Rituximab, Combination Chemotherapy, Filgrastim (G-CSF), and Plerixafor in Treating Patients With Non-Hodgkin Lymphoma Undergoing Mobilization of Autologous Peripheral Blood Stem Cells: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Rituximab, Combination Chemotherapy, Filgrastim (G-CSF), and Plerixafor in Treating Patients With Non-Hodgkin Lymphoma Undergoing Mobilization of Autologous Peripheral Blood Stem Cells (NCT01097057), a na phase 2 study. The primary outcome is Number of Patients to Mobilize ≥5 x 10^6 CD34 Cells/kg Autologous PBSC (Efficacy).

2. Clinical Question

3. Trial Design

This is a completed phase II trial, NCT01097057, sponsored by Fred Hutchinson Cancer Center. The registry lists an enrollment of 20 and a start date of 2010-11-09, with primary completion in 2013-09. Its stated condition is non-Hodgkin lymphoma. The official title focuses on CD20+ lymphoma patients undergoing mobilization of autologous peripheral blood stem cells.

The design is single-group, with allocation listed as not applicable and masking listed as none. Participants received a treatment combination that included rituximab, etoposide, carboplatin, ifosfamide, G-CSF, and plerixafor; leukapheresis is also listed as an intervention. The treatment description indicates that chemotherapy and mobilizing agents were used in preparation for stem-cell collection. There is no separate control arm in the registry record.

The purpose described in the record is to assess mobilization and collection of autologous stem cells. The primary endpoint counts patients who reached the stated CD34-cell collection threshold. The record lists four outcome measures, but the available information does not include their results. It also lists no analysis records. The status is completed, but completion status alone does not establish what the primary outcome was or how precisely it was estimated.

The registry record does not report a statistical analysis plan, details of eligibility beyond the named population, or a sample-size justification. It does not describe randomization, and the design should not be interpreted as a controlled comparison. A single-group study can describe outcomes under the treatment approach used, but without a concurrent comparator it cannot separate the effects of the regimen from other influences on the observed outcome.

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

Reasons for not completing this period:

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Patients to Mobilize ≥5 x 10^6 CD34 Cells/kg Autologous PBSC (Efficacy), measured over One Month.

The primary endpoint is the number of patients who achieved collection of at least 5 x 10^6 CD34 cells/kg autologous peripheral blood stem cells by apheresis. The registry gives a timeframe of one month. This is a binary outcome at the participant level: each patient either meets the stated collection threshold or does not. The reported endpoint is the count of patients meeting it, rather than a measurement of the amount collected for every patient.

A threshold endpoint can make a collection goal explicit and clinically interpretable in the context of the study’s stated purpose: mobilizing stem cells for a future autologous transplant. A count can communicate how many participants reached that goal in the enrolled group. For a fuller statistical interpretation, it would also be useful to know the outcome denominator and an uncertainty interval, because the count alone does not show how precisely the underlying success proportion is estimated.

The registry record gives the endpoint definition and timeframe, but does not report the number who met the threshold. It also does not report an uncertainty interval or a detailed account of how participants without an assessable collection were handled. Consequently, the endpoint’s observed result and its precision are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a single-group design and a binary primary endpoint. These features determine what kinds of statistical summaries are most directly relevant. The endpoint is whether each participant achieved the specified CD34-cell collection threshold by apheresis within one month. A basic descriptive summary would report the number meeting the threshold and the number assessed. Neither the primary outcome count nor its assessment denominator is reported in the registry record.

What does a binary outcome count describe?

A binary outcome classifies each assessed participant into one of two categories: meeting the endpoint or not meeting it. Reporting the count in each category makes the observed data visible. A proportion can also describe the fraction meeting the endpoint among those assessed, but its interpretation depends on a clearly stated denominator. The registry record supplies the enrollment figure but does not report the primary outcome denominator or the number meeting the threshold. No outcome proportion should therefore be inferred from the enrollment information alone.

When a binary outcome is reported, an uncertainty interval can help show how much imprecision remains around the estimated proportion. In general, an interval is wider when information is limited and narrower when information is more precise, though the particular method matters. It is not a guarantee that the true value lies within the interval, and it does not correct for selection, missing outcomes, or the absence of a comparator. The registry record does not report an interval or specify an interval method for this endpoint.

What can a single-group design estimate?

A single-group study can describe outcomes observed among participants who received the treatment regimen. It cannot, without a comparison group, directly estimate what the outcome would have been under a different regimen. Differences between patients, care settings, and other factors may influence outcomes, so an observed result cannot be attributed to the treatment alone on the basis of this design. Here, the registry lists no control group and no randomized allocation.

The allocation field is not applicable, and masking is listed as none. These design descriptions should not be mistaken for evidence that a particular analysis was performed. The statistical analysis plan is not reported in the registry record. There is also no reported adjustment strategy, model, or method for estimating uncertainty. These details cannot be reconstructed from the endpoint definition or trial status.

What do the sample-size assumptions mean?

The planning illustration is described as based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. These are planning assumptions for an illustration, not evidence about the trial’s design or an observed statistical result. In particular, the trial itself is listed as single-group, not as an equal-allocation, two-arm comparison.

Sample-size illustrations are hypothetical calculations about the amount of information that might be needed under their stated assumptions. They are not observed power, and they do not show that a study had a particular probability of detecting an effect after the data were collected. The registry record does not report a sample-size justification for the trial. Since the observed primary endpoint result is not reported, the observed effect used as the basis for the illustration is also not available in the registry record. No sample-size illustration can fill that outcome-reporting gap.

How should safety counts be understood?

For a binary safety summary, a count of affected participants describes how many people in the listed group had the relevant classification. A percentage can offer a scale relative to the group at risk, but it does not show event timing, duration, severity beyond the classification, or whether events were judged related to treatment. Serious adverse events and other adverse events are distinct reporting categories in the registry data. Their participant counts should not be added together, since the record does not establish whether participants could appear in both categories.

More broadly, statistical interpretation requires attention to the population analyzed, outcome ascertainment, missing observations, and multiplicity. The population field for the primary endpoint is blank, and analysis populations are not described. The registry record does not report how missing outcome data were treated, whether there were interim analyses, or whether adjustments were made for multiple outcomes or analyses. These omissions limit the ability to assess how the reported or unreported results were generated.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Treatment (Rituximab, Etoposide, Carboplatin, Ifos")
n      <- c(17)
events <- c(17)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Treatment (Rituximab, Etoposide, Carboplatin, Ifos  17  17  100.0  80.5  100.0  81.6  100.0  81.5  99.9

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists four outcome measures, but the available information does not report their results or describe how the outcomes were prioritized in the analysis. The primary endpoint is identified, but no statistical analysis plan is reported. It is therefore not possible to determine whether any multiplicity adjustment was planned or used, or how conclusions across outcomes were intended to be interpreted. The record lists no analysis records.

Multiplicity arises when several outcomes, comparisons, or analyses are considered: the chance of obtaining a nominally notable result can increase when many opportunities for testing are available. General statistical approaches can address this, but the appropriate method depends on the prespecified analysis strategy. The registry record does not report such a strategy, so no particular adjustment should be assumed.

The primary endpoint population field is blank. The registry record does not state how many participants had an assessable primary outcome, how missing or unevaluable collections were handled, or which analysis population was used. Enrollment is listed as 20, but that figure should not be treated as the denominator for an outcome that is not reported. The reason for any missing data, if present, is also not reported in the registry record.

Likewise, the record does not report interim analyses, stopping rules, or procedures for handling protocol deviations. These are not details that can be inferred from the completed status. Without a statistical plan and a clear account of the analysis population and missingness, the robustness of any endpoint estimate cannot be assessed from the registry record.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: febrile neutropenia (5.0%), atrial fibrillation (5.0%).

The registry lists 20 participants at risk for serious adverse events and 2 affected, and 20 at risk for other adverse events and 3 affected. It separately lists 5 deaths. These are registry counts, not comparative estimates, because the trial has no separate control group. The record does not state whether the deaths were attributed to treatment or how they relate to the serious- and other-adverse-event categories; the categories should not be added or treated as mutually exclusive.

The listed serious-event terms are Febrile Neutropenia at 5.0% and Atrial Fibrillation at 5.0%. The registry record does not provide further detail in the information about event timing, duration, recurrence, severity beyond the serious classification, or attribution. The term percentages should be read as the reported figures, not as evidence of causation.

Safety counts are most informative when accompanied by definitions, follow-up duration, participant-level denominators, and a comparator. The record provides a timeframe of one month for the primary efficacy endpoint, but does not establish that this is the safety follow-up period. The timeframe for the adverse-event figures is not reported in the registry record.

13. What the Result Does, and Does Not, Mean

The registry record documents a phase II, single-group study of a combination intended to mobilize autologous peripheral blood stem cells in patients with non-Hodgkin lymphoma. It defines a primary endpoint based on achieving a specified CD34-cell collection threshold by apheresis within one month. It also records enrollment, treatment components, and selected safety counts.

The record does not report the number of participants who achieved the primary endpoint. It therefore does not establish the observed collection rate or the precision of that result. The endpoint definition can clarify what the study aimed to measure, but it cannot substitute for the outcome itself. Nor does the completed status supply an unreported result.

Because this is a single-group study without a comparator, the design cannot show how outcomes compare with another regimen. Even if an outcome count were available, an observed result would describe the treated group in this study rather than establish a causal advantage over an alternative. Factors other than the regimen may contribute to outcomes, and the registry record does not report a comparative analysis.

The safety entries should likewise be read as descriptive records, not as proof that the treatment caused the listed events or deaths. No control group is available for comparison, and further details on attribution and follow-up are not reported in the registry record.

Overall, the posted information characterizes the study question, treatment approach, and endpoint definition, but leaves the primary result and important analysis details unavailable. It does not support a conclusion about the frequency of meeting the collection threshold, the uncertainty around that frequency, or comparative treatment effects.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of how a clearly defined binary collection endpoint differs from a treatment comparison. The question is whether participants reached a specified stem-cell collection threshold. A count of participants meeting that threshold could describe the observed experience in the study group, while an outcome denominator and uncertainty interval would be needed to understand the estimate’s scale and precision.

The single-group design sets a firm boundary on interpretation. It can characterize what was observed among treated participants, but it does not create a counterfactual comparison with another approach. The registry identifies no control group and no randomized allocation. That limitation is not resolved by the phase II designation or by the trial’s completed status.

The record also illustrates the distinction between a registered endpoint and a reported result. The endpoint definition and one-month timeframe are available, but the number achieving the threshold is not reported. Without that result, statistical precision cannot be evaluated. The record’s safety counts add descriptive information, but do not provide comparative evidence or establish causal attribution.

Finally, planning calculations should be kept separate from trial findings. The stated illustration assumptions concern a hypothetical two-arm calculation, whereas the registered trial is single-group. Such calculations are not observed power and do not replace a reported primary outcome. This distinction helps prevent design illustrations from being misread as evidence about what happened in the study.

15. 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 for interpreting the trial is that the registry record does not report the primary endpoint count. The number meeting the collection threshold, the number assessed, and an uncertainty interval are not reported. Enrollment is listed as 20, but it cannot serve as a substitute for the missing outcome denominator.

The single-group design has no concurrent comparator, and the record identifies no randomized allocation. As a result, the study cannot estimate a treatment contrast against another regimen. Masking is listed as none. The available record does not provide enough information to assess the potential influence of outcome assessment procedures.

The registry record does not report the statistical analysis plan, the analysis population, or the handling of missing or unevaluable outcomes. The primary endpoint population field is blank. It does not report whether any multiplicity procedures were used for the four listed outcome measures, nor does it provide analysis records. The rationale for enrollment and a trial-specific sample-size justification are not reported.

The description of the treatment schedule is incomplete in the available record, ending before all stated timing details are given. Safety information includes participant counts and selected serious-event terms, but the safety follow-up period and attribution details are not reported. The record also does not clarify how deaths overlap with adverse-event categories. These limitations restrict interpretation to the registered design and the descriptive safety figures; they prevent assessment of the primary result and its precision.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Rituximab, Combination Chemotherapy, Filgrastim (G-CSF), and Plerixafor in Treating Patients With Non-Hodgkin Lymphoma Undergoing Mobilization of Autologous Peripheral Blood Stem Cells — NCT01097057.
• Registry record last updated 2018-01-23; status last verified 2017-12.
• Results first posted 2017-07-02.
• 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.

19. Record Summary

NCT01097057 is a completed phase II, single-group trial sponsored by Fred Hutchinson Cancer Center, with enrollment listed as 20. It studied rituximab, ICE chemotherapy, G-CSF, and plerixafor for mobilization of autologous peripheral blood stem cells in patients with non-Hodgkin lymphoma. The primary endpoint was the number of patients reaching at least 5 x 10^6 CD34 cells/kg by apheresis within one month.

The registry record does not report the primary endpoint count, its denominator, or an uncertainty interval. It lists serious adverse events in 2 participants, other adverse events in 3 participants, and 5 deaths, with Febrile Neutropenia and Atrial Fibrillation listed at 5.0% each as serious-event terms. No comparator or statistical analysis records are listed. The analysis plan, missing-data handling, and safety follow-up timeframe are not reported in the registry record.