Quantifying patient centered outcomes associated with the use of bilateral endobronchial coil treatment in patients with severe emphysema

Abstract

Objective: To determine the impact of endobronchial coils on health-related quality-of-life (HRQoL). This paper utilizes trial data to identify the predictors of HRQoL in patients with severe emphysema, and subsequently estimates the impact of a new treatment on HRQoL (measured by utilities). These utility estimates are used to generate indicative long-term QALY estimates for a range of clinically plausible scenarios as a precursor to cost-effectiveness analyses.

Methods: Patient level HRQoL data from RENEW and the National Emphysema Treatment Trial (NETT) were combined and mapped to generic EuroQol 5-dimension health utility questionnaire (EQ-5D) values using a published algorithm. Multilevel statistical models were developed using treatment, time, response, and baseline characteristics (EQ-5D, age, gender, FEV1, lung RV) to predict EQ-5D over time. Lifetime QALY estimates were generated using published survival data from NETT (assuming no impact of treatment on mortality) and four clinically plausible response profiles. Each response profile was combined with assumptions around treatment impact (constant or time varying).

Results: After controlling for baseline characteristics, both treatment and response had a statistically significant impact (p < .001) on utility (+0.101 and +0.061, respectively). When combined with selected baseline characteristics and time, Coils and Standard of Care (SoC) generated more QALYs than SoC alone in all scenarios, with incremental lifetime benefit ranging from 0.29–0.55 QALYs.

Conclusions: Coils and SoC resulted in statistically significant improvements in HRQoL compared to SoC alone in patients with severe emphysema.

Keywords:

• Emphysema
• bronchoscopy
• health-related quality-of-life
• pulmonary disease

Transparency

Declaration of funding

The original work that forms this manuscript and the writing of this manuscript was funded by PneumRx Ltd, a BTG International group company.

Declaration of financial/other relationships

G.D. declares consultancy fees from BTG International and PneumRx Ltd. R.E. declares speakers’ honoraria and travel expenses from PneumRx Ltd and BTG International. R.B. is an employee of PneumRx Ltd. D.J.S. acts as a physician advisor to and declares reimbursement for clinical trial trials, devices for treatments, travel expenses, speakers’ fees from PneumRx Ltd and BTG International. M.M. declares device reimbursement from PneumRx. D.S. declares bronchiectasis Interest Group meeting support from AstraZeneca and Forest Labs, bronchiectasis analysis support from Novartis, speakers’ and advisory board fees from Bayer, advisory board involvement on bronchiectasis from Novartis, and travel bursary from Chiesi. CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgments

None reported.