Ruxolitinib cream improves repigmentation in patients with vitiligo

The application of ruxolitinib cream led to significant improvements in facial and total body repigmentation among adolescents and adults with vitiligo, according to two phase III studies presented at EADV 2021.

TRuE-V1* and TRuE-V2** cohorts consisted of 330 (mean age 15.9 years) and 344 (mean age 14.3 years), respectively, patients diagnosed with vitiligo who had mean baseline facial Vitiligo Area Scoring Index (F-VASI), total body (T-VASI), and facial body surface area (F-BSA) scores of 0.88─0.95, 6.47─6.89, and 0.96─1.09 percent, respectively. Participants were randomized in a 2:1 ratio to receive either ruxolitinib cream 1.5% (TRuE-V1: n=221, TRuE-V2: n=229) or vehicle (TRuE-V1: n=109, TRuE-V2: n=115) twice daily for 24 weeks. [EADV 2021, abstract 2931]

At week 24, a significantly higher percentage of patients treated with ruxolitinib achieved a ≥75 percent improvement in F-VASI score (F-VASI75) from baseline in both TRuE-V1 and TRuE-V2 studies (29.9 percent vs 7.5 percent; p<0.0001 and 12.9 percent; p<0.01, respectively) compared with vehicle treatment.

Significantly more patients on ruxolitinib also achieved F-VASI50 (51.5 percent vs 17.2 percent; p<0.0001 [for TRuE-V1] and 51.4 percent vs 23.4 percent; p<0.0001 [for TRuE-V2]) and F-VASI90 at week 24 from baseline (15.5 percent vs 2.2 percent; p<0.01 [for TRuE-V1] and 15.4 percent vs 1.9 percent; p<0.05 [for TRuE-V2]) than those on vehicle.

Moreover, significantly more ruxolitinib-treated recipients achieved T-VASI50, indicating a ≥50 percent improvement in T-VASI score, from baseline to week 24 in both TRuE-V1 and TRuE-V2 studies (20.6 percent vs 4.9 percent and 26.1 percent vs 11.3 percent, respectively; p<0.01 for both).

A significantly higher percentage of patients treated with ruxolitinib also achieved a Vitiligo Noticeability Scale (VNS) of 4 or 5 (defined as “a lot less noticeable” or “no longer noticeable”) compared with the vehicle cream at week 24 (24.5 percent vs 3.3 percent; p<0.001 [for TRuE-V1] and 21.9 percent vs 6.6 percent; p<0.01 [for TRuE-V2]).

Patients who received ruxolitinib also had an improved F-BSA than those on vehicle at week 24 (least squares mean [LSM] difference from baseline, -19.3 percent; p<0.0001 and -18.5 percent; p<0.05 in TRuE-V1 and TRuE-V2, respectively).

Application site acne and pruritus were the most commonly reported treatment-emergent adverse events (AEs) for the ruxolitinib groups in both TRuE-V1 (5.4 percent and 5.0 percent, respectively) and TRuE-V2 (4.4 percent for both).

Nevertheless, ruxolitinib cream was well tolerated, with no serious AEs.

“In conclusion, ruxolitinib cream monotherapy demonstrated clinically meaningful superiority to vehicle for the primary and all key secondary endpoints in the two phase III TRuE-V studies, … with substantial facial and total body repigmentation evident at 24 weeks, thus confirming the phase II findings,” said lead author Dr David Rosmarin from Tufts Medical Center in Boston, Massachusetts, US.

“As we know that repigmentation may take time, I am particularly looking forward to the 52-week result which will hopefully show [further improvements] … from the week 24 results,” he noted.

*TRuE-V1: Topical Ruxolitinib Evaluation in Vitiligo Study 1