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Genetic Disease

Association of factor expression levels with health-related quality of life and direct medical costs for people with haemophilia B

ORCID Icon, , , , ORCID Icon, , , , ORCID Icon & ORCID Icon show all
Pages 386-392 | Received 10 Jan 2022, Accepted 02 Mar 2022, Published online: 15 Mar 2022

References

  • Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014;12(11):1935–1939.
  • Franchini M. The modern treatment of haemophilia: a narrative review. Blood Transfus. 2013;11(2):178–182.
  • Chen CX, Baker JR, Nichol MB. Economic burden of illness among person with hemophilia B from HUGS Vb: examining the association of severity and treatment regimens with costs and annual bleed rates. Value Health. 2017;20(8):1074–1082.
  • Martin LR, Williams SL, Haskard KB, et al. The challenge of patient adherence. Ther Clin Risk Manag. 2005;1(3):189–199.
  • Thornburg CD, Duncan NA. Treatment adherence in hemophilia. Patient Prefer Adherence. 2017;11:1677–1686.
  • Schrijvers LH, Beijlevelt‐van der Zande M, Peters M, et al. Adherence to prophylaxis and bleeding outcome in haemophilia: a multicentre study. Br J Haematol. 2016;174(3):454–460.
  • O’Hara J, Walsh S, Camp C, et al. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life. Health Qual Life Outcomes. 2018;16(1):84.
  • Witkop M, Neff A, Buckner TW, et al. Self-reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the pain, functional impairment, and quality of life (P-FiQ) study. Haemophilia. 2017;23(4):556–565.
  • Witkop ML, Lambing A, Nichols CD, et al. Interrelationship between depression, anxiety, pain, and treatment adherence in hemophilia: results from a US cross-sectional survey. PPA. 2019;13:1577–1587.
  • O'Hara J, Walsh S, Camp C, et al. The relationship between target joints and direct resource use in severe haemophilia. Health Econ Rev. 2018;8(1):1.
  • Spadarella G, Minno AD, Milan G, et al. Paradigm shift for the treatment of hereditary haemophilia: towards precision medicine. Blood Rev. 2020;39:100618.
  • Dolan G, Benson G, Duffy A, et al. Haemophilia B: where are we now and what does the future hold? Blood Rev. 2018;32(1):52–60.
  • den Uijl IEM, Fischer K, Van Der Bom JG, et al. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels. Haemophilia. 2011;17(1):41–44.
  • Nathwani A. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1–8.
  • Burke T, Shaikh A, Ali TM, et al. Bleeding data across baseline FIX expression level in people with hemophilia B: an analysis using the ‘factor expression study. Blood. 2021;138(Supplement 1):592–592.
  • Burke T, Asghar S, O'Hara J, et al. Clinical, humanistic, and economic burden of severe hemophilia B in the United States: results from the CHESS US and CHESS US + population surveys. Orphanet J Rare Dis. 2021;16(1):143.
  • O'Hara J, Hughes D, Camp C, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis. 2017;12(1):106.
  • Rabin R, de Charro F. EQ-5D: a measure of health status from the EuroQol Group. Ann Med. 2001;33(5):337–343.
  • O'Hara J, Martin AP, Nugent D, et al. Evidence of a disability paradox in patient-reported outcomes in haemophilia. Haemophilia. 2021;27(2):245–252.
  • Harrell FE, Lee KL, Pollock BG. Regression models in clinical studies: determining relationships between predictors and response. J Natl Cancer Inst. 1988;80(15):1198–1202.
  • Pierce GF, Ragni MV, van den Berg HM, et al. Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies. Haemophilia. 2017;23(5):643–644.
  • Orio A, Skinner MW, Clearfield E, et al. Core outcome set for gene therapy in haemophilia: results of the coreHEM multistakeholder project. Haemophilia. 2018;24(4):e167–e172.