High-dose methotrexate with leucovorin rescue: For monumentally severe CNS inflammatory syndromes

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• 作者：Shin C. Beh ; MD^a ; Eric Kildebeck ; MD ; PhD^a ; ^b ; Ram Narayan ; MD^a ; Allen Desena ; MD^a ; Doug Schell ; RN^c ; Elizabeth S. Rowe ; PhD ; MBA^c ; Vernon Rowe ; MD^c ; Dennis Burns ; MD^d ; Louis Whitworth ; MD^e ; Teresa C. Frohman ; PA-C^a ; Benjamin Greenberg ; MD ; MHS^a ; ^{benjamin.greenberg@utsouthwestern.edu} ; Elliot M. Frohman ; MD ; PhD^a ; ^f ; ^{elliot.frohman@utsouthwestern.edu}
• 关键词：Methotrexate ; Leucovorin ; Inflammatory demyelination
• 刊名：Journal of the Neurological Sciences
• 出版年：2017
• 出版时间：15 January 2017
• 年：2017
• 卷：372
• 期：Complete
• 页码：187-195
• 全文大小：987 K
• 卷排序：372

文摘

Methotrexate (HDMTX) achieves substantial CNS penetration, whereas other tissues can be rescued from the effects of HDMTX by leucovorin rescue (LR), which does not penetrate the blood-brain barrier. We report on the efficacy and safety of HDMTX with LR (HDMTX-LR), in the treatment of acute demyelinating inflammatory CNS syndromes refractory to conventional immunotherapy. Ten patients demonstrated both clinical and radiologic evidence of near, if not complete, abolishment of disease activity, in conjunction with impressive reconstitution of neurologic function in the 6-month period following HDMTX-LR. Mean Kurtzke Expanded Disability Status Scale (EDSS) prior to HDMTX-LR was 8.1 (± 1.4). Following HDMTX-LR, mean EDSS was 6.6 (± 2.4) at 1 month, 5.8 (± 2.3) at 3 months, and 5.7 (± 2.3) at 6 months. Our retrospective assessment of treatment-recalcitrant fulminant inflammatory CNS syndromes, HDMTX-LR was observed to be a safe and highly effective treatment, producing the rapid and near complete cessation of disease activity, in conjunction with an important corresponding and ‘durable remission’ in the majority of our small treatment cohort.