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

被引：0

作者：

Gallo, Paolo ^{[1
]}

De Vincentis, Antonio ^{[1
,2
]}

Terracciani, Francesca ^{[1
]}

Falcomata, Andrea ^{[1
]}

Pace Palitti, Valeria ^{[3
]}

Russello, Maurizio ^{[4
]}

Vignone, Anthony ^{[5
]}

Alvaro, Domenico ^{[5
]}

Tortora, Raffaella ^{[6
]}

Biolato, Marco ^{[7
]}

Pompili, Maurizio ^{[7
]}

Calvaruso, Vincenza ^{[8
]}

Marzia, Veneziano ^{[8
]}

Tizzani, Marco ^{[9
]}

Caneglias, Alessandro ^{[9
]}

Frigo, Francesco ^{[9
]}

Gesualdo, Marcantonio ^{[9
]}

Marzano, Alfredo ^{[9
]}

Rosato, Valerio ^{[10
]}

Claar, Ernesto ^{[10
]}

Villani, Rosanna ^{[11
]}

Izzi, Antonio ^{[12
]}

Cozzolongo, Raffaele ^{[13
]}

Cozzolino, Antonio ^{[14
]}

Airoldi, Aldo ^{[15
]}

Mazzarelli, Chiara ^{[15
]}

Distefano, Marco ^{[16
]}

Iegri, Claudia ^{[17
]}

Fagiuoli, Stefano ^{[17
]}

Messina, Vincenzo ^{[18
]}

Ragone, Enrico ^{[19
]}

Sacco, Rodolfo ^{[20
]}

Cacciatore, Pierluigi ^{[3
]}

Masutti, Flora ^{[21
]}

Croce, Saveria Lory ^{[21
]}

Moretti, Alessandra ^{[22
]}

Flagiello, Valentina ^{[1
]}

Di Pasquale, Giulia ^{[1
]}

Picardi, Antonio ^{[1
,23
]}

Vespasiani-Gentilucci, Umberto ^{[1
,23
]}

机构：

[1] Fdn Policlin Univ Campus Biomed, I-00128 Rome, Italy

[2] Univ Campus Biomed Roma, Dept Med & Surg, Res Unit Internal Med, I-00128 Rome, Italy

[3] ASL Pescara, St Spirito Hosp, I-65026 Pescara, Italy

[4] Arnas Garibaldi, Liver Unit, I-95124 Catania, Italy

[5] Sapienza Univ Rome, Dept Translat & Precis Med, I-00185 Rome, Italy

[6] Cardarelli Hosp, Dept Med, Liver Unit, I-80131 Naples, Italy

[7] Fdn Policlin Univ Agostino Gemelli IRCCS, I-01128 Rome, Italy

[8] Univ Palermo, Gastroenterol & Hepatol Unit, I-90128 Palermo, Italy

[9] Univ Turin, AOU Citta Salute & Sci Torino, Div Gastroenterol & Hepatol, I-10126 Turin, Italy

[10] Osped Evangel Betania, UOSD Epatol, I-80147 Naples, Italy

[11] Univ Foggia, CURE Univ Ctr Liver Dis Res & Treatment, Dept Med & Surg Sci, Liver Unit, I-71122 Foggia, Italy

[12] D Cotugno Hosp, Dept Neurol Infect Dis, I-80131 Naples, Italy

[13] Natl Inst Gastroenterol S de Bellis Res Hosp, Gastroenterol Unit, I-70013 Bari, Italy

[14] Osped del Mare, Gastroenterol Unit, I-80147 Naples, Italy

[15] ASST GOM Niguarda, Hepatol & Gastroenterol, I-20162 Milan, Italy

[16] UOSD Epatol Osped Umberto I Siracusa, ASP 8, I-96100 Siracusa, Italy

[17] Univ Milano Bicocca, Dept Med, Gastroenterol, I-20126 Milan, Italy

[18] AORN St Anna & San Sebastiano, Infect Dis Unit, I-81100 Caserta, Italy

[19] UOC Med Infettivolog & Trapianti UOS Eco Intervent, Clin AORN Colli Osped Monaldi, I-80131 Naples, Italy

[20] Foggia Univ Hosp, Gastroenterol & Digest Endoscopy Unit, I-71122 Foggia, Italy

[21] Univ Hosp Trieste Azienda Sanit Giuliano Isontina, Liver Clin, I-34149 Trieste, Italy

[22] San Filippo Neri Hosp, Gastroenterol Unit, I-00135 Rome, Italy

[23] Univ Campus Biomed Roma, Dept Med & Surg, Res Unit Clin Med & Hepatol, I-00128 Rome, Italy

来源：

JOURNAL OF CLINICAL MEDICINE | 2024年 / 13卷 / 13期

关键词：

severe thrombocytopenia; thrombopoietin receptor agonists (TPO-RA); lusutrombopag; portal vein thrombosis (PVT); LIVER-TUMORS;

D O I：

10.3390/jcm13133965

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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/mu L, 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/mu L) to 58,000 (49,000-82,000), p < 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% 1.04-1.26, p 0.01), with a good discrimination power (AUROC: 0.78). Notably, a baseline platelet count <= 29,000/mu L 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/mu L are unlikely to respond to the drug.

引用

页数：11