Activin Receptor II Ligand Traps and Their Therapeutic Potential in Myelodysplastic Syndromes with Ring Sideroblasts

The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia

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Alterations within the Osteo-Hematopoietic Niche in MDS and their Therapeutic Implications

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SF3B1-mutated myelodysplastic syndrome with ring sideroblasts harbors more severe iron overload and corresponding over-erythropoiesis

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Refractory anemia with ring sideroblasts and RARS with thrombocytosis

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Targeting of the bone marrow microenvironment improves outcome in a murine model of myelodysplastic syndrome

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TGF-  signaling in the control of hematopoietic stem cells

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SF3B1 mutation identifies a distinct subset of myelodysplastic syndrome with ring sideroblasts

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Aberrant splicing of genes involved in haemoglobin synthesis and impaired terminal erythroid maturation inSF3B1mutated refractory anaemia with ring sideroblasts

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Cooperative Effect of Erythropoietin and TGF-β Inhibition on Erythroid Development in Human Pluripotent Stem Cells

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Deregulation of Genes Related to Iron and Mitochondrial Metabolism in Refractory Anemia with Ring Sideroblasts

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Sotatercept (ACE-011) for the treatment of chemotherapy-induced anemia in patients with metastatic breast cancer or advanced or metastatic solid tumors treated with platinum-based chemotherapeutic regimens: results from two phase 2 studies

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SF3B1 mutant MDS-initiating cells may arise from the haematopoietic stem cell compartment

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RAP-011, an activin receptor ligand trap, increases hemoglobin concentration in hepcidin transgenic mice

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Myelodysplastic syndromes: 2014 update on diagnosis, risk-stratification, and management

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Live and let die at TEMRA

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An activin receptor IIA ligand trap promotes erythropoiesis resulting in a rapid induction of red blood cells and haemoglobin

(2014) Soraya Carrancio et al.   BRITISH JOURNAL OF HAEMATOLOGY

Sotatercept in patients with osteolytic lesions of multiple myeloma

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Refractory anemia with ring sideroblasts and marked thrombocytosis cases harbor mutations in SF3B1 or other spliceosome genes accompanied by JAK2V617F and ASXL1 mutations

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Disruption of SF3B1 results in deregulated expression and splicing of key genes and pathways in myelodysplastic syndrome hematopoietic stem and progenitor cells

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An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia

(2014) Michael Dussiot et al.   NATURE MEDICINE

Transforming growth factor-β superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis

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Erythropoiesis stimulating agents and other growth factors in low-risk MDS

(2013) Eva Hellström-Lindberg et al.   BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY

Allogeneic stem cell transplantation in MDS: How? When?

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Refractory anemia with ring sideroblasts

(2013) Luca Malcovati et al.   BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY

miR-21 mediates hematopoietic suppression in MDS by activating TGF-  signaling

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How we treat lower-risk myelodysplastic syndromes

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Clinical and biological implications of driver mutations in myelodysplastic syndromes

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Inappropriately low hepcidin levels in patients with myelodysplastic syndrome carrying a somatic mutation of SF3B1

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Age, JAK2V617F and SF3B1 mutations are the main predicting factors for survival in refractory anaemia with ring sideroblasts and marked thrombocytosis

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Prognostic irrelevance of ring sideroblast percentage in World Health Organization-defined myelodysplastic syndromes without excess blasts

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SF3B1 haploinsufficiency leads to formation of ring sideroblasts in myelodysplastic syndromes

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Stromal cell-mediated inhibition of erythropoiesis can be attenuated by Sotatercept (ACE-011), an activin receptor type II ligand trap

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Validation of a Prognostic Model and the Impact of Mutations in Patients With Lower-Risk Myelodysplastic Syndromes

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The transporter ABCB7 is a mediator of the phenotype of acquired refractory anemia with ring sideroblasts

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SF3B1 mutations are prevalent in myelodysplastic syndromes with ring sideroblasts but do not hold independent prognostic value

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Clinical significance of SF3B1 mutations in myelodysplastic syndromes and myelodysplastic/myeloproliferative neoplasms

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From stem cell to red cell: regulation of erythropoiesis at multiple levels by multiple proteins, RNAs, and chromatin modifications

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Outcome of High-Risk Myelodysplastic Syndrome After Azacitidine Treatment Failure

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Recurrent DNMT3A mutations in patients with myelodysplastic syndromes

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Frequent pathway mutations of splicing machinery in myelodysplasia

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SomaticSF3B1Mutation in Myelodysplasia with Ring Sideroblasts

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A soluble activin receptor Type IIA fusion protein (ACE-011) increases bone mass via a dual anabolic-antiresorptive effect in Cynomolgus monkeys

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Reduced SMAD7 Leads to Overactivation of TGF-  Signaling in MDS that Can Be Reversed by a Specific Inhibitor of TGF-  Receptor I Kinase

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Bone progenitor dysfunction induces myelodysplasia and secondary leukaemia

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Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996)

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Treatment with a soluble receptor for activin improves bone mass and structure in the axial and appendicular skeleton of female cynomolgus macaques (Macaca fascicularis)

(2009) Roberto José Fajardo et al.   BONE

Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study

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Erythropoiesis-stimulating agents in the treatment of anemia in myelodysplastic syndromes: a meta-analysis

(2008) Victor Moyo et al.   ANNALS OF HEMATOLOGY

Inhibition of the TGF-  receptor I kinase promotes hematopoiesis in MDS

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Red blood cell transfusions and iron overload in the treatment of patients with myelodysplastic syndromes

(2008) Elias Jabbour et al.   CANCER

Single-Dose, Randomized, Double-Blind, Placebo-Controlled Study of ACE-011 (ActRIIA-IgG1) in Postmenopausal Women*

(2008) Jon Ruckle et al.   JOURNAL OF BONE AND MINERAL RESEARCH

The Role of the Iron Transporter ABCB7 in Refractory Anemia with Ring Sideroblasts

(2008) Jacqueline Boultwood et al.   PLoS One

A soluble activin Type IIA receptor induces bone formation and improves skeletal integrity

(2008) R. S. Pearsall et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA