Informace o publikaci

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

Autoři	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
Rok publikování	2024
Druh	Článek v odborném periodiku
Časopis / Zdroj	Annals of hematology
Fakulta / Pracoviště MU	Lékařská fakulta
Citace
www	https://link.springer.com/article/10.1007/s00277-024-05665-4
Doi	http://dx.doi.org/10.1007/s00277-024-05665-4
Klíčová slova	Myeloproliferative neoplasms (MPNs); Essential thrombocythemia (ET); Ropeginterferon alfa-2b; ROP-ET; Phase III; Disease modification
Popis	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.