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Two Phase 3, Randomized, Controlled
Trials of Ruxolitinib Cream for Vitiligo
David Rosmarin, M.D., Thierry Passeron, M.D., Ph.D., Amit G. Pandya, M.D
Published October 20, 2022
Introduction
• Vitiligo is a chronic autoimmune disease that results in skin
depigmentation and reduced quality of life.
• Skin lesions are characterized by white patches corresponding with a
loss of functioning melanocytes in the epidermis.
• Interferon-γ plays an important role in vitiligo pathogenesis and
signals through the Janus kinase (JAK) signal transducer and activator
of transcription (STAT) pathway, leading to up-regulation of C-X-C
motif chemokine ligand 10 (CXCL10) and promoting CD8+ T-cell
recruitment, which drives melanocyte destruction.
Study design
2019.09.20 - 2021.10.21 2019.10.03- 2021.10.01
Method
• The vitiligo area scoring index (VASI) is a composite measurement of
overall area of vitiligo patches and degree of depigmentation within
patches.
Result
Discussion
• In these two phase 3 trials, ruxolitinib cream showed superiority to
vehicle control in repigmentation of vitiligo. Patient-reported
outcomes suggest that changes were meaningful to patients,
although there were no substantial between-group differences in
quality of life.
• This is the first therapy to receive FDA approval for repigmentation in
patients with vitiligo in patients aged 12 and older.

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Two Phase 3, Randomized, Controlled Trials.pptx

  • 1. Two Phase 3, Randomized, Controlled Trials of Ruxolitinib Cream for Vitiligo David Rosmarin, M.D., Thierry Passeron, M.D., Ph.D., Amit G. Pandya, M.D Published October 20, 2022
  • 2. Introduction • Vitiligo is a chronic autoimmune disease that results in skin depigmentation and reduced quality of life. • Skin lesions are characterized by white patches corresponding with a loss of functioning melanocytes in the epidermis. • Interferon-γ plays an important role in vitiligo pathogenesis and signals through the Janus kinase (JAK) signal transducer and activator of transcription (STAT) pathway, leading to up-regulation of C-X-C motif chemokine ligand 10 (CXCL10) and promoting CD8+ T-cell recruitment, which drives melanocyte destruction.
  • 3. Study design 2019.09.20 - 2021.10.21 2019.10.03- 2021.10.01
  • 4.
  • 5.
  • 6.
  • 7. Method • The vitiligo area scoring index (VASI) is a composite measurement of overall area of vitiligo patches and degree of depigmentation within patches.
  • 9.
  • 10.
  • 11.
  • 12. Discussion • In these two phase 3 trials, ruxolitinib cream showed superiority to vehicle control in repigmentation of vitiligo. Patient-reported outcomes suggest that changes were meaningful to patients, although there were no substantial between-group differences in quality of life. • This is the first therapy to receive FDA approval for repigmentation in patients with vitiligo in patients aged 12 and older.

Editor's Notes

  1. Tufts Medical Center, Boston, National Reference Center for Rare Skin Disorders, Henri Mondor University Hospital and Université Paris-Est Créteil Val de Marne, Paris, the Vitiligo and Pigmentation Institute of Southern California, Los Angeles , Amsterdam University Medical Center
  2. OPZELURA-г (Ruxolitinib) давхар нууцлалтай, санамсаргүй түүврийн аргаар, 2 үе шаттай, эмнэлзүйн туршилт судалгаагаар насанд хүрсэн болон өсвөр насны нийт 674 өвчтөн хамрагдсан. Нэмэх хасах шалгуурт: 1. 12-с дээш насны 2. Нүүрний биеийн гадаргуугийн талбайн ≥0.5%, нүүрний бус биеийн гадаргуугийн талбай ≥3%, 3. Нийт биеийн гадаргуугын талбайн 10% хүртэл витилиго хамарсан 4. Фото эмчилгээнд судалгааны явцад хамрагдахгүй 5. Нүүрний аливаа гэмтэл дотор бүрэн лейкотрихи бүхий өвчтөнүүдийг хассан. OPZELURA буюу Ruxolitinib тосоор өдөрт 2 удаа 24 долоо хоногийн турш плацеботой хамт давхар нууцлалтайгаар, дараа нь 28 долоо хоногийн нээлттэй эмчилгээгээр, 2 бүлэгт дангаар Ruxolitinib-г өдөрт 2 удаа түрхсэн.
  3. Эдгээр хоёр үе шатны туршилтаар ruxolitinib тос нь витилиго өвчнийг арилгахад хяналтын бүлгээс давуу болохыг харуулсан. Өвчтөний мэдээлсэн үр дүнгээс харахад бүлгийн хооронд амьдралын чанарт мэдэгдэхүйц ялгаа байхгүй байсан ч өөрчлөлтүүд өвчтөнүүдэд ач холбогдолтой байсан. Энэ нь 12 ба түүнээс дээш насны витилиго өвчтэй өвчтөнүүдэд дахин пигментаци хийх зорилгоор FDA-аас зөвшөөрөл авсан анхны эмчилгээ юм.