Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set
van Balen, Erna C.; O'Mahony, Brian; Cnossen, Marjon H.; Dolan, Gerard; Blanchette, Victor S.; Fischer, Kathelijn; Gue, Deborah; O'Hara, Jamie; Iorio, Alfonso; Jackson, Shannon; Konkle, Barbara A.; Nugent, Diane J.; Coffin, Donna; Skinner, Mark W.; Smit, Cees; Srivastava, Alok; van Eenennaam, Fred; van der Bom, Johanna G.; Gouw, Samantha C.
(2021) Research and practice in thrombosis and haemostasis, volume 5, issue 4, pp. 1 - 13
(Article)
Abstract
Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all
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individuals with hemophilia. Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons with hemophilia and multidisciplinary health care providers were involved in an iterative nominal consensus process to select the most important health outcomes and risk adjustment variables for persons with hemophilia. Recommendations were made for outcome measurement instruments. Results: Persons with hemophilia were defined as all men and women with an X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII or IX with plasma activity levels <40 IU/dL. We recommend collecting the following 10 health outcomes at least annually, if applicable: (i) cure, (ii) impact of disease on life expectancy, (iii) ability to engage in normal daily activities, (iv) severe bleeding episodes, (v) number of days lost from school or work, (vi) chronic pain, (vii) disease and treatment complications, (viii) sustainability of physical functioning, (ix) social functioning, and (x) mental health. Validated clinical as well as patient-reported outcome measurement instruments were endorsed. Demographic factors, baseline clinical factors, and treatment factors were identified as risk-adjustment variables. Conclusion: A consensus-based international set of health outcomes relevant to all persons with hemophilia, and corresponding measurement instruments, was identified for use in clinical care to facilitate harmonized longitudinal monitoring and comparison of outcomes.
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Keywords: delivery of health care, health care, hemophilia A, hemophilia B, outcome assessment, patient-reported outcome measures, Hematology
ISSN: 2475-0379
Publisher: Wiley-Blackwell Publishing Ltd
Note: Funding Information: The authors thank Vincent Wiersma and Matthijs van der Linde (The Decision Group) for assistance with project management, as well as the Patients and Health Care Professionals Panel for their participation and the International Academic Council for their valuable feedback: Rezan Abdul-Kadir, Sulochana Badagabettu, Marlène Beijlevelt, Petra Buckova, Frederica Cassis, Randall Curtis, Susan Cutter, Judy Ann David, Mariette Driessens, Brian Feldman, Leonard Friedman, Cesar Garrido, Khalid Habaybeh, Lotte Haverman, Mathieu Jackson, Pedro Jardim, Radek Kaczmarek, Kate Khair, Piet de Kleijn, Ilmar Kruis Ed Kuebler, Annamma Kurien, Johnny Mahlangu, Mike Makris, Paul McLaughlin, Pamela Narayan, Rungrote Natesirinilkul, Yasu Nishida, Declan Noone, David Page, Glenn Pierce, Suzie Peterson, Pia Petrini, Suely Rezende, R. Sathyanarayanan, Sheldon Simson, Roberto Solinis Nuño, Fendi Valdez, Pamela Wilton, and Deon York. Affiliations of participants are included in the Supporting Information. We would like to thank Elizabeth Clearfield for sharing the literature search of the CoreHEM project, medical librarian Jan Schoones for his assistance in the literature search update, and Nancy Young for critical feedback on the first draft of the manuscript. Funding Information: EvB, BO, GD, DG, JO, BK, DN, DC, and CS declare no conflicts of interest. MC has received grants from governmental research institutes, such as the Dutch Research Institute (NWO), ZonMW, Innovation fund, NWO‐NWA, and unrestricted investigator initiated research grants, as well as educational and travel funding from various companies (Pfizer, Baxter/Baxalta/Shire, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma), and has served as a member on steering boards of Roche and Bayer. All grants, awards, and fees go to the Erasmus MC. VB’s institution holds the copyright to CHO‐KLAT. KF’s institution holds the copyright to HAL and PedHAL. AI’s institution has received project‐based funding via research or service agreements with Bayer, CSL, Grifols, NovoNordisk, Octapharma, Pfizer, Roche, Sanofi, Sobi, and Takeda. Shannon Jackson has received research funding from Bioverativ, Sanofi, Bayer, and Spark Therapeutics. MS’s institution received independent investigator‐initiated research support for the PROBE study from Bayer, CSL, Novo Nordisk, Roche, and Sanofi, and his institution holds the copyright to PROBE. AS’s institution holds the copyright to the Functional Independence Score in Hemophilia (FISH). FvE’s firm received financial compensation for facilitation of the process. JvdB reports personal fees from Bayer (teaching activities), personal fees from NovoNordisk (consulting), outside the submitted work. SCG reports SOBI medical research grants, outside the submitted work. Publisher Copyright: © 2021 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).
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