Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Hydroxyurea adherence ratio
研究概览
简要总结
This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.
详细描述
This is a single-arm, prospective study of hydroxyurea added to simple chronic transfusions, combination treatment termed hydroxyurea and transfusion (HAT). The primary objective of the study is to determine the feasibility of HAT for patients with sickle cell anemia (SCA) currently being treated only with simple transfusions for stroke prevention. Secondary objectives include: to evaluate the safety of HAT and to determine if HAT decreases transfusion requirements in this patient population. Exploratory objectives include: to evaluate with HAT changes in pre-transfusion laboratories and biomarkers of cerebrovascular disease progression, and to describe changes on brain imaging.
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
- 2 Years 至 24 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of SCA (Hb SS or Sβ0 thalassemia).
- •On simple chronic transfusion therapy for stroke prevention (primary or secondary prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next year.
排除标准
- •Poor adherence to simple transfusion regimen as defined by having an HbS >45% at any time in the last year AND a transfusion interval >5 weeks.
- •Treatment with hydroxyurea in the 12 months prior to study enrollment.
- •Abnormal initial laboratory values (temporary exclusions):
- •Absolute neutrophil count <1.5 x 10^9/L
- •Platelet count <100 x 10^9/L
- •Serum creatinine more than twice upper limit for age
- •Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.
研究组 & 干预措施
Hydroxyurea and Transfusion (HAT)
Combination hydroxyurea and simple chronic transfusion therapy
干预措施: Hydroxyurea (Drug)
结局指标
主要结局
Hydroxyurea adherence ratio
时间窗: 1 year
(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits
Recruitment ratio
时间窗: 1 year
number of participants who enroll on the study / total number of eligible subjects
Retention ratio
时间窗: 1 year
number participants who remain on study 1 year after HAT target dose / total number enrolled participants
次要结局
- Volume of red blood cells transfused per patient weight(1 year)
- Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL(1 year)
研究者
Robert Nickel
Principal Investigator
Children's National Research Institute