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Updated: Jul 28, 2025

A Protocol for Explant Cultures of IDH1-mutant Diffuse Low-grade Gliomas
Published on: May 9, 2025
在IDH1-或IDH2-突变低级质瘤中使用vorasidenib
Ingo K Mellinghoff1, Martin J van den Bent1, Deborah T Blumenthal1
1From Memorial Sloan Kettering Cancer Center, New York (I.K.M.); the Brain Tumor Center, Erasmus MC Cancer Institute, Rotterdam, the Netherlands (M.J.B.); Tel Aviv Medical Center, Tel Aviv University, Tel Aviv (D.T.B., S.Y.-K.), and the Davidoff Cancer Center, Rabin Medical Center, Petah Tikva (S.Y.-K.) - both in Israel; Sorbonne Université, Institut du Cerveau, Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires la Pitié Salpêtrière-Charles Foix, Paris (M.T.), and Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon, Centre de Recherche en Cancérologie de Lyon, Lyon (F.D.) - both in France; Duke University Medical Center, Durham, NC (K.B.P.); the University of California, San Francisco, San Francisco (J.C.); Huntsman Cancer Institute, University of Utah, Salt Lake City (J.M.); the Royal Marsden Hospital, London (L.W.); Princess Margaret Cancer Centre (W.P.M.), and Sunnybrook Health Sciences Centre (J.R.P.), University of Toronto (W.P.M.) - both in Toronto; the University of Michigan Comprehensive Cancer Center, Ann Arbor (Y.U.); the University of Alabama at Birmingham, Birmingham (B.N.); Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore (M.H.); Lundin Family Brain Tumor Research Center, University Hospital of Lausanne, and the University of Lausanne - both in Lausanne, Switzerland (A.F.H.); Kyoto University Graduate School of Medicine, Kyoto, Japan (Y.A.); Hospital Universitario 12 de Octubre, Madrid (J.M.S.); Universitätsklinikum Heidelberg and the German Cancer Research Center - both in Heidelberg, Germany (W.W.); the University of Turin, Turin, Italy (R.S.); Ohio State University Wexner Medical Center, Columbus (P.G.); Sylvester Comprehensive Cancer Center and the Department of Neurology, University of Miami, Miami (M.F.); University of Texas Southwestern Medical Center, Dallas (E.A.M.); Servier Pharmaceuticals (S.S., D.Z., S.S.P., L.S., I.H.) and Dana-Farber Cancer Institute (P.Y.W.) - both in Boston; and the University of California, Los Angeles, Los Angeles (T.F.C.).
沃拉西迪尼布显著延长了IDH突变2级质瘤患者的无进展生存期. 这种口服治疗也延迟了随后对这一患者群体进行抗癌干预的需要.
科学领域:
- 神经瘤学神经瘤学
- 分子向疗法分子向疗法
- 临床试验研究临床试验研究
背景情况:
- 2级IDH突变性质瘤是侵袭性脑瘤,导致显著的发病率和死亡率.
- 目前对这些质瘤的治疗选择有限,这凸显了对新型治疗策略的需求.
研究的目的:
- 评估vorasidenib在患有残留或复发的2级IDH突变质瘤的患者中的疗效和安全性.
- 为了确定vorasidenib是否改善了与安慰剂相比,在这个患者群体的无进展生存期.
主要方法:
- 进行了一项双盲,随机,安慰剂控制的第三期试验 (INDIGO).
- 331名以前未经治疗的 (除手术外) 2 级IDH突变性质瘤患者接受了口服vorasidenib (40 mg/天) 或安慰剂.
- 主要终点是基于成像的无进展生存期,下一次抗癌干预的时间作为关键的次要终点.
主要成果:
- 沃拉西迪尼布显著改善了中位数无进展生存时间 (27.7个月对11.1个月;P<0.001).
- 在vorasidenib组,下一次抗癌干预的时间也显著延迟 (危险比,0.26;P<0.001).
- 3级或更高的不良事件发生在22.8%的vorasidenib受试者和13.5%的安慰剂受试者中,而氨酸转移酶的升高在vorasidenib手臂中更为频繁.
结论:
- 伏拉西代尼布显著提高了2级IDH突变质瘤患者的无进展生存率.
- 该药物有效地延迟了随后抗癌治疗的需要.
- 沃拉西迪尼布对这种具有挑战性的脑瘤类型来说是一个有前途的治疗选择.
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