Rosati Anna, Cosi Alessandra, Basile Massimo, Brambilla Alice, Guerrini Renzo, Cimaz Rolando, Simonini Gabriele
Paediatric Neurology Unit, NEUROFARBA Department, Anna Meyer Children's Hospital, University of Florence, 50139 Firenze, Italy.
Radiology Unit, Anna Meyer Children's Hospital, 50139 Firenze, Italy.
Joint Bone Spine. 2017 May;84(3):353-356. doi: 10.1016/j.jbspin.2016.12.004. Epub 2016 Dec 26.
To report our single centre experience in treating 4 children affected by childhood primary central nervous system vasculitis (cPACNS) using mycophenolate mofetil (MMF).
From December 2011 to August 2015, 4 patients (3 males; age range: 9 months-13 years) affected by cPACNS were collected. Enrolled children received the following treatment protocol: acetylsalicylic acid and/or anticoagulant therapy with low molecular weight heparin (LMWH) 100 U/k BID replaced by acenocoumarol; methyl-prednisolone (30mg/kg/day for 3-5 days) followed by prednisone (2mg/kg/day), tapered and discontinued over 7-8 months; MMF used for induction therapy and subsequent maintenance phase (750-1000mg/m BID, half-dose for the first 10-15 days followed by full-dose).
In all children, no relapse of cerebral vasculitis occurred during the whole follow-up period and all of them improved while in MMF treatment. Magnetic resonance imaging (MRI) and magnetic resonance angiography (MRA), performed at 6, 9 or 12 months intervals, showed no progression or even improvement of the typical radiological findings. Medium period of MMF treatment was 29 months (range: 10-42 months). No major drug-related adverse events were documented.
We report for the first time on the efficacy and safety of MMF in the induction and maintenance of clinical remission in cPACNS. Our single centre experience of MMF use in treating cPACNS seems represent an appealing, alternative and safe option in this clinical setting over a long-term follow-up.
报告我们单中心使用霉酚酸酯（MMF）治疗4例儿童期原发性中枢神经系统血管炎（cPACNS）患儿的经验。
收集2011年12月至2015年8月期间4例cPACNS患儿（3例男性；年龄范围：9个月至13岁）。入选儿童接受以下治疗方案：阿司匹林和/或低分子肝素（LMWH）100 U/kg bid抗凝治疗，后用醋硝香豆素替代；甲泼尼龙（30mg/kg/天，共3 - 5天），随后泼尼松（2mg/kg/天），在7 - 8个月内逐渐减量并停药；MMF用于诱导治疗及后续维持阶段（750 - 1000mg/m² bid，前10 - 15天半量，随后全量）。
所有患儿在整个随访期间均未出现脑血管炎复发，且在MMF治疗期间均有改善。每隔6、9或12个月进行的磁共振成像（MRI）和磁共振血管造影（MRA）显示，典型影像学表现无进展甚至有所改善。MMF治疗的中位时间为29个月（范围：10 - 42个月）。未记录到重大药物相关不良事件。
我们首次报告了MMF在诱导和维持cPACNS临床缓解方面的有效性和安全性。我们单中心使用MMF治疗cPACNS的经验在长期随访中似乎是一种有吸引力、可替代且安全的选择。
