Session: 613. Acute Myeloid Leukemias: Clinical and Epidemiological: Poster II
Hematology Disease Topics & Pathways:
Combination therapy, Therapies
Methods: We enrolled patients with relapsed or newly diagnosed AML with poor prognosis with adverse molecular abnormalities, such as NUP98 rearrangements, FUS::ERG, CBFA2T3::GLIS2, and del7/7q, who did not achieve complete remission (CR) after first-line chemotherapy. Patients were administered the MVHO regimen, which included 1 dose of liposome mitoxantrone 8mg/m2; venetoclax, 300 to 350mg/m2 once daily, from day1 to day 7, (dose escalation for up to 3days from 50mg/m2 if high tumor burden occur); homoharringtonine 2mg/m2 once daily, from day1 to day 7; and Olverembatinib (HQP1351) 20-30mg/m2 every other day, for 4 doses from day 1 to day 7. Prophylactic oral levofloxacin and posaconazole were administered from day 8 through whole myelosuppression period of every cycles. One cycle (28-35 days, depending on hematopoietic recovery) response, total response, cumulative incidence of relapse (CIR), event-free survival (EFS), hematologic toxicity, and infection during this study were evaluated.
Results: A total of 18 patients were enrolled (9 boys and 9 girls), with a median (range) age of 7.3 (4 months-13 years) years, and underwent a total of 27 cycles of MVHO therapy. Half of the patients underwent 1 cycle and the other half, 2 cycles. Per French-American-British (FAB) classification criteria: patients had myelodysplastic syndrome (n = 2); mixed-phenotype acute leukemia (n = 2); and AML subtypes M7 (n = 4), M2 (n = 4), M5 (n = 4), M4 (n = 1), and M0 (n = 1). The median (range) follow-up time was 131 (26-256) days. After cycle one, the objective response rate (ORR, CR plus CR with incomplete hematological recovery [Cri] and partial response [PR]) was 94.4% (17/18), and the remission rate (CR + Cri) was 72.2% (13/18). The total ORR and remission rate before transplantation were 83.3% (15/18) and 66.7% (12/18), respectively. 8 patients among them reached minimum residual disease (MRD) negative remission. For 6 patients with relapsed AML, the ORR and one cycle remission rate was 100% and 66.7%, respectively. A total of 3 patients discontinued because of no response or disease progression, and 12 patients underwent hematopoietic stem cell transplantation (HSCT), after which 1 patient experienced relapse. Remaining 2 patients are being underwent further MVHO cycles. The CIR was 6.7% (1/15). The 8-month mean (± SD) EFS was 60.1% (±19%) and the OS, 100%. Among 26 cycles of MVHO treatment, there were no associated fatal infections or bleeding events. There were 21 episodes of breakthrough infection occurred in 26 cycles of MVHO. There was 1 case of septic shock, and the incidence of ≥grade 3 infection was 80.7%, which included pneumonia, bloodstream infection. Common grade 4 treatment-related adverse events were neutropenia, which was experienced by 100% of patients, and grade 4 thrombocytopenia, which was experienced by 46.1%. Most notably, platelets were stably normal in nearly 20% (5/26) MVHO cycles.
Conclusions: MVHO therapy was effective and reasonably well tolerated in pediatric patients with refractory or relapsed AML, suggesting that it may comprise a suitable first-line treatment option for pediatric AML patients.
Disclosures: No relevant conflicts of interest to declare.
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