Questionnaire sur les saignements chez les enfants

Description

A pediatric-specific bleeding questionnaire used as a screening tool for VWD.The target population is children with VWD.

Contact person(s):
PD James: [email protected]

Date of this review: August 25, 2013
Updated: April 2014

Utility

Overall utility:

• Most useful in the pediatric clinical setting to assess which children should be further evaluated for VWD
• May also be useful in platelet function disorders
• May be used as a self-administered tool once validated appropriately
• Quick and easy to use

Limitations:

Very few studies documented a need for the pediatric specific bleeding symptoms in the “other” category, therefore the need for them is unclear.

Administration

1. Time to complete: 20 minutes
2. Equipment/space required: None
3. Training required: Physician, research nurse or research associate (“trained expert”) administered. One study utilized a self-administered version of the PBQ.⁵
4. Cost: None
5. Scoring/scaling/interpretation of results:
   • Scale –1 to 4 for 2 categories of the 13 symptoms tested: dental extraction and surgery. Maximum score for cutaneous bleeding is 2; maximum score for gastrointestinal tract or post-partum bleeding is 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: –3 to 48
   • Differential features include modification to include pediatric-specific bleeding symptoms in the “other category” (post-circumcision bleeding, umbilical stump bleeding, cephalohematoma, macroscopic hematuria, post-venipuncture bleeding, conjunctival hemorrhage)
   • Abnormal bleeding score (BS) ≥2

Psychometrics

Psychometric properties:

1. Construct validity:
   Convergent validity
   • 32% of boys with VWD scored a clinically significant bleeding score (BS) with post-circumcision bleeding.²
   • 96% of children with a platelet function disorder scored a positive BS.⁴
   • BS was inversely related to VWF:Ag and VWF:RCo when using a self-administered version of the PBQ.⁵
   • BS was significantly higher in affected vs. unaffected family members with VWD.⁶
   Group differences
   • Abnormal BS was derived from assessment of questionnaire in a general control population of children attending school association meetings. The normal range of BS was between –1.5 to 2.5 and only 13% had a score ≥2.¹
   • Median BS was significantly different between children with VWD > positive BS without VWD > negative BS without VWD.¹
   • Median BS was significantly higher in all forms of VWD vs. controls as well as all forms of definite VWD vs. possible VWD.²
2. Criterion validity:
   • For the laboratory diagnosis of VWD, the BS was able to accurately differentiate between those affected and unaffected with VWD.¹
3. Reliability:
   • Repeat testing was performed in one study but results were not recorded.²
4. Responsiveness/sensitivity:
   • Responsiveness by means of ROC analysis had an AUC of 0.88.¹
   • Responsiveness of tool to increased BS with increasing age was shown (Spearman’s correlation coefficient was 0.35, p=0.0004).²
   • The sensitivity of an abnormal BS for diagnosis of VWD was 83%, with a specificity of 79%, positive predictive value (PPV) of 0.14, and negative predictive value (NPV) of 0.99.¹
   • When using a screening question of “Does your child have a problem with bleeding or bruising?” the sensitivity and specificity for a positive BS and diagnostically proven VWD was similar (sensitivity of 94% vs. 91% and specificity of 94% vs. 95%, respectively).
   • Internal consistency of questions within one version of the self-PBQ was good, with a Crohnbach’s alpha of 0.88.⁵
   • PBQ ROC-AUC was 0.768, similar to the ISTH BAT ROC-AUC of 0.764.⁸
   • However, in a tertiary care setting when evaluating for a mild bleeding disorder, the PBQ was found to have an ROC-AUC<0.6 for qualitative BS cut off of 2:
     • Using a cut off of >2, hemorrhagic symptoms sensitivity was 60.5%, and specificity was 50.8%
     • Using a cut off of BS >3 in males and >5 in females, sensitivity was 41.9%, and specificity was 82%.⁷

Languages studied: English, German ⁸

Groups tested with this measure:

• Controls^1,2
• Children being evaluated for VWD because of personal or family history of bleeding^1,6
• Children with known diagnosis of VWD (type 1–3)^2,3,6,7
• Children with a known diagnosis of a platelet function disorder⁴
• Hemophilia A⁵

Age: Children and some adults

Glossary of Psychometric Properties

References

1. Bowman M et al. Evaluation of the diagnostic utility for von Willebrand disease of a pediatric bleeding questionnaire. J Thromb Haemost 2009; 7: 1418-1421.
2. Biss T et al. Quantitation of bleeding symptoms in children with von Willebrand disease: use of a standardized pediatric bleeding questionnaire. J Thromb Haemost 2010; 8: 950-956.
3. Bowman M et al. A Prospective Evaluation of the Prevalence of Symptomatic von Willebrand Disease (VWD) in a Pediatric Primary Care Population. Pediatr Blood Cancer 2010; 55: 171-173.
4. Biss T et al. Use of a quantitative pediatric bleeding questionnaire to assess mucocutaneous bleeding symptoms in children with a platelet function disorder. J Thromb Haemost 2010; 8: 1416-1419.
5. Boelaars MFP et al. Evaluation of a self-administrated pediatric bleeding questionnaire measuring bleeding severity in children. Thromb Haemost 2012; 108: 1006-1007.
6. Robertson J et al. Expanded phenotype–genotype correlations in a pediatric population with type 1 von Willebrand disease. J Thromb Haemost 2011; 9: 1752-60.
7. Marcus PD et al. The power of a standardized bleeding score in diagnosing paediatric type 1 von Willebrand’s disease and platelet function defects. Haemophilia 2011; 17: 223-227.
8. Bidlingmaier C et al. Prospective evaluation of a pediatric bleeding questionnaire and the ISTH bleeding assessment tool in children and parents in routine clinical practice. J Thromb Haemost 2012; 10: 1335-1341.