荷兰阿姆斯特丹UMC血管内科Erik S.G. Stroes团队研究了前激肽释放酶反义抑制控制遗传性血管水肿的疗效。2020年9月24日，该研究发表在《新英格兰医学杂志》上。

遗传性血管性水肿的特征是反复发作且无法预测的皮下和粘膜肿胀，有可能危及生命。IONIS-PKK-LRx是配体偶联的反义寡核苷酸，被研究人员设计用于受体介导的肝细胞递送。

在一项同情用药的先导研究中，研究组招募了两名患有严重缓激肽介导的血管性水肿的患者，最初每周一次皮下注射未结合的母体药物IONIS-PKKRx，持续12至16周。之后每3至4周以80 mg的剂量接受IONIS-PKK-LRx，持续7至8个月。

在治疗的过程中，血管性水肿的发作率显著降低。

附：英文原文

Title: Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema

Author: Danny M. Cohn, M.D., Ph.D.,, Nicholas J. Viney, B.Sc.,, Lauré M. Fijen, M.D.,, Eugene Schneider, M.D.,, Veronica J. Alexander, Ph.D.,, Shuting Xia, M.S.,, Gwendolyn E. Kaeser, Ph.D.,, Charvi Nanavati, Ph.D.,, Brenda F. Baker, Ph.D.,, Richard S. Geary, Ph.D.,, Marcel Levi, M.D., Ph.D.,, Joost C.M. Meijers, Ph.D.,, and Erik S.G. Stroes, M.D., Ph.D.

Issue&Volume: 2020-09-02

Abstract: SummaryHereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-LRx is a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to hepatocytes. In a compassionate-use pilot study, two patients with severe bradykinin-mediated angioedema were initially administered weekly subcutaneous injections of the unconjugated parent drug, IONIS-PKKRx, for 12 to 16 weeks, after which they received IONIS-PKK-LRx at a dose of 80 mg every 3 to 4 weeks for 7 to 8 months. Treatment was accompanied by a reduction in the angioedema attack rate.

DOI: 10.1056/NEJMoa1915035

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1915035