Nonmyeloablative allogeneic hematopoietic stem cell transplantation for congenital sideroblastic anemia.
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.
Medeiros
B C
BC
Department of Medicine, University of Colorado Health Sciences Center, Denver, CO, USA.
Kolhouse
J F
JF
Cagnoni
P J
PJ
Ryder
J
J
Nieto
Y
Y
Rabinovitch
R
R
Shpall
E J
EJ
Bearman
S I
SI
Jones
R B
RB
McSweeney
P A
PA
D000328:Adult; D000756:Anemia, Sideroblastic / Q000151:congenital* / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D017809:Fatal Outcome; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D014916:Whole-Body Irradiation
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D014740:Vidarabine; C024352:fludarabine