This study aims to retrospective investigate the safety and effectiveness of Efgartigimod in the acute phase of neuromyelitis optica spectrum disorders (NMOSD) patients.
This is a multicentre, controlled, retrospective, real-world study which aims to compare the safety and effectiveness of Intravenous methylprednisolone (IVMP) with Efgartigimod injection add-on treatment with IVMP treatment in acute NMOSD patients. Twenty-four patients from 6 centres in China will be enrolled.
Study Type
OBSERVATIONAL
Enrollment
24
IVMP 800-1000mg/day for 3-5 days plus Efgartigimod (Efgartigimod: 10mg/kg IV on Day 1, Day 8, Day 15 and Day 22 after IVMP.)
IVMP 800-1000mg/day for 3-5 days.
Change in Expanded Disability Status Scale (EDSS) score from baseline.
Change in Expanded Disability Status Scale (EDSS) score from baseline to 1 month after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
Time frame: 1 month
Change in Expanded Disability Status Scale (EDSS) score from baseline.
Change in Expanded Disability Status Scale (EDSS) score from baseline to 3 months, 6 months after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
Time frame: 3 months, 6 months
Percentage of Participants with Disability Improvement
Disability improvement is defined as a reduction in EDSS score of: A) ≥1.0 point from the baseline EDSS score when the baseline score was ≤5.5; B) ≥0.5 point when the baseline EDSS score \> 5.5(EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
Time frame: 1 month, 3 months, 6 months
Change in modified Rankin score (mRS) from baseline.
Change in modified Rankin score (mRS) from baseline at 1 month 1, 3 month 3 and 6 month (mRS: Minimum Score 0, Maximum score 6, higher scores mean a worse outcome).
Time frame: 1 month, 3 months, 6 months
Time to first relapse
Time frame: 6 months
Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI)
Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) at the last visit.
Time frame: 6 months
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Change in timed 25 Foot Walk Test from baseline.
Change in time taken to complete the timed 25 Foot Walk Test from baseline.
Time frame: 1 month, 3 months, 6 months
Number of NMOSD attacked during follow-up
Number of NMOSD treatment related to acute attack during follow-up.
Time frame: 6 months
Change in serum GFAP levels from baseline
Change in serum GFAP levels from baseline to the last visit.
Time frame: 1 month, 6 months
Change in AQP4-ab titres from baseline
Change in AQP4-ab titres from baseline to the last visit
Time frame: 1month, 6 months
Change in serum NfL levels from baseline
Change in serum NfL levels from baseline to the last visit
Time frame: 1 month, 6 months
Change in Visual Acuity (VA) from baseline
Change in Visual Acuity (VA) at1 month 1, and 6 month.
Time frame: 1 month, 6 months
Changes in EQ-5D-5L scores from baseline
Changes in EQ-5D scores from baseline to month 6(EQ-5D-5L: Minimum Score 5, Maximum score 25, lower scores mean a better quality of life).
Time frame: 6 months
Change in retinal nerve fibre layer (RNFL) loss from baseline
Change in retinal nerve fibre layer (RNFL) loss measured by optical coherence tomography (OCT) from baseline at month 1, month 6.
Time frame: 1 month,6 months
Adverse reactions during treatment and follow-up
Time frame: 6 months