New England Journal of Medicine November 02, 2023

Perioperative Durvalumab for Resectable Non-Small-Cell Lung Cancer (AEGEAN)

John V. Heymach, David Harpole, Tetsuya Mitsudomi et al.

Bottom Line

In patients with resectable non-small-cell lung cancer, perioperative treatment consisting of neoadjuvant durvalumab plus chemotherapy followed by adjuvant durvalumab monotherapy significantly improved pathological complete response and event-free survival compared to neoadjuvant chemotherapy alone.

Key Findings

1. Pathological complete response (pCR) was significantly higher in the durvalumab arm compared to the placebo arm (17.2% vs. 4.3%; absolute difference, 13.0 percentage points; 95% CI, 8.7 to 17.6; P<0.001) [1.2.1].
2. Event-free survival (EFS) was significantly prolonged in the durvalumab group (hazard ratio for progression, recurrence, or death 0.68; 95% CI, 0.53 to 0.88; P=0.004).
3. At the 12-month landmark analysis, EFS was observed in 73.4% of patients receiving durvalumab (95% CI, 67.9 to 78.1) versus 64.5% of patients receiving placebo (95% CI, 58.8 to 69.6).
4. The addition of durvalumab did not negatively impact surgical feasibility, with 77.6% of patients in the durvalumab arm and 76.7% in the placebo arm completing surgery.
5. Grade 3 or 4 maximum adverse events occurred at similar rates between the two groups (42.4% with durvalumab and 43.2% with placebo), indicating that adding durvalumab did not substantially increase major toxicities.

Study Design

Design
Randomized Controlled Trial
Double-Blind
Sample
802
Patients
Duration
11.7 mo
Median
Setting
Multicenter, International
Population Adult patients with treatment-naive, resectable stage II to IIIB (N2) non-small-cell lung cancer (NSCLC) and an ECOG performance status of 0 or 1, irrespective of PD-L1 expression.
Intervention Durvalumab (1500 mg IV) plus platinum-based chemotherapy every 3 weeks for up to 4 cycles (neoadjuvant), followed by surgery, followed by durvalumab every 4 weeks for up to 12 cycles (adjuvant).
Comparator Placebo plus platinum-based chemotherapy every 3 weeks for up to 4 cycles (neoadjuvant), followed by surgery, followed by placebo every 4 weeks for up to 12 cycles (adjuvant).
Outcome Event-free survival (EFS) assessed by blinded independent central review and pathological complete response (pCR) evaluated by blinded central pathology review.

Study Limitations

The median event-free survival follow-up was relatively short (11.7 months) at the time of the interim analysis, leaving long-term overall survival outcomes immature [1.2.3].
The perioperative trial design does not allow for isolating the independent efficacy contribution of the adjuvant durvalumab phase versus the neoadjuvant chemoimmunotherapy phase.
A protocol amendment excluded patients with documented EGFR mutations or ALK rearrangements from the modified intention-to-treat efficacy analyses, limiting the applicability of these findings to oncogene-driven subgroups.
A notable proportion of randomized patients (around 22-23%) did not ultimately complete surgery, reflecting the real-world attrition often observed in neoadjuvant thoracic oncology trials.

Clinical Significance

The AEGEAN trial demonstrated that incorporating durvalumab both before and after surgery provides a clinically meaningful and statistically significant improvement in event-free survival and pathological complete response for patients with resectable early-stage NSCLC. This established a highly effective multi-modal treatment strategy that avoids excess surgical delays or toxicity, culminating in the FDA approval of the regimen in August 2024 for adults with resectable (tumors ≥ 4 cm and/or node positive) NSCLC with no known EGFR/ALK mutations.

Historical Context

Historically, surgical resection followed by adjuvant platinum-doublet chemotherapy was the standard of care for early-stage NSCLC, though 5-year survival rates remained suboptimal due to high rates of distant relapse. Following the success of immune checkpoint inhibitors in advanced disease, trials like CheckMate 816 validated neoadjuvant chemoimmunotherapy, while trials like IMpower010 and KEYNOTE-091 established the role of adjuvant immunotherapy. The phase 3 AEGEAN trial emerged alongside trials like KEYNOTE-671 to assess a comprehensive, continuous 'perioperative' approach—utilizing immunotherapy both in the neoadjuvant setting with chemotherapy and in the adjuvant setting as monotherapy. This reflects a modern paradigm shift toward intensifying systemic therapy in the curative-intent early-stage lung cancer setting.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the mechanism of action of durvalumab complement traditional cytotoxic chemotherapy in the neoadjuvant setting for NSCLC?

Key Response

Durvalumab is a monoclonal antibody that blocks PD-L1, preventing its interaction with PD-1 and releasing the inhibition of T-cell mediated immune responses. Cytotoxic chemotherapy causes tumor cell death and antigen release, which primes T-cells. Administering durvalumab when the tumor bulk and intact draining lymphatics are present maximizes the systemic immune response against these exposed neoantigens, a foundational concept in tumor immunology.

Resident
Resident

When evaluating a patient with newly diagnosed stage IIIA NSCLC, what clinical parameters and potential surgical complications must you consider before recommending a perioperative chemoimmunotherapy regimen like the one used in the AEGEAN trial?

Key Response

Residents must balance the benefit of improved pathological complete response against the risk of immune-related adverse events and surgical delays. Neoadjuvant immunotherapy can cause pneumonitis or severe inflammatory reactions that may complicate surgery (e.g., dense hilar fibrosis) or delay curative-intent resection. Assessment of PD-L1 status, EGFR/ALK mutations, and baseline pulmonary function is crucial.

Fellow
Fellow

The AEGEAN trial demonstrated improvements in both pCR and EFS. How do we interpret the discordance that sometimes occurs between pCR and long-term EFS in individual patients, and what biomarkers are currently being investigated to better predict which patients actually need the adjuvant durvalumab phase?

Key Response

While pCR is a strong surrogate for survival at the trial level, individual patients achieving pCR might still relapse, and those without pCR might be cured. Furthermore, giving 1 year of adjuvant durvalumab to a patient with a pCR might be overtreatment. Clearance of circulating tumor DNA (ctDNA) representing minimal residual disease is a critical biomarker currently being evaluated to guide escalation or de-escalation of adjuvant therapy.

Attending
Attending

With multiple recent trials showing benefit for neoadjuvant or perioperative immunotherapy in resectable NSCLC, how do you decide between a purely neoadjuvant approach versus a perioperative approach in your clinical practice?

Key Response

Attendings must navigate overlapping data without direct head-to-head comparisons. CheckMate 816 showed benefit with only neoadjuvant therapy, while AEGEAN adds up to a year of adjuvant durvalumab. The decision involves weighing the financial toxicity and potential for chronic immune-related adverse events of a year of immunotherapy against potential EFS benefits, often factoring in final surgical pathology and patient tolerance.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The AEGEAN trial utilized a dual primary endpoint of pCR and EFS. What are the statistical implications of using a surrogate endpoint alongside a clinical endpoint in a phase 3 trial, and how does missing data or informative censoring in the adjuvant phase complicate the EFS analysis?

Key Response

Alpha-spending strategies must strictly control type I error when dual endpoints are used. Furthermore, patients dropping out before the adjuvant phase due to toxicity or surgical complications introduces informative censoring. If not appropriately modeled using techniques like inverse probability weighting or competing risk analysis, this can severely bias Kaplan-Meier EFS estimates.

Journal Editor
Journal Editor

As an editor evaluating the AEGEAN manuscript, how would you scrutinize the contribution of the adjuvant durvalumab phase given that the control arm received placebo after surgery?

Key Response

A major methodological limitation of perioperative trial designs is the lack of a neoadjuvant-only control arm. The control arm gets neoadjuvant chemo, surgery, then placebo. The experimental gets neoadjuvant chemo plus IO, surgery, then IO. If EFS is improved, it is statistically impossible to isolate whether the adjuvant IO was necessary or if the neoadjuvant IO drove the entire benefit. Editors should demand explicit discussion of this confounding factor.

Guideline Committee
Guideline Committee

Based on the AEGEAN trial, should current guidelines be updated to mandate a perioperative approach over a purely neoadjuvant approach for resectable NSCLC, and what level of evidence supports the use of adjuvant IO in patients who achieve a pCR?

Key Response

Current NCCN guidelines include both neoadjuvant and perioperative IO combinations as Category 1 or 2A recommendations. The committee must grapple with the lack of comparative effectiveness research. Recommending against adjuvant IO in pCR patients lacks prospective validation, yet giving it incurs toxicity. Guidelines must carefully reflect the absence of trial designs that isolate the adjuvant phase benefit, keeping both options open rather than mandating the longer perioperative course.

Clinical Landscape

Noteworthy Related Trials

2021

IMpower010

n = 1005 · Lancet

Tested

Adjuvant atezolizumab following resection and chemotherapy

Population

Patients with completely resected stage IB-IIIA NSCLC

Comparator

Best supportive care

Endpoint

Disease-free survival (DFS)

Key result: Adjuvant atezolizumab showed a significant DFS benefit versus best supportive care after adjuvant chemotherapy, particularly in the PD-L1 TC >= 1% stage II-IIIA population.
2022

CheckMate 816

n = 358 · NEJM

Tested

Neoadjuvant nivolumab plus platinum-based chemotherapy

Population

Patients with resectable stage IB to IIIA NSCLC

Comparator

Platinum-based chemotherapy alone

Endpoint

Event-free survival (EFS) and pathological complete response (pCR)

Key result: Neoadjuvant nivolumab plus chemotherapy significantly increased the percentage of patients with a pCR (24.0% vs. 2.2%) and prolonged EFS (31.6 vs. 20.8 months) compared to chemotherapy alone.
2023

KEYNOTE-671

n = 797 · NEJM

Tested

Perioperative pembrolizumab (neoadjuvant with chemo plus adjuvant)

Population

Patients with resectable stage II to IIIB NSCLC

Comparator

Neoadjuvant chemotherapy plus placebo

Endpoint

Event-free survival (EFS) and overall survival (OS)

Key result: Perioperative pembrolizumab significantly improved EFS, pCR, and major pathological response rates compared to neoadjuvant chemotherapy alone, with a demonstrated overall survival benefit.

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