Alicia Ault

NEW ORLEANS  – A review of a stroke registry shows that it may be safe to give clot-busting therapy to "wake-up" stroke patients who have the same clinical and imaging features as those of patients who are traditionally considered eligible for the therapies.

Clinicians have traditionally shied away from giving clot-busters to patients who have stroke symptoms upon awakening because the time of onset is generally unknown. But many of these patients likely have experienced the stroke within a few hours of arriving for treatment and could benefit from a clot-busting intervention, Dr. Dulka Manawadu said during a press briefing at the International Stroke Conference.

Determining how to identify which wake-up patients could benefit "is an area of growing importance because it may allow us to extend the indication for this effective treatment," said Dr. Manawadu, a stroke and general medicine consultant at King’s College Hospital in London.

Dr. Manawadu and her colleagues at King’s College analyzed patients in the hospital’s stroke registry who received alteplase (Activase) between January 2009 and December 2010. The study analyzed data for 326 unselected and consecutive patients with a stroke onset of 0 to 4.5 hours with a National Institute of Health Stroke Scale (NIHSS) score of 5 or greater, and 68 unselected, consecutive patients who awoke with symptoms, had an NIHSS of 5 or greater, and had an unknown time of onset.

At King’s College Hospital, clinicians can give thrombolysis to wake-up patients on a compassionate basis. Decisions are made on a case-by-case basis, and treatment is given with consent, said Dr. Manawadu. The patient’s history, clinical signs of stroke, and scan findings are all taken into consideration. Outside of the time constraint, wake-up patients have to meet all other eligibility criteria for clot-busting treatment. A nonenhanced CT scan must show an Alberta Stroke Program Early CT Score (ASPECTS) of 7 or greater. CT Perfusion (CTP) mismatch was also used to assess eligibility in some wake-up patients, but was not mandatory.

The mean age was 73 years for both arms. There was no significant difference between the groups in most baseline characteristics. However, there was significantly greater use of CTP in the wake-up group: 44 patients, or 65%, compared with 84 patients, or 26% of the comparator group. The wake-up patients also had a higher incidence of large-vessel stroke.

The outcomes were statistically similar for both groups, except for mean NIHSS scores at 24 hours. The mean score was significantly lower for the wake-up group than for the reference group (7.2 vs. 11.5). The rates were similar between the groups for having any intracranial hemorrhage ([ICH], 22% vs. 20%, respectively) or symptomatic ICH (2.9% vs. 3.4%). At 3 months after stroke, wake-up patients had a lower death rate (15% vs. 25%).

When the data for wake-up stroke patients were stratified by age, patients older than 80 years fared significantly worse than did those under 80.

The study was limited in that it was retrospective, but it did include all comers, Dr. Manawadu said. Despite the findings from the registry analysis, there have been no changes in protocol at King’s College; wake-up patients are still given thrombolysis on a case-by-case basis, she said.

Dr. Lee H. Schwamm, director of the telestroke and acute stroke services at Massachusetts General Hospital, Boston, said that parts of the study are reassuring and consistent with what would be expected. A surprising finding was that "the use of CT scanning alone was able to identify this cohort safely, and they could be treated with similar outcomes."

It is surprising in part because CT generally is not very sensitive in detecting ischemic stroke. If the findings hold up, it would be important because CT is more widely available than is MRI, Dr. Schwamm said in an interview.

Currently, few American clinicians are using clot-busting therapy for wake-up patients, and it would be premature to use the King’s College study as a basis for thrombolysis in those patients, Dr. Schwamm said.

The conference was sponsored by the American Heart Association. The study was funded by the Institutional Research and Development Board at King’s College Hospital, London. Dr. Manawadu and her colleagues reported having no relevant disclosures.

NEW ORLEANS  – A review of a stroke registry shows that it may be safe to give clot-busting therapy to "wake-up" stroke patients who have the same clinical and imaging features as those of patients who are traditionally considered eligible for the therapies.

Clinicians have traditionally shied away from giving clot-busters to patients who have stroke symptoms upon awakening because the time of onset is generally unknown. But many of these patients likely have experienced the stroke within a few hours of arriving for treatment and could benefit from a clot-busting intervention, Dr. Dulka Manawadu said during a press briefing at the International Stroke Conference.

Determining how to identify which wake-up patients could benefit "is an area of growing importance because it may allow us to extend the indication for this effective treatment," said Dr. Manawadu, a stroke and general medicine consultant at King’s College Hospital in London.

Dr. Manawadu and her colleagues at King’s College analyzed patients in the hospital’s stroke registry who received alteplase (Activase) between January 2009 and December 2010. The study analyzed data for 326 unselected and consecutive patients with a stroke onset of 0 to 4.5 hours with a National Institute of Health Stroke Scale (NIHSS) score of 5 or greater, and 68 unselected, consecutive patients who awoke with symptoms, had an NIHSS of 5 or greater, and had an unknown time of onset.

At King’s College Hospital, clinicians can give thrombolysis to wake-up patients on a compassionate basis. Decisions are made on a case-by-case basis, and treatment is given with consent, said Dr. Manawadu. The patient’s history, clinical signs of stroke, and scan findings are all taken into consideration. Outside of the time constraint, wake-up patients have to meet all other eligibility criteria for clot-busting treatment. A nonenhanced CT scan must show an Alberta Stroke Program Early CT Score (ASPECTS) of 7 or greater. CT Perfusion (CTP) mismatch was also used to assess eligibility in some wake-up patients, but was not mandatory.

The mean age was 73 years for both arms. There was no significant difference between the groups in most baseline characteristics. However, there was significantly greater use of CTP in the wake-up group: 44 patients, or 65%, compared with 84 patients, or 26% of the comparator group. The wake-up patients also had a higher incidence of large-vessel stroke.

The outcomes were statistically similar for both groups, except for mean NIHSS scores at 24 hours. The mean score was significantly lower for the wake-up group than for the reference group (7.2 vs. 11.5). The rates were similar between the groups for having any intracranial hemorrhage ([ICH], 22% vs. 20%, respectively) or symptomatic ICH (2.9% vs. 3.4%). At 3 months after stroke, wake-up patients had a lower death rate (15% vs. 25%).

When the data for wake-up stroke patients were stratified by age, patients older than 80 years fared significantly worse than did those under 80.

The study was limited in that it was retrospective, but it did include all comers, Dr. Manawadu said. Despite the findings from the registry analysis, there have been no changes in protocol at King’s College; wake-up patients are still given thrombolysis on a case-by-case basis, she said.

Dr. Lee H. Schwamm, director of the telestroke and acute stroke services at Massachusetts General Hospital, Boston, said that parts of the study are reassuring and consistent with what would be expected. A surprising finding was that "the use of CT scanning alone was able to identify this cohort safely, and they could be treated with similar outcomes."

It is surprising in part because CT generally is not very sensitive in detecting ischemic stroke. If the findings hold up, it would be important because CT is more widely available than is MRI, Dr. Schwamm said in an interview.

Currently, few American clinicians are using clot-busting therapy for wake-up patients, and it would be premature to use the King’s College study as a basis for thrombolysis in those patients, Dr. Schwamm said.

The conference was sponsored by the American Heart Association. The study was funded by the Institutional Research and Development Board at King’s College Hospital, London. Dr. Manawadu and her colleagues reported having no relevant disclosures.

NEW ORLEANS  – A review of a stroke registry shows that it may be safe to give clot-busting therapy to "wake-up" stroke patients who have the same clinical and imaging features as those of patients who are traditionally considered eligible for the therapies.

Clinicians have traditionally shied away from giving clot-busters to patients who have stroke symptoms upon awakening because the time of onset is generally unknown. But many of these patients likely have experienced the stroke within a few hours of arriving for treatment and could benefit from a clot-busting intervention, Dr. Dulka Manawadu said during a press briefing at the International Stroke Conference.

Determining how to identify which wake-up patients could benefit "is an area of growing importance because it may allow us to extend the indication for this effective treatment," said Dr. Manawadu, a stroke and general medicine consultant at King’s College Hospital in London.

Dr. Manawadu and her colleagues at King’s College analyzed patients in the hospital’s stroke registry who received alteplase (Activase) between January 2009 and December 2010. The study analyzed data for 326 unselected and consecutive patients with a stroke onset of 0 to 4.5 hours with a National Institute of Health Stroke Scale (NIHSS) score of 5 or greater, and 68 unselected, consecutive patients who awoke with symptoms, had an NIHSS of 5 or greater, and had an unknown time of onset.

At King’s College Hospital, clinicians can give thrombolysis to wake-up patients on a compassionate basis. Decisions are made on a case-by-case basis, and treatment is given with consent, said Dr. Manawadu. The patient’s history, clinical signs of stroke, and scan findings are all taken into consideration. Outside of the time constraint, wake-up patients have to meet all other eligibility criteria for clot-busting treatment. A nonenhanced CT scan must show an Alberta Stroke Program Early CT Score (ASPECTS) of 7 or greater. CT Perfusion (CTP) mismatch was also used to assess eligibility in some wake-up patients, but was not mandatory.

The mean age was 73 years for both arms. There was no significant difference between the groups in most baseline characteristics. However, there was significantly greater use of CTP in the wake-up group: 44 patients, or 65%, compared with 84 patients, or 26% of the comparator group. The wake-up patients also had a higher incidence of large-vessel stroke.

The outcomes were statistically similar for both groups, except for mean NIHSS scores at 24 hours. The mean score was significantly lower for the wake-up group than for the reference group (7.2 vs. 11.5). The rates were similar between the groups for having any intracranial hemorrhage ([ICH], 22% vs. 20%, respectively) or symptomatic ICH (2.9% vs. 3.4%). At 3 months after stroke, wake-up patients had a lower death rate (15% vs. 25%).

When the data for wake-up stroke patients were stratified by age, patients older than 80 years fared significantly worse than did those under 80.

The study was limited in that it was retrospective, but it did include all comers, Dr. Manawadu said. Despite the findings from the registry analysis, there have been no changes in protocol at King’s College; wake-up patients are still given thrombolysis on a case-by-case basis, she said.

Dr. Lee H. Schwamm, director of the telestroke and acute stroke services at Massachusetts General Hospital, Boston, said that parts of the study are reassuring and consistent with what would be expected. A surprising finding was that "the use of CT scanning alone was able to identify this cohort safely, and they could be treated with similar outcomes."

It is surprising in part because CT generally is not very sensitive in detecting ischemic stroke. If the findings hold up, it would be important because CT is more widely available than is MRI, Dr. Schwamm said in an interview.

Currently, few American clinicians are using clot-busting therapy for wake-up patients, and it would be premature to use the King’s College study as a basis for thrombolysis in those patients, Dr. Schwamm said.

The conference was sponsored by the American Heart Association. The study was funded by the Institutional Research and Development Board at King’s College Hospital, London. Dr. Manawadu and her colleagues reported having no relevant disclosures.

NEW ORLEANS – Patients with a very low or a very high hematocrit are at higher risk for death after a stroke, and anemic patients are at greatest risk, according to a study presented at the International Stroke Conference sponsored by the American Heart Association on Feb. 2.

Previous studies have shown that extremes of hematocrit increase mortality after myocardial infarction, congestive heart failure, and kidney disease. Dr. Jason J. Sico of the VA Connecticut Healthcare System and researchers from the Department of Veterans Affairs medical system explored whether there was a similar association in stroke. Previous stroke studies had not adjusted for stroke severity or a large number of comorbidities.

They found that "among stroke patients, severe anemia is a potent predictor of dying throughout the first year after a stroke," said Dr. Sico, who is also an assistant professor of neurology at Yale University, New Haven, Conn.

The researchers abstracted medical records for a sample from 131 Veterans Health Administration (VHA) hospitals of 3,965 patients admitted for a confirmed diagnosis of ischemic stroke in fiscal 2007. Patients with unavailable hematocrits, those who received thrombolytics, or those whose charts had inconsistent death dates were also excluded.

"Among stroke patients, severe anemia is a potent predictor of dying throughout the first year after a stroke."

The hematocrit, taken from 24 hours of admission, was divided into six tiers: less than or equal to 27% (defined as severe anemia); 28%-32% (moderate anemia); 33%-37% (mild anemia); 38%-42% (normal); 43%-47% (normal); and greater than or equal to 48% (polycythemia).

Researchers adjusted for age, National Institutes of Health Stroke Scale (NIHSS) score, comorbidities (including pneumonia, hypertension, hypercholesterolemia, diabetes, and history of cancer and heart disease), and Acute Physiology and Chronic Health Evaluation (APACHE)-III scores.

A total of 2% of the 3,750 patients analyzed had severe anemia, 6.2% had moderate anemia, and 17.9% had mild anemia. About 64% were in the normal categories. A total of 9% had a high hematocrit of greater than or equal to 48%.

People with lower hematocrits tended to be older and have higher APACHE scores, a higher Charlson index, a history of heart disease, and were more likely to have diabetes.

The risk of death was 2.5 to 3.5 times higher for patients with severe anemia (P = .013 for in-hospital and 30-day mortality; P = .002 at 6 months and P = .001 at 1 year). A high hematocrit was independently associated only with in-hospital mortality (OR 2.9, P = .004).

The study showed that having a history of severe anemia put stroke patients at a higher risk of death than having a history of other conditions, including cancer and heart disease, said Dr. Sico.

There are several potential mechanisms to explain why anemia might increase the risk of death, he said in an interview. With lower hematocrits, less blood and less oxygen circulate to various parts of the body. Long-term anemia also impairs the ability of the brain’s blood vessels to respond appropriately to a stroke.

A higher hematocrit also decreases blood flow to brain, said Dr. Sico. It causes a more turbulent blood flow to the brain, which could predispose the stroke patient to having a bad outcome.

Dr. Sico said that the study’s findings were limited to men because no women had been analyzed.

But he concluded that stroke patients with low or high hematocrits should be evaluated for potentially reversible causes, and that they should also be closely monitored during the post-stroke period.

Dr. Sico reported having no financial disclosures. The study was funded by the VA Health Services Research and Development Quality Enhancement Research Initiative.

NEW ORLEANS – Patients with a very low or a very high hematocrit are at higher risk for death after a stroke, and anemic patients are at greatest risk, according to a study presented at the International Stroke Conference sponsored by the American Heart Association on Feb. 2.

Previous studies have shown that extremes of hematocrit increase mortality after myocardial infarction, congestive heart failure, and kidney disease. Dr. Jason J. Sico of the VA Connecticut Healthcare System and researchers from the Department of Veterans Affairs medical system explored whether there was a similar association in stroke. Previous stroke studies had not adjusted for stroke severity or a large number of comorbidities.

They found that "among stroke patients, severe anemia is a potent predictor of dying throughout the first year after a stroke," said Dr. Sico, who is also an assistant professor of neurology at Yale University, New Haven, Conn.

The researchers abstracted medical records for a sample from 131 Veterans Health Administration (VHA) hospitals of 3,965 patients admitted for a confirmed diagnosis of ischemic stroke in fiscal 2007. Patients with unavailable hematocrits, those who received thrombolytics, or those whose charts had inconsistent death dates were also excluded.

"Among stroke patients, severe anemia is a potent predictor of dying throughout the first year after a stroke."

The hematocrit, taken from 24 hours of admission, was divided into six tiers: less than or equal to 27% (defined as severe anemia); 28%-32% (moderate anemia); 33%-37% (mild anemia); 38%-42% (normal); 43%-47% (normal); and greater than or equal to 48% (polycythemia).

Researchers adjusted for age, National Institutes of Health Stroke Scale (NIHSS) score, comorbidities (including pneumonia, hypertension, hypercholesterolemia, diabetes, and history of cancer and heart disease), and Acute Physiology and Chronic Health Evaluation (APACHE)-III scores.

A total of 2% of the 3,750 patients analyzed had severe anemia, 6.2% had moderate anemia, and 17.9% had mild anemia. About 64% were in the normal categories. A total of 9% had a high hematocrit of greater than or equal to 48%.

People with lower hematocrits tended to be older and have higher APACHE scores, a higher Charlson index, a history of heart disease, and were more likely to have diabetes.

The risk of death was 2.5 to 3.5 times higher for patients with severe anemia (P = .013 for in-hospital and 30-day mortality; P = .002 at 6 months and P = .001 at 1 year). A high hematocrit was independently associated only with in-hospital mortality (OR 2.9, P = .004).

The study showed that having a history of severe anemia put stroke patients at a higher risk of death than having a history of other conditions, including cancer and heart disease, said Dr. Sico.

There are several potential mechanisms to explain why anemia might increase the risk of death, he said in an interview. With lower hematocrits, less blood and less oxygen circulate to various parts of the body. Long-term anemia also impairs the ability of the brain’s blood vessels to respond appropriately to a stroke.

A higher hematocrit also decreases blood flow to brain, said Dr. Sico. It causes a more turbulent blood flow to the brain, which could predispose the stroke patient to having a bad outcome.

Dr. Sico said that the study’s findings were limited to men because no women had been analyzed.

But he concluded that stroke patients with low or high hematocrits should be evaluated for potentially reversible causes, and that they should also be closely monitored during the post-stroke period.

Dr. Sico reported having no financial disclosures. The study was funded by the VA Health Services Research and Development Quality Enhancement Research Initiative.

NEW ORLEANS – Patients with a very low or a very high hematocrit are at higher risk for death after a stroke, and anemic patients are at greatest risk, according to a study presented at the International Stroke Conference sponsored by the American Heart Association on Feb. 2.

Previous studies have shown that extremes of hematocrit increase mortality after myocardial infarction, congestive heart failure, and kidney disease. Dr. Jason J. Sico of the VA Connecticut Healthcare System and researchers from the Department of Veterans Affairs medical system explored whether there was a similar association in stroke. Previous stroke studies had not adjusted for stroke severity or a large number of comorbidities.

They found that "among stroke patients, severe anemia is a potent predictor of dying throughout the first year after a stroke," said Dr. Sico, who is also an assistant professor of neurology at Yale University, New Haven, Conn.

The researchers abstracted medical records for a sample from 131 Veterans Health Administration (VHA) hospitals of 3,965 patients admitted for a confirmed diagnosis of ischemic stroke in fiscal 2007. Patients with unavailable hematocrits, those who received thrombolytics, or those whose charts had inconsistent death dates were also excluded.

"Among stroke patients, severe anemia is a potent predictor of dying throughout the first year after a stroke."

The hematocrit, taken from 24 hours of admission, was divided into six tiers: less than or equal to 27% (defined as severe anemia); 28%-32% (moderate anemia); 33%-37% (mild anemia); 38%-42% (normal); 43%-47% (normal); and greater than or equal to 48% (polycythemia).

Researchers adjusted for age, National Institutes of Health Stroke Scale (NIHSS) score, comorbidities (including pneumonia, hypertension, hypercholesterolemia, diabetes, and history of cancer and heart disease), and Acute Physiology and Chronic Health Evaluation (APACHE)-III scores.

A total of 2% of the 3,750 patients analyzed had severe anemia, 6.2% had moderate anemia, and 17.9% had mild anemia. About 64% were in the normal categories. A total of 9% had a high hematocrit of greater than or equal to 48%.

People with lower hematocrits tended to be older and have higher APACHE scores, a higher Charlson index, a history of heart disease, and were more likely to have diabetes.

The risk of death was 2.5 to 3.5 times higher for patients with severe anemia (P = .013 for in-hospital and 30-day mortality; P = .002 at 6 months and P = .001 at 1 year). A high hematocrit was independently associated only with in-hospital mortality (OR 2.9, P = .004).

The study showed that having a history of severe anemia put stroke patients at a higher risk of death than having a history of other conditions, including cancer and heart disease, said Dr. Sico.

There are several potential mechanisms to explain why anemia might increase the risk of death, he said in an interview. With lower hematocrits, less blood and less oxygen circulate to various parts of the body. Long-term anemia also impairs the ability of the brain’s blood vessels to respond appropriately to a stroke.

A higher hematocrit also decreases blood flow to brain, said Dr. Sico. It causes a more turbulent blood flow to the brain, which could predispose the stroke patient to having a bad outcome.

Dr. Sico said that the study’s findings were limited to men because no women had been analyzed.

But he concluded that stroke patients with low or high hematocrits should be evaluated for potentially reversible causes, and that they should also be closely monitored during the post-stroke period.

Dr. Sico reported having no financial disclosures. The study was funded by the VA Health Services Research and Development Quality Enhancement Research Initiative.

NEW ORLEANS – Continuous cardiac outpatient telemetry monitoring for at least 21 days may be necessary to accurately identify atrial fibrillation as the cause of cryptogenic stroke, according to the results of a single-center, retrospective study.

Identifying these patients is important because many could be treated with anticoagulants that would reduce their stroke risk, said Dr. Daniel J. Miller, a neurologist at Henry Ford Hospital, Detroit.

About a third of all strokes are of unknown etiology. Paroxysmal atrial fibrillation (AF) is a potential cause of cryptogenic stroke and transient ischemic attacks (TIAs), and carries a high risk of future strokes, Dr. Miller said during a press briefing at the International Stroke Conference.

Physicians have not been sure about the best way to monitor these patients or how long to monitor them.

He and his colleagues followed-up on two small studies: one conducted by Dr. Ashis H. Tayal and colleagues (Neurology 2008;71:1696-1701) and another by Dr. Archit Bhatt and associates (Stroke Res. Treat. 2011 [doi:10.4061/2011/172074]). The Tayal study showed a 23% AF detection rate in cryptogenic stroke patients monitored for longer than 21 days. The Bhatt study found a similar AF rate in cryptogenic stroke.

Dr. Miller and his coinvestigators reviewed the records of 156 Henry Ford Hospital patients who’d had a cryptogenic stroke or TIA and had undergone monitoring with the Cardionet Mobile Cardiac Outpatient Telemetry (MCOT) device, which is commonly used at Henry Ford

The patients had a mean age of 68 years and half were women. The vast majority (97%) were not taking an anticoagulant. Hypertension was common, present in 87% of the patients. They had a fairly low mean score of 3 on the National Institutes of Health Stroke Scale (NIHSS), indicating that the stroke had not been severe.

After monitoring, 27 (17.3%) of the 156 patients had paroxysmal AF. The AF events lasted less than 30 seconds in two-thirds of the patients and longer than 30 seconds in 26%. The remaining 8% had persistently occurring AF. This finding was not surprising and was consistent with the Tayal report, Dr. Miller said in an interview. The monitors are very sensitive, in particular for those events longer than 30 seconds, but they might miss some episodes of less than 30 seconds, he said.

The review showed that the rate of AF detection rose with increased duration of monitoring. The detection rate on a Kaplan-Meier curve was estimated to be 4% at 48 hours, 9% at 7 days, 14% at 14 days, and 20% by 21 days. "Our study does show that in order to capture all these events you should continue to monitor for at least 21 days," Dr. Miller said.

A multivariate Cox regression analysis showed that for all patients, female gender (P = .002), premature atrial complexes (PAC) on electrocardiogram (P = .001), a 1-cm increase in the diameter of the left atrium (P = .033), and a 10% decline in left ventricular ejection fraction (P = .008) all were associated with increased risk of AF. For stroke patients, female gender, PAC, and increasing stroke severity on the NIHSS were all associated with increased risk.

PAC, a premature beat from the atrium, has been shown to be associated with AF. It was an especially strong predictor of AF in this study, Dr. Miller said.

He urged further study to determine optimal monitoring beyond 21 days for patients with cryptogenic stroke or TIA.

NEW ORLEANS – Continuous cardiac outpatient telemetry monitoring for at least 21 days may be necessary to accurately identify atrial fibrillation as the cause of cryptogenic stroke, according to the results of a single-center, retrospective study.

Identifying these patients is important because many could be treated with anticoagulants that would reduce their stroke risk, said Dr. Daniel J. Miller, a neurologist at Henry Ford Hospital, Detroit.

About a third of all strokes are of unknown etiology. Paroxysmal atrial fibrillation (AF) is a potential cause of cryptogenic stroke and transient ischemic attacks (TIAs), and carries a high risk of future strokes, Dr. Miller said during a press briefing at the International Stroke Conference.

Physicians have not been sure about the best way to monitor these patients or how long to monitor them.

He and his colleagues followed-up on two small studies: one conducted by Dr. Ashis H. Tayal and colleagues (Neurology 2008;71:1696-1701) and another by Dr. Archit Bhatt and associates (Stroke Res. Treat. 2011 [doi:10.4061/2011/172074]). The Tayal study showed a 23% AF detection rate in cryptogenic stroke patients monitored for longer than 21 days. The Bhatt study found a similar AF rate in cryptogenic stroke.

Dr. Miller and his coinvestigators reviewed the records of 156 Henry Ford Hospital patients who’d had a cryptogenic stroke or TIA and had undergone monitoring with the Cardionet Mobile Cardiac Outpatient Telemetry (MCOT) device, which is commonly used at Henry Ford

The patients had a mean age of 68 years and half were women. The vast majority (97%) were not taking an anticoagulant. Hypertension was common, present in 87% of the patients. They had a fairly low mean score of 3 on the National Institutes of Health Stroke Scale (NIHSS), indicating that the stroke had not been severe.

After monitoring, 27 (17.3%) of the 156 patients had paroxysmal AF. The AF events lasted less than 30 seconds in two-thirds of the patients and longer than 30 seconds in 26%. The remaining 8% had persistently occurring AF. This finding was not surprising and was consistent with the Tayal report, Dr. Miller said in an interview. The monitors are very sensitive, in particular for those events longer than 30 seconds, but they might miss some episodes of less than 30 seconds, he said.

The review showed that the rate of AF detection rose with increased duration of monitoring. The detection rate on a Kaplan-Meier curve was estimated to be 4% at 48 hours, 9% at 7 days, 14% at 14 days, and 20% by 21 days. "Our study does show that in order to capture all these events you should continue to monitor for at least 21 days," Dr. Miller said.

A multivariate Cox regression analysis showed that for all patients, female gender (P = .002), premature atrial complexes (PAC) on electrocardiogram (P = .001), a 1-cm increase in the diameter of the left atrium (P = .033), and a 10% decline in left ventricular ejection fraction (P = .008) all were associated with increased risk of AF. For stroke patients, female gender, PAC, and increasing stroke severity on the NIHSS were all associated with increased risk.

PAC, a premature beat from the atrium, has been shown to be associated with AF. It was an especially strong predictor of AF in this study, Dr. Miller said.

He urged further study to determine optimal monitoring beyond 21 days for patients with cryptogenic stroke or TIA.

NEW ORLEANS – Continuous cardiac outpatient telemetry monitoring for at least 21 days may be necessary to accurately identify atrial fibrillation as the cause of cryptogenic stroke, according to the results of a single-center, retrospective study.

Identifying these patients is important because many could be treated with anticoagulants that would reduce their stroke risk, said Dr. Daniel J. Miller, a neurologist at Henry Ford Hospital, Detroit.

About a third of all strokes are of unknown etiology. Paroxysmal atrial fibrillation (AF) is a potential cause of cryptogenic stroke and transient ischemic attacks (TIAs), and carries a high risk of future strokes, Dr. Miller said during a press briefing at the International Stroke Conference.

Physicians have not been sure about the best way to monitor these patients or how long to monitor them.

He and his colleagues followed-up on two small studies: one conducted by Dr. Ashis H. Tayal and colleagues (Neurology 2008;71:1696-1701) and another by Dr. Archit Bhatt and associates (Stroke Res. Treat. 2011 [doi:10.4061/2011/172074]). The Tayal study showed a 23% AF detection rate in cryptogenic stroke patients monitored for longer than 21 days. The Bhatt study found a similar AF rate in cryptogenic stroke.

Dr. Miller and his coinvestigators reviewed the records of 156 Henry Ford Hospital patients who’d had a cryptogenic stroke or TIA and had undergone monitoring with the Cardionet Mobile Cardiac Outpatient Telemetry (MCOT) device, which is commonly used at Henry Ford

The patients had a mean age of 68 years and half were women. The vast majority (97%) were not taking an anticoagulant. Hypertension was common, present in 87% of the patients. They had a fairly low mean score of 3 on the National Institutes of Health Stroke Scale (NIHSS), indicating that the stroke had not been severe.

After monitoring, 27 (17.3%) of the 156 patients had paroxysmal AF. The AF events lasted less than 30 seconds in two-thirds of the patients and longer than 30 seconds in 26%. The remaining 8% had persistently occurring AF. This finding was not surprising and was consistent with the Tayal report, Dr. Miller said in an interview. The monitors are very sensitive, in particular for those events longer than 30 seconds, but they might miss some episodes of less than 30 seconds, he said.

The review showed that the rate of AF detection rose with increased duration of monitoring. The detection rate on a Kaplan-Meier curve was estimated to be 4% at 48 hours, 9% at 7 days, 14% at 14 days, and 20% by 21 days. "Our study does show that in order to capture all these events you should continue to monitor for at least 21 days," Dr. Miller said.

A multivariate Cox regression analysis showed that for all patients, female gender (P = .002), premature atrial complexes (PAC) on electrocardiogram (P = .001), a 1-cm increase in the diameter of the left atrium (P = .033), and a 10% decline in left ventricular ejection fraction (P = .008) all were associated with increased risk of AF. For stroke patients, female gender, PAC, and increasing stroke severity on the NIHSS were all associated with increased risk.

PAC, a premature beat from the atrium, has been shown to be associated with AF. It was an especially strong predictor of AF in this study, Dr. Miller said.

He urged further study to determine optimal monitoring beyond 21 days for patients with cryptogenic stroke or TIA.

NEW ORLEANS – There may be a correlation between infection and stroke in children, according to research presented at the International Stroke Conference.

Acute infection is considered to be a risk factor for stroke in adults, so researchers from the University of California, San Francisco, decided to study whether it might also be a potential risk in children, who experience frequent infections.

Stroke, however, is still extremely rare in children: The incidence of acute arterial ischemic stroke is 2.4 per 100,000 children annually in the United States. A large proportion of those cases have no known cause, said Nancy K. Hills, Ph.D., assistant adjunct professor of neurology at UCSF, at a press briefing. Many of those children, however, have an underlying arteriopathy.

Dr. Hills and her colleagues found that children who had a stroke were eight times more likely to have visited a health provider for an infection within 1 month before the stroke, compared with controls. More than 1 month before the stroke, however, there was no difference in the number of visits for infection between cases and controls. The researchers could not prove a direct link between infection and stroke. "We really believe it’s not the infection that’s causing the stroke," Dr. Hills said.

"It’s not something that parents of healthy children need to worry about," she said, adding that the infections are "probably a trigger for something else." The researchers believe that the children who had infection and stroke "probably have some underlying predisposition that causes them to have an unusual response to a common infection."

The retrospective study examined medical records for 2.5 million children – aged 29 days to 19 years – who were members of Kaiser Permanente from 1993 to 2007. The investigators identified 126 acute ischemic strokes, and then randomly selected 378 age-matched controls (three controls per case) from the Kaiser population. The median age was 10.5 years, and there was a relatively similar mix of male and females. All races were proportionately represented, said Dr. Hills.

The researchers looked at both diagnosed infections and symptoms that were indicative of infection. Any history of infection after a stroke diagnosis was excluded. Once the index stroke was established, the researchers categorized infectious visits according to time frames: 0-2 days, 3-7 days, 8-28 days, 1-3 months, 3-6 months, 6-12 months, and 12-24 months before the stroke.

They found that children who had a stroke were much more likely to have had a visit for infection within 1 month of the index stroke (odds ratio, 8.37). The odds ratio increased to 182 for a visit for infection within 2 days of the stroke. Twenty-nine percent of those who had a stroke had a visit in the 2 days prior to the stroke, compared with 1% of controls for the same dates. In the 3- to 7-day window, 13% of children who had a stroke had an infection, compared with 2% of controls.

The authors concluded that the risk of stroke is substantially elevated within the week after a visit for infection, but that it is likely that these children have some susceptibility to stroke, and that the infection puts them in a prothrombotic state, Dr. Hills said at the meeting, which was sponsored by the American Heart Association.

She pointed out several limitations to the study, including the fact that the number of infections in both the cases and controls was likely underestimated. Also, the infections were not generally confirmed by lab data; they were based on empirical diagnoses.

The study was funded by the National Institute of Neurological Disorders and Stroke. The authors reported no relevant financial conflicts.

NEW ORLEANS – There may be a correlation between infection and stroke in children, according to research presented at the International Stroke Conference.

Acute infection is considered to be a risk factor for stroke in adults, so researchers from the University of California, San Francisco, decided to study whether it might also be a potential risk in children, who experience frequent infections.

Stroke, however, is still extremely rare in children: The incidence of acute arterial ischemic stroke is 2.4 per 100,000 children annually in the United States. A large proportion of those cases have no known cause, said Nancy K. Hills, Ph.D., assistant adjunct professor of neurology at UCSF, at a press briefing. Many of those children, however, have an underlying arteriopathy.

Dr. Hills and her colleagues found that children who had a stroke were eight times more likely to have visited a health provider for an infection within 1 month before the stroke, compared with controls. More than 1 month before the stroke, however, there was no difference in the number of visits for infection between cases and controls. The researchers could not prove a direct link between infection and stroke. "We really believe it’s not the infection that’s causing the stroke," Dr. Hills said.

"It’s not something that parents of healthy children need to worry about," she said, adding that the infections are "probably a trigger for something else." The researchers believe that the children who had infection and stroke "probably have some underlying predisposition that causes them to have an unusual response to a common infection."

The retrospective study examined medical records for 2.5 million children – aged 29 days to 19 years – who were members of Kaiser Permanente from 1993 to 2007. The investigators identified 126 acute ischemic strokes, and then randomly selected 378 age-matched controls (three controls per case) from the Kaiser population. The median age was 10.5 years, and there was a relatively similar mix of male and females. All races were proportionately represented, said Dr. Hills.

The researchers looked at both diagnosed infections and symptoms that were indicative of infection. Any history of infection after a stroke diagnosis was excluded. Once the index stroke was established, the researchers categorized infectious visits according to time frames: 0-2 days, 3-7 days, 8-28 days, 1-3 months, 3-6 months, 6-12 months, and 12-24 months before the stroke.

They found that children who had a stroke were much more likely to have had a visit for infection within 1 month of the index stroke (odds ratio, 8.37). The odds ratio increased to 182 for a visit for infection within 2 days of the stroke. Twenty-nine percent of those who had a stroke had a visit in the 2 days prior to the stroke, compared with 1% of controls for the same dates. In the 3- to 7-day window, 13% of children who had a stroke had an infection, compared with 2% of controls.

The authors concluded that the risk of stroke is substantially elevated within the week after a visit for infection, but that it is likely that these children have some susceptibility to stroke, and that the infection puts them in a prothrombotic state, Dr. Hills said at the meeting, which was sponsored by the American Heart Association.

She pointed out several limitations to the study, including the fact that the number of infections in both the cases and controls was likely underestimated. Also, the infections were not generally confirmed by lab data; they were based on empirical diagnoses.

The study was funded by the National Institute of Neurological Disorders and Stroke. The authors reported no relevant financial conflicts.

NEW ORLEANS – There may be a correlation between infection and stroke in children, according to research presented at the International Stroke Conference.

Acute infection is considered to be a risk factor for stroke in adults, so researchers from the University of California, San Francisco, decided to study whether it might also be a potential risk in children, who experience frequent infections.

Stroke, however, is still extremely rare in children: The incidence of acute arterial ischemic stroke is 2.4 per 100,000 children annually in the United States. A large proportion of those cases have no known cause, said Nancy K. Hills, Ph.D., assistant adjunct professor of neurology at UCSF, at a press briefing. Many of those children, however, have an underlying arteriopathy.

Dr. Hills and her colleagues found that children who had a stroke were eight times more likely to have visited a health provider for an infection within 1 month before the stroke, compared with controls. More than 1 month before the stroke, however, there was no difference in the number of visits for infection between cases and controls. The researchers could not prove a direct link between infection and stroke. "We really believe it’s not the infection that’s causing the stroke," Dr. Hills said.

"It’s not something that parents of healthy children need to worry about," she said, adding that the infections are "probably a trigger for something else." The researchers believe that the children who had infection and stroke "probably have some underlying predisposition that causes them to have an unusual response to a common infection."

The retrospective study examined medical records for 2.5 million children – aged 29 days to 19 years – who were members of Kaiser Permanente from 1993 to 2007. The investigators identified 126 acute ischemic strokes, and then randomly selected 378 age-matched controls (three controls per case) from the Kaiser population. The median age was 10.5 years, and there was a relatively similar mix of male and females. All races were proportionately represented, said Dr. Hills.

The researchers looked at both diagnosed infections and symptoms that were indicative of infection. Any history of infection after a stroke diagnosis was excluded. Once the index stroke was established, the researchers categorized infectious visits according to time frames: 0-2 days, 3-7 days, 8-28 days, 1-3 months, 3-6 months, 6-12 months, and 12-24 months before the stroke.

They found that children who had a stroke were much more likely to have had a visit for infection within 1 month of the index stroke (odds ratio, 8.37). The odds ratio increased to 182 for a visit for infection within 2 days of the stroke. Twenty-nine percent of those who had a stroke had a visit in the 2 days prior to the stroke, compared with 1% of controls for the same dates. In the 3- to 7-day window, 13% of children who had a stroke had an infection, compared with 2% of controls.

The authors concluded that the risk of stroke is substantially elevated within the week after a visit for infection, but that it is likely that these children have some susceptibility to stroke, and that the infection puts them in a prothrombotic state, Dr. Hills said at the meeting, which was sponsored by the American Heart Association.

She pointed out several limitations to the study, including the fact that the number of infections in both the cases and controls was likely underestimated. Also, the infections were not generally confirmed by lab data; they were based on empirical diagnoses.

The study was funded by the National Institute of Neurological Disorders and Stroke. The authors reported no relevant financial conflicts.

WASHINGTON – The historically low growth in health spending in 2009 continued through 2010, driven largely by the recession, officials from the Centers for Medicare and Medicaid announced Jan. 9.

Health spending in the United States grew 3.9% in 2010, to a total of $2.6 trillion or $8,402 per person. That was a 0.1% rise from 2009, which was already at an all-time low growth rate, according to the CMS.

As the nation's economy slumped throughout 2009 and 2010, consumers cut back on elective surgical procedures, emergency department visits, physician office visits, and prescription drug use, according to the officials.

“Even though the recession officially ended in 2009, its impact on the health sector appears to have continued into 2010,” said Anne Martin, an economist with the CMS.

Employers shifted the costs of insurance and care to employees. This, in turn, drove up out-of-pocket spending in 2010.

But overall, consumers spent only 1.8% more out-of-pocket in 2010 than they had in 2009, which was a slow rate of growth when compared with historical patterns, Ms. Martin said.

Consumers reacted to cost-shifting by choosing health insurance plans that offered lower premiums and higher deductibles, and by reducing, where they could, use of personal health care services. Medical prices and the U.S. population remained relatively stable before, during, and after the recession, and yet, personal health spending fell, indicating a willful pullback.

“The slower growth in personal health care spending was mainly driven by the slowdown in the use and intensity of health care goods and services,” Ms. Martin said.

The agency documented a shrinkage in use of hospital care and physician services as compared with historical levels.

Hospital spending grew only 5% to $814 billion in 2010, compared with 6% in 2009. There was a decline in median inpatient admissions, and slower growth in emergency department visits, outpatient visits, and outpatient surgeries.

Overall spending on physician and clinical services – totaling $515 billion in 2010 – accounted for 20% of total health spending. As consumers went to their doctors less frequently, fewer prescriptions were written. And, many of the prescriptions that were dispensed were for less expensive generic drugs. These and other factors led to the slowest rate of growth in prescription drug spending ever recorded – a 1% increase from 2009 to $259 billion. The data were published in the journal Health Affairs (Health Aff. 2012 [doi: 10.1377/hlthaff.2011.1135]).

Growth in spending on physician and clinical services also was historically low, growing 2.5% in 2010 as compared with 3.3% in 2009, according to Ms. Martin.

Meanwhile, as employers and private insurers reduced the amount they spent on health care, the federal government's share of health spending rose – to 29% or a total of $742 billion in 2010. The rise in federal spending also was attributed to federal subsidies to state Medicaid programs. Medicaid accounted for about 15% of the nation's health bill in 2010, at $401 billion.

In 2009, the federal government spent 22% more than it did in 2008; in 2010, spending rose by almost 9%. That compares with a 10% decrease in spending by states and localities in 2008, and a 4% increase in 2010.

Medicare saw an increase in enrollment, both in the Medicare Advantage managed care program and traditional fee-for-service Medicare.

The increase in traditional enrollment reversed a several-year pattern of decline. Overall, Medicare spending increased 5% in 2010 to $524 billion, but per-enrollee spending did not rise as quickly as it had in 2009.

This is because there was a big reduction in payments for certain types of home health services, but also because of low use of physician services.

Small increases in physician fees in 2009 and 2010 also kept a lid on Medicare spending.

Those increases were instituted by Congress in response to cuts that would otherwise have been required by Medicare's Sustainable Growth Rate formula.

The Affordable Care Act had a negligible impact on overall spending, perhaps accounting for less than 0.1% of the slowdown, according to the CMS economists. This is because few provisions were in effect in 2010, and some, such as coverage for patients with preexisting conditions, did not enroll as many people as had been expected.

WASHINGTON – The historically low growth in health spending in 2009 continued through 2010, driven largely by the recession, officials from the Centers for Medicare and Medicaid announced Jan. 9.

Health spending in the United States grew 3.9% in 2010, to a total of $2.6 trillion or $8,402 per person. That was a 0.1% rise from 2009, which was already at an all-time low growth rate, according to the CMS.

As the nation's economy slumped throughout 2009 and 2010, consumers cut back on elective surgical procedures, emergency department visits, physician office visits, and prescription drug use, according to the officials.

“Even though the recession officially ended in 2009, its impact on the health sector appears to have continued into 2010,” said Anne Martin, an economist with the CMS.

Employers shifted the costs of insurance and care to employees. This, in turn, drove up out-of-pocket spending in 2010.

But overall, consumers spent only 1.8% more out-of-pocket in 2010 than they had in 2009, which was a slow rate of growth when compared with historical patterns, Ms. Martin said.

Consumers reacted to cost-shifting by choosing health insurance plans that offered lower premiums and higher deductibles, and by reducing, where they could, use of personal health care services. Medical prices and the U.S. population remained relatively stable before, during, and after the recession, and yet, personal health spending fell, indicating a willful pullback.

“The slower growth in personal health care spending was mainly driven by the slowdown in the use and intensity of health care goods and services,” Ms. Martin said.

The agency documented a shrinkage in use of hospital care and physician services as compared with historical levels.

Hospital spending grew only 5% to $814 billion in 2010, compared with 6% in 2009. There was a decline in median inpatient admissions, and slower growth in emergency department visits, outpatient visits, and outpatient surgeries.

Overall spending on physician and clinical services – totaling $515 billion in 2010 – accounted for 20% of total health spending. As consumers went to their doctors less frequently, fewer prescriptions were written. And, many of the prescriptions that were dispensed were for less expensive generic drugs. These and other factors led to the slowest rate of growth in prescription drug spending ever recorded – a 1% increase from 2009 to $259 billion. The data were published in the journal Health Affairs (Health Aff. 2012 [doi: 10.1377/hlthaff.2011.1135]).

Growth in spending on physician and clinical services also was historically low, growing 2.5% in 2010 as compared with 3.3% in 2009, according to Ms. Martin.

Meanwhile, as employers and private insurers reduced the amount they spent on health care, the federal government's share of health spending rose – to 29% or a total of $742 billion in 2010. The rise in federal spending also was attributed to federal subsidies to state Medicaid programs. Medicaid accounted for about 15% of the nation's health bill in 2010, at $401 billion.

In 2009, the federal government spent 22% more than it did in 2008; in 2010, spending rose by almost 9%. That compares with a 10% decrease in spending by states and localities in 2008, and a 4% increase in 2010.

Medicare saw an increase in enrollment, both in the Medicare Advantage managed care program and traditional fee-for-service Medicare.

The increase in traditional enrollment reversed a several-year pattern of decline. Overall, Medicare spending increased 5% in 2010 to $524 billion, but per-enrollee spending did not rise as quickly as it had in 2009.

This is because there was a big reduction in payments for certain types of home health services, but also because of low use of physician services.

Small increases in physician fees in 2009 and 2010 also kept a lid on Medicare spending.

Those increases were instituted by Congress in response to cuts that would otherwise have been required by Medicare's Sustainable Growth Rate formula.

The Affordable Care Act had a negligible impact on overall spending, perhaps accounting for less than 0.1% of the slowdown, according to the CMS economists. This is because few provisions were in effect in 2010, and some, such as coverage for patients with preexisting conditions, did not enroll as many people as had been expected.

WASHINGTON – The historically low growth in health spending in 2009 continued through 2010, driven largely by the recession, officials from the Centers for Medicare and Medicaid announced Jan. 9.

Health spending in the United States grew 3.9% in 2010, to a total of $2.6 trillion or $8,402 per person. That was a 0.1% rise from 2009, which was already at an all-time low growth rate, according to the CMS.

As the nation's economy slumped throughout 2009 and 2010, consumers cut back on elective surgical procedures, emergency department visits, physician office visits, and prescription drug use, according to the officials.

“Even though the recession officially ended in 2009, its impact on the health sector appears to have continued into 2010,” said Anne Martin, an economist with the CMS.

Employers shifted the costs of insurance and care to employees. This, in turn, drove up out-of-pocket spending in 2010.

But overall, consumers spent only 1.8% more out-of-pocket in 2010 than they had in 2009, which was a slow rate of growth when compared with historical patterns, Ms. Martin said.

Consumers reacted to cost-shifting by choosing health insurance plans that offered lower premiums and higher deductibles, and by reducing, where they could, use of personal health care services. Medical prices and the U.S. population remained relatively stable before, during, and after the recession, and yet, personal health spending fell, indicating a willful pullback.

“The slower growth in personal health care spending was mainly driven by the slowdown in the use and intensity of health care goods and services,” Ms. Martin said.

The agency documented a shrinkage in use of hospital care and physician services as compared with historical levels.

Hospital spending grew only 5% to $814 billion in 2010, compared with 6% in 2009. There was a decline in median inpatient admissions, and slower growth in emergency department visits, outpatient visits, and outpatient surgeries.

Overall spending on physician and clinical services – totaling $515 billion in 2010 – accounted for 20% of total health spending. As consumers went to their doctors less frequently, fewer prescriptions were written. And, many of the prescriptions that were dispensed were for less expensive generic drugs. These and other factors led to the slowest rate of growth in prescription drug spending ever recorded – a 1% increase from 2009 to $259 billion. The data were published in the journal Health Affairs (Health Aff. 2012 [doi: 10.1377/hlthaff.2011.1135]).

Growth in spending on physician and clinical services also was historically low, growing 2.5% in 2010 as compared with 3.3% in 2009, according to Ms. Martin.

Meanwhile, as employers and private insurers reduced the amount they spent on health care, the federal government's share of health spending rose – to 29% or a total of $742 billion in 2010. The rise in federal spending also was attributed to federal subsidies to state Medicaid programs. Medicaid accounted for about 15% of the nation's health bill in 2010, at $401 billion.

In 2009, the federal government spent 22% more than it did in 2008; in 2010, spending rose by almost 9%. That compares with a 10% decrease in spending by states and localities in 2008, and a 4% increase in 2010.

Medicare saw an increase in enrollment, both in the Medicare Advantage managed care program and traditional fee-for-service Medicare.

The increase in traditional enrollment reversed a several-year pattern of decline. Overall, Medicare spending increased 5% in 2010 to $524 billion, but per-enrollee spending did not rise as quickly as it had in 2009.

This is because there was a big reduction in payments for certain types of home health services, but also because of low use of physician services.

Small increases in physician fees in 2009 and 2010 also kept a lid on Medicare spending.

Those increases were instituted by Congress in response to cuts that would otherwise have been required by Medicare's Sustainable Growth Rate formula.

The Affordable Care Act had a negligible impact on overall spending, perhaps accounting for less than 0.1% of the slowdown, according to the CMS economists. This is because few provisions were in effect in 2010, and some, such as coverage for patients with preexisting conditions, did not enroll as many people as had been expected.

The Food and Drug Administration on Jan. 31 approved the drug ivacaftor (Kalydeco) for cystic fibrosis patients aged 6 years and older who have the G551D mutation.

According to ivacaftor maker Vertex Pharmaceuticals, the approval is one of the fastest ever at the FDA. It took only 3 months from submission of data to approval.

Ivacaftor targets the G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. CFTR proteins regulate the flow of salt and water across the cells. A defective CFTR protein causes thick, sticky mucus to build up in the lungs, digestive tract, and other parts of the body, which can lead to respiratory and digestive problems and other complications.

The Vertex drug, taken orally twice a day, is a CFTR potentiator. It targets abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell; that, in turn, thins the mucus and the airways become less clogged, according to Vertex.

The FDA said that about 1,200 people – 4% of the 30,000 with cystic fibrosis in the United States – have the G551D mutation. Ivacaftor can help only those with the mutation. The agency and Vertex recommend FDA-approved molecular diagnostic testing before starting the therapy.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said Dr. Margaret A. Hamburg, FDA commissioner, in a statement.

Added Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, "Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

The FDA’s approval was based on two 48-week, placebo-controlled clinical studies that enrolled 213 patients. One study was in patients aged 12 years and older, and the other was in patients aged 6-11 years.

They all had at least one copy of the G551D mutation. The ivacaftor-treated groups had significant and sustained improvements in lung function, as well as in weight gain and some quality of life measurements, compared with those who received placebo, according to Vertex. They also had fewer pulmonary exacerbations.

The most common side effects were upper respiratory tract infection, headache, stomach ache, rash, diarrhea, and dizziness.

Vertex said it would begin shipping ivacaftor to U.S. pharmacies this week.

The Food and Drug Administration on Jan. 31 approved the drug ivacaftor (Kalydeco) for cystic fibrosis patients aged 6 years and older who have the G551D mutation.

According to ivacaftor maker Vertex Pharmaceuticals, the approval is one of the fastest ever at the FDA. It took only 3 months from submission of data to approval.

Ivacaftor targets the G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. CFTR proteins regulate the flow of salt and water across the cells. A defective CFTR protein causes thick, sticky mucus to build up in the lungs, digestive tract, and other parts of the body, which can lead to respiratory and digestive problems and other complications.

The Vertex drug, taken orally twice a day, is a CFTR potentiator. It targets abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell; that, in turn, thins the mucus and the airways become less clogged, according to Vertex.

The FDA said that about 1,200 people – 4% of the 30,000 with cystic fibrosis in the United States – have the G551D mutation. Ivacaftor can help only those with the mutation. The agency and Vertex recommend FDA-approved molecular diagnostic testing before starting the therapy.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said Dr. Margaret A. Hamburg, FDA commissioner, in a statement.

Added Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, "Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

The FDA’s approval was based on two 48-week, placebo-controlled clinical studies that enrolled 213 patients. One study was in patients aged 12 years and older, and the other was in patients aged 6-11 years.

They all had at least one copy of the G551D mutation. The ivacaftor-treated groups had significant and sustained improvements in lung function, as well as in weight gain and some quality of life measurements, compared with those who received placebo, according to Vertex. They also had fewer pulmonary exacerbations.

The most common side effects were upper respiratory tract infection, headache, stomach ache, rash, diarrhea, and dizziness.

Vertex said it would begin shipping ivacaftor to U.S. pharmacies this week.

The Food and Drug Administration on Jan. 31 approved the drug ivacaftor (Kalydeco) for cystic fibrosis patients aged 6 years and older who have the G551D mutation.

According to ivacaftor maker Vertex Pharmaceuticals, the approval is one of the fastest ever at the FDA. It took only 3 months from submission of data to approval.

Ivacaftor targets the G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. CFTR proteins regulate the flow of salt and water across the cells. A defective CFTR protein causes thick, sticky mucus to build up in the lungs, digestive tract, and other parts of the body, which can lead to respiratory and digestive problems and other complications.

The Vertex drug, taken orally twice a day, is a CFTR potentiator. It targets abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell; that, in turn, thins the mucus and the airways become less clogged, according to Vertex.

The FDA said that about 1,200 people – 4% of the 30,000 with cystic fibrosis in the United States – have the G551D mutation. Ivacaftor can help only those with the mutation. The agency and Vertex recommend FDA-approved molecular diagnostic testing before starting the therapy.

"Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup," said Dr. Margaret A. Hamburg, FDA commissioner, in a statement.

Added Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, "Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

The FDA’s approval was based on two 48-week, placebo-controlled clinical studies that enrolled 213 patients. One study was in patients aged 12 years and older, and the other was in patients aged 6-11 years.

They all had at least one copy of the G551D mutation. The ivacaftor-treated groups had significant and sustained improvements in lung function, as well as in weight gain and some quality of life measurements, compared with those who received placebo, according to Vertex. They also had fewer pulmonary exacerbations.

The most common side effects were upper respiratory tract infection, headache, stomach ache, rash, diarrhea, and dizziness.

Vertex said it would begin shipping ivacaftor to U.S. pharmacies this week.

The Patient-Centered Outcomes Research Institute, established by the Affordable Care Act, has issued a draft of its initial priorities.

PCORI, as it is known, is not a government agency; it is independent and nonprofit and has a 21-member board of governors. But the organization has been, and continues to be, a primary focus of objections by Republican lawmakers who have alleged that it was established to find ways to ration care.

In its mission statement, PCORI says it "helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community."

The board of governors has met eight times since November 2010. In late January, it issued a priority list that focused on five areas:

• Assessment of Options for Prevention, Diagnosis, and Treatment.

• Improving Health Care Systems.

• Communication and Dissemination Research.

• Addressing Disparities.

• Accelerating Patient-Centered Outcomes Research and Methodological Research.

"We want to hear from patients, caregivers, providers, and the wider health care community on whether our draft priorities and initial research agenda capture the broad areas where more evidence-based information is needed to make better decisions," PCORI Board Chair Eugene Washington, said in a statement.

Dr. Joe Selby, PCORI executive director, added, "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point."

The organization did spell out in its draft where it thinks research should focus in each of the five broad areas. For instance, research comparing prevention, diagnosis, and treatment options should focus on "clinical options with emphasis on patient preferences and decision making."

To accelerate patient-centered research, proposals should hone in on "use of registries and clinical data networks to support research about patient-centered outcomes."

The draft is very likely to change. The organization is seeking public comment until March 15. It will hold a "national forum" in the District of Columbia on Feb. 27. PCORI said that once it gathers all the public feedback, it will publish a report summarizing the input and how it led to changes in the draft.

The research priorities then have to be approved by the PCORI board of governors. The organization said it will issue its first grants in May.

PCORI expects to have about $150 million in funding in 2012. Beginning in 2013, it will be funded by a tax on each insured American – through private insurance or Medicare. It will receive an estimated $500 million a year.

Meanwhile, the consulting company Avalere Health is predicting where the Centers for Medicare and Medicaid Services will head with its comparative effectiveness research in 2012. The CMS conducts analyses of various technologies and therapies in making its National Coverage Decisions (NCDs).

In 2008, the CMS issued a list of 20 potential topics for public comment. So far, the agency has acted on 7 of those topics, Avalere noted in its report.

Avalere expects that in 2012, the CMS will focus heavily on oncology and molecular diagnostics, with a concentration on non–small-cell lung cancer therapies, localized prostate cancer treatments, pharmacogenomic testing for breast and colon cancers, and prostate and lung cancer screening.

The consulting company said that oncology is ripe for exploration by the CMS, in part because of an influx of costly new therapies and diagnostic tests, in particular, those that tailor a therapy to the patient. Forty-six percent of the 241 genetic tests that are approved and available for clinical use are for cancer, according to Avalere.

There have been no NCDs on molecular tests for oncology, but there have been decisions by local Medicare contractors, mostly negative.

The Patient-Centered Outcomes Research Institute, established by the Affordable Care Act, has issued a draft of its initial priorities.

PCORI, as it is known, is not a government agency; it is independent and nonprofit and has a 21-member board of governors. But the organization has been, and continues to be, a primary focus of objections by Republican lawmakers who have alleged that it was established to find ways to ration care.

In its mission statement, PCORI says it "helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community."

The board of governors has met eight times since November 2010. In late January, it issued a priority list that focused on five areas:

• Assessment of Options for Prevention, Diagnosis, and Treatment.

• Improving Health Care Systems.

• Communication and Dissemination Research.

• Addressing Disparities.

• Accelerating Patient-Centered Outcomes Research and Methodological Research.

"We want to hear from patients, caregivers, providers, and the wider health care community on whether our draft priorities and initial research agenda capture the broad areas where more evidence-based information is needed to make better decisions," PCORI Board Chair Eugene Washington, said in a statement.

Dr. Joe Selby, PCORI executive director, added, "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point."

The organization did spell out in its draft where it thinks research should focus in each of the five broad areas. For instance, research comparing prevention, diagnosis, and treatment options should focus on "clinical options with emphasis on patient preferences and decision making."

To accelerate patient-centered research, proposals should hone in on "use of registries and clinical data networks to support research about patient-centered outcomes."

The draft is very likely to change. The organization is seeking public comment until March 15. It will hold a "national forum" in the District of Columbia on Feb. 27. PCORI said that once it gathers all the public feedback, it will publish a report summarizing the input and how it led to changes in the draft.

The research priorities then have to be approved by the PCORI board of governors. The organization said it will issue its first grants in May.

PCORI expects to have about $150 million in funding in 2012. Beginning in 2013, it will be funded by a tax on each insured American – through private insurance or Medicare. It will receive an estimated $500 million a year.

Meanwhile, the consulting company Avalere Health is predicting where the Centers for Medicare and Medicaid Services will head with its comparative effectiveness research in 2012. The CMS conducts analyses of various technologies and therapies in making its National Coverage Decisions (NCDs).

In 2008, the CMS issued a list of 20 potential topics for public comment. So far, the agency has acted on 7 of those topics, Avalere noted in its report.

Avalere expects that in 2012, the CMS will focus heavily on oncology and molecular diagnostics, with a concentration on non–small-cell lung cancer therapies, localized prostate cancer treatments, pharmacogenomic testing for breast and colon cancers, and prostate and lung cancer screening.

The consulting company said that oncology is ripe for exploration by the CMS, in part because of an influx of costly new therapies and diagnostic tests, in particular, those that tailor a therapy to the patient. Forty-six percent of the 241 genetic tests that are approved and available for clinical use are for cancer, according to Avalere.

There have been no NCDs on molecular tests for oncology, but there have been decisions by local Medicare contractors, mostly negative.

The Patient-Centered Outcomes Research Institute, established by the Affordable Care Act, has issued a draft of its initial priorities.

PCORI, as it is known, is not a government agency; it is independent and nonprofit and has a 21-member board of governors. But the organization has been, and continues to be, a primary focus of objections by Republican lawmakers who have alleged that it was established to find ways to ration care.

In its mission statement, PCORI says it "helps people make informed health care decisions – and improves health care delivery and outcomes – by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader health care community."

The board of governors has met eight times since November 2010. In late January, it issued a priority list that focused on five areas:

• Assessment of Options for Prevention, Diagnosis, and Treatment.

• Improving Health Care Systems.

• Communication and Dissemination Research.

• Addressing Disparities.

• Accelerating Patient-Centered Outcomes Research and Methodological Research.

"We want to hear from patients, caregivers, providers, and the wider health care community on whether our draft priorities and initial research agenda capture the broad areas where more evidence-based information is needed to make better decisions," PCORI Board Chair Eugene Washington, said in a statement.

Dr. Joe Selby, PCORI executive director, added, "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point."

The organization did spell out in its draft where it thinks research should focus in each of the five broad areas. For instance, research comparing prevention, diagnosis, and treatment options should focus on "clinical options with emphasis on patient preferences and decision making."

To accelerate patient-centered research, proposals should hone in on "use of registries and clinical data networks to support research about patient-centered outcomes."

The draft is very likely to change. The organization is seeking public comment until March 15. It will hold a "national forum" in the District of Columbia on Feb. 27. PCORI said that once it gathers all the public feedback, it will publish a report summarizing the input and how it led to changes in the draft.

The research priorities then have to be approved by the PCORI board of governors. The organization said it will issue its first grants in May.

PCORI expects to have about $150 million in funding in 2012. Beginning in 2013, it will be funded by a tax on each insured American – through private insurance or Medicare. It will receive an estimated $500 million a year.

Meanwhile, the consulting company Avalere Health is predicting where the Centers for Medicare and Medicaid Services will head with its comparative effectiveness research in 2012. The CMS conducts analyses of various technologies and therapies in making its National Coverage Decisions (NCDs).

In 2008, the CMS issued a list of 20 potential topics for public comment. So far, the agency has acted on 7 of those topics, Avalere noted in its report.

Avalere expects that in 2012, the CMS will focus heavily on oncology and molecular diagnostics, with a concentration on non–small-cell lung cancer therapies, localized prostate cancer treatments, pharmacogenomic testing for breast and colon cancers, and prostate and lung cancer screening.

The consulting company said that oncology is ripe for exploration by the CMS, in part because of an influx of costly new therapies and diagnostic tests, in particular, those that tailor a therapy to the patient. Forty-six percent of the 241 genetic tests that are approved and available for clinical use are for cancer, according to Avalere.

There have been no NCDs on molecular tests for oncology, but there have been decisions by local Medicare contractors, mostly negative.

In another example of the continuing scrutiny being given to cardiac procedures, a cardiologist has filed a whistleblower suit against former colleagues, alleging that they knowingly bilked federal health programs over a 4-year period, from 2001-2005.

The suit was filed by Dr. Tullio Emanuele in the U.S. District Court for the Western District of Pennsylvania. It was under seal until Oct. 2011, and was served on the defendants on Jan. 17. The defendants have 20 days to respond.

Dr. Emanuele is now based in Kentucky, but had worked in an Erie-based practice, Medicor Associates. In his suit, he claims that his former colleagues at Medicor and at Flagship Cardiac, Vascular, and Thoracic Surgery of Erie – along with the UPMC Hamot Medical Center of Erie – engaged in billing practices that defrauded Medicare, Medicaid, and Tricare, among other insurers.

Carly Manino, manager of media relations at UPMC Hamot, said that the hospital would not comment. "It’s our policy not to comment on pending litigation," she said.

Whistleblower or "qui tam" suits are filed under the False Claims Act. Such suits allow private citizens with knowledge of false claims against the government to bring a lawsuit on behalf of the United States and to share in any recovery, according to the U.S. Department of Justice.

So far, the federal government has declined to intervene in the case, but the suit can be advanced by private litigators, according to Andy Stone, cocounsel on the Emanuele suit. The government may intervene at a later point, said Mr. Stone, whose practice is in Pittsburgh. The government can seek triple the amount of damages determined by the courts. Mr. Stone said that it’s not clear how much might be at stake in the case before discovery, but that given the potential number of claims, it could be millions of dollars.

The Pennsylvania suit also accuses the cardiologists and the hospital of violating the antikickback statutes that prohibit improper patient referrals.

In addition, the suit alleges that the named physicians performed unnecessary medical procedures, including cardiac catheterizations and vascular surgeries.

Mr. Stone said that he believes that cases alleging overuse of stents or other percutaneous interventions may be brought more often.

"These are important cases because the dollar amounts are big. The incentives are there – and substantial patient risk. You put those two things together and that makes for a real serious abuse situation," he said.

Indeed, the Emanuele suit seems to be the latest in a high-profile battle over the appropriateness of cardiac procedures, particularly stent placement. Last spring, Greensburg, Pa.–based Excela Health said that it had found that stents had been unnecessarily placed in almost 200 patients. In a turnaround, that health system has just won accreditation under the Accreditation for Cardiovascular Excellence (ACE) program that is sponsored by the Society for Cardiovascular Angiography and Interventions and the American College of Cardiology Foundation.

Those two organizations, along with the Maryland Chapter of the ACC, have been working to head off regulation of stent procedures, according to an editorial in the Journal of the American College of Cardiology. Instead, they are hoping to convince the state to require all Maryland hospitals to submit to a mandatory accrediting process through the groups’ ACE program.

The state of Maryland began looking into stenting as the result of another qui tam suit filed by three cardiologists against St. Joseph Medical Center in Towson, Md. The hospital settled for $22 million. The suit alleged that St. Joseph paid kickbacks in return for referrals from MidAtlantic Cardiovascular Associates.

St. Joseph also settled charges that cardiologist Mark Midei performed unnecessary stent procedures.

In another example of the continuing scrutiny being given to cardiac procedures, a cardiologist has filed a whistleblower suit against former colleagues, alleging that they knowingly bilked federal health programs over a 4-year period, from 2001-2005.

The suit was filed by Dr. Tullio Emanuele in the U.S. District Court for the Western District of Pennsylvania. It was under seal until Oct. 2011, and was served on the defendants on Jan. 17. The defendants have 20 days to respond.

Dr. Emanuele is now based in Kentucky, but had worked in an Erie-based practice, Medicor Associates. In his suit, he claims that his former colleagues at Medicor and at Flagship Cardiac, Vascular, and Thoracic Surgery of Erie – along with the UPMC Hamot Medical Center of Erie – engaged in billing practices that defrauded Medicare, Medicaid, and Tricare, among other insurers.

Carly Manino, manager of media relations at UPMC Hamot, said that the hospital would not comment. "It’s our policy not to comment on pending litigation," she said.

Whistleblower or "qui tam" suits are filed under the False Claims Act. Such suits allow private citizens with knowledge of false claims against the government to bring a lawsuit on behalf of the United States and to share in any recovery, according to the U.S. Department of Justice.

So far, the federal government has declined to intervene in the case, but the suit can be advanced by private litigators, according to Andy Stone, cocounsel on the Emanuele suit. The government may intervene at a later point, said Mr. Stone, whose practice is in Pittsburgh. The government can seek triple the amount of damages determined by the courts. Mr. Stone said that it’s not clear how much might be at stake in the case before discovery, but that given the potential number of claims, it could be millions of dollars.

The Pennsylvania suit also accuses the cardiologists and the hospital of violating the antikickback statutes that prohibit improper patient referrals.

In addition, the suit alleges that the named physicians performed unnecessary medical procedures, including cardiac catheterizations and vascular surgeries.

Mr. Stone said that he believes that cases alleging overuse of stents or other percutaneous interventions may be brought more often.

"These are important cases because the dollar amounts are big. The incentives are there – and substantial patient risk. You put those two things together and that makes for a real serious abuse situation," he said.

Indeed, the Emanuele suit seems to be the latest in a high-profile battle over the appropriateness of cardiac procedures, particularly stent placement. Last spring, Greensburg, Pa.–based Excela Health said that it had found that stents had been unnecessarily placed in almost 200 patients. In a turnaround, that health system has just won accreditation under the Accreditation for Cardiovascular Excellence (ACE) program that is sponsored by the Society for Cardiovascular Angiography and Interventions and the American College of Cardiology Foundation.

Those two organizations, along with the Maryland Chapter of the ACC, have been working to head off regulation of stent procedures, according to an editorial in the Journal of the American College of Cardiology. Instead, they are hoping to convince the state to require all Maryland hospitals to submit to a mandatory accrediting process through the groups’ ACE program.

The state of Maryland began looking into stenting as the result of another qui tam suit filed by three cardiologists against St. Joseph Medical Center in Towson, Md. The hospital settled for $22 million. The suit alleged that St. Joseph paid kickbacks in return for referrals from MidAtlantic Cardiovascular Associates.

St. Joseph also settled charges that cardiologist Mark Midei performed unnecessary stent procedures.

In another example of the continuing scrutiny being given to cardiac procedures, a cardiologist has filed a whistleblower suit against former colleagues, alleging that they knowingly bilked federal health programs over a 4-year period, from 2001-2005.

The suit was filed by Dr. Tullio Emanuele in the U.S. District Court for the Western District of Pennsylvania. It was under seal until Oct. 2011, and was served on the defendants on Jan. 17. The defendants have 20 days to respond.

Dr. Emanuele is now based in Kentucky, but had worked in an Erie-based practice, Medicor Associates. In his suit, he claims that his former colleagues at Medicor and at Flagship Cardiac, Vascular, and Thoracic Surgery of Erie – along with the UPMC Hamot Medical Center of Erie – engaged in billing practices that defrauded Medicare, Medicaid, and Tricare, among other insurers.

Carly Manino, manager of media relations at UPMC Hamot, said that the hospital would not comment. "It’s our policy not to comment on pending litigation," she said.

Whistleblower or "qui tam" suits are filed under the False Claims Act. Such suits allow private citizens with knowledge of false claims against the government to bring a lawsuit on behalf of the United States and to share in any recovery, according to the U.S. Department of Justice.

So far, the federal government has declined to intervene in the case, but the suit can be advanced by private litigators, according to Andy Stone, cocounsel on the Emanuele suit. The government may intervene at a later point, said Mr. Stone, whose practice is in Pittsburgh. The government can seek triple the amount of damages determined by the courts. Mr. Stone said that it’s not clear how much might be at stake in the case before discovery, but that given the potential number of claims, it could be millions of dollars.

The Pennsylvania suit also accuses the cardiologists and the hospital of violating the antikickback statutes that prohibit improper patient referrals.

In addition, the suit alleges that the named physicians performed unnecessary medical procedures, including cardiac catheterizations and vascular surgeries.

Mr. Stone said that he believes that cases alleging overuse of stents or other percutaneous interventions may be brought more often.

"These are important cases because the dollar amounts are big. The incentives are there – and substantial patient risk. You put those two things together and that makes for a real serious abuse situation," he said.

Indeed, the Emanuele suit seems to be the latest in a high-profile battle over the appropriateness of cardiac procedures, particularly stent placement. Last spring, Greensburg, Pa.–based Excela Health said that it had found that stents had been unnecessarily placed in almost 200 patients. In a turnaround, that health system has just won accreditation under the Accreditation for Cardiovascular Excellence (ACE) program that is sponsored by the Society for Cardiovascular Angiography and Interventions and the American College of Cardiology Foundation.

Those two organizations, along with the Maryland Chapter of the ACC, have been working to head off regulation of stent procedures, according to an editorial in the Journal of the American College of Cardiology. Instead, they are hoping to convince the state to require all Maryland hospitals to submit to a mandatory accrediting process through the groups’ ACE program.

The state of Maryland began looking into stenting as the result of another qui tam suit filed by three cardiologists against St. Joseph Medical Center in Towson, Md. The hospital settled for $22 million. The suit alleged that St. Joseph paid kickbacks in return for referrals from MidAtlantic Cardiovascular Associates.

St. Joseph also settled charges that cardiologist Mark Midei performed unnecessary stent procedures.

The Food and Drug Administration has approved vismodegib, an oral, once-daily medication for adults with locally advanced and metastatic advanced basal cell carcinoma on Jan. 30.

Vismodegib (Erivedge) is the first medication approved for metastatic BCC and was approved ahead of its March 8 statutory review date, according to a statement from the FDA.

The drug works by inhibiting the hedgehog pathway, which is active in most BCCs. "Our understanding of molecular pathways involved in cancer, such as the hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, in a statement.

"Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions," Dr. Hal Barron, Genentech’s chief medical officer and head of global product development, said in a statement.

The FDA based its approval on a single-arm, multicenter phase II study, ERIVANCE BCC. The open-label study enrolled 104 patients with locally advanced or metastatic BCC but, according to the FDA, approval was based on evaluations of 96 patients. Study participants took 150 mg vismodegib once daily.

The primary end point was objective response rate, that is, the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. A total of 43% of patients with locally advanced BCC had partial or complete response (27 of 63) and 30% with metastatic disease had a partial response (10 of 33), according to Genentech. The median duration of response was 7.6 months.

The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste or loss of taste, decreased appetite, constipation, vomiting, and aching joints.

Vismodegib will carry a boxed warning about a potential risk of death or severe birth effects to a fetus. The FDA is not requiring a birth defects risk management program for vismodegib, but Genentech is advising female patients to use "highly effective" birth control before, during, and for 7 months after the last dose of treatment. Men should use a condom with spermicide, even if they have had a vasectomy, during sex with female partners during treatment and for 2 months after the last dose.

The company said that pregnant women are being encouraged to participate in the Erivedge pregnancy pharmacovigilance program, which collects information about exposure to that drug during pregnancy and the effects on the mother and her unborn child.

Vismodegib will be available in the United States within a few weeks and will be distributed through specialty pharmacies, according to Genentech.

The Food and Drug Administration has approved vismodegib, an oral, once-daily medication for adults with locally advanced and metastatic advanced basal cell carcinoma on Jan. 30.

Vismodegib (Erivedge) is the first medication approved for metastatic BCC and was approved ahead of its March 8 statutory review date, according to a statement from the FDA.

The drug works by inhibiting the hedgehog pathway, which is active in most BCCs. "Our understanding of molecular pathways involved in cancer, such as the hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, in a statement.

"Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions," Dr. Hal Barron, Genentech’s chief medical officer and head of global product development, said in a statement.

The FDA based its approval on a single-arm, multicenter phase II study, ERIVANCE BCC. The open-label study enrolled 104 patients with locally advanced or metastatic BCC but, according to the FDA, approval was based on evaluations of 96 patients. Study participants took 150 mg vismodegib once daily.

The primary end point was objective response rate, that is, the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. A total of 43% of patients with locally advanced BCC had partial or complete response (27 of 63) and 30% with metastatic disease had a partial response (10 of 33), according to Genentech. The median duration of response was 7.6 months.

The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste or loss of taste, decreased appetite, constipation, vomiting, and aching joints.

Vismodegib will carry a boxed warning about a potential risk of death or severe birth effects to a fetus. The FDA is not requiring a birth defects risk management program for vismodegib, but Genentech is advising female patients to use "highly effective" birth control before, during, and for 7 months after the last dose of treatment. Men should use a condom with spermicide, even if they have had a vasectomy, during sex with female partners during treatment and for 2 months after the last dose.

The company said that pregnant women are being encouraged to participate in the Erivedge pregnancy pharmacovigilance program, which collects information about exposure to that drug during pregnancy and the effects on the mother and her unborn child.

Vismodegib will be available in the United States within a few weeks and will be distributed through specialty pharmacies, according to Genentech.

The Food and Drug Administration has approved vismodegib, an oral, once-daily medication for adults with locally advanced and metastatic advanced basal cell carcinoma on Jan. 30.

Vismodegib (Erivedge) is the first medication approved for metastatic BCC and was approved ahead of its March 8 statutory review date, according to a statement from the FDA.

The drug works by inhibiting the hedgehog pathway, which is active in most BCCs. "Our understanding of molecular pathways involved in cancer, such as the hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Dr. Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products, in a statement.

"Today’s approval provides a new treatment for people with advanced basal cell carcinoma who, until now, had no approved medicines to help shrink disfiguring or potentially life-threatening lesions," Dr. Hal Barron, Genentech’s chief medical officer and head of global product development, said in a statement.

The FDA based its approval on a single-arm, multicenter phase II study, ERIVANCE BCC. The open-label study enrolled 104 patients with locally advanced or metastatic BCC but, according to the FDA, approval was based on evaluations of 96 patients. Study participants took 150 mg vismodegib once daily.

The primary end point was objective response rate, that is, the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. A total of 43% of patients with locally advanced BCC had partial or complete response (27 of 63) and 30% with metastatic disease had a partial response (10 of 33), according to Genentech. The median duration of response was 7.6 months.

The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste or loss of taste, decreased appetite, constipation, vomiting, and aching joints.

Vismodegib will carry a boxed warning about a potential risk of death or severe birth effects to a fetus. The FDA is not requiring a birth defects risk management program for vismodegib, but Genentech is advising female patients to use "highly effective" birth control before, during, and for 7 months after the last dose of treatment. Men should use a condom with spermicide, even if they have had a vasectomy, during sex with female partners during treatment and for 2 months after the last dose.

The company said that pregnant women are being encouraged to participate in the Erivedge pregnancy pharmacovigilance program, which collects information about exposure to that drug during pregnancy and the effects on the mother and her unborn child.

Vismodegib will be available in the United States within a few weeks and will be distributed through specialty pharmacies, according to Genentech.