St. George's Respiratory Questionnaire Has Longitudinal Construct Validity in Lymphangioleiomyomatosis

Swigris, Jeffrey J.; Lee, Hye-Seung; Inoue, Yoshikazu; Moss, Joel; Singer, Lianne G.; Young, Lisa R.; McCormack, Francis X.

doi:10.1378/chest.12-0161

Cited by 17 publications

(11 citation statements)

References 20 publications

(20 reference statements)

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“…This was the first intervention for LAM that was able to induce clinically relevant changes in SGRQ. In the MILES trial, sirolimus led to improvement on the EuroQOL instrument compared with placebo, but the groups did not differ in terms of SRGQ, even though patients had baseline SGRQ values similar to those in our study [26]. Although our study did not observe a difference in anxiety, it was also the first intervention to demonstrate a trend towards a reduction in symptoms of depression in LAM.…”

Section: Discussioncontrasting

confidence: 49%

Pulmonary rehabilitation in lymphangioleiomyomatosis: a controlled clinical trial

et al. 2016

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Lymphangioleiomyomatosis (LAM) is a cystic lung disease frequently associated with reduced exercise capacity. The aim of this study was to assess safety and efficacy of pulmonary rehabilitation in LAM.This controlled clinical trial included 40 patients with LAM and a low physical activity level. The pulmonary rehabilitation programme comprised 24 aerobic and muscle strength training sessions and education. The primary outcome was exercise capacity (endurance time during a constant work rate exercise test). Secondary outcomes included health-related quality of life (St George's Respiratory Questionnaire (SGRQ)), 6-min walking distance (6MWD), dyspnoea, peak oxygen consumption (V'O2 ), daily physical activity (pedometer), symptoms of anxiety and depression, lung function and peripheral muscle strength (one-repetition maximum).The baseline characteristics were well balanced between the groups. The pulmonary rehabilitation group exhibited improvements in the following outcomes versus controls: endurance time (median (interquartile range) 169 (2-303) s versus -33 (-129-39) s; p=0.001), SGRQ (median (interquartile range) -8 (-16-2) versus 2 (-4-5); p=0.002) and 6MWD (median (interquartile range) 59 (13-81) m versus 20 (-12-30) m; p=0.002). Dyspnoea, peak V'O2 , daily physical activity and muscle strength also improved significantly. No serious adverse events were observed.Pulmonary rehabilitation is a safe intervention and improves exercise capacity, dyspnoea, daily physical activity, quality of life and muscle strength in LAM.

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Section: Discussioncontrasting

confidence: 49%

Pulmonary rehabilitation in lymphangioleiomyomatosis: a controlled clinical trial

et al. 2016

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“…Increased VEGF-D concentrations were positively associated with forced vital capacity (FVC), total lung capacity, residual volume, functional residual capacity, and several patient-reported outcomes, including scores on the functional performance inventory and the St George’s respiratory questionnaire (table 1), which has recently been validated as a longitudinal measure in lymphangioleiomyomatosis. 23 The associations remained significant when we did sensitivity analyses that excluded either participants with tuberous-sclerosis-complex-associated disease or those who were enrolled in the MILES trial on the basis of increased VEGF-D concentrations (n=12) (data not shown).…”

Section: Resultsmentioning

confidence: 99%

Serum VEGF-D concentration as a biomarker of lymphangioleiomyomatosis severity and treatment response: a prospective analysis of the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES) trial

Young

Lee

Inoue

et al. 2013

The Lancet Respiratory Medicine

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Summary Background VEGF-D is a lymphangiogenic growth factor that has a key role in tumour metastasis. Serum VEGF-D concentrations are increased in most patients with lymphangioleiomyomatosis, a rare neoplasm associated with mTOR-activating tuberous sclerosis gene mutations, lymphadenopathy, metastatic spread, and pulmonary cyst formation. We used data from the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES) trial to assess the usefulness of serum VEGF-D concentration as a marker of severity and therapeutic response to sirolimus in patients with lymphangioleiomyomatosis. Methods In the MILES trial, patients with lymphangioleiomyomatosis who had forced expiratory volume in 1 second (FEV1) of 70% or less of predicted were randomly assigned (1:1) to 12 months masked treatment with sirolimus or placebo. Serum VEGF-D concentrations were measured at baseline, 6 months, and 12 months. We used a linear regression model to assess associations of baseline VEGF-D concentrations with markers of disease severity, and a linear mixed effects model to assess the associations of VEGF-D concentrations with between-group differences in clinical, physiological, and patient-reported outcomes. Findings We included 42 patients from the placebo group and 45 from the sirolimus group in our analysis. Baseline VEGF-D concentrations in individual patients varied from 0·34 ng/mL to 16·7 ng/mL. Baseline VEGF-D concentrations were higher in patients who needed supplemental oxygen than in those who did not need supplemental oxygen (1·7 ng/mL [IQR 0·99–3·36] vs 0·84 ng/mL [0·52–1·39]; p<0·0001) and in those who had a bronchodilator response than in those who did not (2·01 ng/mL [0·99–2·86] vs 1·00 ng/mL [0·61–2·15]; 0·0273). Median serum VEGF-D concentrations were similar at baseline in the sirolimus and placebo groups, and fell from baseline at 6 and 12 months in the sirolimus group but remained roughly stable in the placebo group. Each one-unit increase in baseline log(VEGF-D) was associated with a between-group difference in baseline-to-12-month FEV1 change of 134 mL (p=0·0007). In the sirolimus group, improvement in baseline-to-12-month FEV1 occurred in 15 of 23 (65%) VEGF-D responders (ie, those in whom baseline-to-12-month VEGF-D concentrations decreased by more than they did in any patients in the placebo group) and four of 15 (27%) VEGF-D non-responders (p=0·0448). Interpretation Serum VEGF-D is a biologically plausible and useful biomarker in lymphangioleiomyomatosis that correlates with disease severity and treatment response. Measurement of serum VEGF-D concentrations could inform the risk–benefit analysis of sirolimus therapy in patients with lymphangioleiomyomatosis and reduce the numbers of patients needed for clinical trials.

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“…The SGRQ is a self‐administered questionnaire for assessing HRQoL in respiratory diseases, which was previously validated . It contains three domains, all scored from 0 to 100.…”

Section: Methodsmentioning

confidence: 99%

“…The St George’s Respiratory Questionnaire (SGRQ) was originally developed for patients with chronic obstructive pulmonary disease (COPD), and it has become one of the most extensively used tools for assessing HRQoL in patients with chronic respiratory diseases, including lymphangioleiomyomatosis, cystic fibrosis and idiopathic pulmonary fibrosis (IPF) . Few studies have assessed the performance of the SGRQ in patients with CTD‐ILD .…”

Section: Introductionmentioning

confidence: 99%

Performance of the St George’s Respiratory Questionnaire in patients with connective tissue disease‐associated interstitial lung disease

et al. 2018

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St. George's Respiratory Questionnaire Has Longitudinal Construct Validity in Lymphangioleiomyomatosis

Cited by 17 publications

References 20 publications

Pulmonary rehabilitation in lymphangioleiomyomatosis: a controlled clinical trial

Pulmonary rehabilitation in lymphangioleiomyomatosis: a controlled clinical trial

Serum VEGF-D concentration as a biomarker of lymphangioleiomyomatosis severity and treatment response: a prospective analysis of the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES) trial

Performance of the St George’s Respiratory Questionnaire in patients with connective tissue disease‐associated interstitial lung disease

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