Current and future preparative regimens for bone marrow transplantation in thalassemia.


Journal Article

Preparative regimens for marrow allografts in thalassemia have two objectives. One is eradication of diseased marrow and the other suppression of host-versus-graft (HVG) reactions so that the allograft survives. A common regimen to accomplish these goals has combined high-dose busulfan with cyclophosphamide. Postgrafting immunosuppression with cyclosporine/methotrexate has been used for GVHD prevention. Some patients may die from regimen-related toxicity. Overall event-free survival is 75%. Occasional patients have become mixed donor/host hematopoietic chimeras and, yet, disease symptoms have abated. This has raised the possibility of developing safer and less toxic transplant programs that result in stable mixed hematopoietic chimerism. We have devised such a program in dogs consisting of a nonlethal dose of total body irradiation (200 cGy) before and a novel combination of mycophenolate mofetil and cyclosporine after transplant. Mixed donor/host chimerism (> or = 50% donor cells in all lineages) has persisted for > 80 weeks, even though immunosuppression was discontinued after five weeks.

Full Text

Duke Authors

Cited Authors

  • Storb, R; Yu, C; Deeg, HJ; Georges, G; Kiem, HP; Mcsweeney, PA; Nash, RA; Sandmaier, BM; Sullivan, KM; Wagner, JL; Walters, MC

Published Date

  • June 30, 1998

Published In

Volume / Issue

  • 850 /

Start / End Page

  • 276 - 287

PubMed ID

  • 9668549

Pubmed Central ID

  • 9668549

International Standard Serial Number (ISSN)

  • 0077-8923

Digital Object Identifier (DOI)

  • 10.1111/j.1749-6632.1998.tb10484.x


  • eng

Conference Location

  • United States