Summary of prioritized questions
| . | Population . | Intervention . | Comparator . | Outcome . |
|---|---|---|---|---|
| 1. | Patients with VWD and history of severe and frequent bleeds | Periodic long-term prophylaxis with VWF concentrate | No concentrate (ie, on-demand therapy) | Major bleeding, serious adverse events, joint function, mortality, hospitalization |
| 2. | Patients with VWD | Treat based on results of desmopressin challenge | Not perform desmopressin challenge and treat with VWF concentrate and/or tranexamic acid | Major bleeding, serious adverse events |
| Treat with desmopressin empirically | ||||
| 3. | Patients with VWD and cardiovascular disease | Treat with antiplatelet agents or anticoagulant therapy as indicated | No treatment | Mortality, thrombotic events, serious adverse events, major bleeding, transfusion, hospitalization |
| 4. | Patients with VWD undergoing major surgery | Keep FVIII level ≥0.50 IU/mL for at least 3 d after surgery | Keep VWF activity level ≥0.50 IU/mL for at least 3 d after surgery | Mortality, major bleeding, need for additional surgical procedures, transfusion, serious adverse events, hospitalization, thrombotic events |
| 5. | Patients with VWD undergoing minor surgery or invasive procedures | Increase VWF activity level to ≥0.50 IU/mL with use of VWF concentrate or desmopressin | Tranexamic acid monotherapy | Major bleeding, need for additional hemostatic agents, need for additional surgical procedures, serious adverse events, mortality, hospitalization, transfusion, inability to perform surgery |
| Combination therapy by increasing VWF activity level to ≥0.50 IU/mL with use of VWF concentrate or desmopressin in conjunction with tranexamic acid | ||||
| 6. | Women with VWD with heavy menstrual bleeding | Tranexamic acid | Hormonal therapy (ie, levonorgestrel-releasing intrauterine system or hormonal contraceptives) | Menstrual blood loss and duration, absence from required activities, health-related quality of life, need for additional treatments, need for surgery and blood transfusion |
| Desmopressin | ||||
| 7. | Women with VWD who require or desire neuraxial anesthesia during labor (referring to spinal, epidural, or combined spinal-epidural procedures performed for surgical anesthesia for operative or pain relief) | Administer VWF concentrate to achieve VWF activity level of 0.50-1.50 IU/mL | Administer VWF concentrate to achieve VWF activity level >1.50 IU/mL | Major bleeding, serious adverse event in mother, spinal hematoma, ability to receive epidural anesthesia, mortality |
| 8. | Women with VWD in the postpartum period | Tranexamic acid | No treatment | Primary postpartum hemorrhage, secondary postpartum hemorrhage, serious adverse events in mother, need for other medical procedures, blood loss, mortality, transfusion, hospitalization |
| . | Population . | Intervention . | Comparator . | Outcome . |
|---|---|---|---|---|
| 1. | Patients with VWD and history of severe and frequent bleeds | Periodic long-term prophylaxis with VWF concentrate | No concentrate (ie, on-demand therapy) | Major bleeding, serious adverse events, joint function, mortality, hospitalization |
| 2. | Patients with VWD | Treat based on results of desmopressin challenge | Not perform desmopressin challenge and treat with VWF concentrate and/or tranexamic acid | Major bleeding, serious adverse events |
| Treat with desmopressin empirically | ||||
| 3. | Patients with VWD and cardiovascular disease | Treat with antiplatelet agents or anticoagulant therapy as indicated | No treatment | Mortality, thrombotic events, serious adverse events, major bleeding, transfusion, hospitalization |
| 4. | Patients with VWD undergoing major surgery | Keep FVIII level ≥0.50 IU/mL for at least 3 d after surgery | Keep VWF activity level ≥0.50 IU/mL for at least 3 d after surgery | Mortality, major bleeding, need for additional surgical procedures, transfusion, serious adverse events, hospitalization, thrombotic events |
| 5. | Patients with VWD undergoing minor surgery or invasive procedures | Increase VWF activity level to ≥0.50 IU/mL with use of VWF concentrate or desmopressin | Tranexamic acid monotherapy | Major bleeding, need for additional hemostatic agents, need for additional surgical procedures, serious adverse events, mortality, hospitalization, transfusion, inability to perform surgery |
| Combination therapy by increasing VWF activity level to ≥0.50 IU/mL with use of VWF concentrate or desmopressin in conjunction with tranexamic acid | ||||
| 6. | Women with VWD with heavy menstrual bleeding | Tranexamic acid | Hormonal therapy (ie, levonorgestrel-releasing intrauterine system or hormonal contraceptives) | Menstrual blood loss and duration, absence from required activities, health-related quality of life, need for additional treatments, need for surgery and blood transfusion |
| Desmopressin | ||||
| 7. | Women with VWD who require or desire neuraxial anesthesia during labor (referring to spinal, epidural, or combined spinal-epidural procedures performed for surgical anesthesia for operative or pain relief) | Administer VWF concentrate to achieve VWF activity level of 0.50-1.50 IU/mL | Administer VWF concentrate to achieve VWF activity level >1.50 IU/mL | Major bleeding, serious adverse event in mother, spinal hematoma, ability to receive epidural anesthesia, mortality |
| 8. | Women with VWD in the postpartum period | Tranexamic acid | No treatment | Primary postpartum hemorrhage, secondary postpartum hemorrhage, serious adverse events in mother, need for other medical procedures, blood loss, mortality, transfusion, hospitalization |