Long-term Follow-up of Patients Participating in RACE: the Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 197
- 试验地点
- 1
- 主要终点
- Failure Free Survival
研究概览
简要总结
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
详细描述
Patients will be followed up annually, according to standard of care.
All diagnostic and therapeutic intervention will be performed according to standard of care, at discretion of the treating physician. In particular, during the study no extra Peripheral blood or Bone Marrow sampling will be performed, in addition to routine sampling for morphology and karyotype surveillance.
Molecular analysis by Next Generation Sequencing (NGS) will also be collected if the centre is doing this on a routine basis.
No Investigational Medicinal Product (IMP) or Non-Investigational Medicinal Product (NIMP) will be given to the patients.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 接受健康志愿者
- 否
入选标准
- •Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.
- •Subject has provided informed consent to participate in long-term data collection
排除标准
- 未提供
研究组 & 干预措施
RACE 1 patients
After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study
干预措施: ATGAM plus CsA with or without Eltrombopag (Drug)
结局指标
主要结局
Failure Free Survival
时间窗: 15 years
Failure Free Survival, where treatment failure is defined as one or more of the following: death, relapse, malignant clonal evolution, need for further (e.g. transplant)
次要结局
- Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)(15 years)
- Response Rate: number of patients who reach a hematological response(15 years)
- Overall Survival(15 years)
- Number of patients who need additional IST(15 years)
- Number of patients who need Maintenance IST (e.g. CsA beyond 2 years)(15 years)
- Number of patients who need additional Eltrombopag (EPAG)(15 years)
- Cumulative incidence of relapse after response(15 years)
- Cumulative incidence of clonal evolution(15 years)
- Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)(15 years)
- Cumulative incidence of Solid Tumors(15 years)
- Number of patients who need a Human Stem Cell Transplantation (HSCT)(15 years)
- Number of patients who need any other approved Aplastic Anemia (AA) treatment(15 years)