This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers (NCT01135329), a na phase 2 study. The primary outcome is Chimerism in Unsorted Peripheral Blood.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01135329 |
| Phase | Phase 2 |
| Condition | Lymphoma, Leukemia, Myelodysplastic Syndrome |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with lymphoma, leukemia, or myelodysplastic syndrome. The registry records enrollment of 15 participants. It does not give further eligibility details in the trial summary, so characteristics such as disease status and participant subgroups cannot be described from this record.
Intervention
The study evaluated a reduced-intensity, related-donor allogeneic bone marrow transplant. The preparative regimen was based on fludarabine and busulfan. Graft-versus-host disease prophylaxis used cyclophosphamide, tacrolimus, and mycophenolate mofetil.
Comparator
There was no separate comparison arm: the registry identifies a single-group study, with allocation recorded as not applicable and no masking. The study therefore does not provide a randomized or concurrent control comparison of the transplant regimen against another treatment.
Primary question
The primary endpoint concerned the percentage of participants achieving full-donor chimerism in unsorted peripheral blood at Day 60. In plain terms, the investigators aimed to assess whether donor-derived blood cells were present at the endpoint’s specified level. The registry says the trial was terminated early because stopping-rule criteria were met for a high graft failure rate, and that the data were not analyzed.
3. Trial Design
This was a phase 2, single-group study of reduced-intensity, related-donor allogeneic bone marrow transplantation for hematologic malignancies. The registry lists lymphoma, leukemia, and myelodysplastic syndrome as conditions. Enrollment was 15. The study was sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, began in 2010-08, and had a primary completion date of 2012-05. Its current registry status is terminated.
The treatment approach combined a fludarabine- and busulfan-based preparative regimen with bone marrow transplantation. Cyclophosphamide, tacrolimus, and mycophenolate mofetil were used for graft-versus-host disease prophylaxis. The record lists these drugs as interventions. It does not provide enough detail to describe drug doses, schedules, transplant procedures, or individual treatment exposure.
The design has no randomized allocation and no masking. Since it is a single-group study, outcomes describe the participants in that group; they do not establish a like-for-like comparison with another regimen. The registry does not report a control group, a comparator treatment, or a protocol-based statistical plan in the information available for this record.
The stated primary endpoint was full-donor chimerism in unsorted peripheral blood at Day 60. Other listed outcomes include overall survival, progression-free survival, relapse, non-relapse mortality, graft-versus-host disease, and failure to engraft. The registry reports eight outcome measures but zero analyses. Most importantly, it states that the trial was terminated early after stopping-rule criteria were met for a high graft failure rate and that the primary data were not analyzed. Consequently, the design and stated objectives can be described, but the primary endpoint’s observed result cannot.
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
| Stage | BMT Allogenic Transplantation |
|---|---|
| STARTED | 15 |
| COMPLETED | 15 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: The trial has been terminated early since stopping rule criteria were met (high graft failure rate). Data were not analyzed.
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.
| Characteristic | Unit | BMT Allogenic Transplantation |
|---|---|---|
| Participants analysed | 15 | |
| Age, Continuous | years | 49 (3 to 73) |
| Age, Categorical: <=18 years | Participants | 1 |
| Age, Categorical: Between 18 and 65 years | Participants | 12 |
| Age, Categorical: >=65 years | Participants | 2 |
| Sex/Gender, Customized: Female | participants | 6 |
| Sex/Gender, Customized: Male | participants | 9 |
| Region of Enrollment: United States | participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Chimerism in Unsorted Peripheral Blood, measured over Day 60.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the percentage of participants achieving full-donor chimerism in unsorted peripheral blood at Day 60. Chimerism describes the presence of donor-derived cells in a recipient after a transplant. In this study, the measure was intended to indicate whether the donor marrow had established itself in the blood by the specified assessment time. The registry’s stated main goal was to determine how quickly the donor marrow takes.
In general, a biologic endpoint can help characterize an early process after an intervention. It is different from outcomes such as survival, relapse, or complications, which describe clinical events over time. A chimerism result does not by itself summarize all those other outcomes. The record lists several such outcomes separately, including overall survival, progression-free survival, relapse, non-relapse mortality, graft-versus-host disease, and failure to engraft.
The registry does not state a numerical threshold defining full-donor chimerism, nor does it report an observed percentage or a confidence interval for this endpoint. It does state that the trial stopped early after stopping-rule criteria were met for a high graft failure rate and that the data were not analyzed. Thus, the endpoint’s purpose is clear, but its estimate and precision are not available in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record describes a phase 2 investigation of a reduced-intensity, related-donor transplant strategy in a single group. It records enrollment of 15 participants and identifies Day 60 full-donor chimerism in unsorted peripheral blood as the primary endpoint. The trial was terminated early because stopping-rule criteria were met for a high graft failure rate. The registry explicitly says the primary data were not analyzed.
That statement governs interpretation of the primary question. There is no reported endpoint percentage, numerator, denominator for the endpoint, or estimate of uncertainty. The registry therefore does not permit a quantitative conclusion about the proportion achieving full-donor chimerism at Day 60. The stopping-rule description is an important trial-level result, but it is not a substitute for an analyzed estimate of the primary endpoint. The record does not give the numerical threshold or the detailed calculation used for the stopping rule.
The study’s single-group design also limits the kinds of conclusions it can support. Outcomes observed in the transplant group cannot be separated from other influences by comparison with a concurrent control group. The record does not report randomized allocation or a control arm. Any descriptions of survival, complications, or engraftment would therefore be descriptive for the enrolled group rather than a randomized estimate of a difference between treatments.
The registry lists secondary outcomes, but reports zero analyses. For that reason, the listed outcomes should be understood as planned or registered measures, not as findings with reported estimates. The record does not provide survival estimates, relapse estimates, event-time analyses, or rates for the secondary outcomes. It also does not provide confidence intervals or statistical tests for them.
Some adverse-event information is reported separately. Serious adverse events were recorded for 11 participants in the transplant group, other adverse events for 7 participants, and 9 deaths. The record also lists selected serious adverse-event terms with percentages. These figures are safety descriptions, not adjusted comparisons, and the record does not establish that each event or death was caused by the transplant or a particular drug.
Overall, the record supports a narrow interpretation: the study enrolled participants, specified a biologic primary endpoint, and stopped early after a stopping criterion related to graft failure was met. It does not provide an analyzed primary endpoint result or analyzed secondary outcome estimates. The absence of those estimates means that the size and precision of the intended endpoint finding cannot be evaluated from this registry record.
9. Statistical Methods Explained
The statistical information in the registry is limited. It describes a single-group phase 2 design, lists the primary and secondary outcomes, and records that the primary data were not analyzed after early termination. It does not report the trial’s detailed statistical analysis plan, the stopping-rule threshold, or a set of endpoint estimates. The concepts below explain how such information is generally interpreted; they are not claims about analyses performed in this study.
What does a single-group design estimate?
In general, a single-group study describes outcomes among participants who receive the studied intervention. Its results can characterize what happened in that group, but without a concurrent comparison group they cannot directly estimate how outcomes would have differed under another intervention. This study is recorded as single-group, with allocation not applicable and masking none. It therefore does not have a randomized treatment contrast in the registry record.
A group’s observed outcome can still be useful descriptively. However, the interpretation depends on clear definitions, complete follow-up, and an analysis appropriate to the endpoint. For a percentage endpoint, the numerator and denominator need to be defined, and the estimate’s uncertainty is usually important. The registry does not report the primary endpoint numerator, denominator, percentage, or confidence interval. Because it says the data were not analyzed, no primary endpoint estimate can be interpreted from the record.
What does an uncertainty interval convey?
In general, a confidence interval expresses the range of values compatible with an estimate under a specified statistical procedure. A narrow interval indicates greater precision than a wide interval, while neither width nor location alone establishes causation or clinical importance. An interval for a proportion would depend on the observed data and the chosen method. No such interval is reported for the primary endpoint in this record. It would be inappropriate to invent one or to infer precision from the enrollment count alone.
How should early stopping be read?
The registry states that stopping-rule criteria were met because of a high graft failure rate and that the trial was terminated early. In general, a stopping rule is a prespecified or otherwise defined condition that can lead to ending a study before its planned endpoint assessments are complete. The rule’s exact specification matters when interpreting why a trial stopped and what conclusions can be drawn. The threshold, monitoring schedule, and decision process are not reported in the registry record. The record’s statement can be quoted, but its statistical operating characteristics cannot be assessed from the available description.
Early termination can limit the amount of follow-up and the number of observed outcomes available for analysis. In general, fewer observations can make estimates less precise, and stopping can affect which outcomes are observable. Those are general considerations, not a claim that a particular bias occurred in this trial. The record does not give the timing of individual failures, the number meeting the stopping criterion, or a complete accounting of follow-up.
What do the sample-size illustrations represent?
The registry describes sample-size calculations as planning illustrations based on an observed effect, not as the trial’s own design. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. For time-to-event calculations, the stated output is the total number of events; for other calculations, it is participants per arm. These assumptions describe hypothetical planning calculations, not the actual single-group design recorded for this study.
The registry does not report numerical sample-size illustration results in this record. Further, it says the primary data were not analyzed, so the record does not supply a primary endpoint estimate that can be linked to such an illustration. These calculations should not be read as observed power, proof that the trial was adequately sized, or a description of the original enrollment rationale. The protocol’s sample-size justification is not reported in the registry record.
Why does over-dispersion matter in general?
For count outcomes, a simple statistical model may assume that variability follows a particular relationship to the mean. Over-dispersion means that observed variability is greater than that model expects. If it is ignored, standard errors can be too small and intervals or tests can appear more conclusive than warranted. This is a general issue in count-data analysis. The registry does not identify an over-dispersion analysis for this study, and no such method should be attributed to it.
Likewise, time-to-event outcomes such as overall survival or progression-free survival generally require attention to follow-up duration and censoring. Censoring occurs when a participant’s event time is not observed during the available follow-up period. In general, survival estimates and comparisons depend on how follow-up and censoring are handled. Although these outcomes are listed, the registry reports zero analyses and does not specify an analysis method, follow-up summary, or censoring approach.
These general concepts clarify what would normally be needed to judge estimates: endpoint definitions, observed data, an analysis method, and an account of uncertainty. For this study, the registry provides the endpoint description and the early-termination statement, but not an analyzed primary estimate or a detailed statistical plan. Interpretation must remain within those limits.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several outcomes beyond the primary chimerism measure: overall survival, progression-free survival, incidence of relapse, non-relapse mortality, incidence of graft-versus-host disease, and participants who failed to engraft. It reports eight outcome measures in total and zero analyses. The record does not provide estimates or tests for the listed outcomes.
In general, testing many outcomes can increase the chance of finding at least one apparently notable result by chance. A multiplicity plan may specify which outcomes are primary, how secondary outcomes are interpreted, and whether statistical testing is adjusted or treated as exploratory. The registry record does not report a multiplicity adjustment or an outcome-testing hierarchy. It is therefore not possible to determine whether any formal multiplicity strategy was planned.
The primary outcome description specifies Day 60, but the registry says its data were not analyzed. It does not report a primary endpoint estimate or an analysis population. The record also does not describe whether analyses were intended to include all enrolled participants, only participants who received the transplant, or another defined group. Such analysis-population definitions are not reported in the registry record.
Missing-data handling is likewise not reported. In general, a reader would need to know which outcomes were unavailable, why they were unavailable, and how the analysis treated them. Early termination and incomplete follow-up can make those details particularly relevant, but the record does not provide participant-level follow-up or a missing-data approach. No imputation method, sensitivity analysis, or missingness assumption is described. These absences prevent assessment of how incomplete outcome information might affect an estimate.
The reported absence of analyses should not be mistaken for evidence that the listed outcomes had no events or no differences. It means that the registry record does not provide analyzed results for them. The stopping-rule statement is reported, but it does not disclose a complete outcome-analysis plan or resolve questions about multiplicity, missingness, or analysis populations.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Transplant | 11 | 15 | 73.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Transplant | 9 of 15 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Transplant | 7 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Transplant |
|---|---|---|
| Infection | Infections and infestations | 7 of 15 |
| Fever | General disorders | 2 of 15 |
| Sepsis | Infections and infestations | 2 of 15 |
| Diarrhea | Gastrointestinal disorders | 1 of 15 |
| Febrile neutropenia | Blood and lymphatic system disorders | 1 of 15 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 15 |
| Chills | General disorders | 1 of 15 |
| Dehydration | Metabolism and nutrition disorders | 1 of 15 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Transplant |
|---|---|---|
| Infection | Infections and infestations | 4 of 15 |
| Febrile neutropenia | Infections and infestations | 4 of 15 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 2 of 15 |
| nausea | Gastrointestinal disorders | 1 of 15 |
| fever | General disorders | 1 of 15 |
| tremor | Nervous system disorders | 1 of 15 |
| hallucinations | Psychiatric disorders | 1 of 15 |
| Elevated liver enzymes | Investigations | 1 of 15 |
| Elevated total bilirubin | Investigations | 1 of 15 |
Serious events with the highest arm-level rate: infection (46.7%), fever (13.3%), sepsis (13.3%), diarrhea (6.7%), febrile neutropenia (6.7%).
The registry reports serious adverse events for 11 participants in the transplant group and other adverse events for 7 participants. It records 9 deaths. These are participant counts in the registry’s adverse-event summary. They should be read as reported counts, not as a comparison between treatments, since this was a single-group study with no separate control arm.
The listed serious adverse-event terms include Infection at 46.7%, Fever at 13.3%, Sepsis at 13.3%, Diarrhea at 6.7%, Febrile neutropenia at 6.7%, and Hypoxia at 6.7%. These are registry-reported percentages attached to event terms. The record does not state in this summary whether participants could contribute to more than one term, so the terms should not be added together or treated as mutually exclusive categories.
In general, an adverse event is an event recorded during a study; its presence does not by itself establish that the intervention caused it. A serious adverse event is a regulatory seriousness category, not a measure of how likely an event was to be caused by treatment. The registry’s figures do not provide comparative risk estimates, adjusted analyses, or causal attribution for each event or death.
The safety counts are important descriptive information, but the record does not give event timing, duration, resolution, participant-level overlap, or a complete narrative for the events. It also does not report a safety comparison with another group. These counts should therefore be interpreted as registry-reported observations in the transplant group, with those limitations in mind.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase 2, single-group study of reduced-intensity, related-donor bone marrow transplantation was conducted in participants with hematologic cancers. It records enrollment of 15 participants and identifies full-donor chimerism in unsorted peripheral blood at Day 60 as the primary endpoint. It also reports that the trial was terminated early after stopping-rule criteria were met for a high graft failure rate.
What the record does not establish is the primary endpoint’s observed percentage. The registry explicitly says that the data were not analyzed. It therefore offers no estimate, confidence interval, or statistical test for Day 60 chimerism. The stated stopping reason should not be converted into a numerical graft-failure rate: no such rate is reported in the record.
The trial was not randomized and had no comparator arm. Any treatment-outcome relationship in a single group is descriptive and cannot by itself show what would have happened under a different regimen. The study’s design also does not support a like-for-like comparison of treatment effects. The record does not report comparative estimates for survival, relapse, graft-versus-host disease, or the other listed secondary outcomes.
The adverse-event summary gives participant counts and selected event percentages. Those figures provide safety descriptions, not proof that a specific drug or procedure caused each event. Nor do they supply a comparative estimate of risk. The record does not report the context and timing needed for a more detailed interpretation of individual events.
In general, a clinical study can be informative even when it does not yield a complete endpoint analysis: the design, feasibility, stopping experience, and observed safety information can all be relevant to understanding the research. Here, however, the registry record is not sufficient to quantify the primary chimerism outcome or the secondary outcomes. It supports a description of the study and its early termination, not a numerical assessment of the primary endpoint or a comparative claim about treatment effects.
13. Why This Trial Matters Statistically
This study illustrates why a registered endpoint and an analyzed endpoint are not the same thing. The record identifies a biologic primary measure, full-donor chimerism in unsorted peripheral blood at Day 60, and explains its connection to the study’s goal of determining how quickly donor marrow takes. But the registry also states that the trial was terminated early after stopping-rule criteria were met for a high graft failure rate and that the data were not analyzed. Without an analyzed estimate, the endpoint’s magnitude and precision remain unknown.
It also highlights the importance of design when reading clinical statistics. A single-group study can describe outcomes among participants receiving an intervention, but it does not provide a randomized comparator for estimating a treatment difference. The registry’s enrollment of 15 participants is a description of the study size, not a substitute for an endpoint result or an uncertainty interval.
The record further distinguishes registered outcomes from reported analyses. It lists survival, relapse, non-relapse mortality, graft-versus-host disease, and failure to engraft as secondary outcomes, while reporting zero analyses. A list of outcomes indicates what was tracked or intended for assessment; it does not establish that results were calculated or reported.
Finally, the safety information shows why adverse-event counts require careful interpretation. Counts and event-term percentages describe what the registry records, but without a comparison group and detailed attribution they do not answer causal questions. Together, these features make the record useful for understanding the study’s design and reporting limits, while underscoring why conclusions must depend on the analyses actually available.
14. 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 central limitation is that the primary endpoint data were not analyzed. The registry states that the trial was terminated early because stopping-rule criteria were met for a high graft failure rate. It does not report the stopping threshold, the number of participants meeting it, or an endpoint estimate. As a result, the primary measure cannot be quantified from the registry record.
Enrollment was 15, and the study had a single group. The record does not include a concurrent comparator or randomized allocation, so it cannot provide a randomized estimate of the difference between this regimen and another approach. The registry also does not report participant characteristics in enough detail to assess how the enrolled group was composed across the listed conditions.
Although multiple secondary outcomes are listed, the registry reports zero analyses. It does not provide estimates for overall survival, progression-free survival, relapse, non-relapse mortality, graft-versus-host disease, or failure to engraft. The analysis populations, follow-up details, missing-data methods, and statistical procedures are not reported in the registry record.
The safety summary is limited to participant counts and selected event terms. It does not provide event timing, overlap among terms, detailed causal attribution, or a control-group comparison. These limitations mean that the counts cannot be interpreted as comparative or causal effects.
The record’s sample-size note describes planning illustrations, not the original design rationale, and does not provide numerical illustration results in this record. The protocol’s sample-size justification and detailed statistical analysis plan are not reported. These reporting gaps restrict assessment of precision, planned analyses, and the consequences of early termination.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers — NCT01135329.
- Registry record last updated 2026-05-27; status last verified 2026-05.
- Results first posted 2018-07-03.
- 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.
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18. Record Summary
This phase 2 study evaluated reduced-intensity, related-donor allogeneic bone marrow transplantation using a fludarabine- and busulfan-based preparative regimen, with cyclophosphamide, tacrolimus, and mycophenolate mofetil for graft-versus-host disease prophylaxis. It enrolled 15 participants with lymphoma, leukemia, or myelodysplastic syndrome. The design was single-group, with no masking and no allocation to a separate comparator.
The primary endpoint was full-donor chimerism in unsorted peripheral blood at Day 60. The registry states that the study was terminated early after stopping-rule criteria were met for a high graft failure rate and that the primary data were not analyzed. It lists several secondary outcomes but reports zero analyses. The adverse-event summary records serious adverse events for 11 participants, other adverse events for 7 participants, and 9 deaths. The record does not report an analyzed primary endpoint estimate or comparative treatment effects.