Characterization of patients stopping GH therapy for childhood-onset growth hormone deficiency in Belgium - Luxemburg

Laure Boutsen, Muriel Thomas, Willem Staels, Philippe A Lysy, Marianne Becker, Anne Rochtus, Emese Boros, Kathleen De Waele, Hilde Dotrement, Anne-Simone Parent, Daniel Klink, Olimpia Chivu, Guy Massa, Dominique Beckers, Sylvia Depoorter, Nele Reynaert, Karl Logghe, Jean De Schepper

Research output: Contribution to journalMeeting abstract (Journal)

Abstract

Background: Growth hormone deficiency (GHD) in children comes in different etiologies and can be either isolated or combined with other pituitary hormone deficiencies. The diversity in GHD types and the variable duration of therapy complicate assessments of long-term treatment outcome. We characterized GHD patients at the end of GH therapy in Belgium and Luxemburg and evaluated height and adiposity outcomes in relation to GHD type. Methods: Anthropometric and clinical characteristics of children with GHD who stopped GH therapy between January 2019 and December 2021 were retrieved from the binational BeLUX database (BELGROW). Results: In total, 179 (119 male and 60 female) patients stopped GH therapy after a median (P10; P90) duration of 6.8 (2.6; 12.9) years. Eight patients received GH therapy for less than 2 years. In total, 113 (63 %) patients had isolated GHD and 66 (37%) combined GHD. Forty-five had congenital GHD (i.e., identified structural or genetic cause), 45 acquired and 89 (50%) idiopathic. All diagnostic categories showed male preponderance (>66 %). Isolated GHD was most prevalent in idiopathic GHD (93.3 % vs 44.2 % in congenital vs 22.2 % in acquired GHD). Ten patients stopped GH therapy before 13 years of age. Ninety -two male and 25 female patients stopped GH therapy after the age of 16 years at a mean (SD) height of 171.2 (7.3) cm and 159.5 (8.9) cm, respectively. Their mean (SD) height (-1.05 (1.0) vs -1.11 (1.48)) and BMI (0.06 (1.65) vs 0.18 (1.5)) z-scores were comparable. In this subgroup older than 16 years, the 66 patients with isolated GHD had significantly (P= 0.016) lower height z-scores than the 51 patients with combined GHD (-1.28(1.06) vs.-0.78 (1.13). Mean height and BMI z-scores at the end of GH therapy in patients > 16 years were similar between congenital (n=29), acquired GHD (n=32) and idiopathic GHD (n=56), while mean duration of GH therapy was longest in congenital GHD (9.78 (4.3) vs 6.9 (3.8) vs 6.46(3.7) year; P=0.001).
Conclusion: In GHD patients stopping GH therapy in BeLUx, we see a two-fold male preponderance and 1.7-fold preponderance of isolated GHD. Less than 6 % discontinue GH therapy before 13 years or receive less than 2 years of treatment. In those discontinuing GH therapy after 16 years of age, the height was superior in patients with combined GHD.
Original languageEnglish
Article number10.1530/endoabs.99.P523
Number of pages1
JournalEndocrine Abstracts
Volume99
Issue numberP523
Publication statusPublished - May 2024
EventECE 2024 - Stockholm, Sweden
Duration: 11 May 202414 May 2024
https://www.ese-hormones.org/education-and-training/european-congress-of-endocrinology/ece-2024/programme/

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