HRQoL of European children and adolescents with short stature as assessed with generic (KIDSCREEN) and chronic-generic (DISABKIDS) instruments

Abstract

Short stature may be associated with impairments in health-related quality of life (HRQoL). This study compared the HRQoL of children/adolescents diagnosed with short stature to population norms and examined the effects of height deviation and treatment status on HRQoL. Method: We cross-sectionally assessed 110 children/adolescents aged 8–18, with current short stature (height deviation ≤-2 standard-deviation (SD)) or normal height achieved since diagnosis, and 98 parents, using the generic KIDSCREEN and the chronic-generic DISABKIDS instruments. Results: Generic HRQoL of patients was similar to population norms. Patients with achieved normal height reported better chronic-generic HRQoL when untreated, while patients with current short stature reported better HRQoL upon receiving treatment. Parents reported better HRQoL for treated patients, especially for girls. Conclusion: Although their HRQoL is not significantly compromised, patients diagnosed with short stature may profit from growth-hormone treatment. Specific instruments are needed to adequately assess the effectiveness of treatment.

Keywords::

• children and adolescents
• growth-hormone treatment
• health-related quality of life
• patient- and proxy-reported outcomes
• short stature

Acknowledgements

This study is part of the ‘Quality of Life in Short Stature Youth’ (QoLISSY) project, a joint initiative between the University Medical Centre Hamburg-Eppendorf and ©Pfizer Ltd. The QoLISSY study group, in addition to the German team of M Bullinger, J Quitmann and A Rohenkohl (University Medical Centre Hamburg Eppendorf, Department of Medical Psychology, Hamburg, Germany), includes J E Chaplin (Sahlgrenska Academy at University of Gothenburg, Department of Paediatrics, Växthuset, Queen Silvia’s Children’s Hospital, Gothenburg, Sweden), M Herdman (IMIM University, Insight Consulting and Research, Barcelona, Spain), E Mimoun (University of Toulouse, Department of Pediatric Endocrinology, Toulouse, France), K DeBusk (University of Edinburgh, Department of Clinical Psychology, Edinburgh, Scotland), E Feigerlova (University of Toulouse, Department of Pediatric Endocrinology, Toulouse, France), C Lunde (Sahlgrenska Academy at University of Gothenburg, Department of Pediatrics, Växthuset, Queen Silvia’s Children’s Hospital, Gothenburg, Sweden), M Dellenmark-Blom (Sahlgrenska Academy at University of Gothenburg, Department of Pediatrics, Växthuset, Queen Silvia’s Children’s Hospital, Gothenburg, Sweden), D Sanz (IMIM University, Insight Consulting and Research, Barcelona, Spain), A Pleil (Pfizer, Inc. Specialty Care MDG, Outcomes Research, San Diego, CA), H Wollmann (Pfizer Ltd., Specialty Care MDG, Endocrinology, Walton Oaks, UK) and M Power (University of Edinburgh, Department of Clinical Psychology, Edinburgh, Scotland).

Financial & competing interests disclosure:

The development of the QoLISSY questionnaire was funded through a research grant by Pfizer Ltd. M Bullinger has received honoraria and acted as consultant. Two members of the QoLISSY group are Pfizer Employees. All other authors have no involvement with Pfizer Ltd. nor have financial interests. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. N Silva was supported by a PhD Grant from the Portuguese Foundation for Science and Technology (SFRH/BD/69885/2010). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Key issues

• • Health-related quality of life (HRQoL) is an important patient-reported outcome in pediatric endocrinology which should be obtained from young patients and supplemented by parents.

• • Studies investigating HRQoL impairments in short statured children and adolescents have yielded inconsistent results as regards the comparison to population norms/healthy controls and across patient clinical characteristics.

• • HRQoL assessment in young patients with short stature profits from a modular approach, including generic, chronic-generic and condition-specific instruments, the latter of which have only recently been developed.

• • Generic instruments may underestimate the patient’s burden, while chronic-generic measures may detect more subtle impairments on the basis of clinical and socio-demographic characteristics.

• • Analysis of the cross-sectional data suggests better HRQoL for recombinant human growth hormone-treated patients with current height ≤-2 standard-deviation as compared to treated children who have achieved a normal height.

• • The variability in HRQoL of short statured children/adolescents is better explained by recombinant human growth hormone treatment status than by patients’ developmental characteristics.

• • Patient’s and parent’s HRQoL ratings diverged, with parents observing poorer HRQoL as compared to patient self-assessments.

• • Psychosocial interventions may be helpful to improve adaptation and consequently HRQoL of patients but intervention programs still need to be developed, implemented and evaluated.