Authors: Franziska Scheibe, Harald Prüss, Annerose M. Mengel, Siegfried Kohler, Astrid Nümann, Martin Köhnlein, Klemens Ruprecht, Tobias Alexander, Falk Hiepe, Andreas Meisel
Summary:
Objective: We assessed the therapeutic potential of the plasma-cell-depleting proteasome inhibitor bortezomib in severe and therapy-refractory cases of anti–NMDA receptor (anti-NMDAR) encephalitis.
Methods: Five severely affected patients with anti-NMDAR encephalitis with delayed treatment response or resistance to standard immunosuppressive and B-cell-depleting drugs (corticosteroids, IV immunoglobulins, plasma exchange, immunoadsorption, rituximab, cyclophosphamide) who required medical treatment and artificial ventilation on intensive care units were treated with 1–6 cycles of 1.3 mg/m2 bortezomib. Occurrence of adverse events was closely monitored.
Results: Bortezomib treatment showed clinical improvement or disease remission, which was accompanied by a partial NMDAR antibody titer decline in 4 of 5 patients. With respect to disease severity, addition of bortezomib to the multimodal immunosuppressive treatment regimen was associated with an acceptable safety profile.
Conclusions: Our study identifies bortezomib as a promising escalation therapy for severe and therapy-refractory anti-NMDAR encephalitis.
Source:
Neurology; 2016, 10.1212/WNL.0000000000003536