Classification of BH4 Response Sample Clauses

Classification of BH4 Response. ‌ Fasting plasma phenylalanine concentrations were measured immediately before and after one month of 20 mg/kg/day BH4 therapy (sapropterin dihydrochloride; Kuvan®; BioMarin Pharmaceutical Inc., Novato, California, USA). BH4 response was classified using sequentially applied criteria, detailed elsewhere [185]. Briefly, patients were initially classified based on the percent change in plasma phenylalanine concentrations after one month of BH4 therapy. Non-responders experienced less than a 15% decrease in plasma phenylalanine concentrations, discontinued BH4 therapy, and continued their diet therapy regimen. Patients with at least a 15% decrease in plasma phenylalanine concentrations at month one were further evaluated and differentiated. Definitive responders were able to improve their dietary phenylalanine tolerance by at least 300 mg/day (or consume an unrestricted diet) and could decrease their medical food needs by at least 25% compared to baseline (or no longer needed medical food) while maintaining blood phenylalanine concentrations below 360 µmol/L. Provisional responders, despite an initial decrease in plasma phenylalanine concentrations, could not meet both of the diet liberalization criteria while maintaining blood phenylalanine concentrations below 360 µmol/L.
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