Objectives To assess the efficacy and the safety of biologics in a cohort of patients with relapsing polychondritis (RP). Methods We conducted a French multicentre retrospective cohort study including patients treated with biologics for RP. Efficacy outcomes were clinical response (partial or complete) and complete response during the first 6 months of exposure, plus daily corticosteroid dose at 6 months. Other outcomes were adverse drug reactions (ADRs), persistence of biologics and factors associated with a response. Results This study included 41 patients exposed to 105 biologics (tumour-necrosis factor (TNF) inhibitors, n=60; tocilizumab, n=17; anakinra, n=15; rituximab, n=7; abatacept, n=6). Overall response rate during the first 6 months of exposure was 62.9%. Complete response rate was 19.0%. Reduced corticosteroid doses were highly variable among patients. ADRs were mostly infections (n=42). Reasons for biologic withdrawal (73.3%) were insufficient efficacy (34.3%; ranging from 23.5% for tocilizumab to 72.7% for etanercept), loss of efficacy (18.1%) and ADRs (20.9%; mostly for anakinra: 46.7%). Persistence was comparable among biologic classes. Among TNF inhibitors, the highest persistence was observed with adalimumab. Differences in clinical response rates were observed depending on biologics and organ involvement. There were trends towards a lower response rate in cases with associated myelodysplastic syndrome and for a higher response rate for nasal/auricular chondritis, sternal chondritis and concomitant exposure to non-biologic disease-modifying antirheumatic drugs. Conclusions This study describes the efficacy of biologics for refractory RP. However, the number of complete responses was low and there were concerns about the risk of ADRs, particularly infections.

中文翻译：

---

生物制剂治疗复发性多软骨炎的疗效和安全性：法国国家多中心研究

目的 评估生物制剂在复发性多软骨炎 (RP) 患者队列中的有效性和安全性。方法 我们进行了一项法国多中心回顾性队列研究，包括接受生物制剂治疗的 RP 患者。疗效结果是暴露前 6 个月的临床反应（部分或完全）和完全反应，加上 6 个月的每日皮质类固醇剂量。其他结果包括药物不良反应 (ADR)、生物制剂的持久性以及与反应相关的因素。结果 本研究包括 41 名暴露于 105 种生物制剂的患者（肿瘤坏死因子 (TNF) 抑制剂，n=60；托珠单抗，n=17；阿那白滞素，n=15；利妥昔单抗，n=7；阿巴西普，n=6）。暴露的前 6 个月的总体反应率为 62.9%。完全响应率为 19.0%。减少的皮质类固醇剂量在患者中差异很大。ADR 主要是感染（n=42）。生物制剂停药的原因 (73.3%) 是疗效不足（34.3%；范围从托珠单抗的 23.5% 到依那西普的 72.7%）、疗效丧失（18.1%）和 ADR（20.9%；主要是阿那白滞素：46.7%）。生物类别之间的持久性相当。在 TNF 抑制剂中，阿达木单抗观察到的持久性最高。根据生物制剂和器官受累情况，观察到临床反应率的差异。有相关骨髓增生异常综合征病例的反应率较低的趋势，而鼻/耳软骨炎、胸骨软骨炎和同时暴露于非生物缓解疾病的抗风湿药物的反应率较高。结论 本研究描述了生物制剂对难治性 RP 的疗效。然而，完全反应的数量很少，并且存在对 ADR 风险，尤其是感染风险的担忧。