A Prospective, Two-arm, Non Interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 1,012
- 试验地点
- 1
- 主要终点
- Number of patients with co-morbidities
研究概览
简要总结
This was a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis
详细描述
The purpose of this NIS was to gather data from the daily clinical practice of the Jakavi®-treatment in a broad patient population. In order to evaluate the direct effect of Jakavi®, only JAK inhibitor naive patients were documented in the first study arm; patients pretreated with JAK inhibitors were documented in the second study arm to evaluate the long-term efficacy of Jakavi® in this subpopulation.
The documentation of all patients was carried out prospectively and began after the baseline visit. The medical decision on which therapeutic and diagnostic measures to take was made solely by the responsible physician. The observational period per patient was 36 months. The visit schedule after the baseline visit was set by the responsible physician according to standard clinical care, the clinical condition of the respective patients and the SmPC.
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 120 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients with Primary Myelofibrosis (PMF), post-Polycythemia Vera-Myelofibrosis (PPV-MF), or post-Essential Thrombocythemia-Myelofibrosis (post-ET-MF), for whom Jakavi® therapy is indicated.
- •Patients that were informed about all aspects of this NIS and provided written informed consent.
排除标准
- 未提供
结局指标
主要结局
Number of patients with co-morbidities
时间窗: Up to 36 months
Number of patients with co-morbidities was collected
Assessment of the Quality of Life (QoL) - Short Form-36 (SF-36)
时间窗: Baseline month 6, month 12, month 24 and month 36
This questionnaire consists of questions measuring physical function, physical role limitation, pain, general health, vitality, social function, emotional role limitations, and mental health status. The scores that can be obtained from the scale vary between 0 and 100 and the increase in the scores indicates that the quality of life is high.
Overall survival
时间窗: Up to 36 months
Overall survival for JAK inhibitor naive and pretreated patients
Eastern Cooperative Oncology Group (ECOG) performance status
时间窗: Up to 36 months
The ECOG performance status is a scale used to assess how a patient's disease is progressing, assess how the disease affects the daily living abilities of the patient, and determine appropriate treatment and prognosis. The grade ranges from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead).
Ruxolitinib start and end dose
时间窗: Up to 36 months
Ruxolitinib start and end dose was collected
Change in the number of patients with constitutional symptoms
时间窗: Up to 36 months
Number of patients with change in constitutional symptoms was collected
Assessment of the Quality of Life (QoL) - Myeloproliferative Neoplasm - Symptom Assessment Form (MPN-SAF)
时间窗: Baseline, month 1, month 3, month 6, month 12, month 24 and month 36
The MPN-SAF questionnaire contains important questions that cover MF-specific symptoms whose analysis is part of the standard of care. It includes disease related symptoms each scored from 0 (absent) to 10 (worst imaginable). Total Scores range from 0-100, with higher scores indicating a greater number of symptoms and severity.
Therapy discontinuation and dose adjustments
时间窗: Up to 36 months
Number of participants with therapy discontinuation and dose adjustments was collected
Blood transfusion dependency
时间窗: Up to 36 months
Number of patients with blood transfusion dependency was collected
Safety and tolerability
时间窗: Up to 36 months
Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions
Spleen size (or volume) reduction
时间窗: Up to 36 months
Spleen size (or volume) reduction was measured by palpation
Number of patients with concomitant medications
时间窗: Up to 36 months
Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects was collected
次要结局
未报告次要终点