Lipworth Brian J, Han Joseph K, Desrosiers Martin, Hopkins Claire, Lee Stella E, Mullol Joaquim, Pfaar Oliver, Li Ting, Chen Claudia, Almqvist Gun, Margolis Mary Kay, McLaren Julie, Jagadeesh Shankar, MacKay Jamie, Megally Ayman, Hellqvist Åsa, Mankad Vaishali S, Bahadori Lila, Ponnarambil Sandhia S
Scottish Centre for Respiratory Research, and ENT Rhinology Clinic, Ninewells Hospital and Medical School, University of Dundee, Dundee, United Kingdom.
Department of Otolaryngology, Head and Neck Surgery, Eastern Virginia Medical School, Norfolk.
N Engl J Med. 2025 Mar 27;392(12):1178-1188. doi: 10.1056/NEJMoa2414482. Epub 2025 Mar 1.
Treatment with tezepelumab has been effective for sinonasal symptoms in patients with severe, uncontrolled asthma and a history of chronic rhinosinusitis with nasal polyps, but its efficacy and safety in adults with severe, uncontrolled chronic rhinosinusitis with nasal polyps is unknown.
We randomly assigned adults with physician-diagnosed, symptomatic, severe chronic rhinosinusitis with nasal polyps to receive standard care and either tezepelumab (at a dose of 210 mg) or placebo subcutaneously every 4 weeks for 52 weeks. The coprimary end points were the changes from baseline in the total nasal-polyp score (range, 0 to 4 [for each nostril]; higher scores indicate greater severity) and the mean nasal-congestion score (range, 0 to 3; higher scores indicate greater severity) at week 52. Key secondary end points assessed in the overall population were the loss-of-smell score, the total score on the Sinonasal Outcome Test (SNOT-22; range, 0 to 110; higher scores indicate greater severity), the Lund-Mackay score (range, 0 to 24; higher scores indicate greater severity), the total symptom score (range, 0 to 24; higher scores indicate greater severity), and the first decision to treat with nasal-polyp surgery or use of systemic glucocorticoid therapy, or both, assessed in time-to-event analyses (individual and composite).
In total, 203 patients were assigned to receive tezepelumab and 205 to receive placebo. At week 52, the patients who received tezepelumab had significant improvements in the total nasal-polyp score (mean difference vs. placebo, -2.07; 95% confidence interval [CI], -2.39 to -1.74) and the mean nasal-congestion score (-1.03; 95% CI, -1.20 to -0.86) (P<0.001 for both scores). Tezepelumab significantly improved the loss-of-smell score (mean difference vs. placebo, -1.00; 95% CI, -1.18 to -0.83), SNOT-22 total score (-27.26; 95% CI, -32.32 to -22.21), Lund-Mackay score (-5.72; 95% CI, -6.39 to -5.06), and total symptom score (-6.89; 95% CI, -8.02 to -5.76) (P<0.001 for all scores). Surgery for nasal polyps was indicated in significantly fewer patients in the tezepelumab group (0.5%) than in the placebo group (22.1%) (hazard ratio, 0.02; 95% CI, 0.00 to 0.09); there was significantly less use of systemic glucocorticoids with tezepelumab (5.2%) than with placebo (18.3%) (hazard ratio, 0.12; 95% CI, 0.04 to 0.27) (P<0.001 for both time-to-event analyses).
Tezepelumab therapy led to significantly greater reductions in the size of nasal polyps, the severity of nasal congestion and sinonasal symptoms, and the use of nasal-polyp surgery and systemic glucocorticoids than placebo in adults with severe, uncontrolled chronic rhinosinusitis with nasal polyps. (Funded by AstraZeneca and Amgen; WAYPOINT ClinicalTrials.gov number, NCT04851964.).
对于患有严重、未得到控制的哮喘且有慢性鼻-鼻窦炎伴鼻息肉病史的患者,tezepelumab治疗对鼻窦症状有效,但其在患有严重、未得到控制的慢性鼻-鼻窦炎伴鼻息肉的成人中的疗效和安全性尚不清楚。
我们将经医生诊断为有症状的严重慢性鼻-鼻窦炎伴鼻息肉的成人随机分组,分别接受标准治疗,并每4周皮下注射一次tezepelumab(剂量为210mg)或安慰剂,共治疗52周。共同主要终点为第52周时总鼻息肉评分(范围为0至4[每个鼻孔];分数越高表明病情越严重)和平均鼻充血评分(范围为0至3;分数越高表明病情越严重)相对于基线的变化。在总体人群中评估的关键次要终点包括嗅觉丧失评分、鼻窦结局测试(SNOT-22;范围为0至110;分数越高表明病情越严重)的总分、Lund-Mackay评分(范围为0至24;分数越高表明病情越严重)、总症状评分(范围为0至24;分数越高表明病情越严重),以及在事件发生时间分析(个体和综合分析)中评估的首次决定进行鼻息肉手术或使用全身糖皮质激素治疗或两者兼用的情况。
总共203例患者被分配接受tezepelumab治疗,205例接受安慰剂治疗。在第52周时,接受tezepelumab治疗的患者在总鼻息肉评分(与安慰剂相比,平均差值为-2.07;95%置信区间[CI],-2.39至-1.74)和平均鼻充血评分(-1.03;95%CI,-1.20至-0.86)方面有显著改善(两个评分的P<0.001)。Tezepelumab显著改善了嗅觉丧失评分(与安慰剂相比,平均差值为-1.00;95%CI,-1.18至-0.83)、SNOT-22总分(-27.26;95%CI,-32.32至-22.21)、Lund-Mackay评分(-5.72;95%CI,-6.39至-5.06)和总症状评分(-6.89;95%CI,-8.02至-5.76)(所有评分的P<0.001)。与安慰剂组(22.1%)相比,tezepelumab组中需要进行鼻息肉手术的患者显著减少(0.5%)(风险比,0.02;95%CI,0.00至0.09);与安慰剂(18.3%)相比,使用tezepelumab时全身糖皮质激素的使用显著减少(5.2%)(风险比,0.12;95%CI,0.04至0.27)(两项事件发生时间分析的P<0.001)。
在患有严重、未得到控制的慢性鼻-鼻窦炎伴鼻息肉的成人中,与安慰剂相比,tezepelumab治疗导致鼻息肉大小、鼻充血和鼻窦症状的严重程度显著降低,以及鼻息肉手术和全身糖皮质激素的使用显著减少。(由阿斯利康和安进公司资助;WAYPOINT ClinicalTrials.gov编号,NCT04851964。)