Is the treatment effect of Gilteritinib good?

This study aims to investigate the safety and efficacy of the combination of gilteritinib as a strategy to bridge to allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of FLT3-ITD mutation-positive relapsed and refractory acute myeloid leukemia (AML). At the same time, this study further evaluated the possible impact of gilitinib as maintenance therapy on the prognosis of patients after allo-HSCT.

The study included clinical data from 26 patients with relapsed and refractory AML with FLT3-ITD( ), and comprehensively evaluated the combined complete response rate (CRc), overall survival (OS), disease-free survival (DFS), and adverse events in these patients.

Of the 26 patients, the ratio was balanced between males and females (14 males and 12 females), with a median age of 38 years (range 18-65 years). Among them, 18 cases were refractory and 8 cases were relapsed. After 14~21 days of treatment, the complete response (CR) rate reached 26.9%, the CR rate of incomplete hematological recovery was 57.7%, the partial response (PR) rate was 7.7%, and the comprehensive complete response rate (CRc) was as high as 84.6%. In addition, the negative rate of minimal residual disease (MRD) also reached 65.4%.

In terms of survival analysis, the 12-month cumulative OS rate was 79.0% and the 24-month cumulative OS rate was 72.0% for all patients, and the median OS time was not reached. For patients who responded to treatment, the cumulative DFS rates at 12 and 24 months were 78.0% and 71.0%, respectively, and the median DFS time was also undetermined. Notably, patients who received allo-HSCT had a significantly longer median OS duration than those who did not (3.3 months, 95% CI: 2.2-4.3 months, P=0.005). In addition, the OS time of patients who received maintenance therapy with gilitinib after allo-HSCT was significantly longer than that of those who did not receive maintenance therapy (P=0.019).

In this study, it was also observed that the clearance rate of FLT3-ITD mutations was 38.5%, and the median OS time of patients with mutation clearance was significantly longer than that of those without clearance (15.0 months, P=0.018).

In terms of safety, the most common hematologic adverse events of grade 3 and above in the combination of geritinib included leukopenia (76.9%), neutropenia (76.9%), febrile neutropenia (61.5%), thrombocytopenia (69.2%) and anemia (57.7%). In addition, one patient developed differentiation syndrome during maintenance therapy with gilitinib after allo-HSCT, but his condition improved after treatment.

The combination of gilitinib has demonstrated significant efficacy and acceptable safety profile in the treatment of FLT3-ITD( ) relapsed and refractory AML. This protocol not only improved the negative rates of CRc and MRD, but also significantly improved the survival rate of patients. At the same time, the clearance rate of FLT3-ITD mutations is also high, which further enhances the clinical value of this treatment regimen. In addition, patients receiving maintenance therapy with gilitinib after allo-HSCT had a higher survival rate, suggesting that maintenance therapy may have a positive impact on patient outcomes. However, possible adverse events need to be closely monitored and managed during treatment to ensure patient safety.