Article ID Journal Published Year Pages File Type
5632414 Neuromuscular Disorders 2017 4 Pages PDF
Abstract

•A case of severe and treatment refractory myasthenia gravis (MG) is described.•She underwent an autologous haematopoietic stem cell transplantation (AHSCT).•Dramatic improvement of her MG was seen post transplantation.•AHSCT can be considered for selected cases of treatment refractory MG.

Myasthenia gravis (MG) is an autoimmune disease, with immune reactivity against the post-synaptic endplate of the neuromuscular junction. Apart from symptomatic treatment with choline esterase blockers, many patients also require immunomodulatory treatment. Despite existing treatment options, some patients are treatment refractory. We describe a patient with severe MG refractory to corticosteroids, four oral immunosuppressants, cyclophosphamide, rituximab and bortezomib who was treated with autologous haematopoietic stem cell transplantation. Two years after this, the patient has significantly improved in objective tests and in quality of life and leads an active life. Diplopia is her only remaining symptom and she is completely free of medication for MG. We believe that autologous haematopoietic stem cell transplantation can be an effective therapeutic option for carefully selected cases of severe, treatment refractory MG.

Related Topics
Life Sciences Neuroscience Developmental Neuroscience
Authors
, , , , , , , ,