Allergy, Asthma & Immunology Research JULY 15, 2024

LIBERTY ASTHMA TRAVERSE: Long-term safety and efficacy of dupilumab in patients with moderate-to-severe asthma

Maspero JF, Peters AT, Chapman KR, et al.

Bottom Line

The TRAVERSE open-label extension study demonstrated that long-term treatment with dupilumab in patients with moderate-to-severe asthma is well-tolerated and provides sustained improvements in asthma exacerbation rates, lung function, and asthma control.

Key Findings

1. Long-term treatment with dupilumab was well-tolerated, with a safety profile consistent with that observed in parent phase 3 clinical trials.
2. Dupilumab sustained significant reductions in the annualized severe asthma exacerbation rate (AER) over 96 weeks of treatment in the extension study.
3. Patients who previously received placebo in parent trials experienced rapid and sustained improvements in pre-bronchodilator FEV1 upon initiation of dupilumab in TRAVERSE.
4. Improvements in pre-bronchodilator FEV1 and asthma control (ACQ-5 scores) observed during parent studies were maintained throughout the duration of the TRAVERSE extension, with no apparent loss of treatment effect.
5. Blood eosinophil levels showed progressive decreases during the course of the study.

Study Design

Design
Open-Label Extension Study
Open-Label
Sample
2,282
Patients
Duration
96 wk
Median
Setting
Multinational, multicenter
Population Adolescents (≥12 years) and adults with moderate-to-severe asthma who completed a previous dupilumab asthma clinical trial.
Intervention Dupilumab 300 mg subcutaneous injection every 2 weeks.
Comparator None (single-arm open-label)
Outcome Incidence of treatment-emergent adverse events (TEAEs).

Study Limitations

The study design is open-label without a concurrent control arm during the extension period, which may introduce observer bias regarding patient-reported outcomes.
As an extension study, the population is comprised of patients who successfully completed prior clinical trials, potentially selecting for individuals with higher treatment adherence and better initial tolerance to the medication.
Long-term efficacy results may be subject to the healthy-survivor effect common in longitudinal extension trials.
The study evaluated patients from multiple prior parent trials with different inclusion criteria and baseline characteristics, introducing heterogeneity into the pooled long-term analysis.

Clinical Significance

The study provides essential long-term evidence for the safety and sustained clinical benefits of dupilumab, a monoclonal antibody targeting IL-4 and IL-13 receptors, supporting its use as a durable maintenance therapy for patients with uncontrolled, moderate-to-severe asthma with a type 2 inflammatory phenotype.

Historical Context

Dupilumab was developed to address unmet needs in patients with uncontrolled type 2 inflammatory asthma. Following successful phase 3 parent trials such as QUEST and VENTURE, which established efficacy over 24-52 weeks, the TRAVERSE open-label extension was necessary to characterize the safety and durability of the clinical response over extended periods (up to 96 weeks), fulfilling regulatory requirements for chronic disease management.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the primary molecular target of dupilumab, and how does its mechanism of action specifically address the pathophysiology of Type 2 (T2)-high asthma as observed in the TRAVERSE study population?

Key Response

Dupilumab is a fully human monoclonal antibody that binds to the IL-4 receptor alpha (IL-4Rα) subunit. This binding inhibits the signaling of both IL-4 and IL-13, two key cytokines in the Type 2 inflammatory pathway. By blocking these pathways, it reduces eosinophil recruitment, IgE production, and mucus hypersecretion, which are foundational to the airway inflammation and remodeling seen in moderate-to-severe asthma.

Resident
Resident

In the TRAVERSE study, transient blood eosinophilia was observed in some patients upon initiating dupilumab. How should a clinician manage this laboratory finding, and at what threshold should it prompt further investigation for hypereosinophilic syndromes?

Key Response

Transient eosinophilia is a known pharmacological effect of dupilumab, likely caused by the blockade of IL-4 and IL-13, which prevents eosinophil migration from the blood into the tissues. In most cases, this is asymptomatic and self-limiting. However, clinicians should monitor for clinical signs of eosinophilic granulomatosis with polyangiitis (EGPA) and typically consider further investigation if eosinophil counts exceed 1,500 cells/μL or if systemic symptoms develop.

Fellow
Fellow

The TRAVERSE study included patients from both the QUEST and VENTURE trials. How do the long-term FEV1 improvements seen in TRAVERSE inform our understanding of 'fixed airflow obstruction' versus 'reversible inflammation' in the severe asthma phenotype?

Key Response

The sustained improvement in FEV1 over 96 weeks suggests that dupilumab may do more than just provide temporary bronchodilation; it may modulate the underlying inflammatory processes that lead to airway remodeling. For fellows, this highlights the potential for biologics to alter the natural history of lung function decline, though the degree to which 'fixed' obstruction can be reversed remains a point of active investigation in T2-high populations.

Attending
Attending

Given that the TRAVERSE study demonstrated sustained efficacy for up to 96 weeks, what are the clinical implications for the concept of 'biologic disease remission' and the feasibility of attempting a 'biologic holiday' in patients who achieve stable control?

Key Response

While TRAVERSE shows the drug is safe and effective long-term, it does not provide evidence of a 'cure.' Most experts caution against 'biologic holidays' because the underlying genetic and immunological predisposition to Type 2 inflammation remains. Stopping the biologic often leads to a return of symptoms and a decline in lung function, suggesting that for severe asthma, dupilumab should be viewed as long-term maintenance therapy rather than an induction-to-remission bridge.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The TRAVERSE study is an open-label extension (OLE). Critically evaluate the 'healthy survivor effect' as a source of bias in this study design and how it might lead to an overestimation of long-term safety and efficacy compared to a hypothetical continuous randomized controlled trial.

Key Response

OLE designs inherently suffer from selection bias because patients who experienced severe adverse events or lack of efficacy in the parent trials (QUEST/VENTURE) are less likely to enroll in the extension. This 'healthy survivor' cohort may present a more optimistic view of the drug's profile. Researchers must use sensitivity analyses and compare baseline characteristics of the OLE population versus the original intention-to-treat (ITT) population to quantify this bias.

Journal Editor
Journal Editor

As a reviewer, how would you assess the validity of combining data from the 'placebo-to-dupilumab' group and the 'dupilumab-to-dupilumab' group in TRAVERSE, and what specific statistical adjustments would you require to ensure the 'catch-up' effect doesn't mask long-term waning of efficacy?

Key Response

A journal editor would be concerned with the 'switch' effect. The rapid improvement in the former placebo group (the 'catch-up') provides strong evidence of drug effect but complicates longitudinal trend analysis. A rigorous review would require separate analyses of these cohorts and possibly a longitudinal mixed-effects model to ensure that the rate of improvement or maintenance is consistent across different durations of exposure.

Guideline Committee
Guideline Committee

The TRAVERSE data confirms long-term safety and efficacy in patients with moderate-to-severe asthma regardless of baseline eosinophil count. How should this affect the GINA Step 5 recommendations regarding the timing of biologic initiation compared to the use of long-term oral corticosteroids (OCS)?

Key Response

Current GINA and NAEPP guidelines emphasize minimizing OCS use due to systemic toxicity. The TRAVERSE study, particularly the data from the former VENTURE (OCS-dependent) patients, reinforces the role of dupilumab as a potent OCS-sparing agent. This evidence supports a stronger recommendation to initiate biologics earlier in Step 5 (or even Step 4 in specific phenotypes) to prevent the cumulative morbidity associated with chronic OCS use, moving biologics from 'last resort' to 'preferred steroid-sparing' therapy.

Clinical Landscape

Noteworthy Related Trials

2016

LIBERTY ASTHMA DRI

n = 776 · Lancet

Tested

Dupilumab 200mg or 300mg every 2 weeks

Population

Patients with persistent, moderate-to-severe asthma

Comparator

Placebo

Endpoint

Change from baseline in pre-bronchodilator FEV1 at week 12

Key result: Dupilumab improved lung function and asthma control significantly, particularly in patients with elevated baseline blood eosinophil counts.
2018

LIBERTY ASTHMA QUEST

n = 1,902 · NEJM

Tested

Dupilumab 200mg or 300mg every 2 weeks

Population

Patients with uncontrolled, moderate-to-severe asthma

Comparator

Placebo

Endpoint

Annualized rate of severe asthma exacerbations

Key result: Dupilumab significantly reduced the rate of severe asthma exacerbations and improved pre-bronchodilator FEV1 compared to placebo.
2018

LIBERTY ASTHMA VENTURE

n = 210 · NEJM

Tested

Dupilumab 300mg every 2 weeks

Population

Patients with glucocorticoid-dependent severe asthma

Comparator

Placebo

Endpoint

Percentage reduction in daily oral glucocorticoid dose

Key result: Dupilumab treatment enabled significant reduction in oral glucocorticoid use and reduced exacerbation rates compared to placebo.

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