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 Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias (NCT02443545), a randomized phase 4 study. The primary outcome is Number of Patients With Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02443545 |
| Phase | Phase 4 |
| Condition | Iron Overload, Sickle Cell Disease, Other Anemias |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 134 |
| Arms in the primary outcome | n/a |
| Lead sponsor | ApoPharma |
| Results posted | Yes |
2. Clinical Question
Population
Patients with iron overload associated with sickle cell disease or other anemias, in the setting of regular blood transfusions. The study is described as a long-term follow-up to an earlier study, LA38-0411. The primary safety population is defined as enrolled patients who took at least one dose of study drug.
Intervention
Deferiprone, also identified by the brand name Ferriprox®. The extension study followed patients receiving deferiprone for different durations, depending on their original assignment in LA38-0411.
Comparator
The record describes two groups based on randomization in LA38-0411: patients originally assigned to deferiprone and patients originally assigned to deferoxamine. Both groups received deferiprone in the extension. Thus, the extension does not describe a concurrent group receiving a different treatment.
Primary question
How many patients experienced at least one adverse event, a serious adverse event, or withdrawal from the study due to an adverse event during follow-up? The primary outcome is a count of participants with events, rather than a measure of change in iron burden or a direct treatment-effect comparison.
The registry record describes the study as a long-term safety and efficacy follow-up in people with transfusional iron overload. Its primary endpoint is safety-focused: it counts participants with adverse events over the period from the first study day through the last study visit, defined as Week 104 or early termination. The outcome description includes adverse events of any type, serious adverse events, and withdrawals due to an adverse event.
The treatment-history distinction matters when interpreting the groups. One group had been assigned to deferiprone in LA38-0411 and was described as receiving deferiprone for a total of 3 years across the initial and extension studies. The other group had been assigned to deferoxamine in LA38-0411 and was described as receiving deferiprone for 2 years in the extension. Because both extension groups received deferiprone, their labels reflect prior treatment assignment and duration of exposure, not a continuing comparison between deferiprone and deferoxamine.
The record also names changes in liver iron concentration, cardiac MRI T2*, and serum ferritin as secondary outcomes. Results for those outcomes are not reported in the registry record. The posted primary results therefore address event counts and do not, on their own, answer a comparative efficacy question.
3. Trial Design
This is a Phase 4, randomized, parallel-group study with no masking. The registry lists enrollment of 134 and describes the study as a long-term follow-up to LA38-0411. The sponsor is ApoPharma. The recorded start date is 2015-05-21, primary completion date is 2019-04-30, and status is terminated. The reason for termination is not reported in the registry record.
The extension groups were formed according to randomization in the earlier study. Group 1 comprised patients randomized to deferiprone in LA38-0411; the extension description says they received deferiprone for a total of 3 years, including 1 year in the initial study and 2 years in the extension. Group 2 comprised patients randomized to deferoxamine in LA38-0411; the description says they received deferiprone for 2 years in the extension. In this study, therefore, the earlier assignment distinguishes treatment history and planned duration of deferiprone exposure.
Although the extension is labeled randomized, the registry description does not present it as a new random allocation between deferiprone and another treatment. Both described groups received deferiprone during the extension. The listed adverse-event results contain a single Deferiprone group, and the primary outcome has no two-arm comparison. The registry record does not report a concurrent untreated or active-comparator group for these posted results.
The primary outcome timeframe runs from the first day of the study until the last study visit, specified as Week 104 or early termination. The safety population is defined as all enrolled patients who took at least one dose of study drug. The record does not report the number who met this definition separately from enrollment, or the number contributing to each specific adverse-event category beyond the posted at-risk figures.
Randomization in the earlier study can help explain how treatment histories arose, but it does not make the extension's single-group adverse-event counts a randomized estimate of a deferiprone-versus-deferoxamine effect during the extension. The record does not report details of allocation concealment, a masking procedure, or a new randomization process for the extension. These details are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Group 1: Deferiprone 3 Years | Group 2: Deferiprone 2 Years |
|---|---|---|
| STARTED | 89 | 45 |
| COMPLETED | 50 | 26 |
| NOT COMPLETED | 39 | 19 |
Reasons for not completing this period:
| Reason | Group 1: Deferiprone 3 Years | Group 2: Deferiprone 2 Years |
|---|---|---|
| Adverse Event | 1 | 1 |
| Lost to Follow-up | 0 | 2 |
| Physician Decision | 3 | 0 |
| Protocol Violation | 8 | 1 |
| Pregnancy | 1 | 1 |
| Withdrawal by Subject | 6 | 3 |
| Termination of study | 20 | 11 |
Analysis population for the primary outcome, as defined in the registry: Safety population (all enrolled patients who took at least one dose of study drug)
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 | Deferiprone |
|---|---|---|
| Participants analysed | 134 | |
| Age, Continuous | years | 16.2 (SD 8.6) |
| Sex: Female, Male: Female | Participants | 53 |
| Sex: Female, Male: Male | Participants | 81 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 133 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| 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 | 20 |
| Race (NIH/OMB): White | Participants | 114 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Saudi Arabia | participants | 5 |
| Region of Enrollment: Canada | participants | 2 |
| Region of Enrollment: United States | participants | 14 |
| Region of Enrollment: Egypt | participants | 109 |
| Region of Enrollment: United Kingdom | participants | 4 |
| Liver iron concentration at baseline | mg iron per gram of liver dry weight | 14.93 (SD 7.61) |
| Cardiac iron at baseline | milliseconds | 32.69 (SD 17.66) |
| Serum ferritin level at baseline | micrograms per liter | 3894 (SD 2591) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Adverse Events, measured over From the first day of the study until the last study visit (Week 104 or early termination).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events during follow-up. The registry description specifies three types of information: participants with at least one adverse event of any type, participants with at least one serious adverse event, and participants who withdrew because of an adverse event. The outcome is a participant count, not a count of every event episode. One participant could experience more than one event, so a count of people with an event should not be read as the total number of event occurrences.
Safety endpoints are used to describe the kinds of harms observed and how many participants were affected during the study period. Reporting serious events separately can distinguish events classified as serious from the broader category of adverse events. A withdrawal attributed to an adverse event provides another aspect of tolerability, although the withdrawal count is not included in the posted figures described in the record.
The timeframe is from the first study day to the last study visit, which could be Week 104 or early termination. This means participants may have different observed follow-up durations. The registry record does not report an exposure-adjusted event rate or an analysis accounting for differing time at risk. The endpoint is also not a direct comparison of treatment effects: the posted adverse-event results list one Deferiprone group, and both extension groups received deferiprone.
Changes in liver iron concentration, cardiac MRI T2*, and serum ferritin are listed as secondary outcomes, but their results are not reported in the registry record. The primary endpoint should therefore be interpreted as a description of safety event counts, not as evidence about changes in those measures or comparative efficacy.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted adverse-event results list 134 participants at risk in the Deferiprone group. Within that group, 35 are listed in the serious adverse-event category and 99 in the other adverse-event category. The record also lists 1 death. These are descriptive counts from a single listed treatment group; they do not estimate how event frequency would differ under another treatment during the extension.
The primary outcome description includes withdrawals due to adverse events, but a withdrawal count is not included in the posted figures described in the record. Nor does the record provide an outcome comparison between the two treatment-history groups. It is therefore not possible to use these counts to compare the safety of deferiprone with deferoxamine during the extension, or to establish whether event frequency differed by prior assignment or exposure duration.
The listed serious-event terms include Sickle cell anaemia with crisis at 14.2%, Neutropenia at 9.0%, Pyrexia at 3.7%, Cholecystectomy at 2.2%, Agranulocytosis at 1.5%, and Pneumonia at 1.5%. These percentages describe terms as recorded in the registry, but the denominator and the precise calculation basis for each percentage are not reported in the registry record. They should not be interpreted as percentages of all enrolled patients without that information. A listed event term also does not establish that the study drug caused the event.
In general, a confidence interval communicates the range of effect estimates compatible with a statistical analysis and its assumptions. No confidence interval or treatment-effect estimate for an adverse-event comparison is reported in the registry record. Without a comparison group and a corresponding uncertainty estimate, the posted counts describe what was recorded in this group but do not quantify a between-treatment difference or its precision.
The registry status is terminated, but the reason is not reported in the registry record. Termination status alone does not explain whether follow-up ended as planned, whether the study stopped early for a safety concern, or whether other circumstances led to termination. Those possibilities cannot be distinguished from the status field alone.
Overall, the posted results provide a safety-event account for participants exposed to deferiprone in the extension. Their interpretation is limited by the single listed adverse-event group, the absence of a reported comparative analysis, incomplete reporting of some endpoint components, and the lack of stated denominators for the serious-event term percentages. They do not establish a causal relationship between deferiprone and any individual event, and they do not answer the secondary efficacy questions named in the record.
9. Statistical Methods Explained
What does a participant count represent?
The primary outcome is a count of participants who experienced specified adverse events. This differs from counting event episodes: if a participant experiences several events, a participant-level endpoint can still count that person once within a defined category. Counts are useful for describing how many people were affected, but they do not show how many total episodes occurred, how long each person was observed, or how event timing varied.
The record defines the safety population as enrolled patients who took at least one dose of study drug. This definition focuses safety reporting on people with exposure. It is distinct from an intention-to-treat population, which generally retains participants according to their randomized assignment whether or not they received treatment. The registry record does not report an intention-to-treat analysis for the posted adverse-event outcome.
Why does the comparison structure matter?
In general, a randomized comparison estimates the difference between groups assigned to different strategies, subject to the design and analysis assumptions. Here, the extension descriptions identify groups by their randomization in LA38-0411, but both groups received deferiprone in the extension. The posted adverse-event results list one Deferiprone group rather than a two-group comparison. Accordingly, the results can describe events in the listed group but cannot provide a comparative treatment effect for deferiprone versus deferoxamine during the extension.
The earlier randomization remains relevant to the history of the participants, but the fact that a study is labeled randomized should not be mistaken for evidence that the posted extension outcome compares two concurrently randomized treatment groups. The registry record does not report a new randomization between different extension treatments, nor a statistical analysis comparing the two histories.
What would a rate or time-to-event analysis add?
In general, a simple count does not account for how long each participant was observed. If follow-up duration varies, exposure-adjusted rates can incorporate person-time, and time-to-event methods can use information about when an event first occurred while handling incomplete follow-up under stated assumptions. The recorded timeframe allows the last visit to be Week 104 or early termination, but an exposure-adjusted rate or time-to-event analysis for adverse events is not reported in the registry record. The posted figures should therefore be read as counts, not as rates or time-to-event estimates.
In general, event categories also need clear definitions. A serious adverse event is a classification of an event, while an adverse event of any type is a broader endpoint. The record reports participant counts under serious and other adverse-event categories, but further detail about the coding rules, overlap between categories, and handling of repeated events is not reported in the registry record. The distinction matters because category counts need not represent mutually exclusive groups of people unless the analysis specifies that they do.
How should uncertainty be assessed?
In general, an estimate is more informative when its uncertainty is reported. A confidence interval communicates the range of values reasonably compatible with the data and the statistical model; it does not guarantee that the true value lies in that range. For a comparison, an interval around a difference or ratio can indicate both the direction and precision of the estimated contrast. No such comparative estimate or interval is reported for the primary safety endpoint.
A p-value, in general, summarizes how unusual data at least as incompatible with a specified null hypothesis would be under the assumptions of a statistical test. It does not measure the probability that a treatment works, nor does it measure clinical importance. A p-value for the primary adverse-event comparison is not reported in the registry record. In this setting, a test of a treatment contrast would also require a comparison structure and analysis that are not present in the posted results.
What can the planning illustration tell readers?
The registry record describes a sample-size planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says that the effect to detect is a stated fraction of the observed effect. The illustration is not the trial's own design justification, and the record does not report the original sample-size justification. A planning calculation is not observed power: it describes a hypothetical design under stated assumptions, rather than the probability that this completed study detected an effect.
For participant-count outcomes, planning figures are described as participants per arm; time-to-event planning figures are described as total events. The primary outcome has fewer than two arms in the posted results, so a two-arm planning illustration does not create a valid comparative analysis of these adverse-event counts. The numerical planning results are not repeated in this explanation; their interpretation depends on the assumptions stated in the registry record and should not be confused with observed trial findings.
What analysis details remain unknown?
The registry record does not report the statistical test or model used for the posted primary outcome, any adjustment variables, the handling of repeated events, or a treatment-effect estimate with uncertainty. It also does not report a prespecified statistical analysis plan in the information. Without those details, the most defensible interpretation is descriptive: the registry gives participant counts and selected event terms, but does not establish a comparative effect or its precision.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety outcome and names secondary outcomes for change from baseline in liver iron concentration, cardiac MRI T2*, and serum ferritin. Results for those secondary outcomes are not reported in the registry record. The record also lists four outcome measures and nine analyses, but details of how those analyses relate to specific hypotheses are not reported in the registry record.
When a study examines multiple outcomes or multiple analyses, the chance of at least one apparently notable result can increase. In general, multiplicity procedures can control error rates across a defined family of tests, while a prespecified hierarchy can establish which questions are confirmatory. The registry record does not report a multiplicity adjustment, a testing hierarchy, or how the analyses were grouped for such purposes. No conclusion about whether multiplicity was addressed can be drawn from the listed outcome and analysis counts alone.
For missing data, the record defines the safety population as all enrolled patients who took at least one dose of study drug. It does not report the number of participants with incomplete follow-up, the amount or pattern of missing outcome information, or a method for handling missing data. Because follow-up could end at Week 104 or early termination, the amount of observed follow-up may differ among participants; how that was handled analytically is not reported in the registry record.
The record does not report whether all safety outcomes were assessed for every participant in the safety population, whether any participants were excluded from particular analyses, or whether sensitivity analyses were conducted. The withdrawal count due to an adverse event is part of the primary outcome description, but the corresponding posted count is not included in the record details. These reporting gaps limit assessment of completeness and robustness.
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 |
|---|---|---|---|
| Deferiprone | 35 | 134 | 26.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Deferiprone | 1 of 134 |
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 |
|---|---|
| Deferiprone | 99 of 134 |
Most frequent serious adverse event terms
| Event term | Organ system | Deferiprone |
|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | 19 of 134 |
| Neutropenia | Blood and lymphatic system disorders | 12 of 134 |
| Pyrexia | General disorders | 5 of 134 |
| Cholecystectomy | Surgical and medical procedures | 3 of 134 |
| Agranulocytosis | Blood and lymphatic system disorders | 2 of 134 |
| Pneumonia | Infections and infestations | 2 of 134 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 2 of 134 |
| Splenectomy | Surgical and medical procedures | 2 of 134 |
| Hypotension | Vascular disorders | 2 of 134 |
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 134 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Deferiprone |
|---|---|---|
| Bone pain | Musculoskeletal and connective tissue disorders | 35 of 134 |
| Pyrexia | General disorders | 33 of 134 |
| Abdominal pain | Gastrointestinal disorders | 26 of 134 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 18 of 134 |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 18 of 134 |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | 17 of 134 |
| Nasopharyngitis | Infections and infestations | 17 of 134 |
| Back pain | Musculoskeletal and connective tissue disorders | 17 of 134 |
| Neutrophil count decreased | Investigations | 16 of 134 |
| Headache | Nervous system disorders | 12 of 134 |
Serious events with the highest arm-level rate: sickle cell anaemia with crisis (14.2%), neutropenia (9.0%), pyrexia (3.7%), cholecystectomy (2.2%), agranulocytosis (1.5%).
The registry lists 35 participants in the serious adverse-event category and 99 in the other adverse-event category, with 134 at risk in the Deferiprone group. These figures are participant counts, not a count of every adverse-event episode. The record also lists 1 death. It does not report whether the death was considered related to treatment, nor does it provide additional details about its cause in the information.
Several serious-event terms are listed with percentages: Sickle cell anaemia with crisis, 14.2%; Neutropenia, 9.0%; Pyrexia, 3.7%; Cholecystectomy, 2.2%; Agranulocytosis, 1.5%; and Pneumonia, 1.5%. The denominator and calculation basis for these term percentages are not reported in the registry record. They should therefore be read as registry-listed percentages, not assumed to be percentages of all participants. The listed terms do not establish causation.
The endpoint description includes withdrawals due to adverse events, but a corresponding count is not reported in the registry record. The posted data are descriptive and do not compare serious-event risk between treatments during the extension.
12. What the Result Does, and Does Not, Mean
The record supports a description of adverse events recorded among participants in the listed Deferiprone group during the study follow-up period. It reports participant counts for serious and other adverse-event categories, a death count, and selected serious-event terms with percentages. The endpoint definition also identifies withdrawals due to adverse events as an outcome of interest, although the corresponding withdrawal count is not reported in the registry record.
These observations do not establish that deferiprone caused the listed events. In a clinical study, events can occur during treatment without being caused by treatment; a causal assessment requires an appropriate design and analysis, along with clinical review. The posted counts also do not show what would have happened to similar participants under a concurrent alternative treatment during the extension. Both described extension groups received deferiprone, and the adverse-event results list a single group.
The counts do not provide a measure of event timing or participant-level exposure duration. The follow-up period could end at Week 104 or early termination, and the record does not report an exposure-adjusted rate or time-to-event analysis. The percentages attached to serious-event terms also lack a reported denominator in the registry record, so their population basis cannot be confirmed from the information available.
The results do not answer whether liver iron concentration, cardiac MRI T2*, or serum ferritin changed, because results for those secondary outcomes are not reported in the registry record. They also do not provide a comparative efficacy estimate, a confidence interval for a treatment difference, or a p-value for the primary safety outcome. The statistical precision of any between-treatment effect is therefore not quantified.
In general, safety results are most informative when considered alongside the endpoint definitions, duration and completeness of follow-up, comparator structure, and uncertainty estimates. The posted record offers a descriptive safety account, but it does not support conclusions about comparative safety or efficacy. The terminated status is recorded, while the reason for termination is not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically informative because it illustrates a distinction between a randomized study label and the comparison actually represented by posted results. The registry describes randomization in the earlier study and an extension with two treatment-history groups. During the extension, both groups received deferiprone. The posted primary safety results list one Deferiprone group, so they describe event counts rather than a randomized treatment contrast.
That distinction is important for interpretation. A participant count can summarize the observed burden of events in a group, but it does not by itself say whether the count is higher or lower than it would have been under another treatment. A comparison requires a suitable concurrent comparator, a clearly defined estimand, and an analysis that accounts for the study design. Those elements are not reported for a comparative primary safety analysis in the registry record.
The record also shows why outcome definitions and denominators matter. The primary endpoint includes several kinds of safety information, but the posted figures do not include the withdrawal count. Selected serious-event terms have percentages, yet the basis for those percentages is not reported. Without clear denominators and outcome-category details, readers should avoid interpreting the term percentages as participant-level risks.
Finally, the registry's planning illustration is distinct from the trial's actual design and results. Planning assumptions can help illustrate how a hypothetical study might be sized, but they are not observed power and do not substitute for an estimated effect with an uncertainty interval. In this record, the key statistical contribution is descriptive: it makes clear what was counted, while leaving important comparative and precision questions unanswered.
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 posted primary safety results list one Deferiprone group, although the extension description distinguishes patients by assignment in LA38-0411. Both groups received deferiprone during the extension. Consequently, the adverse-event results do not provide a concurrent treatment comparison, and differences attributable to prior assignment or duration of treatment cannot be evaluated from the posted counts.
The primary outcome covers adverse events from the first study day until Week 104 or early termination. Participants may therefore have different follow-up durations, but the record does not report person-time, exposure-adjusted rates, or an analysis of time to first event. The registry also does not report the number with incomplete follow-up or how missing safety information was handled.
The outcome description includes participants with any adverse event, participants with a serious adverse event, and withdrawals due to an adverse event. The posted details give counts in serious and other adverse-event categories and list a death, but do not include a withdrawal count. Further details about whether categories overlap, event coding, repeated events, and adjudication are not reported in the registry record.
The serious-event term percentages are listed without a reported denominator or calculation basis. Their interpretation as participant percentages or event percentages is therefore uncertain. The record does not report a comparative estimate, confidence interval, or p-value for the primary endpoint, and results for the named secondary outcomes are not reported in the registry record.
The study is marked terminated, but the reason for termination is not reported in the registry record. The registry also does not report the original sample-size justification, details of a statistical analysis plan, or a multiplicity strategy. These limitations constrain assessment of the study's statistical precision, analytic decisions, and generalizability beyond the described population and follow-up.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias — NCT02443545.
- Registry record last updated 2024-01-10; status last verified 2023-12.
- Results first posted 2024-01-10.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT02443545 is a Phase 4, randomized, open-label, parallel-group study sponsored by ApoPharma. It enrolled 134 patients and is described as a long-term follow-up to LA38-0411 in patients with transfusional iron overload associated with sickle cell disease or other anemias. Both extension groups received deferiprone, with treatment duration depending on prior assignment in the earlier study.
The primary outcome counts participants with adverse events, serious adverse events, and withdrawals due to adverse events over follow-up through Week 104 or early termination. Posted results list 134 at risk in the Deferiprone group, 35 in the serious adverse-event category, 99 in the other adverse-event category, and 1 death. Selected serious-event terms are listed with percentages, but their denominators are not reported in the registry record.
The registry lists changes in liver iron concentration, cardiac MRI T2*, and serum ferritin as secondary outcomes; their results are not reported in the registry record. Study status is terminated, and the reason is not reported in the registry record. The posted primary results are descriptive and do not provide a comparative treatment-effect estimate.