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1. Allogeneic hematopoietic stem cell transplantation for adult HLH: a retrospective study by the chronic malignancies and inborn errors working parties of EBMT

Author

Machowicz, Rafal, Suarez, Felipe, Wiktor-Jedrzejczak, Wieslaw, Eikema, Diderik-Jan, de Wreede, Liesbeth C., Blok, Henric-Jan, Isaksson, Cecilia, Einsele, Hermann, Poiré, Xavier, van Dorp, Suzanne, Nikolousis, Emmanouil, Johansson, Jan-Erik, Kobbe, Guido, Zecca, Marco, Arnold, Renate, Gerbitz, Armin, Finke, Jürgen, Díez-Martín, Jose Luis, Bonifazi, Francesca, McQuaker, Grant, Lenhoff, Stig, Rohrlich, Pierre-Simon, Theobald, Matthias, Ljungman, Per, Collin, Matthew, Albert, Michael H., Ehninger, Gerhard, Carlson, Kristina, Halaburda, Kazimierz, Lehmberg, Kai, Schönland, Stefan, Yakoub-Agha, Ibrahim, Gennery, Andrew R., Lankester, Arjan C., and Kröger, Nicolaus

Published
2022
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2. Long-Term Results of Allogeneic Stem Cell Transplantation in Adult Ph- Negative High-Risk Acute Lymphoblastic Leukemia

Author

Beelen, Dietrich W., Arnold, Renate, Stelljes, Matthias, Alakel, Nael, Brecht, Arne, Bug, Gesine, Bunjes, Donald, Faul, Christoph, Finke, Jürgen, Franke, Georg-Nikolaus, Holler, Ernst, Kobbe, Guido, Kröger, Nicolaus, Rösler, Wolf, Scheid, Christof, Schönland, Stefan, Stadler, Michael, Tischer, Johanna, Wagner-Drouet, Eva, Wendelin, Knut, Brüggemann, Monika, Reiser, Lena, Hoelzer, Dieter, and Gökbuget, Nicola

Published
2022
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4. Autologous hematopoietic stem cell transplantation with reduced-intensity conditioning regimens in refractory Takayasu arteritis: a retrospective multicenter case-series from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Laurent, Charlotte, Marjanovic, Zora, Ricard, Laure, Henes, Joerg, Dulery, Remy, Badoglio, Manuela, Farge, Dominique, Snowden, John A., Moraes, Daniela, Dias, Juliana, Mohty, Mohamad, Soussan, Michael, Fain, Olivier, Arnold, Renate, Alexander, Tobias, Oliveira, Maria-Carolina, and Mekinian, Arsène

Published
2020
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8. Matching for the MICA-129 polymorphism is beneficial in unrelated hematopoietic stem cell transplantation

Author

Fuerst, Daniel, Neuchel, Christine, Niederwieser, Dietger, Bunjes, Donald, Gramatzki, Martin, Wagner, Eva, Wulf, Gerald, Glass, Bertram, Pfreundschuh, Michael, Einsele, Hermann, Arnold, Renate, Stuhler, Gernot, Schaefer-Eckart, Kerstin, Freitag, Sebastian, Casper, Jochen, Kaufmann, Martin, Wattad, Mohammed, Hertenstein, Bernd, Klein, Stefan, Ringhoffer, Mark, Mytilineos, Daphne, Tsamadou, Chrysanthi, Mueller, Carlheinz, Schrezenmeier, Hubert, and Mytilineos, Joannis

Published
2016
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9. Sorafenib promotes graft-versus-leukemia activity in mice and humans through IL-15 production in FLT3-ITD-mutant leukemia cells

Author

Mathew, Nimitha R, Baumgartner, Francis, Braun, Lukas, O'Sullivan, David, Thomas, Simone, Waterhouse, Miguel, Müller, Tony A, Hanke, Kathrin, Taromi, Sanaz, Apostolova, Petya, Illert, Anna L, Melchinger, Wolfgang, Duquesne, Sandra, Schmitt-Graeff, Annette, Osswald, Lena, Yan, Kai-Li, Weber, Arnim, Tugues, Sonia, Spath, Sabine, Pfeifer, Dietmar, Follo, Marie, Claus, Rainer, Lübbert, Michael, Rummelt, Christoph, Bertz, Hartmut, Wäsch, Ralph, Haag, Johanna, Schmidts, Andrea, Schultheiss, Michael, Bettinger, Dominik, Thimme, Robert, Ullrich, Evelyn, Tanriver, Yakup, Vuong, Giang Lam, Arnold, Renate, Hemmati, Philipp, Wolf, Dominik, Ditschkowski, Markus, Jilg, Cordula, Wilhelm, Konrad, Leiber, Christian, Gerull, Sabine, Halter, Jörg, Lengerke, Claudia, Pabst, Thomas, Schroeder, Thomas, Kobbe, Guido, Rösler, Wolf, Doostkam, Soroush, Meckel, Stephan, Stabla, Kathleen, Metzelder, Stephan K, Halbach, Sebastian, Brummer, Tilman, Hu, Zehan, Dengjel, Joern, Hackanson, Björn, Schmid, Christoph, Holtick, Udo, Scheid, Christof, Spyridonidis, Alexandros, Stölzel, Friedrich, Ordemann, Rainer, Müller, Lutz P, Sicre-de-Fontbrune, Flore, Ihorst, Gabriele, Kuball, Jürgen, Ehlert, Jan E, Feger, Daniel, Wagner, Eva-Maria, Cahn, Jean-Yves, Schnell, Jacqueline, Kuchenbauer, Florian, Bunjes, Donald, Chakraverty, Ronjon, Richardson, Simon, Gill, Saar, Kröger, Nicolaus, Ayuk, Francis, Vago, Luca, Ciceri, Fabio, Müller, Antonia M, Kondo, Takeshi, Teshima, Takanori, Klaeger, Susan, Kuster, Bernhard, Weisdorf, Daniel, van der Velden, Walter, Dörfel, Daniela, Bethge, Wolfgang, Hilgendorf, Inken, Hochhaus, Andreas, Andrieux, Geoffroy, Börries, Melanie, Busch, Hauke, Magenau, John, Reddy, Pavan, Labopin, Myriam, Antin, Joseph H, Henden, Andrea S, Hill, Geoffrey R, Kennedy, Glen A, Bar, Merav, Sarma, Anita, McLornan, Donal, Mufti, Ghulam, Oran, Betul, Rezvani, Katayoun, Shah, Omid, Negrin, Robert S, Nagler, Arnon, Prinz, Marco, Burchert, Andreas, Neubauer, Andreas, Beelen, Dietrich, Mackensen, Andreas, von Bubnoff, Nikolas, Herr, Wolfgang, Becher, Burkhard, Socié, Gerard, Caligiuri, Michael A, Ruggiero, Eliana, Bonini, Chiara, Häcker, Georg, Duyster, Justus, Finke, Jürgen, Pearce, Erika, Blazar, Bruce R, and Zeiser, Robert

Subjects
Interleukin-15 -- Health aspects, Gene mutation -- Health aspects, Sorafenib -- Patient outcomes, Gene expression -- Health aspects, Acute myelocytic leukemia -- Genetic aspects -- Development and progression -- Drug therapy, Biological sciences, Health
Abstract

Individuals with acute myeloid leukemia (AML) harboring an internal tandem duplication (ITD) in the gene encoding Fms-related tyrosine kinase 3 (FLT3) who relapse after allogeneic hematopoietic cell transplantation (allo-HCT) have a 1-year survival rate below 20%. We observed that sorafenib, a multitargeted tyrosine kinase inhibitor, increased IL-15 production by FLT3-ITD[sup.+] leukemia cells. This synergized with the allogeneic CD8[sup.+] T cell response, leading to long-term survival in six mouse models of FLT3-ITD[sup.+] AML. Sorafenib-related IL-15 production caused an increase in CD8[sup.+]CD107a[sup.+]IFN-[gamma][sup.+] T cells with features of longevity (high levels of Bcl-2 and reduced PD-1 levels), which eradicated leukemia in secondary recipients. Mechanistically, sorafenib reduced expression of the transcription factor ATF4, thereby blocking negative regulation of interferon regulatory factor 7 (IRF7) activation, which enhanced IL-15 transcription. Both IRF7 knockdown and ATF4 overexpression in leukemia cells antagonized sorafenib-induced IL-15 production in vitro. Human FLT3-ITD[sup.+] AML cells obtained from sorafenib responders following sorafenib therapy showed increased levels of IL-15, phosphorylated IRF7, and a transcriptionally active IRF7 chromatin state. The mitochondrial spare respiratory capacity and glycolytic capacity of CD8[sup.+] T cells increased upon sorafenib treatment in sorafenib responders but not in nonresponders. Our findings indicate that the synergism of T cells and sorafenib is mediated via reduced ATF4 expression, causing activation of the IRF7-IL-15 axis in leukemia cells and thereby leading to metabolic reprogramming of leukemia-reactive T cells in humans. Therefore, sorafenib treatment has the potential to contribute to an immune-mediated cure of FLT3-ITD-mutant AML relapse, an otherwise fatal complication after allo-HCT., Author(s): Nimitha R Mathew [1, 2]; Francis Baumgartner [1]; Lukas Braun [1]; David O'Sullivan [3]; Simone Thomas [4]; Miguel Waterhouse [1]; Tony A Müller [1]; Kathrin Hanke [1, 2]; Sanaz [...]

Published
2018
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11. Peptide-specific recognition of human cytomegalovirus strains controls adaptive natural killer cells

Author

Hammer, Quirin, Rückert, Timo, Borst, Eva Maria, Dunst, Josefine, Haubner, André, Durek, Pawel, Heinrich, Frederik, Gasparoni, Gilles, Babic, Marina, Tomic, Adriana, Pietra, Gabriella, Nienen, Mikalai, Blau, Igor Wolfgang, Hofmann, Jörg, Na, Il-Kang, Prinz, Immo, Koenecke, Christian, Hemmati, Philipp, Babel, Nina, Arnold, Renate, Walter, Jörn, Thurley, Kevin, Mashreghi, Mir-Farzin, Messerle, Martin, and Romagnani, Chiara

Published
2018
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13. The impact of HLA-matching on reduced intensity conditioning regimen unrelated donor allogeneic stem cell transplantation for acute myeloid leukemia in patients above 50 years—a report from the EBMT acute leukemia working party

Author

Marie T. Rubio, Bipin N. Savani, Myriam Labopin, Emmanuelle Polge, Dietger Niederwieser, Arnold Ganser, Rainer Schwerdtfeger, Gerhard Ehninger, Jürgen Finke, Arnold Renate, Charles Craddock, Nicolaus Kröger, Michael Hallek, Pavel Jindra, Mohamad Mohty, and Arnon Nagler

Subjects
Allogeneic stem cell transplantation, Unrelated donor, Older patients, HLA matching, Acute leukemia, Toxicity, Diseases of the blood and blood-forming organs, RC633-647.5, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Abstract Background Data comparing fully matched and mismatched-unrelated-donor (M- and mM-URD) allogeneic hematopoietic stem cell transplant (allo-SCT) following reduced intensity conditioning regimens for acute myeloid leukemia are limited. Methods We retrospectively compared the outcome of 3398 patients above the age of 50 years who underwent 10/10 M-URD (n = 2567), 9/10 (n = 723), or 8/10 (n = 108) mM-URD allo-SCT for acute myeloid leukemia after reduced intensity conditioning regimen between 2000 and 2013. The Kaplan-Meier estimator, the cumulative incidence function, and Cox proportional hazards regression models were used where appropriate. Results HLA matching had no impact on engraftment (p = 0.31). In univariate analysis, in comparison to 10/10 M-URD, mM-URD was associated with higher incidence of grade II–IV acute graft-versus-host disease (GVHD) (p = 0.0002), similar rates of chronic GVHD (p = 0.138) but increased incidence of its extensive form (p = 0.047). Compared to 10/10 M-URD, patients transplanted in the first complete remission (CR1) with a 9 or an 8/10 mM-URD had decreased 2-year leukemia free (LFS) (p = 0.005) and overall survivals (OS) (56.7, 46.1, and 50.2 %, respectively, p = 0.005), while outcomes were comparable between all groups for patients transplanted beyond CR1. In multivariate analysis, 9/10 versus 10/10 URD was associated with higher non-relapse mortality (HR 1.34, p = 0.001), similar risk of relapse and chronic GVHD and inferior LFS (HR 1.25, p = 0.0001), and OS (HR 1.27, p = 0.0001). There was no difference in adjusted transplant outcomes between 9/10 and 8/10 mM-URD. Conclusions Reduced intensity conditioned allo-SCT with a 10/10 M-URD remains the preferable option for AML patients above the age of 50 years. The use of a 9/10 or an 8/10 mM-URD in patients not having a fully matched donor represents an alternative therapeutic option that should be compared to other alternative donor transplant strategies.

Published
2016
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16. Treatment of Adult ALL According to Protocols of the German Multicenter Study Group for Adult ALL (GMALL)

Author

German Multicenter Study Group for Adult ALL (GMALL), Gökbuget, Nicola, Arnold, Renate, Böhme, Angelika, Fietkau, Rainer, Freund, Matthias, Ganser, Arnold, Kneba, Michael, Lipp, Thomas, Ludwig, Wolf-Dieter, Maschmeyer, Georg, Messerer, Dorle, Rieder, Harald, Thiel, Eckhard, Hoelzer, Dieter, Estey, E. H., Faderl, S. H., and Kantarjian, H. M.

Published
2008
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19. Splenic irradiation before hematopoietic stem cell transplantation for chronic myeloid leukemia: long-term follow-up of a prospective randomized study

Author

Gratwohl, Alois, Iacobelli, Simona, Bootsman, Natalia, van Biezen, Anja, Baldomero, Helen, Arcese, William, Arnold, Renate, Bron, Dominique, Cordonnier, Catherine, Ernst, Peter, Ferrant, Augustin, Frassoni, Francesco, Gahrton, Gösta, Richard, Carlos, Kolb, Hans Jochem, Link, Hartmut, Niederwieser, Dietger, Ruutu, Tapani, Schattenberg, Anton, Schmitz, Norbert, Torres-Gomez, Antonio, Zwaan, Ferry, Apperley, Jane, Olavarria, Eduardo, Kröger, Nicolaus, and for the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation EBMT

Published
2016
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22. Mobilized Peripheral Blood Stem Cells Compared with Bone Marrow as the Stem Cell Source for Unrelated Donor Allogeneic Transplantation with Reduced-Intensity Conditioning in Patients with Acute Myeloid Leukemia in Complete Remission: An Analysis from the Acute Leukemia Working Party of the European Group for Blood and Marrow Transplantation

Author

Nagler, Arnon, Labopin, Myriam, Shimoni, Avichai, Niederwieser, Dietger, Mufti, Ghulam J., Zander, Axel R., Arnold, Renate, Greinix, Hildegard, Cornelissen, Jan J., Jackson, Graham H., Craddock, Charles, Bunjes, Donald W., Ganser, Arnold, Russell, Nigel H., Kyrcz-Krzemien, Slawomira, Rocha, Vanderson, and Mohty, Mohamad

Published
2012
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23. Adult patients with acute lymphoblastic leukemia and molecular failure display a poor prognosis and are candidates for stem cell transplantation and targeted therapies

Author

Gökbuget, Nicola, Kneba, Michael, Raff, Thorsten, Trautmann, Heiko, Bartram, Claus-Rainer, Arnold, Renate, Fietkau, Rainer, Freund, Mathias, Ganser, Arnold, Ludwig, Wolf-Dieter, Maschmeyer, Georg, Rieder, Harald, Schwartz, Stefan, Serve, Hubert, Thiel, Eckhard, Brüggemann, Monika, and Hoelzer, Dieter

Published
2012
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28. Allogeneic hematopoietic stem cell transplantation (allo SCT) for chronic myeloid leukemia in the imatinib era: evaluation of its impact within a subgroup of the randomized German CML Study IV

Author

Saussele, Susanne, Lauseker, Michael, Gratwohl, Alois, Beelen, Dietrich W., Bunjes, Donald, Schwerdtfeger, Rainer, Kolb, Hans-Jochem, Ho, Anthony D., Falge, Christiane, Holler, Ernst, Schlimok, Günter, Zander, Axel R., Arnold, Renate, Kanz, Lothar, Dengler, Robert, Haferlach, Claudia, Schlegelberger, Brigitte, Pfirrmann, Markus, Müller, Martin C., Schnittger, Susanne, Leitner, Armin, Pletsch, Nadine, Hochhaus, Andreas, Hasford, Joerg, Hehlmann, Rüdiger, and for the German CML Study Group

Published
2010
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30. Comparison of Dynamic International Prognostic Scoring System and MYelofibrosis SECondary to PV and ET Prognostic Model for Prediction of Outcome in Polycythemia Vera and Essential Thrombocythemia Myelofibrosis after Allogeneic Stem Cell Transplantation

Author

Gagelmann, Nico, Eikema, Diderik-Jan, de Wreede, Liesbeth C, Koster, Linda, Wolschke, Christine, Arnold, Renate, Kanz, Lothar, McQuaker, Grant, Marchand, Tony, Socié, Gerard, Bourhis, Jean Henri, Mohty, Mohamad, Cornelissen, Jan J, Chevallier, Patrice, Bernasconi, Paolo, Stelljes, Matthias, Rohrlich, Pierre-Simon, Fanin, Renato, Finke, Jürgen, Maertens, Johan, Blaise, Didier, Itälä-Remes, Maija, Labussière-Wallet, Hélène, Robin, Marie, McLornan, Donal, Chalandon, Yves, Yakoub-Agha, Ibrahim, Kröger, Nicolaus, CMWP of the European Society for Blood and Marrow Transplantation, Hematology, and Medical Oncology

Subjects
Adult, medicine.medical_specialty, Essential thrombocythemia, Gastroenterology, 03 medical and health sciences, 0302 clinical medicine, Polycythemia vera, Internal medicine, Humans, Transplantation, Homologous, Medicine, Allogeneic stem cell transplantation, Secondary myelofibrosis, Hematology, Transplantation, Myelofibrosis, Polycythemia Vera, Survival analysis, Aged, ddc:616, Framingham Risk Score, business.industry, Hematopoietic Stem Cell Transplantation, Middle Aged, Prognosis, medicine.disease, Survival Analysis, Treatment Outcome, Primary Myelofibrosis, International Prognostic Scoring System, 030220 oncology & carcinogenesis, Cohort, business, Thrombocythemia, Essential, 030215 immunology
Abstract

We aimed to validate the MYelofibrosis SECondary to PV and ET prognostic model (MYSEC-PM) in 159 patients with myelofibrosis secondary to polycythemia vera (PV) and essential thrombocythemia (ET) from the European Society for Blood and Marrow Transplantation registry undergoing transplantation from matched siblings or unrelated donors. Furthermore, we aimed to test its prognostic performance in comparison with the Dynamic International Prognostic Scoring System (DIPSS). Score performance was analyzed using the concordance index (C): the probability that a patient who experienced an event had a higher risk score than a patient who did not (C > .5 suggesting predictive ability). Median follow-up of the total cohort was 41 months (range, 34 to 54), 45 months in post-PV and 38 months in post-ET myelofibrosis. Survival at 1, 2, and 4 years was 70% (95% CI, 63% to 77%), 61% (95% CI, 53% to 69%), and 52% (95% CI, 43% to 61%) for the total cohort; 70% (95% CI, 59% to 80%), 61% (95% CI, 49% to 73%), and 51% (95% CI, 38% to 64%) for post-PV; and 71% (95% CI, 61% to 81%), 61% (95% CI, 50% to 72%), and 54% (95% CI, 42% to 66%) for post-ET myelofibrosis (P = .78). Overall, the DIPSS was not significantly predictive of outcome (P = .28). With respect to the MYSEC-PM, overall survival at 4 years was 69% for the low-risk, 55% for the intermediate 1-risk, 47% for the intermediate 2-risk, and 22% (0% to 45%) for the high-risk groups. The prognostic model was predictive of survival overall (P = .05), whereas groups with intermediate 2 and high risk showed no significant difference (P = .44). Assessment of prognostic utility yielded a C-index of .575 (95% CI, .502 to .648) for the DIPSS, whereas assessment of the MYSEC-PM resulted in a C-statistics of .636 (95% CI, .563 to .708), indicating improvement in prediction of post-transplant survival using the new MYSEC-PM. In addition, transplantations from an unrelated donor in comparison with an HLA-identical sibling showed worse outcome (P = .04), and transplant recipients seropositive for cytomegalovirus in comparison with seronegative recipients (P = .01) showed worse survival. In conclusion, incorporating transplant-specific and clinical and mutational information together with the MYSEC-PM may enhance risk stratification. ispartof: Biol Blood Marrow Transplant vol:25 issue:6 pages:e204-e208 ispartof: location:United States status: published

Published
2019

33. Depletion of autoreactive immunologic memory followed by autologous hematopoietic stem cell transplantation in patients with refractory SLE induces long-term remission through de novo generation of a juvenile and tolerant immune system

Author

Alexander, Tobias, Thiel, Andreas, Rosen, Oliver, Massenkeil, Gero, Sattler, Arne, Kohler, Siegfried, Mei, Henrik, Radtke, Hartmut, Gromnica-Ihle, Erika, Burmester, Gerd-Rüdiger, Arnold, Renate, Radbruch, Andreas, and Hiepe, Falk

Published
2009
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34. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini‐Gutzwiller, Federica R., Snowden, John A., Corbacioglu, Selim, Greco, Raffaella, Alexander, Tobias, Snowden, John, Badoglio, Manuela, Labopin, Myriam, Abinun, Mario, Apte, Shashikant, Arnold, Renate, Domenech, Ariadna, Brierley, Charlotte, Burman, Joachim, Castilla‐Llorente, Cristina, Cooper, Nichola, Daghia, Giulia, Daikeler, Thomas, del Papa, Nicoletta, and de Vries‐Bouwstra, Jeska

Subjects
HEMATOPOIETIC stem cell transplantation, AUTOIMMUNE diseases, JUVENILE diseases, PEDIATRICS, BONE marrow, LITERATURE reviews
Abstract

Summary: Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long‐term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment‐refractory autoimmune diseases (ADs) in children. However, transplant‐related outcomes are disease‐dependent and long‐term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non‐transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long‐term disease control or cure, but therapy‐related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence‐based interspeciality guidelines and recommendations. [ABSTRACT FROM AUTHOR]

Published
2022
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36. Impact of postremission consolidation chemotherapy on outcome after reduced-intensity conditioning allogeneic stem cell transplantation for patients with acute myeloid leukemia in first complete remission: A report from the Acute Leukemia Working Party of the European Group for Blood and Marrow Transplantation

Author

Yeshurun, Moshe, Labopin, Myriam, Blaise, Didier, Cornelissen, Jan J., Sengeloev, Henrik, Vindelov, Lars, Kuball, Juergen, Chevallier, Patrice, Craddock, Charles, Socie, Gerard, Bilger, Karin, Schouten, Harry C., Fegueux, Nathalie, Goker, Hakan, Maertens, Johan, Bunjes, Donald, Arnold, Renate, Nagler, Arnon, and Mohty, Mohamad

Published
2014
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42. Mobilized peripheral blood stem cells compared with bone marrow from HLA-identical siblings for reduced-intensity conditioning transplantation in acute myeloid leukemia in complete remission: a retrospective analysis from the Acute Leukemia Working Party of EBMT

Author

Nagler, Arnon, Labopin, Myriam, Shimoni, Avichai, Mufti, Ghulam J., Cornelissen, Jan J., Blaise, Didier, Janssen, Jeroen J. W. M., Milpied, Noel, Vindelov, Lars, Petersen, Eefke, Gribben, John, Bacigalupo, Andrea, Malm, Claes, Niederwieser, Dietger, Socié, Gerard J., Arnold, Renate, Brown, Paul, Goker, Hakan, Rocha, Vanderson, and Mohty, Mohamad

Published
2012
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47. Long-term outcome after allogeneic hematopoietic cell transplantation for myelofibrosis

Author

Robin, Marie, De Wreede, Liesbeth C., Wolschke, Christine, Schetelig, Johannes, Eikema, Diderik-Jan, Van Lint, Maria Teresa, Knelange, Nina Simone, Beelen, Dietrich, Brecht, Arne, Niederwieser, Dietger, Vitek, Antonin, Bethge, Wolfgang, Arnold, Renate, Finke, Jürgen, Volin, Liisa, Yakoub-Agha, Ibrahim, Nagler, Arnon, Poiré, Xavier, Einsele, Hermann, Chevallier, Patrice, Holler, Ernst, Ljungman, Per, Robinson, Stephen, Radujkovic, Alekxandar, McLornan, Donal, Chalandon, Yves, Kröger, Nicolaus, Eikema, Dirk-Jan, Radujkovic, Aleksandar, UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Service d'hématologie, HUS Comprehensive Cancer Center, Hematologian yksikkö, and Department of Oncology

Subjects
Male, Ruxolitinib, BLOOD, Multivariate analysis, Survival, medicine.medical_treatment, Medizin, Graft vs Host Disease, Hematopoietic stem cell transplantation, Disease, PROGNOSTIC SCORING SYSTEM, GRUPPO-ITALIANO, MIDOLLO-OSSEO, 0302 clinical medicine, Risk Factors, Midollo-Osseo, RUXOLITINIB, Registries, SOCIETE FRANCAISE, Cause of death, ddc:616, education.field_of_study, Remission Induction, Hematopoietic Stem Cell Transplantation, Hematology, Middle Aged, 3. Good health, Blood, Treatment Outcome, SURVIVAL, Female, medicine.drug, Adult, medicine.medical_specialty, LATE MORTALITY, Population, Article, Disease-Free Survival, 03 medical and health sciences, Internal medicine, medicine, Humans, Transplantation, Homologous, ddc:610, education, Myelofibrosis, Aged, EUROPEAN GROUP, business.industry, medicine.disease, Transplantation, LATE DEATHS, Primary Myelofibrosis, 3121 General medicine, internal medicine and other clinical medicine, Multivariate Analysis, Myeloproliferative Neoplasms, Neoplasm Recurrence, Local, business, 030215 immunology
Abstract

Allogeneic hematopoietic stem cell transplant remains the only curative treatment for myelofibrosis. Most post-transplantation events Aoccur during the first two years and hence we aimed to analyze the outcome of 2-year disease-free survivors. A total of 1055 patients with myelofibrosis transplanted between 1995 and 2014 and registered in the registry of the European Society for Blood and Marrow Transplantation were included. Survival was compared to the matched general population to determine excess mortality and the risk factors that are associated. In the 2-year survivors, disease-free survival was 64% (60-68%) and overall survival was 74% (71-78%) at ten years; results were better in younger individuals and in women. Excess mortality was 14% (8-21%) in patients aged = 65 years. The main cause of death was relapse of the primary disease. Graft-versus-host disease (GvHD) before two years decreased the risk of relapse. Multivariable analysis of excess mortality showed that age, male sex recipient, secondary myelofibrosis and no GvHD disease prior to the 2-year landmark increased the risk of excess mortality. This is the largest study to date analyzing long-term outcome in patients with myelofibrosis undergoing transplant. Overall it shows a good survival in patients alive and in remission at two years. However, the occurrence of late complications, including late relapses, infectious complications and secondary malignancies, highlights the importance of screening and monitoring of long-term survivors.

Published
2019

48. Hematopoietic stem cell transplantation for autoimmune diseases - a 20 years single-centre experience

Author

Alexander, Tobias, Templin, Lars, Grützkau, Andreas, Hirseland, Heike, Thiel, Andreas, Burmester, Gerd-Rüdiger, Arnold, Renate, and Hiepe, Falk

Subjects
ddc: 610, macromolecular substances, 610 Medical sciences, Medicine
Abstract

Background: Over the past 20 years, haematopoietic stem cell transplantation (HSCT) has emerged as promising treatment option for severe cases of autoimmune diseases (ADs). The goal of this therapy is to induce durable remissions by eradicating the pathologic autoreactive memory and restoration of self-tolerance.[for full text, please go to the a.m. URL], 46. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 32. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), Wissenschaftliche Herbsttagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR)

Published
2019

49. Randomized controlled trials of autologous hematopoietic stem cell transplantation for autoimmune diseases: The evolution from myeloablative to lymphoablative transplant regimens

Author

Burt, Richard K., Marmont, Alberto, Oyama, Yu, Slavin, Shimon, Arnold, Renate, Hiepe, Falk, Fassas, Athanasios, Snowden, John, Schuening, Friedrich, Myint, Han, Patel, Dhavalkumar D., Collier, David, Heslop, Helen, Krance, Robert, Statkute, Laisvyde, Verda, Larissa, Traynor, Ann, Kozak, Tomas, Hintzen, Rogier Q., Rose, John W., Voltarelli, Julio, Loh, Yvonne, Territo, Mary, Cohen, Bruce A., Craig, Robert M., Varga, John, and Barr, Walter G.

Published
2006

50. Hematopoietic Stem Cell Transplantation for Multiple Sclerosis

Author

Burt, Richard K., Cohen, Bruce, Rose, John, Petersen, Finn, Oyama, Yu, Stefoski, Dusan, Katsamakis, George, Carrier, Ewa, Kozak, Tomas, Muraro, Paolo A., Martin, Roland, Hintzen, Roger, Slavin, Shimon, Karussis, Dimitrios, Haggiag, Shalom, Voltarelli, Julio C., Ellison, George W., Jovanovic, Borko, Popat, Uday, McGuirk, Joseph, Statkute, Laisvyde, Verda, Larissa, Haas, Judith, and Arnold, Renate

Published
2005

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