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1. Correction: Need for ICU and outcome of critically ill patients with COVID-19 and haematological malignancies: results from the EPICOVIDEHA survey

Author

Lahmer, Tobias, Salmanton-García, Jon, Marchesi, Francesco, El-Ashwah, Shaimaa, Nucci, Marcio, Besson, Caroline, Itri, Federico, Jaksic, Ozren, Čolović, Natasha, Weinbergerová, Barbora, Seval, Guldane Cengiz, Adžić-Vukičević, Tatjana, Szotkowski, Tomáš, Sili, Uluhan, Dargenio, Michelina, van Praet, Jens, van Doesum, Jaap, Schönlein, Martin, Ráčil, Zdeněk, Žák, Pavel, Poulsen, Christian Bjørn, Magliano, Gabriele, Jiménez, Moraima, Bonuomo, Valentina, Piukovics, Klára, Dragonetti, Giulia, Demirkan, Fatih, Blennow, Ola, Valković, Toni, Gomes Da Silva, Maria, Maertens, Johan, Glenthøj, Andreas, Fernández, Noemí, Bergantim, Rui, Verga, Luisa, Petzer, Verena, Omrani, Ali S., Méndez, Gustavo-Adolfo, Machado, Marina, Ledoux, Marie-Pierre, Bailén, Rebeca, Duarte, Rafael F., Del Principe, Maria Ilaria, Farina, Francesca, Martín-Pérez, Sonia, Dávila-Valls, Julio, Marchetti, Monia, Bilgin, Yavuz M., Fracchiolla, Nicola S., Cattaneo, Chiara, Espigado, Ildefonso, Cordoba, Raul, Collins, Graham P., Labrador, Jorge, Falces-Romero, Iker, Prezioso, Lucia, Meers, Stef, Passamonti, Francesco, Buquicchio, Caterina, López-García, Alberto, Kulasekararaj, Austin, Ormazabal-Vélez, Irati, Cuccaro, Annarosa, Garcia-Vidal, Carolina, Busca, Alessandro, Navrátil, Milan, de Jonge, Nick, Biernat, Monika M., Guidetti, Anna, Abu-Zeinah, Ghaith, Samarkos, Michail, Anastasopoulou, Amalia, de Ramón, Cristina, González-López, Tomás José, Hoenigl, Martin, Finizio, Olimpia, Pinczés, László Imre, Ali, Natasha, Vena, Antonio, Tascini, Carlo, Stojanoski, Zlate, Merelli, Maria, Emarah, Ziad, Kohn, Milena, Barać, Aleksandra, Mladenović, Miloš, Mišković, Bojana, Ilhan, Osman, Çolak, Gökçe Melis, Čerňan, Martin, Gräfe, Stefanie K., Ammatuna, Emanuele, Hanakova, Michaela, Víšek, Benjamín, Cabirta, Alba, Nordlander, Anna, Nunes Rodrigues, Raquel, Hersby, Ditte Stampe, Zambrotta, Giovanni Paolo Maria, Wolf, Dominik, Núñez-Martín-Buitrago, Lucía, Arellano, Elena, Aiello, Tommaso Francesco, García-Sanz, Ramón, Prattes, Juergen, Egger, Matthias, Limongelli, Alessandro, Bavastro, Martina, Cvetanoski, Milche, Dibos, Miriam, Rasch, Sebastian, Rahimli, Laman, Cornely, Oliver A., and Pagano, Livio

Published
2024
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2. Need for ICU and outcome of critically ill patients with COVID-19 and haematological malignancies: results from the EPICOVIDEHA survey

Author

Lahmer, Tobias, Salmanton-García, Jon, Marchesi, Francesco, El-Ashwah, Shaimaa, Nucci, Marcio, Besson, Caroline, Itri, Federico, Jaksic, Ozren, Čolović, Natasha, Weinbergerová, Barbora, Seval, Guldane Cengiz, Adžić-Vukičević, Tatjana, Szotkowski, Tomáš, Sili, Uluhan, Dargenio, Michelina, van Praet, Jens, van Doesum, Jaap, Schönlein, Martin, Ráčil, Zdeněk, Žák, Pavel, Poulsen, Christian Bjørn, Magliano, Gabriele, Jiménez, Moraima, Bonuomo, Valentina, Piukovics, Klára, Dragonetti, Giulia, Demirkan, Fatih, Blennow, Ola, Valković, Toni, Gomes Da Silva, Maria, Maertens, Johan, Glenthøj, Andreas, Fernández, Noemí, Bergantim, Rui, Verga, Luisa, Petzer, Verena, Omrani, Ali S., Méndez, Gustavo-Adolfo, Machado, Marina, Ledoux, Marie-Pierre, Bailén, Rebeca, Duarte, Rafael F., Del Principe, Maria Ilaria, Farina, Francesca, Martín-Pérez, Sonia, Dávila-Valls, Julio, Marchetti, Monia, Bilgin, Yavuz M., Fracchiolla, Nicola S., Cattaneo, Chiara, Espigado, Ildefonso, Cordoba, Raul, Collins, Graham P., Labrador, Jorge, Falces-Romero, Iker, Prezioso, Lucia, Meers, Stef, Passamonti, Francesco, Buquicchio, Caterina, López-García, Alberto, Kulasekararaj, Austin, Ormazabal-Vélez, Irati, Cuccaro, Annarosa, Garcia-Vidal, Carolina, Busca, Alessandro, Navrátil, Milan, de Jonge, Nick, Biernat, Monika M., Guidetti, Anna, Abu-Zeinah, Ghaith, Samarkos, Michail, Anastasopoulou, Amalia, de Ramón, Cristina, González-López, Tomás José, Hoenigl, Martin, Finizio, Olimpia, Pinczés, László Imre, Ali, Natasha, Vena, Antonio, Tascini, Carlo, Stojanoski, Zlate, Merelli, Maria, Emarah, Ziad, Kohn, Milena, Barać, Aleksandra, Mladenović, Miloš, Mišković, Bojana, Ilhan, Osman, Çolak, Gökçe Melis, Čerňan, Martin, Gräfe, Stefanie K., Ammatuna, Emanuele, Hanakova, Michaela, Víšek, Benjamín, Cabirta, Alba, Nordlander, Anna, Nunes Rodrigues, Raquel, Hersby, Ditte Stampe, Zambrotta, Giovanni Paolo Maria, Wolf, Dominik, Núñez-Martín-Buitrago, Lucía, Arellano, Elena, Aiello, Tommaso Francesco, García-Sanz, Ramón, Prattes, Juergen, Egger, Matthias, Limongelli, Alessandro, Bavastro, Martina, Cvetanoski, Milche, Dibos, Miriam, Rasch, Sebastian, Rahimli, Laman, Cornely, Oliver A., and Pagano, Livio

Published
2024
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4. Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria

Author

Schrezenmeier, Hubert, Kulasekararaj, Austin, Mitchell, Lindsay, de Latour, Régis Peffault, Devos, Timothy, Okamoto, Shinichiro, Wells, Richard, Popoff, Evan, Cheung, Antoinette, Wang, Alice, Tomazos, Ioannis, Patel, Yogesh, and Lee, Jong Wook

Published
2024
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6. Mapping aplastic anaemia hospital activity in England

Author

Bamidele Famokunwa, Aman Gupta, Stephen Thomas, Morag Griffin, and Austin Kulasekararaj

Subjects
aplastic anaemia, bone marrow failure, epidemiology, paediatric aplastic anaemia, stem cell transplantation, Diseases of the blood and blood-forming organs, RC633-647.5
Published
2024
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7. Expert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria

Author

David Dingli, Carlos De Castro III, Jamie Koprivnikar, Austin Kulasekararaj, Jaroslaw Maciejewski, Brian Mulherin, Jens Panse, Vinod Pullarkat, Alexander Röth, Jamile Shammo, Louis Terriou, Ilene Weitz, Irina Yermilov, Sarah Gibbs, Michael Broder, David Beenhouwer, and David Kuter

Subjects
(Need 8): Consensus, Aplastic anemia, Bone marrow failure, Coagulation disorders, Complement inhibitors, Hemolysis, Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

ABSTRACTIntroduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, non-malignant hematologic disease characterized by complement-mediated hemolysis (with or without hemoglobinuria), fatigue, increased susceptibility to thrombosis, and bone marrow dysfunction. The development of complement inhibitors has transformed outcomes for patients with PNH, but patients may still experience pharmacodynamic breakthrough hemolysis (BTH), which can be caused by exposure to a complement amplifying condition (CAC), such as vaccination, infection, or surgery.Materials and methods: A 13-member expert panel used a validated methodology (a RAND/UCLA modified Delphi panel) to develop consensus on how to classify pharmacodynamic BTH in patients with complement-inhibitor treated PNH. Physicians reviewed literature, rated the appropriateness of over 400 scenarios, and discussed the ratings at an in-person meeting.Results: After the meeting, the panel agreed on 77% of scenarios. Here, we present the group’s agreed-upon recommendations on how to manage BTH caused by a CAC, as well as provide a severity classification system for BTH and strategies to mitigate risk of BTH in special circumstances (e.g. vaccination, planned or unplanned surgery, and pregnancy).Discussion: In general, as severity of BTH increased, experts agreed more interventions to manage the BTH were appropriate. These recommendations are based on clinical experience and opinion. Without clear data from randomized trials to guide the management of BTH, expert opinion can be useful to support patient care.

Published
2024
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9. Immune‐monitoring of myelodysplastic neoplasms: Recommendations from the i4MDS consortium

Author

Cristina A. Tentori, Lin P. Zhao, Benedetta Tinterri, Kathryn E. Strange, Katharina Zoldan, Konstantinos Dimopoulos, Xingmin Feng, Elena Riva, Benjamin Lim, Yannick Simoni, Vidhya Murthy, Madeline J. Hayes, Antonella Poloni, Eric Padron, Bruno A. Cardoso, Michael Cross, Susann Winter, Aida Santaolalla, Bhavisha A. Patel, Emma M. Groarke, Daniel H. Wiseman, Katy Jones, Lauren Jamieson, Charles Manogaran, Naval Daver, Laura Gallur, Wendy Ingram, P. Brent Ferrell, Katja Sockel, Nicolas Dulphy, Nicolas Chapuis, Anne S. Kubasch, Astrid M. Olsnes, Austin Kulasekararaj, Hugues De Lavellade, Wolfgang Kern, Mieke Van Hemelrijck, Dominique Bonnet, Theresia M. Westers, Sylvie Freeman, Uta Oelschlaegel, David Valcarcel, Marco G. Raddi, Kirsten Grønbæk, Michaela Fontenay, Sanam Loghavi, Valeria Santini, Antonio M. Almeida, Jonathan M. Irish, David A. Sallman, Neal S. Young, Arjan A. van deLoosdrecht, Lionel Adès, Matteo G. Della Porta, Catherine Cargo, Uwe Platzbecker, Shahram Kordasti, and i4MDS consortium

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Abstract Advancements in comprehending myelodysplastic neoplasms (MDS) have unfolded significantly in recent years, elucidating a myriad of cellular and molecular underpinnings integral to disease progression. While molecular inclusions into prognostic models have substantively advanced risk stratification, recent revelations have emphasized the pivotal role of immune dysregulation within the bone marrow milieu during MDS evolution. Nonetheless, immunotherapy for MDS has not experienced breakthroughs seen in other malignancies, partly attributable to the absence of an immune classification that could stratify patients toward optimally targeted immunotherapeutic approaches. A pivotal obstacle to establishing “immune classes” among MDS patients is the absence of validated accepted immune panels suitable for routine application in clinical laboratories. In response, we formed International Integrative Innovative Immunology for MDS (i4MDS), a consortium of multidisciplinary experts, and created the following recommendations for standardized methodologies to monitor immune responses in MDS. A central goal of i4MDS is the development of an immune score that could be incorporated into current clinical risk stratification models. This position paper first consolidates current knowledge on MDS immunology. Subsequently, in collaboration with clinical and laboratory specialists, we introduce flow cytometry panels and cytokine assays, meticulously devised for clinical laboratories, aiming to monitor the immune status of MDS patients, evaluating both immune fitness and identifying potential immune “risk factors.” By amalgamating this immunological characterization data and molecular data, we aim to enhance patient stratification, identify predictive markers for treatment responsiveness, and accelerate the development of systems immunology tools and innovative immunotherapies.

Published
2024
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10. Decoding the historical tale: COVID-19 impact on haematological malignancy patients—EPICOVIDEHA insights from 2020 to 2022Research in context

Author

Jon Salmanton-García, Francesco Marchesi, Francesca Farina, Barbora Weinbergerová, Federico Itri, Julio Dávila-Valls, Sonia Martín-Pérez, Andreas Glenthøj, Ditte Stampe Hersby, Maria Gomes da Silva, Raquel Nunes Rodrigues, Alberto López-García, Raúl Córdoba, Yavuz M. Bilgin, Iker Falces-Romero, Shaimaa El-Ashwah, Ziad Emarah, Caroline Besson, Milena Kohn, Jaap Van Doesum, Emanuele Ammatuna, Monia Marchetti, Jorge Labrador, Giovanni Paolo Maria Zambrotta, Luisa Verga, Ozren Jaksic, Marcio Nucci, Klára Piukovics, Alba Cabirta-Touzón, Moraima Jiménez, Elena Arellano, Ildefonso Espigado, Ola Blennow, Anna Nordlander, Stef Meers, Jens van Praet, Tommaso Francesco Aiello, Carolina Garcia-Vidal, Nicola Fracchiolla, Mariarita Sciumè, Guldane Cengiz Seval, Pavel Žák, Caterina Buquicchio, Carlo Tascini, Stefanie K. Gräfe, Martin Schönlein, Tatjana Adžić-Vukičević, Valentina Bonuomo, Chiara Cattaneo, Summiya Nizamuddin, Martin Čerňan, Gaëtan Plantefeve, Romane Prin, Tomas Szotkovski, Graham P. Collins, Michelina Dargenio, Verena Petzer, Dominik Wolf, Natasha Čolović, Lucia Prezioso, Toni Valković, Francesco Passamonti, Gustavo-Adolfo Méndez, Uluhan Sili, Antonio Vena, Martina Bavastro, Alessandro Limongelli, Rafael F. Duarte, Marie-Pierre Ledoux, Milche Cvetanoski, Zlate Stojanoski, Marina Machado, Josip Batinić, Gabriele Magliano, Monika M. Biernat, Nikola Pantić, Christian Bjørn Poulsen, Annarosa Cuccaro, Maria Ilaria Del Principe, Austin Kulasekararaj, Irati Ormazabal-Vélez, Alessandro Busca, Fatih Demirkan, Marriyam Ijaz, Nikolai Klimko, Igor Stoma, Sofya Khostelidi, Noemí Fernández, Ali S. Omrani, Rui Bergantim, Nick De Jonge, Guillemette Fouquet, Milan Navrátil, Ghaith Abu-Zeinah, Michail Samarkos, Johan Maertens, Cristina De Ramón, Anna Guidetti, Ferenc Magyari, Tomás José González-López, Tobias Lahmer, Olimpia Finizio, Natasha Ali, László Imre Pinczés, Esperanza Lavilla-Rubira, Alessandra Romano, Maria Merelli, Mario Delia, Maria Calbacho, Joseph Meletiadis, Darko Antić, José-Ángel Hernández-Rivas, Joyce Marques de Almeida, Murtadha Al-Khabori, Martin Hoenigl, Maria Chiara Tisi, Nina Khanna, Aleksandra Barać, Noha Eisa, Roberta Di Blasi, Raphaël Liévin, Carolina Miranda-Castillo, Nathan C. Bahr, Sylvain Lamure, Mario Virgilio Papa, Ayel Yahya, Avinash Aujayeb, Jan Novák, Nurettin Erben, María Fernández-Galán, José-María Ribera-Santa Susana, Ikhwan Rinaldi, Rita Fazzi, Monica Piedimonte, Rémy Duléry, Yung Gonzaga, Andrés Soto-Silva, Giuseppe Sapienza, Alexandra Serris, Ľuboš Drgoňa, Ana Groh, Laura Serrano, Eleni Gavriilaki, Athanasios Tragiannidis, Juergen Prattes, Nicola Coppola, Vladimir Otašević, Miloš Mladenović, Mirjana Mitrović, Bojana Mišković, Pavel Jindra, Sofia Zompi, Maria Vittoria Sacchi, Carolin Krekeler, Maria Stefania Infante, Daniel García-Bordallo, Gökçe Melis Çolak, Jiří Mayer, Marietta Nygaard, Michaela Hanáková, Zdeněk Ráčil, Matteo Bonanni, Philipp Koehler, Laman Rahimli, Oliver A. Cornely, Livio Pagano, Francisco Javier Martín-Vallejo, Przemyslaw Zdziarski, Hossein Zarrinfer, Jana Wittig, Sein Win, Vivien Wai-Man, Benjamín Víšek, Donald C. Vinh, Maria Vehreschild, Gina Varricchio, Panagiotis Tsirigotis, Ana Torres-Tienza, Alina Daniela Tanase, Agostino Tafuri, Maria Stamouli, Jiří Sramek, Carole Soussain, Ayten Shirinova, Jörg Schubert, Enrico Schalk, Mohammad Reza Salehi, Modar Saleh, Giorgio Rosati, Elisa Roldán, Florian Reizine, Mayara Rêgo, Isabel Regalado-Artamendi, Marina Popova, Fernando Pinto, Laure Philippe, Hans Martin Orth, Hans-Beier Ommen, Aleš Obr, Lucía Núñez-Martín-Buitrago, Nicolas Noël, Julia Neuhann, Gianpaolo Nadali, Julia A. Nacov, Ana M. Munhoz Alburquerque, Maria Enza Mitra, Malgorzata Mikulska, Sibylle Mellinghoff, Ben Mechtel, Juan-Alberto Martín-González, Sandra Malak, Jorge Loureiro-Amigo, Lisset Lorenzo De La Peña, Giulia Liberti, Marianne Landau, Ira Lacej, Martin Kolditz, Chi Shan Kho, Reham Abdelaziz Khedr, Meinolf Karthaus, Linda Katharina Karlsson, María-Josefa Jiménez-Lorenzo, Macarena Izuzquiza, Baerbel Hoell-Neugebauer, Raoul Herbrecht, Christopher H. Heath, Fabio Guolo, Jan Grothe, Antonio Giordano, Sergey Gerasymchuk, Ramón García-Sanz, Nicole García-Poutón, Vaneuza Araújo Moreira Funke, Monica Fung, Charlotte Flasshove, Luana Fianchi, Jenna Essame, Matthias Egger, Bernard Drenou, Giulia Dragonetti, Maximilian Desole, Roberta Della Pepa, Bénédicte Deau Fischer, Elizabeth De Kort, Erik De Cabo, François Danion, Etienne Daguindau, Tania Cushion, Louise Cremer, Marianna Criscuolo, Gregorio Cordini, Antonella Cingolani, Fabio Ciceri, Fazle Rabbi Chowdhury, Ekaterina Chelysheva, Adrien Chauchet, Louis Yi Ann Chai, M. Mansour Ceesay, Elena Busch, Mathias Brehon, Davimar M.M. Borducchi, Stephen Booth, Serge Bologna, Caroline Berg Venemyr, Rebeca Bailén-Almorox, Anastasia Antoniadou, Amalia N. Anastasopoulou, and Fevzi Altuntaş

Subjects
Vaccination, ICU, COVID-19, Haematological malignancy, Immunosuppression, Medicine (General), R5-920
Abstract

Summary: Background: The COVID-19 pandemic heightened risks for individuals with hematological malignancies due to compromised immune systems, leading to more severe outcomes and increased mortality. While interventions like vaccines, targeted antivirals, and monoclonal antibodies have been effective for the general population, their benefits for these patients may not be as pronounced. Methods: The EPICOVIDEHA registry (National Clinical Trials Identifier, NCT04733729) gathers COVID-19 data from hematological malignancy patients since the pandemic's start worldwide. It spans various global locations, allowing comprehensive analysis over the first three years (2020–2022). Findings: The EPICOVIDEHA registry collected data from January 2020 to December 2022, involving 8767 COVID-19 cases in hematological malignancy patients from 152 centers across 41 countries, with 42% being female. Over this period, there was a significant reduction in critical infections and an overall decrease in mortality from 29% to 4%. However, hospitalization, particularly in the ICU, remained associated with higher mortality rates. Factors contributing to increased mortality included age, multiple comorbidities, active malignancy at COVID-19 onset, pulmonary symptoms, and hospitalization. On the positive side, vaccination with one to two doses or three or more doses, as well as encountering COVID-19 in 2022, were associated with improved survival. Interpretation: Patients with hematological malignancies still face elevated risks, despite reductions in critical infections and overall mortality rates over time. Hospitalization, especially in ICUs, remains a significant concern. The study underscores the importance of vaccination and the timing of COVID-19 exposure in 2022 for enhanced survival in this patient group. Ongoing monitoring and targeted interventions are essential to support this vulnerable population, emphasizing the critical role of timely diagnosis and prompt treatment in preventing severe COVID-19 cases. Funding: Not applicable.

Published
2024
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11. Age, successive waves, immunization, and mortality in elderly COVID-19 hematological patients: EPICOVIDEHA findings

Author

Giuseppe Rossi, Jon Salmanton-García, Chiara Cattaneo, Francesco Marchesi, Julio Dávila-Valls, Sonia Martín-Pérez, Federico Itri, Alberto López-García, Andreas Glenthøj, Maria Gomes da Silva, Caroline Besson, Monia Marchetti, Barbora Weinbergerová, Ozren Jaksic, Moraima Jiménez, Yavuz M. Bilgin, Jaap Van Doesum, Francesca Farina, Pavel Žák, Luisa Verga, Graham P. Collins, Valentina Bonuomo, Jens Van Praet, Marcio Nucci, Stef Meers, Ildefonso Espigado, Nicola S. Fracchiolla, Toni Valković, Christian Bjørn Poulsen, Natasha Čolović, Giulia Dragonetti, Marie-Pierre Ledoux, Carlo Tascini, Caterina Buquicchio, Ola Blennow, Francesco Passamonti, Marina Machado, Jorge Labrador, Rafael F. Duarte, Martin Schönlein, Lucia Prezioso, Iker Falces-Romero, Austin Kulasekararaj, Carolina Garcia-Vidal, Noemí Fernández, Ghaith Abu-Zeinah, Irati Ormazabal-Vélez, Tatjana Adžić-Vukičević, Klára Piukovics, Igor Stoma, Annarosa Cuccaro, Gabriele Magliano, Tomáš Szotkowski, Tomás-José González-López, Shaimaa El-Ashwah, Rui Bergantim, Uluhan Sili, Johan Maertens, Fatih Demirkan, Cristina De Ramón, Verena Petzer, Maria Ilaria Del Principe, Milan Navrátil, Michelina Dargenio, Guldane Cengiz Seval, Michail Samarkos, Zdeněk Ráčil, László Imre Pinczés, Tobias Lahmer, Alessandro Busca, Gustavo-Adolfo Méndez, Antonio Vena, Monika M. Biernat, Maria Merelli, Maria Calbacho, Aleksandra Barać, Martina Bavastro, Alessandro Limongelli, Osman Ilhan, Dominik Wolf, Gökçe Melis Çolak, Ramón García-Sanz, Ziad Emarah, Bojana Mišković, Stefanie K. Gräfe, Miloš Mladenović, Tommaso Francesco Aiello, Lucía Núñez-Martín-Buitrago, Anna Nordlander, Elena Arellano, Giovanni Paolo Maria Zambrotta, Emanuele Ammatuna, Alba Cabirta, Maria Vittoria Sacchi, Raquel Nunes Rodrigues, Ditte Stampe Hersby, Michaela Hanakova, Laman Rahimli, Raul Cordoba, Oliver A. Cornely, Livio Pagano, Joyce MARQUES DE ALMEIDA, José-Ángel HERNÁNDEZ-RIVAS, Anna GUIDETTI, Olimpia FINIZIO, Zlate STOJANOSKI, Milche CVETANOSKI, Joseph MELETIADIS, Nick DE JONGE, Darko ANTIĆ, Natasha ALI, Maria Chiara TISI, Laura SERRANO, Gaëtan PLANTEFEVE, Nina KHANNA, Martin HOENIGL, Martin ČERŇAN, Carolina MIRANDA-CASTILLO, María FERNÁNDEZ-GALÁN, Alexandra SERRIS, Nurettin ERBEN, Rémy DULÉRY, Avinash AUJAYEB, Mario Virgilio PAPA, Jan NOVÁK, Mario DELIA, Giuseppe SAPIENZA, Florian REIZINE, Ali S. OMRANI, Roberta DI BLASI, Sylvain LAMURE, Ľuboš DRGOŇA, Nicola COPPOLA, Josip BATINIĆ, Murtadha AL-KHABORI, José-María RIBERA-SANTA SUSANA, Monica PIEDIMONTE, Jorge LOUREIRO-AMIGO, Guillemette FOUQUET, Rita FAZZI, François DANION, Jörg SCHUBERT, Baerbel HOELL-NEUGEBAUER, Nathan C. BAHR, Ayel Omar YAHIA, Ana TORRES-ATIENZA, Ikhwan RINALDI, Marina POPOVA, Hans-Beier OMMEN, Maria Enza MITRA, Malgorzata MIKULSKA, Ira LACEJ, Sofya KHOSTELIDI, Sein WIN, Donald VINH, Modar SALEH, Juergen PRATTES, Pavel JINDRA, Fabio GUOLO, Roberta DELLA PEPA, Ekaterina CHELYSHEVA, Przemyslaw ZDZIARSKI, Vivien WAI-MAN, Andrés SOTO-SILVA, Hans Martin ORTH, Sandra MALAK, Lisset LORENZO DE LA PEÑA, Martin KOLDITZ, Chi Shan KHO, Christopher H. HEATH, Ana GROH, Eleni GAVRIILAKI, Monica FUNG, Matthias EGGER, Elizabeth DE KORT, Erik DE CABO, Tania CUSHION, Fazle Rabbi CHOWDHURY, M. Mansour CEESAY, Mathias BREHON, Gina VARRICCHIO, Agostino TAFURI, María-Josefa JIMÉNEZ-LORENZO, Nikolai KLIMKO, Panagiotis TSIRIGOTIS, Anastasia ANTONIADOU, and Maria VEHRESCHILD

Subjects
Elderly, SARS-CoV-2, Hematological malignancy, High-risk patient, COVID-19, Infectious and parasitic diseases, RC109-216
Abstract

Objectives: Elderly patients with hematologic malignancies face the highest risk of severe COVID-19 outcomes. The infection's impact on different age groups remains unstudied in detail. Methods: We analyzed elderly patients (age groups: 65-70, 71-75, 76-80, and >80 years old) with hematologic malignancies included in the EPICOVIDEHA registry between January 2020 and July 2022. Univariable and multivariable Cox regression models were conducted to identify factors influencing death in COVID-19 patients with hematological malignancy. Results: The study included data from 3,603 elderly patients (aged 65 or older) with hematological malignancy, with a majority being male (58.1%) and a significant proportion having comorbidities. The patients were divided into four age groups, and the analysis assessed COVID-19 outcomes, vaccination status, and other variables in relation to age and pandemic waves. The 90-day survival rate for patients with COVID-19 was 71.2%, with significant differences between groups. The pandemic waves had varying impacts, with the first wave affecting patients over 80 years old, the second being more severe in 65-70, and the third being the least severe in all age groups. Factors contributing to 90-day mortality included age, comorbidities, lymphopenia, active malignancy, acute leukemia, less than three vaccine doses, severe COVID-19, and using only corticosteroids as treatment. Conclusion: These data underscore the heterogeneity of elderly hematological patients, highlight the different impacts of COVID-19 waves and the pivotal importance of vaccination, and may help in planning future healthcare efforts.

Published
2023
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12. Moving toward Individual Treatment Goals with Pegcetacoplan in Patients with PNH and Impaired Bone Marrow Function

Author

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

Subjects
PNH, bone marrow dysfunction, haematological response, clinical response, pegcetacoplan, Biology (General), QH301-705.5, Chemistry, QD1-999
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 9 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.

Published
2024
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13. Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Author

Montoro, Juan, Eikema, Dirk-Jan, Tuffnell, Joe, Potter, Victoria, Kalwak, Krzysztof, Halkes, Constantijn J. M., Kulagin, Alexander, Collin, Matthew, Wynn, Robert F., Robinson, Stephen, Nicholson, Emma, Sengeloev, Henrik, Clay, Jennifer, Halahleh, Khalid, Skorobogatova, Elena, Sanz, Jaime, Passweg, Jakob, Mielke, Stephan, Ryhänen, Samppa, Carpenter, Ben, Gedde-Dahl, Tobias, Tholouli, Eleni, Fanin, Renato, Lewalle, Philippe, Kulasekararaj, Austin, Risitano, Antonio, and Peffault de Latour, Régis

Published
2024
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15. Decoding the historical tale: COVID-19 impact on haematological malignancy patients—EPICOVIDEHA insights from 2020 to 2022

Author

Martín-Vallejo, Francisco Javier, Zdziarski, Przemyslaw, Zarrinfer, Hossein, Wittig, Jana, Win, Sein, Wai-Man, Vivien, Víšek, Benjamín, Vinh, Donald C., Vehreschild, Maria, Varricchio, Gina, Tsirigotis, Panagiotis, Torres-Tienza, Ana, Tanase, Alina Daniela, Tafuri, Agostino, Stamouli, Maria, Sramek, Jiří, Soussain, Carole, Shirinova, Ayten, Schubert, Jörg, Schalk, Enrico, Salehi, Mohammad Reza, Saleh, Modar, Rosati, Giorgio, Roldán, Elisa, Reizine, Florian, Rêgo, Mayara, Regalado-Artamendi, Isabel, Popova, Marina, Pinto, Fernando, Philippe, Laure, Orth, Hans Martin, Ommen, Hans-Beier, Obr, Aleš, Núñez-Martín-Buitrago, Lucía, Noël, Nicolas, Neuhann, Julia, Nadali, Gianpaolo, Nacov, Julia A., Munhoz Alburquerque, Ana M., Mitra, Maria Enza, Mikulska, Malgorzata, Mellinghoff, Sibylle, Mechtel, Ben, Martín-González, Juan-Alberto, Malak, Sandra, Loureiro-Amigo, Jorge, Lorenzo De La Peña, Lisset, Liberti, Giulia, Landau, Marianne, Lacej, Ira, Kolditz, Martin, Kho, Chi Shan, Khedr, Reham Abdelaziz, Karthaus, Meinolf, Karlsson, Linda Katharina, Jiménez-Lorenzo, María-Josefa, Izuzquiza, Macarena, Hoell-Neugebauer, Baerbel, Herbrecht, Raoul, Heath, Christopher H., Guolo, Fabio, Grothe, Jan, Giordano, Antonio, Gerasymchuk, Sergey, García-Sanz, Ramón, García-Poutón, Nicole, Funke, Vaneuza Araújo Moreira, Fung, Monica, Flasshove, Charlotte, Fianchi, Luana, Essame, Jenna, Egger, Matthias, Drenou, Bernard, Dragonetti, Giulia, Desole, Maximilian, Della Pepa, Roberta, Deau Fischer, Bénédicte, De Kort, Elizabeth, De Cabo, Erik, Danion, François, Daguindau, Etienne, Cushion, Tania, Cremer, Louise, Criscuolo, Marianna, Cordini, Gregorio, Cingolani, Antonella, Ciceri, Fabio, Chowdhury, Fazle Rabbi, Chelysheva, Ekaterina, Chauchet, Adrien, Chai, Louis Yi Ann, Ceesay, M. Mansour, Busch, Elena, Brehon, Mathias, Borducchi, Davimar M.M., Booth, Stephen, Bologna, Serge, Berg Venemyr, Caroline, Bailén-Almorox, Rebeca, Antoniadou, Anastasia, Anastasopoulou, Amalia N., Altuntaş, Fevzi, Salmanton-García, Jon, Marchesi, Francesco, Farina, Francesca, Weinbergerová, Barbora, Itri, Federico, Dávila-Valls, Julio, Martín-Pérez, Sonia, Glenthøj, Andreas, Hersby, Ditte Stampe, Gomes Da Silva, Maria, Nunes Rodrigues, Raquel, López-García, Alberto, Córdoba, Raúl, Bilgin, Yavuz M., Falces-Romero, Iker, El-Ashwah, Shaimaa, Emarah, Ziad, Besson, Caroline, Kohn, Milena, Van Doesum, Jaap, Ammatuna, Emanuele, Marchetti, Monia, Labrador, Jorge, Zambrotta, Giovanni Paolo Maria, Verga, Luisa, Jaksic, Ozren, Nucci, Marcio, Piukovics, Klára, Cabirta-Touzón, Alba, Jiménez, Moraima, Arellano, Elena, Espigado, Ildefonso, Blennow, Ola, Nordlander, Anna, Meers, Stef, Vian Praet, Jens, Aiello, Tommaso Francesco, Garcia-Vidal, Carolina, Fracchiolla, Nicola S., Sciumè, Mariarita, Seval, Guldane Cengiz, Žák, Pavel, Buquicchio, Caterina, Tascini, Carlo, Gräfe, Stefanie K., Schönlein, Martin, Adžić-VUKIČEVIĆ, Tatjana, Bonuomo, Valentina, Cattaneo, Chiara, Nizamuddin, Summiya, Čerňan, Martin, Plantefeve, Gaëtan, Prin, Romane, Szotkovski, Tomas, Collins, Graham P., Dargenio, Michelina, Petzer, Verena, Wolf, Dominik, Čolović, Natasha, Prezioso, Lucia, Valković, Toni, Passamonti, Francesco, Méndez, Gustavo-Adolfo, Sili, Uluhan, Vena, Antonio, Bavastro, Martina, Limongelli, Alessandro, Duarte, Rafael F., Ledoux, Marie-Pierre, Cvetanoski, Milche, Stojanoski, Zlate, Machado, Marina, Batinić, Josip, Magliano, Gabriele, Biernat, Monika M., Pantić, Nikola, Poulsen, Christian Bjørn, Cuccaro, Annarosa, Del Principe, Maria Ilaria, Kulasekararaj, Austin, Ormazabal-Vélez, Irati, Busca, Alessandro, Demirkan, Fatih, Ijaz, Marriyam, Klimko, Nikolai, Stoma, Igor, Khostelidi, Sofya, Fernández, Noemí, Omrani, Ali S., Bergantim, Rui, De Jonge, Nick, Fouquet, Guillemette, Navrátil, Milan, Abu-Zeinah, Ghaith, Samarkos, Michail, Maertens, Johan, De Ramón, Cristina, Guidetti, Anna, Magyari, Ferenc, González-López, Tomás José, Lahmer, Tobias, Finizio, Olimpia, Ali, Natasha, Pinczés, László Imre, Lavilla-Rubira, Esperanza, Romano, Alessandra, Merelli, Maria, Delia, Mario, Calbacho, Maria, Meletiadis, Joseph, Antić, Darko, Hernández-Rivas, José-Ángel, Marques De Almeida, Joyce, Al-Khabori, Murtadha, Hoenigl, Martin, Tisi, Maria Chiara, Khanna, Nina, Barać, Aleksandra, Eisa, Noha, Di Blasi, Roberta, Liévin, Raphaël, Miranda-Castillo, Carolina, Bahr, Nathan C., Lamure, Sylvain, Papa, Mario Virgilio, Yahya, Ayel, Aujayeb, Avinash, Novák, Jan, Erben, Nurettin, Fernández-Galán, María, Ribera-Santa Susana, José-María, Rinaldi, Ikhwan, Fazzi, Rita, Piedimonte, Monica, Duléry, Rémy, Gonzaga, Yung, Soto-Silva, Andrés, Sapienza, Giuseppe, Serris, Alexandra, Drgoňa, Ľuboš, Groh, Ana, Serrano, Laura, Gavriilaki, Eleni, Tragiannidis, Athanasios, Prattes, Juergen, Coppola, Nicola, Otašević, Vladimir, Mladenović, Miloš, Mitrović, Mirjana, Mišković, Bojana, Jindra, Pavel, Zompi, Sofia, Sacchi, Maria Vittoria, Krekeler, Carolin, Shumilov, Evgenii, Infante, Maria Stefania, García-Bordallo, Daniel, Çolak, Gökçe Melis, Mayer, Jiří, Nygaard, Marietta, Hanáková, Michaela, Ráčil, Zdeněk, Quattrone, Martina, Bonanni, Matteo, Koehler, Philipp, Rahimli, Laman, Cornely, Oliver A., and Pagano, Livio

Published
2024
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16. Convergent somatic evolution commences in utero in a germline ribosomopathy

Author

Machado, Heather E., Øbro, Nina F., Williams, Nicholas, Tan, Shengjiang, Boukerrou, Ahmed Z., Davies, Megan, Belmonte, Miriam, Mitchell, Emily, Baxter, E. Joanna, Mende, Nicole, Clay, Anna, Ancliff, Philip, Köglmeier, Jutta, Killick, Sally B., Kulasekararaj, Austin, Meyer, Stefan, Laurenti, Elisa, Campbell, Peter J., Kent, David G., Nangalia, Jyoti, and Warren, Alan J.

Published
2023
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17. COVID-19 and CAR-T cells: current challenges and future directions-a report from the EPICOVIDEHA survey by EHA-IDWP

Author

Busca, Alessandro, Salmanton-García, Jon, Corradini, Paolo, Marchesi, Francesco, Cabirta, Alba, Di Blasi, Roberta, Dulery, Remy, Lamure, Sylvain, Farina, Francesca, Weinbergerová, Barbora, Batinić, Josip, Nordlander, Anna, López-García, Alberto, Drgoňa, Ľuboš, Espigado-Tocino, Ildefonso, Falces-Romero, Iker, García-Sanz, Ramón, García-Vidal, Carolina, Guidetti, Anna, Khanna, Nina, Kulasekararaj, Austin, Maertens, Johan, Hoenigl, Martin, Klimko, Nikolai, Koehler, Philipp, Pagliuca, Antonio, Passamonti, Francesco, Cornely, Oliver A, and Pagano, Livio

Subjects
Prevention, Infection, Good Health and Well Being, COVID-19, Humans, Immunotherapy, Adoptive, Receptors, Chimeric Antigen, T-Lymphocytes
Abstract

The EHA-IDWP developed an observational registry collecting data on COVID-19 infection in patients who received CAR T-cell therapy. Prevalence of COVID-19 was 4.8%, and overall mortality was 50%, highlighting the need for prevention of infection in these patients.

Published
2022

18. Convergent somatic evolution commences in utero in a germline ribosomopathy

Author

Heather E. Machado, Nina F. Øbro, Nicholas Williams, Shengjiang Tan, Ahmed Z. Boukerrou, Megan Davies, Miriam Belmonte, Emily Mitchell, E. Joanna Baxter, Nicole Mende, Anna Clay, Philip Ancliff, Jutta Köglmeier, Sally B. Killick, Austin Kulasekararaj, Stefan Meyer, Elisa Laurenti, Peter J. Campbell, David G. Kent, Jyoti Nangalia, and Alan J. Warren

Subjects
Science
Abstract

Abstract Clonal tracking of cells using somatic mutations permits exploration of clonal dynamics in human disease. Here, we perform whole genome sequencing of 323 haematopoietic colonies from 10 individuals with the inherited ribosomopathy Shwachman-Diamond syndrome to reconstruct haematopoietic phylogenies. In ~30% of colonies, we identify mutually exclusive mutations in TP53, EIF6, RPL5, RPL22, PRPF8, plus chromosome 7 and 15 aberrations that increase SBDS and EFL1 gene dosage, respectively. Target gene mutations commence in utero, resulting in a profusion of clonal expansions, with only a few haematopoietic stem cell lineages (mean 8, range 1-24) contributing ~50% of haematopoietic colonies across 8 individuals (range 4-100% clonality) by young adulthood. Rapid clonal expansion during disease transformation is associated with biallelic TP53 mutations and increased mutation burden. Our study highlights how convergent somatic mutation of the p53-dependent nucleolar surveillance pathway offsets the deleterious effects of germline ribosomopathy but increases opportunity for TP53-mutated cancer evolution.

Published
2023
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20. COVID-19 infection in adult patients with hematological malignancies: a European Hematology Association Survey (EPICOVIDEHA)

Author

Pagano, Livio, Salmanton-García, Jon, Marchesi, Francesco, Busca, Alessandro, Corradini, Paolo, Hoenigl, Martin, Klimko, Nikolai, Koehler, Philipp, Pagliuca, Antonio, Passamonti, Francesco, Verga, Luisa, Víšek, Benjamin, Ilhan, Osman, Nadali, Gianpaolo, Weinbergerová, Barbora, Córdoba-Mascuñano, Raúl, Marchetti, Monia, Collins, Graham P, Farina, Francesca, Cattaneo, Chiara, Cabirta, Alba, Gomes-Silva, Maria, Itri, Federico, van Doesum, Jaap, Ledoux, Marie-Pierre, Čerňan, Martin, Jakšić, Ozren, Duarte, Rafael F, Magliano, Gabriele, Omrani, Ali S, Fracchiolla, Nicola S, Kulasekararaj, Austin, Valković, Toni, Poulsen, Christian Bjørn, Machado, Marina, Glenthøj, Andreas, Stoma, Igor, Ráčil, Zdeněk, Piukovics, Klára, Navrátil, Milan, Emarah, Ziad, Sili, Uluhan, Maertens, Johan, Blennow, Ola, Bergantim, Rui, García-Vidal, Carolina, Prezioso, Lucia, Guidetti, Anna, del Principe, Maria Ilaria, Popova, Marina, de Jonge, Nick, Ormazabal-Vélez, Irati, Fernández, Noemí, Falces-Romero, Iker, Cuccaro, Annarosa, Meers, Stef, Buquicchio, Caterina, Antić, Darko, Al-Khabori, Murtadha, García-Sanz, Ramón, Biernat, Monika M, Tisi, Maria Chiara, Sal, Ertan, Rahimli, Laman, Čolović, Natasa, Schönlein, Martin, Calbacho, Maria, Tascini, Carlo, Miranda-Castillo, Carolina, Khanna, Nina, Méndez, Gustavo-Adolfo, Petzer, Verena, Novák, Jan, Besson, Caroline, Duléry, Rémy, Lamure, Sylvain, Nucci, Marcio, Zambrotta, Giovanni, Žák, Pavel, Seval, Guldane Cengiz, Bonuomo, Valentina, Mayer, Jiří, López-García, Alberto, Sacchi, Maria Vittoria, Booth, Stephen, Ciceri, Fabio, Oberti, Margherita, Salvini, Marco, Izuzquiza, Macarena, Nunes-Rodrigues, Raquel, Ammatuna, Emanuele, Obr, Aleš, Herbrecht, Raoul, Núñez-Martín-Buitrago, Lucía, Mancini, Valentina, Shwaylia, Hawraa, Sciumè, Mariarita, Essame, Jenna, Nygaard, Marietta, and Batinić, Josip

Subjects
Prevention, Cancer, Lymphoma, Rare Diseases, Hematology, Good Health and Well Being, Adult, Aged, Aged, 80 and over, COVID-19, Europe, Female, Hematologic Neoplasms, Hospitalization, Humans, Intensive Care Units, Male, Middle Aged, Registries, Risk Factors, SARS-CoV-2, Young Adult, Pandemic, Hematological malignancies, Epidemiology, EHA, EPICOVIDEHA working group, Cardiorespiratory Medicine and Haematology, Oncology and Carcinogenesis
Abstract

BackgroundPatients with hematological malignancies (HM) are at high risk of mortality from SARS-CoV-2 disease 2019 (COVID-19). A better understanding of risk factors for adverse outcomes may improve clinical management in these patients. We therefore studied baseline characteristics of HM patients developing COVID-19 and analyzed predictors of mortality.MethodsThe survey was supported by the Scientific Working Group Infection in Hematology of the European Hematology Association (EHA). Eligible for the analysis were adult patients with HM and laboratory-confirmed COVID-19 observed between March and December 2020.ResultsThe study sample includes 3801 cases, represented by lymphoproliferative (mainly non-Hodgkin lymphoma n = 1084, myeloma n = 684 and chronic lymphoid leukemia n = 474) and myeloproliferative malignancies (mainly acute myeloid leukemia n = 497 and myelodysplastic syndromes n = 279). Severe/critical COVID-19 was observed in 63.8% of patients (n = 2425). Overall, 2778 (73.1%) of the patients were hospitalized, 689 (18.1%) of whom were admitted to intensive care units (ICUs). Overall, 1185 patients (31.2%) died. The primary cause of death was COVID-19 in 688 patients (58.1%), HM in 173 patients (14.6%), and a combination of both COVID-19 and progressing HM in 155 patients (13.1%). Highest mortality was observed in acute myeloid leukemia (199/497, 40%) and myelodysplastic syndromes (118/279, 42.3%). The mortality rate significantly decreased between the first COVID-19 wave (March-May 2020) and the second wave (October-December 2020) (581/1427, 40.7% vs. 439/1773, 24.8%, p value

Published
2021

21. Diagnosis and investigation of suspected haemophagocytic lymphohistiocytosis in adults: 2023 Hyperinflammation and HLH Across Speciality Collaboration (HiHASC) consensus guideline

Author

Cox, Miriam F, Mackenzie, Strachan, Low, Ryan, Brown, Michael, Sanchez, Emilie, Carr, Aisling, Carpenter, Ben, Bishton, Mark, Duncombe, Andrew, Akpabio, Akpabio, Kulasekararaj, Austin, Sin, Fang En, Jones, Alexis, Kavirayani, Akhila, Sen, Ethan S, Quick, Vanessa, Dulay, Gurdeep S, Clark, Sam, Bauchmuller, Kris, Tattersall, Rachel S, and Manson, Jessica J

Published
2024
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22. HAPLOIDENTICAL TRANSPLANT WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE FOR ACUTE MYELOID LEUKAEMIA AND MYELODYSPLASTIC SYNDROMES PATIENTS: THE ROLE OF PREVIOUS LINES OF THERAPY.

Author

Daniele Avenoso, Fabio Serpenti, Liron Barnea Slonim, Styliani Bouziana, Francesco Dazzi, Guy Hannah, Michelle Kenyon, Varun Mehra, Austin Kulasekararaj, Pramila Krishnamurthy, Mili Naresh Shah, Sharon Lionel, Antonio Pagliuca, and Victoria Potter

Subjects
Haplo-identical stem cell transplantation, AML, MDS, Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Background: Allogeneic haematopoietic stem-cell transplant is a potentially curative option for high-risk acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) patients. Post-transplant cyclophosphamide administration allows for selection of haploidentical donors in patients who are eligible for the procedure, but do not have a fully matched donor, since it can overcome the HLA barrier. There is still an active debate on whether intensification of the conditioning regimen is necessary with haploidentical donors when peripheral blood stem cells are used as the source of the graft. Herein we report our decennial experience of haploidentical stem-cell transplant using peripheral blood stem cells at King’s College Hospital. Objectives: The primary objective was to evaluate overall survival (OS) for patients with less than two previous lines of therapy. Secondary objectives were total OS, OS according to cytomegalovirus (CMV) reactivation, incidence of transplant-related mortality (TRM), graft-versus-host disease (GVHD) and GVHD-relapse-free survival (GRFS). Results: One-year and three-year total OS were 62% and 43%, respectively, with a median OS of 22 months. One-year and three-year OS for patients with ≤2 and in patients with >2 previous lines of therapy were 72% and 55%, and 60% and 22%, respectively (p-value=0.04). The median OS in patients with >2 previous lines of therapy and ≤2 lines of therapy was 16 and 49 months, respectively. Cumulative incidence (CI) of relapse was 25% with a median time to relapse of 5 months (range 1 – 38 months). Conclusions: Haploidentical haematopoietic stem-cell transplant is potentially curative in chemo-sensitive AML and MDS and offers a high rate of prolonged remission. Our cohort further confirms the role of consolidative haploidentical transplant in patients in complete remission and highlights that patients with heavily pre-treated disease may not benefit from this strategy.

Published
2024
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25. Age, successive waves, immunization, and mortality in elderly COVID-19 hematological patients: EPICOVIDEHA findings

Author

MARQUES DE ALMEIDA, Joyce, HERNÁNDEZ-RIVAS, José-Ángel, GUIDETTI, Anna, FINIZIO, Olimpia, STOJANOSKI, Zlate, CVETANOSKI, Milche, MELETIADIS, Joseph, DE JONGE, Nick, ANTIĆ, Darko, ALI, Natasha, TISI, Maria Chiara, SERRANO, Laura, PLANTEFEVE, Gaëtan, KHANNA, Nina, HOENIGL, Martin, ČERŇAN, Martin, MIRANDA-CASTILLO, Carolina, FERNÁNDEZ-GALÁN, María, SERRIS, Alexandra, ERBEN, Nurettin, DULÉRY, Rémy, AUJAYEB, Avinash, PAPA, Mario Virgilio, NOVÁK, Jan, DELIA, Mario, SAPIENZA, Giuseppe, REIZINE, Florian, OMRANI, Ali S., DI BLASI, Roberta, LAMURE, Sylvain, DRGOŇA, Ľuboš, COPPOLA, Nicola, BATINIĆ, Josip, AL-KHABORI, Murtadha, RIBERA-SANTA SUSANA, José-María, PIEDIMONTE, Monica, LOUREIRO-AMIGO, Jorge, FOUQUET, Guillemette, FAZZI, Rita, DANION, François, SCHUBERT, Jörg, HOELL-NEUGEBAUER, Baerbel, BAHR, Nathan C., YAHIA, Ayel Omar, TORRES-ATIENZA, Ana, RINALDI, Ikhwan, POPOVA, Marina, OMMEN, Hans-Beier, MITRA, Maria Enza, MIKULSKA, Malgorzata, LACEJ, Ira, KHOSTELIDI, Sofya, WIN, Sein, VINH, Donald, SALEH, Modar, PRATTES, Juergen, JINDRA, Pavel, GUOLO, Fabio, DELLA PEPA, Roberta, CHELYSHEVA, Ekaterina, ZDZIARSKI, Przemyslaw, WAI-MAN, Vivien, SOTO-SILVA, Andrés, ORTH, Hans Martin, MALAK, Sandra, LORENZO DE LA PEÑA, Lisset, KOLDITZ, Martin, Shan KHO, Chi, HEATH, Christopher H., GROH, Ana, GAVRIILAKI, Eleni, FUNG, Monica, EGGER, Matthias, DE KORT, Elizabeth, DE CABO, Erik, CUSHION, Tania, CHOWDHURY, Fazle Rabbi, CEESAY, M. Mansour, BREHON, Mathias, VARRICCHIO, Gina, TAFURI, Agostino, JIMÉNEZ-LORENZO, María-Josefa, KLIMKO, Nikolai, TSIRIGOTIS, Panagiotis, ANTONIADOU, Anastasia, VEHRESCHILD, Maria, Rossi, Giuseppe, Salmanton-García, Jon, Cattaneo, Chiara, Marchesi, Francesco, Dávila-Valls, Julio, Martín-Pérez, Sonia, Itri, Federico, López-García, Alberto, Glenthøj, Andreas, Gomes da Silva, Maria, Besson, Caroline, Marchetti, Monia, Weinbergerová, Barbora, Jaksic, Ozren, Jiménez, Moraima, Bilgin, Yavuz M., Van Doesum, Jaap, Farina, Francesca, Žák, Pavel, Verga, Luisa, Collins, Graham P., Bonuomo, Valentina, Van Praet, Jens, Nucci, Marcio, Meers, Stef, Espigado, Ildefonso, Fracchiolla, Nicola S., Valković, Toni, Poulsen, Christian Bjørn, Čolović, Natasha, Dragonetti, Giulia, Ledoux, Marie-Pierre, Tascini, Carlo, Buquicchio, Caterina, Blennow, Ola, Passamonti, Francesco, Machado, Marina, Labrador, Jorge, Duarte, Rafael F., Schönlein, Martin, Prezioso, Lucia, Falces-Romero, Iker, Kulasekararaj, Austin, Garcia-Vidal, Carolina, Fernández, Noemí, Abu-Zeinah, Ghaith, Ormazabal-Vélez, Irati, Adžić-Vukičević, Tatjana, Piukovics, Klára, Stoma, Igor, Cuccaro, Annarosa, Magliano, Gabriele, Szotkowski, Tomáš, González-López, Tomás-José, El-Ashwah, Shaimaa, Bergantim, Rui, Sili, Uluhan, Maertens, Johan, Demirkan, Fatih, De Ramón, Cristina, Petzer, Verena, Del Principe, Maria Ilaria, Navrátil, Milan, Dargenio, Michelina, Seval, Guldane Cengiz, Samarkos, Michail, Ráčil, Zdeněk, Pinczés, László Imre, Lahmer, Tobias, Busca, Alessandro, Méndez, Gustavo-Adolfo, Vena, Antonio, Biernat, Monika M., Merelli, Maria, Calbacho, Maria, Barać, Aleksandra, Bavastro, Martina, Limongelli, Alessandro, Ilhan, Osman, Wolf, Dominik, Çolak, Gökçe Melis, García-Sanz, Ramón, Emarah, Ziad, Mišković, Bojana, Gräfe, Stefanie K., Mladenović, Miloš, Aiello, Tommaso Francesco, Núñez-Martín-Buitrago, Lucía, Nordlander, Anna, Arellano, Elena, Zambrotta, Giovanni Paolo Maria, Ammatuna, Emanuele, Cabirta, Alba, Sacchi, Maria Vittoria, Nunes Rodrigues, Raquel, Hersby, Ditte Stampe, Hanakova, Michaela, Rahimli, Laman, Cordoba, Raul, Cornely, Oliver A., and Pagano, Livio

Published
2023
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27. OUTPATIENT SUBCUTANEOUS ALEMTUZUMAB IS FEASIBLE AND SAFE FOR APLASTIC ANEMIA AND ASSOCIATED WITH HIGH RESPONSE RATES

Author

AR Fonseca, CC Justino, VC Molla, PE Yamakawa, IB Rabelo, A Hill, L Arnold, R Kelly, M Griffin, T Munir, E Velloso, T Dalessandro, AM Risitano, P Scheinberg, A Kulasekararaj, and C Arrais-Rodrigues

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

This multicenter international retrospective analysis aimed to evaluate the efficacy and safety of subcutaneous alemtuzumab (ALZ) as an alternative immunosuppressive therapy for aplastic anemia (AA). The study included 57 with AA patients who received ALZ treatment in four centers in Brazil and the United Kingdom. Most patients had severe aplastic anemia (SAA). The median age of the patients was 52 years, with 21% being older than 65 years. Out of the 60 ALZ treatments analyzed, 57% were first-line treatments, 26% were second-line, and 17% were administered after at least two lines of therapy. At 6 months, the overall response rate (ORR) was 54%. At 12 months, the ORR increased to 57%, with a complete response rate of 18% and a partial response rate of 39%. The cumulative incidence of response was significantly higher in patients younger than 65 years (68%) and in those who received a 103 mg subcutaneous ALZ dose (61%). The overall survival (OS) rates were 84% at 1 year and 71% at 4 years. OS at 4 years was significantly higher in patients who responded to ALZ treatment (hazard ratio: 9.88, 95% confidence interval 2.12 - 46.0, p = 0.004). Adverse events associated with ALZ were mostly of low grade, and infectious events were infrequent. In conclusion, subcutaneous ALZ appears to be a feasible, effective, and safe alternative to horse antithymocyte globulin (hATG) in patients with aplastic anemia who require immunosuppressive treatment. This is particularly relevant for centers and countries with limited access to hATG.

Published
2023
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28. Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria

Author

Austin G. Kulasekararaj, Anna-Elina Lehtinen, Cecily Forsyth, Shreyans Gandhi, Morag Griffin, Sixten Körper, Gabor Mikala, Petra Muus, Ulrik Overgaard, Christopher J. Patriquin, Humphrey Pullon, Yu-Min Shen, Ruth Spearing, Jeff Szer, Guillemette de la Borderie, Petra W. Duda, Ramin Farzaneh-Far, Sharan Ragunathan, Camil E. Sayegh, Douangsone D. Vadysirisack, and Hubert Schrezenmeier

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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29. S183: PHASE III RANDOMIZED, MULTICENTER, OPEN-LABEL COMMODORE 1 TRIAL: COMPARISON OF CROVALIMAB VS ECULIZUMAB IN COMPLEMENT INHIBITOR-EXPERIENCED PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

Author

Phillip Scheinberg, Diego Cle, John Edwards, Valentina Giai, Marek Hus, Jin Seok Kim, Cristina Barrenetxea Lekue, Zsolt Nagy, Erfan Nur, Jens Panse, Yasutaka Ueda, Mustafa Nuri Yenerel, Anita Appius, Nadiesh Balachandran, Sammy Chebon, Brittany Gentile, Simon Buatois, and Austin Kulasekararaj

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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30. S182: ORAL IPTACOPAN MONOTHERAPY INCREASES PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) RED BLOOD CELL CLONE SIZE VIA CONTROL OF INTRA- AND EXTRAVASCULAR HEMOLYSIS IN ANTI-C5-TREATED PNH PATIENTS WITH ANEMIA

Author

Antonio Maria Risitano, Alexander Röth, Austin Kulasekararaj, Phillip Scheinberg, Yasutaka Ueda, Carlos de Castro, Eros DI Bona, Morag Griffin, Saskia Langemeijer, Hubert Schrezenmeier, Wilma Barcellini, Vitor Aq Mauad, Jens Panse, Philippe Schafhausen, Suzanne Tavitian, Eloise Beggiato, Anna Gaya, Wei-Han Huang, Toshio Kitawaki, Abdullah Kutlar, Jaroslaw P Maciejewski, Rosario Notaro, Vinod Pullarkat, Jörg Schubert, Louis Terriou, Michihiro Uchiyama, Flore Sicre de Fontbrune, Luana Marano, Ferras Alashkar, Shreyans Gandhi, Cécile Kerloëguen, Rakesh Kumar, Christine Thorburn, Samopriyo Maitra, Marion Dahlke, and Régis Peffault de Latour

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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32. P771: PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND CLINICALLY SIGNIFICANT EXTRAVASCULAR HEMOLYSIS ON RAVULIZUMAB/ECULIZUMAB SHOWED HEMOGLOBIN RESPONSE SUPERIORITY WITH ADD-ON DANICOPAN VS PLACEBO

Author

Jong Wook Lee, Morag Griffin, Jin Seok Kim, Lily L L Wong, Caroline Piatek, Deepak Jain, Peng Liu, Gleb Filippov, Flore Sicre de Fontbrune, Antonio Maria Risitano, and Austin Kulasekararaj

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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33. P774: SUBSTANTIAL INCREASES IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) RED BLOOD CELL CLONE SIZE WITH ORAL IPTACOPAN MONOTHERAPY CONFIRMS CONTROL OF HEMOLYSIS IN COMPLEMENT INHIBITOR-NAÏVE PNH PATIENTS

Author

Régis Peffault de Latour, Bing Han, Jaroslaw P Maciejewski, Yasutaka Ueda, Rong Fu, LI Zhang, Austin Kulasekararaj, Alexander Röth, Lee Ping Chew, Jun Ho Jang, Lily L L Wong, Jens Panse, Eng-Soo Yap, Luana Marano, Flore Sicre de Fontbrune, Chen Yang, Partha Banerjee, Zhixin Wang, Christine Thorburn, Shujie LI, Marion Dahlke, and Antonio Maria Risitano

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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34. P772: LONG-TERM RAVULIZUMAB TREATMENT IN COMPLEMENT INHIBITOR-EXPERIENCED PATIENTS WITH PNH PROVIDES DURABLE CONTROL OF INTRAVASCULAR HEMOLYSIS WITH LOW INCIDENCE OF MAJOR ADVERSE VASCULAR EVENTS AND DEATH.

Author

Austin Kulasekararaj, Robert Brodsky, Morag Griffin, Alexander Röth, Caroline Piatek, Masayo Ogawa, Ji Yu, Yogesh Patel, Fernando Ataulfo Gonzalez Fernandez, Jun-Ichi Nishimura, Régis Peffault de Latour, Jeff Szer, and Jong Wook Lee

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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36. P1288: UNMANIPULATED PERIPHERAL HAPLOIDENTICAL HAEMATOPOIETIC STEM CELL TRANSPLANT IS AN EFFECTIVE CONSOLIDATIVE STRATEGY FOR HIGH-RISK ACUTE MYELOID LEUKAEMIA AND MYELODYSPLASTIC SYNDROME

Author

Fabio Serpenti, Daniele Avenoso, Kenyon Michelle, Pramila Krishnamurthy, Varun Mehra, Austin Kulasekararaj, Shreyans Gandhi, Francesco Dazzi, Shah Mili Naresh, Ye Ting Leung, Sandra Anteh, Madson Correia de Farias, Styliani Bouziana, Christianne Bourlon, Oana Diana Dragoi, Tony Pagliuca, and Victoria Potter

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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40. PB2063: APPULSE-PNH: A PHASE IIIB TRIAL TO EVALUATE THE EFFICACY AND SAFETY OF SWITCHING TO IPTACOPAN IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) ON ANTI-C5 THERAPY WITH HEMOGLOBIN >10G/DL

Author

Antonio Maria Risitano, David J Araten, David Kuter, Bhumika J Patel, Régis Peffault de Latour, Marion Dahlke, Tania Garito, Shujie LI, Samopriyo Maitra, and Austin Kulasekararaj

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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42. Impact of SARS-CoV-2 vaccination and monoclonal antibodies on outcome post–CD19-directed CAR T-cell therapy: an EPICOVIDEHA survey

Author

van Doesum, Jaap A., Salmanton-García, Jon, Marchesi, Francesco, Di Blasi, Roberta, Falces-Romero, Iker, Cabirta, Alba, Farina, Francesca, Besson, Caroline, Weinbergerová, Barbora, Van Praet, Jens, Schönlein, Martin, López-García, Alberto, Lamure, Sylvain, Guidetti, Anna, De Ramón-Sánchez, Cristina, Batinić, Josip, Gavriilaki, Eleni, Tragiannidis, Athanasios, Tisi, Maria Chiara, Plantefeve, Gaëtan, Petzer, Verena, Ormazabal-Vélez, Irati, Marques de Almeida, Joyce, Marchetti, Monia, Maertens, Johan, Machado, Marina, Kulasekararaj, Austin, Hernández-Rivas, José-Ángel, Gomes da Silva, Maria, Fernández, Noemí, Espigado, Ildefonso, Drgoňa, Ľuboš, Dragonetti, Giulia, Metafuni, Elisabetta, Calbacho, Maria, Blennow, Ola, Wolf, Dominik, van Anrooij, Bjorn, Nunes Rodrigues, Raquel, Nordlander, Anna, Martín-González, Juan-Alberto, Liévin, Raphaël, Jiménez, Moraima, Gräfe, Stefanie K., García-Sanz, Ramón, Córdoba, Raúl, Rahimli, Laman, van Meerten, Tom, Cornely, Oliver A., and Pagano, Livio

Published
2023
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43. Current use of androgens in bone marrow failure disorders: a report from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation

Author

Simona Pagliuca, Austin G. Kulasekararaj, Dirk-Jan Eikema, Brian Piepenbroek, Raheel Iftikhar, Tariq Mahmood Satti, Morag Griffin, Marica Laurino, Alphan Kupesiz, Yves Bertrand, Bruno Fattizzo, Ibrahim Yakoub-Agha, Mahmoud Aljurf, Paola Corti, Erika Massaccesi, Bruno Lioure, Marisa Calabuig, Matthias Klammer, Emel Unal, Depei Wu, Patrice Chevallier, Edouard Forcade, John A. Snowden, Hakan Ozdogu, Antonio Risitano, and Régis Peffault de Latour

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Androgens represent the historical therapeutic backbone of bone marrow failure (BMF) syndromes. However, their role has rarely been analyzed in a prospective setting, and systematic and long-term data regarding their usage, effectiveness and toxicity in both acquired and inherited BMF are currently unavailable. Here, taking advantage of a unique disease-specific international dataset, we retrospectively analyzed the largest cohort so far of BMF patients who received androgens before or in the absence of an allogeneic hematopoietic cell transplantation (HCT), re-evaluating their current use in these disorders. We identified 274 patients across 82 European Society for Blood and Marrow Transplantation (EBMT) affiliated centers: 193 with acquired (median age 32 years) and 81 with inherited (median age 8 years) BMF. With a median duration of androgen treatment of 5.6 and 20 months, respectively, complete and partial remission rates at 3 months were 6% and 29% in acquired and 8% and 29% in inherited disorders. Five-year overall survival and failure-free survival (FFS) were respectively 63% and 23% in acquired and 78% and 14% in inherited BMF. Androgen initiation after second-line treatments for acquired BMF, and after >12 months post diagnosis for inherited BMF were identified as factors associated with improved FFS in multivariable analysis. Androgen use was associated with a manageable incidence of organ-specific toxicity, and low rates of solid and hematologic malignancies. Sub-analysis of transplant-related outcomes after exposure to these compounds showed probabilities of survival and complications similar to other transplanted BMF cohorts. This study delivers a unique opportunity to track androgen use in BMF syndromes and represents the basis for general recommendations on this category of therapeutics on behalf of the Severe Aplastic Anemia Working Party of the EBMT.

Published
2023
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45. Decoding the historical tale: COVID-19 impact on haematological malignancy patients—EPICOVIDEHA insights from 2020 to 2022

Author

Salmanton-García, Jon, primary, Marchesi, Francesco, additional, Farina, Francesca, additional, Weinbergerová, Barbora, additional, Itri, Federico, additional, Dávila-Valls, Julio, additional, Martín-Pérez, Sonia, additional, Glenthøj, Andreas, additional, Hersby, Ditte Stampe, additional, Gomes Da Silva, Maria, additional, Nunes Rodrigues, Raquel, additional, López-García, Alberto, additional, Córdoba, Raúl, additional, Bilgin, Yavuz M., additional, Falces-Romero, Iker, additional, El-Ashwah, Shaimaa, additional, Emarah, Ziad, additional, Besson, Caroline, additional, Kohn, Milena, additional, Van Doesum, Jaap, additional, Ammatuna, Emanuele, additional, Marchetti, Monia, additional, Labrador, Jorge, additional, Zambrotta, Giovanni Paolo Maria, additional, Verga, Luisa, additional, Jaksic, Ozren, additional, Nucci, Marcio, additional, Piukovics, Klára, additional, Cabirta-Touzón, Alba, additional, Jiménez, Moraima, additional, Arellano, Elena, additional, Espigado, Ildefonso, additional, Blennow, Ola, additional, Nordlander, Anna, additional, Meers, Stef, additional, Vian Praet, Jens, additional, Aiello, Tommaso Francesco, additional, Garcia-Vidal, Carolina, additional, Fracchiolla, Nicola S., additional, Sciumè, Mariarita, additional, Seval, Guldane Cengiz, additional, Žák, Pavel, additional, Buquicchio, Caterina, additional, Tascini, Carlo, additional, Gräfe, Stefanie K., additional, Schönlein, Martin, additional, Adžić-VUKIČEVIĆ, Tatjana, additional, Bonuomo, Valentina, additional, Cattaneo, Chiara, additional, Nizamuddin, Summiya, additional, Čerňan, Martin, additional, Plantefeve, Gaëtan, additional, Prin, Romane, additional, Szotkovski, Tomas, additional, Collins, Graham P., additional, Dargenio, Michelina, additional, Petzer, Verena, additional, Wolf, Dominik, additional, Čolović, Natasha, additional, Prezioso, Lucia, additional, Valković, Toni, additional, Passamonti, Francesco, additional, Méndez, Gustavo-Adolfo, additional, Sili, Uluhan, additional, Vena, Antonio, additional, Bavastro, Martina, additional, Limongelli, Alessandro, additional, Duarte, Rafael F., additional, Ledoux, Marie-Pierre, additional, Cvetanoski, Milche, additional, Stojanoski, Zlate, additional, Machado, Marina, additional, Batinić, Josip, additional, Magliano, Gabriele, additional, Biernat, Monika M., additional, Pantić, Nikola, additional, Poulsen, Christian Bjørn, additional, Cuccaro, Annarosa, additional, Del Principe, Maria Ilaria, additional, Kulasekararaj, Austin, additional, Ormazabal-Vélez, Irati, additional, Busca, Alessandro, additional, Demirkan, Fatih, additional, Ijaz, Marriyam, additional, Klimko, Nikolai, additional, Stoma, Igor, additional, Khostelidi, Sofya, additional, Fernández, Noemí, additional, Omrani, Ali S., additional, Bergantim, Rui, additional, De Jonge, Nick, additional, Fouquet, Guillemette, additional, Navrátil, Milan, additional, Abu-Zeinah, Ghaith, additional, Samarkos, Michail, additional, Maertens, Johan, additional, De Ramón, Cristina, additional, Guidetti, Anna, additional, Magyari, Ferenc, additional, González-López, Tomás José, additional, Lahmer, Tobias, additional, Finizio, Olimpia, additional, Ali, Natasha, additional, Pinczés, László Imre, additional, Lavilla-Rubira, Esperanza, additional, Romano, Alessandra, additional, Merelli, Maria, additional, Delia, Mario, additional, Calbacho, Maria, additional, Meletiadis, Joseph, additional, Antić, Darko, additional, Hernández-Rivas, José-Ángel, additional, Marques De Almeida, Joyce, additional, Al-Khabori, Murtadha, additional, Hoenigl, Martin, additional, Tisi, Maria Chiara, additional, Khanna, Nina, additional, Barać, Aleksandra, additional, Eisa, Noha, additional, Di Blasi, Roberta, additional, Liévin, Raphaël, additional, Miranda-Castillo, Carolina, additional, Bahr, Nathan C., additional, Lamure, Sylvain, additional, Papa, Mario Virgilio, additional, Yahya, Ayel, additional, Aujayeb, Avinash, additional, Novák, Jan, additional, Erben, Nurettin, additional, Fernández-Galán, María, additional, Ribera-Santa Susana, José-María, additional, Rinaldi, Ikhwan, additional, Fazzi, Rita, additional, Piedimonte, Monica, additional, Duléry, Rémy, additional, Gonzaga, Yung, additional, Soto-Silva, Andrés, additional, Sapienza, Giuseppe, additional, Serris, Alexandra, additional, Drgoňa, Ľuboš, additional, Groh, Ana, additional, Serrano, Laura, additional, Gavriilaki, Eleni, additional, Tragiannidis, Athanasios, additional, Prattes, Juergen, additional, Coppola, Nicola, additional, Otašević, Vladimir, additional, Mladenović, Miloš, additional, Mitrović, Mirjana, additional, Mišković, Bojana, additional, Jindra, Pavel, additional, Zompi, Sofia, additional, Sacchi, Maria Vittoria, additional, Krekeler, Carolin, additional, Shumilov, Evgenii, additional, Infante, Maria Stefania, additional, García-Bordallo, Daniel, additional, Çolak, Gökçe Melis, additional, Cingolani, Antonella, additional, Mayer, Jiří, additional, Criscuolo, Marianna, additional, Nygaard, Marietta, additional, Hanáková, Michaela, additional, Fianchi, Luana, additional, Ráčil, Zdeněk, additional, Giordano, Antonio, additional, Quattrone, Martina, additional, Bonanni, Matteo, additional, Koehler, Philipp, additional, Rahimli, Laman, additional, Cornely, Oliver A., additional, Pagano, Livio, additional, Martín-Vallejo, Francisco Javier, additional, Zdziarski, Przemyslaw, additional, Zarrinfer, Hossein, additional, Wittig, Jana, additional, Win, Sein, additional, Wai-Man, Vivien, additional, Víšek, Benjamín, additional, Vinh, Donald C., additional, Vehreschild, Maria, additional, Varricchio, Gina, additional, Tsirigotis, Panagiotis, additional, Torres-Tienza, Ana, additional, Tanase, Alina Daniela, additional, Tafuri, Agostino, additional, Stamouli, Maria, additional, Sramek, Jiří, additional, Soussain, Carole, additional, Shirinova, Ayten, additional, Schubert, Jörg, additional, Schalk, Enrico, additional, Salehi, Mohammad Reza, additional, Saleh, Modar, additional, Rosati, Giorgio, additional, Roldán, Elisa, additional, Reizine, Florian, additional, Rêgo, Mayara, additional, Regalado-Artamendi, Isabel, additional, Popova, Marina, additional, Pinto, Fernando, additional, Philippe, Laure, additional, Orth, Hans Martin, additional, Ommen, Hans-Beier, additional, Obr, Aleš, additional, Núñez-Martín-Buitrago, Lucía, additional, Noël, Nicolas, additional, Neuhann, Julia, additional, Nadali, Gianpaolo, additional, Nacov, Julia A., additional, Munhoz Alburquerque, Ana M., additional, Mitra, Maria Enza, additional, Mikulska, Malgorzata, additional, Mellinghoff, Sibylle, additional, Mechtel, Ben, additional, Martín-González, Juan-Alberto, additional, Malak, Sandra, additional, Loureiro-Amigo, Jorge, additional, Lorenzo De La Peña, Lisset, additional, Liberti, Giulia, additional, Landau, Marianne, additional, Lacej, Ira, additional, Kolditz, Martin, additional, Kho, Chi Shan, additional, Khedr, Reham Abdelaziz, additional, Karthaus, Meinolf, additional, Karlsson, Linda Katharina, additional, Jiménez-Lorenzo, María-Josefa, additional, Izuzquiza, Macarena, additional, Hoell-Neugebauer, Baerbel, additional, Herbrecht, Raoul, additional, Heath, Christopher H., additional, Guolo, Fabio, additional, Grothe, Jan, additional, Gerasymchuk, Sergey, additional, García-Sanz, Ramón, additional, García-Poutón, Nicole, additional, Funke, Vaneuza Araújo Moreira, additional, Fung, Monica, additional, Flasshove, Charlotte, additional, Essame, Jenna, additional, Egger, Matthias, additional, Drenou, Bernard, additional, Dragonetti, Giulia, additional, Desole, Maximilian, additional, Della Pepa, Roberta, additional, Deau Fischer, Bénédicte, additional, De Kort, Elizabeth, additional, De Cabo, Erik, additional, Danion, François, additional, Daguindau, Etienne, additional, Cushion, Tania, additional, Cremer, Louise, additional, Cordini, Gregorio, additional, Ciceri, Fabio, additional, Chowdhury, Fazle Rabbi, additional, Chelysheva, Ekaterina, additional, Chauchet, Adrien, additional, Chai, Louis Yi Ann, additional, Ceesay, M. Mansour, additional, Busch, Elena, additional, Brehon, Mathias, additional, Borducchi, Davimar M.M., additional, Booth, Stephen, additional, Bologna, Serge, additional, Berg Venemyr, Caroline, additional, Bailén-Almorox, Rebeca, additional, Antoniadou, Anastasia, additional, Anastasopoulou, Amalia N., additional, and Altuntaş, Fevzi, additional

Published
2024
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46. Outcome of COVID-19 in allogeneic stem cell transplant recipients: Results from the EPICOVIDEHA registry

Author

Alessandro Busca, Jon Salmanton-García, Francesco Marchesi, Francesca Farina, Guldane Cengiz Seval, Jaap Van Doesum, Nick De Jonge, Nathan C. Bahr, Johan Maertens, Joseph Meletiadis, Nicola S. Fracchiolla, Barbora Weinbergerová, Luisa Verga, Zdeněk Ráčil, Moraima Jiménez, Andreas Glenthøj, Ola Blennow, Alina Daniela Tanase, Martin Schönlein, Lucia Prezioso, Nina Khanna, Rafael F. Duarte, Pavel Žák, Marcio Nucci, Marina Machado, Austin Kulasekararaj, Ildefonso Espigado, Elizabeth De Kort, José-María Ribera-Santa Susana, Monia Marchetti, Gabriele Magliano, Iker Falces-Romero, Osman Ilhan, Emanuele Ammatuna, Sofia Zompi, Panagiotis Tsirigotis, Anastasia Antoniadou, Giovanni Paolo Maria Zambrotta, Anna Nordlander, Linda Katharina Karlsson, Michaela Hanakova, Giulia Dragonetti, Alba Cabirta, Caroline Berg Venemyr, Stefanie Gräfe, Jens Van Praet, Athanasios Tragiannidis, Verena Petzer, Alberto López-García, Federico Itri, Ana Groh, Eleni Gavriilaki, Michelina Dargenio, Laman Rahimli, Oliver A. Cornely, Livio Pagano, EPICOVIDEHA Consortium, Juergen Prattes, Malgorzata Mikulska, Gustavo-Adolfo Méndez, Tobias Lahmer, Pavel Jindra, Anna Guidetti, Rita Fazzi, Maria Ilaria Del Principe, Cristina De Ramón, Maria Calbacho, Zlate Stojanoski, Andrés Soto, Alexandra Serris, Irati Ormazabal-Vélez, Ali S. Omrani, Milan Navrátil, Sonia Martín-Pérez, Joyce Marques De Almeida, Sylvain Lamure, Martin Kolditz, Ozren Jaksic, Martin Hoenigl, Carolina Garcia-Vidal, Noemí Fernández, Shaimaa El-Ashwah, Natasha Čolović, Martin Čerňan, Caterina Buquicchio, Valentina Bonuomo, Josip Batinić, Murtadha Al-Khabori, Tatjana Adžić-Vukičević, Juan-Alberto Martín-González, Maria Vittoria Sacchi, María-Josefa Jiménez-Lorenzo, Dominik Wolf, Maria Vehreschild, Raul Cordoba, Ramón García-Sanz, Toni Valković, Miloš Mladenović, Nicole García-Poutón, Ziad Emarah, and Julio Dávila-Valls

Subjects
allogeneic HSCT, COVID-19 infection, immunocompromised patients, SARS-CoV-2, hematological malignances, Immunologic diseases. Allergy, RC581-607
Abstract

BackgroundThe outcome of COVID-19 in allogeneic hematopoietic stem cell transplantation (HSCT) recipients is almost uniformely considered poor. The aim of present study was to retrospectively analyse the outcome and risk factors for mortality in a large series of patients who developed COVID-19 infection after an allogeneic HSCT.MethodsThis multicenter retrospective study promoted by the European Hematology Association – Infections in Hematology Study Working Group, included 326 adult HSCT patients who had COVID-19 between January 2020 and March 2022.ResultsThe median time from HSCT to the diagnosis of COVID-19 was 268 days (IQR 86-713; range 0-185 days). COVID-19 severity was mild in 21% of the patients, severe in 39% and critical in 16% of the patients. In multivariable analysis factors associated with a higher risk of mortality were, age above 50 years, presence of 3 or more comorbidities, active hematologic disease at time of COVID-19 infection, development of COVID-19 within 12 months of HSCT, and severe/critical infections. Overall mortality rate was 21% (n=68): COVID-19 was the main or secondary cause of death in 16% of the patients (n=53).ConclusionsMortality in HSCT recipients who develop COVID-19 is high and largely dependent on age, comorbidities, active hematologic disease, timing from transplant and severity of the infection.

Published
2023
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47. 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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48. Germ line DDX41 mutations define a unique subtype of myeloid neoplasms

Author

Makishima, Hideki, Saiki, Ryunosuke, Nannya, Yasuhito, Korotev, Sophia, Gurnari, Carmelo, Takeda, June, Momozawa, Yukihide, Best, Steve, Krishnamurthy, Pramila, Yoshizato, Tetsuichi, Atsuta, Yoshiko, Shiozawa, Yusuke, Iijima-Yamashita, Yuka, Yoshida, Kenichi, Shiraishi, Yuichi, Nagata, Yasunobu, Kakiuchi, Nobuyuki, Onizuka, Makoto, Chiba, Kenichi, Tanaka, Hiroko, Kon, Ayana, Ochi, Yotaro, Nakagawa, Masahiro M., Okuda, Rurika, Mori, Takuto, Yoda, Akinori, Itonaga, Hidehiro, Miyazaki, Yasushi, Sanada, Masashi, Ishikawa, Takayuki, Chiba, Shigeru, Tsurumi, Hisashi, Kasahara, Senji, Muller-Tidow, Carsten, Takaori-Kondo, Akifumi, Ohyashiki, Kazuma, Kiguchi, Toru, Matsuda, Fumihiko, Jansen, Joop H., Polprasert, Chantana, Blombery, Piers, Kamatani, Yoichiro, Miyano, Satoru, Malcovati, Luca, Haferlach, Torsten, Kubo, Michiaki, Cazzola, Mario, Kulasekararaj, Austin G., Godley, Lucy A., Maciejewski, Jaroslaw P., and Ogawa, Seishi

Published
2022
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49. Association between transfusion status and clinical and economic outcomes in patients with myelodysplastic syndromes from the physicians' perspective

Author

Shaloo Gupta, Austin G. Kulasekararaj, Halley Costantino, Jay Grisolano, Jackson Tang, Shalon Jones, and Derek Tang

Subjects
blood transfusion, hospital costs, myelodysplastic syndromes, physicians, survey, treatment outcome, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Abstract Background The current study investigated physicians' understanding of the impact of transfusion status (TS) on clinical and economic outcomes in patients with myelodysplastic syndromes (MDS). Materials & Methods 378 physicians primarily specializing in hematology/oncology across five European countries completed the survey. The survey asked physicians for their perspectives on the impact of TS on risk of death, risk of progression to acute myeloid leukemia (AML), chance of leukemia‐free survival, and number of significant bleeding events, infection events, hospitalizations, and emergency room (ER) visits. Results Physicians estimated that compared to transfusion‐dependent (TD) patients, transfusion‐independent (TI) patients had a 37.6% reduced risk of death, lower risk of progression to AML, and lower risk of non‐leukemic death, for all MDS risk levels. TD patients who became TI after treatment were estimated to have 40.6% reduced risk of death and 34% reduced risk of progression to AML, compared to TD patients who remained TD. Conclusions Compared with TD patients, physicians estimated that TI patients have fewer events of infection and significant bleeding, and experience fewer hospitalizations and ER visits per person per year. Overall, physicians reported better outcomes for TI patients. New treatment options for patients with MDS to reduce or eliminate transfusion burden are warranted.

Published
2023
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