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A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia. NCT02453477

A phase 3, open-label, randomized study to compare the efficacy and safety of luspatercept (ACE-536) versus epoetin alpha for the treatment of anemia due to IPSS-R very low, low or intermediate risk due to myelodysplastic syndrome (MDS) ESA in native subjects who require red blood cell transfusions. NCT03682536

Angelucci E et al. Safety and efficacy, including event-free survival, of deferasirox versus placebo in iron-overloaded patients with low- and int-1-risk myelodysplastic syndromes (MDS): Outcomes from the randomized, double-blind Telesto study. Proc ASH 2018;Abstract 234.

Cappellini MD et al. Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: A phase II, open-label, dose-finding study. Haematologica 2019;104(3):477-84. Abstract

Cappellini M et al. The BELIEVE trial: Results of a phase 3, randomized, double-blind, placebo-controlled study of luspatercept in adult beta-thalassemia patients who require regular red blood cell (RBC) transfusions. Proc ASH 2018;Abstract 163.

Cappellini M, Motta I. New therapeutic targets in transfusion-dependent and -independent thalassemia. Hematology Am Soc Hematol Educ Program 2017;2017(1):278-83. Abstract

Diaz AE, Mesa RA. Pacritinib and its use in the treatment of patients with myelofibrosis who have thrombocytopenia. Future Oncol 2018;14(9):797-807. Abstract

Dussiot M et al. Modulation of activin signaling by RAP-011 (ActRIIA-IgG1) improve anemia, increases hemoglobin levels and corrects ineffective erythropoiesis in β-thalassemia. Proc ASH 2012;Abstract 247.

Fenaux P et al. The MEDALIST trial: Results of a phase 3, randomized, double-blind, placebo-controlled study of luspatercept to treat anemia in patients with very low-, low-, or intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts (RS) who require red blood cell (RBC) transfusions. Proc ASH 2018;Abstract 1.

Harrison CN et al. Momelotinib versus best available therapy in patients with myelofibrosis previously treated with ruxolitinib (SIMPLIFY 2): A randomised, open-label, phase 3 trial. Lancet Haematol 2018;5(2):e73-81. Abstract

Harrison CN et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): A single-arm, open-label, non-randomised, phase 2, multicentre study. Lancet Haematol 2017;4(7):e317-24. Abstract

Komrokji R et al. Sotatercept with long-term extension for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: A phase 2, dose-ranging trial. Lancet Haematol 2018;5(2):e63-72. Abstract

Migdady Y et al. Clinical outcomes with ring sideroblasts and SF3B1 mutations in myelodysplastic syndromes: MDS Clinical Research Consortium analysis. Clin Lymphoma Myeloma Leuk 2018;18(8):528-32. Abstract

Park S et al. Outcome of lower-risk patients with myelodysplastic syndromes without 5q deletion after failure of erythropoiesis-stimulating agents. J Clin Oncol 2017;35(14):1591-7. Abstract

Shammo JM, Komrokji RS. Clinical consequences of iron overload in patients with myelodys­plastic syndromes: The case for iron chelation therapy. Expert Rev Hematol 2018;11(7):577-86. Abstract

Thompson AA et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med 2018;378(16):1479-93. Abstract

Verstovsek S et al. Long-term survival in patients treated with ruxolitinib for myelofibrosis: COMFORT-I and -II pooled analyses. J Hematol Oncol 2017;10(1):156. Abstract

Verstovsek S et al; COMFORT-I Investigators. Long-term treatment with ruxolitinib for patients with myelofibrosis: 5-year update from the randomized, double-blind, placebo-controlled, phase 3 COMFORT-I trial. J Hematol Oncol 2017;10(1):55. Abstract

Zeidan AM et al. Comparison of clinical outcomes and prognostic utility of risk stratification tools in patients with therapy-related vs de novo myelodysplastic syndromes: A report on behalf of the MDS Clinical Research Consortium. Leukemia 2017;31(6):1391-7. Abstract

Zeidan AM et al. Therapy-related myelodysplastic syndromes-specific risk stratification: Are we putting the cart before the horse? Leukemia 2017;31(11):2539-41. Abstract