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

September 8, 2021 updated by: Nantes University Hospital

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

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.

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Anticipated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Thrombosis recurrence :treated and untreated patients with cytoreductive agents

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
24 months follow-up

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
24 months follow-up
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
24 months follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nantes University Hospital

Collaborators

French Intergroup of Myeloproliferative syndromes

Investigators

  • Principal Investigator: Yannick LE BRIS, PhD, Nantes university hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 21, 2019

Primary Completion (Anticipated)

May 21, 2023

Study Completion (Anticipated)

May 21, 2023

Study Registration Dates

First Submitted

August 31, 2020

First Submitted That Met QC Criteria

August 31, 2020

First Posted (Actual)

September 7, 2020

Study Record Updates

Last Update Posted (Actual)

September 9, 2021

Last Update Submitted That Met QC Criteria

September 8, 2021

Last Verified

September 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RC20_0141

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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