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. 2023 Jul 5;23(1):258.
doi: 10.1186/s12883-023-03273-9.

The natural history of primary progressive multiple sclerosis: insights from the German NeuroTransData registry

Stefan Braune  1 , Sandra Bluemich  2 , Carola Bruns  2 , Petra Dirks  3 , Jeanette Hoffmann  2 , Yanic Heer  4 , Erwan Muros-Le Rouzic  3 , Arnfin Bergmann  5 ; NTD Study Group
Collaborators, Affiliations

The natural history of primary progressive multiple sclerosis: insights from the German NeuroTransData registry

Stefan Braune et al. BMC Neurol. .

Abstract

Background: Primary progressive multiple sclerosis (PPMS) is characterised by gradual worsening of disability from symptom onset. Knowledge about the natural course of PPMS remains limited.

Methods: PPMS patients from the German NeuroTransData (NTD) MS registry with data from 56 outpatient practices were employed for retrospective cross-sectional and longitudinal analyses. The cross-sectional analysis included a contemporary PPMS cohort with a documented visit within the last 2 years before index date (1 Jan 2021). The longitudinal analysis included a disease modifying therapy (DMT)-naïve population and focused on the evolution of expanded disability status scale (EDSS) from the first available assessment at or after diagnosis within the NTD registry to index date. Outcome measures were estimated median time from first EDSS assessment to first 24-week confirmed EDSS ≥ 4 and ≥ 7. Besides EDSS change, the proportion of patients on disability pension were described over time.

Results: The cross-sectional analysis included 481 PPMS patients (59.9% female, mean [standard deviation, SD] age 60.5 [11.5] years, mean [SD] EDSS 4.9 [2.1]). Estimated median time from first EDSS assessment after diagnosis to reach 24-week confirmed EDSS ≥ 4 for DMT-naïve patients was 6.9 years. Median time to EDSS ≥ 7 was 9.7 years for 25% of the population. Over a decade mean (SD) EDSS scores increased from 4.6 (2.1) to 5.7 (2.0); the proportion of patients on disability pension increased from 18.9% to 33.3%.

Conclusions: This study provides first insights into the German NTD real-world cohort of PPMS patients. Findings confirm the steadily deteriorating course of PPMS accompanied by increasingly limited quality of life.

Keywords: Cohort studies; Natural history; Primary progressive multiple sclerosis; Quality of life; Retrospective study.

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Conflict of interest statement

Stefan Braune received honoraria from Kassenärztliche Vereinigung Bayerns and health maintenance organisations for patient care, and from Biogen, NeuroTransData, Novartis, and Roche for consulting, project management, clinical studies, and lectures; he also received honoraria and expense compensation as a board member of NeuroTransData.

Sandra Bluemich, Carola Bruns and Jeanette Hoffmann are employees of Roche Pharma AG, Grenzach-Wyhlen, Germany.

Petra Dirks and Erwan Muros-Le Rouzic are employees of Hoffmann-La Roche Ltd, Basel, Switzerland.

Arnfin Bergmann received honoraria from NeuroTransData for project management, clinical studies, and travel expenses from Novartis and Servier; he also received honoraria and expense compensation as a board member of NeuroTransData.

Yanic Heer is an employee of PricewaterhouseCoopers (PwC), Zurich, Switzerland.

Figures

Fig. 1
Fig. 1
Box plot showing median EDSS of DMT-naïve PPMS patients per year, starting with the first EDSS visit
Fig. 2
Fig. 2
Proportion of PPMS patients without DMT on disability pension over time relative to the index 01.01.2021 (index date)
Fig. 3
Fig. 3
Time from first EDSS assessment at or after diagnosis to 24-week confirmed EDSS score of ≥ 4 (A) and ≥ 7 (B) Kaplan–Meier survival curve for all DMT-naïve PPMS patients. Patients were censored at start of any DMT. Patients were considered at-risk until the last assessment visit recorded prior to data cut-off or censored at time of death or at start of any DMT whichever occurred first
Fig. 4
Fig. 4
Time from first EDSS assessment at or after diagnosis to 24-week confirmed EDSS score of ≥ 4 (A) and ≥ 7 (B) Kaplan–Meier survival curve for subgroups of all DMT-naïve PPMS patients ≤ 55 and > 55 years at the time of PPMS diagnosis. Patients were censored at start of any DMT. Patients were considered at-risk until the last assessment visit recorded prior to data cut-off or censored at time of death or at start of any DMT whichever occurred first
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