Trial reportAnnals of neurology2017
A double-blind, placebo-controlled study of rituximab in patients with stiff person syndrome.
Trial report in Annals of neurology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
62 citing papers in PubMed, 4 syntheses or guidelines pooled it, 120 citations in OpenAlex.
- Efficacy of efgartigimod in the treatment of autoimmune encephalitis: a systematic review.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026Pooled it
- Comorbid autoimmune disease in stiff-person syndrome spectrum disorder: a systematic review and meta-analysis.Journal of neurology · 2026Pooled it
- Systematic Review of Immune and Symptomatic Treatments for Stiff-Person Syndrome.European journal of neurology · 2025Pooled it
- Pediatric stiff-person syndrome and related disorders: A systematic review.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025Pooled it
- Trial
- Eptinezumab in episodic migraine: A randomized, double-blind, placebo-controlled study (PROMISE-1).Cephalalgia : an international journal of headache · 2020Trial
- Optimizing the Use of Anti-CD20 Agents in Multiple Sclerosis and Other Autoimmune Central Nervous System Disorders: A Narrative Review.Neurology and therapy · 2026Review
- Phenotypic Spectrum, Diagnostic Challenges, and Clinical Outcomes in Stiff Person Syndrome: A Single-Center Case Series.Neurology international · 2026Article
- Stiff Person Syndrome: Bridging Neuroimmunology and Rehabilitation for Improved Patient Outcomes.Archives of internal medicine research · 2026Article
- Stiff-Person Syndrome: A Comprehensive Review of a Rare Disease with Emphasis on Pathophysiology, Clinical Features, Diagnostic Challenges and Therapeutic Advances.The Yale journal of biology and medicine · 2026Review
- Review
- Promising Effects of CAR T-Cell Therapy in Refractory Stiff Person Syndrome and a Hopeful Future for All Neuroautoimmunities.Neurology(R) neuroimmunology & neuroinflammation · 2026Review
- Sequential efgartigimod and ofatumumab for stiff-person syndrome: a case report illustrating a pathophysiology-informed strategy and literature review.Frontiers in immunology · 2026Review
- Clinical heterogeneity and immunotherapy outcomes in anti-GAD65 antibody-associated autoimmune encephalitis: a retrospective study.Therapeutic advances in neurological disorders · 2026Article
- Anti-CD19 CAR T-Cell Therapy in Advanced Stiff-Person Syndrome and Concomitant Myasthenia Gravis.Neurology(R) neuroimmunology & neuroinflammation · 2025Article
- Progressive encephalomyelitis with rigidity and myoclonus initially diagnosed as an anxiety disorder associated with anti-glycine receptor antibodies: A case report.The Journal of international medical research · 2025Article
- Double trouble: a curious case of non-paraneoplastic anti-voltage gated calcium channel Lambert-Eaton myasthenic syndrome with concurrent anti-glutamic acid decarboxylase-65 antibodies.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025Article
- Review
- Botulinum toxin as a potential adjunct therapy in stiff person syndrome spectrum disorders.Therapeutic advances in neurological disorders · 2025Article
- Chimeric antigen receptor T-cell therapy for stiff-person syndrome: bridging innovation and clinical challenges in neuroimmunology.Therapeutic advances in neurological disorders · 2025Review
2 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
Abstract
objectiveIn 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.
methodsThis 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.
resultsRandomization 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 < 0.01) at 3 months in both groups, but not at 6 months, denoting an early placebo effect. Blinded self-assessment rating of the overall stiffness for individual patients revealed improvement in 4 patients in each group. At 6 months, improvement persisted in 1 patient in the placebo group versus 3 of 4 in the rituximab group, where these meaningful improvements were also captured by video recordings.
interpretationThis is the largest controlled trial conducted in SPS patients and demonstrates no statistically significant difference in the efficacy measures between rituximab and placebo. Rituximab's lack of efficacy could be due to a considerable placebo effect; insensitivity of scales to quantify stiffness, especially in the less severely affected patients; or drug effectiveness in only a small patient subset. Ann Neurol 2017;82:271-277.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.