UK National Haemophilia Database, Manchester, UK.
University Department of Clinical Haematology, Manchester Royal Infirmary, Manchester, UK.
Haemophilia. 2019 Mar;25(2):205-212. doi: 10.1111/hae.13616. Epub 2018 Nov 8.
The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015.
Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint-bleed rate (ABR/AJBR) from Haemtrack (HT) in HT-compliant patients.
We report 1810 patients with SHA and 864 with MHA. Prophylaxis was used in 94.9% (n = 130/137) of HT-compliant children <12 years with SHA, falling to 74.1% (n = 123/166) aged ≥40 years. Median ABR increased with age (1.0, IQR 0.0-5.0, <12 years; 3.0 IQR, 1.0-8.0, ≥40 years). Inhibitors were present in 159 (8.8%) SHA and 34 (3.9%) MHA. Median ABR increased from 2.0 (<12 years) to 21.0 (≥40 years) in SHA inhibitor patients using prophylaxis. Prophylaxis was used by 68.8% of HT-compliant MHA patients (n = 106) (median FVIII baseline 0.01 IU/mL) associated with a median (IQR) ABR of 3.0 (1.0-7.0). Median HJHS (n = 453) increased with age in SHA and MHA. Median (IQR) HJHS was higher in SHA inhibitor (17.0, 0.0-64.5) than non- or past inhibitor patients (7.0, 0.0-23.0).
Increasing ABR with age persists despite current prophylaxis regimens. SHA and MHA had similar ABR/AJBR and HJHS, leading to a suspicion that a subgroup of MHA may be relatively undertreated. More intensive prophylaxis may improve outcomes, but this requires further study.
THUNDER 研究分析了 2015 年英国重度或中度血友病 A（SHA/MHA）患者的治疗模式和结局。
在英国国家血友病数据库（NHD）中登记的 SHA 或 MHA 患者按严重程度、抑制剂状态和年龄进行分组。从 NHD 记录中得出血友病关节健康评分（HJHS），并从 Haemtrack（HT）中获得 HT 依从性患者的治疗方案和年化出血/关节出血率（ABR/AJBR）。
我们报告了 1810 例 SHA 患者和 864 例 MHA 患者。在 HT 依从性儿童中，94.9%（n=130/137）<12 岁的儿童接受预防治疗，而≥40 岁的儿童下降至 74.1%（n=123/166）。ABR 随年龄增加而增加（1.0，IQR 0.0-5.0，<12 岁；3.0 IQR，1.0-8.0，≥40 岁）。159 例（8.8%）SHA 和 34 例（3.9%）MHA 患者存在抑制剂。SHA 抑制剂患者使用预防治疗时，ABR 从 2.0（<12 岁）增加至 21.0（≥40 岁）。68.8%的 HT 依从性 MHA 患者（n=106）接受预防治疗（FVIII 基线中位数为 0.01 IU/mL），ABR 中位数（IQR）为 3.0（1.0-7.0）。SHA 和 MHA 的 HJHS 中位数随年龄增加而增加。SHA 抑制剂患者的 HJHS 中位数（17.0，0.0-64.5）高于非抑制剂或既往抑制剂患者（7.0，0.0-23.0）。
尽管目前采用了预防治疗方案，但年龄相关的 ABR 仍在增加。SHA 和 MHA 的 ABR/AJBR 和 HJHS 相似，这表明 MHA 中有一部分患者可能治疗不足。更强化的预防治疗可能改善结局，但这需要进一步研究。
