Scalzulli Emilia, Costa Alessandro, Carmosino Ida, Musiu Paolo, Bisegna Maria Laura, De Propris Maria Stefania, Ielo Claudia, Diverio Daniela, Minotti Clara, Capria Saveria, Latagliata Roberto, Martelli Maurizio, Breccia Massimo
Hematology, Department of Translational and Precision Medicine, Az. Policlinico Umberto I- Sapienza University, Via Benevento 6, Rome, 00161, Italy.
Hematology Unit, Department of Medical Sciences and Public Health, Businco Hospital, University of Cagliari, Cagliari, Sardegna, Italy.
Ann Hematol. 2024 Dec;103(12):5377-5386. doi: 10.1007/s00277-024-06014-1. Epub 2024 Oct 15.
A comprehensive analysis of 220 patients diagnosed with APL between 1993 and 2022 is here reported. Overall, 214 patients (97.2%) received induction therapy. Complete response (CR) was achieved in 97.4%, 100%, 100%, and 27% of patients treated with AIDA protocol, AIDA + Ara-C, ATRA + ATO, and ATRA monotherapy, respectively. Molecular complete response (CR-) was achieved in 96.8% cases, and 142 patients proceeded to maintenance therapy. Overall, the 3-year and 5-year overall survival (OS) rates were 80.8% (95% CI, 78.1-83.5) and 79.1% (95% CI, 76.4-81.8), respectively. Considering only patients who completed induction and maintenance therapy, the 5-year OS rates were 82.1% (95% CI, 77.5-86.7) for the AIDA0493 cohort, 87.5% (95% CI, 84.4-91.1) for the AIDA2000 cohort, and 100% for the APL0406 cohort (p = 0.044). Additionally, the disease-free survival (DFS) rates were 65.7% (95% CI, 60.4-70.9), 70% (95% CI, 65.8-75.2), and 95.1% (95% CI, 91.7-98.5) (p = 0.016), respectively. Among low and intermediate-risk patients, age > 70 years (p = 0.027) and relapse (p < 0.001) were significantly associated with reduced outcomes. This study contributes to the advancement of our understanding of APL treatment, underscoring the ongoing need for research to enhance outcomes and explore new therapeutic approaches and prognostic factors.
本文报告了对1993年至2022年间诊断为急性早幼粒细胞白血病（APL）的220例患者的综合分析。总体而言，214例患者（97.2%）接受了诱导治疗。接受AIDA方案、AIDA+阿糖胞苷（Ara-C）、全反式维甲酸（ATRA）+三氧化二砷（ATO）和ATRA单药治疗的患者完全缓解（CR）率分别为97.4%、100%、100%和27%。96.8%的病例实现了分子学完全缓解（CR-），142例患者进入维持治疗阶段。总体而言，3年和5年总生存率（OS）分别为80.8%（95%置信区间[CI]，78.1-83.5）和79.1%（95%CI，76.4-81.8）。仅考虑完成诱导和维持治疗的患者，AIDA0493队列的5年OS率为82.1%（95%CI，77.5-86.7），AIDA2000队列的5年OS率为87.5%（95%CI，84.4-91.1），APL0406队列的5年OS率为100%（p=0.044）。此外，无病生存率（DFS）分别为65.7%（95%CI，60.4-70.9）、70%（95%CI，65.8-75.2）和95.1%（95%CI，91.7-98.5）（p=0.016）。在低风险和中风险患者中，年龄>70岁（p=0.027）和复发（p<0.001）与预后降低显著相关。本研究有助于推动我们对APL治疗的理解，强调持续开展研究以改善预后、探索新治疗方法和预后因素的必要性。
