An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® (Avatrombopag) in Adult Patients With Immune Thrombocytopenia (ITP)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 199
- 试验地点
- 55
- 主要终点
- Cumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment.
研究概览
简要总结
This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice. Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
详细描述
This is a multi-center, observational, Phase 4 study in patients with ITP designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice.
Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for the treatment of ITP.
The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective collecting data on usage, effectiveness, safety, patient- and clinician-reported outcomes and health economic parameters whereas the retrospective part will consist of collection of information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Data will be collected for up to 12 months prior to Doptelet treatment start.
Prospective data will be collected at routine clinical visits throughout the study. Patients will be followed for 12 (+6) months and will be enrolled until their first scheduled visit after 12 months of enrollment, or until early termination, whichever occurs first.
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
- •Patient is ≥18 years of age
- •Established and well documented ITP diagnosis
- •Patient is treated with, or at enrollment prescribed, Doptelet for ITP. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study
- •Signed and dated informed consent provided by the patient before any study-related activities are undertaken
- •Willing and able to comply with protocol requirements
排除标准
- •Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
- •ITP secondary to Evan's syndrome, lupus and other autoimmune diseases
- •ITP secondary to other hematological disorders and hematological malignancies
- •ITP secondary to any other malignancies
- •ITP secondary to known drug toxicity
- •ITP secondary to any other disease considered relevant by the Investigator
研究组 & 干预措施
Full-analysis set (FAS)
The FAS includes all enrolled patients. The FAS will be used for all analyses.
干预措施: Avatrombopag (Drug)
结局指标
主要结局
Cumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment.
时间窗: Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.
Laboratory measures of platelet count will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts will be based on local laboratory results. Platelet count is an accepted surrogate marker for bleeding risk. Platelet count is a standard measurement commonly used both in clinical practice and in studies in patients with ITP. Platelet counts during rescue medication use and within 4 weeks after stopping a rescue medication or following splenectomy will not be counted in the cumulative number of weeks.
次要结局
- Cumulative number of weeks with a platelet count ≥50×109/L during Doptelet treatment.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Number and proportion of patients with a platelet count ≥30×109/L, for at least 8 consecutive weeks.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Number and proportion of patients with a platelet count ≥50×109/L for at least 8 consecutive weeks.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Number and proportion of patients experiencing WHO bleeding grade ≥ 2.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Number and proportion of patients requiring rescue medication.(Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Time from Doptelet treatment start to platelet count ≥30×109/L.(Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Time from Doptelet treatment start to platelet count ≥50×109/L.(Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Doptelet dose and dosing frequency per patient (assessed by prescription).(Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Adherence to Doptelet treatment assessed via the 8-item Morisky Medication Adherence Scale (MMAS-8).(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Reason for ITP treatment discontinuation or change from one ITP treatment to another, prior to as well as during the study.(Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Patient satisfaction with outcome of Doptelet treatment using the Treatment Satisfaction Questionnaire for Medication (TSQM-9).(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Physician satisfaction with outcome of Doptelet treatment using a 5 point scale.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Physician assessment of clinical change of Doptelet treatment using the Clinical Global Impression of Change (CGIC) scale.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Change from enrollment in the European Quality of Life - 5 Dimensions (EQ-5D-5L) scale.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Change from enrollment in the Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ).(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Change from enrollment in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue) questionnaire.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Change from enrollment in Patient Global Impression of Change (PGIC) scale.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Change from enrollment in Work Productivity and Activity Impairment (WPAI) questionnaire.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Healthcare resource use including; Inpatient and /or outpatient visits since last routine visit.(Data will be collected retrospectively via the medical records for a time period of 12 months prior to Doptelet start as well as prospectively for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Use of concomitant ITP medications throughout the study.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Serious adverse events (SAEs)(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- Adverse events of special interest (AESIs) (e.g., thromboembolic events, significant bleeding (WHO bleeding scale grade ≥ 3)).(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)
- AEs leading to Doptelet discontinuation.(Data will be collected for all routine visits completed during the study period which is at least 12 but not more than 18 months.)