Quality of life before and after 12-months replacement therapy in children with growth hormone deficiency





quality of life, children, growth hormone deficiency, rhGH therapy


A systematic review of literature in the Embase, MEDLINE, and Cochrane databases reveals incomplete and contradictory data on the psychosocial consequences of short stature in children, including its impact on quality of life (QoL).

The aim of this study was to evaluate the effect of recombinant human growth hormone (rhGH) therapy on the QoL of children diagnosed with growth hormone deficiency (GHD). The QoL was considered as an indicator of psychological and social adaptation of the children.

Materials and methods. A main group (46 prepubescent children with GHD) was selected and compared to a matched control group (80 healthy children). The group of children with short stature included all children with GHD of the main group and 48 children with idiopathic short stature.The Peds QL4.0 questionnaires for children and parents were used to determine QoL on the scales of “total score”, “physical health”, “psycho-social health”, “emotional functioning”, “social functioning”, and “school functioning”. Statistical processing of the results using the Spearman's correlation coefficient and by estimating the differences between the mean values of two independent variation series by the value of "p" was performed.

Results. A more pronounced decrease in QoL was found in children with GHD if they are under 9 years of age  compared to their older peers and in children with height SDS<- 3 compared to those with height SDS between -3 and -2. After 12 months of rhGH treatment, the normalization of global QоL score was found to be associated with optimal growth response, as measured by the Peds QL4.0 scale (rs=0.44, p<0.05). The study also revealed a direct correlation between adherence to replacement therapy and QоL (rs=0.28, p<0.05). Furthermore, there was a positive correlation between high compliance with rhGH therapy and optimal growth response (rs=0.85, p<0.05).

Conclusions. The study found that there was a difference in the scores of the Peds QL 4.0 children's questionnaire and the Peds QL 4.0 parents' questionnaire in terms of awareness of physical health problems. However, both questionnaires indicated that after 12 months of rhGH therapy, all scales of the Peds QL 4.0 were normalized. Furthermore, a positive correlation was found between the QоL of children with GHD and their optimal growth response to rhGH therapy.

The research was carried out in accordance with the principles of the Helsinki Declaration. The study protocol was approved by the Local Ethics Committee of the participating institution. The informed consent of the patient was obtained for conducting the studies.

No conflict of interests was declared by the authors.


Altman DG. (2018). Practical Statistics for Medical Research. London: Chapman and Hall: 611.

Aryayev M, Senkivska L. (2022). Growth response, psychosocial problems, and quality of life in children with growth hormone deficiency. Pediatria Polska - Polish Journal of Paediatrics. 97(3): 236-241. https://doi.org/10.5114/polp.2022.120074

Aryayev M, Senkivska L, Lowe JB. (2021). Psycho-Emotional and Behavioral Problems in Children with Growth Hormone Deficiency. Front. Pediatr. 9: 707648. https://doi.org/10.3389/fped.2021.707648; PMid:34631612 PMCid:PMC8495251

Backeljauw P, Cappa M, Kiess W, Law L, Cookson C, Sert C et al. (2021). Impact of short stature on quality of life: A systematic literature review. Growth hormone & IGF research: official journal of the Growth Hormone Research Society and the International IGF Research Society. 57-58: 101392. https://doi.org/10.1016/j.ghir.2021.101392; PMid:33975197

Bloemeke J, Silva N, Bullinger M, Witt S, Dörr HG, Quitmann J. (2019, Mar 18). Psychometric properties of the quality of life in short statured youth (QoLISSY) questionnaire within the course of growth hormone treatment. Health Qual Life Outcomes. 17(1): 49. https://doi.org/10.1186/s12955-019-1118-9; PMid:30885197 PMCid:PMC6423839

Brod M, Rasmussen MH, Alolga S, Beck JF, Bushnell DM, Lee KW, Maniatis A. (2023). Psychometric Validation of the Growth Hormone Deficiency-Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB). PharmacoEconomics - open. 7(1): 121-138. https://doi.org/10.1007/s41669-022-00373-z; PMid:36255609 PMCid:PMC9929004

Gardner M, Boshart ML, Yeguez CE, Desai KM, Sandberg DE. (2016). Coming Up Short: Risks of Bias in Assessing Psychological Outcomes in Growth Hormone Therapy for Short Stature. The Journal of clinical endocrinology and metabolismю 101(1): 23-30. https://doi.org/10.1210/jc.2015-3256; PMid:26583584

González Briceño LG, Viaud M, Beltrand J et al. (2019). Improved General and Height-Specific Quality of Life in Children with Short Stature After 1 Year on Growth Hormone. The Journal of Clinical Endocrinology & Metabolism. 104(6): 2103-2111. https://doi.org/10.1210/jc.2018-02523; PMid:30649493

Maghnie M, Orso M, Polistena B, Cappa M, Pozzobon G, d'Angela D et al. (2023). Quality of life in children and adolescents with growth hormone deficiency and their caregivers: an Italian survey. Journal of endocrinological investigation. 46(12): 2513-2523. https://doi.org/10.1007/s40618-023-02106-3; PMid:37209402 PMCid:PMC10632207

Quitmann J, Bloemeke J, Dörr HG et al. (2019). First-year predictors of health-related quality of life changes in short-statured children treated with human growth hormone. J. Endocrinol Invest. 42: 1067-1076. https://doi.org/10.1007/s40618-019-01027-4; PMid:30840207

UNICEF & WHO (2020). Levels and trends in child malnutrition: key findings of the 2019 Edition of the Joint Child Malnutrition Estimates. Geneva: World Health Organization.: https://data.unicef.org/topic/nutrition/malnutrition

Varni JW, Limbers CA, Burwinkle TM. (2007). Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales. Health Qual Life Outcomes. 5: 43. https://doi.org/10.1186/1477-7525-5-43; PMid:17634123 PMCid:PMC1964786

Varni JW, Seid M, Knight TS et al. (2002). The PedsQLTM 4.0 Generic Core Scales: Sensitivity, Responsiveness, and Impact on Clinical Decision-Making. J Behav Med. 25(2): 175-193. https://doi.org/10.1023/A:1014836921812; PMid:11977437

Visser-van Balen H, Sinnema G, Geenen R. (2006). Growing up with idiopathic short stature: psychosocial development and hormone treatment; a critical review. Arch Dis Child. 91(5): 433‐439. https://doi.org/10.1136/adc.2005.086942; PMid:16632673 PMCid:PMC2082749

Warrier V, Krishan K, Shedge R, Kanchan T. (2023). Height Assessment. In StatPearls. StatPearls Publishing.





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