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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
Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome May 2024 Journal of Pediatric Hematology/Oncology Aplastic Anemia
Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial Jan 2024 Lancet Hematology Myelodysplastic Syndromes (MDS)
Safety and activity of selinexor in patients with myelodysplastic syndromes or oligoblastic acute myeloid leukaemia refractory to hypomethylating agents: a single-centre, single-arm, phase 2 trial Aug 2020 Lancet Hematology Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
Safety and efficacy of danicopan in patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis Oct 2024 Expert Review of Hematology Paroxysmal Nocturnal Hemoglobinuria (PNH)
Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study Apr 2024 Advances in Therapy Paroxysmal Nocturnal Hemoglobinuria (PNH)
Secondary Myelodysplastic Syndrome and Leukemia in Acquired Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria Jul 2020 Blood Aplastic Anemia, Myelodysplastic Syndromes (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Secondary Myelodysplastic Syndrome and Leukemia in Acquired Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria May 2020 Blood Aplastic Anemia, Myelodysplastic Syndromes (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Severe breakthrough hemolysis during compassionate use of Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: managing an emergency Jul 2024 Blood Transfusion Paroxysmal Nocturnal Hemoglobinuria (PNH)
SF3B1 Mutations Negatively Predict for Response to Immunosuppressive Therapy in Myelodysplastic Syndromes Jun 2020 Clinical Lymphoma , Myeloma & Leukemia Myelodysplastic Syndromes (MDS)
SF3B1-mutant myelodysplastic syndrome as a distinct disease subtype - A Proposal of the International Working Group for the Prognosis of Myelodysplastic Syndromes (IWG-PM) Apr 2020 Blood Myelodysplastic Syndromes (MDS)