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kzn cystic fibrosis association

Management & Approach to Treatment

General Management

The outlook for the individual with CF has improved dramatically. Many of the clinical features previously thought to be inevitable can be prevented, delayed or improved by intensive treatment. The introduction of a more positive attitude to management and the more widespread use of aggressive treatment regimes have been major factors in improving longevity and quality of life. Better survival is associated with more frequent use of antibiotics and more frequent review at CF clinics. As insulin is to people with diabetes, so are regular, high doses of antibiotics to people who have cystic fibrosis.

Communication at the Time of Diagnosis

It is difficult for parents and/or patients to obtain more than a general impression of the condition when it is explained for the first time. Not only is CF a very complex disorder but parents are usually shocked and unable to follow detailed explanations at that time. There is a need to consolidate the information they receive at their first visit.

A team approach must be followed. Clinic visits should include consultation with medical personnel, physiotherapists, dieticians, clinic sisters, pharmacists, social workers, psychologists and parent support groups. Information must be made available to general practitioners, caregivers, teachers, relatives and friends. This information should be available in hard copy from the clinic. Additional sites of information, such as the Internet, may be of use.

It may be helpful for relatives to talk to the families of other affected individuals. Mutual support is generally most beneficial. "Remember, you are not alone".

General Management by a NON-CF Specialist

Some CF patients will be living far from a CF clinic and will find it difficult to attend CF clinics. They will be cared for by general practitioners (GPs) who do not have much experience with CF and who do not have ready access to the support of allied medical staff. It is essential that the GPs align themselves with the nearest CF Clinic and send their CF patient(s) at least yearly for assessment at the CF Clinic. Summaries of CF clinic visits should be sent to primary care doctors. As a relationship is built up with clinic doctors, so the GP will feel more comfortable about telephoning for advice and referring the patient when necessary.

General Facts Discussed at the Time of Diagnosis
  • CF remains a serious disorder despite the major advances of recent years.
  • The condition of the patient and the long-term outlook depends on the effectiveness
  • The outlook continues to improve year by year.
  • Individuals who have CF will always need regular follow-up at a hospital. The condition is so complex and advances in treatment so rapid that all patients must be cared for under the guidance of the CF specialist at the regional CF clinic.
  • The hereditary aspects of CF.
  • Families are told about the Cystic Fibrosis Association. If they agree, their names are forwarded to the relevant CF association.
General Precautions for the Individual who has CF

There are a number of reasonable precautions that should be observed by the CF individual and the family:
  • Immunisation is very important (see Section 4.3, p*).
  • NO SMOKING (active or passive). Smoking is particularly bad for people with CF.
  • Starting nursery school or crèche should be delayed (ideally until 3 years of age).
  • Reduction of exposure to friends and relatives who have just started with a "cold" as this is when they are at their most infectious.
  • If an infant with CF is admitted to hospital, every effort should be made to provide a cubicle to reduce the risk of acquiring an acute viral infection from other acutely ill children.
  • Avoidance of close contact with stables, compost or other forms of rotting vegetation is advised because of the risk of the inhaling Aspergillus spores or infection by Burkholderia cepacia.

CF patients should attend a specialist CF Clinic

  • Patients should attend a CF Clinic every one to three months.
  • Here the patient's progress must be reviewed by the entire team, if possible.
  • At every visit the patient must be weighed and measured.
  • At every visit a sputum sample or cough swab should be sent for microscopy, culture and sensitivity.
  • Parents should have a sputum container at home to send to the laboratory in the event of new respiratory infection or production of unusually purulent sputum.
  • From the age of five years, spirometry should be performed (by experienced personnel).
  • Oxygen saturation should be measured using a pulse oximeter.
  • To avoid cross infection when using all respiratory function equipment, the use of bacterial filters is advised.
  • All staff must wash their hands between patients.
  • A comprehensive CF assessment is recommended at diagnosis and annually
Details of the Initial Comprehensive CF Assessment

History/examination
Anthropometrics (height, weight, etc)
Immunisation status
Family/personal smoking

Confirmation of diagnosis

Sweat test
DNA testing

Lung status and Tests

Respiratory function
Bronchodilator test
Physiotherapist's assessment
X-ray chest
Sputum culture
Aspergillus precipitins & RAST (depending on age and symptoms)
Total IgE

Gastrointestinal status

Dietician's assessment
Electrolytes
Faecal human pancreatic elastase 1 (at diagnosis)
Modified GTT (if >10 years or younger if losing weight or there are symptoms
suggestive of diabetes mellitus)
Vitamin A, D, E serum levels (if available)
Adult diagnosis: ultrasound of liver and portal system

Additional tests

Full blood count
Liver function tests
Adult diagnosis: bone mineral density estimation

Other

Social worker consult
Genetic counselling, diagnostic testing of siblings.

Immunisation

Normal childhood immunisations including pneumococcal vaccination should be administered since viral and bacterial respiratory tract infection can have a detrimental effect on the patient's lung function and disease progression.

An annual influenza vaccine covering the expected strains for that season should be given as a routine in March/April except if there has been anaphylaxis to egg.

Passive immunisation against the respiratory syncytial virus (Synergis®) for children under the age of 2 years is thought to be useful during epidemics.

Immunisation against chicken pox and hepatitis A is recommended.

A vaccine to Pseudomonas aeruginosa is under trial at present. When/If it becomes available it should be given prior to colonisation with the organism.

Annual Review

History/examination
Anthropometrics (height, weight, etc) and review of progress over the year
Immunisation status

Lung status and Tests
Respiratory function and review of the year
Physiotherapist's assessment
X-ray chest preferably a high resolution CT scan of the lungs every 2 years.
Sputum culture and review of the year
Full blood count
Total IgE, Aspergillus precipitins & RAST (depending on age and symptoms)

Gastrointestinal/Nutritional status
Dietician's assessment
Sodium, potassium, urea, creatinine, cholesterol, calcium, magnesium, alkaline phosphatase blood levels
Faecal human pancreatic elastase 1 if pancreatic sufficient at time of review
Modified GTT (if >10 years)
Vitamin A, D, E levels (if available)
Ultrasound of liver and portal system (>10 years)

Additional tests
Adults: bone mineral density estimation
Confirmation of both CF causing mutations re : future treatment modalities.

Other
Social worker review
Review and discussion of genetic/family issues.
The results of assessments and tests should be discussed with the patient and/or parents.

Included in the discussions should be:
  • Current health status
  • Meaning of the changes (if any) over the year reviewed (good and not so good news)
  • Adjustments to treatment regimes for the coming year
  • Aims of the adjustments
  • Discussion of the patient's CF care in general
  • Planning for life events in the coming year e.g. school, employment

Nutrition

The secretion of digestive juice from the pancreas is severely reduced in most CF patients from an early age and, unless treated with pancreatic enzyme supplements, the digestion and absorption of food are severely impaired. Inadequate absorption of food from the bowel will lead to unpleasant digestive symptoms, malnutrition, poor growth and specific deficiencies of fat soluble vitamins A, D, E and K.

Well-nourished patients have fewer infections, better quality of life and increased life span. It is, therefore, essential that CF patients be referred to a dietician experienced in the management of CF. Every effort must be made to achieve normal growth in CF as good nutrition promotes good quality of life and longevity.

Feeding of Infants

Most infants with CF will thrive on breast milk (or a standard infant milk formula if breast feeding is problematic). A predigested medium chain triglyceride fat containing formula may be beneficial for infants who have undergone bowel resection for meconium ileus or those who have co-existing cow milk intolerance. If the infant is breast fed and thriving, this method of feeding should be encouraged. Bottle fed infants may require up to 200ml/kg body weight/day. Nutritional outcome is no better when babies are formula fed. If the infant is failing to thrive despite adequate pancreatic enzyme supplements and an adequate oral intake, additional energy supplements are added to the milk. Early weaning is not usually necessary. Some babies may require sodium supplementation.

Pancreatic Enzyme Supplementation

Virtually all CF patients (95%) require pancreatic enzyme supplementation owing to inadequate pancreatic secretion. A number of preparations are available. Higher doses than those recommended in the manufacturers' literature are usually required. The acid-resistant microsphere preparations are significantly more effective than the older pancreatic enzyme preparations which should NOT be used for CF patients.

High Lipase Pancreatic Preparations

Pancreatic preparations containing three to five times the quantity of lipase (Creon 25000®) are available for older children. Care should be taken to avoid total daily lipase intake of greater than 10000 U/kg/day.

General guidelines on use of pancreatic enzyme supplements

Type

Use one of the acid-resistant microsphere preparations.

Time

Enzymes are best given at the beginning or early in the meal. Half the dose at the beginning and half in the middle of the meal is recommended.

Method

Capsules should be swallowed whole from as early an age as possible. If removed from the capsule, the microspheres should not be sprinkled on or mixed with the whole meal. Microspheres should be mixed with a little fluid and taken in one swallow. If mixed with food or fruit puree, they should be mixed with one teaspoonful and taken in one or two swallows. Microspheres must not be chewed.

Dose

Enzymes are required with all meals and drinks that contain fat. Start with or ½ capsule (i.e. 3000 to 5000 units) in infants and one or two capsules per meal in older patients. Increase gradually until the bowel symptoms are controlled. Increase the dose with more fatty meals. It is advisable not to exceed a dose of 3000 units of lipase/kg body weight/meal or 10 000 units of lipase/kg body weight/day. Some patients may require higher doses.

Changes in dose should be made gradually to avoid constipation.

Insufficient pancreatic enzyme will cause symptoms of malabsorption e.g. abdominal pain, pale, loose, fatty, offensive stools, and will eventually lead to growth failure.

Patients who require larger doses than recommended may warrant the addition of an H2 blocker or proton pump inhibitor to reduce gastric acid secretion. This may permit a reduction in the number of capsules required.

Nutritional Management

Most individuals who have CF have higher than normal energy requirements due to incompletely controlled intestinal malabsorption, increased energy expenditure, chest infections and physical therapy.

A diet that is high in both energy and protein is required to achieve normal weight gain and growth. Individual requirements vary but most patients need 20 to 80% more energy than an unaffected individual of the same age. The food intake of most patients does not meet this increased energy requirement.

Patients are encouraged to take foods rich in energy such as fried foods, crisps and chocolate and those rich in protein such as milk, cheese and meat as part of their total balanced diet. Dietary sources of fat such as butter, margarine, cream or mayonnaise can be added to food to increase the energy density.

Dietary fat intake should never be restricted as this nutrient is essential to achieve a high energy intake and a normal nutritional state with growth. If foods with a high fat content cause abdominal pain or more frequent and paler stools, the dose of pancreatic enzymes should be increased whenever that food is taken. The dose is gradually increased until the food is tolerated and the steatorrhoea resolves.

It is very important that children are given frequent meals and snacks (5-6 per day) from a young age to maximise daily energy intake. Toddlers should get into the habit of regular eating. This habit will stand them in good stead as they grow up.

Psychological factors may play a major role in poor food intake patterns in some children and adolescents.

A booklet by Dr. Tony Westwood of the Red Cross Children's Hospital in Cape Town provides useful recipes and advice on CF nutrition (available from SACFA or CF centres)

Dietary Supplements

If the patient's weight gain is inadequate or the appetite poor, dietary energy supplements can improve energy intake. See Appendix 9 for a list of available products and their use. The type and amount of supplement recommended depends on the patient's age, preference and requirements and should be prescribed on an individual basis. The supplements should be taken in addition to normal food to increase the total daily energy intake. They should not replace a meal. They should be given with a snack between meals or as a drink after meals.

Salt

There is excessive loss of salt in sweat in CF. Most South African diets contain sufficient salt to compensate for this. There are two circumstances under which excess salt loss may cause clinical problems for someone with CF.

All infants with CF lose about 0.5 mmol/kg more sodium than non-CF infants. Salt deficiency can contribute to poor weight gain and must be sought where other explanations (e.g. inadequate pancreatic supplementation) are not present. Hyponatraemic dehydration may be a presenting feature of CF. In these circumstances, 0.5-1mmol/kg of salt per day in a 3% solution should be given until the age when solids constitute most of the child's diet.

Older children who live in and all who exercise in conditions of high environmental temperature should take salt tablets (1-3 per day) or increase their dietary salt and also increase their fluid intake.

Salt intake should not be restricted but excessive salt intake is dangerous.

Vitamins

All CF individuals should receive supplements of the fat soluble vitamins A, D and E. The recommended daily supplements that usually achieve normal plasma levels are considerably greater than the usual daily recommended intake. The plasma fat soluble vitamin levels should be checked annually and the dose adjusted depending on the levels. Dosages are given in Appendix 8.

Vitamin A

Vitamin A deficiency may cause night blindness in older patients. Clinical progress improves when low levels of vitamin A detected at assessment are corrected.

Vitamin D

Vitamin D deficiency may cause rickets (which is rare) and osteomalacia. Osteoporosis and low levels of vitamin D metabolites are well documented, particularly in older patients.

Vitamin E

Vitamin E deficiency may cause haemolytic anaemia in infants. In older CF individuals Vitamin E deficiency may cause neurological problems. Vitamin E appears to be important as an anti-oxidant in CF.

Vitamin K

Vitamin K may be low, particularly if there is an associated liver problem, and supplements of Vitamin K, 10mg daily, may be required if the INR is abnormal or if elective surgery is planned. Vitamin K is also important in bone disease.

Minerals

Iron

Iron supplements are not routinely needed. Full blood count should be monitored annually. Patients with moderate to severe lung disease require iron supplementation.

Calcium

Many CF patients take insufficient dietary calcium. Calcium supplementation is recommended to maximise bone mineral accretion.

Zinc

If the child is malnourished at the time of diagnosis, zinc acetate should be given for a month at a dose of 10mg daily for infants and 20mg a day for those over this age.

Assessment of Growth and Nutritional status

Growth and nutritional status need to be assessed at a specialised CF clinic every 1 to 3 months

Weight, length/height and head circumference

These must be measured and plotted on growth charts at regular intervals, looking at the pattern of growth. Current standards used in South Africa are the WHO z-score charts of weight and length/height for age and weight for height. Body mass index (BMI) is a useful index and plotted on WHO charts for children and standard tables for adults. Children under 5 years of age should have their head circumference measured every 6 months.

Mid arm muscle circumference and triceps skinfold thickness are useful indicators of lean body mass and body fat.

Growth velocity is an important measure and can assist in identifying sub-optimal growth.

Dietary Assessment

Many CF patients do not eat enough. Dietary intakes must be assessed regularly to ensure that energy requirements are being met. As part of their annual assessment, patients should record a 3-day dietary diary from which their nutritional intake is analysed and advice is given accordingly.

Nasogastric & Enterstomy Feeds

Supplemental tube feeds are frequently useful in patients with severe lung involvement. Before embarking on these forms of feeding the diet must be optimised, pancreatic enzyme replacement therapy has to be maximised and H2 blockers/protein pump inhibitors have been introduced.

Indications:
  • Children less than 5 years: weight/height less than 85% expected; weight loss or plateau in weight gain over 4 months.
  • Children 5-18 years: weight/height <85% expected; weight loss or plateau in weight gain over 6 months
  • Adults: a BMI of <19; weight loss of >5% body weight for more than 2 months duration
Methods:
  • Fine nasogastric tube left in permanently or replaced every morning (not usually well tolerated in the long term).
  • Gastrostomy:
  • Percutaneous endoscopic gastrostomy (PEG) with gastrostomy button.
  • Traditional surgical placement.
Technique:
  • Feed for 10-12 hours at night (stop 2 hours before morning physiotherapy session).
  • Eat normally during the day. At least 40-50% of the total daily energy requirement should be given at night.
  • Ideally use a peristaltic pump to avoid the tube blocking.
  • Use Ensure® as food source (not semi-elemental expensive preparations).
  • Take two thirds of enzymes at beginning and one third in morning on wakening or half on starting and half on going to sleep. Dosage to be adjusted according to usual enzyme requirement per gram of fat for the patient.
  • Patients tolerate smaller volumes of higher concentration feeds (1 or 1.5kcal/ml) better than larger volumes of less concentrated formulae.
  • Prokinetic agents may be required. Patients should be encouraged to sleep with the head elevated (30%).
Dangers:
  • Vomiting, aspiration and increase in gastro-oesophageal reflux.
  • Hyperglycaemia. Baseline oral GTT should be done before this type of feeding is commenced. Some patients will require insulin supplementation during feeding at night (see Diabetes mellitus Chapter 8 page *)
  • Leakage, bleeding or ulceration at the gastrostomy site.

Cystic Fibrosis related Diabetes

Management of cystic fibrosis-related diabetes in children and adolescents

There are important differences between CFRD and both type I and type 2 diabetes, which necessitate a unique approach to diagnosis and management (Table 1) and the involvement of a paediatric endocrinologist as part of the management team. Few CF patients have completely normal blood glucose levels at all times. The earliest change is variable, intermittent post-prandial hyperglycemia followed by impaired glucose tolerance (IGT), then diabetes without fasting hyperglycemia, and diabetes with fasting hyperglycemia. A diagnosis of ''normal'' glucose tolerance on oral glucose tolerance testing does not exclude abnormal post-prandial glucose levels at home - when far more than 75 grams of carbohydrate may be consumed.

Factors specific to CF that cause fluctuations in glucose metabolism include:
  • respiratory infection and inflammation,
  • increased energy expenditure,
  • malnutrition, glucagon deficiency and
  • gastrointestinal abnormalities
    • malabsorption,
    • altered gastric emptying and intestinal motility
    • liver disease.
diabetes table1

Pathophysiology

Abnormal chloride channel function in CF results in thick viscous secretions causing obstructive damage to the exocrine pancreas with progressive fibrosis and fatty infiltration. This result in disruption and destruction of islet architecture leading toloss of endocrine beta, alpha and pancreatic polypeptide cells. Beta-cell dysfunction is not related to autoimmune disease in CF, outside of isolated case reports of autoantibody positive individuals with CFRD.

The role of insulin deficiency

The primary defect in CFRD is severe but not absolute insulin deficiency. Virtually all exocrine insufficient patients with CF, with and without diabetes, show evidence of beta-cell dysfunction . Fasting insulin and C-peptide concentrations are normal, but there is delay and blunting of peak insulin secretion during a standard OGTT. This effect is more pronounced with worsening glycemic status. Delayed insulin secretion during the OGTT is related to loss of first phase insulin secretion, which is found even in CF patients with normal glucose tolerance. Secretion of other islet hormones is also impaired in CF, in particular loss of glucagon responses.

The role of insulin resistance

In CF patients without diabetes, insulin sensitivity is variable. While most of these patients are sensitive to insulin in their baseline state of health, infection and inflammation increase insulin resistance. CF patients with diabetes are insulin resistant, due to both decreased peripheral glucose uptake and poor insulin suppression of hepatic glucose production. Insulin resistance can become acutely severe during infectious exacerbations.

Clinical Features

  • Unexplained polyuria or polydipsia
  • Failure to gain or maintain weight despite nutritional intervention
  • Poor growth velocity
  • Delayed progression of puberty
  • Unexplained chronic decline in pulmonary function

Diagnosis of CFRD

  • Oral glucose tolerance test
  • Random and fasting glucose levels - while hyperglycemia is diagnostic for diabetes, normal fasting or random glucose levels do not exclude a diagnosis of diabetes in CF
  • Continuous glucose monitoring - this may aid the diagnosis of CFRD when considered in conjunction with the OGTT result and the clinical scenario.
  • HbA1c has been shown to be unreliable in the diagnosis of CFRD.

Treatment

Insulin Therapy

Insulin is the only recommended medical therapy for CFRD. Insulin therapy may help to stabilise lung function and improves nutritional status in patients with CFRD. There are no definitive data to date on the benefits of insulin therapy for CF children and adolescents with milder forms of abnormal glucose tolerance, although a small case series has demonstrated similar benefit. Choice of the insulin regimen depends on individual needs and characteristics of the patient. The standard basal bolus regimen provides background insulin and a continuous anabolic effect. The short acting insulin controls postprandial hyperglycaemic episodes and provides flexibility for variable eating patterns. Alternatively, effective basal-bolus therapy can be accomplished with insulin pump therapy.

Oral diabetes agents

Oral diabetes agents are currently not recommended in CFRD.

CFRD without fasting hyperglycemia and CF with impaired glucose tolerance

While insulin treatment of CFRD with fasting hyperglycemia has been the accepted standard-of-care for several years, treatment of less severe glucose tolerance
abnormalities has been more controversial. Results from a recent multi-center, randomized, placebo-controlled clinical trial demonstrate that premeal insulin therapy reversed chronic weight loss in adults with CFRD without fasting hyperglycemia and this effect was sustained over one year of treatment. Thus, insulin therapy is now recommended for all patients with CFRD with or without fasting hyperglycemia. There are insufficient data at present to make recommendations for patients with IGT or those who have NGT by OGTT testing but intermittent asymptomatic hyperglycemia when tested at home.
diabetes table2
Routine annual testing for diabetes should be performed in CF patients aged 10 years and older during a time when they are clinically well. The decision to treat should be based on consideration of blood glucose levels and the impact of treatment on the individual's overall condition.

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