@article{5cef006d42fc48b3b1f72ac56bc8a954,
title = "Practical management of adverse events in patients with advanced systemic mastocytosis receiving midostaurin",
abstract = "Introduction: Systemic mastocytosis (SM) is characterized by the overproduction and accumulation of neoplastic mast cells (MCs) in the bone marrow, skin, and visceral organs. The KIT D816V mutation is found in approximately 90% of cases. In advanced SM (advSM), inferior survival often relates to MC-induced organ damage that may impact multiple organ systems. In addition, mediator symptoms related to MC activation can severely impact the quality of life. The oral multikinase/KIT inhibitor midostaurin was approved by the US Food and Drug Administration and the European Medicines Agency as monotherapy for advSM based on data from phase 2 clinical studies.Areas covered: This review discusses the management of common adverse events (AEs) in patients with advSM who participated in phase 2 clinical studies that led to the approval of midostaurin.Expert opinion: In the advSM population undergoing treatment with midostaurin, treatment-related AEs are often difficult to distinguish from disease-related symptoms, which can lead to premature discontinuation and improper dose reduction of midostaurin therapy in patients who might have benefitted from continued therapy. Here we present strategies to help optimize AE management and maximize the potential benefits of midostaurin in advSM.",
keywords = "Advanced systemic mastocytosis, adverse events, cytopenia, KIT D816V mutation, mastocytosis, midostaurin, nausea, tryptase",
author = "Jason Gotlib and Kluin-Nelemans, {Hanneke C.} and Cem Akin and Karin Hartmann and Peter Valent and Andreas Reiter",
note = "Funding Information: This paper was funded by Novartis Pharmaceuticals Corporation. J Gotlib is supported by the Charles and Ann Johnson Foundation. P Valent was supported by the Austrian Science Fund (FWF) under Grant F4704-B20. Editorial assistance for this manuscript was provided by JoAnna Anderson, PhD, of ArticulateScience LLC. Funding Information: J Gotlib was the Chairman of the Study Steering Committee for the global trial of midostaurin and received funding to conduct trials of midostaurin. He also served on an advisory board for Novartis and received honoraria and reimbursement of travel expenses. J Gotlib has also served as Chair or Co-Chair of Study Steering Committees for avapritinib and ripretinib. HC Kluin-Nelemans was a non-paid member of the Study Steering Committee for the global trial of midostaurin and the Department of Hematology Groningen received funding to conduct a phase II trial of midostaurin for patients with ISM. C Akin provided consulting work and served on a scientific advisory board for a mastocytosis study with midostaurin for Novartis. He also provided consulting work and research support for Blueprint. K Hartmann was a member of the Study Steering Committee for the global Novartis trial of midostaurin. She also served on advisory boards for Novartis and received honoraria and reimbursement of travel expenses. P Valent was a member of the Study Steering Committee for the global Novartis trial of midostaurin and received honoraria and a research grant from Novartis. He also received personal fees from Accord, Astellas, Blueprint, Celgene, Daiichi Sankyo, Deciphera, Pfizer, and ThermoFisher. A Reiter was a member of the Study Steering Committee for the global trial of midostaurin and received funding to conduct trials of midostaurin. He also served on an advisory board for Novartis and received honoraria and reimbursement of travel expenses. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Publisher Copyright: {\textcopyright} 2021 Informa UK Limited, trading as Taylor & Francis Group.",
year = "2021",
doi = "10.1080/14712598.2021.1837109",
language = "English",
volume = "21",
pages = "487--498",
journal = "Expert Opinion on Biological Therapy",
issn = "1471-2598",
number = "4",
}