RH Genotype Matched Red Cell Transfusions for Patients With Sickle Cell Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- Determine the treatment efficacy by monitoring the rate of Rh alloimmunization
研究概览
简要总结
To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.
详细描述
This is a Phase 1/2 trial in patients with Sickle Cell Disease requiring chronic red cell transfusions. RH genotyped donor units will be obtained from the New York Blood Center. Patients will be matched with donor units whose RH genotypes predict no foreign Rh protein exposure to the patient. This will provide red cell matching at a level above the current standard of care (serologic C, E, and K matching). Patients will receive RH matched red cells for the duration of their chronic transfusion therapy or up to three years, whichever is shorter. In the pilot phase, we have determined it is feasible to identify RH matched donor units for the patient's RH genotype for every scheduled transfusion. We will now continue to show feasibility as well as determine efficacy by monitoring Rh alloantibody formation.
For subjects with a history of stroke/recurrent transient ischemic attack or other indication who require tight control of Hb S, and RH genotyped blood is not available, standard of care serologic matched blood would be administered rather than delaying transfusion and risking higher Hb S level.
For all subjects, standard of care serologic matched blood would be administered rather than delaying transfusion beyond 7 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects age >6 months
- •Diagnosis of SCD, all genotypes
- •Require a period of chronic red cell transfusion therapy
- •Subject/parental/guardian permission (informed consent) and if appropriate, child assent
排除标准
- •Rare RH genotype that would preclude identification of sufficient RBC units
- •Antigen negative requirements due to alloimmunization that would preclude identification of sufficient RBC units
- •Alloimmunized to D antigen
- •Rh alloimmunized patients for whom providing RH genotype matched blood would expose the patient to an antigen that would not be consistent with standard of care and blood bank protocols
- •Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures
研究组 & 干预措施
RH genotype matched red cell transfusions
Subjects will receive RH genotyped matched red cell units for transfusion in addition to standard serologic C, E, and K antigen matching and being hemoglobin S negative, which is our institutional standard of care for patients with Sickle Cell Disease.
干预措施: Red cell units that are genotype matched at the RHD and RHCE loci (Biological)
结局指标
主要结局
Determine the treatment efficacy by monitoring the rate of Rh alloimmunization
时间窗: 3.5 years
A primary objective is to determine whether providing RH genotype matched red cell units can reduce or prevent Rh alloimmunization.
Determine the feasibility of identifying sufficient RH genotype matched units
时间窗: 3.5 years
A primary objective is to determine the feasibility of identifying sufficient RH genotype matched red cells for chronically transfused patients with SCD with varied RH genotypes. Approximately 20 RHD (Rhesus D) and 20 RHCE (Rhesus CE) variants have been observed in patients with SCD and will determine whether sufficient RH genotyped units can be matched to the patient's own RH genotype.
次要结局
- Determine the rate of non-Rh alloimmunization(3.5 years)