title: Successful autologous haematopoietic stem cell transplantation for refractory myasthenia gravis - a case report.
abstract: 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.
authors.affiliation: Department of Clinical and Experimental Medicine, Linköping University, Linköping, Sweden; Department of Neurology, Region Östergötland, Linköping, Sweden. Electronic address: irene.hakansson@regionostergotland.se.
authors.forename: Irene
authors.initials: I
authors.lastname: Håkansson
authors.affiliation: Department of Haematology, Region Östergötland, Linköping, Sweden.
authors.forename: Anna
authors.initials: A
authors.lastname: Sandstedt
authors.affiliation: Department of Clinical and Experimental Medicine, Linköping University, Linköping, Sweden; Department of Neurology, Region Östergötland, Linköping, Sweden.
authors.forename: Fredrik
authors.initials: F
authors.lastname: Lundin
authors.affiliation: Department of Clinical Neuroscience (Neurology), Uppsala University Hospital, Uppsala, Sweden.
authors.forename: Håkan
authors.initials: H
authors.lastname: Askmark
authors.affiliation: Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.
authors.forename: Ritva
authors.initials: R
authors.lastname: Pirskanen
authors.affiliation: Department of Medical Sciences, Division of Haematology, University Hospital, Uppsala, Sweden.
authors.forename: Kristina
authors.initials: K
authors.lastname: Carlson
authors.affiliation: Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.
authors.forename: Fredrik
authors.initials: F
authors.lastname: Piehl
authors.affiliation: Department of Medical Sciences, Division of Haematology, University Hospital, Uppsala, Sweden.
authors.forename: Hans
authors.initials: H
authors.lastname: Hägglund
mesh_terms: D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008875:Middle Aged; D009157:Myasthenia Gravis / Q000601:surgery*
keywords: Autologous haematopoietic stem cell transplantation; Myasthenia gravis