A. Zabalza
Vall d’Hebron Institut de Recerca Centre d’Esclerosi Múltiple de CatalunyaAuthor Of 2 Presentations
FC03.01 - Defining controversies of benign MS using digital technology
Abstract
Background
Multiple-Sclerosis-Partners-Advancing-Technology-Health-Solutions (MSPATHS) is an international multicentre digital database that collects clinical information provided directly by patients together with standardized MRI and biomarkers.
Objectives
We identify a Benign multiple sclerosis (BMS) population using Patient-Determined-Disease-Steps (PDDS) as a proxy for EDSS. We describe its physical and non-physical characteristics, and explore the features that best discriminate BMS.
Methods
Cross-sectional study of MSPATHS patients (Feb 2019). In patients with disease duration ≥10 years, BMS was considered when PDDS score<2. We compared BMS and non-BMS in terms of (1)socio-demographic and clinical characteristics, (2)physical status (lower and upper extremity function by Neuro-QoL (LUEF-NQ) and neurological performance tests: walking speed test (WST), manual dexterity test (MDT), processing speed test (PST), contrast sensitivity test (CST)) and non-physical symptoms (anxiety, depression, fatigue, among other NQ domains), and (3)MRI (gadolinium enhancement and new T2 lesions). We built a random forest model to estimate the importance of each variable. Cohen’s d was used for descriptive statistics to categorize differences in small (d=0.2-0.5), medium (d=0.5-0.8) and large (d>0.8). A sensitivity analysis with a 1:1 matched cohort by disease duration was performed.
Results
From 15,257 patients included, 8,349 had a disease duration ≥10 years and 3,852 (46.1%) were classified as BMS. (1)BMS and non-BMS patients were similar for gender, age at disease onset and diagnosis, ethnicity, years of education and smoking status. Compared to non-BMS, BMS had small differences in disease duration (median, 17.2 (12,9-23,4) vs. 20.9 (15,1-28,8 years); d=0.39) but medium/large differences in (2)physical status (LUEF-NQ d=2.06 and 1.53, WST d=0.81, MDT d=0.97, PST d=0.82 and CST d=0.56), as well as, in all non-physical symptoms evaluated by NQ (anxiety d=0.53, depression d=0.69, fatigue d=0.84, stigma d=1.32, cognition d=0.69, social role satisfaction (SRS) d=1.11 and participation (SRP) d=1.19). (3)No differences were found on MRI activity. With 0.88 sensitivity and 0.86 specificity, LUEF-NQ was the most contributing variable for the random forest followed by stigma, SRP, WST, and SRS. The sensitivity analysis showed similar results.
Conclusions
PDDS seems to be a useful disability proxy to identify BMS when using digital technology. LUEF-NQ, stigma, SRP and SRS seem to better discriminate BMS.
PS01.05 - Rituximab treatment for MS: an observational multicentric dose comparison
Abstract
Background
Rituximab (RTX) is an anti-CD20 monoclonal antibody, widely used as an off-label treatment for multiple sclerosis (MS). Despite well-known efficacy and safety, RTX regimen has not yet been standardized.
Objectives
We aimed to compare efficacy and safety data of two different rituximab doses at two large Catalan multiple sclerosis centres.
Methods
A two-centre ambispective study considering all MS patients that have received at least one RTX cycle until February 2020 was conducted. In Barcelona centre (BC), RTX regimen used was 2g intravenously (IV), at least during 3 cycles, followed by 1g every 6 months, while in Girona centre (GC), was 2g IV, at least the first cycle, followed by 500mg every 6 months. Patients were clinically followed every 6 months with lab tests, and brain MRI scans were performed at baseline and yearly thereafter. Baseline clinical, radiological and demographic characteristics were collected. Annual relapse rate (ARR), contrast-enhancing lesions (CELs) and new T2 lesions at one and third year on treatment, as well as EDSS changes at last follow-up visit, were evaluated. Also, the dynamics of CD19% lymphocytes and IG immunoglobulin (IgG) values in serum, as well as the incidence of adverse events (AE) were described.
Results
A total of 303 patients (249 at BC and 54 at GC) were included. Main reason to start RTX was clinical progression plus inflammatory activity (clinical, radiological or both) (45.8% BC vs 79.6% GC). No differences on age at RTX onset, gender and disease duration were found between both centres. At baseline, mean ARR was 0.37±0.6 (BC) vs. 0.33±0.5 (GC); median EDSS was 5.5 (1-9.0) (BC) vs. 6.0 (1-8.0) (GC); and proportion of MRI with CELs was 32.4% (BC) vs. 42.6% (GC). ARR decrease to 0.05 (87.5%, p<0.001) for BC vs. 0.03 (90.3%, p=0.018) for GC at first year, and to 0.08 (88.3%, p=0.016) vs. 0 (100%, p=0.172) at third year. Considering only progressive MS phenotypes, 79.4% vs. 71.4% of patients remained stable or improved the EDSS. Regarding MRI findings, percentages of patients with CELs and new T2 lesions (BC vs GC) were 2.7% vs. 8% and 19% vs. 16% at one year; and 0% vs. 0% and 12% vs. 0% at third year. AE incidence was higher at BC during the first year (14.8% vs 4.1%). No difference in the dynamics of CD19% lymphocytes was found, while IgG values decreased significantly in the BC cohort throughout the first 3 years.
Conclusions
In the treatment of multiple sclerosis, low doses of rituximab seem to offer similar effectiveness with better safety profile than high doses.