Avapritinib versus Placebo in Indolent Systemic Mastocytosis.

Jason Gotlib, Mariana Castells, Hanneke Oude Elberink, Frank Siebenhaar, Karin Hartmann, Sigurd Broesby-Olsen, Tracy I George, Jens Panse, Iván Alvarez-Twose, Deepti H Radia, Tsewang Tashi, Cristina Bulai Livideanu, Vito Sabato, Mark Heaney, Paul Van Daele, Sonia Cerquozzi, Ingunn Dybedal, Andreas Reiter, Thanai Pongdee, Stéphane Barete, Celalettin Ustun, Lawrence Schwartz, Brant R Ward, Philippe Schafhausen, Peter Vadas, Prithviraj Bose, Daniel J DeAngelo, Lindsay Rein, Pankit Vachhani, Massimo Triggiani, Patrizia Bonadonna, Mark Rafferty, Nauman M Butt, Stephen T Oh, Friederike Wortmann, Johanna Ungerstedt, Mar Guilarte, Minakshi Taparia, Andrew T Kuykendall, Cecilia Arana Yi, Princess Ogbogu, Caroline Gaudy-Marqueste, Mattias Mattsson, William Shomali, Matthew P Giannetti, Ilda Bidollari, Hui-Min Lin, Erin Sulllivan, Brenton Mar, Robyn Scherber, Maria Roche, Cem Akin, Marcus Maurer

NEJM Evid 2023 June

Avapritinib in Indolent Systemic MastocytosisIn a randomized trial, patients with indolent systemic mastocytosis were treated with avapritinib or placebo along with supportive care. The trial primary end point was the change in mean total symptom scores at 24 weeks. Avapritinib-treated patients had a decrease in mean total symptom score of 15.6 points compared with 9.2 points in the placebo group.

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