Title |
Should HFE p.C282Y homozygotes with moderately elevated serum ferritin be treated? A randomised controlled trial comparing iron reduction with sham treatment (Mi-iron)
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Published in |
BMJ Open, August 2015
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DOI | 10.1136/bmjopen-2015-008938 |
Pubmed ID | |
Authors |
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 |
Abstract |
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. Mi-iron is a prospective, multicentre, randomised patient-blinded trial conducted in three centres in Victoria and Queensland, Australia. Participants who are HFE p.C282Y homozygotes with SF levels between 300 and 1000 μg/L are recruited and randomised to either the treatment group or to the sham treatment group. Those in the treatment group have normalisation of SF by 3-weekly erythrocytapheresis while those in the sham treatment group have 3-weekly plasmapheresis and thus do not have normalisation of SF. Patients are blinded to all procedures. All outcome measures are administered prior to and following the course of treatment/sham treatment. Patient reported outcome measures are the Modified Fatigue Impact Scale (MFIS-primary outcome), Hospital Anxiety and Depression Scale (HADS), Medical Outcomes Study 36-item short form V.2 (SF36v2) and Arthritis Impact Measurement Scale 2 short form (AIMS2-SF). Liver injury and hepatic fibrosis are assessed with transient elastography (TE), Fibrometer and Hepascore, while oxidative stress is assessed by measurement of urine and serum F2-isoprostanes. This study has been approved by the Human Research Ethics Committees of Austin Health, Royal Melbourne Hospital and Royal Brisbane and Women's Hospital. Study findings will be disseminated through peer-reviewed publications and conference presentations. Trial identifier: NCT01631708; Registry: ClinicalTrials.gov. |
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Unknown | 3 | 100% |
Demographic breakdown
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Members of the public | 3 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 81 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Bachelor | 8 | 10% |
Student > Ph. D. Student | 6 | 7% |
Researcher | 6 | 7% |
Student > Master | 6 | 7% |
Other | 6 | 7% |
Other | 24 | 30% |
Unknown | 25 | 31% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 23 | 28% |
Psychology | 6 | 7% |
Nursing and Health Professions | 4 | 5% |
Agricultural and Biological Sciences | 4 | 5% |
Social Sciences | 4 | 5% |
Other | 15 | 19% |
Unknown | 25 | 31% |