Abstract
Purpose
Hemophilia B (HB) is a hereditary bleeding disorder caused by a deficiency of coagulation
factor IX (FIX), which represents 15% to 20% of all patients with hemophilia. Clinical
studies have found significant benefits of prophylaxis treatment with FIX versus on-demand
(OD) treatment. However, these benefits are associated with an increase in FIX consumption
and a considerable increase in cost. Most Chinese children with HB receive OD treatment.
Only a small proportion of patients with HB receive prophylaxis treatment in China.
The patients with inhibitors could result in more complicated bleeding events, joint
status, or treatment patterns. The objective of this study is to assess the cost-effectiveness
of prophylaxis compared with OD treatment in children with HB without inhibitors from
the Chinese health care perspective.
Methods
A Markov model was used to analyze cost-effectiveness by comparing prophylaxis with
OD treatment. The model uses a 17-year time horizon with a yearly cycle. Transition
probabilities and utility weights were estimated using published studies. The cost
data for patients with HB were collected from Beijing Children's Hospital and Capital
Medical University. One-way and probabilistic sensitivity analyses were performed
to assess the robustness of the results.
Findings
The model projects that prophylaxis has an incremental 1.23 quality-adjusted life-years
(QALYs). The incremental cost per QALY gained for individuals with HB receiving prophylaxis
was ¥155,709.80 ($23,530.36) compared with the OD group, which is under the willingness-to-pay
threshold (3 times the gross domestic product per capital according to the World Health
Organization recommendations) in China of ¥193,932 ($29,306.37). Moreover, 1-way sensitivity
analysis found that the results were sensitive to the utility of nonarticular bleeding.
The lower this parameter is, the higher the probability is of the incremental cost-effectiveness
ratio for prophylaxis not being cost-effective. This finding infers that when the
patients have a better QALY (higher utility) at the beginning, the cost for benefit
from prophylaxis treatment is lower. The results of the probabilistic sensitivity
analyses indicate that the probability of prophylaxis being cost-effective is 89.3%.
Implications
Although prophylaxis is a costly treatment, the results of this study indicate that
it is cost-effective compared with OD treatment for children with HB in China.
Key words
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Article info
Publication history
Published online: August 12, 2021
Accepted:
July 2,
2021
Identification
Copyright
© 2021 Published by Elsevier Inc.
