The hallmark of acute promyelocytic leukemia (APL) is the presence of the characteristic fusion transcript of the promyelocytic leukemia gene with the retinoic acid receptor α gene (PML::RARA). The PML::RARA fusion is a molecular target for all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). Therapies based on ATRA plus ATO have excellent outcomes in terms of complete remission rates, overall survival, and achievement of deep and durable molecular responses with a very low incidence of relapse. However, although the combination of ATRA and ATO has lower hematologic toxicity than standard chemotherapy, its use is associated with a spectrum of distinctive toxicities, such as differentiation syndrome, liver toxicity, QT interval prolongation, and neurotoxicity. Rigorous monitoring of patients’ clinical evolution is indispensable for identifying and addressing each complication. The objective is to maintain an equilibrium between treatment-induced adverse events and therapeutic efficacy. This paper focused on non-hematologic complications associated with the combination of ATRA and ATO. Additionally, we discuss late-onset complications of this therapy. In summary, the majority of treatment-related adverse events are manageable, self-limiting, and reversible. More so, there seems to be a lower incidence rate of secondary neoplasms compared to standard chemotherapy. However, further research is required to assess how the ATRA plus ATO regimen affects the emergence of additional comorbidities.
急性早幼粒细胞白血病(APL)的标志性特征是存在早幼粒细胞白血病基因与维甲酸受体α基因的特征性融合转录本(PML::RARA)。PML::RARA融合是全反式维甲酸(ATRA)和三氧化二砷(ATO)的分子靶点。基于ATRA联合ATO的治疗方案在完全缓解率、总生存率以及实现深度持久的分子学反应方面具有优异疗效,且复发率极低。然而,尽管ATRA与ATO联合方案的血液学毒性低于标准化疗,但其使用伴有一系列特征性毒性反应,如分化综合征、肝毒性、QT间期延长和神经毒性。严密监测患者的临床演变对于识别和处理各类并发症至关重要,其目标是在治疗相关不良事件与疗效之间维持平衡。本文重点探讨ATRA联合ATO方案相关的非血液学并发症,同时讨论该疗法可能出现的迟发性并发症。总体而言,大多数治疗相关不良事件具有可控性、自限性和可逆性。与标准化疗相比,该方案继发肿瘤的发生率似乎更低。然而,仍需进一步研究评估ATRA联合ATO方案对患者其他合并症发生发展的影响。