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

Yukiko Kimura; Sriharsha Grevich; Timothy Beukelman; Esi Morgan; Peter A. Nigrovic; Kelly Mieszkalski; T. Brent Graham; Maria Ibarra; Norman Ilowite; Marisa Klein-Gitelman; Karen Onel; Sampath Prahalad; Marilynn Punaro; Sarah Ringold; Dana Toib; Heather Van Mater; Jennifer E. Weiss; Pamela F. Weiss; Laura E. Schanberg; L. Abramson; E. Anderson; M. Andrew; N. Battle; M. Becker; H. Benham; J. Birmingham; P. Blier; A. Brown; H. Brunner; A. Cabrera; D. Canter; D. Carlton; B. Caruso; L. Ceracchio; E. Chalom; J. Chang; P. Charpentier; K. Clark; J. Dean; F. Dedeoglu; B. Feldman; P. Ferguson; M. Fox; K. Francis; M. Gervasini; D. Goldsmith; G. Gorton; B. Gottlieb; T. Griffin; H. Grosbein; The CARRA Registry Investigators

doi:10.1186/s12969-017-0157-1

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

Yukiko Kimura^*, Sriharsha Grevich, Timothy Beukelman, Esi Morgan, Peter A. Nigrovic, Kelly Mieszkalski, T. Brent Graham, Maria Ibarra, Norman Ilowite, Marisa Klein-Gitelman, Karen Onel, Sampath Prahalad, Marilynn Punaro, Sarah Ringold, Dana Toib, Heather Van Mater, Jennifer E. Weiss, Pamela F. Weiss, Laura E. Schanberg, L. AbramsonE. Anderson, M. Andrew, N. Battle, M. Becker, H. Benham, J. Birmingham, P. Blier, A. Brown, H. Brunner, A. Cabrera, D. Canter, D. Carlton, B. Caruso, L. Ceracchio, E. Chalom, J. Chang, P. Charpentier, K. Clark, J. Dean, F. Dedeoglu, B. Feldman, P. Ferguson, M. Fox, K. Francis, M. Gervasini, D. Goldsmith, G. Gorton, B. Gottlieb, T. Griffin, H. Grosbein, The CARRA Registry Investigators

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › peer-review

40 Scopus citations

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 language	English (US)
Article number	23
Journal	Pediatric Rheumatology
Volume	15
Issue number	1
DOIs	https://doi.org/10.1186/s12969-017-0157-1
State	Published - Apr 11 2017

Funding

The authors wish to thank Thomas A. Phillips, M.A. (DCRI) for statistical assistance and Jane Winsor, B.A. (DCRI) for database programming. PAN was supported by the Fundación Bechara. We also acknowledge the following CARRA Registry site investigators and coordinators: L. Abramson, E. Anderson, M. Andrew, N. Battle, M. Becker, H. Benham, T. Beukelman, J. Birmingham, P. Blier, A. Brown, H. Brunner, A. Cabrera, D. Canter, D. Carlton, B. Caruso, L. Ceracchio, E. Chalom, J. Chang, P. Charpentier, K. Clark, J. Dean, F. Dedeoglu, B. Feldman, P. Ferguson, M. Fox, K. Francis, M. Gervasini, D. Goldsmith, G. Gorton, B. Gottlieb, T. Graham, T. Griffin, H. Grosbein, S. Guppy, H. Haftel, D. Helfrich, G. Higgins, A. Hillard, J.R. Hollister, J. Hsu, A. Hudgins, C. Hung, A. Huttenlocher, N. Ilowite, A. Imlay, L. Imundo, C.J. Inman, J. Jaqith, R. Jerath, L. Jung, P. Kahn, A. Kapedani, D. Kingsbury, K. Klein, M. Klein-Gitelman, A. Kunkel, S. Lapidus, S. Layburn, T. Leh-man, C. Lindsley, M. Macgregor-Hannah, M. Malloy, C. Mawhorter, D. McCurdy, K. Mims, N. Moorthy, D. Morus, E. Muscal, M. Natter, J. Olson, K. O’Neil, K. Onel, M. Orlando, J. Palmquist, M. Phillips, L. Ponder, S. Prahalad, M. Punaro, D. Puplava, S. Quinn, A. Quintero, C. Rabinovich, A. Reed, C. Reed, S. Ringold, M. Riordan, S. Roberson, A. Robinson, J. Rossette, D. Rothman, D. Russo, N. Ruth, K. Schikler, A. Sestak, B. Shaham, Y. Sherman, M. Simmons, N. Singer, S. Spalding, H. Stapp, R. Syed, E. Thomas, K. Torok, D. Trejo, J. Tress, W. Upton, R. Vehe, E. von Scheven, L. Walters, J.E. Weiss, P.F. Weiss, N. Welnick, A. White, J. Woo, J. Wootton, A. Yalcindag, C. Zapp, L. Zemel, and A. Zhu. This work was completed with support from an Arthritis Foundation Innovative Research Grant. The National Institute of Arthritis and Musculoskeletal and Skin Disease at the National Institutes of Health (NIH 1RC1AR058605-01) supported the development of the consensus treatment plans. The CARRA Registry was supported by grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, Friends of CARRA, the Arthritis Foundation, and the NIH (RC2AR058934).

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
Rheumatology
Immunology and Allergy

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10.1186/s12969-017-0157-1

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Kimura, Y., Grevich, S., Beukelman, T., Morgan, E., Nigrovic, P. A., Mieszkalski, K., Graham, T. B., Ibarra, M., Ilowite, N., Klein-Gitelman, M., Onel, K., Prahalad, S., Punaro, M., Ringold, S., Toib, D., Van Mater, H., Weiss, J. E., Weiss, P. F., Schanberg, L. E., ... The CARRA Registry Investigators (2017). Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans. Pediatric Rheumatology, 15(1), Article 23. https://doi.org/10.1186/s12969-017-0157-1

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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 = "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 Norman Ilowite 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 and {The CARRA Registry Investigators}",

note = "Publisher Copyright: {\textcopyright} 2017 The Author(s).",

year = "2017",

month = apr,

day = "11",

doi = "10.1186/s12969-017-0157-1",

language = "English (US)",

volume = "15",

journal = "Pediatric Rheumatology",

issn = "1546-0096",

publisher = "BioMed Central",

number = "1",

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Kimura, Y, Grevich, S, Beukelman, T, Morgan, E, Nigrovic, PA, Mieszkalski, K, Graham, TB, Ibarra, M, Ilowite, N, Klein-Gitelman, M, Onel, K, Prahalad, S, Punaro, M, Ringold, S, Toib, D, Van Mater, H, Weiss, JE, Weiss, PF, Schanberg, LE, Abramson, L, Anderson, E, Andrew, M, Battle, N, Becker, M, Benham, H, Birmingham, J, Blier, P, Brown, A, Brunner, H, Cabrera, A, Canter, D, Carlton, D, Caruso, B, Ceracchio, L, Chalom, E, Chang, J, Charpentier, P, Clark, K, Dean, J, Dedeoglu, F, Feldman, B, Ferguson, P, Fox, M, Francis, K, Gervasini, M, Goldsmith, D, Gorton, G, Gottlieb, B, Griffin, T, Grosbein, H & The CARRA Registry Investigators 2017, 'Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans', Pediatric Rheumatology, vol. 15, no. 1, 23. https://doi.org/10.1186/s12969-017-0157-1

TY - JOUR

T1 - Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans

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

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.

AU - The CARRA Registry Investigators

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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UR - http://www.scopus.com/inward/citedby.url?scp=85018462181&partnerID=8YFLogxK

U2 - 10.1186/s12969-017-0157-1

DO - 10.1186/s12969-017-0157-1

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AN - SCOPUS:85018462181

SN - 1546-0096

VL - 15

JO - Pediatric Rheumatology

JF - Pediatric Rheumatology

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ER -

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

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