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Olutasidenib in combination with azacitidine induces durable complete remissions in patients with relapsed or refractory mIDH1 acute myeloid leukemia: a multicohort open-label phase 1/2 trial.
J Hematol Oncol
January 2025
Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL, USA.
Background: Olutasidenib is a potent, selective, oral, small molecule inhibitor of mutant IDH1 (mIDH1) which induced durable remissions in high-risk, relapsed/refractory (R/R) mIDH1 AML patients in a phase 1/2 trial. We present a pooled analysis from multiple cohorts of the phase 1/2 trial of patients with R/R AML who received combination olutasidenib and azacitidine therapy.
Methods: Adult patients with mIDH1 AML received 150 mg olutasidenib twice daily plus standard-of-care azacitidine (OLU + AZA) and were evaluated for response and safety.
CRISPR/Cas9 System as a Promising Therapy in Thalassemia and Sickle Cell Disease: A Systematic Review of Clinical Trials.
Mol Biotechnol
January 2025
Department of Pharmaceutical Chemistry, Faculty of Pharmacy, University of Tabuk, Tabuk, Saudi Arabia.
Article Synopsis
- The CRISPR-Cas9 system is a groundbreaking gene editing tool being researched for treating thalassemia and sickle cell disease (SCD).
- A study reviewed clinical trials from multiple databases, identifying 6 eligible studies involving 115 patients, which used CRISPR/Cas9 to target specific gene enhancers and promoters.
- Results showed that patients experienced increased fetal hemoglobin, improved hemoglobin levels, transfusion independence in thalassemia, and reduced pain episodes in SCD, indicating the potential of CRISPR/Cas9 as a one-time functional cure for these blood disorders.
Existence and significance of anti-HLA-C autoantibodies to primary and persistent platelet transfusion refractoriness in patients with hematologic disorders: a retrospective study from a single centre.
Ann Med
December 2025
Department of Hematology, The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang Provincial Hospital of Chinese Medicine), Hangzhou, China.
Objectives: Platelet transfusion refractoriness (PTR) is a frustrating clinical problem, and primary and persistent (P/P) PTR who experienced persistent PTR since the first transfusion was failed to be well recognized. This study aims to investigate the incidence and risk factors for P/P PTR.
Methods: Patients with hematologic disorders who underwent HLA high-resolution genotyping and donor-specific HLA antibody or panel reactive antibody (PRA) testing between January 2019 and March 2023 were reviewed.
Liberal or Restrictive Transfusion Strategy in Aneurysmal Subarachnoid Hemorrhage.
N Engl J Med
December 2024
From Ottawa Hospital Research Institute, Ottawa (S.W.E., D.A.F., A.T., I.W., T.R., R.M., D.D., S.C.M., L.M.); the Department of Medicine, Division of Critical Care, Faculty of Medicine, University of Ottawa, Ottawa (S.W.E., L.M.); School of Epidemiology and Public Health University of Ottawa, Ottawa (S.W.E., D.A.F., L.M.); Department of Medicine, Faculty of Medicine, University of Ottawa, Ottawa (D.A.F., D.D., S.C.M.); George Institute for Global Health, Sydney (A.D., F.B., N.H., C.R.A., P.T.); Malcolm Fisher Department of Intensive Care Medicine, Royal North Shore Hospital, St. Leonards, NSW, Australia (A.D., N.H., C.R.A., E.F.); the Faculty of Medicine and Health, University of Sydney Northern Clinical School, St. Leonards, NSW, Australia (A.D., C.R.A.); Australian and New Zealand Intensive Care Research Centre, Monash University, Melbourne, VIC, Australia (A.D., A.U.); the Department of Medicine, Centre Hospitalier de l'Université de Montréal, Montreal (M. Chassé); the Department of Medicine, Faculty of Medicine, Université de Montréal, Montreal (M. Chassé); the Department of Anesthesiology and Critical Care Medicine, Division of Critical Care Medicine, Faculty of Medicine, Université Laval, Quebec, QC, Canada (A.F.T., F.L.); Population Health and Optimal Health Practice Research Unit, Centre Hospitalier Universitaire de Québec-Université Laval Research Center, Quebec, QC, Canada (A.F.T., F.L.); the Department of Anesthesia, Critical Care Medicine Service, Hôpital de L'Enfant-Jésus, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec, QC, Canada (A.F.T., F.L.); the Department of Medicine, Faculty of Medicine, Université Laval, Quebec, QC, Canada (F.L.); the Department of Neurology and Neurosurgery, Division of Neurocritical Care, Emory University School of Medicine, Emory University Hospital and Grady Memorial Hospital, Atlanta (O.S.); the Department of Medicine, Division of Critical Care Medicine, Faculty of Medicine, Vancouver General Hospital, University of British Columbia, Vancouver, Canada (D.E.G.); the Division of Neurosurgery, Vancouver General Hospital, Vancouver, BC, Canada (G.R.); the Division of Neurosurgery, Department of Surgery, the University of British Columbia, Vancouver, Canada (G.R.); Neurocritical Care and Anesthesia, Sunnybrook Health Sciences Centre and Sunnybrook Research Institute, Toronto (M. Chapman); McGill University, Montreal (M.H.); the Departments of Critical Care Medicine and Clinical Neurosciences, Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada (A.K.); Nepean Clinical School, University of Sydney, Sydney (I.S.); the Department of Clinical Medicine, Macquarie University, Sydney (I.S.); the Critical Care and Trauma Division, the George Institute for Global Health, Sydney (I.S.); the Department of Intensive Care and Hyperbaric Medicine, the Alfred, Melbourne, VIC, Australia (A.U.); the Department of Critical Care Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Canada (D.J.K.); the Department of Internal Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, Canada (R.Z.); the Department of Medical Oncology/Hematology and the Paul Albrechtsen Research Institute, Cancer Care Manitoba, Winnipeg, Canada (R.Z.); the Department of Anesthesiology, Faculty of Medicine and Health Sciences, Université de Sherbrooke, Sherbrooke, QC, Canada (F.D.); Centre de Recherche du Centre Hospitalier Universitaire de Sherbrooke, Sherbrooke, QC, Canada (F.D.); the Department of Medicine, Division of Neurology, School of Medicine, Queen's University, Kingston, ON, Canada (J.G.B.); Department of Critical Care Medicine, School of Medicine, Queen's University, Kingston, ON, Canada (J.G.B.); the Intensive Care Unit, Prince of Wales Hospital, Randwick, NSW, Australia (G.S.); the Department of Intensive Care, Royal Brisbane and Women's Hospital, Herston, QLD, Australia (J.B.); University of Queensland, Brisbane, Australia (J.B.); the Department of Adult Intensive Care, Island Health Authority, Victoria, BC, Canada (G.W.); University of Colorado School of Medicine, Aurora (L.C.); the Department of Surgery, Division of Neurosurgery, Dalhousie University, Halifax, NS, Canada (G.P.); QEII Health Sciences Centre, Halifax, NS, Canada (G.P.); Lewis Katz School of Medicine, Temple University, Philadelphia (L.K.); Rush University Medical Center, Chicago (L.K.); Royal North Shore Hospital, Sydney (F.B.); the Department of Critical Care Medicine, Sunnybrook Health Sciences Centre, Toronto (D.C.S.); the Interdepartmental Division of Critical Care Medicine, University of Toronto, Toronto (D.C.S.); the Kirby Institute, University of New South Wales, Kensington, Australia (C.R.A.); the Department of Surgery, Division of Neurosurgery, Faculty of Medicine, University of Ottawa, Ottawa (J.S.); Canadian Blood Services, Edmonton, AB, Canada (J.A.); the Department of Laboratory Medicine and Pathology, University of Alberta, Edmonton, Canada (J.A.); and Physical Medicine and Rehabilitation, Bruyere Continuing Care, Ottawa (S.C.M.).
Background: The effect of a liberal red-cell transfusion strategy as compared with a restrictive strategy in patients during the critical care period after an aneurysmal subarachnoid hemorrhage is unclear.
Methods: We randomly assigned critically ill adults with acute aneurysmal subarachnoid hemorrhage and anemia to a liberal strategy (mandatory transfusion at a hemoglobin level of ≤10 g per deciliter) or a restrictive strategy (optional transfusion at a hemoglobin level of ≤8 g per deciliter). The primary outcome was an unfavorable neurologic outcome, defined as a score of 4 or higher on the modified Rankin scale (range, 0 to 6, with higher scores indicating greater disability) at 12 months.
The role of daratumumab in complications post-allogeneic hematopoietic stem cell transplantation: a single-center prospective study on PRCA and AIHA.
Bone Marrow Transplant
November 2024
Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Università Cattolica del Sacro Cuore, Rome, Italy.
Pure red cell aplasia (PRCA) and autoimmune hemolytic anemia (AIHA) post-hematopoietic stem cell transplantation (HSCT) are an unmet medical need with no established standard of care, significantly affecting the patient quality of life and posing a challenge for clinicians. The anti-CD38 IgG-kappa Daratumumab appears to be a safe and efficace treatment compared to prior drugs. Our study is a prospective monocentric investigation assessing the use of daratumumab in these complications following allo-HSCT.
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