ERS statement: a core outcome set for clinical trials evaluating the management of COPD exacerbations

Abstract

Clinical trials evaluating the management of acute exacerbations of COPD assess heterogeneous outcomes, often omitting those that are clinically relevant or more important to patients. We have developed a core outcome set, a consensus-based minimum set of important outcomes that we recommend are evaluated in all future clinical trials on exacerbations management, to improve their quality and comparability. COPD exacerbations outcomes were identified through methodological systematic reviews and qualitative interviews with 86 patients from 11 countries globally. The most critical outcomes were prioritized for inclusion in the core outcome set through a two-round Delphi survey that was completed by 1,063 participants (256 patients, 488 health professionals and 319 clinical academics) from 88 countries in 5 continents. Two global, multi-stakeholder, virtual consensus meetings were conducted to (i) finalize the core outcome set and (ii) prioritize a single measurement instrument to be used for evaluating each of the prioritized outcomes. Consensus was informed by rigorous methodological systematic reviews. The views of patients with COPD were accounted for in all stages of the project. Survival, treatment success, breathlessness, quality of life, activities of daily living, need for higher level of care, arterial blood gases, disease progression, future exacerbations and hospital admissions, treatment safety and adherence were all included in the core outcome set. Focused methodological research was recommended to further validate and optimize some of the selected measurement instruments. The panel did not consider the prioritized set of outcomes and associated measurement instruments burdensome for patients and health professionals to use.

Assessing Treatment Success or Failure as an Outcome in Randomised Clinical Trials of COPD Exacerbations. A Meta-Epidemiological Study

“…While this finding is indirect and based on a small number of observations, it may reflect a limited sensitivity of these instruments. Finally, the ERS COPD exacerbations core outcome set recommends that most of the components of these composite outcomes should be assessed as independent outcomes, thus providing additional granularity in the trial results, while in parallel limiting the utility of composite instruments assessing treatment success or failure [ 12 ].…”

“…In the absence of adequate data to select an optimal measurement instrument for assessing the cure of an exacerbation, the ERS COPD exacerbations core outcome set panel recommended an interim instrument for evaluating this outcome based on the evidence-informed consensus among the panel members and participating patient representatives. It is recommended that treatment success (cure) should be defined as the sufficient improvement of the signs and symptoms of the exacerbation such that no additional systemic treatments (systemic corticosteroids or antibiotics) are required [ 12 ]. This instrument aligns with the definitions of COPD exacerbations severity and is a practical outcome that is routinely considered in daily clinical practice and often used in trials.…”

“…Another major strength is the thorough analysis of the instruments used to assess treatment failure, the timepoints at which they were evaluated, and the results they yielded. Finally, the last part of this manuscript was informed by the consensus discussions of a multi-stakeholder panel with a global representation, described in the main task force report [ 12 ].…”

“…It is crucial that trials evaluating the management of a disease entity assess the same outcomes, those important to patients and health professionals, to facilitate decision making and improve the comparability of the trial results [ 10 ]. For this reason, the European Respiratory Society (ERS) launched a task force that developed a core outcome set for RCTs on the management of COPD exacerbations [ 11 , 12 ]. A core outcome set is an agreed standardised set of critically important outcomes that should be measured and reported as a minimum in all clinical trials in specific areas of health and health care [ 10 ].…”

“…The assessment of the overall outcome of the exacerbation (treatment success/failure, or cure) was selected as a core outcome, to be assessed in all future RCTs [ 12 ]. Moreover, a recent methodological systematic review revealed that this outcome is the second most frequently evaluated outcome in therapeutic trials of COPD exacerbations [ 9 ].…”