Acute promyelocytic leukemia (APL) currently represents one of the malignant hemopathies with the best therapeutic responses, following the introduction of all-trans retinoic acid (ATRA) and subsequently of arsenic trioxide (ATO) treatment. As a result, a large proportion of patients with APL achieve long-term responses after first-line therapy, so performing a hematopoietic stem cell transplant as consolidation of first complete remission (CR) is no longer necessary. Even in the case of relapses, most patients obtain a new remission as a result of therapy with ATO and ATRA, but an effective consolidation treatment is necessary to maintain it. The experience accumulated from studies published in the last two decades shows the effectiveness of hematopoietic stem cell transplantation (HSCT) in improving the outcome of patients who achieve a new CR. Thus, the expert groups recommend transplantation as consolidation therapy in patients with a second CR, with the indication for autologous HSCT in cases with molecular CR and for allogeneic HSCT in patients with the persistence of minimal residual disease (MRD) or with early relapse. However, there is a variety of controversial aspects related to the role of HSCT in APL, ranging from the fact that outcome data are obtained almost exclusively from retrospective studies and historical analyses to questions related to the type of transplantation, the impact of minimal residual disease, conditioning regimens, or the role of other therapeutic options. All these questions justify the need for controlled prospective studies in the following years.
急性早幼粒细胞白血病（APL）自全反式维甲酸（ATRA）及后续三氧化二砷（ATO）疗法应用以来，已成为目前治疗效果最佳的恶性血液病之一。这使得大部分APL患者在一线治疗后能获得长期缓解，因此无需在首次完全缓解（CR）阶段进行造血干细胞移植作为巩固治疗。即使在复发情况下，多数患者通过ATO与ATRA联合治疗仍能再次获得缓解，但需要有效的巩固治疗以维持疗效。近二十年来发表的研究积累表明，造血干细胞移植（HSCT）能显著改善再次获得CR患者的预后。因此，专家建议将移植作为第二次CR患者的巩固治疗方案：达到分子学CR的患者适用自体HSCT，而存在微小残留病（MRD）或早期复发的患者则适用异基因HSCT。然而，关于HSCT在APL治疗中的作用仍存在诸多争议，包括疗效数据主要来源于回顾性研究和历史分析、移植类型的选择、微小残留病的影响、预处理方案的设计以及其他治疗方案的定位等问题。这些争议点都凸显了未来数年开展前瞻性对照研究的必要性。
肿瘤(癌症)患者之家