Laura G González Briceño1,2, Magali Viaud1,3, Jacques Beltrand1,3,4, Isabelle Flechtner1,3, Yamina Dassa1,3, Dinane Samara-Boustani1,3, Caroline Thalassinos1,3, Christian Pauwels1,3, Kanetee Busiah5, Graziella Pinto1,3, Delphine Jaquet1, Michel Polak1,3,4,6. 1. Pediatric Endocrinology, Diabetology and Gynecology Department, Hôpital Universitaire Necker-Enfants Malades [Assistance Publique Hôpitaux de Paris (AP-HP)], Paris, France. 2. European Society for Paediatric Endocrinology (ESPE) Clinical Fellowship, Paris, France. 3. Centre de référence des maladies endocriniennes rares de la croissance et du développement, Paris, France. 4. Université Paris Descartes, Paris, France. 5. Pediatric Endocrinology, Diabetology and Obesity, Centre Hospitalier Universitaire Vaudois, Lausanne, Switzerland. 6. l'Institut des Maladies Génétiques (IMAGINE Institute), Paris, France.
Abstract
OBJECTIVE: Short stature in children and adolescents may lead to social and emotional stress, with negative effects on quality of life (QoL). GH treatment may improve QoL through height normalization. Our objective here was to evaluate general and height-specific QoL after 1 year of GH treatment. DESIGN: Prospective, single-center, observational cohort study. METHODS: Children ≥ 4 years of age starting GH at our center from 2012 to 2015 to treat short stature were studied. Patients with serious diseases, syndromic short stature, or developmental delay were excluded. At treatment initiation and 1 year later, patients and their parents completed the general PedsQL 4.0 and height-specific Quality of Life in Short Stature Youth (QoLiSSY) questionnaires. Correlations between self-report and parent-report scores and between height gain and QoL improvements were assessed based on Pearson correlation coefficients. RESULTS: Seventy-four children (42 boys, 32 girls), median age (± SD), 10.2 ± 3.0 years (range, 4.1 to 16.6 years), were included. The self-report PedsQL indicated significant improvements in emotional (P = 0.02) and social (P = 0.03) QoL. As assessed by the QoLiSSY, children reported improvement of social QoL (+0.2 SD; P = 0.04), and parents reported improvement of children's physical (+0.1 SD; P < 0.0001), emotional (+0.3 SD; P < 0.0001), and social (+0.3 SD; P < 0.0001) QoL. Height SD score (SDS) gains showed moderate positive correlations with QoLISSY self-report score gains (R = 0.53, R2 = 0.28; P < 0.001) and QoLISSY parent-report gains (R = 0.60, R2 = 0.41; P < 0.00001). CONCLUSIONS: After 1 year of GH treatment, children had significant gains in emotional and social QoL, as assessed by a general self-report questionnaire and height-specific parent-report questionnaire.
OBJECTIVE: Short stature in children and adolescents may lead to social and emotional stress, with negative effects on quality of life (QoL). GH treatment may improve QoL through height normalization. Our objective here was to evaluate general and height-specific QoL after 1 year of GH treatment. DESIGN: Prospective, single-center, observational cohort study. METHODS:Children ≥ 4 years of age starting GH at our center from 2012 to 2015 to treat short stature were studied. Patients with serious diseases, syndromic short stature, or developmental delay were excluded. At treatment initiation and 1 year later, patients and their parents completed the general PedsQL 4.0 and height-specific Quality of Life in Short Stature Youth (QoLiSSY) questionnaires. Correlations between self-report and parent-report scores and between height gain and QoL improvements were assessed based on Pearson correlation coefficients. RESULTS: Seventy-four children (42 boys, 32 girls), median age (± SD), 10.2 ± 3.0 years (range, 4.1 to 16.6 years), were included. The self-report PedsQL indicated significant improvements in emotional (P = 0.02) and social (P = 0.03) QoL. As assessed by the QoLiSSY, children reported improvement of social QoL (+0.2 SD; P = 0.04), and parents reported improvement of children's physical (+0.1 SD; P < 0.0001), emotional (+0.3 SD; P < 0.0001), and social (+0.3 SD; P < 0.0001) QoL. Height SD score (SDS) gains showed moderate positive correlations with QoLISSY self-report score gains (R = 0.53, R2 = 0.28; P < 0.001) and QoLISSY parent-report gains (R = 0.60, R2 = 0.41; P < 0.00001). CONCLUSIONS: After 1 year of GH treatment, children had significant gains in emotional and social QoL, as assessed by a general self-report questionnaire and height-specific parent-report questionnaire.
Authors: Meryl Brod; Michael Højby Rasmussen; Suzanne Alolga; Jane F Beck; Donald M Bushnell; Kai Wai Lee; Aristides Maniatis Journal: Pharmacoecon Open Date: 2022-10-18