Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 109
- 试验地点
- 1
- 主要终点
- To evaluate the incidence of aplastic anemia
研究概览
简要总结
This is a national, multicenter, ambispective, observational post-authorization study (EPA-SP for its acronym in Spanish) to describe the incidence, clinical management and outcome of aplastic anemia in hospitals throughout Spain.
详细描述
This is a national, multicenter, ambispective, observational post-authorization study (EPA-SP for its acronym in Spanish) to describe the incidence, clinical management and outcome of aplastic anemia in hospitals throughout Spain.
The study includes a retrospective chart review to identify all cases of aplastic anemia diagnosed between January 2010 and the date of the study initiation and a prospective study to detect new cases of aplastic anemia during an 18-month period since the study initiation in the participant hospitals. The study is also designed to collect both retrospective and prospective data on clinical management and outcome of patients with confirmed aplastic anemia. All patients included in the study, including those cases of aplastic anemia identified since 2010 until the study initiation through the retrospective chart review that are alive at the time of inclusion in the study, will be followed up until death or lost-to-follow-up or until 6 months after the last patient is enrolled in the study.
The study consists of a baseline visit, follow-up visits every 6 months (± 15 days) until death or lost-to follow-up or up to 6 months after the last patient is enrolled in the study, and a final visit (final/premature withdrawal visit) which will be performed at the study end (6 months after the last patient is enrolled) or in case of premature withdrawal, except for patient withdrawal of consent, loss-to-follow-up or death.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of both sexes and of any age
- •Confirmed diagnosis of spinal cord aplasia, regardless of its severity (moderate, severe or very serious), from January 1, 2010 (until the beginning of the study) (retrospective study) and during the inclusion period of 18 months in hospitals Participants (prospective study).
- •Aplasia is defined by a cellularity of the bone marrow <25% and the presence of less than the following: (i) hemoglobin <100 g / l (ii) platelet count <50 x109 / l (iii) Neutrophil count < 1.5 x 109 / l.
- •Any severity of the disease according to hematological and spinal criteria (5): moderate aplasia (absolute neutrophil count [ANC]> 0.5 x 109 / l), severe (ANC 0.2-≤0.5) x 109 / l) or very severe (RAN <0.2 x 109 / l).
- •Patients who voluntarily understand and sign the informed consent (if it can be provided), preferably in writing or orally before a witness, or will be obtained from the legal representative of the patient (for children under 16 years of age) before the start of the study. The deceased patients and the patients with whom they can not be contacted or have lost their follow-up, who have been diagnosed since January 1, 2010 until the beginning of the study, are exempt from the requirement of consent.
排除标准
- •Patients with any medical or psychological alteration that, in the opinion of the investigator, could compromise the patient's ability to grant their informed consent.
- •Evidence of Fanconi anemia, dyskeratosis congenita, congenital spinal cord syndrome, and myelodysplastic syndrome.
结局指标
主要结局
To evaluate the incidence of aplastic anemia
时间窗: 2010-2022
The primary endpoint of the study is the number of cases of aplastic anemia per year recorded in the databases of the participant hospitals from January 2010 and the date of the study initiation (retrospective analysis) and all new cases of aplastic anemia detected per year (prospective analysis) during an 18-month period since study initation.
次要结局
- 1. Number of patients diagnosed with moderate and severe aplastic anemia.(2010-2022)
- 7. Relapse-free survival, defined as the time elapsed since complete or partial remission achievement until relapse or death from any cause(2010-2022)
- 9. Percentage of patients with bleeding events an infections leading to hospitalization or death during the follow-up period.(2010-2022)
- 4. Percentage of responder patients 90, 180, 270 and 360 days after each treatment initiation.(2010-2022)
- 2. Number of patients diagnosed with aplastic anemia according to their age and sex.(2010-2022)
- 3. Type of treatment prescribed as first-line, second-line or further-line treatment for aplastic anemia(2010-2022)
- 5. Best hematological response during each treatment line (CR, PR, and NR).(2010-2022)
- 8. Progression-free survival, defined as the time elapsed since treatment initiation until disease progression or death from any cause.(2010-2022)
- 6. Overall survival, defined as the time elapsed since first-line treatment initiation until death from any cause.(2010-2022)