Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Hereditary Hemochromatosis (HH) Patients

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Patients With Hereditary Hemochromatosis; a Randomised, Single Blinded Sequential, Cross-over Trial

Conditions
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Hereditary Hemochromatosis

Intervention / Treatment
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* Other: Phlebotomy and erythrocytapheresis


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: chelating therapy forced dietary regime aged below 18 years excessive overweight ( BMI more than 35) pregnancy

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 90 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Phlebotomy -intervention phlebotomy<br>Patients are treated with phlebotomy if ferritin level >50 ug/l | Other: Phlebotomy and erythrocytapheresis<br>* Phlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes<br>|
| Experimental: Erythrocytapheresis<br>Patients are treated with erythrocytapheresis if serum ferritin level >50ug/l | Other: Phlebotomy and erythrocytapheresis<br>* Phlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 |  | one year after first phlebotomy treatment and one year after first erythrocytapheresis treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Phlebotomy, Therapeutic erythrocytapheresis, Hereditary Hemochromatosis, maintenance therapy