Behavioral and nutritional treatment for preschool-aged children with cystic fibrosis: a randomized clinical trial.

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.

Comparison of methods for intravenous infusion of fat emulsion during extracorporeal membrane oxygenation.

STUDY OBJECTIVES: To characterize the effects of infusing fat emulsion during neonatal extracorporeal membrane oxygenation (ECMO) by comparing results from patients receiving fat emulsion through the ECMO circuit with those receiving fat emulsion through separate intravenous access. A second goal was to identify the optimal route for administration. DESIGN: Prospective, randomized, open-label trial. SETTING: Neonatal intensive care unit in a 106-bed quaternary care pediatric hospital. SUBJECTS: Nine neonates receiving ECMO who required intravenous nutrition. Intervention. Patients received 1-3 g/kg/day of fat emulsion into either the ecmo circuit or separate intravenous access. MEASUREMENTS AND MAIN RESULTS: The ECMO circuit and samples of blood were evaluated hourly for phase separation, layering out of the emulsion from blood, agglutination, and blood clots. After completion, the oxygenators were dissected and examined. Data were compared with an unpaired t test. The characteristics of the groups were similar, except for a higher mean weight in the ECMO circuit group (3.6 +/- 0.3 kg vs 2.8 +/- 0.4 kg, p=0.03). The mean +/- SD triglyceride level during the study was 87 +/- 79 mg/dl, with no significant difference between the two groups. Two patients in each group had elevated triglyceride levels. No cases of phase separation occurred. In the five patients who received fat emulsion into the ECMO circuit, three had layering out of the emulsion and agglutination, and all developed clots in the circuit despite adequate anticoagulation. Of the four patients in the intravenous-access group, one had layering and agglutination, and two had blood clots. CONCLUSIONS: Although both methods were associated with layering out, agglutination, and clot formation, these effects occurred more frequently with administration into the ECMO circuit, particularly in areas of stasis. This may result in disruption of normal ECMO blood flow and impaired delivery of calories. Fat emulsion should therefore be administered through separate intravenous access during ECMO whenever possible.

Dexamethasone therapy in neonates treated with extracorporeal membrane oxygenation.

OBJECTIVE: To test the hypothesis that infants who received dexamethasone would have a shorter length of time on extracorporeal membrane oxygenation (ECMO). Study design Infants placed on ECMO for respiratory failure were randomly assigned to receive either dexamethasone for 3 days or placebo. Chest radiographs were scored through the use of a validated standard scoring system to assess lung injury. RESULTS: Thirty infants received dexamethasone and 29 received placebo. The median (25th%, 75th%) duration of time on ECMO was 143.5 (100, 313) hours in the dexamethasone group and 160 (111, 303) hours in the placebo group (not significant). Survival was 80% in the dexamethasone group and 83% in the placebo group. Radiographic lung injury scores (mean+/-SEM) were significantly improved in the dexamethasone group (10.5+/-0.6) versus placebo (12.3+/-0.5) on day 3 of ECMO (P=.013). Hypertension developed in 27 of the 30 infants receiving dexamethasone and 13 of the 29 infants in the placebo group during ECMO (P<.01). CONCLUSIONS: Dexamethasone given during the first 3 days of ECMO results in significant improvement in lung injury scores by day 3 of ECMO but does not significantly decrease the duration of ECMO or improve survival. The preponderance of evidence would not support the use of dexamethasone in this setting.