Psychometrics of patient-reported outcomes measurement information system in von Willebrand disease, inherited platelet function disorders, and rare bleeding disorders

Autor: Evelien S. van Hoorn, Sterre P.E. Willems, Wala Al Arashi, Annick S. de Moor, Calvin B. van Kwawegen, Lorynn Teela, Martijn A.H. Oude Voshaar, Idske C.L. Kremer Hovinga, Roger E.G. Schutgens, Saskia E.M. Schols, Frank W.G. Leebeek, Lotte Haverman, Marjon H. Cnossen, Samantha C. Gouw, Hester F. Lingsma, Marjolein Peters, Michiel Coppens, Marieke J.H.A. Kruip, Lize F.D. van Vulpen, Tessa C.M. van Gastel

Publikováno v: Research and Practice in Thrombosis and Haemostasis, Vol 8, Iss 4, Pp 102474- (2024)

Background: Patient-reported outcomes measurement information system (PROMIS) measures can be used to measure patient-reported outcomes. PROMIS measures, including computer adaptive tests (CATs) and short forms, have demonstrated the ability to adequ

Externí odkaz: https://doaj.org/article/2ef8f16a48d044af8da2662bad9acdb2

The ethics of gene therapy for hemophilia

Autor: Lieke Baas, Rieke van der Graaf, Evelien S. van Hoorn, Annelien L. Bredenoord, Karina Meijer

Publikováno v: Journal of Thrombosis and Haemostasis. 21(3):413-420

Gene therapy is expected to become a promising treatment, and potentially even a cure, for hemophilia. After several years of research, the first gene therapy product has been granted conditional market authorization by the European Union in August 2

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8ac8124a5b957ee192f39f8c072ad610
https://doi.org/10.1016/j.jtha.2022.12.027

Generic PROMIS item banks in adults with hemophilia for patient-reported outcome assessment:Feasibility, measurement properties, and relevance

Autor: Lize F D van Vulpen, Michiel Coppens, Evelien S van Hoorn, Jelmer J Muis, Krista Fischer, Isolde A R Kuijlaars, Samantha C. Gouw, Lorynn Teela, Lotte Haverman, Merel A. Timmer, Marjon H. Cnossen, Marieke J. H. A. Kruip, Marjolein Peters

Publikováno v: Kuijlaars, I A R, Teela, L, van Vulpen, L F D, Timmer, M A, Coppens, M, Gouw, S C, Peters, M, Kruip, M J H A, Cnossen, M H, Muis, J J, van Hoorn, E S, Haverman, L & Fischer, K 2021, ' Generic PROMIS item banks in adults with hemophilia for patient-reported outcome assessment : Feasibility, measurement properties, and relevance ', Research and practice in thrombosis and haemostasis, vol. 5, no. 8, e12621 . https://doi.org/10.1002/rth2.12621
Research and practice in thrombosis and haemostasis, 5(8):e12621. Wiley-Blackwell Publishing Ltd
Research and Practice in Thrombosis and Haemostasis, 5(8):e12621. Wiley-Blackwell Publishing Ltd
Research and Practice in Thrombosis and Haemostasis, Vol 5, Iss 8, Pp n/a-n/a (2021)
Research and Practice in Thrombosis and Haemostasis

Background: Legacy hemophilia-specific questionnaires are considered too long, show floor-/ceiling effects, and/or include irrelevant questions. Patient Reported Outcomes Measurement Information System (PROMIS) item banks, including Computer Adaptive

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0a9aa05ad6a2a98343e369fa373e1453
https://research.vumc.nl/en/publications/99431585-dcb3-48f0-b86c-877b0a5be695