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Nat Med: Safety and efficacy of Avapritinib in patients with advanced systemic mastocytosis

 Last Update: 2021-12-24
 Source: Internet
 Author: User

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Systemic mastocytosis (SM) is a rare hematological tumor, and about 95% of the cases are related to KITD816V gene mutation
.

The increased proliferation and accumulation of tumor mast cells driven by mutations in the KITD816V gene lead to severe and often unpredictable symptoms and poor quality of life (QoL) in patients

Quality of life leukemia

Recently, the top medical journal Nature Medicine published a research article in which researchers reported on the pre-specified interim analysis results of an ongoing single-group phase 2 trial of Avapritinib (No.
NCT03580655)
.

Avapritinib is a potent and selective KIT D816V inhibitor.

The primary endpoint of the study is the overall response rate (ORR), and the secondary endpoints include patients with chronic systemic mastocytosis-symptom assessment table total symptom score and average baseline change in quality of life, response time, response duration, and progression-free survival Changes in life, overall survival, disease burden, and safety indicators
.

The study successfully reached the primary endpoint (P=1.
6×10-9).
Among the 32 patients with chronic systemic mastocytosis whose treatment response data can be evaluated, the ORR was 75% (95% confidence interval of 57-89%) ), these patients had sufficient follow-up for response assessment, and 19% of the patients had complete remission and had complete or partial hematological recovery
.

The researchers observed that serum tryptase (93%), bone marrow mast cells (88%), and KITD816V variant allele scores (60%) decreased by more than 50% from baseline

It can be seen that patients with chronic systemic mastocytosis have a high clinical, morphological and molecular response rate to Avapritinib treatment, and they are usually well tolerated in patients with chronic systemic mastocytosis
.

Patients with chronic systemic mastocytosis have a high clinical, morphological and molecular response rate to Avapritinib treatment, and are usually well tolerated in patients with chronic systemic mastocytosis
.

Original source:

Jason Gotlib,et al.

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