Efficacy and Safety of Ruxolitinib Cream in Chinese Children Aged 2-11 Years With Non-segmental Vitiligo

This is a single-center, prospective, interventional, real-world study conducted in China. The study aims to evaluate the efficacy and safety of Ruxolitinib Phosphate Cream in the treatment of non-segmental vitiligo in children aged 2 to 11 years.

A total of eligible participants will receive topical Ruxolitinib Cream applied to vitiligo lesions as prescribed by the investigator. The treatment regimen and dosage follow the product labeling (if applicable) or standard clinical practice, with no additional randomization or blinding procedures.

The study duration is approximately 3 years, including enrollment, a 24-week treatment observation period, and follow-up. Clinical assessments are scheduled at baseline (Week 0) and at Weeks 4, 8, 12, and 24 post-treatment initiation.

Primary Outcome Measures:

• Proportion of participants achieving ≥75% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI75) at Week 24.

Secondary Outcome Measures:

• Proportion of participants achieving F-VASI50, F-VASI90, and Total Vitiligo Area Scoring Index (T-VASI50, T-VASI75) at Weeks 4, 8, 12, and 24.
• Change from baseline in quality of life assessed by the Children's Dermatology Life Quality Index (CDLQI) or appropriate pediatric dermatology quality of life scale.
• Proportion of participants with ≥50% improvement in T-VASI (T-VASI50) at Week 24.

Safety Outcome Measures:

• Incidence and severity of adverse events (AEs), serious adverse events (SAEs), and adverse drug reactions (ADRs) throughout the study period, with special attention to application site acne, application site pruritus, and other local skin reactions.
• Proportion of participants discontinuing treatment or withdrawing from the study due to AEs.

Eligibility Criteria (Key):

• Inclusion: Children aged 2-11 years, clinically diagnosed with non-segmental vitiligo, with depigmented BSA ≥0.1% and T-VASI ≥0.1, total body depigmentation area ≤10% BSA. Legal guardian provides signed informed consent.
• Exclusion: Diagnosis of segmental or other types of vitiligo; other depigmentation disorders; prior use of depigmenting treatments; history of JAK inhibitor failure; allergy to study drug; participation in another clinical trial; judged unsuitable by investigator.

Data will be collected using standardized Case Report Forms (CRFs) and clinical photography. All statistical analyses will be descriptive. This real-world study will provide critical evidence on the use of Ruxolitinib Phosphate Cream in young children with non-segmental vitiligo, filling the current evidence gap in this age group.

Inclusion criteria

• Children aged 2 to 11 years, regardless of sex.
• Clinical diagnosis of non-segmental vitiligo, with affected Body Surface Area (BSA) ≥0.1%, T-VASI score ≥0.1, and total depigmented area not exceeding 10% BSA.
• Legal guardian voluntarily provides signed informed consent and agrees to scheduled follow-ups.

Exclusion criteria

• Diagnosis of other forms of vitiligo (e.g., segmental vitiligo).
• Presence of other hypopigmentary or depigmentary disorders that could interfere with efficacy assessment (e.g., pityriasis alba, leprosy, post-inflammatory hypopigmentation, progressive macular hypomelanosis, nevus depigmentosus, and tinea versicolor).
• Prior or current use of any treatment for depigmentation.
• History of failure with any systemic or topical JAK inhibitor therapy for vitiligo or any other inflammatory condition.
• Known hypersensitivity to the study drug or its excipients.
• Concurrent participation in another clinical trial.
• Considered by the investigator to be unsuitable for the study for any other reason.