神经结节病的治疗：甲氨蝶呤与霉酚酸酯的对比研究
Treatment of neurosarcoidosis: A comparative study of methotrexate and mycophenolate mofetil.
作者信息
Bitoun Samuel, Bouvry Diane, Borie Raphaël, Mahevas Mathieu, Sacre Karim, Haroche Julien, Psimaras Dimitri, Pottier Corinne, Mathian Alexis, Hie Miguel, Boutin Du Le Thi Huong, Papo Thomas, Godeau Bertrand, Valeyre Dominique, Nunes Hilario, Amoura Zahir, Cohen Aubart Fleur
机构信息
From AP-HP, Service de Médecine Interne 2, Institut e3m, Centre National de Référence Maladies Systémiques Rares, Lupus, Syndrome des Anticorps Antiphospholipides (S.B., J.H., A.M., M.H., D.L.T.H.B., Z.A., F.C.A.), and AP-HP, Service de Neurologie 1 (D.P.), Hôpital de la Pitié-Salpêtrière, Paris; AP-HP, Service de Pneumologie (D.B., D.V., H.N.), Hôpital Avicenne, Bobigny; AP-HP, Services de Pneumologie (R.B.) and Médecine Interne (K.S., T.P.), Hôpital Bichat, Paris; AP-HP, Service de Médecine Interne (M.M., B.G.), Hôpital Henri Mondor, Créteil; Université Paris VI, UPMC (J.H., A.M., M.H., D.L.T.H.B., Z.A., C.A.), Sorbonnes Universités, Paris; and Service de Neurologie (C.P.), Centre Hospitalier René Dubos, Pontoise, France.
出版信息
Neurology. 2016 Dec 13;87(24):2517-2521. doi: 10.1212/WNL.0000000000003431. Epub 2016 Nov 16.
OBJECTIVE
To compare the efficacy of methotrexate (MTX) and mycophenolate mofetil (MMF) in the prevention of relapses in neurosarcoidosis.
METHODS
We conducted a retrospective multicenter study including patients who received MTX or MMF for the treatment of histologically proven neurosarcoidosis. The efficacy of the immunosuppressive drug was assessed by determining the time to relapse.
RESULTS
Forty patients with a diagnosis of neurosarcoidosis (24 men, 16 women, median age at diagnosis 43.5 years) who received at least 3 months of MTX (n = 32) or MMF (n = 14) were included. The immunosuppressive drug was always associated with steroids. The rate of relapse was 47% in the MTX group (0.2 relapses per year of exposure) and 79% in the MMF group (0.6 relapses per year of exposure) (p = 0.058). The median time to relapse was significantly shorter in the MMF group (11 months) compared with the MTX group (28 months) (p = 0.049). Adverse events occurred in 11 patients during MTX therapy and in 1 patient during MMF therapy (p = 0.12).
CONCLUSIONS
Relapses of neurosarcoidosis occur frequently, despite the use of an immunosuppressive drug in addition to corticosteroids. MTX significantly increases the survival time without relapse compared to MMF and should be preferred over MMF for the treatment of neurosarcoidosis. This study provides Class IV evidence that for patients with neurosarcoidosis taking steroids, MTX is superior to MMF in reducing the risk of relapse.
目的
比较甲氨蝶呤（MTX）和霉酚酸酯（MMF）预防神经结节病复发的疗效。
方法
我们进行了一项回顾性多中心研究，纳入接受MTX或MMF治疗经组织学证实的神经结节病患者。通过确定复发时间来评估免疫抑制药物的疗效。
结果
纳入40例诊断为神经结节病的患者（24例男性，16例女性，诊断时中位年龄43.5岁），他们接受了至少3个月的MTX（n = 32）或MMF（n = 14）治疗。免疫抑制药物总是与类固醇联合使用。MTX组的复发率为47%（每暴露年复发0.2次），MMF组为79%（每暴露年复发0.6次）（p = 0.058）。MMF组的中位复发时间（11个月）明显短于MTX组（28个月）（p = 0.049）。MTX治疗期间11例患者发生不良事件，MMF治疗期间1例患者发生不良事件（p = 0.12）。
结论
尽管除皮质类固醇外还使用了免疫抑制药物，但神经结节病仍频繁复发。与MMF相比，MTX显著延长了无复发生存时间，在神经结节病治疗中应优先于MMF使用。本研究提供了IV级证据，表明对于服用类固醇的神经结节病患者，MTX在降低复发风险方面优于MMF。
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