Clinical trial NCT05529784: Dupilumab in the Treatment of Severe Uncontrolled CRSwNP: a Multicentre Observational Real-life Study (DUPIREAL)
Official title: Effectiveness of Dupilumab in the Treatment of Severe Uncontrolled CRSwNP: a Multicentre Observational Real-life Study (DUPIREAL)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Chronic Rhinosinusitis With Nasal Polyps
Lead sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sponsor class: OTHER
First posted: 2022-09-07
Enrollment: 600
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: We included all the patients that were referred to Dupilumab therapy, prescribed accordingly to the plan provided by Italian Agency of Drugs.

Brief summary: Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) is a sino-nasal chronic inflammatory disease that strongly affects patients' quality of life, especially in difficult-to-treat cases. In severe uncontrolled CRSwNP, the inflammation is mostly driven by a type 2 inflammatory pathway and its management has been rapidly changing during the last 2 years due to Dupilumab approval. It is a fully human monoclonal antibody that binds the alpha subunit of IL-4 receptors (IL-4Rα type 1 and type 2) to inhibit IL-4 and IL-13 signaling.

So far, randomized clinical trials have assessed efficacy and safety of Dupilumab in a large number of patients, whereas evidences in real life clinical practice are limited to few monocentric series.

Herewith, the investigators present a multicenter, observational nationwide retrospective real-life study with the aim to confirm the effectiveness and the safety of Dupilumab over the first year of treatment in a real life setting.

The primary objective is to evaluate the volumetric reduction of polyps by measuring the variation of total Nasal Polyp Endoscopic Score (NPS).

The secondary objectives are:

* the evaluation of changes in nasal symptoms, olfactory function and nasal obstruction
* the assessment of the patients' quality of life
* the recording of major and minor complications
* the evaluation of the response to the therapy, according to EPOS2020 criteria and EUFOREA2021
* the assessment of efficacy based on concomitant disease (Asthma and ASA triad)
* the evaluation of potential predictors of clinical response to the therapy


Eligibility criteria: Inclusion Criteria:

* confirmed diagnosis of diffuse CRSwNP by endoscopy and CT scan performed at least 6 months prior of the therapy;
* severe disease stage defined by NPS ≥5 or SNOT-22 ≥50;
* inadequate symptom control with INCS therapy;
* failure or intolerance of previous medical treatments (at least 2 cycles of systemic corticosteroid in the last year), and/or failure of previous surgical treatment through nasal endoscopic surgery (ESS), with postoperative complications or no clinical benefit

Exclusion Criteria:

* pregnant women;
* immunosuppressive therapy;
* patients undergoing radio-chemotherapy treatments for cancer in the 12 months before the starting of therapy;
* concomitant long-term corticosteroid therapy for chronic autoimmune disorders

Primary outcomes:
measure: Nasal Polyp Score (NPS)
timeFrame: 12 months
Secondary outcomes:
measure: Nasal Polyp Score (NPS)
timeFrame: 24 months
measure: Nasal congestion score (NCS)
timeFrame: 12 months and 24 months
measure: Sinonasal outcome test (SNOT-22)
timeFrame: 12 months and 24 months
measure: Sniffin' sticks identification test
timeFrame: 12 months and 24 months
measure: Visual Analog Scale (VAS) for nasal congestion
timeFrame: 12 months and 24 months
measure: Visual Analog Scale (VAS) for olfaction loss
timeFrame: 12 months and 24 months
measure: Visual Analog Scale (VAS) for craniofacial pain
timeFrame: 12 months and 24 months
measure: Visual Analog Scale (VAS) for sleeping disorders
timeFrame: 12 months and 24 months
measure: Visual Analog Scale (VAS) for rhinorrhea
timeFrame: 12 months and 24 months
measure: Visual Analog Scale (VAS) for quality of life
timeFrame: 12 months and 24 months
measure: Need for surgery
timeFrame: 12 months and 24 months
measure: Intranasal corticosteroid adherence
timeFrame: 12 months and 24 months
measure: Need for rescue oral corticosteroids
timeFrame: 12 months and 24 months
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: AUSL Bologna
city: Catania
country: Italy
facility: Azienda Ospedaliero-Universitaria Policlinico S. Marco
city: Latina
country: Italy
facility: AUSL Latina
city: Milano
country: Italy
facility: IRCCS Cà Granda Ospedale Maggiore Policlinico
city: Milano
country: Italy
facility: IRCCS San Raffaele
city: Milan
country: Italy
facility: ASST Santi Paolo e Carlo - Presidio San Paolo
city: Modena
country: Italy
facility: Azienda Ospedaliero-Universitaria di Modena
city: Napoli
country: Italy
facility: A.O.R.N. Ospedali dei Colli
city: Napoli
country: Italy
facility: Azienda Ospedaliera Policlinico Federico II
city: Novara
country: Italy
facility: Ospedale maggiore di Novara
city: Orbassano
country: Italy
facility: Ospedale S.Luigi Gonzaga, Università di Torino
city: Padova
country: Italy
facility: Azienda Ospedaliero-Universitaria di Padova
city: Pavia
country: Italy
facility: Policlinico San Matteo-Università di Pavia
city: Pisa
country: Italy
facility: Azienda Ospedaliero-Universitaria Pisana
city: Reggio Emilia
country: Italy
facility: Azienda USL - IRCCS di Reggio Emilia
city: Roma
country: Italy
facility: ASL Roma 5
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A.Gemelli IRCCS
city: Roma
country: Italy
facility: Università La Sapienza
city: Sassari
country: Italy
facility: Azienda Ospedaliero-Universitaria di Sassari
Study officials:
affiliation: Fondazione Policlinico Universitario A. Gemelli, IRCCS
name: Eugenio De Corso, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: