deferasirox plus erythropoietin vs erythropoietin alone in MDS
Research type
Research Study
Full title
An open-label, phase II, randomized, pilot study to assess the efficacy in term of erythroid improvement of deferasirox combined with erythropoietin compared to erythropoietin alone in patients with low- and int-1-risk myelodysplastic syndrome
IRAS ID
134368
Contact name
John Porter
Contact email
Sponsor organisation
Novartis Pharmaceuticals
Eudract number
2013-000981-12
Research summary
The study is designed to test in a prospective way the combination of deferasirox (DFX )with erythropoietin (EPO) in term of their effect on hematopoiesis in lower-risk MDS patients.
Although EPO is not approved in MDS, it is the standard of care in term of improving the hematological parameters in anemic lower risk MDS patients before implementing regular blood transfusion and it is recommended in guidelines for treatment of MDS, however 60-80% of patients do not respond and require ongoing transfusions, there is currently an unmet need for this large group of patients who fail on EPO.
Several case reports and results from clinical trials suggest that some MDS patients improve their hematological parameters if they receive iron chelation therapy with DFX.
The addition of DFX to EPO could lead to a potential synergism with the reduction of reactive oxygen species, the control of free toxic iron and may create a better environment in the bone marrow for a better response with EPO.
DFX and EPO have been administered together in some studies, however this was not part of the study design and the combination was not analysed. In addition there are no published studies of the combination of EPO and Iron chelation agents. However there are different separate scientific elements that lead to a strong hypothesis on the potential synergism of both drugs.REC name
London - Dulwich Research Ethics Committee
REC reference
13/LO/1838
Date of REC Opinion
23 Jan 2014
REC opinion
Further Information Favourable Opinion