Background

The TREAT-MS study (Paul-Ehrlich-Institut registry: 281) is assessing real-world effectiveness of alemtuzumab in relapsing-remitting multiple sclerosis (RRMS) patients in Germany.

Objectives

Subgroup analysis of TREAT-MS to investigate the effect of the number of prior disease-modifying therapies (DMTs) on the efficacy and safety of alemtuzumab 1 year after the 2nd treatment cycle with alemtuzumab.

Methods

TREAT-MS is a 5-year, observational, longitudinal, noninterventional, open-label, multicenter study of alemtuzumab-treated patients.

Results

As of February 2020, 883 patients were enrolled and 571 patients were observed for 2 years after treatment initiation. Of these, 565 (98.9%) patients entered the first treatment period, 538 (94.2%) patients entered two treatment periods. 13.3% of patients were treatment-naive at baseline, 83.7% had received prior DMTs, and in 3.0% pretreatment was unknown. The interim analysis focussed on the data of patients 1 year after the 2nd treatment phase and was differentiated by the number of previously received DMTs. Patients with 0, 1, 2, and ≥3 pretreatments had a mean number of 1.6, 1.7, 1.4, and 1.8 relapses, respectively, during the last 12 months before alemtuzumab treatment. After alemtuzumab treatment initiation, annualized relapse rate (ARR) in patients with 0, 1, 2, ≥3 pretreatments reached levels of 0.13, 0.18, 0.25, and 0.24. ARR after alemtuzumab initiation was significantly higher in patients who received 2 or ≥3 prior DMTs when compared with treatment-naive patients.

The majority of patients were relapse-free 1 year after the 2nd course of alemtuzumab. 82.2%, 77.0%, 66.5%, 73.1% of the patients with 0, 1, 2, ≥3 pretreatments, respectively, had no relapses during the observational period. The proportion of relapse-free patients 1 year after the second alemtuzumab course was highest in treatment-naive patients.

Mean expanded disability status scale (EDSS) score at baseline was 2.2, 2.4, 2.6, and 3.6 for those who received 0, 1, 2, and ≥3 prior DMTs, respectively, and changed by a mean EDSS value of –0.5, –0.2, –0.2, and –0.5 one year after the 2nd treatment phase.

Adverse events were reported for 64.5%, 66.9%, 66.9%, 73.6% of the patients treated with 0, 1, 2, and ≥3 DMTs. No new safety signals were observed.

Conclusions

The interim subgroup analysis of patients 1 year after the 2nd treatment cycle with alemtuzumab has shown that relapse rates were reduced, and EDSS scores were stable regardless of the number of prior DMTs received. These data confirm registration trial findings (CARE-MS I and II) in the real-world setting in patients with longer disease duration and varying treatment history.

Background

For the case of multiple sclerosis, research on gender differences from a health economic perspective has not received much attention. However, cost-of-illness analyses provide valuable information about the diverse impact of the disease and thus help decision-makers to allocate scarce resources.

Objectives

The aim of this study was to describe resource use and associated societal costs from a gender perspective. In particular, the aim was to determine how resource utilization potentially differs in certain cost components between men and women.

Methods

Data were extracted from two prospective, non-interventional, observational multicentre studies in Germany. Information on (health) resource use was obtained from all patients on a quarterly basis using a validated questionnaire. Cost analyses were conducted from the societal perspective, including all direct (healthcare-related) and indirect (work-related) costs, regardless of who ultimately pays them. Costs for men and women were analysed within subgroups of two-year clinical disease activity. Gender-related differences were analysed by multivariate negative binomial regression models (mean quarterly costs) and binary logistic regression models (patients using resources).

Results

In total, 2095 patients (72.9% females) presented a median EDSS of 2 (IQR 1-3.5) and disease duration of 7.55 ±6.12 years (p>0.05 for gender-related differences). Women and men did not statistically differ in total quarterly costs (2329 Euro (€) ±2570€ vs 2361€ ±2612€). For both sexes, costs were higher with advancing disability and indirect costs were the main societal cost driver. Regarding healthcare-related resources, women incurred higher costs for outpatient consultations, complementary medicine, medical consumables and informal care. Among indirect costs, we found higher costs for men for presenteeism and higher costs for women for disability pension (all p<0.05).

Conclusions

Multiple sclerosis poses a significant economic burden on patients, families and society. While the total economic burden did not differ between males and females, we found gender differences in specific cost items that are similar to those in the wider non-MS population. Furthermore, indirect costs were highly dependent on the cost categories included and the way in which they were valued.