New investigational drugs for steroid-refractory acute graft-versus-host disease: a review of the literature.

IF 4.9 2区医学 Q1 PHARMACOLOGY & PHARMACY

Expert opinion on investigational drugs Pub Date : 2024-08-01 Epub Date: 2024-07-09 DOI:10.1080/13543784.2024.2377322

Na Hyun Kim, Mehdi Hamadani, Sameem Abedin

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引用次数: 0

Abstract

Introduction: Steroid-refractory acute graft-versus-host disease (SR-aGVHD) remains a formidable obstacle in the field of allogeneic hematopoietic cell transplantation (allo-HCT), significantly contributing to patient morbidity and mortality. The current therapeutic landscape for SR-aGVHD is limited, often yielding suboptimal results, thereby emphasizing the urgent need for innovative and effective treatments.

Areas covered: In light of the pivotal REACH2 trial, ruxolitinib phosphate, a Janus kinase inhibitor, has gained prominence as the standard treatment for SR-aGVHD. Nevertheless, a considerable number of patients either do not respond to or cannot tolerate this therapy. This review delves into emerging treatments for SR-aGVHD, including mesenchymal stromal cells (MSCs), fecal microbiota transplantation (FMT), CD3/CD7 blockade, neihulizumab, begelomab, tocilizumab, and vedolizumab. While some of these agents have shown encouraging results in early-phase trials, issues such as treatment-related toxicities and inconsistent responses in larger studies highlight the necessity for ongoing research.

Expert opinion: Current trials exploring new agents and combination therapies offer hope for fulfilling the unmet clinical needs in SR-aGVHD, potentially leading to more effective and precise treatment strategies.

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治疗类固醇难治性急性移植物抗宿主病的新研究药物：文献综述。

导言：类固醇难治性急性移植物抗宿主疾病（SR-aGVHD）仍然是异基因造血细胞移植（allo-HCT）领域的一个巨大障碍，严重影响患者的发病率和死亡率。目前SR-aGVHD的治疗方法有限，往往达不到最佳效果，因此迫切需要创新有效的治疗方法：鉴于关键性的 REACH2 试验，Janus 激酶抑制剂磷酸禄索利替尼已成为治疗 SR-aGVHD 的标准疗法。然而，相当多的患者对这种疗法没有反应或无法耐受。本综述深入探讨了SR-aGVHD的新兴治疗方法，包括间充质干细胞（MSCs）、粪便微生物群移植（FMT）、CD3/CD7阻断、内乌珠单抗、贝格罗单抗、托珠单抗和维多珠单抗。虽然其中一些药物在早期试验中取得了令人鼓舞的结果，但治疗相关毒性和大型研究中反应不一致等问题凸显了持续研究的必要性：专家观点：目前探索新药和联合疗法的试验为满足SR-aGVHD未满足的临床需求带来了希望，有可能带来更有效、更精确的治疗策略。

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

Expert opinion on investigational drugs 医学-药学

CiteScore

10.00

自引率

0.00%

发文量

审稿时长

6-12 weeks

期刊介绍： Expert Opinion on Investigational Drugs (ISSN 1354-3784 [print], 1744-7658 [electronic]) is a MEDLINE-indexed, peer-reviewed, international journal publishing review articles and original papers on drugs in preclinical and early stage clinical development, providing expert opinion on the scope for future development. The Editors welcome: Reviews covering preclinical through to Phase II data on drugs or drug classes for specific indications, and their potential impact on future treatment strategies Drug Evaluations reviewing the clinical and pharmacological data on a particular drug Original Research papers reporting the results of clinical investigations on agents that are in Phase I and II clinical trials The audience consists of scientists, managers and decision-makers in the pharmaceutical industry, and others closely involved in R&D.