ECEESPE2025 Oral Communications Oral Communications 14: Growth Axis and Syndromes (6 abstracts)
1Erasmus MC Sophia Childrens Hospital, Rotterdam, Netherlands; 2Willem-Alexander Childrens Hospital, Leiden University Medical Centre, Leiden, Netherlands; 3Reinier de Graaf Gasthuis, Delft, Netherlands; 4Beatrix Childrens Hospital, University Medical Centre Groningen, Groningen, Netherlands; 5Emma Childrens Hospital, Amsterdam University Medical Center, Amsterdam, Netherlands; 6Franciscus Gasthuis & Vlietland, Rotterdam, Netherlands; 7Dutch Growth Research Foundation, Rotterdam, Netherlands; 8Wilhelmina Childrens Hospital, University Medical Center Utrecht, Utrecht, Netherlands; 9Jeroen Bosch Hospital, s-Hertogenbosch, Netherlands; 10Erasmus University Medical Centre, Rotterdam, 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
JOINT378
Background: The majority of children diagnosed with idiopathic isolated growth hormone deficiency (IIGHD) shows normal growth hormone (GH) secretion when retested at near adult height (NAH; height velocity <2cm/y). The question is whether continuing recombinant human GH (rhGH) treatment affects NAH if normal GH secretion is observed in mid-puberty.
Aim: To investigate if withdrawing rhGH treatment from mid-puberty onwards had no negative effect on attained NAH in adolescents who no longer fulfilled a diagnosis of GH deficiency.
Methods: Adolescents diagnosed with IIGHD in childhood (GH peak at diagnosis 1.7-10 µg/l) who started rhGH treatment between 2005-2018 and tested GH sufficient (GH peak >6.7 µg/l) at mid-puberty were included in this prospective multi-center SEENEZ-trial. Mid-puberty was defined as Tanner stage G3/4, testicular volume >12 ml, bone age 13-16 years in males and Tanner stage B3/4, bone age 11-14 years in females. Study participants had the choice to discontinue or continue rhGH treatment until NAH. Primary outcome was NAH-SDS minus target height (TH) SDS. Secondary outcomes were NAH-SDS and total pubertal growth. Additionally, attained versus predicted height gain from mid-puberty to NAH was calculated using a prediction model, developed from retrospective data of an IIGHD cohort who were GH sufficient upon retesting at NAH.
Results: A total of 127 patients (95 male, 75%) participated. Forty-four patients (35%) continued rhGH treatment until NAH (GHcont), and 83 patients (65%) stopped GH treatment (GHstop). Baseline height SDS and age at mid-puberty did not differ significantly between groups. Mean (SD) NAH-SDS minus TH-SDS was -0.16 (0.60) in the GHcont and -0.19 (0.62) in the GHstop group (P=.78). Mean NAH-SDS was -0.91 (0.76) (GHcont) vs -0.79 (0.76) (GHstop) (P=.42). Mean (SD) total pubertal growth in males was 27.4 cm (7.1) (GHcont) vs 25.9 cm (6.2) (GHstop) (P=.30) and in females 20.5 cm (5.7) (GHcont) vs 21.2 cm (7.6) (GHstop) (P=.82). The predicted vs attained height gain based on the prediction model did not differ between groups.
Conclusions: In IIGHD adolescents who tested GH-sufficient in mid-puberty, NAH is comparable between those who continued rhGH treatment until NAH and those who stopped rhGH treatment at mid-puberty. In transient IIGHD adolescents, rhGH treatment can be stopped at mid-puberty. Implementing these results in guidelines reduces the duration of rhGH treatment by 2-3 years in most children with IIGHD, leading to a substantial decrease in patient burden, need for medical care and healthcare costs.