The New England Journal of Medicine July 20, 2023

Dupilumab for COPD with Type 2 Inflammation Indicated by Eosinophil Counts

Surya P. Bhatt, Klaus F. Rabe, Nicola A. Hanania, Claus F. Vogelmeier, Jeremy Cole, Mona Bafadhel, et al.

Bottom Line

In the phase 3 BOREAS trial, patients with uncontrolled COPD and evidence of type 2 inflammation who received dupilumab experienced a significant reduction in moderate or severe exacerbations and meaningful improvements in lung function and quality of life compared to placebo.

Key Findings

1. The annualized rate of moderate or severe exacerbations was significantly lower with dupilumab (0.78) compared to placebo (1.10), representing a 30% relative reduction (rate ratio, 0.70; 95% CI, 0.58 to 0.86; P<0.001) [2.1.1].
2. Prebronchodilator forced expiratory volume in 1 second (FEV1) increased from baseline to week 12 by a least-squares mean of 160 mL with dupilumab versus 77 mL with placebo (mean difference, 83 mL; P<0.001).
3. The improvement in FEV1 was sustained through week 52, with an increase of 153 mL in the dupilumab group versus 70 mL in the placebo group.
4. Dupilumab significantly improved health-related quality of life, reducing the St. George's Respiratory Questionnaire (SGRQ) total score by an additional -3.4 points compared to placebo (P=0.002).

Study Design

Design
Randomized Controlled Trial
Double-Blind
Sample
939
Patients
Duration
52 wk
Median
Setting
Multicenter, Global
Population Current or former smokers (aged 40 to 80 years) with a diagnosis of COPD, a blood eosinophil count of ≥300 cells/μL, and an elevated risk of exacerbations despite receiving standard-of-care maximal inhaled therapy (triple therapy or equivalent).
Intervention Dupilumab 300 mg administered subcutaneously every 2 weeks.
Comparator Matching placebo administered subcutaneously every 2 weeks.
Outcome Annualized rate of moderate or severe COPD exacerbations over the 52-week treatment period.

Study Limitations

The trial population lacked broad ethnic diversity, potentially limiting the generalizability of the findings across different global populations [1.1.6].
The 52-week trial duration may be insufficient to fully assess the long-term safety profile and the durability of lung function preservation.
Efficacy was only demonstrated in a highly selected subgroup with a blood eosinophil count of ≥300 cells/μL; the results cannot be extrapolated to patients with noneosinophilic COPD or lower eosinophil thresholds.
Potential shifts in patient behaviors and exacerbation reporting during the COVID-19 pandemic may have influenced the baseline rates of exacerbations.

Clinical Significance

The BOREAS trial is a landmark study establishing dupilumab as the first targeted biologic to significantly reduce exacerbations and improve lung function in patients with severe, uncontrolled COPD. By defining a specific phenotype (eosinophils ≥300 cells/μL), it pioneers the application of precision medicine in COPD management.

Historical Context

For decades, the pharmacological management of COPD relied on bronchodilators and inhaled corticosteroids. Unlike in severe asthma, earlier trials of eosinophil-depleting biologics (such as anti-IL-5 agents mepolizumab and benralizumab) in COPD yielded inconsistent or negative results. The BOREAS trial represented a paradigm shift by demonstrating that dual blockade of the IL-4 and IL-13 pathways with dupilumab is highly effective in a well-selected, eosinophilic COPD phenotype.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the mechanism of dupilumab target the specific pathophysiology of the COPD patient subset evaluated in the BOREAS trial, and how does this differ from the traditional understanding of COPD inflammation?

Key Response

Dupilumab is a monoclonal antibody that binds to the IL-4R alpha subunit, blocking the signaling of both IL-4 and IL-13. This targets Type 2 (eosinophilic) inflammation, which drives a specific, highly exacerbation-prone phenotype of COPD. Traditionally, COPD has been viewed as a disease of neutrophilic inflammation driven by macrophages and CD8+ T-cells in response to noxious stimuli like cigarette smoke, making the Type 2 subset a distinct therapeutic target.

Resident
Resident

Based on the BOREAS trial inclusion criteria, which specific clinical phenotype of COPD patients in your clinic would be appropriate candidates for initiating dupilumab therapy?

Key Response

Appropriate candidates are those with uncontrolled COPD despite maximal inhaled therapy (typically triple therapy: ICS/LAMA/LABA) who have evidence of Type 2 inflammation, specifically indicated by a peripheral blood eosinophil count of at least 300 cells per microliter and a history of frequent moderate-to-severe exacerbations.

Fellow
Fellow

Previous trials of anti-IL-5 biologics like mepolizumab and benralizumab in eosinophilic COPD yielded mixed results regarding lung function, yet dupilumab (anti-IL-4R alpha) showed robust efficacy in BOREAS. What mechanistic differences between these pathways might explain dupilumab's superior impact on FEV1 and symptoms?

Key Response

Anti-IL-5 biologics primarily deplete eosinophils. In contrast, blocking IL-4 and IL-13 with dupilumab not only reduces eosinophil trafficking but also directly inhibits goblet cell hyperplasia, mucus hypersecretion, and airway smooth muscle contractility. This broader suppression of the Type 2 inflammatory cascade and epithelial remodeling likely explains the significant improvements in FEV1 and quality of life seen in BOREAS, which were largely absent or blunted in the IL-5 trials.

Attending
Attending

The BOREAS trial introduces a highly targeted biologic for a specific COPD endotype. How does transitioning from a generalized bronchodilator approach to a 'treatable traits' endotyping model reshape our long-term management strategy and cost-benefit discussions for severe COPD?

Key Response

This represents a paradigm shift in COPD toward precision medicine. Attendings must now routinely phenotype severe COPD patients using biomarkers (like eosinophils) to identify treatable traits such as Type 2 inflammation. This shifts management from endlessly escalating inhaled therapies or relying on chronic systemic steroids to identifying biologic candidates, which requires complex risk-benefit and cost-efficacy discussions given the high cost of monoclonal antibodies versus the morbidity and healthcare utilization of severe exacerbations.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The BOREAS trial utilized a peripheral blood eosinophil threshold of 300 cells per microliter to define Type 2 inflammation. From a biomarker validation and trial design perspective, what are the limitations of using a single peripheral blood metric as a surrogate for airway inflammation, and how might dynamic or composite biomarker tracking have altered the trial's effect size?

Key Response

Peripheral eosinophil counts can fluctuate significantly due to concurrent infections, oral corticosteroid bursts, or natural diurnal variability, potentially misclassifying endotypes at a single screening visit. A robust trial design might employ longitudinal eosinophil tracking, composite biomarkers (adding FeNO or sputum eosinophils), or transcriptomic profiling to ensure the targeted cohort possesses stable, biologic-responsive Type 2 airway inflammation, which would likely reduce noise and amplify the observed treatment effect size.

Journal Editor
Journal Editor

As a peer reviewer analyzing the BOREAS trial, how might the requirement for patients to be on background triple inhaled therapy (including ICS) confound the true efficacy of dupilumab, considering the known suppressive effects of ICS on Type 2 inflammation?

Key Response

Patients were required to be on maximal therapy, often including high-dose ICS. Since ICS partially suppresses Type 2 inflammation, a baseline eosinophil count of 300+ in these patients indicates a highly refractory subgroup, which is appropriate for a phase 3 biologic trial. However, a tough reviewer would flag that we do not know dupilumab's efficacy in ICS-naïve patients or whether dupilumab could entirely replace ICS in this phenotype, leaving critical questions about the synergistic versus independent effects of these immunomodulators.

Guideline Committee
Guideline Committee

Current GOLD guidelines recommend triple therapy (ICS/LAMA/LABA) for Group E patients with recurrent exacerbations and elevated eosinophils. How does the evidence from the BOREAS trial justify integrating dupilumab into the GOLD treatment algorithm, and what specific evidence grade and placement would you recommend?

Key Response

BOREAS provides high-quality, randomized controlled phase 3 evidence (Level A) that dupilumab reduces exacerbations and improves lung function in patients already maximized on triple therapy with eosinophils >= 300. The committee should consider placing dupilumab as a strong recommendation for add-on therapy in GOLD Group E patients who remain uncontrolled on LAMA/LABA/ICS with a documented Type 2 phenotype, marking the first time a targeted biologic receives a dedicated pathway in major COPD guidelines.

Clinical Landscape

Noteworthy Related Trials

2017

METREX Trial

n = 836 · NEJM

Tested

Mepolizumab 100 mg subcutaneously every 4 weeks

Population

COPD patients with an eosinophilic phenotype and history of exacerbations

Comparator

Placebo

Endpoint

Annual rate of moderate or severe exacerbations

Key result: Mepolizumab resulted in a lower annualized rate of moderate or severe exacerbations than placebo among patients with an eosinophilic phenotype.
2019

GALATHEA Trial

n = 1,656 · NEJM

Tested

Benralizumab 30 mg or 100 mg subcutaneously

Population

COPD patients with moderate to very severe airflow limitation and exacerbation history

Comparator

Placebo

Endpoint

Annualized COPD exacerbation rate

Key result: Benralizumab did not result in a significantly lower annualized rate of COPD exacerbations than placebo.
2024

NOTUS Trial

n = 935 · NEJM

Tested

Dupilumab 300 mg every 2 weeks

Population

Patients with COPD and blood eosinophil count of at least 300 cells per microliter

Comparator

Placebo

Endpoint

Annualized rate of moderate or severe COPD exacerbations

Key result: Dupilumab significantly reduced the annualized rate of moderate or severe exacerbations by 34 percent compared to placebo.

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