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2. Early combination therapy of COVID-19 in high-risk patients

Author

Orth, Hans Martin, Flasshove, Charlotte, Berger, Moritz, Hattenhauer, Tessa, Biederbick, Kaja D., Mispelbaum, Rebekka, Klein, Uwe, Stemler, Jannik, Fisahn, Matthis, Doleschall, Anna D., Baermann, Ben-Niklas, Koenigshausen, Eva, Tselikmann, Olga, Killer, Alexander, de Angelis, Clara, Gliga, Smaranda, Stegbauer, Johannes, Spuck, Nikolai, Silling, Gerda, Rockstroh, Jürgen K., Strassburg, Christian P., Brossart, Peter, Panse, Jens P., Jensen, Björn-Erik Ole, Luedde, Tom, Boesecke, Christoph, Heine, Annkristin, Cornely, Oliver A., and Monin, Malte B.

Published
2024
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4. Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries

Author

Lübke, Johannes, Christen, Deborah, Schwaab, Juliana, Kaiser, Anne, Naumann, Nicole, Shoumariyeh, Khalid, Jentzsch, Madlen, Sockel, Katja, Schaffrath, Judith, Ayuk, Francis A., Stelljes, Matthias, Hilgendorf, Inken, Sala, Elisa, Kaivers, Jennifer, Schönland, Stefan, Wittke, Christoph, Hertenstein, Bernd, Radsak, Markus, Kaiser, Ulrich, Brückl, Valeska, Kröger, Nicolaus, Brümmendorf, Tim H., Hofmann, Wolf-Karsten, Klein, Stefan, Jost, Edgar, Reiter, Andreas, and Panse, Jens

Published
2024
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6. Evolutionary trajectories of small cell lung cancer under therapy

Author

George, Julie, Maas, Lukas, Abedpour, Nima, Cartolano, Maria, Kaiser, Laura, Fischer, Rieke N., Scheel, Andreas H., Weber, Jan-Philipp, Hellmich, Martin, Bosco, Graziella, Volz, Caroline, Mueller, Christian, Dahmen, Ilona, John, Felix, Alves, Cleidson Padua, Werr, Lisa, Panse, Jens Peter, Kirschner, Martin, Engel-Riedel, Walburga, Jürgens, Jessica, Stoelben, Erich, Brockmann, Michael, Grau, Stefan, Sebastian, Martin, Stratmann, Jan A., Kern, Jens, Hummel, Horst-Dieter, Hegedüs, Balazs, Schuler, Martin, Plönes, Till, Aigner, Clemens, Elter, Thomas, Toepelt, Karin, Ko, Yon-Dschun, Kurz, Sylke, Grohé, Christian, Serke, Monika, Höpker, Katja, Hagmeyer, Lars, Doerr, Fabian, Hekmath, Khosro, Strapatsas, Judith, Kambartel, Karl-Otto, Chakupurakal, Geothy, Busch, Annette, Bauernfeind, Franz-Georg, Griesinger, Frank, Luers, Anne, Dirks, Wiebke, Wiewrodt, Rainer, Luecke, Andrea, Rodermann, Ernst, Diel, Andreas, Hagen, Volker, Severin, Kai, Ullrich, Roland T., Reinhardt, Hans Christian, Quaas, Alexander, Bogus, Magdalena, Courts, Cornelius, Nürnberg, Peter, Becker, Kerstin, Achter, Viktor, Büttner, Reinhard, Wolf, Jürgen, Peifer, Martin, and Thomas, Roman K.

Published
2024
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8. Serum chemistry profiling and prognostication in systemic mastocytosis: a registry-based study of the ECNM and GREM

Author

Lübke, Johannes, Schmid, Alicia, Christen, Deborah, Oude Elberink, Hanneke N. G., Span, Lambert F. R., Niedoszytko, Marek, Gorska, Aleksandra, Lange, Magdalena, Gleixner, Karoline V., Hadzijusufovic, Emir, Stefan, Alex, Angelova-Fischer, Irena, Zanotti, Roberta, Bonifacio, Massimiliano, Bonadonna, Patrizia, Shoumariyeh, Khalid, von Bubnoff, Nikolas, Müller, Sabine, Perkins, Cecelia, Elena, Chiara, Malcovati, Luca, Hagglund, Hans, Mattsson, Mattias, Parente, Roberta, Varkonyi, Judit, Fortina, Anna Belloni, Caroppo, Francesca, Brockow, Knut, Zink, Alexander, Breynaert, Christine, Leven, Toon, Yavuz, Akif Selim, Doubek, Michael, Sabato, Vito, Schug, Tanja, Hartmann, Karin, Triggiani, Massimo, Gotlib, Jason, Hermine, Olivier, Arock, Michel, Kluin-Nelemans, Hanneke C., Panse, Jens, Sperr, Wolfgang R., Valent, Peter, Reiter, Andreas, and Schwaab, Juliana

Published
2024
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11. Mast cell leukemia: clinical and molecular features and survival outcomes of patients in the ECNM Registry

Author

Kennedy, Vanessa E., Perkins, Cecelia, Reiter, Andreas, Jawhar, Mohamad, Lübke, Johannes, Kluin-Nelemans, Hanneke C., Shomali, William, Langford, Cheryl, Abuel, Justin, Hermine, Olivier, Niedoszytko, Marek, Gorska, Aleksandra, Mital, Andrzej, Bonadonna, Patrizia, Zanotti, Roberta, Tanasi, Ilaria, Mattsson, Mattias, Hagglund, Hans, Triggiani, Massimo, Yavuz, Akif Selim, Panse, Jens, Christen, Deborah, Heizmann, Marc, Shoumariyeh, Khalid, Müller, Sabine, Elena, Chiara, Malcovati, Luca, Fiorelli, Nicolas, Wortmann, Friederike, Vucinic, Vladan, Brockow, Knut, Fokoloros, Christos, Papageorgiou, Sotirios G., Breynaert, Christine, Bullens, Dominique, Doubek, Michael, Ilerhaus, Anja, Angelova-Fischer, Irena, Solomianyi, Oleksii, Várkonyi, Judit, Sabato, Vito, Rüfer, Axel, Schug, Tanja Daniela, Hermans, Maud A. W., Fortina, Anna Belloni, Caroppo, Francesca, Bumbea, Horia, Gulen, Theo, Hartmann, Karin, Elberink, Hanneke Oude, Schwaab, Juliana, Arock, Michel, Valent, Peter, Sperr, Wolfgang R., and Gotlib, Jason

Published
2023
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15. Prognostic Impact of Organomegaly in Mastocytosis: An Analysis of the European Competence Network on Mastocytosis

Author

Lübke, Johannes, Schwaab, Juliana, Christen, Deborah, Elberink, Hanneke Oude, Span, Bart, Niedoszytko, Marek, Gorska, Aleksandra, Lange, Magdalena, Gleixner, Karoline V., Hadzijusufovic, Emir, Solomianyi, Oleksii, Angelova-Fischer, Irena, Zanotti, Roberta, Bonifacio, Massimiliano, Bonadonna, Patrizia, Shoumariyeh, Khalid, von Bubnoff, Nikolas, Müller, Sabine, Perkins, Cecelia, Elena, Chiara, Malcovati, Luca, Hagglund, Hans, Mattsson, Mattias, Parente, Roberta, Varkonyi, Judit, Fortina, Anna Belloni, Caroppo, Francesca, Zink, Alexander, Brockow, Knut, Breynaert, Christine, Bullens, Dominique, Yavuz, Akif Selim, Doubek, Michael, Sabato, Vito, Schug, Tanja, Niederwieser, Dietger, Hartmann, Karin, Triggiani, Massimo, Gotlib, Jason, Hermine, Olivier, Arock, Michel, Kluin-Nelemans, Hanneke C., Panse, Jens, Sperr, Wolfgang R., Valent, Peter, Reiter, Andreas, and Jawhar, Mohamad

Published
2023
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20. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial

Author

de Latour, Régis Peffault, Szer, Jeff, Weitz, Ilene C, Röth, Alexander, Höchsmann, Britta, Panse, Jens, Usuki, Kensuke, Griffin, Morag, Kiladjian, Jean-Jacques, de Castro, Carlos M, Nishimori, Hisakazu, Ajayi, Temitayo, Al-Adhami, Mohammed, Deschatelets, Pascal, Francois, Cedric, Grossi, Federico, Risitano, Antonio M, and Hillmen, Peter

Published
2022
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21. Standards of Pathology in the Diagnosis of Systemic Mastocytosis: Recommendations of the EU-US Cooperative Group

Author

Sotlar, Karl, George, Tracy I., Kluin, Philip, Reiter, Andreas, Schwaab, Juliana, Panse, Jens, Brockow, Knut, Hartmann, Karin, Sperr, Wolfgang R., Kristensen, Thomas, Nedoszytko, Boguslaw, Carter, Melody, Bonadonna, Patrizia, Lyons, Jonathan J., Kluin-Nelemans, Hanneke C., Hermine, Olivier, Akin, Cem, Broesby-Olsen, Sigurd, Hoermann, Gregor, Triggiani, Massimo, Butterfield, Joseph H., Jawhar, Mohamad, Gotlib, Jason, Metcalfe, Dean D., Orfao, Alberto, Arock, Michel, Valent, Peter, and Horny, Hans-Peter

Published
2022
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22. Refined diagnostic criteria for bone marrow mastocytosis: a proposal of the European competence network on mastocytosis

Author

Zanotti, Roberta, Bonifacio, Massimiliano, Lucchini, Giuseppe, Sperr, Wolfgang R., Scaffidi, Luigi, van Anrooij, Björn, Oude Elberink, Hanneke NC, Rossignol, Julien, Hermine, Olivier, Gorska, Aleksandra, Lange, Magdalena, Hadzijusufovic, Emir, Miething, Cornelius, Müller, Sabine, Perkins, Cecelia, Shomali, William, Elena, Chiara, Illerhaus, Anja, Jawhar, Mohamad, Parente, Roberta, Caroppo, Francesca, Solomianyi, Oleksii, Zink, Alexander, Mattsson, Mattias, Yavuz, Akif Selim, Panse, Jens, Varkonyi, Judit, Doubek, Michael, Sabato, Vito, Breynaert, Christine, Vucinic, Vladan, Schug, Tanja, Hägglund, Hans, Wortmann, Friederike, Brockow, Knut, Angelova-Fischer, Irena, Belloni Fortina, Anna, Triggiani, Massimo, Reiter, Andreas, Hartmann, Karin, Malcovati, Luca, Gotlib, Jason, Shoumariyeh, Khalid, Niedoszytko, Marek, Arock, Michel, Kluin-Nelemans, Hanneke C., Bonadonna, Patrizia, and Valent, Peter

Published
2022
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23. Moss-produced human complement factor H with modified glycans has an extended half-life and improved biological activity

Author

Tschongov, Todor, primary, Konwar, Swagata, additional, Busch, Andreas, additional, Sievert, Christian, additional, Hartmann, Andrea, additional, Noris, Marina, additional, Gastoldi, Sara, additional, Aiello, Sistiana, additional, Schaaf, Andreas, additional, Panse, Jens, additional, Zipfel, Peter F., additional, Dabrowska-Schlepp, Paulina, additional, and Häffner, Karsten, additional

Published
2024
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24. Prevalence of hypersensitivity reactions in various forms of mastocytosis: A pilot study of 2485 adult patients with mastocytosis collected in the ECNM registry

Author

Niedoszytko, Marek, primary, Gorska, Aleksandra, additional, Brockow, Knut, additional, Bonadonna, Patrizia, additional, Lange, Magdalena, additional, Kluin‐Nelemans, Hanneke, additional, Oude‐Elberink, Hanneke, additional, Sabato, Vito, additional, Shoumariyeh, Khalid, additional, von Bubnoff, Dagmar, additional, Müller, Sabine, additional, Illerhaus, Anja, additional, Doubek, Michael, additional, Angelova‐Fischer, Irena, additional, Hermine, Olivier, additional, Arock, Michel, additional, Elena, Chiara, additional, Malcovati, Luca, additional, Yavuz, Akif Selim, additional, Schug, Tanja Daniela, additional, Fortina, Anna Belloni, additional, Judit, Várkonyi, additional, Gotlib, Jason, additional, Panse, Jens, additional, Vucinic, Vladan, additional, Reiter, Andreas, additional, Schwaab, Juliana, additional, Triggiani, Massimo, additional, Mattsson, Mattias, additional, Breynaert, Christine, additional, Romantowski, Jan, additional, Zanotti, Roberta, additional, Olivieri, Elisa, additional, Zink, Alexander, additional, van de Ven, Annick, additional, Stefan, Alex, additional, Barete, Stephane, additional, Caroppo, Francesca, additional, Perkins, Cecelia, additional, Kennedy, Vanessa, additional, Christen, Deborah, additional, Jawhar, Mohamad, additional, Luebke, Johannes, additional, Parente, Roberta, additional, Levedahl, Kerstin, additional, Hadzijusufovic, Emir, additional, Hartmann, Karin, additional, Nedoszytko, Boguslaw, additional, Sperr, Wolfgang R., additional, and Valent, Peter, additional

Published
2024
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26. Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial

Author

Gotlib, Jason, Reiter, Andreas, Radia, Deepti H., Deininger, Michael W., George, Tracy I., Panse, Jens, and Vannucchi, Alessandro M.

Subjects
Event history analysis -- Methods, Mast cell disease -- Diagnosis -- Care and treatment -- Risk factors, Biological sciences, Health
Abstract

Advanced systemic mastocytosis (AdvSM) is a rare, KIT D816V-driven hematologic neoplasm characterized by mast cell infiltration and shortened survival. We report the results of a prespecified interim analysis of an ongoing pivotal single-arm phase 2 trial (no. NCT03580655) of avapritinib, a potent, selective KIT D816V inhibitor administered primarily at a once-daily starting dose of 200 mg in patients with AdvSM (n = 62). The primary endpoint was overall response rate (ORR). Secondary endpoints included mean baseline change in AdvSM-Symptom Assessment Form Total Symptom Score and quality of life, time to response, duration of response, progression-free survival, overall survival, changes in measures of disease burden and safety. The primary endpoint was successfully met (P = 1.6 × 10.sup.-9), with an ORR of 75% (95% confidence interval 57-89) in 32 response-evaluable patients with AdvSM who had sufficient follow-up for response assessment, including 19% with complete remission with full or partial hematologic recovery. Reductions of [greater than or equal to]50% from baseline in serum tryptase (93%), bone marrow mast cells (88%) and KIT D816V variant allele fraction (60%) were observed. The most frequent grade [greater than or equal to]3 adverse events were neutropenia (24%), thrombocytopenia (16%) and anemia (16%). Avapritinib demonstrated a high rate of clinical, morphological and molecular responses and was generally well tolerated in patients with AdvSM. In a prespecified interim analysis of a pivotal phase 2 trial, avapritinib, a selective KIT inhibitor, elicited robust clinical and molecular responses, was generally well tolerated and led to improved patient-reported outcomes in patients with advanced systemic mastocytosis., Author(s): Jason Gotlib [sup.1] , Andreas Reiter [sup.2] , Deepti H. Radia [sup.3] , Michael W. Deininger [sup.4] , Tracy I. George [sup.5] , Jens Panse [sup.6] , Alessandro M. [...]

Published
2021
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27. Clinical Impact of Skin Lesions in Mastocytosis: A Multicenter Study of the European Competence Network on Mastocytosis

Author

Aberer, Elisabeth, Sperr, Wolfgang R., Bretterklieber, Agnes, Avian, Alexander, Hadzijusufovic, Emir, Kluin-Nelemans, Hanneke C., Oude Elberink, Hanneke, van Anrooij, Björn, Niedoszytko, Marek, Lange, Magdalena, Górska, Aleksandra, Elena, Chiara, Brazzelli, Valeria, Belloni Fortina, Anna, Caroppo, Francesca, Hartmann, Karin, Illerhaus, Anja, Reiter, Andreas, Jawhar, Mohamad, Bonadonna, Patrizia, Zanotti, Roberta, Triggiani, Massimo, Parente, Roberta, Gotlib, Jason, Doubek, Michael, von Bubnoff, Nikolas, Fuchs, David, Sabato, Vito, Brockow, Knut, Jäkel, Nadja, Panse, Jens, and Valent, Peter

Published
2021
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28. Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin

Author

Fuchs, David, Kilbertus, Alex, Kofler, Karin, von Bubnoff, Nikolas, Shoumariyeh, Khalid, Zanotti, Roberta, Bonadonna, Patrizia, Scaffidi, Luigi, Doubek, Michael, Elberink, Hanneke Oude, Span, Lambert F.R., Hermine, Olivier, Elena, Chiara, Benvenuti, Pietro, Yavuz, Akif Selim, Brockow, Knut, Zink, Alexander, Aberer, Elisabeth, Gorska, Aleksandra, Romantowski, Jan, Hadzijusufovic, Emir, Fortina, Anna Belloni, Caroppo, Francesca, Perkins, Cecelia, Illerhaus, Anja, Panse, Jens, Vucinic, Vladan, Jawhar, Mohamad, Sabato, Vito, Triggiani, Massimo, Parente, Roberta, Bergström, Anna, Breynaert, Christine, Gotlib, Jason, Reiter, Andreas, Hartmann, Karin, Niedoszytko, Marek, Arock, Michel, Kluin-Nelemans, Hanneke C., Sperr, Wolfgang R., Greul, Rosemarie, and Valent, Peter

Published
2021
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29. Genetic Heterogeneity of MET-Aberrant NSCLC and Its Impact on the Outcome of Immunotherapy

Author

Kron, Anna, Scheffler, Matthias, Heydt, Carina, Ruge, Lea, Schaepers, Carsten, Eisert, Anna-Kristina, Merkelbach-Bruse, Sabine, Riedel, Richard, Nogova, Lucia, Fischer, Rieke Nila, Michels, Sebastian, Abdulla, Diana S.Y., Koleczko, Sophia, Fassunke, Jana, Schultheis, Anne M., Kron, Florian, Ueckeroth, Frank, Wessling, Gabriele, Sueptitz, Juliane, Beckers, Frank, Braess, Jan, Panse, Jens, Grohé, Christian, Hamm, Michael, Kabitz, Hans-Joachim, Kambartel, Kato, Kaminsky, Britta, Krueger, Stefan, Schulte, Clemens, Lorenz, Joachim, Lorenzen, Johann, Meister, Wolfram, Meyer, Andreas, Kappes, Jutta, Reinmuth, Niels, Schaaf, Bernhard, Schulte, Wolfgang, Serke, Monika, Buettner, Reinhard, and Wolf, Jürgen

Published
2021
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30. Paroxysmal Nocturnal Hemoglobinuria, Pathophysiology, Diagnostics, and Treatment.

Author

Panse, Jens Peter, Höchsmann, Britta, and Schubert, Jörg

Subjects
*HEMOLYTIC anemia diagnosis, *THERAPEUTIC use of monoclonal antibodies, *ANEMIA, *FLOW cytometry, *ABDOMINAL pain, *FATIGUE (Physiology), *HEMOGLOBINS, *TREATMENT effectiveness, *DECISION making in clinical medicine, *GENE expression, *MONOCLONAL antibodies, *HEMOLYTIC anemia, *QUALITY of life, *DYSPNEA, *MEMBRANE proteins, *BIOMARKERS
Abstract

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis (IVH) due to diminished or absent inhibition of the complement system because of deficient expression of cell-anchored complement regulating surface proteins. IVH leads to heterogeneous symptoms such as anemia, abdominal pain, dyspnea, fatigue and increased rates of thrombophilia. Inhibitors of the terminal Complement cascade can reverse IVH leading to a significant reduction of disease burden such as thrombembolic events and also mortality. Summary: Therapeutic inhibitors of the terminal complement cascade such as eculizumab or ravulizumab significantly improve overall survival through IVH-inhibition. However, not all patients experience complete disease control with normalization of hemoglobin levels and absolute reticulocyte counts (ARC) under terminal complement inhibition as a significant part of patients develop extravascular hemolysis (EVH). EVH can be clinically relevant causing persistent anemia and fatigue. New proximal complement inhibitors (CI) mainly targeting complement component C3 or factors of the amplification pathway such as pegcetacoplan, danicopan, and iptacopan became available and are meanwhile approved for marketing. Additional complement-inhibiting strategies are under clinical development. A switch from terminal to proximal CI in patients with significant EVH can achieve hemoglobin and ARC normalization and significant improvement in quality of life (QoL). Additional approvals of proximal CI agents for the treatment of hemolytic PNH in the first line are available for pegcetacoplan and iptacopan. So far, no evidence-based algorithm is available for decision-making in first-line treatment of which type of drug should be used for individual patients. Key Messages: Terminal CIs in hemolytic PNH patients can block IVH and have led to a dramatically improved survival. Proximal CIs ameliorate anemia and improve QoL in patients with relevant EVH. However, more (real-world) data are needed to demonstrate long-term improvement in all patients with hemolytic PNH, especially those under first-line treatment with proximal CI. [ABSTRACT FROM AUTHOR]

Published
2024
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31. Phase 3 randomized COMMODORE 1 trial: Crovalimab versus eculizumab in complement inhibitor‐experienced patients with paroxysmal nocturnal hemoglobinuria.

Author

Scheinberg, Phillip, Clé, Diego Villa, Kim, Jin Seok, Nur, Erfan, Yenerel, Mustafa N., Barcellini, Wilma, Bonito, Debora, Giai, Valentina, Hus, Marek, Lee, YooJin, Lekue, Cristina Barrenetxea, Panse, Jens, Ueda, Yasutaka, Buatois, Simon, Gentile, Brittany, Kiialainen, Anna, Patel, Himika, Sreckovic, Sasha, Uguen, Marianne, and Edwards, John

Published
2024
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32. Moving toward Individual Treatment Goals with Pegcetacoplan in Patients with PNH and Impaired Bone Marrow Function.

Author

Szer, Jeff, Panse, Jens, Kulasekararaj, Austin, Oliver, Monika, Fattizzo, Bruno, Nishimura, Jun-ichi, Horneff, Regina, Szamosi, Johan, and Peffault de Latour, Régis

Subjects
*COMPLEMENT (Immunology), *COMPLEMENT inhibition, *HEMOGLOBINS, *CHRONIC diseases, *QUALITY of life
Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, potentially life-threatening haematological disease characterised by chronic complement-mediated haemolysis with multiple clinical consequences that impair quality of life. This post hoc analysis assessed haematological and clinical responses to the first targeted complement C3 inhibitor pegcetacoplan in patients with PNH and impaired bone marrow function in the PEGASUS (NCT03500549) and PRINCE (NCT04085601) studies. For patients with impaired bone marrow function, defined herein as haemoglobin <10 g/dL and absolute neutrophil count <1.5 × 109 cells/L, normalisation of the parameters may be difficult. Indeed, 20% and 43% had normalised haemoglobin in PEGASUS and PRINCE, respectively; 60% and 57% had normalised LDH, and 40% and 29% had normalised fatigue scores. A new set of parameters was applied using changes associated with clinically meaningful improvements, namely an increase in haemoglobin to ≥2 g/dL above baseline, decrease in LDH to ≤1.5× the upper limit of normal, and an increase in fatigue scores to ≥5 points above baseline. With these new parameters, 40% and 71% of PEGASUS and PRINCE patients had improved haemoglobin; 60% and 71% had an improvement in LDH, and 60% and 43% had an improvement in fatigue scores. Thus, even patients with impaired bone marrow function may achieve clinically meaningful improvements with pegcetacoplan. [ABSTRACT FROM AUTHOR]

Published
2024
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33. Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors

Author

Panse, Jens, Daguindau, Nicolas, Okuyama, Sonia, Peffault de Latour, Régis, Schafhausen, Philippe, Straetmans, Nicole, Al-Adhami, Mohammed, Persson, Emmelie, and Wong, Raymond Siu Ming

Subjects
*PAROXYSMAL hemoglobinuria, *COMPLEMENT inhibition, *FATIGUE (Physiology), *HEMOGLOBINS, *ECULIZUMAB, *FETAL hemoglobin, *ATRIAL flutter
Abstract

Background: Although complement component 5 inhibitors (C5is) eculizumab and ravulizumab improve paroxysmal nocturnal hemoglobinuria (PNH) outcomes, patients may experience persistent anemia. This post hoc analysis investigated whether the complement component 3-targeted therapy pegcetacoplan also improved hematologic outcomes and reduced fatigue in patients with PNH and mild/moderate anemia. Methods: Patients with PNH and hemoglobin ≥10.0 g/dL at baseline of PADDOCK (N = 6), PRINCE (N = 8), and PEGASUS (N = 11) were included. Before receiving pegcetacoplan, PADDOCK and PRINCE patients were C5i-naive; PEGASUS patients had hemoglobin <10.5 g/dL despite stably dosed eculizumab. Hemoglobin concentrations, percentages of patients with concentrations ≥12 g/dL, and sex-specific normalization were assessed at baseline and after 16 weeks of pegcetacoplan, as were absolute reticulocyte counts (ARCs) and normalization and fatigue scores and normalization. Results: From baseline to week 16, mean (SD) hemoglobin concentrations increased in C5i-naive patients (PADDOCK: 10.5 [0.4] to 12.7 [1.1] g/dL; PRINCE: 11.3 [1.0] to 14.0 [1.3] g/dL) and those with suboptimal eculizumab responses (PEGASUS: 10.2 [0.2] to 12.8 [2.6] g/dL). Percentage of patients with hemoglobin ≥12 g/dL increased (PADDOCK: 0 to 60.0% [3 of 5 patients]; PRINCE: 25.0% [2 of 8] to 87.5% [7 of 8]; PEGASUS: 0 to 72.7% [8 of 11]). Sex-specific hemoglobin normalization at week 16 occurred in 40.0% (2 of 5) (PADDOCK), 62.5% (5 of 8) (PRINCE), and 63.6% (7 of 11) (PEGASUS). In all studies, mean ARCs decreased from above normal to normal and ARC normalization increased. Mean Functional Assessment of Chronic Illness Therapy–Fatigue scores improved from below to above or near normal. Two patients had serious adverse events (PEGASUS: post-surgery sepsis, breakthrough hemolysis); breakthrough hemolysis resolved without study discontinuation. Conclusion: Patients with PNH and mild/moderate anemia who were C5i-naive or who had suboptimal hemoglobin concentrations despite eculizumab treatment had improved hematologic outcomes and reduced fatigue after initiating or switching to pegcetacoplan. Trial registration: Trial registration numbers: PADDOCK (NCT02588833), PRINCE (NCT04085601; EudraCT, 2018-004220-11), PEGASUS (NCT03500549). [ABSTRACT FROM AUTHOR]

Published
2024
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35. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria

Author

Peffault de Latour, Régis, primary, Röth, Alexander, additional, Kulasekararaj, Austin G., additional, Han, Bing, additional, Scheinberg, Phillip, additional, Maciejewski, Jaroslaw P., additional, Ueda, Yasutaka, additional, de Castro, Carlos M., additional, Di Bona, Eros, additional, Fu, Rong, additional, Zhang, Li, additional, Griffin, Morag, additional, Langemeijer, Saskia M.C., additional, Panse, Jens, additional, Schrezenmeier, Hubert, additional, Barcellini, Wilma, additional, Mauad, Vitor A.Q., additional, Schafhausen, Philippe, additional, Tavitian, Suzanne, additional, Beggiato, Eloise, additional, Chew, Lee Ping, additional, Gaya, Anna, additional, Huang, Wei-Han, additional, Jang, Jun Ho, additional, Kitawaki, Toshio, additional, Kutlar, Abdullah, additional, Notaro, Rosario, additional, Pullarkat, Vinod, additional, Schubert, Jörg, additional, Terriou, Louis, additional, Uchiyama, Michihiro, additional, Wong Lee Lee, Lily, additional, Yap, Eng-Soo, additional, Sicre de Fontbrune, Flore, additional, Marano, Luana, additional, Alashkar, Ferras, additional, Gandhi, Shreyans, additional, Trikha, Roochi, additional, Yang, Chen, additional, Liu, Hui, additional, Kelly, Richard J., additional, Höchsmann, Britta, additional, Kerloeguen, Cécile, additional, Banerjee, Partha, additional, Levitch, Rafael, additional, Kumar, Rakesh, additional, Wang, Zhixin, additional, Thorburn, Christine, additional, Maitra, Samopriyo, additional, Li, Shujie, additional, Verles, Aurelie, additional, Dahlke, Marion, additional, and Risitano, Antonio M., additional

Published
2024
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36. Poor Applicability of Currently Available Prognostic Scoring Systems for Prediction of Outcome in KIT D816V-Negative Advanced Systemic Mastocytosis

Author

Naumann, Nicole, primary, Rudelius, Martina, additional, Lübke, Johannes, additional, Christen, Deborah, additional, Bresser, Jakob, additional, Sotlar, Karl, additional, Metzgeroth, Georgia, additional, Fabarius, Alice, additional, Hofmann, Wolf-Karsten, additional, Panse, Jens, additional, Horny, Hans-Peter, additional, Cross, Nicholas C. P., additional, Reiter, Andreas, additional, and Schwaab, Juliana, additional

Published
2024
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37. Relevance of different prognostic scores in primary CNS lymphoma in the era of intensified treatment regimens: A retrospective, multicenter analysis of 174 patients

Author

Zeremski, Vanja, primary, Adolph, Louisa, additional, Beer, Sina, additional, Berisha, Mirjeta, additional, Jacobs, Benedikt, additional, Kahl, Christoph, additional, Koenecke, Christian, additional, Kropf, Siegfried, additional, Panse, Jens, additional, Petersen, Judith, additional, Schmidt‐Hieber, Martin, additional, Schneider, Jessica, additional, Vucinic, Vladan, additional, Walter, Jeanette, additional, Weigert, Oliver, additional, Witte, Hanno M., additional, and Mougiakakos, Dimitrios, additional

Published
2024
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38. Hemophagocytic lymphohistiocytosis in adults: collaborative analysis of 137 cases of a nationwide German registry

Author

Birndt, Sebastian, Schenk, Thomas, Heinevetter, Babett, Brunkhorst, Frank M., Maschmeyer, Georg, Rothmann, Frank, Weber, Thomas, Müller, Markus, Panse, Jens, Penack, Olaf, Schroers, Roland, Braess, Jan, Frickhofen, Norbert, Ehl, Stephan, Janka, Gritta, Lehmberg, Kai, Pletz, Mathias W., Hochhaus, Andreas, Ernst, Thomas, and La Rosée, Paul

Published
2020
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39. Prognostic impact of eosinophils in mastocytosis: analysis of 2350 patients collected in the ECNM Registry

Author

Kluin-Nelemans, Hanneke C., Reiter, Andreas, Illerhaus, Anja, van Anrooij, Bjorn, Hartmann, Karin, Span, Lambertus F. R., Gorska, Aleksandra, Niedoszytko, Marek, Lange, Magdalena, Scaffidi, Luigi, Zanotti, Roberta, Bonadonna, Patrizia, Perkins, Cecelia, Elena, Chiara, Malcovati, Luca, Shoumariyeh, Khalid, von Bubnoff, Nikolas, Parente, Roberta, Triggiani, Massimo, Schwaab, Juliana, Jawhar, Mohamad, Caroppo, Francesca, Fortina, Anna Belloni, Brockow, Knut, Zink, Alexander, Fuchs, David, Kilbertus, Alex, Yavuz, Akif Selim, Doubek, Michael, Mattsson, Mattias, Hagglund, Hans, Panse, Jens, Sabato, Vito, Aberer, Elisabeth, Niederwieser, Dietger, Breynaert, Christine, Várkonyi, Judit, Kennedy, Vanessa, Lortholary, Olivier, Jakob, Thilo, Hermine, Olivier, Rossignol, Julien, Arock, Michel, Gotlib, Jason, Valent, Peter, and Sperr, Wolfgang R.

Published
2020
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40. Deregulated expression of the HSP40 family members Auxilin-1 and -2 is indicative of proteostasis imbalance and predicts patient outcome in Ph(+) leukemia.

Author

Patterson, John, Panse, Jens, Wilop, Stefan, Samali, Afshin, Chevet, Eric, Kharabi Masouleh, Behzad, Vieri, Margherita, and Geng, Huimin

Subjects
Acute lymphoblastic leukemia, Auxilin-1, Auxilin-2, Chronic myeloid leukemia, DNAJ, HSP40, Unfolded protein response
Abstract

BACKGROUND: Proteostasis is defined by the orchestrated control of anabolic and catabolic protein pathways. Disruption of proteostasis results in cell stress and adaptation to proteostasis imbalance is mediated by adaptive pathways such as the Heat Shock Response (including heat-shock proteins) or the unfolded protein response (UPR). The BCR-ABL1 kinase (Philadelphia chromosome) is the hallmark of chronic myeloid leukemia (CML) and defines a historically poor subset in acute lymphoblastic leukemia (Ph(+) ALL). We previously demonstrated the importance of the UPR and particularly of the IRE1/XBP1 signaling axis in Ph(+) ALL, while others demonstrated the therapeutic relevance of HSP70 in ALL. In this regard, HSP70 is regulated by smaller HSP40 s, whose function is so far poorly characterized. RESULTS: Herein, we characterize the expression of HSP40 s in Ph(+) ALL and CML. We show that these genes are not regulated in a pan-class manner and identify a homologous gene pair, namely Auxilin-1 (DNAJC6) and Auxilin-2 (GAK) with a unique expression profile. Overexpression of Auxilin-2, the ubiquitously expressed homologue of Auxilin-1 correlated with superior clinical outcome in ALL and was tightly linked to both IRE1 RNase and BCR-ABL1 kinase activities. CONCLUSIONS: Our findings suggest that HSP40 gens are uniquely regulated and provide a rationale for further studies between BCR-ABL1/IRE1-based therapies in combination with HSP40 inhibitors, thus opening potentially novel therapeutic avenues.

Published
2015

41. Comprehensive support for families with parental cancer (Family-SCOUT), evaluation of a complex intervention: study protocol for a non-randomized controlled trial

Author

Dohmen, Marc, Petermann-Meyer, Andrea, Blei, Daniel, Bremen, Rebecca, Brock-Midding, Evamarie, Brüne, Manuela, Geiser, Franziska, Haastert, Burkhard, Halbach, Sarah Maria, Heuser, Christian, Holsteg, Steffen, Heier, Lina, Icks, Andrea, Karger, Andre, Montalbo, Joseph, Nakata, Hannah, Panse, Jens, Rottmann, Till-Philip, Sättler, Kristina, Viehmann, Anja, Vomhof, Markus, Ernstmann, Nicole, and Brümmendorf, Tim H.

Published
2021
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42. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

Author

Sperr, Wolfgang R, Kundi, Michael, Alvarez-Twose, Ivan, van Anrooij, Bjorn, Oude Elberink, Joanna N G, Gorska, Aleksandra, Niedoszytko, Marek, Gleixner, Karoline V, Hadzijusufovic, Emir, Zanotti, Roberta, Bonadonna, Patrizia, Bonifacio, Massimiliano, Perkins, Cecelia, Illerhaus, Anja, Elena, Chiara, Merante, Serena, Shoumariyeh, Khalid, von Bubnoff, Nikolas, Parente, Roberta, Jawhar, Mohamad, Belloni Fortina, Anna, Caroppo, Francesca, Brockow, Knut, Zink, Alexander, Fuchs, David, Kilbertus, Alex J, Yavuz, Akif Selim, Doubek, Michael, Hägglund, Hans, Panse, Jens, Sabato, Vito, Bretterklieber, Agnes, Niederwieser, Dietger, Breynaert, Christine, Hartmann, Karin, Triggiani, Massimo, Nedoszytko, Boguslaw, Reiter, Andreas, Orfao, Alberto, Hermine, Olivier, Gotlib, Jason, Arock, Michel, Kluin-Nelemans, Hanneke C, and Valent, Peter

Published
2019
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43. Anschriften der Herausgeber und Autoren

Author

Brunkhorst, Reinhard, primary, Schölmerich, Jürgen, additional, Allescher, Hans-Dieter, additional, Berger, Thomas, additional, Berlit, Peter, additional, Bertram, Franziska, additional, Bollheimer, Cornelius, additional, Bruckmayer, Herbert, additional, Brunotte, Peter, additional, Büttner, Roland, additional, Czudaj, Klaus-Peter, additional, Dellweg, Dominic, additional, Dollinger, Matthias, additional, Dorlars, Dorothee, additional, Ebert, Matthias, additional, Eder, Matthias, additional, Endlicher, Esther, additional, Fahlbusch, Markus, additional, Fickert, Peter, additional, Fluhr, Gabriele, additional, Freundt, Tobias, additional, Gallwitz, Baptist, additional, Geelvink, Mirja, additional, Gerbes, Alexander L., additional, Gerth, Jens, additional, Gleissner, Beate, additional, Glück, Thomas, additional, Gölder, Stefan K., additional, Gottschalk, Laura, additional, Gross, Oliver, additional, Gulba, Dietrich, additional, Haag-Weber, Marianne, additional, Hach-Wunderle, Viola, additional, Hamm, Michael, additional, Herrmann, Thomas R.W., additional, Hermann, Walter, additional, Herth, Felix J.F., additional, Hickstein, Mirja, additional, Hörnschemeyer-Decker, Silke, additional, Holstege, Axel, additional, Kastrup, Oliver, additional, Khattab, Ahmed A., additional, Keller, Jutta, additional, Klemm, Philipp, additional, Klein, Gunnar, additional, Köppen, Stefan, additional, Kreuter, Michael, additional, Kuczyk, Markus A., additional, Lammert, Frank, additional, Landmesser, Ulf, additional, Laurentius, Thea, additional, Lerch, Markus, additional, Lock, Guntram, additional, Löhr, J.-Matthias, additional, Lorenzen, Hans Peter, additional, Mayer, Gert, additional, Mayer, Stephanie, additional, Mayerle, Julia, additional, Mayr, Martina, additional, Merseburger, Axel S., additional, Müller, Gerhard A., additional, Müller-Ladner, Ulf, additional, Müssig, Karsten, additional, Naumann, Ralph, additional, Nebel, Michael, additional, Niedermeyer, Jost, additional, Obermeier, Florian, additional, Otto, Peter, additional, Panse, Jens, additional, Parhofer, Klaus G., additional, Petri, Susanne, additional, Pfeifer, Michael, additional, Prasse, Antje, additional, Raap, Ulrike, additional, Reinisch, Walter, additional, Richardt, Gert, additional, Richter-Bastian, Katrin, additional, Rockmann, Felix, additional, Salzberger, Bernd, additional, Sauerbruch, Tilman, additional, Schafhausen, Philippe, additional, Schmidt, Carsten, additional, Schönhofer, Bernd, additional, Schorr, Friedrich, additional, Schrader, Christoph, additional, Schumann, Michael, additional, Schwartz, Andreas, additional, Seufert, Jochen, additional, Siegmund, Britta, additional, Simon, Peter, additional, Staib, Peter, additional, Stallmach, Andreas, additional, Stark, Erwin, additional, Steinhoff, Bernhard, additional, Strunk, Johannes, additional, Tarner, Ingo H., additional, Teschendorf, Christian, additional, Thomas, Theodoros, additional, Tilg, Herbert, additional, Tölg, Ralph, additional, Trauner, Michael, additional, Unterkofler, Jenny, additional, Wagener, Peter, additional, Wallbach, Manuel, additional, Weiss, Thomas, additional, Weizsäcker, Fritz von, additional, Welker, Martin, additional, Welkoborsky, Hans-Jürgen, additional, Welte, Tobias, additional, Wiechens, Burkhard, additional, Wiegand, Uwe, additional, Wiest, Reiner, additional, Wilke, Jürgen, additional, Woenckhaus, Ulrike, additional, Wolf, Gunter, additional, and Wrede, Christian, additional

Published
2021
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44. The Data Registry of the European Competence Network on Mastocytosis (ECNM): Set Up, Projects, and Perspectives

Author

Valent, Peter, Oude Elberink, Joanna N.G., Gorska, Aleksandra, Lange, Magdalena, Zanotti, Roberta, van Anrooij, Björn, Bonifacio, Massimiliano, Bonadonna, Patrizia, Gleixner, Karoline V., Hadzijusufovic, Emir, Perkins, Cecelia, Hartmann, Karin, Illerhaus, Anja, Merante, Serena, Elena, Chiara, Shoumariyeh, Khalid, von Bubnoff, Nikolas, Parente, Roberta, Triggiani, Massimo, Schwaab, Juliana, Jawhar, Mohamad, Caroppo, Francesca, Fortina, Anna Belloni, Brockow, Knut, David Fuchs, Greul, Rosemarie, Yavuz, Akif Selim, Doubek, Michael, Mattsson, Mattias, Hagglund, Hans, Panse, Jens, Sabato, Vito, Aberer, Elisabeth, Al-Ali, Haifa Kathrin, Morren, Marie-Anne, Varkonyi, Judit, Zink, Alexander, Niedoszytko, Marek, Niederwieser, Dietger, Malcovati, Luca, Reiter, Andreas, Kennedy, Vanessa, Gotlib, Jason, Lortholary, Olivier, Hermine, Olivier, Arock, Michel, Kluin-Nelemans, Hanneke, and Sperr, Wolfgang R.

Published
2019
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45. Improving the Quality of Unstructured Cancer Data Using Large Language Models: A German Oncological Case Study.

Author

Yongli MOU, LEHMKUHL, Jonathan, SAUERBRUNN, Nicolas, KÖCHEL, Anja, PANSE, Jens, TRUH, Daniel, SOWE, Sulayman, BRÜMMENDORF, Tim, and DECKER, Stefan

Abstract

With cancer being a leading cause of death globally, epidemiological and clinical cancer registration is paramount for enhancing oncological care and facilitating scientific research. However, the heterogeneous landscape of medical data presents significant challenges to the current manual process of tumor documentation. This paper explores the potential of Large Language Models (LLMs) for transforming unstructured medical reports into the structured format mandated by the German Basic Oncology Dataset. Our findings indicate that integrating LLMs into existing hospital data management systems or cancer registries can significantly enhance the quality and completeness of cancer data collection - a vital component for diagnosing and treating cancer and improving the effectiveness and benefits of therapies. This work contributes to the broader discussion on the potential of artificial intelligence or LLMs to revolutionize medical data processing and reporting in general and cancer care in particular. [ABSTRACT FROM AUTHOR]

Published
2024
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46. Early combination therapy of COVID-19 in high-risk patients

Author

Orth, Hans Martin, primary, Flasshove, Charlotte, additional, Berger, Moritz, additional, Hattenhauer, Sandra Tessa, additional, Biederbick, Kaja D., additional, Mispelbaum, Rebekka, additional, Klein, Uwe, additional, Stemler, Jannik, additional, Fisahn, Matthis, additional, Doleschall, Anna D., additional, Baermann, Ben-Niklas, additional, Koenigshausen, Eva, additional, Tselikmann, Olga, additional, Killer, Alexander, additional, de Angelis, Clara, additional, Gliga, Smaranda, additional, Stegbauer, Johannes, additional, Spuck, Nikolai, additional, Silling, Gerda, additional, Rockstroh, Jürgen K., additional, Strassburg, Christian P., additional, Brossart, Peter, additional, Panse, Jens P., additional, Jensen, Björn-Erik Ole, additional, Luedde, Tom, additional, Boesecke, Christoph, additional, Heine, Annkristin, additional, Cornely, Oliver A., additional, and Monin, Malte B., additional

Published
2023
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49. Chlorhexidine gluconate‐coated gel pad dressings for prevention of central venous catheter‐related bloodstream infections in patients with hematologic diseases or autologous stem cell transplantation: A registry‐based matched‐pair analysis

Author

Teschner, Daniel, primary, Berisha, Mirjeta, additional, Panse, Jens, additional, Schmitt, Timo, additional, Fiegle, Eva, additional, Naendrup, Jan‐Hendrik, additional, Neitz, Julia, additional, Schmidt‐Hieber, Martin, additional, Hentrich, Marcus, additional, Böll, Boris, additional, and Schalk, Enrico, additional

Published
2023
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50. MPN-295 Avapritinib in Patients With Advanced Systemic Mastocytosis (AdvSM): Efficacy and Safety Analyses from the Phase 2 PATHFINDER Study With 2-Year Follow-Up

Author

Gotlib, Jason, primary, Reiter, Andreas, additional, Radia, Deepti H., additional, Deininger, Michael W., additional, George, Tracy I., additional, Panse, Jens, additional, Vannucchi, Alessandro M., additional, Platzbecker, Uwe, additional, Alvarez-Twose, Iván, additional, Mital, Andrzej, additional, Hermine, Olivier, additional, Dybedal, Ingunn, additional, Hexner, Elizabeth O., additional, Span, Lambert, additional, Mesa, Ruben, additional, Bose, Prithviraj, additional, Pettit, Kristen M, additional, Oh, Stephen T., additional, Lin, Hui-Min, additional, Dimitrijević, Saša, additional, Muñoz-González, Javier I., additional, and DeAngelo, Daniel J., additional

Published
2023
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