Anemia treatment no better than placebo, increases stroke risk, study says
By Elizabeth Cooney, Globe Correspondent
A drug commonly prescribed to treat anemia in patients with both type 2 diabetes and kidney disease worked no better than placebo in reducing deaths or cardiovascular problems it was thought to prevent, according to a paper published online tonight. Instead, people who took the drug, Aranesp, were almost twice as likely to have strokes as patients given a dummy pill.
The study, led by Boston researchers, is the first trial to compare this class of anemia drugs to a placebo in the ability to prevent death and serious complications -- even though the drugs have been in use for decades.
"The risks of the therapy were bigger than we thought, namely stroke, and the benefits were less than we thought, in quality of life," said Dr. Marc Pfeffer, a cardiologist at Brigham and Women's Hospital who led an international group studying Aranesp, which raises red blood cell levels.
Patients with type 2 diabetes and kidney disease often become anemic, and heart and kidney problems can be accelerated by anemia, so doctors have long prescribed Aranesp (darbepoetin alfa) and similar drugs believing that treating the anemia would improve patient's prognosis. The
study confirmed that patients taking Aranesp required fewer blood transfusions and suffered less fatigue, but other hoped-for outcomes were not seen.
Dr. Steven Woloshin of the Dartmouth Institute for Health Policy and Clinical Practice said the findings reinforce a crucial message in medicine. "This current study is an important reminder of how important it is not to assume the treatments work, but to prove them," said Woloshin, who was not involved in the study.
Aranesp, made by California-based Amgen, was approved by the US Food and Drug Administration in 2001 for use in kidney patients, but a similar drug also made by Amgen has been prescribed for 20 years. The drugs are also used to treat anemia in cancer patients. Amgen has sold more than $11 billion of Aranesp.
The study results were released just hours after attorneys general from Massachusetts and 14 other states sued Amgen in federal court in Boston alleging that the company offered kickbacks to doctors to boost sales of Aranesp. The states, which joined a former company sales representative who earlier filed a whistleblower suit under seal, allege that Amgen provided doctors excess amounts of the drug in product vials, and encouraged them to bill insurers, including state Medicaid programs and Medicare, for the free Aranesp.
Amgen issued a statement, saying, "We believe that the allegations are without merit, and we look forward to the opportunity to examine these matters with the states before the Court."
Pfeffer's study, published by the New England Journal of Medicine, randomly assigned more than 4,000 patients -- none of whom was on dialysis -- to receive Aranesp or a placebo. This study design is considered the gold standard of drug trials.
It found that patients who took the drug were just as likely as patients taking placebo to have a heart attack, develop congestive heart failure, progress to end-stage renal disease and need dialysis, or die. In the Aranesp group, almost twice as many patients had strokes: 101 who
took the drug, compared with 53 who didn't.
A spokesman for Amgen, which sponsored the trial and was involved in its design but did not control the publication of its results, said the landmark study answered important questions about Aranesp. "We hoped that we could do more for patients beyond reducing the need
for transfusion and in fact improve cardiovascular disease," said Dr. Preston Klassen, executive medical director of global development at Amgen.
Pfeffer said it was challenging to conduct a trial after use of the drug was widespread. "When we started, we had lot of pushback from people in the medical community, saying you can't do it against placebo because you can't deny these people the right therapy," he said. "Somewhere along the line you have to stand back and say, 'How sound is the data?' "
Aranesp has been under scrutiny before. In 2007, the FDA issued its most severe warning after high doses harmed some kidney disease patients and cancer patients undergoing chemotherapy.
The FDA said the study's results do not change how regulators view the drug. "This study is consistent with what we already know," spokeswoman Karen Riley said in an e-mailed statement. "The ... study findings underscore the importance of adhering to the approved dosing recommendation."
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Elizabeth Cooney is a former
health reporter for the Worcester Telegram & Gazette, where she also was a
business reporter and an editor. Earlier in her career, she edited medical
books and journals at Little, Brown, and worked for Boston magazine.Boston Globe Health and Science staff:
- Gideon Gil, Health and Science Editor
- Ishani Ganguli, Short White Coat blogger
