Journal of the American Academy of Dermatology · 2026
Avapritinib improves cutaneous involvement in patients with indolent systemic mastocytosis: Results from the randomized, phase 2, interventional PIONEER study.
СтатьяРандомизированное исследованиеКлиническое исследование II фазыМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Siebenhaar Frank, Broesby-Olsen Sigurd, Castells Mariana, George Tracy I, Livideanu Cristina Bulai, Álvarez-Twose Iván, Panse Jens, Barete Stéphane, Reiter Andreas, Dybedal Ingunn, Akin Cem, Van Daele Paul, Radia Deepti H, Cerquozzi Sonia, Ustun Celalettin, Sabato Vito, Gotlib Jason, Rafferty Mark, DeAngelo Daniel J, Schafhausen Philippe, Ungerstedt Johanna, Ogbogu Princess U, Florell Scott, Wada David A, Rets Anton, Lin Hui-Min, Bidollari Ilda, Hong Janet, Shaheen Daniel, Lampson Benjamin, Hartmann Karin
Аннотация
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.
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.