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Audit of outcomes in motor neuron disease (MND) patients treated with riluzole
J Douglas Mitchell1, Mary R O'brien, Miland Joshi
1Department of Neurology, Royal Preston Hospital, Fulwood, Preston PR2 9HT, UK. douglas.mitchell@lhtr.nhs.uk
Abstract:
A review of riluzole in MND by NICE concluded that riluzole was modestly effective. NICE however suggested that further studies were needed to gain further insights into its clinical effectiveness. As these studies have not been carried out we audited outcomes in our use of riluzole. Median survival of 148 MND patients receiving riluzole was 3.07 years (2.25 years for 327 patients not given riluzole), hazard ratio 1.66, 95% confidence interval (CI) 1.32-2.12; 3.61 years in 103 limb onset patients given riluzole (2.62 years for 124 limb onset patients not given riluzole), hazard ratio 1.50, 95% CI 1.09-2.05; 2.19 years in 43 bulbar onset patients receiving riluzole (1.84 years for 103 bulbar onset patients not given riluzole), hazard ratio 1.34, 95% CI 0.89-2.07 and 3.80 years in 87 patients with a PEG given riluzole (2.21 years for 261 patients with a PEG not given riluzole), hazard ratio 1.86, 95% CI 1.44-2.39. These findings are comparable with the results of similar analyses from other groups. The uncertainties they raise emphasize the importance of adequate randomized studies being completed prior to licensing of new drugs. They further illustrate the desirability of controlled introduction of new drugs into clinical practice when RCTs have only suggested a modest level of efficacy.
Insights
Riluzole showed modest survival benefits for patients with motor neuron disease (MND). Further randomized studies are crucial to confirm its clinical effectiveness and guide new drug introductions.
Area of Science:
- Neurology
- Pharmacology
- Clinical Effectiveness Research
Background:
- The National Institute for Health and Care Excellence (NICE) previously concluded riluzole offers modest efficacy for motor neuron disease (MND).
- NICE recommended further studies to clarify riluzole's clinical effectiveness, which have not yet been conducted.
- This audit examines real-world outcomes of riluzole use in MND patients in the absence of further randomized controlled trials (RCTs).
Purpose of the Study:
- To audit the clinical outcomes, specifically survival, of patients with motor neuron disease (MND) treated with riluzole.
- To compare survival data between MND patients receiving riluzole and those not receiving the drug.
- To provide real-world evidence on riluzole's effectiveness in different patient subgroups (limb onset, bulbar onset, PEG placement).
Main Methods:
- Retrospective audit of 148 patients with motor neuron disease (MND) receiving riluzole.
- Comparison of median survival between riluzole recipients and 327 matched patients not receiving riluzole.
- Subgroup analysis based on onset type (limb vs. bulbar) and gastrostomy tube (PEG) placement.
Main Results:
- Median survival for MND patients on riluzole was 3.07 years versus 2.25 years for those not on riluzole (Hazard Ratio [HR] 1.66).
- Survival benefits were observed in limb-onset (HR 1.50) and PEG-assisted patients (HR 1.86), but not significantly in bulbar-onset patients (HR 1.34).
- Findings are comparable to other analyses, highlighting persistent uncertainties regarding riluzole's efficacy.
Conclusions:
- The audit results align with previous findings suggesting a modest survival benefit of riluzole in motor neuron disease (MND).
- The observed uncertainties underscore the critical need for robust randomized studies before new drug licensing.
- Controlled introduction of drugs with only modest efficacy suggested by RCTs is desirable.
