Institute of Cancer of São Paulo, University of São Paulo, São Paulo, Brazil.
Institute of Cancer of São Paulo, University of São Paulo, São Paulo, Brazil.
Clin Lymphoma Myeloma Leuk. 2019 Feb;19(2):e116-e122. doi: 10.1016/j.clml.2018.11.004. Epub 2018 Nov 12.
Although a considerable improvement in survival of patients with acute promyelocytic leukemia (APL) has been seen over the past decades, real-life outcomes seem to be worse than those reported by prospective studies. We aim to describe clinical characteristics and outcomes of adult patients diagnosed with APL in an academic hospital from the University of Sao Paulo.
We retrospectively reviewed the medical charts of 61 patients with APL diagnosed between January 2007 and May 2017. Baseline clinical features and follow-up data were collected, focusing on early toxicity variables such as infection, bleeding, and thrombosis in the first 30 days from diagnosis.
Among the 61 patients with APL, 54 received any chemotherapy. All patients also received all-trans retinoic acid (ATRA). Bleeding events were the main cause of death before receiving chemotherapy. Most patients belonged to the intermediate (43%) and high-risk (41%) groups, according to Sanz score. The '7 + 3 + ATRA' regimen was the most used regimen (n = 38). An early death rate of 20% was found, predominantly owing to sepsis. After a median follow-up of 5 years, only 1 relapse was diagnosed. The overall survival at 5 years was 59%.
In comparison with prospective trials with ATRA-based regimens, we found an inferior overall survival, mostly on account of a high early-death rate. Our results are in line with other real-life retrospective reports published in the past decades.
Results of real-life studies differ from those found by prospective trials. Accordingly, early actions and supportive care are still needed, aiming to decrease toxicity, especially in developing countries.
尽管过去几十年来，急性早幼粒细胞白血病（APL）患者的生存率有了显著提高，但实际结果似乎比前瞻性研究报告的要差。我们旨在描述在圣保罗大学附属医院确诊的 APL 成年患者的临床特征和结局。
我们回顾性分析了 2007 年 1 月至 2017 年 5 月期间诊断为 APL 的 61 例患者的病历。收集了基线临床特征和随访数据，重点关注诊断后 30 天内的早期毒性变量，如感染、出血和血栓形成。
在 61 例 APL 患者中，54 例接受了任何化疗。所有患者均接受全反式维甲酸（ATRA）治疗。在接受化疗之前，出血事件是导致死亡的主要原因。根据 Sanz 评分，大多数患者属于中危（43%）和高危（41%）组。最常用的方案是“7+3+ATRA”方案（n=38）。发现早期死亡率为 20%，主要是由于败血症。中位随访 5 年后，仅诊断出 1 例复发。5 年总生存率为 59%。
与基于 ATRA 的方案的前瞻性试验相比，我们发现总体生存率较低，主要是由于早期死亡率较高。我们的结果与过去几十年发表的其他真实世界回顾性报告一致。
真实世界研究的结果与前瞻性试验不同。因此，仍需要采取早期行动和支持性护理，以降低毒性，特别是在发展中国家。
