Who can enter
- Children and adolescents with acute myeloid leukemia (AML) who get a first stem cell transplantation
- Age: 0 up to and including 21 years at transplantation, and 0 up to and including 18 years at the initial diagnosis of AML
Goal
The goal of this study is to compare the efficacy and safety of the conditioning regimens with busulfan, cyclofosfamide and melphalan (BuCyMel) and with clofarabine, fludarabine and busulfan (CloFluBu).
Background
A conditioning regimen with chemotherapy is given prior to a stem cell transplantation to remove all cells from the bone marrow, and to make room for the bone marrow cells from the transplant. This chemotherapy has many side effects. These can be short term side effects such as infections, mucosal damage, and liver problems, but also long term side effects such as damage to the lungs and effects on fertility.
Different conditioning regimens are used in children for a stem cell transplantation. In most countries this is BuCyMel, but in the Princess Máxima Center CloFluBu is often used. The SCRIPT-AML study will compare the BuCyMel and CloFluBu regimens.
The BuCyMel regimen is effective in the treatment of AML, but causes many side effects. Therefore, a regimen is being sought that works equally well but causes fewer side effects. The schedule CloFluBu is also being used for this purpose. The SCRIPT-AML study is investigating whether both regimens work equally well, and whether a difference in side effects can be seen.
This study consists of two parts: an intervention part with randomization, and an observational part. The intervention part compares two conditioning regimens used in pediatric SCT: BuCyMel (standard arm) and CloFluBu (experimental arm).
Patients not eligible for randomization can participate in the observational part of the study. They will then receive the usual conditioning regimen. Only disease and treatment data will be recorded from these patients.
Both regimens are already widely used in the treatment of children with AML, and pose no additional risk to the patient.