Observational Study Protocol REALFed - Real World Evidence of Fedratinib Effectiveness in Myelofibrosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 93
- 试验地点
- 1
- 主要终点
- Splenic response
研究概览
简要总结
This is a multicenter prospective and retrospective observational clinical study in patients with primary or post polycythemia vera or post essential thrombocythemia myelofibrosis to test the efficacy of fedratinib in the rea world. Participants will be managed according to the clinical practice of the participating Center. All Centers will be Italian Hematology Units belonging to the GIMEMA Organization in Italy.
详细描述
This is a multicenter prospective and retrospective observational clinical study with the aim of assessing the efficacy of fedratinib upon AIFA approval in patients with primary myelofibrosis or post polycythemia vera or post essential thrombocythemia myelofibrosis. Patients must meet current diagnostic criteria of MF, according to the WHO (World Health Organization) classification version 5th or the ICC (International Consensus Conference) either published in 2022 or post- polycythemia vera and post-essential thrombocythemia myelofibrosis (according to the ICC classification 2022). Patients enter the study at diagnosis or already in follow-up, at any stage of disease, except if transformed to blast phase. Patients who received fedratinib after June 2022 (time of AIFA reimbursement in Italy) will be enrolled and will be included both those ruxolitinib-naïve and ruxolitinib-exposed.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 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
- •Patients aged 18 years or older.
- •Patients diagnosed with primary myelofibrosis [according to the WHO (World Health Organization) classification version 5th or the ICC (International Consensus Conference) either published in 2022 or post-polycythemia vera and post-essential thrombocythemia myelofibrosis (according to the ICC classification 2022)].
- •Patients who met the reimbursement criteria for fedratinib, in accordance with the AIFA (Agenzia Italiana del Farmaco) after June
- •Patients eligible or not for stem cell transplant (SCT) or patients already undergoing SCT.
- •Patients on non-JAKi cytoreductive treatment.
- •Patients with palpable splenomegaly at baseline of fedratinib treatment.
- •Informed consent signed, if applicable.
排除标准
- •Diagnosis of MPN, unclassifiable, myelodysplastic/myeloproliferative neoplasms, myelodysplastic syndromes, essential thrombocythemia, polycythemia vera.
- •Blast phase of MF.
- •Patients with platelets <50 x10^9/L at baseline of fedratinib treatment.
- •Patients ruxolitinib-exposed for other diseases.
结局指标
主要结局
Splenic response
时间窗: 6 months
Splenic response is defined as 50% palpatory reduction.
次要结局
未报告次要终点