医学
环磷酰胺
造血干细胞移植
地中海贫血
调理疗法
内科学
养生
疾病
免疫系统
胃肠病学
免疫学
化疗
作者
Gaurav Kharya,Atish Bakane,Archana Rauthan
摘要
Abstract Hematopoietic stem cell transplant (HSCT) is currently the only curative option for thalassemia major (TM) and sickle cell disease (SCD). We report our experience of using pretransplant immune suppression (PTIS), augmented Johns Hopkins conditioning, and posttransplant cyclophosphamide (PTCy) as graft‐versus‐host disease (GvHD) prophylaxis for matched unrelated donor (MUD) transplant in TM/SCD. At a median follow‐up of 307.5 days (range 251‐395), all patients (three TM, one SCD) are alive and disease free. MUD HSCT with PTIS, augmented Johns Hopkins conditioning, and PTCy as GvHD prophylaxis is a promising way of treating patients with hemoglobinopathies with low regimen‐related toxicity (RRT), no risk of graft failure (GF) and minimal GvHD rates.
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