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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
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
Eltrombopag in children with severe aplastic anemia Apr 2021 Pediatric Blood & Cancer Aplastic Anemia, Pediatric
Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations Aug 2024 Pediatric Blood Cancer Aplastic Anemia
Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations Aug 2024 Pediatric Blood Cancer Aplastic Anemia, Pediatric
Family reflections: George Elliott: a first-person commentary on pediatric aplastic anemia research Aug 2024 Pediatric Research Aplastic Anemia
Family reflections: pediatric aplastic anemia research; a parent perspective Oct 2024 Pediatric Research Aplastic Anemia, Pediatric
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)
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)
Iron Overload Alters the Energy Metabolism in Patients With Myelodysplastic Syndromes: Results From the Multicenter FISM BIOFER Study Jun 2020 Scientific Reports Myelodysplastic Syndromes (MDS)