A medication used to reduce fluid retention can also safely be used to prevent a dangerous lung condition that affects newborns, particularly those born premature, according to a new study.

Furosemide (Lasix) – which can reduce excess fluid in the body caused by heart failure, liver disease, and kidney trouble – is commonly used off-label to prevent bronchopulmonary dysplasia (BPD), a disorder that causes irritation and poor development of lungs in premature infants. But until now, researchers have not studied its safety in this setting.

BPD often affects babies born more than 2 months early and can sometimes result in breathing difficulties into adolescence and young adulthood.

“There are so few drugs that have been tested for newborns, and there are very little data to help neonatologists decide if certain medications are safe and effective,” said Rachel Greenberg, MD, MHS, a neonatologist and member of the Duke Clinical Research Institute, Durham, N.C. “We found there was no greater risk of safety events for newborns given furosemide.”

Dr. Greenberg presented the findings at the 2022 Pediatric Academic Societies meeting in Denver.

For the 28-day randomized controlled trial, Dr. Greenberg and colleagues enrolled 80 preterm newborns, born at less than 29 weeks’ gestation, at 17 centers within the Eunice Kennedy Shriver National Institute of Child Health and Human Development Pediatric Trials Network. Of those, 61 received furosemide and 19 received a placebo.

Although babies given furosemide had more problems with electrolytes – an expected outcome from the use of diuretic medications – the researchers observed no greater risk for more serious issues, namely hearing loss or kidney stones, Dr. Greenberg told this news organization.

“The mechanism here is we know that extra fluid can damage the lungs and can cause you to have to use more respiratory support and more oxygen,” she said. “The thought from a physiological standpoint is using a diuretic can decrease fluid in the lungs and lead to improvements in lung outcomes.”

The researchers did not observe a reduction in BDP or death in babies who received furosemide, but Dr. Greenberg said the study was underpowered to detect such an effect.

“We were not powered to detect a difference in that outcome; the overall objective of this study was always to evaluate safety,” she said. “Of course, we wanted to capture variables that would measure effectiveness as well.

“Because this was a pragmatic trial, we did not limit the amount of fluids that the clinicians could give the participating infants. This could have impacted the effectiveness of furosemide. We would need a different design and larger study to truly determine effectiveness.” 

Dr. Greenberg said she hoped the new data will provide greater insight to neonatal providers and help bolster future, more large-scale trials using furosemide in premature infants.   

The drug has previously been associated with both kidney stones and ototoxicity, which occurs when medication causes a person to develop hearing or balance problems, said Nicolas Bamat, MD, MSCE, assistant professor of pediatrics at the Perelman School of Medicine, University of Pennsylvania, Philadelphia.

Although the number of children in the latest study was too small to generate any firm conclusions, he said, the trial provides the best data to date on furosemide in premature infants.

The medication is used frequently both on babies at risk of developing BPD and babies who have already reached BPD status. Among newborns with highest risk of dying, furosemide is indeed the “most frequently used pharmacotherapy,” Dr. Bamat said.

“What’s worth noting is that furosemide is an old medication that has been used extensively in the neonatal populations for 40 years, and that is occurring in the absence of data,” Dr. Bamat added. “This is a very important step forward.”

Dr. Greenberg and Dr. Bamat have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

A medication used to reduce fluid retention can also safely be used to prevent a dangerous lung condition that affects newborns, particularly those born premature, according to a new study.

Furosemide (Lasix) – which can reduce excess fluid in the body caused by heart failure, liver disease, and kidney trouble – is commonly used off-label to prevent bronchopulmonary dysplasia (BPD), a disorder that causes irritation and poor development of lungs in premature infants. But until now, researchers have not studied its safety in this setting.

BPD often affects babies born more than 2 months early and can sometimes result in breathing difficulties into adolescence and young adulthood.

“There are so few drugs that have been tested for newborns, and there are very little data to help neonatologists decide if certain medications are safe and effective,” said Rachel Greenberg, MD, MHS, a neonatologist and member of the Duke Clinical Research Institute, Durham, N.C. “We found there was no greater risk of safety events for newborns given furosemide.”

Dr. Greenberg presented the findings at the 2022 Pediatric Academic Societies meeting in Denver.

For the 28-day randomized controlled trial, Dr. Greenberg and colleagues enrolled 80 preterm newborns, born at less than 29 weeks’ gestation, at 17 centers within the Eunice Kennedy Shriver National Institute of Child Health and Human Development Pediatric Trials Network. Of those, 61 received furosemide and 19 received a placebo.

Although babies given furosemide had more problems with electrolytes – an expected outcome from the use of diuretic medications – the researchers observed no greater risk for more serious issues, namely hearing loss or kidney stones, Dr. Greenberg told this news organization.

“The mechanism here is we know that extra fluid can damage the lungs and can cause you to have to use more respiratory support and more oxygen,” she said. “The thought from a physiological standpoint is using a diuretic can decrease fluid in the lungs and lead to improvements in lung outcomes.”

The researchers did not observe a reduction in BDP or death in babies who received furosemide, but Dr. Greenberg said the study was underpowered to detect such an effect.

“We were not powered to detect a difference in that outcome; the overall objective of this study was always to evaluate safety,” she said. “Of course, we wanted to capture variables that would measure effectiveness as well.

“Because this was a pragmatic trial, we did not limit the amount of fluids that the clinicians could give the participating infants. This could have impacted the effectiveness of furosemide. We would need a different design and larger study to truly determine effectiveness.” 

Dr. Greenberg said she hoped the new data will provide greater insight to neonatal providers and help bolster future, more large-scale trials using furosemide in premature infants.   

The drug has previously been associated with both kidney stones and ototoxicity, which occurs when medication causes a person to develop hearing or balance problems, said Nicolas Bamat, MD, MSCE, assistant professor of pediatrics at the Perelman School of Medicine, University of Pennsylvania, Philadelphia.

Although the number of children in the latest study was too small to generate any firm conclusions, he said, the trial provides the best data to date on furosemide in premature infants.

The medication is used frequently both on babies at risk of developing BPD and babies who have already reached BPD status. Among newborns with highest risk of dying, furosemide is indeed the “most frequently used pharmacotherapy,” Dr. Bamat said.

“What’s worth noting is that furosemide is an old medication that has been used extensively in the neonatal populations for 40 years, and that is occurring in the absence of data,” Dr. Bamat added. “This is a very important step forward.”

Dr. Greenberg and Dr. Bamat have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

A medication used to reduce fluid retention can also safely be used to prevent a dangerous lung condition that affects newborns, particularly those born premature, according to a new study.

Furosemide (Lasix) – which can reduce excess fluid in the body caused by heart failure, liver disease, and kidney trouble – is commonly used off-label to prevent bronchopulmonary dysplasia (BPD), a disorder that causes irritation and poor development of lungs in premature infants. But until now, researchers have not studied its safety in this setting.

BPD often affects babies born more than 2 months early and can sometimes result in breathing difficulties into adolescence and young adulthood.

“There are so few drugs that have been tested for newborns, and there are very little data to help neonatologists decide if certain medications are safe and effective,” said Rachel Greenberg, MD, MHS, a neonatologist and member of the Duke Clinical Research Institute, Durham, N.C. “We found there was no greater risk of safety events for newborns given furosemide.”

Dr. Greenberg presented the findings at the 2022 Pediatric Academic Societies meeting in Denver.

For the 28-day randomized controlled trial, Dr. Greenberg and colleagues enrolled 80 preterm newborns, born at less than 29 weeks’ gestation, at 17 centers within the Eunice Kennedy Shriver National Institute of Child Health and Human Development Pediatric Trials Network. Of those, 61 received furosemide and 19 received a placebo.

Although babies given furosemide had more problems with electrolytes – an expected outcome from the use of diuretic medications – the researchers observed no greater risk for more serious issues, namely hearing loss or kidney stones, Dr. Greenberg told this news organization.

“The mechanism here is we know that extra fluid can damage the lungs and can cause you to have to use more respiratory support and more oxygen,” she said. “The thought from a physiological standpoint is using a diuretic can decrease fluid in the lungs and lead to improvements in lung outcomes.”

The researchers did not observe a reduction in BDP or death in babies who received furosemide, but Dr. Greenberg said the study was underpowered to detect such an effect.

“We were not powered to detect a difference in that outcome; the overall objective of this study was always to evaluate safety,” she said. “Of course, we wanted to capture variables that would measure effectiveness as well.

“Because this was a pragmatic trial, we did not limit the amount of fluids that the clinicians could give the participating infants. This could have impacted the effectiveness of furosemide. We would need a different design and larger study to truly determine effectiveness.” 

Dr. Greenberg said she hoped the new data will provide greater insight to neonatal providers and help bolster future, more large-scale trials using furosemide in premature infants.   

The drug has previously been associated with both kidney stones and ototoxicity, which occurs when medication causes a person to develop hearing or balance problems, said Nicolas Bamat, MD, MSCE, assistant professor of pediatrics at the Perelman School of Medicine, University of Pennsylvania, Philadelphia.

Although the number of children in the latest study was too small to generate any firm conclusions, he said, the trial provides the best data to date on furosemide in premature infants.

The medication is used frequently both on babies at risk of developing BPD and babies who have already reached BPD status. Among newborns with highest risk of dying, furosemide is indeed the “most frequently used pharmacotherapy,” Dr. Bamat said.

“What’s worth noting is that furosemide is an old medication that has been used extensively in the neonatal populations for 40 years, and that is occurring in the absence of data,” Dr. Bamat added. “This is a very important step forward.”

Dr. Greenberg and Dr. Bamat have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Caregivers who brought suicidal adolescents to the emergency department reported safer gun storage practices after firearm counseling – a crucial way to cut gun deaths among children, according to researchers from Cincinnati Children’s Hospital.

In the study, which took place between June 2021 and Feb 2022, gun safety counseling and handouts were provided to 99 families of children who had come to the ED with mental health problems. A separate set of 101 families in similar situations received counseling and handouts, along with two cable-style gun locks.

Four weeks later, parents in both groups reported an increase in safe storage practices in which they locked away all guns in the household. Those offered only counseling increased safe storage by 7.2% – from 89.9% to 97.1%.

The gains were greater for families that received locks in addition to counseling. The number of those who locked away all guns rose from 82.2% to 98.5% – a 16.3% increase. (Roughly one-third of families in both arms of the study were lost to follow-up, according to the researchers, which left 68 families in each group for analysis.)

Several caregivers in each group reported that guns had been removed entirely from the home, and more than 60% in each group said they had bought additional gun locks to secure their weapons.

“The main point of our study is that just-in-time counseling is very effective in helping these families of children with mental health concerns in securing all their guns, and an emergency department visit is a great time to do that,” said Bijan Ketabchi, MD, a clinical fellow in the division of emergency medicine at Cincinnati Children’s Hospital Medical Center, who presented the findings at the Pediatric Academic Societies annual meeting.

Dr. Ketabchi said his department sees 500-700 children each month with mental health concerns, most commonly depression. The mean age of adolescent patients in the study was 14 years.

Suicide is the second-leading cause of death among children in the United States. Both pediatric suicides and firearm suicides have increased in the past 2 decades, Dr. Ketabchi said. The number of youth suicides who use guns has risen 90% since 2008. One in three U.S. families own a firearm, and 4.6 million children live in a home with loaded, unlocked guns.

Among children aged 17 years and younger who die by firearm suicide, 82% used guns belonging to a family member.
 

The right time for the message

Interventions to encourage safe gun storage – at a time when caregivers are really listening – can be lifesaving, Dr. Ketabchi said.

“We know that counseling is really helpful for these families, because when they come to the emergency department with a concern, they can have a teachable moment,” he said in an interview. “It resonates with them a lot more than it normally would because they have experienced something traumatic.”

The importance of safe gun storage in households with adolescents can’t be overstated, even if the children are not at risk of suicide, said Naoka Carey, a doctoral candidate at Boston College.

Ms. Carey authored an article on the prevalence of handguns among adolescents that will be published in May in Pediatrics.

“Three kinds of harm for adolescents with access to guns are accidental injury, homicide, and suicide,” she said. “Families who own guns don’t always know their teens have access to the guns.”

The problem is getting worse. Ms. Carey and colleagues found that, between 2002 and 2019, the rate of children aged 12-17 who reported carrying handguns increased 41%. Most of them were White, and their families were in high-income brackets. New data show that firearm injuries have become the leading cause of death among youth in the United States, eclipsing auto accidents for the first time.

“Preventing tragedy in your family is more than reason enough to secure guns you have,” she said.

Dr. Ketabchi disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Caregivers who brought suicidal adolescents to the emergency department reported safer gun storage practices after firearm counseling – a crucial way to cut gun deaths among children, according to researchers from Cincinnati Children’s Hospital.

In the study, which took place between June 2021 and Feb 2022, gun safety counseling and handouts were provided to 99 families of children who had come to the ED with mental health problems. A separate set of 101 families in similar situations received counseling and handouts, along with two cable-style gun locks.

Four weeks later, parents in both groups reported an increase in safe storage practices in which they locked away all guns in the household. Those offered only counseling increased safe storage by 7.2% – from 89.9% to 97.1%.

The gains were greater for families that received locks in addition to counseling. The number of those who locked away all guns rose from 82.2% to 98.5% – a 16.3% increase. (Roughly one-third of families in both arms of the study were lost to follow-up, according to the researchers, which left 68 families in each group for analysis.)

Several caregivers in each group reported that guns had been removed entirely from the home, and more than 60% in each group said they had bought additional gun locks to secure their weapons.

“The main point of our study is that just-in-time counseling is very effective in helping these families of children with mental health concerns in securing all their guns, and an emergency department visit is a great time to do that,” said Bijan Ketabchi, MD, a clinical fellow in the division of emergency medicine at Cincinnati Children’s Hospital Medical Center, who presented the findings at the Pediatric Academic Societies annual meeting.

Dr. Ketabchi said his department sees 500-700 children each month with mental health concerns, most commonly depression. The mean age of adolescent patients in the study was 14 years.

Suicide is the second-leading cause of death among children in the United States. Both pediatric suicides and firearm suicides have increased in the past 2 decades, Dr. Ketabchi said. The number of youth suicides who use guns has risen 90% since 2008. One in three U.S. families own a firearm, and 4.6 million children live in a home with loaded, unlocked guns.

Among children aged 17 years and younger who die by firearm suicide, 82% used guns belonging to a family member.
 

The right time for the message

Interventions to encourage safe gun storage – at a time when caregivers are really listening – can be lifesaving, Dr. Ketabchi said.

“We know that counseling is really helpful for these families, because when they come to the emergency department with a concern, they can have a teachable moment,” he said in an interview. “It resonates with them a lot more than it normally would because they have experienced something traumatic.”

The importance of safe gun storage in households with adolescents can’t be overstated, even if the children are not at risk of suicide, said Naoka Carey, a doctoral candidate at Boston College.

Ms. Carey authored an article on the prevalence of handguns among adolescents that will be published in May in Pediatrics.

“Three kinds of harm for adolescents with access to guns are accidental injury, homicide, and suicide,” she said. “Families who own guns don’t always know their teens have access to the guns.”

The problem is getting worse. Ms. Carey and colleagues found that, between 2002 and 2019, the rate of children aged 12-17 who reported carrying handguns increased 41%. Most of them were White, and their families were in high-income brackets. New data show that firearm injuries have become the leading cause of death among youth in the United States, eclipsing auto accidents for the first time.

“Preventing tragedy in your family is more than reason enough to secure guns you have,” she said.

Dr. Ketabchi disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Caregivers who brought suicidal adolescents to the emergency department reported safer gun storage practices after firearm counseling – a crucial way to cut gun deaths among children, according to researchers from Cincinnati Children’s Hospital.

In the study, which took place between June 2021 and Feb 2022, gun safety counseling and handouts were provided to 99 families of children who had come to the ED with mental health problems. A separate set of 101 families in similar situations received counseling and handouts, along with two cable-style gun locks.

Four weeks later, parents in both groups reported an increase in safe storage practices in which they locked away all guns in the household. Those offered only counseling increased safe storage by 7.2% – from 89.9% to 97.1%.

The gains were greater for families that received locks in addition to counseling. The number of those who locked away all guns rose from 82.2% to 98.5% – a 16.3% increase. (Roughly one-third of families in both arms of the study were lost to follow-up, according to the researchers, which left 68 families in each group for analysis.)

Several caregivers in each group reported that guns had been removed entirely from the home, and more than 60% in each group said they had bought additional gun locks to secure their weapons.

“The main point of our study is that just-in-time counseling is very effective in helping these families of children with mental health concerns in securing all their guns, and an emergency department visit is a great time to do that,” said Bijan Ketabchi, MD, a clinical fellow in the division of emergency medicine at Cincinnati Children’s Hospital Medical Center, who presented the findings at the Pediatric Academic Societies annual meeting.

Dr. Ketabchi said his department sees 500-700 children each month with mental health concerns, most commonly depression. The mean age of adolescent patients in the study was 14 years.

Suicide is the second-leading cause of death among children in the United States. Both pediatric suicides and firearm suicides have increased in the past 2 decades, Dr. Ketabchi said. The number of youth suicides who use guns has risen 90% since 2008. One in three U.S. families own a firearm, and 4.6 million children live in a home with loaded, unlocked guns.

Among children aged 17 years and younger who die by firearm suicide, 82% used guns belonging to a family member.
 

The right time for the message

Interventions to encourage safe gun storage – at a time when caregivers are really listening – can be lifesaving, Dr. Ketabchi said.

“We know that counseling is really helpful for these families, because when they come to the emergency department with a concern, they can have a teachable moment,” he said in an interview. “It resonates with them a lot more than it normally would because they have experienced something traumatic.”

The importance of safe gun storage in households with adolescents can’t be overstated, even if the children are not at risk of suicide, said Naoka Carey, a doctoral candidate at Boston College.

Ms. Carey authored an article on the prevalence of handguns among adolescents that will be published in May in Pediatrics.

“Three kinds of harm for adolescents with access to guns are accidental injury, homicide, and suicide,” she said. “Families who own guns don’t always know their teens have access to the guns.”

The problem is getting worse. Ms. Carey and colleagues found that, between 2002 and 2019, the rate of children aged 12-17 who reported carrying handguns increased 41%. Most of them were White, and their families were in high-income brackets. New data show that firearm injuries have become the leading cause of death among youth in the United States, eclipsing auto accidents for the first time.

“Preventing tragedy in your family is more than reason enough to secure guns you have,” she said.

Dr. Ketabchi disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Stem cell therapy improves motor impairment and is safe and well tolerated in patients with traumatic brain injury (TBI), results from a phase 2 trial indicate. “We proved for the first time that we can affect outcomes in moderately to severely disabled patients with TBI using stem cells,” said study investigator Peter McAllister, MD, cofounder and medical director of the New England Center for Neurology and Headache, Stamford, Conn.

“I think the potential of regenerative medicine was always out there, but we are now getting to the point where we’re living up to that potential,” said Dr. McAllister, associate professor of neurology at Yale University, New Haven, Conn.

The findings were presented at the 2022 annual meeting of the American Academy of Neurology.
 

No effective treatment to date

TBI can lead to motor deficits and chronic disability and currently there are no effective drugs to treat these deficits.

Researchers are increasingly focused on using somatic stem cells to restore lost function. Stem cells can differentiate or proliferate into different types of cells and are thought to promote repair and regeneration of tissues or organs damaged due to illness or injury.

The study included 61 patients with TBI with an average age of 34 years (70% were male and 69% were White). The mean time from injury was 8 years and Glasgow Outcome Scale Extended (GOS-E) ranged from 3 to 6.

Forty-six participants were randomly assigned to receive the stem cell therapy and 15 a sham procedure. In the treatment group, there were three different doses of cells (2.5 x 106, 5 x 106, and 10 x 106).

The treatment involved an investigational regenerative cell medicine comprised of bone marrow-derived mesenchymal stem cells (SB623). The allogeneic cells came from a male donor.

For the 20-minute procedure, a neurosurgeon drilled a tiny hole in the skull and, guided by MRI, injected the stem cells into the area of the lesion.

Patients receiving a surgical sham procedure were brought to the operating room, anesthetized, and had a hole drilled into the head over the area of the lesion. However, the surgeon went only halfway through the skull bone.

Participants were instructed to do specific physiotherapy exercises at home every morning and afternoon for the first 6 months of the study.

The primary efficacy endpoint was change in the Fugl-Meyer Motor Scale score (FMMS). This scale is widely used for clinical assessment of motor function, including range of motion, walking, lower limb movement, and dexterity.

At 24 weeks, the change in FMMS score for SB623-treated patients (least square [LS] mean increase 8.3) compared with controls (LS increase 2.3) was significant (P = .04).

“When we looked at all the data at 6 months, the folks who got the stem cells did statistically significantly better than the group that got the sham,” and that improvement began within the first week or two, said Dr. McAllister.
 

‘A real impact’

The treatment had a real impact on people’s lives, he said. “Some who couldn’t move their arm at all were able to put a nut on a bolt or brush their teeth, and some were able to button and unbutton where they couldn’t do that before.”

One teenager who was previously completely aphasic spoke an entire sentence.

The middle dose (5 x 106) had “by far” the best outcome, said Dr. McAllister. It’s not yet known whether the improvements will be permanent, he added.

At 48 weeks, treated patients experienced improvement over controls in secondary endpoints of the Action Research Arm Test (ARAT), which assesses grasp, grip, pinch, and gross movements; Gait Velocity (walking 10 meters); and NeuroQOL, a self-report measure of ability to carry out various activities.

However, although these endpoints were all numerically better in the stem cell groups, none reached statistical significance. This is likely because of the small study size and the fact the control group improved so much, said Dr. McAllister.

The exact mechanism of stem cell therapy is unclear, but researchers believe it “establishes a milieu of growth” for cells in the brain and promotes anti-inflammatory properties, said Dr. McAllister.

By 48 weeks, all study subjects had experienced at least one adverse event, with no differences between groups and no patient withdrawing as a result of adverse events. “There was no safety signal at all related to the stem cells,” said Dr. McAllister.

A larger phase 3 study of SB623 is planned.

The treatment may be useful in other conditions. A study of stroke survivors “just barely missed statistical significance” likely for methodological reasons and an older, sicker population, but the company plans to do another study in patients who were affected by stroke, said Dr. McAllister.

In addition, there may be potential for this approach with brain hemorrhage, Parkinson’s disease, multiple sclerosis, and other brain-related disorders, he said.
 

‘Modern-day holy grail’

Reached for a comment, TBI specialist Frank Conidi, MD, director of the Florida Center for Headache and Sports Neurology, said stem cell therapy is the most promising potential treatment for brain injury. “It’s the modern-day ‘holy grail.’ “

In this study, “to see a modest improvement in gait in the primary outcome is impressive,” he said.

In addition, the fact the study didn’t have any significant or severe adverse outcomes “is promising,” he added.

Studies like this “are going to help to lay the groundwork for future studies and hopefully one day result in a safe, noninvasive treatment” for Parkinson’s disease, Alzheimer’s disease, and disorders that affect the central nervous system such as spinal cord injury, Dr. Conidi said.

This therapy involves an invasive procedure requiring implantation directly into the brain, he noted. “At present, there’s no way to get stem cells to cross the blood-brain barrier.”

In addition, although motor impairment is definitely a component of TBI, it’s not as prevalent as cognitive impairment, said Dr. Conidi.

The study was supported by SanBio Co Ltd. Dr. McAllister and Dr. Conidi have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Stem cell therapy improves motor impairment and is safe and well tolerated in patients with traumatic brain injury (TBI), results from a phase 2 trial indicate. “We proved for the first time that we can affect outcomes in moderately to severely disabled patients with TBI using stem cells,” said study investigator Peter McAllister, MD, cofounder and medical director of the New England Center for Neurology and Headache, Stamford, Conn.

“I think the potential of regenerative medicine was always out there, but we are now getting to the point where we’re living up to that potential,” said Dr. McAllister, associate professor of neurology at Yale University, New Haven, Conn.

The findings were presented at the 2022 annual meeting of the American Academy of Neurology.
 

No effective treatment to date

TBI can lead to motor deficits and chronic disability and currently there are no effective drugs to treat these deficits.

Researchers are increasingly focused on using somatic stem cells to restore lost function. Stem cells can differentiate or proliferate into different types of cells and are thought to promote repair and regeneration of tissues or organs damaged due to illness or injury.

The study included 61 patients with TBI with an average age of 34 years (70% were male and 69% were White). The mean time from injury was 8 years and Glasgow Outcome Scale Extended (GOS-E) ranged from 3 to 6.

Forty-six participants were randomly assigned to receive the stem cell therapy and 15 a sham procedure. In the treatment group, there were three different doses of cells (2.5 x 106, 5 x 106, and 10 x 106).

The treatment involved an investigational regenerative cell medicine comprised of bone marrow-derived mesenchymal stem cells (SB623). The allogeneic cells came from a male donor.

For the 20-minute procedure, a neurosurgeon drilled a tiny hole in the skull and, guided by MRI, injected the stem cells into the area of the lesion.

Patients receiving a surgical sham procedure were brought to the operating room, anesthetized, and had a hole drilled into the head over the area of the lesion. However, the surgeon went only halfway through the skull bone.

Participants were instructed to do specific physiotherapy exercises at home every morning and afternoon for the first 6 months of the study.

The primary efficacy endpoint was change in the Fugl-Meyer Motor Scale score (FMMS). This scale is widely used for clinical assessment of motor function, including range of motion, walking, lower limb movement, and dexterity.

At 24 weeks, the change in FMMS score for SB623-treated patients (least square [LS] mean increase 8.3) compared with controls (LS increase 2.3) was significant (P = .04).

“When we looked at all the data at 6 months, the folks who got the stem cells did statistically significantly better than the group that got the sham,” and that improvement began within the first week or two, said Dr. McAllister.
 

‘A real impact’

The treatment had a real impact on people’s lives, he said. “Some who couldn’t move their arm at all were able to put a nut on a bolt or brush their teeth, and some were able to button and unbutton where they couldn’t do that before.”

One teenager who was previously completely aphasic spoke an entire sentence.

The middle dose (5 x 106) had “by far” the best outcome, said Dr. McAllister. It’s not yet known whether the improvements will be permanent, he added.

At 48 weeks, treated patients experienced improvement over controls in secondary endpoints of the Action Research Arm Test (ARAT), which assesses grasp, grip, pinch, and gross movements; Gait Velocity (walking 10 meters); and NeuroQOL, a self-report measure of ability to carry out various activities.

However, although these endpoints were all numerically better in the stem cell groups, none reached statistical significance. This is likely because of the small study size and the fact the control group improved so much, said Dr. McAllister.

The exact mechanism of stem cell therapy is unclear, but researchers believe it “establishes a milieu of growth” for cells in the brain and promotes anti-inflammatory properties, said Dr. McAllister.

By 48 weeks, all study subjects had experienced at least one adverse event, with no differences between groups and no patient withdrawing as a result of adverse events. “There was no safety signal at all related to the stem cells,” said Dr. McAllister.

A larger phase 3 study of SB623 is planned.

The treatment may be useful in other conditions. A study of stroke survivors “just barely missed statistical significance” likely for methodological reasons and an older, sicker population, but the company plans to do another study in patients who were affected by stroke, said Dr. McAllister.

In addition, there may be potential for this approach with brain hemorrhage, Parkinson’s disease, multiple sclerosis, and other brain-related disorders, he said.
 

‘Modern-day holy grail’

Reached for a comment, TBI specialist Frank Conidi, MD, director of the Florida Center for Headache and Sports Neurology, said stem cell therapy is the most promising potential treatment for brain injury. “It’s the modern-day ‘holy grail.’ “

In this study, “to see a modest improvement in gait in the primary outcome is impressive,” he said.

In addition, the fact the study didn’t have any significant or severe adverse outcomes “is promising,” he added.

Studies like this “are going to help to lay the groundwork for future studies and hopefully one day result in a safe, noninvasive treatment” for Parkinson’s disease, Alzheimer’s disease, and disorders that affect the central nervous system such as spinal cord injury, Dr. Conidi said.

This therapy involves an invasive procedure requiring implantation directly into the brain, he noted. “At present, there’s no way to get stem cells to cross the blood-brain barrier.”

In addition, although motor impairment is definitely a component of TBI, it’s not as prevalent as cognitive impairment, said Dr. Conidi.

The study was supported by SanBio Co Ltd. Dr. McAllister and Dr. Conidi have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Stem cell therapy improves motor impairment and is safe and well tolerated in patients with traumatic brain injury (TBI), results from a phase 2 trial indicate. “We proved for the first time that we can affect outcomes in moderately to severely disabled patients with TBI using stem cells,” said study investigator Peter McAllister, MD, cofounder and medical director of the New England Center for Neurology and Headache, Stamford, Conn.

“I think the potential of regenerative medicine was always out there, but we are now getting to the point where we’re living up to that potential,” said Dr. McAllister, associate professor of neurology at Yale University, New Haven, Conn.

The findings were presented at the 2022 annual meeting of the American Academy of Neurology.
 

No effective treatment to date

TBI can lead to motor deficits and chronic disability and currently there are no effective drugs to treat these deficits.

Researchers are increasingly focused on using somatic stem cells to restore lost function. Stem cells can differentiate or proliferate into different types of cells and are thought to promote repair and regeneration of tissues or organs damaged due to illness or injury.

The study included 61 patients with TBI with an average age of 34 years (70% were male and 69% were White). The mean time from injury was 8 years and Glasgow Outcome Scale Extended (GOS-E) ranged from 3 to 6.

Forty-six participants were randomly assigned to receive the stem cell therapy and 15 a sham procedure. In the treatment group, there were three different doses of cells (2.5 x 106, 5 x 106, and 10 x 106).

The treatment involved an investigational regenerative cell medicine comprised of bone marrow-derived mesenchymal stem cells (SB623). The allogeneic cells came from a male donor.

For the 20-minute procedure, a neurosurgeon drilled a tiny hole in the skull and, guided by MRI, injected the stem cells into the area of the lesion.

Patients receiving a surgical sham procedure were brought to the operating room, anesthetized, and had a hole drilled into the head over the area of the lesion. However, the surgeon went only halfway through the skull bone.

Participants were instructed to do specific physiotherapy exercises at home every morning and afternoon for the first 6 months of the study.

The primary efficacy endpoint was change in the Fugl-Meyer Motor Scale score (FMMS). This scale is widely used for clinical assessment of motor function, including range of motion, walking, lower limb movement, and dexterity.

At 24 weeks, the change in FMMS score for SB623-treated patients (least square [LS] mean increase 8.3) compared with controls (LS increase 2.3) was significant (P = .04).

“When we looked at all the data at 6 months, the folks who got the stem cells did statistically significantly better than the group that got the sham,” and that improvement began within the first week or two, said Dr. McAllister.
 

‘A real impact’

The treatment had a real impact on people’s lives, he said. “Some who couldn’t move their arm at all were able to put a nut on a bolt or brush their teeth, and some were able to button and unbutton where they couldn’t do that before.”

One teenager who was previously completely aphasic spoke an entire sentence.

The middle dose (5 x 106) had “by far” the best outcome, said Dr. McAllister. It’s not yet known whether the improvements will be permanent, he added.

At 48 weeks, treated patients experienced improvement over controls in secondary endpoints of the Action Research Arm Test (ARAT), which assesses grasp, grip, pinch, and gross movements; Gait Velocity (walking 10 meters); and NeuroQOL, a self-report measure of ability to carry out various activities.

However, although these endpoints were all numerically better in the stem cell groups, none reached statistical significance. This is likely because of the small study size and the fact the control group improved so much, said Dr. McAllister.

The exact mechanism of stem cell therapy is unclear, but researchers believe it “establishes a milieu of growth” for cells in the brain and promotes anti-inflammatory properties, said Dr. McAllister.

By 48 weeks, all study subjects had experienced at least one adverse event, with no differences between groups and no patient withdrawing as a result of adverse events. “There was no safety signal at all related to the stem cells,” said Dr. McAllister.

A larger phase 3 study of SB623 is planned.

The treatment may be useful in other conditions. A study of stroke survivors “just barely missed statistical significance” likely for methodological reasons and an older, sicker population, but the company plans to do another study in patients who were affected by stroke, said Dr. McAllister.

In addition, there may be potential for this approach with brain hemorrhage, Parkinson’s disease, multiple sclerosis, and other brain-related disorders, he said.
 

‘Modern-day holy grail’

Reached for a comment, TBI specialist Frank Conidi, MD, director of the Florida Center for Headache and Sports Neurology, said stem cell therapy is the most promising potential treatment for brain injury. “It’s the modern-day ‘holy grail.’ “

In this study, “to see a modest improvement in gait in the primary outcome is impressive,” he said.

In addition, the fact the study didn’t have any significant or severe adverse outcomes “is promising,” he added.

Studies like this “are going to help to lay the groundwork for future studies and hopefully one day result in a safe, noninvasive treatment” for Parkinson’s disease, Alzheimer’s disease, and disorders that affect the central nervous system such as spinal cord injury, Dr. Conidi said.

This therapy involves an invasive procedure requiring implantation directly into the brain, he noted. “At present, there’s no way to get stem cells to cross the blood-brain barrier.”

In addition, although motor impairment is definitely a component of TBI, it’s not as prevalent as cognitive impairment, said Dr. Conidi.

The study was supported by SanBio Co Ltd. Dr. McAllister and Dr. Conidi have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Key clinical point: Donor sex did not affect post-liver transplantation (LT) recurrence of hepatocellular carcinoma (HCC) and need not be considered during donor selection or organ allocation.

Major finding: After propensity score matching, the female donor (F-D) and male donor (M-D) groups showed comparable 5-year overall recurrence rates (15% vs. 14%; P = .63) and graft recurrence rates (5% vs. 5%; P = .94). Donor sex was not identified as a significant risk factor for HCC recurrence by either univariate or multivariate analysis.

Study details: This study evaluated 1118 patients with HCC who underwent LT receiving a liver graft from the F-D (n = 446) or M-D (n = 672) groups.

Disclosures: The authors did not declare any funding source or conflicts of interest.

Source: Taura K et al. No impact of donor sex on the recurrence of hepatocellular carcinoma after liver transplantation. J Hepatobiliary Pancreat Sci. 2022 (Mar 13). Doi: 10.1002/jhbp.1134

Key clinical point: Donor sex did not affect post-liver transplantation (LT) recurrence of hepatocellular carcinoma (HCC) and need not be considered during donor selection or organ allocation.

Major finding: After propensity score matching, the female donor (F-D) and male donor (M-D) groups showed comparable 5-year overall recurrence rates (15% vs. 14%; P = .63) and graft recurrence rates (5% vs. 5%; P = .94). Donor sex was not identified as a significant risk factor for HCC recurrence by either univariate or multivariate analysis.

Study details: This study evaluated 1118 patients with HCC who underwent LT receiving a liver graft from the F-D (n = 446) or M-D (n = 672) groups.

Disclosures: The authors did not declare any funding source or conflicts of interest.

Source: Taura K et al. No impact of donor sex on the recurrence of hepatocellular carcinoma after liver transplantation. J Hepatobiliary Pancreat Sci. 2022 (Mar 13). Doi: 10.1002/jhbp.1134

Key clinical point: Donor sex did not affect post-liver transplantation (LT) recurrence of hepatocellular carcinoma (HCC) and need not be considered during donor selection or organ allocation.

Major finding: After propensity score matching, the female donor (F-D) and male donor (M-D) groups showed comparable 5-year overall recurrence rates (15% vs. 14%; P = .63) and graft recurrence rates (5% vs. 5%; P = .94). Donor sex was not identified as a significant risk factor for HCC recurrence by either univariate or multivariate analysis.

Study details: This study evaluated 1118 patients with HCC who underwent LT receiving a liver graft from the F-D (n = 446) or M-D (n = 672) groups.

Disclosures: The authors did not declare any funding source or conflicts of interest.

Source: Taura K et al. No impact of donor sex on the recurrence of hepatocellular carcinoma after liver transplantation. J Hepatobiliary Pancreat Sci. 2022 (Mar 13). Doi: 10.1002/jhbp.1134

Key clinical point: When technically feasible, surgical liver resection (LR) should be recommended to patients with hepatocellular carcinoma (HCC) with bile duct tumor thrombus (BDTT) over transcatheter arterial chemoembolization (TACE) because it provides better prognosis.

Major finding: After propensity score matching, patients who underwent LR vs. TACE showed a significantly longer median overall survival (20.0 vs. 11.0 months; P < .001) and disease-free survival (7.0 vs. 2.0 months; P = .007).

Study details: Findings are from a retrospective study including 145 patients with HCC with BDTT who underwent LR (n = 105) or TACE (n = 40).

Disclosures: The study was sponsored by the National Natural Science Foundation of China. The authors declared no conflicts of interest.

Source: Liu Z-H et al. Prognostic comparison between liver resection and transcatheter arterial chemoembolization for hepatocellular carcinoma patients with bile duct tumor thrombus: A propensity-score matching analysis. Front Oncol. 2022;12:835559 (Mar 15). Doi: 10.3389/fonc.2022.835559

Key clinical point: When technically feasible, surgical liver resection (LR) should be recommended to patients with hepatocellular carcinoma (HCC) with bile duct tumor thrombus (BDTT) over transcatheter arterial chemoembolization (TACE) because it provides better prognosis.

Major finding: After propensity score matching, patients who underwent LR vs. TACE showed a significantly longer median overall survival (20.0 vs. 11.0 months; P < .001) and disease-free survival (7.0 vs. 2.0 months; P = .007).

Study details: Findings are from a retrospective study including 145 patients with HCC with BDTT who underwent LR (n = 105) or TACE (n = 40).

Disclosures: The study was sponsored by the National Natural Science Foundation of China. The authors declared no conflicts of interest.

Source: Liu Z-H et al. Prognostic comparison between liver resection and transcatheter arterial chemoembolization for hepatocellular carcinoma patients with bile duct tumor thrombus: A propensity-score matching analysis. Front Oncol. 2022;12:835559 (Mar 15). Doi: 10.3389/fonc.2022.835559

Key clinical point: When technically feasible, surgical liver resection (LR) should be recommended to patients with hepatocellular carcinoma (HCC) with bile duct tumor thrombus (BDTT) over transcatheter arterial chemoembolization (TACE) because it provides better prognosis.

Major finding: After propensity score matching, patients who underwent LR vs. TACE showed a significantly longer median overall survival (20.0 vs. 11.0 months; P < .001) and disease-free survival (7.0 vs. 2.0 months; P = .007).

Study details: Findings are from a retrospective study including 145 patients with HCC with BDTT who underwent LR (n = 105) or TACE (n = 40).

Disclosures: The study was sponsored by the National Natural Science Foundation of China. The authors declared no conflicts of interest.

Source: Liu Z-H et al. Prognostic comparison between liver resection and transcatheter arterial chemoembolization for hepatocellular carcinoma patients with bile duct tumor thrombus: A propensity-score matching analysis. Front Oncol. 2022;12:835559 (Mar 15). Doi: 10.3389/fonc.2022.835559

Key clinical point: Transarterial chemoembolization (TACE) demonstrated a comparable safety profile between elderly and younger patients with intermediate hepatocellular carcinoma (HCC), with its efficacy remaining uncompromised with advancing age.

Major finding: The occurrence rate of at least one serious adverse event was similar between elderly and younger patients (20.5% vs. 21.3%; P = .87). The objective tumor response rate did not decline in the elderly patients (89.5%) compared with that in younger patients (78.7%).

Study details: The data come from a retrospective study including 271 patients aged >18 years with intermediate HCC who underwent the first session of TACE, of which 88 were elderly patients (≥70 years old).

Disclosures: The study received no financial support. The authors declared no conflicts of interest.

Source: Roth GS et al. Safety and efficacy of transarterial chemoembolization in elderly patients with intermediate hepatocellular carcinoma. Cancers. 2022;14(7):1634 (Mar 23). Doi:  10.3390/cancers14071634

Key clinical point: Transarterial chemoembolization (TACE) demonstrated a comparable safety profile between elderly and younger patients with intermediate hepatocellular carcinoma (HCC), with its efficacy remaining uncompromised with advancing age.

Major finding: The occurrence rate of at least one serious adverse event was similar between elderly and younger patients (20.5% vs. 21.3%; P = .87). The objective tumor response rate did not decline in the elderly patients (89.5%) compared with that in younger patients (78.7%).

Study details: The data come from a retrospective study including 271 patients aged >18 years with intermediate HCC who underwent the first session of TACE, of which 88 were elderly patients (≥70 years old).

Disclosures: The study received no financial support. The authors declared no conflicts of interest.

Source: Roth GS et al. Safety and efficacy of transarterial chemoembolization in elderly patients with intermediate hepatocellular carcinoma. Cancers. 2022;14(7):1634 (Mar 23). Doi:  10.3390/cancers14071634

Key clinical point: Transarterial chemoembolization (TACE) demonstrated a comparable safety profile between elderly and younger patients with intermediate hepatocellular carcinoma (HCC), with its efficacy remaining uncompromised with advancing age.

Major finding: The occurrence rate of at least one serious adverse event was similar between elderly and younger patients (20.5% vs. 21.3%; P = .87). The objective tumor response rate did not decline in the elderly patients (89.5%) compared with that in younger patients (78.7%).

Study details: The data come from a retrospective study including 271 patients aged >18 years with intermediate HCC who underwent the first session of TACE, of which 88 were elderly patients (≥70 years old).

Disclosures: The study received no financial support. The authors declared no conflicts of interest.

Source: Roth GS et al. Safety and efficacy of transarterial chemoembolization in elderly patients with intermediate hepatocellular carcinoma. Cancers. 2022;14(7):1634 (Mar 23). Doi:  10.3390/cancers14071634

Key clinical point: In patients with hepatitis B virus (HBV)-related hepatocellular carcinoma (HCC) who had undergone radiofrequency ablation (RFA), tenofovir disoproxil fumarate (TDF) performed better at protecting liver function and reducing HBV DNA loads than entecavir (ETV), without significant difference in recurrence or overall survival.

Major finding: Patients receiving TDF vs. ETV showed significantly faster serum HBV DNA reduction (2.75 vs. 9.13 months; P = .015) and a higher stabilization/improvement rate of the albumin-bilirubin grade (64% vs. 41%; P < .001), but similar 5-year recurrence (40.3% vs. 40.8%; P = .35) and overall survival (93.5% vs. 96.9%; P = .12) rates.

Study details: This single-center retrospective cohort study propensity score-matched patients receiving ETV (n = 130) with those receiving TDF (n = 77) for chronic HBV infection after undergoing RFA as a curative treatment for HCC.

Disclosures: The study was funded by the National Natural Science Foundation of China and Sun Yat-sen University Cancer Center physician scientist funding. No conflicts of interest were reported.

Source: Hu Z et al. Tenofovir vs. entecavir on outcomes of hepatitis B virus-related hepatocellular carcinoma after radiofrequency ablation. Viruses. 2022;14(4):656 (Mar 22). Doi: 10.3390/v14040656

Key clinical point: In patients with hepatitis B virus (HBV)-related hepatocellular carcinoma (HCC) who had undergone radiofrequency ablation (RFA), tenofovir disoproxil fumarate (TDF) performed better at protecting liver function and reducing HBV DNA loads than entecavir (ETV), without significant difference in recurrence or overall survival.

Major finding: Patients receiving TDF vs. ETV showed significantly faster serum HBV DNA reduction (2.75 vs. 9.13 months; P = .015) and a higher stabilization/improvement rate of the albumin-bilirubin grade (64% vs. 41%; P < .001), but similar 5-year recurrence (40.3% vs. 40.8%; P = .35) and overall survival (93.5% vs. 96.9%; P = .12) rates.

Study details: This single-center retrospective cohort study propensity score-matched patients receiving ETV (n = 130) with those receiving TDF (n = 77) for chronic HBV infection after undergoing RFA as a curative treatment for HCC.

Disclosures: The study was funded by the National Natural Science Foundation of China and Sun Yat-sen University Cancer Center physician scientist funding. No conflicts of interest were reported.

Source: Hu Z et al. Tenofovir vs. entecavir on outcomes of hepatitis B virus-related hepatocellular carcinoma after radiofrequency ablation. Viruses. 2022;14(4):656 (Mar 22). Doi: 10.3390/v14040656

Key clinical point: In patients with hepatitis B virus (HBV)-related hepatocellular carcinoma (HCC) who had undergone radiofrequency ablation (RFA), tenofovir disoproxil fumarate (TDF) performed better at protecting liver function and reducing HBV DNA loads than entecavir (ETV), without significant difference in recurrence or overall survival.

Major finding: Patients receiving TDF vs. ETV showed significantly faster serum HBV DNA reduction (2.75 vs. 9.13 months; P = .015) and a higher stabilization/improvement rate of the albumin-bilirubin grade (64% vs. 41%; P < .001), but similar 5-year recurrence (40.3% vs. 40.8%; P = .35) and overall survival (93.5% vs. 96.9%; P = .12) rates.

Study details: This single-center retrospective cohort study propensity score-matched patients receiving ETV (n = 130) with those receiving TDF (n = 77) for chronic HBV infection after undergoing RFA as a curative treatment for HCC.

Disclosures: The study was funded by the National Natural Science Foundation of China and Sun Yat-sen University Cancer Center physician scientist funding. No conflicts of interest were reported.

Source: Hu Z et al. Tenofovir vs. entecavir on outcomes of hepatitis B virus-related hepatocellular carcinoma after radiofrequency ablation. Viruses. 2022;14(4):656 (Mar 22). Doi: 10.3390/v14040656

Key clinical point: Although lenvatinib plus pembrolizumab exhibits similar tolerability between systemic therapy-naive and -experienced patients with unresectable hepatocellular carcinoma (uHCC), the progression-free survival (PFS), objective response rate (ORR), and disease control rate (DCR) may be compromised in patients with prior systemic therapy.

Major finding: After a 9.3-month median follow-up, therapy-naive vs. -experienced patients showed numerically greater median PFS (9.2 vs. 4.9 months; P = .092), ORR (34.1% vs. 18.5%; P = .157), and DCR (84.1% vs. 70.4%; P = .169), but similar incidence rates of treatment-emergent adverse events (96.4% vs. 97.7%).

Study details: Findings are from a prospective study that enrolled 71 patients with uHCC who were systemic therapy-naive (n = 44) or -experienced (n =2 7) and received lenvatinib plus pembrolizumab.

Disclosures: The study received financial support from Taipei Veteran General Hospital, Taiwan. The authors declared no conflicts of interest.

Source: Wu C-J et al. Lenvatinib plus pembrolizumab for systemic therapy-naïve and -experienced unresectable hepatocellular carcinoma. Cancer Immunol Immunother. 2022 (Mar 28). Doi: 10.1007/s00262-022-03185-6

Key clinical point: Although lenvatinib plus pembrolizumab exhibits similar tolerability between systemic therapy-naive and -experienced patients with unresectable hepatocellular carcinoma (uHCC), the progression-free survival (PFS), objective response rate (ORR), and disease control rate (DCR) may be compromised in patients with prior systemic therapy.

Major finding: After a 9.3-month median follow-up, therapy-naive vs. -experienced patients showed numerically greater median PFS (9.2 vs. 4.9 months; P = .092), ORR (34.1% vs. 18.5%; P = .157), and DCR (84.1% vs. 70.4%; P = .169), but similar incidence rates of treatment-emergent adverse events (96.4% vs. 97.7%).

Study details: Findings are from a prospective study that enrolled 71 patients with uHCC who were systemic therapy-naive (n = 44) or -experienced (n =2 7) and received lenvatinib plus pembrolizumab.

Disclosures: The study received financial support from Taipei Veteran General Hospital, Taiwan. The authors declared no conflicts of interest.

Source: Wu C-J et al. Lenvatinib plus pembrolizumab for systemic therapy-naïve and -experienced unresectable hepatocellular carcinoma. Cancer Immunol Immunother. 2022 (Mar 28). Doi: 10.1007/s00262-022-03185-6

Key clinical point: Although lenvatinib plus pembrolizumab exhibits similar tolerability between systemic therapy-naive and -experienced patients with unresectable hepatocellular carcinoma (uHCC), the progression-free survival (PFS), objective response rate (ORR), and disease control rate (DCR) may be compromised in patients with prior systemic therapy.

Major finding: After a 9.3-month median follow-up, therapy-naive vs. -experienced patients showed numerically greater median PFS (9.2 vs. 4.9 months; P = .092), ORR (34.1% vs. 18.5%; P = .157), and DCR (84.1% vs. 70.4%; P = .169), but similar incidence rates of treatment-emergent adverse events (96.4% vs. 97.7%).

Study details: Findings are from a prospective study that enrolled 71 patients with uHCC who were systemic therapy-naive (n = 44) or -experienced (n =2 7) and received lenvatinib plus pembrolizumab.

Disclosures: The study received financial support from Taipei Veteran General Hospital, Taiwan. The authors declared no conflicts of interest.

Source: Wu C-J et al. Lenvatinib plus pembrolizumab for systemic therapy-naïve and -experienced unresectable hepatocellular carcinoma. Cancer Immunol Immunother. 2022 (Mar 28). Doi: 10.1007/s00262-022-03185-6

Key clinical point: Atezolizumab plus bevacizumab (AtezoBev) is an effective therapeutic option for unresectable hepatocellular carcinoma (uHCC) in routine clinical practice and is safe even in patients with Child-Pugh (CP)-B grade liver function.

Major finding: After a 9-month median follow-up, median overall survival was 14.9 months (95% CI 13.6-16.3 months) and median progression-free survival was 6.8 months (95% CI 5.2-8.5 months). Tolerability was similar across CP classes, with comparable bevacizumab-related (CP-A, 48%; CP-B, 46%) and atezolizumab-related (CP-A, 53%; CP-B, 40%) adverse event rates of any grade.

Study details: This was a multicenter retrospective study that included 202 adult patients with uHCC and CP-A (76%) or CP-B (24%) cirrhosis who received AtezoBev as the first-line systemic treatment.

Disclosures: The study was funded by the National Institute of Health Research Imperial Biomedical Research Centre, among others. Some authors declared serving as consultants or advisors for or receiving advisory board honoraria, lecture/speaker fees, research grants, or travel/accommodation expenses from various sources.

Source: D'Alessio A et al. Preliminary evidence of safety and tolerability of atezolizumab plus bevacizumab in patients with hepatocellular carcinoma and Child-Pugh A and B cirrhosis: A real-world study. Hepatology. 2022 (Mar 21). Doi: 10.1002/hep.32468

Key clinical point: Atezolizumab plus bevacizumab (AtezoBev) is an effective therapeutic option for unresectable hepatocellular carcinoma (uHCC) in routine clinical practice and is safe even in patients with Child-Pugh (CP)-B grade liver function.

Major finding: After a 9-month median follow-up, median overall survival was 14.9 months (95% CI 13.6-16.3 months) and median progression-free survival was 6.8 months (95% CI 5.2-8.5 months). Tolerability was similar across CP classes, with comparable bevacizumab-related (CP-A, 48%; CP-B, 46%) and atezolizumab-related (CP-A, 53%; CP-B, 40%) adverse event rates of any grade.

Study details: This was a multicenter retrospective study that included 202 adult patients with uHCC and CP-A (76%) or CP-B (24%) cirrhosis who received AtezoBev as the first-line systemic treatment.

Disclosures: The study was funded by the National Institute of Health Research Imperial Biomedical Research Centre, among others. Some authors declared serving as consultants or advisors for or receiving advisory board honoraria, lecture/speaker fees, research grants, or travel/accommodation expenses from various sources.

Source: D'Alessio A et al. Preliminary evidence of safety and tolerability of atezolizumab plus bevacizumab in patients with hepatocellular carcinoma and Child-Pugh A and B cirrhosis: A real-world study. Hepatology. 2022 (Mar 21). Doi: 10.1002/hep.32468

Key clinical point: Atezolizumab plus bevacizumab (AtezoBev) is an effective therapeutic option for unresectable hepatocellular carcinoma (uHCC) in routine clinical practice and is safe even in patients with Child-Pugh (CP)-B grade liver function.

Major finding: After a 9-month median follow-up, median overall survival was 14.9 months (95% CI 13.6-16.3 months) and median progression-free survival was 6.8 months (95% CI 5.2-8.5 months). Tolerability was similar across CP classes, with comparable bevacizumab-related (CP-A, 48%; CP-B, 46%) and atezolizumab-related (CP-A, 53%; CP-B, 40%) adverse event rates of any grade.

Study details: This was a multicenter retrospective study that included 202 adult patients with uHCC and CP-A (76%) or CP-B (24%) cirrhosis who received AtezoBev as the first-line systemic treatment.

Disclosures: The study was funded by the National Institute of Health Research Imperial Biomedical Research Centre, among others. Some authors declared serving as consultants or advisors for or receiving advisory board honoraria, lecture/speaker fees, research grants, or travel/accommodation expenses from various sources.

Source: D'Alessio A et al. Preliminary evidence of safety and tolerability of atezolizumab plus bevacizumab in patients with hepatocellular carcinoma and Child-Pugh A and B cirrhosis: A real-world study. Hepatology. 2022 (Mar 21). Doi: 10.1002/hep.32468

Key clinical point: Postoperative adjuvant stereotactic body radiotherapy (SBRT) on suboptimal resection margin safely and effectively improves disease-free survival (DFS) and prevents local recurrence in microvascular invasion (MVI)-positive hepatocellular carcinoma (HCC).

Major finding: SBRT vs. surgery alone led to significantly higher 1-year (92.1% vs. 76.3%) and 5-year (56.1% vs. 26.3%) DFS rates (P = .005) and similar local recurrence (P = .236) rates. No grade ≥3 adverse events were noted.

Study details: This randomized controlled trial included 76 adult patients with MVI-positive HCC who underwent marginal resection and were randomly assigned to receive postoperative adjuvant SBRT or surgery alone.

Disclosures: The study was funded by the Clinical Science and Technology Innovation Project of Shenkang Hospital Development Center, Shanghai Jiading District Fund, and Shanghai Municipal Health Commission Program, China. No conflicts of interest were reported.

Source: Shi C et al. Adjuvant stereotactic body radiotherapy after marginal resection for hepatocellular carcinoma with microvascular invasion: A randomised controlled trial. Eur J Cancer. 2022;166:176-184 (Mar 16). Doi: 10.1016/j.ejca.2022.02.012

Key clinical point: Postoperative adjuvant stereotactic body radiotherapy (SBRT) on suboptimal resection margin safely and effectively improves disease-free survival (DFS) and prevents local recurrence in microvascular invasion (MVI)-positive hepatocellular carcinoma (HCC).

Major finding: SBRT vs. surgery alone led to significantly higher 1-year (92.1% vs. 76.3%) and 5-year (56.1% vs. 26.3%) DFS rates (P = .005) and similar local recurrence (P = .236) rates. No grade ≥3 adverse events were noted.

Study details: This randomized controlled trial included 76 adult patients with MVI-positive HCC who underwent marginal resection and were randomly assigned to receive postoperative adjuvant SBRT or surgery alone.

Disclosures: The study was funded by the Clinical Science and Technology Innovation Project of Shenkang Hospital Development Center, Shanghai Jiading District Fund, and Shanghai Municipal Health Commission Program, China. No conflicts of interest were reported.

Source: Shi C et al. Adjuvant stereotactic body radiotherapy after marginal resection for hepatocellular carcinoma with microvascular invasion: A randomised controlled trial. Eur J Cancer. 2022;166:176-184 (Mar 16). Doi: 10.1016/j.ejca.2022.02.012

Key clinical point: Postoperative adjuvant stereotactic body radiotherapy (SBRT) on suboptimal resection margin safely and effectively improves disease-free survival (DFS) and prevents local recurrence in microvascular invasion (MVI)-positive hepatocellular carcinoma (HCC).

Major finding: SBRT vs. surgery alone led to significantly higher 1-year (92.1% vs. 76.3%) and 5-year (56.1% vs. 26.3%) DFS rates (P = .005) and similar local recurrence (P = .236) rates. No grade ≥3 adverse events were noted.

Study details: This randomized controlled trial included 76 adult patients with MVI-positive HCC who underwent marginal resection and were randomly assigned to receive postoperative adjuvant SBRT or surgery alone.

Disclosures: The study was funded by the Clinical Science and Technology Innovation Project of Shenkang Hospital Development Center, Shanghai Jiading District Fund, and Shanghai Municipal Health Commission Program, China. No conflicts of interest were reported.

Source: Shi C et al. Adjuvant stereotactic body radiotherapy after marginal resection for hepatocellular carcinoma with microvascular invasion: A randomised controlled trial. Eur J Cancer. 2022;166:176-184 (Mar 16). Doi: 10.1016/j.ejca.2022.02.012