ESPE2023 Poster Category 1 GH and IGFs (48 abstracts)
Withdrawing growth hormone treatment at mid-puberty in idiopathic isolated growth hormone deficiency: baseline characteristics in patient-preference design study
Joeri Vliegenthart 1 , J.M. Wit 2 , B. Bakker 3 , A.M. Boot 4 , C. de Bruin 2 , M.J.J. Finken 5 , J.C. van der Heyden 6 , E.C.A.M. Houdijk 7 , H.J. van der Kamp 8 , E.G.A.H. van Mil 9 , A.M.J. Reedijk 10 , T.C.J. Sas 1 , D.A. Schott 11 , P. van Setten 12 , S. Straetemans 13 , V. van Tellingen 14 , R.N.H. Touwslager 15 , A.S.P van Trotsenburg 5 , P.G. Voorhoeve 16 & D.C.M. van der Kaay 1
1Erasmus University Medical Centre, Sophia Children's Hospital, Rotterdam, Netherlands. 2Willem-Alexander Children's Hospital, Leiden University Medical Centre, Leiden, Netherlands. 3Reinier de Graaf Gasthuis, Delft, Netherlands. 4Beatrix Children's Hospital, University Medical Centre Groningen, Groningen, Netherlands. 5Emma Children's Hospital, Amsterdam University Medical Center, Amsterdam, Netherlands. 6Franciscus Gasthuis en Vlietland, Rotterdam, Netherlands. 7Juliana Children's Hospital, Haga Teaching Hospital, The Hague, Netherlands. 8Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, Netherlands. 9Jeroen Bosch Hospital, ‘s-Hertogenbosch, Netherlands. 10Dutch Growth Research Foundation, Rotterdam, Netherlands. 11Zuyderland Hospital, Heerlen, Netherlands. 12Amalia Children’s 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
Background: The majority of children diagnosed with idiopathic isolated growth hormone deficiency (IIGHD) show a normal growth hormone (GH) secretion (assessed by GH stimulation tests) when retested at near adult height (NAH). It appears plausible that if normal stimulated GH secretion is observed in mid-puberty, continuing recombinant human GH (rhGH) treatment may only have a minor effect on NAH. The effect on NAH has never been investigated in a prospective study.
Aim: To evaluate patient preference in the choice to (dis-)continue GH treatment from mid-puberty, and to study whether patients who discontinued treatment differed in baseline characteristics from those who continued treatment.
Methods: The study population consists of adolescents who were diagnosed in childhood with IIGHD (GH peak at diagnosis between 1.7 and 10 mg/l; <1.7 mg/l was excluded) and who had started rhGH therapy between 2005-2018. According to the national treatment protocol, GH secretion was retested in mid-puberty (males: Tanner stage G3/4, testicular volume >12 ml and bone age (BA) 13-16 years; females: Tanner stage B3/4 and BA 11-14 years). In this multicentre prospective patient-preference design study, adolescents who tested GH sufficient at mid-puberty (GH peak of >6.7 mg/l) had the choice to discontinue or continue rhGH treatment until NAH (height velocity <2cm/year).
Results: In total 126 patients (94 male, 75%) participated in this study. Forty-three patients (34%) chose to continue GH treatment until NAH (group 1), and 83 patients (66%) chose to discontinue GH treatment until NAH (group 2). Baseline data were available for all patients. Mean height at inclusion was significantly higher in group 2 (group 1: -0.78±0.68 SDS vs group 2: -0.48±0.85 SDS; P=0.042). Target height SDS (-0.73±0.53 SDS and -0.58±0.53 SDS, respectively (P=0.14)) and age at inclusion (14.0±1.2y and 13.8±1.1y, respectively (P=0.81)) did not differ between groups. Males and females did not differ in these characteristics. Currently, 72 patients (57%) have reached NAH.
Conclusions: The majority of patients (66%) preferred to discontinue GH treatment after sufficient GH retesting in mid-puberty. Adolescents who chose to discontinue treatment were taller at baseline. The between-group difference in height SDS at inclusion is too large to show statistically significant “non-inferiority” of discontinuing GH at mid-puberty. A statistical model of expected height gain during GH treatment from mid-puberty until NAH will be constructed from a historic control group with IIGHD who showed normal GH secretion upon retesting at NAH.
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