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Maintaining optimal nutrition in patients with Cystic Fibrosis (CF) is
paramount because of the increased expenditure in resting energy as a
result of deteriorating lung function and chronic sepsis which leads to a
significant weight loss and anorexia in some instances.
Appetite stimulants are widely used
to treat various conditions of weight
loss including anorexia. Agents that
have been used as appetite stimulants
include; cyproheptadine, megasterol,
oxandrolone, dronabinol, mirtazapine,
pizotifen, risperidone and olanzapine.
However, their efficacy and toxicity
have not been adequately evaluated in
patients with CF.3,4
This summary will
present the effectiveness and adverse
events of appetite stimulants used in
patients with CF.
The studies included in the above
mentioned review were randomised
controlled trials (RCTs) and quazi-RCTs
of appetite stimulants, compared to
placebo or no treatment for at least one
month in adults and children diagnosed
with cystic fibrosis.
Quality of the research
Studies included in the report were
of moderate methodological quality.
Biases such as incomplete outcome data
(attrition bias) and selection reporting
were the major drawbacks.
• The following databases were
searched; the Cochrane Cystic Fibrosis
and Genetic Disorders Group’s Cystic
Fibrosis Trials Register, MEDLINE,
Embase, CINAHL, hand searching
reference lists and contacting local and
• The review included three studies
with a total of 47 patients comparing
appetite stimulants cyproheptadine
hydrochloride and megasterol acetate
• The main outcome measures included;
change in body weight (kg), change
in body composition, lean body mass
(LBM), fat mass, body mass index
(BMI) and change in pulmonary
function measured as change in forced
expiratory volume in one second
(FEV1). Other measures included;
Subjective report of anorexia or change
in appetite or both, quality of life;
QoL (subjective report or measured
by a validated questionnaire), dietary
intake and any reported adverse events
directly related to the intervention.
• A meta-analysis of all three studies
showed appetite stimulants resulted
in a larger increase in weight z score
at three months compared to placebo
with a mean difference 0.61 (95%
confidence interval 0.29 to 0.93)
(P <0.001) (n = 40).
• There was no evidence of a difference
in effect between the different
• A meta-analysis of two studies showed
a significantly higher proportion of
patients reporting increased appetite,
odds ratio 45.25 (95% confidence
interval 3.57 to 573.33) (P = 0.003).
• There were no significant differences
in forced expiratory volume at one
second between the appetite stimulant
groups and placebo at follow up,
The efficacy of appetite
stimulants in cystic fibrosis
BY DR HANAN KHALIL
» EVIDENCE SUMMARIES
The purpose of this evidence summary is to provide
the best available evidence for the efficacy of appetite
stimulants in patients with cystic fibrosis. For the full
review, please refer to: Chinuck R, Dewar J, Baldwin
DR, Hendron E. Appetite stimulants for people with
cystic fibrosis. Cochrane Database of Systematic
Reviews 2014, Issue 7. Art. No.: CD008190. DOI:
Dr Hanan Khalil is the Director of the Centre for Chronic
Disease Management, a collaborating centre of the
Joanna Briggs Institute, Faculty of Medicine, Nursing
and Health Sciences, Monash University, and a reviewer
for the consumer group of the Cochrane Collaboration.
with durations ranging from two to
The adverse events reported included;
effects on glucose tolerance, decreased
cortisol levels, increased insulin levels,
insomnia, pulmonary exacerbations,
blocked port-a-cath, constipation,
haemoptysis and mild transient sedation.
However, it was hard to ascertain whether
the active or placebo medications are
contributing to these adverse events.
Implications for research and
From the results shown above, it was
difficult to determine whether one
stimulant is more effective than the
other in increasing appetite in patients
with CF. Moreover, it was not possible to
determine an optimal dose or a duration
of treatment because of the fewer
number of studies included in the review.
Moreover, the studies were also small
and underpowered and unlikely to show
adverse events with a higher certainty.
The current evidence supports that both
megasterol and cyproheptadine are
useful for the management of short-term
(i.e. six months) treatment of anorexia in
adults and children with CF.
1. Chinuck R, Dewar J, Baldwin DR, Hendron E. Appetite
stimulants for people with cystic fibrosis. Cochrane
Database of Systematic Reviews 2014, Issue 7. Art. No.:
CD008190. DOI: 10.1002/14651858.CD008190.pub2.
2. Hardin DS. A review of the management of two common
clinical problems found in patients with cystic fibrosis:
cystic fibrosis-related diabetes and poor growth. Hormone
Research 2007;68(Suppl 5):113–6 .
3. Nasr SZ, Drury D. Appetite stimulants use in cystic fibrosis.
Pediatric Pulmonology 2008;43(3):209–19.
4. Nasrallah, H. A review of the effect atypical antipsychotics on
weight. Psychoneuroendrocrinology. 2003;28:83–96 .
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