Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV, open-label, single-centre trial.
We report the results of an open-label, single-centre phase 4 clinical trial evaluating haploidentical haematopoietic stem cell transplantation (Haplo-HSCT) in patients with transfusion-dependent thalassaemia (TDT). A total of 134 patients with TDT were enrolled; the median age was 8.7 years (interquartile range [IQR],...