Real-World Outcomes in Acute Promyelocytic Leukemia: A 13-Year Cohort from Qatar.

IF 1.9 3区 医学 Q3 ONCOLOGY
Oncology Pub Date : 2026-07-03 DOI:10.1159/000553398
Rola Ghasoub, Hesham Elsabah, Anas Hamad, Nabeel Mohammad Qasem, Hawraa Shwaylia, Feryal Ibrahim, Halima Elomri, Rajvir Singh, Shehab Fareed, Abdulrahman F Al-Mashdali, Anil Ellahie, Honar Cherif
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引用次数: 0

Abstract

Introduction: Acute promyelocytic leukemia (APL) is among the most curable acute leukemias, but real-world early mortality continues to undermine population-level cure. This study evaluated long-term outcomes and prognostic factors among adults with APL treated in a single tertiary center in Qatar.

Methods: We conducted a retrospective cohort study of consecutive adult patients diagnosed with APL between 2012 and 2024. Baseline demographic, clinical, and laboratory data, treatment regimens, and response outcomes were extracted from electronic medical records. Patients were stratified according to Sanz risk criteria. Overall survival (OS) was estimated using the Kaplan-Meier method, and prognostic factors were assessed using log-rank tests and Cox proportional hazards regression.

Results: A total of 87 patients were included (median age: 36 years; 47.1% high-risk groups by Sanz criteria). Early mortality within 30 days occurred in 26.4% (n = 23), predominantly due to intracranial hemorrhage (47.8% of early deaths). Among 71 patients who received treatment, the complete remission (CR) rate was 84.1%, with the highest CR observed in those treated with all-trans retinoic acid (ATRA)-arsenic trioxide (90.9%). Treatment-related complications occurred in 73.2% of patients, most commonly infections (66.2%) and differentiation syndrome (21.1%). Three-year OS was 93% in low-risk groups, 88% in intermediate-risk groups, and 56% in high-risk groups (log-rank p = 0.001). In multivariable analysis, ICU admission (hazard ratio [HR]: 5.42) and lack of treatment initiation (HR: 8.06) were independent predictors of mortality. Excluding patients presenting with intracranial hemorrhage, early mortality decreased to 14.7%, and 5-year OS improved to 87.6%.

Conclusion: Despite the effectiveness of contemporary APL therapies, early mortality, primarily driven by hemorrhagic complications, remains a significant challenge. Strategies to improve outcomes should focus on rapid diagnosis, immediate initiation of empiric ATRA, and aggressive correction of coagulopathy. Integration of APL-specific emergency care pathways may represent a key opportunity to reduce preventable deaths.

急性早幼粒细胞白血病的真实世界结局:来自卡塔尔的13年队列研究
背景:急性早幼粒细胞白血病(APL)是最可治愈的急性白血病之一,但现实世界的早期死亡率继续破坏人群水平的治愈。本研究评估了在卡塔尔单一三级中心治疗的APL成人患者的长期结果和预后因素。方法:我们对2012年至2024年间诊断为APL的连续成人进行了回顾性队列研究。从电子记录中提取人口统计、临床和实验室基线数据、治疗方案和反应。采用Kaplan-Meier法估计总生存期(OS),采用log-rank检验和Cox回归评估预后变量。该回顾性队列包括87名成年人。按照Sanz风险标准对患者进行分层:低危(WBC≤10×10⁹/L,血小板>40×10⁹/L)、中危(WBC≤10×10⁹/L,血小板≤40×10⁹/L)、高危(WBC >10×10⁹/L)。结果:87例患者(中位年龄36岁,47.1%为Sanz高危患者)中,26.4% (n=23)在诊断后30天内死亡,主要死于颅内出血(占早期死亡的47.8%)。71例患者完全缓解(CR)率为84.1%;ATRA-ATO的CR率最高(Lo-Coco, 90.9%)。治疗相关并发症发生率为73.2%;感染(66.2%)和辨证(21.1%)最为常见。3年总生存率(OS)为93%(低风险),88%(中风险)和56%(高风险;log-rank p=0.001)。ICU住院和未接受治疗是死亡的独立预测因素(多变量HR分别为5.42和8.06)。排除伴有脑出血的患者,早期死亡率为14.7%,5年总生存率为87.6%。结论:该研究强调了当代APL治疗的有效性,但也强调了出血并发症导致的持续早期死亡率。及时诊断和快速开始治疗对改善患者预后至关重要。在紧急通道中嵌入apl识别束,确保立即获得经验性ATRA,并及时纠正凝血功能是降低死亡率的最可行的优先事项。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Oncology
Oncology 医学-肿瘤学
CiteScore
6.00
自引率
2.90%
发文量
76
审稿时长
6-12 weeks
期刊介绍: Although laboratory and clinical cancer research need to be closely linked, observations at the basic level often remain removed from medical applications. This journal works to accelerate the translation of experimental results into the clinic, and back again into the laboratory for further investigation. The fundamental purpose of this effort is to advance clinically-relevant knowledge of cancer, and improve the outcome of prevention, diagnosis and treatment of malignant disease. The journal publishes significant clinical studies from cancer programs around the world, along with important translational laboratory findings, mini-reviews (invited and submitted) and in-depth discussions of evolving and controversial topics in the oncology arena. A unique feature of the journal is a new section which focuses on rapid peer-review and subsequent publication of short reports of phase 1 and phase 2 clinical cancer trials, with a goal of insuring that high-quality clinical cancer research quickly enters the public domain, regardless of the trial’s ultimate conclusions regarding efficacy or toxicity.
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