Gilteritinib Treatment in Real-World Setting Among Patients With R/R AML

Results from a Multicohort Ambispective Trial

According to findings from a multicohort ambispective trial recently published in Leukemia, the efficacy of real-world gilteritinib treatment for patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) previously treated with intensive chemotherapy was consistent with results of the past ADMIRAL trial, making it a viable treatment option. 

Gilteritinib, a selective FLT3 inhibitor, has shown promising results in past clinical trials, such as the ADMIRAL trial. However, its real-world efficacy and safety were not yet studied. Pierre-Yves Dumas, MD, Service d’Hématologie Clinique et de Thérapie Cellulaire, Bordeaux, France, and coauthors aimed to assess the efficacy and safety outcomes of gilteritinib treatment for patients with R/R AML in a real-world setting, with endpoints being comparability to ADMIRAL study, composite complete remission, and overall survival. 

167 patients with R/R FLT3-mutated AML were enrolled in this study, including 140 patients who received gilteritinib as a single agent (cohort B) and 67 patients who had undergone previous intensive chemotherapy and midostaurin (cohort C). Patient characteristics were compared with those in the ADMIRAL study, and endpoints were assessed after treatment. 

The comparison of patient characteristics to those of the ADMIRAL study indicated that the main differences were Eastern Cooperative Oncology Group (ECOG) ≥ 2 (83.6% vs. 16.6%), FLT3-TKD mutation (21.0% vs. 8.5%), primary induction failure (15.0% vs. 40.0%) and line of treatment (beyond 2nd in 37.1% vs. 0.0%). 

Efficacy results indicated that the rates of composite complete remissions were similar between cohorts, being 25.4 in cohort B vs 27.5% in cohort C, respectively. The median overall survival in cohort B was 6.4 months and in cohort C was 7.8 months. 

In conclusion, results indicated that gilteritinib treatment in a real-world setting yielded effective results comparable to past study results for patients with R/R AML. As endpoints were met, Dr Dumas et al stated, “Although these patients were more heavily pretreated, these real-world data reproduce the results of ADMIRAL and provide new insights into the course of patients previously treated by intensive chemotherapy and midostaurin and beyond the 2nd line of treatment who can benefit from treatment in an outpatient setting.”

Source: 

Dumas, PY., Raffoux, E., Bérard, E. et al. Gilteritinib activity in refractory or relapsed FLT3-mutated acute myeloid leukemia patients previously treated by intensive chemotherapy and midostaurin: a study from the French AML Intergroup ALFA/FILO. Leukemia. 37, 91–101 (2023). https://doi.org/10.1038/s41375-022-01742-7