Flipping the fetal hemoglobin switch reverses sickle cell symptoms

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SAN DIEGO – Researchers were able to “flip the switch” from the adult to fetal form of hemoglobin using autologous stem cells genetically modified to simultaneously induce the fetal form of hemoglobin and decrease sickle hemoglobin.

The advance was announced by investigators at the Dana-Farber Cancer Institute and Boston Children’s Hospital at the annual meeting of the American Society of Hematology. At 6 months of follow-up, one adult patient in the proof-of-concept study has experienced a reversal of the sickle cell phenotype, with no pain episodes or respiratory or neurologic events.

The fetal form of hemoglobin is known to be protective against the signs and symptoms of sickle cell disease, but apart from a few rare exceptions, people with the disorder begin to experience debilitating symptoms as levels of the fetal form begin to decline in early childhood and levels of the adult form of hemoglobin steadily rise.

In this video interview, Erica B. Esrick, MD, from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, describes the novel approach of using RNA interference to knock down a repressor that suppresses expression of gamma globin in sickle cell disease.

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SAN DIEGO – Researchers were able to “flip the switch” from the adult to fetal form of hemoglobin using autologous stem cells genetically modified to simultaneously induce the fetal form of hemoglobin and decrease sickle hemoglobin.

The advance was announced by investigators at the Dana-Farber Cancer Institute and Boston Children’s Hospital at the annual meeting of the American Society of Hematology. At 6 months of follow-up, one adult patient in the proof-of-concept study has experienced a reversal of the sickle cell phenotype, with no pain episodes or respiratory or neurologic events.

The fetal form of hemoglobin is known to be protective against the signs and symptoms of sickle cell disease, but apart from a few rare exceptions, people with the disorder begin to experience debilitating symptoms as levels of the fetal form begin to decline in early childhood and levels of the adult form of hemoglobin steadily rise.

In this video interview, Erica B. Esrick, MD, from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, describes the novel approach of using RNA interference to knock down a repressor that suppresses expression of gamma globin in sickle cell disease.

SAN DIEGO – Researchers were able to “flip the switch” from the adult to fetal form of hemoglobin using autologous stem cells genetically modified to simultaneously induce the fetal form of hemoglobin and decrease sickle hemoglobin.

The advance was announced by investigators at the Dana-Farber Cancer Institute and Boston Children’s Hospital at the annual meeting of the American Society of Hematology. At 6 months of follow-up, one adult patient in the proof-of-concept study has experienced a reversal of the sickle cell phenotype, with no pain episodes or respiratory or neurologic events.

The fetal form of hemoglobin is known to be protective against the signs and symptoms of sickle cell disease, but apart from a few rare exceptions, people with the disorder begin to experience debilitating symptoms as levels of the fetal form begin to decline in early childhood and levels of the adult form of hemoglobin steadily rise.

In this video interview, Erica B. Esrick, MD, from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, describes the novel approach of using RNA interference to knock down a repressor that suppresses expression of gamma globin in sickle cell disease.

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Child gun deaths lowest in states with strictest firearm laws

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More stringent gun laws are linked to reduced firearm-related pediatric injury and mortality, and laws restricting children’s access to firearms are linked to reduced pediatric firearm suicide rates, according to research.

Bytmonas/ThinkStock

“State-level legislation could play an important role in reducing pediatric firearm-related deaths,” concluded Jordan S. Taylor, MD, of Stanford (Calif.) University and his colleagues.

Dr. Taylor earned top honors among the American Academy of Pediatrics (AAP) Council on Injury, Violence and Poison Prevention research abstracts when he presented his findings at the annual meeting of the American Academy of Pediatrics.

Firearm injuries account for the second leading cause of death among U.S. children: 3,155 youth ages 19 years and younger died from gunshot injuries in 2016, and more than 17,000 were injured. Yet state laws governing the purchase, ownership, carriage, and storage of guns vary widely across the country. Dr. Taylor and his colleagues conducted two studies to assess the effects of firearm legislation on firearm-related injuries and deaths in U.S. children.

In their first study, they analyzed pediatric inpatient admissions for firearm injuries in 2012 relative to the stringency of state firearm legislation. They relied on five data sources for the analysis: the Kids’ Inpatient Database (KID), the Healthcare Cost and Utilization Project, the Agency for Healthcare Research and Quality, the U.S. Census Bureau, and the 2013 Brady scorecard.

The Brady scorecard provides scores for each state based on the presence and strictness of firearm-related laws, including legislation on background checks, ability of dangerous individuals to purchase guns, trafficking laws, and laws governing the sales, carrying, and purchasing of firearms.


The 10 states with the strictest laws (highest Brady scores) are California, Connecticut, Delaware, Hawaii, Illinois, Maryland, Massachusetts, New Jersey, New York, and Rhode Island. The 10 states with the lowest scores (least-strict legislation) are Alaska, Arizona, Arkansas, Florida, Kentucky, Louisiana, Montana, Nevada, Virginia, and Wyoming.

Among the 6,941 youth (aged 0-20 years) hospitalized in 2012 for firearm injuries, 7% died. More than a third of these (36%) occurred in the South, 25% in the Midwest, 22% in the West, and 17% in the Northeast.

Children most likely to be injured were boys, older children, black and Latino children, and children living in low-income zip codes.

The Midwest and South, which have lower average Brady scores (more lax legislation on guns), had 8.30 injuries per 100,000 children, compared with 7.54 injuries per 100,000 children in the Northeast and West, which have higher average Brady scores (more stringent gun laws). This was a difference of 0.76 injuries per 100,000 children (95% confidence interval, 0.38-1.13; P less than 0.001).

Then the researchers conducted a second analysis that looked specifically at firearm mortality within the context of both child access prevention (CAP) laws and states’ Brady scores. CAP laws include safe storage laws and gun lock laws, for example.

This analysis used the Web-Based Injury Statistics Query and Reporting System to capture pediatric firearm deaths from 2014-2015 and compared these to the 2014 Brady scores and CAP laws.

An estimated 2,715 child gun deaths occurred during the study period, of which 62% were homicides and 31% were suicides. The researchers identified “a significant negative correlation between states’ firearm legislation stringency and pediatric firearm mortality (Spearman correlation coefficient = –0.66) and between presence of CAP laws and firearm suicide rates (Spearman correlation coefficient = –0.56).”

Dr. Taylor said in an interview, “states that have both types of child access prevention laws [had] suicide rates four times lower than states that did not have either of those.”

Positive correlations also showed up between unemployment rate and firearm homicide rate (Spearman correlation coefficient = 0.55) and teen tobacco use and firearm suicide rate (Spearman correlation coefficient = 0.50).

The association between Brady scores and pediatric mortality from firearms remained significant after adjustment for poverty, unemployment, and substance abuse (P less than .01). Similarly, the association between the pediatric firearm suicide rate and CAP laws remained significant after controlling for socioeconomic factors and other firearm legislation (P less than .01).

In a video interview, Dr. Taylor discussed his research findings and their importance in clinical practice.

“It’s absolutely important for pediatricians to talk to families about firearms in their home and also in the homes of their friends that they visit,” Dr. Taylor said. “We try to approach it as a public health issue similar to seat belts and car seats.”

No external funding was used, and Dr. Taylor reported no conflicts of interest.

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More stringent gun laws are linked to reduced firearm-related pediatric injury and mortality, and laws restricting children’s access to firearms are linked to reduced pediatric firearm suicide rates, according to research.

Bytmonas/ThinkStock

“State-level legislation could play an important role in reducing pediatric firearm-related deaths,” concluded Jordan S. Taylor, MD, of Stanford (Calif.) University and his colleagues.

Dr. Taylor earned top honors among the American Academy of Pediatrics (AAP) Council on Injury, Violence and Poison Prevention research abstracts when he presented his findings at the annual meeting of the American Academy of Pediatrics.

Firearm injuries account for the second leading cause of death among U.S. children: 3,155 youth ages 19 years and younger died from gunshot injuries in 2016, and more than 17,000 were injured. Yet state laws governing the purchase, ownership, carriage, and storage of guns vary widely across the country. Dr. Taylor and his colleagues conducted two studies to assess the effects of firearm legislation on firearm-related injuries and deaths in U.S. children.

In their first study, they analyzed pediatric inpatient admissions for firearm injuries in 2012 relative to the stringency of state firearm legislation. They relied on five data sources for the analysis: the Kids’ Inpatient Database (KID), the Healthcare Cost and Utilization Project, the Agency for Healthcare Research and Quality, the U.S. Census Bureau, and the 2013 Brady scorecard.

The Brady scorecard provides scores for each state based on the presence and strictness of firearm-related laws, including legislation on background checks, ability of dangerous individuals to purchase guns, trafficking laws, and laws governing the sales, carrying, and purchasing of firearms.


The 10 states with the strictest laws (highest Brady scores) are California, Connecticut, Delaware, Hawaii, Illinois, Maryland, Massachusetts, New Jersey, New York, and Rhode Island. The 10 states with the lowest scores (least-strict legislation) are Alaska, Arizona, Arkansas, Florida, Kentucky, Louisiana, Montana, Nevada, Virginia, and Wyoming.

Among the 6,941 youth (aged 0-20 years) hospitalized in 2012 for firearm injuries, 7% died. More than a third of these (36%) occurred in the South, 25% in the Midwest, 22% in the West, and 17% in the Northeast.

Children most likely to be injured were boys, older children, black and Latino children, and children living in low-income zip codes.

The Midwest and South, which have lower average Brady scores (more lax legislation on guns), had 8.30 injuries per 100,000 children, compared with 7.54 injuries per 100,000 children in the Northeast and West, which have higher average Brady scores (more stringent gun laws). This was a difference of 0.76 injuries per 100,000 children (95% confidence interval, 0.38-1.13; P less than 0.001).

Then the researchers conducted a second analysis that looked specifically at firearm mortality within the context of both child access prevention (CAP) laws and states’ Brady scores. CAP laws include safe storage laws and gun lock laws, for example.

This analysis used the Web-Based Injury Statistics Query and Reporting System to capture pediatric firearm deaths from 2014-2015 and compared these to the 2014 Brady scores and CAP laws.

An estimated 2,715 child gun deaths occurred during the study period, of which 62% were homicides and 31% were suicides. The researchers identified “a significant negative correlation between states’ firearm legislation stringency and pediatric firearm mortality (Spearman correlation coefficient = –0.66) and between presence of CAP laws and firearm suicide rates (Spearman correlation coefficient = –0.56).”

Dr. Taylor said in an interview, “states that have both types of child access prevention laws [had] suicide rates four times lower than states that did not have either of those.”

Positive correlations also showed up between unemployment rate and firearm homicide rate (Spearman correlation coefficient = 0.55) and teen tobacco use and firearm suicide rate (Spearman correlation coefficient = 0.50).

The association between Brady scores and pediatric mortality from firearms remained significant after adjustment for poverty, unemployment, and substance abuse (P less than .01). Similarly, the association between the pediatric firearm suicide rate and CAP laws remained significant after controlling for socioeconomic factors and other firearm legislation (P less than .01).

In a video interview, Dr. Taylor discussed his research findings and their importance in clinical practice.

“It’s absolutely important for pediatricians to talk to families about firearms in their home and also in the homes of their friends that they visit,” Dr. Taylor said. “We try to approach it as a public health issue similar to seat belts and car seats.”

No external funding was used, and Dr. Taylor reported no conflicts of interest.

 

More stringent gun laws are linked to reduced firearm-related pediatric injury and mortality, and laws restricting children’s access to firearms are linked to reduced pediatric firearm suicide rates, according to research.

Bytmonas/ThinkStock

“State-level legislation could play an important role in reducing pediatric firearm-related deaths,” concluded Jordan S. Taylor, MD, of Stanford (Calif.) University and his colleagues.

Dr. Taylor earned top honors among the American Academy of Pediatrics (AAP) Council on Injury, Violence and Poison Prevention research abstracts when he presented his findings at the annual meeting of the American Academy of Pediatrics.

Firearm injuries account for the second leading cause of death among U.S. children: 3,155 youth ages 19 years and younger died from gunshot injuries in 2016, and more than 17,000 were injured. Yet state laws governing the purchase, ownership, carriage, and storage of guns vary widely across the country. Dr. Taylor and his colleagues conducted two studies to assess the effects of firearm legislation on firearm-related injuries and deaths in U.S. children.

In their first study, they analyzed pediatric inpatient admissions for firearm injuries in 2012 relative to the stringency of state firearm legislation. They relied on five data sources for the analysis: the Kids’ Inpatient Database (KID), the Healthcare Cost and Utilization Project, the Agency for Healthcare Research and Quality, the U.S. Census Bureau, and the 2013 Brady scorecard.

The Brady scorecard provides scores for each state based on the presence and strictness of firearm-related laws, including legislation on background checks, ability of dangerous individuals to purchase guns, trafficking laws, and laws governing the sales, carrying, and purchasing of firearms.


The 10 states with the strictest laws (highest Brady scores) are California, Connecticut, Delaware, Hawaii, Illinois, Maryland, Massachusetts, New Jersey, New York, and Rhode Island. The 10 states with the lowest scores (least-strict legislation) are Alaska, Arizona, Arkansas, Florida, Kentucky, Louisiana, Montana, Nevada, Virginia, and Wyoming.

Among the 6,941 youth (aged 0-20 years) hospitalized in 2012 for firearm injuries, 7% died. More than a third of these (36%) occurred in the South, 25% in the Midwest, 22% in the West, and 17% in the Northeast.

Children most likely to be injured were boys, older children, black and Latino children, and children living in low-income zip codes.

The Midwest and South, which have lower average Brady scores (more lax legislation on guns), had 8.30 injuries per 100,000 children, compared with 7.54 injuries per 100,000 children in the Northeast and West, which have higher average Brady scores (more stringent gun laws). This was a difference of 0.76 injuries per 100,000 children (95% confidence interval, 0.38-1.13; P less than 0.001).

Then the researchers conducted a second analysis that looked specifically at firearm mortality within the context of both child access prevention (CAP) laws and states’ Brady scores. CAP laws include safe storage laws and gun lock laws, for example.

This analysis used the Web-Based Injury Statistics Query and Reporting System to capture pediatric firearm deaths from 2014-2015 and compared these to the 2014 Brady scores and CAP laws.

An estimated 2,715 child gun deaths occurred during the study period, of which 62% were homicides and 31% were suicides. The researchers identified “a significant negative correlation between states’ firearm legislation stringency and pediatric firearm mortality (Spearman correlation coefficient = –0.66) and between presence of CAP laws and firearm suicide rates (Spearman correlation coefficient = –0.56).”

Dr. Taylor said in an interview, “states that have both types of child access prevention laws [had] suicide rates four times lower than states that did not have either of those.”

Positive correlations also showed up between unemployment rate and firearm homicide rate (Spearman correlation coefficient = 0.55) and teen tobacco use and firearm suicide rate (Spearman correlation coefficient = 0.50).

The association between Brady scores and pediatric mortality from firearms remained significant after adjustment for poverty, unemployment, and substance abuse (P less than .01). Similarly, the association between the pediatric firearm suicide rate and CAP laws remained significant after controlling for socioeconomic factors and other firearm legislation (P less than .01).

In a video interview, Dr. Taylor discussed his research findings and their importance in clinical practice.

“It’s absolutely important for pediatricians to talk to families about firearms in their home and also in the homes of their friends that they visit,” Dr. Taylor said. “We try to approach it as a public health issue similar to seat belts and car seats.”

No external funding was used, and Dr. Taylor reported no conflicts of interest.

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Key clinical point: Stricter state firearm legislation was associated with reduced firearm-related pediatric mortality.

Major finding: 8.3 injuries per 100,000 children occurred in the Midwest and South, compared with 7.5 injuries per 100,000 children in the Northeast and West.

Study details: The findings are based on two separate analyses that analyzed state Brady scores along with 6,941 firearm-related hospitalizations in 2012 and 2,715 pediatric deaths from firearms in 2014-2015.

Disclosures: No external funding was used, and Dr. Taylor reported no conflicts of interest.

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Sofa and bed injuries very common among young children

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– Injuries related to beds and sofas in children aged under 5 years occur more than twice as frequently than injuries related to stairs, according to new research.

“Findings from our analysis reveal that it is an important source of injury to young children and a leading cause of trauma to infants,” concluded David S. Liu, of Baylor College of Medicine, Houston, who presented the findings at the annual meeting of the American Academy of Pediatrics.

“The rate of bed- and sofa-related injuries is increasing, which underscores the need for increased prevention efforts, including parental education and improved safety design, to decrease soft furniture injuries among young children,” Mr. Liu and his colleagues wrote.

The researchers used the National Electronic Injury Surveillance System of the U.S. Consumer Product Safety Commission to conduct a retrospective analysis of injuries related to sofas and beds from 2007 to 2016.

They found that an estimated 2.3 million children aged under 5 years were treated for injuries related to soft furniture during those years, an average of 230,026 injuries a year, or 115 injuries per 10,000 children. To the surprise of the researchers, injuries related to beds and sofas were the most common types of accidental injury in that age group, occurring 2.5 times more often than stair-related injuries, which occurred at a rate of 47 per 10,000 population.

Boys were slightly more likely to be injured, making up 56% of all the cases. Soft tissue/internal organ injuries were most common, comprising 28% of all injuries, followed by lacerations in 24% of cases, abrasions in 15%, and fractures in 14%.

More than half the children (61%) sustained injuries to the head or face, and 3% were hospitalized for their injuries. Although infants (under 1 year old) only accounted for 28% of children injured, they were twice as likely to be hospitalized than older children.

The researchers also identified increases in injuries over the time period studied. Bed-related injuries increased 17% from 2007 to 2016, and sofa/couch-related injuries increased 17% during that period.

Although the vast majority of children were treated and released, approximately 4% of children were admitted or treated and transferred to another facility. Overall, an estimated 3,361 children died during the 9-year period, translating to a little over 370 children a year.

In a video interview, Mr. Liu discussed the implications of these findings.

“We know how dangerous car accidents and staircases are, and we often recommend car seats and stair gates for those,” Mr. Liu said. “Obviously we can’t put a gate or a barrier on every single sofa, couch, and bed in America, so as clinicians and parents, the best we can do is keep aware of how dangerous these items are. Just because of their soft nature doesn’t mean they’re inherently safer.”

The researchers reported no disclosures and the research received no external funding.

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– Injuries related to beds and sofas in children aged under 5 years occur more than twice as frequently than injuries related to stairs, according to new research.

“Findings from our analysis reveal that it is an important source of injury to young children and a leading cause of trauma to infants,” concluded David S. Liu, of Baylor College of Medicine, Houston, who presented the findings at the annual meeting of the American Academy of Pediatrics.

“The rate of bed- and sofa-related injuries is increasing, which underscores the need for increased prevention efforts, including parental education and improved safety design, to decrease soft furniture injuries among young children,” Mr. Liu and his colleagues wrote.

The researchers used the National Electronic Injury Surveillance System of the U.S. Consumer Product Safety Commission to conduct a retrospective analysis of injuries related to sofas and beds from 2007 to 2016.

They found that an estimated 2.3 million children aged under 5 years were treated for injuries related to soft furniture during those years, an average of 230,026 injuries a year, or 115 injuries per 10,000 children. To the surprise of the researchers, injuries related to beds and sofas were the most common types of accidental injury in that age group, occurring 2.5 times more often than stair-related injuries, which occurred at a rate of 47 per 10,000 population.

Boys were slightly more likely to be injured, making up 56% of all the cases. Soft tissue/internal organ injuries were most common, comprising 28% of all injuries, followed by lacerations in 24% of cases, abrasions in 15%, and fractures in 14%.

More than half the children (61%) sustained injuries to the head or face, and 3% were hospitalized for their injuries. Although infants (under 1 year old) only accounted for 28% of children injured, they were twice as likely to be hospitalized than older children.

The researchers also identified increases in injuries over the time period studied. Bed-related injuries increased 17% from 2007 to 2016, and sofa/couch-related injuries increased 17% during that period.

Although the vast majority of children were treated and released, approximately 4% of children were admitted or treated and transferred to another facility. Overall, an estimated 3,361 children died during the 9-year period, translating to a little over 370 children a year.

In a video interview, Mr. Liu discussed the implications of these findings.

“We know how dangerous car accidents and staircases are, and we often recommend car seats and stair gates for those,” Mr. Liu said. “Obviously we can’t put a gate or a barrier on every single sofa, couch, and bed in America, so as clinicians and parents, the best we can do is keep aware of how dangerous these items are. Just because of their soft nature doesn’t mean they’re inherently safer.”

The researchers reported no disclosures and the research received no external funding.

– Injuries related to beds and sofas in children aged under 5 years occur more than twice as frequently than injuries related to stairs, according to new research.

“Findings from our analysis reveal that it is an important source of injury to young children and a leading cause of trauma to infants,” concluded David S. Liu, of Baylor College of Medicine, Houston, who presented the findings at the annual meeting of the American Academy of Pediatrics.

“The rate of bed- and sofa-related injuries is increasing, which underscores the need for increased prevention efforts, including parental education and improved safety design, to decrease soft furniture injuries among young children,” Mr. Liu and his colleagues wrote.

The researchers used the National Electronic Injury Surveillance System of the U.S. Consumer Product Safety Commission to conduct a retrospective analysis of injuries related to sofas and beds from 2007 to 2016.

They found that an estimated 2.3 million children aged under 5 years were treated for injuries related to soft furniture during those years, an average of 230,026 injuries a year, or 115 injuries per 10,000 children. To the surprise of the researchers, injuries related to beds and sofas were the most common types of accidental injury in that age group, occurring 2.5 times more often than stair-related injuries, which occurred at a rate of 47 per 10,000 population.

Boys were slightly more likely to be injured, making up 56% of all the cases. Soft tissue/internal organ injuries were most common, comprising 28% of all injuries, followed by lacerations in 24% of cases, abrasions in 15%, and fractures in 14%.

More than half the children (61%) sustained injuries to the head or face, and 3% were hospitalized for their injuries. Although infants (under 1 year old) only accounted for 28% of children injured, they were twice as likely to be hospitalized than older children.

The researchers also identified increases in injuries over the time period studied. Bed-related injuries increased 17% from 2007 to 2016, and sofa/couch-related injuries increased 17% during that period.

Although the vast majority of children were treated and released, approximately 4% of children were admitted or treated and transferred to another facility. Overall, an estimated 3,361 children died during the 9-year period, translating to a little over 370 children a year.

In a video interview, Mr. Liu discussed the implications of these findings.

“We know how dangerous car accidents and staircases are, and we often recommend car seats and stair gates for those,” Mr. Liu said. “Obviously we can’t put a gate or a barrier on every single sofa, couch, and bed in America, so as clinicians and parents, the best we can do is keep aware of how dangerous these items are. Just because of their soft nature doesn’t mean they’re inherently safer.”

The researchers reported no disclosures and the research received no external funding.

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Key clinical point: Injuries from beds and sofas/couches are common in children aged under 5 years, occurring 2.5 times more frequently than stairs-related injuries.

Major finding: An estimated 115 bed/sofa-related injuries per 10,000 children occur every year.

Study details: The findings are based on a retrospective analysis of injuries related to sofas and beds from 2007 to 2016.

Disclosures: The researchers reported no disclosures and the research received no external funding.

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AAP speaker emphasizes importance of understanding patients’ ‘lived experience’

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– Pediatricians who learn about their patients’ lived experience have the potential to encourage patients and help them overcome biases, assumptions, and barriers of opioid use disorder, Tamela Milan said at the American Academy of Pediatrics annual meeting.

After her five children were taken into state welfare custody and she began a sixth pregnancy while struggling with opioid use disorder and as a survivor of domestic violence, Ms. Milan’s pediatrician was the one to encourage her to take steps to improve her life. She went on to regain custody of her children and complete college, and has given back by working in community health programs for over 20 years.

In a video interview, Ms. Milan said she would not have been able to overcome these barriers had it not been for the support of her pediatrician, who saw her as a person instead of a mother with opioid use disorder.

“I’ve been on both sides of the fence,” Ms. Milan said. “As someone who’s had to receive treatment and to provide it, it’s really important that we start looking at people for who they are and where they are.”

Tamela Milan has no relevant conflicts of interest.

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– Pediatricians who learn about their patients’ lived experience have the potential to encourage patients and help them overcome biases, assumptions, and barriers of opioid use disorder, Tamela Milan said at the American Academy of Pediatrics annual meeting.

After her five children were taken into state welfare custody and she began a sixth pregnancy while struggling with opioid use disorder and as a survivor of domestic violence, Ms. Milan’s pediatrician was the one to encourage her to take steps to improve her life. She went on to regain custody of her children and complete college, and has given back by working in community health programs for over 20 years.

In a video interview, Ms. Milan said she would not have been able to overcome these barriers had it not been for the support of her pediatrician, who saw her as a person instead of a mother with opioid use disorder.

“I’ve been on both sides of the fence,” Ms. Milan said. “As someone who’s had to receive treatment and to provide it, it’s really important that we start looking at people for who they are and where they are.”

Tamela Milan has no relevant conflicts of interest.

– Pediatricians who learn about their patients’ lived experience have the potential to encourage patients and help them overcome biases, assumptions, and barriers of opioid use disorder, Tamela Milan said at the American Academy of Pediatrics annual meeting.

After her five children were taken into state welfare custody and she began a sixth pregnancy while struggling with opioid use disorder and as a survivor of domestic violence, Ms. Milan’s pediatrician was the one to encourage her to take steps to improve her life. She went on to regain custody of her children and complete college, and has given back by working in community health programs for over 20 years.

In a video interview, Ms. Milan said she would not have been able to overcome these barriers had it not been for the support of her pediatrician, who saw her as a person instead of a mother with opioid use disorder.

“I’ve been on both sides of the fence,” Ms. Milan said. “As someone who’s had to receive treatment and to provide it, it’s really important that we start looking at people for who they are and where they are.”

Tamela Milan has no relevant conflicts of interest.

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Staying up to date on screening may cut risk of death from CRC

Now do the study with colonoscopy
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Wed, 05/26/2021 - 13:48

Patients who died from colorectal cancer were significantly more likely than controls not to have been screened, to have missed screenings, or not to have followed up on an abnormal result, according to the results of a large retrospective case-control study.

Source: American Gastroenterological Association

The findings signify “potentially modifiable” screening failures in a population known for relatively high uptake of colorectal cancer screening, wrote Chyke A. Doubeni, MD, MPH, of the University of Pennsylvania, Philadelphia, and his associates in Gastroenterology. Strikingly, 76% of patients who died from colorectal cancer were not current on screening versus 55% of cancer-free patients, they said. Being up to date on screening decreased the odds of dying from colorectal cancer by 62% (odds ratio, 0.38; 95% confidence interval, 0.33-0.44), even after adjustment for race, ethnicity, socioeconomic status, comorbidities, and frequency of contact with primary care providers, they added.

Colonoscopy, sigmoidoscopy, and fecal testing are effective and recommended screening techniques that help prevent deaths from colorectal cancer. Therefore, most such deaths are thought to result from “breakdowns in the screening process,” the researchers wrote. However, interval cancers and missed lesions also play a role, and no prior study has examined detailed screening histories and their association with colorectal cancer mortality.

Accordingly, the researchers reviewed medical records and registry data for 1,750 enrollees in the Kaiser Permanente Northern and Southern California systems who died from colorectal cancer during 2002-2012 and were part of the health plan for at least 5 years before their cancer diagnosis. They compared these patients with 3,486 cancer-free controls matched by age, sex, study site, and numbers of years enrolled in the health plan. Patients were considered up to date on screening if they were screened at intervals recommended by the 2008 multisociety colorectal cancer screening guidelines – that is, if they had received a colonoscopy within 10 years of colorectal cancer diagnosis or sigmoidoscopy or barium enema within 5 years of it. For fecal testing, the investigators used a 2-year interval based on its efficacy in clinical trials.

Among patients who died from colorectal cancer, only 24% were up to date on screening versus 45% of cancer-free-patients, the investigators determined. Furthermore, 68% of patients who died from colorectal cancer were never screened or were not screened at appropriate intervals, compared with 53% of cancer-free patients.

Additionally, while 8% of colorectal cancer deaths occurred in patients who had not followed up on abnormal screening results, only 2% of controls who had received abnormal screening results had failed to follow up.

“In two health systems with high rates of screening, we observed that most patients dying from colorectal cancer had potentially modifiable failures of the screening process,” the researchers concluded. “This study suggests that, even in settings with high screening uptake, access to and timely uptake of screening, regular rescreening, appropriate use of testing given patient characteristics, completion of timely diagnostic testing when screening is positive, and improving the effectiveness of screening tests, particularly for right colon cancer, remain important areas of focus for further decreasing colorectal cancer deaths.”

The National Institutes of Health funded the work. The investigators reported having no conflicts of interest except that one coinvestigator is editor in chief of the journal Gastroenterology.

SOURCE: Doubeni CA et al. Gastroenterology. 2018 Sep 27. doi: 10.1053/j.gastro.2018.09.040.

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Screening for colorectal cancer (CRC) is a major success story – one of only two cancers (the other being cervical cancer) with an A recommendation for screening from the U.S. Preventive Services Task Force. Multiple randomized trials for two CRC screening modalities, stool-based tests and sigmoidoscopy, have shown significant reductions in CRC incidence and mortality.

Dr. Paul Pinsky
Additionally, U.S. CRC incidence and mortality rates have been steadily decreasing for the past several decades, with much of that decrease attributed to screening.

Within this context, Doubeni et al. examined the association of CRC screening with death from CRC in a real-world HMO setting. Their study is notable for several reasons. First, it showed a highly protective effect on CRC mortality of being up to date with screening (odds ratio, 0.38; 95% confidence interval, 0.33-0.44). Second, it examined CRC screening as a process, with various steps of that process related to CRC mortality. Finally, methodologically, the study’s utilization of electronic medical records and cancer registry linkages highlights the importance of integrated data systems in the efficient performance of epidemiologic research.

Of note, screening was primarily stool-based tests (fecal occult blood test/fecal immunochemical test ) and sigmoidoscopy, in contrast to most of the U.S., where colonoscopy is predominant. Randomized trials of these modalities show mortality reductions of 15%-20% (FOBT/FIT) and 25%-30% (sigmoidoscopy), respectively. Therefore, some of the reported effect is likely due to selection bias, with healthier persons more likely to choose screening. 

It would be of interest to see similar studies performed in a colonoscopy-predominant screening setting and with the effect on CRC incidence as well as mortality examined.

Paul F. Pinsky, PhD, chief of the Early Detection Research Branch, National Cancer Institute, Bethesda, MD. He has no conflicts of interest.
 

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Screening for colorectal cancer (CRC) is a major success story – one of only two cancers (the other being cervical cancer) with an A recommendation for screening from the U.S. Preventive Services Task Force. Multiple randomized trials for two CRC screening modalities, stool-based tests and sigmoidoscopy, have shown significant reductions in CRC incidence and mortality.

Dr. Paul Pinsky
Additionally, U.S. CRC incidence and mortality rates have been steadily decreasing for the past several decades, with much of that decrease attributed to screening.

Within this context, Doubeni et al. examined the association of CRC screening with death from CRC in a real-world HMO setting. Their study is notable for several reasons. First, it showed a highly protective effect on CRC mortality of being up to date with screening (odds ratio, 0.38; 95% confidence interval, 0.33-0.44). Second, it examined CRC screening as a process, with various steps of that process related to CRC mortality. Finally, methodologically, the study’s utilization of electronic medical records and cancer registry linkages highlights the importance of integrated data systems in the efficient performance of epidemiologic research.

Of note, screening was primarily stool-based tests (fecal occult blood test/fecal immunochemical test ) and sigmoidoscopy, in contrast to most of the U.S., where colonoscopy is predominant. Randomized trials of these modalities show mortality reductions of 15%-20% (FOBT/FIT) and 25%-30% (sigmoidoscopy), respectively. Therefore, some of the reported effect is likely due to selection bias, with healthier persons more likely to choose screening. 

It would be of interest to see similar studies performed in a colonoscopy-predominant screening setting and with the effect on CRC incidence as well as mortality examined.

Paul F. Pinsky, PhD, chief of the Early Detection Research Branch, National Cancer Institute, Bethesda, MD. He has no conflicts of interest.
 

Body

Screening for colorectal cancer (CRC) is a major success story – one of only two cancers (the other being cervical cancer) with an A recommendation for screening from the U.S. Preventive Services Task Force. Multiple randomized trials for two CRC screening modalities, stool-based tests and sigmoidoscopy, have shown significant reductions in CRC incidence and mortality.

Dr. Paul Pinsky
Additionally, U.S. CRC incidence and mortality rates have been steadily decreasing for the past several decades, with much of that decrease attributed to screening.

Within this context, Doubeni et al. examined the association of CRC screening with death from CRC in a real-world HMO setting. Their study is notable for several reasons. First, it showed a highly protective effect on CRC mortality of being up to date with screening (odds ratio, 0.38; 95% confidence interval, 0.33-0.44). Second, it examined CRC screening as a process, with various steps of that process related to CRC mortality. Finally, methodologically, the study’s utilization of electronic medical records and cancer registry linkages highlights the importance of integrated data systems in the efficient performance of epidemiologic research.

Of note, screening was primarily stool-based tests (fecal occult blood test/fecal immunochemical test ) and sigmoidoscopy, in contrast to most of the U.S., where colonoscopy is predominant. Randomized trials of these modalities show mortality reductions of 15%-20% (FOBT/FIT) and 25%-30% (sigmoidoscopy), respectively. Therefore, some of the reported effect is likely due to selection bias, with healthier persons more likely to choose screening. 

It would be of interest to see similar studies performed in a colonoscopy-predominant screening setting and with the effect on CRC incidence as well as mortality examined.

Paul F. Pinsky, PhD, chief of the Early Detection Research Branch, National Cancer Institute, Bethesda, MD. He has no conflicts of interest.
 

Title
Now do the study with colonoscopy
Now do the study with colonoscopy

Patients who died from colorectal cancer were significantly more likely than controls not to have been screened, to have missed screenings, or not to have followed up on an abnormal result, according to the results of a large retrospective case-control study.

Source: American Gastroenterological Association

The findings signify “potentially modifiable” screening failures in a population known for relatively high uptake of colorectal cancer screening, wrote Chyke A. Doubeni, MD, MPH, of the University of Pennsylvania, Philadelphia, and his associates in Gastroenterology. Strikingly, 76% of patients who died from colorectal cancer were not current on screening versus 55% of cancer-free patients, they said. Being up to date on screening decreased the odds of dying from colorectal cancer by 62% (odds ratio, 0.38; 95% confidence interval, 0.33-0.44), even after adjustment for race, ethnicity, socioeconomic status, comorbidities, and frequency of contact with primary care providers, they added.

Colonoscopy, sigmoidoscopy, and fecal testing are effective and recommended screening techniques that help prevent deaths from colorectal cancer. Therefore, most such deaths are thought to result from “breakdowns in the screening process,” the researchers wrote. However, interval cancers and missed lesions also play a role, and no prior study has examined detailed screening histories and their association with colorectal cancer mortality.

Accordingly, the researchers reviewed medical records and registry data for 1,750 enrollees in the Kaiser Permanente Northern and Southern California systems who died from colorectal cancer during 2002-2012 and were part of the health plan for at least 5 years before their cancer diagnosis. They compared these patients with 3,486 cancer-free controls matched by age, sex, study site, and numbers of years enrolled in the health plan. Patients were considered up to date on screening if they were screened at intervals recommended by the 2008 multisociety colorectal cancer screening guidelines – that is, if they had received a colonoscopy within 10 years of colorectal cancer diagnosis or sigmoidoscopy or barium enema within 5 years of it. For fecal testing, the investigators used a 2-year interval based on its efficacy in clinical trials.

Among patients who died from colorectal cancer, only 24% were up to date on screening versus 45% of cancer-free-patients, the investigators determined. Furthermore, 68% of patients who died from colorectal cancer were never screened or were not screened at appropriate intervals, compared with 53% of cancer-free patients.

Additionally, while 8% of colorectal cancer deaths occurred in patients who had not followed up on abnormal screening results, only 2% of controls who had received abnormal screening results had failed to follow up.

“In two health systems with high rates of screening, we observed that most patients dying from colorectal cancer had potentially modifiable failures of the screening process,” the researchers concluded. “This study suggests that, even in settings with high screening uptake, access to and timely uptake of screening, regular rescreening, appropriate use of testing given patient characteristics, completion of timely diagnostic testing when screening is positive, and improving the effectiveness of screening tests, particularly for right colon cancer, remain important areas of focus for further decreasing colorectal cancer deaths.”

The National Institutes of Health funded the work. The investigators reported having no conflicts of interest except that one coinvestigator is editor in chief of the journal Gastroenterology.

SOURCE: Doubeni CA et al. Gastroenterology. 2018 Sep 27. doi: 10.1053/j.gastro.2018.09.040.

Patients who died from colorectal cancer were significantly more likely than controls not to have been screened, to have missed screenings, or not to have followed up on an abnormal result, according to the results of a large retrospective case-control study.

Source: American Gastroenterological Association

The findings signify “potentially modifiable” screening failures in a population known for relatively high uptake of colorectal cancer screening, wrote Chyke A. Doubeni, MD, MPH, of the University of Pennsylvania, Philadelphia, and his associates in Gastroenterology. Strikingly, 76% of patients who died from colorectal cancer were not current on screening versus 55% of cancer-free patients, they said. Being up to date on screening decreased the odds of dying from colorectal cancer by 62% (odds ratio, 0.38; 95% confidence interval, 0.33-0.44), even after adjustment for race, ethnicity, socioeconomic status, comorbidities, and frequency of contact with primary care providers, they added.

Colonoscopy, sigmoidoscopy, and fecal testing are effective and recommended screening techniques that help prevent deaths from colorectal cancer. Therefore, most such deaths are thought to result from “breakdowns in the screening process,” the researchers wrote. However, interval cancers and missed lesions also play a role, and no prior study has examined detailed screening histories and their association with colorectal cancer mortality.

Accordingly, the researchers reviewed medical records and registry data for 1,750 enrollees in the Kaiser Permanente Northern and Southern California systems who died from colorectal cancer during 2002-2012 and were part of the health plan for at least 5 years before their cancer diagnosis. They compared these patients with 3,486 cancer-free controls matched by age, sex, study site, and numbers of years enrolled in the health plan. Patients were considered up to date on screening if they were screened at intervals recommended by the 2008 multisociety colorectal cancer screening guidelines – that is, if they had received a colonoscopy within 10 years of colorectal cancer diagnosis or sigmoidoscopy or barium enema within 5 years of it. For fecal testing, the investigators used a 2-year interval based on its efficacy in clinical trials.

Among patients who died from colorectal cancer, only 24% were up to date on screening versus 45% of cancer-free-patients, the investigators determined. Furthermore, 68% of patients who died from colorectal cancer were never screened or were not screened at appropriate intervals, compared with 53% of cancer-free patients.

Additionally, while 8% of colorectal cancer deaths occurred in patients who had not followed up on abnormal screening results, only 2% of controls who had received abnormal screening results had failed to follow up.

“In two health systems with high rates of screening, we observed that most patients dying from colorectal cancer had potentially modifiable failures of the screening process,” the researchers concluded. “This study suggests that, even in settings with high screening uptake, access to and timely uptake of screening, regular rescreening, appropriate use of testing given patient characteristics, completion of timely diagnostic testing when screening is positive, and improving the effectiveness of screening tests, particularly for right colon cancer, remain important areas of focus for further decreasing colorectal cancer deaths.”

The National Institutes of Health funded the work. The investigators reported having no conflicts of interest except that one coinvestigator is editor in chief of the journal Gastroenterology.

SOURCE: Doubeni CA et al. Gastroenterology. 2018 Sep 27. doi: 10.1053/j.gastro.2018.09.040.

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Key clinical point: Being up to date on screening was associated with a significant reduction in the risk of dying from colon cancer.

Major finding: Being up to date on screening decreased the odds of dying from colorectal cancer by 62% (odds ratio, 0.38; 95% confidence interval, 0.33-0.44).

Study details: Retrospective cohort study of 1,750 patients who died from colorectal cancer during 2002-2012 and 3,486 matched controls.

Disclosures: The National Institutes of Health funded the work. The investigators reported having no conflicts of interest except that one coinvestigator is editor in chief of Gastroenterology.

Source: Doubeni CA et al. Gastroenterology. 2018 Sep 27. doi: 10.1053/j.gastro.2018.09.040.

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Gout: new data support treat-to-target approach

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– Failure to reach the therapeutic target of a serum urate level below 6 mg/dL in gout patients is an independent risk factor for all-cause mortality conferring a 139% increased risk, Fernando Perez-Ruiz, MD, PhD, said at the annual meeting of the American College of Rheumatology.

This new finding from a prospective cohort study of 1,193 gout patients constitutes a ringing endorsement that a treat-to-target approach should become the standard in the management of this disease, declared Dr. Perez-Ruiz, a rheumatologist at Hospital Universitario Cruces, Barakaldo, Spain.

“This is encouraging news. We can say to patients and clinicians that we should make every effort to reach the therapeutic target. This is a concept that’s not new in medicine. We do it for diabetes, for hypertension, for hyperlipidemia, and I think now for the first time we will do it for gout,” the rheumatologist said at a press conference highlighting the study findings.

“A lot of physicians including, unfortunately, rheumatologists don’t treat gout to target. They feel like if a patient is doing nicely, that’s good enough. But it’s like lowering cholesterol: If you’re at 400 mg/dL and you go to 300, does that mean it’s fine and you won’t get a myocardial infarction?” he asked rhetorically.

The study included 1,193 gout patients with a mean age at baseline of 60 years, 6.8 years disease duration, and an average of 3-4 flares during the previous year. Mean follow-up was 48 months, translating to 4,830 patient-years of prospective observation. Overall mortality was 13%, mostly from cardiovascular causes.

The mean baseline serum urate level was 9.1 mg/dL. Although both ACR and EULAR guidelines recommend a serum urate level below 6 mg/dL as a therapeutic target, 16.3% of subjects had a level of 6 mg/dL or more despite treatment. The crude mortality rate during follow-up was 80.9 deaths per 1,000 person-years in those with serum urate levels of 6 mg/dL or more, compared with 25.7 per 1,000 person-years in patients with serum urate levels below 6 mg/dL. In a multivariate analysis adjusted for age, prior cardiovascular events, other comorbid conditions, sex, baseline serum urate level, alcohol intake, and other potential confounders, a serum urate of 6 mg/dL or more was independently associated with a 139% increased risk of mortality during follow-up.

“I think the message we would like to give to clinicians is, ‘If you can do that [i.e., maintain the serum urate level below 6 mg/dL], do it. You have the knowledge, you have the means, make the effort. Your patient will benefit from that. Don’t take risks,’” Dr. Perez-Ruiz said.

Session moderator Shraddha Jatwani, MD, a rheumatologist at St. Vincent Hospital in Evansville, Ind., pronounced this a message she will take home to her clinical practice.

“What we usually see in clinical practice is that gout patients are among the most noncompliant. Once they stop hurting they just don’t see the need to take their medication daily. And now that we have this data, we can tell them that their gout medications are like statins, which help reduce the risk of heart attacks. Taking their gout medication will help them reduce their mortality risk. This information will help us to change patient perception,” she said.

[email protected]

SOURCE: Perez-Ruiz F et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 869.

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– Failure to reach the therapeutic target of a serum urate level below 6 mg/dL in gout patients is an independent risk factor for all-cause mortality conferring a 139% increased risk, Fernando Perez-Ruiz, MD, PhD, said at the annual meeting of the American College of Rheumatology.

This new finding from a prospective cohort study of 1,193 gout patients constitutes a ringing endorsement that a treat-to-target approach should become the standard in the management of this disease, declared Dr. Perez-Ruiz, a rheumatologist at Hospital Universitario Cruces, Barakaldo, Spain.

“This is encouraging news. We can say to patients and clinicians that we should make every effort to reach the therapeutic target. This is a concept that’s not new in medicine. We do it for diabetes, for hypertension, for hyperlipidemia, and I think now for the first time we will do it for gout,” the rheumatologist said at a press conference highlighting the study findings.

“A lot of physicians including, unfortunately, rheumatologists don’t treat gout to target. They feel like if a patient is doing nicely, that’s good enough. But it’s like lowering cholesterol: If you’re at 400 mg/dL and you go to 300, does that mean it’s fine and you won’t get a myocardial infarction?” he asked rhetorically.

The study included 1,193 gout patients with a mean age at baseline of 60 years, 6.8 years disease duration, and an average of 3-4 flares during the previous year. Mean follow-up was 48 months, translating to 4,830 patient-years of prospective observation. Overall mortality was 13%, mostly from cardiovascular causes.

The mean baseline serum urate level was 9.1 mg/dL. Although both ACR and EULAR guidelines recommend a serum urate level below 6 mg/dL as a therapeutic target, 16.3% of subjects had a level of 6 mg/dL or more despite treatment. The crude mortality rate during follow-up was 80.9 deaths per 1,000 person-years in those with serum urate levels of 6 mg/dL or more, compared with 25.7 per 1,000 person-years in patients with serum urate levels below 6 mg/dL. In a multivariate analysis adjusted for age, prior cardiovascular events, other comorbid conditions, sex, baseline serum urate level, alcohol intake, and other potential confounders, a serum urate of 6 mg/dL or more was independently associated with a 139% increased risk of mortality during follow-up.

“I think the message we would like to give to clinicians is, ‘If you can do that [i.e., maintain the serum urate level below 6 mg/dL], do it. You have the knowledge, you have the means, make the effort. Your patient will benefit from that. Don’t take risks,’” Dr. Perez-Ruiz said.

Session moderator Shraddha Jatwani, MD, a rheumatologist at St. Vincent Hospital in Evansville, Ind., pronounced this a message she will take home to her clinical practice.

“What we usually see in clinical practice is that gout patients are among the most noncompliant. Once they stop hurting they just don’t see the need to take their medication daily. And now that we have this data, we can tell them that their gout medications are like statins, which help reduce the risk of heart attacks. Taking their gout medication will help them reduce their mortality risk. This information will help us to change patient perception,” she said.

[email protected]

SOURCE: Perez-Ruiz F et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 869.

– Failure to reach the therapeutic target of a serum urate level below 6 mg/dL in gout patients is an independent risk factor for all-cause mortality conferring a 139% increased risk, Fernando Perez-Ruiz, MD, PhD, said at the annual meeting of the American College of Rheumatology.

This new finding from a prospective cohort study of 1,193 gout patients constitutes a ringing endorsement that a treat-to-target approach should become the standard in the management of this disease, declared Dr. Perez-Ruiz, a rheumatologist at Hospital Universitario Cruces, Barakaldo, Spain.

“This is encouraging news. We can say to patients and clinicians that we should make every effort to reach the therapeutic target. This is a concept that’s not new in medicine. We do it for diabetes, for hypertension, for hyperlipidemia, and I think now for the first time we will do it for gout,” the rheumatologist said at a press conference highlighting the study findings.

“A lot of physicians including, unfortunately, rheumatologists don’t treat gout to target. They feel like if a patient is doing nicely, that’s good enough. But it’s like lowering cholesterol: If you’re at 400 mg/dL and you go to 300, does that mean it’s fine and you won’t get a myocardial infarction?” he asked rhetorically.

The study included 1,193 gout patients with a mean age at baseline of 60 years, 6.8 years disease duration, and an average of 3-4 flares during the previous year. Mean follow-up was 48 months, translating to 4,830 patient-years of prospective observation. Overall mortality was 13%, mostly from cardiovascular causes.

The mean baseline serum urate level was 9.1 mg/dL. Although both ACR and EULAR guidelines recommend a serum urate level below 6 mg/dL as a therapeutic target, 16.3% of subjects had a level of 6 mg/dL or more despite treatment. The crude mortality rate during follow-up was 80.9 deaths per 1,000 person-years in those with serum urate levels of 6 mg/dL or more, compared with 25.7 per 1,000 person-years in patients with serum urate levels below 6 mg/dL. In a multivariate analysis adjusted for age, prior cardiovascular events, other comorbid conditions, sex, baseline serum urate level, alcohol intake, and other potential confounders, a serum urate of 6 mg/dL or more was independently associated with a 139% increased risk of mortality during follow-up.

“I think the message we would like to give to clinicians is, ‘If you can do that [i.e., maintain the serum urate level below 6 mg/dL], do it. You have the knowledge, you have the means, make the effort. Your patient will benefit from that. Don’t take risks,’” Dr. Perez-Ruiz said.

Session moderator Shraddha Jatwani, MD, a rheumatologist at St. Vincent Hospital in Evansville, Ind., pronounced this a message she will take home to her clinical practice.

“What we usually see in clinical practice is that gout patients are among the most noncompliant. Once they stop hurting they just don’t see the need to take their medication daily. And now that we have this data, we can tell them that their gout medications are like statins, which help reduce the risk of heart attacks. Taking their gout medication will help them reduce their mortality risk. This information will help us to change patient perception,” she said.

[email protected]

SOURCE: Perez-Ruiz F et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 869.

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Key clinical point: Lowering serum urate in gout patients confers a survival advantage.

Major finding: A serum urate of 6 mg/dL or more in gout patients was independently associated with a 139% increased risk of all-cause mortality.

Study details: This was a prospective study of 1,193 gout patients followed for an average of 4 years.

Disclosures: Dr. Perez-Ruiz reported relationships with Amgen, Grünenthal, and Menarini.

Source: Perez-Ruiz F et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 869.

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Total knee replacement risk soars after arthroscopic surgery for meniscal tear

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– A 5-year follow-up of a major randomized trial comparing methods of meniscal tear management in patients with osteoarthritis showed the risk of total knee replacement was 400% greater in patients who underwent arthroscopic partial meniscectomy than in those who received physical therapy alone, Jeffrey N. Katz, MD, reported at the annual meeting of the American College of Rheumatology.

At 5 years, however, the two divergent initial treatment strategies – arthroscopic surgical repair versus physical therapy – resulted in similar degrees of long-term pain improvement, noted Dr. Katz, a rheumatologist who is professor of medicine and orthopedic surgery at Harvard Medical School, Boston.

“Because that’s the case, a reasonable recommendation – and one that most folks around the world who are thinking about this problem would make – is to have the first choice initially be nonoperative; that is, physical therapy, with surgery reserved for those who don’t improve and who have an interest in undertaking the risks of surgery,” he said.

Dr. Katz presented 5-year follow-up data on 341 participants in the MeTeOR trial, a seven-center study in which middle-age or older subjects with knee pain, a meniscal tear, and osteoarthritic changes on x-ray were randomized to arthroscopic repair or physical therapy. A lot rides on the outcomes of this study, as there is a longstanding debate over the balance of risks and benefits of arthroscopic surgery in this common clinical scenario.

Of the 351 participants, 164 were randomized to and received arthroscopic partial meniscectomy, 109 were randomized to and received a standardized program of physical therapy, and 68 were initially randomized to physical therapy but crossed over to arthroscopic surgery within the first few months because of lack of improvement.

At 5 years of follow-up, all three groups showed similar degrees of improvement in Knee Osteoarthritis and Injury Outcome Score Pain Scale scores, from 40-50 out of a possible 100 at baseline to 20-25 at 6 months, with little change thereafter through 5 years.

The eye-catching finding was the difference in the incidence of total knee replacement (TKR) through 5 years: 10% in those who underwent arthroscopic partial meniscectomy, either as initial therapy or after crossing over from the physical therapy group, compared with 2% in patients who underwent physical therapy alone. Given that more than 400,000 arthroscopic partial meniscectomies are done annually in the United States in patients with knee osteoarthritis, extrapolation from the MeTeOR results suggests an excess of 40,000 total knee replacements in surgically treated patients.

“The higher TKR rates that we observed in surgically treated patients are unexplained, concerning, and require further study. The finding is consistent with the observation in the Osteoarthritis Initiative that TKR rates were higher in patients with arthroscopy as opposed to those treated nonsurgically,” the rheumatologist said.

He proposed two possible explanations for the finding. “It does appear that people who have arthroscopic surgery are then, over the next 5 years, more likely to have total knee replacement. We don’t know whether that is because performing arthroscopic surgery is actually damaging the knee further, leading it to deteriorate more quickly and therefore go on to total knee replacement, or whether when patients develop a relationship with a surgeon and have arthroscopic surgery, they get over some of their apprehension about surgery and may become more likely to accept subsequent surgery for total knee replacement. We hope to find the answer. I think this story is still unfolding because 5 years is a relatively brief period of time in the course of osteoarthritis.

“Arthroscopic surgery certainly offers greater shorter-term improvement, and for some patients that’s worth trading off some downstream risk of joint damage, and for others, they would not want to make that trade-off. So I see it ultimately as a matter of patient choice,” Dr. Katz said.

Knee osteoarthritis affects an estimated 15 million Americans. More than one-half of them have a meniscal tear, the majority of which don’t cause symptoms.

Dr. Katz reported having no financial conflicts regarding MeTeOR, which was funded by the National Institutes of Health.

SOURCE: Katz JN et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 1816.

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– A 5-year follow-up of a major randomized trial comparing methods of meniscal tear management in patients with osteoarthritis showed the risk of total knee replacement was 400% greater in patients who underwent arthroscopic partial meniscectomy than in those who received physical therapy alone, Jeffrey N. Katz, MD, reported at the annual meeting of the American College of Rheumatology.

At 5 years, however, the two divergent initial treatment strategies – arthroscopic surgical repair versus physical therapy – resulted in similar degrees of long-term pain improvement, noted Dr. Katz, a rheumatologist who is professor of medicine and orthopedic surgery at Harvard Medical School, Boston.

“Because that’s the case, a reasonable recommendation – and one that most folks around the world who are thinking about this problem would make – is to have the first choice initially be nonoperative; that is, physical therapy, with surgery reserved for those who don’t improve and who have an interest in undertaking the risks of surgery,” he said.

Dr. Katz presented 5-year follow-up data on 341 participants in the MeTeOR trial, a seven-center study in which middle-age or older subjects with knee pain, a meniscal tear, and osteoarthritic changes on x-ray were randomized to arthroscopic repair or physical therapy. A lot rides on the outcomes of this study, as there is a longstanding debate over the balance of risks and benefits of arthroscopic surgery in this common clinical scenario.

Of the 351 participants, 164 were randomized to and received arthroscopic partial meniscectomy, 109 were randomized to and received a standardized program of physical therapy, and 68 were initially randomized to physical therapy but crossed over to arthroscopic surgery within the first few months because of lack of improvement.

At 5 years of follow-up, all three groups showed similar degrees of improvement in Knee Osteoarthritis and Injury Outcome Score Pain Scale scores, from 40-50 out of a possible 100 at baseline to 20-25 at 6 months, with little change thereafter through 5 years.

The eye-catching finding was the difference in the incidence of total knee replacement (TKR) through 5 years: 10% in those who underwent arthroscopic partial meniscectomy, either as initial therapy or after crossing over from the physical therapy group, compared with 2% in patients who underwent physical therapy alone. Given that more than 400,000 arthroscopic partial meniscectomies are done annually in the United States in patients with knee osteoarthritis, extrapolation from the MeTeOR results suggests an excess of 40,000 total knee replacements in surgically treated patients.

“The higher TKR rates that we observed in surgically treated patients are unexplained, concerning, and require further study. The finding is consistent with the observation in the Osteoarthritis Initiative that TKR rates were higher in patients with arthroscopy as opposed to those treated nonsurgically,” the rheumatologist said.

He proposed two possible explanations for the finding. “It does appear that people who have arthroscopic surgery are then, over the next 5 years, more likely to have total knee replacement. We don’t know whether that is because performing arthroscopic surgery is actually damaging the knee further, leading it to deteriorate more quickly and therefore go on to total knee replacement, or whether when patients develop a relationship with a surgeon and have arthroscopic surgery, they get over some of their apprehension about surgery and may become more likely to accept subsequent surgery for total knee replacement. We hope to find the answer. I think this story is still unfolding because 5 years is a relatively brief period of time in the course of osteoarthritis.

“Arthroscopic surgery certainly offers greater shorter-term improvement, and for some patients that’s worth trading off some downstream risk of joint damage, and for others, they would not want to make that trade-off. So I see it ultimately as a matter of patient choice,” Dr. Katz said.

Knee osteoarthritis affects an estimated 15 million Americans. More than one-half of them have a meniscal tear, the majority of which don’t cause symptoms.

Dr. Katz reported having no financial conflicts regarding MeTeOR, which was funded by the National Institutes of Health.

SOURCE: Katz JN et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 1816.

– A 5-year follow-up of a major randomized trial comparing methods of meniscal tear management in patients with osteoarthritis showed the risk of total knee replacement was 400% greater in patients who underwent arthroscopic partial meniscectomy than in those who received physical therapy alone, Jeffrey N. Katz, MD, reported at the annual meeting of the American College of Rheumatology.

At 5 years, however, the two divergent initial treatment strategies – arthroscopic surgical repair versus physical therapy – resulted in similar degrees of long-term pain improvement, noted Dr. Katz, a rheumatologist who is professor of medicine and orthopedic surgery at Harvard Medical School, Boston.

“Because that’s the case, a reasonable recommendation – and one that most folks around the world who are thinking about this problem would make – is to have the first choice initially be nonoperative; that is, physical therapy, with surgery reserved for those who don’t improve and who have an interest in undertaking the risks of surgery,” he said.

Dr. Katz presented 5-year follow-up data on 341 participants in the MeTeOR trial, a seven-center study in which middle-age or older subjects with knee pain, a meniscal tear, and osteoarthritic changes on x-ray were randomized to arthroscopic repair or physical therapy. A lot rides on the outcomes of this study, as there is a longstanding debate over the balance of risks and benefits of arthroscopic surgery in this common clinical scenario.

Of the 351 participants, 164 were randomized to and received arthroscopic partial meniscectomy, 109 were randomized to and received a standardized program of physical therapy, and 68 were initially randomized to physical therapy but crossed over to arthroscopic surgery within the first few months because of lack of improvement.

At 5 years of follow-up, all three groups showed similar degrees of improvement in Knee Osteoarthritis and Injury Outcome Score Pain Scale scores, from 40-50 out of a possible 100 at baseline to 20-25 at 6 months, with little change thereafter through 5 years.

The eye-catching finding was the difference in the incidence of total knee replacement (TKR) through 5 years: 10% in those who underwent arthroscopic partial meniscectomy, either as initial therapy or after crossing over from the physical therapy group, compared with 2% in patients who underwent physical therapy alone. Given that more than 400,000 arthroscopic partial meniscectomies are done annually in the United States in patients with knee osteoarthritis, extrapolation from the MeTeOR results suggests an excess of 40,000 total knee replacements in surgically treated patients.

“The higher TKR rates that we observed in surgically treated patients are unexplained, concerning, and require further study. The finding is consistent with the observation in the Osteoarthritis Initiative that TKR rates were higher in patients with arthroscopy as opposed to those treated nonsurgically,” the rheumatologist said.

He proposed two possible explanations for the finding. “It does appear that people who have arthroscopic surgery are then, over the next 5 years, more likely to have total knee replacement. We don’t know whether that is because performing arthroscopic surgery is actually damaging the knee further, leading it to deteriorate more quickly and therefore go on to total knee replacement, or whether when patients develop a relationship with a surgeon and have arthroscopic surgery, they get over some of their apprehension about surgery and may become more likely to accept subsequent surgery for total knee replacement. We hope to find the answer. I think this story is still unfolding because 5 years is a relatively brief period of time in the course of osteoarthritis.

“Arthroscopic surgery certainly offers greater shorter-term improvement, and for some patients that’s worth trading off some downstream risk of joint damage, and for others, they would not want to make that trade-off. So I see it ultimately as a matter of patient choice,” Dr. Katz said.

Knee osteoarthritis affects an estimated 15 million Americans. More than one-half of them have a meniscal tear, the majority of which don’t cause symptoms.

Dr. Katz reported having no financial conflicts regarding MeTeOR, which was funded by the National Institutes of Health.

SOURCE: Katz JN et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 1816.

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Key clinical point: Risk of total knee replacement is five times higher after arthroscopic partial meniscectomy.

Major finding: Patients randomized to arthroscopic partial meniscectomy were 400% more likely to subsequently undergo total knee replacement than were those randomized to physical therapy alone.

Study details: This was a presentation of the 5-year follow-up results in 341 participants in the MeTeOR trial, a seven-center study in which middle-age or older subjects with knee pain, a meniscal tear, and osteoarthritic changes on x-ray were randomized to arthroscopic repair or physical therapy.

Disclosures: The presenter reported having no financial conflicts regarding MeTeOR, which was funded by the National Institutes of Health.

Source: Katz JN et al. Arthritis Rheumatol. 2018;70(Suppl 10): Abstract 1816.

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‘Phenomenal’ REDUCE-IT establishes triglyceride theory

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– REDUCE-IT is a phenomenal trial and a game changer because it has shown for the first time that triglyceride reduction with an appropriate therapy – in this case icosapent ethyl – when used in appropriate doses can make a significant difference.

That’s according to Prakash C. Deedwania, MD, chief of the cardiology division at the Veterans Affairs Medical Center/University of California San Francisco Program in Fresno, who joined MDedge reporter Richard Mark Kirkner for a video interview at the American Heart Association scientific sessions.

In the large, placebo-controlled REDUCE-IT trial in patients with or at high risk for cardiovascular disease received who received 2 g of icosapent ethyl (Vascepa) twice daily or placebo saw a 25% lower risk of cardiovascular death or an ischemic event, compared with placebo.

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– REDUCE-IT is a phenomenal trial and a game changer because it has shown for the first time that triglyceride reduction with an appropriate therapy – in this case icosapent ethyl – when used in appropriate doses can make a significant difference.

That’s according to Prakash C. Deedwania, MD, chief of the cardiology division at the Veterans Affairs Medical Center/University of California San Francisco Program in Fresno, who joined MDedge reporter Richard Mark Kirkner for a video interview at the American Heart Association scientific sessions.

In the large, placebo-controlled REDUCE-IT trial in patients with or at high risk for cardiovascular disease received who received 2 g of icosapent ethyl (Vascepa) twice daily or placebo saw a 25% lower risk of cardiovascular death or an ischemic event, compared with placebo.

– REDUCE-IT is a phenomenal trial and a game changer because it has shown for the first time that triglyceride reduction with an appropriate therapy – in this case icosapent ethyl – when used in appropriate doses can make a significant difference.

That’s according to Prakash C. Deedwania, MD, chief of the cardiology division at the Veterans Affairs Medical Center/University of California San Francisco Program in Fresno, who joined MDedge reporter Richard Mark Kirkner for a video interview at the American Heart Association scientific sessions.

In the large, placebo-controlled REDUCE-IT trial in patients with or at high risk for cardiovascular disease received who received 2 g of icosapent ethyl (Vascepa) twice daily or placebo saw a 25% lower risk of cardiovascular death or an ischemic event, compared with placebo.

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CAC scores in type 1 diabetes no higher than general population

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– Roughly 70% of some 1,200 adult patients with type 1 diabetes screened for coronary artery calcium had a score of zero, about the same prevalence as in the general, U.S. adult population, suggesting the unexpected conclusion that a majority of middle-aged patients with type 1 diabetes do not have an elevated risk for coronary artery disease, in contrast to patients with type 2 diabetes.

Among 1,205 asymptomatic people with type 1 diabetes who underwent coronary artery calcium (CAC) measurement and were followed for an average of about 11 years, 71% had a CAC score of zero at baseline followed by a cardiovascular disease event rate of 5.6 events/1,000 patient years of follow-up, a “very low” event rate that made these patients no more likely to have an event than any adult of similar age and sex in the general U.S. population, Matthew J. Budoff, MD, said at the American Heart Association scientific sessions.


In prior reports, about half of patients with type 2 diabetes had a CAC score of zero, noted Dr. Budoff, professor of medicine and a specialist in cardiac CT imaging and preventive cardiology at the University of California, Los Angeles. In a general adult population that’s about 45 years old roughly three-quarters would have a CAC score of zero, he noted.

Until now, little has been known about CAC scores in asymptomatic, middle-aged adults with type 1 diabetes. The findings reported by Dr. Budoff raise questions about the 2018 revision of the cholesterol guideline from the American College of Cardiology and American Heart Association, released during the meeting (J Am Coll Cardiol. 2018. doi: 10.1016/j.jacc.2018.11.003), which lumps type 1 and type 2 diabetes together as a special high-risk category for cholesterol management.


The guideline should instead “advocate for more therapy with a CAC score of more than 100 and less therapy with a CAC score of zero in patients with type 1 diabetes,” Dr. Budoff suggested. “A statin for someone with a CAC score of zero probably won’t result in event reduction. The 70% of patients with type 1 diabetes who have a CAC score of zero potentially may not benefit from a statin,” he said in a video interview.

Dr. Budoff and his associates used CAC scores and outcomes data collected on 1,205 asymptomatic people with type 1 diabetes enrolled in the EDIC (Epidemiology of Diabetes Interventions and Complications) trial who underwent CAC scoring as part of the study protocol when they averaged 43 years of age. Follow-up tracked the incidence of cardiovascular disease events in 1,156 of these patients for an average of about 11 years. During follow-up, 105 patients had a cardiovascular disease event, an overall rate of 8.5 events/1,000 patient years of follow-up.

The results also confirmed the prognostic power of the CAC score in these patients. Compared with the very low event rate among those with a zero score, patients with a score of 1-100 had 71% more events, patients with a CAC score of 101-300 had a 5.4-fold higher event rate as those with no coronary calcium, and patients with a CAC score of greater than 300 had a 6.9-fold higher event rate than those with no coronary calcium, Dr. Budoff reported.

Coronary calcium deposits, a direct reflection of atheroma load, can change over time, but somewhat slowly. A CAC score of zero is very reliable for predicting a very low rate of cardiovascular disease events over the subsequent 5 years, and in many people it can reliably predict for as long as 10 years, Dr. Budoff said. Beyond that, follow-up CAC scoring is necessary to check for changes in coronary status, “especially in patients with type 1 diabetes,”

SOURCE: Budoff M et al. Abstract 13133.

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– Roughly 70% of some 1,200 adult patients with type 1 diabetes screened for coronary artery calcium had a score of zero, about the same prevalence as in the general, U.S. adult population, suggesting the unexpected conclusion that a majority of middle-aged patients with type 1 diabetes do not have an elevated risk for coronary artery disease, in contrast to patients with type 2 diabetes.

Among 1,205 asymptomatic people with type 1 diabetes who underwent coronary artery calcium (CAC) measurement and were followed for an average of about 11 years, 71% had a CAC score of zero at baseline followed by a cardiovascular disease event rate of 5.6 events/1,000 patient years of follow-up, a “very low” event rate that made these patients no more likely to have an event than any adult of similar age and sex in the general U.S. population, Matthew J. Budoff, MD, said at the American Heart Association scientific sessions.


In prior reports, about half of patients with type 2 diabetes had a CAC score of zero, noted Dr. Budoff, professor of medicine and a specialist in cardiac CT imaging and preventive cardiology at the University of California, Los Angeles. In a general adult population that’s about 45 years old roughly three-quarters would have a CAC score of zero, he noted.

Until now, little has been known about CAC scores in asymptomatic, middle-aged adults with type 1 diabetes. The findings reported by Dr. Budoff raise questions about the 2018 revision of the cholesterol guideline from the American College of Cardiology and American Heart Association, released during the meeting (J Am Coll Cardiol. 2018. doi: 10.1016/j.jacc.2018.11.003), which lumps type 1 and type 2 diabetes together as a special high-risk category for cholesterol management.


The guideline should instead “advocate for more therapy with a CAC score of more than 100 and less therapy with a CAC score of zero in patients with type 1 diabetes,” Dr. Budoff suggested. “A statin for someone with a CAC score of zero probably won’t result in event reduction. The 70% of patients with type 1 diabetes who have a CAC score of zero potentially may not benefit from a statin,” he said in a video interview.

Dr. Budoff and his associates used CAC scores and outcomes data collected on 1,205 asymptomatic people with type 1 diabetes enrolled in the EDIC (Epidemiology of Diabetes Interventions and Complications) trial who underwent CAC scoring as part of the study protocol when they averaged 43 years of age. Follow-up tracked the incidence of cardiovascular disease events in 1,156 of these patients for an average of about 11 years. During follow-up, 105 patients had a cardiovascular disease event, an overall rate of 8.5 events/1,000 patient years of follow-up.

The results also confirmed the prognostic power of the CAC score in these patients. Compared with the very low event rate among those with a zero score, patients with a score of 1-100 had 71% more events, patients with a CAC score of 101-300 had a 5.4-fold higher event rate as those with no coronary calcium, and patients with a CAC score of greater than 300 had a 6.9-fold higher event rate than those with no coronary calcium, Dr. Budoff reported.

Coronary calcium deposits, a direct reflection of atheroma load, can change over time, but somewhat slowly. A CAC score of zero is very reliable for predicting a very low rate of cardiovascular disease events over the subsequent 5 years, and in many people it can reliably predict for as long as 10 years, Dr. Budoff said. Beyond that, follow-up CAC scoring is necessary to check for changes in coronary status, “especially in patients with type 1 diabetes,”

SOURCE: Budoff M et al. Abstract 13133.

– Roughly 70% of some 1,200 adult patients with type 1 diabetes screened for coronary artery calcium had a score of zero, about the same prevalence as in the general, U.S. adult population, suggesting the unexpected conclusion that a majority of middle-aged patients with type 1 diabetes do not have an elevated risk for coronary artery disease, in contrast to patients with type 2 diabetes.

Among 1,205 asymptomatic people with type 1 diabetes who underwent coronary artery calcium (CAC) measurement and were followed for an average of about 11 years, 71% had a CAC score of zero at baseline followed by a cardiovascular disease event rate of 5.6 events/1,000 patient years of follow-up, a “very low” event rate that made these patients no more likely to have an event than any adult of similar age and sex in the general U.S. population, Matthew J. Budoff, MD, said at the American Heart Association scientific sessions.


In prior reports, about half of patients with type 2 diabetes had a CAC score of zero, noted Dr. Budoff, professor of medicine and a specialist in cardiac CT imaging and preventive cardiology at the University of California, Los Angeles. In a general adult population that’s about 45 years old roughly three-quarters would have a CAC score of zero, he noted.

Until now, little has been known about CAC scores in asymptomatic, middle-aged adults with type 1 diabetes. The findings reported by Dr. Budoff raise questions about the 2018 revision of the cholesterol guideline from the American College of Cardiology and American Heart Association, released during the meeting (J Am Coll Cardiol. 2018. doi: 10.1016/j.jacc.2018.11.003), which lumps type 1 and type 2 diabetes together as a special high-risk category for cholesterol management.


The guideline should instead “advocate for more therapy with a CAC score of more than 100 and less therapy with a CAC score of zero in patients with type 1 diabetes,” Dr. Budoff suggested. “A statin for someone with a CAC score of zero probably won’t result in event reduction. The 70% of patients with type 1 diabetes who have a CAC score of zero potentially may not benefit from a statin,” he said in a video interview.

Dr. Budoff and his associates used CAC scores and outcomes data collected on 1,205 asymptomatic people with type 1 diabetes enrolled in the EDIC (Epidemiology of Diabetes Interventions and Complications) trial who underwent CAC scoring as part of the study protocol when they averaged 43 years of age. Follow-up tracked the incidence of cardiovascular disease events in 1,156 of these patients for an average of about 11 years. During follow-up, 105 patients had a cardiovascular disease event, an overall rate of 8.5 events/1,000 patient years of follow-up.

The results also confirmed the prognostic power of the CAC score in these patients. Compared with the very low event rate among those with a zero score, patients with a score of 1-100 had 71% more events, patients with a CAC score of 101-300 had a 5.4-fold higher event rate as those with no coronary calcium, and patients with a CAC score of greater than 300 had a 6.9-fold higher event rate than those with no coronary calcium, Dr. Budoff reported.

Coronary calcium deposits, a direct reflection of atheroma load, can change over time, but somewhat slowly. A CAC score of zero is very reliable for predicting a very low rate of cardiovascular disease events over the subsequent 5 years, and in many people it can reliably predict for as long as 10 years, Dr. Budoff said. Beyond that, follow-up CAC scoring is necessary to check for changes in coronary status, “especially in patients with type 1 diabetes,”

SOURCE: Budoff M et al. Abstract 13133.

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Key clinical point: A surprisingly high percentage of patients with type 1 diabetes have a coronary artery calcium score of zero.

Major finding: Seventy-one percent of patients with type 1 diabetes had a coronary artery calcium score of zero.

Study details: Review of data collected from 1,205 patients in the EDIC trial.

Disclosures: The EDIC trial had no commercial funding. Dr. Budoff has received research funding from General Electric.

Source: Budoff M et al. AHA 2018, Abstract 13133.

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A paradigm shift in medical research is necessary

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Thu, 06/10/2021 - 15:18

– What doctors think they know to be true in medicine has changed dramatically in the past several decades and will be different again in the decades to come, leaving them with a dilemma, according to Kevin T. Powell, MD, PhD, a pediatric hospitalist in St. Louis. If half of what doctors teach or know in medicine today will ultimately end up not being true, how do they know what to believe or accept?

While there is not a single satisfactory answer to that question, researchers can select research that gets doctors closer to reliable findings and steer them away from the barrage of poor-quality research that emerges from the current publish-or-perish system, Dr. Powell told his colleagues at the annual meeting of the American Academy of Pediatrics.

During his talk, Dr. Powell discussed the challenges and flaws with medical research as it is currently conducted, citing Doug Altman’s writings on these problems as early as 1994.

“The poor quality of much medical research is widely acknowledged, yet disturbingly the leaders of the medical profession seem only minimally concerned about the problem and make no apparent effort to find a solution,” wrote Mr. Altman, an English medical statistician (BMJ. 1994;308:283).

“We need less research, better research, and research done for the right reasons,” Mr. Altman concluded. “Abandoning using the number of publications as a measure of ability would be a start.”

In an interview, Dr. Powell described an unfortunate consequence of the publish-or-perish pressure in academic medicine: A glut of short-term, small studies with little clinical utility that researchers can complete in 1 or 2 years rather than the large, multicenter studies that take several years – and produce higher-quality findings – but cannot be turned into as many publications.

“We’re generating a lot of medical research findings that end up being false,” he said. “It’s a random walk in terms of getting to the truth rather than having an accurate process of getting to truth through evidence-based medicine.”

But he was hopeful, not cynical, about the way forward. By persuading people that medical research has changed for the worse over time and can change into something better, Dr. Powell saw potential for future research resulting in the same sort of public health achievements that research produced in the past, such as big reductions in smoking or sudden infant death syndrome.

Dr. Powell concluded his talk with a riff on Martin Luther’s 95 Theses, the 9.5 Theses, for a reformation of evidence-based medicine that together address the various shortcomings he discussed.

1. Recognize academic promotion as a bias, just like drug money.

2. Don’t confound statistically significant and clinically significant.

3. Use only significant figures.

4. Use the phrase “we did not DETECT a difference” and include power calculations.

5. Use confidence intervals instead of P values.

6. Use number needed to harm and number needed to treat instead of relative risk.

7. Absence of proof is not proof of absence. When there is insufficient randomized, controlled trial evidence, have an independent party estimate an effect based on non-RCT articles.

8. Any article implying clinical practice should change must include a counterpoint and a benefit cost analysis. Consider both effectiveness and safety.

9. Use postmarketing peer review.

9.5. Beware of research based on surveys.

Dr. Powell reported no relevant financial disclosures.
 

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– What doctors think they know to be true in medicine has changed dramatically in the past several decades and will be different again in the decades to come, leaving them with a dilemma, according to Kevin T. Powell, MD, PhD, a pediatric hospitalist in St. Louis. If half of what doctors teach or know in medicine today will ultimately end up not being true, how do they know what to believe or accept?

While there is not a single satisfactory answer to that question, researchers can select research that gets doctors closer to reliable findings and steer them away from the barrage of poor-quality research that emerges from the current publish-or-perish system, Dr. Powell told his colleagues at the annual meeting of the American Academy of Pediatrics.

During his talk, Dr. Powell discussed the challenges and flaws with medical research as it is currently conducted, citing Doug Altman’s writings on these problems as early as 1994.

“The poor quality of much medical research is widely acknowledged, yet disturbingly the leaders of the medical profession seem only minimally concerned about the problem and make no apparent effort to find a solution,” wrote Mr. Altman, an English medical statistician (BMJ. 1994;308:283).

“We need less research, better research, and research done for the right reasons,” Mr. Altman concluded. “Abandoning using the number of publications as a measure of ability would be a start.”

In an interview, Dr. Powell described an unfortunate consequence of the publish-or-perish pressure in academic medicine: A glut of short-term, small studies with little clinical utility that researchers can complete in 1 or 2 years rather than the large, multicenter studies that take several years – and produce higher-quality findings – but cannot be turned into as many publications.

“We’re generating a lot of medical research findings that end up being false,” he said. “It’s a random walk in terms of getting to the truth rather than having an accurate process of getting to truth through evidence-based medicine.”

But he was hopeful, not cynical, about the way forward. By persuading people that medical research has changed for the worse over time and can change into something better, Dr. Powell saw potential for future research resulting in the same sort of public health achievements that research produced in the past, such as big reductions in smoking or sudden infant death syndrome.

Dr. Powell concluded his talk with a riff on Martin Luther’s 95 Theses, the 9.5 Theses, for a reformation of evidence-based medicine that together address the various shortcomings he discussed.

1. Recognize academic promotion as a bias, just like drug money.

2. Don’t confound statistically significant and clinically significant.

3. Use only significant figures.

4. Use the phrase “we did not DETECT a difference” and include power calculations.

5. Use confidence intervals instead of P values.

6. Use number needed to harm and number needed to treat instead of relative risk.

7. Absence of proof is not proof of absence. When there is insufficient randomized, controlled trial evidence, have an independent party estimate an effect based on non-RCT articles.

8. Any article implying clinical practice should change must include a counterpoint and a benefit cost analysis. Consider both effectiveness and safety.

9. Use postmarketing peer review.

9.5. Beware of research based on surveys.

Dr. Powell reported no relevant financial disclosures.
 

– What doctors think they know to be true in medicine has changed dramatically in the past several decades and will be different again in the decades to come, leaving them with a dilemma, according to Kevin T. Powell, MD, PhD, a pediatric hospitalist in St. Louis. If half of what doctors teach or know in medicine today will ultimately end up not being true, how do they know what to believe or accept?

While there is not a single satisfactory answer to that question, researchers can select research that gets doctors closer to reliable findings and steer them away from the barrage of poor-quality research that emerges from the current publish-or-perish system, Dr. Powell told his colleagues at the annual meeting of the American Academy of Pediatrics.

During his talk, Dr. Powell discussed the challenges and flaws with medical research as it is currently conducted, citing Doug Altman’s writings on these problems as early as 1994.

“The poor quality of much medical research is widely acknowledged, yet disturbingly the leaders of the medical profession seem only minimally concerned about the problem and make no apparent effort to find a solution,” wrote Mr. Altman, an English medical statistician (BMJ. 1994;308:283).

“We need less research, better research, and research done for the right reasons,” Mr. Altman concluded. “Abandoning using the number of publications as a measure of ability would be a start.”

In an interview, Dr. Powell described an unfortunate consequence of the publish-or-perish pressure in academic medicine: A glut of short-term, small studies with little clinical utility that researchers can complete in 1 or 2 years rather than the large, multicenter studies that take several years – and produce higher-quality findings – but cannot be turned into as many publications.

“We’re generating a lot of medical research findings that end up being false,” he said. “It’s a random walk in terms of getting to the truth rather than having an accurate process of getting to truth through evidence-based medicine.”

But he was hopeful, not cynical, about the way forward. By persuading people that medical research has changed for the worse over time and can change into something better, Dr. Powell saw potential for future research resulting in the same sort of public health achievements that research produced in the past, such as big reductions in smoking or sudden infant death syndrome.

Dr. Powell concluded his talk with a riff on Martin Luther’s 95 Theses, the 9.5 Theses, for a reformation of evidence-based medicine that together address the various shortcomings he discussed.

1. Recognize academic promotion as a bias, just like drug money.

2. Don’t confound statistically significant and clinically significant.

3. Use only significant figures.

4. Use the phrase “we did not DETECT a difference” and include power calculations.

5. Use confidence intervals instead of P values.

6. Use number needed to harm and number needed to treat instead of relative risk.

7. Absence of proof is not proof of absence. When there is insufficient randomized, controlled trial evidence, have an independent party estimate an effect based on non-RCT articles.

8. Any article implying clinical practice should change must include a counterpoint and a benefit cost analysis. Consider both effectiveness and safety.

9. Use postmarketing peer review.

9.5. Beware of research based on surveys.

Dr. Powell reported no relevant financial disclosures.
 

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