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A drug designed to fight anemia appears to double the risk of stroke in patients with diabetes and kidney disease without substantially improving their quality of life, a new study finds.

Darbepoetin alfa, marketed as Aranesp and known as an erythropoiesis-stimulating agent (ESA), is often prescribed for diabetic patients with chronic kidney disease and mild anemia.

“The benefits we assumed we would have by treating anemia were less striking and the risks were more striking,” said lead researcher Dr. Marc A. Pfeffer, a professor of medicine in the cardiovascular division of Brigham and Women’s Hospital in Boston.

“This provides new data for doctors and patients to make their own risk-benefit assessment,” he said. “There was a perception that treating anemia would make people feel so much better that we’ll take risks, but the benefit in quality of life was not as great as we thought, and there was a clear doubling of your risk for a stroke.”

The report was published in the Oct. 30 online edition of the New England Journal of Medicine to coincide with its scheduled presentation at the annual meeting of the American Society of Nephrology in San Diego.

For the study, Pfeffer’s team randomly assigned more than 4,000 patients with diabetes, chronic kidney disease and anemia to receive Aranesp or placebo. During the study, 632 patients receiving Aranesp died or suffered a cardiovascular event, compared to 602 of the patients receiving placebo.

As well, 101 patients taking Aranesp had a fatal or non-fatal stroke compared with 53 of the placebo patients, the researchers found. In addition, patients taking Aranesp reported only a modest improvement in their fatigue, the researchers noted.

In earlier studies, Aranesp and a similar drug, epoetin alfa, marketed as Procrit or Epogen, were linked to increased risk of death in cancer and stroke patients.

Pfeffer believes that people with more severe kidney disease, such as those on dialysis, might still find Aranesp beneficial and the risk acceptable.

“People on dialysis generally feel even worse and generally have even more severe anemia, and this class of therapy has been very helpful to them,” he said.

Because the drug was beneficial to these patients, doctors assumed it would help less severely anemic patients, Pfeffer said.

“But this use of ESAs exceeded the data,” he said. “Now we have the data, and we will revisit how the drug is used now.”

Dr. Phillip Marsden, a professor of medicine at the University of Toronto and author of an accompanying journal editorial, said these findings mean that doctors and patients will have to discuss whether or not to start the medication.

“For most of these patients, the modest improvement in quality of life will not be enough to subject themselves to the increased risk of stroke and death,” he said.

ESAs have been used for two decades, Marsden noted. “It is a bit shocking that it took us 20 years to address whether or not these drugs were safe — and now we know more.”

Dr. Ajay Singh, clinical chief of the renal division and director of dialysis at Brigham and Women’s Hospital, said this “landmark study” raises the fundamental question of whether epoetin or darbepoetin should routinely be used in treating anemia of chronic kidney disease.

“Earlier studies raised the specter of increased risk with ESA treatment. This study definitively confirms that there is meaningful risk with routine use of ESAs,” said Singh, also an associate professor of medicine at Harvard Medical School.

“In my own practice, I will be cautious in using ESAs for most patients with chronic kidney disease, balancing risk with benefits and reserving treatment largely for patients who need frequent blood transfusions or who are candidates for a kidney transplant,” he said.

Awareness of COPD — chronic obstructive pulmonary disease — continues to grow in the United States, according to national survey results released today by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health.

Sixty-eight percent of adults are now aware of COPD, a disease that affects 1 in 5 people over age 45, compared with 64 percent last year, and 49 percent in a 2004 survey. Among a high risk group, those who are currently smoking, awareness rose to 74 percent compared to 69 percent a year ago.

Less than half of all adults, 44 percent, understand that the disease can be treated. November is National COPD Awareness Month.

“Awareness is an important first step,” said James P. Kiley, Ph.D., director, NHLBI Division of Lung Diseases. “However, awareness alone is not enough. People at risk of developing the disease need to know what the disease looks and feels like, and most importantly, to understand that it can be treated. The key is to get tested and start treatment as soon as possible.”

COPD, which is sometimes referred to as chronic bronchitis or emphysema, is a serious lung disease affecting 24 million men and women in the United States. However, half of them remain undiagnosed despite recognizable symptoms such as shortness of breath while doing activities that used to be easy, wheezing, or chronic cough (sometimes called a “smoker’s cough”) Eight out of 10 cases of COPD are due to smoking, typically affecting those over 40. The remaining cases are due to genetics or other environmental exposures.

The survey showed that physicians maintain an optimistic view about COPD treatability. Approximately 9 out of 10 primary care physicians agree that available treatments can optimize quality of life for their patients with COPD. However, the survey also showed that this message may not be familiar to their patients.

Symptoms of COPD were approximately two times more common among current smokers than former smokers, but current smokers are only half as likely to talk to their doctors about these symptoms. Survey results also showed that 41 percent of current smokers do not talk to their doctors about these symptoms because they do not want to hear another quit smoking message.

COPD is diagnosed with a simple noninvasive breathing test called spirometry, which can be conducted in a doctor’s office. Taking the test involves breathing hard and fast into a tube connected to a machine which measures the total amount of air exhaled, called the forced vital capacity or FVC, and how much air is exhaled in the first second, called the forced expiratory volume in one second or FEV1.

“We know that for many people, taking the step to talk to a doctor about their smoking and symptoms is difficult,” said Kiley. “But these actions, including testing of lung function, should be seen as proactive for better health.”

The NHLBI analyzed the results of the annual HealthStyles and DocStyles surveys of the public health attitudes, knowledge, practices, and lifestyle habits of consumers and health care professionals, conducted each year by Porter Novelli, communications contractor for NHLBI’s COPD Learn More Breathe Better campaign. The results represent a sample of 4,172 consumers through a mailed survey with a margin of error of plus or minus 1.5 percentage points and 1,000 physicians through a Web-based survey with a margin of error of plus or minus 3.1 percentage points. Both surveys were conducted in summer 2009.

The NHLBI initiated the first national awareness campaign on COPD, called the COPD Learn More Breathe Better campaign, in 2007 to improve knowledge about COPD among those already diagnosed and at risk for COPD, as well as health care providers — particularly those in a primary care setting. The program’s new effort, Country Conquers COPD, aims to reach and raise knowledge of COPD among people at-risk at country-themed fairs and festivals across the country.

Childhood brain cancer survivors have ongoing cognitive problems and achieve lower levels of education, employment and income than their siblings and survivors of other types of cancer, a U.S. study has found.

The findings, published by the American Psychological Association in the November issue of Neuropsychology, highlight the importance of programs to support childhood brain cancer survivors’ transition to adulthood, said Leah Ellenberg, a clinical faculty member of the David Geffen School of Medicine at the University of California, Los Angeles.

Ellenberg and colleagues analyzed responses to a questionnaire filled out by 785 childhood brain cancer survivors 16 years after their diagnosis. The same questionnaire was completed by 5,870 survivors of cancers such as leukemia, Hodgkin’s disease and bone tumors, and 379 siblings of childhood brain cancer survivors.

The study found that childhood brain cancer survivors reported significantly greater neurocognitive dysfunction than their siblings or other cancer survivors. All areas of cognitive function were affected in childhood brain cancer survivors, including organization and emotional regulation.

The most commonly reported problems were in memory and efficiency, such as forgetting what they’re doing in the middle of a task and being slower than others at completing work. More than half of childhood brain cancer survivors reported significant difficulty with at least one task efficiency item, a rate three times higher than among their siblings.

The most serious neurocognitive problems were reported by childhood brain cancer survivors with significant motor or sensory problems after treatment, those who were treated with radiation to their brains, and those who had tumors in the brain cortex rather than in lower brain regions, the researchers found.

The neurocognitive issues reported by childhood brain cancer survivors were associated with significantly poorer adaptation to adult life, including lower achievement in education, full-time employment and income. They were also less likely to be married, the study authors noted.

The study “underscores the need for continued attention to mitigating the long-term negative effects of [childhood brain cancers] and their treatment,” the study authors wrote. They added that it’s “important to investigate the benefits of early and consistent use of compensatory strategies, including assistive technology, transitional facilities to promote independent living, and job placement and coaching, to enhance functional outcomes.”

NIH-supported scientists at Seaside Therapeutics in Cambridge, Mass., are beginning a clinical trial of a potential medication designed to correct a central neurochemical defect underlying Fragile X syndrome, the most common inherited cause of intellectual disability. There has to date been no medication that could alter the disorder’s neurologic abnormalities. The study will evaluate safety, tolerability, and optimal dosage in healthy volunteers.

The work is the outcome of basic research that traced how an error in the fragile X mental retardation gene (FMR1) leads to changes in brain connections, called synapses. The changes in turn appear to be the mechanism for learning deficits in Fragile X syndrome. The new trial tests Seaside Therapeutics’ novel compound, STX107, that selectively and potently targets the synaptic defect.

Thomas R. Insel, M.D., director of the National Institute of Mental Health, said, “This project is the culmination of years of fundamental research, first identifying the genetic mutation and later deciphering the biochemical consequences of this mutation. Now, with the initiation of this first clinical study, we move one step closer to understanding how this novel candidate may play a critical role in improving the lives of individuals with Fragile X Syndrome.”

Randall Carpenter, M.D., president and chief executive officer of Seaside Therapeutics, and Mark Bear, Ph.D., Seaside’s scientific founder, are leading the research. Dr. Bear is a Howard Hughes Medical Institute investigator and a professor of neuroscience at the Massachusetts Institute of Technology, Cambridge, Mass.

The National Institute of Mental Health, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and the National Institute of Neurological Disorders and Stroke (NINDS) have provided grant support. Private foundations providing funding include the advocacy groups Autism Speaks and FRAXA Research Foundation.

Fragile X syndrome is the most common inherited cause of intellectual disability, affecting an estimated 1 in 4,000 males and 1 in 6,000 females.

The syndrome causes a range of developmental problems, including learning disabilities and cognitive impairment. People with Fragile X syndrome may have anxiety and attention deficit hyperactivity disorder. About one-third of males with Fragile X syndrome also have autism or autistic-like behavior that affects communication and social interaction. Usually, males, who have only a single X chromosome, are more severely affected than females.

People with Fragile X have DNA mutations in the FMR1 gene that, in effect, turn off the gene. Research in recent years by Dr. Bear and colleagues has identified the molecular consequences of this silencing of FMR1. Normally, the protein product of the FMR1 gene acts to dampen the synthesis of proteins at synapses that are stimulated via a specific class of receptors on brain cell — metabotropic glutamate receptors (mGluRs). Without the brake provided by FMR protein, synaptic protein synthesis is excessive and connections do not develop normally.

This basic research provided the basis on which to develop medications that could correct the defect.

The current study will focus on a compound, designated STX107, that selectively inhibits one type of mGluR receptor, mGluR5. Evidence in mice with Fragile X-like symptoms suggests that reducing levels of mGluR5 can restore normal synaptic protein synthesis and improve function.

The length of time a patient has to wait between lung cancer diagnosis and treatment is influenced by a number of health-care system factors, a new U.S. study finds.

Researchers at the University of Texas Southwestern Medical Center analyzed data on 482 patients diagnosed with non-small cell lung cancer.

They found that factors such as type of hospital (private or public), insurance coverage, age and race have a major impact on the time it takes for a patient diagnosed with lung cancer to receive treatment.

For example, 59 percent of patients treated at a public hospital had advanced (stage 3) lung cancer, compared with 37 percent of patients treated at a private hospital. The researchers also found significant differences in patient populations at public and private hospitals in terms of age, race and socioeconomic status.

“This study demonstrates that in a contemporary U.S. health-care system, intervals among suspicion, diagnosis and treatment vary widely and are predominately associated with system variables such as insurance and hospital type,” said study author Dr. David E. Gerber. “An organized and timely approach to subsequent diagnostic and therapeutic measures may benefit these individuals and reduce this health-care disparity.”