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1,226 results on '"Rodeghiero, F."'

8. Addressing and proposing solutions for unmet clinical needs in the management of myeloproliferative neoplasm-associated thrombosis: A consensus-based position paper

Author

Barbui, T, De Stefano, V, Falanga, A, Finazzi, G, Martinelli, I, Rodeghiero, F, Vannucchi, A, Barosi, G, Barbui T, De Stefano V, Falanga A, Finazzi G, Martinelli I, Rodeghiero F, Vannucchi AM, Barosi G, Barbui, T, De Stefano, V, Falanga, A, Finazzi, G, Martinelli, I, Rodeghiero, F, Vannucchi, A, Barosi, G, Barbui T, De Stefano V, Falanga A, Finazzi G, Martinelli I, Rodeghiero F, Vannucchi AM, and Barosi G

Abstract

This article presents the results of a group discussion among an ad hoc constituted Panel of experts aimed at highlighting unmet clinical needs (UCNs) in the management of thrombotic risk and thrombotic events associated with Philadelphia-negative myeloproliferative neoplasms (Ph-neg MPNs). With the Delphi technique, the challenges in Ph-neg MPN-associated thrombosis were selected. The most clinically relevant UCNs resulted in: (1) providing evidence of the benefits and risks of direct oral anticoagulants, (2) providing evidence of the benefits and risks of cytoreduction in patients with splanchnic vein thrombosis without hypercythemia, (3) improving knowledge of the role of the mutated endothelium in the pathogenesis of thrombosis, (4) improving aspirin dosing regimens in essential thrombocythemia, (5) improving antithrombotic management of Ph-neg MPN-associated pregnancy, (6) providing evidence for the optimal duration of anticoagulation for prophylaxis of recurrent VTE, (7) improving knowledge of the association between somatic gene mutations and risk factors for thrombosis, and (8) improving the grading system of thrombosis risk in polycythemia vera. For each of these issues, proposals for advancement in research and clinical practice were addressed. Hopefully, this comprehensive overview will serve to inform the design and implementation of new studies in the field.

Published
2019

16. Expression of 14 von Willebrand factor mutations identified in patients with type 1 von Willebrand disease from the MCMDM-1VWD study

Author

EIKENBOOM, J., HILBERT, L, RIBBA, A.S., HOMMAIS, A., HABART, D., MESSENGER, S., AL-BUHAIRAN, A., GUILLIATT, A., LESTER, W., MAZURIER, C., MEYER, D., FRESSINAUD, E., BUDDE, U., WILL, K., SCHNEPPENHEIM, R., OBSER, T., MARGGRAF, O., ECKERT, E., CASTAMAN, G., RODEGHIERO, F., FEDERICI, A.B., BATLLE, J., GOUDEMAND, J., INGERSLEV, J., LETHAGEN, S., HILL, F., PEAKE, I., and GOODEVE, A.

Published
2009
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19. Detailed von Willebrand factor multimer analysis in patients with von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management of type 1 von Willebrand disease (MCMDM‐1VWD)

Author

BUDDE, U., SCHNEPPENHEIM, R., EIKENBOOM, J., GOODEVE, A., WILL, K., DREWKE, E., CASTAMAN, G., RODEGHIERO, F., FEDERICI, A.B., BATLLE, J., PÉREZ, A., MEYER, D., MAZURIER, C., GOUDEMAND, J., INGERSLEV, J., HABART, D., VORLOVA, Z., HOLMBERG, L., LETHAGEN, S., PASI, J., HILL, F., and PEAKE, I.

Published
2008
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24. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers

Author

Cantoni, S, Carpenedo, M, Mazzucconi, M, De Stefano, V, Carrai, V, Ruggeri, M, Specchia, G, Vianelli, N, Pane, F, Consoli, U, Artoni, A, Zaja, F, D'Adda, M, Visentin, A, Ferrara, F, Barcellini, W, Caramazza, D, Baldacci, E, Rossi, E, Ricco, A, Ciminello, A, Rodeghiero, F, Nichelatti, M, Cairoli, R, Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, Specchia G, Vianelli N, Pane F, Consoli U, Artoni A, Zaja F, D'adda M, Visentin A, Ferrara F, Barcellini W, Caramazza D, Baldacci E, Rossi E, Ricco A, Ciminello A, Rodeghiero F, Nichelatti M, Cairoli R, Cantoni, S, Carpenedo, M, Mazzucconi, M, De Stefano, V, Carrai, V, Ruggeri, M, Specchia, G, Vianelli, N, Pane, F, Consoli, U, Artoni, A, Zaja, F, D'Adda, M, Visentin, A, Ferrara, F, Barcellini, W, Caramazza, D, Baldacci, E, Rossi, E, Ricco, A, Ciminello, A, Rodeghiero, F, Nichelatti, M, Cairoli, R, Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, Specchia G, Vianelli N, Pane F, Consoli U, Artoni A, Zaja F, D'adda M, Visentin A, Ferrara F, Barcellini W, Caramazza D, Baldacci E, Rossi E, Ricco A, Ciminello A, Rodeghiero F, Nichelatti M, and Cairoli R

Abstract

Sequential use of the TPO-RAs romiplostim and eltrombopag in ITP patients failing either agent was retrospectively evaluated to assess efficacy and impact of clinical characteristics on outcome. Patients were grouped into 5 categories: efficacy issues: 1st TPO-RA failure; loss of response; non-efficacy issues: platelet fluctuations; patient's preference; adverse event development. Either one TPO-RA sequence was analyzed at 3 month and at last follow-up. 106/546 patients on TPO-RA underwent switch and 65% achieved, regained or maintained a short- term response independent of switch sequence, gender or age; lower response rates were associated with lines of previous therapy; disease duration lowers probability to respond. Clinically, patients switched for efficacy issue did not differ from those switched for non-efficacy issues. Response was achieved/regained in 57.8% of patients switched for efficacy issues, the lowest response rates were observed in non-responders to 1st TPO-RA; 80% of patients switched for non-efficacy issues maintained a response. Platelet fluctuation resolved in 44.4%. Of the 49 patients evaluable for long-term outcome, 27 were in response on therapy; 16 discontinued the TPO-RA for reasons other than efficacy, while only 6 were non responders. We confirm the efficacy of TPO-RA switch; once achieved, response to the 2nd TPO-RA seems durable.

Published
2018

28. Impact of plasma von Willebrand factor levels in the diagnosis of type 1 von Willebrand disease: results from a multicenter European study (MCMDM‐1VWD)

Author

TOSETTO, A., RODEGHIERO, F., CASTAMAN, G., BERNARDI, M., BERTONCELLO, K., GOODEVE, A., FEDERICI, A.B., BATLLE, J., MEYER, D., MAZURIER, C., GOUDEMAND, J., EIKENBOOM, J., SCHNEPPENHEIM, R., BUDDE, U., INGERSLEV, J., VORLOVA, Z., HABART, D., HOLMBERG, L., LETHAGEN, S., PASI, J., HILL, F., and PEAKE, I.

Published
2007
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29. Management of elderly patients with immune thrombocytopenia: Real-world evidence from 451 patients older than 60 years

Author

Palandri, F., Santoro, C., Carpenedo, M., Cantoni, S., Barcellini, W., Carli, G., Carrai, V., Rossi, E., Rivolti, E., Lucchesi, A., Rotondo, F., Baldacci, E., Auteri, G., Sutto, E., Di Pietro, C., Catani, L., Bartoletti, D., De Stefano, V., Ruggeri, M., Mazzucconi, M. G., Cavo, M., Rodeghiero, F., Vianelli, N., Rossi E. (ORCID:0000-0002-7572-9379), De Stefano V. (ORCID:0000-0002-5178-5827), Palandri, F., Santoro, C., Carpenedo, M., Cantoni, S., Barcellini, W., Carli, G., Carrai, V., Rossi, E., Rivolti, E., Lucchesi, A., Rotondo, F., Baldacci, E., Auteri, G., Sutto, E., Di Pietro, C., Catani, L., Bartoletti, D., De Stefano, V., Ruggeri, M., Mazzucconi, M. G., Cavo, M., Rodeghiero, F., Vianelli, N., Rossi E. (ORCID:0000-0002-7572-9379), and De Stefano V. (ORCID:0000-0002-5178-5827)

Abstract

Introduction: Primary Immune thrombocytopenia (ITP) in the elderly is a major clinical challenge which is increasingly frequent due to global ageing population. Materials and methods: To describe baseline ITP features, management, and outcome, a centralized electronic database was established, including data of 451 patients aged ≥60 years that were treated from 2000 onwards and were observed for ≥1 year (total observation of 2704 patient-years). Results: At ITP diagnosis, median age was 71.1 years (age ≥ 75: 42.8%); 237 (53.9%) patients presented with haemorrhages (grade ≥ 3: 7.5%). First-line therapy included prednisone (82.9%), dexamethasone (14.6%), thrombopoietin-receptor agonists (TRAs, 1.3%), and oral immunosuppressive agents (1.1%). Prednisone starting dose ≥1 mg/kg/d (p = .01) and dexamethasone 40 mg/d (p < .001) were mainly reserved to patients aged 60–74, who were more treated with rituximab (RTX, p = .02) and splenectomy (p = .03) second-line. Overall response rates to first and second-line therapies were 83.8% and 84.5%, respectively, regardless of age and treatment type/dose. A total of 178 haemorrhages in 101 patients (grade ≥ 3: n. 52, 29.2%; intracranial in 6 patients), 49 thromboses in 43 patients (grade ≥ 3: n. 26, 53.1%) and 115 infections in 94 patients (grade ≥ 3: n. 23, 20%) were observed during follow-up. Incidence rates of complications per 100 patient-years were: 4.5 (haemorrhages, grade ≥ 3: 1.7), 1.7 (thromboses, grade ≥ 3: 0.9), and 3.9 (infections, grade ≥ 3: 0.7). TRAs use were associated with reduced risk of bleeding and infections, while cardiovascular risk factors (particularly, diabetes) significantly predicted thromboses and infections. Conclusions: Age-adapted treatment strategies are required in elderly and very elderly patients.

Published
2020

39. Linkage analysis in families diagnosed with type 1 von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management of type 1 VWD

Author

EIKENBOOM, J., VAN MARION, V., PUTTER, H., GOODEVE, A., RODEGHIERO, F., CASTAMAN, G., FEDERICI, A.B., BATLLE, J., MEYER, D., MAZURIER, C., GOUDEMAND, J., SCHNEPPENHEIM, R., BUDDE, U., INGERSLEV, J., VORLOVA, Z., HABART, D., HOLMBERG, L., LETHAGEN, S., PASI, J., HILL, F., and PEAKE, I.

Published
2006
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50. 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

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