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Combination of haploidentical haematopoietic stem cell transplantation with an unrelated cord-blood unit in patients with severe aplastic anemia: a report of 146 cases.
Bone Marrow Transplantation ( IF 4.5 ) Pub Date : 2020-03-26 , DOI: 10.1038/s41409-020-0874-9
Limin Liu 1 , Yanming Zhang 2 , Wenjing Jiao 3 , Huifen Zhou 1 , Qingyuan Wang 1 , Huiying Qiu 1 , Xiaowen Tang 1 , Yue Han 1 , Chengcheng Fu 1 , Zhengming Jin 1 , Suning Chen 1 , Aining Sun 1 , Miao Miao 1 , Depei Wu 1
Affiliation  

We analyzed the outcomes of 146 severe aplastic anemia (SAA) patients who received a combination of haploidentical haematopoietic stem cell transplantation (haplo-HSCT) and an unrelated cord-blood (UCB) unit between September 2011 and December 2017. One hundred and seventeen patients underwent transplantation as first-line therapy. Seven patients experienced early mortality, and among the evaluable 139 patients, one patient experienced primary graft failure (GF), while the other 138 patients achieved successful haploidentical donor engraftment; additionally, three patients experienced secondary GF. Six patients demonstrated delayed platelet recovery, and three patients demonstrated platelet GF. The median time for myeloid and platelet engraftment was 11 (range: 9–28) days and 15 (range: 9–330) days, respectively. With a median follow-up of 40 (range: 18–93) months, the cumulative incidences were 31.43% and 10.00% for grades II–IV and grades III–IV acute graft-versus-host disease (GVHD), respectively. The cumulative incidences of chronic GVHD (cGVHD) and moderate–severe cGVHD were 36.23% and 11.71%, respectively. There was no patient relapse. The probabilities of 4-year overall survival and GVHD-free, failure-free survival were 81.4 ± 3.3% and 69.2 ± 3.9%, respectively. These encouraging preliminary results indicated that haplo-HSCT combined with the infusion of UCB is a feasible choice for SAA patients without matched donors.



中文翻译:

重度再生障碍性贫血患者的单倍型造血干细胞移植与无关的脐血单元结合:146例报告。

我们分析了2011年9月至2017年12月间接受单倍型造血干细胞移植(haplo-HSCT)和无关脐带血(UCB)单元治疗的146例严重再生障碍性贫血(SAA)患者的结果。117例患者接受移植作为一线治疗。7例患者早期死亡,并且在139例患者中,1例患者发生了原发性移植失败(GF),而其他138例患者成功完成了单侧供体植入。另外,三名患者经历了继发性GF。6名患者表现出延迟的血小板恢复,3名患者表现出血小板GF。骨髓和血小板移植的中位时间分别为11天(9-28)天和15天(9-330)天。中位随访40个月(范围:18-93个月),II-IV级和III-IV级急性移植物抗宿主病(GVHD)的累积发生率分别为31.43%和10.00%。慢性GVHD(cGVHD)和中重度cGVHD的累积发生率分别为36.23%和11.71%。没有患者复发。4年总生存率和无GVHD,无衰竭生存率分别为81.4±3.3%和69.2±3.9%。这些令人鼓舞的初步结果表明,对于没有匹配供体的SAA患者,单倍HSCT结合UCB输注是可行的选择。慢性GVHD(cGVHD)和中重度cGVHD的累积发生率分别为36.23%和11.71%。没有患者复发。4年总生存率和无GVHD,无衰竭生存率分别为81.4±3.3%和69.2±3.9%。这些令人鼓舞的初步结果表明,对于没有匹配供体的SAA患者,单倍HSCT结合UCB输注是可行的选择。慢性GVHD(cGVHD)和中重度cGVHD的累积发生率分别为36.23%和11.71%。没有患者复发。4年总生存率和无GVHD,无衰竭生存率分别为81.4±3.3%和69.2±3.9%。这些令人鼓舞的初步结果表明,对于没有匹配供体的SAA患者,单倍HSCT联合UCB灌注是可行的选择。

更新日期:2020-04-24
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