Conference summaries


Efficacy and safety of a 52-week, randomized, double-blind trial of ruxolitinib cream for the treatment of vitiligo

Presented by: Dr. Amit G. Pandya University of Texas Southwestern Medical Center, Dallas, TX, USA
  • Ruxolitinib cream monotherapy appears to be a promising therapy for vitiligo lesions with continued improvement for up to 52 weeks.

Vitiligo is a chronic autoimmune disease that targets melanocytes, resulting in patches of skin depigmentation. [1] The pathogenesis of vitiligo is driven by signalling through JAK1/JAK23, and a cream formulation of ruxolitinib, a JAK1/JAK2 inhibitor, [2] is under investigation for the treatment of the condition. [3] Ruxolitinib cream provided significant repigmentation of facial vitiligo lesions after 24 weeks of double-blind, vehicle-controlled treatment (NCT03099304). [4]

  • This trial further investigates the therapeutic potential of ruxolitinib cream in patients with vitiligo after 52 weeks of double-blind treatment.

Type of study, patients, and inclusion criteria

  • Trial NCT03099304 was a randomised, double-blind, dose-ranging study of ruxolitinib phosphate cream in subjects with vitiligo.
  • Patients had to have a clinical diagnosis of vitiligo with depigmented areas including ≥0.5% of total Body Surface Area (BSA) on the face and ≥3% of total BSA on non-facial areas.
  • Patients in the previous groups (0.5% QD, 1.5% QD, 1.5% BID) of trial NCT03099304 continued treatment.
  • Subjects on vehicle and those in the 0.15% group were rerandomised if <25% improvement in facial Vitiligo Area Scoring Index (F-VASI) was seen at week 24.
  • Re-randomisation was to 0.5% QD, 1.5% QD, or 1.5% BID.
  • A total of 157 patients were studied.

Primary outcome measure

  • The primary endpoint was proportion of patients treated with ruxolitinib cream who achieved a ≥50% improvement from baseline in F-VASI (F-VASI50) at week 24 compared with patients treated with vehicle.
  • At week 24, F-VASI50 was achieved by a significantly greater proportion of patients receiving ruxolitinib cream (25.8%–50.0% across doses) vs vehicle (3.1%).
  • At week 52, the proportion of patients achieving an F-VASI50 response was highest in the 1.5% BID group (Figure).
  • At week 52, the proportions of patients achieving F-VASI75 and F-VASI90 responses were highest in the 1.5% BID group.
  • T-VASI50 at week 52 was achieved by patients in a dose-dependent manner.
  • Among patients who treated all depigmented skin (baseline total BSA ≤20%), T-VASI50 response was 45.0% with the 1.5% BID regimen at week 52.
  • The proportion of patients who attained Physician's Global Vitiligo Assessment (F-PhGVA) scores of clear or almost clear at week 24 increased by week 52.
  • Ruxolitinib cream was not associated with clinically significant application site reactions or serious treatment-related adverse events.
  • Most relevant TEAEs include acne and possible application site reactions.
  • Ruxolitinib cream monotherapy produced substantial facial and total body repigmentation of vitiligo lesions after week 24.
  • Continued improvement was seen through 52 weeks of treatment (highest responses with 1.5% BID), suggesting that ruxolitinib cream is an effective treatment option for patients with vitiligo.
  • A longer duration of therapy was associated with greater repigmentation, objectively assessed using the VASI.
    • Near-complete facial repigmentation as assessed by F-VASI75.
    • Substantial total body repigmentation as assessed by T-VASI50.
  • All doses of ruxolitinib cream were well tolerated, and no treatment-related serious AEs were reported.

Key messages/Clinical perspectives

  • Ruxolitinib cream monotherapy appears to be a promising therapy for vitiligo lesions with good long-term durability.



  1. Taïeb A, Picardo M. Clinical practice. Vitiligo. N Engl J Med. 2009;360(2):160-9.
  2. Rashighi M, Harris JE. Interfering with the IFN-γ/CXCL10 pathway to develop new targeted treatments for vitiligo. Ann Transl Med. 2015;3(21):343.
  3. Quintás-Cardama A, Vaddi K, Liu P, et al. Preclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasms. Blood. 2010;115(15):3109-17.
  4. Rosmarin D, et al. Efficacy and safety of ruxolitinib cream for the treatment of vitiligo: results of a 24-week, randomized, double-blind, dose-ranging, vehicle-controlled study. Presented at: World Congress of Dermatology 2019.

Presenter disclosure information: AG Pandya: Aclaris Therapeutics, Clarify Medical, Immune Tolerance Network, Incyte Corporation, Pfizer.

Medical writer: Patrick Moore, PhD

Reviewer: Martina Lambertini, MD

Local reviewers: Martina Lambertini, MD (Italian); Alain Brassard, MD (French); Jorge Moreno González, MD (Spanish); Swen Malte John, MD, PhD (German); Marcelo Arnone, MD (Portuguese)

Scientific Editor: Prof. Brigitte Dréno, MD



Efficacy and safety of ixekizumab in a phase 3, randomized, double-blind, placebo-controlled study in paediatric patients with moderate-to-severe plaque psoriasis

Presented by: Prof. Kim A. Papp, Department of Medicine, Division of Dermatology, University of Toronto, Toronto, Canada


Efficacy and safety of baricitinib in combination with topical corticosteroids in moderate to severe atopic dermatitis: results of a phase 3 randomized, double-blind, placebo-controlled 16-week trial (BREEZE-AD7)

Presented by: Prof. Kristian Reich, Translational Research in Inflammatory Skin Diseases, Institute for Health Services Research in Dermatology and Nursing, University Medical Center Hamburg-Eppendorf, and Skinflammation® Center, Hamburg, Germany


Phase 2b study of nemolizumab in patients with moderate to severe prurigo nodularis and associated severe pruritus

Presented by: Prof. Sonja Ständer, Department of Dermatology and Center for Chronic Pruritus (KCP), University Hospital Münster, Germany


Efficacy and safety of a 52-week, randomized, double-blind trial of ruxolitinib cream for the treatment of vitiligo

Presented by: Dr. Amit G. Pandya, University of Texas Southwestern Medical Center, Dallas, TX, USA



Emerging therapies for psoriasis

Presented by: Prof. Michel Gilliet, Department of Dermatology, Lausanne CHUV, Switzerland


New and emerging therapies in atopic dermatitis

Presented by: Prof. Dagmar Simon, Department of Dermatology, Inselspital, Bern University Hospital, Bern, Switzerland


Emerging treatments for onychomycosis

Presented by: Dr. Ditte Marie L. Saunte, Department of Dermatology, Institute for Clinical Medicine, Zealand University Hospital, Roskilde, Denmark



Isotretinoin for acne and rosacea

Presented by: Dr. Pedro Mendes-Bastos, Dermatology Centre, Hospital CUF Descobertas, Lisbon, Portugal


New drugs for alopecia areata

Presented by: Prof. Spyridon Gkalpakiotis, Department of Dermatovenereology, Third Faculty of Medicine and University Hospital of Kralovske Vinohrady, Prague, Czech Republic.


Update in dermatosurgery

Presented by: Prof. Eduardo Nagore, Department of Dermatology, Instituto Valenciano de Oncología, Valencia, Spain


New start of gene therapy in epidermolysis bullosa

Presented by: Prof. Leena K. Bruckner-Tuderman, University Medical Center, Albert-Ludwigs-University of Freiburg, Germany


Treatment resistance in metastatic melanoma

Presented by: Prof. Martin Röcken, Department of Dermatology, Eberhard-Karls-University Tübingen, Germany


Future of medical scar treatment

Presented by: Prof. Gabriella Fabbrocini, Department of Dermatology, University of Naples Federico II, Naples, Italy



Systemic treatments of non-melanoma skin cancer

Presented by: Prof. Henrik F. Lorentzen, Department of Dermatology, Aarhus University Hospital, Denmark


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