Lenarsky C, Parkman R
Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital, Los Angeles, CA 90027.
Bone Marrow Transplant. 1990 Dec;6(6):361-9.
The first successful allogeneic bone marrow transplants were performed in children with severe combined immune deficiency (SCID). Bone marrow transplants for patients with SCID have been in the forefront of clinical bone marrow transplantation including the first successful use of T lymphocyte-depleted haploidentical bone marrow and matched unrelated donors. Successful bone marrow transplantation for most forms of SCID requires only the engraftment of donor lymphoid stem cells; donor hematopoietic stem cell engraftment is usually not required. The Wiskott-Aldrich syndrome was the first genetic disease involving the hematopoietic stem cell to be completely corrected by allogeneic bone marrow transplantation. The successful transplantation of Wiskott-Aldrich syndrome patients demonstrated that agents with adequate anti-lymphoid and hematopoietic stem cell activity were necessary in order to achieve complete donor lymphoid and hematopoietic stem cell engraftment. Initially, total body irradiation and now busulfan are used to ablate recipient hematopoietic stem cells, while cyclophosphamide is used to ablate recipient lymphoid stem cells. No single agent/drug is capable of eliminating both stem cell populations. Histocompatible bone marrow transplantation has a role in the treatment of patients with immune deficiency due to primary defects of the hematopoietic stem cell. The recent introduction of cytokines (gamma-interferon and granulocyte colony stimulating factor) may reduce the need for bone marrow transplantation for myeloid immune deficiency states. Initial attempts to treat patients with the acquired immune deficiency syndrome by bone marrow transplantation were limited by the lack of effective concomitant anti-viral therapy. Bone marrow transplantation for immune deficiency states continues to be in the forefront of human bone marrow transplantation.
首例成功的异基因骨髓移植是在患有严重联合免疫缺陷（SCID）的儿童中进行的。针对SCID患者的骨髓移植一直处于临床骨髓移植的前沿，包括首次成功使用去除T淋巴细胞的单倍体相合骨髓和匹配的无关供体。对于大多数形式的SCID，成功的骨髓移植仅需要供体淋巴干细胞植入；通常不需要供体造血干细胞植入。维斯科特-奥尔德里奇综合征是第一种通过异基因骨髓移植得到完全纠正的涉及造血干细胞的遗传性疾病。维斯科特-奥尔德里奇综合征患者的成功移植表明，为了实现供体淋巴和造血干细胞的完全植入，具有足够抗淋巴细胞和造血干细胞活性的药物是必要的。最初，全身照射以及现在的白消安用于清除受体造血干细胞，而环磷酰胺用于清除受体淋巴干细胞。没有单一的药物能够消除这两种干细胞群体。组织相容性骨髓移植在治疗因造血干细胞原发性缺陷导致免疫缺陷的患者中发挥作用。细胞因子（γ干扰素和粒细胞集落刺激因子）的近期引入可能会减少骨髓移植治疗髓系免疫缺陷状态的需求。最初尝试通过骨髓移植治疗获得性免疫缺陷综合征患者受到缺乏有效的联合抗病毒治疗的限制。免疫缺陷状态的骨髓移植仍然处于人类骨髓移植的前沿。
