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Status: Bibliographieeintrag

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Verfasst von:Feillet, François [VerfasserIn]   i
 Ficicioglu, Can [VerfasserIn]   i
 Lagler, Florian B. [VerfasserIn]   i
 Longo, Nicola [VerfasserIn]   i
 Muntau, Ania C. [VerfasserIn]   i
 Burlina, Alberto [VerfasserIn]   i
 Trefz, Friedrich K. [VerfasserIn]   i
 van Spronsen, Francjan J. [VerfasserIn]   i
 Arnoux, Jean-Baptiste [VerfasserIn]   i
 Lindstrom, Kristin [VerfasserIn]   i
 Lilienstein, Joshua [VerfasserIn]   i
 Clague, Gillian E. [VerfasserIn]   i
 Rowell, Richard [VerfasserIn]   i
 Burton, Barbara K. [VerfasserIn]   i
Titel:Efficacy and safety of sapropterin before and during pregnancy
Titelzusatz:final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) maternal and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU-MOMs sub-registries
Verf.angabe:François Feillet, Can Ficicioglu, Florian B. Lagler, Nicola Longo, Ania C. Muntau, Alberto Burlina, Friedrich K. Trefz, Francjan J. van Spronsen, Jean-Baptiste Arnoux, Kristin Lindstrom, Joshua Lilienstein, Gillian E. Clague, Richard Rowell, Barbara K. Burton, on behalf of the KAMPER and PKUDOS investigators
E-Jahr:2024
Jahr:July 2024
Umfang:15 S.
Illustrationen:Illustrationen
Fussnoten:Erstveröffentlichung: 03 March 2024 ; Gesehen am 20.08.2024
Titel Quelle:Enthalten in: Journal of inherited metabolic disease
Ort Quelle:Hoboken, NJ : Wiley, 1978
Jahr Quelle:2024
Band/Heft Quelle:47(2024), 4 vom: Juli, Seite 636-650
ISSN Quelle:1573-2665
Abstract:Infants born to mothers with phenylketonuria (PKU) may develop congenital abnormalities because of elevated phenylalanine (Phe) levels in the mother during pregnancy. Maintenance of blood Phe levels between 120 and 360 μmol/L reduces risks of birth defects. Sapropterin dihydrochloride helps maintain blood Phe control, but there is limited evidence on its risk-benefit ratio when used during pregnancy. Data from the maternal sub-registries—KAMPER (NCT01016392) and PKUDOS (NCT00778206; PKU-MOMs sub-registry)—were collected to assess the long-term safety and efficacy of sapropterin in pregnant women in a real-life setting. Pregnancy and infant outcomes, and the safety of sapropterin were assessed. Final data from 79 pregnancies in 57 women with PKU are reported. Sapropterin dose was fairly constant before and during pregnancy, with blood Phe levels maintained in the recommended target range during the majority (82%) of pregnancies. Most pregnancies were carried to term, and the majority of liveborn infants were reported as ‘normal’ at birth. Few adverse and serious adverse events were considered related to sapropterin, with these occurring in participants with high blood Phe levels. This report represents the largest population of pregnant women with PKU exposed to sapropterin. Results demonstrate that exposure to sapropterin during pregnancy was well-tolerated and facilitated maintenance of blood Phe levels within the target range, resulting in normal delivery. This critical real-world data may facilitate physicians and patients to make informed treatment decisions about using sapropterin in pregnant women with PKU and in women of childbearing age with PKU who are responsive to sapropterin.
DOI:doi:10.1002/jimd.12724
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

Volltext: https://doi.org/10.1002/jimd.12724
 Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/jimd.12724
 DOI: https://doi.org/10.1002/jimd.12724
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:BH4
 maternal PKU
 PKU
 pregnancy
 registry
 sapropterin
K10plus-PPN:189920105X
Verknüpfungen:→ Zeitschrift

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