ABSTRACT
Background: Multiple sclerosis (MS) is a chronic disabling disease associated with negative effects on quality of life (QoL), including physical and mental health. The objective of this investigation was to evaluate the change in QoL for patients with highly active relapsing MS at 1 year after initiating treatment with cladribine tablets (CladT), by assessing changes in the physical and mental health composite scores of Multiple Sclerosis Quality of Life-54 (MSQoL-54). Material(s) and Method(s): In CLARIFY-MS (NCT03369665), patients with highly active relapsing MS were assigned to receive CladT 3.5 mg/kg cumulative dose over 2 years. Patients were recruited as per the EU label. Results in this interim analysis, conducted prior to the second year of treatment, were assessed using a mixed-effects linear model. Analyses were also conducted for cohorts separated by treatment naïve/prior disease-modifying therapy (DMT), and MSQoL reporting performed before/after the start of the COVID-19 pandemic, as defined as the first reported fatality within each country. Result(s): Of the 482 patients treated with CladT, 70.1% were female and the mean age was 37.4 years. Of the 426 patients who provided MSQoL-54 data, statistically significant (p<0.0001) improvements from Baseline to Month 12 were observed for physical and mental health composite scores with estimated changes of 4.51 (95% confidence interval [CI] 3.24–5.77) and 4.53 (95% CI 3.00–6.05), respectively. Similar trends were apparent for treatment naïve (n=121) and prior DMT (n=305) cohorts. There was no indication that the start of the COVID-19 pandemic had an impact on MSQoL-54 reporting. Regarding safety, 322 patients (66.8%) experienced at least one treatment-emergent adverse event, most commonly headache (16%), nasopharyngitis (9%), and lymphopenia (9%). The majority of observed post-baseline lymphopenia events were grade 1–2;fewer patients reported grade 3 lymphopenia, no grade 4 lymphopenia was observed. Conclusion(s): With only half a therapeutic dose of CladT, this interim analysis demonstrates a statistically significant improvement in the physical and mental health composite scores of MSQoL-54 at 1 year. No new safety concerns were found in this 1-year interim analysis, with no new severe or opportunistic infections that could have an impact on the established benefit:risk profile of CladT in MS.
ABSTRACT
Background: ConCure-SM is a mixed-methods research project for developing and testing an advance care planning (ACP) intervention for people with progressive multiple sclerosis (pwPMS) in Italy. It consists of a booklet to be used during the ACP conversation (the focus of this presentation) and a training program for neurologists and other MS healthcare professionals (HPs). Aims: To assess the acceptability and comprehensibility of the ConCure-SM booklet. Methods: An inter-disciplinary panel translated and adapted to the Italian legislation and to the MS context a booklet developed by the National ACP programme for New Zealand. The booklet was then probed via individual cognitive interviews with pwPMS and caregivers, and a focus group meeting (FGM) with MS HPs. Two weeks before the interview/ FGM, held on digital platform due to the COVID-19 pandemic, participants received the booklet and were invited to browse it. Results: Between September-January 2021 we conducted 13 interviews which lasted between 36 and 80 minutes. Participants were 10 pwPMS and 3 caregivers (2 spouse, one daughter);8 were men, median age was 54 years. Data saturation was achieved after 11 interviews were analyzed. Twelve HPs participated in the FGM (7 neurologists, 3 psychologists, one nurse and one physiotherapist), which lasted 1.45 min. Thematic analysis (performed by LDP, SV, and LG) identified 4 overarching themes: comprehensibility and clarity;content acceptability and emotional impact;images and layout;suggestions for improvement. Interviews revealed that the booklet was useful and informative, though pwPMS found it emotionally taxing. The FGM was well participated;few experiential data on ACP emerged, lack of training and time constraint emerging as major reasons. Conclusions: Cognitive debriefing was key to refine the ConCure-SM booklet. Interview and FGM results corroborated use of the booklet within the ACP conversation, and the challenge of appraisal as a standalone tool.