auerbach, Scott, Bearl, David W., Boucek, Katerina, Buchholz, Holger, Castleberry, Chesney, Dykes, John, Engelhardt, Kevin, Friedland-Little, Joshua, Hope, Kyle, Iqbal, Mehreen, Jeewa, Aamir, Joong, Anna, Lorts, Angela, May, Lindsay, Mokshagundam, Deepa, Nandi, Deipanjan, O'Connor, Matthew, Parent, John J., Peng, David M., Shwaish, Natalie, Shugh, Svetlana B., VanderPluym, Christina, Vaughn, Gabrielle, and Zinn, Mattew

Published
2024
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14. Multicenter outcomes for ventricular assist device support for failed stage II palliation

Author

Rabinowitz, Edon J., Mehegan, Mary, Joong, Anna, Shezad, Muhammad, Lorts, Angela, Villa, Chet R., Conway, Jennifer, Kobayashi, Ryan, Auerbach, Scott R., Zinn, Matthew, Niebler, Robert, Iqbal, Mehreen, Dykes, John, Choudhry, Swati, Aljohani, Othman, Absi, Mohammed, Ploutz, Michelle S., Griffiths, Eric R., O’Connor, Matthew J., Mokshagundam, Deepa, and Said, Ahmed S.

Published
2024
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15. Multicenter outcomes for ventricular assist device support for failed stage II palliation

Author

Edon J. Rabinowitz, MD, Mary Mehegan, RN, Anna Joong, MD, Muhammad Shezad, MHSA, Angela Lorts, MD, Chet R. Villa, MD, Jennifer Conway, MD, MSc, Ryan Kobayashi, MD, Scott R. Auerbach, MD, Matthew Zinn, DO, Robert Niebler, MD, Mehreen Iqbal, MD, John Dykes, MD, Swati Choudhry, MD, Othman Aljohani, MD, Mohammed Absi, MD, Michelle S. Ploutz, MD, Eric R. Griffiths, MD, Matthew J. O’Connor, MD, Deepa Mokshagundam, MD, and Ahmed S. Said, MD, PhD

Subjects
ventricular assist device, stage 2 palliation, Glenn, transplantation, pediatrics, single ventricle, Surgery, RD1-811, Specialties of internal medicine, RC581-951
Abstract

Background: Ventricular assist device (VAD) use for failed stage II palliation (S2P) is increasing with limited data on outcomes. Methods: To address this knowledge gap, we conducted a multicenter retrospective review of the Advanced Cardiac Therapies Improving Outcomes Network registry. We leveraged the registry to analyze data on the clinical course, complications, and survival of systemic VADs (SVAD) after S2P. Results: We identified 34 patients from 15 centers between 2012 and 2022 implanted at median age of 1.8 years [interquartile range (IQR) 0.9-2.7]; 85% had systemic right ventricles and all patients underwent at least one sternotomy. Preimplant, all but 1 patient had an Interagency Registry for Mechanically Assisted Circulatory Support profile of 1-2, with 62% being on ≥2 inotropes, 50% total parenteral nutrition dependent, 38% mechanically ventilated, and 20% on extracorporeal membrane oxygenation in the week preceding implant. Device strategy was variable with 70% being on continuous flow devices and the remaining on pulsatile support. Multiple device strategies were utilized in 32% of patients. Median time on VAD support was 74 days [IQR 30-186]. Adverse events were frequent and included infection (47%), strokes (29%), bleeding (26%), dialysis (15%), and respiratory failure (9%). Bleeding complications (p = 0.0004), respiratory failure (p = 0.04), and multiple inotropes preimplant (p = 0.046) were associated with in-hospital mortality. Overall survival to transplant/recovery was seen in 76% and to 1-year postexplant in 56%. Conclusion: Encouraging clinical outcomes are seen with SVAD use for failed S2P even in the face of frequent adverse events and wide center variability in device strategy. Ongoing multi-institutional collaboration is required to better understand optimal SVAD support strategies.

Published
2024
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16. Widening care gap in VAD therapy

Author

Conway, Jennifer, Amdani, Shahnawaz, Morales, David L.S., Lorts, Angela, Rosenthal, David N., Jacobs, Jeffrey P., Rossano, Joseph, Koehl, Devin, Kirklin, James K., MD, and Auerbach, Scott R.

Published
2023
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17. Clinical Outcomes of SARS-CoV-2 Infection in Pediatric Patients on Ventricular Assist Device Support: An ACTION Registry Analysis

Author

Torpoco Rivera, Diana M., Joong, Anna, D’Souza, Nikita, Duganiero, Toni, Lorts, Angela, Spinner, Joseph A., Lynch, Aine, Kamsheh, Alicia M., Sheybani, Aryaz, Blanco, Carlos, Tolani, Drishti, Hayes, Emily, Tadros, Hanna J., Masood, Imran R., Boucek, Katerina, Radel, Laura, Townsend, Madeleine, Varma, Manu, Kobayashi, Ryan, and Gupta, Tripti

Published
2024
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18. Abstract 15192: The Impact of Fontan Circulatory Failure on Heart Transplant Survival: A 20 Center Study

Author

Schumacher, Kurt R, Rosenthal, David N, Batazzi, Adriana, Yu, Sunkyung, Reichle, Garrett, Bano, Maria, Deshpande, Shriprasad R, OʼConnor, Matthew, Ahmed, Humera, Chen, Sharon, Wright, Lydia, Kindel, Steven J, Joong, Anna, Ploutz, Michelle, Feingold, Brian D, Godown, Justin, Mao, Chad Y, Lorts, Angela, Simpson, Kathleen, Ybarra, Aecha, Richmond, Marc E, Amdani, Shahnawaz, Conway, Jennifer, Blume, Elizabeth, and Cousino, Melissa

Published
2023
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21. Expanding use of the HeartMate 3 ventricular assist device in pediatric and adult patients within the Advanced Cardiac Therapies Improving Outcomes Network (ACTION)

Author

O’Connor, Matthew J., Shezad, Muhammad, Ahmed, Humera, Amdani, Shahnawaz, Auerbach, Scott R., Bearl, David W., Butto, Arene, Byrnes, Jonathan W., Conway, Jennifer, Dykes, John C., Glass, Lauren, Lantz, Jodie, Law, Sabrina, Mongé, Michael C., Morales, David L.S., Parent, John J., Peng, David M., Ploutz, Michelle S., Puri, Kriti, Shugh, Svetlana, Shwaish, Natalie S., VanderPluym, Christina J., Wilkens, Sarah, Wright, Lydia, Zinn, Matthew D., and Lorts, Angela

Published
2023
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23. Global best practices consensus: Long-term management of patients with hybrid centrifugal flow left ventricular assist device support

Author

Hayward, Christopher, Adachi, Iki, Baudart, Sylvie, Davis, Erin, Feller, Erika D., Kinugawa, Koichiro, Klein, Liviu, Li, Song, Lorts, Angela, Mahr, Claudius, Mathew, Jacob, Morshuis, Michiel, Müller, Marcus, Ono, Minoru, Pagani, Francis D., Pappalardo, Federico, Rich, Jonathan, Robson, Desiree, Rosenthal, David N., Saeed, Diyar, Salerno, Christopher, Sauer, Andrew J., Schlöglhofer, Thomas, Tops, Laurens, and VanderPluym, Christina

Published
2022
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26. Pediatric Learning Health Networks in Solid Organ Transplantation—Engaging all Stakeholders to Achieve Health for Children Who Require Transplantation.

Author

George, Roshan P., Feldman, Amy G., McQueen, Melissa, Krise‐Confair, Cassandra, Smyth, Lauren, Lorts, Angela, Peng, David M., Mazariegos, George V., and Hooper, David K.

Subjects
HEALTH care networks, LIVER transplantation, KIDNEY transplantation, HEART failure, KIDNEY failure
Abstract

Background: Learning Health Networks (LHN) have evolved within medicine over the past two decades, but their integration into transplantation has been more recent. Objectives and Methods: In this paper, we describe three LHNs in end‐stage organ disease/transplantation, their common and unique features, and how their "actor‐oriented" architecture allowed for rapid adaptation to meet the needs of their patients and practitioners during the recent COVID‐19 pandemic. Result: The structure and focus of the Improving Renal Outcomes Collaborative (IROC), Starzl Network for Excellence in Pediatric Transplantation (SNEPT), and the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) are reviewed. We discuss the critical role of patient and family engagement, focusing on collaboration with Transplant Families. Finally, we review challenges common to the LHN concept and potential common areas of alignment to achieve the goal of more rapid and sustained progress to improve health in pediatric transplantation. Conclusion: LHN in transplantation are essential to accelerate knowledge dissemination and improve outcomes. [ABSTRACT FROM AUTHOR]

Published
2024
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27. Sepsis

Author

Carlton, Erin, Lorts, Angela, Shanley, Thomas P., Cornell, Timothy T., Lucking, Steven E., editor, Maffei, Frank A., editor, Tamburro, Robert F., editor, and Zaritsky, Arno, editor

Published
2021
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28. Impact of Weight on Ventricular Assist Device Outcomes in Dilated Cardiomyopathy Patients in Pediatric Centers: An ACTION Registry Study

Author

Kwiatkowski, David M., Shezad, Muhammad, Barnes, Aliessa P., Ploutz, Michelle S., Law, Sabrina P., Zafar, Farhan, Morales, David L.S., O’Connor, Matthew J., Ahmed, Humera, Aljohani, Othman A., Auerbach, Scott R., Bansal, Neha, Bearl, David W, Brickler, Molly, Buchholz, Holger, Byrnes, Jonathan W., Carrington, Sydney, Chandnani, Harsha K., Conway, Jennifer L., Do, Nhue L., Dykes, John C., Honjo, Osami, Jeewa, Aamir, Joong, Anna, Kindel, Steven J., Kleinmahon, Jake A, Lantz, Jodie, Lorts, Angela, Maeda, Katsuhide, Mao, Chad Y., Mauchley, David C., May, Lindsay, McCormick, Amanda D., Nandi, Deipanjan, Peng, David M., Rosenthal, David N., Shugh, Svetlana, Simpson, Kathleen E., Watanbe, Kae, Wilkens, Sarah J., Wright, Lydia K., and Zinn, Matthew D.

Published
2023
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30. Patient-Reported Outcomes in Pediatric VAD Recipients: ACTION Findings Stratified by Device

Author

May, L., primary, Cousino, M., additional, Smyth, L., additional, McQueen, M., additional, Hunter, T., additional, Ventresco, C., additional, Fields, K., additional, Murray, J., additional, Machado, D., additional, Shezad, M., additional, Rosenthal, D., additional, Engelhardt, K., additional, Mehegan, M., additional, Mokshagundam, D., additional, Ploutz, M., additional, Lorts, A., additional, and Blume, E., additional

Published
2024
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31. Collective Five Year ACTION VAD Registry Experience

Author

Edelson, J., primary, Raskin, A., additional, Absi, M., additional, Bearl, D., additional, Bonilla Ramirez, C., additional, Do, N., additional, Knoll, C., additional, Lorts, A., additional, Martinez, H., additional, Maeda, K., additional, O'Connor, M., additional, Rosenthal, D., additional, Shezad, M., additional, Wisotzkey, B., additional, and Niebler, R., additional

Published
2024
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35. Explantation of Durable Ventricular Assist Device for Myocardial Functional Recovery in Children: A Report from The Action Registry

Author

Langanecha, B., primary, Jeewa, A., additional, Adachi, I., additional, Mavroudis, C., additional, Shezad, M., additional, Butts, R., additional, Auerbach, S.R., additional, VanderPluym, C., additional, Zinn, M., additional, Kindel, S., additional, Sutcliffe, D., additional, Khan, S., additional, Byrnes, J., additional, Lorts, A., additional, Castleberry, C., additional, Mettler, B., additional, Absi, M., additional, Rosenthal, D., additional, Joong, A., additional, Law, S., additional, Nandi, D., additional, May, L., additional, Parent, J., additional, Conway, J., additional, Tunuguntla, H., additional, Aljohani, O., additional, Bleiweis, M., additional, O'Connor, M., additional, and Hope, K., additional

Published
2024
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36. The Impact of Resistance Training on Body Composition, Muscle Strength, and Functional Fitness in Older Women (45–80 Years): A Systematic Review (2010–2020)

Author

Lynda B. Ransdell, Heidi A. Wayment, Nanette Lopez, Cori Lorts, Anna L. Schwartz, Karen Pugliesi, Patricia S. Pohl, Dierdra Bycura, and Ricky Camplain

Subjects
weight training, strength training, peri-menopause, post-menopause, intervention, middle-aged women, Medicine, Psychology, BF1-990
Abstract

As women age, they typically experience a progressive decrease in skeletal muscle mass and strength, which can lead to a decline in functional fitness and quality of life. Resistance training (RT) has the potential to attenuate these losses. Although well established for men, evidence regarding the benefits of RT for women is sparse and inconsistent: prior reviews include too few studies with women and do not adequately examine the interactive or additive impacts of workload, modalities, and nutritional supplements on outcomes such as muscle mass (MM), body composition (BC), muscle strength (MS), and functional fitness (FF). The purpose of this review is to identify these gaps. Thirty-eight papers published between 2010 and 2020 (in English) represent 2519 subjects (mean age = 66.89 ± 4.91 years). Intervention averages include 2 to 3 × 50 min sessions across 15 weeks with 7 exercises per session and 11 repetitions per set. Twelve studies (32%) examined the impact of RT plus dietary manipulation. MM, MS, and FF showed positive changes after RT. Adding RT to fitness regimens for peri- to postmenopausal women is likely to have positive benefits.

Published
2021
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39. Genomics of sorghum local adaptation to a parasitic plant

Author

Bellis, Emily S., Kelly, Elizabeth A., Lorts, Claire M., Gao, Huirong, DeLeo, Victoria L., Rouhan, Germinal, Budden, Andrew, Bhaskara, Govinal B., Hu, Zhenbin, Muscarella, Robert, Timko, Michael P., Nebie, Baloua, Runo, Steven M., Chilcoat, N. Doane, Juenger, Thomas E., Morris, Geoffrey P., dePamphilis, Claude W., and Lasky, Jesse R.

Published
2020

42. Definitions of adverse events associated with extracorporeal membrane oxygenation in children: results of an international Delphi process from the ECMO-CENTRAL ARC

Author

Alexander, Peta M A, Di Nardo, Matteo, Combes, Alain, Vogel, Adam M, Antonini, Marta Velia, Barrett, Nicholas, Benedetti, Giulia M, Bettencourt, Amanda, Brodie, Daniel, Gómez-Gutiérrez, René, Gorga, Stephen M, Hodgson, Carol, Kapoor, Poonam Malhotra, Le, Jennifer, MacLaren, Graeme, O’Neil, Erika R, Ostermann, Marlies, Paden, Matthew L, Patel, Neil, Rojas-Peña, Alvaro, Said, Ahmed S, Sperotto, Francesca, Willems, Ariane, Vercaemst, Leen, Yoganathan, Ajit P, Lorts, Angela, del Nido, Pedro J, Barbaro, Ryan P, Abecasis, Francisco, Al Foudri, Huda, Anders, Marc M., Angus, Derek C., Annich, Gail M., Arachchillage, Deepa J., Akcan-Arikan, Ayse, Armstrong, Aimee K., Balcells, Joan, Barrett, Meredith, Bartlett, Robert H., Belda-Hofheinz, Sylvia, Bembea, Melania M., Best, Derek J., Bonadonna, Desiree, Boonstra, Philip S., Brogan, Thomas V, Brown, Katherine L, Butragueño-Laiseca, Laura, Butt, Warwick W., Camporota, Luigi, Cardenas, Barbara, Cascarano, Maria T., Caspi, Joseph, Chen, Eric, Chiletti, Roberto, Colombo, Giovanna G., Conte, Marisa L., Crowe, Liz, Cvetkovic, Mirjana, Dalton, Heidi J., Daverio, Marco, Davidson, Mark G., Davis, Carl, DiGeronimo, Robert J., Egbunine, Ariel, Ellis, W. Cory, Fan, Eddy, Foster, Claire C, Fraser, John F., Friedman, Matthew L., Furlong-Dillard, Jamie, Fynn-Thompson, Francis, Garcia, Alejandro V., Geppert, Jeffery, Gillette, Nicole, Grazioli, Lorenzo, Greene, Christina L, Guerguerian, Anne-Marie, Guilliams, Kristin P., Haji Fortis, Jumana Y., Hao, Jianming, Heinsar, Silver, Hejduk, Mark W., Himebauch, Adam S., Horvat, Christopher M., Hoskote, Aparna, Hou, Xiaotong, Hyslop, Robert W., Ibrahim, Nicole, Ito, Takuya, Karagiannidis, Christian, Karam, Oliver, Kaushik, Shubhi, Kilbaugh, Todd J., Kim-Campbell, Nahmah, Kirsch, Roxanne E, Kormos, Robert L., Kornberger, Angela, Krucoff, Mitchell W., Lakshminrusimha, Satyan, LaRovere, Kerri L, Leger, Pierre-Louis, Levy, Philip T., Lorusso, Roberto, Lyster, Haifa, Malone, Matthew P., McMullan, D. Michael, Mečířová, Soña, Mehran, Roxana, Merz, Scott I., Mochizuki, Shuichi, Moler, Frank W., Molla, Kalkidan, Monagle, Paul, Morgan, Gareth J., Morgan, Catherine, Morice, Marie-Claude, Muszynski, Jennifer A., Najaf, Tasnim A., Nellis, Marianne E., Newburger, Jane W., O'Rourke, P. Pearl, Ostadal, Petr, Ozment, Caroline P., Pagani, Francis D., Peek, Giles J., Polito, Angelo, Priest, John R., Raman, Lakshmi, Rintoul, Natalie E., Roy, Nathalie, Rubin, Juli, Rumford, Martha, Rycus, Peter, Ryerson, Lindsay M., Sakamoto, Wakako, Segura-Matute, Susana, Shekar, Kiran, Spinella, Philip C., Spitzer, Ernest, Starr, Joanne P., Stead, Christine, Steffen, Katherine, Steiner, Marie E., Tan, Lucy, Tasker, Robert C., Taylor, J. Michael, Thiagarajan, Ravi R., Tonna, Joseph E., Ubertini, Gina M., van Leeuwen, Grace, Vang, Eric, Ventetuolo, Corey E., Venugopal, Prem S., Weinberg, Allison, Westrope, Claire, Wu, Changfu, Wylie, Gillian, Zeymer, Uwe, Zivick, Elizabeth E., and Zochios, Vasileios

Abstract

Extracorporeal membrane oxygenation (ECMO) is a high-risk and low-volume life support with increasing clinical study. However, heterogenous outcome definitions impede data assimilation into evidence to guide practice. The Academic Research Consortium (ARC), an international collaborative forum committed to facilitating the creation of stakeholder-driven consensus nomenclature and outcomes for clinical trials of medical devices, supported the ECMO Core Elements Needed for Trials Regulation And quality of Life (ECMO-CENTRAL) ARC. The ECMO-CENTRAL ARC was assembled to develop definitions of paediatric ECMO adverse events for use in clinical trials and regulatory device evaluation. An initial candidate list of ECMO adverse events derived from the mechanical circulatory support ARC was supplemented with a review of ECMO-relevant adverse event definitions collated from literature published between Jan 1, 1988, and Feb 20, 2023. Distinct teams of international topic experts drafted separate adverse event definitions that were harmonised to existing literature when appropriate. Draft definitions were revised for paediatric ECMO relevance with input from patients, families, and an international expert panel of trialists, clinicians, statisticians, biomedical engineers, device developers, and regulatory agencies. ECMO-CENTRAL ARC was revised and disseminated across research societies and professional organisations. Up to three rounds of internet-based anonymous surveys were planned as a modified Delphi process. The expert panel defined 13 adverse event definitions: neurological, bleeding, device malfunction, acute kidney injury, haemolysis, infection, vascular access-associated injury, non-CNS thrombosis, hepatic dysfunction, right heart failure, left ventricular overload, lactic acidaemia, and hypoxaemia. Definitional structure varied. Among 165 expert panel members, 114 were eligible to vote and 111 voted. Consensus was achieved for all proposed definitions. Agreement ranged from 82% to 95%. ECMO-CENTRAL ARC paired rigorous development with methodical stakeholder involvement and dissemination to define paediatric ECMO adverse events. These definitions will facilitate new research and the assimilation of data across clinical trials and ECMO device evaluation in children.

Published
2024
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43. The genomics and physiology of abiotic stressors associated with global elevational gradients in Arabidopsis thaliana.

Author

Gamba, Diana, Lorts, Claire M., Haile, Asnake, Sahay, Seema, Lopez, Lua, Xia, Tian, Takou, Margarita, Kulesza, Evelyn, Elango, Dinakaran, Kerby, Jeffrey, Yifru, Mistire, Bulafu, Collins E., Wondimu, Tigist, Glowacka, Katarzyna, and Lasky, Jesse R.

Subjects
*LOCUS (Genetics), *WATER efficiency, *ARABIDOPSIS thaliana, *PARTIAL pressure, *GENOMICS
Abstract

Summary: Phenotypic and genomic diversity in Arabidopsis thaliana may be associated with adaptation along its wide elevational range, but it is unclear whether elevational clines are consistent among different mountain ranges.We took a multi‐regional view of selection associated with elevation. In a diverse panel of ecotypes, we measured plant traits under alpine stressors (low CO2 partial pressure, high light, and night freezing) and conducted genome‐wide association studies.We found evidence of contrasting locally adaptive regional clines. Western Mediterranean ecotypes showed low water use efficiency (WUE)/early flowering at low elevations to high WUE/late flowering at high elevations. Central Asian ecotypes showed the opposite pattern. We mapped different candidate genes for each region, and some quantitative trait loci (QTL) showed elevational and climatic clines likely maintained by selection. Consistent with regional heterogeneity, trait and QTL clines were evident at regional scales (c. 2000 km) but disappeared globally. Antioxidants and pigmentation rarely showed elevational clines. High elevation east African ecotypes might have higher antioxidant activity under night freezing.Physiological and genomic elevational clines in different regions can be unique, underlining the complexity of local adaptation in widely distributed species, while hindering global trait–environment or genome–environment associations. To tackle the mechanisms of range‐wide local adaptation, regional approaches are thus warranted. [ABSTRACT FROM AUTHOR]

Published
2024
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45. Abstract 12970: Predictors of Stroke and Mortality in Children Who Undergo Ventricular Assist Device Placement: An Evaluation of Pre And Post-Operative Risk Factors Linked From the Advanced Cardiac Therapies Improving Outcomes Network (action) and Pediatric Cardiac Critical Care Consortium (pc4) Registries

Author

Perry, Tanya, Rosenthal, David N, Lorts, Angela, Zafar, Farhan, Zhang, Wenying, Vanderpluym, Christina J, DeWitt, Aaron G, Reichle, Garrett, Banerjee, Mousumi, and Schumacher, Kurt

Published
2022
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47. Early experience with the HeartMate 3 continuous-flow ventricular assist device in pediatric patients and patients with congenital heart disease: A multicenter registry analysis

Author

O'Connor, Matthew J., Lorts, Angela, Davies, Ryan R., Fynn-Thompson, Francis, Joong, Anna, Maeda, Katsuhide, Mascio, Christopher E., McConnell, Patrick I., Mongé, Michael C., Nandi, Deipanjan, Peng, David M., Rosenthal, David N., Si, Ming-Sing, Sutcliffe, David L., VanderPluym, Christina J., Viegas, Melita, Zafar, Farhan, Zinn, Matthew, and Morales, David L.S.

Published
2020
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50. Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.

Author

Hathout, Yetrib, Brody, Edward, Clemens, Paula, Cripe, Linda, DeLisle, Robert, Furlong, Pat, Gordish-Dressman, Heather, Hache, Lauren, Henricson, Erik, Hoffman, Eric, Kobayashi, Yvonne, Lorts, Angela, Mah, Jean, Mehler, Bob, Nelson, Sally, Nikrad, Malti, Singer, Britta, Steele, Fintan, Sterling, David, Sweeney, H, Williams, Steve, Gold, Larry, and McDonald, Craig

Subjects
SOMAmer, SOMAscan, biomarkers, muscular dystrophy, proteomics, Adolescent, Adult, Biomarkers, Blood Proteins, Case-Control Studies, Child, Child, Preschool, Cohort Studies, Humans, Male, Muscular Dystrophy, Duchenne, Young Adult
Abstract

Serum biomarkers in Duchenne muscular dystrophy (DMD) may provide deeper insights into disease pathogenesis, suggest new therapeutic approaches, serve as acute read-outs of drug effects, and be useful as surrogate outcome measures to predict later clinical benefit. In this study a large-scale biomarker discovery was performed on serum samples from patients with DMD and age-matched healthy volunteers using a modified aptamer-based proteomics technology. Levels of 1,125 proteins were quantified in serum samples from two independent DMD cohorts: cohort 1 (The Parent Project Muscular Dystrophy-Cincinnati Childrens Hospital Medical Center), 42 patients with DMD and 28 age-matched normal volunteers; and cohort 2 (The Cooperative International Neuromuscular Research Group, Duchenne Natural History Study), 51 patients with DMD and 17 age-matched normal volunteers. Forty-four proteins showed significant differences that were consistent in both cohorts when comparing DMD patients and healthy volunteers at a 1% false-discovery rate, a large number of significant protein changes for such a small study. These biomarkers can be classified by known cellular processes and by age-dependent changes in protein concentration. Our findings demonstrate both the utility of this unbiased biomarker discovery approach and suggest potential new diagnostic and therapeutic avenues for ameliorating the burden of DMD and, we hope, other rare and devastating diseases.

Published
2015

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