Impact of Oral Nutritional Supplements on Patients Undergoing Haematopoietic Stem Cell Transplantation: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Impact of Oral Nutritional Supplements on Patients Undergoing Haematopoietic Stem Cell Transplantation (NCT05460013), a randomized na study. The primary outcome is The Between-group Change for Body Weight.

2. Clinical Question

The registry frames the question around nutritional outcomes during a period when transplantation-related treatments may make eating difficult. Its background describes symptoms such as sore mouth, mucositis, dysphagia, nausea, vomiting and diarrhoea as possible barriers to oral intake. It also raises interest in gut function and symptoms, including the possible role of fibre-modulated supplements. These statements explain the study rationale; they are not trial findings.

The posted primary results concern body-weight change. They do not by themselves answer every question raised in the background, such as whether a supplement changes gut function, microbiota, or clinical outcomes. Those topics appear among the secondary outcomes, but the registry information summarized for this analysis does not establish a complete pattern of effects across them. The clinical question is therefore best read narrowly: it is a comparison of average weight change between the two randomized groups over the stated period.

3. Trial Design

The study is listed as a completed, randomized, single-masked, parallel-group trial with an enrollment of 100. The registered phase is NA. RenJi Hospital is identified as the sponsor. The start date is 2022-08-01 and the primary completion date is 2025-06-15. The registry record does not report who was masked, how masking was maintained, or additional operational details about recruitment and study conduct.

The intervention listing names Traditional Control and Oral Nutritional Supplement, while the results identify the groups as Placebo Control and Oral Nutritional Intervention. The primary endpoint analysis includes 44 participants in the intervention group and 36 in the control group. The registry states that the number analyzed is consistent with the D28 participant-flow row. The reasons that the analyzed counts differ from enrollment, and the handling of participants without an endpoint measurement, are not reported in the registry record.

The primary outcome is the between-group change in body weight, measured in kilograms, from baseline to 28 days after transplantation. The posted group summaries are means with standard deviations. A two-sided t-test is the reported analysis for the primary comparison. The registry also lists secondary outcomes addressing body composition, physical measures, patient-generated global assessment, microbiota diversity, metabolites, and gut symptoms. The number of outcome measures is 12, with 11 secondary outcomes posted.

Further protocol details, including the original sample-size justification, the intended analysis population, the planned missing-data strategy, and any interim analyses, are not reported in the registry record. The planning illustrations discussed separately are derived from the observed effect and do not describe the trial's original design assumptions.

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: In total, 104 patients were screened, but four participants were excluded from the study before assignment. One patient in the ONS group was discontinued due to non-compliance secondary to psychiatric disease. Three patients in the control group were discontinued due to death, non-compliance or sudden clinical deterioration.

Day of Transplantation (D0)

Reasons for not completing this period:

14 Days of Transplantation (D14)

Reasons for not completing this period:

28 Days of Transplantation (D28)

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The number of participants analyzed is consistent with the numbers provided in the D28 row in the participant flow module.

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: The participants were all Chinese adults recruited from a single centre at Ren Ji Hospital in Shanghai.

6. Primary Endpoint

The registry lists the primary outcome as The Between-group Change for Body Weight, measured over It compares the change in body weight between two timepoints ( at baseline and at 28 days post transplantation) between two arms.

The primary endpoint is the between-group difference in change in body weight. Weight is assessed in kilograms at baseline and 28 days after transplantation, and each group's change over that interval is compared with the other group's change. This targets whether average weight trajectories differed during the specified period, rather than whether the groups had identical weights at baseline or at the final assessment.

Body weight is a direct, continuous measure of nutritional status, and the registry identifies weight loss as a concern in the transplantation setting. The registry rationale notes that treatment-related symptoms may restrict intake, while the endpoint description states that the measure quantifies the difference in weight change between the groups. The outcome is therefore relevant to the study's nutritional question, although body weight alone does not capture every aspect of nutritional status or gut function.

The posted intervention-group mean change is -4.0 kilograms, with a standard deviation of 2.4; the control-group mean change is -3.4 kilograms, with a standard deviation of 2.1. The posted mean difference is -0.6 kilograms. The negative direction reflects a more negative average change in the intervention group for this contrast. These group summaries describe the analyzed participants; they do not by themselves establish a precise or definitive difference between the randomized groups.

The registry record does not report additional details about weighing procedures, the handling of measurements outside the specified timepoints, or whether the endpoint was analyzed under an intention-to-treat principle. Those details matter when assessing how closely the recorded measure corresponds to the intended clinical question.

7. Results Posted in the Registry

• Oral Nutritional Intervention: mean -4.00 versus -3.40 on control; difference -0.60 (95% CI -1.59 to 0.39), standardized effect d = -0.26; z-test p = 0.233.

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.

The Within-group and Between-group Change for Gut Symptoms

• Endpoint type: Binary: the number of participants with the event, compared as proportions.
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Oral Nutritional Intervention: 74.0% (37/50) versus 64.4% (29/45) on control; risk difference 9.6 percentage points (95% CI -9.0 to 28.1), risk ratio 1.15 (0.87–1.51); unadjusted two-proportion z-test p = 0.313.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Oral Nutritional Intervention and the control. The interval gives the range of effects that remain compatible with the data.

The secondary outcomes broaden the study from body weight to other aspects of nutrition, physical function, gut symptoms, and microbiota. The registry lists outcomes for fat-free mass, calf circumference, handgrip, patient-generated subjective global assessment, microbiota diversity, urinary microbiota-derived metabolites, and gut symptoms. Those domains are related to the trial's nutritional and gut-function rationale, but they are not interchangeable with the primary endpoint.

The posted gut-symptom result compares participant counts between the intervention and control groups over the stated collection period. Its reported chi-squared p-value is 0.4, and the recalculated risk-difference interval includes zero. This result does not establish a clear between-group difference. The other secondary outcomes should be interpreted according to their own definitions and estimates; their inclusion does not imply that they support or contradict the primary weight result.

The primary result concerns average change in body weight from baseline to 28 days after transplantation. A secondary measure could show a different pattern without logical contradiction, because body composition, symptoms, functional tests, and microbiota measures capture distinct features and may have different variability and analysis populations. No overall secondary-outcome pattern can be inferred solely from the outcome names. Numeric results for the other secondary measures are not reported in the registry record summarized for this analysis.

Unadjusted secondary results generally carry less confirmatory weight when many outcomes or analyses are examined. Each additional comparison creates more opportunity for a result to appear noteworthy by chance. Multiplicity adjustment, a prespecified hierarchy, and other protections against selective emphasis are not reported in the registry record. Secondary findings are therefore best treated as supportive or exploratory unless the analysis plan establishes otherwise. They can motivate further study, but they do not override the primary estimate or turn an uncertain primary comparison into a conclusive one.

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

• The Within-group Change for Body Weight (Ensure)
• The Within-group Change for Body Weight (Control)
• The Between-group and Within-group Change for Fat-free Mass
• The Between-group and Within-group Change for Calf Circumference
• It Measures the Between-group and Within-group Change for the Handgrip Test.
• The Within-group and Between-group Change for Patient-Generated Subjective Global Assessment
• The Within-group and Between-group Change for Microbiota Diversity
• The Within-group Change for Microbiota-derived Metabolites in Urine

9. Statistical Interpretation

10. Statistical Methods Explained

The registry reports a two-sided t-test for the primary comparison. A t-test evaluates whether the difference between two group means is distinguishable from zero, taking account of the observed variability and the numbers analyzed. “Two-sided” means that departures in either direction are considered: the intervention group could have a more negative or a more positive mean change than control. The registry does not report further details about the exact test implementation, such as whether equal variances were assumed or whether any covariate adjustment was made.

What does the mean difference represent?

The primary effect estimate is the mean difference in change between the intervention and control groups. The posted group means are -4.0 kilograms for intervention and -3.4 kilograms for control, and the posted contrast is -0.6 kilograms. The contrast is expressed in the original measurement units, which makes its scale directly interpretable as a difference in average weight change. It is not the change experienced by every participant: individual responses may vary around each group mean.

The standard deviation describes that variation among participants. It is distinct from the standard error, which describes uncertainty in an estimated mean. The posted group standard deviations are 2.4 and 2.1 kilograms. A pooled standard deviation is also used in the recalculated standardized effect and planning illustration. Standardizing an effect expresses it relative to variability, which can help with planning comparisons, but it is less directly interpretable in kilograms.

How should the p-value be read?

The sponsor's reported primary p-value is 0.24, with the test identified as a two-sided t-test. An unadjusted recalculation from the posted summaries gives p = 0.233. A p-value describes the compatibility of the observed result with a null model under the test's assumptions; it is not the probability that the null hypothesis is true. It also does not measure the size or importance of the estimated difference. The estimate and confidence interval remain essential to interpretation.

“Unadjusted” means the recalculation uses the posted summary statistics without an adjustment for other variables. It is not a replacement for a prespecified analysis using participant-level data. The recalculation is useful as a check on the arithmetic implied by the posted summaries, but the sponsor's analysis is the registry's reported analysis.

Why do confidence intervals add information?

A confidence interval gives a range of estimates reasonably compatible with the observed data under the method used. The primary interval spans values on both sides of zero, so it includes the possibility of no mean difference. Its width conveys that the estimate is not precise enough to isolate a narrow range of effects. A confidence interval does not assign a probability to the true effect after the data are observed, and its interpretation depends on the statistical model and sampling assumptions.

For a continuous endpoint, a mean-based interval is influenced by the variability of the measure and the analysis size. Greater variability or fewer observations generally makes an estimate less precise. This is a general statistical principle; it should not be taken as an explanation of why particular participants lack measurements in this trial. Reasons for missing primary outcome data and methods for handling them are not reported in the registry record.

What does the secondary binary comparison mean?

The registry also reports a gut-symptom outcome as the number of participants, collected from transplantation through 14 days after transplantation. In the posted result, 37 of 50 participants in the intervention group and 29 of 45 in control are counted for the outcome. The reported analysis is a chi-squared test, with p = 0.4. A chi-squared test assesses whether observed counts across categories differ more than would be expected under a no-association model. The registry's result is not enough to infer that symptoms were absent or that the groups were equivalent.

The recalculated risk difference is an unadjusted comparison of the group proportions. For a binary outcome, such a difference is expressed on the probability scale and can be accompanied by a confidence interval. That interval includes zero in this result. Risk ratios and odds ratios are alternative ways to express binary comparisons, but their interpretation differs from an absolute risk difference. These general distinctions do not make any one measure universally preferable; the choice depends on the question and analysis plan.

Why does over-dispersion matter in general?

For count or binary outcomes, the statistical model relies on assumptions about variability. Over-dispersion occurs when the data vary more than the model expects. If ignored, uncertainty can be understated and intervals or tests may appear more decisive than warranted. The registry does not report an over-dispersion assessment for the posted gut-symptom result, so no claim about its presence or absence can be made.

More generally, randomization supports a comparison between assigned groups, but it does not by itself resolve missing-data, adherence, measurement, or multiplicity concerns. The registry record does not describe the analysis population in enough detail to establish the handling of these issues. Interpretation should therefore remain anchored to the outcome, groups, analysis method, and uncertainty that are actually reported.

11. Confidence Intervals and Precision

The recalculated primary confidence interval for the mean difference spans negative and positive values. This is the key precision point: the data are compatible with a range that includes no average difference, rather than locating the effect on only one side of zero. The interval is not so narrow that the estimate can be treated as exact. Its width reflects the uncertainty in the comparison, not a range of individual participants' weight changes.

The primary estimate is -0.6 kilograms, based on group means of -4.0 kilograms and -3.4 kilograms. The observed group standard deviations, 2.4 and 2.1 kilograms, show that individual changes varied around those averages. A mean difference can be estimated even when participant outcomes vary, but variability and the analyzed group sizes affect how precisely that mean contrast is known. The primary analysis includes 44 intervention participants and 36 control participants.

A confidence interval is not a guarantee that the true effect lies within its particular endpoints, nor does inclusion of zero demonstrate equivalence. Equivalence requires a suitable design and a prespecified margin; such a margin is not reported in the registry record. Likewise, a non-significant p-value does not establish that a potentially meaningful difference is absent. The interval should be read as a measure of statistical precision under its assumptions, alongside the point estimate and the test result.

The gut-symptom risk-difference interval also crosses zero. That result similarly leaves uncertainty about the direction and magnitude of the group contrast. Because multiple outcomes are under consideration and adjustment procedures are not reported in the registry record, apparent precision for any individual secondary outcome should not be interpreted as a complete account of uncertainty across the study.

Intervals reflect sampling uncertainty under the statistical method; they do not account automatically for possible bias from missing data, measurement limitations, deviations from assigned intervention, or selective reporting. The registry record does not provide enough information to quantify those additional sources of uncertainty. The most defensible reading is therefore that the primary point estimate is modest in the measurement units, while its interval does not pin down a single direction or magnitude.

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 illustrations express the number of participants per arm, not a total across both arms. They use the observed effect as a starting point and show planning scenarios for detecting the full observed effect, 75% of the observed effect, or 50% of the observed effect. For the primary continuous outcome, the corresponding figures are 225 and 301 participants per arm for the full observed effect at 80% and 90% power; 400 and 535 for 75%; and 899 and 1,204 for 50%.

The assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The 80% power column describes the number per arm in a hypothetical design that would have an 80% chance of detecting the specified effect under those assumptions if that effect were true. The 90% column uses a higher target probability of detecting that effect, and therefore requires more participants per arm in these illustrations. Smaller effects require more participants because they are harder to distinguish from the background variability.

For the gut-symptom binary outcome, the illustrations likewise give participants per arm. They are 366 and 489 for the full observed effect, 664 and 888 for 75%, and 1,524 and 2,039 for 50%, at 80% and 90% power respectively. These figures use the same listed planning assumptions, but apply to the binary endpoint rather than the continuous weight outcome.

These figures are planning illustrations based on the observed effects, not the original trial's sample-size justification. The original design assumptions are not reported in the registry record. This is not observed power: it does not measure the probability that the completed trial detected an effect after seeing its results. The registry's enrollment was 100, whereas the planning scenarios illustrate the larger per-arm sizes associated with the specified effect assumptions.

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 = -0.600; pooled standard deviation σ = 2.270.
2. n = 2 × (1.96 + 0.842)² × 2.270² / -0.600² = 224.7, rounded up to 225 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 <- -0.6                      # observed difference in means
sigma <- 2.27029310205329                     # 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(5460013)
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 = 225 (80% power), 301 (90% power)
 75% of observed effect: n per arm = 400 (80% power), 535 (90% power)
 50% of observed effect: n per arm = 899 (80% power), 1,204 (90% power)
power.t.test: 225.7 
Simulated power at n = 225 per arm: 0.792 (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. Analyses Reported by the Sponsor

The registry reports the sponsor's primary analysis as a two-sided t-test with p = 0.24. The simple unadjusted recalculation from the posted group summaries gives p = 0.233 and a mean difference of -0.6 kilograms. The recalculation uses the posted means, standard deviations, and analysis counts; it is not a participant-level analysis and does not add covariate adjustment.

The two p-values are close, and the registry's discrepancy indicator is false. The recalculation is therefore consistent with the broad message of the sponsor's reported test: the primary comparison does not provide clear evidence of a difference between groups. The sponsor's own analysis takes precedence. The recalculation serves as a transparent check based on the summaries, not as a correction or replacement for the reported analysis.

The registry does not provide the complete sponsor analysis output, such as an adjusted effect estimate or further details of the test implementation. Consequently, the comparison should not be used to infer whether the sponsor used additional information beyond what appears in the posted summaries. In particular, agreement between p-values does not establish that every analysis assumption, population definition, or missing-data decision was identical.

The interpretation should give priority to the estimated contrast and its uncertainty as well as the reported p-value. Neither p-value demonstrates equivalence or proves the absence of an effect. The interval crossing zero means the posted evidence remains compatible with no difference and with effects in either direction.

15. Multiplicity, Missing Data and Analysis Populations

The registry lists 12 outcome measures, including 11 secondary outcomes, and 49 analyses. This creates a broad set of possible statistical comparisons. When many outcomes are tested, the chance of at least one apparently unusual result can rise even if there are no true effects. Statistical approaches such as family-wise error control, false-discovery-rate procedures, or a prespecified hierarchy can address some multiplicity concerns. Which approach, if any, was used is not reported in the registry record.

The reported primary test is two-sided. The registry does not report a detailed statistical analysis plan, the intended ordering of primary and secondary hypotheses, or whether any interim analyses took place. It also does not report how outcome selection was handled when results were posted. Without those details, the primary outcome can still be read as the designated primary measure, but the secondary analyses should not automatically be treated as independent confirmatory tests.

The primary analysis counts are stated to be consistent with the D28 participant-flow row. That correspondence helps connect the reported analysis to participant flow, but it does not explain why the analyzed counts are lower than enrollment. Reasons for missing endpoint data, patterns of missingness, and any imputation or sensitivity analyses are not reported in the registry record. The registry also does not report whether analysis followed randomized assignment regardless of intervention adherence.

Analysis populations matter because exclusions after randomization can affect comparability between groups. Missing-data methods matter because the results may depend on outcomes that were not observed. These are general methodological concerns; the available record does not establish that a particular bias occurred. It also does not describe whether deviations from assigned treatment or other protocol events affected inclusion in the analyses.

Accordingly, the posted test and estimate describe the participants included in the reported analysis, with the documented limitations in analysis detail. The absence of reported multiplicity and missing-data procedures should be treated as uncertainty about the analysis framework, not proof that procedures were omitted. Additional protocol and analysis-plan information would be needed to evaluate those decisions more fully.

16. 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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: death before transplantation (2.1%), death during transplantation (2.1%).

The adverse-event figures use at-risk counts that differ from the primary endpoint analysis counts: 53 participants are listed at risk in the intervention group and 47 in placebo control. The registry reports 0 participants with a serious adverse event in the intervention group and 2 in control. Other adverse events are reported for 1 intervention participant and 0 control participants. Deaths are listed as 0 and 2, respectively. These are counts as recorded, not estimates of comparative risk adjusted for follow-up or other factors.

The registry identifies “Death before transplantation” and “Death during transplantation” as the top serious-event terms, each with a reported percentage of 2.1%. The record does not provide additional event descriptions or enough detail to determine timing, relatedness to the intervention, or the method used to classify events. The listed deaths should not be interpreted as caused by either assigned treatment.

Safety counts are not the same as the primary weight outcome, and their denominators differ from the analyzed primary groups. Small event counts also make simple numerical comparisons imprecise. The registry figures describe the recorded events in the listed at-risk groups; they do not establish a treatment-related safety effect or rule one out.

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

The posted primary result is an estimate of average weight change in the analyzed groups over the period from baseline to 28 days after transplantation. The intervention-group mean change was -4.0 kilograms, compared with -3.4 kilograms in control, and the mean difference was -0.6 kilograms. The reported test gives p = 0.24, and the recalculated confidence interval spans values on both sides of zero. Taken together, the data do not provide a precise, clear between-group difference in this endpoint.

This finding does not show that the groups are equivalent. A result that is not statistically distinguishable from zero can reflect a small effect, considerable variability, limited precision, or some combination of these. Establishing equivalence would require an appropriate design and a prespecified margin, neither of which is reported in the registry record. Nor does the result establish that the supplement has no effect on outcomes other than body weight.

The primary endpoint is a measure of average weight change. It does not fully represent nutritional status, gut function, tolerability, quality of life, or all clinical outcomes. The registry's secondary outcome list reflects some of these broader domains, but the primary result cannot be generalized to them. The posted gut-symptom comparison also does not yield a clear difference, while numerical findings for other listed secondary measures are not reported in the registry record summarized for this analysis.

The trial is described as randomized, which is relevant to interpreting comparisons between assigned groups. Still, details about masking, adherence, missing-data handling, analysis populations, and the original statistical plan are limited or not reported. These gaps constrain assessment of how the analysis was conducted and how broadly the estimates should be applied.

The results therefore support a restrained conclusion about the posted data: the estimated average weight change differed by -0.6 kilograms between groups, but uncertainty remains about the direction and size of the underlying contrast. They do not establish benefit, harm, equivalence, or a definitive answer about nutritional supplementation in all patients undergoing transplantation.

18. Why This Trial Matters Statistically

This trial is statistically interesting because it asks a focused question using a continuous primary endpoint while also addressing several related nutritional and gut-function outcomes. Body-weight change is expressed in kilograms, so the mean contrast is interpretable on the same scale as the measurement. At the same time, the registry's broader outcome set illustrates why a clear distinction between primary and secondary evidence matters.

The primary estimate and p-value demonstrate the difference between an observed direction and a conclusive comparison. The intervention-group average changed by -4.0 kilograms and the control average by -3.4 kilograms, producing a posted contrast of -0.6 kilograms. Yet the p-value is 0.24 and the confidence interval crosses zero. A direction in the point estimate alone is not enough to show that the groups differ reliably.

The planning illustrations also show how sensitive required sample size can be to the effect one aims to detect. Their participant counts rise as the assumed effect becomes smaller, and the target for 90% power is larger than the corresponding target for 80% power. These are general planning relationships applied to the observed effects, not evidence that the trial was designed with those assumptions. The trial's original sample-size justification is not reported in the registry record.

Finally, the registry reports many analyses but does not describe multiplicity control or detailed missing-data methods. These omissions matter for interpretation because a collection of outcomes can generate findings that appear more decisive than the overall evidence warrants, while missing observations can affect the analysis population. The record does not establish that either issue caused bias; it leaves those questions unresolved. Statistical reading therefore depends on keeping the primary estimate, its uncertainty, the broader outcome set, and the limits of the analysis description in view.

19. 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 endpoint analysis includes 44 participants in the intervention group and 36 in control, while total enrollment is 100. The registry says the analysis counts align with the D28 participant-flow row, but reasons for the difference between enrollment and analysis counts are not reported in the registry record. The available information does not describe missingness patterns, imputation, sensitivity analyses, or whether the analysis followed randomized assignment for all participants.

Details about the supplement, control product, dose, timing, adherence, and masking procedures are not reported in the registry record. The study is listed as single masked, but the masked party is not identified. Such details are relevant when interpreting how the intervention was delivered and how outcomes may have been assessed.

The primary result is a mean comparison over the stated time interval. The registry does not report adjusted estimates, baseline covariate handling, or the complete statistical analysis plan. Although the sponsor's t-test and the unadjusted recalculation are consistent in broad interpretation, that agreement does not resolve questions about the analysis population or other analytic choices.

The record lists 49 analyses and multiple outcomes, but multiplicity procedures and any prespecified hierarchy are not reported. Numeric findings for most of the secondary domains are not included in the information summarized for this analysis. This limits assessment of the study's broader nutritional and gut-function questions and makes it difficult to distinguish confirmatory from exploratory findings.

Finally, the registry record does not describe the original sample-size justification, interim analyses, or detailed criteria for event classification. The safety summaries use at-risk counts that differ from the primary analysis counts, and the record does not explain the relationship between those populations. These limitations call for cautious interpretation; they do not, by themselves, show that the trial's conduct or results are biased.

20. Related Tutorials

21. Related Statistical Calculators

22. Sources

• ClinicalTrials.gov: Impact of Oral Nutritional Supplements on Patients Undergoing Haematopoietic Stem Cell Transplantation — NCT05460013.
• Registry record last updated 2026-05-11; status last verified 2026-05.
• Results first posted 2026-05-11.
• 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.

23. Record Summary

The registry record describes a completed, randomized, single-masked, parallel-group trial sponsored by RenJi Hospital, with enrollment of 100 and a registered phase of NA. It compares an oral nutritional intervention with placebo control in patients undergoing haematopoietic stem cell transplantation.

The primary endpoint is between-group change in body weight from baseline to 28 days after transplantation. The posted mean changes are -4.0 kilograms in the intervention group and -3.4 kilograms in control, with a mean difference of -0.6 kilograms. The sponsor reports a two-sided t-test p-value of 0.24; an unadjusted recalculation gives p = 0.233. The interval for the recalculated difference crosses zero, so the estimate remains uncertain.

The registry also lists secondary nutritional, functional, gut-symptom, and microbiota outcomes. It reports a gut-symptom comparison and adverse-event counts, but does not report detailed analysis-plan, missing-data, multiplicity, or original sample-size justification information. The primary result should be read as an imprecise comparison of average weight change, not as proof of equivalence or a conclusion about every outcome.