Hereditary Influences on Pulmonary Fibrosis Trajectories (SHIFT)
An Observational, Prospective, Multicenter Study on Hereditary Influences on Pulmonary Fibrosis Trajectories
Studie Overzicht
Toestand
Gedetailleerde beschrijving
Familial pulmonary fibrosis (FPF) is a genetically driven subset of fibrosing interstitial lung diseases (ILDs) characterised by heterogeneous phenotypes, variable clinical trajectories, and limited evidence to guide prognosis and treatment. Although antifibrotic therapies are effective in idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF), data in FPF, particularly from European populations, remain scarce. The SHIFT (Hereditary Influences on Pulmonary Fibrosis Trajectories) study aims to prospectively characterize disease progression, treatment response, and outcomes in a national Italian cohort.
SHIFT is a prospective, multicenter, observational cohort study enrolling adults (≥18 years) with ILD on high-resolution computed tomography and genetic findings consistent with FPF. Participants are recruited from specialized ILD referral centers across Italy and followed every 6 months for up to 5 years. Diagnostic attribution is standardized through multidisciplinary discussion using a structured confidence framework. All treatments are prescribed according to routine clinical practice. The primary endpoint is annual relative decline in forced vital capacity (FVC). Secondary endpoints include longitudinal changes in diffusing capacity (DLCO), treatment effectiveness and safety, mortality, transplant-free survival, cancer incidence, and genotype-phenotype and genotype-treatment response associations. Longitudinal data will be analyzed using mixed-effects models, and time-to-event outcomes using survival methods. Multivariable analyses and sensitivity analyses will address confounding.
Studietype
Inschrijving (Geschat)
Contacten en locaties
Studie Locaties
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Italy
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Rozzano, Italy, Italië, 20089
- Francesco Amati
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Bemonsteringsmethode
Studie Bevolking
Patients with ILDs discussed in the multidisciplinary discussion to define FPF. Cases discussed will be documented using a standard CRF that detailed complete medical history including genetic test, physical examination, laboratory test results, pulmonary function test (PFT) results and, eventually, lung biopsy results and/or bronchoalveolar lavage (BAL) results.
Pre-MDD diagnoses will be based on the referring pulmonologist's diagnosis and current consensus classification for ILDs.
Post-MDD diagnosis will be classified according to Ryerson confidence terminology, with "confident diagnosis" reserved for >90% clinical likelihood or a provisional diagnosis which was categorized as "high confidence" (70-89% likelihood) or "low confidence" (51-69% likelihood). Patients with less than 50% diagnostic confidence level post-MDD will be categorized as "unclassifiable ILD".
Beschrijving
Inclusion Criteria:
- A HRCT scan consistent with ILD diagnosis
- Age over 18 years old
- A genetic test proved variant or a polymorphism consistent with a diagnosis of FPF
- Ability to give informed consent for the inclusion in the study
Exclusion Criteria:
- Patients unable to perform pulmonary function tests
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
Cohorten en interventies
Groep / Cohort |
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Familial pulmonary fibrosis
Eligible participants will be adults (≥18 years) with ILD documented on HRCT and with a positive genetic test, defined as the presence of rare variants or susceptibility polymorphisms consistent with the diagnosis of FPF.
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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annual relative FVC decline over the observation period
Tijdsspanne: Annual for 5 years
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The annual relative decline is defined as the difference between the final and the initial FVC value divided by the initial value, and it will be calculated for each year and for the entire follow-up period.
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Annual for 5 years
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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relative reduced annual FVC decline when compared to FPF patients treated with immunomodulator agents and FPF patients not treated in a 5-years period of FU.
Tijdsspanne: 5 years
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relative reduced annual FVC decline when compared to FPF patients treated with immunomodulator agents and FPF patients not treated in a 5-years period of FU.
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5 years
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mortality
Tijdsspanne: 5 years
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mortality
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5 years
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Annual relative DLCO decline
Tijdsspanne: 5 years
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Annual relative DLCO decline: The annual relative decline is defined as the difference between the final and the initial DLCO value divided by the initial value, and it will be calculated for each year and for the entire follow-up period.
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5 years
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incidence of lung and non-lung cancer
Tijdsspanne: 5 years
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incidence of lung and non-lung cancer
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5 years
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Medewerkers en onderzoekers
Sponsor
Medewerkers
Onderzoekers
- Hoofdonderzoeker: Francesco Amati, MD, Humanitas Research Hospital IRCCS, Rozzano-Milan
Publicaties en nuttige links
Algemene publicaties
- Zhang D, Newton CA. Familial Pulmonary Fibrosis: Genetic Features and Clinical Implications. Chest. 2021 Nov;160(5):1764-1773. doi: 10.1016/j.chest.2021.06.037. Epub 2021 Jun 26.
- Borie R, Kannengiesser C, Antoniou K, Bonella F, Crestani B, Fabre A, Froidure A, Galvin L, Griese M, Grutters JC, Molina-Molina M, Poletti V, Prasse A, Renzoni E, van der Smagt J, van Moorsel CHM. European Respiratory Society statement on familial pulmonary fibrosis. Eur Respir J. 2023 Mar 16;61(3):2201383. doi: 10.1183/13993003.01383-2022. Print 2023 Mar.
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- Prot. Nr.721/25
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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