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

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.

Reported incidence and exposure-adjusted incidence rates (EAIRs) of treatment-emergent adverse events (TEAEs) were determined.

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.

Ruxolitinib cream demonstrated tolerability, with no unexpected safety findings through 2 years in patients with vitiligo. Graphical Plain Language Summary available for this article.

Clinicaltrials.gov identifiers, NCT04052425 (registered on August 8, 2019), NCT04057573 (registered on August 14, 2019), and NCT04530344 (registered on August 25, 2020).