Nallamothu BK, Hayward RA, Bates ER. Beyond the randomized clinical trial: the role of effectiveness studies in evaluating cardiovascular therapies. Circulation. 2008;118:1294–303.
Article
PubMed
Google Scholar
Milnes F, Bergvall N, Olson M, Capkun-Niggli G, Bonzani I, Makin C. Use of real-world data to assess outcomes in patients with multiple sclerosis in a real-life clinical setting. 10th Annual Meeting of Health Technology Assessment international (HTAi). Seoul: Poster P1082; 2013.
Association of the British Pharmaceutical Industry. Demonstrating value with real world data: a practical guide. 2011. http://www.abpi.org.uk/our-work/library/guidelines/Pages/real-world-data.aspx. Accessed 25 February 2016.
Garrison Jr LP, Neumann PJ, Erickson P, Marshall D, Mullins CD. Using real-world data for coverage and payment decisions: the ISPOR Real-World Data Task Force report. Value Health. 2007;10:326–35.
Article
PubMed
Google Scholar
Ziemssen T, Kempcke R, Eulitz M, Grossmann L, Suhrbier A, Thomas K, Schultheiss T. Multiple sclerosis documentation system (MSDS): moving from documentation to management of MS patients. J Neural Transm (Vienna). 2013;120 Suppl 1:S61–6.
Article
Google Scholar
Agashivala N, Wu N, Abouzaid S, Wu Y, Kim E, Boulanger L, Brandes DW. Compliance to fingolimod and other disease modifying treatments in multiple sclerosis patients, a retrospective cohort study. BMC Neurol. 2013;13:138.
Article
PubMed
PubMed Central
Google Scholar
Bergvall N, Lahoz R, Reynolds T, Korn JR. Healthcare resource use and relapses with fingolimod versus natalizumab for treating multiple sclerosis: a retrospective US claims database analysis. Curr Med Res Opin. 2014;30:1461–71.
Article
CAS
PubMed
Google Scholar
Bergvall N, Petrilla AA, Karkare SU, Lahoz R, Agashivala N, Pradhan A, Capkun G, Makin C, McGuiness CB, Korn JR. Persistence with and adherence to fingolimod compared with other disease-modifying therapies for the treatment of multiple sclerosis: a retrospective US claims database analysis. J Med Econ. 2014;17:696–707.
Article
PubMed
Google Scholar
Butzkueven H, Kappos L, Pellegrini F, Trojano M, Wiendl H, Patel RN, Zhang A, Hotermans C, Belachew S; TYSABRI Observational Program (TOP) Investigators. Efficacy and safety of natalizumab in multiple sclerosis: interim observational programme results. J Neurol Neurosurg Psychiatry. 2014;85:1190–7.
Article
PubMed
PubMed Central
Google Scholar
A dose-blind, multicenter, extension study to determine the long-term safety and efficacy of two doses of BG00012 monotherapy in subjects with relapsing-remitting multiple sclerosis. ClinicalTrials.gov Identifier: NCT00835770. 2015. https://clinicaltrials.gov/show/NCT00835770. Accessed 25 February 2016.
Confavreux C, Vukusic S, Moreau T, Adeleine P. Relapses and progression of disability in multiple sclerosis. N Engl J Med. 2000;343:1430–8.
Article
CAS
PubMed
Google Scholar
Davis MF, Sriram S, Bush WS, Denny JC, Haines JL. Automated extraction of clinical traits of multiple sclerosis in electronic medical records. J Am Med Inform Assoc. 2013;20:e334–40.
Article
PubMed
PubMed Central
Google Scholar
Jokubaitis VG, Li V, Kalincik T, Izquierdo G, Hodgkinson S, Alroughani R, Lechner-Scott J, Lugaresi A, Duquette P, Girard M, et al. Fingolimod after natalizumab and the risk of short-term relapse. Neurology. 2014;82:1204–11.
Article
CAS
PubMed
PubMed Central
Google Scholar
Kalincik T, Horakova D, Spelman T, Jokubaitis V, Trojano M, Lugaresi A, Izquierdo G, Rozsa C, Grammond P, Alroughani R, et al. Switch to natalizumab versus fingolimod in active relapsing-remitting multiple sclerosis. Ann Neurol. 2015;77:425–35.
Article
CAS
PubMed
Google Scholar
Kalincik T, Jokubaitis V, Izquierdo G, Duquette P, Girard M, Grammond P, Lugaresi A, Oreja-Guevara C, Bergamaschi R, Hupperts R, et al. Comparative effectiveness of glatiramer acetate and interferon beta formulations in relapsing-remitting multiple sclerosis. Mult Scler. 2015;21:1159–71.
Article
CAS
PubMed
Google Scholar
Nordin N, Hillert J, Piehl F, Svenningsson A, Lycke J, Fagius J, Walentin F, Martin C, Nilsson P, Feltelius N, et al. The "Immunomodulation and Multiple Sclerosis Epidemiology" (IMSE II) study; A Swedish nationwide pharmaco-epidemiological and genetic study focused on long-term safety and efficacy of fingolimod (Gilenya®), 29th Congress of the European Committee for Research and Treatment in Multiple Sclerosis (ECTRIMS). Copenhagen: Poster; 2013. p. 975.
Google Scholar
He A, Spelman T, Jokubaitis V, Havrdova E, Horakova D, Trojano M, Lugaresi A, Izquierdo G, Grammond P, Duquette P, et al. Comparison of switch to fingolimod or interferon beta/glatiramer acetate in active multiple sclerosis. JAMA Neurol. 2015;72:405–13.
Article
PubMed
Google Scholar
North American Research Committee on Multiple Sclerosis.. Publications validating NARCOMS data and instruments. 2016. http://narcoms.org/publications. Accessed 26 February 2016.
Westerlind H, Ramanujam R, Uvehag D, Kuja-Halkola R, Boman M, Bottai M, Lichtenstein P, Hillert J. Modest familial risks for multiple sclerosis: a registry-based study of the population of Sweden. Brain. 2014;137:770–78.
Article
PubMed
PubMed Central
Google Scholar
Ziemssen T, Kern R, Cornelissen C. The PANGAEA study design – a prospective, multicenter, non-interventional, long-term study on fingolimod for the treatment of multiple sclerosis in daily practice. BMC Neurol. 2015;15:93.
Article
PubMed
PubMed Central
Google Scholar
Willke RJ, Mullins CD. Ten commandments" for conducting comparative effectiveness research using "real-world data. J Managed Care Pharm. 2011;17:S10–5.
Article
Google Scholar
Normand SL, Sykora K, Li P, Mamdani M, Rochon PA, Anderson GM. Readers guide to critical appraisal of cohort studies: 3. Analytical strategies to reduce confounding. BMJ. 2005;330:1021–3.
Article
PubMed
PubMed Central
Google Scholar
Austin PC. An Introduction to propensity score methods for reducing the effects of confounding in observational studies. Multivariate Behav Res. 2011;46:399–424.
Article
PubMed
PubMed Central
Google Scholar
Cepeda MS, Boston R, Farrar JT, Strom BL. Comparison of logistic regression versus propensity score when the number of events is low and there are multiple confounders. Am J Epidemiol. 2003;158:280–7.
Article
PubMed
Google Scholar
Möller HJ. Effectiveness studies: advantages and disadvantages. Dialogues Clin Neurosci. 2011;13:199–207.
PubMed
PubMed Central
Google Scholar
CONSORT. The CONSORT Statement. 2010. http://www.consort-statement.org/. Accessed 12 April 2016.
STROBE. Checklist for cohort studies. 2007. http://www.strobe-statement.org/index.php?id=available-checklists. Accessed 12 April 2016.
Dreyer NA, Velentgas P, Westrich K, Dubois R. The GRACE checklist for rating the quality of observational studies of comparative effectiveness: a tale of hope and caution. J Manag Care Spec Pharm. 2014;20:301–8.
PubMed
Google Scholar
Hurwitz BJ. Analysis of current multiple sclerosis registries. Neurology. 2011;76:S7–13.
Article
PubMed
Google Scholar
Hurwitz BJ. Registry studies of long-term multiple sclerosis outcomes: description of key registries. Neurology. 2011;76:S3–6.
Article
PubMed
Google Scholar
Kremenchutzky M, Rice GP, Baskerville J, Wingerchuk DM, Ebers GC. The natural history of multiple sclerosis: a geographically based study 9: observations on the progressive phase of the disease. Brain. 2006;129:584–94.
Article
CAS
PubMed
Google Scholar
Weinstock-Guttman B, Jacobs LD, Brownscheidle CM, Baier M, Rea DF, Apatoff BR, Blitz KM, Coyle PK, Frontera AT, Goodman AD, et al. Multiple sclerosis characteristics in African American patients in the New York State Multiple Sclerosis Consortium. Mult Scler. 2003;9:293–8.
Article
CAS
PubMed
Google Scholar
Butzkueven H, Chapman J, Cristiano E, Grand'Maison F, Hoffmann M, Izquierdo G, Jolley D, Kappos L, Leist T, Pohlau D, et al. MSBase: an international, online registry and platform for collaborative outcomes research in multiple sclerosis. Mult Scler. 2006;12:769–74.
Article
CAS
PubMed
Google Scholar
Jokubaitis VG, Spelman T, Kalincik T, Izquierdo G, Grand'Maison F, Duquette P, Girard M, Lugaresi A, Grammond P, Hupperts R, et al. Predictors of disability worsening in clinically isolated syndrome. Ann Clin Transl Neurol. 2015;2:479–91.
Article
PubMed
PubMed Central
Google Scholar
Spelman T, Kalincik T, Zhang A, Pellegrini F, Wiendl H, Kappos L, Tsvetkova L, Belachew S, Hyde R, Verheul F, et al. Comparative efficacy of switching to natalizumab in active multiple sclerosis. Ann Clin Transl Neurol. 2015;2:373–87.
Article
CAS
PubMed
PubMed Central
Google Scholar
Kappos L, Radue EW, Comi G, Montalban X, Butzkueven H, Wiendl H, Giovannoni G, Hartung HP, Derfuss T, Naegelin Y, et al. Switching from natalizumab to fingolimod: a randomized, placebo-controlled study in RRMS. Neurology. 2015;85:29–39.
Article
CAS
PubMed
PubMed Central
Google Scholar
Frisell T, Forsberg L, Nordin N, Kiesel C, Alfredsson L, Askling J, Hillert J, Olsson T, Piehl F. Comparative analysis of first-year fingolimod and natalizumab drug discontinuation among Swedish patients with multiple sclerosis. Mult Scler. 2016;22:85–93.
Article
CAS
PubMed
Google Scholar
Hupperts R, Ghazi-Visser L, Martins Silva A, Arvanitis M, Kuusisto H, Marhardt K, Vlaikidis N, Group SS. The STAR Study: a real-world, international, observational study of the safety and tolerability of, and adherence to, serum-free subcutaneous interferon beta-1a in patients with relapsing multiple sclerosis. Clin Ther. 2014;36:1946–57.
Article
CAS
PubMed
Google Scholar
Ziemssen T, Moser S, Schwartz HJ, Fuchs A, van Lockven T, Cornelissen C. Comparison of efficacy and treatment satisfaction of patients on baseline therapy and fingolimod: results of the interim analysis of two German registry studies PANGAEA and PEARL. American Academy of Neurology (AAN) Annual Meeting, April 26–May 3, 2014, Philadelphia PA; Poster P7.232.
Bonafede MM, Johnson BH, Watson C. Health care-resource utilization before and after natalizumab initiation in multiple sclerosis patients in the US. Clinicoecon Outcomes Res. 2013;6:11–20.
Article
PubMed
PubMed Central
Google Scholar
Fox E, Edwards K, Burch G, Wynn DR, LaGanke C, Crayton H, Hunter SF, Huffman C, Kim E, Pestreich L, et al. Outcomes of switching directly to oral fingolimod from injectable therapies: Results of the randomized, open-label, multicenter, Evaluate Patient OutComes (EPOC) study in relapsing multiple sclerosis. Mult Scler Relat Disord. 2014;3:607–19.
Article
PubMed
Google Scholar
Vormfelde SV, Ortler S, Ziemssen T. Multiple Sclerosis Therapy With Disease-Modifying Treatments in Germany: The PEARL (ProspEctive phArmacoeconomic cohoRt evaluation) Noninterventional Study Protocol. JMIR Res Protocol. 2015;5(1), e23.
Article
Google Scholar
Stephenson JJ, Kern DM, Agarwal SS, Zeidman R, Rajagopalan K, Kamat SA, Foley J. Impact of natalizumab on patient-reported outcomes in multiple sclerosis: a longitudinal study. Health Qual Life Outcomes. 2012;10:155.
Article
PubMed
PubMed Central
Google Scholar
Flachenecker P, Buckow K, Pugliatti M, Kes VB, Battaglia MA, Boyko A, Confavreux C, Ellenberger D, Eskic D, Ford D, et al. Multiple sclerosis registries in Europe - results of a systematic survey. Mult Scler. 2014;20:1523–32.
Article
PubMed
Google Scholar
A collaborative initiative to improve MS research and policy across Europe. Better outcomes with better data. 2014. http://www.emsp.org/wp-content/uploads/2015/06/140930-EUReMS-Report.pdf. Accessed 26 February 2016.
Barkhof F, Scheltens P, Frequin ST, Nauta JJ, Tas MW, Valk J, Hommes OR. Relapsing-remitting multiple sclerosis: sequential enhanced MR imaging vs clinical findings in determining disease activity. AJR Am J Roentgenol. 1992;159:1041–7.
Article
CAS
PubMed
Google Scholar
Giorgio A, Stromillo ML, Bartolozzi ML, Rossi F, Battaglini M, De Leucio A, Guidi L, Maritato P, Portaccio E, Sormani MP, et al. Relevance of hypointense brain MRI lesions for long-term worsening of clinical disability in relapsing multiple sclerosis. Mult Scler. 2014;20:214–9.
Article
PubMed
Google Scholar
Sormani MP, Rio J, Tintore M, Signori A, Li D, Cornelisse P, Stubinski B, Stromillo M, Montalban X, De Stefano N. Scoring treatment response in patients with relapsing multiple sclerosis. Mult Scler. 2013;19:605–12.
Article
CAS
PubMed
Google Scholar
Barkhof F, Calabresi PA, Miller DH, Reingold SC. Imaging outcomes for neuroprotection and repair in multiple sclerosis trials. Nat Rev Neurol. 2009;5:256–66.
Article
PubMed
Google Scholar
Simon JH. Brain atrophy in multiple sclerosis: what we know and would like to know. Mult Scler. 2006;12:679–87.
Article
CAS
PubMed
Google Scholar
Calabrese M, Agosta F, Rinaldi F, Mattisi I, Grossi P, Favaretto A, Atzori M, Bernardi V, Barachino L, Rinaldi L, et al. Cortical lesions and atrophy associated with cognitive impairment in relapsing-remitting multiple sclerosis. Arch Neurol. 2009;66:1144–50.
Article
PubMed
Google Scholar
Sormani MP, Arnold DL, De Stefano N. Treatment effect on brain atrophy correlates with treatment effect on disability in multiple sclerosis. Ann Neurol. 2014;75:43–9.
Article
PubMed
Google Scholar
Prinster A, Quarantelli M, Lanzillo R, Orefice G, Vacca G, Carotenuto B, Alfano B, Brunetti A, Morra VB, Salvatore M. A voxel-based morphometry study of disease severity correlates in relapsing-- remitting multiple sclerosis. Mult Scler. 2010;16:45–54.
Article
CAS
PubMed
PubMed Central
Google Scholar
Zivadinov R, Bakshi R. Central nervous system atrophy and clinical status in multiple sclerosis. J Neuroimaging. 2004;14:27S–35S.
Article
PubMed
Google Scholar
Kern R, Haase R, Eisele JC, Thomas K, Ziemssen T. Designing an Electronic Patient Management System for Multiple Sclerosis: Building a Next Generation Multiple Sclerosis Documentation System. Interact J Med Res. 2016;5(1), e2.
Article
PubMed
PubMed Central
Google Scholar