A Novel Sequential Regimen of Efgartigimod and Telitacicept as Fast-Acting Treatment for Impeding Crisis of MuSK-MG.
This case report highlights the potential of using efgartigimod and telitacicept sequentially to manage severe MuSK-MG, but further studies are needed to confirm these findings.
Where it sits
this study against the rest of the efpeglenatide corpusSummary and findings
This case report describes the sequential treatment of a patient with late-onset MuSK-MG using efgartigimod and telitacicept. The patient achieved minimal symptom expression within 1.5 months, with significant improvements in QMG and MG-ADL scores. IgG levels and MuSK-Ab titers were also reduced.
Abstract
<h4>Background</h4>Myasthenia gravis (MG) is an autoimmune disorder of the neuromuscular junction. The muscle-specific kinase (MuSK) antibody-positive subtype often involves bulbar and respiratory muscles, conferring a high risk of myasthenic crisis.<h4>Method</h4>We report a very late-onset MuSK-MG case complicated by lung infection and acute heart failure, with a history of peptic ulcer. The patient was sequentially treated with efgartigimod and telitacicept, achieving minimal symptom expression (MSE) within 1.5 months.<h4>Discussion</h4>Following the first efgartigimod infusion, QMG and MG-ADL scores improved from 31 to 23 and 22 to 12, respectively. After five injections of telitacicept, symptoms resolved completely on day 47, with scores further reducing to 2 (QMG) and 0 (MG-ADL). Treatment also reduced IgG levels and MuSK-Ab titers, while IgM remained stable.<h4>Conclusion</h4>This case demonstrates the successful sequential use of efgartigimod and telitacicept in managing complicated, late-onset MuSK-MG in a state of impeding crisis.
Background
Myasthenia gravis (MG) is an autoimmune disorder affecting the neuromuscular junction, particularly the MuSK antibody-positive subtype, which can lead to severe complications such as myasthenic crisis. Previous treatments have had varied success, and there is a need for effective management strategies in severe cases. This study reports on a novel sequential treatment approach using efgartigimod and telitacicept in a patient with complicated late-onset MuSK-MG.
Methods
This is a case report of a patient with late-onset MuSK-MG who was treated sequentially with efgartigimod and telitacicept. The treatment duration was 1.5 months for symptom expression, with follow-up until day 47. Primary outcome measures included QMG and MG-ADL scores.
Results
The primary endpoint showed a QMG score improvement from 31 to 23 after the first efgartigimod infusion. The MG-ADL score improved from 22 to 12 following the same treatment. After five injections of telitacicept, complete symptom resolution was observed by day 47, with QMG and MG-ADL scores of 2 and 0, respectively.
Interpretation
The findings suggest that the sequential use of efgartigimod and telitacicept may be effective in managing severe MuSK-MG, although the clinical significance of the observed score improvements should be interpreted with caution due to the lack of a control group. The small sample size and absence of long-term follow-up data limit the conclusions that can be drawn about the efficacy and durability of this treatment approach.
Key findings
- QMG score improved from 31 to 23 after the first efgartigimod infusion.
- MG-ADL score improved from 22 to 12 after the first efgartigimod infusion.
- Complete symptom resolution occurred by day 47, with QMG score reducing to 2 and MG-ADL score to 0.
- Treatment reduced IgG levels and MuSK-Ab titers, while IgM remained stable.
Limitations
- single case report with no control group
- lack of long-term follow-up data
- small sample size limits generalizability