M. Hellmann
8Rabin Medical Center, Belinson Hospital

Author Of 2 Presentations

 On-Demand Program
Imaging Late Breaking Abstracts

LB1177 - PAMRINO: International MRI and clinical data repository for neuromyelitis optica spectrum disorder (ID 469)

Speakers
Authors
Presentation Number
LB1177
Presentation Topic
Imaging

Abstract

Background

Neuromyelitis optica spectrum disorders (NMOSD) encompasses a group of rare inflammatory diseases which primarily target the optic nerves, spinal cord, and brain. Typically, magnetic resonance imaging (MRI) data from single-center studies comprise 20-50 patients, limiting statistical power for outcomes research. Using retrospective data from the PArallel MRI in NmOsd (PAMRINO) study, a novel prospective NMOSD image repository (NMOsDIR) representing multiple international sites was coordinated by Charité-Universitätsmedizin Berlin and the Medical Image Analysis Center (Basel).

Objectives

The PAMRINO study aimed to investigate and analyze retrospective MRIs collected from NMOSD-specialized centers, potentially for the evaluation of disease-related brain and spinal cord changes. NMOsDIR serves as an international imaging research resource (comprising standardized retinal optical coherence tomography and MRI scans) and clinical data hub for prospective studies in NMOSD. Linking imaging and clinical data, as well as enabling analysis pipelines for each modality, will facilitate multi-centered studies using sufficient data and statistical power to advance outcomes research in this rare disease.

Methods

For clinical data collection in PAMRINO, a Research Electronic Data Capture (REDCap) platform was used, where participating centers entered data relevant for NMOSD patient monitoring. An image database (XNAT) was established for image uploads. This large collection of MRI data is currently being analyzed in a joint international effort of NMOSD clinical neuroradiologists and scientists.

Results

Brain, spinal cord, and optic nerve MRI scans with associated clinical data were collected from 514 NMOSD patients and 56 healthy controls from 17 international centers. Roughly 20,000 individual MRI scans from patients and healthy controls were collected. Of these, 78% had T1-weighted cerebral MRIs (55% with 3D scans), 80% had T2-weighted cerebral MRIs (54% with 3D scans), 86% had T2-weighted spinal cord MRIs (55% with 3D scans), and 35% had optic nerve MRIs.

Conclusions

We successfully established PAMRINO, an international collaborative retrospective MRI and clinical data repository. The knowledge gained during this process provided important new insights, where the initial analysis of the dataset has underscored the large degree of heterogeneity in image and clinical data collection in NMOSD-specialized centers. Thus, calling for more standardized methods of data acquisition and imaging analysis, as not to limit research opportunities. The new longitudinal, prospective NMOsDIR will help us to answer many pressing - yet open - questions regarding patients seropositive for aquaporin-4-IgG+, myelin oligodendrocyte glycoprotein-IgG+ and other autoimmune-related diseases. In turn, such a strategy will strengthen future capabilities in research, diagnosis, monitoring and improving NMOSD patient care.

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Clinical Trials Late Breaking Abstracts

LB1227 - Glatiramer Acetate Depot (extended-release) phase IIa study in patients with Primary Progressive Multiple Sclerosis: safety and efficacy snapshot (ID 2106)

Speakers
Authors
Presentation Number
LB1227
Presentation Topic
Clinical Trials

Abstract

Background

PPMS is characterized by worsening neurologic function (accumulation of disability) from the onset of symptoms, without early relapses or remissions. GA long-acting injection (GA Depot) consists of extended-release microspheres containing GA, administered intramuscularly (IM) once every 28 days. Results of GA Depot phase IIa for three years in relapsing remitting MS suggest that GA Depot is safe, tolerable and efficacious. The IM administration route together with the slow release formulation may result in a noted effect on PPMS patients as well.

Objectives

To assess the safety and efficacy snapshot data of GA Depot treatment (for up to 112 weeks) in the eleven primary progressive MS (PPMS) subjects enrolled out of the 24 planned.

Methods

Eligibility criteria included: age 18-65 years, subjects diagnosed with PPMS with signs of rapid disease progression (rate of ≥ 1 point increase / year on EDSS score) in the year prior to screening, EDSS score of ≥2.0 and ≤ 6.5 at baseline. Patients are receiving GA Depot IM at a dose of 40 mg every 28 days. Safety is assessed by analysis of adverse events, CBC and blood chemistry. Efficacy is assessed by EDSS, 9HPT, T25FW tests, as well as by MRI analysis.

Results

AEs were mainly mild. Most common AEs included injection site reactions and general weakness. No unexpected AEs were reported. Two SAEs were reported (one related and one not related to study drug). EDSS score remained stable for all patients and no 12 weeks confirmed disability progression (CDP) was detected. Mean 9HPT score and T25FW remained stable. MRI analysis (compared to baseline) revealed findings in three out of the 11 patients’ population.

Conclusions

These interim snapshot data suggest that GA Depot is possibly a safe and effective treatment for patients with PPMS, as demonstrated by stable mean EDSS, mean 9HPT and mean T25FW data, which encourage us to continue this on-going investigation.

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