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Hypertonic saline therapy for cystic fibrosis

Source agency:

ANZHSN

Date of Submission:

07/12/2009

Date of Printing:

05/02/2012

Disclaimer:

This report is work in progress and should not be used for external distribution without permission from the originating agency. Users should be aware that reports are based on information available at the time of research and often on a limited literature search.

Technology, Company & Licensing

Technology name:

Hypertonic saline therapy for cystic fibrosis

Technology - description:

Although it is not fully understood, cystic fibrosis (CF) is thought to affect the liquid layer that lines the air exposed lung tissues. The defective CFTR gene may lead to an excessive reduction in this liquid layer, reducing the clearance of mucus. This can then block airways and acts as a promoter for lung infections, both of which lead to decreased lung function over time and eventual lung failure resulting in death. Hypertonic saline is thought to increase the volume of this liquid layer and hence undo the effects of the faulty CFTR gene, although this is currently speculation. Pharmaceutical hypertonic saline is administered using widely available commercial nebulisers in the patient’s home or clinical settings (Enderby & Doull 2007).

Company or developer:

N/A

Reason for database entry:

Innovative treatment with major expected health and quality of life benefits.

Technology - stage in early warning process:

Assessment complete

Technology - stage of development:

Nearly established

Licensing, reimbursement and other approval:

N/A. The hypertonic saline can be administered via commercially available nebulisers which have Australian Therapeutic Goods Administration approval.

Technology - type(s):

Procedure

Technology - use(s):

Therapeutic

Patient Indication & Setting

Patient indications:

Patients with cystic fibrosis.

Disease description and associated mortality and morbidity:

Antenatal screening exists for subjects who have a CF family history or for partners of known CF patients.

Currently there is no cure for CF and as such therapy is aimed at slowing the progression of the disease. CF patients often require intensive lifelong treatment such as physiotherapy, antibiotic treatment, dietary control, digestive enzyme capsules and other therapies. Additionally, it is believed that loss of lung function begins in infancy and continues throughout the patient’s life. The cause of death for CF patients is most predominantly loss of lung function (Cystic Fibrosis Australia 2009b).

Number of Patients:

One in twenty five Australians are carriers of CF. As the disease is recessive, if both parents are carriers, there is a 25% chance of their child having CF, i.e. 2 copies of the defective gene. Australia has approximately 2500 people with CF. There are 80 babies born with CF every year in Australia equating to an incidence of 1 in 2500 births. Australia has a nationwide neonatal screening program that identifies approximately 95% of CF babies.

Technology - specialities(s):

None

Technology - setting(s):

General hospital and ambulatory care, Community and primary care

Setting - further information:

 

Impact

Alternative and/or complementary technology:

Additive or complementary technology

Current Technology:

There are several techniques available for maintaining/preventing loss of lung function in CF patients. These are often used in combination to improve results. It is likely that hypertonic saline would be used in conjunction with these treatments rather than as a replacement for them. The main treatments for maintaining lung function aim to remove mucus secretions from the lungs and to unblock airways.

Physiotherapy is an integral part of CF treatment with most CF patients undergoing 2-4 sessions per day. Techniques such as chest percussion, vibrations and other physical manipulations are performed with the patient required to be in specific postures to facilitate mucus expulsion. Specific breathing techniques also play an important part in CF therapy. Controlled breathing with or without specific devices is also used to maintain lung function (Cystic Fibrosis Australia 2009a).

Health Impact:

Possible improvements in quality of life and possible prolonging of life from improved lung function.

Diffusion:

At the time of writing there was little evidence of wide spread diffusion of this technique in Australia, however, after discussions with clinicians at HealthPACT it appears that this technique is used in several centres around Australia including Western Australia and Queensland.

Cost, infrastructure and economic consequences:

The hypertonic saline is delivered by a nebuliser. This requires both the nebuliser and compressor. These are widely available on the internet and cost from $AU 90 to 270. The cost of the hypertonic saline is expected to be minimal and this would be available from pharmacists.

Ethical, social, legal, political and cultural impact:

No issues were identified/raised in the sources examined.

Evidence & Policy

Clinical evidence and safety:

Several studies have investigated hypertonic saline in different populations of CF patients.

A study of 164 CF patients in 16 adult or paediatric centres across Australia investigated the effect of hypertonic saline on lung function during a 48 week treatment period. The study was conducted as a parallel, double-blinded trial in which subjects (6 years of age or older) were randomised to the experimental group, inhaling 4 ml of 7 per-cent hypertonic saline, or the control group, inhaling 4 ml of 0.9 percent saline, twice daily for 48 weeks. Both groups were treated with a bronchodilator before inhalation. Subjects continued their normal course of treatment in addition to the hypertonic saline, the randomisation algorithm attempted to correct for differences in treatment between the experimental and control groups. The primary outcome was change in lung function. This was measured as a function of three variables; forced vital capacity [FVC], forced expiratory volume in one second [FEV1], and forced expiratory flow at 25 to 75 percent of FVC [FEF25 –75]. There was no significant improvement in the primary outcome between the experimental and control arms. However, there were several significant improvements in hypertonic saline arm subjects as measured by secondary outcomes such as decreases in the frequency of pulmonary exacerbations, reduced antibiotic use for exacerbations, and reduced absenteeism from work or school. Although the primary outcome was not met, there was an absolute difference between the hypertonic saline and control arms but due to variability in both arms the difference did not reach significance. In light of the safety, low cost of hypertonic saline treatment the authors conclude that it is an effective additional therapy (Elkins et al 2006) (Level II intervention evidence).



CF caused lung damage is thought to begin in infancy and hence investigation of hypertonic saline as a treatment for infants is highly important. A pilot study to assess the tolerability of hypertonic saline in infants and young children enrolled 13 CF subjects. This short term study assessed if there were any negative outcomes to a hypertonic saline treatment. The subjects were all given a sequential treatment regimen consisting of a throat swab for microbiologic assessment, a baseline assessment of pulmonary function, administration of a bronchodilator, a second assessment of pulmonary function, hypertonic saline administration, and a third assessment of pulmonary function. There was no significant difference in the markers of tolerability assessed before or after hypertonic saline treatment. These markers included lung function, respiratory symptoms, respiration rate, heart rate, oxygen saturation, and microbiologic yield from the throat swab. The only side effect was coughing in 3 infants during hypertonic saline inhalation, which resolved within five minutes (Subbarao et al 2007) (Level IV intervention evidence).

A second assessment of hypertonic saline in CF children and infants was carried out by Dellon et al. Two populations were assessed in this study; eight infants (4 months to 3 years old) and seven preschoolers (4 years to 7 years). The treatment protocol assessed involved the administration of a bronchodilator, baseline pulmonary function assessment, treatment with normal saline, a second pulmonary function assessment, treatment with hypertonic saline, then a third pulmonary function assessment. There was no change to lung function during the protocol except in one preschool aged child who had a transient >20% drop in FEV1. This was treated with a second dose of bronchodilator. The authors concluded that there were no significant issues with administering hypertonic saline to this population (Dellon et al 2008)(Level IV intervention evidence).

The administration of hypertonic saline to CF patients seems to be a useful additional therapy that may reduce exacerbations of the lungs and may improve the quality of life of CF patients. Hypertonic saline seems to be safe to administer to infants and children.

Economic evaluation:

 

Ongoing research:

 

Ongoing or planned HTA:

The use of hypertonic saline for the treatment of cystic fibrosis patients appears to be routine in a number of Australian jurisdictions, including Queensland and Western Australia, therefore HealthPACT has recommended that further assessment of this technology is no longer warranted.

Web link:

http://www.horizonscanning.com.au

References and sources:

Cystic Fibrosis Australia (2009a). [Internet]. Available from: http://www.cysticfibrosis.org.au [Accessed 12th May].

Cystic Fibrosis Australia (2009b). Understanding Cystic Fibrosis [Internet]. Cystic Fibrosis Australia. Available from: http://www.cysticfibrosis.org.au/pdf/Understanding_CF.pdf [Accessed 12th May].

Dellon, E. P., Donaldson, S. H. et al (2008). 'Safety and tolerability of inhaled hypertonic saline in young children with cystic fibrosis', Pediatr Pulmonol, 43 (11), 1100-1106.

Elkins, M. R., Robinson, M. et al (2006). 'A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis', N Engl J Med, 354 (3), 229-240.

Enderby, B. & Doull, I. (2007). 'Hypertonic saline inhalation in cystic fibrosis--salt in the wound, or sweet success?' Arch Dis Child, 92 (3), 195-196.

Subbarao, P., Balkovec, S. et al (2007). 'Pilot study of safety and tolerability of inhaled hypertonic saline in infants with cystic fibrosis', Pediatr Pulmonol, 42 (5), 471-476.

Notes: