Min Dong^1,2*, Alina Sadaf, MBBS³, Alexander A. Vinks, PharmD, PhD, FCP^1,2*, Russell E. Ware^2,3 and Charles T. Quinn, MD^2,3

¹Division of Clinical Pharmacology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
³Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Landon C. Power^1*, Amy E. Mirro, MEng^2*, Michael M. Binkley, PhD^3*, Kristin P. Guilliams, MD, MSCI^2,3*, Josiah B. Lewis, PhD^3*, Jade Beshears, BA^3*, Andria L. Ford, MD, MSCI^3,4*, Joshua S. Shimony, MD, PhD^4*, Hongyu An, DSc^4*, Jin-Moo Lee, MD, PhD^3,4,5* and Melanie E. Fields, MD⁶

¹Department of Pediatrics, Washington University School of Medicine, Hastings, NE
²Department of Pediatrics, Washington University School of Medicine, Saint Louis, MO
³Department of Neurology, Washington University School of Medicine, Saint Louis, MO
⁴Mallinckrodt Institute of Radiology, Washington University School of Medicine, Saint Louis, MO
⁵Department of Biomedical Engineering, Washington University School of Medicine, Saint Louis, MO
⁶Division of Pediatric Hematology/Oncology, Washington University in St. Louis, Saint Louis, MO

Akshay Sharma, MBBS¹, John P Manis, MD², David G. Justus, MD^3*, David A. Williams, MD^3*, Sergey Efuni, MD, PhD^4*, Ji Hyun Lee, MD, MPH^4*, Erin Conlin, MS^4*, Shawn Rose, MD, PhD⁴, Jeffrey Humphrey, MD⁴, Kirk Bertelsen, PhD^4*, Nicole Henry, BSN, RN^4*, June Li, PhD^4*, Donald Pardy, MS^4*, David Santos, BS^4*, Christian Zdybowicz, MBA^4*, Alexis Leonard, MD^5* and John Tisdale, MD⁵

¹St. Jude Children's Research Hospital, Memphis, TN
²Department of Laboratory Medicine, Boston Children’s Hospital, Harvard Medical School, Boston, MA
³Boston Children's Hospital, Boston, MA
⁴Magenta Therapeutics, Cambridge, MA
⁵Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Slimane Allali, MD, PhD^1,2,3*, Juliette Elie, MD^2,3*, Mariane de Montalembert, MD, PhD^1,2, Melissa Taylor, MD, PhD^1,2*, Joséphine Brice, MD, PhD^2*, Stéphanie Chhun, PharmD, PhD^4*, Rachel Rignault-Bricard, BSc^1,3*, Claire Heilbronner, MD^5*, Thiago Trovati Maciel, PhD^1,3* and Olivier Hermine, MD, PhD^1,3*

¹Laboratory of Excellence GR-Ex, Paris, France
²Department of General Pediatrics and Pediatric Infectious Diseases, Sickle Cell Center, Necker-Enfants malades Hospital, Assistance Publique – Hôpitaux de Paris (AP-HP), Université Paris Cité, Paris, France
³Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutical Implications, Université Paris Cité, Imagine Institute, Inserm U1163, Paris, France
⁴INEM U1151 "Immunorégulation et Immunopathologie", Laboratoire d’Immunologie biologique, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
⁵Department of Pediatric Intensive care, Necker-Enfants malades Hospital, AP-HP, Université Paris Cité, Paris, France

Ashwin Patel, MD, MPH^1*, Dorothy Kim Waller, PhD^1*, Luis Leon Novelo, PhD^1*, Jonathan M. Flanagan, PhD² and Vivien A. Sheehan, MD, PhD³

¹The University of Texas Health Science Center at Houston (UTHealth), School of Public Health, Houston, TX
²Department of Pediatrics - Hematology/Oncology, Baylor College of Medicine, Houston, TX
³Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

Kuldeepsinh Atodaria, MBBS¹, Richard S Eisenstaedt, MD^1*, Christian Joseph Fidler, MD^2* and Sarin Atam, MD^1*

¹Jefferson Health - Abington, Abington, PA
²Hematology and Medical Oncology, Jefferson Health - Abington, Abington, PA

Maria A. Lizarralde-Iragorri, PhD^1*, Bindu Parachalil Gopalan, PhD^1*, Brenda Merriweather, RN, MSN^1*, Jennifer Brooks, RN^2*, Mai Hill, RN, BSN, MHSA^1*, Anna Conrey, NP^1*, Ruth Pierre Charles, DSc, PA-C^1*, Eveline Gwaabe, CRNP^1*, Neal Jeffries, PhD^3* and Arun S. Shet, MD, PhD¹

¹Sickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD
³Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Bindu Parachalil Gopalan, PhD^1*, Maria A. A. Lizarralde-Iragorri, PhD^1*, Anna Conrey, NP^1*, Mai Hill, RN, BSN, MHSA^1*, Brenda Merriweather, RN, MSN^1*, Jennifer Brooks, RN^2*, Ruth Pierre Charles, DSc, PA-C^1*, Eveline Gwaabe, CRNP^1*, Rafael Villasmil, M.Sc., C.Cy.^3*, Neal Jeffries, PhD^4* and Arun S. Shet, MD, PhD¹

¹Sickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD
³Flow Cytometry Core Facility, National Eye Institute, National Institutes of Health, Bethesda, MD
⁴Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Ada Chang, BSc^1*, Sherri Morris, MSN, AGPCNP-BS^2*, Barbara L LeVarge, MD^3*, Samuel R Wilson, MD, MSCR^2* and Jane Alison Little, MD^4,5,6

¹School of Medicine, UNC Blood Research Center, University of North Carolina, Chapel Hill, Chapel Hill, NC
²Department of Medicine, Division of Hematology, University of North Carolina, Chapel Hill, Chapel Hill, NC
³3. Department of Medicine, Division of Pulmonary Diseases and Critical Care Medicine, University of North Carolina, Chapel Hill, Chapel Hill, NC
⁴Department of Medicine, UNC Adult Sickle Cell Program, Chapel Hill, NC
⁵Departments of Pediatrics and Medicine, UNC Hospitals, Chapel Hill, NC
⁶Department of Medicine, Division of Hematology, University of North Carolina at Chapel Hill, Chapel Hill, NC

Caterina P Minniti, MD¹, Henny H. Billett, MD MSc², Daniel McMahon, MD³, Modupe Idowu, MD⁴, Nirmish Shah, MD⁵, Richard A. Drachtman, MD⁶, Archana Sharma, DO^6*, Alexander Glaros, MD⁷, Maureen Okam Okam Achebe, MD, MPH⁸, Alecia C Nero, MD^9*, Susanna Curtis, MD, PhD¹⁰ and Biree Andemariam, MD¹¹

¹Department of Hematology, Einstein College of Medicine, Chevy Chase, MD
²Division of Hematology, Department of Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY
³Atrium Health Levine Cancer Institute, Charlotte, NC
⁴University of Texas McGovern Medical School, Houston, TX
⁵Division of Hematology, Duke University School of Medicine, Durham, NC
⁶Rutgers Robert Wood Johnson Medical School, New Brunswick, NJ
⁷Children's Hospital of Michigan, Department of Pediatrics, Division of Pediatric Hematology-Oncology, Central Michigan University, Detroit, MI
⁸Hematology Division Brigham and Women's, Boston, MA
⁹University of Texas Southwestern Medical Center, Dallas, TX
¹⁰Icahn School of Medicine at Mount Sinai, New York
¹¹New England Sickle Cell Institute at the University of Connecticut, Farmington, CT

Claudia Rodriguez Rivera, MD^1*, Andrew Srisuwananukorn, MD^2*, Rizma Jalees Bajwa, MD^1*, Victor R. Gordeuk, MD³, Joyce Rauch, PhD^4*, Jerrold Levine, MD^1* and Santosh L. Saraf, MD⁵

¹University of Illinois at Chicago, Chicago
²Mount Sinai Health System, New York, NY
³Sickle Cell Center, Division of Hematology and Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL
⁴McGill University Health Centre, MONTREAL, QC, CAN
⁵Comprehensive Sickle Cell Center, Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL

Syeda Akila Ally, BA^1*, Jin Han, PharmD, PhD^2*, Robert E. Molokie, MD³, James P. Lash, MD^4*, Victor R. Gordeuk, MD⁵ and Santosh L. Saraf, MD⁶

¹University of Illinois at Chicago, Chicago, IL
²Department of Pharmacy Practice, University of Illinois at Chicago, Chicago, IL
³Department of Medicine, University of Illinois at Chicago, Jesse Brown VA Medical Center, Chicago, IL
⁴University of Illinois at Chicago, Chicago
⁵Sickle Cell Center, Division of Hematology and Oncology, Department of Medicine, University of Illinois at Chicago, Carol Stream, IL
⁶Department of Medicine, University of Illinois at Chicago, Chicago, IL

Neha J Desai, DO¹, Ana Lucia Gonzalez-Herrera, MD^2*, Sindhoosha Malay, MPH, PharmD^2*, Tracie Brown, FNP-C^3*, Amma Owusu-Ansah, MD^3* and Sanjay Ahuja, MD⁴

¹Division of Pediatric Hematology & Oncology, University Hospitals Rainbow Babies & Children's Hospital, Cleveland Heights, OH
²Department of Pediatrics, University Hospitals Rainbow Babies & Children's Hospital, Cleveland, OH
³Division of Pediatric Hematology & Oncology, University Hospitals Rainbow Babies & Children's Hospital, Cleveland, OH
⁴Division of Pediatric Hematology & Oncology, Rainbow Babies and Children's Hospital, Cleveland, OH

Jayla Lynn Scott^1*, Jana Christian, MD^2*, Manuela Plazas Montana^2*, Yvette M. Miller, MD^3* and Rakhi P. Naik, MD, MHS²

¹Johns Hopkins University, Baltimore, MD
²Division of Hematology, Department of Medicine, Johns Hopkins University, Baltimore, MD
³American Red Cross, Charlotte, NC

Kayla Baker, MSBHS^1*, Michael Ho^1*, Chibuzo C Ilonze, MD, MPH², Malgorzata Kasztan, PhD¹ and Jeffrey D. Lebensburger, DO, MSPH³

¹University of Alabama at Birmingham, Birmingham, AL
²Division of Pediatric Hematology and Oncology, Department of Pediatrics, The University of Alabama at Birmingham, Birmingham, AL
³Division of Pediatric Hematology Oncology, University of Alabama At Birmingham, Birmingham, AL

Kok Hoe Chan, MD¹, Ruhi Buddharaju, BA^2*, Shandel L Chang, BS^2*, John S Lane, DO^2* and Modupe Idowu, MD¹

¹Department of Internal Medicine, Division of Hematology, McGovern Medical School, University of Texas Health Science Center at Houston, Houston, TX
²McGovern Medical School, University of Texas Health Science Center at Houston, Houston, TX

Reema Kashif, MD¹, Mark Rodeghier, PhD^2*, Shaina M Willen, MD³, Michael R. DeBaun, MD, MPH⁴ and Evans Machogu, MD^5*

¹Department of Pediatrics, Division of Hematology Oncology, University of Florida, Gainesville, FL
²Statistical Consultant, Rodeghier Consultants, Chicago, IL
³Department of Pediatrics, Division of Pulmonary Medicine, University of California - Davis, Sacramento, CA
⁴Department of Pediatrics, Division of Hematology & Oncology, Vanderbilt University School of Medicine, Nashville, TN
⁵Department of Pediatrics, Division of Pulmonology, Allergy, and Sleep Medicine, Riley Hospital for Children at Indiana University Health, Indianapolis, IN

Abisola Baruwa Etti, MBBS^1*, Chia-ling Kuo, Ph.D^2*, Lucas Da Cunha Godoy, M.Sc^3* and Biree Andemariam, MD⁴

¹Internal Medicine, University of Connecticut Health Center, Farmington, CT
²Connecticut Convergence Institute for Translation in Regenerative Engineering, University of Connecticut Health Center, Farmington
³Connecticut Convergence Institute for Translation in Regenerative Engineering, University of Connecticut Health Center, Farmington, CT
⁴New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT

Amy Parker Ruhl, MD, MHS^1*, Ruba Shalhoub, MS^2*, Neal Jeffries, PhD^3*, Alexis Leonard, MD^4*, Emily Limerick, MD^3*, Amisha Barochia, MBBS, MHS^5*, Matthew M. Hsieh, MD⁴ and Courtney D. Fitzhugh, MD³

¹Laboratory of Malaria and Vector Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Washington, DC
²Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
³National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁴Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁵Pulmonary Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD

Rowan O. Brothers, BS^1*, Katherine B. Turrentine, BS^1*, Mariam Akbar, BS^1*, Sydney Triplett, BS^1*, Tara M. Urner, BS^1*, Amy Y. Tang, MD^2,3*, Clark Brown, MD, PhD^2,4,5 and Erin M. Buckley, PhD^1,2,5*

¹Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA
²Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, Atlanta, GA
³Department of Pediatrics, Emory University, Atlanta, GA
⁴Global Blood Therapeutics, South San Francisco, CA
⁵Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

Yang Dai^1*, Kelley Hill^1*, Daniel Harris^1*, Tong Shen^1*, Chao-Xing Yuan^1* and Christoph Gasteyger^2*

¹Alexion, AstraZeneca Rare Disease, Boston, MA
²Alexion, AstraZeneca Rare Disease, Zurich, MA, Switzerland

Christophe Ferreira De Matos, MD^1*, Pierre Cougoul, MD^2*, Oana Zaharie, MD^1*, Aurore Perrot, MD, PhD^3*, Marc Kermorgant, PhD^4*, Anne Pavy-Le Traon, MD, PhD^1*, Celine Gales, PhD^5*, Jean-Michel Senard, MD, PhD^5*, Fabrice Bonneville, MD, PhD^1* and Nathalie Nasr, MD, PhD^6*

¹Centre hospitalier universitaire de Toulouse, Toulouse, France
²Oncopole, Toulouse, FRA
³CHU de Toulouse, IUCT-O, Université de Toulouse, UPS, Service d’Hématologie, Toulouse, France
⁴UMR 1297 Equipe 10 Institute of Metabolic and Cardiovascular Disease (I2MC) Toulouse France, Toulouse, France
⁵UMR 1297 equipe 10 Institute of Metabolic and Cardiovascular Disease (I2MC), Toulouse, France
⁶Department of Neurology Toulouse University Hospital, Centre hospitalier universitaire de Toulouse, Toulouse, France

Stephanie H Guarino, MD^1,2, Mark D Nelms, PhD^3*, David Williams, MEng^4*, Amanda M. Brandow, DO, MS⁵, C. Patrick Carroll, MD^6*, Nitya Bakshi, MBBS, MS⁷, Claudia M Campbell, PhD^8*, Deepika S. Darbari, MD⁹, Martha Kenney, MD^10*, Wally R Smith, MD¹¹, Jennifer Stinson, PhD, CNP, RN^12*, William Zempsky, MD^13*, Wayne Huggins, PhD^4*, Deborah Maiese, MPA^4*, Tabitha Hendershot, BA^4* and Carol Hamilton, PhD^4*

¹Nemours Dupont Hospital For Children, Wilmington, DE
²Center for Special Health Care Needs/Institute on Research in Equity and Community Health, ChristianaCare, Newark, DE
³RTI International, Durham, NC
⁴RTI International, Research Triangle Park, NC
⁵Medical College of Wisconsin, Milwaukee, WI
⁶Johns Hopkins University School of Medicine, Baltimore, MD
⁷Department of Pediatrics/Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA
⁸Department of Psychiatry and Behavioral Health, Johns Hopkins University School of Medicine, Baltimore, MD
⁹Comprehensive Sickle Cell Disease Program, Children's National Hospital, Washington, DC
¹⁰Duke University, Durham, NC
¹¹Internal Medicine, Virginia Commonwealth University, Richmond, VA
¹²Hospital For Sick Children, Toronto, ON, CAN
¹³University of Connecticut School of Medicine, Connecticut Children's Medical Center, Hartford, CT

Macy L. Early, BA¹, A. Kinga Malinowski, MD, MSc, FRCSC², Marissa Solow, MD^3*, Ahizechukwu Eke, MBChB, PhD^4,5*, Nadine Shehata, MD, FRCPC, MSc^6,7,8, Sana Saif Ur Rehman, MD^9*, Kevin H.M. Kuo, MD, MSc, FRCPC^10,11,12 and Lydia H. Pecker, MD¹³

¹Division of Hematology, Department of Medicine, Johns Hopkins Hospital, Baltimore, MD
²Division of Maternal-Fetal Medicine, Department of Obstetrics and Gynecology, Mt. Sinai Hospital, Toronto, ON, CAN
³University Health Network, Toronto, ON, Canada
⁴Division of Clinical Pharmacology, Johns Hopkins School of Medicine, Baltimore, MD
⁵Division of Maternal-Fetal Medicine, Department of Gynecology & Obstetrics, Johns Hopkins University School of Medicine, Baltimore, MD
⁶Department of Medicine, Toronto, Canada
⁷Mount Sinai Hospital, Toronto, ON, Canada
⁸Canadian Blood Services, Toronto, ON, Canada
⁹Division of Hematology, Washington University School of Medicine, Saint Louis, MO
¹⁰Institute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada
¹¹Division of Hematology, Department of Medicine, University of Toronto, Toronto, ON, Canada
¹²Division of Medical Oncology and Hematology, Department of Medicine, University Health Network, Toronto, ON, Canada
¹³Division of Hematology, Department of Medicine, Johns Hopkins Medicine, Baltimore, MD

Fuad El Rassi, MD^1,2, Ross M. Fasano, MD^3* and Morgan L. McLemore^4,5

¹Department of Hematology and Medical Oncology, Emory University School of Medicine and Georgia Comprehensive Sickle Cell Center at Grady Health System, Atlanta, GA
²Department of Hematology and Medical Oncology, Winship Cancer Institute, Atlanta, GA
³Center for Transfusion and Cellular Therapies, Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, GA
⁴Grady Health System, Atlanta, GA
⁵Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA

Nour Belkeziz^1*, Marie-Pierre Gobin-Metteil^2*, Corentin Provost^1*, Suella Martino, MD PhD^3*, Myriam Edjlali^4*, Nicolas Mele^1*, Benjamin Maïer^2*, Anne Blanchet-Deverly^5*, Jean-Benoit Arlet^6*, Laure Joseph, MD^7*, Pablo Bartolucci, MD, PhD^8* and David Calvet, MD, PhD^2*

¹Centre Hospitalier Sainte-Anne, Paris, France
²Centre Hospitalier Sainte-Anne, Paris, FRA
³APHP Hôpital Henri Mondor, Creteil, France, Creteil, France
⁴CENTRE HOSPITALIER SAINTE ANNE, INSERM S 894, PARIS, FRA
⁵CHU de Guadeloupe, Poite à Pitre, France
⁶Hopital Europeen Georges Pompidou, Paris, FRA
⁷Biotherapy Department, French National Sickle Cell Referral Center, Hôpital Necker, Assistance-Publique Hôpitaux de Paris, Paris, DC, France
⁸Université Paris-Est Créteil, INSERM, IMRB, Laboratory of excellence LABEX GRex, Créteil, France

Anoosha Habibi, MD, PhD^1,2, Marion Lefebvre, MD^3*, Octavia Sobcyk, MD^4*, Florence Canouï-Poitrine, MD^5*, Amna Jebali, MD^6*, Gonzalo De Luna, MD^7*, Pablo Bartolucci, MD, PhD^8,9*, Frédéric Galactéros, MD, PhD^10,11*, France Pirenne, MD, PhD^8,12, Diane Redel^13*, Emmanuelle Boutin, MD^14*, Bassam Haddad, MD, PhD^15,16* and Edouard Lecarpentier, MD, PhD^3*

¹IMRB-U955, Equipe 2 émergente: Transfusion et maladies du globule rouge, CRETEIL, France
²Sickle cell disease french referral center , Henri-Mondor university hospital, Créteil, France
³Service de Gynécologie Obstétrique,, Centre Hospitalier Intercommunal de Créteil, Créteil, France
⁴Centre Hospitalier Intercommunal de Créteil, CRETEIL, France
⁵Department of Public Health and Biostatistics, Henri Mondor Hospital, Creteil, France
⁶Sickle cell referral center, APHP Hôpital Henri Mondor, Creteil, France, Creteil, France
⁷Sickle cell referal Center, APHP Hôpital Henri Mondor, Creteil, France, Creteil, France
⁸INSERM-U955, Equipe 2, Laboratoire D'Excellence, GRex, Institut Mondor, Créteil, France
⁹Department of Internal Medicine, Reference Center of Sickle Cell Disease, Mondor University Hospital, Créteil, France
¹⁰Sickle cell referral center, Internal Medicine Unit, Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, Creteil, France
¹¹INSERM-U955, EFS, Institut Mondor, Université Paris-Est Créteil, Team 2 Transfusion et maladies du globule rouge, Laboratoire d´Excellence GR-Ex, Créteil, France
¹²Etablissement Français du Sang, Creteil, France
¹³Service de Gynécologie Obstétrique,, Centre Hospitalier Intercommunal de Créteil, Creteil, France
¹⁴Department of Public Health and Biostatistics, Henri Mondor Hospital, Créteil, France
¹⁵service de gyneco-obstetrique, Centre Hospitalier Intercommunal de Créteil, Creteil, France
¹⁶UPEC, University Paris East Creteil, Creteil, France