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Research Articles

Compiled for your convenience are articles on bone marrow failure research and treatment from the world’s major journals on hematology / oncology.

Article Title Original Publication Date Journal Bone Marrow Disease
Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria Jul 2023 Clinical and Experimental Medicine Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novel therapeutic choices in immune aplastic anemia Sep 2020 F1000Research Aplastic Anemia
Observation and treatment in DDX41-mutated acute myeloid leukemia and myelodysplastic syndrome Apr 2023 Blood Cancer Myelodysplastic Syndromes (MDS)
One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study Oct 2020 Therapeutic Advances in Hematology Paroxysmal Nocturnal Hemoglobinuria (PNH)
One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab Dec 2020 European Journal of Haematology Paroxysmal Nocturnal Hemoglobinuria (PNH)
Oral decitabine-cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study Jan 2024 Lancet Hematology Myelodysplastic Syndromes (MDS)
Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria Mar 2024 The New England Journal of Medicine Paroxysmal Nocturnal Hemoglobinuria (PNH)
Oral therapy for myelodysplastic syndromes/neoplasms and acute myeloid leukemia: a revolution in progress Jul 2023 Expert Review of Anticancer Therapies Myelodysplastic Syndromes (MDS)
Outcome of lower-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) after failure of erythropoiesis- stimulating agents Dec 2020 Leukemia Research Myelodysplastic Syndromes (MDS)
Outcomes of pediatric patients who relapse after first HCT for acute leukemia or MDS Mar 2021 Bone Marrow Transplantation Myelodysplastic Syndromes (MDS)