Disease-modifying treatment preferences and decision-making in a multiple sclerosis randomized and observational clinical trial (DELIVER-MS).
Tallantyre EC., Planchon SM., Howard H., Mays J., Frowd N., Cohen JA., Coles A., Hersh CM., Miller DM., Dever M., Gray EH., LaRocca NG., Nakamura K., Bale C., Covey-Crump G., Alvarez E., Arun T., Brownlee WJ., Craner M., Freeman L., Frost N., Goldman MD., Gupta RK., Harding KE., Hobart J., Hutton GJ., Hyland MH., Kalra S., Kannan M., Kantarci O., Lashley D., Lily O., Mahad D., Nicholas JA., Nicholas R., Paling D., Pearson OR., Rice CM., Samudralwar R., Schmalstieg WF., Singh M., Zune The E., Weinstock-Guttman B., Zabeti A., Love TE., Gunzler DD., Liu Y., Gerry S., Evangelou N., Ontaneda D.
BACKGROUND: There is growing support for high-efficacy disease-modifying therapy (DMT) in multiple sclerosis (MS), but escalation (ESC) approaches remain common. OBJECTIVE: To describe decision-making in a pragmatic trial of early high-efficacy treatment (EHT) versus ESC. METHODS: DELIVER-MS is a multi-center, pragmatic, randomized controlled trial (RCT) with a parallel observational study (OBS), which enrolled treatment-naïve people with RRMS in 31 UK/US sites. Primary outcome was as follows: 36-month brain volume loss according to initial treatment approach (EHT vs. ESC). Stepwise multivariable logistic regression was used to predict participation in RCT versus OBS and choice of EHT versus ESC within the OBS cohort. RESULTS: In total, 816 people with MS were enrolled. Participants declined randomization due to preference for a particular DMT (85%), efficacy concerns (20%), and safety concerns (9%). RCT versus OBS participation was associated with lower relapse rate (p = 0.043) and greater brain parenchymal fraction (p = 0.002). Among 374 in the OBS cohort, 125 (33%) chose ESC and 249 (67%) chose EHT. People commencing EHT had higher education attainment (p < 0.001) and relapse rate (p = 0.025). CONCLUSION: Baseline DELIVER-MS data demonstrate that participants with milder disease are more likely to participate in RCT. The choice of EHT versus ESC was associated with demographic factors and disease activity. CLINICAL TRIAL REGISTRATION: NCT03535298.