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Successful nonmyeloablative bone marrow transplantation in a corticosteroid-resistant infant with Diamond–Blackfan anemia

Bone Marrow Transplantation volume 34, pages 371–372 (2004)Cite this article

Nonmyeloablative bone marrow transplantation is effective in the treatment of autoimmune diseases and hemoglobinopathies.1, 2, 3, 4 Diamond–Blackfan anemia (DBA) is a childhood disorder characterized by normochromic-macrocytic anemia, reticulocytopenia and normocellular bone marrow with a selective deficiency of erythroid progenitors.5 Most patients initially respond to corticosteroids, but about 60% of patients require chronic red cell transfusions and die from hemosiderosis in the second or third decade of life. Allogeneic bone marrow transplantation (BMT) is an effective therapy for steroid-resistant, transfusion-dependent DBA with survival rates of 60–80% in patients transplanted from HLA-matched related donors.6, 7

A conditioning regimen based on fludarabine and low-dose TBI (2 Gy) permits engraftment and full donor chimerism in both malignant and nonmalignant diseases, and can potentially avoid complications such as retarded growth and development, cataract formation and sterility in children.2, 8 We chose this regimen for a transfusion-dependent, 19-month-old boy with DBA.

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Authors and Affiliations

  1. Bone Marrow Transplantation Unit, Real Hospital Português, Recife, PE, Brazil

    M Ostronoff, R Florencio, G Campos, S Arruda, C Matias, M Florencio, M Domingues, A P Souto Maior, A Sucupira, R Calixto, C Tagliari & K Matias

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  1. M Ostronoff
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  2. R Florencio
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  3. G Campos
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  4. S Arruda
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  5. C Matias
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  6. M Florencio
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  7. M Domingues
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  8. A P Souto Maior
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  9. A Sucupira
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  11. C Tagliari
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  12. K Matias
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Ostronoff, M., Florencio, R., Campos, G. et al. Successful nonmyeloablative bone marrow transplantation in a corticosteroid-resistant infant with Diamond–Blackfan anemia. Bone Marrow Transplant 34, 371–372 (2004). https://doi.org/10.1038/sj.bmt.1704592

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