Trial reportAnnals of the New York Academy of Sciences2008
Rebooting the immune system with high-dose cyclophosphamide for treatment of refractory myasthenia gravis.
Trial report in Annals of the New York Academy of Sciences, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers, 3 of them syntheses that pooled it.
What it found
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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
39 citing papers in PubMed, 3 syntheses or guidelines pooled it, 92 citations in OpenAlex.
- Global prevalence of myasthenia gravis and the effectiveness of common drugs in its treatment: a systematic review and meta-analysis.Journal of translational medicine · 2021Pooled it
- Efficacy and Safety of Immunotherapies in Refractory Myasthenia Gravis: A Systematic Review and Meta-Analysis.Frontiers in neurology · 2021Pooled it
- Efficacy and safety of rituximab for myasthenia gravis: a systematic review and meta-analysis.Journal of neurology · 2015Pooled it
- Consistent improvement with eculizumab across muscle groups in myasthenia gravis.Annals of clinical and translational neurology · 2020Trial
- 'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab.Journal of neurology · 2020Trial
- High-dose cyclophosphamide without stem cell rescue in 207 patients with aplastic anemia and other autoimmune diseases.Medicine · 2011Trial
- Trial
- High-dose Cyclophosphamide Without Stem Cell Rescue in Autoimmune Diseases: A Systematic Review.European journal of rheumatology · 2024Review
- Current Treatment of Myasthenia Gravis.Journal of clinical medicine · 2022Review
- Survival, Prognosis, and Clinical Feature of Refractory Myasthenia Gravis: a 15-year Nationwide Cohort Study.Journal of Korean medical science · 2021Article
- Newer Immunotherapies for the Treatment of Acute Neuromuscular Disease in the Critical Care Unit.Current treatment options in neurology · 2020Review
- Management of Juvenile Myasthenia Gravis.Frontiers in neurology · 2020Review
- Long-Lasting Rituximab-Induced Reduction of Specific-But Not Total-IgG4 in MuSK-Positive Myasthenia Gravis.Frontiers in immunology · 2020Article
- Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study.Therapeutic advances in neurological disorders · 2020Article
- Employment in refractory myasthenia gravis: A Myasthenia Gravis Foundation of America Registry analysis.Muscle & nerve · 2019Article
- Article
- Clinical features and outcomes of patients with myasthenia gravis.Neurosciences (Riyadh, Saudi Arabia) · 2019Article
- When myasthenia gravis is deemed refractory: clinical signposts and treatment strategies.Therapeutic advances in neurological disorders · 2018Review
- Chinese Herbal Medicine for Myasthenia Gravis: A Systematic Review and Meta-Analysis.Frontiers in pharmacology · 2018Article
- High-dose cyclophosphamide without stem cell rescue in immune-mediated necrotizing myopathies.Neurology(R) neuroimmunology & neuroinflammation · 2017Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
A small but important proportion of patients with myasthenia gravis (MG) are refractory to conventional immunotherapy. We have treated 12 such patients by "rebooting" the immune system with high-dose cyclophosphamide (Hi Cy, 200 mg/kg), which largely eliminates the mature immune system, while leaving hematopoietic precursors intact. The objective of this report is to describe the clinical and immunologic results of Hi Cy treatment of refractory MG. We have followed 12 patients clinically for 1-9 years, and have analyzed their humoral and cellular immunologic parameters. Hi Cy is safe and effective. All but one of the patients experienced dramatic clinical improvement for variable periods from 5 months to 7.5 years, lasting for more than 1 year in seven of the patients. Two patients are still in treatment-free remission at 5.5 and 7.5 years, and five have achieved responsiveness to immunosuppressive agents that were previously ineffective. Hi Cy typically reduced, but did not completely eliminate, antibodies to the autoantigen AChR or to tetanus or diphtheria toxin; re-immunization with tetanus or diphtheria toxoid increased the antibody levels. Despite prior thymectomy, T cell receptor excision circles, generally considered to reflect thymic emigrant T cells, were produced by all patients. Hi Cy treatment results in effective, but often not permanent, remission in most refractory myasthenic patients, suggesting that the immune system is in fact "rebooted," but not "reformatted." We therefore recommend that treatment of refractory MG with Hi Cy be followed with maintenance immunotherapy.
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