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Table 5 Summary of steps involved in a sapropterin therapy trial

From: Diagnostic and therapeutic recommendations for the treatment of hyperphenylalaninemia in patients 0–4 years of age

Screening Phase
• Diet alone
• Food diary
• Determination of baseline Phe tolerance (PheTol0)
• Determination of the baseline quantity of protein from the amino-acid mixture (amino-acid supplement [AAS0])
• Determination of the baseline blood Phe concentration (Phe0)
Test Phase
• Sapropterin 10–20 mg/kg per day
• Food diary
• Determination of the Phe tolerance on therapy (PheTol1)
• Determination of the quantity of protein from the amino-acid mixture on therapy (AAS1)
• Determination of the Phe concentration on therapy (Phe1)
• Continuing the test phase based on the algorithm until steady state is reached
Evaluation
• Calculate the differences:
­ PheTolx – PheTol0
­ AASx – AAS0
­ Phex – Phe0
• Evaluation and critical benefit analysis in the team