Active clinical trials for "Preleukemia"

Recent retrospective studies have suggested that iron overload is a clinically important problem in patients undergoing ablative stem cell transplantation. However, these studies relied on serum ferritin as a surrogate of iron overload, which limits the conclusions that can be drawn from such analyses. Therefore, the investigators are conducting a prospective study to more rigorously examine the prevalence, mechanisms, and consequences of iron overload in this patient population.

This study performs HLA matched stem cell transplantation from unrelated donors in adults who require stem cell transplantation but do not have a matched related donor available. The incidence of graft-versus-host disease in unrelated stem cell transplantation is recorded. This study also monitors the activity and toxicity of total body irradiation and cyclosphosphamide followed by stem cell transplantation from matched unrelated donors.

The investigators' primary objective is to study prevalences of myocardial iron overload, defined as a cardiac T2*< 20 ms, in 3 populations of multiply transfused patients, affected with thalassemia, sickle cell disease, and myelodysplasia.

The study aims to evaluate the molecular mechanism underlying the erythroid response observed in some patients with myelodysplasia, myelofibrosis and aplastic anemia treated with Deferasirox or Deferoxamina.

The purposes of this study are: 1. To identify and quantify the health utilities and quality of life experienced by patients who have been diagnosed with MDS and what are their predictors. 2. Measure the effects of patient related factors like frailty and comorbidity on quality of life and overall survival or toxicity to therapy. 3. Assess how quality of life changes over time and what are its predictors. This will be valuable information that may guide therapy, transfusion practices, etc., as MDS is a chronic, incurable disease that is often progressive.

Myelodysplastic Syndromes (MDS) are characterized by quantitative and qualitative bone marrow failure and by a disorder of the medullary production which is a pre-leukemic state which can evolve into acute myeloid leukemia. The risk of leukemic transformation is estimated by the score IPSS (International Prognostic Score System). We distinguish the MDS of low risk (IPSS<1) and those of high risk of leukemic transformation (IPSS=1,5). Besides the risk of leukemic transformation, MDS much be complicated of infections which could be life-threatening. The risk of developing first infection after the diagnosis of MDS of high risk is probably influenced by anamnestic (disease duration, comorbidities), clinical (veinous central catheter, previous hospitalization), biological (neutropenia, lymphopenia, serum ferritin) and therapeutics (demethylating agent, lenalidomide, erythropoietin, G-CSF, transfusions, anti-infectious preventive treatment) factors. Their identification will allow for improved targeting of the population which is is likely to benefit from anti-infective prophylaxis Primary objective is to identify risk factors associated with first acute episode of infection in patients with MDS, by comparing index cases and matched control cases who did not develop infection episode since diagnosis. Secondary objectives are to explore nature and severity of infectious episodes, number of recurrences during 1 year of follow up and survival at 6 and 12 months

To assess the efficacy of decitabine and identify predictors for response to decitabine therapy.

RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer. PURPOSE: This research study is looking at blood samples in young patients with cytopenia after undergoing a donor stem cell transplant.

An observational, non-interventional, prospective and multicenter study of Azacitidine in newly diagnosed High Risk Myelodysplastic Syndromes. Primary objectives are to asses mutational status of target genes by Next Generation Sequencing, to evaluate prognostic value of geriatric assessment scales and to evaluate overall survival. The main hypothesis is that mutation status of target genes and geriatric scales have statistical significant impact on overall survival. Study time points will be at diagnosis, 6, 12, 18 and 24 months, always taking into account the routine clinical practice, when sample to assess mutational status will be collected. Geriatric assessment will only be performed at diagnosis. Upon the signature of informed consent and the checking of inclusion criteria, patients will receive treatment with Azacitidine 75 mg/sqm on a 28 days based cycles (both 7-0-0 and 5-0-2 regimens are allowed) until disease progression, unacceptable toxicity or investigator decision. 150 patients are expected to be recruited at study sites.

Myelodysplastic syndromes (MDS) are malignant hematopathies of the elderly characterized by persistent cytopenias and the presence of deregulated clonal hematopoiesis. The risk of progression to acute myeloid leukemia (AML) is variable. Acquired cytogenetic abnormalities are found in less than 50% of de novo cases and up to 80% in secondary MDS. The deletion of the long arm of chromosome 5 (written del(5q)) is the most common abnormality in MDS (15%). Del(5q) MDS has a good prognosis, with a median survival of 6 years and a 15% risk of progression to AML. However, their life expectancy is shorter than the general population, and the quality of life of patients is diminished. These treatments are not that effective over a long period of time or not well tolerated, and the majority of patients die from causes related to their MDS, such as infections (38%), progression to AML (15%), or bleeding (13%). Two genes, RBM22 and SLU7, coding for proteins of the same complex involved in splicing pre-messenger RNA are carried on the long arm of chromosome 5. We investigate the pronostic impact and the predictive value of the double haploinsufficiency of the RBM22 and SLU7 genes in del(5q) myelodysplastic syndromes isolated or not compared to the single haploinsufficiency of RBM22 and normal karyotype myelodysplastic syndromes.