Evolution of clotting factor concentrates prescriptions and impact of recommendations of prophylaxis in children with haemophilia

Summary Prophylaxis treatment is considered as the reference approach for children with severe haemophilia A or B. However, no consensus about the best prophylaxis protocol has yet been identified in term of dosage and timing of infusions. Guidelines were drawn up in France in the early 2000s by an expert group. The objective of this 16 years study (2001 to 2016) was to describe the clotting factor concentrates (CFCs) use in haemophiac outpatients. This is a retrospective monocentric study. Pharmaceutical and clinical data were captured using medical records. Main outcome measure are CFCs use and clinical data in paediatrics. Eighty haemophiliacs A or B under 12 years old with a factor level less than 2% were included (74% of HA), from pharmaceutical outpatient data. Global use of CFCs followed the evolution of the patients’ number and regimen type introduced: increase of prophylaxis and decrease of on demand regimen. The average age at the prophylaxis introduction is significantly different according to the birth year. Prophylaxis introduction was made earlier with an increase of prophylactic regimen joined to an increase of CFCs use. The significant reduction of haemarthrosis in our cohort can be linked to a first infusion age and a prophylaxis introduction much earlier.