Sapropterin for phenylketonuria: A Japanese post-marketing surveillance study
| Autor: | Shuichi Chikada, Kento Wada, Mina Tamura, Yasuyuki Kakurai, Shizuka Seki |
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| Rok vydání: | 2021 |
| Předmět: |
Male
Pediatrics medicine.medical_specialty Surveillance study business.industry Phenylketonurias Phenylalanine Postmarketing surveillance medicine.disease Biopterin Hyperphenylalaninemia Japan Pregnancy Child Preschool Pediatrics Perinatology and Child Health Product Surveillance Postmarketing Medicine Humans Female Alanine aminotransferase business Adverse effect Adverse drug reaction |
| Zdroj: | Pediatrics international : official journal of the Japan Pediatric Society. 64(1) |
| ISSN: | 1442-200X |
| Popis: | The aim of this study was to assess the long-term safety and efficacy of sapropterin in a real-world setting in Japanese patients with tetrahydrobiopterin (BH4)-responsive phenylketonuria.This post-marketing surveillance study enrolled all of the patients in Japan with confirmed BH4-responsive PKU who were administrated sapropterin between July 2008 and October 2017. Patients were observed at least every 3 months during follow up, with key data collected on treatment exposure/duration, effectiveness according to physician's judgement, serum phenylalanine levels, and adverse events.Of 87 enrolled patients, 85 patients (male, 42.4%; outpatients, 96.5%) were included in the safety and efficacy analysis sets. Treatment started at age4 years in 43 (50.6%) patients and the most common starting daily dose was 5-10 mg/kg (n = 41, 48.2%) with the overall duration of treatment between 0.2 and 17.2 years. Serum phenylalanine levels, according to loading tests, reduced from a baseline level of 9.66 mg/dL (range 0.48-36.80 mg/dL) by30% in 84 patients. Treatment was deemed effective in 79 of 85 patients (92.9%, 95% confidence interval: 85.3-97.4). One patient (1.2%) experienced an adverse drug reaction (alanine aminotransferase increased) 50 days after the start of administration, which resolved without complications with continued treatment.Sapropterin appears well tolerated and highly effective in Japanese patients treated in a real-world setting, including those who start treatment at age4 years and pregnant women. |
| Databáze: | OpenAIRE |
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