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Midostaurin (Rydapt)

A first generation kinase inhibitor midostaurin (Rydapt) used in combination with chemotherapy, for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who have the FLT3 genetic mutation.

It is a type 1TKI.
It resembles ATP and binds to the ATP binding site.

It also inhibits FLT3.

The mutation is seen in 30% to 35% of patients with AML and is associated with a worse prognosis.

FLT3 drives the growth of early progenitor cells.

The FLT3 receptor is expressed in most blast cells, which are the malignant cells of acute myelogenous leukemia.

RATIFY trial compared standard 7+3 chemotherapy with midostaurin and standard chemotherapy

resulted at four years with 51% of patients were still alive compared with 44% who receive standard therapy alone.

Patients who receive midostaurin in combination with chemotherapy live longer than patients who received chemotherapy alone.

The median event-free survival rate in the midostaurin-chemotherapy group was 8.2 months vs 3 months for the chemotherapy-alone group.

The most common adverse events include: febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infection.

Patients who experience signs or symptoms of pulmonary toxicity should stop using the drug.

Also approved today for adults with systemic mastocytosis and systemic mastocytosis with associated hematologic neoplasm or mast cell leukemia.

Common side effects include nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, fatigue, upper respiratory tract infection, constipation, fever, headache, and shortness of breath.

Rarely associate with pulmonary toxicity.
 
The pills have an unusual characteristic with a distinctively bad smell.

The first targeted therapy to treat patients with AML, and by detecting the gene mutation with a diagnostic test means one can identify specific patients who may benefit from this treatment.

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