Evidence map›Paper›PMID 39470879›Full record

ArticleAdvances in therapy2024

Risk-Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis.

A Gordon Smith, Gil I Wolfe, Ali A Habib, Cynthia Z Qi, Hongbo Yang, Mandy Du, Xin Chen, Deborah Gelinas, Edward Brauer, Glenn Phillips and 1 more

Abstract readComparative StudyNetwork Meta-Analysis
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

13 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
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  5. Review
  6. Review
  7. Review
  8. Review
  9. Article
  10. New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    Review
  11. Observational
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  13. 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

11 authors.

A Gordon SmithDepartment of Neurology, Virginia Commonwealth University, Richmond, VA, USA.
Gil I WolfeDepartment of Neurology, University at Buffalo/SUNY, Buffalo, NY, USA.
Ali A HabibDepartment of Neurology, University of California-Irvine Medical Center, Irvine, CA, USA.
Cynthia Z QiArgenx, Inc., Boston, MA, USA. cqi@argenx.com.
Hongbo YangAnalysis Group, Inc., Boston, MA, USA.
Mandy DuAnalysis Group, Inc., Boston, MA, USA.
Xin ChenAnalysis Group, Inc., Boston, MA, USA.
Deborah GelinasArgenx, Inc., Boston, MA, USA.
Edward BrauerArgenx, Inc., Boston, MA, USA.
Glenn PhillipsArgenx, Inc., Boston, MA, USA.
Francesco SaccàGENESIS Department, University of Naples Federico II, Naples, Campania, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThis study used network meta-analysis (NMA) to inform and compare the number needed to treat (NNT), number needed to harm (NNH), and cost per improved outcome (CPIO) associated with more recently approved treatments for anti-acetylcholine receptor antibody-positive (anti-AChR Ab+) generalized myasthenia gravis (gMG).

methodsClinical trials of neonatal Fc receptor (FcRn) inhibitors, efgartigimod intravenous (IV) and rozanolixizumab, and complement inhibitors, ravulizumab and zilucoplan, versus placebo (with background conventional treatment) were included in the primary NMA to compare efficacy and safety outcomes. The outputs from the NMAs were used to estimate NNT and NNH of each treatment versus placebo. CPIO (2024 USD) was estimated for a ≥ 3- or ≥ 5-point reduction from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores. Sensitivity analyses were performed adding efgartigimod PH20 subcutaneous (SC) and eculizumab to the NMA.

resultsEfgartigimod IV had the lowest NNT versus placebo for achieving a ≥ 3- and ≥ 5-point reduction in QMG, as well as a ≥ 5-point reduction in MG-ADL, whereas rozanolixizumab had the lowest NNT for a ≥ 3-point reduction in MG-ADL. The NNH versus placebo was similar across comparator treatments. Efgartigimod IV had the lowest CPIO among all treatments for all assessed efficacy outcomes. Sensitivity analyses yielded results consistent with primary analysis and indicated that efgartigimod PH20 SC had comparable NNT and CPIO values to efgartigimod IV, whereas eculizumab had comparable NNT and higher CPIO values compared to other complement inhibitors.

conclusionsFcRn inhibitors and complement inhibitors assessed in this study all demonstrated clinical benefit in terms of NNT as well as an acceptable safety profile in terms of NNH. Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+ gMG.

Indexed as

Myasthenia GravisAntibodies, Monoclonal, HumanizedComplement Inactivating AgentsHumansRisk AssessmentTreatment OutcomeAntibodies, Monoclonal, HumanizedComplement Inactivating AgentsEculizumabEfficacyEfgartigimodGeneralized myasthenia gravisNetwork meta-analysisRavulizumabRisk–benefit analysisRozanolixizumabSafetyZilucoplan

Identifiers

PMID39470879
PMCPMC11550228

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Registered trials

None linked

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.