Faculty Collaboration Database

Validation Study of the Composite Score to Identify Von Willebrand Disease in Children. J Pediatr Hematol Oncol 2016 Mar;38(2):139-42

Date

11/05/2015

Pubmed ID

26535774

DOI

10.1097/MPH.0000000000000460

Scopus ID

2-s2.0-84959568636 (requires institutional sign-in at Scopus site)   10 Citations

Abstract

BACKGROUND: The diagnosis of type 1 von Willebrand disease (VWD) presents a diagnostic challenge in children. In fact, 25% or more of children with VWD may be diagnosed only after they experience postoperative bleeding. We previously described a 4-variable composite score that has 92.5% sensitivity and 95% specificity for diagnosing VWD in children with known VWD when 2 of 4 criteria are positive: (1) Tosetto bleeding score ≥ 1; (2) family history of VWD; (3) personal history of iron deficiency anemia; and/or (4) positive James early bleeding score. The purpose of this study was to prospectively validate a composite score of ≥ 2 for identifying children with VWD.

PROCEDURE: Children without a previously diagnosed bleeding disorder presenting for hematology evaluation were enrolled. Sensitivity, specificity, positive, and negative predictive value of the composite score was determined.

RESULTS: A total of 193 subjects were enrolled from 12 participating centers were included in the analysis. Forty-seven children had type 1 VWD, including 11 with von Willebrand Ristocetin Cofactor (VWF):RCo < 30 IU/dL, 14 subjects with a VWF:RCo 30 to 39 IU/dL, and 22 with a VWF:RCo 40 to 49 IU/dL. Including all 4 variables, a composite score of ≥ 2 had a sensitivity of 63.6% to 76.0%, specificity of 33.5% to 35.1%, negative predictive value of 76.9% to 93.8%, and positive predictive value of 5.5% to 25%.

CONCLUSIONS: The negative predictive value of the composite score was robust, especially at lower VWF:RCo suggesting that VWD testing could be eliminated in nearly a third of children referred for VWD testing.

Author List

Malec LM, Moore CG, Bennett CM, Yee DL, Kerlin BA, Witmer CM, Kulkarni R, Gupta S, Gunawardena S, Kouides PA, Brown D, Ragni MV

Author

Lynn M. Malec MD Associate Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Child
Child, Preschool
Female
Hematology
Humans
Male
Sensitivity and Specificity
von Willebrand Disease, Type 1