Elevated phenylacetic acid levels do not correlate with adverse events in patients with urea cycle disorders or hepatic encephalopathy and can be predicted based on the plasma PAA to PAGN ratio

Molecular Genetics and Metabolism

Volumn 110, Issue 4, 2013, Pages 446-453

  Mokhtarani, M ^a   Diaz, G A ^b   Rhead, W ^c   Berry, S A ^d   Lichter Konecki, U ^e   Feigenbaum, A ^f   Schulze, A ^f   Longo, N ^g   Bartley, J ^h   Berquist, W ⁱ   Gallagher, R ^j   Smith, W ^k   McCandless, S E ^l   Harding, C ^m   Rockey, D C ⁿ   Vierling, J M ^o   Mantry, P ^p   Ghabril, M ^q   Brown, R S ^r   Dickinson, K ^a   Moors, T ^a   Norris, C ^a   Coakley, D ^a   Milikien, D A ^s   Nagamani, S C ^o   LeMons, C ^t   Lee, B ^o   Scharschmidt, B F ^a   more..

^a Hyperion Therapeutics   (United States)

^b MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^c MEDICAL COLLEGE OF WISCONSIN   (United States)

^d UNIVERSITY OF MINNESOTA   (United States)

^e CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^f HOSPITAL FOR SICK CHILDREN   (Canada)

^g UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^h LONG BEACH MEMORIAL MEDICAL CENTER   (United States)

ⁱ STANFORD UNIVERSITY   (United States)

^j CHILDREN S HOSPITAL COLORADO   (United States)

^k MAINE MEDICAL CENTER   (United States)

^l CASE WESTERN RESERVE UNIVERSITY   (United States)

^m OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

ⁿ MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^o BAYLOR COLLEGE OF MEDICINE   (United States)

^p METHODIST DALLAS MEDICAL CENTER   (United States)

^q INDIANA UNIVERSITY   (United States)

^r NewYork Presbyterian Hospital   (United States)

^s Accudata Solutions Inc   (United States)

^t National Urea Cycle Disorders Foundation   (United States)

more..

Author keywords

BUPHENYL; Glycerol phenylbutyrate; HPN 100; Neurological adverse events; RAVICTI; Sodium phenylbutyrate

Indexed keywords

4 PHENYLBUTYRIC ACID; GLUTAMINE DERIVATIVE; GLYCEROL PHENYLBUTYRATE; PHENYLACETIC ACID; PHENYLACETYLEGLUTAMINE; PLACEBO; UNCLASSIFIED DRUG;

ABDOMINAL DISCOMFORT; ADOLESCENT; ADULT; AMNESIA; ARTICLE; BLOOD LEVEL; BLOOD SAMPLING; BRAIN EDEMA; CANCER PATIENT; CHILD; COMA; COMPARATIVE STUDY; CONFUSION; CONTROLLED STUDY; CROSSOVER PROCEDURE; DECOMPENSATED LIVER CIRRHOSIS; DISORIENTATION; DIZZINESS; DRUG BLOOD LEVEL; DRUG SAFETY; FATIGUE; FEMALE; FOOT EDEMA; HEADACHE; HEARING IMPAIRMENT; HEART ARRHYTHMIA; HEPATIC ENCEPHALOPATHY; HUMAN; INFANT; LAVAGE; LETHARGY; LONG TERM CARE; MAJOR CLINICAL STUDY; MALE; MAXIMUM PLASMA CONCENTRATION; METABOLIC ACIDOSIS; METABOLIC ENCEPHALOPATHY; MULTICENTER STUDY (TOPIC); NAUSEA; NEUROPATHY; OPEN STUDY; PHASE 2 CLINICAL TRIAL (TOPIC); PRESCHOOL CHILD; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); RASH; SCHOOL CHILD; SLURRED SPEECH; SOMNOLENCE; TACHYPNEA; TASTE DISORDER; TREATMENT DURATION; UREA CYCLE DISORDER; VOMITING; WEAKNESS;

BUPHENYL; GEE; GENERALIZED ESTIMATING EQUATIONS; GLYCEROL PHENYLBUTYRATE; GLYCEROL PHENYLBUTYRATE (GENERIC NAME FOR GLYCERYL TRI (4-PHENYLBUTYRATE), ALSO REFERRED TO AS HPN-100 OR RAVICTI(®)); GPB; HE; HEPATIC ENCEPHALOPATHY; HPN-100; NAPBA; NEUROLOGICAL ADVERSE EVENTS; PAA; PAA:PAGN RATIO; PAGN; PBA; PHENYLACETIC ACID; PHENYLACETYLGLUTAMINE; PHENYLBUTYRIC ACID; RATIO OF THE CONCENTRATIONS IN ΜG/ML OF PAA TO PAGN IN PLASMA; RAVICTI; SAFETY EXTENSION; SE; SO; SODIUM PHENYLBUTYRATE; SODIUM PHENYLBUTYRATE (BUPHENYL(®)); SWITCHOVER; UCD; UREA CYCLE DISORDER;

BIOLOGICAL MARKERS; DRUG-RELATED SIDE EFFECTS AND ADVERSE REACTIONS; GLUTAMINE; GLYCEROL; HEPATIC ENCEPHALOPATHY; HUMANS; LIVER; NEOPLASMS; PHENYLACETATES; PHENYLBUTYRATES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; UREA CYCLE DISORDERS, INBORN;

EID: 84897089628     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.09.017     Document Type: Article

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