TY - JOUR
T1 - Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD)
T2 - A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey
AU - Chan, Alice Y.
AU - Leiding, Jennifer W.
AU - Liu, Xuerong
AU - Logan, Brent R.
AU - Burroughs, Lauri M.
AU - Allenspach, Eric J.
AU - Skoda-Smith, Suzanne
AU - Uzel, Gulbu
AU - Notarangelo, Luigi D.
AU - Slatter, Mary
AU - Gennery, Andrew R.
AU - Smith, Angela R.
AU - Pai, Sung Yun
AU - Jordan, Michael B.
AU - Marsh, Rebecca A.
AU - Cowan, Morton J.
AU - Dvorak, Christopher C.
AU - Craddock, John A.
AU - Prockop, Susan E.
AU - Chandrakasan, Shanmuganathan
AU - Kapoor, Neena
AU - Buckley, Rebecca H.
AU - Parikh, Suhag
AU - Chellapandian, Deepak
AU - Oshrine, Benjamin R.
AU - Bednarski, Jeffrey J.
AU - Cooper, Megan A.
AU - Shenoy, Shalini
AU - Davila Saldana, Blachy J.
AU - Forbes, Lisa R.
AU - Martinez, Caridad
AU - Haddad, Elie
AU - Shyr, David C.
AU - Chen, Karin
AU - Sullivan, Kathleen E.
AU - Heimall, Jennifer
AU - Wright, Nicola
AU - Bhatia, Monica
AU - Cuvelier, Geoffrey D.E.
AU - Goldman, Frederick D.
AU - Meyts, Isabelle
AU - Miller, Holly K.
AU - Seidel, Markus G.
AU - Vander Lugt, Mark T.
AU - Bacchetta, Rosa
AU - Weinacht, Katja G.
AU - Andolina, Jeffrey R.
AU - Caywood, Emi
AU - Chong, Hey
AU - de la Morena, Maria Teresa
AU - Aquino, Victor M.
AU - Shereck, Evan
AU - Walter, Jolan E.
AU - Dorsey, Morna J.
AU - Seroogy, Christine M.
AU - Griffith, Linda M.
AU - Kohn, Donald B.
AU - Puck, Jennifer M.
AU - Pulsipher, Michael A.
AU - Torgerson, Troy R.
N1 - Funding Information:
We would like to thank all research study coordinators in the PIDTC. We would also like to thank Tara Bani, Catherine Chang, and Elizabeth Dunn in the PIDTC for their administrative help in contacting sites and organizing meetings. Funding. This work was supported by the Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID); and the Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Bethesda, MD; Public Health Service grant/cooperative agreements U54-AI082973 (PIs: MJC; September 2019 forward JP and DK); U54-NS064808 and U01-TR001263 (PI: JK) and R13-AI094943 (PIs: MJC; March 2018 forward JP); and the Division of Intramural Research, NIAID, NIH. LN was supported by the Division of Intramural Research, NIAID, NIH. The PIDTC is a part of the Rare Diseases Clinical Research Network (RDCRN) of ORDR, NCATS. Collaborative work of the PIDTC with the Pediatric Blood and Marrow Transplant Consortium (PBMTC) was supported by the U54 grants above along with support of the PBMTC Operations Center by the St. Baldrick's Foundation and grant/cooperative agreement U10HL069254 (PI: MP) from the National Heart, Lung and Blood Institute (NHLBI), and the NIH. Collaborative work of the PIDTC with the Center for International Blood and Marrow Transplant Research (CIBMTR) was supported by grant/cooperative agreement U24-CA76518 (PI: MH) from the National Cancer Institute (NCI), NHLBI, and NIAID, NIH; and grant/cooperative agreement U01HL069294 from the NHLBI and NCI; contract HHSH250201200016C and HHSH234200637015C with the Health Resources and Services Administration (HRSA/DHHS); and grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research. The content and opinions expressed are solely the responsibility of the authors and do not represent the official policy or position of the NIAID, ORDR, NCATS, NIH, HRSA, or any other agency of the US Government.
Publisher Copyright:
© Copyright © 2020 Chan, Leiding, Liu, Logan, Burroughs, Allenspach, Skoda-Smith, Uzel, Notarangelo, Slatter, Gennery, Smith, Pai, Jordan, Marsh, Cowan, Dvorak, Craddock, Prockop, Chandrakasan, Kapoor, Buckley, Parikh, Chellapandian, Oshrine, Bednarski, Cooper, Shenoy, Davila Saldana, Forbes, Martinez, Haddad, Shyr, Chen, Sullivan, Heimall, Wright, Bhatia, Cuvelier, Goldman, Meyts, Miller, Seidel, Vander Lugt, Bacchetta, Weinacht, Andolina, Caywood, Chong, de la Morena, Aquino, Shereck, Walter, Dorsey, Seroogy, Griffith, Kohn, Puck, Pulsipher and Torgerson.
PY - 2020/2/21
Y1 - 2020/2/21
N2 - 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.
AB - 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.
KW - autoimmunity
KW - genetics
KW - hematopoietic cell transplant
KW - immune dysregulation
KW - primary immune deficiencies
UR - http://www.scopus.com/inward/record.url?scp=85081208356&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85081208356&partnerID=8YFLogxK
U2 - 10.3389/fimmu.2020.00239
DO - 10.3389/fimmu.2020.00239
M3 - Article
C2 - 32153572
AN - SCOPUS:85081208356
SN - 1664-3224
VL - 11
JO - Frontiers in Immunology
JF - Frontiers in Immunology
M1 - 239
ER -