Increase in weight-for-age and vegetable intake in young children with cystic fibrosis treated with lumacaftor/ivacaftor for 24 weeks

ABSTRACT

Background: Modulator therapy has improved nutritional status in individuals with cystic fibrosis (CF), which is associated with favorable outcomes. Because of the high metabolic demands of CF, nutritional recommendations include energy intake of 110% to 200% of daily estimated needs for healthy individuals. With changes in energy balance after initiation of modulator therapy, these recommendations may no longer be appropriate for some people with CFand may lead to excessiveweight gain. Overweight and obesity are being reported, and nutrition concerns now include dietary quality. Dietary quality in relation to growth in young children starting lumacaftor/ivacaftor therapy has not been examined over a 24-week period and may provide new data for future nutrition guidance for individuals with CF. Method(s) The purpose of this observational study was to determine the effect of lumacaftor/ivacaftor treatment on growth and diet in medicationnaive children. Subjects aged 2 to 5 with D508/D508 mutations were recruited from the United States and Canada. Length/height, weight, and body mass index (BMI) were measured in triplicate and averaged. Z-scores were calculated using Centers for Disease Control and Prevention reference data. Dietary data were captured using 3-day weighted food records after study visits. The Healthy Eating Index (HEI) was generated using the U.S. Department of Agriculture scoring system for each recorded day and averaged. Outcomes were assessed before treatment (baseline) and 12 and 24 weeks after beginning medication. Mixed longitudinal models were used for analysis over time. Result(s) Participants (mean age 2.9 +/- 1.4, 50% female) who completed food records for at least their baseline visit plus one other visit (n = 14) had significant increases inweight-for-age z-score (WAZ) 12 (0.6 +/- 1.7, p = 0.02) and 24 (0.21 +/- 1.8, p = 0.001) weeks after therapy. There was no significant change in height-for-age (HAZ), BMI-for-age (BMIZ), or head circumference- for-age (HCZ) z-score at 12 or 24 weeks. Although not statistically significant, percentage estimated energy requirement (%EER) decreased at 12 (-7 +/- 90%, p = 0.54) and 24 (-27 +/- 90%, p = 0.08) weeks. HEI total score did not change over the 24 weeks, although vegetables and greens and beans HEI subgroup scores decreased significantly from baseline to 24 weeks (-0.73 +/- 2.2, p = 0.02;-0.68 +/- 2.1, p = 0.02, respectively). Pooled visit correlation between total vegetables and WAZ indicated a positive association (r = 0.35, p = 0.04). Conclusion(s) WAZ increased significantly over 24 weeks of lumacaftor/ ivacaftor therapy and was positively correlated with total vegetable intake, suggesting that participants with greater WAZ scores consumed more vegetables, although over the course of the study, total vegetable intake and intake of greens and beans decreased, and WAZ increased. %EER decreased over the course of the study, but not statistically significantly so, probably because of variability in energy intake within this small study sample with some COVID-19 interruptions. In summary, WAZ of children aged 2 to 5 with D508/D508 mutations increased, with no significant changes in HAZ, BMIZ, or HCZ, and they consumed fewer total vegetables and greens and beans after 24 weeks of lumacaftor/ivacaftor therapy. Acknowledgements Supported by Vertex Pharmaceutics Inc. and the National Center for Advancing Translational Sciences, National Institutes of Health, through Grant UL1TR001878.Copyright © 2022, European Cystic Fibrosis Society. All rights reserved