Clinical trial NCT01398644: Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Hereditary Hemochromatosis (HH) Patients
Official title: Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Patients With Hereditary Hemochromatosis; a Randomised, Single Blinded Sequential, Cross-over Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Hereditary Hemochromatosis
Keywords: Phlebotomy
Therapeutic erythrocytapheresis
Hereditary Hemochromatosis
maintenance therapy
Lead sponsor: Sanquin Research & Blood Bank Divisions
Sponsor class: OTHER
Collaborators: name: Maastricht University Medical Center
name: Atrium Medical Center
name: Radboud University Medical Center
name: Orbis Medical Centre
First posted: 2011-07-20
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: 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
timeFrame: one year after first phlebotomy treatment and one year after first erythrocytapheresis treatment
Secondary outcomes:
Other outcomes:
Locations:
city: Maastricht
country: Netherlands
facility: University Hospital Maastricht
Study officials:
affiliation: Sanquin Blood Supply
name: Eva Rombout, MD
role: PRINCIPAL_INVESTIGATOR
References: