Pharmacokinetics, Efficacy, and Safety Profiles of Mitoxantrone Hydrochloride Liposome Plus Cytarabine in Pediatric Patients With Newly Diagnosed Acute Myeloid Leukemia.
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BACKGROUND: Mitoxantrone hydrochloride liposome (Lipo-MIT), an innovative anthracycline nano-drug, has demonstrated a favorable pharmacokinetics (PK) profile in lymphoma and preliminary efficacy in adult acute myeloid leukemia (AML). This study investigated the PK profile, efficacy, and safety of Lipo-MIT plus cytarabine in newly diagnosed pediatric AML. METHODS: In this single-arm, open-label trial, eligible patients received Lipo-MIT and cytarabine every 4 weeks for two cycles. The primary endpoint was the PK characteristics of Lipo-MIT after the first dose. RESULTS: Between July 19, 2023, and November 14, 2023, eight pediatric patients were enrolled. The total and free mitoxantrone concentrations increased rapidly during the absorption phase and then declined gradually to minimal concentrations before the administration of the second dose. The mean elimination half-life of total mitoxantrone was 60.2 ± 9.4 h, and the ratio of the area under the plasma concentration-time curve from Time 0 to the time of the last quantifiable concentration (AUC0- t) of free mitoxantrone to total mitoxantrone was 1.8% ± 0.6%. The overall response rate and composite complete remission rate were both 85.7% (6/7). Measurable residual disease negativity was achieved in four (66.7%) patients after Cycle 1 and in six (100.0%) after Cycle 2. The 1-year relapse-free survival rate was 83.3%. The common Grade ≥3 treatment-related adverse events were hematological toxicities (100.0%) and soft tissue infection (28.6%). No treatment-related deaths occurred. CONCLUSION: Lipo-MIT plus cytarabine showed a favorable PK profile and a manageable safety profile in newly diagnosed pediatric AML, with early evidence of clinical activity in this population. CLINICAL TRIAL REGISTRATION: Chinese Clinical Trial Registry: ChiCTR2300067964 (https://www.chictr.org.cn/showproj.html?proj=188738).