Norfloxacin For Hepatopulmonary Syndrome: A Pilot Study of a Rare Disease

• Main Author: Gupta, Samir
• Other Authors: Bayoumi, Ahmed
• Format: Others
• Language: en_ca
• Published: 2008
• Subjects: hepatopulmonary syndrome; rare diseases; pulmonary vascular disease; nitric oxide; cirrhosis; 0564
• Online Access: http://hdl.handle.net/1807/10431

Norfloxacin For Hepatopulmonary Syndrome: A Pilot Study of a Rare Disease Samir Gupta, Masters of Science, 2008 Graduate Department of Health Policy, Management and Evaluation University of Toronto Introduction: Hepatopulmonary Syndrome is a rare disease characterized by abnormal gas-exchange and a poor prognosis, with no known effective medical therapy. A rat model and preliminary human data suggest that this disease may be caused by intestinal bacterial overgrowth, systemic endotoxemia and increased nitric oxide. Methods: We conducted a pilot crossover randomized controlled trial of norfloxacin versus placebo over four weeks, in seven subjects with HPS or a milder condition called pre-HPS, with a primary outcome of alveolar-arterial oxygen gradient (AaDO2). Results: There was no trend toward improved AaDO2, this outcome and other intermediate outcomes were highly variable, and results suggested that a longer treatment course might be necessary. We identified multiple obstacles to recruitment. Conclusion: We believe that a full-scale study of norfloxacin therapy for HPS will require 1) a six-month therapeutic period, 2) more specific HPS diagnostic criteria for clinical and study populations, and 3) creative recruitment maneuvers.