PMID- 34020658
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20210526
IS  - 1476-7961 (Print)
IS  - 1476-7961 (Electronic)
IS  - 1476-7961 (Linking)
VI  - 19
IP  - 1
DP  - 2021 May 21
TI  - Defining type 2 asthma and patients eligible for dupilumab in Italy: a 
      biomarker-based analysis.
PG  - 5
LID - 10.1186/s12948-021-00146-9 [doi]
LID - 5
AB  - BACKGROUND: Asthma is a chronic disease characterized by airway 
      hyperresponsiveness, inflammation and mucus production. In Type 2 asthma, two 
      phenotypic components are often co-expressed (eosinophilic and allergic). 
      Elevated biomarker levels, such as eosinophils (EOS), fraction of exhaled nitric 
      oxide (FeNO) and immunoglobulin E (IgE), are key clinical indicators of Type 2 
      inflammation. Dupilumab has been recently approved for the treatment of 
      uncontrolled severe Type 2 asthma. Type 2 asthma includes allergic and/or 
      eosinophilic phenotypes. The aim of this analysis was to estimate the 
      dupilumab-eligible population in Italy and characterize it by expected biomarker 
      status. METHODS: A 4-step approach was carried out to calculate 
      dupilumab-eligible population. The approach consisted in: (1) estimating the 
      total number of asthma patients in Italy (using 2016-2017 Italian-adapted Global 
      Initiative for Asthma -GINA- guidelines); (2) estimating the number of severe 
      asthma patients with poorly controlled or uncontrolled disease (using the 
      findings of two recent administrative claim analyses conducted in Italy); (3) 
      stratifying the severe uncontrolled population by biomarker levels (EOS, FeNO and 
      IgE) according to the outcomes of the QUEST trial (a clinical study assessing the 
      efficacy of dupilumab in patients with uncontrolled moderate-to-severe asthma; 
      NCT02414854); (4) identifying the sub-populations of severe uncontrolled asthma 
      patients characterised by raised blood EOS and/or FeNO level (thus indicated to 
      receive dupilumab). RESULTS: According to these estimates, about 3.3 million 
      asthmatic patients live in Italy (6.10% of the population). Of them, almost 20 
      thousand (N = 19,960) have uncontrolled severe asthma. Dupilumab-eligible 
      patients would be N = 15,988, corresponding to 80.1% of the total uncontrolled 
      severe population. Most of these patients (89.3%; N = 14,271) have at least an 
      increase of EOS level, while slightly more than half (51.9%; N = 8,303) have 
      raised levels of both biomarkers. Increased FeNO levels without increased EOS are 
      observed less frequently (N = 1,717; 10.7% of the eligible population). 
      CONCLUSIONS: There is a strong rationale for testing all asthma biomarkers during 
      diagnosis and disease follow-up. Given the large availability and the limited 
      costs, these tests are cost-effective tools to detect severe Type 2 asthma, 
      stratify patients by phenotype, and drive appropriate treatment decisions.
FAU - Canonica, Giorgio Walter
AU  - Canonica GW
AD  - Personalized Medicine, Asthma and Allergy, Humanitas Clinical and Research Center 
      IRCCS, Via Manzoni 56, 20089, Rozzano, MI, Italy.
AD  - Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, MI, 
      Italy.
FAU - Blasi, Francesco
AU  - Blasi F
AD  - Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Internal Medicine 
      Department, Respiratory Unit and Cystic Fibrosis Adult Center and Department of 
      Pathophysiology and Transplantation, University of Milan, Milan, Italy.
FAU - Crimi, Nunzio
AU  - Crimi N
AD  - Respiratory Medicine Unit, A.O.U. Policlinico-Vittorio Emanuele, Catania, Italy.
AD  - Department of Clinical and Experimental Medicine, University of Catania, Catania, 
      Italy.
FAU - Paggiaro, Pierluigi
AU  - Paggiaro P
AD  - Department of Surgery, Medicine, Molecular Biology and Critical Care, University 
      of Pisa, Pisa, Italy.
FAU - Papi, Alberto
AU  - Papi A
AD  - Respiratory Medicine Unit, Department of Medical Sciences, University of Ferrara, 
      Ferrara, Italy.
FAU - Fanelli, Francesca
AU  - Fanelli F
AD  - Sanofi S.P.A., Milan, Italy.
FAU - Stassaldi, Annalisa
AU  - Stassaldi A
AD  - Sanofi S.P.A., Milan, Italy.
FAU - Furneri, Gianluca
AU  - Furneri G
AUID- ORCID: 0000-0002-4445-611X
AD  - EBMA Consulting S.R.L., Via per Carpiano 2, 20077, Melegnano, MI, Italy. 
      gianluca.furneri@ebmaconsulting.com.
LA  - eng
PT  - Journal Article
DEP - 20210521
PL  - England
TA  - Clin Mol Allergy
JT  - Clinical and molecular allergy : CMA
JID - 101152195
PMC - PMC8140446
OTO - NOTNLM
OT  - Biomarkers
OT  - Dupilumab
OT  - Italy
OT  - Type 2 asthma
COIS- GF (EBMA Consulting SRL) received consulting fees from Sanofi SpA for conducting 
      the analysis.
EDAT- 2021/05/23 06:00
MHDA- 2021/05/23 06:01
PMCR- 2021/05/21
CRDT- 2021/05/22 05:24
PHST- 2020/07/16 00:00 [received]
PHST- 2021/05/10 00:00 [accepted]
PHST- 2021/05/22 05:24 [entrez]
PHST- 2021/05/23 06:00 [pubmed]
PHST- 2021/05/23 06:01 [medline]
PHST- 2021/05/21 00:00 [pmc-release]
AID - 10.1186/s12948-021-00146-9 [pii]
AID - 146 [pii]
AID - 10.1186/s12948-021-00146-9 [doi]
PST - epublish
SO  - Clin Mol Allergy. 2021 May 21;19(1):5. doi: 10.1186/s12948-021-00146-9.