Clinical Trials. gov identifier:
NCT01956799
Study code:
FISM-BIOFER12
N° of patients expected by the study:
100
Study status:
Closed enrollment
Age:
> 18 years old
Myelodysplasia object of the study:
Myelodysplastic syndromes (MDS), chronic myeloproliferative diseases (PMF, CMML) and aplastic anemia (AA)
Multicentric phase II study
Title of the study:
IDENTIFICATION OF MECHANISM IN THE ERYTHROID RESPONSE IN PATIENTS WITH MYELODYSPLASIA UNDERGOING CHELATION THERAPY
Description of the study:
Myelodysplastic syndromes are a group of hematologic neoplasms characterized essentially by ineffective erythropoiesis and by peripheral cytopenia.
Macrocytic anemia is the major haematochemical alteration occurring in these patients who, especially in the case of lower risk, during the clinical course head to high transfusion needs which often results in a secondary hemostasis.
The iron excess introduced due to periodic blood transfusions can not be eliminated spontaneously from the body as there is no physiological iron elimination system in humans.
For this reason iron deposition is determined in particular at liver and cardiac tissue level with the development of secondary organ failure.
In addition, when transferrin, the protein responsible for transporting circulating iron, is saturate (> 60-70%), it promotes the formation of free radicals that damage cellular membranes, proteins and nucleic acids within the cell.
The prevention of organ dysfunction (especially heart arrhythmias, congestive heart failure, chronic hepatic disease, reduced glycidal tolerance) due to martial overload often makes necessary to introduce in the therapeutic plan of myelodysplastic patients with a relatively good life expectancy a chelating drug to eliminate the excess iron.
The aim of this study is to evaluate the molecular mechanism underlying the erythroid response (increase of hemoglobin values and reduction or elimination of transfusion needs) observed in some patients with myelodysplasia, myelofibrosis and aplastic anemia undergoing iron chelating therapy with Deferasirox or Deferoxamina.
This is a multicenter phase II study, the Sponsor is FISM
The study is expected to enroll up to 100 patients whose biological samples must be collected pre and post chelation therapy.