Quality of life in children and adolescents with growth hormone deficiency and their caregivers: an Italian survey

M Maghnie; M Orso; B Polistena; M Cappa; G Pozzobon; D d'Angela; G Patti; F Spandonaro; S Granato; R Di Virgilio; D La Torre; M Salerno

doi:10.1007/s40618-023-02106-3

Quality of life in children and adolescents with growth hormone deficiency and their caregivers: an Italian survey

J Endocrinol Invest. 2023 Dec;46(12):2513-2523. doi: 10.1007/s40618-023-02106-3. Epub 2023 May 20.

Authors

M Maghnie^{1

2}, M Orso³, B Polistena^{4

5}, M Cappa⁶, G Pozzobon⁷, D d'Angela^{4

5}, G Patti^{1

2}, F Spandonaro^{4

8}, S Granato⁹, R Di Virgilio¹⁰, D La Torre¹¹, M Salerno¹²

Affiliations

¹ Paediatric Clinic and Endocrinology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
² Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy.
³ C.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy. massi.orso@hotmail.it.
⁴ C.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.
⁵ University of Rome Tor Vergata, Rome, Italy.
⁶ Unit of Endocrinology, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
⁷ Pediatric Clinic, IRCCS San Raffaele Hospital, Università Vita-Salute San Raffaele, Milan, Italy.
⁸ San Raffaele University, Rome, Italy.
⁹ Medical Department, Pfizer Italia, Rome, Italy.
¹⁰ Health and Value, Pfizer Italia, Rome, Italy.
¹¹ Global Medical Affairs, Pfizer Rare Disease, Rome, Italy.
¹² Department of Translational Medical Sciences, Paediatric Endocrinology Unit, University of Naples 'Federico II', Naples, Italy.

Abstract

Purpose: The aim of this study was to produce evidence on quality of life (QoL) among Italian growth hormone deficiency (GHD) children and adolescents treated with growth hormone (GH) and their parents.

Methods: A survey was conducted among Italian children and adolescents aged 4-18 with a confirmed diagnosis of GHD and treated with GH therapy and their parents. The European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3L) and the Quality of Life in Short Stature Youth (QoLISSY) questionnaires were administered between May and October 2021 through the Computer-Assisted Personal Interview (CAPI) method. Results were compared with national and international reference values.

Results: The survey included 142 GHD children/adolescents and their parents. The mean EQ-5D-3L score was 0.95 [standard deviation (SD) 0.09], while the mean visual analogue scale (VAS) score was 86.2 (SD 14.2); the scores are similar to those of a reference Italian population aged 18-24 of healthy subjects. As for the QoLISSY child-version, compared to the international reference values for GHD/ idiopathic short stature (ISS) patients, we found a significantly higher score for the physical domain, and lower scores for coping and treatment; compared to the specific reference values for GHD patients, our mean scores were significantly lower for all domains except the physical one. As for the parents, we found a significantly higher score for the physical domain, and a lower score for treatment; compared to reference values GHD-specific, we found lower score in the social, emotional, treatment, parental effects, and total score domains.

Conclusions: Our results suggest that the generic health-related quality of life (HRQoL) in treated GHD patients is high, comparable to that of healthy people. The QoL elicited by a disease specific questionnaire is also good, and comparable with that of international reference values of GHD/ISS patients.

Keywords: Children; Growth hormone deficiency; Italy; Quality of life; Survey.

MeSH terms

Adolescent
Caregivers
Dwarfism, Pituitary* / drug therapy
Dwarfism, Pituitary* / epidemiology
Dwarfism, Pituitary* / psychology
Growth Hormone
Human Growth Hormone* / therapeutic use
Humans
Italy / epidemiology
Quality of Life / psychology
Surveys and Questionnaires

Substances

Human Growth Hormone
Growth Hormone