Journal of Clinical Medicine (Jul 2024)
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
- Paolo Gallo,
- Antonio De Vincentis,
- Francesca Terracciani,
- Andrea Falcomatà,
- Valeria Pace Palitti,
- Maurizio Russello,
- Anthony Vignone,
- Domenico Alvaro,
- Raffaella Tortora,
- Marco Biolato,
- Maurizio Pompili,
- Vincenza Calvaruso,
- Veneziano Marzia,
- Marco Tizzani,
- Alessandro Caneglias,
- Francesco Frigo,
- Marcantonio Gesualdo,
- Alfredo Marzano,
- Valerio Rosato,
- Ernesto Claar,
- Rosanna Villani,
- Antonio Izzi,
- Raffaele Cozzolongo,
- Antonio Cozzolino,
- Aldo Airoldi,
- Chiara Mazzarelli,
- Marco Distefano,
- Claudia Iegri,
- Stefano Fagiuoli,
- Vincenzo Messina,
- Enrico Ragone,
- Rodolfo Sacco,
- Pierluigi Cacciatore,
- Flora Masutti,
- Saveria Lory Crocé,
- Alessandra Moretti,
- Valentina Flagiello,
- Giulia Di Pasquale,
- Antonio Picardi,
- Umberto Vespasiani-Gentilucci
Affiliations
- Paolo Gallo
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Antonio De Vincentis
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Francesca Terracciani
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Andrea Falcomatà
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Valeria Pace Palitti
- Santo Spirito Hospital, ASL Pescara, 65026 Pescara, Italy
- Maurizio Russello
- Liver Unit, Arnas Garibaldi, 95124 Catania, Italy
- Anthony Vignone
- Department of Translational and Precision Medicine, Sapienza University of Rome, 00185 Roma, Italy
- Domenico Alvaro
- Department of Translational and Precision Medicine, Sapienza University of Rome, 00185 Roma, Italy
- Raffaella Tortora
- Liver Unit, Department of Medicine, Cardarelli Hospital, 80131 Napoli, Italy
- Marco Biolato
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 01128 Roma, Italy
- Maurizio Pompili
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 01128 Roma, Italy
- Vincenza Calvaruso
- Gastroenterology and Hepatology Unit, University of Palermo, 90128 Palermo, Italy
- Veneziano Marzia
- Gastroenterology and Hepatology Unit, University of Palermo, 90128 Palermo, Italy
- Marco Tizzani
- Division of Gastroenterology and Hepatology, A.O.U. Città della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy
- Alessandro Caneglias
- Division of Gastroenterology and Hepatology, A.O.U. Città della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy
- Francesco Frigo
- Division of Gastroenterology and Hepatology, A.O.U. Città della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy
- Marcantonio Gesualdo
- Division of Gastroenterology and Hepatology, A.O.U. Città della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy
- Alfredo Marzano
- Division of Gastroenterology and Hepatology, A.O.U. Città della Salute e della Scienza di Torino, University of Turin, 10126 Turin, Italy
- Valerio Rosato
- UOSD Epatologia, Ospedale Evangelico Betania, 80147 Naples, Italy
- Ernesto Claar
- UOSD Epatologia, Ospedale Evangelico Betania, 80147 Naples, Italy
- Rosanna Villani
- C.U.R.E. (University Center for Liver Disease Research and Treatment), Liver Unit, Department of Medical and Surgical Sciences, University of Foggia, 71122 Foggia, Italy
- Antonio Izzi
- Department of Infectious Diseases, D. Cotugno Hospital, 80131 Napoli, Italy
- Raffaele Cozzolongo
- Gastroenterology Unit, National Institute of Gastroenterology, IRCCS “S de Bellis” Research Hospital, 70013 Castellana Grotte, Bari, Italy
- Antonio Cozzolino
- Gastroenterology Unit, Ospedale del Mare, 80147 Napoli, Italy
- Aldo Airoldi
- Hepatology and Gastroenterology, ASST GOM Niguarda, 20162 Milan, Italy
- Chiara Mazzarelli
- Hepatology and Gastroenterology, ASST GOM Niguarda, 20162 Milan, Italy
- Marco Distefano
- UOSD Epatologia-Ospedale Umberto I Siracusa-ASP 8, 96100 Siracusa, Italy
- Claudia Iegri
- Gastroenterology, Department of Medicine, University of Milan Bicocca, 20126 Milan, Italy
- Stefano Fagiuoli
- Gastroenterology, Department of Medicine, University of Milan Bicocca, 20126 Milan, Italy
- Vincenzo Messina
- Infectious Disease Unit, AORN Sant’Anna e San Sebastiano, 81100 Caserta, Italy
- Enrico Ragone
- UOC Medicina Infettivologica e dei Trapianti UOS Eco Interventistica, Clinica AORN Dei Colli-Ospedale Monaldi, 80131 Napoli, Italy
- Rodolfo Sacco
- Gastroenterology and Digestive Endoscopy Unit, Foggia University Hospital, 71122 Foggia, Italy
- Pierluigi Cacciatore
- Santo Spirito Hospital, ASL Pescara, 65026 Pescara, Italy
- Flora Masutti
- Liver Clinic, University Hospital of Trieste (Azienda Sanitaria Giuliano-Isontina), 34149 Trieste, Italy
- Saveria Lory Crocé
- Liver Clinic, University Hospital of Trieste (Azienda Sanitaria Giuliano-Isontina), 34149 Trieste, Italy
- Alessandra Moretti
- Gastroenterology Unit, San Filippo Neri Hospital, 00135 Rome, Italy
- Valentina Flagiello
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Giulia Di Pasquale
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Antonio Picardi
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- Umberto Vespasiani-Gentilucci
- Fondazione Policlinico Universitario Campus Bio-Medico, 00128 Roma, Italy
- DOI
- https://doi.org/10.3390/jcm13133965
- Journal volume & issue
-
Vol. 13,
no. 13
p. 3965
Abstract
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 “Club Epatologi Ospedalieri” (CLEO). Primary and secondary efficacy endpoints were the ability of lusutrombopag to avoid platelet transfusions and to raise the platelet count to ≥50,000/μL, 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 (33,000–44,000/μL) to 58,000 (49,000–82,000), p p 0.01), with a good discrimination power (AUROC: 0.78). Notably, a baseline platelet count ≤ 29,000/μL 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 ≤29,000/μL are unlikely to respond to the drug.
Keywords
- severe thrombocytopenia
- thrombopoietin receptor agonists (TPO-RA)
- lusutrombopag
- portal vein thrombosis (PVT)