首页> 外文期刊>International journal of hematology >B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID
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B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID

机译:X-SCID患者使用最小强度调节方案进行无关脐带血移植后的B细胞功能

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摘要

Patients with X-linked severe combined immunodeficiency (X-SCID) suffer from severe and persistent infections, and usually die early in life unless treated by hematopoietic stem cell transplantation. If a patient has an HLA-identical sibling donor, preparative conditioning is not necessary for T-cell engraftment and B-cell function. However, in the absence of such a donor, long-term reconstitution of full B-cell function is often problematic, leading in many cases to a lifetime requirement for immunoglobulin replacement therapy. Preparative myeloablative conditioning has been shown to improve long-term B-cell function, but may aggravate pre-existing infection and transplant-related toxicity. It is thus important to determine the minimum intensity of conditioning that assures immunoglobulin production. In the present study, we performed reduced-intensity conditioning (RIC), consisting of fludarabine 125 mg/m2 and melphalan 80 mg/m2, prior to unrelated umbilical cord blood transplantation (UCBT) for five patients with X-SCID, none of them had an HLA-identical donor. Four patients survived more than 4 years without sequelae, and none required long-term immunoglobulin replacement therapy. One patient succumbed to sepsis in conjunction with severe GVHD. Our result demonstrates that the RIC regimen described above in combination with UCBT is an effective and less toxic conditioning to correct B-cell function in patients with X-SCID.
机译:X连锁严重合并免疫缺陷病(X-SCID)的患者患有严重和持续性感染,并且通常在生命早期死亡,除非通过造血干细胞移植治疗。如果患者具有与HLA相同的同胞供体,则对于T细胞植入和B细胞功能不需要进行必要的准备。然而,在没有这种供体的情况下,长期重建完整的B细胞功能通常是有问题的,在许多情况下导致终身需要免疫球蛋白替代疗法。制备性清髓性调理已显示可改善长期B细胞功能,但可能加重先前的感染和与移植相关的毒性。因此,重要的是确定确保免疫球蛋白产生的最小调节强度。在本研究中,我们对5例X-SCID患者进行了不相关的脐血移植(UCBT)之前的低强度调节(RIC),包括氟达拉滨125 mg / m2和美法仑80 mg / m2。拥有与HLA相同的捐助者。 4名患者存活超过4年而没有后遗症,并且都没有需要长期免疫球蛋白替代治疗的情况。一名患者因严重的GVHD而败血症。我们的结果表明,上述RIC方案与UCBT联合使用可有效纠正X-SCID患者的B细胞功能,且毒性较小。

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