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193 results on '"Bornhauser M."'

1. Immunotherapy: DIFFERENT SOURCES OF POLYOMAVIRUS-SPECIFIC T CELLS AS TREATMENT OPTION FOR JC POLYOMAVIRUS-TRIGGERED PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY

Author

Morales, J. Marin, primary, Freund, D., additional, Ruhnke, L., additional, Kretschmann, T., additional, Heidrich, K., additional, Tjalf, Z., additional, Bonifacio, E., additional, von Bonin, M., additional, Stölzel, F., additional, Schetelig, J., additional, Bornhauser, M., additional, and Fuchs, A., additional

Published
2023
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3. Biallelic TET2 mutations confer sensitivity to 5 '-azacitidine in acute myeloid leukemia

Author

Stölzel, F. (Friedrich), Fordham, S.E. (Sarah E.), Nandana, D. (Devi), Lin, W.Y. (Wei-Yu), Blair, H. (Helen), Elstob, C. (Claire), Bell, H.L. (Hayden L.), Mohr, B. (Brigitte), Ruhnke, L. (Leo), Kunadt, D. (Desiree), Dill, C. (Claudia), Allsop, D. (Daniel), Piddock, R. (Rachel), Soura, E.N. (Emmanouela-Niki), Park, C. (Catherine), Fadly, M. (Mohd), Rahman, T. (Thahira), Alharbi, A. (Abrar), Wobus, M. (Manja), Altmann, H. (Heidi), Röllig, C. (Christoph), Wagenführ, L. (Lisa), Jones, G.L. (Gail L.), Menne, T. (Tobias), Jackson, G.H. (Graham H.), Marr, H.J. (Helen J.), Fitzgibbon, J. (Jude), Onel, K. (Kenan), Meggendorfer, M. (Manja), Robinson, A. (Amber), Bziuk, Z. (Zuzanna), Bowes, E. (Emily), Heidenreich, O. (Olaf), Haferlach, T. (Torsten), Villar-Fernández, S. (Sara), Ariceta, B. (Beñat), Ayala, R. (Rosa), Altschuler, S.J. (Steven J.), Wu, L.F. (Lani F.), Prosper, F. (Felipe), Montesinos, P. (Pau), Martínez-López, J. (Joaquín), Bornhauser, M. (Martin), and Allan, J.M. (James M.)

Abstract

Precision medicine can significantly improve outcomes for patients with cancer, but implementation requires comprehensive characterization of tumor cells to identify therapeutically exploitable vulnerabilities. Here, we describe somatic biallelic TET2 mutations in an elderly patient with acute myeloid leukemia (AML) that was chemoresistant to anthracycline and cytarabine but acutely sensitive to 5 '-azacitidine (5 '-Aza) hypomethylating monotherapy, resulting in long-term morphological remission. Given the role of TET2 as a regulator of genomic methylation, we hypothesized that mutant TET2 allele dosage affects response to 5 '-Aza. Using an isogenic cell model system and an orthotopic mouse xenograft, we demonstrate that biallelic TET2 mutations confer sensitivity to 5 '-Aza compared with cells with monoallelic mutations. Our data argue in favor of using hypomethylating agents for chemoresistant disease or as first-line therapy in patients with biallelic TET2-mutated AML and demonstrate the importance of considering mutant allele dosage in the implementation of precision medicine for patients with cancer.

Published
2023

4. Bone marrow central memory and memory stem T-cell exhaustion in AML patients relapsing after HSCT

Author

Noviello, M, Manfredi, F, Ruggiero, E, Perini, T, Oliveira, G, Cortesi, F, De Simone, P, Toffalori, C, Gambacorta, V, Greco, R, Peccatori, J, Casucci, M, Casorati, G, Dellabona, P, Onozawa, M, Teshima, T, Griffioen, M, Halkes, C, Falkenburg, J, Stolzel, F, Altmann, H, Bornhauser, M, Waterhouse, M, Zeiser, R, Finke, J, Cieri, N, Bondanza, A, Vago, L, Ciceri, F, Bonini, C, Noviello M., Manfredi F., Ruggiero E., Perini T., Oliveira G., Cortesi F., De Simone P., Toffalori C., Gambacorta V., Greco R., Peccatori J., Casucci M., Casorati G., Dellabona P., Onozawa M., Teshima T., Griffioen M., Halkes C. J. M., Falkenburg J. H. F., Stolzel F., Altmann H., Bornhauser M., Waterhouse M., Zeiser R., Finke J., Cieri N., Bondanza A., Vago L., Ciceri F., Bonini C., Noviello, M, Manfredi, F, Ruggiero, E, Perini, T, Oliveira, G, Cortesi, F, De Simone, P, Toffalori, C, Gambacorta, V, Greco, R, Peccatori, J, Casucci, M, Casorati, G, Dellabona, P, Onozawa, M, Teshima, T, Griffioen, M, Halkes, C, Falkenburg, J, Stolzel, F, Altmann, H, Bornhauser, M, Waterhouse, M, Zeiser, R, Finke, J, Cieri, N, Bondanza, A, Vago, L, Ciceri, F, Bonini, C, Noviello M., Manfredi F., Ruggiero E., Perini T., Oliveira G., Cortesi F., De Simone P., Toffalori C., Gambacorta V., Greco R., Peccatori J., Casucci M., Casorati G., Dellabona P., Onozawa M., Teshima T., Griffioen M., Halkes C. J. M., Falkenburg J. H. F., Stolzel F., Altmann H., Bornhauser M., Waterhouse M., Zeiser R., Finke J., Cieri N., Bondanza A., Vago L., Ciceri F., and Bonini C.

Abstract

The major cause of death after allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for acute myeloid leukemia (AML) is disease relapse. We investigated the expression of Inhibitory Receptors (IR; PD-1/CTLA-4/TIM-3/LAG-3/2B4/KLRG1/GITR) on T cells infiltrating the bone marrow (BM) of 32 AML patients relapsing (median 251 days) or maintaining complete remission (CR; median 1 year) after HSCT. A higher proportion of early-differentiated Memory Stem (T SCM ) and Central Memory BM-T cells express multiple IR in relapsing patients than in CR patients. Exhausted BM-T cells at relapse display a restricted TCR repertoire, impaired effector functions and leukemia-reactive specificities. In 57 patients, early detection of severely exhausted (PD-1 + Eomes + T-bet − ) BM-T SCM predicts relapse. Accordingly, leukemia-specific T cells in patients prone to relapse display exhaustion markers, absent in patients maintaining long-term CR. These results highlight a wide, though reversible, immunological dysfunction in the BM of AML patients relapsing after HSCT and suggest new therapeutic opportunities for the disease.

Published
2019

5. Total body irradiation plus fludarabine compared to busulfan plus fludarabine as 'reduced-toxicity conditioning' for patients with acute myeloid leukemia treated with allogeneic hematopoietic cell transplantation in first complete remission: a study by the Acute Leukemia Working Party of the EBMT

Author

Giebel, S, Labopin, M, Sobczyk-Kruszelnicka, M, Stelljes, M, Byrne, JL, Fegueux, N, Beelen, DW, Rovira, M, Spyridonidis, A, Blaise, D, Bornhauser, M, Karadogan, I, Savani, BN, Nagler, A, Mohty, M, Martin, S, Chevallier, P, Neubauer, A, Damaj, G, Koc, Y, Ganser, A, Collin, M, Yakoub-Agha, I, Ozdogu, H, Araujo, MC, Itala-Remes, M, Orchard, K, Isaksson, C, Bethge, W, Martin, H, Aljurf, M, Faber, E, Caballero, D, Zak, P, Leleu, X, Bay, JO, Rohrlich, PS, Kroger, N, Huynh, A, Schafer-Eckart, K, Milpied, N, Lenhoff, S, Ho, A, Lopez, JLB, Mordini, N, Lioure, B, Halaburda, K, Olivieri, A, Gedde-Dahl, T, Protheroe, R, Tischer, J, Klammer, M, Clausen, J, Potter, V, Ladetto, M, Tilly, H, Deconinck, E, Brecht, A, Muller, LP, Heinicke, T, Carrete, JPT, Bazarbachi, A, Remenyi, P, Rubio, MT, Fanin, R, Perez-Simon, JA, Niels, M, Diez-Martin, JL, Arat, M, Hermine, O, Socie, G, Cornelissen, JJ, Santarone, S, Guyotat, D, Bulabois, CE, Bernasconi, P, Johansson, JE, Vrhovac, R, Greinix, H, Lorenzo, JLL, Apte, S, Craddock, C, Kobbe, G, Zahrani, MA, Dreger, P, Lange, A, Tbakhi, A, Meijer, E, Llamas, CV, Santasusana, JMR, Corradini, P, Benedetti, F, Rambaldi, A, Gandemer, V, Malfuson, JV, Kaare, A, Risitano, A, Petrini, M, Selleri, C, and Wu, DP

Abstract

The optimal conditioning for patients with acute myeloid leukemia in first complete remission treated with allogeneic hematopoietic cell transplantation (allo-HCT) has not been defined so far. In this retrospective study, we compared two "reduced-toxicity" regimens: intravenous busulfan at a total dose of 9.6 mg/kg (3 days) + fludarabine (Bu3/Flu) and total body irradiation at a dose of 8 Gy + fludarabine (TBI8Gy/Flu). In the entire study cohort (n = 518), the probabilities of overall survival (OS), leukemia-free survival (LFS), relapse and non-relapse mortality (NRM) at 2 years for Bu3/Flu and TBI8Gy/Flu were 62% vs. 72.5% (p = 0.051), 59.5% vs. 65% (p = 0.15), 30% vs. 20% (p = 0.01), and 10% vs. 14% (p = 0.18), respectively. In multivariate model for patients

Published
2021

10. Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: A retrospective analysis on behalf of EBMT group

Author

Giardino, S., de Latour, R. P., Aljurf, M., Eikema, D. -J., Bosman, P., Bertrand, Y., Tbakhi, A., Holter, W., Bornhauser, M., Rossig, C., Burkhardt, B., Zecca, M., Afanasyev, B., Michel, G., Ganser, A., Alseraihy, A., Ayas, M., Uckan-Cetinkaya, D., Bruno, B., Patrick, K., Bader, P., Itala-Remes, M., Rocha, V., Jubert, C., Diaz, M. A., Shaw, P. J., Junior, L. G. D., Locatelli, Franco, Kroger, N., Faraci, M., Pierri, F., Lanino, E., Miano, M., Risitano, A., Robin, M., Dufour, C., Locatelli F. (ORCID:0000-0002-7976-3654), Giardino, S., de Latour, R. P., Aljurf, M., Eikema, D. -J., Bosman, P., Bertrand, Y., Tbakhi, A., Holter, W., Bornhauser, M., Rossig, C., Burkhardt, B., Zecca, M., Afanasyev, B., Michel, G., Ganser, A., Alseraihy, A., Ayas, M., Uckan-Cetinkaya, D., Bruno, B., Patrick, K., Bader, P., Itala-Remes, M., Rocha, V., Jubert, C., Diaz, M. A., Shaw, P. J., Junior, L. G. D., Locatelli, Franco, Kroger, N., Faraci, M., Pierri, F., Lanino, E., Miano, M., Risitano, A., Robin, M., Dufour, C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is curative for bone marrow failure in patients with Fanconi anemia (FA), but the presence of a malignant transformation is associated with a poor prognosis and the management of these patients is still challenging. We analyzed outcome of 74 FA patients with a diagnosis of myelodysplastic syndrome (n = 35), acute leukemia (n = 35) or with cytogenetic abnormalities (n = 4), who underwent allo-HSCT from 1999 to 2016 in EBMT network. Type of diagnosis, pre-HSCT cytoreductive therapies and related toxicities, disease status pre-HSCT, donor type, and conditioning regimen were considered as main variables potentially influencing outcome. The 5-year OS and EFS were 42% (30-53%) and 39% (27-51%), respectively. Patients transplanted in CR showed better OS compared with those transplanted in presence of an active malignant disease (OS:71%[48-95] vs 37% [24-50],P =.04), while none of the other variables considered had an impact. Twenty-two patients received pre-HSCT cytoreduction and 9/22 showed a grade 3-4 toxicity, without any lethal event or negative influence on survival after HSCT(OS:toxicity pre-HSCT 48% [20-75%] vs no-toxicity 51% [25-78%],P =.98). The cumulative incidence of day-100 grade II-IV a-GvHD and of 5-year c-GvHD were 38% (26-50%) and 40% (28-52%). Non-relapse-related mortality and incidence of relapse at 5-years were 40% (29-52%) and 21% (11-30%) respectively, without any significant impact of the tested variables. Causes of death were transplant-related events in most patients (34 out of the 42 deaths, 81%). This analysis confirms the poor outcome of transformed FA patients and identifies the importance of achieving CR pre-HSCT, suggesting that, in a newly diagnosed transformed FA patient, a cytoreductive approach pre-HSCT should be considered if a donor have been secured.

Published
2020

12. Randomized comparison of prophylactic and minimal residual disease-triggered imatinib after allogeneic stem cell transplantation for BCR-ABL1-positive acute lymphoblastic leukemia

Author

Pfeifer, H, Wassmann, B, Bethge, W, Dengler, J, Bornhauser, M, Stadler, M, Beelen, D, Vucinic, V, Burmeister, T, Stelljes, M, Faul, C, Dreger, P, Kiani, A, Schafer-Eckart, K, Schwerdtfeger, R, Lange, E, Kubuschok, B, Horst, H A, Gramatzki, M, Brück, P, Serve, H, Hoelzer, D, Gökbuget, N, and Ottmann, O G

Published
2013
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14. 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

17. Reduced intensity conditioning regimens including alkylating chemotherapy do not alter survival outcomes after allogeneic hematopoietic cell transplantation in chronic lymphocytic leukemia compared to low-intensity non-myeloablative conditioning

Author

Andersen, N.S., Bornhauser, M., Gramatzki, M., Dreger, P., Vitek, A., Karas, M., Michallet, M., Moreno, C., Gelder, M. van, Henseler, A., Wreede, L.C. de, Schonland, S., Kroger, N., Schetelig, J., CLL subcomm Chronic, Interne Geneeskunde, MUMC+: MA Hematologie (9), and RS: GROW - R3 - Innovative Cancer Diagnostics & Therapy

Subjects
0301 basic medicine, Oncology, Male, Cancer Research, Transplantation Conditioning, BLOOD, medicine.medical_treatment, Chronic lymphocytic leukemia, Graft vs Host Disease, refractory chronic lymphocytic leukemia, Hematopoietic stem cell transplantation, RETROSPECTIVE ANALYSIS, 0302 clinical medicine, hemic and lymphatic diseases, TOTAL-BODY IRRADIATION, Antineoplastic Combined Chemotherapy Protocols, LYMPHOMA, Medicine, FAILURE, Nonmyeloablative, Melphalan, Hematology, Incidence, Remission Induction, Hematopoietic Stem Cell Transplantation, General Medicine, Middle Aged, Prognosis, Combined Modality Therapy, EUROPEAN-SOCIETY, Survival Rate, 030220 oncology & carcinogenesis, Reduced Intensity Conditioning, Allogeneic hematopoietic stem cell transplantation, Female, FLUDARABINE, Adult, medicine.medical_specialty, Relapsed, 03 medical and health sciences, Young Adult, reduced intensity conditioning, Internal medicine, Humans, Transplantation, Homologous, MARROW-TRANSPLANTATION, Busulfan, Cyclophosphamide, Aged, Retrospective Studies, Chemotherapy, business.industry, medicine.disease, Leukemia, Lymphocytic, Chronic, B-Cell, Intensity (physics), Transplantation, 030104 developmental biology, Conditioning, business, FOLLOW-UP, CLL, Follow-Up Studies
Abstract

Purpose The optimal dose intensity for conditioning prior to allogeneic hematopoietic stem cell transplantation (alloHSCT) for chronic lymphocytic leukemia (CLL) is unknown. Methods We retrospectively compared outcomes of patients who received a first alloHCST after non-myeloablative (NMA) and reduced intensity conditioning (RIC). Data of 432 patients with a median age of 55 years were included, of which 86 patients underwent NMA and 346 RIC. Results The median follow-up after alloHSCT was 4.3 years. Compared to the RIC group, more NMA patients had purine-analog-sensitive disease, were in complete remission and received matched related donor transplantation. After RIC, the probabilities for 5-year OS, EFS, CIR, and NRM were 46%, 38%, 28%, and 35% and after NMA the respective probabilities were 52%, 43%, 25%, and 32%. In multivariate analysis, remission status prior to conditioning but not RIC versus NMA conditioning had a significant impact on CIR, EFS, and OS. Conclusion Presumed higher anti-leukemic activity of RIC versus NMA conditioning did not translate into better outcomes after alloHSCT, but better remission status prior to conditioning did. Effective pathway inhibitor-based salvage therapies combined with NMA conditioning might thus represent the most attractive contemporary approach for alloHSCT for patients with CLL.

Published
2019

18. Impact of primary disease on outcome after allogeneic stem cell transplantation for transformed secondary acute leukaemia

Author

Kroger, N., Eikema, D.J., Koster, L., Beelen, D., Wreede, L.C. de, Finke, J., Koenecke, C., Niederwieser, D., Bornhauser, M., Schoenland, S., Potter, V., Wolschke, C., Maertens, J., Theobald, M., Kobbe, G., Itala-Remes, M., Wulf, G., Kahls, P., Forcade, E., Greinix, H., Masszi, T., Yakoub-Agha, I., Chalandon, Y., Robin, M., and European Soc Blood Marrow Transpla

Subjects
Male, Chronic myelomonocytic leukaemia, myeloproliferative neoplasm, Medizin, Myeloproliferative neoplasm, Graft vs Host Disease, Gastroenterology, 0302 clinical medicine, allogeneic stem cell transplantation, hemic and lymphatic diseases, MDS, Medicine, Cumulative incidence, ddc:616, Hematopoietic Stem Cell Transplantation, Leukemia, Myelomonocytic, Chronic, Hematology, Middle Aged, Haematopoiesis, Leukemia, Myeloid, Acute, 030220 oncology & carcinogenesis, Acute Disease, Disease Progression, Female, Stem cell, chronic myelomonocytic leukaemia, Adult, medicine.medical_specialty, Adolescent, Transplantation, Autologous, Disease-Free Survival, 03 medical and health sciences, Young Adult, Median follow-up, Statistical significance, Internal medicine, Humans, Aged, Secondary acute myeloid leukaemia, Myeloproliferative Disorders, secondary acute myeloid leukaemia, business.industry, Myelodysplastic syndromes, medicine.disease, Allogeneic stem cell transplantation, Transplantation, Myelodysplastic Syndromes, Chronic Disease, Neoplasm Recurrence, Local, business, 030215 immunology
Abstract

Myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN) and chronic myelomonocytic leukaemia (CMML) can progress to secondary acute myeloid leukaemia (sAML). We compared the outcome of 4214 sAML patients who received allogeneic haematopoietic stem cell transplantation (allo-HSCT) from an unrelated (62%) or human leucocyte antigen (HLA)-identical sibling donor (38%) according the underlying disease: MDS (n = 3541), CMML (n = 251) or MPN (n = 422). After a median follow up of 46·5 months, the estimated 3-year progression-free (PFS) and overall survival (OS) for the entire group was 36% (34-37%) and 41% (40-43%), respectively. The cumulative incidence of relapse and non-relapse mortality (NRM) was 37% (35-39%) and 27% (26-29%), respectively. In a multivariable analysis for OS, besides age (P

Published
2019

27. Risk factors for treatment failure after allogeneic transplantation of patients with CLL : A report from the European Society for Blood and Marrow Transplantation

Author

Schetelig, J., Wreede, L.C. de, Gelder, M. van, Andersen, N.S., Moreno, C., Vitek, A., Karas, M., Michallet, M., Machaczka, M., Gramatzki, M., Beelen, D., Finke, J., Delgado, J., Volin, L., Passweg, J., Dreger, P., Henseler, A., Biezen, A. van, Bornhauser, M., Schonland, S.O., Kroger, N., CLL Subcomm, Chronic Malignancies Working Party, Interne Geneeskunde, MUMC+: MA Hematologie (9), and RS: GROW - R3 - Innovative Cancer Diagnostics & Therapy

Subjects
Oncology, Male, medicine.medical_treatment, Chronic lymphocytic leukemia, Medizin, Blood Donors, DISEASE, Targeted therapy, RETROSPECTIVE ANALYSIS, 0302 clinical medicine, Risk Factors, hemic and lymphatic diseases, IDELALISIB, VENETOCLAX, Treatment Failure, IBRUTINIB, Age Factors, Hematopoietic Stem Cell Transplantation, Hematology, Middle Aged, Prognosis, 3. Good health, 030220 oncology & carcinogenesis, Cohort, SURVIVAL, Female, Adult, medicine.medical_specialty, Allogeneic transplantation, Risk Assessment, MECHANISMS, 03 medical and health sciences, Young Adult, Sex Factors, Internal medicine, medicine, Humans, Transplantation, Homologous, Aged, Transplantation, Performance status, Proportional hazards model, business.industry, CHRONIC LYMPHOCYTIC-LEUKEMIA, STEM-CELL TRANSPLANTATION, medicine.disease, Leukemia, Lymphocytic, Chronic, B-Cell, Survival Analysis, Surgery, Graft-versus-host disease, business, FOLLOW-UP, 030215 immunology
Abstract

For young patients with high-risk CLL, BTK-/PI3K-inhibitors or allogeneic stem cell transplantation (alloHCT) are considered. Patients with a low risk of non-relapse mortality (NRM) but a high risk of failure of targeted therapy may benefit most from alloHCT. We performed Cox regression analyses to identify risk factors for 2-year NRM and 5-year event-free survival (using EFS as a surrogate for long-term disease control) in a large, updated EBMT registry cohort (n = 694). For the whole cohort, 2-year NRM was 28% and 5-year EFS 37%. Higher age, lower performance status, unrelated donor type and unfavorable sex-mismatch had a significant adverse impact on 2-year NRM. Two-year NRM was calculated for good-and poor-risk reference patients. Predicted 2-year-NRM was 11 and 12% for male and female good-risk patients compared with 42 and 33% for male and female poor-risk patients. For 5-year EFS, age, performance status, prior autologous HCT, remission status and sex-mismatch had a significant impact, whereas del(17p) did not. The model-based prediction of 5-year EFS was 55% and 64%, respectively, for male and female good-risk patients. Good-risk transplant candidates with high-risk CLL and limited prognosis either on or after failure of targeted therapy should still be considered for alloHCT.

Published
2017

28. Minimum Information about T Regulatory Cells: A Step toward Reproducibility and Standardization

Author

Fuchs, A., Gliwinski, M., Grageda, N., Spiering, R., Abbas, A.K., Appel, S., Bacchetta, R., Battaglia, M., Berglund, D., Blazar, B., Bluestone, J.A., Bornhauser, M., Brinke, A. Ten, Brusko, T.M., Cools, N., Cuturi, M.C., Geissler, E., Giannoukakis, N., Golab, K., Hafler, D.A., Ham, S.M. van, Hester, J., Hippen, K., Ianni, M. Di, Ilic, N., Isaacs, J., Issa, F., Iwaszkiewicz-Grzes, D., Jaeckel, E., Joosten, I., Klatzmann, D., Koenen, H., Kooten, C. van, Korsgren, O., Kretschmer, K., Levings, M., Marek-Trzonkowska, N.M., Martinez-Llordella, M., Miljkovic, D., Mills, K.H., Miranda, J.P., Piccirillo, C.A., Putnam, A.L., Ritter, T., Roncarolo, M.G., Sakaguchi, S., Sanchez-Ramon, S., Sawitzki, B., Sofronic-Milosavljevic, L., Sykes, M., Tang, Q., Vives-Pi, M., Waldmann, H., Witkowski, P., Wood, K.J., Gregori, S., Hilkens, C.M., Lombardi, G., Lord, P., Martinez-Caceres, E.M., Trzonkowski, P., Fuchs, A., Gliwinski, M., Grageda, N., Spiering, R., Abbas, A.K., Appel, S., Bacchetta, R., Battaglia, M., Berglund, D., Blazar, B., Bluestone, J.A., Bornhauser, M., Brinke, A. Ten, Brusko, T.M., Cools, N., Cuturi, M.C., Geissler, E., Giannoukakis, N., Golab, K., Hafler, D.A., Ham, S.M. van, Hester, J., Hippen, K., Ianni, M. Di, Ilic, N., Isaacs, J., Issa, F., Iwaszkiewicz-Grzes, D., Jaeckel, E., Joosten, I., Klatzmann, D., Koenen, H., Kooten, C. van, Korsgren, O., Kretschmer, K., Levings, M., Marek-Trzonkowska, N.M., Martinez-Llordella, M., Miljkovic, D., Mills, K.H., Miranda, J.P., Piccirillo, C.A., Putnam, A.L., Ritter, T., Roncarolo, M.G., Sakaguchi, S., Sanchez-Ramon, S., Sawitzki, B., Sofronic-Milosavljevic, L., Sykes, M., Tang, Q., Vives-Pi, M., Waldmann, H., Witkowski, P., Wood, K.J., Gregori, S., Hilkens, C.M., Lombardi, G., Lord, P., Martinez-Caceres, E.M., and Trzonkowski, P.

Abstract

Contains fulltext : 191000.pdf (publisher's version ) (Open Access), Cellular therapies with CD4+ T regulatory cells (Tregs) hold promise of efficacious treatment for the variety of autoimmune and allergic diseases as well as posttransplant complications. Nevertheless, current manufacturing of Tregs as a cellular medicinal product varies between different laboratories, which in turn hampers precise comparisons of the results between the studies performed. While the number of clinical trials testing Tregs is already substantial, it seems to be crucial to provide some standardized characteristics of Treg products in order to minimize the problem. We have previously developed reporting guidelines called minimum information about tolerogenic antigen-presenting cells, which allows the comparison between different preparations of tolerance-inducing antigen-presenting cells. Having this experience, here we describe another minimum information about Tregs (MITREG). It is important to note that MITREG does not dictate how investigators should generate or characterize Tregs, but it does require investigators to report their Treg data in a consistent and transparent manner. We hope this will, therefore, be a useful tool facilitating standardized reporting on the manufacturing of Tregs, either for research purposes or for clinical application. This way MITREG might also be an important step toward more standardized and reproducible testing of the Tregs preparations in clinical applications.

Published
2017

29. Centre characteristics and procedure-related factors have an impact on outcomes of allogeneic transplantation for patients with CLL: a retrospective analysis from the European Society for Blood and Marrow Transplantation (EBMT)

Author

Schetelig, J., Wreede, L.C. de, Andersen, N.S., Moreno, C., Gelder, M. van, Vitek, A., Karas, M., Michallet, M., Machaczka, M., Gramatzki, M., Beelen, D., Finke, J., Delgado, J., Volin, L., Passweg, J., Dreger, P., Schaap, N.P., Wagner, E., Henseler, A., Biezen, A. van, Bornhauser, M., Iacobelli, S., Putter, H., Schonland, S.O., Kroger, N., Schetelig, J., Wreede, L.C. de, Andersen, N.S., Moreno, C., Gelder, M. van, Vitek, A., Karas, M., Michallet, M., Machaczka, M., Gramatzki, M., Beelen, D., Finke, J., Delgado, J., Volin, L., Passweg, J., Dreger, P., Schaap, N.P., Wagner, E., Henseler, A., Biezen, A. van, Bornhauser, M., Iacobelli, S., Putter, H., Schonland, S.O., and Kroger, N.

Abstract

Contains fulltext : 177313.pdf (publisher's version ) (Open Access), The best approach for allogeneic haematopoietic stem cell transplantations (alloHCT) in patients with chronic lymphocytic leukaemia (CLL) is unknown. We therefore analysed the impact of procedure- and centre-related factors on 5-year event-free survival (EFS) in a large retrospective study. Data of 684 CLL patients who received a first alloHCT between 2000 and 2011 were analysed by multivariable Cox proportional hazards models with a frailty component to investigate unexplained centre heterogeneity. Five-year EFS of the whole cohort was 37% (95% confidence interval [CI], 34-42%). Larger numbers of CLL alloHCTs (hazard ratio [HR] 0.96, P = 0.002), certification of quality management (HR 0.7, P = 0.045) and a higher gross national income per capita (HR 0.4, P = 0.04) improved EFS. In vivo T-cell depletion (TCD) with alemtuzumab compared to no TCD (HR 1.5, P = 0.03), and a female donor compared to a male donor for a male patient (HR 1.4, P = 0.02) had a negative impact on EFS, but not non-myeloablative versus more intensive conditioning. After correcting for patient-, procedure- and centre-characteristics, significant variation in centre outcomes persisted. In conclusion, further research on the impact of centre and procedural characteristics is warranted. Non-myeloablative conditioning appears to be the preferable approach for patients with CLL.

Published
2017

30. Impact of procedure related factors and center effects on outcome of CLL transplantations

Author

Schetelig, J., Wreede, L. de, Gelder, M. van, Andersen, N.S., Moreno, C., Vitek, A., Karas, M., Michallet, M., Machaczka, M., Gramatzki, M., Beelen, D., Finke, J., Delgado, J., Volin, L., Passweg, J., Dreger, P., Henseler, A., Biezen, A. van, Bornhauser, M., Schonland, S.O., Kroger, N., CLL Subcomm, and Chronic Malignancies Working Party

Published
2016

34. Comparison of conditioning regimens of various intensities for allogeneic hematopoietic SCT using HLA-identical sibling donors in AML and MDS with <10% BM blasts: a report from EBMT

Author

Martino, R., Wreede, L. de, Fiocco, M., Biezen, A. van, Borne, P.A. von dem, Hamladji, R.M., Volin, L., Bornhauser, M., Robin, M., Rocha, V., Witte, T. de, Kroger, N., Mohty, M., and European Grp Blood Marrow

Subjects
Oncology, Adult, Male, medicine.medical_specialty, Pediatrics, Myeloid, Transplantation Conditioning, Adolescent, Anemia, medicine.medical_treatment, Hematopoietic stem cell transplantation, Disease-Free Survival, AML, Risk Factors, Internal medicine, hemic and lymphatic diseases, medicine, MDS, Humans, Prospective cohort study, Survival rate, Aged, Transplantation, Anemia, Refractory, with Excess of Blasts, business.industry, Siblings, Hematopoietic Stem Cell Transplantation, Translational research Immune Regulation [ONCOL 3], Retrospective cohort study, Hematology, Middle Aged, medicine.disease, Allografts, Tissue Donors, allogeneic transplantation, Survival Rate, Leukemia, Leukemia, Myeloid, Acute, medicine.anatomical_structure, Female, business, reduced-intensity conditioning, Follow-Up Studies
Abstract

Contains fulltext : 118183.pdf (Publisher’s version ) (Closed access) In this multicenter retrospective study, the long-term outcomes of 878 adults with AML and refractory anemia with excess blasts (RAEB) with BM blasts

Published
2013

35. 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

36. 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

37. CD34 POSITIVE SELECTION OF CRYOPRESERVED PERIPHERAL BLOOD STEM CELLS (PBSC) FOR AUTOLOGOUS TRANSPLANTATION IN A PATIENT WITH WEGENER'S GRANULOMATOSIS

Author

Bornhauser, M., Paul, K.-D., Gahr, M., Nuesslein, H.-G., Meurer, M., Oppermann, J., Pfeiffer, C., Luthke, K., Schroder, H.-E., and Ehninger, G.

Subjects
Wegener's granulomatosis -- Care and treatment, Stem cells -- Transplantation, Health, Care and treatment
Abstract

Background: There is some debate about the role of T and B cell depletion for autologous stem cell transplantation in patients with autoimmune diseases. No clinical study has yet proved [...]

Published
2001

38. The outcome of patients with Philadelphia chromosome negative/bc-abl negative chronic myelogenous leukaemia after allogeneic stem cell transplantation: a restrospective study of the CML subcommittee of the Chronic Leukemia Working Party (EBMT)

Author

Thomssen, H., Van Biezen, A., Brand, R., Russell, N., Apperley, Jane F., Foa, R., Schots, Henri, Koza, V, Bornhauser, M., Olavarria, E., De Witte, T., and Hematology

Subjects
allogeneic stem cell transplantation, chronic myelogenous leukaemia
Published
2010

39. Allogeneic stem cell transplantation for myelofibrosis with leukemic transformation: a study from the Myeloproliferative Neoplasm Subcommittee of the CMWP of the European Group for Blood and Marrow Transplantation

Author

Alchalby, H., Zabelina, T., Stubig, T., Biezen, A. van, Bornhauser, M., Bartolomeo, P. Di, Beelen, D., Cahn, J.Y., Dreger, P., Schroyens, W., Witte, T.J. de, Olavarria, E., Kroger, N., Marrow, T., Alchalby, H., Zabelina, T., Stubig, T., Biezen, A. van, Bornhauser, M., Bartolomeo, P. Di, Beelen, D., Cahn, J.Y., Dreger, P., Schroyens, W., Witte, T.J. de, Olavarria, E., Kroger, N., and Marrow, T.

Abstract

Item does not contain fulltext, Transformed acute myeloid leukemia in myelofibrosis results in a median survival of less than 5 months. We identified 46 of 1048 myelofibrosis patients in the European Group for Blood and Marrow Transplantation registry who received allogeneic stem cell transplantation for acute leukemia evolving from myelofibrosis. The cumulative incidence of treatment-related mortality at 1 year was 28% (95% confidence interval, 14 to 42) and of relapse at 3 years was 47% (95% confidence interval, 31 to 63). The 3-year progression-free (PFS) and overall survival (OS) rates were 26% and 33%, respectively. The only significant factor for survival was complete remission versus no complete remission before transplantation (69% versus 22%, P = .008); however, complete remission was achieved only in 8 patients. Allogeneic stem cell transplantation can cure myelofibrosis patients transformed to leukemia.

Published
2014

40. Hematopoietic Stem-Cell Transplantation for Advanced Systemic Mastocytosis

Author

Ustun, C, Reiter, A, Scott, Bl, Nakamura, R, Damaj, G, Kreil, S, Shanley, R, Hogan, Wj, Perales, M, Shore, T, Baurmann, H, Stuart, R, Gruhn, B, Doubek, M, Hsu, Jw, Tholouli, E, Gromke, T, Godley, La, Pagano, Livio, Gilman, A, Wagner, Em, Shwayder, T, Bornhauser, M, Papadopoulos, Eb, Bohm, A, Vercellotti, G, Van Lint, Mt, Schmid, C, Rabitsch, W, Pullarkat, V, Legrand, F, Yakoub Agha, I, Saber, W, Barrett, J, Hermine, O, Hagglund, H, Sperr, Wr, Popat, U, Alyea, Ep, Devine, S, Deeg, Hj, Weisdorf, D, Akin, C, Valent, P., Pagano, Livio (ORCID:0000-0001-8287-928X), Ustun, C, Reiter, A, Scott, Bl, Nakamura, R, Damaj, G, Kreil, S, Shanley, R, Hogan, Wj, Perales, M, Shore, T, Baurmann, H, Stuart, R, Gruhn, B, Doubek, M, Hsu, Jw, Tholouli, E, Gromke, T, Godley, La, Pagano, Livio, Gilman, A, Wagner, Em, Shwayder, T, Bornhauser, M, Papadopoulos, Eb, Bohm, A, Vercellotti, G, Van Lint, Mt, Schmid, C, Rabitsch, W, Pullarkat, V, Legrand, F, Yakoub Agha, I, Saber, W, Barrett, J, Hermine, O, Hagglund, H, Sperr, Wr, Popat, U, Alyea, Ep, Devine, S, Deeg, Hj, Weisdorf, D, Akin, C, Valent, P., and Pagano, Livio (ORCID:0000-0001-8287-928X)

Abstract

PURPOSE: Advanced systemic mastocytosis (SM), a fatal hematopoietic malignancy characterized by drug resistance, has no standard therapy. The effectiveness of allogeneic hematopoietic stem-cell transplantation (alloHCT) in SM remains unknown. PATIENTS AND METHODS: In a global effort to define the value of HCT in SM, 57 patients with the following subtypes of SM were evaluated: SM associated with clonal hematologic non-mast cell disorders (SM-AHNMD; n = 38), mast cell leukemia (MCL; n = 12), and aggressive SM (ASM; n = 7). Median age of patients was 46 years (range, 11 to 67 years). Donors were HLA-identical (n = 34), unrelated (n = 17), umbilical cord blood (n = 2), HLA-haploidentical (n = 1), or unknown (n = 3). Thirty-six patients received myeloablative conditioning (MAC), and 21 patients received reduced-intensity conditioning (RIC). RESULTS: Responses in SM were observed in 40 patients (70%), with complete remission in 16 patients (28%). Twelve patients (21%) had stable disease, and five patients (9%) had primary refractory disease. Overall survival (OS) at 3 years was 57% for all patients, 74% for patients with SM-AHNMD, 43% for those with ASM, and 17% for those with MCL. The strongest risk factor for poor OS was MCL. Survival was also lower in patients receiving RIC compared with MAC and in patients having progression compared with patients having stable disease or response. CONCLUSION: AlloHCT was associated with long-term survival in patients with advanced SM. Although alloHCT may be considered as a viable and potentially curative therapeutic option for advanced SM in the meantime, given that this is a retrospective analysis with no control group, the definitive role of alloHCT will need to be determined by a prospective trial.

Published
2014

42. Induction of short-term remission with single agent eltrombopag in refractory nucleophosmin-1-mutated acute myeloid leukemia

Author

Platzbecker, U., primary, Sockel, K., additional, Schonefeldt, C., additional, Nowak, D., additional, Helas, S., additional, Rollig, C., additional, Mossner, M., additional, Jann, J. C., additional, Ehninger, G., additional, Hofmann, W.-K., additional, Bornhauser, M., additional, Thiede, C., additional, and Wermke, M., additional

Published
2014
Full Text
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43. Proinflammatory human 6-sulfo LacNAc-positive dendritic cells accumulate in intestinal acute graft-versus-host disease

Author

Sommer, U., primary, Larsson, B., additional, Tuve, S., additional, Wehner, R., additional, Zimmermann, N., additional, Kramer, M., additional, Kloss, A., additional, Gunther, C., additional, Babatz, J., additional, Schmelz, R., additional, Bruckner, S., additional, Schetelig, J., additional, Bornhauser, M., additional, Schakel, K., additional, Bachmann, M. P., additional, Aust, D., additional, Baretton, G., additional, and Schmitz, M., additional

Published
2014
Full Text
View/download PDF

44. Comparison of allogeneic stem cell transplantation and non-transplant approaches in elderly patients with advanced myelodysplastic syndrome: optimal statistical approaches and a critical appraisal of clinical results using non-randomized data

Author

Brand, R., Putter, H., Biezen, A. van, Niederwieser, D., Martino, R., Mufti, G., Onida, F., Symeonidis, A., Schmid, C., Garderet, L., Robin, M., Gelder, M. van, Finke, J., Bornhauser, M., Kobbe, G., Germing, U., Witte, T.J. de, Kroger, N., Brand, R., Putter, H., Biezen, A. van, Niederwieser, D., Martino, R., Mufti, G., Onida, F., Symeonidis, A., Schmid, C., Garderet, L., Robin, M., Gelder, M. van, Finke, J., Bornhauser, M., Kobbe, G., Germing, U., Witte, T.J. de, and Kroger, N.

Abstract

Contains fulltext : 183575.PDF (publisher's version ) (Open Access), Allogeneic stem cell transplantation (ASCT) from related or unrelated donors may cure patients with myelodysplastic syndromes (MDS), a heterogeneous group of clonal stem cell disorders. We analysed 384 elderly patients (55-69 years) with advanced MDS who received either ASCT (n=247) and were reported to The European Group for Blood and Marrow Transplantation (EBMT) or a non -transplant approach (n=137) reported to the Dusseldorf registry. Besides an attempt to answer the question of "comparison", the purpose of this work is to explain the difficulties in comparing a non-transplant with a transplant cohort, when death before transplant is likely but unknown and the selection of patients for transplant is based on assumptions. It shows which methods are almost always biased and that even the most sophisticated approaches crucially rely on clinical assumptions. Using the most appropriate model for our data, we derive an overall univariate non-significant survival disadvantage for the transplant cohort (HR: 1.29, p = 0.11). We show that such an "average" hazard ratio is however misleading due to non-proportionality of the hazards reflecting early treatment related mortality, the occurring of which is logically correlated with the interval between diagnosis and transplant creating a disproportional drop in the (reconstructed) survival curve of the transplanted patients. Also in multivariate analysis (correcting for age > 60 (HR: 1.4, p = 0.02) and abnormal cytogenetics (HR: 1.46, p = 0.01)), transplantation seems to be worse (HR: 1.39, p = 0.05) but only in the (incorrect but commonly applied) model without time varying covariates. The long term (time depending) hazard ratio is shown to be virtually 1 and overall survival is virtually identical in both groups. Nonetheless no conclusion can be reached from a clinical point of view without assumptions which are by their very nature untestable unless all patients would be followed from diagnosis.

Published
2013

46. Cidofovir for CMV infection and disease in allogeneic stem cell transplant

Author

Ljungman, P, LAMBERTENGHI DELILIERS, P, Platzbacker, U, Matthesmartin, S, Bacigalupo, A, Einsele, H, Ullmann, J, Musso, M, Trenschel, R., Ribaud, P, Bornhauser, M, Cesaro, Simone, Crooks, B., Dekker, A., Gratecos, N., Klingebiel, T., Tagliaferri, E., Ullmann, A., Wacker, P., and Cordonnier, C.

Subjects
cidofovir, infection, hematopoietic stem cell transplantation, hematopoietic stem cell transplantation, cidofovir, infection
Published
2001

47. Unrelated stem cell transplantation after reduced intensity conditioning for patients with multiple myeloma relapsing after autologous transplantation.

Author

Kroger, N., Shimoni, A., Schilling, G., Schwerdtfeger, R., Bornhauser, M., Nagler, A., Zander, A.R., Heinzelmann, M., Brand, R., Gahrton, G., Morris, C., Niederwieser, D., Witte, T.J.M. de, Kroger, N., Shimoni, A., Schilling, G., Schwerdtfeger, R., Bornhauser, M., Nagler, A., Zander, A.R., Heinzelmann, M., Brand, R., Gahrton, G., Morris, C., Niederwieser, D., and Witte, T.J.M. de

Abstract

Contains fulltext : 87573.pdf (publisher's version ) (Closed access), From 2002 to 2007, 49 myeloma patients who relapsed following autologous SCT were included in a prospective multicenter trial to determine the efficacy of a reduced melphalan/fludarabine regimen followed by allogeneic SCT from unrelated donors. All patients showed leucocyte and platelet engraftment after a median of 15 and 19 d, respectively. Grade II-IV acute graft-versus-host disease (GvHD) occurred in 25% of patients and 35% had chronic GvHD. Overall response rate at day 100 was 95% including 46% complete remission (CR). Cumulative incidence of non-relapse mortality at 1 year was 25% [95% confidence interval (CI): 13-37%] and was significantly lower for human leucocyte antigen (HLA)-matched compared to -mismatched SCT (10% vs. 53%, P = 0.001). The cumulative incidence of relapse at 3 years was 55% (95% CI: 40-70%). After a median follow up of 43 months, the estimated 5-year progression-free and overall survival rates were 20% and 26% respectively and were significantly better for matched in CR at day 100 (41% vs. 7%, P = 0.04 and 56% vs. 16%, P = 0.02). We conclude that optimal donor selection is mandatory for a low non-relapse mortality and high relapse incidence, which remains a major concern, should be improved by including post-transplant strategies to upgrade remission status.

Published
2010

48. Solid organ transplantation after allogeneic hematopoietic stem cell transplantation: a retrospective, multicenter study of the EBMT.

Author

Koenecke, C., Hertenstein, B., Schetelig, J., Biezen, A. van, Dammann, E., Gratwohl, A., Ganser, A., Schleuning, M., Bornhauser, M., Jacobsen, N., Kroger, N., Niederwieser, D., Witte, T.J.M. de, Ruutu, T., Koenecke, C., Hertenstein, B., Schetelig, J., Biezen, A. van, Dammann, E., Gratwohl, A., Ganser, A., Schleuning, M., Bornhauser, M., Jacobsen, N., Kroger, N., Niederwieser, D., Witte, T.J.M. de, and Ruutu, T.

Abstract

1 augustus 2010, Contains fulltext : 89230.pdf (publisher's version ) (Closed access), To analyze the outcome of solid organ transplantation (SOT) in patients who had undergone allogeneic hematopoietic stem cell transplantation (HSCT), a questionnaire survey was carried out within 107 European Group of Blood and Marrow Transplantation centers. This study covered HSCT between 1984 and 2007 in Europe. Forty-five SOT in 40 patients were reported. Fifteen liver, 15 renal, 13 lung, 1 heart and 1 skin transplantations were performed in 28 centers. Overall survival (OS) of patients after SOT was 78% at 5 years (95% confidence interval [CI], 64% to 92%). OS at 5 years was 100% for renal, 71% (95% CI, 46% to 96%) for liver and 63% (95% CI, 23% to 100%) for lung transplant recipients. The 2-year-incidence of SOT failure was 20% (95% CI, 4% to 36%) in patients with graft-versus-host disease (GvHD) and 7% (95% CI, 0% to 21%) in patients without GvHD before SOT. The relapse incidence for underlying malignant diseases was 4% at 5 years (95% CI, 0% to 12%). In summary, this study shows that selected patients receiving SOT after HSCT have a remarkably good overall and organ survival. These data indicate that SOT should be considered in selected patients with single organ failure after HSCT.

Published
2010

49. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation.

Author

Kroger, N., Holler, E., Kobbe, G., Bornhauser, M., Schwerdtfeger, R., Baurmann, H., Nagler, A., Bethge, W., Stelljes, M., Uharek, L., Wandt, H., Burchert, A., Corradini, P., Schubert, J., Kaufmann, M., Dreger, P., Wulf, G.G., Einsele, H., Zabelina, T., Kvasnicka, H.M., Thiele, J., Brand, R., Zander, A.R., Niederwieser, D., Witte, T.J.M. de, Kroger, N., Holler, E., Kobbe, G., Bornhauser, M., Schwerdtfeger, R., Baurmann, H., Nagler, A., Bethge, W., Stelljes, M., Uharek, L., Wandt, H., Burchert, A., Corradini, P., Schubert, J., Kaufmann, M., Dreger, P., Wulf, G.G., Einsele, H., Zabelina, T., Kvasnicka, H.M., Thiele, J., Brand, R., Zander, A.R., Niederwieser, D., and Witte, T.J.M. de

Abstract

Contains fulltext : 79937.pdf (publisher's version ) (Closed access), From 2002 to 2007, 103 patients with primary myelofibrosis or postessential thrombocythemia and polycythemia vera myelofibrosis and a median age of 55 years (range, 32-68 years) were included in a prospective multicenter phase 2 trial to determine efficacy of a busulfan (10 mg/kg)/fludarabine (180 mg/m(2))-based reduced-intensity conditioning regimen followed by allogeneic stem cell transplantation from related (n = 33) or unrelated donors (n = 70). All but 2 patients (2%) showed leukocyte and platelet engraftment after a median of 18 and 22 days, respectively. Acute graft-versus-host disease grade 2 to 4 occurred in 27% and chronic graft-versus-host disease in 43% of the patients. Cumulative incidence of nonrelapse mortality at 1 year was 16% (95% confidence interval, 9%-23%) and significantly lower for patients with a completely matched donor (12% vs 38%; P = .003). The cumulative incidence of relapse at 3 years was 22% (95% confidence interval, 13%-31%) and was influenced by Lille risk profile (low, 14%; intermediate, 22%; and high, 34%; P = .02). The estimated 5-year event-free and overall survival was 51% and 67%, respectively. In a multivariate analysis, age older than 55 years (hazard ratio = 2.70; P = .02) and human leukocyte antigen-mismatched donor (hazard ratio = 3.04; P = .006) remained significant factors for survival. The study was registered at www.clinicaltrials.gov as #NCT 00599547.

Published
2009

50. Allogeneic hematopoietic stem-cell transplantation for chronic lymphocytic leukemia with 17p deletion: a retrospective European Group for Blood and Marrow Transplantation analysis.

Author

Schetelig, J., Biezen, A. van, Brand, R., Caballero, D., Martino, R., Itala, M., Garcia-Marco, J.A., Volin, L., Schmitz, N., Schwerdtfeger, R., Ganser, A., Onida, F., Mohr, B., Stilgenbauer, S., Bornhauser, M., Witte, T.J.M. de, Dreger, P., Schetelig, J., Biezen, A. van, Brand, R., Caballero, D., Martino, R., Itala, M., Garcia-Marco, J.A., Volin, L., Schmitz, N., Schwerdtfeger, R., Ganser, A., Onida, F., Mohr, B., Stilgenbauer, S., Bornhauser, M., Witte, T.J.M. de, and Dreger, P.

Abstract

Contains fulltext : 69689.pdf (publisher's version ) (Closed access), PURPOSE: Patients with chronic lymphocytic leukemia (CLL) and 17p deletion (17p-) have a poor prognosis. Although allogeneic hematopoietic stem-cell transplantation (HCT) has the potential to cure patients with advanced CLL, it is not known whether this holds true for patients with 17p-CLL. PATIENTS AND METHODS: Baseline data from patients, for whom information on the presence of 17p-CLL was available, were downloaded from the European Group for Blood and Marrow Transplantation database. Additional information on the course of CLL and follow-up was collected with a questionnaire. RESULTS: A total of 44 patients with 17p-CLL received allogeneic HCT between March 1995 and July 2006 from a matched sibling (n = 24) or an alternative donor (n = 20). 17p-CLL had been diagnosed by fluorescent in situ hybridization in 82% of patients and by conventional banding in 18% of patients. The median age was 54 years. Before HCT, a median of three lines of chemotherapy had been administered. At HCT, 53% of patients were in remission. Reduced-intensity conditioning was applied in 89% of patients. Acute, grade 2 to 4 graft-versus-host disease (GVHD) occurred in 43% of patients, and extensive chronic GVHD occurred in 53% of patients. At last follow-up, 19 patients were alive, with a median observation time of 39 months (range, 18 to 101 months). Three-year overall survival and progression-free survival rates were 44% and 37%, respectively. The cumulative incidence of progressive disease at 4 years was 34%. No late relapse occurred in nine patients with a follow-up longer than 4 years. CONCLUSION: Allogeneic HCT has the potential to induce long-term disease-free survival in patients with 17p-CLL.

Published
2008

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