{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,7]],"date-time":"2026-07-07T20:14:04Z","timestamp":1783455244605,"version":"3.55.0"},"reference-count":20,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2026,5,10]],"date-time":"2026-05-10T00:00:00Z","timestamp":1778371200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,5,10]],"date-time":"2026-05-10T00:00:00Z","timestamp":1778371200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra rare haematological disorder. This study aimed to estimate the clinical burden, healthcare resource use (HCRU) and associated costs of cTTP in England using primary and secondary care data. A retrospective cohort study was undertaken using the Clinical Practice Research Datalink (Aurum) linked to Hospital Episode Statistics data. The study period was from 01 January 2000 to 31 December 2019. Patients with cTTP were identified using an algorithm and matched 1:2 to patients without thrombotic thrombocytopenic purpura (TTP) (non\u2010TTP cohort) to contextualise the findings. A total of 36 cTTP patients were identified. The mean follow\u2010up for cTTP patients was 4.4 and 5.7\u2009years in primary and secondary care, respectively, and 4.9 and 5.8\u2009years for the non\u2010TTP cohort. Of the 36 patients, 38.9% experienced an acute episode and 19.4% experienced an organ damage event during follow\u2010up. HCRU (including inpatient admission, outpatient appointments and primary care appointments) was higher in the cTTP cohort relative to the non\u2010TTP cohort. The average yearly cost of healthcare for a cTTP patient was \u00a36155, relative to \u00a3858 in the non\u2010TTP cohort. This study demonstrates the high clinical and economic burden of cTTP and provides evidence to inform healthcare planning.<\/jats:p>","DOI":"10.1111\/bjh.70534","type":"journal-article","created":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:03:28Z","timestamp":1778457808000},"page":"248-256","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Clinical burden and healthcare resource use of congenital thrombotic thrombocytopenic purpura in England: A linked primary and secondary care data analysis"],"prefix":"10.1111","volume":"209","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1692-9028","authenticated-orcid":false,"given":"Erin","family":"Barker","sequence":"first","affiliation":[{"name":"York Health Economics Consortium  Heslington York UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Will","family":"Lester","sequence":"additional","affiliation":[{"name":"Queen Elizabeth Hospital, University Hospitals Birmingham  Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Riley","sequence":"additional","affiliation":[{"name":"York Health Economics Consortium  Heslington York UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5802-3198","authenticated-orcid":false,"given":"Monica","family":"Garrett","sequence":"additional","affiliation":[{"name":"York Health Economics Consortium  Heslington York UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amie","family":"Padhiar","sequence":"additional","affiliation":[{"name":"Takeda UK Ltd  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annie","family":"Berkley","sequence":"additional","affiliation":[{"name":"Takeda UK Ltd  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sudhakar","family":"Manne","sequence":"additional","affiliation":[{"name":"Takeda UK Ltd  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Umesh","family":"Doobaree","sequence":"additional","affiliation":[{"name":"Takeda UK Ltd  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-0087-3798","authenticated-orcid":false,"given":"Oliver","family":"Heard","sequence":"additional","affiliation":[{"name":"Takeda UK Ltd  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,5,10]]},"reference":[{"key":"e_1_2_14_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtha.2022.12.018"},{"key":"e_1_2_14_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-10-709857"},{"key":"e_1_2_14_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(16)30018-7"},{"key":"e_1_2_14_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(16)30125-9"},{"key":"e_1_2_14_6_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm12103365"},{"key":"e_1_2_14_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2008.07276.x"},{"key":"e_1_2_14_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-11-884700"},{"key":"e_1_2_14_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.19026"},{"key":"e_1_2_14_10_1","doi-asserted-by":"publisher","DOI":"10.1093\/ije\/dyv098"},{"key":"e_1_2_14_11_1","unstructured":"CPRD.CPRD Aurum data specification.2023[cited 15 Jul 2025]. 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