Trial of ID-Specific Donor Vaccinated Lymphocyte Infusion for Patients With Myeloma Relapsing or Failing to Achieve a Complete Remission After an Allogenic Transplant: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Trial of ID-Specific Donor Vaccinated Lymphocyte Infusion for Patients With Myeloma Relapsing or Failing to Achieve a Complete Remission After an Allogenic Transplant (NCT01174082), a randomized phase 2 study. The primary outcome is The Rate of Partial Response(PR) and Complete Response(CR) in Patients Receiving DLI From an ID-specific Vaccinated Donor.

2. Clinical Question

In practical terms, the intended question was whether the recipient response outcome could be observed after donor vaccination and DLI, and whether the strategy using myeloma-specific vaccination could be distinguished from non-specific vaccination. The registry describes PR as a 50% reduction disease including serum monoclonal paraprotein, and CR as absence of the original monoclonal paraprotein in serum and urine. The endpoint combines these two response categories into a count of participants.

The record does not establish a usable head-to-head estimate from the posted results. It reports a recipient count of 1 with a value of 1, and a donor count of 0 with a value of 0. Donors are described as supplying the cell product and as not being analyzable for response, so the donor result is not a recipient comparison group. The randomized label and the single-group model are both present in the registry record; how those design labels relate to the posted result is not reported in the registry record.

3. Trial Design

This is registered as a phase II study sponsored by M.D. Anderson Cancer Center. The registry record identifies randomized allocation and triple masking, while also classifying the model as single group. Enrollment is 2. The study status is terminated. The start date is 2010-07-20 and primary completion is listed as 2017-02-23. The reason for termination is not reported in the registry record.

The intervention descriptions outline donor vaccination with KLH or KLH-id before lymphocyte collection. Donors in the KLH group were described as receiving KLH-only vaccine at weeks -8, -6 and -2 before collection; the KLH-id donor description uses the same listed weeks. The recipient arm descriptions say patients received the same vaccine type as their donors after DLI on the same day. GM-CSF, apheresis and DLI also appear in the intervention list. The registry record does not give a complete account of how each intervention was assigned or administered to every participant.

Although allocation is labeled randomized, the posted primary outcome is not reported as two randomized recipient groups. The outcome entries are Recipient and Donor; the recipient entry has n 1.0 and value 1.0, while the donor entry has n 0.0 and value 0.0. The record explains that donors had no disease and could not be analyzed for response. Therefore, those two entries should not be read as randomized treatment arms with comparable outcome populations.

The registry record describes a planned response observation period from DLI up to 5 years post DLI. It does not report the actual follow-up duration for the recipient, a participant flow account, the number assigned to each donor vaccination strategy, or an account of withdrawals. Detailed protocol procedures and the statistical analysis plan are not reported in the registry record. The combination of a very small enrollment, terminated status, and no posted analyses leaves the design's intended randomized comparison distinct from the result that can be interpreted from the record.

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

Analysis population for the primary outcome, as defined in the registry: The donor participant was to supply the cell product to manufacturer the vaccine. The donor has no disease and therefore cannot be analyzed for a response.

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 The Rate of Partial Response(PR) and Complete Response(CR) in Patients Receiving DLI From an ID-specific Vaccinated Donor, measured over DLI up to 5 years post DLI.

The primary endpoint is the rate of partial response (PR) and complete response (CR) in patients receiving DLI from an ID-specific vaccinated donor. The registry lists the unit as participants and the result type as a count of participants. In other words, the posted value is a count, not a response percentage or a time-to-event estimate. The endpoint combines PR and CR rather than posting separate counts for each category.

The registry defines PR as a 50% reduction disease including serum monoclonal paraprotein. It defines CR as absence of the original monoclonal paraprotein in serum and urine. These definitions specify changes in disease-related laboratory findings used by the endpoint; the record does not provide further assessment rules, confirmation requirements, or the timing of response evaluations within the stated observation window.

A combined response endpoint can summarize whether a participant met either of the listed response categories. It is used to describe the primary outcome of interest, but a count alone does not show the precision of an estimated response probability or establish a contrast between treatment strategies. In this record, the recipient count is 1 with a value of 1. The record does not report a confidence interval, a separate PR count, or a separate CR count. It also does not report an analysis linking the result to a comparison between the donor vaccination approaches.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a count endpoint represent?

The primary result is presented as a count of participants meeting a combined PR-or-CR endpoint. A count answers how many qualifying participants were recorded in the analyzed group. A response proportion would relate that count to an eligible denominator, while an effect estimate would compare outcomes across groups or against a specified reference. Those are distinct summaries. The registry identifies the unit as participants and posts a count, so it should be read as a count rather than as a percentage or comparative effect.

The recipient entry has n 1.0 and value 1.0. With such a small posted recipient group, the result has little information about the range of outcomes that might occur in a larger population. A single observed outcome cannot by itself show whether the same result would be common, uncommon, or variable in other recipients. The registry does not report a confidence interval or another measure of uncertainty for this endpoint.

What would a confidence interval add?

In general, a confidence interval expresses the statistical uncertainty around an estimated quantity under a specified analysis. It is not a guarantee that the true value lies in a particular interval, and it does not repair limitations in the underlying design or data. For a small dataset, an interval would ordinarily be expected to reflect substantial uncertainty. No interval for the primary result is reported in the registry record, so the degree of statistical precision cannot be quantified from the posted outcome.

Why does the design label matter?

Randomization is generally used to create groups whose baseline characteristics are balanced on average, supporting a fair comparison of assigned strategies. That purpose requires outcome information for the randomized groups and an analysis that respects the assignment. Here, the registry labels allocation randomized, but its model is single group and the primary outcome entries are Recipient and Donor. The donor population is specifically described as unable to contribute a response outcome. The posted information therefore does not demonstrate a randomized recipient comparison. It also does not identify how many recipients received each donor vaccination strategy or show their outcomes separately.

Masking generally means that specified people involved in a study are not aware of treatment assignment, which can reduce some forms of bias. The registry labels masking triple, but the identities of the masked parties and the procedures used to maintain masking are not reported in the registry record. The label alone cannot show how masking operated in practice or whether it affected outcome assessment.

What can be said about sample-size illustrations?

Planning illustrations based on an observed effect are not observed power and are not evidence that the trial was adequately designed. The record describes an illustration approach using a two-sided significance level 0.05, equal allocation between two arms, a normal approximation, and effect fractions relative to the observed effect. Such calculations are hypothetical planning exercises. They do not describe the actual enrollment, do not establish that a target effect is clinically important, and do not show the probability that the completed study detected an effect.

Because the posted recipient outcome is a single count and a recipient comparison is not reported, any planning exercise anchored to an observed effect has limited interpretive value. The protocol's sample-size justification, target difference, assumptions used at the design stage, and any revision to those assumptions are not reported in the registry record. Enrollment 2 is the registered enrollment figure, not evidence that the intended sample size was achieved or that a planned statistical power was realized.

How should missing analyses be understood?

The registry reports zero analyses and does not post an inferential result for the primary endpoint. This means that a reader cannot assess a model specification, standard error, confidence interval, p-value, or adjusted comparison from the record. The absence of a posted analysis is not equivalent to evidence of no effect. It means that the registry record does not provide those statistical results. No recalculated comparison is warranted from the listed entries because a comparable recipient contrast is not reported.

General statistical interpretation also depends on the analysis population, handling of missing outcomes, and exact endpoint assessment procedures. Those details are not reported in the registry record. Without them, the count can be described, but the basis for inclusion, the consequences of missing observations, and the degree to which the posted result follows the intended analysis cannot be evaluated.

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("Recipient")
n      <- c(1)
events <- c(1)
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  Recipient  1  1  100.0  2.5  100.0  20.7  100.0  15.8  98.7

11. Multiplicity, Missing Data and Analysis Populations

The registry posts one outcome measure and zero secondary outcome measures. It does not report an analysis of the primary endpoint. Whether the protocol included additional outcomes or planned comparisons beyond those posted is not reported in the registry record. The multiplicity plan, including any adjustment for testing multiple outcomes or comparisons, is also not reported in the registry record.

Multiplicity matters when several statistical questions are tested: the chance of at least one apparently notable result can increase as the number of tests grows. General methods for addressing multiplicity include pre-specifying a primary comparison, adjusting significance thresholds, or interpreting secondary analyses as exploratory. The record does not establish which, if any, approach was planned. Since no inferential analyses are posted, there are no reported p-values to evaluate for multiplicity.

Information on missing data and participant disposition is limited. Enrollment is 2, while the posted outcome entries show Recipient n 1.0 and Donor n 0.0. The record does not explain those entries through a participant flow account, nor does it describe withdrawals, exclusions, unavailable endpoint measurements, or the handling of missing outcomes. The reason for termination is not reported in the registry record.

The analysis population is not described beyond the primary endpoint's note that donors posted the cell product and could not be analyzed for response. The registry does not state whether the recipient result reflects an intention-to-treat, per-protocol, or other analysis population. These omissions limit the ability to assess selection after assignment and to determine how completely the posted result represents the intended study population.

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 non-serious adverse event terms

The registry reports serious adverse event figures separately for Recipient and Donor. Each group has 1 participant at risk for serious adverse events, and each has 0 affected by serious adverse events. No serious adverse event terms are listed. The record also reports other adverse event figures: 1 at risk and 1 affected in each group.

Deaths are reported as 1 in the Recipient group and 0 in the Donor group. These are descriptive counts. The registry record does not give a cause, timing, relationship to an intervention, or explanation for the death, so none should be inferred from the count alone. The posted figures do not establish that an intervention caused an adverse event or death.

Safety counts need to be read alongside the population represented and the event definitions. With only 1 participant at risk in each group, these figures offer very limited information about how often events might occur in a larger population. The registry does not report event-level narratives, severity grades beyond the serious-event category, exposure duration, or additional analysis of the safety data. The absence of listed serious event terms is not a substitute for a complete assessment of safety; it describes what is posted in the registry record.

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

The posted result documents a recipient count of 1 with a value of 1 for the combined partial-response and complete-response endpoint. It also records a donor count of 0 with a value of 0, while explaining that donors did not have disease and could not be analyzed for response. These entries are descriptive and should not be read as a comparison of two treatment groups.

The result does not provide a response rate with a reported uncertainty interval, a separate count for PR and CR, or an estimate comparing the KLH-id and KLH donor vaccination approaches. The randomized label does not resolve this limitation because the record does not post recipient outcomes separately for the strategies. No analysis or p-value is reported. As a result, the registry record does not quantify a treatment effect or its precision.

The study's phase II designation and its stated objective identify the research question, not an answer to that question. The record describes intended donor vaccination, lymphocyte collection, DLI, and recipient vaccination. The outcome count shows what was posted for the primary endpoint, but the small enrollment and incomplete comparative information restrict what can be inferred beyond that count.

These results do not establish that the approach changes response outcomes, nor do they establish that it has no effect. They cannot determine the likelihood of response for other patients, distinguish the two response categories, or support a reliable comparative conclusion. The safety counts also should not be treated as evidence of causation or as a comprehensive characterization of risk. They report limited event counts, without the contextual information needed to attribute an event to treatment.

In statistical terms, the main distinction is between observing a count and estimating a generalizable treatment effect. The record supports describing the count, but does not report the comparison, precision, or analysis needed for broader claims. Further details about the intended and actual analysis, participant disposition, and outcome assessment are not reported in the registry record.

14. Why This Trial Matters Statistically

This trial matters statistically because its registry labels create an important distinction between intended design and interpretable posted evidence. The record identifies randomized allocation and triple masking, but also classifies the model as single group. Donor vaccination strategies are described, yet the primary outcome entries do not provide separate recipient results by strategy. Recognizing that distinction prevents the randomized label from being mistaken for a reported randomized comparison.

The endpoint is a combined count of participants with PR or CR, and the posted recipient entry is based on n 1.0. A single observation can document what was recorded for that participant, but offers little basis for estimating variability across patients or making a comparative inference. No confidence interval or statistical analysis is reported. This illustrates why effect estimates and their uncertainty matter in addition to outcome counts.

The record also highlights why enrollment and analysis populations must be interpreted together. Enrollment is 2, while the posted outcome entries distinguish a recipient from a donor, and the donor is explicitly described as not eligible for response analysis. Without a participant flow account or explanation of the analysis population, the relationship between registered enrollment and the posted endpoint result is not clear.

Finally, the terminated status and absent analysis emphasize the difference between a study's question and the evidence available to answer it. General planning calculations do not replace an adequately reported analysis, and hypothetical sample-size illustrations are not observed power. The record leaves the protocol's sample-size justification, multiplicity plan, missing-data procedures, and reason for termination unreported. Careful statistical reading therefore focuses on what is documented, distinguishes descriptive results from comparisons, and keeps uncertainty visible.

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 immediate limitation is the very small registered enrollment of 2 and the single posted recipient outcome entry. The count cannot characterize the range of response outcomes in a broader population, and the registry does not report a confidence interval or inferential analysis. The donor entry is not an appropriate response comparator because the record states that donors had no disease and could not be analyzed for response.

The registry's design descriptions are difficult to reconcile from the posted information: allocation is labeled randomized, masking is labeled triple, and the model is labeled single group. The donor descriptions identify KLH and KLH-id vaccination strategies, but outcomes are not posted separately for recipient groups corresponding to those strategies. The randomization process, assignment counts, masking details, and realized treatment allocation are not reported in the registry record.

The record gives a response window of DLI up to 5 years post DLI but does not report the actual follow-up time or the timing and frequency of response assessments. It combines PR and CR, with no separate category counts. Further assessment procedures, confirmation rules, and endpoint adjudication details are not reported in the registry record.

Participant flow, withdrawals, missing measurements, analysis population, and handling of missing data are not reported. The reason for termination is also not reported in the registry record. No statistical analysis, p-value, or uncertainty estimate is posted. Together, these gaps prevent evaluation of the planned comparison, precision, risk of selection bias, or reasons the study stopped. The safety record is similarly limited: event counts are posted, but causes, timing, intervention relationship, and detailed event descriptions are not.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Trial of ID-Specific Donor Vaccinated Lymphocyte Infusion for Patients With Myeloma Relapsing or Failing to Achieve a Complete Remission After an Allogenic Transplant — NCT01174082.
• Registry record last updated 2018-06-01; status last verified 2018-05.
• Results first posted 2018-06-01.
• 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 phase II study of ID-specific donor vaccinated lymphocyte infusion for patients with myeloma relapsing or failing to achieve complete remission after allogenic transplant. M.D. Anderson Cancer Center is listed as sponsor. The record identifies randomized allocation, triple masking, and a single-group model. Enrollment is 2; status is terminated.

The primary endpoint is the count of participants with partial response or complete response after DLI, with a listed timeframe of DLI up to 5 years post DLI. The posted recipient entry is n 1.0 and value 1.0. The donor entry is n 0.0 and value 0.0, and the record says donors could not be analyzed for response. Zero analyses and zero secondary outcomes are posted.

The registry also reports serious adverse event counts, other adverse event counts, and deaths, but does not provide event attribution or a detailed safety analysis. The analysis plan, sample-size justification, missing-data procedures, reason for termination, and a recipient comparison by donor vaccination strategy are not reported in the registry record.