Comparison of outcomes of myeloablative allogeneic stem cell transplantation for pediatric patients with bone marrow failure, myelodysplastic syndrome and acute myeloid leukemia with and without germline GATA2 mutations

Pediatric myelodysplastic syndromes (MDS) represent a spectrum of rare hematopoietic stem cell disorders characterized by cytopenias, ineffective hematopoiesis, marrow dysplasia and risk for leukemic transformation. While some cases are likely acquired due to de novo transformation, germline mutations in DNA repair pathways, ribosomal pathways, the telomere complex or in hematopoietic transcription factors (e.g., CEBPA, RUNX1, GATA2 and ETV6) are predisposition syndromes underlying many pediatric cases of MDS and acute myeloid leukemia (AML).
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research

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Abstract Myelodysplastic syndrome is a heterogenous group of disorder with clonal dysregulated hematopoiesis characterized by bone marrow failure, cytogenetic and molecular abnormalities and variable risk of progression to acute myeloid leukemia (AML). The bone marrow niche plays a major role in maintaining the homeostasis and is often injured by the chemotherapeutic drugs leading to catastrophic consequences like myelodysplastic syndrome. In the present study, we made an attempt to find out the osteoblastic niche related alterations in the myelodysplastic bone marrow through mainly flowcytometric and fluorescent ...
Source: Molecular and Cellular Biochemistry - Category: Biochemistry Authors: Tags: Mol Cell Biochem Source Type: research
AbstractBackground Patients with haematological malignancies are at high risk of invasive fungal infections. However, there is a lack of information about the utilisation of the recommended Australian antifungal prophylaxis guidelines in haematology outpatients.Objective To assess the impact of a weekly pharmacist review of high-risk adult haematology outpatients on the utilisation of appropriate antifungal prophylaxis.Setting Outpatient cancer centre, tertiary referral hospital in Sydney, Australia.Method A 3-month pre-and post-interventional study was conducted. A retrospective audit was conducted to obtain baseline util...
Source: International Journal of Clinical Pharmacy - Category: Drugs & Pharmacology Source Type: research
Authors: Liu L, Liu X, Sun Y, Zeng Y, Tang X, Li X, Liu J, Xiao R Abstract Esophageal squamous cell carcinoma (ESCC) is one of the most common malignant tumors, and has high incidence and mortality rates, worldwide. Myelodysplastic syndrome (MDS), a disorder of hematopoietic stem or progenitor cells, results in marrow failure, which increases the risk of acute myeloid leukemia (AML). Few studies had reported patients who have suffered from both ESCC and MDS/AML simultaneously. To identify possible potential associations between ESCC and MDS/AML, the present case report describes a patient with both types of these t...
Source: Oncology Letters - Category: Cancer & Oncology Tags: Oncol Lett Source Type: research
Source: Annals of Hematology - Category: Hematology Source Type: research
Allogeneic hematopoietic cell transplantation (HCT) is the only curative therapy for most adults with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Since HCT using conventional myeloablative conditioning (MAC) can be associated with higher toxicity and mortality rates, reduced intensity conditioning (RIC) regimens have been increasingly used in the past two decades for HCT in older and less fit patients with AML or MDS.1,2 A recent prospective randomized Blood and Marrow Transplant Clinical Trials Network (BMT CTN)  0901 trial demonstrated significantly improved disease-free survival (DFS) benefit ...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
We present long-term safety data for niraparib from the ENGOT-OV16/NOVA trial. METHODS: This multicenter, double-blind, randomized, controlled phase III trial evaluated the efficacy and safety of niraparib for the treatment of recurrent ovarian cancer. Patients were randomly assigned 2:1 to receive either once-daily niraparib 300 mg or placebo. Two independent cohorts were enrolled based on germline BRCA mutation status. The primary endpoint was progression-free survival, reported previously. Long-term safety data were from the most recent data cutoff (September 2017). RESULTS: Overall, 367 patients received...
Source: Gynecologic Oncology - Category: Cancer & Oncology Authors: Tags: Gynecol Oncol Source Type: research
The myelodysplastic syndromes (MDS) are a heterogeneous collection of clonal hematopoietic malignancies characterized by poor overall survival owing to ineffective hematopoiesis, progressive cytopenias, and transformation to acute myelogenous leukemia [1]. Current therapeutics for MDS are based primarily on stratification into lower-risk and higher-risk disease using clinical prognostic scoring systems [2,3]. Allogeneic hematopoietic cell transplantation (HCT) is the sole potentially curative therapy for MDS.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: Commentary Source Type: research
Abstract Understanding the Mode of Action (MOA) for a chemical can help guide decisions in development of Occupational Exposure Limits (OELs). Where sufficient information exists, it can provide the OEL developer the basis for selecting either a health-based or risk-based approach. To support the development of an OEL for benzene, scientific information relevant to MOA assessment for risk-based and health-based OEL approaches was reviewed. Direct-acting mutagenicity was considered as a basis for a risk-based OEL, versus MOAs consistent with a health-based approach: indirect mutagenicity via topoisomerase II inhibi...
Source: Toxicology Letters - Category: Toxicology Authors: Tags: Toxicol Lett Source Type: research
(University of Texas M. D. Anderson Cancer Center) The University of Texas MD Anderson Cancer Center and Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Tokyo, Japan, today announce a strategic collaboration agreement aimed at accelerating the clinical evaluation of Astex's pipeline of products for patients with certain types of leukemia, including myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML). The collaboration will combine MD Anderson's clinical trials infrastructure and expertise with Astex's clinical pipeline products.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
esco Albano Immunomodulatory drugs (IMiDs) are analogs of thalidomide. They have immunomodulatory, antiangiogenic and proapoptotic properties and exert a role in regulating the tumor microenvironment. Recently IMiDs have been investigated for their pleiotropic properties and their therapeutic applications in both solid tumors (melanoma, prostate carcinoma and differentiated thyroid cancer) and hematological malignancies. Nowadays, they are applied in de novo and relapsed/refractory multiple myeloma, in myelodysplastic syndrome, in del5q syndrome with specific use of lenalidomide and B-cell lymphoma. Several studies hav...
Source: Cancers - Category: Cancer & Oncology Authors: Tags: Review Source Type: research
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