The pathogenesis of altered bone metabolism leading to bone mass loss and fractures in patients with cystic fibrosis (CF) is complex, and can involve malnutrition, malabsorption, lack of physical activity, vitamin D and K insufficiency, systemic inflammation, respiratory failure, liver disease, hypogonadism, and treatment with glucocorticosteroids. Many studies reported osteopenia, osteoporosis and fractures in adults with CF, with bone loss starting at an earlier age than in healthy subjects. The most common fracture sites are ribs and vertebrae (especially thoracic), with reported fracture rates as much as tenfold (ribs) and 100-fold (vertebrae) higher than in the general population. Less data are available for children, adolescents and young patients. Regular evaluation of bone mineral density (BMD) is now recommended in both adult and young CF patients to minimize the risk of fractures, with their consequent reduced mobility and increased risk of pulmonary infections. Prevention, early recognition, and adequate treatment of bone metabolic derangements are now possible and must be pursued. Nutritional aspects, vitamin D status, and physical activity are the key components of prevention. Regarding treatment, both oral and i.v. bisphosphonates (BPs) have been successfully used in adults with fragility fractures or significant BMD reduction, in particular patients starting long-term treatment with systemic glucocorticosteroids or waiting for lung transplantation. BPs have been rarely used in children and adolescents with CF, and essentially only in the presence of fragility fractures (mainly vertebral fractures), as suggested by the European Cystic Fibrosis Society. A recent multicenter randomized, placebo-controlled trial on 171 patients aged 530 years demonstrated the efficacy and safety of oral alendronate in improving BMD.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology