Nonmyeloablative allogeneic hematopoietic stem cell transplantation for congenital sideroblastic anemia

Bone Marrow Transplant. 2003 Jun;31(11):1053-5. doi: 10.1038/sj.bmt.1704038.

Abstract

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.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Anemia, Sideroblastic / congenital*
  • Anemia, Sideroblastic / therapy*
  • Antilymphocyte Serum / therapeutic use
  • Fatal Outcome
  • Graft vs Host Disease / prevention & control
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Transplantation Conditioning / methods
  • Transplantation, Homologous
  • Vidarabine / analogs & derivatives*
  • Vidarabine / therapeutic use
  • Whole-Body Irradiation

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Vidarabine
  • fludarabine