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Advanced Cancer Randomized Palliative Care NCT01245621

ENABLE III: Complete Statistical Analysis of Early Palliative Care in Advanced Cancer

An independent statistical review of the ENABLE III randomized trial evaluating early versus later entry into a palliative care intervention for patients with advanced cancer and their caregivers.

Dartmouth-Hitchcock Medical Center · Enrollment: 360 · Completed
Scope of this record

This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record.

1. Trial at a Glance

ENABLE III evaluated whether an early palliative care intervention compared with later entry into the same behavioral intervention affected patient quality of life, end-of-life care outcomes, survival measures, and caregiver outcomes in advanced cancer.

360
Enrollment
2
Arms
Randomized
Allocation
2010–2013
Study period
FeatureENABLE III
ConditionAdvanced Cancer
SponsorDartmouth-Hitchcock Medical Center
DesignRandomized parallel assignment
MaskingSingle
Primary purposeSupportive Care
InterventionsLater entry group (behavioral); Early palliative care intervention (behavioral)

2. Clinical Question

Population

Patients with advanced cancer and their caregivers.

Intervention

Early palliative care intervention.

Comparator

Later entry group.

Primary question

Does earlier palliative care intervention change patient-centered and caregiver outcomes over time?

3. Trial Design

Randomize
360 patients
Early entry
Palliative care
Later entry
Comparison
Follow-up
Longitudinal outcomes

Early palliative care intervention

Behavioral intervention beginning earlier in the disease course.

Later entry group

Behavioral intervention with later entry.

4. Endpoints

EndpointTime frameDescription
Change in patient's quality of living over timeBaseline, 6,12,18,24,36 and every 12 weeks until death or end of studyQuality of life, mood, and symptom intensity measures including FACIT-Pal, QUAL-E, and CES-D.
Quality of end of life careChart review at death and caregiver proxy interview 2-3 months after patient deathEnd of Life Care Data Collection Form and Quality of Dying and Death Measure.
Estimate and compare hazard ratios and median survival before and after 1 year from enrollmentEnrollment until death or end of studyOverall survival comparison between early and later entry groups.
Change in caregiver quality of life, burden and grief over timeBaseline, 6,12,18,24,36, and every 12 weeks until patient death or end of studyCaregiver quality of life, burden, depressive symptoms, spiritual well-being, and grief measures.

5. Planned Analysis

The registry lists the planned primary outcomes but does not report statistical results on ClinicalTrials.gov.

The quality-of-life and caregiver endpoints are longitudinal outcomes measured repeatedly over time. A statistical analysis of these outcomes would typically account for correlation among repeated measurements within the same participant rather than treating each time point as an independent observation.

The survival endpoint would typically be analyzed using time-to-event methods. Hazard ratios compare the estimated event rate between randomized groups over follow-up, while median survival describes the time at which half of the population has experienced the event.

Conceptual survival analysis
Survival function: S(t) = Probability of remaining event-free beyond time t

Time-to-event methods account for patients who have different follow-up durations and patients whose event status is not observed before study completion.

6. Statistical Methodology

Longitudinal outcome analysis

Patient quality-of-life and caregiver measures were collected repeatedly over time. Longitudinal statistical models are designed to estimate changes over time while accounting for the fact that measurements from the same person are related.

Time-to-event analysis

The survival endpoint compares outcomes from enrollment until patient death or end of study. Hazard ratios summarize relative differences in event rates between groups during follow-up.

Patient-centered outcomes

Measures such as quality of life, mood, symptoms, caregiver burden, and grief represent multidimensional outcomes. Statistical interpretation requires distinguishing changes in measurement scores from clinically meaningful changes experienced by patients and caregivers.

7. Statistical Methods Explained

Why are repeated measurements important?

ENABLE III collected assessments at multiple time points. Repeated measurements allow investigators to study trajectories rather than only a single baseline-to-endpoint comparison.

Why use survival analysis?

Survival methods are appropriate when the outcome is the time until an event, such as death. They allow inclusion of participants with different lengths of follow-up.

What does a hazard ratio represent?

A hazard ratio describes the relative estimated event rate between groups over time. It is not the same as the proportion of patients experiencing an event.

Why are quality-of-life measures statistically complex?

Quality-of-life scores can change over time, may vary between individuals, and may be affected by missing assessments as disease progresses.

8. Limitations

9. Why This Trial Matters Statistically

ConceptHow it appears in ENABLE III
RandomizationComparison of early versus later behavioral palliative care intervention.
Longitudinal analysisRepeated quality-of-life and caregiver assessments over time.
Survival analysisHazard ratio and median survival comparison from enrollment.
Patient-reported outcomesQuality of life, symptoms, mood, burden, grief, and spiritual well-being.
Missing data considerationsImportant in populations followed until death or end of study.

10. Sources