Remission of severe myasthenia gravis after autologous stem cell transplantation

Author:

Schlatter Monica I.1,Yandamuri Soumya S.23,O'Connor Kevin C.23,Nowak Richard J.2ORCID,Pham Minh C.3,Obaid Abeer H.24,Redman Callee1,Provost Marie1,McSweeney Peter A.1,Pearlman Michael L.5,Tees Michael T.1,Bowen James D.6,Nash Richard A.1ORCID,Georges George E.7

Affiliation:

1. Colorado Blood Cancer Institute Denver Colorado 80218 USA

2. Department of Neurology Yale University School of Medicine New Haven Connecticut 06520 USA

3. Department of Immunobiology Yale University School of Medicine New Haven Connecticut 06520 USA

4. Institute of Biomedical Studies Baylor University Waco Texas 76706 USA

5. Sarah Cannon Research Institute Denver Colorado 80218 USA

6. Swedish Neuroscience Institute Seattle Washington 98122 USA

7. Clinical Research Division Fred Hutchinson Cancer Research Center Seattle Washington USA

Abstract

AbstractObjectiveMyasthenia gravis (MG) is an autoantibody‐mediated neuromuscular junction disorder involving the acetylcholine receptors on the motor endplate. The safety and response to high‐dose chemotherapy (HDIT) and autologous hematopoietic cell transplantation (HCT) were assessed in a patient with severe refractory MG.MethodsAs part of a pilot study of HDIT/HCT for patients with treatment‐resistant autoimmune neurological disorders, a patient with severe refractory MG underwent treatment. After mobilization of hematopoietic stem cells with rituximab, prednisone, and G‐CSF, the patient had HDIT consisting of carmustine, etoposide, cytarabine, melphalan, and rabbit antithymocyte globulin, followed by autologous HCT. The effect of treatment on the autoantibody to the acetylcholine receptor (AChR) was assessed.ResultsThe patient had been diagnosed with AChR antibody‐positive MG 14 years before HDIT/HCT and had failed thymectomy, therapeutic plasma exchange, and multiple immunomodulatory agents. The Myasthenia Gravis Foundation of America (MGFA) clinical classification was IVb before HDIT/HCT. She tolerated HDIT/HCT well and started to improve clinically within days of treatment. At both 1 and 2 years after HDIT/HCT, patients remained symptom‐free. After HDIT/HCT, AChR‐binding autoantibodies persisted, and the relative frequency of immune cell subtypes shifted.InterpretationHDIT/HCT induced a complete response of disease activity in a patient with severe refractory MG. This response may suggest that a cell‐mediated etiology may be a significant contributing factor in refractory MG cases. A phase 2 clinical trial is warranted to establish if HDIT/HCT can be an effective therapy for severe refractory MG and to gain a further understanding of disease pathogenesis.

Funder

Myasthenia Gravis Foundation of America

Rare Diseases Clinical Research Network

Publisher

Wiley

Subject

Neurology (clinical),General Neuroscience

Cited by 4 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. Multiple drugs;Reactions Weekly;2024-08-17

2. Innovationen in der Myasthenietherapie: eine Bestandsaufnahme;DGNeurologie;2024-02-19

3. Stem Cell Therapy in Neuroimmunologic Diseases;Reference Module in Neuroscience and Biobehavioral Psychology;2024

4. HSCT for stiff person syndrome and myasthenia gravis;Handbook of Clinical Neurology;2024

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3