The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease

Abstract

Key Points• The bleeding score helps to predict clinical outcomes in adult patients with von Willebrand disease.• High bleeding scores correlate with intensive ondemand therapy and may identify cases requiring regular prophylaxis.Analyses of the bleeding tendency by means of the bleeding score (BS) have been proposed until now to confirm diagnosis but not to predict clinical outcomes in patients with inherited von Willebrand disease (VWD). We prospectively followed up, for 1 year, 796 Italian patients with different types of VWD to determine whether the previous BS of European VWD1 is useful to predict the occurrence of spontaneous bleeds severe enough to require replacement therapy with desmopressin (DDAVP) and/or von Willebrand factor (VWF)/factor VIII concentrates. Among the 796 patients included, 75 (9.4%) needed treatment of 232 spontaneous bleeding events. BS >10 and VWF:ristocetin cofactor activity <10 U/dL were associated with the risk of bleeding, but only a BS >10 remained highly associated in a multivariable Cox proportional hazard model (adjusted hazard ratio: 7.27 [95% confidence interval, 3.83-13.83]). Although the bleeding event-free survival was different in VWD types, only a BS >10 could predict for each type which patient had bleeding events severe enough to require treatment with DDAVP and/or concentrates. Therefore, BS can be considered a simple predictor of clinical outcomes of VWD and may identify patients needing intensive therapeutic regimens. (Blood. 2014;123(26):4037-4044)

How I treat type 2 variant forms of von Willebrand disease

“…39 Finally, in a recent large prospective investigation performed in 796 patients with all types of VWD, a BS .10 at enrollment was the strongest predictor of bleeding with a 5.5-fold increased risk during 1-year follow-up. 43 These observations suggest that patients with a significant previous bleeding history may be at higher bleeding risk. These patients may require more stringent monitoring after surgery or a bleeding episode to assure that proper therapeutic goals are achieved.…”

Clinical and laboratory variability in a cohort of patients diagnosed with type 1 VWD in the United States

“…In addition, some information would suggest that individuals with higher BS are more likely to bleed in the future. 45,46 This study highlights several critical areas in VWD diagnosis that require additional investigation. First, improved evaluation of phenotype, either through BATs or careful clinical evaluation, including repeat testing, should help define which patients require additional workup and treatment, the subject of further Zimmerman Program investigations.…”

Clinical and laboratory diagnosis of VWD

“…Among the 796 cases included in RENAWI-2, only 75 (9.4%) had at least 1 spontaneous bleeding event requiring treatment during the 1-year follow-up period. 31 Bleeders had median values of BS and baseline VWF:RCo/FVIII:C levels higher and lower than nonbleeders, respectively. Considering the baseline levels of VWF:RCo, all the patients could be classified as mild, moderate, and severe according to the levels of VWF activity.…”

“…Considering the baseline levels of VWF:RCo, all the patients could be classified as mild, moderate, and severe according to the levels of VWF activity. 31 The proportion of mild (31-56 IU/dL), moderate (10-30 IU/dL), and severe (Ͻ10 IU/dL) cases included in RENAWI-2 was 34%, 28%, and 38%, respectively, with different distributions within the VWD1, VWD2A, VWD2B, and VWD2M types. Indeed, the BS measured at the time of inclusion was inversely related to baseline levels of VWF:RCo, reaching a plateau at a mean value of 3.5, corresponding to VWF:RCo levels Ͼ30 U/dL (Figure 2).…”