{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T00:19:01Z","timestamp":1793319541473,"version":"4.5.0"},"reference-count":26,"publisher":"American Society of Hematology","issue":"14","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2024,10,3]]},"abstract":"<jats:sec>\n                  <jats:title>Abstract<\/jats:title>\n                  <jats:p>Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening autoimmune disorder caused by a disintegrin and metalloproteinase with thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency. Caplacizumab, an anti\u2013von Willebrand factor nanobody, is approved for iTTP treatment, reducing the need for therapeutic plasma exchange (TPE) and improving platelet count recovery and survival. We conducted a retrospective study on 42 acute iTTP cases in Austria and Germany, treated with a modified regimen aimed at avoiding TPE if platelet count increased after the first caplacizumab dose. Baseline characteristics and patient outcomes were compared with a control group of 59 patients with iTTP receiving frontline treatment with TPE, caplacizumab, and immunosuppression. The main outcome was the time to platelet count normalization. Secondary outcomes included clinical response, exacerbation, refractory iTTP, iTTP-related deaths, and the time to platelet count doubling. The median time to platelet count normalization was similar between the 2 cohorts (3 and 4 days; P\u00a0=\u00a0.31). There were no significant differences in clinical response, exacerbations, refractoriness, iTTP-related deaths, or time to platelet count doubling, reflecting the short-term treatment response. Four patients did not respond to the first caplacizumab dose, and TPE was subsequently initiated. Cytomegalovirus infection, HIV\/hepatitis B virus coinfection, an ovarian teratoma with associated antiplatelet antibodies, and multiple platelet transfusions before the correct diagnosis may have impeded the immediate treatment response in these patients. In conclusion, caplacizumab and immunosuppression alone, without TPE, rapidly controlled thrombotic microangiopathy and achieved a sustained clinical response in iTTP. Our study provides a basis for TPE-free iTTP management in experienced centers via shared decision-making between patients and treating physicians.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1182\/blood.2023023780","type":"journal-article","created":{"date-parts":[[2024,6,5]],"date-time":"2024-06-05T20:15:30Z","timestamp":1717618530000},"page":"1486-1495","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":38,"title":["Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange"],"prefix":"10.1182","volume":"144","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5990-0279","authenticated-orcid":false,"given":"Lucas","family":"K\u00fchne","sequence":"first","affiliation":[{"name":"1Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7909-7225","authenticated-orcid":false,"given":"Paul","family":"Kn\u00f6bl","sequence":"additional","affiliation":[{"name":"2Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6214-5008","authenticated-orcid":false,"given":"Kathrin","family":"Eller","sequence":"additional","affiliation":[{"name":"3Division of Nephrology, Department of Internal Medicine, Medical University of Graz, Graz, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9613-8658","authenticated-orcid":false,"given":"Johannes","family":"Thaler","sequence":"additional","affiliation":[{"name":"2Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang R.","family":"Sperr","sequence":"additional","affiliation":[{"name":"2Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karoline","family":"Gleixner","sequence":"additional","affiliation":[{"name":"2Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5047-0252","authenticated-orcid":false,"given":"Thomas","family":"Osterholt","sequence":"additional","affiliation":[{"name":"1Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7718-3464","authenticated-orcid":false,"given":"Jessica","family":"Kaufeld","sequence":"additional","affiliation":[{"name":"4Department of Nephrology and Hypertension, Medical School Hannover, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan","family":"Menne","sequence":"additional","affiliation":[{"name":"5KRH Klinikum Mitte\u2013Location Siloah, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7369-9700","authenticated-orcid":false,"given":"Veronika","family":"Buxhofer-Ausch","sequence":"additional","affiliation":[{"name":"6Department of Internal Medicine I with Hematology, Stem Cell Transplantation, Hemostaseology and Medical Oncology, Ordensklinikum Linz Elisabethinnen, Linz, Austria"},{"name":"7Medical Faculty, Johannes Kepler University Linz, Linz, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anja","family":"M\u00fchlfeld","sequence":"additional","affiliation":[{"name":"8Division of Nephrology, Department of Medicine, Uniklinik RWTH Aachen, Aachen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evelyn","family":"Seelow","sequence":"additional","affiliation":[{"name":"9Department of Nephrology and Intensive Care Medicine, Charit\u00e9\u2013Universit\u00e4tsmedizin Berlin, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian","family":"Schreiber","sequence":"additional","affiliation":[{"name":"9Department of Nephrology and Intensive Care Medicine, Charit\u00e9\u2013Universit\u00e4tsmedizin Berlin, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4728-224X","authenticated-orcid":false,"given":"Polina","family":"Todorova","sequence":"additional","affiliation":[{"name":"1Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6427-177X","authenticated-orcid":false,"given":"Sadrija","family":"Cukoski","sequence":"additional","affiliation":[{"name":"1Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfram J.","family":"Jabs","sequence":"additional","affiliation":[{"name":"10Department of Nephrology, Vivantes Klinikum im Friedrichshain, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fedai","family":"\u00d6zcan","sequence":"additional","affiliation":[{"name":"11Klinik f\u00fcr Nephrologie, Klinikum Dortmund, Universit\u00e4t Witten-Herdecke, Campus Dortmund, Dortmund, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anja","family":"G\u00e4ckler","sequence":"additional","affiliation":[{"name":"12Department of Nephrology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-1072-5098","authenticated-orcid":false,"given":"Kristina","family":"Sch\u00f6nfelder","sequence":"additional","affiliation":[{"name":"12Department of Nephrology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Felix S.","family":"Seibert","sequence":"additional","affiliation":[{"name":"13Medical Department 1, Marien Hospital Herne\u2013Universit\u00e4tsklinikum der Ruhr-Universit\u00e4t Bochum, Herne, 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