References
- Barry MJ, Edgman-Levitan S. Shared decision making--pinnacle of patient-centered care. N Engl J Med 2012; 366(9): 780–1. doi: 10.1056/NEJMp1109283.
- Karazivan P, Dumez V, Flora L, et al. The patient-as-partner approach in health care: a conceptual framework for a necessary transition. Acad Med 2015; 90(4): 437–41. doi: 10.1097/ACM.0000000000000603.
- Nossair F, Thornburg CD. The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries. Ther Adv Hematol 2018; 9(8): 239–249. doi: 10.1177/2040620718784830.
- Fillion E. How is medical decision-making shared? The case of haemophilia patients and doctors: the aftermath of the infected blood affair in France. Health Expect 2003; 6(3): 228–41. doi: 10.1046/j.1369-6513.2003.00244.x.
- Berger Z. Navigating the unknown: shared decision-making in the face of uncertainty. J Gen Intern Med 2015; 30(5): 675–8. doi: 10.1007/s11606-014-3074-8.
- Montori VM, Gafni A, Charles C. A shared treatment decision-making approach between patients with chronic conditions and their clinicians: the case of diabetes. Health Expect 2006; 9(1): 25–36. doi: 10.1111/j.1369-7625.2006.00359.x.
- Elwyn G, Frosch D, Thomson R, et al. Shared decision making: a model for clinical practice. J Gen Intern Med 2012; 27(10): 1361–7. doi: 10.1007/s11606-012-2077-6.
- Hauser K, Koerfer A, Kuhr K, Albus C, Herzig S, Matthes J. Outcome-relevant effects of shared decision making. Dtsch Arztebl Int 2015; 112(40): 665–71. doi: 10.3238/arztebl.2015.0665.
- Matlock DD, Spatz ES. Design and testing of tools for shared decision making. Circ Cardiovasc Qual Outcomes 2014; 7(3): 487–92. doi: 10.1161/CIRCOUTCOMES.113.000289.
- Drug and Therapeutics Bulletin. An introduction to patient decision aids. BMJ 2013; 347: f4147. doi: 10.1136/bmj.f4147.
- Agency for Healthcare Research and Quality. The SHARE Approach. Available from https://www.ahrq.gov/professionals/education/curriculum-tools/shareddecisionmaking/index.html (accessed 28 March 2019).
- Lamiani G, Bigi S, Mancuso ME, Coppola A, Vegni E. Applying a deliberation model to the analysis of consultations in haemophilia: Implications for doctor-patient communication. Patient Educ Couns 2017; 100(4): 690–695. doi: 10.1016/j.pec.2016.11.021.
- Dolan JG. Shared decision-making--transferring research into practice: the Analytic Hierarchy Process (AHP). Patient Educ Couns 2008; 73(3): 418–25. doi: 10.1016/j.pec.2008.07.032.
- Shay LA, Lafata JE. Where is the evidence? A systematic review of shared decision making and patient outcomes. Med Decis Making 2015; 35(1): 114–31. doi: 10.1177/0272989X14551638.
- Beers E, Nilsen ML, Johnson JT. The role of patients: shared decision-making. Otolaryngol Clin North Am 2017; 50(4): 689–708. doi: 10.1016/j.otc.2017.03.006.
- Del Río-Lanza AB, Suárez-Álvarez L, Suárez-Vázquez A, Vázquez-Casielles R. Information provision and attentive listening as determinants of patient perceptions of shared decision-making around chronic illnesses. Springerplus 2016; 5(1): 1386. doi: 10.1186/s40064-016-3086-4.
- Joseph-Williams N, Lloyd A, Edwards A, et al. Implementing shared decision making in the NHS: lessons from the MAGIC programme. BMJ 2017; 357: j1744. doi: 10.1136/bmj.j1744.
- Epstein RM. Whole mind and shared mind in clinical decision-making. Patient Educ Couns 2013; 90(2): 200–6. doi: 10.1016/j.pec.2012.06.035.
- Frosch DL, Kaplan RM. Shared decision making in clinical medicine: past research and future directions. Am J Prev Med 1999; 17(4): 285–94. doi: 10.1016/s0749-3797(99)00097-5.
- Elwyn G, Frosch DL, Kobrin S. Implementing shared decision-making: consider all the consequences. Implementation Sci 2015; 11: 114. doi: 10.1186/s13012-016-0480-9.
- Barratt A. Evidence based medicine and shared decision making: the challenge of getting both evidence and preferences into health care. Patient Educ Couns 2008; 73(3): 407–12. doi: 10.1016/j.pec.2008.07.054.
- National Hemophilia Foundation. MASAC Document #241 – Recommendation concerning prophylaxis. February 28, 2016. Available from http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007 (accessed 23 June 2016).
- Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al.; Treatment Guidelines Working Group on Behalf of the World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia 2013; 19(1): e1–e47. doi: 10.1111/j.1365-2516.2012.02909.x.
- Collins PW. Personalized prophylaxis. Haemophilia 2012; 18(Suppl 4): 131–5. doi: 10.1111/j.1365-2516.2012.02838.x.
- Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia 2014; 20(5): 607–15. doi: 10.1111/hae.12438.
- Simhadri VL, Banerjee AS, Simon J, Kimchi-Sarfaty C, Sauna ZE. Personalized approaches to the treatment of hemophilia A and B. Per Med 2015; 12(4): 403–415. doi: 10.2217/pme.15.6.
- Valentino LA, Mamonov V, Hellmann A, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost 2012; 10(3): 359–67. doi: 10.1111/j.1538-7836.2011.04611.x.
- Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood 2014; 123(3): 317–25. doi: 10.1182/blood-2013-10-529974.
- Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood 2019; 133(5): 407–14. doi: 10.1182/blood-2018-07-820720.
- Rodriguez-Merchan EC, Valentino LA. Emicizumab: Review of the literature and critical appraisal. Haemophilia 2019; 25(1): 11–20. doi: 10.1111/hae.13641.
- Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia 2001; 7(4): 392–6. doi: 10.1046/j.1365-2516.2001.00534.x.
- De Moerloose P, Urbancik W, Van Den Berg HM, Richards M.A survey of adherence to haemophilia therapy in six European countries: results and recommendations. Haemophilia 2008; 14(5): 931–8. doi: 10.1111/j.1365-2516.2008.01843.x.
- Du Treil S, Rice J, Leissinger CA. Quantifying adherence to treatment and its relationship to quality of life in a well-characterized haemophilia population. Haemophilia 2007; 13(5): 493–501. doi: 10.1111/j.1365-2516.2007.01526.x.
- Thornburg CD, Carpenter S, Zappa S, Munn J, Leissinger C. Current prescription of prophylactic factor infusions and perceived adherence for children and adolescents with haemophilia: a survey of haemophilia healthcare professionals in the United States. Haemophilia 2012; 18(4): 568–74. doi: 10.1111/j.1365-2516.2012.02756.x.
- Machin N, Ragni MV, Smith KJ. Gene therapy in hemophilia A: a cost-effectiveness analysis. Blood Adv 2018; 2(14): 1792–1798. doi: 10.1182/bloodadvances.2018021345.
- Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357(6): 535–44. doi: 10.1056/NEJMoa067659.
- Ellis AG, Mickle K, Herron-Smith S, et al. Spinraza® and Zolgensma® for spinal muscular atrophy: effectiveness and value. Final evidence report. Institue for Clinical and Economic Review, April 3, 2019. Available from https://34eyj51jerf417itp82ufdoe-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/ICER_SMA_Final_Evidence_Report_110220.pdf (accessed 28 April 2021).
- Pierce GF. Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns. Haemophilia 2020; 27 (S3): 103–13. doi: 10.1111/hae.14023.
- Pierce GF, Pasi KJ, Coffin D, et al.; Members of the WFH Gene Therapy Round Table Program Organizing Committee. Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table. Haemophilia 2020; 26(3): 443–449. doi: 10.1111/hae.13971.
- US Food & Drug Administration. FDA approves emicizumabkxwh for hemophilia A with or without factor VIII inhibitors. 14 December 2018. Available from https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-emicizumabkxwh-hemophilia-or-without-factor-viii-inhibitors (accessed 23 October 2019).
- HEMLIBRA®. HEMLIBRA® (emicizumab-kxwh) injection, for subcutaneous use [package insert]. Genentech, Inc., South San Francisco, CA, October 2018.
- Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: a current update. Blood Transfus 2018; 16(5): 457–461. doi: 10.2450/2018.0272-17.
- High KA, Roncarolo MG. Gene therapy. N Engl J Med 2019; 381(5): 455–464. doi: 10.1056/NEJMra1706910.
- Patel U, Boucher M, de Leseleuc L, Visintini S. Voretigene neparvovec: An emerging gene therapy for the treatment of inherited blindness. 2018 Mar 1. In: CADTH Issues in Emerging Health Technologies, Ottawa (ON) 2016–; 169.
- ZOLGENSMA®, ZOLGENSMA® (onasemnogene abeparvovecxioi) suspension for intravenous infusion [package insert]. AveXis, In., Bannockburn, IL, 2019.
- Brimble MA, Reiss UM, Nathwani AC, Davidoff AM. New and improved AAVenues: current status of hemophilia B gene therapy. Expert Opin Biol Ther 2016; 16(1): 79–92. doi: 10.1517/14712598.2015.1106475.
- High KA, Skinner MW. Cell phones and landlines: the impact of gene therapy on the cost and availability of treatment for hemophilia. Mol Ther 2011; 19(10): 1749–50. doi: 10.1038/mt.2011.203.
- Pipe SW. Gene therapy for hemophilia. Pediatr Blood Cancer 2018; 65(2). doi: 10.1002/pbc.26865.
- Iorio A, Skinner MW, Clearfield E, et al.; coreHEM panel. Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project. Haemophilia 2018; 24(4): e167–e172. doi: 10.1111/hae.13504.
- Sun HL, McIntosh KA, Squire SJ, et al. Patient powered prophylaxis: A 12-month study of individualized prophylaxis in adults with severe haemophilia A. Haemophilia 2017; 23(6): 877–883. doi: 10.1111/hae.13319.
- Arruda VR, Doshi BS, Samelson-Jones BJ. Novel approaches to hemophilia therapy: successes and challenges. Blood 2017; 130(21): 2251–2256. doi: 10.1182/blood-2017-08-742312.
- Pierce GF, Iorio A. Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes. Haemophilia 2018; 24 Suppl 6: 60–67. doi: 10.1111/hae.13489.
- American Society of Gene & Cell Therapy. Gene Therapy 101. Available from https://www.asgct.org/education/gene-therapy-101 (accessed 17 October 2019.
- Klifto K, Klifto C, Slover J. Current concepts of shared decision making in orthopedic surgery. Curr Rev Musculoskelet Med 2017; 10(2): 253–257. doi: 10.1007/s12178-017-9409-4.
- Nathwani AC, Davidoff AM, Tuddenham EGD. Gene therapy for hemophilia. Hematol Oncol Clin North Am 2017; 31(5): 853–868. doi: 10.1016/j.hoc.2017.06.011.
- Stoffman J, Andersson NG, Branchford B, et al. Common themes and challenges in hemophilia care: a multinational perspective. Hematology 2019; 24(1): 39–48. doi: 10.1080/10245332.2018.1505225.