title: Hematopoietic stem cell transplantation in children with genetic defects.
abstract: Seventeen children (mean age: 7.2 years) with genetic defects involving hematopoietic cell production or function, underwent 19 allogeneic stem cell transplantations from HLA identical siblings. Twelve children were suffering from thalassemia major; 2 from Diamond Blackfan anemia; 2 from Fanconi anemia and 1 from congenital dyserythropoietic anemia. The disease free survival was 77% with a mean follow up of 36 months. The major complications were graft versus host disease, veno-occlusive disease, CMV infection and hemorrhage. One case each of thalassaemia major and Fanconi anemia rejected the graft after 1 year and 11 months, respectively. Both patients were successfully transplanted second time from the same donor with some modification in the conditioning regimen and stem cell source.
authors.affiliation: Department of Haematology and Bone Marrow Transplantation, Army Hospital Research and Referral, Delhi Cantonment, Delhi, India. nairvelu2000@yahoo.com
authors.forename: Velu
authors.initials: V
authors.lastname: Nair
authors.forename: Satyaranjan
authors.initials: S
authors.lastname: Das
authors.forename: Ajay
authors.initials: A
authors.lastname: Sharma
mesh_terms: D000293:Adolescent; D029503:Anemia, Diamond-Blackfan / Q000150:complications / Q000628:therapy; D000742:Anemia, Dyserythropoietic, Congenital / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D005199:Fanconi Anemia / Q000628:therapy; D005260:Female; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D006084:Graft Rejection; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000150:complications / Q000628:therapy; D012086:Reoperation; D014184:Transplantation, Homologous; D017086:beta-Thalassemia / Q000628:therapy