Evidence map›Paper›PMID 37726935›Full record

ArticleAnnals of clinical and translational neurology2023

Remission of severe myasthenia gravis after autologous stem cell transplantation.

Monica I Schlatter, Soumya S Yandamuri, Kevin C O'Connor, Richard J Nowak, Minh C Pham, Abeer H Obaid, Callee Redman, Marie Provost, Peter A McSweeney, Michael L Pearlman and 4 more

Open access · goldAbstract read
In one paragraph

Article in Annals of clinical and translational neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
2.5field-weighted citation impact, top 10% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 12 citations in OpenAlex.

  1. Review
  2. New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    Review
  3. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors at 6 institutions in 2 countries.

Monica I SchlatterColorado Blood Cancer Institute, Denver, Colorado, 80218, USA.
Soumya S YandamuriDepartment of Neurology, Yale University School of Medicine, New Haven, Connecticut, 06520, USA.
Kevin C O'ConnorDepartment of Neurology, Yale University School of Medicine, New Haven, Connecticut, 06520, USA.
Richard J NowakDepartment of Neurology, Yale University School of Medicine, New Haven, Connecticut, 06520, USA.ORCID 0000-0001-8438-482X
Minh C PhamDepartment of Immunobiology, Yale University School of Medicine, New Haven, Connecticut, 06520, USA.
Abeer H ObaidDepartment of Neurology, Yale University School of Medicine, New Haven, Connecticut, 06520, USA.
Callee RedmanColorado Blood Cancer Institute, Denver, Colorado, 80218, USA.
Marie ProvostColorado Blood Cancer Institute, Denver, Colorado, 80218, USA.
Peter A McSweeneyColorado Blood Cancer Institute, Denver, Colorado, 80218, USA.
Michael L PearlmanSarah Cannon Research Institute, Denver, Colorado, 80218, USA.
Michael T TeesColorado Blood Cancer Institute, Denver, Colorado, 80218, USA.
James D BowenSwedish Neuroscience Institute, Seattle, Washington, 98122, USA.
Richard A NashColorado Blood Cancer Institute, Denver, Colorado, 80218, USA.ORCID 0009-0005-2683-5182
George E GeorgesClinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.
Colorado Blood Cancer Institute · USYale University · USBaylor University · USFred Hutch Cancer Center · USSarah Cannon Research Institute · GBSwedish Medical Center · US

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
Rare Disease Network for Myasthenia GravisU54NS115054 · NINDS · GEORGE WASHINGTON UNIVERSITY · PI HENRY J KAMINSKI · 2019 to 2026
$11.9M
Mechanisms of autoimmunity in myasthenia gravisR01AI114780 · NIAID · YALE UNIVERSITY · PI Kevin C O'Connor · 2016 to 2026
$4.7M
Molecular determinants driving diverse mechanisms of antibody-mediated pathologyR21AI164590 · NIAID · YALE UNIVERSITY · PI O'CONNOR, KEVIN C · 2021 to 2022
$461k
Mechanisms of autoantibody production in myasthenia gravisR56AI114780 · NIAID · YALE UNIVERSITY · PI O'CONNOR, KEVIN C · 2015 to 2015
$416k
NCATS NIH HHS UL1 TR001863NIAID NIH HHS R01 AI114780NIAID NIH HHS R21 AI164590NIAID NIH HHS R56 AI114780NINDS NIH HHS U54 NS115054
6 · The paper itself

Abstract

objectiveMyasthenia 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.

Indexed as

Hematopoietic Stem Cell TransplantationMyasthenia GravisAutoantibodiesFemaleHumansPilot ProjectsReceptors, CholinergicTransplantation, AutologousTreatment OutcomeAutoantibodiesReceptors, Cholinergic

Identifiers

PMID37726935
PMCPMC10646993
OpenAlexW4386878536

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.