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

Phase 3

• Conditions: Hereditary Hemochromatosis
• Interventions: Other: Phlebotomy and erythrocytapheresis

• Registration Number: NCT01398644
• Lead Sponsor: Sanquin Research & Blood Bank Divisions

Brief Summary

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

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.

Study Timeline

• Study Start: 2008-05
• Study First Submitted: 2011-07-19
• Study First Submitted QC: 2011-07-19
• Study First Posted: 2011-07-20
• Status Verified: 2013-12
• Primary Completion: 2013-12
• Study Completion: 2013-12
• Last Update Submitted: 2013-12-03
• Last Update Posted: 2013-12-04

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Phlebotomy -intervention phlebotomy	Phlebotomy and erythrocytapheresis	Patients are treated with phlebotomy if ferritin level \>50 ug/l
Erythrocytapheresis	Phlebotomy and erythrocytapheresis	Patients are treated with erythrocytapheresis if serum ferritin level \>50ug/l

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

University Hospital Maastricht; 🇳🇱 Maastricht, Netherlands