Splenectomy and/or bone marrow transplantation in the management of the Wiskott-Aldrich syndrome: long-term follow-up of 62 cases.
Splenectomy and/or bone marrow transplantation in the management of the Wiskott-Aldrich syndrome: long-term follow-up of 62 cases.
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脾切除和/或骨髓移植治疗 Wiskott-Aldrich 综合征：62 例长期随访。
DOI：
10.1182/blood.v82.10.2961.2961
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发表时间：
1993
期刊：
影响因子：
20.3
通讯作者：
R. M. Blaese
中科院分区：
文献类型：
--
作者：
C. A. Mullen;K. Anderson;R. M. Blaese
本研究描述了两种主要的治疗选择，脾切除术和/或骨髓移植，对Wiskott-Aldrich（WAS）综合征的自然史的影响。本文回顾了1966年至1992年在美国国立卫生研究院临床中心评估的62例WAS患者的记录。19例患者接受了骨髓移植（BMT）治疗，结果在很大程度上取决于移植物的来源。12例接受HLA匹配同胞骨髓的患者中有12例获得了满意的免疫和血液重建。相比之下，7例接受单倍体相合、亲本或匹配的无关骨髓移植的患者中只有2例在BMT后存活超过1年。39例在病程早期缺乏合适的骨髓供体的患者接受了脾切除术以治疗血小板减少症;大多数患者接受了预防性抗生素治疗，以尽量减少败血症的风险。几乎所有这些患者的血小板计数都达到正常，严重出血的发生率降低了近7倍。非移植脾切除组的中位生存期为25年，而非脾切除组的中位生存期不到5年。我们的结论是，HLA匹配的同胞供体骨髓移植是治疗WAS患者的首选，脾切除术和每日预防性抗生素提供了显着的生存优势，那些男孩没有匹配的同胞供体。脾切除术可能应优先于不匹配的BMT，直到替代供体BMT的结果显着改善或基因治疗变得可用。
This study describes the effects of two major treatment options, splenectomy and/or bone marrow transplantation, on the natural history of the Wiskott-Aldrich (WAS) syndrome. The records of 62 patients with the WAS evaluated at the National Institutes of Health Clinical Center from 1966 to 1992 were reviewed. Nineteen patients were treated with bone marrow transplantation (BMT) and the results were largely dependent on the source of the graft. Twelve of 12 patients receiving HLA-matched sibling marrow achieved satisfactory immunologic and hematologic reconstitution. By contrast, only 2 of 7 patients receiving haploidentical, parental, or matched unrelated marrow survived more than 1 year after BMT. Thirty-nine patients who lacked suitable bone marrow donors early in their course underwent splenectomy for management of their thrombocytopenia; most received prophylactic antibiotics to minimize the risk of sepsis. Nearly all these patients achieved normal platelet counts and the rate of serious bleeding was reduced nearly sevenfold. Median survival in the untransplanted splenectomy group was 25 years, compared with less than 5 years in unsplenectomized patients. We conclude that HLA-matched sibling donor BMT is the treatment of choice for patients with WAS and that splenectomy and daily prophylactic antibiotics provide a significant survival advantage to those boys without a matched sibling donor. Splenectomy should probably be used in preference to unmatched BMT until results with alternative donor BMT significantly improve or gene therapy becomes available.
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DOI：
10.1016/0955-3886(91)90119-n
发表时间：
1991
期刊：
Transfusion science
影响因子：
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DOI：
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发表时间：
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期刊：
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影响因子：
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影响因子：
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作者：
Rimm,IJ;Rappeport,JM
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DOI：
10.1016/s0022-3476(05)83041-0
发表时间：
1991
期刊：
The Journal of pediatrics
影响因子：
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作者：
Brochstein,JA;Gillio,AP;Ruggiero,M;Kernan,NA;Emanuel,D;Laver,J;Small,T;O'Reilly,RJ
通讯作者：
O'Reilly,RJ