Avapritinib for advanced systemic mastocytosis: blood spotlight
Avapritinib, a highly selective inhibitor of KIT D816V, was approved by the Food and Drug Administration in 2021 for treatment of advanced systemic mastocytosis (AdvSM) and by the European Medicines Agency in 2022 for AdvSM after prior systemic therapy. The phase 1 EXPLORER and phase 2 PATHFINDER tr...
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| Main Authors: | , , |
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| Format: | Article (Journal) Editorial |
| Language: | English |
| Published: |
October 13, 2022
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| In: |
Blood
Year: 2022, Volume: 140, Issue: 15, Pages: 1667-1673 |
| ISSN: | 1528-0020 |
| DOI: | 10.1182/blood.2021014612 |
| Online Access: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1182/blood.2021014612 Verlag, lizenzpflichtig, Volltext: https://ashpublications-org.ezproxy.medma.uni-heidelberg.de/blood/article/140/15/1667/486003/Avapritinib-for-advanced-systemic-mastocytosis 
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| Author Notes: | Jason Gotlib, Andreas Reiter, and Daniel J. DeAngelo |
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| 520 | |a Avapritinib, a highly selective inhibitor of KIT D816V, was approved by the Food and Drug Administration in 2021 for treatment of advanced systemic mastocytosis (AdvSM) and by the European Medicines Agency in 2022 for AdvSM after prior systemic therapy. The phase 1 EXPLORER and phase 2 PATHFINDER trials demonstrated that avapritinib can elicit complete and durable clinical responses and molecular remission of KIT D816V. Key management challenges relate to the complex mutational landscape of AdvSM, often found with an associated hematologic neoplasm. | ||
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