Long-term Survival, Organ Function, and Malignancy after Hematopoietic Stem Cell Transplantation for Fanconi Anemia
Long-term Survival, Organ Function, and Malignancy after Hematopoietic Stem Cell Transplantation for Fanconi Anemia
复制标题
DOI：
10.1016/j.bbmt.2016.03.007
复制
发表时间：
2016-07-01
影响因子：
4.3
通讯作者：
Pasquini, Ricardo
中科院分区：
文献类型：
--
作者：
Bonfim, Carmem;Ribeiro, Lisandro;Pasquini, Ricardo
我们报告了157例范可尼贫血（FA）患者的长期生存情况，这些患者在首次移植后存活了2年或更长时间，中位随访时间为9年。骨髓衰竭（80%）是最常见的移植指征。移植后2年内有20例死亡，其中12例死亡发生在移植后5年内。149例患者的供体嵌合体可用：112例（76%）报告> 95%嵌合体，27例（18%）报告90%至95%嵌合体，8例（5%）报告20%至89%供体嵌合体。两名患者具有< 20%的供体嵌合体。10年和15年生存率分别为90%和79%。多变量分析结果显示，2003年以前移植的死亡风险较高（危险比[HR]，7.87; P =.001），慢性移植物抗宿主病（GVHD）（HR，3.80; P =.004）和移植后鳞状细胞癌（HR，38.17; P =.001）。
We report on long-term survival in 157 patients with Fanconi anemia (FA) who survived 2 years or longer after their first transplantation with a median follow-up of 9 years. Marrow failure (80%) was the most common indication for transplantation. There were 20 deaths beyond 2 years after transplantation, with 12 of the deaths occurring beyond 5 years after transplantation. Donor chimerism was available for 149 patients: 112 (76%) reported > 95% chimerism, 27 (18%) reported 90% to 95% chimerism, and 8 (5%) reported 20% to 89% donor chimerism. Two patients have < 20% donor chimerism. The 10- and 15-year probabilities of survival were 90% and 79%, respectively. Results of multivariate analysis showed higher mortality risks for transplantations before 2003 (hazard ratio [HR], 7.87; P =.001), chronic graft-versus-host disease (GVHD) (HR, 3.80; P =.004) and squamous cell carcinoma after transplantation (HR, 38.17; P