Tiziano Barbui
YOU?
Author Swipe
View article: Towards New Therapy Endpoints in Polycythemia Vera: Targeting Clonal and Inflammatory Pathways
Towards New Therapy Endpoints in Polycythemia Vera: Targeting Clonal and Inflammatory Pathways Open
Although phlebotomy and hydroxyurea (HU) are standard first-line therapies for polycythaemia vera (PV), they do not adequately address clonal expansion and chronic inflammation - key drivers of thrombosis, myelofibrosis and mortality. Biom…
View article: Towards New Therapy Endpoints in Polycythemia Vera: Targeting Clonal and Inflammatory Pathways
Towards New Therapy Endpoints in Polycythemia Vera: Targeting Clonal and Inflammatory Pathways Open
Although phlebotomy and hydroxyurea (HU) are standard first-line therapies for polycythaemia vera (PV), they do not adequately address clonal expansion and chronic inflammation - key drivers of thrombosis, myelofibrosis and mortality. Biom…
View article: Ropeginterferon alfa-2b in 248 patients with polycythemia vera: Results from the italian cohort of a prospective, non-interventional, post-authorization study (ROPEG-PV)
Ropeginterferon alfa-2b in 248 patients with polycythemia vera: Results from the italian cohort of a prospective, non-interventional, post-authorization study (ROPEG-PV) Open
Introduction: Ropeginterferon alfa-2b (Ropeg) was approved in Europe (2019) and in the US (2021) for treating polycythemia vera (PV), regardless of risk category or prior treatments. Despite its broad indication, real-world access in Europ…
View article: Triple A Plus ( <scp>AAA</scp> <sup>+</sup> ) Survival Prediction Model for Essential Thrombocythemia: Analysis Involving 7308 Patients
Triple A Plus ( <span>AAA</span> <sup>+</sup> ) Survival Prediction Model for Essential Thrombocythemia: Analysis Involving 7308 Patients Open
Survival prediction models in essential thrombocythemia (ET) include the International Prognostic Scoring System (IPSET) and the more recently introduced triple‐A (AAA) prognostic score. The latter enlists age and absolute neutrophil (ANC)…
View article: European LeukemiaNet criteria for safety and efficacy evaluation of new drugs for myelofibrosis.
European LeukemiaNet criteria for safety and efficacy evaluation of new drugs for myelofibrosis. Open
Most myelofibrosis (MF) drug approvals by Health Authorities are based on 1 or 2 outcome measures such as spleen volume reduction and/or improved quality-of-life. This 1–2 dimensional approach fails to capture the clinical complexity of MF…
View article: Clonal megakaryocyte dysplasia with normal blood values (CMD-NBV): an unique form of early myeloproliferative neoplasm
Clonal megakaryocyte dysplasia with normal blood values (CMD-NBV): an unique form of early myeloproliferative neoplasm Open
We report an in-depth retrospective analysis of an updated series of 30 subjects with clonal megakaryocyte dysplasia with normal blood values (CMD-NBV). Sixteen were men, median age was 47.5 years (IQR, 39–53 years). A thrombosis-driven si…
View article: Thrombosis‐Driven Disease Progression in <i>JAK2</i>‐Mutant Polycythemia Vera and Essential Thrombocythemia: Reassessing Risk‐Based Management
Thrombosis‐Driven Disease Progression in <i>JAK2</i>‐Mutant Polycythemia Vera and Essential Thrombocythemia: Reassessing Risk‐Based Management Open
This paper explores emerging therapies in polycythemia vera and essential thrombocythemia, focusing on thrombosis as a driver of disease progression leading to myelofibrosis, blast phase, second cancers, and mortality. While the thrombosis…
View article: Cost-utility analysis of ropeginterferon alfa-2b to manage low-risk patients with polycythemia vera as compared to phlebotomy only in the Austrian healthcare system
Cost-utility analysis of ropeginterferon alfa-2b to manage low-risk patients with polycythemia vera as compared to phlebotomy only in the Austrian healthcare system Open
Treatment of polycythemia vera (PV) aims to maintain hematocrit on target to reduce risk of thrombotic complications, while preventing disease progression to myelofibrosis (MF) and acute myeloid leukemia (AML). This analysis evaluated cost…
View article: Cytopenic overt primary myelofibrosis at presentation: Analysis of outcomes in the prospective, real‐world ERNEST‐2 registry
Cytopenic overt primary myelofibrosis at presentation: Analysis of outcomes in the prospective, real‐world ERNEST‐2 registry Open
We report herein the analysis of data derived from the European multicenter collaborative ERNEST registry, specifically focusing on clinical characteristics and outcomes of MF patients with CyP. The ERNEST project prospectively enrolled pa…
View article: Essential Thrombocythemia
Essential Thrombocythemia Open
Importance Essential thrombocythemia, a clonal myeloproliferative neoplasm with excessive platelet production, is associated with an increased risk of thrombosis and bleeding. The annual incidence rate of essential thrombocythemia in the U…
View article: Impact of Recent Translational and Therapeutic Developments on Clinical Course of <i>BCR::ABL1‐</i> Positive and ‐Negative Myeloproliferative Neoplasms
Impact of Recent Translational and Therapeutic Developments on Clinical Course of <i>BCR::ABL1‐</i> Positive and ‐Negative Myeloproliferative Neoplasms Open
Despite the study of BCR::ABL1 ‐positive and ‐negative myeloproliferative neoplasms (MPNs) providing seminal insights into cancer biology, tumor evolution and precision oncology over the past half century, significant challenges remain. MP…
View article: Thrombosis Risk Assessment in Polycythemia Vera (TRAP): A 4,636-Patient-Year Analysis of Arterial and Venous Thrombosis in Low-Risk Patients
Thrombosis Risk Assessment in Polycythemia Vera (TRAP): A 4,636-Patient-Year Analysis of Arterial and Venous Thrombosis in Low-Risk Patients Open
Background Conventional thrombosis risk stratification in polycythemia vera (PV) considers age > 60 years and thrombosis history for assignment to low (no risk factor) or high (at least 1 risk factor) risk category (J Clin Oncol.2011;29…
View article: Cancers in Polycythemia Vera and Essential Thrombocythemia: A before and after Diagnosis Survey and Analysis of Risk Factors
Cancers in Polycythemia Vera and Essential Thrombocythemia: A before and after Diagnosis Survey and Analysis of Risk Factors Open
Background An increased risk of second cancers (SC), in patients with myeloproliferative neoplasms (MPN), has been suggested by a number of population-based and cancer registry studies (Blood 2011;118:6515). Such studies often included pat…
View article: Monocytosis Is an Independent Risk Factor for Survival in Essential Thrombocythemia - a Revised Triple-a Prognostic Score (AAA+A) Based on Age and Absolute Monocyte, Neutrophil, and Lymphocyte Counts
Monocytosis Is an Independent Risk Factor for Survival in Essential Thrombocythemia - a Revised Triple-a Prognostic Score (AAA+A) Based on Age and Absolute Monocyte, Neutrophil, and Lymphocyte Counts Open
Background Survival risk models in essential thrombocythemia (ET) include the International Prognostic Scoring System (IPSET; Blood 2012;120:1197) and the more recently described “triple-A” prognostic score that considers Age and absolute …
View article: Neutrophil/lymphocyte ratio identifies low‐risk polycythaemia vera patients for early Ropeginterferon alfa‐2b therapy
Neutrophil/lymphocyte ratio identifies low‐risk polycythaemia vera patients for early Ropeginterferon alfa‐2b therapy Open
Summary We investigated the effect of Ropeginterferon alfa‐2b (Ropeg) versus phlebotomy‐only (Phl‐O) on the neutrophil‐to‐lymphocyte ratio (NLR) in 126 patients randomized in the low‐polycythaemia vera (PV) phase II trial. Patients with a …
View article: Proposals for revised International Working Group–European LeukemiaNet criteria for anemia response in myelofibrosis
Proposals for revised International Working Group–European LeukemiaNet criteria for anemia response in myelofibrosis Open
With emerging new drugs in myelofibrosis (MF), a robust and harmonized framework for defining the severity of anemia and response to treatment will enhance clinical investigation and facilitate interstudy comparisons. Accordingly, the lead…
View article: Exploring the Clinical and Genetic Landscape of Angelman Syndrome: Patient-Reported Insights from an Italian Registry
Exploring the Clinical and Genetic Landscape of Angelman Syndrome: Patient-Reported Insights from an Italian Registry Open
Background: The Angelman Syndrome Registry (RISA) was developed as a retrospective study with the following objectives: to evaluate the clinical history of individuals with Angelman Syndrome (AS) in Italy and compare it with the existing l…
View article: Clinical impact of mutated <scp>JAK2</scp> allele burden reduction in polycythemia vera and essential thrombocythemia
Clinical impact of mutated <span>JAK2</span> allele burden reduction in polycythemia vera and essential thrombocythemia Open
The variant allele frequency (VAF) of driver mutations ( JAK2, CALR ) in myeloproliferative neoplasms is associated with features of advanced disease and complications. Ruxolitinib and interferon were reported to variably reduce the mutant…
View article: Age-stratified analysis reveals arterial thrombosis as a predictor for gender-related second cancers in myeloproliferative neoplasms: a case-control study
Age-stratified analysis reveals arterial thrombosis as a predictor for gender-related second cancers in myeloproliferative neoplasms: a case-control study Open
Over the past decade, observational and population-based studies
\nhave documented an excess of solid tumors and lymphomas in
\npatients with myeloproliferative neoplasms (MPN), including polycythemia vera (PV), essential thrombocythemia (…
View article: Essential thrombocythemia: 2024 update on diagnosis, risk stratification, and management
Essential thrombocythemia: 2024 update on diagnosis, risk stratification, and management Open
Overview Essential thrombocythemia is a Janus kinase 2 ( JAK2 ) mutation‐prevalent myeloproliferative neoplasm characterized by clonal thrombocytosis; clinical course is often indolent but might be interrupted by thrombotic or hemorrhagic …