Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans

The CARRA Registry Investigators

Research output: Contribution to journalArticle

11 Citations (Scopus)

Abstract

Objectives: To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry. Methods: Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone. methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9 months. Trial registration: clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled). Results: Thirty patients were enrolled at 13 sites; eight patients were started on a non-biologic CTP (2 GC, 6 MTX) and 22 patients on a biologic CTP (12 IL1i, 10 IL6i) at disease onset. Demographic and disease features were similar between CTP groups. CTP choice appeared to segregate by site preference. CID off GC was achieved by 37% (11 of 30) including 11/22 (50%) starting a biologic CTP compared to 0/8 starting a non-biologic CTP (p = 0.014). There were four serious adverse events: two infections, one appendicitis and one macrophage activation syndrome. Conclusions: The CARRA systemic JIA CTP pilot study demonstrated successful implementation of CTPs using the CARRA registry infrastructure. Having demonstrated feasibility, a larger study using CTP response to better determine the relative effectiveness of treatments for new-onset systemic JIA is now underway.

Original languageEnglish (US)
Article number23
JournalPediatric Rheumatology
Volume15
Issue number1
DOIs
StatePublished - Apr 11 2017

Fingerprint

Juvenile Arthritis
Rheumatology
Arthritis
Glucocorticoids
Research
Therapeutics
Registries
Methotrexate
Interleukin-6
Macrophage Activation Syndrome
Family Physicians
Appendicitis
Demography

Keywords

  • Biologic response modifiers
  • Comparative effectiveness
  • Pediatric rheumatology
  • Registries
  • Still's disease
  • Systemic Juvenile Idiopathic Arthritis

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Immunology and Allergy
  • Rheumatology

Cite this

Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans. / The CARRA Registry Investigators.

In: Pediatric Rheumatology, Vol. 15, No. 1, 23, 11.04.2017.

Research output: Contribution to journalArticle

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title = "Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans",
abstract = "Objectives: To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry. Methods: Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone. methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9 months. Trial registration: clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled). Results: Thirty patients were enrolled at 13 sites; eight patients were started on a non-biologic CTP (2 GC, 6 MTX) and 22 patients on a biologic CTP (12 IL1i, 10 IL6i) at disease onset. Demographic and disease features were similar between CTP groups. CTP choice appeared to segregate by site preference. CID off GC was achieved by 37{\%} (11 of 30) including 11/22 (50{\%}) starting a biologic CTP compared to 0/8 starting a non-biologic CTP (p = 0.014). There were four serious adverse events: two infections, one appendicitis and one macrophage activation syndrome. Conclusions: The CARRA systemic JIA CTP pilot study demonstrated successful implementation of CTPs using the CARRA registry infrastructure. Having demonstrated feasibility, a larger study using CTP response to better determine the relative effectiveness of treatments for new-onset systemic JIA is now underway.",
keywords = "Biologic response modifiers, Comparative effectiveness, Pediatric rheumatology, Registries, Still's disease, Systemic Juvenile Idiopathic Arthritis",
author = "{The CARRA Registry Investigators} and Yukiko Kimura and Sriharsha Grevich and Timothy Beukelman and Esi Morgan and Nigrovic, {Peter A.} and Kelly Mieszkalski and Graham, {T. Brent} and Maria Ibarra and Ilowite, {Norman Todd} and Marisa Klein-Gitelman and Karen Onel and Sampath Prahalad and Marilynn Punaro and Sarah Ringold and Dana Toib and {Van Mater}, Heather and Weiss, {Jennifer E.} and Weiss, {Pamela F.} and Schanberg, {Laura E.} and L. Abramson and E. Anderson and M. Andrew and N. Battle and M. Becker and H. Benham and J. Birmingham and P. Blier and A. Brown and H. Brunner and A. Cabrera and D. Canter and D. Carlton and B. Caruso and L. Ceracchio and E. Chalom and J. Chang and P. Charpentier and K. Clark and J. Dean and F. Dedeoglu and B. Feldman and P. Ferguson and M. Fox and K. Francis and M. Gervasini and D. Goldsmith and G. Gorton and B. Gottlieb and T. Griffin and H. Grosbein",
year = "2017",
month = "4",
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T1 - Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans

AU - The CARRA Registry Investigators

AU - Kimura, Yukiko

AU - Grevich, Sriharsha

AU - Beukelman, Timothy

AU - Morgan, Esi

AU - Nigrovic, Peter A.

AU - Mieszkalski, Kelly

AU - Graham, T. Brent

AU - Ibarra, Maria

AU - Ilowite, Norman Todd

AU - Klein-Gitelman, Marisa

AU - Onel, Karen

AU - Prahalad, Sampath

AU - Punaro, Marilynn

AU - Ringold, Sarah

AU - Toib, Dana

AU - Van Mater, Heather

AU - Weiss, Jennifer E.

AU - Weiss, Pamela F.

AU - Schanberg, Laura E.

AU - Abramson, L.

AU - Anderson, E.

AU - Andrew, M.

AU - Battle, N.

AU - Becker, M.

AU - Benham, H.

AU - Birmingham, J.

AU - Blier, P.

AU - Brown, A.

AU - Brunner, H.

AU - Cabrera, A.

AU - Canter, D.

AU - Carlton, D.

AU - Caruso, B.

AU - Ceracchio, L.

AU - Chalom, E.

AU - Chang, J.

AU - Charpentier, P.

AU - Clark, K.

AU - Dean, J.

AU - Dedeoglu, F.

AU - Feldman, B.

AU - Ferguson, P.

AU - Fox, M.

AU - Francis, K.

AU - Gervasini, M.

AU - Goldsmith, D.

AU - Gorton, G.

AU - Gottlieb, B.

AU - Griffin, T.

AU - Grosbein, H.

PY - 2017/4/11

Y1 - 2017/4/11

N2 - Objectives: To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry. Methods: Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone. methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9 months. Trial registration: clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled). Results: Thirty patients were enrolled at 13 sites; eight patients were started on a non-biologic CTP (2 GC, 6 MTX) and 22 patients on a biologic CTP (12 IL1i, 10 IL6i) at disease onset. Demographic and disease features were similar between CTP groups. CTP choice appeared to segregate by site preference. CID off GC was achieved by 37% (11 of 30) including 11/22 (50%) starting a biologic CTP compared to 0/8 starting a non-biologic CTP (p = 0.014). There were four serious adverse events: two infections, one appendicitis and one macrophage activation syndrome. Conclusions: The CARRA systemic JIA CTP pilot study demonstrated successful implementation of CTPs using the CARRA registry infrastructure. Having demonstrated feasibility, a larger study using CTP response to better determine the relative effectiveness of treatments for new-onset systemic JIA is now underway.

AB - Objectives: To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry. Methods: Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone. methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9 months. Trial registration: clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled). Results: Thirty patients were enrolled at 13 sites; eight patients were started on a non-biologic CTP (2 GC, 6 MTX) and 22 patients on a biologic CTP (12 IL1i, 10 IL6i) at disease onset. Demographic and disease features were similar between CTP groups. CTP choice appeared to segregate by site preference. CID off GC was achieved by 37% (11 of 30) including 11/22 (50%) starting a biologic CTP compared to 0/8 starting a non-biologic CTP (p = 0.014). There were four serious adverse events: two infections, one appendicitis and one macrophage activation syndrome. Conclusions: The CARRA systemic JIA CTP pilot study demonstrated successful implementation of CTPs using the CARRA registry infrastructure. Having demonstrated feasibility, a larger study using CTP response to better determine the relative effectiveness of treatments for new-onset systemic JIA is now underway.

KW - Biologic response modifiers

KW - Comparative effectiveness

KW - Pediatric rheumatology

KW - Registries

KW - Still's disease

KW - Systemic Juvenile Idiopathic Arthritis

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