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Long-term and short-term outcome of multiple sclerosis: a 3-year follow-up study
B G Weinshenker1, M Issa, J Baskerville
1Department of Neurology, Mayo Clinic and Mayo Foundation, Rochester, Minnesota, USA.
Background:
The anticipated rate of short-term worsening of disability scores is the basis of power estimations in clinical trials of progressive multiple sclerosis (MS). While the clinician is most concerned in modifying the long-term outcome (eg, time to reach the Expanded Disability Status Scale [EDSS]6), the end points studied in clinical trials are those describing short-term outcome (eg, worsening of EDSS scores over 1 to 3 years). However, short-term outcome of MS may not be correlated with long-term outcome.
Objectives:
To validate previously published models predicting time to EDSS 6. To establish predictors of short-term outcome of MS.
Setting:
The Ottawa, Ontario, Regional Multiple Sclerosis Clinic.
Patients:
Two hundred fifty-nine patients were followed up prospectively by a single neurologist.
Main Outcome Measures:
Actuarial analysis of time to reach EDSS 6 and change in EDSS scores over a follow-up period of 1 to 3 years.
Results:
The long-term outcome in the Ottawa population was more favorable than published data from London, Ontario. Predictions of time to EDSS 6 were not strongly correlated with the degree of short-term worsening over the follow-up period. Parameters associated with a higher probability of short-term worsening were proximity of the baseline EDSS score to 4.5 and duration of MS less than 20 years.
Conclusion:
Baseline EDSS and duration of MS must be considered in the design of clinical trials of progressive MS.
Insights
Short-term worsening of multiple sclerosis (MS) disability may not predict long-term outcomes. Baseline Expanded Disability Status Scale (EDSS) score and MS duration are key factors for clinical trial design.
Area of Science:
- Neurology
- Clinical Trials
- Multiple Sclerosis Research
Background:
- Clinical trials for progressive multiple sclerosis (MS) often rely on short-term disability score worsening for power calculations.
- Clinicians aim to modify long-term MS outcomes, but trial endpoints typically focus on short-term changes (1-3 years).
- A potential disconnect exists between short-term MS outcomes and long-term prognosis.
Purpose of the Study:
- To validate existing models that predict the time to reach an Expanded Disability Status Scale (EDSS) score of 6.
- To identify predictors of short-term outcomes in patients with MS.
Main Methods:
- Prospective follow-up of 259 patients at the Ottawa Regional Multiple Sclerosis Clinic by a single neurologist.
- Actuarial analysis was used to determine the time to reach EDSS 6.
- Changes in EDSS scores were analyzed over a 1- to 3-year follow-up period.
Main Results:
- The long-term outcome in the Ottawa cohort was more favorable compared to previously published data from London, Ontario.
- Predictions for time to EDSS 6 did not strongly correlate with the extent of short-term disability worsening.
- Factors associated with increased short-term worsening included a baseline EDSS score near 4.5 and MS duration under 20 years.
Conclusions:
- Baseline EDSS score and the duration of MS are critical considerations when designing clinical trials for progressive MS.
- These factors are important for accurately predicting patient outcomes and ensuring adequate statistical power in trials.