Woods Ashley C, Norsworthy Kelly J
Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20903, USA.
Cancers (Basel). 2023 Sep 28;15(19):4767. doi: 10.3390/cancers15194767.
Differentiation syndrome (DS) is a frequent and potentially life-threatening clinical syndrome first recognized with the advent of targeted therapeutics for acute promyelocytic leukemia (APL). DS was subsequently observed more broadly with targeted therapeutics for acute myeloid leukemia (AML). DS is typically characterized by fever, dyspnea, hypotension, weight gain, pleural or pericardial effusions, and acute renal failure. The incidence in patients with APL ranges from 2 to 37%, with the wide variation likely attributed to different diagnostic criteria, use of prophylactic treatment, and different treatment regimens. Treatment with corticosteroids +/- cytoreductive therapy should commence as soon as DS is suspected to reduce DS-related morbidity and mortality. The targeted anti-leukemic therapy should be discontinued in patients with severe DS. Here, we discuss the pathogenesis of DS, clinical presentations, diagnostic criteria, management strategies, and implementation of prospective tracking on clinical trials.
分化综合征（DS）是一种常见且可能危及生命的临床综合征，随着急性早幼粒细胞白血病（APL）靶向治疗药物的出现首次被认识。随后，在急性髓系白血病（AML）的靶向治疗中更广泛地观察到了DS。DS的典型特征为发热、呼吸困难、低血压、体重增加、胸腔或心包积液以及急性肾衰竭。APL患者中的发病率为2%至37%，差异较大可能归因于不同的诊断标准、预防性治疗的使用以及不同的治疗方案。一旦怀疑有DS，应立即开始使用皮质类固醇+/-细胞减灭疗法，以降低与DS相关的发病率和死亡率。对于严重DS患者，应停用靶向抗白血病治疗。在此，我们讨论DS的发病机制、临床表现、诊断标准、管理策略以及在临床试验中实施前瞻性追踪。
