European Journal of Paediatric Neurology最新文献_第8页

Assessment of somatosensory profiles by quantitative sensory testing in children and adolescents with and without cerebral palsy and chronic pain 通过定量感觉测试评估患有或不患有脑瘫和慢性疼痛的儿童和青少年的体感特征

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-18 DOI: 10.1016/j.ejpn.2024.05.007

Johanne Villars Jørgensen , Mads Utke Werner , Josephine Sandahl Michelsen , Christian Nai En Tierp-Wong

Objective

We investigated differences in somatosensory profiles (SSPs) assessed by quantitative sensory testing in children and adolescents with cerebral palsy (CCP) with and without chronic pain and compared these differences to those in a group of typically developed children and adolescents (TDC) with and without chronic pain.

Method

All included subjects were consecutively recruited from and tested at the same outpatient orthopedic clinic by the same investigator. The subjects had their reaction times tested. The SSP consisted of the following tests: warmth (WDT), cool (CDT), mechanical (MDT), and vibration (VDT) detection thresholds; heat (HPT), pressure (PPT), and mechanical (MPT) pain thresholds; wind-up ratio (WUR); dynamic mechanical allodynia (DMA) and cold pressor test (CPT) using a conditioned pain modulation (CPM) paradigm.

Results

We included 25 CCP and 26 TDC. TDC without chronic pain served as controls. In TDC with chronic pain, WDT, HPT, HPT intensity, and PPT were higher than in controls. No differences in SSPs between CCP with and without chronic pain were observed. In CCP, the MDT, WDT, CDT, and HPT intensity were higher than in controls. CCP had longer reaction times than TDC. There were no differences regarding the remaining variables.

Discussion

In CCP, the SSPs were independent of pain status and findings on MR images. In all CCP the SSPs resembled TDC with chronic pain, compared to TDC without chronic pain. This suggests that CCP do not have the normal neuroplastic adaptive processes that activate and elicit functional changes in the central and peripheral nervous systems.

目的我们研究了通过定量感觉测试评估的患有和不患有慢性疼痛的脑瘫（CCP）儿童和青少年的体感特征（SSPs）的差异，并将这些差异与一组患有和不患有慢性疼痛的发育正常儿童和青少年（TDC）的体感特征进行了比较。受试者均接受了反应时间测试。SSP 包括以下测试：温觉（WDT）、冷觉（CDT）、机械觉（MDT）和振动觉（VDT）检测阈值；热觉（HPT）、压觉（PPT）和机械觉（MPT）疼痛阈值；上风比（WUR）；使用条件性疼痛调制（CPM）范式的动态机械痛觉（DMA）和冷压试验（CPT）。无慢性疼痛的 TDC 作为对照组。有慢性疼痛的 TDC 的 WDT、HPT、HPT 强度和 PPT 均高于对照组。有慢性疼痛和无慢性疼痛的 CCP 之间的 SSP 没有差异。CCP 的 MDT、WDT、CDT 和 HPT 强度均高于对照组。CCP 的反应时间比 TDC 长。讨论在 CCP 中，SSPs 与疼痛状态和磁共振成像结果无关。与无慢性疼痛的 TDC 相比，所有 CCP 的 SSPs 都与伴有慢性疼痛的 TDC 相似。这表明 CCP 没有正常的神经可塑性适应过程来激活和引起中枢和周围神经系统的功能变化。

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引用次数: 0

The when, why, and how of using glycopyrronium to diminish drooling in children with neurodevelopmental disabilities: Implications for clinical practice 何时、为何以及如何使用甘草酸铵来减少神经发育障碍儿童的流口水：对临床实践的启示

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-16 DOI: 10.1016/j.ejpn.2024.05.004

Lynn B. Orriëns , Jan J.W. van der Burg , Frank J.A. van den Hoogen , Michèl A.A.P. Willemsen , Karen van Hulst , Corrie E. Erasmus

Background

Despite the established efficacy of glycopyrronium bromide in reducing drooling among children with neurodevelopmental disabilities, evidence on its impact on the daily lives of children and parents and effectiveness in a real-world setting are scarce, especially among long-term users. This study explored timing and duration of glycopyrronium treatment, effect and impact on daily life, and occurrence of side effects to inform clinical practice.

Methods

This was a retrospective cohort study at a national referral centre for drooling, including 61 children with nonprogressive neurodevelopmental disabilities, treated with glycopyrronium for anterior and/or posterior drooling between 2011 and 2021. Data were obtained from medical records and supplemented by structured telephone interviews with parents.

Results

Anterior drooling severity decreased in 82% of the included children. Changes in the impact of drooling on burden of care, social interaction, and self-esteem were reported in 55%, 31%, and 36%, respectively. Side effects were noted for 71% of cases, yet only 36% of parents deemed these as outweighing the positive impact of treatment. A substantial majority (77%) of the included children were long-term users (≥6 months). Among these, 38% of parents reported decreasing effectiveness and 27% noticed more prominent side effects over time.

Conclusions

Glycopyrronium demonstrated potential in mitigating the impact of drooling on daily life, although variations were observed in the specific aspects and extent of improvement. The real-world context of our study provides important insights for refining clinical practices, emphasizing the need for balanced consideration of treatment benefits and potential side effects to facilitate shared decision-making.

背景尽管甘草溴铵在减少神经发育障碍儿童流口水方面具有公认的疗效，但有关其对儿童和家长日常生活的影响以及在实际环境中的有效性的证据却很少，尤其是在长期使用者中。本研究探讨了甘草酸苷治疗的时间和持续时间、效果和对日常生活的影响以及副作用的发生情况，为临床实践提供参考。方法这是一项在全国流口水转诊中心进行的回顾性队列研究，研究对象包括2011年至2021年间接受甘草酸苷治疗前部和/或后部流口水的61名非进行性神经发育障碍儿童。数据来源于医疗记录，并辅以对家长的结构化电话访谈。据报告，55%、31% 和 36% 的儿童流口水对护理负担、社会交往和自尊的影响有所改变。71%的病例出现了副作用，但只有 36% 的家长认为这些副作用超过了治疗的积极影响。绝大多数儿童（77%）是长期使用者（≥6 个月）。结论甘草酸铵在减轻流口水对日常生活的影响方面具有潜力，但在具体方面和改善程度上存在差异。我们研究的现实背景为完善临床实践提供了重要启示，强调了平衡考虑治疗效果和潜在副作用的必要性，以促进共同决策。

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引用次数: 0

Different trajectories of post-concussive symptom subscales after pediatric mild traumatic brain injury: Data from a prospective longitudinal study 小儿轻度脑外伤后撞击后症状分量表的不同轨迹：前瞻性纵向研究数据

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-08 DOI: 10.1016/j.ejpn.2024.05.003

Martina Studer , Lara Mischler , Fabrizio Romano , Karen Lidzba , Sandra Bigi

Purpose

The aim of this study was to investigate the trajectory of parent-rated post-concussive symptoms (PCS), attentional performance and participation within 6 months in children after mild traumatic brain injury (mTBI).

Methods

For this prospective longitudinal study, we included data on 64 children after mTBI and 57 healthy control children (age 8–16 years). Parents rated PCS using the Post-Concussion Symptom Inventory (PCSI) immediately (T0), 1 week (T1), and 3–6 months after injury (T2). Attentional performance (alertness, selective and divided attention) was measured using the Test of Attentional Performance (TAP) at T1 and T2 and participation was measured using the Child and Adolescent Scale of Participation (CASP) at T2.

Results

Friedman tests showed different trajectories of PCS subscales over time: Compared to pre-injury level, the amount of somatic and cognitive PCS was still elevated at T1, while emotional PCS at T1 were already comparable to pre-injury level. The rating of sleep-related PCS at T2 was significantly elevated compared to the pre-injury rating. Quade ANCOVAs indicated group differences in PCS subscales between patients and controls at T1, but not at T2. Patients and controls showed a similar performance in tests of attention at T1 and T2, but parental rating of participation at school was significantly reduced. Although cognitive PCS and attention were not correlated, there were significantly negative Spearman correlations between participation at home and pre-injury and concurrent PCS at T2.

Conclusions

Our data imply that sleep-related PCS are still elevated weeks after injury and are thus a target for interventions after mTBI.

目的本研究旨在调查轻度脑外伤（mTBI）后儿童在 6 个月内由家长评定的脑震荡后症状（PCS）、注意力表现和参与情况的变化轨迹。方法在这项前瞻性纵向研究中，我们纳入了 64 名轻度脑外伤后儿童和 57 名健康对照组儿童（8-16 岁）的数据。家长使用脑震荡后症状量表（PCSI）对受伤后立即（T0）、一周（T1）和 3-6 个月（T2）的 PCS 进行评分。在 T1 和 T2 期间，使用注意力表现测试（TAP）测量注意力表现（警觉性、选择性和分散注意力），在 T2 期间，使用儿童和青少年参与量表（CASP）测量参与情况：与受伤前的水平相比，躯体和认知方面的 PCS 在 T1 阶段仍然偏高，而情绪方面的 PCS 在 T1 阶段已经与受伤前的水平相当。与受伤前的评分相比，T2 的睡眠相关 PCS 评分明显升高。奎德方差分析显示，患者和对照组在 T1 阶段的 PCS 分量表存在组间差异，但在 T2 阶段则没有。患者和对照组在 T1 和 T2 的注意力测试中表现相似，但家长对其参与学校活动的评分明显降低。虽然认知PCS与注意力没有相关性，但在T2时，在家中的参与度与受伤前和同时的PCS之间存在明显的Spearman负相关。结论我们的数据表明，与睡眠相关的PCS在受伤数周后仍会升高，因此是mTBI后的干预目标。

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引用次数: 0

Cognitive function in SMA patients with 2 or 3 SMN2 copies treated with SMN-modifying or gene addition therapy during the first year of life 有 2 或 3 个 SMN2 拷贝的 SMA 患者在出生后第一年接受 SMN 改性或基因添加疗法治疗后的认知功能

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-08 DOI: 10.1016/j.ejpn.2024.05.002

Paula Steffens , Deike Weiss , Anna Perez , Manuel Appel , Philipp Weber , Claudia Weiss , Corinna Stoltenburg , Ute Ehinger , Maja von der Hagen , Jens Schallner , Birte Claussen , Ilka Lode , Andreas Hahn , Rahel Schuler , Lena Ruß , Andreas Ziegler , Jonas Denecke , Jessika Johannsen

Background

Spinal muscular atrophy (SMA) is a neuromuscular disease, causing progressive muscle weakness due to loss of lower motoneurons. Since 2017, three therapies, two modifying gene transcription and one adding the defective gene, have been approved with comparable efficacy on motor outcome. Data on cognitive outcomes of treated SMA type 1 patients is limited. The aim of this study was to evaluate cognitive function in symptomatic and presymptomatic SMA type 1 patients with two or three SMN2 copies who received SMN-modifying or gene-addition therapy in the first year of life.

Methods

Cognitive testing was performed in 20 patients, including 19 symptomatic SMA type 1 patients with up to three SMN2 copies and 1 pre-symptomatically treated patient. Children were tested using Bayley Scales of Infant Development (BSID-III) at the age of 2 or 3 years or the Wechsler Preschool and Primary Scale of Intelligence (WPSII-IV) at the of age of 5 years.

Results

11/20 patients showed subnormal cognitive development. Boys had significantly lower cognitive scores. Patients requiring assisted ventilation or feeding support were more likely to have cognitive deficits. Achieving more motor milestones was associated with a better cognitive outcome.

Conclusion

Treated patients with SMA type 1 have heterogeneous cognitive function with 55 % of patients showing deficits. Risk factors for cognitive impairment in our cohort were male gender and need for assisted ventilation or feeding support. Therefore, cognitive assessment should be included in the standard of care to allow early identification of deficits and potential therapeutic interventions.

背景脊髓性肌萎缩症（SMA）是一种神经肌肉疾病，因下部运动神经元缺失而导致进行性肌无力。自 2017 年以来，已有三种疗法获得批准，其中两种改变了基因转录，一种增加了缺陷基因，对运动结果的疗效相当。有关接受治疗的SMA 1型患者认知结果的数据十分有限。本研究旨在评估有症状和无症状的SMA 1型患者的认知功能，这些患者有两个或三个SMN2拷贝，并在出生后第一年接受了SMN修饰或基因添加治疗。方法对20名患者进行了认知测试，包括19名有多达三个SMN2拷贝的无症状SMA 1型患者和1名无症状治疗前患者。儿童在2岁或3岁时使用贝利婴儿发展量表（BSID-III）进行测试，或在5岁时使用韦氏学前和小学智能量表（WPSII-IV）进行测试。男孩的认知得分明显较低。需要辅助通气或喂养支持的患者更容易出现认知缺陷。结论经过治疗的1型SMA患者的认知功能参差不齐，55%的患者存在认知障碍。在我们的队列中，认知功能障碍的风险因素包括男性、需要辅助通气或喂养支持。因此，应将认知评估纳入标准护理中，以便及早发现患者的认知功能缺陷，并采取潜在的治疗干预措施。

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引用次数: 0

Insights into Rett Syndrome girls' receptive vocabulary through eye movements and parental perception 通过眼球运动和父母的感知了解雷特综合症女孩的接受性词汇。

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-04 DOI: 10.1016/j.ejpn.2024.05.001

Jucineide S. Xavier , Fernanda T. Orsati , Paulo G. Laurence , Lourenço K. Barbosa , Andressa S. Gouveia , Maria Elóisa F. D'Antino , Katerina Lukasova , Elizeu C. Macedo , José Salomão Schwartzman

Objective

This study evaluated the receptive vocabulary of girls diagnosed with Rett Syndrome (RS) by employing eye-tracking technology and examined how these objective measures compared with parents' perceptions of their daughters' language abilities.

Method

Fourteen girls with RS and eleven typically developing peers participated. Instruments included the Kerr Scale, a parental questionnaire on communication skills, the Peabody Picture Vocabulary Test - PPVT-4, and eye-tracking equipment. The PPVT-4's initial 12 cards, each displaying four images, were presented on screen for 5 s for the child's initial inspection. Subsequently, the same card was shown for 5 s with instructions to identify the picture matching the word, repeated three times in a randomized order.

Results

Eye-tracking assessments revealed that girls with RS with less severe symptoms, performed below typically developing peers with aged 3–4 years. The number of eye fixations emerged as a reliable metric for assessing their performance. A positive correlation was found between parental reports of their daughters' vocabulary knowledge and their ability to identify the correct stimulus, suggesting comprehension and active engagement in the testing process.

Conclusion

This study presents a pioneering approach by combining parental perceptions with eye-tracking measures to assess receptive vocabulary in girls diagnosed with Rett Syndrome. Despite showing lower performance levels compared to significantly younger peers, the findings suggest that these individuals use less effectively eye contact as a mode of communication.

研究目的本研究通过眼动跟踪技术评估了被诊断患有雷特综合症（Rett Syndrome，RS）的女孩的接受词汇量，并研究了这些客观测量结果与父母对女儿语言能力的看法之间的比较：方法：14 名患有雷特综合症的女孩和 11 名发育正常的同龄人参加了研究。测量工具包括克尔量表（Kerr Scale）、家长交流能力问卷、皮博迪图画词汇测验（PPVT-4）和眼动追踪设备。PPVT-4 最初的 12 张卡片（每张卡片显示四幅图像）在屏幕上显示 5 秒钟，供儿童进行初步检查。随后，同样的卡片在屏幕上显示 5 秒钟，并指示儿童识别与单词匹配的图片，以随机顺序重复三次：眼动跟踪评估结果显示，症状较轻的 RS 女童的表现低于 3-4 岁发育正常的同龄儿童。眼睛注视的次数是评估其表现的可靠指标。父母对女儿词汇知识的报告与她们识别正确刺激的能力之间存在正相关，这表明她们能够理解并积极参与测试过程：本研究开创性地将父母的看法与眼动跟踪测量相结合，以评估被诊断为雷特综合症的女孩的接受词汇量。尽管与年龄明显较小的同龄人相比，这些女孩的表现水平较低，但研究结果表明，这些人使用目光接触作为交流方式的效果较差。

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引用次数: 0

Commentary on Phase IV PROVE study: Perampanel in real-world clinical care of pediatric patients with epilepsy [Moretz K, Wheless J, Santos C, Segal E, Lancman M, Patten A, Malhotra M] 对 PROVE IV 期研究的评论：佩兰帕奈在儿科癫痫患者实际临床治疗中的应用[Moretz K、Wheless J、Santos C、Segal E、Lancman M、Patten A、Malhotra M]。

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-01 DOI: 10.1016/j.ejpn.2024.05.008

Frank M.C. Besag, Michael J. Vasey

引用次数: 0

Blood parameters in pediatric myelin oligodendrocyte glycoprotein antibody-associated disorders 小儿髓鞘少突胶质细胞糖蛋白抗体相关疾病的血液参数

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-01 DOI: 10.1016/j.ejpn.2024.04.011

Alina Peternell , Christian Lechner , Markus Breu , Martin Preisel , Mareike Schimmel , Astrid Eisenkölbl , Joachim Zobel , Eva-Maria Wendel , Markus Reindl , Kevin Rostásy , Matthias Baumann

Background and objectives

Patients with myelin oligodendrocyte glycoprotein antibody-associated disorders (MOGAD) clinically present e.g. with acute disseminated encephalomyelitis (ADEM), optic neuritis (ON), transverse myelitis (TM) or aquaporin-4-IgG (AQP4-IgG) negative neuromyelitis optica spectrum disorders (NMOSD)-like phenotypes. We aimed to analyze and compare blood parameters in children with MOGAD, AQP4-IgG-positive NMOSD (hence NMOSD), multiple sclerosis (MS) and healthy controls (HC).

Methods

We evaluated differences in complete blood counts (CBC), neutrophil-to-lymphocyte ratio (NLR), platelet-to-lymphocyte ratio (PLR), monocyte-to-lymphocyte ratio (MLR) and C-reactive protein (CRP) between these four groups and within the groups between clinical attack, acute treatment and remission.

Results

Our cohort consisted of 174 children and adolescents with a total of 550 timepoints: 66 patients had MOGAD (202 timepoints), 11 NMOSD (76 timepoints), 58 MS (219 timepoints) and 39 were HC (53 timepoints). At clinical attack, leukocyte counts were elevated in MOGAD compared to remission (p < 0.001) and compared to all other groups (p < 0.001). NLR was high in MOGAD and NMOSD, and PLR was high in NMOSD, however, after correction for multiple testing these findings did not remain significant. While glucocorticoids caused an increase of leukocyte counts and NLR in NMOSD and MS, these values remained stable during acute treatment in MOGAD. In remission, NLR normalized in MOGAD, while it stayed high in NMOSD. PLR increased in NMOSD and was significantly higher compared to all other groups.

Discussion

Some blood parameters, mainly leukocyte and differential counts, might help clinicians to evaluate disease activity, differentiate relapses from pseudo-relapses and even distinguish between different disease entities.

背景和目的髓鞘少突胶质细胞糖蛋白抗体相关性疾病（MOGAD）患者临床表现为急性播散性脑脊髓炎（ADEM）、视神经炎（ON）、横贯性脊髓炎（TM）或水光素-4-IgG（AQP4-IgG）阴性神经脊髓炎视谱系障碍（NMOSD）样表型。我们旨在分析和比较MOGAD、AQP4-IgG阳性NMOSD（因此是NMOSD）、多发性硬化症（MS）和健康对照组（HC）患儿的血液参数。方法我们评估了这四组之间以及组内临床发作、急性治疗和缓解期间全血细胞计数（CBC）、中性粒细胞与淋巴细胞比值（NLR）、血小板与淋巴细胞比值（PLR）、单核细胞与淋巴细胞比值（MLR）和 C 反应蛋白（CRP）的差异：66 名患者患有 MOGAD（202 个时间点），11 名患者患有 NMOSD（76 个时间点），58 名患者患有 MS（219 个时间点），39 名患者患有 HC（53 个时间点）。在临床发作时，与缓解期相比（p <0.001），以及与所有其他组别相比（p <0.001），MOGAD 的白细胞计数升高。MOGAD和NMOSD的NLR较高，而NMOSD的PLR较高，然而，经多重检验校正后，这些结果并不显著。在 NMOSD 和 MS 中，糖皮质激素导致白细胞计数和 NLR 增加，而在 MOGAD 中，这些数值在急性治疗期间保持稳定。在缓解期，MOGAD 的 NLR 恢复正常，而 NMOSD 的 NLR 则居高不下。讨论一些血液参数，主要是白细胞计数和差值计数，可以帮助临床医生评估疾病的活动性，区分复发和假性复发，甚至区分不同的疾病实体。

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引用次数: 0

Neurologic complications of thiamine (B1) deficiency following bariatric surgery in adolescents 青少年减肥手术后硫胺素（B1）缺乏症的神经并发症

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-01 DOI: 10.1016/j.ejpn.2024.04.008

Lital Cohen Vig , Rachel Straussberg , Noa Ziv , Lior Hirschfeld-Dicker , Osnat Konen , Sharon Aharoni

Background

The prevalence of obesity among children and adolescents is rising and poses a major health concern. Bariatric surgery is well established in adults and has become an option for adolescents. Thiamine (B1) deficiency is common following bariatric surgery in adults. It may present as Beri-Beri, Wernicke encephalopathy, or Korsakoff psychosis.

Objective

Our aim was to describe the clinical features, diagnosis, and treatment of adolescents who presented with B1 deficiency after bariatric surgery at one center, and to summarize the data from the literature.

Patients

Three adolescents with morbid obesity (two boys and one girl, aged 15.5 to- 17-years-old), presented at Schneider Children's Medical Center of Israel with progressive lower limb pain and weakness 2–3 month following a bariatric procedure (sleeve gastrectomy or narrowing of a bariatric band). The girl also had upper limb involvement and cerebellar signs. All three were non-compliant with micronutrient supplementation. After admission, they received intravenous B1 and oral multivitamin supplementation, and their symptoms improved considerably.

Conclusions

Micronutrient supplementation following bariatric surgery is crucial to prevent deficiencies. In adolescents, compliance with micronutrient supplementation should be assessed before and after such surgery. Thiamine deficiency may cause polyneuropathy, among other symptoms. Treatment reduces the severity of neurological complications.

背景儿童和青少年肥胖症发病率不断上升，已成为一个重大的健康问题。减肥手术已在成人中得到广泛应用，并已成为青少年的一种选择。成人减肥手术后常见硫胺素（B1）缺乏症。我们的目的是描述一个中心对减肥手术后出现硫胺素（B1）缺乏症的青少年的临床特征、诊断和治疗，并总结文献数据。患者三名患有病态肥胖症的青少年（两名男孩和一名女孩，年龄在 15.5 岁至 17 岁之间）在以色列施奈德儿童医疗中心接受减肥手术（袖状胃切除术或缩小减肥带）2-3 个月后出现进行性下肢疼痛和无力。这名女孩还伴有上肢受累和小脑症状。三人都不遵守补充微量营养素的规定。入院后，他们接受了静脉注射 B1 和口服多种维生素的补充治疗，症状得到了明显改善。在青少年中，应在手术前后对补充微量元素的依从性进行评估。硫胺素缺乏可能会导致多发性神经病等症状。治疗可降低神经系统并发症的严重程度。

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引用次数: 0

“Deconstructing” upper limb function in dyskinetic cerebral palsy "解构 "运动障碍型脑瘫的上肢功能。

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-01 DOI: 10.1016/j.ejpn.2024.05.005

Roser Pons, Dalivigka Zoi

引用次数: 0

Clinical correlation between disease progression and central vein sign in pediatric onset multiple sclerosis: A binational study 小儿多发性硬化症疾病进展与中央静脉征之间的临床相关性：一项两国研究

IF 3.1 3区医学 Q3 CLINICAL NEUROLOGY

European Journal of Paediatric Neurology

Pub Date : 2024-05-01 DOI: 10.1016/j.ejpn.2024.04.007

Shay Menascu , Simona Halusková , Amir Pollak , Pavel Ryska , Francesco Angelucci , David Magalashvili , Diana Guber , Arthur Yosef , Alon Kalron , Martin Valis , Michael Gurevich

Background

The central vein sign (CVS) has been proposed as a novel MRI biomarker to improve diagnosis of pediatric-onset MS (POMS). However, the role of CVS in POMS progression has yet to be discovered.

Objectives

To investigate the appearance of CVS and its correlation with POMS disease progression.

Methods

One hundred fifty-six POMS from two MS centers in Israel and Czech Republic MS centers were followed for five years. Patient assessment was performed by the Expanded Disability Status Scale (EDSS) and Annual Relapse Rate (ARR). Patients in whom at least 40 % of brain MRI lesions had CVS (“rule of 40”) were determined as CVS-positive.

Results

The total group of POMS consisted of 96 CVS-negative (61.5 %), aged 14.6 ± 1.9 years, EDSS 2.0, 75 % Interquartile Range (IQR) 1.0–3.0, disease duration (DD) 6.28 ± 0.38 years, and 60 CVS-positive (38.5 %), aged 15.1 ± 0.3 years, EDSS 2.0, IQR 1.5–3.0, DD 5.62 ± 0.13 years, were analyzed. After a three and five-year follow-up, the CVS-positive patients had higher EDSS scores than those who were CVS-negative, 2.0, IQR 1.0–2.5, vs 1.0, IQR 1.0–2.0, (p = 0.009) and 2.0, IQR 1.0–3.25 vs 1.0, IQR 1.0–2.0, (p = 0.0003), respectively. Patients with CVS-positive POMS were characterized by a significantly higher ARR (0.78 ± 0.08 vs 0.57 ± 0.04, p = 0.002). These results were confirmed in subgroups of Disease Modifying Treatments (DMT) untreated and treated patients.

Conclusion

CVS-positive POMS is characterized by higher disability progression than CVS-negative, indicating the importance of CVS in disease pathogenesis.

背景中心静脉征（CVS）被认为是一种新型磁共振成像生物标志物，可用于改善儿科多发性硬化症（POMS）的诊断。方法对以色列和捷克共和国两家多发性硬化症中心的 156 名多发性硬化症患者进行了为期五年的随访。患者评估采用扩展残疾状况量表（EDSS）和年复发率（ARR）。至少 40% 的脑 MRI 病灶有 CVS（"40% 规则"）的患者被确定为 CVS 阳性。0，75%四分位数间距（IQR）1.0-3.0，病程（DD）6.28 ± 0.38年；60例CVS阳性（38.5%），年龄15.1 ± 0.3岁，EDSS 2.0，IQR 1.5-3.0，病程（DD）5.62 ± 0.13年。经过三年和五年的随访，CVS 阳性患者的 EDSS 评分高于 CVS 阴性患者，分别为 2.0，IQR 1.0-2.5 vs 1.0，IQR 1.0-2.0（P = 0.009）和 2.0，IQR 1.0-3.25 vs 1.0，IQR 1.0-2.0（P = 0.0003）。CVS阳性POMS患者的ARR明显更高（0.78 ± 0.08 vs 0.57 ± 0.04，p = 0.002）。结论CVS阳性POMS患者的残疾进展高于CVS阴性患者，这表明CVS在疾病发病机制中的重要性。

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引用次数: 0