Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) maternal and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU‐MOMs sub‐registries

Published on 06/07/2024

Reading time: 7 min.

François Feillet ¹, Can Ficicioglu ², Florian B. Lagler ³, Nicola Longo ⁴, Ania C. Muntau ⁵, Alberto Burlina ⁶, Friedrich K. Trefz ⁷, Francjan J. van Spronsen ⁸, Jean‐Baptiste Arnoux ⁹, Kristin Lindstrom ¹⁰, Joshua Lilienstein ¹⁰, Gillian E. Clague ¹⁰, Richard Rowell ¹⁰, Barbara K. Burton ¹¹, the KAMPER and PKUDOS investigators

¹ Hôpital d'enfants Brabois, INSERM 1256 NGERE, Faculty of Medicine Vandoeuvre les Nancy France
² Children's Hospital of Philadelphia Philadelphia Pennsylvania USA
³ Institute of Inherited Metabolic Diseases and Department of Pediatrics Paracelsus Medical Private University Salzburg Austria
⁴ University of Utah Salt Lake City Utah USA
⁵ University Children's Hospital Medical Center Hamburg‐Eppendorf Hamburg Germany
⁶ University Hospital Padova Italy
⁷ Division of Inborn Metabolic Diseases University Children's Hospital, Department of General Pediatrics Heidelberg Germany
⁸ Division of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center of Groningen University of Groningen Groningen The Netherlands
⁹ Necker‐Enfants Malades University Hospital Paris France
¹⁰ BioMarin Pharmaceutical Inc Novato California USA
¹¹ Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois USA

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)...

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Journal of inherited metabolic disease

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