Treatment of patients with rare bleeding disorders in the Netherlands: Real‐life data from the RBiN study

Background Patients with rare inherited bleeding disorders (RBDs) exhibit hemorrhagic symptoms, varying in type and severity, often requiring only on‐demand treatment. Prolonged bleeding after invasive procedures is common. Adequate peri‐procedural therapy may reduce this bleeding risk. Objective To describe general treatment plans of RBD patients and evaluate the use of peri‐procedural hemostatic therapy. Methods In the Rare Bleeding Disorders in the Netherlands (RBiN) study, RBD patients from all six Dutch Hemophilia Treatment Centers were included. General treatment plans were extracted from patient files. Patients with a dental or surgical procedure in their history were interviewed about use of peri‐procedural treatment and bleeding complications. Results Two‐hundred sixty‐three patients with a rare coagulation factor deficiency or fibrinolytic disorder were included. Eighty‐four percent had a documented general treatment plan. General treatment plans of patients with the same RBD were heterogeneous, particularly in factor XI deficiency. Overall, 308 dental and 408 surgical procedures were reported. Bleeding occurred in 50% of dental and 53% of surgical procedures performed without hemostatic treatment and in 28% of dental and 19% of surgical procedures performed with hemostatic treatment. Not only patients with severe RBDs, but also patients with mild deficiencies, experienced increased bleeding without proper hemostatic treatment. Conclusion Large heterogeneity in general treatment plans of RBD patients was found. Bleeding after invasive procedures was reported frequently, both before and after RBD diagnosis, irrespective of factor activity levels and particularly when peri‐procedural treatment was omitted. Improved guidelines should include uniform recommendations for most appropriate hemostatic products per RBD and emphasize the relevance of individual bleeding history.