SAN DIEGO — A new cell-free DNA (cfDNA)-based blood test shows promising performance in detecting colorectal cancer and advanced precancerous lesions, say the authors of new research.

Rachel B. Issaka, MD, MAS, of the Fred Hutchinson Cancer Center, Seattle, presented the clinical data, which was published in The New England Journal of Medicine, at the American Association for Cancer Research annual meeting.

Fred Hutchinson Cancer Center

The authors of the study evaluated the performance of a cfDNA blood-based test in a population eligible for colorectal cancer screening. The researchers found that the test had high sensitivity for the detection of colorectal cancer and high specificity for advanced precancerous lesions.

This novel blood test could improve screening adherence and, ultimately, reduce colorectal cancer-related mortality, Dr. Issaka said during her presentation.

“This test has the potential to help us reach the 80% screening target in colorectal cancer. However, this will depend on many factors, including access, implementation, follow-up colonoscopy, and characteristics of the test,” Dr. Issaka said in an interview.

She added that, when approved for broader use, anyone who wants to use this blood test for colorectal cancer screening should have a frank conversation with their healthcare provider.

“Considering the person’s age, medical history, family history, and any potential symptoms, and how the test performs will dictate if it’s the right test for that person versus another screening strategy,” Dr. Issaka explained.
 

The Blood Test Detects Colorectal Cancer With High Accuracy

The investigators of the observational ECLIPSE trial evaluated the performance of the cfDNA-based blood test in 7861 individuals who were eligible for colorectal cancer screening. The study population included people from more than 200 rural and urban sites across 34 states, including community hospitals, private practices, gastroenterology clinics, and academic centers. “The study enrolled a diverse cohort that is reflective of the demographics of the intended use population in the US,” Dr. Issaka said during her talk.

The co-primary outcomes of the study were the test’s sensitivity for detecting colorectal cancer and its specificity for identifying advanced neoplasia.

In her presentation, Dr. Issaka highlighted that the test had 83.1% (95% confidence interval [CI], 72.2%-90.3%) sensitivity for the detection of colorectal cancer, meaning that it was able to correctly identify most participants with the disease. The test’s sensitivity was even higher (87.5%; 95% CI, 75.3%-94.1%) for stage I, II, or III colorectal cancer. “These are the stages at which early intervention can have the greatest impact on patient prognosis,” Dr. Issaka said.

Moreover, the blood test showed 89.6% (95% CI, 88.8%-90.3%) specificity for advanced neoplasia, including colorectal cancer and advanced precancerous lesions. The specificity of the test for negative colonoscopy results (no colorectal cancer, advanced precancerous lesions, or nonadvanced precancerous lesions) was 89.9% (95% CI, 89.0%-90.7%).

Dr. Issaka highlighted that this cfDNA assay is the first blood-based test with performance comparable to current guideline-recommended noninvasive options for CRC.
 

The Blood Test Shows Limited Ability To Detect Advanced Precancerous Lesions

During her presentation, Dr. Issaka acknowledged that the cfDNA-based blood test had a lower sensitivity (13.2%; 95% CI, 11.3%-15.3%) for the detection of advanced precancerous lesions, suggesting that it may be more effective at identifying established cancers than early-stage precancerous changes. Low sensitivity was also observed for high-grade dysplasia (22.6%; 95% CI, 11.4%-39.8%). However, she emphasized that the test could still play a valuable role in a comprehensive screening approach, potentially serving as a first-line tool to identify individuals who would then undergo follow-up colonoscopy.

“Although blood-based tests perform well at finding cancers, they do not do so well at finding precancerous polyps. This is relevant because colorectal cancer is one of the few cancers that we can prevent by finding and removing precancerous polyps,” Folasade P. May, MD, PhD, MPhil, said in an interview.

“Users must also understand that if the test result is abnormal, a colonoscopy is required to look for cancers and polyps that might have caused the abnormal result,” added Dr. May, associate professor at UCLA. She was not involved in the study.
 

Clinical Implications and Future Steps

According to the study published in the NEJM, colorectal cancer is the third most commonly diagnosed cancer in the United States, and early detection is crucial for effective treatment. However, over a third of eligible individuals are not up to date with recommended screening.

During her talk, Dr. Issaka noted that colonoscopy is the most commonly used screening method for colorectal cancer. What contributes to the low adherence to getting a colonoscopy among the eligible population is that some find it inconvenient, and the test is invasive, she added.

According to Dr. May, the key advantage of cfDNA-based screening is that many people will find it easier to complete a blood test than the currently available screening tests.

“This option may allow us to screen individuals that we have previously struggled to convince to get screened for colorectal cancer,” she said.

In an interview, Dr. Issaka acknowledged that the potential public health impact of any noninvasive screening test depends on how many people with abnormal results complete a follow-up colonoscopy. “This is an important quality metric to track,” she said.

In an interview, Dr. Issaka emphasized that comparing this cfDNA blood test with emerging blood tests and other noninvasive screening strategies will empower patients and clinicians to select the right test at the right time for the right patient.

She added that the study was conducted in an average-risk screening population and that further research is needed to evaluate the test’s performance in higher-risk groups and to assess its real-world impact on screening adherence and colorectal cancer-related outcomes.

Commenting on potential challenges with implementing this cfDNA blood test in clinical practice, Dr. May said, “As we consider incorporating blood-based tests into clinical practice, some challenges include cost, equitable access to tests and follow-up, performance in young adults who are newly eligible for screening, and follow-up after abnormal results.”

She added that, if there is uptake of these tests, it will be important to track how that impacts colorectal cancer screening rates, stage at diagnosis, and whether there is stage migration, incidence, and mortality.

“At this time, I feel that these tests are appropriate for individuals who will not or cannot participate in one of the currently recommended screening tests. These include colonoscopy and stool-based tests, like FIT and FIT-DNA,” Dr. May concluded.

Dr. Issaka reported financial relationships with the National Institutes of Health/National Cancer Institute, American College of Gastroenterology, and Guardant Health Inc. Dr. May reported financial relationships with Takeda, Medtronic, Johnson & Johnson, Saint Supply, Exact Sciences, Freenome, Geneoscopy, Guardant Health, InterVenn, Natura, National Institutes of Health/National Cancer Institute, Veterans Affairs HSR&D, Broad Institute, Stand up to Cancer, and NRG Oncology.

SAN DIEGO — A new cell-free DNA (cfDNA)-based blood test shows promising performance in detecting colorectal cancer and advanced precancerous lesions, say the authors of new research.

Rachel B. Issaka, MD, MAS, of the Fred Hutchinson Cancer Center, Seattle, presented the clinical data, which was published in The New England Journal of Medicine, at the American Association for Cancer Research annual meeting.

Fred Hutchinson Cancer Center

The authors of the study evaluated the performance of a cfDNA blood-based test in a population eligible for colorectal cancer screening. The researchers found that the test had high sensitivity for the detection of colorectal cancer and high specificity for advanced precancerous lesions.

This novel blood test could improve screening adherence and, ultimately, reduce colorectal cancer-related mortality, Dr. Issaka said during her presentation.

“This test has the potential to help us reach the 80% screening target in colorectal cancer. However, this will depend on many factors, including access, implementation, follow-up colonoscopy, and characteristics of the test,” Dr. Issaka said in an interview.

She added that, when approved for broader use, anyone who wants to use this blood test for colorectal cancer screening should have a frank conversation with their healthcare provider.

“Considering the person’s age, medical history, family history, and any potential symptoms, and how the test performs will dictate if it’s the right test for that person versus another screening strategy,” Dr. Issaka explained.
 

The Blood Test Detects Colorectal Cancer With High Accuracy

The investigators of the observational ECLIPSE trial evaluated the performance of the cfDNA-based blood test in 7861 individuals who were eligible for colorectal cancer screening. The study population included people from more than 200 rural and urban sites across 34 states, including community hospitals, private practices, gastroenterology clinics, and academic centers. “The study enrolled a diverse cohort that is reflective of the demographics of the intended use population in the US,” Dr. Issaka said during her talk.

The co-primary outcomes of the study were the test’s sensitivity for detecting colorectal cancer and its specificity for identifying advanced neoplasia.

In her presentation, Dr. Issaka highlighted that the test had 83.1% (95% confidence interval [CI], 72.2%-90.3%) sensitivity for the detection of colorectal cancer, meaning that it was able to correctly identify most participants with the disease. The test’s sensitivity was even higher (87.5%; 95% CI, 75.3%-94.1%) for stage I, II, or III colorectal cancer. “These are the stages at which early intervention can have the greatest impact on patient prognosis,” Dr. Issaka said.

Moreover, the blood test showed 89.6% (95% CI, 88.8%-90.3%) specificity for advanced neoplasia, including colorectal cancer and advanced precancerous lesions. The specificity of the test for negative colonoscopy results (no colorectal cancer, advanced precancerous lesions, or nonadvanced precancerous lesions) was 89.9% (95% CI, 89.0%-90.7%).

Dr. Issaka highlighted that this cfDNA assay is the first blood-based test with performance comparable to current guideline-recommended noninvasive options for CRC.
 

The Blood Test Shows Limited Ability To Detect Advanced Precancerous Lesions

During her presentation, Dr. Issaka acknowledged that the cfDNA-based blood test had a lower sensitivity (13.2%; 95% CI, 11.3%-15.3%) for the detection of advanced precancerous lesions, suggesting that it may be more effective at identifying established cancers than early-stage precancerous changes. Low sensitivity was also observed for high-grade dysplasia (22.6%; 95% CI, 11.4%-39.8%). However, she emphasized that the test could still play a valuable role in a comprehensive screening approach, potentially serving as a first-line tool to identify individuals who would then undergo follow-up colonoscopy.

“Although blood-based tests perform well at finding cancers, they do not do so well at finding precancerous polyps. This is relevant because colorectal cancer is one of the few cancers that we can prevent by finding and removing precancerous polyps,” Folasade P. May, MD, PhD, MPhil, said in an interview.

“Users must also understand that if the test result is abnormal, a colonoscopy is required to look for cancers and polyps that might have caused the abnormal result,” added Dr. May, associate professor at UCLA. She was not involved in the study.
 

Clinical Implications and Future Steps

According to the study published in the NEJM, colorectal cancer is the third most commonly diagnosed cancer in the United States, and early detection is crucial for effective treatment. However, over a third of eligible individuals are not up to date with recommended screening.

During her talk, Dr. Issaka noted that colonoscopy is the most commonly used screening method for colorectal cancer. What contributes to the low adherence to getting a colonoscopy among the eligible population is that some find it inconvenient, and the test is invasive, she added.

According to Dr. May, the key advantage of cfDNA-based screening is that many people will find it easier to complete a blood test than the currently available screening tests.

“This option may allow us to screen individuals that we have previously struggled to convince to get screened for colorectal cancer,” she said.

In an interview, Dr. Issaka acknowledged that the potential public health impact of any noninvasive screening test depends on how many people with abnormal results complete a follow-up colonoscopy. “This is an important quality metric to track,” she said.

In an interview, Dr. Issaka emphasized that comparing this cfDNA blood test with emerging blood tests and other noninvasive screening strategies will empower patients and clinicians to select the right test at the right time for the right patient.

She added that the study was conducted in an average-risk screening population and that further research is needed to evaluate the test’s performance in higher-risk groups and to assess its real-world impact on screening adherence and colorectal cancer-related outcomes.

Commenting on potential challenges with implementing this cfDNA blood test in clinical practice, Dr. May said, “As we consider incorporating blood-based tests into clinical practice, some challenges include cost, equitable access to tests and follow-up, performance in young adults who are newly eligible for screening, and follow-up after abnormal results.”

She added that, if there is uptake of these tests, it will be important to track how that impacts colorectal cancer screening rates, stage at diagnosis, and whether there is stage migration, incidence, and mortality.

“At this time, I feel that these tests are appropriate for individuals who will not or cannot participate in one of the currently recommended screening tests. These include colonoscopy and stool-based tests, like FIT and FIT-DNA,” Dr. May concluded.

Dr. Issaka reported financial relationships with the National Institutes of Health/National Cancer Institute, American College of Gastroenterology, and Guardant Health Inc. Dr. May reported financial relationships with Takeda, Medtronic, Johnson & Johnson, Saint Supply, Exact Sciences, Freenome, Geneoscopy, Guardant Health, InterVenn, Natura, National Institutes of Health/National Cancer Institute, Veterans Affairs HSR&D, Broad Institute, Stand up to Cancer, and NRG Oncology.

SAN DIEGO — A new cell-free DNA (cfDNA)-based blood test shows promising performance in detecting colorectal cancer and advanced precancerous lesions, say the authors of new research.

Rachel B. Issaka, MD, MAS, of the Fred Hutchinson Cancer Center, Seattle, presented the clinical data, which was published in The New England Journal of Medicine, at the American Association for Cancer Research annual meeting.

Fred Hutchinson Cancer Center

The authors of the study evaluated the performance of a cfDNA blood-based test in a population eligible for colorectal cancer screening. The researchers found that the test had high sensitivity for the detection of colorectal cancer and high specificity for advanced precancerous lesions.

This novel blood test could improve screening adherence and, ultimately, reduce colorectal cancer-related mortality, Dr. Issaka said during her presentation.

“This test has the potential to help us reach the 80% screening target in colorectal cancer. However, this will depend on many factors, including access, implementation, follow-up colonoscopy, and characteristics of the test,” Dr. Issaka said in an interview.

She added that, when approved for broader use, anyone who wants to use this blood test for colorectal cancer screening should have a frank conversation with their healthcare provider.

“Considering the person’s age, medical history, family history, and any potential symptoms, and how the test performs will dictate if it’s the right test for that person versus another screening strategy,” Dr. Issaka explained.
 

The Blood Test Detects Colorectal Cancer With High Accuracy

The investigators of the observational ECLIPSE trial evaluated the performance of the cfDNA-based blood test in 7861 individuals who were eligible for colorectal cancer screening. The study population included people from more than 200 rural and urban sites across 34 states, including community hospitals, private practices, gastroenterology clinics, and academic centers. “The study enrolled a diverse cohort that is reflective of the demographics of the intended use population in the US,” Dr. Issaka said during her talk.

The co-primary outcomes of the study were the test’s sensitivity for detecting colorectal cancer and its specificity for identifying advanced neoplasia.

In her presentation, Dr. Issaka highlighted that the test had 83.1% (95% confidence interval [CI], 72.2%-90.3%) sensitivity for the detection of colorectal cancer, meaning that it was able to correctly identify most participants with the disease. The test’s sensitivity was even higher (87.5%; 95% CI, 75.3%-94.1%) for stage I, II, or III colorectal cancer. “These are the stages at which early intervention can have the greatest impact on patient prognosis,” Dr. Issaka said.

Moreover, the blood test showed 89.6% (95% CI, 88.8%-90.3%) specificity for advanced neoplasia, including colorectal cancer and advanced precancerous lesions. The specificity of the test for negative colonoscopy results (no colorectal cancer, advanced precancerous lesions, or nonadvanced precancerous lesions) was 89.9% (95% CI, 89.0%-90.7%).

Dr. Issaka highlighted that this cfDNA assay is the first blood-based test with performance comparable to current guideline-recommended noninvasive options for CRC.
 

The Blood Test Shows Limited Ability To Detect Advanced Precancerous Lesions

During her presentation, Dr. Issaka acknowledged that the cfDNA-based blood test had a lower sensitivity (13.2%; 95% CI, 11.3%-15.3%) for the detection of advanced precancerous lesions, suggesting that it may be more effective at identifying established cancers than early-stage precancerous changes. Low sensitivity was also observed for high-grade dysplasia (22.6%; 95% CI, 11.4%-39.8%). However, she emphasized that the test could still play a valuable role in a comprehensive screening approach, potentially serving as a first-line tool to identify individuals who would then undergo follow-up colonoscopy.

“Although blood-based tests perform well at finding cancers, they do not do so well at finding precancerous polyps. This is relevant because colorectal cancer is one of the few cancers that we can prevent by finding and removing precancerous polyps,” Folasade P. May, MD, PhD, MPhil, said in an interview.

“Users must also understand that if the test result is abnormal, a colonoscopy is required to look for cancers and polyps that might have caused the abnormal result,” added Dr. May, associate professor at UCLA. She was not involved in the study.
 

Clinical Implications and Future Steps

According to the study published in the NEJM, colorectal cancer is the third most commonly diagnosed cancer in the United States, and early detection is crucial for effective treatment. However, over a third of eligible individuals are not up to date with recommended screening.

During her talk, Dr. Issaka noted that colonoscopy is the most commonly used screening method for colorectal cancer. What contributes to the low adherence to getting a colonoscopy among the eligible population is that some find it inconvenient, and the test is invasive, she added.

According to Dr. May, the key advantage of cfDNA-based screening is that many people will find it easier to complete a blood test than the currently available screening tests.

“This option may allow us to screen individuals that we have previously struggled to convince to get screened for colorectal cancer,” she said.

In an interview, Dr. Issaka acknowledged that the potential public health impact of any noninvasive screening test depends on how many people with abnormal results complete a follow-up colonoscopy. “This is an important quality metric to track,” she said.

In an interview, Dr. Issaka emphasized that comparing this cfDNA blood test with emerging blood tests and other noninvasive screening strategies will empower patients and clinicians to select the right test at the right time for the right patient.

She added that the study was conducted in an average-risk screening population and that further research is needed to evaluate the test’s performance in higher-risk groups and to assess its real-world impact on screening adherence and colorectal cancer-related outcomes.

Commenting on potential challenges with implementing this cfDNA blood test in clinical practice, Dr. May said, “As we consider incorporating blood-based tests into clinical practice, some challenges include cost, equitable access to tests and follow-up, performance in young adults who are newly eligible for screening, and follow-up after abnormal results.”

She added that, if there is uptake of these tests, it will be important to track how that impacts colorectal cancer screening rates, stage at diagnosis, and whether there is stage migration, incidence, and mortality.

“At this time, I feel that these tests are appropriate for individuals who will not or cannot participate in one of the currently recommended screening tests. These include colonoscopy and stool-based tests, like FIT and FIT-DNA,” Dr. May concluded.

Dr. Issaka reported financial relationships with the National Institutes of Health/National Cancer Institute, American College of Gastroenterology, and Guardant Health Inc. Dr. May reported financial relationships with Takeda, Medtronic, Johnson & Johnson, Saint Supply, Exact Sciences, Freenome, Geneoscopy, Guardant Health, InterVenn, Natura, National Institutes of Health/National Cancer Institute, Veterans Affairs HSR&D, Broad Institute, Stand up to Cancer, and NRG Oncology.

BUDAPEST, HUNGARY — Twice daily intranasal oxytocin has been associated with improved social functioning, quality of life, and overall symptoms in adults with autism spectrum disorder (ASD), results of a small randomized control trial showed.

“One of the challenges for adults with autism is experiencing poor social interactions and difficulties in making friends. Insufficient social support from peers, friends, and family members can contribute to loneliness in adolescents with ASD, which in turn leads to anxiety, sadness, and social isolation,” said study investigator Saba Faraji Niri, MD, assistant professor of psychiatry, Tehran University of Medical Sciences in Iran. 

Recent US data show it is relatively common. In addition, previous research suggests intranasal oxytocin significantly increases activity in brain regions that play a role in establishing social interactions.

To evaluate the therapeutic effects and safety of intranasal oxytocin the researchers randomly assigned 39 adult patients with ASD to receive intranasal oxytocin or placebo with 24 units administered every 12 hours for 8 weeks. 

Dr. Faraji Niri said study participants were required to stop all psychotropic medications for at least 8 weeks prior to study entry. 

Participants were assessed at baseline and weeks 4 and 8 using the Autism Quotient, Ritvo Autism Asperger Diagnostic Scale — Revised (RAADS-R), Social Responsiveness Scale (SRS), Clinical Global Impression (CGI) scale, and the World Health Organization Quality of Life-BREF (WHOQL-BREF) questionnaire. Adverse events were also evaluated.

Dr. Faraji Niri said that those receiving intranasal oxytocin showed clinical improvement on RAADS-R scores (P = .010), as well as on the social communication subscale of the SRS (P = .002), the CGI scale (P = .000), and the physical (P = .004), psychological (P = .006), and social relationships (P = .046) domains of the WHOQL-BREF. 

However, although the findings were positive, she said at this point it’s not possible to draw any definitive conclusions. She noted the study had several potential confounders. These included differences in baseline levels of endogenous oxytocin among study participants individuals, as well as difference in required treatment doses, which were adjusted by age and sex. The presence of comorbidities and interactions with other treatments could also affect the results.

Commenting on the findings for this news organization, session chair Szabolcs Kéri, PhD, Professor, Sztárai Institute, University of Tokaj, Sárospatak, Hungary, said the use of oxytocin for ASD is controversial. He said that, while the research contributes to the scientific debate, the clinical significance of the findings is unclear. 

The investigators and Dr Keri reported no relevant financial disclosures.

A version of this article appeared on Medscape.com .

BUDAPEST, HUNGARY — Twice daily intranasal oxytocin has been associated with improved social functioning, quality of life, and overall symptoms in adults with autism spectrum disorder (ASD), results of a small randomized control trial showed.

“One of the challenges for adults with autism is experiencing poor social interactions and difficulties in making friends. Insufficient social support from peers, friends, and family members can contribute to loneliness in adolescents with ASD, which in turn leads to anxiety, sadness, and social isolation,” said study investigator Saba Faraji Niri, MD, assistant professor of psychiatry, Tehran University of Medical Sciences in Iran. 

Recent US data show it is relatively common. In addition, previous research suggests intranasal oxytocin significantly increases activity in brain regions that play a role in establishing social interactions.

To evaluate the therapeutic effects and safety of intranasal oxytocin the researchers randomly assigned 39 adult patients with ASD to receive intranasal oxytocin or placebo with 24 units administered every 12 hours for 8 weeks. 

Dr. Faraji Niri said study participants were required to stop all psychotropic medications for at least 8 weeks prior to study entry. 

Participants were assessed at baseline and weeks 4 and 8 using the Autism Quotient, Ritvo Autism Asperger Diagnostic Scale — Revised (RAADS-R), Social Responsiveness Scale (SRS), Clinical Global Impression (CGI) scale, and the World Health Organization Quality of Life-BREF (WHOQL-BREF) questionnaire. Adverse events were also evaluated.

Dr. Faraji Niri said that those receiving intranasal oxytocin showed clinical improvement on RAADS-R scores (P = .010), as well as on the social communication subscale of the SRS (P = .002), the CGI scale (P = .000), and the physical (P = .004), psychological (P = .006), and social relationships (P = .046) domains of the WHOQL-BREF. 

However, although the findings were positive, she said at this point it’s not possible to draw any definitive conclusions. She noted the study had several potential confounders. These included differences in baseline levels of endogenous oxytocin among study participants individuals, as well as difference in required treatment doses, which were adjusted by age and sex. The presence of comorbidities and interactions with other treatments could also affect the results.

Commenting on the findings for this news organization, session chair Szabolcs Kéri, PhD, Professor, Sztárai Institute, University of Tokaj, Sárospatak, Hungary, said the use of oxytocin for ASD is controversial. He said that, while the research contributes to the scientific debate, the clinical significance of the findings is unclear. 

The investigators and Dr Keri reported no relevant financial disclosures.

A version of this article appeared on Medscape.com .

BUDAPEST, HUNGARY — Twice daily intranasal oxytocin has been associated with improved social functioning, quality of life, and overall symptoms in adults with autism spectrum disorder (ASD), results of a small randomized control trial showed.

“One of the challenges for adults with autism is experiencing poor social interactions and difficulties in making friends. Insufficient social support from peers, friends, and family members can contribute to loneliness in adolescents with ASD, which in turn leads to anxiety, sadness, and social isolation,” said study investigator Saba Faraji Niri, MD, assistant professor of psychiatry, Tehran University of Medical Sciences in Iran. 

Recent US data show it is relatively common. In addition, previous research suggests intranasal oxytocin significantly increases activity in brain regions that play a role in establishing social interactions.

To evaluate the therapeutic effects and safety of intranasal oxytocin the researchers randomly assigned 39 adult patients with ASD to receive intranasal oxytocin or placebo with 24 units administered every 12 hours for 8 weeks. 

Dr. Faraji Niri said study participants were required to stop all psychotropic medications for at least 8 weeks prior to study entry. 

Participants were assessed at baseline and weeks 4 and 8 using the Autism Quotient, Ritvo Autism Asperger Diagnostic Scale — Revised (RAADS-R), Social Responsiveness Scale (SRS), Clinical Global Impression (CGI) scale, and the World Health Organization Quality of Life-BREF (WHOQL-BREF) questionnaire. Adverse events were also evaluated.

Dr. Faraji Niri said that those receiving intranasal oxytocin showed clinical improvement on RAADS-R scores (P = .010), as well as on the social communication subscale of the SRS (P = .002), the CGI scale (P = .000), and the physical (P = .004), psychological (P = .006), and social relationships (P = .046) domains of the WHOQL-BREF. 

However, although the findings were positive, she said at this point it’s not possible to draw any definitive conclusions. She noted the study had several potential confounders. These included differences in baseline levels of endogenous oxytocin among study participants individuals, as well as difference in required treatment doses, which were adjusted by age and sex. The presence of comorbidities and interactions with other treatments could also affect the results.

Commenting on the findings for this news organization, session chair Szabolcs Kéri, PhD, Professor, Sztárai Institute, University of Tokaj, Sárospatak, Hungary, said the use of oxytocin for ASD is controversial. He said that, while the research contributes to the scientific debate, the clinical significance of the findings is unclear. 

The investigators and Dr Keri reported no relevant financial disclosures.

A version of this article appeared on Medscape.com .

TOPLINE:

Omitting axillary lymph node dissection does not increase the risk for recurrence or compromise 5-year overall survival outcomes in patients with early-stage, node-negative breast cancer with sentinel-node metastases undergoing surgery and radiation therapy.

METHODOLOGY:

• A growing body of evidence has indicated that patients with one or two positive sentinel nodes undergoing breast-conserving surgery and radiation therapy can skip axillary lymph node dissection and achieve similar outcomes compared with patients receiving axillary dissection.
• However, these earlier studies had notable limitations, such as limited statistical power, uncertain nodal radiotherapy target volumes, and minimal data on relevant clinical subgroups.
• To fill the gaps in the literature, the researchers conducted a trial with a large, inclusive cohort of patients with node-negative stage T1-T3 breast cancer who had one or two sentinel-node macrometastases and had undergone a mastectomy or breast-conserving surgery.
• The trial randomized 2540 patients to either completion axillary lymph node dissection (n = 1205) or sentinel-node biopsy only (n = 1335). Nearly 90% of patients received adjuvant radiation therapy, and the majority also received systematic therapy.
• Earlier recurrence-free survival findings and patient-reported outcomes were reported last December. The researchers now reported overall survival findings as well as secondary endpoints of breast cancer-specific survival.

TAKEAWAY:

• The researchers reported 191 recurrences or deaths over a median follow-up of 46.8 months; 62 patients (4.6%) in the sentinel-node biopsy–only group died, and 69 patients (5.7%) in the dissection group died.
• The biopsy-only group had an estimated 5-year overall survival of 92.9% compared with 92.0% in the dissection group and an estimated 5-year breast cancer-specific survival of 97.1% vs 96.6% in the dissection group.
• The estimated 5-year recurrence-free survival was 89.7% in the biopsy-only group vs 88.7% in the dissection group (hazard ratio [HR], 0.89; 95% CI, 0.66-1.19).
• This non-inferior difference held across all prespecified patient subgroups, except in patients with estrogen receptor-positive, human epidermal growth factor receptor 2–positive disease, in which sentinel biopsy alone appeared to be better (HR, 0.26).

IN PRACTICE:

“This trial provides robust evidence that the omission of completion axillary-lymph-node dissection was safe in patients with clinically node-negative T1, T2, or T3 breast cancer and one or two sentinel-node macrometastases who received adjuvant systemic treatment and radiation therapy according to national guidelines,” the authors concluded.

“It is clear that the role of axillary dissection is rapidly disappearing,” Kandace P. McGuire, MD, of Virginia Commonwealth University, Richmond, wrote in an accompanying editorial. “However, axillary staging continues to be vital with regard to decisions about appropriate breast cancer therapy.”

SOURCE:

This work, led by Jana de Boniface, MD, PhD, from Karolinska Institute, Stockholm, was published online in The New England Journal of Medicine, alongside the accompanying editorial by Dr. McGuire.

LIMITATIONS:

The study limitations include unavailable radiation therapy details for comparison, low male recruitment hindering sex-based analysis, short follow-up for luminal subtype breast cancer, unmet enrollment targets, and higher withdrawal rates in the dissection group.

DISCLOSURES:

This study was supported by the Swedish Research Council, Swedish Cancer Society, Nordic Cancer Union, and Swedish Breast Cancer Association. One coauthor reported receiving consultancy fees from various pharmaceutical companies outside this work.

A version of this article appeared on Medscape.com.

TOPLINE:

Omitting axillary lymph node dissection does not increase the risk for recurrence or compromise 5-year overall survival outcomes in patients with early-stage, node-negative breast cancer with sentinel-node metastases undergoing surgery and radiation therapy.

METHODOLOGY:

• A growing body of evidence has indicated that patients with one or two positive sentinel nodes undergoing breast-conserving surgery and radiation therapy can skip axillary lymph node dissection and achieve similar outcomes compared with patients receiving axillary dissection.
• However, these earlier studies had notable limitations, such as limited statistical power, uncertain nodal radiotherapy target volumes, and minimal data on relevant clinical subgroups.
• To fill the gaps in the literature, the researchers conducted a trial with a large, inclusive cohort of patients with node-negative stage T1-T3 breast cancer who had one or two sentinel-node macrometastases and had undergone a mastectomy or breast-conserving surgery.
• The trial randomized 2540 patients to either completion axillary lymph node dissection (n = 1205) or sentinel-node biopsy only (n = 1335). Nearly 90% of patients received adjuvant radiation therapy, and the majority also received systematic therapy.
• Earlier recurrence-free survival findings and patient-reported outcomes were reported last December. The researchers now reported overall survival findings as well as secondary endpoints of breast cancer-specific survival.

TAKEAWAY:

• The researchers reported 191 recurrences or deaths over a median follow-up of 46.8 months; 62 patients (4.6%) in the sentinel-node biopsy–only group died, and 69 patients (5.7%) in the dissection group died.
• The biopsy-only group had an estimated 5-year overall survival of 92.9% compared with 92.0% in the dissection group and an estimated 5-year breast cancer-specific survival of 97.1% vs 96.6% in the dissection group.
• The estimated 5-year recurrence-free survival was 89.7% in the biopsy-only group vs 88.7% in the dissection group (hazard ratio [HR], 0.89; 95% CI, 0.66-1.19).
• This non-inferior difference held across all prespecified patient subgroups, except in patients with estrogen receptor-positive, human epidermal growth factor receptor 2–positive disease, in which sentinel biopsy alone appeared to be better (HR, 0.26).

IN PRACTICE:

“This trial provides robust evidence that the omission of completion axillary-lymph-node dissection was safe in patients with clinically node-negative T1, T2, or T3 breast cancer and one or two sentinel-node macrometastases who received adjuvant systemic treatment and radiation therapy according to national guidelines,” the authors concluded.

“It is clear that the role of axillary dissection is rapidly disappearing,” Kandace P. McGuire, MD, of Virginia Commonwealth University, Richmond, wrote in an accompanying editorial. “However, axillary staging continues to be vital with regard to decisions about appropriate breast cancer therapy.”

SOURCE:

This work, led by Jana de Boniface, MD, PhD, from Karolinska Institute, Stockholm, was published online in The New England Journal of Medicine, alongside the accompanying editorial by Dr. McGuire.

LIMITATIONS:

The study limitations include unavailable radiation therapy details for comparison, low male recruitment hindering sex-based analysis, short follow-up for luminal subtype breast cancer, unmet enrollment targets, and higher withdrawal rates in the dissection group.

DISCLOSURES:

This study was supported by the Swedish Research Council, Swedish Cancer Society, Nordic Cancer Union, and Swedish Breast Cancer Association. One coauthor reported receiving consultancy fees from various pharmaceutical companies outside this work.

A version of this article appeared on Medscape.com.

TOPLINE:

Omitting axillary lymph node dissection does not increase the risk for recurrence or compromise 5-year overall survival outcomes in patients with early-stage, node-negative breast cancer with sentinel-node metastases undergoing surgery and radiation therapy.

METHODOLOGY:

• A growing body of evidence has indicated that patients with one or two positive sentinel nodes undergoing breast-conserving surgery and radiation therapy can skip axillary lymph node dissection and achieve similar outcomes compared with patients receiving axillary dissection.
• However, these earlier studies had notable limitations, such as limited statistical power, uncertain nodal radiotherapy target volumes, and minimal data on relevant clinical subgroups.
• To fill the gaps in the literature, the researchers conducted a trial with a large, inclusive cohort of patients with node-negative stage T1-T3 breast cancer who had one or two sentinel-node macrometastases and had undergone a mastectomy or breast-conserving surgery.
• The trial randomized 2540 patients to either completion axillary lymph node dissection (n = 1205) or sentinel-node biopsy only (n = 1335). Nearly 90% of patients received adjuvant radiation therapy, and the majority also received systematic therapy.
• Earlier recurrence-free survival findings and patient-reported outcomes were reported last December. The researchers now reported overall survival findings as well as secondary endpoints of breast cancer-specific survival.

TAKEAWAY:

• The researchers reported 191 recurrences or deaths over a median follow-up of 46.8 months; 62 patients (4.6%) in the sentinel-node biopsy–only group died, and 69 patients (5.7%) in the dissection group died.
• The biopsy-only group had an estimated 5-year overall survival of 92.9% compared with 92.0% in the dissection group and an estimated 5-year breast cancer-specific survival of 97.1% vs 96.6% in the dissection group.
• The estimated 5-year recurrence-free survival was 89.7% in the biopsy-only group vs 88.7% in the dissection group (hazard ratio [HR], 0.89; 95% CI, 0.66-1.19).
• This non-inferior difference held across all prespecified patient subgroups, except in patients with estrogen receptor-positive, human epidermal growth factor receptor 2–positive disease, in which sentinel biopsy alone appeared to be better (HR, 0.26).

IN PRACTICE:

“This trial provides robust evidence that the omission of completion axillary-lymph-node dissection was safe in patients with clinically node-negative T1, T2, or T3 breast cancer and one or two sentinel-node macrometastases who received adjuvant systemic treatment and radiation therapy according to national guidelines,” the authors concluded.

“It is clear that the role of axillary dissection is rapidly disappearing,” Kandace P. McGuire, MD, of Virginia Commonwealth University, Richmond, wrote in an accompanying editorial. “However, axillary staging continues to be vital with regard to decisions about appropriate breast cancer therapy.”

SOURCE:

This work, led by Jana de Boniface, MD, PhD, from Karolinska Institute, Stockholm, was published online in The New England Journal of Medicine, alongside the accompanying editorial by Dr. McGuire.

LIMITATIONS:

The study limitations include unavailable radiation therapy details for comparison, low male recruitment hindering sex-based analysis, short follow-up for luminal subtype breast cancer, unmet enrollment targets, and higher withdrawal rates in the dissection group.

DISCLOSURES:

This study was supported by the Swedish Research Council, Swedish Cancer Society, Nordic Cancer Union, and Swedish Breast Cancer Association. One coauthor reported receiving consultancy fees from various pharmaceutical companies outside this work.

A version of this article appeared on Medscape.com.

TOPLINE:

A low-fat vegan diet — high in fiber and carbohydrates and moderate in protein — reduces insulin requirement, increases insulin sensitivity, and improves glycemic control in individuals with type 1 diabetes (T1D) compared with a conventional portion-controlled diet.

METHODOLOGY:

• The effects of a low-fat vegan diet (without carbohydrate or portion restriction) were compared with those of a conventional portion-controlled, carbohydrate-controlled diet in 58 patients with T1D (age, ≥ 18 years) who had been receiving stable insulin treatment for the past 3 months.
• Participants were randomly assigned to receive either the vegan diet (n = 29), comprising vegetables, grains, legumes, and fruits, or the portion-controlled diet (n = 29), which reduced daily energy intake by 500-1000 kcal/d in participants with overweight while maintaining a stable carbohydrate intake.
• The primary clinical outcomes were insulin requirement (total daily dose of insulin), insulin sensitivity, and glycemic control (A1c).
• Other assessments included the blood, lipid profile, blood urea nitrogen, blood urea nitrogen-to-creatinine ratio, and body weight.

TAKEAWAY:

• The study was completed by 18 participants in the vegan-diet group and 17 in the portion-controlled group.
• In the vegan group, the total daily dose of insulin decreased by 12.1 units/d (P = .007) and insulin sensitivity increased by 6.6 g of carbohydrate per unit of insulin on average (P = .002), with no significant changes in the portion-controlled diet group.
• Participants on the vegan diet had lower levels of total and low-density lipoprotein cholesterol and blood urea nitrogen and a lower blood urea nitrogen-to-creatinine ratio (P for all < .001), whereas both vegan and portion-controlled groups had lower A1c levels.
• Body weight decreased by 5.2 kg (P < .001) in the vegan group; there were no significant changes in the portion-controlled group.
• For every 1-kg weight loss, there was a 2.16-unit decrease in the insulin total daily dose and a 0.9-unit increase in insulin sensitivity.

IN PRACTICE:

“This study provides substantial support for a low-fat vegan diet that is high in fiber and carbohydrates, low in fat, and moderate in protein” and suggests the potential therapeutic use of this diet in type 1 diabetes management, the authors wrote.

SOURCE:

The study led by Hana Kahleova, MD, PhD, Physicians Committee for Responsible Medicine, Washington, was published in Clinical Diabetes.

LIMITATIONS:

Dietary intake was recorded on the basis of self-reported data. A higher attrition rate was observed due to meal and blood glucose monitoring. The findings may have limited generalizability as the study participants comprised those seeking help for T1D.

DISCLOSURES:

The study was supported by the Physicians Committee for Responsible Medicine and a grant from the Institute for Technology in Healthcare. Some authors reported receiving compensation, being cofounders of a coaching program, writing books, providing nutrition coaching, giving lectures, or receiving royalties and honoraria from various sources.

A version of this article appeared on Medscape.com.

TOPLINE:

A low-fat vegan diet — high in fiber and carbohydrates and moderate in protein — reduces insulin requirement, increases insulin sensitivity, and improves glycemic control in individuals with type 1 diabetes (T1D) compared with a conventional portion-controlled diet.

METHODOLOGY:

• The effects of a low-fat vegan diet (without carbohydrate or portion restriction) were compared with those of a conventional portion-controlled, carbohydrate-controlled diet in 58 patients with T1D (age, ≥ 18 years) who had been receiving stable insulin treatment for the past 3 months.
• Participants were randomly assigned to receive either the vegan diet (n = 29), comprising vegetables, grains, legumes, and fruits, or the portion-controlled diet (n = 29), which reduced daily energy intake by 500-1000 kcal/d in participants with overweight while maintaining a stable carbohydrate intake.
• The primary clinical outcomes were insulin requirement (total daily dose of insulin), insulin sensitivity, and glycemic control (A1c).
• Other assessments included the blood, lipid profile, blood urea nitrogen, blood urea nitrogen-to-creatinine ratio, and body weight.

TAKEAWAY:

• The study was completed by 18 participants in the vegan-diet group and 17 in the portion-controlled group.
• In the vegan group, the total daily dose of insulin decreased by 12.1 units/d (P = .007) and insulin sensitivity increased by 6.6 g of carbohydrate per unit of insulin on average (P = .002), with no significant changes in the portion-controlled diet group.
• Participants on the vegan diet had lower levels of total and low-density lipoprotein cholesterol and blood urea nitrogen and a lower blood urea nitrogen-to-creatinine ratio (P for all < .001), whereas both vegan and portion-controlled groups had lower A1c levels.
• Body weight decreased by 5.2 kg (P < .001) in the vegan group; there were no significant changes in the portion-controlled group.
• For every 1-kg weight loss, there was a 2.16-unit decrease in the insulin total daily dose and a 0.9-unit increase in insulin sensitivity.

IN PRACTICE:

“This study provides substantial support for a low-fat vegan diet that is high in fiber and carbohydrates, low in fat, and moderate in protein” and suggests the potential therapeutic use of this diet in type 1 diabetes management, the authors wrote.

SOURCE:

The study led by Hana Kahleova, MD, PhD, Physicians Committee for Responsible Medicine, Washington, was published in Clinical Diabetes.

LIMITATIONS:

Dietary intake was recorded on the basis of self-reported data. A higher attrition rate was observed due to meal and blood glucose monitoring. The findings may have limited generalizability as the study participants comprised those seeking help for T1D.

DISCLOSURES:

The study was supported by the Physicians Committee for Responsible Medicine and a grant from the Institute for Technology in Healthcare. Some authors reported receiving compensation, being cofounders of a coaching program, writing books, providing nutrition coaching, giving lectures, or receiving royalties and honoraria from various sources.

A version of this article appeared on Medscape.com.

TOPLINE:

A low-fat vegan diet — high in fiber and carbohydrates and moderate in protein — reduces insulin requirement, increases insulin sensitivity, and improves glycemic control in individuals with type 1 diabetes (T1D) compared with a conventional portion-controlled diet.

METHODOLOGY:

• The effects of a low-fat vegan diet (without carbohydrate or portion restriction) were compared with those of a conventional portion-controlled, carbohydrate-controlled diet in 58 patients with T1D (age, ≥ 18 years) who had been receiving stable insulin treatment for the past 3 months.
• Participants were randomly assigned to receive either the vegan diet (n = 29), comprising vegetables, grains, legumes, and fruits, or the portion-controlled diet (n = 29), which reduced daily energy intake by 500-1000 kcal/d in participants with overweight while maintaining a stable carbohydrate intake.
• The primary clinical outcomes were insulin requirement (total daily dose of insulin), insulin sensitivity, and glycemic control (A1c).
• Other assessments included the blood, lipid profile, blood urea nitrogen, blood urea nitrogen-to-creatinine ratio, and body weight.

TAKEAWAY:

• The study was completed by 18 participants in the vegan-diet group and 17 in the portion-controlled group.
• In the vegan group, the total daily dose of insulin decreased by 12.1 units/d (P = .007) and insulin sensitivity increased by 6.6 g of carbohydrate per unit of insulin on average (P = .002), with no significant changes in the portion-controlled diet group.
• Participants on the vegan diet had lower levels of total and low-density lipoprotein cholesterol and blood urea nitrogen and a lower blood urea nitrogen-to-creatinine ratio (P for all < .001), whereas both vegan and portion-controlled groups had lower A1c levels.
• Body weight decreased by 5.2 kg (P < .001) in the vegan group; there were no significant changes in the portion-controlled group.
• For every 1-kg weight loss, there was a 2.16-unit decrease in the insulin total daily dose and a 0.9-unit increase in insulin sensitivity.

IN PRACTICE:

“This study provides substantial support for a low-fat vegan diet that is high in fiber and carbohydrates, low in fat, and moderate in protein” and suggests the potential therapeutic use of this diet in type 1 diabetes management, the authors wrote.

SOURCE:

The study led by Hana Kahleova, MD, PhD, Physicians Committee for Responsible Medicine, Washington, was published in Clinical Diabetes.

LIMITATIONS:

Dietary intake was recorded on the basis of self-reported data. A higher attrition rate was observed due to meal and blood glucose monitoring. The findings may have limited generalizability as the study participants comprised those seeking help for T1D.

DISCLOSURES:

The study was supported by the Physicians Committee for Responsible Medicine and a grant from the Institute for Technology in Healthcare. Some authors reported receiving compensation, being cofounders of a coaching program, writing books, providing nutrition coaching, giving lectures, or receiving royalties and honoraria from various sources.

A version of this article appeared on Medscape.com.

DENVER — After working for decades in a field littered with promising but failed clinical trials, a prominent researcher in amyotrophic lateral sclerosis (ALS) has decided to turn her attention to prevention, a direction of research that she thinks has more promise.

According to the gene-time hypothesis, duration of exposure to noxious chemicals and genetic susceptibility are key drivers of ALS risk, explained Eva Feldman, MD, PhD, director of the ALS Center of Excellence at the University of Michigan, Ann Arbor. She believes that existing research in risk modification is already promising.

“I think ALS prevention is real and attainable,” she said as this year’s recipient of the Sheila Essey Award for significant contributions in ALS research.

In describing her “pivot” to prevention from treatment at the 2024 annual meeting of the American Academy of Neurology, Dr. Feldman described her growing pessimism about treating a disease that has so consistently resisted even stabilization, let alone cure.

“I spent 10 years trying to repurpose IGF-1 as an ALS therapy. We took it from preclinical work all the way to a phase 3 multicenter trial, but in the end no effect was seen,” Dr. Feldman said,

This was followed by another 10 years spent on the promise of stem cells. In this case, she was eventually involved in two multicenter trials. In fact, trials are still ongoing in Europe, but Dr. Feldman said this strategy is “no longer going forward in the United States,” and she no longer anticipates favorable results.
 

The New Focus on Prevention

The basic concept in the prevention studies she is now working on with Stephen Goutman, MD, a frequent coauthor, and other colleagues at her center, is that the duration of exposure to persistent organic pollutants (POPs), along with some degree of genetic predisposition, determines risk for ALS. The simple idea is the reducing exposure will reduce ALS risk.

There is already substantial support for the underlying time-gene hypothesis, according to Dr. Feldman. Among several examples, she described work with 122 POPS that appear individually and in many cases collectively to correlate with ALS risk. Recent work with an environmental risk score (ERS) that permits studies of risk when accounting for exposure to families of pollutants, has supported these as potentially modifiable risks.

A high ERS “correlates with an ALS risk that is 3 to 4 times higher than a low score,” she said. In addition, those ALS patients with a high relative to a low ERS have a significant 0.6-year reduction in median survival.

Some specific POPs, such as pesticides, correlate with increased risk by themselves, but Dr. Feldman has begun focusing on occupational exposures, particularly in industries that are most likely to increase exposure POPs. Several of the POPs most implicated in ALS, such as polychlorinated biphenyls used in coolants and lubricants, organochlorine pesticides, and polybrominated diphenyl esters, are already banned or mostly banned in the United States, but they persist in the environment and remain legal elsewhere.

Dr. Feldman reported no potential conflicts of interest.

DENVER — After working for decades in a field littered with promising but failed clinical trials, a prominent researcher in amyotrophic lateral sclerosis (ALS) has decided to turn her attention to prevention, a direction of research that she thinks has more promise.

According to the gene-time hypothesis, duration of exposure to noxious chemicals and genetic susceptibility are key drivers of ALS risk, explained Eva Feldman, MD, PhD, director of the ALS Center of Excellence at the University of Michigan, Ann Arbor. She believes that existing research in risk modification is already promising.

“I think ALS prevention is real and attainable,” she said as this year’s recipient of the Sheila Essey Award for significant contributions in ALS research.

In describing her “pivot” to prevention from treatment at the 2024 annual meeting of the American Academy of Neurology, Dr. Feldman described her growing pessimism about treating a disease that has so consistently resisted even stabilization, let alone cure.

“I spent 10 years trying to repurpose IGF-1 as an ALS therapy. We took it from preclinical work all the way to a phase 3 multicenter trial, but in the end no effect was seen,” Dr. Feldman said,

This was followed by another 10 years spent on the promise of stem cells. In this case, she was eventually involved in two multicenter trials. In fact, trials are still ongoing in Europe, but Dr. Feldman said this strategy is “no longer going forward in the United States,” and she no longer anticipates favorable results.
 

The New Focus on Prevention

The basic concept in the prevention studies she is now working on with Stephen Goutman, MD, a frequent coauthor, and other colleagues at her center, is that the duration of exposure to persistent organic pollutants (POPs), along with some degree of genetic predisposition, determines risk for ALS. The simple idea is the reducing exposure will reduce ALS risk.

There is already substantial support for the underlying time-gene hypothesis, according to Dr. Feldman. Among several examples, she described work with 122 POPS that appear individually and in many cases collectively to correlate with ALS risk. Recent work with an environmental risk score (ERS) that permits studies of risk when accounting for exposure to families of pollutants, has supported these as potentially modifiable risks.

A high ERS “correlates with an ALS risk that is 3 to 4 times higher than a low score,” she said. In addition, those ALS patients with a high relative to a low ERS have a significant 0.6-year reduction in median survival.

Some specific POPs, such as pesticides, correlate with increased risk by themselves, but Dr. Feldman has begun focusing on occupational exposures, particularly in industries that are most likely to increase exposure POPs. Several of the POPs most implicated in ALS, such as polychlorinated biphenyls used in coolants and lubricants, organochlorine pesticides, and polybrominated diphenyl esters, are already banned or mostly banned in the United States, but they persist in the environment and remain legal elsewhere.

Dr. Feldman reported no potential conflicts of interest.

DENVER — After working for decades in a field littered with promising but failed clinical trials, a prominent researcher in amyotrophic lateral sclerosis (ALS) has decided to turn her attention to prevention, a direction of research that she thinks has more promise.

According to the gene-time hypothesis, duration of exposure to noxious chemicals and genetic susceptibility are key drivers of ALS risk, explained Eva Feldman, MD, PhD, director of the ALS Center of Excellence at the University of Michigan, Ann Arbor. She believes that existing research in risk modification is already promising.

“I think ALS prevention is real and attainable,” she said as this year’s recipient of the Sheila Essey Award for significant contributions in ALS research.

In describing her “pivot” to prevention from treatment at the 2024 annual meeting of the American Academy of Neurology, Dr. Feldman described her growing pessimism about treating a disease that has so consistently resisted even stabilization, let alone cure.

“I spent 10 years trying to repurpose IGF-1 as an ALS therapy. We took it from preclinical work all the way to a phase 3 multicenter trial, but in the end no effect was seen,” Dr. Feldman said,

This was followed by another 10 years spent on the promise of stem cells. In this case, she was eventually involved in two multicenter trials. In fact, trials are still ongoing in Europe, but Dr. Feldman said this strategy is “no longer going forward in the United States,” and she no longer anticipates favorable results.
 

The New Focus on Prevention

The basic concept in the prevention studies she is now working on with Stephen Goutman, MD, a frequent coauthor, and other colleagues at her center, is that the duration of exposure to persistent organic pollutants (POPs), along with some degree of genetic predisposition, determines risk for ALS. The simple idea is the reducing exposure will reduce ALS risk.

There is already substantial support for the underlying time-gene hypothesis, according to Dr. Feldman. Among several examples, she described work with 122 POPS that appear individually and in many cases collectively to correlate with ALS risk. Recent work with an environmental risk score (ERS) that permits studies of risk when accounting for exposure to families of pollutants, has supported these as potentially modifiable risks.

A high ERS “correlates with an ALS risk that is 3 to 4 times higher than a low score,” she said. In addition, those ALS patients with a high relative to a low ERS have a significant 0.6-year reduction in median survival.

Some specific POPs, such as pesticides, correlate with increased risk by themselves, but Dr. Feldman has begun focusing on occupational exposures, particularly in industries that are most likely to increase exposure POPs. Several of the POPs most implicated in ALS, such as polychlorinated biphenyls used in coolants and lubricants, organochlorine pesticides, and polybrominated diphenyl esters, are already banned or mostly banned in the United States, but they persist in the environment and remain legal elsewhere.

Dr. Feldman reported no potential conflicts of interest.

SYNLAB and ALCEDIAG recently launched the first blood test to assist in mental health diagnosis in France. The test is aimed at differentiating bipolar disorders from depression. The news may be surprising, given the challenges in diagnosing psychiatric conditions, especially when they share common symptoms such as recurrent depression and bipolar disorder.

Psychiatrists’ reactions to the new test are cautious. Many have echoed the sentiments of the French Association of Biological Psychiatry and Neuropsychopharmacology (AFPBN) and Stéphane Jamain, PhD, director of translational neuropsychiatry research (Inserm U955, Mondor Institute of Biomedical Research), who spoke with this news organization.

Early Diagnosis

Depression and bipolar disorders are two distinct psychiatric illnesses requiring different treatments. Early and accurate diagnosis and appropriate treatment are major challenges for clinicians, especially since untreated or inadequately treated bipolar disorder can lead to significant mental and physical health consequences for patients and their families.

Published studies indicate that it takes an average of 8-10 years, and sometimes even longer, to diagnose bipolar disorder. The diagnosis is based on a psychiatric clinical examination, which is conducted by a specialist using validated questionnaires and evaluation scales.

Early and accurate diagnosis of bipolar disorders that allows for appropriate treatment would be a significant advance for patients and their families. This is what the French laboratories SYNLAB, in partnership with ALCEDIAG, propose through myEDIT-B, a blood test described as “the first validated diagnostic aid test to differentiate depression and bipolar disorders.”

Whether this test, the availability of which has somewhat surprised the psychiatric medical and scientific community, will attract psychiatrists remains to be seen.

The AFPBN stated in a press release that “to date, no test meets conditions for clinical use.” For a diagnostic test to be scientifically valid, ethical, and usable in clinical practice, its development must meet strict criteria, as highlighted by the AFPBN. The approximately 10 criteria include the validation of the scientific results in at least two independent clinical studies or cohorts, satisfactory sensitivity (detection of true positives) and specificity (detection of false negatives), and cost that is ethically responsible and allows patient access, independent of commercial interests.

ALCEDIAG has reported two clinical studies, but only one has been published so far (in Translational Psychiatry) involving 400 patients. In this case, “these patients already had a well-established psychiatric condition, did not quite present the same symptoms between patients with recurrent depression and those with bipolar disorder and were not taking the same treatments,” noted Dr. Jamain.

Differentiating between bipolar disorder and depression is crucial, especially regarding treatments, because antidepressants given to a patient with bipolar disorder can induce a manic shift if they are not accompanied by mood stabilizers, Dr. Jamain acknowledged. Nevertheless, he believes that based on what the laboratory has published, it is difficult to comment on the test at this time.

RNA Editing 

Moreover, myEDIT-B is based on a technique that measures RNA editing modifications of specific markers in patients’ blood, which could lead to differences in amino acids within proteins. The technique is unique to the ALCEDIAG laboratory, which coupled it with an artificial intelligence tool that specifically selected 8 RNA sequences for analysis from thousands of edited sequences to obtain a differential signature for unipolar and bipolar depressions. “This method is niche, the trademark of ALCEDIAG,” said Dr. Jamain, who questions the significance of this “editing” on the periphery of the CNS.

“This technique differs from that adopted by most international consortia, which are very active in this research field. The latter technique compares differences in genome [DNA] nucleotides between individuals in large cohorts involving tens of thousands of people and identifies the most frequently occurring patterns associated with a pathology to deduce a risk of developing a psychiatric illness,” said Jamain. “However, the information provided by these large-scale studies does not allow us to define who is at risk for developing the disease any more than the simple observation of the familial recurrence [heritability] of it does.” 

Scientific Validation 

While ALCEDIAG boasts a sensitivity and specificity of more than 80% for its test, the psychiatric world remains cautious. Interviewed by France Info TV, Marion Leboyer, PhD, general director of the FondaMental Foundation, psychiatrist, and researcher (at AP-HP, Inserm in Créteil, France), highlighted the importance of encouraging research on psychiatric illnesses, especially that which will contribute to the understanding and treatment of patients with bipolar disorders. But she expressed caution regarding the test because of the absence of rigorous scientific validation through clinical trials.

Regarding “ALCEDIAG’s test and its commercial aspect, caution is warranted,” said Dr. Jamain. Only time will tell if psychiatrists will prescribe this €899 test, which currently is not reimbursed by social security (see box below). ALCEDIAG plans to submit a validation dossier to the US Food and Drug Administration.

Test Not Reimbursed by Social Security

The ALCEDIAG test will be available beginning in April 2024, by prescription, in SYNLAB France network laboratories. It is intended for patients aged 18 years and older who are being treated for a moderate or severe depressive episode. Test results are transmitted within 4 weeks to the prescribing psychiatrist, who will confirm the diagnosis to the patient during a consultation. Already available in Italy, this in vitro medical device has a CE-IVD marking. In France, however, it costs €899 and is not reimbursed by social security because of insufficient clinical evidence.

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

SYNLAB and ALCEDIAG recently launched the first blood test to assist in mental health diagnosis in France. The test is aimed at differentiating bipolar disorders from depression. The news may be surprising, given the challenges in diagnosing psychiatric conditions, especially when they share common symptoms such as recurrent depression and bipolar disorder.

Psychiatrists’ reactions to the new test are cautious. Many have echoed the sentiments of the French Association of Biological Psychiatry and Neuropsychopharmacology (AFPBN) and Stéphane Jamain, PhD, director of translational neuropsychiatry research (Inserm U955, Mondor Institute of Biomedical Research), who spoke with this news organization.

Early Diagnosis

Depression and bipolar disorders are two distinct psychiatric illnesses requiring different treatments. Early and accurate diagnosis and appropriate treatment are major challenges for clinicians, especially since untreated or inadequately treated bipolar disorder can lead to significant mental and physical health consequences for patients and their families.

Published studies indicate that it takes an average of 8-10 years, and sometimes even longer, to diagnose bipolar disorder. The diagnosis is based on a psychiatric clinical examination, which is conducted by a specialist using validated questionnaires and evaluation scales.

Early and accurate diagnosis of bipolar disorders that allows for appropriate treatment would be a significant advance for patients and their families. This is what the French laboratories SYNLAB, in partnership with ALCEDIAG, propose through myEDIT-B, a blood test described as “the first validated diagnostic aid test to differentiate depression and bipolar disorders.”

Whether this test, the availability of which has somewhat surprised the psychiatric medical and scientific community, will attract psychiatrists remains to be seen.

The AFPBN stated in a press release that “to date, no test meets conditions for clinical use.” For a diagnostic test to be scientifically valid, ethical, and usable in clinical practice, its development must meet strict criteria, as highlighted by the AFPBN. The approximately 10 criteria include the validation of the scientific results in at least two independent clinical studies or cohorts, satisfactory sensitivity (detection of true positives) and specificity (detection of false negatives), and cost that is ethically responsible and allows patient access, independent of commercial interests.

ALCEDIAG has reported two clinical studies, but only one has been published so far (in Translational Psychiatry) involving 400 patients. In this case, “these patients already had a well-established psychiatric condition, did not quite present the same symptoms between patients with recurrent depression and those with bipolar disorder and were not taking the same treatments,” noted Dr. Jamain.

Differentiating between bipolar disorder and depression is crucial, especially regarding treatments, because antidepressants given to a patient with bipolar disorder can induce a manic shift if they are not accompanied by mood stabilizers, Dr. Jamain acknowledged. Nevertheless, he believes that based on what the laboratory has published, it is difficult to comment on the test at this time.

RNA Editing 

Moreover, myEDIT-B is based on a technique that measures RNA editing modifications of specific markers in patients’ blood, which could lead to differences in amino acids within proteins. The technique is unique to the ALCEDIAG laboratory, which coupled it with an artificial intelligence tool that specifically selected 8 RNA sequences for analysis from thousands of edited sequences to obtain a differential signature for unipolar and bipolar depressions. “This method is niche, the trademark of ALCEDIAG,” said Dr. Jamain, who questions the significance of this “editing” on the periphery of the CNS.

“This technique differs from that adopted by most international consortia, which are very active in this research field. The latter technique compares differences in genome [DNA] nucleotides between individuals in large cohorts involving tens of thousands of people and identifies the most frequently occurring patterns associated with a pathology to deduce a risk of developing a psychiatric illness,” said Jamain. “However, the information provided by these large-scale studies does not allow us to define who is at risk for developing the disease any more than the simple observation of the familial recurrence [heritability] of it does.” 

Scientific Validation 

While ALCEDIAG boasts a sensitivity and specificity of more than 80% for its test, the psychiatric world remains cautious. Interviewed by France Info TV, Marion Leboyer, PhD, general director of the FondaMental Foundation, psychiatrist, and researcher (at AP-HP, Inserm in Créteil, France), highlighted the importance of encouraging research on psychiatric illnesses, especially that which will contribute to the understanding and treatment of patients with bipolar disorders. But she expressed caution regarding the test because of the absence of rigorous scientific validation through clinical trials.

Regarding “ALCEDIAG’s test and its commercial aspect, caution is warranted,” said Dr. Jamain. Only time will tell if psychiatrists will prescribe this €899 test, which currently is not reimbursed by social security (see box below). ALCEDIAG plans to submit a validation dossier to the US Food and Drug Administration.

Test Not Reimbursed by Social Security

The ALCEDIAG test will be available beginning in April 2024, by prescription, in SYNLAB France network laboratories. It is intended for patients aged 18 years and older who are being treated for a moderate or severe depressive episode. Test results are transmitted within 4 weeks to the prescribing psychiatrist, who will confirm the diagnosis to the patient during a consultation. Already available in Italy, this in vitro medical device has a CE-IVD marking. In France, however, it costs €899 and is not reimbursed by social security because of insufficient clinical evidence.

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

SYNLAB and ALCEDIAG recently launched the first blood test to assist in mental health diagnosis in France. The test is aimed at differentiating bipolar disorders from depression. The news may be surprising, given the challenges in diagnosing psychiatric conditions, especially when they share common symptoms such as recurrent depression and bipolar disorder.

Psychiatrists’ reactions to the new test are cautious. Many have echoed the sentiments of the French Association of Biological Psychiatry and Neuropsychopharmacology (AFPBN) and Stéphane Jamain, PhD, director of translational neuropsychiatry research (Inserm U955, Mondor Institute of Biomedical Research), who spoke with this news organization.

Early Diagnosis

Depression and bipolar disorders are two distinct psychiatric illnesses requiring different treatments. Early and accurate diagnosis and appropriate treatment are major challenges for clinicians, especially since untreated or inadequately treated bipolar disorder can lead to significant mental and physical health consequences for patients and their families.

Published studies indicate that it takes an average of 8-10 years, and sometimes even longer, to diagnose bipolar disorder. The diagnosis is based on a psychiatric clinical examination, which is conducted by a specialist using validated questionnaires and evaluation scales.

Early and accurate diagnosis of bipolar disorders that allows for appropriate treatment would be a significant advance for patients and their families. This is what the French laboratories SYNLAB, in partnership with ALCEDIAG, propose through myEDIT-B, a blood test described as “the first validated diagnostic aid test to differentiate depression and bipolar disorders.”

Whether this test, the availability of which has somewhat surprised the psychiatric medical and scientific community, will attract psychiatrists remains to be seen.

The AFPBN stated in a press release that “to date, no test meets conditions for clinical use.” For a diagnostic test to be scientifically valid, ethical, and usable in clinical practice, its development must meet strict criteria, as highlighted by the AFPBN. The approximately 10 criteria include the validation of the scientific results in at least two independent clinical studies or cohorts, satisfactory sensitivity (detection of true positives) and specificity (detection of false negatives), and cost that is ethically responsible and allows patient access, independent of commercial interests.

ALCEDIAG has reported two clinical studies, but only one has been published so far (in Translational Psychiatry) involving 400 patients. In this case, “these patients already had a well-established psychiatric condition, did not quite present the same symptoms between patients with recurrent depression and those with bipolar disorder and were not taking the same treatments,” noted Dr. Jamain.

Differentiating between bipolar disorder and depression is crucial, especially regarding treatments, because antidepressants given to a patient with bipolar disorder can induce a manic shift if they are not accompanied by mood stabilizers, Dr. Jamain acknowledged. Nevertheless, he believes that based on what the laboratory has published, it is difficult to comment on the test at this time.

RNA Editing 

Moreover, myEDIT-B is based on a technique that measures RNA editing modifications of specific markers in patients’ blood, which could lead to differences in amino acids within proteins. The technique is unique to the ALCEDIAG laboratory, which coupled it with an artificial intelligence tool that specifically selected 8 RNA sequences for analysis from thousands of edited sequences to obtain a differential signature for unipolar and bipolar depressions. “This method is niche, the trademark of ALCEDIAG,” said Dr. Jamain, who questions the significance of this “editing” on the periphery of the CNS.

“This technique differs from that adopted by most international consortia, which are very active in this research field. The latter technique compares differences in genome [DNA] nucleotides between individuals in large cohorts involving tens of thousands of people and identifies the most frequently occurring patterns associated with a pathology to deduce a risk of developing a psychiatric illness,” said Jamain. “However, the information provided by these large-scale studies does not allow us to define who is at risk for developing the disease any more than the simple observation of the familial recurrence [heritability] of it does.” 

Scientific Validation 

While ALCEDIAG boasts a sensitivity and specificity of more than 80% for its test, the psychiatric world remains cautious. Interviewed by France Info TV, Marion Leboyer, PhD, general director of the FondaMental Foundation, psychiatrist, and researcher (at AP-HP, Inserm in Créteil, France), highlighted the importance of encouraging research on psychiatric illnesses, especially that which will contribute to the understanding and treatment of patients with bipolar disorders. But she expressed caution regarding the test because of the absence of rigorous scientific validation through clinical trials.

Regarding “ALCEDIAG’s test and its commercial aspect, caution is warranted,” said Dr. Jamain. Only time will tell if psychiatrists will prescribe this €899 test, which currently is not reimbursed by social security (see box below). ALCEDIAG plans to submit a validation dossier to the US Food and Drug Administration.

Test Not Reimbursed by Social Security

The ALCEDIAG test will be available beginning in April 2024, by prescription, in SYNLAB France network laboratories. It is intended for patients aged 18 years and older who are being treated for a moderate or severe depressive episode. Test results are transmitted within 4 weeks to the prescribing psychiatrist, who will confirm the diagnosis to the patient during a consultation. Already available in Italy, this in vitro medical device has a CE-IVD marking. In France, however, it costs €899 and is not reimbursed by social security because of insufficient clinical evidence.

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Tocilizumab administration results in high remission rates in adult-onset Still’s disease (AOSD), but the recurrence rate increases on tocilizumab discontinuation, with a longer tocilizumab interval and lower prednisolone dose being critical for successful tocilizumab withdrawal.

METHODOLOGY:

• Tocilizumab is effective in reducing systemic inflammation and lowering glucocorticoid doses in patients with AOSD; however, the possibility of tocilizumab withdrawal has not been explored.
• This retrospective study assessed whether tocilizumab can be discontinued after achieving remission in 48 patients with AOSD.
• The systemic feature score, Pouchot score, and modified Pouchot score were used to evaluate the disease activity.
• Remission was characterized by the absence of symptoms related to Still’s disease, normal levels of erythrocyte sedimentation rate and C-reactive protein, and absence of treatment intensification requirement.
• Recurrence after tocilizumab discontinuation was defined as a disease flare with AOSD treatment intensification that necessitated either a ≥ 1.5-fold increase in glucocorticoid dosage and/or the initiation of a biologic agent.

TAKEAWAY:

• Over a median observation period of 5.1 years, 38 (79.2%) patients achieved remission at 6 months, of which 13 discontinued tocilizumab and the remaining 25 continued it.
• Among patients who discontinued tocilizumab on achieving remission, recurrence was noted in 50% within a year, typically after a mean period of 5.5 months after discontinuation.
• Patients in remission with longer tocilizumab intervals (> 14 days; P < .0002) or lower prednisolone doses (< 7 mg/d; P = .001) at the time of tocilizumab discontinuation showed better recurrence-free rates than those without.
• The duration of tocilizumab use, systemic feature score, and serum ferritin levels at tocilizumab discontinuation were not significantly different between patients who experienced recurrence and those who did not.

IN PRACTICE:

“Stable conditions with extended intervals of tocilizumab administration and very low doses of concomitant glucocorticoids are essential for successful discontinuation,” the authors wrote.

SOURCE:

The study was led by Hiroya Tamai, Division of Rheumatology, Department of Internal Medicine, Keio University School of Medicine, Tokyo, Japan, and published online in Rheumatology (Oxford).

LIMITATIONS:

The study was limited by its retrospective study design and a small sample size. Moreover, there could have been a selection bias as the attending physicians could use their discretion to initiate or stop tocilizumab treatment. The absence of a universally agreed-upon definition for remission or recurrence in AOSD made comparing the findings of this study with those of others challenging.

DISCLOSURES:

This study did not receive any specific funding from any bodies in public, commercial, or nonprofit sectors. Some of the authors reported receiving honoraria and research support from various pharmaceutical companies.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Tocilizumab administration results in high remission rates in adult-onset Still’s disease (AOSD), but the recurrence rate increases on tocilizumab discontinuation, with a longer tocilizumab interval and lower prednisolone dose being critical for successful tocilizumab withdrawal.

METHODOLOGY:

• Tocilizumab is effective in reducing systemic inflammation and lowering glucocorticoid doses in patients with AOSD; however, the possibility of tocilizumab withdrawal has not been explored.
• This retrospective study assessed whether tocilizumab can be discontinued after achieving remission in 48 patients with AOSD.
• The systemic feature score, Pouchot score, and modified Pouchot score were used to evaluate the disease activity.
• Remission was characterized by the absence of symptoms related to Still’s disease, normal levels of erythrocyte sedimentation rate and C-reactive protein, and absence of treatment intensification requirement.
• Recurrence after tocilizumab discontinuation was defined as a disease flare with AOSD treatment intensification that necessitated either a ≥ 1.5-fold increase in glucocorticoid dosage and/or the initiation of a biologic agent.

TAKEAWAY:

• Over a median observation period of 5.1 years, 38 (79.2%) patients achieved remission at 6 months, of which 13 discontinued tocilizumab and the remaining 25 continued it.
• Among patients who discontinued tocilizumab on achieving remission, recurrence was noted in 50% within a year, typically after a mean period of 5.5 months after discontinuation.
• Patients in remission with longer tocilizumab intervals (> 14 days; P < .0002) or lower prednisolone doses (< 7 mg/d; P = .001) at the time of tocilizumab discontinuation showed better recurrence-free rates than those without.
• The duration of tocilizumab use, systemic feature score, and serum ferritin levels at tocilizumab discontinuation were not significantly different between patients who experienced recurrence and those who did not.

IN PRACTICE:

“Stable conditions with extended intervals of tocilizumab administration and very low doses of concomitant glucocorticoids are essential for successful discontinuation,” the authors wrote.

SOURCE:

The study was led by Hiroya Tamai, Division of Rheumatology, Department of Internal Medicine, Keio University School of Medicine, Tokyo, Japan, and published online in Rheumatology (Oxford).

LIMITATIONS:

The study was limited by its retrospective study design and a small sample size. Moreover, there could have been a selection bias as the attending physicians could use their discretion to initiate or stop tocilizumab treatment. The absence of a universally agreed-upon definition for remission or recurrence in AOSD made comparing the findings of this study with those of others challenging.

DISCLOSURES:

This study did not receive any specific funding from any bodies in public, commercial, or nonprofit sectors. Some of the authors reported receiving honoraria and research support from various pharmaceutical companies.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Tocilizumab administration results in high remission rates in adult-onset Still’s disease (AOSD), but the recurrence rate increases on tocilizumab discontinuation, with a longer tocilizumab interval and lower prednisolone dose being critical for successful tocilizumab withdrawal.

METHODOLOGY:

• Tocilizumab is effective in reducing systemic inflammation and lowering glucocorticoid doses in patients with AOSD; however, the possibility of tocilizumab withdrawal has not been explored.
• This retrospective study assessed whether tocilizumab can be discontinued after achieving remission in 48 patients with AOSD.
• The systemic feature score, Pouchot score, and modified Pouchot score were used to evaluate the disease activity.
• Remission was characterized by the absence of symptoms related to Still’s disease, normal levels of erythrocyte sedimentation rate and C-reactive protein, and absence of treatment intensification requirement.
• Recurrence after tocilizumab discontinuation was defined as a disease flare with AOSD treatment intensification that necessitated either a ≥ 1.5-fold increase in glucocorticoid dosage and/or the initiation of a biologic agent.

TAKEAWAY:

• Over a median observation period of 5.1 years, 38 (79.2%) patients achieved remission at 6 months, of which 13 discontinued tocilizumab and the remaining 25 continued it.
• Among patients who discontinued tocilizumab on achieving remission, recurrence was noted in 50% within a year, typically after a mean period of 5.5 months after discontinuation.
• Patients in remission with longer tocilizumab intervals (> 14 days; P < .0002) or lower prednisolone doses (< 7 mg/d; P = .001) at the time of tocilizumab discontinuation showed better recurrence-free rates than those without.
• The duration of tocilizumab use, systemic feature score, and serum ferritin levels at tocilizumab discontinuation were not significantly different between patients who experienced recurrence and those who did not.

IN PRACTICE:

“Stable conditions with extended intervals of tocilizumab administration and very low doses of concomitant glucocorticoids are essential for successful discontinuation,” the authors wrote.

SOURCE:

The study was led by Hiroya Tamai, Division of Rheumatology, Department of Internal Medicine, Keio University School of Medicine, Tokyo, Japan, and published online in Rheumatology (Oxford).

LIMITATIONS:

The study was limited by its retrospective study design and a small sample size. Moreover, there could have been a selection bias as the attending physicians could use their discretion to initiate or stop tocilizumab treatment. The absence of a universally agreed-upon definition for remission or recurrence in AOSD made comparing the findings of this study with those of others challenging.

DISCLOSURES:

This study did not receive any specific funding from any bodies in public, commercial, or nonprofit sectors. Some of the authors reported receiving honoraria and research support from various pharmaceutical companies.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Subcutaneous tocilizumab provides durable disease control rates in patients with polyarticular and systemic juvenile idiopathic arthritis (pJIA and sJIA, respectively).

METHODOLOGY:

• This long-term extension (LTE) study included 44 patients with pJIA and 38 patients with sJIA, according to the International League of Associations for Rheumatology criteria, from two 52-week phase 1b trials (NCT01904292 and NCT01904279).
• In the core trials, the dosing frequency of subcutaneous tocilizumab was determined by weight: Every 3 weeks for those < 30 kg in pJIA and every 2 weeks for those ≥ 30 kg; in sJIA, initially every 10 days for those < 30 kg, transitioning to every 2 weeks, and weekly for those ≥ 30 kg.
• Patients who had adequate disease control with subcutaneous tocilizumab, comparable with the use of intravenous tocilizumab in the core trials, continued to receive subcutaneous tocilizumab.
• The study outcome was the change in Juvenile Arthritis Disease Activity Score on 71 joints (JADAS-71, range 0-101).

TAKEAWAY:

• Disease control remained stable in both groups, with sustained improvements in median JADAS-71 scores in pJIA (−0.2 with lower frequency dosing to −0.5 with higher frequency) and sJIA (−0.1 at both dosing frequencies).
• In the pJIA group, 90% and 53% of patients weighing < 30 kg and ≥ 30 kg achieved inactive disease, respectively, whereas in the sJIA group, the respective rates were 91% and 92%.
• A total of five of 15 patients with pJIA weighing ≥ 30 kg who received subcutaneous tocilizumab every 2 weeks achieved clinical remission, whereas in other groups, the clinical remission rates ranged from 74% to 92%.
• Six patients with pJIA reported seven serious adverse events (SAEs), while five patients with sJIA experienced six SAEs. Five patients with pJIA and one patient with sJIA reported serious infections.

IN PRACTICE:

The authors concluded that subcutaneous tocilizumab treatment provided long-term disease control in patients with pJIA or sJIA, with a safety profile consistent with past studies of tocilizumab.

SOURCE:

The study was led by Hermine I. Brunner, MD, director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center. It was published online in Rheumatology (Oxford).

LIMITATIONS:

The open-label design and lack of a control group limited the analysis. Only a few patients continued the treatment for 5 years.

DISCLOSURES:

This work was supported by F. Hoffmann-La Roche Ltd. Eight authors reported receiving honoraria and consulting or speaker fees from various pharma sources. The remaining authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Subcutaneous tocilizumab provides durable disease control rates in patients with polyarticular and systemic juvenile idiopathic arthritis (pJIA and sJIA, respectively).

METHODOLOGY:

• This long-term extension (LTE) study included 44 patients with pJIA and 38 patients with sJIA, according to the International League of Associations for Rheumatology criteria, from two 52-week phase 1b trials (NCT01904292 and NCT01904279).
• In the core trials, the dosing frequency of subcutaneous tocilizumab was determined by weight: Every 3 weeks for those < 30 kg in pJIA and every 2 weeks for those ≥ 30 kg; in sJIA, initially every 10 days for those < 30 kg, transitioning to every 2 weeks, and weekly for those ≥ 30 kg.
• Patients who had adequate disease control with subcutaneous tocilizumab, comparable with the use of intravenous tocilizumab in the core trials, continued to receive subcutaneous tocilizumab.
• The study outcome was the change in Juvenile Arthritis Disease Activity Score on 71 joints (JADAS-71, range 0-101).

TAKEAWAY:

• Disease control remained stable in both groups, with sustained improvements in median JADAS-71 scores in pJIA (−0.2 with lower frequency dosing to −0.5 with higher frequency) and sJIA (−0.1 at both dosing frequencies).
• In the pJIA group, 90% and 53% of patients weighing < 30 kg and ≥ 30 kg achieved inactive disease, respectively, whereas in the sJIA group, the respective rates were 91% and 92%.
• A total of five of 15 patients with pJIA weighing ≥ 30 kg who received subcutaneous tocilizumab every 2 weeks achieved clinical remission, whereas in other groups, the clinical remission rates ranged from 74% to 92%.
• Six patients with pJIA reported seven serious adverse events (SAEs), while five patients with sJIA experienced six SAEs. Five patients with pJIA and one patient with sJIA reported serious infections.

IN PRACTICE:

The authors concluded that subcutaneous tocilizumab treatment provided long-term disease control in patients with pJIA or sJIA, with a safety profile consistent with past studies of tocilizumab.

SOURCE:

The study was led by Hermine I. Brunner, MD, director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center. It was published online in Rheumatology (Oxford).

LIMITATIONS:

The open-label design and lack of a control group limited the analysis. Only a few patients continued the treatment for 5 years.

DISCLOSURES:

This work was supported by F. Hoffmann-La Roche Ltd. Eight authors reported receiving honoraria and consulting or speaker fees from various pharma sources. The remaining authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Subcutaneous tocilizumab provides durable disease control rates in patients with polyarticular and systemic juvenile idiopathic arthritis (pJIA and sJIA, respectively).

METHODOLOGY:

• This long-term extension (LTE) study included 44 patients with pJIA and 38 patients with sJIA, according to the International League of Associations for Rheumatology criteria, from two 52-week phase 1b trials (NCT01904292 and NCT01904279).
• In the core trials, the dosing frequency of subcutaneous tocilizumab was determined by weight: Every 3 weeks for those < 30 kg in pJIA and every 2 weeks for those ≥ 30 kg; in sJIA, initially every 10 days for those < 30 kg, transitioning to every 2 weeks, and weekly for those ≥ 30 kg.
• Patients who had adequate disease control with subcutaneous tocilizumab, comparable with the use of intravenous tocilizumab in the core trials, continued to receive subcutaneous tocilizumab.
• The study outcome was the change in Juvenile Arthritis Disease Activity Score on 71 joints (JADAS-71, range 0-101).

TAKEAWAY:

• Disease control remained stable in both groups, with sustained improvements in median JADAS-71 scores in pJIA (−0.2 with lower frequency dosing to −0.5 with higher frequency) and sJIA (−0.1 at both dosing frequencies).
• In the pJIA group, 90% and 53% of patients weighing < 30 kg and ≥ 30 kg achieved inactive disease, respectively, whereas in the sJIA group, the respective rates were 91% and 92%.
• A total of five of 15 patients with pJIA weighing ≥ 30 kg who received subcutaneous tocilizumab every 2 weeks achieved clinical remission, whereas in other groups, the clinical remission rates ranged from 74% to 92%.
• Six patients with pJIA reported seven serious adverse events (SAEs), while five patients with sJIA experienced six SAEs. Five patients with pJIA and one patient with sJIA reported serious infections.

IN PRACTICE:

The authors concluded that subcutaneous tocilizumab treatment provided long-term disease control in patients with pJIA or sJIA, with a safety profile consistent with past studies of tocilizumab.

SOURCE:

The study was led by Hermine I. Brunner, MD, director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center. It was published online in Rheumatology (Oxford).

LIMITATIONS:

The open-label design and lack of a control group limited the analysis. Only a few patients continued the treatment for 5 years.

DISCLOSURES:

This work was supported by F. Hoffmann-La Roche Ltd. Eight authors reported receiving honoraria and consulting or speaker fees from various pharma sources. The remaining authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Higher levels of body fat and visceral adipose tissue are associated with increased functional disability and reduced spinal mobility in patients with axial spondyloarthritis (axSpA) receiving biologic disease-modifying antirheumatic drugs (bDMARDs).

METHODOLOGY:

• Research showed that patients with axSpA respond poorly to tumor necrosis factor inhibitors if they have a high body mass index (BMI) or obesity; however, studies delving into the association between biologic therapy and body composition are limited.
• Researchers investigated the association between body composition evaluated by bioimpedance analysis and disease activity, physical function, and mobility in 74 patients with axSpA (mean age, 36.5; 71.6% men) at 6 months and 1 year after initiating bDMARDs.
• These participants from the German Spondyloarthritis Inception Cohort presented with high disease activity despite previous treatment with nonsteroidal anti-inflammatory drugs and initiated bDMARD therapy between 2015 and 2019.
• Bath Ankylosing Spondylitis Disease Activity Index and Axial Spondyloarthritis Disease Activity Score were used to measure disease activity, while Bath Ankylosing Spondylitis Functional Index and Bath Ankylosing Spondylitis Mobility Index assessed physical function and spinal mobility, respectively.
• BMI, fat mass, fat mass index, and visceral adipose tissue (VAT) were used to determine body composition along with other parameters.

TAKEAWAY:

• Higher BMI (parameter estimates [β], 0.081; 95% CI, 0.016-0.145), fat mass (β, 0.037; 95% CI, 0.004-0.070), and fat mass index (β, 0.125; 95% CI, 0.031-0.219) were associated with worse physical function in the overall population.
• VAT was positively associated with reduced spinal mobility (β, 0.201; 95% CI, 0.071-0.332), particularly in men.
• In women, an increase in VAT was linked to worse disease activity and functional disability.
• Treatment with bDMARDs reduced all disease activity parameters but led to an increase in BMI and fat-related parameters, indicating that lifestyle modifications are also necessary to achieve the desired outcomes with bDMARD therapy.

IN PRACTICE:

“Overall, our findings highlight the importance of maintaining a healthy body weight and body composition — characterized by adequate lean mass and reduced FM [fat mass] — to improve physical function and quality of life in patients with SpA,” the authors wrote.

SOURCE:

The study was led by Valeria Rios Rodriguez, MD, department of gastroenterology, infectiology and rheumatology, Charité Universitätsmedizin Berlin, Germany. It was published online March 20, 2024, in Rheumatology (Oxford). 

LIMITATIONS:

This study lacked a control group of patients with axSpA who did not receive biologics. It also did not include dietary habits and comorbidities such as hypertension or diabetes. Additionally, bioimpedance analysis was chosen as the method to assess body composition instead of dual-energy x-ray absorptiometry. 

DISCLOSURES:

The study was funded by the German Federal Ministry of Education and Research and the Berlin Institute of Health. Some of the authors declared receiving personal fees, grants, and consulting fees from various pharmaceutical companies.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Higher levels of body fat and visceral adipose tissue are associated with increased functional disability and reduced spinal mobility in patients with axial spondyloarthritis (axSpA) receiving biologic disease-modifying antirheumatic drugs (bDMARDs).

METHODOLOGY:

• Research showed that patients with axSpA respond poorly to tumor necrosis factor inhibitors if they have a high body mass index (BMI) or obesity; however, studies delving into the association between biologic therapy and body composition are limited.
• Researchers investigated the association between body composition evaluated by bioimpedance analysis and disease activity, physical function, and mobility in 74 patients with axSpA (mean age, 36.5; 71.6% men) at 6 months and 1 year after initiating bDMARDs.
• These participants from the German Spondyloarthritis Inception Cohort presented with high disease activity despite previous treatment with nonsteroidal anti-inflammatory drugs and initiated bDMARD therapy between 2015 and 2019.
• Bath Ankylosing Spondylitis Disease Activity Index and Axial Spondyloarthritis Disease Activity Score were used to measure disease activity, while Bath Ankylosing Spondylitis Functional Index and Bath Ankylosing Spondylitis Mobility Index assessed physical function and spinal mobility, respectively.
• BMI, fat mass, fat mass index, and visceral adipose tissue (VAT) were used to determine body composition along with other parameters.

TAKEAWAY:

• Higher BMI (parameter estimates [β], 0.081; 95% CI, 0.016-0.145), fat mass (β, 0.037; 95% CI, 0.004-0.070), and fat mass index (β, 0.125; 95% CI, 0.031-0.219) were associated with worse physical function in the overall population.
• VAT was positively associated with reduced spinal mobility (β, 0.201; 95% CI, 0.071-0.332), particularly in men.
• In women, an increase in VAT was linked to worse disease activity and functional disability.
• Treatment with bDMARDs reduced all disease activity parameters but led to an increase in BMI and fat-related parameters, indicating that lifestyle modifications are also necessary to achieve the desired outcomes with bDMARD therapy.

IN PRACTICE:

“Overall, our findings highlight the importance of maintaining a healthy body weight and body composition — characterized by adequate lean mass and reduced FM [fat mass] — to improve physical function and quality of life in patients with SpA,” the authors wrote.

SOURCE:

The study was led by Valeria Rios Rodriguez, MD, department of gastroenterology, infectiology and rheumatology, Charité Universitätsmedizin Berlin, Germany. It was published online March 20, 2024, in Rheumatology (Oxford). 

LIMITATIONS:

This study lacked a control group of patients with axSpA who did not receive biologics. It also did not include dietary habits and comorbidities such as hypertension or diabetes. Additionally, bioimpedance analysis was chosen as the method to assess body composition instead of dual-energy x-ray absorptiometry. 

DISCLOSURES:

The study was funded by the German Federal Ministry of Education and Research and the Berlin Institute of Health. Some of the authors declared receiving personal fees, grants, and consulting fees from various pharmaceutical companies.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Higher levels of body fat and visceral adipose tissue are associated with increased functional disability and reduced spinal mobility in patients with axial spondyloarthritis (axSpA) receiving biologic disease-modifying antirheumatic drugs (bDMARDs).

METHODOLOGY:

• Research showed that patients with axSpA respond poorly to tumor necrosis factor inhibitors if they have a high body mass index (BMI) or obesity; however, studies delving into the association between biologic therapy and body composition are limited.
• Researchers investigated the association between body composition evaluated by bioimpedance analysis and disease activity, physical function, and mobility in 74 patients with axSpA (mean age, 36.5; 71.6% men) at 6 months and 1 year after initiating bDMARDs.
• These participants from the German Spondyloarthritis Inception Cohort presented with high disease activity despite previous treatment with nonsteroidal anti-inflammatory drugs and initiated bDMARD therapy between 2015 and 2019.
• Bath Ankylosing Spondylitis Disease Activity Index and Axial Spondyloarthritis Disease Activity Score were used to measure disease activity, while Bath Ankylosing Spondylitis Functional Index and Bath Ankylosing Spondylitis Mobility Index assessed physical function and spinal mobility, respectively.
• BMI, fat mass, fat mass index, and visceral adipose tissue (VAT) were used to determine body composition along with other parameters.

TAKEAWAY:

• Higher BMI (parameter estimates [β], 0.081; 95% CI, 0.016-0.145), fat mass (β, 0.037; 95% CI, 0.004-0.070), and fat mass index (β, 0.125; 95% CI, 0.031-0.219) were associated with worse physical function in the overall population.
• VAT was positively associated with reduced spinal mobility (β, 0.201; 95% CI, 0.071-0.332), particularly in men.
• In women, an increase in VAT was linked to worse disease activity and functional disability.
• Treatment with bDMARDs reduced all disease activity parameters but led to an increase in BMI and fat-related parameters, indicating that lifestyle modifications are also necessary to achieve the desired outcomes with bDMARD therapy.

IN PRACTICE:

“Overall, our findings highlight the importance of maintaining a healthy body weight and body composition — characterized by adequate lean mass and reduced FM [fat mass] — to improve physical function and quality of life in patients with SpA,” the authors wrote.

SOURCE:

The study was led by Valeria Rios Rodriguez, MD, department of gastroenterology, infectiology and rheumatology, Charité Universitätsmedizin Berlin, Germany. It was published online March 20, 2024, in Rheumatology (Oxford). 

LIMITATIONS:

This study lacked a control group of patients with axSpA who did not receive biologics. It also did not include dietary habits and comorbidities such as hypertension or diabetes. Additionally, bioimpedance analysis was chosen as the method to assess body composition instead of dual-energy x-ray absorptiometry. 

DISCLOSURES:

The study was funded by the German Federal Ministry of Education and Research and the Berlin Institute of Health. Some of the authors declared receiving personal fees, grants, and consulting fees from various pharmaceutical companies.
 

A version of this article appeared on Medscape.com.

TOPLINE:

According to this meta-analysis, psoriatic arthritis (PsA) affects 112 out of every 100,000 adults globally, with higher rates observed in Europe and North America than in Asia and South America, according to an analysis of 30 studies.

METHODOLOGY:

• Many previous epidemiological studies have estimated the global prevalence of PsA but have reported marked variations, which could be explained by differences in methodology and inclusion criteria.
• This meta-analysis used data from 30 studies conducted between 1982 and 2020 to estimate the worldwide prevalence of PsA in the general adult population, giving particular attention to methodological differences among the included studies.
• The included studies were either population-based (n = 13) or based on health administrative records (n = 17) and covered over 180 million adults across 24 countries.
• Overall, 15 studies were from Europe, seven from Asia, six from North America, and two from South America.

TAKEAWAY:

• The global prevalence of PsA was estimated at 113 (95% CI, 64-198) and 109 (75-158) cases per 100,000 based on population-based studies and health administrative data studies, respectively.
• The pooled global prevalence of PsA (combining the population-based and health administrative studies) was 112 cases per 100,000 (95% CI, 83-151).
• Combining both study designs, the global prevalence rates of PsA were 188 (95% CI, 128-289) cases per 100,000 for Europe, 48 (95% CI, 20-115) for Asia, 133 (95% CI, 93-191) for North America, and 17 (95% CI, 4-70) for South America.

IN PRACTICE:

“Robust estimates of prevalence are crucial for healthcare planning and resource allocation,” wrote the authors.

SOURCE:

The study was conducted by Stephanie Lembke, MSc, and colleagues from the Aberdeen Centre for Arthritis and Musculoskeletal Health, University of Aberdeen, Scotland. It was published online in Rheumatology (Oxford).

LIMITATIONS:

The meta-analysis had high levels of uncertainty and high heterogeneity between studies. In countries with unequal healthcare access, using data from statutory or private insurance databases to calculate PsA prevalence may systematically exclude uninsured individuals or those covered by private insurers. Moreover, the data were insufficient for a statistically meaningful subgroup analysis.

DISCLOSURES:

The study did not receive any specific funding from any public, commercial, or nonprofit sectors to carry out this work. The authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

According to this meta-analysis, psoriatic arthritis (PsA) affects 112 out of every 100,000 adults globally, with higher rates observed in Europe and North America than in Asia and South America, according to an analysis of 30 studies.

METHODOLOGY:

• Many previous epidemiological studies have estimated the global prevalence of PsA but have reported marked variations, which could be explained by differences in methodology and inclusion criteria.
• This meta-analysis used data from 30 studies conducted between 1982 and 2020 to estimate the worldwide prevalence of PsA in the general adult population, giving particular attention to methodological differences among the included studies.
• The included studies were either population-based (n = 13) or based on health administrative records (n = 17) and covered over 180 million adults across 24 countries.
• Overall, 15 studies were from Europe, seven from Asia, six from North America, and two from South America.

TAKEAWAY:

• The global prevalence of PsA was estimated at 113 (95% CI, 64-198) and 109 (75-158) cases per 100,000 based on population-based studies and health administrative data studies, respectively.
• The pooled global prevalence of PsA (combining the population-based and health administrative studies) was 112 cases per 100,000 (95% CI, 83-151).
• Combining both study designs, the global prevalence rates of PsA were 188 (95% CI, 128-289) cases per 100,000 for Europe, 48 (95% CI, 20-115) for Asia, 133 (95% CI, 93-191) for North America, and 17 (95% CI, 4-70) for South America.

IN PRACTICE:

“Robust estimates of prevalence are crucial for healthcare planning and resource allocation,” wrote the authors.

SOURCE:

The study was conducted by Stephanie Lembke, MSc, and colleagues from the Aberdeen Centre for Arthritis and Musculoskeletal Health, University of Aberdeen, Scotland. It was published online in Rheumatology (Oxford).

LIMITATIONS:

The meta-analysis had high levels of uncertainty and high heterogeneity between studies. In countries with unequal healthcare access, using data from statutory or private insurance databases to calculate PsA prevalence may systematically exclude uninsured individuals or those covered by private insurers. Moreover, the data were insufficient for a statistically meaningful subgroup analysis.

DISCLOSURES:

The study did not receive any specific funding from any public, commercial, or nonprofit sectors to carry out this work. The authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

According to this meta-analysis, psoriatic arthritis (PsA) affects 112 out of every 100,000 adults globally, with higher rates observed in Europe and North America than in Asia and South America, according to an analysis of 30 studies.

METHODOLOGY:

• Many previous epidemiological studies have estimated the global prevalence of PsA but have reported marked variations, which could be explained by differences in methodology and inclusion criteria.
• This meta-analysis used data from 30 studies conducted between 1982 and 2020 to estimate the worldwide prevalence of PsA in the general adult population, giving particular attention to methodological differences among the included studies.
• The included studies were either population-based (n = 13) or based on health administrative records (n = 17) and covered over 180 million adults across 24 countries.
• Overall, 15 studies were from Europe, seven from Asia, six from North America, and two from South America.

TAKEAWAY:

• The global prevalence of PsA was estimated at 113 (95% CI, 64-198) and 109 (75-158) cases per 100,000 based on population-based studies and health administrative data studies, respectively.
• The pooled global prevalence of PsA (combining the population-based and health administrative studies) was 112 cases per 100,000 (95% CI, 83-151).
• Combining both study designs, the global prevalence rates of PsA were 188 (95% CI, 128-289) cases per 100,000 for Europe, 48 (95% CI, 20-115) for Asia, 133 (95% CI, 93-191) for North America, and 17 (95% CI, 4-70) for South America.

IN PRACTICE:

“Robust estimates of prevalence are crucial for healthcare planning and resource allocation,” wrote the authors.

SOURCE:

The study was conducted by Stephanie Lembke, MSc, and colleagues from the Aberdeen Centre for Arthritis and Musculoskeletal Health, University of Aberdeen, Scotland. It was published online in Rheumatology (Oxford).

LIMITATIONS:

The meta-analysis had high levels of uncertainty and high heterogeneity between studies. In countries with unequal healthcare access, using data from statutory or private insurance databases to calculate PsA prevalence may systematically exclude uninsured individuals or those covered by private insurers. Moreover, the data were insufficient for a statistically meaningful subgroup analysis.

DISCLOSURES:

The study did not receive any specific funding from any public, commercial, or nonprofit sectors to carry out this work. The authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.