dc.contributor.author | Rohrer, Tilman R. | |
dc.contributor.author | Abuzzahab, Jennifer | |
dc.contributor.author | Backeljauw, Philippe | |
dc.contributor.author | Birkegård, Anna Camilla | |
dc.contributor.author | Blair, Joanna | |
dc.contributor.author | Dahlgren, Jovanna | |
dc.contributor.author | Juliusson, Petur Benedikt | |
dc.contributor.author | Ostrow, Vlady | |
dc.contributor.author | Pietropoli, Alberto | |
dc.contributor.author | Polak, Michel | |
dc.contributor.author | Romanov, Alicia | |
dc.contributor.author | Ross, Judith | |
dc.contributor.author | Sävendahl, Lars | |
dc.contributor.author | Miller, Bradley S. | |
dc.date.accessioned | 2022-04-21T12:57:01Z | |
dc.date.available | 2022-04-21T12:57:01Z | |
dc.date.created | 2021-03-09T11:22:53Z | |
dc.date.issued | 2021 | |
dc.identifier.issn | 1663-2818 | |
dc.identifier.uri | https://hdl.handle.net/11250/2992045 | |
dc.description.abstract | Introduction: Few data exist on long-term growth hormone (GH) treatment in patients with Noonan syndrome (NS). Objective: To evaluate the effectiveness and safety of GH treatment in NS in clinical practice. Methods: Height gain, near-adult height (NAH), and safety were assessed in 2 complementary non-interventional studies: NordiNet® IOS and ANSWER. The safety analysis included 412 patients, and the effectiveness analysis included 84 GH-treated patients (male, n = 67) with ≥4 years’ height standard deviation score (HSDS) data. HSDS was determined using national reference (NR) and NS-specific (NSS) data. Results: The mean (SD) baseline age was 8.38 (3.57) years; HSDS, −2.76 (1.03); GH dose, 41.6 (11.1) µg/kg/day. The mean (SD) HSDS increase from baseline (ΔHSDS) was 0.49 (0.37) (first year), 0.79 (0.58) (second year), and 1.01 (0.60) (third year) (NR). The mean (SD) HSDS at year 3 was −1.66 (1.00) (NR; 1.06 [1.12] [NSS]). Twenty-four patients achieved NAH. The mean (SD) NAH SDS (NR) was −1.51 (0.60) (154.90 [3.21] cm) in females and −1.79 (1.09) (165.61 [7.19] cm) in males; 70.8% (17/24) had NAH SDS ≥ −2. Adverse drug reactions and GH-unrelated serious adverse events (n = 34) were reported in 22/412 (5.3%) patients. Four neoplasms and 3 cases of scoliosis were reported; no cardiovascular adverse events occurred. Conclusions: GH-treated children with NS achieved substantial height gain during the first 3 years of follow-up. Overall, 24 patients achieved NAH, with 70.8% having NAH SDS ≥ –2. There was no evidence to support a higher prevalence of neoplasm, or cardiac or other comorbidities. | en_US |
dc.language.iso | eng | en_US |
dc.publisher | Karger Publishers | en_US |
dc.rights | Navngivelse-Ikkekommersiell 4.0 Internasjonal | * |
dc.rights.uri | http://creativecommons.org/licenses/by-nc/4.0/deed.no | * |
dc.title | Long-Term Effectiveness and Safety of Childhood Growth Hormone Treatment in Noonan Syndrome | en_US |
dc.type | Journal article | en_US |
dc.type | Peer reviewed | en_US |
dc.description.version | publishedVersion | en_US |
dc.rights.holder | Copyright 2021 The Author(s) | en_US |
cristin.ispublished | true | |
cristin.fulltext | original | |
cristin.qualitycode | 1 | |
dc.identifier.doi | 10.1159/000512429 | |
dc.identifier.cristin | 1896598 | |
dc.source.journal | Hormone Research in Paediatrics | en_US |
dc.source.pagenumber | 380-395 | en_US |
dc.identifier.citation | Hormone Research in Paediatrics. 2021, 93 (6), 380-395. | en_US |
dc.source.volume | 93 | en_US |
dc.source.issue | 6 | en_US |