In the past 25 years, the first targeted therapy for leukemia has been approved in the United States

• by EVOMT
• 2017-06-05

　　On April 28, 2017, the FDA approved Rydapt (midostaurin) for use in combination chemotherapy for adult patients with newly diagnosed acute myeloid leukemia (AML) with FLT3 gene mutations. It also approved the Leukostrat CDx FLT3 mutation detection method for detecting the presence of FLT3 mutations in patients with acute myeloid leukemia

　　Acute myeloid leukemia (AML) originates from the bone marrow and is a rapidly progressing cancer that causes an increase in white blood cells in the bloodstream. According to the National Cancer Institute of the United States, approximately 19930 people were diagnosed with AML in 2016, with an estimated 10430 deaths from the disease

　　"Rydapt is the first targeted therapy for combined chemotherapy for acute myeloid leukemia," said the Executive Director of the Blood and Tumor Products Office of the FDA Drug Evaluation and Research Center "Detecting genetic mutations through diagnostic tests means that doctors can identify specific patients who can benefit from the treatment."

　　Rydapt is a kinase inhibitor that can block various enzymes that promote cell growth. If a FLT3 mutation is detected in a patient's blood and bone marrow samples through Leukostrat CDx FLT3, the patient may be able to receive Rydapt combined with chemotherapy

　　The safety and efficacy of Rydapt in treating patients with acute myeloid leukemia were determined in a randomized trial involving 717 patients with acute myeloid leukemia who had not previously received treatment. In the trial, patients receiving Rydapt in combination with chemotherapy had a longer survival period compared to patients receiving chemotherapy alone, although the specific median survival rate cannot be accurately estimated for the time being, The researchers calculated the patient's event-free survival time and analyzed data on patients who did not achieve complete remission, disease progression, and death within 60 days of starting treatment. It was found that the median event-free survival time in the Rydapt combined chemotherapy group was 8.2 months, while the median event-free survival time in patients receiving only chemotherapy was only 3 months

　　Common side effects of Rydapt in the treatment of acute myeloid leukemia include febrile neutropenia, nausea, mucositis, vomiting, headache, ecchymosis, musculoskeletal pain, epistaxis, hyperglycemia, and upper respiratory tract infections. Patients who are allergic to midostaurin and other components of Rydapt should not use Rydapt. As this drug may cause harm to the fetus and newborn, Therefore, pregnant and lactating women should not use Rydapt. Patients with signs of lung injury should discontinue the drug

　　Rydapt is also approved for use in the treatment of adult patients with several rare blood diseases, including invasive systemic mast cell hyperplasia, hematologic tumor related systemic mast cell hyperplasia, or mast cell leukemia. Common side effects of Rydapt use in these patients include nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, fatigue, upper respiratory tract infections, constipation, fever, headache, and shortness of breath