Journal article

Should HFE p.C282Y homozygotes with moderately elevated serum ferritin be treated? A randomised controlled trial comparing iron reduction with sham treatment (Mi-iron)

Sim Yee Ong, Lara Dolling, Jeannette L Dixon, Amanda J Nicoll, Lyle C Gurrin, Michelle Wolthuizen, Erica M Wood, Greg J Anderson, Grant A Ramm, Katrina J Allen, John K Olynyk, Darrell Crawford, Jennifer Kava, Louise E Ramm, Paul Gow, Simon Durrant, Lawrie W Powell, Martin B Delatycki

BMJ OPEN | BMJ PUBLISHING GROUP | Published : 2015

Abstract

INTRODUCTION: HFE p.C282Y homozygosity is the most common cause of hereditary haemochromatosis. There is currently insufficient evidence to assess whether non-specific symptoms or hepatic injury in homozygotes with moderately elevated iron defined as a serum ferritin (SF) of 300-1000 µg/L are related to iron overload. As such the evidence for intervention in this group is lacking. We present here methods for a study that aims to evaluate whether non-specific symptoms and hepatic fibrosis markers improve with short-term normalisation of SF in p.C282Y homozygotes with moderate elevation of SF. METHODS AND ANALYSIS: Mi-iron is a prospective, multicentre, randomised patient-blinded trial conduct..

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Grants

Awarded by National Health and Medical Research Council of Australia


Funding Acknowledgements

This project is funded by the National Health and Medical Research Council of Australia, grant number 1026394. We acknowledge the contribution of Haemonetics Australia for provision of apheresis machines for the study and Echosens who will provide Fibrometer calculations free of charge. This work was assisted by Victorian State Government Operational Infrastructure Support and Australian Government NHMRC IRIISS.