Long-term observational safety study of deferiprone use in paediatric patients. The study evaluates the nature and incidence of adverse effects of deferiprone (DFP) in children and adolescents with beta-thalassaemia major. Patients treated with deferiprone are followed up throughout their treatment, from its start up to conclusion in October 2015.

Study on Quality of Life, treatment satisfaction and economic burden of disease in patients with b-thalassemia major undergoing iron chelation treatment/Qualità della vita, soddisfazione del paziente e costi della malattia in pazienti affetti da β-talassemia major sottoposti a trattamento di chelazione del ferro (HTA-Thal, “Inter-regional Network for Thalassaemia: HTA for the diagnostic and therapeutic intervention for iron overload”). The study consisted of 24-month observation period with different levels of information recorded in the first and second year of observation. The aim of this study was to assess the economic impact of thalassaemia and the burden of disease in terms of health-related quality of life (HRQoL) in a cohort of 272 adolescent (>12 yrs) and adult thalassaemic patients. The study was also aimed at investigating compliance, quality of life, patients’ satisfaction and costs associated with the different iron chelating treatments, in the perspective of the Italian Health National Care System and the society.

Quality evaluation of the informative booklets for patients involved in the DEEP-2 trial (QuBo). The aim of the study is to investigate the quality of the informative material addressed to paediatric patients involved in the DEEP-2 study, in order to evaluate the level of comprehension and the likeability of the informative documents for paediatric patients. Particularly, QuBo foresees the administration of an age-tailored structured questionnaire to directly collect the children’s and adolescents’ opinion on the DEEP-2 informative material.

Model-based bridging of clinical data for children from 3 months to < 3 years to simulate PK/PD, clinical efficacy and safety data of gabapentin in children from 3 years to less than 18 years of age in chronic pain.

Population pharmacokinetics meta-analysis based on all available published studies and data collected during clinical practice to determine the optimised dosage regimen of vancomycin to be used in children less than 3 months of age.