ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options
Por:
Kiladjian, JJ, Marin, FF, Al-Ali, HK, Alvarez-Larrán, A, Beggiato, E, Bieniaszewska, M, Breccia, M, Buxhofer-Ausch, V, Cerna, O, Crisan, AM, Danaila, CD, De Stefano, V, Döhner, K, Empson, V, Gora-Tybor, J, Griesshammer, M, Grosicki, S, Guglielmelli, P, García-Gutierrez, V, Heidel, FH, Illés, A, Tomuleasa, C, James, C, Koschmieder, S, Krauth, MT, Krejcy, K, Lazaroiu, MC, Mayer, J, Nagy, ZG, Nicolini, FE, Palandri, F, Pappa, V, Reiter, AJ, Sacha, T, Schlager, S, Schmidt, S, Terpos, E, Unger, M, Wölfler, A, Cirici, BX and Klade, C
Publicada:
1 jul 2024
Ahead of Print:
1 mar 2024
Resumen:
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 (R)), 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.
Filiaciones:
Kiladjian, JJ:
Univ Paris Cite, CIC 1427, Inserm, F-75010 Paris, France
Hop St Louis, AP HP, Ctr Invest Clin, F-75010 Paris, France
Marin, FF:
UCAM IMIB Murcia, Morales Meseguer Univ Gen Hosp, Reg Ctr Blood Donat, CIBERER, Murcia, Spain
Al-Ali, HK:
Univ Hosp Halle Saale, Krukenberg Canc Ctr Halle, Halle, Germany
Alvarez-Larrán, A:
Hosp Clin Barcelona, Dept Hematol, Barcelona, Spain
Beggiato, E:
Univ Hosp City Hlth & Sci Turin, Hosp Molinette, Complex Struct Hematol, Turin, Italy
Bieniaszewska, M:
Med Univ Gdansk, Gdansk, Poland
Breccia, M:
Sapienza Univ Rome, Dept Translat & Precis Med, Rome, Italy
Buxhofer-Ausch, V:
Johannes Kepler Univ Linz, Dept Internal Med Hematol Stem Cell Transplantat, Ordensklinikum Linz Elisabethinen, Linz, Austria
Johannes Kepler Univ Linz, Med Fac, Linz, Austria
Cerna, O:
Univ Hosp Kralovske Vinohrady, Clin Internal Hematol, Prague, Czech Republic
Crisan, AM:
Fundeni Clin Inst, Ctr Hematol & Bone Marrow Transplantat, Bucharest, Romania
Danaila, CD:
Reg Inst Oncol, Dept Clin Hematol, Iasi, Romania
De Stefano, V:
Catholic Univ, Fdn Policlin Gemelli IRCCS, Sect Hematol, Rome, Italy
Döhner, K:
Univ Hosp Ulm, Dept Internal Med 3, Ulm, Germany
Empson, V:
AOP Hlth, Vienna, Austria
Gora-Tybor, J:
Copernicus Mem Hosp, Dept Hematooncol, Lodz, Poland
Med Univ Lodz, Dept Hematol, Lodz, Poland
Griesshammer, M:
Ruhr Univ Bochum, Johannes Wesling Hosp Minden, Dept Oncol & Hematol, Minden, Germany
Grosicki, S:
Med Univ Silesia, Katowice, Poland
Guglielmelli, P:
Careggi Univ Hosp, Dept Hematol, Florence, Italy
García-Gutierrez, V:
Hosp Univ Ramon y Cajal IRYCIS, Madrid, Spain
Univ Alcala, Madrid, Spain
Heidel, FH:
Hannover Med Sch MHH, Clin Hematol Hemostasis Oncol & Stem Cell Transpl, Hannover, Germany
Illés, A:
Univ Debrecen, Fac Med, Dept Internal Med, Div Hematol, Debrecen, Hungary
Tomuleasa, C:
Iuliu Hatieganu Univ Med & Pharm, Ion Chiricuta Inst Oncol, Hematol Dept, Cluj Napoca, Romania
Iuliu Hatieganu Univ Med & Pharm, Medfuture Res Ctr Adv Med, Cluj Napoca, Romania
James, C:
Univ Bordeaux, INSERM, BMC, U1034, F-33600 Pessac, France
Bordeaux Univ Hosp, Lab Hematol, Bordeaux, France
Koschmieder, S:
Rhein Westfal TH Aachen, Fac Med, Dept Hematol Oncik Genistasteol & Stem cell Trans, Med Clin 4, Aachen, Germany
Krauth, MT:
Med Univ Vienna, Dept Internal Med 1, Clin Dept Hematol & Hemostaseol, Vienna, Austria
Krejcy, K:
AOP Hlth, Vienna, Austria
Lazaroiu, MC:
Policlin Diagnost Rapid Brasov, Dept Hematol, Brasov, Romania
Mayer, J:
Univ Hosp Brno, Masaryk Univ, Dept Internal Med Hematol & Oncol, Brno, Czech Republic
Nagy, ZG:
Semmelweis Univ, Dept Internal Med & Hematol, Div Hematol, Budapest, Hungary
Nicolini, FE:
Ctr Leon Berard, Lyon, France
Palandri, F:
IRCCS Azienda Osped Univ Bologna, Bologna, Italy
Ist Ematol Seragnoli, Bologna, Italy
Pappa, V:
Univ Gen Hosp Attikon, Athens, Greece
Reiter, AJ:
Univ Hosp Mannheim, Med Clin Hematol & Internist Oncol 3, Mannheim, Germany
Sacha, T:
Jagiellonian Univ Hosp, Dept Hematol, Krakow, Poland
Schlager, S:
AOP Hlth, Vienna, Austria
Schmidt, S:
Med Univ Innsbruck, Dept Internal Med Hematol & Oncol 5, Innsbruck, Austria
Terpos, E:
Natl & Kapodistrian Univ Athens, Sch Med, Dept Clin Therapeut, Athens, Greece
Unger, M:
AOP Hlth, Vienna, Austria
Wölfler, A:
Med Univ Graz, Dept Internal Med, Clin Div Hematol, Graz, Austria
:
Univ Autonoma Barcelona, Hosp Germans Trias i Pujol, Josep Carreras Leukemia Res Inst, Inst Catala Oncol, Barcelona, Spain
Klade, C:
AOP Hlth, Vienna, Austria
Green Published, hybrid
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