Background: Autosomal dominant polycystic kidney disease (ADPKD) commonly results
in end-stage renal disease (ESRD), yet a long-term treatment that is well tolerated is still lacking.
In a small randomized trial in children and adolescents pravastatin administration for 3 years
was associated with reduced renal cyst growth, but no large trial has tested the effect of statins in
Methods: We performed a post-hoc analysis of the HALT PKD trials to compare outcomes of participants
who never used statins with those who used statin for at least 3 years. Because statins were
not randomly allocated, we used propensity score models with inverse probability of treatment
weighting to account for imbalances between the groups. For subjects in Study A (preserved renal
function, n=438) relevant outcomes were percent change in total kidney and liver volume and the
rate of decline in estimated glomerular filtration rate (eGFR); for those in Study B (reduced renal
function, n=352) we compared time to the composite endpoint of death, ESRD or 50% decline in
eGFR. Follow-up was 5-8 years.
Results: There was no difference in any outcome between the 2 groups. However, limitations of
this analysis are the small number of statin users in Study A, different statin drugs and doses used,
non-randomized allocation and advanced disease stage in Study B.
Conclusion: Although this post-hoc analysis of the HALT PKD trials does not demonstrate a benefit
of statin therapy, conclusions remain preliminary. A larger randomized trial in young people
with ADPKD is necessary to answer the question whether statins can slow renal cyst growth and
preserve kidney function.