ESPE Abstracts

Background: The new preventive child health care guideline for referral of short/tall children facilitates early detection of growth disorders. Understanding the impact of early treatment initiation on recombinant human growth hormone (rhGH) adherence and catch-up growth can support the use of the guideline.

Aim: To evaluate the impact of age at rhGH treatment start on adherence (≥85% vs <85% of prescribed doses administered) and catch-up growth (∆Height Standard Deviation Score [HSDS]) in patients with growth disorders.

Patients and Methods: Adherence data were extracted from the easypod™ electromechanical injection device in combination with the easypod™ connect system, which electronically records and transmits objective records of the date, time and dose injected for patients receiving rhGH. Height data from patients receiving rhGH during the easypod™ connect observational study¹ were analysed. For these patients, additional height measurements from easypod™ connect were also included. Adherence and height data were extracted for those patients aged 2-15 years (yr) at treatment start, with height data also extracted for treatment-naïve patients aged 2-18yr with GH deficiency (GHD), small for gestational age (SGA) or Turner syndrome (TS), HSDS <-1 at start and ≥1 height measurement between 0.5-3.5yr of treatment. Regression analyses were applied to study the impact of age at treatment start on adherence and HSDS between 0.5-3.5yr of treatment, adjusted for HSDS at start. ∆HSDS was expressed as predicted HSDS (using the model from the regression analyses) minus HSDS at start.

Results: Adherence and height data were available for 18,562 and 1,212 patients, respectively, (885 GHD, 243 SGA, 84 TS) with 7,485 height measurements. An early age at treatment start was associated with higher adherence (P < 0.001). In the age groups 2-7, 8-11 and 12-15yr at treatment start, adherence was 85%, 83% and 79% at 1yr, 76%, 72% and 70% at 2yr and 73%, 70% and 64% at 3yr of treatment, respectively. An early age at treatment start resulted in a higher ∆HSDS (P < 0.001). Starting from 2yr of age, the predicted ∆HSDS decreased by 0.05 SD with every year of delayed treatment start until the patient reached 10yr, remaining stable thereafter.

Conclusion: Our real-world data show that an early age at treatment start has a positive effect on adherence and catch-up growth. This highlights the importance of early referral of children with growth failure, which is facilitated by using the evidence-based referral criteria for children aged 0-9yr in the guideline.

Reference: ¹Koledova E, et al. Endocr Connect 2018;7(8): 914-923.