Masahiro Ikeda^1*, Muneyoshi Futami, MD, PhD^1*, Bidisha Chanda, MD, PhD^2*, Masayuki Kobayashi, MD, PhD^1*, Kiyoko Izawa, PhD^3* and Arinobu Tojo, MD, PhD¹

¹Division of Molecular Therapy, Advanced Clinical Research Center, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan
²Division of Molecular Therapy, Advanced Clinical Research Center, The Institute of Medical Science, The University of Tokyo, Tokyo, JPN
³Division of Stem Cell Biology, Center for Stem Cell Biology and Regenerative Medicine, The Institute of Medical Science, The University of Tokyo, Tokyo, JPN

Jean Donadieu, MD, PhD¹, Blandine Beaupain, MsC^2*, Hélène Lapillonne, MD, PhD^3*, Odile Fenneteau, PharmD^4*, Flore Sicre de Fontbrune^{5,6,7,8,9,10*}, Yves Bertrand, M.D.^11,12*, Nathalie Aladjidi, M.D.^13,14*, Vincent Barlogis, M.D., Ph.D.^15*, Wadih Abou Chahla, M.D.^16*, Thierry Lamy, PhD, MD^17,18,19, Aline Moignet Autrel, MD^20*, Claire Fieschi, MD, PhD^21*, Thierry Leblanc, M.D.^22,23*, Despina Moshous, MD, PhD^24,25*, Jean Soulier, MD, PhD^26,27, Sarah cohen Beaussant^28*, Francois Delhommeau, MD, PhD^29*, Helene Cavé, PharmD, PhD^30*, Catherine Paillard, MD, PhD^31*, Elodie Gouache, MD^32*, Fares bou Mitri^28*, Damien Bonnet^33*, Laurence Olivier Faivre^34*, Philippe Labrune, MD, PhD^35*, Marlène Pasquet, M.D., Ph.D.^36*, Virginie Gandemer, MD, PhD^37,38*, Nizar Mahlaoui, MD^39*, Karl Balabanian^40*, Francoise Bachelerie, PhD^41* and Christine Bellanne-Chantelot, PhD, PharmD^42,43*

¹Service d'Hémato-Oncologie Pédiatrique, Hopital Trousseau, Paris, Cedex, France
²Registre des Neutropénies, Hopital Trousseau, AP-HP, Paris, France
³Pediatric Hematology and Oncology Department, Hôpital Armand Trousseau, Paris, France
⁴Laboratoire d’Hématologie, APHP, Hôpital Robert Debré,, Paris, France
⁵Hematology and transplantation unit, Saint-Louis Hospital, Paris, France
⁶Hématologie greffe, Hôpital Saint-Louis, Paris, France
⁷Centre de Référence Aplasie Médullaire, Service d’Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France
⁸St-Louis Hospital, APHP, Bone Marrow Transplantation Unit, Paris, France
⁹Bone-marrow transplantation department, CHU Saint-Louis, Paris, France
¹⁰Transplantation Unit, Saint Louis Hospital, APHP, University Paris VII, Paris, France
¹¹Institute of Pediatric Hematology and Oncology, Civil Hospital of Lyon, Claude Bernard University, Lyon, France
¹²Claude Bernard University, Lyon 1, Lyon, France
¹³Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux, France
¹⁴Pediatric Oncology Hematology Unit, Plurithématique CIC (CICP), Centre d’Investigation Clinique (CIC) 1401, INSERM, Bordeaux University Hospital, Bordeaux, France
¹⁵Department of Pediatric Hematology, La Timone Hospital, AP-HM, Marseille, France
¹⁶Department of Pediatric Hematology, Jeanne de Flandre Hospital, CHRU de Lille, Lille, France
¹⁷UMR U1236, Rennes, France
¹⁸INSERM CIC 1414, Hospital Pontchaillou, Rennes, France
¹⁹Department of Hematology, Rennes University Hospital, Rennes, France
²⁰Clinical Hematology Department, Rennes CHU, Rennes, France
²¹Service d’Immunologie clinique, Hôpital Saint-Louis, AP-HP, Paris, France
²²Pediatric Hematology Unit, Robert-Debré Hospital, AP-HP, Paris, France
²³French Reference Center for Aplastic Anemia, Paris, France
²⁴Department of Pediatric Immunology, Hematology and Rheumatology, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
²⁵INSERM UMR 1163, Laboratory of Genome Dynamics in the Immune System, Equipe Labellisée Ligue contre le Cancer, Paris, France
²⁶INSERM U944, Institut Universitaire d'Hématologie, Hopital Saint-Louis, Paris, France
²⁷Hematology Laboratory and INSERM U944, Hopital Saint-Louis Centre Hayem 2ETG, Paris, France
²⁸Centre de référence des neutropénies chroniques Registre des neutropenies, Hopital Trousseau, Paris, France
²⁹APHP, Hôpital Saint Antoine, Service d'hématologie Biologique, Paris, France
³⁰UMRS 1131, Institut de Recherche Saint-Louis, Inserm, University of Paris, Paris, France
³¹Pediatric Hematology Department, Strasbourg University Hospital, Strasbourg, France
³²Pediatric hematology department, Trousseau hospital, APHP, Paris, France
³³Centre de Référence Malformations Cardiaques Congénitales Complexes - M3C, Necker Enfants Malades Université de Paris, Paris, FRA
³⁴Centre de Génétique et Centre de Référence Anomalies du Développement et Syndromes Malformatifs, FHU TRANSLAD, Hôpital d'Enfants, Dijon, France, CHU Dijon, DIJON, France
³⁵Service de Pédiatrie Centre de référence des glycogénoses, APHP Hopital Beclere, CLAMART, France
³⁶Pediatric Oncology Immunology Hematology Unit, Children’s hospital, CHU de Toulouse, Toulouse, France
³⁷CHU Hopital Sud, Rennes, FRA
³⁸CNRS UMR 6290 Institut de Génétique et Développement de Rennes, Rennes, France
³⁹CEREDIH, Grp Hosp. Necker-Enfants Malades, Paris, France
⁴⁰Université de Paris, Saint Louis Research Institute, EMiLy, Inserm U1160, Paris, FRA
⁴¹Immunologie 92296 Châtenay-Malabry, Université Paris Saclay Faculte de Pharmacie Inserm U996, Clamart, FRA
⁴²Hopital Pitié Salpétriére APHP, Département de Génétique, AP-HP Hôpital Pitié- Salpêtrière, UPMC Univ Paris 06, Paris, France
⁴³Département de Neurologie, Hôpital Pitié-Salpêtrière, Paris, France

Yanyan Wang^1*, Christian Rohde, PhD^1*, Fengbiao Zhou, PhD^1*, Marco Hennrich, PhD^2*, Laura Poisa-Beiro, PhD^1*, Volker Eckstein, PhD^1*, Caroline Pabst, MD^1*, Anne-Claude Gavin, PhD^3,4*, Anthony D Ho, MD¹ and Carsten Müller-Tidow, MD^1*

¹Department of Internal Medicine V, University Hospital, Heidelberg University, Heidelberg, Germany
²European Molecular Biology Laboratory, Heidelberg, Germany
³Structural and Computational Biology Unit, EMBL, Heidelberg, Germany
⁴Molecular Medicine Partnership Unit(MMPU), Heidelberg, Germany

Xiaoli Liu, postgraduate^*, Dongyue Zhang, postgraduate^*, Hao Wang, Ph.D. candidate^*, Qian Ren^*, Lina Wang, Ph.D^*, Weiping Yuan, PhD, MD and Guoguang Zheng, PhD^*

State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China

Tomasz W. Kaminski, PharmD¹, Tomasz Brzoska, PhD¹, Egemen Tutuncuoglu, MSc^1*, Margaret V. Ragni, MD, MPH^2,3 and Prithu Sundd, PhD^1,4

¹Pittsburgh Heart, Lung and Blood Vascular Medicine Institute, University of Pittsburgh School of Medicine, Pittsburgh, PA
²University of Pittsburgh, Hemophilia Center of Western Pennsylvania, Pittsburgh, PA
³Department of Medicine - Division of Hematology/Oncology, University of Pittsburgh, Pittsburgh, PA
⁴Department of Bioengineering, University of Pittsburgh, Pittsburgh, PA

Moataz Ellithi, MBBCh¹, Hafez M Abdullah, MD^1*, Waqas Ullah, MD^2*, Radowan Elnair, MD³, Fouad Khalil, MD^1* and Faiz Anwer, MD⁴

¹Department of Internal Medicine, University of South Dakota School of Medicine, Sioux Falls, SD
²Department of Internal Medicine, Abington Hospital - Jefferson Health, Abington, PA
³University of Nebraska Medical Center, Omaha, NE
⁴Taussig Cancer Institute, Department of Hematology and Medical Oncology, Cleveland Clinic, Cleveland, OH

Jennifer Croden, MD^1*, Jennifer Grossman, MD, FRCPC² and Haowei Sun, MD, FRCPC¹

¹Department of Medicine, University of Alberta, Edmonton, AB, Canada
²Department of Hematology and Bone Marrow Transplantation, Department of Medicine, University of Calgary, Calgary, AB, Canada

Antonious Hazim^1*, Gordon Ruan, MD¹, Aishwarya Ravindran, MBBS², Jithma P. Abeykoon, MD³, Caleb J Scheckel, DO⁴, Robert Vassallo, MD^3*, Jay Ryu, MD^3*, W. Oliver Tobin, MB BCh^3*, Matthew Koster, MD^3*, Nabila Nora Bennani, MD⁵, Karen Rech, MD^2*, Jason R Young, MD^3*, Mithun Vinod Shah, MD, PhD⁶, Gaurav Goyal, MD^6,7 and Ronald S. Go, MD⁶

¹Division of Internal Medicine, Mayo Clinic, Rochester, MN
²Division of Hematopathology, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN
³Mayo Clinic, Rochester, MN
⁴Division of Hematology, Department of Medicine, Mayo Clinic, Scottsdale, AZ
⁵Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN
⁶Division of Hematology, Mayo Clinic, Rochester, MN
⁷University of Alabama at Birmingham, Birmingham, AL