Eltrombopag for the treatment of refractory connective tissue disease-related thrombocytopenia: a pilot study of 52 cases
Authors' conclusion
For refractory CTD-ITP patients, Eltrombopag demonstrated significant clinical improvement, safety, and a steroid-sparing effect with prolonged use.
Study context
- Population
- Refractory CTD-ITP patients unresponsive to or intolerant of conventional medicationsmedian follow-up 6 months
- Participants
- 52 participants
- Outcome
- Therapeutic effectiveness (durable overall remission)
Methodological detailssingle-arm
- Single-arm
Main finding
As reported in the abstract
Durable overall remission
Patients achieving durable overall remission
36
90%
Observational data: associations, not effects.
Further findings
Remission rates over time
- Remission rate at month 1
- 67.5%
- Remission rate at month 2
- 87.5%
- Remission rate at month 3
- 97.5%
- Remission rate at month 6
- 95%
Platelet count · within 2 weeks
- Median platelet count reached (significant improvement, p = 0.003)
- 50 × 109/L
Disease activity indices in SLE and pSS
- Disease activity indices reduced in SLE and pSS (p = 0.016)
GC tapering
- Reduced disease activity allowed GC tapering (p = 0.004)
No response and relapse
- No response
- 1
- Relapsed
- 4
Factors associated with efficacy
- Protopathy, comorbidity and prior treatment associated with efficacy
Safety
- Patients with clinically relevant adverse events
- 15 (28.8%)
Original publication
Eltrombopag for the treatment of refractory connective tissue disease-related thrombocytopenia: a pilot study of 52 cases. Arthritis Res Ther. Published 10 January 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →