TY - JOUR
T1 - Treatment of patients with rare bleeding disorders in the Netherlands
T2 - Real-life data from the RBiN study
AU - Maas, Dominique P M S M
AU - Saes, Joline L
AU - Blijlevens, Nicole M A
AU - Cnossen, Marjon H
AU - den Exter, Paul L
AU - Kruis, Ilmar C
AU - Meijer, Karina
AU - Nieuwenhuizen, Laurens
AU - Peters, Marjolein
AU - Schutgens, Roger E G
AU - van Heerde, Waander L
AU - Schols, Saskia E M
N1 - Funding Information:
K. Meijer reports speaker fees from Bayer and Alexion, participation in trial steering committee for Bayer, consulting fees from Uniqure, and participation in data monitoring and endpoint adjudication committee for Octapharma. M.H. Cnossen has received investigator‐initiated research and travel grants as well as speaker fees over the years from the Netherlands Organisation for Scientific Research (NWO), the Netherlands Organization for Health Research and Development (ZonMw), the Dutch “Innovatiefonds Zorgverzekeraars,” Baxter, Baxalta, Shire, Takeda, Pfizer, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma, and has served as a steering board member for Roche, Bayer, and Novartis. All grants, awards, and fees go to the Erasmus MC as institution. R.E.G. Schutgens reports grants from Bayer, Baxalta, Pfizer, and Novo Nordisk outside the submitted work. M. Peters reports a grant from Pfizer outside the submitted work. W.L. van Heerde reports personal fees from Takeda, Bayer, and CSL Behring; other funding from Enzyre; and nonfinancial support from Sobi outside the submitted work. The remaining authors declare no competing financial interests.
Publisher Copyright:
© 2022 The Authors. Journal of Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis.
PY - 2022/4
Y1 - 2022/4
N2 - 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.
AB - 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.
KW - blood coagulation disorders
KW - fibrinolysis
KW - hemorrhage
KW - hemostasis
KW - surgical procedures
UR - http://www.scopus.com/inward/record.url?scp=85125043353&partnerID=8YFLogxK
U2 - 10.1111/jth.15652
DO - 10.1111/jth.15652
M3 - Article
C2 - 35038781
SN - 1538-7933
VL - 20
SP - 833
EP - 844
JO - Journal of thrombosis and haemostasis : JTH
JF - Journal of thrombosis and haemostasis : JTH
IS - 4
ER -