This work represents the most extensive data collection so far made regarding the hematopoietic stem cell transplantation in patients with Wiskott-Aldrich syndrome, a rare genetic disorder linked to the X chromosome that causes severe deficits on platelets as well as on of the immune system cells. The project has involved 12 of the most important American and European transplant centers, allowing the collection of data on transplantation and the subsequent follow-up of 194 patients transplanted since 1980. From this enormous amount of data it was detected that the improvement of the techniques used for transplantation have led to a significant increase in survival over the past ten years for those types of intervention considered more at risk. It also allowed for the first time to statistically demonstrate that there are risks associated with an incomplete engraftment of donor cells, as well as the limits beyond which the effective engraftment for a complete cure of the symptoms linked to the disease could be detected.
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