| アイテムタイプ |
学術雑誌論文 / Journal Article(1) |
| 公開日 |
2025-03-17 |
| タイトル |
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タイトル |
A randomized, double-blind, placebo-controlled phase 3 study to assess efficacy and safety of ropeginterferon alfa-2b in patients with early/lower-risk primary myelofibrosis |
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言語 |
en |
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言語 |
eng |
| キーワード |
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言語 |
en |
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キーワード |
Disease-modifying |
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言語 |
en |
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キーワード |
Early/lower risk PMF |
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言語 |
en |
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キーワード |
Placebo controlled phase 3 |
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言語 |
en |
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キーワード |
Pre-fibrotic PMF |
| キーワード |
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言語 |
en |
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キーワード |
Primary myelofibrosis (PMF) |
| キーワード |
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言語 |
en |
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キーワード |
Randomized |
| キーワード |
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言語 |
en |
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キーワード |
Ropeginterfeon alfa-2b |
| 資源タイプ |
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資源タイプ |
journal article |
| アクセス権 |
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アクセス権 |
open access |
| 著者 |
Abu-Zeinah, Ghaith
Qin, Albert
Gill, Harinder
Komatsu, Norio
Mascarenhas, John
Shih, Weichung Joe
Zagrijtschuk, Oleh
Sato, Toshiaki
下田, 和哉
WEKO
7997
e-Rad_Researcher
90311844
| ja |
下田, 和哉
宮崎大学
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| ja-Kana |
シモダ, カズヤ
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| en |
Shimoda, Kazuya
University of Miyazaki
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Search repository
Silver, Richard T.
Mesa, Ruben
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| 抄録 |
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内容記述タイプ |
Abstract |
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内容記述 |
Primary myelofibrosis (PMF) is the most aggressive of the myeloproliferative neoplasms and patients require greater attention and likely require earlier therapeutic intervention. Currently approved treatment options are limited in their selective suppression of clonal proliferation resulting from driver- and coexisting gene mutations. Janus kinase inhibitors are approved for symptomatic patients with higher-risk PMF. Additionally, most ongoing clinical studies focus on patients with higher-risk disease and/or high rates of transfusion dependency. Optimal treatment of early/lower-risk PMF remains to be identified and needs randomized clinical trial evaluations. Pegylated interferon alfa is recommended for symptomatic lower-risk PMF patients based on phase 2 non-randomized studies and expert opinion. Ropeginterferon alfa-2b (ropeg) is a new-generation pegylated interferon-based therapy with favorable pharmacokinetics and safety profiles, requiring less frequent injections than prior formulations. This randomized, double-blind, placebo-controlled phase 3 trial will assess its efficacy and safety in patients with “early/lower-risk PMF”, defined as pre-fibrotic PMF or PMF at low or intermediate-1 risk according to Dynamic International Prognostic Scoring System-plus. Co-primary endpoints include clinically relevant complete hematologic response and symptom endpoint. Secondary endpoints include progression- or event-free survival, molecular response in driver or relevant coexisting gene mutations, bone marrow response, and safety. Disease progression and events are defined based on the International Working Group criteria and well-published reports. 150 eligible patients will be randomized in a 2:1 ratio to receive either ropeg or placebo. Blinded sample size re-estimation is designed. Ropeg will be administered subcutaneously with a tolerable, higher starting-dose regimen. The study will provide important data for the treatment of early/lower-risk PMF for which an anti-clonal, disease-modifying agent is highly needed. |
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言語 |
en |
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内容記述タイプ |
Other |
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内容記述 |
Citation:
Abu-Zeinah, G., Qin, A., Gill, H. et al. A randomized, double-blind, placebo-controlled phase 3 study to assess efficacy and safety of ropeginterferon alfa-2b in patients with early/lower-risk primary myelofibrosis. Ann Hematol 103, 3573–3583 (2024). https://doi.org/10.1007/s00277-024-05912-8 |
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言語 |
en |
| bibliographic_information |
en : Annals of Hematology
巻 103,
号 9,
p. 3573-3583,
発行日 2024
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| 出版者 |
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出版者 |
Springer Nature |
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言語 |
en |
| ISSN |
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収録物識別子タイプ |
ISSN |
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収録物識別子 |
0939-5555 |
| item_10001_relation_14 |
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関連タイプ |
isVersionOf |
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識別子タイプ |
DOI |
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関連識別子 |
https://doi.org/10.1007/s00277-024-05912-8 |
| 権利 |
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権利情報 |
© 2025 Springer Nature |
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言語 |
en |
| 出版タイプ |
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出版タイプ |
VoR |
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