Personalising haemophilia management with shared decision making

Leonard A Valentino; Victor Blanchette; Claude Negrier; Brian O’Mahony; Val Bias; Thomas Sannié; Mark W Skinner

doi:10.17225/jhp00178

Abstract

Abstract

The current standard of care for treating people with haemophilia (PWH) in the developed world is prophylaxis with regular infusions of clotting factor concentrates. Gene therapy is being investigated as a new treatment paradigm for haemophilia and if approved would potentially eliminate the need for chronic, burdensome infusions. In recent years, shared decision making (SDM) has become increasingly common in patient care settings. SDM is a stepwise process that relies on reciprocal information sharing between the practitioner and patient, resulting in health care decisions stemming from the informed preferences of both parties. SDM represents a departure from the traditional, paternalistic clinical model where the practitioner drives the treatment decision and the patient passively defers to this decision. As the potential introduction of gene therapy in haemophilia may transform the current standard of care, and impact disease management and goals in unique ways, both practitioners and PWH may find their knowledge tested when considering the appropriate use of a novel technology. Therefore, it is incumbent upon haemophilia practitioners to foster an open, trusting, and supportive relationship with their patients, while PWH and their caregivers must be knowledgeable and feel empowered to participate in the decision making process to achieve truly shared treatment decisions.

Article

View Full Article

References

1. Barry MJ, Edgman-Levitan S. Shared decision making--pinnacle of patient-centered care. N Engl J Med 2012; 366(9): 780–1. doi: https://doi.org/10.1056/NEJMp1109283.
2. 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: https://doi.org/10.1097/ACM.0000000000000603.
3. 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: https://doi.org/10.1177/2040620718784830.
4. 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: https://doi.org/10.1046/j.1369-6513.2003.00244.x.
5. Berger Z. Navigating the unknown: shared decision-making in the face of uncertainty. J Gen Intern Med 2015; 30(5): 675–8. doi: https://doi.org/10.1007/s11606-014-3074-8.
6. 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: https://doi.org/10.1111/j.1369-7625.2006.00359.x.
7. 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: https://doi.org/10.1007/s11606-012-2077-6.
8. 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: https://doi.org/10.3238/arztebl.2015.0665.
9. Matlock DD, Spatz ES. Design and testing of tools for shared decision making. Circ Cardiovasc Qual Outcomes 2014; 7(3): 487–92. doi: https://doi.org/10.1161/CIRCOUTCOMES.113.000289.
10. Drug and Therapeutics Bulletin. An introduction to patient decision aids. BMJ 2013; 347: f4147. doi: https://doi.org/10.1136/bmj.f4147.
11. 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).
12. 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: https://doi.org/10.1016/j.pec.2016.11.021.
13. Dolan JG. Shared decision-making--transferring research into practice: the Analytic Hierarchy Process (AHP). Patient Educ Couns 2008; 73(3): 418–25. doi: https://doi.org/10.1016/j.pec.2008.07.032.
14. 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: https://doi.org/10.1177/0272989X14551638.
15. Beers E, Nilsen ML, Johnson JT. The role of patients: shared decision-making. Otolaryngol Clin North Am 2017; 50(4): 689–708. doi: https://doi.org/10.1016/j.otc.2017.03.006.
16. 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: https://doi.org/10.1186/s40064-016-3086-4.
17. 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: https://doi.org/10.1136/bmj.j1744.
18. Epstein RM. Whole mind and shared mind in clinical decision-making. Patient Educ Couns 2013; 90(2): 200–6. doi: https://doi.org/10.1016/j.pec.2012.06.035.
19. 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: https://doi.org/10.1016/s0749-3797(99)00097-5.
20. Elwyn G, Frosch DL, Kobrin S. Implementing shared decision-making: consider all the consequences. Implementation Sci 2015; 11: 114. doi: https://doi.org/10.1186/s13012-016-0480-9.
21. 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: https://doi.org/10.1016/j.pec.2008.07.054.
22. 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).
23. 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: https://doi.org/10.1111/j.1365-2516.2012.02909.x.
24. Collins PW. Personalized prophylaxis. Haemophilia 2012; 18(Suppl 4): 131–5. doi: https://doi.org/10.1111/j.1365-2516.2012.02838.x.
25. Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia 2014; 20(5): 607–15. doi: https://doi.org/10.1111/hae.12438.
26. 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: https://doi.org/10.2217/pme.15.6.
27. 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: https://doi.org/10.1111/j.1538-7836.2011.04611.x.
28. 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: https://doi.org/10.1182/blood-2013-10-529974.
29. Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood 2019; 133(5): 407–14. doi: https://doi.org/10.1182/blood-2018-07-820720.
30. Rodriguez-Merchan EC, Valentino LA. Emicizumab: Review of the literature and critical appraisal. Haemophilia 2019; 25(1): 11–20. doi: https://doi.org/10.1111/hae.13641.
31. Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia 2001; 7(4): 392–6. doi: https://doi.org/10.1046/j.1365-2516.2001.00534.x.
32. 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: https://doi.org/10.1111/j.1365-2516.2008.01843.x.
33. 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: https://doi.org/10.1111/j.1365-2516.2007.01526.x.
34. 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: https://doi.org/10.1111/j.1365-2516.2012.02756.x.
35. Machin N, Ragni MV, Smith KJ. Gene therapy in hemophilia A: a cost-effectiveness analysis. Blood Adv 2018; 2(14): 1792–1798. doi: https://doi.org/10.1182/bloodadvances.2018021345.
36. 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: https://doi.org/10.1056/NEJMoa067659.
37. 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).
38. Pierce GF. Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns. Haemophilia 2020; 27 (S3): 103–13. doi: https://doi.org/10.1111/hae.14023.
39. 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: https://doi.org/10.1111/hae.13971.
40. 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).
41. HEMLIBRA^®. HEMLIBRA^® (emicizumab-kxwh) injection, for subcutaneous use [package insert]. Genentech, Inc., South San Francisco, CA, October 2018.
42. Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: a current update. Blood Transfus 2018; 16(5): 457–461. doi: https://doi.org/10.2450/2018.0272-17.
43. High KA, Roncarolo MG. Gene therapy. N Engl J Med 2019; 381(5): 455–464. doi: https://doi.org/10.1056/NEJMra1706910.
44. 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.
45. ZOLGENSMA^®, ZOLGENSMA^® (onasemnogene abeparvovecxioi) suspension for intravenous infusion [package insert]. AveXis, In., Bannockburn, IL, 2019.
46. 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: https://doi.org/10.1517/14712598.2015.1106475.
47. 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: https://doi.org/10.1038/mt.2011.203.
48. Pipe SW. Gene therapy for hemophilia. Pediatr Blood Cancer 2018; 65(2). doi: https://doi.org/10.1002/pbc.26865.
49. 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: https://doi.org/10.1111/hae.13504.
50. 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: https://doi.org/10.1111/hae.13319.
51. Arruda VR, Doshi BS, Samelson-Jones BJ. Novel approaches to hemophilia therapy: successes and challenges. Blood 2017; 130(21): 2251–2256. doi: https://doi.org/10.1182/blood-2017-08-742312.
52. 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: https://doi.org/10.1111/hae.13489.
53. American Society of Gene & Cell Therapy. Gene Therapy 101. Available from https://www.asgct.org/education/gene-therapy-101 (accessed 17 October 2019.
54. 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: https://doi.org/10.1007/s12178-017-9409-4.
55. Nathwani AC, Davidoff AM, Tuddenham EGD. Gene therapy for hemophilia. Hematol Oncol Clin North Am 2017; 31(5): 853–868. doi: https://doi.org/10.1016/j.hoc.2017.06.011.
56. 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: https://doi.org/10.1080/10245332.2018.1505225.

PDF Download

Download PDF

Open in full-page viewer

Authors

Leonard A Valentino

lvalentino@hemophilia.org

Rush University, Chicago, IL ; National Hemophilia Foundation, New York, NY, USA .
Victor Blanchette

Department of Pediatrics, University of Toronto and Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada
Claude Negrier

Unité d’Hemostase Clinique, Hôpital, Louis Pradel, Hospices Civils de Lyon, Université Claude Bernard, Lyon, France
Brian O’Mahony

Irish Haemophilia Society, Dublin, Ireland
Val Bias

Former CEO, National Hemophilia Foundation, New York, NY, USA
Thomas Sannié

Association française des hémophiles, Paris, France
Mark W Skinner

Institute for Policy Advancement Ltd, Washington, DC, USA ; McMaster University, Hamilton, Ontario, Canada