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Accepted (AM)Open Access (License Unspecified), Open
Journal article
Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials
Molecular genetics and metabolism, v 116(1-2)
Sep 2015
PMID: 26296711
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
Health 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.
Symptoms 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).
After 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.
The 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.
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Details
- Title
- Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials
- Creators
- Sandesh C S Nagamani - Baylor College of MedicineGeorge A Diaz - Icahn School of Medicine at Mount SinaiWilliam Rhead - Medical College of WisconsinSusan A Berry - University of MinnesotaCynthia Le Mons - National Urea Cycle Disorders FoundationUta Lichter-Konecki - Drexel UniversityJames Bartley - Long Beach Memorial Medical CenterAnnette Feigenbaum - Boston Children's HospitalAndreas Schulze - University of TorontoNicola Longo - University of UtahWilliam Berquist - Stanford UniversityRenata Gallagher - University of California, San FranciscoDennis Bartholomew - Nationwide Children's HospitalCary O Harding - Oregon Health & Science UniversityMark S Korson - Tufts UniversityShawn E McCandless - Case Western Reserve UniversityWendy Smith - Maine Medical CenterJerry Vockley - University of PittsburghDavid Kronn - Westchester Medical CenterRobert Zori - University of Florida HealthStephen Cederbaum - University of California at Los AngelesJ Lawrence Merritt, 2nd - Seattle Children's Hospital, 4800 Sand Point Way NE M/S W-65945, Seattle, WA 98105, USADerek Wong - University of California at Los AngelesDion F Coakley - Horizon TherapeuticsBruce F Scharschmidt - Horizon TherapeuticsKlara Dickinson - Anthera PharmaceuticalsMiguel Marino - Oregon Health & Science UniversityBrendan H Lee - Baylor College of MedicineMasoud Mokhtarani - Horizon Therapeutics
- Publication Details
- Molecular genetics and metabolism, v 116(1-2)
- Publisher
- Elsevier
- Grant note
- UL1RR29887 / NCRR NIH HHS UL1RR31973 / NCRR NIH HHS UL1RR29890 / NCRR NIH HHS R01 DK102641 / NIDDK NIH HHS UL1RR25780 / NCRR NIH HHS 1K08DK074573 / NIDDK NIH HHS UL1RR24140 / NCRR NIH HHS UL1TR000423 / NCATS NIH HHS 2013095 / Doris Duke Charitable Foundation UL1 RR031973 / NCRR NIH HHS M01RR00188 / NCRR NIH HHS UL1 TR001085 / NCATS NIH HHS UL1RR31988 / NCRR NIH HHS UL1RR25764 / NCRR NIH HHS UL1RR25752 / NCRR NIH HHS UL1 TR000114 / NCATS NIH HHS UL1 TR002319 / NCATS NIH HHS UL1 RR025744 / NCRR NIH HHS UL1 RR033176 / NCRR NIH HHS UL1 RR033183 / NCRR NIH HHS UL1RR25744 / NCRR NIH HHS U54RR019453 / NCRR NIH HHS UL1TR000005 / NCATS NIH HHS UL1RR33183 / NCRR NIH HHS UL1RR33176 / NCRR NIH HHS
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Pediatrics
- Web of Science ID
- WOS:000361423600005
- Scopus ID
- 2-s2.0-84940895916
- Other Identifier
- 991019167333004721
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- Collaboration types
- Domestic collaboration
- International collaboration
- Web of Science research areas
- Endocrinology & Metabolism
- Genetics & Heredity
- Medicine, Research & Experimental