{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T23:16:54Z","timestamp":1784762214040,"version":"3.55.0"},"reference-count":32,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,6,22]],"date-time":"2021-06-22T00:00:00Z","timestamp":1624320000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100014805","name":"Roche Italia","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014805","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2021,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>A shift from a standard to a personalized prophylaxis has been increasingly adopted in patients with severe haemophilia A (SHA). This approach has raised the likelihood of a significant variability in the prophylactic approaches and the relative Factor VIII (FVIII) consumptions. The aim of our study was to assess the treatment variability of SHA patients without inhibitors and on prophylaxis regimen in Italy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Material and methods<\/jats:title><jats:p>Data reported in the National Registry of Congenital Coagulopathies (NRCC) were analysed to assess treatment distribution within SHA patients without inhibitors, focussing on FVIII consumption in 2017, associated with prophylaxis regimen. The analysis was stratified based on age groups and Italian regions to describe the variability of FVIII consumption in Italy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In 2017, the Registry reported the therapeutic plans of 1068 SHA patients without inhibitors on prophylaxis. The mean (95% CI) individual consumption ranges from 123\u00a0127\u00a0IU (99\u00a0736\u2010146\u00a0518) in the age group 0\u20106\u00a0years to 345\u00a0000\u00a0IU (336\u00a0000\u2010354\u00a0000) in the age group &gt;20\u00a0years. A significant FVIII consumption variability was identified within the adult population. Regions with less than 50 patients reported the higher variability in mean FVIII consumption per patient\u2010year within the different age groups. Similar difference in FVIII consumption variability was reported also in the age groups comparing \u201clow,\u201d \u201cmiddle\u201d and \u201chigh\u201d patient volume regions.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>A reliable estimation of FVIII consumption for patients\u2019 treatment is necessary to manage and plan the appropriate budget and keep treatment's costs affordable. However, without the implementation of a methodology aiming to assess the overall value produced by these FVIII consumptions, the scenario will keep driven by FVIII consumptions, its costs and the budget available. An effort by haemophilic community, haemophilia treatment centres and institutions is required to develop and share this cultural shift in improving haemophilia management and assessment.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.13676","type":"journal-article","created":{"date-parts":[[2021,6,10]],"date-time":"2021-06-10T19:25:56Z","timestamp":1623353156000},"page":"408-415","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Variability of treatment modalities and intensity in patients with severe haemophilia A on prophylaxis: Results from the Italian national registry"],"prefix":"10.1111","volume":"107","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5241-4473","authenticated-orcid":false,"given":"Paolo Angelo","family":"Cortesi","sequence":"first","affiliation":[{"name":"Value\u2010Based Healthcare Unit IRCCS MultiMedica Sesto San Giovanni Italy"},{"name":"Research Centre on Public Health (CESP) University of Milan\u2010Bicocca Monza Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3135-0735","authenticated-orcid":false,"given":"Adele","family":"Giampaolo","sequence":"additional","affiliation":[{"name":"Department of Oncology and Molecular Medicine Italian National Institute of Health Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Abbonizio","sequence":"additional","affiliation":[{"name":"Department of Oncology and Molecular Medicine Italian National Institute of Health Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angelo Claudio","family":"Molinari","sequence":"additional","affiliation":[{"name":"Regional Reference Center for Hemorrhagic Diseases Giannina Gaslini Institute Genova Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4973-1317","authenticated-orcid":false,"given":"Giancarlo","family":"Castaman","sequence":"additional","affiliation":[{"name":"Center for Bleeding Disorders and Coagulation Careggi University Hospital Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mauro","family":"Biffoni","sequence":"additional","affiliation":[{"name":"Department of Oncology and Molecular Medicine Italian National Institute of Health Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorenzo Giovanni","family":"Mantovani","sequence":"additional","affiliation":[{"name":"Value\u2010Based Healthcare Unit IRCCS MultiMedica Sesto San Giovanni Italy"},{"name":"Research Centre on Public Health (CESP) University of Milan\u2010Bicocca Monza Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,6,22]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_10_3_1","first-page":"167","article-title":"Principles of treatment and update of recommendations for the management of haemophilia and congenital bleeding disorders in Italy [published correction appears in Blood Transfus","volume":"13","author":"Rocino A","year":"2015","journal-title":"Blood Transfus"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02909.x"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.217133"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1586\/17474086.2015.1002465"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2018.01.001"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13935"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.15084"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12253"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1080\/16078454.2019.1687798"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-08-872291"},{"issue":"1","key":"e_1_2_10_13_1","first-page":"58","article-title":"New data from the Italian National Register of Congenital Coagulopathies, 2016 Annual Survey","volume":"18","author":"Abbonizio F","year":"2020","journal-title":"Blood Transfus"},{"issue":"4","key":"e_1_2_10_14_1","first-page":"437","article-title":"Italian Association of Haemophilia Centres. 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