Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita
Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita
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DOI：
10.1038/sj.bmt.1703931
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发表时间：
2003-05-01
影响因子：
4.8
通讯作者：
Doyle, JJ
中科院分区：
文献类型：
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作者：
Dror, Y;Freedman, MH;Doyle, JJ
由于先天性角化不良（DC）患者的常规造血干细胞移植（HSCT）结果较差，因为移植相关并发症的发生率较高，我们探索了低强度HSCT方案的使用。我们报告了两名患有严重血细胞减少症的DC儿童，他们在接受氟达拉滨、环磷酰胺和抗胸腺细胞球蛋白预处理后，成功地从一名匹配的无关供体接受了HSCT。移植物抗宿主病（GVHD）预防包括皮质类固醇和环孢菌素A。该方案耐受性良好，未观察到明显的移植相关并发症，植入迅速而完全。在HSCT后15和16个月，这些儿童完全植入，临床状况良好，完全供体嵌合，没有GVHD的迹象。我们得出结论，低强度方案足以诱导DC患者使用匹配的无关供体HSCT的持久植入，1年移植相关毒性最小。更长时间的随访将确定该方案是否也降低了长期毒性。
Since the results of conventional hematopoietic stem-cell transplantation (HSCT) for patients with dyskeratosis congenita (DC) are poor owing to the high incidence of transplant-related complications, we explored the use of a low-intensity HSCT regimen. We report two children with DC with severe cytopenia, who underwent successful HSCT from a matched unrelated donor after conditioning with fludarabine, cyclophosphamide, and antithymocyte globulin. Graft-versus-host-disease (GVHD) prophylaxis consisted of corticosteroids and cyclosporin A. The regimen was well tolerated, no significant transplant-related complications were observed, and engraftment was rapid and complete. At 15 and 16 months after HSCT, the children were fully engrafted, in excellent clinical condition, full-donor chimerism, and no signs of GVHD. We conclude that a low-intensity regimen is sufficient to induce durable engraftment using matched unrelated donor HSCT in DC patients, with minimal 1-year transplant-related toxicity. Longer follow-up will determine whether this regimen also reduces long-term toxicity.