Corticosteroid-Resistant and Corticosteroid-Dependent AIED Patients Treated With Anakinra: A Decade of Experience

Objective Patients with autoimmune inner ear disease (AIED) are particularly vulnerable if they stop responding to corticosteroids or if they are dependent on high-dose corticosteroids to maintain hearing, as they have no therapeutic alternatives. We initially demonstrated the efficacy of anakinra in a small open-label clinical trial in corticosteroid-resistant AIED and are conducting a larger phase 2 placebo-controlled clinical trial. A large number of screened patients were ineligible or refused participation; however, for those we could obtain insurance coverage, we have used anakinra to improve and/or stabilize hearing. Study Design Retrospective case review. Setting Tertiary referral center. Patients/intervention A total of 49 patients representing three cohorts were treated with anakinra from 2013 to 2023, and hearing results followed over time: cohort A, corticosteroid-resistant AIED patients (n = 28); cohort B, corticosteroid-dependent AIED patients (n = 12); and cohort C, AIED patients who have participated in our anakinra trials with hearing improvement but were unable to maintain hearing off drug (n = 9). Outcome measures/results The anakinra response rate was 46% for cohort A, 83% for cohort B, and 78% for cohort C. In cohort A, a positive durable response was measured by hearing improvement by PTA: in this cohort of 13 patients, 14 ears improved by an average of 14 dB PTA. If we adopt a more conservative measure of improvement in both the PTA and WRS domains, eight ears in eight patients improved by 22% in WRS in this PTA-responsive cohort. After removing three responders who exhibited a WRS ceiling effect and three patients deemed nonresponders as they improved by WRS only, the overall rate of improvement drops to 32% or 7 of 22 evaluable patients. In cohort B, a positive response was considered to achieve hearing stability, with a concomitant reduction in corticosteroid dose by at least 50%. In cohort C, a positive response was to achieve hearing stability. Conclusion Anakinra seems to be efficacious in some patients with corticosteroid-resistant and corticosteroid-dependent AIED.