Clinical trial NCT04779593: Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
Official title: Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Haemochromatosis
Lead sponsor: Rennes University Hospital
Sponsor class: OTHER
First posted: 2021-03-03
Enrollment: 240
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* - 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: The primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years)
timeFrame: At Month 24
Secondary outcomes:
measure: the AIMS2- SF (short form of the Arthritis Impact Measurement Scales 2) questionnaire
timeFrame: at Day 0, Month 12 and Month 24 follow-up visit
measure: the WOMAC (Western Ontario and McMaster Universities Arthritis Index) questionnaire
timeFrame: at Day 0, Month 12 and Month 24 follow-up visit
measure: Evolution of the Mental Component Score of the SF-36 questionnaire
timeFrame: At Month 24
measure: Evolution of the Physical Component Score of the SF-36
timeFrame: at Day 0, Month 12 and Month 24 follow-up visit
measure: Evolution of the Mental Component Score of the SF-36
timeFrame: at Day 0, Month 12 and Month 24 follow-up visit
measure: Evolution of of the EQ-5D-5L score
timeFrame: at Day 0, Month 12 and Month 24 follow-up visit
measure: Evolution of Serum ferritin and serum transferrin saturation
timeFrame: Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit
measure: Occurrence of anaemia
timeFrame: Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit
measure: Occurrence of malaise
timeFrame: At Month 24
measure: Total number of phlebotomy performed
timeFrame: At Month 24
measure: Incremental Cost-Effectiveness Ratio
timeFrame: At Month 24
Other outcomes:
Locations:
city: Bobigny
country: France
facility: Hopital Avicenne
city: Limoges
country: France
facility: CHU Dupuytren
city: Lorient
country: France
facility: GHBS site du Scorff
city: Mulhouse
country: France
facility: GHRMSA - Hôpital Emile Muller
city: Orléans
country: France
facility: CHR Orléans
city: Paris
country: France
facility: Hôpital Européen Georges Pompidou
city: Rennes
country: France
facility: CHU Rennes
city: Saint-Brieuc
country: France
facility: CH Yves le Foll
city: Saint-Malo
country: France
facility: CH de St Malo
city: Toulouse
country: France
facility: Hôpital Rangueil
city: Vannes
country: France
facility: Centre hospitalier Bretagne Atlantique
city: Villejuif
country: France
facility: Hôpital Paul Brousse
Study officials:
References: