Kenet Gili, Chen Yeu-Chin, Lowe Gillian, Percy Charles, Tran Huyen, von Drygalski Annette, Trossaërt Marc, Reding Mark, Oldenburg Johannes, Mingot-Castellano Maria Eva, Park Young-Shil, Peyvandi Flora, Ozelo Margareth C, Mahlangu Johnny, Quinn Jennifer, Huang Mei, Reddy Divya B, Kim Benjamin
The National Hemophilia Center, Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Aviv University, Tel Hashomer, Tel Aviv-Yafo 52621, Israel.
Haemophilia Care and Research Center, Tri-Service General Hospital, National Defense Medical Center, Taipei 11490, Taiwan.
J Clin Med. 2021 Dec 18;10(24):5959. doi: 10.3390/jcm10245959.
Regular prophylaxis with exogenous factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data are scarce. Here, we report real-world data from a global cohort. Participants were men ≥18 years old with severe HA (FVIII ≤ 1 IU/dL) receiving regular prophylaxis with FVIII. Participants provided 6 months of retrospective data and were prospectively followed for up to 12 months. Annualised bleeding rate (ABR) and FVIII utilisation and infusion rates were calculated. Differences between geographic regions were explored. Of 294 enrolled participants, 225 (76.5%) completed ≥6 months of prospective follow-up. Pre-baseline and on-study, the median (range) ABR values for treated bleeds were 2.00 (0-86.0) and 1.85 (0-37.8), respectively; the median (range) annualised FVIII utilisation rates were 3629.0 (1008.5-13541.7) and 3708.0 (1311.0-14633.4) IU/kg/year, respectively; and the median (range) annualised FVIII infusion rates were 120.0 (52.0-364.0) and 122.4 (38.0-363.8) infusions/year, respectively. The median (range) Haemo-QoL-A Total Score was 76.3 (9.4-100.0) ( = 289), ranging from 85.1 in Australia to 67.7 in South America. Physical Functioning was the most impacted Haemo-QoL-A domain in 4/6 geographic regions. Despite differences among sites, participants reported bleeding requiring treatment and impaired physical functioning. These real-world data illustrate shortcomings associated with FVIII prophylaxis for this global cohort of individuals with severe HA.
对于重度甲型血友病（HA）患者，建议定期使用外源性凝血因子VIII（FVIII）进行预防，但标准化数据稀缺。在此，我们报告来自一个全球队列的真实世界数据。参与者为年龄≥18岁、患有重度HA（FVIII≤1 IU/dL）且接受FVIII定期预防的男性。参与者提供了6个月的回顾性数据，并进行了长达12个月的前瞻性随访。计算年化出血率（ABR）以及FVIII利用率和输注率。探讨了不同地理区域之间的差异。在294名登记参与者中，225名（76.5%）完成了≥6个月的前瞻性随访。基线前和研究期间，治疗性出血的ABR中位数（范围）分别为2.00（0 - 86.0）和1.85（0 - 37.8）；年化FVIII利用率中位数（范围）分别为3629.0（1008.5 - 13541.7）和3708.0（1311.0 - 14633.4）IU/kg/年；年化FVIII输注率中位数（范围）分别为120.0（52.0 - 364.0）和122.4（38.0 - 363.8）次/年。Haemo - QoL - A总分中位数（范围）为76.3（9.4 - 100.0）（n = 289），从澳大利亚的85.1到南美洲的67.7不等。在6个地理区域中的4个，身体功能是受Haemo - QoL - A影响最大的领域。尽管各地点之间存在差异，但参与者报告了需要治疗的出血情况以及身体功能受损。这些真实世界数据说明了针对这个全球重度HA患者队列进行FVIII预防存在的不足。
