Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor (NCT01350245), a na phase 2 study. The primary outcome is Disease-Free Survival (DFS).

2. Clinical Question

3. Trial Design

This was a phase 2, single-group trial with enrollment of 28 participants. The registry lists the study as completed. Its recorded start was 2010-07, and primary completion was 2013-05. The listed sponsor was Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University. Allocation is marked NA, and masking is recorded as none. These features mean the record describes treatment in a single group rather than allocation between competing study arms.

The study addressed allogeneic hematopoietic stem cell transplantation for patients in remission using partially matched related donors. Participants were treated with the TJU 2 Step Regimen. The intervention list names total body irradiation, donor lymphocyte infusion, cyclophosphamide, mycophenolate mofetil, tacrolimus, and hematopoietic stem cell transplantation. The registry record does not provide enough detail to reconstruct the treatment sequence or determine whether all participants received each listed intervention in the same way.

The eligible disease categories span multiple hematologic malignancies and include aplastic anemia. The arm description states that patients had disease in remission at transplantation and that their diseases would be expected to relapse with standard therapy alone. Counts by diagnosis, age distribution, and other participant characteristics are not reported in the registry record.

The brief summary describes a hypothesis about one-year disease-free survival in relation to historical outcomes after matched sibling transplantation. That historical context does not create a control arm: the registered design is single-group and does not report a concurrent comparison. Randomization, blinding, stratification, and further allocation procedures are not reported in the registry record. The posted results include two outcome measures, but the primary endpoint's observed value and the analyses supporting it are not reported in the registry 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

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 Disease-Free Survival (DFS), measured over 1 year post-transplant.

The primary endpoint is disease-free survival (DFS) at 1 year post-transplant, expressed as a percentage of patients. The registry defines success as being alive and disease free at the 1-year assessment. This is a combined endpoint: a participant must satisfy both the survival and disease-free conditions to count as a success under that definition.

DFS is used to describe an outcome that accounts for both remaining alive and avoiding disease recurrence or persistence, rather than reporting either component alone. In a transplant study for patients whose diseases are in remission, that combination can describe the status of participants at a clinically defined follow-up point. It does not, by itself, show which component accounted for a failure to meet the endpoint.

The registry record states the endpoint definition and timeframe but does not report the observed DFS percentage, the number meeting the success definition, or uncertainty around the estimate. It also does not report how disease status was assessed, how participants without an assessment at the specified time were handled, or whether any additional endpoint rules were applied. Accordingly, the endpoint can be described from the registry definition, but its observed result and precision cannot be evaluated from the reported record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The trial is recorded as single-group and phase 2, with allocation marked NA and masking recorded as none. These design descriptors matter because they determine what kinds of statistical conclusions the data could support. A single-group analysis can estimate an outcome proportion among treated participants, if the result and analysis population are reported. It cannot directly estimate a randomized between-group treatment contrast. A historical benchmark can motivate a study, but it is not a substitute for a concurrent control group.

What does the primary endpoint estimate?

The primary endpoint is a binary status at a specified follow-up time: success means alive and disease free at 1 year post-transplant; failure means not meeting that combined definition. For a binary endpoint, the central summary is often the proportion meeting the definition among an explicitly defined analysis population. The registry labels the unit as a percentage of patients, but does not report the observed percentage, number of successes, or analysis denominator. It is therefore not possible to interpret an estimated proportion or its precision from the record.

A confidence interval, as a general statistical concept, describes a range of values compatible with the data under a stated method and assumptions. It can communicate how much uncertainty surrounds an estimated proportion. A narrower interval generally reflects greater precision, while a wider interval indicates less precision. No confidence interval for the primary endpoint is reported in the registry record, so the precision of the trial's DFS estimate cannot be assessed.

How should the historical benchmarks be read?

The brief summary names 50% or better as meeting the stated criterion for an effective alternative and 75% or better as implying superior efficacy relative to T-replete matched sibling transplantation. These values define the rationale described in the summary; they are not observed outcomes. A comparison between a study proportion and a benchmark requires the observed proportion and an analysis method. Neither the observed DFS result nor a formal comparison with the benchmarks is reported in the registry record.

Even if an outcome estimate were available, historical comparisons would require care. General statistical reasoning is that populations compared across studies may differ in diagnosis, age, disease status, follow-up, and other characteristics. Those differences can influence outcomes independently of treatment. The summary refers to historical patients with similar diagnoses and ages, but the registry record does not report the source, detailed characteristics, or statistical adjustment for such historical data. The strength of any historical comparison therefore cannot be evaluated from this record.

What can the design say about causation?

Randomization is a general method for distributing measured and unmeasured prognostic factors across treatment groups. No randomized comparison is recorded for this trial. With one treated group, an observed outcome proportion would describe the participants' outcomes under the study regimen, but would not alone show what would have happened to the same participants under another regimen. The registry does not report a comparative effect estimate, and a causal treatment effect cannot be derived from the design description alone.

Masking is recorded as none. In general, lack of masking can influence outcomes that rely on judgment or participant reporting, though its relevance depends on endpoint assessment procedures. The primary endpoint combines survival and disease-free status, but the registry does not describe how disease status was evaluated or whether outcome assessors were independent. Those assessment details are not reported in the registry record.

Why do denominators and missing outcomes matter?

For a percentage to be interpretable, readers need to know the numerator, denominator, endpoint definition, and handling of participants whose status is unknown. Enrollment is 28, but that does not establish that all 28 were included in the primary endpoint analysis. Follow-up completeness, losses to follow-up, missing data rules, and the analysis population are not reported in the registry record. In general, excluding participants with missing endpoint status can change an observed proportion if their outcomes differ from those with complete follow-up.

Safety denominators also require definition. The registry records 28 at risk for serious adverse events and 19 affected. It lists serious-event terms with percentages, but does not clarify whether the percentages refer to participants with an event, the share of events, or another convention. General interpretation should distinguish people affected from total event counts, because one participant may experience more than one event. The registry record does not provide enough detail to resolve that distinction for the listed terms.

What statistical plan is documented?

The registry indicates two outcome measures and zero analyses, but does not report the primary endpoint analysis method, the handling of repeated observations, a confidence interval method, or a statistical hypothesis test. The sample-size rationale and any interim analyses are not reported in the registry record. A planning calculation is not an observed outcome, and a sample-size target would not establish the strength of evidence from results. Since the primary result and analysis are absent, no inferential conclusion about the endpoint can be drawn.

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("TJU 2 Step Regimen")
n      <- c(28)
events <- c(22)
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  TJU 2 Step Regimen  28  22  78.6  59.0  91.7  60.5  89.8  60.3  89.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures and zero analyses. It does not identify a statistical analysis plan that explains how outcomes were prioritized beyond labeling DFS as primary, or how multiple outcomes were handled. In general, testing several outcomes can increase the chance of a chance finding if each is assessed separately without an appropriate strategy. Whether that concern applied, and whether any adjustment or hierarchical testing was planned, is not reported in the registry record.

The primary endpoint is defined at 1 year post-transplant, but the record does not report the endpoint's analysis population, number of participants with known status, or missing-data procedures. It is not stated whether all enrolled participants were intended for analysis, whether participants with missing one-year status were counted as failures, or whether another approach was used. These choices can affect an estimated percentage and its interpretation.

The registry record also does not report exclusions after enrollment, protocol deviations, or whether the endpoint was analyzed according to the originally intended treatment assignment. Because the study is single-group, there is no randomized assignment group to compare, but the population used for outcome analysis still needs to be defined. The details needed to assess missingness, analysis-set selection, or sensitivity analyses are not reported in the registry record.

Interim analyses, stopping rules, and any adjustments related to repeated looks at accumulating results are not reported in the registry record. The absence of such information does not establish that these procedures were or were not used; it means they cannot be assessed from the posted record.

12. Safety Results

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

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

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: fever (28.6%), cmv reactivation (14.3%), diarrhea (10.7%), infection (7.1%), hhv-6 infection (7.1%).

The registry records 28 participants at risk for serious adverse events and 19 affected. This is a participant-level summary as labeled in the adverse-event record; it should not be confused with a count of individual events. A person may experience more than one event, and the registry does not provide enough detail to determine the number of distinct serious events or how recurrent events were counted. The death count is not reported in the registry record.

The listed serious-event terms and percentages are fever at 28.6%, CMV reactivation at 14.3%, diarrhea at 10.7%, infection at 7.1%, HHV-6 infection at 7.1%, and hypoxia at 7.1%. These entries describe the registry's reported safety summaries. The record does not specify whether each percentage represents participants affected, the proportion of event reports, or another denominator convention. It also does not report event onset, duration, severity grading beyond the serious-event designation, or attribution to the regimen.

Safety summaries depend on the observation period, event definitions, and completeness of reporting. Those details are not reported in the registry record. The figures therefore should be read as recorded safety data for this study group, not as a comparison with another treatment or as a complete characterization of risk.

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

The registry record establishes that a phase 2, single-group study enrolled 28 participants to evaluate the TJU 2 Step Regimen for patients with hematologic malignancies in remission undergoing transplantation from HLA partially-matched related donors. It also defines the primary outcome as being alive and disease free at 1 year post-transplant. These details clarify the clinical question and the outcome the study intended to assess.

The record does not report the observed one-year DFS result. It therefore does not establish whether the study met the 50% or 75% benchmarks described in its brief summary. It also does not report an estimate of uncertainty around DFS or a formal comparison with historical matched sibling transplant outcomes. Any claim about the direction or size of the primary result would go beyond the registry record.

The design does not include a concurrent control group. Consequently, even an available outcome proportion would describe the treated group rather than isolate the effect of the regimen in comparison with an alternative. Historical context can help frame a research question, but it cannot remove differences between studies unless the relevant data and methods support that comparison. Those details are not reported in the registry record.

The safety record lists serious adverse-event information, including the number affected and several event terms with percentages. Its interpretation is limited by absent details about definitions, event counting, attribution, and observation period. The safety data and missing efficacy result together mean that the registry record supports a description of study design and reported safety entries, but not a complete assessment of benefit, harm, or comparative treatment effects.

14. Why This Trial Matters Statistically

This trial raises statistical issues common to studies of transplantation in heterogeneous patient groups. Its eligible conditions include several hematologic malignancies and aplastic anemia, while the primary endpoint is a single binary status at 1 year post-transplant. The overall result, if reported, would need to be interpreted in light of the population mix and the endpoint's combined definition. The registry does not provide diagnosis-specific results or participant characteristics that would allow readers to explore that context.

The study also illustrates the distinction between a historical benchmark and a controlled comparison. The brief summary frames DFS against historical matched sibling transplantation and names thresholds of 50% and 75%. Those thresholds explain the stated rationale, but the single-group design does not itself provide a concurrent estimate of what would have occurred under matched sibling transplantation. Statistical interpretation should distinguish a prespecified target or benchmark from evidence of a treatment contrast.

Outcome completeness and analysis definitions are equally important. Enrollment of 28 is not necessarily the denominator for one-year DFS; the registry does not report the analysis population, missing-status handling, or endpoint estimate. Without these, neither the point estimate nor its precision can be interpreted. The absence of reported analyses also limits assessment of how the primary outcome was tested and whether other outcomes influenced interpretation.

Finally, the safety entries demonstrate why event rates need clear denominators and counting rules. The record gives serious-event figures but leaves important reporting details unspecified. These gaps do not negate the information that is present; they define the limits of what can be concluded statistically from the registry record.

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 principal limitation for statistical interpretation is that the observed primary endpoint result is not reported in the registry record. The record gives the DFS definition and timeframe, but not the number of participants alive and disease free, the percentage, or an uncertainty interval. It is consequently impossible to assess the primary outcome against the historical benchmarks described in the study summary.

The single-group design has no concurrent control, and the record reports no randomized comparison. The historical comparison mentioned in the brief summary cannot be independently evaluated because the source and detailed characteristics of the historical population, along with any methods for adjustment, are not reported in the registry record. The range of diagnoses further makes the unreported composition of the enrolled group relevant to interpretation.

Enrollment is reported as 28, but the primary endpoint denominator and follow-up completeness are not. The registry record does not describe missing outcome handling, analysis populations, protocol deviations, endpoint assessment procedures, or confidence intervals. It also does not report a statistical analysis method, sample-size justification, or interim-analysis plan.

Safety interpretation is limited by incomplete reporting of event definitions, attribution, time at risk, counting conventions for the listed percentages, and the death count. These limitations mean the posted information supports only a constrained description of study design, endpoint definition, and recorded safety summaries. They do not support a conclusion about comparative efficacy or a comprehensive characterization of safety.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor — NCT01350245.
• Registry record last updated 2025-05-16; status last verified 2025-05.
• Results first posted 2014-10-13.
• 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

NCT01350245 is recorded as a completed phase 2, single-group study sponsored by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University. Enrollment was 28. The study examined the TJU 2 Step Regimen for patients in remission undergoing allogeneic hematopoietic stem cell transplantation with an HLA partially-matched related donor. The primary endpoint was the percentage alive and disease free at 1 year post-transplant.

The brief summary states historical benchmarks of 50% and 75% for framing the study hypothesis. The design records no concurrent comparator, and the observed DFS result and its statistical analysis are not reported in the registry record. Safety reporting lists 19 participants affected by serious adverse events among 28 at risk, with several serious-event terms and percentages. Further endpoint, analysis, follow-up, and safety-reporting details are not reported in the registry record.