The purpose of this project is to study heme arginate for the treatment of the acute porphyrias (acute intermittent porphyria, variegate porphyria, and hereditary coproporphyria). Heme arginate (Normosang(R), Leiras) is a stable heme preparation for intravenous use that was developed and studied in Europe for acute porphyrias, but has not been available in the United States. The heme preparation currently approved by the FDA as an orphan product for the treatment of these disorders has a number of disadvantages, including poor chemical stability, a short shelf life and troublesome side effects such as coagulation abnormalities and phlebitis at the site of intravenous infusion. These features have limited the application of heme treatment in porphyria patients in the United States. Therefore, availability of heme arginate would be a distinct advantage to patients with these disorders.
The aims of these studies are 1) to examine the safety and efficacy of heme arginate in patients with acute attacks of porphyria in terms of producing remissions of symptoms and lowering porphyrin precursors; 2) to study the value of heme arginate infusions for the prevention of frequently occurring attacks of porphyria; 3) to carry out dose-ranging studies of effects of heme arginate on reducing porphyrin precursor levels in stable patients with acute intermittent porphyria. Enrollment in these multicenter studies has been completed and data is being analyzed in cooperation with the manufacturer with the aim of supporting the licensing of heme arginate in the U.S. The efficacy of a preventive regimen using heme therapy has been demonstrated for the first time.

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University of Texas Medical Br Galveston
United States
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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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