{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,6,15]],"date-time":"2025-06-15T04:02:11Z","timestamp":1749960131702,"version":"3.41.0"},"reference-count":27,"publisher":"Springer Science and Business Media LLC","issue":"7","license":[{"start":{"date-parts":[[2025,5,23]],"date-time":"2025-05-23T00:00:00Z","timestamp":1747958400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,5,23]],"date-time":"2025-05-23T00:00:00Z","timestamp":1747958400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["PharmacoEconomics"],"published-print":{"date-parts":[[2025,7]]},"DOI":"10.1007\/s40273-025-01508-w","type":"journal-article","created":{"date-parts":[[2025,5,23]],"date-time":"2025-05-23T18:49:15Z","timestamp":1748026155000},"page":"761-764","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Anchoring the Hemophilia Joint Health Score for Conventional Value Assessment in the Care of People Living with Severe Hemophilia A"],"prefix":"10.1007","volume":"43","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4430-1860","authenticated-orcid":false,"given":"Satoko","family":"Ito","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Manraj","family":"Sra","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"George","family":"Goshua","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2025,5,23]]},"reference":[{"issue":"8","key":"1508_CR1","doi-asserted-by":"publisher","first-page":"540","DOI":"10.7326\/M19-1208","volume":"171","author":"A Iorio","year":"2019","unstructured":"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\u20136. https:\/\/doi.org\/10.7326\/M19-1208.","journal-title":"Ann Intern Med"},{"key":"1508_CR2","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1111\/hae.14046","volume":"26","author":"A Srivastava","year":"2020","unstructured":"Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the management of hemophilia. Haemophilia. 2020;26:1\u2013158. https:\/\/doi.org\/10.1111\/hae.14046.","journal-title":"Haemophilia"},{"issue":"17","key":"1508_CR3","doi-asserted-by":"publisher","first-page":"1484","DOI":"10.1182\/blood.2019001292","volume":"135","author":"E Seth Chhabra","year":"2020","unstructured":"Seth Chhabra E, Liu T, Kulman J, et al. BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice. Blood. 2020;135(17):1484\u201396. https:\/\/doi.org\/10.1182\/blood.2019001292.","journal-title":"Blood"},{"issue":"21","key":"1508_CR4","doi-asserted-by":"publisher","first-page":"2970","DOI":"10.1182\/blood.2020009197","volume":"137","author":"JR Fuller","year":"2021","unstructured":"Fuller JR, Knockenhauer KE, Leksa NC, Peters RT, Batchelor JD. Molecular determinants of the factor VIII\/von Willebrand factor complex revealed by BIVV001 cryo-electron microscopy. Blood. 2021;137(21):2970\u201380. https:\/\/doi.org\/10.1182\/blood.2020009197.","journal-title":"Blood"},{"issue":"7","key":"1508_CR5","doi-asserted-by":"publisher","first-page":"1844","DOI":"10.1016\/j.jtha.2024.04.010","volume":"22","author":"C Hermans","year":"2024","unstructured":"Hermans C, Pierce GF. Ultra-Long factor VIII: a major step forward toward a hemophilia-free mind. J Thromb Haemost. 2024;22(7):1844\u20136. https:\/\/doi.org\/10.1016\/j.jtha.2024.04.010.","journal-title":"J Thromb Haemost"},{"issue":"4","key":"1508_CR6","doi-asserted-by":"publisher","first-page":"310","DOI":"10.1056\/NEJMoa2209226","volume":"388","author":"A von Drygalski","year":"2023","unstructured":"von Drygalski A, Chowdary P, Kulkarni R, et al. Efanesoctocog alfa prophylaxis for patients with severe hemophilia A. N Engl J Med. 2023;388(4):310\u20138. https:\/\/doi.org\/10.1056\/NEJMoa2209226.","journal-title":"N Engl J Med"},{"issue":"3","key":"1508_CR7","doi-asserted-by":"publisher","first-page":"235","DOI":"10.1056\/NEJMoa2312611","volume":"391","author":"L Malec","year":"2024","unstructured":"Malec L, Peyvandi F, Chan AKC, et al. Efanesoctocog alfa prophylaxis for children with severe hemophilia A. N Engl J Med. 2024;391(3):235\u201346. https:\/\/doi.org\/10.1056\/NEJMoa2312611.","journal-title":"N Engl J Med"},{"issue":"2","key":"1508_CR8","doi-asserted-by":"publisher","first-page":"179","DOI":"10.1007\/s41669-024-00540-4","volume":"9","author":"AA El-Sayed","year":"2025","unstructured":"El-Sayed AA, Bolous NS. Economic burden of haemophilia from a societal perspective: a scoping review. Pharmacoecon Open. 2025;9(2):179\u2013205. https:\/\/doi.org\/10.1007\/s41669-024-00540-4.","journal-title":"Pharmacoecon Open."},{"issue":"7","key":"1508_CR9","doi-asserted-by":"publisher","first-page":"632","DOI":"10.18553\/jmcp.2018.24.7.632","volume":"24","author":"T Thorat","year":"2018","unstructured":"Thorat T, Neumann PJ, Chambers JD. Hemophilia burden of disease: a systematic review of the cost-utility literature for hemophilia. J Manag Care Spec Pharm. 2018;24(7):632\u201342. https:\/\/doi.org\/10.18553\/jmcp.2018.24.7.632.","journal-title":"J Manag Care Spec Pharm"},{"issue":"3","key":"1508_CR10","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1007\/s40273-017-0588-z","volume":"36","author":"PA Cortesi","year":"2018","unstructured":"Cortesi PA, D\u2019Angiolella LS, Lafranconi A, Micale M, Cesana G, Mantovani LG. Modern Treatments of haemophilia: review of cost-effectiveness analyses and future directions. Pharmacoeconomics. 2018;36(3):263\u201384. https:\/\/doi.org\/10.1007\/s40273-017-0588-z.","journal-title":"Pharmacoeconomics"},{"issue":"1","key":"1508_CR11","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1080\/13696998.2024.2444157","volume":"28","author":"N Meier","year":"2025","unstructured":"Meier N, Ammann D, Pletscher M, Probst J, Schwenkglenks M. Systematic review of cost-effectiveness modelling studies for haemophilia. J Med Econ. 2025;28(1):89\u2013104. https:\/\/doi.org\/10.1080\/13696998.2024.2444157.","journal-title":"J Med Econ"},{"issue":"7543","key":"1508_CR12","doi-asserted-by":"publisher","first-page":"699","DOI":"10.1136\/bmj.38737.607558.80","volume":"332","author":"CM Bell","year":"2006","unstructured":"Bell CM, Urbach DR, Ray JG, et al. Bias in published cost effectiveness studies: systematic review. BMJ. 2006;332(7543):699\u2013703. https:\/\/doi.org\/10.1136\/bmj.38737.607558.80.","journal-title":"BMJ"},{"key":"1508_CR13","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-025-01484-1","author":"S Hirniak","year":"2025","unstructured":"Hirniak S, Edginton AN, Iorio A, Wong WWL. A Hemophilia Joint Health Score-based model for the economic evaluation of hemophilia A prophylaxis interventions. Pharmacoeconomics. 2025. https:\/\/doi.org\/10.1007\/s40273-025-01484-1.","journal-title":"Pharmacoeconomics"},{"key":"1508_CR14","doi-asserted-by":"publisher","DOI":"10.7326\/ANNALS-24-02749","author":"S Ito","year":"2025","unstructured":"Ito S, Potnis KC, Harvey JP, et al. Prophylactic weekly efanesoctocog alfa versus standard-care factor VIII in people living with severe hemophilia A\u202f: a cost-effectiveness analysis. Ann Intern Med. 2025. https:\/\/doi.org\/10.7326\/ANNALS-24-02749.","journal-title":"Ann Intern Med"},{"key":"1508_CR15","unstructured":"National Institute for Health and Care Excellence (NICE). Efanesoctocog alfa for treating and preventing bleeding episodes in haemophilia A in people 2 years and over. https:\/\/www.nice.org.uk\/guidance\/ta1051. Accessed 24 Apr 2025."},{"issue":"5","key":"1508_CR16","doi-asserted-by":"publisher","first-page":"501","DOI":"10.1080\/13696998.2020.1721508","volume":"23","author":"K Cook","year":"2020","unstructured":"Cook K, Forbes SP, Adamski K, Ma JJ, Chawla A, Garrison LP Jr. Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A. J Med Econ. 2020;23(5):501\u201312. https:\/\/doi.org\/10.1080\/13696998.2020.1721508.","journal-title":"J Med Econ"},{"key":"1508_CR17","unstructured":"Rind DM WS, Agboola F, Herron-Smith S, Quach D, Chapman R, , Pearson SD BP. Valoctocogene roxaparvovec and emicizumab for hemophilia A: effectiveness and value; final report. November 20, 2020. https:\/\/icer-review.org\/material\/hemophilia-a-update-final-evidence-report\/. Accessed 24 Apr 2025."},{"key":"1508_CR18","doi-asserted-by":"publisher","first-page":"153","DOI":"10.1097\/00003086-198006000-00018","volume":"149","author":"H Pettersson","year":"1980","unstructured":"Pettersson H, Ahlberg A, Nilsson IM. A radiologic classification of hemophilic arthropathy. Clin Orthop Relat Res. 1980;149:153\u20139.","journal-title":"Clin Orthop Relat Res"},{"issue":"5","key":"1508_CR19","doi-asserted-by":"publisher","first-page":"518","DOI":"10.1111\/j.1365-2516.2006.01312.x","volume":"12","author":"P Hilliard","year":"2006","unstructured":"Hilliard P, Funk S, Zourikian N, et al. Hemophilia joint health score reliability study. Haemophilia. 2006;12(5):518\u201325. https:\/\/doi.org\/10.1111\/j.1365-2516.2006.01312.x.","journal-title":"Haemophilia"},{"issue":"1","key":"1508_CR20","doi-asserted-by":"publisher","first-page":"e1","DOI":"10.1111\/hae.13631","volume":"25","author":"SC Gouw","year":"2019","unstructured":"Gouw SC, Timmer MA, Srivastava A, et al. Measurement of joint health in persons with haemophilia: a systematic review of the measurement properties of haemophilia-specific instruments. Haemophilia. 2019;25(1):e1\u201310. https:\/\/doi.org\/10.1111\/hae.13631.","journal-title":"Haemophilia"},{"issue":"2","key":"1508_CR21","doi-asserted-by":"publisher","first-page":"223","DOI":"10.1002\/acr.20353","volume":"63","author":"BM Feldman","year":"2011","unstructured":"Feldman BM, Funk SM, Bergstrom BM, et al. Validation of a new pediatric joint scoring system from the International Hemophilia Prophylaxis Study Group: validity of the hemophilia joint health score. Arthritis Care Res (Hoboken). 2011;63(2):223\u201330. https:\/\/doi.org\/10.1002\/acr.20353.","journal-title":"Arthritis Care Res (Hoboken)."},{"issue":"3","key":"1508_CR22","doi-asserted-by":"publisher","first-page":"e180","DOI":"10.1111\/hae.13231","volume":"23","author":"M Osooli","year":"2017","unstructured":"Osooli M, Steen Carlsson K, Baghaei F, et al. The association between health utility and joint status among people with severe haemophilia A: findings from the KAPPA register. Haemophilia. 2017;23(3):e180\u20137. https:\/\/doi.org\/10.1111\/hae.13231.","journal-title":"Haemophilia"},{"key":"1508_CR23","unstructured":"Canadian Hemophilia Society. Health Canada approves Altuviiio. https:\/\/chscontact.ca\/health-canada-approves-altuviiio\/. Accessed 24 Apr 2025."},{"issue":"5","key":"1508_CR24","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12567","volume":"5","author":"E Krumb","year":"2021","unstructured":"Krumb E, Hermans C. Living with a \u201chemophilia-free mind\u201d - The new ambition of hemophilia care? Res Pract Thromb Haemost. 2021;5(5): e12567. https:\/\/doi.org\/10.1002\/rth2.12567.","journal-title":"Res Pract Thromb Haemost."},{"issue":"4","key":"1508_CR25","doi-asserted-by":"publisher","first-page":"951","DOI":"10.1111\/hae.14807","volume":"29","author":"C Hermans","year":"2023","unstructured":"Hermans C, Pierce GF. Towards achieving a haemophilia-free mind. Haemophilia. 2023;29(4):951\u20133. https:\/\/doi.org\/10.1111\/hae.14807.","journal-title":"Haemophilia"},{"issue":"13","key":"1508_CR26","doi-asserted-by":"publisher","first-page":"1709","DOI":"10.1182\/blood.2020009603","volume":"137","author":"CL Kempton","year":"2021","unstructured":"Kempton CL. Prophylaxis in hemophilia: how much is enough? Blood. 2021;137(13):1709\u201311. https:\/\/doi.org\/10.1182\/blood.2020009603.","journal-title":"Blood"},{"issue":"9","key":"1508_CR27","doi-asserted-by":"publisher","first-page":"e689","DOI":"10.1016\/S2352-3026(22)00209-5","volume":"9","author":"GF Pierce","year":"2022","unstructured":"Pierce GF, Adediran M, Diop S, et al. Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years. Lancet Haematol. 2022;9(9):e689\u201397. https:\/\/doi.org\/10.1016\/S2352-3026(22)00209-5.","journal-title":"Lancet Haematol."}],"container-title":["PharmacoEconomics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40273-025-01508-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40273-025-01508-w\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40273-025-01508-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,6,14]],"date-time":"2025-06-14T09:48:28Z","timestamp":1749894508000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40273-025-01508-w"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,5,23]]},"references-count":27,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2025,7]]}},"alternative-id":["1508"],"URL":"https:\/\/doi.org\/10.1007\/s40273-025-01508-w","relation":{},"ISSN":["1170-7690","1179-2027"],"issn-type":[{"type":"print","value":"1170-7690"},{"type":"electronic","value":"1179-2027"}],"subject":[],"published":{"date-parts":[[2025,5,23]]},"assertion":[{"value":"4 May 2025","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"23 May 2025","order":2,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"G.G. is supported by the NOMIS Foundation, Frederick A. DeLuca Foundation, Yale Cancer Center, Yale Bunker Endowment, and the National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI) grant 1\u2009K01 HL175220, and NIH research grant CA-016359 from the National Cancer Institute. The contents of this article are solely the responsibility of the authors and do not necessarily represent the official views of the funding sources.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"None.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Availability of data and material"}}]}}