Vis enkel innførsel

dc.contributor.authorRohrer, Tilman R.
dc.contributor.authorAbuzzahab, Jennifer
dc.contributor.authorBackeljauw, Philippe
dc.contributor.authorBirkegård, Anna Camilla
dc.contributor.authorBlair, Joanna
dc.contributor.authorDahlgren, Jovanna
dc.contributor.authorJuliusson, Petur Benedikt
dc.contributor.authorOstrow, Vlady
dc.contributor.authorPietropoli, Alberto
dc.contributor.authorPolak, Michel
dc.contributor.authorRomanov, Alicia
dc.contributor.authorRoss, Judith
dc.contributor.authorSävendahl, Lars
dc.contributor.authorMiller, Bradley S.
dc.date.accessioned2022-04-21T12:57:01Z
dc.date.available2022-04-21T12:57:01Z
dc.date.created2021-03-09T11:22:53Z
dc.date.issued2021
dc.identifier.issn1663-2818
dc.identifier.urihttps://hdl.handle.net/11250/2992045
dc.description.abstractIntroduction: 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.isoengen_US
dc.publisherKarger Publishersen_US
dc.rightsNavngivelse-Ikkekommersiell 4.0 Internasjonal*
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/deed.no*
dc.titleLong-Term Effectiveness and Safety of Childhood Growth Hormone Treatment in Noonan Syndromeen_US
dc.typeJournal articleen_US
dc.typePeer revieweden_US
dc.description.versionpublishedVersionen_US
dc.rights.holderCopyright 2021 The Author(s)en_US
cristin.ispublishedtrue
cristin.fulltextoriginal
cristin.qualitycode1
dc.identifier.doi10.1159/000512429
dc.identifier.cristin1896598
dc.source.journalHormone Research in Paediatricsen_US
dc.source.pagenumber380-395en_US
dc.identifier.citationHormone Research in Paediatrics. 2021, 93 (6), 380-395.en_US
dc.source.volume93en_US
dc.source.issue6en_US


Tilhørende fil(er)

Thumbnail

Denne innførselen finnes i følgende samling(er)

Vis enkel innførsel

Navngivelse-Ikkekommersiell 4.0 Internasjonal
Med mindre annet er angitt, så er denne innførselen lisensiert som Navngivelse-Ikkekommersiell 4.0 Internasjonal