Department of Neurology, Beijing Tiantan Hospital, Capital Medical University; China National Clinical Research Center for Neurological Diseases, 119 South Fourth Ring West Road, Fengtai District, Beijing, 100070, People's Republic of China.
Department of Neurology, Xuanwu Hospital of Capital Medical University, 45 Chang Chun Road, Xicheng District, Beijing, 100053, People's Republic of China.
BMC Neurosci. 2020 Mar 30;21(1):13. doi: 10.1186/s12868-020-00561-9.
Patients positive for anti-glutamic acid decarboxylase 65 (GAD65) antibodies have attracted increasing attention. Their clinical manifestations are highly heterogeneous and can be comorbid with tumors. Currently, there is no consensus on the therapeutic regimen for anti-GAD65-associated neurological diseases due to the clinical complexity, rarity and sporadic distribution. We reported six anti-GAD65 autoimmune encephalitis (AE) patients who received intravenous methylprednisolone (IVMP) or immunoglobulin (IVIG) or both. Then, we evaluated the therapeutic effect of both by summarizing results in previous anti-GAD65 AE patients from 70 published references.
Our six patients all achieved clinical improvements in the short term. Unfortunately, there was no significant difference between IVMP and IVIG in terms of therapeutic response according to the previous references, and the effectiveness of IVMP and IVIG was 45.56% and 36.71%, respectively. We further divided the patients into different subgroups according to their prominent clinical manifestations. The response rates of IVMP and IVIG were 42.65% and 32.69%, respectively, in epilepsy patients; 60.00% and 77.78%, respectively, in patients with stiff-person syndrome; and 28.57% and 55.56%, respectively, in cerebellar ataxia patients. Among 29 anti-GAD65 AE patients with tumors, the response rates of IVMP and IVIG were 29.41% and 42.11%, respectively. There was no significant difference in effectiveness between the two regimens among the different subgroups.
Except for stiff-person syndrome, we found that this kind of AE generally has a poor response to IVMP or IVIG. Larger prospective studies enrolling large numbers of patients are required to identify the optimal therapeutic strategy in the future.
抗谷氨酸脱羧酶 65（GAD65）抗体阳性患者越来越受到关注。其临床表现高度异质，可与肿瘤并存。由于临床复杂性、罕见性和散发性分布，目前对于抗 GAD65 相关神经疾病尚无共识的治疗方案。我们报告了 6 例抗 GAD65 自身免疫性脑炎（AE）患者，他们接受了静脉注射甲基强的松龙（IVMP）或免疫球蛋白（IVIG）或两者联合治疗。然后，我们通过总结 70 篇已发表文献中以前的抗 GAD65 AE 患者的结果，评估了这两种方法的治疗效果。
我们的 6 例患者在短期内均取得了临床改善。不幸的是，根据以往文献，IVMP 和 IVIG 在治疗反应方面没有显著差异，IVMP 和 IVIG 的有效性分别为 45.56%和 36.71%。我们进一步根据患者突出的临床表现将患者分为不同亚组。在癫痫患者中，IVMP 和 IVIG 的反应率分别为 42.65%和 32.69%；在僵硬人综合征患者中，分别为 60.00%和 77.78%；在小脑共济失调患者中，分别为 28.57%和 55.56%。在 29 例伴有肿瘤的抗 GAD65 AE 患者中，IVMP 和 IVIG 的反应率分别为 29.41%和 42.11%。两种方案在不同亚组之间的疗效无显著差异。
除僵硬人综合征外，我们发现这种 AE 通常对 IVMP 或 IVIG 反应不佳。需要进行更大规模的前瞻性研究，纳入更多患者，以确定未来的最佳治疗策略。
