更新于：2024-05-01

Transverse Myelitis

横贯性脊髓炎

更新于：2024-05-01

基本信息

别名

MYELITIS TRANSVERSE、Myelitis transverse、Myelitis, Postinfectious

+ [26]

简介

Inflammation of a transverse portion of the spinal cord characterized by acute or subacute segmental demyelination or necrosis. The condition may occur sporadically, follow an infection or vaccination, or present as a paraneoplastic syndrome (see also ENCEPHALOMYELITIS, ACUTE DISSEMINATED). Clinical manifestations include motor weakness, sensory loss, and incontinence. (Adams et al., Principles of Neurology, 6th ed, pp1242-6)

关联

项与横贯性脊髓炎相关的药物

Equecabtagene Autoleucel

伊基奥仑赛

靶点

BCMA

作用机制

BCMA抑制剂

在研机构

Innovent Biologics, Inc. [+2]

原研机构

南京驯鹿生物技术股份有限公司初创企业 [+1]

在研适应症

多发性骨髓瘤 [+7]

非在研适应症

POEMS综合征

最高研发阶段批准上市

首次获批国家/地区

中国

首次获批日期2023-06-30

Rozanolixizumab

罗扎诺利珠单抗

靶点

FcRn

作用机制

FcRn拮抗剂 [+1]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2023-06-26

Mitoxantrone liposomal(CSPC Pharmaceutical Group Ltd.)

盐酸米托蒽醌脂质体

靶点

Top II

作用机制

Top II抑制剂

在研机构

石药集团中奇制药技术（石家庄）有限公司 [+5]

原研机构

石药集团有限公司

在研适应症

外周T细胞淋巴瘤 [+23]

非在研适应症

晚期胃癌 [+10]

最高研发阶段批准上市

首次获批国家/地区

中国

首次获批日期2021-06-01

267

项与横贯性脊髓炎相关的临床试验

NCT05199688 / Recruiting临床3期

A Phase III, Multicenter, Open-Label, Uncontrolled Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)

This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.

开始日期2024-06-28

申办/合作机构

Hoffmann-La Roche, Inc.

NCT06374264 / Not yet recruitingN/A

A Single Arm, Prospective, Multi-Center, Feasibility Study to Evaluate the Acceptability and Safety of MR-C-014 in Persons With Neuromyelitis Optica Spectrum Disorder (NMOSD) Who Have an Impaired Gait

The purpose of this proof of concept study is to assess the acceptability and safety of MR-C-014 in persons with Neuromyelitis optica spectrum disorder (NMOSD) who have a gait deficit.

开始日期2024-06-15

申办/合作机构

MedRhythms, Inc.

[+1]

TCTR20240314003 / Not yet recruitingN/AIIT

MRI features and their predictive value for relapsing in Thai Neuromyelitis Optica Spectrum Disorder: a retrospective cohort study

开始日期2024-06-01

申办/合作机构-

100 项与横贯性脊髓炎相关的临床结果

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100 项与横贯性脊髓炎相关的转化医学

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0 项与横贯性脊髓炎相关的专利（医药）

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7,095

项与横贯性脊髓炎相关的文献（医药）

2024-09-01·Neural regeneration research

Statistical evaluation of cluster formation of relapse in neuromyelitis optica spectrum disorder.

Article

作者: Tetsuya Akaishi ; Ichiro Nakashima

2024-03-01·Neurology(R) neuroimmunology & neuroinflammation

CD11c^high B Cell Expansion Is Associated With Severity and Brain Atrophy in Neuromyelitis Optica.

Article

作者: Eiichiro Amano ; Wakiro Sato ; Yukio Kimura ; Atsuko Kimura ; Youwei Lin ; Tomoko Okamoto ; Noriko Sato ; Takanori Yokota ; Takashi Yamamura

BACKGROUND AND OBJECTIVES:

Neuromyelitis optica (NMO) is an autoimmune astrocytopathy mediated by anti-AQP4 antibody-producing B cells. Recently, a B-cell subset highly expressing CD11c and T-bet, originally identified as age-associated B cells, has been shown to be involved in the pathogenesis of various autoimmune diseases. The objective of this study was to determine the relationship between the frequency of CD11c^high B cells per CD19⁺ B cells in the peripheral blood of patients with NMO and the clinical profiles including the brain volume.

METHODS:

In this observational study, 45 patients with anti-AQP4 antibody-positive NMO in remission and 30 healthy control subjects (HCs) were enrolled. Freshly isolated peripheral blood mononuclear cells were analyzed for immune cell phenotypes. The frequency of CD11c^high B cells per CD19⁺ B cells was assessed by flow cytometry and was evaluated in association with the clinical profiles of patients. Brain MRI data from 26 patients were included in the study for the analysis on the correlation between CD11c^high B-cell frequency and brain atrophy.

RESULTS:

We found that the frequency of CD11c^high B cells in CD19⁺ B cells was significantly increased in patients with NMO compared with HCs. The expansion of CD11c^high B cells significantly correlated with EDSS, past relapse numbers, and disease duration. In addition, a higher frequency of CD11c^high B cells negatively correlated with total brain, white matter, and gray matter volumes and positively correlated with T2/FLAIR high lesion volumes. When the past clinical relapse episodes of patients with or without the expansion of CD11c^high B cells were compared, relapses in the brain occurred more frequently in patients with CD11c^high B-cell expansion. CD11c^high B cells had distinct features including expression of chemokine receptors associated with migration into peripheral inflammatory tissues and antigen presentation. CD11c^high B-cell frequency was positively correlated with T peripheral helper-1 (Tph-1) cell frequency.

DISCUSSION:

Even during the relapse-free period, CD11c^high B cells could expand in the long disease context, possibly through the interaction with Tph-1 cells. The increased frequency of CD11c^high B cells associated with brain atrophy and disease severity, indicating that this cell population could be involved in chronic neuroinflammation in NMO.

2024-03-01·Neurology(R) neuroimmunology & neuroinflammation

CNS Inflammation as the First Sign of Complement Factor I Deficiency.

Article

作者: Valérie Massey ; Cam-Tu Emilie Nguyen ; Tine François ; Jean Jacques De Bruycker ; Arnaud Bonnefoy ; Anne-Laure Lapeyraque ; Hélène Decaluwe

OBJECTIVES:

Complement factor I (CFI) deficiency is a rare autosomal recessive inborn error of immunity. In this report, we highlight that complete CFI deficiency may present with isolated and severe CNS inflammation without associated systemic features nor prior non-CNS episodes. This inflammation may respond to complement blockade therapy.

METHODS:

This is a case description of a young girl with severe longitudinal transverse myelitis treated with aggressive immunotherapy that included eculizumab. Published cases of CFI-associated CNS inflammation were reviewed and discussed.

RESULTS:

A primary immunodeficiency panel revealed 2 germline pathogenic variants in the CFI gene. Further complement testing of the index case and her family confirmed complete CFI deficiency.

DISCUSSION:

We describe a unique case of severe spinal inflammation secondary to complete CFI deficiency. Although rare, isolated CNS inflammation may be the primary manifestation of complete CFI deficiency. To halt the uncontrolled complement-mediated inflammation associated with CFI deficiency, prompt targeted blockade of the complement pathway using eculizumab may be life changing in the acute phase. Long-lasting blockade of the complement pathway is also essential to prevent relapse in this subgroup of patients.

515

项与横贯性脊髓炎相关的新闻（医药）

2024-04-03

·医药观澜

荣昌生物「泰它西普」获FDA快速通道资格

▎药明康德内容团队编辑4月2日，荣昌生物宣布，美国FDA授予其泰它西普用于治疗干燥综合征的快速通道资格。2023年底，该适应症全球多中心3期临床已获FDA批准。原发性干燥综合征（pSS）是一种以侵犯泪腺和唾液腺等分泌腺为主的慢性炎症性自身免疫病，患者常出现口干、眼干、皮肤干的症状。目前，pSS尚无满意的治疗措施，无论是干燥、疲乏、疼痛或内脏器官损害均缺乏经循证医学论证的有效药物，现使用的药物多为经验性治疗，或借鉴类似病变的治疗。B细胞通路在pSS发病机制中发挥关键作用，而B淋巴细胞刺激因子（BLyS）和增殖诱导配体（APRIL）在B细胞成熟分化中起到至关重要的作用。根据荣昌生物新闻稿介绍，泰它西普作为新型全人源TACI-Fc融合蛋白，通过重组DNA技术将B细胞表面受体TACI的胞外段与IgG1 Fc段连接制备而成，能同时结合BLyS和APRIL因子，抑制B细胞的成熟分化，减少自身抗体分泌，更好地控制疾病活动。目前，除已获批上市的系统性红斑狼疮适应症外，泰它西普类风湿性关节炎适应症已申报上市，IgA肾炎、干燥综合征、视神经脊髓炎、重症肌无力等适应症的3期临床研究正在进行。此前，一项泰它西普治疗成人pSS患者的随机、双盲、安慰剂对照的2期临床试验结果发表在国际风湿病学期刊RHEUMATOLOGY。研究结论表明，泰它西普治疗pSS患者时展示出良好临床获益。与安慰剂相比，泰它西普治疗组在第12周和24周能明显改善pSS患者的ESSDAI评分（衡量干燥综合征疾病活动的金标准）和多维疲劳量表（MFI-20）评分，并降低免疫球蛋白水平，安全耐受，无严重不良事件发生，试验期间各组均无死亡事件发生。根据荣昌生物新闻稿介绍，该公司迄今已有3款药物先后五次被FDA授予快速通道资格。其中，泰它西普系统性红斑狼疮适应症、维迪西妥单抗胃癌和尿路上皮癌适应症已获批上市，泰它西普重症肌无力适应症正在开展3期临床试验，靶向MSLN的ADC在研药物RC88卵巢癌适应症正处于2期临床试验阶段。参考资料：[1]荣昌生物泰它西普干燥综合征获美国FDA快速通道资格，已获批开展全球多中心Ⅲ期临床. Retrieved Apr 2 , 2024, from https://mp.weixin.qq.com/s/WL5qG0xO8ZNRdZN-RW3Wzg本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

快速通道临床3期临床2期抗体药物偶联物

2024-04-02

·药渡

荣昌生物泰它西普干燥综合征获美国FDA快速通道资格，已获批开展全球多中心Ⅲ期临床

4月2日，荣昌生物制药（烟台）股份有限公司（股票代码：688331.SH / 09995.HK）宣布：美国FDA授予其泰它西普用于治疗干燥综合征的快速通道资格。2023年底，该适应症全球多中心Ⅲ期临床已获美国FDA批准。原发性干燥综合征（pSS）是一种以侵犯泪腺和唾液腺等分泌腺为主的慢性炎症性自身免疫病，患者常出现口干、眼干、皮肤干的症状。目前，pSS尚无满意的治疗措施，无论是干燥、疲乏、疼痛或内脏器官损害均缺乏经循证医学论证的有效药物，现使用的药物多为经验性治疗，或借鉴类似病变的治疗。B细胞通路在pSS发病机制中发挥关键作用，而B淋巴细胞刺激因子（BLyS）和增殖诱导配体（APRIL）在B细胞成熟分化中起到至关重要的作用。泰它西普作为新型全人源TACI-Fc融合蛋白，通过重组DNA技术将B细胞表面受体TACI的胞外段与IgG1 Fc段连接制备而成，能同时结合BLyS和APRIL因子，抑制B细胞的成熟分化，减少自身抗体分泌，更好地控制疾病活动。此前，一项泰它西普治疗成人pSS患者的随机、双盲、安慰剂对照的Ⅱ期临床试验结果发表在国际权威期刊RHEUMATOLOGY。研究结论表明，泰它西普治疗pSS患者时展示出良好临床获益。与安慰剂相比，泰它西普治疗组在第12周和24周能明显改善pSS患者的ESSDAI评分和MFI-20，并降低免疫球蛋白水平，安全耐受，无严重不良事件发生，试验期间各组均无死亡事件发生。目前全球并无用作治疗干燥综合征的生物药获批。而泰它西普有望成为干燥综合征突破性药物，填补这一领域的空白，为更多患者带来治愈希望。关于快速通道认定（FTD）快速通道认定（Fast Track Designation, FTD）是美国FDA为促进用于治疗严重疾病和解决未满足临床需求的新药研发而授予在研药物的一种资格认定。药物获得FTD后，新药研发公司将在后续的药物研发与审评过程中，获得更多与FDA沟通交流的机会，有助于加快药物后续研发和批准上市。此外，新药研发公司可以在提交上市申请（NDA/BLA）时向FDA滚动递交新药研究资料。这些优惠政策都为加快新药的研发提供有利保障。迄今，荣昌生物三款药物先后五次被美国FDA授予快速通道资格，其中，泰它西普系统性红斑狼疮适应症、维迪西妥单抗胃癌和尿路上皮癌适应症已获批上市，泰它西普重症肌无力适应症正在开展Ⅲ期临床试验，靶向MSLN的ADC在研药物RC88卵巢癌适应症正处于Ⅱ期临床试验阶段。关于泰它西普泰它西普是由公司CEO、首席科学官房健民教授发明设计的一个抗体融合蛋白药物分子，是全球首个双靶点治疗系统性红斑狼疮的生物新药。它通过同时抑制BLyS和APRIL两个细胞因子的过度表达，“双管齐下”阻止B细胞的异常分化和成熟，从而治疗自身免疫性疾病。除已获批上市的系统性红斑狼疮外，泰它西普类风湿性关节炎适应症已申报上市，IgA肾炎、干燥综合征、视神经脊髓炎、重症肌无力等适应症的Ⅲ期临床研究正在进行。

快速通道临床3期临床2期突破性疗法抗体药物偶联物

2024-04-02

·Pharmaceutical Technology

Alexion’s Voydeya gains FDA approval for extravascular haemolysis

Alexion is exploring the potential of Voydeya as a monotherapy. Credit: Sai Thaw Kyar/Shutterstock.com. Alexion, AstraZeneca Rare Disease has received approval from the US Food and Drug Administration (FDA) for Voydeya (danicopan) as an add-on therapy to treat extravascular haemolysis (EVH) in adults with paroxysmal nocturnal haemoglobinuria (PNH). A first-in-class, oral Factor D inhibitor, Voydeya has been developed for patients who continue to suffer from EVH despite treatment with C5 inhibitors such as Ultomiris (ravulizumab) or Soliris (eculizumab). The FDA’s decision is grounded in the positive outcomes of the ALPHA Phase III trial, which demonstrated that Voydeya met its primary endpoint of haemoglobin change from baseline to week 12, alongside all key secondary endpoints. The drug was generally well-tolerated with no new safety concerns. As part of the ALPHA double-blind, placebo-controlled, multiple-dose trial, patients were enrolled and randomised to receive Voydeya or placebo apart from their ongoing Soliris or Ultomiris therapy over 12 weeks. See Also: US FDA grants fast track status to LISCure’s LB-P8 drug for PSC FDA extends review period for Applied Therapeutics’ govorestat NDA It aimed to assess the superiority of Voydeya as an adjunct to C5 inhibitor therapy. The trial included a prespecified interim analysis and an option for a two-year extension period for those completing the initial 24 weeks. Japan already approved Voydeya and the European Union is poised to follow suit. The drug has also achieved breakthrough therapy designation in the US and PRIority MEdicines (PRIME) status in the EU. Alexion CEO Marc Dunoyer stated: “The approval of first-in-class, Factor D inhibitor Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science. “As the ALPHA trial suggests, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for this subset of patients with EVH, enabling them to continue with proven standard-of-care therapy.” Alexion is also exploring Voydeya’s potential as a monotherapy for geographic atrophy in a Phase II clinical trial. The FDA recently approved Alexion’s Ultomiris to treat adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.