This protocol targets type 1 von Willebrand disease (VWD) in patients whose baseline von Willebrand factor (VWF) level is below 0.30 IU/mL — a specific sub-population in whom confirming individual treatment responsiveness is the pivotal first step.
Type 1 VWD with a baseline VWF level below 0.30 IU/mL. This lower baseline range places patients in a group where the treatment pathway depends on an upfront assessment of how the individual responds to the recommended initial intervention.
The recommended first-line strategy begins with a desmopressin trial. Ongoing treatment is then determined by each patient's individual response to that trial. The complete protocol specifies the assessment framework and the clinical steps that follow based on results.
The primary aim is to establish responsiveness to desmopressin — defined as a sufficient increase in VWF and factor VIII (FVIII) levels above defined thresholds, evaluated at specified time points after administration. Meeting these targets determines whether the same approach continues.
DOI: 10.1182/bloodadvances.2020003264.
In patients for whom desmopressin is a valid treatment option (primarily type 1 VWD) and who have a baseline VWF level of <0.30 IU/mL, the panel suggests performing a trial of desmopressin and treating based on the results over not performing a trial and treating with tranexamic acid or factor concentrate (conditional recommendation based on very low certainty in the evidence of effects).
The panel considered that an increase of at least 2 times the baseline VWF level and the ability to achieve both VWF and FVIII levels of >0.50 IU/mL were required to consider the patient responsive to desmopressin.
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