Choudhary Dharma, Doval Divya, Sharma Sanjeev Kumar, Khandelwal Vipin, Setia Rasika, Handoo Anil
Centre for Bone Marrow Transplant, BLK Superspeciality Hospital, Pusa Road, New Delhi, India.
Blood Cell Ther. 2021 Jan 15;4(2):29-34. doi: 10.31547/bct-2020-014. eCollection 2021 May 25.
We report herein haplo-identical hematopoietic stem cell transplantation (haplo-HSCT) by T-cell replete graft infusion, with post-transplant cyclophosphamide (PTCy) in patients with hemoglobinopathies. Patients received a conditioning regimen consisting of either busulfan, fludarabine, cyclophosphamide, with antithymocyte globulin or Thiotepa, antithymocyte globulin, fludarabine, cyclophosphamide, and TBI. The median follow-up period was 14.3 months (range, 1-63 months). Overall survival (OS) and disease-free survival (DFS) were 80% and 62.8%, respectively. Incidence of secondary graft failure was 14%. Incidences of acute graft-versus-host disease (aGvHD) and chronic graft-versus-host disease (cGvHD) were 22.5% and 20%, respectively. Cytomegalovirus (CMV) reactivation was observed in 42.5% of cases. The 100-day mortality rate was 20%, with sepsis and aGvHD being the predominant causes of death.
我们在此报告采用富含T细胞的移植物输注并联合移植后环磷酰胺（PTCy）对血红蛋白病患者进行单倍体相合造血干细胞移植（haplo-HSCT）的情况。患者接受了由白消安、氟达拉滨、环磷酰胺联合抗胸腺细胞球蛋白组成的预处理方案，或塞替派、抗胸腺细胞球蛋白、氟达拉滨、环磷酰胺及全身照射（TBI）组成的预处理方案。中位随访期为14.3个月（范围1 - 63个月）。总生存率（OS）和无病生存率（DFS）分别为80%和62.8%。继发性移植物失败发生率为14%。急性移植物抗宿主病（aGvHD）和慢性移植物抗宿主病（cGvHD）的发生率分别为22.5%和20%。42.5%的病例观察到巨细胞病毒（CMV）再激活。100天死亡率为20%，脓毒症和aGvHD是主要死亡原因。
