If you’ve got symptoms that might indicate a serious medical problem, your doctor will first choose the tests that he/she believes will lead to an accurate diagnosis. Then he will choose the treatments that he thinks are most likely to work for your condition.
But how does your doctor make these important decisions? Often, there is no clear evidence and it comes down to an educated guess—but guesses aren’t what you want when you’re facing a life-changing decision…
Traditional medical research has inherent limitations. Industry-sponsored studies, for example, often compare new drugs to placebos. To make real-life decisions, however, doctors and patients need to look to comparative effectiveness research (CER). This type of study makes head-to-head comparisons among existing treatments…and attempts to tailor them to individuals rather than the “average patient.”
The Patient-Centered Outcomes Research Institute (PCORI), authorized by Congress in 2010, funds this type of comparative research (more than 280 studies so far) on medical conditions ranging from depression to stroke and cancer. Among recent CER findings from studies funded by PCORI or the federal government–sponsored Agency for Healthcare Research and Quality…*
Every year, about half a million Americans go to ERs with the fearsome pain of kidney stones. Doctors usually order an abdominal computed tomography (CT) scan to make the diagnosis. CT scans are the most sensitive test, but they’re expensive…often reveal incidental (and usually harmless) abnormalities that lead to unnecessary tests…and expose patients to high doses of radiation that may increase cancer risks. Ultrasound, which is also used to diagnose kidney stones, is cheaper and safer. But until recently, experts didn’t know if it was equally effective.
The study: 2,759 patients suspected of having kidney stones were randomly assigned to have CT scans or ultrasounds.
Results: Ultrasounds and CT scans were found to be equally accurate. Patients given these tests had similar rates of adverse reactions (such as subsequent complications from kidney disease). But the ultrasound patients were exposed to far less radiation than those given CT scans.
Conclusion: Ultrasound should usually be the first choice when diagnosing kidney stones.
More than a decade ago, the landmark Diabetes Prevention Program study found that lifestyle changes (exercise, healthful eating, etc.) reduced the risk for diabetes by 58%. The study also found that the diabetes drug metformin reduced diabetes risk by 31%.
However, few at-risk patients take advantage of either of these approaches, most likely because lifestyle improvement programs can be expensive and time-consuming. PCORI researchers wondered if it might be possible to identify those patients who would strongly benefit from these approaches.
The study: In a re-analysis of data from the original study, complex statistical models were used to correlate diabetes risk factors—age, body mass index, waist circumference, physical activity, etc.—with treatment outcomes.
Conclusion: Metformin was clearly effective, but only in the 25% of patients with the highest risk of developing diabetes (based on highest glucose levels, waist circumference and other factors). Those in lower-risk groups showed little benefit from metformin. Lifestyle changes were found to offer good protection to virtually all patients.
Spinal stenosis, an often-painful narrowing of the spinal canal that may require surgery, is typically treated with injections that combine a glucocorticoid (a type of steroid) with an anesthetic such as lidocaine. It’s estimated that more than 2.2 million of these epidural injections are given every year. But the benefits have never been proved in rigorous, randomized, controlled clinical trials. Nor has it been shown that this drug combination is more effective than a single-drug injection.
The study: 400 patients with spinal stenosis were given one or two injections of either the two-drug shot or the lidocaine alone. They were then evaluated by doctors three and six weeks later.
Results: Patients given glucocorticoids plus lidocaine reported less leg pain and better physical function than those given lidocaine alone—but only at the three-week examination. At six weeks, patients in both groups had the same improvement in pain scores.
Conclusion: The steroid adds very little benefit. This is an important finding because even the short-term use of glucocorticoids can suppress calcium absorption and cause a reduction in bone density, which increases the risk for bone fracture. PCORI has funded a longer-term follow-up to this study.
Previous studies have shown that the blood-thinning drug warfarin (Coumadin) can lower stroke risk for stroke survivors with atrial fibrillation (AFib), a dangerous heartbeat irregularity. But these past studies have tended to exclude the elderly and patients with more than one health problem and not taken into account the impact of warfarin on the quality of life after stroke.
The study: Researchers looked at 12,552 patients in all age groups who had AFib after a stroke to determine if warfarin decreased the number of days spent in the hospital after treatment and discharge.
Results: Patients who were treated with warfarin at discharge from the hospital had 47 more days at home during the two-year follow-up. They also had improvements in standard cardiac outcomes, such as the number of heart attacks, etc.
Conclusion: Warfarin provided these benefits to all patients studied, but the benefit was slightly stronger in women, patients older than age 80 and those who had more severe strokes (groups less likely to be treated with warfarin in the past). Note: Warfarin can raise risk for bleeding, so risks and benefits should be carefully considered.
*For recent studies funded by PCORI, go to PCORI.org and click on “Research & Results.”
