Hematology
Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S14-20. doi: 10.18553/jmcp.2020.26.12-b.s14
Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S3-8. doi: 10.18553/jmcp.2020.26.12-b.s3
Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S8-S14. doi: 10.18553/jmcp.2020.26.12-b.s8
Sun SX, Olayinka-Amao O, DiBenedetti D. Assessing patient experiences with prophylactic treatments for hemophilia A: concept elicitation for gene therapy. Poster presented at the 62nd Annual Virtual Meeting of the American Society of Hematology (ASH); December 5, 2020. [abstract] Blood. 2020 Nov 5; 136(Suppl 1):33-4.
BACKGROUND: Gene therapy for hemophilia A is designed to be a one-time infusion to deliver functional copies of the defective factor VIII (FVIII) gene, to facilitate the endogenous production of therapeutic FVIII levels. The aim is to achieve long-term protection from bleeds without the burden of regular infusions.
Purser M, Gallagher M, Mladsi D, Weber JM, Andemariam B, Kaye JA, Chawla A. Evaluation of published models in sickle cell disease against key criteria for an economic model for a potentially curative one-time treatment. Poster presented at the Virtual ISPOR Europe 2020 Conference; November 2020. [abstract] Value Health. 2020 Dec; 23(S2). doi: 10.1016/j.jval.2020.08.098
Aggarwal K, Vredenburg M, Mladsi D, Barnett C. Cost-effectiveness of avatrombopag for the treatment of thrombocytopenia in patients with chronic liver disease. Poster presented at the 2020 AMCP Meeting (Conference cancelled); April 2020. [abstract] J Manag Care Pharm. 2020 Apr; 26(Supplement 4a):s58.
Purser M, Gallagher M, Mladsi D, Weber JM, Andermariam B, Kaye JA, Chawla A. Development of key criteria for an economic model for a potentially curative one-time treatment for sickle cell disease and evaluation of published models with respect to these criteria. Hemasphere. 2020;p.1098-9.
Gutierrez L, Blin P, Dress J, Droz-Perroteau C, Ehrenstein V, Franzoni C, Kollhorst B, Lassalle R, Linder M, Moore N, Odsbu I, Overbeek J, Perez-Gutthann S, Schink T, Rascher K, Rasouliyan L, Reinold J, Rothman KJ, Saigi-Morgui N, Schaller M, Smits E, Timmer A, Toft G, von Gersdorff G, Intravenous Iron Consortium, Fortuny J. A multinational European study of anaphylaxis among recipients of intravenous iron. Poster presented at the 2020 36th ICPE International Virtual Conference on Pharmacoepidemiology & Therapeutic Risk Management; September 16, 2020.
BACKGROUND: Anaphylaxis (ANA) related to intravenous (IV) iron treatment is a poorly characterized safety concern in Europe. A postauthorization safety study requested by the European Medicines Agency assessed the risk of ANA in IV iron users in Europe.