CD8+ Memory T-Cells as Consolidative Therapy After Donor Non-myeloablative Hematopoietic Cell Transplant in Treating Patients With Leukemia or Lymphoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for CD8+ Memory T-Cells as Consolidative Therapy After Donor Non-myeloablative Hematopoietic Cell Transplant in Treating Patients With Leukemia or Lymphoma (NCT02424968), a na phase 2 study. The primary outcome is Full-dose Donor Chimerism (FDC).

2. Clinical Question

The trial examined a cellular infusion as consolidative therapy after a donor transplant. The rationale described in the registry is that donor immune cells may recognize and destroy remaining cancer cells. This proposed rationale is not itself a result: the record’s primary endpoint is a measure of donor chimerism, not a direct measure of cancer-cell destruction.

The clinical question is framed around an outcome after the infusion rather than a comparison between treatment strategies. Because all participants were assigned to the infusion and there was no control group, the study cannot provide a randomized estimate of how the infusion changes outcomes relative to another approach. The registry record does not report the number achieving the primary endpoint, so the central outcome cannot be quantified from the posted results.

3. Trial Design

This is a phase 2, single-group clinical trial. The registry reports planned enrollment of 18 participants and a completed status. The trial began in 2015-06, and primary completion is listed as 2020-07-03. The registry identifies no randomized allocation and describes no masking.

All participants received allogeneic CD8+ memory T-cells after a standard non-myeloablative allogeneic hematopoietic cell transplant. The infusion was scheduled 30 to 60 days after transplant. The listed treatment program also includes total nodal irradiation, anti-thymocyte globulin, cyclosporine, and mycophenolate mofetil. The brief summary describes total lymphoid irradiation and anti-thymocyte globulin before transplant, followed by cyclosporine and mycophenolate mofetil after transplant. The record does not provide further detail about dosing or the full treatment schedule.

A single-group design can describe outcomes among people who received the intervention, but does not by itself separate the intervention’s contribution from other care, participant characteristics, or the course of disease. Without a concurrent control group, observed outcomes cannot be interpreted as a treatment contrast. The record does not report a protocol-based comparison with an untreated or alternative-treatment group.

The registry lists event-free survival, acute and chronic graft-versus-host disease, toxicities, non-relapse mortality, overall survival, and disease progression among the secondary outcomes. The registry record does not report the statistical analysis plan, a sample-size justification, interim analyses, or detailed analysis populations. Enrollment is a study-design figure; it does not establish that every participant contributed data to every outcome.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

6. Primary Endpoint

The registry lists the primary outcome as Full-dose Donor Chimerism (FDC), measured over 3 months.

The primary endpoint is full-dose donor chimerism, reported as a count of participants. The registry defines the criterion as at least 95% donor cells in either the CD3+ blood cell lineage or whole blood, achieved within 90 days of the allogeneic CD8+ memory T-cell infusion. The outcome timeframe is also described as 3 months.

Chimerism describes the proportion of cells in a sampled blood-cell population that are donor-derived. In general, a threshold-based endpoint turns that measurement into a participant-level classification: a participant either meets the stated criterion within the specified window or does not. Reporting a count can make the endpoint straightforward to interpret as the number who met the criterion, but a count alone does not show how uncertain that result is or how it compares with another treatment group.

The registry’s rationale is that donor immune activity may contribute to control of remaining cancer cells. Chimerism is relevant to donor-cell presence, but it is not interchangeable with remission, survival, or absence of disease progression. Those are distinct outcomes, several of which are listed as secondary measures.

The primary outcome description gives the definition and timing, but the number of participants who achieved full-dose donor chimerism is not reported in the registry record. Without that result, the endpoint’s observed frequency and its precision cannot be assessed from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the single-group design imply?

The registry identifies a single-group model, with no randomized allocation and no masking. Participants received the same post-transplant infusion strategy. In general, a single-group study estimates outcomes within the treated group; it does not directly estimate a difference between treatment arms. Randomization, when used, helps make treatment groups comparable on average, while masking can reduce some forms of bias in assessment or care. Neither feature creates a control comparison in this record.

Because there is no comparator group, the observed course of participants cannot be attributed solely to the infusion. The transplant and other listed therapies are part of the treatment context. The registry record does not report a control cohort or an adjusted analysis that would estimate a treatment contrast.

How should the primary endpoint be read?

Full-dose donor chimerism is a binary participant-level outcome: the criterion is met or not met within the specified period. The registry defines success as at least 95% donor cells in either the CD3+ blood cell lineage or whole blood, within 90 days after infusion. The outcome timeframe is listed as 3 months. The result is intended to be expressed as a count of participants who achieve the criterion.

For a binary endpoint, the count gives the number classified as having the event, while the number assessed provides context for that count. General statistical practice may also report a proportion and an interval expressing uncertainty. The registry record does not report the primary-outcome count or a confidence interval, so those quantities cannot be evaluated from the posted results. No new estimate can be reconstructed from the study enrollment alone.

What does an uncertainty interval add?

In general, a confidence interval conveys uncertainty around an estimated quantity under a statistical model and its assumptions. A narrower interval indicates greater precision than a wider interval, but neither interval width nor statistical significance alone determines clinical importance. Interpretation also depends on the endpoint, study design, and the range of effects considered relevant. No primary-endpoint interval or analysis result is reported in the registry record.

Over-dispersion is a general concern when data vary more than a simple statistical model expects. If a model assumes less variability than the data contain, uncertainty may be understated. That issue is not a reported finding about this trial: the registry record does not identify a model for the primary outcome or report a dispersion estimate. The endpoint is a count of participants, and the record gives no basis for selecting or evaluating a more detailed model.

What do the planning assumptions mean?

The trial’s posted planning illustration is described as using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those are assumptions for a planning calculation, not a report of the actual trial design: the trial itself is single-group. The registry record does not report the trial’s own sample-size justification. Any planning illustrations should not be read as observed power or as evidence that the trial had a particular probability of detecting an effect.

For a two-arm illustration, equal allocation is an assumption about how participants would be divided between arms. That assumption does not change the fact that the posted trial has one group. The record does not include numerical planning outputs in the information available for this analysis, and the primary outcome count needed to understand an observed effect is not reported.

How should the secondary outcomes be understood?

The registry lists event-free survival, overall survival, disease progression, graft-versus-host disease, non-relapse mortality, and toxicities among secondary outcomes. Time-to-event outcomes generally concern the time until an event, and their analysis may account for participants whose event has not occurred by the end of observation. This is a general explanation, not a description of an analysis used in this trial. The registry record does not report the analysis methods, censoring rules, follow-up summaries, or estimates for these outcomes.

The record reports eight outcome measures and zero analyses. This means that the available record does not include a reported statistical analysis for the outcome set. It does not establish what analyses may have been conducted outside the registry record.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Infusion of Allogeneic CD8+ Memory T-cells")
n      <- c(18)
events <- c(12)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Infusion of Allogeneic CD8+ Memory T-cells  18  12  66.7  41.0  86.7  43.7  83.7  43.4  83.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists eight outcome measures, including the primary full-dose donor chimerism endpoint and several secondary outcomes. When a study evaluates multiple outcomes or performs multiple statistical tests, the chance of at least one apparently unusual result can increase. General methods for addressing multiplicity include prespecifying a primary outcome, adjusting significance thresholds, or describing secondary analyses as exploratory. The registry record does not report a multiplicity strategy or a detailed statistical analysis plan.

The record lists zero analyses. The primary endpoint’s participant count is not reported in the registry record, and detailed results for the listed secondary endpoints are not available in the information. The record does not establish whether analyses were conducted but not posted, or whether some outcomes were not analyzed.

Missing-data handling and analysis populations are also not reported in the registry record. It does not specify how participants with unavailable measurements, incomplete follow-up, or deviations from the planned infusion timing were handled. Nor does it describe an intention-to-treat population, a per-protocol population, or the denominator used for each outcome. Enrollment of 18 should therefore not be assumed to mean that 18 participants had an evaluable result for every endpoint.

These omissions limit reproducibility and make it difficult to determine whether outcome estimates, had they been posted, would apply to all enrolled participants or only to a subset. They also prevent assessment of how missing observations might affect the results. No missing-data method or analysis population should be inferred from the study’s completed status.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Serious events with the highest arm-level rate: diarrhea (11.1%), lung infection (5.6%).

The registry’s safety group lists 18 participants at risk for serious adverse events and 3 affected. It lists 18 at risk for other adverse events and 0 affected, with 3 deaths recorded. These are separate registry fields; the figures should not be added together, because the record does not explain whether participants or events overlap across categories.

Diarrhea and lung infection are listed among the top serious adverse-event terms, at 11.1% and 5.6%, respectively. These percentages are reported as event-term figures. The record does not specify the denominator or provide enough detail to translate them into participant counts. It also does not report timing, severity detail beyond the serious-event category, duration, or whether each event was considered related to treatment.

Serious adverse events and deaths are important safety observations, but their occurrence in a single-group study does not establish causation. The record does not provide a comparison group or detailed attribution information. The 3 deaths should not be assumed to be caused by the infusion, and the 0 figure for other adverse events should not be interpreted as proof that no other event occurred outside the registry’s reporting definition or observation period. The posted figures describe what the registry reports, not a complete causal safety assessment.

13. What the Result Does, and Does Not, Mean

The study addresses a relevant question about donor CD8+ memory T-cell infusion after non-myeloablative allogeneic hematopoietic cell transplantation. Its primary endpoint is clearly defined: achievement of at least 95% donor cells in either the CD3+ blood cell lineage or whole blood within 90 days after infusion. The registry record also identifies the intended patient group and describes the timing of the infusion.

However, the result needed to answer the primary question—the number of participants who achieved full-dose donor chimerism—is not reported in the registry record. As a result, the posted information cannot show how often the endpoint occurred or how precisely it was estimated. The completed status does not substitute for a reported outcome value.

The single-group design also limits what even a complete within-group result could establish. It could describe outcomes among those receiving the infusion, but without a concurrent comparator it could not isolate the infusion’s effect from the transplant, other treatment components, or factors related to participants’ disease and clinical course. This is a design limitation, not a claim that the intervention did or did not affect outcomes.

The safety figures provide some information about serious events, other events, and deaths, but do not give the context needed to assess causation or fully characterize risk. The registry lists multiple secondary outcomes, but their estimates and analysis methods are not reported in the information available.

Therefore, the posted record supports a description of the trial’s design and intended primary measure, together with limited safety reporting. It does not support a conclusion that the infusion improves outcomes, fails to improve outcomes, or has a particular causal safety profile. Those judgments require outcome results, uncertainty estimates, and a suitable comparative basis.

14. Why This Trial Matters Statistically

This trial is statistically notable because its primary measure is a clearly thresholded, participant-level endpoint in a single-group study. The definition specifies both the donor-cell threshold and the observation window, which makes the intended classification understandable. At the same time, the primary endpoint count is not reported in the registry record, so the key descriptive result is unavailable.

The design highlights the distinction between measuring an outcome and estimating a treatment effect. A single-group study can document what happened among participants who received the intervention. It cannot, by itself, show how those outcomes differ from what would have happened under another strategy. This distinction is particularly important when the intervention follows a transplant and is delivered alongside other care described in the record.

The record also illustrates why a completed trial is not necessarily a fully interpretable trial record. Enrollment and status provide study context, while outcome values, analysis methods, denominators, and uncertainty estimates are needed to evaluate findings. The posted safety counts offer some information, but event attribution and overlap are not described.

Finally, the registry lists multiple outcomes without reporting the statistical plan or multiplicity handling. In general, prespecifying how primary and secondary outcomes will be interpreted helps readers distinguish confirmatory questions from exploratory ones. Here, those methods are not reported in the registry record. The trial’s statistical contribution therefore lies chiefly in the question and endpoint it defines; its posted results do not permit a quantitative answer to that primary question.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The most consequential limitation of the posted record is that it does not report the count of participants who achieved the primary endpoint. Although the endpoint is defined by a donor-cell threshold and timing window, the observed result and its precision cannot be assessed.

The study is single-group, with no randomized comparator. This limits causal interpretation even if within-group outcomes were available, because outcomes could reflect the transplant, other listed therapies, participant characteristics, or the infusion. The record does not report a control group or a comparative analysis.

Enrollment is listed as 18, but the registry record does not specify endpoint-specific denominators, participant flow, completeness of follow-up, or how missing measurements were handled. It also does not report analysis populations, the statistical analysis plan, the trial’s sample-size justification, or interim analyses. Thus, the posted information does not establish how the study’s planned analyses were intended to address uncertainty or incomplete data.

Eight outcome measures are listed, while no analyses are reported. Results and methods for the secondary outcomes are not available in the information described. The record does not explain any multiplicity strategy, outcome hierarchy beyond the designated primary measure, or criteria for interpreting secondary findings.

Safety reporting is limited to summarized counts and selected serious-event terms. Timing, attribution, event narratives, and overlap between categories are not reported. These constraints limit assessment of both efficacy-related outcomes and the safety profile. The registry’s completed status does not resolve these reporting gaps.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: CD8+ Memory T-Cells as Consolidative Therapy After Donor Non-myeloablative Hematopoietic Cell Transplant in Treating Patients With Leukemia or Lymphoma — NCT02424968.
• Registry record last updated 2021-06-24; status last verified 2021-05.
• Results first posted 2021-06-24.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

The registry record describes a completed phase 2, single-group trial of allogeneic CD8+ memory T-cell infusion after non-myeloablative allogeneic hematopoietic cell transplantation. Planned enrollment was 18, and the infusion was scheduled 30 to 60 days after transplant.

The primary endpoint was full-dose donor chimerism: at least 95% donor cells in either the CD3+ blood cell lineage or whole blood within 90 days after infusion. The registry record does not report the number of participants who met this endpoint. It lists eight outcome measures and zero analyses.

Safety reporting lists 3 participants affected by serious adverse events among 18 at risk, 0 affected by other adverse events among 18 at risk, and 3 deaths. Diarrhea and lung infection are among the top serious-event terms. The record does not provide enough context to infer causation or detailed event-level patterns.

The single-group design supports description of outcomes in the treated group but not a randomized treatment comparison. The registry record does not report the statistical analysis plan, missing-data methods, or analysis populations.