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| http://dx.doi.org/10.1038/s41375-022-01702-1 | DOI Listing |
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Beta 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.
Can Cyprus Afford Luspatercept? A Budget Impact Analysis of the Reimbursement of Luspatercept for the Management of Thalassaemia in Cyprus.
Pharmacoecon Open
May 2024
Department of Health Sciences, Unit of Global Health, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
Objective: This study aims to estimate the budget impact of luspatercept reimbursement as an adjuvant to the standard management of β-thalassaemia major in Cyprus, from a societal perspective, and assess the financial feasibility of its inclusion in the β-thalassaemia armamentarium.
Methods: A 5-year horizon budget impact model was developed to determine the budget impact of reimbursing luspatercept for the management of β-thalassaemia major in Cyprus. Two treatment discontinuation scenarios were elaborated.
Luspatercept mitigates bone loss driven by myelodysplastic neoplasms and estrogen-deficiency in mice.
Leukemia
November 2022
German Cancer Consortium (DKTK), partner site Dresden and German Cancer Research Center (DKFZ), Heidelberg, Germany.
β-Thalassemia: evolving treatment options beyond transfusion and iron chelation.
Hematology Am Soc Hematol Educ Program
December 2021
Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.
After years of reliance on transfusion alone to address anemia and suppress ineffective erythropoiesis in β-thalassemia, many new therapies are now in development. Luspatercept, a transforming growth factor-β inhibitor, has demonstrated efficacy in reducing ineffective erythropoiesis, improving anemia, and possibly reducing iron loading. However, many patients do not respond to luspatercept, so additional therapeutics are needed.
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