Use of oral steroids for cystic fibrosis

Cystic fibrosis causes frequent lung infection and the airways become blocked with mucus. This in turn leads to inflammation, which causes more lung damage and more mucus to be produced. Corticosteroids are strong drugs given to treat this inflammation. The review includes three trials; one for 12 weeks and two with four-year follow up. We could not combine any data. Trials showed that oral steroids of 1mg/kg to 2 mg/kg (prednisolone equivalent) given every other day seemed to slow the advance of lung disease. However, there are serious adverse effects such as cataracts and the slowing of growth at the higher dose. These led to one trial stopping early. Follow-up data show that catch-up growth started two years after treatment ceased. Treatment must use the lowest effective dose and the shortest duration of therapy to reduce the risk of a permanent effect on growth. A dose of 1 mg/kg on alternate days might be considered for up to 24 months, but close attention should be paid to adverse effects. We do not expect any further trials of this treatment to be undertaken, so we do not plan to continue to regularly update the review. However, if any new information is published, we will include this when it is available. This is an update of a previously published review.

Authors' conclusions: 

Oral corticosteroids at prednisolone-equivalent dose of 1 to 2 mg/kg alternate days appear to slow progression of lung disease in CF; benefit should be weighed against occurrence of adverse events. Risk-benefit analysis of low-dose alternate days corticosteroids is important. No further trials of this intervention are anticipated, and hence the review will no longer be regularly updated. However, if any new data are published, these will be incorporate when available.

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Background: 

In cystic fibrosis (CF) airway obstruction and recurrent respiratory infection lead to inflammation, long-term lung damage, respiratory failure and death. Anti-inflammatory agents, e.g. oral corticosteroids are used since inflammation occurs early in disease. This is an update of a previously published review.

Objectives: 

To assess the effectiveness of oral corticosteroids in respiratory complications in CF, particularly lung function and adverse events. We examined long-term use (over 30 days) only.

Search strategy: 

We searched the Cochrane CF and Genetic Disorders Group Trials Register comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings.

Most recent search: 28 August 2015.

Selection criteria: 

Randomised trials comparing oral corticosteroids given for more than 30 days with placebo or no additional therapy in people with CF.

Data collection and analysis: 

Two authors independently assessed study eligibility and quality.

Main results: 

Of eleven studies identified, three (354 participants) were included: two with four-year follow up and one with 12-weeks follow up. Data were lacking on predefined outcomes; common outcomes were examined at different time-points and presented differently. Meta-analyses were not possible.

In one study, oral corticosteroids at prednisolone-equivalent dose of 1 mg/kg alternate days slowed progression of lung disease; at two and four years, % predicted FEV1 in the 1 mg/kg group changed significantly more than in the placebo group (P < 0.02). During the first two years, the 2 mg/kg group was not significantly different from the placebo group. Linear growth retardation was observed from six months in the 2 mg/kg alternate days prednisolone group and from 24 months in the 1 mg/kg alternate days prednisolone group.

Adverse events terminated one four-year study early. Year 10 follow up showed catch-up growth started two years after treatment ceased. Alternate-day treatment with oral corticosteroids may have impaired growth until adulthood in boys.