The researchers found significant improvements in exercise performance during treatment with sildenafil compared to placebo. The findings included improved ventilatory efficiency and, in two subgroups of patients, an improved ability to perform moderate levels of exercise. These changes suggest an overall improvement in the physiology associated with this unique circulation.
"The enhanced exercise performance that we found in the study is exciting and may lead to an improvement in day to day activities for these children and young adults," Dr. Goldberg said. "However, it is important to note that while the results of this study are encouraging, more work is needed to determine whether the short-term benefit found in this study holds up over a longer period of time and whether there are any long-term side effects," he added.
"If the results from this preliminary study are validated in a larger sample over a longer period of time, it may be that this medication has the potential to improve quality of life for patients born with only one ventricle," Dr. Goldberg concluded.
Dr. Goldberg's coauthors were Michael McBride, Ph.D., Bradley Marino, M.D., Nicole Mirarchi, M.D., Brian D. Hanna, M.D., Gil Wernovsky, M.D., Stephen M. Paridon, M.D., and Jack Rychik, M.D., all from The Children's Hospital of Philadelphia; and Benjamin French, M.D., from the University of Pennsylvania School of Medicine.
Grants from The Mark H. and Blanche M. Harrington Foundation and from Big Hearts to Little Hearts provided funding for this study. Dr. Goldberg received support from the NIH T32 grant HL07915.
About The Children's Hospital of Philadelphia: The Children's Hospital of Philadelphia was founded in
|SOURCE The Children's Hospital of Philadelphia|
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