Tofacitinib treatment for active dermatomyositis and anti-synthetase syndrome: a prospective cohort pilot study
Authors' conclusion
Tofacitinib demonstrated significant immunologic and clinical effectiveness in DM and ASS patients, reducing key immune cell populations and downregulating immune activation pathways.
Study context
- Population
- Patients with active dermatomyositis (DM) and anti-synthetase syndrome (ASS) with inadequate response to conventional treatments21 completed
- Participants
- 26 patients
- Outcome
- Reduction in T follicular helper (Tfh) cells
- Follow-up
- Week 24
Methodological detailspilot study, single-arm
- Pilot study
- Single-arm
Main finding
As reported in the abstract
Percentage of T follicular helper (Tfh) cells in peripheral blood decreased in 17/21 patients
81.0%
17/21P = 0.003
Observational data: associations, not effects.
Secondary outcomes
Disease activity (DM and ASS)
- Both DM and ASS patients demonstrated significant improvements in disease activity
Th17 cells in peripheral blood mononuclear cells (PBMCs), in vivo
- Significant reductions in Th17 cells
- P = 0.017
In vitro inhibition of T-cell subsets
- Tfh cells
- 2.88 ± 1.13 vs 2.28 ± 0.92, P< 0.001
- Th17 cells
- 1.42 ± 0.92 vs 1.01 ± 0.74, P = 0.016
- Treg cells
- 2.06 ± 1.26 vs 0.98 ± 0.65, P = 0.019
- Tfh17 cells
- 33.38 ± 15.14 vs 30.28 ± 4.89, P = 0.014
Myositis Disease Activity Assessment Tool (MDAAT) scores improved
- MDAAT scores improved in 21 out of 24 patients
International Myositis Assessment and Clinical Studies (IMACS) definition of improvement (DOI) met
- Patients meeting IMACS DOI criteria
- Fifteen (62.5%)
RNA sequencing (RNAseq) (4 patients)
- Significant downregulation of genes associated with 'herpes simplex virus 1 infection' and 'IL-17 signalling' pathway
Safety
Severe AEs necessitating treatment discontinuation
- No severe adverse events necessitated treatment discontinuation
Original publication
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