Risk Factors and Measures to Prevent Liver and Pancreas Complications in Pediatric Patients After HSCT

The present retrospective study will be conducted at the Bone Marrow Transplant Center, IRCCS Burlo Garofolo, Trieste, Italy. All pediatric patients who underwent one or more allogeneic HSCTs between 2010 and 2018 will be enrolled according to inclusion/exclusion criteria. The following data will be collected in a retrospective manner: * Data on the underlying disease (diagnosis, therapeutic protocol, transfusion regimen, possible relapses, type of transplant, conditioning regimen, infectious complications, immunosuppressive treatment, including use of steroids); * Pre-transplant liver and pancreatic function, as well as quantification of iron content by nuclear magnetic resonance (MRI); * Pre-transplant histological evaluation of liver parenchyma in case of pre-existing liver disease; * Post-transplant liver and pancreatic function and the evaluation of parenchymal accumulation of Fe+; * Length and doses of DFX treatment, and corresponding drug plasma concentrations as per routine drug monitoring protocols; * Treatment tolerability according to CTC-AE grading V5.0, November 27, 2017 The post-transplant data will be collected within 2 years from HSCT (or the last HSCT when more than one). The data entered in an appropriate anonymous database will be processed by descriptive statistics and uni- multivariate statistical analyses according to study endpoints. DFX plasma concentrations will be analyzed by means of to a nonlinear mixed effect modeling approach to elaborate a population pharmacokinetic (POP/PK) model. POP/PK findings will be further analyzed together with clinical and laboratory data.