Clinical Trials. gov identifier:
N° of patients expected by the study:
End of the enrollment
> 18 years old
Myelodysplasia object of the study:
Refractory Anemia (RA), Refractory Anemia with Ring Sideroblasts (RARS), Refractory Cytopenia with Multilineage Dysplasia (RCMD) Refractory Cytopenia with Ring Sideroblasts and Multilineage Dysplasia (RCMD-RS), 5q- Syndrome without excess blasts
Randomized Multicentric phase III study
Title of the study:
MULTICENTRIC STUDY COMPARISON BETWEEN ERYTHROPOIETIN AND ERYTHROPOIETIN ASSOCIATED TO DIFFERENTIATING THERAPY WITH ACID 13-CIS-RETINOIC AND DIHYDROXYVITAMIN D3 IN MYELODYSPLASTIC SYNDROMES WITHOUT EXCESS OF BLASTS
Description of the study:
In low – intermediate risk myelodysplasia, which are the majority of cases, cytopenia complications (particularly anemia) are the main clinical problem.
Over the years, has been increased the interest in recombinant haematopoietic factors such as erythropoietin, whis has been widely used and, as a single drug, has obtained important, though temporary, improvement of anemia in approximately 25% of patients, with beneficial effects on symptomatology, transfusion needs and the quality of life of responsive subjects.To improve the results obtained, it have been evaluated biological agents so-called “differentiating” for their ability to induce cells differentiation.In vitro studies have demonstrated the possibility of synergic action between differentiating agents of different molecular structure, especially when associated with low doses of cytostatic agents.
The primary objective of the study is to compare after 4 months of therapy the erythroid response obtained with conventional therapy with rEPO alone at doses of 40,000 units / week with that obtained with the combination therapy between the same doses of rEPO and differentiating therapy with 13-cis-retinoic acid + Dihydroxycholate vitamin D3.
This is an interventional multicenter randomized phase III study; the Sponsor was AISSM (Italian Association for the Study of Myelodysplastic Syndromes) then evolved in FISM
There are 2 arms of randomization:
- rEPO sc 40.000 unit/week for 4 consecutive months
- rEPO sc 40.000 unit/week for 4 consecutive months in association with acid 13-cis-retinoic 20 mg/die + vitamine D3 Dihydroxylated 1 µg os/die.
The last patient was enrolled in October 2011. Currently there is still a patient in therapy.