Table 1.

Pharmacologic Agents Studied for Effects on Growth and Nutrition in CF.

Drug	Mechanism of Action	Dosage	Adverse Effects
Megestrol acetate	Appetite stimulant, ↑neuropeptide Y	400-800mg/day or 7.5-15mg/kg/day	Glucose dysregulation, insomnia, hyperactivity, irritability, adrenal suppression, concern for increased fat vs lean body mass (76, 77). There are no data regarding a safe duration for long-term use.
Cyproheptadine	Appetite stimulant, antihistamine and serotonin agonist	4mg BID-QID or 0.5mg/kg/day	Transient mild sedation, pill fatigue (78)
Dronabinol	Appetite enhancement via central cannabinoid receptors	2.5mg daily - 5mg BID	Anxiety, confusion, euphoria, somnolence (79)
Olanzapine	Appetite stimulant, atypical antipsychotic	5-20mg daily	Liver dysfunction, sleepiness, hyperglycemia (80)
Mirtazapine	Appetite stimulant, antidepressant - central presynaptic alpha2-adrenergic antagonist, potent antagonist of 5-HT2 and 5-HT3 serotonin receptors and H1histamine receptor	15-45mg daily	Mild sedation, dry mouth, somnolence (81)
Oxandrolone	Anabolic steroid, ↑protein synthesis and skeletal muscle growth	0.03-0.075mg/kg/d, once daily, max 2.5mg per day for children >9-10yrs	Hepatic dysfunction, androgenic effects in females when used at high doses, rapid progression of puberty in males in high doses (61, 62)
rhGH	Anabolic agent, stimulates linear growth of linear bone, skeletal muscle	0.3-0.5 mg/kg/week dose, given as a daily SQ injection	Potential for hyperglycemia (not found to be clinically significant to date), increased intracranial pressure, additional treatment burden due to requirement for daily injections(51-55)
Ivacaftor	Potentiates epithelial cell chloride ion transport of defective (G551D mutant) cell-surface CFTR protein	150mg every 12 hours for age 6 years and up	Skin rash / hypersensitivity reaction, transaminitis, cough(82)