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Myelodysplastic Syndrome

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1. Decitabine (Demylocan) - myelodysplastic syndromes (MDS)

Decitabine (Demylocan) - myelodysplastic syndromes (MDS) Search Page - Drug and Health Product Register Language selection Search and menus Search Search website Search Topics menu You are here: Summary Basis of Decision - - Health Canada Expand all Summary Basis of Decision (SBD) for Contact: Summary Basis of Decision (SBD) documents provide information related to the original authorization of a product. The for is located below. Recent Activity for SBDs written for approved after September 1

2019 Health Canada - Drug and Health Product Register

2. Azacitidine Celgene - myelodysplastic syndrome, chronic myelomonocytic leukaemia, acute myeloid leukaemia

Azacitidine Celgene - myelodysplastic syndrome, chronic myelomonocytic leukaemia, acute myeloid leukaemia Official address Domenico Scarlattilaan 6 ? 1083 HS Amsterdam ? The Netherlands An agency of the European Union Address for visits and deliveries Refer to www.ema.europa.eu/how-to-find-us Send us a question Go to www.ema.europa.eu/contact Telephone +31 (0)88 781 6000 © European Medicines Agency, 2019. Reproduction is authorised provided the source is acknowledged. EMA/384352/2019 EMEA/H/C (...) /005300 Azacitidine Celgene (azacitidine) An overview of Azacitidine Celgene and why it is authorised in the EU What is Azacitidine Celgene and what is it used for? Azacitidine Celgene is used for the treatment of adults with the following diseases, if they cannot have haematopoietic stem cell transplantation (when the patient receives stem cells to restore the bone marrow’s ability to produce healthy blood cells): • myelodysplastic syndromes, a group of conditions where too few blood cells

2019 European Medicines Agency - EPARs

4. Demylocan for Myelodysplastic Syndromes – Details

Demylocan for Myelodysplastic Syndromes – Details Demylocan for Myelodysplastic Syndromes – Details | CADTH.ca Find the information you need Demylocan for Myelodysplastic Syndromes – Details Demylocan for Myelodysplastic Syndromes – Details Brand Name Demylocan Generic Name Decitabine Tumour Type Other Indication Myelodysplastic Syndromes Review Status Not Filed Manufacturer Pharmascience Inc. Submission Type Non-Submission Clarification CADTH is unable to recommend reimbursement

2019 CADTH - Pan Canadian Oncology Drug Review

5. Thrombopoietin mimetics for patients with myelodysplastic syndromes. (Full text)

Thrombopoietin mimetics for patients with myelodysplastic syndromes. Myelodysplastic syndrome (MDS) is one of the most frequent haematologic malignancies of the elderly population and characterised by progenitor cell dysplasia with ineffective haematopoiesis and a high rate of transformation to acute myeloid leukaemia (AML). Thrombocytopenia represents a common problem for patients with MDS. ranging from mild to serious bleeding events and death. To manage thrombocytopenia, the current standard

2017 Cochrane PubMed

6. Myelodysplastic syndrome

Myelodysplastic syndrome Myelodysplastic syndrome - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Myelodysplastic syndrome Last reviewed: February 2019 Last updated: November 2017 Summary Heterogeneous group of clonal stem cell disorders, characterised by ineffective haematopoiesis. Primarily a disease of older adults. Can present with symptoms of anaemia, leukopenia, and thrombocytopenia, but often found during (...) a prolonged course with anaemia and neutropenic infections, or may progress rapidly to acute myelogenous leukaemia (AML). However, most patients die of infection while in the myelodysplastic syndrome (MDS) stage of their disease. Definition Myelodysplastic syndrome (MDS) is a group of clonal stem cell disorders, characterised by ineffective and dysplastic haematopoiesis resulting in 1 or more cytopenias, and a varying predilection to develop acute myeloid leukaemia (AML). Arber DA, Orazi A, Hasserjian R

2017 BMJ Best Practice

7. Granulocyte and granulocyte-macrophage colony stimulating factors for newly diagnosed patients with myelodysplastic syndromes. (Full text)

Granulocyte and granulocyte-macrophage colony stimulating factors for newly diagnosed patients with myelodysplastic syndromes. Myelodysplastic syndromes (MDS) are a heterogeneous group of haematological diseases which are characterised by a uni- or multilineage dysplasia of haematological stem cells. Standard treatment is supportive care of the arising symptoms including red blood cell transfusions or the administration of erythropoiesis-stimulating agents (ESAs) in the case of anaemia

2016 Cochrane PubMed

8. Darbepoetin alfa (Aranesp) for treatment of anaemia in adults with low or intermediate-1-risk myelodysplastic syndromes

Darbepoetin alfa (Aranesp) for treatment of anaemia in adults with low or intermediate-1-risk myelodysplastic syndromes Darbepoetin alfa (Aranesp) for treatment of anaemia in adults with low or intermediate-1-risk myelodysplastic syndromes | Innovation Observatory toggle menu Menu Search View All Filter by Speciality Filter by Year Filter by Category This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed (...) technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders. > > > Darbepoetin alfa (Aranesp) for treatment of anaemia in adults with low or intermediate-1-risk myelodysplastic syndromes Darbepoetin alfa (Aranesp) for treatment of anaemia in adults with low or intermediate-1-risk myelodysplastic syndromes August 2017 Myelodysplastic syndromes (MDS) are a group of blood disorders, in which the bone marrow does not produce

2017 NIHR Innovation Observatory

9. Epoetin alfa (Eprex) for anaemia in adults with low or intermediate-1 risk myelodysplastic syndromes

Epoetin alfa (Eprex) for anaemia in adults with low or intermediate-1 risk myelodysplastic syndromes Epoetin alfa (Eprex) for anaemia in adults with low or intermediate-1 risk myelodysplastic syndromes Epoetin alfa (Eprex) for anaemia in adults with low or intermediate-1 risk myelodysplastic syndromes NIHR HSRIC Record Status This is a bibliographic record of a published health technology assessment. No evaluation of the quality of this assessment has been made for the HTA database. Citation (...) NIHR HSRIC. Epoetin alfa (Eprex) for anaemia in adults with low or intermediate-1 risk myelodysplastic syndromes. Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2016 Authors' conclusions Myelodysplastic syndromes are a group of rare diseases which affect the bone marrow – the part of the body where blood cells are made. In myelodysplastic syndromes, the bone marrow does not make enough healthy blood cells. This causes symptoms such as feeling weak, tired

2016 Health Technology Assessment (HTA) Database.

10. Hypomethylating Properties of Freeze-dried Black Raspberries (BRB) in Patients With Myelodysplastic Syndrome or Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN)

Hypomethylating Properties of Freeze-dried Black Raspberries (BRB) in Patients With Myelodysplastic Syndrome or Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN) Hypomethylating Properties of Freeze-dried Black Raspberries (BRB) in Patients With Myelodysplastic Syndrome or Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN) - Full Text View - ClinicalTrials.gov Hide glossary Glossary Study record managers: refer to the if submitting registration or results information (...) . Search for terms x × Study Record Detail Saved Studies Save this study Warning You have reached the maximum number of saved studies (100). Please remove one or more studies before adding more. Hypomethylating Properties of Freeze-dried Black Raspberries (BRB) in Patients With Myelodysplastic Syndrome or Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN) The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does

2017 Clinical Trials

11. Impact of the number of mutations in survival and response outcomes to hypomethylating agents in patients with myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms (Full text)

Impact of the number of mutations in survival and response outcomes to hypomethylating agents in patients with myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms The prognostic and predictive value of sequencing analysis in myelodysplastic syndromes (MDS) has not been fully integrated into clinical practice. We performed whole exome sequencing (WES) of bone marrow samples from 83 patients with MDS and 31 with MDS/MPN identifying 218 driver mutations in 31 genes in 98 (86

2018 Oncotarget PubMed

12. Mitochondrial tRNA mutations in patients with myelodysplastic syndromes.

Mitochondrial tRNA mutations in patients with myelodysplastic syndromes. Increasing evidence showed that mitochondria play an important role in the development of myelodysplastic syndromes (MDS). Mitochondrial dysfunctions caused by mitochondrial DNA mutations, especially mitochondrial tRNA mutations, were found to be associated with MDS in many studies. However, the link between a candidate mitochondrial tRNA mutation and MDS was not clear. In this study, we investigated the role of some

2018 Mitochondrial DNA. Part A, DNA mapping, sequencing, and analysis

13. Severely impaired terminal erythroid differentiation as an independent prognostic marker in myelodysplastic syndromes (Full text)

Severely impaired terminal erythroid differentiation as an independent prognostic marker in myelodysplastic syndromes Anemia is the defining feature in most patients with myelodysplastic syndromes (MDS), yet defects in erythropoiesis have not been well characterized. We examined freshly obtained bone marrow (BM) samples for stage-specific abnormalities during terminal erythroid differentiation (TED) from 221 samples (MDS, n = 205 from 113 unique patients; normal, n = 16) by measuring

2018 Blood advances PubMed

14. Molecular pathophysiology of the myelodysplastic syndromes: insights for targeted therapy (Full text)

Molecular pathophysiology of the myelodysplastic syndromes: insights for targeted therapy The clinical heterogeneity of the myelodysplastic syndromes (MDSs) relates to the recently discerned panoply of molecular abnormalities extant within this disease spectrum. Despite increasing recognition of these biologic abnormalities, very limited therapeutic options exist to exploit our increasing understanding of the molecular pathophysiology of MDS, with only 1 therapy (lenalidomide) particularly

2018 Blood advances PubMed

15. Hematopoietic cell transplant for acute myeloid leukemia and myelodysplastic syndrome: conditioning regimen intensity (Full text)

Hematopoietic cell transplant for acute myeloid leukemia and myelodysplastic syndrome: conditioning regimen intensity In this study, we sought to identify specific individual high-intensity or reduced-intensity conditioning regimens with the best relapse-free survival (RFS) rather than the global high- vs reduced-intensity regimen comparison. Patients (median age, 58 years) with acute myeloid leukemia (AML; n = 1258), who were in first or subsequent remission, or with MDS (n = 951) who had (...) refractory anemia with unilineage or multilineage dysplasia, 5q- syndrome, or refractory anemia with excess blasts received nonirradiation-containing regimens and were transplanted between 2009 and 2014 in the United States. Three-year RFS with high-intensity busulfan/cyclophosphamide (Bu4/Cy; 44%) was comparable to conditioning with high-intensity fludarabine/busulfan (Flu/Bu4; 44%), reduced-intensity fludarabine/melphalan (Flu/Mel; 52%; P = .53), and Flu/Mel + anti-thymocyte globulin (ATG; 44%; P = .38

2018 Blood advances PubMed

16. Conditioning intensity in secondary AML with prior myelodysplastic syndrome/myeloproliferative disorders: an EBMT ALWP study (Full text)

Conditioning intensity in secondary AML with prior myelodysplastic syndrome/myeloproliferative disorders: an EBMT ALWP study Patients with secondary AML (sAML) with antecedent myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPNs) tend to have high-risk disease based on the older age of patients, high-risk cytogenetics, and higher number of prior treatments. Allogeneic hematopoietic cell transplant (HCT) is the only potentially curative therapy available. Eight hundred and two

2018 Blood advances PubMed

17. Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes (Full text)

Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes Prognosis of lower-risk (International Prognostic Scoring System [IPSS] low/intermediate-1) myelodysplastic syndrome (MDS) is heterogeneous and relies on steady-state assessment of cytopenias. We analyzed relative drops in neutrophil and platelet counts during the first 6 months of follow-up of lower-risk MDS patients. We performed a landmark analysis of overall survival (OS) of lower-risk MDS patients

2018 Blood advances PubMed

18. Evaluation of induction chemotherapies after hypomethylating agent failure in myelodysplastic syndromes and acute myeloid leukemia (Full text)

Evaluation of induction chemotherapies after hypomethylating agent failure in myelodysplastic syndromes and acute myeloid leukemia Hypomethylating agent (HMA) failure in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) carries a poor prognosis with limited treatment options. Although intensive, remission induction chemotherapy is often used subsequently, in particular to bridge to allogeneic transplantation, it is not clear whether an advantage exists for any particular regimen

2018 Blood advances PubMed

19. Deferasirox for managing iron overload in people with myelodysplastic syndrome. (Full text)

Deferasirox for managing iron overload in people with myelodysplastic syndrome. The myelodysplastic syndrome (MDS) comprises a diverse group of haematopoietic stem cell disorders. Due to symptomatic anaemia, most people with MDS require supportive therapy including repeated red blood cell (RBC) transfusions. In combination with increased iron absorption, this contributes to the accumulation of iron resulting in secondary iron overload and the risk of organ dysfunction and reduced life (...) expectancy. Since the human body has no natural means of removing excess iron, iron chelation therapy, i.e. the pharmacological treatment of iron overload, is usually recommended. However, it is unclear whether or not the newer oral chelator deferasirox leads to relevant benefit.To evaluate the effectiveness and safety of oral deferasirox for managing iron overload in people with myelodysplastic syndrome (MDS).We searched the following databases up to 03 April 2014: MEDLINE, EMBASE, The Cochrane Library

2014 Cochrane PubMed

20. Mutation Clearance after Transplantation for Myelodysplastic Syndrome. (Full text)

Mutation Clearance after Transplantation for Myelodysplastic Syndrome. Allogeneic hematopoietic stem-cell transplantation is the only curative treatment for patients with myelodysplastic syndrome (MDS). The molecular predictors of disease progression after transplantation are unclear.We sequenced bone marrow and skin samples from 90 adults with MDS who underwent allogeneic hematopoietic stem-cell transplantation after a myeloablative or reduced-intensity conditioning regimen. We detected

2018 NEJM PubMed

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