ECEESPE2025 ePoster Presentations Growth Axis and Syndromes (132 abstracts)
1Erasmus MC Sophia Childrens Hospital, Rotterdam, Netherlands; 2Erasmus University Medical Centre, Rotterdam, Netherlands; 3Willem-Alexander Childrens Hospital, Leiden University Medical Centre, Leiden, Netherlands; 4Dutch Growth Research Foundation, Rotterdam, Netherlands; 5Reinier de Graaf Gasthuis, Delft, Netherlands; 6Beatrix Childrens Hospital, University Medical Centre Groningen, Groningen, Netherlands; 7Emma Childrens Hospital, Amsterdam University Medical Center, Amsterdam, Netherlands; 8Franciscus Gasthuis & Vlietland, Rotterdam, Netherlands; 9Wilhelmina Childrens Hospital, University Medical Center Utrecht, Utrecht, Netherlands; 10Jeroen Bosch Hospital, s-Hertogenbosch, Netherlands; 11Zuyderland Hospital, Heerlen, Netherlands; 12Amalia Childrens Hospital, Radboud University Medical Center, Nijmegen, Netherlands; 13Maastricht University Medical Center, Maastricht, Netherlands; 14Catharina Hospital, Eindhoven, Netherlands; 15St. Antonius Hospital, Nieuwegein, Netherlands; 16Canisius Wilhelmina Hospital, Nijmegen, Netherlands
JOINT14
Background: Recombinant human growth hormone (rhGH) treatment in children with idiopathic isolated growth hormone deficiency (IIGHD) typically results in catch-up growth for several years followed by a period of normal growth. The efficacy of rhGH for late pubertal height gain in adolescents with IIGHD remains unclear.
Aims: This study aimed to develop and validate a model predicting height gain (cm) from mid-puberty to near adult height (NAH; height velocity <2cm/y) in IIGHD patients who were GH sufficient upon retesting at NAH.
Methods: For model development, data from the Dutch National Registry of Growth Hormone Treatment in Children were used, focusing on 151 patients with IIGHD (98 males, 53 females) who received rhGH treatment until NAH. Participants diagnosed with IIGHD (GH peak at diagnosis 1.7-10 µg/L) received rhGH treatment for a minimum of 2.5 years. GH peak was >10 µg/L upon retesting at NAH, with treatment discontinued for at least 1 month. Mid-puberty was defined for males as Tanner stage G3-G4, testicular volume >12 ml, and bone age (BA) 13-16 years, and for females as Tanner stage B3-B4 and BA 11-14 years. If no GH retest had been conducted, we used IGF-I >0 SDS after cessation of therapy and the pediatric endocrinologists judgment to determine GH sufficiency. Validation was done in 33 males and 7 females from the prospective SEENEZ trial.
Results: In the model, final predictors at mid-puberty included age, BA, Tanner stage, and target height (TH) SDS minus height SDS at mid-puberty. Adjusted for overoptimism the equation for males was: 82.07 - 1.41 * Tanner stage 4 or 5 - 2.55 * age 2.36 * BA + 2.33 * (TH SDS - height SDS). R2 was 48%, residual SD was 4.16 cm. For females: 39.85 - 0.57 * age 1.72 * BA. R2 was 18%, residual SD was 3.64 cm. Validation analysis showed a mean (SD) difference of 1.48cm (2.36) for males and 3.57cm (2.66) for females between predicted and attained NAH.
Conclusions: This study developed a prediction model for height gain in adolescents with IIGHD during rhGH treatment in the final stages of puberty. For females, explained variance was insufficient to reliably predict height gain. For GH sufficient males, the model can be used to assess efficacy of continuing or discontinuing rhGH treatment at mid-puberty in prospective studies and to facilitate shared decision-making regarding treatment continuation at mid-puberty.