Autologous haematopoiesis stem cell transplantation (AHSCT) for treatment-refractory autoimmune diseases in children.

ABSTRACT:

Objectives

To evaluate the long-term effectiveness and safety of autologous haematopoiesis stem cell transplantation (AHSCT) for severe, refractory autoimmune diseases in paediatric patients.

Methods

A single-centre study of consecutive children and adolescents with refractory autoimmune diseases undergoing AHSCT was performed. Demographics, clinical, laboratory features, pre-AHSCT medications, disease activity and functional status were captured. The primary outcome was progression-free survival, secondary outcomes included overall survival, disease-specific treatment responses, disease activity at the last follow-up and AHSCT safety.

Results

The study included seven patients: two systemic sclerosis, one pansclerotic morphoea, one eosinophilic fasciitis, one juvenile

SUBMITTER: Satirer O 

PROVIDER: S-EPMC11253738 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature