What treatment approach would you recommend for a 65-year-old woman with von Willebrand disease, impaired renal function, and a clear bleeding phenotype, as well as a family history of the condition?

Treatment Approach for a 65-Year-Old Woman with Von Willebrand Disease

For a 65-year-old woman with von Willebrand disease, VWF antigen:activity ratio of 0.7, clear bleeding phenotype, and family history, VWF-containing factor concentrates are recommended as the first-line treatment rather than desmopressin due to her age and impaired renal function. 1

Diagnosis Assessment

Based on the clinical presentation and laboratory findings:

• VWF antigen level of 1.33 and activity of 0.993 (ratio of 0.7)
• Platelet count of 125
• Clear bleeding phenotype in patient and family members
• Family history with mother and daughter having antigen:activity ratio of 0.7

This profile is consistent with Type 2 von Willebrand disease, specifically Type 2M, characterized by:

• VWF:RCo/VWF:Ag ratio <0.7 (in this case 0.7)
• Normal multimer pattern but impaired function
• Clear bleeding phenotype despite relatively normal VWF levels 1

Treatment Recommendations

Primary Treatment Option

• VWF-containing factor concentrates are the treatment of choice for this patient due to:
  • Type 2 VWD (qualitative defect) 1, 2
  • Advanced age (65 years) with likely impaired renal function 3
  • Clear bleeding phenotype requiring reliable hemostatic control 2

Dosing Considerations

• Dosing should be based on VWF:RCo activity rather than FVIII levels 2
• Target VWF activity level of ≥50 IU/dL for minor procedures and ≥80-100 IU/dL for major surgeries 1
• Monitor VWF:RCo and FVIII:C levels at 12-24 hours post-treatment 1

Important Cautions

• Avoid desmopressin in this patient due to:
  • Impaired renal function (desmopressin is substantially excreted by kidneys) 3
  • Age-related increased risk of adverse effects 3
  • Type 2 VWD typically responds poorly to desmopressin 1, 4, 5
  • Risk of tachyphylaxis with repeated administration 3

Adjunctive Treatments

• Antifibrinolytic agents (e.g., tranexamic acid) may be used as adjunctive therapy 6
• Topical hemostatic agents for accessible bleeding sites 6
• Strict avoidance of aspirin and other platelet antiaggregants 1
• Avoid metoclopramide which may worsen platelet dysfunction 1

Monitoring and Follow-up

• Regular monitoring of renal function is essential given her age 3
• Document response to interventions to guide future management 1
• Balance hemostasis with thrombosis risk, especially important in older patients 1
• Consider prophylactic treatment before invasive procedures 1

Special Considerations

• The VWF:RCo/VWF:Ag ratio of 0.7 is at the borderline between normal and abnormal, but the clear bleeding phenotype and family history strongly support the diagnosis of Type 2 VWD 1
• The relatively normal VWF levels with abnormal function and clear bleeding phenotype is characteristic of qualitative VWD variants 4, 6
• Haemate P/Humate-P concentrate has been shown to be particularly effective in Type 2 VWD due to its high content of high molecular weight VWF multimers 2