Introduction Treatment of hemophilia A in Brazil is offered to all patients at no cost. However, several unmet medical needs exist. Method In this study, we applied the Delphi method to discuss with seven hemophilia A specialists the challenges that patients and the health system face regarding hemophilia A treatment and opportunities for improvement. Results A consensus was obtained regarding the number of weekly infusions and patient adherence to treatment. The bleeding profile, unfavourable pharmacokinetics (PKs), low adherence and high daily activity were patient profiles that would benefit from using the extended half-life (EHL) recombinant factor VIII (rFVIII). The advantages of treatment with the EHL rFVIII were the lower number of infusions per week, which could increase patient adherence and decrease the risk of bleeds, due to a more constant plasma level, a lower value. Additionally, the EHL rFVIII could improve quality of life, especially in patients with high daily activity, such as adolescents and young adults. The panelists mentioned that EHL rFVIII, if available, could be offered first to the priority group (adolescents between 12 and 19 years old), followed by adults (20 to 64 years old) and elderly people (over 65 years old). Conclusion In summary, the EHL rFVIII offers the optimal prophylaxis by decreasing the dose frequency, increasing the treatment adherence and improving the QoL, without compromising safety and efficacy. cost However exist study improvement profile PKs, PKs , (PKs) halflife half life (EHL rFVIII. . (rFVIII) week bleeds level value Additionally available 1 old, old 20 (2 6 over old. summary frequency QoL efficacy (PKs (rFVIII 2 (

Abstract The aim of this study was to evaluate the impact of oral conditions and health-related quality of life (HRQoL) on oral health-related quality of life (OHRQoL) in children and adolescents with blood coagulation disorders and hemoglobinopathies (BCDH). The study was cross-sectional and included 61 individuals aged 2 to 18 years with BCDH. Exams for dental caries (dmft/DMFT index), oral hygiene (simplified oral hygiene index – OHI-S), and gingival health (modified gingival index – MGI) were performed. The pediatric quality of life inventory™ (PedsQL™) generic core scale and oral health scale were used to measure HRQoL and OHRQoL. Spearman’s correlation coefficient (ρ) and the Mann-Whitney test (α = 0.05) were conducted to assess the relationship between covariates and the PedsQL™ oral health scale. The mean PedsQL™ oral health scale score was 76.66 (SD = 21.36). Worse OHRQoL was correlated with poor oral hygiene (ρ = -0.383; p: 0.004), poor gingival health (ρ = -0.327; p = 0.014), and better HRQoL (ρ = 0.488; p < 0.001). Greater untreated dental caries experience was associated with worse OHRQoL (p = 0.009). Worse oral health status in children and adolescents with BCDH negatively impacts OHRQoL, and OHRQoL and quality of life analyzed from a generic perspective are positively correlated constructs in this population. healthrelated related (HRQoL (OHRQoL . (BCDH) crosssectional cross sectional 6 1 dmft/DMFT dmftDMFT dmft DMFT index, , index) simplified OHIS, OHIS OHI S OHI-S) modified MGI performed inventory PedsQL (PedsQL™ Spearmans Spearman s ρ MannWhitney Mann Whitney α 0.05 005 0 05 7666 76 66 76.6 SD 21.36. 2136 21.36 21 36 21.36) 0.383 0383 383 -0.383 0.004, 0004 0.004 004 0.004) 0.327 0327 327 -0.327 0.014, 0014 0.014 014 0.014) 0.488 0488 488 0.001. 0001 0.001 001 0.001) 0.009. 0009 0.009 009 0.009) population (BCDH OHI-S (PedsQL 0.0 00 766 7 76. 213 21.3 3 0.38 038 38 -0.38 000 0.00 0.32 032 32 -0.32 0.01 01 0.48 048 48 0. 21. 0.3 03 -0.3 0.4 04 4 -0. -0 -