Search

Your search keyword '"Richard K. Vehe"' showing total 31 results
Start Over Author "Richard K. Vehe" Publication Year Range Last 10 years
31 results on '"Richard K. Vehe"'

1. Use of disease assessment tools to increase the value of case reports on Susac syndrome: two case reports

Author

Danielle R. Bullock, Robert T. Spencer, Richard K. Vehe, Sunil Srivastava, and Robert M. Rennebohm

Subjects
Susac syndrome, Disease assessment tools, Clinical course, Outcome, Case report, Medicine
Abstract

Abstract Background Susac syndrome is an immune-mediated, ischemia-producing, occlusive microvascular endotheliopathy that threatens the brain, retina, and inner ear. There is a need for disease assessment tools that can help clinicians and patients to more easily, accurately, and uniformly track the clinical course and outcome of Susac syndrome. Ideally, such tools should simultaneously facilitate the clinical care and study of Susac syndrome and improve the value of future case reports. To meet this need, two novel clinical assessment tools were developed: the Susac Symptoms Form and the Susac Disease Damage Score. The former is a comprehensive self-report form that is completed by patients/families to serially document the clinical status of a patient. The latter documents the extent of damage perceived by individual patients/families and their physicians. Both forms were initially trialed with two particularly representative and instructive patients. The results of this trial are shared in this report. Case presentation Patient 1 is a 21-year-old Caucasian female who presented with an acute onset of headache, paresthesias, cognitive dysfunction, and emotional lability. Patient 2 is a 14-year-old Caucasian female who presented with an acute onset of headache, cognitive dysfunction, urinary incontinence, ataxia, and personality change. Both patients fulfilled criteria for a definite diagnosis of Susac syndrome: both eventually developed brain, retinal, and inner ear involvement, and both had typical “snowball lesions” on magnetic resonance imaging. The Susac Symptoms Form documented initial improvement in both patients, was sufficiently sensitive in detecting a subsequent relapse in the second patient, and succinctly documented the long-term clinical course in both patients. The Disease Damage Score documented minimal disease damage in the first patient and more significant damage in the second. Conclusions The Susac Symptoms Form and the Disease Damage Score are useful disease assessment tools, both for clinical care and research purposes. Their use could enhance the value of future case reports on Susac syndrome and could improve opportunities to learn from a series of such reports.

Published
2023
Full Text
View/download PDF

2. Capturing critical data elements in Juvenile Idiopathic Arthritis: initiatives to improve data capture

Author

Meghan E. Ryan, Andrew Warmin, Bryce A. Binstadt, Colleen K. Correll, Emily Hause, Patricia Hobday, Alison Lerman, Shawn A. Mahmud, Mona M. Riskalla, Zachary Shaheen, Richard K. Vehe, Danielle R. Bullock, the Pediatric Rheumatology Care, and Outcomes Improvement Network

Subjects
Juvenile idiopathic arthritis, Quality Improvement, Data Collection, Virtual visits, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background Documentation of critical data elements is a focus of the Pediatric Rheumatology Care and Outcomes Improvement Network to aid in clinical care and research for patients with juvenile idiopathic arthritis. We aimed to increase data capture for critical data elements and hypothesized that quality improvement methodology would improve data capture. We also hypothesized that data capture for all critical data elements would be lower for virtual visits compared to in-person visits. Methods All visits for patients with JIA between 9/14/2020 and 12/31/2021 at the University of Minnesota were included. We assessed completeness of critical data element capture. Sixteen interventions with providers were conducted, including email reminders, individual discussions, group meetings, and feedback reports. We used statistical process control charts to evaluate change over time. Results Baseline included 355 patient-visits: 221 (62%) in-person and 134 (38%) virtual with critical data elements entry ranging between 50 and 60%. Post-intervention included 1,596 patient-visits: 1,350 (85%) in-person and 246 (15%) virtual, with critical data elements entry reaching 91%. All providers improved data entry during this study. In-person visits had significantly higher data capture rates than virtual visits for all 4 critical data elements. Conclusion We achieved our aim to increase critical data element documentation by focusing on provider buy-in, frequent reminders, and individualized feedback. We also found that collection of critical data elements occurred significantly less often with virtual visits than with in-person visits. Now that we improved capture of critical data elements, we can shift the focus to efforts aimed at improving outcomes for patients with juvenile arthritis.

Published
2022
Full Text
View/download PDF

3. Non-criteria antiphospholipid antibodies and pediatric rheumatic disease: a case series

Author

Shawn A. Mahmud, Danielle R. Bullock, Colleen K. Correll, Patricia M. Hobday, Mona M. Riskalla, Richard K. Vehe, and Bryce A. Binstadt

Subjects
Non-criteria antiphospholipid antibodies, Antiphospholipid syndrome, Rheumatic disease, Lupus anticoagulant, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background Non-criteria antiphospholipid antibodies (NC-aPL) are a relatively undefined subgroup of antiphospholipid antibodies (aPL). Knowledge about NC-aPL in adults is limited and even less is known in pediatric patients. Routine tests for antiphospholipid syndrome (APS)—a clinical state marked by the presence of aPL in association with vascular thrombosis—usually include lupus anticoagulant (LAC), anti-cardiolipin (aCL) and -beta-2 glycoprotein I (aβ2GPI). LAC is a functional screen for prothrombotic aPL, while the latter tests identify specific autoantibodies. Specific targets of NC-aPL include, but are not limited to, phosphatidylethanolamine, phosphatidylserine, and prothrombin. Presentation of cases We present single-center data from eight pediatric patients with NC-aPL identified during a three-year period. All patients had presenting features raising suspicion for APS. Most patients were female with a primary rheumatic disease. One patient had a stroke. Another patient had alveolar hemorrhage and pulmonary hypertension. Raynaud’s phenomenon, rashes involving distal extremities, and headaches were common. Most patients had a positive LAC, yet their routine aPL tests were negative, prompting testing for NC-aPL. Conclusions Our findings suggest NC-aPL are associated with typical signs and symptoms of APS in pediatric patients. Pediatricians and pediatric subspecialists should consider NC-aPL when clinical suspicion is high and routine aPL tests are negative, particularly when LAC is positive. While guidelines for NC-aPL do not yet exist for children or adults, these autoantibodies have pathogenic potential. Actionable items could include evaluation for the presence of other (primary) rheumatic diseases, and consultation with hematologists and/or obstetricians regarding anticoagulation/platelet inhibition and thrombosis education. Future guidelines regarding NC-aPL will only be generated by gathering more data, ideally prospectively.

Published
2022
Full Text
View/download PDF

4. Translating research into practice—implementation recommendations for pediatric rheumatology; Proceedings of the childhood arthritis and rheumatology research alliance 2020 implementation science retreat

Author

Cagri Yildirim-Toruner, Rajdeep Pooni, Y. Ingrid Goh, Emily Becker-Haimes, James W. Dearing, Maria E. Fernandez, Esi M. Morgan, Gareth Parry, Jon M. Burnham, Stacy P. Ardoin, Fatima Barbar-Smiley, Joyce C. Chang, Peter Chiraseveenuprapund, Vincent Del Gaizo, Guy Eakin, Lisa C. Johnson, Yukiko Kimura, Andrea M. Knight, Melanie Kohlheim, Erica F. Lawson, Mindy S. Lo, Nancy Pan, Andrea Ring, Tova Ronis, Rebecca E. Sadun, Emily A. Smitherman, Alysha J. Taxter, Janalee Taylor, Richard K. Vehe, Sheetal S. Vora, Jennifer E. Weiss, Emily von Scheven, and MAS for the CARRA Implementation Science Workgroup

Subjects
Implementation sciences, Knowledge translation, Dissemination, Implementation, Strategy, Framework, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract The translation of research findings into clinical practice is challenging, especially fields like in pediatric rheumatology, where the evidence base is limited, there are few clinical trials, and the conditions are rare and heterogeneous. Implementation science methodologies have been shown to reduce the research- to- practice gap in other clinical settings may have similar utility in pediatric rheumatology. This paper describes the key discussion points from the inaugural Childhood Arthritis and Rheumatology Research Alliance Implementation Science retreat held in February 2020. The aim of this report is to synthesize those findings into an Implementation Science Roadmap for pediatric rheumatology research. This roadmap is based on three foundational principles: fostering curiosity and ensuring discovery, integration of research and quality improvement, and patient-centeredness. We include six key steps anchored in the principles of implementation science. Applying this roadmap will enable researchers to evaluate the full range of research activities, from the initial clinical design and evidence acquisition to the application of those findings in pediatric rheumatology clinics and direct patient care.

Published
2022
Full Text
View/download PDF

5. T cell-intrinsic role for Nod2 in protection against Th17-mediated uveitis

Author

Ruth J. Napier, Ellen J. Lee, Michael P. Davey, Emily E. Vance, João M. Furtado, Paige E. Snow, Kimberly A. Samson, Sydney J. Lashley, Brieanna R. Brown, Reiko Horai, Mary J. Mattapallil, Biying Xu, Michelle C. Callegan, Luke S. Uebelhoer, Christina L. Lancioni, Richard K. Vehe, Bryce A. Binstadt, Justine R. Smith, Rachel R. Caspi, and Holly L. Rosenzweig

Subjects
Science
Abstract

How mutations in the microbial receptor NOD2 induce Blau syndrome in humans and related uveitis is unclear. Here the authors show, using Nod2-deficient mice and experimental uveitis, that Nod2 negatively regulates T cell activation and transcription of autoimmunity-related genes to suppress Th17 responses and uveitis.

Published
2020
Full Text
View/download PDF

6. Physician practices for withdrawal of medications in inactive systemic juvenile arthritis, Childhood Arthritis and Rheumatology Research Alliance (CARRA) survey

Author

Susan Shenoi, Kabita Nanda, Grant S. Schulert, John F. Bohnsack, Ashley M. Cooper, Bridget Edghill, Miriah C. Gillispie-Taylor, Baruch Goldberg, Olha Halyabar, Thomas G. Mason, Tova Ronis, Rayfel Schneider, Richard K. Vehe, Karen Onel, and for the Childhood Arthritis and Rheumatology Research Alliance Systemic Juvenile Idiopathic Arthritis Workgroup

Subjects
Systemic Juvenile Idiopathic Arthritis, Inactive disease, Withdrawal of medications, CARRA, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background We describe a Childhood Arthritis and Rheumatology Research Alliance (CARRA) survey of North American pediatric rheumatologists that assesses physician attitudes on withdrawal of medications in systemic juvenile idiopathic arthritis (SJIA). Methods A REDCap anonymous electronic survey was distributed to 100 random CARRA JIA workgroup physician-voting members. The survey had three broad sections including: A) demographic information; B) physicians’ opinions on clinical inactive disease (CID) in SJIA and C) existing practices for withdrawing medications in SJIA. Results The survey had an 86% response rate. 88 and 93% of participants agreed with the current criteria for CID and clinical remission on medications (CRM) respectively. 78% thought it necessary to meet CRM before tapering medications except steroids. 76% use CARRA SJIA consensus treatment plans always or the majority of the time. All participants weaned steroids first in SJIA patients on combination therapy, 47% waited > 6 months before tapering additional medications. 35% each tapered methotrexate over > 6 months and 2–6 months; however, 39% preferred tapering anakinra, canakinumab and tocilizumab more quickly over 2–6 months and favored spacing the dosing interval for canakinumab and tocilizumab. When patients are on combination therapy with methotrexate and biologics, 58% preferred tapering methotrexate first while others considered patient/family preference and adverse effects to guide their choice. Conclusion Most CARRA members surveyed use published consensus treatment plans for SJIA and agree with validated definitions of CID and CRM. There was agreement with tapering steroids first in SJIA. There was considerable variability with tapering decisions of all other medications.

Published
2019
Full Text
View/download PDF

7. Pharmacokinetic and safety profile of tofacitinib in children with polyarticular course juvenile idiopathic arthritis: results of a phase 1, open-label, multicenter study

Author

Nicolino Ruperto, Hermine I. Brunner, Zbigniew Zuber, Nikolay Tzaribachev, Daniel J. Kingsbury, Ivan Foeldvari, Gerd Horneff, Elzbieta Smolewska, Richard K. Vehe, Anasuya Hazra, Rong Wang, Charles A. Mebus, Christine Alvey, Manisha Lamba, Sriram Krishnaswami, Thomas C. Stock, Min Wang, Ricardo Suehiro, Alberto Martini, Daniel J. Lovell, for the Pediatric Rheumatology International Trials Organization (PRINTO), and the Pediatric Rheumatology Collaborative Study Group (PRCSG)

Subjects
Janus kinase inhibitor, Juvenile idiopathic arthritis, Tofacitinib, Pediatric, Pharmacokinetics, Safety, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background Juvenile idiopathic arthritis (JIA) is the most common pediatric rheumatic disease and a leading cause of childhood disability. The objective of this study was to characterize the PK, safety, and taste acceptability of tofacitinib in patients with JIA. Methods This Phase 1, open-label, multiple-dose (twice daily [BID] for 5 days) study of tofacitinib in patients with active (≥ 5 joints) polyarticular course JIA was conducted from March 2013–December 2015. Patients were allocated to one of three age-based cohorts: Cohort 1, 12 to

Published
2017
Full Text
View/download PDF

8. Telemedicine and other care models in pediatric rheumatology: an exploratory study of parents’ perceptions of barriers to care and care preferences

Author

Danielle R. Bullock, Richard K. Vehe, Lei Zhang, and Colleen K. Correll

Subjects
Access, Barriers, Outreach, Pediatric rheumatology, Telemedicine, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background The United States pediatric rheumatology workforce is committed to a mission of providing children access to pediatric rheumatology care. With a limited number and distribution of pediatric rheumatologists, telemedicine has been proposed as one way to meet this mission, yet the adoption of this modality has been slower than expected. The purpose of this study was to explore the parent perspective on barriers to accessing pediatric rheumatology care and to explore the acceptability of telemedicine and other alternative care models. Methods Over a period of six weeks, all new and return English-speaking parents/guardians of patients visiting a single center were offered an opportunity to complete a survey which assessed barriers to care and interest in alternative models of care. Responses were analyzed using descriptive statistics. Results Survey response rate was 72% (159/221). Twenty-eight percent (45/159) traveled more than three hours to the pediatric rheumatology clinic, and 43% (65/152) reported travel as inconvenient. An overwhelming majority of respondents (95%, 144/152) reported a preference for in-person visits over the option of telemedicine. This preference was similar regardless of whether respondents reported travel to the clinic as inconvenient vs convenient (inconvenient 92%, 60/65; convenient 97%, 84/87; p = 0.2881) and despite those reporting travel as inconvenient also reporting greater difficulty with several barriers to care. Those familiar with telemedicine were more likely to report a preference for telemedicine over in-person visits (27%, 3/11 vs 3%, 4/140; p = 0.0087). The option of an outreach clinic was acceptable to a majority (63%, 97/154); however, adult rheumatology and shared-care options were less acceptable (22%, 35/156 and 34%, 53/156 respectively). Conclusion Among survey respondents, in-person visits were preferred over the option of telemedicine, even when travel was noted to be inconvenient. Telemedicine familiarity increased its acceptability. Outreach clinics were acceptable to a majority. Ultimately, the parent perspective can shape acceptable ways to address barriers and provide accessible care.

Published
2017
Full Text
View/download PDF

9. Proceedings of the 23rd Paediatric Rheumatology European Society Congress: part two

Author

Olga Lomakina, Ekaterina Alekseeva, Sania Valieva, Tatiana Bzarova, Irina Nikishina, Elena Zholobova, Svetlana Rodionovskaya, Maria Kaleda, Yasuo Nakagishi, Masaki Shimizu, Mao Mizuta, Akihiro Yachie, Yuko Sugita, Nami Okamoto, Kousuke Shabana, Takuji Murata, Hiroshi Tamai, Eve M. Smith, Peng Yin, Andrea L. Jorgensen, Michael W. Beresford, on behalf of On behalf of the UK JSLE Cohort Study, Antonio Eleuteri, Beatrice Goilav, Laura Lewandowski, Angel Phuti, Dawn Wahezi, Tamar Rubinstein, Caroline Jones, Paul Newland, Stephen Marks, Rachel Corkhill, Diana Ekdawy, Clarissa Pilkington, Kjell Tullus, Chaim Putterman, Chris Scott, Antony C. Fisher, Andrea Jorgensen, Ezgi Deniz Batu, Can Kosukcu, Ekim Taskiran, Sema Akman, Kubra Ozturk, Betul Sozeri, Erbil Unsal, Zelal Ekinci, Yelda Bilginer, Mehmet Alikasifoglu, Seza Ozen, Hanna Lythgoe, Hermine I. Brunner, Gaurav Gulati, Jordan T. Jones, Mekibib Altaye, Jamie Eaton, Mark Difrancesco, Joo Guan Yeo, Jingyao Leong, Loshinidevi D/O Thana Bathi, Thaschawee Arkachaisri, Salvatore Albani, Nagla Abdelrahman, Michael W Beresford, Valentina Leone, UK JSLE study group supported by the National Institute of Health Research Clinical Research Network, Noortje Groot, D. Shaikhani, I. E. M. Bultink, M. Bijl, R. J. E. M. Dolhain, Y. K. O. Teng, E. Zirkzee, K. de Leeuw, R. Fritsch-Stork, S. S. M. Kamphuis, Rachael D. Wright, Reem Abdawani, Laila Al Shaqshi, Ibrahim Al Zakwani, Natali W. Gormezano, David Kern, Oriany L. Pereira, Gladys C. C. Esteves, Adriana M. Sallum, Nadia E. Aikawa, Rosa M. Pereira, Clovis A. Silva, Eloisa Bonfa, Jessica Beckmann, Nora Bartholomä, Nils Venhoff, Philipp Henneke, Ulrich Salzer, Ales Janda, Alina Lucica Boteanu, Sandra Garrote Corral, Alberto Sifuentes Giraldo, Mariluz Gámir Gámir, Antonio Zea Mendoza, Amra Adrovic, Reyhan Dedeoglu, Sezgin Sahin, Kenan Barut, Aida Koka, Funda Oztunc, Ozgur Kasapcopur, Ana Luisa Rodriguez-Lozano, Francisco Rivas-Larrauri, Silvestre García de la Puente, Andressa G. F. Alves, Maria F. D. A. Giacomin, Juliana Farhat, Alfésio L. F. Braga, Adriana M. E. Sallum, Lúcia M. D. A. Campos, Luiz A. A. Pereira, Ana J. D. F. C. Lichtenfels, Clóvis A. Silva, Sylvia C. L. Farhat, Banu Acar, Z. Birsin Ozcakar, Nilgün Çakar, Nermin Uncu, Gökçe Gür, Semanur Özdel, Fatoş Yalçınkaya, Christiaan Scott, Nicky Brice, Peter Nourse, Christine Arango, Angela C. Mosquera, Clara Malagon, Ana P. Sakamoto, Marco F. C. D. Silva, Ananadreia S. Lopes, Gleice C. S. Russo, Adriana E. M. Sallum, Katia Kozu, Eloisa Bonfá, Claudia Saad-Magalhães, Rosa M. R. Pereira, Claudio A. Len, Maria T. Terreri, Deepti Suri, Siyaram Didel, Amit Rawat, Surjit Singh, Despoina Maritsi, MArgarita Onoufriou, Olga Vougiouka, Maria Tsolia, Edi Paleka Bosak, Mandica Vidović, Mirta Lamot, Lovro Lamot, Miroslav Harjaček, Erika Van Nieuwenhove, Adrian Liston, Carine Wouters, Fatemeh Tahghighi, Vahid Ziaee, Seid-Reza Raeeskarami, Francisca Aguiar, Sandra Pereira, Mariana Rodrigues, Cláudia Moura, Gustavo Rocha, Hercília Guimarães, Iva Brito, Rita Fonseca, Gerd Horneff, Ariane Klein, Kirsten Minden, Hans-Iko Huppertz, Frank Weller-Heinemann, Jasmin Kuemmerle-Deschner, J-Peter Haas, Anton Hospach, BIKER collaborative group, Ricardo Menendez-Castro, Boris Huegle, Johannes-Peter Haas, Joost Swart, Gabriella Giancane, Francesca Bovis, Elio Castagnola, Andreas Groll, Daniel J. Lovell, Tom Wolfs, Michael Hofer, Violeta Panaviene, Susan Nielsen, Jordi Anton, Florence Uettwiller, Valda Stanevicha, Maria Trachana, Denise Pires Marafon, Constantin Ailioaie, Elena Tsitsami, Sylvia Kamphuis, Troels Herlin, Pavla Doležalová, Gordana Susic, Berit Flatø, Flavio Sztajnbok, Angela Pistorio, Alberto Martini, Nico Wulffraat, Nicolino Ruperto, Marco Gattorno, Antonio Brucato, Martina Finetti, George Lazaros, Silvia Maestroni, Mara Carraro, Davide Cumetti, Alessandra Carobbio, Monia Lorini, Alessandro Rimini, Renzo Marcolongo, Anna Valenti, Gian Luca Erre, Riccardo Belli, Fiorenzo Gaita, Maria Pia Sormani, Massimo Imazio, Mario Abinun, Nicola Smith, Tim Rapley, Flora McErlane, Lianne Kearsley-Fleet, Kimme L. Hyrich, Helen Foster, Nikolay Tzaribachev, Andrew Zeft, Rolando Cimaz, John Bohnsack, Thomas Griffin, Ruy Carrasco, Jason Dare, Ivan Foeldvari, Richard Vehe, Teresa Simon, Hermine Brunner, S. Verazza, S. Davì, A. Consolaro, A. Insalaco, V. Gerloni, R. Cimaz, F. Zulian, S. Pastore, F. Corona, G. Conti, P. Barone, M. Cattalini, E. Cortis, L. Breda, A. N. Olivieri, A. Civino, R. Podda, D. Rigante, F. La Torre, G. D’Angelo, M. Jorini, R. Gallizzi, M. C. Maggio, R. Consolini, A. De Fanti, M. G. Alpigiani, A. Martini, A. Ravelli, on behalf of Italian Pediatric Rheumatology Study Group, Aysenur Pac Kısaarslan, Zubeyde Gunduz, Ruhan Dusunsel, Ismail Dursun, Hakan Poyrazoglu, Ekaterina Kuchinskaya, Farida Abduragimova, Mikhail Kostik, Erik Sundberg, Soley Omarsdottir, Lena Klevenvall, Helena Erlandsson-Harris, Gokalp Basbozkurt, Ozge Erdemli, Dogan Simsek, Fatih Yazici, Yildirim Karsioglu, Aysen Tezcaner, Dilek Keskin, Huseyin Ozkan, Cengizhan Acikel, Erkan Demirkaya, Ilonka Orbán, Krisztina Sevcic, Valentin Brodszky, Emese Kiss, Ismaiel A. Tekko, Madeleine Rooney, James McElnay, Cliff Taggart, Helen McCarthy, Ryan F. Donnelly, Drug Delivery Group, Mary Slatter, Zohreh Nademi, Mark Friswell, Sharmila Jandial, Terence Flood, Sophie Hambleton, Andrew Gennery, Andrew Cant, Phoi-Ngoc Duong, Isabelle Koné-Paut, Giovanni Filocamo, María Luz Gamir, Helga Sanner, Laura Carenini, Mesut Topdemir, Yildirim Karslioglu, Faysal Gok, Nadezhda Tsurikova, Elena Ligostaeva, Navdha R. Ramchurn, O. Kostareva, I. Nikishina, S. Arsenyeva, S. Rodionovskaya, M. Kaleda, D. Alexeev, Ismail Dursun Dursun, Sara Murias, Estefania Barral, Rosa Alcobendas, Eugenia Enriquez, Agustin Remesal, Jaime de Inocencio, Tania M. Castro, Simone A. Lotufo, Tatjana Freye, Raffaella Carlomagno, Thomas Zumbrunn, Jan Bonhoeffer, Elvira Cannizzaro Schneider, Daniela Kaiser, Michaël Hofer, Véronique Hentgen, Andreas Woerner, Juvenile Inflammatory Rheumatism (JIR) Cohort, Tobias Schwarz, Jens Klotsche, Martina Niewerth, Gerd Ganser, ICON study group, Jerold Jeyaratnam, Nienke ter Haar, Donato Rigante, Fatma Dedeoglu, Ezgi Baris, Sebastiaan Vastert, Joost Frenkel, Jonathan S. Hausmann, Kathleen G. Lomax, Ari Shapiro, Karen L. Durrant, P. A. Brogan, M. Hofer, J. B. Kuemmerle-Deschner, B. Lauwerys, A. Speziale, K. Leon, X. Wei, R. M. Laxer, Sara Signa, Marta Rusmini, Elena Campione, Sabrina Chiesa, Alice Grossi, Alessia Omenetti, Roberta Caorsi, Gianmaria Viglizzo, Isabella Ceccherini, Silvia Federici, Helen Lachmann, Nicola Ruperto, on behalf of PRINTO and Eurofever Registry, Federica Vanoni, on behalf of PRINTO and Eurofever Project, Sonia Melo Gomes, Ebun Omoyinmi, Juan I. Arostegui, Eva Gonzalez-Roca, Despina Eleftheriou, Nigel Klein, Paul Brogan, Stefano Volpi, Elettra Santori, Paolo Picco, Claudia Pastorino, Gillian Rice, Alessandra Tesser, Yanick Crow, Fabio Candotti, Ada B. Sinoplu, Gozde Yucel, Gizem Pamuk, Laura O. Damian, Cecilia Lazea, Mihaela Sparchez, Paulina Vele, Laura Muntean, Adriana Albu, Simona Rednic, Calin Lazar, Leonardo O. Mendonça, Alessandra Pontillo, Jorge Kalil, Fabio M. Castro, Myrthes T. Barros, Manuela Pardeo, Virginia Messia, Fabrizio De Benedetti, Antonella Insalaco, Giorgia Malighetti, Chiara Gorio, Francesca Ricci, Ilaria Parissenti, Paola Montesano, Barbara Bonafini, Veronica Medeghini, Marco Cattalini, Lucio Giordano, Giulia Zani, Rosalba Ferraro, Donatella Vairo, Silvia Giliani, Maria Cristina Maggio, Girolamo Luppino, Giovanni Corsello, Maria Isabel Gonzalez Fernandez, Berta Lopez Montesinos, Adriana Rodriguez Vidal, Juan I. Arostegui Gorospe, Inmaculada Calvo Penades, Nadia K. Rafiq, Karen Wynne, Khalid Hussain, Paul A. Brogan, Elizabeth Ang, Nicholas Ng, Ayla Kacar, Ozge Altug Gucenmez, Balahan Makay, Sevket Erbil Unsal, Yasin Sahin, Tufan Kutlu, Fugen Cullu-Cokugras, Hasret Ayyildiz-Civan, Tulay Erkan, Sana Al Zuhbi, Eiman Abdalla, Ricardo A. Russo, María M. Katsicas, Francesca Minoia, Angelo Ravelli, Sagar Bhattad, Anju Gupta, Vignesh Pandiarajan, Ritambhra Nada, Kaara Tiewsoh, Philip Hawkins, Dorota Rowczenio, Sarka Fingerhutova, Jana Franova, Leona Prochazkova, Eva Hlavackova, Pavla Dolezalova, Havva Evrengül, Selçuk Yüksel, Mustafa Doğan, Dolunay Gürses, Harun Evrengül, Silvia De Pauli, Serena Pastore, Anna Monica Bianco, Giovanni Maria Severini, Andrea Taddio, Alberto Tommasini, Svetlana O. Salugina, Evgeny Fedorov, Elena Kamenets, Ekaterina Zaharova, Tatiana Sleptsova, Ekaterina Alexeeva, Kirill Savostyanov, Alexander Pushkov, Tatyana Bzarova, Saniya Valieva, Rina Denisova, Kseniya Isayeva, Evgeniya Chistyakova, Margarita Soloshenko, Elena Kaschenko, Utako Kaneko, Chihaya Imai, Akihiko Saitoh, Vitor A. Teixeira, Filipa O. Ramos, Manuela Costa, Yonatan Butbul Aviel, Shafe Fahoum, Riva Brik, Zeynep Birsin Özçakar, Banu Acar Celikel, Fatos Yalcinkaya, Benedetta Schiappapietra, Sergio Davi’, Federica Mongini, Luisa Giannone, Cecilia Bava, Maria Giannina Alpigiani, Alessandro Consolaro, Dragana S. Lazarevic, Jelena Vojinovic, Jelena Basic, Valentina Muratore, Valentina Marzetti, Neus Quilis, Belen Serrano Benavente, Alessandra Alongi, Adele Civino, Lorenzo Quartulli, Giedre Januskeviciute, Pieter van Dijkhuizen, N. Groot, W. van Dijk, A. Kardolus, Raul Gutiérrez Suárez, Ellen B. Nordal, Veronika G. Rypdal, Lillemor Berntson, Maria Ekelund, Kristiina Aalto, Suvi Peltoniemi, Marek Zak, Mia Glerup, Ellen D. Arnstad, Anders Fasth, Marite Rygg, the Nordic Study Group of Pediatric Rheumatology (NoSPeR), Ana Catarina Duarte, Sandra Sousa, Lídia Teixeira, Ana Cordeiro, Mª José Santos, Ana Filipa Mourão, Maria José Santos, Mónica Eusébio, Ana Lopes, Filipa Oliveira-Ramos, Manuel Salgado, Paula Estanqueiro, José Melo-Gomes, Fernando Martins, José Costa, Carolina Furtado, Ricardo Figueira, Jaime C. Branco, João E. Fonseca, Helena Canhão, Ana F. Mourão, Maria Jose Santos, Andrea Coda, Samuel Cassidy, Kerry West, Gordon Hendry, Debra Grech, Julie Jones, Fiona Hawke, Davinder Singh Grewal, Charlene Foley, Orla Killeen, Emma MacDermott, Douglas Veale, Ursula Fearon, Dilek Konukbay, Ela Tarakci, Nilay Arman, Sezgin Şahin, Jane Munro, Esi Morgan, Meredith Riebschleger, Jennifer Horonjeff, Vibeke Strand, Clifton Bingham, Ma. Theresa M. Collante, Margarita Ganeva, Stefan Stefanov, Albena Telcharova, Dimitrina Mihaylova, Radoslava Saraeva, Reni Tzveova, Radka Kaneva, Adelina Tsakova, Katya Temelkova, GRANT Medical University, Sofia 68/, Maria Mercedes C. Picarelli, Luiz C. Danzmann, Florencia Barbé-Tuana, Lucas K. Grun, Marcus H. Jones, Marijan Frković, Karla Ištuk, Ika Birkić, Saša Sršen, Marija Jelušić, Alan Easton, Rachael Quarmby, Raju Khubchandani, Mercedes Chan, Radoslav Srp, Katerina Kobrova, Dana Nemcova, Jozef Hoza, Michal Uher, Melania Saifridova, Lenka Linkova, Sirirat Charuvanij, Isree Leelayuwattanakul, Thita Pacharapakornpong, Sakda A.-O. Vallipakorn, Butsabong Lerkvaleekul, Soamarat Vilaiyuk, Stefano Lanni, Sergio Davì, Randy Q. Cron, Chiara Passarelli, Elisa Pisaneschi, Antonio Novelli, Claudia Bracaglia, Ivan Caiello, Kathy de Graaf, Florence Guilhot, Walter Ferlin, Grant Schulert, Alexi A. Grom, Robert Nelson, Cristina de Min, Dirk Holzinger, Christoph Kessel, Ndate Fall, Alexei Grom, Wilco de Jager, Raffaele Strippoli, Anna Horne, Stephan Ehl, Sandra Ammann, Kai Lehmberg, Karin Beutel, Dirk Foell, AnnaCarin Horne, Laura Pagani, Graciela Espada, Yi-jin Gao, Susan Shenoi, Sheila Weitzman, Giusi Prencipe, Antonia Pascarella, Walter G. Ferlin, Laurence Chatel, Philippe Jacqmin, Kathy De Graaf, Maria Ballabio, Zoë Johnson, Geneviève Lapeyre, Fabrizio de Benedetti, de Min Cristina, Hiroyuki Wakiguchi, Shunji Hasegawa, Reiji Hirano, Fumiko Okazaki, Tamaki Nakamura, Hidenobu Kaneyasu, Shouichi Ohga, Kazuko Yamazaki, Tomo Nozawa, Taichi Kanetaka, Shuichi Ito, Shumpei Yokota, Kirsty McLellan, Ishbel MacGregor, Neil Martin, Joyce Davidson, Sandra Hansmann, Andreas Eikelberg, Iris Haug, Sabrina Schuller, Susanne M. Benseler, Single Hub and Access point for paediatric Rheumatology in Europe (SHARE), Liliia S. Nazarova, Kseniia V. Danilko, Viktor A. Malievsky, Tatiana V. Viktorova, Angela Mauro, Angela Barnicoat, Jane Hurst, Nathalie Canham, Sandrine Lacassagne, Anastasia Wiener, Boris Hügle, Bernd Denecke, Ivan Costa-Filho, Johannes Peter Haas, Klaus Tenbrock, David Popp, Arjan Boltjes, Frank Rühle, Stefanie Herresthal, Femke van Wijk, Joachim Schultze, Monika Stoll, Luisa Klotz, Thomas Vogl, Johannes Roth, Estefania Quesada-Masachs, Daniel Álvarez de la Sierra, Marina Garcia Prat, Ana M. Marín Sánchez, Ricardo Pujol Borrell, Sara Marsal Barril, Mónica Martínez Gallo, Consuelo Modesto Caballero, Iryna Chyzheuskaya, Lyudmyla M. Byelyaeva, Rostislav M. Filonovich, Helena K. Khrustaleva, Larisa I. Zajtseva, Tamara M. Yuraga, Thomas Giner, Lukas Hackl, Julia Albrecht, Reinhard Würzner, Juergen Brunner, Marta Minute, Fulvio Parentin, Agostino Nocerino, Mette Nørgaard, Mikel Alberdi-Saugstrup, Marek S. Zak, Susan M. Nielsen, Ellen Nordal, Klaus G. Müller, Nordic Study Group of Pediatric Rheumatology (NoSPeR), Mojca Zajc Avramovič, Vita Dolžan, Nataša Toplak, Tadej Avčin, N. Ruperto, D. J. Lovell, C. Wallace, M. Toth, I. Foeldvari, J. Bohnsack, D. Milojevic, C. Rabinovich, D. Kingsbury, K. Marzan, P. Quartier, K. Minden, E. Chalom, G. Horneff, R. M. Kuester, J. Dare, M. Heinrich, H. Kupper, J. Kalabic, H. I. Brunner, on behalf of PRINTO and PRCSG, Ruben Burgos-Vargas, Tamas Constantin, Joke Dehoorne, Valda Stanevica, Katarzyna Kobusinska, Zbigniew Zuber, Richard Mouy, Ingrida Rumba-Rozenfelde, Chantal Job-Deslandre, Ronald Pederson, Jack Bukowski, Tina Hinnershitz, Bonnie Vlahos, Paula Keskitalo, Salla Kangas, Paula Vähäsalo, Raul A. Chavez Valencia, David Martino, Anne-Louise Ponsonby, Rachel Chiaroni-Clarke, Braydon Meyer, Roger C. Allen, Jonathan D. Akikusa, Jeffrey M. Craig, Richard Saffrey, Justine A. Ellis, Carol Wallace, Yosef Uziel, Gary Sterba, Rayfel Schneider, Ricardo Russo, Athimalaipet V. Ramanan, Jana Pachlopnik Schmid, Kim E Nichols, Paivi Miettunen, Toshiyuki Kitoh, Norman T. Ilowite, Jan-Inge Henter, Alexei A Grom, Edward M. Behrens, Tadej Avcin, Maurizio Aricò, Sriharsha Grevich, Peggy Lee, Sarah Ringold, Brian Leroux, Hannah Leahey, Megan Yuasa, Jessica Foster, Jeremy Sokolove, Lauren Lahey, William Robinson, Joshua Newson, Anne Stevens, Stephanie J. W. Shoop, Suzanne M. M. Verstappen, Wendy Thomson, Janet E. McDonagh, CAPS, Timothy Beukelman, Yuki Kimura, Marc Natter, Norm Ilowite, Kelly Mieszkalski, Grendel Burrell, Brian Best, Helen Bristow, Shannon Carr, Anne Dennos, Rachel Kaufmann, Laura Schanberg, for the CARRA Registry Investigators, Gabriele Simonini, Francesca Lancini, Margaux Gerbaux, Phu-Quoc Lê, Laurence Goffin, Valérie Badot, Céline La, Laure Caspers, François Willermain, Alina Ferster, Maria Ceci, Francesco Licciardi, Marco Turco, Francesca Santarelli, Davide Montin, Claudia Toppino, Clotilde Alizzi, Bruno Papia, Beatrice Vergara, Umberto Corpora, Luca Messina, Maria Tsinti, Vasiliko Dermentzoglou, Panagiotis Tziavas, Marija Perica, Lana Tambić Bukovac, Mustafa Çakan, Nuray Aktay Ayaz, Gonca Keskindemirci, Michael Lang, Catherine Laing, Susanne Benseler, Tommy Gerschman, Nadia Luca, Heinrike Schmeling, Anastasia Dropol, Jaymi Taiani, Nicole Johnson, Brian Rusted, Panagiota Nalbanti, Polyxeni Pratsidou, Grigoris Pardalos, Vasiliki Tzimouli, Anna Taparkou, Maria Stavrakidou, Fotios Papachristou, Florence Kanakoudi-Tsakalidou, Peter Bale, Emily Robinson, Jason Palman, Elizabeth Ralph, Kimberly Gilmour, Clare Heard, Lucy R. Wedderburn, Yara Barrense-Dias, Antonarakis Gregory, Dhouib Amira, Scolozzi Paolo, Hanquinet Sylviane, Hofer Michaël, Nataliya Panko, Salah Shokry, Liudmila Rakovska, Sally Pino, Adriana Diaz-Maldonado, Pilar Guarnizo, Sofia Torreggiani, Paolo Cressoni, Umberto Garagiola, Giancarla Di Landro, Giampietro Farronato, Fabrizia Corona, Samantha Bell, Parveen Bhatti, Lee Nelson, Beth A. Mueller, T. A. Simon, A. Baheti, N. Ray, Z. Guo, Anasuya Hazra, Thomas Stock, Ronnie Wang, Charles Mebus, Christine Alvey, Manisha Lamba, Sriram Krishnaswami, Umberto Conte, Min Wang, Daniel Kingsbury, Elena Koskova, Elzbieta Smolewska, Richard K. Vehe, Daniel Lovell, Tomohiro Kubota, Junko Yasumura, Toshitaka Kizawa, Masato Yashiro, Tsuyoshi Yamatou, Yuichi Yamasaki, Syuji Takei, Yoshifumi Kawano, Ulrika Järpemo Nykvist, Bo Magnusson, Rikard Wicksell, Karin Palmblad, Gunnar L. Olsson, Mohammadreza Modaressi, Mohammad-Hassan Moradinejad, Valentina Seraya, Alisa Vitebskaya, Veronica Moshe, Gil Amarilyo, Liora Harel, Phillip J Hashkes, Amir Mendelson, Noa Rabinowicz, Yonit Reis, Zane Dāvidsone, Arina Lazareva, Ruta Šantere, Dace Bērziņa, Valda Staņēviča, Giulia Camilla Varnier, Susan Maillard, Cristina Ferrari, Silvia Zaffarano, Juvenile Dermatomyositis Research Group and European Federation of Immunological Societies, Judith Wienke, Felicitas Bellutti Enders, Lucas L. van den Hoogen, Jorre S. Mertens, Timothy R. Radstake, Henny G. Hotten, Ruth Fritsch, Lucy Wedderburn, Kiran Nistala, Berent Prakken, Annet van Royen-Kerkhof, Mohammad Alhemairi, Mohammed Muzaffer, Pieter Van Dijkhuizen, Claire T. Deakin, Stefania Simou, Maria De Iorio, Qiong Wu, Tania Amin, Lee Dossetter, Juvenile Dermatomyositis Research Group (JDRG), Raquel Campanilho-Marques, Claire Deakin, Clarissa A. Pilkington, on behalf of Juvenile Dermatomyositis Research Group (JDRG), Silvia Rosina, Sirisucha Soponkanaporn, on behalf of the UK Juvenile Dermatomyositis Research Group (JDRG), Zehra S. Arıcı, Gökçen D. Tuğcu, Ezgi D. Batu, Hafize E. Sönmez, Deniz Doğru-Ersöz, Beril Talim, Nural Kiper, Seza Özen, Alexander Solyom, Ezgi Batu, John Mitchell, Ariana Kariminejad, Fatemeh Hadipour, Zahra Hadipour, Marta Torcoletti, Carlo Agostoni, Maja Di Rocco, Pranoot Tanpaiboon, Andrea Superti-Furga, Luisa Bonafé, Nur Arslan, Norberto Guelbert, Karoline Ehlert, Giedre Grigelioniene, Ratna Puri, Edward Schuchman, Pilar Gomez, Tatiana Gonzalez, Ricardo Yepez, Camilo Vargas, GRIP study group, Falcini Fernanda, Gemma Lepri, Alessandra Ferrari, Marco Matucci-Cerinic, Antonella Meini, Gian Marco Moneta, Emiliano Marasco, Rebecca Nicolai, Luisa Bracci-Laudiero, Olga Kopchak, Alexander Mushkin, Alexey Maletin, Catalina Mosquera, Rita A. Amorim, Juliana Molina, Gustavo Moreira, Flávia H. Santos, Melissa Fraga, Livia Keppeke, Vanessa M. Silva, Camila Hirotsu, Sergio Tufik, Maria Teresa Terreri, Vinícius L. Braga, Maria Beatriz Fonseca, Vania Schinzel, Maria Teresa R. Terreri, Liliana Jorge, Liana Guerra, Edson Amaro Junior, Maria Cristina Castiglione, Alessandra Tricarico, Emily Boulter, Andre Schultz, Kevin Murray, Fernanda Falcini, Stefano Stagi, Eleonora Bellucci, Ingrid H. R. Grein, Gecilmara Pileggi, Natália B. F. Pinto, Aline L. de Oliveira, Lyudmila Belyaeva, Rostislav Filonovich, Helena Khrustaleva, Larisa Zajtseva, Jaanika Ilisson, Chris Pruunsild, Olivier Gilliaux, Francis Corazza, Christophe Lelubre, on behalf of PANLAR Pediatric Rheumatology Study Group, Zoilo Morel, Claudia Saad-Magalhães C, Luis Lira, Mabel Ladino, Ruth Eraso, Ivonne Arroyo, Clovis Silva, Carlos Rose, and PANLAR Pediatric Rheumatology Study Group

Subjects
Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Published
2017
Full Text
View/download PDF

10. Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II

Author

Lynda E. Polgreen, Alicia Kunin-Batson, Kyle Rudser, Richard K. Vehe, Jeanine J. Utz, Chester B. Whitley, and Patricia Dickson

Subjects
Mucopolysaccharidosis, Inflammation, Joint, Bone, Medicine (General), R5-920, Biology (General), QH301-705.5
Abstract

Mucopolysaccharidosis I and II are lysosomal storage disorders that, despite treatment with hematopoietic cell transplantation (HCT) and/or enzyme replacement therapy (ERT), continue to cause significant skeletal abnormalities leading to pain, stiffness, physical dysfunction, and short stature. Tumor necrosis factor – alpha (TNF-α) is elevated in individuals with MPS I and II and associated with pain and physical dysfunction. Therefore, we evaluated the safety and effects of the TNF-α inhibitor adalimumab in patients with MPS I and II in a 32-week, randomized, double blind, placebo-controlled, crossover study of adalimumab at a dose of 20 mg (weight 15–5° in seven of eight joints in Subject #1 and five of eight joints in Subject #2 (range 7.0° to 52.8°). There was no change in the PPQ, 6MWT, or hand dynamometer. Data from this small pilot study suggest that treatment with adalimumab is safe, tolerable, and may improve ROM, physical function, and possibly pain, in children with MPS I or II. However, additional clinical trials are needed before this therapy should be recommended as part of clinical care.

Published
2017
Full Text
View/download PDF

11. Capturing Critical Data Elements in Juvenile Idiopathic Arthritis: Variations between In-person and Virtual Visits and Initiatives to Improve Data Capture

Author

Meghan E. Ryan, Andrew Warmin, Bryce A. Binstadt, Colleen K. Correll, Emily Hause, Patricia Hobday, Alison Lerman, Shawn A. Mahmud, Mona M. Riskalla, Zachary Shaheen, Richard K. Vehe, and Danielle R. Bullock

Abstract

Background: Documentation of critical data elements is a focus of the Pediatric Rheumatology Care and Outcomes Improvement Network to aid in clinical care and research for patients with juvenile idiopathic arthritis. We aimed to increase data capture for arthritis pain score, patient/parent global assessment of wellbeing, provider global assessment, and active joint count. We hypothesized that data capture for all critical data elements would be lower for virtual visits compared to in-person visits. Methods: All visits for patients with JIA between 9/14/2020 and 12/31/2021 at the University of Minnesota were included. Sixteen interventions with providers were conducted, including email reminders, individual discussions, group meetings, and feedback reports. We used statistical process control charts to evaluate change over time.Results: Baseline included 355 patient-visits: 221 (62%) in-person and 134 (38%) virtual with critical data elements entry ranging between 50-60%. Post-intervention included 1,596 patient-visits: 1,350 (85%) in-person and 246 (15%) virtual with critical data elements entry of 91%. In-person visits had significantly higher data capture rates than virtual visits for all 4 critical data elements.Conclusion: We achieved our aim to increase critical data element documentation and found that collection of critical data elements occurred significantly less often with virtual visits than with in-person visits. We inferred that critical data element capture for virtual visits was lower for multiple reasons, including the lack of intake forms and provider uncertainty about scoring certain elements. Now that we improved capture of critical data elements, we can shift the focus to efforts aimed at improving outcomes for patients with juvenile arthritis.

Published
2022
Full Text
View/download PDF

12. Rapid Infliximab Infusion in the Pediatric Population

Author

Elissa M. Downs, Boris Sudel, Peter J. Bryan, Richard K. Vehe, Colleen K. Correll, Danielle R. Bullock, Bryce A. Binstadt, Catherine Larson-Nath, Erin J. Munsel, and Patricia M. Hobday

Subjects
medicine.medical_specialty, business.industry, Clinical Investigations, Rheumatology, Infliximab, 03 medical and health sciences, 0302 clinical medicine, Patient satisfaction, Infusion therapy, 030220 oncology & carcinogenesis, Anesthesia, Chart review, Internal medicine, Pediatrics, Perinatology and Child Health, medicine, 030211 gastroenterology & hepatology, Pharmacology (medical), Dosing, Infusion reaction, business, medicine.drug, Pediatric population
Abstract

OBJECTIVES To compare infusion reaction rates between rapid infliximab (REMICADE, Janssen Biotech Inc) infusions and previous standard 2- to 3-hour infusions; additionally, to assess patient satisfaction and reduction in chair time associated with rapid infliximab infusions. METHODS Pediatric rheumatology and gastroenterology patients receiving maintenance infliximab therapy using a standard 2- to 3-hour titrated infusion had the opportunity to enroll in the non-titrated rapid 1-hour infusion protocol following tolerance of induction dosing at 0, 2, and 6 weeks. Patients were included from December 1, 2017, to March 31, 2018, via retrospective chart review and patient satisfaction surveys. RESULTS Data were collected on 55 patients receiving a total of 160 rapid infliximab infusions. There were 2 infusion reactions during the enrollment and data collection period, resulting in an overall infusion reaction rate of 1.3%. The patient satisfaction survey results showed all patients were at minimum satisfied with the information provided regarding rapid infliximab, decreased time spent in clinic, ease of scheduling, and overall process. CONCLUSIONS Our data suggest rapid infliximab infusions are safe in pediatric rheumatology and gastroenterology patients receiving maintenance infliximab infusion therapy. The overall infusion reaction rate of 1.3% in this study is well below the accepted infusion reaction rate of standard-length infliximab infusions of 2% to 3%.

Published
2020
Full Text
View/download PDF

13. T cell-intrinsic role for Nod2 in protection against Th17-mediated uveitis

Author

Bryce A. Binstadt, João M. Furtado, Michael P. Davey, Paige E. Snow, Biying Xu, Ruth J. Napier, Christina Lancioni, Kimberly A. Samson, Michelle C. Callegan, E. J. Lee, Mary J. Mattapallil, Holly L. Rosenzweig, Emily E. Vance, Sydney Lashley, Reiko Horai, Luke S. Uebelhoer, Brieanna Brown, Rachel R. Caspi, Richard K. Vehe, and Justine R. Smith

Subjects
0301 basic medicine, CD4-Positive T-Lymphocytes, Male, Receptors, CCR7, Sarcoidosis, T cell, Science, Nod2 Signaling Adaptor Protein, General Physics and Astronomy, C-C chemokine receptor type 7, Autoimmunity, Biology, Signal transduction, General Biochemistry, Genetics and Molecular Biology, Article, Uveitis, 03 medical and health sciences, Mice, 0302 clinical medicine, Immunity, NOD2, NOD-like receptors, medicine, Animals, Humans, T-helper 17 cells, lcsh:Science, Blau syndrome, Multidisciplinary, Synovitis, Arthritis, T-cell receptor, Interleukin-17, MUTAÇÃO GENÉTICA, General Chemistry, medicine.disease, digestive system diseases, Mice, Inbred C57BL, 030104 developmental biology, medicine.anatomical_structure, Immunology, Th17 Cells, Female, lcsh:Q, Ligation, 030215 immunology
Abstract

Mutations in nucleotide-binding oligomerization domain-containing protein 2 (NOD2) cause Blau syndrome, an inflammatory disorder characterized by uveitis. The antimicrobial functions of Nod2 are well-established, yet the cellular mechanisms by which dysregulated Nod2 causes uveitis remain unknown. Here, we report a non-conventional, T cell-intrinsic function for Nod2 in suppression of Th17 immunity and experimental uveitis. Reconstitution of lymphopenic hosts with Nod2−/− CD4+ T cells or retina-specific autoreactive CD4+ T cells lacking Nod2 reveals a T cell-autonomous, Rip2-independent mechanism for Nod2 in uveitis. In naive animals, Nod2 operates downstream of TCR ligation to suppress activation of memory CD4+ T cells that associate with an autoreactive-like profile involving IL-17 and Ccr7. Interestingly, CD4+ T cells from two Blau syndrome patients show elevated IL-17 and increased CCR7. Our data define Nod2 as a T cell-intrinsic rheostat of Th17 immunity, and open new avenues for T cell-based therapies for Nod2-associated disorders such as Blau syndrome., How mutations in the microbial receptor NOD2 induce Blau syndrome in humans and related uveitis is unclear. Here the authors show, using Nod2-deficient mice and experimental uveitis, that Nod2 negatively regulates T cell activation and transcription of autoimmunity-related genes to suppress Th17 responses and uveitis.

Published
2020

14. Using quality improvement methodology and tools to reduce patient wait time in a paediatric subspecialty rheumatology clinic

Author

Bayardo Garay, Denise Erlanson, Bryce A Binstadt, Colleen K Correll, Nora Fitzsimmons, Patricia M Hobday, Allison Hudson, Shawn Mahmud, Mona M Riskalla, Sara Kramer, Sheng Xiong, Richard K Vehe, and Danielle R Bullock

Subjects
Waiting Lists, Leadership and Management, Health Policy, Public Health, Environmental and Occupational Health, Ambulatory Care Facilities, Quality Improvement, paediatrics, Rheumatology, ambulatory care, Patient Satisfaction, Humans, Child, Original Research
Abstract

Our paediatric rheumatology clinic has experienced inefficient patient flow. Our aim was to reduce mean wait time and minimise variation for patients. Baseline data showed that most waiting occurs after a patient has been roomed, while waiting for the physician. Wait time was not associated with a patient’s age, time of day, day of the week or individual physician. We implemented a checkout sheet and staggered start times. After a series of plan–do–study–act cycles, we observed an initial 26% reduction in the variation of wait time and a final 17% reduction in the mean wait time. There was no impact on patient–physician contact time. Overall, we demonstrate how process improvement methodology and tools were used to reduce patient wait time in our clinic, adding to the body of literature on process improvement in an ambulatory setting.

Published
2021

15. Collagen Vascular Diseases: SLE, Dermatomyositis, Scleroderma, and MCTD

Author

Mona Riskalla and Richard K. Vehe

Subjects
medicine.medical_specialty, Anti-nuclear antibody, Extractable nuclear antigens, Disease, Dermatomyositis, Scleroderma, Diagnosis, Differential, Scleroderma, Localized, 03 medical and health sciences, 0302 clinical medicine, Mixed connective tissue disease, 030225 pediatrics, medicine, Humans, Lupus Erythematosus, Systemic, Child, Mixed Connective Tissue Disease, 030203 arthritis & rheumatology, Scleroderma, Systemic, Lupus erythematosus, business.industry, Prognosis, medicine.disease, Dermatology, Connective tissue disease, Pediatrics, Perinatology and Child Health, Female, business
Abstract

1. Richard K. Vehe, MD* 2. Mona M. Riskalla, MD* 1. *Division of Pediatric Rheumatology, Department of Pediatrics, University of Minnesota and the University of Minnesota Masonic Children’s Hospital, Minneapolis, MN * Abbreviations: ANA: : antinuclear antibody CTD: : connective tissue disease CVD: : collagen vascular disease dsDNA: : doubled-stranded DNA DM: : dermatomyositis ENA: : extractable nuclear antigen JIA: : juvenile idiopathic arthritis MCTD: : mixed connective tissue disease NFC: : nail fold capillary RA: : rheumatoid arthritis RF: : rheumatoid factor RP: : Raynaud phenomenon RNP: : ribonucleoprotein SLE: : systemic lupus erythematosus Timely and accurate recognition of collagen vascular disorders (CVDs), and implementation of effective screening and referral processes for patients suspected of having a CVD, remain a challenge for many physicians. The result, too often, is unnecessary testing and referrals, and in some cases unnecessary anxiety for physicians, patients, and parents. After completing this article, readers should be able to: 1. Recognize the common clinical symptoms and signs of systemic lupus erythematosus, dermatomyositis, and scleroderma, and their distinction from common infectious mimics. 2. Recognize the testing that can clarify the likelihood of whether a child has a rheumatic disease, including the limited utility of early serologic testing for autoantibodies. 3. Recognize the prognosis and management objectives for these often-chronic disorders, and practical steps to ensure excellent outcomes. Timely and accurate recognition of collagen vascular diseases (CVDs), and implementation of effective screening and referral processes for patients suspected of having a CVD, remain challenging for many physicians. The result, too often, is unnecessary testing with questionable results, leading in some cases to unnecessary referrals and anxiety for physicians, patients, and parents. (1) Sometimes, CVDs are also referred to as connective tissue diseases (CTDs), but neither name fully captures their essential nature. These are not primarily heritable disorders affecting collagen-containing structures, or disorders of vascular development, but rather are immune-mediated inflammatory diseases. The immunologic targets vary, but the inflammatory disease often affects collagen-containing tissues, including the musculoskeletal system and many other tissues. The targeting of blood vessels can lead to manifestations ranging from reversible vasospasm of Raynaud phenomenon (RP) to vascular injury of 1 or more organ systems, often with serious consequences. The diseases that …

Published
2018
Full Text
View/download PDF

16. Emergent high fatality lung disease in systemic juvenile arthritis

Author

Jenny Lin, Michal Cidon, Richard K. Vehe, Seza Ozen, Aliva De, Christi J. Inman, Suhas M. Radhakrishna, Maria Ibarra, Fabrizio De Benedetti, Raymond R. Balise, Joy Mombourquette, James Birmingham, Maria de los Angeles Ceregido Perez, Ian Ferguson, Marisa Klein-Gitelman, Steven I. Goodman, Layla Bouzoubaa, Karen Onel, Assunta Ho, Khanh Lai, Kathleen A. Haines, Sara O. Vargas, Ann N. Leung, Dieneke Schonenberg-Meinema, Sampath Prahalad, Rayfel Schneider, R. Paul Guillerman, Gail H. Deutsch, Kevin W. Baszis, Alicia Casey, Deborah R. Liptzin, Lu Tian, Vivian E. Saper, Adam L Reinhardt, Grant S. Schulert, Diana Milojevic, Martha P. Fishman, Lauren A. Henderson, Purvesh Khatri, Johannes Roth, Edward M. Behrens, Mona Riskalla, Gill Bejerano, Jacqueline Yang, Clara Lin, Sivia K. Lapidus, Alexei A. Grom, Elizabeth D. Mellins, Matthew L. Stoll, Mark M. Davis, Randy Q. Cron, Karthik A. Jagadeesh, Khalid Abulaban, Khulood Khawaja, Natalie Rosenwasser, Kathryn Phillippi, Hafize Emine Sönmez, Ying Lu, Lisa R. Young, T Brent Graham, Rita Jerath, Daniel J. Kingsbury, Guangbo Chen, Melissa M. Hazen, Robin R. Deterding, Scott W. Canna, Christopher Towe, Johannes Birgmeier, Judith A. Smith, Susan Shenoi, Jianpeng Xu, Tushar J. Desai, Graduate School, Paediatric Infectious Diseases / Rheumatology / Immunology, and AII - Inflammatory diseases

Subjects
Lung Diseases, Male, 0301 basic medicine, medicine.medical_specialty, Biopsy, Immunology, Arthritis, Gastroenterology, General Biochemistry, Genetics and Molecular Biology, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Tocilizumab, Rheumatology, Internal medicine, medicine, Humans, Immunology and Allergy, Child, Lung, Pathological, Retrospective Studies, 030203 arthritis & rheumatology, business.industry, Incidence, Infant, Retrospective cohort study, Prognosis, medicine.disease, Arthritis, Juvenile, United States, Survival Rate, 030104 developmental biology, chemistry, Child, Preschool, Concomitant, Macrophage activation syndrome, Female, Tomography, X-Ray Computed, Trisomy, Pulmonary alveolar proteinosis, business, Follow-Up Studies
Abstract

ObjectiveTo investigate the characteristics and risk factors of a novel parenchymal lung disease (LD), increasingly detected in systemic juvenile idiopathic arthritis (sJIA).MethodsIn a multicentre retrospective study, 61 cases were investigated using physician-reported clinical information and centralised analyses of radiological, pathological and genetic data.ResultsLD was associated with distinctive features, including acute erythematous clubbing and a high frequency of anaphylactic reactions to the interleukin (IL)-6 inhibitor, tocilizumab. Serum ferritin elevation and/or significant lymphopaenia preceded LD detection. The most prevalent chest CT pattern was septal thickening, involving the periphery of multiple lobes ± ground-glass opacities. The predominant pathology (23 of 36) was pulmonary alveolar proteinosis and/or endogenous lipoid pneumonia (PAP/ELP), with atypical features including regional involvement and concomitant vascular changes. Apparent severe delayed drug hypersensitivity occurred in some cases. The 5-year survival was 42%. Whole exome sequencing (20 of 61) did not identify a novel monogenic defect or likely causal PAP-related or macrophage activation syndrome (MAS)-related mutations. Trisomy 21 and young sJIA onset increased LD risk. Exposure to IL-1 and IL-6 inhibitors (46 of 61) was associated with multiple LD features. By several indicators, severity of sJIA was comparable in drug-exposed subjects and published sJIA cohorts. MAS at sJIA onset was increased in the drug-exposed, but was not associated with LD features.ConclusionsA rare, life-threatening lung disease in sJIA is defined by a constellation of unusual clinical characteristics. The pathology, a PAP/ELP variant, suggests macrophage dysfunction. Inhibitor exposure may promote LD, independent of sJIA severity, in a small subset of treated patients. Treatment/prevention strategies are needed.

Published
2019

17. Use of Rheumatology Laboratory Studies Among Primary Pediatricians

Author

Richard K. Vehe, Colleen K. Correll, Logan G. Spector, Bryce A. Binstadt, and Lei Zhang

Subjects
medicine.medical_specialty, Pediatrics, Referral, Primary care, Article, 03 medical and health sciences, 0302 clinical medicine, Rheumatic Diseases, 030225 pediatrics, Internal medicine, medicine, Humans, Rheumatoid factor, Pediatricians, Practice Patterns, Physicians', Pediatric rheumatology, skin and connective tissue diseases, 030203 arthritis & rheumatology, Response rate (survey), Diagnostic Tests, Routine, business.industry, Rheumatology, Pediatrics, Perinatology and Child Health, Physical therapy, Positive relationship, Clinical Competence, Clinical competence, business
Abstract

Rheumatology laboratory tests are often inappropriately ordered in situations for which they are of low diagnostic utility. We surveyed pediatricians to investigate reasons for ordering these tests. The response rate was 15.3% (93/609). The most commonly ordered tests were antinuclear antibody (ANA), anti-double-stranded DNA (dsDNA) antibody, and rheumatoid factor (RF). Of the 89% (83/93) who ordered an ANA, 86% ordered it for correct/possibly correct reasons; of the 73% (68/93) who ordered RF, 80% did so for correct/possibly correct reasons; and among the 59% (54/92) who had ordered anti-dsDNA antibody, 34% ordered it for correct reasons. A positive relationship was seen between years since residency completion and correct use of ANA. However, positive associations were not seen between measures of pediatric rheumatology experience and correct use of other tests. Interventions are needed to improve pediatricians' utilization of rheumatology tests.

Published
2016
Full Text
View/download PDF

18. Novel Method to Collect Medication Adverse Events in Juvenile Arthritis: Results From the Childhood Arthritis and Rheumatology Research Alliance Enhanced Drug Safety Surveillance Project

Author

Steven J. Spalding, Harry Gewanter, John F. Bohnsack, Marisa S. Klein-Gitelman, Beth Gottlieb, Timothy Beukelman, Leslie Abramson, Sampath Prahalad, Joyce J. Hsu, Jorge M. Lopez Benitez, Linda Ray, Karen Onel, Diana Milojevic, C. Egla Rabinovich, Peter R. Blier, Carol A. Wallace, Rachel E. Sobel, Elizabeth C. Chalom, Richard K. Vehe, Reema H. Syed, Audrey Hendrickson, Carol B. Lindsley, Roger Hollister, Andrew J. White, Andrea Hudgins, Michael Shishov, Kelly Rouster-Stevens, Kabita Nanda, Natasha M. Ruth, Norman T. Ilowite, Lakshmi N. Moorthy, Thomas G. Mason, Daniel J. Lovell, Sarah Ringold, Jennifer E. Weiss, and Matthew L. Stoll

Subjects
Drug, medicine.medical_specialty, business.industry, Childhood arthritis, media_common.quotation_subject, MEDLINE, Arthritis, medicine.disease, Confidence interval, Rheumatology, Clinical trial, Internal medicine, parasitic diseases, Physical therapy, medicine, business, Adverse effect, media_common
Abstract

Objective Few data are available regarding the rates of serious adverse events (SAEs) and important medical events (IMEs) outside of product-based registries and clinical trials for juvenile idiopathic arthritis (JIA). The Enhanced Drug Safety Surveillance Project (EDSSP) was developed to pilot a novel system to collect SAEs/IMEs in children with JIA. This analysis reports the results from this 4-year (2008–2012) EDSSP. Methods Participating physicians were surveyed monthly to ascertain whether their JIA patients experienced an SAE or IME. Sites were surveyed every 6 months to determine the number of unique JIA patients seen at each site during that 6-month period. Reporting rates were calculated per 100 person-years and 95% confidence intervals (95% CIs) were calculated based on a Poisson distribution. Results Thirty-seven Childhood Arthritis and Rheumatology Research Alliance sites with 115 physicians participated. The mean response rate to the monthly surveys was 65%. There were 147 total SAEs and 145 total IMEs. The largest proportion of SAEs and IMEs occurred in children with polyarticular JIA (39% and 37%, respectively). The majority of SAEs and IMEs were reported for patients receiving therapy with biologic agents (76% and 69%, respectively). The total event rate for SAEs and IMEs combined was 1.07 events per 100 person-years (95% CI 0.95–1.19). The rates for SAEs and IMEs were 0.54 per 100 person-years (95% CI 0.45–0.63) and 0.53 per 100 person-years (95% CI 0.49–0.62), respectively. Conclusion The EDSSP provided a simple tool for SAE/IME reporting within an established research network and resulted in a similar range of reported events as captured by a traditional product-based registry.

Published
2015
Full Text
View/download PDF

19. Safety of weekly adalimumab in the treatment of juvenile idiopathic arthritis and pediatric chronic uveitis

Author

Danielle R. Bullock, Rachel Cafferty, Richard K. Vehe, and Colleen K. Correll

Subjects
musculoskeletal diseases, Male, medicine.medical_specialty, Adolescent, Arthritis, Drug Administration Schedule, Uveitis, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Internal medicine, medicine, Adalimumab, Humans, 030212 general & internal medicine, Dosing, skin and connective tissue diseases, Adverse effect, Child, Retrospective Studies, 030203 arthritis & rheumatology, business.industry, Retrospective cohort study, General Medicine, medicine.disease, Arthritis, Juvenile, Treatment Outcome, Antirheumatic Agents, Child, Preschool, Methotrexate, Female, business, medicine.drug
Abstract

Weekly adalimumab dosing is used to treat juvenile idiopathic arthritis (JIA), uveitis, and other pediatric rheumatic diseases, but the safety of such dosing has not previously been studied. A retrospective chart review was conducted to assess the safety of weekly adalimumab. Demographic and clinical data were collected. Basic descriptive analysis was performed to assess for adverse events from weekly adalimumab. Sixty-nine patients at the University of Minnesota or Gillette Children's Hospital were identified as treated with weekly adalimumab. Sixty (87%) were eligible for the chart review. Weekly adalimumab was used most commonly to treat uveitis (28%, 17/60) and rheumatoid factor-negative polyarticular JIA (25%, 15/60). Mean age at the start of weekly dosing was 13.9 years. The majority of patients were concurrently treated with a non-steroidal anti-inflammatory drug and methotrexate. Fifty-three (90%) patients continued weekly dosing for greater than 3 months. The mean duration of weekly adalimumab was 2 years. Throughout the duration of weekly dosing, 24/60 (40%) patients had documented minor infections not requiring antimicrobials and 24/60 (40%) had documented infections requiring antimicrobial treatment. Only three patients (5%) had an infection requiring hospitalization. Two patients (3%) developed autoimmune disease. Laboratory abnormalities and injection site reactions were rare. Weekly adalimumab was used most commonly to treat uveitis and rheumatoid factor-negative polyarticular JIA, and mean duration of weekly dosing was 2 years. Serious adverse events were rare.

Published
2017

20. Proceedings of the 23rd Paediatric Rheumatology European Society Congress: part two

Author

David Kern, Masato Yashiro, Gerd Horneff, Ana P. Sakamoto, Berent J. Prakken, Paula Vähäsalo, Juergen Brunner, Ezgi H. Baris, Helen McCarthy, Janet E. McDonagh, A. Grom, Adriana Albu, Lenka Linkova, I. Nikishina, Daniel Álvarez de la Sierra, Bruno Papia, Peggy Lee, Luisa Giannone, Tobias Schwarz, Mekibib Altaye, Margarita Onoufriou, Tatiana Sleptsova, N. Ruperto, O Thana, A. Baheti, Ilonka Orbán, Kai Lehmberg, F. Zulian, Helga Sanner, Karin Palmblad, Kousuke Shabana, Sebastiaan Vastert, Marta Rusmini, Olga Vougiouka, Dirk Holzinger, D. Shaikhani, Shouichi Ohga, Ismail Dursun Dursun, Claire T Deakin, Ingrid Herta Rotstein Grein, Maria Trachana, Ariane Klein, Eugenia Enriquez, Angelo Ravelli, Paul A. Brogan, L.S. Nazarova, Laila Al Shaqshi, Paulina Vele, Liana Guerra, Antonia Pascarella, Jelena Vojinovic, Juliana Molina, Kjell Tullus, S. Rodionovskaya, Chris Scott, A. N. Olivieri, Cliff Taggart, Clare Heard, Ricardo Pujol Borrell, Jens Klotsche, Grendel Burrell, Oriany L. Pereira, Silvia Giliani, Sandra Pereira, Jennifer Horonjeff, Beth A. Mueller, Lyudmyla M. Byelyaeva, Sergio Tufik, Carlo Agostoni, Valentina Muratore, Rostislav M. Filonovich, Fiona Hawke, Virginia Messia, Bo Magnusson, Kerry West, Sara Murias, Mustafa Doğan, Hafize Emine Sönmez, Annet van Royen-Kerkhof, K. Minden, Raquel Campanilho-Marques, Reem Abdawani, Maria Ceci, Maria Ekelund, Seza Ozen, Ratna Puri, Girolamo Luppino, Shannon Carr, Rita A. Amorim, K Kobrová, Rachael D. Wright, Chantal Job-Deslandre, Daniel J. Lovell, Jorge Kalil, Yi-jin Gao, Kubra Ozturk, Fulvio Parentin, Ursula Fearon, Frank Weller-Heinemann, Elizabeth Ang, Charles A Mebus, Andrea Superti-Furga, Alina Ferster, Rikard Wicksell, Mohammadreza Modaressi, F La Torre, Ela Tarakci, Wendy Thomson, Giorgia Malighetti, Antonio Eleuteri, Helena K. Khrustaleva, Alan Easton, Alexander Mushkin, Sara Marsal Barril, Erkan Demirkaya, Florence Kanakoudi-Tsakalidou, Diana Ekdawy, Lana Tambić Bukovac, Suvi Peltoniemi, Nur Arslan, Hermine I. Brunner, Tim Rapley, Donatella Vairo, Kirill Savostyanov, Fumiko Okazaki, Rachel Corkhill, Tufan Kutlu, MG Alpigiani, Fabio Fernandes Morato Castro, Juliana Farhat, Butsabong Lerkvaleekul, Scolozzi Paolo, Akihiko Saitoh, Jason Dare, Gustavo Rocha, Tatiana V. Viktorova, Riva Brik, Jason Palman, Fabrizia Corona, Susan Nielsen, Johannes Roth, Ma. Theresa M. Collante, Leonardo Oliveira Mendonça, D. Alexeev, Randy Q. Cron, Sriharsha Grevich, Andrea L. Jorgensen, Lúcia Maria de Arruda Campos, Kiran Nistala, Fernando Martins, R. Cimaz, Angela C. Mosquera, Ruy Carrasco, Reyhan Dedeoglu, Giovanni Filocamo, J. Dare, Paula Keskitalo, Ana J. D. F. C. Lichtenfels, Florence Uettwiller, Umberto Conte, Gecilmara Salviato Pileggi, Michal Uher, Mercedes Chan, Sarka Fingerhutova, Anne M. Stevens, Peter Bale, Mikel Alberdi-Saugstrup, Olga L Kopchak, Thomas A. Griffin, Constantin Ailioaie, Clifton Bingham, Ekaterina Alexeeva, Loshinidevi D Bathi, Jane Hurst, AnnaCarin Horne, Laura Muntean, Nermin Uncu, Mara Carraro, C Vargas, Lorenzo Quartulli, Ayşenur Paç Kısaarslan, Angela Mauro, F. Corona, Donato Rigante, Helen J. Lachmann, Ana Cordeiro, Ivan Foeldvari, Faysal Gok, Tatiana Gonzalez, S. S. M. Kamphuis, Hasret Ayyildiz-Civan, Claudia Pastorino, Gleice C. S. Russo, J. B. Kuemmerle-Deschner, Serena Pastore, Nigel Klein, M. Jorini, Tatjana Freye, Maria Tsolia, Philippe Jacqmin, Suzanne M M Verstappen, Syuji Takei, Khalid Hussain, Renzo Marcolongo, Yuichi Yamasaki, Sharmila Jandial, K. Leon, Maria Pia Sormani, T. A. Simon, Mohammed Muzaffer, Catalina Mosquera, Clovis Aa Silva, Zelal Ekinci, Zübeyde Gündüz, Bernd Denecke, Felicitas Bellutti Enders, Despina Eleftheriou, Ishbel MacGregor, Andrew Cant, Luisa Bonafé, Valda Staņēviča, Helen E. Foster, Alberto Tommasini, Nora Bartholomä, Nural Kiper, A. Kardolus, Eloisa Bonfa, Alessandro Consolaro, Lillemor Berntson, Umberto Garagiola, Richard K. Vehe, Vanessa Bugni Miotto e Silva, Chihaya Imai, Kathleen G. Lomax, Brian Best, Barbara Bonafini, M. Toth, D. Rigante, Eiman Abdalla, Leona Prochazkova, Lucy Wedderburn, Lovro Lamot, S. Verazza, Raffaella Carlomagno, Gillian I. Rice, Norm Ilowite, K. de Leeuw, Havva Evrengül, Jerold Jeyaratnam, Andrew Zeft, Andrea Taddio, R. Podda, Samuel Cassidy, Grant S. Schulert, Silvia Rosina, Marija Jelušić, Olivier Gilliaux, Rubén Burgos-Vargas, Mao Mizuta, Akihiro Yachie, Angel Phuti, Antonio Zea Mendoza, Emily Boulter, Zane Dāvidsone, Sofia Torreggiani, Marco Cattalini, Natali W. Gormezano, Fatma Dedeoglu, Hercília Guimarães, A. Insalaco, Andrea Coda, Viktor A. Malievsky, Thomas Zumbrunn, Agostino Nocerino, Ronald Pederson, Katarzyna Kobusinska, Anasuya Hazra, Ananadreia S. Lopes, Elena Campione, Toshiyuki Kitoh, Elena Tsitsami, Henny G. Hotten, Radka Kaneva, R. J. E. M. Dolhain, Ndate Fall, Francesco Licciardi, Deepti Suri, G. D’Angelo, Valentina Seraya, Elżbieta Smolewska, Anastasia Dropol, Ezgi Deniz Batu, Andreas Woerner, Christine Arango, Nadia E. Aikawa, Zoilo Morel, Megan Yuasa, Sandra Ammann, Erbil Unsal, Tomohiro Kubota, Toshitaka Kizawa, Fabrizio De Benedetti, Catherine Laing, Liudmila Rakovska, Yonatan Butbul Aviel, J-Peter Haas, Marta Minute, Christine Alvey, Vasiliko Dermentzoglou, Vania Schinzel, Isree Leelayuwattanakul, Ekim Taskiran, Gabriele Simonini, N. Martin, Nathalie Canham, Nicky Brice, Beatrice Vergara, Ika Birkić, Cengizhan Acikel, Johannes-Peter Haas, Ruth Fritsch, Alisa Vitebskaya, Fatih Yazici, Iva Brito, Nataša Toplak, Veronica Moshe, Gordon J Hendry, Nadia Luca, Deniz Doğru-Ersöz, Marco Matucci-Cerinic, Claudia Toppino, Zoë Johnson, Beatrice Goilav, Siyaram Didel, K. Marzan, Tamar B. Rubinstein, Angela Barnicoat, Peter Nourse, Thita Pacharapakornpong, Adele Civino, Inmaculada Calvo Penades, H. I. Brunner, Massimo Imazio, V. Gerloni, Ayla Kacar, Heinrike Schmeling, Marija Perica, Silvestre García de la Puente, Tadej Avcin, Filipa Oliveira-Ramos, S. Arsenyeva, Phillip J Hashkes, Sabrina Schuller, Adriana Rodriguez Vidal, Kathy de Graaf, Giedre Januskeviciute, M. Kaleda, Lee Dossetter, Jelena Basic, Elena Kaschenko, Erik Sundberg, Gizem Pamuk, Marek Zak, I. Foeldvari, Rachael Quarmby, Marc D. Natter, Antonarakis Gregory, P. A. Brogan, João Eurico Fonseca, Andrea Jorgensen, Ana F. Mourão, Gaurav Gulati, Yelda Bilginer, Banu Acar Celikel, Utako Kaneko, Karen L. Durrant, Alice Grossi, Maurizio Aricò, Ibrahim Al Zakwani, Yildirim Karslioglu, Takuji Murata, Monika Stoll, Maria Teresa Terreri, Ariana Kariminejad, Teresa A. Simon, Laura B Lewandowski, Marina Garcia Prat, Walter G. Ferlin, Albena Telcharova, Giovanni Maria Severini, Judith Wienke, Panagiota Nalbanti, Hakan Poyrazoglu, Athimalaipet V. Ramanan, Manisha Lamba, Z. Guo, J. Bohnsack, Norberto Guelbert, John F. Bohnsack, Lucy R. Wedderburn, Elvira Cannizzaro Schneider, Raul Gutiérrez Suárez, Debra Grech, Yonit Reis, Chris Pruunsild, Amit Rawat, Nienke M. ter Haar, Hiroshi Tamai, Alexander Pushkov, A. De Fanti, Valentina Marzetti, Sheila Weitzman, I. E. M. Bultink, Dilek Keskin, Sania Valieva, Klaus Tenbrock, Ana Luisa Rodriguez-Lozano, Lianne Kearsley-Fleet, Luca Messina, Chiara Gorio, Amra Adrovic, Stephanie J. W. Shoop, Davide Cumetti, Sana Al Zuhbi, Helena Khrustaleva, E. Zirkzee, Elio Castagnola, Clarissa Pilkington, Jingyao Leong, Vitor A. Teixeira, Reinhard Würzner, Sonia Melo Gomes, Orla Killeen, Antony C. Fisher, Sevket Erbil Unsal, Edward M. Behrens, Kristiina Aalto, Rebecca Nicolai, Thomas C. Stock, Luiz Cláudio Danzmann, Y. K. O. Teng, Stephen D. Marks, Fotios Papachristou, Valda Stanevicha, Richard Saffrey, Elizabeth Ralph, Johannes Peter Haas, Mary Slatter, Maria Tsinti, Mehmet Alikasifoglu, Mónica Martínez Gallo, Rayfel Schneider, Rosa Maria Rodrigues Pereira, Maria F. D. A. Giacomin, Alfésio Luís Ferreira Braga, Giulia Camilla Varnier, James McElnay, Jessica Foster, Ingrida Rumba-Rozenfelde, Francesca Minoia, Laurence Goffin, Roger C. Allen, Zehra S. Arıcı, Mette Nørgaard, Alberto Martini, Hofer Michaël, Andreas Eikelberg, Junko Yasumura, Maria José Santos, Tom Wolfs, Ada B. Sinoplu, Natalia Balera Ferreira Pinto, Michael Lang, Umberto Corpora, Hidenobu Kaneyasu, Fiorenzo Gaita, Olga Lomakina, Dimitrina Mihaylova, Gian Luca Erre, Fugen Cullu-Cokugras, Mesut Topdemir, Sriram Krishnaswami, Irina Nikishina, Noortje Groot, S. Pastore, Joke Dehoorne, Paula Estanqueiro, Shafe Fahoum, Francisca Aguiar, Mabel Ladino, Nico M Wulffraat, Jana Franova, Helena Erlandsson-Harris, Denise Pires Marafon, Adrian Liston, Edward H. Schuchman, Jaime C. Branco, Maria Teresa R. Terreri, Radoslava Saraeva, Ulrika Järpemo Nykvist, Maria Cristina Maggio, Kazuko Yamazaki, Lídia Teixeira, Hanquinet Sylviane, Ricardo Yepez, Susan Maillard, Tommy Gerschman, G. Horneff, Anne-Louise Ponsonby, Meredith Riebschleger, Alessandra Alongi, José Melo-Gomes, Iris Haug, Maria De Iorio, Ekaterina Alekseeva, Jan-Inge Henter, Elisa Pisaneschi, H. Kupper, Martina Niewerth, Berta López Montesinos, Shunji Hasegawa, Zahra Hadipour, R. M. Kuester, Silvia Maestroni, Pilar Guarnizo, Antonio Brucato, Tamaki Nakamura, Gustavo Antonio Moreira, Chaim Putterman, A. Hospach, Joost Frenkel, Svetlana O Salugina, A. Ravelli, Pavla Dolezalova, Gunnar L. Olsson, Eva González-Roca, Ellen Dalen Arnstad, Mohammad Alhemairi, Tina Hinnershitz, P. Quartier, Yildirim Karsioglu, Davinder Singh Grewal, Sergio Davì, Gökçen D. Tuğcu, Tomo Nozawa, Emily Robinson, M. C. Maggio, Maria Ballabio, Eleonora Bellucci, Alexei A. Grom, Rosa M. Pereira, Federica Vanoni, Shumpei Yokota, Justine A. Ellis, Helen Bristow, Mohammad-Hassan Moradinejad, Ricardo Russo, Harun Evrengül, Mario Abinun, Laura Carenini, Francesca Santarelli, C. Wallace, Maria Beatriz Fonseca, Timothy Beukelman, Saša Sršen, Véronique Hentgen, Sezgin Sahin, Silvia Zaffarano, Salvatore Albani, Valentin Brodszky, Clovis A. Silva, Graciela Espada, Jana Pachlopnik Schmid, Margaux Gerbaux, Stefano Stagi, Valentina Leone, Brian Leroux, Isabelle Koné-Paut, Gabriella Giancane, Soley Omarsdottir, Benedetta Schiappapietra, Alessandra Carobbio, Ricardo Menendez-Castro, Yoshifumi Kawano, Ozge Erdemli, Monia Lorini, Arjan Boltjes, Ellen Nordal, Carol A. Wallace, Mustafa Çakan, Reni Tzveova, Stefano Lanni, Salah Shokry, Kirsty McLellan, Qiong Wu, Nilay Arman, Adelina Tsakova, Michael W. Beresford, A. Consolaro, Francesca Bovis, Margarita Ganeva, Christoph Kessel, A. Martini, Taichi Kanetaka, Andre Schultz, Flora McErlane, Ronnie Wang, Mojca Zajc Avramovič, Thaschawee Arkachaisri, Boris Huegle, Funda Öztunç, Jane E Munro, Yanick J. Crow, Hiroyuki Wakiguchi, Rita Fonseca, Kim E. Nichols, Mia Glerup, Nils Venhoff, R. Filonovich, Erika Van Nieuwenhove, Nadia Rafiq, Elena Kamenets, Yasuo Nakagishi, Giampietro Farronato, Consuelo Modesto Caballero, Tulay Erkan, Jan Bonhoeffer, Jack Bukowski, Myrthes Toledo Barros, Gladys C. C. Esteves, Mirta Lamot, Rosa Alcobendas, Cláudia Moura, Florencia María Barbé-Tuana, Joo Guan Yeo, Mark Friswell, Yuko Sugita, Ari Shapiro, Nagla Abdelrahman, Phu-Quoc Lê, Kevin Murray, Susanne M Benseler, Anna Monica Bianco, J. Kalabic, Ana Catarina Duarte, Ivan Caiello, Joyce Davidson, Maria Isabel Gonzalez Fernandez, Larisa Zajtseva, Ebun Omoyinmi, Jaime de Inocencio, Dragana Lazarevic, Ritambhra Nada, Claudia Saad-Magalhães C, G. Conti, Andrew Gennery, Caroline Jones, Christophe Lelubre, Brian Rusted, Geneviève Lapeyre, Giulia Zani, Alina Boteanu, Maria T. Terreri, Nataliya Panko, Julia Albrecht, Federica Mongini, Lucio Giordano, Daniela Kaiser, Robert Nelson, Hans-Iko Huppertz, Gonca Keskindemirci, Karla Ištuk, Raffaele Strippoli, Dorota Rowczenio, Emma MacDermott, Roberta Caorsi, Emiliano Marasco, Sandra Sousa, Fatoş Yalçınkaya, Jaanika Ilisson, Yuki Kimura, Marco Turco, Nami Okamoto, Parveen Bhatti, Ekaterina M. Kuchinskaya, Stefano Volpi, Min Wang, Jeffrey M. Craig, M. Bijl, Giusi Prencipe, Fatemeh Tahghighi, W. van Dijk, Christiaan Scott, Masaki Shimizu, Alexey Maletin, Braydon Meyer, Joost F Swart, Sylvia Costa Lima Farhat, Anna Taparkou, R. Fritsch-Stork, Paolo Cressoni, Reiji Hirano, I. Chyzheuskaya, Stefania Simou, Kseniya Isayeva, Mariluz Gámir Gámir, Paivi Miettunen, Francesca Ricci, Ruta Šantere, George Lazaros, Madeleine Rooney, Stefan Stefanov, Huseyin Ozkan, Céline La, Boris Hügle, Vita Dolžan, P. Barone, R. Gallizzi, Aline L. de Oliveira, Silvia Federici, Lauren J. Lahey, Kimme L. Hyrich, Claudia Saad-Magalhães, Selçuk Yüksel, Valda Stanevica, Silvia De Pauli, Seid-Reza Raeeskarami, Calin Lazar, Sema Akman, Laurence Chatel, Kirsten Minden, Ismaiel A. Tekko, Philipp Henneke, E. Cortis, Elena Košková, Gil Amarilyo, Ana M. Marín Sánchez, Antonella Insalaco, Z. Birsin Ozcakar, Melissa Mariti Fraga, Lena Klevenvall, Luis Lira, Phoi-Ngoc Duong, Tatiana Bzarova, Neus Quilis, Wilco de Jager, Gary Sterba, Rina Denisova, Miroslav Harjacek, Eve M D Smith, N Ruperto, Gemma Lepri, Evgeniya Chistyakova, Rachel Kaufmann, Liliana Lourenço Jorge, Violeta Panaviene, Helena Canhão, Riccardo Belli, Grigoris Pardalos, Larisa I. Zajtseva, Nicolino Ruperto, Ezgi Batu, Paola Montesano, Alexander Solyom, Nicola Smith, Ales Janda, Sagar Bhattad, Liora Harel, Philip N. Hawkins, Gozde Yucel, François Willermain, Paolo Picco, Alessandro Rimini, Gordana Susic, Esi M. Morgan, Jessica Beckmann, Arina Lazareva, Agustin Remesal, Özge Altuğ Gücenmez, Troels Herlin, Andreas Groll, T. Yuraga, Ekaterina Zaharova, Adriana E. M. Sallum, Zeynep Birsin Özçakar, D. Milojevic, Can Kosukcu, Isabella Ceccherini, Sandrine Lacassagne, Tania M. Castro, R. Consolini, Klaus Müller, Dogan Simsek, Frank Rühle, Katia Kozu, Femke van Wijk, Yasin Sahin, Jonathan S. Hausmann, Gokalp Basbozkurt, M. Cattalini, Mª José Santos, Norman T. Ilowite, Adriana M. E. Sallum, Simona Rednic, Sirisucha Soponkanaporn, Giancarla Di Landro, Semanur Özdel, Timothy R. Radstake, Anastasia Wiener, Betül Sözeri, Estefania Quesada-Masachs, E. Zholobova, Joshua Newson, Ozgur Kasapcopur, Davide Montin, Terence Flood, Amir Mendelson, Manuela Pardeo, Flávia Heloísa dos Santos, Jamie Eaton, Vignesh Pandiarajan, Lyudmila Belyaeva, Edson Amaro Junior, Claudio Arnaldo Len, Tamás Constantin, Livia de Freitas Keppeke, Cristina Ferrari, Margarita Soloshenko, C. Rabinovich, David Popp, Jeremy Sokolove, Jaymi Taiani, Chiara Passarelli, de Min Cristina, José Costa, Stefanie Herresthal, Thomas Giner, Laure Caspers, Dilek Konukbay, Ulrich Salzer, Jorre S. Mertens, Marijan Frković, Yosef Uziel, Sabrina Chiesa, Luisa Bracci-Laudiero, Anders Fasth, Raul A. Chavez Valencia, Jordan T. Jones, Francesca Lancini, Alessandra Ferrari, Dana Nemcova, Mark Difrancesco, Ricardo Figueira, John Mitchell, Zohreh Nademi, E. Fedorov, Thomas Vogl, Carine Wouters, Mónica Eusébio, Hannah Leahey, Alessandra Pontillo, Marco Gattorno, Mandica Vidović, Lucas L. van den Hoogen, Mikhail Kostik, Giovanni Corsello, Gian Marco Moneta, Richard Mouy, Mariana Rodrigues, Veronica Medeghini, Gökçe Gür, Lucas Kich Grun, Stephan Ehl, Edi Paleka Bosak, Walter Ferlin, Hanna Lythgoe, Tsuyoshi Yamatou, Navdha R. Ramchurn, Carolina Furtado, Estefania Barral, Cecilia Lazea, Nikolay Tzaribachev, Vahid Ziaee, Fatemeh Hadipour, Alberto Sifuentes Giraldo, Kimberly Gilmour, Marite Rygg, Anna Valenti, María M Katsicas, Raju Khubchandani, Despoina Maritsi, Alessandra Tesser, R. M. Laxer, Clotilde Alizzi, Francisco Rivas-Larrauri, Aysen Tezcaner, Anne Dennos, Vasiliki Tzimouli, Vibeke Strand, Banu Acar, Fabio Candotti, Kseniia V. Danilko, Joachim Schultze, María Luz Gámir Gámir, Alessia Omenetti, Berit Flatø, Ruth Eraso, Bernard Lauwerys, Angela Pistorio, Andressa G. F. Alves, Gerd Ganser, Sara Signa, Ana Lopes, Emese Kiss, Charlene Foley, Sylvia Kamphuis, Maja Di Rocco, Kenan Barut, Ilaria Parissenti, Aida Koka, Nicholas Ng, Francis Corazza, Vinícius L. Braga, Laura E. Schanberg, Karin Beutel, Camila Hirotsu, Jonathan D Akikusa, Mihaela Sparchez, Karoline Ehlert, Jordi Anton, Adriana M. Sallum, Maria Cristina Castiglione, Surjit Singh, Julie Jones, Katya Temelkova, Tania S. Amin, Jasmin B Kuemmerle-Deschner, Samantha Bell, Sakda A.-O. Vallipakorn, Manuel Salgado, Filipa Ramos, Balahan Makay, Nadezhda Tsurikova, Gianmaria Viglizzo, Rosalba Ferraro, Sandra Hansmann, Nilgün Çakar, Ismail Dursun, Maria Stavrakidou, T. Bzarova, Sally Pino, Dhouib Amira, Salla Kangas, Antonella Meini, Dirk Foell, Dolunay Gürses, Dace Bērziņa, A. Speziale, Juan I. Arostegui Gorospe, Kelly L. Mieszkalski, Dawn M. Wahezi, S. Davì, Radoslav Srp, Daniel J. Kingsbury, Alexei A Grom, Falcini Fernanda, Peng Yin, Claire T. Deakin, Eva Hlavackova, Pavla Doležalová, Maria Mercedes Picarelli, Ezgi D. Batu, Alessandra Tricarico, Soamarat Vilaiyuk, Ivan Costa-Filho, A. Civino, Lukas Hackl, Pilar Gomez, Michael Hofer, M Manuela Costa, Zbigniew Zuber, Elena Ligostaeva, Carlos D. Rose, Jozef Hoza, Pranoot Tanpaiboon, Bonnie Vlahos, Sandra Garrote Corral, Martina Finetti, Giedre Grigelioniene, Susanne M. Benseler, X Wei, Pieter Van Dijkhuizen, Lee Nelson, Elettra Santori, David Martino, Anju Gupta, Nuray Aktay Ayaz, Noa Rabinowicz, Susan Shenoi, Rachel Chiaroni-Clarke, Claudia Bracaglia, Ruhan Düşünsel, M. Hofer, Rolando Cimaz, Juan I. Aróstegui, Ana Filipa Mourão, Ivonne Arroyo, Laura Damian, Marco F. C. D. Silva, D. J. Lovell, Marta Torcoletti, Clara Malagón, Luisa Klotz, Krisztina Sevcic, Douglas Veale, Belen Serrano Benavente, N. Groot, Polyxeni Pratsidou, Nicole Johnson, Karen Wynne, SR Rodionovskaya, Melania Saifridova, Kaara Tiewsoh, Ryan F. Donnelly, Fernanda Falcini, Valérie Badot, M. G. Alpigiani, L. Breda, Farida Abduragimova, Veronika Gjertsen Rypdal, Sophie Hambleton, E. Chalom, Anna Horne, Antonio Novelli, O. Kostareva, Panagiotis Tziavas, Yara Barrense-Dias, Cecilia Bava, Sarah Ringold, William H. Robinson, Sirirat Charuvanij, D. Kingsbury, Shuichi Ito, Luiz A. A. Pereira, Marcus Herbert Jones, S. I. Valieva, Flavio Sztajnbok, Florence Guilhot, Cristina de Min, Adriana Diaz-Maldonado, Simone A. Lotufo, Beril Talim, M. Heinrich, Paul Newland, and Laura Pagani

Subjects
030203 arthritis & rheumatology, 03 medical and health sciences, medicine.medical_specialty, 0302 clinical medicine, Rheumatology, business.industry, 030220 oncology & carcinogenesis, Family medicine, Pediatrics, Perinatology and Child Health, medicine, Immunology and Allergy, business, Paediatric rheumatology
Published
2017
Full Text
View/download PDF

21. Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II

Author

Chester B. Whitley, Alicia Kunin-Batson, Lynda E. Polgreen, Richard K. Vehe, Kyle Rudser, Patricia I. Dickson, and Jeanine Jarnes Utz

Subjects
musculoskeletal diseases, 0301 basic medicine, medicine.medical_specialty, Mucopolysaccharidosis, 030105 genetics & heredity, Short stature, 03 medical and health sciences, 0302 clinical medicine, Endocrinology, Internal medicine, Mucopolysaccharidosis I, Genetics, medicine, Adalimumab, skin and connective tissue diseases, Bone, lcsh:QH301-705.5, Molecular Biology, Inflammation, lcsh:R5-920, business.industry, Enzyme replacement therapy, medicine.disease, Crossover study, 3. Good health, Transplantation, Clinical trial, lcsh:Biology (General), Joint, Physical therapy, medicine.symptom, lcsh:Medicine (General), business, 030217 neurology & neurosurgery, medicine.drug, Research Paper
Abstract

Mucopolysaccharidosis I and II are lysosomal storage disorders that, despite treatment with hematopoietic cell transplantation (HCT) and/or enzyme replacement therapy (ERT), continue to cause significant skeletal abnormalities leading to pain, stiffness, physical dysfunction, and short stature. Tumor necrosis factor – alpha (TNF-α) is elevated in individuals with MPS I and II and associated with pain and physical dysfunction. Therefore, we evaluated the safety and effects of the TNF-α inhibitor adalimumab in patients with MPS I and II in a 32-week, randomized, double blind, placebo-controlled, crossover study of adalimumab at a dose of 20 mg (weight 15– 5° in seven of eight joints in Subject #1 and five of eight joints in Subject #2 (range 7.0° to 52.8°). There was no change in the PPQ, 6MWT, or hand dynamometer. Data from this small pilot study suggest that treatment with adalimumab is safe, tolerable, and may improve ROM, physical function, and possibly pain, in children with MPS I or II. However, additional clinical trials are needed before this therapy should be recommended as part of clinical care.

Published
2016

22. Childhood Arthritis and Rheumatology Research Alliance Consensus Clinical Treatment Plans for Juvenile Dermatomyositis with Persistent Skin Rash

Author

Angela Byun Robinson, Adam M. Huber, Richard K. Vehe, Charles H. Spencer, Sandy D. Hong, Philip Kahn, Ruy Carrasco, Susan Kim, Homaira Rahimi, Jennifer E. Weiss, Ann M. Reed, and Brian M. Feldman

Subjects
Pediatrics, medicine.medical_specialty, Consensus, Childhood arthritis, Immunology, Anti-Inflammatory Agents, Patient characteristics, Dermatomyositis, 030207 dermatology & venereal diseases, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Adrenal Cortex Hormones, Internal medicine, Immunology and Allergy, Medicine, Humans, Child, Clinical treatment, Juvenile dermatomyositis, 030203 arthritis & rheumatology, business.industry, Immunoglobulins, Intravenous, Exanthema, medicine.disease, Rash, Muscle disease, Methotrexate, Antirheumatic Agents, Physical therapy, Drug Therapy, Combination, medicine.symptom, business, medicine.drug
Abstract

Objective.Juvenile dermatomyositis (JDM) is the most common form of idiopathic inflammatory myopathy in children. While outcomes are generally thought to be good, persistence of skin rash is a common problem. The goal of this study was to describe the development of clinical treatment plans (CTP) for children with JDM characterized by persistent skin rash despite complete resolution of muscle involvement.Methods.The Childhood Arthritis and Rheumatology Research Alliance, a North American consortium of pediatric rheumatologists and other healthcare providers, used a combination of Delphi surveys and nominal group consensus meetings to develop CTP that reflected consensus on typical treatments for patients with JDM with persistent skin rash.Results.Consensus was reached on patient characteristics and outcome assessment. Patients should have previously received corticosteroids and methotrexate (MTX). Three consensus treatment plans were developed. Plan A added intravenous immunoglobulin (IVIG) if it was not already being used. Plan B added mycophenolate mofetil, while Plan C added cyclosporine. Continuation of previous treatments, including corticosteroids, MTX, and IVIG, was permitted in plans B and C.Conclusion.Three consensus CTP were developed for use in children with JDM and persistent skin rash despite complete resolution of muscle disease. These CTP reflect typical treatment approaches and are not to be considered treatment recommendations or standard of care. Using prospective data collection and statistical methods to account for nonrandom treatment assignment, it is expected that these CTP will be used to allow treatment comparisons, and ultimately determine the best treatment for these patients.

Published
2016

23. Race, Income, and Disease Outcomes in Juvenile Dermatomyositis

Author

Thomas B. Graham, Lisa Imundo, Steven J. Spalding, Kathleen M. O'Neil, Mara L. Becker, Marilynn Punaro, Joyce J. Hsu, Heather Benham, Beth Gottlieb, Kenneth N. Schikler, Jennifer M. P. Woo, Mary Ellen Riordan, Angela Byun Robinson, Sivia K. Lapidus, Daniel J. Kingsbury, Deborah McCurdy, Pamela F. Weiss, Jenna Tress, Nora G. Singer, Kathryn S. Torok, Deborah Rothman, Jennifer E. Weiss, Karen Onel, Brian M. Feldman, J. R. Hollister, Richard K. Vehe, Gloria C. Higgins, Anna Huttenlocher, Hilary M. Haftel, Thomas A. Griffin, Thomas J. A. Lehman, Donald P. Goldsmith, Polly I. Ferguson, Eyal Muscal, Joni Dean, Sampath Prahalad, Leslie Abramson, Eleanor S. Anderson, Mark F. Hoeltzel, Philip Kahn, Hermine I. Brunner, Reema H. Syed, Ali Yalcindag, Kristin E. Klein, Judyann C. Olson, C. Egla Rabinovich, Carol B. Lindsley, Marisa S. Klein-Gitelman, Natasha M. Ruth, Norman T. Ilowite, Fatma Dedeoglu, Timothy Beukelman, Lydia M. Walters, Lawrence S. Zemel, Andrew J. White, Peter R. Blier, Sarah Ringold, Susan Kim, Emily von Scheven, Rita S. Jerath, Ann M. Reed, Kathryn Phillippi, Lawrence Jung, and Michael A. Malloy

Subjects
Male, Weakness, medicine.medical_specialty, Childhood arthritis, Disease, Family income, Dermatomyositis, 03 medical and health sciences, 0302 clinical medicine, Quality of life, Calcinosis, medicine, Humans, 030212 general & internal medicine, Child, Juvenile dermatomyositis, Retrospective Studies, 030203 arthritis & rheumatology, business.industry, Racial Groups, medicine.disease, Health equity, Child, Preschool, Pediatrics, Perinatology and Child Health, Physical therapy, Income, Female, medicine.symptom, business, Demography
Abstract

To determine the relationships among race, income, and disease outcomes in children with juvenile dermatomyositis (JDM).Data from 438 subjects with JDM enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry were analyzed. Demographic data included age, sex, race, annual family income, and insurance status. Clinical outcomes included muscle strength, presence of rash, calcinosis, weakness, physical function, and quality of life measures. Disease outcomes were compared based on race and income.Minority subjects were significantly more likely to have low annual family income and significantly worse scores on measures of physical function, disease activity, and quality of life measures. Subjects with lower annual family income had worse scores on measures of physical function, disease activity, and quality of life scores, as well as weakness. Black subjects were more likely to have calcinosis. Despite these differences in outcome measures, there were no significant differences among the racial groups in time to diagnosis or duration of disease. Using calcinosis as a marker of disease morbidity, black race, annual family income$50 000 per year, negative antinuclear antibody, and delay in diagnosis12 months were associated with calcinosis.Minority race and lower family income are associated with worse morbidity and outcomes in subjects with JDM. Calcinosis was more common in black subjects. Further studies are needed to examine these associations in more detail, to support efforts to address health disparities in subjects with JDM and improve disease outcomes.

Published
2016

24. Proceedings of the 2016 Childhood Arthritis and Rheumatology Research Alliance (CARRA) Scientific Meeting

Author

Peggy Lee, Victor Hasselblad, Anja Schnabel, Timo Siepmann, Liza Bermudez, Ting Wang, Gloria C. Higgins, Ann M. Reed, Reinhard Berner, Katharine Moore, Sriharsha Grevich, Amy J. Starr, Jerome C. Lane, Mark F. Hoeltzel, Emily von Scheven, Yvonne Chiu, Brian M. Feldman, Edward J Oberle, Susan Kim, Laurence Greenbaum, Adam M. Huber, Judyann C. Olson, Suzanne Li, Lorie Luyrink, Elizabeth Roth Wojcicki, Dana Toib, Doruk Erkan, Michelle Bayer, Rie Karasawa, Lynn Spiegel, Charles Victor, Polly J. Ferguson, T. Brent Graham, Mark Connelly, James N. Jarvis, Laura B Lewandowski, Jacinthe Bisaillon, Elizabeth D. Mellins, Stephanie Luca, Gloria Higgins, Suhas Ganguli, Mary Hintermeyer, Jennifer E. Weiss, Jessica Foster, Phillip I. Tarr, Timothy S. H. Kwok, Kristyn L. Maletta, Christine Smith, Mileka Gilbert, Shirley M. L. Tse, L. Nandini Moorthy, Ciaran Duffy, Beatrice Goilav, Kevin Baszis, Rayfel Schneider, Marc D. Natter, Martha Rodriguez, Chitra Lalloo, Yukiko Kimura, Rob C. Fuhbrigge, Eleanor Pullenayegum, Megan L. Curran, Hernan Correa, Colleen K. Correll, Cassyanne L. Aguiar, Suzanne C. Li, Khaled Alsaeid, Laura Brungs, Tim Beukelman, Sampath Prahalad, Susan D. Thompson, Xiaohu Li, Brian Best, Tao Liu, Stacey M. Martinetti, Kevin W. Baszis, Alexis Boneparth, Monica Tsoras, Darshan H. Mehta, Anne M. Stevens, Annabelle N. Chua, Sangeeta Sule, Gretchen R. Heckel, Argerie Tsimicalis, Lucie Brosseau, Helen Bristow, Sarah Ringold, Thomas G. Mason, Mara L. Becker, Laura E. Schanberg, Ezra M. Cohen, Kaiyu Jiang, Yonit Sterba Ruas, Katie Stewart, Anne Dennos, Alexei A. Grom, Kalpana Manthiram, Yanmin Chen, Joshua Newsom, Peter R. Blier, Jennifer M. P. Woo, Lori B. Tucker, Michelle L. Dossett, Deirdre De Ranieri, Ginger Janow, Nicole Johnson, Hatice Ezgi Baris, Andrew H. Eichenfield, Laiping Wong, Pamela F. Weiss, Marisa S. Klein-Gitelman, Esi M. Morgan, Jennifer Stinson, Christian M. Hedrich, Ashlea Cook, Marsha M. Malloy, Megan Yuasa, M.S.C.E. Mara Becker M.D., Patrick J. McGrath, Max Shenberger, Beth Gottlieb, Daryl M. Okamura, Timothy Beukelman, Esra Meidan, Natasha M. Ruth, Lisha Zhu, Kazuo Yudoh, Kelly L. Mieszkalski, James A. Jegers, Mayumi Tamaki, Peter A. Nigrovic, Karen Onel, Christiaan Scott, Linda Wagner-Weiner, Kader Cetin Gedik, Ursula Range, Vikas R. Dharnidharka, Melissa S Tesher, Daniel J. Lovell, Jason Dare, Natalie J. Shiff, Karine Toupin April, Soko Setoguchi, Michael J. Buck, Yvonne C. Lee, Nadia Luca, Sarah Campillo, Suhas M. Radhakrishna, Lauren J. Lahey, Anna Carmela P. Sagcal-Gironella, Brandi Stevens, William H. Robinson, Hazim Alsaeed, Rachel Kaufmann, Anca D. Askanase, Stacy P. Ardoin, Egla Rabinovich, Paul Dancey, Kathryn S. Torok, Norm Ilowite, Elizabeth L. Roth-Wojcicki, Mario Medvedovic, Brian G. Leroux, Thomas A. Griffin, Megumi Tanaka, Richard K. Vehe, Lampros Fotis, Ronald M. Laxer, Salma Siddique, Joyce Hui-Yuen, Marilynn Punaro, Roberta A. Berard, Gabriele Hahn, Halima Moncrieffe, Kathryn M. Edwards, Dominic O. Co, Lauren Harris, Hannah Leahey, George Tomlinson, Maria Ibarra, Ciarán M. Duffy, Sarah Thomson, Jeremy Sokolove, Stacy Ardoin, Sandy D. Hong, Fatma Dedeoglu, Dustin J. Hahn, Nur Shaikh, Y. Ingrid Goh, Amir B. Orandi, Margalit Rosenkranz, Naweed Tajuddin, Elena Pope, Grendel Burrell, Shannon Carr, C. Egla Rabinovich, Jasim Alfailakawi, Roger Davis, Anabelle Chua, Toshiko Sato, Kathleen M. O'Neil, Anthony R. French, Lisa Imundo, Paula Morris, Joseph A Cafazzo, Maria F. Ibarra, Scott E. Wenderfer, Tamar Rubinstein, Danielle R. Bullock, Huan Xu, Joyce J. Hsu, Ritesh Korumilli, Hamid Alenezi, Julia Barsalou, John Matelski, Nicole Hershey, Heather Van Mater, Marsha Malloy, Lei Zhang, and Mark F. Bennett

Subjects
030203 arthritis & rheumatology, Pediatrics, medicine.medical_specialty, business.industry, Norm (group), 3. Good health, 03 medical and health sciences, 0302 clinical medicine, Alliance, Rheumatology, Griffin, Pediatrics, Perinatology and Child Health, Immunology and Allergy, Medicine, 030212 general & internal medicine, business, Humanities
Abstract

Author(s): Fotis, Lampros; Shaikh, Nur; Baszis, Kevin; French, Anthony; Tarr, Phillip; Grevich, Sriharsha; Lee, Peggy; Ringold, Sarah; Leroux, Brian; Leahey, Hannah; Yuasa, Megan; Foster, Jessica; Sokolove, Jeremy; Lahey, Lauren; Robinson, William; Newsom, Joshua; Stevens, Anne; Karasawa, Rie; Tamaki, Mayumi; Tanaka, Megumi; Sato, Toshiko; Yudoh, Kazuo; Jarvis, James N; Moncrieffe, Halima; Bennett, Mark F; Tsoras, Monica; Luyrink, Lorie; Xu, Huan; Prahalad, Sampath; Morris, Paula; Dare, Jason; Nigrovic, Peter A; Rosenkranz, Margalit; Becker, Mara; O’Neil, Kathleen M; Griffin, Thomas; Lovell, Daniel J; Grom, Alexei A; Medvedovic, Mario; Thompson, Susan D; Zhu, Lisha; Jiang, Kaiyu; Wong, Laiping; Buck, Michael J; Chen, Yanmin; Moncrieffe, Halima; Brungs, Laura; Liu, Tao; Wang, Ting; Jarvis, James N; Alsaeid, Khaled; Alfailakawi, Jasim; Alenezi, Hamid; Alsaeed, Hazim; Beukelman, Tim; Natter, Marc; Ilowite, Norm; Mieszkalski, Kelly; Burrell, Grendel; Best, Brian; Bristow, Helen; Carr, Shannon; Dennos, Anne; Kaufmann, Rachel; Kimura, Yukiko; Schanberg, Laura; Blier, Peter R; Boneparth, Alexis; Wenderfer, Scott E; Moorthy, L Nandini; Radhakrishna, Suhas M; Sagcal-Gironella, Anna Carmela P; von Scheven, Emily; Gedik, Kader Cetin; Siddique, Salma; Aguiar, Cassyanne L; Erkan, Doruk; Cohen, Ezra; Lee, Yvonne; Dossett, Michelle; Mehta, Darshan; Davis, Roger; Gilbert, Mileka; Goilav, Beatrice; Meidan, Esra

Published
2016
Full Text
View/download PDF

25. Inflammatory arthritis in pediatric patients with morphea

Author

Richard K. Vehe, Bryce A. Binstadt, Colleen K. Correll, Sheilagh Maguiness, Patricia M. Hobday, and Sakeen W. Kashem

Subjects
Male, medicine.medical_specialty, Adolescent, Anti-nuclear antibody, Inflammatory arthritis, Arthritis, Comorbidity, Dermatology, Pediatrics, Severity of Illness Index, Cohort Studies, Scleroderma, Localized, 030207 dermatology & venereal diseases, 03 medical and health sciences, Age Distribution, 0302 clinical medicine, Internal medicine, medicine, Humans, Rheumatoid factor, Sex Distribution, Child, Retrospective Studies, 030203 arthritis & rheumatology, business.industry, Incidence, Hydroxychloroquine, Prognosis, medicine.disease, Rheumatology, Methotrexate, Child, Preschool, Female, Polyarthritis, business, Immunosuppressive Agents, Morphea, medicine.drug
Abstract

Background Morphea or localized scleroderma is an inflammatory disorder resulting in fibrosis of the skin and subcutaneous tissues. Joint contractures, arthralgias, and functional compromise are recognized associations of pediatric morphea. The co-existence of inflammatory arthritis and morphea is not well-described in the literature. Objective To investigate the relationship between pediatric morphea and inflammatory arthritis with regards to cutaneous, musculoskeletal, and laboratory findings and treatment regimens. Methods A systematic retrospective chart review of 53 patients with pediatric morphea was performed and analyzed for morphea subtypes, arthritic joint involvement, serum autoantibodies, and therapeutic interventions. Results Eleven out of 53 patients had polyarthritis that involved joints unrelated to the site of the cutaneous morphea. These patients were mostly girls with either the linear or generalized subtypes of morphea. Serum levels of antinuclear antibodies were more significantly elevated in patients with arthritis. All children were treated with methotrexate in addition to other systemic or topical immunosuppressive agents. Limitations This was a small, single-center retrospective study. Conclusion Pediatric morphea co-existed with inflammatory arthritis in 11 of 53 children. Further understanding and appreciation of this relationship may direct more intensive therapy and musculoskeletal screening.

Published
2018
Full Text
View/download PDF

27. Elevated TNF-α is associated with pain and physical disability in mucopolysaccharidosis types I, II, and VI

Author

Lynda E. Polgreen, Jeanine Jarnes Utz, Kyle Rudser, Richard K. Vehe, Patricia I. Dickson, Elsa Shapiro, Chester B. Whitley, and Alicia Kunin-Batson

Subjects
0301 basic medicine, Male, congenital, hereditary, and neonatal diseases and abnormalities, medicine.medical_specialty, Physical disability, Adolescent, Endocrinology, Diabetes and Metabolism, Mucopolysaccharidosis, Health Status, Pain, Inflammation, Biochemistry, Article, 03 medical and health sciences, Endocrinology, Internal medicine, Genetics, medicine, Humans, Disabled Persons, Prospective Studies, Child, Molecular Biology, Pain Measurement, business.industry, Tumor Necrosis Factor-alpha, Chronic pain, nutritional and metabolic diseases, Enzyme replacement therapy, Mucopolysaccharidoses, medicine.disease, Surgery, Transplantation, Patient Outcome Assessment, 030104 developmental biology, Cross-Sectional Studies, Child, Preschool, Disease Progression, Tumor necrosis factor alpha, Female, medicine.symptom, business, Natural history study
Abstract

Children and adults with the lysosomal storage diseases mucopolysaccharidosis (MPS) types I, II and VI live shortened lives permeated by chronic pain and physical disability. Current treatments do not alleviate these problems. Thus there is a critical need to understand the mechanism of chronic pain and disability in MPS in order to improve the way we treat patients. A potential target is inflammation.We hypothesized that excessive inflammation mediated by the tumor necrosis factor-α (TNF-α) inflammatory pathway is the fundamental cause of much of the chronic pain and physical disability in MPS.55 patients with MPS I, II, or VI were enrolled over the course of a 5-year prospective longitudinal natural history study and evaluated annually for 2-5years. 51 healthy controls were enrolled in a separate cross-sectional study of bone and energy metabolism. TNF-α was measured by ELISA. Pain and physical disability were measured by the Children's Health Questionnaire - Parent Form 50 (CHQ-PF50). Differences in log-transformed TNF-α levels and associations with CHQ domains were evaluated using a linear mixed effects model with random intercept.TNF-α levels were measured in 48 MPS (age: 5-17years; 35% female) and 51 controls (age: 8-17years; 53% female). Among MPS, 22 (46%) were treated with hematopoietic cell transplantation (HCT) alone, 24 (50%) with enzyme replacement therapy (ERT) alone, and 2 (4%) with both HCT and ERT. TNF-α levels are higher in MPS compared to healthy controls (p0.001). Higher TNF-α levels are associated with increased pain and decreased physical function, social limitations due to physical health, and physical summary score (all p0.05). TNF-α levels were not significantly associated with the general health score. TNF-α levels did not change significantly over time in MPS.Higher TNF-α levels are implicated in the pain and decreased physical function present in individuals with MPS despite treatment with ERT and/or HCT, suggesting that TNF-a inhibition could potentially be a useful adjunctive therapy. Further investigation into the role of TNF-α inhibition in MPS to decrease pain and improve physical function is indicated.

Published
2015

29. Early Recognition of and Intervention for Susac Syndrome in a Teenager With Encephalopathy

Author

Danielle R. Bullock, Robert M. Beardsley, Erick D. Bothun, Patricia M. Hobday, Richard K. Vehe, Robert M. Rennebohm, and Sean Rivera

Subjects
Pediatrics, medicine.medical_specialty, business.industry, Immunology, Encephalopathy, medicine.disease, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, 030220 oncology & carcinogenesis, Intervention (counseling), medicine, Immunology and Allergy, business, 030217 neurology & neurosurgery, Susac Syndrome
Published
2016
Full Text
View/download PDF

30. AB0879 Pharmacokinetics, Safety, and Tolerability of Tofacitinib in Paediatric Patients from Two To Less than Eighteen Years of Age with Juvenile Idiopathic Arthritis

Author

Zbigniew Zuber, Richard K. Vehe, Charles A Mebus, G. Horneff, Daniel J. Kingsbury, Nikolay Tzaribachev, Elżbieta Smolewska, Ivan Foeldvari, Hermine I. Brunner, Manisha Lamba, Anasuya Hazra, Daniel J. Lovell, N Ruperto, A Martini, Sriram Krishnaswami, U. Conte, M. Wang, Ronnie Wang, E. Koskova, and Christine Alvey

Subjects
030203 arthritis & rheumatology, medicine.medical_specialty, Tofacitinib, business.industry, Immunology, PK Parameters, Tofacitinib 5 MG, Multiple dose, General Biochemistry, Genetics and Molecular Biology, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Pharmacokinetics, Tolerability, Internal medicine, medicine, Immunology and Allergy, 030212 general & internal medicine, business, Paediatric patients, Janus kinase inhibitor
Abstract

Background Tofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). Objectives Report the pharmacokinetics (PK), safety and taste acceptability of tofacitinib following multiple oral doses in patients (pts) 2

Published
2016
Full Text
View/download PDF

31. Using quality improvement methodology and tools to reduce patient wait time in a paediatric subspecialty rheumatology clinic

Author

Richard K Vehe, Bayardo Garay, Denise Erlanson, Bryce A Binstadt, Colleen K Correll, Nora Fitzsimmons, Patricia M Hobday, Allison Hudson, Shawn Mahmud, Mona M Riskalla, Sara Kramer, Sheng Xiong, and Danielle R Bullock

Subjects
Medicine (General), R5-920
Abstract

Our paediatric rheumatology clinic has experienced inefficient patient flow. Our aim was to reduce mean wait time and minimise variation for patients. Baseline data showed that most waiting occurs after a patient has been roomed, while waiting for the physician. Wait time was not associated with a patient’s age, time of day, day of the week or individual physician. We implemented a checkout sheet and staggered start times. After a series of plan–do–study–act cycles, we observed an initial 26% reduction in the variation of wait time and a final 17% reduction in the mean wait time. There was no impact on patient–physician contact time. Overall, we demonstrate how process improvement methodology and tools were used to reduce patient wait time in our clinic, adding to the body of literature on process improvement in an ambulatory setting.

Published
2021
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results