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 Rituximab and Combination Chemotherapy in Treating Older Patients With Diffuse Large B-Cell Lymphoma (NCT00101010), a na phase 2 study. The primary outcome is Disease Response (Complete, Complete Unconfirmed, and Partial Responses) After 4 Courses.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00101010 |
| Phase | Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 80 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Older patients with untreated aggressive B-cell non-Hodgkin lymphoma were the intended population. The trial title specifies patients older than 60 years, and the study summary focuses on older patients with diffuse large B-cell lymphoma. The registry lists lymphoma as a condition. The number enrolled was 80.
Intervention
The study evaluated rituximab with combination chemotherapy that included cyclophosphamide, pegylated liposomal doxorubicin, vincristine and prednisone. Filgrastim or pegfilgrastim was also listed among the interventions. The registry describes a single treatment group.
Comparator
There was no control group or randomized comparison. The record identifies the design as single-group and lists allocation as not applicable. The study therefore does not directly compare this regimen with another treatment or with a control condition.
Primary question
The primary question was how many participants had a complete response, an unconfirmed complete response or a partial response after four courses. Response was assessed after 12 weeks, corresponding to four cycles of 21 days, using International Workshop Criteria and CT and FDG-PET scans.
3. Trial Design
This completed phase II study enrolled 80 participants and evaluated one treatment group. The registry lists no masking and no randomized allocation. The sponsor was M.D. Anderson Cancer Center. The study start is recorded as 2005-09 and primary completion as 2014-09. These dates describe the registry record; they do not establish how long each participant was followed.
The treatment combined rituximab with chemotherapy. The listed drugs were cyclophosphamide, pegylated liposomal doxorubicin hydrochloride, vincristine sulfate and prednisone. Filgrastim and pegfilgrastim were also listed as biological interventions. The arm description gives some administration details, including treatment on day 1 and prednisone on days 1 through 5, but the description ends while specifying how long filgrastim continued. Further schedule details are not reported in the registry record.
A single-group design can describe outcomes among participants receiving the regimen, but it does not provide a concurrent control group for estimating a treatment contrast. Since masking is listed as none, the study was not masked according to the record. The allocation field is not applicable because the record does not describe assignment between groups.
The registry lists survival rate and disease-free survival as secondary outcomes, alongside four outcome measures overall. It lists no analyses. The record does not report the planned statistical analysis, how participants were selected for analysis, or the number who completed endpoint assessment. Those omissions limit interpretation of what the enrollment figure represents for the primary endpoint.
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 | Rituximab - Combination Chemotherapy |
|---|---|
| STARTED | 80 |
| COMPLETED | 79 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Rituximab - Combination Chemotherapy |
|---|---|
| Physician Decision | 1 |
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 | Rituximab - Combination Chemotherapy |
|---|---|---|
| Participants analysed | 80 | |
| Age, Customized: 60 to 69 | participants | 40 |
| Age, Customized: 70 to 79 | participants | 27 |
| Age, Customized: 80 to 89 | participants | 12 |
| Age, Customized: 90 to 99 | participants | 1 |
| Sex: Female, Male: Female | Participants | 42 |
| Sex: Female, Male: Male | Participants | 38 |
| Region of Enrollment: United States | participants | 80 |
6. Primary Endpoint
The registry lists the primary outcome as Disease Response (Complete, Complete Unconfirmed, and Partial Responses) After 4 Courses, measured over Evaluation after 12 weeks (4 cycles of 21 days).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants whose disease response after four courses was classified as complete response, unconfirmed complete response or partial response. Assessment was scheduled after 12 weeks, corresponding to four cycles of 21 days. CT and FDG-PET scans were used under International Workshop Criteria for Tumor Response Criteria.
The definitions distinguish the response categories. Complete response required disappearance of target and non-target lesions in the liver and spleen and regression of lymph node masses to normal size. Partial response required at least 50% reduction in the sum of the products of diameters for measured lymph nodes, splenic lesions and liver lesions, assessed separately against baseline. An unconfirmed complete response did not meet the complete-response definition because of a residual nodal mass or an indeterminate bone marrow.
Combining these categories into a participant count gives a categorical summary of tumor response at a specified assessment time. It does not, by itself, describe how long a response lasted or how participants fared later. The registry record defines this endpoint but does not report the number of participants meeting it, the number assessed, or a response estimate. As a result, the observed primary endpoint cannot be interpreted from the record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a clinically defined response endpoint and a single treatment group, but it does not give the primary response count or the number of participants assessed. Consequently, the posted record does not establish what proportion of participants had complete, unconfirmed complete or partial response after four courses. No response estimate or measure of its precision can be interpreted from the information reported.
The enrollment figure is 80, but enrollment alone is not the endpoint denominator. The record does not state how many participants started treatment, reached the assessment after 12 weeks, had evaluable scans or were included in the response analysis. Without those counts, it is not possible to distinguish an outcome count among all enrolled participants from a count among participants with an assessment. Those are different descriptions of the data and can lead to different interpretations.
The study is single-group, with no randomized allocation or control group. Any observed response count, if available elsewhere, would describe participants in this group under the assessment process; it would not by itself estimate how outcomes would differ under another treatment. Differences between this group and a hypothetical comparison could reflect factors other than treatment, and the registry record does not provide a comparison that addresses them.
The registry lists four outcome measures and zero analyses. It identifies survival rate and disease-free survival as secondary outcomes, but it does not report their results or follow-up details. The primary outcome definition is specific about categories and timing, while the numerical result is absent. The most defensible reading is therefore descriptive: the study set out to assess response at 12 weeks, but the registry record does not report the resulting response data.
Safety information is also limited. The record lists one participant with a serious adverse event among 80 at risk, reported as 1.2%, and zero with other adverse events among 80 at risk. These counts describe what the registry reports for this group; they do not support comparison with another regimen. The serious-event term listed is leptomeningeal. The record does not provide enough detail to infer event timing, attribution, or completeness of event ascertainment.
9. Statistical Methods Explained
What kind of endpoint is this?
The primary endpoint is a count of participants who meet a composite response definition. The count combines complete response, unconfirmed complete response and partial response at the scheduled assessment. It is a binary classification at the participant level: each assessed participant either meets one of the included response categories or does not. The registry labels the measure as a count of participants, not as a time-to-event outcome or a continuous measurement.
General statistical explanation: Counts are often interpreted alongside a denominator, such as the number of participants assessed. A proportion can then describe the frequency of the event in that group. A confidence interval expresses uncertainty about an estimated population value under a statistical model; it does not show the range of individual participant outcomes. The registry record does not report the primary count, its denominator, a proportion or a confidence interval, so none can be interpreted for this study.
Why define response categories carefully?
The response categories rely on imaging and specified criteria. Complete response requires disappearance of the stated target and non-target lesions and regression of lymph node masses to normal size. Partial response uses a threshold based on change in the sum of products of diameters for measured lesions. Unconfirmed complete response accommodates a residual nodal mass or indeterminate bone marrow that prevents classification as complete response. These operational definitions aim to make the endpoint more reproducible than an informal judgment of improvement.
The endpoint combines three categories rather than reporting each separately in its title. A combined count can answer whether a participant reached any of the included response states, but it cannot show the separate frequency of each category. The registry does not report category-specific counts, so the distribution across complete, unconfirmed complete and partial responses is not known from this record.
What does a single-group design allow?
The study has one treatment group and no randomized allocation. Its design can describe outcomes observed in that group, provided the outcome and analysis data are reported. It cannot directly estimate a contrast between treatment groups because the record identifies no comparator arm. General statistical reasoning also cautions that a before-and-after or single-group result is not equivalent to a randomized treatment contrast: the design does not separate treatment effects from other influences on outcomes.
The lack of masking is part of the recorded design. In general, masking can reduce the possibility that knowledge of treatment assignment influences some assessments or decisions. Imaging criteria provide a defined basis for response classification, but the registry does not report who assessed the scans, whether assessment was independently reviewed, or any procedures to limit assessment bias. Those details are not reported in the registry record.
How should precision and planning be understood?
General statistical explanation: An estimate based on a finite group has sampling uncertainty. A confidence interval is one way to communicate that uncertainty, while a point estimate alone gives only a central summary. Precision depends on the information in the data and on the analysis method. Since the endpoint result and its denominator are not reported, the precision of the response estimate cannot be assessed.
The registry describes sample-size planning illustrations as based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. It also states that the illustrations are not the trial's own design. This was a single-group study, so the stated equal-allocation assumption is not a description of its observed design. No numerical planning illustration or observed effect is reported in the record. Such illustrations, when available, are planning aids rather than observed power or evidence that a study detected an effect.
General statistical explanation: A normal approximation is a method for approximating the distribution of a statistic. Whether it gives a useful approximation depends on the data and the setting. Over-dispersion is a general concern when observed variability exceeds what a simple model assumes; accounting for it can affect uncertainty estimates. The registry does not report the method used to analyze this response endpoint, whether an approximation was used for the outcome analysis, or whether over-dispersion was considered. The planning assumptions should not be mistaken for the analysis actually used.
What analyses are documented?
The registry lists zero analyses. That means the record does not describe an analysis result for the primary endpoint. It also does not report a statistical model, hypothesis test, confidence interval method, covariate adjustment, or analysis population. The primary assessment time and response definitions are stated, but an endpoint definition is not an analysis plan. Any explanation of these methods beyond what the record states would be general rather than a description of procedures known to have been used in this trial.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four outcome measures, including disease response as the primary outcome and survival rate and disease-free survival as secondary outcomes. It lists zero analyses. The record does not explain whether additional outcomes were formally tested, how any multiplicity was handled, or whether the outcomes were arranged in a testing hierarchy. A multiplicity procedure is therefore not reported in the registry record.
General statistical explanation: When several outcomes or comparisons are tested, the chance of at least one apparently notable result can increase if each is interpreted separately. Statistical plans sometimes address this through adjustments or a prespecified order of testing. The registry does not say whether such procedures were planned or used, so no conclusion about multiplicity control is warranted.
Missing-data procedures and analysis populations are also not reported in the registry record. The record does not state how participants without an assessment after 12 weeks were handled, whether scans could be unevaluable, or whether participants who stopped treatment remained in the primary analysis. The enrollment figure of 80 does not answer how many participants contributed to the response endpoint.
General statistical explanation: Missing outcomes can affect interpretation when the participants with observed results differ from those without them. An analysis may define who is included and how missing outcomes are handled, but those choices need to be reported to assess their implications. No such approach is described. The registry also does not report interim analyses, stopping rules or a detailed statistical analysis plan. Their status is not reported in the registry record and should not be inferred from the phase or design.
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 |
|---|---|---|---|
| Rituximab - Combination Chemotherapy | 1 | 80 | 1.2% |
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 |
|---|---|
| Rituximab - Combination Chemotherapy | 0 of 80 |
Most frequent serious adverse event terms
| Event term | Organ system | Rituximab - Combination Chemotherapy |
|---|---|---|
| LEPTOMENINGEAL | General disorders | 1 of 80 |
Serious events with the highest arm-level rate: leptomeningeal (1.2%).
The registry lists one participant with a serious adverse event among 80 at risk, reported as 1.2%. It lists zero participants with other adverse events among 80 at risk. These are counts for the single treatment group; they are not a comparison between treatments. The record identifies leptomeningeal as the top serious adverse-event term and reports it as 1.2%.
These figures should be read as registry-reported event summaries, not as proof that the treatment caused or did not cause an event. The record does not report event timing, duration, severity details beyond the serious classification, attribution, or the methods used to collect events. It also does not give a narrative account of the serious event. The listed term alone cannot establish the clinical course or its relationship to treatment.
The at-risk count is 80 for both reported categories, but the record does not explain the observation period or whether all participants had the same duration of safety follow-up. A zero count for other adverse events means none are listed in that summary; it should not be interpreted as evidence that no events occurred outside the registry's reported data. Safety estimates from a single group also cannot establish comparative risk.
12. What the Result Does, and Does Not, Mean
The record identifies a phase II study of rituximab with combination chemotherapy in older patients with untreated aggressive B-cell lymphoma. It specifies a primary response assessment after four courses, at 12 weeks, and defines the response categories using International Workshop Criteria and imaging. Those details make clear what the investigators intended to measure and when they intended to measure it.
However, the registry record does not report the primary response count, the number assessed or an effect estimate. It therefore does not show how many participants met the response definition or how precisely that result was estimated. The stated enrollment of 80 cannot substitute for the missing outcome data. Without reported results, the record cannot establish the observed response frequency.
Even if a response count were available, a single-group study would describe outcomes in the treated group rather than a randomized comparison. It would not establish the difference between this regimen and another treatment. The design does not provide a concurrent control group, and the record contains no comparative analysis.
The response endpoint concerns disease status at a defined assessment. It is not the same as survival or duration of response. Survival rate and disease-free survival are listed as secondary outcomes, but their results and follow-up details are not reported. The registry's safety summary lists one serious adverse event and no other adverse events in the relevant count summaries, but sparse counts without further event details cannot characterize the full safety experience or comparative safety.
Accordingly, the record supports an understanding of the study question, treatment group and endpoint definition, but not a conclusion about the observed efficacy or comparative effects of treatment. It also does not provide enough detail to assess the analysis methods, missing outcomes or follow-up. These distinctions keep the interpretation aligned with what the registry actually reports.
13. Why This Trial Matters Statistically
This study illustrates why an endpoint definition and an endpoint result are separate pieces of evidence. The registry carefully specifies the response categories, imaging approach and assessment time, yet does not report the response count or denominator. Without those data, a reader can understand the intended measure but cannot estimate its observed frequency or uncertainty.
It also illustrates the limits of interpreting a single treatment group. A response count in one group can be informative descriptively, but it does not provide the contrast needed to estimate how outcomes differ from a comparator. The absence of randomized allocation and a control group matters for the question that can be answered, regardless of how precisely a single-group count might be reported.
Statistical interpretation depends not only on the endpoint but also on who was analyzed, how missing assessments were handled and how uncertainty was quantified. Those analysis details are not reported in the registry record. The enrollment count alone does not establish the response denominator, and the stated planning assumptions do not document the trial's own design or actual analysis.
The safety summaries offer another useful distinction: a count and percentage describe what is recorded, while causal attribution and comparative risk require additional evidence. The single-group safety counts do not provide a treatment comparison, and the record lacks event-level context. Together, these features make the study a clear example of why registry interpretation should distinguish study intent, observed data and the strength of the inference that the design permits.
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 results is that the registry record defines the primary response endpoint but does not report its observed count, denominator or estimate. The same record lists zero analyses. It is therefore not possible to assess the magnitude or precision of the primary result from the information reported.
The single-group design provides no randomized comparator. The record lists allocation as not applicable and masking as none. This limits causal and comparative interpretation: an outcome in the treatment group cannot, on its own, show how outcomes would have differed under another treatment. The registry does not describe any external comparison.
The record reports enrollment of 80 but does not state the number assessed after four courses, the number with evaluable scans, or how missing assessments were handled. It also does not report analysis populations, a statistical analysis plan, multiplicity procedures, interim analyses or stopping rules. These details are important for understanding how the endpoint was analyzed and how much confidence to place in any result, but their status is not reported in the registry record.
The intervention description lists several drugs and supportive biological agents, but the arm description ends while describing the duration of filgrastim. Full treatment-duration details are not reported in the registry record. The listed secondary outcomes lack reported results and follow-up details. Safety reporting consists of brief count summaries without event timing, attribution or fuller clinical descriptions. These omissions constrain interpretation beyond the primary endpoint as well.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Rituximab and Combination Chemotherapy in Treating Older Patients With Diffuse Large B-Cell Lymphoma — NCT00101010.
- Registry record last updated 2020-10-19; status last verified 2020-09.
- Results first posted 2020-10-19.
- 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 completed phase II study evaluated rituximab with combination chemotherapy in one group of older patients with untreated aggressive B-cell lymphoma. The registry reports enrollment of 80, no masking and no randomized allocation. The primary outcome was a count of complete, unconfirmed complete and partial responses after four courses, assessed at 12 weeks with International Workshop Criteria and CT and FDG-PET scans.
The registry lists survival rate and disease-free survival as secondary outcomes, four outcome measures overall and zero analyses. It does not report the primary response count, its denominator or an estimate of uncertainty. Safety summaries list one serious adverse event among 80 at risk, reported as 1.2%, and zero other adverse events among 80 at risk. The record does not provide a comparative treatment result or detailed analysis methods.