Success of bone marrow transplantation in congenital Diamond‐Blackfan anaemia: A case report
- 1 February 1987
- journal article
- case report
- Published by Wiley in European Journal of Haematology
- Vol. 38 (2), 204-206
- https://doi.org/10.1111/j.1600-0609.1987.tb01163.x
Abstract
A 6-yr-old girl with congenital corticosteroid-resistant pure red cell aplasia was treated with bone marrow transplant from her HLA-identical, MLC-unreactive sister in November 1984 following conditioning with busulfan and cyclophosphamide. Full engraftment was obtained and the patient at 21 months post-transplant is in excellent clinical condition maintaining normal red cell counts. We conclude that BMT should be considered as a therapy for at least the most severe cases of Diamond-Blackfan anaemia resistant to corticosteroids. Successful outcome of this therapy provides an argument for the stem cell origin of this disorder.Keywords
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