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Acute Myocardial Infarction Phase 3 Win Ratio NCT04564742

DAPA-MI: Complete Statistical Analysis of Dapagliflozin in Acute Myocardial Infarction

An independent statistical review of the randomized phase 3 DAPA-MI trial evaluating dapagliflozin 10 mg versus matching placebo in patients with acute myocardial infarction, with the primary analysis based on a hierarchical composite endpoint and win-ratio methodology.

DAPA-MI  ·  Phase 3  ·  Completed  ·  Enrollment 4017
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. Trial-specific numerical results on this page are restricted to the ClinicalTrials.gov record.

1. Trial at a Glance

DAPA-MI was a randomized, parallel-group, quadruple-masked, phase 3 trial evaluating dapagliflozin 10 mg versus placebo in participants with acute myocardial infarction and heart failure. The primary endpoint was analyzed using a hierarchical composite framework and the win-ratio method.

4017
Enrollment
All randomized participants
2
Treatment arms
Dapagliflozin vs placebo
1.34
Primary win ratio
95% CI 1.20–1.50
<0.001
P-value
Two-sided 95% CI
FeatureDAPA-MI
Trial nameDAPA-MI
PhasePhase 3
ConditionsAcute Myocardial Infarction; Heart Failure
AllocationRandomized
Design modelParallel
MaskingQuadruple
Primary purposeTreatment
Enrollment4017
InterventionsDapagliflozin (drug); Placebo (drug)
Primary endpoint typeCount / rate
Primary hypothesisSuperiority
Primary statistical methodWin ratio
Lead sponsorAstraZeneca
StatusCompleted
Start2020-12-22
Primary completion2023-07-05
ClinicalTrials.govNCT04564742

2. Clinical Question

The statistical question was whether treatment with dapagliflozin 10 mg, given once daily in addition to standard of care, produced a superior result compared with matching placebo when participants were evaluated using the registered hierarchical primary composite endpoint.

Population

Participants in the DAPA-MI trial with the registered conditions of acute myocardial infarction and heart failure.

Intervention

Dapagliflozin 10 mg, given once daily in addition to standard of care.

Comparator

Matching placebo, given once daily in addition to standard of care.

Primary question

Does dapagliflozin provide a superior result to placebo on the hierarchical primary composite endpoint?

3. Trial Design

01
Randomize4017 participants
02
Two armsDapagliflozin or placebo
03
Quadruple maskMasked trial design
04
Follow-upMaximum 29 months
05
Primary analysisHierarchical win ratio
Allocation
Randomized allocation was used in a parallel-group design.
Masking
The registry classifies the trial as quadruple-masked.
Primary purpose
Treatment.
Hypothesis
The registered primary hypothesis type was superiority.
ARM A · DAPA 10 MG

Dapagliflozin

  • Dapagliflozin 10 mg
  • Given once daily
  • In addition to standard of care
ARM B · PLACEBO

Placebo

  • Matching placebo
  • Given once daily
  • In addition to standard of care

The registry reports a mean study duration of 12.0 months, measured as time in study until the last visit, with an accumulated 4023.9 participant-years. The maximum study duration for any participant was 29 months.

4. Endpoints

EndpointRegistered time frameType / analysis
Analysis of the Hierarchical Primary Composite Endpoint (Full Analysis Set) 29 months Binary endpoint in the posted statistical analysis; analyzed using a win-ratio method

The registry describes the primary endpoint as a hierarchical primary composite endpoint. The registry definition states that participants received dapagliflozin 10 mg or matching placebo once daily in addition to standard of care. It also states that the number of events for NYHA corresponds to the number of participants with a non-missing value for NYHA.

Endpoint interpretation: A hierarchical composite is different from simply counting whether a participant experienced any one of several events. The win-ratio framework uses the prespecified hierarchy to determine which participant or treatment arm has the more favorable outcome when two participants are compared. The ClinicalTrials.gov record does not provide enough detail to reconstruct every level of the hierarchy, so this page does not infer or add unreported component definitions.

5. Statistical Methodology

Full Analysis Set

The posted primary analysis is explicitly labeled Analysis of the Hierarchical Primary Composite Endpoint (Full Analysis Set). The statistical-analysis record states that all participants are included in the analysis and that each row item displays the number of participants for the given event without preceding events defined in the hierarchical order.

Intention-to-treat principle

The statistical-analysis record identifies intention-to-treat analysis as a concept used in the primary analysis. In an intention-to-treat framework, randomized participants remain associated with their assigned treatment group for the primary comparison. This preserves the treatment contrast created by randomization rather than redefining groups according to treatment actually received.

Win-ratio analysis

The registry reports Win Ratio Analysis as the method used for the primary endpoint. The win ratio is particularly suited to hierarchical composite endpoints because it can preserve the ordering of clinical outcomes rather than treating all component events as interchangeable.

Conceptual form
WR = number of treatment-group wins ÷ number of control-group wins

This is a conceptual representation rather than a reconstruction of the DAPA-MI calculation. A win is determined using the trial's prespecified hierarchy and comparison rules. The ClinicalTrials.gov record does not provide the complete pairwise event table needed to reproduce the reported value.

Superiority testing

The registered hypothesis type was superiority. The objective was therefore to determine whether the clinical benefit of dapagliflozin was superior to placebo using the hierarchical composite endpoint and win-ratio method.

Multiplicity adjustment

The statistical-analysis record identifies multiplicity adjustment as an additional concept in the analysis text. This is important because hierarchical composite endpoints and clinical-trial analyses can involve multiple ordered comparisons. The ClinicalTrials.gov record does not specify the complete multiplicity procedure, so no additional adjustment scheme is inferred here.

6. Primary Result

Hierarchical Primary Composite Endpoint

Win ratio

1.34

95% CI: 1.20–1.50   ·   P < 0.001

Two-sided confidence interval; superiority hypothesis.

The primary analysis compared dapagliflozin 10 mg versus placebo in the full analysis set. The reported effect measure was the Win Ratio (WR), with an estimate of 1.34 and a two-sided 95% confidence interval of 1.20 to 1.50. The reported P-value was <0.001.

Primary endpointDapagliflozin 10 mg vs placebo
EndpointAnalysis of the Hierarchical Primary Composite Endpoint (Full Analysis Set)
Time frame29 months
AnalysisWin Ratio Analysis
Effect measureWin Ratio (WR)
Estimate1.34
95% CI1.20–1.50
P-value<0.001
HypothesisSuperiority
Analysis populationFull Analysis Set
Clinical Biostats interpretation

A win ratio of 1.34 means that, under the trial's hierarchical comparison framework, the treatment group had an estimated 34% as many wins above the comparator's wins when expressed as the ratio of treatment-group wins to control-group wins. Put differently, the estimated number of favorable pairwise outcomes for dapagliflozin was 1.34 times the corresponding number for placebo under the prespecified hierarchy.

The win ratio does not mean that 34% of participants improved, that 34% more participants benefited, or that an individual participant had a 34% greater probability of a favorable outcome. It is a population-level comparative statistic whose meaning depends on the hierarchy and pairwise rules used by the trial.

The 95% confidence interval of 1.20–1.50 describes statistical uncertainty around the estimated win ratio under the analysis framework. It does not describe the range of treatment effects experienced by individual participants.

The P-value <0.001 addresses the statistical evidence against the relevant null hypothesis under the prespecified analysis. It does not measure the size or clinical importance of the treatment effect. Effect size and uncertainty are better conveyed by the win ratio together with its confidence interval.

The interpretation also depends on the hierarchical structure of the composite endpoint. Because the ClinicalTrials.gov record does not provide the complete hierarchy or pairwise event counts, the numerical win ratio cannot be decomposed here into the contribution made by each individual endpoint component.

7. Understanding the Win Ratio

The win ratio differs fundamentally from a conventional risk ratio or hazard ratio. It is designed for situations in which several outcomes can be ordered by clinical importance. Rather than reducing all outcomes to a single undifferentiated event indicator, the analysis compares outcomes according to their hierarchy.

What a win means

A favorable pairwise comparison is a "win" for one treatment group according to the endpoint hierarchy and prespecified comparison rules.

What a loss means

The opposite treatment group has the more favorable outcome according to those same rules.

Why hierarchy matters

The clinical importance assigned to different outcomes determines which outcome takes precedence when comparing participants.

Why the ratio is useful

A ratio above 1 indicates more favorable pairwise outcomes for the treatment group under the defined comparison framework.

Interpretive anchor
WR = 1  →  equal numbers of treatment and control wins

A value above 1 favors the treatment group in the win-ratio framework, while a value below 1 favors the comparator. The DAPA-MI estimate was 1.34.

Importantly, the win ratio should not be translated into a conventional percentage reduction in risk. A win ratio and a hazard ratio are different effect measures, and the numerical relationship between them is not a simple conversion.

8. Why the Confidence Interval Matters

The primary estimate of 1.34 is only one part of the statistical result. The associated 95% confidence interval, 1.20–1.50, provides information about the precision of the estimate.

Statistical interpretation

The interval lies above 1, consistent with the reported superiority result. Its width reflects uncertainty in estimating the underlying treatment comparison from the available trial data. The interval does not imply that the true effect is equally likely to be any particular value within its limits, nor does it describe individual patient outcomes.

The confidence interval and P-value answer related but different questions. The confidence interval emphasizes the estimated effect and its precision; the P-value quantifies evidence against the null hypothesis under the specified statistical model and testing framework. Neither statistic alone describes clinical importance.

9. Trial Timing and Follow-Up

2020-12-22

Trial start

The DAPA-MI trial began on December 22, 2020.

29 months

Registered endpoint time frame

The hierarchical primary composite endpoint was registered with a 29-month time frame, which was also the maximum study duration for any participant.

2023-07-05

Primary completion

The registry lists July 5, 2023 as the primary completion date.

The registry reports a mean study duration of 12.0 months, measured as time in study until the last visit, and 4023.9 accumulated participant-years. The distinction between mean study duration and the maximum follow-up is important: not every participant necessarily contributed the maximum duration of observation.

10. Safety

The ClinicalTrials.gov record reports serious adverse events by treatment arm. These data are presented separately from the efficacy analysis because safety and efficacy address different statistical questions.

Safety measureDapagliflozin 10 mgPlacebo
Serious adverse events, affected / at risk449 / 2019404 / 1998
Serious adverse events — affected participants
Dapagliflozin 10 mg
449
Placebo
404

The registry provides the affected and at-risk counts but does not provide a formal statistical comparison for serious adverse events in the ClinicalTrials.gov record. The counts should therefore not be converted into an unreported treatment-effect estimate or P-value.

11. Statistical Methods Explained

Why was a win ratio used?

The registered primary endpoint is hierarchical and composite. A win-ratio analysis can preserve the ordering of outcomes by comparing participants pairwise according to that hierarchy. This can be useful when some clinical outcomes are considered more important than others and should therefore take precedence in the analysis.

What does a win ratio of 1.34 mean?

A win ratio of 1.34 indicates that, under the prespecified hierarchical comparison framework, treatment-group wins were estimated to occur 1.34 times as often as control-group wins. It is not a 34% response rate, a 34% reduction in risk, or a 34% increase in survival.

Why does the confidence interval matter?

The 95% confidence interval of 1.20–1.50 quantifies uncertainty around the estimated win ratio. The point estimate alone can conceal how precisely the treatment effect has been estimated. The interval also remains tied to the statistical framework used to generate it.

Why does the P-value not measure effect size?

The P-value of <0.001 indicates strong statistical evidence against the relevant null hypothesis under the prespecified analysis. It does not say whether the effect is small, moderate, or large in clinical terms. Effect size is communicated by the win ratio itself, while precision is communicated by the confidence interval.

Why is the analysis based on the Full Analysis Set?

The registry explicitly labels the primary endpoint analysis as being performed in the Full Analysis Set and states that all participants are included in the analysis. This anchors the primary comparison to the trial population defined for that analysis rather than restricting the analysis to participants who completed treatment.

Why does the hierarchy matter when interpreting the result?

A hierarchical composite does not treat every component as equivalent. The ordering determines which outcome takes precedence in pairwise comparisons. Consequently, the same numerical win ratio cannot be interpreted without reference to the endpoint hierarchy. Because the complete hierarchy is not included in the ClinicalTrials.gov record, this page does not infer component-specific contributions.

Does a win ratio of 1.34 mean that 34% more patients benefited?

No. A win ratio is constructed from favorable and unfavorable pairwise comparisons. It is therefore not equivalent to a proportion of patients benefiting, an absolute risk difference, or a relative risk. Translating it into one of those measures without the underlying data would be statistically incorrect.

12. Intention-to-Treat Analysis

The registry's statistical-analysis record specifically identifies intention-to-treat analysis among the concepts used in the primary analysis. The principle is important because randomization establishes the basis for a causal comparison before treatment adherence, discontinuation, or other post-randomization events occur.

Preserves randomization

Participants remain associated with their randomized treatment assignment for the primary efficacy comparison.

Reduces post-randomization selection

Restricting analysis to participants who behave exactly as planned can introduce selection into a randomized comparison.

In this trial, the statistical-analysis record states that all participants were included in the analysis. That is consistent with the purpose of an intention-to-treat approach: maintaining the treatment contrast generated by randomization.

13. Multiplicity and Hierarchical Testing

The ClinicalTrials.gov record identifies multiplicity adjustment as a concept in the primary analysis text. Multiplicity is a central issue whenever a study involves multiple statistical opportunities to obtain evidence against a null hypothesis.

ConceptRole in DAPA-MI
Primary endpointHierarchical primary composite endpoint
Primary hypothesisSuperiority
Primary effect measureWin Ratio (WR)
MultiplicityIdentified in the posted analysis text
Analysis populationFull Analysis Set

A hierarchical endpoint can itself provide a structured way to prioritize outcomes, but that does not eliminate the need to understand the trial's formal error-control strategy. The ClinicalTrials.gov record identifies multiplicity adjustment as part of the analysis but do not provide enough detail to reconstruct the full procedure. Accordingly, no additional alpha-spending or multiplicity algorithm is attributed to DAPA-MI here.

14. What the Primary Result Does — and Does Not — Establish

What it establishes statistically

The posted primary analysis reports a win ratio of 1.34 with a two-sided 95% confidence interval of 1.20–1.50 and P < 0.001 for dapagliflozin 10 mg versus placebo on the hierarchical primary composite endpoint. The registered hypothesis type was superiority.

What it does not establish by itself

The result does not provide a percentage of participants who benefited, an individual patient's probability of benefit, an absolute risk reduction, a number needed to treat, or a component-specific effect estimate. Those quantities require different data and/or effect measures.

Why the endpoint definition matters

The interpretation of a win ratio is inseparable from the hierarchy used to define wins and losses. Without the complete hierarchy and pairwise event data, the reported 1.34 should be interpreted as the overall registered win-ratio result rather than decomposed into individual components.

15. Design Features That Matter Statistically

Design featureDAPA-MIStatistical relevance
RandomizationRandomizedCreates the treatment comparison used for the primary efficacy analysis.
Parallel designParallelParticipants are compared between two concurrently randomized treatment groups.
MaskingQuadrupleMasking can reduce the potential influence of treatment knowledge on trial conduct and assessment.
Primary endpointHierarchical compositeRequires an analysis that respects the ordering of component outcomes.
Primary methodWin ratioProvides a ratio of treatment-group wins to comparator-group wins under the hierarchy.
HypothesisSuperiorityThe analysis tests whether the treatment produces a superior result rather than establishing non-inferiority.
MultiplicityAdjustment identified in analysis textIndicates that multiplicity was considered in the statistical framework.
ITT conceptIdentified in analysis textSupports analysis according to randomized treatment assignment.

16. Planned vs Reported Statistical Information

The ClinicalTrials.gov record for DAPA-MI contains a formal primary statistical analysis with an effect estimate, confidence interval, and P-value, so the results can be interpreted directly rather than only described as planned.

Reported

The registry reports the primary endpoint, its 29-month time frame, the Full Analysis Set, win-ratio method, estimate of 1.34, 95% CI of 1.20–1.50, and P < 0.001.

Not reconstructed

The ClinicalTrials.gov record does not provide enough information to reconstruct component-level wins, losses, the complete hierarchy, or an independent calculation of the reported win ratio.

This distinction is important in statistical reporting. A published or registry-reported summary statistic should not be reverse-engineered into additional quantities unless the underlying information supports the calculation.

17. Limitations

18. Why This Trial Matters Statistically

DAPA-MI is a useful teaching example because it illustrates a different approach to composite clinical-trial endpoints from the more familiar time-to-event framework. The primary analysis uses a hierarchical composite endpoint and a win ratio, while retaining the randomized comparison and intention-to-treat principles.

ConceptHow it appears in DAPA-MI
RandomizationRandomized parallel-group trial
MaskingQuadruple masking
Intention-to-treatIdentified as a concept in the primary analysis
Composite endpointHierarchical primary composite endpoint
Win ratioPrimary statistical method
SuperiorityRegistered hypothesis type
Confidence interval95% two-sided CI of 1.20–1.50 around the primary estimate
P-value<0.001 for the primary analysis
MultiplicityIdentified in the posted analysis text
Safety analysisSerious adverse-event counts reported by randomized arm

The most important statistical lesson is that the choice of endpoint determines the interpretation of the effect measure. A win ratio should be taught as a win-ratio result, not translated casually into a hazard ratio or risk reduction. Understanding the endpoint construction is therefore just as important as understanding the numerical estimate.

19. A Practical Reading Framework for the DAPA-MI Result

Step 1 · Identify the estimand

The primary question concerns dapagliflozin 10 mg versus placebo using the hierarchical primary composite endpoint.

Step 2 · Identify the analysis

The registry reports a Full Analysis Set and a Win Ratio Analysis.

Step 3 · Read the effect

The reported win ratio is 1.34, indicating more treatment-group wins than control-group wins under the hierarchy.

Step 4 · Read uncertainty

The two-sided 95% CI is 1.20–1.50, describing uncertainty around the estimated ratio.

This four-step approach prevents a common statistical mistake: interpreting the P-value first and the estimand second. The correct order is to understand what was compared, how it was compared, what the effect measure means, and then how precisely it was estimated.

20. Related Tutorials

Learn more about the methods used in this trial:

21. Related Statistical Calculators

22. Sources

Continue through the Clinical Biostats statistical pathway

Use the trial's statistical concepts as a starting point for deeper study of randomization, confidence intervals, intention-to-treat analysis, multiplicity, P-values, and win-ratio methodology.

23. Record Summary

DAPA-MI was a randomized, parallel, quadruple-masked phase 3 trial with 4017 enrolled participants and two treatment arms: dapagliflozin 10 mg and placebo. Its primary endpoint was a hierarchical composite analyzed in the Full Analysis Set using a win-ratio method. The registry reports a primary win ratio of 1.34, with a two-sided 95% confidence interval of 1.20–1.50 and P < 0.001 under a superiority hypothesis.

The central statistical lesson is that the win ratio must be interpreted in the context of its hierarchical endpoint. A value of 1.34 represents the relative frequency of favorable pairwise outcomes under the prespecified comparison framework; it is not interchangeable with a hazard ratio, risk ratio, absolute risk reduction, or percentage of patients benefiting. The confidence interval provides the corresponding measure of statistical precision, while the P-value addresses evidence against the null hypothesis rather than effect magnitude.

The registry also identifies intention-to-treat analysis and multiplicity adjustment among the concepts incorporated into the primary statistical analysis. Serious adverse events were reported as 449/2019 for dapagliflozin 10 mg and 404/1998 for placebo. Because the ClinicalTrials.gov record does not provide the complete endpoint hierarchy, pairwise event data, or a formal safety comparison, this page deliberately does not derive additional component-specific estimates.

Clinical Biostats methodology: A trial-results page should distinguish the reported statistical result from quantities that can only be derived from underlying participant-level or component-level data. For DAPA-MI, the most defensible interpretation is therefore centered on the reported win ratio, its confidence interval and P-value, the Full Analysis Set, and the hierarchical nature of the primary endpoint.