Abstract：
BACKGROUND: Chronic rhinosinusitis with nasal polyps (CRSwNP) is an inflammatory disease, which is usually type 2-mediated in the western hemisphere, associated with severe therapeutic and socioeconomic challenges. The first targeted systemic treatment option for severe uncontrolled CRSwNP is a human monoclonal antibody against the interleukin-4 receptor α (IL-4Rα) subunit called dupilumab, which was approved for subcutaneous administration in Germany in October 2019. The purpose of this study is to investigate the efficacy of dupilumab in real life in patients treated with dupilumab in label according to license in our department in 2019-2021.
METHODS: Since October 2019, we have investigated 40 patients (18 men, 22 women) treated with dupilumab in a single-center, retrospective single-arm longitudinal study. The following parameters were collected before treatment (baseline), at 1 month, 4 months, 7 months, 10 months, and 13 months: the Sino-Nasal Outcome Test-22 (SNOT-22), the forced expiratory pressure in 1 s (FEV-1), the olfactometry using Sniffin\' Sticks-12 identification test (SSIT), a visual analog scale of the total complaints, the Nasal Polyp Score (NPS), histologic findings as well as total serum IgE, eosinophilic cationic protein in serum and blood eosinophils.
RESULTS: The average age was 52.7 years (± 15.3). The follow-up period was 13 months. The SNOT-22 average was 60 points (± 22.2) at the first visit, 28.2 points (± 17.1) after 4 months and 20.8 points (± 17.7) after 13 months. The NPS was 4.3 points (± 1.5), after 4 months 2.1 points (± 1.3) and after 13 months 1.4 points (± 1.1). Olfactometry showed 3.2 points (± 3.7) at the baseline, 7.0 points (± 4.0) after 4 months and 7.8 points (± 3.5) after 13 months. The other parameters also improved. Most parameters showed linear dependence in the slopes under therapy (p < 0.001). Adverse side effects were mostly only mild, and no rescue therapy was needed.
CONCLUSIONS: There is a clear improvement in the medical condition and symptoms in all categories mentioned under therapy with dupilumab, as well as a reduction in the need for systemic glucocorticoids and revision surgery as rescue treatment. Our results show that dupilumab tends to be an effective therapy alternative for severe CRSwNP.
METHODS: Since October 2019, we have investigated 40 patients (18 men, 22 women) treated with dupilumab in a single-center, retrospective single-arm longitudinal study. The following parameters were collected before treatment (baseline), at 1 month, 4 months, 7 months, 10 months, and 13 months: the Sino-Nasal Outcome Test-22 (SNOT-22), the forced expiratory pressure in 1 s (FEV-1), the olfactometry using Sniffin\' Sticks-12 identification test (SSIT), a visual analog scale of the total complaints, the Nasal Polyp Score (NPS), histologic findings as well as total serum IgE, eosinophilic cationic protein in serum and blood eosinophils.
RESULTS: The average age was 52.7 years (± 15.3). The follow-up period was 13 months. The SNOT-22 average was 60 points (± 22.2) at the first visit, 28.2 points (± 17.1) after 4 months and 20.8 points (± 17.7) after 13 months. The NPS was 4.3 points (± 1.5), after 4 months 2.1 points (± 1.3) and after 13 months 1.4 points (± 1.1). Olfactometry showed 3.2 points (± 3.7) at the baseline, 7.0 points (± 4.0) after 4 months and 7.8 points (± 3.5) after 13 months. The other parameters also improved. Most parameters showed linear dependence in the slopes under therapy (p < 0.001). Adverse side effects were mostly only mild, and no rescue therapy was needed.
CONCLUSIONS: There is a clear improvement in the medical condition and symptoms in all categories mentioned under therapy with dupilumab, as well as a reduction in the need for systemic glucocorticoids and revision surgery as rescue treatment. Our results show that dupilumab tends to be an effective therapy alternative for severe CRSwNP.
摘要：
背景：慢性鼻窦炎伴鼻息肉（CRSwNP）是一种炎症性疾病，在西半球通常是2型介导的，与严重的治疗和社会经济挑战有关。严重不受控制的CRSwNP的第一个靶向全身治疗选择是针对白介素4受体α（IL-4Rα）亚基的人单克隆抗体，称为dupilumab。它于2019年10月在德国批准用于皮下给药。这项研究的目的是调查dupilumab在现实生活中根据2019-2021年在我们部门的许可使用dupilumab治疗的患者的疗效。
方法：自2019年10月以来，我们调查了40名患者（18名男性，22名女性）在单中心接受dupilumab治疗，回顾性单臂纵向研究。治疗前（基线）收集以下参数，在1个月，4个月,7个月,10个月,和13个月：Sino-Nasal结果测试-22（SNOT-22），1s内的用力呼气压力（FEV-1），使用Sniffin\'Sticks-12识别测试(SSIT)的嗅觉测定法，总投诉的视觉模拟量表，鼻息肉评分（NPS），组织学发现以及总血清IgE，血清和血液嗜酸性粒细胞中的嗜酸性阳离子蛋白。
结果：平均年龄为52.7岁（±15.3）。随访期为13个月。首次就诊时，SNOT-22的平均值为60分（±22.2），4个月后28.2分(±17.1),13个月后20.8分(±17.7)。NPS为4.3分（±1.5），4个月后2.1分（±1.3），13个月后1.4分（±1.1）。嗅觉显示基线时3.2点（±3.7），4个月后7.0分（±4.0），13个月后7.8分（±3.5）。其他参数也有所改善。大多数参数在治疗下显示斜率的线性依赖性(p<0.001)。副作用大多只是轻微的，不需要任何抢救治疗.
结论：在使用dupilumab治疗的所有类别中，医疗状况和症状均有明显改善，以及减少对全身性糖皮质激素和翻修手术作为抢救治疗的需求。我们的结果表明，dupilumab往往是严重CRSwNP的有效治疗替代方案。
方法：自2019年10月以来，我们调查了40名患者（18名男性，22名女性）在单中心接受dupilumab治疗，回顾性单臂纵向研究。治疗前（基线）收集以下参数，在1个月，4个月,7个月,10个月,和13个月：Sino-Nasal结果测试-22（SNOT-22），1s内的用力呼气压力（FEV-1），使用Sniffin\'Sticks-12识别测试(SSIT)的嗅觉测定法，总投诉的视觉模拟量表，鼻息肉评分（NPS），组织学发现以及总血清IgE，血清和血液嗜酸性粒细胞中的嗜酸性阳离子蛋白。
结果：平均年龄为52.7岁（±15.3）。随访期为13个月。首次就诊时，SNOT-22的平均值为60分（±22.2），4个月后28.2分(±17.1),13个月后20.8分(±17.7)。NPS为4.3分（±1.5），4个月后2.1分（±1.3），13个月后1.4分（±1.1）。嗅觉显示基线时3.2点（±3.7），4个月后7.0分（±4.0），13个月后7.8分（±3.5）。其他参数也有所改善。大多数参数在治疗下显示斜率的线性依赖性(p<0.001)。副作用大多只是轻微的，不需要任何抢救治疗.
结论：在使用dupilumab治疗的所有类别中，医疗状况和症状均有明显改善，以及减少对全身性糖皮质激素和翻修手术作为抢救治疗的需求。我们的结果表明，dupilumab往往是严重CRSwNP的有效治疗替代方案。
