Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Patients With Hereditary Hemochromatosis; a Randomised, Single Blinded Sequential, Cross-over Trial
试验速览
- 阶段
- 3 期
- 发起方
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- The difference in number of required treatments and the interval between treatments per year to keep the serum ferritin levels between 30-50 microg/L
研究概览
简要总结
Hereditary hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload. Phlebotomy is currently the standard therapy. More recently Therapeutic Erythrocytapheresis (TE) has become a new therapeutic modality, which potentially offers a more efficient method to remove iron overload with fewer procedures.In the proposed clinical trial the investigators will examine whether TE can keep the ferritin levels in patients requiring maintenance therapy below 50 microg/L, with minimally half the number of treatment procedures when compared to current standard therapy by P.
详细描述
The research population exists of patients with HH ( by genetic analysis confirmed as homozygous for C282Y) living in south-east of the Netherlands and currently treated with phlebotomy as maintenance treatment to keep their serum ferritin levels < 50 ug/l. Ferritin level at start of the inclusion between 30-50ug/l. Exclusion criteria are: patient receiving other therapies such as chelating therapy or forced dietary regimen, further patients with excessive overweight (BMI>35). After enrollment the patients will be randomized to start either with TE or continue with P. After a year of treatment and being at a serum ferritin level <50ug/l, patients will continue the study but then being treated with the other of the two treatments. Randomization will be done by blocked randomization.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •homozygous for C282Y
- •currently treated with phlebotomy as maintenance therapy for at least 6 month
- •ferritin level between 30-50 micog/L
- •age 18 years an older
- •weight more than 50 kg
- •signed informed consent
- •willingness to fill out additional questionnaires at three points in time
排除标准
- •chelating therapy
- •forced dietary regime
- •aged below 18 years
- •excessive overweight ( BMI more than 35)
- •pregnancy
结局指标
主要结局
The difference in number of required treatments and the interval between treatments per year to keep the serum ferritin levels between 30-50 microg/L
时间窗: one year after first phlebotomy treatment and one year after first erythrocytapheresis treatment
次要结局
未报告次要终点
研究者
Eva Rombout
Transfusion specialist
Sanquin Research & Blood Bank Divisions