# The cure for acute promyelocytic leukemia and China's contributions

## TL;DR

Acute promyelocytic leukemia (APL) treatment has improved dramatically due to the discovery of ATRA and ATO, with China playing a key role in these breakthroughs.

## Contribution

China's pioneering work in developing ATRA and ATO-based therapies revolutionized APL treatment and survival rates.

## Key findings

- ATRA and ATO combination therapy increased APL 5-year survival rates from under 35% to over 90%.
- Chinese researchers identified the PML::RARA fusion gene and developed a synergistic treatment strategy for APL.
- Oral arsenic agents combined with ATRA offer a cost-effective treatment suitable for resource-limited areas.

## Abstract

急性早幼粒细胞白血病（APL）曾因早期高死亡率被称为“最凶险的白血病”，其治疗变革是肿瘤协同靶向治疗的里程碑。全反式维甲酸（ATRA）和三氧化二砷（ATO）的发现与应用，使APL的5年总生存率从不足35％提升至90％以上。中国血液肿瘤学界在此过程中作出重要贡献：王振义教授研究组开创ATRA分化疗法，张亭栋教授研究组验证ATO的临床价值。上海血液学研究所团队克隆了APL特异的15和17号染色体易位形成的PML::RARA融合基因，并发现了首个PLZF::RARA变异型融合基因，在阐明APL分子细胞发病机制和有效药物作用机制基础上，提出了ATRA和ATO协同靶向治疗初发APL的方案，在多中心临床试验中取得了95.7％的7年无复发生存率。之后，国内外数个团队又开发了费效比高的口服砷剂（复方黄黛片或口服ATO溶液）与ATRA的联合疗法，适于在资源有限地区推广。本文系统回顾APL治疗的关键突破，解析科学机制与临床意义，并展望未来机遇和挑战。