Australian Haemophilia Centre Directors Organisation (AHCDO), Melbourne, Vic., Australia.
Queensland Haemophilia Centre, Royal Brisbane and Women's Hospital, Brisbane, QLD, Australia.
Haemophilia. 2018 Mar;24(2):253-260. doi: 10.1111/hae.13375. Epub 2018 Jan 3.
With the emergence of novel treatment products for haemophilia and an increasing focus on the benefits of pharmacokinetic driven individualized prophylaxis, robust national data with regard to current patterns of factor consumption and adherence are required.
To characterize current Australian practice with regard to use of prophylactic clotting factor infusions in patients with moderate or severe haemophilia A (HA) and haemophilia B (HB).
This was a retrospective, non-interventional study utilizing Australian Bleeding Disorder Registry (ABDR) data collected over a 12 month period. Registered and consented patients with moderate or severe HA or HB without inhibitors were included.
A total of 718 HA (551 severe, 167 moderate) and 166 HB (87 severe, 79 moderate) patients were included. Regular prophylaxis was prescribed in 453 patients (82%) with severe HA, 42 patients (25%) with moderate HA, 66 patients (75%) with severe HB and 11 patients (14%) with moderate HB. Near universal prophylaxis was achieved in the paediatric subgroup. The mean weekly dose of factor VIII in severe HA was 84 international units/kg/wk (IU/kg/wk) vs 71 IU/kg/wk of factor IX in severe HB. Most patients on prophylaxis were treated ≥3 times/wk (HA) or 2 times/wk (HB). Non-adherence peaked in the 20-29 year age group. Older individuals on regular prophylaxis used more factor than was expected for their prescribed regimen.
Prophylaxis rates in severe haemophilia are comparable with other developed nations. The benefit of a national registry is demonstrable. Furthermore research into the underlying reasons for non-compliance in young adults with haemophilia is required.
随着新型血友病治疗产品的出现，以及对药代动力学驱动的个体化预防效益的关注度不断提高，我们需要掌握有关当前凝血因子消耗和依从性模式的稳健的国家数据。
描述澳大利亚目前在使用预防性凝血因子输注治疗中度或重度血友病 A（HA）和血友病 B（HB）患者方面的情况。
这是一项回顾性、非干预性研究，利用澳大利亚出血性疾病登记处（ABDR）在 12 个月期间收集的数据。纳入无抑制剂的登记和同意的中重度 HA 或 HB 患者。
共纳入 718 例 HA（551 例重度，167 例中度）和 166 例 HB（87 例重度，79 例中度）患者。453 例（82%）重度 HA 患者、42 例（25%）中度 HA 患者、66 例（75%）重度 HB 患者和 11 例（14%）中度 HB 患者接受了常规预防治疗。在儿科亚组中几乎实现了普遍预防治疗。重度 HA 患者每周因子 VIII 的平均剂量为 84 国际单位/公斤/周（IU/kg/周），而重度 HB 患者每周因子 IX 的平均剂量为 71 IU/kg/周。大多数接受预防治疗的患者每周治疗≥3 次（HA）或每周 2 次（HB）。不依从性在 20-29 岁年龄组中达到峰值。定期接受预防治疗的老年人使用的因子量超过了他们规定方案的预期。
重度血友病的预防治疗率与其他发达国家相当。国家登记处的益处是显而易见的。此外，需要研究年轻血友病患者不遵医嘱的根本原因。
