Summary
Worldwide, haemophilia is the most common hereditary bleeding disorder. The incidence
of haemophilia B, however, is considerably less than haemophilia A and consequently
appears to have received less attention in the research literature. This article aims
to summarise the available evidence documenting the patient and economic burden associated
with haemophilia B and current methods of disease management. Both the immediate and
long-term clinical consequences of haemophilia B can have significant implications
for patients in terms of functional limitations and diminished health-related quality
of life (HRQOL). Evidence demonstrates that primary prophylaxis is the optimal strategy
for replacing missing clotting factor IX (FIX) and managing haemophilia B. Use of
recombinant FIX (rFIX) over plasma-derived FIX (pd-FIX) is also generally preferred
for safety reasons. Prophylaxis using currently available rFIX products, however,
requires a demanding regimen of intravenous infusions 2–3 times a week which may have
significant implications for adherence and ultimately the long-term efficacy of such
regimens. Only limited assessments of the cost-effectiveness of prophylactic versus
on-demand FIX treatment regimens have been conducted to date. Prophylaxis, however,
is generally more costly as greater quantities of FIX are consumed. Any reduction
in FIX replacement dosing frequency is expected to improve patient adherence and contribute
to improved clinical outcomes, further supporting the costeffectiveness of such interventions.
Although a rare disease, as economic constraints for healthcare increase, generating
further information regarding the key clinical, patient and economic outcomes associated
with haemophilia B will be essential for supporting improvements in care for people
with haemophilia B.
keywords
Burden - cost-effectiveness - factor IX - haemophilia B - health-related quality of
life
