Effectiveness and safety of efgartigimod in patients with anti‐NMDAR or anti‐LGI1 encephalitis and its 18 F‐DPA714 PET imaging evaluation: A multicenter, single‐arm, prospective, observational real‐world study
Observational study reveals significant clinical improvement and reduced neuroinflammation in anti-NMDAR and anti-LGI1 encephalitis, highlighting efgartigimod as a viable therapy.
Key Points
To assess the clinical efficacy, safety, and impact of efgartigimod on brain inflammation in patients diagnosed with anti-NMDAR or anti-LGI1 encephalitis.
Conducted a multicenter, single-arm, prospective observational study enrolling 18 patients (10 with anti-NMDAR encephalitis and 8 with anti-LGI1 encephalitis).
Administered intravenous efgartigimod at 10 mg/kg weekly for 2 to 4 doses, with or without concomitant immunosuppression, over a 12-week evaluation period.
Tracked clinical severity using mRS and CASE scales and monitored neuroinflammation using 18F-DPA714 PET/MRI in a subset of patients.
In anti-NMDAR encephalitis, median mRS scores decreased from 3 (range 1–5) to 1 (range 0–3; Z = -2.588, p = 0.007) and CASE scores dropped from 7.5 (range 1–27) to 1.5 (range 1–7; Z = -2.714, p = 0.005).
In anti-LGI1 encephalitis, median CASE scores declined from 3 (range 1–7) to 1.5 (range 0–2; Z = -2.154, p = 0.046) at final follow-up.
Follow-up 18F-DPA714 PET/MRI demonstrated reduced regional tracer uptake correlating with symptom improvement in 4 of 7 examined patients, with only one mild pneumonia event (1/18) reported.