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Repigmentation in non-segmental vitiligo using the Janus kinase inhibitor upadacitinib, a retrospective case series
Jing Zhu1, Lingling Luo1, Youming Guo1
1Hospital for Skin Diseases, Institute of Dermatology, Chinese Academy of Medical Sciences & Peking Union Medical College, No.12 Jiangwangmiao, Xuanwu District, Nanjing, 210042, Jiangsu, China.
Abstract:
Vitiligo is the most frequently diagnosed depigmentation disease, affecting nearly 0.5-2% of individuals worldwide. This disorder is characterized by melanocyte loss, which results in skin and hair depigmentation. Psychological problems are common in patients, especially in those with the involvement of the face and hands. Several studies have recently focused on the use of JAK inhibitors for vitiligo treatment. However, studies on the selective JAK1 inhibitor upadacitinib for vitiligo treatment are limited. This study aimed to assess the efficacy and safety of upadacitinib in the treatment of vitiligo. This retrospective case series included five patients diagnosed with non-segmental vitiligo who were treated with upadacitinib for 4 months or longer. Disease severity was assessed using the Vitiligo Area Scoring Index (VASI). Five patients took 15 mg of upadacitinib orally for 4 consecutive months, and all achieved repigmentation. The plasma collected from the peripheral blood of the four patients showed that the CXCL9 level dropped after upadacitinib treatment. However, the CXCL10 level did not change significantly using enzyme-linked immunosorbent assay (ELISA). Flow cytometry revealed that the ratio of CD4 + CD3+/CD8 + CD3 + T cells in the blood samples tended to decrease through the treatment. And the Th1-like Tregs (CD4 + Foxp3 + IFN-γ + Tregs) were also downregulated in the peripheral blood. In conclusion, the JAK1 inhibitor upadacitinib was found to be effective and safe for treating non-segmental vitiligo.
Insights
Upadacitinib, a JAK1 inhibitor, effectively treated non-segmental vitiligo in five patients, showing significant repigmentation. This JAK inhibitor also demonstrated a favorable safety profile in the study.
Area of Science:
- Dermatology
- Immunology
- Pharmacology
Background:
- Vitiligo is a common depigmentation disorder affecting 0.5-2% globally, characterized by melanocyte loss.
- Psychological distress is prevalent in vitiligo patients, particularly with facial and hand involvement.
- Janus kinase (JAK) inhibitors are emerging as potential treatments for vitiligo, but research on selective JAK1 inhibitors is limited.
Purpose of the Study:
- To evaluate the efficacy and safety of upadacitinib, a selective JAK1 inhibitor, for treating non-segmental vitiligo.
- To assess the impact of upadacitinib on disease severity and relevant immune markers in vitiligo patients.
Main Methods:
- A retrospective case series involving five patients with non-segmental vitiligo treated with upadacitinib (15 mg daily) for at least 4 months.
- Disease severity was quantified using the Vitiligo Area and Severity Index (VASI).
- Peripheral blood samples were analyzed for CXCL9, CXCL10, T cell ratios (CD4+/CD8+), and Th1-like regulatory T cells (Tregs) using ELISA and flow cytometry.
Main Results:
- All five patients achieved significant skin repigmentation after 4 months of upadacitinib treatment.
- Upadacitinib treatment led to a decrease in plasma CXCL9 levels, while CXCL10 levels remained unchanged.
- A trend towards decreased CD4+/CD8+ T cell ratios and downregulation of Th1-like Tregs was observed during treatment.
Conclusions:
- The JAK1 inhibitor upadacitinib demonstrates significant efficacy and a good safety profile in the treatment of non-segmental vitiligo.
- Upadacitinib may modulate immune responses involving T cells and chemokine signaling pathways in vitiligo.
- Further research is warranted to elucidate the precise mechanisms of action and long-term outcomes of upadacitinib in vitiligo management.

