Comparative Efficacy of the Masquelet Versus Titanium Mesh Cage Techniques for the Treatment of Large Long Bone Defects: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Comparative Efficacy of the Masquelet Versus Titanium Mesh Cage Techniques for the Treatment of Large Long Bone Defects (NCT02015390), a randomized na study. The primary outcome is Number of Participants With Defect Healing - Plain Radiography.

2. Clinical Question

3. Trial Design

The registry identifies NCT02015390 as a randomized, parallel-group trial with no masking. It lists an enrollment of 16 participants and marks the study as completed. The start date is 2013-10-01 and the primary completion date is 2019-09-30. The registry record does not report a separate study completion date, so the primary completion date should not be treated as a substitute for one.

The two arms are Masquelet Defect Reconstruction and Titanium Cage Reconstruction. The posted arm counts are 9 participants for Masquelet reconstruction and 7 for titanium cage reconstruction. The record does not give further details about the randomization process, allocation concealment, or whether the allocation ratio was planned to be equal. These design features are not reported in the registry record.

The trial compares surgical reconstruction approaches for segmental long bone defects. The Masquelet arm is described as a two-stage technique; the titanium cage arm is described as a single-stage technique. These are registry descriptions, not a complete account of the procedures or of how care was delivered. Phase is listed as NA.

The primary outcome has a timeframe of two weeks after surgery and concerns plain radiography. Secondary outcomes named in the record include Brief Pain Inventory assessments and an overall functional outcome score. The registry lists 27 outcome measures and 19 posted secondary outcomes, but does not supply numerical results for those secondary outcomes in the information summarized by the record. The statistical analysis results and the planned analysis methods are not reported in the registry record. Consequently, the design can be described, but its intended statistical comparisons cannot be reconstructed from the posted information.

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: There were 8 screen failures

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: 2 week radiographic imaging

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.

Population: 16 patients in both arms with demographic characteristics as outlined below

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Defect Healing - Plain Radiography, measured over 2 weeks postop.

The primary outcome is titled “Number of Participants With Defect Healing - Plain Radiography,” uses participants as its unit, and has a timeframe of two weeks after surgery. Its accompanying description says that biplanar radiography was used to demonstrate adequate hardware placement and defect alignment in participants in both trial arms. These are the specific imaging purposes described in the registry record. It does not give a radiographic threshold, a scoring rule, or an explicit definition of what counted as defect healing; those details are not reported in the registry record.

A binary participant outcome records whether each participant meets a defined condition, rather than measuring a continuous quantity such as a length or score. In general, a clearly specified binary endpoint can make it possible to count participants meeting the criterion in each arm. The clinical interpretation depends on the criterion and the assessment time. In this trial, the two-week timeframe is an early assessment. It should not be read as a measure of later healing, sustained alignment, function, or limb preservation unless those outcomes are separately defined and reported.

The posted primary results are counts: 9 participants in the Masquelet arm and 7 in the titanium cage arm. The registry labels these values as the number with the outcome. The relationship between the title’s wording about defect healing and the description of hardware placement and alignment is not clarified in the registry record. That ambiguity limits what can be concluded about the clinical meaning of the endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What kind of outcome is reported?

The primary outcome is a participant count: the registry records the number of participants with the stated radiographic outcome. General statistical explanation: binary outcomes classify each participant into one of two categories, such as meeting or not meeting a prespecified criterion. A count can be reported for each arm, but interpretation depends on knowing the criterion and the number assessed. Here, the arm counts and outcome counts are posted, while the registry does not give a detailed radiographic rule for classifying defect healing.

The outcome title refers to defect healing, while its description emphasizes adequate hardware placement and defect alignment on biplanar radiography at two weeks after surgery. Those concepts may be clinically related, but the registry record does not define their relationship. Statistical interpretation cannot resolve an ambiguity in the endpoint definition. The result should therefore be described using the registry’s stated wording and timeframe, without treating it as a direct measure of later healing.

What does a comparison require?

General statistical explanation: a two-arm comparison usually expresses the difference between groups using an effect measure suited to the outcome. For a binary outcome, possible approaches include comparing event probabilities or using another prespecified measure of relative or absolute difference. The measure must be accompanied by an account of its uncertainty to show how precisely it is estimated. The registry record does not report a comparative effect estimate or confidence interval for the primary outcome. It states that a two-arm comparison for the binary endpoint could not be estimated.

A confidence interval is a statistical summary of uncertainty around an estimate, calculated using an analysis method and its assumptions. It is not a guarantee that a particular range contains the true value. Because no interval or estimate is reported, the magnitude and precision of a between-arm comparison cannot be evaluated from the registry’s primary results. The arm-specific counts remain useful as descriptive information, but they do not substitute for a comparative analysis.

Why does sample size matter?

General statistical explanation: the amount of information available affects the precision of estimated effects. With fewer observations, an estimate will commonly be less precise, and a small number of outcome classifications can have a substantial influence on the estimate. This is a reason to interpret the counts cautiously, not a basis for calculating or assuming an unreported effect. The registry lists 16 participants enrolled, with 9 in the Masquelet arm and 7 in the titanium cage arm. The sample-size justification and any planned detectable effect are not reported in the registry record.

Planning calculations and observed results answer different questions. A planning illustration describes a hypothetical design under stated assumptions; it is not an observed result and is not evidence that the trial had a particular probability of detecting an effect. The registry record does not report its own sample-size justification. No claim about observed power or the adequacy of the planned sample size can be made from the posted information.

What can be learned from randomization?

General statistical explanation: randomization assigns participants to groups using a chance-based process and is intended to reduce systematic differences at allocation. It does not ensure that every baseline characteristic is identical, particularly in a small trial. Details about the randomization sequence and allocation concealment are not reported in the registry record. The design is listed as randomized and parallel, with no masking. No further description of the allocation process is available in the registry record.

Masking concerns whether participants, clinicians, or outcome assessors know the assigned group. The record lists masking as none, but does not specify which parties were aware of allocation or how radiographs were assessed. That information is not reported in the registry record. For an imaging endpoint, assessor procedures can be relevant to how consistently an outcome is classified; no additional assessor information is posted.

What does the record say about the analysis?

The registry reports no analysis results for the trial in the information available. It does not give the planned statistical model, hypothesis, significance threshold, covariates, or approach to missing outcomes. Each of these details is not reported in the registry record. Without an analysis plan or comparative estimate, readers cannot verify whether a particular statistical method was used or how its assumptions were handled.

General statistical explanation: model choice should reflect the outcome type, study design, and question. A binary participant outcome requires an approach appropriate to counts or probabilities; the analysis should also specify how incomplete observations and the randomized groups are handled. These are general principles, not a description of methods used in this trial. The record’s statement that a two-arm binary comparison could not be estimated means no such comparative result is available to interpret.

The record also does not report interim analyses, stopping rules, or a multiplicity strategy. These elements should not be inferred from the randomized design or the list of outcomes. Their status is not reported in the registry record. The statistical information that can be described is thus restricted to the design label, enrolled and arm counts, endpoint wording and timing, and posted outcome counts.

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("Masquelet Defect Reconstruction", "Titanium Cage Reconstruction")
n      <- c(9, 7)
events <- c(9, 7)
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  Masquelet Defect Reconstruction  9  9  100.0  66.4  100.0  70.1  100.0  69.2  99.7
ARM  Titanium Cage Reconstruction  7  7  100.0  59.0  100.0  64.6  100.0  63.1  99.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 27 outcome measures and 19 posted secondary outcomes. Named secondary outcomes include repeated Brief Pain Inventory assessments and an overall functional outcome score. The record does not explain how repeated entries relate to distinct measurements or time points, nor does it give a complete account of the planned outcome hierarchy. Those details are not reported in the registry record.

General statistical explanation: when a study evaluates multiple outcomes or multiple analyses, the chance of finding an apparently notable result by chance can increase if each result is tested separately. A prespecified primary endpoint, a clear hierarchy, or an adjustment strategy can help define how results should be interpreted. The registry record does not report a multiplicity strategy or an analysis plan for the listed outcomes. No adjustment method should be assumed.

The record does not provide a missing-data plan, analysis population definition, or participant flow for the primary outcome. It therefore does not establish whether results were analyzed by randomized assignment, among participants with observed assessments, or using another population definition. These details are not reported in the registry record.

The safety counts use different at-risk numbers from the arm enrollment counts: 8 participants are listed at risk for safety outcomes in the Masquelet arm and 7 in the titanium cage arm. The reasons for the difference between the Masquelet arm count and its safety at-risk count are not reported in the registry record. This difference should not be attributed to withdrawal, missing data, or any other cause without further information. The record also does not explain how missing outcome assessments, if any, were handled.

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.

Most frequent serious adverse event terms

Serious events with the highest arm-level rate: 6 months post reconstructive surgery (14.3%), 3 weeks post reconstructive surgery (14.3%), 18 months post reconstructive surgery (14.3%).

The safety record lists 8 participants at risk in the Masquelet arm and 7 in the titanium cage arm. It reports 0 participants affected by serious adverse events in the Masquelet arm and 3 in the titanium cage arm. For other adverse events, it reports 0 affected participants in each arm. Deaths are recorded as 0 in both arms. These are the posted counts, not a statistical comparison.

The serious-event entries also list “6 months post reconstructive surgery,” “3 weeks post reconstructive surgery,” and “18 months post reconstructive surgery,” each with 14.3%. The registry information does not identify the nature of an adverse event in those labels or explain how these entries relate to the arm-level counts. The timing labels should not be treated as descriptions of event types.

General statistical explanation: safety counts are interpreted in relation to the number of participants at risk and the period and methods of observation. Differences in the number at risk, event definitions, or follow-up can affect interpretation. The registry record does not report enough detail to determine those factors or to estimate comparative safety effects. The serious-event counts should therefore be read as descriptive registry reports, not as evidence that one procedure is safer or less safe.

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

The registry describes a randomized, parallel-group comparison of Masquelet defect reconstruction and titanium cage reconstruction for segmental long bone defects. The primary outcome is a participant count based on plain radiography at two weeks after surgery. The posted count is 9 in the Masquelet arm and 7 in the titanium cage arm. The record’s stated arm sizes are also 9 and 7, respectively. No comparative estimate or uncertainty interval is reported, and a two-arm comparison for the binary endpoint could not be estimated.

These results do not establish that the approaches differ on the primary outcome. They also do not establish equivalence or show that the approaches have identical outcomes. A failure to report an estimable comparison is not the same as a statistical finding of no difference. The record does not include a comparative statistical result that would support either conclusion.

The endpoint’s meaning is limited by its early timeframe and by an unresolved distinction in the registry wording: the outcome title refers to defect healing, while the description specifies imaging to demonstrate hardware placement and defect alignment. The record does not state that this assessment measures later healing, pain, function, or long-term limb outcomes. Secondary outcomes include pain and functional measures, but their numerical results are not reported in the registry information.

The safety entries report serious adverse events affecting 0 participants in the Masquelet arm and 3 in the titanium cage arm, with at-risk counts of 8 and 7, respectively. Other adverse events are reported for 0 participants in each arm, and deaths are recorded as 0 in each. These descriptive figures do not by themselves provide a controlled estimate of comparative risk; information about event definitions, follow-up, and analysis is not reported in the registry record.

More broadly, the posted record does not provide the analysis plan, sample-size justification, missing-data methods, or a full account of participant flow. Those omissions limit assessment of the precision and robustness of results. The record supports a description of the trial and its posted counts, but not a definitive claim about comparative efficacy or safety.

14. Why This Trial Matters Statistically

This trial is statistically notable because it places two surgical reconstruction approaches in a randomized, parallel-group design, while the reported primary outcome is an early binary radiographic assessment. The registry identifies 16 enrolled participants, with 9 assigned to Masquelet defect reconstruction and 7 to titanium cage reconstruction. Those numbers frame how much outcome information is available, but the registry does not report a sample-size justification or a comparative estimate.

For clinical research, a result is more informative when the endpoint is defined clearly, the analysis is prespecified, and the estimate is accompanied by a measure of uncertainty. Here, the primary outcome title refers to defect healing, while the description focuses on adequate hardware placement and defect alignment at two weeks after surgery. The record does not explain the operational definition or resolve this difference in emphasis. This illustrates why endpoint labels and assessment details both matter when interpreting trial results.

The registry also lists pain and functional outcomes, but does not provide their numerical results in the posted information. With multiple outcomes, an analysis plan is important for distinguishing primary questions from additional assessments and for explaining how multiple statistical comparisons are handled. Such a plan is not reported in the registry record.

Finally, the primary arm counts are descriptive and no comparative statistical result is reported. This distinguishes the existence of randomized groups and observed counts from the availability of a precise estimate of their difference. The trial therefore offers a useful example of how registry design information, endpoint wording, and analysis reporting each shape what can be concluded from a clinical study record.

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’s primary endpoint has an ambiguity that affects interpretation: its title describes participants with defect healing, while its description specifies biplanar radiography to demonstrate hardware placement and defect alignment at two weeks after surgery. The record does not provide a threshold, assessment rubric, or explanation of how these imaging findings correspond to the endpoint label. The primary outcome should therefore be interpreted narrowly and with that uncertainty in mind.

The trial enrolled 16 participants, with 9 listed in the Masquelet arm and 7 in the titanium cage arm. The registry does not report a sample-size justification or a planned detectable effect. It also does not report a comparative estimate, confidence interval, or p-value for the primary endpoint, and notes that a two-arm comparison for the binary endpoint could not be estimated. The posted counts cannot answer the comparative question on their own.

The safety record lists 8 participants at risk in the Masquelet arm and 7 in the titanium cage arm. The reason the Masquelet safety count differs from the arm count is not reported in the registry record. The record does not provide event definitions, detailed follow-up information, or an explanation of the serious-event timing entries. These omissions limit interpretation of the safety counts.

Further limitations include the absence of a reported statistical analysis plan, analysis population, missing-data approach, participant flow, and multiplicity strategy. Masking is listed as none, but the record does not specify who knew the assignment or how radiographic outcomes were assessed. These details are not reported in the registry record. The posted secondary outcomes do not include numerical results in the information summarized by the record, restricting assessment of pain and functional outcomes.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Comparative Efficacy of the Masquelet Versus Titanium Mesh Cage Techniques for the Treatment of Large Long Bone Defects — NCT02015390.
• Registry record last updated 2021-06-28; status last verified 2021-06.
• Results first posted 2021-06-21.
• 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

NCT02015390 is a completed randomized, parallel-group trial with no masking, listed for segmental long bone defects. The registry reports an enrollment of 16, with 9 participants in the Masquelet Defect Reconstruction arm and 7 in the Titanium Cage Reconstruction arm. The start date is 2013-10-01, and the primary completion date is 2019-09-30.

The primary outcome is the number of participants with defect healing by plain radiography at two weeks after surgery. The registry describes the imaging assessment as demonstrating adequate hardware placement and defect alignment. The posted outcome counts are 9 for Masquelet reconstruction and 7 for titanium cage reconstruction. A comparative estimate and confidence interval are not reported, and a two-arm comparison for the binary endpoint could not be estimated.

Safety counts list serious adverse events affecting 0 participants in the Masquelet arm and 3 in the titanium cage arm, other adverse events affecting 0 in each arm, and 0 deaths in each arm. The registry does not report a statistical analysis plan, sample-size justification, or the methods used to address missing data and multiplicity.