Emma Baas¹, Chiara Garita², Judith Renes³, Ardine Reedijk³, Bibian Van der Voorn⁴, Martijn Finken¹

(1) Pediatric Endocrinology, AmsterdamUMC, Amsterdam, Netherlands.

(2) Pediatric Endocrinology, Amsterdam UMC, Amsterdam, Netherlands.

(3) National Registry of Growth hormone treatment in children (LRG), Rotterdam, Netherlands.

(4) Department of Health Sciences, Section Prevention and Public Health, Vrije Universiteit Amsterdam, Amsterdam, Netherlands.

RATIONALE

This study explored practice variation in hydrocortisone replacement therapy (HRT) among infants with adrenal insufficiency (AI), and examined how differences in the timing of transitioning from fixed-dose hydrocortisone replacement therapy (HRT) to a diurnal dosing scheme (DDS) in the first year of life affects their childhood BMI trajectory.

METHODS

This retrospective cohort study used longitudinal data from growth hormone-deficient children with a concomitant diagnosis of AI within the National Registry of Growth Hormone Treatment in Children (LRG) to explore practice variation in the timing of DDS introduction, and to compare BMI Standard Deviation Score (SDS) at ages 2, 5 and 8 years between those who transitioned at early (9 months) age.

RESULTS

Data were available for 83 children (58% boys), of whom 30 transitioned at early age, 20 at intermediate age and 19 at late age. Fourteen could not be categorized due to aberrant dosing schemes. BMI at age 5 years tended to be higher in the early and late groups, as compared to the intermediate group (1.2+-1.2 vs. 1.2+-1.5 vs 0.8+-1.6 SDS, p>0.05). At age 8 years, BMI was 1.5+-1.2 SDS for all groups.

DISCUSSION

A large variation in HRT diurnal dosing schemes was found among growth hormone-deficient infants with AI. In this retrospective observational study, the timing of DDS introduction did not significantly impact later BMI trajectory. However, all groups exhibited on average relatively high BMI SDS at ages 5 and 8 years. Evidence-based guidelines are needed to improve long term outcomes.