Protocol for a multicentre randomiSed controlled TRial of IntraVEnous immunoglobulin versus standard therapy for the treatment of transverse myelitis in adults and children (STRIVE).
Protocol for a multicentre randomiSed controlled TRial of IntraVEnous immunoglobulin versus standard therapy for the treatment of transverse myelitis in adults and children (STRIVE).
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DOI：
10.1136/bmjopen-2015-008312
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发表时间：
2015-05-25
期刊：
影响因子：
2.9
通讯作者：
Lim M
中科院分区：
文献类型：
--
作者：
Absoud M;Gadian J;Hellier J;Brex PA;Ciccarelli O;Giovannoni G;Kelly J;McCrone P;Murphy C;Palace J;Pickles A;Pike M;Robertson N;Jacob A;Lim M
横肌萎缩（TM）是一种免疫介导的脊髓疾病，导致运动和感觉障碍，50%的患者恢复有限。标准治疗是类固醇，更严重的疾病患者似乎对血浆置换（PLEX）有反应。静脉注射免疫球蛋白（IVIG）也被用作类固醇的辅助药物，但缺乏证据。我们建议在成人和儿童中进行首次随机对照试验，以确定IVIG额外治疗的益处。将招募170名患有急性首次发作TM或视神经肌萎缩（伴肌萎缩）的成人和1岁以上儿童，为期2.5年，随访12个月。 随机分配至对照组的受试者将接受静脉注射甲泼尼龙（IVMP）的标准治疗。干预组将接受上述标准治疗，加上额外的IVIG。主要结局将是盲态评估者随机化后6个月时美国脊髓损伤协会（ASIA）损伤量表改善2分。 将收集额外的二级和三级结局指标：ASIA运动和感觉量表、Kurtzke扩展残疾状态量表、国际脊髓损伤（SCI）膀胱/肠数据集、客户服务接收指数、儿科生活质量量表、EQ-5D、SCI疼痛和SCI生活质量数据集。将对生物样本进行生物库储存，用于未来研究。在对前52例招募的患者进行6个月随访后，将进行无效性分析。将进行卫生经济学分析以计算成本效益。招募6个月后，将进行无效性分析。获得研究伦理委员会批准：14/SC/1329。现行方案：v3.0（2015年1月15日）。研究结果将发表在同行评审期刊上。本研究已在EudraCT（REF：2014-002335-34）、Clinicaltrials.gov（REF：NCT 02398994）和ISRCTN（REF：12127581）注册。
Transverse myelitis (TM) is an immune-mediated disorder of the spinal cord which causes motor and sensory disturbance and limited recovery in 50% of patients. Standard treatment is steroids, and patients with more severe disease appear to respond to plasma exchange (PLEX). Intravenous immunoglobulin (IVIG) has also been used as an adjunct to steroids, but evidence is lacking. We propose the first randomised control trial in adults and children, to determine the benefit of additional treatment with IVIG. 170 adults and children aged over 1 year with acute first episode TM or neuromyelitis optica (with myelitis) will be recruited over a 2.5-year period and followed up for 12 months. Participants randomised to the control arm will receive standard therapy of intravenous methylprednisolone (IVMP). The intervention arm will receive the above standard therapy, plus additional IVIG. Primary outcome will be a 2-point improvement on the American Spinal Injury Association (ASIA) Impairment scale at 6 months postrandomisation by blinded assessors. Additional secondary and tertiary outcome measures will be collected: ASIA motor and sensory scales, Kurtzke expanded disability status scale, International Spinal Cord Injury (SCI) Bladder/Bowel Data Set, Client Services Receipt Index, Pediatric Quality of Life Inventory, EQ-5D, SCI Pain and SCI Quality of Life Data Sets. Biological samples will be biobanked for future studies. After 6-months' follow-up of the first 52 recruited patients futility analysis will be carried out. Health economics analysis will be performed to calculate cost-effectiveness. After 6 months’ recruitment futility analysis will be performed. Research Ethics Committee Approval was obtained: 14/SC/1329. Current protocol: v3.0 (15/01/2015). Study findings will be published in peer-reviewed journals. This study is registered with EudraCT (REF: 2014-002335-34), Clinicaltrials.gov (REF: NCT02398994) and ISRCTN (REF: 12127581).
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