Abstract
Secondary acute myeloid leukemia (sAML) poorly responds to conventional treatments and allogeneic stem cell transplantation (HSCT). We evaluated toxicity and efficacy of CPX-351 in 71 elderly patients (median age 66 years) with sAML enrolled in the Italian Named (Compassionate) Use Program. Sixty days treatment-related mortality was 7% (5/71). The response rate at the end of treatment was: CR/CRi in 50/71 patients (70.4%), PR in 6/71 (8.5%), and NR in 10/71 (19.7%). After a median follow-up of 11 months relapse was observed in 10/50 patients (20%) and 12 months cumulative incidence of relapse (CIR) was 23.6%. Median duration of response was not reached. In competing risk analysis, CIR was reduced when HSCT was performed in first CR (12 months CIR of 5% and 37.4%, respectively, for patients receiving (=20) or not (=30) HSCT, p = 0.012). Twelve-months OS was 68.6% (median not reached). In landmark analysis, HSCT in CR1 was the only significant predictor of longer survival (12 months OS of 100 and 70.5%, for patients undergoing or not HSCT in CR1, respectively, p = 0.011). In conclusion, we extend to a real-life setting, the notion that CPX is an effective regimen for high risk AML patients and may improve the results of HSCT.
Highlights
Acute myeloid leukemia (AML) is a highly heterogeneous disease including approximately one fourth of cases secondary to previous hematological disorders or developing after chemotherapy or radiotherapy1–5. sAMLs and tAMLs are more frequent in older patients and their prognosis is often worsened by the presence of adverse/complex cytogenetics, high risk
As the outcome of sAML and tAML is unsatisfactory with conventional chemotherapy, and the probability of performing HSCT is low, the results produced by CPX351 represent a remarkable improvement in the treatment of these diseases[8,9,10,16,17,21,22,23]
Despite the prolonged hematological recovery after CPX-351 induction, related to the extended drug exposure[15,24,25], the incidence of mucositis, severe infectious complications and the mortality rate at 30 and 60 days were lower than those observed after conventional intensive chemotherapy[2,9], and comparable to what reported in previous CPX trials[16,17,18]
Summary
Guolo et al Blood Cancer Journal (2020)10:96 molecular aberrations, and impaired performance status[6,7,8] These unfavorable biologic and clinical features deeply affect the efficacy of conventional treatment that is, generally, able to induce less than 40% short-term complete remissions with poor tolerability[9,10]. HSCT feasibility and overall results are impaired by the low efficacy of available induction therapies, the high median age and comorbidity burden of the majority of the patients[8,11]. Targeted drugs such as gemtuzumab ozogamicin (GO) and midostaurin showed promising efficacy in de novo AML but data on elderly AML patients are lacking[12,13]. Efficacy and toxicity data were subsequently confirmed in a phase 4, multicenter, single-arm, open-label early access program conducted in the United States[18]
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