The number of Americans taking cholesterol-lowering statin drugs to prevent cardiovascular disease could double under treatment recommendations issued Tuesday -- to an estimated 72 million people, or about 30 percent of US adults.
The long-awaited update to the cholesterol treatment guideline lowers the threshold for those deemed to be at significant risk for having a heart attack or stroke and encourages doctors to treat these patients with statins -- even if their cholesterol levels are not elevated.
"When you look at the toll that heart disease and stroke deaths take in this country, we probably need to treat more people with statin drugs," said Dr. Neil Stone, a professor at Northwestern University Feinberg School of Medicine and chairman of the expert panel that wrote the recommendations.
More than 700,000 Americans die every year from cardiovascular diseases, but studies have shown that patients who take statins are, as a group, able to reduce their risk of having a heart attack, stroke, or heart procedure by 25 percent. The new guideline acknowledges, however, that not everyone who qualifies for statin use should necessarily take the drugs. "We also emphasized that physicians and patients discuss the risks and benefits of the therapy, taking into account family history and other factors," Stone said.
Heart specialists said the 84-page guideline, issued by the American Heart Association and American College of Cardiology, is largely an improvement over the old recommendations issued by the federal government a decade ago, because it relies on evidence from large clinical trials. But it includes some drastic changes in the criteria for determining the patients who should take statins, which many cardiologists are likely to consider controversial.
In particular, the guideline urges doctors to use a new assessment tool to determine a patient's risk of having a heart attack or stroke, one that has not been tested in studies, and to prescribe more potent, higher-dose statins as a first-line treatment while abandoning other cholesterol-lowering drugs such as Zetia (ezetimibe) and Lopid (gemfibrozil). There is also concern that people taking statins might feel they don't have to watch what they eat or to exercise -- although the guideline says statins should be taken in addition to adopting lifestyle changes.
Some doctors have also expressed alarm over the possibility that almost one-third of American adults could now qualify for lifelong statin use, even if they do not have high cholesterol levels or any signs of heart disease.
About 50 percent of white men and 60 percent of African-American men in their 50s would now fall in the high-risk category of people who should be treated with statins, as would all men by the time they reach age 70, according to Dr. Roger Blumenthal, director of preventive cardiology at Johns Hopkins Hospital, who provided some input into the guidelines. Two-thirds of African-American women in their 60s would qualify, too, along with one-third of white women in that age group.
"This is a tectonic shift in thinking about how to select people for treatment," said Dr. Steven Nissen, chairman of cardiovascular medicine at the Cleveland Clinic, who was not involved with developing the new guideline. He provided the estimate that statin use could double under the new recommendations.
About half the members of the 15-member advisory panel that drafted the guideline had financial ties to statin makers, but none of those members was allowed to vote on the document, Stone said. While most statins are now available as generics and cost as little as $3 a month, a newer, more-potent one -- rosuvastatin (Crestor), made by AstraZeneca -- remains under patent until 2016 and retails for about $175 per month.
While AstraZeneca stands to gain from the new guideline, Merck, manufacturer of Zetia, which had more than $2 billion in sales last year, could lose big.
The guideline calls on doctors to use a new algorithm -- which takes into account race, gender, age, and heart disease risk factors such as high blood pressure and family history -- to calculate an individual's risk of having a heart attack or stroke in the next 10 years. Physicians are urged to prescribe statins to anyone between 40 and 75 years of age whose 10-year risk is 7.5 percent or greater.
Doctors are still advised to use statins to prevent heart disease deaths in people who have type 2 diabetes or who have had a previous heart attack or stroke.
Nissen said some doctors may be uncomfortable giving a high-dose statin to someone with a relatively low LDL cholesterol level, even if the 10-year risk is above the new threshold, "especially since we know the risks of muscle injury and pain get worse when you scale up the dose."
Studies have found that patients taking statins have an increased risk of muscle aches and a tiny risk of irreversible muscle destruction called rhabdomyolysis. Recent research has found that statin users also have about a 20 to 30 percent increased risk of developing diabetes while taking the drugs.
"Women, in particular, are of concern because they haven't been well represented in clinical trials and we know that diabetes can have more deadly consequences in women than in men," said Dr. Lori Mosca, who is director of preventive cardiology at New YorkC"(euro) "Presbyterian Hospital and authored guidelines on the prevention of heart disease in women. "I'm still going to use my best clinical judgement and won't put a healthy woman on a statin if she's exercising and eating well."
But statins -- which also include atorvastatin (Lipitor) and simvastatin (Zocor) -- are by and large considered safe.
All of the cardiologists who spoke with the Globe applauded the expert panel for advising against using statins to drive down LDL levels to certain target measurements -- which doctors now routinely do. "We never had data to suggest that using a higher dose or extra drugs to get to an LDL level of 70 instead of 90 helped patients do better," said Nissen.
But these experts had little positive to say about the new risk assessment tool. "It overestimates the risk in large numbers of people and is driven by age, smoking, and blood pressure more than cholesterol levels," said Dr. Paul Ridker, a cardiologist at Brigham and Women's Hospital. "A 55-year-old male smoker with high blood pressure would be deemed at high risk, but he should be told to stop smoking and given blood pressure drugs, not a statin."
Likely, many more patients will be put on statins who don't really benefit much from them, and this could have the unintended consequence, Mosca said, of providing a false sense of security. "Someone may loosen up their lifestyle or may not exercise or eat as well if they know they have a statin for protection," she said.