New England Journal of Medicine SEPTEMBER 25, 2014

Mepolizumab as Adjunctive Therapy in Patients with Severe Asthma (MENSA)

Ortega HG, Liu MC, Pavord ID, Brusselle GG, FitzGerald JM, Swales DC, et al.

Bottom Line

In this pivotal Phase III trial, both intravenous and subcutaneous mepolizumab significantly reduced the rate of clinically significant asthma exacerbations in patients with severe eosinophilic asthma compared to placebo.

Key Findings

1. Intravenous mepolizumab reduced the annualized rate of clinically significant asthma exacerbations by 47% (95% CI, 29–61; P<0.001) compared with placebo.
2. Subcutaneous mepolizumab reduced the annualized rate of clinically significant asthma exacerbations by 53% (95% CI, 37–65; P<0.001) compared with placebo.
3. Patients treated with mepolizumab demonstrated statistically significant improvements in pre-bronchodilator forced expiratory volume in 1 second (FEV1) and health-related quality of life, as measured by St. George's Respiratory Questionnaire (SGRQ) scores.
4. The overall incidence of adverse events was similar across the treatment groups, with 84% in the intravenous, 78% in the subcutaneous, and 83% in the placebo group.

Study Design

Design
RCT
Double-Blind
Sample
576
Patients
Duration
32 wk
Median
Setting
Multicenter, multinational
Population Patients with severe asthma, evidence of eosinophilic inflammation, and a history of recurrent exacerbations despite high-dose inhaled glucocorticoids.
Intervention Mepolizumab (75 mg IV or 100 mg SC) administered every 4 weeks.
Comparator Placebo administered every 4 weeks (double-dummy design).
Outcome Annualized rate of clinically significant asthma exacerbations.

Study Limitations

The 32-week study duration provides data on short-term efficacy and safety, but may not fully capture the long-term clinical benefits or potential late-emerging adverse effects of continuous IL-5 inhibition.
The study focused specifically on patients with evidence of eosinophilic inflammation, limiting the generalizability of findings to patients with severe asthma characterized by non-eosinophilic inflammatory pathways.
While statistically significant, the improvements in lung function (FEV1) were modest in absolute terms, highlighting the need to interpret these secondary outcomes in the context of clinical, rather than just statistical, significance.

Clinical Significance

The MENSA trial established mepolizumab as a targeted, effective add-on therapy for patients with severe refractory eosinophilic asthma. It validated the subcutaneous route of administration as a viable, effective alternative to the intravenous route, facilitating easier clinical use and supporting the broader move toward personalized, biomarker-driven management of severe asthma.

Historical Context

Prior to the MENSA trial, the DREAM study (Dose Ranging Efficacy and Safety with Mepolizumab) had demonstrated the clinical efficacy of intravenous mepolizumab in reducing exacerbations in patients with severe eosinophilic asthma. MENSA was designed as a pivotal Phase III trial to confirm these findings, bridge efficacy between intravenous and subcutaneous delivery, and secure regulatory approval, marking a major milestone in the development of monoclonal antibody therapies targeting the IL-5 cytokine pathway for severe asthma.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Mepolizumab is a monoclonal antibody that targets Interleukin-5 (IL-5). Based on the pathophysiology of severe asthma, what is the specific role of IL-5 in the inflammatory cascade, and why was the eosinophil count used as the primary biomarker in the MENSA trial?

Key Response

IL-5 is the major cytokine responsible for the growth, differentiation, recruitment, activation, and survival of eosinophils. In the subset of asthma known as 'eosinophilic asthma,' these cells drive airway inflammation and remodeling. The MENSA trial used blood eosinophil counts (≥150 cells/μL at screening or ≥300 cells/μL in the past year) as a surrogate biomarker to identify patients whose asthma is primarily driven by this IL-5-dependent pathway.

Resident
Resident

A 45-year-old patient with severe asthma experiences three exacerbations per year despite adherence to high-dose ICS-LABA. If their blood eosinophil count is 200 cells/μL, what specific clinical outcome demonstrated in the MENSA trial would justify adding mepolizumab to their regimen?

Key Response

The MENSA trial showed that mepolizumab (both IV and SC) reduced the rate of clinically significant asthma exacerbations by approximately 50% compared to placebo. It also showed significant improvements in the ACQ-5 (Asthma Control Questionnaire) scores and FEV1, providing a multi-faceted benefit for patients who remain uncontrolled on standard-of-care therapy.

Fellow
Fellow

The MENSA trial evaluated both intravenous (75mg) and subcutaneous (100mg) mepolizumab. Discuss the clinical implications of the finding that both routes yielded nearly identical reductions in exacerbation rates (~47% vs. 53%), particularly regarding the pharmacodynamics of IL-5 inhibition.

Key Response

The nearly identical efficacy suggests a 'ceiling effect' where the 100mg SC dose provides sufficient systemic exposure to maximally suppress IL-5 activity. For subspecialists, this indicates that while eosinophil suppression is dose-dependent at lower levels, the clinical benefit (exacerbation reduction) stabilizes once a threshold of IL-5 neutralization is reached, supporting the move toward the more convenient SC administration in clinical practice.

Attending
Attending

The MENSA trial demonstrated a significant reduction in exacerbations, yet many patients in the treatment arm continued to have some symptoms and a subset still experienced exacerbations. How should these findings shape our expectations and the 'treat-to-target' conversation with patients regarding asthma remission versus asthma control?

Key Response

While MENSA is a landmark trial for biologic efficacy, it highlights that anti-IL-5 therapy is not a 'cure.' It successfully addresses the eosinophilic component of the disease (reducing exacerbations), but it may not fully mitigate symptoms driven by airway hyperresponsiveness or non-eosinophilic pathways. This teaches us to use mepolizumab specifically to target the 'exacerbator' phenotype while maintaining other therapies to manage daily symptom burdens.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

Critique the use of the 'blood eosinophil count' as the primary inclusion criterion in MENSA. How might the inclusion of patients based on a single historical value (≥300 cells/μL in the past 12 months) vs. a current value (≥150 cells/μL) introduce phenotypical instability or regression to the mean in the trial results?

Key Response

Eosinophil counts can be highly labile and are suppressed by systemic corticosteroids, which many of these patients take for exacerbations. By allowing a historical high, the researchers ensured they captured patients with the 'potential' for eosinophilic inflammation. However, this risks including patients whose current symptoms might be driven by non-eosinophilic mechanisms, potentially diluting the observed effect size. Researchers must account for this 'phenotype switching' when analyzing longitudinal response to biologics.

Journal Editor
Journal Editor

The MENSA trial reported a significant reduction in exacerbations, but the placebo group also experienced a lower rate of exacerbations than seen in earlier mepolizumab studies (DREAM trial). As a reviewer, what factors in the study design or patient selection would you flag as potential causes for this improved placebo performance, and does it threaten the study's internal validity?

Key Response

A common 'trial effect' occurs where rigorous monitoring and improved adherence to background ICS/LABA in the placebo arm reduce exacerbation rates. Furthermore, MENSA excluded patients with very frequent recently-treated exacerbations or unstable dosing. While this might reduce the absolute difference between groups, the fact that mepolizumab still achieved a ~50% relative reduction despite an optimized placebo group actually strengthens the argument for its efficacy.

Guideline Committee
Guideline Committee

Based on the MENSA trial results, should mepolizumab be prioritized over other biologics (like omalizumab) for patients who qualify for both? How do these findings influence the GINA Step 5 recommendations for the management of difficult-to-treat and severe asthma?

Key Response

MENSA provided high-quality (Level A) evidence that led to the inclusion of anti-IL-5 therapies in GINA Step 5 for patients with a severe eosinophilic phenotype. While GINA does not explicitly prioritize one biologic over another, MENSA (along with the SIRIUS trial) suggests that in patients with high exacerbation rates and clear eosinophilia, mepolizumab offers a more direct mechanistic intervention than anti-IgE, particularly if the patient's IgE levels are low or if they lack a clear allergic trigger.

Clinical Landscape

Noteworthy Related Trials

2012

DREAM Trial

n = 621 · NEJM

Tested

Mepolizumab (75, 250, or 750 mg IV)

Population

Patients with uncontrolled severe asthma

Comparator

Placebo

Endpoint

Rate of clinically significant asthma exacerbations

Key result: Mepolizumab significantly reduced the rate of asthma exacerbations compared with placebo across all dose groups.
2016

SIROCCO Trial

n = 1,205 · Lancet

Tested

Benralizumab (30 mg SC every 4 or 8 weeks)

Population

Patients with severe, uncontrolled asthma with eosinophilic phenotype

Comparator

Placebo

Endpoint

Annual exacerbation rate

Key result: Benralizumab significantly reduced the annual asthma exacerbation rate and improved lung function in patients with high blood eosinophil counts.
2018

LIBERTY ASTHMA QUEST Trial

n = 1,902 · NEJM

Tested

Dupilumab (200 or 300 mg SC)

Population

Patients with uncontrolled, moderate-to-severe asthma

Comparator

Placebo

Endpoint

Annualized rate of severe asthma exacerbations and FEV1 change

Key result: Dupilumab significantly reduced severe asthma exacerbation rates and improved pre-bronchodilator FEV1 in patients with elevated eosinophil levels.

Tailored to your role

Want this tailored to you?

Add your specialty or training stage to get role-specific takeaways and more questions.

Personalize this analysis