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
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 253
- 试验地点
- 1
- 主要终点
- Cumulative incidence of recurrence of thromboembolic events after the initial thrombosis leading to the diagnosis of MPN
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Cumulative incidence of recurrence of thromboembolic events after the initial thrombosis leading to the diagnosis of MPN
时间窗: 24 months follow-up
The diagnosis of thromboembolic events must be confirmed by imaging
次要结局
- Cumulative incidence of recurrence of thromboembolic events according to the type and duration of anticoagulant or anti-aggregant treatment(24 months follow-up)
- Cumulative incidence of hematological progression to essential thrombocythemia, polycythemia vera, secondary myelofibrosis or acute transformation(24 months follow-up)