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Nat Med: The safety and efficacy of Avapritinib in the treatment of advanced systemic mastocytosis

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

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Systemic mastocytosis (SM) is a rare blood system tumor characterized by the accumulation of neoplastic mast cells in the bone marrow (BM), skin, and other organs
.

The release of mast cell mediators and SM-related organ damage (called C discovery) lead to severe, debilitating symptoms

Recently, a research article was published in the top medical journal Nature Medicine.
This phase 1 study (NCT02561988) in patients with advanced systemic mastocytosis was to evaluate Avapritinib (BLU-285), a selective KITD816V The efficacy of inhibitors
.

The primary endpoints of the study are the maximum tolerated dose, the recommended phase 2 dose, and the safety of Avapritinib treatment

Researchers evaluated 86 patients with a therapeutic dose of 30-400mg Avapritinib once a day, 69 of which were diagnosed with advanced systemic mastocytosis
.

This study has not yet reached the maximum tolerated dose.

The incidence of intracranial hemorrhage was 13%, but only 1% of patients did not have severe thrombocytopenia (platelets <50×109/L)
.

Among 53 patients with evaluable response, the overall response rate was 75%, and the complete response rate was 36%

It can be seen that Avapritinib treatment can produce a deep and long-lasting response in patients with advanced systemic mastocytosis, including the molecular remission of KITD816V in patients with advanced systemic mastocytosis, and the patients are resistant to the recommended phase 2 dose of 200 mg per day.
Receptivity is good
.

Avapritinib treatment can produce a deep and long-lasting response in patients with advanced systemic mastocytosis, including the molecular remission of KITD816V in patients with advanced systemic mastocytosis, and it is well tolerated by patients at the recommended phase 2 dose of 200 mg per day
.

Original source:

Daniel J.

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