Congenital, Hereditary, and Neonatal Diseases
The Dipartimento Pediatria e Specialita’ Pediatriche at Azienda Ospedaliera Universitaria Citta’ Della Salute E Della Scienza Di Torino is active in pediatric research on congenital and neonatal conditions, with studies focused on biliary atresia and infant acute lymphoblastic leukemia. These trials are aimed at improving long-term outcomes, supporting organ survival, and testing new treatment strategies for very young patients.
- Research on biliary atresia to help preserve native liver function and extend survival
- Studies in infant acute lymphoblastic leukemia to improve event-free survival in newly diagnosed disease
- Evaluation of new treatment approaches for pediatric hereditary and early-life disorders
These trials reflect a strong focus on rare and serious childhood diseases, where the goal is to refine treatment and improve outcomes for children and infants.
Hemic and Lymphatic Diseases
Clinical research in hemic and lymphatic diseases is a major area at this site, covering haemophilia, acute myeloid leukemia, acute lymphoblastic leukemia, and lymphomas. The studies are designed to evaluate new medicines, improve supportive care, and test better ways to control bleeding disorders and blood cancers in children and young adults.
- Trials in haemophilia A and related bleeding disorders to assess preventive treatment and long-term safety
- Studies in acute myeloid leukemia and acute lymphoblastic leukemia to improve response and survival
- Research in classical Hodgkin lymphoma and other lymphomas to test new immunotherapy and combination approaches
The site also contributes to research on anemia and other blood-related conditions, supporting broader efforts to improve pediatric hematology care.
Immune System Diseases
Work in immune system diseases includes studies for children with immunocompromised conditions, paroxysmal nocturnal hemoglobinuria, and atypical hemolytic uremic syndrome. Research here is centered on testing new therapies that may better control disease activity, prevent complications, and improve safety in vulnerable pediatric patients.
- Evaluation of treatments for PNH and aHUS to improve disease control and patient outcomes
- Studies of herpes zoster prevention in immunocompromised children and adolescents
- Investigation of immune-targeted therapies for lymphoma and other serious pediatric conditions
These trials show a broad interest in strengthening immune-related care, especially where children face high risk from infection or immune-driven disease.
Neoplasms
Oncology research is highly visible at the Turin site, with studies spanning solid tumors, leukemias, and lymphomas. The main purpose of these trials is to test new anticancer therapies, measure treatment effectiveness, and improve outcomes for children and young adults with difficult-to-treat cancers.
- Studies of pediatric solid tumors and advanced cancers to assess antitumor activity
- Trials in relapsed or refractory leukemia to explore better treatment responses
- Research in classical Hodgkin lymphoma and non-Hodgkin lymphoma to evaluate immunotherapy and combination treatment options
This area includes both newly diagnosed and relapsed disease, highlighting a strong focus on improving care where standard treatment is often not enough.
Nutritional and Metabolic Diseases
The site also conducts pediatric studies in nutritional and metabolic diseases, including type 2 diabetes mellitus and anemia associated with chronic kidney disease. These trials are intended to assess whether new treatments can improve metabolic control, support blood health, and offer better long-term management for children and adolescents.
- Research on type 2 diabetes to evaluate better glucose control and treatment safety
- Studies of anemia in chronic kidney disease to test new options for pediatric patients
- Clinical investigation aimed at improving care for metabolic and kidney-related complications
Overall, this portfolio adds important breadth to the site’s pediatric research, linking metabolic care with broader efforts in chronic disease management.


