IF 3.9 3区 医学 Q2 IMMUNOLOGY
Ivan Foeldvari, Kathryn S Torok, Juliana Furtado E Silva, Christopher P Denton, Jörg Henes, Paulina Horvei, Rosser Franziska, Catherine H Orteu, Suzanne C Li, Clare E Pain, Tamás Constantin, Patricia Costa-Reis, Megan L Curran, Maurizio Cutolo, Bernd Hinrichs, Kim Fliglstone, Susan Maillard, Pia Moinzadeh, Clarissa Pilkington, Linda Schraven, Vanessa Smith
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引用次数: 0

摘要

简介:自体干细胞移植(ASCT)和细胞疗法(CT)是成人和幼年发病型系统性硬化症(jSSc)患者的新兴治疗选择。然而,大多数疗效数据来自成人研究,成人干细胞移植标准是否完全适用于儿童系统性硬化症患者,目前仍不清楚。鉴于儿科患者独特的恢复和组织重塑潜力,成人使用的严格标准需要对儿童进行调整:我们回顾了成人和儿童系统性硬化症患者 ASCT 和 CT 的适应症、患者选择和疗效方面的现有数据。在2023年12月于德国汉堡召开的多学科专家研讨会上,我们就系统性硬化症患者何时考虑ASCT和细胞疗法制定了共识指南:造血干细胞移植和CT有望成为jSSc的治疗选择。我们提出的指南旨在在全球范围内统一纳入标准,提高未来各种治疗方法的疗效可比性。建立一致的纳入/排除标准和移植方案将有助于更好地收集和解读数据,并最终改善jSSc患者的治疗效果和护理。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Guidance for stem cell therapy for juvenile systemic sclerosis patients.

Introduction: Autologous stem cell transplantation (ASCT) and cellular therapies (CTs) are emerging therapeutic options for both adult and juvenile-onset systemic sclerosis (jSSc) patients. However, most efficacy data are derived from adult studies, and it remains unclear whether adult stem cell transplant criteria are fully applicable to pediatric patients with jSSc. Given pediatric patients' unique potential for recovery and tissue remodeling, the stringent criteria used in adults need adaptation for children.

Areas covered: We reviewed the current data on indications, patient selection and outcomes of ASCT and CTs in both adult and pediatric patients with systemic sclerosis. At a multidisciplinary expert workshop held in Hamburg, Germany, in December 2023, we developed consensus guidance on when to consider ASCT and cellular therapies in jSSc.

Expert opinion: HSCT and CT hold promise as treatment options for jSSc. Our proposed guidance aims to standardize inclusion criteria globally, enhancing comparability of outcomes across future procedures. Establishing consistent inclusion/exclusion criteria and transplant protocols will enable better data collection, interpretation, and ultimately improve outcomes and care for jSSc patients.

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来源期刊
CiteScore
7.60
自引率
2.30%
发文量
221
审稿时长
6-12 weeks
期刊介绍: Expert Review of Clinical Immunology (ISSN 1744-666X) provides expert analysis and commentary regarding the performance of new therapeutic and diagnostic modalities in clinical immunology. Members of the International Editorial Advisory Panel of Expert Review of Clinical Immunology are the forefront of their area of expertise. This panel works with our dedicated editorial team to identify the most important and topical review themes and the corresponding expert(s) most appropriate to provide commentary and analysis. All articles are subject to rigorous peer-review, and the finished reviews provide an essential contribution to decision-making in clinical immunology. Articles focus on the following key areas: • Therapeutic overviews of specific immunologic disorders highlighting optimal therapy and prospects for new medicines • Performance and benefits of newly approved therapeutic agents • New diagnostic approaches • Screening and patient stratification • Pharmacoeconomic studies • New therapeutic indications for existing therapies • Adverse effects, occurrence and reduction • Prospects for medicines in late-stage trials approaching regulatory approval • Novel treatment strategies • Epidemiological studies • Commentary and comparison of treatment guidelines Topics include infection and immunity, inflammation, host defense mechanisms, congenital and acquired immunodeficiencies, anaphylaxis and allergy, systemic immune diseases, organ-specific inflammatory diseases, transplantation immunology, endocrinology and diabetes, cancer immunology, neuroimmunology and hematological diseases.
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