Larissa Doll^1*, Narges Aghaallaei^1*, Julia Skokowa, MD, PhD¹, Karl Welte² and Baubak Bajoghli^1*

¹Division of Translational Oncology, Department of Hematology, Oncology, Clinical Immunology, University Hospital Tuebingen, Tuebingen, Germany
²Pediatric Hematology and Oncology, University Children Hospital Tuebingen Germany, Tuebingen, Germany

David R Sweet^1*, Asha Thomas, PhD^2*, Stephanie D Lapping, MS^3*, Kenneth A Kalikasingh^2*, Thomas L. Ortel, MD, PhD⁴, Mukesh K Jain, MD^1*, Andrei Maiseyeu^3* and Lalitha V. Nayak, MD²

¹Cardiovascular Research Institute, Case Western Reserve University and University Hospitals Cleveland Medical Center, Cleveland, OH
²Department of Medicine, Division of Hematology and Oncology, Case Western Reserve University and University Hospitals Cleveland Medical Center, Cleveland, OH
³Cardiovascular Research Institute, Case Western Reserve University and University Hospitals Cleveland Medical Center, Cleveland
⁴Department of Medicine, Duke University Medical Center, Durham, NC

Jean Donadieu, MD, PhD¹, Fares bou Mitri^2*, Blandine Beaupain, MsC^3*, Yves Bertrand, M.D.^4,5*, Pierre Simon Rohrlich, MD PhD^6,7*, Nathalie Aladjidi, M.D.^8,9*, Alexia Rouland, MD^10*, Felipe Suarez, MD, PhD¹¹, Andre Vanoli^12*, Jean Fraisse, MD^13*, Olivier Hermine^{14,15,16,17,18,19,20,21}, Philippe Descamps^22*, Sylvie François, MD^23,24*, Hélène Lapillonne, MD, PhD^25*, Marlène Pasquet, M.D., Ph.D.^26*, Matthieu Patient^27*, Ilona Okhremchuck^28*, Didier Blaise, MD²⁹, Faezeh Legrand, MD^30*, Claire Fieschi, MD, PhD^31*, Flore Sicre de Fontbrune^32,33,34*, Sarah cohen Beaussant^2*, didier Simon Kamioner, MD^35*, Francoise Bachelerie, PhD^36*, Claire Deback, MD, PHD^37,38*, Christine Bellanne-Chantelot, PhD, PharmD^39* and Jean-François Emile, MD, PhD^40*

¹Service d'Hémato-Oncologie Pédiatrique, Hopital Trousseau, Paris, Cedex, France
²Centre de référence des neutropénies chroniques Registre des neutropenies, Hopital Trousseau, Paris, France
³Registre des Neutropénies, Hopital Trousseau, AP-HP, Paris, France
⁴Institute of Pediatric Hematology and Oncology, Civil Hospital of Lyon, Claude Bernard University, Lyon, France
⁵Claude Bernard University, Lyon 1, Lyon, France
⁶Hematology-Pediatry, Bensancon, FRA
⁷CHU L'Archet, Nice, FRA
⁸Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux, France
⁹Pediatric Oncology Hematology Unit, Plurithématique CIC (CICP), Centre d’Investigation Clinique (CIC) 1401, INSERM, Bordeaux University Hospital, Bordeaux, France
¹⁰Endocrinology department, CHU Dijon, DIJON, France
¹¹Service d'Hématologie adultes, Hôpital Universitaire Necker-Enfants Malades, AP-HP, Paris, France
¹²Département d'oncologie médicale, Clinique Sainte Marthe, Dijon, Bourgogne, France,, Dijon, France
¹³Department of pathology, Centre Georges-François Leclerc, DIJON, France
¹⁴Imagine Institute, INSERM U1163, University of Paris, Paris, France
¹⁵Department of Hematology, Necker Children's hospital, APHP, Paris, France
¹⁶Laboratory of Excellence Gr-Ex, Paris, France
¹⁷Department of Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
¹⁸Hematology Department, AP-HP Hôpital Necker, University Paris Descartes, Paris, France
¹⁹Clinical Hematology, Necker University Hospital, Paris, France
²⁰Service Hèmatologie Adulte, Centre de référence des mastocytoses, Hôpital Universitaire Necker-Enfants Malades, Paris, France
²¹Centre de référence des déficits immunitaires héréditaires (CEREDIH), Necker Children's hospital, APHP, Paris, France
²²Departement of gynecology, CHU Angers, angers, France
²³Clinical Hematology, Angers University Hospital, Angers, France
²⁴Service Maladies du sang, CHU Angers, Angers, France, Angers, France
²⁵Pediatric Hematology and Oncology Department, Hôpital Armand Trousseau, Paris, France
²⁶Pediatric Oncology Immunology Hematology Unit, Children’s hospital, CHU de Toulouse, Toulouse, France
²⁷Oncology department, Hôpital d'Instruction des armées SAINTE ANNE, Toulon, FRA
²⁸Department of pathology, Hôpital d'Instruction des armées SAINTE ANNE, toulon, France
²⁹Institut Paoli Calmettes, Marseille, France
³⁰Hematology department, Institut Paoli Calmettes, Marseille, France
³¹Service d’Immunologie clinique, Hôpital Saint-Louis, AP-HP, Paris, France
³²Hematology and transplantation unit, Saint-Louis Hospital, Paris, France
³³Centre de Référence Aplasie Médullaire, Service d’Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France
³⁴French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint Louis Hospital and University Paris Diderot, Paris, France
³⁵oncology, Hopital Prive de l'ouest parisien, Trappes, France
³⁶Immunologie 92296 Châtenay-Malabry, Université Paris Saclay Faculte de Pharmacie Inserm U996, Clamart, FRA
³⁷Service de Virologie HOP PAUL BROUSSE, AP-HP. UNIVERSITÉ PARIS SACLAY, villejuif, France
³⁸Inserm U996, inserm, CLAMART, France
³⁹Hopital Pitié Salpétriére APHP, Département de Génétique, AP-HP Hôpital Pitié- Salpêtrière, UPMC Univ Paris 06, Paris, France
⁴⁰Pathology Department,, Hôpital Ambroise-Paré APHP, Université Paris Saclay, Boulogne, FRA

Douglas W. Blayney, MD¹, Ramon Mohanlal, PhD, MD^2* and Lan Huang, Ph.D.^2*

¹Stanford University, Palo Alto, CA
²BeyondSpring Pharmaceuticals, New York, NY

Juliana Vago, PhD, MSc^1,2*, Larissa Froede Brito^3*, Lívia C.R. Teixeira^3*, Izabela Z. Moreira^3*, Thaís C. Miranda^3*, Eliza M. Melo^3*, Alexandre S. Bruno^3*, Michelle A. Sugimoto^3*, Raphael G. Ferreira^4*, Jose C. Alves-Filho^5*, Mauro M. Teixeira^3*, Robert J Parmer, M.D.^6*, Lindsey A Miles, PhD⁷ and Lirlandia P. Sousa^3*

¹Federal University of Minas Gerais, BELO HORIZONTE, Brazil
²Scripps Research, La jolla
³Federal University of Minas Gerais, Belo Horizonte, Brazil
⁴Federal University of Sao Paulo, Ribeirao Preto, Brazil
⁵University of Sao Paulo, Ribeirao Preto, BRA
⁶Department of Medicine, University of California San Diego and Veterans Administration San Diego Healthcare System, San Diego, CA
⁷Department of Molecular Medicine, The Scripps Rsch. Inst., La Jolla, CA

Jean Donadieu, MD, PhD¹, Blandine Beaupain^2*, Fares bou Mitri^3*, Flore Sicre de Fontbrune^4,5,6,7*, Despina Moushous, MD, PhD^8*, Thierry Lamy, PhD, MD^9,10,11, Aline Moignet Autrel, MD^12*, Elodie Gouache^13*, Marlène Pasquet, M.D., Ph.D.^14* and Christine Bellanne-Chantelot, PhD, PharmD^15*

¹Service d'Hémato-Oncologie Pédiatrique, Hopital Trousseau, Paris, Cedex, France
²Registre francais des neutropénies chroniques, HôPital Trousseau, France, FRA
³Centre de référence des neutropénies chroniques Registre des neutropenies, Hopital Trousseau, Paris, France
⁴Hematology and transplantation unit, Saint-Louis Hospital, Paris, France
⁵Centre de Référence Aplasie Médullaire, Service d’Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France
⁶French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint Louis Hospital and University Paris Diderot, Paris, France
⁷St-Louis Hospital, APHP, Bone Marrow Transplantation Unit, Paris, France
⁸Unité d'immuno hématologie rhumatologie pédiatrique, APHP université de Paris Hopital Necker enfants malades, Paris, France
⁹UMR U1236, Rennes, France
¹⁰INSERM CIC 1414, Hospital Pontchaillou, Rennes, France
¹¹Department of Hematology, INSERM U917/University Hospital of Rennes, Rennes, France
¹²Clinical Hematology Department, Rennes CHU, Rennes, France
¹³Department of Pediatric Hemato-Oncology, Hospital Armand Trousseau, Paris, Franc, Paris, ESP
¹⁴Pediatric Oncology Immunology Hematology Unit, Children’s hospital, CHU de Toulouse, Toulouse, France
¹⁵Hopital Pitié Salpétriére APHP, Département de Génétique, AP-HP Hôpital Pitié- Salpêtrière, UPMC Univ Paris 06, Paris, France

Omar Abughanimeh, MD¹, Ayman Qasrawi, MD², James O. Armitage, MD¹, Julie M. Vose, MD, MBA¹, Gerhard C. Hildebrandt, MD² and Muhamed Baljevic, MD¹

¹Division of Oncology and Hematology, University of Nebraska Medical Center- Fred & Pamela Buffett Cancer Center, Omaha, NE
²Department of Internal Medicine, Division of Hematology/BMT, University of Kentucky, Lexington, KY

Christen L. Ebens, MD, MPH¹, Adam S DuVall, MD², Todd E. DeFor, MS^3*, Marketa T. Dimitrov, MD^4*, David Frame^5*, Michael A. Briones^6*, Michael Grimley, MD⁷, Sarita Joshi, MBBS, MD⁸, Rebecca A. Marsh^9*, Evan Shereck, MD¹⁰, Mark Vander Lugt, MD^11*, James A. Connelly, MD^12* and Kelly J. Walkovich, MD¹³

¹Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN
²Departments of Medicine and Pediatrics, Oregon Health & Science University, Portland, OR
³Biostatistics Core, Masonic Cancer Center, University of Minnesota, Minneapolis, MN
⁴Department of Pediatrics, University of Minnesota, Minneapolis, MN
⁵University of Michigan, Dexter, MI
⁶Pediatric Hematology/Oncology, Children's Hospital of Atlanta, Atlanta, GA
⁷Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁸Nationwide Childrens Hospital, Seattle, WA
⁹Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
¹⁰Pediatric Hematology Oncology, Oregon Health & Science University, Portland, OR
¹¹Division of Pediatric Hematology/Oncology; Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, MI
¹²Vanderbilt University Medical Center, Nashville, TN
¹³University of Michigan, Ann Arbor, MI