165
Views
4
CrossRef citations to date
0
Altmetric
Review

Dimethyl Fumarate in the Treatment of Relapsing-Remitting Multiple Sclerosis: Patient Reported Outcomes and Perspectives

ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon & ORCID Icon
Pages 373-384 | Published online: 11 Dec 2019

References

  • Gold R, Kappos L, Arnold DL, et al. Placebo-controlled phase 3 study of oral BG-12 for relapsing multiple sclerosis. N Engl J Med. 2012;367(12):1098–1107. doi:10.1056/NEJMoa111428722992073
  • Fox RJ, Miller DH, Phillips JT, et al. Placebo-controlled phase 3 study of oral BG-12 or glatiramer in multiple sclerosis. N Engl J Med. 2012;367(12):1087–1097. doi:10.1056/NEJMoa120632822992072
  • Bomprezzi R. Dimethyl fumarate in the treatment of relapsing-remitting multiple sclerosis: an overview. Ther Adv Neurol Disord. 2015;8(1):20–30. doi:10.1177/175628561456415225584071
  • Nowinski CJ, Miller DM, Cella D. Evolution of patient-reported outcomes and their role in multiple sclerosis clinical trials. Neurotherapeutics. 2017;14(4):934–944. doi:10.1007/s13311-017-0571-628913785
  • Rothwell PM, McDowell Z, Wong CK, Dorman PJ. Doctors and patients don’t agree: cross sectional study of patients’ and doctors’ perceptions and assessments of disability in multiple sclerosis. BMJ (Clin Res Ed). 1997;314(7094):1580–1583. doi:10.1136/bmj.314.7094.1580
  • Janse AJ, Gemke RJ, Uiterwaal CS, van der Tweel I, Kimpen JL, Sinnema G. Quality of life: patients and doctors don’t always agree: a meta-analysis. J Clin Epidemiol. 2004;57(7):653–661. doi:10.1016/j.jclinepi.2003.11.01315358393
  • United States Food and Drug Adminstration. Guidance for Industry. Patient-reported outcome measures: use in medical product development to support laveling claims. 2009; Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome-mreasures-use-medical-product-development-support-labeling-claims. Accessed 66, 2019.
  • Kremenchutzky M, Walt L. Perceptions of health status in multiple sclerosis patients and their doctors. Can J Neurol Sci. 2013;40(2):210–218. doi:10.1017/S031716710001375523419570
  • Ysrraelit MC, Fiol MP, Gaitan MI, Correale J. Quality of life assessment in multiple sclerosis: different perception between patients and neurologists. Front Neurol. 2017;8:729. doi:10.3389/fneur.2017.0072929375468
  • Vaughn CB, Kavak KS, Dwyer MG, et al. Fatigue at enrollment predicts EDSS worsening in the New York state multiple sclerosis consortium. Mult Scler. 2018;1352458518816619.
  • Gnanasakthy A, Lewis S, Clark M, Mordin M, DeMuro C. Potential of patient-reported outcomes as nonprimary endpoints in clinical trials. Health Qual Life Outcomes. 2013;11:83. doi:10.1186/1477-7525-11-8323675876
  • Khurana V, Sharma H, Afroz N, Callan A, Medin J. Patient-reported outcomes in multiple sclerosis: a systematic comparison of available measures. Eur J Neurol. 2017;24(9):1099–1107. doi:10.1111/ene.2017.24.issue-928695634
  • Cella D, Riley W, Stone A, et al. The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005–2008. J Clin Epidemiol. 2010;63(11):1179–1194. doi:10.1016/j.jclinepi.2010.04.01120685078
  • National Institute on Aging. Patient-Reported Outcomes Measurement Information System (PROMIS). Available from: https://www.nia.nih.gov/research/resource/patient-reported-outcomes-measurement-information-system-promis. Accessed 61, 2019.
  • Lavallee D, Chenok K, Love R, et al. Incorporating patient-reported outcomes into health care to engage patients and enhance care. Health Aff. 2016;35(4):575–582. doi:10.1377/hlthaff.2015.1362
  • D’Amico E, Haase R, Review: ZT. Patient-reported outcomes in multiple sclerosis care. Mult Scler Relat Disord. 2019;33:61–66. doi:10.1016/j.msard.2019.05.01931154262
  • Kornberg MD, Bhargava P, Kim PM, et al. Dimethyl fumarate targets GAPDH and aerobic glycolysis to modulate immunity. Science (New York, NY). 2018;360(6387):449–453. doi:10.1126/science.aan4665
  • da Fonseca RR, Johnson WE, O’Brien SJ, Vasconcelos V, Antunes A. Molecular evolution and the role of oxidative stress in the expansion and functional diversification of cytosolic glutathione transferases. BMC Evol Biol. 2010;10:281. doi:10.1186/1471-2148-10-28120843339
  • Ma Q. Role of nrf2 in oxidative stress and toxicity. Annu Rev Pharmacol Toxicol. 2013;53:401–426. doi:10.1146/annurev-pharmtox-011112-14032023294312
  • Ryter SW, Alam J, Choi AM. Heme oxygenase-1/carbon monoxide: from basic science to therapeutic applications. Physiol Rev. 2006;86(2):583–650. doi:10.1152/physrev.00011.200516601269
  • Siegel D, Gustafson DL, Dehn DL, et al. oxidoreductase 1: role as a superoxide scavenger. Mol Pharmacol. 2004;65(5):1238–1247. doi:10.1124/mol.65.5.123815102952
  • Longbrake EE, Ramsbottom MJ, Cantoni C, Ghezzi L, Cross AH, Piccio L. Dimethyl fumarate selectively reduces memory T cells in multiple sclerosis patients. Mult Scler. 2016;22(8):1061–1070. doi:10.1177/135245851560896126459150
  • Spencer CM, Crabtree-Hartman EC, Lehmann-Horn K, Cree BA, Zamvil SS. Reduction of CD8(+) T lymphocytes in multiple sclerosis patients treated with dimethyl fumarate. Neurol Neuroimmunol Neuroinflamm. 2015;2(3):e76. doi:10.1212/NXI.000000000000007625738172
  • Mathias A, Perriot S, Canales M, et al. Impaired T-cell migration to the CNS under fingolimod and dimethyl fumarate. Neurol Neuroimmunol Neuroinflamm. 2017;4(6):e401. doi:10.1212/NXI.000000000000040129075657
  • Kihara Y, Groves A, Rivera RR, Chun J. Dimethyl fumarate inhibits integrin alpha4 expression in multiple sclerosis models. Ann Clin Transl Neurol. 2015;2(10):978–983. doi:10.1002/acn3.25126478898
  • Peng H, Li H, Sheehy A, Cullen P, Allaire N, Scannevin RH. Dimethyl fumarate alters microglia phenotype and protects neurons against proinflammatory toxic microenvironments. J Neuroimmunol. 2016;299:35–44. doi:10.1016/j.jneuroim.2016.08.00627725119
  • Munoz MA, Kulick CG, Kortepeter CM, Levin RL, Avigan MI. Liver injury associated with dimethyl fumarate in multiple sclerosis patients. Mult Scler. 2017;23(14):1947–1949. doi:10.1177/135245851668835128086032
  • Briner M, Bagnoud M, Miclea A, et al. Time course of lymphocyte repopulation after dimethyl fumarate-induced grade 3 lymphopenia: contribution of patient age. Ther Adv Neurol Disord. 2019;12:1756286419843450. doi:10.1177/175628641984345031105768
  • Kappos L, Gold R, Arnold DL, et al. Quality of life outcomes with BG-12 (dimethyl fumarate) in patients with relapsing-remitting multiple sclerosis: the DEFINE study. Mult Scler. 2014;20(2):243–252. doi:10.1177/135245851350781724150779
  • Kita M, Fox RJ, Phillips JT, et al. Effects of BG-12 (dimethyl fumarate) on health-related quality of life in patients with relapsing-remitting multiple sclerosis: findings from the CONFIRM study. Mult Scler. 2014;20(2):253–257. doi:10.1177/135245851350781824150778
  • Kita M, Fox RJ, Gold R, et al. Effects of delayed-release dimethyl fumarate (DMF) on health-related quality of life in patients with relapsing-remitting multiple sclerosis: an integrated analysis of the phase 3 DEFINE and CONFIRM studies. Clin Ther. 2014;36(12):1958–1971. doi:10.1016/j.clinthera.2014.08.01325315404
  • McHorney C, Raczek JA. The MOS 36-item short-form health survey (SF-36): II. Psychometric and clinical tests of validity in measuring physical and mental health constructs. Medicare Care. 1993;31(3):247–263. doi:10.1097/00005650-199303000-00006
  • EuroQol Group..EuroQol–a new facility for the measurement of health-related quality of life. Health Policy. 1990;16(3):199–208. doi:10.1016/0168-8510(90)90421-910109801
  • Kresa-Reahl K, Repovic P, Robertson D, Okwuokenye M, Meltzer L, Mendoza JP. Effectiveness of delayed-release dimethyl fumarate on clinical and patient-reported outcomes in patients with relapsing multiple sclerosis switching from glatiramer acetate: RESPOND, a prospective observational study. Clin Ther. 2018;40(12):2077–2087. doi:10.1016/j.clinthera.2018.10.01130470580
  • Benedict RH, Fishman I, McClellan MM, Bakshi R, Weinstock-Guttman B. Validity of the beck depression inventory-fast screen in multiple sclerosis. Mult Scler. 2003;9(4):393–396. doi:10.1191/1352458503ms902oa12926845
  • Mills RJ, Young CA, Pallant JF, Tennant A. Rasch analysis of the Modified Fatigue Impact Scale (MFIS) in multiple sclerosis. J Neurol Neurosurg Psychiatry. 2010;81(9):1049–1051. doi:10.1136/jnnp.2008.15134020547635
  • Atkinson MJ, Sinha A, Hass SL, et al. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004;2:12. doi:10.1186/1477-7525-2-1214987333
  • Krousel-Wood M, Islam T, Webber LS, Re RN, Morisky DE, Muntner P. New medication adherence scale versus pharmacy fill rates in seniors with hypertension. Am J Manag Care. 2009;15(1):59–66.19146365
  • Wyrwich K, Tierney W, Babu A, Kroenke K, Wolinsky F. A comparison of clinically important differences in health-related quality of life for patients with chronic lung disease, asthma, or heart disease. Health Serv Res. 2005;40(2):577–592. doi:10.1111/hesr.2005.40.issue-215762908
  • Ward M, Guthrie L, Alba M. Clinically important changes in short-form-36 scales for use in rheumatoid arthritis clinical trials: the impact of low responsiveness. Arthritis Care Res (Hoboken). 2014;66(12):1783–1789. doi:10.1002/acr.2239224980417
  • Glanz BI, Degano IR, Rintell DJ, Chitnis T, Weiner HL, Healy BC. Work productivity in relapsing multiple sclerosis: associations with disability, depression, fatigue, anxiety, cognition, and health-related quality of life. Value Health. 2012;15(8):1029–1035. doi:10.1016/j.jval.2012.07.01023244804
  • Lee A, Pike J, Edwards MR, Petrillo J, Waller J, Jones E. Quantifying the benefits of dimethyl fumarate over beta interferon and Glatiramer acetate therapies on work productivity outcomes in MS Patients. Neurol Ther. 2017;6(1):79–90. doi:10.1007/s40120-016-0061-528093681
  • Gold SM, Heesen C, Schulz H, et al. Disease specific quality of life instruments in multiple sclerosis: validation of the Hamburg Quality of Life Questionnaire in Multiple Sclerosis (HAQUAMS). Mult Scler. 2001;7(2):119–130. doi:10.1177/13524585010070020811424632
  • Dimethyl fumarate (DMF) observational study (ESTEEM). 2019; Available from: https://clinicaltrials.gov/ct2/show/NCT02047097.
  • Giles K, Balashov K, Jones C, et al. Real-World Efficacy of Delayed-Release Dimethyl Fumarate in Early Multiple Sclerosis: Interim Results from ESTEEM. Philadelphia, PA, USA: American Academy of Neurology Annual Meeting; 2019.
  • Everage N, Prada C, Liu S, et al. Safety and efficacy of delayed-release dimethyl fumarate in multiple sclerosis patients treated in routine medical practice: interim analysis of ESTEEM (P6.333). American Academy of Neurology Annual Meeting; 2017; Boston, MA, USA.
  • Berger T, Brochet B, Confalonieri P, et al. Effectiveness of delayed-release dimethyl fumarate on clinical measures and patient-reported outcomes in newly diagnosed and other early relapsing-remitting multiple sclerosis patients: subgroup analysis of PROTEC. American Academy of Neurology Annual Meeting; 2017; Boston, MA, USA.
  • Hauser SL, Bar-Or A, Comi G, et al. Ocrelizumab versus interferon beta-1a in relapsing multiple sclerosis. N Engl J Med. 2017;376(3):221–234. doi:10.1056/NEJMoa160127728002679
  • Cohen JA, Coles AJ, Arnold DL, et al. Alemtuzumab versus interferon beta 1a as first-line treatment for patients with relapsing-remitting multiple sclerosis: a randomised controlled phase 3 trial. Lancet (London, England). 2012;380(9856):1819–1828. doi:10.1016/S0140-6736(12)61769-3
  • Coles AJ, Twyman CL, Arnold DL, et al. Alemtuzumab for patients with relapsing multiple sclerosis after disease-modifying therapy: a randomised controlled phase 3 trial. Lancet (London, England). 2012;380(9856):1829–1839. doi:10.1016/S0140-6736(12)61768-1
  • Fischer JS, LaRocca NG, Miller DM, Ritvo PG, Andrews H, Paty D. Recent developments in the assessment of quality of life in multiple sclerosis (MS). Mult Scler. 1999;5(4):251–259. doi:10.1177/13524585990050041010467384
  • Cella DF, Dineen K, Arnason B, et al. Validation of the functional assessment of multiple sclerosis quality of life instrument. Neurology. 1996;47(1):129–139. doi:10.1212/WNL.47.1.1298710066
  • Vickrey BG, Hays RD, Harooni R, Myers LW, Ellison GW. A health-related quality of life measure for multiple sclerosis. Qual Life Res. 1995;4(3):187–206. doi:10.1007/BF022608597613530
  • Krupp LB, LaRocca NG, Muir-Nash J, Steinberg AD. The fatigue severity scale. Application to patients with multiple sclerosis and systemic lupus erythematosus. Arch Neurol. 1989;46(10):1121–1123. doi:10.1001/archneur.1989.005204601150222803071
  • Meads DM, Doward LC, McKenna SP, Fisk J, Twiss J, Eckert B. The development and validation of the Unidimensional Fatigue Impact Scale (U-FIS). Mult Scler. 2009;15(10):1228–1238. doi:10.1177/135245850910671419556314
  • Penner IK, Raselli C, Stocklin M, Opwis K, Kappos L, Calabrese P. The Fatigue Scale for Motor and Cognitive Functions (FSMC): validation of a new instrument to assess multiple sclerosis-related fatigue. Mult Scler. 2009;15(12):1509–1517. doi:10.1177/135245850934851919995840
  • Mills RJ, Young CA, Pallant JF, Tennant A. Development of a patient reported outcome scale for fatigue in multiple sclerosis: the Neurological Fatigue Index (NFI-MS). Health Qual Life Outcomes. 2010;8:22.20152031
  • Schwartz CE, Vollmer T, Lee H. Reliability and validity of two self-report measures of impairment and disability for MS. North American Research Consortium on Multiple Sclerosis Outcomes Study Group. Neurology. 1999;52(1):63–70. doi:10.1212/WNL.52.1.639921850
  • Gulick EE. Model confirmation of the MS-related symptom checklist. Nurs Res. 1989;38(3):147–153. doi:10.1097/00006199-198905000-000122717438
  • Noordhoff TC, Scheepe JR, t Hoen LA, Sluis TAR, Blok BFM. The Multiple Sclerosis Intimacy and Sexuality Questionnaire (MSISQ-15): validation of the Dutch version in patients with multiple sclerosis and spinal cord injury. Neurourol Urodyn. 2018;37(8):2867–2874. doi:10.1002/nau.2380430168628
  • Burks J, Chancellor M, Bates D, et al. Development and validation of the actionable bladder symptom screening tool for multiple sclerosis patients. Int J MS Care. 2013;15(4):182–192. doi:10.7224/1537-2073.2012-04924453782
  • Learmonth YC, Motl RW, Sandroff BM, Pula JH, Cadavid D. Validation of patient determined disease steps (PDDS) scale scores in persons with multiple sclerosis. BMC Neurol. 2013;13:37. doi:10.1186/1471-2377-13-3723617555
  • Wicks P, Vaughan TE, Massagli MP. The multiple sclerosis rating scale, revised (MSRS-R): development, refinement, and psychometric validation using an online community. Health Qual Life Outcomes. 2012;10:70. doi:10.1186/1477-7525-10-7022709981
  • Hobart J, Lamping D, Fitzpatrick R, Riazi A, Thompson A. The Multiple Sclerosis Impact Scale (MSIS-29): a new patient-based outcome measure. Brain. 2001;124(Pt 5):962–973. doi:10.1093/brain/124.5.96211335698
  • Hobart JC, Riazi A, Lamping DL, Fitzpatrick R, Thompson AJ. Measuring the impact of MS on walking ability: the 12-Item MS Walking Scale (MSWS-12). Neurology. 2003;60(1):31–36. doi:10.1212/WNL.60.1.3112525714
  • Granger C. The LIFEware system. J Rehabil Outcomes Meas. 1999;3:63–69.
  • Finlayson ML, Peterson EW, Fujimoto KA, Plow MA. Rasch validation of the falls prevention strategies survey. Arch Phys Med Rehabil. 2009;90(12):2039–2046. doi:10.1016/j.apmr.2009.07.01319969166
  • Rigby SA, Domenech C, Thornton EW, Tedman S, Young CA. Development and validation of a self-efficacy measure for people with multiple sclerosis: the multiple sclerosis self-efficacy scale. Mult Scler. 2003;9(1):73–81. doi:10.1191/1352458503ms870oa12617272
  • Schwartz CE, Coulthard-Morris L, Zeng Q, Retzlaff P. Measuring self-efficacy in people with multiple sclerosis: a validation study. Arch Phys Med Rehabil. 1996;77(4):394–398. doi:10.1016/S0003-9993(96)90091-X8607766
  • Airlie J, Baker GA, Smith SJ, Young CA. Measuring the impact of multiple sclerosis on psychosocial functioning: the development of a new self-efficacy scale. Clin Rehabil. 2001;15(3):259–265. doi:10.1191/02692150166836264311386395
  • Young CA, Mills RJ, Woolmore J, Hawkins CP, Tennant A. The unidimensional self-efficacy scale for MS (USE-MS): developing a patient based and patient reported outcome. Mult Scler. 2012;18(9):1326–1333. doi:10.1177/135245851243659222492132
  • Giordano A, Uccelli MM, Pucci E, et al. The multiple sclerosis knowledge questionnaire: a self-administered instrument for recently diagnosed patients. Mult Scler. 2010;16(1):100–111. doi:10.1177/135245850935286519995834
  • Benedict RH, Munschauer F, Linn R, et al. Screening for multiple sclerosis cognitive impairment using a self-administered 15-item questionnaire. Mult Scler. 2003;9(1):95–101. doi:10.1191/1352458503ms861oa12617275
  • Lovera J, Bagert B, Smoot KH, et al. Correlations of perceived deficits questionnaire of multiple sclerosis quality of life inventory with beck depression inventory and neuropsychological tests. J Rehabil Res Dev. 2006;43(1):73–82. doi:10.1682/JRRD.2004.09.011816847773
  • Pakenham KI. The nature of caregiving in multiple sclerosis: development of the caregiving tasks in multiple sclerosis scale. Mult Scler. 2007;13(7):929–938. doi:10.1177/135245850707697317881402
  • Pakenham KI. Making sense of caregiving for persons with multiple sclerosis (MS): the dimensional structure of sense making and relations with positive and negative adjustment. Int J Behav Med. 2008;15(3):241–252. doi:10.1080/1070550080222234518696319
  • Pakenham KI, Cox S. Development of the benefit finding in multiple sclerosis (MS) caregiving scale: a longitudinal study of relations between benefit finding and adjustment. Br J Health Psychol. 2008;13(Pt 4):583–602. doi:10.1348/135910707X25084818854061