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Autologous hematopoietic stem cell transplantation in a patient with refractory seropositive myasthenia gravis: A case report
•Refractory myasthenia represents only small percentage of patients with myasthenia.•Stem cell transplantation used in treatment of refractory severe autoimmune diseases.•Patient with refractory myasthenia that underwent stem cell transplantation.•Patient non-responsive to several lines of conventio...
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Published in: | Neuromuscular disorders : NMD 2019-02, Vol.29 (2), p.142-145 |
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Main Authors: | , , , , , , , |
Format: | Article |
Language: | English |
Subjects: | |
Citations: | Items that this one cites Items that cite this one |
Online Access: | Get full text |
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Summary: | •Refractory myasthenia represents only small percentage of patients with myasthenia.•Stem cell transplantation used in treatment of refractory severe autoimmune diseases.•Patient with refractory myasthenia that underwent stem cell transplantation.•Patient non-responsive to several lines of conventional treatment.•54 months after hematopoietic transplantation patient remains in complete remission.
Myasthenia gravis is a neuromuscular autoimmune disease characterized by fatigable weakness of skeletal muscles that results from an antibody-mediated immunological attack directed at acetylcholine postsynaptic receptors. Autologous hematopoietic stem cell transplantation is considered as a treatment option in refractory cases of myasthenia gravis. A 56-year-old Colombian male presented with six months of progressive hoarseness and dysphagia, with a positive repetitive stimulation test suggestive of end plate neuromuscular disease. Myasthenia gravis was confirmed with serology testing that reported presence of circulating acetylcholine postsynaptic receptors antibodies. The patient received several lines of pharmacological treatment and thymectomy without control of symptoms, requiring admission to the intensive care unit and mechanical ventilation in two occasions. Patient underwent autologous hematopoietic stem cell transplantation and has been in complete clinical remission for 65 months. Hematopoietic stem cell transplantation is a well-tolerated treatment that should be considered over conventional therapy in selected patients with refractory myasthenia gravis. |
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ISSN: | 0960-8966 1873-2364 |
DOI: | 10.1016/j.nmd.2018.11.008 |