Validation d'Une Stratégie de Programme Transfusionnel Par Erythraphérèse basée Sur un Algorithme d'Aide à la Prescription Transfusionnelle Chez Les Patients Adultes Drépanocytaires
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 65
- 试验地点
- 13
- 主要终点
- Number of patients with individually achieved objectives in terms of % HbS
研究概览
简要总结
The main objective of this study is to prove the superiority of a procedure which calculates the volume of RBCs to transfuse and the time between apheresis based on this algorithm, compared to the current procedure. The primary endpoint would be the number of patients with individually achieved objectives in terms of % HbS before each apheresis (which reflects the effectiveness of the previous apheresis) over a period of 12 months. The secondary objectives would be to compare the volume differences of transfused RBCs in both groups over a period of 12 months, the occurrence of clinical events and the satisfaction of patients and physicians.
The investigators hope that this study would improve the efficiency and the performance of apheresis in sickle cell patients. The investigators also hope to facilitate the organization of procedures with the flexibility that would allow the use of this algorithm.
详细描述
Sickle cell disease (SCD) is the most common genetic disease leading to abnormal hemoglobin (HbS). Chronic complications can be severe and affect multiple organs. Among them, cerebrovascular disease is one of the most serious leading to a high risk of stroke. These complications often require blood exchange transfusions (BET) in order to replace red blood cells (RBC) containing HbS (from patients) by GR containing HbA (blood donors), and thereby stop the harmful pathophysiological cascade. The indications of long-term apheresis are mostly, but not exclusively, represented by cerebral vasculopathy (85% in our center), and chronic organ damages. Long-term BET in cerebral vasculopathy may considerably reduce the risk of stroke while stopping them leads to a recurrence of this risk, hence there is a need to do them regularly (on average every 4 to 6 weeks) with an objective of HbS ≤ 30%. This objective may be less stringent in the case of other indications.
Two methods are used: a manual method which is feasible anywhere and the apheresis which is preferred because of its better efficacy in achieving the targets of HbS percentage. It also limits transfusion hemochromatosis.
The volume required for BET by apheresis as well as the optimal period between apheresis sessions are empirically determined.
In our practice, the investigators noticed that this method did not allow to steadily obtaining the %HbS objective and the interval between apheresis was variable, in part conditioned by the availability of machines. This implies a real risk of occurrence of recurrent stroke in patients with cerebral vascular disease and may cause a lack of flexibility in the timing of appointments.
Thereby the principal investigator and the biostatistician created an algorithm to compute the volume of blood to be transfused and the interval between apheresis which are necessary to maintain an individual objective of HbS percentage. This algorithm has been obtained by statistical analysis of apheresis performed at Henri Mondor Hospital over a period of 3 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Single (Participant)
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
- •Age 18 years or older
- •Have sickle cell disease, defined as those individuals with HbSS or HbSβ0Thal
- •Included in a Blood Exchange Transfusion program (apherisis)
- •Benefiting from social insurance
- •Accepting to participate in the study and having signed the informed consent
排除标准
- •Have sickle cell disease defined with S-β+thal
- •Receiving EPO treatment
- •Pregnant or breast-feeding women
- •Lack of effective contraception in women in childbearing age
- •Patient under guardianship
结局指标
主要结局
Number of patients with individually achieved objectives in terms of % HbS
时间窗: For each apherisis over a 12 months period
次要结局
- Hemoglobin (g/dL)(For each apherisis over a 12 months period)
- Alanine aminotransferase (UI/L)(For each apherisis over a 12 months period)
- Percentage of alloimmunisation events evaluated with irregular red cell antibodies measure(For each apherisis over a 12 months period)
- Physician satisfaction survey for each participant(Month 12)
- Volume of transfused RBCs(For each apherisis over a 12 months period)
- Number of transfused RBCs(For each apherisis over a 12 months period)
- Number of apherisis per participant(Over a 12 months period)
- Hematocrit (percentage)(For each apherisis over a 12 months period)
- Aspartate aminotransferase (UI/L)(For each apherisis over a 12 months period)
- Bilirubin T (mg/dL)(For each apherisis over a 12 months period)
- Number of reticulocyte (g/L),(For each apherisis over a 12 months period)
- Lactate dehydrogenase (UI/L)(For each apherisis over a 12 months period)
- Quality of life questionnaire (SF-36)(At baseline and in 12 months)
- Percentage of reticulocyte(For each apherisis over a 12 months period)
- Creatinine (mg/L)(For each apherisis over a 12 months period)