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

Sanquin Research & Blood Bank Divisions

Status and phase: Unknown; Phase 3

Conditions: Hereditary Hemochromatosis

Treatments: Other: Phlebotomy and erythrocytapheresis

Study type: Interventional

Funder types: Other

Identifiers

NCT01398644

07-2-104

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 40 estimated patients

Sex: All

Ages: 18 to 90 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Single Blind

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40 participants in 2 patient groups

Phlebotomy -intervention phlebotomy

Active Comparator group

Description:

Patients are treated with phlebotomy if ferritin level \>50 ug/l

Treatment:

Other: Phlebotomy and erythrocytapheresis

Erythrocytapheresis

Experimental group

Description:

Patients are treated with erythrocytapheresis if serum ferritin level \>50ug/l

Treatment:

Other: Phlebotomy and erythrocytapheresis