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 Acute Graft-versus-Host Disease Treatment (BMT CTN 0802) (NCT01002742), a randomized phase 3 study. The primary outcome is GVHD-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01002742 |
| Phase | Phase 3 |
| Condition | Graft-versus-Host Disease, Immune System Disorders |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 236 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Medical College of Wisconsin |
| Results posted | Yes |
2. Clinical Question
Population
People with acute graft-versus-host disease (GVHD) entering a trial of initial systemic treatment. The registry record does not give further eligibility details or a more specific description of the primary-outcome population.
Intervention
Mycophenolate mofetil (MMF) given with systemic corticosteroids. MMF is the additional treatment being evaluated in the comparison.
Comparator
Placebo given with systemic corticosteroids. The placebo group provides the comparison for adding MMF to corticosteroid treatment.
Primary question
Does the registry record show a difference between the treatment groups in the number of participants alive and free of GVHD at Day 56 after randomization? That status defines success; all others are classified as study failures. The record does not report the group results or an estimated treatment effect for this endpoint.
The comparison is about the treatment strategies as assigned: corticosteroids with MMF versus corticosteroids with placebo. It is not a comparison of MMF alone against no treatment. The registry identifies GVHD-free survival at Day 56 as primary and lists additional outcomes concerning response, flares, immune suppression, steroid use, chronic GVHD, and systemic infections. Those secondary outcomes provide other perspectives on disease status and treatment experience, but their presence does not replace the primary question.
The registry lists 236 as enrollment. It does not state the detailed eligibility criteria, the number assessed for eligibility, or the analysis population used for the primary endpoint. The primary outcome definition treats survival and freedom from GVHD at the specified time as a combined success condition. A participant must meet both conditions to count as a success; failure to meet either condition is classified as a study failure under the registry description.
3. Trial Design
This is a randomized, parallel-group Phase III trial. One group received corticosteroids with MMF, and the other received corticosteroids with placebo. The registry describes MMF and placebo as drug interventions, and the arm descriptions identify corticosteroids as common treatment in both groups. Randomization is intended to support a fair comparison of assigned strategies by reducing systematic differences in how participants enter the groups. It does not, by itself, guarantee that every measured or unmeasured characteristic is balanced.
The registry lists the masking as triple, while the official title describes the trial as double blind. The record does not explain which people were masked or how the masking labels relate to one another. It also does not report details of the randomization sequence, allocation concealment, or how treatment assignment was implemented. Those operational details are not reported in the registry record.
The record lists enrollment of 236 and status as completed. It gives a study start of 2010-01 and primary completion of 2012-01. These dates describe the registered study timeline, not the timing or completeness of any particular participant’s follow-up.
The primary endpoint is a binary status at Day 56: alive and free of GVHD is success, while all other statuses are study failures. Secondary outcomes include complete response among surviving participants, GVHD flares requiring increased therapy, discontinuation of immune suppression without flare, cumulative steroid dose, topical or non-absorbable therapy, overall GVHD-free survival after randomization, chronic GVHD, and systemic infections. The registry lists 15 outcome measures, but it does not provide a complete account of how all outcomes were analyzed. The analysis count is listed as 0; the record therefore does not provide a statistical analysis record for these outcomes.
The posted record does not give the planned allocation ratio, sample-size justification, or details of the intended statistical model. It also does not identify a protocol or statistical analysis plan. These design and analysis details 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 | Placebo | Mycophenolate Mofetil |
|---|---|---|
| STARTED | 119 | 117 |
| COMPLETED | 119 | 116 |
| NOT COMPLETED | 0 | 1 |
Reasons for not completing this period:
| Reason | Placebo | Mycophenolate Mofetil |
|---|---|---|
| Determined to be ineligible | 0 | 1 |
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: All randomized patients were included in primary, intention-to-treat analysis.
| Characteristic | Unit | Placebo | Mycophenolate Mofetil | Total |
|---|---|---|---|---|
| Participants analysed | 119 | 116 | 235 | |
| Age, Customized: Median Age | years | 52.9 (9.1 to 76.3) | 54 (9.1 to 76.36) | 53.8 (9.1 to 76.3) |
| Sex: Female, Male: Female | Participants | 41 | 45 | 86 |
| Sex: Female, Male: Male | Participants | 78 | 71 | 149 |
| Primary Disease: Acute myeloid leukemia | participants | 47 | 41 | 88 |
| Primary Disease: Acute lymphoblastic leukemia | participants | 14 | 16 | 30 |
| Primary Disease: Chronic myeloid leukemia | participants | 5 | 3 | 8 |
| Primary Disease: Myelodysplastic syndrome | participants | 17 | 20 | 37 |
| Primary Disease: Lymphoma | participants | 17 | 17 | 34 |
| Primary Disease: Other | participants | 19 | 19 | 38 |
| Unrelated Donor | participants | 72 | 66 | 138 |
| Graft Source: Bone marrow | participants | 16 | 23 | 39 |
| Graft Source: Peripheral blood stem cell | participants | 102 | 91 | 193 |
| Graft Source: Cord blood | participants | 1 | 2 | 3 |
| Myeloablative Conditioning | participants | 74 | 74 | 148 |
| Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis: Grade I | participants | 16 | 11 | 27 |
| Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis: Grade II | participants | 62 | 68 | 130 |
| Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis: Grade III | participants | 34 | 30 | 64 |
| Grade of acute Graft-Vs-Host-Disease (GVHD) at Diagnosis: Grade IV | participants | 7 | 7 | 14 |
| Cutaneous involvement at onset: No Rash | participants | 36 | 31 | 67 |
| Cutaneous involvement at onset: Maculopapular Rash, <25% of Body Surface | participants | 14 | 11 | 25 |
| Cutaneous involvement at onset: Maculopapular Rash, 25-50% of Body Surface | participants | 21 | 27 | 48 |
| Cutaneous involvement at onset: Generalized Erythroderma | participants | 44 | 46 | 90 |
| Cutaneous involvement at onset: Erythroderma and Bullae Formation/Desquamation | participants | 4 | 1 | 5 |
| Upper GI Abnormalities at Diagnosis: No Protracted Nausea and Vomiting | participants | 82 | 85 | 167 |
| Upper GI Abnormalities at Diagnosis: Persistent Nausea, Vomiting or Anorexia | participants | 37 | 31 | 68 |
| Lower GI Abnormalities at Diagnosis: No Diarrhea | participants | 66 | 57 | 123 |
| Lower GI Abnormalities at Diagnosis: Diarrhea Less ≤ 500 mL/day | participants | 19 | 21 | 40 |
| Lower GI Abnormalities at Diagnosis: Diarrhea >500 but ≤ 1000 mL/day | participants | 14 | 21 | 35 |
| Lower GI Abnormalities at Diagnosis: Diarrhea >1000 but ≤ 1500 mL/day | participants | 9 | 8 | 17 |
| Lower GI Abnormalities at Diagnosis: Diarrhea >1500 mL/day | participants | 11 | 7 | 18 |
| Lower GI Abnormalities at Diagnosis: Stool with Frank Blood or Melena | participants | 0 | 2 | 2 |
| Liver Abnormalities at Diagnosis: Bilirubin <2 mg/dL | participants | 110 | 106 | 216 |
| Liver Abnormalities at Diagnosis: Bilirubin 2-3 mg/dL | participants | 5 | 1 | 6 |
| Liver Abnormalities at Diagnosis: Bilirubin 3.1-6 mg/dL | participants | 4 | 6 | 10 |
| Liver Abnormalities at Diagnosis: Bilirubin 6.1-15 mg/dL | participants | 0 | 2 | 2 |
| Liver Abnormalities at Diagnosis: Bilirubin >15 mg/dL | participants | 0 | 1 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as GVHD-free Survival, measured over Day 56.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is GVHD-free survival at Day 56 after randomization. The registry defines success as being alive and free of GVHD at that time. Every other status is considered a study failure. This is a binary endpoint: each participant’s status is classified into one of two categories for the primary definition.
Combining survival with freedom from GVHD makes the endpoint reflect two conditions at once. It does not count survival alone as success if GVHD remains present, and it does not count freedom from GVHD as success if the participant is not alive at the assessment time. The resulting endpoint is a concise way to express the proportion or number meeting both conditions at the specified follow-up point, if those results are reported.
In general, a fixed-time binary endpoint can be straightforward to interpret because it asks whether a defined state was reached by a specified time. It does not describe the timing of events within the follow-up period or distinguish among the different reasons a participant may fail to meet the success definition. The registry record does not report the number meeting the endpoint in either group, an effect estimate, a confidence interval, or a significance test. Therefore, the recorded definition can be explained, but the relative primary outcome results cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies a clinically defined primary outcome and a randomized comparison, but it does not post the primary outcome results in a form that supports a treatment-effect interpretation. The record describes success as alive and free of GVHD at Day 56 and classifies all other statuses as failures. It does not give the number of successes or failures by group, an estimated difference between groups, a confidence interval, or a p-value. Without those results, the direction and precision of the primary comparison are not reported in the registry record.
The study’s registered comparison is MMF plus corticosteroids versus placebo plus corticosteroids. Any estimate from this design would concern the added treatment strategy in the context of corticosteroid use, not MMF in isolation. Random assignment supports comparison of the assigned groups, but interpretation still depends on complete outcome ascertainment, appropriate analysis, and transparent reporting. Those details are not reported in the registry record.
The record lists secondary outcomes spanning response, GVHD flares, immune-suppression discontinuation, steroid dose, topical or non-absorbable therapy, GVHD-free survival after randomization, chronic GVHD, and systemic infections. Listing an outcome does not establish that a result was reported or that the outcome favored either group. The registry lists 15 outcome measures, but the available record does not provide a full set of results and analysis details for them.
The adverse-event record lists 13 serious adverse events in the placebo group among 119 at risk, and 13 in the MMF group among 116 at risk. It lists 0 other adverse events for each group, with the corresponding at-risk counts recorded as 119 and 116. These are descriptive registry counts. They do not, on their own, establish whether the treatments differ in safety: the record does not provide an adjusted comparison, uncertainty interval, or explanation of the adverse-event collection and coding methods.
The serious adverse-event terms listed include haematuria, septic shock, vomiting, orthostatic hypotension, diarrhoea, and cerebellar ataxia, with percentages recorded for those terms. The record does not specify in the available information whether those term percentages are pooled or group-specific, or what denominator was used. They should not be interpreted as a comparison between treatment groups.
In general, a confidence interval would indicate the range of effect estimates compatible with the data under the analysis assumptions, and a p-value would summarize how unusual the data would be under a specified null model. Neither is available for the primary comparison in the registry record. The absence of a posted estimate does not show that the groups were identical; it means the registry record does not allow the size or precision of the primary effect to be judged.
9. Statistical Methods Explained
Randomization assigns participants to treatment strategies by chance. In general, this helps prevent deliberate or systematic selection of participants into one arm and supports a causal comparison of the assigned strategies, provided follow-up and analysis are handled appropriately. It does not ensure that the groups will be exactly alike on every characteristic, particularly in a finite study. The record identifies the study as randomized but does not report the sequence-generation or allocation-concealment procedures.
What does masking mean?
Masking means that specified people involved in a trial do not know which intervention has been assigned. In general, masking can reduce the risk that expectations influence care, outcome assessment, or reporting. The record labels masking as triple, while the official title calls the trial double blind. It does not identify the people masked or explain this difference in wording. Further details are not reported in the registry record.
How should the primary endpoint be understood?
The primary outcome is a binary status at Day 56 after randomization. Success requires both being alive and being free of GVHD at that time; all other statuses count as study failures according to the registry definition. For a binary outcome, a group-level analysis commonly compares the frequency or proportion of participants meeting the defined condition. General methods may express such a comparison as an absolute difference, a relative measure, or another effect estimate. The registry record does not provide the arm-level primary outcome data or state which effect measure was used.
What would a confidence interval add?
In general, a confidence interval communicates uncertainty around an estimated effect. A narrow interval indicates greater precision under the assumptions of the method; a wide interval indicates less precision. It is not a guarantee that the true effect lies within the interval, and its interpretation depends on the design and analysis. No primary endpoint confidence interval is reported in the registry record, so the precision of the primary comparison cannot be assessed from it.
Why do analysis populations matter?
In general, an analysis population specifies which participants contribute to an estimate. An analysis based on assigned groups preserves the original randomized comparison, while analyses restricted by treatment received, adherence, or outcome availability can answer different questions and may introduce bias. The registry record does not report the primary analysis population, how deviations from assigned treatment were handled, or how missing Day 56 outcomes were classified. These details are not reported in the registry record.
How should event counts be read?
Counts describe how many events or affected participants were recorded, depending on the measure’s definition. A count alone does not show the event frequency unless its denominator and counting rules are clear. If people can experience more than one event, event counts may differ from the number of people with at least one event. General interpretation of adverse-event counts therefore depends on whether the registry is reporting events or affected participants, the observation period, and the collection rules. Those methods are not fully described in the available record.
What do planning illustrations mean?
In general, power calculations estimate the sample size needed to detect a prespecified effect under assumptions about the test, allocation, and variability. They are planning tools, not evidence that an observed effect is real or that a completed trial had a particular probability of finding it. The registry’s planning assumptions describe two-sided significance level 0.05, equal allocation, and a normal approximation, with illustrations based on fractions of an observed effect. Such illustrations are not the trial’s original design justification. The record does not report the trial’s own sample-size justification, and the primary endpoint results needed to interpret an observed-effect-based illustration are not reported in the registry record.
For time-to-event outcomes, general methods may account for when events occur and for incomplete observation through censoring. For binary outcomes assessed at a fixed time, methods instead depend on the status observed at that time and the treatment of missing status. The registry lists overall GVHD-free survival after randomization among secondary outcomes, but the methods used for it are not reported in the registry record. The primary endpoint is defined at Day 56, and the record does not state whether a time-to-event method or another statistical model was used.
Over-dispersion is a general issue when count data vary more than a simple model assumes; ignoring it can make uncertainty estimates too small. The registry identifies the primary outcome as participants with an event (counts) for the broader data characterization, but it does not report a model for the primary comparison or describe a dispersion parameter. No claim about over-dispersion handling can be made from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and several secondary outcomes, including response, GVHD flares, immune-suppression discontinuation, steroid dose, topical or non-absorbable therapy, overall GVHD-free survival, chronic GVHD, and systemic infections. Evaluating multiple outcomes can raise the chance of at least one apparently notable result arising by chance. In general, a prespecified hierarchy or adjustment can address some multiplicity concerns, but the record does not report whether such a plan was used, which outcomes were included in any family of tests, or whether adjustments were made. The registry lists 15 outcome measures and 0 analyses.
The primary endpoint is defined at Day 56, but the record does not provide an analysis of it. It also does not report outcome-level estimates or uncertainty for the listed secondary measures in the information available. Without those results and a stated analysis plan, neither multiplicity handling nor the relative emphasis placed on each outcome can be evaluated.
Missing-data procedures are not reported in the registry record. In particular, the record does not explain how missing Day 56 status, withdrawal, loss to follow-up, or other unavailable outcome information was handled. The endpoint definition says that all statuses other than alive and free of GVHD are study failures, but it does not specify how an unknown status was classified analytically.
The analysis populations are also not reported. The registry does not state whether the primary comparison included all randomized participants, whether any participants were excluded, or how treatment changes and protocol deviations were addressed. These omissions limit assessment of whether the reported analysis, if available elsewhere, would preserve the randomized comparison or represent a different estimand.
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 |
|---|---|---|---|
| Placebo | 13 | 119 | 10.9% |
| Mycophenolate Mofetil | 13 | 116 | 11.2% |
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 |
|---|---|
| Placebo | 0 of 119 |
| Mycophenolate Mofetil | 0 of 116 |
Most frequent serious adverse event terms
| Event term | Organ system | Placebo | Mycophenolate Mofetil |
|---|---|---|---|
| Haematuria | Renal and urinary disorders | 3 of 119 | 0 of 116 |
| Septic shock | Infections and infestations | 2 of 119 | 0 of 116 |
| Vomiting | Gastrointestinal disorders | 0 of 119 | 1 of 116 |
| Orthostatic hypotension | Vascular disorders | 0 of 119 | 1 of 116 |
| Diarrhoea | Gastrointestinal disorders | 0 of 119 | 1 of 116 |
| Cerebellar ataxia | Nervous system disorders | 0 of 119 | 1 of 116 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 0 of 119 | 1 of 116 |
| Syncope | Nervous system disorders | 0 of 119 | 1 of 116 |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | 0 of 119 | 1 of 116 |
| Stevens-Johnson syndrome | Skin and subcutaneous tissue disorders | 0 of 119 | 1 of 116 |
Serious events with the highest arm-level rate: haematuria (2.5%), septic shock (1.7%), vomiting (0.9%), orthostatic hypotension (0.9%), diarrhoea (0.9%).
The adverse-event record lists 13 serious adverse events in the placebo group, with 119 at risk, and 13 in the MMF group, with 116 at risk. It lists 0 other adverse events in each group, with the same respective at-risk counts. These are recorded counts and should not be treated as a complete comparative safety analysis. The registry record does not explain whether the counts refer to events or participants affected, or provide a statistical comparison or uncertainty interval.
The record also lists serious adverse-event terms and percentages: haematuria, 2.5%; septic shock, 1.7%; vomiting, 0.9%; orthostatic hypotension, 0.9%; diarrhoea, 0.9%; and cerebellar ataxia, 0.9%. The available information does not state whether these percentages are pooled or specific to an arm, what denominator was used, or whether a person could contribute to more than one term. They cannot be used to infer a between-group difference from the registry record.
In general, safety interpretation depends on how events were defined, collected, attributed, and followed, as well as on the number of people observed and the period of observation. These details are not reported in the registry record. The listed values describe what was recorded; they do not establish that one treatment strategy is safer than the other.
12. What the Result Does, and Does Not, Mean
The record establishes the registered clinical question, treatment groups, and definition of the primary endpoint. It documents a randomized Phase III parallel-group trial comparing corticosteroids with MMF against corticosteroids with placebo for initial systemic treatment of acute GVHD. The primary outcome is success defined as alive and free of GVHD at Day 56 after randomization, with all other statuses counted as failures.
Those design details do not reveal the trial’s primary result. The registry record does not report arm-level counts for the primary outcome, an effect estimate, a confidence interval, or a p-value. Consequently, the size, direction, and precision of any difference in GVHD-free survival cannot be determined from this record. Missing results should not be interpreted as evidence that the treatment strategies had equal outcomes or that one was superior.
The listed secondary outcomes indicate other aspects of clinical course and treatment that the trial registered for evaluation. Their presence does not establish that they were analyzed, that all were reported, or that they support any particular conclusion. The registry lists 15 outcome measures, while the analysis count is 0. The record does not provide a complete statistical analysis plan or outcome results for those measures.
The safety counts and serious-event terms are descriptive as recorded. The event totals are not accompanied by a comparative effect estimate, uncertainty interval, or sufficient detail about collection and denominators to support a firm group-level interpretation. A count is not a substitute for a statistical comparison, and the percentages attached to serious-event terms are not identified as arm-specific in the available information.
In general, a trial result is most informative when the endpoint definition, analysis population, estimate, precision, and handling of missing data are all clear. Several of those elements are not reported in the registry record. The trial’s design can therefore be described, but its primary efficacy result and a comparative interpretation of its safety findings cannot be established from the posted information.
13. Why This Trial Matters Statistically
This trial illustrates the distinction between a clearly stated clinical question and a reportable statistical result. The registry defines a focused primary endpoint: alive and free of GVHD at Day 56 after randomization. It also describes a randomized comparison in which both groups receive corticosteroids and the assigned additional treatment is MMF or placebo. These elements make the intended contrast understandable.
The record also shows why an endpoint definition alone is not enough to interpret a trial. To judge the observed treatment effect, readers need outcome data by group and an estimate that expresses the comparison. A measure of uncertainty is needed to show how precisely the effect is estimated. None of these primary comparison results is reported in the registry record. The result is a substantial gap between knowing what was planned to be measured and knowing what the data showed.
The listed secondary outcomes make multiplicity and reporting practices relevant statistical considerations. Multiple outcomes can describe different dimensions of disease and treatment, but their interpretation depends on prespecification, analysis methods, and complete reporting. The registry lists outcomes but does not report how multiplicity was addressed or provide the statistical analyses.
The safety record provides another teaching point: counts, percentages, and comparative effects are not interchangeable. Counts need clear definitions and denominators; percentages need a stated population and method; and comparisons need uncertainty assessment. The record does not give enough detail to interpret the serious-event terms as arm-specific comparisons.
Overall, the trial is statistically notable as a case where the randomized design and endpoint are registered, while the available record does not provide the analysis needed to quantify the primary comparison. Transparent reporting of estimates, precision, analysis populations, and missing-data handling is essential for readers to distinguish a null or imprecise finding from an unreported result.
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 main limitation for interpreting this trial from the registry record is that the primary endpoint results are not reported. There are no arm-level counts for GVHD-free survival at Day 56, no treatment-effect estimate, and no confidence interval or p-value. The record therefore supports a description of the question and endpoint, but not a conclusion about the observed primary comparison.
Important analysis details are also unavailable. The primary analysis population, statistical model, handling of missing Day 56 outcomes, and treatment of deviations from assigned therapy are not reported in the registry record. The record does not state whether the analysis followed the randomized assignments or used another population. It also does not provide the trial’s sample-size justification, interim-analysis information, or a statistical analysis plan.
The masking description is not fully clarified: the registry labels masking as triple, while the official title says double blind. The record does not explain who was masked or reconcile the labels. The randomization procedure and allocation concealment are also not described.
For safety, the group-level serious and other adverse-event counts are listed, but the collection methods, observation period, attribution procedures, and whether counts represent events or affected participants are not specified. The serious-event term percentages are not identified as pooled or arm-specific, and their denominators are not reported in the available information. These gaps prevent a detailed comparative interpretation of the safety data.
Finally, the registry lists secondary outcomes and 15 outcome measures but does not provide the analysis details needed to assess multiplicity or the completeness of outcome reporting. These limitations concern what can be established from the registry record; they do not independently determine what the full trial report may contain.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Acute Graft-versus-Host Disease Treatment (BMT CTN 0802) — NCT01002742.
- Registry record last updated 2023-01-04; status last verified 2022-12.
- Results first posted 2016-05-27.
- 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, parallel-group Phase III trial of initial systemic treatment for acute GVHD. The comparison is corticosteroids with MMF versus corticosteroids with placebo. Enrollment is listed as 236, with a study start of 2010-01 and primary completion of 2012-01.
The primary endpoint is GVHD-free survival at Day 56 after randomization. Success is defined as alive and free of GVHD; all other statuses are study failures. Secondary outcomes address response, GVHD flares, immune suppression, steroid use, GVHD-free survival after randomization, chronic GVHD, and systemic infections.
The registry does not report the arm-level primary outcome comparison, its estimate, or its precision. It also does not report the primary analysis population, missing-data procedures, or a complete statistical analysis plan. Safety counts and serious-event terms are listed, but the available details do not support a comparative safety conclusion.