Division of Rheumatology, Azienda USL-IRCCS di Reggio Emilia, Reggio Emilia, Italy; Università di Modena e Reggio Emilia, Modena, Italy.
Department of Neurology, Mayo Clinic, Rochester, MN, United States of America.
Autoimmun Rev. 2020 Apr;19(4):102497. doi: 10.1016/j.autrev.2020.102497. Epub 2020 Feb 13.
To evaluate long-term treatment and outcomes of patients with primary central nervous system vasculitis (PCNSV).
In this cohort of 191 consecutive patients with PCNSV seen at Mayo Clinic, Rochester, MN, over 35 years with long-term follow-up we analyzed response to and duration of therapy, frequency of relapses, long-term remission, efficacy of maintenance therapy and initial intravenous glucocorticoid (GC) pulses, survival and degree of disability. We also compared the efficacy of initial IV and oral cyclophosphamide (CYC).
A favorable initial response was observed in 83% of patients treated with prednisone (PDN) alone, 81% of those treated with PDN and CYC and 95% of those initially treated with PDN and an immunosuppressant other than CYC. One or more relapses were observed in 30% of patients, 35% had discontinued therapy by last follow-up, and 21.5% maintained remission for at least 12 months after discontinuing therapy. Maintenance therapy was prescribed in 19% of all patients and 34% of patients initially treated with CYC and PDN. High disability scores (Rankin 4-6) and deaths were less frequently observed in patients receiving maintenance therapy and more frequently in patients with Aβ-related angiitis. Large vessel involvement and cerebral infarction at diagnosis were associated with a poor treatment response. Aspirin use was positively associated with long-term remission and having gadolinium-enhanced cerebral lesions or meninges was negatively associated. A high disability score at last follow-up and higher mortality rate were associated with increasing age, cerebral infarction and cognitive dysfunction at diagnosis. Lymphocytic vasculitis on biopsy was associated with a more benign course with reduced disability and mortality. Patients initially treated with mycophenolate mofetil had better outcomes compared to those treated with CYC and PDN. No therapeutic advantages were observed in the patients initially treated with intravenous GC pulses. Intravenous and oral CYC were equally effective in inducing the remission.
The majority of patients with PCNSV responded to treatment. We found patient subsets with different outcomes. Mycophenolate mofetil may be an effective alternative to CYC.
评估原发性中枢神经系统血管炎（PCNSV）患者的长期治疗和结局。
在这项由明尼苏达州罗切斯特市梅奥诊所的 191 名连续 PCNSV 患者组成的队列研究中，我们对 35 年的长期随访进行了分析，包括治疗反应和持续时间、复发频率、长期缓解、维持治疗和初始静脉内糖皮质激素（GC）脉冲的疗效、生存率和残疾程度。我们还比较了初始静脉内和口服环磷酰胺（CYC）的疗效。
单独使用泼尼松（PDN）治疗的患者中有 83%、PDN 和 CYC 联合治疗的患者中有 81%、最初使用 PDN 和非 CYC 免疫抑制剂治疗的患者中有 95%有良好的初始反应。30%的患者出现一次或多次复发，35%的患者在最后一次随访时已停止治疗，21.5%的患者在停止治疗后至少 12 个月维持缓解。19%的患者接受了维持治疗，34%的最初接受 CYC 和 PDN 治疗的患者接受了维持治疗。接受维持治疗的患者残疾评分较高（Rankin 4-6）和死亡率较低，而 Aβ 相关血管炎患者则较高。大血管受累和诊断时脑梗死与治疗反应不良相关。阿司匹林的使用与长期缓解呈正相关，而钆增强脑病变或脑膜与预后不良呈负相关。最后一次随访时的残疾评分较高和死亡率较高与年龄增加、诊断时脑梗死和认知功能障碍相关。活检的淋巴细胞血管炎与减少残疾和死亡相关的更良性病程相关。与接受 CYC 和 PDN 治疗的患者相比，最初接受霉酚酸酯治疗的患者有更好的结果。在最初接受静脉 GC 脉冲治疗的患者中，没有观察到治疗优势。静脉内和口服 CYC 在诱导缓解方面同样有效。
大多数 PCNSV 患者对治疗有反应。我们发现了具有不同结局的患者亚组。霉酚酸酯可能是 CYC 的有效替代药物。
