{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T23:05:21Z","timestamp":1789427121273,"version":"build-2803163510"},"reference-count":17,"publisher":"Georg Thieme Verlag KG","issue":"09","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Horm Metab Res"],"published-print":{"date-parts":[[2026,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>This retrospective real-world cohort study evaluated the effectiveness and safety of recombinant human growth hormone therapy in Chinese children with Noonan syndrome, with a predefined comparison between short-acting growth hormone and long-acting growth hormone during the first 2 years of treatment. Twenty children with Noonan syndrome were included; 16 were boys, and the median age at recombinant human growth hormone initiation was 5.55 years. The baseline height standard deviation score was\u22123.01. During the first 2 years, 10 patients received short-acting growth hormone and 10 patients received long-acting growth hormone. The median height standard deviation score increased to\u22122.20 after 1 year and\u22121.84 after 2 years, with significant longitudinal improvement over time. The cumulative change in height standard deviation score did not differ significantly between short-acting growth hormone and long-acting growth hormone at year 1 (0.58 vs. 0.71) or year 2 (1.01 vs. 1.30). First-year growth velocity was comparable between groups, whereas the apparent second-year difference was no longer significant after excluding short-acting growth hormone-treated patients who entered puberty and received adjunctive therapy. Among 16 patients with PTPN11 variants, the median cumulative change in height standard deviation score was 1.20 and 56.3% achieved an HtSDS of above\u22122 standard deviation score at the last follow-up. No diabetes mellitus, malignancy, new cardiovascular events, intracranial hypertension, or slipped capital femoral epiphysis occurred. Insulin-like growth factor-1 standard deviation score elevations were transient. Recombinant human growth hormone therapy was associated with sustained linear growth in children with Noonan syndrome, and short-acting growth hormone and long-acting growth hormone showed broadly comparable short-term efficacy under regular safety monitoring.<\/jats:p>","DOI":"10.1055\/a-2900-8976","type":"journal-article","created":{"date-parts":[[2026,7,29]],"date-time":"2026-07-29T23:03:53Z","timestamp":1785366233000},"page":"380-386","source":"Crossref","is-referenced-by-count":0,"title":["Real-World Effectiveness and Safety of Recombinant Human Growth Hormone in Chinese Children with Noonan Syndrome: Long-Term Follow-Up and a 2-Year Comparison of Short- and Long-Acting Formulations"],"prefix":"10.1055","volume":"58","author":[{"given":"Xinying","family":"Gao","sequence":"additional","affiliation":[{"name":"Beijing Geriatric Hospital, China","place":["Beijing"],"department":["Department of Pediatrics"]},{"name":"Beijing Children's Hospital, Capital Medical University, China","place":["Beijing"],"department":["Department of Endocrinology, Genetics and Metabolism"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiajia","family":"Chen","sequence":"additional","affiliation":[{"name":"Beijing Children's Hospital, Capital Medical University, China","place":["Beijing"],"department":["Department of Endocrinology, Genetics and Metabolism"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beibei","family":"Zhang","sequence":"additional","affiliation":[{"name":"Beijing Children's Hospital, Capital Medical University, China","place":["Beijing"],"department":["Department of Endocrinology, Genetics and Metabolism"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manman","family":"Zhao","sequence":"additional","affiliation":[{"name":"Beijing Luhe Hospital, China","place":["Beijing"],"department":["Department of Pediatrics"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yaguang","family":"Peng","sequence":"additional","affiliation":[{"name":"Beijing Children\u2019s Hospital, Capital Medical University, China","place":["Beijing"],"department":["Center for Clinical Epidemiology and Evidence-based Medicine"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chunxiu","family":"Gong","sequence":"additional","affiliation":[{"name":"Beijing Children's Hospital, Capital Medical University, China","place":["Beijing"],"department":["Department of Endocrinology, Genetics and Metabolism"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2026,7,29]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1001\/archpedi.1968.02100020377005","article-title":"Hypertelorism with Turner phenotype. A new syndrome with associated congenital heart disease","volume":"116","author":"J A Noonan","year":"1968","journal-title":"Am J Dis Child"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1016\/j.beem.2010.09.002","article-title":"Noonan syndrome and clinically related disorders","volume":"25","author":"M Tartaglia","year":"2011","journal-title":"Best Pract Res Clin Endocrinol Metab"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1007\/s40142-016-0100-7","article-title":"Expansion of the RASopathies","volume":"4","author":"W E Tidyman","year":"2016","journal-title":"Curr Genet Med Rep"},{"key":"ref4","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1016\/S0140-6736(12)61023-X","article-title":"Noonan syndrome","volume":"381","author":"A E Roberts","year":"2013","journal-title":"Lancet"},{"key":"ref5","doi-asserted-by":"crossref","first-page":"176","DOI":"10.6065\/apem.2018.23.4.176","article-title":"Growth hormone therapy in patients with Noonan syndrome","volume":"23","author":"G H Seo","year":"2018","journal-title":"Ann Pediatr Endocrinol Metab"},{"key":"ref6","doi-asserted-by":"crossref","first-page":"761171","DOI":"10.3389\/fendo.2021.761171","article-title":"Growth in children with Noonan syndrome and effects of growth hormone treatment","volume":"12","author":"A Libraro","year":"2021","journal-title":"Front Endocrinol"},{"key":"ref7","doi-asserted-by":"crossref","first-page":"5377","DOI":"10.1210\/jc.2005-0995","article-title":"PTPN11 mutations are associated with mild growth hormone resistance in individuals with Noonan syndrome","volume":"90","author":"G Binder","year":"2005","journal-title":"J Clin Endocrinol Metab"},{"key":"ref8","first-page":"e210615","article-title":"Outcomes in growth hormone-treated Noonan syndrome children: Impact of PTPN11 mutation status","volume":"11","author":"AA L Jorge","year":"2022","journal-title":"Endocr Connect"},{"key":"ref9","doi-asserted-by":"crossref","first-page":"388","DOI":"10.1159\/000339677","article-title":"Response to growth hormone therapy in children with Noonan syndrome: Correlation with or without PTPN11 gene mutation","volume":"77","author":"J H Choi","year":"2012","journal-title":"Horm Res Paediatr"},{"key":"ref10","doi-asserted-by":"crossref","first-page":"2338","DOI":"10.1210\/jc.2008-2094","article-title":"Growth response, near-adult height, and patterns of growth and puberty in patients with Noonan syndrome treated with growth hormone","volume":"94","author":"A A Romano","year":"2009","journal-title":"J Clin Endocrinol Metab"},{"key":"ref11","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1159\/000498859","article-title":"Treatment with growth hormone in Noonan syndrome observed during 25 years of KIGS: Near adult height and outcome prediction","volume":"91","author":"M B Ranke","year":"2019","journal-title":"Horm Res Paediatr"},{"key":"ref12","first-page":"193","article-title":"Near adult height and body mass index changes in growth hormone treated short children with Noonan syndrome: The Belgian experience","volume":"98","author":"J De Schepper","year":"2025","journal-title":"Horm Res Paediatr"},{"key":"ref13","doi-asserted-by":"crossref","first-page":"e1232","DOI":"10.1210\/clinem\/dgae834","article-title":"Long-acting growth hormone therapy in pediatric growth hormone deficiency: A consensus statement","volume":"110","author":"A K Maniatis","year":"2025","journal-title":"J Clin Endocrinol Metab"},{"key":"ref14","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1093\/ejendo\/lvag041","article-title":"Once-weekly somapacitan in children with Noonan syndrome: Randomized controlled phase 3 trial","volume":"194","author":"AA L Jorge","year":"2026","journal-title":"Eur J Endocrinol"},{"key":"ref15","doi-asserted-by":"crossref","first-page":"1209339","DOI":"10.3389\/fendo.2023.1209339","article-title":"Lipid profile in Noonan syndrome and related disorders: Trend by age, sex and genotype","volume":"14","author":"F Tamburrino","year":"2023","journal-title":"Front Endocrinol"},{"key":"ref16","doi-asserted-by":"crossref","first-page":"e59569","DOI":"10.1371\/journal.pone.0059569","article-title":"Construction of a new growth reference for China based on urban Chinese children: comparison with the WHO growth standards","volume":"8","author":"X N Zong","year":"2013","journal-title":"PLoS One"},{"key":"ref17","doi-asserted-by":"publisher","first-page":"3043","DOI":"10.1007\/s12325-026-03603-6","article-title":"Association between socioeconomic indicators and growth hormone treatment patterns for idiopathic short stature in China: A large-scale real-world study","volume":"43","author":"C Gong","year":"2026","journal-title":"Adv Ther"}],"container-title":["Hormone and Metabolic Research"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/a-2900-8976.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T22:41:45Z","timestamp":1789425705000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/a-2900-8976"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,7,29]]},"references-count":17,"journal-issue":{"issue":"09","published-online":{"date-parts":[[2026,9,14]]},"published-print":{"date-parts":[[2026,9]]}},"URL":"https:\/\/doi.org\/10.1055\/a-2900-8976","archive":["Portico","CLOCKSS"],"relation":{},"ISSN":["0018-5043","1439-4286"],"issn-type":[{"value":"0018-5043","type":"print"},{"value":"1439-4286","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,7,29]]}}}