Selinexor (KPT-330) in Older Patients With Relapsed AML: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Selinexor (KPT-330) in Older Patients With Relapsed AML (NCT02088541), a randomized phase 2 study. The primary outcome is Overall Survival.

2. Clinical Question

The trial was randomized and open label. Its broad eligibility description concerns older patients with relapsed or refractory AML who were not considered candidates for intensive chemotherapy and/or transplantation. The primary outcome comparison reported in the registry is narrower: it covers participants randomized under protocol version 5 or later. The registry describes that intent-to-treat population as including all participants randomized to study treatment under those protocol versions, whether or not they received the assigned treatment.

The clinical question is therefore about the randomized comparison in that specified population, not a comparison across every participant enrolled in the full study. The primary endpoint is time to death from any cause. The registry also reports selected secondary measures related to survival and remission, but those outcomes address different questions and do not replace the primary survival comparison.

3. Trial Design

This was a randomized, multicenter, open-label, phase 2 study with parallel treatment groups. The registry lists total enrollment as 317 and study status as completed. The study began in 2014-03, and the primary completion date is 2018-01-08. The reported primary analysis concerns the protocol-version-5-or-later population rather than the full enrollment: 118 participants appear in the selinexor arm and 57 in physician’s choice 2.

For that primary comparison, selinexor was given as 60 mg, described as equivalent to 35 mg/m², twice weekly on days 1 and 3 of each week of a 28-day cycle. Physician’s choice 2 combined best supportive care with subcutaneous Ara-C, 20 mg twice daily for 10 days, repeated at 28 to 42 day intervals. The broader registry description says physician’s choice could involve one of three potential salvage therapies. The detailed arm descriptions include physician’s choice 1 and physician’s choice 2; the specific details of any other choice are not reported in the registry record.

Protocol versions affected eligibility and treatment descriptions. Before version 5, the described eligibility was one prior line of AML therapy; version 5 and later descriptions include different prior-line requirements. The study also lists earlier selinexor dosing groups and a physician’s choice 1 group. These are not the arms in the primary comparison described by the reported overall-survival result.

The registry identifies one participant randomized to selinexor under the later protocol version who was treated with physician’s choice 2. The overall-survival population description states that participants were analyzed according to randomization regardless of whether they received study treatment. Further details about the allocation procedure, planned enrollment rationale and original statistical design assumptions 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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Intent-to-treat (ITT) population included all participants who were randomized to study treatment under PV\>=5.0, regardless of whether or not they received study treatment. One participant was randomized to receive selinexor 60 mg (PV\>=5) but was treated with physician's choice 2. Hence, this participant was counted under selinexor 60 mg (PV\>=5).

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: Safety Population included all participants who received any amount of study treatment and was the primary population for the analysis of safety outcome measure.

6. Primary Endpoint

The registry lists the primary outcome as Overall Survival, measured over Baseline until disease progression or discontinuation from the study, or death, whichever occurred first (up to a maximum of approximately 104 weeks).

Overall survival measures the time from randomization to death from any cause. Participants last known to be alive were censored at the date of last contact, meaning their observed follow-up contributes information up to that point without treating them as having died. The registry gives the endpoint timeframe as baseline until disease progression or discontinuation from the study, or death, whichever occurred first, up to a maximum of approximately 104 weeks.

Time-to-event outcomes are used when the timing of an event matters and participants may have different lengths of follow-up. They can incorporate both the observed event times and censored follow-up, rather than treating every participant as if the same fixed observation period were available. Overall survival is a broad endpoint because death from any cause counts as the event, regardless of its attributed cause.

The posted primary result reports median overall survival for each arm and a hazard ratio from a log-rank analysis. The median describes the time by which half of the observed survival distribution has reached the event, subject to the analysis and follow-up. The hazard ratio compares event rates over follow-up under the assumptions of the time-to-event analysis; it is not a direct difference between the two reported medians. The confidence interval communicates uncertainty around that relative estimate.

The registry’s endpoint definition, analysis population and censoring rule are essential to interpreting the result. They do not establish that every participant was observed for the full stated timeframe, and the actual follow-up distribution is not reported in the registry record.

7. Results Posted in the Registry

• Selinexor 60 mg (PV >=5) (Equivalent to 35 mg/m^2) vs Physician's Choice 2 (PV >=5): hazard ratio 1.18 (95% CI 0.79–1.75), reported p = 0.422 (as reported in the registry).

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Percentage of Participants With Overall Survival of at Least 3 Months (OS3.0)

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Physician's Choice 2 (PV >=5): 70.2% (40/57) versus 53.4% (63/118) on control; risk difference 16.8 percentage points (95% CI 1.9 to 31.7), risk ratio 1.31 (1.04–1.67); unadjusted two-proportion z-test p = 0.034.
• Interpretation:
  • The 95% confidence interval excludes no difference, so the proportion with the event is statistically higher for Physician's Choice 2 (PV >=5) than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.

Percentage of Participants With Complete Remission Rate (CRR) for Those Who Achieved Complete Remission (CR)

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Physician's Choice 2 (PV >=5): 0.0% (0/57) versus 5.1% (6/118) on control; risk difference -5.1 percentage points (95% CI -9.0 to -1.1); unadjusted two-proportion z-test p = 0.083.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Physician's Choice 2 (PV >=5) and the control. The interval gives the range of effects that remain compatible with the data.

Percentage of Participants With Modified Complete Remission Rate (mCRR) for Those Who Achieved Complete Remission (CR) or Complete Remission With Incomplete Hematologic Recovery (Cri)

• Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Physician's Choice 2 (PV >=5): 3.5% (2/57) versus 11.9% (14/118) on control; risk difference -8.4 percentage points (95% CI -15.9 to -0.8), risk ratio 0.30 (0.07–1.26); unadjusted two-proportion z-test p = 0.072.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Physician's Choice 2 (PV >=5) and the control. The interval gives the range of effects that remain compatible with the data.

The registry lists a range of secondary outcomes, including survival at a fixed time, remission measures, response rates and disease-control measures. The posted results include selected survival and remission outcomes. Across these measures, the observed patterns are not uniformly in the same direction: the fixed-time survival measure is higher in the physician’s choice 2 group, while the posted remission measures show higher percentages in the selinexor group. These endpoints concern distinct aspects of the study and should not be treated as interchangeable confirmations of the primary survival result.

The primary endpoint is overall survival, a time-to-event outcome. A fixed-time survival percentage is a binary summary at a specified point, and remission outcomes assess response categories rather than time to death. Differences in outcome definition, follow-up and analysis mean that secondary measures can complement the primary result but cannot simply be combined into one overall conclusion.

The registry reports multiple secondary outcomes and multiple analyses. An unadjusted secondary p-value does not account for the possibility of chance findings when many outcomes are examined. Without a reported multiplicity plan, those p-values carry less inferential weight than a prespecified primary analysis. The registry’s multiplicity procedure, if any, is not reported in the registry record.

Secondary results are therefore best read as descriptive and supportive context. Their mixed pattern does not remove the uncertainty in the primary overall-survival estimate, and a nominal result on one secondary measure would not by itself settle the broader treatment comparison. The endpoint definitions, confidence intervals and sponsor-reported analyses remain important when considering each result.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Percentage of Participants With Overall Survival of at Least 3 Months (OS3.0)
• Percentage of Participants With Complete Remission Rate (CRR) for Those Who Achieved Complete Remission (CR)
• Median Disease-Free Survival (DFS) for Participants Who Achieved Complete Remission (CR)
• Percentage of Participants With Modified Complete Remission Rate (mCRR) for Those Who Achieved Complete Remission (CR) or Complete Remission With Incomplete Hematologic Recovery (Cri)
• Median Disease-Free Survival (DFS) for Participants Who Achieved Complete Remission or CR With Incomplete Hematologic Recovery (CRi) or Complete Remission With Incomplete Platelet Recovery (CRp)
• Percentage of Participants With Overall Response Rate (ORR)
• Duration of Response (DOR)
• Percentage of Participants With Disease Control Rate (DCR)

9. Statistical Interpretation

10. Statistical Methods Explained

The primary outcome is time to death from any cause, measured from randomization. The registry reports median survival, 95% confidence intervals for the arm-specific medians, and a sponsor-reported hazard ratio with a 95% confidence interval. The comparison method is listed as log rank.

What does censoring mean?

General explanation: In a time-to-event analysis, a participant is censored when the event has not been observed by the last available follow-up point. The analysis uses the observed time at risk up to that point, but does not treat the participant as having experienced the event. In this study, participants last known to be alive were censored at the date of last contact. This method relies on assumptions about how censoring relates to the event process; the registry’s detailed assessment of those assumptions is not reported in the registry record.

What does the median describe?

General explanation: A median time-to-event estimate is the time at which the estimated event-free proportion reaches one half. It is a summary of the survival distribution, not an estimate of how much longer an individual participant will live. The primary result reports medians of 94 days and 170 days for the two groups, with their respective confidence intervals. Comparing those medians alone does not account as fully for event timing across follow-up as a time-to-event comparison does.

What does a hazard ratio describe?

General explanation: A hazard ratio expresses the relative event rate between groups over the follow-up period, subject to the assumptions of the analysis. A value of 1 is the reference for equal hazards. Values on either side indicate different estimated directions, but the estimate must be read with its confidence interval and the analysis design. The reported estimate is 1.18, with a 95% confidence interval from 0.79 to 1.75. Because the interval includes 1, it does not rule out no relative difference, and its width shows that a range of effects remains compatible with the data.

The registry identifies the log-rank method for the primary comparison. General explanation: The log-rank test compares the observed timing of events between groups over follow-up, accounting for censored observations. It tests a null hypothesis of no difference in the survival experience under its assumptions; it does not itself supply a clinically meaningful measure of benefit. The hazard ratio gives a relative-effect estimate alongside that test. The registry does not report the detailed model specification or whether assumptions such as proportional hazards were checked.

How should the p-value be read?

General explanation: A p-value describes how unusual data at least as incompatible with a specified null hypothesis would be if that null hypothesis and the analysis assumptions were true. It is not the probability that the null hypothesis is true, nor does it measure the size or importance of an effect. The sponsor-reported primary p-value is 0.422. It should be read together with the hazard ratio and its confidence interval, not as a standalone verdict.

The trial was randomized and open label. Randomization supports a comparison based on assigned groups, while open-label treatment means participants and study personnel were not masked to treatment assignment. The registry does not report additional details about randomization procedures, stratification factors, missing-data methods beyond the censoring rule, or protocol deviations. These omissions limit how fully the analysis can be independently reconstructed from the registry record.

11. Confidence Intervals and Precision

The arm-specific median estimates are 94 days for selinexor, with a 95% confidence interval from 78 to 158 days, and 170 days for physician’s choice 2, with a 95% confidence interval from 111 to 220 days. The intervals convey uncertainty in the estimated median survival for each group; they are not ranges containing a fixed proportion of individual participants’ survival times.

The primary relative-effect estimate is a hazard ratio of 1.18, with a 95% confidence interval from 0.79 to 1.75. General explanation: A confidence interval is a range produced by a statistical procedure that, under its assumptions, reflects the precision of an estimate. A narrower interval indicates greater precision than a wider interval, while an interval that includes the null value leaves the direction and magnitude less certain. Here, the interval includes 1 and spans values on either side of it.

The reported p-value is 0.422. It is consistent with the uncertainty represented by the interval, but it does not quantify the probability that the treatment effect is absent. The registry does not report the number of deaths contributing to the primary analysis, the censoring pattern, or the number at risk at particular follow-up times. Those details would help readers understand how much information supports the survival estimates.

The confidence intervals should therefore be treated as central to interpretation rather than as decorative bounds around point estimates. The posted estimates do not identify a single precise relative effect, and they do not establish that the groups are equivalent.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The sample-size illustrations are planning calculations based on the observed effects, not the trial’s original design rationale. The original sample-size assumptions are not reported in the registry record. For the binary outcomes, the figures are participants per arm. For the time-to-event outcome, the figures are total events across the comparison, not participants per arm.

The planning assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. Each planning row considers an effect set at the stated fraction of the observed effect. The time-to-event calculations use the Schoenfeld approach. The binary-outcome calculations refer to participants per arm.

The 80% power column gives the planned number of participants per arm, or total events for the time-to-event comparison, for an 80% chance of rejecting the null hypothesis if the assumed effect and other planning assumptions hold. The 90% power column gives the corresponding planning requirement for a 90% chance under those assumptions. These are design illustrations, not descriptions of the study’s actual enrollment targets.

The rows that plan for smaller effects require more participants or events than rows based on a larger effect. That is because smaller differences are harder to distinguish from random variation with the same level of statistical certainty. These figures are not observed power; they do not tell whether the completed trial was adequately powered for its actual result.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

d is the total number of events needed (Schoenfeld's formula) with 1:1 allocation and proportional hazards.

Worked calculation for this trial

1. Hazard ratio HR = 1.180, so ln(HR) = 0.1655.
2. d = 4 × (1.96 + 0.842)² / [ln(1.180)]² = 1,146.0, rounded up to 1,147 events in total for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
hr <- 1.18                              # observed hazard ratio
events_needed <- function(power, frac = 1) ceiling(round(4 * (z_a + qnorm(power))^2 / log(hr^frac)^2, 6))   # Schoenfeld, 1:1 allocation
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: events = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(events_needed(0.80, f), big.mark = ","), format(events_needed(0.90, f), big.mark = ",")))

# Simulation: given d total events under proportional hazards, events in the treatment arm are Binomial(d, hr / (1 + hr)).
# The score (log-rank type) statistic is (D1 - d / 2) / sqrt(d / 4).
set.seed(2088541)
d    <- events_needed(0.80)
reps <- 20000
D1 <- rbinom(reps, d, hr / (1 + hr))
z  <- (D1 - d / 2) / sqrt(d / 4)
cat(sprintf("Simulated power with %s events: %.3f (%d replicates)\n", format(d, big.mark = ","), mean(abs(z) > z_a), reps))

Output from running the code

100% of observed effect: events = 1,147 (80% power), 1,535 (90% power)
 75% of observed effect: events = 2,038 (80% power), 2,728 (90% power)
 50% of observed effect: events = 4,585 (80% power), 6,137 (90% power)
Simulated power with 1,147 events: 0.803 (20000 replicates)

The simulation uses the fact that, with d events in total and proportional hazards, the number of events in the treatment arm is binomial. It is an approximation that conditions on the total number of events, and it should land close to the target power.

14. Analyses Reported by the Sponsor

The sponsor-reported primary analysis gives a hazard ratio of 1.18, with a 95% confidence interval from 0.79 to 1.75, and a log-rank p-value of 0.422. The accompanying recalculation uses the posted summary result and does not produce a discrepancy flag. The sponsor’s reported analysis remains the primary inferential result to cite.

A recalculation from posted summary statistics can be useful as a check or as an illustration, but it is not a substitute for the trial’s own analysis. It may not reproduce all details of the sponsor’s analysis, including data handling or model choices not captured by summary results. No discrepancy is flagged for the primary comparison, so there is no conflict to resolve between the reported estimate and a recalculated estimate in this case.

Some secondary results have both sponsor-reported tests and simple unadjusted recalculations. Those may differ because the reported analysis method and the recalculation need not use the same assumptions or test procedure. Where they differ, the sponsor’s own analysis takes precedence for describing the sponsor-reported result. The recalculations should be understood as unadjusted checks based on posted summary counts, not as replacements for the registry’s reported analyses.

Neither a sponsor-reported p-value nor a recalculated p-value establishes the probability that a treatment effect is true. Both require interpretation alongside the estimated effects, confidence intervals, analysis populations and multiplicity context.

15. Multiplicity, Missing Data and Analysis Populations

The registry record reports a primary endpoint and several secondary outcomes, but the planned approach to multiplicity is not reported in the registry record. This matters because each additional outcome or comparison creates another opportunity for an apparently noteworthy result to arise by chance. A prespecified adjustment or hierarchy can help control that issue; whether the trial used such an approach cannot be established from the registry record.

Consequently, secondary p-values should not be interpreted as if each were the sole confirmatory test. This is especially important where the registry includes distinct survival, remission and response outcomes, and where sponsor-reported tests and simple unadjusted recalculations may use different methods. The primary overall-survival analysis remains the main inferential result described by the registry.

The primary population is described as intent to treat for participants randomized under protocol version 5 or later, regardless of whether they received study treatment. The registry also notes that one participant assigned to selinexor received physician’s choice 2. This indicates that the primary comparison is organized by randomized assignment as described, rather than by treatment received. The details of any additional analysis populations are not reported in the registry record.

Participants last known to be alive were censored at their last contact for overall survival. Other details about missing observations, follow-up completeness, reasons for loss to follow-up, sensitivity analyses or imputation procedures are not reported in the registry record. The pattern and amount of missing data could affect precision and interpretation, but cannot be assessed from the posted information.

The registry record also does not report the original sample-size justification, interim analyses, or a complete statistical analysis plan. These are important details for assessing how outcomes and analyses were selected and whether the published approach followed a prespecified plan. Their absence from the registry record limits assessment; it does not establish that such procedures were or were not used.

16. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: febrile neutropenia (35.6%), pneumonia (14.8%), nervous system disorders (13.0%), sepsis (11.3%), lung infection (7.7%).

The registry lists serious adverse-event information across five treatment categories. For the selinexor 60 mg group under protocol version 5 or later, 115 participants were at risk for serious adverse events and 89 were affected. The primary efficacy population lists 118 participants in that arm; the different figures reflect the denominators presented for these separate registry entries and should not be silently substituted for one another.

The highest listed serious adverse-event terms include febrile neutropenia at 35.6%, pneumonia at 14.8%, nervous system disorders at 13.0%, sepsis at 11.3%, lung infection at 7.7% and dehydration at 7.0%. These are registry-listed terms and percentages; the information presented does not establish that each percentage applies specifically to the primary comparison population. The record also lists serious-event counts and deaths for other treatment categories.

Serious adverse-event figures describe events recorded in the study and should be read with their corresponding at-risk populations and treatment categories. They do not by themselves establish that an event was caused by treatment, and the figures should not be compared across differently defined groups without the relevant context. Further details about event definitions, timing, severity breakdowns and causal attribution are not reported in the registry record.

17. What the Result Does, and Does Not, Mean

The primary result describes an uncertain comparison of overall survival between selinexor and physician’s choice 2 in the registry’s later-protocol intent-to-treat population. The posted hazard ratio is 1.18, with a 95% confidence interval from 0.79 to 1.75 and a log-rank p-value of 0.422. The interval includes the null value for a hazard ratio and covers a range of possible relative effects. The result therefore does not establish a clear difference, but it also does not demonstrate equivalence.

The medians—94 days for selinexor and 170 days for physician’s choice 2—are descriptive estimates with their own uncertainty. They should not be interpreted as a direct estimate of how much one treatment changes survival compared with the other. The hazard ratio and its interval summarize the time-to-event comparison differently from the two medians.

The secondary outcomes add context about survival at a fixed point and remission measures, but they answer different questions. Their mixed observed pattern and the absence of a reported multiplicity plan mean that isolated secondary findings should not be used to make a definitive overall claim. The available record does not provide every detail needed to assess the full statistical analysis plan, follow-up completeness or missing-data sensitivity.

These results describe the posted registry analyses for the stated population and study design. They do not provide individual predictions, establish what would happen for a particular patient, or answer questions about populations outside the trial’s eligibility criteria. They also do not establish causation for individual adverse events or determine the clinical importance of each observed difference. The findings should be understood as estimates with uncertainty, not as a categorical judgment about either treatment.

18. Why This Trial Matters Statistically

This trial illustrates why a time-to-event result should be read through more than its point estimate. The overall-survival medians and hazard ratio present different summaries of the same broad endpoint, while the confidence interval communicates that the relative-effect estimate remains imprecise. A p-value alone would omit much of that information.

It also shows why the analysis population and protocol version matter. Total enrollment was 317, but the primary comparison concerns participants randomized under protocol version 5 or later, with 118 in the selinexor group and 57 in physician’s choice 2. The full trial enrollment is not the denominator for every reported comparison. One participant’s treatment differed from randomized assignment, while the primary population is described according to randomized treatment regardless of treatment received.

The study’s open-label design and multiple outcome measures provide further statistical context. Open-label treatment can be relevant when interpreting outcomes and study procedures, while multiple secondary analyses raise questions about multiplicity. The registry does not report a multiplicity plan, the original sample-size justification or a complete analysis plan, so those aspects cannot be fully evaluated from the record.

Finally, planning calculations based on observed effects should not be confused with actual power or with the trial’s original design. They illustrate how the required number of participants or events changes with the effect a future study aims to detect. Keeping those distinctions clear helps prevent descriptive results, planning examples and confirmatory inference from being conflated.

19. 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 survival comparison covers the later-protocol population, not all 317 enrolled participants. Protocol versions changed eligibility and treatment descriptions, so results from the later-version comparison should not be generalized automatically to the earlier treatment groups or to the entire enrollment. The registry’s primary population description is partly truncated, limiting the detail available about its exact eligibility wording.

The primary arms are unequal in size, with 118 participants in selinexor and 57 in physician’s choice 2. The registry also identifies one participant assigned to selinexor who received physician’s choice 2. The stated intent-to-treat approach addresses the analysis by randomized assignment, but the record does not provide enough detail to assess other treatment deviations or their handling.

The study was open label. The registry does not provide details about whether knowledge of treatment assignment affected assessment, follow-up or decisions to discontinue, so the potential relevance of open-label conduct cannot be quantified from the posted record. The registry also does not report the survival curve, numbers at risk over time, event counts for the primary comparison, or checks of time-to-event model assumptions.

Several analysis details remain unreported in the registry record, including the original sample-size rationale, interim analyses, the multiplicity approach, a complete statistical analysis plan and fuller missing-data information. The secondary outcomes are numerous, and the posted secondary results should be interpreted cautiously where adjustment for multiple testing is not described. Serious adverse-event information is presented across treatment categories, but the registry excerpt does not give all details needed to compare event patterns within the primary efficacy population.

20. Related Tutorials

21. Related Statistical Calculators

22. Sources

• ClinicalTrials.gov: Selinexor (KPT-330) in Older Patients With Relapsed AML — NCT02088541.
• Registry record last updated 2023-01-26; status last verified 2023-01.
• Results first posted 2020-10-08.
• 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.

23. Record Summary

The registry record describes a completed, randomized, open-label, multicenter phase 2 study of selinexor versus specified physician’s choice in older patients with relapsed or refractory AML who were considered unfit for intensive chemotherapy and/or transplantation. Total enrollment was 317.

The primary comparison reported for participants randomized under protocol version 5 or later included 118 participants assigned to selinexor and 57 assigned to physician’s choice 2. Median overall survival was 94 days and 170 days, respectively. The sponsor-reported hazard ratio was 1.18, with a 95% confidence interval from 0.79 to 1.75 and a log-rank p-value of 0.422.

The estimate is uncertain, and the confidence interval includes the null value. The registry also lists secondary outcomes and serious adverse-event information, while several analysis-plan details—including the multiplicity approach and original sample-size rationale—are not reported in the registry record.