The discriminant power of bleeding history for the diagnosis of type 1 von Willebrand disease: an international, multicenter study.
OBJECTIVE
The aim of this study was the validation of the criteria defining a significant mucocutaneous-bleeding history in type 1 von Willebrand disease (VWD).


METHODS
To avoid selection bias, 42 obligatory carriers (OC) of type 1 VWD were identified from a panel of 42 families with type 1 VWD enrolled by 10 expert centers. OC were identified by the presence of an offspring and another first degree relative with type 1 VWD (affected subjects, AFF). A standardized questionnaire was administered to evaluate hemorrhagic symptoms at the time of first examination, using a bleeding score ranging from 0 (no symptom) to 3 (hospitalization, replacement therapy, blood transfusion). Sensitivity, specificity, diagnostic likelihood ratios, positive and negative predictive values for the diagnosis of type 1 VWD were calculated from the data collected in OC and in 215 controls.


RESULTS
Having at least three hemorrhagic symptoms or a bleeding score of 3 in males and 5 in females was very specific (98.6%) for the bleeding history of type 1 VWD, although less sensitive (69.1%). None of the misclassified OC had life-threatening bleeding episodes after diagnosis.


CONCLUSIONS
We suggest that the use of a standardized questionnaire and bleeding score may be useful for the identification of subjects requiring laboratory evaluation for VWD.
Rodeghiero
F
F
Hematology Department, S. Bortolo Hospital, Vicenza, Italy. rodeghiero@hemato.ven.it
Castaman
G
G
Tosetto
A
A
Batlle
J
J
Baudo
F
F
Cappelletti
A
A
Casana
P
P
De Bosch
N
N
Eikenboom
J C J
JC
Federici
A B
AB
Lethagen
S
S
Linari
S
S
Srivastava
A
A
D000328:Adult; D000465:Algorithms; D016022:Case-Control Studies; D005192:Family Health; D005260:Female; D006470:Hemorrhage / Q000175:diagnosis*; D006579:Heterozygote; D006801:Humans; D008297:Male; D008487:Medical History Taking; D011237:Predictive Value of Tests; D012680:Sensitivity and Specificity; D012720:Severity of Illness Index; D012737:Sex Factors; D011795:Surveys and Questionnaires; D014842:von Willebrand Diseases / Q000175:diagnosis*