Key clinical point: Dupilumab rapidly improves the severity of atopic dermatitis (AD) symptoms and shows a favorable safety profile in children with moderate-to-severe AD.

Major finding: Dupilumab significantly reduced the mean Eczema Area and Severity Index (EASI) score at weeks 16, 24, and 52 (all P < .0001) and from weeks 16 to 24 (P < .01) and weeks 16 to 52 (P < .001). By week 52, 86.8% of patients had achieved a ≥ 75% improvement in the EASI score. No serious adverse events were observed, and none of the children discontinued treatment.

Study details: Findings are from a retrospective, observational, real-life study including 96 children (age 6-11 years) with moderate-to-severe AD inadequately controlled with conventional topical therapies who received dupilumab (300 mg on days 1 and 15 and 300 mg every 4 weeks).

Disclosures: This study did not receive any funding. Several authors reported receiving honoraria, travel support, or personal fees from or serving as consultants, investigators, speakers, or advisory board members for or having other ties with various sources.

Source: Patruno C et al. A 52-week multicenter retrospective real-world study on effectiveness and safety of dupilumab in children with atopic dermatitis aged from 6 to 11 years. J Dermatolog Treat. 2023;34:2246602 (Aug 14). doi: 10.1080/09546634.2023.2246602

Key clinical point: Dupilumab rapidly improves the severity of atopic dermatitis (AD) symptoms and shows a favorable safety profile in children with moderate-to-severe AD.

Major finding: Dupilumab significantly reduced the mean Eczema Area and Severity Index (EASI) score at weeks 16, 24, and 52 (all P < .0001) and from weeks 16 to 24 (P < .01) and weeks 16 to 52 (P < .001). By week 52, 86.8% of patients had achieved a ≥ 75% improvement in the EASI score. No serious adverse events were observed, and none of the children discontinued treatment.

Study details: Findings are from a retrospective, observational, real-life study including 96 children (age 6-11 years) with moderate-to-severe AD inadequately controlled with conventional topical therapies who received dupilumab (300 mg on days 1 and 15 and 300 mg every 4 weeks).

Disclosures: This study did not receive any funding. Several authors reported receiving honoraria, travel support, or personal fees from or serving as consultants, investigators, speakers, or advisory board members for or having other ties with various sources.

Source: Patruno C et al. A 52-week multicenter retrospective real-world study on effectiveness and safety of dupilumab in children with atopic dermatitis aged from 6 to 11 years. J Dermatolog Treat. 2023;34:2246602 (Aug 14). doi: 10.1080/09546634.2023.2246602

Key clinical point: Dupilumab rapidly improves the severity of atopic dermatitis (AD) symptoms and shows a favorable safety profile in children with moderate-to-severe AD.

Major finding: Dupilumab significantly reduced the mean Eczema Area and Severity Index (EASI) score at weeks 16, 24, and 52 (all P < .0001) and from weeks 16 to 24 (P < .01) and weeks 16 to 52 (P < .001). By week 52, 86.8% of patients had achieved a ≥ 75% improvement in the EASI score. No serious adverse events were observed, and none of the children discontinued treatment.

Study details: Findings are from a retrospective, observational, real-life study including 96 children (age 6-11 years) with moderate-to-severe AD inadequately controlled with conventional topical therapies who received dupilumab (300 mg on days 1 and 15 and 300 mg every 4 weeks).

Disclosures: This study did not receive any funding. Several authors reported receiving honoraria, travel support, or personal fees from or serving as consultants, investigators, speakers, or advisory board members for or having other ties with various sources.

Source: Patruno C et al. A 52-week multicenter retrospective real-world study on effectiveness and safety of dupilumab in children with atopic dermatitis aged from 6 to 11 years. J Dermatolog Treat. 2023;34:2246602 (Aug 14). doi: 10.1080/09546634.2023.2246602

Key clinical point: Severe atopic dermatitis (AD) is associated with higher risks for venous thromboembolism and cardiovascular diseases in both children and adults.

Major finding: Children with severe AD vs those without AD had a significantly increased risk (adjusted hazard ratio; 95% CI) for cerebrovascular accidents (2.43; 1.13-5.22), diabetes (1.46; 1.06-2.01), and deep vein thrombosis (DVT; 2.13; 1.17-3.87). Among adults, the severe AD vs non-AD group had a significantly higher risk for cerebrovascular accidents (1.21; 1.13-1.30), diabetes (1.15; 1.09-1.22), dyslipidemia (1.11; 1.06-1.17), myocardial infarction (1.27; 1.15-1.39), DVT (1.64; 1.49-1.82), and pulmonary embolism (1.39; 1.21-1.60).

Study details: This population-based cohort study included 409,431 children (age < 18 years) and 625,083 adults with AD who were matched with 1,809,029 children and 2,678,888 adults without AD, respectively.

Disclosures: This study was supported by a contract from Pfizer, Inc. Some authors declared serving as consultants for or receiving research grants, honoraria, or consulting fees from various sources, including Pfizer. AR Lemeshow declared being an employee of Pfizer, Inc.

Source: Wan J, Chiesa Fuxench  ZC, et al. Incidence of cardiovascular disease and venous thromboembolism in patients with atopic dermatitis. J Allergy Clin Immunol Pract. 2023 (Aug 10). doi: 10.1016/j.jaip.2023.08.007

Key clinical point: Severe atopic dermatitis (AD) is associated with higher risks for venous thromboembolism and cardiovascular diseases in both children and adults.

Major finding: Children with severe AD vs those without AD had a significantly increased risk (adjusted hazard ratio; 95% CI) for cerebrovascular accidents (2.43; 1.13-5.22), diabetes (1.46; 1.06-2.01), and deep vein thrombosis (DVT; 2.13; 1.17-3.87). Among adults, the severe AD vs non-AD group had a significantly higher risk for cerebrovascular accidents (1.21; 1.13-1.30), diabetes (1.15; 1.09-1.22), dyslipidemia (1.11; 1.06-1.17), myocardial infarction (1.27; 1.15-1.39), DVT (1.64; 1.49-1.82), and pulmonary embolism (1.39; 1.21-1.60).

Study details: This population-based cohort study included 409,431 children (age < 18 years) and 625,083 adults with AD who were matched with 1,809,029 children and 2,678,888 adults without AD, respectively.

Disclosures: This study was supported by a contract from Pfizer, Inc. Some authors declared serving as consultants for or receiving research grants, honoraria, or consulting fees from various sources, including Pfizer. AR Lemeshow declared being an employee of Pfizer, Inc.

Source: Wan J, Chiesa Fuxench  ZC, et al. Incidence of cardiovascular disease and venous thromboembolism in patients with atopic dermatitis. J Allergy Clin Immunol Pract. 2023 (Aug 10). doi: 10.1016/j.jaip.2023.08.007

Key clinical point: Severe atopic dermatitis (AD) is associated with higher risks for venous thromboembolism and cardiovascular diseases in both children and adults.

Major finding: Children with severe AD vs those without AD had a significantly increased risk (adjusted hazard ratio; 95% CI) for cerebrovascular accidents (2.43; 1.13-5.22), diabetes (1.46; 1.06-2.01), and deep vein thrombosis (DVT; 2.13; 1.17-3.87). Among adults, the severe AD vs non-AD group had a significantly higher risk for cerebrovascular accidents (1.21; 1.13-1.30), diabetes (1.15; 1.09-1.22), dyslipidemia (1.11; 1.06-1.17), myocardial infarction (1.27; 1.15-1.39), DVT (1.64; 1.49-1.82), and pulmonary embolism (1.39; 1.21-1.60).

Study details: This population-based cohort study included 409,431 children (age < 18 years) and 625,083 adults with AD who were matched with 1,809,029 children and 2,678,888 adults without AD, respectively.

Disclosures: This study was supported by a contract from Pfizer, Inc. Some authors declared serving as consultants for or receiving research grants, honoraria, or consulting fees from various sources, including Pfizer. AR Lemeshow declared being an employee of Pfizer, Inc.

Source: Wan J, Chiesa Fuxench  ZC, et al. Incidence of cardiovascular disease and venous thromboembolism in patients with atopic dermatitis. J Allergy Clin Immunol Pract. 2023 (Aug 10). doi: 10.1016/j.jaip.2023.08.007

Key clinical point: Adults with newly diagnosed atopic dermatitis (AD) have a 44% increased risk of subsequently developing type 2 diabetes (T2D).

Major finding: The risk for new-onset T2D was significantly higher in adults with newly diagnosed AD vs control individuals without AD (adjusted hazard ratio 1.44; P < .001), with the risk being significantly greater in both men and women with AD (both P < .001).

Study details: Findings are from a retrospective cohort study including 36,692 adult patients with AD and 36,692 matched control individuals who had never been diagnosed with AD.

Disclosures: This study was supported by the National Research Foundation of Korea funded by the Ministry of Education, Science, and Technology and others. The authors declared no conflicts of interest.

Source: Won Lee S et al. Risk of type 2 diabetes mellitus in adult patients with atopic dermatitis. Diabetes Res Clin Pract. 2023;110883 (Aug 16). doi: 10.1016/j.diabres.2023.110883

Key clinical point: Adults with newly diagnosed atopic dermatitis (AD) have a 44% increased risk of subsequently developing type 2 diabetes (T2D).

Major finding: The risk for new-onset T2D was significantly higher in adults with newly diagnosed AD vs control individuals without AD (adjusted hazard ratio 1.44; P < .001), with the risk being significantly greater in both men and women with AD (both P < .001).

Study details: Findings are from a retrospective cohort study including 36,692 adult patients with AD and 36,692 matched control individuals who had never been diagnosed with AD.

Disclosures: This study was supported by the National Research Foundation of Korea funded by the Ministry of Education, Science, and Technology and others. The authors declared no conflicts of interest.

Source: Won Lee S et al. Risk of type 2 diabetes mellitus in adult patients with atopic dermatitis. Diabetes Res Clin Pract. 2023;110883 (Aug 16). doi: 10.1016/j.diabres.2023.110883

Key clinical point: Adults with newly diagnosed atopic dermatitis (AD) have a 44% increased risk of subsequently developing type 2 diabetes (T2D).

Major finding: The risk for new-onset T2D was significantly higher in adults with newly diagnosed AD vs control individuals without AD (adjusted hazard ratio 1.44; P < .001), with the risk being significantly greater in both men and women with AD (both P < .001).

Study details: Findings are from a retrospective cohort study including 36,692 adult patients with AD and 36,692 matched control individuals who had never been diagnosed with AD.

Disclosures: This study was supported by the National Research Foundation of Korea funded by the Ministry of Education, Science, and Technology and others. The authors declared no conflicts of interest.

Source: Won Lee S et al. Risk of type 2 diabetes mellitus in adult patients with atopic dermatitis. Diabetes Res Clin Pract. 2023;110883 (Aug 16). doi: 10.1016/j.diabres.2023.110883

The years go by, and nothing much changes: The first 2 weeks of the new school year have brought with them a rise in emergency department (ED) admissions for asthma in patients under age 15 years. A more relaxed approach to maintenance therapy for the condition over the summer holidays, exposure to allergens at school, and the surge in viral respiratory infections that accompanies the return to group settings explain this trend, which can be foreseen.

As soon as September begins, asthma cases increase rapidly in children. According to Public Health France, which has just relaunched its epidemiological monitoring, these cases reach their peak around 2 weeks after the start of the new term.

In its first weekly review on Aug. 22, 2023, the authority reported a slight uptick in cases in its Indian Ocean overseas departments, and the calm before the storm in mainland France.

Last year, between weeks 35 and 36, the increases were 82% for SOS Médecins (the French home doctor visit service), 169% for EDs, and 33% for hospital admissions.

These data are similar to the figures obtained over the past 3 years. The authors of this monitoring, using the SurSaUD system, France’s program for monitoring emergency cases and deaths, attribute these increases to the surge in viral respiratory infections seen after the return to group settings after the school summer holidays.

Indeed, viral-induced exacerbations are mostly caused by rhinoviruses, which circulate throughout the year, but more so during the autumn and winter months. These are probably the main culprits behind the epidemics seen once schools have reopened. Yet relaxation of maintenance asthma treatment (inhaled corticosteroids alone or in combination with long-acting bronchodilators) during the summer holidays also plays a significant role in this yearly recurrence.
 

Compliance ends with school

Flore Amat, MD, PhD, pediatric respiratory and allergy specialist and coordinating doctor at the Zephyr asthma clinic (Robert-Debré Hospital, Paris Public Hospitals) acknowledged, “The summer holidays are often a time when compliance with maintenance therapy is relaxed.” Aware of this fact, doctors prefer to strike a deal with their young patients. “For some of our young and teenage asthma patients, we support their relaxed approach to medication during the summer holidays,” she admitted. “In July and August, there are fewer viruses circulating, and the weather is often dry, which limits the risk of an asthma attack, meaning we can ease off the maintenance therapy, or even stop taking it altogether. We tell parents and children to start taking them again 2 weeks before school starts; 2 weeks being the minimum time needed for inhaled corticosteroids to start taking effect again.” Unsurprisingly, some forget to do so or simply don’t.

Two other things contribute to the rise in asthma attacks in children in early September. The first relates to exposure to allergens, especially dust mites. “Ninety percent of asthmatic children are allergic,” said Frédéric le Guillou, MD, respiratory medicine specialist and chair of the French Society for Respiratory Health, an organization aimed at patients and health care professionals. “Don’t forget that asthma is the leading chronic condition in childhood, with a prevalence estimated at between 8% and 10% of children and adolescents. So, we’re talking about considerable numbers of children being affected.”

Although dust mites are a year-round problem, their peak period of reproduction mainly occurs during the wetter months (March to April and September to November). This means that there is a risk of relapse in asthmatic children who are allergic to dust mites when school starts again after the summer holidays. “In such children, any signs of unmanageable allergic rhinitis should be examined,” said Dr. Amat, “these signs being permanent nasal congestion, runny nose, et cetera.”

Finally, we can also add “the stress and anxiety generated by the school setting and settling back into a routine” to the list of likely explanations for this peak in asthma attacks, Dr. Amat concluded.
 

Check-up time

Children and teenagers with asthma should have a check-up with their respiratory medicine specialists at the start of the new term to confirm that their condition is under control and to determine whether any changes need to be made to their maintenance therapy. “Looking back at previous Septembers and winters is informative in adapting a patient’s treatment plan,” said Dr. Amat. “If maintenance therapy has been stopped during the summer, take the opportunity to represcribe it or modify it if, for example, the dose of inhaled corticosteroids has not been enough to prevent attacks in years gone by. Adequate control of symptoms over the summer months suggests that treatment should be bolstered with preventive therapy to cope with the autumn and winter months. Finally, the factors aggravating poor management of asthma should be dealt with, such as intranasal antihistamines and corticosteroids in allergic rhinitis, specific immunotherapy in patients with controlled asthma but with significant allergy symptoms.”

The start-of-term visit to the doctor’s office is also the perfect opportunity to carry out respiratory function testing (RFT), if this has not been done for over a year in patients whose asthma is well managed. “RFT is indicated in the 3 months following any changes to maintenance therapy, every 3 to 6 months in patients with poorly controlled asthma, and after stopping maintenance therapy or when considering stopping treatment permanently or for an extended period of time,” noted Dr. Amat.

The distinction between difficult asthma (suboptimal treatment plan, poor compliance, persisting allergen exposure, etc.) and severe asthma may be made during this back-to-school asthma review. In specialist clinics, children with severe asthma (not controlled by combined treatment with maximum-dose corticosteroids and maximum-dose bronchodilators) may, like adults, benefit from some biotherapies.
 

Commentary from Madiha Ellaffi, MD, respiratory medicine specialist

When children experience relatively calm summers without seasonal summer allergies to certain pollens or molds (such as Alternaria, some grasses, etc.) that require maintenance therapy to be continued, we know full well that compliance is often left up to the child. What would be better would be striking a “deal” with these young people: Maintenance treatment can be stopped over the summer, providing that their usual dose is quite low or their asthma is considered mild to moderate, but it must be restarted before going back to school in September. An action plan should be discussed in the event of an asthma attack, and treatment bolstered to overcome this hurdle, should it occur, such as double inhaled corticosteroid doses, etc. Indeed, this period is conducive to asthma exacerbations due to stress, the return of students to confined classrooms, pollutants released by the deep cleaning of school buildings that occurs at the start of term (particularly the release of volatile organic compounds that irritate the airways), and the lack of ventilation in classrooms, which is conducive to the spread of viruses that can cause worsening asthma symptoms. I’d also like to remind parents of the importance of detecting early symptoms (such as wheezing, cough, bronchitis, itchy throat and nose, etc.) in warding off asthma attacks or severe symptoms. I insist on basic measures, such as nasal irrigation, treating allergic rhinitis, which can exacerbate asthma, and ensuring good habits at home to prevent dust mites and mold, such as vacuuming, airing houses, etc. It is sensible to assess the risk of asthma attacks at the start of term according to the child’s allergy profile and their previous history, like starting treatment for allergic rhinitis if not already being taken.

This article was translated from Medscape’s French edition. A version of this article appeared on Medscape.com.

The years go by, and nothing much changes: The first 2 weeks of the new school year have brought with them a rise in emergency department (ED) admissions for asthma in patients under age 15 years. A more relaxed approach to maintenance therapy for the condition over the summer holidays, exposure to allergens at school, and the surge in viral respiratory infections that accompanies the return to group settings explain this trend, which can be foreseen.

As soon as September begins, asthma cases increase rapidly in children. According to Public Health France, which has just relaunched its epidemiological monitoring, these cases reach their peak around 2 weeks after the start of the new term.

In its first weekly review on Aug. 22, 2023, the authority reported a slight uptick in cases in its Indian Ocean overseas departments, and the calm before the storm in mainland France.

Last year, between weeks 35 and 36, the increases were 82% for SOS Médecins (the French home doctor visit service), 169% for EDs, and 33% for hospital admissions.

These data are similar to the figures obtained over the past 3 years. The authors of this monitoring, using the SurSaUD system, France’s program for monitoring emergency cases and deaths, attribute these increases to the surge in viral respiratory infections seen after the return to group settings after the school summer holidays.

Indeed, viral-induced exacerbations are mostly caused by rhinoviruses, which circulate throughout the year, but more so during the autumn and winter months. These are probably the main culprits behind the epidemics seen once schools have reopened. Yet relaxation of maintenance asthma treatment (inhaled corticosteroids alone or in combination with long-acting bronchodilators) during the summer holidays also plays a significant role in this yearly recurrence.
 

Compliance ends with school

Flore Amat, MD, PhD, pediatric respiratory and allergy specialist and coordinating doctor at the Zephyr asthma clinic (Robert-Debré Hospital, Paris Public Hospitals) acknowledged, “The summer holidays are often a time when compliance with maintenance therapy is relaxed.” Aware of this fact, doctors prefer to strike a deal with their young patients. “For some of our young and teenage asthma patients, we support their relaxed approach to medication during the summer holidays,” she admitted. “In July and August, there are fewer viruses circulating, and the weather is often dry, which limits the risk of an asthma attack, meaning we can ease off the maintenance therapy, or even stop taking it altogether. We tell parents and children to start taking them again 2 weeks before school starts; 2 weeks being the minimum time needed for inhaled corticosteroids to start taking effect again.” Unsurprisingly, some forget to do so or simply don’t.

Two other things contribute to the rise in asthma attacks in children in early September. The first relates to exposure to allergens, especially dust mites. “Ninety percent of asthmatic children are allergic,” said Frédéric le Guillou, MD, respiratory medicine specialist and chair of the French Society for Respiratory Health, an organization aimed at patients and health care professionals. “Don’t forget that asthma is the leading chronic condition in childhood, with a prevalence estimated at between 8% and 10% of children and adolescents. So, we’re talking about considerable numbers of children being affected.”

Although dust mites are a year-round problem, their peak period of reproduction mainly occurs during the wetter months (March to April and September to November). This means that there is a risk of relapse in asthmatic children who are allergic to dust mites when school starts again after the summer holidays. “In such children, any signs of unmanageable allergic rhinitis should be examined,” said Dr. Amat, “these signs being permanent nasal congestion, runny nose, et cetera.”

Finally, we can also add “the stress and anxiety generated by the school setting and settling back into a routine” to the list of likely explanations for this peak in asthma attacks, Dr. Amat concluded.
 

Check-up time

Children and teenagers with asthma should have a check-up with their respiratory medicine specialists at the start of the new term to confirm that their condition is under control and to determine whether any changes need to be made to their maintenance therapy. “Looking back at previous Septembers and winters is informative in adapting a patient’s treatment plan,” said Dr. Amat. “If maintenance therapy has been stopped during the summer, take the opportunity to represcribe it or modify it if, for example, the dose of inhaled corticosteroids has not been enough to prevent attacks in years gone by. Adequate control of symptoms over the summer months suggests that treatment should be bolstered with preventive therapy to cope with the autumn and winter months. Finally, the factors aggravating poor management of asthma should be dealt with, such as intranasal antihistamines and corticosteroids in allergic rhinitis, specific immunotherapy in patients with controlled asthma but with significant allergy symptoms.”

The start-of-term visit to the doctor’s office is also the perfect opportunity to carry out respiratory function testing (RFT), if this has not been done for over a year in patients whose asthma is well managed. “RFT is indicated in the 3 months following any changes to maintenance therapy, every 3 to 6 months in patients with poorly controlled asthma, and after stopping maintenance therapy or when considering stopping treatment permanently or for an extended period of time,” noted Dr. Amat.

The distinction between difficult asthma (suboptimal treatment plan, poor compliance, persisting allergen exposure, etc.) and severe asthma may be made during this back-to-school asthma review. In specialist clinics, children with severe asthma (not controlled by combined treatment with maximum-dose corticosteroids and maximum-dose bronchodilators) may, like adults, benefit from some biotherapies.
 

Commentary from Madiha Ellaffi, MD, respiratory medicine specialist

When children experience relatively calm summers without seasonal summer allergies to certain pollens or molds (such as Alternaria, some grasses, etc.) that require maintenance therapy to be continued, we know full well that compliance is often left up to the child. What would be better would be striking a “deal” with these young people: Maintenance treatment can be stopped over the summer, providing that their usual dose is quite low or their asthma is considered mild to moderate, but it must be restarted before going back to school in September. An action plan should be discussed in the event of an asthma attack, and treatment bolstered to overcome this hurdle, should it occur, such as double inhaled corticosteroid doses, etc. Indeed, this period is conducive to asthma exacerbations due to stress, the return of students to confined classrooms, pollutants released by the deep cleaning of school buildings that occurs at the start of term (particularly the release of volatile organic compounds that irritate the airways), and the lack of ventilation in classrooms, which is conducive to the spread of viruses that can cause worsening asthma symptoms. I’d also like to remind parents of the importance of detecting early symptoms (such as wheezing, cough, bronchitis, itchy throat and nose, etc.) in warding off asthma attacks or severe symptoms. I insist on basic measures, such as nasal irrigation, treating allergic rhinitis, which can exacerbate asthma, and ensuring good habits at home to prevent dust mites and mold, such as vacuuming, airing houses, etc. It is sensible to assess the risk of asthma attacks at the start of term according to the child’s allergy profile and their previous history, like starting treatment for allergic rhinitis if not already being taken.

This article was translated from Medscape’s French edition. A version of this article appeared on Medscape.com.

The years go by, and nothing much changes: The first 2 weeks of the new school year have brought with them a rise in emergency department (ED) admissions for asthma in patients under age 15 years. A more relaxed approach to maintenance therapy for the condition over the summer holidays, exposure to allergens at school, and the surge in viral respiratory infections that accompanies the return to group settings explain this trend, which can be foreseen.

As soon as September begins, asthma cases increase rapidly in children. According to Public Health France, which has just relaunched its epidemiological monitoring, these cases reach their peak around 2 weeks after the start of the new term.

In its first weekly review on Aug. 22, 2023, the authority reported a slight uptick in cases in its Indian Ocean overseas departments, and the calm before the storm in mainland France.

Last year, between weeks 35 and 36, the increases were 82% for SOS Médecins (the French home doctor visit service), 169% for EDs, and 33% for hospital admissions.

These data are similar to the figures obtained over the past 3 years. The authors of this monitoring, using the SurSaUD system, France’s program for monitoring emergency cases and deaths, attribute these increases to the surge in viral respiratory infections seen after the return to group settings after the school summer holidays.

Indeed, viral-induced exacerbations are mostly caused by rhinoviruses, which circulate throughout the year, but more so during the autumn and winter months. These are probably the main culprits behind the epidemics seen once schools have reopened. Yet relaxation of maintenance asthma treatment (inhaled corticosteroids alone or in combination with long-acting bronchodilators) during the summer holidays also plays a significant role in this yearly recurrence.
 

Compliance ends with school

Flore Amat, MD, PhD, pediatric respiratory and allergy specialist and coordinating doctor at the Zephyr asthma clinic (Robert-Debré Hospital, Paris Public Hospitals) acknowledged, “The summer holidays are often a time when compliance with maintenance therapy is relaxed.” Aware of this fact, doctors prefer to strike a deal with their young patients. “For some of our young and teenage asthma patients, we support their relaxed approach to medication during the summer holidays,” she admitted. “In July and August, there are fewer viruses circulating, and the weather is often dry, which limits the risk of an asthma attack, meaning we can ease off the maintenance therapy, or even stop taking it altogether. We tell parents and children to start taking them again 2 weeks before school starts; 2 weeks being the minimum time needed for inhaled corticosteroids to start taking effect again.” Unsurprisingly, some forget to do so or simply don’t.

Two other things contribute to the rise in asthma attacks in children in early September. The first relates to exposure to allergens, especially dust mites. “Ninety percent of asthmatic children are allergic,” said Frédéric le Guillou, MD, respiratory medicine specialist and chair of the French Society for Respiratory Health, an organization aimed at patients and health care professionals. “Don’t forget that asthma is the leading chronic condition in childhood, with a prevalence estimated at between 8% and 10% of children and adolescents. So, we’re talking about considerable numbers of children being affected.”

Although dust mites are a year-round problem, their peak period of reproduction mainly occurs during the wetter months (March to April and September to November). This means that there is a risk of relapse in asthmatic children who are allergic to dust mites when school starts again after the summer holidays. “In such children, any signs of unmanageable allergic rhinitis should be examined,” said Dr. Amat, “these signs being permanent nasal congestion, runny nose, et cetera.”

Finally, we can also add “the stress and anxiety generated by the school setting and settling back into a routine” to the list of likely explanations for this peak in asthma attacks, Dr. Amat concluded.
 

Check-up time

Children and teenagers with asthma should have a check-up with their respiratory medicine specialists at the start of the new term to confirm that their condition is under control and to determine whether any changes need to be made to their maintenance therapy. “Looking back at previous Septembers and winters is informative in adapting a patient’s treatment plan,” said Dr. Amat. “If maintenance therapy has been stopped during the summer, take the opportunity to represcribe it or modify it if, for example, the dose of inhaled corticosteroids has not been enough to prevent attacks in years gone by. Adequate control of symptoms over the summer months suggests that treatment should be bolstered with preventive therapy to cope with the autumn and winter months. Finally, the factors aggravating poor management of asthma should be dealt with, such as intranasal antihistamines and corticosteroids in allergic rhinitis, specific immunotherapy in patients with controlled asthma but with significant allergy symptoms.”

The start-of-term visit to the doctor’s office is also the perfect opportunity to carry out respiratory function testing (RFT), if this has not been done for over a year in patients whose asthma is well managed. “RFT is indicated in the 3 months following any changes to maintenance therapy, every 3 to 6 months in patients with poorly controlled asthma, and after stopping maintenance therapy or when considering stopping treatment permanently or for an extended period of time,” noted Dr. Amat.

The distinction between difficult asthma (suboptimal treatment plan, poor compliance, persisting allergen exposure, etc.) and severe asthma may be made during this back-to-school asthma review. In specialist clinics, children with severe asthma (not controlled by combined treatment with maximum-dose corticosteroids and maximum-dose bronchodilators) may, like adults, benefit from some biotherapies.
 

Commentary from Madiha Ellaffi, MD, respiratory medicine specialist

When children experience relatively calm summers without seasonal summer allergies to certain pollens or molds (such as Alternaria, some grasses, etc.) that require maintenance therapy to be continued, we know full well that compliance is often left up to the child. What would be better would be striking a “deal” with these young people: Maintenance treatment can be stopped over the summer, providing that their usual dose is quite low or their asthma is considered mild to moderate, but it must be restarted before going back to school in September. An action plan should be discussed in the event of an asthma attack, and treatment bolstered to overcome this hurdle, should it occur, such as double inhaled corticosteroid doses, etc. Indeed, this period is conducive to asthma exacerbations due to stress, the return of students to confined classrooms, pollutants released by the deep cleaning of school buildings that occurs at the start of term (particularly the release of volatile organic compounds that irritate the airways), and the lack of ventilation in classrooms, which is conducive to the spread of viruses that can cause worsening asthma symptoms. I’d also like to remind parents of the importance of detecting early symptoms (such as wheezing, cough, bronchitis, itchy throat and nose, etc.) in warding off asthma attacks or severe symptoms. I insist on basic measures, such as nasal irrigation, treating allergic rhinitis, which can exacerbate asthma, and ensuring good habits at home to prevent dust mites and mold, such as vacuuming, airing houses, etc. It is sensible to assess the risk of asthma attacks at the start of term according to the child’s allergy profile and their previous history, like starting treatment for allergic rhinitis if not already being taken.

This article was translated from Medscape’s French edition. A version of this article appeared on Medscape.com.

Key clinical point: Tralokinumab is well tolerated and effective in older adults with moderate-to-severe atopic dermatitis (AD).

Major finding: Compared with the placebo group, a significantly higher proportion of patients in ECZTRA 1 and 2 achieved ≥ 75% improvement in the Eczema Area and Severity Index score (33.9% vs 4.76%; P < .001) and an Investigator’s Global Assessment 0 or 1 score (16.95% vs 0%; P < .001) in the tralokinumab group at week 16. The adverse event (AE) rate was comparable between the groups; however, fewer patients discontinued treatment due to AE in the tralokinumab vs placebo group (5.3% vs 6.9%).

Study details: Findings are from a post hoc analysis of ECZTRA 1, 2, and 3 trials and included 104 older patients (≥65 years) with AD who received tralokinumab (n = 75) or placebo (n = 29).

Disclosures: This study was supported by LEO Pharma. Two authors declared being employees of LEO Pharma. Some authors declared receiving grants, personal fees, or consulting fees from LEO Pharma and other sources.

Source: Merola JF et al. Safety and efficacy of tralokinumab in older adults with moderate-to-severe atopic dermatitis: A secondary analysis. JAMA Dermatol. 2023 (Aug 23). doi: 10.1001/jamadermatol.2023.2626

Key clinical point: Tralokinumab is well tolerated and effective in older adults with moderate-to-severe atopic dermatitis (AD).

Major finding: Compared with the placebo group, a significantly higher proportion of patients in ECZTRA 1 and 2 achieved ≥ 75% improvement in the Eczema Area and Severity Index score (33.9% vs 4.76%; P < .001) and an Investigator’s Global Assessment 0 or 1 score (16.95% vs 0%; P < .001) in the tralokinumab group at week 16. The adverse event (AE) rate was comparable between the groups; however, fewer patients discontinued treatment due to AE in the tralokinumab vs placebo group (5.3% vs 6.9%).

Study details: Findings are from a post hoc analysis of ECZTRA 1, 2, and 3 trials and included 104 older patients (≥65 years) with AD who received tralokinumab (n = 75) or placebo (n = 29).

Disclosures: This study was supported by LEO Pharma. Two authors declared being employees of LEO Pharma. Some authors declared receiving grants, personal fees, or consulting fees from LEO Pharma and other sources.

Source: Merola JF et al. Safety and efficacy of tralokinumab in older adults with moderate-to-severe atopic dermatitis: A secondary analysis. JAMA Dermatol. 2023 (Aug 23). doi: 10.1001/jamadermatol.2023.2626

Key clinical point: Tralokinumab is well tolerated and effective in older adults with moderate-to-severe atopic dermatitis (AD).

Major finding: Compared with the placebo group, a significantly higher proportion of patients in ECZTRA 1 and 2 achieved ≥ 75% improvement in the Eczema Area and Severity Index score (33.9% vs 4.76%; P < .001) and an Investigator’s Global Assessment 0 or 1 score (16.95% vs 0%; P < .001) in the tralokinumab group at week 16. The adverse event (AE) rate was comparable between the groups; however, fewer patients discontinued treatment due to AE in the tralokinumab vs placebo group (5.3% vs 6.9%).

Study details: Findings are from a post hoc analysis of ECZTRA 1, 2, and 3 trials and included 104 older patients (≥65 years) with AD who received tralokinumab (n = 75) or placebo (n = 29).

Disclosures: This study was supported by LEO Pharma. Two authors declared being employees of LEO Pharma. Some authors declared receiving grants, personal fees, or consulting fees from LEO Pharma and other sources.

Source: Merola JF et al. Safety and efficacy of tralokinumab in older adults with moderate-to-severe atopic dermatitis: A secondary analysis. JAMA Dermatol. 2023 (Aug 23). doi: 10.1001/jamadermatol.2023.2626

Key clinical point: Children and adults with atopic dermatitis (AD) have a significantly increased risk of developing inflammatory bowel disease (IBD), including Crohn’s disease (CD).

Major finding: Children with vs without AD had a significantly higher risk for IBD (adjusted HR [aHR] 1.44; 95% CI 1.31-1.58) and CD (aHR 1.74; 95% CI 1.54-1.97), but the risk for ulcerative colitis (UC; aHR 1.65; 95% CI 1.02-2.67) was higher only in children with severe AD. Adults with vs without AD had a significantly increased risk for IBD (aHR 1.34; 95% CI 1.27-1.40), CD (aHR 1.36; 95% CI 1.26-1.47), and UC (aHR 1.32; 95% CI 1.24-1.41).

Study details: This population-based cohort study matched children (n = 409,431; age < 18 years) and adults (n = 625,083) with AD with control children (n = 1,809,029) and adults (n = 2,678,888) without AD, respectively.

Disclosures: This study was supported by a contract from Pfizer Inc. Five authors declared receiving grants, personal fees, and fellowship funding from various sources, including Pfizer Inc.

Source: Chiesa Fuxench ZC et al. Risk of inflammatory bowel disease in patients with atopic dermatitis. JAMA Dermatol. 2023 (Aug 30). doi: 10.1001/jamadermatol.2023.2875

Key clinical point: Children and adults with atopic dermatitis (AD) have a significantly increased risk of developing inflammatory bowel disease (IBD), including Crohn’s disease (CD).

Major finding: Children with vs without AD had a significantly higher risk for IBD (adjusted HR [aHR] 1.44; 95% CI 1.31-1.58) and CD (aHR 1.74; 95% CI 1.54-1.97), but the risk for ulcerative colitis (UC; aHR 1.65; 95% CI 1.02-2.67) was higher only in children with severe AD. Adults with vs without AD had a significantly increased risk for IBD (aHR 1.34; 95% CI 1.27-1.40), CD (aHR 1.36; 95% CI 1.26-1.47), and UC (aHR 1.32; 95% CI 1.24-1.41).

Study details: This population-based cohort study matched children (n = 409,431; age < 18 years) and adults (n = 625,083) with AD with control children (n = 1,809,029) and adults (n = 2,678,888) without AD, respectively.

Disclosures: This study was supported by a contract from Pfizer Inc. Five authors declared receiving grants, personal fees, and fellowship funding from various sources, including Pfizer Inc.

Source: Chiesa Fuxench ZC et al. Risk of inflammatory bowel disease in patients with atopic dermatitis. JAMA Dermatol. 2023 (Aug 30). doi: 10.1001/jamadermatol.2023.2875

Key clinical point: Children and adults with atopic dermatitis (AD) have a significantly increased risk of developing inflammatory bowel disease (IBD), including Crohn’s disease (CD).

Major finding: Children with vs without AD had a significantly higher risk for IBD (adjusted HR [aHR] 1.44; 95% CI 1.31-1.58) and CD (aHR 1.74; 95% CI 1.54-1.97), but the risk for ulcerative colitis (UC; aHR 1.65; 95% CI 1.02-2.67) was higher only in children with severe AD. Adults with vs without AD had a significantly increased risk for IBD (aHR 1.34; 95% CI 1.27-1.40), CD (aHR 1.36; 95% CI 1.26-1.47), and UC (aHR 1.32; 95% CI 1.24-1.41).

Study details: This population-based cohort study matched children (n = 409,431; age < 18 years) and adults (n = 625,083) with AD with control children (n = 1,809,029) and adults (n = 2,678,888) without AD, respectively.

Disclosures: This study was supported by a contract from Pfizer Inc. Five authors declared receiving grants, personal fees, and fellowship funding from various sources, including Pfizer Inc.

Source: Chiesa Fuxench ZC et al. Risk of inflammatory bowel disease in patients with atopic dermatitis. JAMA Dermatol. 2023 (Aug 30). doi: 10.1001/jamadermatol.2023.2875

Key clinical point: Dupilumab significantly improved the overall sleep continuity and quality, itch, and other signs and symptoms of atopic dermatitis (AD) in patients with moderate-to-severe AD.

Major finding: At week 12, dupilumab vs placebo led to a significant improvement in the sleep Numeric Rating Scale (NRS), peak pruritus NRS, SCORing Atopic Dermatitis (SCORAD), SCORAD sleep visual analog scale, Eczema Area and Severity Index, Epworth Sleepiness Scale, and sleep-related impairment T-scores (all P < .001) and a lower overall treatment-emergent adverse event rate (56.7% vs 67.2%).

Study details: Findings are from the prospective phase 4 DUPISTAD study including patients with moderate-to-severe AD who were randomly assigned to receive 300 mg dupilumab every 2 weeks (n = 127) or placebo (n = 61).

Disclosures: This study was sponsored by Sanofi and Regeneron Pharmaceuticals Inc. Three authors declared being employees of and holding stocks or stock options in Sanofi or Regeneron. Other authors declared receiving grant support, travel grants, or speaker fees from or serving as principal investigators, advisory board members, or consultants for various sources.

Source: Merola JF et al. Dupilumab significantly improves sleep in adults with atopic dermatitis: Results from the 12-week placebo-controlled period of the 24-week phase 4 randomized double-blinded placebo-controlled DUPISTAD study. Br J Dermatol. 2023 (Aug 10). doi: 10.1093/bjd/ljad284

Key clinical point: Dupilumab significantly improved the overall sleep continuity and quality, itch, and other signs and symptoms of atopic dermatitis (AD) in patients with moderate-to-severe AD.

Major finding: At week 12, dupilumab vs placebo led to a significant improvement in the sleep Numeric Rating Scale (NRS), peak pruritus NRS, SCORing Atopic Dermatitis (SCORAD), SCORAD sleep visual analog scale, Eczema Area and Severity Index, Epworth Sleepiness Scale, and sleep-related impairment T-scores (all P < .001) and a lower overall treatment-emergent adverse event rate (56.7% vs 67.2%).

Study details: Findings are from the prospective phase 4 DUPISTAD study including patients with moderate-to-severe AD who were randomly assigned to receive 300 mg dupilumab every 2 weeks (n = 127) or placebo (n = 61).

Disclosures: This study was sponsored by Sanofi and Regeneron Pharmaceuticals Inc. Three authors declared being employees of and holding stocks or stock options in Sanofi or Regeneron. Other authors declared receiving grant support, travel grants, or speaker fees from or serving as principal investigators, advisory board members, or consultants for various sources.

Source: Merola JF et al. Dupilumab significantly improves sleep in adults with atopic dermatitis: Results from the 12-week placebo-controlled period of the 24-week phase 4 randomized double-blinded placebo-controlled DUPISTAD study. Br J Dermatol. 2023 (Aug 10). doi: 10.1093/bjd/ljad284

Key clinical point: Dupilumab significantly improved the overall sleep continuity and quality, itch, and other signs and symptoms of atopic dermatitis (AD) in patients with moderate-to-severe AD.

Major finding: At week 12, dupilumab vs placebo led to a significant improvement in the sleep Numeric Rating Scale (NRS), peak pruritus NRS, SCORing Atopic Dermatitis (SCORAD), SCORAD sleep visual analog scale, Eczema Area and Severity Index, Epworth Sleepiness Scale, and sleep-related impairment T-scores (all P < .001) and a lower overall treatment-emergent adverse event rate (56.7% vs 67.2%).

Study details: Findings are from the prospective phase 4 DUPISTAD study including patients with moderate-to-severe AD who were randomly assigned to receive 300 mg dupilumab every 2 weeks (n = 127) or placebo (n = 61).

Disclosures: This study was sponsored by Sanofi and Regeneron Pharmaceuticals Inc. Three authors declared being employees of and holding stocks or stock options in Sanofi or Regeneron. Other authors declared receiving grant support, travel grants, or speaker fees from or serving as principal investigators, advisory board members, or consultants for various sources.

Source: Merola JF et al. Dupilumab significantly improves sleep in adults with atopic dermatitis: Results from the 12-week placebo-controlled period of the 24-week phase 4 randomized double-blinded placebo-controlled DUPISTAD study. Br J Dermatol. 2023 (Aug 10). doi: 10.1093/bjd/ljad284

In June 2023, a multisociety group agreed to change the name of the disease formerly known as nonalcoholic fatty liver disease (NAFLD) to metabolic dysfunction–associated steatotic liver disease (MASLD).

In the week leading up to this, Intercept Pharmaceuticals announced that their program to develop obeticholic acid (OCA) as a first-to-market, long-awaited potential drug therapy for metabolic dysfunction–associated steatohepatitis (MASH), the more pathogenic form of this disease, was being abandoned after the Food and Drug Administration Gastrointestinal Drugs Advisory Committee voted against accelerated approval of the drug. This decision was made in large part owing to data showing modest efficacy coupled with multiple drug-related side effects, including worsening metabolic dysfunction.

The juxtaposition of these two events highlights what could be a fundamental change in perspective when it comes to the management of patients with MASLD and the development of therapies for this disease.
 

A more precise nomenclature for a broad-spectrum disease

The updating of the NAFLD nomenclature to MASLD represents an important change in the way that the medical community is being asked to view and approach the disease of “fatty liver.”

The previous nomenclature clarified that pathogenic liver steatosis can exist in the absence of alcohol. However, it failed to define the primary drivers of the most common form of NAFLD: that is, metabolic syndrome diseases. The new nomenclature not only better defines the broad spectrum of steatotic liver diseases (alcohol, metabolic, drug/genetic, cryptogenic) but also refocuses the attention of providers on the fundamental basis of MASLD as a critical member of the metabolic syndrome spectrum of diseases.

This is in line with the recently updated American Association for the Study of Liver Disease Practice Guidelines for NAFLD (now MASLD), which focus on active screening to identify patients at risk for advanced MASLD, in particular those with medically complicated obesity or high-risk metabolic characteristics such as diabetes. This is a change from previous versions of the guidelines that were cautious to recommend broad screening guidelines in part owing to the lack of “available therapies.”

The increasing clinical burden of MASH has led to the recognition that patients do not have the luxury of waiting for “anticipated therapies” that have frequently shown only marginal or insignificant efficacy. As a result, some providers have refocused their efforts on the development of care pathways that can efficiently provide comprehensive lifestyle interventions to treat MASH and related metabolic comorbidities within hepatology or other subspecialty clinics.
 

Learning from OCA’s limitations

The approach to drug development for MASH seems to be shifting in a similar, metabolic syndrome–focused way.

The risk-benefit analysis from the FDA advisory committee evaluating OCA noted that the drug provided only modest (albeit statistically significant) benefits over placebo in achieving one of two primary clinical endpoints (improvement in fibrosis by one stage without worsening of NASH), but was associated with toxicity as well as significant drug-related side effects, including new or worsening dyslipidemia, accelerated progression to prediabetes or diabetes, and worsening glycemic control in patients with diabetes.

Worsening clinical markers of metabolic health were an important factor in the advisory committee’s decision to not provide accelerated approval of OCA. This informs the criteria upon which future MASH therapies will be evaluated for approval: that an ideal agent for MASH not only will have a significant impact on MASH pathophysiology but will also provide benefit for (or at least not worsen) the metabolic comorbidities associated with MASH.
 

New treatments on the horizon

Fortunately, several new MASH therapeutics in development seem to have promising metabolic profiles.

In the phase 3 MAESTRO-NASH biopsy trial, treatment with resmetirom, a thyroid hormone receptor–beta selective agonist versus placebo resulted in a clinically significant improvement in liver fibrosis by one stage or more (26% vs. 14%) and NASH resolution (30% vs. 10%). Unlike OCA, resmetirom was associated with a clinically significant reduction in LDL cholesterol (–16% compared with placebo).

Resmetirom has received a breakthrough therapy designation, and Madrigal Pharmaceuticals recently completed submission of a new drug application for accelerated approval of this agent to the FDA.

Semaglutide is a glucagonlike peptide–1 receptor agonist that is already FDA approved for treatment of diabetes and obesity and is being studied in a phase 3 analysis for NASH.

In a phase 2 trial, daily dosing of semaglutide vs placebo led to clinically significant improvement in NASH resolution (59% vs. 17%) but not significant improvement in fibrosis (43% vs 33%). Although it is impossible to make a meaningful comparison in outcomes between clinical trials, it should be noted that the absolute rate of response in this trial was higher than is routinely seen in other NASH trials and that the placebo rate for improvement in fibrosis was higher than expected, a common problem with NASH trials.

As expected, treatment with semaglutide was associated with dose-dependent reduction in body weight and hemoglobin A1c, highlighting the global metabolic benefit of this agent.

Another major class of agents being studied for NASH treatment are the fibroblast growth factor (FGF) 21 analogs.

In the recently published phase 2b ENLIVEN trial, treatment with the FGF21 analog pegozafermin versus placebo led to a statistically significant improvement in liver fibrosis by one stage (27% vs. 7%) and a numerical improvement in NASH resolution (26% vs. 2%).

In addition to its effects directly in the liver, FGF21 has been associated with promoting positive global metabolic effects, including improved peripheral insulin sensitivity and amelioration of dyslipidemia. Consistent with this, treatment with pegozafermin resulted in a decrease in serum triglycerides and an increase in serum HDL cholesterol and adiponectin levels.

Ultimately, the best therapeutic strategy for MASH may be one that uses combination therapy to maximize liver-directed effects and also ameliorate concomitant or contributing metabolic dysfunction. This approach is already being investigated. What seems clear, however, is that MASH therapy without concurrent treatment of metabolic comorbidities is probably destined to be ineffective, if not counterproductive.

So, what’s in a name? When it comes to MASLD, it seems quite a bit.
 

Dr. Janardhan is an assistant professor, department of internal medicine, at Rush University Medical Center, Chicago. Dr. Reau is a professor, department of internal medicine, Rush University. Dr. Janardhan reported conflicts of interest with Target RWE, Novo Nordisk, Intercept, and Enanta. Dr. Reau reported conflicts of interest with AbbVie, Gilead, and AASLD.

A version of this article appeared on Medscape.com.

In June 2023, a multisociety group agreed to change the name of the disease formerly known as nonalcoholic fatty liver disease (NAFLD) to metabolic dysfunction–associated steatotic liver disease (MASLD).

In the week leading up to this, Intercept Pharmaceuticals announced that their program to develop obeticholic acid (OCA) as a first-to-market, long-awaited potential drug therapy for metabolic dysfunction–associated steatohepatitis (MASH), the more pathogenic form of this disease, was being abandoned after the Food and Drug Administration Gastrointestinal Drugs Advisory Committee voted against accelerated approval of the drug. This decision was made in large part owing to data showing modest efficacy coupled with multiple drug-related side effects, including worsening metabolic dysfunction.

The juxtaposition of these two events highlights what could be a fundamental change in perspective when it comes to the management of patients with MASLD and the development of therapies for this disease.
 

A more precise nomenclature for a broad-spectrum disease

The updating of the NAFLD nomenclature to MASLD represents an important change in the way that the medical community is being asked to view and approach the disease of “fatty liver.”

The previous nomenclature clarified that pathogenic liver steatosis can exist in the absence of alcohol. However, it failed to define the primary drivers of the most common form of NAFLD: that is, metabolic syndrome diseases. The new nomenclature not only better defines the broad spectrum of steatotic liver diseases (alcohol, metabolic, drug/genetic, cryptogenic) but also refocuses the attention of providers on the fundamental basis of MASLD as a critical member of the metabolic syndrome spectrum of diseases.

This is in line with the recently updated American Association for the Study of Liver Disease Practice Guidelines for NAFLD (now MASLD), which focus on active screening to identify patients at risk for advanced MASLD, in particular those with medically complicated obesity or high-risk metabolic characteristics such as diabetes. This is a change from previous versions of the guidelines that were cautious to recommend broad screening guidelines in part owing to the lack of “available therapies.”

The increasing clinical burden of MASH has led to the recognition that patients do not have the luxury of waiting for “anticipated therapies” that have frequently shown only marginal or insignificant efficacy. As a result, some providers have refocused their efforts on the development of care pathways that can efficiently provide comprehensive lifestyle interventions to treat MASH and related metabolic comorbidities within hepatology or other subspecialty clinics.
 

Learning from OCA’s limitations

The approach to drug development for MASH seems to be shifting in a similar, metabolic syndrome–focused way.

The risk-benefit analysis from the FDA advisory committee evaluating OCA noted that the drug provided only modest (albeit statistically significant) benefits over placebo in achieving one of two primary clinical endpoints (improvement in fibrosis by one stage without worsening of NASH), but was associated with toxicity as well as significant drug-related side effects, including new or worsening dyslipidemia, accelerated progression to prediabetes or diabetes, and worsening glycemic control in patients with diabetes.

Worsening clinical markers of metabolic health were an important factor in the advisory committee’s decision to not provide accelerated approval of OCA. This informs the criteria upon which future MASH therapies will be evaluated for approval: that an ideal agent for MASH not only will have a significant impact on MASH pathophysiology but will also provide benefit for (or at least not worsen) the metabolic comorbidities associated with MASH.
 

New treatments on the horizon

Fortunately, several new MASH therapeutics in development seem to have promising metabolic profiles.

In the phase 3 MAESTRO-NASH biopsy trial, treatment with resmetirom, a thyroid hormone receptor–beta selective agonist versus placebo resulted in a clinically significant improvement in liver fibrosis by one stage or more (26% vs. 14%) and NASH resolution (30% vs. 10%). Unlike OCA, resmetirom was associated with a clinically significant reduction in LDL cholesterol (–16% compared with placebo).

Resmetirom has received a breakthrough therapy designation, and Madrigal Pharmaceuticals recently completed submission of a new drug application for accelerated approval of this agent to the FDA.

Semaglutide is a glucagonlike peptide–1 receptor agonist that is already FDA approved for treatment of diabetes and obesity and is being studied in a phase 3 analysis for NASH.

In a phase 2 trial, daily dosing of semaglutide vs placebo led to clinically significant improvement in NASH resolution (59% vs. 17%) but not significant improvement in fibrosis (43% vs 33%). Although it is impossible to make a meaningful comparison in outcomes between clinical trials, it should be noted that the absolute rate of response in this trial was higher than is routinely seen in other NASH trials and that the placebo rate for improvement in fibrosis was higher than expected, a common problem with NASH trials.

As expected, treatment with semaglutide was associated with dose-dependent reduction in body weight and hemoglobin A1c, highlighting the global metabolic benefit of this agent.

Another major class of agents being studied for NASH treatment are the fibroblast growth factor (FGF) 21 analogs.

In the recently published phase 2b ENLIVEN trial, treatment with the FGF21 analog pegozafermin versus placebo led to a statistically significant improvement in liver fibrosis by one stage (27% vs. 7%) and a numerical improvement in NASH resolution (26% vs. 2%).

In addition to its effects directly in the liver, FGF21 has been associated with promoting positive global metabolic effects, including improved peripheral insulin sensitivity and amelioration of dyslipidemia. Consistent with this, treatment with pegozafermin resulted in a decrease in serum triglycerides and an increase in serum HDL cholesterol and adiponectin levels.

Ultimately, the best therapeutic strategy for MASH may be one that uses combination therapy to maximize liver-directed effects and also ameliorate concomitant or contributing metabolic dysfunction. This approach is already being investigated. What seems clear, however, is that MASH therapy without concurrent treatment of metabolic comorbidities is probably destined to be ineffective, if not counterproductive.

So, what’s in a name? When it comes to MASLD, it seems quite a bit.
 

Dr. Janardhan is an assistant professor, department of internal medicine, at Rush University Medical Center, Chicago. Dr. Reau is a professor, department of internal medicine, Rush University. Dr. Janardhan reported conflicts of interest with Target RWE, Novo Nordisk, Intercept, and Enanta. Dr. Reau reported conflicts of interest with AbbVie, Gilead, and AASLD.

A version of this article appeared on Medscape.com.

In June 2023, a multisociety group agreed to change the name of the disease formerly known as nonalcoholic fatty liver disease (NAFLD) to metabolic dysfunction–associated steatotic liver disease (MASLD).

In the week leading up to this, Intercept Pharmaceuticals announced that their program to develop obeticholic acid (OCA) as a first-to-market, long-awaited potential drug therapy for metabolic dysfunction–associated steatohepatitis (MASH), the more pathogenic form of this disease, was being abandoned after the Food and Drug Administration Gastrointestinal Drugs Advisory Committee voted against accelerated approval of the drug. This decision was made in large part owing to data showing modest efficacy coupled with multiple drug-related side effects, including worsening metabolic dysfunction.

The juxtaposition of these two events highlights what could be a fundamental change in perspective when it comes to the management of patients with MASLD and the development of therapies for this disease.
 

A more precise nomenclature for a broad-spectrum disease

The updating of the NAFLD nomenclature to MASLD represents an important change in the way that the medical community is being asked to view and approach the disease of “fatty liver.”

The previous nomenclature clarified that pathogenic liver steatosis can exist in the absence of alcohol. However, it failed to define the primary drivers of the most common form of NAFLD: that is, metabolic syndrome diseases. The new nomenclature not only better defines the broad spectrum of steatotic liver diseases (alcohol, metabolic, drug/genetic, cryptogenic) but also refocuses the attention of providers on the fundamental basis of MASLD as a critical member of the metabolic syndrome spectrum of diseases.

This is in line with the recently updated American Association for the Study of Liver Disease Practice Guidelines for NAFLD (now MASLD), which focus on active screening to identify patients at risk for advanced MASLD, in particular those with medically complicated obesity or high-risk metabolic characteristics such as diabetes. This is a change from previous versions of the guidelines that were cautious to recommend broad screening guidelines in part owing to the lack of “available therapies.”

The increasing clinical burden of MASH has led to the recognition that patients do not have the luxury of waiting for “anticipated therapies” that have frequently shown only marginal or insignificant efficacy. As a result, some providers have refocused their efforts on the development of care pathways that can efficiently provide comprehensive lifestyle interventions to treat MASH and related metabolic comorbidities within hepatology or other subspecialty clinics.
 

Learning from OCA’s limitations

The approach to drug development for MASH seems to be shifting in a similar, metabolic syndrome–focused way.

The risk-benefit analysis from the FDA advisory committee evaluating OCA noted that the drug provided only modest (albeit statistically significant) benefits over placebo in achieving one of two primary clinical endpoints (improvement in fibrosis by one stage without worsening of NASH), but was associated with toxicity as well as significant drug-related side effects, including new or worsening dyslipidemia, accelerated progression to prediabetes or diabetes, and worsening glycemic control in patients with diabetes.

Worsening clinical markers of metabolic health were an important factor in the advisory committee’s decision to not provide accelerated approval of OCA. This informs the criteria upon which future MASH therapies will be evaluated for approval: that an ideal agent for MASH not only will have a significant impact on MASH pathophysiology but will also provide benefit for (or at least not worsen) the metabolic comorbidities associated with MASH.
 

New treatments on the horizon

Fortunately, several new MASH therapeutics in development seem to have promising metabolic profiles.

In the phase 3 MAESTRO-NASH biopsy trial, treatment with resmetirom, a thyroid hormone receptor–beta selective agonist versus placebo resulted in a clinically significant improvement in liver fibrosis by one stage or more (26% vs. 14%) and NASH resolution (30% vs. 10%). Unlike OCA, resmetirom was associated with a clinically significant reduction in LDL cholesterol (–16% compared with placebo).

Resmetirom has received a breakthrough therapy designation, and Madrigal Pharmaceuticals recently completed submission of a new drug application for accelerated approval of this agent to the FDA.

Semaglutide is a glucagonlike peptide–1 receptor agonist that is already FDA approved for treatment of diabetes and obesity and is being studied in a phase 3 analysis for NASH.

In a phase 2 trial, daily dosing of semaglutide vs placebo led to clinically significant improvement in NASH resolution (59% vs. 17%) but not significant improvement in fibrosis (43% vs 33%). Although it is impossible to make a meaningful comparison in outcomes between clinical trials, it should be noted that the absolute rate of response in this trial was higher than is routinely seen in other NASH trials and that the placebo rate for improvement in fibrosis was higher than expected, a common problem with NASH trials.

As expected, treatment with semaglutide was associated with dose-dependent reduction in body weight and hemoglobin A1c, highlighting the global metabolic benefit of this agent.

Another major class of agents being studied for NASH treatment are the fibroblast growth factor (FGF) 21 analogs.

In the recently published phase 2b ENLIVEN trial, treatment with the FGF21 analog pegozafermin versus placebo led to a statistically significant improvement in liver fibrosis by one stage (27% vs. 7%) and a numerical improvement in NASH resolution (26% vs. 2%).

In addition to its effects directly in the liver, FGF21 has been associated with promoting positive global metabolic effects, including improved peripheral insulin sensitivity and amelioration of dyslipidemia. Consistent with this, treatment with pegozafermin resulted in a decrease in serum triglycerides and an increase in serum HDL cholesterol and adiponectin levels.

Ultimately, the best therapeutic strategy for MASH may be one that uses combination therapy to maximize liver-directed effects and also ameliorate concomitant or contributing metabolic dysfunction. This approach is already being investigated. What seems clear, however, is that MASH therapy without concurrent treatment of metabolic comorbidities is probably destined to be ineffective, if not counterproductive.

So, what’s in a name? When it comes to MASLD, it seems quite a bit.
 

Dr. Janardhan is an assistant professor, department of internal medicine, at Rush University Medical Center, Chicago. Dr. Reau is a professor, department of internal medicine, Rush University. Dr. Janardhan reported conflicts of interest with Target RWE, Novo Nordisk, Intercept, and Enanta. Dr. Reau reported conflicts of interest with AbbVie, Gilead, and AASLD.

A version of this article appeared on Medscape.com.

Fear and a lack of health-related knowledge pose significant barriers to preventative cancer care access and effectiveness, recent survey data from The Harris Poll suggests.

The survey, commissioned by Bayer U.S. to identify patient behaviors and care barriers, indicates that more than one in four adults in the United States (27%) would rather not know if they have cancer, and nearly a third (31%) – particularly younger patients aged 18-44 years – avoid going to the doctor because they are afraid of what they might learn.

Similarly, 26% of 2,079 respondents said that fear and anxiety are the main reasons why they don’t make or keep doctor appointments. Those with lower household income and education levels, those with children under age 18 years, and Hispanic adults were most likely to cite this reason.

Almost half (up to 49%) lacked knowledge about certain cancers and risk factors.

For example, 48% of respondents were unaware that breast density affects breast cancer risk and diagnosis, and 38% said they were not very knowledgeable about breast cancer.

Regarding prostate cancer, 49% were unaware that race impacts risk and 49% said they were not knowledgeable about the disease.

The survey highlighted a lack of trust in treatments and health care processes among most adults, especially those with lower income and education levels. Overall, 53% said they have little or no trust in treatments developed by pharmaceutical companies, and 31% said they have little or no trust in medical tests, test results, and other medical processes.

The findings of the survey, which was conducted online June 6-8, 2023, among U.S. adults aged 18 years and older, underscore the need to better educate individuals about cancer risk factors and the benefits of preventative care.

“The increase of fear and anxiety, heightened by a lack of education and in some cases trust barriers, creates an environment where people may not access basic preventative care to ensure early diagnosis,” Sebastian Guth, president of Bayer U.S. and Pharmaceuticals North America, stated in a press release. “This is compounded by the fact that around 27.4 million people of all ages (8.3%) don’t have access to health insurance.

“Companies like Bayer have a responsibility to provide resources that increase health education on the importance of understanding disease risks, early disease screenings, and preventative health care,” Mr. Guth added, noting that the company is partnering with multiple patient advocacy groups to increase trust, awareness, and knowledge “to help individuals understand the resources available to them and their risks for a specific disease.”

Public health initiatives have had mixed results with respect to changing patient behaviors over time, but Breast Cancer Awareness Month (BCAM) in October of each year is a stand-out initiative that could serve as a model for other patient education initiatives, according to a 2022 study.

The Google trends analysis showed that from 2012 to 2021, BCAM was associated with improved public awareness of breast cancer, whereas Lung Cancer Awareness Month and Prostate Cancer Awareness Month had no impact on lung and prostate cancer awareness, respectively, over time, reported Yoshita Nishimura, MD, of Okayama University Graduate School of Medicine, Dentistry, and Pharmaceutical Sciences in Japan, and Jared D. Acoba, MD, of the University of Hawaii, Honolulu.

Dr. Nishimura and Dr. Acoba concluded that the success of BCAM, which was launched in 1985 and is now led by the National Breast Cancer Foundation, is likely a result of “the effective involvement of non-medical industries, influencers affected by breast cancer, and an awareness symbol.”

As for the role of physicians in raising awareness and increasing knowledge at the patient level, various guidelines focus on assessing patient needs and readiness to learn, communicating clearly, and identifying barriers, such as a lack of support and low health literacy.

An American Society of Clinical Oncology consensus guideline for physician-patient communication, for example, provides guidance on core communication skills that apply across the continuum of care, as well as specific topics to address, such as patient goals, treatment options, and support systems – all with an eye toward using “effective communication to optimize the patient-clinician relationship, patient and clinician well-being and family well-being.”

Fear and a lack of health-related knowledge pose significant barriers to preventative cancer care access and effectiveness, recent survey data from The Harris Poll suggests.

The survey, commissioned by Bayer U.S. to identify patient behaviors and care barriers, indicates that more than one in four adults in the United States (27%) would rather not know if they have cancer, and nearly a third (31%) – particularly younger patients aged 18-44 years – avoid going to the doctor because they are afraid of what they might learn.

Similarly, 26% of 2,079 respondents said that fear and anxiety are the main reasons why they don’t make or keep doctor appointments. Those with lower household income and education levels, those with children under age 18 years, and Hispanic adults were most likely to cite this reason.

Almost half (up to 49%) lacked knowledge about certain cancers and risk factors.

For example, 48% of respondents were unaware that breast density affects breast cancer risk and diagnosis, and 38% said they were not very knowledgeable about breast cancer.

Regarding prostate cancer, 49% were unaware that race impacts risk and 49% said they were not knowledgeable about the disease.

The survey highlighted a lack of trust in treatments and health care processes among most adults, especially those with lower income and education levels. Overall, 53% said they have little or no trust in treatments developed by pharmaceutical companies, and 31% said they have little or no trust in medical tests, test results, and other medical processes.

The findings of the survey, which was conducted online June 6-8, 2023, among U.S. adults aged 18 years and older, underscore the need to better educate individuals about cancer risk factors and the benefits of preventative care.

“The increase of fear and anxiety, heightened by a lack of education and in some cases trust barriers, creates an environment where people may not access basic preventative care to ensure early diagnosis,” Sebastian Guth, president of Bayer U.S. and Pharmaceuticals North America, stated in a press release. “This is compounded by the fact that around 27.4 million people of all ages (8.3%) don’t have access to health insurance.

“Companies like Bayer have a responsibility to provide resources that increase health education on the importance of understanding disease risks, early disease screenings, and preventative health care,” Mr. Guth added, noting that the company is partnering with multiple patient advocacy groups to increase trust, awareness, and knowledge “to help individuals understand the resources available to them and their risks for a specific disease.”

Public health initiatives have had mixed results with respect to changing patient behaviors over time, but Breast Cancer Awareness Month (BCAM) in October of each year is a stand-out initiative that could serve as a model for other patient education initiatives, according to a 2022 study.

The Google trends analysis showed that from 2012 to 2021, BCAM was associated with improved public awareness of breast cancer, whereas Lung Cancer Awareness Month and Prostate Cancer Awareness Month had no impact on lung and prostate cancer awareness, respectively, over time, reported Yoshita Nishimura, MD, of Okayama University Graduate School of Medicine, Dentistry, and Pharmaceutical Sciences in Japan, and Jared D. Acoba, MD, of the University of Hawaii, Honolulu.

Dr. Nishimura and Dr. Acoba concluded that the success of BCAM, which was launched in 1985 and is now led by the National Breast Cancer Foundation, is likely a result of “the effective involvement of non-medical industries, influencers affected by breast cancer, and an awareness symbol.”

As for the role of physicians in raising awareness and increasing knowledge at the patient level, various guidelines focus on assessing patient needs and readiness to learn, communicating clearly, and identifying barriers, such as a lack of support and low health literacy.

An American Society of Clinical Oncology consensus guideline for physician-patient communication, for example, provides guidance on core communication skills that apply across the continuum of care, as well as specific topics to address, such as patient goals, treatment options, and support systems – all with an eye toward using “effective communication to optimize the patient-clinician relationship, patient and clinician well-being and family well-being.”

Fear and a lack of health-related knowledge pose significant barriers to preventative cancer care access and effectiveness, recent survey data from The Harris Poll suggests.

The survey, commissioned by Bayer U.S. to identify patient behaviors and care barriers, indicates that more than one in four adults in the United States (27%) would rather not know if they have cancer, and nearly a third (31%) – particularly younger patients aged 18-44 years – avoid going to the doctor because they are afraid of what they might learn.

Similarly, 26% of 2,079 respondents said that fear and anxiety are the main reasons why they don’t make or keep doctor appointments. Those with lower household income and education levels, those with children under age 18 years, and Hispanic adults were most likely to cite this reason.

Almost half (up to 49%) lacked knowledge about certain cancers and risk factors.

For example, 48% of respondents were unaware that breast density affects breast cancer risk and diagnosis, and 38% said they were not very knowledgeable about breast cancer.

Regarding prostate cancer, 49% were unaware that race impacts risk and 49% said they were not knowledgeable about the disease.

The survey highlighted a lack of trust in treatments and health care processes among most adults, especially those with lower income and education levels. Overall, 53% said they have little or no trust in treatments developed by pharmaceutical companies, and 31% said they have little or no trust in medical tests, test results, and other medical processes.

The findings of the survey, which was conducted online June 6-8, 2023, among U.S. adults aged 18 years and older, underscore the need to better educate individuals about cancer risk factors and the benefits of preventative care.

“The increase of fear and anxiety, heightened by a lack of education and in some cases trust barriers, creates an environment where people may not access basic preventative care to ensure early diagnosis,” Sebastian Guth, president of Bayer U.S. and Pharmaceuticals North America, stated in a press release. “This is compounded by the fact that around 27.4 million people of all ages (8.3%) don’t have access to health insurance.

“Companies like Bayer have a responsibility to provide resources that increase health education on the importance of understanding disease risks, early disease screenings, and preventative health care,” Mr. Guth added, noting that the company is partnering with multiple patient advocacy groups to increase trust, awareness, and knowledge “to help individuals understand the resources available to them and their risks for a specific disease.”

Public health initiatives have had mixed results with respect to changing patient behaviors over time, but Breast Cancer Awareness Month (BCAM) in October of each year is a stand-out initiative that could serve as a model for other patient education initiatives, according to a 2022 study.

The Google trends analysis showed that from 2012 to 2021, BCAM was associated with improved public awareness of breast cancer, whereas Lung Cancer Awareness Month and Prostate Cancer Awareness Month had no impact on lung and prostate cancer awareness, respectively, over time, reported Yoshita Nishimura, MD, of Okayama University Graduate School of Medicine, Dentistry, and Pharmaceutical Sciences in Japan, and Jared D. Acoba, MD, of the University of Hawaii, Honolulu.

Dr. Nishimura and Dr. Acoba concluded that the success of BCAM, which was launched in 1985 and is now led by the National Breast Cancer Foundation, is likely a result of “the effective involvement of non-medical industries, influencers affected by breast cancer, and an awareness symbol.”

As for the role of physicians in raising awareness and increasing knowledge at the patient level, various guidelines focus on assessing patient needs and readiness to learn, communicating clearly, and identifying barriers, such as a lack of support and low health literacy.

An American Society of Clinical Oncology consensus guideline for physician-patient communication, for example, provides guidance on core communication skills that apply across the continuum of care, as well as specific topics to address, such as patient goals, treatment options, and support systems – all with an eye toward using “effective communication to optimize the patient-clinician relationship, patient and clinician well-being and family well-being.”

In the United States this year, approximately 288,300 men will be newly diagnosed with prostate cancer and about 34,700 men will die from this disease. It is the second leading cause of cancer in men, and one out of every eight men will be diagnosed with this cancer at some point in their lives.

As primary care physicians, a large part of our role is to prevent or detect cancers early. Patients look to us for this guidance. However, prostate cancer screening has long been a controversial issue. Earlier this year, the American Urological Association along with the Society of Urologic Oncology published updated guidelines.

Clear recommendations that come from this set of guidelines that are relevant to primary care physicians include:

• using PSA as the screening test of choice.
• repeating PSA in patients with newly elevated results before moving on to other test.
• offering PSA screening every 2-4 years in patients aged 50-69 years.
• offering baseline screening in those between 45-50 years of age.

In high-risk patients, screening can be initiated at 40-45 years of age. All of these recommendations come with the caveat that we give the patient all the pros and cons and leave it up to their “values and preferences.”

The guidelines make recommendations regarding PSA screening and biopsy standards. These guidelines are very specific in their recommendations; however, the question about whether to do PSA screening in the first place is left open to debate. While shared decision-making is important with any testing, it is more difficult with prostate cancer screening. Patients need to understand that there are possible adverse events that can result because of an elevated PSA, such as unneeded biopsies that may come with complications.

The authors of this set of guidelines suggest that physicians talk to patients more often about the benefits of the screening than they do about the negative consequences. This assumes that a negative biopsy result is an unnecessary test, which is not a fair assessment. Negative test results can provide useful clinical information. While a PSA result may lead to a biopsy that could have possibly been avoided, we don’t have any better screening tests available. Missing a prostate cancer that could have been detected by PSA screening is also very harmful. Deciding whether to do PSA screening for any given patient then becomes a difficult question.

More research into biomarkers to detect prostate cancer is needed, as suggested by the guideline authors. As primary care doctors, we’re the first ones to order these tests and make decisions regarding the results. While we may not be the ones to do the biopsies, we do need to know when to refer the patients to specialists or when we can just repeat the test.

Population health is often the benchmark used when looking at screening guidelines. But in the primary care setting, we are responsible for individual patients. Applying guidelines that take whole populations into consideration often doesn’t translate well to single patients. We do need to make them responsible for their own health care decisions but, at the same time, we need to offer them some guidance. If the guidelines are clear, this is easy. When they suggest giving patients all the pros and cons and letting them make their own decision, this is hard. Some of them want us to tell them what to do.

Additionally, patients in the primary care setting develop close relationships with their physicians. They are not an elevated PSA test or a negative biopsy result. They have concerns and fears. When they are high risk, the advice is easy. Keeping in mind that prostate cancer is the second leading cause of cancer in men in the United States, we should have clear screening guidelines, such as we do with mammograms in women. Yes, shared decision-making is important, but we also need to know the answer when our patients ask us whether or not they should have a PSA test done.
 

Dr. Girgis practices family medicine in South River, N.J., and is a clinical assistant professor of family medicine at Robert Wood Johnson Medical School, New Brunswick, N.J.

In the United States this year, approximately 288,300 men will be newly diagnosed with prostate cancer and about 34,700 men will die from this disease. It is the second leading cause of cancer in men, and one out of every eight men will be diagnosed with this cancer at some point in their lives.

As primary care physicians, a large part of our role is to prevent or detect cancers early. Patients look to us for this guidance. However, prostate cancer screening has long been a controversial issue. Earlier this year, the American Urological Association along with the Society of Urologic Oncology published updated guidelines.

Clear recommendations that come from this set of guidelines that are relevant to primary care physicians include:

• using PSA as the screening test of choice.
• repeating PSA in patients with newly elevated results before moving on to other test.
• offering PSA screening every 2-4 years in patients aged 50-69 years.
• offering baseline screening in those between 45-50 years of age.

In high-risk patients, screening can be initiated at 40-45 years of age. All of these recommendations come with the caveat that we give the patient all the pros and cons and leave it up to their “values and preferences.”

The guidelines make recommendations regarding PSA screening and biopsy standards. These guidelines are very specific in their recommendations; however, the question about whether to do PSA screening in the first place is left open to debate. While shared decision-making is important with any testing, it is more difficult with prostate cancer screening. Patients need to understand that there are possible adverse events that can result because of an elevated PSA, such as unneeded biopsies that may come with complications.

The authors of this set of guidelines suggest that physicians talk to patients more often about the benefits of the screening than they do about the negative consequences. This assumes that a negative biopsy result is an unnecessary test, which is not a fair assessment. Negative test results can provide useful clinical information. While a PSA result may lead to a biopsy that could have possibly been avoided, we don’t have any better screening tests available. Missing a prostate cancer that could have been detected by PSA screening is also very harmful. Deciding whether to do PSA screening for any given patient then becomes a difficult question.

More research into biomarkers to detect prostate cancer is needed, as suggested by the guideline authors. As primary care doctors, we’re the first ones to order these tests and make decisions regarding the results. While we may not be the ones to do the biopsies, we do need to know when to refer the patients to specialists or when we can just repeat the test.

Population health is often the benchmark used when looking at screening guidelines. But in the primary care setting, we are responsible for individual patients. Applying guidelines that take whole populations into consideration often doesn’t translate well to single patients. We do need to make them responsible for their own health care decisions but, at the same time, we need to offer them some guidance. If the guidelines are clear, this is easy. When they suggest giving patients all the pros and cons and letting them make their own decision, this is hard. Some of them want us to tell them what to do.

Additionally, patients in the primary care setting develop close relationships with their physicians. They are not an elevated PSA test or a negative biopsy result. They have concerns and fears. When they are high risk, the advice is easy. Keeping in mind that prostate cancer is the second leading cause of cancer in men in the United States, we should have clear screening guidelines, such as we do with mammograms in women. Yes, shared decision-making is important, but we also need to know the answer when our patients ask us whether or not they should have a PSA test done.
 

Dr. Girgis practices family medicine in South River, N.J., and is a clinical assistant professor of family medicine at Robert Wood Johnson Medical School, New Brunswick, N.J.

In the United States this year, approximately 288,300 men will be newly diagnosed with prostate cancer and about 34,700 men will die from this disease. It is the second leading cause of cancer in men, and one out of every eight men will be diagnosed with this cancer at some point in their lives.

As primary care physicians, a large part of our role is to prevent or detect cancers early. Patients look to us for this guidance. However, prostate cancer screening has long been a controversial issue. Earlier this year, the American Urological Association along with the Society of Urologic Oncology published updated guidelines.

Clear recommendations that come from this set of guidelines that are relevant to primary care physicians include:

• using PSA as the screening test of choice.
• repeating PSA in patients with newly elevated results before moving on to other test.
• offering PSA screening every 2-4 years in patients aged 50-69 years.
• offering baseline screening in those between 45-50 years of age.

In high-risk patients, screening can be initiated at 40-45 years of age. All of these recommendations come with the caveat that we give the patient all the pros and cons and leave it up to their “values and preferences.”

The guidelines make recommendations regarding PSA screening and biopsy standards. These guidelines are very specific in their recommendations; however, the question about whether to do PSA screening in the first place is left open to debate. While shared decision-making is important with any testing, it is more difficult with prostate cancer screening. Patients need to understand that there are possible adverse events that can result because of an elevated PSA, such as unneeded biopsies that may come with complications.

The authors of this set of guidelines suggest that physicians talk to patients more often about the benefits of the screening than they do about the negative consequences. This assumes that a negative biopsy result is an unnecessary test, which is not a fair assessment. Negative test results can provide useful clinical information. While a PSA result may lead to a biopsy that could have possibly been avoided, we don’t have any better screening tests available. Missing a prostate cancer that could have been detected by PSA screening is also very harmful. Deciding whether to do PSA screening for any given patient then becomes a difficult question.

More research into biomarkers to detect prostate cancer is needed, as suggested by the guideline authors. As primary care doctors, we’re the first ones to order these tests and make decisions regarding the results. While we may not be the ones to do the biopsies, we do need to know when to refer the patients to specialists or when we can just repeat the test.

Population health is often the benchmark used when looking at screening guidelines. But in the primary care setting, we are responsible for individual patients. Applying guidelines that take whole populations into consideration often doesn’t translate well to single patients. We do need to make them responsible for their own health care decisions but, at the same time, we need to offer them some guidance. If the guidelines are clear, this is easy. When they suggest giving patients all the pros and cons and letting them make their own decision, this is hard. Some of them want us to tell them what to do.

Additionally, patients in the primary care setting develop close relationships with their physicians. They are not an elevated PSA test or a negative biopsy result. They have concerns and fears. When they are high risk, the advice is easy. Keeping in mind that prostate cancer is the second leading cause of cancer in men in the United States, we should have clear screening guidelines, such as we do with mammograms in women. Yes, shared decision-making is important, but we also need to know the answer when our patients ask us whether or not they should have a PSA test done.
 

Dr. Girgis practices family medicine in South River, N.J., and is a clinical assistant professor of family medicine at Robert Wood Johnson Medical School, New Brunswick, N.J.