THE NUMBER NEEDED TO TREAT WITH DUPILUMAB TO PREVENT MODERATE OR SEVERE CHRONIC OBSTRUCTIVE PULMONARY DISEASE EXACERBATIONS AND IMPROVE SYMPTOMS: BOREAS AND NOTUS POST HOC ANALYSIS
Author(s)
Surya P Bhatt, MD1, Nicola A. Hanania, MD, MS2, Min Zhang, MD, PhD3, Changming Xia, PhD4, Ernesto Mayen Herrera, BSc, MSc5, Vijay Alagappan, PhD4, Fernando Martinez, MD, MS6;
1University of Alabama at Birmingham, Birmingham, AL, USA, 2Baylor College of Medicine, Houston, TX, USA, 3Shanghai General Hospital, Shanghai Jiao Tong University, School of Medicine, Shanghai, China, 4Regeneron Pharmaceuticals Inc., Tarrytown, NY, USA, 5Sanofi, Morristown, NJ, USA, 6University of Massachusetts Chan, Worcester, MA, USA
1University of Alabama at Birmingham, Birmingham, AL, USA, 2Baylor College of Medicine, Houston, TX, USA, 3Shanghai General Hospital, Shanghai Jiao Tong University, School of Medicine, Shanghai, China, 4Regeneron Pharmaceuticals Inc., Tarrytown, NY, USA, 5Sanofi, Morristown, NJ, USA, 6University of Massachusetts Chan, Worcester, MA, USA
OBJECTIVES: The number needed to treat (NNT) is a simple and intuitive statistical measure that quantifies treatment benefits for individual patients. In BOREAS and NOTUS, dupilumab reduced exacerbations and improved lung function, quality of life and symptoms in patients with chronic obstructive pulmonary disease (COPD) and type 2 inflammation. This post hoc analysis aimed to estimate the dupilumab NNT to prevent a moderate or severe COPD exacerbation, and the NNT to have 1 responder achieving change in Evaluating Respiratory Symptoms in COPD (E-RS:COPD) total score ≥2 points, using pooled data BOREAS and NOTUS data.
METHODS: BOREAS (NCT03930732) and NOTUS (NCT04456673), phase 3, randomized, double-blind, placebo-controlled trials, enrolled patients with COPD, moderate-to-severe airflow limitation, and type 2 inflammation (screening blood eosinophils ≥300 cells/µL) on triple therapy. Patients received add-on dupilumab 300 mg q2w or placebo for 52 weeks. The risk difference (95% CI) for adjusted annualized moderate or severe exacerbation rates, and the proportion of patients with an E-RS:COPD total score improvement of ≥2 points at Week 52 were derived as the ratio between dupilumab and placebo for each outcome; NNT (95% CI) was 1/risk difference.
RESULTS: In pooled ITT populations, baseline demographic and disease characteristics were broadly similar across placebo and dupilumab groups. Dupilumab vs placebo reduced annualized moderate or severe exacerbation rate (relative risk [95% CI]: 0.69 [0.59, 0.79]; P<0.001); risk difference [95% CI] was -0.36 [-0.51, -0.22] and NNT [95% CI] was 2.7 [2.0, 4.6]). A greater proportion of patients on dupilumab achieved an E-RS:COPD total score improvement of ≥2 points at Week 52 vs placebo (48.0% vs 38.3%; risk difference [95% CI] 0.09 [0.03, 0.16]; NNT [95% CI] 10.9 [6.3, 40.5]).
CONCLUSIONS: Add-on dupilumab prevented 1 moderate or severe exacerbation for every 3 treated patients, and 11 patients required treatment for 1 patient to improve E-RS:COPD total scores by ≥2 points.
METHODS: BOREAS (NCT03930732) and NOTUS (NCT04456673), phase 3, randomized, double-blind, placebo-controlled trials, enrolled patients with COPD, moderate-to-severe airflow limitation, and type 2 inflammation (screening blood eosinophils ≥300 cells/µL) on triple therapy. Patients received add-on dupilumab 300 mg q2w or placebo for 52 weeks. The risk difference (95% CI) for adjusted annualized moderate or severe exacerbation rates, and the proportion of patients with an E-RS:COPD total score improvement of ≥2 points at Week 52 were derived as the ratio between dupilumab and placebo for each outcome; NNT (95% CI) was 1/risk difference.
RESULTS: In pooled ITT populations, baseline demographic and disease characteristics were broadly similar across placebo and dupilumab groups. Dupilumab vs placebo reduced annualized moderate or severe exacerbation rate (relative risk [95% CI]: 0.69 [0.59, 0.79]; P<0.001); risk difference [95% CI] was -0.36 [-0.51, -0.22] and NNT [95% CI] was 2.7 [2.0, 4.6]). A greater proportion of patients on dupilumab achieved an E-RS:COPD total score improvement of ≥2 points at Week 52 vs placebo (48.0% vs 38.3%; risk difference [95% CI] 0.09 [0.03, 0.16]; NNT [95% CI] 10.9 [6.3, 40.5]).
CONCLUSIONS: Add-on dupilumab prevented 1 moderate or severe exacerbation for every 3 treated patients, and 11 patients required treatment for 1 patient to improve E-RS:COPD total scores by ≥2 points.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
CO40
Topic
Clinical Outcomes
Topic Subcategory
Clinician Reported Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas