{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T05:13:05Z","timestamp":1779167585021,"version":"3.51.4"},"reference-count":14,"publisher":"Georg Thieme Verlag KG","issue":"S 01","license":[{"start":{"date-parts":[[2022,10,26]],"date-time":"2022-10-26T00:00:00Z","timestamp":1666742400000},"content-version":"vor","delay-in-days":25,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"Bayer"},{"DOI":"10.13039\/501100025230","name":"Biotest","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100025230","id-type":"DOI","asserted-by":"crossref"}]},{"name":"CSL Behring"},{"DOI":"10.13039\/501100016387","name":"Grifols","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100016387","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Intersero"},{"name":"Novo Nordisk"},{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100012112","name":"Sobi","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100012112","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Takeda"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Hamostaseologie"],"published-print":{"date-parts":[[2022,10]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Objectives\u2003Initial treatment in patients with haemophilia remains challenging. The choice of therapy, timing, dose and frequency have been and are still under intense debate. New treatment options like novel factor concentrates and non-factor therapies broaden the discussion.<\/jats:p>\n                  <jats:p>Design\u2003The German Paediatric Haemophilia Research Database (GEPHARD) is a multicentre prospective observational study including children and adolescents with haemophilia A or B (FVIII or FIX levels &lt;25\u2009IU\/dL) in a German treatment centre after January 1st, 2017. A cross-sectional analysis was performed in June 2021.<\/jats:p>\n                  <jats:p>Results\u2003249 children and adolescents from 22 participating centres in Germany were analysed in this cross-sectional analysis. 203 patients suffered from haemophilia A (PwHA) and 46 from haemophilia B (PwHB). The median age at diagnosis for Pw severe HA or HB was 6 or 2 months, the median age at analysis was 33 or 35 months for Pw severe HA or B, respectively. 117 Pw severe HA received treatment, including plasma derived concentrates (n\u2009=\u200943), standard recombinant concentrates (n\u2009=\u200923), extended half live concentrates (n\u2009=\u200933) and non-replacement therapies (n\u2009=\u200918). For Pw severe HB, plasma derived concentrates (n\u2009=\u20093), standard recombinant concentrates (n\u2009=\u20098) and extended half live concentrates (n\u2009=\u200914) were used. Current inhibitors were reported in 16 PwHA and 1 PwHB.<\/jats:p>\n                  <jats:p>Conclusions\u2003GEPHARD was successfully established as a national cohort for newly diagnosed PwH in Germany. Epidemiological and treatment data were presented. Longitudinal analyses of this growing cohort will allow to value treatment strategies and their outcome in the evolving treatment landscape.<\/jats:p>","DOI":"10.1055\/s-0042-1757552","type":"journal-article","created":{"date-parts":[[2022,10,26]],"date-time":"2022-10-26T18:50:15Z","timestamp":1666810215000},"page":"S24-S31","source":"Crossref","is-referenced-by-count":3,"special_numbering":"S","title":["A Cross-sectional Analysis of Treatment in PUPs in 2021 in Germany \u2013 First Data from the GEPHARD Study Group"],"prefix":"10.1055","volume":"42","author":[{"name":"The GEPHARD Investigators","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"Contributors:","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"Contributors to the cross-sectional analysis:","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"Study commission","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2022,10,26]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"630","DOI":"10.1016\/S0140-6736(20)32722-7","article-title":"The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing","volume":"397","author":"M E Mancuso","year":"2021","journal-title":"Lancet"},{"issue":"01","key":"ref2","doi-asserted-by":"publisher","first-page":"e18","DOI":"10.1111\/j.1365-2516.2011.02659.x","article-title":"Early prophylaxis\/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients\u2013long-term follow-up and continuing experience","volume":"18","author":"G Auerswald","year":"2012","journal-title":"Haemophilia"},{"issue":"02","key":"ref3","doi-asserted-by":"publisher","first-page":"256","DOI":"10.1111\/j.1365-2516.2009.02122.x","article-title":"New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development","volume":"16","author":"K Kurnik","year":"2010","journal-title":"Haemophilia"},{"issue":"05","key":"ref4","doi-asserted-by":"publisher","first-page":"622","DOI":"10.1111\/hae.12666","article-title":"The EPIC study: a lesson to learn","volume":"21","author":"G Auerswald","year":"2015","journal-title":"Haemophilia"},{"issue":"03","key":"ref5","doi-asserted-by":"publisher","first-page":"420","DOI":"10.1111\/hae.13421","article-title":"Clinical trials and registries in haemophilia: Opponents or collaborators? Comparison of PUP data derived from different data sources","volume":"24","author":"C Keipert","year":"2018","journal-title":"Haemophilia"},{"issue":"21","key":"ref6","doi-asserted-by":"publisher","first-page":"2054","DOI":"10.1056\/NEJMoa1516437","article-title":"A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A","volume":"374","author":"F Peyvandi","year":"2016","journal-title":"N Engl J Med"},{"issue":"04","key":"ref7","doi-asserted-by":"publisher","first-page":"e280","DOI":"10.1111\/hae.12448","article-title":"Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry","volume":"20","author":"K Fischer","year":"2014","journal-title":"Haemophilia"},{"issue":"23","key":"ref8","doi-asserted-by":"publisher","first-page":"3398","DOI":"10.1182\/blood-2014-07-586347","article-title":"Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A","volume":"124","author":"T Calvez","year":"2014","journal-title":"Blood"},{"issue":"23","key":"ref9","doi-asserted-by":"publisher","first-page":"3389","DOI":"10.1182\/blood-2014-07-580498","article-title":"Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011","volume":"124","author":"P W Collins","year":"2014","journal-title":"Blood"},{"issue":"03","key":"ref10","doi-asserted-by":"publisher","first-page":"231","DOI":"10.1056\/NEJMoa1208024","article-title":"Factor VIII products and inhibitor development in severe hemophilia A","volume":"368","author":"S C Gouw","year":"2013","journal-title":"N Engl J Med"},{"issue":"01","key":"ref11","doi-asserted-by":"publisher","first-page":"123","DOI":"10.3324\/haematol.2019.239160","article-title":"Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study","volume":"106","author":"C Male","year":"2021","journal-title":"Haematologica"},{"issue":"11","key":"ref12","doi-asserted-by":"publisher","first-page":"E3408","DOI":"10.3390\/jcm9113408","article-title":"The German Hemophilia Registry: Growing with Its Tasks","volume":"9","author":"H Duda","year":"2020","journal-title":"J Clin Med"},{"issue":"06","key":"ref13","first-page":"EREP04","article-title":"Epidemiology of inhibitors and current treatment strategies","volume":"88","author":"W Kreuz","year":"2003","journal-title":"Haematologica"},{"issue":"22","key":"ref14","doi-asserted-by":"publisher","first-page":"5785","DOI":"10.1182\/bloodadvances.2020002731","article-title":"The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development","volume":"4","author":"B M Reipert","year":"2020","journal-title":"Blood Adv"}],"container-title":["H\u00e4mostaseologie"],"original-title":[],"language":"de","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/s-0042-1757552.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,25]],"date-time":"2025-11-25T21:43:39Z","timestamp":1764107019000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/s-0042-1757552"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,10]]},"references-count":14,"journal-issue":{"issue":"S 01","published-online":{"date-parts":[[2022,10,26]]},"published-print":{"date-parts":[[2022,10]]}},"URL":"https:\/\/doi.org\/10.1055\/s-0042-1757552","archive":["Portico","CLOCKSS"],"relation":{},"ISSN":["0720-9355","2567-5761"],"issn-type":[{"value":"0720-9355","type":"print"},{"value":"2567-5761","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,10]]}}}