Background Treatment options delivering sustained efficacy when given as monotherapy in RA are limited.1 Tofacitinib is an oral JAK inhibitor for the treatment of RA. Tofacitinib monotherapy demonstrated efficacy in adult patients (pts) with RA in two Phase (P) 3 index studies (ORAL Solo2 and ORAL Start3).
Objectives To present data through Month 84 for safety and Month 60 for efficacy for pts who stayed on tofacitinib monotherapy in long-term extension (LTE) studies.
Methods Data were pooled from two tofacitinib LTE studies (NCT00413699 [ongoing; database unlocked as of Mar 2015 data cut-off] and NCT00661661 [completed; Apr 2014]). Pts from P 1/2/3 tofacitinib index studies received tofacitinib 5 or 10 mg twice daily (BID) as monotherapy or with background conventional synthetic DMARDs in LTE. For tofacitinib and permitted concomitant RA medications, dose adjustments were allowed for inadequate efficacy or for safety reasons. In this analysis, pts were assigned to tofacitinib 5 or 10 mg BID groups based on average total daily dose in LTE (<15 mg/day or ≥15 mg/day, respectively). Monotherapy was defined as those pts who received tofacitinib without other DMARDs throughout the LTE. Baseline values were those of the index studies for pts enrolling in LTE within 14 days of index study; for all others, baseline was start of LTE. Safety was evaluated through Month 84 and efficacy through Month 60 due to limited sample size.
Results 1750 pts initiated LTE on tofacitinib monotherapy; 1552 (89%) stayed on monotherapy throughout LTE (495 on 5 mg BID; 1057 on 10 mg BID). For pts staying on monotherapy, mean (max) treatment duration was 1248 (2892) and 1011 (2377) days for 5 and 10 mg BID, respectively. Efficacy responses for tofacitinib monotherapy were stable through Month 60 (Table 1). In the 5 and 10 mg groups, 66% and 82% of pts, respectively, stayed on initial dose throughout LTE. The proportion of pts staying on steroids decreased from 56% to 40% and 41% to 35%, in the 5 and 10 mg groups respectively, from baseline to Month 60. Rates of discontinuation due to lack of efficacy and adverse events (AEs), and of serious infection and malignancy (excluding non-melanoma skin cancer) were low (Table 1).
Conclusions In this analysis of pts receiving tofacitinib monotherapy in the LTE studies, nearly 90% of pts stayed on monotherapy, most did not have tofacitinib dose adjustments or add a DMARD and efficacy was sustained over 60 months, with low rates of discontinuation due to lack of efficacy or AEs. Safety was consistent with what has been reported previously.
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Acknowledgement Previously presented (Fleischmann R et al. Arthritis Rheumatol. 2015; 67 (S10): Abstr 1639) and reproduced with permission from Arthritis Rheumatol. This study was funded by Pfizer Inc. Editorial support was provided by S Johnson, PhD, of Complete Medical Communications and funded by Pfizer Inc.
Disclosure of Interest R. Fleischmann Grant/research support from: Pfizer Inc, J. Wollenhaupt Consultant for: Pfizer Inc, Speakers bureau: Pfizer Inc, L. Wang Shareholder of: Pfizer Inc, Employee of: Pfizer Inc, A. Maniccia Shareholder of: Pfizer Inc, Employee of: Pfizer Inc, K. Kwok Shareholder of: Pfizer Inc, Employee of: Pfizer Inc, L. Takiya Shareholder of: Pfizer Inc, Employee of: Pfizer Inc, R. van Vollenhoven Grant/research support from: Pfizer Inc, Consultant for: Pfizer Inc