Clinical Development Programme of the Innovative Mesenchymal Stromal Cell Product MSC-FFM/MC0518 for Steroid-Refractory Acute Graft-Versus-Host Disease: Design of 2 Randomised Controlled Trials in Adult and Paediatric Patients.

IF 1.9 4区医学 Q3 HEMATOLOGY

Transfusion Medicine and Hemotherapy Pub Date : 2026-02-16 DOI:10.1159/000550469

Robert Zeiser, Halvard Bönig, Elena Osswald, Uwe Pichlmeier, Ann-Kristin Möller, Michael Tribanek, Maria Lazarou-Wild, Renate von der Weth, Lisa-Marie Pfeffermann, Peter Bader

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引用次数: 0

Abstract

Introduction: Acute graft-versus-host disease (aGvHD) is a potentially life-threatening complication that can occur following allogeneic haematopoietic stem cell transplantation. Although corticosteroids remain the standard first-line therapy, a considerable number of patients fail to respond adequately, leading to significant morbidity.

Methods: Mesenchymal stromal cells (MSCs) are being investigated as a therapeutic option in this setting due to their immunomodulating and tissue-regenerative properties. Two ongoing clinical trials - IDUNN (NCT04629833) and BALDER (NCT06075706) - are assessing the role of the innovative product MSC-FFM/MC0518 in managing steroid-refractory aGvHD (SR-aGvHD). In parallel, a dedicated surveillance programme is collecting long-term efficacy and safety data to inform clinical use beyond the controlled trial environment.

Conclusion: The IDUNN and BALDER trials will deliver definitive evidence on the efficacy and safety of MC0518 in SR-aGvHD and may shape future therapeutic strategies.

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治疗类固醇难治性急性移植物抗宿主病的创新型间充质基质细胞产品MSC-FFM/MC0518的临床开发计划：成人和儿童患者的2项随机对照试验设计

急性移植物抗宿主病（aGvHD）是异体造血干细胞移植后可能发生的潜在危及生命的并发症。尽管皮质类固醇仍然是标准的一线治疗，但相当多的患者未能充分反应，导致显著的发病率。方法：间充质间质细胞（MSCs）由于其免疫调节和组织再生特性，正在被研究作为一种治疗选择。两项正在进行的临床试验IDUNN （NCT04629833）和BALDER （NCT06075706）正在评估创新产品MSC-FFM/MC0518在治疗类固醇难治性aGvHD （SR-aGvHD）中的作用。与此同时，一个专门的监测规划正在收集长期疗效和安全性数据，以便为受控试验环境之外的临床使用提供信息。结论：IDUNN和BALDER试验将为MC0518治疗SR-aGvHD的有效性和安全性提供明确的证据，并可能形成未来的治疗策略。

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

Transfusion Medicine and Hemotherapy 医学-免疫学

CiteScore

4.00

自引率

9.10%

发文量

审稿时长

6-12 weeks

期刊介绍： This journal is devoted to all areas of transfusion medicine. These include the quality and security of blood products, therapy with blood components and plasma derivatives, transfusion-related questions in transplantation, stem cell manipulation, therapeutic and diagnostic problems of homeostasis, immuno-hematological investigations, and legal aspects of the production of blood products as well as hemotherapy. Both comprehensive reviews and primary publications that detail the newest work in transfusion medicine and hemotherapy promote the international exchange of knowledge within these disciplines. Consistent with this goal, continuing clinical education is also specifically addressed.