Hematopoietic cell transplant in pediatric acute myeloid leukemia after similar upfront therapy; a comparison of conditioning regimens.
Birgitta A B VersluysJaap-Jan BoelensCornelis J H PronkA LankesterVictoria BordonJochen BuechnerM IfversenNatalja JackmannMikael SundinK VettenrantaJ AbrahamssonK MellgrenPublished in: Bone marrow transplantation (2021)
The impact of conditioning regimen prior to hematopoietic cell transplant (HCT) in pediatric AML-patients is not well studied. We retrospectively analyzed the impact of Busulfan-Cyclophosphamide (BuCy), Busulfan-Cyclophosphamide-Melphalan (BuCyMel) and Clofarabine-Fludarabine-Busulfan (CloFluBu) in pediatric AML-patients, with similar upfront leukemia treatment (NOPHO-DBHconsortium), receiving an HCT between 2010 and 2015. Outcomes of interest were LFS, relapse, TRM and GvHD. 103 patients were included; 30 received BuCy, 37 BuCyMel, and 36 CloFluBu. The 5-years LFS was 43.3% (SE ± 9.0) in the BuCy group, 59.2 % (SE ± 8.1) after BuCyMel, and 66.7 % (SE ± 7.9) after CloFluBu. Multivariable Cox regression analysis showed a trend to lower LFS after BuCy compared to CloFluBu (p = 0.07). BuCy was associated with a higher relapse incidence compared to the other regimens (p = 0.06). Younger age was a predictor for relapse (p = 0.02). A strong correlation between Busulfan Therapeutic Drug Monitoring (TDM) and lower incidence of aGvHD (p < 0.001) was found. In conclusion, LFS after BuCyMel and CloFluBu was comparable, lower LFS was found after BuCy, due to higher relapse incidence. CloFluBu was associated with lower incidence of aGvHD, suggesting lower toxicity with this type of conditioning. This finding is also explained by the impact of Busulfan monitoring.
Keyphrases
- acute myeloid leukemia
- allogeneic hematopoietic stem cell transplantation
- end stage renal disease
- risk factors
- newly diagnosed
- chronic kidney disease
- ejection fraction
- bone marrow
- free survival
- single cell
- prognostic factors
- peritoneal dialysis
- cell therapy
- oxidative stress
- stem cells
- acute lymphoblastic leukemia
- mesenchymal stem cells
- cell proliferation
- weight loss
- patient reported outcomes
- insulin resistance
- adipose tissue
- smoking cessation
- patient reported
- signaling pathway