Abstract
Background and purpose: Treatment options in primary progressive multiple sclerosis (PPMS) are scarce and, with the exception of ocrelizumab, anti-inflammatory agents have failed to show efficacy in ameliorating disability progression. The aim of this study was to investigate a potential effect of anti-inflammatory disease-modifying treatment on disability outcomes in PPMS. Methods: Using MSBase, a large, international, observational database, we identified patients with PPMS who were either never treated or treated with a disease-modifying agent. Propensity score matching was used to select subpopulations with similar baseline characteristics. Expanded Disability Status Scale (EDSS) outcomes were compared with an intention-to-treat and an as-treated approach in paired, pairwise-censored analyses. Results: Of the 1284 included patients, 533 were matched (treated, n = 195; untreated n = 338). Median on-study pairwise-censored follow-up was 3.4 years (quartiles 1.2–5.5). No difference in the hazard of experiencing 3-month confirmed EDSS progression events was observed between the groups [hazard ratio (HR), 1.0; 95% confidence interval (CI), 0.6–1.7, P = 0.87]. We did not find significant differences in the hazards of confirmed EDSS improvement (HR, 1.0; 95% CI, 0.6–1.6, P = 0.91) or reaching a confirmed EDSS step ≥7 (HR, 1.1; 95% CI, 0.7–1.6, P = 0.69). Conclusion: Our pooled analysis of disease-modifying agents suggests that these therapies have no substantial effect on short- to medium-term disability outcomes in PPMS.
| Original language | English |
|---|---|
| Pages (from-to) | 363-370 |
| Number of pages | 8 |
| Journal | European Journal of Neurology |
| Volume | 26 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 1 Feb 2019 |
Keywords
- clinical outcomes
- immunomodulation
- multiple sclerosis
- observational study
- primary progressive
Cite this
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Anti-inflammatory disease-modifying treatment and disability progression in primary progressive multiple sclerosis : a cohort study. / Lorscheider, J.; Kuhle, J.; Izquierdo, G.; Lugaresi, A.; Havrdova, E.; Horakova, D.; Hupperts, R.; Duquette, P.; Girard, M.; Prat, A.; Grand'Maison, F.; Grammond, P.; Sola, P.; Ferraro, D.; Trojano, M.; Ramo-Tello, C.; Lechner-Scott, J.; Pucci, E.; Solaro, C.; Slee, M.; Van Pesch, V.; Sanchez Menoyo, J. L.; van der Walt, A.; Butzkueven, H.; Kappos, L.; Kalincik, T.; on behalf of the MSBase Study Group.
In: European Journal of Neurology, Vol. 26, No. 2, 01.02.2019, p. 363-370.Research output: Contribution to journal › Article › Research › peer-review
TY - JOUR
T1 - Anti-inflammatory disease-modifying treatment and disability progression in primary progressive multiple sclerosis
T2 - a cohort study
AU - Lorscheider, J.
AU - Kuhle, J.
AU - Izquierdo, G.
AU - Lugaresi, A.
AU - Havrdova, E.
AU - Horakova, D.
AU - Hupperts, R.
AU - Duquette, P.
AU - Girard, M.
AU - Prat, A.
AU - Grand'Maison, F.
AU - Grammond, P.
AU - Sola, P.
AU - Ferraro, D.
AU - Trojano, M.
AU - Ramo-Tello, C.
AU - Lechner-Scott, J.
AU - Pucci, E.
AU - Solaro, C.
AU - Slee, M.
AU - Van Pesch, V.
AU - Sanchez Menoyo, J. L.
AU - van der Walt, A.
AU - Butzkueven, H.
AU - Kappos, L.
AU - Kalincik, T.
AU - on behalf of the MSBase Study Group
PY - 2019/2/1
Y1 - 2019/2/1
N2 - Background and purpose: Treatment options in primary progressive multiple sclerosis (PPMS) are scarce and, with the exception of ocrelizumab, anti-inflammatory agents have failed to show efficacy in ameliorating disability progression. The aim of this study was to investigate a potential effect of anti-inflammatory disease-modifying treatment on disability outcomes in PPMS. Methods: Using MSBase, a large, international, observational database, we identified patients with PPMS who were either never treated or treated with a disease-modifying agent. Propensity score matching was used to select subpopulations with similar baseline characteristics. Expanded Disability Status Scale (EDSS) outcomes were compared with an intention-to-treat and an as-treated approach in paired, pairwise-censored analyses. Results: Of the 1284 included patients, 533 were matched (treated, n = 195; untreated n = 338). Median on-study pairwise-censored follow-up was 3.4 years (quartiles 1.2–5.5). No difference in the hazard of experiencing 3-month confirmed EDSS progression events was observed between the groups [hazard ratio (HR), 1.0; 95% confidence interval (CI), 0.6–1.7, P = 0.87]. We did not find significant differences in the hazards of confirmed EDSS improvement (HR, 1.0; 95% CI, 0.6–1.6, P = 0.91) or reaching a confirmed EDSS step ≥7 (HR, 1.1; 95% CI, 0.7–1.6, P = 0.69). Conclusion: Our pooled analysis of disease-modifying agents suggests that these therapies have no substantial effect on short- to medium-term disability outcomes in PPMS.
AB - Background and purpose: Treatment options in primary progressive multiple sclerosis (PPMS) are scarce and, with the exception of ocrelizumab, anti-inflammatory agents have failed to show efficacy in ameliorating disability progression. The aim of this study was to investigate a potential effect of anti-inflammatory disease-modifying treatment on disability outcomes in PPMS. Methods: Using MSBase, a large, international, observational database, we identified patients with PPMS who were either never treated or treated with a disease-modifying agent. Propensity score matching was used to select subpopulations with similar baseline characteristics. Expanded Disability Status Scale (EDSS) outcomes were compared with an intention-to-treat and an as-treated approach in paired, pairwise-censored analyses. Results: Of the 1284 included patients, 533 were matched (treated, n = 195; untreated n = 338). Median on-study pairwise-censored follow-up was 3.4 years (quartiles 1.2–5.5). No difference in the hazard of experiencing 3-month confirmed EDSS progression events was observed between the groups [hazard ratio (HR), 1.0; 95% confidence interval (CI), 0.6–1.7, P = 0.87]. We did not find significant differences in the hazards of confirmed EDSS improvement (HR, 1.0; 95% CI, 0.6–1.6, P = 0.91) or reaching a confirmed EDSS step ≥7 (HR, 1.1; 95% CI, 0.7–1.6, P = 0.69). Conclusion: Our pooled analysis of disease-modifying agents suggests that these therapies have no substantial effect on short- to medium-term disability outcomes in PPMS.
KW - clinical outcomes
KW - immunomodulation
KW - multiple sclerosis
KW - observational study
KW - primary progressive
UR - http://www.scopus.com/inward/record.url?scp=85055939122&partnerID=8YFLogxK
U2 - 10.1111/ene.13824
DO - 10.1111/ene.13824
M3 - Article
VL - 26
SP - 363
EP - 370
JO - European Journal of Neurology
JF - European Journal of Neurology
SN - 1351-5101
IS - 2
ER -