Tofacitinib, an oral Janus kinase inhibitor: analysis of malignancies across the rheumatoid arthritis clinical development programme

Ann Rheum Dis. 2016 May;75(5):831-41. doi: 10.1136/annrheumdis-2014-205847. Epub 2015 Apr 22.

Abstract

Objectives: Tofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). To further assess the potential role of Janus kinase inhibition in the development of malignancies, we performed an integrated analysis of data from the tofacitinib RA clinical development programme.

Methods: Malignancy data (up to 10 April 2013) were pooled from six phase II, six Phase III and two long-term extension (LTE) studies involving tofacitinib. In the phase II and III studies, patients with moderate-to-severe RA were randomised to various tofacitinib doses as monotherapy or with background non-biological disease-modifying antirheumatic drugs (DMARDs), mainly methotrexate. The LTE studies (tofacitinib 5 or 10 mg twice daily) enrolled patients from qualifying prior phase I, II and III index studies.

Results: Of 5671 tofacitinib-treated patients, 107 developed malignancies (excluding non-melanoma skin cancer (NMSC)). The most common malignancy was lung cancer (n=24) followed by breast cancer (n=19), lymphoma (n=10) and gastric cancer (n=6). The rate of malignancies by 6-month intervals of tofacitinib exposure indicates rates remained stable over time. Standardised incidence ratios (comparison with Surveillance, Epidemiology and End Results) for all malignancies (excluding NMSC) and selected malignancies (lung, breast, lymphoma, NMSC) were within the expected range of patients with moderate-to-severe RA.

Conclusions: The overall rates and types of malignancies observed in the tofacitinib clinical programme remained stable over time with increasing tofacitinib exposure.

Keywords: DMARDs (synthetic); Inflammation; Rheumatoid Arthritis; Treatment.

Publication types

  • Meta-Analysis
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adolescent
  • Adult
  • Aged
  • Antirheumatic Agents / administration & dosage
  • Antirheumatic Agents / adverse effects*
  • Antirheumatic Agents / therapeutic use
  • Arthritis, Rheumatoid / drug therapy*
  • Clinical Trials, Phase II as Topic
  • Clinical Trials, Phase III as Topic
  • Dose-Response Relationship, Drug
  • Female
  • Humans
  • Incidence
  • Janus Kinase 3 / antagonists & inhibitors
  • Male
  • Middle Aged
  • Multicenter Studies as Topic
  • Neoplasms / chemically induced*
  • Neoplasms / epidemiology
  • Piperidines / administration & dosage
  • Piperidines / adverse effects*
  • Piperidines / therapeutic use
  • Protein Kinase Inhibitors / administration & dosage
  • Protein Kinase Inhibitors / adverse effects*
  • Protein Kinase Inhibitors / therapeutic use
  • Pyrimidines / administration & dosage
  • Pyrimidines / adverse effects*
  • Pyrimidines / therapeutic use
  • Pyrroles / administration & dosage
  • Pyrroles / adverse effects*
  • Pyrroles / therapeutic use
  • Randomized Controlled Trials as Topic
  • Young Adult

Substances

  • Antirheumatic Agents
  • Piperidines
  • Protein Kinase Inhibitors
  • Pyrimidines
  • Pyrroles
  • tofacitinib
  • Janus Kinase 3