ROP-ET : a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options
Kiladjian, Jean-Jacques (Université Paris Cité)
Ferrer Marin, Francisca (Hospital General Universitario Morales Meseguer (Múrcia))
Al-Ali, Haifa Kathrin (University Hospital Halle (Saale, Alemanya))
Álvarez Larrán, Alberto (Hospital Clínic i Provincial de Barcelona)
Beggiato, Eloise (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino)
Bieniaszewska, Maria (Medical University of Gdańsk)
Breccia, Massimo (Università degli Studi di Roma "La Sapienza")
Buxhofer-Ausch, Veronika (Johannes Kepler University Linz)
Cerna, Olga (University Hospital Kralovske Vinohrady)
Crisan, Ana Manuela (Center for Hematology and Bone Marrow Transplantation (Bucarest))
Danaila, Catalin Doru (Regional Institute of Oncology (Iasi, Romania))
De Stefano, Valerio (Fondazione Policlinico "A. Gemelli" IRCCS)
Döhner, Konstanze (University Hospital of Ulm (Alemanya))
Empson, Victoria (AOP Health (Vienna, Austria))
Gora-Tybor, Joanna (Medical University of Lodz)
Griesshammer, Martin (Ruhr University Bochum)
Grosicki, Sebastian (Medical University of Silesia)
Guglielmelli, Paola (Azienda Ospedaliero-Universitaria Careggi)
García-Gutierrez, Valentín (Universidad de Alcalá)
Heidel, Florian H. (Hannover Medical School)
Illés, Arpád (University of Debrecen (Hongria))
Tomuleasa, Ciprian (Iuliu Hatieganu University of Medicine and Pharmacy (Romania))
James, Chloe (Centre Hospitalier Universitaire de Bordeaux)
Koschmieder, Steffen (RWTH Aachen University (Aachen, Alemanya))
Krauth, Maria-Theresa (Medical University of Vienna)
Krejcy, Kurt (AOP Health (Vienna, Austria))
Lazaroiu, Mihaela-Cornelia (Policlinica de Diagnostic Rapid Brasov (Brasov, Romania))
Mayer, Jiri (University Hospital Brno (República Txeca))
Nagy, Zsolt György (Semmelweis University (Budapest, Hongria))
Nicolini, Franck-Emmanuel (Centre Léon Bérard (França))
Palandri, Francesca (IRCCS Azienda Ospedaliero-Universitaria di Bologna)
Pappa, Vassiliki (University General Hospital Attikon (Haidari, Grècia))
Reiter, Andreas Johannes (University Hospital Mannheim (Alemanya))
Sacha, Tomasz (Jagiellonian University Hospital (Cracòvia, Polònia))
Schlager, Stefanie (AOP Health (Vienna, Austria))
Schmidt, Stefan (Medizinische Universität Innsbruck (Innsbruck, Alemanya))
Terpos, Evangelos (National and Kapodistrian University of Athens)
Unger, Martin (AOP Health (Vienna, Austria))
Wölfler, Albert (Medizinischen Universität Graz)
Xicoy, Blanca (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Klade, Christoph (AOP Health (Vienna, Austria))

Data:	2024
Resum:	Interferon-based therapies, such as ropeginterferon alfa-2b have emerged as promising disease-modifying agents for myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET). Current ET treatments aim to normalize hematological parameters and reduce the thrombotic risk, but they do not modify the natural history of the disease and hence, have no impact on disease progression. Ropeginterferon alfa-2b (trade name BESREMi®), a novel, monopegylated interferon alfa-2b with an extended administration interval, has demonstrated a robust and sustained efficacy in polycythemia vera (PV) patients. Given the similarities in disease pathophysiology and treatment goals, ropeginterferon alfa-2b holds promise as a treatment option for ET. The ROP-ET trial is a prospective, multicenter, single-arm phase III study that includes patients with ET who are intolerant or resistant to, and/or are ineligible for current therapies, such as hydroxyurea (HU), anagrelide (ANA), busulfan (BUS) and pipobroman, leaving these patients with limited treatment options. The primary endpoint is a composite response of hematologic parameters and disease-related symptoms, according to modified European LeukemiaNet (ELN) criteria. Secondary endpoints include improvements in symptoms and quality of life, molecular response and the safety profile of ropeginterferon alfa-2b. Over a 3-year period the trial assesses longer term outcomes, particularly the effects on allele burden and clinical outcomes, such as disease-related symptoms, vascular events and disease progression. No prospective clinical trial data exist for ropeginterferon alfa-2b in the planned ET study population and this study will provide new findings that may contribute to advancing the treatment landscape for ET patients with limited alternatives. Trial registration: EU Clinical Trials Register; EudraCT, 2023-505160-12-00; Registered on October 30, 2023.
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Myeloproliferative neoplasms (MPNs) ; Essential thrombocythemia (ET) ; Ropeginterferon alfa-2b ; ROP-ET ; Phase III ; Disease modification
Publicat a:	Annals of hematology, Vol. 103 Núm. 7 (july 2024) , p. 2299-2310, ISSN 1432-0584

DOI: 10.1007/s00277-024-05665-4
PMID: 38438627

12 p, 919.1 KB

El registre apareix a les col·leccions:
Documents de recerca > Documents dels grups de recerca de la UAB > Centres i grups de recerca (producció científica) > Ciències de la salut i biociències > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP) > Institut de Recerca contra la Leucèmia Josep Carreras
Articles > Articles de recerca
Articles > Articles publicats