Efficacy and safety of thalidomide in children with monogenic autoinflammatory diseases: a single-center, real-world-evidence study
Authors' conclusion
The largest cohort of monogenic AIDs with the treatment of thalidomide demonstrated that thalidomide can help reduce disease activity and inflammation, reduce the dosage of glucocorticoids, and improve clinical outcomes. Thalidomide is relatively safe in monogenic AIDs.
Study context
- Population
- Children with monogenic autoinflammatory diseases (Aicardi-Goutières syndrome (AGS), Blau syndrome, CINCA, haploinsufficiency (HA20), DADA2, familial Mediterranean fever (FMF), TRAPS, PLAID, SAVI), September 2016 to August 202112 completed
- Participants
- 16 children
- Comparison
- Thalidomide for 12 months vs baseline
- Thalidomiden = 16
- Outcome
- Efficacy rate
- Follow-up
- 3 and 12 months
Methodological detailssingle-centre, 3-month run-in period
- Single-centre
- 3-month run-in period
Main finding
As reported in the abstract
Thalidomide
56.3%
Further findings
Clinical and laboratory outcomes among completers · 12 months
- Fever improved
- 12
- Rash improved
- 7
- Stopped GC or other immunosuppressive agents
- 5
- CRP normal
- 8
- ESR normal
- 11
- 12 patients completed the study
Safety
| Outcome | Values |
|---|---|
| Anorexia and nausea | Thalidomide 2 events |
Anorexia and nausea
- No other reported drug-related adverse reactions
Original publication
Efficacy and safety of thalidomide in children with monogenic autoinflammatory diseases: a single-center, real-world-evidence study. Pediatr Rheumatol Online J. Published 17 October 2023. doi:10.
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