Hematopoietic SCT in children with Griscelli syndrome: a single-center experience
Hematopoietic SCT in children with Griscelli syndrome: a single-center experience
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DOI：
10.1038/bmt.2009.358
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发表时间：
2010-08-01
影响因子：
4.8
通讯作者：
El-Solh, H.
中科院分区：
文献类型：
--
作者：
Al-Ahmari, A.;Al-Ghonaium, A.;El-Solh, H.
我们回顾了1993至2007年间在本中心接受异基因造血干细胞移植(AHSCT)的连续11例Griscell综合征(GS)2型儿科患者。中位移植年龄为8.2个月(4~36.3个月)，中位诊断至移植时间为3.7个月(1.4~19.5个月)。7名患者出现了加速期，并在移植前接受了化疗。移植时，所有患者均处于临床缓解状态。8例患者的移植物来源是相合相关的骨髓，3例患者的移植物来源是部分不匹配的非血缘关系的脐带。所有患者植骨的平均时间为15天(范围为12-36天)。I~II级急性移植物抗宿主病3例，静脉闭塞1例。目前共有10名患者在接受HSCT后的中位数4.8年内存活并无疾病。4例移植后病程并发巨细胞病毒感染。移植后6个月，1例因败血症休克缓解期死亡。最后一次接触时的嵌合体研究可用于9名患者：6名患者有完全供者嵌合体，3名患者有稳定的混合嵌合体。配型匹配的供者或无血缘关系的脐带血用于GS患儿的早期aHSCT是可行的。骨髓移植(2010年)45,1294年-1299年；DOI：10.1038/bmt.2009.358；2010年1月11日在线发布
In total, 11 consecutive pediatric patients with Griscelli syndrome (GS) type 2, who received allogeneic hematopoietic SCT (aHSCT) at our center between 1993 and 2007, were reviewed. The median age at transplantation was 8.2 months (range, 4-36.3 months) and the median time from diagnosis to transplantation was 3.7 months (range, 1.4-19.5 months). Seven patients developed an accelerated phase and were treated with chemotherapy before transplantation. At the time of transplantation, all patients were in clinical remission. The source of grafts was matched-related marrows in eight patients and partially mismatched unrelated cords in three patients. All patients were engrafted at a median time of 15 days (range, 12-36 days). Grade I-II acute GVHD and veno-occlusive disease occurred in three and one patient, respectively. A total of 10 patients are now alive and disease free at a median of 4.8 years post-HSCT. The post transplant course was complicated by CMV infection in four patients. One patient died in remission from septic shock, 6 months after transplantation. Chimerism studies at the last contact are available for nine patients: six patients have complete donor chimerism and three have stable mixed chimerism. Early aHSCT from matched-related donors or unrelated cord blood for children with GS is feasible. Bone Marrow Transplantation (2010) 45, 1294-1299; doi: 10.1038/bmt.2009.358; published online 11 January 2010