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1. Long-term outcome of autologous haematopoietic stem cell transplantation in patients with systemic sclerosis: a comparison with patients treated with rituximab and with traditional immunosuppressive agents

Author

Del Papa, Nicoletta, Cavalli, Silvia, Rindone, Andrea, Onida, Francesco, Saporiti, Giorgia, Minniti, Antonina, Pellico, Maria Rosa, Iannone, Claudia, Trignani, Giorgia, D’Angelo, Nicoletta, Sette, Manuel, Greco, Raffaella, Vitali, Claudio, and Caporali, Roberto

Published
2024
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3. Innovative cellular therapies for autoimmune diseases: expert-based position statement and clinical practice recommendations from the EBMT practice harmonization and guidelines committee

Author

Greco, Raffaella, Alexander, Tobias, Del Papa, Nicoletta, Müller, Fabian, Saccardi, Riccardo, Sanchez-Guijo, Fermin, Schett, Georg, Sharrack, Basil, Snowden, John A., Tarte, Karin, Onida, Francesco, Sánchez-Ortega, Isabel, Burman, Joachim, Castilla Llorente, Cristina, Cervera, Ricard, Ciceri, Fabio, Doria, Andrea, Henes, Jörg, Lindsay, James, Mackensen, Andreas, Muraro, Paolo A., Ricart, Elena, Rovira, Montserrat, Zuckerman, Tsila, Yakoub-Agha, Ibrahim, and Farge, Dominique

Published
2024
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4. Does the Impact of COVID‐19 on Patients With Systemic Sclerosis Change Over Time?

Author

Deibel, Elisabeth, Carreira, Patricia E., Vonk, Madelon, del Papa, Nicoletta, Bečvář, Radim, Guillén‐Del‐Castillo, Alfredo, Campochiaro, Corrado, Poormoghim, Hadi, Liem, Sophie, Lazzaroni, Maria‐Grazia, Giollo, Alessandro, Mekinian, Arsène, de Vries‐Bouwstra, Jeska, De Santis, Maria, Balbir‐Gurman, Alexandra, Mihai, Carina, De Luca, Giacomo, Moiseev, Sergey, Zanatta, Elisabetta, Foti, Rosario, Rednic, Simona, Denton, Christopher, Cutolo, Maurizio, Belloli, Laura, Airo, Paolo, Garzanova, Liudmila, Moroncini, Gianluca, İnanç, Murat, Panopoulos, Stylianos, Tandaipan, Jose‐Luis, Chatelus, Emmanuel, Rosato, Edoardo, Kuwana, Masataka, Yavuz, Sule, Alegre‐Sancho, Juan J., Smith, Vanessa, Szűcs, Gabriella, Henes, Joerg, Rodríguez‐Pintó, Ignasi, Atzeni, Fabiola, Spierings, Julia, Truchetet, Marie‐Elise, Milchert, Marcin, Brito de Araujo, Daniel, Riemekasten, Gabriela, Bernardino, Vera, Martin, Thierry, del Galdo, Francesco, Vacca, Alessandra, Mendoza, Fabian, Midtvedt, Øyvind, Murdaca, Giuseppe, Santiago, Tânia, Codullo, Veronica, Cacciapaglia, Fabio, Walker, Ulrich, Brunborg, Cathrine, Tirelli, Francesca, Allanore, Yannick, Furst, Daniel E., Matucci, Marco, Gabrielli, Armando, Distler, Oliver, and Hoffmann‐Vold, Anna‐Maria

Published
2024
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5. Rituximab retention rate in systemic sclerosis: a long term real-life multicenter study

Author

De Luca, Giacomo, primary, De Lorenzis, Enrico, additional, Campochiaro, Corrado, additional, Cacciapaglia, Fabio, additional, Del Papa, Nicoletta, additional, Zanatta, Elisabetta, additional, Airò, Paolo, additional, Lazzaroni, Maria Grazia, additional, Giuggioli, Dilia, additional, De Santis, Maria, additional, Alonzi, Gabriella, additional, Stano, Stefano, additional, Binda, Marco, additional, Moccaldi, Beatrice, additional, Tonutti, Antonio, additional, Cavalli, Silvia, additional, Batani, Veronica, additional, Natalello, Gerlando, additional, Iannone, Florenzo, additional, D’Agostino, Maria Antonietta, additional, Dagna, Lorenzo, additional, Matucci-Cerinic, Marco, additional, and Bosello, Silvia Laura, additional

Published
2024
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7. Response to antifibrotic therapy and decrease of circulating BAG3 protein levels in systemic sclerosis patients with reduced forced vital capacity

Author

De Marco, Margot, primary, Basile, Anna, additional, Cammarota, Anna Lisa, additional, Iannone, Claudia, additional, Falco, Antonia, additional, Marzullo, Liberato, additional, Rosati, Alessandra, additional, Caporali, Roberto, additional, Turco, Maria Caterina, additional, and Del Papa, Nicoletta, additional

Published
2024
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10. Does the impact of COVID-19 on patients with systemic sclerosis change over time?

Author

MS Reumatologie/Immunologie/Infectie, Infection & Immunity, Deibel, Elisabeth, Carreira, Patricia E, Vonk, Madelon, Del Papa, Nicoletta, Bečvář, Radim, Guillén-Del-Castillo, Alfredo, Campochiaro, Corrado, Poormoghim, Hadi, Liem, Sophie, Lazzaroni, Maria-Grazia, Giollo, Alessandro, Mekinian, Arsène, de Vries-Bouwstra, Jeska, De Santis, Maria, Balbir-Gurman, Alexandra, Mihai, Carina, De Luca, Giacomo, Moiseev, Sergey, Zanatta, Elisabetta, Foti, Rosario, Rednic, Simona, Denton, Christopher, Cutolo, Maurizio, Belloli, Laura, Airo, Paolo, Garzanova, Liudmila, Moroncini, Gianluca, İnanç, Murat, Panopoulos, Stylianos, Tandaipan, Jose-Luis, Chatelus, Emmanuel, Rosato, Edoardo, Kuwana, Masataka, Yavuz, Sule, Alegre-Sancho, Juan J, Smith, Vanessa, Szűcs, Gabriella, Henes, Joerg, Rodríguez-Pintó, Ignasi, Atzeni, Fabiola, Spierings, Julia, Truchetet, Marie-Elise, Milchert, Marcin, Brito de Araujo, Daniel, Riemekasten, Gabriela, Bernardino, Vera, Martin, Thierry, Del Galdo, Francesco, Vacca, Alessandra, Mendoza, Fabian, Midtvedt, Øyvind, Murdaca, Giuseppe, Santiago, Tânia, Codullo, Veronica, Cacciapaglia, Fabio, Walker, Ulrich, Brunborg, Cathrine, Tirelli, Francesca, Allanore, Yannick, Furst, Daniel E, Matucci, Marco, Gabrielli, Armando, Distler, Oliver, Hoffmann-Vold, Anna-Maria, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, Deibel, Elisabeth, Carreira, Patricia E, Vonk, Madelon, Del Papa, Nicoletta, Bečvář, Radim, Guillén-Del-Castillo, Alfredo, Campochiaro, Corrado, Poormoghim, Hadi, Liem, Sophie, Lazzaroni, Maria-Grazia, Giollo, Alessandro, Mekinian, Arsène, de Vries-Bouwstra, Jeska, De Santis, Maria, Balbir-Gurman, Alexandra, Mihai, Carina, De Luca, Giacomo, Moiseev, Sergey, Zanatta, Elisabetta, Foti, Rosario, Rednic, Simona, Denton, Christopher, Cutolo, Maurizio, Belloli, Laura, Airo, Paolo, Garzanova, Liudmila, Moroncini, Gianluca, İnanç, Murat, Panopoulos, Stylianos, Tandaipan, Jose-Luis, Chatelus, Emmanuel, Rosato, Edoardo, Kuwana, Masataka, Yavuz, Sule, Alegre-Sancho, Juan J, Smith, Vanessa, Szűcs, Gabriella, Henes, Joerg, Rodríguez-Pintó, Ignasi, Atzeni, Fabiola, Spierings, Julia, Truchetet, Marie-Elise, Milchert, Marcin, Brito de Araujo, Daniel, Riemekasten, Gabriela, Bernardino, Vera, Martin, Thierry, Del Galdo, Francesco, Vacca, Alessandra, Mendoza, Fabian, Midtvedt, Øyvind, Murdaca, Giuseppe, Santiago, Tânia, Codullo, Veronica, Cacciapaglia, Fabio, Walker, Ulrich, Brunborg, Cathrine, Tirelli, Francesca, Allanore, Yannick, Furst, Daniel E, Matucci, Marco, Gabrielli, Armando, Distler, Oliver, and Hoffmann-Vold, Anna-Maria

Published
2024

12. Overexpression of BAG3 (Bcl2-Associated Athanogene 3) in Serum and Skin of Patients with Systemic Sclerosis

Author

De Marco, Margot, primary, Armentaro, Giuseppe, additional, Falco, Antonia, additional, Minniti, Antonina, additional, Cammarota, Anna Lisa, additional, Iannone, Claudia, additional, Basile, Anna, additional, D'Ardia, Adriana, additional, Zeppa, Pio, additional, Marzullo, Liberato, additional, Rosati, Alessandra, additional, Vitali, Claudio, additional, Caporali, Roberto, additional, Turco, Maria Caterina, additional, and Del Papa, Nicoletta, additional

Published
2024
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16. Does the impact of COVID‐19 on patients with systemic sclerosis change over time?

Author

Deibel, Elisabeth, primary, Carreira, Patricia E., additional, Vonk, Madelon, additional, del Papa, Nicoletta, additional, Bečvář, Radim, additional, Guillén‐Del‐Castillo, Alfredo, additional, Campochiaro, Corrado, additional, Poormoghim, Hadi, additional, Liem, Sophie, additional, Lazzaroni, Maria‐Grazia, additional, Giollo, Alessandro, additional, Mekinian, Arsène, additional, de Vries‐Bouwstra, Jeska, additional, De Santis, Maria, additional, Balbir‐Gurman, Alexandra, additional, Mihai, Carina, additional, De Luca, Giacomo, additional, Moiseev, Sergey, additional, Zanatta, Elisabetta, additional, Foti, Rosario, additional, Rednic, Simona, additional, Denton, Christopher, additional, Cutolo, Maurizio, additional, Belloli, Laura, additional, Airo, Paolo, additional, Garzanova, Liudmila, additional, Moroncini, Gianluca, additional, İnanç, Murat, additional, Panopoulos, Stylianos, additional, Tandaipan, Jose‐Luis, additional, Chatelus, Emmanuel, additional, Rosato, Edoardo, additional, Kuwana, Masataka, additional, Yavuz, Sule, additional, Alegre‐Sancho, Juan J, additional, Smith, Vanessa, additional, Szűcs, Gabriella, additional, Henes, Joerg, additional, Rodríguez‐Pintó, Ignasi, additional, Atzeni, Fabiola, additional, Spierings, Julia, additional, Truchetet, Marie‐Elise, additional, Milchert, Marcin, additional, Brito de Araujo, Daniel, additional, Riemekasten, Gabriela, additional, Bernardino, Vera, additional, Martin, Thierry, additional, del Galdo, Francesco, additional, Vacca, Alessandra, additional, Mendoza, Fabian, additional, Midtvedt, Øyvind, additional, Murdaca, Giuseppe, additional, Santiago, Tânia, additional, Codullo, Veronica, additional, Cacciapaglia, Fabio, additional, Walker, Ulrich, additional, Brunborg, Cathrine, additional, Tirelli, Francesca, additional, Allanore, Yannick, additional, Furst, Daniel E., additional, Matucci, Marco, additional, Gabrielli, Armando, additional, Distler, Oliver, additional, and Hoffmann‐Vold, Anna‐Maria, additional

Published
2023
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20. Belimumab may decrease flare rate and allow glucocorticoid withdrawal in lupus nephritis (including dialysis and transplanted patient)

Author

Binda, Valentina, Trezzi, Barbara, Del Papa, Nicoletta, Beretta, Lorenzo, Frontini, Giulia, Porata, Giulia, Fabbrini, Paolo, Pozzi, Maria Rosa, Messa, Piergiorgio, Sinico, Renato Alberto, and Moroni, Gabriella

Abstract

Background: Belimumab (Benlysta) is currently approved for the treatment of active Lupus despite standard therapy. Few data are available on the efficacy of this drug in lupus nephritis (LN). Methods: 17 LN female followed in two Nephrology Italian Unit received belimumab for a median period of 36 months (range 6–42 months). The indications were: arthralgia in 3 patients, cutaneous manifestations in 2, residual proteinuria in 8, and the need to reduce steroids for severe side effects in 4. Of interest, 1 patient started therapy during Peritoneal Dialysis and continued after kidney transplantation due to non-responsive arthralgias. Results: Arthralgia and skin manifestations resolved in all patients. Proteinuria normalized in three patients and stabilized in all but one of the others. Steroids were indefinitely stopped in six patients (35%) and reduced to around 40% of the basal dosage in the other patients. During belimumab therapy, three extrarenal and one renal SLE flares were diagnosed accounting for a rate of renal flares of 0.02/patient/year. No major adverse events leading to therapy withdrawal occurred. Clinical case: Arthralgia resolved, immunological parameters improved and prednisone could be reduced within few months in the patient who started belimumab during peritoneal dialysis. After kidney transplantation belimumab was stopped but due to arthralgias unresponsive to standard immunosuppressive therapy it was restarted with success. Conclusions: Belimumab allows the achievement of complete response together with the withdrawal or the reduction of corticosteroids in almost all our patients. Of interest its satisfactory use in a patient in peritoneal dialysis and after kidney transplantation.

Published
2024
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23. Real-life efficacy and safety of nintedanib in systemic sclerosis-interstitial lung disease: data from an Italian multicentre study

Author

Campochiaro, Corrado, primary, De Luca, Giacomo, additional, Lazzaroni, Maria-Grazia, additional, Armentaro, Giuseppe, additional, Spinella, Amelia, additional, Vigone, Barbara, additional, Ruaro, Barbara, additional, Stanziola, Anna, additional, Benfaremo, Devis, additional, De Lorenzis, Enrico, additional, Moccaldi, Beatrice, additional, Bosello, Silvia Laura, additional, Cuomo, Giovanna, additional, Beretta, Lorenzo, additional, Zanatta, Elisabetta, additional, Giuggioli, Dilia, additional, Del Papa, Nicoletta, additional, Airo, Paolo, additional, Confalonieri, Marco, additional, Moroncini, Gianluca, additional, Dagna, Lorenzo, additional, and Matucci-Cerinic, Marco, additional

Published
2023
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25. Profibrotic Effects of Endothelin-1 on Fibroblasts Are Mediated by Aldosterone In Vitro: Relevance to the Pathogenesis and Therapy of Systemic Sclerosis and Pulmonary Arterial Hypertension

Author

Argentino, Giuseppe, primary, Barbieri, Alessandro, additional, Beri, Ruggero, additional, Bason, Caterina, additional, Ruzzenente, Andrea, additional, Olivieri, Oliviero, additional, Tinazzi, Elisa, additional, Puccetti, Antonio, additional, Vitali, Claudio, additional, Del Papa, Nicoletta, additional, Friso, Simonetta, additional, and Lunardi, Claudio, additional

Published
2022
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28. Peripheral Microangiopathy Changes in Pulmonary Arterial Hypertension Related to Systemic Sclerosis: Data From a Multicenter Observational Study

Author

Giuggioli, Dilia, primary, Riccieri, Valeria, additional, Cipolletta, Edoardo, additional, Del Papa, Nicoletta, additional, Ingegnoli, Francesca, additional, Spinella, Amelia, additional, Pellegrino, Greta, additional, Risa, Anna Maria, additional, de Pinto, Marco, additional, Papa, Silvia, additional, Armentaro, Giuseppe, additional, and De Angelis, Rossella, additional

Published
2022
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30. Long-term kidney outcome of patients with rheumatological diseases and antineutrophil cytoplasmic antibody-glomerulonephritis: comparison with a primitive ANCA-glomerulonephritis cohort

Author

Locatelli, Laura, primary, Calatroni, Marta, additional, Reggiani, Francesco, additional, Bonelli, Grazia Dea, additional, Gerosa, Maria, additional, Argolini, Lorenza Maria, additional, Trezzi, Barbara, additional, Del Papa, Nicoletta, additional, Angelini, Claudio, additional, Pozzi, Maria Rosa, additional, Sinico, Renato Alberto, additional, and Moroni, Gabriella, additional

Published
2022
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31. A New Method for the Assessment of Myalgia in Interstitial Lung Disease: Association with Positivity for Myositis-Specific and Myositis-Associated Antibodies

Author

Sambataro, Gianluca, primary, Ferrara, Chiara Alfia, additional, Spadaro, Carla, additional, Torrisi, Sebastiano Emanuele, additional, Vignigni, Giovanna, additional, Vancheri, Ada, additional, Muscato, Giuseppe, additional, Del Papa, Nicoletta, additional, Colaci, Michele, additional, Malatino, Lorenzo, additional, Palmucci, Stefano, additional, Cavagna, Lorenzo, additional, Zanframundo, Giovanni, additional, Ferro, Francesco, additional, Baldini, Chiara, additional, Sambataro, Domenico, additional, and Vancheri, Carlo, additional

Published
2022
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32. Breakthrough Infections in COVID-19 Vaccinated Patients with Systemic Sclerosis: A Survival Analysis from a Multicenter International Patient-Reported Survey

Author

Ahmed, Sakir, Naveen, R., Pauling, John, Thakare, Darpan, Wincup, Chris, Del Papa, Nicoletta, Sambataro, Gianluca, Atzeni, Fabiola, Parisi, Simone, Govoni, Marcello, Bocci, Elena Bartoloni, Sebastiani, Gian Domenico, Fusaro, Enrico, Sebastiani, Marco, Quartuccio, Luca, Franceschini, Franco, Sainaghi, Pier Paolo, Orsolini, Giovanni, Angelis, Rossella, Danielli, Maria Giovanna, Venerito, Vincenzo, Sen, Parikshit, Kim, Minchul, Gracia-Ramos, Abraham Edgar, Akira Yoshida, Lilleker, James B., Agarwal, Vishwesh, Kardes, Sinan, Day, Jessica, Joshi, Mrudula, Milchert, Marcin, Gheita, Tamer A., Salim, Babur, Parodis, Ioannis, Selva O Callaghan, Albert, Nikiphorou, Elena, Chatterjee, Tulika, Tan, Ai Lyn, Nune, Arvind, Cavagna, Lorenzo, Shinjo, Samuel, Ziade, Nelly, Knitza, Johannes, Chinoy, Hector, Distler, Oliver, Kuwana, Masataka, Aggarwal, Rohit, Gupta, Latika, Agarwal, Vikas, and Makol, Ashima

Published
2022

34. BAG3 induces α‐SMA expression in human fibroblasts and its over‐expression correlates with poorer survival in fibrotic cancer patients

Author

De Marco, Margot, primary, Del Papa, Nicoletta, additional, Reppucci, Francesca, additional, Iorio, Vittoria, additional, Basile, Anna, additional, Falco, Antonia, additional, Iaccarino, Roberta, additional, Brongo, Sergio, additional, De Caro, Francesco, additional, Capunzo, Mario, additional, Turco, Maria Caterina, additional, Rosati, Alessandra, additional, and Marzullo, Liberato, additional

Published
2021
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35. Intestinal Microbiome in Hematopoietic Stem Cell Transplantation For Autoimmune Diseases: Considerations and Perspectives on Behalf of Autoimmune Diseases Working Party (ADWP) of the EBMT

Author

Alexander, Tobias, primary, Snowden, John A., additional, Burman, Joachim, additional, Chang, Hyun-Dong, additional, Del Papa, Nicoletta, additional, Farge, Dominique, additional, Lindsay, James O., additional, Malard, Florent, additional, Muraro, Paolo A., additional, Nitti, Rosamaria, additional, Salas, Azucena, additional, Sharrack, Basil, additional, Mohty, Mohamad, additional, and Greco, Raffaella, additional

Published
2021
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38. Impact and outcome of COVID-19 on SSc-ILD

Author

Hoffmann-Vold, Anna Maria, primary, Brunborg, Cathrine, additional, Tirelli, Francesca, additional, Carreira, Patricia E, additional, Del Papa, Nicoletta, additional, Mekinian, Arsène, additional, Vonk, Madelon, additional, Giollo, Alessandro, additional, De Luca, Giacomo, additional, De Santis, Maria, additional, Campochiaro, Corrado, additional, Mihai, Carina, additional, Airo, Paolo, additional, Lazzaroni, Maria-Grazia, additional, Zanatta, Elisabetta, additional, Foti, Rosario, additional, Allanore, Yannick, additional, Furst, Daniel E., additional, Cerinic-Matucci, Marco, additional, Gabrielli, Armando, additional, and Distler, Oliver, additional

Published
2021
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41. Intestinal Microbiome in Hematopoietic Stem Cell Transplantation For Autoimmune Diseases : Considerations and Perspectives on Behalf of Autoimmune Diseases Working Party (ADWP) of the EBMT

Author

Alexander, Tobias, Snowden, John A., Burman, Joachim, Chang, Hyun-Dong, Del Papa, Nicoletta, Farge, Dominique, Lindsay, James O., Malard, Florent, Muraro, Paolo A., Nitti, Rosamaria, Salas, Azucena, Sharrack, Basil, Mohty, Mohamad, Greco, Raffaella, Alexander, Tobias, Snowden, John A., Burman, Joachim, Chang, Hyun-Dong, Del Papa, Nicoletta, Farge, Dominique, Lindsay, James O., Malard, Florent, Muraro, Paolo A., Nitti, Rosamaria, Salas, Azucena, Sharrack, Basil, Mohty, Mohamad, and Greco, Raffaella

Abstract

Over the past decades, hematopoietic stem cell transplantation (HSCT) has been evolving as specific treatment for patients with severe and refractory autoimmune diseases (ADs), where mechanistic studies have provided evidence for a profound immune renewal facilitating the observed beneficial responses. The intestinal microbiome plays an important role in host physiology including shaping the immune repertoire. The relationships between intestinal microbiota composition and outcomes after HSCT for hematologic diseases have been identified, particularly for predicting the mortality from infectious and non-infectious causes. Furthermore, therapeutic manipulations of the gut microbiota, such as fecal microbiota transplant (FMT), have emerged as promising therapeutic approaches for restoring the functional and anatomical integrity of the intestinal microbiota post-transplantation. Although changes in the intestinal microbiome have been linked to various ADs, studies investigating the effect of intestinal dysbiosis on HSCT outcomes for ADs are scarce and require further attention. Herein, we describe some of the landmark microbiome studies in HSCT recipients and patients with chronic ADs, and discuss the challenges and opportunities of microbiome research for diagnostic and therapeutic purposes in the context of HSCT for ADs.

Published
2021
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42. Hematopoietic stem cell transplantation for autoimmune diseases in the time of COVID-19 : EBMT guidelines and recommendations

Author

Greco, Raffaella, Alexander, Tobias, Burman, Joachim, Del Papa, Nicoletta, de Vries-Bouwstra, Jeska, Farge, Dominique, Henes, Joerg, Kazmi, Majid, Kirgizov, Kirill, Muraro, Paolo A., Ricart, Elena, Rovira, Montserrat, Saccardi, Riccardo, Sharrack, Basil, Snarski, Emilian, Withers, Barbara, Jessop, Helen, Boglione, Claudia, Kramer, Ellen, Badoglio, Manuela, Labopin, Myriam, Orchard, Kim, Corbacioglu, Selim, Ljungman, Per, Mikulska, Malgorzata, De la Camara, Rafael, Snowden, John A., Greco, Raffaella, Alexander, Tobias, Burman, Joachim, Del Papa, Nicoletta, de Vries-Bouwstra, Jeska, Farge, Dominique, Henes, Joerg, Kazmi, Majid, Kirgizov, Kirill, Muraro, Paolo A., Ricart, Elena, Rovira, Montserrat, Saccardi, Riccardo, Sharrack, Basil, Snarski, Emilian, Withers, Barbara, Jessop, Helen, Boglione, Claudia, Kramer, Ellen, Badoglio, Manuela, Labopin, Myriam, Orchard, Kim, Corbacioglu, Selim, Ljungman, Per, Mikulska, Malgorzata, De la Camara, Rafael, and Snowden, John A.

Abstract

Coronavirus disease-19 (COVID-19), caused by Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2), represents one of the biggest challenges of 21st century, threatening public health around the globe. Increasing age and presence of co-morbidities are reported risk factors for severe disease and mortality, along with autoimmune diseases (ADs) and immunosuppressive treatments such as haematopoietic stem cell transplantation (HSCT), which are also associated with adverse outcomes. We review the impact of the pandemic on specific groups of patients with neurological, rheumatological, and gastroenterological indications, along with the challenges delivering HSCT in adult and pediatric populations. Moving forward, we developed consensus-based guidelines and recommendations for best practice and quality of patient care in order to support clinicians, scientists, and their multidisciplinary teams, as well as patients and their carers. These guidelines aim to support national and international organizations related to autoimmune diseases and local clinical teams delivering HSCT. Areas of unmet need and future research questions are also highlighted. The waves of the COVID-19 pandemic are predicted to be followed by an "endemic" phase and therefore an ongoing risk within a "new normality". These recommendations reflect currently available evidence, coupled with expert opinion, and will be revised according to necessary modifications in practice.

Published
2021
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43. Interstitial Lung Disease Associated With Autoimmune Rheumatic Diseases: Checklists for Clinical Practice

Author

Bosello, Silvia Laura, Beretta, Lorenzo, Del Papa, Nicoletta, Harari, Sergio, Palmucci, Stefano, Pesci, Alberto, Rechichi, Gilda, Varone, Francesco, Sebastiani, Marco, Bosello, Silvia Laura (ORCID:0000-0002-4837-447X), Bosello, Silvia Laura, Beretta, Lorenzo, Del Papa, Nicoletta, Harari, Sergio, Palmucci, Stefano, Pesci, Alberto, Rechichi, Gilda, Varone, Francesco, Sebastiani, Marco, and Bosello, Silvia Laura (ORCID:0000-0002-4837-447X)

Abstract

Background: Interstitial lung diseases (ILDs) are often associated with rheumatic diseases. Their early diagnosis and management are not only difficult, but also crucial, because they are associated with major morbidity and mortality and can be the first cause of death in autoimmune rheumatic diseases (ARDs).Objectives: By using methodologies, such as Nominal Group Technique (NGT) and Delphi Survey, the aims of this study were (1) to measure consensus between pulmonologists, radiologists, and rheumatologists experienced in the management of ARD-ILD; (2) to highlight the importance of a multidisciplinary approach; and (3) to provide clinicians with a practical tool aimed at improving the prompt recognition and follow-up of ILD associated with ARDs and of any possible rheumatic conditions underlying ILD.Results: During the NGT round, the Steering Committee defined 57 statements to be used in the Delphi survey. A total of 78 experts participated in the Delphi survey, namely 28 pulmonologists, 33 rheumatologists, and 17 radiologists. During this round, consensus on agreement was reached in 47 statements, while disagreement was not reached in any statements. A secondary questionnaire was drafted by the Steering Committee to obtain clearer indications on ILD-ARD "red-flags" and follow-up. Delphi Panelists took part also in the second-questionnaire survey. Answers from both surveys were used to draft two checklists of "red flags" sign or symptom suggestive of ILD and ARD, respectively, and two checklists on identification and monitoring of rheumatoid arthritis (RA) and systemic sclerosis (SSc) ILD.Limitations: This study is a consensus work, which cannot produce empiric data, and is limited to the Italian scenario.Conclusions: This work showed a high level of agreement, but also shows some divergent opinions between different experts. This underlines the importance of a multidisciplinary approach. Eventually, we believe the drafted checklists can help clinicians in the dia

Published
2021

44. A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: The UPSIDE study protocol

Author

MS Reumatologie/Immunologie/Infectie, Infection & Immunity, MS Hematologie, Cancer, Lab Reumatologie/Klinische Immunologie, Regenerative Medicine and Stem Cells, JC onderzoeksprogramma Methodologie, Spierings, Julia, van Rhenen, Anna, Welsing, Paco Mw, Marijnissen, Anne Ca, De Langhe, Ellen, Del Papa, Nicoletta, Dierickx, Daan, Gheorghe, Karina R, Henes, Joerg, Hesselstrand, Roger, Kerre, Tessa, Ljungman, Per, van de Loosdrecht, Arjan A, Marijt, Erik Waf, Mayer, Miro, Schmalzing, Marc, Schroers, Roland, Smith, Vanessa, Voll, Reinhard E, Vonk, Madelon C, Voskuyl, Alexandre E, de Vries-Bouwstra, Jeska K, Walker, Ulrich A, Wuttge, Dirk M, van Laar, Jacob M, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, MS Hematologie, Cancer, Lab Reumatologie/Klinische Immunologie, Regenerative Medicine and Stem Cells, JC onderzoeksprogramma Methodologie, Spierings, Julia, van Rhenen, Anna, Welsing, Paco Mw, Marijnissen, Anne Ca, De Langhe, Ellen, Del Papa, Nicoletta, Dierickx, Daan, Gheorghe, Karina R, Henes, Joerg, Hesselstrand, Roger, Kerre, Tessa, Ljungman, Per, van de Loosdrecht, Arjan A, Marijt, Erik Waf, Mayer, Miro, Schmalzing, Marc, Schroers, Roland, Smith, Vanessa, Voll, Reinhard E, Vonk, Madelon C, Voskuyl, Alexandre E, de Vries-Bouwstra, Jeska K, Walker, Ulrich A, Wuttge, Dirk M, and van Laar, Jacob M

Published
2021

46. Autologous Hematopoietic Stem Cell Transplantation vs Intravenous Pulse Cyclophosphamide in Diffuse Cutaneous Systemic Sclerosis: A Randomized Clinical Trial

Author

van Laar, Jacob M., Farge, Dominique, Sont, Jacob K., Naraghi, Kamran, Marjanovic, Zora, Larghero, Jérôme, Schuerwegh, Annemie J., Marijt, Erik W. A., Vonk, Madelon C., Schattenberg, Anton V., Matucci-Cerinic, Marco, Voskuyl, Alexandre E., van de Loosdrecht, Arjan A., Daikeler, Thomas, Kötter, Ina, Schmalzing, Marc, Martin, Thierry, Lioure, Bruno, Weiner, Stefan M., Kreuter, Alexander, Deligny, Christophe, Durand, Jean-Marc, Emery, Paul, Machold, Klaus P., Sarrot-Reynauld, Francoise, Warnatz, Klaus, Adoue, Daniel F. P., Constans, Joël, Tony, Hans-Peter, Del Papa, Nicoletta, Fassas, Athanasios, Himsel, Andrea, Launay, David, Lo Monaco, Andrea, Philippe, Pierre, Quéré, Isabelle, Rich, Éric, Westhovens, Rene, Griffiths, Bridget, Saccardi, Riccardo, van den Hoogen, Frank H., Fibbe, Willem E., Socié, Gérard, Gratwohl, Alois, and Tyndall, Alan

Published
2014
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48. "Usual" interstitial pneumonia with autoimmune features: a prospective study on a cohort of idiopathic pulmonary fibrosis patients

Author

Sambataro, Gianluca, primary, Ferrara, Chiara A., additional, Torrisi, Sebastiano E., additional, Spadaro, Carla, additional, Vignigni, Giovanna, additional, Vancheri, Ada, additional, Del Papa, Nicoletta, additional, Orlandi, Martina, additional, Colaci, Michele, additional, Malatino, Lorenzo, additional, Palmucci, Stefano, additional, Cavagna, Lorenzo, additional, Sambataro, Domenico, additional, and Vancheri, Carlo, additional

Published
2021
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49. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini‐Gutzwiller, Federica R., Snowden, John A., Corbacioglu, Selim, Greco, Raffaella, Alexander, Tobias, Snowden, John, Badoglio, Manuela, Labopin, Myriam, Abinun, Mario, Apte, Shashikant, Arnold, Renate, Domenech, Ariadna, Brierley, Charlotte, Burman, Joachim, Castilla‐Llorente, Cristina, Cooper, Nichola, Daghia, Giulia, Daikeler, Thomas, del Papa, Nicoletta, and de Vries‐Bouwstra, Jeska

Subjects
HEMATOPOIETIC stem cell transplantation, AUTOIMMUNE diseases, JUVENILE diseases, PEDIATRICS, BONE marrow, LITERATURE reviews
Abstract

Summary: Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long‐term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment‐refractory autoimmune diseases (ADs) in children. However, transplant‐related outcomes are disease‐dependent and long‐term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non‐transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long‐term disease control or cure, but therapy‐related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence‐based interspeciality guidelines and recommendations. [ABSTRACT FROM AUTHOR]

Published
2022
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