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 AlloHCT From Matched Unrelated Donors in Pts w/ Advanced Hematologic Malignancies & Disorders (NCT00547196), a non-randomized na study. The primary outcome is Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00547196 |
| Phase | NA |
| Condition | Leukemia, Lymphoma, Myelodysplastic Syndromes |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | City of Hope Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with leukemia, lymphoma, or myelodysplastic syndromes undergoing allogeneic transplantation with unrelated umbilical cord blood. The registry describes the study purpose as treating patients with relapsed or refractory hematologic cancer. Enrollment was 10.
Intervention
One of four conditioning regimens before transplantation. The regimens combine fludarabine with other chemotherapy drugs, with total-body irradiation included in some regimens. Patients received two combined umbilical cord blood units. The registry also lists supportive and graft-versus-host disease prophylaxis drugs, though some regimen descriptions are incomplete.
Comparator
The four regimens form parallel study arms, but allocation was non-randomized. They are not a like-for-like control comparison, so observed differences between arms cannot be attributed to the conditioning regimen alone.
Primary question
The primary endpoint is the number of patients alive at day 100 after transplantation divided by the number who underwent transplantation. The registry does not report a numerical result for this endpoint. A secondary outcome is survival at day 180, for which a numerical result is also not reported in the registry record.
The registry frames the study as an examination of four chemotherapy regimens, with or without total-body irradiation, before umbilical cord blood transplantation. The intended setting is relapsed or refractory hematologic cancer. The listed conditions also include leukemia, lymphoma, and myelodysplastic syndromes.
The clinical question is descriptive: what survival and safety outcomes were recorded among patients treated in each regimen arm? It is not a randomized test of whether one regimen is superior to another. Because treatment assignment was non-randomized, characteristics that influenced regimen selection could also be related to outcomes. The registry record does not report enough information to assess how such factors were handled.
The planned primary outcome concerns survival through day 100 after transplantation. The registry defines it as a count of surviving patients divided by the number who underwent transplantation. The record does not provide the count or resulting percentage. It also identifies survival at day 180 as a secondary outcome, without reporting a numerical result for that measure. The study therefore raises a clear clinical question, but the posted results do not provide the survival estimates needed to answer it numerically.
3. Trial Design
This completed study, NCT00547196, was sponsored by City of Hope Medical Center. It was listed as non-randomized, unmasked, and parallel, with an enrollment of 10. The phase is recorded as not applicable. The start date was 2005-08-16, and primary completion was 2009-11-11.
The study compared four conditioning approaches before allogeneic hematopoietic stem cell transplantation using umbilical cord blood. Regimen I included fractionated total-body irradiation, cyclophosphamide, and fludarabine. Regimen II included busulfan, fludarabine, and melphalan. Regimen III included total-body irradiation, cyclophosphamide, and fludarabine. Regimen IV included fludarabine and melphalan. The regimen descriptions state that patients received two combined units of umbilical cord blood on transplant day. Some accompanying treatment descriptions are truncated in the registry record.
The record lists cyclosporine and mycophenolate mofetil among the interventions, and describes graft-versus-host disease prophylaxis in the study rationale. Filgrastim is also listed. The study rationale explains that conditioning is intended to suppress disease and reduce the chance of graft rejection, while prophylaxis is intended to reduce immune reactions against normal tissues. These statements describe the study’s rationale, not evidence that a regimen achieved those aims.
Non-randomized allocation means that assignment to a regimen was not determined by randomization. The groups may therefore differ for reasons associated with treatment selection, and an observed difference would not by itself establish a treatment effect. The registry explicitly cautions that the arms are not a like-for-like control comparison.
The trial is marked completed, but completion alone does not indicate that every outcome was reported. The record lists two outcome measures and no analyses. It does not report the method used to assign patients to regimens, a sample-size justification, or a statistical analysis plan. Those design details are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Regimen I (FTBI, Cyclophosphamide, Fludarabine) | Regimen II (Busulfan, Fludarabine, Melphalan) | Regimen III (TBI, Cyclophosphamide, Fludarabine) | Regimen IV (Fludarabine, Melphalan) | Unassigned |
|---|---|---|---|---|---|
| STARTED | 5 | 0 | 1 | 2 | 2 |
| COMPLETED | 5 | 0 | 1 | 2 | 2 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: No subject in the Regimen II arm.
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.
Population: No subject in Regimen II.
| Characteristic | Unit | Regimen I (FTBI, Cyclophosphamide, Fludarabine) | Regimen II (Busulfan, Fludarabine, Melphalan) | Regimen III (TBI, Cyclophosphamide, Fludarabine) | Regimen IV (Fludarabine, Melphalan) | Unassigned | Total |
|---|---|---|---|---|---|---|---|
| Participants analysed | 5 | 0 | 1 | 2 | 2 | 10 | |
| Age, Continuous | years | 14 (11 to 18) | — | 58 (58 to 58) | 35 (16 to 54) | 18.5 (9 to 28) | 15 (9 to 58) |
| Sex: Female, Male: Female | Participants | 1 | — | 1 | 1 | 0 | 3 |
| Sex: Female, Male: Male | Participants | 4 | — | 0 | 1 | 2 | 7 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | — | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 1 | — | 1 | 0 | 0 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | — | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | — | 0 | 0 | 1 | 2 |
| Race (NIH/OMB): White | Participants | 3 | — | 0 | 2 | 1 | 6 |
| Race (NIH/OMB): More than one race | Participants | 0 | — | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | — | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 5 | — | 1 | 2 | 2 | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB), measured over From transplant to Day 100 post-transplant.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of surviving patients |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is survival at day 100 after allogeneic transplantation from umbilical cord blood. The registry defines the measure as the number of surviving patients at day 100 divided by the number of patients who underwent transplantation, expressed as a percentage of surviving patients. The timeframe begins at transplantation and ends at day 100 post-transplant.
Survival at a specified follow-up point is a direct outcome: it records whether patients are alive at that time, rather than relying on a laboratory marker or an indirect measure of disease status. A fixed time point can make outcomes easier to describe consistently, provided the relevant follow-up and denominator are clearly defined. This is general statistical context; it does not establish how completely follow-up was achieved in this study.
The registry’s population field for the primary outcome states, “No subject in the Regimen II arm.” The number alive at day 100 and the resulting percentage are not reported in the registry record. The reason for the population statement, and how it relates to the full enrolled group, is also not reported in the registry record.
Survival at day 180 is listed as a secondary outcome, but a numerical result is not reported in the registry record. Without reported survival counts, percentages, or uncertainty intervals, the outcome definitions alone cannot show the survival experience in any arm or support a numerical comparison among regimens.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies survival at day 100 after transplantation as the primary endpoint, but does not report a numerical estimate for it. The record’s primary outcome population field states that there was no subject in the Regimen II arm. It does not provide the day-100 survivor counts or percentages for the other arms. Survival at day 180 is listed as a secondary outcome, but its result is not reported in the registry record either.
Consequently, the posted outcome information does not establish the observed survival rate in the study or allow the reader to assess its precision. No confidence interval is reported for the primary or secondary survival endpoint. A confidence interval is a statistical expression of the uncertainty around an estimate; its width depends on the information available and the analysis used. Without an estimate and interval, the amount of uncertainty around survival cannot be evaluated from the registry results.
The study’s non-randomized design is central to interpretation. If outcomes differed across the regimen arms, the differences could reflect the regimens, differences among the patients receiving them, or other factors. Randomization is one way to reduce systematic differences between treatment groups, but it was not used. The registry cautions that the arms are not a like-for-like control comparison. The record does not report adjustment methods that might address differences in patient characteristics.
Enrollment was 10, and the adverse-event groups report different numbers at risk across regimens, including no participants at risk in Regimen II. These group counts are not a substitute for the missing survival results. The registry does not explain the relationship between the total enrollment and the populations used for each reported outcome. Without those definitions, it is not possible to determine how much outcome information contributes to an estimate or how missing follow-up might affect it.
The record lists two outcome measures and no analyses. It does not report hypothesis tests, p-values, effect estimates, or confidence intervals for the survival outcomes. Therefore, statistical evidence for differences among regimens is not available in the posted results. This does not demonstrate that the regimens had identical outcomes; it means the registry record does not provide the analysis needed to assess differences.
Safety information is reported separately by regimen, including counts of participants at risk and affected for serious and other adverse events, as well as deaths. These counts describe the record’s safety reporting, but the small and unequal group sizes limit comparisons. The listed serious-event terms and percentages also need to be interpreted within the registry’s reporting framework; the record does not specify all denominator and event-aggregation details needed to infer comparative risk.
Overall, the record documents a completed study and its regimen structure, but the posted outcome information is too limited to quantify day-100 or day-180 survival or to compare regimens statistically. The absence of a reported estimate should not be converted into a claim about the clinical performance of any regimen.
9. Statistical Methods Explained
Statistical methods can help distinguish an observed pattern from a dependable estimate, but the registry record contains little information about the analyses used in this study. It identifies outcome measures and safety counts, yet lists no analyses. The statistical plan, including any prespecified comparisons, is not reported in the registry record.
What does the primary endpoint estimate?
The primary outcome is defined as the number alive at day 100 divided by the number of patients who underwent transplantation. This is a proportion at a fixed follow-up time. To interpret it, readers need both the numerator and denominator, along with a clear account of who was eligible for the endpoint and whether follow-up was complete. The registry does not report the primary outcome value. Its population statement says there was no subject in the Regimen II arm, but the reason and implications of that statement are not reported in the registry record.
A proportion is an estimate based on the observed participants, rather than a guarantee of the outcome for all similar patients. Its precision depends in part on the amount of information contributing to it. A confidence interval can express that precision, but no confidence interval for survival is reported. General statistical context: a wider interval indicates greater uncertainty, while a narrower interval indicates greater precision, conditional on the methods and assumptions used.
Why does allocation matter?
Allocation was non-randomized. In a randomized comparison, assignment by chance is intended to balance measured and unmeasured characteristics across groups over repeated studies. In a non-randomized study, the clinical factors that lead to a regimen choice may also be related to outcomes. As a result, raw differences between arms can be confounded. This is a general statistical concern, not a claim that any specific factor affected this study. The registry does not report a method for adjusting comparisons, nor does it report a direct statistical comparison among the arms.
The study is described as parallel, meaning the arms represent groups receiving different regimens rather than a crossover arrangement. That structure does not make the groups exchangeable. The registry’s own warning that the arms are not a like-for-like control comparison is important when considering any apparent difference in survival or safety counts.
What can the sample-size illustrations tell readers?
The sample-size assumptions describe planning illustrations computed from an observed effect. They specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event calculations, the stated quantity is the total number of events; for other calculations, it is participants per arm. These are planning illustrations, not the study’s original design justification and not observed power. The registry does not report the trial’s sample-size justification.
Such illustrations ask what sample size might be needed under stated assumptions to detect an effect of a chosen share of the observed effect. They do not show that a study with a particular enrollment had a specified probability of finding a result. The study enrollment was 10, and the registry lists no analyses. Any sample-size illustration should therefore be kept separate from the trial’s actual outcomes and from claims about the reliability of its findings. General statistical context: power calculations depend on assumptions about the effect, variability, significance threshold, and allocation; changing assumptions changes the planning estimate.
Why are uncertainty and dispersion relevant?
Survival proportions and adverse-event counts can be unstable when the numbers of participants are small. One participant’s outcome can have a substantial influence on a group estimate when few participants contribute. This is a general statistical principle; it does not supply an unreported estimate for this trial. Uncertainty intervals would help readers understand the precision of an outcome estimate, but survival intervals are not reported in the registry record.
Over-dispersion is a general concern for count outcomes when observed variation exceeds what a simple probability model assumes. It can make standard errors too small if the model fails to account for that extra variation. The registry does not describe a model for the adverse-event counts or report enough analysis detail to determine whether over-dispersion was assessed. No particular model should be inferred from the posted information.
What analyses are actually reported?
The registry lists two outcome measures: survival at day 100 and survival at day 180. It lists no analyses. It does not report survival estimates, hypothesis tests, p-values, confidence intervals, model specifications, or adjustment for baseline differences. It also does not report a statistical analysis plan. These omissions prevent readers from checking how the endpoint definitions were applied or how uncertainty and potential confounding were handled.
Recalculated results, when present in a statistical presentation, are simple unadjusted calculations from posted summary statistics. They do not replace a sponsor’s own analysis. If a discrepancy is identified, the sponsor’s own analysis takes precedence. The registry record does not report an analysis for the survival outcomes to compare with a recalculation. A planning illustration remains a planning illustration and should never be described as observed power.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary survival outcome at day 100 and a secondary survival outcome at day 180. It does not report a statistical analysis plan or explain whether other outcomes, time points, or regimen comparisons were considered. The approach to multiplicity is therefore not reported in the registry record.
Multiplicity arises when several outcomes or comparisons are examined. In general, making many statistical tests can increase the chance of at least one apparently unusual result. Methods to address this depend on which comparisons were planned and how they were prioritized. Without a reported plan or analyses, it is not possible to determine whether any adjustment was intended or necessary in this study.
Missing-data handling is also not reported in the registry record. The primary outcome description defines a denominator as patients who underwent transplantation, while the population field says there was no subject in the Regimen II arm. The record does not explain the population statement, report follow-up completeness, or describe how patients without an observed day-100 status were handled. The same information is not reported for survival at day 180.
Analysis populations are not fully described. The registry does not state whether the survival outcomes were assessed in all enrolled patients, only patients who underwent transplantation, or another defined group. It also does not explain how the enrollment of 10 relates to the group-specific populations reported for safety. These gaps matter because the population included in an analysis determines what its estimate describes.
General statistical context: excluding participants with missing outcomes can bias an estimate if missingness is related to prognosis or treatment. Methods that account for missingness require assumptions, which should be described and assessed. The registry provides no information to judge whether missing outcome data occurred or how any such data were handled.
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 (FTBI, Cyclophosphamide, Fludarabine) | 2 | 5 | 40.0% |
| Regimen III (TBI, Cyclophosphamide, Fludarabine) | 1 | 1 | 100.0% |
| Regimen IV (Fludarabine, Melphalan) | 1 | 2 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Regimen I (FTBI, Cyclophosphamide, Fludarabine) | 3 of 5 |
| Regimen II (Busulfan, Fludarabine, Melphalan) | 0 of 0 |
| Regimen III (TBI, Cyclophosphamide, Fludarabine) | 1 of 1 |
| Regimen IV (Fludarabine, Melphalan) | 2 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 (FTBI, Cyclophosphamide, Fludarabine) | 3 of 5 |
| Regimen II (Busulfan, Fludarabine, Melphalan) | 0 of 0 |
| Regimen III (TBI, Cyclophosphamide, Fludarabine) | 1 of 1 |
| Regimen IV (Fludarabine, Melphalan) | 1 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Regimen I (FTBI, Cyclophosphamide, Fludarabine) | Regimen II (Busulfan, Fludarabine, Melphalan) | Regimen III (TBI, Cyclophosphamide, Fludarabine) | Regimen IV (Fludarabine, Melphalan) |
|---|---|---|---|---|---|
| Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L) | Infections and infestations | 1 of 5 | 0 of 0 | 1 of 1 | 0 of 2 |
| Hemorrhage, GU | Blood and lymphatic system disorders | 0 of 5 | 0 of 0 | 0 of 1 | 1 of 2 |
| Infection, Bacterial (COH) | Infections and infestations | 0 of 5 | 0 of 0 | 1 of 1 | 0 of 2 |
| ALT, SGPT (serum glutamic pyruvic transaminase) | Investigations | 1 of 5 | 0 of 0 | 0 of 1 | 0 of 2 |
| AST, SGOT(serum glutamic oxaloacetic transaminase) | Investigations | 1 of 5 | 0 of 0 | 0 of 1 | 0 of 2 |
| Creatinine | Investigations | 0 of 5 | 0 of 0 | 0 of 1 | 1 of 2 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 1 of 5 | 0 of 0 | 0 of 1 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Regimen I (FTBI, Cyclophosphamide, Fludarabine) | Regimen II (Busulfan, Fludarabine, Melphalan) | Regimen III (TBI, Cyclophosphamide, Fludarabine) | Regimen IV (Fludarabine, Melphalan) |
|---|---|---|---|---|---|
| Hypertension | Cardiac disorders | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Nausea | Gastrointestinal disorders | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Vomiting | Gastrointestinal disorders | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Pain | General disorders | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Glucose, serum-high (hyperglycemia) | Investigations | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Magnesium, serum-low (hypomagnesemia) | Investigations | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Rash/desquamation | Skin and subcutaneous tissue disorders | 3 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Diarrhea | Gastrointestinal disorders | 2 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
| Mucositis/stomatitis (functional/symptomatic) | Gastrointestinal disorders | 3 of 5 | 0 of 0 | 0 of 1 | 1 of 2 |
| Weight gain | General disorders | 2 of 5 | 0 of 0 | 1 of 1 | 1 of 2 |
Serious events with the highest arm-level rate: infection with grade 3 or 4 neutrophils (anc <1.0 x 10e9/l) (100.0%), infection, bacterial (coh) (100.0%), hemorrhage, gu (50.0%), creatinine (50.0%), alt, sgpt (serum glutamic pyruvic transaminase) (20.0%).
The safety record reports group-specific counts of participants at risk and affected for serious adverse events and other adverse events, along with deaths. For Regimen I, the serious-event group lists 5 at risk and 2 affected; the other-event group lists 5 at risk and 3 affected, with 3 deaths. For Regimen II, all these counts are 0. For Regimen III, the serious-event group lists 1 at risk and 1 affected; the other-event group lists 1 at risk and 1 affected, with 1 death. For Regimen IV, the serious-event group lists 2 at risk and 1 affected; the other-event group lists 2 at risk and 1 affected, with 2 deaths.
The serious-event terms listed include “Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)” at 100.0%, “Infection, Bacterial (COH)” at 100.0%, “Hemorrhage, GU” at 50.0%, “Creatinine” at 50.0%, “ALT, SGPT (serum glutamic pyruvic transaminase)” at 20.0%, and “AST, SGOT(serum glutamic oxaloacetic transaminase)” at 20.0%.
These figures are descriptive, not evidence of comparative safety. Group sizes are small and differ, and Regimen II has no participants represented in the safety counts. The registry does not report all details about event definitions, duration of observation, or the denominators underlying the listed percentages. Counts and percentages should therefore be read as registry-reported safety information, not as stable estimates of relative risk.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed, non-randomized study examined four conditioning regimens before umbilical cord blood transplantation. It records enrollment of 10, identifies day-100 survival as the primary endpoint, and lists day-180 survival as a secondary outcome. It also provides group-level adverse-event counts and selected serious-event terms. These are useful descriptions of what the registry set out to study and what safety information it recorded.
The record does not report numerical survival results at either time point. It does not provide the estimates or uncertainty measures needed to describe survival precisely, and it lists no analyses. The absence of a reported result is not evidence that survival was high, low, or equivalent across regimens. It also does not establish that there was no difference between groups.
Because allocation was non-randomized, any outcome differences across arms would need cautious interpretation. The groups cannot be assumed to be comparable, and the registry warns that they are not a like-for-like control comparison. The record does not report adjustment methods or enough detail about patient characteristics to assess possible confounding. The small enrollment and sparse safety group counts further limit how much can be inferred from arm-level descriptions.
The serious-event percentages identify terms appearing in the registry’s safety reporting, but the record does not provide the complete context needed to interpret those percentages as comparative risks. The safety counts do not answer the primary survival question, and they do not establish the cause of any death or event.
In short, the posted information describes the study’s purpose, structure, endpoints, and some safety reporting. It does not provide a numerical basis for judging comparative survival or for claiming that one conditioning regimen performed better or worse. Those conclusions would require outcome estimates, uncertainty measures, clear analysis populations, and appropriate consideration of the non-randomized design.
13. Why This Trial Matters Statistically
This study illustrates a common challenge in clinical trial interpretation: a registry can specify an important outcome without reporting the numerical result needed to evaluate it. Survival at day 100 is clearly defined, and survival at day 180 is listed as a secondary measure, but neither numerical survival result is reported in the registry record. Endpoint definitions are not a substitute for outcome estimates.
It also shows why design labels matter. Four parallel regimen arms may look like a comparative study, but non-randomized allocation does not ensure that the groups are comparable. The registry explicitly warns against treating the arms as like-for-like controls. That warning should guide interpretation of any differences in event counts or other descriptive results.
The enrollment of 10 is important context for the safety figures. The group-specific counts are sparse, and Regimen II has zero participants represented in the reported safety groups. Descriptive percentages can look precise even when based on little information; readers need denominators and uncertainty to understand their stability. The registry does not provide the full analytic context required for that assessment.
The study also demonstrates the distinction between an original design and a planning illustration. A sample-size calculation based on an observed effect can help illustrate what a future study might require under specified assumptions. It does not establish that the completed study had adequate power, and it is not observed power. The registry does not report its original sample-size justification.
For biostatisticians and clinical researchers, the central lesson is to separate what the record documents from what it leaves unresolved. Here, the design, endpoint definitions, and some safety counts are documented. Survival estimates, statistical analyses, missing-data methods, and multiplicity procedures are not. Recognizing these boundaries prevents descriptive registry information from being mistaken for a comparative efficacy analysis.
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 primary limitation is the absence of numerical results for the day-100 survival endpoint. The day-180 secondary survival result is also not reported in the registry record. Without those estimates, readers cannot assess the observed survival experience or its precision.
Enrollment was 10, and the safety counts are sparse and unequal across the regimen groups. Regimen II has no participants in the reported safety groups, and the primary outcome population statement also says there was no subject in that arm. The reason for these zero counts, and the relationship between the enrolled population and each outcome population, are not reported in the registry record.
The study was non-randomized and unmasked. The registry warns that the arms are not a like-for-like control comparison. Patient characteristics, regimen-selection criteria, and adjustment methods are not reported in the registry record, limiting the ability to assess confounding. The unmasked design may also matter for outcomes involving clinical judgment, but the record does not describe outcome assessment procedures.
The regimen descriptions are incomplete in places, and the record does not provide a detailed statistical analysis plan, sample-size justification, missing-data approach, multiplicity method, or full definition of analysis populations. It lists two outcome measures and no analyses. These limitations prevent a detailed evaluation of how the planned endpoints were analyzed and how uncertainty was quantified.
Finally, the listed serious-event percentages are not accompanied by all denominator and follow-up details needed for a comparative safety interpretation. The safety information is best treated as descriptive registry reporting rather than as a reliable ranking of the regimens.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: AlloHCT From Matched Unrelated Donors in Pts w/ Advanced Hematologic Malignancies & Disorders — NCT00547196.
- Registry record last updated 2024-06-14; status last verified 2024-06.
- Results first posted 2023-04-13.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT00547196 is a completed, non-randomized, unmasked, parallel study sponsored by City of Hope Medical Center. Enrollment was 10. The study examined four conditioning regimens before allogeneic transplantation using umbilical cord blood in patients with hematologic malignancies or disorders.
The primary outcome was survival at day 100 after transplantation; survival at day 180 was listed as a secondary outcome. Numerical results for these outcomes are not reported in the registry record, and the record lists no analyses. The primary outcome population field states that there was no subject in the Regimen II arm.
Safety information includes group-specific counts of participants at risk and affected for serious and other adverse events, deaths, and selected serious-event terms. Because allocation was non-randomized, the arms are not a like-for-like control comparison. The posted information supports a description of the study design and recorded safety counts, but not a numerical comparison of survival or a conclusion that one regimen performed better than another.