Frontiers in Immunology (Feb 2020)
Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey
- Alice Y. Chan,
- Jennifer W. Leiding,
- Xuerong Liu,
- Brent R. Logan,
- Lauri M. Burroughs,
- Eric J. Allenspach,
- Suzanne Skoda-Smith,
- Gulbu Uzel,
- Luigi D. Notarangelo,
- Mary Slatter,
- Andrew R. Gennery,
- Angela R. Smith,
- Sung-Yun Pai,
- Sung-Yun Pai,
- Sung-Yun Pai,
- Michael B. Jordan,
- Rebecca A. Marsh,
- Morton J. Cowan,
- Christopher C. Dvorak,
- John A. Craddock,
- Susan E. Prockop,
- Shanmuganathan Chandrakasan,
- Neena Kapoor,
- Rebecca H. Buckley,
- Suhag Parikh,
- Deepak Chellapandian,
- Benjamin R. Oshrine,
- Jeffrey J. Bednarski,
- Megan A. Cooper,
- Shalini Shenoy,
- Blachy J. Davila Saldana,
- Lisa R. Forbes,
- Caridad Martinez,
- Elie Haddad,
- David C. Shyr,
- Karin Chen,
- Kathleen E. Sullivan,
- Jennifer Heimall,
- Nicola Wright,
- Monica Bhatia,
- Geoffrey D. E. Cuvelier,
- Frederick D. Goldman,
- Isabelle Meyts,
- Isabelle Meyts,
- Holly K. Miller,
- Markus G. Seidel,
- Mark T. Vander Lugt,
- Rosa Bacchetta,
- Katja G. Weinacht,
- Jeffrey R. Andolina,
- Emi Caywood,
- Hey Chong,
- Maria Teresa de la Morena,
- Victor M. Aquino,
- Evan Shereck,
- Jolan E. Walter,
- Jolan E. Walter,
- Jolan E. Walter,
- Morna J. Dorsey,
- Christine M. Seroogy,
- Linda M. Griffith,
- Donald B. Kohn,
- Jennifer M. Puck,
- Michael A. Pulsipher,
- Troy R. Torgerson
Affiliations
- Alice Y. Chan
- Division of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States
- Jennifer W. Leiding
- Department of Pediatrics, Johns Hopkins All Children's Hospital, University of South Florida, St. Petersburg, FL, United States
- Xuerong Liu
- Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, United States
- Brent R. Logan
- Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, United States
- Lauri M. Burroughs
- Department of Pediatrics, Fred Hutchinson Cancer Research Center, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States
- Eric J. Allenspach
- Department of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States
- Suzanne Skoda-Smith
- Department of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States
- Gulbu Uzel
- Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States
- Luigi D. Notarangelo
- Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States
- Mary Slatter
- Primary Immunodeficiency Group, Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Translational and Clinical Research Institute, Great North Childrens' Hospital, Newcastle University, Newcastle upon Tyne, United Kingdom
- Andrew R. Gennery
- Primary Immunodeficiency Group, Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Translational and Clinical Research Institute, Great North Childrens' Hospital, Newcastle University, Newcastle upon Tyne, United Kingdom
- Angela R. Smith
- Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, MN, United States
- Sung-Yun Pai
- Division of Pediatric Hematology-Oncology, Boston Children's Hospital, Boston, MA, United States
- Sung-Yun Pai
- 0Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, United States
- Sung-Yun Pai
- 1Department of Pediatrics, Harvard Medical School, Boston, MA, United States
- Michael B. Jordan
- 2Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, United States
- Rebecca A. Marsh
- 2Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, United States
- Morton J. Cowan
- Division of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States
- Christopher C. Dvorak
- Division of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States
- John A. Craddock
- 3Texas Children's Cancer Center, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, United States
- Susan E. Prockop
- 4Stem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, United States
- Shanmuganathan Chandrakasan
- 5Division of Bone Marrow Transplant, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, United States
- Neena Kapoor
- 6Section of Transplantation and Cellular Therapy, Cancer and Blood Disease Institute, Keck School of Medicine, Children's Hospital Los Angeles, University of Southern California, Los Angeles, CA, United States
- Rebecca H. Buckley
- 7Departments of Pediatrics and Immunology, Duke University School of Medicine, Durham, NC, United States
- Suhag Parikh
- 7Departments of Pediatrics and Immunology, Duke University School of Medicine, Durham, NC, United States
- Deepak Chellapandian
- 8Cancer and Blood Disorders Institute, Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital, St. Petersburg, FL, United States
- Benjamin R. Oshrine
- 8Cancer and Blood Disorders Institute, Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital, St. Petersburg, FL, United States
- Jeffrey J. Bednarski
- 9Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States
- Megan A. Cooper
- 9Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States
- Shalini Shenoy
- 9Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States
- Blachy J. Davila Saldana
- 0Division of Blood and Marrow Transplantation, Children's National Health System, George Washington University School of Medicine and Health Sciences, Washington, DC, United States
- Lisa R. Forbes
- 1Department of Pediatrics, Immunology, Allergy, and Retrovirology Baylor College of Medicine, Texas Children's Hospital William T. Shearer Center for Human Immunobiology, Houston, TX, United States
- Caridad Martinez
- 2Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital Cancer Center, Houston, TX, United States
- Elie Haddad
- 3Department of Pediatrics, University of Montreal, Montreal, QC, Canada
- David C. Shyr
- 4Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, United States
- Karin Chen
- 4Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, United States
- Kathleen E. Sullivan
- 5Children's Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA, United States
- Jennifer Heimall
- 5Children's Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA, United States
- Nicola Wright
- 6Department of Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada
- Monica Bhatia
- 7Pediatric Stem Cell Transplantation, Columbia University College of Physicians and Surgeons, New York, NY, United States
- Geoffrey D. E. Cuvelier
- 8Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada
- Frederick D. Goldman
- 9Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL, United States
- Isabelle Meyts
- 0Laboratory of Inborn Errors of Immunity, Department of Immunology, Microbiology and Transplantation, KU Leuven, Leuven, Belgium
- Isabelle Meyts
- 1Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium
- Holly K. Miller
- 2Phoenix Children's Hospital, Phoenix, AZ, United States
- Markus G. Seidel
- 3Research Unit for Pediatric Hematology and Immunology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Graz, Austria
- Mark T. Vander Lugt
- 4Department of Pediatrics, University of Michigan, Ann Arbor, MI, United States
- Rosa Bacchetta
- 5Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, CA, United States
- Katja G. Weinacht
- 5Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, CA, United States
- Jeffrey R. Andolina
- 6Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, United States
- Emi Caywood
- 7Nemours/Alfred I duPont Hospital for Children, Wilmington, DE, United States
- Hey Chong
- 8UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, United States
- Maria Teresa de la Morena
- Department of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States
- Victor M. Aquino
- 9Department of Pediatrics, University of Texas Southwestern Medical Center Dallas, Dallas, TX, United States
- Evan Shereck
- 0Department of Pediatrics, Oregon Health & Science University, Portland, OR, United States
- Jolan E. Walter
- 1Division of Allergy and Immunology, Department of Pediatrics, Morsani College of Medicine, University of South Florida, St. Petersburg, FL, United States
- Jolan E. Walter
- 2Division of Allergy and Immunology, Department of Pediatrics, Johns Hopkins All Children's Hospital, St. Petersburg, FL, United States
- Jolan E. Walter
- 3Division of Allergy and Immunology, Department of Pediatrics, Massachusetts General Hospital for Children, Boston, MA, United States
- Morna J. Dorsey
- Division of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States
- Christine M. Seroogy
- 4Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, United States
- Linda M. Griffith
- 5Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States
- Donald B. Kohn
- 6Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, CA, United States
- Jennifer M. Puck
- Division of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States
- Michael A. Pulsipher
- 6Section of Transplantation and Cellular Therapy, Cancer and Blood Disease Institute, Keck School of Medicine, Children's Hospital Los Angeles, University of Southern California, Los Angeles, CA, United States
- Troy R. Torgerson
- 7Allen Institute for Immunology and Department of Pediatrics, University of Washington, Seattle, WA, United States
- DOI
- https://doi.org/10.3389/fimmu.2020.00239
- Journal volume & issue
-
Vol. 11
Abstract
Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patients have previously focused on a single gene defect. This study surveyed transplanted patients with a phenotypic clinical picture consistent with PIRD treated in 33 Primary Immune Deficiency Treatment Consortium centers and European centers. Our data showed that PIRD patients often had immunodeficient and autoimmune features affecting multiple organ systems. Transplantation resulted in resolution of disease manifestations in more than half of the patients with an overall 5-years survival of 67%. This study, the first to encompass disorders across the PIRD spectrum, highlights the need for further research in PIRD management.
Keywords
- primary immune deficiencies
- autoimmunity
- immune dysregulation
- hematopoietic cell transplant
- genetics
