Featured Student: Zoya Siddiqui

Zoya Siddiqui is a fifth-year medical student in the B.A./M.D. program at the University of Missouri-Kansas City. She enjoys rollerblading, playing tennis, and exploring local coffee shops. Her research interests include inflammatory skin conditions and health disparities in dermatologic care. Writing this research summary has helped her develop a deeper appreciation for study design and its role in translating clinical evidence into patient-centered treatment decisions.

Introduction 

Vitiligo is an acquired disorder of skin pigmentation marked by progressive loss of melanocytes. It carries a significant psychosocial burden and has historically been difficult to treat, with few approved therapies. A major advance came with the JAK inhibitor ruxolitinib cream, which received FDA approval in 2022 for nonsegmental vitiligo in patients aged twelve years and older. A study published in the New England Journal of Medicine in late 2023 by Dr. David Rosmarin and colleagues expanded on this work by evaluating the longer-term efficacy and safety of ruxolitinib cream over a full year of treatment. The trial offers valuable insight into the durability of repigmentation and real-world therapeutic potential. 

Methodology 

This randomized, double blind trial enrolled 674 participants with nonsegmental vitiligo affecting at least 0.5% of facial surface area or 1% of total body surface area. Participants were assigned in a two-to-one ratio to receive ruxolitinib cream 1.5% percent twice daily or vehicle cream. At week twenty-four, responders continued active treatment, and the vehicle group was crossed over to receive ruxolitinib in an open-label extension period through week fifty-two. 

The primary endpoint was the Facial Vitiligo Area Scoring Index (FVASI-75) response at week twenty-four, defined as at least a 75% improvement from baseline. Secondary endpoints included total body repigmentation using the VASI, patient-reported outcome measures, and safety assessments such as application site reactions, acne, laboratory changes, and any systemic adverse events that might signal percutaneous absorption. 

Findings 

At week twenty four, 50% of patients receiving ruxolitinib cream achieved FVASI-75 compared with 8% in the vehicle group. Improvements continued with ongoing therapy. By week fifty-two, almost 60% of patients on continuous ruxolitinib reached FVASI-75, and many also achieved meaningful improvement in total body VASI scores. 

Adverse events were mild and most often included acne at the application site, mild erythema, or pruritus. Laboratory monitoring did not show systemic JAK inhibitor-related effects, and the safety profile remained stable through one year. 

Strengths and Weaknesses 

The study is strengthened by its large sample size, double-blinded design, and long duration of follow-up. Use of validated vitiligo scoring systems improves clarity and clinical relevance. The extension phase also provides valuable insight into the sustained benefit of treatment, which is essential for a chronic depigmenting disorder. 

Limitations include the absence of a direct comparison to other treatments, such as topical calcineurin inhibitors or phototherapy. The population consisted mainly of patients with mild to moderate disease, leaving questions about efficacy in those with extensive involvement. Additionally, repigmentation varied widely, reminding clinicians that response remains patient dependent. 

Applications to Future Practice 

This study reinforces the role of ruxolitinib cream as an effective and well-tolerated therapy for nonsegmental vitiligo. The continued improvement beyond six months suggests that patients may benefit from prolonged treatment, and the favorable safety profile supports its use as a first-line topical option. As interest grows in targeted therapies for pigmentary disorders, this trial provides important evidence that topical JAK inhibition can deliver meaningful repigmentation without systemic risk. Further studies will be useful to guide treatment sequencing, combination therapy with phototherapy, and management of more extensive disease.