Abstract

Acute porphyrias are inherited metabolic disorders that cause life- threatening neurological symptoms, and are treated with intravenous heme. The objectives are to study heme arginate (the preferred preparation of heme) for treatment and prevention of acute attacks of these disorders and to determine the lowest dose that is effective biochemically.
The specific aims are to determine 1) safety and efficacy of heme arginate in acute attacks of porphyria; 2) value of heme arginate infusions for preventing frequent attacks of prophyria; and 3) the lowest effective dose of heme arginate in reducing prophyrin precursor and hemopexin levels in stable patients with acute intermittent prophyria. These studies with heme arginate, an orphan product, will support an application by the manufacturer for approval by the FDA. The studies will significantly improve management of acute porphyrias in the U.S.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000073-36
Application #
6276395
Study Section
Project Start
1997-12-01
Project End
1998-11-30
Budget Start
1997-10-01
Budget End
1998-09-30
Support Year
36
Fiscal Year
1998
Total Cost
Indirect Cost

  Institution

Name
University of Texas Medical Br Galveston
Department
Type
DUNS #
041367053
City
Galveston
State
TX
Country
United States
Zip Code
77555

  Publications

Gelman, Benjamin B; Endsley, Janice; Kolson, Dennis (2018) When do models of NeuroAIDS faithfully imitate ""the real thing""? J Neurovirol 24:146-155
Mourtakos, S P; Tambalis, K D; Panagiotakos, D B et al. (2017) Association between gestational weight gain and risk of obesity in preadolescence: a longitudinal study (1997-2007) of 5125 children in Greece. J Hum Nutr Diet 30:51-58
Ramanujam, V-M S; Nayeem, Fatima; Anderson, Karl E et al. (2017) Riboflavin as an independent and accurate biomarker for adherence in a randomized double-blind and placebo-controlled clinical trial. Biomarkers 22:508-516
Laffer, Cheryl L; Scott 3rd, Robert C; Titze, Jens M et al. (2016) Hemodynamics and Salt-and-Water Balance Link Sodium Storage and Vascular Dysfunction in Salt-Sensitive Subjects. Hypertension 68:195-203
Hosoki, Koa; Ying, Sun; Corrigan, Christopher et al. (2015) Analysis of a Panel of 48 Cytokines in BAL Fluids Specifically Identifies IL-8 Levels as the Only Cytokine that Distinguishes Controlled Asthma from Uncontrolled Asthma, and Correlates Inversely with FEV1. PLoS One 10:e0126035
Murai, Hiroki; Okazaki, Shintaro; Hayashi, Hisako et al. (2015) Alternaria extract activates autophagy that induces IL-18 release from airway epithelial cells. Biochem Biophys Res Commun 464:969-974
Diaz, Eva C; Herndon, David N; Porter, Craig et al. (2015) Effects of pharmacological interventions on muscle protein synthesis and breakdown in recovery from burns. Burns 41:649-57
Tuvdendorj, Demidmaa; Chinkes, David L; Bahadorani, John et al. (2014) Comparison of bolus injection and constant infusion methods for measuring muscle protein fractional synthesis rate in humans. Metabolism 63:1562-7
Sallam, Hanaa S; McNearney, Terry A; Chen, Jiande D Z (2014) Acupuncture-based modalities: novel alternative approaches in the treatment of gastrointestinal dysmotility in patients with systemic sclerosis. Explore (NY) 10:44-52
Petersen, John R; Stevenson, Heather L; Kasturi, Krishna S et al. (2014) Evaluation of the aspartate aminotransferase/platelet ratio index and enhanced liver fibrosis tests to detect significant fibrosis due to chronic hepatitis C. J Clin Gastroenterol 48:370-6

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