Thalassemia, NTDT, MDS, MF, Other Anemia Clinical Trial
Official title:
An Epidemiological Study to Assess the Prevalence of Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)
Iron, one of the most common elements in nature and the most abundant transition metal in
the body, is readily capable of accepting and donating electrons. This capability makes iron
a useful component of various, essential biochemical processes. Despite the essential role
of iron, the excess of iron is toxic to the human body. It is critical for the human body to
maintain iron balance, since humans have no physiologic mechanism for actively removing iron
from the body.
The development of iron overload occurs when iron intake exceeds the body's capacity to
safely store the iron in the liver, which is the primary store for iron. Long-term
transfusion therapy, a life-giving treatment for patients with intractable chronic anemia is
currently the most frequent cause of secondary iron overload.
The mounting evidence regarding the mortality and morbidity due to chronic iron overload in
transfusion dependent anaemias has led to the establishment of guidelines that aim the
improvement of patient outcomes. Further prospective studies are warranted in order to
assess the impact of iron overload in patients with acquired anaemias.
In this study, non-invasive R2- and T2*-MRI techniques will be applied to the liver and the
heart, respectively, to complement the primary variable (serum ferritin) assessed in
patients with various transfusion-dependent anaemias. The main objective of this study is to
assess the prevalence and severity of cardiac and liver siderosis in patients with
transfusional siderosis. This study will also aim to establish possible correlations between
cardiac and liver iron levels with clinical effects in patients with different
transfusion-dependent anaemias. Patients will be eligible for enrollment irrespective of
receiving chelation therapy or not (and irrespective of the chelating agent used).
This study is designed to collect information about a large cohort of patients with anaemias
including MDS, aplastic anemia, Diamond-Blackfan, myeloproliferative disorder, as well as
haemoglobinopathies (e.g. thalassaemia major, SCD) or other anaemias requiring chronic red
blood cell transfusions.
Clinical data will be collected retrospectively (if available), unless specified by this
protocol (e.g. serum ferritin within less than one month prior to enrollment). All
assessments required for this protocol should be performed after the patient informed
consent is signed. The data will be gathered by all study centers and will be combined in
one central database.
Data will be recorded using an electronic case report form (eCRF) at each study site.
Adverse events and serious adverse events will be recorded for all patients from the date of
signed patient informed consent until the MRI tests are performed.
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