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3638 An Analysis of the Worldwide Utilization of Hematopoietic Stem Cell Transplantation for Acute Myeloid LeukemiaClinically Relevant Abstract

Program: Oral and Poster Abstracts
Session: 906. Outcomes Research—Myeloid Malignancies: Poster II
Hematology Disease Topics & Pathways:
Research, Acute Myeloid Malignancies, AML, Non-Biological therapies, epidemiology, Clinical Research, health disparities research, Diseases, real-world evidence, registries, Myeloid Malignancies
Sunday, December 11, 2022, 6:00 PM-8:00 PM

Molly Tokaz, MD1*, Helen Baldomero2*, Andrew J Cowan, MD3,4, Wael Saber, MD, MS5, Hildegard T. Greinix, MD6, Mickey Koh, MD, PhD7*, Nicolaus Kröger, MD8*, Mohamad Mohty, MD PhD9,10, Sebastian Galeano, MD11, Shinichiro Okamoto, MD, PhD12, Naeem A. Chaudhri, MD13, Amado J Karduss, MD14, Fabio Ciceri, MD15*, Vergilio Rensi Colturato, MD16*, Selim Corbacioglu, MD17, Alaa M. Elhaddad, MD18, Lisa Force, MD, MPH19*, Cristobal Augusto Frutos Ortiz, MD20*, Andres Gomez-De Leon, MD21, Nada Hamad, MBBS, BSc, MSc22, Nelson Hamerschlak, MD, PhD23, Naya He, MPH5*, Aloysius YL Ho, MBBS, FRCP, FRCPath24, Xiao-Jun Huang25, Benjamin Jacobs, MD26, Heeje Kim, MD, PhD27, Minako Iida, MD, PhD28*, Leslie E. Lehmann, MD29, Regis Peffault De Latour30*, Mary-Elizabeth M. Percival, MD4,31, Walid Rasheed, MB, ChB, FRACP, FRCPA13, Kirk R. Schultz, MD32, Adriana Seber, MD, MS33, Bor-Sheng Ko, MD, PhD34*, Anderson Simione35*, Alok Srivastava36, Jeff Szer, MBBS, FRACP37, William A. Wood, MD, MPH38, Yoshihisa Kodera, MD39*, Arnon Nagler, MD40, John A Snowden41, Daniel J. Weisdorf, MD42, Jakob R. Passweg, MD, MS43, Marcelo C Pasquini, MD, MS44, Anna Sureda45, Yoshiko Atsuta46*, Mahmoud Aljurf, MD13, Dietger Niederwieser, MD47 and Martina Perdomo48*

1Fred Hutchinson Cancer Center, Seattle, WA
2The Worldwide Network for Blood and Marrow Transplantation (WBMT) Activity Survey Office, University Hospital Basel, Basel, Switzerland
3Division of Medical Oncology, Fred Hutchinson Cancer Research Center, Seattle, WA
4Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA
5CIBMTR® (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI
6Division of Hematology, Department of Internal Medicine, Medical University of Graz, Graz, Austria
7Department of Haematology, St George's Hospital NHS Foundation Trust, London, United Kingdom
8University Medical Center Hamburg-Eppendorf, Department of Stem Cell Transplantation, Hamburg, Germany
9EBMT Paris study office / CEREST-TC, Paris, France, Department of Hematology, Saint Antoine Hospital, Paris, France, INSERM UMR 938, Sorbonne University, Paris, France
10Acute Leukemia Working Party, Paris Study Office, European Society for Blood and Marrow Transplantation, Paris, France
11Hematology Department, British Hospital, Montevideo, Montevideo, Uruguay
12Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan
13Oncology Center, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
14Bone Marrow Transplantation Program, Cancer Institute Las Americas AUNA, Medellin, Colombia
15Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy
16Hospital Amaral Carvalho, Jaú, Brazil
17Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany
18Department of Pediatric Oncology and Stem Cell Transplantation Unit, National Cancer Institute, Cairo University, Cairo, Egypt
19Division of Pediatric Hematology-Oncology and Institute for Health Metrics and Evaluation, University of Washington, Seattle, WA
20Hospital Central Del Instituto De Prevision Social, Asuncion, Paraguay
21Servicio de Hematología, Universidad Autonoma de Nuevo Leon, Hospital Universitario "Dr. José Eleuterio Gonzalez", Monterrey, Mexico
22Department of Haematology, St Vincent's Hospital Sydney, Australia and St Vincent's Clinical School, Sydney, University of New South Wales, Sydney, NSW, Australia
23Hospital Israelita Albert Einstein, Sao Paolo, Brazil
24Department of Hematology, Singapore General Hospital, Singapore, Singapore
25Peking University People’s Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China, Beijing, China
26Hospital of the University of Pennsylvania, Media, PA
27Department of Hematology, Catholic Hematology Hospital, Seoul St. Mary's Hospital, College of Medicine, The Catholic Univ. of Korea, Seoul, Korea, Republic of (South)
28Aichi Medical University School of Medicine, Nagakute-cho, Aichi, JPN
29Division of Pediatric Hematology/Oncology/Stem Cell Transplant Program, Dana-Farber/Boston Children’s Cancer & Blood Disorders Center, Boston, MA
30Assistance Publique–Hôpitaux de Paris, Saint-Louis Hospital, Université Paris Cité, Paris, France
31Division of Hematology, Department of Medicine, University of Washington, Seattle, WA
32B.C. Children's Hospital, Vancouver, BC, Canada
33Hospital Samaritano, Sao Paulo, SP, Brazil
34National Taiwan University Hospital, Taipei, Taiwan
35Hospital Amaral Carvalho, Jaú, BRA
36Department of Hematology, Christian Medical College, Vellore, India
37Department of Clinical Haematology, The Royal Melbourne Hospital and Peter MacCallum Cancer Centre, Melbourne, VIC, Australia
38Division of Hematology, Department of Medicine, University of North Carolina, Chapel Hill, NC
39Department of Promotion for Blood and Marrow Transplantation, Aichi Medical University School of Medicine, Nagoya, Japan
40Hematology and Bone Marrow Transplant Unit, Chaim Sheba Medical Center, Tel Hashomer, Israel
41BSBMTCT, Department of Haematology, Sheffield Teaching Hospitals NHS Trust, Sheffield, United Kingdom
42Department of Medicine, Division of Hematology, Oncology, and Transplantation, University of Minnesota, Minneapolis, MN
43Hematology, University Hospital Basel, Basel, Switzerland
44CIBMTR® (Center for International Blood and Marrow Transplant Research), Department of Medicine,, Froedtert & The Medical College Cancer Ctr., Milwaukee, WI
45Clinical Hematology Department, Institut Català d'Oncologia-Hospitalet; Institut d'Investigació Biomèdica de Bellvitge (IDIBELL); Biomedical Research Institute, Universitat de Barcelona, Barcelona, Spain
46Department of Registry Science for Transplant and Cellular Therapy, Aichi Medical University School of Medicine, Nagakute, Japan
47University of Leipzig, Leipzig, Germany
48Hospital Británico, Montevideo, Uruguay


Acute myeloid leukemia (AML) has an aggressive course and a historically dismal prognosis. For many patients, hematopoietic stem cell transplantation (HSCT) represents the best option for cure, but access, utilization and health inequities on a global scale remain poorly elucidated.


Estimates of AML incident cases in 2016 were obtained from the Global Burden of Disease (GBD) 2019 study. HSCT activities were collected from 2009-2016 by the Worldwide Network for Blood and Marrow Transplantation (WBMT) through the European Society for Blood and Marrow Transplantation (EBMT), the Center for International Blood and Marrow Transplantation (CIBMTR), the Asian Pacific Blood and Marrow Transplant Group (APBMT), the Australasian Bone Marrow Transplant Recipient Registry (ABMTRR), the Eastern Mediterranean Blood and Marrow Transplant Group (EMBMT), the Latin American Bone Marrow Transplantation Group (LABMT), the African Blood and Marrow Transplantation Group (AFBMT) and the Cell Therapy Transplant Canada (CTTC). The primary endpoint was to analyze the global and regional use and utilization of HSCT (number of HSCT/ number of incident cases) for AML. Secondary outcomes included trend from 2009 to 2016, donor type, stem cell source and remission status.


Global AML incidence has steadily increased, from 101,867 (95% uncertainty interval (UI): 90,240-107,950) in 2009 to 118,404 (95% UI 103,620-125,950) in 2016 (+16.2%; see Table 1). Over the same period globally, a 54.9% increase from 9,659 to 14,965 HSCT/year was observed, driven by an increase in allogeneic (+64.9%) with a simultaneous reduction in autologous (-34.9%) HSCT. While highest numbers of HSCT continue to be performed in high- income regions, the largest increases were seen in resource-constrained regions [+94.6% in Africa/East Mediterranean Region (AFR/EMR)] as compared to high-income regions [+34.7% in America-Nord Region (AMR-N)]. HSCT utilization was skewed towards high-income regions [in 2016: AMR-N 18.4%, Europe (EUR) 17.9%, South East Asia/Western Pacific Region (SEAR/WPR) 11.7%, America-South Region (AMR-S) 4.5% and AFR/EMR 2.8%]. For patients <70 years of age this difference in utilization was widened (Figure 1); AMR-N has the highest allogeneic utilization rate, increasing from 2009 to 2016 (30.6% to 39.9%, respectively) with continued low utilization observed in AFR/EMR (1.7% to 2.9%) and AMR-S (3.5% to 5.4%, respectively). In all regions, total HSCT for AML in 1st complete remission (CR1) increased (from 44.1% to 59.0%). Patterns of donor stem cell source from related versus unrelated donors varied widely by geographic region. SEAR/WPR has had a +130.2% increase in related donor from 2009 to 2016 and >95% HSCT donors in AFR/EMR are from related as compared to a predilection for unrelated HSCT in AMR-N and EUR. Globally, stem cell source for allogeneic HSCT was predominantly from peripheral blood with increasing use from 2009 to 2016. Autologous HSCT decreased in all regions except in SEAR/WPR (+18.9%, see Table 1)


HSCT remains a central curative treatment modality in AML and an understanding of global access, practices and unmet needs is important. Allogeneic HSCT for AML is rising globally but there are marked variations in regional utilization and practices, including types of graft source. Resource-constrained regions have the largest growth in HSCT use, but utilization rates remain low with a predilection for familial related donor sources and are typically offered in CR1. Further studies are necessary to elucidate the reasons, including economic factors, to understand and address these health inequalities and improve discrepancies in use of HSCT as a potentially curative treatment globally.

Disclosures: Cowan: Abbvie: Consultancy, Research Funding; Adaptive: Membership on an entity's Board of Directors or advisory committees; Allogene: Consultancy; BMS: Consultancy, Research Funding; EUSA: Consultancy; GSK: Consultancy; Harpoon: Research Funding; Janssen: Consultancy, Research Funding; Nektar: Research Funding; Sanofi: Research Funding; Secura Bio: Consultancy. Greinix: Gilead, Novartis, Sanofi, Cellgene: Consultancy; Amgen, Gilead, Novartis, Sanofi, Takeda, Therakos: Speakers Bureau. Kröger: Takeda: Consultancy, Honoraria; Sanofi: Honoraria; Kite: Honoraria; Neovii: Honoraria, Research Funding; Riemser: Research Funding; DKMS: Research Funding; Amgen: Honoraria; BMS: Honoraria, Research Funding; Novartis: Honoraria, Research Funding; Jazz: Honoraria. Mohty: Oncopeptides: Honoraria; Pfizer,: Honoraria; Adaptive Biotechnologies: Honoraria; Novartis: Honoraria; Astellas: Honoraria; Amgen: Honoraria; Takeda: Honoraria; Bristol Myers Squibb: Honoraria; Celgene: Honoraria; Sanofi: Honoraria, Research Funding; Jazz Pharmaceuticals: Honoraria, Research Funding; GSK: Honoraria; Gilead: Honoraria; Janssen: Honoraria, Research Funding. Ciceri: Kite Pharma: Consultancy. Gomez-De Leon: AbbVie: Honoraria, Other: advisory board. Hamad: Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Peffault De Latour: Samsung: Consultancy, Honoraria; Sobi: Consultancy, Honoraria; MSD: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria, Research Funding; Roche: Consultancy, Honoraria; Keocyte: Consultancy, Honoraria; Jazz: Consultancy, Honoraria, Research Funding; Gilead: Consultancy, Honoraria; Amgen: Consultancy, Honoraria, Research Funding; Alexion: Consultancy, Honoraria, Research Funding; Novartis: Consultancy, Honoraria, Research Funding. Percival: Pfizer: Research Funding; Trillium: Research Funding; Oscotec: Research Funding; Cardiff Oncology: Research Funding; Glycomimetics: Research Funding; Biosight: Research Funding; Celgene/BMS: Research Funding; Abbvie: Research Funding; Ascentage: Research Funding; Telios: Research Funding. Srivastava: Roche: Research Funding; Pfizer: Research Funding; Sanofi: Other: The study was supported by Sanofi, Research Funding; NovoNordisk: Research Funding. Szer: Novartis: Consultancy, Honoraria, Speakers Bureau; Biocryst: Consultancy, Honoraria, Speakers Bureau; Alexion: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CSL: Consultancy, Honoraria, Speakers Bureau; Eli-Lilly: Consultancy, Honoraria, Speakers Bureau; Sobi: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau. Wood: Pfizer: Other: Research funding to my institution; Genentech: Other: Research funding to my institution. Snowden: Mallinckrodt: Speakers Bureau; Novartis: Speakers Bureau; Gilead: Speakers Bureau; Janssen and Jazz: Speakers Bureau; Medac: Membership on an entity's Board of Directors or advisory committees; Kiadis: Other: clinical trial IDMC membership . Weisdorf: Incyte: Other: Research support; FATE Therapeutics: Other: Research Support. Pasquini: Kite: Research Funding; Novartis: Research Funding; Janssen: Research Funding; Bristol Myers Squibb: Consultancy, Research Funding. Sureda: Takeda: Consultancy, Honoraria, Research Funding, Speakers Bureau; BMS/Celgene: Consultancy, Honoraria, Research Funding; MSD: Consultancy, Honoraria; Kite: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; Jannsen: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria; GenMab: Consultancy, Honoraria; Pierre Fabre: Consultancy, Honoraria; Astra Zeneca: Consultancy, Honoraria. Atsuta: Novartis Pharma KK: Honoraria; Kyowa Kirin Co., Ltd: Honoraria; AbbVie GK: Honoraria; Astellas Pharma Inc.: Honoraria; Mochida Pharmaceutical Co., Ltd.: Honoraria; Meiji Seika Pharma Co, Ltd.: Honoraria.

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