Neurology & Neurosurgery

Paper cutouts of silhouette

Successful autism and ADHD tools go digital

Paper cutouts of silhouette

A team is working to implement a successful, evidence-based online training and tele-support system for the Unstuck and On Target (UOT) program.

A team from Children’s National Hospital, Children’s Hospital Colorado and The Institute for Innovation and Implementation at the University of Maryland, Baltimore is working to implement a successful, evidence-based online training and tele-support system for the Unstuck and On Target (UOT) program. The program is now available for free to any parent or educator who needs it.

What is it?

Since 2020, this team has piloted UOT video training with 293 school-based staff across 230 elementary schools in Colorado and Virginia. The work follows a related PCORI-funded research project, Improving Classroom Behaviors Among Students with Symptoms of Autism Spectrum Disorder or Attention Deficit Hyperactivity Disorder, led by Children’s National and Children’s Colorado researchers. That project demonstrated the effectiveness of UOT at improving the executive functioning – or self-regulation skills including flexible thinking, planning and emotional-control – of school-aged children in Title 1 schools. The training focuses on the executive function of elementary school-aged children with autism spectrum disorder (ASD) or attention deficit hyperactivity disorder (ADHD).

In addition to creating more accessible training for educators, the team created short, free videos highlighting executive functioning tips and tricks that parents can employ at home. These videos, evaluated by 100 parents and revised based on their input, are now available to parents nationwide.

The availability of this training is possible due to a $2 million contract awarded to Children’s Hospital Colorado’s (Children’s Colorado) Pediatric Mental Health Institute and Children’s National by the Patient-Centered Outcomes Research Institute (PCORI) in 2020.

Why it matters

There are many children, including those in low-income or rural settings, that don’t have access to clinics that offer services to support executive functioning skills, such as planning and flexibility, that they need. But all children have access to a school. Now, UOT training is online and accessible so any school with internet access can offer UOT where school staff (including special educators, teachers, paraprofessionals and counselors) can actively teach students how to plan, set goals and be flexible. The team’s next goal is to create a comparable video training for the high school version of UOT.

“These free, accessible and effective tools for improving children’s social-emotional development are building skills that are more important today than ever,” said Lauren Kenworthy, Ph.D., director of the Center for Autism Spectrum Disorders at Children’s National. “The vast majority (96%) of caregivers and educators found these tools useful and relevant. That feedback is a testament to our team’s efforts to make sure these resources were created and validated as usable, approachable and actionable for everyone who needs them.”

More information

For educators – Find resources on Unstuck and On Target, including links to the free trainings, tips and tricks and FAQs. Teachers can also receive continuing education credits (CEUs) for this training.

For parents – Find resources on Unstuck and On Target’s parent training videos

For schools – Add free Unstuck and On Target parent videos to your school district’s relevant websites, landing pages and newsletters.

patient undergoing MRI

Brain tumor team performs first ever LIFU procedure on pediatric DIPG patient

patient undergoing MRI

The ultrasound waves activate the drug selectively within the tumor, causing tumor cell death. Credit: Image provided by Insightec.

A multidisciplinary brain tumor team at Children’s National Hospital successfully performed the first treatment with sonodynamic therapy utilizing low intensity focused ultrasound (LIFU) and 5-aminolevulinic acid (5-ALA) medication on a pediatric patient. The treatment, performed on a 5-year-old child diagnosed with a diffuse intrinsic pontine glioma (DIPG), was done noninvasively through an intact skull. The child was discharged from the hospital one day later.

What happened?

Shortly after announcing the use of LIFU, the brain tumor team at Children’s National treated the patient as part of a cutting-edge trial using LIFU combined with a novel medication.

The ultrasound waves – which are given while the child is asleep through an intact skull and does not require an invasive neurosurgical procedure – activate the drug selectively within the tumor, causing tumor cell death.

“This treatment is currently being trialed in adults diagnosed with recurrent glioblastoma tumors, but has never been attempted in pediatric patients,” said Hasan Syed, M.D., co-director of the Focused Ultrasound Program at Children’s National. “Similar to the adult trial, our protocol involves using a medication that is taken up by tumor cells and then targeting those cells with LIFU to induce tumor cell death, and hopefully leading to tumor control.”

Dr. Syed co-directs the program with Roger Packer, M.D., head of the Brain Tumor Institute, and Lindsay Kilburn, M.D., director of the Experimental Therapeutics Program.

How are we leading the way?

The launch and use of LIFU was possible thanks to the efforts of a multidisciplinary team from various departments that understood if too high a dose of ultrasound was utilized, there could be associated brain swelling and even death.

“Our efforts show great teamwork and a commitment from the hospital and our clinical teams to develop innovative means to treat a tumor that kills 90% of those children afflicted within 18 months of diagnosis,” Dr. Syed said.

The work shows expertise of the brain tumor team, as well as radiology, anesthesiology and intensive care units.

“Despite the risks involved, the use of focused ultrasound is a novel way to try to treat these very deep-seated lesions that have been highly resistant to all forms of therapy and is potentially the greatest breakthrough we’ve had in this disease in the past 50 years,” Dr. Packer said.

What has limited therapy in the past?

DIPGs are deep-seated in critical areas of brain, controlling breathing and heart rate and cannot be removed. The brain has an intrinsic system called a blood brain barrier which blocks drugs from getting to the tumor.

Focused ultrasound is a new way to overcome the brain’s ability to stop the drugs from getting there. It can also be used to activate a drug as it passes through the brain stem.

“We are extremely excited to have taken the first step in developing this novel and non-invasive approach to treating one of our most deadly brain tumors,” Dr. Kilburn said. “This is the first step of numerous steps toward evaluating the many potential uses of LIFU as part of combination therapies to treat children with DIPGs and eventually other pediatric brain tumors.”

Children’s National is partnering with other institutions across the world to perform these studies. But because of the commitment of its team and its expertise, it is the first to use this technique in a child.

“I think we’re in a unique position thanks to the collaborations possible at Children’s National and the expertise of those caring for children with brain tumors,” Dr. Packer added.

Why we’re excited

The Brain Tumor Institute at Children’s National is excited about making this a potential treatment option for DIPG patients, which currently have really no surgical options or alternatives. It’s a way to deliver the ultrasound and therapies in a potentially less toxic way, not requiring surgery.

This trial and subsequently others will give doctors more options for children with DIPGs and other malignant tumors.

illustration of the brain

How the circadian clock could help the brain recover after injury

illustration of the brain

A type of brain cell that can renew itself is regulated by circadian rhythms, providing significant insights into how the body’s internal clock may promote healing after traumatic brain injuries (TBI).

A type of brain cell that can renew itself is regulated by circadian rhythms, providing significant insights into how the body’s internal clock may promote healing after traumatic brain injuries (TBI), according to new research from Children’s National Hospital.

Released in the latest issue of eNeuro, the findings open new avenues of investigation for future TBI therapies. These injuries are currently managed only with supportive care and rehabilitation, rather than targeted drug treatment options. The findings also underscore the importance of addressing circadian disturbances to help injured brains heal.

Many of the body’s cells follow a 24-hour rhythm driven by their genes known as the circadian clock. The Children’s National research team found that a relatively newly discovered type of brain cell ­– known as NG2-glia, or oligodendrocyte precursor cells ­– also follow a circadian rhythm. This cell type is one of the few that continually self-renews throughout adulthood and is notably proliferative in the first week after brain injuries.

“We have found evidence for the role of this well-known molecular pathway – the molecular circadian clock – in regulating the ability for these NG2-glia to proliferate, both at rest and after injury,” said Terry Dean, M.D., Ph.D., critical care specialist at Children’s National and the lead author of the paper. “This will serve as a starting point to further investigate the pathways to controlling cellular regeneration and optimize recovery after injury.”

Sometimes called “the silent epidemic,” TBI afflicts an estimated 69 million people worldwide each year, with injuries ranging from mild concussions to severe injuries that cause mortality or lifelong disability. In the United States alone, approximately 2.8 million people sustain TBI annually, including 630,000 children. TBI is the leading cause of death in people under age 45, and those who survive are often left with persistent physical, cognitive and psychological disabilities.

Yet no targeted therapies exist for TBI, creating a critical need to uncover the mechanisms that could unlock the regeneration of these NG2-glia cells, which are the most common type of brain cell known to proliferate and self-renew in adult brains.

“It is essential for researchers to know that cell renewal is coordinated with the time of day,” said Vittorio Gallo, Ph.D., interim chief academic officer and interim director of the Children’s National Research Institute. “With this knowledge, we can dig deeper into the body’s genetic healing process to understand how cells regulate and regenerate themselves.”

Cancer cells

Searching for the key to treating neuroblastoma tumors in kids

Cancer cells

Jianhua Yang, Ph.D., has dedicated his research to finding the molecular mechanism of neuroblastoma development and is working to develop novel therapeutics.

There continues to be an urgent need to identify novel therapies for childhood cancers. Neuroblastoma (NB) is the most common malignant solid tumor in children and contributes to more than 15% of all pediatric cancer-related deaths. Despite strides made in chemotherapy treatment over the past 30 years, NB largely remains an incurable disease. That’s why Jianhua Yang, Ph.D., associate professor and research faculty at the Center for Cancer and Immunology Research at Children’s National Hospital, has dedicated his research to finding the molecular mechanism of NB development and is working to develop novel therapeutics to target molecules he and his team identify in the lab.

Q: What has driven you to do this research?

A: In order to design better and potentially more effective NB treatment approaches, we must further understand the mechanism that activates NB development. We don’t know what that mechanism is yet, and that’s what we’re working to unlock. I felt with my training in cell biology and immunology, I could use that background to help develop novel therapies.

The research is hard and can often times feel frustrating. But I feel I’m working on something that has the potential to make a huge difference. I tell the researchers I work with that you have to really believe in what we’re doing. We’re doing something very different. Before I moved to D.C. to join Children’s National, I sent a text to a former mentor to let him know I was joining the team here to continue my work. His reply said, “I’ve always had confidence in you,” and it’s that type of encouragement that drives me to keep going.

Q: What is your current focus in this area?

A: Specifically, we’re working on two targets right now:

  1. To define the role and regulation of CaM kinase-like vesicle-associated (CAMKV) in NB development and examining the therapeutic potential of CAMKV kinase inhibition for treating NB in pre-clinical models. We’ve found that CAMKV is highly expressed in NB tumor samples and its kinase activity is required for tumor growth. So, if we knock out this gene, tumor cells will die. We’re studying how it is being activated, and if we can find out what causes it, we can find a way to inhibit its activation. Targeting CAMKV is a novel concept for treating NB. CAMKV kinase inhibitors may serve not only as stand-alone therapies but also as effective adjuncts to current chemotherapeutic regimens treating this aggressive pediatric malignancy.
  2. To define the role and regulation of transmembrane protein 108 (TMEM108) in NB development and examine the therapeutic potential of TMEM108 functional blockade for treating NB in pre-clinical models. Evolutionarily, in human genome it has no other family member, it’s a loner. And if you knock it out in NB tumor cells, tumor cells will die. We’re learning how it functions through our basic research, which is quite difficult. But we’re thinking if we can find the antibody to bind to it and block its function, we could stop the tumor from growing or even cause the tumor to die.

Q: What excites you about doing this work within the Center for Cancer and Immunology Research?

A: At Children’s National, I’ll be able to combine my work with the incredible work in immunotherapy that Drs. Catherine Bollard and Muller Fabbri are doing. I’m excited to be here to have that strong collaboration with their labs to develop new therapies.

In the next 5 years, I feel we’ll be able to identify good blocking antibodies that we can then test combinations of to see how it blocks tumor growth. If we can find ways to combine that antibody therapy with traditional chemotherapy options, we can achieve a real cure for NB.

Learn more about the Center for Cancer and Immunology Research.

Brain illustration

Paving the way toward better understanding and treatment of neonatal brain injuries

Brain illustration

The Gallo Lab’s latest research finds reduced expression of Sirt2 in the white matter of premature human infants and characterizes its role in white matter of the brain in normal conditions and during hypoxia.

Changes in myelination due to diffuse white matter injury are a common consequence of premature birth and hypoxic-ischemic injury due to asphyxia of sick term-born newborns. Hypoxic damage during the neonatal period can lead to motor disabilities and cognitive deficits with long-term consequences, including cerebral palsy, intellectual disability or epilepsy, which are often due to cellular and functional abnormalities.

The Gallo Lab, within the Center for Neuroscience Research at Children’s National Hospital, is focused on studying postnatal neural development and the impact of injury and disease on development and regeneration of neurons and glia. Their latest research, published in Nature Communications, finds reduced expression of Sirt2 in the white matter of premature human infants (born earlier than 32 weeks of gestation) and characterizes its role in white matter of the brain in normal conditions as well as during hypoxia.

What it means

The lab previously identified Sirt1 as important for the proliferative regenerative response of oligodendrocyte progenitor cells in response to chronic neonatal hypoxia. This new study characterizes the function of Sirt2 and finds that it acts as a critical promoter of oligodendrocyte differentiation during both normal brain development and after hypoxia.

It’s likely this reduced expression of Sirt2 contributes to the arrest in oligodendrocyte maturation and myelination failure seen in extremely low gestational age neonates. Therefore, targeting Sirt2 may be an opportunity to capture the early and small window of opportunity for therapeutic intervention.

How this moves the field forward

Sirtuins have been shown to play crucial therapeutic roles in various diseases, including aging, neurodegenerative disorders, cardiovascular disease and cancer. Identifying Sirt2 as a major regulator of white matter development and recovery and increasing the understanding of its protein and genomic interactions opens new avenues for Sirt2 as a therapeutic target for white matter injury in premature babies.

Why we’re excited

Interestingly, the team found that overexpression of Sirt2 in oligodendrocyte progenitor cells, but not mature oligodendrocytes, restores oligodendrocyte populations after hypoxia through enhanced proliferation and protection from apoptosis. This is exciting because:

  • It tells us that Sirt2 expression is very important for the transition from progenitor to differentiated oligodendrocyte.
  • It’s the first report, to the team’s knowledge, of Sirt2 regulating cell survival of oligodendrocytes.

Read more in Nature Communications

Mother helping son check blood sugar levels

Supporting parents and children through diabetes diagnosis

Mother helping son check blood sugar levels

Behavioral intervention can improve parents’ mood following their child’s diabetes diagnosis.

Results from a new study show that behavioral intervention improved parents’ mood following young children’s Type 1 diabetes diagnosis.

The study evaluated First STEPS, a stepped-care behavioral intervention designed to support parents’ psychosocial functioning and promote children’s glycemic outcomes. Results indicated likely benefits of parent coach support, supplemented by intervention intensifications, including behavioral intervention and diabetes education.

“We found that parent coaches, or parents of slightly older children with Type 1 diabetes who were trained in offering peer support, were helpful in reducing parent depressive symptoms up to one year and a half following diagnosis for parents in the stepped care group,” says Randi Streisand, Ph.D., C.D.C.E.S., Psychology and Behavioral Health division chief at Children’s National Hospital and senior author of the study. “The second study target, child glycemic control, was not significantly different between the two groups.”

What’s been the hold-up in the field?

There are unique challenges facing families of young children with Type 1 diabetes. However, typical care and management guidelines are not specific to young children.

“Many parents of children diagnosed with diabetes experience distress and symptoms of depression, yet parents are not routinely screened during clinic visits,” Dr. Streisand says. “Further, there are many barriers to mental health support.”

Moving the field forward

Findings also highlighted the potential for training lay people who have a shared lived experience (parent coaches), which could be incorporated into clinical programs.

Most behavioral interventions use behavioral health experts. The study’s experts demonstrated significant outcomes in parent mood by using parent coaches.

“The goal would be to incorporate parent coach programs into the clinic setting, to either offer the support to all families at the time of diagnosis or to screen families and provide support to those in need,” Dr. Streisand adds.

The authors affirm this model has high potential for patient engagement. Additionally, results showed that incorporating targeted behavioral support for intensive diabetes treatment may maximize intervention impact.

Other Children’s National authors include: Carrie Tully, Ph.D.; Christine Wang, Ph.D.; Lauren Clary, Ph.D.; Fran Cogen, M.D.; John Barber and Celia Henderson.

You can read the full study First STEPS: Primary Outcomes of a Randomized, Stepped-Care Behavioral Clinical Trial for Parents of Young Children With New-Onset Type 1 Diabetes in Diabetes Care.

Illustration of brain and brainwaves

Risk factors for pharmacoresistant pediatric epilepsy

Illustration of brain and brainwaves

New study evaluates risk factors for the timing and development of drug-resistant pediatric epilepsy.

Focal cortical dysplasia (FCD) is the most common cause of surgically-treatable epilepsy in children. In a new study published in Neurology, researchers evaluated 143 children with confirmed FCD risk factors for the timing and development of pharmacoresistant epilepsy.

What this means

The current definition of pharmacoresistance requires failure of two appropriately-dosed and selected antiseizure medications before being able to be considered for epilepsy surgery.

“We found that the failure of just one antiseizure medication is associated with an enormous increased incidence and earlier development of pharmacoresistance,” says Nathan Cohen, M.D., neurologist at Children’s National and lead author of the study. “Our data supports the redefinition of pharmacoresistant epilepsy to the failure of just one antiseizure medication in this population, which would potentially allow these patients to benefit from earlier curative surgery.”

Why it matters

The findings showed that in children with FCD the failure of just one antiseizure medication is associated with an enormous risk and earlier incidence of pharmacoresistance. Therefore, the authors advocate for its redefinition in FCD-related epilepsy to the failure of just one antiseizure medication.

“This will allow children to be considered much earlier for potentially curative epilepsy surgery,” adds Dr. Cohen. “We find that the majority of FCD patients develop epilepsy and that the majority of those with epilepsy develop pharmacoresistance.”

In a multivariate analysis, the authors show that the FCD cortical lobar location, pathologic subtype, and age of seizure onset are not important factors in the development of pharmacoresistance.

What’s next

This data supports operational re-definition of pharmacoresistance for surgical planning in FCD-related epilepsy to the failure of one antiseizure medication, and support early, potentially curative surgery to improve outcomes in this patient population.

You can read the full study, Prevalence and Risk Factors for Pharmacoresistance in Children With Focal Cortical Dysplasia–Related Epilepsy, in Neurology.

Digital background depicting innovative technologies in (AI) artificial systems, neural interfaces and internet machine learning technologies

AI algorithm that detects brain abnormalities could help cure epilepsy

Digital background depicting innovative technologies in (AI) artificial systems, neural interfaces and internet machine learning technologies

A new AI algorithm can detect subtle brain abnormalities that cause epileptic seizures.

An artificial intelligence (AI) algorithm that can detect subtle brain abnormalities that cause epileptic seizures has been developed by a UCL-led team of international researchers, including Children’s National Hospital.

To do this, the team quantified features from MRI scans, such as how thick or folded the brain was at nearly 300,000 locations in each case.

They then trained the AI algorithm using examples labelled by expert radiologists as either a healthy brain or one with focal cortical dysplasia (FCD) based on their patterns and features.

The results, published in Brain, showed that in the main cohort of 538 patients, the algorithm was able to detect the FCD in 67% of cases.

“We put an emphasis on creating an AI algorithm that was interpretable and could help doctors make decisions. Showing doctors how the Multicentre Epilepsy Lesion Detection project (MELD) algorithm made its predictions was an essential part of that process,” said Mathilde Ripart, research assistant at UCL and the study’s co-first author.

Around 1% of the population have epilepsy and, of these, 20-30% do not respond to medications.

“We are excited to collaborate with MELD on ways to improve the treatment of pharmacoresistant epilepsy,” said Nathan Cohen, M.D., neurologist at Children’s National Hospital and co-author of the study. “This advanced imaging platform is open source and demonstrates the benefit of team science at the broadest scale.”

In children who have had surgery to control their epilepsy, FCD is the most common cause, and in adults it is the third most common cause.

Additionally, of patients who have epilepsy that have an abnormality in the brain that cannot be found on MRI scans, FCD is the most common cause.

You can read the full UCL press release here.

Tired student studying online on laptop at home

Headache disorders and mental health worsened during pandemic

Tired student studying online on laptop at home

Children’s headaches and mental health worsened during pandemic, new study finds.

Since the beginning of the COVID-19 pandemic, many pediatric patients who suffer from headaches have experienced more frequent headaches and worsening anxiety and mood, and a new study finds links to stress, decreased physical activity and increased screen time.

The findings, published in the Journal of Child Neurology, showed that elevated stress associated with disruptions to daily life, social distancing practices and anxiety about the threat of illness to oneself and others brought on by the pandemic impacted the quality of life for kids with headache disorders.

“These findings are really impactful to me as a physician and a parent. It is important we gain a better understanding about how stress and changes in routine affect children’s wellbeing and mood,” says lead author Marc DiSabella, D.O., director of the Headache Program at Children’s National Hospital. “Things like moving to a virtual environment may have resulted in feelings of isolation and anxiety for kids, and increased screen time may have played a role in more frequent headaches.”

Migraine and other headache disorders are exceedingly common in adolescents and children. For this study, 107 patients completed a questionnaire from summer 2020 to winter 2021 examining changes in headache characteristics and lifestyle factors since the start of the pandemic. The survey found:

  • Pre-pandemic, 60% of patients reported having headaches less than 15 days of the month. After the start of the pandemic, that number dropped to 50%.
  • Patients reporting constant daily headaches went from 22% pre-pandemic to 36% after the start of the pandemic.
  • 49% of patients reported their headaches had worsened since the onset of the pandemic.
  • 54% of patients reported that their physical activity levels decreased because of the pandemic.
  • When asked about screen use during the pandemic, 61% of patients reported using screens for more than six hours a day.

The authors of the study note that whether or not increased screen time worsens headaches has not yet been clearly established; however, patients and families routinely cite screen use as a headache trigger. Lack of physical exercise is also often cited as a migraine trigger.

“Having a headache every day, all the time, with no break in sight, is really frustrating to children and their parents,” Dr. DiSabella adds. “They just want to be a normal child, yet have no control over when the pain increases, and they suddenly are unable to do simple activities like reading a book or seeing friends, which adds to the uncertainty of their future.”

Participants also reported worsened anxiety, mood and workload. According to the authors, this is likely to affect headache patients given their elevated rates of anxiety and depression.

“We already know that patients with headache disorders have disproportionately high rates of mood complaints, including anxious and depressive symptoms,” Dr. DiSabella says. “The fact that our patients reported this worsened during quarantine is an additional stress on their already complex lives, managing pain, school and extra-curricular activities.”

While the study is limited by sample size and observational design, future population-based studies will further explain the impact of this pandemic on kids who suffer from headaches. In the interim, Dr. DiSabella recommends parents talk with their children about how the pandemic has impacted their headaches and mood. He also recommends offering children help, either at home or with a professional trained in child psychology.

William Gaillard

William D. Gaillard, M.D., named as first endowed Professor of Epilepsy and Neurophysiology

William GaillardChildren’s National Hospital named William D. Gaillard, M.D., as the hospital’s first endowed Professor of Epilepsy and Neurophysiology.

Dr. Gaillard serves as Division Chief of Child Neurology, Epilepsy and Neurophysiology, Director of the Comprehensive Pediatric Epilepsy Program and Associate Director of the Center for Neuroscience Research at Children’s National Hospital. He also is Professor of Pediatrics and Neurology at the George Washington University School of Medicine, Professor of Neurology at Georgetown University and Adjunct Professor of Hearing and Speech Sciences at the University of Maryland, College Park.

About the award

Dr. Gaillard joins a distinguished group of 42 Children’s National physicians and scientists who are endowed chairs. Professorships at Children’s National advance groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These prestigious appointments carry honor and reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

Dr. Gaillard is an internationally recognized expert in advanced structural and functional imaging, with a focus on examining the effects of epilepsy on brain structure and function. His pioneering work with functional imaging has changed clinical practice for patients with epilepsy, impacted structural and functional imaging methods and provided fundamental insights on brain plasticity. Dr. Gaillard’s research is improving patient outcomes and the lives of children with epilepsy.

Donors Betsy Williams and Tom Moore, through their vision and generosity, will ensure that Dr. Gaillard and future holders of this professorship can launch bold, new initiatives to rapidly advance the field of pediatric epilepsy and neurophysiology, elevate our leadership and improve the lifetimes of children with epilepsy and seizure disorders.

“This Professorship is a great honor and opportunity for our team and for the future of pediatric epilepsy medicine,” says Dr. Gaillard. “Thanks to Betsy, Tom and the Hess Foundation’s generosity, we can continue to invest in people and programs that show promise. We can have the flexibility to respond to the needs of our current patient families and prepare for those that will need us tomorrow. We are forever grateful.”

About the donors

Betsy and Tom are parents, members of the Epilepsy Council at Children’s National and long-time supporters. To acknowledge their gratitude and respect for Dr. Gaillard and the comprehensive team he’s built, the Professorship will be named for Dr. Gaillard in the future.

boy getting vaccinated

Adolescents with ADHD more hesitant to get COVID-19 vaccine

boy getting vaccinatedAdolescents with attention-deficit/hyperactivity disorder (ADHD) report greater hesitancy and less confidence in COVID-19 vaccine safety compared to adolescents without ADHD, a new study finds.

For all adolescents in the study, those who identified as Black or Latino — and came from families with lower income levels — were more likely to be vaccine hesitant and report lower confidence in the safety of COVID-19 vaccines.

Whereas greater COVID-19 concerns, compliance to social distancing guidelines, media use and perceived negative impact of COVID-19 on relationships was associated with greater vaccination willingness.

The study, led by Melissa Dvorsky, Ph.D., director of ADHD & Learning Differences Program at Children’s National Hospital, also highlighted that:

  • Adolescents with ADHD who engage in large gatherings indoors are at greater risk for vaccine hesitancy.
  • Interventions should target social-cognitive processes for adolescent vaccination.

“Adolescents with ADHD being more vaccine hesitant is perhaps in part due to core risk mechanisms associated with ADHD, likely impacting planning, motivation and execution of vaccination, adolescents’ risk appraisal, and perceived susceptibility to COVID-19,” Dr. Dvorsky said. “Our study also found key social mechanisms predicted increased vaccine acceptance and uptake, and these factors should be leveraged in ongoing initiatives addressing vaccine uptake among teens.”

Findings have important implications for health and mental health providers and educational strategies aimed at promoting COVID-19 vaccinations in adolescents.

Earlier this month, Mayor Muriel Bowser and DC Health announced amplified efforts to encourage families to vaccinate youth ahead of the upcoming 2022-2023 school year, sending a message that students must get caught up on vaccinations over the summer. In addition to expanding access to vaccination services, a concerted effort, Dvorsky added, is needed to increase trust, confidence, motivation and social relevance among adolescents. This is especially true for those with ADHD and from lower socio-economic backgrounds.

“As health and mental health care providers, we are uniquely positioned to offer effective communication using strong, presumptive language with all adolescents in our community to address vaccine hesitancy. Adolescents with ADHD, in particular, can benefit from frequent behavioral ‘nudges’ (such as prompts or reminders, automatic appointments) and social/motivational strategies (such as social network interventions, peer-delivered approaches, motivational interviewing) to increase vaccine uptake.”

It’s important to note that research addressing adolescent COVID-19 vaccination willingness and readiness remains scarce.

The study included 196 adolescents (87 male) ages 16-18 from two sites in the Southeastern and Midwestern United States. Participants were high school students in 11th and 12th grade during the 2020-2021 school year. Participants came from a range of socioeconomic backgrounds, with 21% of families falling below the 2019 U.S. median household income ($68,703). Approximately half of the participants were comprehensively diagnosed with ADHD prior to COVID-19.

US News Badges

Children’s National named to U.S. News & World Report’s Best Children’s Hospitals Honor Roll

US News BadgesChildren’s National Hospital in Washington, D.C., was ranked No. 5 nationally in the U.S. News & World Report 2022-23 Best Children’s Hospitals annual rankings. This marks the sixth straight year Children’s National has made the list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the sixth year in a row.

For the twelfth straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.

“In any year, it would take an incredible team to earn a number 5 in the nation ranking. This year, our team performed at the very highest levels, all while facing incredible challenges, including the ongoing pandemic, national workforce shortages and enormous stress,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “I could not be prouder of every member of our organization who maintained a commitment to our mission. Through their resilience, Children’s National continued to provide outstanding care families.”

“Choosing the right hospital for a sick child is a critical decision for many parents,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The seven Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other three specialties ranked among the top 50 were cardiology and heart surgery, gastroenterology and gastro-intestinal surgery, and urology.

illustration of the brain

LIFU successfully delivers targeted therapies past the blood-brain barrier

illustration of the brain

LIFU offers doctors the first opportunity to open the blood-brain barrier and treat the entire malignant brain tumor.

Children’s National Hospital will leverage low-intensity focused ultrasound (LIFU) to deliver therapy directly to a child’s high-grade glioma. The approach offers doctors the first opportunity to open the blood-brain barrier and treat the entire malignant brain tumor.

Children’s National will be the first hospital in the U.S. to treat high-grade pediatric brain tumors with LIFU to disrupt the blood-brain barrier. Crossing it has been a major hurdle for effective therapy. The barrier, a network of blood vessels and tissue, prevents harmful substances from reaching the brain but also stops molecular targeted therapy and immunotherapy from getting into the tumor site and staying there.

“LIFU gives us a way to potentially transiently open up the barrier, so we can deliver novel therapy directly to the tumor and improve the likelihood of survival,” said Roger Packer, M.D., senior vice president of the Center for Neurosciences and Behavioral Medicine at Children’s National. “It is the greatest breakthrough we’ve potentially had in the past 50 years or more for the management of these tumors. We made great strides in our understanding of molecular genetics and the molecular drivers of tumors, but we have not yet translated that knowledge into better therapies; this may be our most effective mechanism to overcome the barrier.”

In 2020, Children’s National was recognized as the first worldwide Center of Excellence by the Focused Ultrasound Foundation.

Focused ultrasound (FUS) is a non-invasive therapeutic technology with the potential to transform the treatment of many medical disorders by using ultrasonic thermal energy to specifically target tissue deep in the body. The technology can treat without incisions or the need of radiation.

How it works

Doctors at Children’s National will be using LIFU in two different types of procedures:

  • 5-ALA: Doctors will give the patient 5-aminolevulinic acid (5-ALA) with the LIFU treatment. 5-ALA enters rapidly dividing cells and is activated by the ultrasound to a state where it kills the dividing cells of the tumor. The surrounding normal brain cells around the tumor are not dividing, so they do not take up the 5-ALA and are left unharmed after ultrasound therapy.
  • Microbubbles: While receiving different doses of LIFU over a one- to two-hour period, the patient is given “microbubbles,” which are widely used in medical imaging and as carriers for targeted drug delivery. These microbubbles bounce around against the walls like seltzer, opening the blood vessels and transiently opening that space.

Both studies are the first in the world for pediatric gliomas of the brain stem, allowing experts to treat patients 4-6 weeks after radiotherapy. The patient then receives medication orally or intravenously as it passes through the bloodstream. It does not go at high levels anywhere within the brain except where the blood-brain-barrier was opened, allowing oral medication or immune therapies to rush into the tumor.

The launch of this program comes a few months after the hospital successfully performed the first-ever high-intensity focused ultrasound surgery on a pediatric patient with neurofibromatosis.

Watch this video to learn more.

Dr. Limperopoulos talks to a mom

Pandemic-related stressors in pregnant women affect fetal brain development

Dr. Limperopoulos talks to a mom

Dr. Catherine Limperopoulos walking with a mom.

Prolonged levels of stress and depression during the COVID-19 pandemic contributed to altering key features of fetal brain development — even if the mother was not infected by the virus. This is what a study published in Communications Medicine suggests after following more than 200 pregnant women. The study, led by Children’s National Hospital experts, emphasized the need for more scientific inquiry to shed light on the long-term neurodevelopmental consequences of their findings and COVID-19 exposures on fetal brain development.

“Understanding how contemporary stressors may influence fetal brain development during pregnancy has major implications for basic science and informing public policy initiatives,” said Catherine Limperopoulos, Ph.D., chief and director of the Developing Brain Institute at Children’s National and senior author of the study. “With this work, we are able to show there’s a problem, it’s happening prenatally, and we can use this model to start exploring how we can reduce stress in moms and support unborn babies.”

To better understand the effects of environmental exposures on the fetus during pregnancy, further confirmation of the team’s latest findings is needed by ruling out other possibilities, such as maternal nutrition, financial security and genetic factors.

The psychosocial impact of COVID-19 on fetal brain development remains vastly understudied. The neurologic underpinnings of fetal development that turn into psycho-behavioral disorders later in life, including bipolar disorder, mood disorder or anxiety disorder, remain complex and difficult to explain.

Among the 202 participants from the Washington D.C. metropolitan area, 137 were part of the pre-pandemic cohort and 65 were part of the pandemic cohort.

Through advanced MRI imaging techniques and reconstruction of high-resolution 3D brain models, the researchers found a reduction of fetal white matter, hippocampal and cerebellar volumes and delayed brain gyrification in COVID-19 pandemic-era pregnancies. Validated maternal stress, anxiety and depression scales were also used to compare the scores between the two cohorts.

This study builds upon previous work from the Developing Brain Institute led by Limperopoulos, which discovered that anxiety in pregnant women appears to affect the brain development of their babies. Her team also found that maternal mental health, even in high socioeconomic status, alters the structure and biochemistry of the developing fetal brain, emphasizing the importance of mental health support for pregnant women.

“We’re looking at modifiable conditions,” said Limperopoulos. “What’s clear is the next frontier is intervening early to see how we can prevent or reduce stress in the mom’s current setting.”

crawling baby

Gene-targeting may help prevent or recover neonatal brain injuries

crawling baby

The findings of a new pre-clinical study published in The Journal of Neuroscience are helping pave the way toward better understanding, prevention and recovery of neonatal brain injuries.

The findings of a new pre-clinical study published in The Journal of Neuroscience are helping pave the way toward better understanding, prevention and recovery of neonatal brain injuries. During pregnancy, the fetus normally grows in low oxygen conditions. When babies are born preterm, there is an abrupt change into a high oxygen environment which may be higher than the baby can tolerate. These preterm babies often need support to breathe because their lungs are immature. If the oxygen they receive is too high, oxygen-free radicals can form and cause cell death.

Premature infants have underdeveloped antioxidant defenses that prevent or delay some types of cell damage under normal conditions. In a high oxygen environment, these underdeveloped defenses cannot fully protect against oxidative stress, damaging different brain regions without available treatments or preventative measures.

“I am thrilled that we identified a defect in a specific cell population in the hippocampus for memory development,” said Vittorio Gallo, Ph.D., interim chief academic officer and interim director of the Children’s National Research Institute, and principal investigator for the District of Columbia Intellectual and Developmental Disabilities Research Center. “I did not think we would be able to do it at a refined level, identifying cell populations sensitive to oxidative stress and its underlying signaling pathway and molecular mechanism.”

Vittorio Gallo

“I am thrilled that we identified a defect in a specific cell population in the hippocampus for memory development,” said Vittorio Gallo, Ph.D.

Children’s National Hospital experts found that oxidative stress over-activates a glucose metabolism enzyme, GSK3β, altering hippocampal interneuron development and impairing learning and memory, according to the pre-clinical study. The researchers also inhibited GSK3β in hippocampal interneurons, reversing these cellular and cognitive deficits.

The role of oxidative stress in the developing hippocampus, as well as GSK3β involvement in oxidative stress-induced neurodevelopmental disorders and cognitive deficits, have both been unexplored until now. Goldstein et al. suggest the study paves the way for the field as a viable approach to maximize functional recovery after neonatal brain injury.

To better understand the mechanisms underlying neonatal brain injury, the researchers mimicked the brain injury by inducing high oxygen levels in a pre-clinical model for a short time. This quest led to unlocking the underpinnings of the cognitive deficits, including the pathophysiology and molecular mechanisms of oxidative damage in the developing hippocampus.

Once they identified what caused cellular damage, the researchers used a gene-targeted approach to reduce GSK3β levels in POMC-expressing cells or Gad2-expressing interneurons. By regulating the levels of GSK3β in interneurons ⁠— but not in POMC-expressing cells — inhibitory neurotransmission was significantly improved and memory deficits due to high oxygen levels were reversed.

caspase molecule

Caspases may link brain cell degeneration and cardiac surgery

caspase molecule

The review summarizes both the known physiological roles of caspases as well as some of the well-characterized neurotoxic effects of anesthetics in pre-clinical models.

A review article in the journal Cell Press: Trends in Neuroscience outlines the wide variety of cellular signaling roles for caspase proteins — a type of cellular enzyme best known for its documented role in the natural process of cell death (apoptosis). The authors, including Nemanja Saric, Ph.D., Kazue Hashimoto-Torii, Ph.D., and Nobuyuki Ishibashi, M.D., all from Children’s National Research Institute, pay particular attention to what the scientific literature shows about caspases’ non-apoptotic roles in the neurons specifically. They also highlight research showing how, when activated during a cardiac surgery with anesthesia and cardiopulmonary bypass, these enzymes may contribute to the degeneration of brain cells seen in young children who undergo heart surgery for critical congenital heart defects (CHDs).

Why it matters

The review summarizes both the known physiological roles of caspases as well as some of the well-characterized neurotoxic effects of anesthetics in pre-clinical models.

The authors propose that these non-apoptotic activities of caspases may be behind some of the adverse effects on the developing brain related to cardiac surgery and anesthesia. Those adverse effects are known to increase risk of behavioral impairments in children with congenital heart disease who underwent cardiac surgery with both anesthesia and cardiopulmonary bypass at a very young age.

This work is the first to propose a possible link between developmental anesthesia neurotoxicity and caspase-dependent cellular responses.

The patient benefit

Better understanding of the time and dose-dependent effects of general anesthetics on the developing brain, particularly in children who have genetic predispositions to conditions such as CHDs, will help researchers understand their role (if any) in behavioral problems often encountered by these patients after surgery.

If found to be a contributing factor, perhaps new therapies to mitigate this caspase activity might be explored to alleviate some of these adverse effects on the developing brain.

What’s next?

The authors hope to stimulate more in-depth research into caspase signaling events, particularly related to how these signaling events change when an anesthetic is introduced. Deeper understanding of how anesthetics impact caspase activation in the developing brain will allow for better assessments of the risk for children who need major surgery early in life.

Children’s National leads the way

Children’s National Hospital leads studies funded by the U.S. Department of Defense to better understand how these other roles of caspases, which until now have not been well-documented, may contribute to brain cell degeneration when activated by prolonged anesthesia and cardiopulmonary bypass during cardiac surgery for congenital heart disease.

pregnant woman by window

Stress during pregnancy may hinder cognitive development

pregnant woman by window

This is the first study to shed light on an important link between altered in-utero fetal brain development and the long-term cognitive development consequences for fetuses exposed to high levels of toxic stress during pregnancy.

Women’s elevated anxiety, depression and stress during pregnancy altered key features of the fetal brain, which subsequently decreased their offspring’s cognitive development at 18 months. These changes also increased internalizing and dysregulation behaviors, according to a new study by Children’s National Hospital published in JAMA Network Open. Researchers followed a cohort of 97 pregnant women and their babies. The findings further suggest that persistent psychological distress after the baby is born may influence the parent-child interaction and infant self-regulation.

This is the first study to shed light on an important link between altered in-utero fetal brain development and the long-term cognitive development consequences for fetuses exposed to high levels of toxic stress during pregnancy. While in the womb, the researchers observed changes in the sulcal depth and left hippocampal volume, which could explain the neurodevelopment issues seen after birth. Once they grow into toddlers, these children may experience persistent social-emotional problems and have difficulty establishing positive relationships with others, including their mothers. To further confirm this, future studies with a larger sample size that reflect more regions and populations are needed.

“By identifying the pregnant women with elevated levels of psychological distress, clinicians could recognize those babies who are at risk for later neurodevelopmental impairment and might benefit from early, targeted interventions,” said Catherine Limperopoulos, Ph.D., chief and director of the Developing Brain Institute at Children’s National and senior author of the study.

Catherine Limperopoulos

“By identifying the pregnant women with elevated levels of psychological distress, clinicians could recognize those babies who are at risk for later neurodevelopmental impairment and might benefit from early, targeted interventions,” said Catherine Limperopoulos, Ph.D., chief and director of the Developing Brain Institute at Children’s National and senior author of the study.

Regardless of their socioeconomic status, about one of every four pregnant women suffers from stress-related symptoms, the most common pregnancy complication. The relationship between altered fetal brain development, prenatal maternal psychological distress and long-term neurodevelopmental outcomes remain unknown. Studying in utero fetal brain development poses challenges due to fetal and maternal movements, imaging technology, signal-to-noise ratio issues and changes in brain growth.

All pregnant participants were healthy, most had some level of education and were employed. To quantify prenatal maternal stress, anxiety and depression, the researchers used validated self-reported questionnaires. Fetal brain volumes and cortical folding were measured from three-dimensional reconstructed images derived from MRI scans. Fetal brain creatine and choline were quantified using proton magnetic resonance spectroscopy. The 18-month child neurodevelopment was measured using validated scales and assessments.

This study builds upon previous work from the Developing Brain Institute led by Limperopoulos, which discovered that anxiety in pregnant women appears to affect the brain development of their babies. Her team also found that maternal mental health, even for women with high socioeconomic status, alters the structure and biochemistry of the developing fetal brain. The growing evidence underscores the importance of mental health support for pregnant women.

“We’re looking at shifting the health care paradigm and adopting these changes more broadly to better support moms,” said Limperopoulos. “What’s clear is early interventions could help moms reduce their stress, which can positively impact their symptoms and thereby their baby long after birth.”

model of the brain

Treating newborns with hemimegalencephaly by inducing strokes

model of the brain

Experts at Children’s National Hospital have pioneered a novel approach using controlled strokes to stop seizures and improve neurodevelopmental outcomes in newborns under three months born with hemimegalencephaly (HME). They now consider it their new standard of care for babies in this age group with HME and refractory epilepsy.

Asking a physician to induce strokes in newborns is asking her to do something contrary to her training. But over the past eight years, experts at Children’s National Hospital have pioneered a novel approach using controlled strokes to stop seizures and improve neurodevelopmental outcomes in newborns under three months born with hemimegalencephaly (HME). They now consider it their new standard of care for babies in this age group with HME and refractory epilepsy.

“We have demonstrated the ability to intervene and stop the intractable seizures during a critical time of neurodevelopment in which no other effective medical or surgical option exists. That is extremely rewarding,” said Monica Pearl, M.D., director of the Neurointerventional Radiology Program at Children’s National. Children’s National is the only center in the world currently offering this treatment. A multi-disciplinary team led by Dr. Pearl; Taeun Chang, M.D., director of the Neonatal Neurology and Neonatal Neurocritical Care Program; neurophysiologist and neonatal neurologist Tammy Tsuchida, M.D., Ph.D.; and other experts has now successfully treated seven patients using this minimally-invasive approach.

“We want patients and providers to understand this is a better alternative to a delayed hemispherectomy, the standard of care currently offered to newborns with HME,” said Dr. Chang.

The best treatment for newborns with hemimegalencephaly

HME, a rare congenital condition occurring in a handful of newborns each year, is characterized by abnormal growth and enlargement of half of the brain which leads to intractable seizures. The seizures often result in severe cognitive delays and hemiparesis. The standard treatment is an anatomic hemispherectomy — surgical removal of the affected half of the brain, allowing the remaining half of the brain to develop and function without constant seizures.

Such a large and complex surgery poses serious risks for infants younger than three months, leaving doctors with the difficult choice to delay surgery until these newborns grow bigger and stronger, even as they are experiencing seizures. These persistent seizures compromise the development of the healthy half of the brain. One study reports as much as a drop of 10 to 20 IQ points with each month’s delay in surgical hemispherectomy.

“I was willing to consider performing these procedures because there is a clear, unmet medical need and these babies are in dire circumstances,” Dr. Pearl said. “Waiting for curative hemispherectomy means more than just lost time; uncontrolled seizures and anti-seizure medications have detrimental effects on the ‘normal,’ unaffected parts of the brain. We needed a better option for these patients.” Dr. Pearl said that complete embolization of the affected hemisphere as both primary and definitive treatment had never been described. They could only find one example in the literature – a paper from 1995 – suggesting embolization as an adjunct to surgery, and nothing suggesting it as a primary modality.

About the care received

Dr. Pearl is one of only a handful of dedicated pediatric neurointerventionalists across the country with neurovascular expertise in people of all ages, in particular neonates and young infants. For these procedures to be performed safely, the neurointerventionalist must be proficient in obtaining femoral arterial access and navigating small caliber cervicocerebral blood vessels that are less than one millimeter in diameter.

Additionally, one needs a neonatal neurocritical care service and NICU that can medically manage large strokes and their potential complications in newborns. Dr. Chang has developed a specialized protocol based on decades of managing strokes and other acute brain injuries in newborns. She created the neonatal neurocritical care service at Children’s National, the only one in the region and the largest in the world.

“Our teams are fortunate in that we each respectively have extensive prior experience in treating and managing neonates and very young infants for various cerebrovascular disorders,” Dr. Pearl said. “We relied on this collective experience to make this hemispheric embolization pathway possible.”

How it happens

To perform the embolizations, Dr. Chang and her team first optimizes control of the seizures using medications. Dr. Pearl places a sheath in the femoral artery using ultrasound guidance – a delicate task in a neonate whose femoral artery diameter is only two to three millimeters. She then navigates a catheter up the aorta and selects the targeted carotid artery using radiographic guidance. What follows is a set of intricate navigations to direct the microcatheter through small blood vessels in the brain, often less than one millimeter.

Using x-ray guidance, Dr. Pearl injects contrast through the microcatheter to visualize the arterial anatomy and advance the microcatheter into position for embolization. She uses glue that hardens when exposed to blood, blocking off the blood supply to the seizure-inducing areas. The process is repeated until the blood supply to the entire affected hemisphere is occluded. Meanwhile, Dr. Chang and her team monitor the brain’s electrical activity using an electroencephalogram (EEG) to watch how the brain responds to each stroke. The surgical epilepsy, neonatal neurocritical care and neonatology teams are all in constant communication throughout the procedure.

Together, they have to contend with the same symptoms patients have immediately following a stroke, most notably brain swelling that can cause bleeding and herniation. The resultant brain swelling is complicated further by the already enlarged hemisphere of the brain. Using neuroprotective strategies learned from treating over a thousand newborns with perinatal brain injury, Dr. Chang and her team and the NICU coordinate to minimize brain swelling and protect the healthy half of the brain by tightly controlling the brain temperature, glucose, sodium levels, and blood pressure. Over the course of a few weeks, Dr. Pearl performs three to four embolization sessions to halt blood supply to the seizing half of the brain.

“The risks of intracranial vasospasm and hemorrhage during embolization are higher in this distinct group of patients compared to other neonates requiring embolization, such as in vein of Galen malformations. These events must be controlled immediately to prevent complications and I know I only have seconds to react,” Dr. Pearl said.

“Here, we have the cultivation of brain-centric neonatal care, a large level IV tertiary NICU with expertise in keeping critically ill babies alive and rare pediatric neurologic subspecialists like Dr. Pearl and myself. All of this is what makes this level of innovation possible,” Dr. Chang said. Now, they wish this minimally invasive approach to be available to all newborns with HME and refractory epilepsy.

“This is not a fluke. This is not a one-time thing. Our team at Children’s National has been perfecting this method for close to a decade,” Dr. Chang said. As for proof, her answer is clear.

mother and daughter embracing

Understanding end-of-life treatment preferences for adolescents

mother and daughter embracing

FACE-TC effectively increases communication between adolescents with cancer and their families about the patients’ preferences.

Talking about death and dying is taboo. Some families believe it is their role alone to make end-of-life healthcare decisions or they may believe pediatric advance care planning is against their religion.

In a recent trial, Maureen Lyon, Ph.D., a clinical health psychologist at Children’s National Hospital and lead author of the study, analyzed the value of high-quality pediatric advance care planning and how this enabled families to know their adolescents’ end-of-life treatment preferences.

This is the first fully powered randomized controlled trial to focus on adolescents with cancer and their engagement with their families in pediatric advance care planning conversations.

What this means

Some physicians believe it is not their role to discuss the “what ifs.” Others report that they do not have the training or time to do so. As a result, in clinical practice, adolescents living with a serious illness rarely have documented advance care plans. The default is to provide intensive treatments that potentially increase suffering.

“Despite cancer being the leading cause of disease-related death in adolescents, conversations about goals of care and documentation of end-of-life care and treatment preferences for adolescents with cancer are not a routine and standard part of care,” Dr. Lyon said.

Why it matters

Family-centered advance care planning for teens with cancer (FACE-TC) effectively increases communication between adolescents with cancer and their families about the patients’ end-of-life preferences. This meets the first challenge of pediatric advance care planning – knowledge of patient’s preferences.

This low-tech intervention commits to more deeply respecting adolescents with cancer, integrating them into health care decision-making and giving them some control in a low control situation.

The patient and family benefits

“FACE-TC strengthens communication between adolescents with cancer and their families about adolescents’ understanding of their illness, their hopes and fears, their goals of care and their end-of-life treatment preferences,” Dr. Lyon added. “With increased access to palliative care services and pediatric advance care planning, families may better understand that stopping intensive medical interventions when their child is dying is not giving up, but rather choosing how best to spend the final days of one’s life.”

Dr. Lyon and the team at Children’s National have pioneered this effort to give seriously ill adolescents a voice and help families break the ice so they know what their child would want if the worst were to happen. The team also aims to provide an extra level of support for busy clinicians so the first conversation about goals of end-of-life care does not happen in the intensive care unit.

You can read the full trial, An Intervention in Congruence for End-of-Life Treatment Preference: A Randomized Trial, in Pediatrics.

You can also read the last manuscript from this clinical trial, Effect of the Family-Centered Advance Care Planning for Teens with Cancer Intervention on Sustainability of Congruence About End-of-Life Treatment Preferences, in JAMA Network.

girl looking at her phone

TikTok could be causing rising cases of tic-like behaviors

girl looking at her phone

Many teenagers who viewed a high number of Tourette syndrome TikTok videos during the COVID-19 pandemic started portraying similar tic-like behaviors.

The impact of social media on children is once again front and center. During the pandemic, experts noticed the increase in functional tic-like disorders and suggested an association with the rise in popularity of social media videos on TikTok. Many teenagers who viewed a high number of Tourette syndrome (TS) TikTok videos during the COVID-19 pandemic portrayed similar tic-like behaviors.

In a new study published in Pediatric Neurology, experts analyzed the 100 most-viewed videos under #tourettes on the media platform. The authors found the symptoms  portrayed as TS on viewed TikTok videos are an inaccurate representation of TS and are more consistent with functional tic-like behaviors.

“Tourette syndrome symptoms portrayals on highly-viewed TikTok videos are predominantly not representative or typical of Tourette syndrome,” says Alonso Zea Vera, M.D., neurologist at Children’s National Hospital and lead author of the study.

“Although many videos are aimed at increasing Tourette syndrome awareness, I worry that some features of these videos can result in confusion and further stigmatization,” Dr. Zea Vera says. “A common cause of stigmatization in Tourette syndrome is the exaggeration of coprolalia (cursing tics) in the media. We found that many videos portrayed this (often used for a comedic effect) despite being a relatively rare symptom in Tourette syndrome.”

There have been recent discussions about the accuracy of current social media videos of TS. This study highlights the importance of mentioning the source of the medical information and providing guidance. Children’s National has one of the largest movement disorders teams in the U.S. that is trained to differentiate TS from functional tic-like disorders.

“This differentiation can be challenging but important since the treatment is different,” Dr. Zea Vera adds. “Both of these conditions can be very impairing for patients.”

You can read the full study ‘The phenomenology of tics and tic-like behavior in TikTok” here.