View clinical trials related to Preleukemia.
Filter by:The aim of our study is to assess clinical & laboratory parameters of adult Egyptian myelodysplastic syndrome patients in upper Egypt, its correlation with disease-free survival, overall survival (OS) and acute leukemia transformation.
The primary goal of this study is to use qualitative interviews to elicit and confirm concepts related to treatment preferences and understandability of the pTPMQ, cTPMQ, and mTPMQ. The information gathered will be used to support the appropriateness of the questionnaires as a patient-reported, caregiver-reported and clinician-reported outcome measure (PROM) in the population of interest.
Myelodysplastic syndromes (MDS) are a group of malignancies characterized by reduced differentiation and increased apoptosis of hematopoietic progenitor cells, leading to ineffective hematopoiesis. Treatment of MDS varies according to prognosis. Patients with low IPSS-R risk have a low probability of progression to acute myeloid leukemia (AML) and the treatment is aimed at controlling cytopenia and improving quality of life (QOL). Anemia is the most common disease feature, occurring in 80%-85% of low-risk patients, 40% of whom eventually become RBC transfusion-dependent (TD). Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta (TGF-beta) superfamily. Luspatercept binds to GDF11, GDF8, activin B, and other ligands. This binding leads to inhibition of Smad2/3 signaling, which is abnormally high in disease models of ineffective erythropoiesis such as MDS, resulting in erythroid maturation and differentiation. Luspatercept is now approved for the treatment of adult patients with TD anemia due to very low-, low-, and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. FISiM (Fondazione Italiana Sindromi Mielodidplastiche) promotes a multicenter, retrospective observational study to collect information on the efficacy and safety of luspatercept in a real world Italian population of adult patients with transfusion-dependent anemia due to very low- and intermediate-risk MDS with ring sideroblasts
The purpose of this study is to assess the safety and effectiveness in the real-world setting among participants who are treated with Azacitidine in accordance with the China Product Label.
The main objective is to assess the safety, tolerability, and efficacy of AMG 176 as monotherapy and in combination with the 7-day regimen of azacitidine for the treatment of Higher-Risk Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia (HR-MDS/CMML).
The myelodysplastic syndromes (MDS) are a group of myeloid neoplasms characterized by abnormal differentiation and maturation of myeloid cells, reduced bone marrow (BM) function, and a genetic instability with enhanced risk to transform to secondary acute myeloid leukemia, AML
The study will evaluate the safety, tolerability and pharmacokinetics of NEX-18a, a long-acting injectable azacitidine, in patients diagnosed with intermediate 2 or higher-risk MDS, CMML, or AML and already on treatment with azacitidine.
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL. A survey will be used to measure the knowledge and comprehension of the REBLOZYL aRMMs among European Economic Area (EEA) based healthcare professionals (HCPs). The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available, potentially including Austria, Germany, Italy, Norway, Sweden, the Netherlands, Poland, and Spain. Additional EEA countries may be included, as needed, based on commercial availability and reimbursement status.
An open-label study available to all eligible participants from Study B1371019 and participants originating from Study B1371012 continuing on study intervention with azacitidine with or without glasdegib.
The purpose of the study is to find out if the new drug sabatolimab when given in combination with azacitidine and venetoclax, is safe and has beneficial effects in participants with high or very high risk myelodysplastic syndrome (MDS) who are not suitable for treatment with intensive chemotherapy or a stem-cell transplant (HSCT).