Evidence map›Paper›PMID 26296711›Full record

Trial reportMolecular genetics and metabolism

Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials.

Sandesh C S Nagamani, George A Diaz, William Rhead, Susan A Berry, Cynthia Le Mons, Uta Lichter-Konecki, James Bartley, Annette Feigenbaum, Andreas Schulze, Nicola Longo and 19 more

Open access · greenAbstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Molecular genetics and metabolism. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.9field-weighted citation impact, top 27% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 16 citations in OpenAlex.

  1. Review
  2. Observational
  3. Review
  4. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

29 authors at 14 institutions in 1 country.

Sandesh C S NagamaniBaylor College of Medicine, One Baylor Plaza, Room R814, Houston, TX 77030, USA. Electronic address: Nagamani@bcm.edu.
George A DiazIcahn School of Medicine at Mount Sinai, Department of Genetics and Genomic Sciences, Department of Pediatrics, 1428 Madison Avenue, New York, NY 10029, USA.
William RheadThe Medical College of Wisconsin, MS 716, 9000 W. Wisconsin Avenue, Milwaukee, WI 53226, USA.
Susan A BerryUniversity of Minnesota, 420 Delaware St. SE, MMC 75, Minneapolis, MN 55455, USA.
Cynthia Le MonsNational Urea Cycle Disorders Foundation, 75 S. Grand Ave, Pasadena, CA 91105, USA.
Uta Lichter-KoneckiDrexel University, 3141 Chestnut Street, Philadelphia, PA, 19104, USA.
James BartleyLong Beach Memorial Hospital, 2801 Atlantic Avenue, Long Beach, CA 90806, USA.
Annette FeigenbaumRady Children's Hospital, 3020 Children's Way, San Diego, CA 92123, USA.
Andreas SchulzeThe Hospital for Sick Children, 555 University Avenue, Toronto, ON M5G1X8, Canada.
Nicola LongoThe University of Utah, Division of Medical Genetics, 2C412 SOM, 50 North Mario Capecchi Drive, Salt Lake City, UT 84132, USA.
William BerquistStanford University, 750 Welch Road, #116, Palo Alto, CA 94305, USA.
Renata GallagherUCSF School of Medicine, 550 16th Street, San Francisco, CA 94158, USA.
Dennis BartholomewNationwide Children's Hospital, 545 South 18th Street, TH485, Columbus, OH 43205, USA.
Cary O HardingOregon Health & Science University, 3181 SW Sam Jackson Park Road, A036/B198, Mail code L103, Portland, OR 97239, USA.
Mark S KorsonTufts Medical Center, Floating Building, 3rd Floor, 800 Washington Street, Boston, MA 02111, USA.
Shawn E McCandlessCase Western Reserve University, 11100 Euclid Avenue, Cleveland, OH 44106, USA.
Wendy SmithMaine Medical Center, 1577 Congress Street, 2nd Floor, Portland, ME 04102, USA.
Jerry VockleyChildren's Hospital of Pittsburgh, One Children's Hospital Drive, 4401 Penn Avenue, Rangos Floor 5, Pittsburgh, PA 15224, USA.
David KronnWestchester Medical Center, 503 Grasslands Road, Valhalla, NY 10595, USA.
Robert ZoriUniversity of Florida, UFHSC Box 100296, Gainesville, FL 32610, USA.
Stephen CederbaumUniversity of California, Los Angeles, 10833 Le Conte Avenue CHS 32-225, Los Angeles, CA 90095, USA.
J Lawrence MerrittSeattle Children's Hospital, 4800 Sand Point Way NE M/S W-65945, Seattle, WA 98105, USA.
Derek WongUniversity of California, Los Angeles, 10833 Le Conte Avenue CHS 32-225, Los Angeles, CA 90095, USA.
Dion F CoakleyHorizon Therapeutics Inc., 2000 Sierra Point Parkway Suite 400, Brisbane, CA 94005, USA.
Bruce F ScharschmidtHorizon Therapeutics Inc., 2000 Sierra Point Parkway Suite 400, Brisbane, CA 94005, USA.
Klara DickinsonAnthera Pharmaceuticals, 25801 Industrial Blvd. Suite B, Hayward, CA 94545, USA.
Miguel MarinoOregon Health & Science University, 3181 SW Sam Jackson Park Road, A036/B198, Mail code L103, Portland, OR 97239, USA.
Brendan H LeeBaylor College of Medicine, One Baylor Plaza, Room R814, Houston, TX 77030, USA.
Masoud MokhtaraniHorizon Therapeutics Inc., 2000 Sierra Point Parkway Suite 400, Brisbane, CA 94005, USA.
Horizon Therapeutics (United States) · USCase Western Reserve University · USChildren's Hospital of Pittsburgh · USDrexel University · USIcahn School of Medicine at Mount Sinai · USMaine Medical Center · USMedical College of Wisconsin · USNationwide Children's Hospital · USRady Children's Hospital-San Diego · USStanford University · USTufts Medical Center · USUniversity of Florida · USUniversity of Minnesota · USWestchester Medical Center · US

Funding

Transform Dissemination and Implementation Science in CTSA ProgramsUL1TR002319 · NCATS · UNIVERSITY OF WASHINGTON · PI John K. Amory · 2017 to 2026
$100.0M
CTSA INFRASTRUCTURE FOR PEDIATRIC RESEARCHUL1RR025780 · NCRR · UNIVERSITY OF COLORADO DENVER · PI SOKOL, RONALD J. · 2008 to 2011
$61.3M
OREGON CLINICAL AND TRANSLATIONAL SCIENCE INSTITUTEUL1RR024140 · NCRR · OREGON HEALTH & SCIENCE UNIVERSITY · PI ORWOLL, ERIC S. · 2006 to 2011
$61.1M
University of Pittsburgh Clinical and Translational Science InstituteUL1TR000005 · NCATS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2012 to 2015
$50.8M
Institute of Translational Health SciencesUL1TR000423 · NCATS · UNIVERSITY OF WASHINGTON · PI DISIS, MARY L. · 2012 to 2016
$49.4M
Spectrum Stanford Center for clinical and Translational Research and EducationUL1TR001085 · NCATS · STANFORD UNIVERSITY · PI CULLEN, MARK RICHARD, GREENBERG, HARRY BERNARD · 2013 to 2017
$37.1M
University of Minnesota Clinical and Translational Science Institute (UMN CTSI)UL1TR000114 · NCATS · UNIVERSITY OF MINNESOTA · PI BLAZAR, BRUCE R · 2012 to 2015
$35.0M
ZOLENDRONIC ACID IN CHILDREN W/ SEVERE OSTEOGENESIS IMPERFECTAM01RR000188 · NCRR · BAYLOR COLLEGE OF MEDICINE · PI SHEARER, WILLIAM THOMAS · 1985 to 2011
$30.6M
Stanford Center for Clinical and Translational Education and Research (SCCTER)UL1UL1RR025744 · NCRR · STANFORD UNIVERSITY · PI GREENBERG, HARRY BERNARD · 2008 to 2011
$22.4M
UNIVERSITY OF UTAH CENTER FOR CLINICAL AND TRANSLATIONAL SCIENCE-UL1: BPCAUL1RR025764 · NCRR · UNIVERSITY OF UTAH · PI MCCLAIN, DONALD A. · 2008 to 2011
$19.4M
MOUNT SINAI INSTITUTES FOR CLINICAL AND TRANSLATIONAL SCIENCESUL1RR029887 · NCRR · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI SAMPSON, HUGH A · 2009 to 2011
$19.2M
TUFTS CLINICAL AND TRANSLATION SCIENCE INSTITUTE (UL1): BPCAUL1RR025752 · NCRR · TUFTS UNIVERSITY BOSTON · PI SELKER, HARRY P. · 2008 to 2011
$17.6M
Doris Duke Charitable Foundation 2013095NCATS NIH HHS UL1 TR000005NCATS NIH HHS UL1TR000005NCATS NIH HHS UL1 TR000114NCATS NIH HHS UL1 TR000423NCATS NIH HHS UL1TR000423NCATS NIH HHS UL1 TR001085NCATS NIH HHS UL1 TR002319NCRR NIH HHS M01 RR000188NCRR NIH HHS M01RR00188NCRR NIH HHS U54 RR019453NCRR NIH HHS U54RR019453NCRR NIH HHS UL1 RR024140NCRR NIH HHS UL1 RR025744NCRR NIH HHS UL1 RR025752NCRR NIH HHS UL1 RR025764NCRR NIH HHS UL1 RR025780NCRR NIH HHS UL1 RR029887NCRR NIH HHS UL1 RR029890NCRR NIH HHS UL1 RR031973NCRR NIH HHS UL1 RR031988NCRR NIH HHS UL1 RR033176NCRR NIH HHS UL1 RR033183NCRR NIH HHS UL1RR24140NCRR NIH HHS UL1RR25744NCRR NIH HHS UL1RR25752NCRR NIH HHS UL1RR25764NCRR NIH HHS UL1RR25780NCRR NIH HHS UL1RR29887NCRR NIH HHS UL1RR29890NCRR NIH HHS UL1RR31973NCRR NIH HHS UL1RR31988NCRR NIH HHS UL1RR33176NCRR NIH HHS UL1RR33183NIDDK NIH HHS 1K08DK074573NIDDK NIH HHS K08 DK074573NIDDK NIH HHS R01 DK102641
6 · The paper itself

Abstract

backgroundHealth care outcomes have been increasingly assessed through health-related quality of life (HRQoL) measures. While the introduction of nitrogen-scavenging medications has improved survival in patients with urea cycle disorders (UCDs), they are often associated with side effects that may affect patient compliance and outcomes.

methodsSymptoms commonly associated with nitrogen-scavenging medications were evaluated in 100 adult and pediatric participants using a non-validated UCD-specific questionnaire. Patients or their caregivers responded to a pre-defined list of symptoms known to be associated with the use of these medications. Responses were collected at baseline (while patients were receiving sodium phenylbutyrate [NaPBA]) and during treatment with glycerol phenylbutyrate (GPB).

resultsAfter 3 months of GPB dosing, there were significant reductions in the proportion of patients with treatment-associated symptoms (69% vs. 46%; p<0.0001), the number of symptoms per patient (2.5 vs. 1.1; p<0.0001), and frequency of the more commonly reported individual symptoms such as body odor, abdominal pain, nausea, burning sensation in mouth, vomiting, and heartburn (p<0.05). The reduction in symptoms was observed in both pediatric and adult patients. The presence or absence of symptoms or change in severity did not correlate with plasma ammonia levels or NaPBA dose.

conclusionsThe reduction in symptoms following 3 months of open-label GPB dosing was similar in pediatric and adult patients and may be related to chemical structure and intrinsic characteristics of the product rather than its effect on ammonia control.

Indexed as

Quality of LifeSelf ReportAdolescentAdultAgedAmmoniaAntineoplastic AgentsChildChild, PreschoolFemaleGlycerolHumansInfantMaleMiddle AgedPhenylbutyrates4-phenylbutyric acidAmmoniaAntineoplastic AgentsGlycerolglycerol phenylbutyratePhenylbutyratesAmmoniaGlycerol phenylbutyrateHealth-related quality of lifePatient-reported outcomesSodium phenylbutyrateTreatment-related symptoms

Identifiers

PMID26296711
PMCPMC4804346
OpenAlexW2132062202

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.