[Efficacy of iptacopan monotherapy for suboptimal response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria].

IF 0.3 4区医学 Q3 MEDICINE, GENERAL & INTERNAL

Terapevticheskii Arkhiv Pub Date : 2025-02-21 DOI:10.26442/00403660.2025.01.203146

O U Klimova, I K Golubovskaya, Y N Kuznetsov, M V Marchenko, A D Kulagin

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引用次数: 0

Abstract

Aim: To evaluate the hematological response within 4 weeks of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria and suboptimal response to long-term eculizumab therapy.

Materials and methods: The analysis included 8 patients, median age 32 years, with persistent anemia on long-term therapy with eculizumab. The hematological response was assessed when switching to oral monotherapy with iptacopan 200 mg twice daily for 4 weeks.

Results: After 4 weeks of iptacopan therapy, an increase in hemoglobin levels by more than 20 g/l was achieved in 7/8 (88%), and a complete response in 6/8 (75%) patients. The median increase in hemoglobin levels was 32.5 g/l from the initial 85.5 g/l (74-100) to 121.5 g/l (80-141); p=0.00013. Independence from red blood cell transfusions was achieved in 100% of cases. Achieving a hematological response to iptacopan therapy was accompanied by a decrease in the level of absolute reticulocyte count, bilirubin and lactate dehydrogenase, as well as a negative result of the anti-C3d direct antiglobulin test.

Conclusion: The oral complement factor B inhibitor iptacopan is an effective treatment option for paroxysmal nocturnal hemoglobinuria in patients with a suboptimal response to complement inhibitor C5 due to more effective control of extravascular C3-mediated hemolysis.

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[伊普他科泮单药治疗突发性夜间血红蛋白尿患者依曲单抗次优反应的疗效]。

目的：评价阵发性夜间血红蛋白尿患者伊普他科泮单药治疗4周内的血液学反应，以及长期埃珠单抗治疗的次优反应。材料和方法：本研究纳入8例长期接受eculizumab治疗的持续性贫血患者，中位年龄32岁。当切换到口服单药伊普他泮200mg，每日两次，持续4周时，评估血液学反应。结果：伊他科泮治疗4周后，7/8（88%）患者血红蛋白水平升高超过20 g/l， 6/8（75%）患者完全缓解。血红蛋白水平中位数从最初的85.5 g/l（74-100）增加到121.5 g/l(80-141)，增加了32.5 g/l；p = 0.00013。100%的病例实现了红细胞输注的独立性。对伊普他科潘治疗的血液学反应伴随着网状红细胞绝对计数、胆红素和乳酸脱氢酶水平的下降，以及抗c3d直接抗球蛋白试验的阴性结果。结论：补体因子B抑制剂伊普他科泮口服补体因子B抑制剂伊普他科泮对补体抑制剂C5反应不佳的患者可有效控制血管外c3介导的溶血，是治疗突发性夜间血红蛋白尿的有效选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Terapevticheskii Arkhiv 医学-医学：内科

CiteScore

1.40

自引率

33.30%

发文量

171

审稿时长

3-8 weeks

期刊介绍： Терапевтический архив The journal was founded by the prominent Russian therapists M.P. Konchalovsky and G.F. Lang in 1923. Then its editors-in-chief were Professors V.N. Vinogradov and A.G. Gukasyan. Since 1972, E.I. Chazov, Academician of the Russian Academy of Sciences, has been heading the editorial board of the journal. Over 90 years, there have been more than 1000 issues where the authors and editorial staff have done their best for readers to keep abreast of current advances in medical science and practice and for physicians to master the advanced principles of recognition and treatment of a wide spectrum of visceral diseases. The papers published in the journal (editorials, original articles, lectures, reviews, etc.) cover both current scientific achievements and practical experience in diagnosing, treating, and preventing visceral diseases. The authors of publications are not only Russian, but also foreign scientists and physicians. All papers are peer-reviewed by highly qualified Russian specialists. The journal is published monthly. Traditionally, each issue has predominantly certain thematic areas covering individual therapy specializations. Every year, one of the issues is devoted to related problems in practical medicine (allergology and immunology, neurology and psychiatry, obstetrics, oncology, etc.). This all draws the attention of the reading public to the journal. The journal is indexed in RSCI (Russian Science Citation Index), PubMed/Medline, Index Medicus, Scopus/EMBASE, Web of Science Core Collection (Science Citation Index Expanded), Web of Science (Russian Science Citation Index - RSCI, Current Contents Connect, BIOSIS Previews), Google Scholar, Ulrich''s Periodicals Directory. The journal is included in the list of periodicals recommended by the Higher Attestation Committee for publishing the papers containing the basic materials of doctoral and candidate dissertations. By the decision of the Presidium of the Russian Academy of Medical Sciences, the “Therapevticheskiy Arkhiv” was awarded the Botkin medal. It was admitted to the European Association of Sciences Editors (EASE). The journal was honored with the Golden Press Fund decoration at the 13th International Press Professional Exhibition.