The advent of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) has revolutionized the treatment of acute promyelocytic leukemia (APL), resulting in excellent rates of remission and long-term survival. However, real-world outcomes often fall short of those observed in clinical trials due to various factors related to patient demographics and clinical practices. This review examines APL treatment outcomes in real-world settings and highlights the phenomenon of APL clusters. Clinical trials frequently exclude older patients and individuals with significant comorbidities, yet these groups represent a substantial portion of patients in clinical practice. Early mortality remains high in real-world settings, compounded by delayed diagnosis and treatment initiation, as well as the inexperience of some community providers and limited resources of their centers in managing APL and its associated complications. High rates of disease and induction-related complications further exacerbate early mortality. Continuous education and collaboration between community healthcare centers and expert institutions are essential, and international partnerships between resource-limited settings and expert centers can improve global APL outcomes. Ongoing monitoring for measurable residual disease (MRD) recurrence and long-term treatment toxicity, coupled with comprehensive patient evaluations, and experienced management, can enhance long-term outcomes. The clustered incidence of APL, while frequently reported, remains poorly understood. Regular reporting of these clusters could provide valuable insights into disease pathology and aid in developing predictive models for APL incidence, which would guide future resource allocation.
全反式维甲酸（ATRA）与三氧化二砷（ATO）的应用彻底改变了急性早幼粒细胞白血病（APL）的治疗格局，显著提升了缓解率与长期生存率。然而，受患者人口学特征及临床实践差异等多种因素影响，真实世界中的治疗结局常逊于临床试验数据。本文综述真实世界环境下APL的治疗结果，并聚焦APL聚集现象。临床试验常排除高龄患者及存在严重合并症者，但这两类人群在临床实践中占比显著。真实世界中早期死亡率仍居高不下，其原因涉及诊断与治疗启动延迟、部分基层医疗机构对APL及其并发症处理经验不足、医疗资源有限等多重因素。高发的疾病相关及诱导治疗相关并发症进一步加剧了早期死亡风险。加强基层医疗机构与专科中心间的持续教育协作、推动资源有限地区与国际顶尖中心的合作，对提升全球APL治疗水平至关重要。通过持续监测可测量残留病（MRD）复发与长期治疗毒性，结合全面的患者评估与专业化管理，可进一步改善长期预后。尽管APL聚集发病现象屡见报道，其发生机制仍未明确。系统记录此类聚集事件将有助于深入解析疾病病理机制，构建APL发病预测模型，从而为未来医疗资源配置提供科学依据。
肿瘤(癌症)患者之家