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Short term real-world Fingolimod efficacy and safety in Emirati patients with multiple sclerosis.

阿联酋多发性硬化患者的短期真实世界芬戈莫德疗效和安全性。

  • 影响因子:1.63
  • DOI:10.1016/j.jocn.2019.11.010
  • 作者列表:"Ceccarelli A","Mifsud V","Abusamra E","Hussain SI
  • 发表时间:2020-01-01
Abstract

:In clinical trials, Fingolimod was an efficacious and safe treatment for multiple sclerosis (MS). Despite this, few studies have explored its real-world efficacy and safety in the Middle East. The aim of this study was to describe our clinical experience with Fingolimod at Cleveland Clinic Abu Dhabi in Emirati patients with MS. We retrospectively collected clinical and brain and spinal cord MRI activity over time in 30 Emirati MS patients [F/M = 22/8, mean (SD) disease duration at treatment initiation = 3.3 (3.6) years, age at onset = 25.9 (6.9) years] who were commenced on Fingolimod from 2015 and followed for 18 months. The proportion of MS patients clinically and radiologically silent after Fingolimod initiation over time was assessed together with annualized relapse rate (ARR) reduction and adverse events (AEs) occurrence. 70% of MS patients who started Fingolimod were naïve. The baseline ARR [mean (SD; range)] was [1.2 (0.8; 0-4)] and 23.3% of MS patients had Gadolinium enhancing lesions at baseline MRI. Overall, the ARR was reduced by 72% and, relapses and MRI activity were found in only 24% and 38% of MS patients respectively. Mild to moderate AEs were observed in 33% of MS patients. No severe AEs were recorded. In Emirati MS patients, Fingolimod is safe and efficacious as an early treatment choice.

摘要

: 在临床试验中,芬戈莫德是多发性硬化症 (MS) 的有效和安全的治疗方法。尽管如此,很少有研究探讨其在中东的真实世界的疗效和安全性。本研究的目的是描述我们在阿布扎比克利夫兰诊所使用芬戈莫德治疗阿联酋MS患者的临床经验。我们回顾性收集了30例阿联酋MS患者的临床和脑和脊髓MRI随时间的活动 [F/m   =   22/8,治疗开始时的平均 (SD) 病程   =   3.3 (3.6) 年,Onset   的年龄 =   25.9 (6.9) 岁],从2015年开始使用Fingolimod,随访18个月。随着时间的推移,评估了芬戈莫德开始后临床和放射学上沉默的MS患者的比例,以及年复发率 (ARR) 降低和不良事件 (ae) 发生。70% 开始使用芬戈莫德的MS患者是幼稚的。基线ARR [平均值 (SD; 范围)] 为 [1.2 (0.8; 0-4)],23.3% 的MS患者在基线MRI时有钆增强病变。总体而言,ARR降低了72%,并且分别仅在24% 和38% 的MS患者中发现复发和MRI活性。在33% 的MS患者中观察到轻度至中度ae。未记录到严重不良事件。在阿联酋MS患者中,芬戈莫德作为早期治疗选择是安全有效的。

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作者列表:["Apivatthakakul A","Kunavisarut P","Rothova A","Pathanapitoon K"]

METHODS::Purpose: To report on ocular Vogt-Koyanagi-Harada (VKH)-like syndrome under vemurafenib treatment for metastatic melanoma.Design: A case report.Method: Description of clinical and imaging manifestations including fundus photography, fluorescein, and indocyanine green angiography.Results: A 46-year-old Thai female was diagnosed with metastatic melanoma of the skin and had been treated with multiple surgical excisions, radiotherapy, and vemurafenib (initial dose 480 mg orally twice daily, subsequently increased to maximum dose of 960 mg twice daily). After 6 months of vemurafenib use, she complained of bilateral redness and photophobia and was diagnosed with bilateral anterior uveitis, which was topically treated. Two weeks later, her visual acuity (VA) sharply deteriorated to 20/80 and counting fingers. Ocular examination at that stage stronly resembled acute VKH disease. She exhibited intraocular inflammation, and her fundus examination revealed bilateral optic disc swelling and serous retinal detachment. Fluorescein angiogram showed disc leakage and multiple pinpoint hyperfluorescence leakage spots and indocyanine green demonstrated multiple hypofluorescent spots. Oral prednisolone 30 mg/day was commenced while vemurafenib medication was ceased. Three weeks later, her vision improved, and serous retinal detachment subsided. However, her cutaneous melanoma recurred.Conclusions: Vemurafenib, a potential adjunct treatment for metastatic melanoma, was complicated by the development of panuveitis, papillitis, and multiple serous detachments. These ocular symptoms were similar to the presentation of acute VKH syndrome.

翻译标题与摘要 下载文献
影响因子:2.19
发表时间:2020-01-01
DOI:10.1111/dmcn.14268
作者列表:["Crow YJ","Shetty J","Livingston JH"]

METHODS::Comprehensive reviews of the clinical characteristics and pathogenesis of Aicardi-Goutières syndrome (AGS), particularly its contextualization within a putative type I interferonopathy framework, already exist. However, recent reports of attempts at treatment suggest that an assessment of the field from a therapeutic perspective is warranted at this time. Here, we briefly summarize the neurological phenotypes associated with mutations in the seven genes so far associated with AGS, rehearse current knowledge of the pathology as it relates to possible treatment approaches, critically appraise the potential utility of therapies, and discuss the challenges in assessing clinical efficacy. WHAT THIS PAPER ADDS: Progress in understanding AGS disease pathogenesis has led to the first attempts at targeted treatment. Further rational therapies are expected to become available in the short- to medium-term.

关键词: 暂无
翻译标题与摘要 下载文献
影响因子:1.52
发表时间:2020-04-02
DOI:10.1080/09273948.2019.1603312
作者列表:["Takayama K","Obata H","Takeuchi M"]

METHODS::Purpose: To report the efficacy of adalimumab in a case of chronic Vogt-Koyanagi-Harada (VKH) disease refractory to conventional corticosteroids and immunosuppressive therapy and complicated by central serous chorioretinopathy (CSC).Case report: A 66-year-old woman diagnosed with VKH was treated with intravenous corticosteroids followed by oral corticosteroids and cyclosporine. However, systemic corticosteroids could not be tapered because of recurrent ocular inflammation and systemic complications (diabetes mellitus, moon face, bone weakness), while CSC appeared in both eyes. A diagnosis of chronic VKH resistant to medications complicated by corticosteroid-induced CSC was made. Systemic corticosteroids and cyclosporine were tapered and adalimumab initiated. Bilateral ocular inflammation and CSC were gradually reduced and visual acuity improved without any adverse effect. Twelve months after starting adalimumab monotherapy, no signs of active VKH and CSC were present.Conclusions: Adalimumab is one of the effective therapeutic options for refractory VKH disease complicated with corticosteroid-induced adverse effects.

神经系统自身免疫性疾病方向

神经系统自身免疫性疾病是以自身免疫细胞、免疫分子等攻击神经系统为主要致病机制的自身免疫性疾病。在免疫反应中,作用于神经系统自身抗原的致病抗体统称为神经系统自身抗体。

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