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

• Authors: KILADJIAN Jean-Jacques; MARIN Francisca Ferrer; AL-ALI Haifa Kathrin; ALVAREZ-LARRAN Alberto; BEGGIATO Eloise; BIENIASZEWSKA Maria; BRECCIA Massimo; BUXHOFER-AUSCH Veronika; CERNA Olga; CRISAN Ana-Manuela; DANAILA Catalin Doru; VALERIO De Stefano; DOEHNER Konstanze; EMPSON Victoria; GORA-TYBOR Joanna; GRIESSHAMMER Martin; GROSICKI Sebastian; GUGLIELMELLI Paola; GARCIA-GUTIERREZ Valentin; HEIDEL Florian H; ILLES Arpad; TOMULEASA Ciprian; JAMES Chloe; KOSCHMIEDER Steffen; KRAUTH Maria-Theresa; KREJCY Kurt; LAZAROIU Mihaela-Cornelia; MAYER Jiří; NAGY Zsolt Gyoergy; NICOLINI Franck-Emmanuel; PALANDRI Francesca; PAPPA Vassiliki; REITER Andreas Johannes; SACHA Tomasz; SCHLAGER Stefanie; SCHMIDT Stefan; TERPOS Evangelos; UNGER Martin; WOELFLER Albert; CIRICI Blanca Xicoy; KLADE Christoph
• Year of publication: 2024
• Type: Article in Periodical
• MU Faculty or unit: Faculty of Medicine
• Web: https://link.springer.com/article/10.1007/s00277-024-05665-4
• Doi: http://dx.doi.org/10.1007/s00277-024-05665-4
• Keywords: Myeloproliferative neoplasms (MPNs); Essential thrombocythemia (ET); Ropeginterferon alfa-2b; ROP-ET; Phase III; Disease modification
• Description: 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.