Sirolimus is effective for refractory/relapsed idiopathic multicentric Castleman disease: A single-center, retrospective study.
Level 4 - case-series / case-control
Single-center retrospective case series without a control group
PubMed 38691144 · doi:10.1007/s00277-024-05783-z
What was done
A single-center retrospective study evaluated the efficacy and safety of a sirolimus-containing regimen in 26 patients with refractory or relapsed idiopathic multicentric Castleman disease (R/R iMCD). The median age at sirolimus initiation was 40.5 years (range 23–60), and patients had received a median of 2 prior lines of therapy (range 1–5).
What was found
Symptomatic and biochemical response occurred in 18 of 26 patients (69.2%). The median time to at least overall partial remission was 1.9 months (range 0.5–14.6). Over a median follow-up of 11.7 months (range 1.6–50.7), the median time to next treatment was 46.2 months. No patients died during follow-up. Sirolimus was reported as well-tolerated with minor adverse effects, though specific adverse event types and incidence figures were not provided in the abstract.
Why it matters
Treatment options for refractory or relapsed iMCD are scarce. This report provides evidence that mTOR inhibition using sirolimus can induce responses in patients who have failed prior therapies.
Limits
The study is limited by its retrospective single-center design, lack of a control or comparator arm, and small sample size (n = 26). The specific co-administered drugs within the sirolimus-containing regimens and detailed safety event rates were not reported in the abstract.