{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T08:16:07Z","timestamp":1740125767157,"version":"3.37.3"},"reference-count":33,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2018,3,1]],"date-time":"2018-03-01T00:00:00Z","timestamp":1519862400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100005744","name":"Lastentautien Tutkimuss\u00e4\u00e4ti\u00f6","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100005744","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2018,5]]},"abstract":"<jats:sec><jats:title>Aim<\/jats:title><jats:p>For previously untreated patients (<jats:styled-content style=\"fixed-case\">PUP<\/jats:styled-content>s) with severe haemophilia A in Finland for the past 2 decades, the standard practice has been to start early primary prophylaxis. We evaluated the long\u2010term clinical outcomes and costs of treatment with high\u2010dose prophylaxis in <jats:styled-content style=\"fixed-case\">PUP<\/jats:styled-content>s from birth to adolescence, including immune tolerance induction (<jats:styled-content style=\"fixed-case\">ITI<\/jats:styled-content>).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>From the medical records of all <jats:styled-content style=\"fixed-case\">PUP<\/jats:styled-content>s born between June 1994 and May 2013 in Finland, we retrospectively extracted data on clinical outcomes and healthcare use. Using linear mixed models, we analysed longitudinal clinical outcome data. To analyse skewed cost data, including zero costs, we applied hurdle regression.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>All 62 patients received early regular prophylaxis; totally, they have had treatment for nearly 700 patient\u2010years. The median age of starting home treatment was 1.1\u00a0years. The mean (<jats:styled-content style=\"fixed-case\">SD<\/jats:styled-content>) annual treatment costs (\u20ac\u00a0per\u00a0kg) were 4391\u20ac (3852). For ages 1\u20103, <jats:styled-content style=\"fixed-case\">ITI<\/jats:styled-content> comprised over half of the costs; in other groups, prophylactic <jats:styled-content style=\"fixed-case\">FVIII<\/jats:styled-content> treatment dominated. With these high costs, however, clinical outcomes were desirable; median (<jats:styled-content style=\"fixed-case\">IQR<\/jats:styled-content>) <jats:styled-content style=\"fixed-case\">ABR<\/jats:styled-content> was low at 0.19 (0.07\u20100.46) and so was <jats:styled-content style=\"fixed-case\">AJBR<\/jats:styled-content> at 0.06 (0\u20100.24). Thirteen (21%) patients developed a clinically significant inhibitor, 10 (16%) with a high titre. All <jats:styled-content style=\"fixed-case\">ITI<\/jats:styled-content>s were successful. The mean costs for <jats:styled-content style=\"fixed-case\">ITI<\/jats:styled-content> were 383\u00a0448\u20ac (259\u00a0085). The expected <jats:styled-content style=\"fixed-case\">ITI<\/jats:styled-content> payback period was 1.81 (95% <jats:styled-content style=\"fixed-case\">CI<\/jats:styled-content> 0.62\u201012.12) years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Early high\u2010dose prophylaxis leads to excellent long\u2010term clinical outcomes, and early childhood <jats:styled-content style=\"fixed-case\">ITI<\/jats:styled-content> therapy seems to turn cost\u2010neutral generally already in 2\u00a0years.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.13447","type":"journal-article","created":{"date-parts":[[2018,3,1]],"date-time":"2018-03-01T12:31:35Z","timestamp":1519907495000},"page":"436-444","source":"Crossref","is-referenced-by-count":6,"title":["Long\u2010term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A: A nationwide real\u2010world study with 700 person\u2010years"],"prefix":"10.1111","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2831-9518","authenticated-orcid":false,"given":"K.","family":"Veps\u00e4l\u00e4inen","sequence":"first","affiliation":[{"name":"Department of Paediatrics Kuopio University Hospital  Kuopio Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P.","family":"Riikonen","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Kuopio University Hospital  Kuopio Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R.","family":"Lassila","sequence":"additional","affiliation":[{"name":"Coagulation Disorders Unit Department of Haematology Comprehensive Cancer Centre European Haemophilia Comprehensive Care Centre Helsinki University Hospital University of Helsinki  Helsinki Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"Arola","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Tampere University Central Hospital  Tampere Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P.","family":"Huttunen","sequence":"additional","affiliation":[{"name":"Children's Hospital Helsinki University Hospital European Haemophilia Comprehensive Care Centre  Helsinki Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P.","family":"L\u00e4hteenm\u00e4ki","sequence":"additional","affiliation":[{"name":"Department of Paediatrics and Adolescent Medicine Turku University Central Hospital University of Turku  Turku Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"M\u00f6tt\u00f6nen","sequence":"additional","affiliation":[{"name":"Department of Children and Adolescents PEDEGO Research Centre, and Medical Research Centre Oulu Oulu University Hospital University of Oulu  Oulu Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"T.","family":"Selander","sequence":"additional","affiliation":[{"name":"Science Service Centre Kuopio University Hospital  Kuopio Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J.","family":"Martikainen","sequence":"additional","affiliation":[{"name":"Pharmacoeconomics and Outcome Research Unit (PHORU) School of Pharmacy University of Kuopio  Kuopio 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