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 A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma (NCT00434161), a randomized phase 3 study. The primary outcome is Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00434161 |
| Phase | Phase 3 |
| Condition | Multiple Myeloma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 281 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Swedish Orphan Biovitrum |
| Results posted | Yes |
2. Clinical Question
Population
People with multiple myeloma receiving melphalan followed by autologous peripheral blood stem cell transplantation. The registry record describes the efficacy population as the full analysis set: all randomized participants, analyzed according to randomized treatment assignment.
Intervention
Palifermin before high-dose chemotherapy only: three doses before chemotherapy, with matched placebo after chemotherapy. A second active schedule used palifermin both before and after high-dose chemotherapy, for six doses in total.
Comparator
Matched placebo before and after high-dose chemotherapy. The trial also included the palifermin-before-only schedule, so the study was not limited to a single active schedule and a placebo group.
Primary question
How did the maximum severity distribution of oral mucositis differ across the randomized treatment groups at Day 32? Severity was assessed on the World Health Organization scale from grade 0 through grade 4. The category counts and a comparative treatment-effect estimate are not reported in the registry record.
The clinical question is about the distribution of the worst oral mucositis grade recorded by the specified assessment time, rather than about a single yes-or-no event. The scale distinguishes no findings or erythema only, soreness with or without erythema, ulcers with the ability to take solid food, ulcers with only liquids tolerated, and ulcers preventing oral intake. These categories reflect increasing severity as defined in the registry record.
The study also listed secondary outcomes concerning ulcerative mucositis incidence and duration, patient-reported mouth and throat soreness, and cataract and visual-acuity assessments. Those additional outcomes broaden the questions considered, but their presence does not supply an answer to the primary comparison. The registry record does not report the primary category counts, statistical comparison, or uncertainty estimate needed to assess how the treatment groups differed.
3. Trial Design
This was a phase 3, randomized, quadruple-masked, parallel-group study with placebo control. The registry record reports enrollment of 281 participants and identifies Swedish Orphan Biovitrum as the sponsor. The study is marked completed. Its recorded start was 2006-12, and primary completion was 2009-03.
Three treatment schedules are described. Participants in the palifermin-before-only group received palifermin before high-dose chemotherapy and matched placebo after it, for three doses of each. The placebo group received matched placebo before and after chemotherapy. The palifermin-before-and-after group received palifermin on both sides of high-dose chemotherapy, for six doses in total. The registry record identifies the placebo intervention as a drug; it does not provide further details about matching procedures beyond the arm descriptions.
The primary efficacy population was the full analysis set, defined in the record as all randomized participants and analyzed according to randomized treatment assignment. This description indicates that efficacy analyses were organized by assigned group, rather than by treatment received. The registry record does not report the exact statistical model, test, or method used to compare the groups.
The stated primary outcome was maximum oral mucositis severity at Day 32, categorized using the World Health Organization scale. Secondary outcomes included ulcerative mucositis incidence and duration, an area-under-the-curve measure based on patient-reported mouth and throat soreness, and eye-related outcomes. The record notes that an amendment in April 07 introduced cataract assessments at Screening, Month 6, and Month 12 in response to FDA and EMEA follow-up measures.
Although the title describes a double-blind study, the registry design fields describe masking as quadruple. The record does not explain which roles were masked or how masking was implemented, so the details cannot be reconciled further from the registry information. A sample-size justification, interim-analysis plan, and complete prespecified 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 | Palifermin Before Only | Placebo | Palifermin Before and After |
|---|---|---|---|
| STARTED | 109 | 57 | 115 |
| COMPLETED | 99 | 54 | 104 |
| NOT COMPLETED | 10 | 3 | 11 |
Reasons for not completing this period:
| Reason | Palifermin Before Only | Placebo | Palifermin Before and After |
|---|---|---|---|
| Adverse Event | 5 | 1 | 4 |
| Lost to Follow-up | 0 | 1 | 0 |
| Death | 1 | 0 | 1 |
| Non-Compliance | 1 | 1 | 0 |
| Withdrawal by Subject | 2 | 0 | 3 |
| Logistical error study assessments | 1 | 0 | 3 |
Analysis population for the primary outcome, as defined in the registry: Full analysis set that includes all randomized subjects, and was used to compare treatment effects for all efficacy endpoints. This set of subjects was analyzed according to their randomized treatment assignment.
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.
| Characteristic | Unit | Palifermin Before Only | Placebo | Palifermin Before and After | Total |
|---|---|---|---|---|---|
| Participants analysed | 109 | 57 | 115 | 281 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 90 | 49 | 100 | 239 |
| Age, Categorical: >=65 years | Participants | 19 | 8 | 15 | 42 |
| Age, Continuous | years | 55.3 (SD 8.4) | 57.1 (SD 7.0) | 56.1 (SD 7.5) | 56 (SD 7.7) |
| Sex: Female, Male: Female | Participants | 50 | 24 | 52 | 126 |
| Sex: Female, Male: Male | Participants | 59 | 33 | 63 | 155 |
| Region of Enrollment: Germany | participants | 16 | 9 | 23 | 48 |
| Region of Enrollment: Hungary | participants | 22 | 9 | 16 | 47 |
| Region of Enrollment: France | participants | 15 | 10 | 16 | 41 |
| Region of Enrollment: Austria | participants | 16 | 5 | 9 | 30 |
| Region of Enrollment: Belgium | participants | 8 | 1 | 11 | 20 |
| Region of Enrollment: Czech Republic | participants | 7 | 4 | 7 | 18 |
| Region of Enrollment: Italy | participants | 4 | 4 | 9 | 17 |
| Region of Enrollment: Switzerland | participants | 4 | 5 | 6 | 15 |
| Region of Enrollment: United Kingdom | participants | 4 | 2 | 5 | 11 |
| Region of Enrollment: Finland | participants | 3 | 3 | 4 | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4), measured over at Day 32.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was maximum severity of oral mucositis, assessed at Day 32. The registry record reports the number of participants in each of the World Health Organization grades: 0, 1, 2, 3, or 4. Grade 0 indicates no findings or erythema only; grade 1 indicates soreness, with or without erythema; grade 2 indicates ulcers while solid food remains possible; grade 3 indicates ulcers with only liquids possible; and grade 4 indicates ulcers preventing oral intake.
This endpoint is ordinal: its categories have an ordered severity, but the distances between adjacent grades are not specified as equal. Recording the maximum grade captures the most severe mucositis experienced during the assessment period rather than only whether any mucositis occurred. It can therefore preserve distinctions that would be lost if the categories were combined into a simple event/no-event outcome. The registry record does not specify the exact assessment schedule used to determine the maximum beyond identifying the Day 32 timeframe.
For interpretation, the category distribution matters. A treatment-group comparison could differ depending on whether changes occur among lower grades, higher grades, or across several categories. However, the registry record does not report the grade-specific counts, a summary contrast, or a confidence interval for this primary outcome. The endpoint definition alone therefore establishes what was measured, but not the direction, size, or precision of any treatment-group difference.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the trial question and identifies the primary outcome, but it does not report the primary outcome results. In particular, it gives no counts for the participants in each mucositis grade, no comparative estimate between treatment groups, and no confidence interval or p-value for the primary comparison. The treatment effect on maximum oral mucositis severity therefore cannot be characterized from the registry record.
This distinction is important because the endpoint has several ordered categories. A result for one grade alone would not necessarily describe the full distribution, and collapsing grades into a binary outcome would discard information unless a justified threshold had been specified. The record itself notes that the primary outcome has several categories or time points and was not reduced to one comparison. Accordingly, there is no single primary effect estimate in the posted results to interpret as a complete summary of the outcome.
The registry does list safety counts by group. Serious adverse events were reported for 13 of 111 participants in the palifermin-before-only group, 3 of 57 in the placebo group, and 18 of 109 in the palifermin-before-and-after group. These are descriptive counts in the registry record. They do not establish a causal safety difference: the record does not provide an adjusted comparison, uncertainty interval, event-level timing, or a detailed account of how events were collected. Counts for other adverse events are also listed, but those figures do not replace the missing primary efficacy results.
Planning illustrations based on an observed effect should not be mistaken for trial findings or for the trial's original design justification. The stated planning assumptions use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a fraction of the observed effect. Such illustrations depend on the effect estimate chosen as their starting point. A planning calculation is not observed power and does not supply missing evidence about the primary endpoint.
General statistical interpretation: an estimate describes the observed contrast, while a confidence interval indicates the range of values compatible with the data under the analysis assumptions. Neither can be assessed for the primary endpoint because the registry record does not report an estimate or interval. A non-reported result should not be treated as evidence of no difference, nor as evidence of a difference. The available record supports describing the design, endpoint, and listed adverse-event counts, but not a conclusion about comparative efficacy.
9. Statistical Methods Explained
General statistical explanation: a randomized parallel-group trial assigns participants to treatment strategies and compares outcomes across the assigned groups. Random assignment is intended to make groups comparable on average at the point of assignment, though chance imbalances can still occur. The registry describes this study as randomized and parallel-group, but does not report the sequence-generation or allocation-concealment procedures. Those procedural details are not reported in the registry record.
What does an ordinal endpoint mean?
General: an ordinal outcome has categories with a meaningful order, but the gaps between adjacent categories are not necessarily equal. The oral mucositis grades in this study run from no findings or erythema only through progressively more limiting ulceration. The grade labels therefore communicate rank and clinical description; they do not establish that a one-grade change has the same quantitative meaning at every point on the scale.
For an ordinal outcome, analysts may compare the full distribution, estimate the probability of being in a more severe category, or use another prespecified summary. These approaches answer related but not identical questions. A full distribution preserves all category information, while a threshold-based summary groups categories and can make the result depend on where that threshold is set. The registry record names the categories and the maximum-severity endpoint but does not identify the statistical method used to compare them. No particular model or test should therefore be attributed to the trial.
How should effect estimates and uncertainty be read?
General: an effect estimate quantifies a contrast between groups on a stated scale. A confidence interval communicates uncertainty about that estimate under the model and sampling assumptions; a wider interval generally indicates less precision. The interval does not show the probability that a particular treatment effect is true. In this record, the primary category counts, estimate, and interval are not reported in the registry record, so neither the magnitude nor precision of the primary comparison can be evaluated.
A p-value, when reported, addresses how unusual data at least as inconsistent with a specified null hypothesis would be under the analysis assumptions. It does not measure effect size, clinical importance, or the probability that the null hypothesis is true. The registry record does not report a primary-outcome p-value or describe the test used. It is therefore not possible to infer statistical significance from the endpoint description or the study design.
What does the planning illustration represent?
General: sample-size planning asks how many participants would be needed under stated assumptions to detect a specified effect with a chosen error rate and power. A planning illustration based on an observed effect is retrospective and depends on that estimated effect; it is not the same as the original trial design calculation. The registry record states that its planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. It also states that time-to-event planning gives total events using the Schoenfeld approach, whereas other planning results give participants per arm.
These assumptions are planning conventions, not evidence that the trial used the same design assumptions. They do not establish the original sample-size justification, the trial's actual power, or a treatment effect. The sample-size justification and the trial's own statistical plan are not reported in the registry record. Calculations based on an observed effect also inherit uncertainty from that effect estimate, which is especially relevant when the source estimate is imprecise.
Why do multiplicity and over-dispersion matter generally?
General: when several outcomes, time points, or comparisons are examined, the chance of at least one apparently unusual result can increase if each is tested separately without adjustment. A prespecified testing strategy or multiplicity adjustment can address this issue, but the appropriate approach depends on the scientific questions and analysis plan. The registry lists 16 outcome measures and 17 analyses, but does not report a multiplicity strategy. The number of listed measures alone does not establish how the analyses were conducted or whether any adjustment was applied.
General: over-dispersion occurs when observed variability exceeds what a statistical model assumes. If unaddressed, standard errors and uncertainty intervals can be misleading. This concept is relevant to some count outcomes, but the primary endpoint is described as categorical, and the registry record does not identify a model or report a dispersion parameter. No claim about over-dispersion in this trial is supported by the record.
Finally, the registry defines the efficacy population as all randomized participants analyzed according to randomized assignment. This is the population description available for interpreting the intended efficacy comparisons. The record does not describe exclusions, missing-outcome handling, sensitivity analyses, or deviations from the assigned treatment. Those omissions limit assessment of how robust any unreported comparison would be.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 16 outcome measures and 17 analyses. The outcomes extend beyond the primary maximum-severity measure to ulcerative mucositis incidence and duration, patient-reported mouth and throat soreness, and cataract and visual-acuity measures. Considering multiple outcomes or analyses can raise multiplicity concerns: when many statistical tests are conducted, the chance of at least one apparently notable finding may increase. Whether that concern was addressed depends on the prespecified analysis strategy.
A multiplicity adjustment or hierarchical testing plan is not reported in the registry record. The primary endpoint's category counts and inferential results are also not reported, so it is not possible to determine how the primary analysis related to secondary analyses or whether any result was interpreted within a formal testing hierarchy. The existence of multiple listed outcomes does not by itself establish that the reported analyses were unplanned or improperly conducted.
The efficacy population is described as the full analysis set, comprising all randomized participants and analyzed according to assigned treatment. This supports an assignment-based interpretation of the intended efficacy analysis. The registry record does not report how missing outcome data were handled, whether participants were excluded from particular analyses, or whether sensitivity analyses were conducted. It also does not describe adherence-related analyses or how protocol deviations were treated. These details are important because missingness and exclusions can affect the interpretation of group comparisons, especially if they differ by group or relate to outcome severity. Their absence from the registry record means the robustness of the efficacy analysis cannot be assessed from the available description.
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 |
|---|---|---|---|
| Palifermin Before Only | 13 | 111 | 11.7% |
| Placebo | 3 | 57 | 5.3% |
| Palifermin Before and After | 18 | 109 | 16.5% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Palifermin Before Only | 110 of 111 |
| Placebo | 56 of 57 |
| Palifermin Before and After | 109 of 109 |
Most frequent serious adverse event terms
| Event term | Organ system | Palifermin Before Only | Placebo | Palifermin Before and After |
|---|---|---|---|---|
| Cardiac failure | Cardiac disorders | 1 of 111 | 1 of 57 | 1 of 109 |
| Sepsis | Infections and infestations | 1 of 111 | 1 of 57 | 1 of 109 |
| Dehydration | Metabolism and nutrition disorders | 1 of 111 | 1 of 57 | 0 of 109 |
| Diarrhoea | Gastrointestinal disorders | 1 of 111 | 1 of 57 | 0 of 109 |
| Infection | Infections and infestations | 1 of 111 | 0 of 57 | 1 of 109 |
| Nausea | Gastrointestinal disorders | 0 of 111 | 1 of 57 | 1 of 109 |
| Pneumonia | Infections and infestations | 0 of 111 | 0 of 57 | 2 of 109 |
| Rash | Skin and subcutaneous tissue disorders | 0 of 111 | 0 of 57 | 2 of 109 |
| Vomiting | Gastrointestinal disorders | 1 of 111 | 1 of 57 | 0 of 109 |
| Atrial fibrillation | Cardiac disorders | 0 of 111 | 0 of 57 | 1 of 109 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Palifermin Before Only | Placebo | Palifermin Before and After |
|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | 87 of 111 | 44 of 57 | 77 of 109 |
| Diarrhoea | Gastrointestinal disorders | 80 of 111 | 42 of 57 | 81 of 109 |
| Pyrexia | General disorders | 48 of 111 | 22 of 57 | 47 of 109 |
| Vomiting | Gastrointestinal disorders | 46 of 111 | 25 of 57 | 44 of 109 |
| Rash | Skin and subcutaneous tissue disorders | 24 of 111 | 12 of 57 | 41 of 109 |
| Erythema | Skin and subcutaneous tissue disorders | 20 of 111 | 4 of 57 | 26 of 109 |
| Thrombocytopenia | Blood and lymphatic system disorders | 22 of 111 | 9 of 57 | 19 of 109 |
| Constipation | Gastrointestinal disorders | 23 of 111 | 11 of 57 | 15 of 109 |
| Febrile neutropenia | Blood and lymphatic system disorders | 16 of 111 | 12 of 57 | 19 of 109 |
| Headache | Nervous system disorders | 18 of 111 | 6 of 57 | 20 of 109 |
Serious events with the highest arm-level rate: pneumonia (1.8%), rash (1.8%), cardiac failure (1.8%), dehydration (1.8%), diarrhoea (1.8%).
The registry record lists serious adverse-event counts and counts for other adverse events separately for each treatment group. Serious adverse events were reported for 13 of 111 participants in the palifermin-before-only group, 3 of 57 in the placebo group, and 18 of 109 in the palifermin-before-and-after group. Other adverse events were reported for 110 of 111, 56 of 57, and 109 of 109 participants in those respective groups. These are the posted counts; no percentages or comparative effect estimates are needed to describe them.
The record also identifies pneumonia, rash, cardiac failure, dehydration, diarrhoea, and dizziness among the listed serious adverse-event terms, each reported as 1.8%. The registry does not provide additional event-level detail in this summary, such as timing, recurrence, or severity beyond the serious-event classification. Death counts are not reported in the registry record.
These descriptive safety figures should not be read as adjusted comparisons between treatments. The record does not report uncertainty intervals, a statistical test, or methods for attributing events. Group sizes differ, and raw counts alone do not quantify comparative risk. They also do not establish whether an event was caused by treatment. The registry record supports reporting the listed counts and terms, but not a causal conclusion about safety differences.
12. What the Result Does, and Does Not, Mean
The registry record describes a randomized comparison of palifermin schedules and placebo in people with multiple myeloma receiving melphalan followed by autologous peripheral blood stem cell transplantation. It specifies a primary endpoint that captures maximum oral mucositis severity by Day 32, using ordered World Health Organization grades. It also reports certain adverse-event counts and identifies secondary mucositis and eye-related outcomes.
What the record does not establish is the comparative primary outcome result. It does not report how many participants fell into each severity grade, an estimate contrasting groups, an uncertainty interval, or a p-value. Without those results, the direction and size of any efficacy difference cannot be described, and the precision of a possible difference cannot be assessed. The absence of a reported result is not evidence that groups were alike, and it is not evidence that they differed.
The listed serious adverse-event counts are descriptive. They do not on their own show that treatment caused an event or that one schedule had a higher or lower risk. The registry record does not provide adjusted safety comparisons or uncertainty estimates. Similarly, the listed secondary outcomes do not answer the primary question unless their results and analyses are reported and interpreted on their own terms.
General statistical interpretation: a study's design and endpoint definitions explain what investigators intended to compare, while results and uncertainty estimates are needed to evaluate what the observed data support. A planning illustration based on an observed effect can inform hypothetical future study sizes under its assumptions, but it cannot stand in for the missing outcome distribution or serve as observed power. The available record is therefore informative about study structure and measurement, but insufficient for a conclusion about the comparative efficacy of the palifermin schedules.
13. Why This Trial Matters Statistically
This trial raises a useful statistical issue: the primary outcome is an ordered severity scale rather than a single binary event. Preserving its categories can matter because the distribution may shift in different ways across severity levels. A simple incidence measure may answer whether an event occurred, but it cannot necessarily describe how severe the maximum event was. The registry record identifies the scale and endpoint clearly, even though it does not report the outcome distribution.
The comparison also involves more than one active schedule. Palifermin was administered before chemotherapy only in one group and both before and after chemotherapy in another, alongside a placebo group. That structure makes the choice and interpretation of contrasts important. A result for one schedule versus placebo would answer a different question from a direct comparison between schedules. The registry record does not report the statistical comparisons or estimates needed to evaluate those questions.
The number of listed outcomes also illustrates why a statistical analysis plan matters. Mucositis, patient-reported soreness, and eye-related outcomes address distinct questions, and multiple analyses can complicate interpretation if their roles are not specified. The registry record does not report a multiplicity strategy or the complete analysis plan. This limits understanding of how confirmatory and exploratory findings, if any, were distinguished.
Finally, the case demonstrates the difference between a detailed design record and a results record. Randomization, masking, population definitions, and endpoint descriptions provide essential context, but they cannot replace category counts and effect estimates. Here, the primary results are not reported in the registry record, so statistical interpretation must remain focused on what was measured and what information is missing.
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.
A central limitation is that the registry record does not report the primary outcome results. It describes maximum oral mucositis severity at Day 32 and defines the grading scale, but provides no grade-specific counts, treatment contrast, confidence interval, or p-value. As a result, comparative efficacy and precision cannot be assessed from the record.
The record does not provide the complete statistical analysis plan, sample-size justification, interim-analysis information, or details of the statistical test used for the primary endpoint. It also does not report the multiplicity strategy despite listing multiple outcomes and analyses. These omissions prevent assessment of whether the reported analyses followed a prespecified hierarchy or how uncertainty was quantified.
Although the efficacy population is defined as all randomized participants analyzed according to assigned treatment, missing-data handling, analysis-specific exclusions, adherence analyses, and sensitivity analyses are not reported in the registry record. The exact assessment procedures used to establish maximum mucositis severity are also not detailed beyond the endpoint timeframe and scale description.
The registry design fields describe quadruple masking, while the title describes a double-blind study. The record does not explain the roles masked or provide enough information to clarify the difference. Safety reporting is also limited: serious adverse-event counts and selected terms are listed, but event timing, comparative inference, attribution methods, and death counts are not reported in the registry record. These limitations constrain interpretation without establishing that the study itself lacked such procedures or information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma — NCT00434161.
- Registry record last updated 2015-03-30; status last verified 2015-03.
- Results first posted 2015-03-10.
- 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 describes a completed, randomized, quadruple-masked, parallel-group, phase 3, placebo-controlled study sponsored by Swedish Orphan Biovitrum. Enrollment was 281. The study compared palifermin before chemotherapy only, placebo before and after chemotherapy, and palifermin before and after chemotherapy in people with multiple myeloma receiving melphalan followed by autologous blood stem cell transplantation.
The primary endpoint was maximum oral mucositis severity at Day 32, measured on the World Health Organization scale from grade 0 through grade 4. The efficacy population was the full analysis set, defined as all randomized participants analyzed according to assigned treatment. The registry record does not report primary category counts or a comparative efficacy estimate.
Secondary outcomes included ulcerative mucositis, mouth and throat soreness, cataract measures, and visual acuity. Serious adverse-event counts are listed by group, but deaths are not reported in the registry record. The record does not report the full statistical analysis plan, missing-data methods, or multiplicity strategy.