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首页> 外文期刊>Clinical immunology: The official journal of the Clinical Immunology Society >Stem cell transplants for patients with X-linked agammaglobulinemia.
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Stem cell transplants for patients with X-linked agammaglobulinemia.

机译：X连锁无球蛋白血症患者的干细胞移植。

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摘要

Six young patients with X-linked agammaglobulinemia and proven mutations in Btk were treated with cord blood or bone marrow transplants from HLA-matched siblings. Complete blood counts, serum chemistries, serum immunoglobulin concentrations, lymphocyte cell surface markers, and physical findings were evaluated at 3- to 5-day intervals for the first 2 weeks after transplant and then every 3 to 6 months. The first three patients were not given any preparative regimen or antirejection drugs and at 24 to 42 months posttransplant these patients have shown no benefit or harm related to the transplants. The second three patients were not given a preparative regimen but were treated with cyclosporine A (70 days) and mycophenolate mophetil (28 days) after transplant. Two of these patients have developed normal sized, nontender cervical lymph nodes 3 to 12 months after transplant but none of the three patients have shown an increase in serum IgM or an increase in the number of peripheral blood B cells. It is likely that successful engraftment will require more aggressive immunosupressive medications.
机译：用来自HLA匹配兄弟姐妹的脐带血或骨髓移植治疗了6名年轻的X连锁无球蛋白血症和Btk突变的年轻患者。在移植后的最初2周中，每隔3至5天评估一次全血细胞计数，血清化学成分，血清免疫球蛋白浓度，淋巴细胞表面标志物和物理发现，然后每3至6个月评估一次。前三名患者未接受任何准备方案或抗排斥药，并且在移植后24至42个月，这些患者未显示与移植相关的益处或伤害。后三名患者未接受任何准备，但在移植后接受了环孢霉素A（70天）和霉酚酸酯（28天）治疗。这些患者中有两个在移植后3至12个月出现了正常大小的非嫩颈淋巴结，但三位患者中没有一个显示出血清IgM升高或外周血B细胞数量升高。成功的植入可能需要更积极的免疫抑制药物。

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