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151. The european hematology association roadmap for european hematology research: A consensus document

Author

Engert, A, Balduini, C, Brand, A, Coiffier, B, Cordonnier, C, Döhner, H, De Wit, T, Eichinger, S, Fibbe, W, Green, T, De Haas, F, Iolascon, A, Jaffredo, T, Rodeghiero, F, Sall Es, G, Schuringa, J, André, M, Andre Schmutz, I, Bacigalupo, A, Bochud, P, Den Boer, M, Bonini, C, Camaschella, C, Cant, A, Cappellini, M, Cazzola, M, Celso, C, Dimopoulos, M, Douay, L, Dzierzak, E, Einsele, H, Ferreri, A, De Franceschi, L, Gaulard, P, Gottgens, B, Greinacher, A, Gresele, P, Gribben, J, De Haan, G, Hansen, J, Hochhaus, A, Kadir, R, Kaveri, S, Kouskoff, V, Kühne, T, Kyrle, P, Ljungman, P, Maschmeyer, G, Méndez Ferrer, S, Milsom, M, Mummery, C, Ossenkoppele, G, Pecci, A, Peyvandi, F, Philipsen, S, Reitsma, P, Ribera, J, Risitano, A, Rivella, S, Ruf, W, Schroeder, T, Scully, M, Socie, G, Staal, F, Stanworth, S, Stauder, R, Stilgenbauer, S, Tamary, H, Theilgaard Mönch, K, Thein, S, Tilly, H, Trneny, M, Vainchenker, W, Vannucchi, A, Viscoli, C, Vrielink, H, Zaaijer, H, Zanella, A, Zolla, L, Zwaginga, J, Martinez, P, Van Den Akker, E, Allard, S, Anagnou, N, Andolfo, I, Andrau, J, Angelucci, E, Anstee, D, Aurer, I, Avet Loiseau, H, Aydinok, Y, Bakchoul, T, Balduini, A, Barcellini, W, Baruch, D, Baruchel, A, Bayry, J, Bento, C, Van Den Berg, A, Bernardi, R, Bianchi, P, Bigas, A, Biondi, A, Bohonek, M, Bonnet, D, Borchmann, P, Borregaard, N, Brækkan, S, Van Den Brink, M, Brodin, E, Bullinger, L, Buske, C, Butzeck, B, Cammenga, J, Campo, E, Carbone, A, Cervantes, F, Cesaro, S, Charbord, P, Claas, F, Cohen, H, Conard, J, Coppo, P, Vives Corron, J, Da Costa, L, Davi, F, Delwel, R, Dianzani, I, Domanović, D, Donnelly, P, Drnovšek, T, Dreyling, M, Du, M, Dufour, C, Durand, C, Efremov, D, Eleftheriou, A, Elion, J, Emonts, M, Engelhardt, M, Ezine, S, Falkenburg, F, Favier, R, Federico, M, Fenaux, P, Fitzgibbon, J, Flygare, J, Foà, R, Forrester, L, Galacteros, F, Garagiola, I, Gardiner, C, Garraud, O, Van Geet, C, Geiger, H, Geissler, J, Germing, U, Ghevaert, C, Girelli, D, Godeau, B, Gökbuget, N, Goldschmidt, H, Goodeve, A, Graf, T, Graziadei, G, Griesshammer, M, Gruel, Y, Guilhot, F, Von Gunten, S, Gyssens, I, Halter, J, Harrison, C, Harteveld, C, Hellström Lindberg, E, Hermine, O, Higgs, D, Hillmen, P, Hirsch, H, Hoskin, P, Huls, G, Inati, A, Johnson, P, Kattamis, A, Kiefel, V, Kleanthous, M, Klump, H, Krause, D, Hovinga, J, Lacaud, G, Lacroix Desmazes, S, Landman Parker, J, Legouill, S, Lenz, G, Von Lilienfeld Toal, M, Von Lindern, M, Lopez Guillermo, A, Lopriore, E, Lozano, M, Macintyre, E, Makris, M, Mannhalter, C, Martens, J, Mathas, S, Matzdorff, A, Medvinsky, A, Menendez, P, Migliaccio, A, Miharada, K, Mikulska, M, Minard, V, Montalbán, C, De Montalembert, M, Montserrat, E, Morange, P, Mountford, J, Muckenthaler, M, Müller Tidow, C, Mumford, A, Nadel, B, Navarro, J, El Nemer, W, Noizat Pirenne, F, O’Mahony, B, Oldenburg, J, Olsson, M, Oostendorp, R, Palumbo, A, Passamonti, F, Patient, R, De Latour, R, Pflumio, F, Pierelli, L, Piga, A, Pollard, D, Raaijmakers, M, Radford, J, Rambach, R, Koneti Rao, A, Raslova, H, Rebulla, P, Rees, D, Ribrag, V, Rijneveld, A, Rinalducci, S, Robak, T, Roberts, I, Rodrigues, C, Rosendaal, F, Rosenwald, A, Rule, S, Russo, R, Saglio, G, Sanchez, M, Scharf, R, Schlenke, P, Semple, J, Sierra, J, So Osman, C, Soria, J, Stamatopoulos, K, Stegmayr, B, Stunnenberg, H, Swinkels, D, Barata, J, Taghon, T, Taher, A, Terpos, E, Thachil, J, Tissot, J, Touw, I, Toye, A, Trappe, R, Traverse Glehen, A, Unal, S, Vaulont, S, Viprakasit, V, Vitolo, U, Van Wijk, R, Wójtowicz, A, Zeerleder, S, Zieger, B, Zieger, B., ZANELLA, ALBERTO, BIONDI, ANDREA, Engert, A, Balduini, C, Brand, A, Coiffier, B, Cordonnier, C, Döhner, H, De Wit, T, Eichinger, S, Fibbe, W, Green, T, De Haas, F, Iolascon, A, Jaffredo, T, Rodeghiero, F, Sall Es, G, Schuringa, J, André, M, Andre Schmutz, I, Bacigalupo, A, Bochud, P, Den Boer, M, Bonini, C, Camaschella, C, Cant, A, Cappellini, M, Cazzola, M, Celso, C, Dimopoulos, M, Douay, L, Dzierzak, E, Einsele, H, Ferreri, A, De Franceschi, L, Gaulard, P, Gottgens, B, Greinacher, A, Gresele, P, Gribben, J, De Haan, G, Hansen, J, Hochhaus, A, Kadir, R, Kaveri, S, Kouskoff, V, Kühne, T, Kyrle, P, Ljungman, P, Maschmeyer, G, Méndez Ferrer, S, Milsom, M, Mummery, C, Ossenkoppele, G, Pecci, A, Peyvandi, F, Philipsen, S, Reitsma, P, Ribera, J, Risitano, A, Rivella, S, Ruf, W, Schroeder, T, Scully, M, Socie, G, Staal, F, Stanworth, S, Stauder, R, Stilgenbauer, S, Tamary, H, Theilgaard Mönch, K, Thein, S, Tilly, H, Trneny, M, Vainchenker, W, Vannucchi, A, Viscoli, C, Vrielink, H, Zaaijer, H, Zanella, A, Zolla, L, Zwaginga, J, Martinez, P, Van Den Akker, E, Allard, S, Anagnou, N, Andolfo, I, Andrau, J, Angelucci, E, Anstee, D, Aurer, I, Avet Loiseau, H, Aydinok, Y, Bakchoul, T, Balduini, A, Barcellini, W, Baruch, D, Baruchel, A, Bayry, J, Bento, C, Van Den Berg, A, Bernardi, R, Bianchi, P, Bigas, A, Biondi, A, Bohonek, M, Bonnet, D, Borchmann, P, Borregaard, N, Brækkan, S, Van Den Brink, M, Brodin, E, Bullinger, L, Buske, C, Butzeck, B, Cammenga, J, Campo, E, Carbone, A, Cervantes, F, Cesaro, S, Charbord, P, Claas, F, Cohen, H, Conard, J, Coppo, P, Vives Corron, J, Da Costa, L, Davi, F, Delwel, R, Dianzani, I, Domanović, D, Donnelly, P, Drnovšek, T, Dreyling, M, Du, M, Dufour, C, Durand, C, Efremov, D, Eleftheriou, A, Elion, J, Emonts, M, Engelhardt, M, Ezine, S, Falkenburg, F, Favier, R, Federico, M, Fenaux, P, Fitzgibbon, J, Flygare, J, Foà, R, Forrester, L, Galacteros, F, Garagiola, I, Gardiner, C, Garraud, O, Van Geet, C, Geiger, H, Geissler, J, Germing, U, Ghevaert, C, Girelli, D, Godeau, B, Gökbuget, N, Goldschmidt, H, Goodeve, A, Graf, T, Graziadei, G, Griesshammer, M, Gruel, Y, Guilhot, F, Von Gunten, S, Gyssens, I, Halter, J, Harrison, C, Harteveld, C, Hellström Lindberg, E, Hermine, O, Higgs, D, Hillmen, P, Hirsch, H, Hoskin, P, Huls, G, Inati, A, Johnson, P, Kattamis, A, Kiefel, V, Kleanthous, M, Klump, H, Krause, D, Hovinga, J, Lacaud, G, Lacroix Desmazes, S, Landman Parker, J, Legouill, S, Lenz, G, Von Lilienfeld Toal, M, Von Lindern, M, Lopez Guillermo, A, Lopriore, E, Lozano, M, Macintyre, E, Makris, M, Mannhalter, C, Martens, J, Mathas, S, Matzdorff, A, Medvinsky, A, Menendez, P, Migliaccio, A, Miharada, K, Mikulska, M, Minard, V, Montalbán, C, De Montalembert, M, Montserrat, E, Morange, P, Mountford, J, Muckenthaler, M, Müller Tidow, C, Mumford, A, Nadel, B, Navarro, J, El Nemer, W, Noizat Pirenne, F, O’Mahony, B, Oldenburg, J, Olsson, M, Oostendorp, R, Palumbo, A, Passamonti, F, Patient, R, De Latour, R, Pflumio, F, Pierelli, L, Piga, A, Pollard, D, Raaijmakers, M, Radford, J, Rambach, R, Koneti Rao, A, Raslova, H, Rebulla, P, Rees, D, Ribrag, V, Rijneveld, A, Rinalducci, S, Robak, T, Roberts, I, Rodrigues, C, Rosendaal, F, Rosenwald, A, Rule, S, Russo, R, Saglio, G, Sanchez, M, Scharf, R, Schlenke, P, Semple, J, Sierra, J, So Osman, C, Soria, J, Stamatopoulos, K, Stegmayr, B, Stunnenberg, H, Swinkels, D, Barata, J, Taghon, T, Taher, A, Terpos, E, Thachil, J, Tissot, J, Touw, I, Toye, A, Trappe, R, Traverse Glehen, A, Unal, S, Vaulont, S, Viprakasit, V, Vitolo, U, Van Wijk, R, Wójtowicz, A, Zeerleder, S, Zieger, B, Zieger, B., ZANELLA, ALBERTO, and BIONDI, ANDREA

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at ∈ European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better fu treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine ‘sections’ in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

Published
2016

153. Rabbit ATG for aplastic anaemia treatment: a backward step?

Author

European Blood, Marrow Transplant Group, Severe Aplastic Anaemia Working P.a.r.t.y. Dufour C, Bacigalupo A, Oneto R, Socie G, Tichelli A, Passweg JR, Schrezenmeier H, Locasciulli A, Aljurf M, Marsh J.C., RISITANO, ANTONIO MARIA, European, Blood, Marrow Transplant, Group, Dufour C, Severe Aplastic Anaemia Working P. a. r. t. y., Bacigalupo, A, Oneto, R, Socie, G, Tichelli, A, Passweg, Jr, Risitano, ANTONIO MARIA, Schrezenmeier, H, Locasciulli, A, Aljurf, M, and Marsh, J. C.

Subjects
business.industry, Anemia, Aplastic, General Medicine, Prognosis, Molecular biology, Risk Assessment, Sensitivity and Specificity, Severity of Illness Index, Survival Analysis, Treatment Outcome, Cyclosporine, Medicine, Animals, Humans, Rabbits, Rabbit ATG, business, Immunosuppressive Agents, Antilymphocyte Serum, Randomized Controlled Trials as Topic
Published
2011

154. Should irradiated blood products be given routinely to all patients with aplastic anaemia undergoing immunosuppressive therapy with antithymocyte globulin (ATG)? A survey from the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party

Author

Marsh J, Socie G, Tichelli A, Schrezenmeier H, Hochsmann B, Fuehrer M, Bekassy AN, Korthof ET, Locasciulli A, Ljungman P, Bacigalupo A, Camitta B, Young NS, Passweg J, European Group for Blood, Marrow Transplantation Severe Aplastic Anaemia Working P.a.r.t.y., RISITANO, ANTONIO MARIA, Marsh, J, Socie, G, Tichelli, A, Schrezenmeier, H, Hochsmann, B, Risitano, ANTONIO MARIA, Fuehrer, M, Bekassy, An, Korthof, Et, Locasciulli, A, Ljungman, P, Bacigalupo, A, Camitta, B, Young, N, Passweg, J, European Group for, Blood, and Marrow Transplantation Severe Aplastic Anaemia Working, P. a. r. t. y.

Published
2010

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

Author

Laar, J.M. van, Farge, D., Sont, J.K., Naraghi, K., Marjanovic, Z., Larghero, J., Schuerwegh, A.J., Marijt, E.W.A., Vonk, M.C., Schattenberg, A.V., Matucci-Cerinic, M., Voskuyl, A.E., Loosdrecht, A.A. van de, Daikeler, T., Kotter, I., Schmalzing, M., Martin, T., Lioure, B., Weiner, S.M., Kreuter, A., Deligny, C., Durand, J.M., Emery, P., Machold, K.R., Sarrot-Reynauld, F., Warnatz, K., Adoue, D.F.P., Constans, J., Tony, H.P., Papa, N. del, Fassas, A., Himsel, A., Launay, D., Monaco, A. lo, Philippe, P., Quere, I., Rich, E., Westhovens, R., Griffiths, B., Saccardi, R., Hoogen, F.H. van den, Fibbe, W.E., Socie, G., Gratwohl, A., Tyndall, A., EBMT EULAR Scleroderma Study Grp, Rheumatology, Hematology, and CCA - Innovative therapy

Subjects
medicine.medical_specialty, Cyclophosphamide, business.industry, Cancer development and immune defence Radboud Institute for Molecular Life Sciences [Radboudumc 2], medicine.medical_treatment, Hazard ratio, General Medicine, Hematopoietic stem cell transplantation, 3. Good health, Surgery, law.invention, Clinical trial, Transplantation, Autologous stem-cell transplantation, Randomized controlled trial, law, Internal medicine, Inflammatory diseases Radboud Institute for Health Sciences [Radboudumc 5], medicine, Clinical endpoint, business, medicine.drug
Abstract

Contains fulltext : 136804.pdf (Publisher’s version ) (Open Access) IMPORTANCE: High-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HSCT) have shown efficacy in systemic sclerosis in phase 1 and small phase 2 trials. OBJECTIVE: To compare efficacy and safety of HSCT vs 12 successive monthly intravenous pulses of cyclophosphamide. DESIGN, SETTING, AND PARTICIPANTS: The Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial, a phase 3, multicenter, randomized (1:1), open-label, parallel-group, clinical trial conducted in 10 countries at 29 centers with access to a European Group for Blood and Marrow Transplantation-registered transplant facility. From March 2001 to October 2009, 156 patients with early diffuse cutaneous systemic sclerosis were recruited and followed up until October 31, 2013. INTERVENTIONS: HSCT vs intravenous pulse cyclophosphamide. MAIN OUTCOMES AND MEASURES: The primary end point was event-free survival, defined as time from randomization until the occurrence of death or persistent major organ failure. RESULTS: A total of 156 patients were randomly assigned to receive HSCT (n = 79) or cyclophosphamide (n = 77). During a median follow-up of 5.8 years, 53 events occurred: 22 in the HSCT group (19 deaths and 3 irreversible organ failures) and 31 in the control group (23 deaths and 8 irreversible organ failures). During the first year, there were more events in the HSCT group (13 events [16.5%], including 8 treatment-related deaths) than in the control group (8 events [10.4%], with no treatment-related deaths). At 2 years, 14 events (17.7%) had occurred cumulatively in the HSCT group vs 14 events (18.2%) in the control group; at 4 years, 15 events (19%) had occurred cumulatively in the HSCT group vs 20 events (26%) in the control group. Time-varying hazard ratios (modeled with treatment x time interaction) for event-free survival were 0.35 (95% CI, 0.16-0.74) at 2 years and 0.34 (95% CI, 0.16-0.74) at 4 years. CONCLUSIONS AND RELEVANCE: Among patients with early diffuse cutaneous systemic sclerosis, HSCT was associated with increased treatment-related mortality in the first year after treatment. However, HCST conferred a significant long-term event-free survival benefit. TRIAL REGISTRATION: isrctn.org Identifier: ISRCTN54371254.

Published
2014

156. Impact of Postremission Consolidation Chemotherapy on Outcome After Reduced-Intensity Conditioning Allogeneic Stem Cell Transplantation for Patients With Acute Myeloid Leukemia in First Complete Remission

Author

Yeshurun, M, Labopin, M, Blaise, D, Cornelissen, Jan, Sengeloev, H, Vindelov, L, Kuball, J, Chevallier, P, Craddock, C, Socie, G, Bilger, K, Schouten, HC (Harry), Fegueux, N, Goker, H, Maertens, J, Bunjes, D, Arnold, R, Nagler, A, Mohty, M, RS: GROW - Oncology, RS: GROW - R3 - Innovative Cancer Diagnostics & Therapy, Hematology, and Cardiology

Subjects
SDG 3 - Good Health and Well-being, complete remission, allogeneic stem cell transplantation (alloSCT), acute myeloid leukemia, consolidation, reduced-intensity conditioning
Abstract

BACKGROUNDThe objective of the current study was to investigate the role of postremission consolidation chemotherapy before reduced-intensity conditioning (RIC) allogeneic stem cell transplantation (alloSCT) for patients with acute myeloid leukemia (AML) in first complete remission (CR1). METHODSOf the 789 consecutive patients with AML in CR1 who underwent RIC alloSCT from a human leukocyte antigen-matched sibling or matched unrelated donor peripheral stem cell grafts between 2001 and 2010, 591 patients received at least 1 cycle of consolidation chemotherapy and 198 patients did not receive any consolidation chemotherapy before alloSCT. To minimize inherent survival bias in favor of patients who underwent transplant long after achieving CR1, the study focused on 373 patients who underwent transplant within the median time frame between achievement of CR1 and alloSCT (3 months for patients who underwent alloSCT from matched siblings and 4 months for patients who underwent alloSCT from matched unrelated donors). In this subgroup, 151 patients did not receive any consolidation chemotherapy and 222 patients received 1 consolidation chemotherapy cycle. RESULTSWith a median follow-up of 36 months (range, 2 months-135 months), the 3-year cumulative recurrence incidence (RI) was not significantly different between the groups (36%4% for the group treated without consolidation chemotherapy vs 38%3% for patients who received consolidation chemotherapy; P=.89). In addition, leukemia-free survival was similar between the groups (45%+/- 4% and 47%+/- 3%, respectively; P=.41). Dose intensity of cytarabine given during consolidation chemotherapy appeared to have no influence on RI. On multivariate analysis, pretransplant consolidation (1 cycle vs 0 cycles) was found to have no significant impact on RI (hazards ratio, 1.29; 95% confidence interval, 0.84-1.97 [P=.24]) or leukemia-free survival (hazards ratio, 1.00; 95% confidence interval, 0.71-1.42 [P=.99]). CONCLUSIONSThe data from the current study suggest no apparent advantage for postremission consolidation chemotherapy before RIC alloSCT, provided a donor is readily available. Cancer 2014;120:855-863. (c) 2013 American Cancer Society. There is no apparent advantage for postremission consolidation chemotherapy before reduced-intensity conditioning allogeneic stem cell transplantation for patients with acute myeloid leukemia in first complete response. Provided a suitable donor is readily available, transplantation should be offered promptly at the time the first complete response is achieved, without undue delay.

Published
2014

157. Erratum: Impact of HLA mismatch direction on outcomes after umbilical cord blood transplantation for hematological malignant disorders: A retrospective Eurocord-EBMT analysis (Bone Marrow Transplantation (2014) 49 (864))

Author

Cunha, R., Loiseau, P., Ruggeri, A., Sanz, G., Michel, G., Iori, A. P., Socie, G., Arcese, W., Picardi, A., Dias De Heredia, C., Rio, B., Locatelli, F., O'Brien, T. A., Yakoub-Agha, I., Diaz, M. A., Milpied, N., Bittencourt, H., Souza, M. P., Aljurf, M., Charron, D., Boudjedir, K., Labopin, M., Gluckman, E., and Rocha, V.

Published
2014

159. Unrelated alternative donor transplantation for severe acquired aplastic anemia: a study from the French Society of Bone Marrow Transplantation and Cell Therapies and the EBMT Severe Aplastic Anemia Working Party

Author

Devillier, R., primary, Dalle, J.-H., additional, Kulasekararaj, A., additional, Daveni, M., additional, Clement, L., additional, Chybicka, A., additional, Vigouroux, S., additional, Chevallier, P., additional, Koh, M., additional, Bertrand, Y., additional, Michallet, M., additional, Zecca, M., additional, Yakoub-Agha, I., additional, Cahn, J.-Y., additional, Ljungman, P., additional, Bernard, M., additional, Loiseau, P., additional, Dubois, V., additional, Maury, S., additional, Socie, G., additional, Dufour, C., additional, and de Latour, R. P., additional

Published
2016
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160. Improved graft-versus-host disease-free, relapse-free survival associated with bone marrow as the stem cell source in adults

Author

Mehta, R. S., primary, de Latour, R. P., additional, DeFor, T. E., additional, Robin, M., additional, Lazaryan, A., additional, Xhaard, A., additional, Bejanyan, N., additional, de Fontbrune, F. S., additional, Arora, M., additional, Brunstein, C. G., additional, Blazar, B. R., additional, Weisdorf, D. J., additional, MacMillan, M. L., additional, Socie, G., additional, and Holtan, S. G., additional

Published
2016
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161. Allogeneic hematopoietic stem cell transplantation after reduced intensity conditioning regimen for elderly patients (60 years and older) with hematologic malignancies using unrelated donors: a retrospective study from the French society for stem cell transplantation (SFGM-TC)

Author

Cheikh, J. E., primary, Sfumato, P., additional, Sobh, M., additional, Fegueux, N., additional, Mohty, M., additional, Vigouroux, S., additional, Beguin, Y., additional, Yakoub-Agha, I., additional, Socie, G., additional, Cornillon, J., additional, Mercier, M., additional, Bay, J. O., additional, Blaise, D., additional, Michallet, M., additional, and de Latour, R. P., additional

Published
2016
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162. APRIL levels are associated with disease activity in human chronic graft-versus-host disease

Author

Chasset, F., primary, de Masson, A., additional, Le Buanec, H., additional, Xhaard, A., additional, de Fontbrune, F. S., additional, Robin, M., additional, Rybojad, M., additional, Parquet, N., additional, Brignier, A. C., additional, Coman, T., additional, Bengoufa, D., additional, Bergeron, A., additional, de Latour, R. P., additional, Bagot, M., additional, Bensussan, A., additional, Socie, G., additional, and Bouaziz, J.-D., additional

Published
2016
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163. Expanding transplant options to patients over 50 years. Improved outcome after reduced intensity conditioning mismatched-unrelated donor transplantation for patients with acute myeloid leukemia: a report from the Acute Leukemia Working Party of the EBMT

Author

Savani, B. N., primary, Labopin, M., additional, Kroger, N., additional, Finke, J., additional, Ehninger, G., additional, Niederwieser, D., additional, Schwerdtfeger, R., additional, Bunjes, D., additional, Glass, B., additional, Socie, G., additional, Ljungman, P., additional, Craddock, C., additional, Baron, F., additional, Ciceri, F., additional, Gorin, N. C., additional, Esteve, J., additional, Schmid, C., additional, Giebel, S., additional, Mohty, M., additional, and Nagler, A., additional

Published
2016
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164. Natural killer cell licensing after double cord blood transplantation is driven by the self-HLA class I molecules from the dominant cord blood

Author

Guillaume, N., primary, Loiseau, P., additional, Gagne, K., additional, Moins-Teissserenc, H., additional, Cayuela, J.-M., additional, Henry, G., additional, Robin, M., additional, de Latour, R. P., additional, Gluckman, E., additional, Socie, G., additional, Retiere, C., additional, Dulphy, N., additional, and Toubert, A., additional

Published
2016
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165. Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey

Author

Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M.M., Maas-Bauer, K., Metzelder, S.K., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., Fontbrune, F.S. de, Na, I.K., Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Lubbert, M., Wasch, R., Scheid, C., Stolzel, F., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graff, A., Velden, W.J. van der, Huls, G.A., Mielke, S., Grigoleit, G.U., Kuball, J., Flynn, R., Ihorst, G., Du, J., Blazar, B.R., Arnold, R., Kroger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., Bubnoff, N. von, Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M.M., Maas-Bauer, K., Metzelder, S.K., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., Fontbrune, F.S. de, Na, I.K., Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Lubbert, M., Wasch, R., Scheid, C., Stolzel, F., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graff, A., Velden, W.J. van der, Huls, G.A., Mielke, S., Grigoleit, G.U., Kuball, J., Flynn, R., Ihorst, G., Du, J., Blazar, B.R., Arnold, R., Kroger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., and Bubnoff, N. von

Abstract

Item does not contain fulltext, Despite major improvements in allogeneic hematopoietic cell transplantation over the past decades, corticosteroid-refractory (SR) acute (a) and chronic (c) graft-versus-host disease (GVHD) cause high mortality. Preclinical evidence indicates the potent anti-inflammatory properties of the JAK1/2 inhibitor ruxolitinib. In this retrospective survey, 19 stem cell transplant centers in Europe and the United States reported outcome data from 95 patients who had received ruxolitinib as salvage therapy for SR-GVHD. Patients were classified as having SR-aGVHD (n=54, all grades III or IV) or SR-cGVHD (n=41, all moderate or severe). The median number of previous GVHD-therapies was 3 for both SR-aGVHD (1-7) and SR-cGVHD (1-10). The overall response rate was 81.5% (44/54) in SR-aGVHD including 25 complete responses (46.3%), while for SR-cGVHD the ORR was 85.4% (35/41). Of those patients responding to ruxolitinib, the rate of GVHD-relapse was 6.8% (3/44) and 5.7% (2/35) for SR-aGVHD and SR-cGVHD, respectively. The 6-month-survival was 79% (67.3-90.7%, 95% confidence interval (CI)) and 97.4% (92.3-100%, 95% CI) for SR-aGVHD and SR-cGVHD, respectively. Cytopenia and cytomegalovirus-reactivation were observed during ruxolitinib treatment in both SR-aGVHD (30/54, 55.6% and 18/54, 33.3%) and SR-cGVHD (7/41, 17.1% and 6/41, 14.6%) patients. Ruxolitinib may constitute a promising new treatment option for SR-aGVHD and SR-cGVHD that should be validated in a prospective trial.

Published
2015

166. Paediatric reduced intensity conditioning : analysis of centre strategies on regimens and definitions by the EBMT Paediatric Diseases and Complications and Quality of Life WP

Author

Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., Peters, C., Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., and Peters, C.

Published
2015

167. High response rates in patients treated with ruxolitinib for corticosteroid-refractory Graft-versus-host disease: analysis including 13 Transplant Centers

Author

Zeiser, R., Lengerke, C., Spoerl, S., Maas-Bauer, K., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Sicre-de-Fontbrune, F., Na, I-K, Ajib, S., Verbeek, M., Nogai, H., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Illert, L., Bertz, H., Marks, R., Waesch, R., Hackanson, B., Scheid, C., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Finke, J., Peschel, C., Duyster, J., von Bubnoff, N., Zeiser, R., Lengerke, C., Spoerl, S., Maas-Bauer, K., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Sicre-de-Fontbrune, F., Na, I-K, Ajib, S., Verbeek, M., Nogai, H., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Illert, L., Bertz, H., Marks, R., Waesch, R., Hackanson, B., Scheid, C., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Finke, J., Peschel, C., Duyster, J., and von Bubnoff, N.

Published
2015

168. Ruxolitinib for corticosteroid-refractory graft-versus-host disease: analysis of 95 patients treated at multiple medical centers

Author

Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M., Maas-Bauer, K., Metzelder, S., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., de Fontbrune, Sicre F., Na, I. -K, Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Luebbert, M., Waesch, R., Scheid, C., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graeff, A., van der Velden, W., Huls, G., Grigoleit, G. U., Kuball, J., Blazar, B. R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., von Bubnoff, N., Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M., Maas-Bauer, K., Metzelder, S., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., de Fontbrune, Sicre F., Na, I. -K, Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Luebbert, M., Waesch, R., Scheid, C., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graeff, A., van der Velden, W., Huls, G., Grigoleit, G. U., Kuball, J., Blazar, B. R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., and von Bubnoff, N.

Published
2015

169. Predicting survival using clinical risk scores and non-HLA immunogenetics

Author

Balavarca, Y., Pearce, K., Norden, J., Collin, M., Jackson, G., Holler, E., Dressel, R., Kolb, H-J, Greinix, H., Socie, G., Toubert, A., Rocha, V., Gluckman, E., Hromadnikova, I., Sedlacek, P., Wolff, D., Holtick, U., Dickinson, A., Bickeboeller, H., Balavarca, Y., Pearce, K., Norden, J., Collin, M., Jackson, G., Holler, E., Dressel, R., Kolb, H-J, Greinix, H., Socie, G., Toubert, A., Rocha, V., Gluckman, E., Hromadnikova, I., Sedlacek, P., Wolff, D., Holtick, U., Dickinson, A., and Bickeboeller, H.

Abstract

Previous studies of non-histocompatibility leukocyte antigen (HLA) gene single-nucleotide polymorphisms (SNPs) on subgroups of patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT) revealed an association with transplant outcome. This study further evaluated the association of non-HLA polymorphisms with overall survival in a cohort of 762 HSCT patients using data on 26 polymorphisms in 16 non-HLA genes. When viewed in addition to an already established clinical risk score (EBMT-score), three polymorphisms: rs8177374 in the gene for MyD88-adapter-like (MAL; P = 0.026), rs9340799 in the oestrogen receptor gene (ESR; P = 0.003) and rs1800795 in interleukin-6 (IL-6; P = 0.007) were found to be associated with reduced overall survival, whereas the haplo-genotype (ACC/ACC) in IL-10 was protective (P = 0.02). The addition of these non-HLA polymorphisms in a Cox regression model alongside the EBMT-score improved discrimination between risk groups and increased the level of prediction compared with the EBMT-score alone (gain in prediction capability for EBMT-genetic-score 10.8%). Results also demonstrated how changes in clinical practice through time have altered the effects of non-HLA analysis. The study illustrates the significance of non-HLA genotyping prior to HSCT and the importance of further investigation into non-HLA gene polymorphisms in risk prediction.

Published
2015

170. Paediatric reduced intensity conditioning: analysis of centre strategies on regimens and definitions by the EBMT Paediatric Diseases and Complications and Quality of Life WP

Author

Child Health, Infection & Immunity, SCT onderzoek, Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., Peters, C., Child Health, Infection & Immunity, SCT onderzoek, Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., and Peters, C.

Published
2015

171. Tyrosine kinase inhibitors improve long-term outcome of allogeneic hematopoietic stem cell transplantation for adult patients with Philadelphia chromosome positive acute lymphoblastic leukemia

Author

Brissot, E, Labopin, M, Beckers, MM, Socie, G, Rambaldi, A, Volin, L, Finke, J, Lenhoff, S, Kroger, N, Ossenkoppele, GJ, Craddock, CF, Yakoub-Agha, I, Gurman, G, Russell, NH, Aljurf, M, Potter, MN, Nagler, A, Ottmann, O, Cornelissen, Jan, Esteve, J, Mohty, M, Brissot, E, Labopin, M, Beckers, MM, Socie, G, Rambaldi, A, Volin, L, Finke, J, Lenhoff, S, Kroger, N, Ossenkoppele, GJ, Craddock, CF, Yakoub-Agha, I, Gurman, G, Russell, NH, Aljurf, M, Potter, MN, Nagler, A, Ottmann, O, Cornelissen, Jan, Esteve, J, and Mohty, M

Abstract

This study aimed to determine the impact of tyrosine kinase inhibitors given pre- and post- allogeneic stem cell transplantation on long- term outcome of patients allografted for Philadelphia chromosome- positive acute lymphoblastic leukemia. This retrospective analysis from the EBMT Acute Leukemia Working Party included 473 de novo Philadelphia chromosome- positive acute lymphoblastic leukemia patients in first complete remission who underwent an allogeneic stem cell transplantation using a human leukocyte antigen- identical sibling or human leukocyte antigen- matched unrelated donor between 2000 and 2010. Three hundred and ninety patients received tyrosine kinase inhibitors before transplant, 329 at induction and 274 at consolidation. Kaplan- Meier estimates of leukemia- free survival, overall survival, cumulative incidences of relapse incidence, and non- relapse mortality at five years were 38%, 46%, 36% and 26%, respectively. In multivariate analysis, tyrosine- kinase inhibitors given before allogeneic stem cell transplantation was associated with a better overall survival ( HR= 0.68; P= 0.04) and was associated with lower relapse incidence ( HR= 0.5; P= 0.01). In the post- transplant period, multivariate analysis identified prophylactic tyrosine- kinase inhibitor administration to be a significant factor for improved leukemiafree survival ( HR= 0.44; P= 0.002) and overall survival ( HR= 0.42; P= 0.004), and a lower relapse incidence ( HR= 0.40; P= 0.01). Over the past decade, administration of tyrosine kinase inhibitors before allogeneic stem cell transplantation has significantly improved the long- term allogeneic stem cell transplantation outcome of adult Philadelphia chromosome- positive acute lymphoblastic leukemia. Prospective studies will be of great interest to further confirm the potential benefit of the prophylactic use of tyrosine kinase inhibitors in the post- transplant setting.

Published
2015

172. Lenalidomide as salvage treatment for multiple myeloma relapsing after allogeneic hematopoietic stem cell transplantation: a report from the SFGM-TC

Author

Coman , T., Bachy , E., Michallet , M., Socie , G., Uzunov , M., J.H. , Bourhis, Lapusan , S., Brebion , A., Vigouroux , S., Maury , S., Francois , S., Huynh , A., Lioure , B., Yakoub-Agha , I., Hermine , O., Milpied , M., Mohty , M., M.T. , Rubio, Cytokines, hématopoïèse et réponse immune (CHRI), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Slama, Catherine, Cytokines, hématopoïèse et réponse immune ( CHRI ), and Université Paris Descartes - Paris 5 ( UPD5 ) -Institut National de la Santé et de la Recherche Médicale ( INSERM ) -Centre National de la Recherche Scientifique ( CNRS )

Subjects
ComputingMilieux_MISCELLANEOUS
Abstract

International audience

Published
2013

173. Distinct factors determine the kinetics of disease relapse in adults transplanted for acute myeloid leukaemia.

Author

Craddock, C., Versluis, J., Labopin, M., Socie, G., Huynh, A., Deconinck, E., Volin, L., Milpied, N., Bourhis, J. H., Rambaldi, A., Chevallier, P., Blaise, D., Manz, M., Vellenga, E., Vekemans, M‐C., Maertens, J., Passweg, J., Vyas, P., Schmid, C., and Löwenberg, B.

Subjects
MYELOID leukemia, CANCER chemotherapy, DISEASE relapse, CAUSES of death, CELLULAR therapy
Abstract

Background: Disease recurrence remains the major cause of death in adults with acute myeloid leukaemia (AML) treated using either intensive chemotherapy (IC) or allogenic stem cell transplantation (allo-SCT).Aims: The timely delivery of maintenance drug or cellular therapies represent emerging strategies with the potential to reduce relapse after both treatment modalities, but whilst the determinants of overall relapse risk have been extensively characterized the factors determining the timing of disease recurrence have not been characterized.Materials and Methods: We have therefore examined, using a series of sequential landmark analyses, relapse kinetics in a cohort of 2028 patients who received an allo-SCT for AML in CR1 and separately 570 patients treated with IC alone.Results: In the first 3 months after allo-SCT, the factors associated with an increased risk of relapse included the presence of the FLT3-ITD (P < 0.001), patient age (P = 0.012), time interval from CR1 to transplant (P < 0.001) and donor type (P = 0.03). Relapse from 3 to 6 months was associated with a higher white cell count at diagnosis (P = 0.001), adverse-risk cytogenetics (P < 0.001), presence of FLT3-ITD mutation (P < 0.001) and time interval to achieve first complete remission (P = 0.013). Later relapse was associated with adverse cytogenetics, mutated NPM1, absence of chronic graft-versus-host disease (GVHD) and the use of in vivo T-cell depletion. In patients treated with IC alone, the factors associated with relapse in the first 3 months were adverse-risk cytogenetics (P < 0.001) and FLT3-ITD status (P = 0.001). The factors predicting later relapse were the time interval from diagnosis to CR1 (P = 0.22) and time interval from CR1 to IC (P = 0.012).Discussion and Conclusion: Taken together, these data provide novel insights into the biology of disease recurrence after both allo-SCT and IC and have the potential to inform the design of novel maintenance strategies in both clinical settings. [ABSTRACT FROM AUTHOR]

Published
2018
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174. The European LeukemiaNet AML Working Party consensus statement on allogeneic HSCT for patients with AML in remission: an integrated-risk adapted approach

Author

Cornelissen, JJ, Gratwohl, A, Schlenk, RF, Sierra, J, Bornhauser, M, Juliusson, G, Racil, Z, Rowe, JM, Russell, N, Mohty, M, Lowenberg, B, Socie, G, Niederwieser, D, and Ossenkoppele, GJ

Abstract

Allogeneic haematopoietic stem-cell transplantation (HSCT) is frequently applied as part of the treatment in patients with acute myeloid leukaemia (AML) in their first or subsequent remission. Allogeneic HSCT reduces relapse, but nonrelapse mortality and morbidity might counterbalance this beneficial effect. Here, we review recent studies reporting new disease-specific prognostic markers, in addition to allogeneic-HSCT-related risk factors, which can be assessed at specific time points during treatment. We propose risk assessment as a dynamic process during treatment, incorporating both disease-related and transplant-related factors for the decision to proceed either to allogeneic HSCT or to apply a nontransplant strategy. We suggest that allogeneic HSCT might be favoured if the projected disease-free survival is expected to improve by at least 10% based on an individual's risk assessment. The approach requires initial disease risk assessment, identifying a sibling or unrelated donor soon after diagnosis and the incorporation of time-dependent risk factors, all within the context of an integrated therapeutic management approach.

Published
2012

175. Prognostic Factors Affecting Outcome after Allogeneic Transplantation for Hematological Malignancies from Unrelated Donors: Results from a Randomized Trial

Author

Finke, J, Schmoor, C, Bethge, WA, Ottinger, HD, Stelljes, M, Zander, AR, Volin, L, Heim, DA, Schwerdtfeger, R, Kolbe, K, Mayer, J, Maertens, JA, Linkesch, W, Holler, E, Koza, V, Bornhauser, M, Einsele, H, Bertz, H, Grishina, O, Socie, G, Sierra J., and ATG-Fresenius Trial Grp

Subjects
Graft-versus-host-disease prophylaxis, Allogeneic hematopoietic stem-cell transplantation, HLA-matching
Abstract

Several prognostic factors for the outcome after allogeneic hematopoietic stem-cell transplant (HSCT) from matched unrelated donors have been postulated from registry data; however, data from randomized trials are lacking. We present analyses on the effects of patient-related, donor-related, and treatment-related prognostic factors on acute GVHD (aGVHD), chronic GVHD (cGVHD), relapse, nonrelapse mortality (NRM), disease-free survival (DFS), and overall survival (OS) in a randomized, multicenter, open-label, phase III trial comparing standard graft-versus-host-disease (GVHD) prophylaxis with and without pretransplantation ATG-Fresenius (ATG-F) in 201 adult patients receiving myeloablative conditioning before HSCT from HLA-A, HLA-B antigen, HLA-DRBI, HLA-DQBI allele matched unrelated donors. High-resolution testing (allele) of HLA-A, HLA-B, and HLA-C were obtained after study closure, and the impact of an HLA 10/10 4-digit mismatch on outcome and on the treatment effect of ATG-F versus control investigated. Advanced disease was a negative factor for relapse, DFS, and OS. Donor age >= 40 adversely affected the risk of aGVHD III-IV, extensive cGVHD, and OS. Younger donors are to be preferred in unrelated donor transplantation. Advanced disease patients need special precautions to improve outcome. The degree of mismatch had no major influence on the positive effect of ATG-F on the reduction of aGVHD and cGVHD. Biol Blood Marrow Transplant 18: 1716-1726 (2012) (C) 2012 American Society for Blood and Marrow Transplantation

Published
2012

176. Class I and II HLA Typing After a 10 Gy-4 I-iour Therayeutic Total Body Irradiation

Author

E Grimaud, T. Girinski, Socie G, Jean-Marc Cosset, Follezou Jy, Raffoux C, B Dubray, Chaillet Mp, and Briot E

Subjects
Adult, Male, Time Factors, Adolescent, Epidemiology, Health, Toxicology and Mutagenesis, medicine.medical_treatment, Human leukocyte antigen, Antigen, Allogeneic bone marrow graft, Neoplasms, medicine, Humans, Radiology, Nuclear Medicine and imaging, Typing, Child, Radiation Injuries, Bone Marrow Transplantation, business.industry, Histocompatibility Testing, Histocompatibility Antigens Class I, Histocompatibility Antigens Class II, Middle Aged, Total body irradiation, Radiation therapy, medicine.anatomical_structure, Accidents, Child, Preschool, Immunology, Female, Bone marrow, business, Whole-Body Irradiation, Class II Antigens
Abstract

Class I and II HLA typing was investigated before and at various intervals after a 10 Gy total body irradiation delivered over 4 h, prior to allogeneic bone marrow graft for various hematological malignancies, in 14 patients. A reliable class I HLA typing appeared to be possible in almost all cases 6-8 hours after the start of irradiation but was only possible in 5 patients after 24 h. Preliminary results with class II antigens might suggest a more marked "fragility" of this antigen class after irradiation. These results encourage the drawing of blood samples for HLA grouping as soon as possible after accidental whole-body irradiation.

Published
1993

177. Prospective Study of the Clinical Symptoms of Therapeutic Whole Body Irradiation

Author

E Grimaud, Jose-Luis Pico, Jean-Marc Cosset, Socie G, Chaillet Mp, C Alapetite, Theodore Girinsky, and B Dubray

Subjects
Adult, Male, medicine.medical_specialty, Adolescent, Consciousness, Fever, Lymphoma, Cyclophosphamide, Vomiting, Epidemiology, Nausea, Health, Toxicology and Mutagenesis, Xerostomia, Ondansetron, Institut Gustave Roussy, Tachycardia, medicine, Humans, Radiology, Nuclear Medicine and imaging, Prospective Studies, Child, Prospective cohort study, Leukemia, business.industry, Headache, Middle Aged, Total body irradiation, Surgery, Blood pressure, Child, Preschool, Anesthesia, Hypertension, Female, France, medicine.symptom, business, Parotitis, Whole-Body Irradiation, medicine.drug
Abstract

Thirty-one selected patients with various haematological malignancies who received a 10 Gy-4 h total body irradiation (TBI) at the Institut Gustave Roussy 24 h before high dose cyclophosphamide for bone marrow transplantation, were prospectively evaluated for gastrointestinal symptoms, body temperature, consciousness, headache, xerostomia, parotiditis, ocular symptoms, blood pressure, and respiratory and cutaneous signs for 24 h. In spite of prophylactic administration of various anti-emetic agents, 90% of the patients experienced nausea and 80% experienced vomiting. An almost constant body temperature peak--up to 40.8 degrees C--was registered 6 h after the start of irradiation. No drowsiness was reported since the introduction of the new anti-emetic agent Ondansetron. Nearly half the patients (42%) complained of headache. The proportion of patients experiencing early (during TBI) xerostomia was 61%. 74% of patients complained of parotiditis in the first 24 h. Although this low dose rate whole body irradiation is not likely to be exactly replicated in many accidental human exposures, the incidence rate and the time-course of the observed prodromal phase symptoms may prove helpful for early triage in the case of accidental irradiation.

Published
1993

178. Monosomal karyotype as an adverse prognostic factor in patients with acute myeloid leukemia treated with allogeneic hematopoietic stem-cell transplantation in first complete remission: a retrospective survey on behalf of the ALWP of the EBMT

Author

Brands-Nijenhuis, A. V. M., primary, Labopin, M., additional, Schouten, H. C., additional, Volin, L., additional, Socie, G., additional, Cornelissen, J. J., additional, Huynh, A., additional, Ljungman, P., additional, Malard, F., additional, Esteve, J., additional, Nagler, A., additional, and Mohty, M., additional

Published
2015
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179. Prevalence, risk factors, and impact on clinical outcome of extended-spectrum beta-lactamase-producing Escherichia coli bacteraemia: a five-year study

Author

Denis, B., primary, Lafaurie, M., additional, Donay, J.-L., additional, Fontaine, J.-P., additional, Oksenhendler, E., additional, Raffoux, E., additional, Hennequin, C., additional, Allez, M., additional, Socie, G., additional, Maziers, N., additional, Porcher, R., additional, and Molina, J.-M., additional

Published
2015
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180. Allogeneic hematopoietic cell transplantation in acute myeloid leukemia with normal karyotype and isolated Nucleophosmin-1 (NPM1) mutation: outcome strongly correlates with disease status

Author

Bazarbachi, A., primary, Labopin, M., additional, Kharfan-Dabaja, M. A., additional, Schwerdtfeger, R., additional, Volin, L., additional, Bourhis, J. H., additional, Socie, G., additional, Daguindau, E., additional, Gedde-Dahl, T., additional, Rambaldi, A., additional, Karas, M., additional, Schlimok, G., additional, Blaise, D., additional, Chevallier, P., additional, Malard, F., additional, Schmid, C., additional, Esteve, J., additional, Nagler, A., additional, and Mohty, M., additional

Published
2015
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181. Should irradiated blood products be given routinely to all patients with aplastic anaemia undergoing immunosuppressive therapy with antithymocyte globulin (ATG)? A survey from the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party

Author

Marsh, J., Socie, G., Tichelli, A., Schrezenmeier, H., Hochsmann, B., Risitano, A.M., Fuehrer, M., Bekassy, A.N., Korthof, E.T., Locasciulli, A., Ljungman, P., Bacigalupo, A., Camitta, B., Young, N.S., Passweg, J., and European Grp Blood Marrow Transpla

Subjects
antithymocyte globulin, transfusion-associated graft-versus-host disease, irradiated blood products, aplastic anaemia
Published
2010

182. The impact of graft-versus-host disease prophylaxis in reduced-intensity conditioning allogeneic stem cell transplant in acute myeloid leukemia: a study from the Acute Leukemia Working Party of the European Group for Blood and Marrow Transplantation

Author

Rubio, M. T., primary, Labopin, M., additional, Blaise, D., additional, Socie, G., additional, Contreras, R. R., additional, Chevallier, P., additional, Sanz, M. A., additional, Vigouroux, S., additional, Huynh, A., additional, Shimoni, A., additional, Bulabois, C.-E., additional, Caminos, N., additional, Lopez-Corral, L., additional, Nagler, A., additional, and Mohty, M., additional

Published
2015
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183. Fludarabine, cyclophosphamide, anti-thymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia (SAA): a retrospective study from the EBMT-SAA Working Party

Author

Bacigalupo, A, Socie', G, Lanino, E, Prete, A, Locatelli, F, Locasciulli, A, Cesaro, Simone, Shimoni, A, Marsh, J, Brune, M, van Lint MT, Oneto, R, and Passweg, J.

Subjects
Thymocyte globulin, alternative donor transplants, severe aplastic anemia
Published
2010

184. Mobilized peripheral blood stem cells compared with bone marrow for related reduced-intensity transplantation in acute myeloid leukaemia in complete remission: a retrospective analysis from the ALWP of EBMT

Author

Nagler, A., Labopin, M., Shimoni, A., Mufti, G., Cornelissen, J. J., Didier Blaise, Janssen, J. J. W. M., Milpied, N., Vindelov, L., Petersen, E., Gribben, J., Bacigalupo, A., Malm, C., Niederwieser, D., Socie, G., Arnold, R., Brown, P., Goker, H., Mohty, M., Rocha, V., Hematology, and CCA - Innovative therapy

Published
2010

185. Impact of in vivo T-cell depletion on outcome of AML patients in first CR given peripheral blood stem cells and reduced-intensity conditioning allo-SCT from a HLA-identical sibling donor : a report from the Acute Leukemia Working Party of the European group for Blood and Marrow Transplantation

Author

Baron, F., Labopin, M., Blaise, D., Lopez-Corral, L., Vigouroux, S., Craddock, C., Attal, M., Jindra, P., Goker, H., Socie, G., Chevallier, P., Browne, P., Sandstedt, A., Duarte, R. F., Nagler, A., Mohty, M., Baron, F., Labopin, M., Blaise, D., Lopez-Corral, L., Vigouroux, S., Craddock, C., Attal, M., Jindra, P., Goker, H., Socie, G., Chevallier, P., Browne, P., Sandstedt, A., Duarte, R. F., Nagler, A., and Mohty, M.

Abstract

The impact of in vivo T-cell depletion on transplantation outcomes in patients transplanted with reduced-intensity conditioning (RIC) remains controversial. This study assessed the outcome of 1250 adult patients with de novo AML in first CR (CR1) given PBSC from HLA-identical siblings after chemotherapy-based RIC. A total of 554 patients did not receive any form of in vivo T-cell depletion (control group), whereas antithymocyte globulin (ATG) and alemtuzumab were given in 444 and 252 patients, respectively. The incidences of grade II-IV acute GVHD were 21.4, 17.6 and 10.2% in control, ATG and alemtuzumab patients, respectively (P less than 0.001). In multivariate analysis, the use of ATG and the use of alemtuzumab were each associated with a lower risk of chronic GVHD (P less than 0.001 each), but a similar risk of relapse, and of nonrelapse mortality, and similar leukemia-free survival and OS. Further, among patients given BU-based RIC, the use of less than 6 mg/kg ATG did not increase the risk of relapse (hazard ratio, HR=1.1), whereas there was a suggestion for higher relapse risk in patients given greater than= 6 mg/kg ATG (HR=1.4, P=0.08). In summary, these data suggest that a certain amount of in vivo T-Cell depletion can be safely used in the conditioning of AML patients in CR1 given PBSC after chemotherapy-based RIC.

Published
2014
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186. Outcome of Allogeneic Stem Cell Transplantation for Patients Transformed to Myelodysplastic Syndrome or Leukemia from Severe Aplastic Anemia: A Report from the MDS Subcommittee of the Chronic Malignancies Working Party and the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

Author

Hussein, A.A., Halkes, C.M., Socie, G., Tichelli, A., Borne, P.A. von dem, Schaap, M.N., Foa, R., Ganser, A., Dufour, C., Bacigalupo, A., Locasciulli, A., Aljurf, M., Peters, C., Robin, M., Biezen, A.A. van, Volin, L., Witte, T.J. de, Marsh, J., Passweg, J.R., Kroger, N., et al., Hussein, A.A., Halkes, C.M., Socie, G., Tichelli, A., Borne, P.A. von dem, Schaap, M.N., Foa, R., Ganser, A., Dufour, C., Bacigalupo, A., Locasciulli, A., Aljurf, M., Peters, C., Robin, M., Biezen, A.A. van, Volin, L., Witte, T.J. de, Marsh, J., Passweg, J.R., Kroger, N., and et al.

Abstract

Item does not contain fulltext, One hundred and forty patients who had undergone hematopoietic stem cell transplantation (HSCT) for myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML) transformation after treatment of severe aplastic anemia (SAA) were identified in the European Group for Blood and Marrow Transplantation (EBMT) database. The median age at HSCT was 29 years (range, 1 to 66 years). The transplant donor was related in 49% cases and unrelated in 51% cases. The 5-year probability of relapse was 17%, and that of nonrelapse mortality was 41%. The 5-year overall survival was 45% +/- 9%, better for patients untreated and patients in remission compared with patients with refractory disease. Our data indicate that allogeneic HSCT leads to prolonged survival in close to one-half of the patients transforming to MDS or AML from SAA.

Published
2014

187. Prophylaxis and treatment of GVHD: EBMT-ELN working group recommendations for a standardized practice

Author

Ruutu, T., Gratwohl, A., Witte, T.J. de, Afanasyev, B., Apperley, J., Bacigalupo, A., Dazzi, F., Dreger, P., Duarte, R., Finke, J., Garderet, L., Greinix, H., Holler, E., Kroger, N., Lawitschka, A., Mohty, M., Nagler, A., Passweg, J., Ringden, O., Socie, G., Sierra, J., Sureda, A., Wiktor-Jedrzejczak, W., Madrigal, A., Niederwieser, D., Ruutu, T., Gratwohl, A., Witte, T.J. de, Afanasyev, B., Apperley, J., Bacigalupo, A., Dazzi, F., Dreger, P., Duarte, R., Finke, J., Garderet, L., Greinix, H., Holler, E., Kroger, N., Lawitschka, A., Mohty, M., Nagler, A., Passweg, J., Ringden, O., Socie, G., Sierra, J., Sureda, A., Wiktor-Jedrzejczak, W., Madrigal, A., and Niederwieser, D.

Abstract

Item does not contain fulltext, GVHD remains the major impediment to broader application of allogeneic haematopoietic SCT. It can be prevented completely, but at the expense of other complications, rejection, relapse or delayed immune reconstitution. No optimal prevention or treatment method has been defined. This is reflected by enormous heterogeneity in approaches in Europe. Retrospective comparisons between different policies, although warranted, do not give definite answers. In order to improve the present situation, an European Group for Blood and Marrow Transplantation and the European LeukemiaNet working group has developed in a Delphi-like approach recommendations for prophylaxis and treatment of GVHD in the most common allogeneic transplant setting, transplantation from an HLA-identical sibling or unrelated donor for standard risk malignant disease. The working group proposes these guidelines to be adopted as routine standard in transplantation centres and to be used as comparator in systematic studies evaluating the advantages and disadvantages of practices differing from these recommendations.

Published
2014

188. Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry

Author

Schrezenmeier, H., Muus, P., Socie, G., Szer, J., Urbano-Ispizua, A., Maciejewski, J.P., Brodsky, R.A., Bessler, M., Kanakura, Y., Rosse, W., Khursigara, G., Bedrosian, C., Hillmen, P., Schrezenmeier, H., Muus, P., Socie, G., Szer, J., Urbano-Ispizua, A., Maciejewski, J.P., Brodsky, R.A., Bessler, M., Kanakura, Y., Rosse, W., Khursigara, G., Bedrosian, C., and Hillmen, P.

Abstract

Contains fulltext : 136926.pdf (publisher's version ) (Open Access), Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life. The International PNH Registry is a worldwide, observational, non-interventional study collecting safety, effectiveness, and quality-of-life data from patients with a confirmed paroxysmal nocturnal hemoglobinuria diagnosis or detectable paroxysmal nocturnal hemoglobinuria clone, irrespective of treatment. In addition to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, optimize patient management and outcomes, and enhance the understanding of the natural history of paroxysmal nocturnal hemoglobinuria. Here we report the characteristics of the first 1610 patients enrolled. Median disease duration was 4.6 years. Median granulocyte paroxysmal nocturnal hemoglobinuria clone size was 68.1% (range 0.01-100%). Overall, 16% of patients had a history of thrombotic events and 14% a history of impaired renal function. Therapies included anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Frequently reported symptoms included fatigue (80%), dyspnea (64%), hemoglobinuria (62%), abdominal pain (44%), and chest pain (33%). Patients suffered from poor quality of life; 23% of patients had been hospitalized due to paroxysmal nocturnal hemoglobinuria-related complications and 17% stated that paroxysmal nocturnal hemoglobinuria was the reason they were not working or were working less. This international registry will provide an ongoing, valuable resource to further the clinical understanding of paroxysmal nocturnal hemoglobinuria.

Published
2014

189. THE REVISED IPSS (IPSS-R) AT TRANSPLANT PREDICTS OVERALL AND RELAPSE-FREE SURVIVAL AFTER ALLOGENEIC STEM CELL TRANSPLANTATION IN MDS/SAML: A RETROSPECTIVE ANALYSIS OF THE EBMT CHRONIC MALIGNANCIES WORKING PARTY

Author

Scheid, C., de Wreede, L., van Biezen, A., Goehring, G., Koenecke, C., Volin, L., Maertens, J., Finke, J., Schaap, N., Socie, G., Passweg, J., Cornelissen, J., Beelen, D., de Witte, T., Kroeger, N., Scheid, C., de Wreede, L., van Biezen, A., Goehring, G., Koenecke, C., Volin, L., Maertens, J., Finke, J., Schaap, N., Socie, G., Passweg, J., Cornelissen, J., Beelen, D., de Witte, T., and Kroeger, N.

Published
2014

190. Tyrosine kinase inhibitors improve long-term outcome of allogeneic hematopoietic stem cell transplantation for adult patients with Philadelphia chromosome positive acute lymphoblastic leukemia

Author

Brissot, E., primary, Labopin, M., additional, Beckers, M. M., additional, Socie, G., additional, Rambaldi, A., additional, Volin, L., additional, Finke, J., additional, Lenhoff, S., additional, Kroger, N., additional, Ossenkoppele, G. J., additional, Craddock, C. F., additional, Yakoub-Agha, I., additional, Gurman, G., additional, Russell, N. H., additional, Aljurf, M., additional, Potter, M. N., additional, Nagler, A., additional, Ottmann, O., additional, Cornelissen, J. J., additional, Esteve, J., additional, and Mohty, M., additional

Published
2014
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191. Matched unrelated donor stem cell transplant in 131 patients with follicular lymphoma: an analysis from the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation

Author

Avivi, I, Montoto, S, Canals, C, Maertens, J, Al-Ali, H, Mufti, GJ, Finke, J, Schattenberg, A, Fanin, R, Cornelissen, JJ, Vernant, JP, Russell, N, Beguin, Y, Thomson, K, Verdonck, LF, Kobbe, G, Tilly, H, Socie, G, Sureda, A, and Hematology

Subjects
matched unrelated donor transplant, surgical procedures, operative, follicular lymphoma, Translational research [ONCOL 3], hemic and lymphatic diseases, parasitic diseases, conditioning regimen
Abstract

Contains fulltext : 79862schattenberg.pdf (Publisher’s version ) (Closed access) Matched unrelated donor stem cell transplantation (MUD-SCT) provides the only curative option for patients with follicular lymphoma (FL) who fail conventional therapies and do not have a sibling donor. The purpose of this study was to analyse the outcome of patients with FL treated with MUD-SCT included in the European Group for Blood and Marrow Transplantation registry. 131 patients treated with reduced-intensity conditioning (RIC, n = 87) or conventional myeloablative (CONV, n = 44) MUD-SCT between 2000 and 2005 were included. Median time from diagnosis to MUD-SCT was 47 months and the median number of previous therapeutic regimens was 4 (previous autograft: 47%). RIC recipients were significantly older, with a longer interval from diagnosis to MUD-SCT and had failed a previous autograft more frequently than CONV recipients. Non-relapse mortality (NRM) was 24% and 30% at 100-d and 1-year, respectively. After a median follow-up of 36 months, 17% of the patients developed disease progression, the 3-year progression-free survival (PFS) being 47%. Three-year overall survival (OS) for the whole series was 51%. On multivariate analysis, RIC regimens were associated with at lower NRM and a significantly longer PFS and OS. This retrospective study demonstrated that MUD-SCT results, even in heavily pre-treated populations, in a meaningful PFS and OS.

Published
2009

193. Failure of zanamivir therapy for pneumonia in a bone-marrow transplant recipient infected by a zanamivir-sensitive influenza A (H1N1) virus

Author

Medeiros, R., Rameix-Welti, M. -A, Lorin, V., Ribaud, P., Manuguerra, J. -C, Socie, G., Scieux, C., Naffakh, N., Sylvie van der WERF, Science et Ingénierie des Matériaux et Procédés (SIMaP), Université Joseph Fourier - Grenoble 1 (UJF)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-Institut National Polytechnique de Grenoble (INPG)-Institut de Chimie du CNRS (INC)-Centre National de la Recherche Scientifique (CNRS), Service greffe de moelle osseuse, 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), Génétique Moléculaire des Virus Respiratoires, Centre National de la Recherche Scientifique (CNRS)-Université Paris Diderot - Paris 7 (UPD7)-Institut Pasteur [Paris], CHU Saint Louis, Institut Pasteur [Paris]-Université Paris Diderot - Paris 7 (UPD7)-Centre National de la Recherche Scientifique (CNRS), Université Joseph Fourier - Grenoble 1 (UJF)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP)-Institut National Polytechnique de Grenoble (INPG)-Centre National de la Recherche Scientifique (CNRS)-Université Grenoble Alpes (UGA), 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), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Université Paris Diderot - Paris 7 (UPD7)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], and Institut Pasteur [Paris] (IP)-Université Paris Diderot - Paris 7 (UPD7)-Centre National de la Recherche Scientifique (CNRS)

Subjects
Male, Pharmacology, viruses, Pneumonia, Viral, virus diseases, Middle Aged, biochemical phenomena, metabolism, and nutrition, Antiviral Agents, respiratory tract diseases, Influenza A Virus, H1N1 Subtype, Infectious Diseases, Recurrence, Influenza, Human, [SDV.MP.VIR]Life Sciences [q-bio]/Microbiology and Parasitology/Virology, Humans, Zanamivir, Pharmacology (medical), Treatment Failure, Bone Marrow Transplantation
Abstract

International audience; Influenza A viruses are responsible for significant morbidity and mortality after bone marrow transplantation. Here we report failure of inhaled zanamivir treatment in a bone-marrow transplant recipient with pneumonia caused by an influenza A (H1N1) virus, although the influenza viruses isolated from bronchoalveolar lavages before and after treatment were clearly found to be sensitive to zanamivir using cell-based and enzymatic assays. Subsequent oral treatment with oseltamivir allowed complete recovery. Poor bioavailability of zanamivir in the peripheral lungs might have been limiting treatment efficacy in such an immunocompromised patient.

Published
2007

198. Favorable impact of natural killer cell reconstitution on chronic graft-versus-host disease and cytomegalovirus reactivation after allogeneic hematopoietic stem cell transplantation

Author

Kheav, V. D., primary, Busson, M., additional, Scieux, C., additional, de Latour, R. P., additional, Maki, G., additional, Haas, P., additional, Mazeron, M.-C., additional, Carmagnat, M., additional, Masson, E., additional, Xhaard, A., additional, Robin, M., additional, Ribaud, P., additional, Dulphy, N., additional, Loiseau, P., additional, Charron, D., additional, Socie, G., additional, Toubert, A., additional, and Moins-Teisserenc, H., additional

Published
2014
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199. Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

Author

Dufour, C., primary, Pillon, M., additional, Passweg, J., additional, Socie, G., additional, Bacigalupo, A., additional, Franceschetto, G., additional, Carraro, E., additional, Oneto, R., additional, Risitano, A. M., additional, de Latour, R. P., additional, Tichelli, A., additional, Rovo, A., additional, Peters, C., additional, Hoechsmann, B., additional, Samarasinghe, S., additional, Kulasekararaj, A. G., additional, Schrezenmeier, H., additional, Aljurf, M., additional, and Marsh, J., additional

Published
2014
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