(226) Evaluation of the Use of Appetite Stimulants in Pediatric Patients with Cystic Fibrosis

Background: Poor nutrition in patients with cystic fibrosis (CF) has been associated with reduced lung function and increased morbidity and mortality. [1] Conversely, improved nutritional status has been associated with improved pulmonary function and fewer CF associated complications. [2] There is currently no consensus regarding appetite stimulant therapy. The Cystic Fibrosis Foundation, ESPEN-ESPGHAN-ECFS, and the Australia and New Zealand Cystic Fibrosis nutrition guidelines do not make recommendations for use of appetite stimulants due to insufficient evidence. [2,3,4] This study aimed to evaluate the association between weight gain and the use of appetite stimulants.

Methods: This was a retrospective study that evaluated 64 pediatric patients diagnosed with CF who received cyproheptadine, mirtazapine, megestrol, or dronabinol for appetite stimulation for at least 6 consecutive months between January 1, 2009 and December 31, 2019. Absolute weight, weight z-score, and weight percentiles were collected for each patient at baseline, 3 months of therapy, 6 months of therapy, and 12 months of therapy, if available. The primary objective of this study was to determine if these appetite stimulants were associated with weight changes in pediatric cystic fibrosis patients in the ambulatory care setting.

Results: Fifty-six (88%) of patients were taking cyproheptadine, six patients were taking mirtazapine, one patient was taking megestrol, and one patient was taking dronabinol. Increase in weight (absolute, percentile, and Z-score) after 3 months of therapy was statistically significant based on both univariable and multivariable longitudinal regression models. The adjusted mean difference for change in weight Z-score from baseline to month 3 was 0.31 (p < 0.0001), from month 3 to month 6 was 0.07 (p = 0.0907), and from month 6 to month 12 was -0.03 (p = 0.7235) (Figure 1). For patients who received cyproheptadine, a significant improvement in pulmonary function was observed after 3 months of therapy with an adjusted mean difference change in FEV₁ of 3.88 (p< 0.0001).

Conclusions: Appetite stimulant therapy is associated with improvement in weight Z-score in the first 3 months of treatment. Weight Z-score continues to trend upwards from months 3 – 6 and plateaus from months 6 – 12. Cyproheptadine was associated with an improvement in pulmonary function in the first 3 months of therapy, which supports the relationship between weight gain and improved pulmonary function in patients with CF. These findings suggest that appetite stimulants likely contribute to weight gain in pediatric patients with CF, particularly within the first 3 months of therapy.

Acknowledgements:

References: [1] Chinuck R, Fortnum H, Baldwin D. Appetite stimulants in cystic fibrosis: a systematic review. Journal of Human Nutrition and Dietetics. 2007;20(6):526-537
[2] van der Haak N, Kench A, Saxby N, et al. Nutrition guidelines for cystic fibrosis in Australia and New Zealand: development of evidence based and consensus recommendations. Journal of Cystic Fibrosis. 2017;16:S146
[3] Stallings V, Stark L, Robinson K, et al. Evidence-Based Practice Recommendations for Nutrition-Related Management of Children and Adults with Cystic Fibrosis and Pancreatic Insufficiency: Results of a Systematic Review. Journal of the American Dietetic Association. 2008;108(5):832-839
[4] Turck D, Braegger C, Colombo C, et al. ESPEN-ESPGHAN-ECFS guidelines on nutrition care for infants, children, and adults with cystic fibrosis. Clinical Nutrition. 2016;35(3):557-577