A Multimodal Intervention to Address Sexual Dysfunction in Hematopoietic Stem Cell Transplant Survivors: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Multimodal Intervention to Address Sexual Dysfunction in Hematopoietic Stem Cell Transplant Survivors (NCT02492100), a na na study. The primary outcome is Feasibility Primary Endpoint.

2. Clinical Question

The central question is about the practical feasibility of enrolling patients and retaining them for follow-up, not whether the intervention improved sexual function or other health outcomes. The registry describes the study as evaluating feasibility and impact on sexual function, quality of life, and mood. It lists measures for those outcomes, but results for them are not reported in the registry record. The reported feasibility figures can be considered against the study’s stated thresholds. They do not establish what would have happened without the intervention, nor do they support a treatment-effect estimate from a controlled comparison. The study population is described as transplant survivors in remission more than 6 months after allogeneic bone marrow transplant; whether the findings apply to other groups is not established by this record.

3. Trial Design

This completed study was registered as a single-group behavioral intervention study. The registry lists enrollment of 47 participants and an expected participation of about 50 stem cell transplant survivors. It lists no phase and no allocation method. Masking is listed as none. The study began in 2015-06, reached primary completion in 2017-09, and is recorded as completed.

The intervention is named a multi-modality sexual dysfunction intervention. The description refers to patients in remission more than 6 months after allogeneic bone marrow transplant and outlines an initial enrollment and baseline data collection, followed by an intervention visit. That visit included comprehensive assessment, normalization and education, therapeutic interventions, and referral to sexual health care. Further detail about the intervention schedule, its full content, and the intended number or spacing of visits is not reported in the registry record.

There was no randomized comparator and no like-for-like control group. The posted figures are therefore descriptive of participants who entered this single-group study. They do not provide a randomized estimate of how much an outcome changed because of the intervention. The registry identifies sexual function, quality of life, and psychological distress as secondary outcome areas, but it does not report their results or the timing of their assessments. The primary feasibility endpoint has a 6-month timeframe. The precise follow-up schedule and the population included in each reported outcome are not reported in the registry record.

The registry gives no statistical analysis entries. The specific analysis methods, handling of missing observations, and analysis populations are not reported in the registry record. The study’s feasibility criteria are described as two separate requirements rather than a composite outcome: the study is deemed feasible if both are met. The posted participation and visit-attendance figures can be read against those requirements, while keeping in mind that the record does not provide additional detail about how the figures were calculated or their uncertainty.

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:

Analysis population for the primary outcome, as defined in the registry: overall 94% (47 of 50) patients who screened positive for sexual dysfunction agreed to participate in the study and 80% attended at least 2 intervention visits

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 Feasibility Primary Endpoint, measured over 6 months.

The primary endpoint is a feasibility measure expressed as a count of participants, with a 6-month timeframe. It concerns two practical steps: whether patients screening positive for sexual dysfunction causing distress agree to participate, and whether participants complete at least one additional follow-up visit. The registry explicitly states that this is not a composite outcome, while defining the overall feasibility decision as positive only when both criteria are met.

These criteria address whether recruitment and continued participation appear workable for this intervention and population. Agreement to participate reflects willingness to enroll among those who screened positive. Follow-up attendance reflects whether participants return for further contact after enrollment. Such measures can inform whether a study approach is practicable; they are distinct from measures of clinical benefit or change in symptoms.

The registry reports that 94% (47 of 50) of patients who screened positive agreed to participate, and that 80% attended at least 2 intervention visits. These figures meet the stated participation and follow-up thresholds: at least 75% agreement and at least 80% completion of one additional follow-up visit. The registry’s attendance wording is not identical to the endpoint wording, but attendance at at least 2 intervention visits is consistent with having attended an additional visit beyond an initial visit. The record does not report uncertainty intervals for these percentages or further details about visit completion and participant disposition.

Feasibility is useful to distinguish from effectiveness. The endpoint does not quantify improvement in sexual function, quality of life, or mood. Although those outcomes are listed as secondary measures, their results are not reported in the registry record. The feasibility endpoint therefore answers a narrower question about participation and follow-up, not whether the intervention changed those outcomes.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a feasibility endpoint, not a treatment-effect endpoint. It uses participant counts to represent two distinct stages: agreement to join among patients screening positive for distressing sexual dysfunction, and attendance at follow-up after enrollment. The registry states that both criteria must be met for the study to be deemed feasible. Keeping the two requirements separate matters because success on recruitment alone would not satisfy the stated definition if follow-up were below its threshold, and the reverse would also be true.

The registry reports 94% (47 of 50) agreement to participate. It reports 80% attendance at at least 2 intervention visits. These are descriptive proportions: each expresses a count relative to a stated group. The record does not provide further information about how screening was conducted, whether all eligible patients were approached, how visit attendance was recorded, or how participants with incomplete follow-up were handled. Those details are not reported in the registry record.

As a general statistical principle, a proportion is an estimate of the frequency of an outcome in the group observed. Its precision depends in part on the amount of information in that group and on the outcome frequency. A confidence interval can convey uncertainty around the estimate, rather than presenting the point estimate alone. No confidence intervals for the feasibility figures are reported in the registry record, so the uncertainty around those estimates cannot be evaluated from the posted results.

The study is described as single group, with no randomized allocation. In a randomized comparison, outcome patterns between groups can be used to estimate a contrast, subject to the design and analysis assumptions. This trial has no such comparison group. Changes over time, if they had been reported, could reflect the intervention, other care, natural variation, or other influences. The registry does not report results for the listed secondary outcomes, so there are no posted change estimates to interpret in any case.

The outcome list includes sexual function and satisfaction, quality of life, and psychological distress measures. The registry identifies the PROMIS Sexual Function and Satisfaction Measure, the Functional Assessment of Cancer Therapy-Bone Marrow Transplant, the Hospital Anxiety and Depression Scale, and the Patient Health Questionnaire-9. The listed outcome descriptions concern change, but the result values, scoring details, assessment timepoints, and analysis methods are not reported in the registry record. The registry lists 4 outcome measures and 0 analyses. These entries do not establish that all measures were collected or analyzed; additional details are not reported.

The registry’s general sample-size assumptions describe a planning illustration based on an observed effect, with two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. They state that the effect to detect is a stated fraction of the observed effect, and distinguish total events for time-to-event outcomes from participants per arm for other outcomes. These are planning assumptions, not evidence that this single-group study was designed as a two-arm trial. The registry does not report the numerical planning results, an effect value for this feasibility endpoint, or a trial-specific sample-size justification. Such planning illustrations do not represent observed power.

General statistical concepts can help explain the limits of a small descriptive feasibility study. An analysis plan specifies how outcomes will be summarized or compared, which participants will be included, and how incomplete data will be treated. Prespecification can reduce ambiguity in interpreting results, but the analysis plan is not reported in the registry record. Over-dispersion is a general concern when count data vary more than a simple model assumes; it is not a documented feature of the posted feasibility results, and no count model is reported. No modeling assumptions should be inferred from the endpoint’s count format alone.

Likewise, the record does not report interim analyses, adjustment for multiple outcomes, or a strategy for missing data. These omissions mean that readers cannot determine whether all planned analyses were completed or how any missing outcome observations would have affected estimates. The published registry information supports a descriptive reading of the two feasibility figures. It does not support reconstructing an unreported inferential analysis or treating planning assumptions as the study’s actual analysis method.

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("Single Arm Study")
n      <- c(47)
events <- c(38)
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  Single Arm Study  47  38  80.9  66.7  90.9  67.5  89.6  67.4  89.5

11. Multiplicity, Missing Data and Analysis Populations

The registry lists several outcome domains: feasibility, sexual function and satisfaction, quality of life, and psychological distress. It reports 4 outcome measures, but lists 0 analyses. Results for the listed secondary measures are not reported in the registry record. The record also does not report whether a particular outcome was designated as primary among the secondary measures, whether a hierarchy was specified, or whether statistical tests were adjusted for multiple comparisons.

Multiplicity is a general statistical issue that can arise when several outcomes or tests are considered: the more results examined, the greater the chance that at least one may appear notable by chance, depending on the testing approach. Without an analysis plan or reported results, it is not possible to assess how multiplicity was handled in this study. No adjustment method should be assumed.

Missing-data handling is also not reported in the registry record. The participation and visit-attendance summaries do not describe participant flow in detail, reasons for missed visits, or whether outcome measures were incomplete. The record does not state whether analyses used all enrolled participants, only participants with observed data, or another population. It also does not report imputation or sensitivity analyses.

These gaps limit assessment of the precision and completeness of the outcome evidence. They do not change the arithmetic meaning of the posted feasibility summaries, but they leave unanswered how missing observations or unreported analyses might affect broader conclusions. No analysis population or missing-data approach can be inferred from the fact that the study enrolled 47 participants.

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 registry reports one study group with 47 participants at risk for serious adverse events and 0 affected. For other adverse events, it likewise reports 47 at risk and 0 affected. The group entry also records 1 death. The registry does not list any top serious adverse-event terms.

These figures describe what was recorded for the study group; they do not establish whether an event was caused by the intervention. The record does not report the cause or timing of the death, whether it was considered related to study participation, or further details about the event-monitoring period. Those details are not reported in the registry record. The death count is distinct from the serious and other adverse-event counts in the posted entry, and should not be combined with them without additional information.

A zero count in the registry is not proof that risk is absent. As a general statistical point, a study with limited enrollment may not characterize uncommon events precisely. The posted safety information should therefore be read as event counts in this study group, not as a comparative safety estimate. There is no control group from which to estimate a difference in adverse-event frequency.

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

The registry’s feasibility results answer a practical question: did the study meet its stated recruitment and follow-up targets? The reported agreement rate was 94% (47 of 50), compared with a threshold of at least 75%. The reported attendance figure was 80% at at least 2 intervention visits, compared with a threshold of at least 80% for completing one additional follow-up visit. Under the registry’s rule that both conditions must be met, these reported figures satisfy the stated criteria.

That conclusion is bounded by what the endpoint measures. It concerns willingness to enroll and attendance, not improvement in sexual function, quality of life, or psychological distress. Although those outcome measures are listed, their results are not reported in the registry record. The feasibility findings therefore cannot be used to say whether participants’ symptoms or quality of life changed.

The study is single group and has no randomized control condition. It cannot estimate a comparative intervention effect. Even if before-and-after outcome results were available, a single-group change would not by itself separate intervention effects from other changes over time. No such secondary outcome estimates are reported in the registry record.

The findings also do not establish that the same participation and retention experience would occur in other clinical settings or among other transplant survivor groups. The population described is limited to patients in remission more than 6 months after allogeneic bone marrow transplant who screened positive for distressing sexual dysfunction. Details on broader eligibility, setting, and recruitment are not reported in the registry record.

In short, the posted data support a threshold-based feasibility interpretation for this study. They do not demonstrate clinical benefit, establish comparative effectiveness, or provide a complete account of safety. The registry does not report uncertainty intervals for the feasibility estimates, so their precision beyond the point summaries cannot be judged from the record.

14. Why This Trial Matters Statistically

Feasibility is often a necessary statistical and practical question before a larger or more controlled evaluation can be considered. Recruitment and follow-up determine whether a study can enroll the intended population and collect later observations. In this trial, the registry’s criteria make those practical targets explicit: agreement to participate and completion of an additional follow-up visit. Reporting both measures allows readers to see that feasibility was not defined solely by enrollment.

The single-group design also clarifies what the results can answer. The study can describe participation and attendance within its enrolled group, but it cannot produce a randomized comparison of intervention outcomes. Separating feasibility from treatment effect helps prevent a common interpretive error: treating successful recruitment or retention as evidence that an intervention improves health outcomes.

The record lists measures for sexual function, quality of life, and mood, but does not report results for them. That distinction is statistically important. Naming an outcome measure does not provide an estimate of change, its uncertainty, or evidence of a difference. Without those results, the clinical impact question remains unanswered by the registry record.

The reported feasibility thresholds and observed figures make the practical aim legible, while the missing analysis details mark the limits of what can be assessed. No analysis plan, missing-data strategy, or uncertainty intervals are reported in the registry record. These omissions matter because they affect how fully readers can judge robustness and precision, even when a descriptive result meets its stated target.

The study thus illustrates why a trial’s design, endpoint, and available results must be interpreted together. The posted data are informative about recruitment and visit attendance in this cohort. They are not a substitute for comparative outcome evidence, and they do not answer whether the intervention changed the secondary outcomes listed in the registry.

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 registry record provides limited detail about how the intervention was delivered. It describes assessment, education, therapeutic interventions, and referral, but the full content, visit schedule, and criteria for referral are not reported in the registry record. This makes it difficult to judge how consistently the intervention could be reproduced from the description alone.

The record also does not provide a detailed account of participant flow. It reports 47 enrolled from 50 patients who screened positive and reports attendance at at least 2 intervention visits, but does not report reasons for nonparticipation, missed visits, or completion of other assessments. Further detail on eligibility and recruitment procedures is not reported in the registry record.

Although sexual function, quality of life, and psychological distress are listed as secondary outcomes, their results are not reported. Nor are assessment timepoints, scoring details, or change estimates. The registry lists 0 analyses, and does not report an analysis plan, missing-data method, or multiplicity approach. These omissions prevent assessment of the statistical methods and the completeness of outcome reporting.

The study has no control group, so it cannot estimate an intervention effect relative to another condition. The feasibility figures are also not accompanied by confidence intervals in the registry record. As a result, the precision of the observed participation and attendance summaries is not reported. The registry’s expected participation of about 50 is not a formal sample-size justification, which is not reported.

The safety entry records 1 death but gives no cause, timing, or relationship assessment. The event counts therefore cannot establish whether the death was related to the intervention. Taken together, these trial-specific limitations support a narrow interpretation focused on the reported feasibility thresholds rather than broader clinical or causal conclusions.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Multimodal Intervention to Address Sexual Dysfunction in Hematopoietic Stem Cell Transplant Survivors — NCT02492100.
• Registry record last updated 2019-11-27; status last verified 2019-11.
• Results first posted 2019-11-27.
• 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

This completed, single-group behavioral study evaluated a multimodal intervention for sexual dysfunction in hematopoietic stem cell transplant survivors. The registry reports enrollment of 47 participants and a primary feasibility timeframe of 6 months. The population description refers to patients in remission more than 6 months after allogeneic bone marrow transplant.

The feasibility definition required both at least 75% of patients screening positive for distressing sexual dysfunction to agree to participate and at least 80% to complete at least one additional follow-up visit. The posted results report 94% (47 of 50) agreement and 80% attendance at at least 2 intervention visits, meeting both stated thresholds as reported.

Sexual function, quality of life, and psychological distress are listed as secondary outcomes, but their results are not reported in the registry record. The study has no randomized comparator, and the record does not report an analysis plan, missing-data approach, or confidence intervals for the feasibility estimates. Safety counts include 0 serious adverse events, 0 other adverse events, and 1 death; further detail about the death is not reported in the registry record.