Neuromyelitis Optica (NMO) is a rare, devastating demyelinating disease of the central nervous system (CNS) that has different causes and treatments from the more common demyelinating disease multiple sclerosis (MS). Current NMO therapies are nonspecific and have varying and often suboptimal benefit. The investigators will evaluate whether use of alpha1-antitrypsin (A1AT, an FDA-approved medication for patients with congenital deficiency of A1AT associated with emphysema) can benefit acute attacks of NMO, improving patient disability and quality of life.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
3-5 days 1000mg IV methylprednisolone at first presentation with acute attack.
Stanford University
Stanford, California, United States
Mean change in disability from baseline/nadir to week 24 as assessed by Opticospinal Impairment Score (OSIS) subscale.
Time frame: Baseline, Week 1: Day 2, Week 2, 3, 4, 8, 16, and 24.
Mean change in disability from baseline/nadir to week 24 as assessed by Expanded Disability Status Scale (EDSS).
Time frame: Baseline, Week 1: Day 2, Week 2, 3, 4, 8, 16, and 24.
For patients experiencing optic neuritis, mean change in visual acuity from baseline/nadir to week 24 as assessed by Sloan 2.5% low contrast visual acuity chart.
Time frame: Baseline, Week 1: Day 2, Week 2, 3, 4, 8, 16, and 24.
Mean change in retinal nerve fiber layer from baseline/nadir to week 24 as assessed by optical coherence tomography (OCT).
Time frame: Baseline and Week 24
Mean change in length of spinal cord lesion from baseline/nadir to week 24 as assessed by magnetic resonance imaging (MRI) T2 sequences.
Time frame: Baseline, Week 24
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