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Abstract Number: L09

Efficacy and Safety of Tofacitinib in Patients with Active Systemic Juvenile Idiopathic Arthritis

Hermine Brunner1, Caifeng Li2, Kogie Chinniah3, Yosef Uziel4, Olga Synoverska5, Sujata Sawhney6, Inmaculada Calvo Penades7, Ingrid Clara Louw8, Meiping Lu9, Pooja Nikunj Patel10, Pamela F. Weiss11, Cheng Chang12, Ivana Vranic13, Shixue Liu14, Annette Diehl15, Jose L. Rivas16, Carol A. Connell17, Gary G. Koch18, Alberto Martini19, Daniel J. Lovell1, Nicolino Ruperto20 and the PRINTO and PRCSG investigators, 1Cincinnati Children’s Hospital Medical Center, and University of Cincinnati, Cincinnati, OH, 2Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Bejing, China, 3Department of Paediatrics and Child Health, University of Kwa-Zulu, and Enhancing Care Foundation, Durban, South Africa, 4Pedriatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center and Israel Tel Aviv University School of Medicine, Kfar Saba, Israel, 5Ivano-Frankivsk National Medical University, Ivano-Frankivsk, Ukraine, 6Sir Ganga Ram Hospital, New Delhi, India, 7Hospital Universitario y Politécnico La Fe, Valencia, Spain, 8Panorama Medical Centre, Cape Town, South Africa, 9Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China, 10Ann & Robert H. Lurie Children’s Hospital of Chicago; Northwestern University School of Medicine, Chicago, IL, 11Division of Rheumatology, Children's Hospital of Philadelphia, and University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 12Pfizer Inc, New York, NY, 13Pfizer Ltd, Tadworth, United Kingdom, 14Pfizer Inc, Shanghai, China, 15Pfizer Inc, Collegeville, PA, 16Pfizer SLU, Madrid, Spain, 17Pfizer Inc, Groton, CT, 18University of North Carolina at Chapel Hill, Chapel Hill, NC, 19University of Genoa, Genoa, Italy, 20Università Milano Bicocca, Milano, and IRCCS Fondazione San Gerardo dei Tintori, Monza, Italy

Meeting: ACR Convergence 2024

Date of first publication: October 24, 2024

Keywords: clinical trial, Disease Activity, Disease-Modifying Antirheumatic Drugs (Dmards), Juvenile idiopathic arthritis, Late-Breaking 2024, Still's disease

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Session Information

Date: Monday, November 18, 2024

Title: (L01–L14) Late-Breaking Posters

Session Type: Poster Session C

Session Time: 10:30AM-12:30PM

Background/Purpose: Tofacitinib (TOF) has been shown to be efficacious in the treatment of polyarticular course JIA, including systemic JIA (sJIA) without active systemic features. Here we report efficacy/safety/tolerability of TOF in participants (pts) with sJIA with active disease.

Methods: This was a Phase 3, global, 2-phase, randomized withdrawal study (NCT03000439), enrolling pts aged 2–< 18 years with active sJIA. In the open-label (OL) phase, pts were treated with TOF (5 mg twice daily or equivalent weight-based dose): in Part 1 (up to 16 wks), pts had to achieve and maintain an adapted (a) JIA-ACR30 response for ≥4 wks to proceed to OL Part 2 (up to 24 wks), in which pts treated with CS ˃0.2 mg/kg/day attempted to taper while maintaining clinical response. Responders (aJIA-ACR30, tapered CS) were eligible to enter the double-blind (DB) phase and were randomized to either continue TOF or switch to placebo (PBO). The primary endpoint was time to sJIA disease flare in the DB phase. Secondary efficacy endpoints and safety were assessed during the OL and DB phases. A prespecified interim analysis was performed after 28 pts reported an sJIA flare in the DB phase.

Results: Overall, 100 pts were enrolled in the OL phase from members of the PRINTO/PRCSG networks; 59 were randomized into the DB phase (28 TOF, 31 PBO). The prespecified futility stopping criterion was met and the study did not meet the primary objective of demonstrating statistically significant prolongation in time to sJIA disease flare in the DB phase (HR 0.633; 95% CI 0.296, 1.354; 1-sided p=0.1171) for TOF vs PBO (Table). However, fewer pts experienced flares with TOF (11/28; 39.3%) vs PBO (17/31; 54.8%). Median time to flare was 295 days with PBO and could not be calculated for TOF as < 50% of pts had a flare. During OL Part 1, aJIA-ACR30/70/90 responses were reached by 71/82 (86.6%), 28/82 (34.1%) and 15/82 (18.3%) of pts at wk 8 (Fig 1a). Clinically meaningful improvements in CHAQ-DI (Fig 1b) and JADAS-27 CRP (Fig 1c) were noted during OL Part 1. Responses were generally maintained during OL Part 2 despite CS taper (Fig 1); 38/54 (70.4%) pts achieved tapering criteria. Imbalances in disease activity were observed at DB randomization, including greater proportions of pts with JADAS-27 CRP inactive disease and aJIA-ACR100 responses randomized to PBO compared with TOF (Fig 2). The TOF group generally maintained the efficacy response for these outcomes during the DB phase (Fig 2). In the DB phase, frequencies of treatment‑emergent (TE) adverse events (AEs) were comparable between groups; no pts in the TOF group had severe/serious AEs. A higher percentage of pts had TEAEs leading to discontinuation in the PBO group vs the TOF group, driven by a greater number of sJIA flare TEAEs for PBO (14/31; 45.2%) vs TOF (8/28; 28.6%).

Conclusion: Improvements in sJIA disease activity were observed with OL TOF treatment after 7 days, with >80% of pts achieving an aJIA-ACR30 response by 8 wks. However, the primary efficacy objective in the DB withdrawal phase was not met. Important imbalances at randomization may have influenced the results and been driven by the overall small sample size. Safety findings were consistent with the known profile of TOF in adults and children.

Supporting image 1

Table 1

Supporting image 2

Figure 1

Supporting image 3

Figure 2


Disclosures: H. Brunner: AbbVie/Abbott, 2, Amgen, 2, AstraZeneca, 2, Boehringer-Ingelheim, 2, Bristol-Myers Squibb(BMS), 2, Eli Lilly, 1, 2, Genentech, 2, 5, Janssen, 1, 2, Novartis, 2, Pfizer, 2, 5, UCB, 2; C. Li: None; K. Chinniah: None; Y. Uziel: None; O. Synoverska: None; S. Sawhney: None; I. Calvo Penades: AbbVie, 5, 6, GSK, 2, 6, Lilly, 5, Novartis, 5, 6, Pfizer Inc, 5, 6, Sanofi, 5, Sobi, 6; I. Louw: Bristol Myers Squibb, 5, Idorsia, 5, Novartis, 5, Pfizer Inc, 5, Roche, 5, Sanofi, 5; M. Lu: None; P. Patel: AbbVie, 5, AstraZeneca, 5, Century Therapeutics, 7, CVS / Oak Street Health, 8, 11, Pfizer Inc, 5; P. Weiss: AbbVie, 5, Biogen, 1, Cerecor, 2, Lilly, 1, NIH, 5, Novartis, 1, Patient-Centered Outcomes Research Institute, 5, Pfizer inc, 2, 5, RheumNow, 6, Spondylitis Association of America, 5, Spondyloarthritis Research and Treatment Network, 6; C. Chang: Pfizer inc, 3, 11; I. Vranic: Pfizer Ltd, 3; S. Liu: Pfizer Inc, 3; A. Diehl: Pfizer Inc, 3, 11; J. Rivas: Pfizer SLU, 3, 11; C. Connell: Pfizer Inc, 3, 8; G. Koch: AbbVie, 5, Acadia Pharmaceuticals, 5, Aerovant Therapeutics, 5, Amgen, 5, AstraZeneca, 5, Bayer, 5, Cytokinetics, 5, Ensho Therapeutics, 5, Galderma Research Development, 5, Gilead, 5, GSK, 5, HUYA Bioscience International, 5, Intra-Cellular Therapies, 5, Ironwood Pharmaceuticals, 5, Johnson & Johnson, 5, Momentum, 5, Novan, 5, Otsuka, 5, Pfizer Inc, 5, Priovant Therapeutics, 5, Prometheus Biosciences, 5, Regeneron Pharmaceuticals, 5, Scholar Rock, 5, Summit Therapeutics, 5, Televant, Inc, 5, UCB, 5, vTv Therapeutics, 5, XyloCor Therapeutics, 5; A. Martini: Janssen, 2, Pfizer Inc, 2; D. Lovell: AstraZeneca, 2, Bristol Myers Squibb, 5, GSK, 2, Janssen, 5, Novartis, 2, Pfizer Inc, 2, Roche, 5, United Bioscience Corporation, 2; N. Ruperto: AbbVie, 2, 6, Aclaris, 2, 6, AlfaSigma, 2, 6, Amgen, 2, 6, AstraZeneca, 2, 6, Aurinia, 2, 6, Boehringer Ingelheim, 2, 6, Bristol Myers Squibb, 2, 6, Eli Lilly, 2, 6, Galapagos, 2, 6, Guidepoint, 2, 6, Idorsia, 2, 6, Janssen, 2, 6, Novartis, 2, 6, Pfizer Inc, 2, 6, Roche Genentech, 2, 6, Takeda, 2, 6.

To cite this abstract in AMA style:

Brunner H, Li C, Chinniah K, Uziel Y, Synoverska O, Sawhney S, Calvo Penades I, Louw I, Lu M, Patel P, Weiss P, Chang C, Vranic I, Liu S, Diehl A, Rivas J, Connell C, Koch G, Martini A, Lovell D, Ruperto N. Efficacy and Safety of Tofacitinib in Patients with Active Systemic Juvenile Idiopathic Arthritis [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/efficacy-and-safety-of-tofacitinib-in-patients-with-active-systemic-juvenile-idiopathic-arthritis/. Accessed .
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