A double-blind, placebo-controlled study of rituximab in patients with stiff person syndrome.

ABSTRACT:

Objective

In stiff person syndrome (SPS), an antibody-mediated impaired γ-aminobutyric acidergic (GABAergic) neurotransmission is believed to cause muscle stiffness and spasms. Most patients improve with GABA-enhancing drugs and intravenous immunoglobulin, but some respond poorly and remain disabled. The need for more effective therapy prompted a trial with the anti-CD20 monoclonal antibody rituximab.

Methods

This was a placebo-controlled randomized trial of rituximab (2 biweekly infusions of 1g each). The primary outcome was a change in stiffness scores at 6 months. Secondary outcomes were changes in heightened-sensitivity and quality of life scores. Enrolling 24 patients was calculated to detect 50% change in stiffness scores.

Results

Randomization was balanced for

SUBMITTER: Dalakas MC 

PROVIDER: S-EPMC5755707 | biostudies-literature | 2017 Aug

REPOSITORIES: biostudies-literature