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: Velu Nair, Satyaranjan Das, Ajay Sharma
• Journal: Indian pediatrics (Indian Pediatr) Vol. 46 Issue 3 Pg. 241-3 (Mar 2009) ISSN: 0019-6061 [Print] India
• PMID: 19213990 (Publication Type: Journal Article)

Topics

• Adolescent
• Anemia, Diamond-Blackfan (complications, therapy)
• Anemia, Dyserythropoietic, Congenital (therapy)
• Child
• Child, Preschool
• Fanconi Anemia (therapy)
• Female
• Genetic Diseases, Inborn (therapy)
• Graft Rejection
• Hematopoietic Stem Cell Transplantation
• Humans
• Male
• Muscular Dystrophy, Duchenne (complications, therapy)
• Reoperation
• Transplantation, Homologous
• beta-Thalassemia (therapy)