FibroGen to initiate Phase II/III trial of roxadustat to treat MDS related anaemia in China

US-based biopharmaceutical firm FibroGen has secured approval from the China Food and Drug Administration (CFDA) to initiate a Phase II/III clinical trial of roxadustat to treat anaemia associated with lower risk myelodysplastic syndromes (MDS).
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

Related Links:

Immune-mediated processes are considered important in the pathogenesis of bone marrow failure syndromes (BFS). We previously reported that natural killer group 2D (NKG2D) ligands were expressed on pathological blood cells of patients with BFS and that NKG2D immunity may be involved in bone marrow failure. In addition to membranous NKG2D ligands on the cell surface, soluble NKG2D ligands can exist in plasma. We therefore examined the relationship between soluble NKG2D ligands and blood cell counts in 86 patients with BFS, including aplastic anemia, myelodysplastic syndrome with single lineage dysplasia, and paroxysmal noctu...
Source: Acta Haematologica - Category: Hematology Source Type: research
Contributors : Sabrina Bondu ; Anne-Sophie Alary ; Carine Lef èvre ; Alexandre Houy ; Grace Jung ; Thibaud Lefebvre ; David Rombaut ; Ismael Boussaid ; Abderrahmane Bousta ; François Guillonneau ; Prunelle Perrier ; Samar Alsafadi ; Michel Wassef ; Raphaël Margueron ; Alice Rousseau ; Nathalie Droin ; Nicolas Cagnard ; Sophie Kaltenbach ; Sus ann Winter ; Anne-Sophie Kubasch ; Didier Bouscary ; Valeria Santini ; Andrea Toma ; Mathilde Hunault ; Aspasia Stamatoullas ; Emmanuel Gyan ; Thomas Cluzeau ; Uwe Platzbecker ; Lionel Adès ; Hervé Puy ; Marc-Henri Stern ; Zoubida Karim ; Patrick Mayeu...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Homo sapiens Source Type: research
AbstractMyelodysplastic syndromes are clonal hematopoietic stem cell disorders characterized by cytopenia and intramedullary apoptosis. BCL-2 Ovarian Killer (BOK) is a pro-apoptotic member of the BCL-2 family of proteins which, when stabilized from endoplasmic reticulum-associated degradation (ERAD), induces apoptosis in response to ER stress. Although ER stress appropriately activates the unfolded protein response (UPR) in BOK-disrupted cells, the downstream effector signaling that includes ATF4 is defective. We used Nup98-HoxD13 (NHD13) transgenic mice to evaluate the consequences of BOK loss on hematopoiesis and leukemo...
Source: Annals of Hematology - Category: Hematology Source Type: research
AbstractPurpose of ReviewAlleviating cytopenias, namely anemia, is the main goal of therapy in lower-risk myelodysplastic syndromes (MDS). Current available treatment options remain limited. We review the role of TGF-B pathway in MDS, the current available data on luspatercept and sotatercept development.Recent FindingsTGF-B pathway is overactivated in MDS contributing to observed myelosuppression. SMADs, the downstream proteins of TGF-B pathway, are upregulated. GDF-11 is a negative regulator of terminal erythroid differentiation and an activin receptor ligand. Sotatercept and luspatercept are fusion ligand trap novel age...
Source: Current Hematologic Malignancy Reports - Category: Hematology Source Type: research
Several prognostic scoring systems have been developed to assess prognosis in myelodysplastic syndrome (MDS). However, currently there are no systems that list gender as a prognostic factor. We queried a National Cancer Institute database to investigate the prognostic influence of gender on the survival of patients with MDS. We first identified 34,681 qualified patients diagnosed with MDS from 2001-2014 in the Surveillance, Epidemiology, and End Results (SEER) database, and then analyzed the characteristics of these patients using chi-squared tests. The Kaplan-Meier method and the multivariate Cox regression model were use...
Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
Abstract Eltrombopag, an oral thrombopoietin-receptor agonist, stimulates hematopoiesis in patients with acquired aplastic anemia (AA) and has higher exposure in patients of East Asian origin. We evaluated the pharmacokinetics, efficacy, and safety of eltrombopag in Japanese patients with AA refractory or intolerant to immunosuppressive therapy (IST). Twenty-one patients (15 with non-severe AA, six with severe AA) with platelet counts
Source: International Journal of Hematology - Category: Hematology Authors: Tags: Int J Hematol Source Type: research
Publication date: Available online 25 May 2019Source: Leukemia Research ReportsAuthor(s): Jihane Belkhair, Abderahim Raissi, Hicham Elyahyaoui, Mustapha Ait Ameur, Mohamed ChakourAbstractAtypical chronic myeloid leukemia (aCML), BCR-ABL1 negative is a rare myelodysplastic syndrome /myeloproliferative neoplasm for which no current standard of care exists. The blood smear of patients with aCML showed prominent immature granulocytosis, and granulocytic dysplasia. We admitted a 58-year-old man with splenomegaly, hyperleukocytosis, anemia, and thrombocytopenia; then cytology, cytogenetic and molecular biology analysis of bone m...
Source: Leukemia Research Reports - Category: Hematology Source Type: research
Allogeneic HLA-matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) for adults with acquired severe aplastic anemia (SAA) is currently recommended after failure to respond to a course of immunosuppressive therapy (IST) 1,2. This guidance reflects the historical developments in HSCT and IST, and the improving outcomes of MUD HSCT over time. Although SAA patients are surviving long term after both HSCT and IST, the long-term complications of clonal transformation to myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) and hemolytic PNH after IST in up to 15-26% of patients at 10 years are notable 3-7.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Allogeneic HLA-matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) for adults with acquired severe aplastic anemia (SAA) is currently recommended after failure to respond to a course of immunosuppressive therapy (IST) [1,2]. This guidance reflects the historical developments in HSCT and IST, as well as the improving outcomes of MUD HSCT over time. Although patients with SAA are surviving long term after both HSCT and IST, the long-term complications of clonal transformation to myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) and hemolytic paroxysmal nocturnal hemoglobinuria (PNH)...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
This study is registered with ClinicalTrials.gov, number NCT01261312.FindingsBetween July 9, 2012, and April 7, 2014, 105 patients were enrolled: 55 (52%) were allocated to guadecitabine 60 mg/m2 (28 patients were treatment-naive and 27 had relapsed or refractory disease after previous hypomethylating agent treatment) and 50 (48%) patients to 90 mg/m2 (23 patients were treatment-naive and 27 had relapsed or refractory disease). Three (3%) patients of 105 did not receive study treatment and were excluded from analyses. Median follow-up was 3·2 years (IQR 2·8–3·5). The proportion of patients achiev...
Source: The Lancet Haematology - Category: Hematology Source Type: research
More News: Anemia | Clinical Trials | Food and Drug Administration (FDA) | Myelodysplastic Syndrome | Pharmaceuticals