Construction and validation of a quality of life questionnaire for neuromuscular disease (INQoL)

Vincent, Kelly; Carr, Alison; Walburn, Jessica; Scott, David; Rose, Michael R.

doi:10.1212/01.wnl.0000257819.47628.41

Cited by 147 publications

(165 citation statements)

References 23 publications

Supporting

Mentioning

158

Contrasting

Unclassified

Order By: Relevance

“…The internal consistency of the Serbian version of INQoL was excellent except for treatment section. This may be explained by the fact that treatment section includes questions about positive and negative effects of perceived and expected treatment [11], thus intercorrelation between these items was expected to be low.…”

Section: Resultsmentioning

confidence: 99%

“…This is due to the specifi c calculation of the treatment scores: they are expressed as the trade-off between the positive and side effects of the treatment [11].…”

Section: Resultsmentioning

confidence: 99%

“…INQoL has also been validated in Italy confi rming that INQoL is a reliable, valid and practical measure for QoL assessment able to capture issues specifi cally relevant to the muscle condition [12]. These studies suggested that validity of INQoL should be extended to other countries using larger and more homogenous diagnostic groups [11,12].…”

Section: Introductionmentioning

confidence: 90%

“…The Individualized Neuromuscular Quality of Life questionnaire (INQoL) is a muscle disease specifi c QoL measure which has been developed and validated in the United Kingdom on a heterogeneous group of patients with neuromuscular disorders [11]. INQoL has also been validated in Italy confi rming that INQoL is a reliable, valid and practical measure for QoL assessment able to capture issues specifi cally relevant to the muscle condition [12].…”

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Serbian Validation of the Individualized Neuromuscular Quality of Life Questionnaire (INQoL) in Adults With Myotonic Dystrophy Type 1

Peric

2011

J Neurol Res

View full text Add to dashboard Cite

show abstract

Section: Resultsmentioning

confidence: 99%

“…This is due to the specifi c calculation of the treatment scores: they are expressed as the trade-off between the positive and side effects of the treatment [11].…”

Section: Resultsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 90%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Serbian Validation of the Individualized Neuromuscular Quality of Life Questionnaire (INQoL) in Adults With Myotonic Dystrophy Type 1

Peric

2011

J Neurol Res

View full text Add to dashboard Cite

show abstract

“…Finally, having been developed for use in other conditions, these existing measures lacked adequate content validity in the specific 'context of use' of GNEM studies. As such, none of the measures were deemed adequately specific to GNEM and the unique functional limitations experienced in these patients as a result of their distinct pattern of weakness [5][6][7][8][9][10][11] and thus, the need to develop an instrument specific to functional impairment in GNEM was identified. The GNEM functional activity scale (GNEM-FAS) was therefore developed.…”

mentioning

confidence: 99%

Development and preliminary evidence of the psychometric properties of the GNE myopathy functional activity scale

et al. 2018

View full text Add to dashboard Cite

Aim: GNE myopathy, a rare, severe, progressive myopathy, presents with lower extremity distal muscle weakness. The GNE myopathy functional activity scale (GNEM-FAS) evaluates the impact of GNE myopathy on functioning in adults. This paper presents the psychometric validation of the GNEM-FAS. Patients & methods: Validation of the GNEM-FAS was performed using data from a randomized, double-blind, placebo-controlled Phase-II study (n = 46). Results: Domain score distributions were acceptable. Moderate inter-item correlations (typical range, 0.40-0.70), strong item convergent and discriminant validity and high internal consistency reliability (α = 0.88-0.92) supported the instrument structure. Test-retest reliability was strong (ICC range: 0.87-0.95). Scale scores distinguished among subjects with differing disease severity (p < 0.05). Conclusion: This study provides preliminary evidence of the GNEM-FAS as a valid, reliable assessment.

show abstract

Mexiletine for Symptoms and Signs of Myotonia in Nondystrophic Myotonia

Statland

Bundy

Rayan

et al. 2012

JAMA

166

173

View full text Add to dashboard Cite

Context Non-dystrophic myotonias (NDM) are rare diseases caused by mutations in skeletal muscle ion channels. Patients experience delayed muscle relaxation causing functionally-limiting stiffness and pain. Mexiletine-induced sodium channel blockade reduced myotonia in case studies and one single blind trial. As is common in rare diseases, larger studies of safety and efficacy have not previously been considered feasible. Objective To determine the effects of mexiletine for symptoms and signs of myotonia in NDM. Design, Setting, and Participation Fifty-nine patients with NDM participated in a randomized, double-blind, placebo-controlled two-period crossover study conducted between December 23, 2008 and March 30, 2011 at 7 neuromuscular referral centers in 4 countries, as part of the NIH-funded Rare Disease Clinical Research Network. Intervention Oral 200 mg mexiletine or placebo capsules three times daily for 4 weeks, followed by the opposite intervention for 4 weeks, with 1 week wash-out between periods. Main Outcome Measures Patient-reported stiffness recorded on an interactive voice response diary (IVR) was the primary endpoint (1 ‘minimal’ to 9 ‘worst ever experienced’). Secondary endpoints included IVR-reported changes in pain, weakness, and tiredness, clinical myotonia assessment, quantitative grip myotonia, Individualized Neuromuscular Quality of Life (INQoL, percent of maximal detrimental impact), SF-36, electrophysiological exercise testing, and needle EMG. Results Mexiletine significantly improved patient-reported stiffness on the IVR. Because of a statistically significant interaction between treatment and period for this outcome, primary endpoint is presented by period (period 1 means were mexiletine 2.53 versus placebo 4.21, difference −1.68, 95% Confidence Interval [CI] −2.66, −0.706, P<0.001; period 2 means were mexiletine 1.60 versus placebo 5.27, difference −3.68, 95% CI −3.85, −0.139, P=0.04). Mexiletine improved the INQoL QOL score (mexiletine 14.0, placebo 16.7, difference −2.69, 95% CI −4.07, −1.30, P<0.001) and decreased handgrip myotonia on clinical exam (seconds: mexiletine 0.164, placebo 0.494, difference −0.330, 95% CI −0.633, −0.142, P<0.001). The most common adverse effect was gastrointestinal (9 mexiletine, 1 placebo). Two participants experienced transient cardiac effects that did not require stopping the study (1 placebo, 1 mexiletine). One serious adverse event was determined to be not study-related. Conclusion In this preliminary study of patients with NDM, the use of mexiletine compared with placebo resulted in improved patient-reported stiffness over 4 weeks of treatment, despite some concern about the maintenance of blinding. Trial Registration Clinicaltrials.gov identifier: NCT 00832000

show abstract

Construction and validation of a quality of life questionnaire for neuromuscular disease (INQoL)

Abstract: The Individualized Neuromuscular Quality of Life is a validated muscle disease specific measure of quality of life developed from the experiences of patients with muscle disease and can be used for individuals or large samples.

Cited by 147 publications

References 23 publications

Serbian Validation of the Individualized Neuromuscular Quality of Life Questionnaire (INQoL) in Adults With Myotonic Dystrophy Type 1

Serbian Validation of the Individualized Neuromuscular Quality of Life Questionnaire (INQoL) in Adults With Myotonic Dystrophy Type 1

Development and preliminary evidence of the psychometric properties of the GNE myopathy functional activity scale

Mexiletine for Symptoms and Signs of Myotonia in Nondystrophic Myotonia

Contact Info

Product

Resources

About