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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 ?-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 age, sex, disease duration, and glutamic acid decarboxylase autoantibody titers. No significant changes were noted at 6 months after treatment in all outcomes. Specifically, no differences were noted in the stiffness index, the primary outcome, or sensitivity scores, the secondary outcome, at 3 or 6 months. Quality of life scores improved significantly (p?

SUBMITTER: Dalakas MC

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

REPOSITORIES: biostudies-literature

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

Dalakas Marinos C MC Rakocevic Goran G Dambrosia James M JM Alexopoulos Harry H McElroy Beverly B

Annals of neurology 20170809 2

<h4>Objective</h4>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.<h4>Methods</h4>This was a placebo-controlled randomized trial of rituximab (2 biweekly ...[more]

PMID: 28749549

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

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

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