Clinical Trials Logo

Clinical Trial Summary

Philadelphia-negative myeloproliferative neoplasms (MPN) are frequent and chronic myeloid malignancies including Polycythemia Vera (PV), essential thrombocythemia (ET), Primary Myelofibrosis (PMF) and Prefibrotic myelofibrosis (PreMF). These MPNs are caused by the acquisition of mutations affecting activation/proliferation pathways in hematopoietic stem cells. The principal mutations are JAK2V617F, calreticulin (CALR exon 9) and MPL W515. ET or MFP/PreMF patients who do not carry one of these three mutations are declared as triple-negative (3NEG) cases even if they are real MPN cases. These diseases are at high risk of thrombo-embolic complications and with high morbidity/mortality. This risk varies from 4 to 30% depending on MPN subtype and mutational status. In terms of therapy, all patients with MPNs should also take daily low-dose aspirin (LDA) as first antithrombotic drug, which is particularly efficient to reduce arterial but not venous events. Despite the association of a cytoreductive drug and LDA, thromboses still occur in 5-8% patients/year. All these situations have been explored in biological or clinical assays. All of them could increase the bleeding risk. We should look at different ways to reduce the thrombotic incidence: Direct Oral Anticoagulants (DOAC)? In the general population, in medical or surgical contexts, DOACs have demonstrated their efficiency to prevent or cure most of the venous or arterial thrombotic events. At the present time, DOAC can be used in cancer populations according to International Society on Thrombosis and Haemostasis (ISTH) recommendations, except in patients with cancer at high bleeding risk (gastro-intestinal or genito-urinary cancers). Unfortunately, in trials evaluating DOAC in cancer patients, most patients have solid rather than hematologic cancers (generally less than 10% of the patients, mostly lymphoma or myeloma). In cancer patients, DOAC are also highly efficient to reduce the incidence of thrombosis (-30 to 60%), but patients are exposed to a higher hemorrhagic risk, especially in digestive cancer patients. In the cancer population, pathophysiology of both thrombotic and hemorrhagic events may be quite different between solid cancers and MPN. If MPN patients are also considered to be cancer patients in many countries, the pathophysiology of thrombosis is quite specific (hyperviscosity, platelet abnormalities, clonality, specific cytokines…) and they are exposed to a lower risk of digestive hemorrhages. It is thus difficult to extend findings from the "general cancer population" to MPN patients. Unfortunately, only scarce, retrospective data regarding the use of DOAC in MPNs are available data. We were the first to publish a "real-life" study about the use, the impact, and the risks in this population. In this local retrospective study, 25 patients with MPN were treated with DOAC for a median time of 2.1 years. We observed only one thrombosis (4%) and three major hemorrhages (12%, after trauma or unprepared surgery). Furthermore, we have compared the benefit/risk balance compared to patients treated with LDA without difference. With the increasing evidences of efficacy and tolerance of DOAC in large cohorts of patients including cancer patients, with their proven efficacy on prevention of both arterial and venous thrombotic events and because of the absence of prospective trial using these drugs in MPN patients, we propose to study their potential benefit as primary thrombotic prevention in MPN.

Clinical Trial Description


Study Design

Related Conditions & MeSH terms

NCT number NCT05198960
Study type Interventional
Source University Hospital, Brest
Contact Jean-Christophe IANOTTO, Pr
Phone +33298223421
Status Recruiting
Phase Phase 3
Start date July 13, 2022
Completion date July 13, 2027

See also
  Status Clinical Trial Phase
Recruiting NCT05558696 - A Study of Bomedemstat (MK-3543) in Participants With Polycythemia Vera (MK-3543-004) Phase 2
Completed NCT02912884 - Treatment of Polycythaemia Vera and Essential Thrombocythaemia: Influence on the Clot Structure
Recruiting NCT02897297 - Myeloproliferative Neoplastic Diseases Observatory From Brest
Completed NCT01949805 - Pegylated Interferon Alpha-2b Versus Hydroxyurea in Polycythemia Vera Phase 3
Completed NCT00666549 - Research Tissue Bank
Completed NCT00241241 - Efficacy and Safety of Pegylated Interferon Alfa in Polycythemia Vera Phase 2
Completed NCT00052520 - Biological Therapy in Treating Patients With Advanced Myelodysplastic Syndrome, Acute or Chronic Myeloid Leukemia, or Acute Lymphoblastic Leukemia Who Are Undergoing Stem Cell Transplantation Phase 1/Phase 2
Active, not recruiting NCT05485948 - A Study to Access Efficacy and Safety of P1101 in Chinese PV Patients Who Are Intolerant or Resistance to HU Phase 2
Completed NCT01588015 - Vaccine Therapy in Preventing Cytomegalovirus Infection in Patients With Hematological Malignancies Undergoing Donor Stem Cell Transplant Phase 1
Completed NCT01243944 - Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial) Phase 3
Recruiting NCT05481151 - A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV Phase 3
Recruiting NCT05031897 - Reduced-Intensity Conditioning for the Prevention of Treatment-Related Mortality in Patients Who Undergo a Hematopoietic Stem Cell Transplant Phase 2
Recruiting NCT04116502 - MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera Phase 3
Completed NCT01901432 - A Two-part Study to Assess the Safety and Preliminary Efficacy of Givinostat in Patients With Polycythemia Vera Phase 1/Phase 2
Recruiting NCT04262141 - IMG-7289 in Patients With Essential Thrombocythemia (ET) or Polycythemia Vera (PV) Phase 2
Not yet recruiting NCT05566535 - Changes in QoL and Symptoms in Patients With Polycythemia Vera Receiving Ruxo in a Routine Clinical Practice
Active, not recruiting NCT04057040 - Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE) Phase 2
Completed NCT03907436 - The NUTRIENT Trial (NUTRitional Intervention Among myEloproliferative Neoplasms): Feasibility Phase N/A
Completed NCT01981850 - A Phase 2 Study of RO7490677 In Participants With Myelofibrosis Phase 2
Active, not recruiting NCT00588991 - Veliparib and Topotecan With or Without Carboplatin in Treating Patients With Relapsed or Refractory Acute Leukemia, High-Risk Myelodysplasia, or Aggressive Myeloproliferative Disorders Phase 1