过敏性鼻炎与哮喘患者对NPP-SLIT疗法的感知获益：ERAPP真实世界队列研究初步结果
2026/04/02
摘要
背景：NPP-SLIT疗法在法国应用广泛，但关于患者感知获益的真实世界证据仍然有限。本研究旨在使用患者获益指数及经过验证的患者报告结局指标，评估近期开始NPP-SLIT治疗的患者在12-15个月期间的治疗期望和感知获益。
方法：ERAPP是一项针对IgE介导的呼吸道过敏儿童和成人的前瞻性、多中心、观察性研究。在基线时开始治疗（治疗时间≤6个月）的受试者，在基线、第6个月和第12-15个月完成数字化PROM评估。主要终点是在第12-15个月时PBI ≥ 1的患者比例。次要终点是PROMs的变化；探索性分析考察了更高的PBI阈值及各单项获益目标的达成情况。
结果：在9439名入组者中，4794名为治疗起始者（儿童950名；青少年/成人3844名）。在第12-15个月时，83.8%的儿童和84.0%的青少年/成人达到了PBI ≥ 1。症状负担和鼻炎严重程度（通过T5SS和ARIA评估）的改善超过了已发表的最小重要差异。哮喘控制（ACT）得到改善，而鼻炎控制（ARCT，≥ 12岁者）和日间嗜睡（ESS，成人）的变化低于其各自的最小重要差异。治疗满意度（ESPIA-Q11）有所提高，而从基线到随访期间，治疗依从性（GIRERD）有所下降。近半数患者达到PBI ≥ 2，约五分之一达到PBI ≥ 3。单项分析显示，鼻塞缓解、睡眠改善和整体症状缓解的获益达成率最高；疲劳、情绪和社交方面的改善则较少被报告。儿童报告的获益达成率普遍略高于青少年/成人。
结论：从患者视角来看，NPP-SLIT在两个年龄组中均能提供持续且有临床意义的获益。ERAPP研究支持将PBI作为一种将患者期望与治疗结局相联系、并为以患者为中心的过敏性疾病诊疗提供信息的补充性终点。
Patient-Perceived Benefits of Named-Patient Product Sublingual Immunotherapy in Allergic Rhinitis and Asthma: Primary Results From the ERAPP Real-World Cohort Study
Caimmi D, Abouelfath A, Lassalle R, Lignot-Maleyran S, Bignon E, Blin P, Clark E, Cottet J, Carcaillon-Bentata L, Demoly P.
Abstract
BACKGROUND:Named-patient product sublingual immunotherapy (NPP-SLIT) is widely used in France, yet real-world evidence on patient-perceived benefit remains limited. To assess treatment expectations and patient-perceived benefit over 12-15 months among recent NPP-SLIT initiators using the Patient Benefit Index (PBI) and validated patient-reported outcome measures (PROMs).
METHODS:ERAPP is a prospective, multicenter, observational study in children and adults with IgE-mediated respiratory allergy. Initiators (≤ 6 months on NPP-SLIT at baseline) completed digital PROMs at baseline, Month 6, and Month 12-15. The primary endpoint was the proportion with PBI ≥ 1 at Months 12-15. Secondary endpoints were changes in PROMs; exploratory analyses examined higher PBI thresholds and item-level fulfillment.
RESULTS:Of 9439 enrolled, 4794 were initiators (950 children; 3844 adolescents/adults). At Month 12-15, PBI ≥ 1 was achieved by 83.8% of children and 84.0% of adolescents/adults. Symptom burden (T5SS) and rhinitis severity (ARIA) improved beyond published MIDs. Asthma control (ACT) improved, whereas changes in rhinitis control (ARCT, ≥ 12 years) and daytime sleepiness (ESS, adults) were below their respective MIDs. Treatment satisfaction (ESPIA-Q11) increased, while adherence (GIRERD) decreased from baseline to follow-up. Nearly half reached PBI ≥ 2 and about one-fifth PBI ≥ 3. Item-level analyses showed highest fulfillment for nasal obstruction relief, improved sleep, and overall symptom relief; fatigue, mood, and social aspects were less frequently fulfilled. Children generally reported slightly higher fulfillment than adolescents/adults.
CONCLUSION:NPP-SLIT provides sustained, clinically meaningful benefit from the patient's perspective in both age groups. ERAPP supports the PBI as a complementary endpoint that links patient expectations to outcomes and informs patient-centered allergy care.
背景：NPP-SLIT疗法在法国应用广泛，但关于患者感知获益的真实世界证据仍然有限。本研究旨在使用患者获益指数及经过验证的患者报告结局指标，评估近期开始NPP-SLIT治疗的患者在12-15个月期间的治疗期望和感知获益。
方法：ERAPP是一项针对IgE介导的呼吸道过敏儿童和成人的前瞻性、多中心、观察性研究。在基线时开始治疗（治疗时间≤6个月）的受试者，在基线、第6个月和第12-15个月完成数字化PROM评估。主要终点是在第12-15个月时PBI ≥ 1的患者比例。次要终点是PROMs的变化；探索性分析考察了更高的PBI阈值及各单项获益目标的达成情况。
结果：在9439名入组者中，4794名为治疗起始者（儿童950名；青少年/成人3844名）。在第12-15个月时，83.8%的儿童和84.0%的青少年/成人达到了PBI ≥ 1。症状负担和鼻炎严重程度（通过T5SS和ARIA评估）的改善超过了已发表的最小重要差异。哮喘控制（ACT）得到改善，而鼻炎控制（ARCT，≥ 12岁者）和日间嗜睡（ESS，成人）的变化低于其各自的最小重要差异。治疗满意度（ESPIA-Q11）有所提高，而从基线到随访期间，治疗依从性（GIRERD）有所下降。近半数患者达到PBI ≥ 2，约五分之一达到PBI ≥ 3。单项分析显示，鼻塞缓解、睡眠改善和整体症状缓解的获益达成率最高；疲劳、情绪和社交方面的改善则较少被报告。儿童报告的获益达成率普遍略高于青少年/成人。
结论：从患者视角来看，NPP-SLIT在两个年龄组中均能提供持续且有临床意义的获益。ERAPP研究支持将PBI作为一种将患者期望与治疗结局相联系、并为以患者为中心的过敏性疾病诊疗提供信息的补充性终点。
（中日友好医院呼吸与危重症医学科 沈焜路 摘译 林江涛 审校）
（Allergy. 2026 Mar 8; DOI: 10.1111/all.70270）
（Allergy. 2026 Mar 8; DOI: 10.1111/all.70270）
Patient-Perceived Benefits of Named-Patient Product Sublingual Immunotherapy in Allergic Rhinitis and Asthma: Primary Results From the ERAPP Real-World Cohort Study
Caimmi D, Abouelfath A, Lassalle R, Lignot-Maleyran S, Bignon E, Blin P, Clark E, Cottet J, Carcaillon-Bentata L, Demoly P.
Abstract
BACKGROUND:Named-patient product sublingual immunotherapy (NPP-SLIT) is widely used in France, yet real-world evidence on patient-perceived benefit remains limited. To assess treatment expectations and patient-perceived benefit over 12-15 months among recent NPP-SLIT initiators using the Patient Benefit Index (PBI) and validated patient-reported outcome measures (PROMs).
METHODS:ERAPP is a prospective, multicenter, observational study in children and adults with IgE-mediated respiratory allergy. Initiators (≤ 6 months on NPP-SLIT at baseline) completed digital PROMs at baseline, Month 6, and Month 12-15. The primary endpoint was the proportion with PBI ≥ 1 at Months 12-15. Secondary endpoints were changes in PROMs; exploratory analyses examined higher PBI thresholds and item-level fulfillment.
RESULTS:Of 9439 enrolled, 4794 were initiators (950 children; 3844 adolescents/adults). At Month 12-15, PBI ≥ 1 was achieved by 83.8% of children and 84.0% of adolescents/adults. Symptom burden (T5SS) and rhinitis severity (ARIA) improved beyond published MIDs. Asthma control (ACT) improved, whereas changes in rhinitis control (ARCT, ≥ 12 years) and daytime sleepiness (ESS, adults) were below their respective MIDs. Treatment satisfaction (ESPIA-Q11) increased, while adherence (GIRERD) decreased from baseline to follow-up. Nearly half reached PBI ≥ 2 and about one-fifth PBI ≥ 3. Item-level analyses showed highest fulfillment for nasal obstruction relief, improved sleep, and overall symptom relief; fatigue, mood, and social aspects were less frequently fulfilled. Children generally reported slightly higher fulfillment than adolescents/adults.
CONCLUSION:NPP-SLIT provides sustained, clinically meaningful benefit from the patient's perspective in both age groups. ERAPP supports the PBI as a complementary endpoint that links patient expectations to outcomes and informs patient-centered allergy care.
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