Author Of 1 Presentation
YI02.02 - Assessing No Evidence of Disease Activity in Pediatric Onset Multiple Sclerosis
Abstract
Background
Pediatric onset multiple sclerosis (POMS) account for 5-10% of all MS cases. The optimal treatment target in POMS is not known. No Evidence of Disease Activity (NEDA), defined as the lack of clinical and radiologic evidence of MS activity, has been proposed as a possible outcome measure and treatment target in MS.
Objectives
We aim to determine POMS NEDA rate, compare NEDA and evidence of disease activity (EDA) patient characteristics, and determine NEDA predictors.
Methods
Retrospective analyses of POMS cases from the Multiple Sclerosis and other Demyelinating Diseases Database (PedMSDD) were conducted. Multiple imputation was employed to reduce bias relative to complete-case methods. NEDA proportions at 12, 18, and 24 months post-diagnosis were calculated. Demographic and clinical disease characteristics between NEDA and EDA were compared. The odds of achieving NEDA at each time interval were modeled using separate univariable logistic regressions, and significant predictors were included in final multivariable models.
Results
Demographics and clinical characteristics were similar between NEDA and EDA. In patients with at least six months of follow-up (N=913), an estimated 56%, 64%, and 69% of patients achieve NEDA at months 12, 18, and 24, respectively. A significant proportion (>70%) of patients were not on disease modifying therapy (DMT) in the first 6 months. Among those on DMT, intravenous (IV) DMT was more common among the NEDA group compared to EDA. Recent DMT use was more important than initial DMT use in its ability to explain NEDA status at 18 and 24 months. Those on IV DMT had increased odds of achieving NEDA compared to those on no DMT or other DMTs.
Conclusions
A majority of POMS patients achieved NEDA at some point within 2 years of MS diagnosis, despite being treatment-naïve initially. In those on DMT, recent intravenous DMT use was associated with higher likelihood of NEDA.
Author Of 1 Presentation
P1082 - Therapeutic Response in Pediatric Neuromyelitis Optics Spectrum Disorder (ID 1820)
Abstract
Background
Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune condition which can led to significant disability. Approximately 4% of the NMOSD cases are pediatric onset. At present, there are limited studies that aim at guiding physicians in their treatment choices for NMOSD in children.
Objectives
To evaluate the effect of different disease modifying therapies (DMT) with respect to attack prevention in children with NMOSD.
Methods
Cohort study that included 12 clinical centers participating in the US Network of Pediatric MS Centers. Cases were validated for NMOSD diagnostic criteria and classified via serostatus as AQP4+, MOG+, or double-seronegative (DS). Clinical data, including demographics, attack details, type of initial DMT (rituximab, mycophenolate mofetil, azathioprine, IVIg) and neurological visit data were extracted from charts, centrally collected in a database, and analyzed. Treatment response in the three serostatus subgroups was evaluated. Effect of DMTs on annualized relapse rate (ARR) was assessed by negative binomial regression.
Results
111 pediatric patients with NMOSD were identified: 80 AQP4+, 10 MOG+, 14 double seronegative (DS), and 7 with unknown serostatus (94 females and 17 males; 48 white, 47 African American, 13 other races). Mean follow-up duration was 1.9 years (SD±2.2). About 6% of patients were treatment-naive. First-line DMTs varied by serostatus: in the AQP4+ subgroup 42% used rituximab, 16% mycophenolate mofetil, 16% azathioprine, and 8% IVIg. Among MOG+ patients, 13% received rituximab, 13% azathioprine, 13% mycophenolate, and 38% IVIg. Within the DS group, rituximab was used in 21% of cases, azathioprine in 7%, mycophenolate in 21%, and IVIg in 21%. In the unknown serogroup, 33% received rituximab, 17% azathioprine, 0% mycophenolate, and 33% IVIg. The ARR calculated in all the serogroups was 0.25 (95% CI 0.13-0.46) for rituximab, 0.73 (95% CI 0.27-2.00) for azathioprine, 0.40 (95% CI 0.18-0.89) for mycophenolate, and 0.56 (95% CI 0.26-1.20) for IVIg. In the AQP4+ subgroup, the patients started on rituximab showed an ARR of 0.25 (95% CI 0.13-0.48), those on azathioprine an ARR of 0.76 (95% CI 0.24-2.39), those on mycophenolate an ARR 0.43 (95% CI 0.17-1.07), and those on IVIg an ARR of 0.63 (95% CI 0.26-1.55).
Conclusions
This retrospective study showed that rituximab is associated with a lowered annual relapse rate in pediatric NMOSD and in particular in the AQP4+ subgroup.