Abstract |
Children and adolescents with homozygous beta-thalassemia can be cured by transplantation of normal stem cells after eradication of the thalassemic hematopoietic system. In an attempt to achieve durable engraftment and to minimize regimen-related toxicity (RRT), we have initiated a fludarabine-based pilot protocol not containing cyclophosphamide. Between 1999 and 2004, five children with beta-thalassemia major were enrolled. Median age at transplantation was 11.5 years (range 4-14 years). Three patients received conditioning with fludarabine (30 mg/m2/day x 6), oral busulfan (3.5 mg/kg/day x 4), and ATG rabbit Fresenius (10 mg/kg/day x 4). Two children received intravenous busulfan instead of oral busulfan at a dose of 2 x 1.4 mg/kg/day x 4 days. All children were transplanted with a fresh bone marrow graft from an HLA-identical sibling. Mean cell doses given were 3.7 x 10(8) nucleated cells/kg BW (range 2.4-6.2 x 10(8)/kg). Overall, 5/5 patients achieved donor engraftment and are alive and well. No GVHD exceeding grade I was observed, and 2/5 children maintained donor chimerism at 100%. One patient maintains mixed hematopoietic donor chimerism being between 94 and 97% nearly 5 years after transplant.
|
Authors | M Sauer, C Bettoni, M Lauten, A Ghosh, K Rehe, L Grigull, A Beilken, K Welte, K W Sykora |
Journal | Bone marrow transplantation
(Bone Marrow Transplant)
Vol. 36
Issue 5
Pg. 383-7
(Sep 2005)
ISSN: 0268-3369 [Print] England |
PMID | 15995711
(Publication Type: Journal Article)
|
Chemical References |
|
Topics |
- Adolescent
- Bone Marrow Transplantation
(methods)
- Case-Control Studies
- Child
- Child, Preschool
- Female
- Graft vs Host Disease
(prevention & control)
- Histocompatibility Testing
- Humans
- Immunosuppressive Agents
(administration & dosage)
- Male
- Transplantation Chimera
- Transplantation Conditioning
(methods)
- Transplantation, Homologous
- beta-Thalassemia
(therapy)
|