Long-standing inhibitors present many day-today difficulties for the affected individual; the unpredictability of bleeds, bleed management, pain and treatment efficacy all affect quality of life. This study explored these issues through focus groups of affected individuals aged 16-25 in the UK. The data from the focus groups was analysed for recurring themes, which were coded under three umbrella headings: ‘daily impact’, ‘education and future’ and ‘resilience and support’. Participants felt isolated through geography and being extra ‘rare’ within the bleeding disorders community; used pain as a gauge of bleed resolution, often without use of analgesia; described transition to adult care as particularly worrying; and explained the potential impact of living with an inhibitor on future career options. Peer-to-peer networking could provide emotional support for these young adults, who could also be role models for the future. Despite the burden of living with an inhibitor and its treatment, participants described a good quality of life from their own perspectives. With new therapeutic options for these individuals on the horizon, they look forward to a future with fewer bleeds and less pain.
1 Collins PW, Palmer BP, Chalmers EA, et al; UK Haemophilia Centre Doctors’ Organisation. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011. Blood 2014; 124: 3389-97.
2 Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med 2016; 374: 2054-64.
3 Collins P, Chalmers E, Alamelu J, et al. First-line immune tolerance induction for children with severe haemophilia A: a protocol from the UK Haemophilia Centre Doctors' Organisation Inhibitor and Paediatric Working Parties. Haemophilia. 2017 Sep;23(5):654-9. doi: 10.1111/hae.13264.
4 Dekoven M, Wisniewski T, Petrilla A, et al. Health-related quality of life in haemophilia patients with inhibitors and their caregivers. Haemophilia 2013; 19: 287-93.
5 DeKoven M, Karkare S, Lee WC, et al. Impact of haemophilia with inhibitors on caregiver burden in the United States. Haemophilia 2014; 20: 822-30.
6 Lindvall K, von Mackensen S, Elmståhl S, et al. Increased burden on caregivers of having a child with haemophilia complicated by inhibitors. Pediatr Blood Cancer. 2014 Apr;61(4):706-11. doi: 10.1002/pbc.24856.
7 Neufeld EJ, Recht M, Sabio H. Effect of acute bleeding on daily quality of life assessments in patients with congenital hemophilia with inhibitors and their families: observations from the dosing observational study in hemophilia. Value Health 2012; 15: 916-25.
8 Recht M, Neufeld EJ, Sharma VR, et al. Impact of acute bleeding on daily activities of patients with congenital hemophilia with inhibitors and their caregivers and families: observations from the Dosing Observational Study in Hemophilia (DOSE). Value Health 2014; 17: 744-8.
9 duTreil S. Physical and psychosocial challenges in adult hemophilia patients with inhibitors. J Blood Med 2014; 5: 115-22.
10 Hermans C, Auerswald G, Benson G. Outcome measures for adult and pediatric hemophilia patients with inhibitors. Eur J Haematol 2017; 99: 103-11.
11 Young G. Transitioning issues in adolescent to young adult hemophilia patients with inhibitors: an approach for a growing population. Blood Coagul Fibrinolysis 2010; 21 Suppl 1: S7-10.
12 Phillott A, Khair K, Loran C, et al. HaemophiliaLIVE: an ethnographic study on the impact of haemophilia on daily life. J Haem Pract 2014; 1(3): 14-20. doi: 10.17225/jhp00030.
13 Charmaz K. ‘Discovering’ chronic illness: using grounded theory. Soc Sci Med 1990; 30 (11): 1161-72.
14 Morfini M, Auerswald G, Kobelt RA,et al. Prophylactic treatment of haemophilia patients with inhibitors: clinical experience with recombinant factor VIIa in European Haemophilia Centres. Haemophilia 2007 Sep; 13(5): 502-7.
15 Negrier C, Voisin S, Baghaei F, et al; FEIBA PASS Study Group. Global Post-Authorization Safety Surveillance Study: realworld data on prophylaxis and on-demand treatment using FEIBA (an activated prothrombin complex concentrate). Blood Coagul Fibrinolysis 2016 Jul; 27(5): 551-6.
16 Young G, Escober MA, Pipe SW, Cooper DL. Safety and efficacy of recombinant activated coagulation factor VII in congenital hemophilia with inhibitors in the home treatment setting: a review of clinical studies and registries. Am J Hematol 2017 Sep:92 (9): 940-5.
17 Duncan N, Kronenberger W, Roberson C, Shapiro A. VERITASPro: a new measure of adherence to prophylactic regimens in haemophilia. Haemophilia 2010; 20(1): 39-43.
18 Van Os SB, Troop NA, Sullivan KR, Hart DP. Adherence to prophylaxis in adolescents and young adults with severe haemophilia: a quantitative study with patients. PLoS One 2017 Jan 19; 12(1): e0169880. doi: 10.1371/journal.pone.0169880.
19 Young G. Transitioning issues in adolescent to young adult hemophilia patients with inhibitors: an approach for a growing population. Blood Coag and Fibrinolysis 2010; 21 (Suppl 1): S7-10.
20 Khair K, Holland M, Bladen M, et al; SO-FIT Study Group. Study of physical function in adolescents with haemophilia: the SOFIT study. Haemophilia. 2017 Nov; 23(6): 918-25. doi: 10.1111/hae.13323.
21 Poti S, Palareti L, Emiliani F, Rodorigo G, Valdre L. The subjective experience of living with haemophilia in the transition from early adolescence to young adulthood: the effect of age and the therapeutic regimen. International Journal of Adolescence and Youth 2017. doi: 10.1080/02673843.2017.1299017.
22 Palareti L, Poti S, Cassis F, Emiliani F, Mayino D, Iorio A. Shared topics on the experience of people with haemophilia living in the UK and the USA and the influence of individual and contextual variables: results from the HERO qualitative study. Int J Qual Stud Health Well-being 2015; 10:28915. doi: 10.3402/qhw.v10.28915.
23 Khair K, Gibson F, Meerabeau L. ‘I can always rely on them’: the importance of social support for boys with haemophilia. J Haem Pract 2014; 1(1): 10-16. doi: 10.17225/jhp00005.
24 Limperg P, Peters M, Gibbons E, et al. Themes in daily life of adolescents and young adults with congenital bleeding disorders: a qualitative study. Haemophilia 2016; 22(4): e330-3. doi: 10.1111/hae.12961.
25 Garrino LE, Picco I, Fniguerra D, Rossi P, Roccatello D. Living with and treating rare diseases: experience of patients and professional health care providers. Qual Health Res 2015; 25(5): 636-51. doi: 10.1177/1049732315570116.
26 Brand B, Dunn S, Kulkarni R. Challenges in the management of haemophilia on transition from adolescence to adulthood. Eur J Haematol 2015 Dec;95 Suppl 81: 30-5. doi: 10.1111/ejh.12582.
27 Khair K, Holland M, Carrington S. Social networking for adolescents with severe haemophilia. Haemophilia 2012 May; 18(3): e290-6. doi: 10.1111/j.1365-2516.2011.02689.x.
28 European Haemophilia Consortium. The European Inhibitor Network. Available from https://inhibitor.ehc.eu (accessed 28 January 2018).
29 Lincoln YS, Garuba EG. Naturalistic Inquiry. Newbury Park, Ca: SAGE; 1985.