Autoinflammatory & rare systemic diseases

Anakinra improves retention rate of targeted treatments in Erdheim–Chester disease

Rheumatology (Oxford)Published Retrospective cohort study

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

OutcomeP value
Drug retention (survival analysis), anakinra vs no anakinraDrug retention better in the anakinra grouplog-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

Composed by AI from the published abstract and checked against it. How Briefs are made →

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