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现实世界中的急性早幼粒细胞白血病:理解结局差异以及我们如何改善这些差异。

Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them.

作者信息

Bidikian Aram, Bewersdorf Jan Philipp, Kewan Tariq, Stahl Maximilian, Zeidan Amer M

机构信息

Department of Internal Medicine, Yale School of Medicine, Yale New Haven Hospital, New Haven, CT 06510, USA.

Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine, Yale Comprehensive Cancer Center, New Haven, CT 06510, USA.

出版信息

Cancers (Basel). 2024 Dec 6;16(23):4092. doi: 10.3390/cancers16234092.

Abstract

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.

摘要

全反式维甲酸(ATRA)和三氧化二砷(ATO)的出现彻底改变了急性早幼粒细胞白血病(APL)的治疗方式,带来了极高的缓解率和长期生存率。然而,由于与患者人口统计学和临床实践相关的各种因素,实际治疗效果往往不及临床试验中的观察结果。本综述考察了真实世界环境下的APL治疗效果,并突出了APL聚集现象。临床试验通常会排除老年患者和有严重合并症的个体,但这些人群在临床实践中占了相当大的比例。在真实世界环境中,早期死亡率仍然很高,诊断和治疗启动延迟、一些社区医疗服务提供者经验不足以及其所在中心管理APL及其相关并发症的资源有限,都使情况更加复杂。疾病和诱导相关并发症的高发生率进一步加剧了早期死亡率。社区医疗中心与专家机构之间持续的教育与合作至关重要,资源有限地区与专家中心之间的国际合作可以改善全球APL的治疗效果。持续监测可测量残留病(MRD)复发情况和长期治疗毒性,同时进行全面的患者评估和经验丰富的管理,可以提高长期治疗效果。虽然APL聚集性发病经常被报道,但人们对其仍知之甚少。定期报告这些聚集性发病情况可以为疾病病理学提供有价值的见解,并有助于开发APL发病率预测模型,从而指导未来的资源分配。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c4a/11640703/da54780e2750/cancers-16-04092-g001.jpg

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