Systemic treatment of recessive dystrophic epidermolysis bullosa with mesenchymal stromal cells: a scoping review of the literature and conclusions for future clinical research.

Elke Niebergall-Roth, Kathrin Dieter, Markus H Frank, Mark A Kluth
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Abstract

Background: The ability of mesenchymal stromal cells (MSCs) to facilitate regenerative responses in inflamed and injured tissues, coupled with preclinical data suggesting potential to restore defective collagen VII at the dermo-epidermal junction, has raised the hope that MSCs may provide an effective disease-modifying therapy for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).

Methods: We present a descriptive analysis of the clinical research on systemic MSC administration to RDEB patients available in PubMed, including six early-phase studies and one case report, involving 59 patients who received 1-3 intravenous infusions of MSCs from various sources.

Results: Based on 133 MSC infusions, a total of 44 mostly mild adverse events were reported as definitely, possibly or likely related to the study treatment, only two of which led to treatment discontinuation. Improvements were seen in skin manifestations, disease activity, pain, pruritus and quality of life, with considerable heterogeneity in reported outcome variables and measurement tools between studies, and large inter-patient variability within studies.

Conclusions: Although the current evidence base is limited, reflecting the typical challenges of clinical research in rare diseases, the reported results suggest potential treatment benefits for patients and provide a rationale for continuing to pursue this therapeutic approach.

间充质基质细胞治疗隐性萎缩性表皮松解症:文献综述及未来临床研究结论。
背景:间充质基质细胞(MSCs)能够促进炎症和损伤组织的再生反应,而且临床前数据表明间充质基质细胞具有恢复真皮表皮交界处缺陷胶原蛋白VII的潜力,这使人们希望间充质基质细胞能够为隐性萎缩性表皮松解症(RDEB)患者提供一种有效的疾病调节疗法:我们对PubMed上有关RDEB患者全身性间充质干细胞应用的临床研究进行了描述性分析,其中包括6项早期研究和1项病例报告,共有59名患者接受了1-3次不同来源间充质干细胞的静脉输注:在133例间叶干细胞输注中,共有44例轻微不良事件被报告为肯定、可能或很可能与研究治疗有关,其中只有2例导致治疗中止。皮肤表现、疾病活动、疼痛、瘙痒和生活质量均有所改善,但不同研究间报告的结果变量和测量工具存在很大的异质性,研究内部患者间的差异也很大:尽管目前的证据基础有限,反映了罕见病临床研究的典型挑战,但所报告的结果表明治疗可能会给患者带来益处,并为继续采用这种治疗方法提供了依据。
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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