Removing Transfusion Dependence as a Barrier to Hospice Enrollment: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Removing Transfusion Dependence as a Barrier to Hospice Enrollment (NCT05063591), a na na study. The primary outcome is Proportion of Eligible Patients Choosing to Participate in the Care Model Under Study Rather Than Standard of Care..

2. Clinical Question

3. Trial Design

This completed study, NCT05063591, is titled Removing Transfusion Dependence as a Barrier to Hospice Enrollment. It is recorded as a single-group study of transfusion support, with enrollment of 18. The registry lists the phase and allocation as NA and masking as none. These entries do not describe a randomized, masked comparison. The design information therefore supports a descriptive account of a care model, not a treatment-effect comparison against a concurrent control arm.

The eligible population is described as patients with aggressive hematologic malignancies who are hospice eligible, are not pursuing further cancer-directed therapy, and whose primary hematologist plans to initiate a hospice-transition conversation. The study objective is to evaluate whether external funding for transfusions during hospice encourages earlier hospice enrollment and improves end-of-life care for patients and caregivers. The description explains the rationale: patients with blood cancers may need transfusions for symptom control, while hospice coverage arrangements can make those transfusions financially unfeasible for hospice agencies. This is the study rationale, not an outcome finding.

The record lists the study start as 2021-07-09, primary completion as 2023-12-31, and status as completed. It names transfusion support as the intervention. The primary outcome concerns eligible patients choosing to participate in the care model rather than standard care. Secondary outcomes concern time enrolled on hospice, intensive-care days in the last 30 days of life, death in an acute-care hospital, and chemotherapy in the last 14 days of life. Results for these outcomes are not reported in the registry record. The registry record also does not describe recruitment flow, treatment delivery, or the timing and details of follow-up beyond the stated primary-outcome timeframe.

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.

Before assignment: Of 22 patients and 9 caregivers screened, 10 patients (45%) and 8 caregivers enrolled. 4 screened patients died before consenting.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Eligible patients approached for participation

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 Proportion of Eligible Patients Choosing to Participate in the Care Model Under Study Rather Than Standard of Care., measured over Through study completion, on average 2 years.

The primary endpoint is the count-based measure of eligible patients approached for participation and those who elect to enter the study and participate in the novel care model rather than the traditional hospice care model. The registry describes the population as eligible patients approached for participation and gives the timeframe as through study completion, on average 2 years. Although the endpoint title refers to a proportion, its description identifies the approached and electing counts as the quantities used to measure it. The registry record does not report either count or a resulting proportion.

This endpoint addresses the immediate feasibility and uptake question behind the study: whether eligible patients choose the transfusion-support care model when it is offered. It is not itself a measure of how long patients remain enrolled on hospice, the quality of end-of-life care, or the effects on caregivers. Those topics are reflected in the study objective and secondary outcomes, but the primary endpoint is participation choice. Interpreting it requires knowing how many eligible patients were approached and how many elected to participate. Without those results, uptake and its precision cannot be evaluated. A single-group participation measure can describe choices within the enrolled study setting, but by itself it cannot establish what would have happened to the same patients under a concurrent alternative care model.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design tell us?

In a single-group study, participants receive or are offered the study care model without a concurrent control group serving as a direct comparison. The design can describe what happened among people who entered or were approached for the model, provided those outcomes and denominators are reported. It cannot, by itself, separate the influence of the care model from other explanations for an observed pattern. For example, differences in who is eligible, who is approached, who chooses to participate, and what happens over time may all shape the observed experience. This is a general design principle, not a finding about this study.

Here, the registry lists a single-group model and allocation as NA. The primary outcome asks whether eligible patients approached choose the study model rather than traditional hospice care. That wording identifies a choice of care model, but it does not create a concurrent comparison group. The record does not describe random assignment or an alternative arm, and the study should not be interpreted as a randomized comparison.

How should the primary outcome be understood?

The primary outcome is described through two counts: eligible patients approached and those who elect to participate. A proportion based on these counts would summarize participation among those approached, rather than among all patients with the listed conditions or all hospice-eligible patients. This distinction matters because the approach process defines who can appear in the denominator. If the number approached is not known, the participation result cannot be reconstructed from the enrollment figure alone. The registry record does not report the needed primary outcome counts.

In general, a proportion is an estimate of a frequency in a defined group. Its interpretation depends on a clear numerator, denominator, and observation period. A confidence interval, when reported, describes statistical uncertainty around an estimate under the assumptions of the method used; it does not account automatically for selection differences or missing information. No confidence interval or primary outcome analysis is reported in the registry record.

What can the listed sample-size planning assumptions show?

The planning illustration described for this page is not the study’s own design justification. It uses an observed effect as its planning basis, considers a two-sided significance level of 0.05, assumes equal allocation between two arms, and uses a normal approximation. For time-to-event planning, the illustration expresses the required information as a total number of events; other planning results are expressed as participants per arm. The stated target effect is a fraction of the observed effect.

These are general planning conventions, not evidence that this study had two arms or used those assumptions in its protocol. In particular, the registry describes a single-group study, so equal allocation between two arms does not describe the recorded trial design. A planning illustration asks how much information a hypothetical design might need under stated assumptions. It is not an observed outcome, an estimate of the study’s precision, or observed power. The registry record does not report a protocol-based sample-size justification, and it does not report the primary outcome result needed to assess the stated observed-effect basis against the posted results.

Why do the analysis details matter?

A statistical analysis plan normally specifies how outcomes will be summarized or compared, how missing observations will be handled, and which participants belong in each analysis. Those decisions can affect the interpretation of an estimate. For a count-based participation outcome, the analysis would need to preserve the intended approached-patient denominator and describe how incomplete or unavailable participation information is treated. For outcomes such as time enrolled on hospice, the analysis also depends on the definition of the time origin, the event or end point, and how follow-up ending before an event is handled. These are general methodological considerations; the registry record does not report such details.

The registry lists zero analyses. That means no statistical analysis results are listed in the record excerpt; it does not establish what analyses may or may not have been performed outside the registry. The tables and plots are generated from recorded results where available, but the primary outcome and the listed secondary outcomes have no reported results in this record. Therefore, there is no estimate, interval, or test result to interpret for those outcomes.

How should uncertainty and over-dispersion be considered?

Uncertainty is especially important in a study with a limited enrollment and an outcome defined by counts among people approached. In general, estimates based on counts can vary substantially when the number of observations is limited, and a point estimate alone does not show the range of values compatible with the data. A confidence interval can communicate sampling uncertainty, while the denominator helps readers judge how much information supports the estimate. Neither resolves limitations arising from selective approach, selective participation, or missing outcome reporting.

Over-dispersion is a general issue for count models: it occurs when observed variation is greater than the model’s assumptions allow, which can make model-based uncertainty appear too small if ignored. The registry record does not report an over-dispersion assessment or a count-model analysis for this study. It would be inappropriate to assume that such a model was used or needed. More broadly, no statistical model, covariate adjustment, interim analysis, or inferential testing plan is reported in the registry record.

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("Intervention Arm")
n      <- c(22)
events <- c(10)
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  Intervention Arm  22  10  45.5  24.4  67.8  26.9  65.3  26.8  65.5

11. Multiplicity, Missing Data and Analysis Populations

The registry identifies a primary outcome and lists secondary outcomes concerning time enrolled on hospice, intensive-care days in the last 30 days of life, death in an acute-care hospital, and chemotherapy in the last 14 days of life. The record reports zero analyses, and results for these outcomes are not reported in the registry record. It does not state whether the outcomes were analyzed, whether any outcomes were prioritized beyond the primary designation, or whether a plan for handling multiple outcome tests was specified.

Multiplicity is a general concern when several outcomes or analyses are examined: the chance of observing at least one apparently notable result can depend on how many comparisons are made and how they are interpreted. A prespecified analysis plan can clarify which results are primary, which are exploratory, and whether any adjustment or other interpretive approach is intended. No multiplicity strategy is reported in the registry record, so the page cannot describe one for this study.

The registry record does not report missing-data amounts, reasons for missingness, imputation, censoring rules, or sensitivity analyses. It also does not describe analysis populations, such as whether analyses would include everyone eligible, everyone approached, or only those who enrolled. Those distinctions are particularly relevant to the participation endpoint, whose denominator is eligible patients approached. The record does not provide that denominator or the number electing participation. The stated enrollment of 18 should not be treated as either count without further information. As a result, neither missingness handling nor the analysis population can be evaluated from the registry information.

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 0% reporting threshold.

The adverse-event summary lists an intervention arm with 10 participants at risk for serious adverse events and 0 affected. It also lists 10 at risk for other adverse events and 0 affected. The summary lists 10 deaths. These are the reported counts; the record does not provide event terms for serious adverse events, and no serious-event terms are listed.

Read these figures as a registry summary, not as a complete narrative of safety monitoring. The record does not describe the observation window for these counts, whether events were assessed for relationship to the intervention, or how death timing relates to the adverse-event categories. It also does not explain the relationship between the intervention-arm safety denominator and the stated study enrollment of 18. The listed deaths should not be interpreted as caused by the intervention; attribution is not reported in the registry record.

Zero affected in the listed safety categories means no events were recorded in those categories in this summary. It does not establish that the care model is free of risk, particularly when the event definitions, follow-up details, and fuller clinical context are not reported. The safety figures also do not answer the primary participation question or provide results for the secondary end-of-life outcomes.

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

The study addresses a practical barrier described in its rationale: patients with blood cancers may need transfusions near the end of life, while hospice agencies may find transfusion coverage financially unfeasible. The study objective was to evaluate whether external funding for transfusions during hospice could encourage earlier hospice enrollment and improve end-of-life care for patients and caregivers. This gives the study a clear service-delivery question, distinct from a conventional comparison of two assigned treatments.

The registry record establishes that the study was completed, had enrollment of 18, and used a single-group design. It also defines a primary outcome focused on the choice to participate among eligible patients approached. However, the primary outcome counts and results are not reported in the registry record. The record likewise does not report results for the listed secondary outcomes. Therefore, the posted information cannot show how often eligible patients chose the model, how long participants remained on hospice, or whether the listed end-of-life outcomes differed from another care approach.

Even if a participation result were available, a single-group measure would describe uptake in the study setting rather than establish what would have happened under a concurrent alternative. Selection into the approach and enrollment process could shape who is represented. The registry does not report a control arm, an analysis plan, or results that would allow those issues to be assessed. The study’s stated aim and its clinical rationale should not be confused with demonstrated outcomes.

In statistical terms, the record offers design and context but insufficient outcome reporting for an estimate of effect or its precision. It does not support a conclusion that the care model increased hospice enrollment, changed end-of-life care, or improved patient or caregiver outcomes. Those are questions the study was designed to examine, not findings established by the information recorded.

14. Why This Trial Matters Statistically

This study is statistically notable because its primary question is about choosing a care model, not a conventional clinical event comparison between assigned groups. The endpoint depends on an approach process: eligible patients must be identified and approached before their participation choices can be summarized. For that reason, reporting both the number approached and the number who elect to participate is essential. Without those figures, the primary endpoint cannot be interpreted, even though total enrollment is recorded.

The single-group design also makes the boundary between description and causal interpretation important. A participation measure can describe uptake among the eligible patients who were approached, but without a concurrent control arm it cannot isolate the effect of transfusion support from other influences on hospice decisions. The study’s motivating question remains important, but the design information alone does not answer it.

The record also illustrates why outcome availability matters as much as outcome definitions. It lists a primary endpoint and several secondary outcomes, yet does not report their results or analyses. Readers therefore cannot assess the estimates, their uncertainty, or whether outcomes align with the study objective. The safety counts are a separate summary and do not fill that gap.

Finally, the sample-size planning illustration should be kept distinct from the design actually recorded. Its assumptions describe a hypothetical two-arm planning framework, while the registry lists a single-group study. Planning quantities are not observed power and do not substitute for reported outcome data. Clear separation of rationale, design, planning, and results is central to sound interpretation of clinical research.

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 this study from the registry record is the absence of the primary outcome result. The record describes eligible patients approached and those electing participation as the basis of the outcome, but reports neither count nor a resulting proportion. Enrollment of 18 does not identify the number approached, and the record does not explain how enrollment relates to the endpoint denominator.

The single-group design offers no concurrent control group. As a result, the registry information cannot support a direct comparison with traditional hospice care or determine whether the intervention itself led to any observed change. The primary outcome is a choice to participate, which is informative about uptake but does not directly measure hospice duration, intensive-care use, acute-care death, chemotherapy near the end of life, or caregiver experience.

The record lists secondary outcomes but does not report their results. It also does not report statistical analyses, confidence intervals, an analysis plan, missing-data methods, analysis populations, or the handling of follow-up that may end before an outcome occurs. Details of how transfusion support and external funding were implemented are not reported in the registry record. These omissions limit assessment of the intervention’s delivery and the completeness of outcome ascertainment.

The safety summary has limited context: it gives counts for participants at risk, affected participants, and deaths, but does not provide event timing, attribution, or further clinical descriptions. The record also does not provide a protocol-based sample-size justification. The planning illustration is not evidence of the study’s actual design rationale. Together, these limitations mean that the registry record can describe the intended question and basic design, but not establish the study’s primary or secondary outcome findings.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Removing Transfusion Dependence as a Barrier to Hospice Enrollment — NCT05063591.
• Registry record last updated 2026-01-15; status last verified 2025-12.
• Results first posted 2026-01-15.
• 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

NCT05063591 is a completed, single-group study titled Removing Transfusion Dependence as a Barrier to Hospice Enrollment. The registry lists enrollment of 18, transfusion support as the intervention, and eligible patients with aggressive hematologic malignancies who are hospice eligible and not pursuing further cancer-directed therapy as the intended population.

The primary outcome concerns eligible patients approached and whether they elect to participate in the novel care model rather than the traditional hospice care model. The registry record does not report the primary outcome counts or results. It lists secondary outcomes relating to hospice duration, intensive-care days near death, acute-care hospital death, and chemotherapy near death, but their results are not reported either.

The adverse-event summary lists 10 at risk and 0 affected for both serious and other adverse events, with 10 deaths listed. No statistical analyses are recorded. The study dates are 2021-07-09 to 2023-12-31, with status recorded as completed.