| Title: |
Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia : A prospective multicentre trial of the I-BFM Study Group |
| Authors: |
Pichler, Herbert; Sedlacek, Petr; Meisel, Roland; Beier, Rita; Faraci, Maura; Kalwak, Krzysztof; Ifversen, Marianne; Müller, Ingo; Stein, Jerry; Vettenranta, Kim; Kropshofer, Gabriele; Kolenova, Alexandra; Karlhuber, Susanne; Glogova, Evgenia; Poetschger, Ulrike; Peters, Christina; Suttorp, Meinolf; Matthes-Leodolter, Susanne; Balduzzi, Adriana |
| Contributors: |
Clinicum; Children's Hospital; Lastentautien yksikkö; HUS Children and Adolescents |
| Publisher Information: |
John Wiley and Sons Inc |
| Publication Year: |
2025 |
| Collection: |
Helsingfors Universitet: HELDA – Helsingin yliopiston digitaalinen arkisto |
| Subject Terms: |
allogeneic stem cell transplantation; children and adolescents; chronic myeloid leukaemia; reduced intensity conditioning; General medicine; internal medicine and other clinical medicine |
| Description: |
This prospective multicentre trial evaluated the safety and the efficacy of a thiotepa/melphalan-based reduced intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT) in children and adolescents with chronic myeloid leukaemia (CML) in chronic phase (CP). Thirty-two patients were transplanted from matched siblings or matched unrelated donors. In 22 patients, HSCT was performed due to insufficient molecular response or loss of response to first- or second-generation tyrosine kinase inhibitor (TKI), with pretransplant BCR::ABL1 transcripts ranging between 0.001% and 33%. The protocol included a BCR::ABL1-guided intervention with TKI retreatment in the first year and donor lymphocyte infusions (DLI) in the second-year post-transplant. All patients engrafted. The 1-year transplant-related mortality was 3% (confidence interval [CI]: 0%–6%). After a median follow-up of 6.3 years, 5-year overall survival and event-free survival are 97% (CI: 93%–100%) and 91% (CI: 79%–100%) respectively. The current 5-year leukaemia-free survival with BCR::ABL1 |
| Document Type: |
article in journal/newspaper |
| File Description: |
application/pdf |
| Language: |
English |
| Relation: |
https://hdl.handle.net/10138/590319; 85194875268; 001231997300001 |
| Availability: |
https://hdl.handle.net/10138/590319 |
| Rights: |
cc_by_nc_nd ; info:eu-repo/semantics/openAccess ; openAccess |
| Accession Number: |
edsbas.55B469D3 |
| Database: |
BASE |