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. 2017 Mar 2;4(2):124-131.
doi: 10.15326/jcopdf.4.2.2017.0130.

Responder Analyses for Treatment Effects in COPD Using the St George's Respiratory Questionnaire

Paul W Jones  1   2 , Heather Gelhorn  3 , Hilary Wilson  3 , Niklas Karlsson  4 , Shailendra Menjoge  5 , Hana Müllerova  2 , Stephen I Rennard  6   7 , Ruth Tal-Singer  8 , Debora Merrill  9 , Maggie Tabberer  2
Affiliations

Responder Analyses for Treatment Effects in COPD Using the St George's Respiratory Questionnaire

Paul W Jones et al. Chronic Obstr Pulm Dis. .

Abstract

Background: Patient-reported outcomes data in clinical trials are usually reported as mean values, interpreted in comparison to a minimum clinically important difference (MCID) and ignoring the possibility of a sizable proportion of patients experiencing a worthwhile benefit when the majority did not. This analysis tested the reliability of calculated responder rates (from chronic obstructive pulmonary disease [COPD] patients) with the St George's Respiratory Questionnaire (SGRQ) using a range of responder cut-points above and below the MCID (4 units). Methods: Individual patient data (i.e., data from long-acting bronchodilator [LAB] and inhaled corticosteroids [ICS]/long-acting beta2-agonist [LABA] randomized clinical studies) in the COPD Biomarker Qualification Consortium database were used: short-term (≤1-year duration; 14,814 patients,) and medium-term (2-4 years; 12,043 patients). Responder rates versus placebo across SGRQ score change thresholds ranging from -1.5 to -8.0 were tested; differences were expressed as the odds ratio (OR) of a patient exceeding the threshold versus no change or deterioration. Results: The ORs measuring benefit of active treatment were similar across thresholds in short-term studies (LAB, ORs 1.40-1.42; LABA/ICS, 1.50-1.56) and medium-term LAB studies (ORs 1.34-1.43), whereas ORs in medium-term studies with LABA/ICS intervention showed a trend for higher response rates at higher values of threshold cut-points (1.64-1.79). In short-term studies, different thresholds had little effect on the OR between active drugs versus a trend for lower ORs with lower thresholds in medium-term studies. Conclusions: The OR for a treatment effect compared with placebo appears consistent across a range of responder cut-points. In medium-term trials, the treatment difference between active drugs suggests that use of a lower threshold would not increase the odds of observing a measured treatment difference.

Keywords: CBQC; COPD Biomarkers Qualification Consortium; SGRQ; SGRQ threshold; St George’s Respiratory Questionnaire; forced expiratory volume in 1 second; responder rates.

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Conflict of interest statement

PWJ, NK, SM, HM, SIR, RTS and MT are employees of the pharma companies who funded this analysis. KR is a former employee of Pfizer. HG and HW participated in this project as employees of Evidera, a company which performs work for hire for multiple pharmaceutical and device companies in outcomes research. DM has nothing to declare.

References

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