Clinical and demographical characteristics in a cohort of MND patients treated with riluzole. Differences between tablets and oral suspension

Abstract

Objective: To describe the clinical and demographic characteristics of patients with MND treated with riluzole by comparing two dosage forms (oral suspension and tablets), as well as the impact on survival in patients with and without dysphagia according to the form of dosage.

Methods: Retrospective and prospective cohort of patients diagnosed with MND at the multidisciplinary functional unit of Motor Neuron Disease in our center in the period between 1 of January 2011 and 31 of December 2020 (n = 742). A descriptive analysis (univariate and bivariate) was carried out and survival curves were estimated.

Results: During the follow-up period, 402 males (54.18%) and 340 females (45.82%) were diagnosed with MND. Of these patients, 632 (97.23%) were being treated with 100mg riluzole: 282 (54.55%) patients took this in tablet form and 235 (45.45%) oral suspension. Riluzole in tablet form is taken more frequently by men than women, in younger age ranges, and mostly without dysphagia (78.31%). Also, it is the predominant dosage form for classic spinal ALS and respiratory phenotypes. Dosages via oral suspension are taken by patients in the older age ranges (over 64.8 years), mostly with dysphagia (53.67%) and more frequently with bulbar phenotypes such as classic bulbar ALS and PBP. Because of this, patients using oral suspension (most of them with dysphagia) had a poorer survival rate (at 90% CI) than patients using tablets (most of them without dysphagia).

Conclusions: The most appropriate dosage form should be given according to the patient’s needs at each stage of the disease and, furthermore, oral suspension could improve adherence to treatment because it avoids having to change from one form (tablet) to the other (suspension) when swallowing disorders appear.

Keywords:

• Amyotrophic lateral sclerosis (ALS)
• motor neuron disease
• dysphagia
• riluzole

Acknowledgements

We would like to express our gratitude to Ramon Moreno Fuentes, Head of Economic Management in our centre, for his generous work in collecting retrospective information; and to ITF Research Pharma, S.L.U. for partly supporting the study and assisting its publication. We appreciate the comments of two anonymous reviewers of a previous version of this work who, without doubt, helped us to improve our work. The usual disclaimer applies.

Declaration of interest

There are no relevant financial or non-financial competing interests to report. This research did not receive any specific grants from funding agencies in the public, commercial, or not-for-profit sectors.