9 results on '"Badoglio, M"'
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2. 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
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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
3. 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
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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
4. 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
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Tappenden, P., Wang, Y., Sharrack, B., Burman, Joachim, Kazmi, M., Saccardi, R., Bermejo, I., Harvey, R., Badoglio, M., Farge, D., Snowden, J. A., Tappenden, P., Wang, Y., Sharrack, B., Burman, Joachim, Kazmi, M., Saccardi, R., Bermejo, I., Harvey, R., Badoglio, M., Farge, D., and Snowden, J. A.
- Published
- 2020
- Full Text
- View/download PDF
5. 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
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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.
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- 2017
- Full Text
- View/download PDF
6. No improvement of survival with reduced- versus high-intensity conditioning for allogeneic stem cell transplants in Ewing tumor patients
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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
7. Onset and outcome of pregnancy after autologous haematopoietic SCT (AHSCT) for autoimmune diseases : a retrospective study of the EBMT autoimmune diseases working party (ADWP)
- Author
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Snarski, E, Snowden, J A, Oliveira, M C, Simoes, B, Badoglio, M, Carlson, K, Burman, Joachim, Moore, J, Rovira, M, Clark, R E, Saiz, A, Hadj-Khelifa, S, Tan, J, Crescimanno, A, Musso, M, Martin, T, Farge, D, Snarski, E, Snowden, J A, Oliveira, M C, Simoes, B, Badoglio, M, Carlson, K, Burman, Joachim, Moore, J, Rovira, M, Clark, R E, Saiz, A, Hadj-Khelifa, S, Tan, J, Crescimanno, A, Musso, M, Martin, T, and Farge, D
- Abstract
Autologous haematopoietic SCT (AHSCT) is increasingly used to control severe and refractory autoimmune diseases (AD). Many patients are women of reproductive age with a potential desire for children. We present a multicentre retrospective analysis of pregnancy and childbirth in patients who underwent AHSCT for AD. The databases of the European Blood and Marrow Transplantation and University of Sao Paulo, Ribeirão Preto, Brazil were searched for female patients aged 18-50 years who had received AHSCT for AD between 1994-2011. In 324 adult female patients, 22 pregnancies were reported in 15 patients between 1997-2011. Indications for AHSCT included multiple sclerosis (n=7), systemic sclerosis (n=5), rheumatoid arthritis (n=1), juvenile idiopathic arthritis (n=1) and Takayasu disease (n=1). Of the 22 reported pregnancies, 20 followed natural conception. 15 pregnancies (68%) resulted in healthy life births, whereas 7 (32%) failed. Exacerbations of AD occurred in two patients during second pregnancies. No maternal mortality was associated with pregnancy or postpartum. There were no reports of congenital, developmental or any other disease in the children. This retrospective analysis confirms the possibility of pregnancy and childbirth following AHSCT for severe AD. The outcome of pregnancy is generally good and most led to the birth of a healthy child.
- Published
- 2015
- Full Text
- View/download PDF
8. Allogeneic stem cell transplantation for patients with advanced rhabdomyosarcoma: a retrospective assessment
- Author
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Thiel, U, Koscielniak, E, Blaeschke, F, Grunewald, T G P., Badoglio, M, Diaz, M A., 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, Petar, Styczynski, J, Krueger, W H., Castagna, L, Rohrlich, P, Ouachee-Chardin, M, Salmon, A, Peters, C, Bregni, M, Burdach, S, Thiel, U, Koscielniak, E, Blaeschke, F, Grunewald, T G P., Badoglio, M, Diaz, M A., 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, Petar, Styczynski, J, Krueger, W H., Castagna, L, Rohrlich, P, Ouachee-Chardin, M, Salmon, A, Peters, C, Bregni, M, and Burdach, S
- Abstract
Background: Allogeneic haematopoietic stem cell transplantation (allo-SCT) may provide donor cytotoxic T cell-/NK cell-mediated disease control in patients with rhabdomyosarcoma (RMS). However, little is known about the prevalence of graft-vs-RMS effects and only a few case experiences have been reported. less thanbrgreater than less thanbrgreater thanMethods: We evaluated allo-SCT outcomes of 30 European Group for Blood and Marrow Transplantation (EBMT)-registered patients with advanced RMS regarding toxicity, progression-free survival (PFS) and overall survival (OS) after allo-SCT. Twenty patients were conditioned with reduced intensity and ten with high-dose chemotherapy. Twenty-three patients were transplanted with HLA-matched and seven with HLA-mismatched grafts. Three patients additionally received donor lymphocyte infusions (DLIs). Median follow-up was 9 months. less thanbrgreater than less thanbrgreater thanResults: Three-year OS was 20% (s. e.+/- 8%) with a median survival time of 12 months. Cumulative risk of progression was 67% (s. e.+/- 10%) and 11% (s. e.+/- 6%) for death of complications. Thirteen patients developed acute graft-vs-host disease (GvHD) and five developed chronic GvHD. Eighteen patients died of disease and four of complications. Eight patients survived in complete remission (CR) (median: 44 months). No patients with residual disease before allo-SCT were converted to CR. less thanbrgreater than less thanbrgreater thanConclusion: The use of allo-SCT in patients with advanced RMS is currently experimental. In a subset of patients, it may constitute a valuable approach for consolidating CR, but this needs to be validated in prospective trials., Funding Agencies|Wilhelm Sander-Stiftung|2006.109.1|Else Kroner-Fresenius-Stiftung|P31/08//A123/07|BMBF (TranSarNet FK)|01GM087001GM1104B|Deutsche Forschungsgemeinschaft (DFG)|GR3728/2-1|AmGen and Chugai and the Deutsche Kinderkrebsstiftung|DKS 2010.07
- Published
- 2013
- Full Text
- View/download PDF
9. No improvement of survival with reduced- versus high-intensity conditioning for allogeneic stem cell transplants in Ewing tumor patients
- Author
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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
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