Platelet Transfusion During Neonatal Open Heart Surgery: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Platelet Transfusion During Neonatal Open Heart Surgery (NCT03045068), a randomized phase 4 study. The primary outcome is Amount of Blood Products Transfused.

2. Clinical Question

The trial addresses whether timing platelet transfusion before the end of bypass is associated with a different subsequent blood-product volume. The result is a comparison of group averages, not a direct measure of every aspect of bleeding, coagulation, or recovery. The randomized allocation is relevant to interpreting the group comparison, but the trial was unmasked, and the registry record does not report the analysis population or a fuller statistical plan. The posted result therefore needs to be read alongside its uncertainty and the limitations of registry reporting. The clinical question is specific to neonatal open-heart surgery involving CPB; the registry result alone does not establish how the comparison applies to other populations, procedures, or transfusion approaches.

3. Trial Design

The registry record describes a randomized, parallel-group, open-label Phase 4 trial titled “Platelet Transfusion During Neonatal Open Heart Surgery.” Enrollment is recorded as 46. The posted primary outcome lists 21 participants in the Study Group and 21 in the Control Group. These are the outcome-specific group counts; the record does not explain the difference between the enrollment figure and the counts contributing to this outcome.

The intervention compared platelet transfusion before termination of CPB with no pre-termination intervention in the control group. The study-group description specifies a pre-termination transfusion of 10 mL/kg when the patient was rewarmed to 35°C. It also contains a post-CPB platelet-management description, but that text is truncated in the registry record, so its full regimen cannot be established from the record. The control description specifies post-CPB transfusion of 20 mL/kg at 100 mL/hour until completion.

Masking is listed as none. The registry lists additional interventions involving FFP and cryoprecipitate, PRBC and cell-saver transfusion, and factor concentrate (Bebulin). It does not describe enough detail to establish how those co-interventions were protocolized or how they were incorporated into the primary comparison. The primary outcome is a continuous measure summarized as a mean and standard deviation. Its listed timeframe is 0-72 hours; the outcome description separately specifies blood products from CPB termination to the first 24 hours after surgery. The record does not explain this difference in wording.

The trial is marked completed. Its recorded start and primary completion dates are 2017-04-11 and 2018-10-01. The registry record does not report the original sample-size justification, allocation-concealment procedures, interim analyses, or a detailed statistical analysis plan. Those design details should not be inferred from the posted group summaries.

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

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 Amount of Blood Products Transfused, measured over 0-72 hours.

The primary endpoint is the amount of blood products transfused, measured in milliliters per kilogram and summarized as a group mean with standard deviation. It includes PRBC, FFP, cryoprecipitate, and platelets. The outcome description says that it covers products administered from termination of CPB to the first 24 hours after surgery, while the registry lists the timeframe as 0-72 hours. The registry record does not reconcile those descriptions, so the measure should be understood using both stated definitions rather than assuming they are identical.

This endpoint is used to quantify transfusion exposure over the stated postoperative period. A continuous volume measure allows the group averages to be compared and gives a concise summary of the amount administered. It does not by itself identify which product accounted for a group’s average, how transfusion decisions were made, or whether a particular patient had bleeding or another complication. Individual values can differ from the mean, and the standard deviation describes variability within the group rather than uncertainty about the mean difference.

The posted means are 58.0 mL/kg for the Study Group and 103.0 mL/kg for the Control Group. The posted comparison is a mean difference of -45.0 mL/kg, with the Study Group as the comparison arm. This is a group-level result for the defined measure. Its interpretation depends on the precision of the estimate, the measurement window, and the registry’s incomplete reporting of analysis details.

7. Results Posted in the Registry

• Study Group: mean 58.00 versus 103.00 on control; difference -45.00 (95% CI -81.39 to -8.61), standardized effect d = -0.75; z-test p = 0.015.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Time From End of CPB to Sternal Closure (Chest Approximation)

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Study Group: mean 1.48 versus 1.95 on control; difference -0.47 (95% CI -1.13 to 0.19), standardized effect d = -0.43; z-test p = 0.164.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Study Group and the control. The interval gives the range of effects that remain compatible with the data.

Chest Tube Output

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Study Group: mean 20.00 versus 23.00 on control; difference -3.00 (95% CI -12.02 to 6.02), standardized effect d = -0.20; z-test p = 0.515.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Study Group and the control. The interval gives the range of effects that remain compatible with the data.

Inotropic Support at the Time of Entry to the Pediatric Intensive Care Unit (PICU) as Indicated by Inotrope Score

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Study Group: mean 7.10 versus 5.60 on control; difference 1.50 (95% CI -0.51 to 3.51), standardized effect d = 0.45; z-test p = 0.144.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Study Group and the control. The interval gives the range of effects that remain compatible with the data.

The secondary outcomes add clinical dimensions beyond the primary measure of blood-product volume. The registry lists outcomes concerning time to sternal closure, chest tube output, inotropic support at PICU entry, exposure to factor products, ventilation duration, mortality, CPR events, and arrhythmia. The continuous summaries available for sternal closure, chest tube output, and inotrope score do not all point in the same direction: the Study Group averages are lower for the first two measures and higher for inotrope score. Their posted confidence intervals include zero. These outcomes therefore do not form a consistent directional pattern that independently reinforces the primary result.

The secondary measures are related to the primary question in that they describe aspects of bleeding, support, and postoperative course, but they measure different phenomena. A lower volume of transfused products cannot be assumed to imply a change in chest drainage, closure timing, support needs, or other outcomes. Conversely, an individual secondary measure does not explain the primary difference by itself. The endpoints should be considered as complementary descriptions rather than interchangeable evidence.

Unadjusted secondary comparisons carry less weight for confirmatory interpretation when several outcomes are examined. In general, testing many outcomes increases the chance that at least one result appears notable even if there is no underlying difference for that outcome. The registry record does not report a multiplicity strategy, and the secondary entries list no reported analyses. This makes it difficult to know which outcomes were intended to be confirmatory, how their tests were prioritized, or whether any adjustment was planned. The secondary summaries remain useful descriptively, but they do not replace a prespecified analysis plan or provide independent confirmation of the primary comparison.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Number of Exposures of 4-PCC and Factor 7
• Time From End of CPB to Sternal Closure (Chest Approximation)
• Chest Tube Output
• Inotropic Support at the Time of Entry to the Pediatric Intensive Care Unit (PICU) as Indicated by Inotrope Score
• Length of Mechanical Ventilation
• Number of Participants With Mortality at 30 Days
• Number of Participants With Cardiopulmonary Resuscitation (CPR) Events
• Number of Participants With Arrhythmia

9. Statistical Interpretation

10. Statistical Methods Explained

The primary outcome is continuous: blood-product volume in mL/kg, summarized by a mean and standard deviation in each group. The posted comparison is the difference between the Study Group mean and the Control Group mean. A negative mean difference therefore points toward a lower average in the Study Group. The comparison is reported as -45.0 mL/kg. This direction is a matter of how the difference is defined; it does not by itself establish why the group averages differ.

What does the mean summarize?

In general, the mean is the arithmetic average of a set of observations. It offers a useful summary for a continuous measure, but it can be influenced by unusually high or low observations. Blood-product volumes may vary across participants, and a group average does not show the distribution’s full shape. The registry reports standard deviations of 29.0 mL/kg and 80.0 mL/kg for the Study and Control Groups, respectively. In general, a standard deviation describes the spread of individual observations around a group mean. It is not a confidence interval and should not be read as the uncertainty around the difference between means.

What does the mean difference represent?

The reported mean difference compares the two group averages on the original measurement scale. Here, the estimate is -45.0 mL/kg. The confidence interval accompanies that estimate to express uncertainty about the mean difference under the analysis used. It ranges from -81.39 to -8.605. In general, a confidence interval is a range of values that are reasonably compatible with the data and the model used to calculate it. It does not guarantee that the true difference lies within the interval, and it is not a range of individual participant outcomes.

The interval excludes zero. In general, zero represents no difference between the group means on this scale. An interval that excludes zero is consistent with a nonzero difference under the corresponding interval procedure. This interpretation depends on the assumptions and analysis used. The registry record does not report enough detail to verify every analytical choice, so the result is best described using the posted estimate and interval rather than attributing it to an unreported model.

How should the p-value be understood?

The primary comparison has an unadjusted p-value of 0.015. In general, a p-value describes how unusual the observed data, or data more extreme, would be if a specified null model and its assumptions were true. It is not a measure of the size of the effect, the probability that a result arose “by chance,” or the probability that the null hypothesis is true. The word “unadjusted” matters because the registry lists multiple outcomes and does not report a multiplicity correction. A single p-value does not account for the broader set of questions examined.

The registry record does not state the exact test or model used for the primary comparison. It also does not report adjustment variables, handling of missing outcome values, or whether the analysis followed randomized assignment. Those details should not be inferred from the fact that the groups were randomized or from the form of the posted summary. The estimates are presented as group means and an unadjusted comparison, which supports a straightforward description but not claims about unreported analytical procedures.

Why can variability affect the comparison?

In general, the precision of a difference in means depends on both the amount of information and the variability in the observations. Greater variability makes it harder to estimate a group mean difference precisely when other features are held constant. The posted standard deviations differ between the groups, so it is useful to keep the within-group spread in mind when reading the interval. The registry also reports a pooled standard deviation of 60.17 for the comparison, but it does not state the role of that quantity in the sponsor’s analysis. A pooled spread is a combined summary and does not mean that the two groups had identical variability.

In general, over-dispersion means that observed variability is greater than a model expects. When a model underestimates variability, standard errors may be too small and intervals may be too narrow. The registry record does not report an over-dispersion assessment or a model for this endpoint, so over-dispersion cannot be claimed as a feature of this trial’s analysis. The broader point is that a statistical model must represent the data’s variability adequately for its uncertainty estimates to be dependable.

What can the secondary comparisons add?

The registry reports continuous secondary outcomes using group means, standard deviations, mean differences, confidence intervals, and unadjusted p-values. The time from CPB end to sternal closure, chest tube output, and inotrope score are different measures and should not be treated as interchangeable with blood-product volume. Their confidence intervals include zero. In general, an interval crossing zero leaves both no difference and differences in either direction compatible with the estimate at the interval’s confidence level. The secondary registry entries list no reported analyses, and a detailed plan for how these outcomes were to be tested is not reported in the registry record.

Because multiple outcomes can generate multiple opportunities for apparently notable results, secondary comparisons need cautious interpretation. In general, multiplicity adjustment can control the chance of false-positive findings across a defined family of tests, while an unadjusted analysis treats each test without such a correction. The registry record does not report an adjustment strategy. This does not erase the descriptive value of the secondary summaries, but it limits the weight that can be placed on isolated p-values.

What are the planning figures?

The sample-size illustrations are based on the observed effect, with two-sided significance level 0.05, equal allocation, and a normal approximation. They consider planning for 100%, 75%, and 50% of the observed effect, and show participants per arm for continuous outcomes. In general, power is the probability that a planned study would detect an effect of a specified size under the assumptions used. The registry record does not report whether these illustrations were used in the original trial design; the original sample-size justification and design assumptions are not reported in the registry record. These planning calculations are not a description of the trial’s observed analysis.

11. Confidence Intervals and Precision

The primary mean difference is -45.0 mL/kg, with a 95% confidence interval from -81.39 to -8.605. The interval excludes zero, which is compatible with a lower mean blood-product volume in the Study Group under the comparison as reported. Its width also shows that the exact size of the difference remains uncertain. Values nearer either end of the interval remain compatible with the data and the interval procedure; the point estimate should not be treated as an exact or guaranteed effect.

Precision refers to how narrowly an estimate is determined. In general, a narrower confidence interval indicates greater precision, while a wider interval indicates more uncertainty about the estimate’s magnitude. Precision is not the same as clinical importance. The interval is expressed in mL/kg, so it retains the primary outcome’s units and communicates uncertainty on the same scale as the mean difference. It does not describe where individual participants’ blood-product volumes fell.

The two group standard deviations, 29.0 mL/kg and 80.0 mL/kg, indicate substantial within-group variation, especially in the Control Group. A standard deviation is a measure of spread among observations, whereas the confidence interval concerns uncertainty about the comparison of group means. They answer different questions and should not be substituted for one another.

The primary p-value is unadjusted, and the record does not report the statistical model, analysis population, or missing-data procedure. The interval and p-value should therefore be read as the posted summary, not as a complete account of all analytical uncertainty. Randomization does not eliminate uncertainty, and an estimate from this trial need not apply unchanged to other settings. The numerical interval expresses sampling uncertainty under the analysis assumptions; it does not incorporate every possible limitation in measurement, conduct, or reporting.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are participants per arm for continuous outcomes, not total events. They are illustrations based on the observed primary effect and use a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The effect to detect is set in turn to 100%, 75%, or 50% of the observed effect. These assumptions describe the planning illustrations; the registry record does not report the original trial’s sample-size justification or design assumptions.

The 80% power column gives the illustrated number of participants per arm for a hypothetical study to have an 80% chance of detecting the chosen effect, under the stated assumptions. The 90% power column gives the corresponding planning number for a 90% chance. These are design targets, not guarantees: actual performance would depend on whether the assumptions and chosen effect reflect the study that is conducted. The figures use participants per arm, so they should not be interpreted as the combined enrollment target.

For planning a smaller effect, more participants are generally needed, because a smaller difference is harder to distinguish from random variation when other assumptions stay the same. The entries for 75% and 50% of the observed effect illustrate that relationship. The sample-size illustrations are not observed power: observed data cannot establish the probability that the completed trial had to detect an effect. They are forward-looking planning calculations based on specified assumptions, not a measure of the trial’s achieved evidence.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = -45.000; pooled standard deviation σ = 60.171.
2. n = 2 × (1.96 + 0.842)² × 60.171² / -45.000² = 28.1, rounded up to 29 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- -45                      # observed difference in means
sigma <- 60.1705908230923                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(3045068)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 29 (80% power), 38 (90% power)
 75% of observed effect: n per arm = 50 (80% power), 67 (90% power)
 50% of observed effect: n per arm = 113 (80% power), 151 (90% power)
power.t.test: 29.1 
Simulated power at n = 29 per arm: 0.797 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary outcome and multiple secondary outcomes. The record does not report a multiplicity adjustment or a hierarchy specifying how the outcomes were to be interpreted. When several outcomes are tested, unadjusted p-values do not account for the number of comparisons. In general, this can increase the chance of at least one apparently notable result across the set of tests. The primary result’s unadjusted p-value should therefore not be treated as if the full outcome family had been accounted for.

The registry identifies the primary outcome as blood-product volume and reports a group comparison. For the secondary outcomes, the entries list no reported analyses. The record does not explain whether this means no formal analyses were performed, whether analyses were performed but not posted, or whether the available summaries are the entirety of the reported analysis. That distinction is not reported in the registry record and should not be guessed.

The record does not report how missing outcome data were handled, whether all randomized participants were included in the primary analysis, or which analysis population was used. It also does not report reasons for any difference between the enrollment entry and the outcome-specific counts. These are important details because excluding participants or handling unavailable measurements in different ways can affect estimates and their uncertainty. No missingness pattern or extent can be inferred from the posted summaries.

Details about the statistical analysis plan, including the exact primary test, any covariate adjustment, interim analyses, and rules for secondary analyses, are not reported in the registry record. The trial’s randomized design is known, but it does not establish that the posted comparison used an intention-to-treat analysis or any other particular analysis population. Interpretation should therefore distinguish what is posted—the group summaries and comparison—from methods that remain undocumented.

15. 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

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cardiopulmonary resuscitation (cpr) event (19.0%).

The registry’s adverse-event record reports serious adverse events affecting 0 participants in the Study Group and 4 in the Control Group; each group has 21 participants listed at risk. Other adverse events are reported for 3 participants in the Study Group and 6 in the Control Group. The record lists 0 deaths in each group. It also identifies Cardiopulmonary Resuscitation (CPR) Event as the top serious adverse-event term, at 19.0%. The record does not specify an arm attribution for that term in the top-term entry.

These are descriptive registry counts. They do not by themselves establish that the groups differ in safety, and the count of participants affected is not necessarily the same as the total number of events. The record does not provide enough information about event definitions, severity grading beyond the serious-event label, timing, relatedness, or adjudication to interpret the counts as a complete safety comparison. The posted figures should be read with those reporting limits in mind, not as proof of benefit or harm.

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

The primary result describes a lower posted mean blood-product volume in the Study Group than in the Control Group over the outcome’s stated period. The estimated mean difference is -45.0 mL/kg, and its 95% confidence interval ranges from -81.39 to -8.605. This is evidence about the trial’s recorded group averages and the uncertainty around their comparison. It is not a guarantee that every participant in the Study Group received less product, nor does it specify how large a difference would be important in clinical practice.

Randomized allocation is a useful feature for comparing assigned groups, but the trial was open-label. The registry record does not report the analysis population, missing-data handling, or detailed statistical plan. The outcome is also based on products administered during a period whose description is not fully consistent: the timeframe is listed as 0-72 hours, while the endpoint description specifies from CPB termination to the first 24 hours after surgery. These reporting details constrain interpretation.

The result does not establish why the averages differ or whether the difference is attributable only to the timing of platelet transfusion. The registry also lists other interventions and does not provide complete detail about their use. The primary volume measure does not, on its own, show that bleeding, recovery, mortality, arrhythmia, or another clinical outcome changed. Secondary results are not uniform in direction, and the registry does not report a multiplicity plan.

The findings should be understood as a comparison from this trial of neonatal open-heart surgery with CPB. They do not establish a result for other patient populations, settings, procedures, or transfusion regimens. Nor do the planning illustrations establish how well the completed trial was powered. The registry record supports a precise description of the posted estimate and its interval, while leaving important questions about design assumptions and analysis methods unanswered.

17. Why This Trial Matters Statistically

This trial raises statistical issues common to clinical research where a randomized comparison is paired with several outcomes and incomplete reporting of analysis details. The primary measure is continuous and has a clear unit, which allows the group contrast to be expressed directly as a mean difference in mL/kg. The interval makes the uncertainty in that difference visible rather than reducing interpretation to a single p-value.

At the same time, the comparison illustrates why effect size, precision, and variability need to be considered together. The posted group means differ, but the estimate has a broad interval and the standard deviations show within-group dispersion. The point estimate alone does not capture this uncertainty. The unadjusted p-value also answers a narrower question than whether the result is important or robust across multiple outcomes.

Endpoint definition matters. The registry’s primary outcome lists a 0-72 hour timeframe while its description refers to termination of CPB through the first 24 hours after surgery. Such differences in timing can affect what observations are included and how a result is understood. Transparent, consistent definitions are essential for interpretation and for reproducing an analysis.

The record also shows why randomized design does not remove the need to report analysis populations, missing-data procedures, multiplicity plans, and protocol-based sample-size rationale. Without those details, readers can describe the posted comparison but cannot fully assess whether the analysis matches the intended design. The sample-size illustrations are useful as hypothetical planning information, but they are distinct from the trial’s original design assumptions and from evidence of achieved power.

18. 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.

Several limitations specific to this registry record affect interpretation. First, the primary endpoint’s timeframe is described as 0-72 hours in one field and as termination of CPB to the first 24 hours after surgery in its measure description. The record does not reconcile those statements. Second, the Study Group’s arm description is truncated during its post-CPB transfusion instructions. The pre-termination instruction is stated, but the complete study-group regimen cannot be reconstructed from the registry record.

The trial is open-label, and blood-product administration is a care measure that can reflect decisions made during treatment. The record does not describe how those decisions were standardized or whether outcome assessment was masked. The enrollment entry is 46, while the primary outcome lists 21 participants in each arm; reasons for the outcome-specific counts are not reported in the registry record. No missing-data approach or analysis population is reported.

The registry does not report the original sample-size justification, the exact primary statistical test, a multiplicity plan, or interim analyses. The secondary entries list no reported analyses, limiting their role in confirming the primary result. The record includes adverse-event counts, but does not provide details about definitions, relatedness, or adjudication. These limits do not invalidate the posted summaries, but they mean that they should not be treated as a complete account of trial conduct or analysis.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: Platelet Transfusion During Neonatal Open Heart Surgery — NCT03045068.
• Registry record last updated 2023-03-08; status last verified 2023-03.
• Results first posted 2023-03-08.
• 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.

22. Record Summary

The completed Phase 4 trial was randomized, parallel-group, and unmasked. Enrollment is recorded as 46. The primary outcome is mean blood-product volume in mL/kg, with posted means of 58.0 for the Study Group and 103.0 for the Control Group. The reported mean difference is -45.0 mL/kg, with a 95% confidence interval from -81.39 to -8.605 and an unadjusted p-value of 0.015.

The primary endpoint description specifies products from CPB termination to the first 24 hours after surgery, while the listed timeframe is 0-72 hours. Secondary outcomes cover several aspects of postoperative care and course; available continuous comparisons have intervals that include zero and do not show a uniform direction. The registry record does not report the original sample-size rationale, analysis population, missing-data handling, or multiplicity strategy. The result is best read as a posted group-average comparison with meaningful uncertainty and limits in registry reporting.