MPN-524 Reductions in Indolent Systemic Mastocytosis Biomarker Burden With Avapritinib in the Registrational, Double-Blind, Placebo-Controlled PIONEER Trial

Indolent systemic mastocytosis (ISM) is a clonal mast-cell (MC) disease driven by the KIT D816V mutation. PIONEER (NCT03731260) is a phase II, randomized, double-blind, placebo-controlled study of avapritinib, a potent, highly selective inhibitor of D816V-mutant c-KIT protein in symptomatic ISM. Her...

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Published in:Clinical lymphoma, myeloma and leukemia myeloma and leukemia, 2023-09, Vol.23, p.S398-S399
Main Authors: Gotlib, Jason, Castells, Mariana, Elberink, Hanneke Oude, Siebenhaar, Frank, Hartmann, Karin, Broesby-Olsen, Sigurd, George, Tracy I., Panse, Jens, Alvarez-Twose, Iván, Radia, Deepti H., Tashi, Tsewang, Livideanu, Cristina Bulai, Sabato, Vito, Van Daele, Paul, Cerquozzi, Sonia, Dybedal, Ingunn, Reiter, Andreas, Ustun, Celalettin, Schafhausen, Philippe, Bose, Prithviraj, DeAngelo, Daniel J., Rein, Lindsay, Vachhani, Pankit, Triggiani, Massimo, Rafferty, Mark, Butt, Nauman M., Oh, Stephen T., Wortmann, Friederike, Ungerstedt, Johanna, Taparia, Minakshi, Kuykendall, Andrew T., Yi, Cecilia Arana, Mattsson, Mattias, Shomali, William, Giannetti, Matthew P., Bidollari, Ilda, Lin, Hui-Min, Scherber, Robyn, Roche, Maria, Akin, Cem, Maurer, Marcus
Format: Article
Language:English
Subjects:
avapritinib
C-KIT
KIT D816V
myeloproliferative neoplasm
systemic mastocytosis
tyrosine kinase inhibitor
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Summary:Indolent systemic mastocytosis (ISM) is a clonal mast-cell (MC) disease driven by the KIT D816V mutation. PIONEER (NCT03731260) is a phase II, randomized, double-blind, placebo-controlled study of avapritinib, a potent, highly selective inhibitor of D816V-mutant c-KIT protein in symptomatic ISM. Here we assess the impact of avapritinib vs placebo on objective biomarkers of MC burden in ISM. Adults were randomized 2:1 to once-daily avapritinib 25 mg or placebo, both with best supportive care and stratified by baseline serum tryptase (
ISSN:2152-2650
2152-2669
DOI:10.1016/S2152-2650(23)01250-8