Real-world effectiveness of mitoxantrone hydrochloride liposome containing regimens in acute myeloid leukemia

Abstract Background Mitoxantrone hydrochloride liposome (Lipo-MIT) exhibits encouraging efficacy in treating multiple hematologic malignancies. This study evaluated the real-world efficacy and safety of Lipo-MIT containing regimens in the treatment of acute myeloid leukemia (AML). Methods This non-interventional, ambispective cohort study was conducted at 42 centers in China. Patients with newly diagnosed or relapsed/refractory AML who received Lipo-MIT containing regimens were included. The primary endpoint was the composite complete remission (CRc) rate. Results Between January 2022 and December 2023, 117 patients with newly diagnosed AML and 122 patients with relapsed/refractory AML were included. The CRc and objective response rates were 84.6% (95% confidence interval CI, 76.8–90.6) and 91.5% (95% CI, 84.8–95.8), respectively, in the newly diagnosed AML, and 45.1% (95% CI, 36.1–54.3) and 55.7% (95% CI, 46.5–64.7), respectively, in relapsed/refractory AML. The 2-year-event-free survival and overall survival rates for newly diagnosed AML were 67.0% (95% CI, 54.4–76.8) and 77.8% (95% CI, 63.8–86.9), respectively, while the rates for relapsed/refractory AML were 23.9% (95% CI, 12.5-37.3) and 56.9% (95% CI, 39.6-70.9), respectively. Univariate and multivariate analyses suggested age and the number of prior failed induction therapy cycles as independent prognostic factors for CRc in relapsed/refractory AML (all p 0.05). Treatment-related adverse events (AEs) were reported in 216 patients (90.4%). Thrombocytopenia (64.4%), leukopenia (63.6%), and neutropenia (60.7%) were the most common AEs of grade 3–4. No treatment-related deaths occurred. Conclusions Lipo-MIT containing regimens were effective and well-tolerated in AML under real-world circumstances.