ABSTRACT
BACKGROUND: The treatment landscape for neuromyelitis optica spectrum disorder (NMOSD) has changed in recent years with the approval of therapies with different efficacy, safety and administration profiles. OBJECTIVE: The aim of this study was to assess neurologists' preferences for different NMOSD treatment attributes using conjoint analysis (CA). METHODS: We conducted an online, non-interventional, cross-sectional study in collaboration with the Spanish Society of Neurology. Our CA assessed five drugs' attributes: prevention of relapse, prevention of disability accumulation, safety risk, management during pregnancy, and route and frequency of administration. Participants were presented with eight hypothetical treatment scenarios to rank based on their preferences from the most preferred to the least. An ordinary least squares method was selected to estimate weighted preferences. RESULTS: A total of 104 neurologists were included. Mean age (standard deviation-SD) was 37.7 (10.3) years, 52.9 % were male, and median time (interquartile range) of experience managing NMOSD was 5.0 (2.9, 10.8) years. Neurologists placed the greatest importance on efficacy attributes, time to relapse (44.1 %) being the most important, followed by preventing disability accumulation (36.8 %). In contrast, route and frequency of administration (4.6 %) was the least important characteristic. Participants who prioritised efficacy attributes felt more comfortable in decision-making, had fewer past experiences of care-related regret and a lower attitude to risk taking than their counterparts. CONCLUSION: Neurologists' treatment preferences in NMOSD were mainly driven by efficacy attributes. These results may be useful to design policy decisions and treatment guidelines for this condition.
ABSTRACT
Introduction and objective: Limited information is available on how neurologists make therapeutic decisions in neuromyelitis optica spectrum disorder (NMOSD), especially when new treatments with different mechanisms of action, administration, and safety profile are being approved. Decision-making can be complex under this uncertainty and may lead to therapeutic inertia (TI), which refers to lack of treatment initiation or intensification when therapeutic goals are not met. The study aim was to assess neurologists' TI in NMOSD. Methods: An online, cross-sectional study was conducted in collaboration with the Spanish Society of Neurology. Neurologists answered a survey composed of demographic characteristics, professional background, and behavioral traits. TI was defined as the lack of initiation or intensification with high-efficacy treatments when there is evidence of disease activity and was assessed through five NMOSD aquaporin-4 positive (AQP4+) simulated case scenarios. A multivariate logistic regression analysis was used to determine the association between neurologists' characteristics and TI. Results: A total of 78 neurologists were included (median interquartile range [IQR] age: 36.0 [29.0-46.0] years, 55.1% male, median [IQR] experience managing demyelinating conditions was 5.2 [3.0-11.1] years). The majority of participants were general neurologists (59.0%) attending a median (IQR) of 5.0 NMOSD patients (3.0-12.0) annually. Thirty participants (38.5%) were classified as having TI. Working in a low complexity hospital and giving high importance to patient's tolerability/safety when choosing a treatment were predictors of TI. Conclusion: TI is a common phenomenon among neurologists managing NMOSD AQP4+. Identifying TI and implementing specific intervention strategies may be critical to improving therapeutic decisions and patient care.
ABSTRACT
BACKGROUND AND PURPOSE: disease-modifying treatments (DMT) for Multiple Sclerosis (MS) have expanded in recent years making the shared-decision process challenging. Moreover, no head-to-head studies are available within the first-line options. Our aim is to compare therapeutic persistence within first-line DMT: teriflunomide (TER), dimethyl fumarate (DMF), and injectable drugs (INJ) in a real-world setting. METHODS: Retrospective observational study analyzing diagnosed with Relapsing-Remitting Multiple Sclerosis (RRMS) who started DMT between January 2015 and April 2022 (TER=117, DMF=117, INJ=123). Clinical, radiological, and demographic variables were collected. The primary outcome was the median time to discontinuation of any DMT. Dropout was defined as discontinuation for 6 months for any reason. RESULTS: Of the total of 357 patients, 155 withdraw with a median time-to-discontinuation of 1.427 years (IQR 2.410). The discontinuation rate was higher in the injectable group, 49.6%; compared to teriflunomide 40.2%, and dimethyl fumarate 39.8% (p = 0.201). The most frequent reason of discontinuation differs within groups (lack of efficacy in TER, 63.8%, and adverse effects in DMF and INJ (40.4% and 40.9% respectively). No difference in persistence was observed between DMT (p = 0.30). After 2018 there has been a tendency to treat in a quick and early manner (lower EDSS; relapse rate and number of naïve patients), statistically significant for TER (p = 0.005, p = 0.010, and p = 0.045). CONCLUSIONS: Our study demonstrated no differences in persistence between the actual first-line DMT in a real-world setting, although a trend to favor oral-DMT was seen. Reasons for discontinuation differs within groups.
ABSTRACT
In the last years, therapeutic decisions in multiple sclerosis (MS) have become challenging due to expanded options with different treatment profiles attending to efficacy, safety, and route and frequency of administration. Moreover, patients with multiple sclerosis (PwMS) increasingly wish to be involved in their therapeutic decision process. Therefore, a new, patient-centric shared decision model (SDM), is gaining relevance. However, validated scales oriented to assess the quality of the process itself are lacking. The AGA-25 scale is a fit-for-purpose 25-item scale based on two validated scales in MS (Treatment Satisfaction Questionnaire for Medication (TSQM) and Decisional Conflict Scale (DCS)). The aim of this work is to develop and validate the AGAS-25 in Spanish. Two hundred and three PwMS (aged 17 to 67; 155 [76.4%] females) undergoing stable disease modifying treatment in the last 6 months were consecutively recruited. The Principal Component Analysis suggested a four-factor structure for the 25-item version of the questionnaire: 1) satisfaction with the SDM process 2) adverse events with the DMT, 3) convenience of the chosen-DMT and 4) information reliability. The internal consistency of the measurement was adequate (Cronbach's alpha = 0.88). Our results support the use of the AGAS-25 scale to assist SDM in Spanish-speaking PwMS.
Subject(s)
Multiple Sclerosis , Female , Humans , Male , Multiple Sclerosis/drug therapy , Patients , Psychometrics , Reproducibility of Results , Surveys and QuestionnairesABSTRACT
INTRODUCCIÓN: La efectividad y seguridad del fingolimod en pacientes con esclerosis múltiple remitente recurrente (EMRR) se demostró en ensayos clínicos. Sin embargo, por las limitaciones de éstos, es importante saber cómo se comporta en condiciones de práctica clínica habitual. Así, el objetivo de este estudio es evaluar la efectividad y seguridad del fingolimod después de 12 meses de uso en la práctica clínica en Galicia. PACIENTES Y MÉTODOS: Estudio retrospectivo y multicéntrico (n = 8) de pacientes con EMRR y tratados con una o más dosis de fingolimod, 0,5 mg/día. Se evaluó la efectividad -tasa anualizada de brotes (TAB), cambio en la puntuación de la escala expandida del estado de discapacidad (EDSS), porcentaje de pacientes libres de brotes, libres de progresión de discapacidad y libres de actividad en resonancia- para el total de pacientes y según tratamiento previo. Se evaluó la seguridad a partir del porcentaje de pacientes que discontinuaron y que presentaron efectos adversos. RESULTADOS: Después de 12 meses de uso, el fingolimod redujo un 87% la TAB (de 1,7 a 0,23; p < 0,0001) y, en consecuencia, un 81% de pacientes estuvo libre de brotes. La puntuación de la EDSS disminuyó un 9%. Un 91% de pacientes estuvo libre de progresión de discapacidad y un 72%, libre de actividad en resonancia. En el 43% de los pacientes no se evidenciaron signos de la actividad de la enfermedad. La mayoría de los beneficios del fingolimod difirieron según el tratamiento previo. Alrededor de un tercio de los pacientes comunicaron efectos adversos, pero sólo el 2% discontinuó debido a ellos. CONCLUSIONES: La mayoría de los resultados de efectividad de los ensayos clínicos del fingolimod se observa durante los 12 primeros meses de tratamiento en la práctica clínica. Se observó un mejor perfil de seguridad al comunicado en los ensayos clínicos
INTRODUCTION: The effectiveness and safety of fingolimod in patients with relapsing-remitting multiple sclerosis (RRMS) have been proven in clinical trials. Yet, due to their limitations, it is important to know how it behaves under everyday clinical practice conditions. Hence, the aim of this study is to evaluate the effectiveness and safety of fingolimod after 12 months' usage in clinical practice in Galicia. PATIENTS AND METHODS: We conducted a retrospective, multi-centre study (n = 8) of patients with RRMS who were treated with one or more doses of fingolimod, 0.5 mg/day. Effectiveness was assessed -annualised relapse rate (ARR), changes in the score on the Expanded Disability Status Scale (EDSS), percentage of patients free from relapses, free from progression of disability and free from activity in resonance- for the total number of patients and according to previous treatment. Safety was assessed based on the percentage of patients who withdrew and presented adverse side effects. RESULTS: After 12 months' use, fingolimod reduced the ARR by 87% (1.7 to 0.23; p < 0.0001) and, consequently, 81% of patients were free from relapses. The score was reduced by 9%. In all, 91% of patients were free from progression of disability and 72% were free from resonance activity. No signs of disease activity were found in 43% of the PATIENTS: Most of the benefits of fingolimod differed depending on previous treatment. About a third of the patients reported adverse side effects, but only 2% of them withdrew for this reason. CONCLUSIONS: In clinical practice, most of the results on the effectiveness of the clinical trials conducted with fingolimod were observed during the first 12 months of treatment. A better safety profile was observed than that reported in the clinical trials (AU)
