ESPE Abstracts (2024) 98 RFC7.5

1Erasmus University Medical Centre, Sophia Children's Hospital, Rotterdam, Netherlands. 2Erasmus University Medical Centre, Rotterdam, Netherlands. 3Leiden University Medical Centre, Willem-Alexander Children's Hospital, Leiden, Netherlands. 4Reinier de Graaf Gasthuis, Delft, Netherlands. 5University Medical Centre Groningen, Beatrix Children's Hospital, Groningen, Netherlands. 6Amsterdam University Medical Center, Emma Children's Hospital, Amsterdam, Netherlands. 7Franciscus Gasthuis & Vlietland, Rotterdam, Netherlands. 8Dutch Growth Research Foundation, Rotterdam, Netherlands. 9University Medical Center Utrecht, Wilhelmina Children's Hospital, Utrecht, Netherlands. 10Juliana Children's Hospital, Haga Teaching Hospital, Den Haag, Netherlands. 11Jeroen Bosch Hospital, ‘s-Hertogenbosch, Netherlands. 12Zuyderland Hospital, Heerlen, Netherlands. 13Radboud University Medical Centre, Amalia Children’s Hospital, Nijmegen, Netherlands. 14Maastricht University Medical Center, MosaKids Children's Hospital, Maastricht, Netherlands. 15Catharina Hospital, Eindhoven, Netherlands. 16St. Antonius Hospital, Nieuwegein, Netherlands. 17Canisius Wilhelmina Hospital, Nijmegen, Netherlands


Introduction: Health related Quality of Life (QoL) outcome is used as an argument when continuation or stopping growth hormone (GH) treatment is considered in patients with idiopathic isolated growth hormone deficiency (IIGHD) who tested GH sufficient in mid-puberty (inclusion moment for this study).

Aim: To assess potential differences in QoL between patients with IIGHD who discontinued treatment and those who continued treatment.

Materials and Methods: Multicenter patient-preference study in patients at mid-puberty who were no longer GH deficient and chose to continue or stop GH treatment until near adult height (NAH). QoL was measured with a short stature specific questionnaire (QoLISSY) completed by patients and their parents. The questionnaire contained three core QoL domains (physical, emotional and social), three additional domains (coping, beliefs and treatment) and two specific domains for parents (future and effects on parents). Scores were transformed from raw scores to 0-100 scores. Higher values indicate better QoL.

Results: In total 127 patients participated: 44 continued GH treatment (GHcont) and 83 stopped treatment (GHstop). Height SDS (GHcont: -0.76±0.69 SDS vs GHstop: -0.47±0.85 SDS; P = 0.06) and age (GHcont: 14.0±1.1y vs GHstop: 13.9±1.1y; P = 0.69) were similar between groups. 74 patients (58%) completed the questionnaire; 22 in the GHcont (50%) and 52 in the GHstop (63%) group. Patient-reported total QoL scores were not significantly different (GHcont: 90.8±10SD vs GHstop: 92.7±11.1SD; P = 0.49), neither were the domain scores. Mean QoL was comparable with age- and sex-adjusted reference values. Parent-reported total QoL score was significantly higher in the GHstop than in the GHcont group (GHstop: 93.3±6.36SD vs GHcont: 85.1±14.7SD; P = 0.02), with significantly higher scores on the social, emotional and future domains.

Conclusion: Fifty-eight percent completed the questionnaires. Similar patient-reported QoL was observed between groups, while parents reported higher QoL when GH treatment was stopped at mid-puberty. These results establish a solid foundation for further exploration of QoL in this health care efficiency study.

Volume 98

62nd Annual ESPE (ESPE 2024)

Liverpool, UK
16 Nov 2024 - 18 Nov 2024

European Society for Paediatric Endocrinology 

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