Avapritinib improves cutaneous involvement in patients with indolent systemic mastocytosis: results from the randomized, phase 2, interventional PIONEER study
Background - Indolent systemic mastocytosis (ISM), a clonal mast cell disease driven by the KIT D816V mutation, can often cause debilitating dermatologic symptoms. - Objective - Assess improvement of ISM-related skin manifestations after treatment with avapritinib, a highly selective KIT D816V inhib...
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| Autori principali: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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| Natura: | Article (Journal) |
| Lingua: | inglese |
| Pubblicazione: |
June 2026
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| In: |
Journal of the American Academy of Dermatology
Year: 2026, Volume: 94, Fascicolo: 6, Pages: 1705-1713 |
| ISSN: | 1097-6787 |
| DOI: | 10.1016/j.jaad.2026.02.025 |
| Accesso online: | Verlag, kostenfrei, Volltext: https://doi.org/10.1016/j.jaad.2026.02.025 Verlag, kostenfrei, Volltext: https://www.sciencedirect.com/science/article/pii/S0190962226002215 
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| Note sull'autore: | Frank Siebenhaar, MD, Sigurd Broesby-Olsen, MD, Mariana Castells, MD, PhD, Tracy I. George, MD, Cristina Bulai Livideanu, MD, PhD,Iván Ávarez-Twose, MD, PhD, Jens Panse, MD, Stéphane Barete, MD, PhD, Andreas Reiter, MD, Ingunn Dybedal, MD, PhD, Cem Akin, MD, PhD, Paul Van Daele, MD, PhD, Deepti H. Radia, MD, Sonia Cerquozzi, MD, Celalettin Ustun, MD, Vito Sabato, MD, PhD, Jason Gotlib, MD, MS, Mark Rafferty, MBChB, BSC(Med Sci), MRCP(UK), Daniel J. DeAngelo, MD, PhD, Philippe Schafhausen, MD, Johanna Ungerstedt, MD, PhD, Princess U. Ogbogu, MD, Scott Florell, MD, David A. Wada, MD, Anton Rets, MD, PhD, Hui-Min Lin, PhD, Ilda Bidollari, MD, MBA, Janet Hong, PharmD, Daniel Shaheen, PharmD, Benjamin Lampson, MD, PhD, and Karin Hartmann, MD |
| Riassunto: | Background - Indolent systemic mastocytosis (ISM), a clonal mast cell disease driven by the KIT D816V mutation, can often cause debilitating dermatologic symptoms. - Objective - Assess improvement of ISM-related skin manifestations after treatment with avapritinib, a highly selective KIT D816V inhibitor, vs placebo in Part 2 of the PIONEER study (NCT03731260). - Methods - Patients with moderate to severe ISM received avapritinib 25 mg once daily (n = 141) or placebo (n = 71). Endpoints included skin lesion area and pigmentation at week 24, skin mast cell burden, and change in symptoms. - Results - Mean percent reduction in lesional surface area was −36.6% with avapritinib vs −1.8% with placebo in the most affected area; 86% vs 0% had improved skin lesion color. Mean percent change in skin mast cell burden decreased with avapritinib (−22.1%) vs placebo (10.1%). Avapritinib vs placebo significantly improved skin symptom domain score (mean change −7.2 vs −2.8; P < .0001), including the individual skin symptoms itching, flushing, and spots. Avapritinib was well tolerated. - Limitations - Photography was optional, so analysis population for lesion area and color were smaller (n = 111) than the overall study population (n = 212). - Conclusion - Avapritinib treatment improved dermatologic symptoms, decreased skin lesion size, normalized skin lesion color, and reduced skin mast cell burden in patients with ISM. |
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| Descrizione del documento: | Online verfügbar: 12. Februar 2026, Artikelversion: 19. Mai 2026 Gesehen am 02.07.2026 |
| Descrizione fisica: | Online Resource |
| ISSN: | 1097-6787 |
| DOI: | 10.1016/j.jaad.2026.02.025 |