학술논문
The natural history of primary progressive multiple sclerosis: insights from the German NeuroTransData registry
Document Type
Report
Author
Braune, Stefan; Bluemich, Sandra; Bruns, Carola; Dirks, Petra; Hoffmann, Jeanette; Heer, Yanic; Rouzic, Erwan Muros-Le; Bergmann, Arnfin; Albrecht, Walter; Bischof, Felix; Bittkau, Foroogh; Bittkau, Simon; Bohr, Kin-Arno; Borries, Bettina; Brockmeier, Bernd; Brummer, Dagmar; Bühler, Bernhard; Butz, Wolfgang; Cepek, Lukas; Claassen, Lars; Dee, Jürgen; Dieterle, Lienhard; Drees, Eckehard; Engelmann, Christoph; Ernst, Michael; Fasold, Oliver; Fischer, Johannes; Flach, Michael; Fleischer, Robert; Friedrich, Lea; Friedrich, Anke; Fritzinger, Michael; Gehring, Klaus; Gierer, Stephanie; Gierer, Stephan; Gössling, Jens; Grips, Eva; Haldenwanger, Andreas Hans-Joachim; Harth, Andreas; Hartmann, Rolf; Helm, Roland; Herbst, Heinz-Peter; Hofer, Christian; Hofmann, Werner Erwin; Hoge, Alexander; Hummel, Sibylla; Ikenberg, Benno; Israel-Willner, Heike; Jankovits, Ralf; Kallmann, Boris-Alexander; Kausch, Ulrich; Keppler, Marc; Kessler, Kirn; Kirchhöfer, Ulrike; Kirchmeier, Jürgen; Knoblich, Rupert; Knoll, Thomas; Knorn, Philipp; Köchling, Monika; Kornhuber, Anselm Wolfgang; Kramer, Bernd; Krause, Michaela; Krauss, Martin; Kubalek, Ralf; Kunz, Jürgen; Landefeld, Harald; Lange, Thomas; Lehmann-Horn, Klaus; Lippert, Esther; Lippmann, Karla; Maier-Janson, Walter; Märkl, Martin; Masri, Said; Moser, Christof; Neusch, Clemens; Niemann, Julius; Paschke, Tilmann; Peikert, Anna Sybilla; Peikert, Andreas; Peters, Henning; Pfister, Robert; Reifschneider, Gerd; Ries, Stefan; Rieth, Christoph; Roick, Holger; Roth, Gerhard Dieter; Roth, Roland; Safavi, Ali; Saur, Joachim; Schmitt-Roth, Brigitte; Scholz, Erich Franz; Schreiber, Herbert; Schreiber, Klaus; Schrey, Christoph; Schumann, Carsten; Seiler, Martin; Sigel, Karl-Otto; Sikora, Viola; Sotiriadis, Nikolaos; Spiegel, Stefanie; Städt, Detlef; Sühnel, Torsten; Tiel-Wilck, Klaus; Ulzheimer, Jochen Christoph; Unsorg, Barbara Sofie; Voith, Silvia; Wannenmacher, Achim Stephan; Weber, Hildegund; Weih, Markus; Wendtland, Bernd; Wiborg, Andreas; Wimmer, Martin; Winker, Thomas; Wontroba, Isaak; Wüstenhagen, Monika
Source
BMC Neurology. July 5, 2023, Vol. 23 Issue 1
Subject
Language
English
ISSN
1471-2377
Abstract
Author(s): Stefan Braune[sup.1] , Sandra Bluemich[sup.2] , Carola Bruns[sup.2] , Petra Dirks[sup.3] , Jeanette Hoffmann[sup.2] , Yanic Heer[sup.4] , Erwan Muros-Le Rouzic[sup.3] , Arnfin Bergmann[sup.1] , Walter Albrecht[sup.] , Felix [...]
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 [greater than or equal to] 4 and [greater than or equal to] 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 [greater than or equal to] 4 for DMT-naïve patients was 6.9 years. Median time to EDSS [greater than or equal to] 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: Primary progressive multiple sclerosis, Natural history, Retrospective study, Cohort studies, Quality of life
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 [greater than or equal to] 4 and [greater than or equal to] 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 [greater than or equal to] 4 for DMT-naïve patients was 6.9 years. Median time to EDSS [greater than or equal to] 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: Primary progressive multiple sclerosis, Natural history, Retrospective study, Cohort studies, Quality of life