JAMA Neurology has recently published the results of a clinical trial performed on 7 cases of severe Myasthenia gravis (MG) treated with autologous HSCT in which consistent, durable, symptom-free, and treatment-free remission was achieved. Myasthenia gravis (MG) is a neuromuscular disease that leads to fluctuating muscle weakness and fatigue. In the most common cases, muscle weakness is caused by circulating antibodies that block acetylcholine receptors at the postsynaptic neuromuscular junction, inhibiting the excitatory effects of the neurotransmitter acetylcholine on nicotinic receptors at neuromuscular junctions.
Alternatively, in a much rarer form, muscle weakness is caused by a genetic defect in some portion of the neuromuscular junction that is inherited at birth as opposed to developing through passive transmission from the mother’s immune system at birth or through autoimmunity later in life.Some patients with myasthenia gravis (MG) do not respond to conventional treatment and have severe or life-threatening symptoms. Alternate and emerging therapies have not yet proved consistently or durably effective. Autologous haematopoietic stem cell transplant (HSCT) has been effective in treating other severe autoimmune neurologic conditions and may have similar application in MGThis study reports outcomes at The Ottawa Hospital, a large, Canadian, tertiary care referral center with expertise in neurology and HSCT, from January 1, 2001, through December 31, 2014, with a median follow-up of 40 months (range, 29-149 months).
Data collection and analysis were performed from February 1 through August 31, 2015. All patients with MG treated with autologous HSCT at The Ottawa Hospital were included. All had persistent severe or life-threatening MG-related symptoms despite continued use of intensive immunosuppressive therapies.Autologous haematopoietic stem cell grafts were mobilized with cyclophosphamide and granulocyte colony-stimulating factor, collected by peripheral blood leukapheresis, and purified away from contaminating lymphocytes using CD34 immunomagnetic selection. Patients were treated with intensive conditioning chemotherapy regimens to destroy the autoreactive immune system followed by graft reinfusion for blood and immune reconstitution.All patients achieved durable MG complete stable remission with no residual MG symptoms and freedom from any ongoing MG therapy. There were no treatment- or MG-related deaths.
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