Cargando…

Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?

Patients with polycythaemia vera (PV) are at increased risk of thrombosis and haemorrhages. Although hydroxyurea (HU) has been the frontline therapy for patients at high risk of vascular complications, about 25% of patients develop resistance/intolerance to this therapy. The aim of this non-interven...

Descripción completa

Detalles Bibliográficos
Autores principales: Sarmento, Maria, Duarte, Marta, Ponte, Sandra, Sanchez, Juan, Roriz, Diana, Fernandes, Laura, Silva, Maria José Monteiro, Pacheco, Judite, Ferreira, Gisela, Freitas, Jorge, Costa, Inês, Brás, Daniel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10530755/
https://www.ncbi.nlm.nih.gov/pubmed/37754671
http://dx.doi.org/10.3390/hematolrep15030056
_version_ 1785111560100773888
author Sarmento, Maria
Duarte, Marta
Ponte, Sandra
Sanchez, Juan
Roriz, Diana
Fernandes, Laura
Silva, Maria José Monteiro
Pacheco, Judite
Ferreira, Gisela
Freitas, Jorge
Costa, Inês
Brás, Daniel
author_facet Sarmento, Maria
Duarte, Marta
Ponte, Sandra
Sanchez, Juan
Roriz, Diana
Fernandes, Laura
Silva, Maria José Monteiro
Pacheco, Judite
Ferreira, Gisela
Freitas, Jorge
Costa, Inês
Brás, Daniel
author_sort Sarmento, Maria
collection PubMed
description Patients with polycythaemia vera (PV) are at increased risk of thrombosis and haemorrhages. Although hydroxyurea (HU) has been the frontline therapy for patients at high risk of vascular complications, about 25% of patients develop resistance/intolerance to this therapy. The aim of this non-interventional, multicentre cohort study was to understand the clinical characteristics and HU treatment response of Portuguese PV patients. HU resistance/intolerance was defined according to adjusted European LeukemiaNet (ELN) criteria. In total, 134 PV patients with a mean (SD) disease duration of 4.8 (5.0) years were included and followed up for 2 years. At baseline, most patients were ≥60 years old (83.2%), at high risk for thrombotic events (87.2%), and receiving HU therapy (79.1%). A total of 10 thrombotic events and 8 haemorrhagic events were reported, resulting in a 5-year probability of thrombo-haemorrhagic events of 17.2%. Haematocrit (p = 0.007), haemoglobin (p = 0.012) and MPN10 symptom score (12.0 (11.6) vs. 10.3 (9.1); p = 0.041) decreased significantly at the 24-month visit compared to baseline. Overall, 75.9% of patients met at least one of the adjusted ELN criteria for HU resistance, and 14.4% of patients remained on HU throughout the study. The results from this real-world study may help identify the subset of patients at higher risk for disease sequelae who may benefit from earlier second-line treatment.
format Online
Article
Text
id pubmed-10530755
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-105307552023-09-28 Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality? Sarmento, Maria Duarte, Marta Ponte, Sandra Sanchez, Juan Roriz, Diana Fernandes, Laura Silva, Maria José Monteiro Pacheco, Judite Ferreira, Gisela Freitas, Jorge Costa, Inês Brás, Daniel Hematol Rep Article Patients with polycythaemia vera (PV) are at increased risk of thrombosis and haemorrhages. Although hydroxyurea (HU) has been the frontline therapy for patients at high risk of vascular complications, about 25% of patients develop resistance/intolerance to this therapy. The aim of this non-interventional, multicentre cohort study was to understand the clinical characteristics and HU treatment response of Portuguese PV patients. HU resistance/intolerance was defined according to adjusted European LeukemiaNet (ELN) criteria. In total, 134 PV patients with a mean (SD) disease duration of 4.8 (5.0) years were included and followed up for 2 years. At baseline, most patients were ≥60 years old (83.2%), at high risk for thrombotic events (87.2%), and receiving HU therapy (79.1%). A total of 10 thrombotic events and 8 haemorrhagic events were reported, resulting in a 5-year probability of thrombo-haemorrhagic events of 17.2%. Haematocrit (p = 0.007), haemoglobin (p = 0.012) and MPN10 symptom score (12.0 (11.6) vs. 10.3 (9.1); p = 0.041) decreased significantly at the 24-month visit compared to baseline. Overall, 75.9% of patients met at least one of the adjusted ELN criteria for HU resistance, and 14.4% of patients remained on HU throughout the study. The results from this real-world study may help identify the subset of patients at higher risk for disease sequelae who may benefit from earlier second-line treatment. MDPI 2023-09-13 /pmc/articles/PMC10530755/ /pubmed/37754671 http://dx.doi.org/10.3390/hematolrep15030056 Text en © 2023 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
Sarmento, Maria
Duarte, Marta
Ponte, Sandra
Sanchez, Juan
Roriz, Diana
Fernandes, Laura
Silva, Maria José Monteiro
Pacheco, Judite
Ferreira, Gisela
Freitas, Jorge
Costa, Inês
Brás, Daniel
Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?
title Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?
title_full Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?
title_fullStr Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?
title_full_unstemmed Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?
title_short Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?
title_sort real-world clinical characterisation of polycythaemia vera patients from a prospective registry in portugal: is resistance to hydroxyurea a reality?
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10530755/
https://www.ncbi.nlm.nih.gov/pubmed/37754671
http://dx.doi.org/10.3390/hematolrep15030056
work_keys_str_mv AT sarmentomaria realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT duartemarta realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT pontesandra realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT sanchezjuan realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT rorizdiana realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT fernandeslaura realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT silvamariajosemonteiro realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT pachecojudite realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT ferreiragisela realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT freitasjorge realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT costaines realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality
AT brasdaniel realworldclinicalcharacterisationofpolycythaemiaverapatientsfromaprospectiveregistryinportugalisresistancetohydroxyureaareality