Lupus & antiphospholipid syndrome
Sirolimus for the treatment of patients with refractory connective tissue disease-related thrombocytopenia: a pilot study
Authors' conclusion
Our results do support sirolimus as an alternative regimen for refractory CTD-ITP patients, including systemic lupus erythematosus and primary SS.
Study context
- Population
- Hospitalised patients with CTD-ITP unresponsive to, or intolerant of, conventional medicationsconsecutively hospitalised
- Participants
- 12 patients
- Outcome
- Changes in platelet count, and overall response according to the ITP International Working Group Criteria
Methodological detailsprospective, Enrolment November 2020 to February 2022
- Prospective
- Enrolment November 2020 to February 2022
Main finding
As reported in the abstract
Response (ITP International Working Group Criteria)
Complete response
6
50%
Partial response
2
16.7%
No response
4
33.3%
Observational data: associations, not effects.
Further findings
Overall response by underlying CTD (subgroups)
- pSS: overall response
- 3 of 4
- SLE: overall response
- 2 of 3
- Overlapping SjD and SLE: complete response at 6 months
- 1 of 2
Safety
- No severe drug-related toxicities were observed
Original publication
Sirolimus for the treatment of patients with refractory connective tissue disease-related thrombocytopenia: a pilot study. Rheumatology (Oxford). Published 20 April 2023. doi:10.
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