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NCT06533813

Clinical Epidemiology in Contemporary Patients With Myelofibrosis.

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

617 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-10-14

Study Completion Date

2028-12-31

Brief Summary

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Multicenter retrospective and prospective European observational study. At each site, all consecutive patients with a 2016- or 2022 World Health Organization (WHO) confirmed diagnosis of myelofibrosis (MF) established from 01/01/2018 to 31/12/2027 will be enrolled into the study. Yearly follow-up updates will be scheduled until the end of data collection on 31/12/2028 or until the last available patient visit, whichever comes first. At least 1 year of follow-up will be ensured from the last patient enrolled.

Detailed Description

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Myeloproliferative neoplasms (MPNs) are rare bone marrow disorders characterized by clonal proliferation of hematopoietic cell lineages, and include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). MF has worse prognosis, with main causes of death including acute leukemia transformation, comorbid conditions, and consequences of cytopenia. MF is characterized by progressive anemia, bone marrow fibrosis, and extramedullary hematopoiesis with splenomegaly. Moreover, the disease is associated with a heavy symptom burden including night sweats, fever, bone pain, and weight loss and worsening the quality of life.

On the beginning of 2013 the European Registry for Myeloproliferative Neoplasms (ERNEST) observational study was launched and approved by several IRBs of European hematological centers. The study focused on overt Primary (PMF) and Secondary myelofibrosis (SMF; i.e., post-Essential Thrombocythemia myelofibrosis (post-ET MF) and post-polycythemia (post-PV MF)) and aimed at describing the clinical epidemiology of large series of patients observed in clinical practice. This research was justified as the landscape of both pathophysiological and clinical knowledge in MPNs was rapidly evolving, prompting to revise diagnostic criteria, prognostication and therapy recommendations.

ERNEST retrospectively enrolled 1292 patients in whom the proposed prognostic models were confirmed to differentiate treatments in clinical practice, while ERNEST-2 reported results on critical events observed in 1010 of these cases during a median follow-up period of 5.4 years.4,5 The two studies closed in December 2022.

In the last decade, new diagnostic and prognostic findings have been accumulated and the availability of new approved drugs, based on results of several new clinical trials, influenced the therapy decision making in the real-world clinical practice. Therefore, the continuation of observational studies in present ERNEST-3 on large multicenter case series of patients with MF is timely and might refine the results of clinical trials.

The purpose of this study observational retrospective/prospective study is to gain information on MF associated cytopenias that represent a significant challenge in the contemporary patients with MF. Currently, there are few agents aimed at treating cytopenic MF, including immunomodulatory drugs, hypomethylating agents, and JAK inhibitors such as momelotinib and pacritinib, and development of new agents specifically tailored to this patient population remains an unmet need. Therefore, this study can provide data on these patients, focusing on clinical status, quality of life, comorbidities, and treatment results over time.

Conditions

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Myelofibrosis

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

RETROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of primary myelofibrosis (PMF) or secondary (i.e., post-ET/PV MF) myelofibrosis according to 2016- or 2022-WHO criteria ascertained between 01/01/2018 and 31/12/2027
* Age ≥ 18 years
* Signed informed consent where applicable, in line with current European General Data Protection Regulation (GDPR) directives

Exclusion Criteria

* Diagnosis of early/prefibrotic primary myelofibrosis
* Concurrent participation to interventional clinical trials in MF

Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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TIZIANO BARBUI, MD

Role: STUDY_DIRECTOR

FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

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TIZIANO BARBUI, MD

Role: CONTACT

Phone: 0352675134

Email: [email protected]

FENILI

Role: CONTACT

Email: [email protected]

Facility Contacts

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Role: primary

Role: backup

Clara Deambrogi

Role: primary

Elisabetta Cosi

Role: primary

Clinical Epidemiology in Contemporary Patients With Myelofibrosis.

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Study Design

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

GlaxoSmithKline

FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

Responsible Party

Principal Investigators

TIZIANO BARBUI, MD

Locations

Hematology Center after Prof.R.H. Yeolyan

University of Copenhagen's Roskilde Hospital

Clinical Investigation Center of the Saint Louis Hospital

Internal Medicine C, Hematology, Oncology, Stem Cell Transplantation and Palliative Care, University Medicine Greifswald

University Hospital Halle Department of Hematology/Oncology

University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care Johannes Wesling Medical Center Minden UKRUB, University of Bochum

Division of Haematology, Sasson University Hospital Assuta Ashdod, Ashdod, Ben-Gurion University of Negev

A.S.O. SS. Antonio e Biagio e C.Arrigo, SC Ematologia

ASST Papa Giovanni XXIII, SC Ematologia

Policlinico S. Orsola - Malpighi, Unità di Ematologia

Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di ematologia con Trapianto di midollo Osseo

AOU Careggi di Firenze, Divisione di Ematologia

ASST Grande Ospedale Metropolitano Niguarda, Divisione di Ematologia

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia

Ospedale San Raffaele, Unità Operativa di Ematologia e Trapianto Midollo Osseo

Fondazione IRCCS San Gerardo dei Tintori

Azienda Ospedaliera Universitaria Federico II, Divisione di Ematologia

Azienda Ospedaliero Universitaria Maggiore della Carità, SCDU Ematologia

Azienda Ospedale-Università di Padova, Dipartimento di Medicina

Università degli Studi di Padova - Policlinico Universitario, Clinica Medica 1

Fondazione IRCCS Policlinico San Matteo, Divisione di Ematologia

Fondazione Policlinico Universitario A. Gemelli - Università Cattolica del Sacro Cuore, UCSC Ematologia

Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia

A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U

ASST dei Sette Laghi (Ospedale di Circolo e F. Macchi), Divisione U.O. Ematologia

Azienda Ospedaliera Universitaria Integrata - Ospedale Borgo Roma, Unità di Ematologia

Azienda ULSS 8 Berica - Ospedale San Bortolo, Divisione di Ematologia

Institute of Oncology, State University of Medicine and Pharmacy - Department of Oncology, Hematology and Radiotherapy

Department of Hematology, University Hospital, Jagiellonian University Medical College

Department and Clinic of Haematology, Blood Neoplasms, and Bone Marrow Transplantation, Wroclaw Medical University

Department of Hematology and Bone Marrow Transplantation, Fundeni Clinical Institute

Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS) - Hospital Clinic

Hematology and Hemotherapy Department, 12 de Octubre University Hospital

Servicio de Hematología - Hospital Clínico Universitario de Valencia

School of Medicine, Dentistry and Biomedical Sciences Queen's University Belfast

Guy's and St. Thomas' NHS Foundation Trust.

Countries

Central Contacts

TIZIANO BARBUI, MD

FENILI

Facility Contacts

Monia Marchetti

Maria Luisa Ferrari

Valerio Michelucci

Chiara Paoli

Mattia La Monica

Silvia Artuso

Antonella Danese

Alessia Picozzi

Clara Deambrogi

Elisabetta Cosi

Antonella Mosca

Francesca Pirillo

Roberta Mattarucchi

Other Identifiers

ERNEST-3