Following the positive evaluation of the Scientific Committee, the Gianni Benzi Pharmacological Research Foundation awarded a research grant for a project entitled “Endothelial function and cardiovascular risk in a cohort of haemophilic children”.
The research will compare some haematological parameters (blood glucose, insulin, triglycerides, etc..) in a cohort of children with haemophilia and in healthy subjects, in order to verify the level of cardiovascular risk and promote physical activity.
On this occasion, we had the pleasure to interview the research leader, Doctor Giuseppe Lassandro, paediatrician and researcher in the Department of Biomedical Sciences and Oncology of the University of Bari.

Below is the full text of the interview, which clarifies the relevance of the research for the health of haemophilic children.

• What are the molecular basis of the increased cardiovascular risk in children with haemophilia?

Until few years ago, it was thought that haemophiliacs were “protected” from thrombotic diseases because of the lack of FVIII/IX. Replacement therapy (and prophylaxis protocols) has prolonged life expectancy, but in the adult patient population it is noted, however, a cardiovascular risk similar (if not increased) compared to the healthy adult population.

• What are the main innovative elements of your research project?

If there is a cardiovascular risk in haemophilic population, we must identify the first metabolic alarm bells, that can arise in youth, even without clinical evidence. Haemophiliacs are often sedentary children who are “kept away” from the places of physical activity for fear (often not legitimate) of the consequences of trauma.

• How will study results improve the quality of life of haemophilic children?

We hope that study results may direct towards a greater promotion of the culture of the movement.

• What is the current position of Sports Medicine, the National Olympic Committee, Sports Federations and Clubs towards children with haemophilia who want to practice sports?

There is no law regulating the physical fitness in haemophiliacs. We often rely on common sense or experience. The time is almost ripe to sit around a table and discuss the problem objectively.

• What is the risk of bleeding in these patients during exercise?

Low, if the patient is in regular prophylaxis with the replacement factor.

• Gene therapy, what is the status of the research for haemophilia?

Although the time is still immature, the first experimental results are encouraging so we can safely say that haemophilia will be the first monogenic disease to be cured by gene therapy with the ‘beta-thalassemia’, for which the first gene therapy treatments have already been carried out.

Updates related to the research project will be regularly published on the website of the Foundation.