Design and rationale of the Fontan Udenafil Exercise Longitudinal (FUEL) trial.

Autor: Goldberg DJ; Division of Cardiology, The Children's Hospital of Philadelphia, Perelman School of Medicine, Philadelphia, PA 19104. Electronic address: goldbergda@email.chop.edu., Zak V; New England Research Institutes, Watertown, MA 02472., Goldstein BH; Division of Cardiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229., McCrindle BW; Division of Cardiology, The Hospital for Sick Children, University of Toronto, Toronto, Ontario M5G 1X8., Menon SC; Division of Pediatric Cardiology, University of Utah, Salt Lake City, UT 84132., Schumacher KR; Division of Cardiology, C.S. Mott Children's Hospital, Ann Arbor, MI 48109., Payne RM; Division of Cardiology, Riley Hospital for Children, Indianapolis, IN 46202., Rhodes J; Department of Cardiology, Children's Hospital Boston, Boston, MA 02115., McHugh KE; Division of Pediatric Cardiology, Medical University of South Carolina, Charleston, SC 29425., Penny DJ; Division of Cardiology, Texas Children's Hospital, Houston, TX 77030., Trachtenberg F; New England Research Institutes, Watertown, MA 02472., Hamstra MS; Division of Cardiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229., Richmond ME; Division of Pediatric Cardiology, Morgan Stanley Children's Hospital, Columbia University Medical Center, New York, NY 10032., Frommelt PC; Division of Cardiology, Medical College of Wisconsin, Children's Hospital of Wisconsin, Milwaukee, WI 53226., Files MD; Division of Cardiology, Seattle Children's Hospital, Seattle, WA 98105., Yeager JL; Consultant to Mezzion Pharma Co. Ltd., Mezzion Pharma Co. Ltd., Seoul, South Korea 135-879., Pemberton VL; Division of Cardiovascular Sciences, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD 20892., Stylianou MP; Division of Cardiovascular Sciences, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD 20892., Pearson GD; Division of Cardiovascular Sciences, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD 20892., Paridon SM; Division of Cardiology, The Children's Hospital of Philadelphia, Perelman School of Medicine, Philadelphia, PA 19104.
Jazyk: angličtina
Zdroj: American heart journal [Am Heart J] 2018 Jul; Vol. 201, pp. 1-8. Date of Electronic Publication: 2018 Apr 03.
DOI: 10.1016/j.ahj.2018.03.015
Abstrakt: The Fontan operation creates a circulation characterized by elevated central venous pressure and low cardiac output. Over time, these characteristics result in a predictable and persistent decline in exercise performance that is associated with an increase in morbidity and mortality. A medical therapy that targets the abnormalities of the Fontan circulation might, therefore, be associated with improved outcomes. Udenafil, a phosphodiesterase type 5 inhibitor, has undergone phase I/II testing in adolescents who have had the Fontan operation and has been shown to be safe and well tolerated in the short term. However, there are no data regarding the long-term efficacy of udenafil in this population. The Fontan Udenafil Exercise Longitudinal (FUEL) Trial is a randomized, double-blind, placebo-controlled phase III clinical trial being conducted by the Pediatric Heart Network in collaboration with Mezzion Pharma Co, Ltd. This trial is designed to test the hypothesis that treatment with udenafil will lead to an improvement in exercise capacity in adolescents who have undergone the Fontan operation. A safety extension trial, the FUEL Open-Label Extension Trial (FUEL OLE), offers the opportunity for all FUEL subjects to obtain open-label udenafil for an additional 12 months following completion of FUEL, and evaluates the long-term safety and tolerability of this medication. This manuscript describes the rationale and study design for FUEL and FUEL OLE. Together, these trials provide an opportunity to better understand the role of medical management in the care of those who have undergone the Fontan operation.
(Copyright © 2018 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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