Hematology

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


OBJECTIVES: To adapt a patient-reported outcome (PRO) measure, the Western Ontario McMaster Universities Osteoarthritis Index (WOMAC), into efficacy attributes for a discrete-choice experiment (DCE) survey designed to quantify the relative importance of endpoints commonly used in knee osteoarthritis (KOA) trials.

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


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


OBJECTIVE: Sickle cell disease (SCD) is a genetic hematologic disorder with multi-system impacts contributing to long-term end-organ damage. Our objective was to assess the extent to which associations among SCD complications have been quantified in the literature to inform cost-effectiveness modeling for SCD therapies with curative intent.

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


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.


BACKGROUND: Patients (pts) with LR-MDS experience a mean life-year loss of 6 years, and the treatment focus is improvement of quality of life and prolongation of life expectancy.

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


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).


OBJECTIVES: A recent FDA-sponsored PPI study of knee osteoarthritis (KOA) treatments incorporated assessments of comprehension and response consistency to evaluate the logical soundness of the data.

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


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


PURPOSE Anaphylaxis (ANA) is an important adverse drug reaction. We examined positive predictive values (PPV) and other test characteristics of ICD-10-GM code algorithms for detecting ANA as used in a multinational safety study (PASS).

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


PURPOSE: This post-authorisation safety study estimated the risk of anaphylaxis in patients receiving intravenous (IV) iron in Europe, with interest in iron dextran and iron non-dextrans. Studies conducted in the United States have reported risk of anaphylaxis to intravenous iron ranging from 2.0 to 6.8 per 10,000 first treatments.

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