Clinical Epidemiology in Contemporary Patients With Myelofibrosis.
- Conditions
- Myelofibrosis
- Registration Number
- NCT06533813
- Lead Sponsor
- FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 617
- 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 โฅ 18 years
- Signed informed consent where applicable, in line with current European General Data Protection Regulation (GDPR) directives
- Diagnosis of early/prefibrotic primary myelofibrosis
- Concurrent participation to interventional clinical trials in MF
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Overall survival according to the presence of cytopenias at diagnosis At diagnosis during the baseline Obtained by medical health records normally filled out in clinical practice
- Secondary Outcome Measures
Name Time Method Frequency and types of cytopenias At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Incidence of adverse events (AEs) of interest, overall, by type, severity and treatments received At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Incidence of disease progressions (MF accelerated phase, MF blast phase/acute myeloid leukemia (AML)), overall and by presence of cytopenias and treatments received At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Incidence of major CV events (thrombosis and bleeding), overall and by presence of cytopenias and treatments received At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Incidence of infections, overall and by presence of cytopenias and treatments received At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Incidence of death, overall, by cause and by presence of cytopenias and treatments received At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Treatments response and duration (according to modified IWG-MRT and ELN 2013 criteria, overall and by presence of cytopenias At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Incidence of secondary neoplasia, overall, by site and by presence of cytopenias and treatments received At baseline; 1 year follow-up. Obtained by medical health records normally filled out in clinical practice
Trial Locations
- Locations (36)
Hematology Center after Prof.R.H. Yeolyan
๐ฆ๐ฒYerevan, Armenia
University of Copenhagen's Roskilde Hospital
๐ฉ๐ฐCopenhagen, Denmark
Clinical Investigation Center of the Saint Louis Hospital
๐ซ๐ทParis, France
Internal Medicine C, Hematology, Oncology, Stem Cell Transplantation and Palliative Care, University Medicine Greifswald
๐ฉ๐ชGreifswald, Germany
University Hospital Halle Department of Hematology/Oncology
๐ฉ๐ชHalle, Germany
University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care Johannes Wesling Medical Center Minden UKRUB, University of Bochum
๐ฉ๐ชMinden, Germany
Division of Haematology, Sasson University Hospital Assuta Ashdod, Ashdod, Ben-Gurion University of Negev
๐ฎ๐ฑAshdod, Israel
A.S.O. SS. Antonio e Biagio e C.Arrigo, SC Ematologia
๐ฎ๐นAlessandria, Italy
ASST Papa Giovanni XXIII, SC Ematologia
๐ฎ๐นBergamo, Italy
Policlinico S. Orsola - Malpighi, Unitร di Ematologia
๐ฎ๐นBologna, Italy
Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di ematologia con Trapianto di midollo Osseo
๐ฎ๐นCatania, Italy
AOU Careggi di Firenze, Divisione di Ematologia
๐ฎ๐นFirenze, Italy
ASST Grande Ospedale Metropolitano Niguarda, Divisione di Ematologia
๐ฎ๐นMilano, Italy
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia
๐ฎ๐นMilano, Italy
Ospedale San Raffaele, Unitร Operativa di Ematologia e Trapianto Midollo Osseo
๐ฎ๐นMilano, Italy
Fondazione IRCCS San Gerardo dei Tintori
๐ฎ๐นMonza, Italy
Azienda Ospedaliera Universitaria Federico II, Divisione di Ematologia
๐ฎ๐นNapoli, Italy
Azienda Ospedaliero Universitaria Maggiore della Caritร , SCDU Ematologia
๐ฎ๐นNovara, Italy
Azienda Ospedale-Universitร di Padova, Dipartimento di Medicina
๐ฎ๐นPadova, Italy
Universitร degli Studi di Padova - Policlinico Universitario, Clinica Medica 1
๐ฎ๐นPadova, Italy
Fondazione IRCCS Policlinico San Matteo, Divisione di Ematologia
๐ฎ๐นPavia, Italy
Fondazione Policlinico Universitario A. Gemelli - Universitร Cattolica del Sacro Cuore, UCSC Ematologia
๐ฎ๐นRoma, Italy
Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia
๐ฎ๐นRoma, Italy
A.O.U. Cittร della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U
๐ฎ๐นTorino, Italy
ASST dei Sette Laghi (Ospedale di Circolo e F. Macchi), Divisione U.O. Ematologia
๐ฎ๐นVarese, Italy
Azienda Ospedaliera Universitaria Integrata - Ospedale Borgo Roma, Unitร di Ematologia
๐ฎ๐นVerona, Italy
Azienda ULSS 8 Berica - Ospedale San Bortolo, Divisione di Ematologia
๐ฎ๐นVicenza, Italy
Institute of Oncology, State University of Medicine and Pharmacy - Department of Oncology, Hematology and Radiotherapy
๐ฒ๐ฉChisinau, Moldova, Republic of
Department of Hematology, University Hospital, Jagiellonian University Medical College
๐ต๐ฑCracovia, Poland
Department and Clinic of Haematology, Blood Neoplasms, and Bone Marrow Transplantation, Wroclaw Medical University
๐ต๐ฑWrocลaw, Poland
Department of Hematology and Bone Marrow Transplantation, Fundeni Clinical Institute
๐ท๐ดBucharest, Romania
Institut d'Investigacions Biomรจdiques August Pi i Sunyer (IDIBAPS) - Hospital Clinic
๐ช๐ธBarcellona, Spain
Hematology and Hemotherapy Department, 12 de Octubre University Hospital
๐ช๐ธMadrid, Spain
Servicio de Hematologรญa - Hospital Clรญnico Universitario de Valencia
๐ช๐ธValencia, Spain
School of Medicine, Dentistry and Biomedical Sciences Queen's University Belfast
๐ฌ๐งBelfast, United Kingdom
Guy's and St. Thomas' NHS Foundation Trust.
๐ฌ๐งLondra, United Kingdom