Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy (FORTE)
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 16
- 试验地点
- 11
- 主要终点
- Platelet counts over time during the course of fostamatinib therapy
研究概览
简要总结
Evaluate patient characteristics, treatment patterns, clinical outcomes and safety of fostamatinib as second-line therapy in real-world clinical practice, for the treatment of ITP in adult patients who have an insufficient response to prior therapy (steroids ± immunoglobulins).
详细描述
Evaluate patient characteristics, treatment patterns, clinical outcomes and safety of fostamatinib as second-line therapy in real-world clinical practice, for the treatment of ITP in adult patients who have an insufficient response to prior therapy (steroids ± immunoglobulins).
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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to provide written informed consent
- •Diagnosis of ITP in adult patients (≥ 18 years of age) with an insufficient response to steroids ± immunoglobulins and requiring a change in therapy
- •For Cohort 1, intent to initiate fostamatinib at enrollment as change in therapy
- •For Cohort 2, currently undergoing treatment with fostamatinib as second line therapy for at least 12 weeks, with the intent to continue, at the time of enrollment
排除标准
- •Any prior ITP therapy/treatment other than steroids ± immunoglobulins (e.g. rituximab, thrombopoietin receptor agonists (TPO), splenectomy)
- •Participation in any interventional study during the course of this study
- •Lack of historical platelet counts, at time of fostamatinib initiation for those entering Cohort 2
- •Pregnant and/or nursing
研究组 & 干预措施
Initiating treatment with fostamatinib as second-line therapy
干预措施: Fostamatinib (Drug)
Treated with fostamatinib for at least 12 weeks as second-line therapy
干预措施: Fostamatinib (Drug)
结局指标
主要结局
Platelet counts over time during the course of fostamatinib therapy
时间窗: 12-month observation period
Platelet count and change from baseline in platelet count over time.
Safety of fostamatinib
时间窗: 12-month observation period
Evaluate continued safety of fostamatinib in ITP patients through adverse events
ITP-PAQ Quality of life measure
时间窗: 12-month observation period
Use of the score from questionnaire to assess disease-specific quality of life in adults with ITP.
Treatment satisfaction measure (MSQ)
时间窗: 12-month observation period
Fostamatinib dosing
时间窗: 12-month observation period
Changes in fostamatinib dosing
Use of ITP Rescue Medication
时间窗: 12-month observation period
The number of patients with ITP rescue medication use at any time during the study.
SF-36 Quality of life measure
时间窗: 12-month observation period
Scale of functional health and well-being from the patient's point of view.
Use of concomitant medications related to ITP
时间窗: 12-month observation period
Use of concomitant medications related to ITP during the study.
次要结局
未报告次要终点