B. Trambusti General and Specialised Paediatrics Unit, Giovanni XXIII Hospital, University of Bari, Bari, Italy.
Regional Reference Centre for Inherited Bleeding and Thrombotic Disorders, Transfusion Medicine, Regina Margherita Children Hospital, Turin, Italy.
Eur J Haematol. 2024 May;112(5):765-775. doi: 10.1111/ejh.14168. Epub 2024 Jan 15.
To evaluate pattern of use and clinical outcomes in pediatric/adolescent patients enrolled in the IDEAL study.
This post-hoc analysis of IDEAL retrospective-prospective observational study focused on patients <18 years, 100% on prophylaxis during the entire observation period.
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.
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.
评估 IDEAL 研究中入组的儿科/青少年患者的使用模式和临床结局。
本研究是 IDEAL 回顾性前瞻性观察研究的事后分析，主要关注在整个观察期间 100%接受预防治疗的<18 岁患者。
分析了 13 名患者（中位年龄 10.0 岁；61.5%≤11 岁）。在之前使用 rFIX 的患者中，输注频率从 84.6%（N=11）的每周 2 次，变为 rIX-FP 的 76.9%（N=9）的每周少于 1 次，每年输注次数减少了 57%（p=0.002），从平均±SD 的 95.1±22.77 降至 40.4±6.79。每年平均消耗量减少了约 56%（p=0.001），从之前使用 rFIX 的 3748.4±1155.40IU/kg，降至 rIX-FP 的 1656.8±456.63IU/kg。平均 FIX 谷底水平从 3.0%±1.98%变为 10.92%±3.6%。所有预防方案的平均年化出血率均保持较低水平（0.8±1.69 与 0.3±0.89），且零出血患者从之前使用 rFIX 的 69.2%（N=9）增加至 rIX-FP 的 84.6%（N=11）（p=0.63）。两名患者发生了两起不良事件，均与 rIX-FP 无关。未报告抑制剂的产生。
该儿科/青少年亚组的结果支持 rIX-FP 预防治疗可减少输注频率，同时提供较高的 FIX 谷底水平、稳定的年化出血率和良好的安全性。
