ESPE2024 Poster Category 2 GH and IGFs (22 abstracts)
Quality of Life and Treatment Burden of Greek children and adolescents with growth hormone deficiency
Athanasios Christoforidis 1 , Fotini-Eleni Karachaliou 2 , Assimina Galli-Tsinopoulou 3 , Dionisios Chrysis 4 , Christina Kanaka-Gantenbein 5 , Evangelia Baxevanidi 6 , Ioannis Skiadas 6 , Oresteia Zisimopoulou 6 , Dimitris Tsilakis 6 , Apostolia Poimenidou 6 & Elpis-Athina Vlachopapadopoulou 7
11st Department of Pediatrics, School of Medicine, Faculty of Health Sciences, Aristotle University of Thessaloniki, Ippokratio General Hospital, Thessaloniki, Greece. 2University Pediatric Clinic, Attikon Hospital, Athens, Greece. 32nd Department of Pediatrics, School of Medicine, Faculty of Health Sciences, Aristotle University of Thessaloniki, AHEPA University General Hospital, Thessaloniki, Greece. 4Department of Pediatrics, University Hospital of Patras, Patras, Greece. 5Division of Endocrinology, Diabetes and Metabolism and Aghia Sophia Children’s Hospital Endo-ERN representative, First Department of Pediatrics, Medical School, National and Kapodistrian University of Athens, Aghia Sofia Children’s Hospital, Athens, Greece. 6Pfizer Hellas S.A., Athens, Greece. 7Department of Endocrinology-Growth and Development, Athens General Children's Hospital P. & A. Kyriakou, Athens, Greece
Pediatric growth hormone deficiency (pGHD) is a rare disorder characterized by inadequate growth hormone secretion. Recombinant human growth hormone (rhGH) is the current standard of care; however, daily injections may represent a burden to patients and caregivers, affecting patients’ health-related quality of life (HRQoL). This study evaluated the HRQoL of Greek patients receiving daily rhGH, and the burden of patients and caregivers. This cross-sectional study enrolled patients with pGHD aged 3-17 years receiving daily rhGH for at least 12 months, and caregivers. Two questionnaires were administered to both: The Quality of Life in Short Stature Youth (QoLISSY) assessing patients’ HRQoL (higher score denoting better HRQoL) and the dyad-administered Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD) evaluating treatment burden (higher score denoting greater life interference). This study (July 2022–October 2023) included 250 patients (237 aged ≥8 years) and caregivers from 6 pediatric endocrinology clinics. Τhe mean (SD) age of diagnosis was 7.80 (±3.22) years. Time to treatment initiation was 4.96 (±7.54) months. Patients aged ≥8 years reported higher QoLISSY scores than caregivers: Least Squares Mean (LSM) difference 3.47 (P = 0.040) in 8-12 years and 4.55 (P = 0.001) in >12 years. Among LIQ-GHD domains, EoIS, LI, and WtC mainly comprised the burden. PEoU and EoIS appeared higher in patients <8 years (Table 1). Injection Signs and Symptoms burden was higher in 8-12 years than >12 years (P = 0.005). In the CLI/FLI domain, burden was associated with age groups (P = 0.021), with FLI subdomain driving this association (P = 0.002). This study revealed a better HRQoL perceived by patients than caregivers. A moderate treatment burden was reported, mainly attributed to EoIS, LI, and WtC domains. Εvaluation of HRQoL and burden may help pGHD management and treatment decisions.
| Domain Scores Mean (SD) | Overall n = 250 | <8 years n = 13 | 8-12 years n = 93 | >12 years n = 144 |
| PEoU | 8.34(±13.95) | 13.08(±14.94) | 9.41(±13.93) | 7.22(±13.83) |
| EoIS | 19.88(±22.02) | 25.38(±21.06) | 21.08(±22.53) | 18.61(±21.79) |
| LI | 19.36(±18.39) | 17.86(±13.83) | 20.01(±20.59) | 19.07(±17.31) |
| WtC | 19.25(±19.60) | 18.27(±14.98) | 18.41(±18.94) | 19.88(±20.45) |
| SS | 16.91(±14.66) | - | 20.22(±15.36) | 14.77(±13.83) |
| CS | 7.31(±6.96) | 7.31(±6.96) | - | - |
| CLI/FLI | 11.87(±14.87) | 9.47(±10.81) | 15.26(±17.84) | 9.90(±12.60) |
| SD: standard deviation; PEoU: Pen Ease of Use; EoIS: Ease of Injection Schedule; LI: Life Interference; WtC: Willingness to Continue; SS: Injection Signs and Symptoms reported by patients ≥8 years; CS: Injection Signs reported by caregivers for patients <8 years; CLI/FLI: Caregiver/Family Life Interference | ||||
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