Highlights
- •The benefits of diagnosis and treatment of people with familial hypercholesterolaemia are large.
- •Considering cholesterol burden in cost-effectiveness modelling shows larger benefits from diagnosis and treatment.
- •The magnitude of benefits also depends on prognostic factors, in particular age and low-density lipoprotein cholesterol.
Abstract
Background and aims
This study aimed to ascertain how the long-term benefits and costs of diagnosis and
treatment of familial hypercholesterolaemia (FH) vary by prognostic factors and ‘cholesterol
burden’, which is the effect of long-term exposure to low-density lipoprotein cholesterol
(LDL-C) on cardiovascular disease (CVD) risk.
Methods
A new cost-effectiveness model was developed from the perspective of the UK National
Health Service (NHS), informed by routine data from individuals with FH. The primary
outcome was net health gain (i.e., health benefits net of the losses due to costs),
expressed in quality-adjusted life years (QALYs) at the £15,000/QALY threshold. Prognostic
factors included pre-treatment LDL-C, age, gender, and CVD history.
Results
If cholesterol burden is considered, diagnosis resulted in positive net health gain
(i.e., it is cost-effective) in all individuals with pre-treatment LDL-C ≥ 4 mmol/L,
and in those with pre-treatment LDL-C ≥ 2 mmol/L aged ≥50 years or who have CVD history.
If cholesterol burden is not considered, diagnosis resulted in lower net health gain,
but still positive in children aged 10 years with pre-treatment LDL-C ≥ 6 mmol/L and
adults aged 30 years with pre-treatment LDL-C ≥ 4 mmol/L.
Conclusions
Diagnosis and treatment of most people with FH results in large net health gains,
particularly in those with higher pre-treatment LDL-C. Economic evaluations of FH
interventions should consider the sensitivity of the study conclusions to cholesterol
burden, particularly where interventions target younger patients, and explicitly consider
prognostic factors such as pre-treatment LDL-C, age, and CVD history.
Graphical abstract
Graphical Abstract
Keywords
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Article info
Publication history
Published online: December 29, 2022
Accepted:
December 6,
2022
Received in revised form:
November 19,
2022
Received:
August 27,
2022
Publication stage
In Press Journal Pre-ProofIdentification
Copyright
© 2022 Published by Elsevier B.V.
