新诊断急性早幼粒细胞白血病早期死亡率的风险分层：一项多中心、非选择性、回顾性队列研究。

Risk stratification for early mortality in newly diagnosed acute promyelocytic leukemia: a multicenter, non-selected, retrospective cohort study.

作者信息

Kim Suhyeon, Jung Jiye, Ahn Seo-Yeon, Kim Mihee, Jeon So Yeon, Lee Chang-Hoon, Kim Dae Sik, Lee Se Ryeon, Sung Hwa Jung, Choi Chul Won, Kim Byung-Soo, Kim Hyeoung-Joon, Kwak Jae-Yong, Park Yong, Ahn Jae-Sook, Yhim Ho-Young

机构信息

Department of Internal Medicine, Jeonbuk National University Medical School, Research Institute of Clinical Medicine of Jeonbuk National University-Biomedical Research Institute of Jeonbuk National University Hospital, Jeonju, Republic of Korea.

Department of Internal Medicine, Chonnam National University Medical School, Chonnam National University Hwasun Hospital, Jeollanam-do, Republic of Korea.

出版信息

Front Oncol. 2024 Jan 30;14:1307315. doi: 10.3389/fonc.2024.1307315. eCollection 2024.

DOI:10.3389/fonc.2024.1307315

PMID:38352893

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10861669/

Abstract

INTRODUCTION

Despite the current effective treatments for acute promyelocytic leukemia (APL), early mortality (EM), defined as death within 30 days of presentation, is a major hurdle to long-term survival.

METHODS

We performed a multicenter retrospective study to evaluate the incidence and clinical characteristics of EM in patients with newly diagnosed APL and to develop a risk stratification model to predict EM.

RESULTS

We identified 313 eligible patients diagnosed between 2000 and 2021 from five academic hospitals. The median age was 50 years (range 19-94), and 250 (79.9%) patients were <65 years. Most patients (n=274, 87.5%) received their first dose of all-trans retinoic acid (ATRA) within 24 hours of presentation. EM occurred in 41 patients, with a cumulative incidence of 13.1%. The most common cause of EM was intracranial hemorrhage (n=22, 53.6%), and most EMs (31/41, 75.6%) occurred within the first seven days of APL presentation. In a multivariable analysis, we identified three independent factors predicting EM: age ≥65 years (HR, 2.56), white blood cell count ≥8.0 x 10/L (HR, 3.30), and ATRA administration >24 hours of presentation (HR, 2.95). Based on these factors, patients were stratified into three categories with a significantly increasing risk of EM: 4.1% for low risk (54.3%; no risk factors; HR 1), 18.5% for intermediate risk (34.5%; 1 factor; HR 4.81), and 40.5% for high risk (11.2%; 2-3 factors; HR 13.16).

DISCUSSION

The risk of EM is still not negligible in this era of ATRA-based therapies. Our risk model serves as a clinically useful tool to identify high-risk patients for EM who may be candidates for novel treatments and aggressive supportive strategies.

摘要

引言

尽管目前有针对急性早幼粒细胞白血病（APL）的有效治疗方法，但早期死亡率（EM），即就诊后30天内死亡，仍是长期生存的主要障碍。

方法

我们进行了一项多中心回顾性研究，以评估新诊断APL患者中EM的发生率和临床特征，并建立一个风险分层模型来预测EM。

结果

我们从五家学术医院中确定了2000年至2021年间诊断的313例符合条件的患者。中位年龄为50岁（范围19 - 94岁），250例（79.9%）患者年龄<65岁。大多数患者（n = 274，87.5%）在就诊后24小时内接受了第一剂全反式维甲酸（ATRA）。41例患者发生了EM，累积发生率为13.1%。EM最常见的原因是颅内出血（n = 22，53.6%），大多数EM（31/41，75.6%）发生在APL就诊后的前七天内。在多变量分析中，我们确定了三个预测EM的独立因素：年龄≥65岁（HR，2.56）、白细胞计数≥8.0×10⁹/L（HR，3.30）以及在就诊>24小时后给予ATRA（HR，2.95）。基于这些因素，患者被分为三类，EM风险显著增加：低风险为4.1%（54.3%；无风险因素；HR 1），中风险为18.5%（34.5%；1个因素；HR 4.81），高风险为40.5%（11.2%；2 - 3个因素；HR 13.16）。