Immune Reconstitution After Autologous Hematopoietic Stem Cell Transpl for High-Risk Lymphoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Immune Reconstitution After Autologous Hematopoietic Stem Cell Transpl for High-Risk Lymphoma (NCT00569309), a na na study. The primary outcome is Number of Participants Experiencing Immune Reconstitution.

2. Clinical Question

In plain terms, the study was set up to describe immune measures after transplantation in a group receiving the vaccine on the stated schedule. It also lists secondary questions about regulatory T-cells, inflammatory cytokines, and quality of life, including pain and fatigue. These outcomes broaden the study’s focus beyond the primary immune-reconstitution measure, but their results are not reported in the registry record. The study can therefore be read as a planned assessment of post-transplant immune and quality-of-life measures, not as a randomized test of vaccination against a concurrent control.

3. Trial Design

The registry identifies NCT00569309 as a completed, single-group study sponsored by Ohio State University Comprehensive Cancer Center. Enrollment is recorded as 30. The start date is 2007-12-12, and the primary completion date is 2011-07-29. The phase is recorded as NA, allocation as NA, and masking as NONE. These entries describe the registry classification; they do not establish additional design features that are not recorded.

The trial title focuses on immune reconstitution after autologous hematopoietic stem cell transplantation for high-risk lymphoma and myeloma. The brief purpose describes vaccine therapy after transplantation. The intervention description identifies the conjugate vaccine for Streptococcus pneumoniae, Prevnar, with administration planned in weeks 9, 17, and 25 after autologous HSCT. The registry also lists laboratory correlative studies and quality-of-life assessment.

Because the model is single-group and no allocation to a comparison arm is recorded, the design does not support a randomized between-group treatment contrast. Outcomes can be described for the enrolled group, if results are available, but changes over time or comparisons with an external expectation would need a specified analysis and appropriate context. Such details are not reported in the registry record.

The record lists six outcome measures and zero analyses. It names immune, cellular, cytokine, and quality-of-life outcomes, but does not report numeric results for the primary outcome. The protocol’s sample-size justification, recruitment details, analysis populations, and any interim analyses are not reported in the registry record. The posted planning illustration is not a substitute for the study’s own design rationale, and its two-arm assumptions do not describe this single-group design.

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 Number of Participants Experiencing Immune Reconstitution, measured over Up to 2 years.

The primary endpoint is titled “Number of Participants Experiencing Immune Reconstitution.” It is a participant count, rather than a continuous average or a time-to-event measure, and its stated timeframe is up to 2 years. The registry description identifies several immune-response components: response to the conjugate pneumococcal vaccine Prevnar (PCV7), natural killer cell activity against autologous lymphoblastoid cell lines, and CMV and EBV tetramer responses after autologous transplant for myeloma.

As a general statistical matter, a participant count can summarize how many people meet a defined outcome criterion. To interpret that count, readers need to know the operational definition of “experiencing immune reconstitution,” how the listed components contribute to that definition, when assessments occur, and how missing or unevaluable measurements are handled. Those details, including any rule combining the components, are not reported in the registry record. The denominator used for the primary outcome analysis is also not reported.

This kind of endpoint is relevant to the stated study question because the registry is focused on immune measures after transplantation. However, an endpoint’s relevance does not by itself establish the meaning or clinical importance of a particular value. The registry record does not report a primary endpoint count, an uncertainty estimate, or an analysis linking the endpoint to a clinical outcome.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Several statistical ideas help clarify what can and cannot be inferred from this registry record. The distinction between a planned outcome, an observed result, and an analysis is important: an outcome description explains what investigators intended to measure; a result reports the data; and an analysis explains how those data were summarized or compared. The record describes the primary immune-reconstitution outcome, but does not report its numeric result or an analysis.

What does a participant count tell readers?

As a general statistical explanation, a count gives the number of participants meeting a stated criterion. Counts are easiest to interpret when paired with the number assessed and with a clear definition of who qualifies as an event. For a binary participant-level endpoint, the event definition distinguishes participants counted as experiencing the outcome from those not counted. Here, the registry names the outcome and its immune measures, but does not report a specific rule for combining those measures or the analyzed denominator. The enrollment figure alone cannot fill that gap.

A count also does not convey precision on its own. General methods can attach an uncertainty interval to a count or to a proportion, but the appropriate calculation depends on the observed data and the analysis population. No primary count, proportion, or interval estimate is reported in the registry record. Consequently, there is no trial-specific uncertainty estimate to interpret.

Why does the single-group design matter?

In general, a randomized control group can help distinguish differences associated with an intervention from differences that might otherwise occur between groups. This study is recorded as single-group, and allocation is NA; the registry does not describe a concurrent comparator. Its design therefore does not yield a randomized between-group estimate. Descriptive measurements within a group may still be informative, but causal interpretation requires more than observing an outcome after an intervention. Timing alone does not establish that the intervention caused a measured response.

The study concerns patients after autologous transplantation and schedules vaccination during weeks 9, 17, and 25 after transplant. Both the post-transplant setting and the timing of assessment are relevant context for describing the study, but the record does not provide a comparison that separates the vaccine’s contribution from other influences. No adjusted or external comparison is reported.

What is a confidence interval, in general?

A confidence interval is a statistical way to express uncertainty around an estimate under a specified analysis. A narrower interval generally indicates greater precision, while a wider interval indicates more uncertainty. It does not by itself address bias, the suitability of the endpoint, or whether an association is causal. No confidence interval for the primary outcome is reported in the registry record, so these general principles cannot be applied to a trial-specific estimate.

What is the role of a planning illustration?

In general, sample-size planning uses assumptions about the outcome, the effect worth detecting, and the planned design. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a stated fraction of the observed effect. For time-to-event calculations, the assumption describes a total number of events; other calculations are described in participants per arm. These are planning conventions, not observed trial outcomes.

Those assumptions do not match the registry’s single-group design, which has no recorded allocation to two arms. The planning illustration should therefore not be interpreted as the study’s actual design or as observed power. The registry does not report its own sample-size justification, the effect assumed by the investigators, or an analysis of the primary endpoint. No planning count should be presented as an observed result.

Why can multiple outcomes complicate interpretation?

As a general matter, examining several outcomes creates multiple opportunities to identify patterns. If many hypotheses are tested, chance findings can become more likely unless the analysis plan addresses multiplicity. This study lists six outcome measures, but the registry does not report a multiplicity strategy, a hierarchy among outcomes, or inferential analyses. The list alone does not establish that all outcomes were tested or that any result was statistically significant.

Finally, immune endpoints may involve repeated assessments and multiple laboratory measures. General methods for repeated measurements or correlated outcomes depend on the schedule, the data structure, and the prespecified analysis. The registry provides a primary timeframe and descriptions of measures, but does not report the assessment-level data, a statistical model, or rules for handling incomplete measurements. Those methods cannot be inferred from the outcome names.

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("Prevnar")
n      <- c(30)
events <- c(30)
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  Prevnar  30  30  100.0  88.4  100.0  88.6  100.0  88.8  99.9

11. Multiplicity, Missing Data and Analysis Populations

The record lists six outcome measures, including the primary immune-reconstitution count and secondary measures concerning regulatory T-cells, cytokine production, and quality of life. It does not report how the outcomes were prioritized for statistical testing, whether a hierarchy was prespecified, or whether any adjustment for multiple testing was planned. The number of outcome measures does not establish the number of hypotheses actually tested; the registry records zero analyses.

As a general statistical matter, multiplicity arises when investigators make several comparisons or test several outcomes. Without a stated strategy, readers cannot determine whether the interpretation of any hypothetical finding accounts for the opportunity to observe chance patterns across outcomes. Since numeric outcome results are not reported in the registry record, there are no trial-specific tests to evaluate for multiplicity.

Missing-data handling is also not reported. The registry does not state how incomplete immune assessments, missed visits, or unavailable quality-of-life measures were handled. It does not report an analysis population for the primary outcome or whether participants were included according to a specified analysis principle. Enrollment of 30 does not establish how many contributed data to each outcome.

These omissions matter because incomplete measurements can affect both the denominator and the interpretation of an outcome count. In general, methods may describe the amount and reasons for missingness and specify how analyses handle it; such details cannot be inferred. The registry also does not report an interim-analysis plan or stopping rules. Their presence or absence in the study protocol is not reported in the registry record.

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 5% reporting threshold.

Most frequent non-serious adverse event terms

The registry’s adverse-event summary lists 30 participants at risk for serious events and zero affected in that category. For the category labeled “other,” it lists 30 at risk and 30 affected. The death count is zero. No top serious-event terms are listed. These are the reported figures and categories; the record does not define the “other” category in further detail.

As a general interpretive point, “at risk” describes the recorded denominator for a category, while “affected” is a count. Those entries should not be conflated with the primary immune-reconstitution endpoint. Nor do the category labels alone explain the event types, timing, duration, severity, or relationship to vaccination or transplantation. The registry record does not report those details or an event-level breakdown.

The serious-event summary is limited to what is recorded in those fields. Zero affected in the serious-event category does not establish that no adverse experiences of any kind occurred; the other-event category records affected participants. Conversely, the other-event count does not indicate the nature or clinical importance of those events. These figures are a registry summary, not a complete safety interpretation.

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

The record describes a completed study designed to assess immune reconstitution after autologous stem cell transplantation in the setting of high-risk lymphoma or myeloma, with a pneumococcal conjugate vaccine intervention. It identifies the primary endpoint, its timeframe, the planned vaccine schedule, and additional immune and quality-of-life outcomes. This information helps define the questions the study addressed.

What the record does not establish is the observed primary outcome. No numeric count of participants experiencing immune reconstitution, denominator for that endpoint, or uncertainty estimate is reported. Without those results, the magnitude and precision of any immune-reconstitution finding cannot be assessed. The outcome descriptions should not be mistaken for evidence that a response occurred in a particular number of participants.

The single-group design also limits what can be concluded about cause and effect. The registry does not describe a randomized comparison group, so it does not provide a controlled estimate of the effect attributable to vaccination. Any observed post-transplant immune measure, if reported elsewhere, would need interpretation in light of the study design and relevant sources of change. The registry record itself does not report such an analysis.

The secondary outcomes signal broader interests, including regulatory T-cells, inflammatory cytokines, pain, fatigue, and quality of life. They do not establish improvement or deterioration. Likewise, the adverse-event counts are limited to the recorded categories and do not give a detailed account of event types or attribution. The posted information supports understanding the study’s scope and design, but not a claim that the intervention did or did not produce a clinical benefit or harm.

In statistical terms, the distinction is between what was planned and what was demonstrated in the record. The planned endpoint and outcome descriptions are available; primary and secondary analysis results are not reported in the registry record. That boundary should guide any interpretation.

14. Why This Trial Matters Statistically

This study is statistically notable because its central question is framed around immune reconstitution after transplantation, while the registry records a single-group design. That combination makes clear the difference between describing an outcome in a treated group and estimating an intervention effect against a concurrent comparison group. A descriptive study can address what was measured in its participants, but a controlled contrast requires a suitable comparator and a reported analysis.

The primary outcome also illustrates why operational definitions matter. “Immune reconstitution” is represented through several kinds of immune measurements, including vaccine response, natural killer cell activity, and CMV and EBV tetramer responses. A reader needs to know whether these components are considered separately or combined, and what qualifies a participant as experiencing the endpoint. The registry record does not report those decision rules or a numeric outcome result.

The record further lists laboratory and quality-of-life questions alongside the primary endpoint. This breadth can be useful for characterizing a study’s intended scope, but multiple outcomes require transparent priorities and analysis methods for clear interpretation. The record lists six outcome measures and zero analyses, without a reported multiplicity plan or results.

Finally, the planning assumptions describe a two-arm illustration even though the recorded study model is single-group. This highlights a general lesson: planning outputs must be matched to the design they represent. A hypothetical two-arm calculation is not a justification for this study’s enrollment, a result, or evidence of observed power. The registry does not report the trial’s own sample-size rationale. These distinctions are important when readers encounter design information without corresponding outcome analyses.

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.

Several trial-specific limitations follow directly from the registry record. The design is single-group, with allocation recorded as NA and no comparator described. Consequently, the registry does not present a randomized control comparison. The record also does not report a numeric primary endpoint result, denominator, or uncertainty estimate, which prevents assessment of the magnitude or precision of the stated immune-reconstitution outcome.

The endpoint description includes multiple immune measures, but the record does not state how those measures are combined into a participant-level outcome or what threshold defines immune reconstitution. It does not report the schedule of endpoint assessments beyond the timeframe of up to 2 years, or explain how incomplete measurements are handled. Analysis populations and reasons for missing data are not reported in the registry record.

The registry lists six outcomes and zero analyses. It does not report an analysis plan, multiplicity approach, sample-size justification, interim analyses, or the statistical methods used for the outcomes. The planning assumptions describe a two-arm illustration, which is not the recorded single-group design; the trial’s own planning rationale is not reported.

The adverse-event summary is also limited: the record does not give event descriptions for the “other” category, event timing, severity details, attribution, or a fuller event-level breakdown. Finally, the registry lists Small Intestine Cancer among the conditions while its title and brief purpose focus on high-risk lymphoma and myeloma. Eligibility criteria and the relationship of that listed condition to the analyzed population are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Immune Reconstitution After Autologous Hematopoietic Stem Cell Transpl for High-Risk Lymphoma — NCT00569309.
• Registry record last updated 2018-03-12; status last verified 2018-03.
• Results first posted 2018-03-12.
• 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

NCT00569309 is recorded as a completed, single-group study sponsored by Ohio State University Comprehensive Cancer Center. Enrollment is 30. Its start date is 2007-12-12, and its primary completion date is 2011-07-29. The registry lists no masking and records phase and allocation as NA.

The study concerns immune reconstitution after autologous hematopoietic stem cell transplantation, with Prevnar (PCV7) scheduled during weeks 9, 17, and 25 after transplantation. The primary endpoint is the number of participants experiencing immune reconstitution, assessed up to 2 years. Six outcomes are listed, and the registry records zero analyses.

A numeric primary outcome result and the methods for handling missing data, defining the endpoint, and analyzing outcomes are not reported in the registry record. The design is not a randomized comparison. The adverse-event summary records zero affected in the serious-event category, 30 affected in the other-event category, and zero deaths.