Real-world outcomes after switching from standard therapy to efgartigimod in five patients with chronic inflammatory demyelinating polyradiculoneuropathy: a case series study in Japan - PubMed
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- #efgartigimod
- #CIDP treatment
- #real-world evidence
- Efgartigimod, a neonatal Fc receptor blocker, was approved in Japan in December 2024 for treating chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
- A case series study assessed switching five CIDP patients from standard therapy to efgartigimod, evaluating effectiveness via clinical scales and monitoring adverse events.
- All five patients showed a clinical response in at least one effectiveness measure after switching to efgartigimod.
- Three patients with typical CIDP experienced significant effectiveness, even with prior inadequate responses to intravenous or subcutaneous immunoglobulin.
- One patient with distal CIDP did not respond to efgartigimod treatment.
- One patient had a manageable severe headache as an adverse event.
- Effectiveness varied between typical CIDP and CIDP variants, possibly due to differences in immune pathophysiology among subtypes.
- Efgartigimod is considered a useful treatment option for CIDP in real-world practice, but further validation of findings is needed.