New England Journal of Medicine JUNE 05, 2017

Alectinib versus Crizotinib in Patients with ALK-Positive Non-Small-Cell Lung Cancer (ALEX)

Shaw AT, Peters S, Mok TSK, et al.

Bottom Line

The ALEX trial demonstrated that first-line alectinib significantly improved progression-free survival and reduced the risk of central nervous system progression compared to crizotinib in patients with treatment-naive advanced ALK-positive non-small-cell lung cancer.

Key Findings

1. Alectinib significantly improved progression-free survival (PFS) compared to crizotinib, with an investigator-assessed hazard ratio (HR) of 0.47 (95% CI, 0.34-0.65; P<0.0001) at the primary analysis.
2. Alectinib demonstrated superior central nervous system (CNS) efficacy, with a cause-specific hazard ratio for CNS progression of 0.16 (95% CI, 0.10-0.28; P<0.0001) compared to crizotinib.
3. The 12-month cumulative incidence of CNS progression was markedly lower in the alectinib group (9.4%) compared to the crizotinib group (41.4%).
4. Alectinib maintained a favorable safety profile with lower rates of grade 3-5 adverse events (41%) compared to crizotinib (50%).
5. Mature follow-up (up to 5 years) showed a 5-year overall survival rate of 62.5% for alectinib versus 45.5% for crizotinib, although the survival benefit did not reach formal statistical significance due to trial design and crossover (HR 0.67; 95% CI, 0.46-0.98).

Study Design

Design
RCT
Open-Label
Sample
303
Patients
Duration
48.2 mo
Median
Setting
Multicenter, International
Population Treatment-naive patients with stage IIIB/IV ALK-positive non-small-cell lung cancer
Intervention Alectinib 600 mg orally twice daily
Comparator Crizotinib 250 mg orally twice daily
Outcome Investigator-assessed progression-free survival (PFS)

Study Limitations

The trial was open-label, which may introduce bias in assessment, particularly for subjective endpoints.
The primary analysis for OS was confounded by the potential impact of subsequent therapies after disease progression in the crizotinib arm.
The study was not originally powered to detect a statistically significant difference in overall survival.
Patients with high-grade (symptomatic) CNS metastases were excluded, potentially limiting the generalizability of results to the most severe neurological presentations.

Clinical Significance

The ALEX trial established alectinib as the new standard-of-care for first-line treatment of advanced ALK-positive NSCLC, shifting the paradigm from the first-generation TKI crizotinib to more potent, CNS-penetrant second-generation inhibitors, significantly improving disease control and durability of response.

Historical Context

Prior to the ALEX trial, crizotinib was the first approved ALK inhibitor and standard-of-care, but patients frequently experienced early disease progression, particularly in the central nervous system due to poor blood-brain barrier penetration. ALEX was the pivotal global phase 3 study that demonstrated the superiority of next-generation ALK inhibitors, following early suggestions of efficacy from the smaller J-ALEX trial.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the specific molecular driver involved in ALK-positive non-small-cell lung cancer (NSCLC), and what pharmacological advantage does alectinib possess over crizotinib regarding the blood-brain barrier?

Key Response

ALK-positive NSCLC is typically driven by an EML4-ALK gene fusion resulting from a chromosomal inversion. Alectinib is a second-generation TKI designed to be more selective and, crucially, is not a substrate for the P-glycoprotein efflux pump, allowing it to achieve much higher concentrations in the central nervous system compared to crizotinib.

Resident
Resident

In a patient presenting with newly diagnosed ALK-positive advanced NSCLC and multiple asymptomatic brain metastases, how do the results of the ALEX trial dictate the first-line management strategy?

Key Response

The ALEX trial showed that alectinib significantly reduced the risk of CNS progression compared to crizotinib (12-month cumulative incidence of 9.4% vs 41.4%). Because of its superior CNS efficacy and systemic PFS, alectinib is the preferred first-line therapy, often allowing for the deferral of cranial radiation in asymptomatic patients.

Fellow
Fellow

The ALEX trial reported a significantly improved PFS for alectinib, but how should a clinician interpret the lack of mature overall survival (OS) data at the time of the primary analysis, and what role does crossover play in this context?

Key Response

While PFS was the primary endpoint and was clearly superior (HR 0.47), OS is often confounded by subsequent lines of therapy in ALK+ disease. In ALEX, many patients in the crizotinib arm crossed over to alectinib or other potent ALK inhibitors upon progression, which tends to dilute the OS benefit, making PFS a more sensitive measure of the initial regimen's efficacy.

Attending
Attending

How does the toxicity profile of alectinib observed in the ALEX trial shift the paradigm of long-term survivorship management compared to the previous standard of crizotinib?

Key Response

Alectinib demonstrated a more favorable safety profile despite longer treatment duration, with lower rates of Grade 3-5 adverse events (41% vs 50%). Clinically, this means moving away from managing the gastrointestinal and visual disturbances common with crizotinib toward monitoring for specific alectinib-related issues like myalgias (increased CPK), photosensitivity, and bradycardia.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

Considering the design of the ALEX trial, how does the use of a competing-risk analysis for CNS progression provide a more accurate assessment of drug efficacy than standard Kaplan-Meier estimates?

Key Response

Standard Kaplan-Meier estimates for CNS progression can be biased by the 'competing risk' of systemic progression or death. By using a cumulative incidence function (CIF) that accounts for death and non-CNS progression as competing events, the ALEX trial researchers more accurately isolated the specific CNS-protective effect of alectinib.

Journal Editor
Journal Editor

As a reviewer, what are the implications of the ALEX trial's open-label design on the investigator-assessed PFS, and was the inclusion of an Independent Review Committee (IRC) sufficient to mitigate potential bias?

Key Response

Open-label designs can lead to 'ascertainment bias' where investigators might be more likely to declare progression in the control arm. In ALEX, while the investigator HR was 0.47, the IRC HR was 0.50. The consistency between these two values suggests the treatment effect was robust and not significantly inflated by the lack of blinding.

Guideline Committee
Guideline Committee

Based on the ALEX trial, why should alectinib be designated as a 'Category 1' recommendation over crizotinib in global guidelines such as NCCN or ESMO, and what criteria were met to justify this shift?

Key Response

Alectinib met all criteria for a Category 1 recommendation: high-quality evidence from a large, randomized Phase III trial showing a clear, statistically significant, and clinically meaningful improvement in its primary endpoint (PFS). Current guidelines (NCCN Version 1.2024) list alectinib as 'Preferred' because it outperformed crizotinib in both systemic and intracranial efficacy while maintaining better tolerability.

Clinical Landscape

Noteworthy Related Trials

2014

PROFILE 1014 Trial

n = 343 · NEJM

Tested

Crizotinib

Population

Treatment-naive patients with advanced ALK-positive NSCLC

Comparator

Pemetrexed-platinum chemotherapy

Endpoint

Progression-free survival

Key result: Crizotinib showed superior progression-free survival compared to standard chemotherapy in the first-line setting.
2017

ASCEND-4 Trial

n = 376 · NEJM

Tested

Ceritinib

Population

Treatment-naive patients with advanced ALK-positive NSCLC

Comparator

Platinum-based chemotherapy

Endpoint

Progression-free survival

Key result: Ceritinib significantly prolonged progression-free survival compared to chemotherapy, with a manageable safety profile.
2018

ALTA-1L Trial

n = 275 · NEJM

Tested

Brigatinib

Population

Treatment-naive patients with advanced ALK-positive NSCLC

Comparator

Crizotinib

Endpoint

Progression-free survival

Key result: Brigatinib demonstrated superior progression-free survival compared to Crizotinib, with improved systemic and intracranial efficacy.

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