Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 120
- 试验地点
- 6
- 主要终点
- Complete hematological response
研究概览
简要总结
First-line treatment for patients with polycythemia vera, essential thrombocythemia, and pre-myelofibrosis is based on hydroxyurea or pegylated interferon. The objective of treatment is to prevent thrombotic complications and leukemic transformation. Despite overall good response rates, some patients do not respond to treatment and others lose their response over time. Both situations are associated with worse survival and there are to date no clear predictive factors for response although the existence of additional mutations seems unfavorable.
In this exploratory study, we hypothesize that biological factors at diagnosis are associated with hematological response at 12 months. We will more specifically study the association between mutational profile, assessed by next-generation sequencing, and cytokine profile with hematological response.
This study will help in identifying patients who will not respond to hydroxyurea or pegylated interferon and give the opportunity to try other treatments upfront, in the perspective of precision medicine. On the basic science side, this study will help in understanding the molecular and immunological factors involved in resistance to treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis.
- •Indication for first-line treatment with hydroxyurea or pegylated interferon.
- •Consent to participate.
- •Affiliated to social security.
排除标准
- •Previous treatment.
- •Other on-going malignancy, including overt myelofibrosis.
- •Other treatment such as phlebotomy solely, ruxolitinib, anagrelide, or pipobroman.
结局指标
主要结局
Complete hematological response
时间窗: 12 months
ELN-2013 criteria by meeting all of the following: * Durable resolution of disease-related signs including palpable hepatosplenomegaly, large symptoms improvement, AND * Durable peripheral blood count remission, defined as: platelet count ≤400 ×109/L, WBC count \<10 × 109/L, Ht lower than 45% without phlebotomies (for PV patients), absence of leukoerythroblastosis, AND * Without signs of progressive disease, and absence of any hemorrhagic or thrombotic events.
次要结局
- Complete hematological response(24, 36, 48, and 60 months)
- Molecular response(12 and 24 months)