The New England Journal of Medicine MAY 31, 2018

Pembrolizumab plus Pemetrexed and Platinum in Nonsquamous Non–Small-Cell Lung Cancer (KEYNOTE-189)

Leena Gandhi, Delvys Rodríguez-Abreu, Shirish Gadgeel, et al.

Bottom Line

The addition of pembrolizumab to pemetrexed and platinum-based chemotherapy significantly improves overall survival and progression-free survival in patients with previously untreated metastatic nonsquamous non-small cell lung cancer, regardless of PD-L1 expression.

Key Findings

1. Pembrolizumab in combination with chemotherapy significantly reduced the risk of death compared to chemotherapy alone, with a hazard ratio (HR) for overall survival (OS) of 0.49 (95% CI, 0.38 to 0.64; P<0.001) in the initial analysis.
2. Progression-free survival (PFS) was notably improved in the pembrolizumab arm, showing a median PFS of 8.8 months versus 4.9 months in the placebo arm (HR 0.52; 95% CI, 0.43 to 0.64; P<0.001).
3. The clinical benefit of adding pembrolizumab was consistent across all subgroups, including those with low (<1%) or absent PD-L1 tumor proportion scores.
4. Five-year long-term follow-up data demonstrated sustained efficacy, with an OS rate of 19.4% in the pembrolizumab-combination arm compared to 11.3% in the placebo-combination arm.

Study Design

Design
RCT
Double-Blind
Sample
616
Patients
Duration
5.4 yr
Median
Setting
Multicenter, International
Population Patients with previously untreated metastatic nonsquamous non–small-cell lung cancer without EGFR or ALK genomic aberrations
Intervention Pembrolizumab (200 mg) plus pemetrexed and investigator's choice of platinum (cisplatin or carboplatin) every 3 weeks for four cycles, followed by maintenance pembrolizumab and pemetrexed for up to 35 cycles
Comparator Placebo plus pemetrexed and investigator's choice of platinum (cisplatin or carboplatin) every 3 weeks for four cycles, followed by maintenance placebo and pemetrexed
Outcome Overall survival and progression-free survival

Study Limitations

The study allowed for crossover of patients from the placebo-chemotherapy arm to anti-PD-(L)1 therapy upon disease progression, which may confound the overall survival results.
Subgroup analyses, particularly regarding specific PD-L1 expression levels or secondary endpoints, may be exploratory and were not powered to definitively demonstrate superiority in smaller strata.
The study excluded patients with active autoimmune diseases or those requiring immunosuppressive therapy, limiting the generalizability of these results to these specific patient populations.
The trial population did not include individuals with EGFR or ALK genomic alterations, excluding these distinct biological subgroups from the study's conclusions.

Clinical Significance

This trial established the 'triplet' regimen of pembrolizumab, pemetrexed, and platinum-based chemotherapy as a standard-of-care, first-line treatment for metastatic nonsquamous non-small cell lung cancer lacking targetable EGFR or ALK mutations, effectively doubling the efficacy of traditional chemotherapy and setting a new benchmark for patient outcomes in the first-line setting.

Historical Context

Before KEYNOTE-189, platinum-based doublet chemotherapy was the standard of care for metastatic NSCLC without sensitizing driver mutations. Following promising Phase 2 data from KEYNOTE-021 (Cohort G), KEYNOTE-189 served as the pivotal Phase 3 confirmatory study that solidified the role of immune checkpoint inhibitors in the first-line therapeutic landscape, even for patients with low or negative PD-L1 expression.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the biological rationale for combining cytotoxic chemotherapy with pembrolizumab, and how does this synergistic approach overcome immune evasion in nonsquamous non-small-cell lung cancer (NSCLC)?

Key Response

Cytotoxic chemotherapy promotes 'immunogenic cell death,' which releases tumor-associated antigens and pro-inflammatory cytokines. This increases the visibility of the tumor to the immune system. When combined with pembrolizumab (a PD-1 inhibitor), which prevents the tumor from 'turning off' T-cells via the PD-1/PD-L1 checkpoint, the immune system is better primed and unleashed to mount a more effective anti-tumor response than either modality could achieve alone.

Resident
Resident

In a patient with metastatic nonsquamous NSCLC and a PD-L1 Tumor Proportion Score (TPS) of <1%, how should the findings of KEYNOTE-189 influence your first-line treatment recommendation compared to chemotherapy alone?

Key Response

KEYNOTE-189 demonstrated a significant survival benefit for the addition of pembrolizumab to pemetrexed-platinum chemotherapy across all PD-L1 expression levels. Specifically, even in the PD-L1-negative group (TPS <1%), the hazard ratio for death was 0.59. Therefore, the presence of pembrolizumab should be part of the first-line regimen regardless of low or negative PD-L1 expression, unless specific contraindications to immunotherapy exist.

Fellow
Fellow

When considering the OS benefit observed in KEYNOTE-189, how do you reconcile the choice between pembrolizumab monotherapy (per KEYNOTE-024) and the KEYNOTE-189 triplet for a patient with PD-L1 TPS ≥50%?

Key Response

While both are Category 1 options, the choice involves balancing tumor burden and patient fitness. The KEYNOTE-189 triplet generally offers higher objective response rates (47.6%) and may be preferred in patients with high tumor burden or symptomatic disease where a rapid response is prioritized. Conversely, monotherapy (KEYNOTE-024) might be preferred for patients who are less fit for intensive chemotherapy, as it spares them the toxicities of platinum-pemetrexed while still offering durable survival benefit.

Attending
Attending

How does the 'tail of the curve' observed in KEYNOTE-189 change the clinical dialogue regarding prognosis and long-term maintenance in metastatic lung cancer compared to the previous platinum-doublet era?

Key Response

Before KEYNOTE-189, metastatic NSCLC was viewed almost exclusively through a palliative lens with a predictable decline. The plateauing of the survival curve suggests that a subset of patients may achieve long-term, durable responses. This necessitates a shift in teaching toward managing immunotherapy-related adverse events (irAEs) over long durations and discussing the potential for a 'chronic disease' state with patients rather than just short-term survival extensions.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The KEYNOTE-189 trial allowed for a crossover rate of nearly 50% from the placebo-combination group to pembrolizumab monotherapy upon progression. From a statistical standpoint, how does this crossover affect the interpretation of the Hazard Ratio for Overall Survival (OS)?

Key Response

Crossover typically biases the results toward the null hypothesis (an HR of 1.0) because the control group eventually receives the active treatment. The fact that KEYNOTE-189 still achieved a highly significant OS Hazard Ratio (0.49) despite this crossover suggests an exceptionally strong treatment effect for early (first-line) versus delayed (second-line) administration of pembrolizumab.

Journal Editor
Journal Editor

A critical reviewer might point out that KEYNOTE-189 excluded patients with EGFR or ALK mutations. How does this exclusion impact the study's generalizability and its significance in the evolving landscape of precision oncology?

Key Response

Excluding EGFR/ALK-mutated patients was essential for internal validity because these populations have a distinct biology, often respond poorly to PD-1 inhibitors, and have established targeted therapies (TKIs). However, it means the study's results cannot be generalized to all 'nonsquamous' patients. A tough reviewer would ensure the manuscript clearly defines its 'driver-negative' population to avoid clinicians inappropriately using this triplet as first-line therapy for patients who should receive targeted TKIs.

Guideline Committee
Guideline Committee

Based on the OS and PFS data from KEYNOTE-189, how should current guidelines grade the recommendation for pembrolizumab-pemetrexed-platinum as a first-line therapy, and how does it compare to the ASCO/NCCN standing for PD-L1 TPS 1-49% patients?

Key Response

KEYNOTE-189 provides Level 1, Category 1 evidence. For patients with TPS 1-49%, the triplet regimen is now the preferred standard because pembrolizumab monotherapy has not shown as robust a benefit in this subgroup as it has in the ≥50% group (per KEYNOTE-042). Guidelines were updated to reflect this, moving the chemo-IO combination to a 'preferred' status over platinum-doublet alone, which was the previous standard of care.

Clinical Landscape

Noteworthy Related Trials

2016

KEYNOTE-021G

n = 123 · Lancet Oncol

Tested

Pembrolizumab plus carboplatin and pemetrexed

Population

Treatment-naive stage IIIB/IV nonsquamous NSCLC

Comparator

Carboplatin and pemetrexed

Endpoint

Progression-free survival

Key result: The addition of pembrolizumab significantly improved progression-free survival compared to chemotherapy alone.
2018

IMpower150

n = 1202 · NEJM

Tested

Atezolizumab plus carboplatin, paclitaxel, and bevacizumab

Population

Metastatic nonsquamous NSCLC

Comparator

Carboplatin plus paclitaxel and bevacizumab

Endpoint

Progression-free survival and overall survival

Key result: Atezolizumab plus chemotherapy and bevacizumab significantly improved progression-free and overall survival in patients with metastatic nonsquamous NSCLC.
2018

KEYNOTE-407

n = 559 · NEJM

Tested

Pembrolizumab plus carboplatin and paclitaxel/nab-paclitaxel

Population

Treatment-naive metastatic squamous NSCLC

Comparator

Placebo plus carboplatin and paclitaxel/nab-paclitaxel

Endpoint

Overall survival and progression-free survival

Key result: Pembrolizumab plus chemotherapy significantly prolonged both overall and progression-free survival compared to chemotherapy alone in patients with squamous NSCLC.

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