Observational, Retrospective and Prospective Study on the Use of Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 620
- 试验地点
- 12
- 主要终点
- Rate of vascular events after ruxolitinib exposure in myelofibrosis patients
研究概览
简要总结
The RUXOREL-MF observational study includes patients with primary and post-essential thrombocythemia/post-polycythemia vera myelofibrosis (MF) being treated with the oral JAK1-/JAK2-inhibitor ruxolitinib in a "real world" setting. Patients are treated according to current indications in Italy (i.e., primary and secondary MF patients with intermediate-1, intermediate-2, and high risk IPSS (International Prognostic Scoring System) scores and symptomatic splenomegaly and/or systemic symptoms). Patients are treated at facilities pertaining to the regional Hematology Network of Lombardy (Rete Ematologica Lombarda) in Italy. Efficacy data, data related to infectious and vascular events, data related to second primary malignancies, data regarding disease progression/transformation, and molecular information in relationship to ruxolitinib treatment will be collected and analyzed.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •Age >= 18 years
- •Diagnosis of primary myelofibrosis diagnosis according to the WHO 2016 classification or post-essential thrombocythemia/post-polycythemia vera myelofibrosis according to the IWG-MRT 2008 classification
- •Patients with an intermediate-1, intermediate-2, or high risk score according to the IPSS (International Prognostic Scoring System)
- •Patients treated with ruxolitinib in accordance with current indications in Italy
- •Patients eligible or ineligible to hematopoietic stem cell transplant or who have already undergone a hematopoietic stem cell transplant
排除标准
- •Diagnoses other than primary myelofibrosis or post-essential thrombocythemia/post-polycythemia vera myelofibrosis
- •Patients treated with ruxolitinib having a platelet count at treatment initiation <50 x10^9/L
- •Patients treated with ruxolitinib for conditions other than primary myelofibrosis or post-essential thrombocythemia/post-polycythemia vera myelofibrosis
结局指标
主要结局
Rate of vascular events after ruxolitinib exposure in myelofibrosis patients
时间窗: Through study completion, an average of 1 year
Rate of infectious events after ruxolitinib exposure in myelofibrosis patients
时间窗: Through study completion, an average of 1 year
次要结局
- Spleen response rate(At 3 and 6 months from ruxolitinib start)
- Rate of primary secondary malignancies(Through study completion, an average of 1 year)
- Acute myeloid leukemia transformation rate(Through study completion, an average of 1 year)
- Rate of primary secondary malignancies according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)(Through study completion, an average of 1 year)
- Acute myeloid leukemia transformation rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)(Through study completion, an average of 1 year)
- Rate of infectious events according to the presence of additional mutations(Through study completion, an average of 1 year)
- Rate of vascular events according to the presence of additional mutations(Through study completion, an average of 1 year)
- Rate of infectious events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)(Through study completion, an average of 1 year)
- Rate of vascular events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)(Through study completion, an average of 1 year)
- Spleen response rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)(Through study completion, an average of 1 year)
- Spleen response rate according to the presence of additional mutations(Through study completion, an average of 1 year)
- Rate of primary secondary malignancies according to the presence of additional mutations(Through study completion, an average of 1 year)
- Acute myeloid leukemia transformation rate according to the presence of additional mutations(Through study completion, an average of 1 year)
- Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation(Through study completion, an average of 1 year)
研究者
Margherita Maffioli
Principal Investigator
Ospedale di Circolo - Fondazione Macchi