Background

Background: In multiple sclerosis (MS), randomized controlled trials (RCT) have provided relevant information about the efficacy and safety in ideal scenarios. While RCT are powerful tools for developing scientific evidence based on their high internal validity, there is always uncertainty about the generalizability, especially since the populations enrolled in such studies may differ in significant ways from those seen in clinical practice.

Objectives

Objective: to describe the frequency and clinical profile of MS patients under disease modifying treatment in Argentina that would have not fulfilled inclusion criteria in RCT.

Methods

Methods: MS patients included in the Argentinean MS and NMOSD registry (RelevarEM, NCT 03375177) were analyzed. RelevarEM is a longitudinal, strictly observational MS and NMOSD registry in Argentina. From May 2018 to March 2020, the centers and principal investigators were contacted and incorporated into the Registry. All patients with definite MS and receiving DMT at 31 December 2019 were screened, those with EDSS >6, phenotypes secondary progressive (SP) and primary progressive (PP)(with other DMT than ocrelizumab) and age <18 and >55 years old were included in the analysis.

Results

Results: A total of 1782 patients with MS receiving DMT were screened, of whom 465 (26%)would not have been included in a pivotal trial. From the 465,218 had and EDSS >6, 67 had phenotype SP and 19 PP; 292 were patients with <18 and >55 years of age (2 under 18 years old). Most prescribed DMT among patients with EDSS >6 was fingolimod (31%), among age >55 was beta interferon (35%), phenotype SP fingolimod (30%) and PP fingolimod and glatiramer acetate (each 26%).

Conclusions

Conclusion: in our registry, we found a significant number of MS patients who would have not been included in pivotal trials, receiving DMT. Real life evidence is highly relevant to assess effectiveness as well as safety of DMT in this subset of patients.

Background

Adherence to prescribed treatment in chronic disease is a critical factor for a successful therapeutic response. However, in conditions like multiple sclerosis (MS), where the treatment is mainly preventive, it might be inadequate. The reasons for poor adherence may be related to multiple factors.

Objectives

The objective is to evaluate adherence to Multiple Sclerosis treatment and identify predictors that could affect it.

Methods

This is a cross-sectional study consisting of a cohort of MS patients conducted at a National Medical Care Program: PAMI (Programa Atención Médica Integral) in Argentina, during January 1st and October 1st 2017 available in the database of drug dispensing. Variables related to disease, patient, health system and treatment were evaluated from a brief telephone survey. The medication possession ratio (MPR) was used to estimate adherence, MPR<80% defined nonadherence. The association between predictor variables and adherence were assessed with a logistic regression model.

Results

Out of the 648 patients included in the database, a total of 360 (55.5%) surveys were conducted. 311/360 (86.4%) stated that they were receiving treatment for MS at the moment of the survey. Mean age was 55.3 (SD 12), 216 (60%) female. The optimal adherence to treatment was 45.3%.

Median disease duration was 14.5 years (IQR 13). During last year, 117 (32.5%) had relapses. Fatigue was moderate to severe in 297 (82.5%) of patients, 201 (56%) patients required assistance to walk. 107 (33.6%) of patients forgot to take the medication and 103 (29%) presented symptoms suggestive of depression.The mean of neurological controls were 3.8 (SD 2.6) per year. The median delay to authorization of medication was 2.3 (IQR 10) weeks.

198 (63.7%) received injectables therapies, most commonly used were interferons. In the multivariate model, we only found an association between adherence and the oral route of administration (OR 1.96 CI95% 1.20-3.20, p= 0.006). In a secondary post hoc analysis we found that the predictors of receiving oral drugs were higher educational level (OR 2.86 CI95% 1.06-7.66) and the presence of associated comorbidities (OR 1.59, CI95% 0.98-2.57).

Conclusions

Treatment adherence has been suboptimal. The use of injectable drugs was associated with nonadherence to treatment. No adherence predictors associated with the patient, disease and health system were found.

Background

Several retrospective studies have demonstrated the clinical benefits of immunosuppressive therapies (IST) such as azathioprine (AZA), mycophenolate mofetil (MMF) and rituximab (RTX) for reducing relapse rates in neuromyelitis optica spectrum disorders (NMOSD) patients. However, there is considerable uncertainty regarding the relative benefits and harms associated with each of these IST in real world clinical practice and current data describing the strategies are limited

Objectives

The objective of this study was to describe the incidence of relapses in patients with NMOSD under IST included in the Argentinean MS and NMOSD registry (RelevarEM, NCT 03375177).

Methods

We conducted a retrospective cohort study from RelevarEM. RelevarEM is a longitudinal, strictly observational MS and NMOSD registry in Argentina. From May 2018 to June 2020, the centers and principal investigators were contacted, and patients were incorporated into the Registry. NMOSD patients were defined based on the 2015 International Consensus Diagnostic Criteria for NMOSD. Relapses during the study period, demographics and radiological (e.g. new/enlarging and/or enhancing-contrast MRI lesions) data were collected. Only patients under IST were included in the analysis. Patients contributed person-years of follow-up for the study period. Incidence rates and 95% CI were calculated. Thus, global and associated with each IST incidence density rate of relapses was estimated.

Results

We included a total of 132 (77% women) NMOSD patients with a median age at diagnosis of 36 years (27-47) and a disease duration of 6 years (4-10). Aquaporin-4 antibody was positive in 54.8%. At the time of entering the registry, 39.4% were treated with RTX, 33.3% with AZA, 3.6% MMF. The global incidence density rate of relapse was 0.032/person-year (CI95% 0,021-0,048), for RTX 0.051 (CI95% 0,024-0,1) and for AZA 0,031 (CI95% 0,016-0,06). There were no relapses in the group of MMF during this period of time.

Conclusions

This study showed a low incidence density rate of relapses in NMOSD patients under IST during this study period. Further studies will help expand our initial findings, hopefully leading to improve treatment options for NMOSD patients.