Article
Successful hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia type II.
Pediatric transplantation - 1 Jun 2014
Unal Sule, Russo Roberta, Gumruk Fatma, Kuskonmaz Baris, Cetin Mualla, Sayli Tulin, Tavil Betul, Langella Concetta, Iolascon Achille, Uckan Cetinkaya Duygu
Abstract excerpt
CDA are a group of inherited, rare diseases that are characterized by dyserythropoiesis and ineffective erythropoiesis associated with transfusion dependency in approximately 10% of cases. For these latter patients, the only curative treatment is HSCT. There are very limited data on HSCT experience in this rare disease. Herein, we report a five-yr six-month-old girl with compound heterozygous mutations in SEC23B...
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