Development of a patient-reported outcomes tool to monitor changes in joint health and wellbeing for young people with haemophilia B

1. Bertamino M, Riccardi F, Banov L, Svahn J, Molinari AC. Hemophilia care in the pediatric age. J Clin Med 2017; 6(5): 54. doi: 10.3390/jcm6050054. Bertamino M Riccardi F Banov L Svahn J Molinari AC. , Hemophilia care in the pediatric age . J Clin Med 2017 ; 6 ( 5 ): 54 . doi: 10.3390/jcm6050054 . Open DOISearch in Google Scholar

2. Iorio A, Stonebraker JS, Chambost H, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2019; 171(8): 540-546. doi: 10.7326/m19-1208. Iorio A Stonebraker JS Chambost H , Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries . Ann Intern Med 2019 ; 171 ( 8 ): 540 - 546 . doi: 10.7326/m19-1208 . Open DOISearch in Google Scholar

3. Knobe K, Berntorp E. Haemophilia and joint disease: pathophysiology, evaluation, and management. J Comorb 2011; 1: 51-59. doi: 10.15256/joc.2011.1.2. Knobe K Berntorp E. , Haemophilia and joint disease: pathophysiology, evaluation, and management . J Comorb 2011 ; 1 : 51 - 59 . doi: 10.15256/joc.2011.1.2 . Open DOISearch in Google Scholar

4. Kearney S, Raffini LJ, Pham TP, et al. Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII. Patient Prefer Adherence 2019; 13: 497-513. doi:10.2147/ppa.s196103. Kearney S Raffini LJ Pham TP , Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII . Patient Prefer Adherence 2019 ; 13 : 497 - 513 . doi: 10.2147/ppa.s196103 . Open DOISearch in Google Scholar

5. Manco-Johnson MJ, Soucie JM, Gill JC. Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. Blood 2017; 129(17): 2368-2374. doi:10.1182/blood-2016-02-683169. Manco-Johnson MJ Soucie JM Gill JC. , Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project . Blood 2017 ; 129 ( 17 ): 2368 - 2374 . doi: 10.1182/blood-2016-02-683169 . Open DOISearch in Google Scholar

6. Timmer MA, Pisters MF, de Kleijn P, et al. How do patients and professionals differentiate between intra-articular joint bleeds and acute flare-ups of arthropathy in patients with haemophilia? Haemophilia 2016; 22(3): 368-73. doi: 10.1111/hae.12858. Timmer MA Pisters MF de Kleijn P , How do patients and professionals differentiate between intra-articular joint bleeds and acute flare-ups of arthropathy in patients with haemophilia? Haemophilia 2016 ; 22 ( 3 ): 368 - 73 . doi: 10.1111/hae.12858 . Open DOISearch in Google Scholar

7. Rodriguez-Merchan EC. Hemophilic arthropathy: how to diagnose subclinical bleeding early and how to orthopedically treat a damaged joint. Expert Rev Hematol 2023; 16(9): 651-658. doi: 10.1080/17474086.2023.2232547. Rodriguez-Merchan EC. Hemophilic arthropathy: how to diagnose subclinical bleeding early and how to orthopedically treat a damaged joint . Expert Rev Hematol 2023 ; 16 ( 9 ): 651 - 658 . doi: 10.1080/17474086.2023.2232547 . Open DOISearch in Google Scholar

8. Gooding R, Thachil J, Alamelu J, Motwani J, Chowdary P. asymptomatic joint bleeding and joint health in hemophilia: a review of variables, methods, and biomarkers. J Blood Med 2021; 12: 209-220. doi: 10.2147/jbm.s304597. Gooding R Thachil J Alamelu J Motwani J Chowdary P. , asymptomatic joint bleeding and joint health in hemophilia: a review of variables, methods, and biomarkers . J Blood Med 2021 ; 12 : 209 - 220 . doi: 10.2147/jbm.s304597 . Open DOISearch in Google Scholar

9. APPG on Haemophilia and Contaminated Blood. APPG on Haemophilia and Contaminated Blood: Inquiry into Access to Treatment. APPG on Haemophilia and Contaminated Blood. Updated 2020. Available from https://haemophilia.org.uk/wp-content/uploads/pdf/APPG_report_web.pdf (accessed March 2022). APPG on Haemophilia and Contaminated Blood . APPG on Haemophilia and Contaminated Blood: Inquiry into Access to Treatment . APPG on Haemophilia and Contaminated Blood . Updated 2020. Available from https://haemophilia.org.uk/wp-content/uploads/pdf/APPG_report_web.pdf (accessed March 2022 ). Search in Google Scholar

10. National Health Service. Is my study research? Updated 10/2022. Available from https://www.hra-decisiontools.org.uk/research/ (accessed January 2024). National Health Service . Is my study research? Updated 10/2022. Available from https://www.hra-decisiontools.org.uk/research/ (accessed January 2024 ). Search in Google Scholar

11. Riley AW. Evidence that school-age children can self-report on their health. Ambul Pediatr 2004; 4(4 Suppl): 371-6. doi: 10.1367/a03-178r.1 Riley AW. , Evidence that school-age children can self-report on their health . Ambul Pediatr 2004 ; 4 ( 4 Suppl ): 371 - 6 . doi: 10.1367/a03-178r.1 Open DOISearch in Google Scholar

12. Varni JW, Limbers CA, Burwinkle TM. How young can children reliably and validly self-report their health-related quality of life?: an analysis of 8,591 children across age subgroups with the PedsQL 4.0 Generic Core Scales. Health QualLife Outcomes 2007; 5: 1. doi: 10.1186/1477-7525-5-1. Varni JW Limbers CA Burwinkle TM. , How young can children reliably and validly self-report their health-related quality of life?: an analysis of 8,591 children across age subgroups with the PedsQL 4.0 Generic Core Scales . Health QualLife Outcomes 2007 ; 5 : 1 . doi: 10.1186/1477-7525-5-1 . Open DOISearch in Google Scholar

13. Arbuckle R, Abetz-Webb L. "Not just little adults": qualitative methods to support the development of pediatric patient-reported outcomes. Patient 2013; 6(3): 143-159. doi: 10.1007/s40271-013-0022-3. Arbuckle R Abetz-Webb L. , " Not just little adults": qualitative methods to support the development of pediatric patient-reported outcomes . Patient 2013 ; 6 ( 3 ): 143 - 159 . doi: 10.1007/s40271-013-0022-3 . Open DOISearch in Google Scholar

14. Baek HJ, Park YS, Yoo KY, Cha JH, Kim YJ, Lee KS. Health-related quality of life of moderate and severe haemophilia patients: Results of the haemophilia-specific quality of life index in Korea. PLoS One 2020; 15(9): e0238686. doi: 10.1371/journal.pone.0238686. Baek HJ Park YS Yoo KY Cha JH Kim YJ Lee KS. , Health-related quality of life of moderate and severe haemophilia patients: Results of the haemophilia-specific quality of life index in Korea . PLoS One 2020 ; 15 ( 9 ): e0238686 . doi: 10.1371/journal.pone.0238686 . Open DOISearch in Google Scholar

15. Flood E, Pocoski J, Michaels LA, Bell JA, Valluri S, Sasane R. Illustrating the impact of mild/moderate and severe haemophilia on health-related quality of life: hypothesised conceptual models. Eur J Haematol 2014; 93 Suppl 75: 9-18. doi: 10.1111/ejh.12328. Flood E Pocoski J Michaels LA Bell JA Valluri S Sasane R. , Illustrating the impact of mild/moderate and severe haemophilia on health-related quality of life: hypothesised conceptual models . Eur J Haematol 2014 ; 93 Suppl 75 : 9 - 18 . doi: 10.1111/ejh.12328 . Open DOISearch in Google Scholar

16. Flood E, Pocoski J, Michaels LA, McCoy A, Beusterien K, Sasane R. Patient-reported experience of bleeding events in haemophilia. Eur J Haemotol 2014; 93(75): 19-28. doi: 10.1111/ejh.12329. Flood E Pocoski J Michaels LA McCoy A Beusterien K Sasane R. , Patient-reported experience of bleeding events in haemophilia . Eur J Haemotol 2014 ; 93 ( 75 ): 19 - 28 . doi: 10.1111/ejh.12329 . Open DOISearch in Google Scholar

17. Kempton CL, Recht M, Neff A, et al. Impact of pain and functional impairment in US adults with haemophilia: Patient-reported outcomes and musculoskeletal evaluation in the pain, functional impairment and quality of life (P-FiQ) study. Haemophilia 2018; 24(2): 261-270. doi: 10.1111/hae.13377. Kempton CL Recht M Neff A , Impact of pain and functional impairment in US adults with haemophilia: Patient-reported outcomes and musculoskeletal evaluation in the pain, functional impairment and quality of life (P-FiQ) study . Haemophilia 2018 ; 24 ( 2 ): 261 - 270 . doi: 10.1111/hae.13377 . Open DOISearch in Google Scholar

18. Stephensen D, Classey S, Hopper D. Exploring patient perceptions of ultrasound “selfies” to engage patient confidence in recognizing and managing musculoskeletal bleeding episodes. Haemophilia 2018; 24(S1)(P013): 32-135. doi: 10.1111/hae.13393. Stephensen D Classey S Hopper D. , Exploring patient perceptions of ultrasound “selfies” to engage patient confidence in recognizing and managing musculoskeletal bleeding episodes . Haemophilia 2018 ; 24 ( S1)(P013 ): 32 - 135 . doi: 10.1111/hae.13393 . Open DOISearch in Google Scholar

19. Tagliaferri A, Franchini M, Rivolta GF, Farace S, Quintavalle G, Coppola A. Pain assessment and management in haemophilia: A survey among Italian patients and specialist physicians. Haemophilia 2018; 24(5): 766-773. doi: 10.1111/hae.13600. Tagliaferri A Franchini M Rivolta GF Farace S Quintavalle G Coppola A. , Pain assessment and management in haemophilia: A survey among Italian patients and specialist physicians . Haemophilia 2018 ; 24 ( 5 ): 766 - 773 . doi: 10.1111/hae.13600 . Open DOISearch in Google Scholar

20. Khanom S, McDonagh JE, Briggs M, McBeth J. Characterizing pain flares in adolescent inflammatory and non-inflammatory musculoskeletal disorders: A qualitative study using an interpretative phenomenological approach. Eur J Pain 2020; 24(9): 1785-1796. doi: 10.1002/ejp.1626. Khanom S McDonagh JE Briggs M McBeth J. , Characterizing pain flares in adolescent inflammatory and non-inflammatory musculoskeletal disorders: A qualitative study using an interpretative phenomenological approach . Eur J Pain 2020 ; 24 ( 9 ): 1785 - 1796 . doi: 10.1002/ejp.1626 . Open DOISearch in Google Scholar

21. Betz CL, Mannino JE, Cleverley K, et al. Self-management for youth and young adults with special health needs: protocol for a scoping review of health care transition planning literature. JBI Evid Synth 2021; 19(7): 1682-1690. doi: 10.11124/jbies-20-00265. Betz CL Mannino JE Cleverley K , Self-management for youth and young adults with special health needs: protocol for a scoping review of health care transition planning literature . JBI Evid Synth 2021 ; 19 ( 7 ): 1682 - 1690 . doi: 10.11124/jbies-20-00265 . Open DOISearch in Google Scholar

22. Farre A, Wood V, McDonagh JE, Parr JR, Reape D, Rapley T. Health professionals' and managers' definitions of developmentally appropriate healthcare for young people: conceptual dimensions and embedded controversies. Arch Dis Child 2016; 101(7): 628-33. doi: 10.1136/archdischild-2015-309473. Farre A Wood V McDonagh JE Parr JR Reape D Rapley T. , Health professionals' and managers' definitions of developmentally appropriate healthcare for young people: conceptual dimensions and embedded controversies . Arch Dis Child 2016 ; 101 ( 7 ): 628 - 33 . doi: 10.1136/archdischild-2015-309473 . Open DOISearch in Google Scholar

23. Farre A, McDonagh JE. Helping health services to meet the needs of young people with chronic conditions: towards a developmental model for transition. Healthcare (Basel) 2017; 5(4): 77. doi: 10.3390/healthcare5040077. Farre A McDonagh JE. , Helping health services to meet the needs of young people with chronic conditions: towards a developmental model for transition . Healthcare (Basel) 2017 ; 5 ( 4 ): 77 . doi: 10.3390/healthcare5040077 . Open DOISearch in Google Scholar

24. Nyrop KA, Deal AM, Reeve BB, et al. Congruence of patient- and clinician-reported toxicity in women receiving chemotherapy for early breast cancer. Cancer 2020; 126(13): 3084-3093. doi: 10.1002/cncr.32898. Nyrop KA Deal AM Reeve BB , Congruence of patient- and clinician-reported toxicity in women receiving chemotherapy for early breast cancer . Cancer 2020 ; 126 ( 13 ): 3084 - 3093 . doi: 10.1002/cncr.32898 . Open DOISearch in Google Scholar

25. Xiao C, Polomano R, Bruner DW. Comparison between patient-reported and clinician-observed symptoms in oncology. Cancer Nurs 2013; 36(6): E1-e16. doi: 10.1097/NCC.0b013e318269040f. Xiao C Polomano R Bruner DW. , Comparison between patient-reported and clinician-observed symptoms in oncology . Cancer Nurs 2013 ; 36 ( 6 ): E1 - e16 . doi: 10.1097/NCC.0b013e318269040f . Open DOISearch in Google Scholar

26. Varni JW, Thissen D, Stucky BD, et al. Item-level informant discrepancies between children and their parents on the PROMIS(^®) pediatric scales. Qual Life Res 2015; 24(8): 1921-37. doi: 10.1007/s11136-014-0914-2. Varni JW Thissen D Stucky BD , Item-level informant discrepancies between children and their parents on the PROMIS(^®) pediatric scales . Qual Life Res 2015 ; 24 ( 8 ): 1921 - 37 . doi: 10.1007/s11136-014-0914-2 . Open DOISearch in Google Scholar

27. Tzelepis F, Sanson-Fisher RW, Zucca AC, Fradgley EA. Measuring the quality of patient-centered care: why patient-reported measures are critical to reliable assessment. Patient Prefer Adherence 2015; 9: 831-5. doi: 10.2147/ppa.S81975. Tzelepis F Sanson-Fisher RW Zucca AC Fradgley EA. , Measuring the quality of patient-centered care: why patient-reported measures are critical to reliable assessment . Patient Prefer Adherence 2015 ; 9 : 831 - 5 . doi: 10.2147/ppa.S81975 . Open DOISearch in Google Scholar

28. Øvretveit J, Zubkoff L, Nelson EC, Frampton S, Knudsen JL, Zimlichman E. Using patient-reported outcome measurement to improve patient care. Int J Qual Health Care 2017; 29(6): 874-879. doi: 10.1093/intqhc/mzx108. Øvretveit J Zubkoff L Nelson EC Frampton S Knudsen JL Zimlichman E. , Using patient-reported outcome measurement to improve patient care . Int J Qual Health Care 2017 ; 29 ( 6 ): 874 - 879 . doi: 10.1093/intqhc/mzx108 . Open DOISearch in Google Scholar

29. Bevans KB, Moon J, Carle AC, et al. Patient reported outcomes as indicators of pediatric health care quality. Acad Pediatr 2014; 14(5 Suppl): S90-6. doi: 10.1016/j.acap.2014.06.002. Bevans KB Moon J Carle AC , Patient reported outcomes as indicators of pediatric health care quality . Acad Pediatr 2014 ; 14 ( 5 Suppl ): S90 - 6 . doi: 10.1016/j.acap.2014.06.002 . Open DOISearch in Google Scholar

30. van Riel P, Alten R, Combe B, et al. Improving inflammatory arthritis management through tighter monitoring of patients and the use of innovative electronic tools. RMD Open 2016; 2(2): e000302. doi: 10.1136/rmdopen-2016-000302. van Riel P Alten R Combe B , Improving inflammatory arthritis management through tighter monitoring of patients and the use of innovative electronic tools . RMD Open 2016 ; 2 ( 2 ): e000302 . doi: 10.1136/rmdopen-2016-000302 . Open DOISearch in Google Scholar

31. Matza LS, Patrick DL, Riley AW, et al. Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force. Value Health 2013; 16(4): 461-479. doi: 10.1016/j.jval.2013.04.004. Matza LS Patrick DL Riley AW , Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force . Value Health 2013 ; 16 ( 4 ): 461 - 479 . doi: 10.1016/j.jval.2013.04.004 . Open DOISearch in Google Scholar

32. Krohe M, Lamoureux R, Banderas C, Turner-Bowker DM. Exploring and leveraging known resources to support pediatric clinical outcomes assessment (COA) development. Curr Pediatr Rev 2020; 16(3): 232-240. doi: 10.2174/1573396316666200331132259. Krohe M Lamoureux R Banderas C Turner-Bowker DM. , Exploring and leveraging known resources to support pediatric clinical outcomes assessment (COA) development . Curr Pediatr Rev 2020 ; 16 ( 3 ): 232 - 240 . doi: 10.2174/1573396316666200331132259 . Open DOISearch in Google Scholar