越来越多的急性髓系白血病 (AML) 治疗方法已被开发出来，它们不是细胞毒性疗法，而是能够恢复正常分化中异常和致白血病“阻断”的靶向药物。全反式视黄酸（ATRA）和三氧化二砷（ATO）是治疗急性早幼粒细胞白血病（APL）的分化剂的经典例子；通过分化发挥作用的新疗法包括异柠檬酸脱氢酶 (IDH) 1 和 2 抑制剂、FMS 样酪氨酸激酶 3 (FLT3) 抑制剂和 menin 抑制剂。通过分化剂治疗对白血病原始细胞进行终末分化可导致一系列体征和症状，最初称为“视黄酸综合征”，现在称为“分化综合征”(DS)，其主要特征是全身炎症反应系统 (SIRS)呼吸困难、肺部浸润、胸膜和心包积液、不明原因发烧、低血压、水肿和肾功能不全等类似特征。新诊断的 APL 患者的 DS 通常很容易识别，但多发性 AML 患者的 DS 诊断起来可能更具挑战性，因为非特异性体征和症状可能被错误地归因于感染性病因或潜在的难治性白血病本身。及时考虑 DS、快速开始全身性皮质类固醇治疗以及伴有白细胞增多症的早期细胞减灭术对于最佳治疗至关重要。版权所有 © 2024 美国血液学会。

An increasing number of acute myeloid leukemia (AML) therapeutics have been developed, not as cytotoxic therapies, but rather as targeted agents able to restore the aberrant and leukemogenic "block" in normal differentiation. All-trans retinoic acid (ATRA) and arsenic trioxide (ATO) are classic examples of differentiating agents for treatment of acute promyelocytic leukemia (APL); newer therapies functioning through differentiation include isocitrate dehydrogenase (IDH) 1 and 2 inhibitors, FMS-like tyrosine kinase 3 (FLT3) inhibitors, and menin inhibitors. The terminal differentiation of leukemic blasts via differentiating agent therapy can lead to a constellation of signs and symptoms, originally referred to as "retinoic acid syndrome" and now termed "differentiation syndrome" (DS), characterized predominantly by systemic inflammatory response system (SIRS)-like features of dyspnea, pulmonary infiltrates, pleural and pericardial effusions, unexplained fevers, hypotension, edema, and renal insufficiency. DS in patients with newly diagnosed APL is generally straightforward to identify, however DS in patients with multiply relapsed AML can be more challenging to diagnose, due to non-specific signs and symptoms which can be mistakenly attributed to infectious etiologies or the underlying refractory leukemia itself. Prompt consideration of DS, rapid initiation of systemic corticosteroids, and early cytoreduction in the setting of concomitant hyperleukocytosis, are essential for optimal management.Copyright © 2024 American Society of Hematology.