This is a multicenter, single-arm, continuous, prospective, interventional registry study designed to systematically evaluate the efficacy and safety of low-dose blinatumomab in patients with antibody-mediated refractory autoimmune encephalitis (AE) and autoimmune cerebellitis. Eligible participants will be patients with a confirmed diagnosis of refractory AE or autoimmune cerebellitis who have provided written informed consent. All enrolled patients will receive blinatumomab treatment according to a unified protocol, consisting of two cycles:
Cycle 1 (Week 1): Continuous intravenous infusion at 9 µg/day for 5 consecutive days (total dose: 45 µg).
Cycle 2 (Week 3): Continuous intravenous infusion at 9 µg/day for 5 consecutive days (total dose: 45 µg). If there is no improvement in the modified Rankin Scale (mRS) score at Week 3 and the proportion of peripheral blood B cells (CD3-/CD19+) remains \>1%, the dose may be optimized to 15 µg/m²/day (maximum 28 µg/day).
During the study, all patients will undergo regular follow-up visits to collect data on clinical symptoms, functional scores, immunological biomarkers, and adverse events, to comprehensively assess the efficacy and safety of the treatment. This study uses a non-randomized, open-label design with no blinding or control group.
Aged ≥18 years, male or female.
Confirmed diagnosis of antibody-positive autoimmune encephalitis (AE) or autoimmune cerebellitis (ACA).
Modified Rankin Scale (mRS) score >3 (stable for at least 24 hours) at baseline.
Received first-line acute therapy more than 6 weeks prior to baseline visit, defined as at least 3 days of intravenous methylprednisolone (≥500 mg/day) or equivalent oral corticosteroids, and/or at least 3 days of IVIG and/or plasma exchange (PE), or any combination thereof.
Systemic or central nervous system tumors (e.g., gliomatosis cerebri), history of cancer (excluding ovarian/extra-ovarian teratoma, skin squamous cell carcinoma, or basal cell carcinoma with documented cure ≥3 months prior to enrollment); hereditary diseases (e.g., mitochondrial encephalopathy); neurodegenerative diseases (e.g., Lewy body dementia); prior epilepsy with ongoing seizures; severe traumatic brain injury; metabolic/toxic encephalopathy (e.g., Wernicke encephalopathy).
Infectious diseases (e.g., viral encephalitis); active or uncontrolled infection requiring systemic treatment within 1 week prior to screening; history of severe recurrent or chronic infections, especially respiratory infections.
Positive hepatitis B surface antigen/核心 antigen and/or positive hepatitis C PCR at screening.
Active tuberculosis at screening.
Single group
Blinatumomab
DrugLow-dose blinatumomab administered intravenously in two cycles: Cycle 1 (Week 1) at 9 μg/day for 5 consecutive days, and Cycle 2 (Week 3) at 9 μg/day for 5 consecutive days (45 μg per cycle). If no clinical improvement is observed at Week 3 and peripheral blood B-cell proportion (CD3-/CD19+) remains \>1%, the dose may be optimized to 15 µg/m²/day (maximum 28 µg/day).
Proportion of participants with mRS score 0-2
Proportion of participants with modified Rankin Scale (mRS) score of 0 to 2 points at scheduled follow-up time points.
Distribution of mRS scores
Distribution of modified Rankin Scale (mRS) scores at scheduled follow-up time points.
Proportion of participants with mRS improvement ≥1 point
Proportion of participants with a decrease of ≥1 point in modified Rankin Scale (mRS) score from baseline at scheduled follow-up time points.
Change in CASE score from baseline
Change in Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score from baseline at scheduled follow-up time points.
Changes in MMSE scores from baseline
Changes in Mini-Mental State Examination (MMSE) scores from baseline.
Changes in MoCA scores from baseline
Changes in Montreal Cognitive Assessment (MoCA) scores from baseline.
Changes in PSQI scores from baseline
Changes in Pittsburgh Sleep Quality Index (PSQI) scores from baseline.
Changes in NPI scores from baseline
Changes in Neuropsychiatric Inventory (NPI) scores from baseline.
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