Guidance for stem cell therapy for juvenile systemic sclerosis patients

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.

Keywords: Autologous bone marrow transplantation; CAR–T cell therapy; autologous stem cell transplantation; cellular therapies; indication for transplantation; juvenile systemic scleroderma; juvenile systemic sclerosis.