A PIONEERING study has shown that allogeneic haematopoietic stem-cell transplantation (HSCT) could be a life-saving treatment for children with systemic juvenile idiopathic arthritis-related lung disease (sJIA-LD) who do not respond to conventional therapies.
The international study reviewed data from 13 children across the USA and Europe who underwent HSCT after failing an average of six immunosuppressive treatments. The results suggest that HSCT can provide significant improvements in survival and quality of life for this challenging patient group.
At a median follow-up of 16 months, nine of the 13 children achieved a complete response, with no active sJIA symptoms, no need for systemic immunosuppressive therapy, and no supplemental oxygen dependence. All patients engrafted successfully, though complications such as acute graft-versus-host disease, bacteraemia, and cytomegalovirus reactivation were observed.
Four patients died due to severe complications, highlighting the risks associated with this intensive treatment. However, for the majority, HSCT proved transformative, offering a path to recovery from a condition that had previously been untreatable.
“This study highlights HSCT as a potential therapeutic option for children with refractory sJIA-LD, particularly those with worsening oxygen dependence or severe treatment-related complications,” the authors wrote.
The findings open new possibilities for treating severe sJIA-LD, a life-threatening complication of systemic juvenile arthritis, and underscore the need for further research to refine this approach and improve outcomes.
Aleksandra Zurowska, EMJ
Reference
Matt MG. Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study. Lancet Rheumatol. 2024; DOI: 10.1016/S2665-9913(24)00275-3.
