Successful use of reduced-intensity conditioning and matched-unrelated hematopoietic stem cell transplant in a child with Diamond-Blackfan anemia and cirrhosis.

Abstract

For patients with DBA who are transfusion dependent, HSCT is the only cure. Chronic transfusions can lead to cirrhosis secondary to iron overload, making them poor candidates for myeloablative HSCT. RIC regimens are associated with lower morbidity and mortality compared to myeloablative regimens, but use of RIC in DBA has been limited. Here we present a 14-yr-old girl with DBA and multiple comorbidities including liver cirrhosis, who underwent MUD HSCT utilizing a RIC regimen that is novel to this condition. She tolerated the regimen well, and at 21 months, she remains transfusion independent with chimerisms at 99%.

• Authors: Justin M Asquith, Jessica Copacia, Mark J Mogul, Rajinder P S Bajwa
• Journal: Pediatric transplantation (Pediatr Transplant) Vol. 19 Issue 6 Pg. E157-9 (Sep 2015) ISSN: 1399-3046 [Electronic] Denmark
• PMID: 26103586 (Publication Type: Case Reports, Journal Article)
• Copyright: © 2015 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

Topics

• Adolescent
• Anemia, Diamond-Blackfan (complications, therapy)
• Erythrocyte Transfusion (adverse effects)
• Female
• Hematopoietic Stem Cell Transplantation (methods)
• Humans
• Liver Cirrhosis (etiology)
• Transplantation Conditioning (methods)