Patient-Perceived Outcomes and Quality of Life in ALS

Neurotherapeutics, Dec 2014

A variety of outcome measures are used in clinical practice and in research to assess patients with amyotrophic lateral sclerosis (ALS). However, there may be discordance between traditional outcome measures such as strength and physical function, and patient-perceived measures of well-being. One such self-perceived measure, reflecting the patient’s view, is quality of life (QOL). QOL in patients with severe medical disorder is often underestimated by others. Patients with ALS often have high QOL, and this may persist throughout the disease due to shifting expectations and to reprioritization of factors contributing to QOL. QOL instruments can measure health-related QOL (HRQOL) or global QOL, and can be generic or disease-specific. HRQOL refers primarily to physical and mental health. Global QOL is much broader, and is also determined by non-health-related factors. The choice of a QOL instrument depends on whether the setting is routine patient care or clinical research, whether or not the outcome of a specific intervention is being assessed, and upon the expected efficacy or toxicity of the intervention. Global QOL instruments are best for individual clinical patient care or for comparing groups. HRQOL or a combination of HRQOL and global QOL instruments are most appropriate for assessing specific interventions.

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Patient-Perceived Outcomes and Quality of Life in ALS

Neurotherapeutics Patient-Perceived Outcomes and Quality of Life in ALS Zachary Simmons 0 Introduction: Why Patient-Perceived Outcomes Matter 0 0 Z. Simmons ( 1 ) Department of Neurology, Penn State Hershey Medical Center , EC 037, Hershey, PA 17033 , USA A variety of outcome measures are used in clinical practice and in research to assess patients with amyotrophic lateral sclerosis (ALS). However, there may be discordance between traditional outcome measures such as strength and physical function, and patient-perceived measures of wellbeing. One such self-perceived measure, reflecting the patient's view, is quality of life (QOL). QOL in patients with severe medical disorder is often underestimated by others. Patients with ALS often have high QOL, and this may persist throughout the disease due to shifting expectations and to reprioritization of factors contributing to QOL. QOL instruments can measure health-related QOL (HRQOL) or global QOL, and can be generic or disease-specific. HRQOL refers primarily to physical and mental health. Global QOL is much broader, and is also determined by non-health-related factors. The choice of a QOL instrument depends on whether the setting is routine patient care or clinical research, whether or not the outcome of a specific intervention is being assessed, and upon the expected efficacy or toxicity of the intervention. Global QOL instruments are best for individual clinical patient care or for comparing groups. HRQOL or a combination of HRQOL and global QOL instruments are most appropriate for assessing specific interventions. Amyotrophic lateral sclerosis; Quality of life; Health-related quality of life; Psychological health; Outcome measures - Published online: 11 December 2014 # The American Society for Experimental NeuroTherapeutics, Inc. 2014 A variety of outcome measures are used to assess patients with ALS in clinical practice and in research. Common measures in routine clinical care include limb strength as measured by manual muscle testing or hand-held dynamometry, physical function as measured by the ALS Functional Rating Scale (ALSFRS) and its revised version (ALSFRS-R) [ 1, 2 ], and respiratory parameters such as forced vital capacity, maximum inspiratory pressure and sniff nasal inspiratory force. Research outcome measures have varied widely, due to the lack of a specific biomarker to measure ALS disease progression. The most common have been survival, rate of change in strength, and rate of change in function [3]. However, there is not always concordance between traditional outcome measures and patient-perceived measures of well-being. This is not unique to ALS, but has been found in many neurological and non-neurological diseases [ 4–7 ]. To date, only one drug, riluzole, has been shown to be effective in altering the natural history of ALS [ 8, 9 ], and it is not clear if the very modest average benefit conferred by riluzole is perceived by patients as being meaningful. As the field moves forward, it will be important to determine whether the benefits of new therapies are meaningful to patients. A cure for ALS would, of course, be meaningful to the individuals being treated. But current drug trials for ALS are geared toward extending survival, slowing rate of progression of weakness, or slowing loss of function. The concept of “clinically meaningful” changes in ALS measures is just beginning to be studied [10]. A different, or at least complementary, way to assess the perceived value of a new therapy is to measure its impact on quality of life (QOL), as perceived by the patient. For drug trials, a critical question is whether the benefits outweigh the costs. Ignoring the economic implications, the costs can be thought of primarily as being of two types: 1) adverse effects of medications, and 2) the physical and psychological costs of remaining alive longer with a progressive, degenerative neurological condition. One can imagine scenarios in which an experimental drug prolonged survival or slowed rate of disease progression, but with profound fatigue, or persistent nausea, or an increase in pain and cramping. Using “objective” clinical trial endpoints such as survival or rate of change in strength and function, such a treatment would be considered successful. However, from the patient’s perspective, the treatment might be considered a failure if they deemed their QOL to be poorer and if this led them to question the value of the added survival time. QOL must be considered not only in the context of therapeutic trials, but in standard clinical care as well. Noninvestigational clinical care measures for ALS are currently palliative, including the optimization of mobility and activities of daily living, nutrition, communication, respiratory function, psychological well-being, pseudobulbar affect, sialorrhea, pain, cramps, spasticity, fatigue, sleep, and end-of-life decisions [ 11, 12 ]. It is important to assess whether our standard clinical m (...truncated)


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Zachary Simmons. Patient-Perceived Outcomes and Quality of Life in ALS, Neurotherapeutics, 2015, pp. 394-402, Volume 12, Issue 2, DOI: 10.1007/s13311-014-0322-x