Myeloablative and reduced-intensity conditioning in HLA-haploidentical peripheral blood stem cell transplantation using post-transplant cyclophosphamide
Myeloablative and reduced-intensity conditioning in HLA-haploidentical peripheral blood stem cell transplantation using post-transplant cyclophosphamide
复制标题
DOI：
10.1038/s41409-018-0279-1
复制
发表时间：
2019-03-01
影响因子：
4.8
通讯作者：
Teshima, Takanori
中科院分区：
文献类型：
--
作者：
Sugita, Junichi;Kagaya, Yusuke;Teshima, Takanori
我们进行了两项平行、前瞻性、多中心、II期研究，以评估在清骨髓调节（MAC, n = 50）和降低强度调节（RIC, n = 77）后使用移植后环磷酰胺（pcy - haplopbsct）进行hla -单倍体外周血干细胞移植的安全性和有效性。MAC组和RIC组的1年无事件生存率（EFS）分别为64%和43%。MAC组和RIC组的中性粒细胞移植率分别为98%和94%。II-IV级和III-IV级急性移植物抗宿主病（GVHD）的发生率在MAC组分别为18%和8%，在RIC组分别为14%和5%。2年时，MAC组中所有级别和中重度慢性GVHD的发生率分别为36%和20%，RIC组为27%和20%。MAC组的总生存率（OS）、EFS、非复发死亡率和2年复发率分别为68%、54%、10%和36%，RIC组为44%、35%、20%和45%。值得注意的是，在MAC组和RIC组中，分别有83%和86%的患者在2年后停止使用免疫抑制剂。我们的研究结果表明，MAC和RIC都是成人恶性血液病患者pcy - haplopbsct的有效选择。
We conducted two parallel prospective, multicenter, phase II studies to evaluate the safety and efficacy of HLA-haploidentical peripheral blood stem cell transplantation using post-transplant cyclophosphamide (PTCy-haploPBSCT) following myeloablative conditioning (MAC, n = 50) and reduced-intensity conditioning (RIC, n = 77). Event-free survival (EFS) at 1-year as for primary endpoint was 64% and 43% in the MAC and RIC groups, respectively. Neutrophil engraftment was achieved in 98% and 94% in the MAC and RIC groups, respectively. The incidences of grades II-IV and III-IV acute graft-versus-host disease (GVHD) were 18% and 8% in the MAC group, and 14% and 5% in the RIC group, respectively. Those of all grade and moderate to severe chronic GVHD at 2-year were 36% and 20% in the MAC group, and 27% and 20% in the RIC group, respectively. Overall survival (OS), EFS, nonrelapse mortality, and relapse rate at 2-year were 68%, 54%, 10%, and 36% in the MAC group, and 44%, 35%, 20%, and 45% in the RIC group, respectively. Notably, 83% and 86% of patients who survived without relapse stopped immunosuppressant at 2-year in the MAC and RIC groups, respectively. Our results indicate that both MAC and RIC are valid options for PTCy-haploPBSCT for adults with hematological malignancies.