Journal of the Neurological Sciences最新文献_第9页

Feasibility of differentiating gait in Parkinson's disease and spinocerebellar degeneration using a pose estimation algorithm in two-dimensional video 利用二维视频中的姿势估计算法区分帕金森病和脊髓小脑变性患者步态的可行性

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-30 DOI: 10.1016/j.jns.2024.123158

{"title":"Feasibility of differentiating gait in Parkinson's disease and spinocerebellar degeneration using a pose estimation algorithm in two-dimensional video","authors":"","doi":"10.1016/j.jns.2024.123158","DOIUrl":"10.1016/j.jns.2024.123158","url":null,"abstract":"<div><h3>Background</h3><p>Although pose estimation algorithms have been used to analyze videos of patients with Parkinson's disease (PD) to assess symptoms, their feasibility for differentiating PD from other neurological disorders that cause gait disturbances has not been evaluated yet. We aimed to determine whether it was possible to differentiate between PD and spinocerebellar degeneration (SCD) by analyzing video recordings of patient gait using a pose estimation algorithm.</p></div><div><h3>Methods</h3><p>We videotaped 82 patients with PD and 61 patients with SCD performing the timed up-and-go test. A pose estimation algorithm was used to extract the coordinates of 25 key points of the participants from these videos. A transformer-based deep neural network (DNN) model was trained to predict PD or SCD using the extracted coordinate data. We employed a leave-one-participant-out cross-validation method to evaluate the predictive performance of the trained model using accuracy, sensitivity, and specificity. As there were significant differences in age, weight, and body mass index between the PD and SCD groups, propensity score matching was used to perform the same experiment in a population that did not differ in these clinical characteristics.</p></div><div><h3>Results</h3><p>The accuracy, sensitivity, and specificity of the trained model were 0.86, 0.94, and 0.75 for all participants and 0.83, 0.88, and 0.78 for the participants extracted by propensity score matching.</p></div><div><h3>Conclusion</h3><p>The differentiation of PD and SCD using key point coordinates extracted from gait videos and the DNN model was feasible and could be used as a collaborative tool in clinical practice and telemedicine.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141796259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Additional Relevant Intracranial Findings in Persons Screened with MR for Intracranial Aneurysms 通过 MR 筛查颅内动脉瘤患者的其他相关颅内检查结果

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-30 DOI: 10.1016/j.jns.2024.123160

{"title":"Additional Relevant Intracranial Findings in Persons Screened with MR for Intracranial Aneurysms","authors":"","doi":"10.1016/j.jns.2024.123160","DOIUrl":"10.1016/j.jns.2024.123160","url":null,"abstract":"<div><h3>Background</h3><p>Radiological screening for intracranial aneurysms (IAs) may identify other relevant intracranial findings. We investigated their prevalence on MR in persons screened for IAs.</p></div><div><h3>Methods</h3><p>We included all persons who were screened for the presence of IAs with brain MRI/MRA between 1996 and 2022 because of a family history of aneurysmal subarachnoid haemorrhage (aSAH) or autosomal dominant polycystic kidney disease (ADPKD). We reviewed radiology reports of initial and repeated brain MR to identify additional intracranial findings that needed follow-up or treatment, or carried a risk of becoming symptomatic.</p></div><div><h3>Results</h3><p>We included 766 persons (positive family history of aSAH: <em>n</em> = 681; ADPKD: <em>n</em> = 85) who had 1446 MRI/MRAs. At initial screening, 49 additional relevant intracranial findings were reported in 47 persons (6.1%, 95% CI 4.7–8.1%). Of all included persons, 338 (44%) underwent one (<em>n</em> = 154) or more (<em>n</em> = 184) follow-up screenings (total MRI/MRAs at follow-up: <em>n</em> = 680). In 15/338 persons (4.4%, 95% CI 2.7–7.2%), 16 new additional relevant findings were reported at a median follow-up duration of 10 years (IQR 5–12).</p></div><div><h3>Conclusions</h3><p>Persons who are counselled for screening for IAs should be informed that there is a six percent chance of identifying an additional finding that requires follow-up or treatment, or may become symptomatic. Additionally, after 10-year follow-up screening there is a four percent chance of identifying a new additional relevant finding. The impact of such findings on quality of life needs further study.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0022510X24002958/pdfft?md5=ac7bacc97205b7812b2843ae023ae0c0&pid=1-s2.0-S0022510X24002958-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141796268","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Factor XI inhibitors – Rising stars in anti-thrombotic therapy? 因子 XI 抑制剂--抗血栓治疗的新星？

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-29 DOI: 10.1016/j.jns.2024.123157

{"title":"Factor XI inhibitors – Rising stars in anti-thrombotic therapy?","authors":"","doi":"10.1016/j.jns.2024.123157","DOIUrl":"10.1016/j.jns.2024.123157","url":null,"abstract":"<div><p>The “holy grail” of preventing and treating thrombosis and thromboembolism would be a drug that was highly effective (preventing clots) and at the same time had a low risk of bleeding. From a hemostasiological perspective, the inhibition of factor XI represents a promising target because a reduced level of factor XI protects against thrombosis without significantly increasing the risk of spontaneous bleeding.</p><p>Currently, three different classes of drugs of factor XI-inhibition are tested. These are (1) monoclonal antibodies (mAbs), (2) so-called synthetic, small molecules and (3) antisense oligonucleotides (ASOs). This article provides a narrative overview of the current status of studies on all three classes of drugs.</p><p>Tests with mAbs have been conducted primarily in DVT prevention after knee replacement surgery. One large phase 3 study is testing the mAbs Abelacimab in patients with atrial fibrillation. The synthetic, small molecules Asundexian and Milvexian are tested in several phase 3 trials, mainly in patients with non-cardioembolic ischemic stroke. Results can be expected in the coming years. Clinical testing of ASOs to inhibit factor XI are still in their infancies.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0022510X24002922/pdfft?md5=51373c83185167fe86eaa1598e9b023b&pid=1-s2.0-S0022510X24002922-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141796511","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Impact of obese body mass index on inflammasome blood biomarkers and neurocognitive performance following traumatic brain injury with Glasgow coma scale 13 to 15 肥胖体重指数对格拉斯哥昏迷量表 13 至 15 级创伤性脑损伤后炎性体血液生物标志物和神经认知能力的影响

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-29 DOI: 10.1016/j.jns.2024.123159

{"title":"Impact of obese body mass index on inflammasome blood biomarkers and neurocognitive performance following traumatic brain injury with Glasgow coma scale 13 to 15","authors":"","doi":"10.1016/j.jns.2024.123159","DOIUrl":"10.1016/j.jns.2024.123159","url":null,"abstract":"<div><p>Activation of the NOD-like receptor family pyrin domain-containing 3 (NLRP3) inflammasome is a moderating factor between obesity and cognitive impairment in animals, but this has never been tested in humans following mild traumatic brain injury (mTBI). This is a retrospective cohort analysis of subjects enrolled at a single level 1 trauma center (<em>n</em> = 172). Participants completed Trail Making Test Part A and B (TMT-A and <img>B) at six- and twelve-months, Blood samples were obtained within 24 h of mTBI and apoptosis-associated speck-like protein containing a caspase recruitment domain (ASC), caspase-1, interleukin-18 (IL-18), and IL-1β were assayed. Obese participants (BMI = 30–34.9) were associated with higher IL-18 (<em>p</em> = 0.03) and IL-1β (<em>p</em> = 0.05) and severely obese participants (BMI > 35.0) were associated with higher IL-1β (<em>p</em> = 0.005) than healthy weight participants. IL-1β was associated with TMT-A at six- (<em>p</em> = 0.01) and twelve-months (p = 0.03) and TMT-B at twelve-months (<em>p</em> = 0.046). The interaction of severely obese BMI and IL-1β was associated with TMT-B at six- (<em>p</em> = 0.049) and twelve-months (<em>p</em> = 0.02). ASC (p = 0.03) and the interaction of ASC with severely obese BMI was associated with TMTB at six- (p = 0.02) and twelve-months (p = 0.02). Obesity may augment acute inflammasome response to mTBI and influence worse long-term cognitive outcomes up to one-year post-injury.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141796371","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comorbid onset of cardiovascular diagnosis and long-term confirmed disability progression in multiple sclerosis: A 15-year follow-up study 多发性硬化症患者合并心血管疾病诊断和长期确诊残疾进展：15 年随访研究

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-29 DOI: 10.1016/j.jns.2024.123156

{"title":"Comorbid onset of cardiovascular diagnosis and long-term confirmed disability progression in multiple sclerosis: A 15-year follow-up study","authors":"","doi":"10.1016/j.jns.2024.123156","DOIUrl":"10.1016/j.jns.2024.123156","url":null,"abstract":"<div><h3>Background</h3><p>People with multiple sclerosis (pwMS) have greater prevalence of comorbid cardiovascular diseases (CVD) when compared to the general population despite similar frequency of CV risk factors.</p></div><div><h3>Objective</h3><p>Determine the impact of comorbid-onset of CVD diagnosis on long-term confirmed disability progression (CDP).</p></div><div><h3>Methods</h3><p>276 pwMS (29 clinically isolated syndrome, 130 relapsing-remitting and 117 progressive) were clinically followed an average of 14.9 years, with a mean of 14.4 clinical visits. Retrospective electronic medical records (EMR) review determined CVD diagnoses (hypertension, hyperlipidemia, diabetes, and heart disease) at baseline and over the follow-up. CDP was determined with ≥1.0 point Expanded Disability Status Scale (EDSS) increase from EDSS <5.5, or ≥ 0.5-point increase from ≥5.5, and was sustained on next clinical visit.</p></div><div><h3>Results</h3><p>A significantly shorter time to overall CDP was detected in 213 pwMS who had an existing (28 pwMS) or developed new onset (185 pwMS) of CVD, compared to 63 CVD-healthy pwMS over the follow-up (13.4 vs 15.9 years, Mantel-Cox <em>p</em> < 0.001), independent of baseline age and EDSS score. The CVD diagnosis preceded the CDP in 103 pwMS (55.7%), occurred after CDP in 71 pwMS (38.4%) and was concurrent in 11 pwMS (5.9%). Using mixed-effect models adjusted for significant age (F = 56.5, <em>p</em> < 0.001) and time effects (F = 67.8, p < 0.001), the CVD-onset diagnosis was associated with greater accrual of disability, as measured by longitudinal increase in EDSS score (F = 4.207, <em>p</em> = 0.04). Sex was not significant predictor of future disability in our cohort.</p></div><div><h3>Conclusion</h3><p>PwMS with an existing or new onset of comorbid CVD diagnosis showed accelerated disability worsening over long-term. There was no temporal relationship between the onset of CVD and CDP within the group that had CVD-onset diagnosis.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141796387","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Chorioretinopathy presenting as bitemporal hemianopia 表现为位颞侧偏盲的脉络膜视网膜病变

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-27 DOI: 10.1016/j.jns.2024.123151

{"title":"Chorioretinopathy presenting as bitemporal hemianopia","authors":"","doi":"10.1016/j.jns.2024.123151","DOIUrl":"10.1016/j.jns.2024.123151","url":null,"abstract":"<div><h3>Introduction</h3><p>Bitemporal hemianopia is usually caused by chiasmal pathology. Rarely, chorioretinal lesions may develop symmetrically in both eyes and mimic chiasmopathy.</p></div><div><h3>Methods</h3><p>This case series included three patients who presented to a tertiary neuro-ophthalmology centre with bitemporal hemianopic defects between 2021 and 2023 and were subsequently diagnosed with bilateral chorioretinopathy. All patients received comprehensive examinations from a fellowship-trained neuro-ophthalmologist and uveitis specialist to rule out other causes of visual dysfunction.</p></div><div><h3>Results</h3><p>Three males aged 64, 62, and 72 years were included. All patients showed bitemporal hemianopic defects crossing the vertical midline on automated perimetry and binasal thinning of the macular ganglion cell complex on spectral-domain optical coherence tomography (OCT). Fundus autofluorescence (FAF) showed classical features of acute zonal occult outer retinopathy (AZOOR) in two patients and central serous chorioretinopathy (CSCR) in another. AZOOR diagnosis was preceded by neuroimaging in both cases, whereas the patient with CSCR had longstanding, electroretinography-confirmed lesions and did not require neuroimaging. Fundus appearance and visual field defects remained stable in all patients across 3–6 months of follow-up.</p></div><div><h3>Conclusions</h3><p>Bilateral chorioretinopathy should be considered in the differential diagnosis of bitemporal hemianopia in specific cases, including when visual field defects cross the vertical midline and when neuroimaging fails to reveal chiasmal pathology. FAF and macular OCT have high diagnostic yield as initial investigations.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141847474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Robust epidemiological evidence supports the association between nodding syndrome and epilepsy 大量流行病学证据支持点头综合征与癫痫之间的关联

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-26 DOI: 10.1016/j.jns.2024.123152

引用次数: 0

Indications for a brain biopsy in neurological diseases of unknown etiology: The role of magnetic resonance imaging findings and liquid biopsy in yielding definitive pathological diagnoses 病因不明的神经系统疾病的脑活检指征：磁共振成像结果和液体活检在明确病理诊断中的作用。

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-24 DOI: 10.1016/j.jns.2024.123150

{"title":"Indications for a brain biopsy in neurological diseases of unknown etiology: The role of magnetic resonance imaging findings and liquid biopsy in yielding definitive pathological diagnoses","authors":"","doi":"10.1016/j.jns.2024.123150","DOIUrl":"10.1016/j.jns.2024.123150","url":null,"abstract":"<div><p>Brain biopsies are often considered for patients who cannot be diagnosed with various laboratory test results. However, physicians tend to be hesitant regarding their application in possibly non-neoplastic brain diseases, due to the invasiveness and risks. The aim was to determine the indications for brain biopsies in cases of neurological diseases of unknown etiology. We retrospectively evaluated diagnostic accuracy, laboratory findings (including a liquid biopsy for malignant lymphoma), magnetic resonance imaging (MRI) characteristics and the post-treatment outcomes of patients undergoing brain biopsies for neurological diseases of unknown etiology. The data of patients who had undergone a brain biopsy during their admission to Niigata University Hospital, between 2011 and 2024, were reviewed. Moreover, the laboratory data and MRI findings between patients with definitive and nonspecific biopsy diagnoses were compared. Twenty-six patients underwent a brain biopsy, and a definitive diagnosis was obtained in 14 patients (53.8%). Even in cases where a nonspecific diagnosis was made, biopsy findings helped rule out malignancy and guide clinical diagnosis and treatment decisions. The liquid biopsy for malignant lymphoma was performed in eight patients, with one yielding a positive result, consistent with primary central nervous system lymphoma. The sensitivity and specificity of liquid biopsy were 0.5 and 1, respectively. Diffusely contrasted cortical lesions and the presence of mass effects on MRI, were significantly associated with a definitive diagnosis, compared to a nonspecific diagnosis. In conclusion, brain MRI and liquid biopsies can assist in determining the appropriate indications for brain biopsies in neurological diseases of unknown etiology.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141788504","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Age-associated gadolinium leakage into ocular structures in patients with acute traumatic brain injury 急性脑外伤患者眼部结构中的钆渗漏与年龄有关

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-22 DOI: 10.1016/j.jns.2024.123149

{"title":"Age-associated gadolinium leakage into ocular structures in patients with acute traumatic brain injury","authors":"","doi":"10.1016/j.jns.2024.123149","DOIUrl":"10.1016/j.jns.2024.123149","url":null,"abstract":"<div><h3>Background</h3><p>Gadolinium Leakage into Ocular Structures (GLOS) is common following acute cerebrovascular events. The objective of this study was to investigate the occurrence of GLOS in an acute traumatic brain injury (TBI) cohort without acute cerebrovascular injury and to explore associated factors.</p></div><div><h3>Methods</h3><p>Enrolled acute TBI patients had a baseline MRI ≤48 h of injury (TP1) and follow-up MRI ≤72 h after baseline (TP2). Vitreous chamber enhancement and signal intensity ratios (SIRs) were calculated using pre- and post-contrast Fluid Attenuated Inversion Recovery (FLAIR). White matter hyperintensities (WMHs) were assessed using the Fazekas scale.</p></div><div><h3>Results</h3><p>Of the 128 TBI patients included, median age was 47 years, 70% male, and 66% presented with Glasgow Coma Scale of 15. No GLOS was detected at TP1 but was present in 23% of patients at TP2. GLOS+ patients were older (68 years [56–76] vs 39 years [27–53], <em>p</em> < 0.001), more likely to report falls as injury mechanism (62% vs 36%, <em>p</em> = 0.006), report history of hypertension (41% vs 19%, <em>p</em> = 0.025), and had a higher burden of WMHs (59% vs 14% with a total Fazekas ≥2, <em>p</em> < 0.001). Quantitative SIRs confirmed qualitative assessments: GLOS+ patients had higher SIRs at TP2 (0.43 vs 0.22, <em>p</em> < 0.001). Age (OR 3.28, 95%CI [1.88–5.71], p < 0.001) and prior TBI history (OR 4.99, 95%CI [1.46–17.06], <em>p</em> = 0.010) were independent predictors of GLOS. When age was removed, total Fazekas score (OR 2.53, 95%CI [1.60–4.00], <em>p</em> < 0.001) was an independent predictor of GLOS.</p></div><div><h3>Conclusions</h3><p>GLOS is primarily associated with age and may serve as another imaging marker of chronic vascular disease.</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141851835","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind, placebo-controlled pilot study 三氯匹林（CER-0001）用于偏头痛的预防性治疗：第 2 期随机、双盲、安慰剂对照试验研究

IF 3.6 3区医学

Journal of the Neurological Sciences Pub Date : 2024-07-22 DOI: 10.1016/j.jns.2024.123147

{"title":"Tricaprilin (CER-0001) for the preventive treatment of migraine: A phase 2 randomised, double-blind, placebo-controlled pilot study","authors":"","doi":"10.1016/j.jns.2024.123147","DOIUrl":"10.1016/j.jns.2024.123147","url":null,"abstract":"<div><h3>Background</h3><p>Increasing evidence indicates a metabolic etiology for migraines, with ketosis potentially rectifying metabolic and clinical features. We conducted a pilot study to evaluate CER-0001, a ketogenic agent, for migraine prevention without dietary changes.</p></div><div><h3>Methods</h3><p>This was a 2-part, double-blind, randomised, placebo-controlled study conducted in Australia. Adults with at least a 1-year history of migraine and ≥ 1 prior preventive treatment failure were randomised to either oral CER-0001 (up to 30 g twice a day) or placebo for 12 weeks. The primary endpoint was Month 3 change in Migraine Headache Days from baseline.</p></div><div><h3>Results</h3><p>Part 1 results are presented. 81 participants were randomised and dosed (<em>n</em> = 40 CER-0001, <em>n</em> = 41 placebo), and 61 participants had evaluable efficacy data. No statistically significant difference was observed in the primary endpoint (LSMean difference 0.92 days; <em>p</em> = 0.586). During Month 2, a mean improvement of −2.8 days was observed for CER-0001 (<em>p</em> = 0.056). Withdrawal rates were 45.0% and 53.7% (CER-0001; placebo). The proportion of participants reporting at least one treatment-emergent adverse event was similar between arms (90.0% CER-0001, 82.9% placebo), mostly gastrointestinal (85.0% CER-0001, 70.7% placebo).</p></div><div><h3>Conclusion</h3><p>Results suggest positive directional promise over 2–3 months for CER-0001. A new formulation will be used for larger, fully powered phase 2/3 studies.</p></div><div><h3>Trial registration</h3><p>This study is registered at <span><span>ClinicalTrials.gov</span><svg><path></path></svg></span> (<span><span>NCT04437199</span><svg><path></path></svg></span>).</p></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141847372","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0