Description

The MCMDM-1 VWD bleeding questionnaire is an expert-administered questionnaire studied primarily in the research setting to distinguish between types of VWD and to assess bleeding pattern and severity. The target population is patients with VWD and their family members.

Contact person:
F Rodeghiero: [email protected]

Date of this review: August 19, 2013
Updated: April 2014

Utility

Overall utility:

Useful in the research setting for assessment of VWD and differentiating between types of VWD.

Limitations:

• Time to complete is the main limitation of this questionnaire
• Requires a skilled professional to administer

Administration

1. Time to complete: 40 minutes.
2. Equipment/space required: None.
3. Training required: Expert-administered; no training specific to the administration of the scoring tool is required.
4. Cost: None
5. Scoring/scaling and interpretation of results:
   • Scale: –1 to 4 for each of 12 symptoms, HOWEVER, maximum score for cutaneous bleeding: 2; maximum score for gastrointestinal tract bleeding: 3; score of –1 is assigned for lack of bleeding on 2 or more challenges for each of post-surgery bleeding, post-partum hemorrhage, and bleeding post-tooth extraction
   • Possible range of total score: –3 to +45
   • Differentiating features: to attempt to improve sensitivity over the Vicenza score, the range of possible scores for a symptom was increased, with a score of –1 possible in the absence of bleeding after significant hemostatic challenges

Psychometrics

Psychometric properties:

1. Construct validity:
   Convergent validity
   
   • Bleeding score (BS) was greater in index cases > affected family members > unaffected family members > controls or type 3 VWD > type 2 VWD > type 1 VWD > control.^1,2,3 
   • Higher BS was associated with lower VWF:RCo, longer PFA-100 closure times, lower VWF:Ag, lower FVIII:C, lower platelet count.^4,5,6,7
   • In subjects with either a clinical bleeding history or a hemostatic laboratory abnormality, almost half (46%) of those with a BS ≥4 had an underlying coagulation disorder (platelet secretion defect, VWD, coagulation factor deficiency, defect of fibrinolysis, secondary coagulation disorders).⁸
   Predictive validity
   • Was shown in that an increased BS was associated with increased skin bleeding time, increased need for prophylaxis, increased visits for bleeding episodes requiring treatment, decreased health related quality of life, and higher risk of post-operative bleeding.^6,9,10,11
   Group differences
   • When used in unaffected family members and control group.^1,2
   • No discriminant validity was demonstrated between BS in those with a BS ≥4 and various patterns of platelet secretion defects or number of agonists that elicited a reduced response.⁸
2. Criterion validity:
   • Increased BS quintiles had a strong inverse association with VWF:Ag, FVIII:C, and VWF:RCo levels.¹
3. Reliability:
   • No studies identified.
4. Responsiveness/sensitivity:
   • Responsiveness: Clear trend of increasing BS with age in index cases and affected family members, with no trend in controls or unaffected family members.
   • Using a cut off BS ≥2, in comparing index cases and affected family members to controls and unaffected family members, there was a statistically significant increased likelihood ratio of having VWD (based on laboratory cut offs).¹ 

Languages studied: English

Groups tested with this measure:

• Type 1 VWD (index cases)
• Affected and unaffected family members of index cases
• Controls with no history of bleeding problems
• Type 2A, 2B, 2M VWD
• Type 3 VWD
• Type 3 VWD obligate carriers
• Adults referred for a bleeding disorder evaluation

Age: Children and adults

Glossary of Psychometric Properties

References

1. Tosetto A et al. A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1 VWD). J Thromb Haemost 2006; 4: 766-773.
2. Eikenboom J, et al. Linkage analysis in families diagnosed with type 1 von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management of type 1 VWD. J Thromb Haemost 2006; 4: 774-782.
3. Akin M et al. An evaluation of DDAVP infusion test with PFA-100 and vWF activity assays to distinguish vWD types in children. Clin Appl Thromb Hemost 2011; 17: 441-448.
4. Castaman G et al. The impact of bleeding history, von Willebrand factor and PFA-100 on the diagnosis of type 1 von Willebrand disease: results from the European study MCMDM-1VWD. Br J Haematol 2010; 151: 245-251.
5. de Wee EM et al. Effect of fibrinolysis on bleeding phenotype in moderate and severe von Willebrand disease. Haemophilia 2012; 18: 444-451.
6. Federici AB et al. Efficacy and safety of highly purified, doubly virus-inactivated VWF/FVIII concentrates in inherited von Willebrand’s disease: results of an Italian cohort study on 120 patients characterized by bleeding severity score. Haemophilia 2010; 16: 101-110.
7. Federici AB et al. Clinical and molecular predictors of thrombocytopenia and risk of bleeding in patients with von Willebrand disease type 2B: a cohort study of 67 patients. Blood 2009; 113(3): 526-34.
8. Lotta LA et al. Prevalence of disease and relationships between laboratory phenotype and bleeding severity in platelet primary secretion defects. PLoS ONE 2013; 8: 1-6.
9. Shamsakhzari S Hemorrhagic symptoms and bleeding risk in obligatory carriers of type 3 von Willebrand disease in southern Iran. Blood Coagul Fibrinolysis 2011; 22: 325-330.
10. de Wee EM et al. Health-related quality of life among adult patients with moderate and severe von Willebrand disease. J Thromb Haemost 2010; 8: 1492-1499.
11. Castaman G et al. Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients. J Thromb Haemost 2012; 10: 632-638.
12. Rydz N, James P The evolution and value of bleeding assessment tools. J Thromb Haemost 2012; 10: 2223-2229.