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1. Idiopathic pulmonary fibrosis: current treatment options and critical appraisal of nintedanib

Author

Bonella F, Stowasser S, and Wollin L

Subjects
Tyrosine kinase, disease progression, treatment outcome, usual interstitial pneumonia, therapeutics, Therapeutics. Pharmacology, RM1-950
Abstract

Francesco Bonella,1 Susanne Stowasser,2 Lutz Wollin3 1Interstitial and Rare Lung Disease Unit, Ruhrlandklinik, University Hospital, University of Duisburg-Essen, Essen, 2Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim am Rhein, 3Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach an der Riss, Germany Abstract: Idiopathic pulmonary fibrosis (IPF) is the most common type of idiopathic interstitial pneumonia and is characterized by a poor prognosis, with an estimated 5-year survival of approximately 20%. Progressive and irreversible lung functional impairment leads to chronic respiratory insufficiency with a severely impaired quality of life. In the last 2 decades, novel treatments for IPF have been developed as a consequence of an increasing understanding of disease pathogenesis and pathobiology. In IPF, injured dysfunctional alveolar epithelial cells promote fibroblast recruitment and proliferation, resulting in scarring of the lung tissue. Recently, pirfenidone and nintedanib have been approved for the treatment of IPF, having shown efficacy to slow functional decline and disease progression. This article focuses on the pharmacologic characteristics and clinical evidence supporting the use of nintedanib, a potent small-molecule tyrosine kinase inhibitor, as therapy for IPF. After introducing the mechanism of action and pharmacokinetics, an overview of the safety and efficacy results from the most recent clinical trials of nintedanib in IPF is presented. Keywords: tyrosine kinase, disease progression, treatment outcome, usual interstitial pneumonia, therapeutics

Published
2015

2. Update on therapeutic management of idiopathic pulmonary fibrosis

Author

Tzouvelekis A, Bonella F, and Spagnolo P

Subjects
Therapeutics. Pharmacology, RM1-950
Abstract

Argyris Tzouvelekis,1 Francesco Bonella,2 Paolo Spagnolo31Department of Internal Medicine, Section of Pulmonary, Critical Care and Sleep Medicine, Yale School of Medicine, New Haven, CT, USA; 2Interstitial and Rare Lung Disease Unit, Ruhrlandklinik, University Hospital, University of Duisburg-Essen, Germany; 3Medical University Clinic, Canton Hospital Baselland and University of Basel, Liestal, SwitzerlandAbstract: Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive diffuse parenchymal lung disease of unknown origin, with a mortality rate exceeding that of many cancers. The diagnostic process is complex and relies on the clinician integrating clinical, laboratory, radiological, and histological data. In the last decade, major advances in our understanding of the pathogenesis of IPF have shifted the paradigm from a primarily inflammatory process evolving to fibrosis to a condition driven by aberrant wound healing following alveolar epithelial cell injury that results in scarring of the lung, architectural distortion, and irreversible loss of function. Improved understanding of disease pathogenesis has led to the identification of several therapeutic targets and the design of high-quality clinical trials evaluating novel compounds. However, the results of these studies have been mostly disappointing, probably due to the plethora of mediators, growth factors, and signaling pathways involved in the fibrotic process. Most recently, pirfenidone and nintedanib, two compounds with pleiotropic anti-fibrotic properties, have been proven effective in reducing functional decline and disease progression in IPF. This is a major breakthrough. Nevertheless, we still have a long way to go. In fact, neither pirfenidone nor nintedanib is a cure for IPF, and most patients continue to progress despite treatment. As such, comprehensive care of patients with IPF, including management of concomitant conditions and physical debility, as well as timely referral for lung transplantation, remains essential. Several agents with a high potential are currently being tested, and many more are ready for clinical trials. Their completion is critical for achieving the ultimate goal of curing patients with IPF.Keywords: pulmonary fibrosis, therapy, nintedanib, pirfenidone, treatment

Published
2015

3. Defining anti-synthetase syndrome: a systematic literature review

Author

Zanframundo, G, Faghihi-Kashani, S, Scire, C, Bonella, F, Corte, T, Doyle, T, Fiorentino, D, Gonzalez-Gay, M, Hudson, M, Kuwana, M, Lundberg, I, Mammen, A, Mchugh, N, Miller, F, Monteccucco, C, Oddis, C, Rojas-Serrano, J, Schmidt, J, Selva-O'Callaghan, A, Werth, V, Sakellariou, G, Aggarwal, R, Cavagna, L, Zanframundo G., Faghihi-Kashani S., Scire C. A., Bonella F., Corte T. J., Doyle T. J., Fiorentino D., Gonzalez-Gay M. A., Hudson M., Kuwana M., Lundberg I. E., Mammen A., McHugh N., Miller F. W., Monteccucco C., Oddis C. V., Rojas-Serrano J., Schmidt J., Selva-O'Callaghan A., Werth V. P., Sakellariou G., Aggarwal R., Cavagna L., Zanframundo, G, Faghihi-Kashani, S, Scire, C, Bonella, F, Corte, T, Doyle, T, Fiorentino, D, Gonzalez-Gay, M, Hudson, M, Kuwana, M, Lundberg, I, Mammen, A, Mchugh, N, Miller, F, Monteccucco, C, Oddis, C, Rojas-Serrano, J, Schmidt, J, Selva-O'Callaghan, A, Werth, V, Sakellariou, G, Aggarwal, R, Cavagna, L, Zanframundo G., Faghihi-Kashani S., Scire C. A., Bonella F., Corte T. J., Doyle T. J., Fiorentino D., Gonzalez-Gay M. A., Hudson M., Kuwana M., Lundberg I. E., Mammen A., McHugh N., Miller F. W., Monteccucco C., Oddis C. V., Rojas-Serrano J., Schmidt J., Selva-O'Callaghan A., Werth V. P., Sakellariou G., Aggarwal R., and Cavagna L.

Abstract

Anti-synthetase syndrome (ASSD) is a heterogeneous autoimmune disease characterised by multi-system involvement with a wide variety of manifestations. Validated classification criteria are necessary to improve recognition and prevent misclassification, especially given the lack of reliable and standardised autoantibody testing. We systematically reviewed the literature to analyse proposed ASSD criteria, characteristics, and diagnostic performance. Methods We searched PubMed and Embase databases (01/01/1984 to 06/11/2018) and the ACR and EULAR meeting abstracts (2017-2018). Sensitivities, specificities, positive, negative likelihood ratios and risk of bias were calculated for ASSD criteria and key variables reported in the literature. We performed meta-analysis when appropriate. Results We retrieved 4,358 studies. We found 85 proposed ASSD criteria from a total of 82 studies. All but one study included anti-synthetase autoantibody (ARS) positivity in the ASSD criteria. Most studies required only one ASSD feature plus anti-ARS to define ASSD (n=64, 78%), whereas 16 studies required more than one ASSD variable plus anti-ARS. The only criteria not including anti-ARS positivity required 5 ASSD clinical features. We found limited data and wide variability in the diagnostic performance of each variable and definition proposed in the literature. Given these limitations we only meta-analysed the performance of individual muscle biopsy and clinical variables in diagnosing ASSD, which performed poorly. Conclusion The current ASSD criteria include a variety of serological, clinical, and histological features with wide variability amongst proposed definitions and the performance of these definitions has not been tested. This systematic literature review suggests the need for additional data and consensus-driven classification criteria for ASSD.

Published
2022

4. Impact of systemic sclerosis-associated interstitial lung disease with and without pulmonary hypertension on survival in over 5,000 patients

Author

Moinzadeh, P, Sunderkötter, C, Ehrchen, J, Kötter, I, Korsten, P, Lorenz, HM, Müller-Ladner, U, Oberste, M, Kreuter, A, Susok, L, Hunzelmann, N, Siegert, E, Worm, M, Pfeiffer, C, Riemekasten, G, Jandova, I, Schmalzing, M, Kreuter, M, Ramming, A, Bonella, F, Henes, J, Schmeiser, T, Zeidler, G, Görl, N, Juche, A, Günther, C, Keyßer, G, Blank, N, Moinzadeh, P, Sunderkötter, C, Ehrchen, J, Kötter, I, Korsten, P, Lorenz, HM, Müller-Ladner, U, Oberste, M, Kreuter, A, Susok, L, Hunzelmann, N, Siegert, E, Worm, M, Pfeiffer, C, Riemekasten, G, Jandova, I, Schmalzing, M, Kreuter, M, Ramming, A, Bonella, F, Henes, J, Schmeiser, T, Zeidler, G, Görl, N, Juche, A, Günther, C, Keyßer, G, and Blank, N

Published
2023

6. Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

Author

Kalluri, M., Luppi, F., Vancheri, A., Vancheri, C., Balestro, E., Varone, F., Mogulkoc, N., Cacopardo, G., Bargagli, E., Renzoni, E., Torrisi, S., Calvello, M., Libra, A., Pavone, M., Bonella, F., Cottin, V., Valenzuela, C., Wijsenbeek, M., Bendstrup, E., 3rd International Summit for ILD (ISILD), Erice collaborators listed below: Carlo Albera, Goksel, Altinisik, Kjetil, Ask, Elisabetta, Balestro, Elena, Bargagli, Elisabeth, Bendstrup, Marialuisa, Bocchino, Francesco, Bonella, Martina, Bonifazi, Giulia, Cacopardo, Maria, Calvello, Diego, M Castillo, Nazia, Chaudhuri, Ulrich, Costabel, Vincent, Cottin, Bruno, Crestani, Manuela, Funke-Chambour, Jack, Gauldie, Peter, M George, Johannes, C Grutters, Sergio, Harari, Richard, G Jenkins, Kerri, A Johannson, Mark, G Jones, Meena, Kalluri, Michael, P Keane, Maria, A Kokosi, Michael, Kreuter, Donato, Lacedonia, Brett, Ley, Alessandro, Libra, Fabrizio, Luppi, Toby, M Maher, George, A Margaritopoulos, Fernando, J Martinez, Jelle, Miedema, Nesrin, Mogulkoc, Maria, Molina-Molina, Philip, L Molyneaux, Julie, Morisset, Stefano, Palmucci, Mauro, Pavone, Ganesh, R Raghu, Elisabetta, A Renzoni, Luca, Richeldi, Gianluca, Sambataro, Alfredo, Sebastiani, Paolo, Spagnolo, Giulia Maria Stella, Martina, Sterclova, Irina, Strambu, Tomassetti, Sara, Sebastiano, Torrisi, Jacopo, Simonetti, Haluk, Turktas, Argyrios, Tzouvelekis, Claudia, Valenzuela, Ada, Vancheri, Carlo, Vancheri, Francesco, Varone, Patrizio, Vitulo, Athol, U Wells, Marlies, S Wijsenbeek, Wim, A Wuyts, Kalluri, M, Luppi, F, Vancheri, A, Vancheri, C, Balestro, E, Varone, F, Mogulkoc, N, Cacopardo, G, Bargagli, E, Renzoni, E, Torrisi, S, Calvello, M, Libra, A, Pavone, M, Bonella, F, Cottin, V, Valenzuela, C, Wijsenbeek, M, Bendstrup, E, Kalluri, M., Luppi, F., Vancheri, A., Vancheri, C., Balestro, E., Varone, F., Mogulkoc, N., Cacopardo, G., Bargagli, E., Renzoni, E., Torrisi, S., Calvello, M., Libra, A., Pavone, M., Bonella, F., Cottin, V., Valenzuela, C., Wijsenbeek, M., Bendstrup, E., and Bocchino, M.

Subjects
Pulmonary and Respiratory Medicine, medicine.medical_specialty, Concordance, Health Status, interstitial lung dieseases, MEDLINE, Medizin, Prom, 03 medical and health sciences, 0302 clinical medicine, Medicine, Humans, 030212 general & internal medicine, Patient Reported Outcome Measures, Intensive care medicine, business.industry, Minimal clinically important difference, Interstitial lung disease, respiratory system, medicine.disease, Idiopathic Pulmonary Fibrosis, female genital diseases and pregnancy complications, respiratory tract diseases, Clinical trial, patient reported outcomes, patient reported outcome measures, idiopathic pulmonary fibrosi, 030228 respiratory system, Patient-reported outcome, Computerized adaptive testing, Patient Participation, business, Lung Diseases, Interstitial
Abstract

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

Published
2021

8. Communicating with patients with idiopathic pulmonary fibrosis: can we do it better?

Author

Wijsenbeek, M. S., Bonella, F., Orsatti, L., Russell, A. M., Valenzuela, C., Wuyts, W. A., Baile, W. F., Wijsenbeek, M. S., Bonella, F., Orsatti, L., Russell, A. M., Valenzuela, C., Wuyts, W. A., and Baile, W. F.

Abstract

Communications between clinicians and patients with idiopathic pulmonary fibrosis (IPF) have the potential to be challenging. The variable course and poor prognosis of IPF complicate discussions around life expectancy but should not prevent clinicians from having meaningful conversations about patients’ fears and needs, while acknowledging uncertainties. Patients want information about the course of their disease and management options, but the provision of information needs to be individualised to the needs and preferences of the patient. Communication from clinicians should be empathetic and take account of the patient’s perceptions and concerns. Models, tools and protocols are available that can help clinicians to improve their interactions with patients. In this article, we consider the difficulties inherent in discussions with patients with IPF and their loved ones, and how clinicians might communicate with patients more effectively, from breaking the news about the diagnosis to providing support throughout the course of the disease.

Published
2022

9. Nailfold capillaroscopy characteristics of antisynthetase syndrome and possible clinical associations: Results of a multicenter international study

Author

Sebastiani, M, Triantafyllias, K, Manfredi, A, Gonzalez-Gay, M, Palmou-Fontana, N, Cassone, G, Drott, U, Delbruck, C, Rojas-Serrano, J, Bertolazzi, C, Nuno, L, Giannini, M, Iannone, F, Vicente, E, Castaneda, S, Selva-O'Callaghan, A, Araguas, E, Emmi, G, Iuliano, A, Bauhammer, J, Miehle, N, Parisi, S, Cavagna, L, Codullo, V, Montecucco, C, Lopez-Longo, F, Martinez-Barrio, J, Nieto-Gonzalez, J, Vichi, S, Confalonieri, M, Tomietto, P, Bergner, R, Sulli, A, Bonella, F, Furini, F, Scire, C, Bortoluzzi, A, Specker, C, Barsotti, S, Neri, R, Mosca, M, Caproni, M, Weinmann-Menke, J, Schwarting, A, Smith, V, Cutolo, M, Sebastiani M., Triantafyllias K., Manfredi A., Gonzalez-Gay M. A., Palmou-Fontana N., Cassone G., Drott U., Delbruck C., Rojas-Serrano J., Bertolazzi C., Nuno L., Giannini M., Iannone F., Vicente E. F., Castaneda S., Selva-O'Callaghan A., Araguas E. T., Emmi G., Iuliano A., Bauhammer J., Miehle N., Parisi S., Cavagna L., Codullo V., Montecucco C., Lopez-Longo F. J., Martinez-Barrio J., Nieto-Gonzalez J. C., Vichi S., Confalonieri M., Tomietto P., Bergner R., Sulli A., Bonella F., Furini F., Scire C. A., Bortoluzzi A., Specker C., Barsotti S., Neri R., Mosca M., Caproni M., Weinmann-Menke J., Schwarting A., Smith V., Cutolo M., Sebastiani, M, Triantafyllias, K, Manfredi, A, Gonzalez-Gay, M, Palmou-Fontana, N, Cassone, G, Drott, U, Delbruck, C, Rojas-Serrano, J, Bertolazzi, C, Nuno, L, Giannini, M, Iannone, F, Vicente, E, Castaneda, S, Selva-O'Callaghan, A, Araguas, E, Emmi, G, Iuliano, A, Bauhammer, J, Miehle, N, Parisi, S, Cavagna, L, Codullo, V, Montecucco, C, Lopez-Longo, F, Martinez-Barrio, J, Nieto-Gonzalez, J, Vichi, S, Confalonieri, M, Tomietto, P, Bergner, R, Sulli, A, Bonella, F, Furini, F, Scire, C, Bortoluzzi, A, Specker, C, Barsotti, S, Neri, R, Mosca, M, Caproni, M, Weinmann-Menke, J, Schwarting, A, Smith, V, Cutolo, M, Sebastiani M., Triantafyllias K., Manfredi A., Gonzalez-Gay M. A., Palmou-Fontana N., Cassone G., Drott U., Delbruck C., Rojas-Serrano J., Bertolazzi C., Nuno L., Giannini M., Iannone F., Vicente E. F., Castaneda S., Selva-O'Callaghan A., Araguas E. T., Emmi G., Iuliano A., Bauhammer J., Miehle N., Parisi S., Cavagna L., Codullo V., Montecucco C., Lopez-Longo F. J., Martinez-Barrio J., Nieto-Gonzalez J. C., Vichi S., Confalonieri M., Tomietto P., Bergner R., Sulli A., Bonella F., Furini F., Scire C. A., Bortoluzzi A., Specker C., Barsotti S., Neri R., Mosca M., Caproni M., Weinmann-Menke J., Schwarting A., Smith V., and Cutolo M.

Abstract

Objective. To describe nailfold videocapillaroscopy (NVC) features of patients with antisynthetase syndrome (AS) and to investigate possible correlations with clinical and serological features of the disease. Methods. We retrospectively analyzed NVC images of 190 patients with AS [females/males 3.63, mean age 49.7 ± 12.8 yrs, median disease duration 53.7 mos (interquartile range 82), 133 anti-Jo1 and 57 non-anti-Jo1-positive patients]. For each patient, we examined number of capillaries, giant capillaries, microhemorrhages, avascular areas, ramified capillaries, and the presence of systemic sclerosis (SSc)-like pattern. Finally, we correlated NVC features with clinical and serological findings of patients with AS. Concomitantly, a historical cohort of 75 patients with antinuclear antibody-negative primary Raynaud phenomenon (RP) and longterm followup was used as a control group (female/male ratio 4.13/1, mean age 53.9 ± 17.6 yrs) for NVC measures. Results. NVC abnormalities were observed in 62.1% of AS patients compared with 29.3% of primary RP group (p < 0.001). An SSc-like pattern was detected in 67 patients (35.3%) and it was associated with anti-Jo1 antibodies (p = 0.002) and also with a longer disease duration (p = 0.004). Interestingly, there was no significant correlation between the presence of SSc-like pattern and RP, and only 47% of patients with SSc-like pattern had RP. Conclusion. NVC abnormalities are commonly observed in AS, independently from the occurrence of RP. The presence of an SSc-like pattern could allow identification of a more defined AS subtype, and prospective studies could confirm the association with clinical and serological features of AS.

Published
2019

10. Mechanisms of progressive fibrosis in connective tissue disease (CTD)-associated interstitial lung diseases (ILDs)

Author

Spagnolo, P. Distler, O. Ryerson, C.J. Tzouvelekis, A. Lee, J.S. Bonella, F. Bouros, D. Hoffmann-Vold, A.-M. Crestani, B. Matteson, E.L.

Subjects
respiratory system, behavioral disciplines and activities, respiratory tract diseases
Abstract

Interstitial lung diseases (ILDs), which can arise from a broad spectrum of distinct aetiologies, can manifest as a pulmonary complication of an underlying autoimmune and connective tissue disease (CTD-ILD), such as rheumatoid arthritis-ILD and systemic sclerosis (SSc-ILD). Patients with clinically distinct ILDs, whether CTD-related or not, can exhibit a pattern of common clinical disease behaviour (declining lung function, worsening respiratory symptoms and higher mortality), attributable to progressive fibrosis in the lungs. In recent years, the tyrosine kinase inhibitor nintedanib has demonstrated efficacy and safety in idiopathic pulmonary fibrosis (IPF), SSc-ILD and a broad range of other fibrosing ILDs with a progressive phenotype, including those associated with CTDs. Data from phase II studies also suggest that pirfenidone, which has a different -yet largely unknown -mechanism of action, may also have activity in other fibrosing ILDs with a progressive phenotype, in addition to its known efficacy in IPF. Collectively, these studies add weight to the hypothesis that, irrespective of the original clinical diagnosis of ILD, a progressive fibrosing phenotype may arise from common, underlying pathophysiological mechanisms of fibrosis involving pathways associated with the targets of nintedanib and, potentially, pirfenidone. However, despite the early proof of concept provided by these clinical studies, very little is known about the mechanistic commonalities and differences between ILDs with a progressive phenotype. In this review, we explore the biological and genetic mechanisms that drive fibrosis, and identify the missing evidence needed to provide the rationale for further studies that use the progressive phenotype as a target population. © Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Published
2021

11. LONG-TERM OUTCOME OF SSC ASSOCIATED ILD: IMPROVED SURVIVAL IN PPI TREATED PATIENTS

Author

Kreuter, M., Bonella, F., Kathrin, K., Henes, J., Siegert, E., Riemekasten, G., Blank, N., Pfeiffer, C., Mueller-Ladner, U., Kreuter, A., Korsten, P., Juche, A., Schmalzing, M., Worm, M., Jandova, I., Susok, L., Schmeiser, T., Guenther, C., Keyszer, G., Ehrchen, J., Ramming, A., Koetter, I., Lorenz, H. M., Moinzadeh, P., Hunzelmann, N., Kreuter, M., Bonella, F., Kathrin, K., Henes, J., Siegert, E., Riemekasten, G., Blank, N., Pfeiffer, C., Mueller-Ladner, U., Kreuter, A., Korsten, P., Juche, A., Schmalzing, M., Worm, M., Jandova, I., Susok, L., Schmeiser, T., Guenther, C., Keyszer, G., Ehrchen, J., Ramming, A., Koetter, I., Lorenz, H. M., Moinzadeh, P., and Hunzelmann, N.

Published
2021

12. Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

Author

Kalluri, M, Luppi, F, Vancheri, A, Vancheri, C, Balestro, E, Varone, F, Mogulkoc, N, Cacopardo, G, Bargagli, E, Renzoni, E, Torrisi, S, Calvello, M, Libra, A, Pavone, M, Bonella, F, Cottin, V, Valenzuela, C, Wijsenbeek, M, Bendstrup, E, Kalluri, Meena, Luppi, Fabrizio, Vancheri, Ada, Vancheri, Carlo, Balestro, Elisabetta, Varone, Francesco, Mogulkoc, Nesrin, Cacopardo, Giulia, Bargagli, Elena, Renzoni, Elizabeth, Torrisi, Sebastiano, Calvello, Mariarosaria, Libra, Alessandro, Pavone, Mauro, Bonella, Francesco, Cottin, Vincent, Valenzuela, Claudia, Wijsenbeek, Marlies, Bendstrup, Elisabeth, Kalluri, M, Luppi, F, Vancheri, A, Vancheri, C, Balestro, E, Varone, F, Mogulkoc, N, Cacopardo, G, Bargagli, E, Renzoni, E, Torrisi, S, Calvello, M, Libra, A, Pavone, M, Bonella, F, Cottin, V, Valenzuela, C, Wijsenbeek, M, Bendstrup, E, Kalluri, Meena, Luppi, Fabrizio, Vancheri, Ada, Vancheri, Carlo, Balestro, Elisabetta, Varone, Francesco, Mogulkoc, Nesrin, Cacopardo, Giulia, Bargagli, Elena, Renzoni, Elizabeth, Torrisi, Sebastiano, Calvello, Mariarosaria, Libra, Alessandro, Pavone, Mauro, Bonella, Francesco, Cottin, Vincent, Valenzuela, Claudia, Wijsenbeek, Marlies, and Bendstrup, Elisabeth

Abstract

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

Published
2021

13. Shedding light on developmental drugs for idiopathic pulmonary fibrosis

Author

Spagnolo, P. Bonella, F. Ryerson, C.J. Tzouvelekis, A. Maher, T.M.

Abstract

Introduction: Idiopathic pulmonary fibrosis (IPF) is an age-related disease of unknown cause. The disease is characterized by relentless scarring of the lung parenchyma resulting in respiratory failure and death. Two antifibrotic drugs (pirfenidone and nintedanib) are approved for the treatment of IPF worldwide, but they do not offer a cure and are associated with tolerability issues. Owing to its high unmet medical need, IPF is an area of dynamic research activity. Areas covered: There is a growing portfolio of novel therapies that target different pathways involved in the complex pathogenesis of IPF. In this review, we discuss the mechanisms of action and available data for compounds in the most advanced stages of clinical development. We searched PubMed for articles on this topic published from 1 January 2000, to 6 June 2020. Expert opinion: The approval of pirfenidone and nintedanib has fueled IPF drug discovery and development. New drugs are likely to reach the clinic in the near future. However, numerous challenges remain; the lack of animal models that reproduce the complexity of human disease and the poor translation of preclinical and early-phase positive effects to late stage clinical trials must be tackled. © 2020 Informa UK Limited, trading as Taylor & Francis Group.

Published
2020

14. Inhaled molgramostim therapy in autoimmune pulmonary alveolar proteinosis

Author

Trapnell, B.C. Inoue, Y. Bonella, F. Morgan, C. Jouneau, S. Bendstrup, E. Campo, I. Papiris, S.A. Yamaguchi, E. Cetinkaya, E. Ilkovich, M.M. Kramer, M.R. Veltkamp, M. Kreuter, M. Baba, T. Ganslandt, C. Tarnow, I. Waterer, G. Jouhikainen, T. IMPALA Trial Investigators

Abstract

BACKGROUND Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease characterized by progressive surfactant accumulation and hypoxemia. It is caused by disruption of granulocyte-macrophage colony-stimulating factor (GM-CSF) signaling, which pulmonary alveolar macrophages require to clear surfactant. Recently, inhaled GM-CSF was shown to improve the partial pressure of arterial oxygen in patients with aPAP. METHODS In a double-blind, placebo-controlled, three-group trial, we randomly assigned patients with aPAP to receive the recombinant GM-CSF molgramostim (300 μg once daily by inhalation), either continuously or intermittently (every other week), or matching placebo. The 24-week intervention period was followed by an open-label treatment-extension period. The primary end point was the change from baseline in the alveolar-arterial difference in oxygen concentration (A-aDo2) at week 24. RESULTS In total, 138 patients underwent randomization; 46 were assigned to receive continuous molgramostim, 45 to receive intermittent molgramostim, and 47 to receive placebo. Invalid A-aDo2 data for 4 patients (1 in each molgramostim group and 2 in the placebo group) who received nasal oxygen therapy during arterial blood gas measurement were replaced by means of imputation. For the primary end point - the change from baseline in the A-aDo2 at week 24 - improvement was greater among patients receiving continuous molgramostim than among those receiving placebo (-12.8 mm Hg vs. -6.6 mm Hg; estimated treatment difference, -6.2 mm Hg; P = 0.03 by comparison of least-squares means). Patients receiving continuous molgramostim also had greater improvement than those receiving placebo for secondary end points, including the change from baseline in the St. George's Respiratory Questionnaire total score at week 24 (-12.4 points vs. -5.1 points; estimated treatment difference, -7.4 points; P = 0.01 by comparison of least-squares means). For multiple end points, improvement was greater with continuous molgramostim than with intermittent molgramostim. The percentages of patients with adverse events and serious adverse events were similar in the three groups, except for the percentage of patients with chest pain, which was higher in the continuous-molgramostim group. CONCLUSIONS In patients with aPAP, daily administration of inhaled molgramostim resulted in greater improvements in pulmonary gas transfer and functional health status than placebo, with similar rates of adverse events. (Funded by Savara Pharmaceuticals; IMPALA ClinicalTrials.gov number, NCT02702180.). Copyright © 2020 Massachusetts Medical Society.

Published
2020

15. Long term outcomes of immunmodulatory drugs in SSc-ILD - data rom the German SSc network

Author

Kreuter, M., Bonella, F., Blank, N., Siegert, E., Henes, J., Worm, M., Sunderkoetter, C., Schmalzing, M., Kreuter, A., Guenther, C., Susok, L., Zeidler, G., Koetter, I, Mueller-Ladner, U., Krieg, T., Juche, A., Schmeiser, T., Riemekasten, G., Aberer, E., Gaebelein-Wissing, N., Distler, J. H. W., Sardy, M., Pfeiffer, C., Kuhr, K., Lorenz, H. M., Moinzadeh, P., Hunzelmann, N., Kreuter, M., Bonella, F., Blank, N., Siegert, E., Henes, J., Worm, M., Sunderkoetter, C., Schmalzing, M., Kreuter, A., Guenther, C., Susok, L., Zeidler, G., Koetter, I, Mueller-Ladner, U., Krieg, T., Juche, A., Schmeiser, T., Riemekasten, G., Aberer, E., Gaebelein-Wissing, N., Distler, J. H. W., Sardy, M., Pfeiffer, C., Kuhr, K., Lorenz, H. M., Moinzadeh, P., and Hunzelmann, N.

Published
2020

16. Does anti-acid treatment influence disease progression in SSc-ILD? Data form the German SSc-network

Author

Kreuter, M., Bonella, F., Blank, N., Siegert, E., Henes, J., Worm, M., Sunderkoetter, C., Schmalzing, M., Kreuter, A., Guenther, C., Susok, L., Zeidler, G., Koetter, I, Mueller-Ladner, U., Krieg, T., Juche, A., Schmeiser, T., Riemekasten, G., Aberer, E., Gaebelein-Wissing, N., Distler, J. H. W., Sardy, M., Pfeiffer, C., Kuhr, K., Lorenz, H. M., Moinzadeh, P., Hunzelmann, N., Kreuter, M., Bonella, F., Blank, N., Siegert, E., Henes, J., Worm, M., Sunderkoetter, C., Schmalzing, M., Kreuter, A., Guenther, C., Susok, L., Zeidler, G., Koetter, I, Mueller-Ladner, U., Krieg, T., Juche, A., Schmeiser, T., Riemekasten, G., Aberer, E., Gaebelein-Wissing, N., Distler, J. H. W., Sardy, M., Pfeiffer, C., Kuhr, K., Lorenz, H. M., Moinzadeh, P., and Hunzelmann, N.

Published
2020

17. DOES ANTI-ACID TREATMENT INFLUENCE DISEASE PROGRESSION IN SYSTEMIC SCLEROSIS INTERSTITIAL LUNG DISEASE (SSC-ILD)? DATA FROM THE GERMAN SSC-NETWORK

Author

Kreuter, M., Bonella, F., Riemekasten, G., Mueller-Ladner, U., Henes, J., Siegert, E., Guenther, C., Koetter, I., Blank, N., Pfeiffer, C., Schmalzing, M., Zeidler, G., Korsten, P., Susok, L., Juche, A., Worm, M., Jandova, I., Ehrchen, J., Sunderkoetter, C., Keyszer, G., Ramming, A., Schmeiser, T., Kreuter, A., Kuhr, K., Lorenz, H. M., Moinzadeh, P., Hunzelmann, N., Kreuter, M., Bonella, F., Riemekasten, G., Mueller-Ladner, U., Henes, J., Siegert, E., Guenther, C., Koetter, I., Blank, N., Pfeiffer, C., Schmalzing, M., Zeidler, G., Korsten, P., Susok, L., Juche, A., Worm, M., Jandova, I., Ehrchen, J., Sunderkoetter, C., Keyszer, G., Ramming, A., Schmeiser, T., Kreuter, A., Kuhr, K., Lorenz, H. M., Moinzadeh, P., and Hunzelmann, N.

Published
2020

18. Treatment of systemic sclerosis-associated interstitial lung disease

Author

Prasse, A., Bonella, F., Mueller-Ladner, U., Witte, T., Hunzelmann, N., Distler, J., Prasse, A., Bonella, F., Mueller-Ladner, U., Witte, T., Hunzelmann, N., and Distler, J.

Abstract

Background Systemic sclerosis (SSc) is a fibrosing autoimmune disease of the connective tissue. In addition to skin fibrosis, pulmonary involvement and interstitial lung disease (ILD) in particular are the most common and severe manifestations of SSc. The disease is associated with a substantial risk of morbidity and mortality, especially in progressive ILD. In the last 5 years new treatment concepts for SSc-ILD have been investigated in numerous clinical studies. Material and methods This review is based on a literature search in PubMed, focusing on the most relevant papers published up to the end of 2018 with the keywords SSc and treatment. Results The treatment of SSc-ILD has changed over the last few years due to the results of many clinical studies. The updated guidelines of the European League Against Rheumatism (EULAR) recommend the use of cyclophosphamide or hematopoietic stem cell transplantation. Data for a positive influence on SSc-ILD are also available for mycophenolate, tocilizumab and anabasum. Because of the pathophysiological similarities to idiopathic pulmonary fibrosis, the use of the antifibrotic agents nintedanib and pirfenidone is currently being investigated in randomized, multicenter clinical trials and could be a novel and promising therapeutic strategy. Conclusion Current drug studies may provide innovative therapeutic perspectives for SSc-ILD and could significantly improve the prognosis of affected patients in the future.

Published
2020

19. Nintedanib in patients with progressive fibrosing interstitial lung diseases—subgroup analyses by interstitial lung disease diagnosis in the INBUILD trial: a randomised, double-blind, placebo-controlled, parallel-group trial

Author

Wells, A. U., Flaherty, K. R., Brown, K. K., Inoue, Y., Devaraj, A., Richeldi, Luca, Moua, T., Crestani, B., Wuyts, W. A., Stowasser, S., Quaresma, M., Goeldner, R. -G., Schlenker-Herceg, R., Kolb, M., Abe, S., Aburto, M., Acosta, O., Andrews, C., Antin-Ozerkis, D., Arce, G., Arias, M., Avdeev, S., Barczyk, A., Bascom, R., Bazdyrev, E., Beirne, P., Belloli, E., Bergna, M. A., Bergot, E., Bhatt, N., Blaas, S., Bondue, B., Bonella, F., Britt, E., Buch, K., Burk, J., Cai, H., Cantin, A., Castillo Villegas, D. M., Cazaux, A., Cerri, S., Chaaban, S., Chaudhuri, N., Cottin, V., Criner, G., Dahlqvist, C., Danoff, S., Dematte D'Amico, J., Dilling, D., Elias, P., Ettinger, N., Falk, J., Fernandez Perez, E. R., Gamez-Dubuis, A., Giessel, G., Gifford, A., Glassberg, M., Glazer, C., Golden, J., Gomez Carrera, L., Guiot, J., Hallowell, R., Hayashi, H., Hetzel, J., Hirani, N., Homik, L., Hope-Gill, B., Hotchkin, D., Ichikado, K., Ilkovich, M., Izumi, S., Jassem, E., Jones, L., Jouneau, S., Kaner, R., Kang, J., Kawamura, T., Kessler, R., Kim, Y., Kishi, K., Kitamura, H., Kondoh, Y., Kono, C., Koschel, D., Kreuter, M., Kulkarni, T., Kus, J., Lebargy, F., Leon Jimenez, A., Luo, Q., Mageto, Y., Maher, T. M., Makino, S., Marchand-Adam, S., Marquette, C., Martinez, Sara, Martinez, M., Maturana Rozas, R., Miyazaki, Y., Moiseev, S., Molina-Molina, M., Malcolm, Joan Morrison, Morrow, L., Nambiar, A., Nishioka, Y., Nunes, H., Okamoto, M., Oldham, J., Otaola, M., Padilla, M., Park, J. S., Patel, N., Pesci, Riccardo, Piotrowski, W., Pitts, L., Poonyagariyagorn, H., Prasse, A., Quadrelli, S., Randerath, W., Refini, R., Reynaud-Gaubert, M., Riviere, F., Rodriguez Portal, J. A., Rosas, I., Rossman, M., Safdar, Z., Saito, T., Sakamoto, N., Salinas Fenero, M., Sauleda, J., Schmidt, S., Scholand, M. B., Schwartz, M., Shapera, S., Shlobin, O., Sigal, B., Silva Orellana, A., Skowasch, D., Song, J. W., Stieglitz, S., Stone, H., Strek, M., Suda, T., Sugiura, H., Takahashi, H., Takaya, H., Takeuchi, T., Thavarajah, K., Tolle, L., Tomassetti, S., Tomii, K., Valenzuela, C., Vancheri, C., Varone, Francesco, Veeraraghavan, S., Villar, A., Weigt, S., Wemeau, L., Wuyts, W., Xu, Z., Yakusevich, V., Yamada, Y., Yamauchi, H., Ziora, D., Richeldi L. (ORCID:0000-0001-8594-1448), Martinez R., Morrison L., Pesci A., Varone F., Wells, A. U., Flaherty, K. R., Brown, K. K., Inoue, Y., Devaraj, A., Richeldi, Luca, Moua, T., Crestani, B., Wuyts, W. A., Stowasser, S., Quaresma, M., Goeldner, R. -G., Schlenker-Herceg, R., Kolb, M., Abe, S., Aburto, M., Acosta, O., Andrews, C., Antin-Ozerkis, D., Arce, G., Arias, M., Avdeev, S., Barczyk, A., Bascom, R., Bazdyrev, E., Beirne, P., Belloli, E., Bergna, M. A., Bergot, E., Bhatt, N., Blaas, S., Bondue, B., Bonella, F., Britt, E., Buch, K., Burk, J., Cai, H., Cantin, A., Castillo Villegas, D. M., Cazaux, A., Cerri, S., Chaaban, S., Chaudhuri, N., Cottin, V., Criner, G., Dahlqvist, C., Danoff, S., Dematte D'Amico, J., Dilling, D., Elias, P., Ettinger, N., Falk, J., Fernandez Perez, E. R., Gamez-Dubuis, A., Giessel, G., Gifford, A., Glassberg, M., Glazer, C., Golden, J., Gomez Carrera, L., Guiot, J., Hallowell, R., Hayashi, H., Hetzel, J., Hirani, N., Homik, L., Hope-Gill, B., Hotchkin, D., Ichikado, K., Ilkovich, M., Izumi, S., Jassem, E., Jones, L., Jouneau, S., Kaner, R., Kang, J., Kawamura, T., Kessler, R., Kim, Y., Kishi, K., Kitamura, H., Kondoh, Y., Kono, C., Koschel, D., Kreuter, M., Kulkarni, T., Kus, J., Lebargy, F., Leon Jimenez, A., Luo, Q., Mageto, Y., Maher, T. M., Makino, S., Marchand-Adam, S., Marquette, C., Martinez, Sara, Martinez, M., Maturana Rozas, R., Miyazaki, Y., Moiseev, S., Molina-Molina, M., Malcolm, Joan Morrison, Morrow, L., Nambiar, A., Nishioka, Y., Nunes, H., Okamoto, M., Oldham, J., Otaola, M., Padilla, M., Park, J. S., Patel, N., Pesci, Riccardo, Piotrowski, W., Pitts, L., Poonyagariyagorn, H., Prasse, A., Quadrelli, S., Randerath, W., Refini, R., Reynaud-Gaubert, M., Riviere, F., Rodriguez Portal, J. A., Rosas, I., Rossman, M., Safdar, Z., Saito, T., Sakamoto, N., Salinas Fenero, M., Sauleda, J., Schmidt, S., Scholand, M. B., Schwartz, M., Shapera, S., Shlobin, O., Sigal, B., Silva Orellana, A., Skowasch, D., Song, J. W., Stieglitz, S., Stone, H., Strek, M., Suda, T., Sugiura, H., Takahashi, H., Takaya, H., Takeuchi, T., Thavarajah, K., Tolle, L., Tomassetti, S., Tomii, K., Valenzuela, C., Vancheri, C., Varone, Francesco, Veeraraghavan, S., Villar, A., Weigt, S., Wemeau, L., Wuyts, W., Xu, Z., Yakusevich, V., Yamada, Y., Yamauchi, H., Ziora, D., Richeldi L. (ORCID:0000-0001-8594-1448), Martinez R., Morrison L., Pesci A., and Varone F.

Abstract

Background: The INBUILD trial investigated the efficacy and safety of nintedanib versus placebo in patients with progressive fibrosing interstitial lung diseases (ILDs) other than idiopathic pulmonary fibrosis (IPF). We aimed to establish the effects of nintedanib in subgroups based on ILD diagnosis. Methods: The INBUILD trial was a randomised, double-blind, placebo-controlled, parallel group trial done at 153 sites in 15 countries. Participants had an investigator-diagnosed fibrosing ILD other than IPF, with chest imaging features of fibrosis of more than 10% extent on high resolution CT (HRCT), forced vital capacity (FVC) of 45% or more predicted, and diffusing capacity of the lung for carbon monoxide (DLco) of at least 30% and less than 80% predicted. Participants fulfilled protocol-defined criteria for ILD progression in the 24 months before screening, despite management considered appropriate in clinical practice for the individual ILD. Participants were randomly assigned 1:1 by means of a pseudo-random number generator to receive nintedanib 150 mg twice daily or placebo for at least 52 weeks. Participants, investigators, and other personnel involved in the trial and analysis were masked to treatment assignment until after database lock. In this subgroup analysis, we assessed the rate of decline in FVC (mL/year) over 52 weeks in patients who received at least one dose of nintedanib or placebo in five prespecified subgroups based on the ILD diagnoses documented by the investigators: hypersensitivity pneumonitis, autoimmune ILDs, idiopathic non-specific interstitial pneumonia, unclassifiable idiopathic interstitial pneumonia, and other ILDs. The trial has been completed and is registered with ClinicalTrials.gov, number NCT02999178. Findings: Participants were recruited between Feb 23, 2017, and April 27, 2018. Of 663 participants who received at least one dose of nintedanib or placebo, 173 (26%) had chronic hypersensitivity pneumonitis, 170 (26%) an autoimmune IL

Published
2020

21. Significance of pulmonary involvement in systemic sclerosis (SSc) – data from the German SSc-network

Author

Kreuter, M, Bonella, F, Blank, N, Henes, J, Worm, M, Sunderkötter, C, Schmalzing, M, Kreuter, A, Günther, C, Susok, L, Zeidler, G, Kötter, I, Müller-Ladner, U, Krieg, T, Juche, A, Schmeiser, T, Riemekasten, G, Aberer, E, Gäbelein-Wissing, N, Distler, JHW, Sardy, M, Pfeiffer, C, Kuhr, K, Lorenz, HM, Moinzadeh, P, and Hunzelmann, N

Subjects
ddc: 610, respiratory system, 610 Medical sciences, Medicine, behavioral disciplines and activities, respiratory tract diseases
Abstract

Background: Pulmonary involvement is the leading cause of death in SSc and can manifest as interstitial lung disease (ILD), pulmonary hypertension (PAH) or a combination (ILD-PH). Aim of this analysis was to determine prevalence, clinical characteristics and outcomes of these different forms within [for full text, please go to the a.m. URL], 46. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 32. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), Wissenschaftliche Herbsttagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR)

Published
2019
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22. The characterisation of interstitial lung disease multidisciplinary team meetings: A global study

Author

Richeldi, L. Launders, N. Martinez, F. Walsh, S.L.F. Myers, J. Wang, B. Jones, M. Chisholm, A. Flaherty, K.R. David-Wang, A. Morais, A. Azuma, A. Crestani, B. Vancheri, C. Youakim, C. Fell, C.D. Ryerson, C.J. Bouros, D. Bendstrup, E. Morell, F. Bonella, F. Raghu, G. Christoff, G. Ferrara, G. Glaspole, I. Rosas, I. Behr, J. DeBoer, K. Antoniou, K.M. Dheda, K. Brown, K. Planas-Cerezales, L. Sköld, M. Funke, M. Molina-Molina, M. Mazzei, M. Kolb, M. Selman, M. Rottoli, P. Spagnolo, P. Rivera-Ortega, P. Avdeev, S. Quadrelli, S. Corte, T.J. Maher, T.M. Cottin, V. Wuyts, W. Xu, Z.J.

Subjects
body regions, respiratory system
Abstract

Multidisciplinary team (MDT) diagnosis of interstitial lung disease (ILD) has been proposed as a gold standard, but there are no formal recommendations for MDT process or composition and limited knowledge regarding prevalence in routine practice. We performed a systematic evaluation of ILD diagnostic practice across a range of healthcare settings around the world. Electronic questionnaires were distributed across all global regions via society and collaborators networks. Responses from 457 unique centres across 64 countries were included in the analysis. Of the 350 (76.6%) centres holding formal meetings, the majority held face-to-face MDT meetings (80%), for a minimum of 30 min (93%), and discussed diagnosis (96.9%) and patient management (94.9%) at the meetings. Compared with non-academic and academic non-ILD centres, ILD academic centres reported a higher ILD caseload, held more formal MDT meetings, and were more likely to include histopathology and rheumatology specialists in their diagnostic team. Of the centres holding MDT meetings, 5.5% routinely discussed all new cases at such meetings. An MDT approach to ILD diagnosis is consistently interpreted and widely implemented across a range of routine care settings around the world. This observation will inform future ILD diagnostic agreement studies and diagnostic pathway recommendations. © ERS 2019.

Published
2019

23. The Burden of Sarcoidosis Symptoms from a Patient Perspective

Author

Voortman, M., Hendriks, C. M.R., Elfferich, M. D.P., Bonella, F., Møller, J., De Vries, J., Costabel, U., Drent, M., Voortman, M., Hendriks, C. M.R., Elfferich, M. D.P., Bonella, F., Møller, J., De Vries, J., Costabel, U., and Drent, M.

Published
2019

24. Gaps in care of patients living with pulmonary fibrosis

Author

Moor, C.C. (Karen), Wijsenbeek-Lourens, M.S. (Marlies), Balestro, E. (Elisabetta), Biondini, D. (Davide), Bondue, B. (Benjamin), Cottin, V. (Vincent), Kreuter, M. (Michael), Bonella, F. (Francesco), Moor, C.C. (Karen), Wijsenbeek-Lourens, M.S. (Marlies), Balestro, E. (Elisabetta), Biondini, D. (Davide), Bondue, B. (Benjamin), Cottin, V. (Vincent), Kreuter, M. (Michael), and Bonella, F. (Francesco)

Abstract

Introduction: Pulmonary fibrosis (PF) and its most common form, idiopathic pulmonary fibrosis (IPF), are chronic, progressive diseases resulting in increasing loss of lung function and impaired quality of life and survival. The aim of this joint expert and patient statement was to highlight the most pressing common unmet needs of patients with PF/IPF, putting forward recommendations to improve the quality of life and health outcomes throughout the patient journey. Methods: Two online surveys for patients and healthcare professionals (HCPs) were conducted by the European Idiopathic Pulmonary Fibrosis and Related Disorders Federation (EU-IPFF) in 14 European countries. Results: The surveys were answered by 286 patients and 69 HCPs, including physicians and nurses. Delays in diagnosis and timely access to interstitial lung disease specialists and pharmacological treatment have been identified as important gaps in care. Additionally, patients and HCPs reported that a greater focus on symptomcentred management, adequate information, trial information and increasing awareness of PF/IPF is required. Conclusions: The surveys offer important insights into the current unmet needs of PF/IPF patients. Interventions at different points of the care pathway are needed to improve patient experience.

Published
2019
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25. Cardiovascular safety of nintedanib in subgroups by cardiovascular risk at baseline in the TOMORROW and INPULSIS trials

Author

Noth, I. (Imre), Wijsenbeek-Lourens, M.S. (Marlies), Kolb, M. (Martin), Bonella, F. (Francesco), Moros, L. (Lizette), Wachtlin, D. (Daniel), Corte, T.J. (Tamera J.), Noth, I. (Imre), Wijsenbeek-Lourens, M.S. (Marlies), Kolb, M. (Martin), Bonella, F. (Francesco), Moros, L. (Lizette), Wachtlin, D. (Daniel), and Corte, T.J. (Tamera J.)

Abstract

Nintedanib is a tyrosine kinase inhibitor used to treat idiopathic pulmonary fibrosis (IPF). We investigated the cardiovascular safety of nintedanib using pooled data from the TOMORROW and INPULSIS trials.Cardiovascular events were assessed post hoc in patients with a history of atherosclerotic cardiovascular disease (CVD) and/or one or more cardiovascular risk factors at baseline ("higher cardiovascular risk") and patients with no history of atherosclerotic CVD and no cardiovascular risk factors at baseline ("lower cardiovascular risk").Incidence rates were calculated for 1231 patients (n=723 nintedanib and n=508 placebo), of whom 89.9% had higher cardiovascular risk. Incidence rates of major adverse cardiovascular events were similar in the nintedanib and placebo groups in patients with higher cardiovascular risk (3.88 (95% CI 2.58-5.84) and 3.49 (95% CI 2.10-5.79) per 100 patient-years, respectively) and lower cardiovascular risk (4.78 (95% CI 1.54-14.82) and 5.37 (95% CI 1.73-16.65) per 100 patient-years, respectively). Incidence rates of myocardial infarction in the nintedanib and placebo groups, respectively, were 3.03 (95% CI 1.91-4.81) and 1.16 (95% CI 0.48-2.79) per 100 patient-years in patients with higher cardiovascular risk and 1.59 (95% CI 0.22-11.29) and 1.78 (95% CI 0.25-12.64) per 100 patient-years in patients with lower cardiovascular risk. Incidence rates of other ischaemic heart disease in the nintedanib and placebo groups, respectively, were 1.85 (95% CI 1.02-3.34) and 3.28 (95% CI 1.94-5.54) per 100 patient-years in patients with higher cardiovascular risk and 0 and 1.80 (95% CI 0.25-12.78) per 100 patient-years in patients with lower cardiovascular risk.These data help to establish the cardiovascular safety profile of nintedanib in IPF.

Published
2019
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26. Resequencing study confirms that host defense and cell senescence gene variants contribute to the risk of idiopathic pulmonary fibrosis

Author

Moore, C., Blumhagen, R.Z., Yang, I.V., Walts, A., Powers, J., Walker, T., Bishop, M., Russell, P., Vestal, B., Cardwell, J., Markin, C.R., Mathai, S.K., Schwarz, M.I., Steele, M.P., Lee, J., Brown, K.K., Loyd, J.E., Crapo, J.D., Silverman, E.K., Cho, M.H., James, J.A., Guthridge, J.M., Cogan, J.D., Kropski, J.A., Swigris, J.J., Bair, C., Kim, D.S., Ji, W., Kim, H., Song, J.W., Maier, L.A., Pacheco, K.A., Hirani, N., Poon, A.S., Li, F., Jenkins, R.G., Braybrooke, R., Saini, G., Maher, T.M., Molyneaux, P.L., Saunders, P., Zhang, Y., Gibson, K.F., Kass, D.J., Rojas, M., Sembrat, J., Wolters, P.J., Collard, H.R., Sundy, J.S., O’Riordan, T., Strek, M.E., Noth, I., Ma, S-F, Porteous, M.K., Kreider, M.E., Patel, N.B., Inoue, Y., Hirose, M., Arai, T., Akagawa, S., Eickelberg, O., Fernandez, I.E., Behr, J., Mogulkoc, N., Corte, T.J., Glaspole, I., Tomassetti, S., Ravaglia, C., Poletti, V., Crestani, B., Borie, R., Kannengiesser, C., Parfrey, H., Fiddler, C., Rassl, D., Molina-Molina, M., Machahua, C., Worboys, A.M., Gudmundsson, G., Isaksson, H.J., Lederer, D.J., Podolanczuk, A.J., Montesi, S.B., Bendstrup, E., Danchel, V., Selman, M., Pardo, A., Henry, M.T., Keane, M.P., Doran, P., Vašáková, M., Sterclova, M., Ryerson, C.J., Wilcox, P.G., Okamoto, T., Furusawa, H., Miyazaki, Y., Laurent, G., Baltic, S., Prêle, C., Moodley, Y., Shea, B.S., Ohta, K., Suzukawa, M., Narumoto, O., Nathan, S.D., Venuto, D.C., Woldehanna, M.L., Kokturk, N., de Andrade, J.A., Luckhardt, T., Kulkarni, T., Bonella, F., Donnelly, S.C., McElroy, A., Armstong, M.E., Aranda, A., Carbone, R.G., Puppo, F., Beckman, K.B., Nickerson, D.A., Fingerlin, T.E., Schwartz, D.A., Moore, C., Blumhagen, R.Z., Yang, I.V., Walts, A., Powers, J., Walker, T., Bishop, M., Russell, P., Vestal, B., Cardwell, J., Markin, C.R., Mathai, S.K., Schwarz, M.I., Steele, M.P., Lee, J., Brown, K.K., Loyd, J.E., Crapo, J.D., Silverman, E.K., Cho, M.H., James, J.A., Guthridge, J.M., Cogan, J.D., Kropski, J.A., Swigris, J.J., Bair, C., Kim, D.S., Ji, W., Kim, H., Song, J.W., Maier, L.A., Pacheco, K.A., Hirani, N., Poon, A.S., Li, F., Jenkins, R.G., Braybrooke, R., Saini, G., Maher, T.M., Molyneaux, P.L., Saunders, P., Zhang, Y., Gibson, K.F., Kass, D.J., Rojas, M., Sembrat, J., Wolters, P.J., Collard, H.R., Sundy, J.S., O’Riordan, T., Strek, M.E., Noth, I., Ma, S-F, Porteous, M.K., Kreider, M.E., Patel, N.B., Inoue, Y., Hirose, M., Arai, T., Akagawa, S., Eickelberg, O., Fernandez, I.E., Behr, J., Mogulkoc, N., Corte, T.J., Glaspole, I., Tomassetti, S., Ravaglia, C., Poletti, V., Crestani, B., Borie, R., Kannengiesser, C., Parfrey, H., Fiddler, C., Rassl, D., Molina-Molina, M., Machahua, C., Worboys, A.M., Gudmundsson, G., Isaksson, H.J., Lederer, D.J., Podolanczuk, A.J., Montesi, S.B., Bendstrup, E., Danchel, V., Selman, M., Pardo, A., Henry, M.T., Keane, M.P., Doran, P., Vašáková, M., Sterclova, M., Ryerson, C.J., Wilcox, P.G., Okamoto, T., Furusawa, H., Miyazaki, Y., Laurent, G., Baltic, S., Prêle, C., Moodley, Y., Shea, B.S., Ohta, K., Suzukawa, M., Narumoto, O., Nathan, S.D., Venuto, D.C., Woldehanna, M.L., Kokturk, N., de Andrade, J.A., Luckhardt, T., Kulkarni, T., Bonella, F., Donnelly, S.C., McElroy, A., Armstong, M.E., Aranda, A., Carbone, R.G., Puppo, F., Beckman, K.B., Nickerson, D.A., Fingerlin, T.E., and Schwartz, D.A.

Abstract

Rationale: Several common and rare genetic variants have been associated with idiopathic pulmonary fibrosis, a progressive fibrotic condition that is localized to the lung. Objectives: To develop an integrated understanding of the rare and common variants located in multiple loci that have been reported to contribute to the risk of disease. Methods: We performed deep targeted resequencing (3.69 Mb of DNA) in cases (n = 3,624) and control subjects (n = 4,442) across genes and regions previously associated with disease. We tested for associations between disease and 1) individual common variants via logistic regression and 2) groups of rare variants via sequence kernel association tests. Measurements and Main Results: Statistically significant common variant association signals occurred in all 10 of the regions chosen based on genome-wide association studies. The strongest risk variant is the MUC5B promoter variant rs35705950, with an odds ratio of 5.45 (95% confidence interval, 4.91–6.06) for one copy of the risk allele and 18.68 (95% confidence interval, 13.34–26.17) for two copies of the risk allele (P = 9.60 × 10−295). In addition to identifying for the first time that rare variation in FAM13A is associated with disease, we confirmed the role of rare variation in the TERT and RTEL1 gene regions in the risk of IPF, and found that the FAM13A and TERT regions have independent common and rare variant signals. Conclusions: A limited number of common and rare variants contribute to the risk of idiopathic pulmonary fibrosis in each of the resequencing regions, and these genetic variants focus on biological mechanisms of host defense and cell senescence.

Published
2019

27. Influence of Antisynthetase Antibodies Specificities on Antisynthetase Syndrome Clinical Spectrum Time Course

Author

Cavagna, L, Trallero-Araguás, E, Meloni, F, Cavazzana, I, Rojas-Serrano, J, Feist, E, Zanframundo, G, Morandi, V, Meyer, A, Pereira da Silva, J, Matos Costa, C, Molberg, O, Andersson, H, Codullo, V, Mosca, M, Barsotti, S, Neri, R, Scirè, C, Govoni, M, Furini, F, Lopez-Longo, F, Martinez-Barrio, J, Schneider, U, Lorenz, H, Doria, A, Ghirardello, A, Ortego-Centeno, N, Confalonieri, M, Tomietto, P, Pipitone, N, Rodriguez Cambron, A, Blázquez Cañamero, M, Voll, R, Wendel, S, Scarpato, S, Maurier, F, Limonta, M, Colombelli, P, Giannini, M, Geny, B, Arrigoni, E, Bravi, E, Migliorini, P, Mathieu, A, Piga, M, Drott, U, Delbrueck, C, Bauhammer, J, Cagnotto, G, Vancheri, C, Sambataro, G, De Langhe, E, Sainaghi, P, Monti, C, Gigli Berzolari, F, Romano, M, Bonella, F, Specker, C, Schwarting, A, Villa Blanco, I, Selmi, C, Ceribelli, A, Nuno, L, Mera-Varela, A, Perez Gomez, N, Fusaro, E, Parisi, S, Sinigaglia, L, Del Papa, N, Benucci, M, Cimmino, M, Riccieri, V, Conti, F, Sebastiani, G, Iuliano, A, Emmi, G, Cammelli, D, Sebastiani, M, Manfredi, A, Bachiller-Corral, J, Sifuentes Giraldo, W, Paolazzi, G, Saketkoo, L, Giorgi, R, Salaffi, F, Cifrian, J, Caporali, R, Locatelli, F, Marchioni, E, Pesci, A, Dei, G, Pozzi, M, Claudia, L, Distler, J, Knitza, J, Schett, G, Iannone, F, Fornaro, M, Franceschini, F, Quartuccio, L, Gerli, R, Bartoloni, E, Bellando Randone, S, Zampogna, G, Gonzalez Perez, M, Mejia, M, Vicente, E, Triantafyllias, K, Lopez-Mejias, R, Matucci-Cerinic, M, Selva-O'Callaghan, A, Castañeda, S, Montecucco, C, Gonzalez-Gay, M, Cavagna, Lorenzo, Trallero-Araguás, Ernesto, Meloni, Federica, Cavazzana, Ilaria, Rojas-Serrano, Jorge, Feist, Eugen, Zanframundo, Giovanni, Morandi, Valentina, Meyer, Alain, Pereira da Silva, Jose Antonio, Matos Costa, Carlo Jorge, Molberg, Oyvind, Andersson, Helena, Codullo, Veronica, Mosca, Marta, Barsotti, Simone, Neri, Rossella, Scirè, Carlo, Govoni, Marcello, Furini, Federica, Lopez-Longo, Francisco Javier, Martinez-Barrio, Julia, Schneider, Udo, Lorenz, Hanns-Martin, Doria, Andrea, Ghirardello, Anna, Ortego-Centeno, Norberto, Confalonieri, Marco, Tomietto, Paola, Pipitone, Nicolò, Rodriguez Cambron, Ana Belen, Blázquez Cañamero, María Ángeles, Voll, Reinhard Edmund, Wendel, Sarah, Scarpato, Salvatore, Maurier, Francois, Limonta, Massimiliano, Colombelli, Paolo, Giannini, Margherita, Geny, Bernard, Arrigoni, Eugenio, Bravi, Elena, Migliorini, Paola, Mathieu, Alessandro, Piga, Matteo, Drott, Ulrich, Delbrueck, Christiane, Bauhammer, Jutta, Cagnotto, Giovanni, Vancheri, Carlo, Sambataro, Gianluca, De Langhe, Ellen, Sainaghi, Pier Paolo, Monti, Cristina, Gigli Berzolari, Francesca, Romano, Mariaeva, Bonella, Francesco, Specker, Christof, Schwarting, Andreas, Villa Blanco, Ignacio, Selmi, Carlo, Ceribelli, Angela, Nuno, Laura, Mera-Varela, Antonio, Perez Gomez, Nair, Fusaro, Enrico, Parisi, Simone, Sinigaglia, Luigi, Del Papa, Nicoletta, Benucci, Maurizio, Cimmino, Marco Amedeo, Riccieri, Valeria, Conti, Fabrizio, Sebastiani, Gian Domenico, Iuliano, Annamaria, Emmi, Giacomo, Cammelli, Daniele, Sebastiani, Marco, Manfredi, Andreina, Bachiller-Corral, Javier, Sifuentes Giraldo, Walter Alberto, Paolazzi, Giuseppe, Saketkoo, Lesley Ann, Giorgi, Roberto, Salaffi, Fausto, Cifrian, Jose, Caporali, Roberto, Locatelli, Francesco, Marchioni, Enrico, Pesci, Alberto, Dei, Giulia, Pozzi, Maria Rosa, Claudia, Lomater, Distler, Jorg, Knitza, Johannes, Schett, George, Iannone, Florenzo, Fornaro, Marco, Franceschini, Franco, Quartuccio, Luca, Gerli, Roberto, Bartoloni, Elena, Bellando Randone, Silvia, Zampogna, Giuseppe, Gonzalez Perez, Montserrat I, Mejia, Mayra, Vicente, Esther, Triantafyllias, Konstantinos, Lopez-Mejias, Raquel, Matucci-Cerinic, Marco, Selva-O'Callaghan, Albert, Castañeda, Santos, Montecucco, Carlomaurizio, Gonzalez-Gay, Miguel Angel, Cavagna, L, Trallero-Araguás, E, Meloni, F, Cavazzana, I, Rojas-Serrano, J, Feist, E, Zanframundo, G, Morandi, V, Meyer, A, Pereira da Silva, J, Matos Costa, C, Molberg, O, Andersson, H, Codullo, V, Mosca, M, Barsotti, S, Neri, R, Scirè, C, Govoni, M, Furini, F, Lopez-Longo, F, Martinez-Barrio, J, Schneider, U, Lorenz, H, Doria, A, Ghirardello, A, Ortego-Centeno, N, Confalonieri, M, Tomietto, P, Pipitone, N, Rodriguez Cambron, A, Blázquez Cañamero, M, Voll, R, Wendel, S, Scarpato, S, Maurier, F, Limonta, M, Colombelli, P, Giannini, M, Geny, B, Arrigoni, E, Bravi, E, Migliorini, P, Mathieu, A, Piga, M, Drott, U, Delbrueck, C, Bauhammer, J, Cagnotto, G, Vancheri, C, Sambataro, G, De Langhe, E, Sainaghi, P, Monti, C, Gigli Berzolari, F, Romano, M, Bonella, F, Specker, C, Schwarting, A, Villa Blanco, I, Selmi, C, Ceribelli, A, Nuno, L, Mera-Varela, A, Perez Gomez, N, Fusaro, E, Parisi, S, Sinigaglia, L, Del Papa, N, Benucci, M, Cimmino, M, Riccieri, V, Conti, F, Sebastiani, G, Iuliano, A, Emmi, G, Cammelli, D, Sebastiani, M, Manfredi, A, Bachiller-Corral, J, Sifuentes Giraldo, W, Paolazzi, G, Saketkoo, L, Giorgi, R, Salaffi, F, Cifrian, J, Caporali, R, Locatelli, F, Marchioni, E, Pesci, A, Dei, G, Pozzi, M, Claudia, L, Distler, J, Knitza, J, Schett, G, Iannone, F, Fornaro, M, Franceschini, F, Quartuccio, L, Gerli, R, Bartoloni, E, Bellando Randone, S, Zampogna, G, Gonzalez Perez, M, Mejia, M, Vicente, E, Triantafyllias, K, Lopez-Mejias, R, Matucci-Cerinic, M, Selva-O'Callaghan, A, Castañeda, S, Montecucco, C, Gonzalez-Gay, M, Cavagna, Lorenzo, Trallero-Araguás, Ernesto, Meloni, Federica, Cavazzana, Ilaria, Rojas-Serrano, Jorge, Feist, Eugen, Zanframundo, Giovanni, Morandi, Valentina, Meyer, Alain, Pereira da Silva, Jose Antonio, Matos Costa, Carlo Jorge, Molberg, Oyvind, Andersson, Helena, Codullo, Veronica, Mosca, Marta, Barsotti, Simone, Neri, Rossella, Scirè, Carlo, Govoni, Marcello, Furini, Federica, Lopez-Longo, Francisco Javier, Martinez-Barrio, Julia, Schneider, Udo, Lorenz, Hanns-Martin, Doria, Andrea, Ghirardello, Anna, Ortego-Centeno, Norberto, Confalonieri, Marco, Tomietto, Paola, Pipitone, Nicolò, Rodriguez Cambron, Ana Belen, Blázquez Cañamero, María Ángeles, Voll, Reinhard Edmund, Wendel, Sarah, Scarpato, Salvatore, Maurier, Francois, Limonta, Massimiliano, Colombelli, Paolo, Giannini, Margherita, Geny, Bernard, Arrigoni, Eugenio, Bravi, Elena, Migliorini, Paola, Mathieu, Alessandro, Piga, Matteo, Drott, Ulrich, Delbrueck, Christiane, Bauhammer, Jutta, Cagnotto, Giovanni, Vancheri, Carlo, Sambataro, Gianluca, De Langhe, Ellen, Sainaghi, Pier Paolo, Monti, Cristina, Gigli Berzolari, Francesca, Romano, Mariaeva, Bonella, Francesco, Specker, Christof, Schwarting, Andreas, Villa Blanco, Ignacio, Selmi, Carlo, Ceribelli, Angela, Nuno, Laura, Mera-Varela, Antonio, Perez Gomez, Nair, Fusaro, Enrico, Parisi, Simone, Sinigaglia, Luigi, Del Papa, Nicoletta, Benucci, Maurizio, Cimmino, Marco Amedeo, Riccieri, Valeria, Conti, Fabrizio, Sebastiani, Gian Domenico, Iuliano, Annamaria, Emmi, Giacomo, Cammelli, Daniele, Sebastiani, Marco, Manfredi, Andreina, Bachiller-Corral, Javier, Sifuentes Giraldo, Walter Alberto, Paolazzi, Giuseppe, Saketkoo, Lesley Ann, Giorgi, Roberto, Salaffi, Fausto, Cifrian, Jose, Caporali, Roberto, Locatelli, Francesco, Marchioni, Enrico, Pesci, Alberto, Dei, Giulia, Pozzi, Maria Rosa, Claudia, Lomater, Distler, Jorg, Knitza, Johannes, Schett, George, Iannone, Florenzo, Fornaro, Marco, Franceschini, Franco, Quartuccio, Luca, Gerli, Roberto, Bartoloni, Elena, Bellando Randone, Silvia, Zampogna, Giuseppe, Gonzalez Perez, Montserrat I, Mejia, Mayra, Vicente, Esther, Triantafyllias, Konstantinos, Lopez-Mejias, Raquel, Matucci-Cerinic, Marco, Selva-O'Callaghan, Albert, Castañeda, Santos, Montecucco, Carlomaurizio, and Gonzalez-Gay, Miguel Angel

Abstract

Antisynthetase syndrome (ASSD) is a rare clinical condition that is characterized by the occurrence of a classic clinical triad, encompassing myositis, arthritis, and interstitial lung disease (ILD), along with specific autoantibodies that are addressed to different aminoacyl tRNA synthetases (ARS). Until now, it has been unknown whether the presence of a different ARS might affect the clinical presentation, evolution, and outcome of ASSD. In this study, we retrospectively recorded the time of onset, characteristics, clustering of triad findings, and survival of 828 ASSD patients (593 anti-Jo1, 95 anti-PL7, 84 anti-PL12, 38 anti-EJ, and 18 anti-OJ), referring to AENEAS (American and European NEtwork of Antisynthetase Syndrome) collaborative group's cohort. Comparisons were performed first between all ARS cases and then, in the case of significance, while using anti-Jo1 positive patients as the reference group. The characteristics of triad findings were similar and the onset mainly began with a single triad finding in all groups despite some differences in overall prevalence. The "ex-novo" occurrence of triad findings was only reduced in the anti-PL12-positive cohort, however, it occurred in a clinically relevant percentage of patients (30%). Moreover, survival was not influenced by the underlying anti-aminoacyl tRNA synthetase antibodies' positivity, which confirmed that antisynthetase syndrome is a heterogeneous condition and that antibody specificity only partially influences the clinical presentation and evolution of this condition.

Published
2019

28. Video-kapillarmikroskopische Veränderungen bei Patienten mit Antisynthetase-Syndrom. Ergebnisse einer internationalen, multizentrischen Studie des American and European Network of Antisynthetase Syndrome (AENEAS)

Author

Triantafyllias, K, Sebastiani, M, Manfredi, A, Codullo, V, Schwarting, A, Drott, U, Delbrück, C, Weinmann-Menke, J, Bauhammer, J, Miehle, N, Bonella, F, Bergner, R, Specker, C, Lütgendorf, F, Cavagna, L, Triantafyllias, K, Sebastiani, M, Manfredi, A, Codullo, V, Schwarting, A, Drott, U, Delbrück, C, Weinmann-Menke, J, Bauhammer, J, Miehle, N, Bonella, F, Bergner, R, Specker, C, Lütgendorf, F, and Cavagna, L

Published
2019

29. The Burden of Sarcoidosis Symptoms from a Patient Perspective

Author

Longziekten, Voortman, M., Hendriks, C. M.R., Elfferich, M. D.P., Bonella, F., Møller, J., De Vries, J., Costabel, U., Drent, M., Longziekten, Voortman, M., Hendriks, C. M.R., Elfferich, M. D.P., Bonella, F., Møller, J., De Vries, J., Costabel, U., and Drent, M.

Published
2019

30. Research highlights from the 2018 ERS International Congress: interstitial lung diseases

Author

Alfaro, T, Moor, Karen, Alfieri, V, Jeny, F, Kreuter, M, Wijsenbeek - Lourens, Marlies, Renzoni, E A, Bargagli, E, Nunes, H, Spagnolo, P, Bonella, F, Molina-Molina, M, Antoniou, KM, Poletti, V, Alfaro, T, Moor, Karen, Alfieri, V, Jeny, F, Kreuter, M, Wijsenbeek - Lourens, Marlies, Renzoni, E A, Bargagli, E, Nunes, H, Spagnolo, P, Bonella, F, Molina-Molina, M, Antoniou, KM, and Poletti, V

Published
2019

37. Research highlights from the 2018 ERS International Congress

Author

Alfaro, T.M. (Tiago), Moor, C.C. (Karen), Alfieri, V. (Veronica), Jeny, F. (Florence), Kreuter, M. (Michael), Wijsenbeek-Lourens, M.S. (Marlies), Renzoni, E. A. (Elisabetta), Bargagli, E. (Elena), Nunes, H. (Hilario), Spagnolo, P. (Paolo), Bonella, F. (Francesco), Molina-Molina, M. (Maria), Antoniou, K.M. (Katerina), Poletti, V. (Venerino), Alfaro, T.M. (Tiago), Moor, C.C. (Karen), Alfieri, V. (Veronica), Jeny, F. (Florence), Kreuter, M. (Michael), Wijsenbeek-Lourens, M.S. (Marlies), Renzoni, E. A. (Elisabetta), Bargagli, E. (Elena), Nunes, H. (Hilario), Spagnolo, P. (Paolo), Bonella, F. (Francesco), Molina-Molina, M. (Maria), Antoniou, K.M. (Katerina), and Poletti, V. (Venerino)

Abstract

This article reviews a selection of the scientific presentations on interstitial lung disease (ILD)/diffuse parenchymal lung disease (DPLD) that were made at the 2018 European Respiratory Society (ERS) International Congress in Paris. A number of advances in the epidemiology, pathogenesis, diagnosis and treatment of these disorders were presented and discussed by clinicians and researchers. The research topics span over all four groups of ERS Assembly 12: Interstitial Lung Diseases.

Published
2018
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38. European Respiratory Society International Congress 2017: highlights from the Clinical Assembly

Author

Kahn, N, Mekov, E, Fregonese, L, Andrianopoulos, V, Franssen, F, Grgic, A, Coolen, J, Bonta, P, Gompelmann, D, Annema, J, Faverio, P, Bonella, F, Daines, L, Pinnock, H, Kocks, J, Herth, F, Kahn, Nicolas, Mekov, Evgeni, Fregonese, Laura, Andrianopoulos, Vasileios, Franssen, Frits M E, Grgic, Aleksandar, Coolen, Johan, Bonta, Peter I, Gompelmann, Daniela, Annema, Jouke T, Faverio, Paola, Bonella, Francesco, Daines, Luke, Pinnock, Hillary, Kocks, Janwillem, Herth, Felix, Kahn, N, Mekov, E, Fregonese, L, Andrianopoulos, V, Franssen, F, Grgic, A, Coolen, J, Bonta, P, Gompelmann, D, Annema, J, Faverio, P, Bonella, F, Daines, L, Pinnock, H, Kocks, J, Herth, F, Kahn, Nicolas, Mekov, Evgeni, Fregonese, Laura, Andrianopoulos, Vasileios, Franssen, Frits M E, Grgic, Aleksandar, Coolen, Johan, Bonta, Peter I, Gompelmann, Daniela, Annema, Jouke T, Faverio, Paola, Bonella, Francesco, Daines, Luke, Pinnock, Hillary, Kocks, Janwillem, and Herth, Felix

Abstract

This article contains highlights and a selection of the scientific advances from the European Respiratory Society's Clinical Assembly (Assembly 1 and its six respective groups) that were presented at the 2017 European Respiratory Society International Congress in Milan, Italy. The most relevant topics from each of the groups will be discussed, covering a wide range of areas including clinical problems, rehabilitation and chronic care, thoracic imaging, interventional pulmonology, diffuse and parenchymal lung diseases, and general practice and primary care. In this comprehensive review, the newest research and actual data as well as award-winning abstracts and highlight sessions will be discussed.

Published
2018

39. Timing of onset affects arthritis presentation pattern in antisyntethase syndrome

Author

González-Gay, M, Montecucco, C, Selva-O'Callaghan, A, Trallero-Araguas, E, Molberg, O, Andersson, H, Rojas-Serrano, J, Perez-Roman, D, Bauhammer, J, Fiehn, C, Neri, R, Barsotti, S, Lorenz, H, Doria, A, Ghirardello, A, Iannone, F, Giannini, M, Franceschini, F, Cavazzana, I, Triantafyllias, K, Benucci, M, Infantino, M, Manfredi, M, Conti, F, Schwarting, A, Sebastiani, G, Iuliano, A, Emmi, G, Silvestri, E, Govoni, M, Scirè, C, Furini, F, Lopez-Longo, F, Martínez-Barrio, J, Sebastiani, M, Manfredi, A, Bachiller-Corral, J, Sifuentes Giraldo, W, Cimmino, M, Cosso, C, Belotti Masserini, A, Cagnotto, G, Codullo, V, Romano, M, Paolazzi, G, Pellerito, R, Saketkoo, L, Ortego-Centeno, N, Quartuccio, L, Batticciotto, A, Bartoloni Bocci, E, Gerli, R, Specker, C, Bravi, E, Selmi, C, Parisi, S, Salaffi, F, Meloni, F, Marchioni, E, Pesci, A, Dei, G, Confalonieri, M, Tomietto, P, Nuno, L, Bonella, F, Pipitone, N, Mera-Valera, A, Perez-Gomez, N, Gerzeli, S, Lopez-Mejias, R, Matos-Costa, C, Pereira da Silva, J, Cifrian, J, Alpini, C, Olivieri, I, Blázquez Cañamero, M, Rodriguez Cambrón, A, Castañeda, S, Cavagna, L, González-Gay, Miguel A, Montecucco, Carlomaurizio, Selva-O'Callaghan, Albert, Trallero-Araguas, Ernesto, Molberg, Ovynd, Andersson, Helena, Rojas-Serrano, Jorge, Perez-Roman, Diana Isabel, Bauhammer, Jutta, Fiehn, Christoph, Neri, Rossella, Barsotti, Simone, Lorenz, Hannes M, Doria, Andrea, Ghirardello, Anna, Iannone, Florenzo, Giannini, Margherita, Franceschini, Franco, Cavazzana, Ilaria, Triantafyllias, Konstantinos, Benucci, Maurizio, Infantino, Maria, Manfredi, Mariangela, Conti, Fabrizio, Schwarting, Andreas, Sebastiani, Giandomenico, Iuliano, Annamaria, Emmi, Giacomo, Silvestri, Elena, Govoni, Marcello, Scirè, Carlo Alberto, Furini, Federica, Lopez-Longo, Francisco Javier, Martínez-Barrio, Julia, Sebastiani, Marco, Manfredi, Andreina, Bachiller-Corral, Javier, Sifuentes Giraldo, Walter Alberto, Cimmino, Marco A, Cosso, Claudio, Belotti Masserini, Alessandro, Cagnotto, Giovanni, Codullo, Veronica, Romano, Mariaeva, Paolazzi, Giuseppe, Pellerito, Raffaele, Saketkoo, Lesley Ann, Ortego-Centeno, Norberto, Quartuccio, Luca, Batticciotto, Alberto, Bartoloni Bocci, Elena, Gerli, Roberto, Specker, Christof, Bravi, Elena, Selmi, Carlo, Parisi, Simone, Salaffi, Fausto, Meloni, Federica, Marchioni, Enrico, Pesci, Alberto, Dei, Giulia, Confalonieri, Marco, Tomietto, Paola, Nuno, Laura, Bonella, Francesco, Pipitone, Nicolò, Mera-Valera, Antonio, Perez-Gomez, Nair, Gerzeli, Simone, Lopez-Mejias, Raquel, Matos-Costa, Carlo Jorge, Pereira da Silva, Jose Antonio, Cifrian, José, Alpini, Claudia, Olivieri, Ignazio, Blázquez Cañamero, María Ángeles, Rodriguez Cambrón, Ana Belén, Castañeda, Santos, Cavagna, Lorenzo, González-Gay, M, Montecucco, C, Selva-O'Callaghan, A, Trallero-Araguas, E, Molberg, O, Andersson, H, Rojas-Serrano, J, Perez-Roman, D, Bauhammer, J, Fiehn, C, Neri, R, Barsotti, S, Lorenz, H, Doria, A, Ghirardello, A, Iannone, F, Giannini, M, Franceschini, F, Cavazzana, I, Triantafyllias, K, Benucci, M, Infantino, M, Manfredi, M, Conti, F, Schwarting, A, Sebastiani, G, Iuliano, A, Emmi, G, Silvestri, E, Govoni, M, Scirè, C, Furini, F, Lopez-Longo, F, Martínez-Barrio, J, Sebastiani, M, Manfredi, A, Bachiller-Corral, J, Sifuentes Giraldo, W, Cimmino, M, Cosso, C, Belotti Masserini, A, Cagnotto, G, Codullo, V, Romano, M, Paolazzi, G, Pellerito, R, Saketkoo, L, Ortego-Centeno, N, Quartuccio, L, Batticciotto, A, Bartoloni Bocci, E, Gerli, R, Specker, C, Bravi, E, Selmi, C, Parisi, S, Salaffi, F, Meloni, F, Marchioni, E, Pesci, A, Dei, G, Confalonieri, M, Tomietto, P, Nuno, L, Bonella, F, Pipitone, N, Mera-Valera, A, Perez-Gomez, N, Gerzeli, S, Lopez-Mejias, R, Matos-Costa, C, Pereira da Silva, J, Cifrian, J, Alpini, C, Olivieri, I, Blázquez Cañamero, M, Rodriguez Cambrón, A, Castañeda, S, Cavagna, L, González-Gay, Miguel A, Montecucco, Carlomaurizio, Selva-O'Callaghan, Albert, Trallero-Araguas, Ernesto, Molberg, Ovynd, Andersson, Helena, Rojas-Serrano, Jorge, Perez-Roman, Diana Isabel, Bauhammer, Jutta, Fiehn, Christoph, Neri, Rossella, Barsotti, Simone, Lorenz, Hannes M, Doria, Andrea, Ghirardello, Anna, Iannone, Florenzo, Giannini, Margherita, Franceschini, Franco, Cavazzana, Ilaria, Triantafyllias, Konstantinos, Benucci, Maurizio, Infantino, Maria, Manfredi, Mariangela, Conti, Fabrizio, Schwarting, Andreas, Sebastiani, Giandomenico, Iuliano, Annamaria, Emmi, Giacomo, Silvestri, Elena, Govoni, Marcello, Scirè, Carlo Alberto, Furini, Federica, Lopez-Longo, Francisco Javier, Martínez-Barrio, Julia, Sebastiani, Marco, Manfredi, Andreina, Bachiller-Corral, Javier, Sifuentes Giraldo, Walter Alberto, Cimmino, Marco A, Cosso, Claudio, Belotti Masserini, Alessandro, Cagnotto, Giovanni, Codullo, Veronica, Romano, Mariaeva, Paolazzi, Giuseppe, Pellerito, Raffaele, Saketkoo, Lesley Ann, Ortego-Centeno, Norberto, Quartuccio, Luca, Batticciotto, Alberto, Bartoloni Bocci, Elena, Gerli, Roberto, Specker, Christof, Bravi, Elena, Selmi, Carlo, Parisi, Simone, Salaffi, Fausto, Meloni, Federica, Marchioni, Enrico, Pesci, Alberto, Dei, Giulia, Confalonieri, Marco, Tomietto, Paola, Nuno, Laura, Bonella, Francesco, Pipitone, Nicolò, Mera-Valera, Antonio, Perez-Gomez, Nair, Gerzeli, Simone, Lopez-Mejias, Raquel, Matos-Costa, Carlo Jorge, Pereira da Silva, Jose Antonio, Cifrian, José, Alpini, Claudia, Olivieri, Ignazio, Blázquez Cañamero, María Ángeles, Rodriguez Cambrón, Ana Belén, Castañeda, Santos, and Cavagna, Lorenzo

Abstract

Objective To evaluate if the timing of appearance with respect to disease onset may influence the arthritis presentation pattern in antisynthetase syndrome (ASSD). Methods The patients were selected from a retrospective large international cohort of ASSD patients regularly followed-up in centres referring to AENEAS collaborative group. Patients were eligible if they had an antisynthetase antibody testing positive in at least two determinations along with arthritis occurring either at ASSD onset (Group 1) or during the course of the disease (Group 2). Results 445 (70%; 334 females, 110 males, 1 transsexual) out of the 636 ASSD we collected had arthritis, in the majority of cases (367, 83%) from disease onset (Group 1). Patients belonging to Group 1 with respect to Group 2 had an arthritis more commonly polyarticular and symmetrical (p=0.015), IgM-Rheumatoid factor positive (p=0.035), erosions at hands and feet plain x-rays (p=0.036) and more commonly satisfying the 1987 revised classification criteria for rheumatoid arthritis (RA) (p=0.004). Features such as Raynaud's phenomenon, mechanic's hands and fever (e.g. accompanying findings) were more frequently reported in Group 2 (p=0.005). Conclusion In ASSD, the timing of appearance with respect to disease onset influences arthritis characteristics. In particular, RA features are more common when arthritis occurs from ASSD onset, suggesting an overlap between RA and ASSD in these patients. When arthritis appears during the follow-up, it is very close to a connective tissue disease-related arthritis. Also, the different prevalence of accompanying features between these two groups is in line with this possibility

Published
2018

40. Zeitlicher Verlauf klinischer Manifestationen im Vergleich zwischen PL-7-, PL-12- und EJ-positiven Patienten mit Antisynthetase Syndrom

Author

Triantafyllias, K, Cavagna, L, Bauhammer, J, Delbrück, C, Drott, U, Specker, C, Weinmann-Menke, J, Wendel, S, Voll, R, Bonella, F, Lorenz, HM, Lütgendorf, F, Castaneda, S, Gonzalez-Gay, MA, Selva-O' Callaghan, A, and Schwarting, A

Subjects
ddc: 610, 610 Medical sciences, Medicine
Abstract

Einleitung: Arthritis, Myositis und interstitielle Lungenerkrankung (ILD) bilden die klassische klinische Trias des Antisynthetase-Syndroms (ASSD). Bei Jo-1-positiven Patienten können diese Charakteristika auch während der Follow-Up-Periode auftreten. Auch wenn eine ähnliche Tendenz bei[zum vollständigen Text gelangen Sie über die oben angegebene URL], 45. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 31. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), 27. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR)

Published
2017
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41. Supplementary Material for: Antacid Therapy and Disease Progression in Patients with Idiopathic Pulmonary Fibrosis Who Received Pirfenidone

Author

Kreuter, M., Spagnolo, P., Wuyts, W., Renzoni, E., Koschel, D., Bonella, F., Maher, T.M., Kolb, M., Weycker, D., Kirchgässler, K.-U., and Costabel, U.

Subjects
congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Background: Gastroesophageal reflux disease is a potential risk factor for idiopathic pulmonary fibrosis (IPF) progression; however, the impact of antacid therapy (AAT) is under debate. Objective: To evaluate the effect of AAT on IPF progression in pirfenidone-treated patients. Methods: This post hoc analysis included patients with IPF who received pirfenidone in 3 trials (CAPACITY [PIPF-004/PIPF-006] and ASCEND [PIPF-016]). Pulmonary function, exercise tolerance, survival, hospitalizations, and adverse events (AEs) over 52 weeks were analyzed by baseline AAT use. Disease progression was defined as a decrease in forced vital capacity (FVC) of ≥10%, a decrease in 6-min walking distance of ≥50 m, or death over 1 year. Results: Of 623 patients, 44% received AAT. No significant differences were found at 52 weeks (AAT versus non-AAT, respectively) in disease progression (24.9 vs. 30.6%; p = 0.12), all-cause mortality rate (2.9 vs. 4.0%; p = 0.47), IPF-related mortality rate (1.1 vs. 2.0%; p = 0.37), all-cause hospitalization rate (16.1 vs. 18.3%; p = 0.48), or mean change in percent FVC (-2.7 vs. -3.1%; p = 0.44). A relative, but not absolute, FVC decline of ≥10% favored AAT (15 vs. 22%; p = 0.03). Severe gastrointestinal AEs (3.7 vs. 0.9%; p = 0.015) and severe pulmonary infections (3.7 vs. 1.1%; p = 0.035) were more frequent with AAT. Conclusions: AAT and pirfenidone had outcomes comparable to those of pirfenidone alone in patients with IPF, underscoring the need for prospective trials to elucidate the role of AAT with or without antifibrotic drugs as a treatment for IPF.

Published
2017
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42. Diffusing capacity and clinical characteristics of patients with systemic sclerosis – data from the german network for systemic sclerosis

Author

Distler, JHW, Blank, N, Zeidler, G, Moinzadeh, P, Hunzelmann, N, Bonella, F, Kuhr, K, Riemekasten, G, Kreuter, M, Pfeiffer, C, Gaebelein-Wissing, N, Gunther, C, Sunderkoetter, C, Mueller-Ladner, U, Henes, J, Susok, L, Sárdy, M, Aberer, E, Schmeiser, T, Schmalzing, M, Worm, M, Siegert, E, Koetter, I, Krieg, T, Juche, A, and Kreuter, A

Subjects
Medizin
Published
2017

43. DIFFUSING CAPACITY AND CLINICAL CHARACTERISTICS OF PATIENTS WITH SYSTEMIC SCLEROSIS - DATA FROM THE GERMAN NETWORK FOR SYSTEMIC SCLEROSIS

Author

Moinzadeh, P., Blank, N., Siegert, E., Henes, J., Worm, M., Sunderkoetter, C., Schmalzing, M., Kreuter, A., Gunther, C., Susok, L., Zeidler, G., Koetter, I., Mueller-Ladner, U., Krieg, T., Juche, A., Schmeiser, T., Riemekasten, G., Aberer, E., Gaebelein-Wissing, N., Distler, J. H. W., Sardy, M., Pfeiffer, C., Kuhr, K., Hunzelmann, N., Bonella, F., Kreuter, M., Moinzadeh, P., Blank, N., Siegert, E., Henes, J., Worm, M., Sunderkoetter, C., Schmalzing, M., Kreuter, A., Gunther, C., Susok, L., Zeidler, G., Koetter, I., Mueller-Ladner, U., Krieg, T., Juche, A., Schmeiser, T., Riemekasten, G., Aberer, E., Gaebelein-Wissing, N., Distler, J. H. W., Sardy, M., Pfeiffer, C., Kuhr, K., Hunzelmann, N., Bonella, F., and Kreuter, M.

Published
2017

48. Whole lung lavage therapy for pulmonary alveolar proteinosis: a global survey of current practices and procedures

Author

University of Helsinki, Keuhkosairaudet (-2009), Campo, I., Luisetti, M., Griese, M., Trapnell, B.C., Bonella, F., Grutters, J., Nakata, K., Van Moorsel, C.H., Costabel, U., Cottin, V., Ichiwata, T., Inoue, Y., Braschi, A., Bonizzoni, G., Iotti, G.A., Tinelli, C., Rodi, G., Halme, Maija, University of Helsinki, Keuhkosairaudet (-2009), Campo, I., Luisetti, M., Griese, M., Trapnell, B.C., Bonella, F., Grutters, J., Nakata, K., Van Moorsel, C.H., Costabel, U., Cottin, V., Ichiwata, T., Inoue, Y., Braschi, A., Bonizzoni, G., Iotti, G.A., Tinelli, C., Rodi, G., and Halme, Maija

Published
2016

50. Pharmacological Treatment of Idiopathic Pulmonary Fibrosis: Current Approaches, Unsolved Issues, and Future Perspectives

Author

Kreuter, M. (Michael), Bonella, F. (Francesco), Wijsenbeek-Lourens, M.S. (Marlies), Maher, T.M. (Toby M.), Spagnolo, P. (Paolo), Kreuter, M. (Michael), Bonella, F. (Francesco), Wijsenbeek-Lourens, M.S. (Marlies), Maher, T.M. (Toby M.), and Spagnolo, P. (Paolo)

Abstract

Idiopathic pulmonary fibrosis (IPF) is a devastating condition with a 5-year survival of approximately 20%. The disease primarily occurs in elderly patients. IPF is a highly heterogeneous disorder with a clinical course that varies from prolonged periods of stability to episodes of rapid deterioration. In the last decade, improved understanding of disease mechanisms along with a more precise disease definition has allowed the design and completion of a number of high-quality clinical trials. Yet, until recently, IPF was essentially an untreatable disease. Finally, pirfenidone and nintedanib, two compounds with antifibrotic properties, have consistently proven effective in reducing functional decline and disease progression in IPF. This is a major breakthrough for patients and physicians alike, but there is still a long way to go. In fact, neither pirfenidone nor nintedanib is a cure for IPF, and most patients continue to progress despite treatment. As such, comprehensive care of patients with IPF, including manage

Published
2015
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