Peter Elise, Ciano-Petersen Nicolas Lundahl, Do Le-Duy, Perrot Jimmy, Ngo Thomas, Pluvinage John, Bartley Christopher M, Zorn Kelsey C, Miske Ramona, Scharf Madeleine, Villagrán-García Macarena, Farina Antonio, Rogemond Véronique, Antoine Jean-Christophe, Tranchant Christine, Dubois Valérie, DeRisi Joseph L, Pleasure Samuel J, Wilson Michael R, Gelfand Jeffrey M, Traverse-Glehen Alexandra, Honnorat Jérôme, Desestret Virginie
French Reference Center on Paraneoplastic Neurological Syndromes and Autoimmune Encephalitis, Hospices Civils de Lyon, Hôpital Neurologique, Bron, France.
MeLiS - UCBL - CNRS UMR 5284 - INSERM U1314, Université de Lyon, Université Claude Bernard Lyon 1, Lyon, France.
J Neurol. 2024 Oct;271(10):6839-6846. doi: 10.1007/s00415-024-12618-4. Epub 2024 Aug 29.
Ataxia with anti-regulator of G-protein signaling 8 autoantibodies (RGS8-Abs) is an autoimmune disease recently described in four patients. The present study aimed to identify other patients with RGS8-Abs, describe their clinical features, including the link between RGS8-related autoimmune cerebellar ataxia (ACA) and cancer. Patients with RGS8-Abs were identified retrospectively in the biological collections of the French Reference Center for Paraneoplastic Neurological Syndrome and the University of California San Francisco Center for Encephalitis and Meningitis. Clinical data were collected, and cerebrospinal fluid, serum, and tumor pathological samples were retrieved to characterize the autoantibodies and the associated malignancies. Only three patients with RGS8-Abs were identified. All of them presented with a pure cerebellar ataxia of mild to severe course, unresponsive to current immunotherapy regimens for ACA. Two patients presented with a Hodgkin lymphoma of the rare specific subtype called nodular lymphocyte-predominant Hodgkin lymphoma, with very mild extension. Autoantibodies detected in all patients enriched the same epitope on the RGS8 protein, which is an intracellular protein physiologically expressed in Purkinje cells but also ectopically expressed specifically in lymphoma cells of patients with RGS8-related ACA. The present results and those of the four cases previously described suggest that RGS8-Abs define a new paraneoplastic neurological syndrome of extreme rarity found mostly in middle-aged males that associates pure cerebellar ataxia and a particular lymphoma specifically expressing the RGS8 antigen. As in other paraneoplastic ACA with intracellular antigen, the disease course is severe, and patients tend to exhibit a poor response to immune therapy.
伴有抗G蛋白信号调节因子8自身抗体(RGS8-Abs)的共济失调是最近在4例患者中描述的一种自身免疫性疾病。本研究旨在识别其他患有RGS8-Abs的患者,描述他们的临床特征,包括RGS8相关自身免疫性小脑共济失调(ACA)与癌症之间的联系。通过法国副肿瘤性神经综合征参考中心和加利福尼亚大学旧金山分校脑炎和脑膜炎中心的生物样本库,对患有RGS8-Abs的患者进行回顾性鉴定。收集临床数据,并获取脑脊液、血清和肿瘤病理样本,以鉴定自身抗体和相关恶性肿瘤。仅鉴定出3例患有RGS8-Abs的患者。他们均表现为轻度至重度的单纯小脑共济失调,对目前用于ACA的免疫治疗方案无反应。2例患者患有罕见的特定亚型霍奇金淋巴瘤,即结节性淋巴细胞为主型霍奇金淋巴瘤,病变范围非常小。在所有患者中检测到的自身抗体富集了RGS8蛋白上的相同表位,RGS8蛋白是一种在浦肯野细胞中生理性表达的细胞内蛋白,但在患有RGS8相关ACA的患者的淋巴瘤细胞中也有异位特异性表达。目前的结果以及先前描述的4例病例的结果表明,RGS8-Abs定义了一种极为罕见的新的副肿瘤性神经综合征,主要见于中年男性,与单纯小脑共济失调和一种特异性表达RGS8抗原的特定淋巴瘤相关。与其他具有细胞内抗原的副肿瘤性ACA一样,该病病程严重,患者往往对免疫治疗反应不佳。