OBJECTIVES: Treatment for hemophilia patients with inhibitors is costly and challenging for its complexity, without clear agreement on drug selection or optimal dosing regimen for the first-line management of bleeding episodes. This study sought to identify treatment attributes that are important to hematologists in the United States.
METHODS: A conjoint analysis was conducted to elicit preferences using a discrete choice experiment. Twelve attributes were assessed: risk of human viral infections, possibility that the titer of the inhibitor may rise, reduction in the likelihood of dose-related thromboembolic events, the number of infusions required to stop hemorrhage, infusion preparation time, infusion time, infusion volume, time required to stop bleeding, time required to alleviate pain, prophylaxis use, ability to undergo major surgery, and cost of medications. Thirty hematologists completed questionnaires involving twelve choice tasks with trade-offs between three scenarios (most likely to use, no preference, and least likely to use). Data were analyzed using a multinomial logit model to obtain relative importance of each attribute.
RESULTS: Responding hematologists (with an average of 13 years of experience treating hemophilia patients with inhibitors) treat on average a total of 28 patients including four inhibitor patients per month. ?Time required to stop bleeding' was the most important factor affecting treatment decisions (relative importance (RI) = 16.3%). Physicians also preferred treatment products that possessed quick pain relief (RI=12.9%), no possibility that the titer of inhibitor may rise (RI=12.8%), fewer number of infusions required to stop a hemorrhage (RI=12.7%), and absence of risk of human viral infection (RI=10.8%).
CONCLUSION: The study revealed the most important attributes of treatment for hemophilia patients with inhibitors from the physician perspective. Future studies should compare physician preferences with those of hemophilia patients with inhibitors.