Unrelated hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia and iron overload.
CDA is a heterogeneous group of disorders that result in morphologically abnormal erythroid maturation and ineffective erythropoiesis. Curative therapy for CDA focuses on the use of HSCT using fully matched sibling donors. This is the first report of a Type II CDA patient with severe iron overload who was successfully treated with HSCT using a HLA-matched unrelated donor after aggressive chelation therapy. Given the challenges of HSCT in any patient with CDA and severe iron overload, the role of novel approaches to iron chelation and HSCT is discussed.
Division of Hematology, CHOC Children's Hospital and UC Irvine Medical Center, Orange, CA 92868, USA. dbuchbinder@choc.org
David
D
Buchbinder
Diane
D
Nugent
Dan
D
Vu
Amit
A
Soni
Jill
J
Stites
Loan
L
Hsieh
Geetha
G
Puthenveetil
D000742:Anemia, Dyserythropoietic, Congenital / Q000150:complications*; D001565:Benzoates / Q000494:pharmacology; D016026:Bone Marrow Transplantation / Q000379:methods; D002675:Child, Preschool; D000077588:Deferasirox; D003676:Deferoxamine / Q000494:pharmacology; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D007501:Iron / Q000737:chemistry; D007502:Iron Chelating Agents / Q000494:pharmacology; D019190:Iron Overload / Q000150:complications*; D008297:Male; D014230:Triazoles / Q000494:pharmacology; D061349:Unrelated Donors
D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D003676:Deferoxamine; D000077588:Deferasirox