Autoinflammatory & rare systemic diseases
Efficacy and safety of baricitinib in Japanese patients with autoinflammatory type I interferonopathies (NNS/CANDLE, SAVI, And Aicardi-Goutières syndrome (AGS))
Authors' conclusion
Baricitinib may offer a potential therapeutic option for patients with NNS/CANDLE, SAVI, and AGS, with a positive benefit/risk profile in a vulnerable patient population with multiple comorbidities.
Study context
- Population
- Adult and pediatric Japanese patients with NNS/CANDLE, SAVI or AGS treated with baricitinib (JAKi)55.6% female
- Participants
- 9 participants
- Comparison
- NNS/CANDLE vs SAVI vs AGS
- NNS/CANDLEn = 5
- SAVIn = 3
- AGSn = 1
- Outcome
- Change in mean daily diary score (DDS) from baseline
- Follow-up
- End of primary treatment period
Methodological detailsmulticentre, single-arm, 52 weeks
- Multicentre
- Single-arm
- 52 weeks
Main finding
As reported in the abstract
Change in mean DDS from baseline
NNS/CANDLE
0.22
mean
SAVI
0.21
mean
AGS
0.07
mean
Further findings
| Outcome | NNS/CANDLEMean | SAVI | AGSMean |
|---|---|---|---|
| Change in mean DDS from baseline · end of maintenance treatment period | 0.18 | mean 0.27 | 0.04 |
| Mean percent decrease in corticosteroid use | 18.4% | mean 62.9% | — not stated in the abstract |
| Decrease in mean PGA score from baseline | 1.60 | mean 1.33 | 1.0 |
| DeathIntracranial hemorrhage, not related to study drug | — not stated in the abstract | 1 events | — not stated in the abstract |
— not stated in the abstract
- Change in mean daily diary score (DDS) from baseline: decreased in NNS/CANDLE and SAVI; increased in the AGS patient
- Mean percent decrease in corticosteroid use: 3 out of 5 NNS/CANDLE; 1 out of 3 SAVI
Mean daily symptom-specific score (DSSS)
- Improved from baseline
Safety
≥ 1 TEAE
- All patients
Frequent AEs (all patients)
- BK polyomavirus detection
- 3; 33.3%
- Increased blood creatine phosphokinase
- 2; 22.2%
- Anemia
- 2; 22.2%
- Upper respiratory tract infection
- 2; 22.2%
Serious adverse events (all patients)
- Patients with SAEs
- 3 (33.3%)
- SAEs related to study drug
- 1
Original publication
Efficacy and safety of baricitinib in Japanese patients with autoinflammatory type I interferonopathies (NNS/CANDLE, SAVI, And AGS). Pediatr Rheumatol Online J. Published 22 April 2023. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →