New Medications Are Needed for Children with Juvenile Idiopathic Arthritis

Hermine Brunner¹, Laura Schanberg ², Yukiko Kimura ³, Anne Dennos ⁴, Guy Eakin ⁵, Dominic Co ⁶, Robert Colbert ⁷, Robert Fuhlbrigge ⁸, Ellen Goldmuntz ⁹, Daniel Kingsbury ¹⁰, Sandra Mintz ¹¹, Karen Onel ¹², Cathy Patty-Resk ¹³, Lisa G. Rider ¹⁴, Rayfel Schneider ¹⁵, Allen Watts ¹⁶, Emily von-Scheven ¹⁷, Daniel J. Lovell ¹⁸, Timothy Beukelman ¹⁹ and for PRCSG Advisory Council and CARRA Registry Investigators ²⁰, ¹Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, ²Duke University Medical Center, Durham, NC, ³Joseph M Sanzari Children's Hospital, Hackensack University Medical Center, Cincinnati, OH, ⁴Duke Clinical Research Institute, Durham, NC, ⁵Arthritis Foundation, Atlanta, ⁶of Wisconsin School of Medicine and Public Health, Madison, WI, ⁷Pediatric Clinical Trials Unit, Pediatric Translational Research Branch, NIAMS, NIH, Bethesda, MD, ⁸Department of Rheumatology, Children’s Hospital Colorado, Denver, CO, ⁹NIAID, Washingto, DC, ¹⁰LHS Pediatrics, Prtland, OR, ¹¹Children's Hospital of Los Angeles, Los Angeles, CA, ¹²Hospital for Special Surgery, Division of Pediatric Rheumatology, New York, NY, ¹³Children's Hospital of Michigan, Detroit, MI, ¹⁴Environmental Autoimmunity Group, Clinical Research Branch, NIEHS, NIH, Bethesda, MD, ¹⁵Hospital for Sick Children, Toronto, ON, Canada, ¹⁶Cincinnati Children's Hospital MEdical Center, Cincinnati, OH, ¹⁷University of California San Francisco, San Francisco, ¹⁸Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children’s Hospital Medical Center, Cincinnati, ¹⁹University of Alabama Birmingham, Birmingham, AL, ²⁰Pediatric Rheumatology Collaborative Study Group, Cincinnati, OH

Meeting: 2019 ACR/ARP Annual Meeting

Keywords: medical necessity and biologic drugs, Pediatric rheumatology, registries

Session Information

Date: Monday, November 11, 2019

Title: 4M092: Pediatric Rheumatology – Clinical II: JIA (1812–1817)

Session Type: ACR Abstract Session

Session Time: 2:30PM-4:00PM

Background/Purpose: Existing legislation in the United States (US) promotes the study of new medications in children. Biologic disease-modifying-drugs (bDMARDs) and small molecules proven effective and safe in adults with rheumatoid arthritis and other forms of inflammatory arthritis require testing in children with juvenile idiopathic arthritis (JIA) to establish proper dosing, effectiveness, and safety. Several bDMARDs have been approved for the treatment of JIA in the US, but not all children with JIA have a complete clinical response to the available medications and are treated with medications off-label (i.e., anakinra, golimumab, infliximab, rituximab, secukinumab, tofacitinib, ustekinumab). The purpose of this research was to document the continuing medical need for new approved medications for the treatment of JIA.

Methods: The electronic medical record of JIA patients (n= 1599) treated at Cincinnati Children’s Hospital Medical Center (CCHMC) since 2008 were reviewed for medication use and disease activity over time. JIA patients enrolled in the Childhood Arthritis & Rheumatology Research Alliance (CARRA) Registry (n=7,379) were assessed for medication use and disease activity at the most recent Registry visit. Medication need was defined as active JIA despite sequential use of ≥2 bDMARDs. Active JIA was defined as either a) physician-global assessment of JIA activity (MD-global; 0-10; 0 = inactive) >3 OR b) number of active joints (AJC) >3 OR c) patient-global assessment of well-being (Pt-global; 0-10; 0=very well) >3. Medication failure was only assessed for patients with complete data (a-c).

Results: At CCHMC, polyarticular-course JIA (40%), systemic JIA (9%) and juvenile psoriatic arthritis/enthesitis-related arthritis (JPSA/ERA; 17%) were common, and only 16% had persistent-oligoarticular JIA. Overall, use of bDMARDs (n=829; 53%) was common. Systemic JIA (85%) and jPSA/ERA patients (79%) were most commonly treated with bDMARDs. At least 5% (25/1599) of patients had failed >5 bDMARDs. Of 829 biologic users in the CCHMC cohort, 304 (37%) children were exposed to non-approved bDMARDs. Among 278 CCHMC patients with jPSA /ERA 27 (9.7%) had failed >2 bDMARDs. In the CARRA Registry, 46% of JIA patients had polyarticular disease course, 8% systemic JIA, and 18% jPSA/ERA; 4766 (65%) children had received a bDMARD with 1122 (24%) of these children receiving bDMARDs off label. Among 1351 jPSA/ERA patients in the CARRA Registry, about 10% failed >2 bDMARDs with active disease at their most recent visit. Table 1 summarizes medication failures in both cohorts.

Conclusion: Data from a population-based cohort and a large national registry demonstrate a profound medical need for additional therapies to control JIA signs and symptoms, despite the availability of several approved biologic DMARDs. Given FDA approval ensures bDMARD access, the testing of new medications in JIA as they become available to treat adults is critical to further improve JIA outcomes.

Table 1

Disclosure: H. Brunner, ., 2, 5, 8, AbbVie, 5, AstraZeneca, 5, Bayer, 5, Bristol-Myers Squibb, 2, 5, EMD Serono, 5, Genentech, 2, 5, 8, GlaxoSmithKline, 5, Janssen, 5, Lilly, 5, Novartis, 5, 8, Pfizer, 2, 5, R-Pharm, 5, Sanofi, 5, UCB, 5; L. Schanberg, CARRA, 9, Childhood Arthritis and Rheumatology Research Alliance, 2, Sanofi, 5, 9, SOBI, 5, UCB, 5; Y. Kimura, Novartis, 5, Sobi, 5; A. Dennos, Childhood Arthritis and Rheumatology Research Alliance, 3; G. Eakin, Arthritis Foundation, 3, 6; D. Co, None; R. Colbert, Eli Lilly and Company, 2, 9; R. Fuhlbrigge, CARRA, 6, 9; E. Goldmuntz, None; D. Kingsbury, None; S. Mintz, Arthritis Research Professionals, 6; K. Onel, None; C. Patty-Resk, None; L. Rider, ., 2, 9, aTyr, 9, Bristol Myers Squibb, 2, Cure JM Foundation, 2, 9, Eli Lilly and Company, 9, Hope Pharmaceuticals, 2, Lilly-drug, 9, MedImmune / AstraZeneca, 9, MedImmune/AstraZeneca, 9, NIEHS, 2, NIEHS, NIH, 2, NIH, 2; R. Schneider, Novimmune, 5, Sobi, 5, Novartis, 5; A. Watts, None; E. von-Scheven, CARRA, 6, 9; D. Lovell, Abbott, 5, 9, AbbVie, 5, 9, Amgen, 5, 9, AstraZeneca, 5, Astra-Zeneca Pharm, 5, Biogen, 5, Boehringer Ingelheim, 5, Boeringher Ingelheim, 5, Bristol-Myers Squibb, 5, 9, Celgene, 5, Forest Research, 9, Forest Research Institute, 5, Genentech, 5, 8, GlaxoSmithKline, 5, Hoffmann-La Roche, 5, 9, Horizon, 5, Jannsen, 5, Janssen, 5, 9, Johnson & Johnson, 5, Novartis, 5, 9, Pfizer, 5, 9, Roche, 5, 9, Takeda, 5, 9, UBC, 5, Wyeth Pharm, 5, 8; T. Beukelman, CARRA, 6, UCB, 5; f. and CARRA Registry Investigators, None.

To cite this abstract in AMA style:

Brunner H, Schanberg L, Kimura Y, Dennos A, Eakin G, Co D, Colbert R, Fuhlbrigge R, Goldmuntz E, Kingsbury D, Mintz S, Onel K, Patty-Resk C, Rider L, Schneider R, Watts A, von-Scheven E, Lovell D, Beukelman T, and CARRA Registry Investigators f. New Medications Are Needed for Children with Juvenile Idiopathic Arthritis [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/new-medications-are-needed-for-children-with-juvenile-idiopathic-arthritis/. Accessed .

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