Advancing Treatment for Vitiligo: 52-Week Ruxolitinib Cream Study | Insights from AAD 2023

Vitiligo, a chronic autoimmune disorder characterized by skin depigmentation, has long posed challenges for effective treatment. Recent progress, however, has been made in the form of a cream containing ruxolitinib, a Janus kinase (JAK) 1/JAK2 inhibitor. This formulation has shown promising safety and efficacy results over the course of a 52-week period, offering hope to patients with this condition.¹

The studies, TRuE-V1 (ClinicalTrials.gov Identifier: NCT04052425) and TRuE-V2 (ClinicalTrials.gov Identifier: NCT04057573),^2,3 enrolled 674 participants aged 12 years and older, who had nonsegmental vitiligo with depigmented areas covering no more than 10% of their total body surface area. Participants were radomized 2:1 to recieve either a twice-daily 1.5% ruxolitinib cream or a placebo (vehicle) cream over the course of 24 weeks. Following this initial phase, all participants were given the opportunity to use the 1.5% ruxolitinib cream up until the end of the 52-week study period. This approach allowed researchers to evaluate the safety and efficacy of the treatment over an extended duration.

Dr Amit G Pandya, a prominent figure in the field, highlighted that the phase III results from TRuE-V1 and TRuE-V2 were consistent with those of phase II studies. The Facial Vitiligo Area Scoring Index (F-VASI) 75, indicating a 75% colour return to vitiligo lesions on the face, was achieved by 50% of participants after one year of treatment. Similarly, for the body, the F-VASI 50 was attained by 50% of participants, signifying an average of 50% colour return across the whole body. Notably, patients generally experienced better results on areas like the face, upper arms and upper legs, while outcomes were less favourable on the hands, feet, ankle and wrists. These findings shed light on the potential effectiveness of ruxolitinib cream in treating vitiligo lesions.

Dr Pandya also emphasized the positive safety profile observed in the TRUE-V studies. He pointed out that approximately 6% of patients reported acne at the application site as a side effect. However, crucially, side effects commonly associated with the oral form of ruxolitinib, such as bone marrow toxicity and increased infections, were notably absent. This underscores the cream’s safety when applied to the skin.

The study assessed the occurrence of treatment-emergent adverse events (TEAEs), which were reported by 332 participants (52.1%). The most frequently reported TEAEs included COVID-19 (6.1%), acne at the application site (5.3%), and nasopharyngitis (4.9%). Notably, only 2.2% of participants experienced serious TEAEs, and these were not considered to be linked to the treatment. Of particular interest were the JAK-related TEAEs, which encompassed serious infections (0.5%), malignancies (0.6%), and thromboembolic events (0.2%). Examples of these included cases of appendiceal abscess, basal cell carcinoma, and transient ischaemic attack, among others. Haematopoietic TEAEs were also monitored and included conditions such as erythropenia (0.8%), leukopenia (0.8%), thrombocytopenia (0.2%) and thrombocytosis (0.3%). Importantly, no fatal TEAEs or major adverse cardiovascular events were observed.

The study delved into the systemic effects of JAK inhibition, which were limited in nature and occurred in less than 1% of participants who applied the ruxolitinib cream for the full 52 weeks. These findings underscore the overall positive safety profile of the treatment, providing encouraging insights into its long-term use.

In conclusion, results from TRuE-V1 and TRuE-V2 on the 52-week application of a ruxolitinib cream for vitiligo patients has yielded promising results in terms of safety and tolerability. With the majority of participants experiencing manageable and non-treatment-related adverse events, and with the occurrence of serious TEAEs being notably low, this treatment avenue holds potential for offering an effective and safe long-term option for individuals living with vitiligo. Further research and clinical studies are warranted to validate and expand upon these findings, but the insights gained from this study mark a step forward in addressing the challenges posed by this chronic autoimmune condition. Dr Amit G Pandya’s commentary further emphasizes the importance of these findings, highlighting both the encouraging efficacy and the excellent safety profile observed in the trials.

The full video interview with Dr Amit G Pandya can be viewed on touchIMMUNOLOGY here.

The abstract discussed in this interview is the following:

Treatment-Emergent Adverse Events of Interest for Janus Kinase Inhibitors: Pooled Analysis of the 52-Week TRuE-V Phase 3 Studies of Ruxolitinib Cream Treatment for Vitiligo

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References

1. Pandya AG, Ezzedine K, Rosmarin D et al. Treatment-Emergent Adverse Events of Interest for Janus Kinase Inhibitors: Pooled Analysis of the 52-Week TRuE-V Phase 3 Studies of Ruxolitinib Cream Treatment for Vitiligo. Presented at: ADD 2023, New Orleans, Louisiana, 17–21 March, 2023; Abstr. #43978.
2. ClinicalTrials.gov. Topical Ruxolitinib Evaluation in Vitiligo Study 1 (TRuE-V1). ClinicalTrials.gov Identifier: NCT04052425. Available at: https://classic.clinicaltrials.gov/ct2/show/NCT04052425 (accessed 21 August 2023).
3. ClinicalTrials.gov. Topical Ruxolitinib Evaluation in Vitiligo Study 2 (TRuE-V2). ClinicalTrials.gov Identifier: NCT04057573. Available at: https://classic.clinicaltrials.gov/ct2/show/NCT04057573 (accessed 21 August 2023).