Nonmyeloablative allogeneic hematopoietic stem cell transplantation for congenital sideroblastic anemia.


Journal Article

Congenital sideroblastic anemia (CSA) is a dyserythropoietic disorder that leads to transfusion dependency and subsequent iron overload. Nonmyeloablative allogeneic hematopoietic stem cell transplantation (NST) was performed for a patient with CSA, who had contraindications to conventional allografting. Conditioning was fludarabine, low-dose total body irradiation and antithymocyte globulin, followed by peripheral blood stem cell transplant. Cyclosporine and mycophenolate mofetil were used for graft-versus-host disease prophylaxis. Complete donor chimerism was observed day +131. Early after transplant, the patient became transfusion independent, allowing a regular phlebotomy program. On day +190, refractory lactic acidosis followed by fatal cardiovascular collapse developed, without evidence of infection. Data from this case demonstrates that NST may correct the erythropoietic defect of CSA.

Full Text

Duke Authors

Cited Authors

  • Medeiros, BC; Kolhouse, JF; Cagnoni, PJ; Ryder, J; Nieto, Y; Rabinovitch, R; Shpall, EJ; Bearman, SI; Jones, RB; McSweeney, PA

Published Date

  • June 2003

Published In

Volume / Issue

  • 31 / 11

Start / End Page

  • 1053 - 1055

PubMed ID

  • 12774059

Pubmed Central ID

  • 12774059

International Standard Serial Number (ISSN)

  • 0268-3369

Digital Object Identifier (DOI)

  • 10.1038/sj.bmt.1704038


  • eng

Conference Location

  • England