C. R. Martin and P. G. Blanco contributed equally to this study.
The safety and efficacy of oral docosahexaenoic acid supplementation for the treatment of primary sclerosing cholangitis – a pilot study
Version of Record online: 30 NOV 2011
© 2011 Blackwell Publishing Ltd
Alimentary Pharmacology & Therapeutics
Volume 35, Issue 2, pages 255–265, January 2012
How to Cite
Martin, C. R., Blanco, P. G., Keach, J. C., Petz, J. L., Zaman, M. M., Bhaskar, K. R., Cluette-Brown, J. E., Gautam, S., Sheth, S., Afdhal, N. H., Lindor, K. D. and Freedman, S. D. (2012), The safety and efficacy of oral docosahexaenoic acid supplementation for the treatment of primary sclerosing cholangitis – a pilot study. Alimentary Pharmacology & Therapeutics, 35: 255–265. doi: 10.1111/j.1365-2036.2011.04926.x
- Issue online: 15 DEC 2011
- Version of Record online: 30 NOV 2011
- Publication data Submitted 3 October 2011 First decision 16 October 2011 Resubmitted 21 October 2011 Accepted 2 November 2011 EV Pub Online 30 November 2011
Aliment Pharmacol Ther 2012; 35: 255–265
Background Primary sclerosing cholangitis (PSC) is characterised by progressive inflammatory and fibrotic destruction of the biliary ducts. There are no effective medical therapies and presently high dose ursodeoxycholic acid is no longer recommended due to significant adverse events in a recent clinical trial. Cystic fibrosis transmembrane conductance regulator (CFTR) dysfunction is associated with PSC in both children and adults. Since CFTR dysfunction leads to altered fatty acid metabolism, specifically reduced docosahexaenoic acid (DHA), we hypothesised that DHA supplementation might be an effective therapy for patients with PSC.
Aim To determine the safety and efficacy of oral DHA supplementation for the treatment of PSC.
Methods We conducted a 12 month open-label pilot study to evaluate safety of oral DHA and its effects on serum alkaline phosphatase as a primary outcome measure in 23 patients with PSC. DHA was administered orally at 800 mg twice per day. Secondary outcomes included changes in other liver function tests and fibrosis biomarkers.
Results A 1.7-fold increase in serum DHA levels was observed with supplementation. The mean alkaline phosphatase level (±S.E.) at baseline was 357.8 ± 37.1 IU compared to 297.1 ± 23.7 IU (P < 0.05) after 12 months of treatment. There were no changes in other liver function tests and fibrosis biomarkers. No adverse events were reported.
Conclusions Oral DHA supplementation is associated with an increase in serum DHA levels and a significant decline in alkaline phosphatase levels in patients with PSC. These data support the need for a rigorous trial of DHA therapy in PSC.