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<PubmedArticle><MedlineCitation Status="MEDLINE" Owner="NLM" IndexingMethod="Curated"><PMID Version="1">37169697</PMID><DateCompleted><Year>2023</Year><Month>06</Month><Day>09</Day></DateCompleted><DateRevised><Year>2023</Year><Month>06</Month><Day>09</Day></DateRevised><Article PubModel="Print-Electronic"><Journal><ISSN IssnType="Print">1473-0502</ISSN><JournalIssue CitedMedium="Internet"><Volume>62</Volume><Issue>3</Issue><PubDate><Year>2023</Year><Month>Jun</Month></PubDate></JournalIssue><Title>Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis</Title><ISOAbbreviation>Transfus Apher Sci</ISOAbbreviation></Journal><ArticleTitle>Real-world data of the use and experience of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura: Case series.</ArticleTitle><Pagination><StartPage>103722</StartPage><MedlinePgn>103722</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1016/j.transci.2023.103722</ELocationID><ELocationID EIdType="pii" ValidYN="Y">S1473-0502(23)00096-4</ELocationID><Abstract><AbstractText Label="PURPOSE" NlmCategory="OBJECTIVE">Caplacizumab was licensed for acquired thrombotic thrombocytopenic purpura (aTTP) based on prospective controlled trials. Real-world evidence is crucial in rare diseases. We aim to describe a patient population with aTTP, receiving caplacizumab in a real-world setting, reporting their outcomes, including safety and tolerability, and contrasting them with a historical cohort from our center.</AbstractText><AbstractText Label="METHODS" NlmCategory="METHODS">We describe data collected retrospectively from 2012 to 2022 for 16 patients with aTTP (8 received caplacizumab and 8 the historical standard-of-care). Patients' characteristics and outcomes were compared between groups.</AbstractText><AbstractText Label="RESULTS" NlmCategory="RESULTS">Patients' demographic and baseline characteristics were similar in both groups. Caplacizumab led to a rapid normalization of the platelet count of 3.5 (IQR, 2-6) versus 16 (IQR, 9.5-23.5) days in the historical cohort: (p&#xa0;=&#xa0;.002). The median number of plasma exchanges and the length of days requiring them, between the caplacizumab group versus the historical cohort, was 6 (IQR, 6-10) versus 19.5 (IQR, 12.5-29.5) plasma exchanges (p&#xa0;=&#xa0;.006); and 9 (IQR, 8.5-13.5) versus 22 (15-31) days (p&#xa0;=&#xa0;.049), respectively. There were no refractory cases in the caplacizumab group in comparison with 37.5 % in the historical cohort. None of patients treated with caplacizumab experienced a recurrence after 1081 (IQR, 511-3125) days of follow-up. Safety was in line with data reported in clinical trials, with mild adverse events (mostly grade&#x2264;2).</AbstractText><AbstractText Label="CONCLUSION" NlmCategory="CONCLUSIONS">We provided real-world evidence in the treatment of aTTP, confirming the results obtained in clinical trials. Caplacizumab reduced the time to platelet count recovery and the number and length of plasma exchanges.</AbstractText><CopyrightInformation>Copyright &#xa9; 2023 Elsevier Ltd. All rights reserved.</CopyrightInformation></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Albanell-Fern&#xe1;ndez</LastName><ForeName>Marta</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Monge-Escart&#xed;n</LastName><ForeName>In&#xe9;s</ForeName><Initials>I</Initials><AffiliationInfo><Affiliation>Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Carcelero-San Mart&#xed;n</LastName><ForeName>Esther</ForeName><Initials>E</Initials><AffiliationInfo><Affiliation>Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Riu Viladoms</LastName><ForeName>Gisela</ForeName><Initials>G</Initials><AffiliationInfo><Affiliation>Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Ruiz-Boy</LastName><ForeName>Sonia</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lozano</LastName><ForeName>Miquel</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>Apheresis &amp; Cellular Therapy Unit, Department of Hemotherapy and Hemostasis, ICMHO, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain; August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Soy</LastName><ForeName>Dolors</ForeName><Initials>D</Initials><AffiliationInfo><Affiliation>Pharmacy Service, Division of Medicines, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain; August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Barcelona, Spain; Department of Pharmacology, Toxicology and Therapeutic Chemistry. School of Pharmacy. University of Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Moreno-Casta&#xf1;o</LastName><ForeName>Ana Bel&#xe9;n</ForeName><Initials>AB</Initials><AffiliationInfo><Affiliation>August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Barcelona, Spain; Hemostasis and Erythropathology Laboratory, Pathology Department. Biomedical Diagnosis Center (CDB), Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Diaz-Ricart</LastName><ForeName>Maribel</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Barcelona, Spain; Hemostasis and Erythropathology Laboratory, Pathology Department. Biomedical Diagnosis Center (CDB), Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Cid</LastName><ForeName>Joan</ForeName><Initials>J</Initials><AffiliationInfo><Affiliation>Apheresis &amp; Cellular Therapy Unit, Department of Hemotherapy and Hemostasis, ICMHO, Hospital Clinic of Barcelona, University of Barcelona, Barcelona, Spain; August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Barcelona, Spain. Electronic address: jcid@clinic.cat.</Affiliation></AffiliationInfo></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D016454">Review</PublicationType></PublicationTypeList><ArticleDate DateType="Electronic"><Year>2023</Year><Month>05</Month><Day>08</Day></ArticleDate></Article><MedlineJournalInfo><Country>England</Country><MedlineTA>Transfus Apher Sci</MedlineTA><NlmUniqueID>101095653</NlmUniqueID><ISSNLinking>1473-0502</ISSNLinking></MedlineJournalInfo><ChemicalList><Chemical><RegistryNumber>2R27AB6766</RegistryNumber><NameOfSubstance UI="C585343">caplacizumab</NameOfSubstance></Chemical></ChemicalList><SupplMeshList><SupplMeshName Type="Disease" UI="C536901">Thrombotic thrombocytopenic purpura, acquired</SupplMeshName></SupplMeshList><MeshHeadingList><MeshHeading><DescriptorName UI="D006801" MajorTopicYN="N">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D010951" MajorTopicYN="N">Plasma Exchange</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D011446" MajorTopicYN="N">Prospective Studies</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D011697" MajorTopicYN="Y">Purpura, Thrombotic Thrombocytopenic</DescriptorName><QualifierName UI="Q000628" MajorTopicYN="N">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D012189" MajorTopicYN="N">Retrospective Studies</DescriptorName></MeshHeading></MeshHeadingList><KeywordList Owner="NOTNLM"><Keyword MajorTopicYN="N">ADAMTS13</Keyword><Keyword MajorTopicYN="N">Acquired thrombotic thrombocytopenic purpura</Keyword><Keyword MajorTopicYN="N">Caplacizumab</Keyword><Keyword MajorTopicYN="N">Plasma exchange</Keyword><Keyword MajorTopicYN="N">Platelet count</Keyword><Keyword MajorTopicYN="N">Real-world evidence</Keyword></KeywordList><CoiStatement>Declarations of Competing Interest Joan Cid has received research funding from Cerus, Kawasumi Laboratories, and Sanofi. He has also received speaker or advisory fees from Cerus, Fresenius Kabi, Grifols, MacoPharma, Sanofi, and TerumoBCT. Maribel Diaz-Ricart has received research funding from Zacros (Fujimori Kogyo Co., Ltd., Japan), Cellphire Therapeutics (US), and CSL Behring (Spain). The other authors declare no conflicts of interest.</CoiStatement></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="medline"><Year>2023</Year><Month>6</Month><Day>9</Day><Hour>6</Hour><Minute>42</Minute></PubMedPubDate><PubMedPubDate PubStatus="pubmed"><Year>2023</Year><Month>5</Month><Day>12</Day><Hour>1</Hour><Minute>7</Minute></PubMedPubDate><PubMedPubDate PubStatus="entrez"><Year>2023</Year><Month>5</Month><Day>11</Day><Hour>22</Hour><Minute>2</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="pubmed">37169697</ArticleId><ArticleId IdType="doi">10.1016/j.transci.2023.103722</ArticleId><ArticleId IdType="pii">S1473-0502(23)00096-4</ArticleId></ArticleIdList></PubmedData></PubmedArticle></PubmedArticleSet>