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More than one-third of adults in the US could have NAFLD by 2050

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Mon, 12/04/2023 - 12:26

More than one out of three adults in the United States could have nonalcoholic fatty liver disease (NAFLD) by 2050, substantially increasing the national clinical burden, according to investigators.

These findings suggest that health care systems should prepare for “large increases” in cases of hepatocellular carcinoma (HCC) and need for liver transplants, reported lead author Phuc Le, PhD, MPH, of the Cleveland Clinic, and colleagues.

Cleveland Clinic
Dr. Phuc Le

“Following the alarming rise in prevalence of obesity and diabetes, NAFLD is projected to become the leading indication for liver transplant in the United States in the next decade,” Dr. Le and colleagues wrote in their abstract for the annual meeting of the American Association for the Study of Liver Diseases. “A better understanding of the clinical burden associated with NAFLD will enable health systems to prepare to meet this imminent demand from patients.”

To this end, Dr. Le and colleagues developed an agent-based state transition model to predict future prevalence of NAFLD and associated outcomes.

In the first part of the model, the investigators simulated population growth in the United States using Census Bureau data, including new births and immigration, from the year 2000 onward. The second part of the model simulated natural progression of NAFLD in adults via 14 associated conditions and events, including steatosis, nonalcoholic steatohepatitis (NASH), HCC, liver transplants, liver-related mortality, and others.

By first comparing simulated findings with actual findings between 2000 and 2018, the investigators confirmed that their model could reliably predict the intended epidemiological parameters.

Next, they turned their model toward the future.

It predicted that the prevalence of NAFLD among US adults will rise from 27.8% in 2020 to 34.3% in 2050. Over the same timeframe, prevalence of NASH is predicted to increase from 20.0% to 21.8%, proportion of NAFLD cases developing cirrhosis is expected to increase from 1.9% to 3.1%, and liver-related mortality is estimated to rise from 0.4% to 1% of all deaths.

The model also predicted that the burden of HCC will increase from 10,400 to 19,300 new cases per year, while liver transplant burden will more than double, from 1,700 to 4,200 transplants per year.

“Our model forecasts substantial clinical burden of NAFLD over the next three decades,” Dr. Le said in a virtual press conference. “And in the absence of effective treatments, health systems should plan for large increases in the number of liver cancer cases and the need for liver transplant.”

During the press conference, Norah Terrault, MD, president of the AASLD from the University of Southern California, Los Angeles, noted that all of the reported outcomes, including increasing rates of liver cancer, cirrhosis, and transplants are “potentially preventable.”

Keck School of Medicine
Dr. Norah Terrault

Dr. Terrault went on to suggest ways of combating this increasing burden of NAFLD, which she referred to as metabolic dysfunction–associated steatotic liver disease (MASLD), the name now recommended by the AASLD.

“There’s no way we’re going to be able to transplant our way out of this,” Dr. Terrault said. “We need to be bringing greater awareness both to patients, as well as to providers about how we seek out the diagnosis. And we need to bring greater awareness to the population around the things that contribute to MASLD.”

Rates of obesity and diabetes continue to rise, Dr. Terrault said, explaining why MASLD is more common than ever. To counteract these trends, she called for greater awareness of driving factors, such as dietary choices and sedentary lifestyle.

“These are all really important messages that we want to get out to the population, and are really the cornerstones for how we approach the management of patients who have MASLD,” Dr. Terrault said.

In discussion with Dr. Terrault, Dr. Le agreed that increased education may help stem the rising tide of disease, while treatment advances could also increase the odds of a brighter future.

“If we improve our management of NAFLD, or NAFLD-related comorbidities, and if we can develop an effective treatment for NAFLD, then obviously the future would not be so dark,” Dr. Le said, noting promising phase 3 data that would be presented at the meeting. “We are hopeful that the future of disease burden will not be as bad as our model predicts.”

The study was funded by the Agency for Healthcare Research and Quality. The investigators disclosed no conflicts of interest.

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More than one out of three adults in the United States could have nonalcoholic fatty liver disease (NAFLD) by 2050, substantially increasing the national clinical burden, according to investigators.

These findings suggest that health care systems should prepare for “large increases” in cases of hepatocellular carcinoma (HCC) and need for liver transplants, reported lead author Phuc Le, PhD, MPH, of the Cleveland Clinic, and colleagues.

Cleveland Clinic
Dr. Phuc Le

“Following the alarming rise in prevalence of obesity and diabetes, NAFLD is projected to become the leading indication for liver transplant in the United States in the next decade,” Dr. Le and colleagues wrote in their abstract for the annual meeting of the American Association for the Study of Liver Diseases. “A better understanding of the clinical burden associated with NAFLD will enable health systems to prepare to meet this imminent demand from patients.”

To this end, Dr. Le and colleagues developed an agent-based state transition model to predict future prevalence of NAFLD and associated outcomes.

In the first part of the model, the investigators simulated population growth in the United States using Census Bureau data, including new births and immigration, from the year 2000 onward. The second part of the model simulated natural progression of NAFLD in adults via 14 associated conditions and events, including steatosis, nonalcoholic steatohepatitis (NASH), HCC, liver transplants, liver-related mortality, and others.

By first comparing simulated findings with actual findings between 2000 and 2018, the investigators confirmed that their model could reliably predict the intended epidemiological parameters.

Next, they turned their model toward the future.

It predicted that the prevalence of NAFLD among US adults will rise from 27.8% in 2020 to 34.3% in 2050. Over the same timeframe, prevalence of NASH is predicted to increase from 20.0% to 21.8%, proportion of NAFLD cases developing cirrhosis is expected to increase from 1.9% to 3.1%, and liver-related mortality is estimated to rise from 0.4% to 1% of all deaths.

The model also predicted that the burden of HCC will increase from 10,400 to 19,300 new cases per year, while liver transplant burden will more than double, from 1,700 to 4,200 transplants per year.

“Our model forecasts substantial clinical burden of NAFLD over the next three decades,” Dr. Le said in a virtual press conference. “And in the absence of effective treatments, health systems should plan for large increases in the number of liver cancer cases and the need for liver transplant.”

During the press conference, Norah Terrault, MD, president of the AASLD from the University of Southern California, Los Angeles, noted that all of the reported outcomes, including increasing rates of liver cancer, cirrhosis, and transplants are “potentially preventable.”

Keck School of Medicine
Dr. Norah Terrault

Dr. Terrault went on to suggest ways of combating this increasing burden of NAFLD, which she referred to as metabolic dysfunction–associated steatotic liver disease (MASLD), the name now recommended by the AASLD.

“There’s no way we’re going to be able to transplant our way out of this,” Dr. Terrault said. “We need to be bringing greater awareness both to patients, as well as to providers about how we seek out the diagnosis. And we need to bring greater awareness to the population around the things that contribute to MASLD.”

Rates of obesity and diabetes continue to rise, Dr. Terrault said, explaining why MASLD is more common than ever. To counteract these trends, she called for greater awareness of driving factors, such as dietary choices and sedentary lifestyle.

“These are all really important messages that we want to get out to the population, and are really the cornerstones for how we approach the management of patients who have MASLD,” Dr. Terrault said.

In discussion with Dr. Terrault, Dr. Le agreed that increased education may help stem the rising tide of disease, while treatment advances could also increase the odds of a brighter future.

“If we improve our management of NAFLD, or NAFLD-related comorbidities, and if we can develop an effective treatment for NAFLD, then obviously the future would not be so dark,” Dr. Le said, noting promising phase 3 data that would be presented at the meeting. “We are hopeful that the future of disease burden will not be as bad as our model predicts.”

The study was funded by the Agency for Healthcare Research and Quality. The investigators disclosed no conflicts of interest.

More than one out of three adults in the United States could have nonalcoholic fatty liver disease (NAFLD) by 2050, substantially increasing the national clinical burden, according to investigators.

These findings suggest that health care systems should prepare for “large increases” in cases of hepatocellular carcinoma (HCC) and need for liver transplants, reported lead author Phuc Le, PhD, MPH, of the Cleveland Clinic, and colleagues.

Cleveland Clinic
Dr. Phuc Le

“Following the alarming rise in prevalence of obesity and diabetes, NAFLD is projected to become the leading indication for liver transplant in the United States in the next decade,” Dr. Le and colleagues wrote in their abstract for the annual meeting of the American Association for the Study of Liver Diseases. “A better understanding of the clinical burden associated with NAFLD will enable health systems to prepare to meet this imminent demand from patients.”

To this end, Dr. Le and colleagues developed an agent-based state transition model to predict future prevalence of NAFLD and associated outcomes.

In the first part of the model, the investigators simulated population growth in the United States using Census Bureau data, including new births and immigration, from the year 2000 onward. The second part of the model simulated natural progression of NAFLD in adults via 14 associated conditions and events, including steatosis, nonalcoholic steatohepatitis (NASH), HCC, liver transplants, liver-related mortality, and others.

By first comparing simulated findings with actual findings between 2000 and 2018, the investigators confirmed that their model could reliably predict the intended epidemiological parameters.

Next, they turned their model toward the future.

It predicted that the prevalence of NAFLD among US adults will rise from 27.8% in 2020 to 34.3% in 2050. Over the same timeframe, prevalence of NASH is predicted to increase from 20.0% to 21.8%, proportion of NAFLD cases developing cirrhosis is expected to increase from 1.9% to 3.1%, and liver-related mortality is estimated to rise from 0.4% to 1% of all deaths.

The model also predicted that the burden of HCC will increase from 10,400 to 19,300 new cases per year, while liver transplant burden will more than double, from 1,700 to 4,200 transplants per year.

“Our model forecasts substantial clinical burden of NAFLD over the next three decades,” Dr. Le said in a virtual press conference. “And in the absence of effective treatments, health systems should plan for large increases in the number of liver cancer cases and the need for liver transplant.”

During the press conference, Norah Terrault, MD, president of the AASLD from the University of Southern California, Los Angeles, noted that all of the reported outcomes, including increasing rates of liver cancer, cirrhosis, and transplants are “potentially preventable.”

Keck School of Medicine
Dr. Norah Terrault

Dr. Terrault went on to suggest ways of combating this increasing burden of NAFLD, which she referred to as metabolic dysfunction–associated steatotic liver disease (MASLD), the name now recommended by the AASLD.

“There’s no way we’re going to be able to transplant our way out of this,” Dr. Terrault said. “We need to be bringing greater awareness both to patients, as well as to providers about how we seek out the diagnosis. And we need to bring greater awareness to the population around the things that contribute to MASLD.”

Rates of obesity and diabetes continue to rise, Dr. Terrault said, explaining why MASLD is more common than ever. To counteract these trends, she called for greater awareness of driving factors, such as dietary choices and sedentary lifestyle.

“These are all really important messages that we want to get out to the population, and are really the cornerstones for how we approach the management of patients who have MASLD,” Dr. Terrault said.

In discussion with Dr. Terrault, Dr. Le agreed that increased education may help stem the rising tide of disease, while treatment advances could also increase the odds of a brighter future.

“If we improve our management of NAFLD, or NAFLD-related comorbidities, and if we can develop an effective treatment for NAFLD, then obviously the future would not be so dark,” Dr. Le said, noting promising phase 3 data that would be presented at the meeting. “We are hopeful that the future of disease burden will not be as bad as our model predicts.”

The study was funded by the Agency for Healthcare Research and Quality. The investigators disclosed no conflicts of interest.

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Food insecurity increases risk of adolescent MASLD

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Mon, 12/04/2023 - 12:21

Adolescents facing food insecurity have a significantly increased risk of metabolic dysfunction-associated steatotic liver disease (MASLD), likely due to overconsumption of low-cost, ultra-processed, unbalanced diets, according to a recent study.

These findings suggest that more work is needed to ensure that eligible adolescents can access Supplemental Nutrition Assistance Program (SNAP) benefits and have opportunities to engage in physical activities through school-associated programs, reported principal investigator Zobair M. Younossi, MD, MPH, professor and chairman of the Beatty Liver and Obesity Research Program, Inova Health System, Falls Church, Virginia, and colleagues.

Dr. Zobair M. Younossi

Dr. Younossi presented the findings in November during a press conference at the annual meeting of the American Association for the Study of Liver Diseases.

“Food insecurity among children is about 10.2% in the United States,” Dr. Younossi said. “[Food insecurity has] been shown to be a risk factor for MASLD among adults, but the data and children and adolescents are really lacking at the moment.”

To address this knowledge gap, Dr. Younossi and colleagues analyzed data from 771 adolescents aged 12-18 years in the National Health and Nutrition Examination Survey (2017-2018). Among these participants, 9.8% reported food insecurity and 10.8% had MASLD. Rates of obesity and central obesity were 22.5% and 45.4%, respectively, while 1.0% had diabetes and 20.9% had prediabetes.

Among adolescents facing food insecurity, more than half (51.5%) did not eat enough food, a vast majority (93.2%) could not access a balanced meal, and almost all (98.9%) relied upon low-cost food for daily sustenance.

The prevalence of MASLD in the food insecure group was almost twice as high as in the food secure group (18.7% vs 9.9%), and advanced fibrosis was about 9 times more common (2.8% vs. 0.3%). Food insecure participants were also more likely to come from a low-income household (70.4% vs. 25.7%) and participate in SNAP (62.4% vs. 25.1%).

Adjusting for SNAP participation, demographic factors, and metabolic disease showed that food insecurity independently increased risk of MASLD by more than twofold (odds ratio [OR], 2.62; 95% CI, 1.07–6.41). The negative effect of food insecurity was almost twice as strong in participants living in a low-income household (OR, 4.79; 95% CI, 1.44–15.86).

“The association between food insecurity and MASLD/NAFLD is most likely the result of not being able to eat a balanced meal and more likely having to purchase low-cost food,” Dr. Younossi said. “Together, these factors may lead to a cycle of overeating along with the overconsumption of ultra-processed foods and sugar-sweetened food and beverages.”

He went on to suggest that more work is needed to remove “systemic and structural barriers” that prevent eligible adolescents from participating in SNAP, while offering support so they can participate in “more physical activity in school and in after-school programs.”

Elliot Benjamin Tapper, MD, associate professor of medicine at the University of Michigan, Ann Arbor, recently published a similar study in the Journal of Clinical Gastroenterology linking food scarcity and MASLD in adults.

Michigan Medicine
Dr. Elliot Benjamin Tapper

In an interview, Dr. Tapper praised this new study by Dr. Younossi and colleagues because it “identifies a serious unmet need” among younger individuals, who may stand to benefit most from early intervention.

“The goal [of screening] is to prevent the development of progressive disease,” Dr. Tapper said. “Our current guidelines for screening for advanced liver disease and people with risk factors focus exclusively on adults. If you waited longer, then there’s a risk that these [younger] people [in the study] would have progressed to a later stage of disease.”

Dr. Tapper predicted increased enthusiasm for MAFLD screening among adolescents in response to these findings, but he cautioned that conventional educational intervention is unlikely to yield significant benefit.

“If you’re food insecure, you can’t go out and buy salmon and olive oil to follow the Mediterranean diet,” Dr. Tapper said. In this era, where the people who are at risk tomorrow are young and food insecure, we have to come up with a way of tailoring our interventions to the means that are available to these patients.”

To this end, health care providers need to collaborate with individuals who have personally dealt with food scarcity to implement practicable interventions.

“Referral to social work has to be paired with some kind of standard teaching,” Dr. Tapper said. “How would I use social and nutritional assistance programs to eat in a liver-healthy way? What can I avoid? [Educational materials] should be written by and edited by people with lived experience; i.e., people who have food insecurity or have walked a mile in those shoes.”

Dr. Younossi disclosed relationships with Merck, Abbott, AstraZeneca, and others. Dr. Tapper disclosed relationships with Takeda, Novo Nordisk, Madrigal, and others.

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Adolescents facing food insecurity have a significantly increased risk of metabolic dysfunction-associated steatotic liver disease (MASLD), likely due to overconsumption of low-cost, ultra-processed, unbalanced diets, according to a recent study.

These findings suggest that more work is needed to ensure that eligible adolescents can access Supplemental Nutrition Assistance Program (SNAP) benefits and have opportunities to engage in physical activities through school-associated programs, reported principal investigator Zobair M. Younossi, MD, MPH, professor and chairman of the Beatty Liver and Obesity Research Program, Inova Health System, Falls Church, Virginia, and colleagues.

Dr. Zobair M. Younossi

Dr. Younossi presented the findings in November during a press conference at the annual meeting of the American Association for the Study of Liver Diseases.

“Food insecurity among children is about 10.2% in the United States,” Dr. Younossi said. “[Food insecurity has] been shown to be a risk factor for MASLD among adults, but the data and children and adolescents are really lacking at the moment.”

To address this knowledge gap, Dr. Younossi and colleagues analyzed data from 771 adolescents aged 12-18 years in the National Health and Nutrition Examination Survey (2017-2018). Among these participants, 9.8% reported food insecurity and 10.8% had MASLD. Rates of obesity and central obesity were 22.5% and 45.4%, respectively, while 1.0% had diabetes and 20.9% had prediabetes.

Among adolescents facing food insecurity, more than half (51.5%) did not eat enough food, a vast majority (93.2%) could not access a balanced meal, and almost all (98.9%) relied upon low-cost food for daily sustenance.

The prevalence of MASLD in the food insecure group was almost twice as high as in the food secure group (18.7% vs 9.9%), and advanced fibrosis was about 9 times more common (2.8% vs. 0.3%). Food insecure participants were also more likely to come from a low-income household (70.4% vs. 25.7%) and participate in SNAP (62.4% vs. 25.1%).

Adjusting for SNAP participation, demographic factors, and metabolic disease showed that food insecurity independently increased risk of MASLD by more than twofold (odds ratio [OR], 2.62; 95% CI, 1.07–6.41). The negative effect of food insecurity was almost twice as strong in participants living in a low-income household (OR, 4.79; 95% CI, 1.44–15.86).

“The association between food insecurity and MASLD/NAFLD is most likely the result of not being able to eat a balanced meal and more likely having to purchase low-cost food,” Dr. Younossi said. “Together, these factors may lead to a cycle of overeating along with the overconsumption of ultra-processed foods and sugar-sweetened food and beverages.”

He went on to suggest that more work is needed to remove “systemic and structural barriers” that prevent eligible adolescents from participating in SNAP, while offering support so they can participate in “more physical activity in school and in after-school programs.”

Elliot Benjamin Tapper, MD, associate professor of medicine at the University of Michigan, Ann Arbor, recently published a similar study in the Journal of Clinical Gastroenterology linking food scarcity and MASLD in adults.

Michigan Medicine
Dr. Elliot Benjamin Tapper

In an interview, Dr. Tapper praised this new study by Dr. Younossi and colleagues because it “identifies a serious unmet need” among younger individuals, who may stand to benefit most from early intervention.

“The goal [of screening] is to prevent the development of progressive disease,” Dr. Tapper said. “Our current guidelines for screening for advanced liver disease and people with risk factors focus exclusively on adults. If you waited longer, then there’s a risk that these [younger] people [in the study] would have progressed to a later stage of disease.”

Dr. Tapper predicted increased enthusiasm for MAFLD screening among adolescents in response to these findings, but he cautioned that conventional educational intervention is unlikely to yield significant benefit.

“If you’re food insecure, you can’t go out and buy salmon and olive oil to follow the Mediterranean diet,” Dr. Tapper said. In this era, where the people who are at risk tomorrow are young and food insecure, we have to come up with a way of tailoring our interventions to the means that are available to these patients.”

To this end, health care providers need to collaborate with individuals who have personally dealt with food scarcity to implement practicable interventions.

“Referral to social work has to be paired with some kind of standard teaching,” Dr. Tapper said. “How would I use social and nutritional assistance programs to eat in a liver-healthy way? What can I avoid? [Educational materials] should be written by and edited by people with lived experience; i.e., people who have food insecurity or have walked a mile in those shoes.”

Dr. Younossi disclosed relationships with Merck, Abbott, AstraZeneca, and others. Dr. Tapper disclosed relationships with Takeda, Novo Nordisk, Madrigal, and others.

Adolescents facing food insecurity have a significantly increased risk of metabolic dysfunction-associated steatotic liver disease (MASLD), likely due to overconsumption of low-cost, ultra-processed, unbalanced diets, according to a recent study.

These findings suggest that more work is needed to ensure that eligible adolescents can access Supplemental Nutrition Assistance Program (SNAP) benefits and have opportunities to engage in physical activities through school-associated programs, reported principal investigator Zobair M. Younossi, MD, MPH, professor and chairman of the Beatty Liver and Obesity Research Program, Inova Health System, Falls Church, Virginia, and colleagues.

Dr. Zobair M. Younossi

Dr. Younossi presented the findings in November during a press conference at the annual meeting of the American Association for the Study of Liver Diseases.

“Food insecurity among children is about 10.2% in the United States,” Dr. Younossi said. “[Food insecurity has] been shown to be a risk factor for MASLD among adults, but the data and children and adolescents are really lacking at the moment.”

To address this knowledge gap, Dr. Younossi and colleagues analyzed data from 771 adolescents aged 12-18 years in the National Health and Nutrition Examination Survey (2017-2018). Among these participants, 9.8% reported food insecurity and 10.8% had MASLD. Rates of obesity and central obesity were 22.5% and 45.4%, respectively, while 1.0% had diabetes and 20.9% had prediabetes.

Among adolescents facing food insecurity, more than half (51.5%) did not eat enough food, a vast majority (93.2%) could not access a balanced meal, and almost all (98.9%) relied upon low-cost food for daily sustenance.

The prevalence of MASLD in the food insecure group was almost twice as high as in the food secure group (18.7% vs 9.9%), and advanced fibrosis was about 9 times more common (2.8% vs. 0.3%). Food insecure participants were also more likely to come from a low-income household (70.4% vs. 25.7%) and participate in SNAP (62.4% vs. 25.1%).

Adjusting for SNAP participation, demographic factors, and metabolic disease showed that food insecurity independently increased risk of MASLD by more than twofold (odds ratio [OR], 2.62; 95% CI, 1.07–6.41). The negative effect of food insecurity was almost twice as strong in participants living in a low-income household (OR, 4.79; 95% CI, 1.44–15.86).

“The association between food insecurity and MASLD/NAFLD is most likely the result of not being able to eat a balanced meal and more likely having to purchase low-cost food,” Dr. Younossi said. “Together, these factors may lead to a cycle of overeating along with the overconsumption of ultra-processed foods and sugar-sweetened food and beverages.”

He went on to suggest that more work is needed to remove “systemic and structural barriers” that prevent eligible adolescents from participating in SNAP, while offering support so they can participate in “more physical activity in school and in after-school programs.”

Elliot Benjamin Tapper, MD, associate professor of medicine at the University of Michigan, Ann Arbor, recently published a similar study in the Journal of Clinical Gastroenterology linking food scarcity and MASLD in adults.

Michigan Medicine
Dr. Elliot Benjamin Tapper

In an interview, Dr. Tapper praised this new study by Dr. Younossi and colleagues because it “identifies a serious unmet need” among younger individuals, who may stand to benefit most from early intervention.

“The goal [of screening] is to prevent the development of progressive disease,” Dr. Tapper said. “Our current guidelines for screening for advanced liver disease and people with risk factors focus exclusively on adults. If you waited longer, then there’s a risk that these [younger] people [in the study] would have progressed to a later stage of disease.”

Dr. Tapper predicted increased enthusiasm for MAFLD screening among adolescents in response to these findings, but he cautioned that conventional educational intervention is unlikely to yield significant benefit.

“If you’re food insecure, you can’t go out and buy salmon and olive oil to follow the Mediterranean diet,” Dr. Tapper said. In this era, where the people who are at risk tomorrow are young and food insecure, we have to come up with a way of tailoring our interventions to the means that are available to these patients.”

To this end, health care providers need to collaborate with individuals who have personally dealt with food scarcity to implement practicable interventions.

“Referral to social work has to be paired with some kind of standard teaching,” Dr. Tapper said. “How would I use social and nutritional assistance programs to eat in a liver-healthy way? What can I avoid? [Educational materials] should be written by and edited by people with lived experience; i.e., people who have food insecurity or have walked a mile in those shoes.”

Dr. Younossi disclosed relationships with Merck, Abbott, AstraZeneca, and others. Dr. Tapper disclosed relationships with Takeda, Novo Nordisk, Madrigal, and others.

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Prognostic tool identifies alcohol relapse risk after liver transplant

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Thu, 11/30/2023 - 11:40

A novel prognostic tool based on social determinants effectively predicted increased risk of alcohol use relapse in adults who underwent liver transplants for alcoholic liver disease, based on data from 140 individuals.

Alcohol relapse after liver transplant ranges from 4% to as high as 95% among patients with alcoholic liver disease (ALD) and better tools are needed to identify those at increased risk, Jiten P. Kothadia, MD, of the University of Tennessee Health Science Center, Memphis, said in a presentation given in October at the annual meeting of the American College of Gastroenterology.

Dr. Kothadia and colleagues evaluated the effectiveness of the Social Determinant Acuity Tool (S-DAT), which stratified patients in terms of successful post-liver transplant outcomes from excellent (S-DAT scores 0-6) to poor candidates (scores 35-40). The S-DAT categories included cognitive function, mental health, social support, coping skills, financial status, compliance, alcohol abuse, substance abuse, reliability, legal issues, understanding the transplant process, and desire for transplant.

The study population included 140 adults with alcoholic liver disease who underwent a liver transplant between January 2016 and November 2021 at a single center. Before surgery, all patients underwent a thorough psychosocial evaluation using the S-DAT. The mean age of the participants was 53.4 years, 107 were male, and 67.9% had abstained from alcohol for more than 6 months prior to transplant.

The primary outcome of post-liver transplant alcohol relapse was defined as any alcohol use regardless of the amount or frequency, based on patient interviews or blood or urine tests.

Overall, the rate of relapse was 23.6%; and the rate within a year was 18.6%. In a multivariate analysis, S-DAT score was a significant predictor of relapse (odds ratio [OR] 1.65, P = .000). Other independent predictors of relapse were post-LT alcohol treatment (OR 7.11, P = .02), smoking history (OR 0.15, P = .03), and marital status (OR 60.28, P = .000). The area under the receiver operative curves (AUROC) for the S-DAT score to predict alcohol relapse within 1 year after LT was 0.77.

The sensitivity of the S-DAT for predicting relapse risk was 96.2%, and specificity was 40.4%; positive and negative predictive values were 26.9% and 97.9%, respectively.

The high sensitivity and negative predictive values of the S-DAT make it a useful screening tool for identifying patients at low risk of alcohol relapse after a liver transplant, Dr. Kothadia said in an interview. “Our score will guide risk-based interventions post-LT to reduce post-LT relapse and improve long-term outcomes.”

The findings included only data from a single center, which may limit generalizability, Dr. Kothadia said. The tool is not yet clinically available, he noted.

“We would like to perform external validation of our S-DAT score as it stresses the importance of these psychosocial variables,” and to confirm the findings in larger, multicenter, prospective clinical trials, he said.

The study received no outside funding. Dr. Kothadia indicated no relevant financial relationships.

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A novel prognostic tool based on social determinants effectively predicted increased risk of alcohol use relapse in adults who underwent liver transplants for alcoholic liver disease, based on data from 140 individuals.

Alcohol relapse after liver transplant ranges from 4% to as high as 95% among patients with alcoholic liver disease (ALD) and better tools are needed to identify those at increased risk, Jiten P. Kothadia, MD, of the University of Tennessee Health Science Center, Memphis, said in a presentation given in October at the annual meeting of the American College of Gastroenterology.

Dr. Kothadia and colleagues evaluated the effectiveness of the Social Determinant Acuity Tool (S-DAT), which stratified patients in terms of successful post-liver transplant outcomes from excellent (S-DAT scores 0-6) to poor candidates (scores 35-40). The S-DAT categories included cognitive function, mental health, social support, coping skills, financial status, compliance, alcohol abuse, substance abuse, reliability, legal issues, understanding the transplant process, and desire for transplant.

The study population included 140 adults with alcoholic liver disease who underwent a liver transplant between January 2016 and November 2021 at a single center. Before surgery, all patients underwent a thorough psychosocial evaluation using the S-DAT. The mean age of the participants was 53.4 years, 107 were male, and 67.9% had abstained from alcohol for more than 6 months prior to transplant.

The primary outcome of post-liver transplant alcohol relapse was defined as any alcohol use regardless of the amount or frequency, based on patient interviews or blood or urine tests.

Overall, the rate of relapse was 23.6%; and the rate within a year was 18.6%. In a multivariate analysis, S-DAT score was a significant predictor of relapse (odds ratio [OR] 1.65, P = .000). Other independent predictors of relapse were post-LT alcohol treatment (OR 7.11, P = .02), smoking history (OR 0.15, P = .03), and marital status (OR 60.28, P = .000). The area under the receiver operative curves (AUROC) for the S-DAT score to predict alcohol relapse within 1 year after LT was 0.77.

The sensitivity of the S-DAT for predicting relapse risk was 96.2%, and specificity was 40.4%; positive and negative predictive values were 26.9% and 97.9%, respectively.

The high sensitivity and negative predictive values of the S-DAT make it a useful screening tool for identifying patients at low risk of alcohol relapse after a liver transplant, Dr. Kothadia said in an interview. “Our score will guide risk-based interventions post-LT to reduce post-LT relapse and improve long-term outcomes.”

The findings included only data from a single center, which may limit generalizability, Dr. Kothadia said. The tool is not yet clinically available, he noted.

“We would like to perform external validation of our S-DAT score as it stresses the importance of these psychosocial variables,” and to confirm the findings in larger, multicenter, prospective clinical trials, he said.

The study received no outside funding. Dr. Kothadia indicated no relevant financial relationships.

A novel prognostic tool based on social determinants effectively predicted increased risk of alcohol use relapse in adults who underwent liver transplants for alcoholic liver disease, based on data from 140 individuals.

Alcohol relapse after liver transplant ranges from 4% to as high as 95% among patients with alcoholic liver disease (ALD) and better tools are needed to identify those at increased risk, Jiten P. Kothadia, MD, of the University of Tennessee Health Science Center, Memphis, said in a presentation given in October at the annual meeting of the American College of Gastroenterology.

Dr. Kothadia and colleagues evaluated the effectiveness of the Social Determinant Acuity Tool (S-DAT), which stratified patients in terms of successful post-liver transplant outcomes from excellent (S-DAT scores 0-6) to poor candidates (scores 35-40). The S-DAT categories included cognitive function, mental health, social support, coping skills, financial status, compliance, alcohol abuse, substance abuse, reliability, legal issues, understanding the transplant process, and desire for transplant.

The study population included 140 adults with alcoholic liver disease who underwent a liver transplant between January 2016 and November 2021 at a single center. Before surgery, all patients underwent a thorough psychosocial evaluation using the S-DAT. The mean age of the participants was 53.4 years, 107 were male, and 67.9% had abstained from alcohol for more than 6 months prior to transplant.

The primary outcome of post-liver transplant alcohol relapse was defined as any alcohol use regardless of the amount or frequency, based on patient interviews or blood or urine tests.

Overall, the rate of relapse was 23.6%; and the rate within a year was 18.6%. In a multivariate analysis, S-DAT score was a significant predictor of relapse (odds ratio [OR] 1.65, P = .000). Other independent predictors of relapse were post-LT alcohol treatment (OR 7.11, P = .02), smoking history (OR 0.15, P = .03), and marital status (OR 60.28, P = .000). The area under the receiver operative curves (AUROC) for the S-DAT score to predict alcohol relapse within 1 year after LT was 0.77.

The sensitivity of the S-DAT for predicting relapse risk was 96.2%, and specificity was 40.4%; positive and negative predictive values were 26.9% and 97.9%, respectively.

The high sensitivity and negative predictive values of the S-DAT make it a useful screening tool for identifying patients at low risk of alcohol relapse after a liver transplant, Dr. Kothadia said in an interview. “Our score will guide risk-based interventions post-LT to reduce post-LT relapse and improve long-term outcomes.”

The findings included only data from a single center, which may limit generalizability, Dr. Kothadia said. The tool is not yet clinically available, he noted.

“We would like to perform external validation of our S-DAT score as it stresses the importance of these psychosocial variables,” and to confirm the findings in larger, multicenter, prospective clinical trials, he said.

The study received no outside funding. Dr. Kothadia indicated no relevant financial relationships.

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Liver-resident T cells provide early protection against Listeria infection

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Mon, 11/13/2023 - 09:50

Liver-resident gamma delta T cells that produce interleukin(IL)-17 coordinate with hepatic macrophages to offer early protection against Listeria monocytogenes infection, according to investigators.

These finding suggest that gamma delta T17 cells could be a target for novel cell-based therapies against liver diseases, reported lead author Yanan Wang, PhD, of Shandong University, Jinan, China, and colleagues.

“Gamma delta T cells are located in mucosal tissues and other peripheral lymphoid tissues and are considered to act as the first line of defense within the immune system,” the investigators wrote in Cellular and Molecular Gastroenterology and Hepatology. “Several studies have reported that IL-17A produced by gamma delta T cells plays a critical role in host defense after Listeria monocytogenes [infection] in the liver. However, in those studies, the details of the phenotypes, dynamic changes, proliferation activity, and cytokine production of the responding gamma delta T cell populations in the overall process of hepatic infection are unclear, and how they accumulated into the infection sites has not been elucidated.”

To address this knowledge gap, Dr. Wang and colleagues conducted a series of experiments involving gamma delta T cells from murine liver samples.

First, using single-cell RNA-sequencing (scRNA-seq), the investigators identified six clusters of hepatic gamma delta T cells.

“[This first step] revealed the unique gene expression characteristics and indicated the possible important roles in immune responses of hepatic gamma delta T17 cells,” they noted.

Next, the investigators measured expression of CD44 and CD27 in liver gamma delta cells.

“Expression of CD44 and CD27 has been used to distinguish IL-17A–, interferon gamma–producing, and other subsets of gamma delta T cells in the thymus, lymph nodes, lungs, and other peripheral lymphoid tissues,” they wrote.

These efforts revealed three subsets of hepatic gamma delta T cells, of which CD44hiCD27– gamma delta T cells were most abundant. Further analysis revealed expression profiles consistent with liver residency.

The next phases of the study characterized the immune roles of hepatic gamma delta T cells.

A comparison of Listeria monocytogenes infection in wild-type versus T-cell antigen receptor knockout mice, for example, showed that knockout mice had significantly more weight loss than did wild-type mice, greater bacterial load in the liver, and shorter survival times.

“As expected, the proportion and absolute numbers of gamma delta T cells in the liver of wild-type mice increased at day 3 and reached a peak at day 7 after infection,” the investigators wrote. “These data suggested that hepatic gamma delta T cells proliferated after infection and contributed to Lm clearance.”

Parabiosis experiments showed that the increased number of CD44hiCD27– gamma delta T cells in the livers of Listeria monocytogenes-infected mice were due to migration and proliferation of liver-resident gamma delta T cells instead of circulating gamma delta T cells. A transwell assay revealed that Kupffer cells and monocyte-derived macrophages promoted migration of CD44hiCD27– gamma delta T cells upon infection.

“Our study provides additional insight into liver-resident lymphocytes and will aid in targeting such tissue-resident lymphocyte populations to promote local immune surveillance,” the investigators concluded.

The study was supported by grants from the National Natural Science Foundation of China and the Shandong Provincial Natural Science Foundation. The investigators disclosed no conflicts of interest.

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Liver-resident gamma delta T cells that produce interleukin(IL)-17 coordinate with hepatic macrophages to offer early protection against Listeria monocytogenes infection, according to investigators.

These finding suggest that gamma delta T17 cells could be a target for novel cell-based therapies against liver diseases, reported lead author Yanan Wang, PhD, of Shandong University, Jinan, China, and colleagues.

“Gamma delta T cells are located in mucosal tissues and other peripheral lymphoid tissues and are considered to act as the first line of defense within the immune system,” the investigators wrote in Cellular and Molecular Gastroenterology and Hepatology. “Several studies have reported that IL-17A produced by gamma delta T cells plays a critical role in host defense after Listeria monocytogenes [infection] in the liver. However, in those studies, the details of the phenotypes, dynamic changes, proliferation activity, and cytokine production of the responding gamma delta T cell populations in the overall process of hepatic infection are unclear, and how they accumulated into the infection sites has not been elucidated.”

To address this knowledge gap, Dr. Wang and colleagues conducted a series of experiments involving gamma delta T cells from murine liver samples.

First, using single-cell RNA-sequencing (scRNA-seq), the investigators identified six clusters of hepatic gamma delta T cells.

“[This first step] revealed the unique gene expression characteristics and indicated the possible important roles in immune responses of hepatic gamma delta T17 cells,” they noted.

Next, the investigators measured expression of CD44 and CD27 in liver gamma delta cells.

“Expression of CD44 and CD27 has been used to distinguish IL-17A–, interferon gamma–producing, and other subsets of gamma delta T cells in the thymus, lymph nodes, lungs, and other peripheral lymphoid tissues,” they wrote.

These efforts revealed three subsets of hepatic gamma delta T cells, of which CD44hiCD27– gamma delta T cells were most abundant. Further analysis revealed expression profiles consistent with liver residency.

The next phases of the study characterized the immune roles of hepatic gamma delta T cells.

A comparison of Listeria monocytogenes infection in wild-type versus T-cell antigen receptor knockout mice, for example, showed that knockout mice had significantly more weight loss than did wild-type mice, greater bacterial load in the liver, and shorter survival times.

“As expected, the proportion and absolute numbers of gamma delta T cells in the liver of wild-type mice increased at day 3 and reached a peak at day 7 after infection,” the investigators wrote. “These data suggested that hepatic gamma delta T cells proliferated after infection and contributed to Lm clearance.”

Parabiosis experiments showed that the increased number of CD44hiCD27– gamma delta T cells in the livers of Listeria monocytogenes-infected mice were due to migration and proliferation of liver-resident gamma delta T cells instead of circulating gamma delta T cells. A transwell assay revealed that Kupffer cells and monocyte-derived macrophages promoted migration of CD44hiCD27– gamma delta T cells upon infection.

“Our study provides additional insight into liver-resident lymphocytes and will aid in targeting such tissue-resident lymphocyte populations to promote local immune surveillance,” the investigators concluded.

The study was supported by grants from the National Natural Science Foundation of China and the Shandong Provincial Natural Science Foundation. The investigators disclosed no conflicts of interest.

Liver-resident gamma delta T cells that produce interleukin(IL)-17 coordinate with hepatic macrophages to offer early protection against Listeria monocytogenes infection, according to investigators.

These finding suggest that gamma delta T17 cells could be a target for novel cell-based therapies against liver diseases, reported lead author Yanan Wang, PhD, of Shandong University, Jinan, China, and colleagues.

“Gamma delta T cells are located in mucosal tissues and other peripheral lymphoid tissues and are considered to act as the first line of defense within the immune system,” the investigators wrote in Cellular and Molecular Gastroenterology and Hepatology. “Several studies have reported that IL-17A produced by gamma delta T cells plays a critical role in host defense after Listeria monocytogenes [infection] in the liver. However, in those studies, the details of the phenotypes, dynamic changes, proliferation activity, and cytokine production of the responding gamma delta T cell populations in the overall process of hepatic infection are unclear, and how they accumulated into the infection sites has not been elucidated.”

To address this knowledge gap, Dr. Wang and colleagues conducted a series of experiments involving gamma delta T cells from murine liver samples.

First, using single-cell RNA-sequencing (scRNA-seq), the investigators identified six clusters of hepatic gamma delta T cells.

“[This first step] revealed the unique gene expression characteristics and indicated the possible important roles in immune responses of hepatic gamma delta T17 cells,” they noted.

Next, the investigators measured expression of CD44 and CD27 in liver gamma delta cells.

“Expression of CD44 and CD27 has been used to distinguish IL-17A–, interferon gamma–producing, and other subsets of gamma delta T cells in the thymus, lymph nodes, lungs, and other peripheral lymphoid tissues,” they wrote.

These efforts revealed three subsets of hepatic gamma delta T cells, of which CD44hiCD27– gamma delta T cells were most abundant. Further analysis revealed expression profiles consistent with liver residency.

The next phases of the study characterized the immune roles of hepatic gamma delta T cells.

A comparison of Listeria monocytogenes infection in wild-type versus T-cell antigen receptor knockout mice, for example, showed that knockout mice had significantly more weight loss than did wild-type mice, greater bacterial load in the liver, and shorter survival times.

“As expected, the proportion and absolute numbers of gamma delta T cells in the liver of wild-type mice increased at day 3 and reached a peak at day 7 after infection,” the investigators wrote. “These data suggested that hepatic gamma delta T cells proliferated after infection and contributed to Lm clearance.”

Parabiosis experiments showed that the increased number of CD44hiCD27– gamma delta T cells in the livers of Listeria monocytogenes-infected mice were due to migration and proliferation of liver-resident gamma delta T cells instead of circulating gamma delta T cells. A transwell assay revealed that Kupffer cells and monocyte-derived macrophages promoted migration of CD44hiCD27– gamma delta T cells upon infection.

“Our study provides additional insight into liver-resident lymphocytes and will aid in targeting such tissue-resident lymphocyte populations to promote local immune surveillance,” the investigators concluded.

The study was supported by grants from the National Natural Science Foundation of China and the Shandong Provincial Natural Science Foundation. The investigators disclosed no conflicts of interest.

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Nano drug delivery could overcome toxicity in HCC to enable safer, more effective therapy

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Wed, 10/11/2023 - 18:14

Employing a targeted nano drug delivery system for patients with hepatocellular carcinoma (HCC) could overcome issues with liver toxicity, leading to safer treatment and better outcomes, according to a recent review.

Nanomedicines homing in on the Wnt/beta-catenin signaling pathway could be particularly impactful, Mamatha Bhat, MD, PhD, a hepatologist and clinician-scientist at Toronto General Hospital Research Institute, and colleagues reported, as this is one of the most up-regulated pathways in HCC.

To date, however, agents addressing this pathway have been hindered by off-target toxicity, suggesting that more work is needed to develop the right payload for nanoparticle delivery, the investigators wrote in Gastro Hep Advances.

“Although nanotherapeutics offers an unmatched improvement in drug delivery, due to the limited impact and treatment-resistance demonstrated by the current systemic therapies, there is currently no approved nanomedicine for the treatment of HCC,” the investigators wrote. “Therefore, it is of utmost importance to dig deeper into understanding the signaling pathways that govern hepatocarcinogenesis and identify novel targets that can be used to develop more specific and targeted nanotherapies.”

Their review focused on the Wnt/beta-catenin signaling pathway, but first, Dr. Bhat and colleagues discussed the characteristics of inorganic versus lipid nanoparticles, as these differences can determine liver uptake.

Inorganic nanoparticles have a high surface-to-volume ratio, leading to increased surface charges that enhance cellular uptake. However, they are prone to oxidation, requiring surface modifications or short circulation times to prevent degradation. These nanoparticles are limited in delivering chemotherapeutic drugs and peptides, and are not suitable for encapsulating nucleic acids.

In contrast, lipid nanoparticles are preferred for targeted delivery in HCC, according to the investigators. They have a natural affinity for apolipoprotein E (apo E), resembling lipoproteins, which aids in specific liver cell targeting. When lipid nanoparticles enter the bloodstream, they interact with apo E–rich lipoproteins like HDL cholesterol and LDL cholesterol, leading to formation of complexes recognized by LDL cholesterol receptors on liver cells. This triggers receptor-mediated endocytosis, internalizing apo E–lipid nanoparticle complexes into HCC cells.

The other major variable is the selected treatment target. Dr. Bhat and colleagues made the case for the Wnt/beta-catenin signaling pathway based on alterations found in approximately two-thirds of patients with HCC.

“Aberrant activation of this pathway and mutations in genes encoding key components are characteristic to hepatocarcinogenesis and promote tumor growth and dedifferentiation,” they wrote.

Although beta-catenin itself makes for an obvious molecular target, especially considering known associations with drug resistance, its flat structure lacks deep binding pockets that would be suitable for small-molecule inhibitors, and any available pockets may be altered by numerous posttranscriptional modifications. Instead, beta-catenin could be indirectly modulated by nanoparticle-mediated siRNA therapy, as this would allow for precise delivery of siRNA to cancer cells, minimizing off-target toxicity.

Alternative approaches could involve targeting proteasomal degradation of beta-catenin, transcriptional coactivators of beta-catenin, or different oncogenes in HCC, all of which are described in further detail in the review, along with promising preclinical findings.

“With ongoing advancements in nanotechnology, there is optimism that it will continue to play a vital role in overcoming the challenges associated with HCC management and contribute to further advancements in therapeutic outcomes for patients,” the authors concluded.

One coauthor disclosed external funding by a Mitacs Elevate postdoctoral fellowship in collaboration with Highland Therapeutics. The remaining authors disclosed no conflicts of interest.

Body

 

Hepatocellular Carcinoma (HCC) remains a major health problem associate with increasing prevalence and mortality rates worldwide. Around 50-60% of HCC patients are exposed to systemic therapies during their natural history. Atezolizumab plus bevacizumab (median OS: 19.2mo, ORR 30%), and durvalumab plus tremelimumab (median OS: 16.4mo, ORR: 20%) are considered first line treatment options for advanced HCC, and sorafenib or lenvatinib are recommended for patients with any contraindication for immune checkpoint inhibitors. These therapies are indicated for ‘all comers’ an no molecular markers /personalize medicine is currently available for this cancer. The lack of precision oncology relates to the fact that the most common mutations ( i.e TERT, TP53,CTNNB1) are unactionable targets. In this scenario, advances in precision oncology are an unmet medical need.

The Wnt/B-catenin signaling pathway is a master regulator of oncogenesis in HCC and defines one of the molecular subclasses characterized by CTNNB1 mutations (~25-30%) or AXIN1 mutations (~5-10%). Most of these tumors have an immune excluded/desert phenotype. Thus, targeting this pathway is expected to provide a primary antitumoral effect along with an immune-modulatory effect rescuing cases with an immune excluded phenotype.

In this review, the authors discuss the applicability of precision oncology in HCC targeting the WNT/B-catenin pathway by inhibiting the interaction with transcriptional coactivators of B-catenin such as CBP and TCF or by enhancing the proteasomal degradation of B-catenin, reducing pathway activation, with drugs like Tankyrase inhibitors and casein kinase 1a activators. These approaches are challenging due to its associated off-target toxicity and its complexity. To overcome these caveats, the author propose to utilization of nanotechnology to deliver Wnt inhibitors, an approach that currently requires further research to refine the most promising strategies and drugs suitable for clinical implementation.

Josep M. Llovet, MD, PhD, FAASLD, director, Mount Sinai Liver Cancer Program in New York, and head of translational research in the Liver Cancer Group, Liver Unit, IDIBAPS, Hospital Clínic Barcelona. Dr. Llovet receives research support from Bayer Pharmaceuticals, Eisai Inc, Bristol-Myers Squibb and Ipsen.

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Body

 

Hepatocellular Carcinoma (HCC) remains a major health problem associate with increasing prevalence and mortality rates worldwide. Around 50-60% of HCC patients are exposed to systemic therapies during their natural history. Atezolizumab plus bevacizumab (median OS: 19.2mo, ORR 30%), and durvalumab plus tremelimumab (median OS: 16.4mo, ORR: 20%) are considered first line treatment options for advanced HCC, and sorafenib or lenvatinib are recommended for patients with any contraindication for immune checkpoint inhibitors. These therapies are indicated for ‘all comers’ an no molecular markers /personalize medicine is currently available for this cancer. The lack of precision oncology relates to the fact that the most common mutations ( i.e TERT, TP53,CTNNB1) are unactionable targets. In this scenario, advances in precision oncology are an unmet medical need.

The Wnt/B-catenin signaling pathway is a master regulator of oncogenesis in HCC and defines one of the molecular subclasses characterized by CTNNB1 mutations (~25-30%) or AXIN1 mutations (~5-10%). Most of these tumors have an immune excluded/desert phenotype. Thus, targeting this pathway is expected to provide a primary antitumoral effect along with an immune-modulatory effect rescuing cases with an immune excluded phenotype.

In this review, the authors discuss the applicability of precision oncology in HCC targeting the WNT/B-catenin pathway by inhibiting the interaction with transcriptional coactivators of B-catenin such as CBP and TCF or by enhancing the proteasomal degradation of B-catenin, reducing pathway activation, with drugs like Tankyrase inhibitors and casein kinase 1a activators. These approaches are challenging due to its associated off-target toxicity and its complexity. To overcome these caveats, the author propose to utilization of nanotechnology to deliver Wnt inhibitors, an approach that currently requires further research to refine the most promising strategies and drugs suitable for clinical implementation.

Josep M. Llovet, MD, PhD, FAASLD, director, Mount Sinai Liver Cancer Program in New York, and head of translational research in the Liver Cancer Group, Liver Unit, IDIBAPS, Hospital Clínic Barcelona. Dr. Llovet receives research support from Bayer Pharmaceuticals, Eisai Inc, Bristol-Myers Squibb and Ipsen.

Body

 

Hepatocellular Carcinoma (HCC) remains a major health problem associate with increasing prevalence and mortality rates worldwide. Around 50-60% of HCC patients are exposed to systemic therapies during their natural history. Atezolizumab plus bevacizumab (median OS: 19.2mo, ORR 30%), and durvalumab plus tremelimumab (median OS: 16.4mo, ORR: 20%) are considered first line treatment options for advanced HCC, and sorafenib or lenvatinib are recommended for patients with any contraindication for immune checkpoint inhibitors. These therapies are indicated for ‘all comers’ an no molecular markers /personalize medicine is currently available for this cancer. The lack of precision oncology relates to the fact that the most common mutations ( i.e TERT, TP53,CTNNB1) are unactionable targets. In this scenario, advances in precision oncology are an unmet medical need.

The Wnt/B-catenin signaling pathway is a master regulator of oncogenesis in HCC and defines one of the molecular subclasses characterized by CTNNB1 mutations (~25-30%) or AXIN1 mutations (~5-10%). Most of these tumors have an immune excluded/desert phenotype. Thus, targeting this pathway is expected to provide a primary antitumoral effect along with an immune-modulatory effect rescuing cases with an immune excluded phenotype.

In this review, the authors discuss the applicability of precision oncology in HCC targeting the WNT/B-catenin pathway by inhibiting the interaction with transcriptional coactivators of B-catenin such as CBP and TCF or by enhancing the proteasomal degradation of B-catenin, reducing pathway activation, with drugs like Tankyrase inhibitors and casein kinase 1a activators. These approaches are challenging due to its associated off-target toxicity and its complexity. To overcome these caveats, the author propose to utilization of nanotechnology to deliver Wnt inhibitors, an approach that currently requires further research to refine the most promising strategies and drugs suitable for clinical implementation.

Josep M. Llovet, MD, PhD, FAASLD, director, Mount Sinai Liver Cancer Program in New York, and head of translational research in the Liver Cancer Group, Liver Unit, IDIBAPS, Hospital Clínic Barcelona. Dr. Llovet receives research support from Bayer Pharmaceuticals, Eisai Inc, Bristol-Myers Squibb and Ipsen.

Employing a targeted nano drug delivery system for patients with hepatocellular carcinoma (HCC) could overcome issues with liver toxicity, leading to safer treatment and better outcomes, according to a recent review.

Nanomedicines homing in on the Wnt/beta-catenin signaling pathway could be particularly impactful, Mamatha Bhat, MD, PhD, a hepatologist and clinician-scientist at Toronto General Hospital Research Institute, and colleagues reported, as this is one of the most up-regulated pathways in HCC.

To date, however, agents addressing this pathway have been hindered by off-target toxicity, suggesting that more work is needed to develop the right payload for nanoparticle delivery, the investigators wrote in Gastro Hep Advances.

“Although nanotherapeutics offers an unmatched improvement in drug delivery, due to the limited impact and treatment-resistance demonstrated by the current systemic therapies, there is currently no approved nanomedicine for the treatment of HCC,” the investigators wrote. “Therefore, it is of utmost importance to dig deeper into understanding the signaling pathways that govern hepatocarcinogenesis and identify novel targets that can be used to develop more specific and targeted nanotherapies.”

Their review focused on the Wnt/beta-catenin signaling pathway, but first, Dr. Bhat and colleagues discussed the characteristics of inorganic versus lipid nanoparticles, as these differences can determine liver uptake.

Inorganic nanoparticles have a high surface-to-volume ratio, leading to increased surface charges that enhance cellular uptake. However, they are prone to oxidation, requiring surface modifications or short circulation times to prevent degradation. These nanoparticles are limited in delivering chemotherapeutic drugs and peptides, and are not suitable for encapsulating nucleic acids.

In contrast, lipid nanoparticles are preferred for targeted delivery in HCC, according to the investigators. They have a natural affinity for apolipoprotein E (apo E), resembling lipoproteins, which aids in specific liver cell targeting. When lipid nanoparticles enter the bloodstream, they interact with apo E–rich lipoproteins like HDL cholesterol and LDL cholesterol, leading to formation of complexes recognized by LDL cholesterol receptors on liver cells. This triggers receptor-mediated endocytosis, internalizing apo E–lipid nanoparticle complexes into HCC cells.

The other major variable is the selected treatment target. Dr. Bhat and colleagues made the case for the Wnt/beta-catenin signaling pathway based on alterations found in approximately two-thirds of patients with HCC.

“Aberrant activation of this pathway and mutations in genes encoding key components are characteristic to hepatocarcinogenesis and promote tumor growth and dedifferentiation,” they wrote.

Although beta-catenin itself makes for an obvious molecular target, especially considering known associations with drug resistance, its flat structure lacks deep binding pockets that would be suitable for small-molecule inhibitors, and any available pockets may be altered by numerous posttranscriptional modifications. Instead, beta-catenin could be indirectly modulated by nanoparticle-mediated siRNA therapy, as this would allow for precise delivery of siRNA to cancer cells, minimizing off-target toxicity.

Alternative approaches could involve targeting proteasomal degradation of beta-catenin, transcriptional coactivators of beta-catenin, or different oncogenes in HCC, all of which are described in further detail in the review, along with promising preclinical findings.

“With ongoing advancements in nanotechnology, there is optimism that it will continue to play a vital role in overcoming the challenges associated with HCC management and contribute to further advancements in therapeutic outcomes for patients,” the authors concluded.

One coauthor disclosed external funding by a Mitacs Elevate postdoctoral fellowship in collaboration with Highland Therapeutics. The remaining authors disclosed no conflicts of interest.

Employing a targeted nano drug delivery system for patients with hepatocellular carcinoma (HCC) could overcome issues with liver toxicity, leading to safer treatment and better outcomes, according to a recent review.

Nanomedicines homing in on the Wnt/beta-catenin signaling pathway could be particularly impactful, Mamatha Bhat, MD, PhD, a hepatologist and clinician-scientist at Toronto General Hospital Research Institute, and colleagues reported, as this is one of the most up-regulated pathways in HCC.

To date, however, agents addressing this pathway have been hindered by off-target toxicity, suggesting that more work is needed to develop the right payload for nanoparticle delivery, the investigators wrote in Gastro Hep Advances.

“Although nanotherapeutics offers an unmatched improvement in drug delivery, due to the limited impact and treatment-resistance demonstrated by the current systemic therapies, there is currently no approved nanomedicine for the treatment of HCC,” the investigators wrote. “Therefore, it is of utmost importance to dig deeper into understanding the signaling pathways that govern hepatocarcinogenesis and identify novel targets that can be used to develop more specific and targeted nanotherapies.”

Their review focused on the Wnt/beta-catenin signaling pathway, but first, Dr. Bhat and colleagues discussed the characteristics of inorganic versus lipid nanoparticles, as these differences can determine liver uptake.

Inorganic nanoparticles have a high surface-to-volume ratio, leading to increased surface charges that enhance cellular uptake. However, they are prone to oxidation, requiring surface modifications or short circulation times to prevent degradation. These nanoparticles are limited in delivering chemotherapeutic drugs and peptides, and are not suitable for encapsulating nucleic acids.

In contrast, lipid nanoparticles are preferred for targeted delivery in HCC, according to the investigators. They have a natural affinity for apolipoprotein E (apo E), resembling lipoproteins, which aids in specific liver cell targeting. When lipid nanoparticles enter the bloodstream, they interact with apo E–rich lipoproteins like HDL cholesterol and LDL cholesterol, leading to formation of complexes recognized by LDL cholesterol receptors on liver cells. This triggers receptor-mediated endocytosis, internalizing apo E–lipid nanoparticle complexes into HCC cells.

The other major variable is the selected treatment target. Dr. Bhat and colleagues made the case for the Wnt/beta-catenin signaling pathway based on alterations found in approximately two-thirds of patients with HCC.

“Aberrant activation of this pathway and mutations in genes encoding key components are characteristic to hepatocarcinogenesis and promote tumor growth and dedifferentiation,” they wrote.

Although beta-catenin itself makes for an obvious molecular target, especially considering known associations with drug resistance, its flat structure lacks deep binding pockets that would be suitable for small-molecule inhibitors, and any available pockets may be altered by numerous posttranscriptional modifications. Instead, beta-catenin could be indirectly modulated by nanoparticle-mediated siRNA therapy, as this would allow for precise delivery of siRNA to cancer cells, minimizing off-target toxicity.

Alternative approaches could involve targeting proteasomal degradation of beta-catenin, transcriptional coactivators of beta-catenin, or different oncogenes in HCC, all of which are described in further detail in the review, along with promising preclinical findings.

“With ongoing advancements in nanotechnology, there is optimism that it will continue to play a vital role in overcoming the challenges associated with HCC management and contribute to further advancements in therapeutic outcomes for patients,” the authors concluded.

One coauthor disclosed external funding by a Mitacs Elevate postdoctoral fellowship in collaboration with Highland Therapeutics. The remaining authors disclosed no conflicts of interest.

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Neutrophils may offer therapeutic target for Wilson’s disease

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Mon, 10/30/2023 - 17:12

Modulating N2-neutrophil activity could offer a new therapeutic approach to Wilson’s disease, according to a preclinical study.

Inhibiting neutrophil function via transforming growth factor (TGF-beta 1) inhibition or methylation inhibition reduced parenchymal liver fibrosis and injury while improving liver function in a mouse model of Wilson’s disease, shows new research published in Cellular and Molecular Gastroenterology and Hepatology.

Also called progressive hepatolenticular degeneration, Wilson’s disease is an inherited nervous system disorder that can occur as a result of severe liver disease. It is caused by variants in the ATP7B gene which can lead to abnormalities in copper metabolism that lead to accumulation of the heavy metal in the liver and brain, resulting in damage to both organs. Approximately 60% of patients with Wilson’s disease present with hepatic syndromes, and of those 50%-60% go on to develop liver cirrhosis.

Current treatments aim to address metal deposition, but this approach is poorly tolerated by many patients, wrote investigators who were led by Junping Shi, MD, PhD, of the Institute of Hepatology and Metabolic Diseases, The Affiliated Hospital of Hangzhou Normal University, China.

“Drug interventions (such as copper chelators and zinc salts) reduce pathologic copper deposition, but side effects can be observed in up to 40% of patients during treatment and even after years of treatment, particularly nephropathy, autoimmune conditions, and skin changes,” the investigators wrote. “Liver transplantation is an effective treatment for Wilson’s disease, particularly for patients with end-stage liver disease, but donor shortages and lifelong immunosuppression limit its use. Therefore, alternative treatments with higher specificity in Wilson’s disease patients are urgently needed.”

The present study explored the underlying metabolic abnormalities in Wilson’s disease that result in liver injury and fibrosis, and related therapeutic approaches. Based on previous studies that have shown a relationship between persistent neutrophil infiltration and chronic tissue inflammation and damage, the investigators sought to explore the role of neutrophils in Wilson’s disease, with a focus on the N2 subtype.

First, they analyzed neutrophil populations in the livers of Atp7b–/– mice and atp7b–/– zebrafish, both of which are established animal models of Wilson’s disease. Compared with the wild-type comparison animals, the livers of disease model animals showed increased neutrophil infiltration, in terms of both count and density.

In one of several related experiments, administering a neutrophil agonist in the presence of copper led to significantly greater neutrophil infiltration in mutant versus wild-type fish, as well as greater increases in lipid droplets and disorganized tissue structure, which serve as markers of disease activity.

“Collectively, these data suggested that neutrophils infiltrated the liver and accelerated liver defects in Wilson’s disease,” the investigators wrote.

Additional experiments with the mouse model showed that pharmacologic ablation of N2 neutrophils via two approaches led to reduced liver fibrosis, offering a glimpse at therapeutic potential.

These findings were further supported by experiments involving a cellular model of Wilson’s disease with isolated bone marrow neutrophils. These analyses revealed the role of the TGF1–DNMT3A/STAT3 signaling axis in neutrophil polarization, and resultant liver disease progression, in Wilson’s disease.

“Neutrophil heterogeneity shows therapeutic potential, and pharmacologic modulation of N2-neutrophil activity should be explored as an alternative therapeutic to improve liver function in Wilson’s disease,” the investigators concluded, noting that TGF-beta 1, DNMT3A, or STAT3 could all serve as rational therapeutic targets.

Beyond Wilson’s disease, the findings may offer broader value for understanding the mechanisms driving other neutrophil-related diseases, as well as possible therapeutic approaches for those conditions, the authors added.

The authors disclosed no conflicts of interest.

Body

The treatment of Wilson disease relies on use of chelators (D-pencilliamine; trientine) that promote urinary copper excretion and zinc, which blocks intestinal absorption. 
These drugs, which must be taken continuously, are effective but are associated with significant side effects. Another chelator, bis-choline-­tetrathiomolybdate (TTM), promotes biliary, rather than urinary copper excretion.
TTM improved neurological function in clinical trials; however, dose-dependent transaminase elevations were noted. 
Thus, there is a need to identify new therapeutic approaches to reduce impact of copper toxicity in hepatocytes.
In the current issue of CMGH, Mi and colleagues utilize zebrafish and mouse models of Wilson disease to generate novel insights into the pathogenesis and molecular basis of liver injury and fibrosis caused by ATP7B mutations. In the zebrafish model, they first showed that fluorescently-labeled neutrophils accumulate in the livers of live, mutant animals, which are transparent, and thus, uniquely suited to these studies. Gene expression analyses showed that the liver neutrophils are metabolically active and sensitize hepatocytes to copper-induced injury, thus providing a therapeutic rational for neutrophil inhibition. Next, the authors confirmed these findings in the mouse model, showing specifically that the N2-neutrophil subtype predominated and correlated with the degree of liver injury. Subsequent gene expression studies in the mouse, combined with in vitro analysis of bone marrow-derived neutrophils, identified a molecular signaling pathway originating in hepatocytes that triggered N2 differentiation. This pathway, which was previously shown to drive N2 differentiation in cancer models, involves TGF-beta induced methylation (and hence repression) of a gene (SOCS3) that itself, blocks expression of STAT3, a gene that drives N2 differentiation. Importantly, liver injury and fibrosis were reduced in the mouse model by drugs that inhibit TGF-beta or DNA methylation, and hence N2 differentiation, or by directly blocking the activity of N2 neutrophils.

In summary, this new study provides novel insights into not only into the pathogenesis and potential treatment of Wilson disease, but also demonstrates how signaling pathways, such as the one involving TGFbeta-SOCS3-STAT3, are reiteratively used in a variety of pathologic contexts. Going forward, it will be important to determine whether this pharmacologically modifiable signaling pathway is activated in Wilson disease patients, and whether it impacts the pathogenesis of more common liver disorders.

Michael Pack, M.D., is professor of medicine at Perelman School of Medicine, University of Pennsylvania. He has no conflicts.

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The treatment of Wilson disease relies on use of chelators (D-pencilliamine; trientine) that promote urinary copper excretion and zinc, which blocks intestinal absorption. 
These drugs, which must be taken continuously, are effective but are associated with significant side effects. Another chelator, bis-choline-­tetrathiomolybdate (TTM), promotes biliary, rather than urinary copper excretion.
TTM improved neurological function in clinical trials; however, dose-dependent transaminase elevations were noted. 
Thus, there is a need to identify new therapeutic approaches to reduce impact of copper toxicity in hepatocytes.
In the current issue of CMGH, Mi and colleagues utilize zebrafish and mouse models of Wilson disease to generate novel insights into the pathogenesis and molecular basis of liver injury and fibrosis caused by ATP7B mutations. In the zebrafish model, they first showed that fluorescently-labeled neutrophils accumulate in the livers of live, mutant animals, which are transparent, and thus, uniquely suited to these studies. Gene expression analyses showed that the liver neutrophils are metabolically active and sensitize hepatocytes to copper-induced injury, thus providing a therapeutic rational for neutrophil inhibition. Next, the authors confirmed these findings in the mouse model, showing specifically that the N2-neutrophil subtype predominated and correlated with the degree of liver injury. Subsequent gene expression studies in the mouse, combined with in vitro analysis of bone marrow-derived neutrophils, identified a molecular signaling pathway originating in hepatocytes that triggered N2 differentiation. This pathway, which was previously shown to drive N2 differentiation in cancer models, involves TGF-beta induced methylation (and hence repression) of a gene (SOCS3) that itself, blocks expression of STAT3, a gene that drives N2 differentiation. Importantly, liver injury and fibrosis were reduced in the mouse model by drugs that inhibit TGF-beta or DNA methylation, and hence N2 differentiation, or by directly blocking the activity of N2 neutrophils.

In summary, this new study provides novel insights into not only into the pathogenesis and potential treatment of Wilson disease, but also demonstrates how signaling pathways, such as the one involving TGFbeta-SOCS3-STAT3, are reiteratively used in a variety of pathologic contexts. Going forward, it will be important to determine whether this pharmacologically modifiable signaling pathway is activated in Wilson disease patients, and whether it impacts the pathogenesis of more common liver disorders.

Michael Pack, M.D., is professor of medicine at Perelman School of Medicine, University of Pennsylvania. He has no conflicts.

Body

The treatment of Wilson disease relies on use of chelators (D-pencilliamine; trientine) that promote urinary copper excretion and zinc, which blocks intestinal absorption. 
These drugs, which must be taken continuously, are effective but are associated with significant side effects. Another chelator, bis-choline-­tetrathiomolybdate (TTM), promotes biliary, rather than urinary copper excretion.
TTM improved neurological function in clinical trials; however, dose-dependent transaminase elevations were noted. 
Thus, there is a need to identify new therapeutic approaches to reduce impact of copper toxicity in hepatocytes.
In the current issue of CMGH, Mi and colleagues utilize zebrafish and mouse models of Wilson disease to generate novel insights into the pathogenesis and molecular basis of liver injury and fibrosis caused by ATP7B mutations. In the zebrafish model, they first showed that fluorescently-labeled neutrophils accumulate in the livers of live, mutant animals, which are transparent, and thus, uniquely suited to these studies. Gene expression analyses showed that the liver neutrophils are metabolically active and sensitize hepatocytes to copper-induced injury, thus providing a therapeutic rational for neutrophil inhibition. Next, the authors confirmed these findings in the mouse model, showing specifically that the N2-neutrophil subtype predominated and correlated with the degree of liver injury. Subsequent gene expression studies in the mouse, combined with in vitro analysis of bone marrow-derived neutrophils, identified a molecular signaling pathway originating in hepatocytes that triggered N2 differentiation. This pathway, which was previously shown to drive N2 differentiation in cancer models, involves TGF-beta induced methylation (and hence repression) of a gene (SOCS3) that itself, blocks expression of STAT3, a gene that drives N2 differentiation. Importantly, liver injury and fibrosis were reduced in the mouse model by drugs that inhibit TGF-beta or DNA methylation, and hence N2 differentiation, or by directly blocking the activity of N2 neutrophils.

In summary, this new study provides novel insights into not only into the pathogenesis and potential treatment of Wilson disease, but also demonstrates how signaling pathways, such as the one involving TGFbeta-SOCS3-STAT3, are reiteratively used in a variety of pathologic contexts. Going forward, it will be important to determine whether this pharmacologically modifiable signaling pathway is activated in Wilson disease patients, and whether it impacts the pathogenesis of more common liver disorders.

Michael Pack, M.D., is professor of medicine at Perelman School of Medicine, University of Pennsylvania. He has no conflicts.

Modulating N2-neutrophil activity could offer a new therapeutic approach to Wilson’s disease, according to a preclinical study.

Inhibiting neutrophil function via transforming growth factor (TGF-beta 1) inhibition or methylation inhibition reduced parenchymal liver fibrosis and injury while improving liver function in a mouse model of Wilson’s disease, shows new research published in Cellular and Molecular Gastroenterology and Hepatology.

Also called progressive hepatolenticular degeneration, Wilson’s disease is an inherited nervous system disorder that can occur as a result of severe liver disease. It is caused by variants in the ATP7B gene which can lead to abnormalities in copper metabolism that lead to accumulation of the heavy metal in the liver and brain, resulting in damage to both organs. Approximately 60% of patients with Wilson’s disease present with hepatic syndromes, and of those 50%-60% go on to develop liver cirrhosis.

Current treatments aim to address metal deposition, but this approach is poorly tolerated by many patients, wrote investigators who were led by Junping Shi, MD, PhD, of the Institute of Hepatology and Metabolic Diseases, The Affiliated Hospital of Hangzhou Normal University, China.

“Drug interventions (such as copper chelators and zinc salts) reduce pathologic copper deposition, but side effects can be observed in up to 40% of patients during treatment and even after years of treatment, particularly nephropathy, autoimmune conditions, and skin changes,” the investigators wrote. “Liver transplantation is an effective treatment for Wilson’s disease, particularly for patients with end-stage liver disease, but donor shortages and lifelong immunosuppression limit its use. Therefore, alternative treatments with higher specificity in Wilson’s disease patients are urgently needed.”

The present study explored the underlying metabolic abnormalities in Wilson’s disease that result in liver injury and fibrosis, and related therapeutic approaches. Based on previous studies that have shown a relationship between persistent neutrophil infiltration and chronic tissue inflammation and damage, the investigators sought to explore the role of neutrophils in Wilson’s disease, with a focus on the N2 subtype.

First, they analyzed neutrophil populations in the livers of Atp7b–/– mice and atp7b–/– zebrafish, both of which are established animal models of Wilson’s disease. Compared with the wild-type comparison animals, the livers of disease model animals showed increased neutrophil infiltration, in terms of both count and density.

In one of several related experiments, administering a neutrophil agonist in the presence of copper led to significantly greater neutrophil infiltration in mutant versus wild-type fish, as well as greater increases in lipid droplets and disorganized tissue structure, which serve as markers of disease activity.

“Collectively, these data suggested that neutrophils infiltrated the liver and accelerated liver defects in Wilson’s disease,” the investigators wrote.

Additional experiments with the mouse model showed that pharmacologic ablation of N2 neutrophils via two approaches led to reduced liver fibrosis, offering a glimpse at therapeutic potential.

These findings were further supported by experiments involving a cellular model of Wilson’s disease with isolated bone marrow neutrophils. These analyses revealed the role of the TGF1–DNMT3A/STAT3 signaling axis in neutrophil polarization, and resultant liver disease progression, in Wilson’s disease.

“Neutrophil heterogeneity shows therapeutic potential, and pharmacologic modulation of N2-neutrophil activity should be explored as an alternative therapeutic to improve liver function in Wilson’s disease,” the investigators concluded, noting that TGF-beta 1, DNMT3A, or STAT3 could all serve as rational therapeutic targets.

Beyond Wilson’s disease, the findings may offer broader value for understanding the mechanisms driving other neutrophil-related diseases, as well as possible therapeutic approaches for those conditions, the authors added.

The authors disclosed no conflicts of interest.

Modulating N2-neutrophil activity could offer a new therapeutic approach to Wilson’s disease, according to a preclinical study.

Inhibiting neutrophil function via transforming growth factor (TGF-beta 1) inhibition or methylation inhibition reduced parenchymal liver fibrosis and injury while improving liver function in a mouse model of Wilson’s disease, shows new research published in Cellular and Molecular Gastroenterology and Hepatology.

Also called progressive hepatolenticular degeneration, Wilson’s disease is an inherited nervous system disorder that can occur as a result of severe liver disease. It is caused by variants in the ATP7B gene which can lead to abnormalities in copper metabolism that lead to accumulation of the heavy metal in the liver and brain, resulting in damage to both organs. Approximately 60% of patients with Wilson’s disease present with hepatic syndromes, and of those 50%-60% go on to develop liver cirrhosis.

Current treatments aim to address metal deposition, but this approach is poorly tolerated by many patients, wrote investigators who were led by Junping Shi, MD, PhD, of the Institute of Hepatology and Metabolic Diseases, The Affiliated Hospital of Hangzhou Normal University, China.

“Drug interventions (such as copper chelators and zinc salts) reduce pathologic copper deposition, but side effects can be observed in up to 40% of patients during treatment and even after years of treatment, particularly nephropathy, autoimmune conditions, and skin changes,” the investigators wrote. “Liver transplantation is an effective treatment for Wilson’s disease, particularly for patients with end-stage liver disease, but donor shortages and lifelong immunosuppression limit its use. Therefore, alternative treatments with higher specificity in Wilson’s disease patients are urgently needed.”

The present study explored the underlying metabolic abnormalities in Wilson’s disease that result in liver injury and fibrosis, and related therapeutic approaches. Based on previous studies that have shown a relationship between persistent neutrophil infiltration and chronic tissue inflammation and damage, the investigators sought to explore the role of neutrophils in Wilson’s disease, with a focus on the N2 subtype.

First, they analyzed neutrophil populations in the livers of Atp7b–/– mice and atp7b–/– zebrafish, both of which are established animal models of Wilson’s disease. Compared with the wild-type comparison animals, the livers of disease model animals showed increased neutrophil infiltration, in terms of both count and density.

In one of several related experiments, administering a neutrophil agonist in the presence of copper led to significantly greater neutrophil infiltration in mutant versus wild-type fish, as well as greater increases in lipid droplets and disorganized tissue structure, which serve as markers of disease activity.

“Collectively, these data suggested that neutrophils infiltrated the liver and accelerated liver defects in Wilson’s disease,” the investigators wrote.

Additional experiments with the mouse model showed that pharmacologic ablation of N2 neutrophils via two approaches led to reduced liver fibrosis, offering a glimpse at therapeutic potential.

These findings were further supported by experiments involving a cellular model of Wilson’s disease with isolated bone marrow neutrophils. These analyses revealed the role of the TGF1–DNMT3A/STAT3 signaling axis in neutrophil polarization, and resultant liver disease progression, in Wilson’s disease.

“Neutrophil heterogeneity shows therapeutic potential, and pharmacologic modulation of N2-neutrophil activity should be explored as an alternative therapeutic to improve liver function in Wilson’s disease,” the investigators concluded, noting that TGF-beta 1, DNMT3A, or STAT3 could all serve as rational therapeutic targets.

Beyond Wilson’s disease, the findings may offer broader value for understanding the mechanisms driving other neutrophil-related diseases, as well as possible therapeutic approaches for those conditions, the authors added.

The authors disclosed no conflicts of interest.

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MASLD/MASH and Weight Loss

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References
  1. Younossi ZM et al. Gastroenterology. 2021;160(3):912-918. doi:10.1053/j.astro.2020.11.051
  2. Cusi K et al. Endocr Pract. 2022;28(5):528-562. doi:10.1016/j.eprac.2022.03.010
  3. Rinella ME et al. Hepatology. 2023;77(5):1797-1835. doi:10.1097/HEP.0000000000000323
  4. World obesity atlas 2023. World Obesity Day. Published March 2023. Accessed July 23, 2023. https://www.worldobesityday.org/assets/downloads/World_Obesity_Atlas_2023_Report.pdf
  5. Le MH et al. Clin Mol Hepatol. 2022;28(4):841-850. doi:10.3350/cmh.2022.0239
  6. Vilar-Gomez E et al. Gastroenterology. 2015;149(2):367-78.e5. doi:10.1053/j.gastro.2015.04.005
  7. Koutoukidis DA et al. Metabolism. 2021;115:154455. doi:10.1016/j.metabol.2020.154455
  8. Ma J et al. Gastroenterology. 2018;155(1):107-117. doi:10.1053/j.gastro.2018.03.038
  9. Ahern AL et al. Lancet. 2017;389(10085):2214-2225. doi:10.1016/S0140-6736(17)30647-5
  10. Newsome PN et al; NN9931-4296 Investigators. N Engl J Med. 2021;384(12):1113-1124. doi:10.1056/NEJMoa2028395
  11. Armstrong MJ et al. Lancet. 2016;387(10019):679-690. doi:10.1016/S0140-6736(15)00803-X
  12. Gastaldelli A et al. Lancet Diabetes Endocrinol. 2022;10(6):393-406. doi:10.1016/S2213-8587(22)00070-5
  13. Kahl S et al. Diabetes Care. 2020;43(2):298-305. doi:10.2337/dc19-0641
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Assistant Professor of Medicine
Boston Medical Center
Boston University Chobanian & Avedisian School of Medicine
Boston, MA

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Boston University Chobanian & Avedisian School of Medicine
Boston, MA

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Boston, MA

References
  1. Younossi ZM et al. Gastroenterology. 2021;160(3):912-918. doi:10.1053/j.astro.2020.11.051
  2. Cusi K et al. Endocr Pract. 2022;28(5):528-562. doi:10.1016/j.eprac.2022.03.010
  3. Rinella ME et al. Hepatology. 2023;77(5):1797-1835. doi:10.1097/HEP.0000000000000323
  4. World obesity atlas 2023. World Obesity Day. Published March 2023. Accessed July 23, 2023. https://www.worldobesityday.org/assets/downloads/World_Obesity_Atlas_2023_Report.pdf
  5. Le MH et al. Clin Mol Hepatol. 2022;28(4):841-850. doi:10.3350/cmh.2022.0239
  6. Vilar-Gomez E et al. Gastroenterology. 2015;149(2):367-78.e5. doi:10.1053/j.gastro.2015.04.005
  7. Koutoukidis DA et al. Metabolism. 2021;115:154455. doi:10.1016/j.metabol.2020.154455
  8. Ma J et al. Gastroenterology. 2018;155(1):107-117. doi:10.1053/j.gastro.2018.03.038
  9. Ahern AL et al. Lancet. 2017;389(10085):2214-2225. doi:10.1016/S0140-6736(17)30647-5
  10. Newsome PN et al; NN9931-4296 Investigators. N Engl J Med. 2021;384(12):1113-1124. doi:10.1056/NEJMoa2028395
  11. Armstrong MJ et al. Lancet. 2016;387(10019):679-690. doi:10.1016/S0140-6736(15)00803-X
  12. Gastaldelli A et al. Lancet Diabetes Endocrinol. 2022;10(6):393-406. doi:10.1016/S2213-8587(22)00070-5
  13. Kahl S et al. Diabetes Care. 2020;43(2):298-305. doi:10.2337/dc19-0641
References
  1. Younossi ZM et al. Gastroenterology. 2021;160(3):912-918. doi:10.1053/j.astro.2020.11.051
  2. Cusi K et al. Endocr Pract. 2022;28(5):528-562. doi:10.1016/j.eprac.2022.03.010
  3. Rinella ME et al. Hepatology. 2023;77(5):1797-1835. doi:10.1097/HEP.0000000000000323
  4. World obesity atlas 2023. World Obesity Day. Published March 2023. Accessed July 23, 2023. https://www.worldobesityday.org/assets/downloads/World_Obesity_Atlas_2023_Report.pdf
  5. Le MH et al. Clin Mol Hepatol. 2022;28(4):841-850. doi:10.3350/cmh.2022.0239
  6. Vilar-Gomez E et al. Gastroenterology. 2015;149(2):367-78.e5. doi:10.1053/j.gastro.2015.04.005
  7. Koutoukidis DA et al. Metabolism. 2021;115:154455. doi:10.1016/j.metabol.2020.154455
  8. Ma J et al. Gastroenterology. 2018;155(1):107-117. doi:10.1053/j.gastro.2018.03.038
  9. Ahern AL et al. Lancet. 2017;389(10085):2214-2225. doi:10.1016/S0140-6736(17)30647-5
  10. Newsome PN et al; NN9931-4296 Investigators. N Engl J Med. 2021;384(12):1113-1124. doi:10.1056/NEJMoa2028395
  11. Armstrong MJ et al. Lancet. 2016;387(10019):679-690. doi:10.1016/S0140-6736(15)00803-X
  12. Gastaldelli A et al. Lancet Diabetes Endocrinol. 2022;10(6):393-406. doi:10.1016/S2213-8587(22)00070-5
  13. Kahl S et al. Diabetes Care. 2020;43(2):298-305. doi:10.2337/dc19-0641
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Metabolic dysfunction–associated steatotic liver disease (MASLD), formerly known as nonalcoholic fatty liver disease (NAFLD), affects about 20% to 30% of the world population. About 1 in 4 of these patients have metabolic dysfunction–associated steatohepatitis (MASH; formerly known as nonalcoholic steatohepatitis)—which is associated with significant morbidity and mortality.

Although they affect the liver, these conditions are associated with extrahepatic diseases such as obesity, type 2 diabetes mellitus, and cardiovascular disease, which contribute to poor outcomes. Evidence has shown that weight loss is a key intervention for reduction of steatosis, MASH, and fibrosis in both lean and obese patients with MASLD. Modifications recommended for weight loss include potential pharmacological, surgical, or endoscopic approaches (in cases of severe obesity) that can prevent progression of MASLD as well as improve diabetes and reduce cardiovascular events.1-3

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Gastroenterology Data Trends 2023

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GI&Hepatology News and the American Gastroenterological Association present the 2023 issue of Gastroenterology Data Trends, a special report on hot topics in gastroenterology told through original infographics and visual storytelling.

In this issue:

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GI&Hepatology News and the American Gastroenterological Association present the 2023 issue of Gastroenterology Data Trends, a special report on hot topics in gastroenterology told through original infographics and visual storytelling.

In this issue:

GI&Hepatology News and the American Gastroenterological Association present the 2023 issue of Gastroenterology Data Trends, a special report on hot topics in gastroenterology told through original infographics and visual storytelling.

In this issue:

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AGA CPU focuses on noninvasive tests in patients with NAFLD

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Noninvasive testing allows for routine risk stratification and long-term monitoring of patients with nonalcoholic fatty liver disease (NAFLD), offering a safer, more practical approach than biopsy, according to a recent Clinical Practice Update Expert Review by the American Gastroenterological Association.

The update, published online in Gastroenterology, includes eight best practice advice statements.

“The health care burden of longitudinal management of patients with NAFLD is significant. The emergence and utilization of noninvasive testing (NIT) in gastroenterology practices has the potential to significantly enhance the care of patients with NAFLD by improving detection of patients with advanced fibrosis who are at increased risk for cirrhosis, hepatic decompensation, and hepatocellular carcinoma (HCC), thereby facilitating timely clinical management,” wrote authors who were led by Julia J. Wattacheril, MD, MPH, of the Columbia University–New York Presbyterian Hospital nonalcoholic fatty liver disease program and center for liver disease and transplantation.

“In this Expert Review, we have provided clinicians with best practice advice for optimal utilization of NITs in patients with NAFLD,” the authors wrote.

Consensus best practice for implementing NITs in practice are scarce, giving rise to the present clinical practice update. The expert panel reviewed available evidence for these tests during longitudinal care of patients with advanced fibrosis as a means of predicting liver-related outcomes and informing treatment decisions.

The first statement encourages use of NITs for risk stratification during the diagnosis of NAFLD, typically in the form of clinical calculators like fibrosis 4 index (FIB-4), vibration controlled transient elastography (VCTE), shear wave

elastography (SWE), or magnetic resonance elastography (MRE), all of which have been validated in NAFLD.

“Ultrasound-based 3-dimensional elastography (Velacur) and iron-corrected T1 magnetic resonance imaging, although used less frequently, are emerging technologies,” the panelists noted.

Second, the update suggests that patients with a FIB-4 less than 1.3 are unlikely to have advanced hepatic fibrosis, based on this threshold’s strong negative predictive value (NPV).

Still, clinicians should remember that this FIB-4 threshold may be less reliable among patients younger than 35 years or older than 65 years, making it necessary to also consider other clinical measurements, according to the update. The third best practice advice encourages use of two or more NITs among patients with a FIB-4 score greater than 1.3.

The fourth piece of best practice advice suggests that clinicians follow manufacturer’s specifications when implementing NITs, as misuse may lead to “discordant results and adverse events.”

Fifth, to increase the positive predictive value (PPV) for detecting advanced fibrosis, NITs are best interpreted in the context of relevant clinical data, such as physical exam and endoscopy findings.

Next, the document encourages use of liver biopsy when NIT findings are discordant or indeterminate, conflict with findings from other test modalities, or if alternative, non–NAFLD etiologies are suspected.

The penultimate best practice advice suggests use of NITs for serial longitudinal disease monitoring, with signs of progression or regression used to guide clinical decisions.

“Additional evidence for longitudinal prediction of fibrosis regression and progression and response to intervention (lifestyle and pharmacologic) is needed in trials and real-world clinical practice,” Dr. Wattacheril and colleagues noted.

Finally, the clinical practice update advises surveillance of liver complications, such as hepatocellular carcinoma, among patients with NIT results that suggest advanced fibrosis (F3) or cirrhosis (F4).

This clinical practice update was commissioned by the AGA Institute. The investigators disclosed relationships with AstraZeneca, BMS, Novo Nordisk, and others.
 

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Noninvasive testing allows for routine risk stratification and long-term monitoring of patients with nonalcoholic fatty liver disease (NAFLD), offering a safer, more practical approach than biopsy, according to a recent Clinical Practice Update Expert Review by the American Gastroenterological Association.

The update, published online in Gastroenterology, includes eight best practice advice statements.

“The health care burden of longitudinal management of patients with NAFLD is significant. The emergence and utilization of noninvasive testing (NIT) in gastroenterology practices has the potential to significantly enhance the care of patients with NAFLD by improving detection of patients with advanced fibrosis who are at increased risk for cirrhosis, hepatic decompensation, and hepatocellular carcinoma (HCC), thereby facilitating timely clinical management,” wrote authors who were led by Julia J. Wattacheril, MD, MPH, of the Columbia University–New York Presbyterian Hospital nonalcoholic fatty liver disease program and center for liver disease and transplantation.

“In this Expert Review, we have provided clinicians with best practice advice for optimal utilization of NITs in patients with NAFLD,” the authors wrote.

Consensus best practice for implementing NITs in practice are scarce, giving rise to the present clinical practice update. The expert panel reviewed available evidence for these tests during longitudinal care of patients with advanced fibrosis as a means of predicting liver-related outcomes and informing treatment decisions.

The first statement encourages use of NITs for risk stratification during the diagnosis of NAFLD, typically in the form of clinical calculators like fibrosis 4 index (FIB-4), vibration controlled transient elastography (VCTE), shear wave

elastography (SWE), or magnetic resonance elastography (MRE), all of which have been validated in NAFLD.

“Ultrasound-based 3-dimensional elastography (Velacur) and iron-corrected T1 magnetic resonance imaging, although used less frequently, are emerging technologies,” the panelists noted.

Second, the update suggests that patients with a FIB-4 less than 1.3 are unlikely to have advanced hepatic fibrosis, based on this threshold’s strong negative predictive value (NPV).

Still, clinicians should remember that this FIB-4 threshold may be less reliable among patients younger than 35 years or older than 65 years, making it necessary to also consider other clinical measurements, according to the update. The third best practice advice encourages use of two or more NITs among patients with a FIB-4 score greater than 1.3.

The fourth piece of best practice advice suggests that clinicians follow manufacturer’s specifications when implementing NITs, as misuse may lead to “discordant results and adverse events.”

Fifth, to increase the positive predictive value (PPV) for detecting advanced fibrosis, NITs are best interpreted in the context of relevant clinical data, such as physical exam and endoscopy findings.

Next, the document encourages use of liver biopsy when NIT findings are discordant or indeterminate, conflict with findings from other test modalities, or if alternative, non–NAFLD etiologies are suspected.

The penultimate best practice advice suggests use of NITs for serial longitudinal disease monitoring, with signs of progression or regression used to guide clinical decisions.

“Additional evidence for longitudinal prediction of fibrosis regression and progression and response to intervention (lifestyle and pharmacologic) is needed in trials and real-world clinical practice,” Dr. Wattacheril and colleagues noted.

Finally, the clinical practice update advises surveillance of liver complications, such as hepatocellular carcinoma, among patients with NIT results that suggest advanced fibrosis (F3) or cirrhosis (F4).

This clinical practice update was commissioned by the AGA Institute. The investigators disclosed relationships with AstraZeneca, BMS, Novo Nordisk, and others.
 

Noninvasive testing allows for routine risk stratification and long-term monitoring of patients with nonalcoholic fatty liver disease (NAFLD), offering a safer, more practical approach than biopsy, according to a recent Clinical Practice Update Expert Review by the American Gastroenterological Association.

The update, published online in Gastroenterology, includes eight best practice advice statements.

“The health care burden of longitudinal management of patients with NAFLD is significant. The emergence and utilization of noninvasive testing (NIT) in gastroenterology practices has the potential to significantly enhance the care of patients with NAFLD by improving detection of patients with advanced fibrosis who are at increased risk for cirrhosis, hepatic decompensation, and hepatocellular carcinoma (HCC), thereby facilitating timely clinical management,” wrote authors who were led by Julia J. Wattacheril, MD, MPH, of the Columbia University–New York Presbyterian Hospital nonalcoholic fatty liver disease program and center for liver disease and transplantation.

“In this Expert Review, we have provided clinicians with best practice advice for optimal utilization of NITs in patients with NAFLD,” the authors wrote.

Consensus best practice for implementing NITs in practice are scarce, giving rise to the present clinical practice update. The expert panel reviewed available evidence for these tests during longitudinal care of patients with advanced fibrosis as a means of predicting liver-related outcomes and informing treatment decisions.

The first statement encourages use of NITs for risk stratification during the diagnosis of NAFLD, typically in the form of clinical calculators like fibrosis 4 index (FIB-4), vibration controlled transient elastography (VCTE), shear wave

elastography (SWE), or magnetic resonance elastography (MRE), all of which have been validated in NAFLD.

“Ultrasound-based 3-dimensional elastography (Velacur) and iron-corrected T1 magnetic resonance imaging, although used less frequently, are emerging technologies,” the panelists noted.

Second, the update suggests that patients with a FIB-4 less than 1.3 are unlikely to have advanced hepatic fibrosis, based on this threshold’s strong negative predictive value (NPV).

Still, clinicians should remember that this FIB-4 threshold may be less reliable among patients younger than 35 years or older than 65 years, making it necessary to also consider other clinical measurements, according to the update. The third best practice advice encourages use of two or more NITs among patients with a FIB-4 score greater than 1.3.

The fourth piece of best practice advice suggests that clinicians follow manufacturer’s specifications when implementing NITs, as misuse may lead to “discordant results and adverse events.”

Fifth, to increase the positive predictive value (PPV) for detecting advanced fibrosis, NITs are best interpreted in the context of relevant clinical data, such as physical exam and endoscopy findings.

Next, the document encourages use of liver biopsy when NIT findings are discordant or indeterminate, conflict with findings from other test modalities, or if alternative, non–NAFLD etiologies are suspected.

The penultimate best practice advice suggests use of NITs for serial longitudinal disease monitoring, with signs of progression or regression used to guide clinical decisions.

“Additional evidence for longitudinal prediction of fibrosis regression and progression and response to intervention (lifestyle and pharmacologic) is needed in trials and real-world clinical practice,” Dr. Wattacheril and colleagues noted.

Finally, the clinical practice update advises surveillance of liver complications, such as hepatocellular carcinoma, among patients with NIT results that suggest advanced fibrosis (F3) or cirrhosis (F4).

This clinical practice update was commissioned by the AGA Institute. The investigators disclosed relationships with AstraZeneca, BMS, Novo Nordisk, and others.
 

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Hepatic presentations of celiac disease

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Mon, 10/02/2023 - 10:24

Celiac disease can present with extraintestinal manifestations, including liver disease, with a spectrum ranging from elevated liver biochemistries to advanced cirrhosis. Liver biopsy findings may include variable degrees of steatosis, inflammation, and fibrosis.

Dr. Lawrence S. Friedman

In one case we have seen, the patient presented with unexplained ascites and features suggestive of Budd-Chiari syndrome. The serum ascites albumin gradient was 2.3 with a total protein of 0.8 g/dL, and albumin 0.5 g/dL, with an ascitic WBC count of 88/mm3.

Dr. Paul Martin


Echocardiography showed an ejection fraction of 80%. Transjugular liver biopsy revealed a normal hepatic venous pressure gradient but marked sinusoidal dilatation and congestion with hepatocyte atrophy and focal necrosis suggestive of vascular outlet obstruction (Figure 1).

Courtesy Gastro Hep Advances/AGA


Hepatic venography, however, showed no evidence of Budd-Chiari syndrome. When seen in consultation, pertinent observations included Irish ancestry, a history of occasional diarrhea, short stature, osteoporosis, and an atrophic spleen on computed tomography. An IgA transglutaminase antibody was positive, and a small-bowel biopsy confirmed celiac disease (Figure 2).

Courtesy Gastro Hep Advances/AGA


On a gluten-free diet, the patient’s symptoms resolved, with clinical and laboratory abnormalities returning to normal. She lived another 20 years before dying of primary pulmonary hypertension. Recognition of an unusual hepatic manifestation of celiac disease led to effective management.
 

Dr. Friedman is the Anton R. Fried, MD, Chair of the department of medicine at Newton-Wellesley Hospital in Newton, Mass., and assistant chief of medicine at Massachusetts General Hospital, and a professor of medicine at Harvard Medical School and Tufts University School of Medicine, all in Boston. Dr. Martin is chief of the division of digestive health and liver diseases at the Miller School of Medicine, University of Miami, where he is the Mandel Chair of Gastroenterology. The authors disclose no conflicts.

Previously published in Gastro Hep Advances. 2023. doi: 10.1016/j.gastha.2023.03.018.

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Celiac disease can present with extraintestinal manifestations, including liver disease, with a spectrum ranging from elevated liver biochemistries to advanced cirrhosis. Liver biopsy findings may include variable degrees of steatosis, inflammation, and fibrosis.

Dr. Lawrence S. Friedman

In one case we have seen, the patient presented with unexplained ascites and features suggestive of Budd-Chiari syndrome. The serum ascites albumin gradient was 2.3 with a total protein of 0.8 g/dL, and albumin 0.5 g/dL, with an ascitic WBC count of 88/mm3.

Dr. Paul Martin


Echocardiography showed an ejection fraction of 80%. Transjugular liver biopsy revealed a normal hepatic venous pressure gradient but marked sinusoidal dilatation and congestion with hepatocyte atrophy and focal necrosis suggestive of vascular outlet obstruction (Figure 1).

Courtesy Gastro Hep Advances/AGA


Hepatic venography, however, showed no evidence of Budd-Chiari syndrome. When seen in consultation, pertinent observations included Irish ancestry, a history of occasional diarrhea, short stature, osteoporosis, and an atrophic spleen on computed tomography. An IgA transglutaminase antibody was positive, and a small-bowel biopsy confirmed celiac disease (Figure 2).

Courtesy Gastro Hep Advances/AGA


On a gluten-free diet, the patient’s symptoms resolved, with clinical and laboratory abnormalities returning to normal. She lived another 20 years before dying of primary pulmonary hypertension. Recognition of an unusual hepatic manifestation of celiac disease led to effective management.
 

Dr. Friedman is the Anton R. Fried, MD, Chair of the department of medicine at Newton-Wellesley Hospital in Newton, Mass., and assistant chief of medicine at Massachusetts General Hospital, and a professor of medicine at Harvard Medical School and Tufts University School of Medicine, all in Boston. Dr. Martin is chief of the division of digestive health and liver diseases at the Miller School of Medicine, University of Miami, where he is the Mandel Chair of Gastroenterology. The authors disclose no conflicts.

Previously published in Gastro Hep Advances. 2023. doi: 10.1016/j.gastha.2023.03.018.

Celiac disease can present with extraintestinal manifestations, including liver disease, with a spectrum ranging from elevated liver biochemistries to advanced cirrhosis. Liver biopsy findings may include variable degrees of steatosis, inflammation, and fibrosis.

Dr. Lawrence S. Friedman

In one case we have seen, the patient presented with unexplained ascites and features suggestive of Budd-Chiari syndrome. The serum ascites albumin gradient was 2.3 with a total protein of 0.8 g/dL, and albumin 0.5 g/dL, with an ascitic WBC count of 88/mm3.

Dr. Paul Martin


Echocardiography showed an ejection fraction of 80%. Transjugular liver biopsy revealed a normal hepatic venous pressure gradient but marked sinusoidal dilatation and congestion with hepatocyte atrophy and focal necrosis suggestive of vascular outlet obstruction (Figure 1).

Courtesy Gastro Hep Advances/AGA


Hepatic venography, however, showed no evidence of Budd-Chiari syndrome. When seen in consultation, pertinent observations included Irish ancestry, a history of occasional diarrhea, short stature, osteoporosis, and an atrophic spleen on computed tomography. An IgA transglutaminase antibody was positive, and a small-bowel biopsy confirmed celiac disease (Figure 2).

Courtesy Gastro Hep Advances/AGA


On a gluten-free diet, the patient’s symptoms resolved, with clinical and laboratory abnormalities returning to normal. She lived another 20 years before dying of primary pulmonary hypertension. Recognition of an unusual hepatic manifestation of celiac disease led to effective management.
 

Dr. Friedman is the Anton R. Fried, MD, Chair of the department of medicine at Newton-Wellesley Hospital in Newton, Mass., and assistant chief of medicine at Massachusetts General Hospital, and a professor of medicine at Harvard Medical School and Tufts University School of Medicine, all in Boston. Dr. Martin is chief of the division of digestive health and liver diseases at the Miller School of Medicine, University of Miami, where he is the Mandel Chair of Gastroenterology. The authors disclose no conflicts.

Previously published in Gastro Hep Advances. 2023. doi: 10.1016/j.gastha.2023.03.018.

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