This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study Evaluating the Safety and Effectiveness of Etanercept for the Treatment of Pediatric Psoriasis (NCT01100034), a registered phase not stated study. The primary outcome is Number of Participants With Serious Infections, Opportunistic Infections of Interest and Malignancies: Prospective Participants.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01100034 |
| Phase | Phase not stated |
| Condition | Psoriasis |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 72 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
Children and adolescents aged 17 or younger with plaque psoriasis diagnosed by a dermatologist were eligible. The registry describes prospective participants as those who started etanercept within 30 days before enrollment or at any time after enrollment. Enrollment was 72.
Intervention
The treatment being observed was etanercept. The registry says patients were invited to participate only after a clinical decision had been made to prescribe it. Treatment therefore reflected care in a naturalistic setting rather than assignment by the study.
Comparator
No like-for-like control comparison is described. The registry lists an Etanercept arm and notes that allocation was not stated; the listed arms are not a like-for-like control comparison. The results therefore cannot establish a treatment contrast against an untreated or alternative-treatment group.
Primary question
The primary outcome concerns the number of prospective participants with serious infections, opportunistic infections of interest, and malignancies from baseline up to 5 years. These outcomes were selected to describe important safety events during follow-up. The primary endpoint result is not reported in the registry record, so the observed count for this specific outcome cannot be stated.
The study also describes an aim to assess how well etanercept works in pediatric psoriasis. However, the primary endpoint listed in the registry is a safety count, and no effectiveness result is reported in the registry record. The clinical question addressed by the posted information is therefore narrower than the full study aim: it documents the planned safety assessment and some general adverse-event figures, but does not provide a reported primary endpoint result or a comparative estimate of effectiveness.
3. Trial Design
This was a prospective, observational cohort study of etanercept use in pediatric psoriasis, described in its official title as a long-term post-authorisation safety study in a naturalistic setting. The registry lists 72 enrolled participants and marks the study as completed. The recorded start date is 2010-11-19 and the primary completion date is 2018-09-24.
Participation was offered after a clinical decision to prescribe etanercept had already been made. The study therefore observed treatment in clinical practice rather than assigning treatment through a randomized allocation. The registry lists an Etanercept arm for pediatric patients with plaque psoriasis on etanercept. Allocation is not stated, and the registry warns that the arms are not a like-for-like control comparison. No control group suitable for a direct treatment comparison is described.
The registry summary describes follow-up lasting 5 years, with visits every 3 months for the first 2 years and every 6 months for the next 3 years, or until the end of the study. The primary endpoint timeframe is baseline up to 5 years. The endpoint was planned for prospective participants only, defined as participants who began etanercept within 30 days before enrollment or at any time after enrollment.
The study's stated aim was to assess safety and effectiveness in Europe. The registry lists a secondary outcome concerning duration of subsequent etanercept treatment after completion of the initial treatment period. Detailed eligibility criteria beyond the stated age and diagnosis, the treatment protocol, rules for changing or stopping treatment, and the statistical analysis plan are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Etanercept |
|---|---|
| STARTED | 72 |
| COMPLETED | 29 |
| NOT COMPLETED | 43 |
Reasons for not completing this period:
| Reason | Etanercept |
|---|---|
| Lost to Follow-up | 11 |
| Withdrawal of parent/guardian consent | 1 |
| Withdrawal by Subject | 4 |
| Sponsor decision | 21 |
| Participated in another clinical trial | 1 |
| Participants moved to another hospital | 4 |
| Site closed due to non-responsiveness | 1 |
Analysis population for the primary outcome, as defined in the registry: Prospective Participants: participants who started etanercept within 30 days before the enrollment date or any time after enrollment date. This outcome measure was planned to be analyzed in prospective participants only.
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: Analysis population included all enrolled participants who were documented as having received at least 1 dose of etanercept.
| Characteristic | Unit | Etanercept |
|---|---|---|
| Participants analysed | 72 | |
| Age, Continuous | Years | 14.5 (SD 3.3) |
| Sex: Female, Male: Female | Participants | 35 |
| Sex: Female, Male: Male | Participants | 37 |
| Race/Ethnicity, Customized: White or Caucasian | Participants | 56 |
| Race/Ethnicity, Customized: Black | Participants | 1 |
| Race/Ethnicity, Customized: Other Asian | Participants | 2 |
| Race/Ethnicity, Customized: Other | Participants | 2 |
| Race/Ethnicity, Customized: Unknown | Participants | 11 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Serious Infections, Opportunistic Infections of Interest and Malignancies: Prospective Participants, measured over Baseline up to 5 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of prospective participants with serious infections, opportunistic infections of interest, and malignancies. Its timeframe is baseline up to 5 years, and its unit is participants. Serious infections were defined as infections that were life-threatening, resulted in disability, or required intravenous antibiotic treatment and hospitalisation. The endpoint also includes a protocol-specified range of bacterial, fungal, protozoan, and viral opportunistic infections.
Counting participants with these events is a way to describe the occurrence of safety outcomes that may be clinically important during treatment and follow-up. Combining the named event categories into a participant count gives a broad safety summary, but does not describe when events occurred, how long they lasted, or how many events an affected participant experienced. Those details are not reported in the registry record.
The endpoint was planned for prospective participants only, as defined in the registry. That distinction matters because the enrolled total and the primary endpoint population need not be identical: the record does not state how many participants met the prospective definition. Nor does it report the primary endpoint count. The serious adverse-event figures posted separately should not be treated as the primary endpoint result: they are a broader adverse-event summary, and the record does not establish that those figures correspond exactly to the infections, opportunistic infections, and malignancies specified for the primary outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes an observational study of etanercept use in pediatric psoriasis, with 72 participants enrolled. It gives a planned primary safety outcome and a follow-up timeframe, but does not report the primary endpoint result. In particular, the number of prospective participants with serious infections, opportunistic infections of interest, or malignancies is not reported in the registry record. This prevents a direct reading of the main outcome the study was designed to assess from the posted information.
The adverse-event summary reports 11 participants affected by serious adverse events among 72 at risk, and 44 affected by other adverse events among 72 at risk. These are descriptive counts for the Etanercept group. The record also reports 0 deaths. They do not, by themselves, give a comparison with another treatment, nor are they presented as an estimate of the primary endpoint. The event terms listed as the most frequent serious events include Psoriasis at 6.9%, Drug ineffective at 2.8%, and Angina pectoris, Hepatic steatosis, Chronic tonsillitis, and Cholesteatoma at 1.4% each. These terms and percentages should be read as registry safety reporting, not as a complete account of every event or a causal assessment.
Because treatment was prescribed as part of clinical care before registry participation, the study does not separate treatment effects from differences in the patients who received treatment, their underlying condition, or other aspects of care. A descriptive event count can document what was reported during observation, but without a suitable comparison it cannot determine whether event occurrence was higher or lower because of etanercept.
The registry does not report an effectiveness endpoint result, a treatment-effect estimate, or uncertainty intervals for the outcomes described in the posted safety summary. It is therefore not possible to assess the precision of a treatment contrast or to infer an effectiveness result from these data. The number enrolled and the reported adverse-event counts describe the cohort, but do not substitute for the missing primary endpoint result or a comparative analysis.
The study summary describes long-term follow-up, which is relevant to monitoring safety outcomes that may occur after treatment begins. However, the actual follow-up achieved by participants, losses to follow-up, event timing, and completeness of outcome ascertainment are not reported in the registry record. Without those details, the strength and limits of the event counts as a description of longer-term safety cannot be fully assessed.
9. Statistical Methods Explained
The registry describes a prospective observational cohort. In general, a prospective cohort follows people over time after a defined starting point and records outcomes as they occur. An observational design does not assign treatment randomly: in this study, the decision to prescribe etanercept preceded the invitation to participate. This makes the results useful as a description of treatment in a naturalistic setting, while limiting causal interpretation. Differences between people receiving treatment and any potential comparison group can arise from clinical decisions and patient characteristics, not only from treatment itself.
What does a participant count measure?
The primary endpoint is a count of participants with specified safety events, rather than a continuous measurement such as a score or laboratory value. A count of affected participants tells readers how many people in the defined analysis population experienced an event category. It is different from a count of total event episodes: a person with more than one event may still be counted as an affected participant for a participant-based endpoint. The registry defines the endpoint unit as participants, but does not report whether any participant experienced multiple events or how repeated events were handled in other analyses.
The endpoint is restricted to prospective participants. The registry defines that group by when etanercept was started in relation to enrollment. This population definition matters because people included in an analysis must be identified consistently with the outcome question. The number of enrolled participants is reported as 72, but the size of the prospective endpoint population is not reported in the registry record. Consequently, the denominator for the primary endpoint cannot be established from the posted information.
How should the safety counts be understood?
The adverse-event figures are descriptive: 11 participants were affected by serious adverse events among 72 at risk, and 44 were affected by other adverse events among 72 at risk. The registry also reports 0 deaths. These figures do not estimate a relative treatment effect because no suitable comparator is reported. A participant count also does not communicate event duration, severity beyond the registry classification, time to onset, or recurrence. Those additional details are not reported in the registry record.
In general, an uncertainty interval communicates a range of values compatible with the data and the statistical method used. It helps distinguish an estimate that is relatively precise from one that is uncertain. No confidence intervals for the posted adverse-event counts are reported in the registry record. The primary endpoint result itself is also not reported, so its precision cannot be evaluated from these data.
What can a planning illustration tell readers?
The stated planning assumptions describe illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is defined as a stated fraction of the observed effect. For time-to-event outcomes, the illustration is expressed as a total event count using the Schoenfeld method; other outcomes are expressed as participants per arm. Such calculations are planning illustrations, not observed trial results and not observed power. The registry does not describe them as the study's own design justification. The specific effect estimates and resulting planning counts are not reported in the registry record.
In general, planning calculations depend on assumptions about the effect to detect, allocation, statistical threshold, and analysis method. A change in those assumptions can change the required sample size. Because the underlying observed effect and the resulting counts are not reported in the registry record, the assumptions alone do not establish whether the enrolled cohort was adequate for a particular comparison or endpoint.
Why is over-dispersion relevant in general?
For count outcomes, a simple statistical model may assume that variation in the observed counts follows a specified distribution. Over-dispersion means the data vary more than that model expects. If over-dispersion is present and ignored, uncertainty can be understated and interval estimates or tests can be misleading. This is a general consideration for count data; the registry record does not report whether over-dispersion was assessed or whether a count model was used.
The registry reports no analyses in its analysis count, and no detailed statistical methods, model specifications, adjustment factors, or confidence intervals are reported in the registry record. That limits the ability to judge how the posted descriptive figures were analyzed beyond their stated participant counts and percentages.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 6 outcome measures, including the primary safety outcome and a secondary outcome concerning duration of subsequent etanercept treatment after completion of the initial treatment period. It does not report a multiplicity strategy for interpreting several outcomes or any multiple statistical tests. In general, when many outcomes or comparisons are tested, the chance of at least one apparently notable result arising by chance can increase. A prespecified approach to multiplicity can help readers interpret such results, but the relevant plan is not reported in the registry record. No outcome analyses are listed in the registry analysis count.
Missing-data handling is not reported in the registry record. The record does not state how incomplete follow-up, missed visits, unavailable event information, or withdrawal from observation was handled. This matters in a longitudinal cohort because event ascertainment depends on how much follow-up information is available for each participant. The follow-up schedule is described, but the actual follow-up completeness and the number of participants remaining under observation over time are not reported.
The registry defines the primary endpoint population as prospective participants, but does not report how many of the 72 enrolled participants met that definition or how membership in that population was determined in practice. It also does not report an analysis population for the separate adverse-event counts beyond the group at risk figures. The planned population and the analyzed population should not be assumed to be the same without a reported accounting of participants.
In general, prespecified analysis populations, missing-data rules, and multiplicity methods help clarify how outcome estimates were produced and how much confidence to place in them. Their absence from the registry record means that the analytical handling of these issues cannot be evaluated from the posted information.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Etanercept | 11 | 72 | 15.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Etanercept | 0 of 72 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Etanercept | 44 of 72 |
Most frequent serious adverse event terms
| Event term | Organ system | Etanercept |
|---|---|---|
| Psoriasis | Skin and subcutaneous tissue disorders | 5 of 72 |
| Drug ineffective | General disorders | 2 of 72 |
| Angina pectoris | Cardiac disorders | 1 of 72 |
| Hepatic steatosis | Hepatobiliary disorders | 1 of 72 |
| Chronic tonsillitis | Infections and infestations | 1 of 72 |
| Cholesteatoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 72 |
| Paraesthesia | Nervous system disorders | 1 of 72 |
| Anorexia nervosa | Psychiatric disorders | 1 of 72 |
| Erythema nodosum | Skin and subcutaneous tissue disorders | 1 of 72 |
| Erythrodermic psoriasis | Skin and subcutaneous tissue disorders | 0 of 72 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Etanercept |
|---|---|---|
| Nasopharyngitis | Infections and infestations | 18 of 72 |
| Drug ineffective | General disorders | 17 of 72 |
| Influenza | Infections and infestations | 5 of 72 |
| Ear infection | Infections and infestations | 3 of 72 |
| Respiratory tract infection viral | Infections and infestations | 3 of 72 |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 3 of 72 |
| Abdominal pain upper | Gastrointestinal disorders | 2 of 72 |
| Injection site reaction | General disorders | 2 of 72 |
| Bronchitis | Infections and infestations | 2 of 72 |
| Enterovirus infection | Infections and infestations | 2 of 72 |
Serious events with the highest arm-level rate: psoriasis (6.9%), drug ineffective (2.8%), angina pectoris (1.4%), hepatic steatosis (1.4%), chronic tonsillitis (1.4%).
The safety summary lists 72 participants at risk for serious adverse events, with 11 affected; for other adverse events, 72 were at risk and 44 were affected. The registry reports 0 deaths. These are counts of participants, not counts of all event episodes, and the record does not provide event timing or duration. The percentages listed for selected serious event terms are Psoriasis at 6.9%, Drug ineffective at 2.8%, and Angina pectoris, Hepatic steatosis, Chronic tonsillitis, and Cholesteatoma at 1.4% each.
These figures are descriptive and apply to the Etanercept group. They do not show whether events were caused by treatment, and they do not support a comparison with another treatment group. The listed serious-event terms are not the same as the primary endpoint definition, which specifically concerns serious infections, opportunistic infections of interest, and malignancies. The primary endpoint count is not reported in the registry record. Readers should therefore keep the general adverse-event summary separate from the prespecified primary safety outcome and should not interpret the terms or percentages as a causal assessment.
12. What the Result Does, and Does Not, Mean
The posted information establishes that the registry followed an observational cohort of pediatric psoriasis patients treated with etanercept and recorded descriptive adverse-event figures. It reports 72 enrolled participants, 11 participants affected by serious adverse events among 72 at risk, 44 affected by other adverse events among 72 at risk, and 0 deaths. These figures describe the events recorded for the group; they do not establish why the events occurred.
The information does not establish whether etanercept changed the risk of serious infection, opportunistic infection, malignancy, or another outcome. The primary endpoint count is not reported in the registry record, and no suitable control comparison is described. There is also no reported effectiveness result. Thus, the record does not support a conclusion about comparative safety or effectiveness from the posted results.
In general, an observational cohort can describe what happened among participants receiving a treatment in clinical practice. A comparison intended to estimate a treatment effect needs a defensible basis for comparing outcomes between treatment groups, including attention to differences in who received each treatment. Here, the registry cautions that its arms are not a like-for-like control comparison. A descriptive count should not be read as a treatment-attributable effect.
The absence of a reported primary endpoint result also means that the central safety question stated in the registry cannot be answered from the posted information. The summary does not provide enough detail about follow-up completeness, event timing, or the analysis population to determine how fully the counts represent the planned observation period. No confidence intervals are reported, so precision cannot be judged from the record.
Accordingly, the appropriate interpretation is limited: the registry records a completed observational study and some adverse-event counts, but does not report the primary safety outcome or an effectiveness estimate. The figures should be understood as descriptive reporting, not proof for or against a treatment effect.
13. Why This Trial Matters Statistically
This study is statistically relevant because it addresses safety monitoring in a pediatric population in a naturalistic treatment setting. The registry describes a long-term follow-up plan and identifies serious infections, specified opportunistic infections, and malignancies as the primary outcome. A prospective cohort can be useful for documenting events observed during routine treatment, particularly when the research question concerns safety over time.
The design also makes clear why descriptive safety figures and treatment comparisons answer different questions. Participants entered the registry after a clinical decision to prescribe etanercept. Without a like-for-like control group, event counts can describe what was reported among treated participants but cannot isolate a treatment effect. The registry explicitly warns against treating the listed arms as a valid control comparison.
The record highlights the importance of separating an endpoint's definition from the availability of its result. The primary endpoint is described in detail, including the participant population, timeframe, and event categories, yet its result is not reported in the registry record. The separate adverse-event summary does not fill that gap because it is broader and is not identified as the primary endpoint result.
It also illustrates why a reported denominator and follow-up detail matter. Although enrollment was 72 and the adverse-event figures list 72 at risk, the primary outcome population is prospectively defined and its size is not stated. Actual follow-up completeness and event timing are also not reported. These omissions limit interpretation of long-term event counts and prevent a full assessment of precision.
Finally, the record distinguishes planning assumptions from observed evidence. Sample-size illustrations based on an observed effect are not evidence of achieved power or a guarantee that a study can answer a particular question. Without the underlying effect estimates and calculated planning counts, those assumptions cannot be used to judge the study's ability to detect an effect.
14. 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 most direct limitation is that the primary endpoint result is not reported in the registry record. The outcome was defined as a participant count for serious infections, opportunistic infections of interest, and malignancies, but the posted information does not give the number affected. The separate adverse-event summary cannot be substituted for this result because it is not identified as the primary endpoint analysis.
The observational design and absence of a like-for-like control comparison limit causal interpretation. Treatment was prescribed before participation, and the registry notes that the listed arms are not a control comparison. Differences in patient characteristics or clinical circumstances could influence who received treatment and what outcomes were recorded. A treatment effect cannot be isolated from the descriptive counts.
The registry defines a prospective endpoint population, but does not state how many participants met that definition. It also does not report how many participants completed follow-up, whether follow-up varied between participants, or how missing outcome information was handled. Although a 5-year follow-up plan is described, the actual observation time and event timing are not reported in the registry record.
Detailed statistical methods, confidence intervals, adjustment methods, and a multiplicity plan are not reported in the registry record. No effectiveness result is reported, and the listed secondary outcome has no result in the posted information. The adverse-event figures provide counts and selected percentages, but not enough detail to evaluate event recurrence, duration, or attribution to treatment.
These limitations mean that the record supports a descriptive account of selected adverse-event figures, but not a complete assessment of the planned primary safety endpoint, comparative safety, effectiveness, or the precision of treatment effects.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study Evaluating the Safety and Effectiveness of Etanercept for the Treatment of Pediatric Psoriasis — NCT01100034.
- Registry record last updated 2019-10-08; status last verified 2019-10.
- Results first posted 2019-10-08.
- 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.
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18. Record Summary
NCT01100034 is a completed prospective observational cohort study of etanercept in pediatric psoriasis, described as a post-authorisation safety study in a naturalistic setting. The registry reports 72 enrolled participants and a planned follow-up of 5 years. The primary endpoint concerns prospective participants with serious infections, opportunistic infections of interest, and malignancies from baseline up to 5 years.
The posted safety summary reports 11 participants affected by serious adverse events among 72 at risk, 44 affected by other adverse events among 72 at risk, and 0 deaths. The primary endpoint result and an effectiveness result are not reported in the registry record. No like-for-like control comparison is described, so the adverse-event figures are descriptive rather than estimates of a comparative treatment effect.