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Hematopoietic stem cell transplantation rescues the hematological, immunological and vascular phenotype in DADA2

Blood Oct 07, 2017

Hashem H, et al. - In this study, researchers analyzed a cohort of patients from 6 countries who received hematopoietic stem cell transplantation (HSCT) for deficiency of adenosine deaminase 2 (DADA2). In these patients, HSCT proved an effective and definitive treatment for DADA2. Thereby suggesting that HSCT have the potential for curing the immunological, hematological, and vascular phenotype of DADA2 with 100% survival at median follow-up of 18 months.
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