Department of Pediatric Neurology, Hôpital Erasme and Université Libre de Bruxelles, Brussels, Belgium.
Pediatrics. 2011 Apr;127(4):e1086-90. doi: 10.1542/peds.2009-2729. Epub 2011 Mar 14.
Primary angiitis of the central nervous system (PACNS) is an idiopathic vasculitis confined to the central nervous system. In children with PACNS, small-vessel (SV) involvement is characterized clinically by progressive neurologic symptoms, multifocal lesions on brain imaging, occasional pseudo-tumor presentation, and normal angiogram results in most patients. Small case series of patients with SV PACNS with short follow-up usually reveal favorable outcomes in children treated with immunosuppressive therapy. We report here the cases of 3 children with biopsy-confirmed SV PACNS and long-term follow-up who developed different patterns of neurologic deterioration despite immunosuppressive therapy. One patient had transient ischemic attacks shortly after initiation of corticosteroid treatment. Early ischemic events probably result from residual thrombogenicity or residual inflammation of recently affected vessels, which supports the use of antiplatelet agents and suggests potential benefits of stronger immunosuppressive therapy. In contrast, the other 2 patients had later neurologic deterioration after corticosteroid withdrawal, which suggests failure of initial immunosuppressant treatment and the need for stronger agents, longer treatment duration, or both. All patients responded to long-term treatment with corticosteroids combined with cytotoxic agents. This particular combination is probably indicated in many cases of SV PACNS, including those with neurologic deterioration that occurs during maintenance corticotherapy or after corticosteroid withdrawal. In 1 case, SV PACNS recurred several years after discontinuation of combination therapy. Long-term relapses may reflect intrinsic predispositions to SV PACNS rather than treatment failure. These cases highlight different chronological patterns of neurologic deterioration despite immunosuppressive therapy, which supports the relevance of monitoring clinical, laboratory, and radiologic responses to treatment and of long-term follow-up of patients with SV PACNS.
原发性中枢神经系统血管炎（PACNS）是一种局限于中枢神经系统的特发性血管炎。在患有 PACNS 的儿童中，小血管（SV）受累的临床表现为进行性神经症状、脑影像学上的多灶性病变、偶尔出现假性肿瘤表现，以及大多数患者的血管造影结果正常。小系列 SV PACNS 患者的短期随访通常显示接受免疫抑制治疗的儿童有良好的结局。我们在此报告 3 例经活检证实的 SV PACNS 患儿的病例，他们在长期随访中尽管接受了免疫抑制治疗，但出现了不同模式的神经恶化。1 例患者在开始皮质类固醇治疗后不久发生短暂性脑缺血发作。早期缺血事件可能是由于新受影响的血管的残留血栓形成或残留炎症引起的，这支持使用抗血小板药物，并提示更强的免疫抑制治疗可能有潜在益处。相比之下，另外 2 例患者在皮质类固醇停药后出现晚期神经恶化，这表明初始免疫抑制剂治疗失败，需要更强的药物、更长的治疗时间或两者兼而有之。所有患者均对长期皮质激素联合细胞毒性药物治疗有反应。这种特殊的联合治疗可能适用于许多 SV PACNS 病例，包括在维持皮质激素治疗期间或停药后发生神经恶化的病例。在 1 例患者中，SV PACNS 在停止联合治疗数年后复发。长期复发可能反映了 SV PACNS 的内在易感性，而不是治疗失败。这些病例强调了尽管进行了免疫抑制治疗，但仍存在不同的神经恶化时间模式，这支持对治疗的临床、实验室和影像学反应进行监测，并对 SV PACNS 患者进行长期随访的重要性。
