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 An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families (NCT04723693), a registered phase not stated study. The primary outcome is Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04723693 |
| Phase | Phase not stated |
| Condition | Hemophilia A With Inhibitor |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Haemnet |
| Results posted | Yes |
2. Clinical Question
Population
People with haemophilia A and inhibitors using emicizumab in clinical practice, together with close family members such as parents, carers, children, partners, or siblings. The study was designed for English-speaking patients and families. The registry describes 16 people with haemophilia and inhibitors as interviewed, with 12 having family members take part.
Intervention
Use of emicizumab for haemophilia inhibitor therapy. Participants could be receiving emicizumab through early access to medicine, after taking part in clinical trials, or as part of routine haemophilia care. The study did not assign treatment; it explored experiences of people already using emicizumab.
Comparator
No concurrent comparison group is described. The interviews included discussion of previous treatment and current treatment with emicizumab, but the registry does not report a controlled comparison or a standardized before-and-after assessment.
Primary question
How do participants and their families describe the experience of emicizumab use, including any perceived change in the burden of haemophilia and its treatment? Topics included bleeding, pain, treatment burden, control, freedom, and missed opportunities. The study sought narratives and themes rather than a numerical estimate of treatment effect.
3. Trial Design
This is a prospective, observational cohort qualitative research study. Its stated aim was to capture the real-life experience of people with haemophilia and inhibitors using emicizumab, as well as the impact of treatment on close family members. Participants and family members were considered a study dyad. Because treatment was observed in routine practice rather than assigned by investigators, the record does not describe a randomized trial or a treatment-control comparison.
The intervention listed is a qualitative interview. Each participant and family member was to take part in a one-hour semi-structured interview covering experiences of the condition, previous treatment, and current treatment with emicizumab. The interviews were intended to let English-speaking patients and families tell their own life stories. The narratives were to be thematically analysed and reported.
The registry lists an enrollment of 30 and a completed status. It reports that 16 people with haemophilia and inhibitors were interviewed, and that 12 had family members take part. Of the interviews, 14 took place online, one by telephone, and one in person. The registry record does not explain how the enrollment count relates to the number interviewed or describe reasons for any difference. It also does not report recruitment procedures, participant characteristics beyond the condition and treatment context, or the number and roles of interviewers.
The study start date is 2020-02-28 and the primary completion date is 2021-01-31. Haemnet is listed as the sponsor. The record identifies treatment satisfaction, treatment expectations, and treatment impact among the secondary outcomes. It does not report a protocol-level statistical analysis plan or a quantitative efficacy comparison.
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.
Before assignment: Individual participants and a family member (if available and consented) were interviewed as a dyad pair. Individual participants with no available family member were interviewed on their own
Overall Study
| Stage | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| STARTED | 30 |
| COMPLETED | 28 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| Withdrawal by Subject | 2 |
Analysis population for the primary outcome, as defined in the registry: 16 People with Haemophilia and inhibitors were interviewed 12 with family members 14 interviews took place online, one by telephone and one in person. 6 themes emerged: bleeds; pain; treatment burden; control; freedom (for both PwHi and family members) and missed potential.
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: 16 people with haemophilia and inhibitors and 12 family members
| Characteristic | Unit | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|---|
| Participants analysed | 28 | |
| Age, Categorical: <=18 years | Participants | 9 |
| Age, Categorical: Between 18 and 65 years | Participants | 19 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 12 |
| Sex: Female, Male: Male | Participants | 16 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: United Kingdom | participants | 28 |
6. Primary Endpoint
The registry lists the primary outcome as Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities), measured over Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome concerns reduction in the burden of the condition, including treatment burden, bleed frequency, pain, control, freedom, and missed opportunities. In this study, the endpoint is approached through participants’ accounts rather than through a reported numeric scale or a count of clinical events. The record describes interviews about the condition, previous treatment, and current emicizumab treatment, followed by thematic analysis.
This approach is used to understand how participants and family members describe their experiences and what aspects of daily life they consider important. It can capture meanings and concerns that may not be represented by a single numerical measure. The registry reports six themes: bleeds; pain; treatment burden; control; freedom, for both people with haemophilia and family members; and missed potential.
The theme labels identify topics that arose in the analysis; they are not, by themselves, measurements of how much burden changed. The registry record does not report a validated burden scale, a pre-specified numeric threshold for improvement, or counts of participants endorsing each theme. It also does not report a quantified reduction in bleeding, pain, or treatment burden. The primary endpoint therefore describes the subject of the interviews and the themes reported, rather than a numerical treatment-effect estimate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted results describe experiences, not a controlled estimate of the effect of emicizumab. The study’s observational qualitative design is suited to exploring how participants and family members make sense of treatment and its place in daily life. It cannot, on the information reported, establish how those experiences compare with what would have happened under another treatment or without emicizumab.
The registry reports six themes: bleeds, pain, treatment burden, control, freedom, and missed potential. These topics indicate what the thematic analysis identified as relevant in the narratives. Their presence does not establish that every participant experienced each issue, that the issues changed in the same direction for everyone, or that a particular theme was more common or important than another. Theme frequencies, quotations, and participant-level patterns are not reported in the registry record.
The primary outcome title refers to reduction in burden, but the registry’s description is qualitative. There is no numeric estimate of change in bleed frequency, pain, control, freedom, missed opportunities, or treatment burden. Consequently, there is no reported effect size or confidence interval with which to judge the magnitude or precision of a change. A confidence interval, as a general statistical concept, expresses uncertainty around a numerical estimate; it is not a substitute for detailed qualitative evidence and is not reported for this outcome.
The registry lists an enrollment of 30, while its primary-outcome description says that 16 people with haemophilia and inhibitors were interviewed and that 12 had family members take part. The record does not explain this distinction. It reports interview modes, but not how participants were selected for interview, whether interviews were stopped when no new themes were emerging, or how family-member accounts were integrated with patient accounts. These omissions limit assessment of how fully the themes represent the enrolled group.
Safety information is reported separately from the narrative findings. Among the 28 people listed as at risk, the registry reports zero serious adverse events, zero other adverse events, and zero deaths. Those counts describe the posted registry safety summary; they do not quantify the completeness of event ascertainment or establish that no events occurred outside the recorded reporting process. The record does not provide event definitions, exposure duration, or detailed methods for safety monitoring.
Overall, the results are best read as a thematic account of experiences among the people interviewed. They provide context about concerns and perceived treatment impact, but they do not support a numerical comparison, a causal conclusion, or a claim that the same experience applies to all people with haemophilia and inhibitors.
9. Statistical Methods Explained
The design determines what kinds of conclusions the study can support. This record describes a prospective, observational cohort qualitative study in routine clinical practice. Prospective means experiences were gathered as part of a planned study rather than solely reconstructed from a pre-existing dataset. Observational means investigators did not assign emicizumab or a control treatment in the study described. Qualitative means the central evidence is narrative material interpreted for themes, rather than a treatment effect calculated from a numeric endpoint.
What does thematic analysis do?
In general, thematic analysis organizes narrative material into recurring topics or patterns. Analysts examine accounts, develop or apply codes, and group related content into themes that can be described in a report. This method can help make sense of how people explain experiences, priorities, and perceived changes. A theme is an interpretive category, not automatically a count, a clinical outcome, or evidence of a causal effect.
The registry says that interview accounts were to be thematically analysed and reports six themes: bleeds; pain; treatment burden; control; freedom, for both people with haemophilia and family members; and missed potential. It does not report the coding framework, whether themes were decided before or after reviewing interviews, how many analysts coded the material, how disagreements were resolved, or whether participants reviewed the interpretations. Those analytic details are not reported in the registry record, so the consistency and reproducibility of the coding cannot be assessed from the record alone.
Why is there no ordinary treatment-effect estimate?
In a quantitative comparative study, an effect estimate usually contrasts outcomes across treatment groups or across defined measurement times. Here, no concurrent comparator group or standardized numeric pre-treatment and post-treatment measure is reported. Interviews included discussion of previous treatment and current treatment with emicizumab, but recollections of earlier experience are not the same as measurements collected under a controlled comparison. Differences in memory, context, and individual circumstances can shape narrative comparisons.
A confidence interval is, in general, a way to express uncertainty around a numerical estimate under specified assumptions. A p-value is, in general, a measure calculated under a statistical model to assess how compatible data are with a specified null hypothesis. Neither statistic can be interpreted for the primary qualitative findings because the registry does not report a corresponding numerical estimate, model, or hypothesis test. Any p-values, confidence intervals, or quantitative effect estimates for these outcomes are not reported in the registry record.
What can sample-size planning mean in general?
For quantitative trials, sample-size planning often uses assumptions about the effect to be detected, outcome variability, significance level, power, and allocation. Such calculations are design illustrations, not evidence that an observed result had a particular probability of success. In qualitative research, adequacy is often considered in relation to the depth and range of accounts and the analytic approach, rather than by using a conventional power calculation for a treatment effect. These are general distinctions; the registry record does not report a qualitative sample-size justification, a target for thematic saturation, or a quantitative power calculation for the interview findings.
How should counts and groups be understood?
The registry lists 30 participants as the enrollment figure and describes 16 people with haemophilia and inhibitors as interviewed, with 12 having family members take part. These counts refer to different reported aspects of the study, and the record does not explain their relationship. It does not report an analysis population, exclusions, or a participant flow account. For that reason, the interview findings should be interpreted in relation to the reported interview group, without assuming that all enrolled participants contributed narrative data.
The record lists 4 outcome measures and 2 analyses, but does not describe those analyses in enough detail to determine their methods or purpose. The primary outcome description specifies thematic analysis; further details of any quantitative analysis are not reported in the registry record. In particular, there is no reported model specification, adjustment strategy, missing-data method, or statistical plan that could be used to reproduce an inferential analysis.
The safety counts are descriptive. A count of events among people listed as at risk can summarize the posted safety record, but interpretation also depends on how events were defined, how long participants were observed, and how events were solicited and verified. Those details are not reported in the registry record. With no event counts beyond the registry summary and no exposure-time information, a rate or comparative safety estimate cannot be assessed.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 4 outcome measures and 2 analyses. It names treatment satisfaction, treatment expectations, and treatment impact as secondary outcomes, alongside a primary outcome focused on condition and treatment burden. The record does not describe a prespecified hierarchy among all outcome measures, an adjustment for multiple statistical tests, or a plan for interpreting several possible findings. Any multiplicity strategy is not reported in the registry record.
Multiplicity is a general concern when many outcomes or hypotheses are tested: the more opportunities there are to identify a result as noteworthy, the more important it is to understand whether the analyses were prespecified and how results were interpreted. In qualitative work, the issue is not simply a set of p-values. Researchers may examine a range of topics and develop themes from the accounts. Transparency about interview prompts, coding decisions, and theme development is therefore important. Those details are not reported in the registry record.
The record does not report how incomplete interviews, missing narrative material, or participants who did not complete an interview were handled. It also does not identify a defined analysis population or explain how patient and family-member accounts were combined or kept distinct. The relationship between the enrollment figure of 30 and the 16 people interviewed is not explained.
These omissions do not show that particular procedures were or were not used; they mean the registry record does not report them. As a result, readers cannot determine how missing information, analysis choices, or multiple outcomes may have influenced the reported themes.
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 |
|---|---|---|---|
| Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | 0 | 28 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | 0 of 28 |
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 |
|---|---|
| Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | 0 of 28 |
The registry safety summary lists 28 people at risk for serious adverse events and 28 at risk for other adverse events. It reports zero people affected in each category and zero deaths. These are the counts recorded for the study’s safety groups; they are not a comparison between emicizumab and another treatment, because no control group is described.
A zero count in a registry summary means that no events in that category were recorded in the posted results. It does not, on its own, establish that no events occurred in every participant or outside the period and procedures captured by the report. The record does not explain the event definitions, observation time, method of collection, or whether the listed safety group covers all people enrolled or only a particular reporting set.
For a fuller assessment of safety, readers would need information about exposure and follow-up as well as event ascertainment and classification. These details are not reported in the registry record. The counts should therefore be read narrowly as the posted descriptive safety figures, not as a comparative risk estimate or a comprehensive account of possible harms.
12. What the Result Does, and Does Not, Mean
The study offers a qualitative account of how interviewed people with haemophilia and inhibitors, and some of their family members, described life with the condition and treatment with emicizumab. Its themes identify areas that participants’ narratives addressed: bleeds, pain, treatment burden, control, freedom, and missed potential. This can help frame questions about what patients and families consider meaningful when discussing treatment experience.
The results do not quantify how often each experience occurred or how much any experience changed. The record gives no numerical estimate of reduction in bleeding, pain, or treatment burden, and no standardized measure comparing experience before and during emicizumab use. It therefore cannot show the size or precision of a treatment effect. It also does not establish that emicizumab caused any perceived change, because the study was observational and no concurrent comparison group is reported.
The themes should not be read as universal experiences. The registry describes 16 interviewed people with haemophilia and inhibitors, with 12 having family members take part, but does not report theme counts or detailed participant characteristics. The record also does not explain the relationship between the enrollment of 30 and the interview group. Readers cannot infer from the listed themes how many participants shared each view or how representative those views are of people outside the interview group.
What the results do provide is a structured account of topics that arose in the interviews, including both treatment-related and broader life impacts. The findings can inform understanding of the kinds of experiences researchers chose to capture, while remaining distinct from evidence that compares clinical outcomes across treatments. The registry record does not report enough analytic detail to assess how consistently the themes were developed or how family and patient accounts were weighed.
13. Why This Trial Matters Statistically
Clinical research is not limited to measuring events or comparing numeric outcomes. Qualitative work can examine how a condition and its treatment are experienced by patients and families, including matters that may be difficult to represent in a single scale. In this study, the stated focus on treatment burden, control, freedom, and missed opportunities makes the patient and family perspective central to the research question.
The dyad approach is relevant because the registry explicitly includes close family members alongside people with haemophilia. The reported themes include freedom for both people with haemophilia and family members, recognizing that treatment experience may reach beyond the individual receiving care. The interviews also covered previous treatment and current emicizumab use, allowing participants to describe their own perspectives on treatment history and daily life.
Statistically, the study illustrates why design and outcome type must guide interpretation. A thematic finding is not interchangeable with a treatment-effect estimate, and counts of participants interviewed are not a substitute for a randomized comparison. The registry’s qualitative findings can describe themes but cannot quantify comparative benefit, precision, or causation.
The record also illustrates the importance of transparent reporting. Information about coding, missing interviews, participant selection, and the relationship between enrollment and interview counts would help readers judge how the themes were generated and whose experiences they represent. The posted information identifies important topics while leaving those methodological questions unresolved.
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 registry record provides limited detail for evaluating the qualitative methods. It does not report the interview guide, how interviewers were trained, who conducted the interviews, how transcripts or narrative accounts were prepared, or what coding framework was used. It also does not report whether more than one analyst reviewed the accounts, how differences in interpretation were handled, or whether participants were asked to comment on the themes.
The reported participant counts need cautious interpretation. The record lists enrollment of 30 and says 16 people with haemophilia and inhibitors were interviewed, with 12 having family members take part. It does not explain why these counts differ, whether all enrolled participants were eligible for interview, or whether any interview data were incomplete. Participant characteristics such as age, location, duration of emicizumab use, and treatment history are not reported in the registry record.
The study drew on participants using emicizumab in different care contexts, including early access, clinical trials, and routine care. The record does not report how many participants came from each context or whether their experiences were analysed separately. That limits assessment of how varied treatment settings may have shaped the narratives.
The primary outcome is described in terms of potential reduction in burden, but the results are presented as themes rather than quantified change. Theme frequencies and participant-level accounts are not reported in the registry record. No concurrent comparator, standardized outcome measure, or causal analysis is described. These limitations mean the findings cannot quantify treatment effects or be assumed to represent all people with haemophilia and inhibitors.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families — NCT04723693.
- Registry record last updated 2024-08-19; status last verified 2024-03.
- Results first posted 2024-08-19.
- 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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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, prospective, observational qualitative study sponsored by Haemnet. The study examined experiences of emicizumab use among people with haemophilia and inhibitors and close family members. It listed enrollment of 30, with interviews reported for 16 people with haemophilia and inhibitors and family members taking part with 12 of them. Interviews took place online, by telephone, and in person.
The primary outcome focused on condition and treatment burden. The registry reports six themes: bleeds, pain, treatment burden, control, freedom, and missed potential. It does not report a numerical estimate of change or a controlled treatment comparison. The safety summary lists zero serious adverse events, zero other adverse events, and zero deaths among 28 people at risk. Further details about analysis methods, missing data, and event ascertainment are not reported in the registry record.