&lt;p&gt;Minimal Clinically Important Differences for Patient-Reported Outcome Measures of Cough and Sputum in Patients with COPD&lt;/p&gt;

Rebelo, Patrícia; Oliveira, Ana; Paixão, Cátia; Valente, Carla; Andrade, Lília; Marques, Alda

doi:10.2147/copd.s219480

Cited by 26 publications

(33 citation statements)

References 70 publications

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“…The increases in LCQ score in the current study (1.09 and 1.53 points versus placebo at 200 and 750 mg, respectively) are close to the minimal clinically important difference for this measure, generally reported as 1.3 points [ 32 – 34 ] (although higher values have been suggested [ 33 ]). These results should be viewed with caution because the LCQ is a validated assessment of the impact of cough on quality of life during the preceding 14 days rather than the 1-week duration of treatment at each dose here, which may be too short to see substantial changes in quality of life.…”

Section: Discussionsupporting

confidence: 79%

Eliapixant (BAY 1817080), a P2X3 receptor antagonist, in refractory chronic cough: a randomised, placebo-controlled, crossover phase 2a study

et al. 2021

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ATP acting via P2X3 receptors is an important mediator of refractory chronic cough (RCC). This phase 2a double-blinded crossover study assessed the safety, tolerability and efficacy of eliapixant (BAY 1817080), a selective P2X3 receptor antagonist, in adults with RCC attending specialist centres.In period A, patients received placebo for 2 weeks then eliapixant 10 mg for 1 week. In period B, patients received eliapixant 50, 200 and 750 mg twice daily for 1 week per dose level. Patients were randomised 1:1 to period A–B (n=20) or B–A (n=20). The primary efficacy endpoint was change in cough frequency assessed over 24 h (VitaloJAK). Primary safety endpoint was frequency and severity of adverse events (AEs).Thirty-seven patients completed randomised therapy. Mean cough frequency fell by 17.4% versus baseline with placebo. Eliapixant reduced cough frequency at doses ≥50 mg (reduction versus placebo at 750 mg, 25%: 90% confidence interval, 11.5–36.5%; p=0.002). Doses ≥50 mg also significantly reduced cough severity. AEs, mostly mild or moderate, were reported in 65% of patients with placebo and 41–49% receiving eliapixant. Cumulative rates of taste-related AEs were 3% with placebo and 5–21% with eliapixant: all were mild.Selective P2X3 antagonism with eliapixant significantly reduced cough frequency and severity, confirming this as a viable therapeutic pathway for RCC. Taste-related side-effects were lower at therapeutic doses than with the less selective P2X3 antagonist gefapixant. Selective P2X3 antagonism appears to be a novel therapeutic approach for RCC.

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Section: Discussionsupporting

confidence: 79%

Eliapixant (BAY 1817080), a P2X3 receptor antagonist, in refractory chronic cough: a randomised, placebo-controlled, crossover phase 2a study

et al. 2021

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“…The thresholds for worsening of the patient-reported outcomes, i.e. an increase in SGRQ total score ≥ 7, increase in SGRQ activity score ≥ 5, increase in SGRQ impact score ≥ 7, increase in SGRQ symptoms score ≥ 8, decrease in CASA-Q cough domains ≥ 11, decrease in SF-12 mental component summary (MCS) score ≥ 6, decrease in SF-12 physical component summary (PCS) score ≥ 5, decrease in EQ-5D index score ≥ 0.06 and decrease in EQ-5D VAS ≥ 8, were based on published estimates for minimal clinically important differences [ 14 – 18 ].…”

Section: Methodsmentioning

confidence: 99%

Associations between resources and practices of ILD centers and outcomes in patients with idiopathic pulmonary fibrosis: data from the IPF-PRO Registry

et al. 2022

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Background Performance benchmarks for the management of idiopathic pulmonary fibrosis (IPF) have not been established. We used data from the IPF-PRO Registry, an observational registry of patients with IPF managed at sites across the US, to examine associations between the characteristics of the enrolling sites and patient outcomes. Methods An online survey was used to collect information on the resources, operations, and self-assessment practices of IPF-PRO Registry sites that enrolled ≥ 10 patients. Site variability in 1-year event rates of clinically relevant outcomes, including death, death or lung transplant, and hospitalization, was assessed. Models were adjusted for differences in patient case mix by adjusting for known predictors of each outcome. We assessed whether site-level heterogeneity existed for each patient-level outcome, and if so, we investigated potential drivers of the heterogeneity. Results All 27 sites that enrolled ≥ 10 patients returned the questionnaire. Most sites were actively following > 100 patients with IPF (70.4%), had a lung transplant program (66.7%), and had a dedicated ILD nurse leader (77.8%). Substantial heterogeneity was observed in the event rates of clinically relevant outcomes across the sites. After controlling for patient case mix, there were no outcomes for which the site variance component was significantly different from 0, but the p-value for hospitalization was 0.052. Starting/completing an ILD-related quality improvement project in the previous 2 years was associated with a lower risk of hospitalization (HR 0.60 [95% CI 0.44, 0.82]; p = 0.001). Conclusions Analyses of data from patients with IPF managed at sites across the US found no site-specific characteristics or practices that were significantly associated with clinically relevant outcomes after adjusting for patient case mix. Trial registration ClinicalTrials.gov, NCT01915511. Registered 5 August 2013, https://clinicaltrials.gov/ct2/show/NCT01915511

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“…The patient-reported outcomes assessed were the St. George's Respiratory Questionnaire (SGRQ) [6], the Cough and Sputum Assessment Questionnaire (CASA-Q) [7], the Short Form-12 (SF-12) questionnaire [8] and the EuroQoL 5-D (EQ-5D) index score and visual analog scale (VAS) [9]. The proportions of patients who had categorical changes in these patient-reported outcomes that reflected minimal clinically important differences for worsening proposed based on previous studies, i.e., an increase in SGRQ total score ≥7, increase in SGRQ activity score ≥5, increase in SGRQ impact score ≥7, increase in SGRQ symptoms score ≥8, decrease in CASA-Q cough domains ≥11, decrease in EQ-5D index score ≥0.06, decrease in EQ-5D VAS ≥8, decrease in SF-12 mental component summary (MCS) score ≥6, and decrease in SF-12 physical component summary (PCS) score ≥5, were assessed [10][11][12][13][14]. Associations between implementation score and categorical changes in FVC, DLco and patient-reported outcomes were analyzed using logistic regression models.…”

Section: Methodsmentioning

confidence: 99%

Implementation of guideline recommendations and outcomes in patients with idiopathic pulmonary fibrosis: Data from the IPF-PRO registry

Andrade

Kulkarni

Neely

et al. 2021

Respiratory Medicine

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Background: Few data are available on the extent to which clinical practice is aligned with international guidelines for the management of idiopathic pulmonary fibrosis (IPF). We investigated the extent to which management guidelines for IPF have been implemented in the US IPF-PRO Registry and associations between implementation of guidelines and clinical outcomes. Methods: We assessed the implementation of eight recommendations in clinical practice guidelines within the 6 months after enrollment: visit to a specialized clinic; pulmonary function testing; use of oxygen in patients with resting hypoxemia and exercise-induced hypoxemia; referral for pulmonary rehabilitation; treatment of gastroesophageal reflux disease; initiation of anti-fibrotic therapy; referral for lung transplant evaluation. An implementation score was calculated as the number of recommendations achieved divided by the number for which the patient was eligible. Associations between implementation score and outcomes were analyzed using logistic regression and Cox proportional hazards models. Results: Among 727 patients, median (Q1, Q3) implementation score was 0.6 (0.5, 0.8). Patients with an implementation score >0.6 had greater disease severity than those with a lower score. Implementation was lowest for referral for pulmonary rehabilitation (19.5%) and lung transplant evaluation (22.3%). In unadjusted models, patients with higher implementation scores had a greater risk of death, death or lung transplant, and hospitalization, but no significant associations were observed in adjusted models. Conclusions: Management guidelines were more likely to be implemented in patients with IPF with greater disease severity. When adjusted for disease severity, no association was found between implementation of management guidelines and clinical outcomes.

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<p>Minimal Clinically Important Differences for Patient-Reported Outcome Measures of Cough and Sputum in Patients with COPD</p>

Cited by 26 publications

References 70 publications

Eliapixant (BAY 1817080), a P2X3 receptor antagonist, in refractory chronic cough: a randomised, placebo-controlled, crossover phase 2a study

Eliapixant (BAY 1817080), a P2X3 receptor antagonist, in refractory chronic cough: a randomised, placebo-controlled, crossover phase 2a study

Associations between resources and practices of ILD centers and outcomes in patients with idiopathic pulmonary fibrosis: data from the IPF-PRO Registry

Implementation of guideline recommendations and outcomes in patients with idiopathic pulmonary fibrosis: Data from the IPF-PRO registry

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