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To tame prescription prices, HHS dips a toe into drug importation stream
It came as something of a surprise when Health & Human Services Secretary Alex Azar announced that the administration was exploring the importation of prescription drugs to fight high domestic prices. Sec. Azar and Scott Gottlieb, MD, commissioner of the Food and Drug Administration, who also endorsed the new proposal, had previously opposed the idea.
But drug prices in the United States have continued to rise and more than 80% of Americans say the government should take action. President Donald Trump has said drugmakers are “getting away with murder” and has angrily tweeted at companies about individual price hikes.
Although candidate Trump supported the idea of allowing patients to import medicines, since he was elected, he has not mentioned that option – which is strongly opposed by drug companies.
Now, determined to explore more avenues to curb price hikes, the administration is signaling that it is willing to consider what the industry regards as something of a nuclear option to address a recalcitrant problem. Carefully tailored to focus solely on specific situations in which a high-priced drug is made by only one company, it is finding support where broader proposals have failed.
“They’re approaching it incrementally and wisely, they’re focusing on prices where there’s a need,” said Dan Mendelson, the founder of health care consultant company Avalere and an official in the Clinton White House. “It is certainly more narrow than the way others have conceptualized it.”
Far from a blanket legalization of imported medicines, the working group Sec. Azar convened will study importation to combat sudden price increases in specific drugs. The focus is on temporarily bringing in cheaper similar or identical drugs to introduce competition into the U.S. market. The medicines must be off patent and have only one manufacturer here.
The secretary’s memo said the effort is designed to avoid the kind of overnight increases seen with Daraprim in 2015. That price hike was engineered by “pharma bro” Martin Shkreli, then CEO of Turing Pharmaceuticals. He purchased the rights to the single-sourced medication that treats parasitic infections and began charging $750 for a pill that formerly cost $13.50 and costs a little more than a dollar in much of the world. Turing was the only U.S. producer.
“This is a workable solution to a discrete problem,” said Ameet Sarpatwari, PhD, JD, an instructor in medicine at Harvard Medical School in Boston.
But those who support more sweeping importation policies decried the plan’s limited scope and suspected the announcement was part theatrics and part a threatening signal to drugmakers.
“This could just be a dog-and-pony show, where they’re calling in an expert group to explore avenues of importation – but when all is said and done, they find that they don’t want to do this,” said Gabriel Levitt, the cofounder of PharmacyChecker.com, a private company that verifies international online pharmacies and compares prescription drug prices for consumers.
“At that point, we’ll learn that the exercise was lip service,” he added. “Frankly, there’s a good chance that that is the case.”
This isn’t the first time officials have suggested importing drugs from other countries to find better prices. Bills have been offered in Congress to allow it, and George W. Bush administration officials investigated the issue and produced two reports questioning the safety of such efforts in 2004.
Overall, the measure is by no means a silver bullet to the larger problem of rising drug prices, said Rachel Sachs, JD, an associate professor of law at Washington University School of Law in St. Louis.
“It’s a really smart move to solve one of the many drug pricing problems we observe, but, of course, it won’t address every problem,” Sachs said.
Mr. Mendelson suggested this working group might be an effort to placate patients who have seen little movement to bring down drug costs, despite the president’s repeated promises to provide help.
“If the goal is to make policy changes that are visible and help with the 2018 and 2020 election, I think it’s right up there with a lot of the things they’re doing,” Mendelson said. “If the goal is truly to help consumers with drug prices, not so much.”
In addition, since the group’s work applies primarily to the generic drug market, a new policy would stop short of taming the price spirals and high launch prices of blockbuster brand-name drugs, which Harvard’s Sarpatwari said were the “elephants in the room” of the drug pricing debate.
Mr. Levitt pointed out that, while a big overnight increase on a drug might trigger action to allow importation, the move would do nothing to stop the yearly increases that drug companies tack on to medicines. Depending on how possible regulations are written, such increases might even be encouraged. The administration has been pressuring pharmaceutical makers to hold down those rising prices but finding tepid support among the companies.
Even if the policy targets just a slice of the overall problem, it could still make a difference for Americans struggling to pay for off-patent drugs and provide more competition.
“If Azar is serious about this proposal, even though it’s very limited in scope, it could help deter the most egregious forms of drug price gouging where there are single-source meds,” Mr. Levitt said.
KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
It came as something of a surprise when Health & Human Services Secretary Alex Azar announced that the administration was exploring the importation of prescription drugs to fight high domestic prices. Sec. Azar and Scott Gottlieb, MD, commissioner of the Food and Drug Administration, who also endorsed the new proposal, had previously opposed the idea.
But drug prices in the United States have continued to rise and more than 80% of Americans say the government should take action. President Donald Trump has said drugmakers are “getting away with murder” and has angrily tweeted at companies about individual price hikes.
Although candidate Trump supported the idea of allowing patients to import medicines, since he was elected, he has not mentioned that option – which is strongly opposed by drug companies.
Now, determined to explore more avenues to curb price hikes, the administration is signaling that it is willing to consider what the industry regards as something of a nuclear option to address a recalcitrant problem. Carefully tailored to focus solely on specific situations in which a high-priced drug is made by only one company, it is finding support where broader proposals have failed.
“They’re approaching it incrementally and wisely, they’re focusing on prices where there’s a need,” said Dan Mendelson, the founder of health care consultant company Avalere and an official in the Clinton White House. “It is certainly more narrow than the way others have conceptualized it.”
Far from a blanket legalization of imported medicines, the working group Sec. Azar convened will study importation to combat sudden price increases in specific drugs. The focus is on temporarily bringing in cheaper similar or identical drugs to introduce competition into the U.S. market. The medicines must be off patent and have only one manufacturer here.
The secretary’s memo said the effort is designed to avoid the kind of overnight increases seen with Daraprim in 2015. That price hike was engineered by “pharma bro” Martin Shkreli, then CEO of Turing Pharmaceuticals. He purchased the rights to the single-sourced medication that treats parasitic infections and began charging $750 for a pill that formerly cost $13.50 and costs a little more than a dollar in much of the world. Turing was the only U.S. producer.
“This is a workable solution to a discrete problem,” said Ameet Sarpatwari, PhD, JD, an instructor in medicine at Harvard Medical School in Boston.
But those who support more sweeping importation policies decried the plan’s limited scope and suspected the announcement was part theatrics and part a threatening signal to drugmakers.
“This could just be a dog-and-pony show, where they’re calling in an expert group to explore avenues of importation – but when all is said and done, they find that they don’t want to do this,” said Gabriel Levitt, the cofounder of PharmacyChecker.com, a private company that verifies international online pharmacies and compares prescription drug prices for consumers.
“At that point, we’ll learn that the exercise was lip service,” he added. “Frankly, there’s a good chance that that is the case.”
This isn’t the first time officials have suggested importing drugs from other countries to find better prices. Bills have been offered in Congress to allow it, and George W. Bush administration officials investigated the issue and produced two reports questioning the safety of such efforts in 2004.
Overall, the measure is by no means a silver bullet to the larger problem of rising drug prices, said Rachel Sachs, JD, an associate professor of law at Washington University School of Law in St. Louis.
“It’s a really smart move to solve one of the many drug pricing problems we observe, but, of course, it won’t address every problem,” Sachs said.
Mr. Mendelson suggested this working group might be an effort to placate patients who have seen little movement to bring down drug costs, despite the president’s repeated promises to provide help.
“If the goal is to make policy changes that are visible and help with the 2018 and 2020 election, I think it’s right up there with a lot of the things they’re doing,” Mendelson said. “If the goal is truly to help consumers with drug prices, not so much.”
In addition, since the group’s work applies primarily to the generic drug market, a new policy would stop short of taming the price spirals and high launch prices of blockbuster brand-name drugs, which Harvard’s Sarpatwari said were the “elephants in the room” of the drug pricing debate.
Mr. Levitt pointed out that, while a big overnight increase on a drug might trigger action to allow importation, the move would do nothing to stop the yearly increases that drug companies tack on to medicines. Depending on how possible regulations are written, such increases might even be encouraged. The administration has been pressuring pharmaceutical makers to hold down those rising prices but finding tepid support among the companies.
Even if the policy targets just a slice of the overall problem, it could still make a difference for Americans struggling to pay for off-patent drugs and provide more competition.
“If Azar is serious about this proposal, even though it’s very limited in scope, it could help deter the most egregious forms of drug price gouging where there are single-source meds,” Mr. Levitt said.
KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
It came as something of a surprise when Health & Human Services Secretary Alex Azar announced that the administration was exploring the importation of prescription drugs to fight high domestic prices. Sec. Azar and Scott Gottlieb, MD, commissioner of the Food and Drug Administration, who also endorsed the new proposal, had previously opposed the idea.
But drug prices in the United States have continued to rise and more than 80% of Americans say the government should take action. President Donald Trump has said drugmakers are “getting away with murder” and has angrily tweeted at companies about individual price hikes.
Although candidate Trump supported the idea of allowing patients to import medicines, since he was elected, he has not mentioned that option – which is strongly opposed by drug companies.
Now, determined to explore more avenues to curb price hikes, the administration is signaling that it is willing to consider what the industry regards as something of a nuclear option to address a recalcitrant problem. Carefully tailored to focus solely on specific situations in which a high-priced drug is made by only one company, it is finding support where broader proposals have failed.
“They’re approaching it incrementally and wisely, they’re focusing on prices where there’s a need,” said Dan Mendelson, the founder of health care consultant company Avalere and an official in the Clinton White House. “It is certainly more narrow than the way others have conceptualized it.”
Far from a blanket legalization of imported medicines, the working group Sec. Azar convened will study importation to combat sudden price increases in specific drugs. The focus is on temporarily bringing in cheaper similar or identical drugs to introduce competition into the U.S. market. The medicines must be off patent and have only one manufacturer here.
The secretary’s memo said the effort is designed to avoid the kind of overnight increases seen with Daraprim in 2015. That price hike was engineered by “pharma bro” Martin Shkreli, then CEO of Turing Pharmaceuticals. He purchased the rights to the single-sourced medication that treats parasitic infections and began charging $750 for a pill that formerly cost $13.50 and costs a little more than a dollar in much of the world. Turing was the only U.S. producer.
“This is a workable solution to a discrete problem,” said Ameet Sarpatwari, PhD, JD, an instructor in medicine at Harvard Medical School in Boston.
But those who support more sweeping importation policies decried the plan’s limited scope and suspected the announcement was part theatrics and part a threatening signal to drugmakers.
“This could just be a dog-and-pony show, where they’re calling in an expert group to explore avenues of importation – but when all is said and done, they find that they don’t want to do this,” said Gabriel Levitt, the cofounder of PharmacyChecker.com, a private company that verifies international online pharmacies and compares prescription drug prices for consumers.
“At that point, we’ll learn that the exercise was lip service,” he added. “Frankly, there’s a good chance that that is the case.”
This isn’t the first time officials have suggested importing drugs from other countries to find better prices. Bills have been offered in Congress to allow it, and George W. Bush administration officials investigated the issue and produced two reports questioning the safety of such efforts in 2004.
Overall, the measure is by no means a silver bullet to the larger problem of rising drug prices, said Rachel Sachs, JD, an associate professor of law at Washington University School of Law in St. Louis.
“It’s a really smart move to solve one of the many drug pricing problems we observe, but, of course, it won’t address every problem,” Sachs said.
Mr. Mendelson suggested this working group might be an effort to placate patients who have seen little movement to bring down drug costs, despite the president’s repeated promises to provide help.
“If the goal is to make policy changes that are visible and help with the 2018 and 2020 election, I think it’s right up there with a lot of the things they’re doing,” Mendelson said. “If the goal is truly to help consumers with drug prices, not so much.”
In addition, since the group’s work applies primarily to the generic drug market, a new policy would stop short of taming the price spirals and high launch prices of blockbuster brand-name drugs, which Harvard’s Sarpatwari said were the “elephants in the room” of the drug pricing debate.
Mr. Levitt pointed out that, while a big overnight increase on a drug might trigger action to allow importation, the move would do nothing to stop the yearly increases that drug companies tack on to medicines. Depending on how possible regulations are written, such increases might even be encouraged. The administration has been pressuring pharmaceutical makers to hold down those rising prices but finding tepid support among the companies.
Even if the policy targets just a slice of the overall problem, it could still make a difference for Americans struggling to pay for off-patent drugs and provide more competition.
“If Azar is serious about this proposal, even though it’s very limited in scope, it could help deter the most egregious forms of drug price gouging where there are single-source meds,” Mr. Levitt said.
KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Screening for osteoporosis to prevent fractures: USPSTF recommendation statement
The U.S. Preventive Services Task Force commissioned a systematic evidence review of 168 fair-good quality articles to examine newer evidence on screening for and treatment of osteoporotic fracture in women and men and update its 2011 guideline. For postmenopausal women older than 65 years and those younger than 65 years with increased risk of osteoporosis, USPSTF found convincing evidence that screening can detect osteoporosis and that treatment can provide at least a moderate benefit in preventing fractures (grade B). For men, they report inadequate evidence on the benefits and harms of screening for osteoporosis to reduce the risk of fractures (I statement).
Importance
Osteoporosis leads to increased bone fragility and risk of fractures, specifically hip fractures, that are associated with limitations in ambulation, chronic pain, disability and loss of independence, and decreased quality of life: 21%-30% of those who suffer hip fractures die within 1 year. Osteoporosis is usually asymptomatic until a fracture occurs, thus preventing fractures is the main goal of an osteoporosis screening strategy. With the increasing life expectancy of the U.S. population, the potential preventable burden is likely to increase in future years.
Screening tests
The most commonly used test is central dual energy x-ray absorptiometry (DXA), which provides measurement of bone mineral density (BMD) of the hip and lumbar spine. Most treatment guidelines already use central DXA BMD to define osteoporosis and the threshold at which to start drug therapies for prevention. Other lower-cost and more accessible alternatives include peripheral DXA, which measures BMD at lower forearm and heel, and quantitative ultrasound (QUS), which also evaluates peripheral sites like the calcaneus. QUS does not measure BMD. USPSTF found that the harms associated with screening were small (mainly radiation exposure from DXA and opportunity costs).
Population and risk assessment
The review included adults older than 40 years of age, mostly postmenopausal women, without a history of previous low-trauma fractures, without conditions or medications that may cause secondary osteoporosis, and without increased risk of falls.
Patients at increased risk of osteoporotic fractures include those with parental history of hip fractures, low body weight, excessive alcohol consumption, and smokers. For postmenopausal women younger than 65 years of age with at least one risk factor, a reasonable approach to determine who should be screened with BMD is to use one of the various clinical risk assessment tools available. The most frequently studied tools in women are the Osteoporosis Risk Assessment Instrument (ORAI), Osteoporosis Index of Risk (OSIRIS), Osteoporosis Self-Assessment Tool (OST), and Simple Calculated Osteoporosis Risk Estimation (SCORE). The Fracture Risk Assessment (FRAX) tool calculates the 10-year risk of a major osteoporotic fracture (MOF) using clinical risk factors. For example, one approach is to perform BMD in women younger than 65 years with a FRAX risk greater than 8.4% (the FRAX risk of a 65-year-old woman of mean height and weight without major risk factors).
In men, the prevalence of osteoporosis (4.3%) is generally lower than in women (15.4%). In the absence of other risk factors, it is not till age 80 that the prevalence of osteoporosis in white men starts to reach that of a 65-year-old white woman. While men have similar risk factors as women described above, men with hypogonadism, history of cerebrovascular accident, and history of diabetes are also at increased risk of fracture.
Preventative measures to reduce osteoporotic fractures
Approved drug therapies. The majority of studies were conducted in postmenopausal women. Bisphosphonates, most commonly used and studied, significantly reduced vertebral and nonvertebral fractures but not hip fractures (possibly because of underpowered studies). Raloxifene and parathyroid hormone reduced vertebral fractures but not nonvertebral fractures. Denosumab significantly reduced all three types of fractures. A 2011 review identified that estrogen reduced vertebral fractures, but no new studies were identified for the current review. Data from the Women’s Health Initiative show that women receiving estrogen with or without progesterone had an elevated risk of stroke, venous thromboembolism, and gallbladder disease; their risk for urinary incontinence was increased during the first year of follow-up. In addition, women receiving estrogen plus progestin had a higher risk of invasive breast cancer, coronary heart disease, and probable dementia. The risk of serious adverse events, upper-gastrointestinal events, or cardiovascular events associated with the most common class of medications used, bisphosphonates, is small. Evidence on the effectiveness of medications to treat osteoporosis in men is lacking (only two studies conducted).
Exercise. Engagement in 120-300 minutes of weekly moderate-intensity aerobic activity can reduce the risk of hip fractures, and performance of weekly balance and muscle-strengthening activities can help prevent falls in older adults.
Supplements. In a separate recommendation, USPSTF recommends against daily supplementation with less than 400 IU of vitamin D and less than 1,000 mg of calcium for the primary prevention of fractures in community-dwelling, postmenopausal women. They found insufficient evidence on supplementation with higher doses of vitamin D and calcium in postmenopausal women, or at any dose in men and premenopausal women.
Recommendations from others
The National Osteoporosis Foundation and the International Society for Clinical Densitometry recommend BMD testing in all women older than 65 years, all men over 70 years, postmenopausal women younger than 65 years, and men aged 50-69 years with increased risk factors. The American Academy of Family Physicians recommends against DXA screening in women younger than 65 years and men younger than 70 years with no risk factors.
The bottom line
For all women older than 65 years and postmenopausal women younger than 65 years who are at increased risk, screen for and treat osteoporosis to prevent fractures. For men, there is insufficient evidence to screen.
Dr. Shrestha is a second-year resident in the Family Medicine Residency Program at Abington (Pa.) - Jefferson Health. Dr. Skolnik is a professor of family and community medicine at Jefferson Medical College, Philadelphia, and an associate director of the family medicine residency program at Abington - Jefferson Health.
References
1. U.S. Preventative Services Task Force. JAMA. 2018 Jun 26;319(24):2521-31.
2. U.S. Preventative Services Task Force. JAMA. 2018 Jun 26;319(24):2532-51.
The U.S. Preventive Services Task Force commissioned a systematic evidence review of 168 fair-good quality articles to examine newer evidence on screening for and treatment of osteoporotic fracture in women and men and update its 2011 guideline. For postmenopausal women older than 65 years and those younger than 65 years with increased risk of osteoporosis, USPSTF found convincing evidence that screening can detect osteoporosis and that treatment can provide at least a moderate benefit in preventing fractures (grade B). For men, they report inadequate evidence on the benefits and harms of screening for osteoporosis to reduce the risk of fractures (I statement).
Importance
Osteoporosis leads to increased bone fragility and risk of fractures, specifically hip fractures, that are associated with limitations in ambulation, chronic pain, disability and loss of independence, and decreased quality of life: 21%-30% of those who suffer hip fractures die within 1 year. Osteoporosis is usually asymptomatic until a fracture occurs, thus preventing fractures is the main goal of an osteoporosis screening strategy. With the increasing life expectancy of the U.S. population, the potential preventable burden is likely to increase in future years.
Screening tests
The most commonly used test is central dual energy x-ray absorptiometry (DXA), which provides measurement of bone mineral density (BMD) of the hip and lumbar spine. Most treatment guidelines already use central DXA BMD to define osteoporosis and the threshold at which to start drug therapies for prevention. Other lower-cost and more accessible alternatives include peripheral DXA, which measures BMD at lower forearm and heel, and quantitative ultrasound (QUS), which also evaluates peripheral sites like the calcaneus. QUS does not measure BMD. USPSTF found that the harms associated with screening were small (mainly radiation exposure from DXA and opportunity costs).
Population and risk assessment
The review included adults older than 40 years of age, mostly postmenopausal women, without a history of previous low-trauma fractures, without conditions or medications that may cause secondary osteoporosis, and without increased risk of falls.
Patients at increased risk of osteoporotic fractures include those with parental history of hip fractures, low body weight, excessive alcohol consumption, and smokers. For postmenopausal women younger than 65 years of age with at least one risk factor, a reasonable approach to determine who should be screened with BMD is to use one of the various clinical risk assessment tools available. The most frequently studied tools in women are the Osteoporosis Risk Assessment Instrument (ORAI), Osteoporosis Index of Risk (OSIRIS), Osteoporosis Self-Assessment Tool (OST), and Simple Calculated Osteoporosis Risk Estimation (SCORE). The Fracture Risk Assessment (FRAX) tool calculates the 10-year risk of a major osteoporotic fracture (MOF) using clinical risk factors. For example, one approach is to perform BMD in women younger than 65 years with a FRAX risk greater than 8.4% (the FRAX risk of a 65-year-old woman of mean height and weight without major risk factors).
In men, the prevalence of osteoporosis (4.3%) is generally lower than in women (15.4%). In the absence of other risk factors, it is not till age 80 that the prevalence of osteoporosis in white men starts to reach that of a 65-year-old white woman. While men have similar risk factors as women described above, men with hypogonadism, history of cerebrovascular accident, and history of diabetes are also at increased risk of fracture.
Preventative measures to reduce osteoporotic fractures
Approved drug therapies. The majority of studies were conducted in postmenopausal women. Bisphosphonates, most commonly used and studied, significantly reduced vertebral and nonvertebral fractures but not hip fractures (possibly because of underpowered studies). Raloxifene and parathyroid hormone reduced vertebral fractures but not nonvertebral fractures. Denosumab significantly reduced all three types of fractures. A 2011 review identified that estrogen reduced vertebral fractures, but no new studies were identified for the current review. Data from the Women’s Health Initiative show that women receiving estrogen with or without progesterone had an elevated risk of stroke, venous thromboembolism, and gallbladder disease; their risk for urinary incontinence was increased during the first year of follow-up. In addition, women receiving estrogen plus progestin had a higher risk of invasive breast cancer, coronary heart disease, and probable dementia. The risk of serious adverse events, upper-gastrointestinal events, or cardiovascular events associated with the most common class of medications used, bisphosphonates, is small. Evidence on the effectiveness of medications to treat osteoporosis in men is lacking (only two studies conducted).
Exercise. Engagement in 120-300 minutes of weekly moderate-intensity aerobic activity can reduce the risk of hip fractures, and performance of weekly balance and muscle-strengthening activities can help prevent falls in older adults.
Supplements. In a separate recommendation, USPSTF recommends against daily supplementation with less than 400 IU of vitamin D and less than 1,000 mg of calcium for the primary prevention of fractures in community-dwelling, postmenopausal women. They found insufficient evidence on supplementation with higher doses of vitamin D and calcium in postmenopausal women, or at any dose in men and premenopausal women.
Recommendations from others
The National Osteoporosis Foundation and the International Society for Clinical Densitometry recommend BMD testing in all women older than 65 years, all men over 70 years, postmenopausal women younger than 65 years, and men aged 50-69 years with increased risk factors. The American Academy of Family Physicians recommends against DXA screening in women younger than 65 years and men younger than 70 years with no risk factors.
The bottom line
For all women older than 65 years and postmenopausal women younger than 65 years who are at increased risk, screen for and treat osteoporosis to prevent fractures. For men, there is insufficient evidence to screen.
Dr. Shrestha is a second-year resident in the Family Medicine Residency Program at Abington (Pa.) - Jefferson Health. Dr. Skolnik is a professor of family and community medicine at Jefferson Medical College, Philadelphia, and an associate director of the family medicine residency program at Abington - Jefferson Health.
References
1. U.S. Preventative Services Task Force. JAMA. 2018 Jun 26;319(24):2521-31.
2. U.S. Preventative Services Task Force. JAMA. 2018 Jun 26;319(24):2532-51.
The U.S. Preventive Services Task Force commissioned a systematic evidence review of 168 fair-good quality articles to examine newer evidence on screening for and treatment of osteoporotic fracture in women and men and update its 2011 guideline. For postmenopausal women older than 65 years and those younger than 65 years with increased risk of osteoporosis, USPSTF found convincing evidence that screening can detect osteoporosis and that treatment can provide at least a moderate benefit in preventing fractures (grade B). For men, they report inadequate evidence on the benefits and harms of screening for osteoporosis to reduce the risk of fractures (I statement).
Importance
Osteoporosis leads to increased bone fragility and risk of fractures, specifically hip fractures, that are associated with limitations in ambulation, chronic pain, disability and loss of independence, and decreased quality of life: 21%-30% of those who suffer hip fractures die within 1 year. Osteoporosis is usually asymptomatic until a fracture occurs, thus preventing fractures is the main goal of an osteoporosis screening strategy. With the increasing life expectancy of the U.S. population, the potential preventable burden is likely to increase in future years.
Screening tests
The most commonly used test is central dual energy x-ray absorptiometry (DXA), which provides measurement of bone mineral density (BMD) of the hip and lumbar spine. Most treatment guidelines already use central DXA BMD to define osteoporosis and the threshold at which to start drug therapies for prevention. Other lower-cost and more accessible alternatives include peripheral DXA, which measures BMD at lower forearm and heel, and quantitative ultrasound (QUS), which also evaluates peripheral sites like the calcaneus. QUS does not measure BMD. USPSTF found that the harms associated with screening were small (mainly radiation exposure from DXA and opportunity costs).
Population and risk assessment
The review included adults older than 40 years of age, mostly postmenopausal women, without a history of previous low-trauma fractures, without conditions or medications that may cause secondary osteoporosis, and without increased risk of falls.
Patients at increased risk of osteoporotic fractures include those with parental history of hip fractures, low body weight, excessive alcohol consumption, and smokers. For postmenopausal women younger than 65 years of age with at least one risk factor, a reasonable approach to determine who should be screened with BMD is to use one of the various clinical risk assessment tools available. The most frequently studied tools in women are the Osteoporosis Risk Assessment Instrument (ORAI), Osteoporosis Index of Risk (OSIRIS), Osteoporosis Self-Assessment Tool (OST), and Simple Calculated Osteoporosis Risk Estimation (SCORE). The Fracture Risk Assessment (FRAX) tool calculates the 10-year risk of a major osteoporotic fracture (MOF) using clinical risk factors. For example, one approach is to perform BMD in women younger than 65 years with a FRAX risk greater than 8.4% (the FRAX risk of a 65-year-old woman of mean height and weight without major risk factors).
In men, the prevalence of osteoporosis (4.3%) is generally lower than in women (15.4%). In the absence of other risk factors, it is not till age 80 that the prevalence of osteoporosis in white men starts to reach that of a 65-year-old white woman. While men have similar risk factors as women described above, men with hypogonadism, history of cerebrovascular accident, and history of diabetes are also at increased risk of fracture.
Preventative measures to reduce osteoporotic fractures
Approved drug therapies. The majority of studies were conducted in postmenopausal women. Bisphosphonates, most commonly used and studied, significantly reduced vertebral and nonvertebral fractures but not hip fractures (possibly because of underpowered studies). Raloxifene and parathyroid hormone reduced vertebral fractures but not nonvertebral fractures. Denosumab significantly reduced all three types of fractures. A 2011 review identified that estrogen reduced vertebral fractures, but no new studies were identified for the current review. Data from the Women’s Health Initiative show that women receiving estrogen with or without progesterone had an elevated risk of stroke, venous thromboembolism, and gallbladder disease; their risk for urinary incontinence was increased during the first year of follow-up. In addition, women receiving estrogen plus progestin had a higher risk of invasive breast cancer, coronary heart disease, and probable dementia. The risk of serious adverse events, upper-gastrointestinal events, or cardiovascular events associated with the most common class of medications used, bisphosphonates, is small. Evidence on the effectiveness of medications to treat osteoporosis in men is lacking (only two studies conducted).
Exercise. Engagement in 120-300 minutes of weekly moderate-intensity aerobic activity can reduce the risk of hip fractures, and performance of weekly balance and muscle-strengthening activities can help prevent falls in older adults.
Supplements. In a separate recommendation, USPSTF recommends against daily supplementation with less than 400 IU of vitamin D and less than 1,000 mg of calcium for the primary prevention of fractures in community-dwelling, postmenopausal women. They found insufficient evidence on supplementation with higher doses of vitamin D and calcium in postmenopausal women, or at any dose in men and premenopausal women.
Recommendations from others
The National Osteoporosis Foundation and the International Society for Clinical Densitometry recommend BMD testing in all women older than 65 years, all men over 70 years, postmenopausal women younger than 65 years, and men aged 50-69 years with increased risk factors. The American Academy of Family Physicians recommends against DXA screening in women younger than 65 years and men younger than 70 years with no risk factors.
The bottom line
For all women older than 65 years and postmenopausal women younger than 65 years who are at increased risk, screen for and treat osteoporosis to prevent fractures. For men, there is insufficient evidence to screen.
Dr. Shrestha is a second-year resident in the Family Medicine Residency Program at Abington (Pa.) - Jefferson Health. Dr. Skolnik is a professor of family and community medicine at Jefferson Medical College, Philadelphia, and an associate director of the family medicine residency program at Abington - Jefferson Health.
References
1. U.S. Preventative Services Task Force. JAMA. 2018 Jun 26;319(24):2521-31.
2. U.S. Preventative Services Task Force. JAMA. 2018 Jun 26;319(24):2532-51.
Fitbit Flex is feasible, provides nuanced step count data in MS patients
LOS ANGELES – The commercially available Fitbit Flex accelerometer proved useful and feasible for longitudinal measurement of average daily steps in a prospective, 1-year study of patients with multiple sclerosis.
The wrist-worn device was well received, with 79 of 96 participants (82%) completing follow-up, and it revealed changes in daily functioning that were not captured using more traditional disability metrics, Valerie J. Block, DPTSc, reported at the annual meeting of the American Academy of Neurology.
The study involved 61 adults with relapsing multiple sclerosis (MS) and 35 with progressive MS who were recruited into the University of California, San Francisco (UCSF) FITriMS cohort. All were able to walk at least 2 minutes at baseline, and their daily physical activity was recorded continuously by the device over 1 year. Performance-based measures, including the Expanded Disability Status Scale (EDSS) and timed 25-foot walk test, were evaluated at baseline and at 1 year, and patient-reported outcomes such as the 12-item MS walking scale were completed at baseline and at 1.5, 3, 6, 9, and 12 months. Nine patients withdrew, and eight were lost to follow-up.
“This was a fairly stable, actively treated cohort, and overall, there was no significant change in average daily steps (STEPS) over 1 year. However, there was a trend toward a decrease (–315 steps/day; P = .117), and 53% of patients had a decrease of more than 800 steps per day, which is a proposed minimal clinically important difference threshold,” Dr. Block, a postdoctoral scholar at UCSF, said in an interview.
Despite the modest, gradual reduction in STEPS over the year, most participants were able to complete the study, providing at least 1 valid week of STEPS data per month throughout the year, she said, noting that there was no difference in device use between relapsing MS and progressive MS participants.
A significant association between decreasing STEPS and worsening of clinic-based and patient-reported outcomes was demonstrated, but declining STEPS occurred even when disability levels, as measured by the EDSS, remained stable.
“Participants who started off the study with lower STEPS, below the cohort median of 4,766 STEPS, had fourfold higher odds of clinically-meaningful disability worsening at 1 year after adjusting for age, sex, and disease duration,” she said.
Further, earlier data published by Dr. Block and her colleagues showed there is wide variability in the change in steps over 1 year. In that study, the change in step count in patients whose EDSS score remained unchanged ranged from +814 to –3,718, suggesting that popular mainstream wearable technology is not only feasible but also can provide a more detailed and nuanced measure of how patients are functioning in their daily lives, she said.
Together, these findings suggest that remote accelerometry provides useful continuous information about physical activity in real-world setting, she said, concluding that “these results support the possibility of using STEPS as a more sensitive and granular outcome measure in clinical trials and for targeted interventions in clinical care.”
This study was supported by the National Center for Advancing Translational Sciences. Dr. Block reported having no disclosures.
SOURCE: Block VJ et al. Neurology. 2018 Apr 10;90(15 Suppl):N5.001.
LOS ANGELES – The commercially available Fitbit Flex accelerometer proved useful and feasible for longitudinal measurement of average daily steps in a prospective, 1-year study of patients with multiple sclerosis.
The wrist-worn device was well received, with 79 of 96 participants (82%) completing follow-up, and it revealed changes in daily functioning that were not captured using more traditional disability metrics, Valerie J. Block, DPTSc, reported at the annual meeting of the American Academy of Neurology.
The study involved 61 adults with relapsing multiple sclerosis (MS) and 35 with progressive MS who were recruited into the University of California, San Francisco (UCSF) FITriMS cohort. All were able to walk at least 2 minutes at baseline, and their daily physical activity was recorded continuously by the device over 1 year. Performance-based measures, including the Expanded Disability Status Scale (EDSS) and timed 25-foot walk test, were evaluated at baseline and at 1 year, and patient-reported outcomes such as the 12-item MS walking scale were completed at baseline and at 1.5, 3, 6, 9, and 12 months. Nine patients withdrew, and eight were lost to follow-up.
“This was a fairly stable, actively treated cohort, and overall, there was no significant change in average daily steps (STEPS) over 1 year. However, there was a trend toward a decrease (–315 steps/day; P = .117), and 53% of patients had a decrease of more than 800 steps per day, which is a proposed minimal clinically important difference threshold,” Dr. Block, a postdoctoral scholar at UCSF, said in an interview.
Despite the modest, gradual reduction in STEPS over the year, most participants were able to complete the study, providing at least 1 valid week of STEPS data per month throughout the year, she said, noting that there was no difference in device use between relapsing MS and progressive MS participants.
A significant association between decreasing STEPS and worsening of clinic-based and patient-reported outcomes was demonstrated, but declining STEPS occurred even when disability levels, as measured by the EDSS, remained stable.
“Participants who started off the study with lower STEPS, below the cohort median of 4,766 STEPS, had fourfold higher odds of clinically-meaningful disability worsening at 1 year after adjusting for age, sex, and disease duration,” she said.
Further, earlier data published by Dr. Block and her colleagues showed there is wide variability in the change in steps over 1 year. In that study, the change in step count in patients whose EDSS score remained unchanged ranged from +814 to –3,718, suggesting that popular mainstream wearable technology is not only feasible but also can provide a more detailed and nuanced measure of how patients are functioning in their daily lives, she said.
Together, these findings suggest that remote accelerometry provides useful continuous information about physical activity in real-world setting, she said, concluding that “these results support the possibility of using STEPS as a more sensitive and granular outcome measure in clinical trials and for targeted interventions in clinical care.”
This study was supported by the National Center for Advancing Translational Sciences. Dr. Block reported having no disclosures.
SOURCE: Block VJ et al. Neurology. 2018 Apr 10;90(15 Suppl):N5.001.
LOS ANGELES – The commercially available Fitbit Flex accelerometer proved useful and feasible for longitudinal measurement of average daily steps in a prospective, 1-year study of patients with multiple sclerosis.
The wrist-worn device was well received, with 79 of 96 participants (82%) completing follow-up, and it revealed changes in daily functioning that were not captured using more traditional disability metrics, Valerie J. Block, DPTSc, reported at the annual meeting of the American Academy of Neurology.
The study involved 61 adults with relapsing multiple sclerosis (MS) and 35 with progressive MS who were recruited into the University of California, San Francisco (UCSF) FITriMS cohort. All were able to walk at least 2 minutes at baseline, and their daily physical activity was recorded continuously by the device over 1 year. Performance-based measures, including the Expanded Disability Status Scale (EDSS) and timed 25-foot walk test, were evaluated at baseline and at 1 year, and patient-reported outcomes such as the 12-item MS walking scale were completed at baseline and at 1.5, 3, 6, 9, and 12 months. Nine patients withdrew, and eight were lost to follow-up.
“This was a fairly stable, actively treated cohort, and overall, there was no significant change in average daily steps (STEPS) over 1 year. However, there was a trend toward a decrease (–315 steps/day; P = .117), and 53% of patients had a decrease of more than 800 steps per day, which is a proposed minimal clinically important difference threshold,” Dr. Block, a postdoctoral scholar at UCSF, said in an interview.
Despite the modest, gradual reduction in STEPS over the year, most participants were able to complete the study, providing at least 1 valid week of STEPS data per month throughout the year, she said, noting that there was no difference in device use between relapsing MS and progressive MS participants.
A significant association between decreasing STEPS and worsening of clinic-based and patient-reported outcomes was demonstrated, but declining STEPS occurred even when disability levels, as measured by the EDSS, remained stable.
“Participants who started off the study with lower STEPS, below the cohort median of 4,766 STEPS, had fourfold higher odds of clinically-meaningful disability worsening at 1 year after adjusting for age, sex, and disease duration,” she said.
Further, earlier data published by Dr. Block and her colleagues showed there is wide variability in the change in steps over 1 year. In that study, the change in step count in patients whose EDSS score remained unchanged ranged from +814 to –3,718, suggesting that popular mainstream wearable technology is not only feasible but also can provide a more detailed and nuanced measure of how patients are functioning in their daily lives, she said.
Together, these findings suggest that remote accelerometry provides useful continuous information about physical activity in real-world setting, she said, concluding that “these results support the possibility of using STEPS as a more sensitive and granular outcome measure in clinical trials and for targeted interventions in clinical care.”
This study was supported by the National Center for Advancing Translational Sciences. Dr. Block reported having no disclosures.
SOURCE: Block VJ et al. Neurology. 2018 Apr 10;90(15 Suppl):N5.001.
REPORTING FROM AAN 2018
Key clinical point: Wearable accelerometers like the Fitbit Flex are feasible and useful for daily step counts in MS patients.
Major finding: 79 of 96 participants (82%) completed follow-up,
Study details: A prospective, 1-year study of 96 patients with MS
Disclosures: This study was supported by the National Center for Advancing Translational Sciences. Dr. Block reported having no disclosures.
Source: Block VJ et al. Neurology. 2018 Apr 10;90(15 Suppl):N5.001.
Patients with chronic pain feel caught in an opioid-prescribing debate
It started with a rolled ankle during a routine Army training exercise. Shannon Hubbard never imagined it was the prologue to one of the most debilitating pain conditions known to exist, called complex regional pain syndrome.
The condition causes the nervous system to go haywire, creating pain disproportionate to the actual injury. It also can affect how the body regulates temperature and blood flow.
For Ms. Hubbard, it manifested years ago following surgery on her foot – a common way for it to take hold.
“My leg feels like it’s on fire pretty much all the time. It spreads to different parts of your body,” the 47-year-old veteran said.
Ms. Hubbard props up her leg, careful not to graze it against the kitchen table in her home east of Phoenix. It’s red and swollen, still scarred from an ulcer that landed her in the hospital a few months ago.
“That started as a little blister and 4 days later, it was like the size of a baseball,” she said. “They had to cut it open and then it got infected, and because I have blood flow issues, it doesn’t heal.”
She knows it’s likely to happen again.
“Over the past 3 years, I’ve been prescribed over 60 different medications and combinations; none have even touched the pain,” she said.
Ms. Hubbard said she’s had injections and even traveled across the country for infusions of ketamine, an anesthetic that can be used for pain in extreme cases. Her doctors have discussed amputating her leg because of the frequency of the infections.
“All I can do is manage the pain,” she said. “Opioids have become the best solution.”
For about 9 months, Ms. Hubbard was on a combination of short- and long-acting opioids. She said it gave her enough relief to start leaving the house again and doing physical therapy.
But in April that changed. At her monthly appointment, her pain doctor informed her the dose was being lowered. “They had to take one of the pills away,” she said.
Ms. Hubbard knew the rules were part of Arizona’s new opioid law, which places restrictions on prescribing and limits the maximum dose for most patients. She also knew the law wasn’t supposed to affect her – an existing patient with chronic pain.
She argued with the doctor, without success. “They didn’t indicate there was any medical reason for cutting me back. It was simply because of the pressure of the opioid rules.”
Her dose was lowered from 100 morphine milligram equivalents daily (MME) to 90, the highest dose allowed for many new patients in Arizona. She said her pain has been “terrible” ever since.
“It just hurts,” she said. “I don’t want to walk, I pretty much don’t want to do anything.”
Ms. Hubbard’s condition may be extreme, but her situation isn’t unique. Faced with skyrocketing drug overdoses, states are cracking down on opioid prescribing. Increasingly, some patients with chronic pain like Ms. Hubbard say they are becoming collateral damage.
New limits on prescribing
More than two dozen states have implemented laws or policies limiting opioid prescriptions in some way. The most common is to restrict a patient’s first prescription to a number of pills that should last a week or less. But some states like Arizona have gone further by placing a ceiling on the maximum dose for most patients.
The Arizona Opioid Epidemic Act, the culmination of months of outreach and planning by state health officials, was passed earlier this year with unanimous support.
It started in June 2017, when Arizona Gov. Doug Ducey, a Republican, declared a public health emergency, citing new data, showing that two people were dying every day in the state from opioid overdoses.
He has pledged to come after those responsible for the rising death toll.
“All bad actors will be held accountable – whether they are doctors, manufacturers, or just plain drug dealers,” the governor said in his annual State of the State address, in January 2018.
He cited statistics from one rural county in which four doctors prescribed 6 million pills in a single year, concluding “something has gone terribly, terribly wrong.”
Later in January, Gov. Ducey called a special session of the Arizona legislature and, in less than a week, he signed the Arizona Opioid Epidemic Act into law. He called it the “most comprehensive and thoughtful package any state has passed to address this issue and crisis to date.”
The law expands access to addiction treatment, ramps up oversight of prescribing and protects from prosecution drug users who call 911 to report an overdose, among other things.
Initially, Arizona’s major medical associations cautioned against what they saw as too much interference in clinical practice, especially since opioid prescriptions already were on the decline.
Gov. Ducey’s administration offered assurances that the law would “maintain access for chronic pain sufferers and others who rely on these drugs.” Restrictions would apply to new patients only. Cancer, trauma, end-of-life, and other serious cases were exempt. Ultimately, the medical establishment came out in favor of the law.
Pressure on doctors
Since the law’s passage, some doctors in Arizona report feeling pressure to lower patient doses, even for patients who have been on stable regimens of opioids for years without trouble.
Julian Grove, MD, knows the nuances of Arizona’s new law better than most physicians. A pain physician, Dr. Grove worked with the state on the prescribing rules.
“We moved the needle to a degree so that many patients wouldn’t be as severely affected,” said Dr. Grove, president of the Arizona Pain Society. “But I’ll be the first to say, this has certainly caused a lot of patients problems [and] anxiety.”
“Many people who are prescribing medications have moved to a much more conservative stance and, unfortunately, pain patients are being negatively affected.”
Like many states, Arizona has looked to its prescription-monitoring program as a key tool for tracking overprescribing. State law requires prescribers to check the online database. Report cards are sent out comparing each prescriber to the rest of their cohort. Clinicians consider their scores when deciding how to manage patients’ care, he said.
“A lot of practitioners are reducing opioid medications, not from a clinical perspective, but more from a legal and regulatory perspective for fear of investigation,” Dr. Grove said. “No practitioner wants to be the highest prescriber.”
Arizona’s new prescribing rules don’t apply to board-certified pain specialists like Dr. Grove, who are trained to care for patients with complex chronic pain. But, he said, the reality is that doctors – even pain specialists – were already facing pressure on many fronts to curtail opioids – from the Drug Enforcement Agency to health insurers down to state medical boards.
The new state law has made the reduction of opioids only “more fast and furious,” he said.
Dr. Grove traces the hypervigilance back to guidelines put out by the Centers for Disease Control and Prevention in 2016. The CDC spelled out the risks associated with higher doses of opioids and advised clinicians when starting a patient on opioids to prescribe the lowest effective dosage.
Sally Satel, MD, a psychiatrist and a fellow at the American Enterprise Institute, said those guidelines stipulated the decision to lower a patient’s dose should be decided on a case-by-case basis, not by means of a blanket policy.
“[The guidelines] have been grossly misinterpreted,” Dr. Satel said.
The guidelines were not intended for pain specialists, but rather for primary care physicians, a group that accounted for nearly half of all opioids dispensed from 2007 to 2012.
“There is no mandate to reduce doses on people who have been doing well,” Dr. Satel said.
In the rush to address the nation’s opioid overdose crisis, she said, the CDC’s guidelines have become the model for many regulators and state legislatures. “It’s a very, very unhealthy, deeply chilled environment in which doctors and patients who have chronic pain can no longer work together,” she said.
Dr. Satel called the notion that new prescribing laws will reverse the tide of drug overdose deaths “misguided.”
The rate of opioid prescribing nationally has declined in recent years, though it still soars above the levels of the 1990s. Meanwhile, more people are dying from illicit drugs like heroin and fentanyl than prescription opioids.
In Arizona, more than 1,300 people have died from opioid-related overdoses since June 2017, according to preliminary state numbers. Only a third of those deaths involved just a prescription painkiller.
Heroin is now almost as common as oxycodone in overdose cases in Arizona.
A range of views
Some physicians support the new rules, said Pete Wertheim, executive director of the Arizona Osteopathic Medical Association.
“For some, it has been a welcome relief,” he said. “They feel like it has given them an avenue, a means to confront patients.” Some doctors tell him it’s an opportunity to have a tough conversation with patients they believe to be at risk for addiction or overdose because of the medication.
The organization is striving to educate its members about Arizona’s prescribing rules and the exemptions. But, he said, most doctors now feel the message is clear: “We don’t want you prescribing opioids.”
Long before the law passed, Mr. Wertheim said, physicians were already telling him that they had stopped prescribing, because they “didn’t want the liability.”
He worries the current climate around prescribing will drive doctors out of pain management, especially in rural areas. There’s also a fear that some patients who can’t get prescription pills will try stronger street drugs, said Gerald Harris II, MD, an addiction treatment specialist in Glendale, Ariz.
Dr. Harris said he has seen an increase in referrals from doctors concerned that their patients with chronic pain are addicted to opioids. He receives new patients – almost daily, he said – whose physicians have stopped prescribing altogether.
“Their doctor is afraid and he’s cut them off,” Dr. Harris said. “Unfortunately, a great many patients turn to street heroin and other drugs to self-medicate because they couldn’t get the medications they need.”
Arizona’s Department of Health Services is working to reassure providers and dispel the myths, said Cara M. Christ, MD, who heads the agency and helped design the state’s opioid response. She pointed to the recently launched Opioid Assistance and Referral Line, created to help health care providers with complex cases. The state has also released a set of detailed prescribing guidelines for doctors.
Dr. Christ characterizes this as an “adjustment period” while physicians learn the new rules.
“The intent was never to stop prescribers from utilizing opioids,” she said. “It’s really meant to prevent a future generation from developing opioid use disorder, while not impacting current chronic pain patients.”
Dr. Christ said she just hasn’t heard of many patients losing access to medicine.
It’s still too early to gauge the law’s success, she said, but opioid prescriptions continue to decline in Arizona.
Arizona saw a 33% reduction in the number of opioid prescriptions in April, compared with the same period last year, state data show. Dr. Christ’s agency reports that more people are getting help for addiction: There has been about a 40% increase in hospitals referring patients for behavioral health treatment following an overdose.
Shannon Hubbard, the woman living with complex regional pain syndrome, considers herself fortunate that her doctors didn’t cut back her painkiller dose even more.
“I’m actually kind of lucky that I have such a severe case because at least they can’t say I’m crazy or it’s in my head,” she said.
Ms. Hubbard is well aware that people are dying every day from opioids. One of her family members struggles with heroin addiction, and she’s helping raise his daughter. But she’s adamant that there’s a better way to address the crisis.
“What they are doing is not working. They are having no effect on the guy who is on the street shooting heroin and is really in danger of overdosing,” she said. “Instead, they are hurting people that are actually helped by the drugs.”
This story is part of a partnership that includes KJZZ, NPR, and Kaiser Health News. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
It started with a rolled ankle during a routine Army training exercise. Shannon Hubbard never imagined it was the prologue to one of the most debilitating pain conditions known to exist, called complex regional pain syndrome.
The condition causes the nervous system to go haywire, creating pain disproportionate to the actual injury. It also can affect how the body regulates temperature and blood flow.
For Ms. Hubbard, it manifested years ago following surgery on her foot – a common way for it to take hold.
“My leg feels like it’s on fire pretty much all the time. It spreads to different parts of your body,” the 47-year-old veteran said.
Ms. Hubbard props up her leg, careful not to graze it against the kitchen table in her home east of Phoenix. It’s red and swollen, still scarred from an ulcer that landed her in the hospital a few months ago.
“That started as a little blister and 4 days later, it was like the size of a baseball,” she said. “They had to cut it open and then it got infected, and because I have blood flow issues, it doesn’t heal.”
She knows it’s likely to happen again.
“Over the past 3 years, I’ve been prescribed over 60 different medications and combinations; none have even touched the pain,” she said.
Ms. Hubbard said she’s had injections and even traveled across the country for infusions of ketamine, an anesthetic that can be used for pain in extreme cases. Her doctors have discussed amputating her leg because of the frequency of the infections.
“All I can do is manage the pain,” she said. “Opioids have become the best solution.”
For about 9 months, Ms. Hubbard was on a combination of short- and long-acting opioids. She said it gave her enough relief to start leaving the house again and doing physical therapy.
But in April that changed. At her monthly appointment, her pain doctor informed her the dose was being lowered. “They had to take one of the pills away,” she said.
Ms. Hubbard knew the rules were part of Arizona’s new opioid law, which places restrictions on prescribing and limits the maximum dose for most patients. She also knew the law wasn’t supposed to affect her – an existing patient with chronic pain.
She argued with the doctor, without success. “They didn’t indicate there was any medical reason for cutting me back. It was simply because of the pressure of the opioid rules.”
Her dose was lowered from 100 morphine milligram equivalents daily (MME) to 90, the highest dose allowed for many new patients in Arizona. She said her pain has been “terrible” ever since.
“It just hurts,” she said. “I don’t want to walk, I pretty much don’t want to do anything.”
Ms. Hubbard’s condition may be extreme, but her situation isn’t unique. Faced with skyrocketing drug overdoses, states are cracking down on opioid prescribing. Increasingly, some patients with chronic pain like Ms. Hubbard say they are becoming collateral damage.
New limits on prescribing
More than two dozen states have implemented laws or policies limiting opioid prescriptions in some way. The most common is to restrict a patient’s first prescription to a number of pills that should last a week or less. But some states like Arizona have gone further by placing a ceiling on the maximum dose for most patients.
The Arizona Opioid Epidemic Act, the culmination of months of outreach and planning by state health officials, was passed earlier this year with unanimous support.
It started in June 2017, when Arizona Gov. Doug Ducey, a Republican, declared a public health emergency, citing new data, showing that two people were dying every day in the state from opioid overdoses.
He has pledged to come after those responsible for the rising death toll.
“All bad actors will be held accountable – whether they are doctors, manufacturers, or just plain drug dealers,” the governor said in his annual State of the State address, in January 2018.
He cited statistics from one rural county in which four doctors prescribed 6 million pills in a single year, concluding “something has gone terribly, terribly wrong.”
Later in January, Gov. Ducey called a special session of the Arizona legislature and, in less than a week, he signed the Arizona Opioid Epidemic Act into law. He called it the “most comprehensive and thoughtful package any state has passed to address this issue and crisis to date.”
The law expands access to addiction treatment, ramps up oversight of prescribing and protects from prosecution drug users who call 911 to report an overdose, among other things.
Initially, Arizona’s major medical associations cautioned against what they saw as too much interference in clinical practice, especially since opioid prescriptions already were on the decline.
Gov. Ducey’s administration offered assurances that the law would “maintain access for chronic pain sufferers and others who rely on these drugs.” Restrictions would apply to new patients only. Cancer, trauma, end-of-life, and other serious cases were exempt. Ultimately, the medical establishment came out in favor of the law.
Pressure on doctors
Since the law’s passage, some doctors in Arizona report feeling pressure to lower patient doses, even for patients who have been on stable regimens of opioids for years without trouble.
Julian Grove, MD, knows the nuances of Arizona’s new law better than most physicians. A pain physician, Dr. Grove worked with the state on the prescribing rules.
“We moved the needle to a degree so that many patients wouldn’t be as severely affected,” said Dr. Grove, president of the Arizona Pain Society. “But I’ll be the first to say, this has certainly caused a lot of patients problems [and] anxiety.”
“Many people who are prescribing medications have moved to a much more conservative stance and, unfortunately, pain patients are being negatively affected.”
Like many states, Arizona has looked to its prescription-monitoring program as a key tool for tracking overprescribing. State law requires prescribers to check the online database. Report cards are sent out comparing each prescriber to the rest of their cohort. Clinicians consider their scores when deciding how to manage patients’ care, he said.
“A lot of practitioners are reducing opioid medications, not from a clinical perspective, but more from a legal and regulatory perspective for fear of investigation,” Dr. Grove said. “No practitioner wants to be the highest prescriber.”
Arizona’s new prescribing rules don’t apply to board-certified pain specialists like Dr. Grove, who are trained to care for patients with complex chronic pain. But, he said, the reality is that doctors – even pain specialists – were already facing pressure on many fronts to curtail opioids – from the Drug Enforcement Agency to health insurers down to state medical boards.
The new state law has made the reduction of opioids only “more fast and furious,” he said.
Dr. Grove traces the hypervigilance back to guidelines put out by the Centers for Disease Control and Prevention in 2016. The CDC spelled out the risks associated with higher doses of opioids and advised clinicians when starting a patient on opioids to prescribe the lowest effective dosage.
Sally Satel, MD, a psychiatrist and a fellow at the American Enterprise Institute, said those guidelines stipulated the decision to lower a patient’s dose should be decided on a case-by-case basis, not by means of a blanket policy.
“[The guidelines] have been grossly misinterpreted,” Dr. Satel said.
The guidelines were not intended for pain specialists, but rather for primary care physicians, a group that accounted for nearly half of all opioids dispensed from 2007 to 2012.
“There is no mandate to reduce doses on people who have been doing well,” Dr. Satel said.
In the rush to address the nation’s opioid overdose crisis, she said, the CDC’s guidelines have become the model for many regulators and state legislatures. “It’s a very, very unhealthy, deeply chilled environment in which doctors and patients who have chronic pain can no longer work together,” she said.
Dr. Satel called the notion that new prescribing laws will reverse the tide of drug overdose deaths “misguided.”
The rate of opioid prescribing nationally has declined in recent years, though it still soars above the levels of the 1990s. Meanwhile, more people are dying from illicit drugs like heroin and fentanyl than prescription opioids.
In Arizona, more than 1,300 people have died from opioid-related overdoses since June 2017, according to preliminary state numbers. Only a third of those deaths involved just a prescription painkiller.
Heroin is now almost as common as oxycodone in overdose cases in Arizona.
A range of views
Some physicians support the new rules, said Pete Wertheim, executive director of the Arizona Osteopathic Medical Association.
“For some, it has been a welcome relief,” he said. “They feel like it has given them an avenue, a means to confront patients.” Some doctors tell him it’s an opportunity to have a tough conversation with patients they believe to be at risk for addiction or overdose because of the medication.
The organization is striving to educate its members about Arizona’s prescribing rules and the exemptions. But, he said, most doctors now feel the message is clear: “We don’t want you prescribing opioids.”
Long before the law passed, Mr. Wertheim said, physicians were already telling him that they had stopped prescribing, because they “didn’t want the liability.”
He worries the current climate around prescribing will drive doctors out of pain management, especially in rural areas. There’s also a fear that some patients who can’t get prescription pills will try stronger street drugs, said Gerald Harris II, MD, an addiction treatment specialist in Glendale, Ariz.
Dr. Harris said he has seen an increase in referrals from doctors concerned that their patients with chronic pain are addicted to opioids. He receives new patients – almost daily, he said – whose physicians have stopped prescribing altogether.
“Their doctor is afraid and he’s cut them off,” Dr. Harris said. “Unfortunately, a great many patients turn to street heroin and other drugs to self-medicate because they couldn’t get the medications they need.”
Arizona’s Department of Health Services is working to reassure providers and dispel the myths, said Cara M. Christ, MD, who heads the agency and helped design the state’s opioid response. She pointed to the recently launched Opioid Assistance and Referral Line, created to help health care providers with complex cases. The state has also released a set of detailed prescribing guidelines for doctors.
Dr. Christ characterizes this as an “adjustment period” while physicians learn the new rules.
“The intent was never to stop prescribers from utilizing opioids,” she said. “It’s really meant to prevent a future generation from developing opioid use disorder, while not impacting current chronic pain patients.”
Dr. Christ said she just hasn’t heard of many patients losing access to medicine.
It’s still too early to gauge the law’s success, she said, but opioid prescriptions continue to decline in Arizona.
Arizona saw a 33% reduction in the number of opioid prescriptions in April, compared with the same period last year, state data show. Dr. Christ’s agency reports that more people are getting help for addiction: There has been about a 40% increase in hospitals referring patients for behavioral health treatment following an overdose.
Shannon Hubbard, the woman living with complex regional pain syndrome, considers herself fortunate that her doctors didn’t cut back her painkiller dose even more.
“I’m actually kind of lucky that I have such a severe case because at least they can’t say I’m crazy or it’s in my head,” she said.
Ms. Hubbard is well aware that people are dying every day from opioids. One of her family members struggles with heroin addiction, and she’s helping raise his daughter. But she’s adamant that there’s a better way to address the crisis.
“What they are doing is not working. They are having no effect on the guy who is on the street shooting heroin and is really in danger of overdosing,” she said. “Instead, they are hurting people that are actually helped by the drugs.”
This story is part of a partnership that includes KJZZ, NPR, and Kaiser Health News. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
It started with a rolled ankle during a routine Army training exercise. Shannon Hubbard never imagined it was the prologue to one of the most debilitating pain conditions known to exist, called complex regional pain syndrome.
The condition causes the nervous system to go haywire, creating pain disproportionate to the actual injury. It also can affect how the body regulates temperature and blood flow.
For Ms. Hubbard, it manifested years ago following surgery on her foot – a common way for it to take hold.
“My leg feels like it’s on fire pretty much all the time. It spreads to different parts of your body,” the 47-year-old veteran said.
Ms. Hubbard props up her leg, careful not to graze it against the kitchen table in her home east of Phoenix. It’s red and swollen, still scarred from an ulcer that landed her in the hospital a few months ago.
“That started as a little blister and 4 days later, it was like the size of a baseball,” she said. “They had to cut it open and then it got infected, and because I have blood flow issues, it doesn’t heal.”
She knows it’s likely to happen again.
“Over the past 3 years, I’ve been prescribed over 60 different medications and combinations; none have even touched the pain,” she said.
Ms. Hubbard said she’s had injections and even traveled across the country for infusions of ketamine, an anesthetic that can be used for pain in extreme cases. Her doctors have discussed amputating her leg because of the frequency of the infections.
“All I can do is manage the pain,” she said. “Opioids have become the best solution.”
For about 9 months, Ms. Hubbard was on a combination of short- and long-acting opioids. She said it gave her enough relief to start leaving the house again and doing physical therapy.
But in April that changed. At her monthly appointment, her pain doctor informed her the dose was being lowered. “They had to take one of the pills away,” she said.
Ms. Hubbard knew the rules were part of Arizona’s new opioid law, which places restrictions on prescribing and limits the maximum dose for most patients. She also knew the law wasn’t supposed to affect her – an existing patient with chronic pain.
She argued with the doctor, without success. “They didn’t indicate there was any medical reason for cutting me back. It was simply because of the pressure of the opioid rules.”
Her dose was lowered from 100 morphine milligram equivalents daily (MME) to 90, the highest dose allowed for many new patients in Arizona. She said her pain has been “terrible” ever since.
“It just hurts,” she said. “I don’t want to walk, I pretty much don’t want to do anything.”
Ms. Hubbard’s condition may be extreme, but her situation isn’t unique. Faced with skyrocketing drug overdoses, states are cracking down on opioid prescribing. Increasingly, some patients with chronic pain like Ms. Hubbard say they are becoming collateral damage.
New limits on prescribing
More than two dozen states have implemented laws or policies limiting opioid prescriptions in some way. The most common is to restrict a patient’s first prescription to a number of pills that should last a week or less. But some states like Arizona have gone further by placing a ceiling on the maximum dose for most patients.
The Arizona Opioid Epidemic Act, the culmination of months of outreach and planning by state health officials, was passed earlier this year with unanimous support.
It started in June 2017, when Arizona Gov. Doug Ducey, a Republican, declared a public health emergency, citing new data, showing that two people were dying every day in the state from opioid overdoses.
He has pledged to come after those responsible for the rising death toll.
“All bad actors will be held accountable – whether they are doctors, manufacturers, or just plain drug dealers,” the governor said in his annual State of the State address, in January 2018.
He cited statistics from one rural county in which four doctors prescribed 6 million pills in a single year, concluding “something has gone terribly, terribly wrong.”
Later in January, Gov. Ducey called a special session of the Arizona legislature and, in less than a week, he signed the Arizona Opioid Epidemic Act into law. He called it the “most comprehensive and thoughtful package any state has passed to address this issue and crisis to date.”
The law expands access to addiction treatment, ramps up oversight of prescribing and protects from prosecution drug users who call 911 to report an overdose, among other things.
Initially, Arizona’s major medical associations cautioned against what they saw as too much interference in clinical practice, especially since opioid prescriptions already were on the decline.
Gov. Ducey’s administration offered assurances that the law would “maintain access for chronic pain sufferers and others who rely on these drugs.” Restrictions would apply to new patients only. Cancer, trauma, end-of-life, and other serious cases were exempt. Ultimately, the medical establishment came out in favor of the law.
Pressure on doctors
Since the law’s passage, some doctors in Arizona report feeling pressure to lower patient doses, even for patients who have been on stable regimens of opioids for years without trouble.
Julian Grove, MD, knows the nuances of Arizona’s new law better than most physicians. A pain physician, Dr. Grove worked with the state on the prescribing rules.
“We moved the needle to a degree so that many patients wouldn’t be as severely affected,” said Dr. Grove, president of the Arizona Pain Society. “But I’ll be the first to say, this has certainly caused a lot of patients problems [and] anxiety.”
“Many people who are prescribing medications have moved to a much more conservative stance and, unfortunately, pain patients are being negatively affected.”
Like many states, Arizona has looked to its prescription-monitoring program as a key tool for tracking overprescribing. State law requires prescribers to check the online database. Report cards are sent out comparing each prescriber to the rest of their cohort. Clinicians consider their scores when deciding how to manage patients’ care, he said.
“A lot of practitioners are reducing opioid medications, not from a clinical perspective, but more from a legal and regulatory perspective for fear of investigation,” Dr. Grove said. “No practitioner wants to be the highest prescriber.”
Arizona’s new prescribing rules don’t apply to board-certified pain specialists like Dr. Grove, who are trained to care for patients with complex chronic pain. But, he said, the reality is that doctors – even pain specialists – were already facing pressure on many fronts to curtail opioids – from the Drug Enforcement Agency to health insurers down to state medical boards.
The new state law has made the reduction of opioids only “more fast and furious,” he said.
Dr. Grove traces the hypervigilance back to guidelines put out by the Centers for Disease Control and Prevention in 2016. The CDC spelled out the risks associated with higher doses of opioids and advised clinicians when starting a patient on opioids to prescribe the lowest effective dosage.
Sally Satel, MD, a psychiatrist and a fellow at the American Enterprise Institute, said those guidelines stipulated the decision to lower a patient’s dose should be decided on a case-by-case basis, not by means of a blanket policy.
“[The guidelines] have been grossly misinterpreted,” Dr. Satel said.
The guidelines were not intended for pain specialists, but rather for primary care physicians, a group that accounted for nearly half of all opioids dispensed from 2007 to 2012.
“There is no mandate to reduce doses on people who have been doing well,” Dr. Satel said.
In the rush to address the nation’s opioid overdose crisis, she said, the CDC’s guidelines have become the model for many regulators and state legislatures. “It’s a very, very unhealthy, deeply chilled environment in which doctors and patients who have chronic pain can no longer work together,” she said.
Dr. Satel called the notion that new prescribing laws will reverse the tide of drug overdose deaths “misguided.”
The rate of opioid prescribing nationally has declined in recent years, though it still soars above the levels of the 1990s. Meanwhile, more people are dying from illicit drugs like heroin and fentanyl than prescription opioids.
In Arizona, more than 1,300 people have died from opioid-related overdoses since June 2017, according to preliminary state numbers. Only a third of those deaths involved just a prescription painkiller.
Heroin is now almost as common as oxycodone in overdose cases in Arizona.
A range of views
Some physicians support the new rules, said Pete Wertheim, executive director of the Arizona Osteopathic Medical Association.
“For some, it has been a welcome relief,” he said. “They feel like it has given them an avenue, a means to confront patients.” Some doctors tell him it’s an opportunity to have a tough conversation with patients they believe to be at risk for addiction or overdose because of the medication.
The organization is striving to educate its members about Arizona’s prescribing rules and the exemptions. But, he said, most doctors now feel the message is clear: “We don’t want you prescribing opioids.”
Long before the law passed, Mr. Wertheim said, physicians were already telling him that they had stopped prescribing, because they “didn’t want the liability.”
He worries the current climate around prescribing will drive doctors out of pain management, especially in rural areas. There’s also a fear that some patients who can’t get prescription pills will try stronger street drugs, said Gerald Harris II, MD, an addiction treatment specialist in Glendale, Ariz.
Dr. Harris said he has seen an increase in referrals from doctors concerned that their patients with chronic pain are addicted to opioids. He receives new patients – almost daily, he said – whose physicians have stopped prescribing altogether.
“Their doctor is afraid and he’s cut them off,” Dr. Harris said. “Unfortunately, a great many patients turn to street heroin and other drugs to self-medicate because they couldn’t get the medications they need.”
Arizona’s Department of Health Services is working to reassure providers and dispel the myths, said Cara M. Christ, MD, who heads the agency and helped design the state’s opioid response. She pointed to the recently launched Opioid Assistance and Referral Line, created to help health care providers with complex cases. The state has also released a set of detailed prescribing guidelines for doctors.
Dr. Christ characterizes this as an “adjustment period” while physicians learn the new rules.
“The intent was never to stop prescribers from utilizing opioids,” she said. “It’s really meant to prevent a future generation from developing opioid use disorder, while not impacting current chronic pain patients.”
Dr. Christ said she just hasn’t heard of many patients losing access to medicine.
It’s still too early to gauge the law’s success, she said, but opioid prescriptions continue to decline in Arizona.
Arizona saw a 33% reduction in the number of opioid prescriptions in April, compared with the same period last year, state data show. Dr. Christ’s agency reports that more people are getting help for addiction: There has been about a 40% increase in hospitals referring patients for behavioral health treatment following an overdose.
Shannon Hubbard, the woman living with complex regional pain syndrome, considers herself fortunate that her doctors didn’t cut back her painkiller dose even more.
“I’m actually kind of lucky that I have such a severe case because at least they can’t say I’m crazy or it’s in my head,” she said.
Ms. Hubbard is well aware that people are dying every day from opioids. One of her family members struggles with heroin addiction, and she’s helping raise his daughter. But she’s adamant that there’s a better way to address the crisis.
“What they are doing is not working. They are having no effect on the guy who is on the street shooting heroin and is really in danger of overdosing,” she said. “Instead, they are hurting people that are actually helped by the drugs.”
This story is part of a partnership that includes KJZZ, NPR, and Kaiser Health News. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
FDA rejects mepolizumab on efficacy, but supports safety for COPD
Asthma drug mepolizumab could be added safely to inhaled corticosteroids for maintenance therapy to help reduce exacerbations in chronic obstructive pulmonary disease (COPD) patients who meet criteria for eosinophil counts, but the current data do not support its efficacy strongly enough for approval, according to a majority of members of the Food and Drug Administration’s Pulmonary-Allergy Drugs Advisory Committee.
The committee voted 16-3 that there was insufficient evidence of efficacy to support guided by eosinophil levels; they also voted 16-3 that the risk-benefit profile was not adequate to support approval.
However, on a voting question of safety, the committee voted 17-2 that the safety data on mepolizumab were sufficient to support approval.
Mepolizumab, a humanized monoclonal antibody, is currently approved for the treatment of asthma with eosinophilic phenotype for patients aged 12 years and older and for adults with eosinophilic granulomatosis with polyangiitis. Manufacturer GlaxoSmithKline is seeking approval for its use as an add-on therapy in COPD patients at a subcutaneous dose of 100 mg every 4 weeks. Mepolizumab works by binding to interleukin-5 (IL-5) and reducing eosinophil maturation and survival, which prompted GlaxoSmithKline to pursue an indication for COPD patients in a high-eosinophil stratum.
The application was supported in part by two concurrent randomized trials of 52 weeks’ duration.
Banu A. Karimi-Shah, MD, clinical team leader of the FDA’s Division of Pulmonary, Allergy, and Rheumatology Products, presented data from the two studies, referred to as Study 106 and Study 113.
In Study 106, researchers found statistically significant reductions in exacerbations for patients in the highest eosinophil group. However, challenges of the studies included a lack of consensus over the definition and possible relevance of an eosinophilic COPD phenotype, Dr. Karimi-Shah said in a presentation at the meeting.
In Study 113, mepolizumab had no significant impact on reducing moderate to severe exacerbations at either a 100-mg or 300-mg dose, Dr. Karimi-Shah said. In addition, most secondary endpoints, with the exception of reducing time to the first exacerbation among patients in the highest eosinophil group, did not consistently support the primary endpoint of exacerbation reduction in either study, she said.
Robert Busch, MD, also of the FDA’s Division of Pulmonary, Allergy, and Rheumatology products, served as a clinical reviewer and presented data on safety, efficacy, and risk-benefit profile of mepolizumab.
Dr. Busch noted that the variability in blood eosinophils make it challenging to use as a potential marker to identify patients who would benefit from mepolizumab as an add-on therapy.
Overall, most of the committee agreed on the existence of an eosinophilic COPD phenotype, but expressed concern about the threshold being used.
“The studies were not particularly well controlled regarding the characterization of patients,” said William J. Calhoun, MD, of the University of Texas Medical Branch, Galveston, who cast one of the ‘no’ votes on the question of efficacy.
By contrast, Jeffrey S. Wagener, MD, of the University of Colorado at Denver, Aurora, referenced his background in cystic fibrosis, and voted “yes” on the question of efficacy. “For patients that have no other option, this is a step forward,” he said.
Committee members on both sides of the vote emphasized the need for more research with larger numbers, better patient characterization, and more female patients. The committee members reported no relevant conflicts of interest.
Asthma drug mepolizumab could be added safely to inhaled corticosteroids for maintenance therapy to help reduce exacerbations in chronic obstructive pulmonary disease (COPD) patients who meet criteria for eosinophil counts, but the current data do not support its efficacy strongly enough for approval, according to a majority of members of the Food and Drug Administration’s Pulmonary-Allergy Drugs Advisory Committee.
The committee voted 16-3 that there was insufficient evidence of efficacy to support guided by eosinophil levels; they also voted 16-3 that the risk-benefit profile was not adequate to support approval.
However, on a voting question of safety, the committee voted 17-2 that the safety data on mepolizumab were sufficient to support approval.
Mepolizumab, a humanized monoclonal antibody, is currently approved for the treatment of asthma with eosinophilic phenotype for patients aged 12 years and older and for adults with eosinophilic granulomatosis with polyangiitis. Manufacturer GlaxoSmithKline is seeking approval for its use as an add-on therapy in COPD patients at a subcutaneous dose of 100 mg every 4 weeks. Mepolizumab works by binding to interleukin-5 (IL-5) and reducing eosinophil maturation and survival, which prompted GlaxoSmithKline to pursue an indication for COPD patients in a high-eosinophil stratum.
The application was supported in part by two concurrent randomized trials of 52 weeks’ duration.
Banu A. Karimi-Shah, MD, clinical team leader of the FDA’s Division of Pulmonary, Allergy, and Rheumatology Products, presented data from the two studies, referred to as Study 106 and Study 113.
In Study 106, researchers found statistically significant reductions in exacerbations for patients in the highest eosinophil group. However, challenges of the studies included a lack of consensus over the definition and possible relevance of an eosinophilic COPD phenotype, Dr. Karimi-Shah said in a presentation at the meeting.
In Study 113, mepolizumab had no significant impact on reducing moderate to severe exacerbations at either a 100-mg or 300-mg dose, Dr. Karimi-Shah said. In addition, most secondary endpoints, with the exception of reducing time to the first exacerbation among patients in the highest eosinophil group, did not consistently support the primary endpoint of exacerbation reduction in either study, she said.
Robert Busch, MD, also of the FDA’s Division of Pulmonary, Allergy, and Rheumatology products, served as a clinical reviewer and presented data on safety, efficacy, and risk-benefit profile of mepolizumab.
Dr. Busch noted that the variability in blood eosinophils make it challenging to use as a potential marker to identify patients who would benefit from mepolizumab as an add-on therapy.
Overall, most of the committee agreed on the existence of an eosinophilic COPD phenotype, but expressed concern about the threshold being used.
“The studies were not particularly well controlled regarding the characterization of patients,” said William J. Calhoun, MD, of the University of Texas Medical Branch, Galveston, who cast one of the ‘no’ votes on the question of efficacy.
By contrast, Jeffrey S. Wagener, MD, of the University of Colorado at Denver, Aurora, referenced his background in cystic fibrosis, and voted “yes” on the question of efficacy. “For patients that have no other option, this is a step forward,” he said.
Committee members on both sides of the vote emphasized the need for more research with larger numbers, better patient characterization, and more female patients. The committee members reported no relevant conflicts of interest.
Asthma drug mepolizumab could be added safely to inhaled corticosteroids for maintenance therapy to help reduce exacerbations in chronic obstructive pulmonary disease (COPD) patients who meet criteria for eosinophil counts, but the current data do not support its efficacy strongly enough for approval, according to a majority of members of the Food and Drug Administration’s Pulmonary-Allergy Drugs Advisory Committee.
The committee voted 16-3 that there was insufficient evidence of efficacy to support guided by eosinophil levels; they also voted 16-3 that the risk-benefit profile was not adequate to support approval.
However, on a voting question of safety, the committee voted 17-2 that the safety data on mepolizumab were sufficient to support approval.
Mepolizumab, a humanized monoclonal antibody, is currently approved for the treatment of asthma with eosinophilic phenotype for patients aged 12 years and older and for adults with eosinophilic granulomatosis with polyangiitis. Manufacturer GlaxoSmithKline is seeking approval for its use as an add-on therapy in COPD patients at a subcutaneous dose of 100 mg every 4 weeks. Mepolizumab works by binding to interleukin-5 (IL-5) and reducing eosinophil maturation and survival, which prompted GlaxoSmithKline to pursue an indication for COPD patients in a high-eosinophil stratum.
The application was supported in part by two concurrent randomized trials of 52 weeks’ duration.
Banu A. Karimi-Shah, MD, clinical team leader of the FDA’s Division of Pulmonary, Allergy, and Rheumatology Products, presented data from the two studies, referred to as Study 106 and Study 113.
In Study 106, researchers found statistically significant reductions in exacerbations for patients in the highest eosinophil group. However, challenges of the studies included a lack of consensus over the definition and possible relevance of an eosinophilic COPD phenotype, Dr. Karimi-Shah said in a presentation at the meeting.
In Study 113, mepolizumab had no significant impact on reducing moderate to severe exacerbations at either a 100-mg or 300-mg dose, Dr. Karimi-Shah said. In addition, most secondary endpoints, with the exception of reducing time to the first exacerbation among patients in the highest eosinophil group, did not consistently support the primary endpoint of exacerbation reduction in either study, she said.
Robert Busch, MD, also of the FDA’s Division of Pulmonary, Allergy, and Rheumatology products, served as a clinical reviewer and presented data on safety, efficacy, and risk-benefit profile of mepolizumab.
Dr. Busch noted that the variability in blood eosinophils make it challenging to use as a potential marker to identify patients who would benefit from mepolizumab as an add-on therapy.
Overall, most of the committee agreed on the existence of an eosinophilic COPD phenotype, but expressed concern about the threshold being used.
“The studies were not particularly well controlled regarding the characterization of patients,” said William J. Calhoun, MD, of the University of Texas Medical Branch, Galveston, who cast one of the ‘no’ votes on the question of efficacy.
By contrast, Jeffrey S. Wagener, MD, of the University of Colorado at Denver, Aurora, referenced his background in cystic fibrosis, and voted “yes” on the question of efficacy. “For patients that have no other option, this is a step forward,” he said.
Committee members on both sides of the vote emphasized the need for more research with larger numbers, better patient characterization, and more female patients. The committee members reported no relevant conflicts of interest.
FROM AN FDA ADVISORY COMMITTEE MEETING
ED key to reducing pediatric asthma x-rays
ATLANTA – but accomplishing this goal takes more than a new clinical practice guideline, according to a quality improvement team at the Monroe Carell Jr. Children’s Hospital at Vanderbilt University, Nashville, Tenn.
The team eventually reduced the chest x-ray rate for pediatric asthma exacerbations from 30% to 15% without increasing 3-day all-cause readmissions, but it took some sleuthing in the ED and good relations with staff. “We were way out in left field when we started this. Working in silos is never ideal,” said senior project member David Johnson, MD, a pediatric hospitalist and assistant professor of pediatrics at Vanderbilt.
It’s been known for a while that chest x-rays are almost always a waste of time and money for asthma exacerbations, and national guidelines recommend against them. X-rays don’t improve outcomes and needlessly expose children to radiation.
In 2014, some of the providers at Vanderbilt, which has about 1,700 asthma encounters a year, realized that the institution’s 30% x-ray rate was a problem. The quality improvement team hoped a new guideline would address the issue, but that didn’t happen. “We roll out clinical practice guidelines” from on high, “and think people will magically change their behavior,” but they don’t, Dr. Johnson said at the annual Pediatric Hospital Medicine meeting.
The guideline was not being fully implemented. So the team asked the ED what was the standard procedure for a child presenting with asthma exacerbation. It turned out that the ED had a dyspnea order set that the team ”had no idea existed.” Chest x-rays were at the top of the list; next came blood gases, ventilation-perfusion scans, and leg Dopplers, he said.
The investigators tried to get rid of the whole order set but were unsuccessful. The ED department did, however, let the team eliminate chest x-rays in the default order set in July 2015. That helped, but more changes were needed.
The next conversation was to figure out why x-rays were being ordered in the first place. ED staff said they were worried about missing something, especially pneumonia. They also thought they were helping hospitalists by getting x-rays before sending kids to the ward even though, in reality, it didn’t matter whether x-rays were done a few hours later on the floor. ED providers also said that ill-appearing children often got better after a few hours but were kept back from discharge because x-ray results were still pending and that sometimes these results revealed problems at 3 a.m. that had nothing to do with why the patients were in the ED but still required a work-up.
This discussion opened a door. The ED staff didn’t want to order unnecessary x-rays, either. That led to talks about letting kids declare themselves a bit before x-rays were ordered. ED staff liked the idea, so the guidelines were updated in early 2016 to say that chest x-rays should only be ordered if there is persistent severe respiratory distress with hypoxia, there are focal findings that don’t improve after 12 hours of treatment, or there were concerns for pneumomediastinum or collapsed lung. The updated guidelines were posted in work areas and brought home by resident education. A reminder was added to the electronic medical record system that popped up when someone tried to order a chest x-ray for an child with asthma.
It worked. Chest x-ray rates in asthma fell to 15%, and have remained there since.
“We gave them permission to take their foot off the throttle and wait a little bit, and we don’t have more kids bouncing back from reduced x-rays.” The approach is “probably generalizable everywhere,” Dr. Johnson said.
It was essential that an ED fellow, Caroline Watnick, MD, led the effort and eventually bridged the gap between hospitalists and ED providers. In the end, “the change wasn’t something from the outside,” Dr. Johnson said.
There was no industry funding, and Dr. Johnson didn’t have any disclosures. The Pediatric Hospital Medicine meeting is sponsored by the Society of Hospital Medicine, the American Academy of Pediatrics, and the Academic Pediatric Association.
ATLANTA – but accomplishing this goal takes more than a new clinical practice guideline, according to a quality improvement team at the Monroe Carell Jr. Children’s Hospital at Vanderbilt University, Nashville, Tenn.
The team eventually reduced the chest x-ray rate for pediatric asthma exacerbations from 30% to 15% without increasing 3-day all-cause readmissions, but it took some sleuthing in the ED and good relations with staff. “We were way out in left field when we started this. Working in silos is never ideal,” said senior project member David Johnson, MD, a pediatric hospitalist and assistant professor of pediatrics at Vanderbilt.
It’s been known for a while that chest x-rays are almost always a waste of time and money for asthma exacerbations, and national guidelines recommend against them. X-rays don’t improve outcomes and needlessly expose children to radiation.
In 2014, some of the providers at Vanderbilt, which has about 1,700 asthma encounters a year, realized that the institution’s 30% x-ray rate was a problem. The quality improvement team hoped a new guideline would address the issue, but that didn’t happen. “We roll out clinical practice guidelines” from on high, “and think people will magically change their behavior,” but they don’t, Dr. Johnson said at the annual Pediatric Hospital Medicine meeting.
The guideline was not being fully implemented. So the team asked the ED what was the standard procedure for a child presenting with asthma exacerbation. It turned out that the ED had a dyspnea order set that the team ”had no idea existed.” Chest x-rays were at the top of the list; next came blood gases, ventilation-perfusion scans, and leg Dopplers, he said.
The investigators tried to get rid of the whole order set but were unsuccessful. The ED department did, however, let the team eliminate chest x-rays in the default order set in July 2015. That helped, but more changes were needed.
The next conversation was to figure out why x-rays were being ordered in the first place. ED staff said they were worried about missing something, especially pneumonia. They also thought they were helping hospitalists by getting x-rays before sending kids to the ward even though, in reality, it didn’t matter whether x-rays were done a few hours later on the floor. ED providers also said that ill-appearing children often got better after a few hours but were kept back from discharge because x-ray results were still pending and that sometimes these results revealed problems at 3 a.m. that had nothing to do with why the patients were in the ED but still required a work-up.
This discussion opened a door. The ED staff didn’t want to order unnecessary x-rays, either. That led to talks about letting kids declare themselves a bit before x-rays were ordered. ED staff liked the idea, so the guidelines were updated in early 2016 to say that chest x-rays should only be ordered if there is persistent severe respiratory distress with hypoxia, there are focal findings that don’t improve after 12 hours of treatment, or there were concerns for pneumomediastinum or collapsed lung. The updated guidelines were posted in work areas and brought home by resident education. A reminder was added to the electronic medical record system that popped up when someone tried to order a chest x-ray for an child with asthma.
It worked. Chest x-ray rates in asthma fell to 15%, and have remained there since.
“We gave them permission to take their foot off the throttle and wait a little bit, and we don’t have more kids bouncing back from reduced x-rays.” The approach is “probably generalizable everywhere,” Dr. Johnson said.
It was essential that an ED fellow, Caroline Watnick, MD, led the effort and eventually bridged the gap between hospitalists and ED providers. In the end, “the change wasn’t something from the outside,” Dr. Johnson said.
There was no industry funding, and Dr. Johnson didn’t have any disclosures. The Pediatric Hospital Medicine meeting is sponsored by the Society of Hospital Medicine, the American Academy of Pediatrics, and the Academic Pediatric Association.
ATLANTA – but accomplishing this goal takes more than a new clinical practice guideline, according to a quality improvement team at the Monroe Carell Jr. Children’s Hospital at Vanderbilt University, Nashville, Tenn.
The team eventually reduced the chest x-ray rate for pediatric asthma exacerbations from 30% to 15% without increasing 3-day all-cause readmissions, but it took some sleuthing in the ED and good relations with staff. “We were way out in left field when we started this. Working in silos is never ideal,” said senior project member David Johnson, MD, a pediatric hospitalist and assistant professor of pediatrics at Vanderbilt.
It’s been known for a while that chest x-rays are almost always a waste of time and money for asthma exacerbations, and national guidelines recommend against them. X-rays don’t improve outcomes and needlessly expose children to radiation.
In 2014, some of the providers at Vanderbilt, which has about 1,700 asthma encounters a year, realized that the institution’s 30% x-ray rate was a problem. The quality improvement team hoped a new guideline would address the issue, but that didn’t happen. “We roll out clinical practice guidelines” from on high, “and think people will magically change their behavior,” but they don’t, Dr. Johnson said at the annual Pediatric Hospital Medicine meeting.
The guideline was not being fully implemented. So the team asked the ED what was the standard procedure for a child presenting with asthma exacerbation. It turned out that the ED had a dyspnea order set that the team ”had no idea existed.” Chest x-rays were at the top of the list; next came blood gases, ventilation-perfusion scans, and leg Dopplers, he said.
The investigators tried to get rid of the whole order set but were unsuccessful. The ED department did, however, let the team eliminate chest x-rays in the default order set in July 2015. That helped, but more changes were needed.
The next conversation was to figure out why x-rays were being ordered in the first place. ED staff said they were worried about missing something, especially pneumonia. They also thought they were helping hospitalists by getting x-rays before sending kids to the ward even though, in reality, it didn’t matter whether x-rays were done a few hours later on the floor. ED providers also said that ill-appearing children often got better after a few hours but were kept back from discharge because x-ray results were still pending and that sometimes these results revealed problems at 3 a.m. that had nothing to do with why the patients were in the ED but still required a work-up.
This discussion opened a door. The ED staff didn’t want to order unnecessary x-rays, either. That led to talks about letting kids declare themselves a bit before x-rays were ordered. ED staff liked the idea, so the guidelines were updated in early 2016 to say that chest x-rays should only be ordered if there is persistent severe respiratory distress with hypoxia, there are focal findings that don’t improve after 12 hours of treatment, or there were concerns for pneumomediastinum or collapsed lung. The updated guidelines were posted in work areas and brought home by resident education. A reminder was added to the electronic medical record system that popped up when someone tried to order a chest x-ray for an child with asthma.
It worked. Chest x-ray rates in asthma fell to 15%, and have remained there since.
“We gave them permission to take their foot off the throttle and wait a little bit, and we don’t have more kids bouncing back from reduced x-rays.” The approach is “probably generalizable everywhere,” Dr. Johnson said.
It was essential that an ED fellow, Caroline Watnick, MD, led the effort and eventually bridged the gap between hospitalists and ED providers. In the end, “the change wasn’t something from the outside,” Dr. Johnson said.
There was no industry funding, and Dr. Johnson didn’t have any disclosures. The Pediatric Hospital Medicine meeting is sponsored by the Society of Hospital Medicine, the American Academy of Pediatrics, and the Academic Pediatric Association.
REPORTING FROM PHM 2018
Key clinical point: Reduction of chest x-rays for routine pediatric asthma exacerbations in the ED can be accomplished with a team effort.
Major finding: A team project reduced x-rays for pediatric asthma exacerbations from 30% to 15% without increasing 3-day, all-cause readmissions.
Study details: Pre/post quality improvement analysis of asthma encounters in the Monroe Carell Jr. Children’s Hospital, Nashville, Tenn., starting in 2014.
Disclosures: There was no industry funding, and the presenter didn’t have any disclosures.
Denial of science isn’t fiction
Summer is often a time when we can take a break from our usual frenetic schedules. It is a time to catch up on nonurgent reading. I just completed 3 books of interest, including Bad Blood, by John Carreyrou, What the Eyes Don’t See, by Mona Hanna-Attisha, and Overcharged, by Charles Silver and David Hyman.
The common thread among them is their focus on the interplay among American medicine, politics, money, and denial of science. Carreyrou is a Wall Street Journal investigative reporter who describes the spectacular rise and ignominious fall of Theranos, a privately held company in the business of blood testing and diagnostics. Theranos claimed to have a secret process enabling them to run over 200 diagnostic blood tests on blood derived from a finger prick. At its peak, the company was valued at $10 billion but their secret process proved to be false science. They are now subject to multiple lawsuits and their leaders are under criminal investigation.
Dr. Attisha’s book describes the decisions made by Michigan political and administrative leaders that resulted in high levels of lead in Flint’s water supply. Dr. Attisha is a pediatrician in Flint who documented elevated lead levels in her small patients. When she tried to bring this to public attention, her data was met with enormous backlash by leaders who tried to deny facts and discredit her personally. Neurological damage from childhood lead poisoning is not reversible.
Hyman and Silver’s book (published by the Cato Institute) asserts that politics, third-party payers, and the health care industry, together, have devised a system of wealth transfer from taxpayers to health care providers. It provides examples where this system (that separates payment from health value) does real harm to individual people and the nation. Reading this book makes us think about who should hold the assets from which first-dollar medical payments derive. It reminded me of the famous saying by James Madison, “If all men were angels, there would be no need for government.”
In some circles, science, data, and the scientific method are not valued. In the end, as these books point out, everyone can be entitled to their opinion, but no one has the power to alter facts.
John I. Allen, MD, MBA, AGAF
Editor in Chief
Summer is often a time when we can take a break from our usual frenetic schedules. It is a time to catch up on nonurgent reading. I just completed 3 books of interest, including Bad Blood, by John Carreyrou, What the Eyes Don’t See, by Mona Hanna-Attisha, and Overcharged, by Charles Silver and David Hyman.
The common thread among them is their focus on the interplay among American medicine, politics, money, and denial of science. Carreyrou is a Wall Street Journal investigative reporter who describes the spectacular rise and ignominious fall of Theranos, a privately held company in the business of blood testing and diagnostics. Theranos claimed to have a secret process enabling them to run over 200 diagnostic blood tests on blood derived from a finger prick. At its peak, the company was valued at $10 billion but their secret process proved to be false science. They are now subject to multiple lawsuits and their leaders are under criminal investigation.
Dr. Attisha’s book describes the decisions made by Michigan political and administrative leaders that resulted in high levels of lead in Flint’s water supply. Dr. Attisha is a pediatrician in Flint who documented elevated lead levels in her small patients. When she tried to bring this to public attention, her data was met with enormous backlash by leaders who tried to deny facts and discredit her personally. Neurological damage from childhood lead poisoning is not reversible.
Hyman and Silver’s book (published by the Cato Institute) asserts that politics, third-party payers, and the health care industry, together, have devised a system of wealth transfer from taxpayers to health care providers. It provides examples where this system (that separates payment from health value) does real harm to individual people and the nation. Reading this book makes us think about who should hold the assets from which first-dollar medical payments derive. It reminded me of the famous saying by James Madison, “If all men were angels, there would be no need for government.”
In some circles, science, data, and the scientific method are not valued. In the end, as these books point out, everyone can be entitled to their opinion, but no one has the power to alter facts.
John I. Allen, MD, MBA, AGAF
Editor in Chief
Summer is often a time when we can take a break from our usual frenetic schedules. It is a time to catch up on nonurgent reading. I just completed 3 books of interest, including Bad Blood, by John Carreyrou, What the Eyes Don’t See, by Mona Hanna-Attisha, and Overcharged, by Charles Silver and David Hyman.
The common thread among them is their focus on the interplay among American medicine, politics, money, and denial of science. Carreyrou is a Wall Street Journal investigative reporter who describes the spectacular rise and ignominious fall of Theranos, a privately held company in the business of blood testing and diagnostics. Theranos claimed to have a secret process enabling them to run over 200 diagnostic blood tests on blood derived from a finger prick. At its peak, the company was valued at $10 billion but their secret process proved to be false science. They are now subject to multiple lawsuits and their leaders are under criminal investigation.
Dr. Attisha’s book describes the decisions made by Michigan political and administrative leaders that resulted in high levels of lead in Flint’s water supply. Dr. Attisha is a pediatrician in Flint who documented elevated lead levels in her small patients. When she tried to bring this to public attention, her data was met with enormous backlash by leaders who tried to deny facts and discredit her personally. Neurological damage from childhood lead poisoning is not reversible.
Hyman and Silver’s book (published by the Cato Institute) asserts that politics, third-party payers, and the health care industry, together, have devised a system of wealth transfer from taxpayers to health care providers. It provides examples where this system (that separates payment from health value) does real harm to individual people and the nation. Reading this book makes us think about who should hold the assets from which first-dollar medical payments derive. It reminded me of the famous saying by James Madison, “If all men were angels, there would be no need for government.”
In some circles, science, data, and the scientific method are not valued. In the end, as these books point out, everyone can be entitled to their opinion, but no one has the power to alter facts.
John I. Allen, MD, MBA, AGAF
Editor in Chief
Foster cultural competence when examining hair, scalp of ethnic patients
LAKE TAHOE, CALIF. – The way Susan C. Taylor, MD, sees it,
At the annual meeting of the Society for Pediatric Dermatology, Dr. Taylor, a dermatologist at the University of Pennsylvania, Philadelphia, defined culturally competent care as a patient-centered approach in which clinicians establish a rapport with the patient and the caregiver. “It’s important that we ask the right questions,” she said. “In doing so, we have to be familiar with common hair care practices. It’s important that we respect our patients’ values, their goals, their health needs, and, of course, their cultural background. Finally, we have to engage in shared decision making. That’s where we can improve compliance and lead to an overall very satisfactory patient visit.”
To illustrate this point, she discussed the case of a four-year-old black female with a 9-month history of bad dandruff. The child’s mother reports thick flakes that never go away. She takes pride in caring for her daughter’s hair, and shampoos it every two weeks. “She tells me that during the 2.5 hours that it takes her on Saturdays to shampoo, detangle, and comb her daughter’s hair, which she then braids or cornrows and adorns with barrettes or balls, it is a great bonding experience for the two of them,” Dr. Taylor said. “The flakes are temporarily better after she ‘greases’ her daughter’s scalp, but after 2-3 days, they are back.”
In a case like this, Dr. Taylor recommends asking the parent or caregiver to join you while you examine the scalp. This way, the focus becomes the child’s scalp, and the parent is not just staring at your expressions. “The child also observes the pediatric dermatologist and parent/caregiver working together as a team,” she said. “You also want to ask the parent to remove the hair adornments. This makes the child feel more comfortable. It also allows you to observe how the hair is being managed. Are the adornments being removed gently? Is there aggressive pulling of the hair when they take out the braids? Is the child visibly wincing in pain? If the latter two happen this is a teachable moment. You can point out, ‘It looks like Susie is in pain. Let’s do it a little more gently. That might prevent further hair breakage.’ ”
The differential diagnosis of a scaly pediatric scalp includes infrequent shampooing, seborrheic dermatitis, tinea capitis, atopic dermatitis, psoriasis, and sebopsoriasis. The type of hairstyle also factors in. For example, cornrows are a popular hair styling option among ethnic patients. “These are very popular and very time consuming and may lead to infrequent shampooing,” she said. “If they’re put in very tightly or if they have beads or other adornments, it can lead to traction alopecia.” Twists, meanwhile, can create tension on the hair, while puffs can cause traction alopecia if they’re pulled too tightly. “Although dreadlocks are more common among adolescents, we’re seeing them more commonly in young children,” she said. “They can be very long and wavy and lead to traction alopecia.”
The time required to shampoo, detangle, and style tightly coiled African American hair can be significant. For example, in children with cornrows or braids with extensions, Dr. Taylor said that it might require 30 minutes to as long as 2 or 3 hours to remove their current hairstyle, followed by shampooing and conditioning. “Detangling can take at least 15 minutes. In tightly coiled African American hair, studies have demonstrated that detangling while the hair is wet is best, because you have fewer forces on that comb and the hair is less likely to break, as opposed to detangling when the hair is dry. After the wet hair is detangled and the conditioner is rinsed out, a leave-in conditioner is often applied. Then the hair is detangled again, which can take up to an hour, followed by styling, which can take 1-3 hours. That gives you some insight as to why there can often be infrequent shampooing.”
The recommended frequency of shampooing depends on the hairstyle selected. Many children with braids and extensions will have those braids and extensions taken out every six to 12 weeks, “but that doesn’t mean that the scalp can’t be shampooed,” Dr. Taylor said. “The scalp should be shampooed more often. Economics and socioeconomic status play into the frequency of shampooing. For example, if a parent or a caregiver sends a child to a hair stylist, that can range in price from $45 to $65 or more. Time also factors in. In the black community in particular, it’s a ritual on a Saturday to get your hair done. If the parent or caregiver works on weekends, that’s going to impact the frequency of shampooing.”
Dr. Taylor underscored the importance of framing the history-taking process to avoid common pitfall questions like “Do you wash the child’s hair every day or every other day?” or “Do you use dandruff shampoo every day?” It is important to remember that “the parent’s inherent perception is of a doctor who does not have my hair probably does not understand my hair or my child’s hair,” she said. “It’s unlikely that you’re going to find a parent who shampoos their child’s hair every day or every other day. Maybe once a week, probably biweekly. It’s important to ask culturally competent questions.”
She also advises against asking about shampooing when you’re examining the child’s hair, “because there’s going to be the perception that you may think the scalp is dirty,” Dr. Taylor explained. “You probably want to ask that when gathering the history of present illness. The culturally competent question is going to be, ‘Do you wash her/his hair weekly, every other week, monthly, or does it depend on the hair style?’ ”
Body language is also important. “Don’t lean in from afar when examining the patient,” she said. “Get up close and touch the child’s hair.” If you choose to wear surgical gloves for the exam “don’t hold your hands in the surgical scrub position,” she recommended. “Hold your hands in a more neutral position. I think it’s important to touch the hair.”
Referring back to the 4-year-old black child with bad dandruff, she said that a diagnosis of seborrheic dermatitis is unlikely since that condition usually occurs during puberty. “You should have a high index of suspicion for tinea capitis,” she said. “If the patient has occipital lymphadenopathy plus scaling of the scalp or alopecia, that’s enough to presumptively treat for tinea capitis. There are studies that support that.”
For established tinea capitis, Dr. Taylor advises parents to wash barrettes and other hair adornments in hot soapy water or in the dishwasher. She also recommends disposal of hair oil, pomade, and grease and shampooing the child’s hair with ketoconazole and use a conditioner to decrease household and patient spread, which decreases transmissible fungal spores. “There’s a misperception that the application of hair oils and grease can increase the rate of tinea capitis,” she noted. “That’s not true. However, if hair grease and hair oil is applied to the scalp within one week of culture, it could produce a false negative culture.”
For established seborrheic dermatitis, antifungal shampoos including ketoconazole, ciclopirox, and selenium sulfide may be too drying for ethnic hair, “which already has a propensity to break,” she said. “Instead, we recommend a 5-10 minute scalp contact time with the shampoo and avoid contact with strands of hair. Shampoo hair strands with a conditioning shampoo followed by a conditioner to limit hair breakage. We suggest once weekly or biweekly shampooing.”
Dr. Taylor disclosed that she has advisory board and/or investigator relationships with Aclaris Therapeutics, Allergan, Beiersdorf, Croma Pharmaceuticals, Galderma, Isdin, Johnson & Johnson, and Unilever. She also acknowledged Candrice R. Heath, MD, a dermatologist based in Newark, Delaware, for her assistance with the presentation content.
[email protected]
LAKE TAHOE, CALIF. – The way Susan C. Taylor, MD, sees it,
At the annual meeting of the Society for Pediatric Dermatology, Dr. Taylor, a dermatologist at the University of Pennsylvania, Philadelphia, defined culturally competent care as a patient-centered approach in which clinicians establish a rapport with the patient and the caregiver. “It’s important that we ask the right questions,” she said. “In doing so, we have to be familiar with common hair care practices. It’s important that we respect our patients’ values, their goals, their health needs, and, of course, their cultural background. Finally, we have to engage in shared decision making. That’s where we can improve compliance and lead to an overall very satisfactory patient visit.”
To illustrate this point, she discussed the case of a four-year-old black female with a 9-month history of bad dandruff. The child’s mother reports thick flakes that never go away. She takes pride in caring for her daughter’s hair, and shampoos it every two weeks. “She tells me that during the 2.5 hours that it takes her on Saturdays to shampoo, detangle, and comb her daughter’s hair, which she then braids or cornrows and adorns with barrettes or balls, it is a great bonding experience for the two of them,” Dr. Taylor said. “The flakes are temporarily better after she ‘greases’ her daughter’s scalp, but after 2-3 days, they are back.”
In a case like this, Dr. Taylor recommends asking the parent or caregiver to join you while you examine the scalp. This way, the focus becomes the child’s scalp, and the parent is not just staring at your expressions. “The child also observes the pediatric dermatologist and parent/caregiver working together as a team,” she said. “You also want to ask the parent to remove the hair adornments. This makes the child feel more comfortable. It also allows you to observe how the hair is being managed. Are the adornments being removed gently? Is there aggressive pulling of the hair when they take out the braids? Is the child visibly wincing in pain? If the latter two happen this is a teachable moment. You can point out, ‘It looks like Susie is in pain. Let’s do it a little more gently. That might prevent further hair breakage.’ ”
The differential diagnosis of a scaly pediatric scalp includes infrequent shampooing, seborrheic dermatitis, tinea capitis, atopic dermatitis, psoriasis, and sebopsoriasis. The type of hairstyle also factors in. For example, cornrows are a popular hair styling option among ethnic patients. “These are very popular and very time consuming and may lead to infrequent shampooing,” she said. “If they’re put in very tightly or if they have beads or other adornments, it can lead to traction alopecia.” Twists, meanwhile, can create tension on the hair, while puffs can cause traction alopecia if they’re pulled too tightly. “Although dreadlocks are more common among adolescents, we’re seeing them more commonly in young children,” she said. “They can be very long and wavy and lead to traction alopecia.”
The time required to shampoo, detangle, and style tightly coiled African American hair can be significant. For example, in children with cornrows or braids with extensions, Dr. Taylor said that it might require 30 minutes to as long as 2 or 3 hours to remove their current hairstyle, followed by shampooing and conditioning. “Detangling can take at least 15 minutes. In tightly coiled African American hair, studies have demonstrated that detangling while the hair is wet is best, because you have fewer forces on that comb and the hair is less likely to break, as opposed to detangling when the hair is dry. After the wet hair is detangled and the conditioner is rinsed out, a leave-in conditioner is often applied. Then the hair is detangled again, which can take up to an hour, followed by styling, which can take 1-3 hours. That gives you some insight as to why there can often be infrequent shampooing.”
The recommended frequency of shampooing depends on the hairstyle selected. Many children with braids and extensions will have those braids and extensions taken out every six to 12 weeks, “but that doesn’t mean that the scalp can’t be shampooed,” Dr. Taylor said. “The scalp should be shampooed more often. Economics and socioeconomic status play into the frequency of shampooing. For example, if a parent or a caregiver sends a child to a hair stylist, that can range in price from $45 to $65 or more. Time also factors in. In the black community in particular, it’s a ritual on a Saturday to get your hair done. If the parent or caregiver works on weekends, that’s going to impact the frequency of shampooing.”
Dr. Taylor underscored the importance of framing the history-taking process to avoid common pitfall questions like “Do you wash the child’s hair every day or every other day?” or “Do you use dandruff shampoo every day?” It is important to remember that “the parent’s inherent perception is of a doctor who does not have my hair probably does not understand my hair or my child’s hair,” she said. “It’s unlikely that you’re going to find a parent who shampoos their child’s hair every day or every other day. Maybe once a week, probably biweekly. It’s important to ask culturally competent questions.”
She also advises against asking about shampooing when you’re examining the child’s hair, “because there’s going to be the perception that you may think the scalp is dirty,” Dr. Taylor explained. “You probably want to ask that when gathering the history of present illness. The culturally competent question is going to be, ‘Do you wash her/his hair weekly, every other week, monthly, or does it depend on the hair style?’ ”
Body language is also important. “Don’t lean in from afar when examining the patient,” she said. “Get up close and touch the child’s hair.” If you choose to wear surgical gloves for the exam “don’t hold your hands in the surgical scrub position,” she recommended. “Hold your hands in a more neutral position. I think it’s important to touch the hair.”
Referring back to the 4-year-old black child with bad dandruff, she said that a diagnosis of seborrheic dermatitis is unlikely since that condition usually occurs during puberty. “You should have a high index of suspicion for tinea capitis,” she said. “If the patient has occipital lymphadenopathy plus scaling of the scalp or alopecia, that’s enough to presumptively treat for tinea capitis. There are studies that support that.”
For established tinea capitis, Dr. Taylor advises parents to wash barrettes and other hair adornments in hot soapy water or in the dishwasher. She also recommends disposal of hair oil, pomade, and grease and shampooing the child’s hair with ketoconazole and use a conditioner to decrease household and patient spread, which decreases transmissible fungal spores. “There’s a misperception that the application of hair oils and grease can increase the rate of tinea capitis,” she noted. “That’s not true. However, if hair grease and hair oil is applied to the scalp within one week of culture, it could produce a false negative culture.”
For established seborrheic dermatitis, antifungal shampoos including ketoconazole, ciclopirox, and selenium sulfide may be too drying for ethnic hair, “which already has a propensity to break,” she said. “Instead, we recommend a 5-10 minute scalp contact time with the shampoo and avoid contact with strands of hair. Shampoo hair strands with a conditioning shampoo followed by a conditioner to limit hair breakage. We suggest once weekly or biweekly shampooing.”
Dr. Taylor disclosed that she has advisory board and/or investigator relationships with Aclaris Therapeutics, Allergan, Beiersdorf, Croma Pharmaceuticals, Galderma, Isdin, Johnson & Johnson, and Unilever. She also acknowledged Candrice R. Heath, MD, a dermatologist based in Newark, Delaware, for her assistance with the presentation content.
[email protected]
LAKE TAHOE, CALIF. – The way Susan C. Taylor, MD, sees it,
At the annual meeting of the Society for Pediatric Dermatology, Dr. Taylor, a dermatologist at the University of Pennsylvania, Philadelphia, defined culturally competent care as a patient-centered approach in which clinicians establish a rapport with the patient and the caregiver. “It’s important that we ask the right questions,” she said. “In doing so, we have to be familiar with common hair care practices. It’s important that we respect our patients’ values, their goals, their health needs, and, of course, their cultural background. Finally, we have to engage in shared decision making. That’s where we can improve compliance and lead to an overall very satisfactory patient visit.”
To illustrate this point, she discussed the case of a four-year-old black female with a 9-month history of bad dandruff. The child’s mother reports thick flakes that never go away. She takes pride in caring for her daughter’s hair, and shampoos it every two weeks. “She tells me that during the 2.5 hours that it takes her on Saturdays to shampoo, detangle, and comb her daughter’s hair, which she then braids or cornrows and adorns with barrettes or balls, it is a great bonding experience for the two of them,” Dr. Taylor said. “The flakes are temporarily better after she ‘greases’ her daughter’s scalp, but after 2-3 days, they are back.”
In a case like this, Dr. Taylor recommends asking the parent or caregiver to join you while you examine the scalp. This way, the focus becomes the child’s scalp, and the parent is not just staring at your expressions. “The child also observes the pediatric dermatologist and parent/caregiver working together as a team,” she said. “You also want to ask the parent to remove the hair adornments. This makes the child feel more comfortable. It also allows you to observe how the hair is being managed. Are the adornments being removed gently? Is there aggressive pulling of the hair when they take out the braids? Is the child visibly wincing in pain? If the latter two happen this is a teachable moment. You can point out, ‘It looks like Susie is in pain. Let’s do it a little more gently. That might prevent further hair breakage.’ ”
The differential diagnosis of a scaly pediatric scalp includes infrequent shampooing, seborrheic dermatitis, tinea capitis, atopic dermatitis, psoriasis, and sebopsoriasis. The type of hairstyle also factors in. For example, cornrows are a popular hair styling option among ethnic patients. “These are very popular and very time consuming and may lead to infrequent shampooing,” she said. “If they’re put in very tightly or if they have beads or other adornments, it can lead to traction alopecia.” Twists, meanwhile, can create tension on the hair, while puffs can cause traction alopecia if they’re pulled too tightly. “Although dreadlocks are more common among adolescents, we’re seeing them more commonly in young children,” she said. “They can be very long and wavy and lead to traction alopecia.”
The time required to shampoo, detangle, and style tightly coiled African American hair can be significant. For example, in children with cornrows or braids with extensions, Dr. Taylor said that it might require 30 minutes to as long as 2 or 3 hours to remove their current hairstyle, followed by shampooing and conditioning. “Detangling can take at least 15 minutes. In tightly coiled African American hair, studies have demonstrated that detangling while the hair is wet is best, because you have fewer forces on that comb and the hair is less likely to break, as opposed to detangling when the hair is dry. After the wet hair is detangled and the conditioner is rinsed out, a leave-in conditioner is often applied. Then the hair is detangled again, which can take up to an hour, followed by styling, which can take 1-3 hours. That gives you some insight as to why there can often be infrequent shampooing.”
The recommended frequency of shampooing depends on the hairstyle selected. Many children with braids and extensions will have those braids and extensions taken out every six to 12 weeks, “but that doesn’t mean that the scalp can’t be shampooed,” Dr. Taylor said. “The scalp should be shampooed more often. Economics and socioeconomic status play into the frequency of shampooing. For example, if a parent or a caregiver sends a child to a hair stylist, that can range in price from $45 to $65 or more. Time also factors in. In the black community in particular, it’s a ritual on a Saturday to get your hair done. If the parent or caregiver works on weekends, that’s going to impact the frequency of shampooing.”
Dr. Taylor underscored the importance of framing the history-taking process to avoid common pitfall questions like “Do you wash the child’s hair every day or every other day?” or “Do you use dandruff shampoo every day?” It is important to remember that “the parent’s inherent perception is of a doctor who does not have my hair probably does not understand my hair or my child’s hair,” she said. “It’s unlikely that you’re going to find a parent who shampoos their child’s hair every day or every other day. Maybe once a week, probably biweekly. It’s important to ask culturally competent questions.”
She also advises against asking about shampooing when you’re examining the child’s hair, “because there’s going to be the perception that you may think the scalp is dirty,” Dr. Taylor explained. “You probably want to ask that when gathering the history of present illness. The culturally competent question is going to be, ‘Do you wash her/his hair weekly, every other week, monthly, or does it depend on the hair style?’ ”
Body language is also important. “Don’t lean in from afar when examining the patient,” she said. “Get up close and touch the child’s hair.” If you choose to wear surgical gloves for the exam “don’t hold your hands in the surgical scrub position,” she recommended. “Hold your hands in a more neutral position. I think it’s important to touch the hair.”
Referring back to the 4-year-old black child with bad dandruff, she said that a diagnosis of seborrheic dermatitis is unlikely since that condition usually occurs during puberty. “You should have a high index of suspicion for tinea capitis,” she said. “If the patient has occipital lymphadenopathy plus scaling of the scalp or alopecia, that’s enough to presumptively treat for tinea capitis. There are studies that support that.”
For established tinea capitis, Dr. Taylor advises parents to wash barrettes and other hair adornments in hot soapy water or in the dishwasher. She also recommends disposal of hair oil, pomade, and grease and shampooing the child’s hair with ketoconazole and use a conditioner to decrease household and patient spread, which decreases transmissible fungal spores. “There’s a misperception that the application of hair oils and grease can increase the rate of tinea capitis,” she noted. “That’s not true. However, if hair grease and hair oil is applied to the scalp within one week of culture, it could produce a false negative culture.”
For established seborrheic dermatitis, antifungal shampoos including ketoconazole, ciclopirox, and selenium sulfide may be too drying for ethnic hair, “which already has a propensity to break,” she said. “Instead, we recommend a 5-10 minute scalp contact time with the shampoo and avoid contact with strands of hair. Shampoo hair strands with a conditioning shampoo followed by a conditioner to limit hair breakage. We suggest once weekly or biweekly shampooing.”
Dr. Taylor disclosed that she has advisory board and/or investigator relationships with Aclaris Therapeutics, Allergan, Beiersdorf, Croma Pharmaceuticals, Galderma, Isdin, Johnson & Johnson, and Unilever. She also acknowledged Candrice R. Heath, MD, a dermatologist based in Newark, Delaware, for her assistance with the presentation content.
[email protected]
EXPERT ANALYSIS FROM THE SPD ANNUAL MEETING
Taking the sting out of nail surgery: Postoperative pain pearls
CHICAGO – In a busy clinic it can be hard to find the time to stop, talk, and listen. But doing so will “pay dividends in time spent later – and in reduced complications” of nail surgery, according to Molly A. Hinshaw, MD.
Dr. Hinshaw, director of the nail clinic at the University of Wisconsin–Madison, shared her
“One pearl is the importance of patient education before we start,” Dr. Hinshaw said. Preoperatively, she takes time to talk through the entire surgery and expected postoperative course. Critically, she reassures patients that pain will be controlled; she also reviews in detail what the pharmacologic and nonpharmacologic pain control strategies will be.
In addition, it’s important to address patients’ natural anxiety about what the surgical site will look and feel like and how healing will progress, particularly in those first few days after surgery. “I offer a first dressing change in my practice, either at 24 or 48 hours. This can be very anxiolytic for the patient,” she said.
At the preoperative stage, Dr. Hinshaw also tells patients that, from a healing and pain management standpoint, to make sure they plan “to have a restful 48 hours after surgery.” Her patient instructions for the immediate postoperative period include keeping the limb elevated and avoiding unnecessary activity with the affected limb while the digit, whether a finger or toe, is still anesthetized. To stay on top of the pain, the appropriate oral pain medication should be started once sensation starts to return to the digit. She recommends patients also take a dose of their pain medication at bedtime, as this will help them get a restful night of sleep.
“One thing that I’ve learned over the years is that throbbing and a little bit of swelling after surgery is not uncommon,” said Dr. Hinshaw, who uses elastic self-adherent wrap for the top layer of wound dressings after nail surgery. She tells her patients, “if you’re feeling throbbing, you’re welcome to unwrap it and rewrap it more loosely.” Just giving the patient the ability to find a comfortable level of pressure on the affected digit is often enough to alleviate the throbbing, as opposed to treating that throbbing with pain medication.
Dr. Hinshaw said she’s learned to tailor her postoperative analgesia to the surgery and to the patient. With all patients, she is sure to make medication and dosing choices that take comorbidities and potential drug-drug interactions into account. She does not ask patients to stop anticoagulation before nail procedures.
For phenolization procedures and punch biopsies, she’ll advise patients to use acetaminophen or NSAIDs. Some procedures are going to have a more painful recovery course, said Dr. Hinshaw, so she’ll use an opioid such as hydrocodone with acetaminophen for shave excisions and fusiform longitudinal excisions.
The physician and patient can also plan ahead for a brief course of more potent opioids for some procedures. “Certainly for lateral longitudinal excisions, they will need narcotic pain management for at least 48 hours after surgery,” she noted. “It’s a painful surgery.”
Other procedures that will need more postoperative analgesia include flaps and nail unit grafts, she said. In general, NSAIDs are useful to add after the first 24 hours. In addition, “I always call my patients the day after surgery to see how they’re doing. This helps identify any issues and questions early and is comforting to the patient,” she added.
Dr. Hinshaw disclosed that she has an ownership stake in and sits on the board of directors of Accure Medical.
CHICAGO – In a busy clinic it can be hard to find the time to stop, talk, and listen. But doing so will “pay dividends in time spent later – and in reduced complications” of nail surgery, according to Molly A. Hinshaw, MD.
Dr. Hinshaw, director of the nail clinic at the University of Wisconsin–Madison, shared her
“One pearl is the importance of patient education before we start,” Dr. Hinshaw said. Preoperatively, she takes time to talk through the entire surgery and expected postoperative course. Critically, she reassures patients that pain will be controlled; she also reviews in detail what the pharmacologic and nonpharmacologic pain control strategies will be.
In addition, it’s important to address patients’ natural anxiety about what the surgical site will look and feel like and how healing will progress, particularly in those first few days after surgery. “I offer a first dressing change in my practice, either at 24 or 48 hours. This can be very anxiolytic for the patient,” she said.
At the preoperative stage, Dr. Hinshaw also tells patients that, from a healing and pain management standpoint, to make sure they plan “to have a restful 48 hours after surgery.” Her patient instructions for the immediate postoperative period include keeping the limb elevated and avoiding unnecessary activity with the affected limb while the digit, whether a finger or toe, is still anesthetized. To stay on top of the pain, the appropriate oral pain medication should be started once sensation starts to return to the digit. She recommends patients also take a dose of their pain medication at bedtime, as this will help them get a restful night of sleep.
“One thing that I’ve learned over the years is that throbbing and a little bit of swelling after surgery is not uncommon,” said Dr. Hinshaw, who uses elastic self-adherent wrap for the top layer of wound dressings after nail surgery. She tells her patients, “if you’re feeling throbbing, you’re welcome to unwrap it and rewrap it more loosely.” Just giving the patient the ability to find a comfortable level of pressure on the affected digit is often enough to alleviate the throbbing, as opposed to treating that throbbing with pain medication.
Dr. Hinshaw said she’s learned to tailor her postoperative analgesia to the surgery and to the patient. With all patients, she is sure to make medication and dosing choices that take comorbidities and potential drug-drug interactions into account. She does not ask patients to stop anticoagulation before nail procedures.
For phenolization procedures and punch biopsies, she’ll advise patients to use acetaminophen or NSAIDs. Some procedures are going to have a more painful recovery course, said Dr. Hinshaw, so she’ll use an opioid such as hydrocodone with acetaminophen for shave excisions and fusiform longitudinal excisions.
The physician and patient can also plan ahead for a brief course of more potent opioids for some procedures. “Certainly for lateral longitudinal excisions, they will need narcotic pain management for at least 48 hours after surgery,” she noted. “It’s a painful surgery.”
Other procedures that will need more postoperative analgesia include flaps and nail unit grafts, she said. In general, NSAIDs are useful to add after the first 24 hours. In addition, “I always call my patients the day after surgery to see how they’re doing. This helps identify any issues and questions early and is comforting to the patient,” she added.
Dr. Hinshaw disclosed that she has an ownership stake in and sits on the board of directors of Accure Medical.
CHICAGO – In a busy clinic it can be hard to find the time to stop, talk, and listen. But doing so will “pay dividends in time spent later – and in reduced complications” of nail surgery, according to Molly A. Hinshaw, MD.
Dr. Hinshaw, director of the nail clinic at the University of Wisconsin–Madison, shared her
“One pearl is the importance of patient education before we start,” Dr. Hinshaw said. Preoperatively, she takes time to talk through the entire surgery and expected postoperative course. Critically, she reassures patients that pain will be controlled; she also reviews in detail what the pharmacologic and nonpharmacologic pain control strategies will be.
In addition, it’s important to address patients’ natural anxiety about what the surgical site will look and feel like and how healing will progress, particularly in those first few days after surgery. “I offer a first dressing change in my practice, either at 24 or 48 hours. This can be very anxiolytic for the patient,” she said.
At the preoperative stage, Dr. Hinshaw also tells patients that, from a healing and pain management standpoint, to make sure they plan “to have a restful 48 hours after surgery.” Her patient instructions for the immediate postoperative period include keeping the limb elevated and avoiding unnecessary activity with the affected limb while the digit, whether a finger or toe, is still anesthetized. To stay on top of the pain, the appropriate oral pain medication should be started once sensation starts to return to the digit. She recommends patients also take a dose of their pain medication at bedtime, as this will help them get a restful night of sleep.
“One thing that I’ve learned over the years is that throbbing and a little bit of swelling after surgery is not uncommon,” said Dr. Hinshaw, who uses elastic self-adherent wrap for the top layer of wound dressings after nail surgery. She tells her patients, “if you’re feeling throbbing, you’re welcome to unwrap it and rewrap it more loosely.” Just giving the patient the ability to find a comfortable level of pressure on the affected digit is often enough to alleviate the throbbing, as opposed to treating that throbbing with pain medication.
Dr. Hinshaw said she’s learned to tailor her postoperative analgesia to the surgery and to the patient. With all patients, she is sure to make medication and dosing choices that take comorbidities and potential drug-drug interactions into account. She does not ask patients to stop anticoagulation before nail procedures.
For phenolization procedures and punch biopsies, she’ll advise patients to use acetaminophen or NSAIDs. Some procedures are going to have a more painful recovery course, said Dr. Hinshaw, so she’ll use an opioid such as hydrocodone with acetaminophen for shave excisions and fusiform longitudinal excisions.
The physician and patient can also plan ahead for a brief course of more potent opioids for some procedures. “Certainly for lateral longitudinal excisions, they will need narcotic pain management for at least 48 hours after surgery,” she noted. “It’s a painful surgery.”
Other procedures that will need more postoperative analgesia include flaps and nail unit grafts, she said. In general, NSAIDs are useful to add after the first 24 hours. In addition, “I always call my patients the day after surgery to see how they’re doing. This helps identify any issues and questions early and is comforting to the patient,” she added.
Dr. Hinshaw disclosed that she has an ownership stake in and sits on the board of directors of Accure Medical.
EXPERT ANALYSIS FROM SUMMER AAD 2018
Does supplemental oxygen help COPD patients who have chronic stable moderate hypoxia?
New study a departure from previous research
Case
An 85-year-old man with long-standing chronic obstructive pulmonary disease (COPD) has a witnessed aspiration event while undergoing an outpatient procedure requiring conscious sedation. He is admitted to the hospital for observation overnight. The next morning, he feels well, but his oxygen saturation dips to 85% with ambulation. He reports this is not new for him, but he vehemently does not want supplemental oxygen.
Background
Patients with COPD and severe resting hypoxemia – arterial oxygen partial pressure less than or equal to 55 mm Hg or peripheral capillary oxygen saturation (SpO2) less than or equal to 88% – commonly are prescribed supplemental oxygen. The evidence supporting this practice is limited to two small trials from the 1970s that showed a survival benefit of long-term oxygen therapy (LTOT) in this population,1,2 but these trials may not be generalizable to patients today.
For patients with COPD and mild to moderate resting hypoxemia (SpO2, 89%-93%) or patients with exercise-induced hypoxemia, LTOT has not been shown to improve survival, although it may improve symptoms of dyspnea, exercise tolerance, and other patient reported outcomes. Given the costs, risks, and burdens associated with LTOT, a high-quality clinical trial assessing the effects of LTOT on clinically meaningful outcomes, such as survival or hospitalization, in patients with COPD and moderate hypoxemia has been long overdue.
Overview of the data
The utility of long-term treatment with supplemental oxygen in patients with stable COPD and moderate resting or exercise-induced desaturation was examined by the Long-Term Oxygen Treatment Trial (LOTT) Research Group. Results were published in the New England Journal of Medicine in October 2016 in the article, “A Randomized Trial of Long-Term Oxygen for COPD with Moderate Desaturation.”3
The study was initially designed to test whether the use of supplemental oxygen would lead to longer time until death as compared with no supplemental oxygen in the subgroup of COPD patients with stable disease and moderate resting desaturation (defined as resting SpO2 of 89%-93%). However, because of an enrollment of only 34 patients after 7 months, the trial was redesigned to include exercise-induced desaturation (defined as SpO2 of greater than or equal to 80% for at least 5 minutes, and less than 90% for at least 10 seconds, on a 6-minute walk test) and the secondary outcome of all-cause hospitalization. Hospitalization for any cause was combined with mortality into a new composite primary outcome.
This study was a randomized, controlled trial which enrolled patients at a total of 14 regional clinical centers and their associated sites for a total of 42 centers in the United States. The experimental arm consisted of a long term supplemental oxygen group, and the control group did not receive long term supplemental oxygen. Patients were assigned to groups in a 1:1 ratio and the study was not blinded. Patients with moderate resting desaturation were prescribed 24 hour oxygen at 2 L/min, and patients with moderate exercise-induced desaturation were prescribed oxygen at 2 L/min during exercise and sleep only. The primary outcome was a composite outcome of time until death or time until first hospitalization for any cause. There were multiple secondary outcomes, including incidence of COPD exacerbation, incidence of severe resting desaturation and severe exercise-induced desaturation, quality of life, sleep quality, depression and anxiety, adherence to regimen, 6-minute walk distance, spirometric measurements, risk of cardiovascular disease, and neurocognitive function.
Data were gathered via yearly visits, biannual telephone interviews, and questionnaires mailed at 4 months and 16 months. Adherence was assessed by inquiring about oxygen use every 4 months. If patients in the supplemental oxygen group used stationary oxygen concentrators, logs of meter readings were kept as well. The necessary final sample size was calculated using a time to composite event survival model with the use of the log-rank test statistic.
A total of 738 patients were enrolled in the trial between January 2009 and September 2015 and were followed for 1-6 years. A total of 97% of participants had at least 1 year of follow-up. Out of the 738 randomized patients, 133 (18%) had only resting desaturations, 319 (43%) had only exercise-induced desaturations, and 286 (39%) had both resting and exercise-induced desaturations. Baseline characteristics including age, sex, race, smoking status, quality of life scores, resting SpO2, and nadir SpO2 during the 6-minute walk test were similar between the two groups. The only significant difference noted by the authors between the two groups was a lower BODE (body mass index, airflow obstruction, dyspnea, and exercise) index, which was lower in the group with no supplemental oxygen.
In the time-to-event analysis, there was no significant difference between the two groups in the time to death or first hospitalization (hazard ratio, 0.94; 95% confidence interval, 0.79-1.12; P = .52). There were no significant differences in the rates of all hospitalizations (rate ratio, 1.01; 95% CI, 0.91-1.13), COPD exacerbations (RR 1.08; 95% CI, 0.98-1.19), and COPD related hospitalizations (RR, 0.99; 95% CI, 0.83-1.17). There were also no differences between the experimental and control groups in quality of life, lung function, and 6-minute walk distance. There were no significant differences in the subgroups classified by desaturation profile, sex, race, nadir SpO2 during the 6-minute walk test, and forced expiratory volume in 1 second.
The findings in this study show that, in the subgroup of chronic obstructive pulmonary disease patients with stable COPD and moderate resting or exercise-induced desaturation, supplemental oxygen did not affect the time to death or first hospitalization, time to death, time to first hospitalization, time to first COPD exacerbation, time to first hospitalization for a COPD exacerbation, rate of all hospitalizations, rate of all COPD exacerbations, or changes in metrics surrounding quality of life, anxiety/depression, or functional status. This supports earlier studies that demonstrated that long-term treatment with oxygen does not result in longer survival than does no long-term treatment with oxygen in patients with COPD and resting SpO2 of more than 88%.
The results of this study are a departure from previous studies that had shown improved mortality in patients with COPD and severe desaturation who were treated with LTOT. The authors hypothesized that this may have been caused by physiological effects of oxygen saturation on pulmonary vasoconstriction, release of mediators, and ventilator drive, which occur at an O2 saturation of 88% or less and may be more significant in patients with chronic hypoxemia. This trial also contrasted previous studies that had shown that oxygen therapy may reduce dyspnea in COPD patients with mild or no hypoxia because the LOTT trial showed no improvement in quality of life, anxiety, and depression measures in patients treated with long-term oxygen as compared with those treated with no oxygen.
Some limitations of the study included the absence of highly symptomatic patients or patients who the providers believed were too ill to participate, the effect of the unblinded nature of the study on outcomes that were patient reported, the lack of assessment of immediate effects of oxygen on exercise performance or symptoms, possible variability in amount of oxygen delivered, and the fact that patients may have overestimated their oxygen use.
In patients with stable COPD and moderate resting or exercise induced desaturation, long-term supplemental oxygen did not provide any benefit in regard to time until death or first hospitalization or any of the other measured outcomes.
Application of data to the case
Our patient has stable COPD and had only moderate exercise-induced desaturation. Long-term supplemental oxygen would not produce a benefit for him.
This study shows us that it would not increase his survival at this point; however, if he were to have worsening exercise-induced or new resting desaturation at some point in the future, supplemental oxygen would then be beneficial. At this point supplemental oxygen would not even affect his rate of hospitalization for COPD- or non-COPD–related reasons. Perhaps most importantly, adding oxygen therapy would not affect his overall quality of life, including his functional status and mood.
Bottom line
The addition of supplemental oxygen is not helpful for patients with COPD who have chronic stable moderate hypoxia.
Dr. Farber is a medical instructor in the Duke University Health System in Durham, N.C. Dr. Sata is a medical instructor in the Duke University Hospital. Dr. Wachter is an assistant professor of medicine at Duke University. Dr. Sharma is associate medical director for clinical education in hospital medicine at Duke Regional Hospital and an assistant professor of medicine at Duke University.
References
1. Nocturnal Oxygen Therapy Trial Group. Continuous or nocturnal oxygen therapy in hypoxemic chronic obstructive lung disease: A clinical trial. Ann Intern Med. 1980 Sep;93(3):391-8.
2. Medical Research Council Working Party. Long term domiciliary oxygen therapy in chronic hypoxic cor pulmonale complicating chronic bronchitis and emphysema: Report of the Medical Research Council Working Party. Lancet 1981 Mar 28;1(8222):681-6.
3. Long-term oxygen treatment trial research group et al. A randomized trial of long-term oxygen for COPD with moderate desaturation. N Engl J Med. 2016 Oct 27;375(17):1617-27.
Additional reading
Stoller JK et al. Oxygen therapy for patients with COPD: Current evidence and the Long-term Oxygen Treatment Trial. Chest. 2010 July;138:179-87.
Qaseem A et al. Diagnosis and management of stable chronic obstructive pulmonary disease: A clinical practice guideline update from the American College of Physicians, American College of Chest Physicians, American Thoracic Society, and European Respiratory Society. Ann Intern Med. 2011 Aug 2;155(3):179-91.
Ameer F et al. Ambulatory oxygen for people with chronic obstructive pulmonary disease who are not hypoxaemic at rest. Cochrane Database Syst Rev. 2014 Jun 24;(6):CD000238.
Vestbo J et al. Global strategy for the diagnosis, management, and prevention of chronic obstructive pulmonary disease: GOLD executive summary. Am J Respir Crit Care Med. 2013 Feb 15;187(4):347-65.
Quiz: Does this patient need oxygen?
You are caring for a 72-year-old man with stable COPD who was admitted for cellulitis. He is improving clinically on appropriate antibiotics, and he has been stable on room air every time you examine him. The nurse pages you on the day of discharge – a Sunday – informing you that his oxygen saturation dropped to 88% while he was walking the halls this morning. She asks whether he needs to stay in the hospital so you can arrange home supplemental oxygen therapy. What should you do?
A. Keep him in the hospital until you can arrange home oxygen therapy.
B. Discharge him home Sunday but have the oxygen company go out to his house first thing on Monday.
C. Discharge him home without supplemental oxygen therapy.
D. Check an arterial blood gas to help decide if you should set up oxygen therapy.
The answer is C. He meets the description of stable COPD with mild to moderate exercise-induced desaturation. The LOTT trial supports our clinical decision that he would not benefit from supplemental oxygen therapy at this point.
Key Points
- Long-term oxygen therapy (LTOT) is beneficial in patients with COPD and severe resting hypoxemia (arterial oxygen partial pressure ≤ 55 mm Hg or SpO2 ≤ 88%) and should be prescribed to improve survival in this population.
- Patients with COPD and mild to moderate resting hypoxemia or exercised-induced hypoxemia should not be routinely prescribed LTOT given the associated costs, risks, and burdens and the lack of evidence of benefit.
New study a departure from previous research
New study a departure from previous research
Case
An 85-year-old man with long-standing chronic obstructive pulmonary disease (COPD) has a witnessed aspiration event while undergoing an outpatient procedure requiring conscious sedation. He is admitted to the hospital for observation overnight. The next morning, he feels well, but his oxygen saturation dips to 85% with ambulation. He reports this is not new for him, but he vehemently does not want supplemental oxygen.
Background
Patients with COPD and severe resting hypoxemia – arterial oxygen partial pressure less than or equal to 55 mm Hg or peripheral capillary oxygen saturation (SpO2) less than or equal to 88% – commonly are prescribed supplemental oxygen. The evidence supporting this practice is limited to two small trials from the 1970s that showed a survival benefit of long-term oxygen therapy (LTOT) in this population,1,2 but these trials may not be generalizable to patients today.
For patients with COPD and mild to moderate resting hypoxemia (SpO2, 89%-93%) or patients with exercise-induced hypoxemia, LTOT has not been shown to improve survival, although it may improve symptoms of dyspnea, exercise tolerance, and other patient reported outcomes. Given the costs, risks, and burdens associated with LTOT, a high-quality clinical trial assessing the effects of LTOT on clinically meaningful outcomes, such as survival or hospitalization, in patients with COPD and moderate hypoxemia has been long overdue.
Overview of the data
The utility of long-term treatment with supplemental oxygen in patients with stable COPD and moderate resting or exercise-induced desaturation was examined by the Long-Term Oxygen Treatment Trial (LOTT) Research Group. Results were published in the New England Journal of Medicine in October 2016 in the article, “A Randomized Trial of Long-Term Oxygen for COPD with Moderate Desaturation.”3
The study was initially designed to test whether the use of supplemental oxygen would lead to longer time until death as compared with no supplemental oxygen in the subgroup of COPD patients with stable disease and moderate resting desaturation (defined as resting SpO2 of 89%-93%). However, because of an enrollment of only 34 patients after 7 months, the trial was redesigned to include exercise-induced desaturation (defined as SpO2 of greater than or equal to 80% for at least 5 minutes, and less than 90% for at least 10 seconds, on a 6-minute walk test) and the secondary outcome of all-cause hospitalization. Hospitalization for any cause was combined with mortality into a new composite primary outcome.
This study was a randomized, controlled trial which enrolled patients at a total of 14 regional clinical centers and their associated sites for a total of 42 centers in the United States. The experimental arm consisted of a long term supplemental oxygen group, and the control group did not receive long term supplemental oxygen. Patients were assigned to groups in a 1:1 ratio and the study was not blinded. Patients with moderate resting desaturation were prescribed 24 hour oxygen at 2 L/min, and patients with moderate exercise-induced desaturation were prescribed oxygen at 2 L/min during exercise and sleep only. The primary outcome was a composite outcome of time until death or time until first hospitalization for any cause. There were multiple secondary outcomes, including incidence of COPD exacerbation, incidence of severe resting desaturation and severe exercise-induced desaturation, quality of life, sleep quality, depression and anxiety, adherence to regimen, 6-minute walk distance, spirometric measurements, risk of cardiovascular disease, and neurocognitive function.
Data were gathered via yearly visits, biannual telephone interviews, and questionnaires mailed at 4 months and 16 months. Adherence was assessed by inquiring about oxygen use every 4 months. If patients in the supplemental oxygen group used stationary oxygen concentrators, logs of meter readings were kept as well. The necessary final sample size was calculated using a time to composite event survival model with the use of the log-rank test statistic.
A total of 738 patients were enrolled in the trial between January 2009 and September 2015 and were followed for 1-6 years. A total of 97% of participants had at least 1 year of follow-up. Out of the 738 randomized patients, 133 (18%) had only resting desaturations, 319 (43%) had only exercise-induced desaturations, and 286 (39%) had both resting and exercise-induced desaturations. Baseline characteristics including age, sex, race, smoking status, quality of life scores, resting SpO2, and nadir SpO2 during the 6-minute walk test were similar between the two groups. The only significant difference noted by the authors between the two groups was a lower BODE (body mass index, airflow obstruction, dyspnea, and exercise) index, which was lower in the group with no supplemental oxygen.
In the time-to-event analysis, there was no significant difference between the two groups in the time to death or first hospitalization (hazard ratio, 0.94; 95% confidence interval, 0.79-1.12; P = .52). There were no significant differences in the rates of all hospitalizations (rate ratio, 1.01; 95% CI, 0.91-1.13), COPD exacerbations (RR 1.08; 95% CI, 0.98-1.19), and COPD related hospitalizations (RR, 0.99; 95% CI, 0.83-1.17). There were also no differences between the experimental and control groups in quality of life, lung function, and 6-minute walk distance. There were no significant differences in the subgroups classified by desaturation profile, sex, race, nadir SpO2 during the 6-minute walk test, and forced expiratory volume in 1 second.
The findings in this study show that, in the subgroup of chronic obstructive pulmonary disease patients with stable COPD and moderate resting or exercise-induced desaturation, supplemental oxygen did not affect the time to death or first hospitalization, time to death, time to first hospitalization, time to first COPD exacerbation, time to first hospitalization for a COPD exacerbation, rate of all hospitalizations, rate of all COPD exacerbations, or changes in metrics surrounding quality of life, anxiety/depression, or functional status. This supports earlier studies that demonstrated that long-term treatment with oxygen does not result in longer survival than does no long-term treatment with oxygen in patients with COPD and resting SpO2 of more than 88%.
The results of this study are a departure from previous studies that had shown improved mortality in patients with COPD and severe desaturation who were treated with LTOT. The authors hypothesized that this may have been caused by physiological effects of oxygen saturation on pulmonary vasoconstriction, release of mediators, and ventilator drive, which occur at an O2 saturation of 88% or less and may be more significant in patients with chronic hypoxemia. This trial also contrasted previous studies that had shown that oxygen therapy may reduce dyspnea in COPD patients with mild or no hypoxia because the LOTT trial showed no improvement in quality of life, anxiety, and depression measures in patients treated with long-term oxygen as compared with those treated with no oxygen.
Some limitations of the study included the absence of highly symptomatic patients or patients who the providers believed were too ill to participate, the effect of the unblinded nature of the study on outcomes that were patient reported, the lack of assessment of immediate effects of oxygen on exercise performance or symptoms, possible variability in amount of oxygen delivered, and the fact that patients may have overestimated their oxygen use.
In patients with stable COPD and moderate resting or exercise induced desaturation, long-term supplemental oxygen did not provide any benefit in regard to time until death or first hospitalization or any of the other measured outcomes.
Application of data to the case
Our patient has stable COPD and had only moderate exercise-induced desaturation. Long-term supplemental oxygen would not produce a benefit for him.
This study shows us that it would not increase his survival at this point; however, if he were to have worsening exercise-induced or new resting desaturation at some point in the future, supplemental oxygen would then be beneficial. At this point supplemental oxygen would not even affect his rate of hospitalization for COPD- or non-COPD–related reasons. Perhaps most importantly, adding oxygen therapy would not affect his overall quality of life, including his functional status and mood.
Bottom line
The addition of supplemental oxygen is not helpful for patients with COPD who have chronic stable moderate hypoxia.
Dr. Farber is a medical instructor in the Duke University Health System in Durham, N.C. Dr. Sata is a medical instructor in the Duke University Hospital. Dr. Wachter is an assistant professor of medicine at Duke University. Dr. Sharma is associate medical director for clinical education in hospital medicine at Duke Regional Hospital and an assistant professor of medicine at Duke University.
References
1. Nocturnal Oxygen Therapy Trial Group. Continuous or nocturnal oxygen therapy in hypoxemic chronic obstructive lung disease: A clinical trial. Ann Intern Med. 1980 Sep;93(3):391-8.
2. Medical Research Council Working Party. Long term domiciliary oxygen therapy in chronic hypoxic cor pulmonale complicating chronic bronchitis and emphysema: Report of the Medical Research Council Working Party. Lancet 1981 Mar 28;1(8222):681-6.
3. Long-term oxygen treatment trial research group et al. A randomized trial of long-term oxygen for COPD with moderate desaturation. N Engl J Med. 2016 Oct 27;375(17):1617-27.
Additional reading
Stoller JK et al. Oxygen therapy for patients with COPD: Current evidence and the Long-term Oxygen Treatment Trial. Chest. 2010 July;138:179-87.
Qaseem A et al. Diagnosis and management of stable chronic obstructive pulmonary disease: A clinical practice guideline update from the American College of Physicians, American College of Chest Physicians, American Thoracic Society, and European Respiratory Society. Ann Intern Med. 2011 Aug 2;155(3):179-91.
Ameer F et al. Ambulatory oxygen for people with chronic obstructive pulmonary disease who are not hypoxaemic at rest. Cochrane Database Syst Rev. 2014 Jun 24;(6):CD000238.
Vestbo J et al. Global strategy for the diagnosis, management, and prevention of chronic obstructive pulmonary disease: GOLD executive summary. Am J Respir Crit Care Med. 2013 Feb 15;187(4):347-65.
Quiz: Does this patient need oxygen?
You are caring for a 72-year-old man with stable COPD who was admitted for cellulitis. He is improving clinically on appropriate antibiotics, and he has been stable on room air every time you examine him. The nurse pages you on the day of discharge – a Sunday – informing you that his oxygen saturation dropped to 88% while he was walking the halls this morning. She asks whether he needs to stay in the hospital so you can arrange home supplemental oxygen therapy. What should you do?
A. Keep him in the hospital until you can arrange home oxygen therapy.
B. Discharge him home Sunday but have the oxygen company go out to his house first thing on Monday.
C. Discharge him home without supplemental oxygen therapy.
D. Check an arterial blood gas to help decide if you should set up oxygen therapy.
The answer is C. He meets the description of stable COPD with mild to moderate exercise-induced desaturation. The LOTT trial supports our clinical decision that he would not benefit from supplemental oxygen therapy at this point.
Key Points
- Long-term oxygen therapy (LTOT) is beneficial in patients with COPD and severe resting hypoxemia (arterial oxygen partial pressure ≤ 55 mm Hg or SpO2 ≤ 88%) and should be prescribed to improve survival in this population.
- Patients with COPD and mild to moderate resting hypoxemia or exercised-induced hypoxemia should not be routinely prescribed LTOT given the associated costs, risks, and burdens and the lack of evidence of benefit.
Case
An 85-year-old man with long-standing chronic obstructive pulmonary disease (COPD) has a witnessed aspiration event while undergoing an outpatient procedure requiring conscious sedation. He is admitted to the hospital for observation overnight. The next morning, he feels well, but his oxygen saturation dips to 85% with ambulation. He reports this is not new for him, but he vehemently does not want supplemental oxygen.
Background
Patients with COPD and severe resting hypoxemia – arterial oxygen partial pressure less than or equal to 55 mm Hg or peripheral capillary oxygen saturation (SpO2) less than or equal to 88% – commonly are prescribed supplemental oxygen. The evidence supporting this practice is limited to two small trials from the 1970s that showed a survival benefit of long-term oxygen therapy (LTOT) in this population,1,2 but these trials may not be generalizable to patients today.
For patients with COPD and mild to moderate resting hypoxemia (SpO2, 89%-93%) or patients with exercise-induced hypoxemia, LTOT has not been shown to improve survival, although it may improve symptoms of dyspnea, exercise tolerance, and other patient reported outcomes. Given the costs, risks, and burdens associated with LTOT, a high-quality clinical trial assessing the effects of LTOT on clinically meaningful outcomes, such as survival or hospitalization, in patients with COPD and moderate hypoxemia has been long overdue.
Overview of the data
The utility of long-term treatment with supplemental oxygen in patients with stable COPD and moderate resting or exercise-induced desaturation was examined by the Long-Term Oxygen Treatment Trial (LOTT) Research Group. Results were published in the New England Journal of Medicine in October 2016 in the article, “A Randomized Trial of Long-Term Oxygen for COPD with Moderate Desaturation.”3
The study was initially designed to test whether the use of supplemental oxygen would lead to longer time until death as compared with no supplemental oxygen in the subgroup of COPD patients with stable disease and moderate resting desaturation (defined as resting SpO2 of 89%-93%). However, because of an enrollment of only 34 patients after 7 months, the trial was redesigned to include exercise-induced desaturation (defined as SpO2 of greater than or equal to 80% for at least 5 minutes, and less than 90% for at least 10 seconds, on a 6-minute walk test) and the secondary outcome of all-cause hospitalization. Hospitalization for any cause was combined with mortality into a new composite primary outcome.
This study was a randomized, controlled trial which enrolled patients at a total of 14 regional clinical centers and their associated sites for a total of 42 centers in the United States. The experimental arm consisted of a long term supplemental oxygen group, and the control group did not receive long term supplemental oxygen. Patients were assigned to groups in a 1:1 ratio and the study was not blinded. Patients with moderate resting desaturation were prescribed 24 hour oxygen at 2 L/min, and patients with moderate exercise-induced desaturation were prescribed oxygen at 2 L/min during exercise and sleep only. The primary outcome was a composite outcome of time until death or time until first hospitalization for any cause. There were multiple secondary outcomes, including incidence of COPD exacerbation, incidence of severe resting desaturation and severe exercise-induced desaturation, quality of life, sleep quality, depression and anxiety, adherence to regimen, 6-minute walk distance, spirometric measurements, risk of cardiovascular disease, and neurocognitive function.
Data were gathered via yearly visits, biannual telephone interviews, and questionnaires mailed at 4 months and 16 months. Adherence was assessed by inquiring about oxygen use every 4 months. If patients in the supplemental oxygen group used stationary oxygen concentrators, logs of meter readings were kept as well. The necessary final sample size was calculated using a time to composite event survival model with the use of the log-rank test statistic.
A total of 738 patients were enrolled in the trial between January 2009 and September 2015 and were followed for 1-6 years. A total of 97% of participants had at least 1 year of follow-up. Out of the 738 randomized patients, 133 (18%) had only resting desaturations, 319 (43%) had only exercise-induced desaturations, and 286 (39%) had both resting and exercise-induced desaturations. Baseline characteristics including age, sex, race, smoking status, quality of life scores, resting SpO2, and nadir SpO2 during the 6-minute walk test were similar between the two groups. The only significant difference noted by the authors between the two groups was a lower BODE (body mass index, airflow obstruction, dyspnea, and exercise) index, which was lower in the group with no supplemental oxygen.
In the time-to-event analysis, there was no significant difference between the two groups in the time to death or first hospitalization (hazard ratio, 0.94; 95% confidence interval, 0.79-1.12; P = .52). There were no significant differences in the rates of all hospitalizations (rate ratio, 1.01; 95% CI, 0.91-1.13), COPD exacerbations (RR 1.08; 95% CI, 0.98-1.19), and COPD related hospitalizations (RR, 0.99; 95% CI, 0.83-1.17). There were also no differences between the experimental and control groups in quality of life, lung function, and 6-minute walk distance. There were no significant differences in the subgroups classified by desaturation profile, sex, race, nadir SpO2 during the 6-minute walk test, and forced expiratory volume in 1 second.
The findings in this study show that, in the subgroup of chronic obstructive pulmonary disease patients with stable COPD and moderate resting or exercise-induced desaturation, supplemental oxygen did not affect the time to death or first hospitalization, time to death, time to first hospitalization, time to first COPD exacerbation, time to first hospitalization for a COPD exacerbation, rate of all hospitalizations, rate of all COPD exacerbations, or changes in metrics surrounding quality of life, anxiety/depression, or functional status. This supports earlier studies that demonstrated that long-term treatment with oxygen does not result in longer survival than does no long-term treatment with oxygen in patients with COPD and resting SpO2 of more than 88%.
The results of this study are a departure from previous studies that had shown improved mortality in patients with COPD and severe desaturation who were treated with LTOT. The authors hypothesized that this may have been caused by physiological effects of oxygen saturation on pulmonary vasoconstriction, release of mediators, and ventilator drive, which occur at an O2 saturation of 88% or less and may be more significant in patients with chronic hypoxemia. This trial also contrasted previous studies that had shown that oxygen therapy may reduce dyspnea in COPD patients with mild or no hypoxia because the LOTT trial showed no improvement in quality of life, anxiety, and depression measures in patients treated with long-term oxygen as compared with those treated with no oxygen.
Some limitations of the study included the absence of highly symptomatic patients or patients who the providers believed were too ill to participate, the effect of the unblinded nature of the study on outcomes that were patient reported, the lack of assessment of immediate effects of oxygen on exercise performance or symptoms, possible variability in amount of oxygen delivered, and the fact that patients may have overestimated their oxygen use.
In patients with stable COPD and moderate resting or exercise induced desaturation, long-term supplemental oxygen did not provide any benefit in regard to time until death or first hospitalization or any of the other measured outcomes.
Application of data to the case
Our patient has stable COPD and had only moderate exercise-induced desaturation. Long-term supplemental oxygen would not produce a benefit for him.
This study shows us that it would not increase his survival at this point; however, if he were to have worsening exercise-induced or new resting desaturation at some point in the future, supplemental oxygen would then be beneficial. At this point supplemental oxygen would not even affect his rate of hospitalization for COPD- or non-COPD–related reasons. Perhaps most importantly, adding oxygen therapy would not affect his overall quality of life, including his functional status and mood.
Bottom line
The addition of supplemental oxygen is not helpful for patients with COPD who have chronic stable moderate hypoxia.
Dr. Farber is a medical instructor in the Duke University Health System in Durham, N.C. Dr. Sata is a medical instructor in the Duke University Hospital. Dr. Wachter is an assistant professor of medicine at Duke University. Dr. Sharma is associate medical director for clinical education in hospital medicine at Duke Regional Hospital and an assistant professor of medicine at Duke University.
References
1. Nocturnal Oxygen Therapy Trial Group. Continuous or nocturnal oxygen therapy in hypoxemic chronic obstructive lung disease: A clinical trial. Ann Intern Med. 1980 Sep;93(3):391-8.
2. Medical Research Council Working Party. Long term domiciliary oxygen therapy in chronic hypoxic cor pulmonale complicating chronic bronchitis and emphysema: Report of the Medical Research Council Working Party. Lancet 1981 Mar 28;1(8222):681-6.
3. Long-term oxygen treatment trial research group et al. A randomized trial of long-term oxygen for COPD with moderate desaturation. N Engl J Med. 2016 Oct 27;375(17):1617-27.
Additional reading
Stoller JK et al. Oxygen therapy for patients with COPD: Current evidence and the Long-term Oxygen Treatment Trial. Chest. 2010 July;138:179-87.
Qaseem A et al. Diagnosis and management of stable chronic obstructive pulmonary disease: A clinical practice guideline update from the American College of Physicians, American College of Chest Physicians, American Thoracic Society, and European Respiratory Society. Ann Intern Med. 2011 Aug 2;155(3):179-91.
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Quiz: Does this patient need oxygen?
You are caring for a 72-year-old man with stable COPD who was admitted for cellulitis. He is improving clinically on appropriate antibiotics, and he has been stable on room air every time you examine him. The nurse pages you on the day of discharge – a Sunday – informing you that his oxygen saturation dropped to 88% while he was walking the halls this morning. She asks whether he needs to stay in the hospital so you can arrange home supplemental oxygen therapy. What should you do?
A. Keep him in the hospital until you can arrange home oxygen therapy.
B. Discharge him home Sunday but have the oxygen company go out to his house first thing on Monday.
C. Discharge him home without supplemental oxygen therapy.
D. Check an arterial blood gas to help decide if you should set up oxygen therapy.
The answer is C. He meets the description of stable COPD with mild to moderate exercise-induced desaturation. The LOTT trial supports our clinical decision that he would not benefit from supplemental oxygen therapy at this point.
Key Points
- Long-term oxygen therapy (LTOT) is beneficial in patients with COPD and severe resting hypoxemia (arterial oxygen partial pressure ≤ 55 mm Hg or SpO2 ≤ 88%) and should be prescribed to improve survival in this population.
- Patients with COPD and mild to moderate resting hypoxemia or exercised-induced hypoxemia should not be routinely prescribed LTOT given the associated costs, risks, and burdens and the lack of evidence of benefit.