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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Exposed group | Intravenous methylprednisolone (IVMP) plus Efgartigimod |
| |
| Control group | IVMP |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Intravenous methylprednisolone (IVMP) and Efgartigimod | Drug | 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.) |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 1 month |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 3 months, 6 months |
| Percentage of Participants with Disability Improvement |
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Inclusion Criteria:
Exclusion Criteria:
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NMOSD patients with acute attacks
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jinzhou Feng, Ph.D | Contact | 02389012487 | 203756@cqmu.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Jinzhou Feng, Ph.D | First Affiliated Hospital of Chongqing Medical University | Principal Investigator |
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| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D009188 | Myelitis, Transverse |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
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Blood samples are collected before the treatment and at the last visit.
| IVMP | Drug | IVMP 800-1000mg/day for 3-5 days. |
|
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). |
| 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). | 1 month, 3 months, 6 months |
| Time to first relapse | 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. | 6 months |
| Change in timed 25 Foot Walk Test from baseline. | Change in time taken to complete the timed 25 Foot Walk Test from baseline. | 1 month, 3 months, 6 months |
| Number of NMOSD attacked during follow-up | Number of NMOSD treatment related to acute attack during follow-up. | 6 months |
| Change in serum GFAP levels from baseline | Change in serum GFAP levels from baseline to the last visit. | 1 month, 6 months |
| Change in AQP4-ab titres from baseline | Change in AQP4-ab titres from baseline to the last visit | 1month, 6 months |
| Change in serum NfL levels from baseline | Change in serum NfL levels from baseline to the last visit | 1 month, 6 months |
| Change in Visual Acuity (VA) from baseline | Change in Visual Acuity (VA) at1 month 1, and 6 month. | 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). | 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. | 1 month,6 months |
| Adverse reactions during treatment and follow-up | 6 months |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |