Hematology
DiBenedetti D, Neme D, Pan-Petesch B, Willemze A, Wynn T, Kragh N, Wilson A. Patient experience with efanesoctocog alfa for severe hemophilia A: results from the XTEND-1 phase 3 clinical study exit interviews. Clin Ther. 2024 Dec;46(12):1016-23. doi: 10.1016/j.clinthera.2024.09.010
Campelo MD, Yucel A, Goyal RK, Glassberg MB, Esterberg L, Rombi J, Davis KL, Jimenez M, Miteva D, Germing U. Luspatercept utilization patterns in lower-risk myelodysplastic syndrome (LR-MDS): findings from a multinational medical record review study. Poster presented at the European Hematology Association (EHA) 2024 Hybrid Congress; June 13, 2024. Madrid, Spain.
BACKGROUND: Limited evidence exists from routine clinical practices on patient characteristics and treatment patterns in patients treated with luspatercept for lower-risk myelodysplastic syndrome (LRMDS).
Fermann GJ, Cash BD, Coelho-Prabhu N, Maegele M, Bingisser R, Sehgal V, Cohen AT, Golden AH, Russo J, Price M, Mangel A, Koch B, Christoph MJ, Milling, Jr. TJ. Definition of factor Xa inhibitor–related, life-threatening gastrointestinal bleeding and guidance on when to use reversal therapy: a Delphi panel. J Am Coll Emerg Physicians Open. 2023 Oct 2;4(5):e13043. doi: 10.1002/emp2.13043
OBJECTIVE: To define and contextualize life-threatening gastrointestinal (GI) bleeding in the setting of factor Xa (FXa) inhibitor therapy and to derive a consensus-based, clinically oriented approach to the administration of FXa inhibitor reversal therapy.
Herring WL, Gallagher ME, Shah N, Morse KC, Mladsi D, Dong OM, Chawla A, Leiding JW, Zhang L, Paramore C, Andemariam B. Cost-effectiveness of lovotibeglogene autotemcel (lovo-cel) gene therapy for patients with sickle cell disease and recurrent vaso-occlusive events in the United States. Pharmacoeconomics. 2024 Jun;42(6):693-714. doi: 10.1007/s40273-024-01385-9
Wilson A, Kragh N, DiBenedetti D, Pan-Petesch B, Wynn T, Neme D, Willemze A. Patient experience with efanesoctocog alfa: results from the XTEND-1 phase 3 clinical trial exit interviews in patients with severe haemophilia A. Poster presented at the 75th Bleeding Disorders Conference (BDC); August 17, 2023. Baltimore, MD. Previously presented at the 16th Annual Congress of European Association for Haemophilia and Allied Disorders 2023.
Fan Q, Hawe E, Yang J, Burgess B, Bullano M, Mokdad A. Cost outcomes of noninhibitor patients with hemophilia A switching from prophylaxis with factor VIII to emicizumab: a meta-analysis of real-world evidence studies in the United States. Poster presented at the AMCP Nexus 2023; October 16, 2023. Orlando, FL. [abstract] J Manag Care Pharm. 2023 Oct; 29(10-a Suppl):S35.
BACKGROUND: Individuals with hemophilia A (HA) may be prophylactically treated with factor VIII (FVIII) or nonfactor therapies (eg, emicizumab) to prevent or reduce the fre-quency of bleeding episodes.
Curtis R, Decker-Palmer M, Wilson MR, McDade CL, Lin CW, Wallick C, Ko RH. Cost-effectiveness of emicizumab vs efanesoctocog alfa, standard half life (SHL) and other extended half life (EHL) FVIII products for prophylaxis in people with severe hemophilia a without inhibitors. Poster presented at the 65th ASH Annual Meeting & Exposition 2023; December 11, 2023. San Diego, CA. [abstract] Blood. 2023 Nov; 142(Supplement 1):5053-4. doi: 10.1182/blood-2023-181062
Malec L, Carcao M, Mathias M, Kragh N, Dumont J, Willemze A, Bystricka L, Wilson A, DiBenedetti D. Experiences with efanesoctocog alfa: exit interviews with caregivers of previously treated patients with hemophilia a from the XTEND-Kids phase 3 clinical trial. Presented at the 65th ASH Annual Meeting & Exposition 2023; December 10, 2023. San Diego, CA.
Wilson M, McDade C, Thiruvillakkat K, Rouse R, Sivamurthy K, Yan S. Long-term impact of the gene therapy etranacogene dezaparvovec for the treatment of hemophilia B in the United States. Poster presented at the AMCP Nexus 2023; October 17, 2023. Orlando, FL. [abstract] J Manag Care Pharm. 2023 Oct; 29(10-a Suppl):S36-7.
BACKGROUND: Etranacogene dezaparvovec (EDZ) is a gene therapy recently approved for use in people with hemophilia B (PwHB). EDZ increases FIX activity levels, reduces the risk of breakthrough bleeding episodes and eliminates the need for routine, FIX prophylaxis replacement therapy.