Bleeding and Treatment in Factor XIII Deficiency
European Registry (PRO-RBDD) Reports Bleeding Patterns and Treatment Regimen in Congenital Factor XIII Deficiency by Severity
ASH 2014 (San Francisco, December 6-9) Interim results from the PRO-RBDD study, a global initiative focused in part on assessment of patients with fibrinogen and factor XIII deficiency over 3 years, were presented at the American Society of Hematology (ASH) 2014 Annual Meeting. To date, 15 sites submitted 109 cases of factor XIII deficiency. Factor levels were found to be correlated with bleeding severity (extent of symptoms). Milder (>30%) deficiency was diagnosed at an older age than severe (<5%) deficiency (average 30 years old vs 5 years old). In women, 16/26 pregnancies resulted in spontaneous abortions. For patients with severe deficiency, 71/74 had spontaneous bleeds, most with major spontaneous bleeds and 67/75 were treated with routine FXIII replacement (prophylaxis). For patients with moderate deficiency, 7/10 had spontaneous bleeding; many with major bleeding and 2/10 were treated with routine replacement (prophylaxis). In the few patients with 6 month follow-up, no factor XIII deficient patients on prophylaxis experienced bleeding.
The PRO-RBDD Study is an independent study that receives financial support from Novo Nordisk Healthcare AG.