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Updated: May 23, 2025

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Guidance for stem cell therapy for juvenile systemic sclerosis patients

Ivan Foeldvari1, Kathryn S Torok2, Juliana Silva3

  • 1Hamburg Centre for Pediatric and Adolescent Rheumatology, Schön Klinik Hamburg Eilbek, Hamburg, Germany.

Expert Review of Clinical Immunology
|March 8, 2025
PubMed

Insights

Autologous stem cell transplantation (ASCT) and cellular therapies (CTs) show promise for systemic sclerosis. Guidelines are proposed to adapt adult criteria for pediatric patients with juvenile systemic sclerosis (jSSc) to improve outcomes.

Area of Science:

  • Hematology
  • Immunology
  • Pediatrics

Background:

  • Autologous stem cell transplantation (ASCT) and cellular therapies (CTs) are emerging treatments for systemic sclerosis (SSc).
  • Current efficacy data primarily derive from adult studies, raising questions about applicability to pediatric patients with juvenile-onset SSc (jSSc).
  • Pediatric patients have unique recovery and tissue remodeling potential, necessitating adaptation of adult transplant criteria.

Purpose of the Study:

  • To review current data on ASCT and CTs in adult and pediatric SSc.
  • To develop consensus guidance for considering ASCT and CTs in jSSc.
  • To standardize global inclusion criteria for jSSc transplantation.

Main Methods:

  • Multidisciplinary expert workshop.
  • Review of existing literature on ASCT and CTs for SSc.
  • Development of consensus-based guidance for jSSc.

Main Results:

  • Consensus guidance for ASCT and CTs in jSSc was developed.
  • The guidance addresses indications and patient selection.
  • Standardized criteria aim to improve data comparability and patient care.

Conclusions:

  • ASCT and CTs offer potential therapeutic benefits for jSSc.
  • Standardized inclusion criteria are crucial for consistent outcomes and research.
  • Adapted criteria for pediatric patients are essential for optimizing treatment and care in jSSc.
Abstract

Keywords:
Autologous bone marrow transplantationCAR–T cell therapyautologous stem cell transplantationcellular therapiesindication for transplantationjuvenile systemic sclerodermajuvenile systemic sclerosis

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