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Practical Management of Adverse Events in Patients With Advanced Systemic Mastocytosis Receiving Midostaurin.
Practical Management of Adverse Events in Patients With Advanced Systemic Mastocytosis Receiving Midostaurin. Expert opinion on biological therapy Gotlib, J., Kluin-Nelemans, H. C., Akin, C., Hartmann, K., Valent, P., Reiter, A. 2020Abstract
INTRODUCTION: Systemic mastocytosis (SM) is characterized by the over-production 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 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.
View details for DOI 10.1080/14712598.2021.1837109
View details for PubMedID 33063554