Effects of the oral Janus kinase inhibitor tofacitinib on patient-reported outcomes in patients with active rheumatoid arthritis: results of two Phase 2 randomised controlled trials

Clin Exp Rheumatol. 2016 May-Jun;34(3):430-42. Epub 2016 Apr 28.


Objectives: Tofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). Here we investigated the effects of tofacitinib on patient-reported outcomes (PRO) in patients with active RA.

Methods: Two, 6-month, double-blind, placebo-controlled Phase 2b studies were performed. The combination study evaluated patients with inadequate response to methotrexate who received tofacitinib 1-15 mg twice daily (BID), 20 mg once daily or placebo, on background methotrexate. In the monotherapy study, patients with inadequate response to disease-modifying anti-rheumatic drugs received tofacitinib 1-15 mg BID, adalimumab 40 mg once every other week or placebo. PROs measured were: Patient's Assessment of Arthritis Pain (PAAP), Patient's Assessment of Disease Activity, HAQ-DI, FACIT-F and SF-36.

Results: In the combination study (n=507), significant improvements (p<0.05) versus placebo were observed at Week 12 in PAAP (visual analogue scale) and HAQ-DI for all tofacitinib groups. In the monotherapy study (n=384), significant improvements in PAAP were observed at Week 12 for tofacitinib 5, 10 and 15 mg BID, and in HAQ-DI for tofacitinib 3, 5, 10 and 15 mg BID. Significant improvements versus placebo were seen at Week 2 in PAAP (both studies) and HAQ‑DI (monotherapy study) with tofacitinib, and were maintained throughout each study. In both studies, improvements in several domains of the SF-36 in the tofacitinib groups were observed at Weeks 12 and 24.

Conclusions: In patients with active RA, tofacitinib, either in combination with methotrexate or as monotherapy, demonstrated rapid and sustained improvement in pain, physical functioning and health-related quality of life.

Trial registration: ClinicalTrials.gov NCT00413660 NCT00550446.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antirheumatic Agents / administration & dosage
  • Antirheumatic Agents / adverse effects
  • Arthralgia* / drug therapy
  • Arthralgia* / physiopathology
  • Arthralgia* / psychology
  • Arthritis, Rheumatoid* / diagnosis
  • Arthritis, Rheumatoid* / drug therapy
  • Arthritis, Rheumatoid* / immunology
  • Arthritis, Rheumatoid* / physiopathology
  • Disease Resistance
  • Drug Administration Schedule
  • Drug Monitoring
  • Female
  • Humans
  • Janus Kinases / antagonists & inhibitors*
  • Male
  • Middle Aged
  • Pain Measurement / methods
  • Piperidines* / administration & dosage
  • Piperidines* / adverse effects
  • Pyrimidines* / administration & dosage
  • Pyrimidines* / adverse effects
  • Pyrroles* / administration & dosage
  • Pyrroles* / adverse effects
  • Quality of Life*
  • Treatment Outcome


  • Antirheumatic Agents
  • Piperidines
  • Pyrimidines
  • Pyrroles
  • tofacitinib
  • Janus Kinases

Associated data

  • ClinicalTrials.gov/NCT00413660
  • ClinicalTrials.gov/NCT00550446