{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T19:08:43Z","timestamp":1791572923212,"version":"4.3.4"},"reference-count":25,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T00:00:00Z","timestamp":1697673600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Hereditary thrombotic thrombocytopenia purpura (hTTP) is an ultra\u2010rare disorder resulting from an inherited deficiency of ADAMTS13, a von Willebrand factor (VWF)\u2010cleaving metalloprotease. The plasma\u2010derived factor VIII\/VWF Koate (FVIII\/VWF<jats:sup>Koate<\/jats:sup>) has been shown to contain ADAMTS13, allowing for its use to treat hTTP at home by the patient\/caregiver.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>Based on prior demonstration of safe and effective use of FVIII\/VWF<jats:sup>Koate<\/jats:sup> in eight patients with hTTP, we conducted a retrospective study to gather additional data regarding the use of FVIII\/VWF<jats:sup>Koate<\/jats:sup> for hTTP.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was a multicentre, retrospective, noninterventional chart review of patients who had received FVIII\/VWF<jats:sup>Koate<\/jats:sup> for the management of hTTP. Data collected included demographics, medical history, relevant family history, past use and tolerability of fresh frozen plasma, and details regarding FVIII\/VWF<jats:sup>Koate<\/jats:sup> therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The cohort included 11 patients (seven males, four females) with hTTP, ranging in age at study entry from 2 to 28\u00a0years. The average duration of FVIII\/VWF<jats:sup>Koate<\/jats:sup> therapy was 4.8\u00a0years (range, 0.5\u20136.5\u00a0years). Among nine patients using FVIII\/VWF<jats:sup>Koate<\/jats:sup> as prophylaxis, the normalized annual rate of breakthrough TTP episodes ranged from 0.2 to 1.1 episodes\/year. All nine patients who received FVIII\/VWF<jats:sup>Koate<\/jats:sup> prophylaxis had thrombocytopenia recorded at baseline, while eight (88.9%) did not have thrombocytopenia after using FVIII\/VWF<jats:sup>Koate<\/jats:sup>. There was one AE (unspecified) attributed to FVIII\/VWF<jats:sup>Koate<\/jats:sup>.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>These data suggest that FVIII\/VWF<jats:sup>Koate<\/jats:sup> is a safe and well\u2010tolerated source of the missing ADAMTS13 enzyme in patients with hTTP, producing a marked reduction in thrombocytopenia prevalence, low frequency of TTP episodes, and with the added benefit of self\u2010 or caregiver\u2010administration.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14891","type":"journal-article","created":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T14:15:26Z","timestamp":1697724926000},"page":"1638-1645","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Long\u2010term follow\u2010up of patients with congenital thrombotic thrombocytopenia purpura receiving a plasma\u2010derived factor VIII (Koate) that contains ADAMTS13"],"prefix":"10.1111","volume":"29","author":[{"given":"Tammuella","family":"Chrisentery\u2010Singleton","sequence":"first","affiliation":[{"name":"American Thrombosis and Hemostasis Network  Rochester New York USA"},{"name":"Ochsner Clinic Foundation  New Orleans Louisiana USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa N.","family":"Boggio","sequence":"additional","affiliation":[{"name":"Rush Hemophilia &amp; Thrombophilia Center Rush University Medical Center  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5350-1763","authenticated-orcid":false,"given":"Manuel D.","family":"Carcao","sequence":"additional","affiliation":[{"name":"Division of Haematology\/Oncology Department of Paediatrics The Hospital for Sick Children  Toronto ON Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sami","family":"Ibrahimi","sequence":"additional","affiliation":[{"name":"OUHSC\/Stephenson Cancer Center  Oklahoma City Oklahoma USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Osman","family":"Khan","sequence":"additional","affiliation":[{"name":"University of Oklahoma Health Sciences Center  Oklahoma City Oklahoma USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arash","family":"Mahajerin","sequence":"additional","affiliation":[{"name":"Children's Hospital of Orange County  Orange California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anita","family":"Rajasekhar","sequence":"additional","affiliation":[{"name":"University of Florida \u2013 Shands Hospital  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vivek","family":"Sharma","sequence":"additional","affiliation":[{"name":"University of Louisville  Louisville Kentucky USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"MacGregor","family":"Steele","sequence":"additional","affiliation":[{"name":"Alberta Children's Hospital  Edmonton AB Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcela","family":"Torres","sequence":"additional","affiliation":[{"name":"Department of Hematology Cook Children's Medical Center  Fort Worth Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank J.","family":"Rodino","sequence":"additional","affiliation":[{"name":"Churchill Outcomes Research  Red Bank New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shannon L.","family":"Carpenter","sequence":"additional","affiliation":[{"name":"Children's Mercy Hospital  Kansas City Missouri USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,10,19]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-11-884700"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM197902153000717"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1080\/09537104.2022.2138306"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.13571"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/ijlh.13295"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.216796"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2013.1.292"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1312353"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020009801"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1097\/MPH.0000000000000917"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15010"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1537-2995.2012.03663.x"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40271-019-00365-y"},{"key":"e_1_2_11_15_1","doi-asserted-by":"crossref","unstructured":"ScullyM WindygaJ Mellg\u00e5rdB et\u00a0al.Phase 3 prospective randomized controlled open\u2010label multicenter crossover study of recombinant ADAMTS13 in patients with congenital thrombotic thrombocytopenic purpura.Paper presented at: International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress; 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