Autologous haematopoiesis stem cell transplantation (AHSCT) for treatment-refractory autoimmune diseases in children
Authors' conclusion
AHSCT was an effective and safe approach for children and adolescents with treatment-refractory autoimmune diseases.
Study context
- Population
- Consecutive children and adolescents with severe, refractory autoimmune diseases (2 SSc, 1 pansclerotic morphoea, 1 eosinophilic fasciitis, 1 JDM, 2 sJIA); median age at AHSCT 10 years (7–19)4 women · 3 men
- Participants
- 7 children
- Outcome
- Progression-free survival
Methodological detailsconsecutive patients
- Consecutive patients
Main finding
As reported in the abstract
Progression-free survival, median
| Group | Value | PFS rate at 1 year | PFS rate at 2 years |
|---|---|---|---|
| AHSCT | median 4.2 (95% CI 0.98–8.3) years | 100% | 77% |
AHSCT
Valuemedian 4.2 (95% CI 0.98–8.3) years
PFS rate at 1 year100%
PFS rate at 2 years77%
Observational data: associations, not effects.
Secondary outcomes
| Outcome | Values |
|---|---|
| Overall survival, median | AHSCT median 17 (95% CI 11.8–22.1) years |
Overall survival, median
- All children survived
Disease activity at last follow-up
- Require ongoing immunotherapy
- 4
- All patients in clinical remission
Safety
| Outcome | Values |
|---|---|
| Infections (HHV6, Candida and Ralstonia sepsis) | AHSCT 3 events |
| Systemic inflammatory response syndrome | AHSCT 1 events |
| New onset secondary autoimmune diseases (autoimmune haemolytic anaemia, Graves' disease) | AHSCT 2 events |
| Breast fibroadenoma | AHSCT 1 events |
| CYC-associated transient renal failure and pericardial effusion | AHSCT 1 events |
| Amenorrhoea/infertility | AHSCT 1 events |
Original publication
Autologous haematopoiesis stem cell transplantation (AHSCT) for treatment-refractory autoimmune diseases in children. RMD Open. Published 14 July 2024. doi:10.
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