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SYTHROM Cohort, Myeloproliferative Neoplasia With Normal CBC and Thrombotic Complications (SYTHROM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04539678
Recruitment Status : Recruiting
First Posted : September 7, 2020
Last Update Posted : September 9, 2021
French Intergroup of Myeloproliferative syndromes
Information provided by (Responsible Party):
Nantes University Hospital

Brief Summary:

Among the etiologies of thrombosis, myeloproliferative neoplasia (MPN) is quite rare but should be investigated in case of thrombosis of atypical localization (digestive or cerebral) or in the context of recurrent idiopathic thrombosis in a young subject. Thrombosis could reveal an underlying MPN through the identification of a JAK2 V617F mutation. Rarely, MPN with thrombotic complications present with normal complete blood count(CBC). In case of a MPN with a thrombotic event but without CBC abnormality, anti-thrombotic treatment is recommended. But there is no recommendation for the indication of cytoreductive therapy and the clinician's decision is often empirical.

One of the major complications of for essential thrombocythemia (ET) or polycythemia vera (PV) is thrombosis and an age over 60 is a major risk factor. The treatment of thrombosis associated with TE or PV is based on recommendations the main therapeutic objective of which is to reduce the thrombotic risk. The combination of a cytoreducing agent and antithrombotic treatment is thus proposed in high-risk patients. The efficacy of this management is monitored by assessing CBC with the objective of normalization at <400 G/L of platelets for ET patients and <45% hematocrit in case of PV.

The absence of abnormal CBC makes it difficult to justify cytoreduction. The benefit of such a therapy in this context has not been clinically demonstrated. If a cytoreductive therapy is initiated, no biological parameters are available to assess the response to treatment.

The objective of this observational study is to evaluate the incidence of recurrence of thrombosis in patients whose thrombotic event revealed an underlying MPN with normal CBC. A comparison of groups treated or not with cytoreductive agents will be performed. Longitudinal monitoring of the patients will provide a better understanding of the nature and kinetics of hematological changes in these patients.

Condition or disease
Thrombotic Patients Diagnosis of Myeloproliferative Neoplasia Impact of Treatment With Cytoreducing Agent

Detailed Description:
This study is observational and multicentric.In a first part, patients will be retrospectively included. Baseline clinical and biological data obtainedat the time of MPN diagnosis of MPN with normal CBC following venous or arterial thrombosis will be recorded and follow-up data will be collected in an e-CRF. In a second part, patients will be included prospectively and diagnostic and follow-up data will be collected. Whether or not to initiate treatment with a cytoreduction treatment is left to the discretion of the clinician.

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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Retrospective and Prospective Multicenter Study Evaluating the Impact of Treatment With Cytoreducing Agents on the Recurrence of Thrombosis in Thrombotic Patients With a Diagnosis of Myeloproliferative Neoplasia and Normal Blood Counts - a FIM Study
Actual Study Start Date : May 21, 2019
Estimated Primary Completion Date : May 21, 2023
Estimated Study Completion Date : May 21, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Blood Clots

Primary Outcome Measures :
  1. Cumulative incidence of recurrence of thromboembolic events after the initial thrombosis leading to the diagnosis of MPN [ Time Frame: 24 months follow-up ]
    The diagnosis of thromboembolic events must be confirmed by imaging

Secondary Outcome Measures :
  1. Cumulative incidence of hematological progression to essential thrombocythemia, polycythemia vera, secondary myelofibrosis or acute transformation [ Time Frame: 24 months follow-up ]
    Hematological progression criteria will be based on the WHO classification

  2. Cumulative incidence of recurrence of thromboembolic events according to the type and duration of anticoagulant or anti-aggregant treatment [ Time Frame: 24 months follow-up ]
    The diagnosis of thromboembolic event must be confirmed by imaging

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Thrombosis recurrence :treated and untreated patients with cytoreductive agents

Inclusion Criteria :

  • Adult ≥18 years old
  • Diagnosis of a deep arterial or venous thrombotic event verified by imaging (Doppler or CT scan)
  • Diagnosis of MPN with normal CBC according to WHO 2017 criteria characterized by the detection of a molecular abnormality JAK2V617F mutation (regardless of allelic load), CALR or MPL and/or a bone marrow biopsy with abnormalities in favor of MPN.
  • Normal blood cell count not suggestive of polycythemia vera (hematocrit<48% and hemoglobin<16g/dL for women; hematocrit<49% and hemoglobin<16.5g/dL for men), essential thrombocythemia (platelets<450 G/L) nor myelofibrosis (white blood cell count<11 G/L, no anemia or erythromyelemia associated with splenomegaly) at the time of the thrombotic event.

Exclusion Criteria :

  • Minors (<18 years of age)
  • Microcirculation disorders (erythromelalgia, headaches, paresthesia, ischemia of the extremities)
  • Superficial or deep arterial or venous thrombosis NOT verified by imaging
  • Diagnosis or history of TE, PV or myelofibrosis at time of first thrombotic event
  • Diagnosis of mixed myelodysplastic/myeloproliferative syndrome
  • Diagnosis of unclassifiable MPN with excess blasts at the onset or signs of myelodysplasia as defined by the WHO 2017 classification

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04539678

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Contact: Yannick LE BRIS, PhD 330240084630

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Nantes University Hospital Recruiting
Nantes, Loire-Atlantique, France, 44093
Contact: Yannick LE BRIS, Phd    02 40 08 46 30   
Contact: Annick COULON   
Sponsors and Collaborators
Nantes University Hospital
French Intergroup of Myeloproliferative syndromes
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Principal Investigator: Yannick LE BRIS, PhD Nantes University Hospital

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Responsible Party: Nantes University Hospital Identifier: NCT04539678    
Other Study ID Numbers: RC20_0141
First Posted: September 7, 2020    Key Record Dates
Last Update Posted: September 9, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Nantes University Hospital:
Myeloproliferative neoplasm
Additional relevant MeSH terms:
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Pathologic Processes