Siddiqui Shahid, Bachert Claus, Chaker Adam M, Han Joseph K, Hellings Peter W, Peters Anju T, Heffler Enrico, Kamat Siddhesh, Zhang Haixin, Nash Scott, Khan Asif H, De Prado Gomez Lucia, Jacob-Nara Juby A, Rowe Paul J, Deniz Yamo
Medical Affairs, Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA.
Upper Airways Research Laboratory and Department of Otorhinolaryngology, Ghent University, Ghent, Belgium.
ERJ Open Res. 2022 Nov 28;8(4). doi: 10.1183/23120541.00085-2022. eCollection 2022 Oct.
Chronic rhinosinusitis with nasal polyps (CRSwNP) is a predominantly type 2 inflammatory disease of the nasal and paranasal sinuses. Dupilumab is a monoclonal antibody that blocks the shared receptor component for interleukin-4 and interleukin-13, which are key and central drivers of type 2 inflammation. In clinical trials, dupilumab significantly improved objective and patient-reported measures of CRSwNP placebo and was well tolerated. Dupilumab is approved in the European Union, USA and Japan as add-on maintenance treatment for adults with inadequately controlled CRSwNP. There exists an important evidence gap between efficacy and effectiveness data for dupilumab in severe CRSwNP. In order to bridge this gap, the AROMA prospective global registry (ClinicalTrials.gov: NCT04959448) was established. AROMA will collect long-term data on the utilisation, effectiveness and safety of dupilumab for CRSwNP treatment in real-world clinical practice. AROMA will enrol approximately 1000 adults starting dupilumab for severe CRSwNP across 120 global sites. Baseline data will include patient demographics, medical/surgical history and presence of type 2 comorbidities. Effectiveness outcome assessments will include objective measures of CRSwNP assessed as part of routine clinical care and various patient-reported questionnaires. Treatment patterns, concomitant medications and long-term safety will also be recorded. Results from AROMA, the first prospective, real-world, global registry to characterise patients with severe CRSwNP starting dupilumab, will provide evidence on the real impact of dupilumab in patients with CRSwNP and complement the data from randomised clinical trials. The registry will also provide evidence on disease progression in patients with CRSwNP, including those with coexisting diseases.
伴鼻息肉的慢性鼻-鼻窦炎（CRSwNP）主要是一种鼻腔和鼻窦的2型炎症性疾病。度普利尤单抗是一种单克隆抗体，可阻断白细胞介素-4和白细胞介素-13的共享受体成分，这两种细胞因子是2型炎症的关键驱动因素。在临床试验中，度普利尤单抗显著改善了CRSwNP的客观指标和患者报告指标，优于安慰剂，且耐受性良好。度普利尤单抗在欧盟、美国和日本被批准作为对控制不佳的CRSwNP成人患者的附加维持治疗。在重度CRSwNP中，度普利尤单抗的疗效和有效性数据之间存在重要的证据差距。为了弥补这一差距，建立了AROMA前瞻性全球注册研究（ClinicalTrials.gov：NCT04959448）。AROMA将收集度普利尤单抗在真实世界临床实践中用于CRSwNP治疗的使用情况、有效性和安全性的长期数据。AROMA将在全球120个地点招募约1000名开始使用度普利尤单抗治疗重度CRSwNP的成人患者。基线数据将包括患者人口统计学信息、医疗/手术史以及2型合并症的存在情况。有效性结局评估将包括作为常规临床护理一部分评估的CRSwNP客观指标以及各种患者报告问卷。还将记录治疗模式、伴随用药情况和长期安全性。AROMA是首个对开始使用度普利尤单抗的重度CRSwNP患者进行特征描述的前瞻性、真实世界、全球注册研究，其结果将提供度普利尤单抗对CRSwNP患者实际影响的证据，并补充随机临床试验的数据。该注册研究还将提供CRSwNP患者疾病进展的证据，包括那些患有合并症的患者。
