Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies (NCT01093586), a na phase 2 study. The primary outcome is Overall Survival.

2. Clinical Question

In statistical terms, this is a descriptive question about survival within one treated group, not a randomized comparison of transplant against another treatment or a control condition. Without a control group, the registry record cannot establish how outcomes would have differed under another approach. The broad condition list also means the study population spans multiple diagnoses and clinical contexts; a single group-level count would not by itself describe outcomes for each condition separately. The registry record does not report diagnosis-specific primary endpoint results or an adjusted analysis.

3. Trial Design

The registry describes a phase II, single-group study of allogeneic umbilical cord blood transplantation for hematologic malignancies. Enrollment is listed as 14, the status is completed, allocation is not applicable, and masking is none. The sponsor is Case Comprehensive Cancer Center. The recorded study start is 2007-09, and the primary completion is 2015-12.

The intervention is a double-unit umbilical cord blood transplant. The arm description lists a preparative regimen with oral busulfan on days -8 to -5, intravenous cyclophosphamide on days -4 to -3, and intravenous anti-thymocyte globulin or methylprednisolone on days -3 to -1. Transplantation is described for day 0. The intervention list also includes cytogenetic analysis, bone marrow aspiration, and fluorescence in situ hybridization. The registry record does not provide a complete arm description beyond the stated text.

Because there is one study group and no comparator, outcomes describe participants in that group; they do not estimate a between-group treatment effect. The absence of masking is recorded, but the registry record does not describe whether any endpoint assessment was independently adjudicated. Details about recruitment procedures, eligibility thresholds, treatment adherence, or deviations from the intended regimen are not reported in the registry record.

The registry lists a broad range of hematologic diagnoses, including adult and childhood conditions and recurrent or refractory disease categories. The enrollment total is small relative to the breadth of that list, but diagnosis-specific enrollment counts are not reported in the registry record. The design therefore supports a limited descriptive reading of the registered group and does not establish that results are comparable across the listed conditions.

No randomized allocation, control arm, or masking procedure is recorded. The registry record does not report the protocol’s sample-size justification, interim analyses, or full statistical analysis plan. These omissions limit what can be inferred about how the study was designed to evaluate its endpoints.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All participants that went on study, whether completing engraftment or not.

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 Overall Survival, measured over On day +180.

The primary endpoint is a count: the number of participants alive at day +180. Its description specifies alive 180 days after engraftment. The stated population includes all participants who went on study, whether or not they completed engraftment. The number meeting this endpoint is not reported in the registry record.

Overall survival is used in clinical research because it records whether participants are alive at a defined follow-up point, without requiring a judgment about the cause of death. A count at a fixed time point is a straightforward summary, but it does not show the timing of deaths or how survival changes across follow-up. General statistical interpretation: a time-to-event analysis can use information about when events occur and can account for differing follow-up, while a single fixed-time count does not convey those details by itself.

The endpoint description leaves a timing detail to consider: it names day +180 and also defines the time point as 180 days after engraftment, while the population includes participants who may not complete engraftment. The registry record does not explain how the time origin or endpoint status was handled for participants without completed engraftment. It also does not report a survival estimate, confidence interval, or analysis of censoring. Therefore the endpoint’s intended measure is identifiable, but its observed result and some implementation details are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registered design is single-group, and the primary outcome is a participant count. That makes the main question descriptive: how many participants met the stated alive-at-day-+180 condition? The record identifies the endpoint, population, and timeframe, but it does not give the resulting count or an analysis entry. No p-value, confidence interval, or other inferential result for the primary endpoint is reported in the registry record.

What does a single-group design estimate?

General statistical explanation: in a single-group study, an outcome summary estimates what was observed among participants in that group. A treatment contrast requires an appropriate comparison, such as a concurrent control group, because the outcome under an alternative treatment is not observed in the same participants. Here, allocation is listed as not applicable and the model as single-group. Thus, the registry record does not provide a randomized treatment contrast or an estimate of a difference between groups.

This distinction matters even if a one-group outcome count is available. The count would not separate the contribution of the transplant regimen from the participants’ underlying conditions, disease status, other care, or events that occur over follow-up. General statistical explanation: randomization can help balance prognostic factors across comparison groups on average, while a single-group design cannot make that balance through random assignment.

How should a count be read?

The primary outcome parameter is a count of participants, not a continuous measurement or a rate per unit of follow-up. The count is meaningful only with its defined population and endpoint window. The record says the population includes everyone who went on study, regardless of whether engraftment was completed, and defines the endpoint as participants alive at 180 days post engraftment under a day +180 timeframe. The record does not explain how the endpoint was assigned for participants who did not complete engraftment, or provide the result.

General statistical explanation: a count alone does not describe uncertainty unless accompanied by an inferential method and an interval or other uncertainty measure. Nor does a fixed-time count show when events occurred. A time-to-event method typically uses event timing and follow-up information, but the registry’s primary endpoint is specified as a participant count. No time-to-event analysis details or primary endpoint uncertainty estimate are reported in the registry record.

What do the safety counts represent?

The serious-event summary lists Arm I with 14 participants at risk and 9 affected. The other-event fields list 0 at risk and 0 affected. The deaths field is 11. These are separate reported quantities; the record does not explain their overlap or provide participant-level event histories. Selected serious-event terms are accompanied by percentages. Those percentages should be read as registry-listed event summaries, not as evidence of a causal relationship or as comparisons between treatments.

General statistical explanation: event terms can overlap within a participant, and one participant can experience more than one event. Without definitions and participant-level details, event-term percentages should not be added together or treated as mutually exclusive outcomes. The registry record does not report a comparative safety model or an attribution analysis.

What do the planning illustrations mean?

The stated sample-size illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are framed as planning illustrations based on a stated fraction of an observed effect, not as the trial’s own design justification or as observed power. For time-to-event outcomes, the description specifies total events; for other outcomes, it specifies participants per arm. The actual illustration values and the observed effect estimate are not reported in the registry record.

General statistical explanation: a planning calculation asks what sample size might be needed under assumed conditions and a target effect. It does not show that a study had a particular probability of finding a result after the data were collected. Such a calculation depends on its assumptions, including the effect used for planning and the endpoint’s statistical model. The registry record does not report a protocol-based sample-size justification, and the single-group registered design is distinct from the equal-allocation, two-arm assumption described for the planning illustrations.

Other methodological details—including handling of missing endpoint data, censoring, covariate adjustment, and prespecified analysis choices—are not reported in the registry record. No analyses are listed. These gaps prevent reconstruction of an inferential analysis from the registered information.

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 I")
n      <- c(14)
events <- c(8)
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 I  14  8  57.1  28.9  82.3  32.6  78.6  32.3  78.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 15 outcome measures, with secondary outcome names that include hematologic engraftment, overall survival, disease free, recurrence or relapse, and transplant related mortality. Some names appear more than once in the listed outcomes. The registry record does not report how those repeated entries correspond to distinct time points, definitions, or analyses.

When several outcomes or analyses are examined, general statistical explanation: the chance of at least one apparently unusual result can increase if each is tested separately without accounting for the number of tests. A multiplicity strategy may specify a hierarchy, adjustment, or distinction between confirmatory and exploratory outcomes. No multiplicity strategy is reported in the registry record, and no analysis entries are listed. It is therefore not possible to determine whether any inferential claims were adjusted for multiple outcomes or comparisons.

The stated primary endpoint population is all participants who went on study, whether or not they completed engraftment. This identifies the intended population for the endpoint, but the registry record does not report how missing status, withdrawal, incomplete follow-up, or death before the stated time point was handled in analysis. It also does not provide a participant flow or the number with endpoint data available.

The record does not report an analysis population for secondary outcomes, a missing-data method, censoring rules, or sensitivity analyses. These details matter because different handling choices can change the interpretation of a count or survival estimate. Since the primary result and analysis are not reported in the registry record, neither the effect of missing data nor the robustness of any result can be assessed.

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 serious adverse event terms

Serious events with the highest arm-level rate: hypoxia (28.6%), diarrhea (21.4%), death not associated with ctcae term - multi-organ failure (21.4%), death not associated with ctcae term - death nos (14.3%), infection with normal anc or grade 1 or 2 neutrophils (14.3%).

For Arm I, the registry lists 14 participants at risk for serious adverse events and 9 participants affected. It lists 0 at risk and 0 affected for other adverse events, and records 11 deaths. These are the registry’s group-level figures; the record does not provide further detail about the relationship between the death count and the serious-event counts.

The selected serious-event terms include hypoxia (28.6%), diarrhea (21.4%), death not associated with a CTCAE term — multi-organ failure (21.4%), death not associated with a CTCAE term — death NOS (14.3%), infection with normal ANC or Grade 1 or 2 neutrophils (14.3%), and supraventricular and nodal arrhythmia — sinus tachycardia (7.1%). The percentages are reported as event-term summaries. The registry record does not specify whether participants could be counted under more than one term or give additional detail on event timing and severity.

These figures describe safety events recorded in the study group; they are not a comparison with another group and do not establish causation. General statistical explanation: adverse events occurring during a study may have multiple possible causes, and attribution requires information and methods beyond a group event count. No comparative or causal safety analysis is reported in the registry record.

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

The registry record establishes that this was a completed phase II study of double-unit umbilical cord blood transplantation, recorded as a single-group study with enrollment of 14. It specifies overall survival at day +180 as the primary endpoint and describes the endpoint population as all participants who went on study, whether or not they completed engraftment. These details define the study question, but the primary endpoint count is not reported in the registry record.

The record also reports safety summaries for Arm I, including 9 participants affected by serious adverse events among 14 at risk and 11 deaths, plus selected serious-event terms and percentages. These figures can be described as recorded events in the study group. They do not show how the outcomes compare with another treatment or establish that the intervention caused any particular event.

The design does not include a control group, and no randomized comparison is recorded. Consequently, the registry information cannot quantify a treatment effect relative to an alternative. The broad range of listed hematologic conditions further limits interpretation of a pooled group summary as representative of any one diagnosis. Diagnosis-specific outcomes, detailed participant characteristics, and adjusted analyses are not reported in the registry record.

General statistical explanation: an outcome estimate and its uncertainty answer different questions. The estimate describes the observed data, while an interval can indicate statistical precision under a specified analysis. Neither a small interval nor a large count would resolve limitations caused by lack of a comparator or incomplete reporting. Here, no primary result, uncertainty interval, or analysis is reported, so the magnitude and precision of the primary outcome cannot be assessed.

The registry record therefore supports a description of the study’s design, intended endpoint, and reported safety counts. It does not support a conclusion about comparative benefit, comparative harm, or outcome differences across the diagnoses listed.

14. Why This Trial Matters Statistically

This study illustrates why statistical interpretation begins with the design and the endpoint, not only with the treatment name. The registry identifies a phase II, single-group study and a day +180 overall-survival count. Because there is no comparator, the registered design can describe outcomes in its participants but cannot estimate a randomized difference between treatment strategies.

It also highlights the importance of defining the endpoint population and time origin. The primary endpoint description refers to 180 days after engraftment, while its population includes all participants who went on study whether or not engraftment was completed. The registry record does not explain how that circumstance was handled. Clear reporting of the time origin and endpoint status is essential for interpreting a fixed-time outcome consistently.

The study’s condition list spans many hematologic malignancies and age groups, whereas enrollment is 14. The registry record does not provide diagnosis-specific results. This makes it important not to treat a group-level summary as evidence of a common outcome pattern across all listed conditions.

The record also separates the existence of safety figures from the ability to interpret them causally. Serious-event counts and percentages are useful descriptions of what was recorded, but without a comparison group and details on attribution, they do not establish whether event frequency differs from another setting or treatment.

Finally, the record demonstrates the value of complete results reporting. The endpoint is named, but its result and analysis are not reported. Without those, readers cannot assess the observed outcome, its precision, or how missing data and multiple outcomes were addressed. The statistical lesson is that a defined design and endpoint are necessary, but they are not substitutes for reported 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.

A central limitation for interpreting this specific study is that the primary endpoint result is not reported in the registry record. The endpoint is defined as the number alive at day +180, but no observed count or uncertainty estimate is available. The record also lists no analysis entries, so there is no reported primary analysis to assess.

The single-group design has no concurrent comparison arm. Thus, it cannot support a randomized comparison with another treatment. Allocation is listed as not applicable and masking as none. The record does not report whether outcome assessment was independently adjudicated or whether any safeguards against assessment bias were used.

The listed conditions span a broad range of adult and childhood hematologic malignancies, including recurrent and refractory conditions. Enrollment is 14, but diagnosis-specific enrollment and outcomes are not reported. A pooled description therefore cannot be assumed to represent each diagnosis or subgroup. The registry record also does not report participant characteristics that could help readers understand the clinical composition of the group.

The primary endpoint’s time definition may be difficult to apply to the full stated population: it is described as 180 days after engraftment, while the population includes participants regardless of whether engraftment was completed. The registry record does not explain the time origin or endpoint handling for participants without completed engraftment.

Other details not reported in the registry record include missing-data procedures, censoring rules, analysis populations for secondary outcomes, multiplicity handling, and the protocol’s sample-size justification. The safety record provides group counts and selected terms but no comparative analysis or event attribution. These limitations concern what can be concluded from the registry information; they do not by themselves establish how the study was conducted beyond the recorded details.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies — NCT01093586.
• Registry record last updated 2019-01-23; status last verified 2019-01.
• Results first posted 2019-01-04.
• 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

This completed phase II study is recorded as a single-group, unmasked trial of double-unit umbilical cord blood transplantation for hematologic malignancies. Enrollment is 14, and the sponsor is Case Comprehensive Cancer Center. The primary endpoint is the number of participants alive at day +180; the result is not reported in the registry record.

The registry lists 15 outcome measures and no analysis entries. Its Arm I safety summary records 14 participants at risk for serious adverse events, 9 affected, and 11 deaths, with selected serious-event terms and percentages. No comparator is recorded, so these data describe the study group rather than a treatment contrast. The registry record does not report the primary endpoint analysis, uncertainty estimates, or several key analysis details.