PMID- 33063554
OWN - NLM
STAT- MEDLINE
DCOM- 20211125
LR  - 20211125
IS  - 1744-7682 (Electronic)
IS  - 1471-2598 (Linking)
VI  - 21
IP  - 4
DP  - 2021 Apr
TI  - Practical management of adverse events in patients with advanced systemic 
      mastocytosis receiving midostaurin.
PG  - 487-498
LID - 10.1080/14712598.2021.1837109 [doi]
AB  - 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.
FAU - Gotlib, Jason
AU  - Gotlib J
AD  - Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, 
      USA.
FAU - Kluin-Nelemans, Hanneke C
AU  - Kluin-Nelemans HC
AUID- ORCID: 0000-0003-2617-9427
AD  - Department of Hematology, University Medical Center, University of Groningen, 
      Groningen, The Netherlands.
FAU - Akin, Cem
AU  - Akin C
AD  - Division of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, 
      MI, USA.
FAU - Hartmann, Karin
AU  - Hartmann K
AD  - Division of Allergy, Department of Dermatology, University of Basel, Basel, 
      Switzerland.
AD  - Department of Biomedicine, University of Basel, Basel, Switzerland.
FAU - Valent, Peter
AU  - Valent P
AD  - Department of Internal Medicine I, Division of Hematology and Hemostaseology, 
      Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of 
      Vienna, Vienna, Austria.
FAU - Reiter, Andreas
AU  - Reiter A
AD  - University Hospital Mannheim, Heidelberg University, Mannheim, Germany.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
DEP - 20210115
PL  - England
TA  - Expert Opin Biol Ther
JT  - Expert opinion on biological therapy
JID - 101125414
RN  - H88EPA0A3N (Staurosporine)
RN  - ID912S5VON (midostaurin)
SB  - IM
MH  - Humans
MH  - *Mastocytosis, Systemic/diagnosis/drug therapy
MH  - Mutation
MH  - Quality of Life
MH  - Staurosporine/adverse effects/analogs & derivatives
OTO - NOTNLM
OT  - Advanced systemic mastocytosis
OT  - KIT D816V mutation
OT  - adverse events
OT  - cytopenia
OT  - mastocytosis
OT  - midostaurin
OT  - nausea
OT  - tryptase
EDAT- 2020/10/17 06:00
MHDA- 2021/11/26 06:00
CRDT- 2020/10/16 08:37
PHST- 2020/10/17 06:00 [pubmed]
PHST- 2021/11/26 06:00 [medline]
PHST- 2020/10/16 08:37 [entrez]
AID - 10.1080/14712598.2021.1837109 [doi]
PST - ppublish
SO  - Expert Opin Biol Ther. 2021 Apr;21(4):487-498. doi: 
      10.1080/14712598.2021.1837109. Epub 2021 Jan 15.