Abstract Number: PB0529
Meeting: ISTH 2021 Congress
Background: The last decade has seen significant advances in treatment options of haemophilia.
Aims: The Austrian Haemophilia Registry is a nationwide registry collecting data on all patients with haemophilia cared for in its participating centres. Here, we investigated how treatment of severe haemophilia has changed over the last decade in Austria.
Methods: We included all patients with severe haemophilia recorded between 2009 and 2019 (338). We excluded those with positive inhibitors (50, 14.8%), immune tolerance induction therapy (34, 10.1%), or receiving inhibitor bypassing agents (21, 6.2%). The final study cohort consisted of 278 patients (82.2%). Loss to follow-up was defined as no visit in the last 5 years.
Results: Patients with severe haemophilia (mean age, 24.3 years [±16.8 years]; 92.1% haemophilia A; 7.9% haemophilia B) were followed for a median observation period of 9.2 years (25th-75th percentile, 5.0-10.5 years; total observation time, 2,514.9 years) during which 37 (13.3%) patients died or were lost to follow-up. The proportion of patients on prophylaxis increased from initially 72.1% to 85.0% (Figure 1A). Following marketing authorisation, the end of the decade saw a sharp increase in treatment with extended half-life products (35.7%), whereas the use of plasma-derived (pd) and recombinant standard half-life products declined to their lowest levels in 2019 (15.4% and 45.1%, respectively) (Figure 1B). In patients with haemophilia A aged <18 years, however, an initially decreasing usage of pd products reversed again after 2015. In fact, in this age group, we recorded the highest rate of treatment with pd products in the two very last years (24.0% and 20.5%, respectively) (Figure 2), possibly reflecting the impact of the SIPPET study on the initial treatment strategy for children with haemophilia.
Conclusions: Our study provides an overview of changes in haemophilia therapy in Austria over the recent decade, reflecting significant advances in therapeutic options.
To cite this abstract in AMA style:Kraemmer D, Ritschl V, Rejtő J, Feistritzer C, Gallistl S, Jäger S, Jones N, Kurringer A, Neumeister P, Rettl J, Schuster G, Schwarz R, Sohm M, Streif W, Schindl T, Stamm T, Thom K, Male C, Pabinger I, Ay C. Changes in Treatment Patterns in Patients with Severe Haemophilia – a Longitudinal Analysis of the Austrian Haemophilia Registry [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/changes-in-treatment-patterns-in-patients-with-severe-haemophilia-a-longitudinal-analysis-of-the-austrian-haemophilia-registry/. Accessed August 15, 2022.
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