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

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 d...

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Bibliographic Details
Published in:Annals of hematology 2024-07, Vol.103 (7), p.2299-2310
Main Authors: Kiladjian, Jean-Jacques, Marin, Francisca Ferrer, Al-Ali, Haifa Kathrin, Alvarez-Larrán, Alberto, Beggiato, Eloise, Bieniaszewska, Maria, Breccia, Massimo, Buxhofer-Ausch, Veronika, Cerna, Olga, Crisan, Ana-Manuela, Danaila, Catalin Doru, De Stefano, Valerio, Döhner, Konstanze, Empson, Victoria, Gora-Tybor, Joanna, Griesshammer, Martin, Grosicki, Sebastian, Guglielmelli, Paola, García-Gutierrez, Valentin, Heidel, Florian H., Illés, Arpád, Tomuleasa, Ciprian, James, Chloe, Koschmieder, Steffen, Krauth, Maria-Theresa, Krejcy, Kurt, Lazaroiu, Mihaela-Cornelia, Mayer, Jiri, Nagy, Zsolt György, Nicolini, Franck-Emmanuel, Palandri, Francesca, Pappa, Vassiliki, Reiter, Andreas Johannes, Sacha, Tomasz, Schlager, Stefanie, Schmidt, Stefan, Terpos, Evangelos, Unger, Martin, Wölfler, Albert, Cirici, Blanca Xicoy, Klade, Christoph
Format: Article
Language:English
Subjects:
Blood
Bone marrow
Contraindications
Disease
Hematology
Hepatitis
Interferon
Medicine
Medicine & Public Health
Oncology
Pathophysiology
Patients
Phlebotomy
Quality of life
Remission (Medicine)
Skin cancer
Tumors
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Summary: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.
ISSN:0939-5555
1432-0584
1432-0584
DOI:10.1007/s00277-024-05665-4