The DRAIHA Study: Data Registry of AutoImmune Hemolytic Anemia, to Improve Diagnostic Testing for the Development of Personalized Treatment Protocols in AIHA Patients
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 720
- 试验地点
- 2
- 主要终点
- Immunological characteristics of autoantibodies in autoimmune hemolytic anemia (AIHA) patients - laboratory tests.
研究概览
简要总结
In autoimmune hemolytic anemia (AIHA) auto-antibodies directed against red blood cells (RBCs) lead to increased RBC clearance (hemolysis). This can result in a potentially life-threatening anemia. AIHA is a rare disease with an incidence of 1-3 per 100,000 individuals. An unsolved difficulty in diagnosis of AIHA is the laboratory test accuracy. The current 'golden standard' for AIHA is the direct antiglobulin test (DAT). The DAT detects autoantibody- and/or complement-opsonized RBCs. The DAT has insufficient test characteristics since it remains falsely negative in approximate 5-10% of patients with AIHA, whereas a falsely positive DAT can be found in 8% of hospitalized individuals. Also apparently healthy blood donors can have a positive DAT. The consequences of DAT positivity are not well known and may point to early, asymptomatic disease, or to another disease associated with formation of RBC autoantibodies, such as a malignancy or (systemic) autoimmune disease. Currently, there are no guidelines to follow-up DAT positive donors.
A second unsolved difficulty is the choice of treatment in AIHA. Hemolysis can be stopped or at least attenuated with corticosteroids, aiming to inhibit autoantibody production and/or RBC destruction. Many patients do not respond adequately to corticosteroid treatment or develop severe side effects.
Currently, it is advised to avoid RBC transfusions since these may lead to aggravation of hemolysis and RBC alloantibody formation. But in case symptomatic anemia occurs, RBC transfusions need to be given. An evidence-based transfusion strategy for AIHA patients is needed to warrant safe transfusion in this complex patient group.
To design optimal diagnostic testing and (supportive) treatment algorithms, the investigators will study a group well-characterized patients with AIHA and blood donors without AIHA, via a prospective centralized clinical data collection and evaluation of new laboratory tests. With this data the knowledge of the AIHA pathophysiology and to evaluate diagnostic testing in correlation with clinical features and treatment outcome can be improved.
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
- 3 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Sufficient comprehension of the Dutch language
- •Signed informed consent by patient and/or parent/caretaker or donor
- •Patients older than 3 months
- •Patients with a positive DAT, a positive eluate and signs of hemolysis*
- •Patients with a positive DAT with complement only, negative eluate, but with signs of hemolysis
- •Donors with a (repeatedly) positive DAT and a positive eluate and/or clinically relevant cold auto-antibodies
排除标准
- •Prior inclusion in the DRAIHA study
结局指标
主要结局
Immunological characteristics of autoantibodies in autoimmune hemolytic anemia (AIHA) patients - laboratory tests.
时间窗: 12-18 months
Documentation of characteristics of autoantibodies (e.g. isotype, subtype, titer, thermal amplitude).
Assessment of hemolysis before and after therapy, reported per class of auto-immune hemolytic anemia. - laboratory tests
时间窗: 12-18 months
Documentation of hemolysis parameters (hemoglobin level (g/dL), reticulocytes (%), haptoglobin (mg/dL), bilirubin (μmol/L) and LDH(U/L)) before and after each type of therapy. AIHA classification as IgG/IgA only, IgG/IgA with complement activation or complement activation only.
次要结局
- Incidence of underlying disease that causes or is associated with AIHA.(12-18 months)
- Type of treatment prescribed as first-line, second-line or further-line treatment for AIHA.(12-18 months)
- Hematological response after each treatment line (CR, CR-u, PR and NR)(12-18 months)
- Relapse-free survival, defined as the time since the achievement of complete or partial remission until relapse of AIHA or dead from any cause.(12-18 month)
- Documentation of adverse events during the treatment of AIHA.(12-18 month)
- Characteristics of autoantibodies of DAT positive blood donors.(12-18 months)
- Assessment of hemolysis parameters after red blood cell transfusion.(1 and 7 days after transfusion)
- Change in the incidence of auto- and alloantibodies after red blood cell transfusion.(12-18 months)