THU0497 MAINTENANCE OF MINIMAL DISEASE ACTIVITY OR INACTIVE DISEASE STATUS AND PATIENT-REPORTED OUTCOMES IN INDIVIDUAL PAEDIATRIC PATIENTS WITH JUVENILE IDIOPATHIC ARTHRITIS TREATED WITH SUBCUTANEOUS ABATACEPT
Authors: H. Brunner, N. Tzaribachev, I. Louw, I. Calvo, F. Zapata, G. Horneff, I. Foeldvari, D. Kingsbury, M. Gastanaga, C. Wouters, J. Breedt, R. Wong, M. Nys, M. Askelson, J. Zhuo, A. Martini, D. J. Lovell, N. Ruperto
Journal: Annals of the Rheumatic Diseases (2020)

Background: Maintenance of clinical response over time has been shown in individual patients (pts) with polyarticular-course juvenile idiopathic arthritis (pJIA) treated with SC abatacept (ABA). 1 It is unknown whether each individual pt with sustained efficacy also consistently maintains the previously reported shorter-term benefits on patient-reported outcomes (PROs) 2,3 over time. Objectives: Investigate whether combined efficacy and stringent, optimal PRO responses to ABA treatment are maintained by individual pts with pJIA over time. Methods: In this analysis of the intent-to-treat population, pts in two age cohorts (2–5 and 6–17 yrs) who achieved clinical response to weekly SC ABA (10–&lt;25 kg [50 mg], 25–&lt;50 kg [87.5 mg], ≥50 kg [125 mg]) at Mo 4 (time point of primary pharmacokinetic endpoint 4 ) were followed for 2 yrs. Stringent efficacy outcomes (Juvenile Arthritis Disease Activity Score 27 [JADAS27] minimal disease activity [MDA; ≤3.8] and inactive disease [ID; ≤1] status) were combined with optimal PRO endpoints (childhood [C]HAQ-DI=0, Parental Global Assessment [PaGA] ≤1 and Pain visual analogue scale [VAS] &lt;35). Combined efficacy and PRO responses were analysed at Mos 4, 13 and 21. Results: 219 pts entered the study (46 [21.0%] 2–5 yrs; 173 [79.0%] 6–17 yrs); a subgroup of these pts achieved a clinical response at Mo 4 (Table 1). Many pts who achieved JADAS27 MDA or JADAS27 ID combined with optimal PROs at Mo 4 sustained their response at Mo 13, and at both Mo 13 and Mo 21 in the 2–5-yr and 6–17-yr cohorts (Table 1). Across the cohorts, 33–88% of pts maintained a combined JADAS27 MDA with optimal PRO responses through Mo 21. Where estimable, median times to combined efficacy and specific optimal PRO responses were consistent across the cohorts (Table 2; Figs 1, 2). Table 1. Proportion of pts with combined efficacy and optimal PRO responses at Mos 4, 13 and 21 Endpoint Responders at Mo 4 Responders at Mos 4 and 13* Responders at Mos 4, 13 and 21* 2–5 yrs (n=46 ) 6–17 yrs (n=173 ) 2–5 yrs 6–17 yrs 2–5 yrs 6–17 yrs JADAS27 MDA and CHAQ-DI=0 9 (20) 34 (20) 5/9 (56) 25/34 (74) 3/9 (33) 16/34 (47) JADAS27 MDA and PaGA ≤1 8 (17) 14 (8) 8/8 (100) 7/14 (50) 7/8 (88) 5/14 (36) JADAS27 MDA and Pain VAS &lt;35 mm 28 (61) 70 (41) 25/28 (89) 58/70 (83) 21/28 (75) 43/70 (61) JADAS27 ID and CHAQ-DI=0 7 (15) 20 (12) 2/7 (29) 13/20 (65) 1/7 (14) 9/20 (45) JADAS27 ID and PaGA ≤1 6 (13) 10 (6) 4/6 (67) 4/10 (40) 4/6 (67) 4/10 (40) JADAS27 ID and Pain VAS &lt;35 mm 17 (37) 31 (18) 10/17 (59) 22/31 (71) 8/17 (47) 17/31 (55) Data are n (%) or n/N (%). *% based on n of pts who achieved response at Mo 4 (denominator) Table 2. Kaplan–Meier estimates for median (95% CI) times (mos) to achieving combined efficacy and optimal PRO responses Endpoint 2–5 yrs 6–17 yrs JADAS27 MDA and CHAQ-DI=0 21.5 (6.8, NE) 21.5 (13.1, 24.4) JADAS27 MDA and PaGA ≤1 NE (15.9, NE) 24.6 (24.3, NE) JADAS27 MDA and Pain VAS &lt;35 mm 2.8 (1.9, 2.9) 3.8 (3.7, 6.6) JADAS27 ID and CHAQ-DI=0 NE (18.4, NE) 24.4 (18.7, NE) JADAS27 ID and PaGA ≤1 NE (21.3, NE) 24.6 (24.3, NE) JADAS27 ID and Pain VAS &lt;35 mm 3.8 (3.8, 10.3) 13.2 (10.3, 15.9) NE=not estimable Conclusion: Many individuals with pJIA who achieved stringent efficacy and PRO measures with weekly SC abatacept by Mo 4 sustained them over 2 years. Time to achieve combined efficacy and Pain VAS &lt;35 response was shorter than that for PaGA ≤1 and CHAQ-DI=0. References: [1]Ruperto N, et al. Ann Rheum Dis 2019;78:99–100 (abstr OP0056) [2]Brunner H, et al. Arthritis Rheumatol 2019;71(suppl 10):abstr 2707 [3]Ruperto N, et al. Ann Rheum Dis 2017;76:75 (abstr OP0058) [4]Brunner HI, et al. Arthritis Rheumatol 2018;70:1144–54 Acknowledgments: Katerina Kumpan, PhD, Caudex; funding: Bristol-Myers Squibb Disclosure of Interests: : Hermine Brunner Consultant of: Hoffman-La Roche, Novartis, Pfizer, Sanofi Aventis, Merck Serono, AbbVie, Amgen, Alter, AstraZeneca, Baxalta Biosimilars, Biogen Idec, Boehringer, Bristol-Myers Squibb, Celgene, EMD Serono, Janssen, MedImmune, Novartis, Pfizer, and UCB Biosciences, Speakers bureau: GSK, Roche, and Novartis, Nikolay Tzaribachev: None declared, Ingrid Louw Consultant of: Amgen, Novartis, Pfizer, Roche (advisory boards), Inmaculada Calvo Grant/research support from: Bristol-Myers Squibb, Clementia, GlaxoSmithKline, Hoffman-La Roche, Merck Sharpe &amp; Dohme, Novartis, Pfizer, Sanofi, Speakers bureau: AbbVie, GlaxoSmithKline, Hoffman-La Roche, Novartis, Francisco Zapata: None declared, Gerd Horneff Grant/research support from: AbbVie, Chugai, Merck Sharp &amp; Dohme, Novartis, Pfizer, Roche, Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp &amp; Dohme, Novartis, Pfizer, Roche, Ivan Foeldvari Consultant of: AbbVie, Amgen, Bristol-Myers Squibb, Chugai, Eli Lilly, Novartis, Pfizer, Daniel Kingsbury: None declared, Maria Gastanaga Grant/research support from: Amgen, Bristol-Myers Squibb, GlaxoSmithKline, Novartis, Pfizer, Roche, Speakers bureau: AbbVie, Roche, Carine Wouters Grant/research support from: GlaxoSmithKline, Pfizer, Roche, Johannes Breedt: None declared, Robert Wong Shareholder of: Bristol-Myers Squibb, Employee of: Bristol-Myers Squibb, Marleen Nys Shareholder of: Bristol-Myers Squibb, Employee of: Bristol-Myers Squibb, Margarita Askelson Consultant of: Bristol-Myers Squibb, Joe Zhuo Shareholder of: Bristol-Myers Squibb, Employee of: Bristol-Myers Squibb, Alberto Martini Consultant of: AbbVie, Eli Lily, EMD Serono, Janssen, Novartis, Pfizer, UCB, Daniel J Lovell Consultant of: Abbott (consulting and PI), AbbVie (PI), Amgen (consultant and DSMC Chairperson), AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb (PI), Celgene, Forest Research (DSMB Chairman), GlaxoSmithKline, Hoffman-La Roche, Janssen (co-PI), Novartis (consultant and PI), Pfizer (consultant and PI), Roche (PI), Takeda, UBC (consultant and PI), Wyeth, Employee of: Cincinnati Children’s Hospital Medical Center, Speakers bureau: Wyeth, Nicolino Ruperto Grant/research support from: Bristol-Myers Squibb, Eli Lily, F Hoffmann-La Roche, GlaxoSmithKline, Janssen, Novartis, Pfizer, Sobi (paid to institution), Consultant of: Ablynx, AbbVie, AstraZeneca-Medimmune, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lily, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, Sanofi, Servier, Sinergie, Sobi, Takeda, Speakers bureau: Ablynx, AbbVie, AstraZeneca-Medimmune, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lily, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, Sanofi, Servier, Sinergie, Sobi, Takeda