Phase II trial of natalizumab for the treatment of anti-Hu associated paraneoplastic neurological syndromes.

ABSTRACT:

Background

Paraneoplastic neurological syndromes with anti-Hu antibodies (Hu-PNS) have a very poor prognosis: more than half of the patients become bedridden and median survival is less than 12 months. Several lines of evidence suggest a pathogenic T cell-mediated immune response. Therefore, we conducted a prospective open-label phase II trial with natalizumab.

Methods

Twenty Hu-PNS patients with progressive disease were treated with a maximum of three monthly natalizumab cycles (300 mg). The primary outcome measure was functional improvement, this was defined as at least one point decrease in modified Rankin Scale (mRS) score at the last treatment visit. In addition, treatment response was assessed wherein a mRS score ≤3 after treatment was defined as treatment responsive.<

SUBMITTER: Bastiaansen AEM 

PROVIDER: S-EPMC8528262 | biostudies-literature | 2021 Jan-Dec

REPOSITORIES: biostudies-literature