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| Allografting |
| Transfusion-dependent congenital dyserythropoietic anemia type I successfully treated with allogeneic stem cell transplantation |
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| M Ayas, A Al-Jefri, A Baothman, M Al-Mahr, M M Mustafa, S Khalil, M Karaoui and H Solh |
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Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center (KFSH&RC), Riyadh, Saudi Arabia
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Correspondence to: Dr M Ayas, Department of Oncology, King Faisal Specialist Hospital and Research Center, MBC 53, PO Box 3354, Riyadh 11211, Saudi Arabia |
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| Abstract |
| Until recently, therapy for patients with severe congenital dyserythropoietic anemia (CDA) has been limited to blood transfusions and chelation therapy. Three children with transfusion-dependent CDA type I underwent allogeneic stem cell transplantation (SCT) from matched sibling donors. Conditioning was with cyclophosphamide 50 mg/kg/day for 4 days, busulphan 4 mg/kg/day for 4 days, and antithymocyte globulin (ATG) 30 mg/kg for four doses pre-SCT. All patients engrafted and are alive, and transfusion independent. To our knowledge, this is the first report of successful SCT in the management of CDA type I. Bone Marrow Transplantation (2002) 29, 681-682. DOI: 10.1038/sj/bmt/1703526 |
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| Keywords |
| stem cell transplantation; dyserythropoietic anemia; congenital anemia; ineffective erythropoiesis; interferon |
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| Received 16 October 2001; accepted 24 January 2002 |
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| April (2) 2002, Volume 29, Number 8, Pages 681-682 |
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