Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study
Authors' conclusion
Allogeneic HSCT might be a valuable treatment option for patients with refractory sJIA and sJIA-LD
Study context
- Population
- Children with systemic juvenile idiopathic arthritis (sJIA)-related lung disease refractory to immunosuppressive treatment (average of six different treatment agents) who received allogeneic haematopoietic stem-cell transplantation (HSCT), nine hospitals in the USA and Europe, 2018–202210 female, 3 male
- Participants
- 13 children
- Outcome
- Overall outcome: complete response:
- Resolution of sJIA signs without systemic immunomodulatory therapy
- Plus discontinuation of supplemental oxygen
- Follow-up
- Median 16 months (IQR 6-24)
Methodological detailsinternational, multicentre (nine hospitals, USA and Europe), Medical charts reviewed and data extracted using a standardised form
- International
- Multicentre (nine hospitals, USA and Europe)
- Medical charts reviewed and data extracted using a standardised form
Main finding
As reported in the abstract
Complete response in all surviving patients (no active sJIA, no biological therapy or GC use, no supplemental oxygen dependence)
9
Deaths
4
Observational data: associations, not effects.
Further findings
Engraftment
- Secondary graft failure, second transplant from a different donor
- 1
- All patients engrafted
Deaths by cause
- Cytomegalovirus pneumonitis
- 2
- Intracranial haemorrhage
- 1
- Progressive sJIA-LD
- 1
Safety
Post-transplantation complications
- Acute graft-versus-host disease
- 5
- Bacteraemia
- 8
- Cytomegalovirus reactivation
- 6
- Post-transplantation macrophage activation syndrome
- 3
Original publication
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. Published 20 December 2024. doi:10.
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