Cargando…

Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry

Background: Intracranial hemorrhage (ICH) is a highly serious event in patients with haemophilia (PWH) which leads to disability and in some cases to death. ICH occurs among all ages but is particularly frequent in newborns. Aim: The primary aim was to assess the incidence and mortality due to ICH i...

Descripción completa

Detalles Bibliográficos
Autores principales: Zanon, Ezio, Pasca, Samantha, Demartis, Francesco, Tagliaferri, Annarita, Santoro, Cristina, Cantori, Isabella, Molinari, Angelo Claudio, Biasoli, Chiara, Coppola, Antonio, Luciani, Matteo, Sottilotta, Gianluca, Ricca, Irene, Pollio, Berardino, Borchiellini, Alessandra, Tosetto, Alberto, Peyvandi, Flora, Frigo, Anna Chiara, Simioni, Paolo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8999820/
https://www.ncbi.nlm.nih.gov/pubmed/35407576
http://dx.doi.org/10.3390/jcm11071969
_version_ 1784685282524659712
author Zanon, Ezio
Pasca, Samantha
Demartis, Francesco
Tagliaferri, Annarita
Santoro, Cristina
Cantori, Isabella
Molinari, Angelo Claudio
Biasoli, Chiara
Coppola, Antonio
Luciani, Matteo
Sottilotta, Gianluca
Ricca, Irene
Pollio, Berardino
Borchiellini, Alessandra
Tosetto, Alberto
Peyvandi, Flora
Frigo, Anna Chiara
Simioni, Paolo
author_facet Zanon, Ezio
Pasca, Samantha
Demartis, Francesco
Tagliaferri, Annarita
Santoro, Cristina
Cantori, Isabella
Molinari, Angelo Claudio
Biasoli, Chiara
Coppola, Antonio
Luciani, Matteo
Sottilotta, Gianluca
Ricca, Irene
Pollio, Berardino
Borchiellini, Alessandra
Tosetto, Alberto
Peyvandi, Flora
Frigo, Anna Chiara
Simioni, Paolo
author_sort Zanon, Ezio
collection PubMed
description Background: Intracranial hemorrhage (ICH) is a highly serious event in patients with haemophilia (PWH) which leads to disability and in some cases to death. ICH occurs among all ages but is particularly frequent in newborns. Aim: The primary aim was to assess the incidence and mortality due to ICH in an Italian population of PWH. Secondary aims were to evaluate the risk factors for ICH, the role of prophylaxis, and the clinical management of patients presenting ICH. Methods: A retrospective-prospective registry was established in the network of the Italian Association of Haemophilia Centers to collect all ICHs in PWH from 2009 to 2019 reporting clinical features, treatments, and outcomes. Results: Forty-six ICHs were collected from 13 Centers. The ICHs occurred in 15 children (10 < 2 years), and in 31 adults, 45.2% of them with mild hemophilia. Overall, 60.9% patients had severe haemophilia (15/15 children). Overall ICH incidence (×1000 person/year) was 0.360 (0.270–0.480 95% CI), higher in children <2 years, 1.995 (1.110–3.442 95% CI). Only 7/46 patients, all with severe haemophilia, had received a prophylactic regimen before the ICH, none with mild. Inhibitors were present in 10.9% of patients. In adult PWHs 17/31 suffered from hypertension; 85.7% of the mild subjects and 29.4% of the moderate/severe ones (p < 0.05). ICH was spontaneous in the 69.6% with lower rate in children (46.7%). Surgery was required in 21/46 patients for cerebral hematoma evacuation. Treatment with coagulation factor concentrates for at least three weeks was needed in 76.7% of cases. ICH was fatal in 30.4% of the cases. Of the survivors, 50.0% became permanently disabled. Only one-third of adult patients received long term prophylaxis after the acute treatment. Conclusion: The results from our Registry confirm the still high incidence of ICH in infants <2 years and in adults, particularly in mild PWHs presenting hypertension and its unfavorable outcomes. The majority of PWHs were treated on-demand before ICH occurred, suggesting the important role of prophylaxis in preventing such life-threatening bleeding.
format Online
Article
Text
id pubmed-8999820
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-89998202022-04-12 Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry Zanon, Ezio Pasca, Samantha Demartis, Francesco Tagliaferri, Annarita Santoro, Cristina Cantori, Isabella Molinari, Angelo Claudio Biasoli, Chiara Coppola, Antonio Luciani, Matteo Sottilotta, Gianluca Ricca, Irene Pollio, Berardino Borchiellini, Alessandra Tosetto, Alberto Peyvandi, Flora Frigo, Anna Chiara Simioni, Paolo J Clin Med Article Background: Intracranial hemorrhage (ICH) is a highly serious event in patients with haemophilia (PWH) which leads to disability and in some cases to death. ICH occurs among all ages but is particularly frequent in newborns. Aim: The primary aim was to assess the incidence and mortality due to ICH in an Italian population of PWH. Secondary aims were to evaluate the risk factors for ICH, the role of prophylaxis, and the clinical management of patients presenting ICH. Methods: A retrospective-prospective registry was established in the network of the Italian Association of Haemophilia Centers to collect all ICHs in PWH from 2009 to 2019 reporting clinical features, treatments, and outcomes. Results: Forty-six ICHs were collected from 13 Centers. The ICHs occurred in 15 children (10 < 2 years), and in 31 adults, 45.2% of them with mild hemophilia. Overall, 60.9% patients had severe haemophilia (15/15 children). Overall ICH incidence (×1000 person/year) was 0.360 (0.270–0.480 95% CI), higher in children <2 years, 1.995 (1.110–3.442 95% CI). Only 7/46 patients, all with severe haemophilia, had received a prophylactic regimen before the ICH, none with mild. Inhibitors were present in 10.9% of patients. In adult PWHs 17/31 suffered from hypertension; 85.7% of the mild subjects and 29.4% of the moderate/severe ones (p < 0.05). ICH was spontaneous in the 69.6% with lower rate in children (46.7%). Surgery was required in 21/46 patients for cerebral hematoma evacuation. Treatment with coagulation factor concentrates for at least three weeks was needed in 76.7% of cases. ICH was fatal in 30.4% of the cases. Of the survivors, 50.0% became permanently disabled. Only one-third of adult patients received long term prophylaxis after the acute treatment. Conclusion: The results from our Registry confirm the still high incidence of ICH in infants <2 years and in adults, particularly in mild PWHs presenting hypertension and its unfavorable outcomes. The majority of PWHs were treated on-demand before ICH occurred, suggesting the important role of prophylaxis in preventing such life-threatening bleeding. MDPI 2022-04-01 /pmc/articles/PMC8999820/ /pubmed/35407576 http://dx.doi.org/10.3390/jcm11071969 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Zanon, Ezio
Pasca, Samantha
Demartis, Francesco
Tagliaferri, Annarita
Santoro, Cristina
Cantori, Isabella
Molinari, Angelo Claudio
Biasoli, Chiara
Coppola, Antonio
Luciani, Matteo
Sottilotta, Gianluca
Ricca, Irene
Pollio, Berardino
Borchiellini, Alessandra
Tosetto, Alberto
Peyvandi, Flora
Frigo, Anna Chiara
Simioni, Paolo
Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry
title Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry
title_full Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry
title_fullStr Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry
title_full_unstemmed Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry
title_short Intracranial Haemorrhage in Haemophilia Patients Is Still an Open Issue: The Final Results of the Italian EMO.REC Registry
title_sort intracranial haemorrhage in haemophilia patients is still an open issue: the final results of the italian emo.rec registry
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8999820/
https://www.ncbi.nlm.nih.gov/pubmed/35407576
http://dx.doi.org/10.3390/jcm11071969
work_keys_str_mv AT zanonezio intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT pascasamantha intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT demartisfrancesco intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT tagliaferriannarita intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT santorocristina intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT cantoriisabella intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT molinariangeloclaudio intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT biasolichiara intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT coppolaantonio intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT lucianimatteo intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT sottilottagianluca intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT riccairene intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT pollioberardino intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT borchiellinialessandra intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT tosettoalberto intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT peyvandiflora intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT frigoannachiara intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry
AT simionipaolo intracranialhaemorrhageinhaemophiliapatientsisstillanopenissuethefinalresultsoftheitalianemorecregistry