Behavioral and Nutritional Treatment for Preschool-Aged Children With Cystic Fibrosis

Abstract

IMPORTANCE Evidence-based treatments that achieve optimal energy intake and improve growth in preschool-aged children with cystic fibrosis (CF) are a critical need. OBJECTIVE To test whether behavioral and nutritional treatment (intervention) was superior to an education and attention control treatment in increasing energy intake, weight z (WAZ) score, and height z (HAZ) score. DESIGN, SETTING, AND PARTICIPANTS This randomized clinical trial included 78 children aged 2 to 6 years (mean age, 3.8 years) with CF and pancreatic insufficiency (intervention, n = 36 and control, n = 42). The study was conducted at 7 CF centers between January 2006 and November 2012; all 78 participants who met intent-to-treat criteria completed through follow-up. INTERVENTIONS Behavioral intervention combined individualized nutritional counseling targeting increased energy intake and training in behavioral child management skills. The control arm provided education and served as a behavioral placebo controlling for attention and contact frequency. Both treatments were delivered in person or telehealth (via telephone). Sessions occurred weekly for 8 weeks then monthly for 4 months (6 months). Participants then returned to standard care for 1 year, with 12-month follow-up thereafter. MAIN OUTCOMES AND MEASURES Changes in energy intake and WAZ score were examined from pretreatment to posttreatment (6 months) and change in HAZ score was assessed pretreatment to follow-up (18 months). Covariates included sex, Pseudomonas aeruginosa status at baseline, and treatment modality (in person vs telehealth). RESULTS At baseline, mean (SD) energy intake was 1462 (329) kcals/d, WAZ score was −0.44 (0.81), and HAZ score was −0.55 (0.84). From pretreatment to posttreatment, the intervention increased daily energy intake by 485 calories vs 58 calories for the control group (adjusted difference, 431 calories; 95% CI, 282 to 581; P < .001) and increased the WAZ score by 0.12 units vs 0.06 for the control (adjusted difference, 0.09; 95% CI, −0.06 to 0.24; P = .25). From pretreatment to follow-up, the intervention increased the HAZ score by 0.09 units vs −0.02 for the control (adjusted difference, 0.14 units; 95% CI, 0.001 to 0.27; P = .049). Measured treatment integrity and credibility were high for both groups. CONCLUSIONS AND RELEVANCE Behavioral and nutritional intervention improved energy intake and HAZ score outcomes but not WAZ score outcomes. Our results provide evidence that behavioral and nutritional treatment may be efficacious as a nutritional intervention for preschoolers aged 2 to 6 years with CF and pancreatic insufficiency. TRIAL REGISTRATION clinicaltrials.gov Identifier: NCT00241969

Cystic Fibrosis is Associated with Adverse Neonatal Outcomes in Washington State, 1996-2013

“…The overall birth prevalence of CF in Washington State can be calculated based on vital statistics data; between 2006 and 2013, there were 702,670 live births in Washington State (17), giving an estimated birth prevalence of CF of 1 per 5,128 live births. The birth prevalence in the overall period, 1996-2013, should not have differed substantially from the prevalence in the sub-cohort.…”

“…Since 2006, newborn screening for CF has been mandated in Washington State, and nearly all infants with CF are diagnosed within weeks of delivery. Poor neonatal outcomes could be a harbinger of more severe disease in infants with CF, and when infants are diagnosed through screening, these adverse neonatal outcomes may identify infants that need earlier and more aggressive nutritional interventions (16, 17) or targeted CF therapies (18, 19). …”

Clinical Practice Guidelines From the Cystic Fibrosis Foundation for Preschoolers With Cystic Fibrosis

“…10, [136][137][138][139] In multiple studies, families consistently reported challenges with mealtime energy-intake goals due to demanding mealtime behaviors, leading to significant stress. 8, 9, 124-127 These mealtime behavioral challenges can predict calorie intake and weight gain.…”

Parental Depression and Pancreatic Enzymes Adherence in Children With Cystic Fibrosis

“…1,2,4,11 The CFF recommends that these children consume 110% to 200% of the energy intake requirement for healthy peers and eat at least 3 meals and 3 snacks per day to achieve this elevated intake. [3][4][5] Using electronic monitors, participants' adherence was estimated at 42% (2.5 bottle openings) at home and 94% (0.9 bottle opening) at school.…”

“…3,7,8 Adherence to pancreatic enzyme regimens is also low, ranging from 27% to 46%. 9 Although effective behavioral interventions have been developed to improve nutritional outcomes in young children, 10,11 less is known about adherence to pancreatic enzyme therapy.…”