PMID- 36527328
OWN - NLM
STAT- MEDLINE
DCOM- 20230124
LR  - 20230202
IS  - 1097-4598 (Electronic)
IS  - 0148-639X (Linking)
VI  - 67
IP  - 2
DP  - 2023 Feb
TI  - Autologous hematopoietic stem cell transplant for the treatment of refractory 
      myasthenia gravis with anti-muscle specific kinase antibodies.
PG  - 154-157
LID - 10.1002/mus.27772 [doi]
AB  - INTRODUCTION/AIMS: Up to 25% of patients with myasthenia gravis (MG) have 
      refractory disease despite trials of multiple immunosuppressants. Several case 
      series describe acetylcholine receptor antibody-positive (AChR) MG patients 
      treated with autologous hematopoietic stem cell transplant (HSCT). In this 
      report, we describe three patients with anti-muscle-specific kinase (MuSK) MG 
      treated with HSCT. METHODS: We included all patients who had undergone HSCT with 
      anti-MuSK myasthenia gravis identified through the records of the Alberta Blood 
      and Marrow Transplant Program. We collected demographic and clinical data 
      including validated MG scales as well as questionnaire data. RESULTS: All 3 
      patients had severe disease (Myasthenia Gravis Foundation of America score IVb-V) 
      and were refractory to multiple treatments, including rituximab. All patients 
      improved with no clinical manifestations or mild symptoms and remained as such 
      for 2, 3.5, and 5.5 y. Adverse events ranged from treatable infections and 
      transient dyspnea to persistent fatigue and premature menopause. The average 
      worst Myasthenia Gravis Activities of Daily Living (MG-ADL) scores improved from 
      14.7 before to 0.3 after HSCT. The mean worst Myasthenia Gravis Quality of Life 
      Questionnaire (MG-QoL15) scores improved from 26.7 to 0. All patients reported 
      they would undergo transplant again for their MG. DISCUSSION: We describe three 
      patients with anti-MuSK MG treated with HSCT, all of whom became symptom free 
      from MG with a tolerable side effect profile. In patients with severe refractory 
      anti-MuSK MG, it may be reasonable to consider HSCT.
CI  - (c) 2022 Wiley Periodicals LLC.
FAU - Beland, Benjamin
AU  - Beland B
AUID- ORCID: 0000-0002-9574-1412
AD  - Division of Neurology, Department of Clinical Neurosciences, Cumming School of 
      Medicine, University of Calgary, Calgary, Canada.
FAU - Hahn, Christopher
AU  - Hahn C
AD  - Division of Neurology, Department of Clinical Neurosciences, Cumming School of 
      Medicine, University of Calgary, Calgary, Canada.
FAU - Jamani, Kareem
AU  - Jamani K
AD  - Division of Hematology, Department of Medicine, Cumming School of Medicine, 
      University of Calgary, Calgary, Canada.
FAU - Chhibber, Sameer
AU  - Chhibber S
AD  - Division of Neurology, Department of Clinical Neurosciences, Cumming School of 
      Medicine, University of Calgary, Calgary, Canada.
FAU - White, Christopher
AU  - White C
AD  - Division of Neurology, Department of Clinical Neurosciences, Cumming School of 
      Medicine, University of Calgary, Calgary, Canada.
FAU - Atkins, Harold
AU  - Atkins H
AD  - Transplant and Cell Therapy Program, The Ottawa Hospital, Ottawa, Canada.
FAU - Storek, Jan
AU  - Storek J
AD  - Division of Hematology, Department of Medicine, Cumming School of Medicine, 
      University of Calgary, Calgary, Canada.
LA  - eng
PT  - Journal Article
DEP - 20221226
PL  - United States
TA  - Muscle Nerve
JT  - Muscle & nerve
JID - 7803146
RN  - 0 (Receptors, Cholinergic)
RN  - 0 (Autoantibodies)
SB  - IM
MH  - Female
MH  - Humans
MH  - Activities of Daily Living
MH  - Quality of Life
MH  - *Myasthenia Gravis/surgery/diagnosis
MH  - Receptors, Cholinergic
MH  - Autoantibodies
MH  - *Hematopoietic Stem Cell Transplantation
OTO - NOTNLM
OT  - MuSK
OT  - anti-acetylcholine receptor antibody
OT  - hematopoietic stem cell transplant
OT  - immunotherapy
OT  - myasthenia gravis
OT  - stem cells
EDAT- 2022/12/18 06:00
MHDA- 2023/01/25 06:00
CRDT- 2022/12/17 04:52
PHST- 2022/12/06 00:00 [revised]
PHST- 2022/06/06 00:00 [received]
PHST- 2022/12/11 00:00 [accepted]
PHST- 2022/12/18 06:00 [pubmed]
PHST- 2023/01/25 06:00 [medline]
PHST- 2022/12/17 04:52 [entrez]
AID - 10.1002/mus.27772 [doi]
PST - ppublish
SO  - Muscle Nerve. 2023 Feb;67(2):154-157. doi: 10.1002/mus.27772. Epub 2022 Dec 26.