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| http://dx.doi.org/10.1177/20406207231195594 | DOI Listing |
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Luspatercept: a treatment for ineffective erythropoiesis in thalassemia.
Hematology Am Soc Hematol Educ Program
December 2024
Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
Patients with β-thalassemia continue to have several unmet needs. In non-transfusion-dependent patients, untreated ineffective erythropoiesis and anemia have been associated with a variety of clinical sequelae, with no treatment currently available beyond supportive transfusions. In transfusion-dependent forms, lifelong transfusion and iron chelation therapy are associated with considerable clinical, psychological, and economic burden on the patient and health care system.
View Article and Find Full Text PDFReal-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study.
Leuk Res
November 2024
Carolina Blood and Cancer Care Associates, Rock Hill, SC, USA.
Myelodysplastic syndromes (MDS) are associated with anemia and the need for blood transfusions. In clinical trials, luspatercept reduced transfusion dependency among patients with lower-risk MDS. This United States (US) study describes real-world clinical outcomes pre- and post-luspatercept initiation among patients with MDS.
View Article and Find Full Text PDFBeta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing.
J Clin Med
November 2024
Karma Association for Diseased Children and Adolescents, Furn El Chebbak, Beirut VG9G+3GV, Lebanon.
Article Synopsis
- Beta thalassemia rates may be decreasing, but it still poses a major global health challenge, particularly in countries with limited resources.
- With proper management of transfusion therapy and iron chelation, patients, especially children in high-resource settings, can lead healthy lives into adulthood.
- New treatments, including gene therapy and medications like luspatercept, are emerging but access remains limited in many regions, affecting the overall care of patients with beta thalassemia.
A phase 2 clinical trial of luspatercept in non-transfusion-dependent patients with myelodysplastic syndromes.
Int J Hematol
November 2024
Division of Hematology/Oncology, Department of Internal Medicine, Kameda Medical Center, Kamogawa, Japan.
Luspatercept has shown durable clinical efficacy for the treatment of anemia in transfusion-dependent patients with lower-risk myelodysplastic syndromes (LR-MDS). We report the results of a prespecified primary analysis of a phase 2 trial of luspatercept in non-transfusion-dependent (NTD) Japanese patients with anemia due to LR-MDS. Luspatercept (starting dose 1.
View Article and Find Full Text PDFExperience with luspatercept therapy in patients with transfusion-dependent low-risk myelodysplastic syndromes in real-world clinical practice: exploring the positive effect of combination with erythropoietin alfa.
Front Oncol
October 2024
Center of Oncocytogenomics, Institute of Clinical Biochemistry and Laboratory Diagnostics, General University Hospital and First Faculty of Medicine, Charles University, Prague, Czechia.
Background: Luspatercept, an inhibitor of the transforming growth factor beta (TGF-β) pathway, is a novel treatment for anemic patients with lower-risk myelodysplastic syndromes (MDS) with transfusion dependence (TD) who do not respond to erythropoiesis-stimulating agents (ESA) therapy or are not suitable candidates for this treatment. We present real-world experience with luspatercept therapy from two hematology centers in the Czech Republic.
Methods: By January 2024, 54 MDS patients (33 men, 21 women) with a median age of 74 years (range, 55-95) were treated with luspatercept ± ESA at two Charles University hematology centers in Prague and Hradec Králové.
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