Hematology
Megias-Vericat JE, Escolar G, Wilson MR, Mendez P, McDade CL, Barrientos LV, Tomic R, Panebianco M, Linden S, Yan S. Cost-effectiveness and cost-utility analysis of haemate-P versus other von willebrand disease treatments in Spain. J Med Econ. 2025 Mar 7;28(1):436-45. doi: 10.1080/13696998.2025.2474886
BACKGROUND: Von Willebrand Disease (vWD) is the most common congenital bleeding disorder, with an estimated prevalence in Spain of 0.01%. The aim was to assess the cost-utility of Haemate-P compared with present alternatives in the treatment of vWD in Spain.
Cappellini MD, Glassberg MB, Meyers J, Jimenez M, Nham T, Bueno L, Sieluk J, Yucel A, Alashkar F. Demographics, clinical characteristics, and real-world treatment patterns among patients with beta-thalassemia: a retrospective medical record abstraction study. Ther Adv Hematol. 2024 Dec 15;15. doi: 0.1177/20406207241298088
BACKGROUND: Beta-thalassemias (BTs) are characterized by deficient or absent synthesis of the beta-globin subunit, leading to anemia. Patient characteristics and treatment patterns in these patients may vary.
Wilson MR, Castaman G, Thomas W, Millar CM, Escolar G, Miesbach WA, McDade CL, Tomic R, Yan S. Cost-effectiveness of voncento prophylaxis versus on-demand treatment in von willebrand disease in the United Kingdom. Blood Adv. 2025 Mar 25;9(6):1312-9. doi: 10.1182/bloodadvances.2024014376
Passamonti F, Korgaonkar S, Parikh RC, Chevli M, Yucel A, Rombi J, Jones S, Zissler D, Davis KL, Slaff S. Real-world treatment patterns and health outcomes for patients with myelofibrosis treated with fedratinib. Future Oncol. 2025 Feb;21(5):579-91. doi: 10.1080/14796694.2025.2454895
AIM: Assess real-world fedratinib (FEDR) treatment patterns and clinical outcomes in patients with primary or secondary myelofibrosis following discontinuation of ruxolitinib (RUX).
Khanji C, Nuabor W, Kim HK, Yubin SA, Kim R, Njue A. Systematic literature review of the humanistic burden of hemophilia A and B. Poster presented at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Supplement):S613. doi: 10.1016/j.jval.2024.10.3082
OBJECTIVES: Hemophilia causes substantial clinical burden, which can lead to reduced health-related quality of life (HRQOL) in patients. This systematic literature review aimed to examine the humanistic burden of hemophilia A or B, with or without inhibitors in patients ≥ 12 years and their caregivers.
Khanji C, Nuabor W, Kim HK, Yubin SA, Kim R, Njue A. Systematic literature review of the economic burden of hemophilia A and B. Poster presented at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Supplement):S629. doi: 10.1016/j.jval.2024.10.3157
OBJECTIVES: This systematic literature review (SLR) aimed to investigate the economic burden of hemophilia A and B.
Yubin Sohn A, Khanji C, Nuabor W, Gould T, Kim HK, Barrows SM, Herbel B, Njue A. Effect of adherence to hemophilia drug therapy on outcomes: a systematic literature review. Poster presented at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Supplement):S634. doi: 10.1016/j.jval.2024.10.3181
Kosa K, Vanegas A, Colavecchia C, Coulter J, Christopher SA, Fasano RM, Chou ST, Drachtman RA, Purdie DM, Baker CL, Meier ER. Qualitative interview study to characterize the treatment experiences of participants with sickle cell disease and assess perceptions of red blood cell transfusions. Poster presented at the American Society of Hematology (ASH) Annual Meeting & Exposition; December 8, 2024. San Diego, CA.
Yan S, McDade C, Thiruvillakkat K, Rouse R, Sivamurthy K, Wilson M. Analysis of long-term clinical and cost impact of etranacogene dezaparvovec for the treatment of hemophilia B population in the United States. J Med Econ. 2024 Dec;27(1):758-65. doi: 10.1080/13696998.2024.2351762
INTRODUCTION: Etranacogene dezaparvovec (EDZ), HemgenixⓇ,is a gene therapy recently approved for people with hemophilia B (PwHB).
OBJECTIVE: To estimate long-term clinical impact and cost of EDZ in the United States (US).