Annals of the Child Neurology Society最新文献

{"title":"Who Will Protect the Children?","authors":"William D. Graf, Leon G. Epstein","doi":"10.1002/cns3.70049","DOIUrl":"https://doi.org/10.1002/cns3.70049","url":null,"abstract":"<p>At a recent news conference, Florida Surgeon General Dr. Joseph A. Lapado announced plans “to end all vaccine mandates in Florida law.” Dr. Lapado claimed that vaccine requirements were an “immoral” infringement on personal liberty and declared, “Who am I, as a government … to tell you what you should put in your body?” In fact, this is precisely his job. As the state's leading public health advocate for wellness and disease prevention, his responsibility is to promote evidence-based health policies and to inform those Florida citizens who cannot gather, research, or interpret the vast epidemiological data about how infectious diseases arise, spread, and are controlled. In this regard, both Dr. Lapado and the Secretary of the US Department of Health and Human Services (HHS), Robert F. Kennedy Jr., have failed to act solely for the public good and have violated the fundamental principle that “public service is a public trust.”</p><p>Public health imperatives should not compete with personal liberties. Individual freedoms are valued in American culture, but the prevention of contagious diseases requires civic responsibilities and collective action. All adult citizens in the US have autonomy—the ethical principle that they can make decisions for themselves. The autonomy principle is easily defended if an individual lives alone and has no contact with others. However, any infected individual who shops at the supermarket during a viral epidemic can expose vulnerable fellow citizens to the risk of major illness, hospitalization, or death.</p><p>Young children do not have autonomy and must rely on their parents to make health care decisions on their behalf. Children do have an ethical right to a healthy future, independent of their parents. If parents receive misinformation about the risks and benefits of childhood vaccines and decide against the measles, mumps, rubella (MMR) vaccine for their children, those children may die during a future measles epidemic or cause the death of an immunocompromised classmate at school. Before the measles vaccine became available in 1963, 3–4 million cases occurred annually in the United States, with 48,000 hospitalizations, 500 deaths, and 1000 cases of severe encephalitis [<span>1</span>]. Public health officials are responsible for protecting the right of children to a healthy future, even if some parents are vaccine-hesitant.</p><p>There is a long and tragic history of misinformation in the public sphere causing great harm to citizens. AIDS denialism in the early 1990s—the pernicious hypothesis that HIV did not cause AIDS—led to significant public health consequences [<span>2</span>]. A broad body of scientific evidence demonstrated that specific anti-retroviral therapy (ART) decreased viral replication, blocked the spread of HIV between individuals, and prevented maternal transmission during pregnancy. Despite the scientific consensus, AIDS denialists influenced public health policy against the use of ART in So","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"101-102"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70049","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147565877","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lesions Associated With Autism Symptoms Map to a Cerebellar Brain Network in Tuberous Sclerosis Complex","authors":"Wendy Xiao Herman,&nbsp;Gillian N. Miller,&nbsp;Mustafa Sahin,&nbsp;Jurriaan M. Peters,&nbsp;Simon K. Warfield,&nbsp;Darcy A. Krueger,&nbsp;E. Martina Bebin,&nbsp;Hope Northrup,&nbsp;Joyce Y. Wu,&nbsp;Michael D. Fox,&nbsp;Alexander L. Cohen","doi":"10.1002/cns3.70045","DOIUrl":"https://doi.org/10.1002/cns3.70045","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>Autism spectrum disorder (ASD) affects 1 in 36 individuals in the United States and is characterized by impaired social communication and restrictive/repetitive behaviors. Individuals with tuberous sclerosis complex (TSC) have a high incidence of ASD (40%) and exhibit congenital brain lesions (tubers), offering a unique lesion-based model to investigate the neural circuits underlying ASD symptoms. We tested whether tuber connectivity is associated with specific ASD symptom profiles in TSC patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Brain tuber locations were analyzed in 115 children with TSC from the TSC Autism Centers of Excellence Research Network. Lesion network mapping was performed using normative resting-state functional magnetic resonance imaging data from 1000 typically developing 9-year-olds to identify brain networks functionally connected to tuber locations. Multivariable linear regression analyses assessed associations between these networks and specific domain subscores on the Autism Diagnostic Observation Schedule (ADOS).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Greater severity of social affect symptoms was associated with tubers connected to bilateral deep cerebellar nuclei, particularly the right side, localizing to Crus V despite no cerebellar tubers being included in this analysis. No significant associations were found for total ADOS scores or repetitive/restrictive behavior subscores. These effects were independent of age, sex, lesion burden, epilepsy severity, and language ability.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Interpretation</h3>\u0000 \u0000 <p>Social affect impairments in TSC patients with ASD are associated with to functional connectivity between tubers and cerebellar crus V. This symptom-specific neuroanatomical association supports the concept that different ASD features map to distinct brain circuits and highlights the role of cortico-cerebellar pathways in early social development.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"64-73"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70045","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147564946","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vaccination as a Patriotic Activity","authors":"Nina F. Schor","doi":"10.1002/cns3.70050","DOIUrl":"https://doi.org/10.1002/cns3.70050","url":null,"abstract":"<p>There have been times and circumstances in our country's history when taking a risk to protect other Americans has been judged worthy of reward and honor. But getting a vaccine or having one's child vaccinated is not viewed in this light. It seems most people see vaccination as being done to protect the person who is vaccinated. If that person is young and healthy, why take a risk of vaccine side effects when the risk of the illness itself is small and rare? But this view misses the point of vaccination entirely.</p><p>The whole idea of everyone who is old and healthy enough getting a vaccine is to protect those who are not old or healthy enough to be protected by a vaccine. For example, one person with measles in a large room is almost certain to infect every nonimmune other person in that room, and all those people can then spread the infection everywhere they go. One in every 500 infants who gets measles will then get the universally disabling, usually fatal condition called subacute sclerosing panencephalitis, or SSPE, within 10 years of getting measles. During the 2024–2025 season, 37 cases of acute necrotizing encephalitis, or ANE, a severe complication of flu that can cause brain swelling, hemorrhage, and cell death, were identified among 109 cases of flu in people who were not or could not be effectively immunized. And regarding COVID, approximately one in five people who get COVID will have long-term effects that keep them from working, going to school, or even living independently.</p><p>Why should healthy people take the one-in-a-million risk of vaccine injury if they are unlikely to get serious illness or aftereffects from infectious disease? Because healthy people who are immune to an infectious disease cannot give that disease to those who cannot become immune for whom that disease might be disabling or life-threatening. Like military service or being a member of the police or fire department, getting vaccinated is a public service that involves taking a very small risk to protect those fellow Americans for whom infectious disease is a much bigger and sometimes lethal risk. Getting everyone who can be vaccinated a vaccine is the patriotic thing to do.</p><p><b>Nina F. Schor:</b> conceptualization, writing – original draft, writing – review and editing.</p><p>The author received no specific funding for this work.</p><p>The author declares no conflicts of interest.</p>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70050","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147565876","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Not Just Half a Doctor: Promoting Humanism During Stressful Times","authors":"Nigel S. Bamford,&nbsp;Nomazulu Dlamini,&nbsp;Bruce H. Cohen,&nbsp;Ann Tilton,&nbsp;Scott L. Pomeroy,&nbsp;Phillip L. Pearl,&nbsp;Nina F. Schor,&nbsp;E. Steve Roach","doi":"10.1002/cns3.70053","DOIUrl":"10.1002/cns3.70053","url":null,"abstract":"&lt;p&gt;More than 30 years ago, Arnold and Sandra Gold, supported by a Robert Wood Johnson Foundation grant, convened a meeting of 50 medical school deans to discuss ways to preserve humanism in medicine, which, even then, was increasingly compromised by the pressing demands on physicians and trainees [&lt;span&gt;1&lt;/span&gt;]. From this meeting, the white coat ceremony was born—a ritual that enables medical students and other future health care professionals to set aside personal desires and commit to a lifetime of caring. Importantly, the white coat ceremony takes place as the students are matriculating, not at graduation like the traditional reciting of the Hippocratic oath. Subsequent Gold Foundation humanism initiatives include the Gold Humanism Societies at many medical schools, the annual Gold Humanism Award at the Child Neurology Society, and the biennial Gold Humanism in Medicine Workshop at the Child Neurology Society.&lt;/p&gt;&lt;p&gt;Child neurologists face many practice and academic challenges. Stress can negatively affect the way we provide care. A humanistic approach to medicine is always needed, especially under challenging conditions. The sixth biennial Gold Humanism in Medicine workshop at the Child Neurology Society's annual meeting was organized by Nigel Bamford and featured a panel of well-known senior child neurologists. Each panelist was asked to reflect on the insights, experiences, and coping skills that they have found useful during stressful times. Audience members were encouraged to offer their own insights.&lt;/p&gt;&lt;p&gt;The result was a meeting room packed with highly engaged colleagues. As the powerful personal stories and observations began to emerge, the hushed atmosphere reflected the group's common experiences and feelings. Here we try to capture some of the magic of this year's Gold Humanism in Medicine Workshop for those who could not attend.&lt;/p&gt;&lt;p&gt;When I was asked to participate in this panel, I must admit that my understanding of &lt;i&gt;humanism&lt;/i&gt; was limited. So I looked it up and found that, amongst many things, humanism advocates for compassion, justice, equality, and the flourishing of all people. So, what does that have to do with you and your practice as a neurologist, particularly in these challenging times?&lt;/p&gt;&lt;p&gt;One of my early teachers taught me that medicine is more than science; it is also an art. And beneath all of that, medicine is, at its heart, a profoundly &lt;i&gt;human&lt;/i&gt; endeavor. Humanism in medicine reminds us that healing begins not with a smart diagnosis or treatment but with &lt;i&gt;connection&lt;/i&gt;. It entails truly &lt;i&gt;seeing&lt;/i&gt; the whole person, not just the patient, and trying to understand their experience and what matters to them by &lt;i&gt;listening&lt;/i&gt; to their story, and not just symptom hunting. By finding a way to truly see people, in the time that we have, with &lt;i&gt;empathy&lt;/i&gt; and not just efficiency, we restore a sense of one's own humanity and dignity that no prescription alone can provide.&lt;/p&gt;&lt;p&gt;And so I ask you, when wa","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"6-11"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12893704/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146183646","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advantages of Exome Sequencing Over Panel Testing for Individuals With a Seizure Indication","authors":"Michelle M. Morrow,&nbsp;Elizabeth Butler,&nbsp;Melanie P. Napier,&nbsp;Lindsay Havens-Dyer,&nbsp;Annabelle Tuttle,&nbsp;Patricia C. Lopes,&nbsp;Britt A. Johnson,&nbsp;Paul Kruszka,&nbsp;Kirsty McWalter","doi":"10.1002/cns3.70051","DOIUrl":"https://doi.org/10.1002/cns3.70051","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>Our aim was to investigate the advantages of exome sequencing versus panel testing for patients with unexplained seizures.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We reviewed the diagnostic outcomes of exome sequencing by a commercial genetics laboratory for more than 16 000 individuals with a clinical history of seizures or suspected seizures.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Exome sequencing identified diagnostic variants (pathogenic and/or likely pathogenic; L/PATH) in 778 seizure-related genes, resulting in diagnoses in 4035 individuals (24.6% of the cohort). Of the 778 genes, 454 (58.4%) were not present on a comparison sample of six commercially available epilepsy panel tests. The analysis predicted that the epilepsy panels would have missed 52%–63% of diagnostic cases reported by exome sequencing. Nineteen percent (753/4035) of the cohort had a previous epilepsy panel. Exome sequencing added new diagnostic information in this panel-tested sub-cohort, including L/PATH variants not reported on the epilepsy panel (74.2% of patients) and L/PATH variants that were originally classified as variants of uncertain significance by the panel (11.2% of patients). Exome sequencing identified L/PATH variants likely to have immediate implications for clinical intervention in 17.9% of diagnostic cases and in 10.0% of cases with a previous nondiagnostic epilepsy panel.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Significance</h3>\u0000 \u0000 <p>The data demonstrated limitations of epilepsy panels and several advantages of exome sequencing over panels for patients with unexplained seizures. Exome sequencing offered a higher diagnostic rate than previously published for panels and the identification of variants with implications for clinical intervention among patients with prior nondiagnostic epilepsy panels. Another advantage of exome sequencing was the identification of clinically relevant findings unrelated to seizures. Together, these data further support the use of exome sequencing as a first-tier test for patients with unexplained seizures and demonstrate the potential clinical benefits of comprehensive genetic testing.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>Not applicable</p>\u0000 </section>\u0000 </div>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"29-37"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70051","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147566750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early-Life Clinical Factors Shape Cerebellar Structure in Preterm-Born Children","authors":"Victoria Rapos,&nbsp;Steven Ufkes,&nbsp;Cecil Chau,&nbsp;Jessie van Dyk,&nbsp;Ting Guo,&nbsp;Ruth E. Grunau,&nbsp;Steven P. Miller","doi":"10.1002/cns3.70054","DOIUrl":"https://doi.org/10.1002/cns3.70054","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>Preterm birth alters typical brain development due to complex exposures related to neonatal illness, pain management, and brain injury. Despite an increased understanding of neonatal brain injury on developmental outcomes, the association of cerebellar maturation with school-age motor and cognitive function remains incompletely understood. The current study evaluated neonatal clinical factors associated with regionally specific cerebellar volumes and white matter pathways at 8 years of age in children born preterm (24–32 weeks of gestational age).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Neuroimaging, clinical, and behavioral data were collected from 130 children born very preterm (24–32 weeks' gestational age) at BC Women's Hospital. Cerebellar subregions were automatically segmented using CerebNet, and cerebellar white matter connectivity was assessed using tractography. The relationships between cerebellar subregion volumes and neonatal clinical factors were examined using constrained principal component analysis. Cognitive and motor neurodevelopmental outcomes related to cerebellar volume and connectivity were also investigated.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Neonatal clinical factors including mechanical ventilation, number of invasive procedures, and opioid exposures were associated with reduced cerebellar volumes in specific subregions. Acute neonatal mortality risk (i.e., SNAPPE-II) was negatively associated with cerebellar connectivity at 8 years. Reduced cerebellar volumes and structural connectivity were related to poorer cognitive and motor outcomes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Interpretation</h3>\u0000 \u0000 <p>In very preterm children, neonatal illness severity and exposure to invasive procedures and opioids are associated with reduced cerebellar volumes, connectivity, and poorer neurodevelopmental outcomes at 8 years.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"74-86"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70054","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147569785","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bringing the Universe to the University: The Place of International Students in American Academia","authors":"Pedro Weisleder","doi":"10.1002/cns3.70060","DOIUrl":"https://doi.org/10.1002/cns3.70060","url":null,"abstract":"&lt;p&gt;Forty years ago, I arrived in the United States from Mexico, full of excitement and optimism. I was going to be an international student at the University of Arizona. I was ready to learn, explore, and immerse myself in American culture. What I found exceeded my expectations—a country that welcomed me, a system that supported me, and mentors who saw my potential. Over the course of four decades, I transitioned from student to researcher to mentor and, now, a professor emeritus at The Ohio State University—what a journey! I owe much of my professional success to the opportunities afforded to me by this country, opportunities that would have been nearly impossible elsewhere.&lt;/p&gt;&lt;p&gt;Lately, however, I find myself increasingly worried. Recent political developments have shaken my faith in the system that once embraced me. I am concerned that the American academic landscape is closing itself off from the international voices that have enriched it.&lt;/p&gt;&lt;p&gt;I still remember my first day as a postdoctoral fellow at The University of Texas at Austin in 1992. Like many international students and scholars, I attended an orientation session meant to help us understand and integrate into our new academic environment. The dean of the graduate school stepped up to the microphone and welcomed us with words that stayed with me ever since: “Welcome. Thanks for being here. Your presence brings the universe to the University.” It was not just a witty statement. It was an acknowledgment that international students and scholars play a vital role in American academia. We bring the world's view, new ideas and perspectives, and dreams to be fulfilled. That welcoming environment is what has made American universities world-renowned centers of learning.&lt;/p&gt;&lt;p&gt;But that culture is now under threat. Today, international students find themselves caught in the crossfire of contentious political debates. Rhetoric that casts suspicion on outsiders is making it harder for students to fulfill their dreams of an American education. The message is shifting from “You bring the universe” to “You are not welcomed here.”&lt;/p&gt;&lt;p&gt;This change is not merely discouraging—it is concerning. According to the National Foundation for American Policy, between 2000 and 2021, 38% of the United States' Nobel Prize winners in chemistry, medicine, and physics were immigrants, many of whom first came as international students [&lt;span&gt;1&lt;/span&gt;]. When we close the doors to international students, we close the door to progress. The strength of American higher education has come from its openness to ideas, to debate, and to people from all over the world. That openness is being eroded, and the US risks isolating itself. Fewer international students may choose to come here. Some who are already here feel unwelcome or uncertain about their future. Others must navigate complex bureaucratic hurdles simply to study and contribute.&lt;/p&gt;&lt;p&gt;Many international students are not visitors just passing through—we become par","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"4-5"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70060","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147570325","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method","authors":"Erin O'Connor Prange,&nbsp;Arthur Beisang,&nbsp;Davut Pehlivan,&nbsp;Bernhard Suter,&nbsp;Paula Schleifer","doi":"10.1002/cns3.70062","DOIUrl":"https://doi.org/10.1002/cns3.70062","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>Trofinetide is the first approved treatment for Rett syndrome (RTT) in the United States and Canada. Trofinetide improved the core symptoms of RTT in clinical trials, and real-world evidence supports the findings of clinical trials. As RTT experts in the United States have now gained nearly 3 years of real-world experience with trofinetide, a modified Delphi process was conducted in the United States to establish consensus recommendations for the practical use of trofinetide in the treatment of RTT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A multidisciplinary steering group of five trofinetide-experienced RTT experts practicing at an International Rett Syndrome Foundation (IRSF)-designated center of excellence convened and developed 72 consensus statements across six domains: first-line use, pre-treatment assessment, initiation, benefit evaluation, tolerability management, and discontinuation strategies. The statements were then assigned a 4-point Likert scale for testing via email by the Delphi panel identified by the steering group. Consensus was reached for individual statements if they met the pre-specified consensus threshold of ≥ 75% agreement.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Two rounds of assessments were completed by 25 respondents in each round, resulting in agreement ≥ 75% being reached across the final statement set. The consensus supports trofinetide as the standard of care for eligible patients with early initiation and an individualized approach to titration to maximize efficacy and tolerability. The panel reflected the steering group's view that trofinetide is efficacious. While side effects can emerge with trofinetide, they are manageable in most cases.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Recommendations highlight a flexible, patient-centered approach to trofinetide use to optimize efficacy while addressing tolerability challenges and supporting adherence.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"38-51"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70062","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147563701","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Non-Syndromic Tumefactive Demyelinating Lesions in the Pediatric Population: Four Case Reports and Review of the Literature","authors":"Elizabeth C. Ballinger,&nbsp;Hannah F. Todd,&nbsp;Jonathan M. Yarimi,&nbsp;Timothy E. Lotze,&nbsp;Nikita M. Shukla,&nbsp;Kristen S. Fisher,&nbsp;Alexander J. Sandweiss","doi":"10.1002/cns3.70055","DOIUrl":"https://doi.org/10.1002/cns3.70055","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Tumefactive demyelinating lesions (TDLs) are large lesions (&gt; 2 cm) seen in demyelinating syndromes such as multiple sclerosis, myelin oligodendrocyte glycoprotein antibody-associated disorder, and neuromyelitis optica spectrum disorder. They rarely occur in children and most often have a monophasic course. TDLs can cause severe presentations given their size, edema, and associated mass effect, making prompt diagnosis and effective treatment crucial.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patient Description</h3>\u0000 \u0000 <p>We present four adolescents with TDLs who tested negative for aquaporin-4-IgG and myelin oligodendrocyte glycoprotein (MOG)-IgG and did not meet diagnostic criteria for any known demyelinating disorder. All four patients underwent extensive and inconclusive workup for the etiology of their radiographic and clinical findings and received multiple immunomodulators with some clinical improvement, although three of the four had multiple relapses.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>TDLs that do not meet criteria for the known demyelinating syndromes can cause significant neurological sequelae. We describe four adolescents with neurological symptoms associated with TDLs. Given the rare but serious occurrence of these non-syndromic TDLs, more well-powered studies are critical to guide diagnosis, management, and response to therapies and eventually develop evidence-based protocols to manage other patients with similar presentations.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"87-94"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70055","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147567932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retrospective, Multicenter Study of Lacosamide to Treat Neonatal Seizures","authors":"Alexandra Santana Almansa,&nbsp;Jessica R. Landers,&nbsp;Nicholas S. Abend,&nbsp;Giulia M. Benedetti,&nbsp;Catherine J. Chu,&nbsp;Andrew T. Knox,&nbsp;Shavonne Massey,&nbsp;Steffany Moen,&nbsp;Andrea C. Pardo,&nbsp;Arnold Sansevere,&nbsp;Renée A. Shellhaas,&nbsp;Cameron Thomas,&nbsp;Tammy Tsuchida,&nbsp;Sonya Wang,&nbsp;Bo Zhang,&nbsp;Janet S. Soul","doi":"10.1002/cns3.70044","DOIUrl":"https://doi.org/10.1002/cns3.70044","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>Most antiseizure medications (ASMs) are prescribed off label for neonates. Lacosamide's efficacy in infants and availability in intravenous formulation suggest potential utility for neonates. We evaluated the safety and efficacy of lacosamide for neonatal seizures.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This 10-center, retrospective study of neonates with seizures and lacosamide treatment initiated by ≤ 48 weeks postmenstrual age collected clinical data from medical records and electroencephalogram recordings. Lacosamide efficacy was determined by changes in seizure burden with lacosamide treatment and seizure cessation by hospital discharge. Potential adverse events were reviewed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among 62 eligible neonates, 33 had acute provoked seizures while 29 had neonatal-onset epilepsy; there was no difference in seizure type or baseline seizure severity between groups. There were high rates of pretreatment status epilepticus (48%) and treatment-resistant seizures, with 93% receiving ≥ 3 ASMs before lacosamide. Most received intravenous lacosamide, with a median loading dose of 5.0 mg/kg and median daily dose of 7.3 mg/kg. Seizure cessation occurred in 37% of neonates; 21% had no additional ASM administered after lacosamide. Seizure burden, measured in seizure minutes per hour, was lower at both 4 h and 7 days following lacosamide administration. In addition, there was a median reduction in seizure frequency of 30 seizures per day at 7 and 30 days posttreatment (<i>p</i> &lt; .05). Lacosamide was continued at discharge in most neonates (72%). Seventy adverse events were reported in 35 (56%) neonates. Four transient events with possible or unknown relationship to lacosamide were likely multifactorial in origin; none were cardiac arrhythmias.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Summary</h3>\u0000 \u0000 <p>Despite high rates of treatment-resistant seizures in this neonatal cohort, 37% experienced seizure cessation and most remained on lacosamide at hospital discharge. Most adverse events were not attributed to lacosamide. These results favor use of lacosamide and provide a rationale for future prospective studies.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72232,"journal":{"name":"Annals of the Child Neurology Society","volume":"4 1","pages":"52-63"},"PeriodicalIF":0.0,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cns3.70044","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147566908","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}