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 G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome (NCT00228813), a na na study. The primary outcome is Engraftment Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00228813 |
| Phase | NA |
| Condition | Hematologic Malignancies |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Emory University |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with hematologic malignancies or bone marrow failure syndrome receiving bone marrow from partially mismatched related donors. The registry condition listing names hematologic malignancies; it does not report how many participants had either condition or describe other participant characteristics.
Intervention
Donors underwent Granulocyte Colony Stimulating Factor (G-CSF) stimulation before bone marrow collection. The study description specifies that recipients received in vivo T-cell-depleted G-CSF-stimulated bone marrow.
Comparator
There was no control group. The study is recorded as single-group, with allocation listed as NA, and the registry explicitly identifies it as not a randomized comparison. Its findings therefore cannot provide a like-for-like comparison against another treatment or against an untreated group.
Primary question
The primary question was how many participants receiving this transplant approach reached engraftment by Day 45. The study also aimed to evaluate the incidence and severity of acute and chronic graft-versus-host disease. Numeric results for these outcomes are not reported in the registry record.
The record describes a feasibility study rather than a comparative efficacy trial. Its primary outcome is a participant count tied to a specified follow-up point, while the brief summary identifies additional clinical outcomes of interest. The registry lists enrollment as 8 and the status as terminated. It does not report why the study was terminated, whether enrollment reached a planned target, or the participant-level circumstances behind termination.
The clinical question is thus about whether engraftment was observed within the stated timeframe among participants receiving the specified transplant approach, and what graft-versus-host disease outcomes were recorded. The posted registry results do not give the engraftment count, the results for graft-versus-host disease, or a comparison that would isolate the effects of G-CSF stimulation or T-cell depletion.
3. Trial Design
This was a single-group study sponsored by Emory University. The registry lists allocation as NA and masking as NONE. It explicitly notes that the study was not a randomized comparison and that its arms are not a like-for-like control comparison. No control group or randomized allocation is described, so the design does not support a direct treatment contrast.
The intervention is listed as Granulocyte Colony Stimulating Factor, classified as a drug. The arm description clarifies that donors received G-CSF stimulation before bone marrow collection; recipients were to receive the resulting bone marrow with in vivo T-cell depletion from partially mismatched related donors. This describes the transplant approach under study, not a separate recipient-only drug comparison.
The record lists enrollment as 8, phase as NA, and status as TERMINATED. The study start is recorded as 2004-04 and primary completion as 2015-01. The registry does not report the reason for termination, a detailed recruitment timeline, or whether enrollment matched a planned target. The study title and description refer to patients with hematologic malignancies or bone marrow failure syndrome, while the condition listing names hematologic malignancies. The number of enrolled participants in each condition is not reported in the registry record.
The brief summary identifies engraftment and acute and chronic graft-versus-host disease as objectives. It does not give a protocol-level account of assignment, follow-up procedures, eligibility criteria, or rules for handling deviations. Those details are not reported in the registry record. As a single-group feasibility study, its descriptive results would characterize outcomes in the enrolled participants; without a control group, they would not establish how those outcomes compare with another approach.
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 | Granulocyte Colony Stimulating Factor (G-CSF) Stimulation |
|---|---|
| STARTED | 8 |
| COMPLETED | 4 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Granulocyte Colony Stimulating Factor (G-CSF) Stimulation |
|---|---|
| Death | 4 |
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 | Granulocyte Colony Stimulating Factor (G-CSF) Stimulation |
|---|---|---|
| Participants analysed | 8 | |
| Age, Categorical: <=18 years | Participants | 7 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Engraftment Rate, measured over Day 45.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | particpants |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of participants who received in vivo T-cell-depleted G-CSF-stimulated bone marrow from a partially mismatched related donor and reached engraftment by Day 45. This is a count of participants meeting a defined milestone, not a time-to-event estimate or a measure of the degree of engraftment. The outcome description specifies the time point but does not give additional criteria for determining that engraftment occurred.
Engraftment is a transplant-process outcome: it records whether the transplanted marrow has established sufficiently to meet the study's stated criterion. In a feasibility study, documenting whether and when this milestone is reached can help describe how the transplant approach performed in the enrolled group. The endpoint is therefore relevant to the study's stated aim of examining engraftment, but it does not by itself describe other outcomes such as graft-versus-host disease, survival, or longer-term health.
The primary result—the number reaching engraftment by Day 45—is not reported in the registry record. The endpoint description also does not state a separate denominator or specify how participants without an evaluable Day 45 assessment would be treated. Those details are not reported in the registry record, so the result cannot be interpreted as a reported proportion or rate from the information available.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically specific primary outcome: the number of participants reaching engraftment by Day 45 after receiving the described transplant approach. However, it does not report that number. Without the endpoint result, the observed engraftment experience cannot be quantified, and the record does not support an estimate of the outcome's precision. The absence of a posted result is not evidence that engraftment did or did not occur.
Enrollment is listed as 8, but enrollment alone does not reveal the number who completed the relevant follow-up, had an evaluable assessment, or reached the endpoint. The primary endpoint's analysis population and denominator are not reported in the registry record. It would therefore be inappropriate to treat enrollment as the outcome denominator or to infer a success count from the study's size.
The study is single-group and is explicitly not a randomized comparison. Any outcome observed in this group would describe the enrolled participants under this transplant approach. It would not establish what would have happened to similar participants receiving another approach, nor would it separate the contributions of donor stimulation, T-cell depletion, the donor relationship, or other aspects of care. A comparator is not available in this record.
The brief summary also names acute and chronic graft-versus-host disease as outcomes of interest, but no numeric results for either are reported. Three outcome measures are listed in the record, while no analyses are listed. The status is TERMINATED, but the reason for termination and its relationship, if any, to outcome availability are not reported in the registry record. It is not possible to infer whether outcomes were collected but not posted, whether follow-up was incomplete, or whether the study ended before planned assessments.
Safety information is posted separately: the registry lists serious adverse events affecting 4 participants among 8 at risk, and other adverse events affecting 0 among 8 at risk. These safety entries do not fill the gap in the primary endpoint result and do not establish the occurrence or absence of engraftment. The serious-event terms and their percentages should also be interpreted cautiously because their event-specific denominators and counting rules are not reported in the registry record.
Overall, the posted record describes the question and design, but its missing primary outcome result sharply limits statistical interpretation. No treatment effect, comparative benefit, or lack of benefit can be estimated from the information posted. The feasibility question remains unanswered by the posted primary result.
9. Statistical Methods Explained
The registry describes a single-group study with a participant-count primary endpoint. These design features determine what kinds of statistical conclusions are possible. A single-group result can describe the observed experience in that group, but it cannot estimate a randomized contrast against a control group. Here, allocation is listed as NA, masking as NONE, and the record explicitly states that this is not a randomized comparison.
What does a participant count represent?
The primary outcome counts participants who reached engraftment by Day 45. It is not a count of repeated events, and it is not a measurement of how long each participant took to engraft. In general, a participant count needs a clearly defined eligible population and an outcome denominator to be interpreted as a proportion. The registry gives the enrollment figure but does not report the primary result or its denominator. Enrollment should not be substituted for the endpoint denominator without evidence that every enrolled participant was assessed and included.
For a binary outcome such as reaching or not reaching a milestone, statistical uncertainty depends on both the observed count and the number of participants assessed. A confidence interval, when reported, expresses the range of values reasonably compatible with the data under the chosen method. It does not remove design limitations, and it cannot be reconstructed from the posted record when the endpoint count and denominator are absent. No primary confidence interval or other precision measure is reported in the registry record.
Why does the design matter?
Random assignment can make treatment groups comparable in expectation, which supports interpretation of differences as treatment contrasts when the trial is properly conducted and analyzed. This study has no randomized comparator, so that framework does not apply. Even if a group-level outcome were posted, it would remain descriptive. Differences from outcomes in other studies or from historical expectations would not be randomized comparisons and could reflect differences in participants, care, assessment, or follow-up.
Masking is listed as NONE. Masking can reduce certain forms of bias in outcome assessment or care, depending on the outcome and study procedures. The registry record does not report whether engraftment assessment used independent review or specified objective criteria. It therefore does not permit an assessment of how outcome ascertainment was protected from bias.
What can be said about modeling?
No statistical analysis is listed in the registry record. The primary endpoint is described as a number of participants, but no fitted model, estimation method, hypothesis test, confidence interval, or adjustment strategy is reported. For participant-level binary outcomes, analysts commonly report the count and denominator, and may report a proportion with a confidence interval. Those are general methods, not analyses documented for this study.
Over-dispersion is a concern in some count-data models when variability exceeds what a model assumes, particularly for rates or counts of recurrent events. This endpoint is different: it counts participants reaching a milestone rather than recurrent events per participant. A rate model with person-time or a recurrent-event model is not specified in the record and should not be presumed. More importantly, the missing endpoint result precludes choosing or checking a model based on the observed data.
How should the planning illustrations be read?
The registry's planning illustrations are described as calculations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are planning illustrations, not observed power and not the design of this single-group study. The stated assumptions refer to a hypothetical two-arm design, whereas the actual study is recorded as single-group. The illustrations do not constitute a comparison observed in this trial.
The record states that the effect to detect is a stated fraction of the observed effect. It does not report a trial-specific sample-size justification, and a sample-size rationale is not reported in the registry record. No planning estimate should be read as proof that the study had a particular probability of detecting an effect. The planning output is also not a substitute for the unreported primary result. Where event-based planning is described generally for time-to-event outcomes, that does not change the nature of this primary participant-count endpoint.
Because the sponsor's analysis is not posted and no recalculated estimate is available in the registry record, there is no basis for comparing an independent calculation with a sponsor analysis. The registry lists no analyses. Statistical interpretation should therefore stay with the study's documented design, endpoint definition, and the outcomes actually posted, while distinguishing those details from general statistical principles.
10. Multiplicity, Missing Data and Analysis Populations
The record lists 3 outcome measures and identifies engraftment as the primary outcome. Its brief summary also names acute and chronic graft-versus-host disease objectives. Considering several outcomes raises a general multiplicity issue: when many hypotheses are tested, the chance of at least one apparently notable result can increase unless the analysis plan accounts for the number and relationships of the tests. The registry does not list any analyses or report a multiplicity adjustment. Whether any adjustment was planned or performed is not reported in the registry record.
Missing outcome information is a separate concern. The primary result is not reported, and the registry does not state how many participants had an evaluable Day 45 assessment or how missing assessments were handled. It does not describe whether participants were analyzed according to an intention-to-treat principle, another analysis population, or a per-protocol definition. The analysis populations are not reported in the registry record.
The enrollment figure does not resolve these questions: it does not establish that each enrolled participant contributed to each endpoint. Reasons for missing outcome data, follow-up completeness, and sensitivity analyses are not reported in the registry record. These gaps prevent assessment of whether missingness could have affected the reported outcomes, particularly because the outcomes themselves are not posted. No imputation method or other missing-data procedure is described.
Although the record lists no analyses, that should not be interpreted as confirmation that no analyses were conducted outside the registry. It means the registry record does not present them. The distinction matters: without reported methods and results, readers cannot evaluate whether outcome selection, missing data, or multiple outcomes influenced the findings.
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 |
|---|---|---|---|
| Granulocyte Colony Stimulating Factor (G-CSF) Stimulation | 4 | 8 | 50.0% |
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 |
|---|---|
| Granulocyte Colony Stimulating Factor (G-CSF) Stimulation | 0 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | Granulocyte Colony Stimulating Factor (G-CSF) Stimulation |
|---|---|---|
| Recurrent Disease | Blood and lymphatic system disorders | 2 of 8 |
| Multi-organ system failure | General disorders | 2 of 8 |
Serious events with the highest arm-level rate: recurrent disease (25.0%), multi-organ system failure (25.0%).
The registry lists serious adverse events for the G-CSF stimulation group: 4 participants are recorded as affected among 8 at risk. For other adverse events, 0 participants are recorded as affected among 8 at risk. These are the posted group-level figures; the record does not explain the event-collection window or give detailed rules for attribution.
The serious-event terms listed are recurrent disease and multi-organ system failure, each with a reported percentage of 25.0%. The registry does not state the denominator or counting method for these term-specific percentages. They should not be converted into participant counts or interpreted as percentages of all participants without that information. A participant may also experience more than one event, so event terms and affected-participant counts are not necessarily interchangeable.
Deaths are not reported in the registry record. The posted safety figures do not establish a causal relationship between the intervention and any event, and they do not provide a comparison with another group. They are descriptive safety entries in a small, single-group study; the absence of a listed other adverse event is not proof that no other events occurred outside the recorded data.
12. What the Result Does, and Does Not, Mean
The record establishes that a feasibility study was conducted to examine engraftment and graft-versus-host disease outcomes after recipients received in vivo T-cell-depleted, G-CSF-stimulated bone marrow from partially mismatched related donors. It identifies engraftment by Day 45 as the primary endpoint and lists enrollment as 8. These details define the research question and context.
However, the number of participants who reached engraftment by Day 45 is not reported in the registry record. Numeric results for acute or chronic graft-versus-host disease are also not reported. Consequently, the posted record does not show whether the primary milestone was reached, how often it was reached, or how precisely the outcome was estimated. It cannot establish that the approach did or did not produce a particular outcome.
The single-group design also limits what an observed result could mean. Without a control group, a result would describe the experience of participants in this study; it would not determine what would have occurred under a different transplant approach. No randomized comparison is available to separate the effect of the intervention from other influences. The posted record therefore does not support a comparative treatment effect.
The serious adverse event figures are descriptive, not a comparative safety analysis. The registry lists 4 affected participants among 8 at risk for serious adverse events and 0 among 8 at risk for other adverse events. It does not report deaths, event attribution, or the term-specific denominators. Those figures cannot establish causation or a relative safety profile.
The appropriate interpretation is limited: the record describes a terminated feasibility study, its intended outcomes, and some safety entries, but does not post the primary endpoint result or analyses. Missing results should not be treated as either a favorable or unfavorable finding. The reason for termination and the completeness of outcome follow-up are not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why a clearly stated clinical question and a posted result are both necessary for statistical interpretation. The registry defines an endpoint—engraftment by Day 45—and describes the transplant approach, but it does not report the endpoint count. Without that result and its denominator, readers cannot assess the observed outcome or its precision.
Its single-group structure also demonstrates the difference between feasibility description and comparative inference. A study can describe outcomes among participants receiving an approach, but a control group is needed for a direct comparison within the trial. Here, the registry explicitly notes that the study is not a randomized comparison. Even a complete single-group result would not by itself answer whether outcomes differ from those under another approach.
The record further shows why enrollment, endpoint results, and safety figures must not be conflated. Enrollment is listed as 8; the primary endpoint denominator is not reported; and serious adverse events are recorded separately. These pieces of information serve different purposes and do not substitute for one another.
Finally, the record lists 3 outcome measures but no analyses. This makes reporting of analytic methods, missing-data handling, and the relationship between primary and additional outcomes especially important for interpretation. Their absence leaves readers unable to assess precision, multiplicity, or the effect of incomplete follow-up. The statistical lesson is not about the success or failure of the intervention, but about the limits imposed when results and analysis details are unavailable.
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.
Several limitations are specific to this record. The most consequential is that the primary endpoint result is not reported in the registry record. The endpoint description names the Day 45 milestone, but gives neither the number reaching it nor the denominator used for analysis. No estimate of precision can be assessed from the posted primary outcome information.
The study is single-group, with allocation listed as NA and no masking. The registry explicitly says it is not a randomized comparison. Thus the design does not support a controlled estimate of the effect of G-CSF stimulation, T-cell depletion, or the combined transplant approach. The record also does not report detailed eligibility criteria, baseline characteristics, or participant counts by diagnosis, limiting assessment of who the results would represent even if outcomes were available.
The study status is TERMINATED, but the reason for termination is not reported in the registry record. The record does not explain whether all planned follow-up was completed or whether termination affected the availability of outcome data. It also does not describe missing-data handling, analysis populations, outcome-assessment procedures, or a statistical analysis plan. These omissions prevent assessment of potential bias and analytic choices.
Safety entries include serious and other adverse events, but details about timing, attribution, and term-specific denominators are not reported. Deaths are not reported in the registry record. The registry also lists no analyses, so the posted record cannot be used to evaluate model assumptions or statistical adjustments. Taken together, these limitations make the record useful for understanding the study's stated aims and design, but insufficient for drawing conclusions about its primary outcome or comparative effects.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome — NCT00228813.
- Registry record last updated 2017-09-11; status last verified 2017-08.
- Results first posted 2016-07-06.
- 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
The registry describes a terminated, single-group feasibility study sponsored by Emory University. It lists enrollment as 8, status as TERMINATED, allocation as NA, and masking as NONE. The study concerned patients with hematologic malignancies or bone marrow failure syndrome receiving in vivo T-cell-depleted G-CSF-stimulated bone marrow from partially mismatched related donors.
The primary endpoint was the number reaching engraftment by Day 45. The study also aimed to evaluate acute and chronic graft-versus-host disease. The primary result and numeric graft-versus-host disease results are not reported in the registry record. Three outcome measures are listed, and no analyses are listed.
Serious adverse events are recorded for 4 participants among 8 at risk; other adverse events are recorded for 0 among 8 at risk. The reasons for termination, missing-data procedures, analysis populations, and deaths are not reported in the registry record. The single-group design does not provide a randomized comparison.