PPI in secondary hemochromatosis.

Completed

Conditions: Secondary hemochromatosisHereditary anemiaProton pump inhibitor

Registration Number: NL-OMON25811

Lead Sponsor: niversity Medical Center Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 30

Inclusion Criteria

o diagnosis of hereditary anemia: hemoglobinopathy (including all sickle cell syndromes and beta-thalassemia), sideroblastic anemia, congenital dyserythropoietic anemia or an erythrocyte enzyme deficiency.
o hemoglobin level before study inclusion <7.0 mmol/L.
o clinically stable and relevant iron overload defined as either one of:
o a baseline LIC measurement by MRI between 3 and 15 mg Fe/g without having received iron chelation 2 months prior to entering the study.
o OR a baseline LIC measurement by MRI between 3 and 15 mg Fe/g on stable chelation therapy (deferasirox, deferoxamine or deferiprone), with documented stable dosage the preceding 2 months and no expected dose reductions or increases the next two years.
o aged more than 18 years and able to sign informed consent.
o serum transferrin saturation higher than 0.40 once during the preceding 24 months.
o received less than 10 units of blood during the preceding 12 months.
o is expected to receive less than 4 units of blood during the following 12 months
o is not splenectomized during the preceding 24 months.

Exclusion Criteria

o Pregnancy.
o Liver cirrhosis.
o Heart failure.
o Severe cardiac iron overload defined as MRI T2* < 20 ms.
o Severe liver iron overload defined as MRI LIC > 15 mg Fe/g dw.
o Expected poor compliance.
o Currently taking PPI and not able to stop for personal or medical reasons.
o Patients that are being phlebotomized as treatment for iron overload.
o Current peptic ulcer disease, gastro-intestinal bleeding or other causes of blood loss.
o Contra-indication for esomeprazole use.
o Concomitant use of clopidogrel.
o Contra-indication for MRI.
o Received more than 4 units blood during one of the treatment periods of 12 months.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The change in LIC measured by MRI of the liver expressed in milgram Fe/gram dry weight after one year of treatment with esomeprazole compared to one year treatment with placebo.

Secondary Outcome Measures

Name	Time	Method
1. Tolerability of esomeprazole: the incidence of side effect / adverse events will be monitored every 3 months during study visits. <br /><br>2. Quality of life: this will be assessed with EQ5D-forms, with time intervals of 3 months. <br /><br>3. Cost-effectiveness analysis of esomeprazole in treatment of iron overload in hereditary anemia. This will be assessed by a prospective cost-effectiveness analysis. IMCQ and iPCQ questionnaires will be filled in with time intervals of 3 months.<br /><br>4. Related changes in markers of iron metabolism: plasma hepcidin, serum ferritin.<br /><br>5. Compliance to study drug.<br /><br>6. Need for chelation therapy.<br>