Secondary hemochromatosis Hereditary anemia Proton pump inhibitor
Conditions
Interventions
12 months treatment with esomeprazole 40 mg twice daily or 12 months treatment with placebo twice daily.
Sponsors
University Medical Center Utrecht
Eligibility
Inclusion criteria
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
Exclusion criteria: o Pregnancy. o Liver cirrhosis. o Heart failure. o Severe cardiac iron overload defined as MRI T2* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 1. Tolerability of esomeprazole: the incidence of side effect / adverse events will be monitored every 3 months during study visits. 2. Quality of life: this will be assessed with EQ5D-forms, with time intervals of 3 months. 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. 4. Related changes in markers of iron metabolism: plasma hepcidin, serum ferritin. 5. Compliance to study drug. 6. Need for chelation therapy. | — |
Contacts
Public ContactA.J. van Vuren
Huispost C01.425 Postbus 85500
Outcome results
None listed