The development of an inhibitory antibody in non-severe haemophilia A patients may aggravate the bleeding phenotype considerably. Effective treatment of bleeding episodes may be challenging, with ensuing severe complications. At present, evidence is scarce for optimal treatment of bleeding episodes in this patient group. The aim of this study was to describe the incidence and the treatment of bleeding episodes in inhibitor patients in a population-based unselected cohort of non-severe haemophilia A patients with clinically relevant inhibitors. Data were available for 100 of the 107 non-severe haemophilia A patients (factor VIII (FVIII) baseline, 2-40 IU/dl) from 29 centres in Europe and one centre in Australia who had developed a clinically relevant inhibitor between 1980 and 2011. The majority (89 %) of the patients were treated during the inhibitor period for bleeding episodes or a surgical intervention: 66 % needed treatment for bleeding episodes, at a median annual bleeding rate (ABR) of 1.1 (interquartile range (IQR) 0.1-2.5) and a median total of 2 (IQR 1-6) bleeding episodes. Compared to the median ABR before inhibitor development of 0.095 bleeds per year (IQR 0.02-0.42), the increase in ABR is more than a 10-fold. More than 90 % of the bleeding episodes were treated with only one type of product, most frequently (51 %) FVIII concentrates. This study provides the incidence of bleeding episodes and treatment choices in non-severe haemophilia A patients with inhibitors. The 10-fold increase to a median ABR of 1.1 episodes per year emphasizes the impact of inhibitor development for non-severe haemophilia A patients.

The incidence and treatment of bleeding episodes in non-severe haemophilia A patients with inhibitors / van Velzen, Alice S.; Eckhardt, Corien L.; Streefkerk, Nina; Peters, Marjolein; Hart, Daniel P.; Hamulyak, Karly; Klamroth, Robert; Meijer, Karina; Nijziel, Marten; Schinco, Piercarla; Yee, Thynn T.; van der Bom, Johanna G.; Fijnvandraat, Karin; Santoro, Cristina. - In: THROMBOSIS AND HAEMOSTASIS. - ISSN 0340-6245. - 115:3(2016), pp. 543-550. [10.1160/TH15-03-0212]

The incidence and treatment of bleeding episodes in non-severe haemophilia A patients with inhibitors

SANTORO, Cristina
2016

Abstract

The development of an inhibitory antibody in non-severe haemophilia A patients may aggravate the bleeding phenotype considerably. Effective treatment of bleeding episodes may be challenging, with ensuing severe complications. At present, evidence is scarce for optimal treatment of bleeding episodes in this patient group. The aim of this study was to describe the incidence and the treatment of bleeding episodes in inhibitor patients in a population-based unselected cohort of non-severe haemophilia A patients with clinically relevant inhibitors. Data were available for 100 of the 107 non-severe haemophilia A patients (factor VIII (FVIII) baseline, 2-40 IU/dl) from 29 centres in Europe and one centre in Australia who had developed a clinically relevant inhibitor between 1980 and 2011. The majority (89 %) of the patients were treated during the inhibitor period for bleeding episodes or a surgical intervention: 66 % needed treatment for bleeding episodes, at a median annual bleeding rate (ABR) of 1.1 (interquartile range (IQR) 0.1-2.5) and a median total of 2 (IQR 1-6) bleeding episodes. Compared to the median ABR before inhibitor development of 0.095 bleeds per year (IQR 0.02-0.42), the increase in ABR is more than a 10-fold. More than 90 % of the bleeding episodes were treated with only one type of product, most frequently (51 %) FVIII concentrates. This study provides the incidence of bleeding episodes and treatment choices in non-severe haemophilia A patients with inhibitors. The 10-fold increase to a median ABR of 1.1 episodes per year emphasizes the impact of inhibitor development for non-severe haemophilia A patients.
2016
Bypassing agents; Factor VIII inhibitors; Haemophilia A; Haemostasis; Management of disease; Adolescent; Adult; Antibodies; Cohort Studies; Factor VIII; Female; Hemophilia A; Hemorrhage; Humans; Incidence; Middle Aged; Phenotype; Young Adult; Hematology
01 Pubblicazione su rivista::01a Articolo in rivista
The incidence and treatment of bleeding episodes in non-severe haemophilia A patients with inhibitors / van Velzen, Alice S.; Eckhardt, Corien L.; Streefkerk, Nina; Peters, Marjolein; Hart, Daniel P.; Hamulyak, Karly; Klamroth, Robert; Meijer, Karina; Nijziel, Marten; Schinco, Piercarla; Yee, Thynn T.; van der Bom, Johanna G.; Fijnvandraat, Karin; Santoro, Cristina. - In: THROMBOSIS AND HAEMOSTASIS. - ISSN 0340-6245. - 115:3(2016), pp. 543-550. [10.1160/TH15-03-0212]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/986880
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