Pattern of use and clinical outcomes with rIX‐FP in pediatric/adolescent patients with haemophilia B in Italy: Results from IDEAL real‐world study

Abstract

ObjectivesTo evaluate pattern of use and clinical outcomes in pediatric/adolescent patients enrolled in the IDEAL study.MethodsThis post‐hoc analysis of IDEAL retrospective‐prospective observational study focused on patients <18 years, 100% on prophylaxis during the entire observation period.ResultsThirteen 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.ConclusionsThe 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.

The good, the bad and the ugly of pain in haemophilia: Recent evidence on the epidemiology, molecular mechanisms and knowledge gaps preventing optimal treatment

“…The latter, however, seems to be still a bit too ambitious aim for current clinical practice. 8,9 Spontaneous bleeds are common in severe forms of haemophilia, while in people with moderate haemophilia, bleeding occurs after minor injuries and spontaneous bleeding is rare. 2 People with mild haemophilia experience sporadic bleeding because of a trauma, injury or surgery and often remain undiagnosed until a prolonged bleeding occurs.…”