Autoinflammatory & rare systemic diseases
How to treat VEXAS syndrome: a systematic review on effectiveness and safety of current treatment strategies
Authors' conclusion
Current data on VEXAS treatment are limited and inhomogeneous.
Study context
- Population
- Patients with VEXAS syndrome in published reports (three databases searched)36 publications
- Participants
- 116 patients
- Comparison
- Current treatment strategies for VEXAS syndrome
- Azacytidinen = 36
- Janus kinase inhibitors (JAKi)n = 33
- Tocilizumabn = 15
- Allogeneic stem cell transplantationn = 7
- Anakinran = 5
- Canakinumabn = 2
- GC monotherapyn = 6
- Outcome
- Treatment response: complete (CR), partial (PR) or none (NR), by changes in clinical symptoms and laboratory parameters
Methodological detailsprotocolized, PRISMA, narrative synthesis
- Protocolized
- PRISMA
- Narrative synthesis
Pooled result
As reported in the abstract
Treatment response (CR, PR, NR)
| Group | CR | Partial (PR) | Deaths | NR |
|---|---|---|---|---|
| Azacytidine | 9/36, 25% | 14/36, 38.9% | ||
| JAKi | 11/33, 33% | 9/33, 27.3% | ||
| Tocilizumab | 3/15, 20% | 6/15, 40% | ||
| Allogeneic stem cell transplantation | 6/7, 85.7% | 1 | ||
| Anakinra | 4/5, 80% | 1/5, 20% | ||
| Canakinumab | 1/2, 50% | 1/2, 50% | ||
| GC monotherapy | 1/6, 16.7% | 4/6, 66.7% |
CR
Azacytidine9/36, 25%
JAKi11/33, 33%
Tocilizumab3/15, 20%
Allogeneic stem cell transplantation6/7, 85.7%
Anakinra4/5, 80%
Canakinumab1/2, 50%
GC monotherapy1/6, 16.7%
Partial (PR)
Azacytidine14/36, 38.9%
JAKi9/33, 27.3%
Tocilizumab6/15, 40%
Allogeneic stem cell transplantation— not stated in the abstract
Anakinra— not stated in the abstract
Canakinumab1/2, 50%
GC monotherapy4/6, 66.7%
Deaths
Azacytidine— not stated in the abstract
JAKi— not stated in the abstract
Tocilizumab— not stated in the abstract
Allogeneic stem cell transplantation1
Anakinra— not stated in the abstract
Canakinumab— not stated in the abstract
GC monotherapy— not stated in the abstract
NR
Azacytidine— not stated in the abstract
JAKi— not stated in the abstract
Tocilizumab— not stated in the abstract
Allogeneic stem cell transplantation— not stated in the abstract
Anakinra1/5, 20%
Canakinumab— not stated in the abstract
GC monotherapy— not stated in the abstract
Safety
Adverse events (data available for 67/116, 57.8%)
- Pneumonia
- 12/67, 17.9%
- Other infections
- 9/67, 13.4%
- VTE
- 6/67, 8.9%
- Cytopenias
- 4/67, 5.9%
- Acute graft-vs-host disease
- 4/67, 5.9%
- Chronic graft-vs-host disease
- 2/67, 2.9%
Original publication
How to treat VEXAS syndrome: a systematic review on effectiveness and safety of current treatment strategies. Rheumatology (Oxford). Published 26 May 2023. doi:10.
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