Prevalence of perennial severe allergic asthma in Italy and effectiveness of omalizumab in its management: PROXIMA - an observational, 2 phase, patient reported outcomes study.

BACKGROUND: We designed the PROXIMA study (Patient-Reported Outcomes and Xolair(®) In the Management of Asthma) to determine the proportion of patients with severe asthma sensitive to perennial allergens, and to evaluate asthma control and treatment adherence up to 12 months in patients treated with omalizumab in Italian population. In addition, an ancillary study was designed to explore protein biomarkers and characterize them in relation to severe allergic asthma and treatment effects by proteomic approach.

METHODS: PROXIMA is an observational, multicenter, cross-sectional and prospective cohort study conducted at 25 centers in Italy, in outpatient settings. The study consists of two phases: 1) a cross-sectional phase plans to enroll 600 patients with severe allergic asthma, in step 4 therapy as per GINA guidelines, aged ≥18 years, needing a step up in therapy, and 2) a longitudinal phase on patients who will start omalizumab add-on therapy per clinician's judgment at baseline visit (approximately 180-240 patients). The primary variable of the cross-sectional phase is the proportion of patients with severe asthma presenting with perennial form of allergy (skin prick test or in vitro test). The primary variable of longitudinal phase is proportion of patients who achieve disease control (assessed by Asthma Control Questionnaire [ACQ]) with omalizumab at 6 months, and maintain it at 12 months. Secondary variables are patient compliance to omalizumab, patient-reported perception of cognitive and emotional impact of the illness, assessed by Brief Illness Perception Questionnaire (Brief IPQ) and the health related quality of life evaluated by the EuroQoL 5D-3 L (EQ-5D-3 L). Safety endpoints will be recorded during the course of the study. Patients participating in the longitudinal phase will be enrolled for ancillary study if they provide additional informed consent. Protein species in complex mixtures will be identified using innovative MudPIT (Multidimensional Protein Identification Technology) method.

CONCLUSIONS: The results of this observational study will provide estimate of patient population allergic to perennial allergens in Italy and information on patient-reported outcomes with omalizumab therapy in a real-world setting. The exploratory proteomic analysis on asthma biomarkers could eventually provide new data to identify responder patients to anti IgE therapy.