Mi-iron - Moderately Increased Iron - is Reducing Iron Overload Necessary?

Murdoch Childrens Research Institute

Status: Completed

Conditions: Hereditary Haemochromatosis

Treatments: Procedure: Erythrocytapheresis; Procedure: Plasmapheresis

Study type: Interventional

Funder types: Other

Identifiers

NCT01631708

04609

Details and patient eligibility

About

Haemochromatosis is a preventable genetic iron overload disorder. Untreated, it can shorten life due mainly to liver cirrhosis and cancer. It can be prevented by blood donation to maintain normal iron levels. It is unclear, however, whether treatment is necessary when individuals have moderate elevation of iron in the body. This research project will study the effects of treatment in this group by assessing a number of scans, questionnaires and blood tests in treated and untreated individuals.

Full description

There is mounting evidence that treatment of moderate iron overload in HFE related hereditary haemochromatosis (HH) is not necessary. This project aims to undertake a randomised patient-blinded trial of erythrocytapheresis compared to sham erythrocytapheresis (using plasmapheresis) in individuals who have serum ferritin (SF) above the upper limit of the normal range but < 1000ug/L (defined here as moderate iron overload) due to HFE mutations and to compare the prevalence of symptoms and objective markers of disease in the two treatment arms.

Enrollment: 100 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

1. HFE C282Y homozygous.
2. Aged 18 - 70 years .
3. SF above the upper limit of the normal range of 300µg/L but less than 1000µg/L with a currently or previously raised TS (>greater than the upper limit of normal for the testing laboratory).

Exclusion criteria

1. HH due to genotypes other than HFE C282Y homozygosity.
2. Normal SF, SF > 1000µg/L.
3. Other major risk factor(s) for liver toxicity or other significant co-morbidities including positivity for hepatitis B or C, excess alcohol consumption (> 60g/day in males and 40g/day in females) or body mass index > 35.
4. Has had venesection therapy for HH in the last two years.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: Single Blind

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100 participants in 2 patient groups

Erythrocytapheresis

Active Comparator group

Description:

Erythrocytapheresis is a procedure whereby whole blood is drawn from an individual and all elements except erythrocytes are returned to the donor. An automated filtration process removes the erythrocytes. Those in arm 1 will have third weekly erythrocytapheresis until their SF is returned to the normal range.

Treatment:

Procedure: Erythrocytapheresis

Plasmapheresis

Sham Comparator group

Description:

In plasmapheresis, the plasma is removed by the automated filtration process whilst other blood elements including erythrocytes are returned to the subject. Those in arm 2 will have plasmapheresis with the approximate number of episodes of apheresis that would be required to reduce their SF to normal had they been randomised to the true treatment arm.

Treatment:

Procedure: Plasmapheresis