Lauren E. Merz, MD, MSc^1,2, Yating Wang, MS^3*, Elad Sharon, MD, MPH^4*, Thomas P Walsh^5*, Gregory Abel, MD, MPH⁵ and Andrew Hantel, MD⁶

¹Division of Hematology/Oncology, Mass General Brigham, Boston, MA
²Department of Medical Oncology, Dana-Farber Cancer Institute, Brookline, MA
³Department of Data Science, Dana-Farber Cancer Institute, Boston, MA
⁴Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
⁵Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
⁶HMS Center for Bioethics, Dana-Farber Cancer Institute, Boston, MA

Elizabeth A. Griffiths, MD¹, Andrew Messali^2*, Mohammed Mahdi^2*, Maral DerSarkissian^3*, Rose Chang^3*, Brendan Rabideau^3* and David Dingli, MD, PhD⁴

¹Roswell Park Comprehensive Cancer Center, Buffalo, NY
²Alexion, AstraZeneca Rare Disease, Boston, MA
³Analysis Group, Inc., Boston, MA
⁴Mayo Clinic, Rochester, MN

Juliana Schwaab, MD^1*, Paul Dolin^2*, Priya Jain², Jennifer Rowell^3*, Chris Edmonds^4*, Josepine Persson^5*, Daniel Mascia^6*, Tia Pennant^6*, Fritha Hennessy^6* and Stephanie Yanjing Chen^7*

¹Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany
²BioPharmaceuticals Medical, AstraZeneca, Cambridge, United Kingdom
³Market Access and Pricing, AstraZeneca, Cambridge, United Kingdom
⁴Market Access and Pricing, AstraZeneca, Gaithersburg, MD
⁵Market Access and Pricing, AstraZeneca, Gothenburg, Sweden
⁶Adelphi Real World, Bollington, United Kingdom
⁷BioPharmaceuticals Medical, AstraZeneca, Gaithersburg, MD

Emma P Deloughery, MD¹ and Thomas G Deloughery, MD²

¹Oregon Health & Science University, Portland, OR
²Division of Hematology and Medical Oncology, Oregon Health and Science Univ., Portland, OR

Víctor Jiménez-Yuste, MD, PhD^1*, Cléa Percier, PharmD^2*, Naveen Shridhar, MBBS, PhD^3*, Neil Reynolds^4*, Olivera Rajkovic-Hooley^4*, Thom Dewar^4* and Giancarlo Castaman^5*

¹Hospital Universitario La Paz, Universidad Autónoma de Madrid, Madrid, Spain
²Novo Nordisk Health Care AG, Zürich, Switzerland
³Novo Nordisk Service Centre India Private Limited, Bangalore, India
⁴Adelphi Real World, Bollington, United Kingdom
⁵Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy

Michael Recht, MD, PhD, MBA^1,2*, Hanna Salehi, MSc^3*, Chris Blazos, BSc^4*, Sophie Lai, BSc^4*, Ella Morton, BSc^4*, Lucy Lee, PharmD, MPH^5*, Juliana M.L. Biondo, MD⁵ and Janet S Lee^5*

¹National Bleeding Disorders Foundation, New York, NY
²Yale School of Medicine, New Haven, CT
³Adelphi Real World, Bollington, United Kingdom
⁴Adelphi Real World, Macclesfield, United Kingdom
⁵Genentech, Inc., South San Francisco, CA

Claire Booth, MBBS, PhD^1,2*, Patrick Gilbert^3*, Luuk Gras^4*, Hawazen Alsaedi^5*, Amir Ali Hamidieh^6*, Mor Seny Gueye^7*, Mary Slatter, MD^8*, Ashrafsadat Mousavi^9*, Amos Toren^10*, Musa Karakukcu, MD^11*, Abdelghani Tbakhi^12*, Polina Stepensky^13*, Gulyuz Ozturk^14*, Peter Bader¹⁵, Henrik Sengeloev^16*, Tayfun Güngör^17*, R.F. Wynn^18*, Franco Locatelli, MD¹⁹, Maria Chitty-Lopez, MD^20*, Joana E Matos^20*, Stuart Turner^20*, Gayatri R Rao, MD, JD^20*, Jonathan D Schwartz, MD²⁰, Michael H Albert^21* and Bénédicte Neven^22*

¹University College London Great Ormond Street Institute of Child Health, London, United Kingdom
²Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital NHS Trust, London, United Kingdom
³EBMT Leiden Study Unit, Leiden, Netherlands
⁴EBMT, Leiden, Netherlands
⁵King Faisal Specialist Hospital and Research Centre, Riyadh, SAU
⁶Pediatric Cell and Gene Therapy Research Centre, Gene, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Tehran, Iran (Islamic Republic of)
⁷Hospital Necker-Enfants Malades, Paris, France
⁸Paediatric Stem Cell Transplant Unit, Great North Children’s Hospital, Newcastle Upon Tyne, United Kingdom
⁹Shariati Hospital, Teheran, Iran (Islamic Republic of)
¹⁰Sheba Medical Center, Tel-Hashomer, Israel
¹¹Pediatri Hematoloji Onkoloji Başkanı, Erciyes Üniversitesi, Kayseri, Turkey
¹²King Hussein Cancer Foundation and Center, Amman, Jordan
¹³Hadassah University Hospital, Jerusalem, Israel
¹⁴Acibadem Saglik Hizmetleri ve Ticaret Anonim Sirketi, Istanbul, TUR
¹⁵Universitätsklinikum Frankfurt Goethe-Universität, Frankfurt, Germany
¹⁶Rigshospitalet, Copenhagen, Denmark
¹⁷Hematology/Oncology/Immunology, Gene Therapy, and Stem Cell Transplantation, University Children's Hospital Zurich-Eleonore Foundation & Children's Research Center, Zürich, Switzerland
¹⁸Royal Manchester Children's Hospital, Manchester, United Kingdom
¹⁹IRCCS Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome, Italy
²⁰Rocket Pharmaceuticals, Inc., Cranbury, NJ
²¹Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, Ludwig-Maximilians-Universität München, Munich, Germany
²²Unité d'Immuno-Hématologie et Rhumatologie Pédiatrique, Assistance Publique-Hôpitaux de Paris, Paris, France

Elizabeth Bowhay-Carnes, MD^1*, Yuliya Kreychman^2*, Lucas Buxman^3* and Leslie Todd^2*

¹Division of Hematology-Oncology, Mays Cancer Center at UT Health MD Anderson, San Antonio, TX
²Rigel Pharmaceuticals, Inc., South San Francisco, CA
³Claritas Rx, Inc., South San Francisco

Maya Birhiray, MS^*, Sam Ranger, M.S.^* and Ruemu Birhiray

Indy Hematology Education Inc., Carmel, IN

Valeria Merla, MPH^1*, Sheena Thakkar, MPH^1*, Jasmine Healy, PhD^2*, Ella Morton, BSc^3*, Kieran Wynne-Cattanach, MSc^3*, Nathan Ball, BSc^3*, Sophie Lai, BSc^3*, Chris Blazos, BSc^3* and Hanna Salehi, MSc^3*

¹Pfizer Inc, New York, NY
²Pfizer Canada ULC, Kirkland, QC, Canada
³Adelphi Real World, Bollington, United Kingdom

Meera Karunakaran^1,2*, Fatema Hossain^1,2*, Erin Jamula, MSc^1,2*, Ishac Nazy, PhD^1,2,3 and Donald Arnold, MD^1,2

¹Michael G. DeGroote Centre for Transfusion Research, McMaster University, Hamilton, ON, Canada
²Department of Medicine, McMaster University, Hamilton, ON, Canada
³Department of Biochemistry and Biomedical Sciences, McMaster University, Hamilton, ON, Canada

Cho Han Chiang^1*, Shuwen Lin, MD^2*, Yu-Cheng Chang, MD^3*, Junmin Song, MD⁴, Kuan-Yu Chi, MD^4*, Nutchapon Xanthavanij, MD^5*, Yu Chang, MD^6*, Cho-Hung Chiang, MD^7* and Xiaocao/Haze Xu, MBBS^8*

¹Department of Medicine, Mount Auburn Hospital, Cambridge, MA
²Montefiore Medical Center, Montefiore Einstein Cancer Center, Bronx, NY
³Department of Medicine, Danbury Hospital, Danbury, CT
⁴Department of Medicine, Jacobi Medical Center, Albert Einstein College of Medicine, Bronx, NY
⁵Mount Auburn Hospital, Cambridge, MA
⁶Section of Neurosurgery, Department of Surgery, National Cheng Kung University Hospital, College of Medicine, National Cheng Kung University, Tainan, Taiwan
⁷National Taiwan University Hospital, Taipei, Taiwan
⁸University of Vermont Medical Center, Burlington, VT

Jens Panse, MD¹, Juan Carlos Vallejo Llamas, MD^2*, Koo Wilson, MSc^3*, Zalmai Hakimi, PhD^3*, Barbara Czech, PhD^3*, Brianne Kerr, BSc^4*, Sam Williamson, BSc^4*, Jade Garratt-Wheeldon, BSc^4*, Yasmin Taylor, MSc, BSc^4*, Niall Hatchell, MSc, BSc^4* and Christopher Patriquin, MD, MSc, FRCPC⁵

¹Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany & Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen, Germany
²University Clinical Hospital of Santiago, Santiago De Compostela, Spain
³Swedish Orphan Biovitrum AB, Stockholm, Sweden
⁴Adelphi Real World, Bollington, United Kingdom
⁵Division of Medical Oncology & Hematology, University Health Network, Toronto, ON, Canada

Kristi Upadhyaya^1*, Xiaoying Chen^2*, Alok A. Khorana, MD¹ and Dana Elizabeth Angelini, MD¹

¹Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
²Cleveland Clinic, Quantitative Health Sciences, Lerner Research Institute, Cleveland, OH

Sumera Ackbarali^1*, Tariqa Ackbarali, MSc^2*, Katie Kowalski, MPH^3*, Shirley D'Sa, FRCP FRCPath MD(Res)^4*, Carlos M de Castro, MD⁵, Satheesh Chonat, MD⁶ and Catherine M. Broome, MD⁷

¹PlatformQ Health Education, LLC, Lake Worth, FL
²PlatformQ Health, Lake Worth, FL
³National Organization for Rare Disorders, Danbury, CT
⁴University College London Hospitals NHS Foundation Trust, London, United Kingdom
⁵Duke University, Durham, NC
⁶Cincinnati Children's Hospital, Atlanta, GA
⁷Georgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, DC

Alexis Brantley, MD^1*, Nali Gillespie, M.D.^2*, Morgan Viviano, MD^1*, Leah Smith, DO^1*, Zhide Feng, PhD^3*, Maria C. Velez-Yanguas, MD⁴ and Zachary LeBlanc, MD^5*

¹Louisiana State University Health Sciences Center School of Medicine, New Orleans
²Louisiana State University Health Sciences Center School of Medicine, New Orleans, LA
³Department of Biostatistics, Louisiana State University Health Sciences Center, New Orleans
⁴Division of Pedatric Hematology and Oncology, Louisiana State University Health Sciences Center School of Medicine, New Orleans, LA
⁵Division of Pediatric Hematology and Oncology, Louisiana State University Health Sciences Center School of Medicine, New Orleans, LA

Alexander J Xiang^1*, Sarah Sadafi, BHSc^2*, Rachael Principato^3*, Asim Shaikh, BHSc^2*, Mehrad Nowrouzi^2*, Allen Li, MD⁴, Ali Eshaghpour, MD⁵ and Mark A. Crowther, MD, MSc, FRSC, FRCPC, LLM⁵

¹Department of Medicine, McMaster University, Kitchener, ON, Canada
²McMaster University, Hamilton, Canada
³University of Guelph, Guelph, Canada
⁴Department of Vascular Surgery, University of Toronto, Toronto, ON, Canada
⁵Department of Medicine, McMaster University, Hamilton, Canada

Priya Jain¹, Jennifer Rowell^2*, Chris Edmonds^3*, Michael Stokes^4*, Ning Ding^5* and Stephanie Yanjing Chen^6*

¹BioPharmaceuticals Medical, AstraZeneca, Cambridge, United Kingdom
²Market Access and Pricing, AstraZeneca, Cambridge, United Kingdom
³Market Access and Pricing, AstraZeneca, Gaithersburg, MD
⁴Data Analytics, Evidera, Saint-Laurent, QC, Canada
⁵Data Analytics, Evidera, Bethesda, MD
⁶BioPharmaceuticals Medical, AstraZeneca, Gaithersburg, MD

Amber Afzal, MD, MSc¹, Brian F. Gage, MD, MSc^2*, Luo Suhong^3* and Kristen M. Sanfilippo, MD^4,5

¹Washington University School of Medicine St Louis, Saint Louis, MO
²Washington University School of Medicine in St. Louis, St Louis, MO
³Veterans Health Administration, Saint Louis, MO
⁴Washington University School of Medicine St Louis, Chesterfield, MO
⁵St Louis Veterans Affairs Medical Center, Saint Louis, MO