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Research Articles

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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
Treatment of eculizumab refractory paroxysmal nocturnal hemoglobinuria: A systematic review about current treatment options and future direction Jun 2023 SAGE Open Medicine Paroxysmal Nocturnal Hemoglobinuria (PNH)
Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. Sep 2020 PloS One Paroxysmal Nocturnal Hemoglobinuria (PNH)
Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors Jul 2024 PLOS One Paroxysmal Nocturnal Hemoglobinuria (PNH)
A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune-suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the Nort Aug 2020 Pediatric Blood & Cancer Aplastic Anemia, Pediatric
Diagnosis and treatment of pediatric myelodysplastic syndromes: A survey of the North American Pediatric Aplastic Anemia Consortium Aug 2020 Pediatric Blood & Cancer Aplastic Anemia, Myelodysplastic Syndromes (MDS), Pediatric
Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations May 2024 Pediatric Blood & Cancer Aplastic Anemia
Eltrombopag in children with severe aplastic anemia Apr 2021 Pediatric Blood & Cancer Aplastic Anemia, Pediatric
Experience of life quality from patients with aplastic anemia: a descriptive qualitative study Dec 2023 Orphanet Journal of Rare Diseases Aplastic Anemia
Patterns of Diagnostic Evaluation and Determinants of Treatment in Older Patients With Non-transfusion Dependent Myelodysplastic Syndromes Oct 2023 Oncologist Myelodysplastic Syndromes (MDS)
Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes Aug 2020 Nature Medicine Myelodysplastic Syndromes (MDS)