Therapeutic status quo in patients with relapsing-remitting multiple sclerosis: a sign of poor self-perception of their clinical status?

Background Status quo (SQ) bias is defined as patient´s tendency to continue taking a previously selected but inferior therapeutic option. Objective To assess the presence of SQ bias and its associated factors in patients with relapsing-remitting multiple sclerosis (RRMS). Methods A multicenter, non-interventional study involving 211 patients with RRMS was conducted. Participants answered questions regarding risk preferences and management of simulated MS case-scenarios. The SymptoMScreen (SMSS) questionnaire was used to assess the perception of severity from the patients´ perspective. SQ bias was defined as patients’ preference to maintain the current treatment despite evidence of disease activity. Mixed linear models adjusting for clustering assessed the association of candidate predictors with the outcome of interest. Results The mean age (SD) was 39.1 (9.5) years and 70.6% were women. SQ bias was observed in 74.4% (n=161) participants. Univariate analysis showed that SMSS score was associated with SQ bias (OR 1.04; 95% CI 1.01-1.07). Mixed linear regression models suggest that for every point increase in SMSS, there was a 4% increase in the likelihood of SQ bias (β 0.04; 95%CI 0.015-0.06; p<0.002). Among the different symptomatic dimensions included in the SMSS, only vision impairment (β 0.32; 95%CI 0.05-0.50) and depression (β 0.29; 95%CI 0.006-0.58) remained associated with SQ bias in the multivariate analysis. There was no association between participants’ risk preferences and SQ bias. Conclusions Unwillingness to pursue treatments that are more effective is a common phenomenon affecting over 7 out of 10 patients with RRMS. This phenomenon appears to be driven by patients’ negative self-perception of their clinical status

Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study.

Outcome measures traditionally used in spinal muscular atrophy (SMA) clinical trials are inadequate to assess the full range of disease severity. The aim of this study was to assess the psychometric properties of a set of existing questionnaires and new items, gathering information on the impact of SMA from the patient and caregiver perspectives. This was a multicenter, prospective, noninterventional study including patients with a confirmed diagnosis of 5q-autosomal-recessive SMA aged 8 years and above, or their parents (if aged between 2 and 8 years). The set of outcome measurements included the SMA Independence Scale (SMAIS) patient and caregiver versions, the Neuro-QoL Fatigue Computer Adaptive Test (CAT), the Neuro-QoL Pain Short Form-Pediatric Pain, the PROMIS adult Pain Interference CAT, and new items developed by Fundación Atrofia Muscular España: perceived fatigability, breathing and voice, sleep and rest, and vulnerability. Reliability, construct validity, discriminant validity, and sensitivity to change (4 months from baseline) were measured. A total of 113 patients were included (59.3% 2-17 years old, 59.3% male, and 50.4% with SMA type II). Patients required moderate assistance [mean patient and caregiver SMAIS (SD) scores were 31.1 (12.8) and 7.6 (11.1), respectively]. Perceived fatigability was the most impacted domain, followed by vulnerability. Cronbach's alpha coefficient for perceived fatigability, breathing and voice, and vulnerability total scores were 0.92, 0.88, and 0.85, respectively. The exploratory factor analysis identified the main factors considered in the design, except in the sleep and rest domain. All questionnaires were able to discriminate between the Clinical Global Impression-Severity scores and SMA types. Sensitivity to change was only found for the SMAIS caregiver version and vulnerability items. This set of outcome measures showed adequate reliability, construct validity, and discriminant validity and may constitute a valuable option to measure symptom severity in patients with SMA