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 Use of Biosimilar Nivestim® to Prevent Chemo-induced Neutropenia. Real Life Study (NCT02454530), a registered phase not stated study. The primary outcome is Percentage of Participants by Level of Importance of Factors Determining the Use of Nivestim.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02454530 |
| Phase | Phase not stated |
| Condition | Chemotherapy-Induced Neutropenia |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 1160 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
The study’s stated focus is patients receiving chemotherapy for solid tumours and Nivestim® for primary or secondary prophylaxis. The primary-outcome analysis population included participants who received at least one dose of Nivestim® and had no major protocol deviation. The registry record reports an enrollment of 1160.
Intervention
Nivestim®, a G-CSF biosimilar, was used as prophylaxis for chemotherapy-induced neutropenia. The registry describes its use in real-life conditions rather than specifying a treatment regimen for comparison.
Comparator
The study distinguishes adjuvant from metastatic chemotherapy context. These are clinical settings, not like-for-like control arms. The registry record does not state an allocation method, and the record cautions that this is not a randomized comparison.
Primary question
At the inclusion visit (Week 1), how important were different factors in determining Nivestim® use? Listed factors include participant characteristics, medical history, life expectancy, and occupational or family activity. The primary outcome describes the percentages of participants assigned to importance categories; those percentages are not reported in the registry record.
The registry’s stated aim is descriptive: to characterize the determinants of Nivestim® use in primary or secondary prophylaxis and to consider whether use differs by chemotherapy context. It also lists outcomes concerning chemotherapy continuation, discontinuation, dose reduction, delay, protocol change, neutropenia, and febrile neutropenia. These outcomes could help describe treatment-course events in the recorded population, but their numerical results are not reported in the registry record.
3. Trial Design
The record describes a real-life study of Nivestim® use among patients receiving chemotherapy for solid tumours. Its focus is prophylactic use in adjuvant and metastatic settings. The registry lists Nivestim® as the intervention and describes one arm of cancer patients treated with Nivestim®. It does not provide an allocation method. The record explicitly cautions that this is not a randomized comparison and that the arms are not a like-for-like control comparison.
Enrollment is listed as 1160. The study start is recorded as 2014-09, and primary completion as 2016-10-18. The status is TERMINATED. The registry record does not state why the study was terminated, so the status alone does not establish whether termination was planned or what effect it had on data collection or analysis.
The primary outcome is the percentage of participants categorized by how important various factors were in determining Nivestim® use. The timeframe is the inclusion visit (Week 1). The primary-outcome population is described as participants who received at least one dose of Nivestim® and had no major protocol deviation; the record notes that the number analyzed refers to participants evaluable for the specified categories.
Seventeen outcome measures are listed, including the primary measure and secondary measures. No analyses are listed in the registry record. Numerical results for the primary outcome and the listed secondary outcomes are not reported in the registry record. The record does not report the full protocol, a statistical analysis plan, an allocation procedure, or a sample-size justification. The study’s descriptive aim and the stated limitations on comparison therefore matter when interpreting any observed differences between clinical settings.
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 | Adjuvant Chemotherapy | Metastatic Chemotherapy |
|---|---|---|
| STARTED | 552 | 608 |
| COMPLETED | 482 | 481 |
| NOT COMPLETED | 70 | 127 |
Reasons for not completing this period:
| Reason | Adjuvant Chemotherapy | Metastatic Chemotherapy |
|---|---|---|
| Other | 8 | 13 |
| Missing information | 56 | 69 |
| Death | 2 | 41 |
| Lost to Follow-up | 4 | 4 |
Analysis population for the primary outcome, as defined in the registry: Analysis population included all participants who received at least one dose of Nivestim and did not had a major deviation from the protocol. Here 'Number analyzed' signifies number of participants evaluable for specified categories.
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 participants who received at least one dose of Nivestim® and did not had a major deviation from the protocol.
| Characteristic | Unit | Adjuvant Chemotherapy | Metastatic Chemotherapy | Total |
|---|---|---|---|---|
| Participants analysed | 551 | 603 | 1154 | |
| Age, Continuous | years | 60.3 (SD 12.1) | 65.0 (SD 11.0) | 62.7 (SD 11.8) |
| Sex/Gender, Customized: Female | Participants | 399 | 324 | 723 |
| Sex/Gender, Customized: Male | Participants | 149 | 277 | 426 |
| Sex/Gender, Customized: Missing | Participants | 3 | 2 | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants by Level of Importance of Factors Determining the Use of Nivestim, measured over Inclusion visit (Week 1).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is the percentage of participants assigned to each level of importance for factors that determined Nivestim® use. The listed levels are very important, important, relatively important, not important, and not applicable. Factors include sex, young or elderly participant status, past infection, comorbidities, life expectancy, past febrile or severe neutropenia, occupational activity, family activity, and other important criteria. The stated timeframe is the inclusion visit (Week 1).
This outcome is used to describe clinical decision-making: it records which considerations were judged relevant to Nivestim® use and how strongly they were rated. It is not itself a measure of neutropenia prevention, chemotherapy delivery, or a treatment effect. The outcome is also not a randomized comparison of Nivestim® against a control treatment. It characterizes reported importance within the analysis population.
General statistical context: a percentage expresses the share of an eligible group assigned to a category. Its interpretation depends on knowing the denominator for that category and how missing or unevaluable responses were handled. The registry record defines the analysis population and says that the number analyzed means the number evaluable for a specified category, but category-specific denominators and percentages are not reported in the registry record. Without those results, the distribution of importance ratings cannot be described or compared across settings from the record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a descriptive question: which factors influenced Nivestim® use, and how important were those factors in the study’s chemotherapy contexts? The primary outcome is framed as percentages across importance categories at the inclusion visit (Week 1). The record lists the factors and categories, but it does not report the category percentages or the number evaluable for each category. Consequently, it does not establish which factors were most often considered important, whether ratings differed between adjuvant and metastatic settings, or how precise any such estimates were.
The study reports an enrollment of 1160, but enrollment should not be mistaken for the denominator of every outcome. For the primary outcome, the analysis population was participants who received at least one dose of Nivestim® and had no major protocol deviation. The record further explains that the number analyzed is the number evaluable for the specified category. Category-level denominators are not reported in the registry record, so the enrolled total cannot be substituted for them.
Seventeen outcome measures are listed, including measures concerning chemotherapy continuation and discontinuation, neutropenia-related dose reduction and cycle delay, changes in chemotherapy protocol, and neutropenia or febrile neutropenia. Numerical results for these outcomes are not reported in the registry record. The record lists no analyses. Thus, the listed outcomes describe what the study intended to assess, not what was observed in the posted results available in the registry record.
The safety information is reported by chemotherapy context. In the adjuvant group, 3 of 552 participants at risk were affected in the serious adverse-event category, and 78 of 552 were affected in the other adverse-event category. In the metastatic group, the corresponding figures were 2 of 608 and 67 of 608. Deaths are listed as 2 and 41, respectively. These are descriptive counts. The record does not report a randomized comparison, event attribution, or details sufficient to interpret the death counts as treatment-related.
The record explicitly warns that the groups are not a like-for-like control comparison. Any contrast between adjuvant and metastatic counts may reflect differences in the underlying clinical contexts, participants, or other factors; the registry record does not report adjustment methods that would address those differences. The counts therefore describe what was recorded in each group, not a causal effect of Nivestim® or of chemotherapy context.
Precision cannot be evaluated from the primary outcome because estimates and confidence intervals are not reported in the registry record. General statistical context: confidence intervals communicate the uncertainty around an estimate, conditional on the analysis and assumptions used. No such interval is reported for the primary percentages in the record. The study status is TERMINATED, but the registry record does not state the reason or its implications for the completeness of the results.
9. Statistical Methods Explained
The primary outcome is categorical: participants are classified by the importance assigned to factors influencing Nivestim® use. General statistical context: categorical outcomes summarize observations in distinct groups rather than on a continuous measurement scale. Percentages can make category distributions easier to interpret, but they require a clearly defined denominator. In this study, the registry record says that the primary analysis included participants receiving at least one Nivestim® dose without a major protocol deviation, and that the number analyzed is the number evaluable for the specified category. It does not report the actual category-specific counts or percentages.
What does a percentage describe?
General statistical context: a percentage describes the proportion of eligible observations that fall into a category. It does not, by itself, explain why an observation falls there. For the primary outcome, a percentage in an importance category would describe the distribution of ratings among evaluable participants. It would not demonstrate that the factor caused Nivestim® use or that the medicine changed a clinical outcome. Because the category results are not reported in the registry record, no such distribution can be characterized from the record.
The listed response categories—very important, important, relatively important, not important, and not applicable—are ordered in terms of importance, with a separate category for not applicable. General statistical context: ordered categories preserve rank information, but the distance between categories is not necessarily equal. Treating these categories as if the step from one level to the next had a uniform numeric meaning would require a justified analysis plan. Such a plan is not reported in the registry record. The record also does not explain how a participant’s response was obtained or how multiple factors were summarized.
How should the study groups be read?
The record distinguishes adjuvant and metastatic chemotherapy contexts. General statistical context: a comparison between groups is most interpretable when the groups are comparable in factors that affect the outcome, or when suitable methods account for relevant differences. Random allocation is one way to support comparability, but the registry record states that this is not a randomized comparison and that the arms are not a like-for-like control comparison. It does not report an allocation procedure or adjusted comparative analyses. The group-specific counts should therefore be read descriptively, not as estimates of a causal contrast.
The record’s primary question concerns factors determining Nivestim® use, while the safety information and secondary outcomes concern events during follow-up. These are different types of questions. A description of decision factors does not itself estimate a treatment effect, and a count of events does not establish that an event was caused by treatment. General statistical context: causal interpretation generally depends on design, measurement, and analysis assumptions that support comparison. The registry record does not report enough methodological detail to assess such assumptions for a causal analysis.
What do uncertainty and statistical significance mean?
General statistical context: an estimate is a summary of observed data, while a confidence interval describes a range of values compatible with the data under a stated statistical procedure and assumptions. A narrower interval generally indicates greater precision; a wider interval indicates more uncertainty. A significance test addresses compatibility with a specified null hypothesis, but it does not measure clinical importance or prove causation. The registry record does not report confidence intervals or significance tests for the primary outcome, and no analyses are listed. Therefore, precision and statistical significance for the primary percentages cannot be assessed from the registry record.
General statistical context: sparse event counts can produce substantial uncertainty, especially when an outcome is uncommon. The safety information gives counts and numbers at risk for the serious and other adverse-event categories, but it does not provide confidence intervals or inferential comparisons. The figures should be treated as observed descriptive counts. They do not, without more detail, establish differences between the chemotherapy settings or attribute events to Nivestim®.
How do planning illustrations differ from trial results?
The registry record describes planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is specified as a fraction of the observed effect. For time-to-event outcomes, the illustrations concern the total number of events; for other outcomes, they concern participants per arm. These are planning illustrations, not the study’s reported enrollment rationale and not observed power. The registry record does not report the study’s own sample-size justification, nor does it report the illustration values in the available information.
General statistical context: a sample-size calculation is conditional on its assumptions, including the target effect, variability or event rate, allocation, and statistical method. A planning illustration can show what a hypothetical design might require under those assumptions, but it cannot reveal the chance that a completed study detected an effect after seeing its results. Calling such a calculation observed power would confuse a planning exercise with an analysis of the study data. No new calculation or outcome estimate can be inferred from the information in the registry record.
The registry lists 17 outcome measures and zero analyses. General statistical context: a registry outcome list and an analysis plan are not interchangeable. A prespecified analysis plan can clarify outcome definitions, populations, handling of missing observations, and any adjustments for multiple testing. The analysis plan is not reported in the registry record. As a result, details about model choice, covariate adjustment, and inferential procedures remain unknown. The outcome descriptions and population note support a limited understanding of what was intended to be measured, but not a reconstruction of the complete statistical methods.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 17 outcome measures, including the primary measure and several secondary measures. It lists zero analyses. A statistical analysis plan is not reported in the registry record, so it is not possible to determine whether outcomes were assigned a testing hierarchy, whether any adjustment for multiple comparisons was planned, or whether the analyses were descriptive only. General statistical context: when many hypotheses are tested, the chance of at least one apparently unusual result can increase. Methods for controlling that issue depend on the analysis objectives and plan; the registry record does not report which, if any, were used.
The primary-outcome population is described as participants who received at least one dose of Nivestim® and had no major protocol deviation. The record says that the number analyzed is the number evaluable for the specified categories. It does not report category-specific denominators or how missing, incomplete, or unevaluable responses were handled. The registry record also does not report whether the listed secondary outcomes used the same analysis population.
General statistical context: missing data can affect both the number of observations contributing to an estimate and the interpretation of that estimate. The impact depends on why information is missing and on the methods used to handle it. Without the missing-data approach and evaluable counts, the completeness and potential selection effects of the primary outcome cannot be assessed. No assumptions about missingness or imputation should be inferred from the registry record.
The record does not report interim analyses, outcome-specific analysis methods, or a multiplicity strategy. The status is TERMINATED, but the reason for termination and any consequences for outcome availability are not reported in the registry record.
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 |
|---|---|---|---|
| Adjuvant Chemotherapy | 3 | 552 | 0.5% |
| Metastatic Chemotherapy | 2 | 608 | 0.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Adjuvant Chemotherapy | 2 of 552 |
| Metastatic Chemotherapy | 41 of 608 |
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 |
|---|---|
| Adjuvant Chemotherapy | 78 of 552 |
| Metastatic Chemotherapy | 67 of 608 |
Most frequent serious adverse event terms
| Event term | Organ system | Adjuvant Chemotherapy | Metastatic Chemotherapy |
|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 1 of 552 | 0 of 608 |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | 0 of 552 | 1 of 608 |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 1 of 552 | 0 of 608 |
| Bronchitis | Infections and infestations | 1 of 552 | 0 of 608 |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | 1 of 552 | 0 of 608 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 0 of 552 | 1 of 608 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Adjuvant Chemotherapy | Metastatic Chemotherapy |
|---|---|---|---|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 44 of 552 | 29 of 608 |
| Nausea | Gastrointestinal disorders | 11 of 552 | 10 of 608 |
| General physical health deterioration | General disorders | 13 of 552 | 6 of 608 |
| Diarrhoea | Gastrointestinal disorders | 4 of 552 | 7 of 608 |
| Alopecia | Skin and subcutaneous tissue disorders | 5 of 552 | 5 of 608 |
| Chest pain | Musculoskeletal and connective tissue disorders | 5 of 552 | 4 of 608 |
| Anaemia | Blood and lymphatic system disorders | 2 of 552 | 7 of 608 |
| Vomiting | Gastrointestinal disorders | 3 of 552 | 4 of 608 |
| Mucite | Gastrointestinal disorders | 2 of 552 | 5 of 608 |
| Headache | Nervous system disorders | 4 of 552 | 3 of 608 |
Serious events with the highest arm-level rate: diarrhoea (0.2%), musculoskeletal pain (0.2%), bronchitis (0.2%), lung disorder (0.2%), lung infiltration (0.2%).
The registry reports serious and other adverse-event counts separately for adjuvant and metastatic chemotherapy groups. In the adjuvant group, 3 of 552 participants at risk were affected in the serious adverse-event category, and 78 of 552 were affected in the other adverse-event category. In the metastatic group, 2 of 608 participants at risk were affected in the serious category, and 67 of 608 in the other category. These figures are descriptive counts within the recorded groups, not results of a randomized comparison.
The registry also lists 2 deaths in the adjuvant group and 41 in the metastatic group. It does not report the cause of death, timing, or relationship to Nivestim® in the information available. The counts should not be interpreted as treatment-attributable deaths or as evidence that chemotherapy context caused a difference.
The listed serious-event terms include Diarrhoea, Musculoskeletal pain, Bronchitis, Lung disorder, Lung infiltration, and Pulmonary embolism, each reported as 0.2%. The registry record does not provide further details about event timing, recurrence, severity beyond the serious classification, or individual attribution. General statistical context: adverse-event summaries are most useful when the event definition, observation period, denominator, and attribution method are clear. The counts alone do not establish causality, comparative safety, or risk for an individual.
12. What the Result Does, and Does Not, Mean
The registry record documents a real-life study intended to describe how Nivestim® was used for primary or secondary prophylaxis during chemotherapy for solid tumours. Its primary outcome concerns the importance assigned to factors influencing use, with attention to adjuvant and metastatic settings. The registry lists the relevant factors and response categories, but does not report the category-level percentages. The primary outcome therefore cannot establish which factors predominated or whether importance ratings differed by setting.
The record lists outcomes related to chemotherapy continuation, discontinuation, dose reduction, cycle delay, protocol change, neutropenia, and febrile neutropenia. Their results are not reported in the registry record. Listing an outcome indicates that it was included in the registry record; it does not show how often it occurred or what its association with Nivestim® was.
The adverse-event counts describe recorded participants in each chemotherapy context. Because the registry cautions that the groups are not a like-for-like control comparison and does not report a randomized allocation, the counts do not establish a causal difference between settings. They also do not determine whether an event was caused by Nivestim®. The record does not report the event attribution or additional detail needed to make that judgment.
General statistical context: descriptive results can characterize what was observed in a defined group, while causal conclusions require stronger support from design and analysis. Even a difference between group summaries may reflect differences in who entered each group or in the clinical context, rather than an effect of the treatment under study. No adjusted comparative analysis is listed in the registry record.
The available information supports a narrow conclusion: the study was registered to describe factors associated with Nivestim® use and to record clinical and safety outcomes by chemotherapy context. Numerical primary and secondary outcome results are not reported in the registry record, and no analyses are listed. Thus, the record does not establish a treatment benefit, lack of benefit, or comparative treatment effect. The status TERMINATED is recorded, but the reason and consequences are not reported.
13. Why This Trial Matters Statistically
This study is statistically relevant because it asks a descriptive question about treatment decisions in routine clinical contexts rather than setting up a randomized treatment comparison. The primary outcome focuses on the importance assigned to factors influencing Nivestim® use. That kind of outcome can help characterize decision-making, but its interpretation depends on clear category definitions, evaluable denominators, and a transparent approach to summarizing responses. The registry record gives the categories and analysis-population description, while category-level results and analysis details are not reported.
The distinction between adjuvant and metastatic settings is central to the record, but the groups are not identified as like-for-like controls. This makes it important to separate description from causal inference. Group-specific counts can document what was recorded, but they do not isolate the effect of treatment or chemotherapy context. The record’s explicit warning about comparison supports cautious interpretation.
The registry also illustrates why outcome registration and reported results are different. Seventeen outcome measures are listed, yet zero analyses are listed and numerical results for the primary and secondary outcomes are not reported in the registry record. Without outcome estimates, denominators, and uncertainty measures, readers cannot evaluate the size or precision of the study’s main descriptive findings.
General statistical context: a well-interpreted clinical study needs alignment between its question, design, defined outcomes, analysis population, and analysis methods. A registry record can clarify some of these elements and reveal where information is absent. In this case, the available record identifies the study’s aims, setting, enrollment, primary outcome, and selected safety counts, while leaving important details about analysis and results unreported. Recognizing that boundary is part of sound statistical interpretation.
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 registry record does not report numerical results for the primary outcome. Although it lists importance categories and factors, it does not provide category percentages or the number evaluable for each category. The primary finding therefore cannot be described quantitatively from the record. Numerical results for the listed secondary outcomes are also not reported.
The record lists enrollment of 1160, but the primary analysis population is narrower: participants who received at least one dose of Nivestim® and had no major protocol deviation. Category-specific denominators are not reported, and the registry does not describe how missing or unevaluable responses were handled. Enrollment cannot be assumed to equal the denominator for an outcome.
The record states that this was not a randomized comparison and that the arms are not a like-for-like control comparison. It does not report an allocation method or adjusted comparative analyses. Adjuvant and metastatic groups therefore should not be treated as equivalent groups suitable for attributing outcome differences to chemotherapy context or Nivestim®.
The analysis plan, sample-size justification, interim analyses, multiplicity approach, and detailed statistical methods are not reported in the registry record. No analyses are listed. The status is TERMINATED, but the reason and implications for follow-up or result completeness are not reported. The safety summary lacks reported details on event attribution and timing; the death counts likewise have no reported cause or relationship to treatment. These limitations restrict interpretation to the descriptive information recorded, rather than conclusions about comparative effects or causation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Use of Biosimilar Nivestim® to Prevent Chemo-induced Neutropenia. Real Life Study — NCT02454530.
- Registry record last updated 2019-06-28; status last verified 2019-03.
- Results first posted 2019-06-28.
- 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
The registry record concerns NCT02454530, titled “Use of Biosimilar Nivestim® to Prevent Chemo-induced Neutropenia. Real Life Study.” Pfizer is listed as sponsor. The study aimed to describe Nivestim® use for primary or secondary prophylaxis among patients receiving chemotherapy for solid tumours, considering adjuvant and metastatic settings.
Enrollment is listed as 1160. The start is recorded as 2014-09, primary completion as 2016-10-18, and status as TERMINATED. The primary outcome measures percentages of participants across importance categories for factors determining Nivestim® use at the inclusion visit (Week 1). Seventeen outcome measures are listed and zero analyses are listed.
Numerical results for the primary and secondary outcomes are not reported in the registry record. The record cautions that the study is not a randomized comparison and its groups are not like-for-like controls. The reason for termination and detailed analysis methods are not reported.