Patient-led Surveillance Compared to Clinician-led Surveillance in People Treated for Localised Melanoma.: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Patient-led Surveillance Compared to Clinician-led Surveillance in People Treated for Localised Melanoma. (NCT03581188), a randomized na study. The primary outcome is The Percentage of Eligible and Contacted Patients Who Were Randomised Into the Trial.

2. Clinical Question

The trial’s broader purpose was to evaluate digitally supported skin self-examination compared with usual care. Its secondary outcomes cover self-examination frequency and thoroughness, submission of dermoscopic images for teledermatology in the intervention group, clinic visits, excised skin lesions, subsequent primary or recurrent melanoma diagnoses, diagnoses prompted by visit type, and anxiety, stress, and depression measured using the Depression Anxiety Stress Scales-21.

The registry does not report results for these secondary outcomes. It also does not report the primary outcome’s numerator, denominator, or estimated percentage. Consequently, the record does not establish how many eligible and contacted patients entered the trial, or how the two surveillance approaches compared on clinical or behavioural outcomes.

3. Trial Design

The registry describes a randomised, parallel-group pilot trial with no masking. It lists the study as completed and identifies the University of Sydney as sponsor. The registry lists enrollment as 100. The recorded start date is 2018-11-01 and the primary completion date is 2020-02-17. The phase is listed as NA.

Participants were assigned to patient-led surveillance or clinician-led surveillance. In the patient-led arm, the described approach combined smartphone-supported lesion photography for teledermatology with guidance and reminders for skin self-examination. The description refers to a mobile dermatoscope attachment, the ASICA skin checker App, and reminders to perform self-examination every 2 months. The description of this arm is truncated in the registry record, so its full booklet content and any further intervention details are not reported in the registry record.

The clinician-led arm is described as receiving the booklet “Your guide to early melanoma” and attending scheduled clinician visits as required. The record does not report the schedule of those visits, whether clinical surveillance differed in other respects, or the extent to which each intervention was delivered as intended.

The pilot framing matters for interpretation: the stated primary outcome concerns the proportion of eligible and contacted patients who were randomised, rather than a treatment effect on melanoma diagnosis or another health outcome. Although the record lists 100 as the enrollment, it does not give the number screened, eligible, contacted, or randomised for the primary outcome. It also does not report the analysis population or participant flow. Therefore, the registry description alone cannot show how recruitment proceeded or whether the intended comparison was completed as planned.

Further design details, including the randomisation procedure, allocation concealment, follow-up duration, and protocol deviations, are not reported in the registry record. The lack of masking is explicitly recorded; reasons for that design choice are not reported in the registry record.

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 the 481 participants screened from November 1, 2018, to May 24, 2019, for eligibility, 125 were ineligible and 30 could not be contacted, leaving 326 participants who were eligible and contacted. Of them, 100 participants were randomized and enrolled in the trial.

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 The Percentage of Eligible and Contacted Patients Who Were Randomised Into the Trial, measured over Baseline.

The primary endpoint is titled “The Percentage of Eligible and Contacted Patients Who Were Randomised Into the Trial.” It measures recruitment feasibility: the numerator is the number of patients randomised, and the denominator is the number of screened patients who were eligible and contacted. The registry specifies the timeframe as baseline.

This endpoint is useful in a pilot because it describes whether people who meet eligibility criteria and are reached can be recruited into a randomised study. Recruitment information can help researchers assess the practical feasibility of conducting a larger study. It is distinct from an outcome that measures whether either surveillance approach changes melanoma diagnoses, clinic attendance, self-examination, or wellbeing.

The registry record gives the endpoint definition but does not report the numerator, denominator, or resulting percentage. The listed enrollment of 100 does not supply those missing elements: enrollment does not identify how many people were screened, eligible, contacted, or randomised for this endpoint. No confidence interval or other measure of precision for the recruitment percentage is reported in the registry record.

Because the endpoint is a single recruitment proportion rather than a reported outcome in each randomised arm, the record does not support a between-arm comparison for this primary measure. The primary outcome result is therefore not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does randomisation contribute?

In general, randomisation assigns participants to study groups using a chance-based process. If implemented successfully, it can reduce systematic differences between groups at the start of a trial and support a fair comparison of outcomes. This trial is recorded as randomised and parallel-group, but the specific sequence-generation and allocation-concealment methods are not reported in the registry record. The record also states that masking was none. It does not describe whether outcome assessors were masked.

What does the primary endpoint estimate?

The primary outcome is a proportion. Its numerator is the number of patients randomised; its denominator is the screened patients who were eligible and contacted. In general, a proportion is interpretable only when its numerator and denominator are clear and the population contributing to each is defined. Here, the endpoint definition is recorded, but the counts and estimated percentage are not reported. The enrollment figure does not substitute for the endpoint denominator because it does not identify how many patients were screened, eligible, and contacted.

How should a confidence interval be understood?

In general, a confidence interval expresses statistical uncertainty around an estimate. A narrower interval indicates greater precision under the method used, while a wider interval indicates greater uncertainty. Its interpretation depends on the data, the statistical method, and the assumptions behind that method. The registry record does not report a confidence interval for the recruitment endpoint, or the data needed to assess one. It is therefore not possible to describe the precision of the primary estimate.

What do the binary outcome counts mean?

Some listed outcomes can be represented as counts of participants with an event. In general, such counts need to be read alongside the number of participants assessed, the outcome definition, and the follow-up period. A count alone does not describe the risk in a population if its denominator or observation period is unclear. The registry lists several secondary outcome topics, but does not report their result counts, denominators, or estimates. It is not possible to infer results from the outcome titles.

What is over-dispersion, in general?

For count outcomes, some statistical models assume a particular relationship between the average count and its variability. Over-dispersion means that the observed variability is greater than the model assumes. If ignored, uncertainty estimates can be too small and inferences can appear more precise than the data justify. Whether over-dispersion is relevant depends on the outcome distribution and model. The registry record does not report a model for the listed count outcomes, so it is not possible to say whether over-dispersion was assessed or addressed in this trial.

What do the planning illustrations represent?

The record describes planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The effect to detect is expressed as a stated fraction of the observed effect. For outcomes other than time-to-event outcomes, the illustrations are expressed as participants per arm; for time-to-event outcomes, they are expressed as the total number of events. These are planning illustrations, not observed power and not evidence that the trial was designed with those sample sizes. The registry does not report the trial’s own sample-size justification. Since the primary endpoint result is not reported, the observed effect on that endpoint cannot be evaluated from the registry record.

What can be recalculated?

A recalculated estimate would be a simple, unadjusted calculation from posted summary statistics. Such a calculation can help readers understand a reported result, but it cannot replace the sponsor’s analysis when the registry reports a discrepancy. The record contains no primary-outcome counts or estimate to recalculate, and does not report a sponsor analysis of that endpoint. No recalculated primary result is available.

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("Patients Who Were Eligible and Contacted")
n      <- c(326)
events <- c(100)
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  Patients Who Were Eligible and Contacted  326  100  30.7  25.7  36.0  25.9  35.9  25.9  35.9

11. Multiplicity, Missing Data and Analysis Populations

The registry lists multiple secondary outcomes, including self-examination frequency and thoroughness, image submission for teledermatology in the intervention group, clinic visits, surgically excised lesions, melanoma diagnoses, visit-type-related diagnoses, and psychological measures. Testing many outcomes can raise the chance of at least one apparently notable finding arising by chance. In general, a prespecified analysis plan may address this through a defined hierarchy or an adjustment for multiple comparisons. The registry record does not report whether multiplicity was addressed, which outcomes were treated as confirmatory, or how secondary analyses were prioritised.

The record does not report missing-data amounts, reasons for missingness, or methods for handling missing outcomes. It also does not specify the analysis population, such as whether analyses followed the randomised assignment regardless of intervention uptake. The registry does not describe participant flow after randomisation or the handling of withdrawals and incomplete assessments.

These omissions limit interpretation because the meaning of an outcome estimate depends on who contributed data and how unavailable observations were handled. They do not justify assuming that data were complete, that a particular analysis population was used, or that the missingness was harmless. The registry record also does not report a statistical analysis plan, so the planned treatment of multiplicity and missing data is not reported in the registry record.

Since the primary result and secondary results are not reported, no claim about multiplicity-adjusted or unadjusted findings can be made 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 1% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event record lists the group “Self-examination of the Skin” with 49 at risk for serious adverse events and 0 affected; its other adverse-event figures are 49 at risk and 1 affected, with 0 deaths. The “Control” group lists 51 at risk for serious adverse events and 0 affected; its other adverse-event figures are 51 at risk and 0 affected, with 0 deaths. No serious adverse-event terms are listed.

These are counts as recorded, not evidence that the groups had equivalent safety. A zero count means no events were recorded in that category in the registry entry; it does not establish that an event could never occur. The record does not define the “other” adverse-event category in further detail, provide event descriptions for the nonzero count, or state the observation period and ascertainment procedures.

The adverse-event counts should not be treated as a comparison of treatment-related risk. The registry does not report whether events were judged related to an intervention, how events were collected, or whether reporting differed by group. The group labels in this safety record also do not provide a complete account of exposure or follow-up. These figures describe what the registry lists, with those limitations.

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

The record establishes that a randomised pilot trial compared patient-led surveillance with clinician-led surveillance in people treated for localised melanoma. It describes a digitally supported approach involving smartphone lesion photography, teledermatology, self-examination instructions, and reminders, and a clinician-led approach involving an educational booklet and scheduled visits as required.

The primary outcome asks about recruitment feasibility: the percentage of screened patients who were eligible and contacted and then randomised. That is a useful study-process measure, but it is not itself a measure of melanoma recurrence, new diagnoses, or the clinical effectiveness of surveillance. The registry record does not report the primary outcome result. It also does not report results for the listed secondary outcomes.

Accordingly, the posted record does not show whether patient-led surveillance changed self-examination behaviour, resulted in successful image submissions, altered clinic attendance or excisions, or affected melanoma diagnoses or psychological measures. It does not establish that the approaches were the same or different. Absence of reported results should not be interpreted as a finding of no effect.

The safety entry records no serious adverse events in either listed group and gives the other adverse-event counts, but event definitions, attribution, and follow-up details are not reported. Those entries alone cannot support a broad conclusion about comparative safety.

Interpretation should remain tied to the information actually recorded: a completed pilot with a stated recruitment endpoint, a listed enrollment of 100, and descriptions of the intended surveillance approaches. The registry does not report enough outcome information to judge recruitment performance, estimate comparative effects, or assess their precision.

14. Why This Trial Matters Statistically

This trial is statistically notable because its primary endpoint addresses whether a randomised evaluation of surveillance can recruit eligible and contacted patients. Feasibility outcomes can inform whether a research question can be studied in practice. They are different from clinical outcomes and should be interpreted on their own terms.

The distinction between a study’s intended question and its reported result is central. The record defines a recruitment proportion with a clear numerator and denominator, but does not report either count or the estimated percentage. Without those data, readers cannot judge recruitment success or quantify uncertainty. The listed enrollment cannot answer that question because it does not identify the relevant screened, eligible, and contacted population.

The trial also illustrates why outcome definitions alone do not establish findings. The record names secondary measures spanning behaviour, healthcare use, melanoma diagnoses, and psychological outcomes, but reports no results for them. A detailed intervention description and a completed status are not substitutes for estimates, denominators, and measures of precision.

Finally, the record makes visible the limits of registry-based statistical interpretation. It documents randomisation, parallel groups, and no masking, while leaving important analysis details unreported. Recognising those gaps avoids overstating what a pilot can establish and keeps feasibility, clinical outcomes, and safety claims distinct.

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 interpretation is that the registry record does not report the primary recruitment result. It gives neither the number randomised for that outcome nor the number screened who were eligible and contacted, and no percentage or confidence interval is reported. The listed enrollment of 100 does not resolve those omissions.

Results for the secondary outcomes are also not reported. This prevents assessment of self-examination frequency or thoroughness, image submission, clinic visits, excisions, melanoma diagnoses, or psychological measures. Follow-up periods for those outcomes are not reported in the registry record, apart from the primary endpoint timeframe of baseline.

Several design and analysis details are missing. The randomisation procedure, allocation concealment, eligibility criteria, participant characteristics, analysis population, missing-data approach, multiplicity strategy, and statistical analysis plan are not reported in the registry record. The absence of masking is recorded, but the record does not clarify assessor masking. The patient-led arm description is truncated, so its full content is unavailable.

Safety information is limited to the listed counts. Definitions, event descriptions, attribution to an intervention, observation duration, and ascertainment methods are not reported. Therefore, the safety entries should not be interpreted as a complete comparative safety analysis.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Patient-led Surveillance Compared to Clinician-led Surveillance in People Treated for Localised Melanoma. — NCT03581188.
• Registry record last updated 2023-08-01; status last verified 2023-07.
• Results first posted 2023-08-01.
• 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 randomized, parallel-group pilot trial compared patient-led with clinician-led surveillance for people treated for localised melanoma. The registry lists enrollment as 100, no masking, and the University of Sydney as sponsor. The recorded study start date is 2018-11-01 and primary completion date is 2020-02-17.

The primary outcome measures the percentage of screened patients who were eligible and contacted and then randomised. Its result is not reported in the registry record. The registry lists secondary outcomes concerning self-examination, teledermatology images, clinic use, excisions, melanoma diagnoses, and psychological measures, but does not report their results.

The safety record lists no serious adverse events in either group, with other adverse-event counts of 1 in Self-examination of the Skin and 0 in Control, and 0 deaths in each group. Further safety and analysis details are not reported in the registry record.