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 Combination Chemotherapy and Peripheral Blood Stem Cell Transplant Followed By Aldesleukin and Sargramostim in Treating Patients With Inflammatory Stage IIIB or Metastatic Stage IV Breast Cancer (NCT00003199), a na phase 2 study. The primary outcome is Event-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00003199 |
| Phase | Phase 2 |
| Condition | Estrogen Receptor-negative Breast Cancer, Estrogen Receptor-positive Breast Cancer, Inflammatory Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 50 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fred Hutchinson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with inflammatory stage IIIB or responsive metastatic stage IV breast cancer. The registry record lists an enrollment of 50 participants and includes several breast cancer conditions, including inflammatory breast cancer and stage IIIB and stage IV disease.
Intervention
The study concerns combination chemotherapy using busulfan, melphalan and thiotepa, followed by peripheral blood stem cell transplantation and post-engraftment immunotherapy with low-dose IL-2 and GM-CSF. The listed interventions also include tamoxifen citrate and radiation therapy; further details of their use are not reported in the registry record.
Comparator
There is no separate comparator group in the registry design. The study is identified as single-group, and allocation is recorded as NA. The record therefore does not support a like-for-like comparison with an untreated group or another treatment group.
Primary question
The primary endpoint is event-free survival, reported as a count of participants. The registry describes the primary outcome as event-free survival among patients treated with the stated chemotherapy, stem cell support and low-dose immunotherapy, with a timeframe of 11 years. The posted population description says 20 patients out of 50 have event-free survival. The exact event definition and the analysis used to summarize time to event are not reported in the registry record.
3. Trial Design
This is a phase II, single-group study sponsored by Fred Hutchinson Cancer Center. The registry lists enrollment of 50 and describes the target population as patients with inflammatory stage IIIB or responsive metastatic stage IV breast cancer. The study began in 1997-11, had primary completion in 2009-12 and is recorded as completed.
The treatment strategy in the title and official title combines busulfan, melphalan and thiotepa with autologous or syngeneic peripheral blood stem cell rescue, followed by 12 weeks of post-engraftment immunotherapy with low-dose IL-2 and GM-CSF. The intervention list names aldesleukin and sargramostim, as well as the chemotherapy drugs and peripheral blood stem cell transplantation. It also lists tamoxifen citrate and radiation therapy. Details about how those latter interventions were used are not reported in the registry record.
Allocation is recorded as NA, masking as none, and the study model as single-group. Thus, the record describes no randomized allocation or masking and does not provide a concurrent control arm. The arm description says “See Detailed Description,” but the detailed regimen and treatment sequence beyond the stated study strategy are not reported in the registry record. No protocol rationale, eligibility detail beyond the listed disease setting, or sample-size justification is reported in the registry record.
The record names overall survival and the number of participants with toxicity of low-dose IL-2 and GM-CSF as secondary outcomes. It lists 3 outcome measures and 0 analyses. That information identifies outcomes but does not specify how they were measured or analyzed. The design can describe outcomes within the treated group, but by itself it cannot separate treatment-associated changes from the natural course of disease or other influences.
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 | TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer |
|---|---|
| STARTED | 50 |
| COMPLETED | 28 |
| NOT COMPLETED | 22 |
Reasons for not completing this period:
| Reason | TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer |
|---|---|
| Refused Post-Transplant Immunotherapy | 5 |
| Lack of insurance clearance | 2 |
| Screen fail | 15 |
Analysis population for the primary outcome, as defined in the registry: Study-wide, 20 patients out of 50 have event-free survival.
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 | TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer |
|---|---|---|
| Participants analysed | 50 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 50 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 50 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 50 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 |
| Race (NIH/OMB): Asian | Participants | 7 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 4 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 37 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 50 |
6. Primary Endpoint
The registry lists the primary outcome as Event-free Survival, measured over 11 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is event-free survival, expressed in the registry as a count of participants. The posted description gives a timeframe of 11 years and states that 20 patients out of 50 have event-free survival. This is a reported count, not a complete time-to-event estimate: the record does not give the event definition, timing for individual participants, a survival curve, or a confidence interval.
General: event-free survival is a time-to-event concept. It can summarize how long participants remain without a specified event, with the event definition determined by the study protocol. It is often used when the research question concerns the period before disease progression, recurrence, death, or another prespecified event. Which events count, and how participants are followed or censored, are essential to interpreting the measure.
For this study, those operational details are not reported in the registry record. The count therefore identifies the posted primary result but does not reveal which events were included or how follow-up contributed to the result. A count alone also does not show the distribution of event times or the precision of a time-to-event estimate. The endpoint is relevant to the stated treatment question because it concerns participants’ event-free status after the treatment strategy, but the registry result does not establish a comparison with another strategy.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main posted result is that 20 patients out of 50 are described as having event-free survival, with a primary timeframe of 11 years. The outcome is a count of participants, rather than a reported hazard ratio, median event-free survival, or other comparative effect estimate. No confidence interval or p-value for the primary result is reported in the registry record. The count is informative as a description of the reported study group, but it does not quantify uncertainty around a time-to-event estimate.
The study is single-group and has no like-for-like comparator. Consequently, the posted outcome cannot establish how event-free survival would have differed under another treatment or in an untreated group. A within-group result cannot by itself attribute outcomes to the treatment components, distinguish the effect of one component from another, or account for changes that might occur over time for reasons unrelated to treatment. These are limitations of the design, not evidence for or against treatment benefit.
The endpoint description does not define what counted as an event. It also does not describe follow-up completeness, censoring, the timing of events, or the method used to estimate event-free survival. Without these details, it is not possible to judge whether the count represents a fixed-time status, a time-to-event analysis, or another summary. The registry does not report an uncertainty interval, so the precision of the primary finding cannot be assessed from the posted result.
The secondary outcomes include overall survival and the number of participants with toxicity of a combination of low-dose IL-2 and GM-CSF. Although the record names these outcomes, corresponding results and analysis details are not reported in the registry record. The adverse-event entry reports 2 participants affected by serious events among 50 at risk, lists two serious-event terms at 2.0% each, and reports 32 deaths. The record does not establish that the deaths were caused by treatment or specify how deaths relate to the primary endpoint.
Overall, the registry supports a descriptive reading of a single-group study with a reported event-free survival count. It does not supply the information needed for a controlled treatment-effect estimate, a detailed assessment of time-to-event precision, or a complete benefit-risk interpretation. Those limits should remain distinct in the registry record that the record does report.
9. Statistical Methods Explained
The registry identifies the primary outcome as event-free survival and gives its unit as participants and its parameter as a count of participants. It reports 20 patients out of 50 as having event-free survival, with a timeframe of 11 years. No analysis is listed, and the statistical method used for this outcome is not reported in the registry record. The label “event-free survival” suggests a time-to-event endpoint, but the record does not define the qualifying event or explain whether the posted count is based on a fixed-time assessment or a time-to-event analysis.
What does a count show?
A count reports how many participants are represented in a category. It does not, on its own, describe when events occurred, the duration of follow-up for each participant, or the uncertainty around a time-to-event estimate. General: a time-to-event analysis usually accounts for both event timing and participants whose event status is not observed for the full follow-up period. The treatment of those participants, often described through censoring rules, matters to interpretation. Those rules and participant-level timing are not reported in the registry record.
What would a confidence interval add?
General: a confidence interval expresses statistical uncertainty around an estimate, under the assumptions of the method used. It is not a guarantee that the true value lies within the interval, and it does not resolve limitations such as lack of a comparator or an unclear endpoint definition. No confidence interval for the primary result is reported in the registry record. Therefore, the precision of the reported count as an estimate of event-free survival cannot be assessed from the record.
What does the design permit?
The study is listed as single-group, with allocation recorded as NA and masking as none. General: random allocation can help make treatment groups comparable in expectation, while a concurrent comparator can provide a reference for interpreting outcomes. Neither a separate comparator nor randomized allocation is described. The posted result can therefore be read as an outcome description for the study group, not as an estimate of a difference between treatment strategies. It cannot isolate the contribution of chemotherapy, stem cell transplantation, IL-2 or GM-CSF.
The sample-size assumptions describe planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect is a fraction of the observed effect. For time-to-event outcomes, the assumptions specify a total number of events; for other outcomes, they specify participants per arm. These are planning conventions and do not document the trial’s own design justification. No sample-size justification is reported in the registry record, and a planning illustration is not observed power. The design itself is single-group, so the two-arm illustration should not be mistaken for the trial’s actual allocation.
Why can outcome analysis be difficult?
General: time-to-event data may require methods that account for event timing and incomplete follow-up. A count without event dates, censoring rules, or a defined event cannot be converted into a full survival analysis. No hazard ratio, median event-free survival, or survival curve is reported in the registry record. Such estimates should not be inferred from the participant count.
General: over-dispersion matters for some count outcomes when variability exceeds what a simple probability model expects; it can affect standard errors and uncertainty estimates. The primary measure is described as a count of participants, but no dispersion estimate or model is reported. There is no basis in the registry record to say that over-dispersion was assessed or that a particular count model was used.
For the safety results, denominators and event categories are necessary for context. The record gives 50 at risk for serious and other adverse events, 2 serious-event-affected participants, 50 participants affected in the other category, and 32 deaths. It does not report the collection window, grading system, handling of repeated events, or analysis approach. The listed serious-event terms each have a reported value of 2.0%. Their presentation does not establish whether participants could contribute to multiple terms or explain the relationship between those terms and the overall serious-event count.
The registry lists 3 outcome measures and 0 analyses. This is not enough to identify a statistical analysis plan, covariate adjustments, interim analyses, missing-data procedures, or an analysis population. Those details are not reported in the registry record. As a result, methodological explanations can clarify what the outcome labels and design imply in general, but cannot fill in unreported protocol choices.
10. Multiplicity, Missing Data and Analysis Populations
The registry names event-free survival as the primary outcome and lists overall survival and the number of participants with toxicity of a combination of low-dose IL-2 and GM-CSF as secondary outcomes. It reports 3 outcome measures and 0 analyses. The statistical plan, including any hierarchy among secondary outcomes or adjustment for multiple testing, is not reported in the registry record. It is therefore not possible to determine whether multiplicity was addressed or how multiple results were intended to be interpreted.
General: when several outcomes or analyses are examined, the chance of at least one apparently unusual result can increase. Prespecified outcome priorities and multiplicity procedures can help readers understand whether reported tests are confirmatory or exploratory. The registry record does not provide the information needed to assess those issues for this study. No p-values or analysis results for the named secondary outcomes are reported in the registry record.
Missing-data handling and analysis populations are also not described. The record does not state how many participants completed follow-up, whether all enrolled participants were included in the primary outcome, or how participants with unknown event status were treated. It does not report censoring rules, exclusions after enrollment, or a missing-data method. These details are important for interpreting a time-to-event endpoint and assessing whether the reported count represents the full enrolled group.
General: missing outcome data can affect both the estimated result and its uncertainty, especially when the likelihood of missingness is related to participants’ outcomes. A clear account of follow-up and analysis populations helps readers judge this risk. No such account is reported in the registry record, so the direction or magnitude of any missing-data effect cannot be determined.
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 |
|---|---|---|---|
| TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer | 2 | 50 | 4.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer | 32 of 50 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer | 50 of 50 |
Most frequent serious adverse event terms
| Event term | Organ system | TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer |
|---|---|---|
| Prolonged hospitalization for post-transplant complications | Infections and infestations | 1 of 50 |
| Pulmonary Emboli | Respiratory, thoracic and mediastinal disorders | 1 of 50 |
Most frequent non-serious adverse event terms
| Event term | Organ system | TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer |
|---|---|---|
| Myelosuppression | Blood and lymphatic system disorders | 50 of 50 |
| Infections | Infections and infestations | 16 of 50 |
| Hematologic | Blood and lymphatic system disorders | 6 of 28 |
| Infection | Infections and infestations | 3 of 28 |
Serious events with the highest arm-level rate: prolonged hospitalization for post-transplant complications (2.0%), pulmonary emboli (2.0%).
The serious adverse-event entry lists 50 participants at risk and 2 affected. It identifies “Prolonged hospitalization for post-transplant complications” and “Pulmonary Emboli” as the top serious-event terms, each with a reported value of 2.0%. The record also lists 50 participants at risk and 50 affected in the other adverse-event category, and reports 32 deaths. The registry does not provide a timeframe or a detailed explanation of how these categories relate to one another.
These figures should be read as reported counts and terms, not as evidence that a specific event was caused by treatment. The record does not report event onset, severity grading, recurrence, exposure duration, or adjudication procedures. It also does not explain whether one participant could appear under multiple terms or categories. The 32 deaths are not identified as treatment-related, and the record does not specify how they relate to event-free survival or overall survival. The secondary outcome naming toxicity does not itself provide a separate toxicity analysis. Without the missing context, the serious-event figures describe the registry entries but do not support a causal safety conclusion or a comparison with another treatment group.
12. What the Result Does, and Does Not, Mean
The result establishes that the registry reports 20 patients out of 50 as having event-free survival, with an 11 years timeframe. It also identifies the study as a phase II, single-group investigation of a treatment strategy combining chemotherapy, peripheral blood stem cell support and post-engraftment immunotherapy for the stated breast cancer population. These registry entries describe the study and its posted primary outcome.
The result does not establish how outcomes would compare with another regimen, supportive care, or no study treatment. There is no concurrent control group in the registry design. Nor does the count isolate the contribution of any single treatment component. The record does not report the event definition, time-to-event analysis, confidence interval, or individual follow-up information that would be needed to judge the precision and construction of an event-free survival estimate.
The posted result should not be read as proof that the treatment strategy caused any observed outcome. A single-group outcome can reflect treatment, disease characteristics, follow-up, and other influences, and the record does not provide a method to distinguish them. Likewise, the reported deaths and serious adverse events do not establish treatment causation. Their timing, attribution and relationship to the primary endpoint are not reported in the registry record.
General: statistical evidence is most informative when the design, outcome definition, analysis method and uncertainty are considered together. Here, the registry provides a count and a broad timeframe, but not those additional details. The defensible interpretation is consequently descriptive: the record states the number of participants reported as having event-free survival, without a comparative effect estimate or a reported measure of precision.
The secondary outcomes are named, but their results are not reported in the registry record. A reader should not infer unreported survival or toxicity findings from the outcome names or from the primary count. The available information is insufficient for a full assessment of efficacy, safety, or comparative clinical value.
13. Why This Trial Matters Statistically
This study illustrates why the endpoint label alone is not enough to interpret a clinical result. Event-free survival is a time-to-event concept, while the posted primary result is expressed as a participant count. Without a definition of the event, an account of follow-up and an analysis method, readers cannot tell how that count corresponds to event timing or how precisely an underlying time-to-event outcome is estimated.
The single-group design is also central to statistical interpretation. The record describes treatment outcomes in one study group but offers no like-for-like control comparison. A result in such a group can be useful as a description of what was recorded among participants, yet it cannot estimate a treatment contrast on its own. This distinction helps prevent a reported outcome from being mistaken for evidence that a regimen caused a particular result.
The safety entries demonstrate a related issue: event counts need clear definitions and context. Serious-event terms and a death count are reported, but the registry does not provide the timing, attribution, collection window or relationship among those entries. Such omissions limit causal interpretation, even when counts and denominators are available.
Finally, the record lists outcomes but no analyses. Planning illustrations based on an observed effect do not establish the trial’s original design rationale, and they are not observed power. Statistical reporting is most useful when it separates planning assumptions, observed summaries and comparative analyses. This record offers a focused example of why those distinctions matter when reading a completed clinical study.
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 trial-specific details needed to interpret the primary endpoint are not reported in the registry record. The event-free survival definition is absent, as are event dates, censoring rules, follow-up completeness and the method used to derive the reported count. The stated 11 years timeframe does not, by itself, establish how long each participant was followed or how event status was assessed.
The study has no comparator group and is listed as single-group, with allocation recorded as NA. Its posted result is therefore not a controlled treatment-effect estimate. Masking is recorded as none. The record does not describe any approach to reduce bias in outcome assessment or to address differences among participants.
The treatment details are incomplete in the registry record. The overall strategy and intervention names are listed, but the arm description directs readers to a detailed description that is not included in the posted record. The use of tamoxifen citrate and radiation therapy is not explained. The registry does not report a sample-size justification, interim analyses, a statistical analysis plan, or the method used for the primary analysis.
There is no reported confidence interval or comparative estimate for the primary endpoint. The secondary outcomes are named, but their results are not reported in the registry record. Follow-up losses, analysis populations and missing-data procedures are also not reported. The serious-event entries and death count lack timing and attribution details. These limitations constrain interpretation of the posted outcome and safety information; they do not determine what unreported analyses or protocol documents may have contained.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Combination Chemotherapy and Peripheral Blood Stem Cell Transplant Followed By Aldesleukin and Sargramostim in Treating Patients With Inflammatory Stage IIIB or Metastatic Stage IV Breast Cancer — NCT00003199.
- Registry record last updated 2017-07-12; status last verified 2017-06.
- Results first posted 2017-05-17.
- 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 record describes a completed phase II, single-group study sponsored by Fred Hutchinson Cancer Center, with enrollment of 50. It concerns chemotherapy with busulfan, melphalan and thiotepa, peripheral blood stem cell support, and post-engraftment immunotherapy with low-dose IL-2 and GM-CSF for inflammatory stage IIIB or responsive metastatic stage IV breast cancer. Allocation is recorded as NA and masking as none.
The primary outcome is event-free survival, reported as a count of participants over a timeframe of 11 years. The posted result states that 20 patients out of 50 have event-free survival. The registry lists overall survival and toxicity as secondary outcomes, 3 outcome measures and 0 analyses. Serious adverse-event entries include 2 affected participants among 50 at risk and two listed terms at 2.0% each; 32 deaths are also reported. Outcome definitions, analysis methods, missing-data procedures and causal attribution details are not reported in the registry record.