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

J 2024

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

KILADJIAN, Jean-Jacques, Francisca Ferrer MARIN, Haifa Kathrin AL-ALI, Alberto ALVAREZ-LARRAN, Eloise BEGGIATO et. al.

Basic information

Original name

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, Francisca Ferrer MARIN, Haifa Kathrin AL-ALI, Alberto ALVAREZ-LARRAN, Eloise BEGGIATO, Maria BIENIASZEWSKA, Massimo BRECCIA, Veronika BUXHOFER-AUSCH, Olga CERNA, Ana-Manuela CRISAN, Catalin Doru DANAILA, De Stefano VALERIO, Konstanze DOEHNER, Victoria EMPSON, Joanna GORA-TYBOR, Martin GRIESSHAMMER, Sebastian GROSICKI, Paola GUGLIELMELLI, Valentin GARCIA-GUTIERREZ, Florian H HEIDEL, Arpad ILLES, Ciprian TOMULEASA, Chloe JAMES, Steffen KOSCHMIEDER, Maria-Theresa KRAUTH, Kurt KREJCY, Mihaela-Cornelia LAZAROIU, Jiří MAYER (203 Czech Republic, belonging to the institution), Zsolt Gyoergy NAGY, Franck-Emmanuel NICOLINI, Francesca PALANDRI, Vassiliki PAPPA, Andreas Johannes REITER, Tomasz SACHA, Stefanie SCHLAGER, Stefan SCHMIDT, Evangelos TERPOS, Martin UNGER, Albert WOELFLER, Blanca Xicoy CIRICI and Christoph KLADE

Edition

Annals of hematology, New York, Springer Verlag, 2024, 0939-5555

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.500 in 2022

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1007/s00277-024-05665-4

UT WoS

001182326400002

Keywords in English

Myeloproliferative neoplasms (MPNs); Essential thrombocythemia (ET); Ropeginterferon alfa-2b; ROP-ET; Phase III; Disease modification

Abstract

V originále

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.

Citovat

KILADJIAN, Jean-Jacques, Francisca Ferrer MARIN, Haifa Kathrin AL-ALI, Alberto ALVAREZ-LARRAN, Eloise BEGGIATO, Maria BIENIASZEWSKA, Massimo BRECCIA, Veronika BUXHOFER-AUSCH, Olga CERNA, Ana-Manuela CRISAN, Catalin Doru DANAILA, De Stefano VALERIO, Konstanze DOEHNER, Victoria EMPSON, Joanna GORA-TYBOR, Martin GRIESSHAMMER, Sebastian GROSICKI, Paola GUGLIELMELLI, Valentin GARCIA-GUTIERREZ, Florian H HEIDEL, Arpad ILLES, Ciprian TOMULEASA, Chloe JAMES, Steffen KOSCHMIEDER, Maria-Theresa KRAUTH, Kurt KREJCY, Mihaela-Cornelia LAZAROIU, Jiří MAYER, Zsolt Gyoergy NAGY, Franck-Emmanuel NICOLINI, Francesca PALANDRI, Vassiliki PAPPA, Andreas Johannes REITER, Tomasz SACHA, Stefanie SCHLAGER, Stefan SCHMIDT, Evangelos TERPOS, Martin UNGER, Albert WOELFLER, Blanca Xicoy CIRICI and Christoph KLADE. ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options. Annals of hematology. New York: Springer Verlag, 2024, vol. 103, No 7, p. 2299-2310. ISSN 0939-5555. Available from: https://dx.doi.org/10.1007/s00277-024-05665-4.

@article{2418102,
   author = {Kiladjian, JeanandJacques and Marin, Francisca Ferrer and AlandAli, Haifa Kathrin and AlvarezandLarran, Alberto and Beggiato, Eloise and Bieniaszewska, Maria and Breccia, Massimo and BuxhoferandAusch, Veronika and Cerna, Olga and Crisan, AnaandManuela and Danaila, Catalin Doru and Valerio, De Stefano and Doehner, Konstanze and Empson, Victoria and GoraandTybor, Joanna and Griesshammer, Martin and Grosicki, Sebastian and Guglielmelli, Paola and GarciaandGutierrez, Valentin and Heidel, Florian H and Illes, Arpad and Tomuleasa, Ciprian and James, Chloe and Koschmieder, Steffen and Krauth, MariaandTheresa and Krejcy, Kurt and Lazaroiu, MihaelaandCornelia and Mayer, Jiří and Nagy, Zsolt Gyoergy and Nicolini, FranckandEmmanuel and Palandri, Francesca and Pappa, Vassiliki and Reiter, Andreas Johannes and Sacha, Tomasz and Schlager, Stefanie and Schmidt, Stefan and Terpos, Evangelos and Unger, Martin and Woelfler, Albert and Cirici, Blanca Xicoy and Klade, Christoph},
   article_location = {New York},
   article_number = {7},
   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},
   language = {eng},
   issn = {0939-5555},
   journal = {Annals of hematology},
   title = {ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options},
   url = {https://link.springer.com/article/10.1007/s00277-024-05665-4},
   volume = {103},
   year = {2024}
}

TY  - JOUR
ID  - 2418102
AU  - 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
PY  - 2024
TI  - ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options
JF  - Annals of hematology
VL  - 103
IS  - 7
SP  - 2299-2310
EP  - 2299-2310
PB  - Springer Verlag
SN  - 09395555
KW  - Myeloproliferative neoplasms (MPNs)
KW  - Essential thrombocythemia (ET)
KW  - Ropeginterferon alfa-2b
KW  - ROP-ET
KW  - Phase III
KW  - Disease modification
UR  - https://link.springer.com/article/10.1007/s00277-024-05665-4
N2  - 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.
ER  -

KILADJIAN, Jean-Jacques, Francisca Ferrer MARIN, Haifa Kathrin AL-ALI, Alberto ALVAREZ-LARRAN, Eloise BEGGIATO, Maria BIENIASZEWSKA, Massimo BRECCIA, Veronika BUXHOFER-AUSCH, Olga CERNA, Ana-Manuela CRISAN, Catalin Doru DANAILA, De Stefano VALERIO, Konstanze DOEHNER, Victoria EMPSON, Joanna GORA-TYBOR, Martin GRIESSHAMMER, Sebastian GROSICKI, Paola GUGLIELMELLI, Valentin GARCIA-GUTIERREZ, Florian H HEIDEL, Arpad ILLES, Ciprian TOMULEASA, Chloe JAMES, Steffen KOSCHMIEDER, Maria-Theresa KRAUTH, Kurt KREJCY, Mihaela-Cornelia LAZAROIU, Jiří MAYER, Zsolt Gyoergy NAGY, Franck-Emmanuel NICOLINI, Francesca PALANDRI, Vassiliki PAPPA, Andreas Johannes REITER, Tomasz SACHA, Stefanie SCHLAGER, Stefan SCHMIDT, Evangelos TERPOS, Martin UNGER, Albert WOELFLER, Blanca Xicoy CIRICI and Christoph KLADE. ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options. \textit{Annals of hematology}. New York: Springer Verlag, 2024, vol.~103, No~7, p.~2299-2310. ISSN~0939-5555. Available from: https://dx.doi.org/10.1007/s00277-024-05665-4.

Detailed Information on Publication Record