Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey

被引：53

作者：

Chan, Alice Y. ^{[1
]}

Leiding, Jennifer W. ^{[2
]}

Liu, Xuerong ^{[3
]}

Logan, Brent R. ^{[3
]}

Burroughs, Lauri M. ^{[4
]}

Allenspach, Eric J. ^{[5
]}

Skoda-Smith, Suzanne ^{[5
]}

Uzel, Gulbu ^{[6
]}

Notarangelo, Luigi D. ^{[6
]}

Slatter, Mary ^{[7
]}

Gennery, Andrew R. ^{[7
]}

Smith, Angela R. ^{[8
]}

Pai, Sung-Yun ^{[9
,10
,11
]}

Jordan, Michael B. ^{[12
]}

Marsh, Rebecca A. ^{[12
]}

Cowan, Morton J. ^{[1
]}

Dvorak, Christopher C. ^{[1
]}

Craddock, John A. ^{[13
]}

Prockop, Susan E. ^{[14
]}

Chandrakasan, Shanmuganathan ^{[15
]}

Kapoor, Neena ^{[16
]}

Buckley, Rebecca H. ^{[17
,18
]}

Parikh, Suhag ^{[17
,18
]}

Chellapandian, Deepak ^{[19
]}

Oshrine, Benjamin R. ^{[19
]}

Bednarski, Jeffrey J. ^{[20
]}

Cooper, Megan A. ^{[20
]}

Shenoy, Shalini ^{[20
]}

Saldana, Blachy J. Davila ^{[21
]}

Forbes, Lisa R. ^{[22
]}

Martinez, Caridad ^{[23
]}

Haddad, Elie ^{[24
]}

Shyr, David C. ^{[25
]}

Chen, Karin ^{[25
]}

Sullivan, Kathleen E. ^{[26
]}

Heimall, Jennifer ^{[26
]}

Wright, Nicola ^{[27
]}

Bhatia, Monica ^{[28
]}

Cuvelier, Geoffrey D. E. ^{[29
]}

Goldman, Frederick D. ^{[30
]}

Meyts, Isabelle ^{[31
,32
]}

Miller, Holly K. ^{[33
]}

Seidel, Markus G. ^{[34
]}

Vander Lugt, Mark T. ^{[35
]}

Bacchetta, Rosa ^{[36
]}

Weinacht, Katja G. ^{[36
]}

Andolina, Jeffrey R. ^{[37
]}

Caywood, Emi ^{[38
]}

Chong, Hey ^{[39
]}

de la Morena, Maria Teresa ^{[5
]}

机构：

[1] Univ Calif San Francisco, Benioff Childrens Hosp, Div Pediat Allergy, BMT,Immunol, San Francisco, CA 94143 USA

[2] Univ S Florida, Johns Hopkins All Childrens Hosp, Dept Pediat, St Petersburg, FL USA

[3] Med Coll Wisconsin, Div Biostat, Milwaukee, WI 53226 USA

[4] Univ Washington, Fred Hutchinson Canc Res Ctr, Seattle Childrens Hosp, Dept Pediat,Sch Med, Seattle, WA 98195 USA

[5] Univ Washington, Sch Med, Dept Pediat, Seattle Childrens Hosp, Seattle, WA 98195 USA

[6] NIAID, Lab Clin Infect Dis, NIH, 9000 Rockville Pike, Bethesda, MD 20892 USA

[7] Newcastle Univ, Great North Childrens Hosp, Translat & Clin Res Inst, Primary Immunodeficiency Grp,Paediat Immunol & Ha, Newcastle Upon Tyne, Tyne & Wear, England

[8] Univ Minnesota, Pediat Blood & Marrow Transplant, Minneapolis, MN USA

[9] Boston Childrens Hosp, Div Pediat Hematol Oncol, Boston, MA USA

[10] Dana Farber Canc Inst, Dept Pediat Oncol, Boston, MA 02115 USA

[11] Harvard Med Sch, Dept Pediat, Boston, MA 02115 USA

[12] Univ Cincinnati, Dept Pediat, Cincinnati Childrens Hosp Med Ctr, Div Bone Marrow Transplantat & Immune Deficiency, Cincinnati, OH USA

[13] Baylor Coll Med, Texas Childrens Canc Ctr, Ctr Cell & Gene Therapy, Houston, TX 77030 USA

[14] Mem Sloan Kettering Canc Ctr, Dept Pediat, Stem Cell Transplant & Cellular Therapy Serv, 1275 York Ave, New York, NY 10021 USA

[15] Emory Univ, Sch Med, Div Bone Marrow Transplant, Childrens Healthcare Atlanta,Aflac Canc & Blood D, Atlanta, GA USA

[16] Univ Southern Calif, Childrens Hosp Los Angeles, Keck Sch Med, Sect Transplantat & Cellular Therapy,Canc & Blood, Los Angeles, CA 90007 USA

[17] Duke Univ, Sch Med, Dept Pediat, Durham, NC USA

[18] Duke Univ, Sch Med, Dept Immunol, Durham, NC USA

[19] Johns Hopkins All Childrens Hosp, Canc & Blood Disorders Inst, Blood & Marrow Transplant Program, St Petersburg, FL USA

[20] Washington Univ, Sch Med, Dept Pediat, St Louis, MO 63110 USA

[21] George Washington Univ, Sch Med & Hlth Sci, Childrens Natl Hlth Syst, Div Blood & Marrow Transplantat, Washington, DC 20052 USA

[22] Texas Childrens Hosp William T Shearer Ctr Human, Baylor Coll Med, Dept Pediat Immunol Allergy & Retrovirolocgy, Houston, TX USA

[23] Baylor Coll Med, Texas Childrens Hosp Canc Ctr, Ctr Cell & Gene Therapy, Houston, TX 77030 USA

[24] Univ Montreal, Dept Pediat, Montreal, PQ, Canada

[25] Univ Utah, Sch Med, Dept Pediat, Salt Lake City, UT USA

[26] Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USA

[27] Univ Calgary, Alberta Childrens Hosp, Dept Pediat, Calgary, AB, Canada

[28] Columbia Univ Coll Phys & Surg, Pediat Stem Cell Transplantat, 630 W 168th St, New York, NY 10032 USA

[29] Univ Manitoba, Canc Care Manitoba, Manitoba Blood & Marrow Transplant Program, Winnipeg, MB, Canada

[30] Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USA

[31] Katholieke Univ Leuven, Lab Inborn Errors Immun, Dept Immunol Microbiol & Transplantat, Leuven, Belgium

[32] Univ Hosp Leuven, Dept Pediat, Leuven, Belgium

[33] Phoenix Childrens Hosp, Phoenix, AZ USA

[34] Med Univ Graz, Dept Pediat & Adolescent Med, Res Unit Pediat Hematol & Immunol, Graz, Austria

[35] Univ Michigan, Dept Pediat, Ann Arbor, MI 48109 USA

[36] Stanford Sch Med, Dept Pediat, Div Stem Cell Transplantat & Regenerat Med, Stanford, CA 94305 USA

[37] Univ Rochester, Med Ctr, Dept Pediat, Golisano Childrens Hosp, Rochester, NY 14642 USA

[38] Nemours Alfred I DuPont Hosp Children, Wilmington, DE USA

[39] UPMC Childrens Hosp Pittsburgh, Pittsburgh, PA USA

[40] Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, Dallas, TX USA

[41] Oregon Hlth & Sci Univ, Dept Pediat, 3181 Sw Sam Jackson Pk Rd, Portland, OR 97201 USA

[42] Univ S Florida, Div Allergy & Immunol, Morsani Coll Med, Dept Pediat, St Petersburg, FL USA

[43] Johns Hopkins All Childrens Hosp, Dept Pediat, Div Allergy & Immunol, St Petersburg, FL USA

[44] Massachusetts Gen Hosp Children, Dept Pediat, Div Allergy & Immunol, Boston, MA USA

[45] Univ Wisconsin, Dept Pediat, Sch Med & Publ Hlth, Madison, WI USA

[46] NIAID, Div Allergy Immunol & Transplantat, NIH, 9000 Rockville Pike, Bethesda, MD 20892 USA

[47] Univ Calif Los Angeles, David Geffen Sch Med, Dept Pediat, Los Angeles, CA 90095 USA

[48] Univ Washington, Allen Inst Immunol, Seattle, WA 98195 USA

[49] Univ Washington, Dept Pediat, Seattle, WA 98195 USA

来源：

FRONTIERS IN IMMUNOLOGY | 2020年 / 11卷

基金：

美国国家卫生研究院;

关键词：

primary immune deficiencies; autoimmunity; immune dysregulation; hematopoietic cell transplant; genetics; C1Q DEFICIENCY;

D O I：

10.3389/fimmu.2020.00239

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

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.

引用

页数：7

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