{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,9]],"date-time":"2026-07-09T14:19:22Z","timestamp":1783606762181,"version":"3.55.0"},"reference-count":21,"publisher":"MDPI AG","issue":"13","license":[{"start":{"date-parts":[[2024,7,6]],"date-time":"2024-07-06T00:00:00Z","timestamp":1720224000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background and aims: Management of severe thrombocytopenia poses significant challenges in patients with chronic liver disease. Here, we aimed to evaluate the first real-world European post-marketing cohort of cirrhotic patients treated with lusutrombopag, a thrombopoietin receptor agonist, verifying the efficacy and safety of the drug. Methods: In the REAl-world Lusutrombopag treatment in ITalY (REALITY) study, we collected data from consecutive cirrhotic patients treated with lusutrombopag in 19 Italian hepatology centers, mostly joined to the \u201cClub Epatologi Ospedalieri\u201d (CLEO). Primary and secondary efficacy endpoints were the ability of lusutrombopag to avoid platelet transfusions and to raise the platelet count to \u226550,000\/\u03bcL, respectively. Treatment-associated adverse events were also collected. Results: A total of 66 patients and 73 cycles of treatment were included in the study, since 5 patients received multiple doses of lusutrombopag over time for different invasive procedures. Fourteen patients (19%) had a history of portal vein thrombosis (PVT). Lusutrombopag determined a significant increase in platelet count [from 37,000 (33,000\u201344,000\/\u03bcL) to 58,000 (49,000\u201382,000), p &lt; 0.001]. The primary endpoint was met in 84% of patients and the secondary endpoint in 74% of patients. Baseline platelet count was the only independent factor associated with response in multivariate logistic regression analysis (OR for any 1000 uL of 1.13, CI95% 1.04\u20131.26, p 0.01), with a good discrimination power (AUROC: 0.78). Notably, a baseline platelet count \u2264 29,000\/\u03bcL was identified as the threshold for identifying patients unlikely to respond to the drug (sensitivity of 91%). Finally, de novo PVT was observed in four patients (5%), none of whom had undergone repeated treatment, and no other safety or hemorrhagic events were recorded in the entire population analyzed. Conclusions: In this first European real-world series, lusutrombopag demonstrated efficacy and safety consistent with the results of registrational studies. According to our results, patients with baseline platelet counts \u226429,000\/\u03bcL are unlikely to respond to the drug.<\/jats:p>","DOI":"10.3390\/jcm13133965","type":"journal-article","created":{"date-parts":[[2024,7,8]],"date-time":"2024-07-08T09:01:19Z","timestamp":1720429279000},"page":"3965","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Final Results from the First European Real-World Experience on Lusutrombopag Treatment in Cirrhotic Patients with Severe Thrombocytopenia: Insights from the REAl-World Lusutrombopag Treatment in ITalY Study"],"prefix":"10.3390","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8292-1134","authenticated-orcid":false,"given":"Paolo","family":"Gallo","sequence":"first","affiliation":[{"name":"Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"De Vincentis","sequence":"additional","affiliation":[{"name":"Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy"},{"name":"Research Unit of Internal Medicine, Department of Medicine and Surgery, Universit\u00e0 Campus Bio-Medico di Roma, 01128 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Terracciani","sequence":"additional","affiliation":[{"name":"Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4523-130X","authenticated-orcid":false,"given":"Andrea","family":"Falcomat\u00e0","sequence":"additional","affiliation":[{"name":"Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valeria","family":"Pace Palitti","sequence":"additional","affiliation":[{"name":"Santo Spirito Hospital, ASL Pescara, 65026 Pescara, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-3650-5541","authenticated-orcid":false,"given":"Maurizio","family":"Russello","sequence":"additional","affiliation":[{"name":"Liver Unit, Arnas Garibaldi, 95124 Catania, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6009-2398","authenticated-orcid":false,"given":"Anthony","family":"Vignone","sequence":"additional","affiliation":[{"name":"Department of Translational and Precision Medicine, Sapienza University of Rome, 00185 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3771-9644","authenticated-orcid":false,"given":"Domenico","family":"Alvaro","sequence":"additional","affiliation":[{"name":"Department of Translational and Precision Medicine, Sapienza University of Rome, 00185 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raffaella","family":"Tortora","sequence":"additional","affiliation":[{"name":"Liver Unit, Department of Medicine, Cardarelli Hospital, 80131 Napoli, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5172-8208","authenticated-orcid":false,"given":"Marco","family":"Biolato","sequence":"additional","affiliation":[{"name":"Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 01128 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maurizio","family":"Pompili","sequence":"additional","affiliation":[{"name":"Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 01128 Roma, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0287-1059","authenticated-orcid":false,"given":"Vincenza","family":"Calvaruso","sequence":"additional","affiliation":[{"name":"Gastroenterology and Hepatology Unit, University of Palermo, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Veneziano","family":"Marzia","sequence":"additional","affiliation":[{"name":"Gastroenterology and Hepatology Unit, University of Palermo, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco","family":"Tizzani","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology, A.O.U. Citt\u00e0 della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandro","family":"Caneglias","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology, A.O.U. Citt\u00e0 della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Frigo","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology, A.O.U. Citt\u00e0 della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcantonio","family":"Gesualdo","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology, A.O.U. Citt\u00e0 della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8843-8645","authenticated-orcid":false,"given":"Alfredo","family":"Marzano","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology, A.O.U. 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Gastroenterol."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1097\/00001721-199104000-00026","article-title":"Clinical aspects of platelet transfusions","volume":"2","author":"Murphy","year":"1991","journal-title":"Blood Coagul. Fibrinolysis"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1111\/liv.13317","article-title":"Thrombocytopenia in chronic liver disease","volume":"37","year":"2017","journal-title":"Liver Int."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"1192","DOI":"10.1016\/j.cgh.2018.11.047","article-title":"Lusutrombopag Reduces Need for Platelet Transfusion in Patients with Thrombocytopenia Undergoing Invasive Procedures","volume":"17","author":"Hidaka","year":"2019","journal-title":"Clin. Gastroenterol. Hepatol."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"1336","DOI":"10.1002\/hep.30561","article-title":"Lusutrombopag for the Treatment of Thrombocytopenia in Patients with Chronic Liver Disease Undergoing Invasive Procedures (L-PLUS 2)","volume":"70","author":"Simon","year":"2019","journal-title":"Hepatology"},{"key":"ref_6","doi-asserted-by":"crossref","unstructured":"Takada, H., Kurosaki, M., Nakanishi, H., Takahashi, Y., Itakura, J., Tsuchiya, K., Yasui, Y., Tamaki, N., Takaura, K., and Komiyama, Y. (2019). Real-life experience of lusutrombopag for cirrhotic patients with low platelet counts being prepared for invasive procedures. PLoS ONE, 14.","DOI":"10.1371\/journal.pone.0211122"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s12876-020-01573-9","article-title":"Lusutrombopag is effective and safe in patients with chronic liver disease and severe thrombocytopenia: A multicenter retrospective study","volume":"20","author":"Nomoto","year":"2020","journal-title":"BMC Gastroenterol."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"590","DOI":"10.1111\/hepr.13305","article-title":"Efficacy and safety of repeated use of lusutrombopag prior to radiofrequency ablation in patients with recurrent hepatocellular carcinoma and thrombocytopenia","volume":"49","author":"Ishikawa","year":"2019","journal-title":"Hepatol. 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Gastroenterol."}],"container-title":["Journal of Clinical Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2077-0383\/13\/13\/3965\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T15:11:08Z","timestamp":1760109068000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2077-0383\/13\/13\/3965"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,6]]},"references-count":21,"journal-issue":{"issue":"13","published-online":{"date-parts":[[2024,7]]}},"alternative-id":["jcm13133965"],"URL":"https:\/\/doi.org\/10.3390\/jcm13133965","relation":{},"ISSN":["2077-0383"],"issn-type":[{"value":"2077-0383","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,7,6]]}}}