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Long-term combination therapy with metformin and oxymetholone in a Fanconi anemia mouse model.
Pediatr Blood Cancer
August 2024
Department of Pediatrics, Papé Family Pediatric Research Institute, Stem Cell Center, Pediatric Blood & Cancer Biology Program, Oregon Health & Science University, Portland, Oregon, USA.
Fanconi anemia (FA) is a disease caused by defective deoxyribonucleic acid (DNA) repair that manifests as bone marrow failure, cancer predisposition, and developmental defects. We previously reported that monotherapy with either metformin (MET) or oxymetholone (OXM) improved peripheral blood (PB) counts and the number and functionality of bone marrow hematopoietic stem progenitor cells (HSPCs) number in Fancd2 mice. To evaluate whether the combination treatment of these drugs has a synergistic effect to prevent bone marrow failure in FA, we treated cohorts of Fancd2 mice and wildtype controls with either MET alone, OXM alone, MET+OXM, or placebo diet from age 3 weeks to 18 months.
View Article and Find Full Text PDFLong-term combination therapy with Metformin and Oxymetholone in a Fanconi Anemia mouse model.
bioRxiv
August 2023
Department of Pediatrics, Papé Family Pediatric Research Institute, Pediatric Blood & Cancer Biology Program, Stem Cell Center; Oregon Health & Science University, Portland, OR.
Fanconi Anemia (FA) is a disease caused by defective DNA repair which manifests as bone marrow failure, cancer predisposition, and developmental defects. Mice containing inactivating mutations in one or more genes in the FA pathway partially mimic the human disease. We previously reported that monotherapy with either metformin (MET) or oxymetholone (OXM) improved peripheral blood (PB) counts and the number and functionality of bone marrow (BM) hematopoietic stem progenitor cells (HSPCs) number in mice.
View Article and Find Full Text PDFReal-World Costs and Cost-Effectiveness Analysis of Rabbit-Antithymocyte Globulin Versus Oxymetholone in Acquired Aplastic Anemia in Thailand.
Value Health Reg Issues
September 2023
Department of Social and Administrative Pharmacy, Faculty of Pharmaceutical Sciences, Prince of Songkla University, Songkhla, Thailand. Electronic address:
Objectives: This study aimed to compare rabbit-antithymocyte globulin and cyclosporine (rATG/CsA) with oxymetholone in terms of direct medical expenditures and economic evaluation in severe acquired aplastic anemia (SAA) and very severe acquired aplastic anemia (vSAA) patients.
Methods: Patients with SAA/vSAA who initiated treatment with rATG/CsA or oxymetholone between 2004 and 2018 were included. Trial-based cost-effectiveness evaluation in healthcare provider perspective was performed.
Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis.
J Blood Med
December 2022
Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand.
Background: Bone marrow transplantation, antithymocyte globulin/cyclosporine and eltrombopag are recommended as first-line therapy of severe aplastic anemia (SAA). However, androgens could be considered as front-line treatment among any patients ineligible for better methods although unsatisfactory efficacy is presented.
Objective: This retrospective study aimed to evaluate response and survival rate of practical-based treatment with oxymetholone.
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