Haematopoietic stem cell transplantation (HSCT) was an effective and definitive treatment in 14 patients with deficiency of adenosine deaminase 2 (DADA2), a monogenic autoinflammatory vasculopathy. All patients reported resolution of immunological and haematological phenotype, with no new vascular events at an average of 18 months follow-up, and adenosine deaminase 2 activity normalized as early as 14 days post-HSCT in those patients tested (n = 7). Adverse events were reported in 11 of the 14 patients (four incidents of cytopenia and seven incidents of moderate or acute graft-versus-host disease).
References
Hashem H. et al. Hematopoietic stem cell transplantation rescues the hematological, immunological and vascular phenotype in DADA2. Blood http://dx.doi.org/10.1182/blood-2017-07-798660 (2017)
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Collison, J. Stem cell transplantation for DADA2. Nat Rev Rheumatol 13, 694 (2017). https://doi.org/10.1038/nrrheum.2017.175
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DOI: https://doi.org/10.1038/nrrheum.2017.175
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