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Physical, Mental, and Social Characteristics Associated With Happiness in Individuals With Schizophrenia in Japan: A Cross-Sectional Study. 日本精神分裂症患者与幸福相关的生理、心理和社会特征:一项横断面研究。
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70045
Junna Hattori, Masaaki Matsunaga, Yupeng He, Kenji Sakuma, Taro Kishi, Shinichi Tanihara, Nakao Iwata, Atsuhiko Ota

Objective: To examine the characteristics associated with happiness in Japanese individuals with schizophrenia.

Methods: A self-reported online survey was conducted in 2022 among individuals aged 20-75 years, including 223 and 1776 individuals with and without schizophrenia, respectively. We used a modified Poisson regression to assess the factors associated with happiness by calculating the age- and sex-adjusted prevalence ratios (PRs). We examined within-schizophrenia group differences by age and sex strata, and compared these stratified PRs between groups with and without schizophrenia.

Results: Among participants with schizophrenia, happiness was significantly associated with self-rated health status (PR = 1.75), Ikigai (PR = 5.02), depressive symptoms (PR = 0.43), perceived stress (PR = 0.52), cognitive social capital (PR = 2.07), structural social capital (PR = 1.70), social support (PR = 2.40), close friends (PR = 1.88), close relatives (PR = 2.34), and a cohabiting partner (PR = 1.57). Within the schizophrenia group, sex differences were significant for cognitive social capital (men: PR = 3.45; women: PR = 1.43) and cohabiting partners (men: PR = 2.26; women: PR = 1.25), whereas no significant age differences were found. Factors demonstrating a stronger association in participants with schizophrenia than in those without schizophrenia included: Ikigai (with, PR = 5.02; without, PR = 2.91), cognitive social capital (with, PR = 2.07; without, PR = 1.49), and structural social capital (with, PR = 1.70; without, PR = 1.24).

Conclusion: Happiness in individuals with schizophrenia is associated with physical, mental, and social factors, with social factors exhibiting sex-related differences.

目的:研究日本精神分裂症患者与幸福感相关的特征。方法:在2022年对20-75岁的个体进行了一项自我报告的在线调查,其中分别包括223名和1776名患有和非精神分裂症的个体。我们通过计算年龄和性别调整的患病率比率(pr),使用修正泊松回归来评估与幸福相关的因素。我们检查了精神分裂症组内年龄和性别阶层的差异,并比较了精神分裂症组和非精神分裂症组之间这些分层的pr。结果:在精神分裂症患者中,幸福感与自评健康状况(PR = 1.75)、Ikigai (PR = 5.02)、抑郁症状(PR = 0.43)、感知压力(PR = 0.52)、认知社会资本(PR = 2.07)、结构性社会资本(PR = 1.70)、社会支持(PR = 2.40)、亲密朋友(PR = 1.88)、近亲(PR = 2.34)和同居伴侣(PR = 1.57)显著相关。在精神分裂症组中,认知社会资本(男性:PR = 3.45;女性:PR = 1.43)和同居伴侣(男性:PR = 2.26;女性:PR = 1.25)的性别差异显著,而年龄差异不显著。精神分裂症患者比非精神分裂症患者表现出更强相关性的因素包括:Ikigai(有,PR = 5.02;没有,PR = 2.91)、认知社会资本(有,PR = 2.07;没有,PR = 1.49)和结构性社会资本(有,PR = 1.70;没有,PR = 1.24)。结论:精神分裂症患者的幸福感与生理、心理和社会因素有关,其中社会因素表现出性别差异。
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引用次数: 0
Successful Early Treatment of Anti-N-Methyl-d-Aspartate Receptor Encephalitis Associated With Small Cell Lung Cancer in an Elderly Male Patient: A Case Report. 抗n -甲基-d-天冬氨酸受体脑炎合并小细胞肺癌的早期成功治疗1例
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70044
Kohei Kamikawa, Ryohei Takada, Yuya Honda, Harue Goto, Takashi Okada

Background: Anti-N-methyl-d-aspartate receptor (NMDAR) encephalitis, a type of autoimmune encephalitis, characterized by acute onset neuropsychiatric symptoms, predominantly affects young females and is often associated with ovarian teratomas. Although small cell lung cancer (SCLC) is a known cause of paraneoplastic encephalitis, its association with anti-NMDAR encephalitis is rare and often carries a poor prognosis due to limited response to immunotherapy.

Case presentation: An 80-year-old male with no psychiatric history presented with flu-like symptoms, followed by the acute onset of neuropsychiatric symptoms, including pressured speech, agitation, memory impairment, and abnormal behavior. Autoimmune encephalitis was suspected due to mildly elevated cerebrospinal fluid (CSF) white cell count and a mass in the right upper lung detected by whole-body computed tomography (CT) on the first day of hospitalization. High-dose intravenous corticosteroids were administered on Day 1, resulting in prompt and sustained improvement in symptoms. CSF was later confirmed positive for anti-NMDAR antibodies, and a bronchoscopy biopsy of the pulmonary mass diagnosed SCLC. The patient recovered without neurological deficits and was discharged in stable condition on hospital Day 30.

Conclusion: This was a rare case of anti-NMDAR encephalitis associated with SCLC in an elderly male patient. Diagnosis in elderly individuals is often challenging because of the atypical presentations and lower tumor association. Nevertheless, timely intervention initiation may lead to favorable outcomes. Clinicians should consider autoimmune encephalitis, including anti-NMDAR encephalitis, when evaluating acute onset neuropsychiatric symptoms in elderly individuals and initiate early immunotherapy alongside tumor screening.

背景:抗n -甲基-d-天冬氨酸受体(NMDAR)脑炎是一种自身免疫性脑炎,以急性发作的神经精神症状为特征,主要影响年轻女性,常伴有卵巢畸胎瘤。虽然小细胞肺癌(SCLC)是一种已知的副肿瘤脑炎的病因,但它与抗nmdar脑炎的关联是罕见的,并且由于对免疫治疗的反应有限,通常预后较差。病例介绍:80岁男性,无精神病史,出现流感样症状,随后出现急性神经精神症状,包括言语压力、躁动、记忆障碍和异常行为。住院第一天全身计算机断层扫描(CT)发现脑脊液(CSF)白细胞计数轻度升高和右上肺肿块,怀疑为自身免疫性脑炎。在第1天给予大剂量静脉注射皮质类固醇,导致症状迅速和持续改善。脑脊液后来证实抗nmdar抗体阳性,肺肿块的支气管镜活检诊断为SCLC。患者康复后无神经功能缺损,入院第30天病情稳定出院。结论:这是一例罕见的老年男性患者抗nmdar脑炎合并SCLC的病例。由于不典型的表现和较低的肿瘤相关性,老年人的诊断往往具有挑战性。然而,及时的干预可能会导致良好的结果。临床医生在评估老年人急性发作的神经精神症状时应考虑自身免疫性脑炎,包括抗nmdar脑炎,并在肿瘤筛查的同时进行早期免疫治疗。
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引用次数: 0
Clinical Characteristics of Patients With Enlarged Ventricles and Cognitive Impairment (EVCI): Case Series. 脑室增大合并认知障碍(EVCI)患者的临床特征:病例系列。
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70029
Yuka Yasuda, Satsuki Ito, Junya Matsumoto, Toshiaki Onitsuka, Hidenaga Yamamori, Michiko Fujimoto, Naomi Hasegawa, Manabu Ikeda, Ryota Hashimoto

Background: Since research on the pathophysiology of psychiatric disorders diagnosed by symptoms has not succeeded, a data-driven analysis incorporating biological and cross-disease perspectives has been proposed. We have reported a new subgroup of psychiatric disorders by a data-driven analysis of subcortical volumes of brain MRI in 5602 subjects, including patients with psychiatric disorders and controls. This subgroup of patients is characterized by enlarged ventricle and cognitive impairment (EVCI) with a high proportion of schizophrenia, electroencephalography abnormalities, and rare pathological copy number variations.

Case presentation: Of the nine patients with EVCI, eight patients had schizophrenia, and one patient had autism spectrum disorder. Early onset of age was observed in eight patients with schizophrenia. Treatment responses were poor in seven patients with schizophrenia, and two of three treatment-resistant schizophrenia patients responded to clozapine. Four patients showed ischemic changes in cerebral white matter. In electroencephalography, abnormal findings were observed in five patients, borderline findings in two patients, and normal findings in two patients. Rare pathogenic copy number variations were found in three patients (22q11.21 deletion, 7q11.23 duplication, and 7q36.2 deletion).

Conclusions: The results of this case series showed additional clinical features of treatment response and ischemic changes in cerebral white matter, which could be a clue to the treatment and diagnosis of EVCI. This case series might help elucidate the pathophysiology of EVCI.

背景:由于通过症状诊断精神障碍的病理生理学研究尚未成功,因此提出了一种结合生物学和跨疾病观点的数据驱动分析。我们报告了一个新的精神疾病亚组,通过对5602名受试者(包括精神疾病患者和对照组)的脑MRI皮质下体积的数据驱动分析。该亚组患者的特点是脑室增大和认知障碍(EVCI),精神分裂症的比例高,脑电图异常,罕见的病理拷贝数变异。病例介绍:在9例EVCI患者中,8例患有精神分裂症,1例患有自闭症谱系障碍。在8例精神分裂症患者中观察到年龄早发。七名精神分裂症患者的治疗反应较差,三名治疗抵抗的精神分裂症患者中有两名对氯氮平有反应。4例患者出现脑白质缺血性改变。脑电图异常5例,交界性2例,正常2例。在3例患者中发现罕见的致病性拷贝数变异(22q11.21缺失、7q11.23重复和7q36.2缺失)。结论:本病例系列的结果显示了治疗反应和脑白质缺血性改变的额外临床特征,可为EVCI的治疗和诊断提供线索。本病例系列可能有助于阐明EVCI的病理生理学。
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引用次数: 0
Clinical Characteristics of Patients With Cannabis-Related Mental Disorders and an Examination of Factors Influencing Their Access to Medical and Nonmedical Resources: Comparison of Methamphetamine-Related Mental Disorders. 大麻相关精神障碍患者的临床特征及影响其获得医疗和非医疗资源的因素:甲基苯丙胺相关精神障碍的比较
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70051
Toshihiko Matsumoto, Takashi Usami, Akiho Nishimura, Sayako Higuchi, Kyoji Okita, Takuya Shimane

Purpose: This study aims to identify the characteristics of patients with cannabis use disorder and to determine whether the challenges in treating cannabis use disorder stem from the pharmacological properties of cannabis as an abused substance or from other contributing factors.

Methods: The subjects of this study were the 208 male cases of methamphetamine-related mental disorders (MAP group) and 82 male cases of cannabis-related mental disorders (CAN group), which drawn from the "2024 Nationwide Survey on the Actual Conditions of Drug-Related Mental Disorders in Psychiatric Hospitals in Japan." Clinical variables were compared between the two groups, and logistic regression analyses were conducted to examine the use of medical and nonmedical resources.

Results: The CAN group was found to be younger, to have fewer histories of drug-related criminal offenses, incarceration in correctional facilities, or comorbid psychiatric disorders, compared to the MAP group, and to exhibit less severe forms of substance use disorder and have less experience participating in self-help groups or utilizing private recovery support facilities. Logistic regression analyses of the use of self-help groups and private recovery support facilities indicated that the utilization of these nonmedical resources was more strongly associated with older age and greater severity of substance use disorder than with the specific type of substance abused.

Conclusion: Recently, an increasing number of young patients in Japan have been arrested for cannabis-related offenses and seek addiction treatment. There is growing concern that current nonmedical support resources may not adequately address the specific needs of these individuals.

目的:本研究旨在确定大麻使用障碍患者的特征,并确定治疗大麻使用障碍的挑战是否源于大麻作为滥用物质的药理学特性或其他因素。方法:选取《2024年日本精神病院药物相关精神障碍实际情况调查》中208例男性甲基苯丙胺相关精神障碍(MAP组)和82例男性大麻相关精神障碍(CAN组)为研究对象。比较两组的临床变量,并进行logistic回归分析,以检查医疗和非医疗资源的使用情况。结果:与MAP组相比,CAN组被发现更年轻,有更少的与毒品有关的犯罪史,在惩教机构的监禁,或共病精神障碍,表现出更少的严重形式的物质使用障碍,较少参与自助团体或使用私人康复支持设施的经验。对自助团体和私人康复支持设施使用情况的Logistic回归分析表明,这些非医疗资源的使用与年龄和物质使用障碍的严重程度的关系比与滥用物质的具体类型的关系更强。结论:最近,日本越来越多的年轻患者因大麻相关犯罪而被捕,并寻求成瘾治疗。人们越来越担心,目前的非医疗支助资源可能无法充分满足这些人的具体需求。
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引用次数: 0
Amelioration of Adults' Mental Health Conditions and Symptoms Through Spiritual Energy Therapy: Randomized Controlled Trial. 通过精神能量疗法改善成人心理健康状况和症状:随机对照试验。
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70050
Mahendra Kumar Trivedi, Alice Branton, Dahryn Trivedi, Sambhu Mondal, Snehasis Jana

Reduced physical activities, social support, old age, and increased exposure to regular stressful life events are crucial factors for poor mental health. Although several medicines are available to support mental health-related symptoms, due to more side effects and non-compliance for long-term use, these conventional medicines are not appropriate to tackle these symptoms. In this context, the study aimed to determine whether spiritual energy therapy with an experienced spiritual energy practitioner could manage adults' psychological and mental health-related symptoms. A single-blind, active-controlled, randomized trial was performed. Enrolled participants (n = 80) exhibited at least one psychological symptom. Spiritual energy therapy was given to subjects at two time points (Days 0 and 90) in physical presence. Psychological scores and physiological biomarkers were assessed. Treatment group subjects were reported to have a significant (p ≤ 0.0001) improvement in psychological scores related to stress, depression, mental restlessness, emotional trauma, hopelessness, etc., compared to the control group subjects. Additionally, subjects in the treatment group also showed significant improvement in biological markers compared to those in the control group. No treatment-related adverse effects were observed. The current study data unveiled a significant effect of spiritual energy therapy on improving adults' mental health conditions and symptoms, and significantly decreased elevated proinflammatory and oxidative stress biomarker levels in the treatment group.

体力活动减少、社会支持减少、年龄增大以及经常接触有压力的生活事件是导致心理健康状况不佳的关键因素。虽然有几种药物可用于治疗与精神健康有关的症状,但由于副作用较多且长期使用不符合规定,这些常规药物不适合治疗这些症状。在此背景下,本研究旨在确定经验丰富的精神能量从业者的精神能量疗法是否可以管理成年人的心理和精神健康相关症状。进行了一项单盲、主动对照、随机试验。入选的参与者(n = 80)表现出至少一种心理症状。精神能量疗法在两个时间点(第0天和第90天)进行。评估心理评分和生理生物标志物。与对照组受试者相比,治疗组受试者在应激、抑郁、精神不安、情绪创伤、绝望等方面的心理评分均有显著改善(p≤0.0001)。此外,与对照组相比,治疗组的受试者在生物标志物方面也有显著改善。未观察到与治疗相关的不良反应。目前的研究数据揭示了精神能量疗法对改善成人心理健康状况和症状的显着作用,并显着降低治疗组中升高的促炎和氧化应激生物标志物水平。
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引用次数: 0
Clarifying the Gateway Hypothesis and Conflict of Interest in Cannabis Research. 澄清大麻研究中的门户假说和利益冲突。
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70056
Kenjiro Shiraishi

This image visualizes the weighing of commercial incentives against research integrity, highlighting the challenge of maintaining transparency in CBD-related studies.

该图像可视化了商业激励与研究诚信之间的权衡,突出了在cbd相关研究中保持透明度的挑战。
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引用次数: 0
Effect of Lemborexant-Based Sleep Medication Formulary on Benzodiazepine Reduction and Clinical Outcomes: A Single-Center Retrospective Study. 以lemborexant为基础的睡眠药物处方对苯二氮卓类药物减少和临床结果的影响:一项单中心回顾性研究
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70054
Shunya Aoki, Katsutoshi Takada, Tatsuru Sugama, Mitsugi Kimiwada, Tatsuya Hoshino, Kaori Koike, Hirokazu Akada, Takahisa Saiga, Shigeki Sato, Ryosuke Shinkai, Yukihiro Shibata, Takashi Tomita

Benzodiazepine and non-benzodiazepine hypnotics (Z-drugs) are known risk factors for adverse events, including delirium and falls. Although formularies are intended to promote appropriate prescribing, few comprehensive studies have assessed their clinical impact in the context of sleep medications. This study aimed to evaluate changes in hypnotic prescribing patterns and associated clinical outcomes following the implementation of a sleep medication formulary. A psychiatric liaison team developed and implemented a formulary in April 2024, recommending lemborexant as the first-line treatment and eszopiclone as the second-line option. This single-center, retrospective study compared patients admitted and discharged during the 12 months before (April 2023 to March 2024; n = 12 633) and after (April 2024 to March 2025; n = 12 931) implementation. Outcome measures included monthly prescription volumes, diazepam equivalents, use in clinical pathways and prescription sets, delirium incidence, nighttime falls, and length of hospital stay. Statistical analyses were performed using the Mann-Whitney U-test and Fisher's exact test. Following implementation, prescription volumes of lemborexant and eszopiclone increased significantly, whereas diazepam equivalents decreased from 10 682 mg to 4117 mg. All 104 clinical pathways and prescription sets previously using benzodiazepine hypnotics or Z-drugs were converted to lemborexant. Monthly delirium cases declined from 12.5 to 8.0, and the proportion of nighttime falls among patients receiving benzodiazepine hypnotics or Z-drugs decreased from 24.0% to 11.5%. The median hospital stay also decreased from 8 to 7 days. These findings suggest that formulary implementation effectively optimized hypnotic prescribing and contributed to improved clinical outcomes and patient safety in an acute care setting.

苯二氮卓类和非苯二氮卓类催眠药(z -药物)是已知的不良事件的危险因素,包括谵妄和跌倒。虽然处方集旨在促进适当的处方,但很少有综合研究评估它们在睡眠药物方面的临床影响。本研究旨在评估催眠处方模式的变化和睡眠药物处方实施后的相关临床结果。精神病学联络小组于2024年4月制定并实施了一份处方,建议将lemborexant作为一线治疗,将eszopiclone作为二线选择。这项单中心回顾性研究比较了实施前(2023年4月至2024年3月,n = 12 633)和实施后(2024年4月至2025年3月,n = 12 931) 12个月内入院和出院的患者。结果测量包括月处方量、地西泮当量、临床途径和处方组的使用、谵妄发生率、夜间跌倒和住院时间。采用Mann-Whitney u检验和Fisher精确检验进行统计分析。实施后,lemborexant和eszopiclone的处方量显著增加,而地西泮当量从10682毫克减少到4117毫克。将先前使用苯二氮卓类催眠药或z类药物的104条临床路径和处方集全部转换为lemborexant。每月谵妄病例从12.5例下降到8.0例,服用苯二氮卓类安眠药或z类药物的患者夜间跌倒的比例从24.0%下降到11.5%。中位住院时间也从8天减少到7天。这些发现表明,处方的实施有效地优化了催眠处方,并有助于改善急性护理环境中的临床结果和患者安全。
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引用次数: 0
Correction to "Clinical Characteristics of Patients With Enlarged Ventricles and Cognitive Impairment (EVCI): Case Series". 修正了“脑室增大和认知障碍(EVCI)患者的临床特征:病例系列”。
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70058
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引用次数: 0
Reporting Frequency of Antipsychotics-Induced Tardive Dyskinesia and Other Extrapyramidal Symptoms: Analysis Based on a Spontaneous Reporting System Database in Japan. 抗精神病药物引起的迟发性运动障碍和其他锥体外系症状的报告频率:基于日本自发报告系统数据库的分析
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70049
Yosuke Saga, Hiroshi Horio, Chih-Lin Chiang, Akihide Wakamatsu

First- and second-generation antipsychotics (FGAs and SGAs, respectively) with dopamine-antagonizing properties may cause involuntary movement-related adverse drug reactions (ADRs). However, the risk in the Japanese population is not well characterized. In this study, we analyzed spontaneous ADR reports from the Japanese Adverse Drug Event Report (JADER) database and evaluated the reporting odds ratios (RORs) of tardive dyskinesia (TD) and other extrapyramidal symptoms (EPS) associated with antipsychotics. SGAs were evaluated both as a whole class and as subgroups based on their primary pharmacological mode of action. From 1 April 2011 to 31 March 2020, 1 197 065 ADRs, including 760 TD and 6059 EPS cases, were identified for this study. By calculating RORs, risk signals were detected with both FGAs and SGAs for TD and EPS compared with non-antipsychotics, with an ROR (95% confidence interval (CI)) of 153.9 (125.64-188.34) with FGAs for TD and 95.3 (80.61-112.65) with SGAs total for TD. No risk signals were detected for SGAs total data or any SGA subgroups versus FGAs. The ROR (95% CI) with SGAs total versus FGAs for TD was 0.62 (0.51-0.75), for dyskinesia: 0.55 (0.42-0.72), and for parkinsonism: 0.43 (0.35-0.52), showing that SGAs were associated with lower reporting frequency versus FGAs, but not for akathisia and dystonia. In conclusion, both FGAs and SGAs were associated with risks for TD and EPS compared with non-antipsychotics in the Japanese population, and SGAs total or all SGA subgroups showed no risk signals compared with FGAs.

第一代和第二代抗精神病药物(分别为FGAs和SGAs)具有多巴胺拮抗特性,可能导致不自主运动相关的药物不良反应(adr)。然而,日本人口的风险并没有很好地描述。在这项研究中,我们分析了来自日本不良药物事件报告(JADER)数据库的自发性不良反应报告,并评估了与抗精神病药物相关的迟发性运动障碍(TD)和其他锥体外系症状(EPS)的报告优势比(RORs)。根据SGAs的主要药理作用方式,将其作为一个整体类别和一个亚组进行评估。从2011年4月1日至2020年3月31日,本研究共发现1 197 065例adr,包括760例TD和6059例EPS。通过计算RORs,与非抗精神病药物相比,FGAs和SGAs对TD和EPS的风险信号均检测到,FGAs对TD的ROR(95%可信区间(CI))为153.9 (125.64-188.34),SGAs对TD的ROR(95%可信区间(CI))为95.3(80.61-112.65)。与FGAs相比,SGA总数据或任何SGA亚组未检测到风险信号。对于TD, SGAs总与FGAs的ROR (95% CI)为0.62(0.51-0.75),对于运动障碍:0.55(0.42-0.72),对于帕金森病:0.43(0.35-0.52),表明SGAs与FGAs相比报告频率较低,但与无运动障碍和肌张力障碍无关。总之,在日本人群中,与非抗精神病药物相比,FGAs和SGAs都与TD和EPS的风险相关,与FGAs相比,SGAs的总亚组或所有SGA亚组没有显示出风险信号。
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引用次数: 0
Nonrandomized Allocation of Steroid Therapy in Patients With Fukuyama Congenital Muscular Dystrophy: Study Protocol for a Phase II Clinical Trial. 福山先天性肌萎缩症患者非随机分配类固醇治疗:II期临床试验研究方案
IF 2 Q3 NEUROSCIENCES Pub Date : 2025-09-01 DOI: 10.1002/npr2.70043
Terumi Murakami, Takatoshi Sato, Takami Ishizuka, Harumasa Nakamura, Hisateru Tachimori, Hiroko Harada, Hideki Oi, Kenji Hatano, Mari S Oba, Kumiko Ishiguro, Minobu Shichiji, Yuki Kihara, Yasuhiro Takeshima, Mariko Taniguchi-Ikeda, Ayako Hattori, Yuko Shimizu-Motohashi, Hiroyuki Awano, Ryosuke Bo, Satoru Nagata, Keiko Ishigaki

Aim: This study aimed to evaluate the efficacy and safety of steroid therapy in patients with Fukuyama congenital muscular dystrophy (FCMD).

Methods: This is a noncontrolled, nonblinded, multicenter collaborative phase 2 trial in patients with a definite diagnosis of 3-kb insertion mutation homozygous or compound heterozygous FCMD based on genetic testing. The first part of the study evaluates efficacy; patients with homozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 24 weeks. The second part of the study evaluates safety; patients with homozygous and heterozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 48 weeks. Homozygous patients will be evaluated in the first part of the study for up to 24 weeks after prednisolone administration, after which they will continue receiving prednisolone in the second part for an additional 24 weeks. The primary endpoints were the changes in motor function evaluated using the gross motor function measure after treatment with prednisolone in the first part and the safety profiles based on the results of physical examination, vital signs, 12-lead electrocardiography (ECG), echocardiography, ophthalmic testing, SpO2, laboratory tests, immunological tests, and adverse events in the second part.

Discussion: Based on previous clinical research, prednisolone shows great potential as a therapeutic drug in patients with FCMD. To achieve this goal, we planned an investigator-initiated trial to confirm the effectiveness and safety of prednisolone.

目的:本研究旨在评价类固醇治疗福山先天性肌营养不良(FCMD)患者的疗效和安全性。方法:这是一项非对照、非盲、多中心合作的2期临床试验,研究对象是基于基因检测明确诊断为3kb插入突变纯合型或复合杂合型口蹄疫的患者。研究的第一部分评估疗效;纯合子型口蹄疫患者口服强的松龙,剂量为1mg /kg,隔天服用(0.5 mg/kg/天),疗程24周。研究的第二部分评估安全性;纯合型和杂合型口蹄疫患者口服强的松龙,剂量为1mg /kg,隔天服用(0.5 mg/kg/天),连续48周。纯合子患者将在强的松龙给药后24周在研究的第一部分进行评估,之后他们将在第二部分继续接受强的松龙治疗24周。主要终点是第一部分使用强的松龙治疗后使用大运动功能测量评估的运动功能变化,以及第二部分基于体格检查、生命体征、12导联心电图(ECG)、超声心动图、眼科检查、SpO2、实验室检查、免疫检查和不良事件结果的安全性概况。讨论:基于以往的临床研究,强的松龙作为手足口病患者的治疗药物显示出巨大的潜力。为了实现这一目标,我们计划了一项由研究者发起的试验,以确认强的松龙的有效性和安全性。
{"title":"Nonrandomized Allocation of Steroid Therapy in Patients With Fukuyama Congenital Muscular Dystrophy: Study Protocol for a Phase II Clinical Trial.","authors":"Terumi Murakami, Takatoshi Sato, Takami Ishizuka, Harumasa Nakamura, Hisateru Tachimori, Hiroko Harada, Hideki Oi, Kenji Hatano, Mari S Oba, Kumiko Ishiguro, Minobu Shichiji, Yuki Kihara, Yasuhiro Takeshima, Mariko Taniguchi-Ikeda, Ayako Hattori, Yuko Shimizu-Motohashi, Hiroyuki Awano, Ryosuke Bo, Satoru Nagata, Keiko Ishigaki","doi":"10.1002/npr2.70043","DOIUrl":"10.1002/npr2.70043","url":null,"abstract":"<p><strong>Aim: </strong>This study aimed to evaluate the efficacy and safety of steroid therapy in patients with Fukuyama congenital muscular dystrophy (FCMD).</p><p><strong>Methods: </strong>This is a noncontrolled, nonblinded, multicenter collaborative phase 2 trial in patients with a definite diagnosis of 3-kb insertion mutation homozygous or compound heterozygous FCMD based on genetic testing. The first part of the study evaluates efficacy; patients with homozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 24 weeks. The second part of the study evaluates safety; patients with homozygous and heterozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 48 weeks. Homozygous patients will be evaluated in the first part of the study for up to 24 weeks after prednisolone administration, after which they will continue receiving prednisolone in the second part for an additional 24 weeks. The primary endpoints were the changes in motor function evaluated using the gross motor function measure after treatment with prednisolone in the first part and the safety profiles based on the results of physical examination, vital signs, 12-lead electrocardiography (ECG), echocardiography, ophthalmic testing, SpO<sub>2</sub>, laboratory tests, immunological tests, and adverse events in the second part.</p><p><strong>Discussion: </strong>Based on previous clinical research, prednisolone shows great potential as a therapeutic drug in patients with FCMD. To achieve this goal, we planned an investigator-initiated trial to confirm the effectiveness and safety of prednisolone.</p>","PeriodicalId":19137,"journal":{"name":"Neuropsychopharmacology Reports","volume":"45 3","pages":"e70043"},"PeriodicalIF":2.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12355001/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144855888","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}
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Neuropsychopharmacology Reports
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