Treatment of Primary Autoimmune Cerebellar Ataxia with Mycophenolate
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ORIGINAL ARTICLE
Treatment of Primary Autoimmune Cerebellar Ataxia with Mycophenolate M. Hadjivassiliou 1 N. Hoggard 3
&
R. A. Grunewald 1 & P. D. Shanmugarajah 1 & P. G. Sarrigiannis 1 & P. Zis 1 & V. Skarlatou 2 &
# The Author(s) 2020
Abstract Immune-mediated ataxias account for a substantial number of sporadic otherwise idiopathic ataxias. Despite some wellcharacterised entities such as paraneoplastic cerebellar degeneration where diagnostic markers exist, the majority of immune ataxias remained undiagnosed and untreated. We present here our experience in the treatment of suspected primary autoimmune cerebellar ataxia (PACA) using mycophenolate. All patients reported attend the Sheffield Ataxia Centre on a regular basis and had undergone extensive investigations, including genetic testing using next-generation sequencing, with other causes of ataxia excluded. The diagnosis of PACA was strongly suspected based on investigations, pattern of disease progression, and cerebellar involvement. Patients were treated with mycophenolate and monitored using MR spectroscopy of the cerebellar vermis. Thirty patients with PACA are reported here. Of these, 22 received mycophenolate (group 1). The remaining 8 were not on treatment (group 2-control group). Out of the 22 treated patients, 4 underwent serial MR spectroscopy prior to starting treatment and thus were used as controls making the total number of patients in the control group 12. The mean change of the MRS within the vermis (NAA/Cr area ratio) in the treatment group was + 0.144 ± 0.09 (improved) and in the untreated group − 0.155 ± 0.06 (deteriorated). The difference was significant. We also demonstrated a strong correlation between the spectroscopy and the SARA score. We have demonstrated the effectiveness of mycophenolate in the treatment of PACA. The results suggest that immune-mediated ataxias are potentially treatable, and that there is a need for early diagnosis to prevent permanent neurological deficit. The recently published diagnostic criteria for PACA would hopefully aid the diagnosis and treatment of this entity. Keywords Immune ataxias . Primarry autoimmune cerebellar ataxia . Mycophenolate . MR spectroscopy
Introduction Immune-mediated ataxias include paraneoplastic cerebellar degeneration (PCD), gluten ataxia (GA) and post-infective cerebellitis. Unlike PCD, GA and post-infective cerebellitis where an antigenic trigger is known, in most suspected autoimmune ataxias, the antigenic trigger is not known and any
* M. Hadjivassiliou [email protected] 1
Academic Department of Neurosciences, Sheffield Teaching Hospitals NHS Trust, Sheffield, UK
2
Department of Neurology, Evaggelismos General Hospital, Athens, Greece
3
IICD insigneo, University of Sheffield, Sheffield, UK
associated neuronal antibodies are not well characterized or proven pathogenic. The term Primary Autoimmune Cerebellar Ataxia (PACA) was introduced to describe this later group [1]. Progress in our ability to genetically characterise the ataxias, using next-generation
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