-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

508.O1.6 508. Bone Marrow Failure: Acquired: Emerging Data in the Treatment of Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Symposia: Bone Marrow Failure: Acquired Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Acquired Marrow Failure Syndromes, Research, Clinical trials, Clinical Practice (Health Services and Quality), Bone Marrow Failure Syndromes, Drug development, Translational Research, Aplastic Anemia, Clinical Research, Paroxysmal Nocturnal Hemoglobinuria, Diseases, Treatment Considerations, Real-world evidence, Biological therapies, Immunotherapy, Registries
Saturday, December 7, 2024: 4:00 PM-5:30 PM
Room 11 (San Diego Convention Center)
Moderators:
Jaroslaw Maciejewski, Taussig Cancer Center and Gloria F. Gerber, MD, Johns Hopkins University
Disclosures:
Gerber: Apellis Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees; Alexion Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees; Merck: Honoraria; Pfizer: Other: spouse employment and received stock.

This session will cover the most recent results of clinical experiences dealing with the treatment of aplastic anemia, including the well-established use of thrombopoietin-mimetic agents in assiciation with immunosuppression, innovative cellular therapies, complement inhibition and hematopoietic stem cell transplantation
4:00 PM

Nazia Matto, PhD1*, Shreyans Gandhi, MD, MBBS, DNB, FRCPath, MPhil, MRCP1,2*, Marta Rzepkowska1*, Sila Gerlevik1*, Mohammad M Karimi3*, Austin G. Kulasekararaj, MD, MBBS, FRCPath, MRCP1,4, Abdel Douiri, PhD5*, Angela Miller6*, Lynne Duran2*, Haili Cui2*, Jen Lewis2*, Shahram Kordasti, MD, PhD7, Giovanna Lombardi, PhD8*, Giorgio Napolitani, PhD1* and Ghulam Jeelani Mufti, MB, DM, FRCP, FRCPath1,2*

1Department of Hemato-oncology, Kings College London, London, United Kingdom
2Kings College Hospital, NHS Foundation Trust, London, United Kingdom
3Comprehensive Cancer Centre, Kings College London, London, United Kingdom
4King's College Hospital NHS Foundation Trust, London, United Kingdom
5Division of Population Health Sciences, King's College London, London, United Kingdom
6LifeArc, London, United Kingdom
7Kings College London, London, United Kingdom
8MRC Centre for Transplantation, King's College London, London, United Kingdom

4:15 PM

Antonio M Risitano, MD, PhD1,2, Simona Iacobelli3*, Austin G. Kulasekararaj, MD, MBBS, FRCPath, MRCP4, Marleen Van Os5*, Sophie Terwel6*, Morag Griffin, MBChB, MRCP7*, Constantijn J.M. Halkes, MD8*, Christian Recher, MD, PhD9*, Fiorenza Barraco10*, Edouard Forcade, MD, PhD11*, Carlos Vallejo12*, Beatrice Drexler, MD13*, Jean-Baptiste Méar, MD14*, Alexander Smith15*, Emanuele Angelucci, MD16, Reinier Raymakers, MD17, Marco R De Groot18*, Etienne Daguindau, MD19*, Erfan Nur20*, Wilma Barcellini, MD21*, Nigel H. Russell, MD22, Louis Terriou, MD PhD23*, Anna Paola Iori24*, Walter Barberi25*, Anna Sureda Balari, MD, PhD26, Isabel Sanchez-Ortega27*, Blanca Xicoy, MD28*, Isidro Jarque Ramos29*, Jamie Cavenagh30*, Flore Sicre De Fontbrune31,32*, Camilla Frieri33*, Talha Munir, MBBS, PhD34, Jennifer Tjon35*, Suzanne Tavitian, MD36*, Aline Praire37*, Laurence Clement38*, Florence Rabian, MD39*, Luana Marano, MD2*, Anita Hill40,41*, Elena Palmisani42*, Petra Muus, MD43, Serena Marotta44*, Fabiana Cacace45*, Marica Laurino, MD46*, Jakob Passweg Sr.47, Judith Marsh, MD48*, Gerard Socie49,50, Ghulam Mufti51*, Carlo Dufour, MD1,52 and Regis Peffault De Latour31,53,54*

1Severe Aplastic Anemia Working Party, European Group for Blood and Marrow Transplantation (EBMT), Leiden, Netherlands
2Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy
3Centro Di Biostatistica E Bioinformatica Università Tor Vergata, Rome, ITA
4King’s College Hospital-NHS Foundation Trust, NIHR/Wellcome King’s Clinical Research Facility, and King’s College London, London, United Kingdom
5EBMT Data Office, Leiden, NLD
6EBMT Data Office, Leiden, Netherlands
7Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom
8Hematology, Leiden University Medical Center, Leiden, Netherlands
9ICUT oncopole, Toulouse, FRA
10Service d’Hematologie Clinique, Hôpital universitaire, Lyon, France
11Service d'Hématologie Clinique et Thérapie Cellulaire, CHU Bordeaux, Bordeaux, France
12Hospital Clínico Universitario de Santiago, Oviedo, Spain
13Department of Hematology, University Hospital Basel, Basel, Switzerland
14Hematology Clinic, Rennes University Hospital, Rennes, France
15King's College London Rayne Institute, London, GBR
16Hematology and Cellular Therapy, IRCCS Ospedale Policlinico San Martino, Genova, ITA
17University Medical Centre, Utrecht, Netherlands
18University Medical Center Groningen, Groningen, Netherlands
19Department of Clinical Hematology, Hopital Jean Minjoz, Service d'Onco-hématologie, Besançon, France
20Department of Hematology, Amsterdam University Medical Centers, Amsterdam, Netherlands
21Hematology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
22Nottingham University Hospital (City Hospital Campus), Nottingham, ENG, United Kingdom
23Lille University Hospital, Lille, FRA
24Department of Translational and Precision Medicine, Policlinico Umberto I Hospital, Sapienza University of Rome, Roma, Roma, ITA
25Policlinico Umberto I Hospital, Sapienza University of Rome, Department of Translational and Precision Medicine, ITA, Rome, Italy
26Hematology Department, Institut Catala d'Oncologia, Hospital Duran i Reynals, L'Hospitalet De Llobregat, Spain
27Institut Catalá d'Oncologia – Hospital Duran i Reynals, Barcelona, Spain
28Hematology Service, ICO-Hospital Germans Trias i Pujol, Institut de Recerca contra la Leucèmia Josep Carreras (IJC), Universitat Autònoma de Barcelona, Badalona, Spain
29University Hospital La Fe, Valencia, Spain
30St. Bartholomew's Hospital London, London, United Kingdom
31Hematology and Transplant Unit, Saint Louis Hospital, APHP, Paris, France
32French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint Louis Hospital, Paris, France
33Hematology and Transplant Unit, A.O.R.N. S. Giuseppe Moscati, Avellino, Italy
34Department of Haematology, St. James's University Hospital, Leeds, United Kingdom
35Leiden University Medical Center, Leiden, NLD
36Department of Hematology, Toulouse University Cancer Institute Oncopole, Toulouse University Hospital, Toulouse, France
37Centre Hospitalier Lyon Sud, Lyon, France
38CHU Bordeaux, Hopital Haut-Leveque, Pessac, France
39Hematology Adolescents and Young Adult Unit, Saint-Louis Hospital, APHP, Paris, France, Paris, France
40Alexion Pharmaceuticals Inc, London, GBR
41St James University Hospital Leeds, Leeds, United Kingdom
42Gaslini Children Hospital, Genoa, Italy
43Department of Haematology, St James's University Hospital, Leeds, United Kingdom
44AORN "A. Cardarelli", Napoli, ITA
45Stem Cell Transplantation and Cell Therapy Unit, AORN Santobono Pausilipon, Naples, Italy
46UO Ematologia e terapie cellulari, IRCCS Ospedale Policlinico San martino, Genova, Italy
47University Hospital Basel, Basel, Switzerland
48King's College Hospital, London, GBR
49Hematology Transplantation, Paris, France
50French Reference Center for Aplastic Anemia, CHU Saint Louis, Paris, France
51King's College Hospital, London, United Kingdom
52Hematology Unit, IRCCS G. Gaslini Children's Hospital, Genova, Italy
53French Référence Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France
54Hematology and Transplantation Unit, Hôpital Saint Louis, AP-HP, Paris, France

4:30 PM

Zoe K McQuilten, MBBS, PhD1, Lucy C Fox, BSc, FRACP, FRCPA, MBBS2,3, Stephane Heritier, PhD4*, Vanessa Fox, BSc4*, Lauren Young, PhD4*, Piers Blombery, MBBS5, Ilona Cunningham, MBBS FRACP FRCPA6, Jennifer Curnow, MBBS, FRACP, FRCPA, PhD7*, Alisa Higgins2*, Devendra Hiwase, MD, MBBS, PhD, FRACP, FRCPA8,9, Robin Filshie, PhD, FRACP, FRCPA10, Frank Caleb Firkin11*, Paul Lacaze12*, Kylie Mason, MBBS, PhD, FRACP, FRCPA13, Anthony K. Mills, MBBS, FRACP, FRCPA14, Dominic Pepperell15*, Sushrut S. Patil16*, Robyn Sutherland, BN, BSc4*, William S. Stevenson, MBBS, PhD17, Jeffrey Szer, MBBS18, Le Thi Phuong Thao, PhD19*, Neil A Waters, BSc20*, Kate Wilson21*, Stephen Ting, MBBS, FRACP, FRCPA, PhD22 and Erica M. Wood, MD2

1Transfusion Research Unit, School of Public Health and Preventive Medicine, Monash University, Melbourne, Australia
2Monash University, Melbourne, Australia
3Peter MacCallum Cancer Centre, Melbourne, Australia
4School of Public Health and Preventative Medicine, Monash University, Melbourne, Australia
5Peter Maccallum Cancer Centre, East Melbourne, VIC, Australia
6Haematology Department Concord Hospital, Concord and University of Sydney, Sydney, Australia
7Westmead Hospital, Sydney, AUS
8South Australian Health and Medical Research Institute, Adelaide, SA, Australia
9Royal Adelaide Hospital, Adelaide, SA, Australia
10St. Vincent's Hospital Melbourne, Fitzroy, VIC, AUS
11St. Vincent's Hospital, Fitzroy, VIC, AUS
12Monash University, Melbourne, AUS
13Royal Melbourne Hospital, Parkville, VIC, AUS
14Division of Cancer Services, Princess Alexandra Hospital, Brisbane, QLD, Australia
15Fiona Stanley Hospital, Murdoch, AUS
16Department of Malignant Haematology & Stem Cell Transplantation, The Alfred Hospital, Melbourne, VIC, Australia
17Royal North Shore Hospital, Redfern, NSW, Australia
18Department of Clinical Haematology, Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, Melbourne, VIC, Australia
19School of Public Health and Preventive Medicine, Monash University, Melbourne, AUS
20Transfusion Research Unit, School of Public Health and Preventive Medicine, Monash University, Melbourne, AUS
21University of Sydney, Sydney, Australia
22Department of Haematology, Eastern Health and Monash University, Elwood, VIC, Australia

4:45 PM

Fengkui Zhang1*, Xin Zhao1, Hong Chang2*, Bing Han3*, Sujun Gao4*, Miao Miao, MD5*, Jianda Hu, PhD6, Yanjuan Lin6*, Xinjian Liu7*, Lina Zhang7*, Chenghao Jin8*, Yingmei Li9*, Shengyun Lin10*, Jinsong Jia11*, Yi Wang12*, Chunkang Chang13*, Zheng Ge, MD, PhD14, Guangsheng He15*, Ruixiang Xia16*, Xiangjun Fu17*, Yuhua Li18*, Hong Liu19*, Kai Sun20*, Shunqing Wang21*, Linghui Xia22*, Weihua Zhang23*, Runzi Li24*, Junye Xiong24* and Wanyi Zhai24*

1National Clinical Research Center for Blood Diseases, State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
2West China Hospital of Sichuan University, Chengdu, China
3Department of Hematology, Chinese Academy of Medical Sciences, Peking Union Medical College Hospital, Beijing, China
4The First Hospital of Jilin University, Changchun, China
5The First Affiliated Hospital of Soochow University, Suzhou, China
6Fujian Medical University Union Hospital, Fuzhou, China
7Henan Cancer Hospital, Affiliated Cancer Hospital of Zhengzhou University, Zhengzhou, China
8Jiangxi Provincial People's Hospital, Nanchang, China
9The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China
10The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, China
11Peking University People’s Hospital, Beijing, China
12Shaanxi Provincial People's Hospital, Xi'an, China
13Shanghai Sixth People's Hospital, Shanghai, China
14Zhongda Hospital Southeast University, Nanjing, China
15The First Affiliated Hospital with Nanjing Medical University, NANJING, China
16The First Affiliated Hospital of Anhui Medical University, Hefei, China
17Hainan General Hospital, Haikou, China
18Zhujiang Hospital, Southern Medical University, Guangzhou, China
19Affiliated Hospital of Nantong University, Nantong, China
20Henan Provincial People's Hospital, Zhengzhou, China
21Guangzhou First People's Hospital, Guangzhou, China
22Union Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
23First Hospital of Shanxi Medical University, Taiyuan, China
24Jiangsu Hengrui Pharmaceuticals Co., Ltd., Shanghai, China

5:00 PM

Camilla Frieri1,2*, Dirk-Jan Eikema Sr.3*, Joe Tuffnell4*, Brian Piepenbroek5*, Malek Benakli, MD6*, Grzegorz Helbig, MD, PhD7*, Thomas Cluzeau, MD, PhD8*, Cécile Renard, MD9*, Philippe Lewalle10*, Victoria Potter11*, Krzysztof Kalwak Sr.12*, Johan Maertens, MD, PhD13*, Gerard Michel, MD, PhD14*, Andrew Bruce McDonald, MBChB15, Péter Pál Reményi, MD16*, Ladislav Sopko17*, Kazimierz Halaburda18*, Depei Wu, MD, PhD19, Zubeyde Nur Ozkurt20*, Xavier Poiré, MD, PhD21*, Morag Griffin, MBChB, MRCP22*, Mahmoud Aljurf23*, Ashrafsadat Mousavi24*, Jose Antonio Pérez-Simón, MD, PhD25, Henrik Sengeloev26*, Ben Carpenter, MD27*, Eric Deconinck28, Arancha Bermúdez29*, Stéphanie Nguyen-Quoc, MD, PhD30*, Matteo Parma, MD31*, Ibrahim Yakoub-Agha, MD, PhD32*, Shankara Paneesha33*, Lutz Peter Hermann Mueller, MD34*, Regis Peffault De Latour35,36*, Austin G. Kulasekararaj, MD, MBBS, FRCPath, MRCP37 and Antonio M Risitano, MD, PhD38,39

1Hematology and Transplant Unit, A.O.R.N. S. Giuseppe Moscati, Avellino, Italy
2European Bone Marrow Transplantation society, Severe Aplastic Anemia Working Party, Leiden, Netherlands
3EBMT Statistical Unit, Leiden, Netherlands
4EBMT Leiden Study Unit, Leiden, Netherlands
5EBMTClinical Study Unit, Leiden, Netherlands
6Pierre and Marie Curie Center, Algiers, Algeria
7Department of Hematology and Bone Marrow Transplantation, Silesian Medical Academy, Katowice, Poland
8Centre Hospitalier Universitaire de Nice, Nice, France
9Institute of Hematology and Pediatric Oncology, Lyon, France
10Jules Bordet Institute (HUB/ULB), Brussels, Belgium
11Dept. of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, United Kingdom
12Fundacja "Na Ratunek Dzieciom z Choroba Nowotworowa", Wroclaw, Dolnoslaskie, Poland
13Department of Hematology, University Hospital Gasthuisberg, Leuven, Belgium
14CHU de Marseille Hôpital de la Timone Enfants, Marseille, France
15This, Randburg, ZAF
16Department of Hematology and Stem Cell Transplantation, Central Hospital of Southern Pest, National Institute of Hematology and Infectious Diseases, Budapest, Hungary
17University Hospital, Bratislava, Slovakia
18Institute of Hematology and Transfusion Medicine, Warsaw, Poland
19Department of Hematology, Collaborative Innovation Center of Hematology, Institute of Blood and Marrow Transplantation, the First Affiliated Hospital of Soochow University, Suzhou, China
20Gazi University Faculty of Medicine, Ankara, Turkey
21Section of Hematology, Institut Roi Albert II, Cliniques Universitaires St-Luc, Brussels, Belgium
22Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom
23King Faisal Specialist Hospital & Research Centre, Oncology (Section of Adult Haematolgy/BMT) – Riyadh, Saudi Arabia, Riyadh, Saudi Arabia
24Shariati Hospital, Teheran, Iran (Islamic Republic of)
25Department of Hematology, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBIS), CSIC, University of Sevilla, Sevilla, Spain
26Rigshospitalet, Copenhagen, Denmark
27University College London Hospitals NHS Foundation Trust, London, United Kingdom
28Université de Franche-Comté, EFS, INSERM, UMR RIGHT, Besançon, France
29Department of Hematology, Hospital Universitario Marqués de Valdecilla, Cantabria, Spain
30Hematology Department, Pitié-Salpêtrière University Hospital, AP-HP, Paris, France
31Hematology Department, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
32CHU de Lille, Université de Lille, INSERM U1286, Infinite, 59000, Lille, France
33Birmingham Centre for Cellular Therapy and Transplant (BCCTT), Birmingham, United Kingdom
34Department of Internal Medicine IV, Martin-Luther-Universität Halle-Wittenberg, Halle, Germany
35BMT unit, Hôpital Saint-Louis,, Paris, France
36French Référence Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France
37King’s College Hospital-NHS Foundation Trust, NIHR/Wellcome King’s Clinical Research Facility, and King’s College London, London, United Kingdom
38Severe Aplastic Anemia Working Party, European Group for Blood and Marrow Transplantation (EBMT), Leiden, Netherlands
39Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy

5:15 PM

Christopher Patriquin1*, Jun-Ho Jang, MD., PhD.2, Lisa Aurand3, Deepak Taneja3*, Andrew Magyar3*, Bradley Dain3*, Karoline Meagher3*, Lorah Perlee3*, Amal Souttou3* and Morag Griffin, MBChB, MRCP4*

1University Health Network, Toronto General Hospital, Toronto, ON, Canada
2Samsung Medical Center, Seoul, Korea, Republic of (South)
3Regeneron Pharmaceuticals, Inc., Tarrytown, NY
4St James’s University Hospital, Leeds, United Kingdom

*signifies non-member of ASH