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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Low-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).
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, China
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.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Distribution of mRS scores
Distribution of modified Rankin Scale (mRS) scores at scheduled follow-up time points.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
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.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Change in CASE score from baseline
Change in Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score from baseline at scheduled follow-up time points.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Relapse rate within 48 weeks
Relapse rate of autoimmune encephalitis and autoimmune cerebellitis within 48 weeks after treatment initiation.
Time frame: Up to Week 48 (±10 days)
Changes in MMSE scores from baseline
Changes in Mini-Mental State Examination (MMSE) scores from baseline.
Time frame: Week 3 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Changes in MoCA scores from baseline
Changes in Montreal Cognitive Assessment (MoCA) scores from baseline.
Time frame: Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Changes in PSQI scores from baseline
Changes in Pittsburgh Sleep Quality Index (PSQI) scores from baseline.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Changes in NPI scores from baseline
Changes in Neuropsychiatric Inventory (NPI) scores from baseline.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Changes in SDMT scores from baseline
Changes in Symbol Digit Modalities Test (SDMT) scores from baseline.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Changes in serum and CSF autoantibody titers from baseline
Changes in autoantibody titers in serum and cerebrospinal fluid (CSF) from baseline.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
Changes in peripheral blood lymphocyte subsets from baseline
Changes in peripheral blood lymphocyte subsets from baseline at scheduled follow-up time points.
Time frame: Week 3 (±1 day), Week 4 (±1 day), Week 5 (±1 day), Week 6 (±1 day), Week 8 (±1 day), Week 12 (±1 day), Week 24 (±3 days), Week 36 (±5 days), Week 48 (±10 days)
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