TY - JOUR
T1 - Assessing treatment outcomes in multiple sclerosis trials and in the clinical setting
AU - Tur, Carmen
AU - Moccia, Marcello
AU - Barkhof, Frederik
AU - Chataway, Jeremy
AU - Sastre-Garriga, Jaume
AU - Thompson, Alan J.
AU - Ciccarelli, Olga
PY - 2018/2/1
Y1 - 2018/2/1
N2 - Increasing numbers of drugs are being developed for the treatment of multiple sclerosis (MS). Measurement of relevant outcomes is key for assessing the efficacy of new drugs in clinical trials and for monitoring responses to disease-modifying drugs in individual patients. Most outcomes used in trial and clinical settings reflect either clinical or neuroimaging aspects of MS (such as relapse and accrual of disability or the presence of visible inflammation and brain tissue loss, respectively). However, most measures employed in clinical trials to assess treatment effects are not used in routine practice. In clinical trials, the appropriate choice of outcome measures is crucial because the results determine whether a drug is considered effective and therefore worthy of further development; in the clinic, outcome measures can guide treatment decisions, such as choosing a first-line disease-modifying drug or escalating to second-line treatment. This Review discusses clinical, neuroimaging and composite outcome measures for MS, including patient-reported outcome measures, used in both trials and the clinical setting. Its aim is to help clinicians and researchers navigate through the multiple options encountered when choosing an outcome measure. Barriers and limitations that need to be overcome to translate trial outcome measures into the clinical setting are also discussed.
AB - Increasing numbers of drugs are being developed for the treatment of multiple sclerosis (MS). Measurement of relevant outcomes is key for assessing the efficacy of new drugs in clinical trials and for monitoring responses to disease-modifying drugs in individual patients. Most outcomes used in trial and clinical settings reflect either clinical or neuroimaging aspects of MS (such as relapse and accrual of disability or the presence of visible inflammation and brain tissue loss, respectively). However, most measures employed in clinical trials to assess treatment effects are not used in routine practice. In clinical trials, the appropriate choice of outcome measures is crucial because the results determine whether a drug is considered effective and therefore worthy of further development; in the clinic, outcome measures can guide treatment decisions, such as choosing a first-line disease-modifying drug or escalating to second-line treatment. This Review discusses clinical, neuroimaging and composite outcome measures for MS, including patient-reported outcome measures, used in both trials and the clinical setting. Its aim is to help clinicians and researchers navigate through the multiple options encountered when choosing an outcome measure. Barriers and limitations that need to be overcome to translate trial outcome measures into the clinical setting are also discussed.
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85041323047&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/29326424
UR - http://www.scopus.com/inward/record.url?scp=85041323047&partnerID=8YFLogxK
U2 - https://doi.org/10.1038/nrneurol.2017.171
DO - https://doi.org/10.1038/nrneurol.2017.171
M3 - Review article
C2 - 29326424
SN - 1759-4758
VL - 14
SP - 75
EP - 93
JO - Nature Reviews. Neurology
JF - Nature Reviews. Neurology
IS - 2
ER -