1. Pattern of use and clinical outcomes with rIX‐FP in pediatric/adolescent patients with haemophilia B in Italy: Results from IDEAL real‐world study.
- Author
-
Giordano, Paola, Pollio, Berardino, Sottilotta, Gianluca, Biasoli, Chiara, Daniele, Filomena, De Cristofaro, Raimondo, Peyvandi, Flora, Villa, Maria Rosaria, Castaman, Giancarlo, Banov, Laura, Borchiellini, Alessandra, Coppola, Antonio, Cultrera, Dorina, Cipolla, Antonino, Demartis, Francesco, Marchesini, Emanuela, Margaglione, Maurizio, Marino, Renato, Radossi, Paolo, and Santoro, Cristina
- Subjects
HEMOPHILIACS ,TREATMENT effectiveness ,TEENAGERS - Abstract
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]
- Published
- 2024
- Full Text
- View/download PDF