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184 results on '"Badoglio, M"'

1. High-dose chemotherapy with autologous stem cell transplants in adult primary non-seminoma mediastinal germ-cell tumors. A report from the Cellular Therapy and Immunobiology working party of the EBMT

Author

Secondino, S., Badoglio, M., Rosti, G., Labopin, M., Delaye, M., Bokemeyer, C., Seidel, C., Kanfer, E., Metafuni, E., Finke, J., Bouhris, J.-H., Kosmas, C., Malard, F., Pagani, A., Kuball, J., Koehl, U., Ruggeri, A., De Giorgi, U., and Pedrazzoli, P.

Published
2024
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2. High-dose chemotherapy with autologous stem cell transplants in adult primary non-seminoma mediastinal germ-cell tumors. A report from the Cellular Therapy and Immunobiology working party of the EBMT

Author

CTI Kuball, MS Hematologie, Cancer, Infection & Immunity, Regenerative Medicine and Stem Cells, Secondino, S, Badoglio, M, Rosti, G, Labopin, M, Delaye, M, Bokemeyer, C, Seidel, C, Kanfer, E, Metafuni, E, Finke, J, Bouhris, J-H, Kosmas, C, Malard, F, Pagani, A, Kuball, J, Koehl, U, Ruggeri, A, De Giorgi, U, Pedrazzoli, P, EBMT Cellular Therapy & Immunobiology WP, CTI Kuball, MS Hematologie, Cancer, Infection & Immunity, Regenerative Medicine and Stem Cells, Secondino, S, Badoglio, M, Rosti, G, Labopin, M, Delaye, M, Bokemeyer, C, Seidel, C, Kanfer, E, Metafuni, E, Finke, J, Bouhris, J-H, Kosmas, C, Malard, F, Pagani, A, Kuball, J, Koehl, U, Ruggeri, A, De Giorgi, U, Pedrazzoli, P, and EBMT Cellular Therapy & Immunobiology WP

Published
2024

5. Evaluating the clinical effectiveness of autologous haematopoietic stem cell transplantation versus disease-modifying therapy in multiple sclerosis using a matching-adjusted indirect comparison: an exploratory study from the Autoimmune Diseases Working Party (ADWP) of the European Society of Bone and Marrow Transplantation (EBMT)

Author

Tappenden, P., Wang, Y., Sharrack, B., Burman, J., Kazmi, M., Saccardi, R., Bermejo, I., Harvey, R., Badoglio, M., Farge, D., and Snowden, J. A.

Published
2020
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8. 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, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., Yesilipek A., Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., and Yesilipek A.

Abstract

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.

Published
2022

10. 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, FR, Snowden, JA, Corbacioglu, S, Greco, R, Alexander, T, Snowden, J, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla‐Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries‐Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, HU, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, CH, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, MG, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, AP, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, and Yesilipek, A

Subjects
autoimmune diseases, haematopoietic stem cell transplantation, paediatric, ddc:610, paediatric, surgical procedures, operative, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, haematopoietic stem cell transplantation, 610 Medizin, autoimmune diseases, autoimmune disease, Hematology
Abstract

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 (

Published
2022

13. Prognostic impact of progression to induction chemotherapy and prior paclitaxel therapy in patients with germ cell tumors receiving salvage high-dose chemotherapy in the last 10 years: a study of the European Society for Blood and Marrow Transplantation Solid Tumors Working Party

Author

Necchi, A, Miceli, R, Bregni, M, Bokemeyer, C, Berger, L A, Oechsle, K, Schumacher, K, Kanfer, E, Bourhis, J H, Massard, C, Laszlo, D, Montoro, J, Flechon, A, Arpaci, F, Secondino, S, Wuchter, P, Dreger, P, Crysandt, M, Worel, N, Kruger, W, Ringhoffer, M, Unal, A, Nagler, A, Campos, A, Wahlin, A, Michieli, M, Sucak, G, Donnini, I, Schots, R, Ifrah, N, Badoglio, M, Martino, M, Raggi, D, Giannatempo, P, Rosti, G, Pedrazzoli, P, and Lanza, F

Published
2016
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14. OP021 Autologous haematopoietic stem cell transplantation for Crohn’s disease: a retrospective study from the European Society for Blood & Marrow Transplantation (EBMT) Autoimmune Diseases Working Party

Author

Brierley, C, Castilla-Llorente, C, Labopin, M, Badoglio, M, Rovira, M, Ricart, E, Dierickx, D, Vermeire, S, Hasselblatt, P, Finke, J, Onida, F, Cassinotti, A, Satsangi, J, Kazmi, M, López-Sanromán, A, Farge, D, Travis, S, Hawkey, C, and Snowden, J

Published
2018
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15. 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, F. R., Snowden, J. A., Corbacioglu, S., Greco, R., Alexander, T., Snowden, J., Badoglio, M., Labopin, M., Abinun, M., Apte, S., Arnold, R., Domenech, A., Brierley, C., Burman, J., Castilla-Llorente, C., Cooper, N., Daghia, G., Daikeler, T., del Papa, N., de Vries-Bouwstra, J., Farge, D., Finke, J., Hagglund, H., Hawkey, C., Henes, J., Hiepe, F., Jessop, H., Kiely, D., Kazmi, M., Kirgizov, K., Kramer, E., Mancardi, G., Marjanovic, Z., Martin, R., Martin, T., Ma, D., Moore, J., Miller, P., Muraro, P., Oliveira, M. -C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Ricart, E., Rovira, M., Saccardi, R., Saif, M., Sakellari, I., Sharrack, B., Snarski, E., Scherer, H. U., Sossa, C., Withers, B., Wulffraat, N., Zaccara, E., Amrolia, P., Ansari, M., Balduzzi, A., Dalassier, A., Dalle, J. -H., Diaz, C. H., Feuchtinger, T., Locatelli, Franco, Lucchini, G., Galimard, J. -E., Vincent, M. G., Handgretinger, R., Kleinschmidt, K., Lawitschka, A., Martinez, A. P., Peters, C., Rocha, V., Ruggeri, A., Sedlacek, P., Svec, P., Toporski, J., Yesilipek, A., Locatelli F. (ORCID:0000-0002-7976-3654), Achini-Gutzwiller, F. R., Snowden, J. A., Corbacioglu, S., Greco, R., Alexander, T., Snowden, J., Badoglio, M., Labopin, M., Abinun, M., Apte, S., Arnold, R., Domenech, A., Brierley, C., Burman, J., Castilla-Llorente, C., Cooper, N., Daghia, G., Daikeler, T., del Papa, N., de Vries-Bouwstra, J., Farge, D., Finke, J., Hagglund, H., Hawkey, C., Henes, J., Hiepe, F., Jessop, H., Kiely, D., Kazmi, M., Kirgizov, K., Kramer, E., Mancardi, G., Marjanovic, Z., Martin, R., Martin, T., Ma, D., Moore, J., Miller, P., Muraro, P., Oliveira, M. -C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Ricart, E., Rovira, M., Saccardi, R., Saif, M., Sakellari, I., Sharrack, B., Snarski, E., Scherer, H. U., Sossa, C., Withers, B., Wulffraat, N., Zaccara, E., Amrolia, P., Ansari, M., Balduzzi, A., Dalassier, A., Dalle, J. -H., Diaz, C. H., Feuchtinger, T., Locatelli, Franco, Lucchini, G., Galimard, J. -E., Vincent, M. G., Handgretinger, R., Kleinschmidt, K., Lawitschka, A., Martinez, A. P., Peters, C., Rocha, V., Ruggeri, A., Sedlacek, P., Svec, P., Toporski, J., Yesilipek, A., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

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.

Published
2022

16. No improvement of survival with reduced- versus high-intensity conditioning for allogeneic stem cell transplants in Ewing tumor patients

Author

Thiel, U., Wawer, A., Wolf, P., Badoglio, M., Santucci, A., Klingebiel, T., Basu, O., Borkhardt, A., Laws, H.-J., Kodera, Y., Yoshimi, A., Peters, C., Ladenstein, R., Pession, A., Prete, A., Urban, E.-C., Schwinger, W., Bordigoni, P., Salmon, A., Diaz, M.A., Afanasyev, B., Lisukov, I., Morozova, E., Toren, A., Bielorai, B., Korsakas, J., Fagioli, F., Caselli, D., Ehninger, G., Gruhn, B., Dirksen, U., Abdel-Rahman, F., Aglietta, M., Mastrodicasa, E., Torrent, M., Corradini, P., Demeocq, F., Dini, G., Dreger, P., Eyrich, M., Gozdzik, J., Guilhot, F., Holler, E., Koscielniak, E., Messina, C., Nachbaur, D., Sabbatini, R., Oldani, E., Ottinger, H., Ozsahin, H., Schots, R., Siena, S., Stein, J., Sufliarska, S., Unal, A., Ussowicz, M., Schneider, P., Woessmann, W., Jürgens, H., Bregni, M., and Burdach, S.

Published
2011
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19. Hematopoietic stem cell transplantation for autoimmune diseases in the time of COVID-19: EBMT guidelines and recommendations

Author

Greco, R., Alexander, T., Burman, J., Papa, N. del, Vries-Bouwstra, J. de, Farge, D., Henes, J., Kazmi, M., Kirgizov, K., Muraro, P.A., Ricart, E., Rovira, M., Saccardi, R., Sharrack, B., Snarski, E., Withers, B., Jessop, H., Boglione, C., Kramer, E., Badoglio, M., Labopin, M., Orchard, K., Corbacioglu, S., Ljungman, P., Mikulska, M., Camara, R. de la, Snowden, J.A., European Soc Blood Marrow Transp, Autoimmune Dis Working Party ADWP, Infect Dis Working Party IDWP, Pediat Working Party PWP, Joint Accreditation Comm Int Soc C, EBMT Nurses Grp, and Patient Advocacy Comm

Subjects
Feature, Adult, medicine.medical_specialty, Coronavirus disease 2019 (COVID-19), medicine.medical_treatment, Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), Best practice, MEDLINE, Hematopoietic stem cell transplantation, Inflammatory bowel disease, Autoimmune Diseases, 03 medical and health sciences, Rheumatic diseases, 0302 clinical medicine, Pandemic, medicine, Humans, Hematologi, Child, Intensive care medicine, Pandemics, 030203 arthritis & rheumatology, Transplantation, SARS-CoV-2, business.industry, Public health, Hematopoietic Stem Cell Transplantation, COVID-19, Hematology, Infectious diseases, Immunotherapy, business, Neurological disorders, 030217 neurology & neurosurgery
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

20. ALLOGENEIC STEM CELL TRANSPLANTATION FOR PATIENTS WITH ADVANCED RHABDOMYOSARCOMA: A RETROSPECTIVE ASSESSMENT: PH-AB118

Author

Thiel, U., Koscielniak, E., Blaeschke, F., Grunewald, T. G. P., Badoglio, M., Diaz, Angel M., Paillard, C., Prete, A., Ussowicz, M., Lang, P., Fagioli, F., Lutz, P., Ehninger, G., Schneider, P., Santucci, A., Bader, P., Gruhn, B., Faraci, M., Antunovic, P., Styczynski, J., Krüger, W. H., Castagna, L., Rohrlich, P., Ouachee, M., Salmon, A., Peters, C., Bregni, M., and Burdach, S.

Published
2014

22. AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION IN NEUROMYELITIS OPTICA: A RETROSPECTIVE STUDY OF THE EBMT AUTOIMMUNE DISEASES WORKING PARTY IN COLLABORATION WITH THE UNIVERSITY OF SAO PAOLO RIBEIRÃO PRETO BRAZIL: PH-P308

Author

Greco, R., Bondanza, A., Oliveira, M. C., Badoglio, M., Burman, J., Piehl, F., Hagglund, H., Krasulova, E., Simoes, Pinto B., Carlson, K., Pohlreich, D., Labopin, M., Saccardi, R., Comi, G., Mancardi, G. L., Bacigalupo, A., Ciceri, F., and Farge, D.

Published
2014

25. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party

Author

Henes, J., Oliveira, M.C., Labopin, M., Badoglio, M., Scherer, H.U., Papa, N. del, Daikeler, T., Schmalzing, M., Schroers, R., Martin, T., Pugnet, G., Simoes, B., Michonneau, D., Marijt, E.W.A., Lioure, B., Bay, J.O., Snowden, J.A., Rovira, M., Huynh, A., Onida, F., Kanz, L., Marjanovic, Z., Farge, D., NISSC1 Members, University of São Paulo (USP), CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre International des greffes [CHU Saint-Antoine] (EBMT), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), Leiden University Medical Center (LUMC), University of Basel (Unibas), CHU Strasbourg, Hôpital Purpan [Toulouse], CHU Toulouse [Toulouse], Hopital Saint-Louis [AP-HP] (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), CHU Clermont-Ferrand, Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-CHU Toulouse [Toulouse]-Institut National de la Santé et de la Recherche Médicale (INSERM), and Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Subjects
Adult, medicine.medical_specialty, Transplantation Conditioning, Cyclophosphamide, [SDV]Life Sciences [q-bio], medicine.medical_treatment, Aucun, Hematopoietic stem cell transplantation, Transplantation, Autologous, MESH: Scleroderma, Systemic, Article, Autoimmune Diseases, 03 medical and health sciences, 0302 clinical medicine, Autologous stem-cell transplantation, Bone Marrow, MESH: Autoimmune Diseases, Internal medicine, Clinical endpoint, Humans, Medicine, Prospective Studies, Progression-free survival, MESH: Hematopoietic Stem Cell Transplantation, MESH: Transplantation Conditioning, Aged, MESH: Aged, 030203 arthritis & rheumatology, MESH: Humans, Scleroderma, Systemic, business.industry, Hazard ratio, Hematopoietic Stem Cell Transplantation, MESH: Cyclophosphamide, MESH: Adult, Hematology, TRANSPLANTE AUTÓLOGO, MESH: Transplantation, Autologous, MESH: Prospective Studies, 3. Good health, Transplantation, MESH: Scleroderma, Diffuse, 030220 oncology & carcinogenesis, Scleroderma, Diffuse, MESH: Bone Marrow, Stem cell, business, medicine.drug
Abstract

Three randomized controlled trials in early severe systemic sclerosis demonstrated that autologous hematopoietic stem cell transplantation was superior to standard cyclophosphamide therapy. This European Society for Blood and Marrow Transplantation multicenter, prospective, non-interventional study was designed to further decipher efficacy and safety of this procedure for severe systemic sclerosis patients in real-life practice and to search for prognostic factors. All consecutive adult patients with systemic sclerosis undergoing a first autologous hematopoietic stem cell transplant between December 2012 and February 2016 were prospectively included in the study. The primary endpoint was progression-free survival. Secondary endpoints were overall survival, non-relapse mortality, response and incidence of progression. Eighty patients with systemic sclerosis were included. The median duration of the follow-up was 24 (range, 6-57) months after stem cell transplantation using cyclophosphamide plus antithymocyte globulin conditioning for all, with CD34+ selection in 35 patients. At 2 years, the progression- free survival rate was 81.8%, the overall survival rate was 90%, the response rate was 88.7% and the incidence of progression was 11.9%. The 100-day non-relapse mortality rate was 6.25% (n=5) with four deaths from cardiac events, including three due to cyclophosphamide toxicity. Modified Rodnan skin score and forced vital capacity improved with time (P24 and older age at transplantation were associated with lower progression-free survival (hazard ratios 3.32 and 1.77, respectively). CD34+-cell selection was associated with better response (hazard ratio 0.46). This study confirms the efficacy of autologous stem cell transplantation, using nonmyeloablative conditioning, in real-life practice for severe systemic sclerosis. Careful cardio-pulmonary assessment to identify organ involvement at the time of the patient’s referral, reduced cyclophosphamide doses and CD34+-cell selection may improve outcomes. The study was registered at ClinicalTrials.gov: NCT02516124.

Published
2021
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29. Diagnosis of Lupus, age at HSCT and CD34+ graft selection are the main risk factors for secondary autoimmune diseases occurring after HSCT for autoimmune diseases: a retrospective study of the EBMT Autoimmune Disease Working Party: O428

Author

Daikeler, T., Labopin, M., Di Gioia, M., Abinun, M., Alexander, T., Miniati, I., Gualandi, F., Fassas, A., Martin, T., Schwarze, C.-P., Wulffraat, N., Buch, M., Sampol, A., Carreras, E., Dubois, B., Gruhn, B., Guengoer, T., Pohlreich, D., Schuerwegh, A., Snarski, E., Snowden, J., Veys, P., Fasth, A., Lenhoff, S., Messina, C., Voswinkel, J., Badoglio, M., Henes, J., Launay, D., Tyndall, A., Gluckman, E., and Farge-Bancel, D.

Published
2011

32. THU0312 AUTOLOGOUS NON-MYELOABLATIVE HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY TAKAYASU ARTERITIS: A RETROSPECTIVE MULTICENTRE CASE-SERIES FROM THE AUTOIMMUNE DISEASES WORKING PARTY (ADWP) OF THE EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION (EBMT)

Author

Laurent, C., primary, Marjanovic, Z., additional, Henes, J., additional, Farge, D., additional, Badoglio, M., additional, Snowden, J., additional, Fain, O., additional, Alexander, T., additional, Oliveira, M. C., additional, and Mekinian, A., additional

Published
2020
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33. Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases : updated guidelines and recommendations from the EBMT autoimmune diseases working party (ADWP) and the joint accreditation committee of EBMT and ISCT (JACIE)

Author

Sharrack, B., Saccardi, R., Alexander, T., Badoglio, M., Burman, J., Farge, D., Greco, R., Jessop, H., Kazmi, M., Kirgizov, K., Labopin, M., Mancardi, G., Martin, R., Moore, J., Muraro, P.A., Rovira, M., Sormani, M.P., and Snowden, J.A.

Subjects
surgical procedures, operative
Abstract

These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

Published
2020

34. General information for patients and carers considering haematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (ADs) : A position statement from the EBMT Autoimmune Diseases Working Party (ADWP), the EBMT Nurses Group, the EBMT Patient, Family and Donor Committee and the Joint Accreditation Committee of ISCT and EBMT (JACIE)

Author

Jessop, H., Farge, D., Saccardi, R., Alexander, T., Rovira Tarrats, Montserrat, Sharrack, B., Greco, R., Wulffraat, N., Moore, J., Kazmi, M., Badoglio, M., Adams, G., Verhoeven, B., Murray, J., Snowden, J.A., Universitat Autònoma de Barcelona, Service de Médecine Interne [Saint-Louis], Université Paris Diderot - Paris 7 (UPD7)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Recherche clinique appliquée à l'hématologie ((EA_3518)), Université Paris Diderot - Paris 7 (UPD7), McGill University = Université McGill [Montréal, Canada], Charité - UniversitätsMedizin = Charité - University Hospital [Berlin], University of Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Universitat de Barcelona (UB), University of Sheffield [Sheffield], San Raffaele Scientific Institute, Vita-Salute San Raffaele University and Center for Translational Genomics and Bioinformatics, St. Vincent's Hospital, Sydney, Guy's Hospital [London], Laboratoire d'Hématologie et d'Immunologie [CHU Saint-Antoine], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), CEREST-TC [CHU Saint-Antoine], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre International des greffes [CHU Saint-Antoine] (EBMT), Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Université Paris Diderot - Paris 7 (UPD7)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (APHP), McGill University, Charité - Universitätsmedizin Berlin / Charite - University Medicine Berlin, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-CHU Saint-Antoine [APHP], and Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Sorbonne Université (SU)-CHU Saint-Antoine [APHP]

Subjects
medicine.medical_specialty, Autoimmune diseases, MEDLINE, Disease, Stem cells, Hospitals, Special, Transplantation, Autologous, Accreditation, 03 medical and health sciences, 0302 clinical medicine, Risk Factors, Medicine, Humans, Intensive care medicine, 030203 arthritis & rheumatology, Autoimmune disease, Transplantation, business.industry, Standard treatment, Hematopoietic Stem Cell Transplantation, Hematology, medicine.disease, Tissue Donors, 3. Good health, Haematopoiesis, surgical procedures, operative, Caregivers, 030220 oncology & carcinogenesis, Perspective, [SDV.IMM]Life Sciences [q-bio]/Immunology, Stem cell, business
Abstract

International audience; Over the last 20 years, haematopoietic stem cell transplantation (HSCT) has been used to treat patients with severe autoimmune and inflammatory diseases whose response to standard treatment options has been limited, resulting in a poor long-term prognosis in terms of survival or disability. The vast majority of patients have received autologous HSCT where an increasing evidence-base supports its use in a wide range of autoimmune diseases, particularly relapsing remitting MS, systemic sclerosis and Crohn’s disease. Compared with standard treatments for autoimmune diseases, HSCT is associated with greater short-term risks, including a risk of treatment-related mortality and long-term complications. There is a need for a careful appraisal of potential benefits and risks by disease and transplant specialists working closely together with patients and carers to determine individual suitability for HSCT. HSCT should be conducted in accredited transplant centres with robust arrangements for long-term follow-up with both disease and transplant specialists. The aim of this open-access position statement is to provide plainly worded guidance for patients and non-specialist clinicians considering HSCT for an autoimmune disease, especially when treatment abroad is being considered. Recent technical publications in the field have been referenced to support the statement and provide more detail for clinicians advising patients.

Published
2019
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36. Autologous haematopoietic stem cell transplantation for Crohn's disease: a retrospective study from the European Society for Blood & Marrow Transplantation (EBMT) Autoimmune Diseases Working Party

Author

Brierley, C, Castilla-Llorente, C, Labopin, M, Badoglio, M, Rovira, M, Ricart, E, Dierickx, D, Vermeire, S, Hasselblatt, P, Finke, J, Onida, F, Cassinotti, A, Satsangi, J, Kazmi, M, Lopez-Sanroman, A, Farge, D, Travis, S, Hawkey, C, and Snowden, J

Abstract

Autologous haematopoietic stem cell transplantation (AHSCT) is an emerging therapeutic option for refractory Crohn’s disease (CD). The ASTIC trial of AHSCT failed to meet its stringent composite primary endpoint, but 1-year follow-up data demonstrate sustained efficacy. The real-world use and outcomes of AHSCT for CD are unknown. Using the EBMT registry, we evaluated long-term outcomes for patients (pts) undergoing AHSCT for CD in Europe.

Published
2018

38. Evaluating the clinical effectiveness of autologous haematopoietic stem cell transplantation versus disease-modifying therapy in multiple sclerosis using a matching-adjusted indirect comparison: an exploratory study from the Autoimmune Diseases Working Party (ADWP) of the European Society of Bone and Marrow Transplantation (EBMT)

Author

Tappenden, P., primary, Wang, Y., additional, Sharrack, B., additional, Burman, J., additional, Kazmi, M., additional, Saccardi, R., additional, Bermejo, I., additional, Harvey, R., additional, Badoglio, M., additional, Farge, D., additional, and Snowden, J. A., additional

Published
2019
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39. Management of growth failure and growth hormone deficiency after pediatric allogeneic HSCT: Endocrinologists are of importance for further guidelines and studies

Author

Lawitschka, A., primary, Schwarze, P., additional, Rovelli, A., additional, Badoglio, M., additional, Socie, G., additional, Tichelli, A., additional, Bauer, D., additional, Rovo, A., additional, Basak, G., additional, Schoemans, H., additional, Peters, C., additional, and Salooja, N., additional

Published
2019
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41. Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases

Author

Snowden, J.A., Badoglio, M., Labopin, M., Giebel, S., McGrath, E., Marjanovic, Z., Burman, J., Moore, J., Rovira, M., Wulffraat, N.M., Kazmi, M., Greco, R., Snarski, E., Kozak, T., Kirgizov, K., Alexander, T., Bader, P., Saccardi, R., Farge, D., European Society for Blood and Marrow Transplantation (EBMT) Aut, EBMT Paediatric Working Party (PWP), Joint Accreditation Committee of the International Society for C, and EBMT (JACIE)

Subjects
surgical procedures, operative
Abstract

Hematopoietic stem cell transplantation (HSCT) has evolved for >20 years as a specific treatment of patients with autoimmune disease (AD). Using European Society for Blood and Marrow Transplantation registry data, we summarized trends and identified factors influencing activity and outcomes in patients with AD undergoing first autologous HSCT (n = 1951; median age, 37 years [3-76]) and allogeneic HSCT (n = 105; median age, 12 years [

Published
2017

45. Does ex vivo CD34+ positive selection influence outcome after autologous hematopoietic stem cell transplantation in systemic sclerosis patients?

Author

Oliveira, M.C., Labopin, M., Henes, J., Moore, J., Papa, N.D., Cras, A., Sakellari, I., Schroers, R., Scherer, H.U., Cuneo, A., Kyrcz-Krzemien, S., Daikeler, T., Alexander, T., Finke, J., Badoglio, M., Simoes, B., Snowden, J.A., Farge, D., and Party, E.A.D.W.

Subjects
0301 basic medicine, Oncology, Adult, Male, medicine.medical_specialty, Adolescent, medicine.medical_treatment, Antigens, CD34, Hematopoietic stem cell transplantation, Disease-Free Survival, law.invention, NO, Scleroderma, 03 medical and health sciences, 0302 clinical medicine, Randomized controlled trial, law, Autografts, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Middle Aged, Scleroderma, Systemic, Survival Rate, Hematology, Transplantation, Internal medicine, medicine, Antigens, Survival rate, CÉLULAS-TRONCO, business.industry, Hazard ratio, Systemic, medicine.disease, Confidence interval, Clinical trial, 030104 developmental biology, Graft-versus-host disease, Immunology, CD34, business, 030215 immunology
Abstract

This EBMT Autoimmune Disease Working Party study aimed to evaluate the influence of CD34+ positive graft selection (CD34+) on the outcome of systemic sclerosis (SSc) patients after autologous hematopoietic stem cell transplantation (AHSCT). Clinical and laboratory data from 138 SSc patients at diagnosis, before and after AHSCT were retrospectively analyzed. CD34+ selection was performed in 47.1% (n=65) patients. By multivariate analysis adjusting for all factors differing between the two groups (without or with CD34+), there was no statistically significant difference in terms of overall survival (hazard ratio (HR): 0.98, 95% confidence interval (CI) 0.40-2.39, P=0.96), PFS (HR: 1.55, 95% CI 0.83-2.88, P=0.17) and incidence of relapse or progression (HR: 1.70, 95% CI 0.85-3.38, P=0.13). We demonstrate that CD34+ does not add benefit to the outcome of SSc patient treated with AHSCT. These findings should be further confirmed by prospective randomized trials.

Published
2015

46. Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis : a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Burman, Joachim, Kirgizov, K, Carlson, Kristina, Badoglio, M, Mancardi, G L, De Luca, G, Casanova, B, Ouyang, J, Bembeeva, R, Haas, J, Bader, P, Snowden, J, Farge, D, Burman, Joachim, Kirgizov, K, Carlson, Kristina, Badoglio, M, Mancardi, G L, De Luca, G, Casanova, B, Ouyang, J, Bembeeva, R, Haas, J, Bader, P, Snowden, J, and Farge, D

Abstract

Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.

Published
2017
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47. No improvement of survival with reduced- versus high-intensity conditioning for allogeneic stem cell transplants in Ewing tumor patients

Author

Thiel, U., Wawer, A., Wolf, P., Badoglio, M., Santucci, A., Klingebiel, T., Basu, O., Borkhardt, A., Laws, H.-J, Kodera, Y., Yoshimi, A., Peters, C., Ladenstein, R., Pession, A., Prete, A., Urban, E.-C, Schwinger, W., Bordigoni, P., Salmon, A., Diaz, M. A., Afanasyev, B., Lisukov, I., Morozova, E., Toren, A., Bielorai, B., Korsakas, J., Fagioli, F., Caselli, D., Ehninger, G., Gruhn, B., Dirksen, U., Abdel-Rahman, F., Aglietta, M., Mastrodicasa, E., Torrent, M., Corradini, P., Demeocq, F., Dini, G., Dreger, P., Eyrich, M., Gozdzik, J., Guilhot, F., Holler, E., Koscielniak, E., Messina, C., Nachbaur, D., Sabbatini, R., Oldani, E., Ottinger, H., Ozsahin, H., Schots, R., Siena, S., Stein, J., Sufliarska, S., Unal, A., Ussowicz, M., Schneider, P., Woessmann, W., Jürgens, H., Bregni, M., Burdach, S., Thiel, U., Wawer, A., Wolf, P., Badoglio, M., Santucci, A., Klingebiel, T., Basu, O., Borkhardt, A., Laws, H.-J, Kodera, Y., Yoshimi, A., Peters, C., Ladenstein, R., Pession, A., Prete, A., Urban, E.-C, Schwinger, W., Bordigoni, P., Salmon, A., Diaz, M. A., Afanasyev, B., Lisukov, I., Morozova, E., Toren, A., Bielorai, B., Korsakas, J., Fagioli, F., Caselli, D., Ehninger, G., Gruhn, B., Dirksen, U., Abdel-Rahman, F., Aglietta, M., Mastrodicasa, E., Torrent, M., Corradini, P., Demeocq, F., Dini, G., Dreger, P., Eyrich, M., Gozdzik, J., Guilhot, F., Holler, E., Koscielniak, E., Messina, C., Nachbaur, D., Sabbatini, R., Oldani, E., Ottinger, H., Ozsahin, H., Schots, R., Siena, S., Stein, J., Sufliarska, S., Unal, A., Ussowicz, M., Schneider, P., Woessmann, W., Jürgens, H., Bregni, M., and Burdach, S.

Abstract

Background: Outcomes of Ewing tumor (ET) patients treated with allogeneic stem cell transplantation (allo-SCT) were compared regarding the use of reduced-intensity conditioning (RIC) and high-intensity conditioning (HIC) regimens as well as human leukocyte antigen (HLA)-matched and HLA-mismatched grafts. Patients and methods: We retrospectively analyzed data of 87 ET patients from the European Group for Blood and Marrow Transplantation, Pediatric Registry for Stem Cell Transplantations, Asia Pacific Blood and Marrow Transplantation and MetaEICESS registries treated with allo-SCT. Fifty patients received RIC (group A) and 37 patients received HIC (group B). Twenty-four patients received HLA-mismatched grafts and 63 received HLA-matched grafts. Results: Median overall survival was 7.9 months [±1.24, 95% confidence interval (CI) 5.44-10.31] for group A and 4.4 months (±1.06, 95% CI 2.29-6.43) for group B patients (P = 1.3). Death of complications (DOC) occurred in 4 of 50 (0.08) and death of disease (DOD) in 33 of 50 (0.66) group A and in 16 of 37 (0.43) and 17 of 37 (0.46) group B patients, respectively. DOC incidence was decreased (P < 0.01) and DOD/relapse increased (P < 0.01) in group A compared with group B. HLA mismatch was not generally associated with graft-versus-Ewing tumor effect (GvETE). Conclusions: There was no improvement of survival with RIC compared with HIC due to increased DOD/relapse incidence after RIC despite less DOC incidence. This implicates general absence of a clinically relevant GvETE with current protocols

Published
2017

50. Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: a retrospective study of the EBMT Autoimmune Disease Working Party

Author

Daikeler, T., Labopin, M., Gioia, M. di, Abinun, M., Alexander, T., Miniati, I., Gualandi, F., Fassas, A., Martin, T., Schwarze, C.P., Wulffraat, N., Buch, M., Sampol, A., Carreras, E., Dubois, B., Gruhn, B., Gungor, T., Pohlreich, D., Schuerwegh, A., Snarski, E., Snowden, J., Veys, P., Fasth, A., Lenhoff, S., Messina, C., Voswinkel, J., Badoglio, M., Henes, J., Launay, D., Tyndall, A., Gluckman, E., Farge, D., EBMT Autoimmune Disease Working, Department of Rheumatology, University Hospital Basel [Basel], Service d'hématologie clinique et de thérapie cellulaire [CHU Saint-Antoine], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), Haematology Department, Careggi University Hospital, Newcastle General Hospital, Department of Rheumatology & Clinical Immunology, Charité - UniversitätsMedizin = Charité - University Hospital [Berlin], Department of Biomedicine, Università degli Studi di Firenze = University of Florence [Firenze] (UNIFI), Department of Haematology II, Ospedale San Martino, Neurology & Haematology, George Papanicolau Hospital, Immunologie et chimie thérapeutiques (ICT), Cancéropôle du Grand Est-Centre National de la Recherche Scientifique (CNRS), Paediatric Haematology & Endocrinology, University Children's Hospital, University Medical Center, VU University Medical Center [Amsterdam], Section of Musculoskeletal Disease, University of Leeds, Hospital Universitari Son Espases, Department of Hematology, Hospital Clinic Barcelona, Department of Neurology, University Hospitals Leuven [Leuven], Department of Pediatrics, Friedrich-Schiller-Universität = Friedrich Schiller University Jena [Jena, Germany], Division of Immunology/Hematology/BMT, Charles University Hospital, Leiden University Medical Center (LUMC), Department of Hematology & Oncology, Medical University of Warsaw - Poland, Department of Haematology & Department of Oncology, NHS & University of Sheffield, Great Ormond Street Hospital for Children [London] (GOSH), University of Gothenburg (GU), University Hospital Lund, Dipartimento di Pediatria, Cinica di Oncoematologia Pediatrica, CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre de Recherche Saint-Antoine (UMRS893), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM), Department of Hematology & Rheumatology, University Hospital Tübingen, Department of Internal Medicine, Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Clinical Research Unit, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Immunologie, dermatologie, oncologie, Oncodermatologie, immunologie et cellules souches cutanées (IDO (U976 / UMR_S 976)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Diderot - Paris 7 (UPD7), EULAR Grant & Freiwillige Akademische Gesellschaft Basel, Università degli Studi di Firenze = University of Florence (UniFI), Universiteit Leiden-Universiteit Leiden, University of Sheffield [Sheffield], and Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM)

Subjects
Male, stem-cell transplantation bone-marrow-transplantation hemolytic-anemia blood, medicine.medical_treatment, Hematopoietic stem cell transplantation, Biochemistry, Autoimmune thrombocytopenia, Antibodies, Monoclonal, Murine-Derived, 0302 clinical medicine, Prednisone, immune system diseases, Risk Factors, hemic and lymphatic diseases, Child, Hematopoietic Stem Cell Transplantation, [SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology, Hematology, Middle Aged, 3. Good health, Europe, surgical procedures, operative, Treatment Outcome, 030220 oncology & carcinogenesis, Child, Preschool, Rituximab, Female, Autoimmune hemolytic anemia, Immunosuppressive Agents, medicine.drug, Adult, medicine.medical_specialty, Adolescent, Immunology, Autoimmune Diseases, 03 medical and health sciences, Young Adult, Antiphospholipid syndrome, Internal medicine, medicine, Humans, Immunologic Factors, Cyclophosphamide, Glucocorticoids, Retrospective Studies, Autoimmune disease, Lupus erythematosus, business.industry, Infant, Cell Biology, [SDV.IMM.IMM]Life Sciences [q-bio]/Immunology/Immunotherapy, medicine.disease, Multivariate Analysis, business, 030215 immunology
Abstract

To specify the incidence and risk factors for secondary autoimmune diseases (ADs) after HSCT for a primary AD, we retrospectively analyzed AD patients treated by HSCT reported to EBMT from 1995 to 2009 with at least 1 secondary AD (cases) and those without (controls). After autologous HSCT, 29 of 347 patients developed at least 1 secondary AD within 21.9 (0.6-49) months and after allogeneic HSCT, 3 of 16 patients. The observed secondary ADs included: autoimmune hemolytic anemia (n = 3), acquired hemophilia (n = 3), autoimmune thrombocytopenia (n = 3), antiphospholipid syndrome (n = 2), thyroiditis (n = 12), blocking thyroid-stimulating hormone receptor antibody (n = 1), Graves disease (n = 2), myasthenia gravis (n = 1), rheumatoid arthritis (n = 2), sarcoidosis (n = 2), vasculitis (n = 1), psoriasis (n = 1), and psoriatic arthritis (n = 1). After autologous HSCT for primary AD, the cumulative incidence of secondary AD was 9.8% ± 2% at 5 years. Lupus erythematosus as primary AD, and antithymocyte globulin use plus CD34+ graft selection were important risk factors for secondary AD by multivariate analysis. With a median follow-up of 6.2 (0.54-11) years after autologous HSCT, 26 of 29 patients with secondary AD were alive, 2 died during their secondary AD (antiphospholipid syndrome, hemophilia), and 1 death was HSCT-related. This European multicenter study underlines the need for careful management and follow-up for secondary AD after HSCT.

Published
2011
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