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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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