Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study
Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study
复制标题
DOI：
10.1182/blood-2010-11-319376
复制
发表时间：
2011-08-11
期刊：
影响因子：
20.3
通讯作者：
Notarangelo, Luigi D.
中科院分区：
文献类型：
--
作者：
Moratto, Daniele;Giliani, Silvia;Notarangelo, Luigi D.
在这项回顾性合作研究中，我们分析了1980-2009年期间接受造血细胞移植（HCT）治疗的194例Wiskott-Aldrich综合征（WAS）患者的长期结局和供体细胞植入。总生存率为84.0%，自2000年以来接受HCT的患者的总生存率更高（89.1%的5年生存率），反映了从不匹配的家庭供体移植后以及从5岁以上的无关供体接受HCT的患者的近期结局改善。在更好的临床条件下进行移植的患者HCT后并发症的发生率较低。对谱系特异性供体细胞移植的回顾性分析显示，72.3%的HCT后存活至少1年的患者获得了稳定的完全供体嵌合体。混合嵌合体与淋巴细胞计数不完全重建和HCT后自身免疫的风险增加相关，髓系供体细胞嵌合体< 50%与持续性血小板减少相关。这些观察结果表明，WAS的HCT后结局持续改善，并可能对开发旨在完全纠正疾病和减少HCT后并发症的新方案具有重要意义。（血。2011;118（6）：1675-1684）
In this retrospective collaborative study, we have analyzed long-term outcome and donor cell engraftment in 194 patients with Wiskott-Aldrich syndrome (WAS) who have been treated by hematopoietic cell transplantation (HCT) in the period 1980-2009. Overall survival was 84.0% and was even higher (89.1% 5-year survival) for those who received HCT since the year 2000, reflecting recent improvement of outcome after transplantation from mismatched family donors and for patients who received HCT from an unrelated donor at older than 5 years. Patients who went to transplantation in better clinical conditions had a lower rate of post-HCT complications. Retrospective analysis of lineage-specific donor cell engraftment showed that stable full donor chimerism was attained by 72.3% of the patients who survived for at least 1 year after HCT. Mixed chimerism was associated with an increased risk of incomplete reconstitution of lymphocyte count and post-HCT autoimmunity, and myeloid donor cell chimerism < 50% was associated with persistent thrombocytopenia. These observations indicate continuous improvement of outcome after HCT for WAS and may have important implications for the development of novel protocols aiming to obtain full correction of the disease and reduce post-HCT complications. (Blood. 2011;118(6):1675-1684)