Archives For research

PillRandomized controlled trials (RCTs) may have lots of problems, but they remain the “gold standard” to determine whether a drug or treatment works. A recent study published in the BMJ, again raises concerns about trusting clinical trial results. The situation is outlined in an article in the New York Times about the antidepressant drug Paxil (paroxetine) and its safety in teens. The original study was published in 2001 in the Journal of the American Academy of Child and Adolescent Psychiatry (JAACAP) and the authors concluded that Paxil is “generally well tolerated and effective” for adolescents with major depression. However, since that time, experts have questioned whether the data really supports this conclusion and whether Paxil is really safe in young adults with depression.

In the recent BMJ analysis, the authors looked at the data from the original 2001 study and also some additional data they were able to get from the drug company that makes Paxil, GlaxoSmithKline. They concluded that Paxil is neither safe nor effective in adolescents with depression.

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Scalpel combinedSeveral years ago a close friend asked me to recommend a surgeon for an elective procedure. I told her I had a colleague with a great bedside manner who also had great technical skills. He was absolutely the person I would go to if I needed general surgery. My friend went to see him but ended up using a different surgeon. When I asked her what she liked about the other guy, she said that he told her that he was “the best” surgeon to perform the procedure. She didn’t like his personality but he instilled her with confidence.

The truth is that there is no single “best” surgeon for everyone. And there are many factors that go into picking a surgeon: insurance issues, convenience, etc. And there are advantages to going to a surgeon who works well with our primary care physician (communication) and in having surgery in a place that has an electronic medical record that our primary care physician can access (coordination of care). But the most important thing is how skilled the surgeon is at performing the surgery we need. How do we figure that out? The short answer is that we don’t.

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N-of-1During my medical training, we were taught that if a patient responds to a treatment, it doesn’t necessarily mean that every patient will respond in the same way. The results in a single patient might be due to chance so it was important to look at the results of well-designed research studies before we could conclude that the treatment really worked.  In statistics, “N” refers to the sample size in an experiment so we referred to these individual observations as “N-of-1” experiments (and we did not look at them very favorably).

Unfortunately, there are lots of problems with research studies – they take a long time to complete, the patients in the studies are very carefully selected and may be very different from you, there is often bias in the way the results are interpreted, etc.

What if doctors and patients had tools that allowed them to design high-quality experiments specifically for the individual patient?

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microbiome2Remember in elementary school science class when we put samples of our hair and saliva in Petri dishes to see what grew? We even used cotton swabs to test the surfaces of our desks and bacteria grew in a few days. These experiments were designed to show us that we have lots of bacteria inside and on our bodies – and all around us. There are microbes (bacteria, viruses, fungi, etc.) everywhere in our “built environment” – the buildings we live and work in – as seen in this incredible animation.

Research suggests that the microbes in our guts play an important role in the development of disease. This collection of organisms, referred to as the microbiome (although technically the collection of organisms is called the microbiota and the genes of those organisms are called the microbiome), may play a role in the development of many diseases including diabetes, obesity, rheumatoid arthritis, asthma, allergies, Crohn’s disease, autism and cancer.

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Health is life

January 30, 2015 — 1 Comment

Healthislife

When I was an infectious diseases specialist, most of the patients I saw were hospital inpatients but I also saw a few outpatients. They came to see me because of weeks or months of symptoms that their doctors couldn’t figure out and were often worried that they had a mysterious infection that was hard to diagnose. All of these patients had real symptoms – they were extremely tired, had headaches, muscle pains and sore throats. They generally arrived with stacks of medical records – numerous lab tests and notes from other doctors. I also noticed that many of them had serious “real life” problems – bad marriages, difficulties at work, housing problems, sick relatives and more. Perhaps they really did have an infection that I couldn’t find but I also began to wonder if their symptoms were caused by the stress.

My intuition was that many of these patients would benefit from speaking with a social worker, marriage counselor, psychologist, an expert in finding affordable housing or a financial planner. Unfortunately, these services were not part of our health care system. I suspected that many of my patients would get better if we were able to treat the whole patient, not just the symptoms.

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The gut and the brain

December 19, 2014 — Leave a comment

Gut BrainWe don’t normally think about the gut and the brain being connected. And yet many of us have gotten a stomach ache, nausea or diarrhea from stress or a feeling of “butterflies” from excitement. Or we may experience pleasure from certain foods or feel a need to eat when under stress.

The vagus nerve travels between the brain and other organs in the body and can transmit messages in both directions. The brain can send messages to the gut through chemicals (called neurotransmitters). The gut has its own nervous system (called the enteric nervous system or ENS) that controls digestion. But scientists now think that the ENS can also produce neurotransmitters to send to the brain.

The big question is whether the gut can actually cause symptoms and diseases of the nervous system.

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The power of placebos

November 19, 2014 — Leave a comment

placeboLots of people talk about the placebo effect but what exactly is it?

The most reliable clinical studies compare a treatment that is being tested with a fake treatment (called a placebo). Generally, half the people in the study get the treatment and half get the placebo and the then the two groups are compared. In the case of pills the placebo is often a sugar pill. Researchers can even test the effectiveness of a surgical procedure by comparing it with a sham or fake procedure. In these studies (called randomized controlled trials or RCTs), patients (and their healthcare teams) don’t know who is getting the pill or procedure being studied and who is getting the placebo. The reason for this is that patients sometimes get better when they are given a placebo because they believe they will get better (called the “placebo effect”) or because their disease got better on its own.

So can patients get better just by believing they will get better? And can doctors actually prescribe placebos to help people get better?

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Research in real time

October 15, 2014 — Leave a comment

Big Data2This past week, I was reminded of the problem of fraud in medical research when the British courts ruled that the mother of a child with autism had lied about her child’s symptoms. The woman was a supporter of Dr. Andrew Wakefield, the British doctor who published studies connecting the Measles Mumps and Rubella – MMR – vaccine to autism based on “data” he made up.

Published medical studies, especially randomized controlled trials, remain the most reliable way for doctors to make treatment decisions. But, in addition to fraud, there are many other problems with medical research studies including the fact that they cost a lot of money to perform and take a long time to complete. An even bigger problem is that research studies have not been done for most of the questions that doctors and patients want answered. And even if there is a relevant published study, the people in the study may be very different from you.

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ExpertThanks to the work of organizations like the Society for Participatory Medicine and patient advocates like e-patient Dave, the voice of the patient is being heard. And I’ve written before about organizations like the ImproveCareNow network where patients and families are treated as equal partners in quality improvement efforts.

I love that patients and families are being recognized for their expertise and that healthcare organizations are starting to involve patients as team members from the beginning of projects. I also love that organizations like PCORI (Patient Centered Outcomes Research Institute) are recognizing the importance of asking patients the research questions and outcomes that are most important to them.

So this is all really good, right? Yes, but…

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Bacteria are our friends…but not all the time.

While I believe that we need to keep the bacteria in our bodies happy and that the improved cleanliness of modern life may be causing problems, there is also no question that bacteria are our enemies as well. You don’t have to look very far to see examples of how bacteria can cause serious illness or even death – meningococal meningitis, pneumococcal pneumonia, salmonella and tuberculosis to name a few. In most cases, antibiotics are required to treat these infections (or vaccines to prevent the infections).

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