Intravenous iron in adults with cystic fibrosis
Research type
Research Study
Full title
A pilot trial of intravenous iron for the treatment of iron deficiency in adult patients with cystic fibrosis
IRAS ID
204823
Contact name
Nick Talbot
Contact email
Sponsor organisation
University of Oxford / Clinical Trials and Research Governance
Eudract number
2018-002366-39
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Research Summary
Cystic fibrosis (CF) is an inherited condition affecting over 10,000 patients in the UK, which leads to recurrent chest infections and poor absorption of nutrients from the gut. In the past, patients with CF died in childhood, but most now survive into adulthood. However, they require frequent contact with healthcare services, expensive and time-consuming treatments, and have poor health-related quality of life.
Adults with cystic fibrosis often have low iron levels (iron deficiency). This is partly due to poor absorption of iron from the gut, and partly to the trapping of iron within cells of the immune system during periods of infection. Unfortunately, iron tablets are often ineffective in this setting, and may cause significant side effects in patients with cystic fibrosis.
In other patient groups, intravenous iron is used routinely to correct iron deficiency. In these patients it has been shown to be safe, and to improve energy levels, exercise tolerance, cognitive function and quality of life, even in the absence of anaemia. Recent research suggests that iron may be particularly beneficial in patients with heart and lung disease.
Despite these possible benefits, IV iron is rarely used in patients with CF, due in part to concerns about encouraging the growth of bacterial in the lungs. Iron deficiency therefore often goes untreated. However, since no clinical trials have examined the use of IV iron in CF patients to date, the risks and benefits remain uncertain.
We plan to undertake a small pilot study examining the effects of IV iron in 20 adults with CF and low iron levels. We will primarily assess whether intravenous iron is safe and effective at treating iron deficiency in this group, but will also study various other clinical parameters, to guide the design of future larger studies.
Summary of Results
Cystic fibrosis (CF) is an inherited condition affecting over 10,000 people in the UK. Patients with CF experience problems in the lungs and bowel. In the lungs, long-term bacterial infection may lead to lung damage over many years. In the bowel, food and nutrients, including iron, are poorly absorbed.
In the past, patients with CF died in childhood, but highly-effective therapies are now available, and patients routinely survive into adulthood. They often remain active and may work full-time, but they may still have poor health-related quality of life, require time-consuming treatments, and make frequent visits to hospital.
Low iron levels are common in patients with cystic fibrosis. In other long-term conditions associated with low iron levels, iron tablets are ineffective, so iron is given intravenously (directly into a vein). In patients with heart failure, for example, intravenous (IV) iron is now used routinely, and improves symptoms and exercise capacity.
Despite these potential benefits, IV iron is rarely used in patients with CF, due to a theoretical concern that it could encourage the growth of iron-dependent bacteria in the lungs, increasing the risk of infection. To address this issue, we performed a preliminary clinical trial in which 20 people with cystic fibrosis and low iron levels were treated with a single dose of IV iron, and the effects were studied over the following 12 weeks.
In this study, IV iron corrected low iron levels, without increasing the rate of infection. Our results also showed a modest improvement in exercise capacity following IV iron, and demonstrated that the treatment was acceptable to the majority of participants. These findings suggest that a larger trial of IV iron should be considered in this patient group, and these preliminary results would be valuable in the design of any such trial.
This study was funded by the National Institute for Health Research (NIHR), and took place in Oxford. We are very grateful to the study participants for taking part.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
18/SC/0415
Date of REC Opinion
23 Aug 2018
REC opinion
Favourable Opinion