Audit of outcomes in motor neuron disease (MND) patients treated with riluzole

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.