Department of Hematology, Hokkaido University Hospital, Sapporo, Japan.
Japanese Data Center for Hematopoietic Cell Transplantation, Nagakute, Japan.
Bone Marrow Transplant. 2022 Nov;57(11):1681-1688. doi: 10.1038/s41409-022-01770-y. Epub 2022 Aug 20.
HLA-haploidentical stem cell transplantation using post-transplant cyclophosphamide (PTCy-haplo) and umbilical cord blood transplantation (UCBT) are alternative to HLA-matched stem cell transplantation. We conducted a matched-pair analysis of PTCy-haplo and UCBT using the Japanese registry data. We identified 136 patients aged between 16 and 69 years who received PTCy-haplo as their first transplantation for acute leukemia or myelodysplastic syndromes. Control group included 408 UCBT recipients selected to match the PTCy-haplo group. Overall and relapse-free survival probabilities at 2 years were comparable between the PTCy-haplo and UCBT groups: 55% vs. 53% for overall survival (p = 0.46), and 47% vs. 48% for relapse-free survival (p = 0.79), respectively. The cumulative incidence of relapse was significantly higher (43% vs. 29%, respectively, p = 0.006), while the cumulative incidence of non-relapse mortality (NRM) was significantly lower (9% vs. 23%, respectively, p < 0.001) in the PTCy-haplo group. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was lower in the PTCy-haplo group compared to the UCBT group (29% vs. 41%, respectively, p = 0.016), while those of grade III-IV acute GVHD and chronic GVHD were not statistically different between the two groups. Our results suggest that both PTCy-haplo and UCBT are viable alternatives to HLA-matched stem cell transplantation.
HLA 单倍体相合造血干细胞移植（PTCy-haplo）和脐带血移植（UCBT）是 HLA 匹配造血干细胞移植的替代方案。我们使用日本注册数据进行了 PTCy-haplo 和 UCBT 的配对分析。我们确定了 136 名年龄在 16 岁至 69 岁之间的患者，他们接受 PTCy-haplo 作为急性白血病或骨髓增生异常综合征的首次移植。对照组包括 408 名选择与 PTCy-haplo 组匹配的 UCBT 受者。2 年时，PTCy-haplo 和 UCBT 组的总生存率和无复发生存率相当：总生存率分别为 55%和 53%（p=0.46），无复发生存率分别为 47%和 48%（p=0.79）。PTCy-haplo 组的复发累积发生率明显较高（43%比 29%，p=0.006），而非复发死亡率（NRM）累积发生率明显较低（9%比 23%，p<0.001）。与 UCBT 组相比，PTCy-haplo 组的 II-IV 级急性移植物抗宿主病（GVHD）累积发生率较低（29%比 41%，p=0.016），但 III-IV 级急性 GVHD 和慢性 GVHD 的发生率在两组间无统计学差异。我们的研究结果表明，PTCy-haplo 和 UCBT 都是 HLA 匹配造血干细胞移植的可行替代方案。
