Joeri Vliegenthart1, J.M. Wit2, B. Bakker3, A.M. Boot4, C. de Bruin4, M.J.J. Finken5, J.C. van der Heyden6, A.C.S. Hokken-Koelega7, E.C.A.M. Houdijk8, H.J. van der Kamp9, E.G.A.H. van Mil10, A. Reedijk10, T.C.J. Sas11, D.A. Schott12, P. van Setten13, S. Straetemans14, V. van Tellingen15, R.N.H. Touwslager16, A.S.P. van Trotsenburg16, P.G. Voorhoeve17, D.C.M. van der Kaay18,
(1) Division of Pediatric Endocrinology, Department of Pediatrics, Erasmus University Medical Centre, Sophia Children\’s Hospital, Rotterdam, The Netherlands. (2) Division of Pediatric Endocrinology, Department of Pediatrics, Willem-Alexander Children\’s Hospital, Leiden University Medical Centre, Leiden, The Netherlands. (3) Division of Pediatric Endocrinology, Department of Pediatrics, Reinier de Graaf Gasthuis, Delft, The Netherlands. (4) Division of Pediatric Endocrinology, Beatrix Children\’s Hospital, University Medical Centre Groningen, Groningen, The Netherlands. (5) Division of Pediatric Endocrinology, Department of Pediatrics, Emma Children\’s Hospital, Amsterdam University Medical Center, University of Amsterdam, Amsterdam, The Netherlands. (6) Division of Pediatric Endocrinology, Department of Pediatrics, Franciscus Gasthuis en Vlietland, Rotterdam, The Netherlands. (7) Dutch Growth Research Foundation, Rotterdam, The Netherlands. (8) Division of Pediatric Endocrinology, Department of Pediatrics, Juliana Children\’s Hospital, Haga Teaching Hospital, The Hague, The Netherlands. (9) Division of Pediatric Endocrinology, Department of Pediatrics, Division of Endocrinology, Wilhelmina Children\’s Hospital, University Medical Center Utrecht, Utrecht, The Netherlands. (10) Division of Pediatric Endocrinology, Department of Pediatrics, Jeroen Bosch Hospital, ‘s-Hertogenbosch, The Netherlands. (11) Division of Pediatric Endocrinology, Department of Pediatrics, Erasmus University Medical Centre, Sophia Children\’s Hospital, Rotterdam, The Netherlands. (12) Division of Pediatric Endocrinology, Department of Pediatrics, Zuyderland Hospital, Heerlen, The Netherlands. (13) Division of Pediatric Endocrinology, Department of Pediatrics, Amalia Children’s Hospital, Radboud University Medical Center, Nijmegen, The Netherlands. (14) Division of Pediatric Endocrinology, Department of Pediatrics, division of Endocrinology, Maastricht university medical center, Maastricht, The Netherlands. (15) Division of Pediatric Endocrinology, Department of Pediatrics, Catharina Hospital, Eindhoven, The Netherlands. (16) Division of Pediatric Endocrinology, Department of Pediatrics, St. Antonius Hospital, Nieuwegein, The Netherlands. (17) Division of Pediatric Endocrinology, Department of Pediatrics, Canisius Wilhelmina Hospital, Nijmegen, The Netherlands. (18) Division of Pediatric Endocrinology, Department of Pediatrics, Erasmus University Medical Centre, Sophia Children\’s Hospital, Rotterdam, The Netherlands.
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.
Aims: 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.
The study population consisted of adolescents who were diagnosed in childhood with IIGHD (GH peak at diagnosis between 1.7 ug/L and 10 ug/L) 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 >6.7 ug/L) had the choice to discontinue or continue rhGH treatment until NAH (height velocity <2cm/year).
In total 127 patients (95 male, 75%) participated in this study. 44 patients (35%) chose to continue GH treatment until NAH (GHcont), and 83 patients (65%) chose to discontinue GH treatment (GHstop). Mean height at inclusion (GHcont: -0.76±0.69 SDS vs GHstop: -0.47±0.85 SDS; P=0.06), target height SDS (GHcont: -0.73±0.54 SDS vs GHstop: -0.58±0.53 SDS, P=0.15), mean height SDS minus TH-SDS (GHcont: -0.03±0.69 SDS vs GHstop: 0.11±0.77 SDS, P=0.31) and age at inclusion (GHcont: 14.0±1.1y vs GHstop: 13.9±1.1y, P=0.69) did not significantly differ between groups.
The majority of patients (65%) preferred to discontinue rhGH treatment after sufficient GH retesting in mid-puberty. An anticipated difference in baseline data between the groups could not be confirmed. Data are being collected until all patients reach NAH.