Up-front alternative donor HCT in severe aplastic anemia: gaps and opportunities to translate evidence into practice.

Immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporine has long been a frontline treatment option in SAA; however, its limited durability and risk of long-term complications such as secondary malignancies remain a drawback in this treatment modality.

长期以来，抗胸腺细胞球蛋白和环孢素的免疫抑制治疗（IST）一直是SAA的一线治疗选择；然而，其有限的持久性和长期并发症（如继发性恶性肿瘤）的风险仍然是这种治疗方式的缺点。

Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative option with significantly improved outcomes over the long term, particularly with HLA-matched related donors.

异基因造血干细胞移植（HCT）是一种潜在的治愈性选择，长期预后显著改善，特别是HLA相合的亲属供者。

However, the use of alternative donors, such as haploidentical, mismatched, or matched unrelated donors, has previously been limited due to increased transplant-related morbidity, particularly graft-versus-host disease (GVHD).

然而，使用替代供者，如半相合、不相合或相合无关供者的使用，由于移植相关发病率增加，特别是移植物抗宿主病（GVHD），之前受到了限制。

HCTs have therefore been limited to young recipients and those with HLA-matched related donors, creating significant disparity for older adults and those who lack matched donor options.

因此，造血干细胞移植（HCTs）主要限于年轻的受者和拥有HLA匹配相关供体的患者，这为老年患者和缺乏匹配供体选项的患者造成了显著的差异。

Nevertheless, more recent advances in HCT, such as posttransplant cyclophosphamide for GVHD prophylaxis, have led to improved outcomes of HCT with alternative donors; however, alternative donor HCT remains underused as up-front therapy, in part because of limited multicenter trial data.

然而，最近在HCT方面的进步，如移植后环磷酰胺用于预防移植物抗宿主病（GVHD），已经改善了使用替代供体的HCT的治疗结果；然而，由于缺乏多中心试验数据，替代供体HCT作为首选治疗的使用仍然不足。

This review discusses current SAA treatment approaches, including both IST and HCT, and highlights remaining gaps.

本综述讨论了当前重型再生障碍性贫血(SAA)的治疗方法，包括免疫抑制治疗(IST)和造血干细胞移植(HCT)，并指出了尚存的空白。

It also discusses how ongoing clinical trials such as CureAA and TransIT could help address these gaps.

此外，本综述还讨论了正在进行的临床试验，如CureAA和TransIT，如何帮助解决这些空白。

Furthermore, we discuss the importance of stakeholder engagement and implementation science in the integration of research-based evidence into clinical practice.

此外，我们讨论了利益相关者参与和实施科学在将基于研究的证据整合到临床实践中的重要性。

Bridging these gaps is necessary for achieving equitable access for patients historically excluded from frontline HCT, including older adults and racially or ethnically diverse populations.

弥合这些差距对于实现历史上被排除在前线造血干细胞移植（HCT）之外的患者群体的公平访问至关重要，包括老年人以及种族或民族多样性的人群。