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: Irene Håkansson, Anna Sandstedt, Fredrik Lundin, Håkan Askmark, Ritva Pirskanen, Kristina Carlson, Fredrik Piehl, Hans Hägglund
• Journal: Neuromuscular disorders : NMD (Neuromuscul Disord) Vol. 27 Issue 1 Pg. 90-93 (Jan 2017) ISSN: 1873-2364 [Electronic] England
• PMID: 27863874 (Publication Type: Case Reports, Journal Article)
• Copyright: Copyright © 2016 Elsevier B.V. All rights reserved.

Topics

• Female
• Hematopoietic Stem Cell Transplantation (methods)
• Humans
• Middle Aged
• Myasthenia Gravis (surgery)