Noonan syndrome and Turner syndrome patients respond similarly to 4 years' growth-hormone therapy: longitudinal analysis of growth-hormone-naïve patients enrolled in the NordiNet® International Outcome Study and the ANSWER Program

International Journal of Pediatric Endocrinology
Peter A LeeHenrik T Christesen

Abstract

Turner syndrome (TS) and Noonan syndrome (NS) are distinct syndromes associated with short stature and other similar phenotypic features. We compared the responses to growth hormone (GH) therapy of TS and NS patients enrolled in the NordiNet® International Outcome Study (IOS) or the American Norditropin Studies: Web-Enabled Research (ANSWER) Program, which collect information on GH therapy in clinical practice. Repeated-measures regression analysis was performed on change in height standard deviation score (HSDS) and target-height-corrected HSDS, based on national normal references and treatment-naïve disease-specific references. Models were adjusted for baseline age and HSDS, and average GH dose. The study population was paediatric patients with TS and NS in the NordiNet® IOS and ANSWER Program. Longitudinal growth responses over 4 years were evaluated. In 30 NS patients (24 males; baseline age 8.39 ± 3.45 years) and 294 TS patients (7.81 ± 3.22 years), 4-year adjusted ΔHSDS were +1.14 ± 0.13 and +1.03 ± 0.04, respectively (national references). Based on untreated, disease-specific references, 4-year adjusted ΔHSDS for NS and TS were +1.48 ± 0.10 and +1.79 ± 0.04. The analyses showed a significant increase in HSDS over time fo...Continue Reading

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Citations

May 18, 2016·Anales de pediatría : publicación oficial de la Asociación Española de Pediatría (A.E.P.)·Silvia Beatriz Sánchez MarcoJesús María Garagorri Otero
Apr 14, 2018·Journal of Pediatric Endocrinology & Metabolism : JPEM·Michel PolakMarta Šnajderová
Aug 25, 2018·Endocrine Connections·Robert RapaportGiuseppe Piccoli
Jul 16, 2019·The Journal of Clinical Endocrinology and Metabolism·Lars SävendahlJudith Ross
Aug 15, 2017·Clinical Pediatric Endocrinology : Case Reports and Clinical Investigations : Official Journal of the Japanese Society for Pediatric Endocrinology·Tsuyoshi IsojimaSusumu Yokoya
Jun 22, 2021·Frontiers in Endocrinology·Fernando RodríguezFernando Cassorla

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NCT00960128
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