Collins P, Watts M, Brocklesby M, Gerritsen B, Veys P
Department of Haematology, Institute of Child Health and Great Ormond Street Hospital for Children NHS Trust, London.
Br J Haematol. 1997 Mar;96(3):644-6. doi: 10.1046/j.1365-2141.1997.d01-2050.x.
Familial haemophagocytic lymphohistiocytosis (HLH) is a disease with a very poor prognosis unless patients receive a bone marrow transplant. It is often difficult to find an HLA-matched donor and haploidentical familial donors may be considered. The main complication of this type of transplant is graft rejection. We describe a patient with familial HLH who received a haploidentical transplant using both mobilized peripheral blood and bone marrow stem cells in an attempt to overcome graft rejection by increasing the stem cell dose. The peripheral blood stem cell inoculum was CD34 enriched using a Cellpro column and T-cell depleted by Campath-1M, the patient received conditioning for a matched sibling donor transplant with the addition of Campath 1G. There was rapid and full engraftment and the patient remains disease free at 5 months. This technique may be applicable for other fatal inborn errors in the absence of an HLA-matched donor.
家族性噬血细胞性淋巴组织细胞增生症（HLH）是一种预后极差的疾病，除非患者接受骨髓移植。通常很难找到 HLA 匹配的供体，此时可考虑单倍体相合的家族供体。这类移植的主要并发症是移植物排斥反应。我们描述了一名患有家族性 HLH 的患者，该患者接受了单倍体相合移植，使用了动员外周血和骨髓干细胞，试图通过增加干细胞剂量来克服移植物排斥反应。外周血干细胞接种物使用 Cellpro 柱进行 CD34 富集，并通过 Campath-1M 去除 T 细胞，患者接受了与匹配同胞供体移植相同的预处理，并加用了 Campath 1G。移植后迅速实现了完全植入，患者在 5 个月时仍无疾病。在没有 HLA 匹配供体的情况下，这项技术可能适用于其他致命的先天性疾病。
