Epidemiology and Treatment of Patients with Haemophilia in Austria—Update from the Austrian Haemophilia Registry

Judit Rejtő; Sylvia Reitter-Pfoertner; Sylvia Kepa; Clemens Feistritzer; Michael Grundbichler; Alexander Hörbst; Neil Jones; Wolfgang Muntean; Peter Neumeister; Stefan Oberbichler; Gerhard Schuster; Rudolf Schwarz; Katharina Thom; Karl Zwiauer; Werner Streif; Christoph Male; Ingrid Pabinger

doi:10.1055/s-0038-1675354

Hämostaseologie, Inhaltsverzeichnis

Hamostaseologie 2019; 39(03): 284-293
DOI: 10.1055/s-0038-1675354

Original Article

Georg Thieme Verlag KG Stuttgart · New York

Epidemiology and Treatment of Patients with Haemophilia in Austria—Update from the Austrian Haemophilia Registry

Epidemiologie und Therapie der Hämophilie in Österreich—Update aus dem Österreichischen Hämophilie Register

Judit Rejtő

¹Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria

,

Sylvia Reitter-Pfoertner

¹Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria

,

Sylvia Kepa

¹Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria

,

Clemens Feistritzer

²Department of Internal Medicine V – Haematology and Oncology, Medical University of Innsbruck, Innsbruck, Austria

,

Michael Grundbichler

³IIIrd Medical Department, Paracelsus Medical University Salzburg, Salzburg Cancer Research Institute, Salzburg, Austria

,

Alexander Hörbst

⁴eHealth Research and Innovation Unit, UMIT – University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Austria

,

Neil Jones

⁵Department of Pediatrics and Adolescent Medicine, Paracelsus Private Medical University, Salzburg, Austria

,

Wolfgang Muntean

⁶Department of General Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria

,

Peter Neumeister

⁷Division of Haematology, Department of Internal Medicine, Medical University of Graz, Graz, Austria

,

Stefan Oberbichler

⁴eHealth Research and Innovation Unit, UMIT – University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Austria

,

Gerhard Schuster

⁸Austrian Red Cross, Blood Transfusion Service for Upper Austria, Linz, Austria

,

Rudolf Schwarz

⁹Department of Paediatric and Adolescent Medicine, Kepler University Hospital Linz, Linz, Austria

,

Katharina Thom

¹⁰Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria

,

Karl Zwiauer

¹¹Department of Paediatrics, Universitätsklinikum St. Pölten, Karl Landsteiner University of Health Sciences Austria, St. Pölten, Austria

,

Werner Streif

¹²Department of Pediatrics, Medical University of Innsbruck, Innsbruck, Austria

,

Christoph Male

¹⁰Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria

,

Ingrid Pabinger

¹Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria

› Institutsangaben

Abstract

The Austrian Haemophilia Registry collects epidemiological data on patients with haemophilia, on treatment modalities and potential side effects. The Registry covers more than 85% of the assumed total number of haemophilia patients in Austria. This report summarizes data on 753 patients: 84.3% (635) have haemophilia A and 15.7% (118) have haemophilia B. Patients' median age is 34 years (range: 1–93 years). Of the total cohort, 39.0% (294) patients have severe haemophilia, 11.3% (85) moderate haemophilia, and 49.4% (372) mild haemophilia. Of the patients with severe haemophilia, 38.4% (113) have been infected with hepatitis C virus (HCV) and 12.6% (37) are human immunodeficiency virus (HIV) positive. Overall, 10.6% (67) of patients with haemophilia A and 1.7% (2) of those with haemophilia B have had an inhibitor in their history. Among patients with severe haemophilia, 68.4% (201) receive prophylaxis and 28.6% (84) receive on-demand therapy. There are 65.0% (191) patients with severe haemophilia who are treated with recombinant products. In conclusion, most patients with severe haemophilia receive prophylactic treatment. HCV and HIV infections are still important issues in the Austrian haemophilia population.

Zusammenfassung

Das Österreichische Hämophilieregister sammelt epidemiologische Daten über Patienten mit Hämophilie, über die Behandlungsmodalitäten und über die möglichen Nebenwirkungen. Das Österreichische Hämophilie Register erfasst mehr als 85% der zu erwartenden Anzahl von Hämophilen in Österreich. Im Register sind 753 Patienten erfasst, von denen 84.3% (635) Hämophilie A und 15.7% (118) Hämophilie B haben. Das mediane Alter ist 34 Jahre (1–93). Es haben 39.0% (294) eine schwere, 11.3% (85) eine mittelschwere und 49.4% (372) eine leichte Hämophilie. Von den Patienten mit schwerer Hämophilie wurden 38.4% (113) mit HCV infiziert und 12.6% (37) sind HIV positiv. Insgesamt haben 10.6% (67) aller Patienten mit Hämophilie A und 1.7% (2) aller Patienten mit Hämophilie B einen Hemmkörper in ihrer Anamnese. Von den Patienten mit schwerer Hämophilie erhalten 68.4% (201) eine Prophylaxe und 28.6% (84) eine Bedarfsbehandlung. Von den Patienten mit schwerer Hämophilie erhalten 65.0% (191) ein rekombinantes Produkt. Zusammenfassend erhalten die meisten Patienten mit schwerer Hämophilie eine Prophylaxe. HCV und HIV Infektionen sind immer noch von großer Bedeutung für die österreichischen Hämophilie-Patienten.

Keywords

Austrian Haemophilia Registry - hepatitis C virus - congenital bleeding disorder

Schlüsselwörter

Österreichisches Hämophilieregister - Hepatitis C Virus - angeborene Blutungsstörung

Volltext

Referenzen

References
1 Srivastava A, Brewer AK, Mauser-Bunschoten EP. , et al; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia 2013; 19 (01) e1-e47
2 Beschluss der Kommission vom 10. Februar 2006 über die Annahme des Arbeitsplanes 2006 für die Durchführung des Aktionsprogramms der Gemeinschaft im Bereich der öffentlichen Gesundheit (2003–2008), einschließlich Jahresarbeitsprogramm für Finanzhilfen (2006/89/EG); 2006
3 EMEA Report. Expert meeting on factor VIII products and inhibitor development; 2007 https://www.ema.europa.eu/documents/report/report-expert-meeting-factor-viii-products-inhibitor-development_en.pdf , accessed 17 Jan 2018
4 Evatt B. Guide to developing a national patient registry. World Federation of Haemophilia; 2005. http://www1.wfh.org/publication/files/pdf-1288.pdf , accessed 23 Oct 2018
5 Reitter S, Streif W, Schabetsberger T. , et al. Austrian Hemophilia Registry: design, development and set of variables. Wien Klin Wochenschr 2009; 121 (5-6): 196-201
6 Austrian Data Protection Law (DSG 2000): Bundesgesetzblatt für die Republik Österreich vom 17. August 1999
7 White II GC, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J. ; Factor VIII and Factor IX Subcommittee. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 2001; 85 (03) 560
8 Blanchette VS, Srivastava A. Definitions in hemophilia: resolved and unresolved issues. Semin Thromb Hemost 2015; 41 (08) 819-825
9 Iorio A, Oliovecchio E, Morfini M, Mannucci PM. ; Association of Italian Hemophilia Centres Directors. Italian Registry of Haemophilia and Allied Disorders. Objectives, methodology and data analysis. Haemophilia 2008; 14 (03) 444-453
10 Aznar JA, Lucía F, Abad-Franch L. , et al. Haemophilia in Spain. Haemophilia 2009; 15 (03) 665-675
11 Zdziarska J, Chojnowski K, Klukowska A. , et al. Registry of inherited bleeding disorders in Poland--current status and potential role of the HemoRec database. Haemophilia 2011; 17 (01) e189-e195
12 Windyga J, Lopaciuk S, Stefanska E. , et al. Haemophilia in Poland. Haemophilia 2006; 12 (01) 52-57
13 Teixeira L, Ferreira C, Santos BS, Saavedra V. Web-enabled registry of inherited bleeding disorders in Portugal: conditions and perception of the patients. Haemophilia 2012; 18 (01) 56-62
14 Pabinger I, Heistinger M, Muntean W. , et al. [Treatment of haemophilia in Austria]. Wien Klin Wochenschr 2015; 127 (Suppl. 03) S115-S130
15 Berntorp E, Dolan G, Hay C. , et al. European retrospective study of real-life haemophilia treatment. Haemophilia 2017; 23 (01) 105-114
16 Peyvandi F, Mannucci PM, Garagiola I. , et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med 2016; 374 (21) 2054-2064
17 Iorio A, Halimeh S, Holzhauer S. , et al. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost 2010; 8 (06) 1256-1265
18 Berntorp E. Plasma-derived versus recombinant factor concentrates in PUPs: a never ending debate?. Hamostaseologie 2017; 37 (01) 53-57
19 Darby SC, Kan SW, Spooner RJD. , et al; UK Haemophilia Centre Doctors' Organisation. The impact of HIV on mortality rates in the complete UK haemophilia population. AIDS 2004; 18 (03) 525-533
20 Lassila R, Makris M. Safety surveillance in haemophilia and allied disorders. J Intern Med 2016; 279 (06) 515-523
21 Larsson SA. Life expectancy of Swedish haemophiliacs, 1831-1980. Br J Haematol 1985; 59 (04) 593-602
22 Ferenci P. Treatment of hepatitis C in difficult-to-treat patients. Nat Rev Gastroenterol Hepatol 2015; 12 (05) 284-292
23 von Köckritz L, Dufour J-F. Management of chronic hepatitis C in 2017. Hamostaseologie 2017; 37 (03) 186-195
24 Berntorp E. Future of haemophilia outcome assessment: registries are key to optimized treatment. J Intern Med 2016; 279 (06) 498-501
25 Osooli M, Berntorp E. Registry-based outcome assessment in haemophilia: a scoping study to explore the available evidence. J Intern Med 2016; 279 (06) 502-514
26 Reitter S, Sturn R, Horvath B. , et al; Austrian Molecular Haemophilia Study Group. Spectrum of causative mutations in patients with haemophilia A in Austria. Thromb Haemost 2010; 104 (01) 78-85
27 Margaglione M, Castaman G, Morfini M. , et al; AICE-Genetics Study Group. The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype. Haematologica 2008; 93 (05) 722-728
28 Kepa S, Horvath B, Reitter-Pfoertner S. , et al. Parameters influencing FVIII pharmacokinetics in patients with severe and moderate haemophilia A. Haemophilia 2015; 21 (03) 343-350
29 Hofbauer CJ, Kepa S, Schemper M. , et al. FVIII-binding IgG modulates FVIII half-life in patients with severe and moderate hemophilia A without inhibitors. Blood 2016; 128 (02) 293-296
30 Dolan G, Makris M, Bolton-Maggs PHB, Rowell JA. Enhancing haemophilia care through registries. Haemophilia 2014; 20 (Suppl. 04) 121-129