Variability of treatment modalities and intensity in patients with severe haemophilia A on prophylaxis: Results from the Italian national registry.

Background A shift from a standard to a personalized prophylaxis has been increasingly adopted in patients with severe haemophilia A (SHA). This approach has raised the likelihood of a significant variability in the prophylactic approaches and the relative Factor VIII (FVIII) consumptions. The aim of our study is to assess the treatment variability of SHA patients without inhibitors and on prophylaxis regimen in Italy. Material and methods Data reported in the National Registry of Congenital Coagulopathies (NRCC) were analysed to assess treatment distribution within SHA patients without inhibitors, focusing on FVIII consumption in 2017, associated to prophylaxis regimen. The analysis was stratified based on age groups and Italian regions to describe the variability of FVIII consumption in Italy. Results In 2017 the Registry reported the therapeutic plans of 1,068 SHA patients without inhibitors on prophylaxis. The mean (95% CI) individual consumption ranges from 123,127 IU (99,736-146,518) in the age group 0-6 years to 345,000 IU (336,000-354,000) in the age group >20 years. A significant FVIII consumption variability was identified within the adult population. Regions with less than 50 patients reported the higher variability in mean FVIII consumption per patients-year within the different age groups. Similar difference in FVIII consumption variability was reported also in the age groups comparing "low", "middle" and "high" patient volume regions. Discussion A reliable estimation of FVIII consumption for patients' treatment is necessary to manage and plan the appropriate budget and keep treatment's costs affordable. However, without the implementation of a methodology aiming to assess the overall value produced by these FVIII consumptions, the scenario will keep driven by FVIII consumptions, its costs and the budget available. An effort by haemophilic community, haemophilia treatment centres and institutions is required to develop and share this cultural shift in improving haemophilia management and assessment.