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Pilot Study of the Juvenile Dermatomyositis Consensus Treatment Plans: A CARRA Registry Study.
Liu, Kuan; Tomlinson, George; Reed, Ann M; Huber, Adam M; Saarela, Olli; Bout-Tabaku, Sharon M; Curran, Megan; Dvergsten, Jeffrey A; Eberhard, Barbara A; Jung, Lawrence K; Kim, Susan; Ringold, Sarah; Rouster-Steven, Kelly A; Tesher, Melissa; Wahezi, Dawn M; Feldman, Brian M.
Afiliación
  • Liu K; K. Liu, MMath, B.M. Feldman, MD, MSc, FRCPC, The Hospital for Sick Children, Toronto, and Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.
  • Tomlinson G; G. Tomlinson, PhD, Department of Medicine, University Health Network, Toronto, Ontario, Canada.
  • Reed AM; G. Tomlinson, PhD, Department of Medicine, University Health Network, Toronto, Ontario, Canada.
  • Huber AM; A.M. Reed, MD, Department of Pediatrics, Duke University Medical Center, Durham, North Carolina, USA.
  • Saarela O; A.M. Huber, MD, MSc, IWK Health Centre, Halifax, Nova Scotia, Canada.
  • Bout-Tabaku SM; O. Saarela, PhD, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.
  • Curran M; S.M. Bout-Tabaku, MD, MSCE, Department of Pediatric Medicine, Sidra Medicine, Doha, Weill Cornell Medicine - Qatar, Doha, Qatar.
  • Dvergsten JA; M.L. Curran, MD, Department of Pediatrics, University of Colorado, Denver, Colorado, USA.
  • Eberhard BA; J.A. Dvergsten, MD, Department of Pediatrics, Duke University Medical Center, Durham, and Department of Pediatrics, Duke Children's Hospital, Durham, North Carolina, USA.
  • Jung LK; B.A. Eberhard, MBBS, MS, Division of Pediatric Rheumatology, Steven and Alexandra Cohen Children's Medical Center of New York, Lake Success, New York, and Department of Pediatrics, Hofstra Northwell School of Medicine, Hempstead, New York, USA.
  • Kim S; L.K. Jung, MD, Division of Rheumatology, Children's National Health System, Washington, D.C., USA.
  • Ringold S; S. Kim, MD, MMSc, Department of Pediatrics, University of California San Francisco, San Francisco, California, USA.
  • Rouster-Steven KA; S. Ringold, MD, Seattle Children's Hospital and Research Institute, Seattle, Washington, USA.
  • Tesher M; K.A. Rouster-Steven, MD, Department of Pediatrics, Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta, Georgia, USA.
  • Wahezi DM; M. Tesher, MD, Comer Children's Hospital, University of Chicago Medical Center, Chicago, Illinois, USA.
  • Feldman BM; D.M. Wahezi, MD, Msc, Division of Pediatric Rheumatology, the Children's Hospital at Montefiore, Bronx, New York, USA.
J Rheumatol ; 48(1): 114-122, 2021 01 01.
Article en En | MEDLINE | ID: mdl-32238513
OBJECTIVES: To determine the feasibility of comparing the Childhood Arthritis and Rheumatology ResearchAlliance (CARRA) consensus treatment plans (CTP) in treating moderate new-onset juvenile dermatomyositis (JDM) using the CARRA registry, and to establish appropriate analytic methods to control for confounding by indication and missing data. METHODS: A pilot cohort of 39 patients with JDM from the CARRA registry was studied. Patients were assigned by the treating physician, considering patient/family preferences, to 1 of 3 CTP: methotrexate (MTX) and prednisone (MP); intravenous (IV) methylprednisolone, MTX, and prednisone (MMP); or IV methylprednisolone, MTX, prednisone, and IV immunoglobulin (MMPI). The primary outcome was the proportion of patients achieving moderate improvement at 6 months under each CTP. Statistical methods including multiple imputation and inverse probability of treatment weighting were used to handle missing data and confounding by indication. RESULTS: Patients received MP (n = 13), MMP (n = 18) and MMPI (n = 8). Patients in all CTP had significant improvement in disease activity. Of the 36 patients who remained in our pilot study at 6 months, 16 (44%) of them successfully achieved moderate improvement at 6 months (6/13, 46% for MP; 7/15, 47% for MMP; 3/8, 38% for MMPI). After correcting for confounding, there were no statistically significant pairwise differences between the CTP (P = 0.328-0.88). CONCLUSION: We gained valuable experience and insight from our pilot study that can be used to guide the design and analysis of comparative effectiveness studies using the CARRA registry CTP approach. Our analytical methods can be adopted for future comparative effectiveness studies and applied to other rare disease observational studies.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Artritis Juvenil / Reumatología / Dermatomiositis Tipo de estudio: Guideline / Observational_studies Límite: Child / Humans Idioma: En Revista: J Rheumatol Año: 2021 Tipo del documento: Article País de afiliación: Canadá Pais de publicación: Canadá
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Artritis Juvenil / Reumatología / Dermatomiositis Tipo de estudio: Guideline / Observational_studies Límite: Child / Humans Idioma: En Revista: J Rheumatol Año: 2021 Tipo del documento: Article País de afiliación: Canadá Pais de publicación: Canadá