Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 239
- 试验地点
- 24
- 主要终点
- The primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years)
研究概览
简要总结
Patients in maintenance treatment for HFE hemochromatosis since at least one year will be included in a two year study period and randomized in two groups experimental and control group. Because proton pump inhibitors are widely used as chronic medication, and because they can significantly modify iron absorption, patients will be stratified according to the use of proton pump inhibitors and gender. A first bloodletting will be performed at inclusion with the same volume as usually performed by the patients. Results of the biological test performed at this visit will guide the time schedule and volume of the next bloodletting according to randomization group (patients treated with bloodletting according to current guidelines "ferritin alone" versus patients treated with bloodletting according to "transferrin saturation and serum ferritin").Blood count and iron metabolism parameters will be performed at each bloodletting and follow-up visits. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit. Volume and schedule for bloodlettings will be determined according to guidelines specifically designed for this study to assure harmonization of treatment management, and centrally validated through the recording of the biological tests in the electronic Case Report Form which will provide the investigator with the volume and schedule of the next bloodletting. There will be two ways of treatment modification: either change of schedule or volume of bloodletting. Patients will undergo follow-up visit every six months with clinical examination, questionnaires at J0, M12 and M24 (SF-36; AIMS2-SF, WOMAC, EQ-5D-5L), and biological test. For health economics analysis, data will be obtained thanks to a dedicated extraction from SNDS database SNDS database will allow to gather hospital stays, visits, and other healthcare-related costs as well as vital status (date (month/year) of death) and cause of death. A de-identified copy of the clinical database, restricted to the relevant variables, will be sent for semideterministic matching purpose with SNDS extraction using four key variables: gender, same date (month/year) of birth, same date (day/month/year) of visit for bloodletting; pending, of course, regulatory authorization.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
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
- •- Patients treated with iron chelators;
- •Patients treated with erythroid growth factors (erythropoietin);
- •Patient with excessive alcohol consumption (> 20g/day and > 30 g/day for women and men respectively);
- •Patients with chronic haematological condition;
- •Patients having uncontrolled chronic blood loss (of digestive or gynaecological origin);
- •Patients with chronic kidney failure;
- •Patients with a diagnosis of cancer or history of cancer in the last year;
- •Pregnancy or breast feeding.
- •Patient who are included in another research protocol
- •Adults legally protected (judicial protection, guardianship, or supervision), persons deprived of their freedom.
- •with C282Y homozygous HFE hemochromatosis;
- •having finished the initial phase of HFE hemochromatosis treatment and in maintenance treatment for at least one year;
- •having signed an informed consent form.
排除标准
- •Patients treated with iron chelators;
- •Patients treated with erythroid growth factors (erythropoietin);
- •Patient with excessive alcohol consumption (> 20g/day and > 30 g/day for women and men respectively);
- •Patients with chronic haematological condition;
- •Patients having uncontrolled chronic blood loss (of digestive or gynaecological origin);
- •Patients with chronic kidney failure;
- •Patients with a diagnosis of cancer or history of cancer in the last year;
- •Pregnancy or breast feeding.
- •Patient who are included in another research protocol
- •Adults legally protected (judicial protection, guardianship, or supervision), persons deprived of their freedom.
研究组 & 干预措施
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Clinical examination (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Clinical examination (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: SF36 questionnaire (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: WOMAC questionnaire (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: EQ-5D-5L questionnaire (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Blood Sample Complete blood count (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Blood Sample Iron panel (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Blood Sample Fasting Glucose (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Blood sample lipid panel (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Blood sample liver panel (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Blood sample C reactive protein (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: BioBank (Biological)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Medico-economical (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: Bloodletting - experimental group (Procedure)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: AIMS2_SF questionnaire (Other)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: SF36 questionnaire (Other)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: WOMAC questionnaire (Other)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: EQ-5D-5L questionnaire (Other)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Blood Sample Complete blood count (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Blood Sample Iron panel (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Blood Sample Fasting Glucose (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Blood sample lipid panel (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Blood sample liver panel (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Blood sample C reactive protein (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Bloodletting - control group (Procedure)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: BioBank (Biological)
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
干预措施: Medico-economical (Other)
experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
干预措施: AIMS2_SF questionnaire (Other)
结局指标
主要结局
The primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years)
时间窗: At Month 24
The primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years). Physical Component score of the SF-36 has been chosen because SF-36 it is a widely validated and reproducible questionnaire, and this component is best susceptible to reflect both fatigue and joint involvement.
次要结局
- Evolution of the Physical Component Score of the SF-36(at Day 0, Month 12 and Month 24 follow-up visit)
- the AIMS2- SF (short form of the Arthritis Impact Measurement Scales 2) questionnaire(at Day 0, Month 12 and Month 24 follow-up visit)
- Total number of phlebotomy performed(At Month 24)
- the WOMAC (Western Ontario and McMaster Universities Arthritis Index) questionnaire(at Day 0, Month 12 and Month 24 follow-up visit)
- Evolution of the Mental Component Score of the SF-36(at Day 0, Month 12 and Month 24 follow-up visit)
- Occurrence of malaise(At Month 24)
- Evolution of the Mental Component Score of the SF-36 questionnaire(At Month 24)
- Evolution of of the EQ-5D-5L score(at Day 0, Month 12 and Month 24 follow-up visit)
- Evolution of Serum ferritin and serum transferrin saturation(Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit)
- Occurrence of anaemia(Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit)
- Incremental Cost-Effectiveness Ratio(At Month 24)