Unrelated hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia and iron overload.

Abstract

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.

• Authors: David Buchbinder, Diane Nugent, Dan Vu, Amit Soni, Jill Stites, Loan Hsieh, Geetha Puthenveetil
• Journal: Pediatric transplantation (Pediatr Transplant) Vol. 16 Issue 3 Pg. E69-73 (May 2012) ISSN: 1399-3046 [Electronic] Denmark
• PMID: 21108711 (Publication Type: Case Reports, Journal Article)
• Copyright: © 2010 John Wiley & Sons A/S.

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Iron
• Deferoxamine
• Deferasirox

Topics

• Anemia, Dyserythropoietic, Congenital (complications)
• Benzoates (pharmacology)
• Bone Marrow Transplantation (methods)
• Child, Preschool
• Deferasirox
• Deferoxamine (pharmacology)
• Female
• Hematopoietic Stem Cell Transplantation (methods)
• Humans
• Infant
• Iron (chemistry)
• Iron Chelating Agents (pharmacology)
• Iron Overload (complications)
• Male
• Triazoles (pharmacology)
• Unrelated Donors