Azacitidine Plus Deferasirox (ICL670) in Higher Risk Myelodysplastic Syndromes (MDS)
Status:
Terminated
Trial end date:
2016-09-29
Target enrollment:
Participant gender:
Summary
Myelodysplastic syndromes are common blood disorders that can affect as many as one in 1000
Canadians over the age of 65. They are characterized by low blood counts that require
frequent blood transfusions. The development of iron overload in these patients is
inevitable. The iron deposits in vital organs such as the heart and the liver and can lead to
organ dysfunction. Deferasirox is a well-studied drug that helps remove iron from the body.
Most people with this disorder die due to progression of their disease to acute leukemia
through multiple mechanisms. Iron overload in patients with myelodysplastic syndromes has
been shown to be associated with shorter survival, and potentially a higher chance of
leukemia. In a certain sub-group of higher risk patients, the drug azacitidine has been shown
to decrease the chance of progression to leukemia and death from it. Thus, it is presently
the standard of care for these patients. However, 50% of higher risk patients are still
unresponsive to this medication, leaving a large group of patients for which other treatment
options are emergently needed. Given that a large proportion of higher risk MDS patients fail
to respond to azacitidine, and the evidence that iron deposition may lead to increased
leukemic transformation, we would like to study whether iron removal from the body with
deferasirox potentiates azacitidine in its effects on overall survival, as well as the chance
of leukemia transformation. This question needs to be addressed in a randomized controlled
trial, and the first step is a preliminary study to determine if the combination of
azacitidine and deferasirox has any biologic effect. This study will determine whether this
combination leads to blood count improvement over azacitidine alone. If this drug combination
ultimately proves more useful than azacitidine alone with respect to survival, this has the
potential to impact the care of a large proportion of patients with myelodysplastic
syndromes.