Summary of prioritized questions
| . | Population . | Intervention . | Comparator . | Outcome . |
|---|---|---|---|---|
| 1 | Patients suspected of VWD | Using a BAT to screen for VWD | Nonstandardized clinical assessment (not using a BAT) | Accurate VWD diagnosis |
| 2 | Patients suspected of VWD with negative/normal BAT (eg, men and children) | Blood testing | No blood testing | Accurate VWD diagnosis |
| 3 | Patients suspected of VWD | VWF:RCo (automated and nonautomated assays) | Newer assays that reflect the platelet-binding activity of VWF (eg, VWF:GPIbM, VWF:GPIbR). | Accurate VWD diagnosis/classification |
| 4 | Patients with a historic diagnosis of type 1 VWD but who now have normal VWF levels | Reconsider the diagnosis | Remove the diagnosis | Accurate VWD diagnosis |
| 5 | Patients with abnormal initial VWD screen (low VWF:Ag and/or platelet-dependent VWF activity) and suspected type 1 VWD | Diagnosis of type 1 VWD at VWF:Ag and/or platelet-dependent VWF activity <0.30 IU/mL | Diagnosis of type 1 VWD at VWF:Ag and/or platelet-dependent VWF activity <0.50 IU/mL | Accurate VWD diagnosis (to identify type 1 VWD) |
| 6 | Patients suspected of type 1C VWD | VWFpp/VWF:Ag | Desmopressin trial with 1- and 4-h blood work | Accurate VWD classification (to identify type 1C) |
| 7 | Patients with abnormal initial screen (low VWF:Ag and/or platelet-dependent VWF activity) and suspected of type 2 VWD | Platelet-dependent VWF activity/VWF:Ag ratio <0.5 | Higher cutoff <0.7 | Accurate VWD classification (to identify those suspected of having type 2A, 2B, or 2M in need of additional testing) |
| 8 | Patients suspected of type 2A, 2B, or 2M in need of additional testing | VWF multimer analysis | VWF:CB/VWF:Ag | Accurate VWD classification (to identify type 2A, 2B, or 2M) |
| 9 | Patients suspected of type 2A or 2B VWD | Low-dose RIPA | Targeted genetic testing | Accurate VWD classification (to identify type 2B) |
| 10 | Patients suspected of type 2N in need of additional testing | VWF:FVIII binding | Targeted genetic testing | Accurate VWD classification (to identify type 2N) |
| . | Population . | Intervention . | Comparator . | Outcome . |
|---|---|---|---|---|
| 1 | Patients suspected of VWD | Using a BAT to screen for VWD | Nonstandardized clinical assessment (not using a BAT) | Accurate VWD diagnosis |
| 2 | Patients suspected of VWD with negative/normal BAT (eg, men and children) | Blood testing | No blood testing | Accurate VWD diagnosis |
| 3 | Patients suspected of VWD | VWF:RCo (automated and nonautomated assays) | Newer assays that reflect the platelet-binding activity of VWF (eg, VWF:GPIbM, VWF:GPIbR). | Accurate VWD diagnosis/classification |
| 4 | Patients with a historic diagnosis of type 1 VWD but who now have normal VWF levels | Reconsider the diagnosis | Remove the diagnosis | Accurate VWD diagnosis |
| 5 | Patients with abnormal initial VWD screen (low VWF:Ag and/or platelet-dependent VWF activity) and suspected type 1 VWD | Diagnosis of type 1 VWD at VWF:Ag and/or platelet-dependent VWF activity <0.30 IU/mL | Diagnosis of type 1 VWD at VWF:Ag and/or platelet-dependent VWF activity <0.50 IU/mL | Accurate VWD diagnosis (to identify type 1 VWD) |
| 6 | Patients suspected of type 1C VWD | VWFpp/VWF:Ag | Desmopressin trial with 1- and 4-h blood work | Accurate VWD classification (to identify type 1C) |
| 7 | Patients with abnormal initial screen (low VWF:Ag and/or platelet-dependent VWF activity) and suspected of type 2 VWD | Platelet-dependent VWF activity/VWF:Ag ratio <0.5 | Higher cutoff <0.7 | Accurate VWD classification (to identify those suspected of having type 2A, 2B, or 2M in need of additional testing) |
| 8 | Patients suspected of type 2A, 2B, or 2M in need of additional testing | VWF multimer analysis | VWF:CB/VWF:Ag | Accurate VWD classification (to identify type 2A, 2B, or 2M) |
| 9 | Patients suspected of type 2A or 2B VWD | Low-dose RIPA | Targeted genetic testing | Accurate VWD classification (to identify type 2B) |
| 10 | Patients suspected of type 2N in need of additional testing | VWF:FVIII binding | Targeted genetic testing | Accurate VWD classification (to identify type 2N) |