ESPE Abstracts (2015) 84 P-2-469

Major Improvement in Parental Perception of their Children's Height-Specific Quality of Life after 1 Year of GH Treatment: Our Experience with the QoLiSSY Questionnaire

Laura Gabriela González-Briceñoa,b, Magali Viauda,c, Isabelle Flechtnera,c, Yamina Dassaa,c, Dinane Samara-Boustania,c, Caroline Thalassinosa,c, Christian Pauwelsa,c, Jacques Beltranda,c, Kanetee Busiaha,c, Graziella Pintoa,c & Michel Polaka,c

aAP-HP, Hôpital Universitaire Necker Enfants Malades, Endocrinologie, gynécologie et diabétologie pédiatrique, Université Paris Descartes, Institut IMAGINE (affiliate), Paris, France; bESPE Clinical Fellowship, Paris, France; cCentre de référence des maladies endocriniennes rares de la croissance (CRMERC), Paris, France

Background: Short stature may be a source of social and affective stress in children and their parents, and thus impact negatively on their quality of life (QoL). Treatment by GH may improve QoL through normalisation of height.

Objective: To evaluate height-specific QoL in short stature children after 1 year of GH treatment.

Methods: Prospective study. Inclusion criteria were: having started GH treatment at Hôpital Necker-Enfants Malades between April 2012 and December 2014, age ≥ 4 y.o and short stature (≤−2 SDS). Exclusion criteria were the presence of a serious chronic disease (e.g. brain tumour, idiopathic juvenile arthritis), syndromic cause (e.g. Turner syndrome, Prader–Willi syndrome), or developmental delay. Two questionnaires (general PedsQoL 4.0 and height-specific QoLISSY) were completed on the day of first GH injection (T0) and one year later (T12), both in parents and in children. Paired t-test was used to evaluate changes in QoL.

Results: Of 66 patients who filled in the T0 questionnaires, 30 have completed the T12 evaluation, 12 girls and 18 boys. Median age: 10.3 y.o. (4.2–13.7). In PedsQoL questionnaires, parents reported a significant improvement of their child’s emotional QoL (P=0.009). In QoLISSY scores, a very statistically significant improvement was noted in parental perception of their child’s physical (P=0.006), emotional (P=0.002) and social (P=0.006) QoL. Children’s questionnaires showed a significant improvement in their beliefs concerning importance of height (P=0.015), and a trend towards improvement of their emotional (P=0.051) and social (P=0.058) QoL. There was no correlation between height gain in SDS (mean: +0.8 SDS) and improvement in QoL.

Conclusions: Our preliminary results show that after one year of treatment, children’s height-specific QoL (physical, emotional, social) is significantly improved, according to parental perception. Results obtained after completion of the study will further clarify if there exists a change in children’s QoL.

Funding: This study was supported partially by Pfizer.