Natasja Nielsen Viller^1,2*, Saman Maleki Vareki^2,3*, Karen Dodge^1*, Hui Chen^1*, Vivian Lee^1*, Vien Chai^1*, Xinli Pang^1*, Mark Wong^1*, Suzanne Trudel, MD⁴, Rene Figueredo^2,3*, Macarena Pampillo^2*, James Koropatnick^2,3*, Penka S. Petrova^1* and Robert A. Uger^1*

¹Trillium Therapeutics Inc., Toronto, ON, Canada
²London Regional Cancer Program, London Health Sciences Centre, Lawson Health Research Institute, London, ON, Canada
³Department of Oncology, University of Western Ontario, London, ON, Canada
⁴Department of Medical Oncology and Hematology, Princess Margaret Cancer Centre, Toronto, ON, Canada

Jeremy Lagrange, PhD^1*, Sabine Kossmann, PhD^1,2*, Jäckel Sven, DVM^1*, Jurk Kerstin, PhD^1*, Moritz Ehlken, MD^1,2*, Tanja Schönfelder, DVM^1*, Maike Knorr, MD^1,2*, Moritz Brandt, MD^1,2*, Susanne Karbach, MD^1,2*, Andreas Daiber, PhD^2*, Matthias Oelze, PhD^2*, Christoph Reinhardt, PhD^1*, Karl Lackner, MD^3*, Brett P. Monia, PhD⁴, Ulrich Walter, MD^1*, Zaverio M. Ruggeri, MD⁵, Thomas Renné, MD^6,7*, Wolfram Ruf, MD^1,8, Thomas Münzel, MD^1,2* and Philip Wenzel, MD^1,2*

¹Center for Thrombosis and Hemostasis, University Medical Center, Mainz, Germany
²Department of Medicine 2, University Medical Center, Mainz, Germany
³Institute of Clinical Chemistry and Laboratory Medicine, University Medical Center, Mainz, Germany
⁴Isis Pharmaceuticals, Inc., Carlsbad, CA
⁵Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, CA
⁶Department of Molecular Medicine and Surgery, Clinical Chemistry, Karolinska Institutet, Stockholm, Sweden
⁷Department of Clinical Chemistry/Central Laboratories, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
⁸Immunology and Microbial Science, The Scripps Research Institute, La Jolla, CA

Satoshi Saito, MD^1*, Shiho Nishimura, MD^1*, Miyuki Tsumura, PhD^1*, Yoko Mizoguchi, MD, PhD^1*, Sonoko Sakata, MD^1*, Aya Furue, MD^1*, Mizuka Miki, MD^1*, Hiroshi Kawaguchi, MD, PhD^1*, Takafumi Hiramoto, PhD^2*, Yasuhiro Ebihara, MD, PhD², Kohichiro Tsuji, MD^2*, Tatsutoshi Nakahata, MD., PhD.³ and Masao Kobayashi, MD, PhD¹

¹Department of Pediatrics, Hiroshima University Graduate School of Biomedical & Health Sciences, Hiroshima, Japan
²Center for Stem Cell Biology and Regenerative Medicine, University of Tokyo, Tokyo, Japan
³Center for iPS Cell Research and Application, Kyoto University, Kyoto, Japan

Olga Klimenkova, PhD^1*, Maksim Klimiankou, PhD^1*, Lothar Kanz, MD², Cornelia Zeidler, MD^3*, Karl Welte, MD^4* and Julia Skokowa, Prof. Dr.^1*

¹Division of Translational Oncology, Department of Oncology, Hematology, Immunology, Rheumatology and Pulmonology, University Hospital Tuebingen, Tuebingen, Germany
²Hematology and Oncology, Eberhard Karls University, Tubingen, Germany
³Pediatric Hematology / Oncology, SCNER, Medical School Hannover, Hannover, Germany
⁴University Children’s Hospital Tuebingen, Tuebingen, Germany

Colin G. Steward, MD, PhD¹, Sarah J. Groves^2*, Carolyn L. Taylor, MD^3*, Melissa K. Maisenbacher, MS, CGC^4*, Birgitta Versluys, MD^5*, Ruth Newbury-Ecob, MD, FRCP^6*, Hulya Ozsahin, MD^7*, Leo Hamilton, MD⁸, Michaela K. Damin, MSc, CGC^9*, Valerie M. Bowen^10*, Katherine R. McCurdy^10*, Michael C. Mackey, PhD, FRSC^11*, Audrey Anna Bolyard, RN, BS^12* and David C. Dale, MD¹³

¹Department of Haemaology, Oncology and BMT, Bristol Royal Hospital for Children, Bristol, United Kingdom
²School of Cellular and Molecular Medicine, University of Bristol, Bristol, United Kingdom
³Department of Pediatrics, Division of Cardiology, Medical University of South Carolina, Charleston, SC
⁴NATERA, San Carlos, CA
⁵Paediatric Blood and Marrow Transplantation Program, University Medical Center Utrecht, Wilhelmina Children's Hospital, Utrecht, Netherlands
⁶Department of Clinical Genetics, St. Michael's Hospital, Bristol, United Kingdom
⁷Departement de Pediatrie, Unite d'Onco-hematologie, Hopitaux Universitaires de Geneve, Geneva, Switzerland
⁸Paediatrician, International Paediatric Clinic, Camden, Singapore
⁹Barth Syndrome Trust, Hampshire, United Kingdom
¹⁰Barth Syndrome Foundation, Inc., Larchmont, NY
¹¹Department of Physiology, McGill University, Montreal, QC, Canada
¹²Department of Medicine, University of Washington, Severe Chronic Neutropenia International Registry, Seattle, WA
¹³Department of Medicine, University of Washington, Seattle, WA

Roberta Chessa, PhD^1*, Ritama Gupta, MSc^2*, Bart J Crielaard, MD, PhD^3*, Carla Casu, PhD^4*, Rick Feldman, PhD^5* and Stefano Rivella, PhD⁶

¹Hematology, Children Hospital of Philadelphia, Philadelphia, PA
²Children's Hospital of Philadelphia, Philadelphia, PA
³Children Hospital of Philadelphia, Philadelphia, PA
⁴Children Hospital of Philadelphia, philadelphia, PA
⁵Bayer, san francisco, CA
⁶Pediatrics/Hematology, Children's Hospital of Philadelphia, Philadelphia, PA

Kandace Gollomp, MD¹, Johnston Ian^2*, Diarra Fatoumata^3*, Guohua Zhao^4*, Sriram Krishnaswamy, PhD. MD^4,5, Mortimer Poncz, MD^6,7 and M. Anna Kowalska, PhD⁸

¹Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Pharmacology, University of Pennsylvania, Philadelphia, PA
³University of Pennsylvania, Philadelphia, PA
⁴Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
⁵The Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁶Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
⁷Department of Pediatrics, University of Pennsylvania, Philadelphia, PA
⁸Children's Hospital of Philadelphia, Philadelphia, PA

Derek NJ Hart^1,2, Pablo Silveira^1*, Tsun Ho Lo^1,3*, Nirupama Verma^1*, Ai Vu^4*, Masato Kato^5* and Georgina Clark^1,2*

¹Dendritic Cell Research Group, ANZAC Research Institute, Concord, Australia
²Sydney Medical School, The University of Sydney, Sydney, Australia
³Sydney Medical School, Sydney, Australia
⁴ANZAC Research Institute, Concord, Australia
⁵Mater Medical Research Institute, South Brisbane, Australia

Bardia Samareh, PhD^1*, Masoud Nasri, M.Sc^1*, Inna Zimmer^2*, Olga Klimenkova, PhD^1*, Leonie Keller^1*, Lothar Kanz, Prof. Dr. med.³, Karl Welte, Prof. Dr. med.^2,4 and Julia Skokowa, Prof. Dr.^1*

¹Division of Translational Oncology, Department of Oncology, Hematology, Immunology, Rheumatology and Pulmonology, University Hospital Tuebingen, Tuebingen, Germany
²Department of Molecular Hematopoiesis, Hannover Medical School, Hannover, Germany
³Department of Oncology, Hematology, Immunology, Rheumatology and Pulmonology, University Hospital Tuebingen, Tuebingen, Germany
⁴Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation, University Hospital Tuebingen, Tuebingen, Germany

Akshay A D'Cruz^1,2, Meghan Bliss-Moreau^1,2, Maria Ericcson^2* and Ben A Croker^1,2

¹Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA
²Harvard Medical School, Boston, MA

Dafna Gilboa^1*, Yasmin Haim-Ohana^1*, Nathali Ben-Califa, PhD^1*, Naamit Deshet-Unger, MS^2*, Sahar Bab-Hiram, PhD^1*, Max Gassmann, DVM^3*, Moshe Mittelman, MD⁴ and Drorit Neumann, PhD²

¹Department of cell and developmental biology, Sackler faculty of medicine Tel Aviv University, Tel Aviv, Israel
²Department of Cell and Developmental Biology, Sackler Faculty of Medicine Tel Aviv University, Tel Aviv, Israel
³Institute of Veterinary Physiology, University of Zurich, Zurich, Switzerland
⁴Tel Aviv Sourasky Medical Center, Tel Aviv, Israel

Corinna Cavan Pedersen, M.Sc. Eng.^1*, Rehannah Borup, M.Sc.^2*, Anne Fischer-Nielsen, MD, PhD^3*, Helena Mora-Jensen, PhD^1*, Anna Fossum, M.Sc.^4*, Jack B. Cowland⁵ and Niels Borregaard⁵

¹Granulocyte Research Laboratory, Copenhagen University Hospital, Copenhagen, Denmark
²Department of Clinical Biochemistry, Copenhagen University Hospital, Copenhagen, Denmark
³Department of Clinical Immunology, Copenhagen University Hospital, Copenhagen, Denmark
⁴Biotech and Research Innovation Centre, University of Copenhagen, Copenhagen, Denmark
⁵The Granulocyte Research Laboratory, Department of Hematology, Copenhagen University Hospital, Copenhagen, Denmark

Heiko Bruns, PhD^1*, Regina Jitschin, PhD, MD^1*, Dimitrios Mougiakakos^2*, Joerg Thomas Bittenbring, MD,^3*, Jens Nolting^4*, Maike Büttner^2*, Michael Rehli, PhD^5*, Julia Wimmer^6*, Frank Neumann, PhD^3*, Christian Bach^2*, Heidi Balzer^2*, Stephanie Moi^7*, Peter Brossart, MD⁸, Armin Gerbitz, MD⁷ and Andreas Mackensen, MD⁹

¹University of Erlangen, Erlangen, Germany
²University Hospital Erlangen, Erlangen, Germany
³Department of Internal Medicine I, Saarland University Medical School, Homburg, Germany
⁴University Hospital Bonn, Bonn, Germany
⁵Internal Medicine III, University Hospital, Regensburg, Germany
⁶University Hospital of Regensburg, Regensburg, Germany
⁷University Erlangen, Erlangen, Germany
⁸Internal Medicine III, Oncology, Hematology and Rheumatology, University Clinic Bonn, Bonn, Germany
⁹Dept. of Internal Medicine 5 - Hematology/Oncology, University of Erlangen-Nuernberg, Erlangen, Germany

Liana M Toia, BS^1*, John Mariano, BS², Michael Weinstein Winter, MD¹ and Jessica C Shand, MD³

¹University of Rochester Medical Center, Rochester, NY
²University of Rochester School of Medicine and Dentistry, Rochester, NY
³Division of Pediatric Hematology-Oncology, University of Rochester, Rochester, NY

Francesca Vinchi, PhD^1,2*, Milene Costa da Silva, PhD student^1,2*, Giada Ingoglia, PhD student^3*, Sara Petrillo, PhD student^3*, Nathan Brinkman^4*, Adrian Zuercher^5*, Adelheid Cerwenka, PhD^6*, Emanuela Tolosano, PhD³ and Martina U. Muckenthaler, PhD^1,2

¹Department of Pediatric Oncology, Hematology, Immunology and Pulmunology, University of Heidelberg, Heidelberg, Germany
²Molecular Medicine Partnership Unit (MMPU), University of Heidelberg and EMBL, Heidelberg, Germany
³Department of Molecular Biotechnology and Health Sciences, Molecular Biotechnology Center, University of Torino, Torino, Italy
⁴CSL Behring, Research & Development, Kanakee, IL
⁵CSL Behring, Research & Development, Bern, Switzerland
⁶German Cancer Research Center (DKFZ), Heidelberg, Germany

Inna Zimmer^1*, Bardia Samareh, PhD^2*, Olga Klimenkova, PhD^2*, Maksim Klimiankou, PhD^2*, Murat Ünalan, PhD^1*, Lothar Kanz, Prof. Dr. med.³, Karl Welte, Prof. Dr. med.⁴ and Julia Skokowa, Prof. Dr.^2*

¹Department of Molecular Hematopoiesis, Hannover Medical School, Hannover, Germany
²Division of Translational Oncology, Department of Oncology, Hematology, Immunology, Rheumatology and Pulmonology, University Hospital Tuebingen, Tuebingen, Germany
³Department of Oncology, Hematology, Immunology, Rheumatology and Pulmonology, University Hospital Tuebingen, Tuebingen, Germany
⁴Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation, University Hospital Tuebingen, Tuebingen, Germany

Jae Min Shin^1*, Young-Jun Kim, Ph.D.^1*, Sun Young Yoon, Ph.D.^2*, Myung-Hwan Kim, MD, Ph.D.^3*, Cheolwon Suh⁴, Ki-Young Sohn^2*, Heung-Jae Kim, Ph.D.^2*, Yong-Hae Han, Ph.D.^2*, Saeho Chong, Ph.D.^2* and Jae Wha Kim, Ph.D.^1*

¹Biomedical Translational Research Center, Korea Research Institute of Bioscience and Biotechnology, Daejeon, South Korea
²Enzychem Lifesciences Corporation, Weehawken, NJ
³Department of Gastroenterology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea
⁴Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea

Wulin Aerbajinai, MD, PhD^1*, Jungsoo Joo^2*, Ming Yan^2*, Chutima Kumkhaek^1* and Griffin P. Rodgers¹

¹Molecular and Clinical Hematology Branch, NHLBI/NIH, Bethesda, MD
²Laboratory of Biochemistry and Genetics, NIDDK/NIH, Bethesda

Deepak Chellapandian, MD, MBBS¹, Polyzois Makras, MD, PhD^2*, Gregory Kaltsas, MD, PhD^3*, Cor van den Bos, MD PhD^4*, Anne-Sophie Carret, MD^5*, Sheila Weitzman, MB BCh, FRCP^6*, Maarten Egeler, MD, PhD^7* and Oussama Abla, MD⁸

¹Pediatric Hematology and Oncology/ University of Toronto, The Hospital For Sick Children, Toronto, ON, Canada
²Department of Endocrinology & Diabetes, Hellenic Airforce & VA General Hospital, Athens, Greece
³Department of Pathophysiology, National University of Athens, Athens, Greece
⁴Department of Pediatric Oncology, Academic Medical Center, Amsterdam, Netherlands
⁵Division of Hematology-Oncology, Department of Pediatrics, Centre Hospitalier Universitaire (CHU) Sainte-Justine, Université de Montréal, Montreal, Canada
⁶Pediatric Hematology-Oncology/ Universiy of Toronto, Hospital For Sick Children, Toronto, ON, Canada
⁷Pediatric Hematology-Oncology and Bone Marrow Transplantation/ Hospital for Sick Children/University of Toronto, Toronto, Canada
⁸Division of Haematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada

Jae Wha Kim, Ph.D.^1*, Yong-Jae Kim^1*, Young-Jun Kim, Ph.D.^1*, Sun Young Yoon, Ph.D.^2*, Jinseon Jeong^1*, Young-Eun Ko^1*, Su-Hyun Shin^1*, Nina Yoo^1*, Myung-Hwan Kim, MD, Ph.D.^3*, Cheolwon Suh, MD, Ph.D.⁴, Ki-Young Sohn^2*, Heung-Jae Kim, Ph.D.^2*, Yong-Hae Han, Ph.D.^2* and Saeho Chong, Ph.D.^2*

¹Biomedical Translational Research Center, Korea Research Institute of Bioscience and Biotechnology, Daejeon, South Korea
²Enzychem Lifesciences Corporation, Weehawken, NJ
³Department of Gastroenterology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea
⁴Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea

Laurence A. Boxer, MD¹, Audrey Anna Bolyard, RN, BS^2*, Tracy M. Marrero^2*, Emily L. Tran, BA^2*, Mary Ann Bonilla, MD³, Peter E. Newburger, MD⁴ and David C. Dale, MD⁵

¹Pediatric Hematology/Oncology, University of Michigan, Ann Arbor, MI
²Department of Medicine, University of Washington, Severe Chronic Neutropenia International Registry, Seattle, WA
³Pediatric Hematology/Oncology, St. Joseph's Children's Hospital, Paterson, NJ
⁴University of Massachusetts Medical School, Worcester, MA
⁵Department of Medicine, University of Washington, Seattle, WA

Su-Hyun Shin^1*, Ha-Reum Lee, Ph.D.^1*, Nina Yoo^1*, Sun Young Yoon, Ph.D.^2*, Myung-Hwan Kim, MD, Ph.D.^3*, Cheolwon Suh⁴, Ki-Young Sohn^2*, Heung-Jae Kim, Ph.D.^2*, Yong-Hae Han, Ph.D.^2*, Saeho Chong, Ph.D.^2* and Jae Wha Kim, Ph.D.^1*

¹Biomedical Translational Research Center, Korea Research Institute of Bioscience and Biotechnology, Daejeon, South Korea
²Enzychem Lifesciences Corporation, Weehawken, NJ
³Department of Gastroenterology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea
⁴Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, South Korea

Stine N. Clemmensen, MD^1*, Sara Heebøll^2*, Claus Koch^3* and Niels Borregaard¹

¹Granulocyte Research Laboratory, State University Hospital, Copenhagen, Denmark
²Department of Hepatology and Gastroenterology, Aarhus University Hospital, Aarhus, Denmark
³Department of Biomedicine, University of Southern Denmark, Odense, Denmark

Cory M Alvey, BS Biology and Chemistry^1*, Kyle Spinler^2*, Manu Tewari, Ph.D Biochemistry^2* and Dennis Discher, Ph.D Cell Membrane Biophysics & Biochemistry^2*

¹University of Pennsylvania, Philadelphia, PA
²University of Pennsylvania, Philadelphia

Burkhard Otremba, MD^1* and Wolfhard Erdlenbruch, MD^2*

¹Oncological practice Oldenburg / Delmenhorst, Oldenburg, Germany
²Hospira Germany, Munich, Germany