Recombinant interferon alfa in BCR/ABL-negative chronic myeloproliferative neoplasms.

IF 1.1 Q4 ONCOLOGY
Sandy El Bitar, Murat O Arcasoy
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引用次数: 0

Abstract

The treatment landscape for BCR/ABL-negative myeloproliferative neoplasms (MPNs), driven by JAK2, CALR, and MPL mutations, has evolved significantly over the last decade. Recent regulatory approvals in polycythemia vera (PV) include the JAK inhibitor ruxolitinib, and more recently, a novel recombinant interferon alfa-2 (IFN-α) therapeutic agent. Many clinical trials have documented the safety and efficacy of IFN-α therapy in PV and essential thrombocythemia, the classical BCR/ABL-negative MPNs. Used off-label for more than 30 years as a cytoreductive agent, IFN-α therapy promotes significant clinical, hematologic, and molecular responses. In some IFN-α-treated patients, partial or complete reduction of the mutant JAK2 allele burden may lead to a durable measurable residual disease state, owing to the ability of long-term IFN-α therapy to selectively deplete mutant JAK2-harboring hematopoietic stem cells. Pegylated IFN-α forms were developed to improve the drug stability and tolerability of first-generation IFN-α therapeutics. More recently, a novel pegylated IFN-α, ropeginterferon alfa-2b, received approval for PV by the European Medicines Agency and the US Food and Drug Administration in 2019 and 2021, respectively. This article reviews the clinical research and recent advances that led to the first regulatory approval of IFN-α in a BCR/ABL-negative MPN and its future promise as a disease-modifying therapeutic agent.

重组干扰素 alfa 用于 BCR/ABL 阴性慢性骨髓增生性肿瘤。
过去十年来,由 JAK2、CALR 和 MPL 突变驱动的 BCR/ABL 阴性骨髓增殖性肿瘤(MPN)的治疗形势发生了重大变化。最近获得监管部门批准用于治疗真性红细胞增多症(PV)的药物包括 JAK 抑制剂 ruxolitinib,以及最近推出的新型重组干扰素 alfa-2 (IFN-α)治疗剂。许多临床试验都证明了 IFN-α 治疗 PV 和原发性血小板增多症(典型的 BCR/ABL 阴性 MPNs)的安全性和有效性。IFN-α 作为一种细胞再生剂在标签外使用已有 30 多年,可促进显著的临床、血液学和分子反应。在一些接受过IFN-α治疗的患者中,由于长期的IFN-α治疗能够选择性地清除突变的JAK2携带造血干细胞,因此部分或完全减轻突变的JAK2等位基因负担可能会导致持久的可测量残留疾病状态。为了提高第一代 IFN-α 疗法的药物稳定性和耐受性,人们开发了聚乙二醇化 IFN-α。最近,一种新型聚乙二醇化 IFN-α--干扰素 alfa-2b 分别于 2019 年和 2021 年获得欧洲药品管理局和美国食品和药物管理局的 PV 批准。本文回顾了导致IFN-α在BCR/ABL阴性MPN中首次获得监管部门批准的临床研究和最新进展,以及其作为疾病改变治疗药物的未来前景。
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来源期刊
CiteScore
1.10
自引率
0.00%
发文量
99
期刊介绍: Clinical Advances in Hematology & Oncology (CAH&O) is a monthly peer-reviewed journal reaching more than 27,000 hematology and oncology clinicians. CAH&O provides editorial content encompassing a wide array of topics relevant and useful to the fields of oncology and hematology, both separately and together. Content is directed by the strong input of today’s top thought leaders in hematology & oncology, including feature-length review articles, monthly columns consisting of engaging interviews with experts on current issues in solid tumor oncology, hematologic malignancies, hematologic disorders, drug development, and clinical case studies with expert commentary. CAH&O also publishes industry-supported meeting highlights, clinical roundtable monographs, and clinical review supplements.
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