ABSTRACT
Background
Named-patient product sublingual immunotherapy (NPP-SLIT) is widely used in France, yet real-world evidence on patient-perceived benefit remains limited.
Objective
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.
Background
Named-patient product sublingual immunotherapy (NPP-SLIT) is widely used in France, yet real-world evidence on patient-perceived benefit remains limited.
Objective
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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