Search

Objectives: To evaluate pattern of use and clinical outcomes in pediatric/adolescent patients enrolled in the IDEAL study. Methods: This post‐hoc analysis of IDEAL retrospective‐prospective observational study focused on patients <18 years, 100% on prophylaxis during the entire observation period. Results: Thirteen subjects (median age 10.0 years; 61.5% ≤ 11 years) were analyzed. The infusion frequency changed from 2/week in 84.6% (N = 11) of patients with previous rFIX, to less than 1/weekly in 76.9% (N = 9) with rIX‐FP and the annualized number of infusions reduced of 57% (p =.002), from a mean ± SD of 95.1 ± 22.77 to 40.4 ± 6.79, respectively. Annualized mean consumption decreased of about 56% (p =.001), from 3748.4 ± 1155.40 IU/kg with previous rFIX, to 1656.8 ± 456.63 IU/kg of rIX‐FP. Mean FIX trough level changed from 3.0% ± 1.98% to 10.92% ± 3.6%. Low mean Annualized Bleeding Rate was maintained across all prophylaxis regimens (0.8 ± 1.69 vs. 0.3 ± 0.89) and zero bleeding patients moved from 69.2% (N = 9) with previous rFIX to 84.6% (N = 11) with rIX‐FP (p =.63). Two adverse events, none related to rIX‐FP, occurred in two patients. No inhibitors development was reported. Conclusions: The results in this pediatric/adolescent subgroup support rIX‐FP prophylaxis may reduce infusion frequency, while providing high FIX trough levels, stable annualized bleeding rate and a good safety profile. [ABSTRACT FROM AUTHOR]

The article discusses the use of pharmacological thromboprophylaxis in individuals with hemophilia undergoing major orthopedic surgery. The guidelines recommend thromboprophylaxis to prevent postoperative venous thromboembolism (VTE), but the prevalence of VTE in individuals with hemophilia is still not well-known. The article presents the experience of a cohort of individuals with hemophilia who underwent major orthopedic surgery without receiving pharmacological thromboprophylaxis. The incidence of clinically significant VTE was found to be low (0.78%), suggesting that routine pharmacological thromboprophylaxis may not be necessary in all cases. However, it should be considered in individuals with multiple high-risk factors for VTE. [Extracted from the article]

Introduction: Factor IX replacement therapy is used for treatment and prophylaxis of bleeding in haemophilia B. rIX‐FP is an extended half‐life albumin‐fusion protein, which, in clinical studies, has demonstrated prolonged dosing intervals up to 21 days for routine prophylaxis, providing therapeutic benefit. Aims: To describe dosing frequency and consumption (primary endpoint), efficacy and safety of rIX‐FP treatment during routine clinical practice in Italy. Methods: Patients with moderate/severe haemophilia B on prophylaxis with rIX‐FP for ≥6 months, were enrolled in this observational study from October 2017 to February 2019 and followed‐up for 2 years. Descriptive analysis included prospective and retrospective data (12 months prior to switching to rIX‐FP). Results: Data were collected from 59 male patients (median age 30.1 years) enrolled by 23 Italian centres. Of them, 50 were on prophylaxis during the entire observation period and completed the study. The infusion frequency changed from 2–3 times/week in 86.0% of patients with previous treatment, to less than once a week in 84.0% of patients treated with rIX‐FP at the 2nd‐year follow‐up. The annual number of infusions decreased by about 70%, whereas the mean FIX activity trough level increased from 3.8% to 14.4% (mean > 10% in all the infusion regimens). Median Annualised Bleeding Rate of.0 was achieved across all prophylaxis regimens. Subjects with zero bleedings increased from 66.0% to 78.0% with rIX‐FP. Conclusion: Treatment with rIX‐FP reduced infusion frequency, while providing higher FIX trough levels with substantial benefit in terms of annualised bleeding rate and a good safety profile. [ABSTRACT FROM AUTHOR]