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1,470 results on '"Platzbecker U"'

1. Allogeneic Stem Cell Transplantation with Sequential Melphalan-Based Conditioning in AML: Residual Morphological Blast Count Determines the Risk of Relapse

Author

Sockel K, Stölzel F, Hönl F, Baldauf H, Röllig C, Wermke M, von Bonin M, Teipel R, Link-Rachner C, Brandt K, Kroschinsky F, Hänel M, Morgner A, Klesse C, Ehninger G, Platzbecker U, Bornhäuser M, Schetelig J, and Middeke JM

Subjects
acute myeloid leukemia, eln high risk, aml relapse/refractory, early allogeneic transplantation in aplasia, blast count, melphalan-based conditioning, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Katja Sockel,1 Friedrich Stölzel,1 Franziska Hönl,1 Henning Baldauf,2 Christoph Röllig,1 Martin Wermke,1 Malte von Bonin,1 Raphael Teipel,1 Cornelia Link-Rachner,1 Kalina Brandt,1 Frank Kroschinsky,1 Mathias Hänel,3 Anke Morgner,3 Christian Klesse,2 Gerhard Ehninger,1 Uwe Platzbecker,4 Martin Bornhäuser,1 Johannes Schetelig,1,2,* Jan Moritz Middeke1,* 1Medical Clinic and Policlinic I, University Hospital Carl Gustav Carus and Medical Faculty of the TU Dresden, Dresden, Germany; 2Clinical Trials Unit, DKMS, Dresden, Germany; 3Department of Medicine III, Chemnitz Hospital, Chemnitz, Germany; 4Department of Hematology, Cellular Therapy and Hemostaseology, Leipzig University Hospital, Leipzig, Germany*These authors contributed equally to this workCorrespondence: Jan Moritz Middeke, Medical Clinic and Policlinic I, University Hospital Carl Gustav Carus and Medical Faculty of the TU Dresden, Fetscherstr. 74, Dresden, 01307, Germany, Tel +49-0351-458-15603, Fax +49-0351-458-4373, Email JanMoritz.Middeke@uniklinikum-dresden.deIntroduction: Allogeneic hematopoietic cell transplantation (HCT) during chemotherapy-induced aplasia may offer long-term survival in acute myeloid leukemia (AML) with otherwise poor prognosis including ELN adverse risk, relapsed or refractory disease. However, the value of residual morphologic disease prior HCT in this context has not been conclusively settled until yet. Therefore, we aimed to investigate variables predicting outcome in this unique setting of sequential conditioning therapy, with a focus on pretreatment morphologic blast count. In contrast to the most popular FLAMSA-RIC protocol, we used a melphalan-based conditioning regimen during aplasia.Methods: We retrospectively analyzed data from 173 AML patients who underwent a sequential melphalan-based conditioning therapy between 2003 and 2015 at our centre. All patients participated either in the prospective Phase 2 BRIDGE trial (NCT01295307), the Phase 3 AML2003 study (NCT00180102) or were treated according to this protocol and underwent allogeneic HCT after melphalan-based conditioning in treatment-induced aplasia.Results: Median bone marrow blast count prior to conditioning was 10% (range, 0– 96%). Four year probabilities of EFS and OS were 34% (95% CI, 28– 43%) and 43% (95% CI, 36– 52%), respectively. In multivariate analysis, blast count > 20% was associated with worse EFS (HR = 1.93; p = 0.009) and OS (HR = 1.80; p = 0.026). This effect was not significant anymore for HCT during 1st line therapy.Conclusion: Allogeneic HCT in aplasia with a melphalan-based conditioning regimen has the potential to cure a subset of adverse risk AML patients, even with persistent morphological disease prior HCT. However, a high pre-transplant blast count still indicates patients with a dismal prognosis, especially in the relapsed patient group, for whom post-transplant strategies should be considered to further optimize post HCT outcome.Keywords: acute myeloid leukemia, ELN high risk, AML relapse/refractory, early allogeneic transplantation in aplasia, blast count, melphalan-based conditioning, sequential conditioning

Published
2022

2. Artificial intelligence methods to estimate overall mortality and non-relapse mortality following allogeneic HCT in the modern era: an EBMT-TCWP study

Author

Mussetti, A., Rius-Sansalvador, B., Moreno, V., Peczynski, C., Polge, E., Galimard, J. E., Kröger, N., Blaise, D., Peffault de Latour, R., Kulagin, A., Mousavi, A., Stelljes, M., Hamladji, R. M., Middeke, J. M., Salmenniemi, U., Sengeloev, H., Forcade, E., Platzbecker, U., Reményi, P., Angelucci, E., Chevallier, P., Yakoub-Agha, I., Craddock, C., Ciceri, F., Schroeder, T., Aljurf, M., Ch, Koenecke, Moiseev, I., Penack, O., Schoemans, H., Mohty, M., Glass, B., Sureda, A., Basak, G., and Peric, Z.

Published
2024
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3. Response-guided first-line therapy and treatment of relapse in aggressive lymphoma: 10-year follow-up of the PETAL trial

Author

Wilop, S., Tometten, M., Korfel, A., Keller, U., de Wit, M., Weissinger, F., Stark, U., Behringer, D., Bückner, U., Nückel, H., Schroers, R., Trenn, G., Hertenstein, B., Bernhard, H., Heike, M., Wörns, M.-A., Kroschinsky, F., Prange-Krex, G., Giagounidis, A., Grigoleit, G. U., Selbach, J., Petrasch, S., Balleisen, J. S., Schütte, J., Dienst, A., Germing, U., Dührsen, U., von Verschuer, U., Reimer, P., Höffkes, H.-G., Runde, V., Spohn, C., Moeller, R., Dürk, H., Kofahl-Krause, D., Heuser, M., Witzens-Harig, M., Müller-Tidow, C., Martens, U. M., Strumberg, D., La Rosée, P., Hochhaus, A., Link, H., Held, G., Kneba, M., Baldus, C., Naumann, R., Chemnitz, J. M., Schulte, K., Limmroth, C., Schwarzer, A., Niederwieser, D., Platzbecker, U., Heil, G., Schwalenberg, M., Grieshammer, M., Beck, C., Stephany, M., Mesters, R., Karsten, I. E., Held, H., Mahlmann, S., Steiniger, H., Gaska, T., Südhoff, T., Kreisel-Büstgens, C., Moorahrend, E., Maschmeyer, G., Jordan, K., Kloke, O., Klein, M., Höhler, T., Grube, M., Herr, W., Hahn, D., Raghavachar, A., Schmalz, O., Fett, W., Sandmann, M., Krohn, T., Brenner, W., Plotkin, M., Franzius, C., Kotzerke, J., Hautzel, H., Bockisch, A., Hertel, A., Bengel, F. M., Haberkorn, U., Freesmeyer, M., Lützen, U., Klein, A., Kluge, R., Larisch, R., Fricke, E., Holzinger, J., Schäfer, W., Weckesser, M., Nyuyki, F., Römer, W., Brink, I., Marienhagen, J., Pöpperl, G., Jöckel, K.-H., Nonnemacher, M., Rekowski, J., Scherag, A., Neuhäuser, M., Dührsen, Ulrich, Bockisch, Andreas, Hertenstein, Bernd, Karsten, Imke E., Kroschinsky, Frank, Heuser, Michael, Hochhaus, Andreas, Höffkes, Heinz-Gert, Behringer, Dirk, Prange-Krex, Gabriele, Tometten, Mareike, Grieshammer, Martin, Grigoleit, Götz U., Schmalz, Oliver, Jordan, Karin, Bernhard, Helga, Gaska, Tobias, Giagounidis, Aristoteles, Schroers, Roland, Martens, Uwe M., Held, Gerhard, Klapper, Wolfram, Jöckel, Karl-Heinz, Nonnemacher, Michael, and Hüttmann, Andreas

Published
2024
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4. Efficacy and safety of bemcentinib in patients with advanced myelodysplastic neoplasms or acute myeloid leukemia failing hypomethylating agents- the EMSCO phase II BERGAMO trial

Author

Kubasch, A. S., Peterlin, P., Cluzeau, T., Götze, K. S., Sockel, K., Teipel, R., Jentzsch, M., Attalah, H., Sebert, M., Chermat, F., Gloaguen, S., Puttrich, M., Cross, M., Schneider, M., Kayser, S., Schipp, D., Giagounidis, A., Tirado-Gonzalez, I., Descot, A., van de Loosdrecht, A., Weigert, A., Metzeler, K. H., Fenaux, P., Medyouf, H., Platzbecker, U., and Ades, L.

Published
2023
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5. Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial

Author

Zeidan, A, Ando, K, Rauzy, O, Turgut, M, Wang, M, Cairoli, R, Hou, H, Kwong, Y, Arnan, M, Meers, S, Pullarkat, V, Santini, V, Malek, K, Kiertsman, F, Niolat, J, Ramos, P, Menssen, H, Fenaux, P, Miyazaki, Y, Platzbecker, U, Zeidan A. M., Ando K., Rauzy O., Turgut M., Wang M. -C., Cairoli R., Hou H. -A., Kwong Y. -L., Arnan M., Meers S., Pullarkat V., Santini V., Malek K., Kiertsman F., Niolat J., Ramos P. M., Menssen H. D., Fenaux P., Miyazaki Y., Platzbecker U., Zeidan, A, Ando, K, Rauzy, O, Turgut, M, Wang, M, Cairoli, R, Hou, H, Kwong, Y, Arnan, M, Meers, S, Pullarkat, V, Santini, V, Malek, K, Kiertsman, F, Niolat, J, Ramos, P, Menssen, H, Fenaux, P, Miyazaki, Y, Platzbecker, U, Zeidan A. M., Ando K., Rauzy O., Turgut M., Wang M. -C., Cairoli R., Hou H. -A., Kwong Y. -L., Arnan M., Meers S., Pullarkat V., Santini V., Malek K., Kiertsman F., Niolat J., Ramos P. M., Menssen H. D., Fenaux P., Miyazaki Y., and Platzbecker U.

Abstract

Background: Sabatolimab is an immunotherapy targeting T-cell immunoglobulin domain and mucin domain-3 (TIM-3), an immuno-myeloid regulator expressed on immune cells and leukaemic stem cells. In this trial, we compared the efficacy and safety of sabatolimab plus hypomethylating agent with placebo plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes. Methods: STIMULUS-MDS1 was a multicentre, randomised, double-blind, placebo-controlled, phase 2 study done at 54 investigational sites in 17 countries. Adult patients (aged ≥18 years) with intermediate-risk, high-risk, and very high-risk myelodysplastic syndromes (according to Revised International Prognostic Scoring System criteria) who had not received previous treatment were included. Patients were randomly assigned (1:1) to intravenous sabatolimab (400 mg on day 8 and 22) or placebo plus a hypomethylating agent (intravenous decitabine 20 mg/m2 on day 1–5 or intravenous or subcutaneous azacitidine 75 mg/m2 on day 1–7 or day 1–5 and day 8 and 9) every 28 days until treatment discontinuation. The two primary endpoints were complete response rate and progression-free survival, assessed in the full analysis set, which included all randomly assigned patients. Complete response was analysed, as prespecified, 7 months after the last patient was randomly assigned. All other analyses presented, including progression-free survival, were done at the final data cutoff prespecified via a protocol amendment on Sept 2, 2021. Safety was assessed in in all patients who received at least one dose of study treatment. This study is registered with ClinicalTrials.gov, NCT03946670, and is ongoing. Findings: Between July 29, 2019, and Aug 10, 2020, 127 patients were randomly assigned to sabatolimab plus a hypomethylating agent group (sabatolimab group; n=65) or placebo plus a hypomethylating agent (placebo group; n=62). The median age of participants was 73 years (IQR 69–77), of whom 86 (68%

Published
2024

6. Trends in allogeneic haematopoietic cell transplantation for myelofibrosis in Europe between 1995 and 2018: a CMWP of EBMT retrospective analysis

Author

McLornan, D., Eikema, D. J., Czerw, T., Kröger, N., Koster, L., Reinhardt, Hans Christian, Angelucci, E., Robin, M., Bornhäuser, M., Passweg, J., Clark, A., Vydra, J., Blau, I. E., Niittyvuopio, R., Platzbecker, U., Cornelissen, J. J., Chevallier, P., Srour, M., Stamatovic, D., Martinez-Lopez, J., de Wreede, L., Hayden, P., Hernández-Boluda, J. C., and Yakoub-Agha, I.

Published
2021
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8. Improving post-cellular therapy care with an interdisciplinary, cross-regional, cross-sectoral, digital-aided approach

Author

Schneider, M, Egger-Heidrich, K, Müller, G, Schmädig, R, Schmidt, F, Leppla, L, Geest, SD, Teynor, A, Wolfien, M, Sedlmayr, M, Hänel, M, Vucinic, V, Schmitt, J, Platzbecker, U, Schetelig, J, Bornhäuser, M, Middeke, JM, Schneider, M, Egger-Heidrich, K, Müller, G, Schmädig, R, Schmidt, F, Leppla, L, Geest, SD, Teynor, A, Wolfien, M, Sedlmayr, M, Hänel, M, Vucinic, V, Schmitt, J, Platzbecker, U, Schetelig, J, Bornhäuser, M, and Middeke, JM

Published
2024

9. EFFICACY AND SAFETY OF LUSPATERCEPT VERSUS EPOETIN ALFA IN ERYTHROPOIESIS-STIMULATING AGENT (ESA)-NAIVE PATIENTS WITH TRANSFUSION-DEPENDENT LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS): FULL ANALYSIS OF THE COMMANDS TRIAL

Author

Garcia-Manero, G, Platzbecker, U, Santini, V, Zeidan, A, Fenaux, P, Komrokji, R, Shortt, J, Valcarcel, D, Jonasova, A, Dimicoli-Salazar, S, Tiong, IS, Lin, C-C, Li, J, Zhang, J, Giuseppi, AC, Kreitz, S, Pozharskaya, V, Keeperman, K, Rose, S, Prebet, T, Degulys, A, Paolini, S, Cluzeau, T, Porta, MD, Garcia-Manero, G, Platzbecker, U, Santini, V, Zeidan, A, Fenaux, P, Komrokji, R, Shortt, J, Valcarcel, D, Jonasova, A, Dimicoli-Salazar, S, Tiong, IS, Lin, C-C, Li, J, Zhang, J, Giuseppi, AC, Kreitz, S, Pozharskaya, V, Keeperman, K, Rose, S, Prebet, T, Degulys, A, Paolini, S, Cluzeau, T, and Porta, MD

Published
2024

10. Outcomes of CMML patients undergoing allo-HCT are significantly worse compared to MDS-a study of the CMWP of the EBMT

Author

Rovó, A, Gras, L, Piepenbroek, B, Kroeger, N, Reinhardt, HC, Radujkovic, A, Blaise, D, Kobbe, G, Niityvuopio, R, Platzbecker, U, Sockel, K, Hunault-Berger, M, Cornelissen, JJ, Forcade, E, Bourhis, JH, Chalandon, Y, Kinsella, F, Nguyen-Quoc, S, Maertens, J, Elmaagacli, A, Mordini, N, Hayden, P, Raj, K, Drozd-Sokolowska, J, de Wreede, LC, Mclornan, DP, Robin, M, Yakoub-Agha, I, Onida, F, Rovó, A, Gras, L, Piepenbroek, B, Kroeger, N, Reinhardt, HC, Radujkovic, A, Blaise, D, Kobbe, G, Niityvuopio, R, Platzbecker, U, Sockel, K, Hunault-Berger, M, Cornelissen, JJ, Forcade, E, Bourhis, JH, Chalandon, Y, Kinsella, F, Nguyen-Quoc, S, Maertens, J, Elmaagacli, A, Mordini, N, Hayden, P, Raj, K, Drozd-Sokolowska, J, de Wreede, LC, Mclornan, DP, Robin, M, Yakoub-Agha, I, and Onida, F

Abstract

Although CMML since long has been separated from MDS, many studies continue to evaluate the outcomes of both diseases after hematopoietic cell transplantation (allo-HCT) together. Data evaluating outcomes of a large CMML cohort after allo-HCT compared to MDS are limited. We aim to compare outcomes of CMML to MDS patients who underwent allo-HCT between 2010 and 2018. Patients ≥18 years with CMML and MDS undergoing allo-HCT reported to the EBMT registry were analyzed. Progression to AML before allo-HCT was an exclusion criterion. Overall survival (OS), progression/relapse-free survival (PFS), relapse incidence (including progression) (REL), and non-relapse mortality (NRM) were evaluated in univariable and multivariable (MVA) Cox proportional hazard models including interaction terms between disease and confounders. In total, 10832 patients who underwent allo-HCT were included in the study, there were a total of 1466 CMML, and 9366 MDS. The median age at time of allo-HCT in CMML (median 60.5, IQR 54.3–65.2 years) was significantly higher than in the MDS cohort (median 58.8, IQR 50.2–64.5 years; p < .001). A significantly higher percentage of CMML patients were male (69.4%) compared to MDS (61.2%; p < .001). There were no clinically meaningful differences in the distribution of Karnofsky score, Sorror HCT-CI score at allo-HCT, and donor type, between the CMML and MDS patients. RIC platforms were utilized in 63.9% of CMML allo-HCT, and in 61.4% of MDS patients (p = .08). In univariable analyses, we found that OS, PFS, and REL were significantly worse in CMML when compared with MDS (all p < .0001), whereas no significant difference was observed in NRM (p = .77). In multivariable analyses, the HR comparing MDS versus CMML for OS was 0.81 (95% CI, 0.74–0.88, p < .001), PFS 0.76 (95% CI 0.70–0.82, p < .001), relapse 0.66 (95% CI 0.59–0.74, p < .001), and NRM 0.87 (95% CI 0.78–0.98, p = .02), respectively. The association between baseline variables and outco

Published
2024

11. Impact of post-transplant cyclophosphamide (PTCy)-based prophylaxis in matched sibling donor allogeneic haematopoietic cell transplantation for patients with myelodysplastic syndrome: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT

Author

Salas, M. Q., Eikema, D. -J., Koster, L., Maertens, J., Passweg, J., Finke, J., Broers, A. E. C., Koc, Y., Kroger, N., Ozkurt, Z. N., Pascual-Cascon, M. J., Platzbecker, U., Van Gorkom, G., Schroeder, T., Lopez-Lorenzo, J. L., Martino, Michelangelo, Chiusolo, Patrizia, Kaufmann, M., Onida, F., Gurnari, C., Scheid, C., Drozd-Sokolowska, J., Raj, K., Robin, M., Mclornan, D. P., Martino M., Chiusolo P. (ORCID:0000-0002-1355-1587), Salas, M. Q., Eikema, D. -J., Koster, L., Maertens, J., Passweg, J., Finke, J., Broers, A. E. C., Koc, Y., Kroger, N., Ozkurt, Z. N., Pascual-Cascon, M. J., Platzbecker, U., Van Gorkom, G., Schroeder, T., Lopez-Lorenzo, J. L., Martino, Michelangelo, Chiusolo, Patrizia, Kaufmann, M., Onida, F., Gurnari, C., Scheid, C., Drozd-Sokolowska, J., Raj, K., Robin, M., Mclornan, D. P., Martino M., and Chiusolo P. (ORCID:0000-0002-1355-1587)

Abstract

We retrospectively compared outcomes of 404 MDS patients undergoing 1st matched sibling donor allo-HCT receiving either PTCy-based (n = 66) or other “conventional prophylaxis” (n = 338; mostly calcineurin inhibitor + methotrexate or MMF). Baseline characteristics were balanced, except for higher use of myeloablative regimens in the PTCy group (52.3% vs. 38.2%, p = 0.047). Incidences of neutrophil (Day +28: 89% vs. 97%, p = 0.011) and platelet (Day +100: 89% vs. 97%, p < 0.001) engraftment were lower for PTCy-based. Day +100 cumulative incidences of grade II–IV and III–IV aGVHD, and 5-year CI of extensive cGVHD were 32%, 18% and 18% for PTCy-based and 25% (p = 0.3), 13% (p = 0.4) and 31% (p = 0.09) for the conventional cohort. Five-year OS (51% vs. 52%, p = 0.6) and GRFS (33% vs. 25%, p = 0.6) were similar between groups. Patients receiving PTCy had a trend to a lower cumulative incidence of relapse (20% vs. 33%, p = 0.06), not confirmed on multivariable analysis (p = 0.3). Although higher NRM rates were observed in patients receiving PTCy (32% vs. 21%, p = 0.02) on univariate analysis, this was not confirmed on multivariate analysis (HR 1.46, p = 0.18), and there was no resultant effect on OS (HR 1.20, p = 0.5). Based on these data, PTCy prophylaxis appears to be an attractive option for patients with MDS undergoing MSD allo-HCT.

Published
2024

12. Therapy-related myelodysplastic syndromes deserve specific diagnostic sub-classification and risk-stratification—an approach to classification of patients with t-MDS

Author

Kuendgen, A., Nomdedeu, M., Tuechler, H., Garcia-Manero, G., Komrokji, R. S., Sekeres, M. A., Della Porta, M. G., Cazzola, M., DeZern, A. E., Roboz, G. J., Steensma, D. P., Van de Loosdrecht, A. A., Schlenk, R. F., Grau, J., Calvo, X., Blum, S., Pereira, A., Valent, P., Costa, D., Giagounidis, A., Xicoy, B., Döhner, H., Platzbecker, U., Pedro, C., Lübbert, M., Oiartzabal, I., Díez-Campelo, M., Cedena, M. T., Machherndl-Spandl, S., López-Pavía, M., Baldus, C. D., Martinez-de-Sola, M., Stauder, R., Merchan, B., List, A., Ganster, C., Schroeder, T., Voso, M. T., Pfeilstöcker, M., Sill, H., Hildebrandt, B., Esteve, J., Nomdedeu, B., Cobo, F., Haas, R., Sole, F., Germing, U., Greenberg, P. L., Haase, D., and Sanz, G.

Published
2021
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13. CO77 Impact of Imetelstat Treatment on PROs and Health Care Resource Utilization in Heavily Transfused Non-Del(5Q) Lower-Risk Myelodysplastic Syndromes Relapsed/Refractory to Erythropoiesis Stimulating Agents: IMerge Phase 3 Trial

Author

Sekeres, M., primary, Santini, V., additional, Diez-Campelo, M., additional, Komrokji, R., additional, Fenaux, P., additional, Savona, M., additional, Madanat, Y., additional, Valcárcel-Ferreiras, D., additional, Illmer, T., additional, Jonášová, A., additional, Bělohlávková, P., additional, Regnault, A., additional, Creel, K., additional, Sengupta, N., additional, Sherman, L., additional, Berry, T., additional, Dougherty, S., additional, Shah, S., additional, Xia, Q., additional, Sun, L., additional, Wan, Y., additional, Huang, F., additional, Ikin, A., additional, Navada, S., additional, Feller, F., additional, Zeidan, A., additional, and Platzbecker, U., additional

Published
2023
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14. Synthetic Data Generation by Artificial Intelligence to Accelerate Research and Precision Medicine in Hematology

Author

D'Amico, S, Dall'Olio, D, Sala, C, Dall'Olio, L, Sauta, E, Zampini, M, Asti, G, Lanino, L, Maggioni, G, Campagna, A, Ubezio, M, Russo, A, Bicchieri, M, Riva, E, Tentori, C, Travaglino, E, Morandini, P, Savevski, V, Santoro, A, Prada-Luengo, I, Krogh, A, Santini, V, Kordasti, S, Platzbecker, U, Diez-Campelo, M, Fenaux, P, Haferlach, T, Castellani, G, Della Porta, M, D'Amico S., Dall'Olio D., Sala C., Dall'Olio L., Sauta E., Zampini M., Asti G., Lanino L., Maggioni G., Campagna A., Ubezio M., Russo A., Bicchieri M. E., Riva E., Tentori C. A., Travaglino E., Morandini P., Savevski V., Santoro A., Prada-Luengo I., Krogh A., Santini V., Kordasti S., Platzbecker U., Diez-Campelo M., Fenaux P., Haferlach T., Castellani G., Della Porta M. G., D'Amico, S, Dall'Olio, D, Sala, C, Dall'Olio, L, Sauta, E, Zampini, M, Asti, G, Lanino, L, Maggioni, G, Campagna, A, Ubezio, M, Russo, A, Bicchieri, M, Riva, E, Tentori, C, Travaglino, E, Morandini, P, Savevski, V, Santoro, A, Prada-Luengo, I, Krogh, A, Santini, V, Kordasti, S, Platzbecker, U, Diez-Campelo, M, Fenaux, P, Haferlach, T, Castellani, G, Della Porta, M, D'Amico S., Dall'Olio D., Sala C., Dall'Olio L., Sauta E., Zampini M., Asti G., Lanino L., Maggioni G., Campagna A., Ubezio M., Russo A., Bicchieri M. E., Riva E., Tentori C. A., Travaglino E., Morandini P., Savevski V., Santoro A., Prada-Luengo I., Krogh A., Santini V., Kordasti S., Platzbecker U., Diez-Campelo M., Fenaux P., Haferlach T., Castellani G., and Della Porta M. G.

Abstract

PURPOSE: Synthetic data are artificial data generated without including any real patient information by an algorithm trained to learn the characteristics of a real source data set and became widely used to accelerate research in life sciences. We aimed to (1) apply generative artificial intelligence to build synthetic data in different hematologic neoplasms; (2) develop a synthetic validation framework to assess data fidelity and privacy preservability; and (3) test the capability of synthetic data to accelerate clinical/translational research in hematology. METHODS: A conditional generative adversarial network architecture was implemented to generate synthetic data. Use cases were myelodysplastic syndromes (MDS) and AML: 7,133 patients were included. A fully explainable validation framework was created to assess fidelity and privacy preservability of synthetic data. RESULTS: We generated MDS/AML synthetic cohorts (including information on clinical features, genomics, treatment, and outcomes) with high fidelity and privacy performances. This technology allowed resolution of lack/incomplete information and data augmentation. We then assessed the potential value of synthetic data on accelerating research in hematology. Starting from 944 patients with MDS available since 2014, we generated a 300% augmented synthetic cohort and anticipated the development of molecular classification and molecular scoring system obtained many years later from 2,043 to 2,957 real patients, respectively. Moreover, starting from 187 MDS treated with luspatercept into a clinical trial, we generated a synthetic cohort that recapitulated all the clinical end points of the study. Finally, we developed a website to enable clinicians generating high-quality synthetic data from an existing biobank of real patients. CONCLUSION: Synthetic data mimic real clinical-genomic features and outcomes, and anonymize patient information. The implementation of this technology allows to increase the scientific

Published
2023

15. Integrating biological HLA-DPB1 mismatch models to predict survival after unrelated hematopoietic cell transplantation

Author

Ruggeri, A, de Wreede, L, Muller, C, Crivello, P, Bonneville, E, Petersdorf, E, Socie, G, Dubois, V, Niittyvuopio, R, Perasaari, J, Yakoub-Agha, I, Cornelissen, J, Wieten, L, Gedde-Dahl, T, Forcade, E, Crawley, C, Marsh, S, Gandemer, V, Tholouli, E, Bulabois, C, Huynh, A, Choi, G, Deconinck, E, Itala-Remes, M, Lenhoff, S, Bengtsson, M, Johansson, J, van Gorkom, G, Hoogenboom, J, Vago, L, Rocha, V, Bonini, C, Chabannon, C, Fleischhauer, K, Clausen, J, Holter, W, Kalhs, P, Beguin, Y, Bron, D, Deeren, D, Lung, W, Kerre, T, Poire, X, Selleslag, D, Schroyens, W, Jindra, P, Mayer, J, Vydra, J, Zak, P, Nielsen, B, Sengeloev, H, Kaare, A, Partanen, A, Bay, J, Bertrand, Y, Blaise, D, Bourhis, J, Chevallier, P, Cluzeau, T, Damaj, G, Fegueux, N, Guyotat, D, Hunault-Berger, M, Labussiere-Wallet, H, Leleu, X, Lioure, B, Maury, S, Michel, G, Mohty, M, Rubio, M, Tilly, H, Turlure, P, Bethge, W, Casper, J, Einsele, H, Ganser, A, Kroger, N, Martin, S, Platzbecker, U, Reinhardt, C, Schafer-Eckart, K, Thurner, L, Valerius, T, Wulf, G, Karakasis, D, Spyridonidis, A, Hauser, P, Remenyi, P, Reykdal, S, Mousavi, A, Angelucci, E, Arcese, W, Benedetti, F, Bernasconi, P, Biondi, A, Bonifazi, F, Carella, A, Carluccio, P, Casini, M, Cavanna, L, Ciceri, F, Cimino, G, Corradini, P, Fanin, R, Galieni, P, Grillo, G, Iori, A, La Nasa, G, Locatelli, F, Marotta, G, Martino, M, Mazza, P, Mordini, N, Musso, M, Olivieri, A, Pavone, V, Pane, F, Petrini, M, Pioltelli, P, Rambaldi, A, Ruggeri, M, Saccardi, R, Santarone, S, Scime, R, Sica, S, Tarella, C, Velardi, A, Visani, G, Zecca, M, Tanase, A, Kulagin, A, Savchenko, V, Lopez, C, Amor, A, Lopez, J, Caballero, D, Duarte, R, Cascon, M, Porras, R, Perez-Simon, J, Rovira, M, Sanz, J, Carrete, J, Cammenga, J, Isaksson, C, Mielke, S, Chalandon, Y, Passweg, J, Schanz, U, Meijer, E, Kuball, J, Veelken, J, Apperley, J, Bloor, A, Byrne, J, Carpenter, B, Clark, A, Collin, M, Craddock, C, Gibson, B, Khan, A, Martin, M, Medd, P, Nicholson, E, Orchard, K, Patel, A, Peniket, A, Potter, V, Snowden, J, Wilson, K, Ruggeri A., de Wreede L. C., Muller C. R., Crivello P., Bonneville E. F., Petersdorf E. W., Socie G., Dubois V., Niittyvuopio R., Perasaari J., Yakoub-Agha I., Cornelissen J. J., Wieten L., Gedde-Dahl T., Forcade E., Crawley C. R., Marsh S. G. E., Gandemer V., Tholouli E., Bulabois C. -E., Huynh A., Choi G., Deconinck E., Itala-Remes M., Lenhoff S., Bengtsson M., Johansson J. -E., van Gorkom G., Hoogenboom J. D., Vago L., Rocha V., Bonini C., Chabannon C., Fleischhauer K., Clausen J., Holter W., Kalhs P., Beguin Y., Bron D., Deeren D., Lung W. K., Kerre T., Poire X., Selleslag D., Schroyens W., Jindra P., Mayer J., Vydra J., Zak P., Nielsen B., Sengeloev H., Kaare A., Partanen A., Bay J., Bertrand Y., Blaise D., Bourhis J. H., Chevallier P., Cluzeau T., Damaj G., Fegueux N., Guyotat D., Hunault-Berger M., Labussiere-Wallet H., Leleu X., Lioure B., Maury S., Michel G., Mohty M., Rubio M. T., Tilly H., Turlure P., Bethge W., Casper J., Einsele H., Ganser A., Kroger N., Martin S., Platzbecker U., Reinhardt C., Schafer-Eckart K., Thurner L., Valerius T., Wulf G. G., Karakasis D., Spyridonidis A., Hauser P., Remenyi P., Reykdal S., Mousavi A., Angelucci E., Arcese W., Benedetti F., Bernasconi P., Biondi A., Bonifazi F., Carella A. M., Carluccio P., Casini M., Cavanna L., Ciceri F., Cimino G., Corradini P., Fanin R., Galieni P., Grillo G., Iori A. P., La Nasa G., Locatelli F., Marotta G., Martino M., Mazza P., Mordini N., Musso M., Olivieri A., Pavone V., Pane F., Petrini M., Pioltelli P., Rambaldi A., Ruggeri M., Saccardi R., Santarone S., Scime R., Sica S., Tarella C., Velardi A., Visani G., Zecca M., Tanase A., Kulagin A., Savchenko V., Lopez C. A., Amor A. A., Lopez J. L. B., Caballero D., Duarte R., Cascon M. J. P., Porras R. P., Perez-Simon J. A., Rovira M., Sanz J., Carrete J. P. T., Cammenga J., Isaksson C., Mielke S., Chalandon Y., Passweg J., Schanz U., Meijer E., Kuball J., Veelken J. H., Apperley J., Bloor A., Byrne J., Carpenter B., Clark A., Collin M., Craddock C., Gibson B. E., Khan A., Martin M., Medd P., Nicholson E., Orchard K., Patel A., Peniket A., Potter V., Snowden J., Wilson K. M. O., Ruggeri, A, de Wreede, L, Muller, C, Crivello, P, Bonneville, E, Petersdorf, E, Socie, G, Dubois, V, Niittyvuopio, R, Perasaari, J, Yakoub-Agha, I, Cornelissen, J, Wieten, L, Gedde-Dahl, T, Forcade, E, Crawley, C, Marsh, S, Gandemer, V, Tholouli, E, Bulabois, C, Huynh, A, Choi, G, Deconinck, E, Itala-Remes, M, Lenhoff, S, Bengtsson, M, Johansson, J, van Gorkom, G, Hoogenboom, J, Vago, L, Rocha, V, Bonini, C, Chabannon, C, Fleischhauer, K, Clausen, J, Holter, W, Kalhs, P, Beguin, Y, Bron, D, Deeren, D, Lung, W, Kerre, T, Poire, X, Selleslag, D, Schroyens, W, Jindra, P, Mayer, J, Vydra, J, Zak, P, Nielsen, B, Sengeloev, H, Kaare, A, Partanen, A, Bay, J, Bertrand, Y, Blaise, D, Bourhis, J, Chevallier, P, Cluzeau, T, Damaj, G, Fegueux, N, Guyotat, D, Hunault-Berger, M, Labussiere-Wallet, H, Leleu, X, Lioure, B, Maury, S, Michel, G, Mohty, M, Rubio, M, Tilly, H, Turlure, P, Bethge, W, Casper, J, Einsele, H, Ganser, A, Kroger, N, Martin, S, Platzbecker, U, Reinhardt, C, Schafer-Eckart, K, Thurner, L, Valerius, T, Wulf, G, Karakasis, D, Spyridonidis, A, Hauser, P, Remenyi, P, Reykdal, S, Mousavi, A, Angelucci, E, Arcese, W, Benedetti, F, Bernasconi, P, Biondi, A, Bonifazi, F, Carella, A, Carluccio, P, Casini, M, Cavanna, L, Ciceri, F, Cimino, G, Corradini, P, Fanin, R, Galieni, P, Grillo, G, Iori, A, La Nasa, G, Locatelli, F, Marotta, G, Martino, M, Mazza, P, Mordini, N, Musso, M, Olivieri, A, Pavone, V, Pane, F, Petrini, M, Pioltelli, P, Rambaldi, A, Ruggeri, M, Saccardi, R, Santarone, S, Scime, R, Sica, S, Tarella, C, Velardi, A, Visani, G, Zecca, M, Tanase, A, Kulagin, A, Savchenko, V, Lopez, C, Amor, A, Lopez, J, Caballero, D, Duarte, R, Cascon, M, Porras, R, Perez-Simon, J, Rovira, M, Sanz, J, Carrete, J, Cammenga, J, Isaksson, C, Mielke, S, Chalandon, Y, Passweg, J, Schanz, U, Meijer, E, Kuball, J, Veelken, J, Apperley, J, Bloor, A, Byrne, J, Carpenter, B, Clark, A, Collin, M, Craddock, C, Gibson, B, Khan, A, Martin, M, Medd, P, Nicholson, E, Orchard, K, Patel, A, Peniket, A, Potter, V, Snowden, J, Wilson, K, Ruggeri A., de Wreede L. C., Muller C. R., Crivello P., Bonneville E. F., Petersdorf E. W., Socie G., Dubois V., Niittyvuopio R., Perasaari J., Yakoub-Agha I., Cornelissen J. J., Wieten L., Gedde-Dahl T., Forcade E., Crawley C. R., Marsh S. G. E., Gandemer V., Tholouli E., Bulabois C. -E., Huynh A., Choi G., Deconinck E., Itala-Remes M., Lenhoff S., Bengtsson M., Johansson J. -E., van Gorkom G., Hoogenboom J. D., Vago L., Rocha V., Bonini C., Chabannon C., Fleischhauer K., Clausen J., Holter W., Kalhs P., Beguin Y., Bron D., Deeren D., Lung W. K., Kerre T., Poire X., Selleslag D., Schroyens W., Jindra P., Mayer J., Vydra J., Zak P., Nielsen B., Sengeloev H., Kaare A., Partanen A., Bay J., Bertrand Y., Blaise D., Bourhis J. H., Chevallier P., Cluzeau T., Damaj G., Fegueux N., Guyotat D., Hunault-Berger M., Labussiere-Wallet H., Leleu X., Lioure B., Maury S., Michel G., Mohty M., Rubio M. T., Tilly H., Turlure P., Bethge W., Casper J., Einsele H., Ganser A., Kroger N., Martin S., Platzbecker U., Reinhardt C., Schafer-Eckart K., Thurner L., Valerius T., Wulf G. G., Karakasis D., Spyridonidis A., Hauser P., Remenyi P., Reykdal S., Mousavi A., Angelucci E., Arcese W., Benedetti F., Bernasconi P., Biondi A., Bonifazi F., Carella A. M., Carluccio P., Casini M., Cavanna L., Ciceri F., Cimino G., Corradini P., Fanin R., Galieni P., Grillo G., Iori A. P., La Nasa G., Locatelli F., Marotta G., Martino M., Mazza P., Mordini N., Musso M., Olivieri A., Pavone V., Pane F., Petrini M., Pioltelli P., Rambaldi A., Ruggeri M., Saccardi R., Santarone S., Scime R., Sica S., Tarella C., Velardi A., Visani G., Zecca M., Tanase A., Kulagin A., Savchenko V., Lopez C. A., Amor A. A., Lopez J. L. B., Caballero D., Duarte R., Cascon M. J. P., Porras R. P., Perez-Simon J. A., Rovira M., Sanz J., Carrete J. P. T., Cammenga J., Isaksson C., Mielke S., Chalandon Y., Passweg J., Schanz U., Meijer E., Kuball J., Veelken J. H., Apperley J., Bloor A., Byrne J., Carpenter B., Clark A., Collin M., Craddock C., Gibson B. E., Khan A., Martin M., Medd P., Nicholson E., Orchard K., Patel A., Peniket A., Potter V., Snowden J., and Wilson K. M. O.

Published
2023

17. Artificial intelligence methods to estimate overall mortality and non-relapse mortality following allogeneic HCT in the modern era: an EBMT-TCWP study

Author

Mussetti, A., primary, Rius-Sansalvador, B., additional, Moreno, V., additional, Peczynski, C., additional, Polge, E., additional, Galimard, J. E., additional, Kröger, N., additional, Blaise, D., additional, Peffault de Latour, R., additional, Kulagin, A., additional, Mousavi, A., additional, Stelljes, M., additional, Hamladji, R. M., additional, Middeke, J. M., additional, Salmenniemi, U., additional, Sengeloev, H., additional, Forcade, E., additional, Platzbecker, U., additional, Reményi, P., additional, Angelucci, E., additional, Chevallier, P., additional, Yakoub-Agha, I., additional, Craddock, C., additional, Ciceri, F., additional, Schroeder, T., additional, Aljurf, M., additional, Ch, Koenecke, additional, Moiseev, I., additional, Penack, O., additional, Schoemans, H., additional, Mohty, M., additional, Glass, B., additional, Sureda, A., additional, Basak, G., additional, and Peric, Z., additional

Published
2023
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20. Intermediate-dose cytarabine plus mitoxantrone versus standard-dose cytarabine plus daunorubicin for acute myeloid leukemia in elderly patients

Author

Röllig, C., Kramer, M., Gabrecht, M., Hänel, M., Herbst, R., Kaiser, U., Schmitz, N., Kullmer, J., Fetscher, S., Link, H., Mantovani-Löffler, L., Krümpelmann, U., Neuhaus, T., Heits, F., Einsele, H., Ritter, B., Bornhäuser, M., Schetelig, J., Thiede, C., Mohr, B., Schaich, M., Platzbecker, U., Schäfer-Eckart, K., Krämer, A., Berdel, W.E., Serve, H., Ehninger, G., and Schuler, U.S.

Published
2018
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22. Cellular dynamics and their impact on outcome in patients with mantle cell lymphoma during treatment with chimeric antigen receptor (CAR) T cells

Author

Weiss, R., primary, Fernandez, C. P., additional, Boldt, A., additional, Hoffmann, S., additional, Krauss, S., additional, Bach, E., additional, Kirchberg, J., additional, Kurch, L., additional, Merz, M., additional, Metzeler, K., additional, Herling, C., additional, Jentzsch, M., additional, Schwind, S., additional, Petermann, N., additional, Franz, P., additional, Herling, M., additional, Denecke, T., additional, Sabri, O., additional, Fricke, S., additional, Kluge, R., additional, Sack, U., additional, Franke, G., additional, Koehl, U., additional, Platzbecker, U., additional, Georgi, T., additional, and Vucinic, V., additional

Published
2023
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25. Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: IMPROVED PROGNOSIS OF PATIENTS WITH MDS OVER FOUR DECADES: ANALYSIS FROM THE GERMAN MDS REGISTRY

Author

Kasprzak, A., primary, Strupp, C., additional, Haase, D., additional, Ganster, C., additional, Pfeilstöcker, M., additional, Blum, S., additional, Hofmann, W.-K., additional, Platzbecker, U., additional, Valent, P., additional, Beier, F., additional, Götze, K., additional, Parmentier, S., additional, Lübbert, M., additional, Stauder, R., additional, Thol, F., additional, Giagounidis, A., additional, Tüchler, H., additional, Lauseker, M., additional, Gattermann, N., additional, Kobbe, G., additional, Nachtkamp, K., additional, and Germing, U., additional

Published
2023
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26. Topic: AS04-MDS Biology and Pathogenesis/AS04d-Somatic mutations: TP53 ALLELIC STATE DID NOT INFLUENCE THE PROGNOSIS IN MYELODYSPLASTIC SYNDROMES (MDS) WITH 5Q DELETION

Author

Montoro, M.J., primary, Palomo, L., additional, Haferlach, C., additional, Fuster-Tormo, F., additional, Meggendorfer, M., additional, Xicoy, B., additional, Schulz, F., additional, Della Porta, M., additional, Gonzalez, T., additional, López-Cadenas, F., additional, Acha, P., additional, Diez-Campelo, M., additional, Jerez, A., additional, Such, E., additional, Bernal, T., additional, Santini, V., additional, Platzbecker, U., additional, Germing, U., additional, Solé, F., additional, Haferlach, T., additional, and Valcárcel, D., additional

Published
2023
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28. Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: INTERMEDIATE RISK KARYOTYPE BY IPSS-R CAN BE SEPARATED INTO 5 PROGNOSTICALLY DISTINCT SUBGROUPS IN MDS PATIENTS

Author

Nachtkamp, K., primary, Flatten, V., additional, Hildebrandt, B., additional, Kasprzak, A., additional, Schulz, F., additional, Strupp, C., additional, Haase, D., additional, Ganster, C., additional, Shirneshan, K., additional, Rittscher, K., additional, Götze, K., additional, Hofmann, W.-K., additional, Platzbecker, U., additional, Pfeilstöcker, M., additional, Valent, P., additional, Blum, S., additional, Lübbert, M., additional, Parmentier, S., additional, Beier, F., additional, Sockel, K., additional, Nusch, A., additional, Gattermann, N., additional, and Germing, U., additional

Published
2023
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29. Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: A STEP TOWARDS IDENTIFICATION AND CAUSAL INTERPRETATION OF THERAPY-RELATED MDS (T-MDS)

Author

Kuendgen, A., primary, Nomdedeu, M., additional, Tuechler, H., additional, Garcia-Manero, G., additional, Komrokji, R., additional, Sekeres, M., additional, Della Porta, M., additional, Cazzola, M., additional, Dezern, A., additional, Roboz, G., additional, Steensma, D., additional, Van De Loosdrecht, A., additional, Malcovati, L., additional, Schlenk, R., additional, Grau, J., additional, Calvo, X., additional, Blum, S., additional, Pereira, A., additional, Valent, P., additional, Costa, D., additional, Giagounidis, A., additional, Xicoy, B., additional, Döhner, H., additional, Platzbecker, U., additional, Pedro, C., additional, Lübbert, M., additional, Oiartzabal, I., additional, Diez-Campelo, M., additional, Cedena, M.T., additional, Machherndl-Spandl, S., additional, Lopez-Pavia, M., additional, Martinez-De-Sola, M., additional, Stauder, R., additional, Merchan, B., additional, Ganster, C., additional, Schroeder, T., additional, Voso, M.T., additional, Pfeilstöcker, M., additional, Sill, H., additional, Hildebrandt, B., additional, Esteve, J., additional, Cobo, F., additional, Solé, F., additional, Germing, U., additional, Greenberg, P., additional, Haase, D., additional, and Sanz, G., additional

Published
2023
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30. TARGETING THE DEFECTIVE COA PATHWAY TO IMPROVE ERYTHROPOIESIS IN SF3B1-MUTANT MDS-RS PATIENTS

Author

Philippe, C., primary, Mian, S., additional, Maniati, E., additional, Protopapa, P., additional, Bergot, T., additional, Piganeau, M., additional, Nemkov, T., additional, Di Bella, D., additional, Morales, V., additional, Finch, A., additional, D’Alessandro, A., additional, Bianchi, K., additional, Wang, J., additional, Gallipoli, P., additional, Kordasti, S., additional, Kubasch, A.S., additional, Cross, M., additional, Platzbecker, U., additional, Wiseman, D., additional, Bonnet, D., additional, Bernard, D., additional, Gribben, J., additional, and Rouault-Pierre, K., additional

Published
2023
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31. Immunotherapy: IN-DEPTH CHARACTERIZATION OF THE IMMUNE MICROENVIRONMENT OF PATIENTS UNDERGOING CAR T CELL THERAPY

Author

Weirauch, U., primary, Alb, M., additional, Weiß, R., additional, Löffler, D., additional, Kopfnagel, V., additional, Blumert, C., additional, Düll, J., additional, Scheller, L., additional, Boldt, A., additional, Vucinic, V., additional, Platzbecker, U., additional, Köhl, U., additional, Hudecek, M., additional, and Reiche, K., additional

Published
2023
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32. Topic: AS01-Diagnosis/AS01c-Molecular aberrations (cytogenetic, genetic, gene expression): GENOMIC CLASSIFICATION OF MYELODYSPLASTIC SYNDROMES

Author

Bernard, E., primary, Hasserjian, R., additional, Greenberg, P., additional, Ossa, J. Arango, additional, Creignou, M., additional, Nannya, Y., additional, Tuechler, H., additional, Medina-Martinez, J., additional, Levine, M., additional, Jädersten, M., additional, Germing, U., additional, Sanz, G., additional, Van De Loosdrecht, A., additional, Kosmider, O., additional, Follo, M., additional, Thol, F., additional, Zamora, L., additional, Pinheiro, R., additional, Pellagatti, A., additional, Elias, H., additional, Haase, D., additional, Ganster, C., additional, Ades, L., additional, Tobiasson, M., additional, Palomo, L., additional, Della Porta, M., additional, Fenaux, P., additional, Belickova, M., additional, Savona, M., additional, Klimek, V., additional, Santos, F., additional, Boultwood, J., additional, Kotsianidis, I., additional, Santini, V., additional, Solé, F., additional, Platzbecker, U., additional, Heuser, M., additional, Valent, P., additional, Finelli, C., additional, Voso, M.T., additional, Shih, L.-Y., additional, Fontenay, M., additional, Jansen, J., additional, Cervera-Zamora, J., additional, Gattermann, N., additional, Ebert, B., additional, Bejar, R., additional, Malcovati, L., additional, Cazzola, M., additional, Ogawa, S., additional, Hellstrom-Lindberg, E., additional, and Papaemmanuil, E., additional

Published
2023
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34. Topic: AS03-Health Economics & Outcome Research/AS03a-Cost of care: ARE WE READY TO PERFORM NGS FOR ALL MDS PATIENTS ?

Author

Santini, V., primary, Bejar, R., additional, Belli, C., additional, Buckstein, R., additional, Campelo, M., additional, Dezern, A., additional, Fontenay, M., additional, Griffiths, E., additional, Grille, S., additional, Haferlach, T., additional, Iastrebner, M., additional, Kosmider, O., additional, Magalhaes, S., additional, Mittelman, M., additional, Platzbecker, U., additional, Wei, A., additional, and Zeidan, A., additional

Published
2023
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35. Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes.

Author

Zeidan, A.M., Platzbecker, U., Bewersdorf, J.P., Stahl, M., Adès, L., Borate, U., Bowen, D., Buckstein, R., Brunner, A., Carraway, H.E., Daver, N., Díez-Campelo, M., Witte, T.J. de, DeZern, A.E., Efficace, F., Garcia-Manero, G., Garcia, J.S., Germing, U., Giagounidis, A., Griffiths, E.A., Hasserjian, R.P., Hellström-Lindberg, E., Iastrebner, M., Komrokji, R., Kulasekararaj, A.G., Malcovati, L., Miyazaki, Y., Odenike, O., Santini, V., Sanz, G., Scheinberg, P., Stauder, R., Loosdrecht, A.A. van de, Wei, A.H., Sekeres, M.A., Fenaux, P., Zeidan, A.M., Platzbecker, U., Bewersdorf, J.P., Stahl, M., Adès, L., Borate, U., Bowen, D., Buckstein, R., Brunner, A., Carraway, H.E., Daver, N., Díez-Campelo, M., Witte, T.J. de, DeZern, A.E., Efficace, F., Garcia-Manero, G., Garcia, J.S., Germing, U., Giagounidis, A., Griffiths, E.A., Hasserjian, R.P., Hellström-Lindberg, E., Iastrebner, M., Komrokji, R., Kulasekararaj, A.G., Malcovati, L., Miyazaki, Y., Odenike, O., Santini, V., Sanz, G., Scheinberg, P., Stauder, R., Loosdrecht, A.A. van de, Wei, A.H., Sekeres, M.A., and Fenaux, P.

Abstract

Item does not contain fulltext, Myelodysplastic syndromes/myelodysplastic neoplasms (MDS) are associated with variable clinical presentations and outcomes. The initial response criteria developed by the International Working Group (IWG) in 2000 have been used in clinical practice, clinical trials, regulatory reviews, and drug labels. Although the IWG criteria were revised in 2006 and 2018 (the latter focusing on lower-risk disease), limitations persist in their application to higher-risk MDS (HR-MDS) and their ability to fully capture the clinical benefits of novel investigational drugs or serve as valid surrogates for longer-term clinical end points (eg, overall survival). Further, issues related to the ambiguity and practicality of some criteria lead to variability in interpretation and interobserver inconsistency in reporting results from the same sets of data. Thus, we convened an international panel of 36 MDS experts and used an established modified Delphi process to develop consensus recommendations for updated response criteria that would be more reflective of patient-centered and clinically relevant outcomes in HR-MDS. Among others, the IWG 2023 criteria include changes in the hemoglobin threshold for complete remission (CR), the introduction of CR with limited count recovery and CR with partial hematologic recovery as provisional response criteria, the elimination of marrow CR, and specific recommendations for the standardization of time-to-event end points and the derivation and reporting of responses. The updated criteria should lead to a better correlation between patient-centered outcomes and clinical trial results in an era of multiple emerging new agents with novel mechanisms of action.

Published
2023

36. Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes

Author

Sauta, E, Robin, M, Bersanelli, M, Travaglino, E, Meggendorfer, M, Zhao, L, Caballero Berrocal, J, Sala, C, Maggioni, G, Bernardi, M, Di Grazia, C, Vago, L, Rivoli, G, Borin, L, D'Amico, S, Tentori, C, Ubezio, M, Campagna, A, Russo, A, Mannina, D, Lanino, L, Chiusolo, P, Giaccone, L, Voso, M, Riva, M, Oliva, E, Zampini, M, Riva, E, Nibourel, O, Bicchieri, M, Bolli, N, Rambaldi, A, Passamonti, F, Savevski, V, Santoro, A, Germing, U, Kordasti, S, Santini, V, Diez-Campelo, M, Sanz, G, Sole, F, Kern, W, Platzbecker, U, Ades, L, Fenaux, P, Haferlach, T, Castellani, G, Della Porta, M, Sauta, Elisabetta, Robin, Marie, Bersanelli, Matteo, Travaglino, Erica, Meggendorfer, Manja, Zhao, Lin-Pierre, Caballero Berrocal, Juan Carlos, Sala, Claudia, Maggioni, Giulia, Bernardi, Massimo, Di Grazia, Carmen, Vago, Luca, Rivoli, Giulia, Borin, Lorenza, D'Amico, Saverio, Tentori, Cristina Astrid, Ubezio, Marta, Campagna, Alessia, Russo, Antonio, Mannina, Daniele, Lanino, Luca, Chiusolo, Patrizia, Giaccone, Luisa, Voso, Maria Teresa, Riva, Marta, Oliva, Esther Natalie, Zampini, Matteo, Riva, Elena, Nibourel, Olivier, Bicchieri, Marilena, Bolli, Niccolo', Rambaldi, Alessandro, Passamonti, Francesco, Savevski, Victor, Santoro, Armando, Germing, Ulrich, Kordasti, Shahram, Santini, Valeria, Diez-Campelo, Maria, Sanz, Guillermo, Sole, Francesc, Kern, Wolfgang, Platzbecker, Uwe, Ades, Lionel, Fenaux, Pierre, Haferlach, Torsten, Castellani, Gastone, Della Porta, Matteo Giovanni, Sauta, E, Robin, M, Bersanelli, M, Travaglino, E, Meggendorfer, M, Zhao, L, Caballero Berrocal, J, Sala, C, Maggioni, G, Bernardi, M, Di Grazia, C, Vago, L, Rivoli, G, Borin, L, D'Amico, S, Tentori, C, Ubezio, M, Campagna, A, Russo, A, Mannina, D, Lanino, L, Chiusolo, P, Giaccone, L, Voso, M, Riva, M, Oliva, E, Zampini, M, Riva, E, Nibourel, O, Bicchieri, M, Bolli, N, Rambaldi, A, Passamonti, F, Savevski, V, Santoro, A, Germing, U, Kordasti, S, Santini, V, Diez-Campelo, M, Sanz, G, Sole, F, Kern, W, Platzbecker, U, Ades, L, Fenaux, P, Haferlach, T, Castellani, G, Della Porta, M, Sauta, Elisabetta, Robin, Marie, Bersanelli, Matteo, Travaglino, Erica, Meggendorfer, Manja, Zhao, Lin-Pierre, Caballero Berrocal, Juan Carlos, Sala, Claudia, Maggioni, Giulia, Bernardi, Massimo, Di Grazia, Carmen, Vago, Luca, Rivoli, Giulia, Borin, Lorenza, D'Amico, Saverio, Tentori, Cristina Astrid, Ubezio, Marta, Campagna, Alessia, Russo, Antonio, Mannina, Daniele, Lanino, Luca, Chiusolo, Patrizia, Giaccone, Luisa, Voso, Maria Teresa, Riva, Marta, Oliva, Esther Natalie, Zampini, Matteo, Riva, Elena, Nibourel, Olivier, Bicchieri, Marilena, Bolli, Niccolo', Rambaldi, Alessandro, Passamonti, Francesco, Savevski, Victor, Santoro, Armando, Germing, Ulrich, Kordasti, Shahram, Santini, Valeria, Diez-Campelo, Maria, Sanz, Guillermo, Sole, Francesc, Kern, Wolfgang, Platzbecker, Uwe, Ades, Lionel, Fenaux, Pierre, Haferlach, Torsten, Castellani, Gastone, and Della Porta, Matteo Giovanni

Abstract

Purpose: Myelodysplastic syndromes (MDS) are heterogeneous myeloid neoplasms in which a risk-adapted treatment strategy is needed. Recently, a new clinical-molecular prognostic model, the Molecular International Prognostic Scoring System (IPSS-M) was proposed to improve the prediction of clinical outcome of the currently available tool (Revised International Prognostic Scoring System [IPSS-R]). We aimed to provide an extensive validation of IPSS-M. Methods: A total of 2,876 patients with primary MDS from the GenoMed4All consortium were retrospectively analyzed. Results: IPSS-M improved prognostic discrimination across all clinical end points with respect to IPSS-R (concordance was 0.81 v 0.74 for overall survival and 0.89 v 0.76 for leukemia-free survival, respectively). This was true even in those patients without detectable gene mutations. Compared with the IPSS-R based stratification, the IPSS-M risk group changed in 46% of patients (23.6% and 22.4% of subjects were upstaged and downstaged, respectively).In patients treated with hematopoietic stem cell transplantation (HSCT), IPSS-M significantly improved the prediction of the risk of disease relapse and the probability of post-transplantation survival versus IPSS-R (concordance was 0.76 v 0.60 for overall survival and 0.89 v 0.70 for probability of relapse, respectively). In high-risk patients treated with hypomethylating agents (HMA), IPSS-M failed to stratify individual probability of response; response duration and probability of survival were inversely related to IPSS-M risk.Finally, we tested the accuracy in predicting IPSS-M when molecular information was missed and we defined a minimum set of 15 relevant genes associated with high performance of the score. Conclusion: IPSS-M improves MDS prognostication and might result in a more effective selection of candidates to HSCT. Additional factors other than gene mutations can be involved in determining HMA sensitivity. The definition of a minimum set of relevan

Published
2023

37. Integrating biological HLA-DPB1 mismatch models to predict survival after unrelated hematopoietic cell transplantation

Author

Ruggeri, A., de Wreede, L. C., Muller, C. R., Crivello, P., Bonneville, E. F., Petersdorf, E. W., Socie, G., Dubois, V., Niittyvuopio, R., Perasaari, J., Yakoub-Agha, I., Cornelissen, J. J., Wieten, L., Gedde-Dahl, T., Forcade, E., Crawley, C. R., Marsh, S. G. E., Gandemer, V., Tholouli, E., Bulabois, C. -E., Huynh, A., Choi, G., Deconinck, E., Itala-Remes, M., Lenhoff, S., Bengtsson, M., Johansson, J. -E., van Gorkom, G., Hoogenboom, J. D., Vago, L., Rocha, V., Bonini, C., Chabannon, C., Fleischhauer, K., Clausen, J., Holter, W., Kalhs, P., Beguin, Y., Bron, D., Deeren, D., Lung, W. K., Kerre, T., Poire, X., Selleslag, D., Schroyens, W., Jindra, P., Mayer, J., Vydra, J., Zak, P., Nielsen, B., Sengeloev, H., Kaare, A., Partanen, A., Bay, J., Bertrand, Y., Blaise, D., Bourhis, J. H., Chevallier, P., Cluzeau, T., Damaj, G., Fegueux, N., Guyotat, D., Hunault-Berger, M., Labussiere-Wallet, H., Leleu, X., Lioure, B., Maury, S., Michel, G., Mohty, M., Rubio, M. T., Tilly, H., Turlure, P., Bethge, W., Casper, J., Einsele, H., Ganser, A., Kroger, N., Martin, S., Platzbecker, U., Reinhardt, C., Schafer-Eckart, K., Thurner, L., Valerius, T., Wulf, G. G., Karakasis, D., Spyridonidis, A., Hauser, P., Remenyi, P., Reykdal, S., Mousavi, A., Angelucci, E., Arcese, W., Benedetti, F., Bernasconi, P., Biondi, A., Bonifazi, F., Carella, A. M., Carluccio, P., Casini, M., Cavanna, L., Ciceri, F., Cimino, G., Corradini, P., Fanin, R., Galieni, P., Grillo, G., Iori, A. P., La Nasa, G., Locatelli, Franco, Marotta, G., Martino, M., Mazza, P., Mordini, N., Musso, M., Olivieri, A., Pavone, V., Pane, F., Petrini, M., Pioltelli, P., Rambaldi, A., Ruggeri, M., Saccardi, R., Santarone, S., Scime, R., Sica, S., Tarella, C., Velardi, A., Visani, G., Zecca, M., Tanase, A., Kulagin, A., Savchenko, V., Lopez, C. A., Amor, A. A., Lopez, J. L. B., Caballero, D., Duarte, R., Cascon, M. J. P., Porras, R. P., Perez-Simon, J. A., Rovira, M., Sanz, J., Carrete, J. P. T., Cammenga, J., Isaksson, C., Mielke, S., Chalandon, Y., Passweg, J., Schanz, U., Meijer, E., Kuball, J., Veelken, J. H., Apperley, J., Bloor, A., Byrne, J., Carpenter, B., Clark, A., Collin, M., Craddock, C., Gibson, B. E., Khan, A., Martin, M., Medd, P., Nicholson, E., Orchard, K., Patel, A., Peniket, A., Potter, V., Snowden, J., Wilson, K. M. O., Locatelli F. (ORCID:0000-0002-7976-3654), Ruggeri, A., de Wreede, L. C., Muller, C. R., Crivello, P., Bonneville, E. F., Petersdorf, E. W., Socie, G., Dubois, V., Niittyvuopio, R., Perasaari, J., Yakoub-Agha, I., Cornelissen, J. J., Wieten, L., Gedde-Dahl, T., Forcade, E., Crawley, C. R., Marsh, S. G. E., Gandemer, V., Tholouli, E., Bulabois, C. -E., Huynh, A., Choi, G., Deconinck, E., Itala-Remes, M., Lenhoff, S., Bengtsson, M., Johansson, J. -E., van Gorkom, G., Hoogenboom, J. D., Vago, L., Rocha, V., Bonini, C., Chabannon, C., Fleischhauer, K., Clausen, J., Holter, W., Kalhs, P., Beguin, Y., Bron, D., Deeren, D., Lung, W. K., Kerre, T., Poire, X., Selleslag, D., Schroyens, W., Jindra, P., Mayer, J., Vydra, J., Zak, P., Nielsen, B., Sengeloev, H., Kaare, A., Partanen, A., Bay, J., Bertrand, Y., Blaise, D., Bourhis, J. H., Chevallier, P., Cluzeau, T., Damaj, G., Fegueux, N., Guyotat, D., Hunault-Berger, M., Labussiere-Wallet, H., Leleu, X., Lioure, B., Maury, S., Michel, G., Mohty, M., Rubio, M. T., Tilly, H., Turlure, P., Bethge, W., Casper, J., Einsele, H., Ganser, A., Kroger, N., Martin, S., Platzbecker, U., Reinhardt, C., Schafer-Eckart, K., Thurner, L., Valerius, T., Wulf, G. G., Karakasis, D., Spyridonidis, A., Hauser, P., Remenyi, P., Reykdal, S., Mousavi, A., Angelucci, E., Arcese, W., Benedetti, F., Bernasconi, P., Biondi, A., Bonifazi, F., Carella, A. M., Carluccio, P., Casini, M., Cavanna, L., Ciceri, F., Cimino, G., Corradini, P., Fanin, R., Galieni, P., Grillo, G., Iori, A. P., La Nasa, G., Locatelli, Franco, Marotta, G., Martino, M., Mazza, P., Mordini, N., Musso, M., Olivieri, A., Pavone, V., Pane, F., Petrini, M., Pioltelli, P., Rambaldi, A., Ruggeri, M., Saccardi, R., Santarone, S., Scime, R., Sica, S., Tarella, C., Velardi, A., Visani, G., Zecca, M., Tanase, A., Kulagin, A., Savchenko, V., Lopez, C. A., Amor, A. A., Lopez, J. L. B., Caballero, D., Duarte, R., Cascon, M. J. P., Porras, R. P., Perez-Simon, J. A., Rovira, M., Sanz, J., Carrete, J. P. T., Cammenga, J., Isaksson, C., Mielke, S., Chalandon, Y., Passweg, J., Schanz, U., Meijer, E., Kuball, J., Veelken, J. H., Apperley, J., Bloor, A., Byrne, J., Carpenter, B., Clark, A., Collin, M., Craddock, C., Gibson, B. E., Khan, A., Martin, M., Medd, P., Nicholson, E., Orchard, K., Patel, A., Peniket, A., Potter, V., Snowden, J., Wilson, K. M. O., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

NO ABSTRACT

Published
2023

38. A sex-informed approach to improve the personalised decision making process in myelodysplastic syndromes: a multicentre, observational cohort study

Author

Maggioni, G, Bersanelli, M, Travaglino, E, Alfonso Piérola, A, Kasprzak, A, Sangerman Montserrat, A, Sauta, E, Sala, C, Matteuzzi, T, Meggendorfer, M, Gnocchi, M, Zhao, L, Astrid Tentori, C, Nachtkamp, K, Dall'Olio, D, Mosca, E, Ubezio, M, Campagna, A, Russo, A, Rivoli, G, Bernardi, M, Borin, L, Teresa Voso, M, Riva, M, Oliva, E, Zampini, M, Riva, E, Saba, E, D'Amico, S, Lanino, L, Tinterri, B, Re, F, Bicchieri, M, Giordano, L, Angelotti, G, Morandini, P, Sophie Kubasch, A, Passamonti, F, Rambaldi, A, Savevski, V, Santoro, A, A van de Loosdrecht, A, Brogi, A, Santini, V, Kordasti, S, Sanz, G, Sole, F, Gattermann, N, Kern, W, Platzbecker, U, Ades, L, Fenaux, P, Haferlach, T, Castellani, G, Germing, U, Diez-Campelo, M, G Della Porta, M, Giulia Maggioni, Matteo Bersanelli, Erica Travaglino, Ana Alfonso Piérola, Annika Kasprzak, Arnan Sangerman Montserrat, Elisabetta Sauta, Claudia Sala, Tommaso Matteuzzi, Manja Meggendorfer, Matteo Gnocchi, Lin-Pierre Zhao, Cristina Astrid Tentori, Kathrin Nachtkamp, Daniele Dall'Olio, Ettore Mosca, Marta Ubezio, Alessia Campagna, Antonio Russo, Giulia Rivoli, Massimo Bernardi, Lorenza Borin, Maria Teresa Voso, Marta Riva, Esther Oliva, Matteo Zampini, Elena Riva, Elena Saba, Saverio D'Amico, Luca Lanino, Benedetta Tinterri, Francesca Re, Marilena Bicchieri, Laura Giordano, Giovanni Angelotti, Pierandrea Morandini, Anne Sophie Kubasch, Francesco Passamonti, Alessandro Rambaldi, Victor Savevski, Armando Santoro, Arjan A van de Loosdrecht, Alice Brogi, Valeria Santini, Shahram Kordasti, Guillermo Sanz, Francesc Sole, Norbert Gattermann, Wolfgang Kern, Uwe Platzbecker, Lionel Ades, Pierre Fenaux, Torsten Haferlach, Gastone Castellani, Ulrich Germing, Maria Diez-Campelo, Matteo G Della Porta, Maggioni, G, Bersanelli, M, Travaglino, E, Alfonso Piérola, A, Kasprzak, A, Sangerman Montserrat, A, Sauta, E, Sala, C, Matteuzzi, T, Meggendorfer, M, Gnocchi, M, Zhao, L, Astrid Tentori, C, Nachtkamp, K, Dall'Olio, D, Mosca, E, Ubezio, M, Campagna, A, Russo, A, Rivoli, G, Bernardi, M, Borin, L, Teresa Voso, M, Riva, M, Oliva, E, Zampini, M, Riva, E, Saba, E, D'Amico, S, Lanino, L, Tinterri, B, Re, F, Bicchieri, M, Giordano, L, Angelotti, G, Morandini, P, Sophie Kubasch, A, Passamonti, F, Rambaldi, A, Savevski, V, Santoro, A, A van de Loosdrecht, A, Brogi, A, Santini, V, Kordasti, S, Sanz, G, Sole, F, Gattermann, N, Kern, W, Platzbecker, U, Ades, L, Fenaux, P, Haferlach, T, Castellani, G, Germing, U, Diez-Campelo, M, G Della Porta, M, Giulia Maggioni, Matteo Bersanelli, Erica Travaglino, Ana Alfonso Piérola, Annika Kasprzak, Arnan Sangerman Montserrat, Elisabetta Sauta, Claudia Sala, Tommaso Matteuzzi, Manja Meggendorfer, Matteo Gnocchi, Lin-Pierre Zhao, Cristina Astrid Tentori, Kathrin Nachtkamp, Daniele Dall'Olio, Ettore Mosca, Marta Ubezio, Alessia Campagna, Antonio Russo, Giulia Rivoli, Massimo Bernardi, Lorenza Borin, Maria Teresa Voso, Marta Riva, Esther Oliva, Matteo Zampini, Elena Riva, Elena Saba, Saverio D'Amico, Luca Lanino, Benedetta Tinterri, Francesca Re, Marilena Bicchieri, Laura Giordano, Giovanni Angelotti, Pierandrea Morandini, Anne Sophie Kubasch, Francesco Passamonti, Alessandro Rambaldi, Victor Savevski, Armando Santoro, Arjan A van de Loosdrecht, Alice Brogi, Valeria Santini, Shahram Kordasti, Guillermo Sanz, Francesc Sole, Norbert Gattermann, Wolfgang Kern, Uwe Platzbecker, Lionel Ades, Pierre Fenaux, Torsten Haferlach, Gastone Castellani, Ulrich Germing, Maria Diez-Campelo, and Matteo G Della Porta

Abstract

BACKGROUND: Sex is a major source of diversity among patients and a sex-informed approach is becoming a new paradigm in precision medicine. We aimed to describe sex diversity in myelodysplastic syndromes in terms of disease genotype, phenotype, and clinical outcome. Moreover, we sought to incorporate sex information into the clinical decision-making process as a fundamental component of patient individuality. METHODS: In this multicentre, observational cohort study, we retrospectively analysed 13 284 patients aged 18 years or older with a diagnosis of myelodysplastic syndrome according to 2016 WHO criteria included in the EuroMDS network (n=2025), International Working Group for Prognosis in MDS (IWG-PM; n=2387), the Spanish Group of Myelodysplastic Syndromes registry (GESMD; n=7687), or the Düsseldorf MDS registry (n=1185). Recruitment periods for these cohorts were between 1990 and 2016. The correlation between sex and genomic features was analysed in the EuroMDS cohort and validated in the IWG-PM cohort. The effect of sex on clinical outcome, with overall survival as the main endpoint, was analysed in the EuroMDS population and validated in the other three cohorts. Finally, novel prognostic models incorporating sex and genomic information were built and validated, and compared to the widely used revised International Prognostic Scoring System (IPSS-R). This study is registered with ClinicalTrials.gov, NCT04889729. FINDINGS: The study included 7792 (58·7%) men and 5492 (41·3%) women. 10 906 (82·1%) patients were White, and race was not reported for 2378 (17·9%) patients. Sex biases were observed at the single-gene level with mutations in seven genes enriched in men (ASXL1, SRSF2, and ZRSR2 p<0·0001 in both cohorts; DDX41 not available in the EuroMDS cohort vs p=0·0062 in the IWG-PM cohort; IDH2 p<0·0001 in EuroMDS vs p=0·042 in IWG-PM; TET2 p=0·031 vs p=0·035; U2AF1 p=0·033 vs p<0·0001) and mutations in two genes were enriched in women (DNMT3A p<0·000

Published
2023

39. Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes

Author

Sauta, E., Robin, M., Bersanelli, M., Travaglino, E., Meggendorfer, M., Zhao, L. -P., Caballero Berrocal, J. C., Sala, C., Maggioni, G., Bernardi, M., Di Grazia, C., Vago, L., Rivoli, G., Borin, L., D'Amico, S., Tentori, C. A., Ubezio, M., Campagna, A., Russo, A., Mannina, D., Lanino, L., Chiusolo, Patrizia, Giaccone, L., Voso, Maria Teresa, Riva, M., Oliva, E. N., Zampini, M., Riva, E., Nibourel, O., Bicchieri, M., Bolli, N., Rambaldi, A., Passamonti, F., Savevski, V., Santoro, A., Germing, U., Kordasti, S., Santini, V., Diez-Campelo, M., Sanz, G., Sole, F., Kern, W., Platzbecker, U., Ades, L., Fenaux, P., Haferlach, T., Castellani, G., Della Porta, M. G., Chiusolo P. (ORCID:0000-0002-1355-1587), Voso M. T., Sauta, E., Robin, M., Bersanelli, M., Travaglino, E., Meggendorfer, M., Zhao, L. -P., Caballero Berrocal, J. C., Sala, C., Maggioni, G., Bernardi, M., Di Grazia, C., Vago, L., Rivoli, G., Borin, L., D'Amico, S., Tentori, C. A., Ubezio, M., Campagna, A., Russo, A., Mannina, D., Lanino, L., Chiusolo, Patrizia, Giaccone, L., Voso, Maria Teresa, Riva, M., Oliva, E. N., Zampini, M., Riva, E., Nibourel, O., Bicchieri, M., Bolli, N., Rambaldi, A., Passamonti, F., Savevski, V., Santoro, A., Germing, U., Kordasti, S., Santini, V., Diez-Campelo, M., Sanz, G., Sole, F., Kern, W., Platzbecker, U., Ades, L., Fenaux, P., Haferlach, T., Castellani, G., Della Porta, M. G., Chiusolo P. (ORCID:0000-0002-1355-1587), and Voso M. T.

Abstract

PURPOSEMyelodysplastic syndromes (MDS) are heterogeneous myeloid neoplasms in which a risk-adapted treatment strategy is needed. Recently, a new clinical-molecular prognostic model, the Molecular International Prognostic Scoring System (IPSS-M) was proposed to improve the prediction of clinical outcome of the currently available tool (Revised International Prognostic Scoring System [IPSS-R]). We aimed to provide an extensive validation of IPSS-M.METHODSA total of 2,876 patients with primary MDS from the GenoMed4All consortium were retrospectively analyzed.RESULTSIPSS-M improved prognostic discrimination across all clinical end points with respect to IPSS-R (concordance was 0.81 v 0.74 for overall survival and 0.89 v 0.76 for leukemia-free survival, respectively). This was true even in those patients without detectable gene mutations. Compared with the IPSS-R based stratification, the IPSS-M risk group changed in 46% of patients (23.6% and 22.4% of subjects were upstaged and downstaged, respectively).In patients treated with hematopoietic stem cell transplantation (HSCT), IPSS-M significantly improved the prediction of the risk of disease relapse and the probability of post-transplantation survival versus IPSS-R (concordance was 0.76 v 0.60 for overall survival and 0.89 v 0.70 for probability of relapse, respectively). In high-risk patients treated with hypomethylating agents (HMA), IPSS-M failed to stratify individual probability of response; response duration and probability of survival were inversely related to IPSS-M risk.Finally, we tested the accuracy in predicting IPSS-M when molecular information was missed and we defined a minimum set of 15 relevant genes associated with high performance of the score.CONCLUSIONIPSS-M improves MDS prognostication and might result in a more effective selection of candidates to HSCT. Additional factors other than gene mutations can be involved in determining HMA sensitivity. The definition of a minimum set of relevant genes may

Published
2023

41. Accuracy of physician assessment of treatment preferences and health status in elderly patients with higher-risk myelodysplastic syndromes

Author

Caocci, G., Voso, M.T., Angelucci, E., Stauder, R., Cottone, F., Abel, G., Nguyen, K., Platzbecker, U., Beyne-Rauzy, O., Gaidano, G., Invernizzi, R., Molica, S., Criscuolo, M., Breccia, M., Lübbert, M., Sanpaolo, G., Buccisano, F., Ricco, A., Palumbo, G.A., Niscola, P., Zhang, H., Fenu, S., La Nasa, G., Mandelli, F., and Efficace, F.

Published
2015
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42. Characteristics and outcome of patients with therapy-related acute promyelocytic leukemia front-line treated with or without arsenic trioxide

Author

Kayser, S, Krzykalla, J, Elliott, M A, Norsworthy, K, Gonzales, P, Hills, R K, Baer, M R, Ráčil, Z, Mayer, J, Novak, J, Žák, P, Szotkowski, T, Grimwade, D, Russell, N H, Walter, R B, Estey, E H, Westermann, J, Görner, M, Benner, A, Krämer, A, Smith, B D, Burnett, A K, Thiede, C, Röllig, C, Ho, A D, Ehninger, G, Schlenk, R F, Tallman, M S, Levis, M J, and Platzbecker, U

Published
2017
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48. P091 - Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: A STEP TOWARDS IDENTIFICATION AND CAUSAL INTERPRETATION OF THERAPY-RELATED MDS (T-MDS)

Author

Kuendgen, A., Nomdedeu, M., Tuechler, H., Garcia-Manero, G., Komrokji, R., Sekeres, M., Della Porta, M., Cazzola, M., Dezern, A., Roboz, G., Steensma, D., Van De Loosdrecht, A., Malcovati, L., Schlenk, R., Grau, J., Calvo, X., Blum, S., Pereira, A., Valent, P., Costa, D., Giagounidis, A., Xicoy, B., Döhner, H., Platzbecker, U., Pedro, C., Lübbert, M., Oiartzabal, I., Diez-Campelo, M., Cedena, M.T., Machherndl-Spandl, S., Lopez-Pavia, M., Martinez-De-Sola, M., Stauder, R., Merchan, B., Ganster, C., Schroeder, T., Voso, M.T., Pfeilstöcker, M., Sill, H., Hildebrandt, B., Esteve, J., Cobo, F., Solé, F., Germing, U., Greenberg, P., Haase, D., and Sanz, G.

Published
2023
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49. P094 - Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: INTERMEDIATE RISK KARYOTYPE BY IPSS-R CAN BE SEPARATED INTO 5 PROGNOSTICALLY DISTINCT SUBGROUPS IN MDS PATIENTS

Author

Nachtkamp, K., Flatten, V., Hildebrandt, B., Kasprzak, A., Schulz, F., Strupp, C., Haase, D., Ganster, C., Shirneshan, K., Rittscher, K., Götze, K., Hofmann, W.-K., Platzbecker, U., Pfeilstöcker, M., Valent, P., Blum, S., Lübbert, M., Parmentier, S., Beier, F., Sockel, K., Nusch, A., Gattermann, N., and Germing, U.

Published
2023
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50. P089 - Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: IMPROVED PROGNOSIS OF PATIENTS WITH MDS OVER FOUR DECADES: ANALYSIS FROM THE GERMAN MDS REGISTRY

Author

Kasprzak, A., Strupp, C., Haase, D., Ganster, C., Pfeilstöcker, M., Blum, S., Hofmann, W.-K., Platzbecker, U., Valent, P., Beier, F., Götze, K., Parmentier, S., Lübbert, M., Stauder, R., Thol, F., Giagounidis, A., Tüchler, H., Lauseker, M., Gattermann, N., Kobbe, G., Nachtkamp, K., and Germing, U.

Published
2023
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