Publications
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Displaying 1 through 50 of 113 publications.
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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
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
Khanji C, Nuabor W, Gould T, Kim HK, Barrows S, Herbel B, Njue A. Systematic literature review of outcomes associated with adherence to haemophilia drug therapy. Haemophilia. 2025 Mar;31(2):187-206. doi: 10.1111/hae.15153
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
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
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.
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
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
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
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
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
Passamonti F, Parikh RC, Korgaonkar S, Chevli M, Yucel A, Rombi J, Zissler D, Davis KL, Slaff S. Patient characteristics, treatment patterns, and health outcomes in a real-world population of patients with myelofibrosis treated with fedratinib. Poster presented at the European Hematology Association (EHA) 2024 Hybrid Congress; June 13, 2024. Madrid, Spain.
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.
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
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.
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
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
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.
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.
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.
Stothers Rosenberg S, Ng X, Mansfield C, Poulos C, Peay H, Lee TH, Irony T, Ho M. Adaptation of the WOMAC for use in a patient preference study. Ther Innov Regul Sci. 2023 Jul;57(4):702-11. doi: 10.1007/s43441-023-00510-8
Dong O, Purser M, Herring WL, Mladsi DM, Gallagher M, Chawla A, Leiding JW, Andemariam B. Relationships among sickle cell disease complications and their implications for cost-effectiveness modeling for therapies with curative intent. Poster presented at the ISPOR 2023 Conference; May 7, 2023. Boston, MA. [abstract] Value Health. 2023 Jun; 26(6 supplement):S115-6. doi: 10.1016/j.jval.2023.03.611
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 16th Annual Congress of European Association for Haemophilia and Allied Disorders 2023; February 7, 2023. Manchester, England. [abstract] Haematologica. 2023 Feb 5; 29(S1):137-8. doi: 10.1111/hae.14715
Ronquest N, Clinkscales M, Paret K. How are United States ICER'S evidence ratings determined?: A systematic review of ICER'S evidence ratings in evidence reports for new drugs in 2020 and 2021. Poster presented at the ISPOR Europe 2022; November 7, 2022. Vienna, Austria. [abstract] Value Health. 2022 Dec 1; 25(12):299-300. doi: 10.1016/j.jval.2022.09.1479
Srivastava A, Rangarajan S, Ross C, Slota C, DiBenedetti D, Cano V, Andersson S, DasMahapatra P, Bartelt-Hofer J, Afonso M. Fitusiran reaches people's with hemophilia and their caregivers' treatment expectations: qualitative semi-structured interviews of participants of ATLAS-OLE Trial (Interim analysis). Poster presented at the American Society of Hematology (ASH) Annual Meeting 2022; December 11, 2022. New Orleans, LA. [abstract] Blood. 2022 Nov 15; 140(Suppl 1):7989-90. doi: 10.1182/blood-2022-166895
Diez-Campelo M, Yucel A, Goyal R, Parikh R, Dhuliawala S, Jimenez M, Sluga-O'Callaghan M, Hughes C, Tang D, Germing U. Systemic therapy utilization and hematologic outcomes in lower-risk myelodysplastic syndromes (LR-MDS): findings from a real-world medical record review study in the US, UK, and Europe (EU). Poster presented at the 2022 European Hematology Association Hybrid Congress; June 10, 2022. Vienna, Austria.
Poulos C, Ng X, Mansfield C, Rosenberg S, Peay H, Lee J, Irony T, Ho M. Logical soundness in a discrete choice experiment study of benefit risk preferences. Presented at the ISPOR 2022 Conference; May 15, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).
de Sordi D, Kappen S, Otto-Sobotka F, Kulschewski A, Weyland A, Gutierrez L, Fortuny J, Reinold J, Schink T, Timmer A. Validity of hospital ICD-10-GM codes to identify anaphylaxis. Pharmacoepidemiol Drug Saf. 2021 Dec;12:1643-52. doi: 10.1002/pds.5348
Walters MC, Tisdale JF, Mapara MY, Krishnamurti L, Kwiatkowski JL, Aygun B, Kasow KA, Rifkin-Zenenberg S, Jaroscak J, Garbinsky D, Chirila C, Gallagher ME, Zhang X, Ho PR, Thompson AA, Kanter J. Sustained improvements in patient-reported quality of life up to 24 months post-treatment with LentiGlobin for sickle cell disease (bb1111) gene therapy. Presented at the 2021 63rd American Society of Hematology (ASH) Annual Meeting & Exposition; December 11, 2021. Atlanta, Georgia. [abstract] Blood. 2021 Nov 23; 138(Suppl 1):7. doi: 10.1182/blood-2021-146905
Ng X, Stothers S, Mansfield C, Poulos C, Peay H, Lee TS, Irony T, Ho M. Adaptation of the Western Ontario McMaster Universities (WOMAC) osteoarthritis index for use in a patient preference study to inform regulatory decision-making. Poster presented at the 2021 SMDM Virtual Annual Meeting; October 19, 2021.
Fortuny J, von Gersdorff G, Lassalle R, Linder M, Overbeek J, Reinold J, Toft G, Timmer A, Dress J, Blin P, Droz-Perroteau C, Ehrenstein V, Franzoni C, Herings R, Kollhorst B, Moore N, Odsbu I, Perez-Gutthann S, Schink T, Rascher K, Rasouliyan L, Rothman KJ, Saigi-Morgui N, Schaller M, Smits E, Forstner M, Intravenous Iron Consortium, Benichou J, Bircher AJ, Garbe E, Rampton DS, Gutierrez L. Use of intravenous iron and risk of anaphylaxis: a multinational observational post-authorisation safety study in Europe. Pharmacoepidemiol Drug Saf. 2021 Oct;30(10):1447-57. doi: 10.1002/pds.5319
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
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: 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
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
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.
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.
Mladsi D, Barnett C, Aggarwal K, Vredenburg M, Dieterich D, Kim R. Cost-effectiveness of avatrombopag for the treatment of thrombocytopenia in patients with chronic liver disease. Clinicoecon Outcomes Res. 2020 Sep 14;2020(12):515-26. doi: 10.2147/CEOR.S262772
Banerji A, Davis KH, Brown TM, Hollis K, Hunter SM, Long J, Jain G, Devercelli G. Patient-reported burden of hereditary angioedema: findings from a patient survey in the United States. Ann Allergy Asthma Immunol. 2020 Jun;124(6):600-7. doi: 10.1016/j.anai.2020.02.018
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.
Allen LF, Aggarwal K, Vredenburg M, Barnett C, Mladsi D, Kim R. Cost-effectiveness of avatrombopag for the treatment of thrombocytopenia in patients with chronic liver disease. Poster presented at the 61st ASH American Society of Hematology Annual Meeting and Exposition; December 8, 2019. San Diego, CA. [abstract] Blood. 2019 Nov 13; 134(Supplement_1):3454. doi: 10.1182/blood-2019-131822
Gutierrez L, Bezemer ID, Dress J, Ehrenstein V, Forstner M, Franzoni C, Lassalle R, Linder M, Droz-Perroteau C, Odsbu I, Overbeek JA, Perez-Gutthann S, Pisa FE, Rascher K, Rasouliyan L, Reinold J, Rothman KJ, Saigi-Morgui N, Schaller M, Smits E, Timmer A, Toft G, von Gersdorff G, Fortuny J. Challenges in conducting a multinational European study of severe hypersensitivity reactions among recipients of intravenous iron. Poster presented at the 35th ICPE International Conference on Pharmacoepidemiology & Therapeutic Risk Management; August 27, 2019. Philadelphia, PA. [abstract] Pharmacoepidemiol Drug Saf. 2019 Aug 20; 28(S2):640. doi: 10.1002/pds.4864
Mladsi DM, Barnett CL, Kaye JA. Identification of potential bias from data sources and study designs used to estimate platelet transfusion adverse event rates. Poster presented at the 2018 ISPOR 21st Annual European Congress; November 12, 2018. Barcelona, Spain.
Assimon MM, Brookhart MA, Fine JP, Heiss G, Layton JB, Flythe JE. A comparative study of carvedilol versus metoprolol initiation and 1-year mortality among individuals receiving maintenance hemodialysis. Am J Kidney Dis. 2018 Sep;72(3):337-48. doi: 10.1053/j.ajkd.2018.02.350.
Barnett CL, Mladsi D, Vredenburg M, Aggarwal K. Cost estimate of platelet transfusion in the United States for patients with chronic liver disease and associated thrombocytopenia undergoing elective procedures. J Med Econ. 2018 Aug;21(8):827-34. doi: 10.1080/13696998.2018.1490301
Gamble JF, Kurian DJ, Hopkins TJ, Guinn NR, Grimsley A, Jimenez M, Aronson S. Preoperative anemia clinic: optimizing hemoglobin for elective hip and knee arthroplasty. Poster presented at the American Society of Anesthesiologists 2016 Annual Conference; October 25, 2016. Chicago, IL.
Kurian DJ, Gamble JF, Hopkins TJ, Guinn NR, Grimsley A, Jimenez MI, Aronson S. Preoperative blood management strategy for elective hip and knee arthroplasty: preliminary findings. Poster presented at the American Society of Anesthesiologists 2016 Annual Conference; October 22, 2016. Chicago, IL.
Guinn NR, Guercio JR, Hopkins TJ, Grimsley A, Kurian TJ, Jimenez ML, Bolognesi MP, Schroeder R, Aronson S, Duke Perioperative Enhancement Team (POET). How do we develop and implement a preoperative anemia clinic designed to improve perioperative outcomes and reduce cost? Transfusion. 2016 Feb;56(2):297-303. doi: 10.1111/trf.13426
Brockbank J, Wolowacz S. Economic evaluations of new oral anticoagulants for the prevention of venous thromboembolism after total hip or total knee replacement. Poster presented at the 2015 ISPOR 18th Annual European Congress; November 2015. Milan, Italy.