Long-term effectiveness and safety of baricitinib treatment on refractory or severe juvenile dermatomyositis
Authors' conclusion
We demonstrated the general long-term effectiveness and safety of BAR treatment in patients with rsJDM over a follow-up period of 36 months.
Study context
- Population
- Children with refractory or severe JDM (rsJDM) and poor response to corticosteroids (CS) and immunosuppressants, single centremean follow-up 25.1 months
- Participants
- 27 children
- Outcome
- Long-term effectiveness and safety of BAR add-on therapy; disease response by PRINTO criteria
- Follow-up
- 0, 6, 12, 24 and 36 months
Methodological detailssingle-centre, real-world, baricitinib (BAR) add-on therapy
- Single-centre
- Real-world
- BAR add-on therapy
Main finding
As reported in the abstract
Patients with improvement in skin rashes
77%
21 out of 27
Patients with complete resolution of skin rashes
62%
Observational data: associations, not effects.
Further findings
Disease activity score (DAS) of the skin, median, baseline vs 12 months
- Median DAS skin, baseline vs 12 months (significant decrease)
- 6.0 vs 0.8, P < 0.01
Childhood myositis assessment scale (CMAS) score, median [IQR], baseline vs 12 months
- CMAS, baseline vs 12 months (significant increase)
- 40.5 [30.8, 45.0] vs 52.0 [50.0, 52.0], P < 0.05
- Last observation carried forward for the three non-responders at month 12
MMT-8 score, median [IQR], baseline vs 12 months
- MMT-8, baseline vs 12 months (notable increase)
- 66.0 [47.8, 72.3] vs 79.0 [79.0, 80.0], P = 0.004
- Last observation carried forward for the three non-responders at month 12
Inactive disease (ID) over follow-up · 36 months
- Patients achieving ID over 36-month follow-up
- 70% (19 out of 27)
CS dosage, median [IQR], baseline vs 36 months
- CS dose, baseline vs 36 months
- 0.50 [0.34, 0.89] mg/kg/day vs 0 [0, 0.10] mg/kg/day (P < 0.001)
ID by observation point · 6, 12, 24 and 36 months
- ID at 6 months
- 48.1% (13/27)
- ID at 12 months
- 70.8% (17/24)
- ID at 24 months
- 90.0% (18/20)
- ID at 36 months
- 92.3% (12/13)
CS discontinuation
- Patients who discontinued CS
- One-third (9 out of 27)
Calcinosis (radiographic)
- Improved
- 1
- Stabilised
- 2
ILD
- Improved
- 3
- Stabilised
- 3
Macrophage activation syndrome
- Resolved
- 2
Safety
| Outcome | Values |
|---|---|
| Herpes zoster infection requiring hospitalisation and temporary BAR cessation | BAR 1 events |
Original publication
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