Department of Pediatrics, University of Ulm, Eythstrasse 24, 89075 Ulm, Germany.
Immunol Allergy Clin North Am. 2010 Feb;30(1):31-44. doi: 10.1016/j.iac.2009.11.004.
Curative treatment of Severe Combined Immunodeficiency (SCID) by Hematopoietic Cell Transplantation (HCT) remains a challenge, in particular in infants presenting with serious, poorly controllable complications. In the absence of a matched family donor, HLA-haploidentical transplantation from parental donors represents a uniformly and readily available treatment option, offering a high chance to be successful. Concerning outcomes of HCT in SCID, other important parameters beside survival need to be taken into consideration, in particular the stability and robustness of the graft and its function, as well as potential late complications, related either to the disease or to the treatment.
造血干细胞移植（HCT）治疗严重联合免疫缺陷（SCID）仍然是一个挑战，尤其是在出现严重、难以控制的并发症的婴儿中。在没有匹配的家族供体的情况下，来自父母供体的 HLA 单倍体移植是一种普遍且易于获得的治疗选择，成功率很高。关于 SCID 患者 HCT 的结果，除了生存率之外，还需要考虑其他重要参数，特别是移植物的稳定性和功能，以及潜在的迟发性并发症，这些并发症可能与疾病或治疗有关。
