PMID- 25724073 OWN - NLM STAT- MEDLINE DCOM- 20160122 LR - 20231213 IS - 1096-7206 (Electronic) IS - 1096-7192 (Linking) VI - 114 IP - 4 DP - 2015 Apr TI - Long-term safety and efficacy of sapropterin: the PKUDOS registry experience. PG - 557-63 LID - S1096-7192(15)00046-3 [pii] LID - 10.1016/j.ymgme.2015.02.003 [doi] AB - The Phenylketonuria (PKU) Demographics, Outcomes and Safety (PKUDOS) registry is designed to provide longitudinal safety and efficacy data on subjects with PKU who are (or have been) treated with sapropterin dihydrochloride. The PKUDOS population consists of 1189 subjects with PKU: N = 504 who were continuously exposed to sapropterin from date of registry enrollment, N = 211 who had intermittent exposure to the drug, and N = 474 with some other duration of exposure. Subjects continuously exposed to sapropterin showed an average 34% decrease in blood phenylalanine (Phe)--from 591 +/- 382 mumol/L at baseline to 392 +/- 239 mumol/L (p = 0.0009) after 5 years. This drop in blood Phe was associated with an increase in dietary Phe tolerance [from 1000 +/- 959 mg/day (pre-sapropterin baseline) to 1539 +/- 840 mg/day after 6 years]. Drug-related adverse events (AEs) were reported in 6% of subjects, were mostly considered non-serious, and were identified in the gastrointestinal, respiratory, and nervous systems. Serious drug-related AEs were reported in