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
{"title":"Guidance for stem cell therapy for juvenile systemic sclerosis patients.","authors":"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","doi":"10.1080/1744666X.2025.2474216","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Areas covered: </strong>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.</p><p><strong>Expert opinion: </strong>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.</p>","PeriodicalId":12175,"journal":{"name":"Expert Review of Clinical Immunology","volume":" ","pages":""},"PeriodicalIF":3.9000,"publicationDate":"2025-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Review of Clinical Immunology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/1744666X.2025.2474216","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"IMMUNOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
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.
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.