Oevermann, Lena and Schulte, Johannes H. and Hundsdoerfer, Patrick and Hakimeh, Dani and Kogel, Friederike and Lang, Peter and Corbacioglu, Selim and Eggert, Angelika and Sodani, Pietro (2019) HLA-haploidentical hematopoietic stem cell transplantation in pediatric patients with hemoglobinopathies: current practice and new approaches. BONE MARROW TRANSPLANTATION, 54. pp. 743-748. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
We review current approaches in HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for pediatric patients with hemoglobinopathies with a focus on recent developments using TCR alpha/beta+/CD19+ depleted grafts in patients with beta-thalassemia major (TM) or sickle cell disease (SCD) in two European transplant units. Eleven TM and three SCD patients (Roma cohort) received a preparative regimen consisting of busulfan/thiotepa/cyclophosphamide/ATG preceded by fludarabine/hydroxyurea/azathioprine. The preparative regimen for 5 SCD patients included treosulfan/thiotepa/fludarabine/ATG (Berlin pilot cohort). All grafts were PBSC engineered by TCR-alpha/beta+/CD19+ depletion. In both cohorts, rates for graft failure, treatment related mortality (TRM) and GvHD were encouraging. Overall survival (OS) and disease-free survival (DFS) in the Roma cohort were 84 and 69%, respectively, while OS and DFS are 100% in the Berlin cohort. Immune reconstitution was satisfactory. Although asymptomatic viral reactivation was common, no severe viral infection occured. These data confirm that TCR-alpha/beta+/CD19+ depletion is a well-suited haplo-HSCT strategy for children with hemoglobinopathies. We discuss the results in the context of additional optimization strategies and introduce our concepts for multicenter trial protocols in Germany.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; T-CELLS; POSTTRANSPLANTATION CYCLOPHOSPHAMIDE; CONDITIONING REGIMEN; IMPROVED OUTCOMES; REDUCED-TOXICITY; IMMUNE RECOVERY; THALASSEMIA; CHILDREN; DONORS; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Petra Gürster |
| Date Deposited: | 02 Apr 2020 06:06 |
| Last Modified: | 02 Apr 2020 06:06 |
| URI: | https://pred.uni-regensburg.de/id/eprint/26478 |
Actions (login required)
 |
View Item |
