Clinicohematologic and molecular response of essential thrombocythemia patients treated with pegylated interferon-α: a multi-center study of the German Study Group-Myeloproliferative Neoplasms (GSG-MPN)

Stegelmann, Frank; Teichmann, Lino L.; Heidel, Florian H.; Crodel, Carl C.; Ernst, Thomas; Kreil, Sebastian; Reiter, Andreas; Otten, Sara; Schauer, Stefanie; Körber, R; Kricheldorf, Kim; Isfort, Susanne; Döhner, Hartmut; Bruemmendorf, Tim H.; Grießhammer, Martin; Döhner, Konstanze; Koschmieder, Steffen

doi:10.1038/s41375-023-01837-9

Cited by 2 publications

(1 citation statement)

References 15 publications

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“…The quantification of JAK2V617F allele burden is a helpful marker of treatment response and a measure of MRD after stem cell transplantation. However, there is a certain degree of overlap, and allelic burden cannot be considered pathognomonic of disease (Rumi et al, 2020;Sørensen et al, 2020;Stegelmann et al, 2023).…”

Section: Jak2mentioning

confidence: 99%

BCR::ABL1-negative myeloproliferative neoplasms in the era of next-generation sequencing

Mroczkowska-Bękarciak,

Wróbel

2023

Front. Genet.

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The classical BCR::ABL1-negative myeloproliferative neoplasms such as polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF) are clonal diseases with the presence of characteristic “driver mutations” in one of the genes: JAK2, CALR, or MPL. The search for mutations in these three genes is required for the diagnosis of MPNs. Nevertheless, the progress that has been made in the field of molecular genetics has opened a new era in medicine. The search for additional mutations in MPNs is helpful in assessing the risk stratification, disease progression, transformation to acute myeloid leukemia (AML), or choosing the right treatment. In some cases, advanced technologies are needed to find a clonal marker of the disease and establish a diagnosis. This review focuses on how the use of new technologies like next-generation sequencing (NGS) helps in the diagnosis of BCR::ABL1-negative myeloproliferative neoplasms.

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Section: Jak2mentioning

confidence: 99%

BCR::ABL1-negative myeloproliferative neoplasms in the era of next-generation sequencing

Mroczkowska-Bękarciak,

Wróbel

2023

Front. Genet.

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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,

Marin,

Al-Ali

et al. 2024

Ann Hematol

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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.

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Clinicohematologic and molecular response of essential thrombocythemia patients treated with pegylated interferon-α: a multi-center study of the German Study Group-Myeloproliferative Neoplasms (GSG-MPN)

Cited by 2 publications

References 15 publications

BCR::ABL1-negative myeloproliferative neoplasms in the era of next-generation sequencing

BCR::ABL1-negative myeloproliferative neoplasms in the era of next-generation sequencing

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

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