Amyotrophic lateral sclerosis and other motor neuron disorders : official publication of the World Federation of Neurology, Research Group on Motor Neuron Diseases
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Amyotroph. Lateral Scler. Other Motor Neuron Disord. · Jun 2004
ReviewAmyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial.
In November 2002, an advisory board meeting was convened by Novartis Pharma to provide recommendations and rationale for clinical trials designed to evaluate new treatments, such as TCH346, for amyotrophic lateral sclerosis (ALS). In terms of selecting appropriate outcome measures, the panel recommended the use of the ALS Functional Rating Scale (ALSFRS-R) to measure primary endpoints. A review of other key issues in this area including regional variations in the epidemiology, diagnosis and management of ALS, defining patient populations and doses of trial medication, and accommodating the likelihood of co-medication with pre-existing treatment in trial design, are discussed.
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Amyotroph. Lateral Scler. Other Motor Neuron Disord. · Mar 2004
Comparative StudyValidation of quality of life instruments in ALS.
Non-invasive ventilation (NIV) probably improves survival in ALS, but its effect on quality of life (QoL) is less clear. Most potentially useful QoL instruments have not been validated in this context. ⋯ Of the generic instruments the SF-36 was most useful, but limited by floor effects. Of the specific instruments, the SAQLI symptoms domain was most responsive to NIV, but this instrument took longer to complete, and several items were inappropriate. The CRQ and ESS are valid and responsive alternatives.
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Amyotroph. Lateral Scler. Other Motor Neuron Disord. · Mar 2004
Comparative StudyMajor stressors facing patients with amyotrophic lateral sclerosis (ALS): a survey to identify their concerns and to compare with those of their caregivers.
To identify and compare the primary existential, physical, and psychosocial stressors affecting patients with ALS and their caregivers. Health care providers, together with patients and their caregivers, are challenged to identify both physical and psychosocial concerns that have the greatest impact on quality of life over the course of a serious illness. It is also helpful to understand the priorities of these concerns from the patients' and caregivers' perspectives so that we can render optimal care and help patients and their families with the myriad problems that accompany a progressive and fatal disease. ⋯ Health care professionals should apply a holistic approach to treatment and care of patients with ALS. Families should be included in the process, and it should not be assumed that patients and caregivers will agree on all issues. Future research should focus on therapeutic interventions to help ALS patients and their families cope with the multiple stressors accompanying a catastrophic illness.
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Amyotroph. Lateral Scler. Other Motor Neuron Disord. · Sep 2003
ReviewRiluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).
Riluzole 100 mg probably prolongs survival in patients with amyotrophic lateral sclerosis by about two months and the safety of the drug is not a major concern. The evidence from randomized controlled trials indicates that patients taking riluzole probably survive longer than patients taking placebo. The beneficial effects are very modest and the drug is expensive. Adverse effects from riluzole are relatively minor and for the most part reversible after stopping the drug. Riluzole has been approved for treatment of patients with amyotrophic lateral sclerosis in many countries but not all. Questions persist about its clinical utility because of high cost, modest efficacy and concern over adverse effects. ⋯ Riluzole 100 mg daily is reasonably safe and probably prolongs survival by about two months in patients with ALS. More studies are needed, especially to clarify its effect in older patients (over 75 years), and those with more advanced disease.