Impact of Steroid Therapy on Early Growth in Infants with Biliary Atresia: The Multicenter Steroids in Biliary Atresia Randomized Trial

J Pediatr. 2018 Nov;202:179-185.e4. doi: 10.1016/j.jpeds.2018.07.002. Epub 2018 Sep 21.

Abstract

Objective: To investigate the impact of corticosteroid therapy on the growth of participants in the Steroids in Biliary Atresia Randomized Trial (START) conducted through the Childhood Liver Disease Research Network. The primary analysis in START indicated that steroids did not have a beneficial effect on drainage in a cohort of infants with biliary atresia. We hypothesized that steroids would have a detrimental effect on growth in these infants.

Study design: A total of 140 infants were enrolled in START, with 70 randomized to each treatment arm: steroid and placebo. Length, weight, and head circumference were obtained at baseline and follow-up visits to 24 months of age.

Results: Patients treated with steroids had significantly lower length and head circumference z scores during the first 3 months post-hepatoportoenterostomy (HPE), and significantly lower weight until 12 months. Growth trajectories in the steroid and placebo arms differed significantly for length (P < .0001), weight (P = .009), and head circumference (P < .0001) with the largest impact noted for those with successful HPE. Growth trajectory for head circumference was significantly lower in patients treated with steroids irrespective of HPE status, but recovered during the second 6 months of life.

Conclusions: Steroid therapy following HPE in patients with biliary atresia is associated with impaired length, weight, and head circumference growth trajectories for at least 6 months post-HPE, especially impacting infants with successful bile drainage.

Trial registration: ClinicalTrials.gov: NCT00294684.

Keywords: chronic liver disease; failure to thrive; sarcopenia.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural

MeSH terms

  • Adrenal Cortex Hormones / adverse effects*
  • Adrenal Cortex Hormones / therapeutic use
  • Biliary Atresia / drug therapy*
  • Biliary Atresia / mortality
  • Biliary Atresia / surgery*
  • Body Weight / drug effects
  • Cephalometry / methods
  • Child Development / drug effects
  • Child Development / physiology
  • Child, Preschool
  • Double-Blind Method
  • Failure to Thrive / chemically induced*
  • Failure to Thrive / epidemiology
  • Failure to Thrive / physiopathology
  • Female
  • Follow-Up Studies
  • Humans
  • Infant
  • Male
  • Monitoring, Physiologic / methods
  • Portoenterostomy, Hepatic / methods
  • Portoenterostomy, Hepatic / mortality
  • Postoperative Care / methods
  • Prospective Studies
  • Reference Values
  • Risk Assessment
  • Sarcopenia / chemically induced*
  • Sarcopenia / epidemiology
  • Sarcopenia / physiopathology
  • Time Factors
  • Treatment Outcome

Substances

  • Adrenal Cortex Hormones

Associated data

  • ClinicalTrials.gov/NCT00294684