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 Autologous Stem Cell Transplant Followed By Maintenance Therapy in Treating Elderly Patients With Multiple Myeloma (NCT01849783), a na phase 2 study. The primary outcome is Median Progression Free Survival (mPFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01849783 |
| Phase | Phase 2 |
| Condition | Extramedullary Plasmacytoma, Isolated Plasmacytoma of Bone, Light Chain Deposition Disease |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 41 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Margarida Magalhaes-Silverman |
| Results posted | Yes |
2. Clinical Question
Population
Participants aged 65 years or older with multiple myeloma or a related plasma cell malignancy who received at least one day of protocol treatment. The registry lists 41 enrolled participants.
Intervention
A treatment sequence centered on autologous stem cell transplant, with induction chemotherapy and planned post-transplant consolidation and maintenance. The listed drugs include dexamethasone, cisplatin, doxorubicin, cyclophosphamide, etoposide, bortezomib, thalidomide, and melphalan.
Comparator
There was no concurrent comparison group in this single-group study. The brief summary refers to non-transplant approaches as a context for the question, but the registry record does not describe a randomized or like-for-like control group.
Primary question
What was the median progression-free survival after the protocol treatment sequence? The registry defines progression-free survival as time from the start of DPACE to documented disease progression or death from any cause. The median and its confidence interval are not reported in the registry record.
The clinical question concerns outcomes following a transplant-based treatment sequence in older participants with myeloma or a related plasma cell malignancy. The brief summary frames the question as whether transplant followed by maintenance is associated with better outcomes than non-transplant approaches, with progression-free survival as the primary measure. However, the design recorded is single-group, with allocation listed as not applicable. There is therefore no concurrent non-transplant group in this trial to estimate a comparative treatment effect.
The eligibility description spans multiple conditions, including several stages of multiple myeloma and other plasma cell disorders. That breadth is relevant to interpretation: a single outcome summary across the enrolled population would describe the participants included in this study, not necessarily each listed diagnosis separately. Diagnosis-specific outcome results are not reported in the registry record. The primary endpoint also includes death from any cause, not only progression, so it captures more than a change in disease status alone.
3. Trial Design
This was a Phase 2, single-group, unmasked study with allocation listed as not applicable. The registry reports enrollment of 41 participants and a completed status. The recorded start date is 2013-04-04, and the primary completion date is 2020-09-30. The registry record does not report a concurrent control group, random allocation, or masking.
The study population was described as participants aged 65 years or older with multiple myeloma or a related plasma cell malignancy. The primary outcome population is narrower in operational terms: participants who had at least one day of protocol treatment. The registry lists a range of conditions, including extramedullary plasmacytoma, isolated plasmacytoma of bone, light chain deposition disease, primary systemic amyloidosis, and Stage I, Stage II, and Stage III multiple myeloma.
The arm description begins with DPACE induction chemotherapy and stem cell collection. It states that additional collection and/or chemotherapy may be required, and that participants receive dexamethasone for four days every 14 days after collection. The listed intervention drugs also include bortezomib, thalidomide, and melphalan. Because the arm description is incomplete in the registry record, the full timing and role of every listed drug, as well as all details of the transplant, consolidation, and maintenance sequence, are not reported in the registry record.
The single-group design is central to how results can be read. A cohort outcome can describe what happened among participants treated under the study protocol, but it cannot by itself estimate how outcomes would have differed under a separate treatment approach. The registry record does not report the protocol’s sample-size justification or a prespecified comparative analysis plan.
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 | Autologous Stem Cell Transplant |
|---|---|
| STARTED | 41 |
| COMPLETED | 19 |
| NOT COMPLETED | 22 |
Reasons for not completing this period:
| Reason | Autologous Stem Cell Transplant |
|---|---|
| Adverse Event | 5 |
| Death | 2 |
| Physician Decision | 1 |
| Withdrawal by Subject | 1 |
| Unable to comply | 2 |
| Disease progression | 11 |
Analysis population for the primary outcome, as defined in the registry: Participants who have had at least one day of protocol treatment.
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 | Autologous Stem Cell Transplant |
|---|---|---|
| Participants analysed | 41 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 41 |
| Age, Continuous | years | 69 (65 to 75) |
| Sex: Female, Male: Female | Participants | 19 |
| Sex: Female, Male: Male | Participants | 22 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 39 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 38 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Region of Enrollment: United States | participants | 41 |
6. Primary Endpoint
The registry lists the primary outcome as Median Progression Free Survival (mPFS), measured over From the start of DPACE for all participants who have had at least one day of protocol treatment. Up to 6 years..
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is median progression-free survival, measured in months. The registry defines progression-free survival as the time from the start of DPACE to the first documentation of disease progression under International Myeloma Working Group response criteria, or death from any cause. Progression is described as an increase of at least 25% from the lower response value. The timeframe is stated as up to 6 years, and the endpoint population consists of participants with at least one day of protocol treatment.
A time-to-event endpoint incorporates both whether an event occurs and how long participants remain without that event. In general, progression-free survival is used to describe the duration until progression or death, combining these events into a single outcome. The median is the time by which half of the relevant event-time distribution has experienced an event, subject to the methods used to handle participants whose follow-up ends before an event is recorded. Those handling details are not reported in the registry record.
The registry names a 95% confidence interval as the measure of uncertainty around the median. A confidence interval communicates the precision of an estimate under the statistical method used; it is not a range containing every participant’s individual time. The median value and its confidence interval are not reported in the registry record. Therefore, the endpoint definition is available, but the study’s estimated progression-free survival cannot be interpreted from the posted registry outcome information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies median progression-free survival as the primary outcome, but it does not report the median estimate or its 95% confidence interval. Without those values, the posted record does not establish the observed duration of progression-free survival or the precision of that estimate. The record also lists zero analyses. No hazard ratio or other sponsor-reported comparative effect is reported in the registry record.
The endpoint definition is clear about the origin of time and the events counted: time starts at DPACE, and an event is either documented disease progression or death from any cause. This composite definition matters because the endpoint can be reached through either component. The registry does not report separate outcome results for progression and death, so their individual contributions to the primary outcome cannot be described.
The design was single-group, and allocation was not applicable. Accordingly, even if a cohort-level median were available, it would describe progression-free survival among the treated study population. It would not directly quantify a difference versus non-transplant treatment. The brief summary raises that broader comparison, but the study design recorded in the registry does not include a concurrent non-transplant arm. Comparisons with outcomes from other populations or studies would require information not reported in this record and would not be equivalent to a randomized comparison.
Enrollment was 41, but that number alone does not establish the precision of the primary estimate. Precision depends on the number and timing of observed events and on follow-up, among other features. The registry’s stated timeframe extends up to 6 years, but the actual follow-up distribution and event count for the primary endpoint are not reported in the registry record. Nor are the number of participants included in the endpoint analysis, the number censored, or the handling of missing outcome information reported.
The study is marked completed, but completion status is not itself an outcome result. Likewise, the listed dates describe the trial record’s timeline, not the magnitude or reliability of the primary endpoint. The appropriate interpretation is therefore limited: the registry describes the population, treatment approach, and definition of the primary endpoint, but does not post the primary numerical result needed to characterize the cohort’s progression-free survival.
General statistical point: estimates from time-to-event outcomes are usually interpreted together with their uncertainty and the amount of follow-up supporting them. A confidence interval that is narrow indicates more precision than a wide interval, while a median without an interval gives less information about uncertainty. Here, neither the median nor its interval is reported, so this kind of precision assessment cannot be made.
9. Statistical Methods Explained
Median progression-free survival is a time-to-event summary. Each participant contributes follow-up from the defined starting point until the first qualifying event or the end of their observed follow-up. In this study, the starting point is the start of DPACE, and the qualifying events are disease progression under the specified criteria or death from any cause. The registry describes follow-up as extending up to 6 years.
What does a median time-to-event estimate describe?
In general, a median time-to-event estimate identifies the point in the event-time distribution at which half of the population has experienced the event, with the estimation method accounting for participants whose event has not been observed by the time their follow-up ends. Such participants are commonly described as censored: they contribute information up to their last known event-free time, rather than being treated as if an event occurred then. This is a general explanation of time-to-event analysis, not a claim about the exact method used for this study. The registry record does not report the estimator or detailed censoring rules.
The median differs from an average because it is a position in the event-time distribution, not the arithmetic mean of all observed durations. It can be useful when the distribution of times is asymmetric or when some participants have not experienced an event by the end of their observed follow-up. However, the estimate depends on the observed event and censoring information. The primary result is not reported in the registry record, so the median and its precision cannot be evaluated.
How should the confidence interval be understood?
The registry names a 95% confidence interval for the median. In general, a confidence interval describes statistical uncertainty around an estimate under the assumptions and method used to calculate it. It does not describe the spread of individual participant outcomes, and it does not mean that a specified proportion of individual event times fall within its limits. A narrower interval conveys greater precision than a wider one, all else equal. The numerical limits and the method used to calculate the interval are not reported in the registry record.
For time-to-event outcomes, the interval can depend on how many events have occurred, how follow-up is distributed, and how many participants remain under observation over time. These are general methodological considerations. The registry record does not report the primary-event count, the follow-up distribution, or the number at risk at particular times. No further inference about the quality or stability of the estimate is possible from the endpoint definition alone.
What is the relevance of the single-group design?
A single-group outcome analysis estimates the observed experience of the enrolled participants under the study protocol. It does not create a counterfactual estimate of what would have happened to those same participants under another approach. General statistical interpretation therefore distinguishes an outcome observed in a cohort from a treatment effect estimated by comparing groups. Because this record describes a single-group design and no control arm, it cannot provide a randomized between-group estimate of the effect of transplant followed by maintenance versus non-transplant care.
The study’s brief summary frames a comparison with non-transplant approaches, but that framing does not change the recorded design. The registry does not report an external comparison group or a method for adjusting comparisons with another population. A numerical contrast with an outside cohort, if attempted, would depend on comparability of participants, care, outcome definitions, and follow-up; those details are not available in this record. No comparative effect should be inferred from the single-group design.
What do the planning assumptions mean?
The registry describes sample-size calculations as planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event calculations, the stated quantity is the total number of events, using the Schoenfeld approach. These are planning assumptions, not observed trial results. A planning illustration does not show that the study had a particular probability of detecting an effect, and it is not observed power. The registry’s actual sample-size justification is not reported in the registry record.
The stated planning framework assumes a two-arm comparison, while the recorded study itself is single-group. Thus, such planning illustrations should not be mistaken for the actual design or as evidence that a comparator was enrolled. The numerical planning results and the effect fractions used are not reported in the registry record. No participant-per-arm calculation can be described from the information recorded here, and no such calculation would alter the fact that this trial enrolled a single group.
Why does over-dispersion matter in general?
General statistical point: over-dispersion occurs when the variability in observed data is greater than a model assumes. If a model underestimates variability, its standard errors can be too small and its confidence intervals too narrow, making results appear more precise than warranted. Appropriate methods may account for extra variability, depending on the outcome and design. This is a general concept only. The registry record does not report a model for the primary endpoint, an over-dispersion assessment, or an adjustment of that kind.
More broadly, a complete statistical assessment would need the numerical endpoint estimate, uncertainty interval, event and censoring information, and the analysis method. Those items are not reported in the registry record. The available description permits explanation of the endpoint and design, but not a reconstruction of the sponsor’s primary analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies one primary outcome, median progression-free survival, and lists a secondary outcome concerning mean change in quality-of-life indicators after transplant. It also reports four outcome measures, but does not provide a complete analysis description for those measures. A prespecified statistical plan, including whether and how multiplicity was addressed, is not reported in the registry record.
Multiplicity arises when several hypotheses or outcomes are examined. In general, testing many outcomes can increase the chance that at least one result appears statistically notable by chance. Researchers may define a hierarchy, adjust significance thresholds, or treat secondary analyses as exploratory. The registry record does not state which approach, if any, was planned or used. The available information therefore does not support a claim that multiplicity was controlled or that it was ignored.
Missing-data methods are also not reported in the registry record. For a time-to-event endpoint, the status and timing of follow-up are important, including how participants who stop attending or have incomplete records are handled. The registry does not report missingness, loss to follow-up, censoring rules, or sensitivity analyses. These omissions limit assessment of how robust the primary estimate would be to incomplete follow-up, even if its numerical value were available.
The primary outcome population is defined as participants who had at least one day of protocol treatment. The registry does not report whether this population is identical to all enrolled participants, nor does it describe an intention-to-treat analysis population or another prespecified analysis set. The number analyzed for each outcome and the reasons for any exclusions are not reported in the registry record. Interim analyses, stopping rules, and the statistical plan are also not reported in the registry record. As a result, the record does not allow a full appraisal of outcome selection, analysis populations, missing data, or multiplicity.
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 |
|---|---|---|---|
| D-PACE/Induction | 14 | 41 | 34.1% |
| Post D-PACE/Pre-Transplant | 2 | 41 | 4.9% |
| Transplant | 8 | 39 | 20.5% |
| Post-transplant/Consolidation | 4 | 38 | 10.5% |
| Maintenance Therapy, Year 1 | 11 | 37 | 29.7% |
| Maintenance Therapy, Year 2 | 9 | 31 | 29.0% |
| Long Term Follow-up | 1 | 24 | 4.2% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| D-PACE/Induction | 0 of 41 |
| Post D-PACE/Pre-Transplant | 0 of 41 |
| Transplant | 1 of 39 |
| Post-transplant/Consolidation | 0 of 38 |
| Maintenance Therapy, Year 1 | 1 of 37 |
| Maintenance Therapy, Year 2 | 0 of 31 |
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 |
|---|---|
| D-PACE/Induction | 41 of 41 |
| Post D-PACE/Pre-Transplant | 8 of 41 |
| Transplant | 38 of 39 |
| Post-transplant/Consolidation | 8 of 38 |
| Maintenance Therapy, Year 1 | 27 of 37 |
| Maintenance Therapy, Year 2 | 22 of 31 |
Most frequent serious adverse event terms
| Event term | Organ system | D-PACE/Induction | Post D-PACE/Pre-Transplant | Transplant | Post-transplant/Consolidation | Maintenance Therapy, Year 1 | Maintenance Therapy, Year 2 |
|---|---|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | 2 of 41 | 0 of 41 | 0 of 39 | 0 of 38 | 0 of 37 | 0 of 31 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 41 | 0 of 41 | 0 of 39 | 1 of 38 | 0 of 37 | 0 of 31 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 41 | 0 of 41 | 1 of 39 | 0 of 38 | 0 of 37 | 0 of 31 |
| Atrial fibrillation | Cardiac disorders | 0 of 41 | 0 of 41 | 0 of 39 | 0 of 38 | 0 of 37 | 1 of 31 |
| Colitis | Gastrointestinal disorders | 1 of 41 | 0 of 41 | 0 of 39 | 0 of 38 | 0 of 37 | 0 of 31 |
| Diarrhea | Gastrointestinal disorders | 0 of 41 | 0 of 41 | 0 of 39 | 1 of 38 | 0 of 37 | 0 of 31 |
| Diarrhea | Gastrointestinal disorders | 0 of 41 | 0 of 41 | 1 of 39 | 0 of 38 | 0 of 37 | 0 of 31 |
| Nausea | Gastrointestinal disorders | 0 of 41 | 0 of 41 | 0 of 39 | 0 of 38 | 0 of 37 | 1 of 31 |
| Vomiting | Gastrointestinal disorders | 0 of 41 | 0 of 41 | 0 of 39 | 0 of 38 | 0 of 37 | 1 of 31 |
| Edema limbs | General disorders | 0 of 41 | 0 of 41 | 1 of 39 | 0 of 38 | 0 of 37 | 0 of 31 |
Most frequent non-serious adverse event terms
| Event term | Organ system | D-PACE/Induction | Post D-PACE/Pre-Transplant | Transplant | Post-transplant/Consolidation | Maintenance Therapy, Year 1 | Maintenance Therapy, Year 2 |
|---|---|---|---|---|---|---|---|
| Lymphocyte count decreased | Investigations | 41 of 41 | 8 of 41 | 36 of 39 | 8 of 38 | 27 of 37 | 22 of 31 |
| White blood cell decreased | Investigations | 39 of 41 | 0 of 41 | 38 of 39 | 3 of 38 | 4 of 37 | 4 of 31 |
| Platelet count decreased | Investigations | 35 of 41 | 1 of 41 | 38 of 39 | 3 of 38 | 3 of 37 | 5 of 31 |
| Neutrophil count decreased | Investigations | 33 of 41 | 1 of 41 | 31 of 39 | 2 of 38 | 3 of 37 | 3 of 31 |
| Hypophosphatemia | Metabolism and nutrition disorders | 10 of 41 | 1 of 41 | 22 of 39 | 3 of 38 | 11 of 37 | 9 of 31 |
| Anemia | Blood and lymphatic system disorders | 15 of 41 | 2 of 41 | 30 of 39 | 1 of 38 | 3 of 37 | 2 of 31 |
| CD4 lymphocytes decreased | Investigations | 2 of 41 | 7 of 41 | 1 of 39 | 6 of 38 | 23 of 37 | 14 of 31 |
| Hyperglycemia | Metabolism and nutrition disorders | 4 of 41 | 1 of 41 | 4 of 39 | 1 of 38 | 4 of 37 | 4 of 31 |
| Hypocalcemia | Metabolism and nutrition disorders | 4 of 41 | 0 of 41 | 13 of 39 | 0 of 38 | 0 of 37 | 0 of 31 |
| Febrile neutropenia | Blood and lymphatic system disorders | 3 of 41 | 1 of 41 | 8 of 39 | 2 of 38 | 0 of 37 | 0 of 31 |
Serious events with the highest arm-level rate: febrile neutropenia (4.9%), myelodysplastic syndrome (4.2%), atrial fibrillation (3.2%), nausea (3.2%), vomiting (3.2%).
The registry lists adverse-event summaries across treatment periods: D-PACE/Induction, Post D-PACE/Pre-Transplant, Transplant, Post-transplant/Consolidation, Maintenance Therapy, Year 1, Maintenance Therapy, Year 2, and Long Term Follow-up. For each period, the record gives numbers at risk and numbers affected for serious and other adverse events, alongside deaths. These are phase-specific summaries, not a single overall comparison between treatment groups.
For example, during D-PACE/Induction, 14 of 41 at-risk participants were listed as affected by serious adverse events, and 41 of 41 were listed as affected by other adverse events. During Transplant, 8 of 39 at-risk participants were listed as affected by serious adverse events, and 38 of 39 by other adverse events; 1 death is listed for that period. During Maintenance Therapy, Year 1, 11 of 37 at-risk participants were listed as affected by serious adverse events, and 27 of 37 by other adverse events; 1 death is listed. These phase entries should be read as posted, without adding counts across periods, since the record does not establish that participants are unique across those summaries.
The listed serious adverse-event terms include febrile neutropenia at 4.9%, myelodysplastic syndrome at 4.2%, atrial fibrillation at 3.2%, nausea at 3.2%, vomiting at 3.2%, and lung infection at 3.2%. The registry record does not report the denominator or additional calculation details for these percentages. Safety summaries describe events recorded during the study; without a concurrent control group, they do not estimate comparative risks attributable to treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a Phase 2, single-group study of a transplant-based treatment sequence in participants aged 65 years or older with multiple myeloma or a related plasma cell malignancy. It defines progression-free survival as the primary outcome and specifies the starting point, qualifying events, and outcome population. It also lists adverse-event summaries by treatment period. These are useful descriptions of the study’s scope and recorded outcomes.
The record does not report the numerical median progression-free survival or its 95% confidence interval. It therefore does not establish how long participants remained free of progression or death, nor how precisely that duration was estimated. It also does not provide a comparative outcome against non-transplant treatment. Although the brief summary frames the study question in relation to non-transplant approaches, the recorded single-group design cannot answer a randomized comparative question by itself.
General statistical point: a treatment outcome and a treatment effect are different quantities. An outcome describes what was observed in a group; an effect compares outcomes under alternative treatment conditions, ideally with a design that supports a fair comparison. The single-group study can describe its enrolled cohort, but the registry record does not supply the comparison needed to estimate a difference between treatment strategies.
The adverse-event summaries also require a limited interpretation. They report counts by study period and selected serious-event terms, but do not constitute a controlled comparison. They cannot establish how event frequency would differ under another approach. The record’s adverse-event counts should not be combined across periods as though they necessarily represented distinct people or independent observations.
Completion status, enrollment, and the stated follow-up timeframe do not substitute for the missing primary outcome estimate. A completed study can still have an incomplete registry outcome record. The appropriate conclusion is that the registry documents the study design and outcome definitions, while the primary numerical result and several key analytical details are not reported in the registry record. No conclusion about a treatment benefit or lack of benefit can be drawn from these entries alone.
13. Why This Trial Matters Statistically
Statistically, this study illustrates why a clearly defined endpoint and a reported numerical result are both necessary for interpretation. The registry specifies progression-free survival, its time origin, its event definition, the population to be included, and a confidence-interval measure. Those details describe what the investigators intended to measure. But without the median and interval, readers cannot assess the observed outcome or its precision.
The single-group design is equally important. The brief summary poses a question that invokes non-transplant approaches, whereas the recorded design has no concurrent comparison group. This distinction separates a descriptive cohort result from a comparative treatment effect. A study can contribute information about outcomes and safety observations within its enrolled group while remaining unable to isolate how those outcomes compare with another strategy.
The registry also demonstrates the importance of distinguishing protocol descriptions from analysis reporting. The treatment sequence is only partially described, and the statistical plan, missing-data methods, analysis populations, and multiplicity approach are not reported in the registry record. These details help readers judge how outcomes were assembled and whether the reported analysis matches the intended question. Their absence limits reproducibility and critical appraisal.
Finally, time-to-event outcomes depend on follow-up and event information, not enrollment alone. The enrollment of 41 participants gives the scale of the study but does not, by itself, tell readers how many progression or death events supported the primary estimate. With no posted primary result, the registry record cannot support an assessment of endpoint precision. The statistical importance of this trial record therefore lies in the contrast between a defined clinical question and the limited outcome and analysis information available for evaluating it.
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 primary limitation for interpretation is that the registry record does not report the median progression-free survival or its 95% confidence interval. It also does not report the number of primary endpoint events, follow-up distribution, or the number of participants included in the endpoint analysis. As a result, the primary outcome cannot be quantitatively characterized from the registry information.
The single-group design limits causal comparison. No concurrent non-transplant group is recorded, and allocation is listed as not applicable. The study therefore cannot provide a randomized estimate of how outcomes differ between the transplant-based sequence and a non-transplant approach. The brief summary’s comparative framing should not be mistaken for a comparative trial design.
The registry lists multiple eligible diagnoses, including several myeloma stages and related plasma cell malignancies, but does not report diagnosis-specific outcome results. The available record consequently does not show whether outcomes varied across these conditions. The arm description is incomplete, so the timing and full details of the treatment sequence cannot be reconstructed from the record.
Important analytical details are absent: the statistical plan, sample-size justification, interim analyses, analysis populations, missing-data methods, censoring rules, and multiplicity approach are not reported in the registry record. The adverse-event summaries are organized by treatment period, and the record does not establish whether affected counts across periods refer to distinct participants. These limitations restrict interpretation of both efficacy outcomes and safety summaries. The trial’s completed status does not resolve these reporting gaps.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Autologous Stem Cell Transplant Followed By Maintenance Therapy in Treating Elderly Patients With Multiple Myeloma — NCT01849783.
- Registry record last updated 2022-05-11; status last verified 2022-04.
- Results first posted 2022-05-11.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry describes a completed Phase 2, unmasked, single-group study with 41 enrolled participants. It concerns autologous stem cell transplant followed by consolidation and maintenance for participants aged 65 years or older with multiple myeloma or a related plasma cell malignancy. The recorded start date is 2013-04-04, and the primary completion date is 2020-09-30.
The primary outcome is median progression-free survival in months, defined from the start of DPACE to disease progression or death from any cause. The registry specifies a 95% confidence interval and a timeframe of up to 6 years, but the estimate and interval are not reported in the registry record. The record lists quality-of-life change as a secondary outcome and includes adverse-event summaries by treatment period.
No concurrent comparator is recorded. The primary numerical result, analysis methods, sample-size justification, missing-data approach, and analysis populations are not reported in the registry record. The record therefore describes the study question and design more fully than it documents the primary outcome result.