A short and validated multiple sclerosis‐specific health‐related quality of life measurement for routine medical practice

Pompe disease/glycogen storage disease type II, is a rare, lysosomal storage disorder associated with progressive proximal myopathy, causing a gradual loss of muscular function and respiratory insufficiency. Studies of patients with late-onset Pompe disease have used endpoints such as the 6-minute walking test (6MWT) and forced vital capacity (FVC) to assess muscular and respiratory function during disease progression or treatment. However, the relevance of these markers to late-onset Pompe disease and the minimal clinically important difference (MCID) for these endpoints in late-onset Pompe disease have not yet been established. A literature search was carried out to identify studies reporting the MCID (absolute and relative) for the 6MWT and FVC in other diseases. The MCIDs determined in studies of chronic respiratory diseases were used to analyze the results of clinical studies of enzyme replacement therapy in late-onset Pompe disease. In 9 of the 10 late-onset Pompe disease studies reviewed, changes from baseline in the 6MWT were above or within the MCID established in respiratory diseases. Clinical improvement was perceived by patients in 6 of the 10 studies. In 6 of the 9 late-onset Pompe disease studies that reported FVC, the changes from baseline in percentage predicted FVC were above or within the MCID established in respiratory diseases and the difference was perceived as either an improvement or stabilization by patients. However, applying the 6MWT and FVC MCIDs from studies of chronic respiratory diseases to late-onset Pompe disease has several important limitations. Outcome measures in muscular dystrophies include composite measures of muscle function and gait, as well as Rasch-designed and validated tools to assess disease-related quality of life and activities of daily living. Given that the relevance to patients with late-onset Pompe disease of the 6MWT or FVC MCIDs established for chronic respiratory diseases is unclear, these measures should be evaluated specifically in late-onset Pompe disease and alternative outcome measures more specific to neuromuscular disease considered.

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“…Disease-specific tools developed for measuring QoL/ADL in MS and ALS patients [69,70] might also be applicable to late-onset Pompe patients.…”

Section: Methodsmentioning

confidence: 99%

The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

Lachmann

Schöser

2013

Orphanet J Rare Dis

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“…At baseline, we measured alexithymia (TAS-20) scale [31], Beck Depression inventory [31], Anxiety STA-I [31], emotional distress (POMS) [31], assertiveness and selfesteem scales [31], [32]. Neuropathic pain scale (DN4), severity fatigue scale (FSS), sleeping disorder scales (Epworth), EDSS [33] and self-filled validated short TLS-QoL 10 [27] and DC10 [28] scales were used at every visit. All the scales were administered in conformity with CBT.…”

Section: Measurementsmentioning

confidence: 99%

“…The essential concern of QoL in this pathology motivated us to provide further investigation on the beneficial effect of CBT. Aware of these methodological problems, we postponed the therapy trial, in concentrating first to the development of adapted measurement tools: a literature review [27], [28] showed that there was no scale of QoL or coping conducted in the same time specific to MS and adapted to routine practice. A first cross-sectional study of 331 patients [27] identified a short MS-specific optimized QoL scale of 10 items (TLS-QoL 10).…”

Section: Introductionmentioning

confidence: 99%

“…Aware of these methodological problems, we postponed the therapy trial, in concentrating first to the development of adapted measurement tools: a literature review [27], [28] showed that there was no scale of QoL or coping conducted in the same time specific to MS and adapted to routine practice. A first cross-sectional study of 331 patients [27] identified a short MS-specific optimized QoL scale of 10 items (TLS-QoL 10). In the same study, the authors developed an optimized short MS-specific coping scale [28], measuring the negative and positive coping and providing a total score (Devy Coping Scale (DC-10)).…”

Section: Introductionmentioning

confidence: 99%

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A Group Cognitive Behavioural Therapy Improves Quality of Life of Multiple Sclerosis Patients and Delays Disease Progression: A Multi-Centre Controlled Trial

Devy¹,

Lehert

Genty³

2018

ABNE

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Background: Multiple Sclerosis (MS) affects quality of life (QoL). Pharmacological treatments demonstrated benefits on clinical endpoints without improving QoL. We evaluated the effects of a group Cognitive Behavioural Therapy (CBT) on QoL disease progression. Methods: One-year multi-centre controlled multivariate-matched study was organised on Relapsing-Remitting MS (RRMS) patients with Expanded Disability Status Scale (EDSS) < 4, MS duration < 2 years, treated by interferon I? in 11 French centres. For each new patient, the two best-matching patients for age, gender, EDSS, mood, illness duration baseline variables were selected in the other centres. The self-filled Two Lives Scale (TLS)-QoL10 was used at months (M) 0-3-6-9-12-15; the post-baseline mean QoL was the endpoint. We compared CBT + I? to I? alone. The effect of disease progression on QoL was evaluated by modelling, for each visit, the effect of EDSS on QoL at later visits.Results: 19 + 32 patients were recruited. Compared to placebo, improvements of 1.10 (95%CI [0.31-1.89], p = 0.009) and 1.43*** [0.72, 2.15] were observed in the CBT group on QoL and coping scales, respectively. Coping explained 81%*** [57, 100] of the effect of CBT on QoL. QoL was negatively affected by disease progression (0.95*** [-1.21; 0.63]), whereas EDSS was influenced by QoL values (-0.10*** [-0.14; -0.06]).Conclusions: We observed a clinically significant beneficial effect of CBT on QoL, the effect of CBT essentially explained by an increase of coping, a positive influence of QoL on disease progression. QoL is both the most important target for patients and a factor of slowing disease progression.

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“…Multiple sclerosis is considered the prototypic neuro‐inflammatory disease. It is now recognized to have a significant genetic component, although no single gene mutations have been identified, so again this is likely to be a complex genetic influence on risk.…”

mentioning

confidence: 99%

Recent advances in neurology 2013–2014

Schapira¹

2014

Euro J of Neurology

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A short and validated multiple sclerosis‐specific health‐related quality of life measurement for routine medical practice

Abstract: Compared with existing alternatives, our 10-item three-dimensional Hr-QoL measurement tool is adapted to RMP, and constitutes an adequate compromise between precision and ease of use in patients with MS.

Cited by 8 publications

References 24 publications

The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

A Group Cognitive Behavioural Therapy Improves Quality of Life of Multiple Sclerosis Patients and Delays Disease Progression: A Multi-Centre Controlled Trial

Recent advances in neurology 2013–2014

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