Kapoor N, Crooks G, Kohn D B, Parkman R
Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital Los Angeles, CA 90027, USA.
Semin Hematol. 1998 Oct;35(4):346-53.
Hematopoietic stem cell (HSC) transplantation is curative therapy for many primary immunodeficiencies. All forms of severe combined immune deficiency (SCID) can be cured, but the extent of the immunologic correction is dependent on the pathophysiology of the primary defect. Defects involving lymphocyte differentiation are more easily corrected than defects in lymphocyte function because a selective advantage exists for the progeny of the normal donor HSC when the primary defect affects lymphocyte differentiation. T-cell-depleted (TCD), haploidentical-HSC transplantation can cure many forms of SCID, but not other primary immunodeficiencies like the Wiskott-Aldrich syndrome (WAS). Unrelated bone marrow and umbilical cord blood are alternative sources of HSC for patients who do not have histocompatible donors.
造血干细胞（HSC）移植是治疗多种原发性免疫缺陷的有效疗法。所有形式的重症联合免疫缺陷（SCID）都可治愈，但免疫纠正的程度取决于原发性缺陷的病理生理学。涉及淋巴细胞分化的缺陷比淋巴细胞功能缺陷更容易纠正，因为当原发性缺陷影响淋巴细胞分化时，正常供体造血干细胞的后代具有选择性优势。去除T细胞的（TCD）单倍体相合造血干细胞移植可以治愈多种形式的SCID，但不能治愈其他原发性免疫缺陷，如维斯科特-奥尔德里奇综合征（WAS）。对于没有组织相容性供体的患者，无关供体骨髓和脐带血是造血干细胞的替代来源。
