ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options.

Autor: Kiladjian JJ; CIC 1427, Inserm, Université Paris Cité, 75010, Paris, France. jean-jacques.kiladjian@aphp.fr.; Centre d'Investigations Cliniques, AP-HP, Hôpital Saint-Louis, Paris, 75010, France. jean-jacques.kiladjian@aphp.fr., Marin FF; Morales Meseguer University General Hospital, Regional Center of Blood Donation. CIBERER. UCAM. IMIB-Murcia, Murcia, Spain., Al-Ali HK; University Hospital Halle (Saale), Krukenberg Cancer Center Halle, Halle, Germany., Alvarez-Larrán A; Department of Hematology, Hospital Clínic of Barcelona, Barcelona, Spain., Beggiato E; University Hospital City of Health and Science of Turin - Hospital Molinette, Complex Structure of Hematology, Torino, Italy., Bieniaszewska M; Medical University of Gdańsk, Gdańsk, Poland., Breccia M; Department of Translational and Precision Medicine, Sapienza University of Rome, Rome, Italy., Buxhofer-Ausch V; Department of Internal Medicine I for Hematology with Stem Cell Transplantation, Hemostaseology and Medical Oncology, Ordensklinikum Linz Elisabethinen, Johannes Kepler University Linz, Linz, Austria.; Medical Faculty, Johannes Kepler University Linz, Linz, Austria., Cerna O; Clinic of Internal Hematology, University Hospital Kralovske Vinohrady, Prague, Czech Republic., Crisan AM; Fundeni Clinical Institute, Center for Hematology and Bone Marrow Transplantation, București, Romania., Danaila CD; Department of Clinical Hematology, Regional Institute of Oncology, Iasi, Romania., De Stefano V; Fondazione Policlinico Gemelli IRCCS, Section of Hematology, Catholic University, Rome, Italy., Döhner K; Department of Internal Medicine III, University Hospital Ulm, Ulm, Germany., Empson V; AOP Health, Vienna, Austria., Gora-Tybor J; Department of Hematooncology, Copernicus Memorial Hospital, Lodz, Poland.; Department of Hematology, Medical University of Lodz, Lodz, Poland., Griesshammer M; Department of Oncology and Hematology, Ruhr University Bochum, Johannes Wesling Hospital Minden, Minden, Germany., Grosicki S; Medical University of Silesia, Katowice, Poland., Guglielmelli P; Department of Hematology, Careggi University Hospital, Florence, Italy., García-Gutierrez V; Hospital Universitario Ramón y Cajal, Madrid (IRYCIS), Madrid, Spain.; Universidad de Alcalá, Madrid, Spain., Heidel FH; Clinic for Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School (MHH), Hannover, Germany., Illés A; Faculty of Medicine, Department of Internal Medicine, Division of Hematology, University of Debrecen, Debrecen, Hungary., Tomuleasa C; Ion Chiricuta Institute of Oncology, Hematology Department and Medfuture Research Center for Advanced Medicine, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj-Napoca, Romania., James C; University Bordeaux, INSERM, BMC, U1034, F-33600, Pessac, France.; Laboratory of Hematology, Bordeaux University Hospital, Bordeaux, France., Koschmieder S; Faculty of Medicine, Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation (Medical Clinic IV), RWTH Aachen University, Aachen, Germany., Krauth MT; Department of Internal Medicine I, Clinical Department of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria., Krejcy K; AOP Health, Vienna, Austria., Lazaroiu MC; Department of Hematology, Policlinica de Diagnostic Rapid Brasov, Brasov, Romania., Mayer J; University Hospital Brno, Department of Internal Medicine, Hematology and Oncology, Masaryk University, Brno, Czech Republic., Nagy ZG; Department of Internal Medicine and Hematology, Division of Hematology, Semmelweis University, Budapest, Hungary., Nicolini FE; Centre Léon Bérard, Lyon, France., Palandri F; IRCCS Azienda Ospedaliero-Universitaria di Bologna and Istituto di Ematologia Seràgnoli, Bologna, Italy., Pappa V; University General Hospital Attikon, Athens, Greece., Reiter AJ; Medical Clinic III, Hematology and Internistic Oncology, University Hospital Mannheim, Mannheim, Germany., Sacha T; Department of Hematology, Jagiellonian University Hospital, Kraków, Poland., Schlager S; AOP Health, Vienna, Austria., Schmidt S; Department of Internal Medicine V (Hematology and Oncology), Medical University Innsbruck, Innsbruck, Austria., Terpos E; Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece., Unger M; AOP Health, Vienna, Austria., Wölfler A; Department of Internal Medicine, Clinical Divison of Hematology, Medical University Graz, Graz, Austria., Cirici BX; Institut Català d' Oncologia- Hospital Germans Trias i Pujol, Josep Carreras Leukemia Research Institute, Universitat Autònoma de Barcelona, Barcelona, Spain., Klade C; AOP Health, Vienna, Austria.
Jazyk: angličtina
Zdroj: Annals of hematology [Ann Hematol] 2024 Jul; Vol. 103 (7), pp. 2299-2310. Date of Electronic Publication: 2024 Mar 04.
DOI: 10.1007/s00277-024-05665-4
Abstrakt: 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.
(© 2024. The Author(s).)
Databáze: MEDLINE
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