National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Department of Hematology, The Affiliated Huai'an Hospital of Xuzhou Medical University and The Second People's Hospital of Huai'an, No 62, Huaihai Road (S.), Huai'an, China.
Bone Marrow Transplant. 2020 Oct;55(10):2017-2025. doi: 10.1038/s41409-020-0874-9. Epub 2020 Mar 26.
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.
我们分析了 146 例重型再生障碍性贫血（SAA）患者的结果，这些患者于 2011 年 9 月至 2017 年 12 月期间接受了单倍体相合造血干细胞移植（haplo-HSCT）和无关脐带血（UCB）联合治疗。117 例患者作为一线治疗进行移植。7 例患者早期死亡，在可评估的 139 例患者中，1 例患者发生原发性移植物失败（GF），而其他 138 例患者成功实现了单倍体供者植入；此外，3 例患者发生继发性 GF。6 例患者出现血小板延迟恢复，3 例患者出现血小板 GF。骨髓和血小板植入的中位时间分别为 11（范围：9-28）天和 15（范围：9-330）天。中位随访 40（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、无失败生存率分别为 81.4±3.3%和 69.2±3.9%。这些令人鼓舞的初步结果表明，haplo-HSCT 联合 UCB 输注对于没有匹配供体的 SAA 患者是一种可行的选择。
