Nicholas Williams, PhD^1*, Joe Lee, MBBS^1,2*, Luiza Moore, PhD^1,3*, Joanna E Baxter, PhD^4*, James Hewinson, PhD^1*, Kevin J Dawson, PhD^1*, Andrew Menzies, MSc^1*, Anna L Godfrey, PhD^3*, Anthony R Green, PhD FRCPath FMedSci^2,4, Peter J Campbell, PhD^1* and Jyoti Nangalia, MBBChir^1,2,3

¹Wellcome Sanger Institute, Hinxton, United Kingdom
²University of Cambridge, Wellcome-MRC Cambridge Stem Cell Institute, Cambridge, United Kingdom
³Cambridge Universities NHS Trust, Cambridge, United Kingdom
⁴University of Cambridge, Department of Haematology, Cambridge, United Kingdom

Charlotte A Bradbury, MD, MSc, FRCP, FRCPath, PhD^1,2*, Rosemary Greenwood^3*, Julie Pell^4*, Katie Breheny^5*, Rebecca Kandiyali^5*, Jenny Ingram^5*, Ian Thomas^6*, Catherine Bagot, MD, FRCPath, MBBS^7*, Quentin Hill^8*, HaemSTAR Collaborators^9* and Nichola Cooper^10*

¹Faculty of Translational Health Sciences, University of Bristol, Bristol, United Kingdom
²Bristol Haematology and Oncology Centre, University Hospitals Bristol and Weston NHS Trust, Bristol, United Kingdom
³Research Design Service, University of Bristol, Bristol, United Kingdom
⁴Cardiff University, Centre for Trials Research, Cardiff, United Kingdom
⁵University Hospitals Bristol and Weston NHS Trust, Bristol, United Kingdom
⁶Centre for Trials Research, Cardiff University, Cardiff, United Kingdom
⁷Glasgow Royal Infirmary, Glasgow, United Kingdom
⁸Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom
⁹http://haemstar.org/flight/, UK, United Kingdom
¹⁰Dept Immunology and Inflammation, Imperial College, London, United Kingdom

Lindsey Montefiori, PhD¹, Sonja Seliger, MS^2*, Zhaohui Gu, PhD³, Xiaotu Ma, PhD^4*, Beisi Xu, PhD^5*, Xiaolong Chen, PhD^4*, Kirsten Dickerson, BS¹, Tamara Westover, BS^1*, Anna Stengel, PhD², Ilaria Iacobucci, PhD¹, Shunsuke Kimura, MD, PhD^1*, Chunxu Qu, PhD^1*, Jing Ma, PhD^1*, Marc Valentine, BA^6*, Wolfgang Kern, MD², Torsten Haferlach, MD², Gang Wu, PhD^5*, Jeffery M. Klco, MD, PhD¹, Claudia Haferlach, MD² and Charles G Mullighan, MBBS, MD¹

¹Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN
²MLL Munich Leukemia Laboratory, Munich, Germany
³Department of Computational and Quantitative Medicine, City of Hope National Medical Center, Monrovia, CA
⁴Department of Computational Biology, St. Jude Children's Research Hospital, Memphis, TN
⁵Center for Applied Bioinformatics, St. Jude Children's Research Hospital, Memphis, TN
⁶Cytogenetics Core Facility, St. Jude Children's Research Hospital, Memphis, TN

Andreas Hochhaus, MD¹, Carla Boquimpani, MD^2*, Delphine Rea, MD, PhD³, Yosuke Minami, MD, PhD⁴, Elza Lomaia, MD, PhD^5*, Sergey Voloshin, MD, PhD^6*, Anna G. Turkina, Prof., MD⁷, Dong-Wook Kim, MD⁸, Jane Apperley, FRCP, FRCPath, MB ⁹, Jorge E. Cortes, MD¹⁰, Andre Abdo, MD^11*, Laura Maria Fogliatto, MD, PhD¹², Dennis Dong Hwan Kim, MD, PhD^13*, Philipp D le Coutre, MD¹⁴, Susanne Saussele, MD¹⁵, Mario Annunziata, MD^16*, Timothy P. Hughes, MD, MBBS, FRACP, FRCPA¹⁷, Naeem A. Chaudhri, MD¹⁸, Lynette C.Y. Chee, MBBS, PhD, FRACP, FRCPA¹⁹, Valentín García Gutiérrez, MD, PhD²⁰, Koji Sasaki, MD²¹, Paola Aimone, MD^22*, Alex Allepuz, MD, MPH^23*, Sarah Quenet^24*, Véronique Bédoucha^24* and Michael J. Mauro, MD²⁵

¹Klinik für Innere Medizin II, Jena, Germany
²HEMORIO, State Institute of Hematology Arthur de Siquiera Cavalcanti, Rio de Janeiro, Brazil
³Hopital Saint Louis, Paris, France
⁴National Cancer Center Hospital East, Kashiwa, Japan
⁵Institute of Hematology, Almazov National Medical Research Centre, St. Petersburg, Russian Federation
⁶Russian Research Institute of Hematology and Transfusiology, Saint Petersburg, Russian Federation
⁷National Research Center for Hematology, Moscow, Russian Federation
⁸Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea, Republic of (South)
⁹Centre for Haematology, Department of Medicine, Hammersmith Hospital, Imperial College, London, United Kingdom
¹⁰Georgia Cancer Center Augusta University, Augusta, GA
¹¹Instituto do Câncer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil
¹²Serviço Hematologia e Transplante de Medula Ossea, Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil
¹³Princess Margaret Cancer Centre, University of Toronto, Messner Allogeneic Blood and Marrow Transplantation Program, Toronto, ON, Canada
¹⁴Charité – Universitätsmedizin Berlin, Berlin, Berlin, Germany
¹⁵Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg, Germany
¹⁶Hematology Unit, Cardarelli Hospital, Naples, Italy
¹⁷Precision Medicine Theme, South Australian Health and Medical Research Institute, Adelaide, SA, Australia
¹⁸Oncology center, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
¹⁹Clinical Haematology and Bone Marrow Transplant Service, Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, Melbourne, VIC, Australia
²⁰Hematology and Hemotherapy Department, Hospital Universitario Ramón y Cajal, Madrid, Spain
²¹Department of Leukemia, MD Anderson Cancer Center, Houston, TX
²²Novartis Pharma AG, Basel, Switzerland
²³Novartis Pharma AG, Basil, NJ, Switzerland
²⁴Novartis Pharma AG, Basil, Switzerland
²⁵Memorial Sloan-Kettering Cancer Center, New York, NY

Diletta Fontana, PhD^1*, Mario Mauri, PhD^2*, Rossella Renso, MD^3*, Mattia Docci, MD^4*, Ilaria Crespiatico, MS^5*, Lisa M Røst, PhD^6*, Mi Jang, PhD^6*, Antonio Niro, MS^7*, Deborah D'Aliberti, PhD^5*, Luca Massimino, PhD^8*, Mayla Bertagna, MS^5*, Giovanni Zambrotta, MD^5*, Mario Bossi, MS^5*, Stefania Citterio, MS^9*, Barbara Crescenzi, PhD^10*, Francesca Fanelli, PhD^11*, Valeria Cassina, PhD^5*, Roberta Corti, PhD^5*, Domenico Salerno, PhD^5*, Luca Nardo, PhD^5*, Clizia Chinello, PhD^5*, Francesco Mantegazza, PhD^5*, Cristina Mecucci, MD, PhD¹², Fulvio Magni, PhD^5*, Guido Cavaletti, MD^5*, Per Bruheim, PhD^13*, Delphine Rea, MD, PhD¹⁴, Steen Larsen, PhD^15*, Rocco Piazza, MD, PhD^16* and Carlo Gambacorti-Passerini, MD¹⁷

¹Department of Medicine and Surgery, University of Milano - Bicocca, Monza, MB, Italy
²Department of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy
³Hematology and Transplant Department, ASST-Monza San Gerardo Hospital, Monza, Italy
⁴University of Milano - Bicocca, Monza, Italy
⁵Department of Medicine and Surgery, University of Milano - Bicocca, Monza, Italy
⁶Department of Biotechnology and Food Science, Norwegian University of Science and Technology, Trondheim, Norway
⁷Department of Medicine and Surgery, University of Milano-Bicocca, Monza, ITA
⁸Dept. of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy
⁹University of Milano Bicocca, Milano, Italy
¹⁰University of Perugia, Perugia, Italy
¹¹Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy
¹²University of Perugia, Perugia, ITA
¹³Norwegian University of Science and Technology, Trondheim, NOR
¹⁴Université de Paris, Hematology department, Saint-Louis hospital, APHP, Paris, France
¹⁵Department of Biomedical Sciences, University of Copenhagen, Copenhagen, Denmark
¹⁶Department of Medicine, University of Milano-Bicocca, Monza, Italy
¹⁷Dept of Medicine and Surgery, University of Milano Bicocca, Monza, MB, Italy

Steven W. Pipe, MD¹, Michael Recht, MD, PhD², Nigel S. Key, MD³, Frank W.G. Leebeek, MD, PhD⁴, Giancarlo Castaman, MD^5*, Susan U. Lattimore, R.N.^6*, Paul Van Der Valk^7,8,9*, Kathelijne Peerlinck, MD, PhD^10*, Michiel Coppens, MD^11*, Niamh O'Connell, MD, PhD^12*, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH¹³, Peter Kampmann, MD^14*, Karina Meijer, MD, PhD¹⁵, Annette von Drygalski, MD, Pharm D¹⁶, Guy Young, MD¹⁷, Cedric Hermans, MD, MRCP, PhD¹⁸, Jan Astermark, MD, PhD^19,20*, Robert Klamroth, MD, PhD^21*, Richard S. Lemons, MD^22*, Nathan Visweshwar, MD^23*, Shelley Crary, MD, MS²⁴, Rashid Kazmi, MBBS^25*, Emily Symington^26*, Miguel A. Escobar, M.D.^27*, Esteban Gomez, MD²⁸, Rebecca Kruse-Jarres, MD²⁹, Adam Kotowski, M.D.^30*, Doris Quon, MD, PhD³¹, Michael Wang, MD³², Allison P. Wheeler, MD³³, Eileen K Sawyer, PhD^34*, Stephanie Verweij, BSc^35*, Valerie Colletta, MSc^36*, Naghmana Bajma, M.D.^37*, Robert Gut, MD, PhD^36* and Wolfgang A. Miesbach, MD, PhD^38*

¹University of Michigan, Ann Arbor, MO
²Oregon Health & Science University, Portland, OR
³University of North Carolina, Chapel Hill, NC
⁴Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands
⁵Azienda Ospedaliera Universitaria Careggi, Florence, Italy
⁶Oregon Health & Science University, Portland
⁷Vrije Universiteit Medical Center, Amsterdam, NLD
⁸Universitair Medisch Centrum Utrecht, Utrecht, Netherlands
⁹Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
¹⁰Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium
¹¹Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands
¹²The National Coagulation Centre, St James Hospital, Dublin, Ireland
¹³Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom
¹⁴Rigshospitalet, Copenhagen, Denmark
¹⁵University Medical Center Groningen, Groningen, Netherlands
¹⁶University of California San Diego, La Jolla, CA
¹⁷University of Southern California Keck School of Medicine, Children's Hospital Los Angeles, Los Angeles, CA
¹⁸Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussels, Belgium
¹⁹Department of Translational Medicine-Clinical Coagulation, Lund University, Malmo, Sweden
²⁰Skane University Hospital, Malmo, Sweden
²¹Vivantes Klinikum im Friedrichshain, Berlin, Germany
²²University of Utah, Salt Lake City, UT
²³University of South Florida, Tampa, FL
²⁴University of Arkansas for Medical Sciences, Little Rock, AR
²⁵University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
²⁶Cambridge University - Addenbrooke's Hospital, Cambridge, United Kingdom
²⁷University of Texas Health Science Center at Houston, Houston, TX
²⁸Phoenix Children's Hospital, Phoenix, AZ
²⁹BloodWorks NorthWest, Seattle, WA
³⁰Hemophilia Center of Western New York, New York
³¹Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, CA
³²Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia and Thrombosis Center, Aurora, CO
³³Vanderbilt University Medical Center, Nashville, TN
³⁴uniQure Inc., Lexington, MA
³⁵uniQure, Lexington, MA
³⁶uniQure Inc, Lexington, MA
³⁷Uniqure Inc, Lexington, MA
³⁸University Hospital Frankfurt, Frankfurt, Germany