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X MONTH JUNE library at www . ausdoc . com . au / therapy-update
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2022
2023
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Figure 3 . Organs affected by cystic fibrosis . |
Water and NaCl
Sweat contains excess salt
Sweat gland
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Vein |
To the small intestine |
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started at diagnosis . In infants and toddlers , this involves providing a mechanical stimulus to the chest wall that creates vibrations in the airways and accentuates airflow to help loosen secretions ; these include chest wall percussions , Swiss ball bouncing and assisted huffing . In the preschool years , children are taught blowing games , including blowing bubbles or playing a recorder , which progresses to blowing against resistance , such as blowing up balloons or blowing into water ( bubble-positive expiratory pressure [ PEP ]). Once children can blow against resistance , it is formalised with a PEP device that can be oscillatory or non-oscillatory . This is the cornerstone of treatment from school age through the lifespan .
Inhaled mucolytics are used as adjuncts to improve secretion clearance with directed techniques . Nebulised 6 % hypertonic saline has been shown to result in improvements in lung function in infants , preschool and school-aged children . 20 , 23 In infants , the use of nebulised hypertonic saline also reduces structural lung disease . 21 Inhaled dry-powder mannitol improves lung function in children and adults with CF , and dornase alfa ( recombinant human deoxyribonuclease I , an enzyme that selectively cleaves DNA ) is effective in improving lung function and reducing pulmonary exacerbations .
24 , 25
The timing of inhaled mucolytics and airway clearance is very important . Short-acting mucolytics , such as hypertonic saline or mannitol , are given immediately before or during airway clearance , whereas long-acting mucolytics ( for example , dornase alfa ), inhaled antibiotics ( for example , tobramycin ), LABAs and ICS are administered after directed clearance . Exercise — particularly moderate – high intensity — is also beneficial in promoting airway clearance ; encourage at least 30 minutes of daily exercise . Cough in CF is generally spontaneous and strong and does not need to be externally elicited .
Diet
In the past , the diet for patients with CF typically focused on high-energy and high-fat intake to meet higher energy requirements . 27 Optimising the growth and nutrition in people with CF positively influences lung function and overall survival . The expert CF dietitian closely monitors patients ’
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Figure 4 . Clinical manifestations of cystic fibrosis . |
growth , intake , pancreatic enzyme replacement therapy ( PERT ) dosing / adherence in pancreatic insufficiency , sodium and fluid requirements and supplemental energy needs .
About 85 % of those with CF have pancreatic insufficiency . 9 The dietitian tailors individualised PERT dosing according to intake at meal and snack times , and this is adjusted based on growth , intake and gastrointestinal symptoms . PERT should be taken at the start of each meal and snack containing fat . Routine supplementation of fat-soluble vitamins ( A , D , E , K ) is also encouraged , particularly in those who are pancreatic insufficient .
Children with CF lose large amounts of sodium and chloride in their sweat . Additional salt is supplied through diet and / or supplements . Insufficient salt intake can interfere with growth , reduce appetite , cause gastrointestinal symptoms and dehydration . Salt is usually added to food and drink , and a specific dose
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is prescribed based on patient size , physical activity and time of year ( people sweat more in the summer months ). If a patient is unable to meet their sodium requirements , the CF team will initiate salt tablets .
In recent decades , the nutritional status and growth of patients with CF have shown marked improvements secondary to adherence to a high-fat diet and the use of CFTR modulators . 28 , 29 The dietary recommendations for patients with CF are moving closer to general population guidelines to prevent overweight and obesity — a new and emerging issue in those with CF . 30 , 31 Studies have shown that children with CF consumed significantly more energy-dense , nutrient-poor foods ( such as fast foods ) to meet dietary targets than controls . 26 This is concerning for the future health of children with CF , who are now expected to survive well into adulthood and will experience other comorbidities . 29
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Despite overall improvements in nutritional status , some patients continue to struggle to maintain their weight with a high-energy / high-fat diet . The use of oral supplements and enteral feeding are considered in patients who are persistently underweight . 28
Medications
Pharmacological interventions ( see table 3 ) to address pulmonary manifestations now include agents that target airway mucus and airway surface liquid hydration , as well as antimicrobial therapy , including antibiotic eradication treatment in early infection and protocols for maintenance therapy in chronic infection . 6
CFTR MODULATORS The medications mentioned above minimise the effects of CFTR dysfunction through airway clearance , treatment of complications and infection prophylaxis . CFTR
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Maen K Abu Househ , Mikael Häggström : bit . ly / 3NN3p4E
modulators allows restoration of channel function by enhancing or restoring epithelial chloride and bicarbonate transport in people with certain CFTR mutations . 18 There are two types : potentiators , which increase the activity of the transport channels already on the epithelial surface ( for example , ivacaftor ), and correctors , which improve the traffic of the defective protein to the cell membrane ( such as lumacaftor , tezacaftor and elexacaftor ). 18 Those currently PBS approved appear in table 4 .
Trikafta , also known as triple combination therapy , is the newest of the CFTR modulators . Studies have found a significantly decreased frequency of pulmonary exacerbations compared with placebo , as well as substantial improvements in pulmonary function and weight gain . 18 Trikafta became available on the PBS in April 2022 for those aged 12 and older who have at least one F508del
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