Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.
Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.
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DOI：
10.1111/bjh.17311
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发表时间：
2021-03
影响因子：
6.5
通讯作者：
Hsieh MM
中科院分区：
文献类型：
--
作者：
Alzahrani M;Damlaj M;Jeffries N;Alahmari B;Singh A;Rondelli D;Tisdale JF;Saraf SL;Hsieh MM
血缘相合供者的非清髓性造血祖细胞移植(HPCT)在镰状细胞病(SCD)中的应用日益广泛。122例患者接受300cGy全身照射(TBI)、阿伦图珠单抗、未经处理的非格列汀动员的外周血HPC和西罗莫司。中位随访期为四年；HPCT的中位年龄为29岁。中性粒细胞和血小板植入分别发生在第22天和第19天；41例患者不需要输注血小板。1年和5年的总生存率和无镰刀生存率分别为93%和85%。移植失败和移植患者的年龄、性别、HPCT前镰刀并发症、铁蛋白和输注的HPC数量相似。供者骨髓嵌合率在移植后1年和5年分别为84%和88%，CD3分别为48%和53%。两名患者发展为1级和2级皮肤移植物抗宿主病(GVHD)，没有慢性GVHD。接受免疫抑制的受者的平均天数为489天；83%的移植患者已停止免疫抑制。HPCT后，患者的血红蛋白、溶血参数和肝铁水平均有改善。肺功能测试、肝脏组织学和神经血管成像保持稳定，表明进一步的镰刀相关损伤已经停止。14名患者有孩子。在这一最大的成年SCD患者组中，尽管HPCT前器官功能受损，但该方案非常有效，耐受性良好，并且没有临床意义的GVHD。
Non-myeloablative haematopoietic progenitor cell transplantation (HPCT) from matched related donors (MRD) has been increasingly utilized in sickle cell disease (SCD). A total of 122 patients received 300 cGy of total body irradiation (TBI), alemtuzumab, unmanipulated filgrastim-mobilized peripheral blood HPC and sirolimus. The median follow-up was four years; median age at HPCT was 29 years. Median neutrophil and platelet engraftment occurred on day 22 and 19 respectively; 41 patients required no platelet transfusions. Overall and sickle-free survival at one and five years were 93% and 85% respectively. Age, sex, pre-HPCT sickle complications, ferritin and infused HPC numbers were similar between graft failure and engrafted patients. Mean donor myeloid chimaerism at one and five years post HPCT were 84% and 88%, and CD3 was 48% and 53% respectively. Two patients developed grade 1 and 2 skin graft-versus-host disease (GVHD) with no chronic GVHD. Median days of recipients taking immunosuppression were 489; 83% of engrafted patients have discontinued immunosuppression. Haemoglobin, haemolytic parameters and hepatic iron levels improved post HPCT. Pulmonary function testing, hepatic histology and neurovascular imaging remained stable, suggesting cessation of further sickle-related injury. Fourteen patients had children. In this largest group of adult SCD patients, this regimen was highly efficacious, well-tolerated despite compromised organ functions pre HPCT, and without clinically significant GVHD.
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影响因子：
4.8
作者：
Nollet, F;Billiet, J;Criel, A
通讯作者：
Criel, A
影响因子：
20.3
作者：
Hsieh, Matthew M.;Fitzhugh, Courtney D.;Tisdale, John F.
通讯作者：
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影响因子：
10.1
作者：
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通讯作者：
Socie, Gerard
影响因子：
4.3
作者：
de la Fuente, Josu;Dhedin, Nathalie;Kassim, Adetola A.
通讯作者：
Kassim, Adetola A.
影响因子：
158.5
作者：
Niihara, Yutaka;Miller, Scott T.;Vichinsky, Elliott P.
通讯作者：
Vichinsky, Elliott P.