Developmental Medicine and Child Neurology最新文献

{"title":"Glycopyrronium 320 μg/mL in children and adolescents with severe sialorrhoea and neurodisabilities: An open-label study extension of the SALIVA trial","authors":"","doi":"10.1111/dmcn.16268","DOIUrl":"10.1111/dmcn.16268","url":null,"abstract":"<p>Drooling (sialorrhoea) is common in children with neurodisabilities, such as cerebral palsy. Severe drooling can cause irritated skin, dehydration, retention of urine, and chest infections. Drooling may also affect the child's quality of life, self-esteem, and social interactions. In addition, it can add to the burden of parents and caregivers, for example, leading to frequent changes of bibs and clothing. The SALIVA (<span>S</span>ialanar plus or<span>A</span>l rehabi<span>L</span>itation against placebo plus oral rehabilitation for ch<span>I</span>ldren and adolescents with se<span>V</span>ere sialorrhoe<span>A</span> and neurodisabilities) trial investigated 320 μg/mL glycopyrronium, a medicine that has been specifically designed to meet the needs of children.</p><p>An open label (or non-blinded) study is one in which both the healthcare providers and the patients are aware of the drug or treatment being given.</p><p>In the first part of the trial, 87 children (aged 3–17 years) with neurodisabilities and severe drooling were randomly allocated to receive either 320 μg/mL glycopyrronium or placebo for 12 weeks. Treatment with 320 μg/mL glycopyrronium significantly reduced drooling versus placebo and the impact of drooling on quality of life was decreased, with no unexpected side effects.</p><p>In the second part of the trial, all children received 320 μg/mL glycopyrronium for 24 weeks. Improvements in drooling seen in the first part were sustained with continued 320 μg/mL glycopyrronium. Around 80% of children responded to treatment and 70% showed a good response. In addition, reductions in drooling's impact on quality of life were sustained with continued 320 μg/mL glycopyrronium.</p><p>Overall, the side effects seen with 320 μg/mL glycopyrronium were typical of this type of medicine, most commonly constipation and dry mouth. Side effects were more frequent during the first 4 weeks, when the dose was being adjusted, with the frequency of side effects subsequently reducing.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":"67 4","pages":"e85"},"PeriodicalIF":3.8,"publicationDate":"2025-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/dmcn.16268","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143384086","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Involving people with lived experience when setting cerebral palsy research priorities: A scoping review","authors":"","doi":"10.1111/dmcn.16269","DOIUrl":"10.1111/dmcn.16269","url":null,"abstract":"<p>Scoping reviews identify key concepts within a particular research area and types of evidence currently available. This scoping review examines how people with lived experience of cerebral palsy (CP) have contributed to setting priorities for CP research in order to characterize how the current body of literature addresses the high priority needs of those directly affected. This review specifically focused on characteristics of the participants who were involved, methods used, and research priorities identified. Five projects were identified that involved people with lived experience of CP in setting research priorities, conducted in North America and Australia.</p><p>Most participants with lived experience were caregivers, with fewer individuals with CP making up the total number of participants. The methods varied, including surveys, workshops, and webinars. The most commonly identified areas of research were related to optimizing interventions. Four of the five projects actively involved participants with lived experience of CP as research partners, influencing decisions at every stage of the research.</p><p>This review highlighted significant gaps in representation of the CP community. People with CP, particularly from low- and middle-income countries were underrepresented. Similarly, perspectives from children and young adults with CP and individuals with more complex needs, such as those using augmentative communication devices to supplement or replace speech or writing, were rarely included.</p><p>The findings emphasize the importance of including a broader range of voices in future research priority-setting projects. This can ensure that the unique needs and perspectives of all individuals with CP, regardless of background or condition severity, are reflected in the literature. Employing rigorous methods and collaborating with people with lived experience as research partners is also necessary to improve transparency and reliability of new research.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":"67 4","pages":"e86"},"PeriodicalIF":3.8,"publicationDate":"2025-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/dmcn.16269","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143384089","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Wearable sensors in paediatric neurology","authors":"","doi":"10.1111/dmcn.16267","DOIUrl":"10.1111/dmcn.16267","url":null,"abstract":"<p>Wearable sensors are small, non-invasive devices that can easily be attached to the body to monitor signals such as movement, heart rate, breathing, and temperature. These sensors allow data to be collected in familiar environments, such as at home or during school, and capture real-world patterns and behaviours that may not be evident during traditional clinical assessments. This approach reduces the stress of being examined, minimizes bias, and overcomes poor cooperation, especially in younger patients.</p><p>Wearable sensors have been studied for various paediatric neurological conditions, including cerebral palsy, epilepsy, autism spectrum disorder, attention-deficit/hyperactivity disorder, Rett syndrome, Down syndrome, Angelman syndrome, Prader–Willi syndrome, and neuromuscular disorders such as Duchenne muscular dystrophy and spinal muscular atrophy. Other areas of application are ataxia, Gaucher disease, headaches, and sleep disorders.</p><p>The data collected by wearable sensors can be used to detect early signs of neurological disorders and monitor changes over time. For example, subtle changes in walking patterns that might go unnoticed through conventional clinical assessments can be detected using these devices. This information could enable earlier interventions, more accurate diagnoses, and personalized treatment plans tailored to each child's specific needs.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":"67 4","pages":"e84"},"PeriodicalIF":3.8,"publicationDate":"2025-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/dmcn.16267","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143383293","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and effectiveness of stiripentol in patients with Dravet syndrome: A prospective, 3-year, postmarketing surveillance study.","authors":"Yuki Kitamura, Hiroaki Ohyabu, Tatsuo Miura, Naomi Takei-Masuda, Daisuke Matsui, Yushi Inoue, Yoko Ohtsuka","doi":"10.1111/dmcn.16252","DOIUrl":"https://doi.org/10.1111/dmcn.16252","url":null,"abstract":"<p><strong>Aim: </strong>To conduct a postmarketing surveillance study of patients with Dravet syndrome in Japan to investigate the safety and effectiveness of long-term, real-world, clinical use of stiripentol (STP).</p><p><strong>Method: </strong>This prospective study was conducted over 156 weeks in all patients with Dravet syndrome who started STP treatment from its launch in Japan in November 2012 until August 2017. Adverse drug reactions (ADRs) were investigated by degree of seriousness. Effectiveness was determined based on a comprehensive assessment by the physician in charge as well as on the percentage change in the number of seizures from the pretreatment period.</p><p><strong>Results: </strong>In total, 520 patients (266 males, 254 females; mean age [SD] 10 years 6 months [9 years 10 months]; age range 0-50 years) were included in the safety analysis set, and 515 patients in the effectiveness analysis set. ADRs occurred in 69.2%, including somnolence, decreased appetite, dizziness, in order of frequency. Twelve deaths occurred, the rate of which was not higher than the reported rates. No new safety concerns were identified. The rate of overall improvement (marked or moderate) after 156 weeks or at treatment discontinuation was 37.7%. Decreases in the number of all seizure types over the long term were confirmed.</p><p><strong>Interpretation: </strong>In real-world clinical settings, long-term STP treatment can be safe and effective in patients with Dravet syndrome.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143384097","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Developmental trajectories of impaired hand function in unilateral cerebral palsy: Clinical insights and future directions.","authors":"Nava Gelkop, Hanoch Cassuto","doi":"10.1111/dmcn.16257","DOIUrl":"https://doi.org/10.1111/dmcn.16257","url":null,"abstract":"","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143191246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Glycopyrronium 320 μg/mL in children and adolescents with severe sialorrhoea and neurodisabilities: An open-label study extension of the SALIVA trial.","authors":"Pierre Fayoux, Mickael Dinomais, Helen Shaw, Frédéric Villain, Déborah Schwartz, Vincent Gautheron, Guy Letellier, Stéphane Auvin","doi":"10.1111/dmcn.16251","DOIUrl":"https://doi.org/10.1111/dmcn.16251","url":null,"abstract":"<p><strong>Aims: </strong>To test the long-term efficacy, safety, and impact on quality of life (QoL) of an oral paediatric formulation of 320 μg/mL glycopyrronium in the 36-week SALIVA (Sialanar plus orAl rehabiLitation against placebo plus oral rehabilitation for chIldren and adolescents with seVere sialorrhoeA and neurodisabilities) trial.</p><p><strong>Method: </strong>In the initial 12-week blinded period, 87 children with neurodisabilities and severe sialorrhoea were randomized to 320 μg/mL glycopyrronium versus placebo. In the subsequent 24-week open-label study extension, 74 children received 320 μg/mL glycopyrronium (37 continued glycopyrronium, 37 switched from placebo).</p><p><strong>Results: </strong>The open-label study extension population included 39 males and 35 females. The median age was 10 years 2 months (quartile 1, quartile 3: 7 years 5 months, 14 years 7 months; range: 3 years 5 months-17 years 8 months). Over 36 weeks, continued 320 μg/mL glycopyrronium resulted in a median 39-point reduction in Drooling Impact Scale (DIS) score from baseline (quartile 1, quartile 3: -51, -21; p < 0.001), with an 81.1% response rate (DIS improvement ≥ 13.6 points) and a 70.3% good response rate (≥ 28 points). Improvements in the impact of drooling on QoL seen in the blinded period were sustained with continued glycopyrronium. Treatment-related adverse events occurred most frequently during titration (0-4 weeks: 40.9%; 5-20 weeks: 32.4% in those who switched). Constipation was the most common adverse event.</p><p><strong>Interpretation: </strong>Long-term treatment with 320 μg/mL glycopyrronium resulted in significant sustained improvements in drooling and QoL, with fewer adverse events after initial titration and overall good tolerability.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143070804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Wearable sensors in paediatric neurology.","authors":"Camila González Barral, Laurent Servais","doi":"10.1111/dmcn.16239","DOIUrl":"https://doi.org/10.1111/dmcn.16239","url":null,"abstract":"<p><p>Wearable sensors have the potential to transform diagnosis, monitoring, and management of children who have neurological conditions. Traditional methods for assessing neurological disorders rely on clinical scales and subjective measures. The snapshot of the disease progression at a particular time point, lack of cooperation by the children during assessments, and susceptibility to bias limit the utility of these measures. Wearable sensors, which capture data continuously in natural settings, offer a non-invasive and objective alternative to traditional methods. This review examines the role of wearable sensors in various paediatric neurological conditions, including cerebral palsy, epilepsy, autism spectrum disorder, attention-deficit/hyperactivity disorder, as well as Rett syndrome, Down syndrome, Angelman syndrome, Prader-Willi syndrome, neuromuscular disorders such as Duchenne muscular dystrophy and spinal muscular atrophy, ataxia, Gaucher disease, headaches, and sleep disorders. The review highlights their application in tracking motor function, seizure activity, and daily movement patterns to gain insights into disease progression and therapeutic response. Although challenges related to population size, compliance, ethics, and regulatory approval remain, wearable technology promises to improve clinical trials and outcomes for patients in paediatric neurology.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143071159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Historia natural de la distonía mioclónica asociada a variantes de SGCE en niños y adolescentes","authors":"Valeria De Francesch,&nbsp;Ana Cazurro-Gutiérrez,&nbsp;Elze R. Timmers,&nbsp;Gemma Español-Martín,&nbsp;Julia Ferrero-Turrión,&nbsp;David Gómez-Andrés,&nbsp;Anna Marcé-Grau,&nbsp;Lucía Dougherty-de Miguel,&nbsp;Victoria González,&nbsp;Antonio Moreno-Galdó,&nbsp;Marina A. J. Tijssen,&nbsp;Belén Pérez-Dueñas","doi":"10.1111/dmcn.16215","DOIUrl":"10.1111/dmcn.16215","url":null,"abstract":"<p>Children and adolescents with <i>SGCE</i>-myoclonus dystonia showed a progression of motor symptoms during a mean follow-up of 4 years. Patients developed a significant increase in the severity of axial and limb myoclonus, as well as dystonia during writing. Consequently, patients reported a marked decline in their speech, writing, and walking abilities. Up to 74% of patients had a psychiatric diagnosis, most commonly anxiety, obsessive-compulsive disorders, and attention-deficit/hyperactivity disorder.\u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure></p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":"67 6","pages":"e104-e114"},"PeriodicalIF":3.8,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143071156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cerebral palsy and perinatal mortality in children born in Norway to immigrant mothers.","authors":"Maria Wiedswang Sigholt, Guro L Andersen, Stian Lydersen, Liv Cecilie Vestrheim Thomsen, Torstein Vik, Sandra Julsen Hollung","doi":"10.1111/dmcn.16253","DOIUrl":"https://doi.org/10.1111/dmcn.16253","url":null,"abstract":"<p><strong>Aim: </strong>To compare the prevalence and clinical characteristics of cerebral palsy (CP), and perinatal mortality, in children born to non-immigrant mothers with children born to immigrant mothers.</p><p><strong>Method: </strong>This was a registry-based cross-sectional study. Data on children born from 2000 to 2016 were extracted from the Medical Birth Registry of Norway and linked to the Norwegian Quality and Surveillance Registry for Cerebral Palsy. The mother's country of birth was categorized into three groups: non-immigrant (born in Norway); immigrant from high-income countries (HICs); and immigrant from low- and middle-income countries (LMICs) (born abroad giving birth in Norway). Birth prevalence of CP and prevalence of perinatal mortality per 1000 live births were calculated. Odds ratios (ORs) for CP among live-born children of non-immigrant mothers compared to mothers from HICs and LMICs were calculated using logistic regression, both unadjusted and adjusted for each risk factor for CP. Pearson χ² tests were used to compare the proportions of clinical characteristics.</p><p><strong>Results: </strong>The prevalence of CP among non-immigrant mothers was 2.11 per 1000 live births, 1.44 among mothers from HICs, and 1.71 among mothers from LMICs. The OR for CP in mothers from HICs was 0.68 and 0.81 in mothers from LMICs. Despite mothers from LMICs having higher proportions of consanguinity and lower folate intake, and their children having lower Apgar scores, the ORs for CP were unchanged after adjusting for these. Yet, children born to mothers from LMICs had higher perinatal mortality; their children with CP had higher proportions of intellectual disability.</p><p><strong>Interpretation: </strong>The lower birth prevalence of CP among children born to mothers from LMICs was unexpected. Yet, children born to mothers from LMICs had higher perinatal mortality, which could impact the number of live-born children with CP.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069357","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Consciousness trajectories and functional independence after acute brain injury in children with prolonged disorder of consciousness.","authors":"Ningning Chen, Helin Zheng, Ying Feng, Congjie Chen, Li Xie, Duan Wang, Xiaoling Duan, Ting Zhang, Nong Xiao, Tingsong Li","doi":"10.1111/dmcn.16244","DOIUrl":"https://doi.org/10.1111/dmcn.16244","url":null,"abstract":"<p><strong>Aim: </strong>To explore the trajectories of consciousness recovery and prognosis-associated predictors in children with prolonged disorder of consciousness (pDoC).</p><p><strong>Method: </strong>This single-centre, retrospective, observational cohort involved 134 (87 males, 47 females) children diagnosed with pDoC and hospitalized at the Department of Rehabilitation at the Children's Hospital of Chongqing Medical University in China. The median onset age was 30 (interquartile range [IQR] 18-54) months, with onset ages ranging from 3 to 164 months. Least absolute shrinkage and selection operator (LASSO) regression and logistic regression analyses were performed to identify the independent predictors of consciousness recovery at 1 year after brain injury. Discrimination and calibration were assessed using 1000 bootstrap resamples. The potential predictors of resultant living independence were also explored.</p><p><strong>Results: </strong>The predictors for consciousness recovery at 1-year postinjury were: traumatic brain injury (odds ratio [OR]: 3.26, 95% confidence interval [95% CI]: 1.21-9.46), electroencephalogram (EEG) grade IV or below based on Young's classification (OR: 3.41, 95% CI: 1.38-8.70), and no bilateral impairments in the basal ganglia (OR: 3.75, 95% CI: 1.50-9.91) or posterior cingulate (OR: 5.61, 95% CI: 2.20-15.54). A nomogram was constructed with the area under the curve of 0.845 (95% CI: 0.780-0.911). Additionally, EEG grade IV or below, and the absence of bilateral impairments in the frontal lobes and occipital lobes were associated with favorable functional outcomes.</p><p><strong>Interpretation: </strong>These findings underscore the importance of comprehensive early-stage assessments in evaluating consciousness and function, assisting clinicians and families in making clinical decisions.</p>","PeriodicalId":50587,"journal":{"name":"Developmental Medicine and Child Neurology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143054266","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}