Existing drug therapies
haemoptysis and pneumothorax. The former may require bronchial artery embolisation (or even lobectomy) but may be managed conservatively with tranexamic acid. Pneumothorax at this stage carries a poor prognosis. Patients may gain symptomatic relief from supplemental oxygen therapy and may benefit from non-invasive ventilation, either full time or during sleep. Ultimately, lung transplantation is the only option for these patients.
The frequent use of antibiotics can lead to a number of complications, including antimicrobial resistance and the emergence of newer organisms, including fungi, and renal and oto-toxicity; these are a particular problem with the aminoglycoside antibiotics, for which monitoring must be performed carefully. Even without documented toxic levels, high-frequency deafness occurs in a proportion of adult patients and appears to be related to cumulative dose. Multiple courses of corticosteroids may lead to growth suppression, glucose intolerance, cataracts and reduced bone density. These complications should all be monitored for at regular intervals.
Treatment of extra-pulmonary disease
Gastrointestinal (Table 2): over 90% of patients will have exocrine pancreatic insufficiency and require pancreatic enzyme replacement therapy (PERT); antacids, most commonly H2-blockers or omeprazole, may be used to enhance the efficacy of PERT if high doses are required. CF patients have higher than normal energy requirements because of subclinical malabsorption and a higher energy consumption secondary to infection. Increased metabolic rate is thought by some to be part of the underlying defect. Weight should be measured, and body mass index calculated, at least 3-monthly. Good nutrition should be maintained, if necessary with calorie supplements +/- gastrostomy feeding. There are gastrointestinal causes of weight loss that are unrelated to pancreatic insufficiency, such as coeliac disease (more common in CF) and undiagnosed glucose intolerance, which should be screened for. Bad nutrition is a very poor prognostic feature. Gastroesophageal reflux is common and should be treated as for non-CF patients.9 Distal intestinal obstruction syndrome (DIOS) presents with abdominal pain
Table 3: Treatment for disease in other organs Organ
Upper airway Nasal polyps Sinusitis
Insulin deficiency Frank diabetes
Sweat gland Electrolyte depletion, often leading to acute collapse
Bones and joints
Osteopenia (CFTR is expressed in bones)
CF arthropathy (large or small joint)
Bilateral absence of vas deferens leading to male infertility
Vaginal candidiasis Stress incontinence
Topical steroids Surgery if medical management fails, recurrence is common
Topical steroids and antibiotics; surgery; sinus drainage tubes for antibiotic instillationrg
Screen regularly with annual glucose tolerance test. Increasingly, continuous glucose monitoring is helpful
Low threshold for starting Insulin, especially in women, who have a worse prognosis if they develop diabetes Continue high fat diet, adjust insulin doses accordingly
Sodium and potassium chloride supplementation Measure bone mineral density at least every two years
Prevention: weight bearing exercise, high dairy intake, Vitamin D and K therapy. Treat with bisphosphonates if severe
Non-steroidal anti-inflammatory agents, prednisolone; seek specialist rheumatological advice if more than mild
Sperm aspiration and in vitro fertilisation; genetic counselling prior to procedure
Topical anti-fungal agents Seek gynaecological advice
+/- difficulty passing stool or overflow diarrhoea. Treatment is with oral (or nasogastric if required) gastrograffin or Kleanprep; hydration must be carefully maintained and might require IV fluids. Adherence to pancreatic enzymes should be reviewed as should general fluid intake. Endocrine: glucose intolerance and frank CF-related diabetes mellitus are common as patients grown older and should be routinely screened for.10 Treatment is with insulin; there is no evidence to support the use of oral hypoglycaemic agents. Table 3 lists other organs commonly affected in CF and usual treatments.
Psychological issues Cystic fibrosis clearly poses a huge burden to patients and families, both in terms of the life-shortening nature of the disease, the time-consuming treatments prescribed and the ongoing morbidity. Times of particular stress include diagnosis, adolescence, when adherence to therapy can often become poor, and end of life. Support and coping strategies from clinical psychologists with experience of the disease are often invaluable. l
References 1. Rosenfeld M et al. ISIS Study Group. Inhaled hypertonic saline in infants and children younger
than 6 years with cystic fibrosis: the ISIS randomized controlled trial. J Am Med Assoc 2012;307:2269–77.
2. Amin R et al. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax 2010;65:379–83.
3. Konstan MW, Ratjen F. Effect of dornase alfa on inflammation and lung function: potential role in the early treatment of cystic fibrosis. J Cyst Fibros 2012;11:78–83.
4. Bilton D et al. Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study. Eur Respir J 2011;38:1071–80.
5. Balfour-Lynn IM, Welch K. Inhaled corticosteroids for cystic fibrosis. Cochrane Database Syst Rev 2012;11:CD001915.
6. Flume PA et al. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med 2007;176:957–69.
7. Southern KW et al. Macrolide antibiotics for cystic fibrosis. Cochrane Database Syst Rev 2004;2:CD002203.
8. de Boer K et al. Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis. Thorax 2011; 66: 680–5.
9. Ledson MJ et al. Prevalence and mechanisms of gastro-oesophageal reflux in adult cystic fibrosis patients. J R Soc Med 1998;91:7–9.
10. Onady GM, Stolfi A. Insulin and oral agents for managing cystic fibrosis-related diabetes. Cochrane Database Syst Rev 2005;3:CD004730.
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