TACE Using Doxorubicin-eluting Beads for Patients With HCC and Marginal Hepatic Reserve: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for TACE Using Doxorubicin-eluting Beads for Patients With HCC and Marginal Hepatic Reserve (NCT02147301), a na na study. The primary outcome is Best Observed Radiographic Response Rate (Measured by mRECIST).

2. Clinical Question

The study asked whether transarterial chemoembolization using doxorubicin-eluting beads could be used safely and effectively in people with liver-only hepatocellular carcinoma and baseline hepatic dysfunction. Its primary outcome was the best observed radiographic response, assessed using modified RECIST criteria for hepatocellular carcinoma (mRECIST) over 1 year. The outcome grouped complete response, partial response, and stable disease as responses, with the eligible imaging population defined as participants with at least one follow-up cross-sectional imaging study.

Because the study has no comparator group, its design cannot estimate a treatment effect against another treatment or against no treatment. It can describe outcomes observed in the treated group, subject to the registry's endpoint definition and the availability of imaging. The record does not report the primary response count or the number of participants included in that endpoint analysis, so the response result and its precision cannot be assessed from the registry record.

3. Trial Design

The registry describes a prospective phase II study of chemoembolization with doxorubicin-eluting microspheres in liver-transplant candidates with hepatocellular carcinoma and marginal hepatic reserve. The study is recorded as single-group, with allocation listed as NA and masking listed as none. The phase field is recorded as NA. These entries do not describe a randomized comparison or a blinded assessment process.

The intervention is DEB-TACE using doxorubicin-loaded LC Beads. The registry describes delivery through a hepatic artery catheter under fluoroscopic visualization. The enrollment is 17. The study began on 2014-12-17, and the primary completion date is listed as 2017-01-27. Its overall status is terminated. The reason for termination is not reported in the registry record; the status alone does not establish why the study stopped or what effect termination had on outcome collection.

The primary outcome is best observed radiographic response by mRECIST, with a 1-year timeframe. The record also lists secondary outcomes covering radiographic response, progression and survival measures, alpha-fetoprotein response, left ventricular ejection fraction, and area under the curve. There are 10 outcome measures listed, but no analyses are listed in the registry record. That distinction matters: an outcome definition indicates what was intended to be measured, but does not establish that a result was collected, analyzed, or reported.

Since this is a single-group study, any observed response or safety frequency would describe participants who received the intervention. Without a concurrent control group, such results cannot separate outcomes associated with treatment from the course of disease, other care, or participant characteristics. The registry record does not report a detailed statistical analysis plan, the reason for termination, or the number assessed 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

Analysis population for the primary outcome, as defined in the registry: Best observed radiographic response for all patients who had at least one follow-up cross-sectional imaging study

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 Best Observed Radiographic Response Rate (Measured by mRECIST), measured over 1 year.

The primary endpoint is the best observed radiographic response rate measured by mRECIST, with results intended to be assessed over 1 year. The registry defines the measure as the number of patients whose best observed response was complete response (CR), partial response (PR), or stable disease (SD), divided by the total number of patients with at least one available CT or MRI. The primary population is described as patients with at least one follow-up cross-sectional imaging study.

mRECIST is used in hepatocellular carcinoma to assess viable tumor using arterial enhancement on imaging. In the registry definition, CR means disappearance of intratumoral arterial enhancement in all target lesions. PR requires at least a 30% decrease in the sum of viable target-lesion diameters from baseline. SD covers cases that do not meet the criteria for PR or progressive disease. The record describes progressive disease as an increase of at least 20% in the sum of viable target-lesion diameters, using the smallest such sum as the reference.

Combining CR, PR, and SD gives a broad measure of disease control on imaging rather than a measure limited to tumor shrinkage. It is useful for describing radiographic status, but it does not by itself establish a survival benefit, symptom improvement, or a causal treatment effect. The registry record does not report the observed primary endpoint count or its denominator, so the endpoint's result cannot be interpreted from the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a single-group study and a binary primary endpoint built from participant response categories. The central statistical issue is therefore not a comparison between randomized groups, but the estimation and interpretation of a response frequency within the imaged study population. The record defines the endpoint but does not report the estimate or the number of participants contributing to it.

What does the response endpoint count?

The endpoint classifies each eligible participant according to their best observed imaging response. Participants whose best response was CR, PR, or SD count toward the response measure; the definition uses patients with at least one available CT or MRI as its denominator. This is a participant-level binary summary: each eligible person is either included as having one of the qualifying response categories or is not. The endpoint does not count the number of lesions that respond, and it does not describe the degree of response beyond the category definitions.

Best observed response compresses a participant's imaging assessments into one category. That can be useful for describing the most favorable response recorded during the observation period, but it does not show the sequence or duration of imaging findings. The registry does not report assessment schedules, the number of scans, or rules for handling scans obtained outside the stated timeframe.

Why does the denominator matter?

A response frequency depends on both the response count and the population used as its denominator. Here, the endpoint definition restricts the denominator to participants with at least one available CT or MRI, while the population description refers to at least one follow-up cross-sectional imaging study. The record does not report how many participants met this imaging condition. If participants lack follow-up imaging, their response status may not be classifiable under this definition. The number and handling of such participants are not reported in the registry record.

In general, a confidence interval describes the uncertainty around an estimated quantity, such as a response frequency. Wider intervals indicate less precision, while narrower intervals indicate greater precision, given the analysis and assumptions used. The registry record does not provide a primary response estimate or confidence interval, so the precision of this endpoint cannot be assessed from the record.

What can a single-group design estimate?

A single-group study can describe outcomes observed among the participants treated, provided the outcomes and analysis population are reported. It cannot, on its own, estimate how outcomes would have differed under a comparator treatment or no treatment. In a randomized comparison, allocation helps make groups comparable on average; this study has no separate comparison group and is not recorded as randomized. Differences between the observed study experience and an external expectation would not substitute for a concurrent control, because the groups and circumstances may differ.

Masking is listed as none. In general, masking can reduce the risk that knowledge of treatment assignment influences some assessments or decisions. The registry does not report whether radiographic response was reviewed independently, whether assessors were unaware of clinical information, or whether any other safeguards were used. These details should not be inferred from the masking field.

How should planning illustrations be read?

The registry describes sample-size assumptions for planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a specified fraction of the observed effect. Time-to-event illustrations are described as giving a total number of events, while other illustrations are described in participants per arm. These are planning conventions, not observed trial results or evidence of observed power.

Those assumptions describe a hypothetical two-arm planning exercise and do not match the recorded single-group design. They should not be read as the trial's design rationale, a prospective sample-size justification, or a report of the trial's statistical power. The registry record does not report the study's own sample-size justification or a detailed statistical plan. Because the primary observed response result is not reported, the record also does not permit an independent assessment of how an effect-based planning illustration relates to the study's actual findings.

What statistical detail is missing?

No analyses are listed in the registry record. The record does not report a response estimate, its uncertainty, an inferential test, or a model for the primary endpoint. It also does not report whether any adjustment was made for participant characteristics or how protocol deviations were handled. For the listed time-to-event outcomes, the record names the outcomes but does not provide estimates or analysis details in the registry record for this interpretation. These omissions limit what can be concluded about the analysis, beyond the stated endpoint definition and design.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 10 outcome measures, including the primary radiographic response endpoint and secondary measures related to response, progression, survival, alpha-fetoprotein, cardiac function, and drug exposure. When a study examines several outcomes, the chance of finding at least one apparently notable result can increase if each is tested without accounting for the collection of tests. Statistical procedures can address multiplicity, but the registry record does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or an analysis plan that explains how multiple results would be interpreted.

No analyses are listed in the registry record, and the primary response result is not reported. Accordingly, there are no posted primary or secondary effect estimates to compare, and no inferential results from which to assess whether multiplicity was handled in practice. The fact that an outcome appears in the registry does not establish that it was measured or analyzed.

The primary endpoint's definition requires at least one available CT or MRI. This makes imaging availability part of the endpoint population. The registry describes the target population for the primary outcome as patients with at least one follow-up cross-sectional imaging study, but does not report the number meeting that condition, the number without follow-up imaging, or how missing imaging was handled. It also does not describe other missing-data procedures, imputation, exclusions, or sensitivity analyses.

The analysis population for each secondary outcome is not reported in the registry record. Without participant flow and outcome-specific denominators, the completeness of follow-up and the comparability of analysis populations cannot be assessed. The record's enrollment count should not be assumed to be the denominator for every outcome.

11. 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: hepatic failure (23.5%), hyponatremia (23.5%), hyperbilirubinemia (17.6%), ast elevation (17.6%), thrombocytopenia (17.6%).

The registry's DEB-TACE group summary lists 17 at risk and 13 affected for serious adverse events. For other adverse events, it lists 17 at risk and 17 affected. The group summary also reports 12 deaths. These are registry counts; the record does not provide enough detail to determine when events occurred, whether they were considered related to treatment, or whether the same participants contributed to more than one event category.

The most frequently listed serious adverse event terms are hepatic failure and hyponatremia, each reported as 23.5%. Hyperbilirubinemia, AST elevation, and thrombocytopenia are each reported as 17.6%, while edema of legs is reported as 11.8%. These percentages describe the listed terms in the registry safety summary. They are not a comparative estimate of excess risk, because the study has no control group. The record does not provide event-level detail, severity grades, duration, timing, or attribution for these terms.

The reported deaths should not be interpreted as treatment-caused deaths: cause and timing are not reported in the registry record. Likewise, safety counts from a single group cannot establish how those counts compare with outcomes under another approach. The figures are best read as descriptive safety reporting, with important context and causal details unavailable.

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

The registry record identifies a clinically focused question: whether DEB-TACE could be used in people with hepatocellular carcinoma and marginal hepatic reserve who were candidates for liver transplantation. It also specifies a radiographic endpoint based on best observed mRECIST response, and records selected adverse event information. These details show what the study set out to describe and what safety information is listed.

The record does not report the primary response count, its denominator, or an estimate of response precision. Consequently, it does not establish the observed radiographic response result. The enrollment figure is not a substitute for the endpoint denominator, which is defined by imaging availability. Nor does the list of outcome definitions show that all outcomes were measured or analyzed.

The single-group design also limits causal interpretation. Without a comparator group, observed outcomes cannot show what would have happened to similar participants under another treatment or without the intervention. The registry record does not support a comparative claim about efficacy or safety. Its terminated status does not explain why the study ended, and should not be treated as evidence for any particular outcome or reason.

The safety summary offers descriptive counts and percentages, but the registry does not report attribution, timing, or detailed event context. The record's 12 deaths cannot be assigned to the intervention based on the information reported. Safety frequencies also cannot be directly contrasted with another treatment in this single-group study.

In general, a trial result is most informative when the outcome estimate, uncertainty, analysis population, missing-data handling, and study design are considered together. Here, several of those elements are not reported in the registry record. The appropriate interpretation is therefore limited: the record documents the study design, intended endpoint, and some safety observations, but it does not establish the primary outcome result or a treatment effect.

13. Why This Trial Matters Statistically

This study illustrates why a registered endpoint and a reported result are not the same thing. The registry gives a detailed definition of best observed mRECIST response, including the response categories and imaging-based population. Yet it does not report the primary response count or denominator. Without those elements, readers cannot assess the observed response frequency or its precision.

The study also highlights the distinction between descriptive evidence and comparative evidence. A single-group design can describe what was observed among treated participants, but it cannot isolate the effect of treatment from other influences. This matters particularly when the stated question includes both safety and effectiveness: descriptive safety counts may identify events recorded during the study, but a comparator is needed for a direct treatment-group contrast, and event attribution also depends on detailed clinical reporting.

The endpoint's dependence on follow-up imaging makes outcome availability important. The registry definition specifies an imaging-evaluable population, yet the size and completeness of that population are not reported. Statistical interpretation therefore depends not only on the endpoint formula but on who contributed data and how missing assessments were handled.

Finally, the record lists multiple outcomes and marks the study as terminated, but does not report analyses or a detailed statistical plan. These features reinforce the value of distinguishing planned measurement, observed data, analysis, and interpretation. For this trial, the registry provides enough information to explain the question and design, but not enough to reconstruct or evaluate the main efficacy result.

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 limitations constrain interpretation of the registry record. First, the primary radiographic response result is not reported. The record defines the endpoint and its imaging-based denominator, but does not give the response count, denominator, or uncertainty estimate. Enrollment of 17 cannot be substituted for the endpoint population because the definition requires imaging availability.

Second, the study is recorded as single-group, without allocation to a comparator. It therefore cannot provide a randomized treatment contrast. The masking field is listed as none, and the registry record does not describe independent imaging review or other procedures that might limit assessment bias. The phase field is NA, and the record does not provide a detailed statistical analysis plan.

Third, the registry status is terminated, but the reason is not reported. The record does not clarify whether planned follow-up was complete, whether the enrollment target was met, or whether termination affected data collection. No explanation should be inferred from the status alone.

Fourth, missing-data information is limited. The primary outcome population depends on available CT or MRI, but the number lacking imaging, their characteristics, and the handling of missing assessments are not reported. Outcome-specific analysis populations and secondary outcome estimates are also not reported in the information available for interpretation.

Finally, the safety summary is descriptive and lacks timing, attribution, severity grading, and event-level details. The reported deaths have no stated cause or timing. The listed outcomes and safety counts can therefore characterize what the registry records, but they cannot resolve comparative benefit, causal safety, or the reasons the study ended.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: TACE Using Doxorubicin-eluting Beads for Patients With HCC and Marginal Hepatic Reserve — NCT02147301.
• Registry record last updated 2019-09-10; status last verified 2019-08.
• Results first posted 2019-09-10.
• 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.

18. Record Summary

NCT02147301 is a prospective, single-group study of DEB-TACE using doxorubicin-eluting LC Beads in liver-transplant candidates with hepatocellular carcinoma and marginal hepatic reserve. Enrollment is 17; allocation is listed as NA, masking as none, phase as NA, and status as terminated. The primary endpoint is best observed radiographic response by mRECIST over 1 year, defined among participants with at least one available CT or MRI. The registry record does not report the primary response result or its analysis denominator. It lists 10 outcome measures and no analyses. The safety summary reports serious and other adverse event counts, selected serious adverse event terms, and 12 deaths, without the detailed context needed to establish cause or comparative risk. The reason for termination is not reported in the registry record.