Patient powered prophylaxis: A 12-month study of individualized prophylaxis in adults with severe haemophilia A
Haemophilia Sep 16, 2017
Sun HL, et al. - 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 severe haemophilia A (SHA) was assessed. This study reported reduced ABR and increased FVIII consumption in patients with SHA who switched from standard to individualized prophylaxis. In addition, these patients had improved their health-related quality of life. The mechanism was independent of adherence to the prescribed prophylactic regimen.
Methods
- Patients with baseline FVIII:C <2% and ABR >3 on weight-based prophylaxis received a standardized approach to individualized prophylaxis, in this prospective cohort study.
- Experts compared changes in ABR, annualized FVIII consumption and adherence from the 12-month prestudy and 12-month intervention period.
- Also, they investigated changes in Haemo-QoL-A total score, Physical Functioning (PF) subscale and physical activity level measured by accelerometry.
Results
- This study enrolled eighteen patients (median age 26 years).
- By 69%, individualized prophylaxis decreased total bleeds in the population and traumatic bleeds by 73%.
- From 7.5 to 2 (P<.001), the median ABR decreased.
- Moreover, 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.
- There was no change in adherence scores for frequency and dosing.
- A significant increase was observed in the Haemo-QoL-A total score (P=.02) and PF score (P=.01) from baseline to 4 months.
- However, no change was seen in physical activity.
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