Department of Hematology, Aarhus University Hospital, Aarhus, Denmark.
Department of Hematology, Rigshospitalet, Copenhagen, Denmark.
Eur J Haematol. 2019 Jun;102(6):479-485. doi: 10.1111/ejh.13229. Epub 2019 Apr 7.
Acute promyelocytic leukemia (APL) is highly curable. To achieve high cure rates, targeted therapy with retinoic acid (ATRA) must be started promptly at time of suspected diagnosis. Early death rates (EDRs, ≤30 days from diagnosis) differ markedly in patients treated on clinical trials compared to the general population.
We used the comprehensive Danish National Acute Leukemia Registry (DNLR) to investigate the incidence, treatment, EDR, and long-term clinical outcome in APL between 2000 and 2014.
Twenty-two of 41 deaths occurring in 122 APL patients were EDs which were primarily caused by intracranial hemorrhage, disseminated intravascular coagulation (DIC), sepsis, and multiorgan failure. The overall EDR was 18.0%, whereas clinical trial participants had an EDR of 6.7%. Fifteen patients recruited to the NCRI AML17 APL trial from 2010 to 2013 were younger and had decreased mortality (HR 0.18, CI 0.04-0.86, P = 0.02) compared to contemporarily treated patients (n = 15) not recruited to a clinical trial. Performance status, leukemia origin, and Sanz-score were independent prognostic variables.
The very low EDR for on-trial patients is not observed in the general cohort of APL patients. Diagnostic awareness emerges as the greatest clinical challenge in management of APL.
急性早幼粒细胞白血病（APL）是高度可治愈的。为了达到高治愈率，必须在怀疑诊断时立即进行靶向治疗，使用维甲酸（ATRA）。与临床试验相比，接受治疗的患者的早期死亡率（EDR，从诊断起 30 天内死亡）在临床试验中差异显著。
我们使用综合丹麦国家急性白血病登记处（DNLR），调查 2000 年至 2014 年期间 APL 的发病率、治疗、EDR 和长期临床结局。
在 122 例 APL 患者中，41 例死亡中有 22 例为 EDR，主要由颅内出血、弥漫性血管内凝血（DIC）、败血症和多器官衰竭引起。总的 EDR 为 18.0%，而临床试验参与者的 EDR 为 6.7%。2010 年至 2013 年，NCRI AML17 APL 试验从 15 名患者中招募了 15 名患者，他们年龄较小，死亡率降低（HR 0.18，CI 0.04-0.86，P=0.02）与未招募至临床试验的同期治疗患者（n=15）相比。表现状态、白血病起源和 Sanz 评分是独立的预后变量。
试验组患者的 EDR 非常低，而在 APL 患者的一般队列中并未观察到。诊断意识是管理 APL 的最大临床挑战。
