Autologous and Allogeneic Transplant for Relapsed Lymphoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Autologous and Allogeneic Transplant for Relapsed Lymphoma (NCT00802113), a non-randomized phase 1, phase 2 study. The primary outcome is Total Number of Subjects With a Complete Response (CR) Following Myeloablative Conditioning (MAC) and Autologous Stem Cell Transplantation (AutoSCT).

2. Clinical Question

3. Trial Design

The registry identifies this as a Phase 1 and Phase 2 study with non-randomized allocation, no masking, and a single-group model. Enrollment is listed as 30. The study status is completed; the start is recorded as 2003-06 and primary completion as 2014-10. These registry dates describe the study record, not the timing of any particular participant's treatment or follow-up.

The proposed sequence combined myeloablative therapy and autologous peripheral blood stem cell transplantation with a subsequent reduced intensity allogeneic transplant and post-transplant adoptive cellular immunotherapy. The registry's brief summary states that the sequence was intended to be well tolerated in patients with refractory or recurrent non-Hodgkin's lymphoma and Hodgkin's disease. This stated aim is not itself evidence of tolerability or of a treatment effect.

The arm descriptions distinguish participants with a matched family donor from those without one. Arm A is labeled Family Donor; its description mentions fludarabine and busulfan before infusion of donor stem cells. Arm B is labeled Unrelated Cord Blood or Adult; its description mentions fludarabine, busulfan, and anti-thymocyte globulin and a search for an appropriate unrelated donor. The registry descriptions are incomplete, so further details of the regimens and pathways are not reported in the registry record.

Because allocation was non-randomized and the study model is single-group, the arm labels should not be interpreted as randomized treatment groups. The registry explicitly cautions that the arms are not a like-for-like control comparison. The planned enrollment and completed status do not establish how many participants contributed data to the primary endpoint or to each safety measure. The registry record does not report those analysis populations or a primary endpoint result.

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.

Before assignment: Out of the 30 enrolled subjects, only 23 subjects received both MAC and AutoSCT and Reduced Intensity Conditioning (RIC) and allogeneic hematopoietic cell transplantation (AlloHCT). 7 subjects did not complete the transplantation and therefore were not included into data analysis.

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 Total Number of Subjects With a Complete Response (CR) Following Myeloablative Conditioning (MAC) and Autologous Stem Cell Transplantation (AutoSCT), measured over Up to 1 year post-transplantation.

The primary endpoint is the total number of participants with a complete response following myeloablative conditioning and autologous stem cell transplantation. Its timeframe is up to 1 year post-transplantation. A complete response is defined in the registry as complete resolution of B symptoms—weight loss, night sweats, and fever—and normalization of all sites of disease based on physical examination, bone marrow biopsy, and imaging studies.

This endpoint represents a participant count, rather than a continuous measurement or a time-to-event measure. A response count can describe how many participants met the registry's stated disease-response definition within the specified timeframe. It is clinically interpretable as a categorical outcome, but its meaning depends on how response was assessed, how missing or unevaluable assessments were handled, and which participants were included in the analysis. Those details are not reported in the registry record.

The endpoint is used to assess disease response after the autologous transplant stage of the sequence. It is distinct from the registry's other listed outcomes, which include time to neutrophil and platelet engraftment, grade II-IV acute graft-versus-host disease, and transplant-related mortality. The registry record describes the endpoint definition but does not report the number of participants with a complete response, an estimate of response frequency, uncertainty around that estimate, or a comparison with another treatment.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes the primary endpoint as a count of participants with an event: in this case, meeting the defined complete-response criteria. For a binary endpoint, each participant is classified according to whether the event occurred under the study's assessment rules. The count is meaningful only alongside the number of participants assessed and the rules used to handle participants whose outcome is unknown or unevaluable. The registry record does not report the primary count, its denominator, or those handling rules.

What a confidence interval would convey

General statistical explanation: a confidence interval expresses uncertainty around an estimated quantity. For a response outcome, an interval around a response proportion would help readers understand how much precision the observed data provide. A narrow interval generally indicates greater precision than a wide interval, while neither width nor the estimate alone resolves limitations in study design. No confidence interval for the primary endpoint is reported in the registry record.

What a comparison can establish

General statistical explanation: a randomized comparison can help distinguish treatment-group differences from differences that existed before treatment, because random allocation is designed to balance prognostic factors on average. This study is registered as non-randomized and single-group. The two donor-pathway labels therefore should not be read as a randomized comparison, and the registry explicitly cautions that the arms are not a like-for-like control comparison. The record does not report an adjusted or unadjusted treatment comparison.

In a single-group study, outcome counts can describe what occurred among assessed participants, but they do not on their own identify what would have happened to the same participants under a different treatment. This is a general limitation of interpreting outcomes without a suitable comparator. It is especially important not to infer a causal treatment effect from a response count when the registry does not report that count or a comparator outcome.

Outcome definitions and assessment

The response definition combines symptom resolution with normalization of disease sites using physical examination, bone marrow biopsy, and imaging. Such a composite definition requires consistent assessment and clear rules for the timing and interpretation of each component. The registry gives the definition and a timeframe of up to 1 year post-transplantation, but does not report further assessment procedures, adjudication methods, or the handling of conflicting or incomplete component assessments.

The registry lists time to neutrophil engraftment and time to platelet engraftment as secondary outcomes. General statistical explanation: time-to-event outcomes require both an event definition and a way to account for participants who have not had the event by the end of observation or whose follow-up ends earlier. The registry record does not report analysis methods, censoring rules, or results for these outcomes. The listed adverse outcomes are counts, and they likewise require clear denominators and ascertainment periods to support interpretation.

What is not available for statistical review

The registry record does not report a statistical analysis plan, a sample-size justification, interim analyses, or results of statistical tests. It also does not report outcome-specific analysis populations, methods for missing data, or an explanation of how the enrollment figure relates to participants included in each outcome. Without these details, readers cannot verify whether the endpoint analysis followed a prespecified plan or determine how incomplete follow-up may have affected the results.

General statistical explanation: statistical significance and clinical importance are different ideas. A test result, if reported, would address compatibility with a specified statistical model and null hypothesis; it would not by itself establish the importance of an outcome or remove limitations of design. Here, no primary endpoint test result or estimate is reported, so statistical significance and precision for the response outcome cannot be assessed from the registry record.

The registry lists seven outcome measures and zero analyses. This means the record as described does not include posted analysis results for those outcome measures. It does not establish why results are absent. The appropriate interpretation is limited to the registered design, outcome definitions, and the posted counts that are available, without filling in unreported methods or results.

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("Arm A - Family Donor", "Arm B - Unrelated Cord Blood or Adult")
n      <- c(6, 17)
events <- c(4, 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  Arm A - Family Donor  6  4  66.7  22.3  95.7  30.0  90.3  29.0  90.1
ARM  Arm B - Unrelated Cord Blood or Adult  17  12  70.6  44.0  89.7  46.9  86.7  46.5  86.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists seven outcome measures, including the primary complete-response count and secondary outcomes related to engraftment, graft-versus-host disease, and transplant-related mortality. It does not report a multiplicity strategy, such as a hierarchy of hypotheses or an adjustment for testing multiple outcomes. The registry record also lists zero analyses, so it does not provide reported statistical tests across these outcomes. Without a reported analysis plan, the approach to multiple testing is not reported in the registry record.

The record does not describe missing-data procedures, the number of participants with missing or unevaluable primary assessments, or the handling of incomplete follow-up. It also does not define the primary endpoint analysis population. The enrollment figure of 30 should not be assumed to be the denominator for the endpoint because the record does not report that denominator.

These omissions matter because outcome estimates can depend on who is included and how unknown outcomes are treated. General statistical explanation: excluding participants with unavailable assessments, counting them as nonresponders, or using a model to address missingness can lead to different estimates; the appropriate approach depends on the study design and prespecified analysis plan. None of those approaches is specified in the registry record. Consequently, the complete-response result and its sensitivity to missing data cannot be evaluated from the registry information.

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.

The registry reports serious and other adverse-event figures for Arm A - Family Donor and Arm B - Unrelated Cord Blood or Adult. For Arm A, 6 participants are listed as at risk for serious events and 6 for other events; zero are listed as affected in either category, and deaths are listed as 2. For Arm B, 17 are listed as at risk for serious events and 17 for other events; zero are listed as affected in either category, and deaths are listed as 6.

These are the registry's posted counts, not a complete account of all safety outcomes. The record does not report serious-event terms among the top terms, and the figures do not explain event timing, duration, relatedness to treatment, or the relationship between deaths and adverse-event categories. The number of participants at risk for these safety counts is not necessarily the denominator for other outcomes. The registry does not explain how these safety denominators relate to the enrollment figure of 30.

The groups are not randomized control groups, and the registry cautions that they are not a like-for-like comparison. Their counts therefore should not be interpreted as evidence that one donor pathway had a different safety profile. These figures describe what is recorded, with substantial limits on what can be inferred from them.

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

The registry record describes a study of sequential myeloablative therapy and autologous stem cell transplantation followed by reduced intensity allogeneic transplantation and post-transplant adoptive cellular immunotherapy. It identifies complete response after the autologous transplant stage as the primary endpoint and provides a response definition and timeframe. These details explain what the study set out to assess, but they do not reveal the observed response outcome.

The primary response count and statistical analysis are not reported in the registry record. Consequently, the record does not establish how many participants achieved complete response, how precise an estimate would be, or whether outcomes differed from an alternative treatment. No claim about the treatment's effectiveness or lack of effectiveness can be drawn from the missing primary result.

The design further limits causal interpretation. Allocation was non-randomized, the model was single-group, and the two donor-based pathways are not described as a like-for-like control comparison. Any differences between those pathways could reflect factors associated with donor availability or other differences; the record does not report an analysis that addresses such factors.

The safety counts offer a narrow description of serious and other adverse events and deaths in the named groups. They do not provide event details or a comparative safety analysis. The registry also lists secondary outcomes, but their results are not reported in the record. Overall, the available information characterizes the study design and some recorded safety counts, while leaving the primary response result, outcome precision, and broader statistical interpretation unresolved.

14. Why This Trial Matters Statistically

This trial raises several important statistical issues for clinical research. First, its primary endpoint is a participant-level response count with a defined assessment timeframe. Interpreting such a result requires both the number meeting the response definition and the number assessed. The registry record provides the endpoint definition but does not report either the primary count or its analysis denominator.

Second, the study's non-randomized, single-group model illustrates why a treatment sequence and an outcome occurring afterward do not alone establish a causal effect. The donor-based pathways are not randomized control groups, and the registry warns against treating them as like-for-like comparisons. This makes careful description of the observed outcomes, rather than causal comparison, particularly important.

Third, outcome interpretation depends on reporting the analysis plan, missing-data rules, and outcome populations. Those details are not reported in the registry record. Their absence prevents readers from determining how incomplete assessments were handled or whether the analysis followed a prespecified approach. The record also lists multiple outcomes without reporting a multiplicity strategy or analysis results.

Finally, the safety figures show why counts need context: the registry lists participants at risk, affected counts, and deaths, but provides little information about event characteristics or how the denominators relate to the overall enrollment. The study is therefore a useful example of the distinction between a registered design, posted outcome data, and the evidence needed for a complete statistical interpretation.

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 limitations are specific to the information available for this study. The central limitation is that the primary complete-response result is not reported in the registry record. Although the endpoint definition and timeframe are stated, the response count, denominator, estimate, and uncertainty are absent. The record also lists zero analyses, leaving no reported statistical tests or outcome analyses to evaluate.

The registry gives an enrollment of 30, but does not report how many participants entered each donor pathway, received each treatment stage, completed response assessment, or contributed to each outcome. The available safety denominators are outcome-specific, and their relationship to overall enrollment is not explained. The arm descriptions are incomplete, limiting a full account of the treatment pathways.

Design features also constrain interpretation. The study is non-randomized and single-group, and the registry explicitly says the arms are not a like-for-like control comparison. The record does not report methods for confounding adjustment, a statistical analysis plan, sample-size justification, interim analyses, missing-data handling, or analysis populations. It also does not report a multiplicity approach despite listing seven outcome measures.

For safety, the registry provides counts but not the event terms, timing, duration, attribution, or details of the deaths. The top serious adverse-event terms are not listed. These gaps limit interpretation of both the event counts and any comparison between donor pathways. The available record therefore supports a limited design and count-level description, not a comprehensive efficacy or safety assessment.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Autologous and Allogeneic Transplant for Relapsed Lymphoma — NCT00802113.
• Registry record last updated 2019-03-27; status last verified 2019-03.
• Results first posted 2019-03-27.
• 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

NCT00802113 is a completed, non-randomized, single-group Phase 1 and Phase 2 study sponsored by Columbia University. Enrollment is listed as 30; the study start is recorded as 2003-06 and primary completion as 2014-10. The study concerns refractory or recurrent non-Hodgkin's lymphoma and Hodgkin's disease and describes sequential autologous and reduced intensity allogeneic stem cell transplantation.

The primary endpoint is the count of participants with complete response after myeloablative conditioning and autologous stem cell transplantation, assessed up to 1 year post-transplantation. The registry record does not report the primary result, outcome analyses, or the statistical analysis plan. It lists secondary outcomes for engraftment, acute graft-versus-host disease, and transplant-related mortality. Safety counts are recorded for two donor-based pathways, which are not a like-for-like control comparison.