Evaluation of the Severity and Pattern of Epistaxis In Children with a Mucocutaneous Bleeding Disorder and In Healthy Children Using a Standardized Questionnaire

Abstract 713

Epistaxis is a common symptom in children with mucocutaneous bleeding disorders such as von Willebrand disease (VWD) or a platelet function disorder (PFD), but also occurs in healthy children. Determining the features of epistaxis that suggest underlying hemostatic defects would help distinguish which children with epistaxis should undergo laboratory testing to screen for an inherited bleeding disorder. This is particularly significant in young children who may not yet have been exposed to a hemostatic challenge. However, there is no standardized approach to determine which children presenting with epistaxis should undergo testing. The aim of this study was to describe the severity and pattern of epistaxis in children with VWD or a PFD vs. healthy children using the PBQ, a standardized, validated Pediatric Bleeding Questionnaire (Bowman et al, J Thromb Haemost 2009:7;1418). PBQ epistaxis scores were assigned depending on clinical severity of the most severe episode (0: no epistaxis or trivial, ≤5/yr; 1: >5/yr or >10 min duration; 2: consultation with a healthcare professional; 3: packing, cauterization or antifibrinolytics; 4: blood transfusion, replacement therapy or desmopressin); scores of ≥2 were considered clinically significant. Epistaxis scores and features (frequency, duration, onset (spontaneous or not), site (uni- or bilateral), presence of seasonal correlation, whether cessation required medical intervention) were retrospectively reviewed in 107 questionnaires administered to patients/parents of patients with a known diagnosis of VWD (type 1, n=60 [definite, with abnormal laboratory results at least twice + a significant bleeding history, n=37; possible, with abnormal laboratory results at least once ± a significant bleeding history, n=23]; type 2, n=8; type 3, n=16) or a PFD (Glanzmann thrombasthenia, dense granule disorder, MYH9-related disorder, Noonan syndrome, Ehlers-Danlos syndrome, unspecified; n=23) and in 163 questionnaires administered to healthy children (Bowman et al, J Thromb Haemost 2009:7;1418; Biss et al, J Thromb Haemost 2010:8;950; Biss et al, J Thromb Haemost 2010:8;1416). Epistaxis was present in 66/107 patients (62%) (definite type 1 VWD: 62%; possible type 1 VWD: 48%; type 2 VWD: 38%; type 3 VWD: 75%; PFD: 74%) with a median age of 12 yrs (range: 0.6–18.3 yrs), and in 56/163 healthy children (34%), with a median age of 11.5 yrs (range: 1.5–17.0 yrs). 59% of the 66 patients with epistaxis were males, as were 50% of the 56 controls with epistaxis. The mean PBQ epistaxis score in these 66 patients with VWD or a PFD was greater, 2.1 (range: 0–4), than in the 56 controls, 0.4 (range: 0–2) (P<0.0001). The mean PBQ epistaxis score in patients varied according to underlying diagnosis and was greater in those with type 3 VWD (3.1) than in the patients with definite type 1 VWD (2.2), possible type 1 VWD (0.8), type 2 VWD (2.0) or a PFD (2.1) (P<0.05). There was no effect of age or gender on PBQ epistaxis scores within patient and control groups. 38/66 patients (58%) had a clinically significant score compared to only 5/56 (9%) of controls (P<0.0001). Epistaxis frequency (>5 episodes/yr) was greater in patients than controls, 44/66 (67%) vs. 11/56 (20%) (P<0.0001). More patients (41/66; 62%) had epistaxis lasting >10 min compared with controls (5/56; 9%) (P<0.0001). Most patients and controls reported spontaneous onset of epistaxis − 57/66 (86%) and 44/56 (79%), respectively (P=0.2316). However, a greater number of patients bled from both nostrils (51/66; 77%) than controls (15/56; 27%) (P<0.0001). Seasonal correlation of epistaxis occurred less frequently in the patient group; 14/66 patients (21%) had epistaxis only during 1 or 2 specified seasons of the year while 24/56 controls (43%) had seasonal correlation of epistaxis (P=0.0219). More patients (38/66; 58%) required medical intervention for cessation of epistaxis than controls (5/56; 9%) (P<0.0001). In conclusion, the standardized PBQ is useful in the assessment of epistaxis pattern and severity in children with a mucocutaneous bleeding disorder – VWD or a PFD. The severity score is significantly greater in patients vs. controls. Features of epistaxis suggestive of an underlying bleeding disorder include: epistaxis frequency >5 episodes/yr; duration >10 min; epistaxis occurring from both nostrils; lack of seasonal correlation; and need for medical intervention for cessation.