Blood: Prognosis for hematopoietic cell transplantation treatment for Wiskott-Aldrich syndrome

Aldridge syndrome (WAS) is an X-chromosome chain disease caused by mutations in the WAS gene that can lead to platelet reduction, eczema, recurrent infections, autoimmune diseases and malignant tumorsHematopoietic Cell Transplantation (HCT) is the main treatment designed to correct potential immunodeficiency and platelet reductionthe prognosis of HCT improved over time, especially in patients with siblings and unrelated donors that matched HLAThis study reported on the prognosis of 129 WAS patients treated with HCT at 29 combination centers for primary immunodeficiency therapy from 2005 to 2015The median age at HCT is 1.2 yearsMost patients (65%) underwent bone marrow removal-based conditioningMedian follow-up was 4.5 years, with an overall 5-year survival rate (OS) of 91%The five-year OS performed HCT was significantly better (94 percent vs 66 percent) compared to patients who performed HCT at the age of 5OS is very good (90%) even in cord blood receptors, regardless of donor typeThe regulatory intensity does not affect OS, but is related to hcT post-donor T-cell and myelin cell transplantationIn particular, patients who received a low-intensity treatment based on fluodabin/mefalenarewered to be 50% more likely to have donor bone marrow chinos in the early stages of HCT, the platelet count of recipients performing whole myelin cell transplantation was higher than those with a low proportion (5-49%)myelin cell transplantall in all, was the HCT prognosis of WAS has improved from previous reports since 2005A high proportion of donor myelin cell transplantation is essential for platelet reconstruction