Parent-Reported Symptom Assessments in Children Taking Multiple Medications: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Parent-Reported Symptom Assessments in Children Taking Multiple Medications (NCT03849066), a registered phase not stated study. The primary outcome is Global Symptom Score.

2. Clinical Question

The study asks how parents can report symptoms in children with special healthcare needs who take multiple medications. Its focus is symptom assessment, including symptoms that may change over time or emerge after a medication is started or its dose is changed. The registry describes a cross-sectional assessment, so the recorded question concerns symptoms at baseline rather than a comparison of symptom changes over time.

The record also lists medication count and the Medication Regimen Complexity Index Score as secondary outcomes. These measures describe medication burden and regimen complexity; they do not create a treatment comparison. The registry does not report a hypothesis comparing PRSA with another assessment method, nor does it report a primary outcome value. Accordingly, the research question is best read as descriptive: it concerns parent-reported symptom burden in the enrolled population, not whether a medication or intervention changes symptoms.

3. Trial Design

The registry classifies this as an “OTHER” study and describes a cross-sectional analysis of children with neurologic impairment and polypharmacy. It lists one PRSA group, with no like-for-like control comparison; allocation is not stated. The record reports enrollment of 136 and a status of COMPLETED. The recorded study start is 2019-04-01, and the primary completion date is 2022-08-01.

Parents completed a proxy symptom assessment. The population description includes children aged 0-17 years, inclusive, who spoke English or Spanish, had special healthcare needs and polypharmacy, and received primary care at a large, hospital-based special health care needs clinic. The brief summary describes the study’s interest in symptoms such as tiredness or rash, and in symptoms that can change or appear after medication changes. That motivation does not change the recorded cross-sectional design: the primary outcome is specified at baseline.

The PQ-MSAS is described as an adapted pediatric-specific version of the validated adult MSAS. The registry says it has versions tailored for age groups 0-3 and 3-18 years, and Spanish versions are available for both. The PRSA intervention is the parent-report assessment itself; the record does not describe a randomized treatment or a separate comparison group.

The record lists three outcome measures, including the primary global symptom score and the secondary outcomes medication count and MRCI score. It reports zero analyses. A statistical analysis plan, detailed recruitment procedures, and any prespecified comparison are not reported in the registry record. Therefore, the design supports a descriptive account of the assessed population and measures, but not a causal treatment-effect interpretation.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Analysis population for the primary outcome, as defined in the registry: We obtained parental written informed consent and enrolled and assessed English-speaking and Spanish-speaking children 0-17 years-old (inclusive) with SNI and polypharmacy (≥5 medications) who received primary care in a large, hospital-based special health care needs clinic.

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: 13 participants enrolled but did not complete the study activities and were excluded from analysis.

6. Primary Endpoint

The registry lists the primary outcome as Global Symptom Score, measured over Baseline.

The primary endpoint is the baseline Global Symptom Score from the PQ-MSAS. The instrument assesses 28 physical and psychological symptoms over the past week. Parents complete the full-proxy version, reporting symptoms on the child’s behalf. The registry describes each symptom item as having four-point scores for frequency, severity, and extent of bother; these components are used to calculate global and individual symptom scores on a 0-100 scale, with 100 representing the worst score.

A global score offers a summary of symptom burden across the instrument’s symptom items. The separate frequency, severity, and bother domains retain distinctions that a single summary can conceal, so the global measure is useful as an overall description rather than a complete account of each child’s symptom experience. Parent proxy reporting is central to the study question: the investigators’ stated rationale is that parents can assess symptoms their child may be experiencing.

The primary outcome is specified as a median with an inter-quartile range at baseline. These statistics describe the center and spread of observed scores without assuming that the distribution is symmetric. However, the registry record does not report the median or inter-quartile range, so the symptom burden observed in the enrolled participants cannot be quantified from the posted results described in the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry specifies a median and inter-quartile range for the primary outcome. These summaries are appropriate descriptive statistics for a score when the distribution may be skewed or contain values that are far from the center. The median marks the center of the ordered observations, while the inter-quartile range describes the span of the middle portion of those observations. In general, the inter-quartile range is less affected by extreme values than the standard deviation. The record does not report the actual summary values, so these methods can be described but not interpreted numerically for this study.

What does a global symptom score represent?

The PQ-MSAS covers 28 physical and psychological symptoms over the past week. Each symptom item includes scores for frequency, severity, and extent of bother, and the registry describes four-point scoring for each domain. A global score combines information from these components into a 0-100 scale, where 100 is the worst score. This creates a compact overall summary, but a global score can obscure differences among individual symptoms or among frequency, severity, and bother. For clinical interpretation, a single overall statistic should therefore be understood as a summary of the instrument’s assessment, not as a full description of every symptom.

Because parents complete the instrument as proxies, the outcome represents parent-reported assessment rather than direct self-report by each child. Proxy reporting can be useful when a child cannot reliably communicate symptoms, and the study specifically centers parents’ role. At the same time, a parent’s report is an assessment made on the child’s behalf. The registry record does not report a comparison between parent and child reports or provide results evaluating agreement between them. Such agreement cannot be inferred from the instrument description alone.

Why is the design important?

A cross-sectional assessment describes measurements collected at a point in the study rather than following a randomized contrast over time. Here, the primary outcome is specified at baseline. This makes a baseline distribution an appropriate descriptive target, but it does not reveal whether symptoms changed after medication initiation or dose adjustment. To assess change, repeated outcome measurements with their timing and an analysis of change would be needed; those details are not reported in the registry record. General methodological principle: a temporal sequence is necessary to describe change, and a suitable comparison is needed to separate a treatment effect from other explanations.

The registry lists one PRSA group and explicitly notes that the arms are not a like-for-like control comparison. Allocation is not stated. Consequently, a between-arm treatment estimate, a randomized contrast, or a causal effect cannot be reconstructed from this design description. Medication count and MRCI are listed as secondary outcomes, but their presence does not make them control groups. They are outcomes describing medication burden or regimen complexity.

What can confidence intervals add?

In general, a confidence interval expresses the statistical uncertainty around an estimated quantity under the assumptions of the analysis. A narrow interval indicates more precision than a wide interval, while neither width nor location alone establishes clinical importance. The registry record does not report a confidence interval for the primary outcome or an inferential analysis from which one could be interpreted. The median and inter-quartile range, if reported, would describe the sample distribution; they are not interchangeable with a confidence interval for a population parameter.

How should planning calculations be read?

The record describes planning illustrations based on a fraction of an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event settings, the stated convention is to express the planning quantity as a total number of events; for other settings, it is participants per arm. These assumptions describe an illustration and are not reported as the study’s own design justification. Since the registered study is cross-sectional and does not describe a two-arm control comparison, those assumptions should not be treated as a description of the observed analysis.

General explanation: a planning sample size depends on the target effect, variability, type I error threshold, allocation, and desired power, among other design choices. A hypothetical calculation based on an observed effect is especially dependent on that effect estimate and should not be interpreted as observed power. The registry does not report a protocol-based sample-size justification, a prespecified target effect, or a statistical plan establishing that the illustration governed enrollment. No sample-size outputs or trial-specific inferential calculations are reported in the registry record.

What does over-dispersion mean in general?

For count outcomes, over-dispersion means that observed variability is greater than a model’s assumptions allow. If ignored, standard errors can be too small and confidence intervals too narrow. This general issue may matter when analyzing symptom counts or event counts, but the registry does not report a count-model analysis or a finding of over-dispersion. The primary endpoint is described as a score summarized by its median and inter-quartile range, not as a count outcome.

Finally, reproducible statistical interpretation requires knowing which participants were included in each analysis, how incomplete assessments were handled, and whether any outcome definitions changed. These details are not reported in the registry record. Without them, the stated endpoint and design can be explained, but the analysis population, missing-data impact, and robustness of any numerical estimate cannot be assessed.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists three outcome measures: the primary Global Symptom Score and the secondary outcomes medication count and Medication Regimen Complexity Index Score. It does not report a multiplicity strategy, such as a hierarchy among secondary outcomes, an adjustment to account for multiple statistical tests, or a distinction between confirmatory and exploratory analyses. With several outcomes, testing each one can increase the chance of at least one apparently notable finding by chance. General statistical practice addresses this by defining the primary question in advance and interpreting secondary results in light of their exploratory or confirmatory status. The registry record does not provide enough analysis detail to determine how that issue was handled.

Missing-data procedures are not reported in the registry record. The record does not state how incomplete parent assessments, missing symptom items, or missing secondary outcome values were handled. It also does not report the amount or pattern of missing outcome data. These details matter because incomplete assessments can alter the analyzed sample and may affect estimates if missingness is related to symptom burden or other participant characteristics. No assumptions about the missingness mechanism or imputation approach can be inferred from the endpoint description.

The analysis populations are not reported in the registry record. It is therefore unclear whether the primary summary was intended to include every enrolled participant, only those with a completed baseline instrument, or another defined group. The record gives enrollment of 136, but does not specify the number included in any outcome analysis. It reports zero analyses, and no participant flow or analysis denominators are reported. These gaps prevent an assessment of how enrollment translated into analyzed observations.

The record also does not report whether the scoring rules allowed partial completion of the PQ-MSAS or how individual symptom items were combined when responses were missing. As a result, neither the handling of incomplete measures nor the sensitivity of the global score to missing items can be evaluated.

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

The study’s central contribution, as registered, is its focus on parent-reported symptom assessment among children with special healthcare needs and polypharmacy. The registry identifies the PQ-MSAS, its past-week recall period, the symptom domains, and the intended baseline summary. This describes what the primary measure is designed to capture and the population in which it was assessed.

The record does not report the primary Global Symptom Score or its inter-quartile range. It therefore does not show the level or variability of symptom burden observed in the enrolled participants. The presence of an outcome definition is not itself an outcome finding. Similarly, the completed status and enrollment figure do not establish that the primary question was answered in a way that can be evaluated from the posted registry information.

The study is not a randomized treatment comparison. It does not establish that a medication caused a symptom, that symptoms changed after a medication adjustment, or that parent reporting improved outcomes. The brief summary describes medication changes as a context in which symptoms may change, but the registered cross-sectional baseline measure cannot determine that temporal relationship. No control comparison is described, and allocation is not stated.

The adverse-event entries list zero for the recorded event counts, but that information should not be treated as evidence that participants had no symptoms. The primary symptom assessment and adverse-event reporting are different records with different purposes. Nor does the entry establish that all symptoms were assessed as adverse events.

General interpretation: descriptive scores can characterize what was reported in the assessed group, while causal conclusions require an appropriate design and analysis that address alternative explanations. Here, the registry description supports an understanding of the measurement approach and intended population, but the missing score estimates and analysis details limit conclusions about the observed symptom burden. The record is not evidence for a treatment benefit or lack of benefit.

12. Why This Trial Matters Statistically

Symptoms can be difficult to assess in children who have complex health needs, particularly when communication is limited or medication regimens are complex. This study makes parent proxy reporting the focus of its measurement question. Statistically, that shifts attention toward the properties and interpretation of the outcome measure: what is being reported, over what recall period, and how multiple symptom domains are combined into a summary.

The PQ-MSAS includes physical and psychological symptoms and scores frequency, severity, and bother. A global score can support a concise description, while item-level scores may carry distinctions obscured by an overall summary. Understanding which level of measurement is being interpreted is important when evaluating any reported result. The registry’s specification of a median and inter-quartile range also signals a descriptive approach centered on the distribution of participant scores rather than a treatment contrast.

The registry’s cross-sectional design illustrates why a carefully framed question matters. Assessing symptoms at baseline can describe reported burden, but it cannot evaluate change following a medication event without longitudinal measurement and an appropriate analysis. Making that distinction prevents a rationale about possible symptom changes from being mistaken for evidence that change was observed or attributable to medication.

The record also highlights an important reporting issue: a detailed endpoint definition is not enough to judge results when estimates, uncertainty, analysis populations, and missing-data handling are not reported. Those elements help readers assess what the data show and how broadly the estimates may apply. In this case, the posted record identifies a relevant measurement approach and target population, while leaving the primary numerical result and its precision unavailable in the registry record.

13. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The primary limitation for interpretation is that the registry record does not report the Global Symptom Score result, its inter-quartile range, or individual symptom estimates. Without these, the magnitude and distribution of parent-reported symptom burden cannot be assessed. The record also reports zero analyses, so no statistical tests, confidence intervals, or adjusted estimates are available for interpretation.

The cross-sectional design and absence of a like-for-like control comparison limit the questions that can be answered. A baseline assessment cannot establish symptom change over time or attribute a symptom to medication initiation or dose adjustment. Allocation is not stated. The record does not describe a longitudinal follow-up analysis, a comparator, or a causal model.

The population is specific: English-speaking and Spanish-speaking children aged 0-17 years, inclusive, with special healthcare needs, neurologic impairment, and polypharmacy, receiving primary care in a large, hospital-based special health care needs clinic. Findings from such a setting, if reported, would not automatically describe children outside those eligibility and care contexts. The registry does not provide enough information to assess representativeness or recruitment-related selection.

Parent proxy reporting is appropriate to the stated study focus, but the record does not report agreement with child self-report or another symptom assessment. The instrument is described, but no information is provided about completion rates, scoring of incomplete items, or measurement performance in this study population. The registry also does not report missing-data methods, analysis populations, or a multiplicity strategy.

Finally, the adverse-event entries do not resolve these limitations or substitute for symptom outcome reporting. They list zero event counts, but the primary symptom measure is not reported. The stated planning assumptions are illustrative and do not establish the study’s own sample-size rationale or statistical plan.

14. Related Tutorials

15. Related Statistical Calculators

16. Sources

• ClinicalTrials.gov: Parent-Reported Symptom Assessments in Children Taking Multiple Medications — NCT03849066.
• Registry record last updated 2023-02-21; status last verified 2023-01.
• Results first posted 2023-02-21.
• 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.

17. Record Summary

NCT03849066, titled “Parent-Reported Symptom Assessments in Children Taking Multiple Medications,” is registered as COMPLETED. The sponsor is University of Colorado, Denver. The study start date is 2019-04-01, primary completion date is 2022-08-01, and enrollment is 136.

The record describes a cross-sectional PRSA assessment among children with neurologic impairment and polypharmacy. The primary outcome is the baseline PQ-MSAS Global Symptom Score, specified as a median with an inter-quartile range. Medication count and MRCI score are listed as secondary outcomes. The registry reports three outcome measures and zero analyses.

The primary score, its dispersion, analysis populations, and missing-data methods are not reported in the registry record. The record does not describe a like-for-like control comparison, and allocation is not stated. Its planning assumptions are described as an illustration rather than the study’s own design justification.