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Osteoporosis drug aids young people with CF not helped by standard treatments

Recent medical developments have increased the life expectancy of people with cystic fibrosis – but this has also put them at high risk of long-term complications like bone fractures and osteoporosis.

Image: Sebastian Kaulitzki via Shutterstock

COMPELLING NEW EVIDENCE has indicated that a drug used to treat osteoporosis can help young people with cystic fibrosis who are not benefiting from standard treatments.

The first randomised trial to investigate the treatment of low bone density in young people with cystic fibrosis (CF), published in The Lancet medical journal, showed that alendronate, a drug used to curb bone loss, is safe and can help increase bone mineral density (BMD) in young patients with CF after standard treatment with calcium and vitamin D has failed.

Recent medical developments have increased the life expectancy of people with CF to about 40 years, however this has put them at high risk of long-term complications like bone fractures and osteoporosis.

Treatments to prevent osteoporosis in younger patients include calcium and vitamin D but these are not always enough to maintain BMD. Treatment with bisphosphonates is recommended in adults with CF but, until now, they have not been tested in clinical trials of children and adolescents.

The first randomised trial involved 171 young people in Italy (aged 5–30 years) with CF and low BMD, who received counselling to increase their dietary calcium intake to the daily recommended allowance plus a daily dose of vitamin D (calcifediol).

Researchers at the Istituto Auxologico Italiano IRCCS in Milan measured changes in BMD of the lumbar spine using dual energy x-ray absorptiometry and changes in serum and urine bone markers every 3–6 month; they found that, after 12 months of calcium and calcifediol, BMD had increased by 5 per cent or more in just a quarter of patients (43). That marked that the minimum annual increase expected for patients of this age range.

The second stage of the trial randomly assigned the 128 patients not responding to calcium plus calcifediol treatment after one year to a daily dose of oral alendronate or placebo for a further year. In this timeframe, BMD increased by 16.3 per cent in those taking alendronate compared to 3.1 per cent in those given placebo. Importantly, a third of young people attained a normal-for-age Z-score (a measured BMD value to the average value obtained from a healthy population of the same sex and age as the patient) after just 12 months of alendronate treatment.

Maria Luisa Bianchi, who led the study, said the study show bisphosphonates can be “prudently used” in young patients, when other and simpler measures (eg, calcium and vitamin D) have failed – but said further research is needed to assess the effects of bisphosphonates on fracture risk.

Read: Negotiations underway over new CF drug deemed ‘too expensive’
Read: St Vincent’s to provide two extra rooms for Cystic Fibrosis patients
Read: Committee seek clarification on St Vincent’s Cystic Fibrosis unit

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