ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)
1Erasmus MC Sophia Childrens Hospital, Rotterdam, Netherlands; 2Erasmus University Medical Centre, Rotterdam, Netherlands; 3Franciscus Gasthuis & Vlietland, Rotterdam, Netherlands; 4Reinier de Graaf Gasthuis, Delft, Netherlands; 5Beatrix Childrens Hospital, University Medical Centre Groningen, Groningen, Netherlands; 6Willem-Alexander Childrens Hospital, Leiden University Medical Centre, Leiden, Netherlands; 7Emma Childrens Hospital, Amsterdam University Medical Center, Amsterdam, Netherlands; 8Dutch Growth Research Foundation, 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?
JOINT1327
Background: Idiopathic isolated growth hormone deficiency (IIGHD) is treated with recombinant human growth hormone (rhGH) to achieve normal adult height (AH). While effective in improving height, rhGH treatment is expensive, requiring hospital visits, laboratory investigations, and X-rays. It also imposes a significant treatment burden due to the necessity of daily injections. The optimal duration of rhGH treatment remains debated, with current protocols recommending continuation until near adult height (NAH). However, studies (including our SEENEZ-trial) suggest that many adolescents may not need prolonged rhGH treatment, as 70-80% of patients with IIGHD show a sufficient GH peak when retested. This raises the question whether earlier discontinuation could be cost-effective.
Aims: We aimed to analyze the costs savings of withdrawing rhGH treatment at mid-puberty in GH-sufficient adolescents with IIGHD by comparing early rhGH discontinuation with continuation until NAH.
Methods: A cost analysis was conducted alongside a multi-center patient preference trial (SEENEZ-trial). Adolescents treated with rhGH for partial IIGHD were eligible if they were in mid-puberty and had been receiving rhGH treatment for at least 3 years. Adolescents who tested GH sufficient (GH peak >6. 7 µg/l) had the choice to stop or continue rhGH treatment until NAH. Adolescents who continued GH treatment received standard care, those who stopped visited the outpatient clinic twice yearly. A comprehensive analysis of the costs for each individual patient up to 3 years was performed, including healthcare costs (relating to GH medication, outpatient clinic visits, laboratory tests, and X-rays) and costs outside the healthcare sector (travel costs).
Results: A total of 127 patients (95 male, 75%) participated in the SEENEZ-trial. Forty-four patients (35%) continued rhGH treatment until NAH (GHcont), while 83 patients (65%) stopped treatment 2-3 years earlier (GHstop). Mean costs per patient in the GHcont group equaled C= 11, 928 per year or C= 27, 868 from mid-puberty until NAH. For the GHstop group, the total costs per patient averaged C= 222 per year and C= 692 from mid-puberty until NAH. On a nation-wide scale, this suggests that early discontinuation of rhGH could potentially reduce costs in the Netherlands by approximately C= 2 million annually.
Conclusion: Withdrawing rhGH treatment 2-3 years earlier in GH-sufficient adolescents with transient IIGHD significantly decreases medical consumption and reduces healthcare costs. Even greater cost savings will be achieved if outpatient follow-up visits after withdrawing rhGH treatment are eliminated. Future research should focus on the cost-effectiveness of rhGH withdrawal, considering final height, health-related quality of life, and quality-adjusted life years.