Precision Medicine: Making Warfarin Safer

Blood sample for PT INR test, diagnosis for coagulation disease

Caption: Finding the right dose of the drug warfarin can be tricky, even with this standard test to measure how fast a person’s blood clots.
Credit: Thinkstock/jarun011

Every year, thousands of older Americans require emergency treatment to stop bleeding caused by taking warfarin, a frequently prescribed blood-thinning pill. My own mother received this drug in her later years, and her doctors encountered significant challenges getting the dose right. The problem is too much warfarin causes potentially serious bleeding, while too little leaves those who need the drug vulnerable to developing life-threatening clots in their legs or heart. The difference between too little and too much is distressingly small. But what if before writing a prescription, doctors could test for known genetic markers to help them gauge the amount of warfarin that a person should take?

Such tests have been available to doctors and patients for a few years, but they have not been widely used. The recent results of a national clinical trial offer some of the most convincing evidence that it’s time for that to change. In this study of 1,650 older adults undergoing elective hip or knee surgery, patients whose genetic makeup was used to help determine their dose of warfarin were less likely to suffer adverse events, including major bleeding. This trial marks an encouraging success story for the emerging field of pharmacogenomics, the study of how the variations in our genes affect our responses to medicines.

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Protecting Kids: Developing a Vaccine for Respiratory Syncytial Virus

Baby at the Doctor's OfficeVaccines are one of biomedicine’s most powerful and successful tools for protecting against infectious diseases. While we currently have safe and effective vaccines to prevent measles, mumps, and a great many other common childhood diseases, we still lack a vaccine to guard against respiratory syncytial virus (RSV)—a leading cause of pneumonia among infants and young children.

Each year, more than 2 million U.S. children under the age of 5 require medical care for pneumonia and other potentially life-threatening lower respiratory infections caused by RSV [1,2]. Worldwide, the situation is even worse, with more than 30 million infections estimated to occur annually, most among kids in developing countries, where as many as 200,000 deaths may result [3]. So, I’m pleased to report some significant progress in biomedical research’s long battle against RSV: encouraging early results from a clinical trial of an experimental vaccine specifically designed to outwit the virus.

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Team Approach Helps Teen Depression

Team of Support

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As many as one in five U.S. teenagers experience an episode of major depression by the time they turn 18. Sadly, depression among teens often goes unrecognized, increasing the risk of suicide, substance abuse, and many other problems. Even among those who are diagnosed, few receive proper treatment. But now there’s a ray of hope from a new NIH-funded study that’s found success using a team approach that pairs depressed teens and their parents with a counselor [1].

Faced with a shortage of psychiatrists who specialize in child mental health, a multidisciplinary team from the Seattle Children’s Research Institute, University of Washington School of Medicine, and Group Health in Seattle decided to use a strategy called “collaborative care” to treat depressed teenagers. There are more than 70 clinical trials showing that team-based care approaches work well for adults with depression, but there were only two such previous studies in teens—and results were mixed.

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PCORnet: Meeting Clinical Trials’ Need for Speed

Doctor with an iPad-type device talking with a patient

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Comparative effectiveness research (CER) studies aim to determine what works (and what doesn’t) in the real world of clinical medicine. Such studies are essential to identify the safest, most effective ways of detecting, treating, and preventing disease. Yet, both patients and researchers are often frustrated by the agonizingly slow pace at which many CER studies proceed. Under our current system, it’s often a long, costly process to identify trial sites, recruit volunteers, run the study, compile data, and, finally, analyze the results. The time is ripe to revamp the way we do this.

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Bracing Successfully Treats Spine Curvature in Adolescents

BrAISTWhen the results of a clinical trial are remarkably good, or bad, we end the trial early to translate the findings into action. Today I’m happy to share exciting news about a trial that was stopped early because of a good outcome. This was an NIH-funded clinical trial that investigated the benefits of wearing a back brace for tweens and teens who suffer from a spine curvature called adolescent idiopathic scoliosis (AIS), which affects up to 3% of children and teens between ages 10 and 16. The trial revealed, overwhelmingly, that such a brace stops the progression of curves before they require surgery [1]. Continue reading