Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study

S Steven W. Pipe (Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor) L Laurel A. Menapace (17Sanofi, Cambridge, MA) C Chuanwu Zhang (9Sanofi, Waltham, United States) Y Yuqian Shen (4Sanofi, Morristown, United States) M Marja Puurunen (17Sanofi, Cambridge, MA) M Marek Demissie (2Sanofi, Cambridge, United States) T Teh-Liane Khoo (8Royal Prince Alfred Hospital, Sydney, Australia) S Scott Dunkley S Stephanie P'ng (4The Haemophilia and Haemostasis Centre, Fiona Stanley Hospital, Murdoch, Australia) D Dominic Pepperell S Stephanie Lam E Elizabeth Steinepreis J Jacques Malherbe (3Farmovs, Bloemfontein, South Africa) T Tulene Kendrick M Marcus Lombard H Huyen Tran (8Department of Clinical Haematology, Alfred Hospital, Melbourne, VIC, Australia) J Jessie Zhao H Hannah Stevens C Caroline Dix J James McFadyen J Jean St-Louis J Jeannine Kassis G Georges Rivard J Jing Sun X Xiaofang Li (Key Laboratory of Cluster Science of Ministry of Education, Beijing Key Laboratory of Photoelectronic/Electrophotonic Conversion Materials, School of Chemistry and Chemical Engineering, Beijing Institute of Technology, 5 South Zhongguancun Street, Haidian District, Beijing 100081, China) H Huiping Li J Jieyu Ye (1Department of Hematology, Nanfang Hospital, Southern medical University, Guangzhou, China) X Xuan Zhou (State Key Laboratory of Agricultural and Forestry Biosecurity, Key Laboratory of Ministry of Education for Genetics, Breeding and Multiple Utilization of Crops, Plant Immunity Center, Fujian Agriculture and Forestry University) D Dong Du X Xuefeng Wang (Beijing National Laboratory for Condensed Matter Physics) J Jing Dai Q Quilan Ding W Wenman Wu Y Yiyi Lu R Runhui Wu G Guoqing Liu (School of Physics and Astronomy, Shanghai Jiao Tong University) W Wanru Yao Y Yan Wang Y Yunyun Wei W Weiqun Xu (20Children's Hospital, Zhejiang University School of Medicine, Hangzhou, China., Hangzhou, China) Y Yu Du (Key Laboratory of Material Simulation Methods and Software of Ministry of Education, College of Physics) L Liyao Xu D Di Wang C Chan Liao H Hongwei Zhang (Key Laboratory of Development and Application of Rural Renewable Energy) Z ziqiang Yu (3National Clinical Research Center for Hematologic Disease, The First Affiliated Hospital of Soochow University, Suzhou, China) H Hong Tian H Haibo Wang J Jie Yin (School of Psychology, Beijing Sport University) T Tienan Zhu (2Peking Union Medical College Hospital, Beijing, China) H Huacong Cai (1Chinese Academy of Medical Sciences & Peking Union Medical College, Peking, China) J Juan Xiao S Shujie Wang (School of Chemical Engineering and Technology, Key Laboratory for Green Chemical Technology of Ministry of Education, Tianjin University) S Shufen Liu K Kaini Shen Y Yun Chen Y Yali Chang T Tan Sang (1Shandong Cancer Hospital and Institute, Shandong First Medical University and Shandong Academy of Medical Sciences, Department of Lymphohematology Ward 2, Jinan, China) J Jun Zhao (Department of Thoracic Oncology Beijing Cancer Hospital Beijing China) Y Yawei Zhou L Liya Zhai X Xiadong Qiu Y Yulei He H Hui Li Y Yi Xia M Min Zhou F Feng Gao S Shipin Feng X Xiaoli Luo D Dan Li Z Zhongguang Cui C Chunzhu Zhao H Hong Xu (Institute of Nuclear and New Energy Technology) L Lingjie Sun (State Key Laboratory of Advanced Materials for Intelligent Sensing, Key Laboratory of Organic Integrated Circuit, Ministry of Education & Tianjin Key Laboratory of Molecular Optoelectronic Sciences, Department of Chemistry, School of Science & Institute of Molecular Aggregation Science, Tianjin University) X Xue Shi X Xiaojing Zeng L Lu Qi (Shandong Provincial Key Laboratory for Science of Material Creation and Energy Conversion, Science Center for Material Creation and Energy Conversion, Institute of Frontier Chemistry, School of Chemistry and Chemical Engineering) S Shuyun Cao Z ZePing Zhou (1The Second Affiliated Hospital of Kunming Medical University, Department of Hematology, kunming, China) F Fuhou Zhang L Lingmei Yin H Hongli Mu H Hui Bi (Shanghai Institute of Ceramics, Chinese Academy of Sciences) Z Zhengchao Li Y Yaping Miu Y Yaming Xi M Mingfeng Jia L Long Zhao (Key Laboratory of Evolution and Marine Biodiversity (Ministry of Education) and Institute of Evolution and Marine Biodiversity, Ocean University of China, Qingdao, China.) X Xielan Zhao (13Xiangya Hospital Central South University, Changsha, China) Y Yaochun Li J Jie Peng (Institute of Intelligent Innovation, Henan Academy of Sciences) C Chia-Yau Chang (2Taipei Medical University, Division of Pediatric Hematology/Oncology, Department of Pediatrics, Taipei, Taiwan) Y Yen-Lin Liu (1Division of Pediatric Hematology/Oncology, Department of Pediatrics, Taipei Medical University Hospital, Taiwan, Taipei, Taiwan) M Ming-Ching Shen (5Department of Internal Medicine, Changhua Christian Hospital, Changhua, Taiwan) C Cheng-Wei Huang S Shih-Chung Wang (7Changhua Christian Hospital, Changhua, Taiwan) H Hsuan-Yu Lin C Ching-Yeh Lin S Sheng-Chieh Chou (9Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan) M Ming-Kai Chuang S Szu-Chun Hsu J Jiaan-Der Wang (6Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan) M Ming-Yang Shih L Liang-Tsai Hsiao G Giun-Yi Hung H Hsiu-ju Yen (11Taipei Veterans General Hospital and National Yang-Ming University, Taipei, Taiwan) P Po-Shen Ko (1Taipei Veterans General Hospital, Division of Hematology and Oncology, Department of Medicine, Taipei, Taiwan) H Hung Chang (20Division of Hematology-Oncology, Department of Internal Medicine, Chang Gung Memorial Hospital, Taipei, Taiwan) S Shih-Hsaing Chen T Te-Fu Weng Y Yu-Hua Chao S Sherig-Wei Zeng P Peter Kampmann (5Rigshospitalet, Department of Hematology, Copenhagen, Denmark) E Eva Funding Y Yesim Dargaud (1Hospices Civils de Lyon, Service d'Hématologie et d'Hémostase Clinique, Lyon, France) C Claude Negrier S Stéphanie Desag L Laurent Frenzel G Guillemette Fouquet C Cécile Bally R Robert Klamroth (14Vivantes Clinic Friedrichshain, Internal Medicine, Berlin, Germany) A Ana-Marija Orlovic C Christian Pfrepper (4Division of Hemostaseology, Department of Hematology, Cellular Therapy, Hemostaseology and Infectiology, University of Leipzig Medical Centre, Leipzig, Germany) M Michael Metze R Rima Chakaroun M Maria Weise L László Nemes A Agnes Ronatas C Cecil Ross (1St John's Medical College Hospital, Department of Clinical Hematology, Bengaluru, India) S Seetharam Anandram (1St John's Medical College Hospital, Department of Clinical Hematology, Bengaluru, India) V Vandana Bharadwaj S Sonali Salvi S Sunil Lohande A Alok Srivastava (Hematology Research Unit, St. John’s Research Institute, St. John’s National Academy of Health Sciences, Bengaluru, India) A Aby Abraham (3Christian Medical College, Vellore, India) F Fouzia Aboobacker S Shailendra Verma G Gaurav Datta S Savita Rangarajan (6University Hospital Southampton NHS Foundation Trust, South Hampton, United Kingdom) S Shweta Bansal S Shashikant Apte K Kannan Subramanian (35Sahyadri Speciality Hospital Deccan Gymkhana, Pune, India) B Beatrice Nolan (13Children's Health Ireland at Crumlin, Department of Paediatric Haematology, Dublin, Ireland) S Saad Ahmed N Niamh O’Connell J James O’Donnell K Kevin Ryan (3National Coagulation Centre, St James’s Hospital, Dublin, Ireland) M Michelle Lavin (National Coagulation Centre, St. James's Hospital, Dublin, Ireland) G Gili Kenet (1The Amalia Biron Research Institute of Thrombosis & Hemostasis, Faculty of Medical & Health Sciences, Tel Aviv University, Tel Aviv, Israel) O Omri Cohen M Mudi Misgav A Assaf Barg S Sarina Levy Mendelovich F Flora Peyvandi R Roberta Gualtierotti E Ezio Zanon L Luca Spiezia C Chiara Simion T Tadashi Matsushita (Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan) S Shuichi Okamoto N Nobuaki Suzuki T Takeshi Kanematsu N Naruko Suzuki Y Yuka Kawaguchi (4Aichi Cancer Center, Department of Hematology and Cell Therapy, Nagoya, Japan) S Satoshi Higasa (14Hyogo College of Medicine, Department of Hematology, Nishinomiya, Japan) T Tazuko Tokugawa A Akihiro Sawada R Rie Shirayama M Masaru Kawamura H Hiroshi Asai K Koichi Oshida T Tetsuji Sato T Takuma Ito (Department of Chemistry, Graduate School of Science) K Katsuyoshi Koh (23Japan Children’s Cancer Group ALL Committee, Nagoya, Japan) Y Yoshitaka Mizushima T Takahiro Ishikawa H Hirohito Kubota R Ryota Kaneko T Tomoya Irikura M Mamoru Honda K Katsuaki Taira Y Yuki Arakawa (Department of Hematology/Oncology, Saitama Children’s Medical Center) M Makiko Mori (6Saitama Children's Medical Center, Saitama, Japan) K Kohei Fukoka K Koichi Oshima Y Yuichi Mitani Y Yoshiaki Ogawa (24Tokai University of School of Medicine, Kanagawa, Japan) K Kiyoshi Ando R Rikio Suzuki R Ryujiro Hara Y Yasuyuki Aoyama M Masako Toyosaki D Daisuke Ogiya (9Tokai University School of Medicine, Department of Hematology and Oncology, Kanagawa, Japan) S Shinichiro Machida (11Tokai University School of Medicine, Department of Hematology and Oncology, Kanagawa, Japan) H Hiroshi Kawada Y Yukari Shirasugi M Makoto Onizuka K Ken Omachi K Kagehiro Amano T Tomoko Yamaguchi E Ei Kinai T Takeshi Hagiwara M Mihoko Yotsumoto R Ryoko Sekiya M Masato Bingo Y Yushi Chikasawa A Akito Ichiki Y Yoshiko Kamikubo R Ryui Miyashita T Takashi Muramatsu H Ho-jin Shin (Pusan National University Hospital, Busan, Korea, Republic of) D Doyoung Kim C Chur-Woo You (2Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, Republic of Korea) J Ju Young Kim S Seung Min Hahn C Chuhl Joo Lyu (Department of Pediatrics, Yonsei University Severance Hospital, Seoul, South Korea) J Jung Woo Han (1Yonsei University College of Medicine, Department of Pediatrics, Seoul, Korea) Y Young Shil Park J Ja Hyang Cho A Azizan Shariq S Soo Min Lim (7Sultanah Aminah Hospital, Johor Bahru, Malaysia) L Lily Wong H Heng Chang Choo G Gilbert Wilfred M Mohd Tarmizi Ahmad Zakhi Y Yin Man Lim K Khaiteri Raghunathan J Jameela Sathar S Syazarin Fidhora Nasution Y Yee Yee Yap V Veena Selvaratnam D Daniel Malan Z Zelda Erika Punt A Ali Bulent Antmen I Ilgen Sasmaz U Utku Aygunes (29Konya City Hospital, Hematology, Konya, Türkiye) K Kaan Kavakli (3Ege University Faculty of Medicine Children's Hospital, Department of Pediatric Hematology and Oncology, Izmir, Türkiye) N Nihal Karadas D Deniz Karapinar A Alphan Kupesiz (2Akdeniz University School of Medicine, Pediatric Hematology and Oncology, Antalya, Türkiye) M Mine Erkan K Kupesiz Funda Tayfun E Elif Guler B Bulent Zulfikar (2Department of Pediatric Hematology-Oncology, Istanbul University, Oncology Institute, Istanbul, Türkiye) B Basak Koc V Vahap Okan S Sahin Handan Haydaroglu C Canan Albayrak I Ibrahim Kartal E Ebru Yilmaz M Musa Karakükcü (24Erciyes University, Faculty of Medicine, Kayseri, Turkey, Kayseri, Türkiye) E Ekrem Unal A Alper Ozcan (8Erciyes University School of Medicine, Pediatric Hematology and Oncology, Kayseri, Türkiye) F Fahri Sahin B Betul Tuzun G Güray Saydam (9Ege University Medical Faculty Hospital, Department of Internal Diseases, Division of Hematology, Izmir, Türkiye) I Ievgenii Averianov O Oksana Kovtunenko O Oleksandra Stasyshyn M Maksym Semerak O Orest Tushnytskyy K Kateryna Vilchevska I Iryna Bakhchyvandzhy N Nataliya Romanyuk S Stanislav Nikulin A Alina Kobets J Jayanthi Alamelu MRCPCH S Samah Babiker C Catherine Bagot (18Glasgow Royal Infirmary, Glasgow, United Kingdom) J James Tyan Rodgers L Lisa Boggio (4Rush Hemophilia and Thrombophilia Center, Chicago, United States) M Mindy Simpson (1Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago, United States) E Erin Cockrell (11St. Joseph's Hospitals Center for Bleeding and Clotting Disorders, Tampa, United States) C Candance DeBerry D Dana Obzut J John Fargo (1Ann & Robert H. Lurie Children's Hospital, Chicago, United States) J Jeffrey Hord S Stephanie Savelli J Joseph Lasky J Jennifer Buitrago W Waseem Alhushki C Cynthia Gauger D Darren Klawinski E Eric Sandler S Scott Bradfield P Paul Pitel M Michael Joyce (20Department of Pediatrics, Nemours Children’s Health, Jacksonville, FL) H Howard Katzenstein M Manisha Bansal A Allison Bechtel C Christine Knoll E Esteban Gomez S Shanna White (1Phoenix Children's Hospital, Phoenix, United States) S Sanjay Shah (1Phoenix Children's Hospital, Phoenix, United States) J Joanna Gendreau (3Johnson & Johnson, Global Medical Organization, Horsham, United States) L Lori Wagner M Maa-Ohui Quarmyne (1Phoenix Children's Hospital, Phoenix, United States) L Larissa Bornikova C Colleen Cleary CNP P Pamela G. Hodges (Division of Hematology–Oncology, Massachusetts General Hospital, Harvard Medical School, Boston) A Alexandra Mossali R Rachel Rosovsky V Victoria Turbini V Valerie Wood H Hanny Al-Samkari (Department of Medicine, Massachusetts General Hospital, Boston) S Steven Pipe (4University of Michigan, Departments of Pediatrics and Pathology, Ann Arbor, United States) A Angela Weyand (4Department of Pediatrics, University of Michigan Medical School, Ann Arbor, United States) J Jordan Shavit (1University of Michigan, Department of Pediatrics, Ann Arbor, United States) A Amit Soni D Diane Nugent (13Children's Hospital of Orange County, Orange, United States) N Nina Hwang (2Center for Inherited Blood Disorders, Orange, United States) V Vanessa Salinas G Guy Young (Children’s Hospital Los Angeles) T Tom Hofstra J Jennifer Donkin J Jacquelyn Keegan Z Zaheer Sibgha W Wendy Leung J Julie Jaffray (1Department of Pediatrics, University of California, San Diego, San Diego, CA)

Abstract

Abstract Fitusiran, a subcutaneous investigational small interfering RNA therapeutic, lowers antithrombin (AT) to increase thrombin generation and rebalance hemostasis in people with hemophilia. This phase 3 open-label extension study (ATLAS-OLE) evaluated safety and efficacy of an AT-based dose regimen (AT-DR) in males aged ≥12 years with severe hemophilia A/B, with/without inhibitors. The original dose regimen (ODR) of 80 mg monthly was optimized to AT-DR targeting AT activity levels 15% to 35% to mitigate thrombotic risk (starting dose of 50 mg once every 2 months, individually adjusted to 20 mg once every 2 months, or 20/50/80 mg monthly as needed). Primary and secondary end points were safety and efficacy, respectively. Integrated safety analyses assessed safety of AT-DR and ODR across all fitusiran studies and integrated efficacy analyses compared efficacy of AT-DR in ATLAS-OLE with phase 3 parent study control groups. At interim data cutoff, 213 participants were enrolled on AT-DR (78% on regimens of once every 2 months). Integrated safety analyses of participants receiving AT-DR (n = 286) demonstrated that AT-DR was well tolerated. In ATLAS-OLE, median observed annualized bleeding rate (ABR) with AT-DR was 3.7 (interquartile range, 0.0-7.5). Integrated efficacy analyses demonstrated superiority of AT-DR over on-demand clotting factor concentrates (CFCs; 71% mean ABR reduction; P< .0001), and on-demand bypassing agents (BPAs; 73% mean ABR reduction; P = .0006); improvement over BPA prophylaxis (70% mean ABR reduction); and ABR comparable with that observed with CFC prophylaxis. Fitusiran AT-DR was well tolerated and maintained bleed protection with as few as 6 injections per year. This trial was registered at www.ClinicalTrials.gov as #NCT03754790.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 25
Published June 19, 2025
Pages 2966-2977
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (319)

S

Steven W. Pipe

Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor

L

Laurel A. Menapace

17Sanofi, Cambridge, MA

C

Chuanwu Zhang

9Sanofi, Waltham, United States

Y

Yuqian Shen

4Sanofi, Morristown, United States

M

Marja Puurunen

17Sanofi, Cambridge, MA

M

Marek Demissie

2Sanofi, Cambridge, United States

T

Teh-Liane Khoo

8Royal Prince Alfred Hospital, Sydney, Australia

S

Scott Dunkley

S

Stephanie P'ng

4The Haemophilia and Haemostasis Centre, Fiona Stanley Hospital, Murdoch, Australia

D

Dominic Pepperell

S

Stephanie Lam

E

Elizabeth Steinepreis

J

Jacques Malherbe

3Farmovs, Bloemfontein, South Africa

T

Tulene Kendrick

M

Marcus Lombard

H

Huyen Tran

8Department of Clinical Haematology, Alfred Hospital, Melbourne, VIC, Australia

J

Jessie Zhao

H

Hannah Stevens

C

Caroline Dix

J

James McFadyen

J

Jean St-Louis

J

Jeannine Kassis

G

Georges Rivard

J

Jing Sun

X

Xiaofang Li

Key Laboratory of Cluster Science of Ministry of Education, Beijing Key Laboratory of Photoelectronic/Electrophotonic Conversion Materials, School of Chemistry and Chemical Engineering, Beijing Institute of Technology, 5 South Zhongguancun Street, Haidian District, Beijing 100081, China

H

Huiping Li

J

Jieyu Ye

1Department of Hematology, Nanfang Hospital, Southern medical University, Guangzhou, China

X

Xuan Zhou

State Key Laboratory of Agricultural and Forestry Biosecurity, Key Laboratory of Ministry of Education for Genetics, Breeding and Multiple Utilization of Crops, Plant Immunity Center, Fujian Agriculture and Forestry University

D

Dong Du

X

Xuefeng Wang

Beijing National Laboratory for Condensed Matter Physics

J

Jing Dai

Q

Quilan Ding

W

Wenman Wu

Y

Yiyi Lu

R

Runhui Wu

G

Guoqing Liu

School of Physics and Astronomy, Shanghai Jiao Tong University

W

Wanru Yao

Y

Yan Wang

Y

Yunyun Wei

W

Weiqun Xu

20Children's Hospital, Zhejiang University School of Medicine, Hangzhou, China., Hangzhou, China

Y

Yu Du

Key Laboratory of Material Simulation Methods and Software of Ministry of Education, College of Physics

L

Liyao Xu

D

Di Wang

C

Chan Liao

H

Hongwei Zhang

Key Laboratory of Development and Application of Rural Renewable Energy

Z

ziqiang Yu

3National Clinical Research Center for Hematologic Disease, The First Affiliated Hospital of Soochow University, Suzhou, China

H

Hong Tian

H

Haibo Wang

J

Jie Yin

School of Psychology, Beijing Sport University

T

Tienan Zhu

2Peking Union Medical College Hospital, Beijing, China

H

Huacong Cai

1Chinese Academy of Medical Sciences & Peking Union Medical College, Peking, China

J

Juan Xiao

S

Shujie Wang

School of Chemical Engineering and Technology, Key Laboratory for Green Chemical Technology of Ministry of Education, Tianjin University

S

Shufen Liu

K

Kaini Shen

Y

Yun Chen

Y

Yali Chang

T

Tan Sang

1Shandong Cancer Hospital and Institute, Shandong First Medical University and Shandong Academy of Medical Sciences, Department of Lymphohematology Ward 2, Jinan, China

J

Jun Zhao

Department of Thoracic Oncology Beijing Cancer Hospital Beijing China

Y

Yawei Zhou

L

Liya Zhai

X

Xiadong Qiu

Y

Yulei He

H

Hui Li

Y

Yi Xia

M

Min Zhou

F

Feng Gao

S

Shipin Feng

X

Xiaoli Luo

D

Dan Li

Z

Zhongguang Cui

C

Chunzhu Zhao

H

Hong Xu

Institute of Nuclear and New Energy Technology

L

Lingjie Sun

State Key Laboratory of Advanced Materials for Intelligent Sensing, Key Laboratory of Organic Integrated Circuit, Ministry of Education & Tianjin Key Laboratory of Molecular Optoelectronic Sciences, Department of Chemistry, School of Science & Institute of Molecular Aggregation Science, Tianjin University

X

Xue Shi

X

Xiaojing Zeng

L

Lu Qi

Shandong Provincial Key Laboratory for Science of Material Creation and Energy Conversion, Science Center for Material Creation and Energy Conversion, Institute of Frontier Chemistry, School of Chemistry and Chemical Engineering

S

Shuyun Cao

Z

ZePing Zhou

1The Second Affiliated Hospital of Kunming Medical University, Department of Hematology, kunming, China

F

Fuhou Zhang

L

Lingmei Yin

H

Hongli Mu

H

Hui Bi

Shanghai Institute of Ceramics, Chinese Academy of Sciences

Z

Zhengchao Li

Y

Yaping Miu

Y

Yaming Xi

M

Mingfeng Jia

L

Long Zhao

Key Laboratory of Evolution and Marine Biodiversity (Ministry of Education) and Institute of Evolution and Marine Biodiversity, Ocean University of China, Qingdao, China.

X

Xielan Zhao

13Xiangya Hospital Central South University, Changsha, China

Y

Yaochun Li

J

Jie Peng

Institute of Intelligent Innovation, Henan Academy of Sciences

C

Chia-Yau Chang

2Taipei Medical University, Division of Pediatric Hematology/Oncology, Department of Pediatrics, Taipei, Taiwan

Y

Yen-Lin Liu

1Division of Pediatric Hematology/Oncology, Department of Pediatrics, Taipei Medical University Hospital, Taiwan, Taipei, Taiwan

M

Ming-Ching Shen

5Department of Internal Medicine, Changhua Christian Hospital, Changhua, Taiwan

C

Cheng-Wei Huang

S

Shih-Chung Wang

7Changhua Christian Hospital, Changhua, Taiwan

H

Hsuan-Yu Lin

C

Ching-Yeh Lin

S

Sheng-Chieh Chou

9Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan

M

Ming-Kai Chuang

S

Szu-Chun Hsu

J

Jiaan-Der Wang

6Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan

M

Ming-Yang Shih

L

Liang-Tsai Hsiao

G

Giun-Yi Hung

H

Hsiu-ju Yen

11Taipei Veterans General Hospital and National Yang-Ming University, Taipei, Taiwan

P

Po-Shen Ko

1Taipei Veterans General Hospital, Division of Hematology and Oncology, Department of Medicine, Taipei, Taiwan

H

Hung Chang

20Division of Hematology-Oncology, Department of Internal Medicine, Chang Gung Memorial Hospital, Taipei, Taiwan

S

Shih-Hsaing Chen

T

Te-Fu Weng

Y

Yu-Hua Chao

S

Sherig-Wei Zeng

P

Peter Kampmann

5Rigshospitalet, Department of Hematology, Copenhagen, Denmark

E

Eva Funding

Y

Yesim Dargaud

1Hospices Civils de Lyon, Service d'Hématologie et d'Hémostase Clinique, Lyon, France

C

Claude Negrier

S

Stéphanie Desag

L

Laurent Frenzel

G

Guillemette Fouquet

C

Cécile Bally

R

Robert Klamroth

14Vivantes Clinic Friedrichshain, Internal Medicine, Berlin, Germany

A

Ana-Marija Orlovic

C

Christian Pfrepper

4Division of Hemostaseology, Department of Hematology, Cellular Therapy, Hemostaseology and Infectiology, University of Leipzig Medical Centre, Leipzig, Germany

M

Michael Metze

R

Rima Chakaroun

M

Maria Weise

L

László Nemes

A

Agnes Ronatas

C

Cecil Ross

1St John's Medical College Hospital, Department of Clinical Hematology, Bengaluru, India

S

Seetharam Anandram

1St John's Medical College Hospital, Department of Clinical Hematology, Bengaluru, India

V

Vandana Bharadwaj

S

Sonali Salvi

S

Sunil Lohande

A

Alok Srivastava

Hematology Research Unit, St. John’s Research Institute, St. John’s National Academy of Health Sciences, Bengaluru, India

A

Aby Abraham

3Christian Medical College, Vellore, India

F

Fouzia Aboobacker

S

Shailendra Verma

G

Gaurav Datta

S

Savita Rangarajan

6University Hospital Southampton NHS Foundation Trust, South Hampton, United Kingdom

S

Shweta Bansal

S

Shashikant Apte

K

Kannan Subramanian

35Sahyadri Speciality Hospital Deccan Gymkhana, Pune, India

B

Beatrice Nolan

13Children's Health Ireland at Crumlin, Department of Paediatric Haematology, Dublin, Ireland

S

Saad Ahmed

N

Niamh O’Connell

J

James O’Donnell

K

Kevin Ryan

3National Coagulation Centre, St James’s Hospital, Dublin, Ireland

M

Michelle Lavin

National Coagulation Centre, St. James's Hospital, Dublin, Ireland

G

Gili Kenet

1The Amalia Biron Research Institute of Thrombosis & Hemostasis, Faculty of Medical & Health Sciences, Tel Aviv University, Tel Aviv, Israel

O

Omri Cohen

M

Mudi Misgav

A

Assaf Barg

S

Sarina Levy Mendelovich

F

Flora Peyvandi

R

Roberta Gualtierotti

E

Ezio Zanon

L

Luca Spiezia

C

Chiara Simion

T

Tadashi Matsushita

Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan

S

Shuichi Okamoto

N

Nobuaki Suzuki

T

Takeshi Kanematsu

N

Naruko Suzuki

Y

Yuka Kawaguchi

4Aichi Cancer Center, Department of Hematology and Cell Therapy, Nagoya, Japan

S

Satoshi Higasa

14Hyogo College of Medicine, Department of Hematology, Nishinomiya, Japan

T

Tazuko Tokugawa

A

Akihiro Sawada

R

Rie Shirayama

M

Masaru Kawamura

H

Hiroshi Asai

K

Koichi Oshida

T

Tetsuji Sato

T

Takuma Ito

Department of Chemistry, Graduate School of Science

K

Katsuyoshi Koh

23Japan Children’s Cancer Group ALL Committee, Nagoya, Japan

Y

Yoshitaka Mizushima

T

Takahiro Ishikawa

H

Hirohito Kubota

R

Ryota Kaneko

T

Tomoya Irikura

M

Mamoru Honda

K

Katsuaki Taira

Y

Yuki Arakawa

Department of Hematology/Oncology, Saitama Children’s Medical Center

M

Makiko Mori

6Saitama Children's Medical Center, Saitama, Japan

K

Kohei Fukoka

K

Koichi Oshima

Y

Yuichi Mitani

Y

Yoshiaki Ogawa

24Tokai University of School of Medicine, Kanagawa, Japan

K

Kiyoshi Ando

R

Rikio Suzuki

R

Ryujiro Hara

Y

Yasuyuki Aoyama

M

Masako Toyosaki

D

Daisuke Ogiya

9Tokai University School of Medicine, Department of Hematology and Oncology, Kanagawa, Japan

S

Shinichiro Machida

11Tokai University School of Medicine, Department of Hematology and Oncology, Kanagawa, Japan

H

Hiroshi Kawada

Y

Yukari Shirasugi

M

Makoto Onizuka

K

Ken Omachi

K

Kagehiro Amano

T

Tomoko Yamaguchi

E

Ei Kinai

T

Takeshi Hagiwara

M

Mihoko Yotsumoto

R

Ryoko Sekiya

M

Masato Bingo

Y

Yushi Chikasawa

A

Akito Ichiki

Y

Yoshiko Kamikubo

R

Ryui Miyashita

T

Takashi Muramatsu

H

Ho-jin Shin

Pusan National University Hospital, Busan, Korea, Republic of

D

Doyoung Kim

C

Chur-Woo You

2Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, Republic of Korea

J

Ju Young Kim

S

Seung Min Hahn

C

Chuhl Joo Lyu

Department of Pediatrics, Yonsei University Severance Hospital, Seoul, South Korea

J

Jung Woo Han

1Yonsei University College of Medicine, Department of Pediatrics, Seoul, Korea

Y

Young Shil Park

J

Ja Hyang Cho

A

Azizan Shariq

S

Soo Min Lim

7Sultanah Aminah Hospital, Johor Bahru, Malaysia

L

Lily Wong

H

Heng Chang Choo

G

Gilbert Wilfred

M

Mohd Tarmizi Ahmad Zakhi

Y

Yin Man Lim

K

Khaiteri Raghunathan

J

Jameela Sathar

S

Syazarin Fidhora Nasution

Y

Yee Yee Yap

V

Veena Selvaratnam

D

Daniel Malan

Z

Zelda Erika Punt

A

Ali Bulent Antmen

I

Ilgen Sasmaz

U

Utku Aygunes

29Konya City Hospital, Hematology, Konya, Türkiye

K

Kaan Kavakli

3Ege University Faculty of Medicine Children's Hospital, Department of Pediatric Hematology and Oncology, Izmir, Türkiye

N

Nihal Karadas

D

Deniz Karapinar

A

Alphan Kupesiz

2Akdeniz University School of Medicine, Pediatric Hematology and Oncology, Antalya, Türkiye

M

Mine Erkan

K

Kupesiz Funda Tayfun

E

Elif Guler

B

Bulent Zulfikar

2Department of Pediatric Hematology-Oncology, Istanbul University, Oncology Institute, Istanbul, Türkiye

B

Basak Koc

V

Vahap Okan

S

Sahin Handan Haydaroglu

C

Canan Albayrak

I

Ibrahim Kartal

E

Ebru Yilmaz

M

Musa Karakükcü

24Erciyes University, Faculty of Medicine, Kayseri, Turkey, Kayseri, Türkiye

E

Ekrem Unal

A

Alper Ozcan

8Erciyes University School of Medicine, Pediatric Hematology and Oncology, Kayseri, Türkiye

F

Fahri Sahin

B

Betul Tuzun

G

Güray Saydam

9Ege University Medical Faculty Hospital, Department of Internal Diseases, Division of Hematology, Izmir, Türkiye

I

Ievgenii Averianov

O

Oksana Kovtunenko

O

Oleksandra Stasyshyn

M

Maksym Semerak

O

Orest Tushnytskyy

K

Kateryna Vilchevska

I

Iryna Bakhchyvandzhy

N

Nataliya Romanyuk

S

Stanislav Nikulin

A

Alina Kobets

J

Jayanthi Alamelu MRCPCH

S

Samah Babiker

C

Catherine Bagot

18Glasgow Royal Infirmary, Glasgow, United Kingdom

J

James Tyan Rodgers

L

Lisa Boggio

4Rush Hemophilia and Thrombophilia Center, Chicago, United States

M

Mindy Simpson

1Rush University Medical Center, Rush Hemophilia and Thrombophilia Center, Chicago, United States

E

Erin Cockrell

11St. Joseph's Hospitals Center for Bleeding and Clotting Disorders, Tampa, United States

C

Candance DeBerry

D

Dana Obzut

J

John Fargo

1Ann & Robert H. Lurie Children's Hospital, Chicago, United States

J

Jeffrey Hord

S

Stephanie Savelli

J

Joseph Lasky

J

Jennifer Buitrago

W

Waseem Alhushki

C

Cynthia Gauger

D

Darren Klawinski

E

Eric Sandler

S

Scott Bradfield

P

Paul Pitel

M

Michael Joyce

20Department of Pediatrics, Nemours Children’s Health, Jacksonville, FL

H

Howard Katzenstein

M

Manisha Bansal

A

Allison Bechtel

C

Christine Knoll

E

Esteban Gomez

S

Shanna White

1Phoenix Children's Hospital, Phoenix, United States

S

Sanjay Shah

1Phoenix Children's Hospital, Phoenix, United States

J

Joanna Gendreau

3Johnson & Johnson, Global Medical Organization, Horsham, United States

L

Lori Wagner

M

Maa-Ohui Quarmyne

1Phoenix Children's Hospital, Phoenix, United States

L

Larissa Bornikova

C

Colleen Cleary CNP

P

Pamela G. Hodges

Division of Hematology–Oncology, Massachusetts General Hospital, Harvard Medical School, Boston

A

Alexandra Mossali

R

Rachel Rosovsky

V

Victoria Turbini

V

Valerie Wood

H

Hanny Al-Samkari

Department of Medicine, Massachusetts General Hospital, Boston

S

Steven Pipe

4University of Michigan, Departments of Pediatrics and Pathology, Ann Arbor, United States

A

Angela Weyand

4Department of Pediatrics, University of Michigan Medical School, Ann Arbor, United States

J

Jordan Shavit

1University of Michigan, Department of Pediatrics, Ann Arbor, United States

A

Amit Soni

D

Diane Nugent

13Children's Hospital of Orange County, Orange, United States

N

Nina Hwang

2Center for Inherited Blood Disorders, Orange, United States

V

Vanessa Salinas

G

Guy Young

Children’s Hospital Los Angeles

T

Tom Hofstra

J

Jennifer Donkin

J

Jacquelyn Keegan

Z

Zaheer Sibgha

W

Wendy Leung

J

Julie Jaffray

1Department of Pediatrics, University of California, San Diego, San Diego, CA