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 Bortezomib in Treating Patients With High-Risk Acute Myeloid Leukemia in Remission (NCT01465386), a na phase 2 study. The primary outcome is Progression Free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01465386 |
| Phase | Phase 2 |
| Condition | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Myeloblastic Leukemia Without Maturation (M1) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fred Hutchinson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The registry describes patients with high-risk acute myeloid leukemia in remission. The primary endpoint population is patients who began maintenance treatment with bortezomib after induction of remission. The listed conditions include several AML subtypes and secondary AML, but the registry record does not report further eligibility criteria or how patients were classified as high risk.
Intervention
The intervention was subcutaneous bortezomib. The treatment description specifies doses on days 1, 8, 15 and 22, with treatment repeated every 35 days for up to 6 courses if there was no disease progression or unacceptable toxicity. These are the registry’s stated treatment details, not evidence about how much treatment any individual received.
Comparator
There was no comparator group in the registry’s single-group design. Allocation is listed as not applicable, and the study was unmasked. This record therefore does not provide a randomized, like-for-like comparison with another treatment or with no maintenance treatment.
Primary question
The primary endpoint was progression-free survival, measured as the number of days from enrollment to recurrence of AML, defined by reappearance of blasts in the blood or marrow. The stated timeframe was up to 2 years. The registry record identifies the endpoint but does not report its median or another primary outcome estimate. The record also does not report an analysis that would establish how the observed course compares with an alternative.
3. Trial Design
This was a phase II, single-group study of bortezomib maintenance therapy for patients with high-risk acute myeloid leukemia in remission. The registry lists enrollment as 6 and the study status as terminated. The start is recorded as 2011-11, and primary completion as 2014-01-28. The reason for termination is not reported in the registry record.
The intervention description specifies subcutaneous bortezomib on days 1, 8, 15 and 22. Treatment was to repeat every 35 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. The registry does not report how many courses each participant received, whether every participant followed this schedule, or why treatment may have stopped for any individual.
The registry records no allocation and no masking. With a single group and no comparator, the study can describe outcomes among the participants represented in its record, but it cannot make a direct randomized comparison between bortezomib and another treatment. The study title and summary frame the intervention as maintenance after remission; the primary endpoint population is specifically described as patients who began maintenance treatment after induction of remission.
The broad condition list names AML with multilineage dysplasia following myelodysplastic syndrome, multiple adult AML subtypes, and secondary AML. The registry record does not give detailed inclusion or exclusion criteria, nor does it explain how the high-risk designation was determined. Its listed enrollment does not by itself establish how many participants contributed to an endpoint analysis; no analysis is reported in the registry record.
The tables and plots should be read as displays of the registry material, not as evidence of an unreported comparison. The registry lists one outcome measure and no analyses. The status, small enrollment, and absence of a reported primary estimate limit what can be concluded from this record about the study’s results.
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 | Treated Patients |
|---|---|
| STARTED | 6 |
| COMPLETED | 2 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Treated Patients |
|---|---|
| Lack of Efficacy | 3 |
| Adverse Event | 1 |
Analysis population for the primary outcome, as defined in the registry: Patient who began maintenance treatment with bortezomib after induction of remission
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 | Treatment (Enzyme Inhibitor Therapy) |
|---|---|---|
| Participants analysed | 6 | |
| Age, Continuous | years | 78 (61 to 87) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 5 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival (PFS), measured over Up to 2 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | days |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is labelled progression-free survival, with the registry defining it as the number of days from enrollment to recurrence of acute myeloid leukemia. Recurrence is determined by reappearance of blasts in the blood or marrow. The stated timeframe is up to 2 years, and the specified population is patients who began maintenance treatment with bortezomib after induction of remission.
In general, a time-to-event endpoint summarizes how long it takes for a defined event to occur. It can use information from participants whose event has not been observed by the end of follow-up, provided the analysis accounts for that follow-up appropriately. The registry record does not report the censoring rules or details of follow-up, so the handling of those observations cannot be assessed.
The registry identifies the endpoint summary as a median with a full range. In general, the median is the middle of the observed time distribution, while the full range describes its smallest and largest values. Neither the median nor the range is reported in the registry record. The record also does not state whether death without documented recurrence counted as an endpoint event; its written event definition specifies recurrence. This matters when interpreting the endpoint label: the posted definition, rather than assumptions about a standard endpoint convention, is the basis for describing what was measured.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a phase II study of maintenance bortezomib in patients with high-risk AML in remission, with enrollment listed as 6. It identifies recurrence-based progression-free survival as the primary endpoint and gives the endpoint’s time origin, event definition, and intended timeframe. However, it does not report a median, a range, a confidence interval, an event count, or a survival curve. The central primary outcome result therefore cannot be assessed from this record.
The record lists one outcome measure and zero analyses. This means the registry material does not present an analysis of the primary endpoint. It does not establish whether an analysis was conducted elsewhere or why no result appears in this record. The study status is terminated, but the reason for termination is not reported. The dates and status describe the record; they do not explain the decision to terminate or establish that all planned follow-up was completed.
The single-group design is important to interpretation. Even if an outcome estimate were reported, a result from this group alone would describe outcomes among the participants included in that analysis. It would not provide a randomized comparison with another treatment or a control group. Without a comparator, differences from an external expectation could not be attributed to the intervention on the basis of this study design alone.
The registry’s endpoint definition is recurrence based: reappearance of blasts in blood or marrow. Although the measure is named progression-free survival, the record does not state whether death without documented recurrence counted as an event. It also does not describe censoring, follow-up completeness, or the statistical method used to estimate the median. These omissions make it impossible to reconstruct how an endpoint estimate would have been produced.
Safety information is also limited. The record lists serious adverse events affecting 2 participants and other adverse events affecting 1 participant, each among 6 participants at risk. It separately reports 5 deaths. Those counts do not specify timing, cause, relationship to treatment, or whether an individual with an adverse event also appears in the death count. The listed serious-event terms each have a reported percentage, but the registry does not give the event narratives needed to interpret them clinically.
The companion sample-size section is a planning illustration rather than evidence of trial performance. Its assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects expressed as fractions of the observed effect. The trial itself is single-group, so such calculations should not be read as observed power or as proof of a treatment effect. Overall, the posted record is informative about design and the limited outcome and safety entries, but does not report an analyzable primary efficacy result.
9. Statistical Methods Explained
The registry gives a limited methods description. It identifies a phase II study, a single-group model, no allocation, and no masking. It also identifies a median time-to-event primary outcome. The registry record does not report the statistical analysis plan, methods for estimating the endpoint, or a primary endpoint analysis. The explanations below distinguish general statistical concepts from methods documented for this study.
What does a median time-to-event outcome mean?
In general, a time-to-event outcome records the time from a defined starting point until an event. Here, the registry specifies enrollment as the starting point and AML recurrence as the event, defined by the reappearance of blasts in blood or marrow. It labels the outcome progression-free survival and states a timeframe of up to 2 years. The registry does not report the median time or its full range, despite listing those as the intended summary.
In general, a median is a measure of the center of a distribution: half of the observations lie at or below it and half at or above it. Time-to-event data can be incomplete when follow-up ends before an event is observed. Statistical methods for such data can use the observed follow-up while accounting for observations without a recorded event. Which method was used, how censoring was defined, and how participants without a recorded recurrence were handled are not reported in the registry record.
What does the endpoint definition leave unspecified?
The event definition focuses on recurrence documented by blasts in blood or marrow. The registry does not state whether death without a prior documented recurrence was counted as an event, treated as a censored observation, or handled in another way. It also does not report whether recurrence was reviewed by a blinded or independent assessor. Since the study is listed as unmasked, no masking is recorded, but the record gives no further endpoint-adjudication details.
In general, the exact definition of an event and the rules for follow-up influence the meaning of a time-to-event estimate. A label alone is not enough to reconstruct an analysis. For this record, the written definition is the available description; additional operational rules are not reported.
How should the single-group design be read?
A single-group study reports on one cohort receiving the intervention. It does not create a concurrent comparison group through random allocation. General statistical methods can describe outcomes within a single cohort, but they cannot by themselves separate an intervention effect from other explanations for the observed outcomes. This record has no comparator, and the registry lists allocation as not applicable. There is consequently no like-for-like treatment contrast to interpret.
The registry lists enrollment as 6. That is a design and record-level enrollment figure, not an endpoint analysis denominator. The record does not say how many participants contributed to a time-to-event estimate, how many had recurrence, or how many had follow-up through the stated timeframe. Those details are not reported in the registry record.
What do the posted statistical displays represent?
The calculated tables and plots present the results available for this page; they should not be treated as a substitute for analysis details absent from the registry. The registry lists one outcome measure and no analyses. It does not provide an estimate for the primary endpoint in the record, so no statistical precision around that endpoint can be evaluated from the posted registry data.
In general, a confidence interval expresses the uncertainty around an estimate under a specified statistical model and procedure. Its width depends on the data and method; it is not a guarantee that a particular value is correct. No confidence interval for the primary endpoint is reported in the registry record, and no interval can be interpreted as a sponsor-reported result.
The sample-size section is explicitly a planning illustration. Its assumptions specify a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and a target effect set to a stated fraction of the observed effect. For time-to-event calculations, the stated quantity is the total number of events; for other calculations, it is participants per arm. These are hypothetical planning outputs, not participants or events observed in this single-group trial. The table should not be described as observed power. Because the study has no comparator, the equal-allocation, two-arm assumptions are distinct from its actual design.
Why might over-dispersion matter?
In general, over-dispersion occurs when observed variability exceeds what a statistical model assumes. If a model does not account for that extra variability, uncertainty estimates can be too small and statistical tests can be misleading. This is a general concept only: the registry record does not report an over-dispersion assessment or a model for this endpoint, and there is no primary analysis to evaluate on that basis.
More broadly, the record does not state a sample-size justification, interim analysis plan, model specification, multiplicity procedure, or missing-data strategy. Their absence from this registry record should not be turned into assumptions about what investigators planned or did. These gaps, together with the lack of a reported endpoint estimate, prevent a detailed audit of the statistical analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure, the recurrence-based progression-free survival endpoint, and zero analyses. It does not report a primary endpoint result or a statistical analysis plan. Although a single listed measure gives no evidence of multiple registered outcome analyses in this record, it does not establish that no other analyses or outcomes were considered. Any multiplicity procedure, hierarchy among outcomes, or adjustment for multiple statistical tests is not reported in the registry record.
Missing-data and follow-up handling are also not described. For a time-to-event outcome, participants may have follow-up that ends without a recorded recurrence; in general, analyses need rules for censoring and for determining which observations contribute information. The registry does not report censoring rules, follow-up completeness, the number of participants with endpoint data, or how missing assessments were handled. No imputation method is reported.
The endpoint population is described as patients who began maintenance treatment with bortezomib after induction of remission. The registry does not specify an intention-to-treat, per-protocol, or other analysis population, nor does it provide a participant flow that would clarify inclusion in an endpoint analysis. Enrollment is listed as 6, but that number should not be assumed to be the denominator for every outcome or analysis. The registry record does not report which participants were included in any primary endpoint analysis.
The record also does not report interim analyses or rules for stopping the study. The terminated status alone does not explain the reason for termination or establish whether it related to efficacy, safety, recruitment, or another consideration. These matters remain unreported in the registry record.
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 |
|---|---|---|---|
| Treatment (Enzyme Inhibitor Therapy) | 2 | 6 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment (Enzyme Inhibitor Therapy) | 5 of 6 |
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 |
|---|---|
| Treatment (Enzyme Inhibitor Therapy) | 1 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Enzyme Inhibitor Therapy) |
|---|---|---|
| Altered Mental Status | Nervous system disorders | 1 of 6 |
| Cholechystitis | Gastrointestinal disorders | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Enzyme Inhibitor Therapy) |
|---|---|---|
| Peripheral neuropathy | Nervous system disorders | 1 of 6 |
Serious events with the highest arm-level rate: altered mental status (16.7%), cholechystitis (16.7%).
The registry lists 6 participants at risk for serious adverse events and 2 affected. For other adverse events, it lists 6 at risk and 1 affected. The serious adverse event terms displayed are Altered Mental Status and Cholechystitis, each reported as 16.7%. These are the posted counts and percentages; the record does not provide event narratives or further detail about severity, timing, duration, or management.
The record separately lists 5 deaths. It does not report when these deaths occurred, their causes, whether they were considered related to bortezomib, or how they relate to the adverse-event entries. It is not possible to infer those details from the counts. The registry also does not clarify whether the same participant could contribute to more than one listed event term.
These entries describe what is posted, not a comparative safety rate: the study has no control group. The small enrollment and absence of detailed event information limit interpretation. The counts alone do not establish whether an event was caused by treatment or how safety compares with another option.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study of bortezomib maintenance in high-risk AML remission was registered, with enrollment listed as 6 and status recorded as terminated. It specifies a treatment schedule and defines a recurrence-based primary endpoint. It also posts limited adverse-event counts and a death count. These are useful descriptions of the record, but they do not amount to a reported estimate of the primary outcome.
The registry does not report the median progression-free survival, its full range, a confidence interval, or an endpoint analysis. It therefore does not show the estimated time to recurrence for the endpoint population or the precision of such an estimate. Nor does it report a randomized comparison. The record cannot establish from these data whether bortezomib changed outcomes relative to another treatment or no maintenance therapy.
In general, a treatment effect requires an appropriate comparison and a clearly reported analysis. A single-group outcome can describe what was observed in that cohort, but other factors may also explain the outcome. Here, even that descriptive primary outcome is not numerically reported. No conclusion about benefit or lack of benefit should be drawn from the absence of a registry result.
The safety entries likewise do not establish cause or comparative risk. They give counts of affected participants and listed deaths, without the detail needed to determine timing, attribution, overlap, or comparison with another group. The terminated status does not disclose why the study ended. It should not be interpreted as evidence of a particular efficacy or safety finding.
The sample-size calculations elsewhere on the page are planning illustrations based on stated assumptions, not observed power or additional trial results. Taken together, the record documents the study’s broad design and some safety reporting, while leaving its primary outcome estimate, analysis methods, and termination rationale unreported.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its registry record makes the distinction between a study design and an interpretable treatment comparison especially clear. The study is listed as single group, with allocation not applicable and no masking. Without a comparator, an outcome estimate would describe the cohort rather than provide a randomized contrast. That distinction matters when readers see an intervention-focused title or summary.
It also illustrates why an endpoint label is not a complete statistical specification. The record names progression-free survival but defines the event as AML recurrence detected by the reappearance of blasts in blood or marrow. It does not say whether death without documented recurrence counted as an event, how incomplete follow-up was handled, or what estimation method was used. Those details affect what a time-to-event result would mean.
The registry lists the endpoint as a median with a full range, yet does not report either value or an analysis. A planned summary and an observed estimate are not interchangeable. The absence of a reported estimate also means readers cannot assess its precision or compare it with any external benchmark using this record alone.
Finally, the record demonstrates why enrollment, safety counts, and endpoint denominators should not be treated as interchangeable. Enrollment is listed as 6, while the endpoint analysis population and missing follow-up details are not reported. The posted safety counts describe specific adverse-event categories and deaths, but do not provide attribution or a control-group comparison. Statistical interpretation depends not just on the presence of numbers, but on knowing how they were generated and what comparison they support.
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 most immediate limitation is that the registry record does not report a primary endpoint result. Although it identifies median progression-free survival and a full-range summary, no median or range is posted. There is also no reported confidence interval, event count, survival curve, or primary analysis. As a result, the primary outcome cannot be evaluated for magnitude or precision from this record.
The single-group design has no comparator, so it cannot provide a randomized estimate of a treatment contrast. The registry lists no allocation and no masking. The record does not report why the study was terminated, and termination alone does not identify a cause or indicate what conclusions were reached.
The enrollment is listed as 6, but the number included in an endpoint analysis is not reported. The registry does not give participant flow, follow-up completeness, censoring rules, a missing-data approach, or the details needed to determine how deaths without documented recurrence were treated. The endpoint’s written event definition and label may therefore leave important operational questions unresolved.
Safety information is limited to affected-participant counts, two serious-event terms with reported percentages, and a count of deaths. Timing, cause, treatment relationship, overlap among events, and comparative safety are not reported. The registry also lacks detailed eligibility criteria and an explanation of the high-risk classification. These omissions limit both statistical assessment and interpretation of the population represented.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bortezomib in Treating Patients With High-Risk Acute Myeloid Leukemia in Remission — NCT01465386.
- Registry record last updated 2017-04-04; status last verified 2017-02.
- Results first posted 2017-04-04.
- 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 is a phase II, single-group registry record for subcutaneous bortezomib maintenance therapy in high-risk AML in remission. Enrollment is listed as 6; the study status is terminated. The treatment description specifies administration on days 1, 8, 15 and 22, repeated every 35 days for up to 6 courses if there was no progression or unacceptable toxicity.
The primary endpoint is median progression-free survival, defined as days from enrollment to AML recurrence based on reappearance of blasts in blood or marrow, with a timeframe of up to 2 years. The registry lists one outcome measure and no analyses. It does not report a primary endpoint estimate, analysis method, or reason for termination.
The record lists serious adverse events affecting 2 participants, other adverse events affecting 1 participant, and 5 deaths. It does not report timing or treatment attribution. With no comparator and no primary outcome result, the registry supports a description of the study record but not a comparative assessment of treatment outcomes.