Autoinflammatory & rare systemic diseases
Anakinra improves retention rate of targeted treatments in Erdheim–Chester disease
Authors' conclusion
ECD patients on targeted therapies showed an overall 18-month DRR of 72%, with no significant differences between specific molecules. The combination of anakinra and targeted therapies was associated with a significantly improved drug retention.
Study context
- Population
- Patients with Erdheim-Chester disease receiving targeted therapies, two Italian referral centres60 treatment courses
- Participants
- 52 patients
- Exposure
- Targeted therapies combined with anakinra vs targeted therapies alone
- Anakinran = 16
- Outcome
- Drug retention rate (DRR) of targeted therapies
- Follow-up
- 18 months
Methodological detailstwo Italian referral centres, Drugs used: vemurafenib (72%), cobimetinib (23%), trametinib (3%), dabrafenib (2%)
- Two Italian referral centres
- Drugs used: vemurafenib (72%), cobimetinib (23%), trametinib (3%), dabrafenib (2%)
Main finding
As reported in the abstract
18-month DRR, overall (all treatment courses)
Overall 18-month drug retention rate (DRR), all treatment courses
72%
Observational data: associations, not effects.
Further findings
| Outcome | P value |
|---|---|
| Drug retention (survival analysis), anakinra vs no anakinraDrug retention better in the anakinra group | log-rank test P = 0.040 |
- 18-month DRR, combined anakinra + targeted therapy vs targeted therapy without anakinra · 18 months — 18-month DRR, combination therapy vs comparator · significantly higher 18-month DRR
- 94% vs 65% · P = 0.0251
- 18-month DRR, combined anakinra + targeted therapy vs targeted therapy without anakinra: 16 patients (27%) received combined treatment with anakinra
18-month DRR by agent
- No significant differences between agents
Safety
Adverse reactions as cause of discontinuation
- Adverse reactions were the leading cause of discontinuation
- 76%
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
Anakinra improves retention rate of targeted treatments in Erdheim–Chester disease. Rheumatology (Oxford). Published 15 January 2025. doi:10. 1093/ rheumatology/ keaf023
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