Kato, G. J., Piel, F. B., Reid, C. D., Gaston, M. H., Ohene-Frempong, K., Krishnamurti, L., et al. (2018). Sickle cell disease. Nat Rev Dis Primers, 4, 18010. https://doi.org/10.1038/nrdp.2018.10
Piel, F. B., Steinberg, M. H., & Rees, D. C. (2017). Sickle cell disease. New England Journal of Medicine, 376(16), 1561–1573. https://doi.org/10.1056/NEJMra1510865
Article CAS PubMed Google Scholar
Osunkwo, I., Andemariam, B., Minniti, C. P., Inusa, B. P. D., El Rassi, F., Francis-Gibson, B., et al. (2021). Impact of sickle cell disease on patients’ daily lives, symptoms reported, and disease management strategies: Results from the international sickle cell world assessment survey (SWAY). American Journal of Hematology, 96(4), 404–417. https://doi.org/10.1002/ajh.26063
Article PubMed PubMed Central Google Scholar
Esham, K. S., Rodday, A. M., Smith, H. P., Noubary, F., Weidner, R. A., Buchsbaum, R. J., et al. (2020). Assessment of health-related quality of life among adults hospitalized with sickle cell disease vaso-occlusive crisis. Blood Adv, 4(1), 19–27. https://doi.org/10.1182/bloodadvances.2019000128
Holdford, D., Vendetti, N., Sop, D. M., Johnson, S., & Smith, W. R. (2021). Indirect economic burden of sickle cell disease. Value in Health: the Journal of the international Society for Pharmacoeconomics and Outcomes Research, 24(8). https://doi.org/10.1016/j.jval.2021.02.014. 1095– 101.
Haywood, C. Jr., Tanabe, P., Naik, R., Beach, M. C., & Lanzkron, S. (2013). The impact of race and disease on sickle cell patient wait times in the emergency department. American Journal of Emergency Medicine, 31(4), 651–656. https://doi.org/10.1016/j.ajem.2012.11.005
(2023). 30th Annual Conference of the International Society for Quality of Life Research. Quality of Life Research. 32 (2), 23–220. https://doi.org/10.1007/s11136-023-03530-x
U.S (2014). Department of Health and Human Services, Health NIo. Evidence-Based Management of Sickle Cell Disease: Expert Panel Report.
Lee, L., Smith-Whitley, K., Banks, S., & Puckrein, G. (2019). Reducing health care disparities in sickle cell disease: A review. Public Health Reports, 134(6), 599–607. https://doi.org/10.1177/0033354919881438
Article PubMed PubMed Central Google Scholar
Drahos, J., Boateng-Kuffour, A., Calvert, M., Valentine, A., Mason, A., Lauren, L. (2023). Health-Related Quality of Life Impacts Associated With Sickle Cell Disease in the United States and United Kingdom: A Qualitative Assessment. International Society for Quality of Life Research (ISOQOL) 2023 Annual Conference. Oral Session 307: Clinical Research and Application.
von Elm, E., Altman, D. G., Egger, M., Pocock, S. J., Gotzsche, P. C., Vandenbroucke, J. P., et al. (2008). The strengthening the reporting of observational studies in epidemiology (STROBE) statement: Guidelines for reporting observational studies. Journal of Clinical Epidemiology, 61(4), 344–349. https://doi.org/10.1016/j.jclinepi.2007.11.008
Brucker, P. S., Yost, K., Cashy, J., Webster, K., & Cella, D. (2005). General population and cancer patient norms for the functional assessment of cancer Therapy-General (FACT-G). Evaluation and the Health Professions, 28(2), 192–211. https://doi.org/10.1177/0163278705275341
Cella, D., Lai, J. S., Chang, C. H., Peterman, A., & Slavin, M. (2002). Fatigue in cancer patients compared with fatigue in the general united States population. Cancer, 94(2), 528–538. https://doi.org/10.1002/cncr.10245
Jiang, R., Janssen, M. F. B., & Pickard, A. S. (2021). US population norms for the EQ-5D-5L and comparison of norms from face-to-face and online samples. Quality of Life Research, 30(3). https://doi.org/10.1007/s11136-020-02650-y. 803– 16.
The Association of the British Pharmaceutical Industry Code of Practice for the Pharmaceutical Industry 2021 2021 [updated 20 September 2023. Available from: https://www.abpi.org.uk/publications/code-of-practice-for-the-pharmaceutical-industry-2021/
European Pharmaceutical Market Research Association Code of Conduct 2022 [Available from: https://www.ephmra.org/sites/default/files/2022-08/EPHMRA%202022%20Code%20of%20Conduct.pdf
British Healthcare Business Intelligence Association. Legal and Ethical Guidelines for Healthcare Market Research Your essential guide 2023 [updated September 2023. Available from: https://www.bhbia.org.uk/guidelines-and-legislation/legal-and-ethical-guidelines
Henry, E. B., Barry, L. E., Hobbins, A. P., McClure, N. S., & O’Neill, C. (2020). Estimation of an Instrument-Defined minimally important difference in EQ-5D-5L index scores based on scoring algorithms derived using the EQ-VT version 2 valuation protocols. Value in Health: the Journal of the international Society for Pharmacoeconomics and Outcomes Research, 23(7), 936–944. https://doi.org/10.1016/j.jval.2020.03.003
Pickard, A. S., Neary, M. P., & Cella, D. (2007). Estimation of minimally important differences in EQ-5D utility and VAS scores in cancer. Health and Quality of Life Outcomes, 5, 70. https://doi.org/10.1186/1477-7525-5-70
Article PubMed PubMed Central Google Scholar
Maziarz, R. T., Waller, E. K., Jaeger, U., Fleury, I., McGuirk, J., Holte, H., et al. (2020). Patient-reported long-term quality of life after tisagenlecleucel in relapsed/refractory diffuse large B-cell lymphoma. Blood Adv, 4(4), 629–637. https://doi.org/10.1182/bloodadvances.2019001026
Article PubMed PubMed Central Google Scholar
Shah, J., Shacham, S., Kauffman, M., Daniele, P., Tomaras, D., Tremblay, G., et al. (2021). Health-related quality of life and utility outcomes with Selinexor in relapsed/refractory diffuse large B-cell lymphoma. Future Oncology, 17(11), 1295–1310. https://doi.org/10.2217/fon-2020-0946
Article CAS PubMed Google Scholar
Montan, I., Löwe, B., Cella, D., Mehnert, A., & Hinz, A. (2018). General population norms for the functional assessment of chronic illness therapy (FACIT)-Fatigue scale. Value in Health: the Journal of the international Society for Pharmacoeconomics and Outcomes Research, 21(11), 1313–1321. https://doi.org/10.1016/j.jval.2018.03.013
Keller, S., Yang, M., Evensen, C., & Cowans, T. (2017). ASCQ-ME USER’S MANUAL.
Keller, S. D., Yang, M., Treadwell, M. J., Werner, E. M., & Hassell, K. L. (2014). Patient reports of health outcome for adults living with sickle cell disease: Development and testing of the ASCQ-Me item banks. Health and Quality of Life Outcomes, 12, 125. https://doi.org/10.1186/s12955-014-0125-0
Article PubMed PubMed Central Google Scholar
Dampier, C., LeBeau, P., Rhee, S., Lieff, S., Kesler, K., Ballas, S., et al. (2011). Health-related quality of life in adults with sickle cell disease (SCD): A report from the comprehensive sickle cell centers clinical trial consortium. American Journal of Hematology, 86(2), 203–205. https://doi.org/10.1002/ajh.21905
Article PubMed PubMed Central Google Scholar
Rizio, A. A., Bhor, M., Lin, X., McCausland, K. L., White, M. K., Paulose, J., et al. (2020). The relationship between frequency and severity of vaso-occlusive crises and health-related quality of life and work productivity in adults with sickle cell disease. Quality of Life Research, 29(6), 1533–1547. https://doi.org/10.1007/s11136-019-02412-5
Article PubMed PubMed Central Google Scholar
Elendu, C., Amaechi, D. C., Alakwe-Ojimba, C. E., Elendu, T. C., Elendu, R. C., Ayabazu, C. P., et al. (2023). Understanding sickle cell disease: Causes, symptoms, and treatment options. Medicine (Baltimore), 102(38), e35237. https://doi.org/10.1097/md.0000000000035237
Article CAS PubMed Google Scholar
Anderson, L. M., Allen, T. M., Thornburg, C. D., & Bonner, M. J. (2015). Fatigue in children with sickle cell disease: Association with neurocognitive and Social-Emotional functioning and quality of life. Journal of Pediatric Hematology/Oncology, 37(8), 584–589. https://doi.org/10.1097/MPH.0000000000000431
Udeze, C., Evans, K. A., Yang, Y., Lillehaugen, T., Manjelievskaia, J., Mujumdar, U., et al. (2023). Economic and clinical burden of managing sickle cell disease with recurrent Vaso-Occlusive crises in the united States. Adv Ther, 40(8), 3543–3558. https://doi.org/10.1007/s12325-023-02545-7
Article PubMed PubMed Central Google Scholar
Bradt, P., Spackman, E., Synnott, P., Chapman, R., Beinfeld, M., Rind, D. (2020). Crizanlizumab, voxelotor, and L-glutamine for sickle cell disease: Effectiveness and value. Institute for Clinical and Economic Review. 23.
Lubeck, D., Agodoa, I., Bhakta, N., Danese, M., Pappu, K., Howard, R., et al. (2019). Estimated life expectancy and income of patients with sickle cell disease compared with those without sickle cell disease. JAMA Netw Open, 2(11), e1915374. https://doi.org/10.1001/jamanetworkopen.2019.15374
Article PubMed PubMed Central Google Scholar
Power-Hays, A., & McGann, P. T. (2020). When actions speak louder than Words - Racism and sickle cell disease. New England Journal of Medicine, 383 (20), 1902–1903. https://doi.org/10.1056/NEJMp2022125
FDA Approves First (2023). Gene Therapies to Treat Patients with Sickle Cell Disease [press release].
Power-Hays, A., & McGann, P. T. (2020). When actions speak louder than Words — Racism and sickle cell disease. New England Journal of Medicine, 383(20), 1902–1903. https://doi.org/10.1056/NEJMp2022125
Inusa, B. P. D., James, J., Tinga, B., Ba, D., Ingoli, E., Hartfield, R. (2023). 5612992 Sickle Cell Health Awareness, Perspectives, and Experiences (Shape) Survey: Findings on the Burden of Sickle Cell Disease and Impact on the Quality of Life of Patients and Caregivers in the Uk. HemaSphere. 7 (S1), 10– 1. https://doi.org/10.1097/01.HS9.0000928200.69096.c5
Pickard, A. S., Law, E. H., Jiang, R., Pullenayegum, E., Shaw, J. W., Xie, F., et al. (2019). United States valuation of EQ-5D-5L health States using an international protocol. Value in Health: the Journal of the international Society for Pharmacoeconomics and Outcomes Research, 22(8). https://doi.org/10.1016/j.jval.2019.02.009. 931– 41.
Hernández Alava, M., Pudney, S., & Wailoo, A. (2023). Estimating the relationship between EQ-5D-5L and EQ-5D-3L: Results from a UK population study. Pharmacoeconomics, 41(2), 199–207. https://doi.org/10.1007/s40273-022-01218-7
Andrade, L. F., Ludwig, K., Goni, J. M. R., Oppe, M., & de Pouvourville, G. (2020). A French Value Set for the EQ-5D-5L. Pharmacoeconomics. 38 (4), 413– 25. https://doi.org/10.1007/s40273-019-00876-4
Ludwig, K., von der Graf, J. M., & Greiner, W. (2018). German value set for the EQ-5D-5L. Pharmacoeconomics, 36(6). https://doi.org/10.1007/s40273-018-0615-8. 663– 74.
Finch, A. P., Meregaglia, M., Ciani, O., Roudijk, B., & Jommi, C. (2022). An EQ-5D-5L value set for Italy using videoconferencing interviews and feasibility of a new mode of administration. Social Science and Medicine, 292, 114519.
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