To share our experience of bortezomib use in patients with refractory autoimmune-mediated neurological disorders and it’s safety characteristics.

Bortezomib use has not been widely used in the treatment of autoimmune neurological disorders.

In our single-center (AIMS, Kochi, India) retrospective cohort study, we describe clinical outcomes of combination immunosuppressive treatment including bortezomib (29 patients, the first bortezomib dose received from June, 2017 till September, 2021). We used the Wilcoxon signed rank test and univariate binary logistic regression (SPSS v.20) to assess outcomes.

Our cohort included 29 patients (aged 44±21 years, 15 males) with refractory and aggressive course of anti-NMDAR autoimmune encephalitis, seronegative autoimmune encephalitis, anti-DR2-encephalitis, Hashimoto encephalopathy, seropositive neuromyelitis optica spectrum disorder, seronegative optic neuritis, myasthenia gravis, transverse myelitis, post-infectious demyelination, vasculitic peripheral neuropathy, autoimmune atypical parkinsonism, autoimmune ataxia, cramp-fasciculation syndrome. A median number of subcutaneous bortezomib injections at the standard dose was 8 (4-9), with a maximum of 89. A follow-up from the first bortezomib injection was 19.4 (6.9-26.8) months. A median mRS-9Q score was 4 at bortezomib initiation; 3 at 60 days after bortezomib initiation (z=-3.59; p< .001) and the last documented mRS-9Q score in patients who had longer follow-up (n=27 [93%]; z=-2.40; p=.016). Out of 28 patients who had follow-up, 13 (46%) patients had no registered AE, 4 (14%) had mild and moderate AE, 8 (29%) had severe but not immediately life-threatening AE, 1 (4%) – life-threatening AE, and 2 died because of SARS-CoV2 infection on active treatment with BTZ and RIX or several months after the last dose of these drugs.

In our cohort of patients with refractory and aggressive autoimmune neurological diseases treatment with bortezomib appeared to improve mRS-9Q scores; infection was the most common side effect (around half of cases) which, however, could not be attributed to bortezomib use alone.