ESPE Abstracts (2023) 97 P1-103

11st Pediatric Department, Ippokratio General Hospital, Thessaloniki, Greece. 2Aristotle University of Thessaloniki, Thessaloniki, Greece. 32nd Department of Pediatrics, Aristotle University of Thessaloniki, Thessaloniki, Greece. 4AΗEPA University General Hospital, Thessaloniki, Greece. 5University Pediatric Clinic, Attikon Hospital, Athens, Greece. 6Department of Pediatrics, University Hospital of Patras, Patras, Greece. 7Division of Endocrinology, Diabetes and Metabolism and Aghia Sophia Children’s Hospital Endo-ERN representative, Athens, Greece. 81st Department of Pediatrics, Medical School, National and Kapodistrian University of Athens, Aghia Sofia Children’s Hospital, Athens, Greece. 9Pfizer Hellas S.A., Athens, Greece. 10Pfizer Corporation Austria Gesellschaft M.B.H., Vienna, Austria

Background: Pediatric growth hormone deficiency (pGHD) is associated with growth attenuation/deceleration, short stature, delayed bone maturation, and metabolic defects. Daily recombinant human growth hormone (rhGH) treatment promotes linear growth and increases growth rate; however, it may present a substantial burden for patients and caregivers. This may lead to low adherence, limiting the clinical effect of treatment. This study assesses the health-related quality of life (HRQoL) of children and adolescents with GHD, as well as rhGH treatment burden for patients and caregivers in Greece.

Methods: This ongoing cross-sectional study enrolls patients aged 3-17 years receiving daily rhGH for at least 12 months. The Quality of Life in Short Stature Youth (QoLISSY) questionnaire assessed patients’ HRQoL (higher score denoting better HRQoL) from both patients’ and caregivers’ perspective, whereas the Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD) evaluated patients’ and caregivers’ treatment burden (higher score denoting greater life interference).

Results: Overall, 84 patients (77 aged >8 years) from 5 pediatric endocrinology outpatient clinics were included in this interim analysis (July 2022-January 2023). The mean QoLISSY scores reported from patients and caregivers were 81.18 and 74.92, respectively. Coping was the lowest scored domain in both groups, followed by Treatment and Beliefs domains (Table 1). The LIQ-GHD results were similar to literature reports, revealing a mean (SD) overall patient life interference score of 22.36 (±19.36) with no substantial differences among age groups. The mean (SD) caregiver life interference score was 15.86 (±18.48). In a four-week period, 46.43% of patients missed ≥1 injections. Adolescents (>12 years) demonstrated lower adherence than patients 8-12 and <8 years of age (47.62% vs. 60.00% vs. 57.14%, respectively).

Table 1: Mean overall QoLISSY scores for each domain
QoLISSY scores Overall (patient report*) n= 77 Mean (SD) Overall (caregiver report**) n= 84 Mean (SD)
Total 81.18 (±15.05) 74.92 (±18.63)
Physical domain 82.25 (±15.30) 78.37 (±19.28)
Social domain 80.64 (±18.56) 75.74 (±21.63)
Emotional domain 80.64 (±17.01) 70.65 (±20.13)
Coping domain 46.88 (±23.51) 50.54 (±20.39)
Beliefs domain 70.45 (±27.41) 66.67 (±27.43)
Treatment domain 56.19 (±21.36) 61.37 (±19.90)
SD: standard deviation; *patients aged >8 years; **patients aged 3-17 years

Conclusion: This analysis showed an overall good HRQoL and a moderate treatment burden for patients receiving daily rhGH and their caregivers. However, sub-optimal adherence rates may affect clinical outcomes. New treatment options could further improve HRQoL and treatment experience, as well as increase adherence rates that may lead to better health outcomes.

Volume 97

61st Annual ESPE (ESPE 2023)

The Hague, Netherlands
21 Sep 2023 - 23 Sep 2023

European Society for Paediatric Endocrinology 

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