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环磷酰胺治疗肿块样多发性硬化。

Cyclophosphamide in treatment of tumefactive multiple sclerosis.

机构信息

Department of Neurology, Mayo Clinic Rochester, MN, USA; Center for Multiple Sclerosis and Autoimmune Neurology, Mayo Clinic Rochester, MN, USA.

Department of Neurology, Mayo Clinic Rochester, MN, USA; Center for Multiple Sclerosis and Autoimmune Neurology, Mayo Clinic Rochester, MN, USA.

出版信息

Mult Scler Relat Disord. 2021 Jan;47:102627. doi: 10.1016/j.msard.2020.102627. Epub 2020 Nov 11.

Abstract

OBJECTIVE

. To review the Mayo clinic experience of cyclophosphamide in the treatment of tumefactive Multiple Sclerosis (TMS).

METHODS

. Retrospective chart review was performed to identify patients with TMS who did not respond to corticosteroid and plasma exchange and were treated with cyclophosphamide. Demographic, clinical, treatment, and outcome parameters were collected.

RESULTS

. We identified 12 patients with TMS who were treated with cyclophosphamide. Median follow-up was 17 (range 1-72) months after cyclophosphamide administration. The tumefactive attack was the first clinical symptom in 5/12 patients. The median age at index attack onset was 36 (range 22-57). The median age at the first demyelinating attack was 29.3 (range 20-55). The most common clinical presentation at the index attack was the visual disturbance, followed by motor symptoms. The index attack was accompanied by headache in 5/12 patients. Median attack related Expanded Disability Status Scale (EDSS) score was 6 (range 3-9.5), which improved to 5.25 (range 1-10) after treatment with cyclophosphamide. All patients were treated with corticosteroids and plasma exchange prior to treatment with cyclophosphamide. EDSS score improved in 8/12 patients and targeted neurological deficits improved to a moderate or marked degree in 9/12 patients after cyclophosphamide administration. The number of gadolinium enhancing lesions decreased in 10/12 and 8/12 patients had lack of disease activity (no new T2 or GAD lesions) in a median (range) time of 11 months (1-16) after the last dose of cyclophosphamide.

CONCLUSION

. Cyclophosphamide could be an effective option for the treatment of TMS, refractory to corticosteroids and plasmapheresis, and is associated with improvement of clinical and radiological disease activity.

摘要

目的

总结梅奥诊所应用环磷酰胺治疗肿块型多发性硬化(TMS)的经验。

方法

回顾性分析 12 例对激素和血浆置换治疗无效的 TMS 患者,给予环磷酰胺治疗,收集患者的人口统计学、临床、治疗和预后等参数。

结果

12 例 TMS 患者接受环磷酰胺治疗,环磷酰胺治疗后中位随访时间 17(1-72)个月。5 例患者以肿块样发作为首发症状,中位发病年龄为 36(22-57)岁,中位首次临床缓解后复发年龄为 29.3(20-55)岁。首发症状以视力障碍最常见,其次为运动障碍。5 例患者首发时有头痛。首发时扩展残疾状态量表(EDSS)评分中位数为 6(3-9.5)分,经环磷酰胺治疗后降至 5.25(1-10)分。所有患者均在应用环磷酰胺前接受激素和血浆置换治疗。8 例患者 EDSS 评分改善,9 例患者神经系统定位体征改善至中度或明显改善。10 例患者钆增强病灶数减少,8 例患者在末次环磷酰胺治疗后 11 个月(1-16)中位时间内无疾病活动(无新 T2 或 GAD 病灶)。

结论

环磷酰胺可能是治疗激素和血浆置换治疗无效的 TMS 的有效方法,可改善临床和影像学疾病活动度。

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