, Volume 20, Issue 11, pp 759–774 | Cite as

Cost-Utility Analysis of Primary Prophylaxis versus Treatment On-Demand for Individuals with Severe Haemophilia

  • Alexander H. MinersEmail author
  • Caroline A. Sabin
  • Keith H. Tolley
  • Christine A. Lee
Original Research Article


Objective: To assess the cost effectiveness of primary prophylaxis with clotting factor instead of treatment following a bleed (on-demand) for individuals with severe haemophilia.

Design: Different data sources on the clinical effects and costs of treatments were combined using a Markov model.

Setting: English treatment centres.

Perspective: UK societal.

Participants: Hypothetical cohorts of 100 individuals with severe haemophilia A or B or severe von Willebrands disease.

Interventions: Primary prophylaxis treatment on-demand with clotting factor.

Outcome measures: Costs, quality-adjusted life-years (QALYs) and incremental cost per QALY in UK pounds (£, 1999/2000 values).

Results: The baseline results showed that treating individuals with severe haemophilia A/severe von Willebrands disease or severe haemophilia B with primary prophylaxis instead of treatment on-demand cost an additional £46 500 and £8600 per QALY gained, respectively. However, the results were extremely sensitive to a number of factors including the clotting factor unit cost, the time between prophylactic doses and the discount rate.

Conclusions: Despite the high costs of treatment, primary prophylaxis was cost effective compared with treatment on-demand in some scenarios. Primary prophylaxis is more likely to be cost effective for individuals with severe haemophilia B compared with individuals with severe haemophilia A/severe von Willebrands disease. Further research is required to assess the relationship between methods of clotting factor infusion and health-related quality-of-life.


Clotting Factor Major Surgery Bolus Dose Primary Prophylaxis Utility Weight 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.



The authors would like to thank Martin Buxton for comments on an earlier draft of the manuscript.


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Copyright information

© Adis International Limited 2002

Authors and Affiliations

  • Alexander H. Miners
    • 1
    Email author
  • Caroline A. Sabin
    • 2
  • Keith H. Tolley
    • 3
  • Christine A. Lee
    • 4
  1. 1.Health Economics Research GroupBrunel UniversityUxbridgeUK
  2. 2.Department of Primary Care and Population SciencesRoyal Free Hospital and University College Medical SchoolLondonUK
  3. 3.GlaxoSmithKlineStockley ParkUK
  4. 4.Royal Free Hampstead NHS TrustKatharine Dormandy Haemophilia CentreLondonUK

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