Background/Purpose: Tofacitinib (CP-690,550) is a novel, oral Janus kinase (JAK) inhibitor investigated as a targeted immunomodulator for treatment of rheumatoid arthritis (RA). The efficacy and safety of tofacitinib vs placebo (PBO) were evaluated in pts with active RA with an inadequate response to ≥1 DMARD (traditional or biologic). Primary efficacy analyses have previously been reported. Here we report patient‑reported outcomes (PRO).
Methods: In this 12-month (Mo) study (NCT00856544), pts receiving traditional background DMARDs were randomized (4:4:1:1) to: tofacitinib 5 mg twice daily (BID); 10 mg BID; PBO advanced to 5 mg BID; and PBO advanced to 10 mg BID. At Mo 3 all ‘non-responder' PBO pts (<20% reduction from baseline in swollen/tender joint counts) were advanced to tofacitinib 5 or 10 mg BID. At Mo 6 all remaining PBO pts were advanced to tofacitinib. Most PROs were secondary endpoints and included: patient global assessment of disease activity (PtGA), pain, physical function (HAQ-DI); health-related quality of life (Medical Outcomes Survey [MOS] Short Form 36 [SF-36]), fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue [FACIT-F]), and sleep (MOS Sleep Scale).
Results: 792 pts were treated; mean disease duration 8.1 - 10.2 years (y); mean age 50.8 - 53.3 y. At Mo 3 improvements from baseline in all PROs were statistically significantly greater in tofacitinib 5 and 10 mg BID groups than PBO (Table). Based on pts reporting improvements ≥ minimally clinically important differences [MCID], numbers needed to treat (NNTs) at Mo 3 for tofacitinib 5 and 10 mg BID, respectively, ranged from 4.6 - 6.0 and 3.7 - 8.3, respectively (full analysis set [FAS], no imputation).
Conclusions: In this Phase 3 study of tofacitinib in combination with traditional DMARDs, treatment with 5 and 10 mg BID resulted in consistent statistically significant and clinically meaningful improvements in multiple PROs vs PBO at Mo 3.
Disclosure: V. Strand, Amgen, AstraZeneca, BMS, Genentech, GlaxoSmithKline, Human Genome Sciences, Idera, Lilly, MedImmune, Merck Serono, NovoNordisk, Ono, Pfizer, Rigel, Roche, Sanofi-Aventis, Schering Plough, UCB, 5 ; J. M. Kremer, Pfizer Inc, 2, Pfizer Inc, 5 ; Z. G. Li, Pfizer Inc, 5 ; S. Hall, Pfizer Inc, 5 ; R. M. Fleischmann, Pfizer Inc, 2, Pfizer Inc, 5 ; M. C. Genovese, Pfizer Inc., 2, Pfizer Inc., 5 ; E. Martin-Mola, Pfizer Inc, 9 ; J. Isaacs, Pfizer Inc., 5 ; D. Gruben, Pfizer Inc, 3 ; G. Wallenstein, Pfizer Inc, 3, Pfizer Inc, 1 ; S. Krishnaswami, Pfizer Inc, 3 ; S. H. Zwillich, Pfizer Inc, 3, Pfizer Inc, 1 ; T. Koncz, Pfizer Inc, 3 ; R. Riese, Pfizer Inc, 3 ; J. D. Bradley, Pfizer Inc, 3 .