Pathogenicity and impact of HLA class I alleles in aplastic anemia patients of different ethnicities

Research ArticleHematology Open Access | 10.1172/jci.insight.163040

Timothy S. Olson,1,2 Benjamin F. Frost,2,3 Jamie L. Duke,4 Marian Dribus,5 Hongbo M. Xie,6 Zachary D. Prudowsky,7,8 Elissa Furutani,9 Jonas Gudera,9,10 Yash B. Shah,1,2,11 Deborah Ferriola,4 Amalia Dinou,4 Ioanna Pagkrati,4 Soyoung Kim,12,13 Yixi Xu,12 Meilun He,14 Shannon Zheng,3 Sally Nijim,3 Ping Lin,3 Chong Xu,15,16 Taizo A. Nakano,17 Joseph H. Oved,1,18 Beatriz M. Carreno,15,16 Yung-Tsi Bolon,14 Shahinaz M. Gadalla,19 Steven G.E. Marsh,20 Sophie Paczesny,21 Stephanie J. Lee,12,22 Dimitrios S. Monos,4,23 Akiko Shimamura,9 Alison A. Bertuch,7,8 Loren Gragert,5 Stephen R. Spellman,14 and Daria V. Babushok1,3

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Olson, T. in: JCI | PubMed | Google Scholar |

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Frost, B. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Duke, J. in: JCI | PubMed | Google Scholar |

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Dribus, M. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Xie, H. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Prudowsky, Z. in: JCI | PubMed | Google Scholar |

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Furutani, E. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Gudera, J. in: JCI | PubMed | Google Scholar |

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Shah, Y. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Ferriola, D. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Dinou, A. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Pagkrati, I. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

16Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

17Center for Cancer and Blood Disorders, Children’s Hospital Colorado, Aurora, Colorado, USA.

18Department of Pediatric Transplant and Cell Therapy, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

19Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, NIH, Rockville, Maryland, USA.

20Anthony Nolan Research Institute and University College London Cancer Institute, Royal Free Campus, London, United Kingdom.

21Department of Microbiology and Immunology, Medical University of South Carolina, Charleston, South Carolina, USA.

22Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

23Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Address correspondence to: Daria Babushok, BRB II/III Room 808, 421 Curie Blvd., Philadelphia, Pennsylvania 19104, USA. Phone: 215.573.0667; Email: daria.babushok@pennmedicine.upenn.edu.

Authorship note: TSO and BFF contributed equally to this work.

Find articles by Kim, S. in: JCI | PubMed | Google Scholar

1Comprehensive Bone Marrow Failure Center and

2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

3Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

4Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

5Department of Pathology and Laboratory Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA.

6Department of Biomedical and Health Informatics, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

7Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.

8Texas Children’s Cancer and Hematology Center, Houston, Texas, USA.

9Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.

10Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Klinikum Munich, Munich, Germany.

11Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

12Center for International Blood and Marrow Transplant Research and

13Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

14Center for International Blood and Marrow Transplant Research, National Marrow Donor Program/Be The Match, Minneapolis, Minneapolis, USA.

15Department of Pathology and Laboratory Medicine,

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