A Prospective Observational Study of Switching Avatrombopag and Rh-TPO in Chinese Adult Patients With Primary Immune Thrombocytopenia
试验速览
- 阶段
- 不适用
- 入组人数
- 100
- 主要终点
- Initial response after switching
研究概览
简要总结
Thrombopoietin receptor agonists (TPO-RAs) represent a highly effective and well-tolerated second-line ITP treatment that provides excellent responses.If there is cross-resistance between 2 drugs for the treatment of adult ITP is still unkonwn.The purpose of this study is to investigate the efficacy and safety of switching avatrombopag and rh-TPO in adults with ITP.
详细描述
Thrombopoietin Receptor Agonists (TPO-RAs) are novel treatments for patients with chronic Primary Immune Thrombocytopenia (ITP). According to the findings of mechanism-based studies, rhTPO competes with endogenous TPO for binding to TPO-R while avatrombopag has an additive effect with endogenous TPO, indicating that the treatment mechanism and side-effect profiles could be somewhat different between these drugs. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. The purpose of this study is to investigate the efficacy and safety of switching avatrombopag and rh-TPO in adults with ITP.This is a non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to avatrombopag or vice versa will be enrolled. The reason for switch will be recorded. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1.18 years or older 2.Primary ITP 3.Failed initial glucocorticosteroid treatment, 4.Applying rhTPO or Eltrombopag as subsequent treatment 5.Switch from rh-TPO to eltrombopag or vice versa 6.Normal neutrophils 7.Available follow-up at least 6 weeks after switching
排除标准
- •HIV positive status, or active infection of HBV or HCV
- •Suffering from a serious or progressive disease, which, in the investigator's judgment, put the subject at undue risk for participation in this study (i.e. cancer or pre-cancer, immunocompromised, uncontrolled diabetes, epilepsy, severe cardio-cerebrovascular disease(s) (i.e. stroke, idiopathic aortic stenosis, aneurysm, hypertrophic obstructive cardiomyopathy, ischaemic heart disease, tachyarrhythmias, severe heart failure [classified as NYHA III-IV], severe lung dysfunctions, etc))
- •History of thrombosis plus two or more risk factors as defined in Caprini thrombosis risk assessment model
- •Lactating or pregnant women, or WOCBP who are unwilling to use highly effective contraceptive measures during the study period
- •Abnormal liver and renal functions: AST or ALT or total bilirubin ≥1.5 × ULN, and/or creatinine ≥176.8 μmol/L
- •Women of childbearing potential (WOCBP) that are pregnant or wish to become pregnant during the prospective phase of the study.
- •Other conditions which the investigator considers inappropriate for enrollment
结局指标
主要结局
Initial response after switching
时间窗: 4 weeks
Rate of response at 4 weeks after switching from rhTPO to avatrombopag or vise versa
次要结局
- Rate of response at 12 weeks after switching according to the reasons of switching(12 weeks)
- Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)(24 weeks)
- Safety assessment(24 weeks)
- Response rate at 12 weeks after switching(12 weeks)
- Time to response(4 weeks)
- Initial response after switching according to the reasons of switching(4 weeks)
- Durable response(24 weeks)
- Incidence of bleeding events(24 weeks)
- Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)(24 weeks)
研究者
Fuhaixia
associate chief physician
Peking University People's Hospital