mPCST for BMT Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for mPCST for BMT Patients (NCT01984671), a randomized na study. The primary outcome is Feasibility as Measured by the Overall Percentage of Completed Sessions.

2. Clinical Question

The clinical question is framed around whether a pain coping skills program can be delivered and used after transplant. Its primary endpoint is the overall percentage of completed sessions, calculated from session counts across participants rather than from a reported patient-level clinical change. The registry describes the study goal as developing an intervention that could help patients manage pain following transplant and be feasible and acceptable. The posted primary result addresses session completion; a separate acceptability result is not reported in the registry record.

The trial also lists secondary outcomes concerning changes from baseline to 10 weeks in pain disability, pain self-efficacy, fatigue, and the 2-minute walk test. The outcome titles indicate what was intended to be examined, but numerical results for those outcomes are not reported in the registry record. Accordingly, the primary feasibility result should not be read as evidence about comparative changes in pain or function.

3. Trial Design

This was a randomized, parallel-group trial with no masking, and the registry status is completed. Enrollment is listed as 36. The trial is titled “mPCST for BMT Patients,” with the official title “Home-Based Tablet Computer Pain Coping Skills Following Stem Cell Transplant.” Duke University is listed as the sponsor. The recorded study start date is 2013-10-02 and the primary completion date is 2015-12-01.

The two named groups were Mobile Pain Coping Skills Training (mPCST) and Usual Care Control. The registry lists 18 participants for the mPCST primary endpoint entry. The usual-care primary endpoint entry has a participant count of 0 and no value. That entry is consistent with the endpoint definition: usual-care participants did not attend sessions, so session completion for the intervention sessions is not reported as a comparable usual-care outcome. This does not mean that the trial had no usual-care participants; the safety results list 18 participants at risk in that group.

Random allocation is intended to create groups that can be compared, while a parallel design means participants are assigned to one group rather than crossing between interventions. Those design features do not by themselves establish that a particular outcome was analyzed or that a group difference was estimated. The registry does not report the sequence-generation method, allocation concealment, protocol deviations, or an analysis plan. Masking is listed as none, so the trial was unmasked; the registry does not specify which individuals, if any, were aware of assignment beyond this design label.

The available primary result is a feasibility measure for the mPCST sessions. A randomized design is not equivalent to a reported randomized comparison: no comparative estimate, uncertainty interval, or test of the primary endpoint is reported in the registry record. The registry does not report a rationale for the enrollment target, and the available information does not establish whether enrollment or follow-up differed from the original plan.

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: Usual care participants did not attend sessions.

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 as Measured by the Overall Percentage of Completed Sessions, measured over 10 weeks.

The primary endpoint is feasibility measured as the overall percentage of completed sessions over 10 weeks. The registry defines it as the total number of completed sessions for all participants divided by the total number of planned sessions for all participants, multiplied by 100. The posted mPCST value is 83%.

This measure is used to describe whether the intervention sessions were delivered and completed as planned. It is an aggregate across sessions and participants, not a patient-level average or a measure of how much pain changed. The registry’s stated study goal includes developing an intervention that is feasible and acceptable for patients after transplant. Session completion speaks to the feasibility component; a separate acceptability result is not reported in the registry record.

The control group did not attend mPCST sessions, according to the registry. Its primary endpoint entry has no value and lists a participant count of 0 for this session-based measure. Thus, the 83% result is not a comparison between the randomized groups. The registry does not report the underlying number of planned or completed sessions, or an uncertainty interval for the percentage. Without those details, the precision of the aggregate estimate cannot be assessed from the posted result alone.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The trial is identified as randomized and parallel, with no masking. In general, randomization is used to reduce systematic differences between groups at assignment, and a parallel design keeps participants in their assigned group. These design labels describe how the study was organized; they do not guarantee that every outcome was analyzed as a group comparison. The registry does not report the randomization procedure, allocation concealment, or a statistical analysis plan.

The primary endpoint is a count-based feasibility measure expressed as a percentage: completed sessions divided by planned sessions across participants, multiplied by 100. The posted mPCST result is 83%. Because the measure pools sessions across participants, it is not equivalent to an average of individual completion percentages. In general, an aggregate can be influenced by how session opportunities are distributed among participants. The underlying completed and planned session counts, and participant-level completion data, are not reported in the registry record.

There is no reported estimate for the usual-care group on this endpoint. The registry explicitly says that usual-care participants did not attend sessions. As a general statistical principle, a value that is structurally undefined for a group should not be treated as an observed zero on a common outcome. Here, the usual-care entry has no result and a participant count of 0 for the session endpoint, while safety data list participants at risk in that group. The endpoint is therefore descriptive for mPCST rather than a two-arm comparison.

A confidence interval would ordinarily help show the uncertainty around an estimated percentage or treatment contrast. In general, such an interval reflects both the amount of information and the assumptions of the statistical method; it is not a guarantee that a particular range contains the true value. No confidence interval is reported for the 83% session-completion result. The registry also does not give the session counts needed to independently reconstruct its precision. Consequently, the percentage should be read as a point estimate without a reported precision measure.

The secondary outcome titles concern changes from baseline to 10 weeks in pain disability, pain self-efficacy, fatigue, and the 2-minute walk test. Several titles refer to Cohen’s D effect size. In general, Cohen’s D expresses a standardized difference relative to a measure of variability, allowing effects measured on different scales to be described in a common way. Its interpretation depends on how the difference and variability are defined and on the study design. The registry provides no numerical effect-size results, no underlying group summaries, and no analysis entries, so no effect size or direction of change can be interpreted from the record.

Over-dispersion is a general concern for count outcomes when observed variability is greater than expected under a simple count model. If session counts vary substantially across participants, a model that assumes less variability may yield uncertainty estimates that are too narrow. This endpoint is reported as a pooled percentage, and the registry does not describe a model for session counts or report the distribution of individual completion. There is therefore no basis to determine whether over-dispersion was assessed or how it would affect inference in this trial.

The record lists no analysis entries. It does not report whether the primary endpoint was analyzed with a formal statistical test, whether any adjustment was made for baseline characteristics, or how deviations from the assigned intervention were handled. It also does not report an inferential comparison for the clinical secondary outcomes. No p-value, standard error, confidence interval, or model estimate for a between-group effect is available in the registry record.

The sample-size assumptions describe planning illustrations based on a fraction of an observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. In general, a planning calculation estimates the sample size that would be needed under specified assumptions; it is not a measure of the probability that this completed trial detected an effect. Such illustrations are not observed power. The registry does not report the trial’s own design rationale or sample-size justification, and the planning assumptions alone do not establish that those calculations governed enrollment.

These distinctions matter because the trial’s primary result is a feasibility percentage, while sample-size calculations commonly address a specified statistical contrast. The available record does not report a comparative primary effect or the assumptions needed to connect the session-completion result to a treatment-effect calculation. No new contrast or test can be reconstructed from the endpoint entry alone.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists six outcome measures and identifies four secondary outcomes: change from baseline to 10 weeks in pain disability, pain self-efficacy, fatigue, and the 2-minute walk test. The record has no analysis entries, and numerical results for those secondary outcomes are not reported in the registry record. It does not describe a prespecified hierarchy among outcomes or a procedure for accounting for multiple statistical tests.

In general, multiplicity arises when several outcomes or comparisons are tested. If many tests are interpreted separately without an appropriate plan, the chance of at least one apparently notable result can increase. Methods such as defining a primary outcome in advance, adjusting significance thresholds, or treating secondary results as exploratory can address different multiplicity concerns. The registry does not report which, if any, approach was used. No claim about statistical significance or multiplicity-adjusted results can be made from the available outcome listings.

Missing-data methods and analysis populations are also not reported in the registry record. It does not state how missing outcome assessments were handled, whether the primary result included all randomized participants or only those with session data, or whether the secondary outcomes used an intention-to-treat or another analysis population. The primary endpoint entry lists 18 participants for mPCST and 0 for usual care; the latter reflects that usual-care participants did not attend the intervention sessions, rather than a reported missing-data result. The registry does not provide additional participant-flow details with which to assess outcome availability or attrition.

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 0% reporting threshold.

The registry lists zero serious adverse events and zero other adverse events among 18 participants at risk in the mPCST group. It also lists zero serious adverse events and zero other adverse events among 18 participants at risk in the usual-care group. Deaths are listed as zero in both groups. No serious adverse event terms are listed.

These are the posted event counts, not estimates of comparative risk. In general, observing no events does not establish that an event cannot occur or that the underlying risk is zero. Interpretation depends on the amount of observation, the way events were collected, and the completeness of reporting. The registry does not report a safety observation timeframe, event definitions, or further details about ascertainment, so the counts cannot be used to characterize those aspects.

The safety entries should be kept separate from the primary feasibility result. The 83% session-completion result concerns completed intervention sessions, not adverse events. The registry’s zero-event entries do not provide evidence about the comparative clinical outcomes listed as secondary measures.

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

The posted primary result means that the mPCST arm completed 83% of planned sessions in the registry’s 10-week feasibility measure. The measure pools sessions across participants, so it describes overall session completion rather than the experience of a typical participant. No underlying session counts or confidence interval are reported, limiting assessment of how precisely the percentage is estimated.

The result does not establish that mPCST improved pain, function, fatigue, or pain self-efficacy. Although the registry lists secondary outcomes in these areas, their numerical results are not reported in the registry record. The result also does not show a difference between mPCST and usual care. Usual-care participants did not attend mPCST sessions, and no usual-care value is reported for this session-based endpoint. There is no posted comparative estimate for a shared clinical outcome.

Feasibility and acceptability are related but distinct ideas. Session completion offers information about intervention delivery and participation. The registry describes acceptability as part of the study goal, but a separate acceptability measure or result is not reported in the registry record. The session-completion percentage alone cannot stand in for a broader assessment of acceptability.

Randomization is an important design feature, but interpretation of a randomized trial still depends on outcome definitions, data availability, analysis choices, and reported estimates. The registry does not report the statistical analysis plan, analysis populations, missing-data methods, or a comparative outcome analysis. Therefore, the narrow conclusion supported by the posted primary result is descriptive: the mPCST group’s aggregate session completion was reported as 83% over 10 weeks. Broader claims about clinical benefit, comparative effectiveness, or generalizability are not established by that result.

13. Why This Trial Matters Statistically

This trial illustrates why the endpoint must be matched to the question. Its primary outcome is a feasibility percentage based on completed and planned intervention sessions. That is informative about whether the program’s session activity was completed, but it is not a clinical outcome and it does not provide a direct contrast with usual care. Treating the 83% result as evidence of a treatment effect would confuse delivery of an intervention with its effect on patients.

The record also demonstrates how endpoint structure affects comparability. Usual-care participants did not attend mPCST sessions, so the session-completion endpoint has no usual-care result. A randomized design does not make every endpoint comparable across groups; the outcome must be meaningful and defined for each arm. For a comparative question, an outcome measured in both groups and a reported between-group estimate would be needed, but those are not present for this primary feasibility measure.

Precision is another central issue. A point estimate can be useful descriptively, while an interval or other uncertainty measure helps readers judge how much information supports it. No such precision measure or underlying session counts are reported for the 83% result. The secondary outcome titles likewise do not substitute for estimates, uncertainty, and analysis details.

Finally, the registry lists no analysis entries. A clear record of analysis populations, missing-data handling, and multiplicity decisions helps distinguish planned analyses from exploratory ones and supports fair interpretation. Their absence does not erase the feasibility result, but it limits what can be concluded from the recorded information. The trial is a useful example of the distinction between documenting feasibility and establishing comparative clinical effects.

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 primary outcome is an aggregate session-completion percentage, and the underlying counts of completed and planned sessions are not reported in the registry record. Participant-level completion patterns are also unavailable. As a result, the registry does not show whether session completion was broadly similar across participants or concentrated among some participants.

No confidence interval or other precision measure accompanies the 83% result. The record also gives no statistical analysis plan, no analysis entries, and no description of how missing session or outcome information was handled. The primary endpoint entry does not provide a usual-care value because that group did not attend mPCST sessions, so the feasibility result does not support a direct group comparison.

Although the registry lists secondary outcomes covering pain disability, pain self-efficacy, fatigue, and walking performance, their numerical results are not reported. The available record therefore cannot establish whether those outcomes changed, in which direction, or by how much. The trial’s allocation method, concealment procedures, and details about who knew group assignment are not reported; masking is listed as none.

The registry describes the study as completed, but further details about participant flow, follow-up, protocol deviations, and outcome ascertainment are not reported in the registry record. Safety entries list no events, but the observation timeframe and event-collection methods are not reported. Finally, the registry does not report the trial’s sample-size rationale. These limitations constrain assessment of precision, comparative effects, and the extent to which the result applies beyond the recorded feasibility measure.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: mPCST for BMT Patients — NCT01984671.
• Registry record last updated 2025-10-28; status last verified 2025-10.
• Results first posted 2025-10-28.
• 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

NCT01984671, “mPCST for BMT Patients,” is a completed randomized, parallel-group trial with no masking, sponsored by Duke University. Enrollment is listed as 36. The study evaluated Mobile Pain Coping Skills Training against usual care after hematopoietic stem-cell transplant.

The primary endpoint was overall session completion over 10 weeks. The mPCST result is 83%; usual-care participants did not attend sessions, and no comparable usual-care endpoint value is reported. The registry lists secondary outcomes for changes in pain disability, pain self-efficacy, fatigue, and the 2-minute walk test, but their numerical results are not reported in the registry record. Safety entries list zero serious adverse events, zero other adverse events, and zero deaths in both groups. No analysis entries are listed.