Successful treatment of an infant with CDA type II by intrauterine transfusions and postnatal stem cell transplantation. Issue 4 (3rd October 2013)

• Record Type: Journal Article
• Authors: Braun, M.; Wölfl, M.; Wiegering, V.; Winkler, B.; Ertan, K.; Bald, R.; Schwarz, K.; Heimpel, H.; Eyrich, M.; Schlegel, P.G.
• Is Part Of: Pediatric blood & cancer. Volume 61:Issue 4(2014:Apr.)
• Page Start: 743
• Page End: 745
• Publication Date: 2013-10-03
• Subjects: Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.24786
• Languages: English
• ISSNs: 1545-5009

Abstract

<abstract abstract-type="main" xml:lang="en"> <title>Abstract</title> <sec id="pbc24786-sec-0001" sec-type="section"> <p>Congenital dyserythropoietic anemias are rare hematological disorders leading to ineffective erythropoiesis with chronic anemia, complicated by iron overload. Here we present a remarkable clinical course of an infant with CDA type II who first presented as a severe fetal hydrops, requiring serial <italic>intrauterine</italic> red cell transfusions. While postnatal transfusion dependency persisted, the patient was successfully transplanted with a myeloablative conditioning regimen and peripheral blood stem cells of a matched donor. We believe that allogeneic HSCT is a reasonable therapeutic approach for patients with very severe CDA, even if only a matched unrelated donor is available. Pediatr Blood Cancer 2014;61:743–745. © 2013 Wiley Periodicals, Inc.</p> </sec> </abstract>