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 - 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 - https://www.scopus.com/pages/publications/85081208356
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 -