Abstract
Objectives: There have been attempts to establish biomarkers for motor neuron disease, without success. The study aim to seek possible markers to be used in the clinical routine evaluation, to optimize timing for palliative interventions. Methods: A cohort study evaluated clinical, respiratory and neurophysiological variables every 3-4 months across 20 months in 101 patients with motor neuron disease using riluzole. Primary endpoint was death or tracheostomy. The most significant parameters in cox regression analysis created a predictive model. Results: There were 58 men and 43 women with a mean age of 57.2 ± 11.7 years. 77 patients (76.2%) had spinal onset and 24 (23.8%) had bulbar onset. The mean survival time was 43.5 ± 5.7 months (CI 95% 32.3-54.8). The variables related to worse prognosis were: age > 65 years (HR=2.50, CI 95% (1.23–5.08); involvement of a second site in less than six months (HR=2.02, CI 95% (1.04 – 3.94); supine Forced Vital Capacity <63% (HR=2.78, CI 95% 1.03–7.48), neck weakness (HR=2.28, CI 95% (1.03–5.05) and presence of pyramidal syndrome (HR= 2.36, CI 95% (1.05–5.33). Conclusion: It was created a five-factors set that predicts evolution to death or tracheostomy within one year.
Highlights
Motor Neuron Disease (MND) is a rare neurodegenerative disease characterized by progressive loss of upper motor neurons in the brain and lower motor neurons in the brain stem and spinal cord, resulting in generalized weakness and muscle atrophy
This study aims to create a predictive model by combining simple possible markers to be used in the routine evaluation of patients to improve timing to palliative measures
The inclusion criteria were a) diagnosis of motor neuron disease done by a neurologist that eliminated other similar diseases; b) age over 18 years; c) a written informed consent that was signed by patients; d) a forced vital capacity (FVC) greater than or equal to 50%, except in progressive bulbar palsy; and d) El Escorial ALS diagnostic criteria that were either definite, probable and possible
Summary
Motor Neuron Disease (MND) is a rare neurodegenerative disease characterized by progressive loss of upper motor neurons in the brain and lower motor neurons in the brain stem and spinal cord, resulting in generalized weakness and muscle atrophy. The pathogenesis and course of the disease are heterogeneous, the disorder is inexorably progressive, and up to 70% of patients die within 3 years from symptom onset [1]. There are few treatment options to reduce disease progression and most of measures are palliative. Decision making about end of life in the late stages generally includes nutritional and respiratory support, gastrostomy insertion, non-invasive ventilation (NIV), and invasive mechanical ventilation [1,3]. These practices are often delayed or triggered in a crisis by the occurrence of life-threatening complications [3]
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