• CAMBRIDGE, Mass., Nov. 20, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that clinical proof-of-concept has been achieved in the Phase 2a portion of a study of investigational pyruvate kinase (PK) activator AG-946 as a potential treatment for anemia in adults with lower-risk myelodysplastic syndromes (LR-MDS). (ktla.com)
  • Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis (MF). (globenewswire.com)
  • See also Pediatric Myelodysplastic Syndromes , Pathology of Acute Myeloid Leukemia With Myelodysplasia-Related Changes , Pathology of Therapy-Related Myeloid Neoplasms (t-MNs) , Pathology of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) , and Pathology of Acute Myeloid Leukemia Not Otherwise Specified (AML NOS) . (medscape.com)
  • Myelodysplastic syndromes (MDS) are highly heterogeneous myeloid neoplasms, and a large number of patients are difficult to diagnose and classify by blood and bone marrow examination. (techscience.com)
  • The Program for Drug Development in Leukemia offers patients the most advanced treatments available for acute leukemia, myeloproliferative neoplasms, myelodysplastic syndromes, and chronic myeloid leukemia through participation in phase 1 clinical trials. (mskcc.org)
  • This is a multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). (nih.gov)
  • Researchers from the University of Texas MD Anderson Cancer Center, Houston, have conducted a comprehensive analysis of a large cohort of patients with myeloid neoplasms, including myelodysplastic syndromes (MDS), harboring DDX41 germline and somatic variants. (rarediseaseadvisor.com)
  • A Phenogenetic Axis that Modulates Clinical Manifestation and Predicts Treatment Outcome in Primary Myeloid Neoplasms. (cdc.gov)
  • The results of four clinical trials of ezatiostat in MDS have been reported in peer-reviewed scientific journals. (ascopost.com)
  • The Parkville Cancer Clinical Trials Unit was established in 2016, from the cancer clinical trials units of Peter Mac, Royal Melbourne and the Women's . (petermac.org)
  • The Early Drug Development (EDD) / Phase One team specialises in complex and first in human clinical trials, run by dedicated investigators and research staff with early drug development experience and expertise. (petermac.org)
  • Search clinical trials for adult and pediatric cancers and blood disorders offered through Dana-Farber and our clinical partners. (dana-farber.org)
  • Abstracts for this category describe innovative clinical trials that have not reached their primary endpoint to provide opportunities for early engagement and collaboration amongst investigators, translational research, clinical and industry investigators, statisticians and regulators. (globenewswire.com)
  • With the average cost of research and development to bring one drug to market at $802 million and given that 1 in 1,000 new compounds that enter preclinical testing ever make it to human testing and only 1 in 5 agents that enter human trials receive FDA approval ( 4, 5 ), it is little wonder that pharmaceutical companies would hesitate to invest in pediatric cancer treatments. (aacrjournals.org)
  • In randomised clinical trials, a subcutaneous dose of 230 µg/m 2 /day (4.0 to 8.4 µg/kg/day) was used. (medicines.org.uk)
  • Moffitt participates in over 350 clinical trials. (moffitt.org)
  • Phase I and II clinical trials) for patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) based on the underlying mutational drivers of each disease. (moffitt.org)
  • He has published significantly on this topic including recently in highly regarded journals including Leukemia and Haematologica and these work form the foundation of the clinical trials that he is bringing to patients in order to improve their quality of life and survival. (moffitt.org)
  • There was an increased incidence of venous and arterial thrombotic events in patients with multiple myeloma who received lenalidomide in combination with dexamethasone compared with dexamethasone alone in clinical trials. (pdr.net)
  • This article reviews current anticancer small-molecule inhibitors targeting epigenetic modified enzymes and displays their performances in different stages of clinical trials. (frontiersin.org)
  • Researchers at Memorial Sloan Kettering continue to pursue these and other treatment approaches in our clinical trials. (mskcc.org)
  • Here you can find a continually updated listing of Memorial Sloan Kettering's current clinical trials for adults with leukemia. (mskcc.org)
  • This drug is currently in phase III clinical trials at multiple sites, including Mount Sinai, for the treatment of myelodysplastic syndrome (MDS). (oncologynurseadvisor.com)
  • Our patients have access to advanced treatments and diagnosis, including DNA mutation identification and ongoing clinical trials investigating new treatments. (dana-farber.org)
  • I hope that now the development would continue," Dr. Mittelman said, "(that) clinical trials will be activated and will eventually lead to available drugs in the market for the patients who really need it. (medscape.com)
  • Furthermore, survival outcomes by specific treatment type extend knowledge about the effectiveness of drug regimens among all patients, not just those eligible for clinical trials. (cdc.gov)
  • 5-azacytidine (AZA) has become standard treatment for patients with higher-risk myelodysplastic syndrome (MDS). (biomedcentral.com)
  • Seven patients (29%) had previously received intensive chemotherapy, and five had previous HDAC inhibitor treatment. (biomedcentral.com)
  • In recent years, epigenetic therapy has become a treatment option for patients with higher-risk myelodysplastic syndrome (MDS) who are not considered candidates for intensive induction chemotherapy or allogeneic stem cell transplantation (SCT). (biomedcentral.com)
  • 2001 ). Stimulated by these findings, we studied the clinical effect of VPA at serum concentrations of 50-100 μg/ml in 23 patients with AML or MDS as monotherapy or in combination with (ATRA) (Kuendgen et al. (biomedcentral.com)
  • In general, patients with myelodysplastic syndrome (MDS) typically present with features associated with their cytopenia(s), such as fatigue, easy bleeding or bruising, or infection. (medscape.com)
  • Patients with this type of MDS can often live a long time, even without treatment. (cancer.org)
  • Ezatiostat has been shown to cause clinically significant and sustained reduction in red blood cell transfusions, transfusion independence, and multilineage responses in patients with multidysplastic syndromes. (ascopost.com)
  • Four of the 10 patients with low transfusion burden (LTB) achieved the transfusion independence endpoint, and one of the 22 patients treated in the study achieved the hemoglobin response endpoint in the 16-week treatment (core) period. (ktla.com)
  • We are grateful to all of the patients who participated in this trial, our collaborators, study investigators and advisors in the patient and clinical communities for their partnership in achieving this milestone, and we look forward to advancing the clinical program to Phase 2b," said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. (ktla.com)
  • Agios is proud to be the leader in PK activation with two distinct PK activators - AG-946 and mitapivat - that have the potential to be meaningful treatment options for a wide range of blood disorders and positively impact many patients' lives. (ktla.com)
  • Treatment options for MDS patients are limited, especially for patients with relapsed or refractory disease, and are often associated with myelosuppressive side effects. (medicalnewstoday.com)
  • We look forward to expanding our experience with CPI-613 as a monotherapy in the treatment of relapsed / refractory MDS patients in the recently launched Phase II MDS clinical trial as well as the possibility of establishing CPI-613 as an alternative treatment option for this rare and devastating disease. (medicalnewstoday.com)
  • Survival of patients with SDS who develop myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) is poor 10 . (nature.com)
  • Therefore, a central goal in clinical care of SDS patients is to identify incipient leukemic transformation and initiate pre-emptive treatment with allogeneic stem cell transplantation. (nature.com)
  • Current surveillance strategies for patients with SDS and other leukemia predisposition syndromes rely on monitoring hematologic status by serial peripheral blood counts to identify worsening cytopenias and bone marrow examinations to identify morphologic changes or development of clonal chromosomal abnormalities 11 . (nature.com)
  • [ 7 ] The first report of a paraneoplastic syndrome has been attributed to a French physician, M. Auchè, who described peripheral nervous system involvement in cancer patients in 1890. (medscape.com)
  • We offer a wide range of advanced treatments for many of the Peter Mac and Royal Melbourne patients. (petermac.org)
  • Most patients with myelodysplastic syndrome have greater than one mutation and a unique pattern of mutation with marked heterogeneity. (standardofcare.com)
  • Somatic mutation is not a static process in myelodysplastic syndrome and additional mutations will accumulate leading to more profound phenotypic worsening cytopenias, and approximately 30% of patients will eventually experience progression to secondary AML. (standardofcare.com)
  • Patients with myelodysplastic syndromes (MDS) collectively have a high symptom burden and are also at risk of death from complications of cytopenias and acute myeloid leukemia. (standardofcare.com)
  • Validation of the revised international prognostic scoring system in treated patients with myelodysplastic syndromes. (aamds.org)
  • Notably, this unique cDC1 repertoire was subverted in a group of patients with myelodysplastic syndromes included in the study. (nih.gov)
  • He compared paired samples from AML patients at various stages of the disease and treatment and found that blood samples from AML patients at diagnosis contained the ancestral stem and progenitor cells that carried the initiating genetic events in AML. (weizmann.ac.il)
  • He recently initiated a clinical study to track large cohorts of AML patients by taking samples from diagnosis, after therapy induction, and every three months during remission until relapse. (weizmann.ac.il)
  • Since 1947, Dana-Farber's sole focus has been to provide expert cancer care and groundbreaking treatments for adult and pediatric patients. (dana-farber.org)
  • Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment. (globenewswire.com)
  • Salman Fazal, MD, shares opinions regarding the role of hypomethylating agents (HMAs) in treatment for patients with MDS. (ajmc.com)
  • In the initial clinical trial, hypomethylating agents were studied in patients with high-risk myelodysplastic syndrome, where it showed that it improved hematologic parameters, reduced risk of transformation to acute myeloid leukemia, and improved the overall survival. (ajmc.com)
  • We offer patients who have high-risk disease IV [intravenous] azacitidine, a 7-day treatment, and then there's IV decitabine, which is given over 5 days. (ajmc.com)
  • For high-risk myelodysplastic syndrome, we're able to offer the oral decitabine-cedazuridine combination for these patients. (ajmc.com)
  • Having treated patients with myelodysplastic syndrome for years, one of the frequently asked questions by my patients was: "Do we have any oral therapy? (ajmc.com)
  • and a Phase 2a combination trial of ALRN-6924 and palbociclib in patients with tumors harboring MDM2 amplifications co-amplifications, as well as for patients with p53-mutated small cell lung cancer that has completed Phase 1b clinical trial. (yahoo.com)
  • Over 100 free commonly used clinical formulas and calculator tools to help diagnose, treat, prevent, and determine prognosis of patients at point of care. (guidelinecentral.com)
  • Identify clinical preventive services that are appropriate for your patients. (guidelinecentral.com)
  • Our results indicated that CD10-positive granulocytes were significantly decreased in BM of MDS patients than AA and IDA patients, and the level of CD10-positive mature granulocytes was not associated with the clinical stages of malignancy. (techscience.com)
  • Neupogen is indicated for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in patients treated with established cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes) and for the reduction in the duration of neutropenia in patients undergoing myeloablative therapy followed by bone marrow transplantation considered to be at increased risk of prolonged severe neutropenia. (medicines.org.uk)
  • Neupogen is indicated for the treatment of persistent neutropenia (ANC less than or equal to 1.0 × 10 9 /l) in patients with advanced HIV infection, in order to reduce the risk of bacterial infections when other options to manage neutropenia are inappropriate. (medicines.org.uk)
  • Physicians and researchers work close together to provide innovative treatment options for patients. (moffitt.org)
  • Dr. Sallman's research focuses on the development of novel, targeted therapeutic strategies for patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). (moffitt.org)
  • Dr. Sallman studies the genetic drivers of myeloid diseases in order to improve prognostication for patients and also to allow for more personalized treatment. (moffitt.org)
  • As an example, Dr. Sallman has focused research and clinical trial efforts on patients who have TP53 mutation (often associated with complex genes/cytogenetics) given their high risk of transformation to acute leukemia and poor survival. (moffitt.org)
  • Therapy will continue as long as the cancer does not get worse and patients are tolerating treatment. (facingourrisk.org)
  • In patients with myelodysplastic syndromes (MDS), preexisting autoimmune disease is associated with a decreased risk for mortality, according to findings from a large population-based cohort analysis published in the journal Blood Advances. (rarediseaseadvisor.com)
  • A phase 3b, open-label study is set to assess the efficacy and safety of luspatercept administered at the maximum approved dose in low-risk patients with myelodysplastic syndromes (MDS) who require red blood cell transfusions. (rarediseaseadvisor.com)
  • In patients with low-risk myelodysplastic syndromes (LR-MDS), transfusion dependence is a common challenge and can be associated with complications and decreased overall survival (OS), according to findings from a discrete-event simulation-based disease model presented at the Society of Hematologic Oncology 11th Annual Meeting and published in the journal Clinical Lymphoma, Myeloma & Leukemia. (rarediseaseadvisor.com)
  • Researchers from France have identified a potential predictor of treatment outcomes in patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) receiving azacitidine therapy. (rarediseaseadvisor.com)
  • At the same time, we often include patients in suitable clinical studies, if they are available. (uhkt.cz)
  • Lymphadenopathy is present in 40-76% of patients with JMML but is present in less than 10% of patients with adult-type myelodysplastic syndrome (MDS). (medscape.com)
  • Myelodysplasia syndrome and acute myeloid leukemia in patients with congenital neutropenia receiving G-CSF therapy. (medscape.com)
  • Identification of new prognosis factors from the clinical and epidemiologic analysis of a registry of 229 Diamond-Blackfan anemia patients. (medscape.com)
  • Despite the fact that most follicular lymphomas are advanced at the time of diagnosis, many patients may not require treatment for prolonged periods of time, and the median survival of patients with follicular lymphomas has historically been approximately 8-10 years. (medscape.com)
  • Some patients have developed leukemia or myelodysplastic syndrome following treatment with GCSF. (lu.se)
  • Grade 3 or higher treatment-related adverse events and treatment-related serious adverse events occurred in 20.5% and 11.4% of patients in cohort A and in 29.3% and 10.0% of patients in cohort B, respectively. (bvsalud.org)
  • Despite the recent success of tyrosine kinase inhibitors (TKIs) in the treatment of chronic myeloid leukemia (CML), approximately 2-17% of patients develop clonal cytogenetic changes in the Philadelphia-negative (Ph(-)) cell population. (lu.se)
  • A fraction of these patients, in particular those displaying trisomy 8 or monosomy 7, are at risk of developing a myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). (lu.se)
  • Wide variation in use and interpretation of gene mutation profiling panels among health care providers of patients with myelodysplastic syndromes: results of a large web-based survey. (cdc.gov)
  • Genomic Biomarkers to Predict Resistance to Hypomethylating Agents in Patients With Myelodysplastic Syndromes Using Artificial Intelligence. (cdc.gov)
  • A genomics-informed computational biology platform prospectively predicts treatment responses in AML and MDS patients. (cdc.gov)
  • Based on findings of excess blast cells and high proportions of patients diagnosed with AML after starting romiplostim treatment in a phase 2, placebo-controlled trial, romiplostim's label includes a warning that "in some patients with MDS, (romiplostim) increases blast-cell counts and increases the risk of progression to AML. (medscape.com)
  • After the safety committee recommendation, 69 of 250 patients (28%) discontinued treatment early. (medscape.com)
  • Despite initial concerns about increased acute myeloid leukemia transformation in patients treated with romiplostim, long-term follow-up showed that treatment with romiplostim had no significant negative impact on acute myeloid leukemia transformation or survival," the researchers conclude. (medscape.com)
  • Physicians should take into account our findings of no increased AML risk with romiplostim, the warning language from the prescribing information, and the characteristics of individual patients when making treatment decisions for patients with lower-risk MDS and thrombocytopenia," Dr. Mac concluded. (medscape.com)
  • Registry has specific treatment information for patients in an unstructured free-text format. (cdc.gov)
  • CHALLENGE: Because specific treatment information for patients in the California Cancer Registry is contained in an unstructured free-text format and manual review is laborious, the information is infrequently used. (cdc.gov)
  • RESULTS: Manual review of 24,845 text field records associated with 17,310 patients diagnosed with stage IV NSCLC from 2012 to 2014 found specific treatment information for 78% of patients. (cdc.gov)
  • Findings indicated that there has been variable utilization of treatments and almost a third (31.7%) of patients did not receive systemic treatment. (cdc.gov)
  • HSCT) has significantly modified the prognosis of when the pathological process involves BM or when patients with hereditary or acquired hematological, hematopoietic toxicity is the limiting factor in the oncological and immunological diseases and it is the aggressive treatment of the disease. (bvsalud.org)
  • Also, drastic changes in daily living habits, qualified and responsible for the implementation of changes in body image, long duration of treatment, HSCT since 2004, agreed to the Brazilian Unified periods of hospitalization and protective isolation, Health System, which meets the needs of the feeling of loss of control, fear of death and lack of patients with an indication for transplantation. (bvsalud.org)
  • This edition reflects the numerous advances that are redefining the field and dramatically influencing new approaches to diagnosis, treatment, and outcomes. (elsevierhealth.com)
  • The U.S. Orphan Drug Act aims to encourage the development of drugs for the diagnosis, prevention and treatment of medical conditions affecting fewer than 200,000 people in the United States. (ascopost.com)
  • Diagnosis of leukemia predisposition provides potential opportunities for early intervention, but data to guide precision medicine approaches to clinical surveillance are lacking. (nature.com)
  • When a patient without a known cancer presents with one of the "typical" paraneoplastic syndromes, a diagnosis of cancer should be considered and investigated. (medscape.com)
  • This finding holds significant promise for treatment, earlier diagnosis, and new screening methods for AML and perhaps other cancers as well. (weizmann.ac.il)
  • Quick Medical Diagnosis & Treatment 2023 Papadakis MA, McPhee SJ. (mhmedical.com)
  • From diagnosis to treatment, our experts provide the care and support you need, when you need it. (mskcc.org)
  • A Geno-Clinical Decision Model for the Diagnosis of Myelodysplastic Syndromes. (cdc.gov)
  • Targeted Next-Generation Sequencing Is a Sensitive Tool for Differential Diagnosis of Myelodysplastic Syndromes in Bone Marrow Trephines. (cdc.gov)
  • The results from the Phase I single agent trial were presented at the 2013 American Society for Clinical Oncology (ASCO) Annual Meeting and hand-selected for inclusion in the Best of ASCO 2013 educational series. (medicalnewstoday.com)
  • Compiled for your convenience are articles on bone marrow failure research and treatment from the world's major journals on hematology / oncology. (aamds.org)
  • Aileron Therapeutics, Inc. operates as a clinical stage chemoprotection oncology company in the United States. (yahoo.com)
  • Neupogen therapy should only be given in collaboration with an oncology centre which has experience in G-CSF treatment and haematology and has the necessary diagnostic facilities. (medicines.org.uk)
  • Aptevo Therapeutics Inc (Aptevo) is a clinical-stage biotechnology company that develops and commercializes oncology and hematology therapeutics. (researchandmarkets.com)
  • In a study to be presented at the American Society of Clinical Oncology (ASCO) 2020 virtual meeting, a Roswell Park Comprehensive Cancer Center team reports that in many cases, these newer treatment approaches may reduce the risk of therapy-related myelodysplastic syndrome or acute myeloid leukemia (tMDS/AML) compared to chemotherapy-based treatment strategies. (newswise.com)
  • We hypothesized that increased immune surveillance after treatment with these drugs could halt clonal evolution and reduce risk of therapy-related hematologic cancers - specifically, myelodysplastic syndrome or acute myeloid leukemia," says Dr. Thota, Assistant Professor of Oncology in the Department of Medicine. (newswise.com)
  • Oncology Nurse Advisor offers clinical updates and evidence-based guidance to the oncology nurse community online and in print. (oncologynurseadvisor.com)
  • Several ongoing questions in the realm of myelodysplastic syndromes were discussed at the 11th Annual Meeting of the Society of Hematologic Oncology. (rarediseaseadvisor.com)
  • Myelodysplastic syndromes (MDS) are clonal hematological disorders arising from hematopoietic stem cells that have accumulated various genetic abnormalities. (go.jp)
  • Mutation-driver genes cause clonal outgrowth and propagation of myelodysplastic hematopoiesis. (standardofcare.com)
  • Myelodysplastic syndrome (MDS) is a clonal disorder characterized by dyshematopoiesis and high susceptibility to acute myeloid leukemia (AML). (karger.com)
  • Particular drivers of clonal hematopoiesis are associated with a higher chance of developing myelodysplastic syndromes (MDS) and may yield significant changes in blood cell parameters, a study published in Nature Genetics suggested. (rarediseaseadvisor.com)
  • Myelodysplastic syndrome and acute myelogenous leukemia as a late clonal complication in children with acquired aplastic anemia. (medscape.com)
  • Myelodysplastic syndromes are a group of clonal hematopoietic stem cell disorders unified by the presence of distinct mutations of hematopoietic stem cells, most frequently in genes involved in RNA splicing. (msdmanuals.com)
  • Consequently, there is a need to characterize the clinical features of such cases and to increase our understanding of the pathogenetic mechanisms underlying the emergence of clonal cytogenetic changes in Ph(-) cells. (lu.se)
  • Geron is a clinical stage biopharmaceutical company focused on the development and potential commercialization of a first-in-class telomerase inhibitor, imetelstat, in hematologic myeloid malignancies. (globenewswire.com)
  • Newswise - BUFFALO, N.Y. - While breakthrough treatments have emerged for several cancers over the last two decades, driving striking improvements in survival and other clinical outcomes, too little is known about the risk of therapy-related hematologic cancers following targeted and immunotherapeutic approaches. (newswise.com)
  • The study, Abstract 7516 , will be displayed on board 289 in the Hematologic Malignancies-Leukemia, Myelodysplastic Syndromes, and Allotransplant virtual poster session and discussed in the poster discussion session "Battle of the Clones: Establishing the Leukemia Roadmap" from 8 a.m. to 11 a.m. (newswise.com)
  • Clinical applicability of proposed algorithm for identifying individuals at risk for hereditary hematologic malignancies. (cdc.gov)
  • Do You Have Higher-Risk Myelodysplastic Syndrome? (webmd.com)
  • This article is for people who are living with higher-risk myelodysplastic syndrome (MDS) and their care partners or anyone who wants to learn about higher-risk MDS. (webmd.com)
  • Based on these findings, novel agents targeting driver mutations of MDS are currently under development and expected to improve the clinical outcome of MDS in the coming decades. (go.jp)
  • de Souza Fernandez T, Menezes de Souza J, Macedo Silva ML, Tabak D, Abdelhay E: Correlation of N-ras point mutations with specific chromosomal abnormalities in primary myelodysplastic syndrome. (karger.com)
  • Clinical Outcomes With Ring Sideroblasts and SF3B1 Mutations in Myelodysplastic Syndromes: MDS Clinical Research Consortium Analysis. (cdc.gov)
  • The causes of the paraneoplastic syndromes associated with underlying cancers are not well known. (medscape.com)
  • Structural experiments by the research team confirmed the mode of action for rigosertib and also demonstrated the potential for this targeted mechanism in the treatment of several RAS-driven cancers. (oncologynurseadvisor.com)
  • Trisomy 8/8q is a common cytogenetic event in myelocytic malignancies, ranging from myelodysplastic syndrome (MDS) to acute myelocytic leukemia (AML) to blastic transformation of chronic myelocytic leukemia. (nih.gov)
  • Cytogenetic studies of Brazilian pediatric myelodysplastic syndrome cases: challenges and difficulties in a large and emerging country. (medscape.com)
  • ABSTRACT This study examined haematopoietic stem cells of 19 high-risk cases of myelodysplastic syndrome (MDS) for apoptotic and anti-apoptotic signals and cellular proliferation and correlated these with clinical and cytogenetic subtypes, particularly trisomy 8. (who.int)
  • Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. (globenewswire.com)
  • Treatment usually also includes intrathecal chemotherapy since systemic chemotherapy can have limited penetration into the central nervous system and the central nervous system is a common site for relapse of acute lymphoblastic leukemia. (wikipedia.org)
  • The clinical features of 11 cases of myelocytic leukemia with pentasomy and hexasomy 8/8q were summarized. (nih.gov)
  • To understand the mechanisms that mediate germline genetic leukemia predisposition, we studied the inherited ribosomopathy Shwachman-Diamond syndrome (SDS), a bone marrow failure disorder with high risk of myeloid malignancies at an early age. (nature.com)
  • These results mechanistically link leukemia predisposition to germline genetic constraints on cellular fitness, and provide a rational framework for clinical surveillance strategies. (nature.com)
  • When acute myeloid leukemia is first diagnosed, it appears as an overwhelming disease in need of immediate and aggressive treatment. (weizmann.ac.il)
  • In 2012, he packed his bags for the University of Toronto, where he spent the next few years as both a postdoctoral research fellow and a clinical fellow with a focus on leukemia at Princess Margaret Cancer Centre. (weizmann.ac.il)
  • Au WY, Fung AT, Ma ES, Liang RH, Kwong YL: Low frequency of FLT3 gene internal tandem duplication and activating loop mutation in therapy-related acute myelocytic leukemia and myelodysplastic syndrome. (karger.com)
  • Aptevo has received Orphan Drug Designation (ODD) for its pipeline product APVO436 from the US FDA for the treatment of acute myelogenous leukemia (AML). (researchandmarkets.com)
  • EA9181, A Phase III Randomized Trial of Steroids+Tyrosine Kinase Inhibitor Induction With Chemotherapy or Blinatumomab for Newly Diagnosed BCR-ABL-Positive Acute Lymphoblastic Leukemia in Adults Rochester, Minn. This phase III trial compares the effect of usual treatment of chemotherapy and steroids and a tyrosine kinase inhibitor (TKI) to the same treatment plus blinatumomab. (mayo.edu)
  • The objective of this study is to assess the safety and tolerability, including the maximum tolerated dose, of ASP2215 in subjects with relapsed or treatment-refractory acute myeloid leukemia (AML). (mayo.edu)
  • Thus, our care covers congenital or acquired non-tumor diseases (anemias, coagulation disorders, etc.), as well as tumor diseases such as leukemia or myelodysplastic syndrome. (uhkt.cz)
  • Distinctive demography, biology, and outcome of acute myeloid leukemia and myelodysplastic syndrome in children with Down syndrome: Children's Cancer Group Studies 2861 and 2891. (medscape.com)
  • The drug is approved for the treatment of refractory B-cell chronic lymphocytic leukemia ( 2 ) and also has been used after stem cell ( 3 ) and organ transplantations ( 4 ). (cdc.gov)
  • A 65-year-old man with refractory chronic lymphocytic leukemia had been receiving treatment with alemtuzumab for 3 months. (cdc.gov)
  • NEW YORK (Reuters Health) - Contrary to initial reports, romiplostim treatment of thrombocytopenia does not appear to increase the risk of progression from myelodysplastic syndrome (MDS) to acute myeloid leukemia (AML). (medscape.com)
  • They are defined as clinical syndromes involving nonmetastatic systemic effects that accompany malignant disease. (medscape.com)
  • A 17-year-old woman with severe systemic lupus erythematosus and secondary myelodysplastic syndrome received an unrelated T-cell depleted bone marrow transplant. (cdc.gov)
  • SUMMARY: Surveillance of systemic treatment utilization at the population level can provide insight into dissemination of new or existing cancer treatments. (cdc.gov)
  • Using SAS- based text mining, the CalCARES Program developed an algorithm to extract specific systemic treatment information for stage IV non-small cell lung cancer (NSCLC) from the free-text treatment text fields. (cdc.gov)
  • Following induction and consolidation treatment for acute myeloid leukaemia the duration of treatment may be substantially longer (up to 38 days) depending on the type, dose and schedule of cytotoxic chemotherapy used. (medicines.org.uk)
  • Your treatment plan may also be based on the prognostic score (outlook for survival) of the disease. (healthline.com)
  • Compared with trisomy and tetrasomy 8, significant features included reduced median survival (90 days), treatment refractoriness (even with transplantation), monocytic differentiation, trilineage dysplasia, and radiation or toxin exposure. (nih.gov)
  • Treatment with recombinant human granulocyte colony-stimulating factor (GCSF) elevates the granulocyte counts, helps resolve preexisting infections, diminishes the number of new infections and results in significant improvements in survival and quality of life. (lu.se)
  • The Phase 3 IMerge clinical trial will be presented as a poster in this new category, and the details of the trial design are described in the abstract. (globenewswire.com)
  • As stated in the abstract, these findings suggest that an additive inhibitory activity against malignant myelofibrosis hematopoietic stem and progenitor cells can be achieved using a sequential treatment regimen of ruxolitinib followed by imetelstat. (globenewswire.com)
  • Well-illustrated and clinically focused, it details the basic science and clinical practice of hematology and hematopoietic cellular therapy-covering virtually all aspects of hematology in one definitive resource. (elsevierhealth.com)
  • Dr. Shlush continues to collaborate with his postdoctoral mentor, Dr. John E. Dick, and clinical mentor, Dr. Mark Minden, at Princess Margaret to study AML and other blood malignancies. (weizmann.ac.il)
  • Challenges in the introduction of next-generation sequencing (NGS) for diagnostics of myeloid malignancies into clinical routine use. (cdc.gov)
  • For patient education information, see the Cancer Center , as well as Lung Cancer , Lymphoma , and Carcinoid Syndrome . (medscape.com)
  • Multiagent chemotherapy in combination with rituximab is the most common first-line treatment strategy for follicular lymphoma. (medscape.com)
  • Ezatiostat is an investigational agent in development for the treatment of MDS and idiopathic chronic neutropenia. (ascopost.com)
  • [ 9 ] In cases in which the patient has received previous chemotherapy, MDS in which del(5q) is the sole abnormality should be classified as a therapy-associated myelodysplastic syndrome (t-MDS). (medscape.com)
  • After getting chemotherapy or other treatments to destroy the stem cells in your bone marrow, you'll receive an intravenous (IV) infusion of healthy stem cells from a donor. (webmd.com)
  • Following established chemotherapy for solid tumours, lymphomas, and lymphoid leukaemia, it is expected that the duration of treatment required to fulfil these criteria will be up to 14 days. (medicines.org.uk)
  • Therapy often involves several cycles of chemotherapy, targeted biological treatment, immunological, immunosuppressive and instrument treatment (TPE and depletion procedures performed in cooperation with the IHBT Apheresis Department ), as well as different forms of hematopoietic stem cell transplantation . (uhkt.cz)
  • Treatment options include transfusions, drug therapy, chemotherapy, and blood or bone marrow stem cell transplants. (medlineplus.gov)
  • Current standard treatment for all of these cancer types relies on drugs that stimulate the immune system - tyrosine kinase inhibitors (TKIs) and immune checkpoint inhibitors (ICIs), in the case of NSCLC, RCC and melanoma, and immunomodulatory imide drugs (IMiDs), in the case of multiple myeloma. (newswise.com)
  • Genetic Predisposition to Myelodysplastic Syndrome in Clinical Practice. (cdc.gov)
  • The pathophysiology of paraneoplastic syndromes is complex and intriguing. (medscape.com)
  • Luspatercept-aamt demonstrated superiority against epoetin alfa in the treatment of anemia in adults with ESA-naïve transfusion-dependent LR‑MDS. (rarediseaseadvisor.com)
  • We are pleased with the results of the Phase 2a study, which underscore the potential of AG-946 to be a first-in-class, oral, safe and effective option for the treatment of anemia in adults with LR-MDS by improving red blood cell health through its unique mechanism of action. (ktla.com)
  • A common clinical finding in benzene hematotoxicity is cytopenia, which is a decrease in various cellular elements of the circulating blood manifested as anemia, leukopenia, or thrombocytopenia in humans and in animals. (cdc.gov)
  • We show that germline SBDS deficiency establishes a fitness constraint that drives selection of somatic clones via two distinct mechanisms with different clinical consequences. (nature.com)
  • Since epigenetic treatment aims at reversing pathological gene silencing, and DNA methylation cooperates with histone modification to control gene expression, it appears logical to combine AZA with inhibitors of histone deacetylases. (biomedcentral.com)
  • Epigenetic drugs show significant effects on both preclinical and clinical studies in which the target development and research offer a promising direction for cancer therapy. (frontiersin.org)
  • In this review, we summarize the basic principles manipulating the abovementioned epigenetic pathways and highlight the evidence of the promising clinical and preclinical results using small-molecule inhibitors against chromatin regulators for cancer treatment. (frontiersin.org)
  • Evolution of myelodysplastic syndrome and acute myelogenous leukaemia in children with hepatitis-associated aplastic anaemia. (medscape.com)
  • The experiments explored the hypothesis that the combination of imetelstat and ruxolitinib might create a treatment regimen for MF that could be more efficacious than using either drug alone in reducing myelofibrosis hematopoietic stem cells and hematopoietic progenitor cells. (globenewswire.com)
  • If you have a myelodysplastic syndrome, the stem cells do not mature into healthy blood cells. (medlineplus.gov)
  • 2 Lab of Clinical and Experimental Immunology, Humanitas Clinical and Research Center, Rozzano, Italy. (nih.gov)
  • Cancer treatment is a significant challenge for the global health system, although various pharmacological and therapeutic discoveries have been made. (frontiersin.org)
  • Paraneoplastic syndromes are rare disorders that are triggered by an altered immune system response to a neoplasm. (medscape.com)
  • This translational research conference will bring together biologists and clinicians with an interest in Myelodysplastic Syndromes (MDS) in order to explore the most recent advances in the understanding of the pathogenesis and clinical behavior of the disease. (esh.org)
  • Next, we'll discuss each of the main treatment options for this group of diseases. (healthline.com)
  • Some therapies listed on our site are considered experimental for the treatment of bone marrow failure diseases. (aamds.org)
  • Behçet disease (BD) and BD-like clinical phenotypes: NF-kB pathway in mucosal ulcerating diseases. (cdc.gov)
  • Based on the favorable efficacy data and positive benefit-risk profile in the Phase 2a core period, Agios intends to advance its clinical program evaluating AG-946 in LR-MDS by initiating the placebo-controlled Phase 2b portion of the study in mid-2024. (ktla.com)
  • Telik, Inc, announced that its product candidate, ezatiostat hydrochloride (Telintra), has been granted orphan drug designation by the FDA for the treatment of myelodysplastic syndromes (MDS). (ascopost.com)
  • Cornerstone Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CPI-613, the Company's lead Altered Energy Metabolism Directed (AEMD) drug candidate, for the treatment of myelodysplastic syndrome (MDS). (medicalnewstoday.com)
  • In addition to MDS, Cornerstone Pharmaceuticals has previously received orphan drug designation for the use of CPI-613 in the treatments of AML and pancreatic carcinoma . (medicalnewstoday.com)
  • In a broad sense, these syndromes are collections of symptoms that result from substances produced by the tumor, and they occur remotely from the tumor itself. (medscape.com)
  • In other cases, paraneoplastic syndromes result from the production and release of physiologically active substances by the tumor. (medscape.com)
  • Applied to the characterization of DCs in the tumor microenvironment, this panel has the potential to provide valuable information to be used for investigating the role of DC subsets in cancer, guiding DC-targeting treatments, and possibly identifying predictive biomarkers for clinical response to cancer immunotherapy. (nih.gov)
  • Dana-Farber assumes no liability for inaccuracies that may result from using this third-party tool, which is for website translation and not clinical interactions. (dana-farber.org)
  • There is substantial discrepancy in the treatment of ITP among clinicians, even among high-income countries, who are likely to over-treat [4,5]. (who.int)
  • Some treatments try to slow MDS down, while others help manage symptoms. (webmd.com)
  • Myelodysplastic syndromes often do not cause early symptoms and are sometimes found during a routine blood test. (medlineplus.gov)