The New England Journal of Medicine MAY 31, 2019

Apalutamide in Patients with Metastatic Castration-Sensitive Prostate Cancer (TITAN Study)

Kim N. Chi, et al.

Bottom Line

In the phase 3 TITAN trial, the addition of apalutamide to androgen-deprivation therapy (ADT) significantly improved overall survival and radiographic progression-free survival in a broad population of patients with metastatic castration-sensitive prostate cancer.

Key Findings

1. Apalutamide combined with ADT significantly reduced the risk of death by 35% compared to placebo plus ADT (hazard ratio, 0.65; 95% CI, 0.53 to 0.79; P < 0.0001) after a median follow-up of 44.0 months.
2. After adjusting for crossover, the risk of death was reduced by 48% (hazard ratio, 0.52; 95% CI, 0.42 to 0.64; P < 0.0001).
3. Median overall survival was not reached in the apalutamide group, compared to 52.2 months in the placebo group.
4. Apalutamide significantly delayed the time to radiographic disease progression or death, with a 52% reduction in risk (hazard ratio, 0.48; 95% CI, 0.39 to 0.60; P < 0.001) at the primary analysis.
5. Health-related quality of life was maintained throughout the study in both groups.

Study Design

Design
RCT
Double-Blind
Sample
1,052
Patients
Duration
44.0 mo
Median
Setting
Multicenter, international
Population Patients with metastatic castration-sensitive prostate cancer (mCSPC) receiving continuous androgen-deprivation therapy (ADT), regardless of disease volume, prior docetaxel use, or prior treatment for localized disease.
Intervention Apalutamide (240 mg daily) plus ADT
Comparator Placebo plus ADT
Outcome Overall survival and radiographic progression-free survival

Study Limitations

The study design included a crossover provision for placebo-treated patients, which required sensitivity analyses to estimate the true effect on overall survival.
The final analysis was performed without formal statistical retesting or adjustments for multiplicity after the study was unblinded.
The benefit in the pre-specified subgroup of patients who had received prior docetaxel was less pronounced.

Clinical Significance

The TITAN trial provides robust evidence supporting the use of apalutamide in combination with ADT as an effective first-line treatment for a broad population of patients with mCSPC, including those with both high- and low-volume disease, regardless of prior systemic therapy for localized disease.

Historical Context

The TITAN trial, published in 2019, followed established data showing that adding second-generation antiandrogens or chemotherapy to ADT improves survival in mCSPC. It expanded the therapeutic options by demonstrating efficacy in a more heterogeneous patient population than previous landmark trials like CHAARTED or LATITUDE.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the mechanism of action of apalutamide differ from first-generation antiandrogens like bicalutamide, and why is this significant in the treatment of metastatic castration-sensitive prostate cancer (mCSPC)?

Key Response

Apalutamide is a next-generation androgen receptor (AR) inhibitor. Unlike first-generation agents like bicalutamide, which only compete for ligand binding, apalutamide also inhibits AR nuclear translocation and AR-mediated DNA binding. This more potent inhibition is crucial in mCSPC because it overcomes common resistance mechanisms seen in first-generation drugs, where the receptor can occasionally act as an agonist in the setting of rising AR expression levels.

Resident
Resident

In a patient newly diagnosed with metastatic castration-sensitive prostate cancer, what factors would lead you to choose the TITAN regimen (Apalutamide + ADT) over the CHAARTED regimen (Docetaxel + ADT)?

Key Response

The choice involves balancing volume of disease, fitness, and toxicity. While docetaxel (CHAARTED) showed primary benefit in high-volume disease and carries risks of myelosuppression and neuropathy, apalutamide (TITAN) showed benefit in both high- and low-volume disease. Apalutamide is generally preferred for patients seeking to avoid chemotherapy or those with low-volume disease, though clinicians must monitor for specific side effects like skin rash and hypothyroidism.

Fellow
Fellow

The TITAN trial included a subset of patients who received prior docetaxel. How should the survival benefit observed in this subgroup inform the clinical adoption of 'triplet therapy' (ADT + Docetaxel + ARPI) in modern uro-oncology?

Key Response

TITAN demonstrated that adding apalutamide improved outcomes even in those with prior docetaxel exposure (hazard ratio for death 0.66). This provided early evidence that intensifying therapy beyond ADT+Docetaxel was beneficial. This has been further validated by trials like PEACE-1 and ARASENS, establishing triplet therapy as a standard of care for patients with high-volume mCSPC who are fit for chemotherapy.

Attending
Attending

Considering the long-term results of the TITAN study, how do you manage the 27% incidence of skin rash and the increased risk of fractures in patients on apalutamide compared to other AR signaling inhibitors?

Key Response

Management of apalutamide-specific toxicities is key for adherence. The rash is typically maculopapular and often resolves with topical steroids or temporary dose interruption/reduction. Fracture risk (reported at roughly 9% vs 3% in placebo) necessitates baseline and periodic bone mineral density (BMD) scans and the early introduction of bone-protective agents (bisphosphonates or denosumab) alongside calcium and Vitamin D, which is a critical teaching point for managing long-term survivors.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The TITAN trial utilized a dual primary endpoint of radiographic progression-free survival (rPFS) and overall survival (OS). What are the statistical implications of this design regarding alpha-spending, and what are the limitations of rPFS as a surrogate for OS in the castration-sensitive setting?

Key Response

Using dual primary endpoints requires the pre-specification of alpha-splitting to control the family-wise type I error rate. In TITAN, alpha was partitioned (e.g., 0.005 for rPFS and 0.045 for OS). While rPFS is a validated surrogate in mCRPC, its correlation with OS in mCSPC is weaker due to the longer duration of subsequent lines of therapy, which can dilute the OS signal, making the achievement of a statistically significant OS benefit in TITAN particularly robust.

Journal Editor
Journal Editor

Following the primary analysis of TITAN, the trial was unblinded and approximately 40% of the placebo group crossed over to the apalutamide arm. As a reviewer, how would you evaluate the impact of this crossover on the final OS hazard ratio, and which statistical models would you require the authors to use to address this?

Key Response

Crossover can lead to an underestimation of the true survival benefit (Type II error). To maintain methodological rigor, an editor would expect the authors to perform sensitivity analyses such as the Rank Preserving Structural Failure Time (RPSFT) model or Inverse Probability of Censoring Weighting (IPCW). These models estimate the treatment effect as if the crossover had not occurred, ensuring the reported efficacy is not confounded by the ethical necessity of unblinding.

Guideline Committee
Guideline Committee

Given that TITAN demonstrated benefit across both high- and low-volume mCSPC, should current guidelines (NCCN or EAU) prioritize ARPIs over docetaxel for low-volume disease, and what is the strength of this recommendation?

Key Response

Current NCCN and EAU guidelines have been updated to reflect TITAN results, giving a Category 1 (Strong) recommendation for adding an ARPI (apalutamide, enzalutamide, or abiraterone) to ADT for mCSPC regardless of volume. Because docetaxel showed no significant OS benefit in low-volume disease (per CHAARTED/STAMPEDE long-term data), guidelines now explicitly favor ARPIs for low-volume patients, whereas for high-volume patients, both ARPIs and triplets are viable options.

Clinical Landscape

Noteworthy Related Trials

2015

CHAARTED Trial

n = 790 · NEJM

Tested

Docetaxel plus Androgen Deprivation Therapy (ADT)

Population

Metastatic castration-sensitive prostate cancer

Comparator

ADT alone

Endpoint

Overall survival

Key result: Adding docetaxel to ADT significantly improved overall survival compared to ADT alone in patients with metastatic castration-sensitive prostate cancer.
2017

LATITUDE Trial

n = 1199 · NEJM

Tested

Abiraterone acetate plus prednisone with ADT

Population

High-risk metastatic castration-sensitive prostate cancer

Comparator

ADT plus dual placebos

Endpoint

Overall survival and radiographic progression-free survival

Key result: The addition of abiraterone acetate and prednisone to ADT significantly improved both overall survival and radiographic progression-free survival.
2019

ARCHES Trial

n = 1150 · JCO

Tested

Enzalutamide plus ADT

Population

Metastatic castration-sensitive prostate cancer

Comparator

ADT plus placebo

Endpoint

Radiographic progression-free survival

Key result: Enzalutamide significantly reduced the risk of radiographic disease progression or death compared to placebo in patients receiving ADT.

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