1. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey
- Author
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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, 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, 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, Morna J. Dorsey, Christine M. Seroogy, Linda M. Griffith, Donald B. Kohn, Jennifer M. Puck, Michael A. Pulsipher, and Troy R. Torgerson
- Subjects
0301 basic medicine ,hematopoietic cell transplant ,T-Lymphocytes ,Disease ,medicine.disease_cause ,T-Lymphocytes, Regulatory ,Autoimmunity ,0302 clinical medicine ,Surveys and Questionnaires ,Immunology and Allergy ,Medicine ,genetics ,Child ,Original Research ,autoimmunity ,Hematopoietic Stem Cell Transplantation ,Middle Aged ,Regulatory ,3. Good health ,Treatment Outcome ,medicine.anatomical_structure ,Medical Microbiology ,Child, Preschool ,primary immune deficiencies ,Development of treatments and therapeutic interventions ,lcsh:Immunologic diseases. Allergy ,Adult ,Adolescent ,Regulatory T cell ,Primary Immunodeficiency Diseases ,Immunology ,Young Adult ,03 medical and health sciences ,Rare Diseases ,Immune system ,Clinical Research ,immune dysregulation ,Animals ,Humans ,In patient ,Preschool ,Transplantation ,5.2 Cellular and gene therapies ,Hematopoietic cell ,business.industry ,Inflammatory and immune system ,Infant ,Immune dysregulation ,030104 developmental biology ,lcsh:RC581-607 ,business ,030215 immunology - 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. ispartof: FRONTIERS IN IMMUNOLOGY vol:11 ispartof: location:Switzerland status: published
- Published
- 2020
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