Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
NCT ID: NCT04779593
Last Updated: 2023-01-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
NA
240 participants
INTERVENTIONAL
2021-07-02
2027-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
Clinical examination
Clinical data will be recorded (general clinical examination, height, weight, blood pressure,heart beat, alcohol and tobacco consumption, antecedent) as well as concurrent medication at each follow-up visit.
SF36 questionnaire
At D0, M12 and M24. This 36 item patient reported survey of patient's health is the most commonly used and validated health survey instrument for appraising quality of life.
Items are grouped in 8 scaled scores exploring multiple dimension of global health (vitality, physical functioning, bodily pain, general health, perceptions, physical role functioning, emotional role functioning, social role functioning, mental health).
Scoring will be performed as recommended by the SF-36 instruction manual to create the eight scale scores. Furthermore, these subscales sum to obtain the total SF-36 score and will be summarized into two composite scores (physical and mental quality of life).
AIMS2_SF questionnaire
At D0, M12 and M24. The Arthritis Impact Measurement Scales 2 Short Form (AIMS2-SF) is a specific tool to measure changes in global health, pain, mobility and social function in patients with arthritis. It was described in 1992 in patients with rheumatoid arthritis and osteoarthritis and include 26 items that are summarized in scales according to a predefined scoring system: mobility, physical activity (walking, bending, lifting), dexterity, household activity (managing money and medications, housekeeping), social activities, activities of daily living, pain, depression, and anxiety. The French translation has been validated and this questionnaires has been widely used in the rheumatology field to assess quality of life of patients with arthritis. Because HFE related arthropathy is very similar to osteoarthritis this questionnaire is ought to be adequate in this setting.
WOMAC questionnaire
At D0, M12 and M24. The Western Ontario and McMaster Universities Arthritis Index (WOMAC) is a widely used questionnaires specifically assessing lower limb (hips and knee) osteoarthritis. It measures five items for pain, two for stiffness and 17 for functional limitation and had been translated in French.
EQ-5D-5L questionnaire
At D0, M12 and M24. The EQ-5D is a standardized instrument which evaluates the generic quality of life (http://www.euroqol.org/). It is a preference-based health-related quality of life measure with one question for each of the five dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The answers given to EQ-5D permit to find 243 health states that can be converted into utility score anchored at 0 for death and 1 for perfect health. EQ-5D is an instrument developed in Europe, widely used in cost-utility analysis. It has been validated in a representative sample of French population.
Blood Sample Complete blood count
Blood Sample Complete blood count at D0, M6, M12, M18 and M24/end follow-up visit
Blood Sample Iron panel
Blood Sample Iron panel (serum ferritin, serum iron and serum transferrin to determine transferrin saturation according to randomization group (at M6, M12 and M18) at D0, M6, M12, M18 and M24/end follow-up visit and at each bloodletting.
Blood Sample Fasting Glucose
Blood Sample Fasting Glucose at D0 and M24/end follow-up visit
Blood sample lipid panel
Blood sample lipid panel (total cholesterol, triglycerides, HDL, LDL) at D0 and M24/end follow-up visit
Blood sample liver panel
Blood sample liver panel (total bilirubin, Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Gamma-Glutamyl Transferase) at D0 and M24/end follow-up visit
Blood sample C reactive protein
Blood sample C reactive protein at D0 and M24/end follow-up visit
BioBank
Blood sample will be collected for BioBank at D0, M12 and M24.
Medico-economical
Medico-economic data will be collected at each follow up visit.
Bloodletting - experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin". Patients will undergo bloodletting with a goal of maintaining a serum transferrin saturation equal or lower than 50 % and a serum ferritin lower than the upper limit of the normal range (300 g/L for men and 200 g/L for women).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. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit.
control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
Clinical examination
Clinical data will be recorded (general clinical examination, height, weight, blood pressure,heart beat, alcohol and tobacco consumption, antecedent) as well as concurrent medication at each follow-up visit.
SF36 questionnaire
At D0, M12 and M24. This 36 item patient reported survey of patient's health is the most commonly used and validated health survey instrument for appraising quality of life.
Items are grouped in 8 scaled scores exploring multiple dimension of global health (vitality, physical functioning, bodily pain, general health, perceptions, physical role functioning, emotional role functioning, social role functioning, mental health).
Scoring will be performed as recommended by the SF-36 instruction manual to create the eight scale scores. Furthermore, these subscales sum to obtain the total SF-36 score and will be summarized into two composite scores (physical and mental quality of life).
AIMS2_SF questionnaire
At D0, M12 and M24. The Arthritis Impact Measurement Scales 2 Short Form (AIMS2-SF) is a specific tool to measure changes in global health, pain, mobility and social function in patients with arthritis. It was described in 1992 in patients with rheumatoid arthritis and osteoarthritis and include 26 items that are summarized in scales according to a predefined scoring system: mobility, physical activity (walking, bending, lifting), dexterity, household activity (managing money and medications, housekeeping), social activities, activities of daily living, pain, depression, and anxiety. The French translation has been validated and this questionnaires has been widely used in the rheumatology field to assess quality of life of patients with arthritis. Because HFE related arthropathy is very similar to osteoarthritis this questionnaire is ought to be adequate in this setting.
WOMAC questionnaire
At D0, M12 and M24. The Western Ontario and McMaster Universities Arthritis Index (WOMAC) is a widely used questionnaires specifically assessing lower limb (hips and knee) osteoarthritis. It measures five items for pain, two for stiffness and 17 for functional limitation and had been translated in French.
EQ-5D-5L questionnaire
At D0, M12 and M24. The EQ-5D is a standardized instrument which evaluates the generic quality of life (http://www.euroqol.org/). It is a preference-based health-related quality of life measure with one question for each of the five dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The answers given to EQ-5D permit to find 243 health states that can be converted into utility score anchored at 0 for death and 1 for perfect health. EQ-5D is an instrument developed in Europe, widely used in cost-utility analysis. It has been validated in a representative sample of French population.
Blood Sample Complete blood count
Blood Sample Complete blood count at D0, M6, M12, M18 and M24/end follow-up visit
Blood Sample Iron panel
Blood Sample Iron panel (serum ferritin, serum iron and serum transferrin to determine transferrin saturation according to randomization group (at M6, M12 and M18) at D0, M6, M12, M18 and M24/end follow-up visit and at each bloodletting.
Blood Sample Fasting Glucose
Blood Sample Fasting Glucose at D0 and M24/end follow-up visit
Blood sample lipid panel
Blood sample lipid panel (total cholesterol, triglycerides, HDL, LDL) at D0 and M24/end follow-up visit
Blood sample liver panel
Blood sample liver panel (total bilirubin, Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Gamma-Glutamyl Transferase) at D0 and M24/end follow-up visit
Blood sample C reactive protein
Blood sample C reactive protein at D0 and M24/end follow-up visit
Bloodletting - control group
Patients treated with bloodletting according to current guidelines (ferritin alone). Patient will undergo bloodletting with a goal of maintaining a serum ferritin equal or lower than 50 g/L according to current clinical practice guidelines (French and European).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. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit.
BioBank
Blood sample will be collected for BioBank at D0, M12 and M24.
Medico-economical
Medico-economic data will be collected at each follow up visit.
Interventions
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Clinical examination
Clinical data will be recorded (general clinical examination, height, weight, blood pressure,heart beat, alcohol and tobacco consumption, antecedent) as well as concurrent medication at each follow-up visit.
SF36 questionnaire
At D0, M12 and M24. This 36 item patient reported survey of patient's health is the most commonly used and validated health survey instrument for appraising quality of life.
Items are grouped in 8 scaled scores exploring multiple dimension of global health (vitality, physical functioning, bodily pain, general health, perceptions, physical role functioning, emotional role functioning, social role functioning, mental health).
Scoring will be performed as recommended by the SF-36 instruction manual to create the eight scale scores. Furthermore, these subscales sum to obtain the total SF-36 score and will be summarized into two composite scores (physical and mental quality of life).
AIMS2_SF questionnaire
At D0, M12 and M24. The Arthritis Impact Measurement Scales 2 Short Form (AIMS2-SF) is a specific tool to measure changes in global health, pain, mobility and social function in patients with arthritis. It was described in 1992 in patients with rheumatoid arthritis and osteoarthritis and include 26 items that are summarized in scales according to a predefined scoring system: mobility, physical activity (walking, bending, lifting), dexterity, household activity (managing money and medications, housekeeping), social activities, activities of daily living, pain, depression, and anxiety. The French translation has been validated and this questionnaires has been widely used in the rheumatology field to assess quality of life of patients with arthritis. Because HFE related arthropathy is very similar to osteoarthritis this questionnaire is ought to be adequate in this setting.
WOMAC questionnaire
At D0, M12 and M24. The Western Ontario and McMaster Universities Arthritis Index (WOMAC) is a widely used questionnaires specifically assessing lower limb (hips and knee) osteoarthritis. It measures five items for pain, two for stiffness and 17 for functional limitation and had been translated in French.
EQ-5D-5L questionnaire
At D0, M12 and M24. The EQ-5D is a standardized instrument which evaluates the generic quality of life (http://www.euroqol.org/). It is a preference-based health-related quality of life measure with one question for each of the five dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The answers given to EQ-5D permit to find 243 health states that can be converted into utility score anchored at 0 for death and 1 for perfect health. EQ-5D is an instrument developed in Europe, widely used in cost-utility analysis. It has been validated in a representative sample of French population.
Blood Sample Complete blood count
Blood Sample Complete blood count at D0, M6, M12, M18 and M24/end follow-up visit
Blood Sample Iron panel
Blood Sample Iron panel (serum ferritin, serum iron and serum transferrin to determine transferrin saturation according to randomization group (at M6, M12 and M18) at D0, M6, M12, M18 and M24/end follow-up visit and at each bloodletting.
Blood Sample Fasting Glucose
Blood Sample Fasting Glucose at D0 and M24/end follow-up visit
Blood sample lipid panel
Blood sample lipid panel (total cholesterol, triglycerides, HDL, LDL) at D0 and M24/end follow-up visit
Blood sample liver panel
Blood sample liver panel (total bilirubin, Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Gamma-Glutamyl Transferase) at D0 and M24/end follow-up visit
Blood sample C reactive protein
Blood sample C reactive protein at D0 and M24/end follow-up visit
Bloodletting - control group
Patients treated with bloodletting according to current guidelines (ferritin alone). Patient will undergo bloodletting with a goal of maintaining a serum ferritin equal or lower than 50 g/L according to current clinical practice guidelines (French and European).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. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit.
BioBank
Blood sample will be collected for BioBank at D0, M12 and M24.
Medico-economical
Medico-economic data will be collected at each follow up visit.
Bloodletting - experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin". Patients will undergo bloodletting with a goal of maintaining a serum transferrin saturation equal or lower than 50 % and a serum ferritin lower than the upper limit of the normal range (300 g/L for men and 200 g/L for women).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. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit.
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
18 Years
ALL
No
Sponsors
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Rennes University Hospital
OTHER
Responsible Party
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Locations
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Hopital Avicenne
Bobigny, , France
CHU Dupuytren
Limoges, , France
GHBS site du Scorff
Lorient, , France
GHRMSA - Hôpital Emile Muller
Mulhouse, , France
CHR Orléans
Orléans, , France
Hôpital Européen Georges Pompidou
Paris, , France
CHU Rennes
Rennes, , France
CH Yves le Foll
Saint-Brieuc, , France
CH de St Malo
St-Malo, , France
Hôpital Rangueil
Toulouse, , France
Centre hospitalier Bretagne Atlantique
Vannes, , France
Hôpital Paul Brousse
Villejuif, , France
Countries
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Facility Contacts
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Prunelle GETTEN
Role: backup
Rodolphe Sobesky
Role: backup
Other Identifiers
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35RC19_8985_Quali-SAT
Identifier Type: -
Identifier Source: org_study_id
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