An expert discusses how data from the pivotal phase 3 trials of topical ruxolitinib showed 30% of patients achieved 75% improvement in facial vitiligo at 24 weeks, with real-world experience confirming these results and demonstrating that longer treatment periods and combination with phototherapy can further enhance repigmentation outcomes
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The pivotal TruE-V1 and TruE-V2 phase 3 trials established topical ruxolitinib’s efficacy through identical, vehicle-controlled studies enrolling patients aged 12 and older with nonsegmental vitiligo. Participants received either ruxolitinib 1.5% cream or vehicle cream twice daily, with the primary end point measuring 75% or greater reduction in facial vitiligo severity (F-VASI 75) at week 24. Results demonstrated significantly higher response rates in the ruxolitinib group, with approximately 30% of patients achieving F-VASI 75 compared with vehicle controls. Secondary end points including total body VASI scores also showed significant improvements with active treatment.
Real-world clinical experience aligns closely with trial outcomes, confirming the 30% response rate at 6 months while highlighting the need for extended treatment duration in many patients. Data from post hoc analyses and longer-term studies demonstrate that patients showing limited 24-week responses often achieve 75% or greater improvement at 1 or 2 years with continued therapy. This finding emphasizes that vitiligo treatment represents “a marathon rather than a sprint,” requiring patience and persistence from both patients and practitioners to achieve optimal outcomes.
Clinical practice often necessitates combination approaches when monotherapy proves insufficient at 6 months. Adding phototherapy to topical ruxolitinib can accelerate repigmentation and enhance treatment responses, with evidence from phase 4 studies confirming superior outcomes when combining narrow-band UVB with topical ruxolitinib. Success metrics vary individually, depending on patient goals and affected body areas, with facial vitiligo typically showing highest and fastest response rates compared with more challenging areas such as hands. Patient education about the gradual repigmentation process—beginning with pigmented dots within white patches—proves crucial for treatment adherence and long-term success.
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