OFFICIAL HIGHLIGHTS

American Diabetes Association

Conference summaries


Vitiligo

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.


References

References


  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


CLINICAL TRIALS

PSORIASIS

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

ATOPIC DERMATITIS

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

PRURIGO NODULARIS

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

VITILIGO

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 TERAPIES

PSORIASIS

Emerging therapies for psoriasis

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

ATOPIC DERMATITIS

New and emerging therapies in atopic dermatitis

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

ONYCHOMYCOSIS

Emerging treatments for onychomycosis

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

REVIEW & UPDATES

ACNE & ROSACEA

Isotretinoin for acne and rosacea

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

ALOPECIA AREATA

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.

DERMATOSURGERY

Update in dermatosurgery

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

EPIDERMOLYSIS BULLOSA

New start of gene therapy in epidermolysis bullosa

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

MELANOMA

Treatment resistance in metastatic melanoma

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

SCAR TREATMENT

Future of medical scar treatment

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

EDUCATION FORUM

NON-MELANOMA SKIN CANCER

Systemic treatments of non-melanoma skin cancer

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

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