AI Article Synopsis
- Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only cure for marrow failure caused by dyskeratosis congenita (DC), with limited data on transplants from alternative donors.
- A boy with DC and severe aplastic anemia successfully underwent haploidentical T-cell depleted HSCT using a reduced-intensity conditioning regimen, achieving engraftment without toxicity or graft-versus-host disease (GVHD).
- His recovery featured a complication of EBV reactivation, which was treated effectively, and after 26 months, he shows complete chimerism, normal blood counts, and no signs of GVHD or pulmonary issues.
Article Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only cure for marrow failure associated with dyskeratosis congenita (DC). Data on transplants from alternative donors are limited. We describe a boy with DC and severe aplastic anemia who underwent haploidentical T-cell depleted HSCT using a reduced-intensity conditioning regimen. He underwent engraftment without toxicity or GVHD. His posttransplant course was complicated by EBV reactivation, treated with rituximab and EBV-specific T lymphocytes. After 26 months, he is in complete chimerism, with normal blood count and no sign of GVHD or pulmonary dysfunction. To the best of our knowledge, this is the first report of DC successfully treated with allogeneic HSCT from a haploidentical family donor.
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| http://dx.doi.org/10.1097/MPH.0000000000000283 | DOI Listing |
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