Sapropterin Dihydrochloride Tablets

Autor: Dennis J. Cada, Danial E. Baker, Terri Levien
Rok vydání: 2008
Předmět:
Zdroj: Hospital Pharmacy. 43:307-314
ISSN: 1945-1253
0018-5787
DOI: 10.1310/hpj4304-307
Popis: INDICATIONS Sapropterin dihydrochloride is indicated to reduce blood phenyl alanine levels in patients with hyperphenylalaninemia caused by tetrahydrobiopterin (BH4)-responsive phenylketonuria. Sapropterin should be used in conjunction with a phenylalanine-restricted diet. Phenylketonuria is an autosomal recessive metabolic disease caused by mutations in the gene that encodes phenylalanine hydroxylase. These mutations prevent hepatic conversion of phenylalanine to tyrosine, which leads to hyperphenylalaninemia. Prolonged elevations in phenylalanine concentration in the blood can produce profound neurocognitive and developmental defects so early detection and dietary restrictions are important to avoid these complications. However, compliance with the dietary restrictions are difficult, and noncompliance after successful treatment in early childhood can result in below-average IQ scores, behavioral problems, and emotional dysfunction. Dietary restriction and supplementation is the mainstay of therapy for patients with phenylketonuria. The phenylalanine content of the food must be counted daily. Dietary restriction is recommended for life with a treatment goal of blood phenylalanine levels between 120 and 360 mcmol/L through 12 years of age and concentrations between 120 and 900 mcmol/L acceptable for those 13 years of age and older (120 to 600 mcmol/L preferred). High-protein foods are restricted and a medical food is often used to provide adequate protein with additional tyrosine and no phenylalanine.
Databáze: OpenAIRE
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