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2111 Worldwide Network for Blood and Marrow Transplantation (WBMT) Global Study on Baseline Characteristics and Clinical Outcomes in NEWLY Diagnosed Multiple Myeloma Patients Undergoing Upfront Autologous STEM Cell Transplantation, a Study Off 61,725 Patients from 629 Centers

Program: Oral and Poster Abstracts
Session: 731. Autologous Transplantation: Clinical and Epidemiological: Poster I
Hematology Disease Topics & Pathways:
Plasma Cell Disorders, Diseases, Lymphoid Malignancies
Saturday, December 10, 2022, 5:30 PM-7:30 PM

Laurent Garderet, MD, PhD1, Luuk Gras2*, Linda Koster3*, Anita D'Souza, MD, MS4, Noel Estrada-Merly5*, Parameswaran Hari, MD, MRCP6, Wael Saber, MD, MS5, Andrew J Cowan, MD7, Minako Iida, MD, PhD8*, Shinichiro Okamoto9*, Hiroyuki Takamatsu, MD, PhD10, Shohei Mizuno, MD, PhD11*, Koji Kawamura12*, Yoshihisa Kodera, MD13*, Nada Hamad, MBBS, BSc, MSc14, Bor-Sheng Ko, MD, PhD15*, Christopher Liam16, KIM Wah HO, MBBS, MRCP17*, A Sim Goh18*, S Keat Tan18*, Alaa M. Elhaddad, MD19, Ali Bazarbachi, MD, PhD20, Qamar un Nisa Chaudhry21*, Rozan Alfar, MBBS22*, Mohamed-Amine Bekadja23*, Malek Benakli, MD, PhD24, Cristobal Augusto Frutos Ortiz, MD25*, Eloisa Riva, MD, MEd26, Sebastian Galeano, MD27, Francisca Bass, MD28*, Hira S Mian, MD29, Arleigh McCurdy, MD, BSc30, Feng Rong Wang31*, Daniel Neumann32*, Mickey Koh, MD, PhD33*, John A Snowden34, Stefan Schönland35*, Donal P. McLornan, MD, PhD36*, Patrick John Hayden, MD37, Anna Sureda38, Hildegard T. Greinix, MD39, Mahmoud Aljurf, MD40, Yoshiko Atsuta41* and Dietger Niederwieser, MD42

1Service Hematologie, Sorbonne Université, Hopital Pitié Salpêtière APHP, Paris, France
2EBMT Statistical Unit, Leiden, The Netherlands, Leiden, Netherlands
3EBMT Data Office, Leiden, Netherlands
4Department of medicine, medical college of Wisconsin, Milwaukee
5CIBMTR® (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI
6Medical College of Wisconsin, Milwaukee, WI
7Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA
8Aichi Medical University School of Medicine, Nagakute-cho, Aichi, JPN
9Keio university school of medicine, Tokyo, Japan
10​School of Entrepreneurial and Innovation Studies, Kanazawa University, Kanazawa, Japan
11Department of Hematology, Aichi Medical University School of Medicine, Nagakute, AIC, Japan
12Division of Hematology, Jichi Medical University Saitama Medical Center, Saitama, SAI, Japan
13Department of Promotion for Blood and Marrow Transplantation, Aichi Medical University School of Medicine, Nagoya, Japan
14Department of Haematology, St Vincent's Hospital Sydney, Australia and St Vincent's Clinical School, Sydney, University of New South Wales, Sydney, NSW, Australia
15National Taiwan University Hospital, Taipei, Taiwan
16Hospital Ampang, Ampang, Selangor, Malaysia
17Ampang Hospital, Ampang Jaya, Malaysia
18Penang general hospital, Penang, Malaysia
19Department of Pediatric Oncology and Stem Cell Transplantation Unit, National Cancer Institute, Cairo University, Cairo, Egypt
20American University of Beirut Dept. of Medicine, Beirut, Lebanon
21AFBMTC/NIBMT, Rawalpindi, Pakistan
22King Hussein cancer center, Amman, ON, Jordan
23Department of Hematology and Cell Therapy, EHU 1st Novembre 1954 Bir el Djir Usto, University Ahmed Benbella 1, Oran, Algeria
24Centre Pierre et Marie Curie (CPMC), Algiers, Algeria
25Hospital Central Del Instituto De Prevision Social, Asuncion, Paraguay
26Cátedra de Hematología, Hospital de Clínicas, Facultad de Medicina, Montevideo, Montevideo, Uruguay
27Hematology Department, British Hospital, Montevideo, Montevideo, Uruguay
28Hospital del Salvador, Santiago, Chile
29Department of Oncology, McMaster University, Hamilton, ON, Canada
30Department of Medicine, Division of Hematology, The Ottawa Hospital, Ottawa, ON, Canada
31Peking University, Beijing, China
32IMISE, university of Leipzig, Leipzig, Germany
33Department of Haematology, St George's Hospital NHS Foundation Trust, London, United Kingdom
34BSBMTCT, Department of Haematology, Sheffield Teaching Hospitals NHS Trust, Sheffield, United Kingdom
35Department of Internal Medicine V, Amyloidosis Center, Heidelberg University Hospital, Heidelberg, Germany
36Department of Haematology, Kings's College Hospital, London, ENG, United Kingdom
37Department of Haematology, Trinity College Dublin, St. James's Hospital, Dublin 8, Leinster, Ireland
38Institut Català d'Oncologia, Hospital Duran i Reynals. Institut d’Investigació Biomèdica de Bellvitge (IDIBELL). Universitat de Barcelona, Barcelona, Spain
39Division of Hematology, Department of Internal Medicine, Medical University of Graz, Graz, Austria
40Oncology Center, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
41Japanese Data Center for Hematopoietic Cell Transplantation, Nagakute, Japan
42University of Leipzig, Leipzig, Germany


Patients with newly diagnosed multiple myeloma (NDMM) undergoing upfront autologous stem cell transplantation (ASCT) have different characteristics worldwide, likely due to variances in transplant activity, patient factors and health economic factors including access to new myeloma therapies. The first aim of this retrospective study was to analyze the global baseline patient characteristics and outcomes of upfront ASCT in NDMM.


Data on patient characteristics and transplant outcomes for patients with NDMM who received an upfront ASCT between 2013 and 2017 were provided to the Worldwide Network for Blood and Marrow Transplantation (WBMT) by 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 Australia and New Zealand Transplant and Cellular Therapies Registry (ANZTCT), the Eastern Mediterranean Blood and Marrow Transplant Group (EMBMT), the Latin American Bone Marrow Transplant Group (LABMT), and the Ottawa Canadian Registry. There primary endpoints were overall (OS) and non-relapse mortality (NRM) and the secondary were progression-free survival (PFS) and relapse incidence (RI). The Kaplan-Meier estimator and log-rank test were used for OS and PFS, and the crude cumulative incidence estimator and Gray’s test were used for competing events (RI and NRM). Age at ASCT, sex, year of ASCT, disease stage, Karnofsky score, MM classification, preparative regimen, time between diagnosis and ASCT, ISS stage at diagnosis, cytogenetic risk, and HCT-CI score were studied for prognostic value. Multivariable (cause specific) models included a random effect for each country and were censored at 3 years for OS and PFS and at 1 year for RI and NRM.


61,725 patients from 629 centers (median patients/center = 67) were included: 61% from EBMT, 26% CIBMTR, 6% APBMT (5% Japan, 1% Taiwan, 0.3% Malaysia), 5% AZTCT, 1% EMBMT, 0.5% LABMT and 0.3% Ottawa, Canada. The patient baseline characteristics are shown in Table 1. Males comprised 58% and the median age at diagnosis was 60 years. The predominant phenotypes were IgG (54%), light chain (24%) and IgA (19%). The ISS stage at diagnosis was I (38%), II (35%) or III (27%) and cytogenetic risk was standard in 70% and high in 30%. The median time from diagnosis to ASCT was 7.1 months. The year of ASCT was equally distributed between 2013 and 2017 (the annual percentage varied between 18% and 22%). The median age at ASCT was 60.8 years with 5.1% of patients older than 70 years. The HCT-CI risk at transplant was low in 52%, intermediate in 25% and high in 23%. The Karnofsky score at ASCT was 100 in 40% and ≤90 in 60%.Disease status was CR in 19%, VGPR in 38%, PR in 36%, MR/SD in 5% and refractory in 2%. The most frequent preparative regimen was melphalan 200 mg/m2 (82 %) and 140 mg/m2 comprised 14%. Tandem ASCT was reported in 6.7%. Of the 11% of patients with data on post-ASCT maintenance treatment, 51% received lenalidomide. The median follow-up was 41.1 months (95% CI: 40.5 to 41.6, IQR:19.2-60.4). Outcomes: non-relapse mortality (NRM) at 12 months was 1% (95% CI 1-2%); OS at 4 and 8 years was 76% (75-76%) and 45% (42-48%) respectively; PFS at 2 and 4 years was 65% (95% CI: 64-65%) and 40% (40-41%) respectively; RI at 6 and 12 months was 7% (7-7%) and 16% (15-16%) respectively. In the multivariate analysis (Table 2), later calendar year of ASCT, a better disease response at time of ASCT, higher Karnofsky score, and an IgG phenotype were all associated with an improved OS. A lower ISS stage, a lower HCT-CI score and standard risk cytogenetics were also associated with better OS (Table 2). Overall NRM was low. Younger age, higher Karnofsky score, higher melphalan dose, better disease response at ASCT, lower ISS stage at diagnosis and lower HCT-CI score were associated with a lower NRM.


This study represents the largest study to date characterizing the outcomes of ASCT performed worldwide. The most frequent preparative regimen was melphalan 200 mg/m2. Unmeasured confounding (pre-existing toxicities/frailties) may partially explain the higher NRM in those with low dose melphalan. Globally, NRM was low at 1% at 12 months, while RI was 16%. Median OS was 7 years, median PFS was 3 years and differed across various risk factors. ASCT remains an effective and safe procedure for patients with NDMM world-wide.

Disclosures: Garderet: Sanofi: Honoraria; BMS: Honoraria; Janssen: Honoraria. D'Souza: Pfizer, Janssen Oncology, Bristol Myers Squibb/Celgene, Prothena: Consultancy, Membership on an entity's Board of Directors or advisory committees; Takeda, Sanofi, TeneoBio, Prothena, Caelum Biosciences, Janssen Oncology, Regeneron, AbbVie: Research Funding. Hari: BMS: Consultancy, Honoraria, Research Funding; Janssen: Consultancy, Honoraria; Karyopharm: Consultancy, Honoraria; AbbVie: Honoraria; Millennium: Research Funding; Kite: Consultancy, Honoraria; Spectrum Pharmaceuticals: Research Funding; Takeda: Consultancy, Honoraria; Incyte: Honoraria; Pharmacyclics: Consultancy; GlaxoSmithKline: Honoraria; Novartis: Honoraria; Amgen: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria; Iovance: Current Employment. 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. Takamatsu: Bristol-Myers Squibb: Honoraria; Sanofi: Honoraria; Ono: Honoraria; Janssen: Honoraria; SRL: Consultancy. Hamad: Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Mian: Takeda: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Janssen: Consultancy, Honoraria, Research Funding; BMS: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria. McCurdy: Takeda: Honoraria; BMS: Honoraria, Research Funding; Janssen: Honoraria; GSK: Honoraria; Sanofi: Honoraria; Amgen: Honoraria; Forus: Honoraria. Snowden: Medac: Membership on an entity's Board of Directors or advisory committees; Janssen and Jazz: Speakers Bureau; Mallinckrodt: Speakers Bureau; Gilead: Speakers Bureau; Kiadis: Other: clinical trial IDMC membership ; Novartis: Speakers Bureau. Schönland: Pfizer: Honoraria; Takeda: Honoraria, Other: Travel Support; Janssen: Honoraria, Other: travel support, Research Funding; Prothena: Honoraria, Other: Travel Support, Research Funding. McLornan: NOVARTIS: Honoraria, Research Funding, Speakers Bureau; JAZZ: Honoraria, Speakers Bureau; ABBVIE: Speakers Bureau; CELGENE BMS: Research Funding, Speakers Bureau. Hayden: Amgen: Other: Participation in Advisory Board. Sureda: SANOFI: Consultancy, Honoraria; BMS: Consultancy, Honoraria; GILEAD: Consultancy; MSD: Honoraria; JANSSEN: Consultancy, Honoraria; ROCHE: Consultancy, Honoraria; NOVARTIS: Consultancy, Honoraria; TAKEDA: Consultancy, Honoraria, Speakers Bureau. Greinix: Gilead, Novartis, Sanofi, Cellgene: Consultancy; Amgen, Gilead, Novartis, Sanofi, Takeda, Therakos: Speakers Bureau. Atsuta: Novartis Pharma KK: Honoraria; Astellas Pharma Inc.: Honoraria; Kyowa Kirin Co., Ltd: Honoraria; AbbVie GK: Honoraria; Mochida Pharmaceutical Co., Ltd.: Honoraria; Meiji Seika Pharma Co, Ltd.: Honoraria.

*signifies non-member of ASH