British Columbia Provincial Bleeding Disorders Program - Adult Division, St. Paul's Hospital, Vancouver, BC, Canada.
Division of Hematology, Department of Medicine, University of British Columbia (UBC) and St. Paul's Hospital, Vancouver, BC, Canada.
Haemophilia. 2017 Nov;23(6):877-883. doi: 10.1111/hae.13319. Epub 2017 Aug 29.
Adults with severe haemophilia A (SHA) may experience breakthrough bleeds despite standard weight-based FVIII prophylaxis three times weekly. Individualized prophylaxis has evolved to optimize patient outcomes.
This study aimed to evaluate the impact of a standardized approach to individualized prophylaxis on annualized bleeding rates (ABR), factor utilization, physical activity and quality of life in adults with SHA.
In this prospective cohort study, patients with baseline FVIII:C <2% and ABR >3 on weight-based prophylaxis received a standardized approach to individualized prophylaxis. Changes in ABR, annualized FVIII consumption and adherence from the 12-month prestudy and 12-month intervention period were compared. Changes in Haemo-QoL-A total score, Physical Functioning (PF) subscale and physical activity level measured by accelerometry were also examined.
Eighteen patients participated (median age 26 years). Individualized prophylaxis decreased total bleeds in the population by 69% and traumatic bleeds by 73%. The median ABR decreased from 7.5 to 2 (P<.001). Annualized factor consumption increased by 7.3%, as a result of 66% reduction in factor utilization for treatment of bleeds and 25% increase in factor utilization for prophylaxis. Adherence scores for frequency and dosing did not change. There was a significant increase in the Haemo-QoL-A total score (P=.02) and PF score (P=.01) from baseline to 4 months but no change in physical activity.
Patients with SHA who switched from standard to individualized prophylaxis show reduced ABR and increased FVIII consumption, and also improved their health-related quality of life. The mechanism is independent of adherence to prescribed prophylactic regimen.
尽管每周三次按照体重给予标准的因子 VIII 预防治疗，重度血友病 A（SHA）成人仍可能出现突破性出血。个体化预防治疗已发展成为优化患者结局的手段。
本研究旨在评估标准化个体化预防方案对 SHA 成人的年化出血率（ABR）、因子利用、身体活动和生活质量的影响。
在这项前瞻性队列研究中，基线因子 VIII：C <2%且基于体重的预防治疗下 ABR >3 的患者接受了标准化的个体化预防方案。比较了从基线前 12 个月和干预后 12 个月的 ABR、年化因子 VIII 消耗和依从性的变化。还检查了 Haemo-QoL-A 总评分、身体功能（PF）子量表和加速度计测量的身体活动水平的变化。
18 名患者参与（中位年龄 26 岁）。个体化预防方案使人群中的总出血减少了 69%，创伤性出血减少了 73%。ABR 中位数从 7.5 降至 2（P<.001）。年化因子消耗增加了 7.3%，这是由于出血治疗的因子利用减少了 66%，预防治疗的因子利用增加了 25%。频率和剂量的依从性评分没有变化。从基线到 4 个月，Haemo-QoL-A 总评分（P=.02）和 PF 评分（P=.01）显著增加，但身体活动没有变化。
从标准预防方案转为个体化预防方案的 SHA 患者的 ABR 降低，因子 VIII 消耗增加，生活质量也得到改善。这种机制与对预防性治疗方案的依从性无关。
