Clearfield, E., Ayoub, E., Ziemele, B., Delaney, M., Youttananukorn, T., Coffin, D., Brennan, F., Kucher, A., Skinner, M. W., & James, P. (2025). Mapping outcomes to coreVWD: Moving toward a fully reportable core outcome set to improve access to treatment for people with von Willebrand Disease (VWD) [Abstract]. ISTH. https://www.rpthjournal.org/issue/S2475-0379(24)X0006-0
Background
The VWD treatment landscape is expanding; to assess upcoming products for efficacy and cost-effectiveness, outcomes must be harmonized across clinical trials, registries, and post-market studies. The coreVWD Initiative (Clearfield, Haemophilia 2024) convened a multi-stakeholder group to align on a recommended core outcome set important to decision-makers across the product lifecycle. Outcomes were prioritized for prophylaxis, perioperative treatment, and for women, girls, and people with the potential to menstruate (WGPPM); adverse events were also considered. To fully utilize these recommendations, we must now identify how to most efficiently measure, collect and report them.
Aims
Using coreVWD as a benchmark, to map the core outcomes identified to those currently collected in prominent ongoing studies and identify points of overlap and gaps to be covered by updated or new measurement tools.
Methods
We compared the coreVWD core outcomes to the World Federation of Hemophilia’s World Bleeding Disorders Registry (WBDR) and myWBDR app datasets and to variables collected in the Patient Reported Outcomes Burdens and Experiences (PROBE) Study, a quality-of-life study for people with hemophilia.
Results
Frequency of bleeds and bleeds requiring treatment are well covered by WBDR, myWBDR, and PROBE, but other descriptions of bleeding are only captured dependent on what Bleeding Assessment Tool (BAT) is used (Table). Information about perioperative bleed control is only collected in WBDR. All three tools could better cover the core outcomes if they enhanced or added a section for WGPPM on menstrual bleeding and pregnancy experiences.
Conclusion
coreVWD prioritized outcomes important to patients; the coreVWD outcomes will require both patient and clinically reported data. This initial analysis provides a foundation to assess completeness and fitness for purpose. Future research will assess data gaps and explore possible revisions to allow WBDR, myWBDR, or PROBE to capture additional coreVWD outcomes. Comprehensively collecting patient-important outcomes will support treatment advances for people with VWD.
| Mapping of coreVWD core outcomes to WBDR, myWBDR, and the PROBE Study Data Elements | ||||
| Type of Study | coreVWD core set | WFH WBDR Section (Clinician-reported) | myWBDR Section (Patient-reported) | PROBE Study* Question (Patient-reported) |
| All | Severity of bleeds | BAT Score1,2 | Patient-reported bleed | |
| Duration of bleeds | BAT Score1,2 | |||
| Bleeds requiring treatment | BAT Score1,2, Treatment History | Patient-reported bleed | Treatment product and treatment regimen | |
| Prophylaxis Treatment | Frequency of bleeds | Bleeding Events Assessment | Patient-reported bleed | Number of bleeds in last year, and last two weeks |
| Mucocutaneous bleeds | BAT Score1,2 (focused on epistaxis and mouth bleeding), Bleeding Events Assessment | Patient-reported bleed | ||
| Musculoskeletal bleeds | BAT Score1, Bleeding Events Assessment | Patient-reported bleed | Problem joints and range of motion | |
| Bleed control: non-surgical bleeds requiring additional treatment | BAT Score1,2, Treatment Module | Patient-reported bleed, Patient-reported treatment | Treatment product and treatment regimen | |
| Quality of life (QOL)† | EQ-5D-5L | EQ-5D-5L | EQ-5D-5L | |
| Perioperative Treatment | Re-admission to hospital | |||
| Ability to undergo invasive diagnostic or surgical procedures | BAT Score2,3 | Treatment regimen and question about invasive procedures | ||
| Bleed control: with prophylaxis prior to surgery | BAT Score2, Treatment Module | Patient-reported bleed, Patient-reported treatment | ||
| Bleed control: without prophylaxis prior to surgery | BAT Score2, Treatment Module | |||
| Number of administrations needed to treat surgical bleeds | ||||
| WGPPM Health | Menstrual blood loss | BAT Score1,2,3, Bleeding Events Assessment, Hospital Admission Module | Patient-reported bleed | |
| Menstrual period duration | BAT Score1,3 | |||
| Heavy menstrual bleeding requiring treatment | BAT Score1,2,3 | Patient-reported bleed | ||
| Need for blood transfusion from menstrual blood loss | BAT Score1,2,3 | Invasive procedures | ||
| Postpartum hemorrhage | BAT Score1,2,3, Hospital Admission Module, Pregnancy Module | Patient-reported bleed | Health problems and conditions | |
| Need for blood transfusion peri-partum | BAT Score1,2,3 | Invasive procedures | ||
| Adverse Events | Inhibitor development | VWF Inhibitor History, Inhibitor Assessment Module | Current inhibitor and question about ever having an inhibitor | |
| Thromboembolic events | Adverse Events Module | Health problems and conditions | ||
| Mortality | Mortality Section | |||
| Pregnancy: serious adverse event in mother | Pregnancy Module | Patient-reported bleed | ||
Empty cells represent data gaps. Grayed cells indicate partial coverage or that outcome can be derived from data dependent on type of assessment used and/or detail provided in open text boxes.
1 BAT Scores assessed Self-BAT
2 BAT Scores assessed by ISTH BAT
3 BAT Scores assessed by MCMDM-1
*PROBE is collected as part of the myWBDR PRO module, or can be administered independently
† PROBE is a quality-of-life study, further description of what should be included as QOL for those with VWD is needed
Abstract: HERE
