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

M. Mokhtarani; G. A. Diaz; W. Rhead; S. A. Berry; U. Lichter-Konecki; A. Feigenbaum; A. Schulze; N. Longo; J. Bartley; W. Berquist; R. Gallagher; W. Smith; S. E. McCandless; C. Harding; D. C. Rockey; J. M. Vierling; P. Mantry; M. Ghabril; R. S. Brown; K. Dickinson; T. Moors; C. Norris; D. Coakley; D. A. Milikien; S. C. Nagamani; C. LeMons; B. Lee; B. F. Scharschmidt

doi:10.1016/j.ymgme.2013.09.017

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

M. Mokhtarani, G. A. Diaz, W. Rhead, S. A. Berry, U. Lichter-Konecki, A. Feigenbaum, A. Schulze, N. Longo, J. Bartley, W. Berquist, R. Gallagher, W. Smith, S. E. McCandless, C. Harding, D. C. Rockey, J. M. Vierling, P. Mantry, M. Ghabril, R. S. Brown, K. DickinsonT. Moors, C. Norris, D. Coakley, D. A. Milikien, S. C. Nagamani, C. LeMons, B. Lee, B. F. Scharschmidt

Research output: Contribution to journal › Article › peer-review

Original language	English
Pages (from-to)	446-453
Number of pages	8
Journal	Molecular Genetics and Metabolism
Volume	110
Issue number	4
DOIs	https://doi.org/10.1016/j.ymgme.2013.09.017
State	Published - Dec 2013
Externally published	Yes

ASJC Scopus Subject Areas

Endocrinology, Diabetes and Metabolism
Biochemistry
Molecular Biology
Genetics
Endocrinology

Keywords

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

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10.1016/j.ymgme.2013.09.017

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Mokhtarani, M., Diaz, G. A., Rhead, W., Berry, S. A., Lichter-Konecki, U., Feigenbaum, A., Schulze, A., Longo, N., Bartley, J., Berquist, W., Gallagher, R., Smith, W., McCandless, S. E., Harding, C., Rockey, D. C., Vierling, J. M., Mantry, P., Ghabril, M., Brown, R. S., ... Scharschmidt, B. F. (2013). 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, 110(4), 446-453. https://doi.org/10.1016/j.ymgme.2013.09.017

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. / Mokhtarani, M.; Diaz, G. A.; Rhead, W. et al.
In: Molecular Genetics and Metabolism, Vol. 110, No. 4, 12.2013, p. 446-453.

Research output: Contribution to journal › Article › peer-review

Mokhtarani, M, Diaz, GA, Rhead, W, Berry, SA, Lichter-Konecki, U, Feigenbaum, A, Schulze, A, Longo, N, Bartley, J, Berquist, W, Gallagher, R, Smith, W, McCandless, SE, Harding, C, Rockey, DC, Vierling, JM, Mantry, P, Ghabril, M, Brown, RS, Dickinson, K, Moors, T, Norris, C, Coakley, D, Milikien, DA, Nagamani, SC, LeMons, C, Lee, B & Scharschmidt, BF 2013, '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, vol. 110, no. 4, pp. 446-453. https://doi.org/10.1016/j.ymgme.2013.09.017

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title = "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",

keywords = "BUPHENYL, Glycerol phenylbutyrate, HPN-100, Neurological adverse events, RAVICTI, Sodium phenylbutyrate",

author = "M. Mokhtarani and Diaz, {G. A.} and W. Rhead and Berry, {S. A.} and U. Lichter-Konecki and A. Feigenbaum and A. Schulze and N. Longo and J. Bartley and W. Berquist and R. Gallagher and W. Smith and McCandless, {S. E.} and C. Harding and Rockey, {D. C.} and Vierling, {J. M.} and P. Mantry and M. Ghabril and Brown, {R. S.} and K. Dickinson and T. Moors and C. Norris and D. Coakley and Milikien, {D. A.} and Nagamani, {S. C.} and C. LeMons and B. Lee and Scharschmidt, {B. F.}",

note = "Funding Information: The authors gratefully acknowledge and thank the efforts of the Study Coordinators and nursing staff who made these trials possible, including D. Bartholomew (Nationwide Children's Hospital), S. Cederbaum, D. Wong (University of California), J. Vockley (Children's Hospital of Pittsburgh), S. Bart, M. Al-Ibraham (SNBL), M.S. Korson (Tufts Medical Center), D. Kronn (Westchester Medical Center), R. Zori (University of Florida), J.L. Merritt (Seattle Children's Hospital), N. Schrager (Mount Sinai School of Medicine), A. Donovan, J. Crawford, Pediatric TRU Staff, K. Defouw, J. Balliet (The Medical College of Wisconsin), M. Keuth, N. O'Donnell (Long Beach Memorial Hospital), M. Hussain, E. Bailey, M. Ambreen (The Hospital for Sick Children, University of Toronto, ON, Canada), C. Bailey, A. Lang (The University of Utah), J. Perry, V. de Leon, A. Niemi, K. Cusmano (Stanford University),T. Carlson, J. Parker, S. Elsbecker (University of Minnesota), K. Simpson (Children's National Medical Center), K. Regis (Nationwide Children's Hospital), A. Behrend, T. Marrone, J. Martin (Oregon Health Sciences University), N. Dorrani (University of California, Los Angeles), M.B. Frohnapfel, S. Bergant, J. Haky, C. Tasi, C. Heggie (Case Western Reserve University), S. Mortenson (Maine Medical Center), S. Deward (Children's Hospital of Pittsburgh), S. Burr (Children's Hospital Colorado), K. Bart, C. Duggan (SNBL), K. Murray, C. Dedomenico (Tufts Medical Center), C. Gross (University of Florida), L. Brody (Seattle Children's Hospital), M. Mullins, S. Carter, A. Tran, J. Stuff, TCH General Clinical Research Center nursing staff (Baylor), B. McGuire (University of Alabama), D. Wolf (New York Medical College), C. O'Brien (University of Miami), R. O'Shea (Cleveland Clinic), I. Zupanets (National University of Pharmacy of MH of Ukraine), Kathy Lisam (Hyperion), as well as the Clinical and Translational Science Awards/General Clinical Research Center grants ( Baylor College of Medicine , M01RR00188 ; Case Western Reserve University , UL1RR024989 ; Clinical and Translational Science Institute at Children's National Medical Center NIH/NCRR , UL1RR31988 ; Medical College of Wisconsin , UL1RR31973 ; Mount Sinai School of Medicine , UL1RR29887 ; Oregon Health & Science University , UL1RR24140 ; Stanford University , UL1RR25744 ; Tufts University , UL1RR25752 ; University of California, Los Angeles , UL1RR33176 ; University of Colorado , UL1RR25780 ; University of Florida , UL1RR29890 ; University of Minnesota , UL1RR33183 ; University of Pittsburgh , UL1RR24153 , and UL1TR000005 ; University of Utah , UL1RR25764 ; University of Washington , UL1RR25014 ), the Urea Cycle Disorders Consortium ( NIH grant U54RR019453 ) and grants from the O'Malley Foundation and Kettering Fund which provided support. SCS. Nagamani is an awardee of the National Urea Cycle Disorders Foundation Research Fellowship. The authors also thank G. Enns (Stanford) for his comments on the manuscript. ",

year = "2013",

month = dec,

doi = "10.1016/j.ymgme.2013.09.017",

language = "English",

volume = "110",

pages = "446--453",

journal = "Molecular Genetics and Metabolism",

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T1 - 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

AU - Mokhtarani, M.

AU - Diaz, G. A.

AU - Rhead, W.

AU - Berry, S. A.

AU - Lichter-Konecki, U.

AU - Feigenbaum, A.

AU - Schulze, A.

AU - Longo, N.

AU - Bartley, J.

AU - Berquist, W.

AU - Gallagher, R.

AU - Smith, W.

AU - McCandless, S. E.

AU - Harding, C.

AU - Rockey, D. C.

AU - Vierling, J. M.

AU - Mantry, P.

AU - Ghabril, M.

AU - Brown, R. S.

AU - Dickinson, K.

AU - Moors, T.

AU - Norris, C.

AU - Coakley, D.

AU - Milikien, D. A.

AU - Nagamani, S. C.

AU - LeMons, C.

AU - Lee, B.

AU - Scharschmidt, B. F.

N1 - Funding Information: The authors gratefully acknowledge and thank the efforts of the Study Coordinators and nursing staff who made these trials possible, including D. Bartholomew (Nationwide Children's Hospital), S. Cederbaum, D. Wong (University of California), J. Vockley (Children's Hospital of Pittsburgh), S. Bart, M. Al-Ibraham (SNBL), M.S. Korson (Tufts Medical Center), D. Kronn (Westchester Medical Center), R. Zori (University of Florida), J.L. Merritt (Seattle Children's Hospital), N. Schrager (Mount Sinai School of Medicine), A. Donovan, J. Crawford, Pediatric TRU Staff, K. Defouw, J. Balliet (The Medical College of Wisconsin), M. Keuth, N. O'Donnell (Long Beach Memorial Hospital), M. Hussain, E. Bailey, M. Ambreen (The Hospital for Sick Children, University of Toronto, ON, Canada), C. Bailey, A. Lang (The University of Utah), J. Perry, V. de Leon, A. Niemi, K. Cusmano (Stanford University),T. Carlson, J. Parker, S. Elsbecker (University of Minnesota), K. Simpson (Children's National Medical Center), K. Regis (Nationwide Children's Hospital), A. Behrend, T. Marrone, J. Martin (Oregon Health Sciences University), N. Dorrani (University of California, Los Angeles), M.B. Frohnapfel, S. Bergant, J. Haky, C. Tasi, C. Heggie (Case Western Reserve University), S. Mortenson (Maine Medical Center), S. Deward (Children's Hospital of Pittsburgh), S. Burr (Children's Hospital Colorado), K. Bart, C. Duggan (SNBL), K. Murray, C. Dedomenico (Tufts Medical Center), C. Gross (University of Florida), L. Brody (Seattle Children's Hospital), M. Mullins, S. Carter, A. Tran, J. Stuff, TCH General Clinical Research Center nursing staff (Baylor), B. McGuire (University of Alabama), D. Wolf (New York Medical College), C. O'Brien (University of Miami), R. O'Shea (Cleveland Clinic), I. Zupanets (National University of Pharmacy of MH of Ukraine), Kathy Lisam (Hyperion), as well as the Clinical and Translational Science Awards/General Clinical Research Center grants ( Baylor College of Medicine , M01RR00188 ; Case Western Reserve University , UL1RR024989 ; Clinical and Translational Science Institute at Children's National Medical Center NIH/NCRR , UL1RR31988 ; Medical College of Wisconsin , UL1RR31973 ; Mount Sinai School of Medicine , UL1RR29887 ; Oregon Health & Science University , UL1RR24140 ; Stanford University , UL1RR25744 ; Tufts University , UL1RR25752 ; University of California, Los Angeles , UL1RR33176 ; University of Colorado , UL1RR25780 ; University of Florida , UL1RR29890 ; University of Minnesota , UL1RR33183 ; University of Pittsburgh , UL1RR24153 , and UL1TR000005 ; University of Utah , UL1RR25764 ; University of Washington , UL1RR25014 ), the Urea Cycle Disorders Consortium ( NIH grant U54RR019453 ) and grants from the O'Malley Foundation and Kettering Fund which provided support. SCS. Nagamani is an awardee of the National Urea Cycle Disorders Foundation Research Fellowship. The authors also thank G. Enns (Stanford) for his comments on the manuscript.

PY - 2013/12

Y1 - 2013/12

KW - BUPHENYL

KW - Glycerol phenylbutyrate

KW - HPN-100

KW - Neurological adverse events

KW - RAVICTI

KW - Sodium phenylbutyrate

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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

ASJC Scopus Subject Areas

Keywords

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OpenUrl availability

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