Predictors for the severity of acute aquaporin-4 immunoglobulin G-seropositive neuromyelitis optica spectrum disorders: a prospective cohort study

Clinical spectrum and prognostic features of patients seropositive for anti-GD1a antibody

Background Anti-GD1a antibodies are associated with a range of immune-mediated neuropathies, particularly acute motor axonal neuropathy (AMAN) and other Guillain-Barré syndrome (GBS) variants. However, their full clinical spectrum and prognostic significance remain unclear due to limited systematic studies. Here, we investigated the clinical phenotypes, serological profiles, cerebrospinal fluid (CSF) characteristic, and therapeutic outcomes associated with anti-GD1a antibodies. Methods The clinical, paraclinical and therapeutic data were retrospectively collected and analyzed from 19 Chinese patients who tested positive for anti-GD1a antibodies. Results The mean age at onset in this cohort was 50.6 years. Among the 19 patients, 14 (74%) presented with acute syndromes. The remaining five patients (26%) exhibited features consistent with chronic neuropathies. Common manifestations included absent tendon reflexes (84%), limb weakness (84%), sensory impairment (58%), and cranial nerve involvement (53%). Isolated anti-GD1a antibody positivity was observed in only 5 patients, while others had co-existing antiganglioside antibodies, most commonly anti-GQ1b, and anti-GM1. Electrophysiological studies revealed axonal and demyelinating neuropathic patterns. Patients with cranial nerve involvement and albuminocytologic dissociation were associated with higher modified Erasmus GBS Outcome Score (mEGOS). Immunotherapy was effective in acute cases, with 57% achieving complete recovery at 6-month follow-up. Conclusions Patients seropositive for anti-GD1a antibody exhibits significant clinical and electrophysiological heterogeneity and frequently coexists with other antiganglioside antibodies. Acute presentations generally respond well to immunotherapy, while chronic cases require individualized management.

Patient-reported outcomes in Chinese patients with AQP4-IgG–positive neuromyelitis optica spectrum disorder on inebilizumab therapy: a cross-sectional study

The treatment of neuromyelitis optica spectrum disorder (NMOSD) has entered the era of targeted biological agents. While relapse control has improved substantially, patient-reported outcomes (PROs) remain understudied. This study aimed to explore real-world patient-reported perceptions of inebilizumab therapy in AQP4-IgG–positive NMOSD. We conducted a nationwide cross-sectional survey of AQP4-IgG–positive NMOSD patients receiving inebilizumab using an anonymous online questionnaire (April–September 2025). Data collected included demographics, medication history, and multidimensional quality-of-life changes following inebilizumab initiation. Descriptive statistics and correlation analyses were performed. Key outcomes were patient-reported without objective validation. A total of 275 valid questionnaires were analyzed. Respondents were predominantly female (90.5%) with a mean age of 39.7 ± 11.8 years. Mean inebilizumab treatment duration was 14.5 ± 8.4 months. Of all patients, 33.1% received inebilizumab as initial maintenance therapy, 42.9% had previously used conventional immunosuppressants, and 24.0% had switched from other biologics. Among respondents, 92.4% reported meaningful symptom improvement across multiple domains including bodily pain and mental health. Overall, 96.4% expressed willingness to continue therapy, with symptom improvement cited as the predominant reason (91.7%). Correlation analysis revealed weak associations between treatment duration and satisfaction (r = 0.12, p < 0.05) and between medication cost burden and satisfaction (r = − 0.24, p < 0.01). In this cross-sectional survey, Chinese patients with AQP4-IgG–positive NMOSD receiving inebilizumab reported high satisfaction, perceived multidimensional benefit, and strong treatment continuation willingness. These findings provide exploratory patient-reported evidence regarding the acceptability of inebilizumab in routine practice. However, the observed improvements are self-reported and associative rather than causal; interpretation should account for the cross-sectional, non-comparative design, potential selection and recall biases, and lack of objective outcome validation. Prospective controlled studies are required to confirm these preliminary findings.

Clinical and serological features of systemic autoimmune overlap in AQP4-IgG positive neuromyelitis optica spectrum disorder: a Mexican cohort study.

Systemic autoimmune overlap was present in approximately one in five patients and the overlap group showed more frequent systemic autoantibody positivity and a predominance of optic neuritis at onset, but disability measures, treatment patterns, and most clinical outcomes were similar between groups.

Age-stratified clinical characteristics and disability outcomes in neuromyelitis optica spectrum disorder: A cross-sectional study.

Therapeutic Plasma Exchange in Neuromyelitis Optica Spectrum Disorder: Real-World Clinical Outcomes and Safety From a Tertiary Care Center in Northwest India.

BACKGROUND AND OBJECTIVES Neuromyelitis Optica Spectrum Disorder (NMOSD) is a severe autoimmune demyelinating disorder of the central nervous system characterized by relapsing attacks that can result in permanent neurological disability. Therapeutic Plasma Exchange (TPE) is recommended for steroid-refractory acute exacerbations. This study evaluated the efficacy and safety of TPE in patients with acute NMOSD attacks. MATERIALS AND METHODS A prospective observational study was conducted over 18 months at SMS Jaipur, India. Forty adult patients fulfilling the 2015 International Panel for NMO Diagnosis criteria and presenting with steroid-refractory acute attacks were enrolled. TPE was performed using continuous-flow centrifugation, exchanging 1.0-1.4 estimated plasma volumes per session for 3-7 alternate-day procedures. Clinical outcomes were assessed using the EDSS, Snellen visual acuity, and MRC muscle strength grading. RESULTS The cohort had a female predominance (65.0%) with a mean age of 36.5 years. Acute first presentations accounted for 77.5% of cases, while 22.5% were relapses. Anti-aquaporin-4 antibody positivity was detected in 45.0% of patients. Among 37 patients with motor deficits, 94.6% showed clinical improvement following TPE, including marked (35.1%), moderate (43.2%), and mild (16.2%) responses. Mean EDSS improved significantly from 7.4 to 4.1 (95% CI and p < 0.001). Visual improvement was observed in 84.4% of patients with baseline visual impairment. Adverse events occurred in 25.0% of patients, predominantly transient hypotension and mild citrate-related hypocalcemia, with no procedure-related treatment discontinuation. CONCLUSION TPE is a safe and effective rescue therapy for steroid-refractory NMOSD exacerbations, producing substantial neurological and visual recovery with a favorable safety profile.

Serum SERPINA3 as a Candidate Non-Invasive Biomarker for Neuromyelitis Optica Spectrum Disorder: Proteomic Discovery and Same-Center Validation

SERPINA3 is therefore an exploratory adjunctive serum biomarker candidate rather than a stand-alone diagnostic test; prospective external validation in clinically representative cohorts is required.