Đơn trị liệu với Gilteritinib cho người bệnh bạch cầu cấp dòng tủy tái phát có đột biến FLT3: Báo cáo ca

Acute Myeloid Leukemia (AML) is a severe hematological malignancy with poor prognosis, especially in relapsed cases, necessitating advanced therapeutic strategies. The presence of Fms-like tyrosine kinase 3 (FLT3) mutations significantly worsens outcomes, being identified in a substantial portion of AML patients and contributing to uncontrolled cell proliferation. This underscores the critical need for targeted therapies that can effectively address this genetic aberration. Gilteritinib, a potent second-generation FLT3 inhibitor, has emerged as a promising agent for FLT3-mutated relapsed/refractory AML. This case report aims to highlight the application and outcomes of gilteritinib monotherapy in a patient with relapsed acute myeloid leukemia carrying an FLT3 mutation, providing valuable insights into its real-world efficacy and tolerability as a salvage or bridging treatment.

Medical professionals, oncologists, hematologists, clinical researchers, and pharmaceutical scientists specializing in acute myeloid leukemia and targeted therapies.

This case report details the successful application of gilteritinib monotherapy in a 24-year-old male patient diagnosed with high-risk relapsed acute myeloid leukemia (AML) characterized by a FLT3-TKD D389A mutation. The patient initially achieved complete remission with standard chemotherapy combined with midostaurin but subsequently experienced a relapse with severe granulocytopenia. Due to concerns about the significant side effects of intensive chemotherapy, the patient opted for single-agent gilteritinib. Following five cycles of gilteritinib treatment, he achieved a partial response, with bone marrow blast cells significantly reduced from 65% at relapse to 10%. Importantly, the patient demonstrated good tolerability to gilteritinib, experiencing no severe adverse events, and showing recovery of all three blood cell lines. The findings underscore the value of gilteritinib in managing relapsed acute myeloid leukemia with FLT3 mutations, particularly for patients who are not candidates for intensive chemotherapy or are wary of its toxicity. Although the response in this specific case was observed to be slow, gilteritinib effectively controlled the disease and stabilized hematological parameters, acting as a viable bridging therapy towards potentially curative treatments like allogeneic stem cell transplantation. This case provides crucial real-world evidence for the efficacy and safety profile of gilteritinib as a targeted approach in a challenging patient population, suggesting its critical role in improving outcomes for relapsed acute myeloid leukemia with FLT3 mutation.