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2. Late treatment-related mortality versus competing causes of death after allogeneic transplantation for myelodysplastic syndromes and secondary acute myeloid leukemia

Author

Schetelig, Johannes, de Wreede, Liesbeth C., van Gelder, Michel, Koster, Linda, Finke, Jürgen, Niederwieser, Dietger, Beelen, Dietrich, Mufti, G. J., Platzbecker, Uwe, Ganser, Arnold, Heidenreich, Silke, Maertens, Johan, Socié, Gerard, Brecht, Arne, Stelljes, Matthias, Kobbe, Guido, Volin, Liisa, Nagler, Arnon, Vitek, Antonin, Luft, Thomas, Ljungman, Per, Yakoub-Agha, Ibrahim, Robin, Marie, and Kröger, Nicolaus

Published
2019
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7. Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study.

Author

Gattermann N., Finelli C., Della Porta M., Fenaux P., Ganser A., Guerci-Bresler A., Schmid M., Taylor K., Vassilieff D., Habr D., Domokos G., Roubert B., Rose C., Agaoglu L., Alimena G., Alonso D., Ame S., Angelucci E., Arrizabalaga B., Athanasiou-Metaxa M., Augustson B., Aydinok Y., Baba A., Baccarani M., Beck J., Beris P., Beyne-Rauzy O., Birgens H., Bordessoule D., Borgna-Pignatti C., Bosly A., Bouabdallah K., Bowden D., Bowen D., Bron D., Cappellini M.D., Capra M., Cartron G., Cazzola M., Chalkias C., Chan L.L., Chancharunee S., Chapman C., Charoenkwan P., Chasapopoulou E., Cheze S., Chuansumrit A., Cianciulli P., Dauriac C., Delforge M., Dolken G., Dombret H., Duyster J., Economopoulos T., Ehninger G., Elalfy M., El-Beshlawy A., Enggaard L., Fillet G., Filosa A., Forni G., Galanello R., Gastl G., Giraudier S., Goldfarb A., Grigg A., Gumruk F., Ha S.Y., Haase D., Heinrich B., Hertzberg M., Ho J., Hsu H.-C., Huang S., Hunault-Berger M., Inusa B., Jaulmes D., Jensen J., Kattamis A., Kilinc Y., Kim K.-H., Kinsey S., Kjeldsen L., Koren A., Lai M.E., Lai Y., Lee J.-W., Lee K.-H., Lee S.-H., Legros L., Li C., Li C.-K., Li Q., Lin K.-H., Linkesch W., Lubbert M., Lutz D., Mohamed Thalha A.J., Mufti G., Muus P., Nobile F., Papadopoulos N., Perrotta S., Petrini M., Pfeilstocker M., Piga A., Poole J., Porter J.B., Pungolino E., Quarta G., Ravoet C., Jolimont Lobbes H.H., Remacha A.F., Roy L., Saglio G., Sanz G., Schmugge M., Schots H., Secchi G., Seymour J.F., Shah F., Shah H., Shen Z., Slama B., Sutcharitchan P., Taher A., Tamary H., Tesch H.-J., Thein S.L., Troncy J., Villegas A., Viprakasit V., Wainwright L., Wassmann B., Wettervald M., Will A., Wormann B., Wright J., Yeh S.-P., Yoon S.-S., Zoumbos N.C., Zweegman S., Gattermann N., Finelli C., Della Porta M., Fenaux P., Ganser A., Guerci-Bresler A., Schmid M., Taylor K., Vassilieff D., Habr D., Domokos G., Roubert B., Rose C., Agaoglu L., Alimena G., Alonso D., Ame S., Angelucci E., Arrizabalaga B., Athanasiou-Metaxa M., Augustson B., Aydinok Y., Baba A., Baccarani M., Beck J., Beris P., Beyne-Rauzy O., Birgens H., Bordessoule D., Borgna-Pignatti C., Bosly A., Bouabdallah K., Bowden D., Bowen D., Bron D., Cappellini M.D., Capra M., Cartron G., Cazzola M., Chalkias C., Chan L.L., Chancharunee S., Chapman C., Charoenkwan P., Chasapopoulou E., Cheze S., Chuansumrit A., Cianciulli P., Dauriac C., Delforge M., Dolken G., Dombret H., Duyster J., Economopoulos T., Ehninger G., Elalfy M., El-Beshlawy A., Enggaard L., Fillet G., Filosa A., Forni G., Galanello R., Gastl G., Giraudier S., Goldfarb A., Grigg A., Gumruk F., Ha S.Y., Haase D., Heinrich B., Hertzberg M., Ho J., Hsu H.-C., Huang S., Hunault-Berger M., Inusa B., Jaulmes D., Jensen J., Kattamis A., Kilinc Y., Kim K.-H., Kinsey S., Kjeldsen L., Koren A., Lai M.E., Lai Y., Lee J.-W., Lee K.-H., Lee S.-H., Legros L., Li C., Li C.-K., Li Q., Lin K.-H., Linkesch W., Lubbert M., Lutz D., Mohamed Thalha A.J., Mufti G., Muus P., Nobile F., Papadopoulos N., Perrotta S., Petrini M., Pfeilstocker M., Piga A., Poole J., Porter J.B., Pungolino E., Quarta G., Ravoet C., Jolimont Lobbes H.H., Remacha A.F., Roy L., Saglio G., Sanz G., Schmugge M., Schots H., Secchi G., Seymour J.F., Shah F., Shah H., Shen Z., Slama B., Sutcharitchan P., Taher A., Tamary H., Tesch H.-J., Thein S.L., Troncy J., Villegas A., Viprakasit V., Wainwright L., Wassmann B., Wettervald M., Will A., Wormann B., Wright J., Yeh S.-P., Yoon S.-S., Zoumbos N.C., and Zweegman S.

Abstract

The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500. ng/mL, ~50% were chelation-naive. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-naive or previously chelated; changes were dependent on dose adjustments and ongoing iron intake. Sustained reductions in labile plasma iron were observed. Discontinuation rate (48.7%) and adverse event profile were consistent with previously reported deferasirox data in MDS. Alanine aminotransferase levels decreased significantly; change correlated significantly with reduction in serum ferritin (p<0.0001). This large dataset prospectively confirms the efficacy and well characterizes the safety profile of deferasirox in MDS. © 2010 Elsevier Ltd.

Published
2021

8. Standardization of flow cytometry in myelodysplastic syndromes: a report from an international consortium and the European LeukemiaNet Working Group

Author

Westers, T M, Ireland, R, Kern, W, Alhan, C, Balleisen, J S, Bettelheim, P, Burbury, K, Cullen, M, Cutler, J A, Della Porta, M G, Dräger, A M, Feuillard, J, Font, P, Germing, U, Haase, D, Johansson, U, Kordasti, S, Loken, M R, Malcovati, L, te Marvelde, J G, Matarraz, S, Milne, T, Moshaver, B, Mufti, G J, Ogata, K, Orfao, A, Porwit, A, Psarra, K, Richards, S J, Subirá, D, Tindell, V, Vallespi, T, Valent, P, van der Velden, V H J, de Witte, T M, Wells, D A, Zettl, F, Béné, M C, and van de Loosdrecht, A A

Published
2012
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15. Allogeneic stem cell transplantation in second complete remission for core binding factor acute myeloid leukemia: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Author

Halaburda, K, Labopin, M, Mailhol, A, Socie, G, Craddock, C, Aljurf, M, Beelen, D, Cornelissen, Jan, Bourhis, J H, Labussiere-Wallet, H, Blaise, D, Gedde-Dahl, T, Gilleece, M, Yakoub-Agha, I, Mufti, G, Esteve, J, Mohty, M, Nagler, A, Halaburda, K, Labopin, M, Mailhol, A, Socie, G, Craddock, C, Aljurf, M, Beelen, D, Cornelissen, Jan, Bourhis, J H, Labussiere-Wallet, H, Blaise, D, Gedde-Dahl, T, Gilleece, M, Yakoub-Agha, I, Mufti, G, Esteve, J, Mohty, M, and Nagler, A

Published
2020

22. Transplant results in adults with Fanconi anaemia

Author

Bierings M., Bonfim C., Peffault De Latour R., Aljurf M., Mehta P. A., Knol C., Boulad F., Tbakhi A., Esquirol A., McQuaker G., Sucak G. A., Othman T. B., Halkes C. J. M., Carpenter B., Niederwieser D., Zecca M., Kroger N., Michallet M., Risitano A. M., Ehninger G., Porcher R., Dufour C., Bonfirm C., Socie G., Gurman G., Ghavamzadeh A., Hamladji R. -M., van Lint M. T., Stepensky P., Koh M., Ozkurt Z. N., Veelken J. H., Bunjes D., Beelen D., Campos A., Robinson S., Alessandrino E. P., Unal A., Fernandez Navarro J. M., Mufti G. J., Velardi A., Passweg J., Apperley J., Sengeloev H., Ljungman P., Foa R., Alegre A., Espiga C. R., Cornelissen J. J., Di Bartolomeo P., Cordonnier C., Browne P., Jubert C., Gastl G., Pierelli L., Johansson J. -E., Fagioli F., Moraleda J., Zuckerman T., Bazarbachi A., Sedlacek P., Rossig C., Wynn R. F., Hallek M., Toren A., Zudaire T., Clausen J., Spencer A., Grazon Lopez S., Schots R., Komarnicki M., Gonzalez Muniz S., Vitek A., Rambaldi A., Merli F., Rubio M. T., Cabrera Marin J. R., Porto F., Kerre T., Metzner B., Stein J., Bertrand Y., Ciceri F., Chybicka A., Diez-Martin J. L., Bayoumy M., de la Fuente J., Fegueux N., Bierings, M., Bonfim, C., Peffault De Latour, R., Aljurf, M., Mehta, P. A., Knol, C., Boulad, F., Tbakhi, A., Esquirol, A., Mcquaker, G., Sucak, G. A., Othman, T. B., Halkes, C. J. M., Carpenter, B., Niederwieser, D., Zecca, M., Kroger, N., Michallet, M., Risitano, A. M., Ehninger, G., Porcher, R., Dufour, C., Bonfirm, C., Socie, G., Gurman, G., Ghavamzadeh, A., Hamladji, R. -M., van Lint, M. T., Stepensky, P., Koh, M., Ozkurt, Z. N., Veelken, J. H., Bunjes, D., Beelen, D., Campos, A., Robinson, S., Alessandrino, E. P., Unal, A., Fernandez Navarro, J. M., Mufti, G. J., Velardi, A., Passweg, J., Apperley, J., Sengeloev, H., Ljungman, P., Foa, R., Alegre, A., Espiga, C. R., Cornelissen, J. J., Di Bartolomeo, P., Cordonnier, C., Browne, P., Jubert, C., Gastl, G., Pierelli, L., Johansson, J. -E., Fagioli, F., Moraleda, J., Zuckerman, T., Bazarbachi, A., Sedlacek, P., Rossig, C., Wynn, R. F., Hallek, M., Toren, A., Zudaire, T., Clausen, J., Spencer, A., Grazon Lopez, S., Schots, R., Komarnicki, M., Gonzalez Muniz, S., Vitek, A., Rambaldi, A., Merli, F., Rubio, M. T., Cabrera Marin, J. R., Porto, F., Kerre, T., Metzner, B., Stein, J., Bertrand, Y., Ciceri, F., Chybicka, A., Diez-Martin, J. L., Bayoumy, M., de la Fuente, J., Fegueux, N., Clinical sciences, Hematology, Bierings, Marc, Bonfim, Carmem, Peffault De Latour, Regi, Aljurf, Mahmoud, Mehta, Parinda A., Knol, Cora, Boulad, Farid, Tbakhi, Abdelghani, Esquirol, Albert, Mcquaker, Grant, Sucak, Gulsan A., Othman, Tarek B., Halkes, Constantijn J. M., Carpenter, Ben, Niederwieser, Dietger, Zecca, Marco, Kröger, Nicolau, Michallet, Mauricette, Risitano, Antonio M., Ehninger, Gerhard, Porcher, Raphael, Dufour, Carlo, Bonfirm, Carmem, Socié, Gerard, Gurman, Gunham, Ghavamzadeh, Ardesir, Hamladji, Rise-Marie, Stepensky, Polina, Koh, Mickey, Ozkurt, Zubeyde Nur, Veelken, Joan Hendrik, Bunjes, Donald, Beelen, Dietrich, Campos, Antonio, Robinson, Stephen, Alessandrino, E. Paolo, Unal, Ali, Fernandez Navarro, José Maria, Velardi, Andrea, Passweg, Jakob, Apperley, Jane, Sengeloev, Henrik, Ljungman, Per, Foá, Roberto, Alegre, Adrián, Espiga, Carlos Richard, Di Bartolomeo, Paolo, Cordonnier, Catherine, Browne, Paul, Jubert, Charlotte, Gastl, Günther, Pierelli, Luca, Johansson, Jan-Erik, Fagioli, Franca, Moraleda, José, Zuckerman, Tsila, Bazarbachi, Ali, Sedlacek, Petr, Rössig, Claudia, Hallek, Michael, Toren, Amo, Zudaire, Teresa, Clausen, Joahanne, Spencer, Andrew, Grazon Lopez, Sebastian, Schots, Rik, González Muniz, Soledad, Vitek, Antonin, Rambaldi, Alessandro, Merli, Francesco, Rubio, Marie Thérese, Rossig, Claudia, Cabrera Marín, José Rafael, Porto, Fulvio, Kerre, Tessa, Metzner, Bernd, Stein, Jerry, Bertrand, Yve, Ciceri, Fabio, Chybicka, Alicja, Bayoumy, Mohamed, de la Fuente, Josu, and Fegueux, Nathalie

Subjects
Transplantation Conditioning, Graft vs Host Disease, Disease, Gastroenterology, 0302 clinical medicine, Retrospective Studie, Risk Factors, Inborn bone marrow failure syndrome, Neoplasms, Cause of Death, risk factors, inborn bone marrow failure syndrome, Transplantation, Homologou, Medicine(all), allogeneic transplant, Fanconi anaemia, myelodysplasia, Adolescent, Adult, Fanconi Anemia, Humans, Middle Aged, Neoplasms, Second Primary, Prognosis, Retrospective Studies, Survival Analysis, Tissue Donors, Transplantation, Homologous, Treatment Outcome, Young Adult, Hematopoietic Stem Cell Transplantation, Hematology, Incidence (epidemiology), Fludarabine, Second Primary, medicine.anatomical_structure, surgical procedures, operative, 030220 oncology & carcinogenesis, young adult, Survival Analysi, Human, medicine.drug, Homologous, medicine.medical_specialty, Cyclophosphamide, Prognosi, Myelodysplasia, Tissue Donor, Graft vs Host Disease/etiology, 03 medical and health sciences, Internal medicine, medicine, Hematopoietic Stem Cell Transplantation/adverse effects, Sibling, Transplantation, business.industry, Risk Factor, Neoplasms, Second Primary/etiology, Confidence interval, Allogeneic transplant, Surgery, Bone marrow, business, Fanconi Anemia/diagnosis, 030215 immunology
Abstract

The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16years of age when diagnosed with FA, and underwent transplantation at a median age of 23years. Time between diagnosis and transplant was shortest (median 2years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76–87%), acute graft-versus-host disease (GvHD) grade II–IV in 22% (95% CI 16–28%) and the incidence of chronic GvHD at 96months was 26% (95% CI 20–33). Non-relapse mortality at 96months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58months. Patients transplanted after 2000 had improved survival (84% at 36months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.

Published
2018

23. Long-term survival of patients with CLL after allogeneic transplantation: A report from the European Society for Blood and Marrow Transplantation

Author

Van Gelder, M, De Wreede, L, Bornhauser, M, Niederwieser, D, Karas, M, Anderson, N, Gramatzki, M, Dreger, P, Michallet, M, Petersen, E, Bunjes, D, Potter, M, Beelen, D, Cornelissen, J, Yakoub-Agha, I, Russell, N, Finke, J, Schoemans, H, Vitek, A, Urbano-Ispizua, A, Blaise, D, Volin, L, Chevallier, P, Caballero, D, Putter, H, Van Biezen, A, Henseler, A, Schonland, S, Kroger, N, Schetelig, J, Ehninger, G, Jindra, P, Sengeloev, H, Ispizua, A, Arnold, R, Veelken, J, Mufti, G, Milpied, N, Benedetto, B, Schaap, M, Leblond, V, Nikolousis, M, Hallek, M, Passweg, J, Ljungman, P, Masszi, T, Stelljes, M, Browne, P, Glass, B, Espiga, C, Bourhis, J, Roussy, G, Gribben, J, Foa, R, Sierra, J, Mayer, J, Thomson, K, Meijer, E, Blau, W, Holler, E, Bacigalupo, A, Guilhot, F, Carlson, K, Zachee, P, Ifrah, N, Marin, J, Socie, G, Mcquaker, G, Cortelezzi, A, Lenhoff, S, Tischer, J, Irrera, G, Fanin, R, Beguin, Y, Nagler, A, Mackinnon, S, Itala-Remes, M, Deconinck, E, Wulf, G, Corradini, P, Gilleece, M, Wing, B, Peniket, A, Ganser, A, Stuhler, G, Faber, E, Komarnicki, M, Kanz, L, Brune, M, Lamy, T, Sanz, M, Kyrcz-Krzemien, S, Orchard, K, Hunter, A, Sandstedt, A, Fegueux, N, Bandini, G, Robinson, S, Craddock, C, Crawley, C, Griskevicius, L, Bloor, A, Reman, O, Hilgendorf, I, Cannell, P, Ciceri, F, Kalhs, P, Sica, S, Greinix, H, Scime, R, Selleslag, D, Kruger, W, Huynh, A, Einsele, H, Bittenbring, J, Olivieri, A, Hermine, O, Gedde-Dahl, T, Zsiros, J, Guyotat, D, Cordonnier, C, Campos, A, Casini, M, Martinelli, G, Muller, L, Van Imhoff, G, Neubauer, A, Lioure, B, Hamladji, R, Noens, L, Theobald, M, Salvi, F, Ram, R, Poire, X, Or, R, Chalandon, Y, Solano, C, Wilson, K, Santasusana, J, Karakasis, D, Schafer-Eckart, K, Wahlin, A, Mohty, M, Velardi, A, Bron, D, Alegre, A, Cairoli, R, Marotta, G, Lange, A, Narni, F, Fauser, A, Rambaldi, A, Guillerm, G, Heras, I, Snowden, J, Wiktor-Jedrzejczak, W, Schanz, U, Cahn, J, Abecasis, M, Kobbe, G, Salim, R, Junghanss, C, Segel, E, Clement, L, Zak, P, Metzner, B, Espigado, I, Tilly, H, Schroyens, W, Favre, C, Russo, D, Gastl, G, Bay, J, Alessandrino, E, Majolino, I, Bosi, A, Zuckerman, T, Aljurf, M, Thomson, J, Pioltelli, P, Anagnostopoulos, A, Schouten, H, Tholouli, E, Gurman, G, Vural, F, Zver, S, Muniz, S, Afanasyev, B, Pohlreich, D, Hellmann, A, Rosler, W, Martin, S, Apperley, J, Finnegan, D, Renaud, M, Nemet, D, Culligan, D, Castagna, L, Cascavilla, N, Koh, M, Chacon, M, Ozdogu, H, Spencer, A, Llamas, C, Grasso, M, Lopez, S, Benedetti, F, Deeren, D, De Revel, T, Musso, M, Halaburda, K, Sureda, A, Angelucci, E, Diez-Martin, J, Hunter, H, Koc, Y, Bordessoule, D, Fouillard, L, Di Bartolomeo, P, Mazza, P, Novitzky, N, Peschel, C, Lopez, J, Cascon, M, Romeril, K, Schots, R, Brussel, H, Koistinen, P, Arcese, W, Aktan, M, Rodeghiero, F, Butler, A, Pizzuti, M, Melpignano, A, Carella, A, Valcarcel, D, De Toledo Codina, J, Galieni, P, Bader, P, Hahn, Cavanna, L, Sucak, G, Broom, A, Garcia, P, Nicolas-Virelizier, E, Rizzoli, V, Witz, F, Collin, M, Ringhoffer, M, Kansu, E, Martin, H, Moraleda, J, Pranger, D, Greil, R, Bazarbachi, A, Ozturk, M, Fagioli, F, Jantunen, E, Yeshurun, M, Altuntas, F, Bassan, R, Rohrlich, P, Jimenez, S, Glaisner, S, Vinante, O, Clausen, J, Lopez-Jimenez, J, Theunissen, K, Specchia, G, Pavone, V, Krauter, J, Edwards, D, Rifon, J, Everaus, H, Da Prada, G, Wattad, M, Milone, G, Walewski, J, Thieblemont, C, Nasa, G, Duchosal, M, Ferrara, F, Devidas, A, Delmer, A, Degos, L, Van Gelder M., De Wreede L. C., Bornhauser M., Niederwieser D., Karas M., Anderson N. S., Gramatzki M., Dreger P., Michallet M., Petersen E., Bunjes D., Potter M., Beelen D., Cornelissen J. J., Yakoub-Agha I., Russell N. H., Finke J., Schoemans H., Vitek A., Urbano-Ispizua A., Blaise D., Volin L., Chevallier P., Caballero D., Putter H., Van Biezen A., Henseler A., Schonland S., Kroger N., Schetelig J., Ehninger G., Jindra P., Sengeloev H., Russell N., Ispizua A. U., Arnold R., Veelken J. H., Mufti G., Milpied N., Benedetto B., Schaap M., Leblond V., Nikolousis M., Hallek M., Passweg J., Ljungman P., Masszi T., Stelljes M., Browne P., Glass B., Espiga C. R., Bourhis J. H., Roussy G., Gribben J., Foa R., Sierra J., Mayer J., Thomson K., Meijer E., Blau W., Holler E., Bacigalupo A., Guilhot F., Carlson K., Zachee P., Ifrah N., Marin J. R. C., Socie G., McQuaker G., Cortelezzi A., Lenhoff S., Tischer J., Irrera G., Fanin R., Beguin Y., Nagler A., Mackinnon S., Itala-Remes M., Deconinck E., Wulf G., Corradini P., Gilleece M., Wing B., Peniket A., Ganser A., Stuhler G., Faber E., Komarnicki M., Kanz L., Brune M., Lamy T., Sanz M., Kyrcz-Krzemien S., Orchard K., Hunter A., Sandstedt A., Fegueux N., Bandini G., Robinson S., Craddock C., Crawley C., Griskevicius L., Bloor A., Reman O., Hilgendorf I., Cannell P., Ciceri F., Kalhs P., Sica S., Greinix H., Scime R., Selleslag D., Kruger W., Huynh A., Einsele H., Bittenbring J., Olivieri A., Hermine O., Gedde-Dahl T., Zsiros J., Guyotat D., Cordonnier C., Campos A., Casini M., Martinelli G., Muller L. P., Van Imhoff G., Neubauer A., Lioure B., Hamladji R. -M., Noens L., Theobald M., Salvi F., Ram R., Poire X., Or R., Chalandon Y., Solano C., Wilson K., Santasusana J. M. R., Karakasis D., Schafer-Eckart K., Wahlin A., Mohty M., Velardi A., Bron D., Alegre A., Cairoli R., Marotta G., Lange A., Narni F., Fauser A., Rambaldi A., Guillerm G., Heras I., Snowden J., Wiktor-Jedrzejczak W., Schanz U., Cahn J. Y., Abecasis M., Kobbe G., Salim R., Junghanss C., Segel E. K., Clement L., Zak P., Metzner B., Espigado I., Tilly H., Schroyens W., Favre C., Russo D., Gastl G., Bay J. -O., Alessandrino E. P., Majolino I., Bosi A., Zuckerman T., Aljurf M., Thomson J., Pioltelli P., Anagnostopoulos A., Schouten H., Tholouli E., Gurman G., Vural F., Zver S., Muniz S. G., Afanasyev B., Pohlreich D., Hellmann A., Rosler W., Martin S., Apperley J., Finnegan D., Renaud M., Nemet D., Culligan D., Castagna L., Cascavilla N., Koh M., Chacon M. J., Ozdogu H., Spencer A., Llamas C. V., Grasso M., Lopez S. G., Benedetti F., Deeren D., De Revel T., Musso M., Halaburda K., Sureda A., Angelucci E., Diez-Martin J. L., Hunter H., Koc Y., Bordessoule D., Fouillard L., Di Bartolomeo P., Mazza P., Novitzky N., Peschel C., Lopez J. L. B., Cascon M. J. P., Romeril K. R., Schots R., Brussel H., Koistinen P., Arcese W., Aktan M., Rodeghiero F., Butler A., Pizzuti M., Melpignano A., Carella A. M., Valcarcel D., De Toledo Codina J. S., Galieni P., Bader P., Cavanna L., Sucak G., Broom A. J. M., Garcia P. G., Nicolas-Virelizier E., Rizzoli V., Witz F., Collin M., Ringhoffer M., Kansu E., Martin H., Moraleda J., Pranger D., Greil R., Bazarbachi A., Ozturk M., Fagioli F., Jantunen E., Yeshurun M., Altuntas F., Bassan R., Rohrlich P. -S., Jimenez S., Glaisner S., Vinante O., Clausen J., Lopez-Jimenez J., Theunissen K., Specchia G., Pavone V., Krauter J., Edwards D., Rifon J., Everaus H., Da Prada G. A., Wattad M., Milone G., Walewski J., Thieblemont C., Nasa G. L., Duchosal M., Ferrara F., Devidas A., Delmer A., Degos L., Van Gelder, M, De Wreede, L, Bornhauser, M, Niederwieser, D, Karas, M, Anderson, N, Gramatzki, M, Dreger, P, Michallet, M, Petersen, E, Bunjes, D, Potter, M, Beelen, D, Cornelissen, J, Yakoub-Agha, I, Russell, N, Finke, J, Schoemans, H, Vitek, A, Urbano-Ispizua, A, Blaise, D, Volin, L, Chevallier, P, Caballero, D, Putter, H, Van Biezen, A, Henseler, A, Schonland, S, Kroger, N, Schetelig, J, Ehninger, G, Jindra, P, Sengeloev, H, Ispizua, A, Arnold, R, Veelken, J, Mufti, G, Milpied, N, Benedetto, B, Schaap, M, Leblond, V, Nikolousis, M, Hallek, M, Passweg, J, Ljungman, P, Masszi, T, Stelljes, M, Browne, P, Glass, B, Espiga, C, Bourhis, J, Roussy, G, Gribben, J, Foa, R, Sierra, J, Mayer, J, Thomson, K, Meijer, E, Blau, W, Holler, E, Bacigalupo, A, Guilhot, F, Carlson, K, Zachee, P, Ifrah, N, Marin, J, Socie, G, Mcquaker, G, Cortelezzi, A, Lenhoff, S, Tischer, J, Irrera, G, Fanin, R, Beguin, Y, Nagler, A, Mackinnon, S, Itala-Remes, M, Deconinck, E, Wulf, G, Corradini, P, Gilleece, M, Wing, B, Peniket, A, Ganser, A, Stuhler, G, Faber, E, Komarnicki, M, Kanz, L, Brune, M, Lamy, T, Sanz, M, Kyrcz-Krzemien, S, Orchard, K, Hunter, A, Sandstedt, A, Fegueux, N, Bandini, G, Robinson, S, Craddock, C, Crawley, C, Griskevicius, L, Bloor, A, Reman, O, Hilgendorf, I, Cannell, P, Ciceri, F, Kalhs, P, Sica, S, Greinix, H, Scime, R, Selleslag, D, Kruger, W, Huynh, A, Einsele, H, Bittenbring, J, Olivieri, A, Hermine, O, Gedde-Dahl, T, Zsiros, J, Guyotat, D, Cordonnier, C, Campos, A, Casini, M, Martinelli, G, Muller, L, Van Imhoff, G, Neubauer, A, Lioure, B, Hamladji, R, Noens, L, Theobald, M, Salvi, F, Ram, R, Poire, X, Or, R, Chalandon, Y, Solano, C, Wilson, K, Santasusana, J, Karakasis, D, Schafer-Eckart, K, Wahlin, A, Mohty, M, Velardi, A, Bron, D, Alegre, A, Cairoli, R, Marotta, G, Lange, A, Narni, F, Fauser, A, Rambaldi, A, Guillerm, G, Heras, I, Snowden, J, Wiktor-Jedrzejczak, W, Schanz, U, Cahn, J, Abecasis, M, Kobbe, G, Salim, R, Junghanss, C, Segel, E, Clement, L, Zak, P, Metzner, B, Espigado, I, Tilly, H, Schroyens, W, Favre, C, Russo, D, Gastl, G, Bay, J, Alessandrino, E, Majolino, I, Bosi, A, Zuckerman, T, Aljurf, M, Thomson, J, Pioltelli, P, Anagnostopoulos, A, Schouten, H, Tholouli, E, Gurman, G, Vural, F, Zver, S, Muniz, S, Afanasyev, B, Pohlreich, D, Hellmann, A, Rosler, W, Martin, S, Apperley, J, Finnegan, D, Renaud, M, Nemet, D, Culligan, D, Castagna, L, Cascavilla, N, Koh, M, Chacon, M, Ozdogu, H, Spencer, A, Llamas, C, Grasso, M, Lopez, S, Benedetti, F, Deeren, D, De Revel, T, Musso, M, Halaburda, K, Sureda, A, Angelucci, E, Diez-Martin, J, Hunter, H, Koc, Y, Bordessoule, D, Fouillard, L, Di Bartolomeo, P, Mazza, P, Novitzky, N, Peschel, C, Lopez, J, Cascon, M, Romeril, K, Schots, R, Brussel, H, Koistinen, P, Arcese, W, Aktan, M, Rodeghiero, F, Butler, A, Pizzuti, M, Melpignano, A, Carella, A, Valcarcel, D, De Toledo Codina, J, Galieni, P, Bader, P, Hahn, Cavanna, L, Sucak, G, Broom, A, Garcia, P, Nicolas-Virelizier, E, Rizzoli, V, Witz, F, Collin, M, Ringhoffer, M, Kansu, E, Martin, H, Moraleda, J, Pranger, D, Greil, R, Bazarbachi, A, Ozturk, M, Fagioli, F, Jantunen, E, Yeshurun, M, Altuntas, F, Bassan, R, Rohrlich, P, Jimenez, S, Glaisner, S, Vinante, O, Clausen, J, Lopez-Jimenez, J, Theunissen, K, Specchia, G, Pavone, V, Krauter, J, Edwards, D, Rifon, J, Everaus, H, Da Prada, G, Wattad, M, Milone, G, Walewski, J, Thieblemont, C, Nasa, G, Duchosal, M, Ferrara, F, Devidas, A, Delmer, A, Degos, L, Van Gelder M., De Wreede L. C., Bornhauser M., Niederwieser D., Karas M., Anderson N. S., Gramatzki M., Dreger P., Michallet M., Petersen E., Bunjes D., Potter M., Beelen D., Cornelissen J. J., Yakoub-Agha I., Russell N. H., Finke J., Schoemans H., Vitek A., Urbano-Ispizua A., Blaise D., Volin L., Chevallier P., Caballero D., Putter H., Van Biezen A., Henseler A., Schonland S., Kroger N., Schetelig J., Ehninger G., Jindra P., Sengeloev H., Russell N., Ispizua A. U., Arnold R., Veelken J. H., Mufti G., Milpied N., Benedetto B., Schaap M., Leblond V., Nikolousis M., Hallek M., Passweg J., Ljungman P., Masszi T., Stelljes M., Browne P., Glass B., Espiga C. R., Bourhis J. H., Roussy G., Gribben J., Foa R., Sierra J., Mayer J., Thomson K., Meijer E., Blau W., Holler E., Bacigalupo A., Guilhot F., Carlson K., Zachee P., Ifrah N., Marin J. R. C., Socie G., McQuaker G., Cortelezzi A., Lenhoff S., Tischer J., Irrera G., Fanin R., Beguin Y., Nagler A., Mackinnon S., Itala-Remes M., Deconinck E., Wulf G., Corradini P., Gilleece M., Wing B., Peniket A., Ganser A., Stuhler G., Faber E., Komarnicki M., Kanz L., Brune M., Lamy T., Sanz M., Kyrcz-Krzemien S., Orchard K., Hunter A., Sandstedt A., Fegueux N., Bandini G., Robinson S., Craddock C., Crawley C., Griskevicius L., Bloor A., Reman O., Hilgendorf I., Cannell P., Ciceri F., Kalhs P., Sica S., Greinix H., Scime R., Selleslag D., Kruger W., Huynh A., Einsele H., Bittenbring J., Olivieri A., Hermine O., Gedde-Dahl T., Zsiros J., Guyotat D., Cordonnier C., Campos A., Casini M., Martinelli G., Muller L. P., Van Imhoff G., Neubauer A., Lioure B., Hamladji R. -M., Noens L., Theobald M., Salvi F., Ram R., Poire X., Or R., Chalandon Y., Solano C., Wilson K., Santasusana J. M. R., Karakasis D., Schafer-Eckart K., Wahlin A., Mohty M., Velardi A., Bron D., Alegre A., Cairoli R., Marotta G., Lange A., Narni F., Fauser A., Rambaldi A., Guillerm G., Heras I., Snowden J., Wiktor-Jedrzejczak W., Schanz U., Cahn J. Y., Abecasis M., Kobbe G., Salim R., Junghanss C., Segel E. K., Clement L., Zak P., Metzner B., Espigado I., Tilly H., Schroyens W., Favre C., Russo D., Gastl G., Bay J. -O., Alessandrino E. P., Majolino I., Bosi A., Zuckerman T., Aljurf M., Thomson J., Pioltelli P., Anagnostopoulos A., Schouten H., Tholouli E., Gurman G., Vural F., Zver S., Muniz S. G., Afanasyev B., Pohlreich D., Hellmann A., Rosler W., Martin S., Apperley J., Finnegan D., Renaud M., Nemet D., Culligan D., Castagna L., Cascavilla N., Koh M., Chacon M. J., Ozdogu H., Spencer A., Llamas C. V., Grasso M., Lopez S. G., Benedetti F., Deeren D., De Revel T., Musso M., Halaburda K., Sureda A., Angelucci E., Diez-Martin J. L., Hunter H., Koc Y., Bordessoule D., Fouillard L., Di Bartolomeo P., Mazza P., Novitzky N., Peschel C., Lopez J. L. B., Cascon M. J. P., Romeril K. R., Schots R., Brussel H., Koistinen P., Arcese W., Aktan M., Rodeghiero F., Butler A., Pizzuti M., Melpignano A., Carella A. M., Valcarcel D., De Toledo Codina J. S., Galieni P., Bader P., Cavanna L., Sucak G., Broom A. J. M., Garcia P. G., Nicolas-Virelizier E., Rizzoli V., Witz F., Collin M., Ringhoffer M., Kansu E., Martin H., Moraleda J., Pranger D., Greil R., Bazarbachi A., Ozturk M., Fagioli F., Jantunen E., Yeshurun M., Altuntas F., Bassan R., Rohrlich P. -S., Jimenez S., Glaisner S., Vinante O., Clausen J., Lopez-Jimenez J., Theunissen K., Specchia G., Pavone V., Krauter J., Edwards D., Rifon J., Everaus H., Da Prada G. A., Wattad M., Milone G., Walewski J., Thieblemont C., Nasa G. L., Duchosal M., Ferrara F., Devidas A., Delmer A., and Degos L.

Abstract

Even with the availability of targeted drugs, allogeneic hematopoietic cell transplantation (allo-HCT) is the only therapy with curative potential for patients with CLL. Cure can be assessed by comparing long-term survival of patients to the matched general population. Using data from 2589 patients who received allo-HCT between 2000 and 2010, we used landmark analyses and methods from relative survival analysis to calculate excess mortality compared with an age-, sex- and calendar year-matched general population. Estimated event-free survival, overall survival and non-relapse mortality (NRM) 10 years after allo-HCT were 28% (95% confidence interval (CI), 25-31), 35% (95% CI, 32-38) and 40% (95% CI, 37-42), respectively. Patients who passed the 5-year landmark event-free survival (N=394) had a 79% probability (95% CI, 73-85) of surviving the subsequent 5 years without an event. Relapse and NRM contributed equally to treatment failure. Five-year mortality for 45- and 65-year-old reference patients who were event-free at the 5-year landmark was 8% and 47% compared with 3% and 14% in the matched general population, respectively. The prospect of long-term disease-free survival remains an argument to consider allo-HCT for young patients with high-risk CLL, and programs to understand and prevent late causes of failure for long-term survivors are warranted, especially for older patients.

Published
2017

25. The JAK2 V617F mutation identifies a subgroup of MDS patients with isolated deletion 5q and a proliferative bone marrow

Author

Ingram, W, Lea, N C, Cervera, J, Germing, U, Fenaux, P, Cassinat, B, Kiladjian, J J, Varkonyi, J, Antunovic, P, Westwood, N B, Arno, M J, Mohamedali, A, Gaken, J, Kontou, T, Czepulkowski, B H, Twine, N A, Tamaska, J, Csomer, J, Benedek, S, Gattermann, N, Zipperer, E, Giagounidis, A, Garcia-Casado, Z, Sanz, G, and Mufti, G J

Published
2006
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27. Optimized EBMT transplant-specific risk score in myelodysplastic syndromes after allogeneic stem-cell transplantation

Author

Gagelmann, N., Eikema, D.J., Stelljes, M., Beelen, D., de Wreede, L., Mufti, G., Knelange, N.S., Niederwieser, D., Friis, L.S., Ehninger, G., Nagler, A., Yakoub-Agha, I., Meijer, E., Ljungman, P., Maertens, J., Kanz, L., Lopez-Corral, L., Brecht, A., Craddock, C., Finke, J., Cornelissen, J.J., Bernasconi, P., Chevallier, P., Sierra, Jorge, Robin, M., Kröger, N., Universitat Autònoma de Barcelona, Hematology, CCA - Imaging and biomarkers, and CCA - Cancer Treatment and quality of life

Subjects
Male, BLOOD, IMPACT, medicine.medical_treatment, CYTOMEGALOVIRUS, Medizin, Hematopoietic stem cell transplantation, PROPHYLAXIS, Cohort Studies, 0302 clinical medicine, Risk Factors, Medicine, OUTCOMES, Framingham Risk Score, Hazard ratio, Hematopoietic Stem Cell Transplantation, Hematology, Middle Aged, Prognosis, Survival Rate, Female, Life Sciences & Biomedicine, Adult, medicine.medical_specialty, Adolescent, ACUTE MYELOID-LEUKEMIA, Risk Assessment, Article, 03 medical and health sciences, Young Adult, Internal medicine, Humans, Transplantation, Homologous, Survival rate, Aged, Science & Technology, Models, Statistical, business.industry, Proportional hazards model, MUTATIONS, Myelodysplastic syndromes, Nomogram, medicine.disease, Transplantation, MONOSOMAL KARYOTYPE, Nomograms, Myelodysplastic Syndromes, business, SYSTEM, 030215 immunology, Follow-Up Studies
Abstract

The aim of this study was to develop and validate a clinical and transplant-specific prognostic score using data from a large cohort of patients with myelodysplastic syndromes reported to the European Society for Blood and Marrow Transplantation registry. A Cox model was fitted to detect clinical and transplant-related variables prognostic of outcome. Then, cross-validation was performed to evaluate the validity and consistency of the model. Seven independent risk factors for survival were identified: age ≥50 years, matched unrelated donor, Karnofsky Performance Status 1%, and platelet count ≤50 × 109/L prior to transplantation. Incorporating these factors into a four-level risk score yielded hazard ratios for death, with low-risk (score of 0-1) as reference, of 2.02 (95% CI: 1.41-2.90) for the intermediate-risk group (score of 2-3), 3.49 (95% CI: 2.45-4.97) for the high-risk group (score of 4-5), and 5.90 (95% CI: 4.01-8.67) for the very high-risk group (score of >5). The score was predictive of survival, relapse-free survival, relapse, and non-relapse mortality (P

Published
2019

34. Late treatment-related mortality versus competing causes of death after allogeneic transplantation for myelodysplastic syndromes and secondary acute myeloid leukemia

Author

Schetelig, Johannes, primary, de Wreede, Liesbeth C., additional, van Gelder, Michel, additional, Koster, Linda, additional, Finke, Jürgen, additional, Niederwieser, Dietger, additional, Beelen, Dietrich, additional, Mufti, G. J., additional, Platzbecker, Uwe, additional, Ganser, Arnold, additional, Heidenreich, Silke, additional, Maertens, Johan, additional, Socié, Gerard, additional, Brecht, Arne, additional, Stelljes, Matthias, additional, Kobbe, Guido, additional, Volin, Liisa, additional, Nagler, Arnon, additional, Vitek, Antonin, additional, Luft, Thomas, additional, Ljungman, Per, additional, Yakoub-Agha, Ibrahim, additional, Robin, Marie, additional, and Kröger, Nicolaus, additional

Published
2018
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39. Trends in patient outcome over the past two decades following allogeneic stem cell transplantation for acute myeloid leukaemia: anALWP/EBMTanalysis

Author

Canaani, J., primary, Beohou, E., additional, Labopin, M., additional, Ghavamzadeh, A., additional, Beelen, D., additional, Hamladji, R.‐M., additional, Niederwieser, D., additional, Volin, L., additional, Markiewicz, M., additional, Arnold, R., additional, Mufti, G., additional, Ehninger, G., additional, Socié, G., additional, Kröger, N., additional, Mohty, M., additional, and Nagler, A., additional

Published
2018
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48. Validation of the revised IPSS at transplant in patients with myelodysplastic syndrome/transformed acute myelogenous leukemia receiving allogeneic stem cell transplantation: a retrospective analysis of the EBMT chronic malignancies working party

Author

Scheid, C., Wreede, L. de, Biezen, A. van, Koenecke, C., Gohring, G., Volin, L., Maertens, J., Finke, J., Passweg, J., Beelen, D., Cornelissen, J.J., Itala-Remes, M., Chevallier, P., Russell, N., Petersen, E., Milpied, N., Espiga, C.R., Peniket, A., Sierra, J., Mufti, G., Crawley, C., Veelken, J.H., Ljungman, P., Cahn, J.Y., Alessandrino, E.P., Witte, T.J. de, Robin, M., Kroger, N., Scheid, C., Wreede, L. de, Biezen, A. van, Koenecke, C., Gohring, G., Volin, L., Maertens, J., Finke, J., Passweg, J., Beelen, D., Cornelissen, J.J., Itala-Remes, M., Chevallier, P., Russell, N., Petersen, E., Milpied, N., Espiga, C.R., Peniket, A., Sierra, J., Mufti, G., Crawley, C., Veelken, J.H., Ljungman, P., Cahn, J.Y., Alessandrino, E.P., Witte, T.J. de, Robin, M., and Kroger, N.

Abstract

Contains fulltext : 181828.pdf (Publisher’s version ) (Open Access), The International Prognostic Scoring System has been revised (IPSS-R) to predict prognosis of patients with myelodysplastic syndromes at diagnosis. To validate the use of the IPSS-R assessed before transplant rather than at diagnosis we performed a retrospective analysis of the EBMT database. A total of 579 patients had sufficient information available to calculate IPSS-R at transplant. Median overall survival (OS) from transplant was significantly different according to IPSS-R: very low 23.6 months, low 55.0 months, intermediate 19.7 months, high 13.5 months, very high 7.8 months (P<0.001). In a multivariate Cox model the following parameters were significant risk factors for OS: IPSS-R, graft source, age and prior treatment. Median relapse free survival also showed significant differences according to IPSS-R: very low: 23.6 months, low: 24.8 months, intermediate 10.6 months, high 7.9 months, very high 5.5 months (P<0.001). Multivariate risk factors for relapse-free survival (RFS) were: IPSS-R, reduced intensity conditioning, graft source and prior treatment. A trend for an increased relapse incidence was noted for very high risk IPSS-R. We conclude that the IPSS-R at transplant is a useful prognostic score for predicting OS and RFS after transplantation, capturing both disease evolution and response to prior treatment before transplant.

Published
2017

49. Allogeneic haematopoietic stem cell transplant in patients with lower risk myelodysplastic syndrome: a retrospective analysis on behalf of the Chronic Malignancy Working Party of the EBMT

Author

Robin, M., Porcher, R., Zinke-Cerwenka, W., Biezen, A. van, Volin, L., Mufti, G., Craddock, C., Finke, J., Richard, C., Passweg, J., Peniket, A., Maertens, J., Sucak, G., Gedde-Dahl, T., Vitek, A., Nagler, A., Blaise, D., Beelen, D., Maillard, N., Schwerdtfeger, R., Witte, T.J.M. de, Kroger, N., Robin, M., Porcher, R., Zinke-Cerwenka, W., Biezen, A. van, Volin, L., Mufti, G., Craddock, C., Finke, J., Richard, C., Passweg, J., Peniket, A., Maertens, J., Sucak, G., Gedde-Dahl, T., Vitek, A., Nagler, A., Blaise, D., Beelen, D., Maillard, N., Schwerdtfeger, R., Witte, T.J.M. de, and Kroger, N.

Abstract

Item does not contain fulltext, We report a retrospective analysis of 246 myelodysplastic syndrome (MDS) patients in the EBMT (The European Society for Blood and Marrow Transplantation) database who were transplanted for International Prognostic Scoring System (IPSS) low or intermediate-1 disease. The majority of these patients (76%) were reclassified as intermediate or higher risk according to R-IPSS. The 3-year overall survival (OS) and PFS were 58% and 54%, respectively. In a multivariate analysis, adverse risk factors for PFS were marrow blast percentage (hazard ratio (HR): 1.77, P=0.037), donor/recipient CMV serostatus (donor-/recipient+: HR: 2.02, P=0.011) and source of stem cells (marrow and non-CR: HR: 5.72, P<0.0001, marrow and CR: HR: 3.17, P=0.027). Independent risk factors for OS were disease status at time of transplant and the use of in vivo T-cell depletion (TCD). Patients who did not receive TCD and were transplanted from an unrelated donor had worse OS (HR: 4.08, P<0.0001). In conclusion, 'lower' risk MDS patients have better outcome than those with 'higher risk' after haematopoietic stem cell transplant (HSCT). Selecting the right source of stem cells, a CMV-positive donor for CMV-positive patients and using in vivo TCD results in the best outcome in these patients. More studies are needed to evaluate the role of HSCT in these patients as compared with conventional treatment.

Published
2017

50. In the Era of Chemoimmunotherapy, Relapse Post Autologous Stem Cell Transplantation for Follicular Lymphoma Is Associated with Prolonged Overall Survival. an Analysis By the Lymphoma Working Party of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Robinson, S, Finel, H, Boumendil, A, Tilly, H, Salles, G, Corradini, P, Cairoli, R, Pytlik, R, Schouten, H, Leweri, N, Thieblemont, C, Metzner, B, Rambaldi, A, Ringhoffer, M, Lown, R, Rossi, G, Bittenbring, J, Bloor, A, Illes, A, Feugier, P, Turlure, P, Mufti, G, Heinicke, T, Peggs, K, Poire, X, Dreger, P, Montoto, S, Schouten, HC, Lewerin. C, Bittenbring, JT, Robinson, S, Finel, H, Boumendil, A, Tilly, H, Salles, G, Corradini, P, Cairoli, R, Pytlik, R, Schouten, H, Leweri, N, Thieblemont, C, Metzner, B, Rambaldi, A, Ringhoffer, M, Lown, R, Rossi, G, Bittenbring, J, Bloor, A, Illes, A, Feugier, P, Turlure, P, Mufti, G, Heinicke, T, Peggs, K, Poire, X, Dreger, P, Montoto, S, Schouten, HC, Lewerin. C, and Bittenbring, JT

Published
2017

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