Predictive factors for response and survival in elderly acute myeloid leukemia patients treated with hypomethylating age
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ORIGINAL ARTICLE
Predictive factors for response and survival in elderly acute myeloid leukemia patients treated with hypomethylating agents: a real-life experience Sara Pepe 1 & Emilia Scalzulli 1 & Gioia Colafigli 1 & Alessio Di Prima 1 & Daniela Diverio 1 & Marco Mancini 1 & Roberto Latagliata 1 & Maurizio Martelli 1 & Robin Foà 1 & Massimo Breccia 1 Received: 28 May 2020 / Accepted: 10 August 2020 # Springer-Verlag GmbH Germany, part of Springer Nature 2020
Abstract Predictive factors of response to hypomethylating agents (HMA) in elderly acute myeloid leukemia (AML) patients remain unclear in the real-life setting and no direct comparison between azacitidine (AZA) and decitabine (DEC) has been carried out. We retrospectively evaluated 110 AML patients treated with HMA (78 AZA, 32 DEC) as first-line therapy outside of clinical trials. Median age was 75 years (range 58–87). The median overall survival (OS) of the entire cohort was 8.0 months (95% CI 6.1–10), without significant differences among the subgroups: AZA 8.8 months vs DEC 6.3 months (p = 0.291). HMA treatment yielded an overall response rate (ORR) of 40% (AZA 37% vs DEC 47%, p = 0.237). A stable disease (SD) after 4 HMA cycles was not associated with a worse survival outcome compared with an early optimal response. Factors independently associated with a better OS were transfusion independence during treatment (p = 0.049), achievement of an optimal response to treatment (p < 0.001), and a baseline hemoglobin level ≥ 9.25 (p = 0.018). A bone marrow (BM) blast count ≥ 30% (p < 0.001) and a therapyrelated AML (p = 0.008) remain poor survival predictors. Of the available biologic features, an adverse risk category according to the ELN classification was significantly associated with a shorter survival over the intermediate risk category (p = 0.034). Disease progression remains the primary cause of death. Infectious complications were more severe (p = 0.036) and occurred earlier (p = 0.006) in the DEC group compared with that of the AZA group. In conclusion, clinical prognostic factors associated to response and survival have been identified without significant associations concerning overall outcomes between the two HMAs. Keywords Azacitidine . Decitabine . Hypomethylating agents . Unfit patients . Acute myeloid leukemia . Real-life
Introduction Acute myeloid leukemia (AML) is the most common form of acute leukemia in adults and is characterized by a clinically and biologically heterogeneous disease [1]. According to the most recent data from the NCI’s Surveillance, Epidemiology
Electronic supplementary material The online version of this article (https://doi.org/10.1007/s00277-020-04217-w) contains supplementary material, which is available to authorized users. * Massimo Breccia [email protected] 1
Hematology, Department of Translational and Precision Medicine, Azienda Ospedaliera Policlinico Umberto I, Sapienza University, Via Benevento 6, 00161 Rome, Italy
and End Results (SEER) program, the median age at diagnosis is 68 years. More than hal
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