The efficacy and clinical evaluation of Midostaurin in patients with acute leukemia

Midostaurin is a multi-target tyrosine kinase inhibitor that mainly targets FLT3, KIT, PDGFR and other signaling pathways have a significant effect on acute myeloid leukemia (AML), especially patients with FLT3 mutations. FLT3 mutations account for approximately 25%–30% of AML patients and generally indicate more aggressive disease, higher risk of recurrence, and lower overall survival. Midostaurin inhibits leukemia cell proliferation and induces apoptosis by inhibiting abnormally activated tyrosine kinase signaling, providing a new treatment option for high-risk AML patients, especially showing good promise in first-line chemotherapy combination therapy.

From a clinical efficacy perspective, multiple large-scale randomized controlled trials have shown that midostaurin combined with standard chemotherapy regimens (cytarabine and daiomycin) can significantly improve FLT3 mutations AML Patients’ overall survival (OS) and progression-free survival (EFS). In the RATIFY study, the median overall survival of patients in the midostaurin combined with chemotherapy group was significantly higher than that of the placebo group, and the recurrence rate was reduced. This shows that targeted therapy targeting specific molecular markers can effectively change the prognosis of high-risk AML, especially in patients with ITD and TKD FLT3 FLT3 mutations.

In efficacy evaluation, midostaurin not only showed advantages in overall survival and recurrence rate, but also could be objectively monitored through hematological indicators and bone marrow assessment. Clinically, the efficacy is usually evaluated by regularly monitoring blood routine, bone marrow blast cell ratio and molecular mutation load. Effective patients usually have a gradual recovery of platelet, white blood cell and red blood cell levels within a few weeks after chemotherapy combined with midostaurin, a significant decrease in bone marrow blasts, and a significant reduction in FLT3 mutation load. Through this multi-dimensional monitoring method, doctors can promptly adjust the treatment plan according to the efficacy, optimize the dose and course of treatment, and ensure that patients get the best benefit.

In terms of safety, the overall tolerability of midostaurin is controllable, but it is still necessary to pay attention to drug-related adverse reactions, such as gastrointestinal symptoms (nausea, vomiting, diarrhea), rash, hematological toxicity (neutropenia, thrombocytopenia), abnormal liver function, etc. Most adverse reactions can be managed with supportive care or dose adjustment, and the incidence of serious events is relatively low. Especially in the long-term maintenance phase, doctors will combine the patient's blood picture, liver function and electrocardiogram monitoring to evaluate tolerance, and make individual adjustments to the maintenance dose if necessary.

Overall, midostaurin provides a clear survival benefit and disease control advantage for FLT3 mutated AML patients, and its clinical application in combination with chemotherapy has been recommended by guidelines as one of the first-line treatment standards. Through standardized efficacy evaluation and safety monitoring, midostaurin can not only prolong overall survival and reduce recurrence rate, but also improve patients' quality of life. In the future, with the advancement of research on more combination regimens and individualized strategies, midostaurin is expected to play a more important role in precision treatment of AML and bring more stable long-term efficacy and more controllable treatment safety to high-risk patients.

Reference materials:https://www.nlm.nih.gov/