Andrea Vambutas, Virginia Mullooly, Sharon Weber, Shresh Pathak
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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.</p><p><strong>Study design: </strong>Retrospective case review.</p><p><strong>Setting: </strong>Tertiary referral center.</p><p><strong>Patients/intervention: </strong>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).</p><p><strong>Outcome measures/results: </strong>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.</p><p><strong>Conclusion: </strong>Anakinra seems to be efficacious in some patients with corticosteroid-resistant and corticosteroid-dependent AIED.</p>","PeriodicalId":19732,"journal":{"name":"Otology & Neurotology","volume":" ","pages":"1059-1069"},"PeriodicalIF":2.0000,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Corticosteroid-Resistant and Corticosteroid-Dependent AIED Patients Treated With Anakinra: A Decade of Experience.\",\"authors\":\"Andrea Vambutas, Virginia Mullooly, Sharon Weber, Shresh Pathak\",\"doi\":\"10.1097/MAO.0000000000004553\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objective: </strong>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.</p><p><strong>Study design: </strong>Retrospective case review.</p><p><strong>Setting: </strong>Tertiary referral center.</p><p><strong>Patients/intervention: </strong>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).</p><p><strong>Outcome measures/results: </strong>The anakinra response rate was 46% for cohort A, 83% for cohort B, and 78% for cohort C. 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引用次数: 0
摘要
目的：自身免疫性内耳疾病（AIED）患者如果停止使用皮质类固醇或依赖大剂量皮质类固醇来维持听力，因为他们没有其他治疗方案，因此特别容易受到伤害。我们最初在一项小型开放标签临床试验中证明了anakinra对皮质类固醇抵抗性AIED的疗效，目前正在进行一项更大的2期安慰剂对照临床试验。大量筛选的患者不符合条件或拒绝参与；然而，对于那些我们可以获得保险的人，我们使用阿那白来改善和/或稳定听力。研究设计：回顾性病例回顾。单位：三级转诊中心。患者/干预：从2013年到2023年，共有49名患者代表三个队列接受了anakinra治疗，听力结果随时间推移而随访：队列A，皮质类固醇抵抗的AIED患者（n = 28）；B组：皮质类固醇依赖性AIED患者（n = 12）；C组，参加我们的阿那那试验的AIED患者，听力改善，但无法维持药物后的听力（n = 9）。结果测量/结果：队列A的阿那金反应率为46%，队列B为83%，队列c为78%。在队列A中，通过PTA听力改善测量了积极的持久反应：在这个13例患者的队列中，平均14 dB PTA改善了14只耳朵。如果我们在PTA和WRS领域都采用更保守的改善措施，在PTA应答队列中，8名患者中的8只耳朵的WRS改善了22%。在去除3名表现出WRS上限效应的应答者和3名仅通过WRS改善而被视为无应答者后，总体改善率降至32%，即22名可评估患者中的7名。在队列B中，阳性反应被认为达到听力稳定，同时皮质类固醇剂量减少至少50%。在队列C中，积极的反应是达到听力稳定。结论：阿那金对部分皮质激素抵抗和皮质激素依赖性AIED患者有效。
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.
期刊介绍：
Otology & Neurotology publishes original articles relating to both clinical and basic science aspects of otology, neurotology, and cranial base surgery. As the foremost journal in its field, it has become the favored place for publishing the best of new science relating to the human ear and its diseases. The broadly international character of its contributing authors, editorial board, and readership provides the Journal its decidedly global perspective.
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