AI Article Synopsis
- Avapritinib is a selective inhibitor of the KIT D816V mutation, approved for treating advanced systemic mastocytosis (AdvSM) by the FDA in 2021 and by the EMA in 2022.
- Clinical trials (EXPLORER and PATHFINDER) showed that avapritinib can achieve significant and long-lasting responses, including molecular remission of the mutation.
- Managing AdvSM is complicated due to its intricate mutational patterns, often occurring alongside other blood disorders.
Article Abstract
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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| http://dx.doi.org/10.1182/blood.2021014612 | DOI Listing |
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