ESPE2023 Poster Category 1 Growth and Syndromes (75 abstracts)
15-year experience with the IGF1 generation test in the Netherlands
Anne Kruijsen 1 , Kirsten de Groote 1 , Lauren Punt 1 , Paul van Trotsenburg 2 , Karijn Pijnenburg-Kleizen 3 , Gianni Bocco 4 , Lizanne Berkenbosch 5 , Petra van Setten 6 , Hedi Claahsen - van der Grinten 6 , Danielle van der Kaay 7 , Nina Schott 8 , Vera van Tellingen 9 , Edgar van Mill 10 , Josine van der Heyden 11 , Annelies Brandsma 12 , Yvonne Hendriks 1 , Monique Losekoot 1 , Hermine van Duyvenvoorde 1 , Anita Hokken-Koelega 7,13 , Judith Renes 13,14 , Jan Maarten Wit 1 , Christiaan de Bruin 1 & Sjoerd Joustra 1
1Leiden University Medical Center, Leiden, Netherlands. 2Amsterdam University Medical Center, Amsterdam, Netherlands. 3Canisius Wilhelmina Hospital, Nijmegen, Netherlands. 4University Medical Center Groningen, Groningen, Netherlands. 5Maastricht University Medical Center, Maastricht, Netherlands. 6Radboud University Medical Center, Nijmegen, Netherlands. 7ErasmusMC, Rotterdam, Netherlands. 8Zuyderland Hospital, Heerlen, Netherlands. 9Catharina Hospital, Eindhoven, Netherlands. 10Jeroen Bosch Hospital, 's-Hertogenbosch, Netherlands. 11Franciscus Gasthuis & Vlietland, Rotterdam, Netherlands. 12Maasstad Hospital, Rotterdam, Netherlands. 13Dutch Growth Research Foundation, Rotterdam, Netherlands. 14Albert Schweitzer Hospital, Dordrecht, Netherlands
Introduction: Among children with short stature, some show persistent IGF-I levels <-2.0 SDS despite a normal growth hormone (GH) response in a stimulation test. This may be caused by conditions that could benefit from recombinant human GH (rhGH) therapy (e.g. GH neurosecretory dysfunction, bioinactive GH, partial GH insensitivity). Therefore, the IGF-I generation test (IGFIGT) was implemented in 2006 using a national, standardized protocol. Children with a positive response in the IGF1IGT started rhGH treatment. In this study, we describe the long-term outcome of this strategy.
Methods: Our retrospective cohort includes children with height <-2.5 SDS, repeated IGF-I <-2.0 SDS, maximum GH peak >10 µg/L, BMI >-2.0 SDS, and a positive IGF1IGT as indicated by an IGF-I rise of ≥1.0 SDS after one week of rhGH at 0.7 mg/m2/day or (in case of insufficient rise) 1.4 mg/m2/day or 2.8 mg/m2/day. The rhGH dose at which a sufficient IGF-I increase was observed determined the starting dose for therapy. Children who were treated for ≥1 year were included.
Results: We identified 111 children in our National Registry (28% females), aged 2.4-16.7 years. Mean birthweight and birth length were -0.3 SDS. At start of treatment, mean height was -2.9 SDS, with normal BMI and sitting height/height ratio. Bone age delay was 0.9-2.5 years (interquartile range). Available genetic analyses identified Okur-Chung syndrome (n=1), Noonan syndrome (n=1), and FGFR3 variants (n=5). Fifty-eight patients responded to the 0.7 mg/m2/day dose in the IGFIGT, 38 to 1.4 mg/m2/day, and 15 only to 2.4 mg/m2/day. Most patients (84.2%) were prepubertal at start of therapy. After one year of rhGH therapy in patients who remained prepubertal (n=86), height increased by 0.8±0.3 SDS. After three years, height increase in patients who remained prepubertal (n=56) was 1.5±0.5 SDS and their predicted adult height increased by 1.1±0.4 SDS. In 50 patients who reached near adult height (NAH) after an average of 7.1 years of rhGH therapy, height increased from -3.1±0.7 SDS at start to -1.6±1.2 SDS at NAH. The dose of response in the IGF1GT had no direct correlation with outcome, but better growth responses (NAH) were seen with earlier age at start of treatment.
Conclusion: Patients with short stature, reduced IGF-I levels, normal stimulated GH response, and a positive IGFIGT respond well to rhGH therapy. The IGFIGT may identify a subgroup among children with short stature that benefit from rhGH therapy.
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