Instituto Nacional de Cancer, Transplante de Medula Ossea, Rio de Janeiro, RJ, Brazil.
Universidade Federal do Paraná, Transplante de Medula Ossea, Curitiba, PR, Brazil.
Biol Blood Marrow Transplant. 2020 Sep;26(9):e222-e226. doi: 10.1016/j.bbmt.2020.05.023. Epub 2020 Jun 3.
Patients with severe aplastic anemia (SAA) who fail immunosuppressive therapy have a dismal prognosis. Hematopoietic stem cell transplantation (HSCT) from an unrelated donor (URD) is one of the most effective treatment options. Two institutions have independently adopted a post-transplantation cyclophosphamide (PTCy) approach for patients with SAA undergoing HSCT from a URD. Thirteen patients were included, 11 of whom had been treated with immunosuppressive therapy. Eight patients had a mismatched URD. All patients were conditioned with fludarabine, cyclophosphamide, and total body irradiation, in various dosage combinations. PTCy was given at a dose of 100 mg/kg. Two patients died, and overall survival was 85% at 2 years. All patients engrafted, but 1 patient developed secondary graft failure. Of the 11 patients alive after 2 years, 9 had complete donor chimerism. All surviving patients were transfusion-independent. Ten patients (77%) had cytomegalovirus reactivation, and 2 patients had more than 1 reactivation. No Epstein-Barr virus reactivation or post-transplantation lymphoproliferative disease was observed. Four patients had mild hemorrhagic cystitis. In summary, our findings show that PTCy is a promising treatment for patients with SAA undergoing URD HSCT.
对于重型再生障碍性贫血（SAA）患者，免疫抑制治疗失败预后较差。异基因造血干细胞移植（HSCT）是最有效的治疗选择之一。两个机构均独立采用了移植后环磷酰胺（PTCy）方案治疗接受异基因供体 HSCT 的 SAA 患者。共纳入 13 例患者，其中 11 例接受了免疫抑制治疗。8 例患者的供体不匹配。所有患者均采用氟达拉滨、环磷酰胺和全身照射，不同剂量组合进行预处理。PTCy 的剂量为 100mg/kg。2 例患者死亡，2 年总生存率为 85%。所有患者均植活，但 1 例发生继发性移植物失败。2 年后存活的 11 例患者中，9 例完全供者嵌合。所有存活患者均无需输血。10 例（77%）患者出现巨细胞病毒再激活，2 例患者发生 1 次以上再激活。未观察到 EBV 再激活或移植后淋巴组织增生性疾病。4 例患者发生轻度出血性膀胱炎。总之，我们的研究结果表明，PTCy 是治疗接受异基因供体 HSCT 的 SAA 患者的一种有前途的治疗方法。
