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

DOI
https://doi.org/10.3390/jcm13133965
Journal volume & issue
Vol. 13, no. 13
p. 3965

Abstract

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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.

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