Clinical Coagulation Research, Department of Translational Medicine, Lund University, Malmö, Sweden.
Department of Haematology, Oncology and Radiation Physics, Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.
Haemophilia. 2021 May;27(3):366-374. doi: 10.1111/hae.14299. Epub 2021 Mar 29.
Data on outcome in persons with haemophilia B (PwHB) are limited and mainly extrapolated from studies of haemophilia A (HA).
To characterize treatment outcomes in persons with severe HB in the Nordic region, with a focus on joint health, compared with matched controls with HA.
PwHB attending haemophilia centres in Denmark, Finland, Norway and Sweden were enrolled and matched with controls with HA. Joint assessment using Haemophilia Joint Health Score (HJHS) and ultrasound according to Haemophilia Early Arthropathy Detection protocol (HEAD-US) was conducted. Adherence was evaluated using the Validated Haemophilia Regimen Treatment Adherence Scale (VERITAS).
Seventy-nine males with HB, with median age of 30 years (range 1-75), were enrolled. Eleven patients (14%) had a history of or current inhibitor. Twenty-nine PwHB (37%) reported joint bleeds during the prior year, and 35% had previously undergone joint surgery. Ninety-five per cent were on prophylaxis, and 70% used recombinant concentrates, with a median factor consumption of 3,900 IU/kg/year for standard half-life products. Only two patients had a VERITAS score corresponding to 'non-adherence'. Joint health, assessed with HJHS, showed a significant lower score among PwHB compared with HA controls, explained by a difference in the 18-49 age group, without observed differences in older or younger subgroups. The HEAD-US scores were overall low.
The Nordic cohort of PwHB is well treated by prophylaxis, but the goal of zero bleeds for all is not reached. Our findings suggest that patients with severe HB suffer from a milder arthropathy than patients with severe HA.
关于乙型血友病（PwHB）患者结局的数据有限，主要是从甲型血友病（HA）的研究中推断出来的。
描述北欧地区严重 HB 患者的治疗结局，并与 HA 患者的匹配对照进行比较，重点关注关节健康。
丹麦、芬兰、挪威和瑞典的血友病中心招募 PwHB 患者，并与 HA 患者的对照进行匹配。使用血友病关节健康评分（HJHS）和根据血友病早期关节病检测方案（HEAD-US）进行的超声进行关节评估。使用验证后的血友病治疗依从性量表（VERITAS）评估依从性。
共纳入 79 名男性 HB 患者，中位年龄 30 岁（范围 1-75 岁）。11 名患者（14%）有或目前有抑制剂病史。29 名 PwHB（37%）在过去一年中报告有关节出血，35%曾接受过关节手术。95%的患者接受预防治疗，70%使用重组浓缩物，标准半衰期产品的中位因子消耗为 3900IU/kg/年。只有两名患者的 VERITAS 评分对应“不依从”。使用 HJHS 评估关节健康，PwHB 的评分明显低于 HA 对照组，这一差异主要归因于 18-49 岁年龄组，在年龄较小或较大的亚组中未观察到差异。HEAD-US 评分总体较低。
北欧的 PwHB 患者队列通过预防治疗得到了很好的治疗，但尚未达到所有患者零出血的目标。我们的研究结果表明，严重 HB 患者的关节病比严重 HA 患者更轻。
