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

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

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.

Study Overview

• Status: Unknown
• Conditions: Hereditary Hemochromatosis
• Intervention / Treatment: Other: Phlebotomy and erythrocytapheresis

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 Type: Interventional
• Enrollment (Anticipated): 40
• Phase: Phase 3

Contacts and Locations

Study Locations

• Netherlands
  • Maastricht, Netherlands, 6300
    • University Hospital Maastricht

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 86 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Sanquin Research & Blood Bank Divisions
• Collaborators: Radboud University Medical Center; Maastricht University Medical Center; Atrium Medical Center; Orbis Medical Centre
• Investigators: Principal Investigator: Eva Rombout, MD, Sanquin Blood Supply

Study record dates

Study Major Dates

• Study Start: May 1, 2008
• Primary Completion (Anticipated): December 1, 2013
• Study Completion (Anticipated): December 1, 2013

Study Registration Dates

• First Submitted: July 19, 2011
• First Submitted That Met QC Criteria: July 19, 2011
• First Posted (Estimate): July 20, 2011

Study Record Updates

• Last Update Posted (Estimate): December 4, 2013
• Last Update Submitted That Met QC Criteria: December 3, 2013
• Last Verified: December 1, 2013

More Information

Terms related to this study

• Keywords: Phlebotomy; Hereditary Hemochromatosis; maintenance therapy; Therapeutic erythrocytapheresis
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Iron Metabolism Disorders; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Iron Overload; Hemochromatosis; Hemosiderosis
• Other Study ID Numbers: 07-2-104