Bertaina Alice, Pitisci Angela, Sinibaldi Matilde, Algeri Mattia
Department of Pediatric Hematology-Oncology, IRCCS, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165, Rome, Italy.
Curr Hematol Malig Rep. 2017 Feb;12(1):68-78. doi: 10.1007/s11899-017-0364-3.
Hematopoietic stem cell transplantation (HSCT) is a treatment option for children with malignant and non-malignant disorders as well as an expanding number of inherited disorders. However, only a limited portion of patients in the need of an allograft have an HLA-compatible, either related or unrelated, donor. Haploidentical HSCT is now considered a valid treatment option, especially in view of the recent insights in terms of graft manipulation. This review will offer an overview of clinical results obtained through the use of haploidentical HSCT in non-malignant diseases. We will analyze major advantages and drawbacks of both T cell depleted and unmanipulated HSCT, discussing future challenges for further improving patients' outcome.
T cell depletion (TCD) aims to reduce the morbidity and mortality associated with graft-versus-host disease (GvHD). However, the delayed immune recovery and the risk of graft failure still remain potential problems. In the last years, the use of post-transplant cyclophosphamide has been shown to be an alternative effective strategy to prevent GvHD in recipients of haploidentical HSCT. Recent data suggest that both T cell depleted and T cell-replete haplo-HSCT are suitable options to treat children with several types of non-malignant disorders lacking an HLA-identical donor.
造血干细胞移植（HSCT）是治疗患有恶性和非恶性疾病以及越来越多遗传性疾病儿童的一种治疗选择。然而，需要同种异体移植的患者中，只有有限一部分有 HLA 相合的供者，无论是亲属供者还是非亲属供者。单倍体 HSCT 现在被认为是一种有效的治疗选择，特别是鉴于最近在移植物操作方面的见解。本综述将概述通过使用单倍体 HSCT 治疗非恶性疾病所获得的临床结果。我们将分析 T 细胞去除和未处理的 HSCT 的主要优缺点，讨论进一步改善患者预后的未来挑战。
T 细胞去除（TCD）旨在降低与移植物抗宿主病（GvHD）相关的发病率和死亡率。然而，免疫恢复延迟和移植物失败的风险仍然是潜在问题。在过去几年中，移植后环磷酰胺的使用已被证明是预防单倍体 HSCT 受者 GvHD 的另一种有效策略。最近的数据表明，T 细胞去除和 T 细胞充足的单倍体 HSCT 都是治疗缺乏 HLA 相同供者的几种类型非恶性疾病儿童的合适选择。
