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 Donor Atorvastatin Treatment for Preventing Severe Acute Graft-Versus-Host Disease in Patients Undergoing Myeloablative Peripheral Blood Stem Cell Transplantation (NCT01525407), a na phase 2 study. The primary outcome is Grade 3-4 Acute GVHD.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01525407 |
| Phase | Phase 2 |
| Condition | Malignant Neoplasm |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 83 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fred Hutchinson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with malignant neoplasms undergoing myeloablative peripheral blood stem cell transplantation, with donors receiving the study treatment. The record reports enrollment of 83 participants but does not further describe the endpoint analysis population.
Intervention
Donors received oral atorvastatin calcium beginning on day -14 and continuing until the last day of stem cell collection. The transplant procedures listed include allogeneic hematopoietic stem cell transplantation and peripheral blood stem cell transplantation.
Comparator
No concurrent control group is identified. The registry lists allocation as not applicable and the model as single group, so the study does not provide a randomized, like-for-like comparison of donor atorvastatin with an untreated donor group.
Primary question
What was the cumulative incidence of grade 3-4 acute graft-versus-host disease by day 100 after transplant, with death treated as a completing risk? The registry record describes this endpoint but does not report its numerical estimate or confidence interval.
The clinical rationale described in the record is that transplanted donor cells can mount an immune response against the recipient’s normal cells. The trial studied whether treating donors with atorvastatin before stem cell collection could prevent severe acute GVHD in recipients undergoing myeloablative transplantation. This is a question about an observed endpoint in a single-group study; without a concurrent control, the design cannot directly estimate how the outcome would differ under another donor-treatment strategy.
3. Trial Design
This was a phase II, single-group study of donor atorvastatin treatment in the setting of myeloablative peripheral blood stem cell transplantation. The registry reports enrollment of 83 and a completed status. The recorded study period began in 2012-05, with primary completion in 2015-09. Fred Hutchinson Cancer Center is listed as the sponsor.
The treatment was given to donors, not described as a recipient medication. Donors received atorvastatin calcium by mouth starting on day -14 and continuing through the last day of stem cell collection. The listed procedures include allogeneic hematopoietic stem cell transplantation and peripheral blood stem cell transplantation. The brief summary describes chemotherapy and total-body irradiation before the transplant, but further treatment details are not reported in the registry record.
The registry lists allocation as not applicable, masking as none, and the study model as single group. These features mean that no randomized allocation or masking is reported, and no concurrent control arm is identified. Therefore, outcomes can be described for the treated study group, but the design does not support a direct randomized comparison with another donor-treatment group. Differences between this group and outcomes in other settings could reflect differences in participants, care, measurement, or follow-up rather than the donor treatment alone.
The primary outcome was grade 3-4 acute GVHD, measured as cumulative incidence at day 100 after transplant, with death treated as a completing risk. The registry also lists secondary outcomes including chronic extensive GVHD, disease-free survival, grades II-IV acute GVHD, non-relapse mortality, overall survival, donor discontinuation of atorvastatin because of toxicity, and recipient need for secondary systemic immunosuppressive therapy. Numerical results for these outcomes are not reported in the registry record. The protocol details, eligibility criteria, sample-size justification, and 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 | Donors | Patients |
|---|---|---|
| STARTED | 41 | 42 |
| COMPLETED | 38 | 38 |
| NOT COMPLETED | 3 | 4 |
Reasons for not completing this period:
| Reason | Donors | Patients |
|---|---|---|
| Change in recipient treatment plan | 2 | 2 |
| Withdrawal by Subject | 0 | 1 |
| Transplant was delayed | 0 | 1 |
| Delay in Transplant | 1 | 0 |
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 | Donors | Patients | Total |
|---|---|---|---|---|
| Participants analysed | 38 | 38 | 76 | |
| Age, Continuous | years | 54 (22 to 64) | 52 (24 to 69) | 53 (22 to 69) |
| Sex: Female, Male: Female | Participants | 17 | 16 | 33 |
| Sex: Female, Male: Male | Participants | 21 | 22 | 43 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 4 | 4 | 8 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 28 | 27 | 55 |
| Race (NIH/OMB): More than one race | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 6 | 6 | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Grade 3-4 Acute GVHD, measured over First 100 days after transplant.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | probability |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the cumulative incidence of grade 3-4 acute graft-versus-host disease (GVHD) assessed at day 100 after transplant. The registry describes death as a completing risk. In practical terms, the endpoint concerns the probability of experiencing severe acute GVHD by the specified assessment time while accounting for death as an event that can prevent a later GVHD event from being observed.
General statistical context: a cumulative-incidence measure is useful when another event can alter the chance of observing the outcome of interest. Treating such an event as a competing risk distinguishes the probability of experiencing GVHD from a calculation that would act as though participants who died could still subsequently develop the measured endpoint. The exact analytical method used for this estimate is not reported in the registry record.
Severity is part of the endpoint definition: it includes grade 3-4 acute GVHD rather than all acute GVHD. The registry separately lists grades II-IV acute GVHD as a secondary outcome. These are distinct outcome definitions and should not be treated as interchangeable. The primary endpoint’s numerical estimate and its 95% confidence interval are not reported in the registry record. Consequently, the record does not establish the observed incidence or the precision with which it was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the clinical outcome of interest and the intended assessment time, but it does not report a numerical primary endpoint result. It states that grade 3-4 acute GVHD was assessed by day 100 after transplant as a cumulative incidence, with death as a completing risk. It also identifies 95% confidence interval as the endpoint’s dispersion measure. Without the estimate and interval, the observed incidence and its statistical precision cannot be evaluated from this record.
The design is important to interpretation. This was a single-group study with no concurrent control arm identified. Any endpoint estimate, if reported elsewhere, would describe the study group under the treatment and care conditions used; it would not by itself show how the same patients’ outcomes would have differed if their donors had not received atorvastatin. The registry explicitly indicates that the study is not a randomized comparison. Thus a causal treatment effect cannot be read directly from a single-group outcome rate.
The enrollment figure is 83, but the registry does not report how many participants contributed to the primary endpoint analysis, how many experienced the endpoint, or how many were censored or otherwise excluded. Enrollment should not be assumed to equal the analyzed population. The record also does not report follow-up completeness, the handling of missing endpoint information, or the statistical method used to estimate cumulative incidence.
The registry lists secondary outcomes across GVHD, survival, mortality, and treatment tolerability. It reports 10 outcome measures, but reports 0 analyses. No numerical secondary-outcome estimates or inferential results are included in the record. A list of intended outcomes is not evidence of their observed values, and the record does not permit conclusions about those outcomes.
General statistical context: a confidence interval communicates the uncertainty around an estimated quantity; a wider interval generally indicates less precision than a narrower one. It does not make a single-group estimate comparative, and it does not resolve design limitations. Since the primary estimate and interval are not reported, no statement about the size, direction, or precision of the observed endpoint result can be made from this registry record. The posted information supports describing the study question and design, not claiming that donor treatment did or did not change clinical outcomes.
9. Statistical Methods Explained
The statistical methods that were actually used are not reported in the registry record. The endpoint is described as a cumulative incidence rate, with 95% confidence interval, evaluated at day 100 and with death as a completing risk. No numerical endpoint estimate or interval is posted. The explanations below are general statistical context for understanding the stated endpoint, not a description of an unreported analysis plan.
What does cumulative incidence describe?
General statistical context: cumulative incidence estimates the probability that an event of interest has occurred by a particular time. For this trial, the event of interest is grade 3-4 acute GVHD, and the stated assessment time is day 100 after transplant. Unlike a simple count at a fixed time, a time-based estimate can account for when events occur and for participants whose follow-up ends before the assessment point. The registry does not report how follow-up or censoring was handled in this analysis.
Death matters because a participant who dies before developing GVHD can no longer experience a later GVHD event. The registry describes death as a completing risk. General statistical context: when an event prevents the outcome of interest from occurring later, it is commonly treated as a competing risk. A cumulative-incidence analysis accounts for this competing event rather than treating it as ordinary loss to follow-up. The specific estimator or software used in this trial is not reported in the registry record.
What does the confidence interval add?
The registry identifies a 95% confidence interval as the measure of dispersion for the primary endpoint. General statistical context: a confidence interval gives a range of values compatible with the data and the assumptions of the analysis, and conveys uncertainty about the estimated incidence. It is not a guarantee that the true value lies in that particular interval, nor does it correct for bias from the study design or incomplete data. Because the interval endpoints are not reported, the precision of the primary estimate cannot be assessed.
Why does the single-group design matter?
The study model is single group, and no concurrent control group is identified. General statistical context: an outcome rate observed in one group can describe that group, but a treatment effect requires a comparison that represents what would have happened under an alternative strategy. In a randomized trial, allocation is intended to make groups comparable on average; this registry does not describe randomized allocation. Comparison with an external group would require careful attention to differences in eligibility, care, measurement, and follow-up, and no such comparative analysis is reported in the registry record.
Masking is listed as none. The record does not explain whether endpoint assessment was blinded or how the outcome was adjudicated. General statistical context: when outcome assessment involves judgment, awareness of treatment can potentially influence classification. The record does not provide enough detail to assess whether that concern applied to the endpoint measurement in this study.
What can be inferred from the outcomes listed?
The record lists a primary endpoint and secondary outcomes, but reports 0 analyses. An outcome listing establishes what was planned or tracked in the registry; it does not supply a result, an effect estimate, or a test of a hypothesis. The statistical model, any covariate adjustment, handling of competing events for secondary outcomes, and any interim analyses are not reported in the registry record. The sample-size justification and any prespecified statistical thresholds are also not reported. General statistical context: without those details and the results, readers cannot evaluate whether analyses followed a prespecified plan, how much uncertainty surrounds estimates, or whether reported findings would be sensitive to analytic choices.
The record includes a 95% confidence interval designation for the primary endpoint, but does not state an inferential comparison or p-value. A confidence interval for a single-group incidence is not itself a test of treatment benefit against a control. Accordingly, statistical interpretation should remain descriptive and tied to the outcome definition, follow-up time, competing-risk description, and single-group design that the registry records.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 10 outcome measures, including the primary grade 3-4 acute GVHD endpoint and secondary outcomes covering other GVHD outcomes, survival, mortality, and treatment tolerability. Non-relapse mortality appears twice in the secondary-outcome list. The registry reports 0 analyses, so no numerical results are posted for these outcomes.
Multiplicity concerns arise when a study examines several outcomes or makes several statistical comparisons. General statistical context: as the number of inferential tests increases, the chance of at least one apparently notable result can increase unless the analysis plan addresses that issue. Whether the investigators used a prespecified hierarchy, adjustment, or other approach to multiplicity is not reported in the registry record. No p-values or comparative tests are reported, so the degree to which multiplicity affects any result cannot be assessed.
Missing-data handling and analysis populations are also not reported in the registry record. The record gives enrollment of 83, but does not state how many participants were included in the primary endpoint analysis, how missing observations were handled, or whether distinct intention-to-treat, per-protocol, or safety populations were defined. Follow-up completeness and reasons for any exclusions are not reported. These omissions limit assessment of whether the endpoint estimate, if available elsewhere, represents all enrolled participants or a subset.
The sample-size justification, interim analysis plans, and detailed statistical analysis plan are not reported in the registry record. Without these details, it is not possible to evaluate the planned precision, the prespecification of analyses, or the rules governing any repeated examination of accumulating data. These are reporting limitations; they do not establish that a particular analytic practice was or was not used.
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 |
|---|---|---|---|
| Patient Adverse Events | 21 | 38 | 55.3% |
| Donor Adverse Events | 0 | 41 | 0.0% |
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 |
|---|---|
| Patient Adverse Events | 0 of 38 |
| Donor Adverse Events | 0 of 41 |
Most frequent serious adverse event terms
| Event term | Organ system | Patient Adverse Events | Donor Adverse Events |
|---|---|---|---|
| Fever/Infection | Infections and infestations | 9 of 38 | 0 of 41 |
| GVHD | Immune system disorders | 7 of 38 | 0 of 41 |
| Nausea/Vomiting | Gastrointestinal disorders | 3 of 38 | 0 of 41 |
| Multi-Organ Failure | General disorders | 2 of 38 | 0 of 41 |
| DAH | Respiratory, thoracic and mediastinal disorders | 1 of 38 | 0 of 41 |
| Metabolic Acidosis | Metabolism and nutrition disorders | 1 of 38 | 0 of 41 |
| Pneumotosis | Gastrointestinal disorders | 1 of 38 | 0 of 41 |
| Acute Kidney Injury | Renal and urinary disorders | 1 of 38 | 0 of 41 |
| Pulmonary Emboli | Respiratory, thoracic and mediastinal disorders | 1 of 38 | 0 of 41 |
| Relapse | Blood and lymphatic system disorders | 1 of 38 | 0 of 41 |
Serious events with the highest arm-level rate: fever/infection (23.7%), gvhd (18.4%), nausea/vomiting (7.9%), multi-organ failure (5.3%), dah (2.6%).
The registry separates patient adverse events from donor adverse events. For patients, 38 were listed as at risk for serious adverse events and 21 as affected. For donors, 41 were listed as at risk and 0 as affected. The record lists 0 other adverse events for each group. These are the posted counts; the registry does not provide further detail about event timing, duration, severity grading, attribution, or the criteria used to classify events as serious.
The listed serious-event terms include Fever/Infection (23.7%), GVHD (18.4%), Nausea/Vomiting (7.9%), Multi-Organ Failure (5.3%), DAH (2.6%), and Metabolic Acidosis (2.6%). These percentages should be read as the registry’s reported figures. The record does not explain whether terms could overlap for the same participant, how the percentages were calculated, or whether the list is exhaustive. It also does not report a death count in the adverse-event groups.
General statistical context: adverse-event counts describe reported events within the stated group and at-risk population; they do not by themselves establish that treatment caused an event. Here, donor atorvastatin exposure and patient transplant outcomes concern different participant groups, so the patient and donor figures should be kept distinct. Because the record does not provide event-level details or a control group, these figures cannot establish comparative safety or attribute events to a specific treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase II, single-group study evaluated donor atorvastatin in connection with myeloablative peripheral blood stem cell transplantation. It specifies a clinically relevant primary endpoint: cumulative incidence of grade 3-4 acute GVHD by day 100 after transplant, with death treated as a completing risk. It also records serious adverse-event counts for patients and donors and lists secondary outcomes.
However, the record does not report the numerical primary endpoint result, its 95% confidence interval, or numerical results for secondary outcomes. The central clinical result therefore cannot be determined from the registry record. The absence of a posted estimate is not evidence that the event did or did not occur, and it cannot be interpreted as evidence for or against an effect of atorvastatin.
The single-group design further limits what an observed rate could mean. Without a concurrent comparison group, an outcome rate would characterize the study group, not establish what would have happened under a different donor-treatment strategy. The registry does not report randomized allocation or masking. Even if a numerical rate were available, it would not alone establish a causal treatment effect.
The adverse-event counts provide descriptive information, but do not by themselves identify cause or comparative risk. The registry does not report event-level timing, attribution, or enough methodological detail to assess how the listed percentages were derived. The counts should therefore be read as registry safety reporting rather than as proof that a treatment caused or prevented particular events.
General statistical context: estimates are most informative when accompanied by uncertainty intervals, transparent analysis populations, complete follow-up information, and a suitable comparison. Those elements are not fully reported in this record. The appropriate conclusion is limited: the registry describes the study question, design, endpoint, and some safety counts, while leaving the numerical efficacy outcome and important analysis details unreported.
13. Why This Trial Matters Statistically
This trial is statistically instructive because it asks a comparative clinical question using a single-group design. The registry describes donor atorvastatin as a strategy intended to prevent severe acute GVHD in recipients, but identifies no concurrent control arm and lists allocation as not applicable. This distinction matters: an observed outcome rate can describe what happened in a group, while estimating a treatment effect requires a credible comparison with an alternative.
The endpoint also illustrates why outcome definitions and event structure matter. The primary outcome is grade 3-4 acute GVHD by day 100, not acute GVHD of any grade, and death is identified as a completing risk. General statistical context: treating death as a competing event is relevant when it can preclude later observation of the outcome. The registry names cumulative incidence and a 95% confidence interval, but does not provide the estimate or interval needed to judge the result or its precision.
The record also demonstrates the difference between outcome registration and results reporting. It lists secondary outcomes across GVHD, survival, mortality, and tolerability, while reporting 0 analyses. The listed outcomes show the scope of measurement described in the registry, but do not answer those questions without numerical results and methods.
Finally, the safety record separates patient and donor counts, reflecting that the intervention was administered to donors while the principal clinical endpoint was measured in recipients. Keeping those populations distinct is essential when interpreting both benefits and harms. The study’s design and registry entries can frame careful questions, but missing endpoint results and analysis details prevent a complete statistical assessment.
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.
Several trial-specific limitations of interpretation follow from the registry record. First, the study is listed as single group, with allocation not applicable and no concurrent control group identified. This prevents a direct randomized comparison and limits causal interpretation of any outcome rate. The record also lists masking as none, but does not describe how endpoints were assessed or whether outcome classification involved blinded review.
Second, the primary endpoint’s numerical result and 95% confidence interval are not reported. Although the endpoint is defined as cumulative incidence of grade 3-4 acute GVHD by day 100, the record does not report the number of primary endpoint events, the analyzed population, or the method used to estimate cumulative incidence. It describes death as a completing risk but does not provide further methodological detail.
Third, enrollment of 83 does not establish the number included in each outcome analysis. The registry does not report missing-data handling, follow-up completeness, analysis populations, or reasons participants may not have contributed outcome data. Sample-size justification, interim analyses, and the statistical analysis plan are not reported in the registry record.
Finally, secondary outcomes are listed, but the record reports 0 analyses and no numerical results for them. The safety information is limited to group counts and a list of serious-event terms and percentages; event timing, attribution, overlap, and ascertainment details are not reported. These limitations concern what can be evaluated from the registry record and should not be taken as proof that unreported procedures or analyses were not performed.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Donor Atorvastatin Treatment for Preventing Severe Acute Graft-Versus-Host Disease in Patients Undergoing Myeloablative Peripheral Blood Stem Cell Transplantation — NCT01525407.
- Registry record last updated 2017-08-17; status last verified 2017-07.
- Results first posted 2017-08-17.
- 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 describes a completed phase II, single-group study sponsored by Fred Hutchinson Cancer Center, with enrollment of 83. Donors received oral atorvastatin calcium beginning on day -14 and continuing until the last day of stem cell collection for myeloablative peripheral blood stem cell transplantation. The study began in 2012-05 and reached primary completion in 2015-09.
The primary endpoint was cumulative incidence of grade 3-4 acute GVHD by day 100 after transplant, with death treated as a completing risk and 95% confidence interval listed as the dispersion measure. The numerical endpoint result and interval are not reported in the registry record. The record lists secondary outcomes but reports 0 analyses.
Patient serious adverse events were reported for 38 at risk, with 21 affected; donor serious adverse events were reported for 41 at risk, with 0 affected. Because no concurrent control group is identified and key results and analysis details are absent, the registry record does not support a comparative estimate of treatment effect.