Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease

Bone Marrow Transplantation
Koen van BesienR Hoffman

Abstract

Although allogeneic transplantation can be curative for patients with sickle cell disease, the toxicity of conditioning regimens has precluded its use in adults with significant end-organ damage. Newer conditioning regimens have been developed that are less toxic and that may broaden the applicability of allogeneic transplantation in this disorder. We report two adults with end-stage sickle cell disease, who underwent allogeneic transplantation from an HLA-identical sibling donor after conditioning with fludarabine/melphalan and ATG. Both patients had been extensively transfused and one had multiple RBC antibodies. One of the patients also had end-stage renal disease, and was dialysis dependent. Engraftment occurred promptly in both patients. Both achieved 100% donor chimerism and both were free of pain crises after transplant. The first patient died of a respiratory failure related to chronic graft-versus-host disease (GVHD) on day 335 after transplantation. The second patient developed severe gastro-intestinal GVHD and TTP and died on day 147 after transplantation. Conditioning with fludarabine/melphalan and ATG followed by allogeneic stem cell transplantation resulted in prompt and reliable engraftment in adults with end-sta...Continue Reading

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Related Concepts

Fludarabine
Anemia, Sickle Cell
Graft Survival
Graft-vs-Host Disease
Immunosuppressive Agents
Son
Transplantation, Homologous
Vira-A
Fatal Outcome
Transplantation Chimera

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