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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.

Background: Hemophilia A and B are X-linked inherited bleeding disorders. Hematuria is a common manifestation among patients, resulting in a worsening of quality of life. Results of previous studies regarding the association between hematuria and hemophilia have been inconsistent, leading to the need for further analysis. Aims: Determine the prevalence of macroscopic or microscopic hematuria in patients with hemophilia, as well as its association with comorbidities. Methods: Transversal unicenter study with adult patients (≥18 years old) bearing hemophilia A or B under active outpatient clinic in a reference center over the year of 2019. Hematuria was defined as any macroscopic episode or ≥3 red blood cells per high power field in urine analysis. Using simple and multivariable logistic analysis, age, diagnosis type, disease severity, comorbidities and creatinine clearance have been studied as potential characteristics associated with the prevalence of hematuria. Results: In total, 179 male patients were included. Mean age was 26 years (sd = 15), the majority of them were diagnosed with hemophilia A (76%) and 66% presented a severe disease. Despite our young population, the prevalence of comorbidities was high, mainly hypertension (10%), dyslipidemia (10%), diabetes (5%) and obesity (10%). The prevalence of hematuria was 39% (95% CI 32% - 47%), of these a half presented at least one macroscopic episode. Age (OR = 1.03[1.01-1.05]), hemophilia B (OR = 0.47[0.21-0.99]), hypertension (OR = 3.88[1.45-11.55]), obesity (OR = 2.92[1.11-8.24]) and creatinine clearance (OR = 0.99[0.98-1.00]) were associated with hematuria in univariable analysis. No independent factors were found in multivariable analysis. Conclusions: Hematuria is common among patients with hemophilia and further studies are needed to elucidate the relationship between hematuria and increased morbidity.