Camille Roussel, MD, PhD^1,2,3*, Sandra Manceau^2,4*, Zeynep Cacan^3*, Celine Gounou^5*, Laurent Sabbah^6*, Alain Brisson^5*, Lucie Dumas^2,7*, Delphine Borgel, PhD, PharmD^3,8* and Laure Joseph, MD^9*

¹Université Paris Cité and Université des Antilles, Inserm, BIGR, Paris, France
²Laboratoire d’Excellence GR-Ex, Paris, France
³General hematology department, Hôpital Necker, Assistance-Publique Hôpitaux de Paris, Paris, France
⁴Biotherapy Department, French National Sickle Cell Referral Center, Hôpital Necker, Assistance-Publique Hôpitaux de Paris, Paris, France
⁵University of Bordeaux, CNRS, Bordeaux INP, CBMN, UMR 5248, Pessac, France
⁶Unité de cardiologie adulte, Hôpital Necker, Assistance-Publique Hôpitaux de Paris, Paris, France
⁷Université Paris Cité and Université des Antilles, Inserm, BIGR, PARIS, France
⁸Université Paris-Saclay, HITh, UMR_S1176, INSERM, le Kremlin Bicêtre, France
⁹Biotherapy Department, French National Sickle Cell Referral Center, Hôpital Necker, Assistance-Publique Hôpitaux de Paris, Paris, DC, France

Kristine Anne Karkoska, MD, MS¹, Russell Sawyer, MD^2* and Hyacinth I Hyacinth, MD, PhD, MPH³

¹Division of Hematology/Oncology, Dept of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, OH
²Department of Neurology and Rehabilitation Medicine, University of Cincinnati Medical Center, Cincinnati, OH
³Department of Neurology and Rehabilitation Medicine, University of Cincinnati, Cincinnati, OH

Nirmish Shah, MD¹, Clark Brown, MD, PhD², Biree Andemariam, MD³, Modupe Idowu, MD⁴, Alexander Glaros, MD⁵, Jeffrey Glassberg, MD^6*, Robert M Cronin, MD, MS⁷, Barbara Moehring, PharmD^8*, Carolyn Hoppe, MD^8*, Sandy Dixon^8* and Deepa Manwani, MD⁹

¹Department of Medicine, Division of Hematology - Duke Sickle Cell Comprehensive Care Unit, Duke University Hospital, Durham, NC
²Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
³University of Connecticut Health, West Hartford, CT
⁴University of Texas McGovern Medical School, Houston, TX
⁵Children's Hospital of Michigan, Department of Pediatrics, Division of Pediatric Hematology-Oncology, Central Michigan University, Detroit, MI
⁶Icahn School of Medicine at Mount Sinai, New York City, NY
⁷Department of Internal Medicine, The Ohio State University, Hilliard, OH
⁸Global Blood Therapeutics, South San Francisco, CA
⁹Division of Pediatric Hematology/Oncology, Department of Pediatrics, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

Mycal Casey, DO, MPH¹, Brandon Gramazio, PhD^2,3*, Florence Urum, BS^4*, Niren Patel, MBBS^2*, Wenbo Zhi, PhD^5* and Abdullah Kutlar, MD⁶

¹Internal Medicine, Medical College of Georgia At Augusta University, Augusta, GA
²Center for Blood Disorders, Augusta University/Medical College of Georgia, Augusta, GA
³Augusta University, Augusta, GA
⁴Augusta University, Medical College of Georgia, Augusta, GA
⁵Center for Biotechnology and Genomic Medicine, Augusta University, Augusta, GA
⁶Department of Medicine, Sickle Cell Center, Augusta University, Augusta, GA

Abena Appiah-Kubi, MD, MPH¹, Seethal A Jacob, MD, MS², Matthew M. Heeney, MD³, Clark Brown, MD, PhD⁴, Susan E. Creary, MD, MSc⁵, Christine R Hollenkamp, APRN^6*, Emily Riehm Meier, MD, MSHS^4*, Omar Niss, MD⁷, Connie M. Piccone, MD⁸, Maa-Ohui Quarmyne, MBChB MS⁹, Charles T. Quinn, MD⁷, Allison Remiker, MD^10*, Kay L Saving, MD¹¹, Min Dong^12* and Patrick T. McGann, MD, PhD^13,14

¹Cohen Children's Medical Center, Northwell Health, New Hyde Park, NY
²Center for Pediatric and Adolescent Comparative Effectiveness Research, Riley Hospital For Children, Indianapolis, IN
³Pediatric Hematology-Oncology, Children's Hospital - Boston, Boston, MA
⁴Global Blood Therapeutics, South San Francisco, CA
⁵Center for Innovation in Pediatric Practice, Division of Pediatric Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH
⁶Children's Minnesota, Minneapolis, MN
⁷Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁸Carle Foundation Hospital, Urbana, IL
⁹Hematology/Oncology, Phoenix Children's Medical Group, Phoenix, AZ
¹⁰Medical College of Wisconsin, Wisconsin
¹¹University of Illinois Chicago, Peoria, IL
¹²Division of Clinical Pharmacology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
¹³Department of Pediatrics, The Warren Alpert Medical School of Brown University, Providence, RI
¹⁴Lifespan Comprehensive Sickle Cell Center, Providence, RI

Keli C Coleman^1*, Kenneth McKinley, MD^2*, Angela Ellison^3*, Amanda M. Brandow, DO, MS⁴, Blake Nielsen, MS^5*, Lawrence Cook, PhD^5*, Elizabeth Alpern, MD, MSCE^6*, Selena Hariharan, MD^7*, Irina Topoz, MD^8*, Morgan Wurtz, MD^9*, Claudia R. Morris, MD^10,11, Andrew D. Campbell, MD¹², Robert Liem, MD¹³, Rachelle Nuss, MD^14*, Charles T. Quinn, MD¹⁵, Alexis A. Thompson, MD, MPH¹⁶, Anthony Villella, MD^9*, Ana Baumann, PhD^17*, Allison A. King, MD, PhD, MPH¹⁸, Warren Frankenberger, PhD^16* and David C Brousseau, MD, MS¹⁹

¹Medical College of Wisconsin and the Children's Research Institute of Children's Wisconsin, Milwaukee, WI
²Children's National Medical Center, Washington, DC
³Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Pediatric Hematology/Oncology, Medical College of Wisconsin and Children’s Research Institute of the Children’s Hospital of Wisconsin, Milwaukee, WI
⁵University of Utah, Salt Lake City, UT
⁶Lurie Children's Hospital, Chicago, IL
⁷Cincinnati Children's Hospital, Cincinnati, OH
⁸Department of Pediatrics, Children's Colorado, Denver, CO
⁹Nationwide Children's Hospital, Columbus, OH
¹⁰Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
¹¹Childrens Healthcare of Atlanta, Atlanta, GA
¹²Center for Cancer and Blood Disorders, Childrens National Hospital, Washington, DC
¹³Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
¹⁴Center for Cancer and Blood Disorders, University of Colorado Anschutz Medical Campus, Aurora, CO
¹⁵Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Med. Ctr., Cincinnati, OH
¹⁶Children's Hospital of Philadelphia, Philadelphia, PA
¹⁷Washington University School of Medicine, St Louis, MO
¹⁸Pediatric Hematology/Oncology, Washington University School of Medicine, Clayton, MO
¹⁹Department of Pediatrics, Medical College of Wisconsin and the Children's Research Institute at Children's Wisconsin, Milwaukee, WI

Biree Andemariam, MD¹, Pernille Højlund Nielsen^2*, Rikke Zillmer, MSc, PhD^2* and Julie Derving Karsbøl, MD^2*

¹New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT
²Novo Nordisk A/S, Soeborg, Denmark

Patrick T. McGann, MD, PhD^1,2, Christine Briscoe, MSN, RN^2*, Margarida Muhongo^3*, Rui Pascoal^3*, J. Ray Tanzer, PhD^4*, Roxanne Wood^5*, Anastacia da Conceicao Nguvela, MD^6* and Eugenia Alberto Mateus, MSN^6*

¹Department of Pediatrics, The Warren Alpert Medical School of Brown University, Providence, RI
²Lifespan Comprehensive Sickle Cell Center, Providence, RI
³Hospital Pediátrico David Bernardino, Luanda, Angola
⁴Lifespan Biostatistics, Epidemiology, and Research Design, Providence, RI
⁵Brown University Oncology Research Group, Providence, RI
⁶Hospital Geral dos Cajueiros, Luanda, Angola

Deva Sharma, MD, MS¹ and Ilknur Kozanoglu, MD, Prof.^2,3*

¹Hematology-Oncology & Transfusion Medicine, Vanderbilt University Medical Center, Nashville, TN
²Hematology, Baskent University, Adana, Turkey
³Faculty of Medicine, Baskent University, Ankara, Turkey

Xiaoshu Dai Gurov, PhD^1*, Elizabeth Merica^2*, Varsha Iyer, PhD^1*, Annie Claeys^1*, Spurthi Patil^1*, Rolandas Urbstonaitis, MBA, PharmD^1*, James Xiao, PhD^1* and Michael U. Callaghan, MD¹

¹Agios Pharmaceuticals, Inc., Cambridge, MA
²Merica Clinical Consulting LLC, Boston, MA

Landon C. Power^1*, Michael M. Binkley, PhD^2*, Kristin P. Guilliams, MD, MSCI^2,3*, Jade Beshears, BA^2*, Amal Hamed, BA^3*, Anna M Hood, PhD^4*, Jin-Moo Lee, MD, PhD^2,5,6*, Andria L. Ford, MD, MSCI^2,5*, Allison A. King, MD, PhD, MPH⁷ and Melanie E. Fields, MD⁸

¹Department of Pediatrics, Washington University School of Medicine, Hastings, NE
²Department of Neurology, Washington University School of Medicine, Saint Louis, MO
³Department of Pediatrics, Washington University School of Medicine, Saint Louis, MO
⁴Division of Psychology & Mental Health, The University of Manchester, Manchester, United Kingdom
⁵Mallinckrodt Institute of Radiology, Washington University School of Medicine, Saint Louis, MO
⁶Department of Biomedical Engineering, Washington University School of Medicine, Saint Louis, MO
⁷Pediatric Hematology/Oncology, Washington University School of Medicine, Clayton, MO
⁸Division of Pediatric Hematology/Oncology, Washington University in St. Louis, Saint Louis, MO

Chibuzo C Ilonze, MD, MPH¹, Najibah Aliyu Galadanci, MBBS, MPH², Dakshin Padmanabhan, MBBS^3*, Ammar Saadoon, MD^4*, Brandi Pernell, DNP^5* and Jeffrey D. Lebensburger, DO, MSPH⁶

¹Division of Pediatric Hematology and Oncology, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL
²School of Public Health/Epidemiology, University of Alabama at Birmingham, Birmingham, AL
³Pediatric Hematology/ Oncology, University of Alabama at Birmingham, Birmingham, AL
⁴Division of Pulmonology and Sleep Medicine, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL
⁵Division of Pediatric Hematology Oncology, University of Alabama at Birmingham, Birmingham, AL
⁶Division of Pediatric Hematology Oncology, University of Alabama At Birmingham, Birmingham, AL

Jinxia Yao^1*, Andrew RW O'Brien, MD², Yunjie Tong, PhD^1* and Ying Wang, MD, PhD^3*

¹Purdue University, West Lafayette, IN
²Division of Hematology/Oncology, Indiana University School of Medicine, Indianapolis, IN
³Department of Anesthesia, Stark Neurosciences Research Institute, Indiana University School of Medicine, Indianapolis, IN

Emily M. Harris, MD^1*, Emily Vilk, NP^2*, Carolina Donado, MD, MBI^3,4*, Alexis Williams, MD^5*, Matthew M. Heeney, MD⁶, Jean Solodiuk, RN, PhD^3,4*, Christine Greco, MD^3,4* and Natasha M. Archer, MD¹

¹Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
²Division of Hematology,, Boston Children’s Hospital, Boston, MA
³Department of Anesthesiology, Critical Care, and Pain Medicine, Boston Children's Hospital, Boston, MA
⁴Department of Anaesthesia, Harvard Medical School, Boston, MA
⁵Department of Pediatrics, Boston Children's Hospital, Boston, MA
⁶Pediatric Hematology-Oncology, Children's Hospital - Boston, Boston, MA

William Beau Mitchell, MD^1,2, Kaitlin L Strumph, DO, MSc^3,4, Kerry A Morrone, MD⁵, Parmpreet Dhillon, MD^1*, Kevin Hsu, MD^1* and Deepa Manwani, MD⁶

¹Children's Hospital at Montefiore, Bronx, NY
²Albert Einstein College of Medicine, Bronx, NY
³Division of Pediatric Hematology/Oncology, Department of Pediatrics, Children's Hospital at Montefiore, Albert Einstein College of Medicine, New York, NY
⁴Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY
⁵Division of Pediatric Hematology, Oncology and Cellular Therapy, Children's Hospital At Montefiore, Bronx, NY
⁶Division of Pediatric Hematology/Oncology, Department of Pediatrics, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

Angela Liu, MD^1*, Susanna Curtis, MD, PhD², Paola Sebastiani, PhD^3*, Donald Brambilla, PhD⁴ and Jeffrey A. Glassberg, MD⁵

¹Tisch Cancer Institute, Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY
²Icahn School of Medicine at Mount Sinai, New York
³Statistics, Boston University School of Public Health, Boston, MA
⁴RTI International, Rockville, MD
⁵Division of Hematology and Oncology, Department of Medicine and Department of Emergency Medicine, Mount Sinai Medical Center, New York, NY

Kiranveer Kaur, MD, Annum Faisal, MD, Katie Kennedy, DO, Charles L. Knupp, MD and Darla Liles, MD

Division of Hematology Oncology, East Carolina University, Greenville, NC

Ryan Sun, MD^1*, Jin Han, PharmD, PhD^2*, James P. Lash, MD^1*, Victor R. Gordeuk, MD³ and Santosh L. Saraf, MD⁴

¹University of Illinois at Chicago, Chicago
²Department of Pharmacy Practice, University of Illinois at Chicago, Chicago, IL
³Department of Medicine, University of Illinois at Chicago, Chicago, IL
⁴Comprehensive Sickle Cell Center, Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL

Tyler J Barrett^1*, Andrew Pucka, Undergraduate^1*, Brandon Reyes, BS^1*, Seethal A Jacob, MD, MS², Andrew RW O'Brien, MD³, Richard E. Harris, PhD^4*, Steven Harte, PhD^4* and Ying Wang, MD, PhD^1*

¹Department of Anesthesia, Indiana University School of Medicine, Indianapolis, IN
²Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN
³Division of Hematology/Oncology, Indiana University School of Medicine, Indianapolis, IN
⁴Department of Anesthesiology, University of Michigan Medical School, Ann Arbor, MI

Joanna E. Papadakis, BSc^1*, Paulina Piwowarczyk, BA^2*, Daniel S. Weber, BS^2*, Anna L. Slingerland, BS^2*, Dylan S. Keusch^2*, Matthew M. Heeney, MD³, Edward R. Smith, MD^2*, Alfred P. See, MD^2* and Natasha M. Archer, MD⁴

¹Department of Neurosurgery, Boston Children's Hospital, BOSTON, MA
²Department of Neurosurgery, Boston Children's Hospital, Boston, MA
³Pediatric Hematology-Oncology, Children's Hospital - Boston, Boston, MA
⁴Pediatric Hematology-Oncology, Dana-Farber / Boston Children’s Cancer and Blood Disorders Center, Boston, MA

Kennedy N Goldsborough, BSc^1*, Molly Sonenklar, MD^2*, Kalpna Gupta, PhD³, M Imad Damaj, PhD^1*, Wally R Smith, MD⁴, Joyce Lloyd, PhD^5* and Aron Lichtman, PhD^1*

¹Pharmacology and Toxicology, Virginia Commonwealth University, Richmond, VA
²Pediatric Hematology/Oncology, Children's Hospital of Richmond, Richmond
³Department of Medicine, Division of Hematology/Oncology, University of California, Irvine, Irvine, CA
⁴Internal Medicine, Virginia Commonwealth University, Richmond, VA
⁵Virginia Commonwealth University, Richmond, VA

Ibrahim Musa Idris, MBBS, MPH, FMCPath¹, Aminu Abba Yusuf, MBBS, MSc, FMCPath^1*, Ismail Isa Ismail, MBBS^2*, Awwal Musa Borodo, MBBS, FMCPath^3*, Mustapha Shuaibu Hikima, MBBS, FMCRad^4*, Shehu Kana, MBBS, FWACP^5*, Sani A Aji, MBBS, FWACS^6*, Aisha Kuliya_Gwarzo, MBBS, MSc, FMCPath^7*, Mohammad Kabir, BPharm^8*, Jamil Aliyu Galadanci, MSc^9*, Rukayya Alkassim, MSc^10*, Nafiu Hussain, PhD^11*, Mark Rodeghier, PhD^12*, Aurthur Burnett, MD, PhD^13* and Michael R. DeBaun, MD, MPH¹⁴

¹Hematology, Aminu Kano Teaching Hospital/Bayero University Kano, Kano, Nigeria
²Aminu Kano Teaching Hospital, Kano, Nigeria
³Department of Hematology, Murtala Mohammed Specialist Hospital, Kano, Nigeria
⁴Radiology, Muhammad Abdullahi Wase Teaching Hospital, Kano, Nigeria
⁵Medicine, Aminu Kano Teaching Hospital, Kano, Nigeria
⁶Surgery, Aminu Kano Teaching Hospital/Bayero University Kano, Kano, Nigeria
⁷Department of Hematology, Aminu Kano Teaching Hospital/Bayero University Kano, Kano, Nigeria
⁸Department of Pharmacy, Aminu Kano Teaching Hospital, Kano, Nigeria
⁹Department of Computer Science, Bayero University Kano, Kano, Nigeria
¹⁰Department of Mathematical Science, Kano University of Science and Technology,, Kano, Nigeria
¹¹Mathematical Science, Bayero University Kano, Kano, Nigeria
¹²Statistical Consultant, Rodeghier Consultants, Chicago, IL
¹³School of Medicine, Johns Hopkins University, Baltimore, MD
¹⁴Department of Pediatrics, Division of Hematology & Oncology, Vanderbilt University School of Medicine, Nashville, TN

Raffaella Colombatti, MD, PhD^1*, Carolyn Hoppe, MD^2*, Elliott P. Vichinsky, MD³, Mona Hamdy, MD, MCRA^4*, Adeseye Michael Akinsete, MBBS^5*, Mark Davis^2*, Halima Bello-Manga, MBBS, MPH, FMCPath⁶, Iheanyi Okpala, FWACP, FRCPath⁷, Ana Cristina Silva Pinto, MD, PhD^8* and Adlette Inati, MD, FAAP^9,10

¹Department of Woman's and Child's Health, Pediatric Hematology Oncology, University of Padova, Padova, Italy
²Global Blood Therapeutics, South San Francisco, CA
³UCSF Benioff Children's Hospital Oakland, Oakland, CA
⁴Clinical Research center, Faculty of Medicine, Cairo University, Cairo, Egypt
⁵College of Medicine, Division of Pediatric Hematology & Oncology, Lagos University Teaching Hospital, Lagos, Nigeria
⁶Barau Dikko Teaching Hospital, Kaduna State University, Kaduna, Nigeria
⁷Department of Hematology, University of Nigeria, Enugu, Nigeria
⁸Center for Cell-Based Therapy, Hemotherapy Center of Ribeirão Preto, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil
⁹Lebanese American University, Beirut and Nini Hospital, Tripoli, Lebanon
¹⁰Division of Pediatric Hematology and Oncology, Nini Hospital, Tripoli, Lebanon

Anthea Louise Greenway, MBBS, FRACP, FRCPA, QIA^1,2,3, Zane Kaplan, MBBS FRACP FRCPA PhD^4,5,6, Pasquale Barbaro, MBBS FRACP FRCPA PhD^7*, Tina Carter, PhD, FRACP, FRCPA⁸, Juliana Teo, MBBS, FRACP, FRCPA⁹ and Manika Pal, MBBS, FRACP FRCPA^10*

¹Congenital Blood Disorders, Monash Medical Centre, Clayton, VIC, Australia
²Royal Children's Hospital, Parkville, VIC, Australia
³Dept of Paediatrics, University of Melbourne, Parkville, VIC, Australia
⁴Monash Medical Centre, Clayton, Australia
⁵Australian Centre for Blood Diseases, Melbourne, AUS
⁶Monash University, Melbourne, Australia
⁷Queensland Children's Hospital, Brisbane, QLD, Australia
⁸Perth Children's Hospital, Nedlands, WEA, AUS
⁹The Children's Hospital Westmead, Westmead, NSW, AUS
¹⁰Dept of Haematology and Oncology, Womens and Children's Hospital, North Adelaide, SA, Australia

Ashrei Bayewitz and Sai Shalini Pillarisetty, MD

Coney Island Hospital, Brooklyn, NY

Olufunke Y. Martin, MD¹, Stefanie L. Margulies, MS^1*, Deepika S. Darbari, MD^1,2, Patrick C. Hines, MD/PhD^3,4 and Andrew D. Campbell, MD^2,5

¹Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC
²The George Washington University School of Medicine and Health Sciences, Washington, DC
³Functional Fluidics, Detroit, MI
⁴Carman and Ann Adams Department of Pediatrics, Division of Pediatric Critical Care, Children’s Hospital of Michigan/Wayne State University, Detroit, MI
⁵Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC

Alexandra Power-Hays, MD¹, Min Dong^2,3*, Kathryn McElhinney, BS^4*, Andrea L. Conroy, PhD^5,6*, Teresa Latham, MA^7,8*, Adam Lane, PhD^7,9*, Robert O. Opoka, MMED, PhD^10,11*, Alexander A. Vinks, PharmD, PhD, FCP^2,3* and Russell E. Ware^3,4,12,13

¹Cancer and Blood Disorders Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Division of Clinical Pharmacology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
³Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
⁴Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁵Indiana University, Indianapolis, IN
⁶Mulago Hospital, Global Health Uganda, Kampala, Uganda
⁷Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁸Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati
⁹University of Cincinnati - College of Medicine, Cincinnati, OH
¹⁰Global Health Uganda, Kampala, Uganda
¹¹Paediatrics and Child Health, Makerere University, Kampala, Uganda
¹²Division of Hematology, Children's Hospital Medical Center, Cincinnati, OH
¹³Global Health Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH