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 Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders (NCT00544115), a non-randomized phase 2 study. The primary outcome is Neutrophil Engraftment - The Days Till ANC Recovery.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00544115 |
| Phase | Phase 2 |
| Condition | Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 260 |
| Arms in the primary outcome | n/a |
| Lead sponsor | City of Hope Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerns patients with advanced hematologic malignancies or hematological disorders receiving an allogeneic peripheral blood stem cell transplant from a matched unrelated donor. The listed conditions include leukemia, lymphoma, multiple myeloma and plasma cell neoplasm, myelodysplastic syndromes, chronic myeloproliferative disorders, and other hematologic or nonmalignant conditions. The registry does not give a more detailed description of the analyzed population for the primary endpoint.
Intervention
The intervention is transplantation after one of six conditioning regimens. The schedules use combinations of total-body irradiation and drugs including cyclophosphamide, busulfan, etoposide, fludarabine phosphate, or melphalan. The registry also lists drugs used in the transplant setting, including medicines intended to prevent or manage immune complications. The regimens are not described as a single uniform treatment.
Comparator
The allocation is nonrandomized. The record describes six regimens, but does not establish a randomized control group or a like-for-like comparison among them. Differences between regimen groups could reflect differences in the patients, clinical circumstances, or treatment selection as well as the regimens themselves. The registry does not report an adjusted comparison that would resolve those concerns.
Primary question
The primary endpoint asks how long it takes after transplant for neutrophil engraftment, defined by the first of three consecutive days with an absolute neutrophil count > 500/µL. The endpoint is assessed up to 180 days post transplant. The registry identifies the summary measure as a median with a 95% confidence interval, but the numerical median and confidence interval are not reported in the registry record. Consequently, the record does not provide a numerical estimate with which to describe the observed time to engraftment.
3. Trial Design
This is a Phase II, nonrandomized, unmasked, parallel-group trial sponsored by City of Hope Medical Center. The registry records enrollment of 260. Its title and official title describe allogeneic peripheral blood stem cell transplantation from matched unrelated donors in people with advanced hematologic malignancies or hematological disorders. The planned treatment approaches are organized as six conditioning regimens rather than as a randomized comparison against a control treatment.
The regimens vary in their use and timing of total-body irradiation and chemotherapy. Regimen I uses total-body irradiation with cyclophosphamide, with an alternative schedule also described. Regimen II uses busulfan and cyclophosphamide; Regimen III uses total-body irradiation and etoposide; Regimen IV uses fludarabine phosphate and melphalan; Regimen V uses fludarabine phosphate and total-body irradiation; and Regimen VI uses busulfan and fludarabine phosphate. These descriptions identify clinically different schedules, not interchangeable versions of one treatment.
The registry lists a start date of 2001-10-16 and a primary completion date of 2007-03-13. Its status is recorded as active, not recruiting. The study summary describes the rationale for transplant conditioning and for medicines used around transplant to reduce immune complications. These explanations describe the study’s rationale; they are not evidence of the outcomes.
Because assignment was nonrandomized, the groups should not be read as if treatment allocation had balanced their characteristics by design. The registry record does not report the allocation process, baseline characteristics by regimen, a control group, or an adjusted analysis that would support a causal contrast among regimens. It also does not report the protocol’s sample-size justification or a detailed statistical analysis plan. Those omissions limit what can be inferred from comparisons between the regimen groups.
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 | Regimen I | Regimen II | Regimen III | Regimen IV | Regimen V | Regimen VI |
|---|---|---|---|---|---|---|
| STARTED | 62 | 17 | 130 | 4 | 40 | 2 |
| COMPLETED | 62 | 17 | 130 | 4 | 40 | 2 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Some patients had engraftment failure and some had missing data. Therefore, the overall number of participants analyzed in each regimen is less than the overall number in the Participant Flow module.
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.
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | Regimen I | Regimen II | Regimen III | Regimen IV | Regimen V | Regimen VI |
|---|---|---|---|---|---|---|---|
| Participants analysed | 62 | 17 | 130 | 4 | 40 | 2 | |
| Age, Continuous | years | 38 (14 to 62) | 52 (12 to 62) | 50 (6 to 67) | 22 (18 to 42) | 37 (7 to 58) | 44 (41 to 47) |
| Sex: Female, Male: Female | Participants | 24 | 11 | 65 | 2 | 17 | 1 |
| Sex: Female, Male: Male | Participants | 38 | 6 | 65 | 2 | 23 | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 2 | 0 | 8 | 1 | 2 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 3 | 1 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 55 | 17 | 118 | 2 | 38 | 2 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 4 | 0 | 1 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 62 | 17 | 130 | 4 | 40 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Neutrophil Engraftment - The Days Till ANC Recovery, measured over Up to 180 days post transplant.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | days |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is time from transplant to neutrophil engraftment, summarized as a median with a 95% confidence interval. The registry defines engraftment as the first of three consecutive days on which the absolute neutrophil count is > 500/µL, with follow-up described as up to 180 days post transplant. The measure therefore captures how long it takes to reach a specified blood-cell recovery criterion, rather than simply whether the criterion was reached at one particular visit.
As a general statistical point, a time-to-event endpoint retains information about the timing of an event. A median gives the point by which half of the relevant event-time distribution has occurred, when it can be estimated. A confidence interval describes uncertainty around that estimate; it is not a range expected to contain individual patients’ times. The registry identifies this type of summary but does not report the median or its confidence interval, so neither the observed timing nor its precision can be assessed from the record.
The record notes that some patients had engraftment failure and some had missing data. It also says the number analyzed in each regimen is lower than the corresponding number in Participant Flow. How failure and missing observations were handled in the endpoint calculation is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies time to neutrophil engraftment as the primary endpoint and specifies a median with a 95% confidence interval as its summary. However, it does not report a numerical median or confidence interval. There is therefore no posted numerical primary result from which to describe the typical time to engraftment or the precision of that estimate. The endpoint definition and timeframe describe what was intended to be measured; they do not substitute for the result.
The enrollment figure is 260, but it should not be treated as the analyzed denominator for each regimen or for the primary endpoint. The registry explicitly says that the number analyzed in each regimen is smaller than the number in Participant Flow because of engraftment failure and missing data. The actual endpoint analysis counts by regimen are not reported in the registry record. It would be inappropriate to infer them from the enrollment figure or from the adverse-event counts.
The study has six regimens and nonrandomized allocation. Any apparent difference among regimens would be descriptive unless the record also established a sound basis for a comparative interpretation. Patients may have entered different regimens for reasons related to their clinical circumstances, and the registry does not provide baseline comparisons or an adjusted treatment-effect analysis. No numerical between-regimen estimates for the primary endpoint are reported.
The registry lists two outcome measures and zero analyses. This means no analysis records are listed; it does not establish that analyses were not conducted outside the registry. A secondary outcome is two-year overall survival, but its numerical result is not reported in the registry record. The posted information therefore does not support a numerical account of survival or a comparison of survival across regimens.
Safety fields report serious adverse-event affected counts, other adverse-event affected counts, and deaths for each regimen. These are useful descriptions of what the record lists, but they do not establish that an event was caused by a regimen. The nonrandomized design, differing regimen group sizes, and absence of a reported causal analysis prevent these counts from serving as direct evidence of comparative safety. The record also does not describe event attribution or provide enough detail to interpret the listed terms as comparative risks.
Overall, the registry describes the study question, the treatment schedules, and some safety counts, but it does not post the numerical primary endpoint result or a treatment-effect estimate. Any conclusion about the amount of time to engraftment, precision, or differences between regimens would go beyond the reported results.
9. Statistical Methods Explained
The primary outcome is a time-to-event measure: the event is neutrophil engraftment, and the time origin is transplant. The registry defines the event using a threshold of absolute neutrophil count > 500/µL sustained for three consecutive days. This operational definition matters because the time recorded depends on the stated threshold and confirmation rule. The endpoint is not a general measure of all aspects of recovery, nor does it directly summarize every transplant outcome.
What does a median time summarize?
As a general statistical explanation, the median time is the midpoint of an event-time distribution: half of the relevant observations have event times at or below it and half at or above it, when the median is estimable. It is less sensitive to unusually long or short times than an arithmetic mean. For time-to-event outcomes, some participants may not have a recorded event during follow-up. Methods can account for incomplete follow-up, but the registry record does not say which method was used for this endpoint. It also does not post the numerical median, so the summary cannot be interpreted quantitatively.
A 95% confidence interval expresses uncertainty about an estimated quantity under the assumptions of the method used. It is not a statement that a specified proportion of individual event times falls within the interval. A narrower interval generally indicates greater precision, while a wider interval indicates more uncertainty. Here, the interval is named as the dispersion measure, but its limits are not reported in the registry record. Its width, and therefore the precision of the estimate, cannot be assessed.
Why can participants be absent from an endpoint analysis?
The registry specifically notes engraftment failure and missing data, and says each regimen’s analyzed count is lower than its Participant Flow count. These situations are not necessarily equivalent. Failure to meet an event definition, loss of information needed to determine a time, and incomplete follow-up can have different statistical implications. General methods may handle these situations in different ways, but the registry does not report the endpoint-specific rules, the number affected for each reason, or any sensitivity analysis. The direction and possible size of bias from missing or unevaluable outcomes therefore cannot be determined from the record.
What does the nonrandomized design imply?
In a randomized study, assignment by chance is intended to reduce systematic differences between treatment groups. This trial is recorded as nonrandomized, so that protection is not established. As a general principle, group differences in outcomes can reflect treatment, patient characteristics, treatment-selection decisions, or other differences. A parallel-group structure means that regimens are organized into separate groups, but it does not by itself make those groups comparable. The registry does not report a randomized allocation procedure, a detailed adjustment strategy, or adjusted treatment-effect estimates.
The record describes six conditioning schedules, with differences in drug combinations and timing of total-body irradiation. A comparison across these groups would require careful attention to who received each regimen and why. Without baseline characteristics and a reported comparative analysis, a simple difference in group summaries could not separate regimen effects from other explanations. No numerical comparative primary-endpoint results are posted.
How should planning calculations be understood?
As a general distinction, a sample-size calculation is a planning exercise, whereas power is a design property under assumptions about the effect and variability. The record’s planning assumptions describe illustrations based on an observed effect, two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. For time-to-event illustrations, the stated convention is to report the total number of events; other illustrations use participants per arm. These are planning conventions, not observed trial outcomes and not observed power. Numerical planning outputs are not reported in the registry record, and the record does not report that these illustrations formed the trial’s original design rationale.
The record’s method note says that only sponsor-reported hazard ratios are used for time-to-event outcomes. No sponsor-reported hazard ratio for this primary endpoint is included in the posted results. A hazard ratio, as a general concept, compares event rates over time between groups under a model; it is not the same quantity as a median time. No hazard-ratio interpretation or reconstructed estimate is warranted without a reported estimate and its uncertainty.
In short, the record identifies the endpoint, summary measure, and some planning conventions, but does not provide the primary numerical result, the analysis details, or an inferential comparison among regimens. General statistical concepts can clarify what these methods would mean, but they cannot fill in the missing trial-specific estimates.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures, including the primary neutrophil-engraftment endpoint and the secondary outcome of two-year overall survival. It lists zero analyses. This registry entry therefore does not provide analysis records for either outcome. The number of outcome measures alone does not establish that a multiplicity adjustment was needed or used; the planned hypotheses and analysis hierarchy are not reported in the registry record.
As a general statistical point, examining multiple outcomes or multiple comparisons can increase the chance of a false-positive finding if each is assessed independently. Methods such as prespecifying a primary comparison or adjusting for multiple testing can address particular multiplicity concerns. Whether any such approach was used in this trial is not reported in the registry record. There is also no reported interim-analysis plan, stopping rule, or statistical analysis plan to clarify how repeated or multiple analyses were managed.
For missingness, the registry says that some patients had engraftment failure and some had missing data, so the number analyzed in each regimen is lower than the number in Participant Flow. It does not report the counts attributable to each reason, the endpoint analysis population definition, or the handling of those observations. The record does not state whether missingness was assumed to be related to observed or unobserved information, nor does it describe imputation or sensitivity analyses. These details matter because the treatment of missing or unevaluable event times can affect the estimate and its uncertainty.
The enrollment figure is not a substitute for the endpoint analysis population. The registry does not provide regimen-specific analyzed denominators for the primary endpoint, and they should not be reconstructed from other counts. Accordingly, the posted record does not allow assessment of whether missing data or multiplicity materially affected the reported 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 |
|---|---|---|---|
| Regimen I | 4 | 62 | 6.5% |
| Regimen II | 1 | 17 | 5.9% |
| Regimen III | 7 | 130 | 5.4% |
| Regimen IV | 0 | 4 | 0.0% |
| Regimen V | 3 | 40 | 7.5% |
| Regimen VI | 1 | 2 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Regimen I | 45 of 62 |
| Regimen II | 14 of 17 |
| Regimen III | 103 of 130 |
| Regimen IV | 2 of 4 |
| Regimen V | 32 of 40 |
| Regimen VI | 1 of 2 |
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 |
|---|---|
| Regimen I | 14 of 62 |
| Regimen II | 4 of 17 |
| Regimen III | 38 of 130 |
| Regimen IV | 0 of 4 |
| Regimen V | 10 of 40 |
| Regimen VI | 1 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Regimen I | Regimen II | Regimen III | Regimen IV | Regimen V | Regimen VI |
|---|---|---|---|---|---|---|---|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 62 | 0 of 17 | 4 of 130 | 0 of 4 | 2 of 40 | 0 of 2 |
| Infection with unknown ANC | Infections and infestations | 1 of 62 | 0 of 17 | 2 of 130 | 0 of 4 | 1 of 40 | 1 of 2 |
| Infection without neutropenia | Infections and infestations | 1 of 62 | 0 of 17 | 1 of 130 | 0 of 4 | 1 of 40 | 1 of 2 |
| Supraventricular and nodal arrhythmia | Cardiac disorders | 2 of 62 | 0 of 17 | 0 of 130 | 0 of 4 | 1 of 40 | 0 of 2 |
| Bilirubin associated with GVHD for BMT studies, if specified in the protocol. | Hepatobiliary disorders | 2 of 62 | 0 of 17 | 0 of 130 | 0 of 4 | 1 of 40 | 0 of 2 |
| Bilirubin (hyperbilirubinemia) | Investigations | 1 of 62 | 0 of 17 | 2 of 130 | 0 of 4 | 0 of 40 | 0 of 2 |
| Somnolence/depressed level of consciousness | Nervous system disorders | 2 of 62 | 0 of 17 | 1 of 130 | 0 of 4 | 0 of 40 | 0 of 2 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 0 of 62 | 0 of 17 | 2 of 130 | 0 of 4 | 1 of 40 | 0 of 2 |
| Melena/GI bleeding | Blood and lymphatic system disorders | 1 of 62 | 0 of 17 | 0 of 130 | 0 of 4 | 1 of 40 | 0 of 2 |
| Platelets | Blood and lymphatic system disorders | 2 of 62 | 0 of 17 | 0 of 130 | 0 of 4 | 0 of 40 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Regimen I | Regimen II | Regimen III | Regimen IV | Regimen V | Regimen VI |
|---|---|---|---|---|---|---|---|
| Acute graft versus host disease (GVHD) (COH) | Investigations | 4 of 62 | 0 of 17 | 19 of 130 | 0 of 4 | 2 of 40 | 0 of 2 |
| Stomatitis/pharyngitis (oral/pharyngeal mucositis) for BMT studies, if specified in the protocol. | Gastrointestinal disorders | 6 of 62 | 0 of 17 | 15 of 130 | 0 of 4 | 3 of 40 | 0 of 2 |
| Glucose, serum-high (hyperglycemia) | Investigations | 6 of 62 | 1 of 17 | 12 of 130 | 0 of 4 | 5 of 40 | 0 of 2 |
| ALT, SGPT (serum glutamic pyruvic transaminase) | Investigations | 5 of 62 | 2 of 17 | 12 of 130 | 0 of 4 | 2 of 40 | 1 of 2 |
| Graft versus host disease | Investigations | 3 of 62 | 0 of 17 | 17 of 130 | 0 of 4 | 2 of 40 | 0 of 2 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 4 of 62 | 0 of 17 | 15 of 130 | 0 of 4 | 1 of 40 | 0 of 2 |
| Infection, Bacterial (COH) | Infections and infestations | 3 of 62 | 0 of 17 | 14 of 130 | 0 of 4 | 2 of 40 | 0 of 2 |
| Potassium, serum-low (hypokalemia) | Investigations | 4 of 62 | 1 of 17 | 10 of 130 | 0 of 4 | 4 of 40 | 0 of 2 |
| Febrile neutropenia (ANC <1.0 x 10e9/L, fever >=38.5 degrees C) | Infections and infestations | 6 of 62 | 0 of 17 | 9 of 130 | 0 of 4 | 3 of 40 | 0 of 2 |
| Diarrhea associated with GVHD for BMT studies, if specified in the protocol. | Gastrointestinal disorders | 3 of 62 | 0 of 17 | 12 of 130 | 0 of 4 | 2 of 40 | 0 of 2 |
Serious events with the highest arm-level rate: infection with unknown anc (50.0%), infection without neutropenia (50.0%), neuropathy: motor (5.9%), hypoxia (5.0%), platelets (3.2%).
The safety record lists serious adverse-event counts, other adverse-event counts, and deaths by regimen. For Regimen I, it records 4 serious-event affected entries among 62 at risk, 14 other-event affected entries among 62 at risk, and 45 deaths. For Regimen II, the corresponding entries are 1 among 17, 4 among 17, and 14 deaths. Regimen III lists 7 among 130, 38 among 130, and 103 deaths. Regimen IV lists 0 among 4, 0 among 4, and 2 deaths. Regimen V lists 3 among 40, 10 among 40, and 32 deaths. Regimen VI lists 1 among 2, 1 among 2, and 1 death.
These are registry counts, not proof that an event or death was caused by a regimen. The groups differ in size, and allocation was nonrandomized, so the counts should not be treated as direct comparative risks. The record does not report event attribution or a comparative safety analysis. It also lists serious-event terms and percentages, including “Infection with unknown ANC” at 50.0% and “Infection without neutropenia” at 50.0%; the denominator and calculation context for those percentages are not reported in the registry record. The counts and terms describe the posted safety information, not a causal conclusion.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study examined matched unrelated-donor allogeneic peripheral blood stem cell transplantation after six conditioning regimens in a Phase II, nonrandomized design. It identifies neutrophil engraftment as the primary endpoint, defines the event, specifies follow-up up to 180 days post transplant, and names the median and 95% confidence interval as the summary. These details explain the question and measurement, but do not reveal the numerical result.
The record does not report the primary median or its confidence interval. It therefore does not show how long engraftment took in the analyzed population or how precisely that time was estimated. It also does not report numerical results for the secondary two-year overall survival outcome. The record’s description of endpoint definitions cannot be read as evidence that a particular result occurred.
Because the regimen groups were not randomized, the study description does not support treating them as equivalent groups that differ only in conditioning. The record does not report an adjusted comparison that could account for differences in patient characteristics or treatment selection. Nor does it provide a numerical estimate of a regimen’s effect relative to another regimen. Any causal claim about one schedule versus another would exceed the information posted.
The adverse-event fields provide counts by regimen and several event terms. They are not enough to determine whether an event was attributable to treatment, whether groups had comparable risks, or whether one regimen was safer than another. Death counts likewise do not, by themselves, indicate cause or treatment relationship.
Thus, the posted information is best read as a description of trial design, endpoint definition, incomplete endpoint analysis information, and registry safety counts. It does not establish treatment benefit or lack of benefit, and it does not permit a quantitative estimate of comparative effectiveness. The absence of a numerical primary result in the registry record should be kept separate from any clinical interpretation of transplantation or its regimens.
13. Why This Trial Matters Statistically
This trial is statistically notable because its main outcome is a time-to-event measure in a setting with multiple conditioning schedules and a nonrandomized design. The endpoint is defined by a specific neutrophil-count threshold sustained over consecutive days, so a clear operational definition is essential to make the event measurable. Reporting a median and confidence interval would ordinarily convey both the typical timing and the uncertainty around it. In this record, the summary measure is identified but the numerical estimate is not reported.
The study also illustrates the difference between enrollment and analysis populations. The registry reports enrollment of 260, yet explicitly notes that the analyzed number in each regimen is lower than the Participant Flow number because some patients had engraftment failure or missing data. Recognizing that difference is important: the denominator relevant to an endpoint may not be the same as overall enrollment, and the reasons for exclusion or missingness can affect interpretation.
Nonrandomized assignment is another central statistical feature. Six regimens can describe treatment practice and generate useful questions, but groups formed without random assignment may differ for reasons beyond the regimen itself. A causal comparison requires more than observing different outcomes; it requires a design or analysis that addresses plausible sources of group imbalance. The registry does not report a comparative adjusted estimate.
Finally, this record separates outcome definition from outcome reporting. It provides a defined primary endpoint and safety counts, while the primary numerical estimate and secondary survival result are not reported in the registry record. That distinction helps prevent readers from mistaking a detailed protocol description or registry count for a complete estimate of treatment effect.
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 principal limitation for interpreting the primary endpoint is that its numerical result is not reported in the registry record. Although the endpoint is defined and the intended summary is stated, there is no median or 95% confidence interval to assess. The secondary outcome of two-year overall survival is named, but its numerical result is also not reported.
The design is nonrandomized and includes six distinct conditioning regimens. The registry does not report the allocation process, baseline characteristics by regimen, or an adjusted comparison. Differences between groups therefore cannot be attributed to regimen alone from the posted record. No like-for-like randomized control comparison is described.
The registry notes engraftment failure and missing data, with fewer analyzed participants per regimen than in Participant Flow. It does not state the regimen-specific primary-endpoint analysis counts, how failure was handled, why observations were missing, or whether sensitivity analyses were conducted. The extent to which missing or unevaluable outcomes could change the endpoint estimate is consequently unknown.
The record lists zero analyses and does not provide a statistical analysis plan, multiplicity approach, interim-analysis information, or sample-size justification. These items are not reported in the registry record; their absence from the entry does not establish what was done outside it. Safety fields do not report event attribution or a comparative safety analysis, and their denominators and group sizes differ. The record’s status is active, not recruiting, but that status does not supply missing outcome estimates or clarify the analysis methods.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders — NCT00544115.
- Registry record last updated 2026-06-02; status last verified 2026-05.
- Results first posted 2023-05-10.
- 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 registry record describes NCT00544115, a Phase II, nonrandomized, unmasked, parallel-group study sponsored by City of Hope Medical Center. It concerns matched unrelated-donor allogeneic peripheral blood stem cell transplantation for advanced hematologic malignancies and hematological disorders, with six conditioning regimens. Enrollment is recorded as 260.
The primary endpoint is time from transplant to neutrophil engraftment, defined as the first of three consecutive days with an absolute neutrophil count > 500/µL and assessed up to 180 days post transplant. The planned summary is a median with a 95% confidence interval, but the numerical result is not reported in the registry record. Two-year overall survival is listed as a secondary outcome, without a numerical result. The registry also records serious and other adverse-event counts and deaths by regimen. Nonrandomized allocation, missing or unevaluable endpoint data, and the absence of reported comparative analyses limit interpretation of differences among regimens.