Safety and efficacy of empirical interleukin-1 inhibition using anakinra in AA amyloidosis of uncertain aetiology.

OBJECTIVE

AA amyloidosis is a serious complication of persistent inflammation, which, untreated will progress to renal failure and death. Effective suppression of the underlying inflammatory disease is the focus of treatment. However, in approximately 20% of cases the underlying condition remains uncertain, presenting a dilemma as to choice of treatment.

METHODS

We conducted a retrospective study of a cohort of 11 patients diagnosed with AA amyloidosis of unknown aetiology, who had been empirically treated with anakinra.

RESULTS

In anakinra-responders, median pre-treatment SAA was 74 (IQR 34-190) mg/L, and median on-treatment SAA was 6 (4-16) mg/L (p = .0047), with the response having been maintained for a median on-treatment follow-up of 1.8 (1-7.6) years. Six dialysis patients were treated effectively and safely with 100 mg anakinra three times weekly post-dialysis. Four patients remained well on daily anakinra post-renal transplant. Five anakinra-responders showed regression and three showed stabilization of amyloid load on serial SAP scintigraphy.

CONCLUSIONS

This small cohort shows that even in potentially high risk cases with organ damage secondary to AA amyloidosis or in the presence of a renal graft, anakinra, when used appropriately and carefully monitored, has proved remarkably effective and well tolerated. Longer follow-up of this off-label use is required.