Response Rate and Response Duration After Discontinuation of Treatment With Thrombopoietin Receptor Agonists in Patients Affected by Primary Immune Thrombocytopenia (pITP): Retrospective Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 148
- 试验地点
- 13
- 主要终点
- Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation.
研究概览
简要总结
The primary objective of the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included.
详细描述
Increasing evidence exists in the literature on the persistent response after TPO-RAs discontinuation. However, the available data consist of description of case reports from different patients' series. At this time, systematic data collections on this topic do not exist and the real incidence of such persistent response is unknown. Therefore, because of the increasing interest in this new and challenging therapeutic field, also for the therapeutic implications, we would like to investigate the impact of TPO- RAs on the achievement of sustained and persistent responses after their discontinuation in the Italian adult patients affected by pITP who have been referred to the GIMEMA Centers.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
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
- •Signed written informed consent according to ICH/EU/GCP and national local laws;
- •Persistent or chronic pITP adult patients who have been treated with TPO-RAs therapy after failure of one or more therapy lines, splenectomy included;
- •Age > 18 years.
排除标准
- •Active malignancy;
- •Active malignancy;
- •HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- •Chronic liver disease;
- •Treatment with Rituximab less than 8 weeks before TPO-RAs start;
- •Recent splenectomy (less than 8 weeks) before TPO-RAs start. Active malignancy;
- •HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- •Chronic liver disease;
- •Treatment with Rituximab less than 8 weeks before TPO-RAs start;
- •Recent splenectomy (less than 8 weeks) before TPO-RAs start. HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- •Chronic liver disease;
- •Treatment with Rituximab less than 8 weeks before TPO-RAs start;
- •Recent splenectomy (less than 8 weeks) before TPO-RAs start.
结局指标
主要结局
Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation.
时间窗: One year from start of recruitment.
次要结局
- Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.(One year from start of recruitment.)
- Frequency of the type of response in patients treated with TPO-RAs in absence of concomitant or rescue medication, in order to estimate the rate of Response (R) and Complete Response (CR).(One year from start of recruitment.)
- Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.(One year from start of recruitment.)
- Description of Platelet levels and TPO-RAs dosage at stop.(One year from start of recruitment.)
- Description of patients' characteristics in term of: biological and clinical features, disease duration at the beginning of TPO-RAs, number and type of therapy lines preceding TPO-RAs treatment, platelet levels at TPO-RAs start.(One year from start of recruitment.)
- Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.(One year from start of recruitment.)
- Estimation of the duration of sustained response in terms of patients alive in CR or R at 6 and 12 months after sustained response achievement.(One year from start of recruitment.)
- Estimation of treatment duration starting from the date of first dose of TPO-RAs.(One year from start of recruitment.)
- Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.(One year from start of recruitment.)