Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial

IntroductionExtracorporeal photopheresis (ECP) is a viable treatment that slows the progression of chronic lung allograft dysfunction. Despite its immunoregulatory potential, data on extracorporeal photopheresis as an induction therapy remain rather limited.MethodsWe conducted a pilot randomized con...

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Main Authors: Ilaria Righi, Claudio Fenizia, Daria Trabattoni, Mario Nosotti, Giacomo Grisorio, Claudia Vanetti, Sonia di Tella, Cristina Mocellin, Norma Fantini, Daniele Prati, Letizia Corinna Morlacchi, Valeria Rossetti, Francesco Blasi, Mario Clerici, Lorenzo Paolo Rosso
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Language:English
Published: Frontiers Media S.A. 2025-05-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1583460/full
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author Ilaria Righi
Claudio Fenizia
Daria Trabattoni
Mario Nosotti
Mario Nosotti
Giacomo Grisorio
Claudia Vanetti
Claudia Vanetti
Sonia di Tella
Cristina Mocellin
Norma Fantini
Daniele Prati
Letizia Corinna Morlacchi
Valeria Rossetti
Francesco Blasi
Francesco Blasi
Mario Clerici
Mario Clerici
Lorenzo Paolo Rosso
Lorenzo Paolo Rosso
author_facet Ilaria Righi
Claudio Fenizia
Daria Trabattoni
Mario Nosotti
Mario Nosotti
Giacomo Grisorio
Claudia Vanetti
Claudia Vanetti
Sonia di Tella
Cristina Mocellin
Norma Fantini
Daniele Prati
Letizia Corinna Morlacchi
Valeria Rossetti
Francesco Blasi
Francesco Blasi
Mario Clerici
Mario Clerici
Lorenzo Paolo Rosso
Lorenzo Paolo Rosso
author_sort Ilaria Righi
collection DOAJ
description IntroductionExtracorporeal photopheresis (ECP) is a viable treatment that slows the progression of chronic lung allograft dysfunction. Despite its immunoregulatory potential, data on extracorporeal photopheresis as an induction therapy remain rather limited.MethodsWe conducted a pilot randomized controlled study on ECP as induction therapy in cystic fibrosis patients undergoing primary lung transplantation. Primary endpoints included safety, assessed based on the incidence of adverse events, treatment-related toxicity, and procedure-related complication rates; and feasibility, evaluated through the completion rate of scheduled ECP sessions, patient tolerability, and treatment discontinuation rates. Secondary endpoint consisted of an exploratory assessment of efficacy, using a composite measure that included three key components: freedom from biopsy-proven acute rejection within the first 12 months, absence of chronic lung allograft dysfunction at 36 months, and optimal graft function, defined as a predicted forced expiratory volume in the first second ≥ 90% at 36 months. Finally, exploratory endpoints included cell phenotypic and functional analyses, secreted immune protein profiling, and gene expression analysis for mechanistic insights. Patients were randomly assigned to receive either standard immunosuppressive therapy alone or standard therapy plus six sessions of extracorporeal photopheresis, with a follow-up period of 36 months.ResultsAmong 36 cystic fibrosis patients who underwent lung transplantation between 2018 and 2021 and met the eligibility criteria, 21 were randomized (9 to the study group and 12 to the control group). No patients in the treatment group experienced adverse events. The enrollment rate was 61%, and the treatment discontinuation rate was 22%. The clinical composite endpoint was achieved by 28.6% of patients in the treatment group and 16.7% in the control group. Exploratory endpoint analyses revealed significant decreases in pro-inflammatory cytokines, degranulating CD8+ T lymphocytes, and NK cells in the treatment group. Moreover, significant increases in Treg lymphocytes, IL-10-producing NK cells, and anti-inflammatory cytokines appeared to be associated with improved pulmonary function in the treatment group.ConclusionsInduction therapy with extracorporeal photopheresis is safe and feasible in lung transplantation for cystic fibrosis. Some clinical benefits appear to persist for the first 36 months of follow-up. Interestingly, a correlation between immunological modulation induced by extracorporeal photopheresis and pulmonary function was observed.Clinical Trial Registrationhttps://clinicaltrials.gov/study/NCT03500575?cond=NCT03500575&rank=1, identifier NCT03500575.
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spelling doaj-art-4e334af81e0f47f3bc24f6308030ccfc2025-08-20T02:30:59ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-05-011610.3389/fimmu.2025.15834601583460Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trialIlaria Righi0Claudio Fenizia1Daria Trabattoni2Mario Nosotti3Mario Nosotti4Giacomo Grisorio5Claudia Vanetti6Claudia Vanetti7Sonia di Tella8Cristina Mocellin9Norma Fantini10Daniele Prati11Letizia Corinna Morlacchi12Valeria Rossetti13Francesco Blasi14Francesco Blasi15Mario Clerici16Mario Clerici17Lorenzo Paolo Rosso18Lorenzo Paolo Rosso19Thoracic Surgery and Lung Transplant Unit, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyDepartment of Biomedical and Clinical Sciences, University of Milan, Milan, ItalyThoracic Surgery and Lung Transplant Unit, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyUniversity of Milan, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyDepartment of Biomedical and Clinical Sciences, University of Milan, Milan, ItalyDepartment of Psychology, Catholic University of the Sacred Heart, Milan, ItalyDepartment of Transfusion Medicine and Hematology, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Transfusion Medicine and Hematology, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Transfusion Medicine and Hematology, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyRespiratory Unit and Cystic Fibrosis Adult Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyRespiratory Unit and Cystic Fibrosis Adult Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyIRCCS Don C. Gnocchi Foundation, Milan, ItalyThoracic Surgery and Lung Transplant Unit, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Ospedale Maggiore Policlinico, Milan, ItalyDepartment of Pathophysiology and Transplantation, University of Milan, Milan, ItalyIntroductionExtracorporeal photopheresis (ECP) is a viable treatment that slows the progression of chronic lung allograft dysfunction. Despite its immunoregulatory potential, data on extracorporeal photopheresis as an induction therapy remain rather limited.MethodsWe conducted a pilot randomized controlled study on ECP as induction therapy in cystic fibrosis patients undergoing primary lung transplantation. Primary endpoints included safety, assessed based on the incidence of adverse events, treatment-related toxicity, and procedure-related complication rates; and feasibility, evaluated through the completion rate of scheduled ECP sessions, patient tolerability, and treatment discontinuation rates. Secondary endpoint consisted of an exploratory assessment of efficacy, using a composite measure that included three key components: freedom from biopsy-proven acute rejection within the first 12 months, absence of chronic lung allograft dysfunction at 36 months, and optimal graft function, defined as a predicted forced expiratory volume in the first second ≥ 90% at 36 months. Finally, exploratory endpoints included cell phenotypic and functional analyses, secreted immune protein profiling, and gene expression analysis for mechanistic insights. Patients were randomly assigned to receive either standard immunosuppressive therapy alone or standard therapy plus six sessions of extracorporeal photopheresis, with a follow-up period of 36 months.ResultsAmong 36 cystic fibrosis patients who underwent lung transplantation between 2018 and 2021 and met the eligibility criteria, 21 were randomized (9 to the study group and 12 to the control group). No patients in the treatment group experienced adverse events. The enrollment rate was 61%, and the treatment discontinuation rate was 22%. The clinical composite endpoint was achieved by 28.6% of patients in the treatment group and 16.7% in the control group. Exploratory endpoint analyses revealed significant decreases in pro-inflammatory cytokines, degranulating CD8+ T lymphocytes, and NK cells in the treatment group. Moreover, significant increases in Treg lymphocytes, IL-10-producing NK cells, and anti-inflammatory cytokines appeared to be associated with improved pulmonary function in the treatment group.ConclusionsInduction therapy with extracorporeal photopheresis is safe and feasible in lung transplantation for cystic fibrosis. Some clinical benefits appear to persist for the first 36 months of follow-up. Interestingly, a correlation between immunological modulation induced by extracorporeal photopheresis and pulmonary function was observed.Clinical Trial Registrationhttps://clinicaltrials.gov/study/NCT03500575?cond=NCT03500575&rank=1, identifier NCT03500575.https://www.frontiersin.org/articles/10.3389/fimmu.2025.1583460/fulllung transplanationcystic fibrosisphotopheresisimmunomodulationinduction therapy
spellingShingle Ilaria Righi
Claudio Fenizia
Daria Trabattoni
Mario Nosotti
Mario Nosotti
Giacomo Grisorio
Claudia Vanetti
Claudia Vanetti
Sonia di Tella
Cristina Mocellin
Norma Fantini
Daniele Prati
Letizia Corinna Morlacchi
Valeria Rossetti
Francesco Blasi
Francesco Blasi
Mario Clerici
Mario Clerici
Lorenzo Paolo Rosso
Lorenzo Paolo Rosso
Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial
Frontiers in Immunology
lung transplanation
cystic fibrosis
photopheresis
immunomodulation
induction therapy
title Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial
title_full Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial
title_fullStr Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial
title_full_unstemmed Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial
title_short Extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis: a pilot randomized trial
title_sort extracorporeal photopheresis as induction therapy in lung transplantation for cystic fibrosis a pilot randomized trial
topic lung transplanation
cystic fibrosis
photopheresis
immunomodulation
induction therapy
url https://www.frontiersin.org/articles/10.3389/fimmu.2025.1583460/full
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