Low dose of aPCC after the initial treatment in acquired haemophilia A is useful to reduce bleeding relapses: Data from the FAIR registry

Sergio Siragusa, Mariasanta Napolitano, Corrado Santoro, Rocino, Cristina Santoro, Ezio Zanon, Samantha Pasca, Mameli

Risultato della ricerca: Article

Abstract

Background: Bypassing agents are the first line therapy in patients with acquired haemophilia A (AHA). Activated prothrombin complex concentrate (aPCC) proved to be effective as initial treatment, but 20% of patients (pts) had relapses. aPCC as short-term prophylaxis to reduce subsequent bleeds is still not clear. Aim: To evaluate whether a short-term prophylaxis with low dose of aPCC can reduce bleeding relapses after initial AHA treatment, maintaining safety. Methods: The FAIR Registry is a retrospective-prospective study started on December 2012, that collected data on all pts with AHA treated with aPCC in 12 Italian Haemophilia Centers. All statistical analyses were carried out in the 56 pts included in the registry. Results: 31 retrospective and 25 prospective pts were evaluated.101 bleeds requiring treatment were reported, 84.1% spontaneous, 71.3% involving muscles or skin. Major bleeds were 38,6%. Low-dose aPCC as short-term prophylaxis was started after the first resolved episode in 15/56 pts, 58% of whom prospective, in a mean dose of 54.2 ± 23.0 IU/kg, higher (61.4 ± 23.4 IU/kg) in the prospective group than in the retrospective one (44.3 ± 19.7 IU/kg) and it was continued up to a mean of 20.5 ± 17.6 days, similar in both groups. A total of 32 bleeding relapses were reported, 87.5% in the retrospective group. Only 9.4% occurred during short-term prophylaxis (p < 0.05). In our Registry no thromboembolic events were found. Conclusion: Initial AHA treatment with aPCC proved to be highly effective, but a consecutive low dose as short-term prophylaxis seems to demonstrate a significant reduction in bleeding relapses maintaining safety.
Lingua originaleEnglish
pagine (da-a)24-26
Numero di pagine3
RivistaThrombosis Research
Volume174
Stato di pubblicazionePublished - 2019

All Science Journal Classification (ASJC) codes

  • Hematology

Cita questo

@article{b0b877ed6f57461c9b0f2cb533a0155b,
title = "Low dose of aPCC after the initial treatment in acquired haemophilia A is useful to reduce bleeding relapses: Data from the FAIR registry",
abstract = "Background: Bypassing agents are the first line therapy in patients with acquired haemophilia A (AHA). Activated prothrombin complex concentrate (aPCC) proved to be effective as initial treatment, but 20{\%} of patients (pts) had relapses. aPCC as short-term prophylaxis to reduce subsequent bleeds is still not clear. Aim: To evaluate whether a short-term prophylaxis with low dose of aPCC can reduce bleeding relapses after initial AHA treatment, maintaining safety. Methods: The FAIR Registry is a retrospective-prospective study started on December 2012, that collected data on all pts with AHA treated with aPCC in 12 Italian Haemophilia Centers. All statistical analyses were carried out in the 56 pts included in the registry. Results: 31 retrospective and 25 prospective pts were evaluated.101 bleeds requiring treatment were reported, 84.1{\%} spontaneous, 71.3{\%} involving muscles or skin. Major bleeds were 38,6{\%}. Low-dose aPCC as short-term prophylaxis was started after the first resolved episode in 15/56 pts, 58{\%} of whom prospective, in a mean dose of 54.2 ± 23.0 IU/kg, higher (61.4 ± 23.4 IU/kg) in the prospective group than in the retrospective one (44.3 ± 19.7 IU/kg) and it was continued up to a mean of 20.5 ± 17.6 days, similar in both groups. A total of 32 bleeding relapses were reported, 87.5{\%} in the retrospective group. Only 9.4{\%} occurred during short-term prophylaxis (p < 0.05). In our Registry no thromboembolic events were found. Conclusion: Initial AHA treatment with aPCC proved to be highly effective, but a consecutive low dose as short-term prophylaxis seems to demonstrate a significant reduction in bleeding relapses maintaining safety.",
keywords = "Acquired haemophilia; Bleeding relapses; Bypassing agents; Prophylaxis; Hematology",
author = "Sergio Siragusa and Mariasanta Napolitano and Corrado Santoro and Rocino and Cristina Santoro and Ezio Zanon and Samantha Pasca and Mameli",
year = "2019",
language = "English",
volume = "174",
pages = "24--26",
journal = "Thrombosis Research",
issn = "0049-3848",
publisher = "Elsevier Limited",

}

TY - JOUR

T1 - Low dose of aPCC after the initial treatment in acquired haemophilia A is useful to reduce bleeding relapses: Data from the FAIR registry

AU - Siragusa, Sergio

AU - Napolitano, Mariasanta

AU - Santoro, Corrado

AU - Rocino, null

AU - Santoro, Cristina

AU - Zanon, Ezio

AU - Pasca, Samantha

AU - Mameli, null

PY - 2019

Y1 - 2019

N2 - Background: Bypassing agents are the first line therapy in patients with acquired haemophilia A (AHA). Activated prothrombin complex concentrate (aPCC) proved to be effective as initial treatment, but 20% of patients (pts) had relapses. aPCC as short-term prophylaxis to reduce subsequent bleeds is still not clear. Aim: To evaluate whether a short-term prophylaxis with low dose of aPCC can reduce bleeding relapses after initial AHA treatment, maintaining safety. Methods: The FAIR Registry is a retrospective-prospective study started on December 2012, that collected data on all pts with AHA treated with aPCC in 12 Italian Haemophilia Centers. All statistical analyses were carried out in the 56 pts included in the registry. Results: 31 retrospective and 25 prospective pts were evaluated.101 bleeds requiring treatment were reported, 84.1% spontaneous, 71.3% involving muscles or skin. Major bleeds were 38,6%. Low-dose aPCC as short-term prophylaxis was started after the first resolved episode in 15/56 pts, 58% of whom prospective, in a mean dose of 54.2 ± 23.0 IU/kg, higher (61.4 ± 23.4 IU/kg) in the prospective group than in the retrospective one (44.3 ± 19.7 IU/kg) and it was continued up to a mean of 20.5 ± 17.6 days, similar in both groups. A total of 32 bleeding relapses were reported, 87.5% in the retrospective group. Only 9.4% occurred during short-term prophylaxis (p < 0.05). In our Registry no thromboembolic events were found. Conclusion: Initial AHA treatment with aPCC proved to be highly effective, but a consecutive low dose as short-term prophylaxis seems to demonstrate a significant reduction in bleeding relapses maintaining safety.

AB - Background: Bypassing agents are the first line therapy in patients with acquired haemophilia A (AHA). Activated prothrombin complex concentrate (aPCC) proved to be effective as initial treatment, but 20% of patients (pts) had relapses. aPCC as short-term prophylaxis to reduce subsequent bleeds is still not clear. Aim: To evaluate whether a short-term prophylaxis with low dose of aPCC can reduce bleeding relapses after initial AHA treatment, maintaining safety. Methods: The FAIR Registry is a retrospective-prospective study started on December 2012, that collected data on all pts with AHA treated with aPCC in 12 Italian Haemophilia Centers. All statistical analyses were carried out in the 56 pts included in the registry. Results: 31 retrospective and 25 prospective pts were evaluated.101 bleeds requiring treatment were reported, 84.1% spontaneous, 71.3% involving muscles or skin. Major bleeds were 38,6%. Low-dose aPCC as short-term prophylaxis was started after the first resolved episode in 15/56 pts, 58% of whom prospective, in a mean dose of 54.2 ± 23.0 IU/kg, higher (61.4 ± 23.4 IU/kg) in the prospective group than in the retrospective one (44.3 ± 19.7 IU/kg) and it was continued up to a mean of 20.5 ± 17.6 days, similar in both groups. A total of 32 bleeding relapses were reported, 87.5% in the retrospective group. Only 9.4% occurred during short-term prophylaxis (p < 0.05). In our Registry no thromboembolic events were found. Conclusion: Initial AHA treatment with aPCC proved to be highly effective, but a consecutive low dose as short-term prophylaxis seems to demonstrate a significant reduction in bleeding relapses maintaining safety.

KW - Acquired haemophilia; Bleeding relapses; Bypassing agents; Prophylaxis; Hematology

UR - http://hdl.handle.net/10447/333005

UR - http://www.elsevier.com/locate/thromres

M3 - Article

VL - 174

SP - 24

EP - 26

JO - Thrombosis Research

JF - Thrombosis Research

SN - 0049-3848

ER -