Long-Term Integrated Safety Summary of Ruxolitinib Cream in Phase 3 Clinical Trials of Patients with Vitiligo

INTRODUCTION: Ruxolitinib cream demonstrated superior repigmentation versus vehicle at week 24 with continued improvement through week 104 in phase 3 studies of patients (aged ≥ 12 years) with nonsegmental vitiligo. Here, we evaluated long-term safety of ruxolitinib cream in an integrated analysis of phase 3 vitiligo studies. METHODS: Reported incidence and exposure-adjusted incidence rates (EAIRs) of treatment-emergent adverse events (TEAEs) were determined. RESULTS: Over 104 weeks, 673 patients with vitiligo applied either ruxolitinib cream (n = 637; 867.9 person-years [PY]) or vehicle (n = 270; 131.1 PY). TEAEs (EAIR, patients/100 PY) with ruxolitinib cream versus vehicle occurred in 62.6% (46.0) versus 37.0% (76.3) of patients, most commonly nasopharyngitis (7.2% [5.3] vs 2.6% [5.3]) and application site acne (6.0% [4.4] vs 1.1% [2.3]). No serious treatment-related TEAEs were reported with ruxolitinib cream. EAIRs (patients/100 PY) were low for acne-related TEAEs (7.1), skin and subcutaneous tissue infections (4.0), cytopenias (2.4), and liver enzyme elevations (2.2). Malignancies, serious infections, and thromboembolic events were rare (0.7, 0.5, and 0.2 patients/100 PY, respectively), and none were considered related to treatment. No major adverse cardiovascular events or deaths occurred. CONCLUSION: Ruxolitinib cream demonstrated tolerability, with no unexpected safety findings through 2 years in patients with vitiligo. Graphical Plain Language Summary available for this article. TRIAL REGISTRATION: Clinicaltrials.gov identifiers, NCT04052425 (registered on August 8, 2019), NCT04057573 (registered on August 14, 2019), and NCT04530344 (registered on August 25, 2020).