Primary Immune Deficiency Treatment Consortium (PIDTC) report.
Identifieur interne : 001959 ( Main/Exploration ); précédent : 001958; suivant : 001960Primary Immune Deficiency Treatment Consortium (PIDTC) report.
Auteurs : Linda M. Griffith ; Morton J. Cowan ; Luigi D. Notarangelo ; Donald B. Kohn ; Jennifer M. Puck ; Sung-Yun Pai ; Barbara Ballard ; Sarah C. Bauer ; Jack J H. Bleesing [États-Unis] ; Marcia Boyle ; Amy Brower ; Rebecca H. Buckley [États-Unis] ; Mirjam Van Der Burg [Pays-Bas] ; Lauri M. Burroughs ; Fabio Candotti ; Andrew J. Cant [Royaume-Uni] ; Talal Chatila ; Charlotte Cunningham-Rundles [États-Unis] ; Mary C. Dinauer [États-Unis] ; Christopher C. Dvorak ; Alexandra H. Filipovich [États-Unis] ; Thomas A. Fleisher ; Hubert Bobby Gaspar [Royaume-Uni] ; Tayfun Gungor [Suisse] ; Elie Haddad [Canada] ; Emily Hovermale ; Faith Huang [États-Unis] ; Alan Hurley ; Mary Hurley ; Sumathi Iyengar ; Elizabeth M. Kang ; Brent R. Logan ; Janel R. Long-Boyle ; Harry L. Malech ; Sean A. Mcghee ; Fred Modell [États-Unis] ; Vicki Modell [États-Unis] ; Hans D. Ochs ; Richard J. O'Reilly [États-Unis] ; Robertson Parkman ; David J. Rawlings ; John M. Routes ; William T. Shearer ; Trudy N. Small [États-Unis] ; Heather Smith ; Kathleen E. Sullivan ; Paul Szabolcs ; Adrian Thrasher [Royaume-Uni] ; Troy R. Torgerson ; Paul Veys [Royaume-Uni] ; Kenneth Weinberg ; Juan Carlos Zuniga-Pflucker [Canada]Source :
- The Journal of allergy and clinical immunology [ 1097-6825 ] ; 2014.
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Abstract
The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.
DOI: 10.1016/j.jaci.2013.07.052
PubMed: 24139498
Affiliations:
- Canada, Pays-Bas, Royaume-Uni, Suisse, États-Unis
- Angleterre, Canton de Zurich, Caroline du Nord, Grand Londres, Hollande-Méridionale, Missouri (État), Ohio, Ontario, État de New York
- Londres, Rotterdam, Saint-Louis (Missouri), Toronto, Zurich
- University College de Londres, Université de Toronto, Université de Zurich, École de médecine (Université Washington de Saint-Louis)
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Puck</name><affiliation><nlm:affiliation>Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif; Institute for Human Genetics, University of California San Francisco, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Pai, Sung Yun" sort="Pai, Sung Yun" uniqKey="Pai S" first="Sung-Yun" last="Pai">Sung-Yun Pai</name><affiliation><nlm:affiliation>Pediatric Hematology/Oncology, Children's Hospital, Harvard Medical School, Boston, Mass.</nlm:affiliation><wicri:noCountry code="subField">Mass</wicri:noCountry></affiliation></author><author><name sortKey="Ballard, Barbara" sort="Ballard, Barbara" uniqKey="Ballard B" first="Barbara" last="Ballard">Barbara Ballard</name><affiliation><nlm:affiliation>Immune Deficiency Foundation, Towson, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Bauer, Sarah C" sort="Bauer, Sarah C" uniqKey="Bauer S" first="Sarah C" last="Bauer">Sarah C. Bauer</name><affiliation><nlm:affiliation>Developmental and Behavioral Pediatrics, Lurie Children's Hospital of Chicago, Northwestern Feinberg School of Medicine, Chicago, Ill.</nlm:affiliation><wicri:noCountry code="subField">Ill</wicri:noCountry></affiliation></author><author><name sortKey="Bleesing, Jack J H" sort="Bleesing, Jack J H" uniqKey="Bleesing J" first="Jack J H" last="Bleesing">Jack J H. 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Burroughs</name><affiliation><nlm:affiliation>Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="Candotti, Fabio" sort="Candotti, Fabio" uniqKey="Candotti F" first="Fabio" last="Candotti">Fabio Candotti</name><affiliation><nlm:affiliation>Genetics & Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Cant, Andrew J" sort="Cant, Andrew J" uniqKey="Cant A" first="Andrew J" last="Cant">Andrew J. 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Dinauer</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Hematology/Oncology, Washington University School of Medicine, St Louis, Mo.</nlm:affiliation><orgName type="university">École de médecine (Université Washington de Saint-Louis)</orgName><country>États-Unis</country><placeName><settlement type="city">Saint-Louis (Missouri)</settlement><region type="state">Missouri (État)</region></placeName></affiliation></author><author><name sortKey="Dvorak, Christopher C" sort="Dvorak, Christopher C" uniqKey="Dvorak C" first="Christopher C" last="Dvorak">Christopher C. Dvorak</name><affiliation><nlm:affiliation>Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Filipovich, Alexandra H" sort="Filipovich, Alexandra H" uniqKey="Filipovich A" first="Alexandra H" last="Filipovich">Alexandra H. 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Fleisher</name><affiliation><nlm:affiliation>Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Bobby Gaspar, Hubert" sort="Bobby Gaspar, Hubert" uniqKey="Bobby Gaspar H" first="Hubert" last="Bobby Gaspar">Hubert Bobby Gaspar</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region><settlement type="city">Londres</settlement></placeName><orgName type="university">University College de Londres</orgName></affiliation></author><author><name sortKey="Gungor, Tayfun" sort="Gungor, Tayfun" uniqKey="Gungor T" first="Tayfun" last="Gungor">Tayfun Gungor</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Immunology and Blood and Marrow Transplantation, Children's Hospital Zurich, University of Zurich, Zurich, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Pediatric Immunology and Blood and Marrow Transplantation, Children's Hospital Zurich, University of Zurich, Zurich</wicri:regionArea><orgName type="university">Université de Zurich</orgName><placeName><settlement type="city">Zurich</settlement><region nuts="3" type="region">Canton de Zurich</region></placeName></affiliation></author><author><name sortKey="Haddad, Elie" sort="Haddad, Elie" uniqKey="Haddad E" first="Elie" last="Haddad">Elie Haddad</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Pediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec</wicri:regionArea><wicri:noRegion>Quebec</wicri:noRegion></affiliation></author><author><name sortKey="Hovermale, Emily" sort="Hovermale, Emily" uniqKey="Hovermale E" first="Emily" last="Hovermale">Emily Hovermale</name><affiliation><nlm:affiliation>Immune Deficiency Foundation, Towson, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Huang, Faith" sort="Huang, Faith" uniqKey="Huang F" first="Faith" last="Huang">Faith Huang</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Allergy/Immunology, Mount Sinai Medical Center, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatric Allergy/Immunology, Mount Sinai Medical Center, New York</wicri:cityArea></affiliation></author><author><name sortKey="Hurley, Alan" sort="Hurley, Alan" uniqKey="Hurley A" first="Alan" last="Hurley">Alan Hurley</name><affiliation><nlm:affiliation>Chronic Granulomatous Disease Association, San Marino, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Hurley, Mary" sort="Hurley, Mary" uniqKey="Hurley M" first="Mary" last="Hurley">Mary Hurley</name><affiliation><nlm:affiliation>Chronic Granulomatous Disease Association, San Marino, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Iyengar, Sumathi" sort="Iyengar, Sumathi" uniqKey="Iyengar S" first="Sumathi" last="Iyengar">Sumathi Iyengar</name><affiliation><nlm:affiliation>Wiskott-Aldrich Foundation, Smyrna, Ga.</nlm:affiliation><wicri:noCountry code="subField">Ga</wicri:noCountry></affiliation></author><author><name sortKey="Kang, Elizabeth M" sort="Kang, Elizabeth M" uniqKey="Kang E" first="Elizabeth M" last="Kang">Elizabeth M. Kang</name><affiliation><nlm:affiliation>Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Logan, Brent R" sort="Logan, Brent R" uniqKey="Logan B" first="Brent R" last="Logan">Brent R. Logan</name><affiliation><nlm:affiliation>Center for International Blood and Marrow Transplant Research and Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.</nlm:affiliation><wicri:noCountry code="subField">Wis</wicri:noCountry></affiliation></author><author><name sortKey="Long Boyle, Janel R" sort="Long Boyle, Janel R" uniqKey="Long Boyle J" first="Janel R" last="Long-Boyle">Janel R. Long-Boyle</name><affiliation><nlm:affiliation>Department of Clinical Pharmacy, School of Pharmacy, University of California, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Malech, Harry L" sort="Malech, Harry L" uniqKey="Malech H" first="Harry L" last="Malech">Harry L. Malech</name><affiliation><nlm:affiliation>Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Mcghee, Sean A" sort="Mcghee, Sean A" uniqKey="Mcghee S" first="Sean A" last="Mcghee">Sean A. Mcghee</name><affiliation><nlm:affiliation>Pediatric Allergy/Immunology, Lucile Packard Children's Hospital, Stanford University Medical Center, Stanford, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Modell, Fred" sort="Modell, Fred" uniqKey="Modell F" first="Fred" last="Modell">Fred Modell</name><affiliation wicri:level="2"><nlm:affiliation>Jeffrey Modell Foundation, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Jeffrey Modell Foundation, New York</wicri:cityArea></affiliation></author><author><name sortKey="Modell, Vicki" sort="Modell, Vicki" uniqKey="Modell V" first="Vicki" last="Modell">Vicki Modell</name><affiliation wicri:level="2"><nlm:affiliation>Jeffrey Modell Foundation, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Jeffrey Modell Foundation, New York</wicri:cityArea></affiliation></author><author><name sortKey="Ochs, Hans D" sort="Ochs, Hans D" uniqKey="Ochs H" first="Hans D" last="Ochs">Hans D. Ochs</name><affiliation><nlm:affiliation>Center for Immunity and Immunotherapy, Seattle Children's Hospital Research Institute, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="O Reilly, Richard J" sort="O Reilly, Richard J" uniqKey="O Reilly R" first="Richard J" last="O'Reilly">Richard J. O'Reilly</name><affiliation wicri:level="2"><nlm:affiliation>Pediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York</wicri:cityArea></affiliation></author><author><name sortKey="Parkman, Robertson" sort="Parkman, Robertson" uniqKey="Parkman R" first="Robertson" last="Parkman">Robertson Parkman</name><affiliation><nlm:affiliation>Division of Research Immunology/B.M.T., Children's Hospital Los Angeles, Los Angeles, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Rawlings, David J" sort="Rawlings, David J" uniqKey="Rawlings D" first="David J" last="Rawlings">David J. Rawlings</name><affiliation><nlm:affiliation>Pediatric Immunology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="Routes, John M" sort="Routes, John M" uniqKey="Routes J" first="John M" last="Routes">John M. Routes</name><affiliation><nlm:affiliation>Pediatric Allergy and Clinical Immunology, Children's Hospital of Wisconsin, Medical College of Wisconsin, Milwaukee, Wis.</nlm:affiliation><wicri:noCountry code="subField">Wis</wicri:noCountry></affiliation></author><author><name sortKey="Shearer, William T" sort="Shearer, William T" uniqKey="Shearer W" first="William T" last="Shearer">William T. Shearer</name><affiliation><nlm:affiliation>Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex.</nlm:affiliation><wicri:noCountry code="subField">Tex</wicri:noCountry></affiliation></author><author><name sortKey="Small, Trudy N" sort="Small, Trudy N" uniqKey="Small T" first="Trudy N" last="Small">Trudy N. Small</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York</wicri:cityArea></affiliation></author><author><name sortKey="Smith, Heather" sort="Smith, Heather" uniqKey="Smith H" first="Heather" last="Smith">Heather Smith</name><affiliation><nlm:affiliation>SCID Angels for Life Foundation, Lakeland, Fla.</nlm:affiliation><wicri:noCountry code="subField">Fla</wicri:noCountry></affiliation></author><author><name sortKey="Sullivan, Kathleen E" sort="Sullivan, Kathleen E" uniqKey="Sullivan K" first="Kathleen E" last="Sullivan">Kathleen E. Sullivan</name><affiliation><nlm:affiliation>Pediatric Immunology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa.</nlm:affiliation><wicri:noCountry code="subField">Pa</wicri:noCountry></affiliation></author><author><name sortKey="Szabolcs, Paul" sort="Szabolcs, Paul" uniqKey="Szabolcs P" first="Paul" last="Szabolcs">Paul Szabolcs</name><affiliation><nlm:affiliation>Bone Marrow Transplantation and Cellular Therapies, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pa.</nlm:affiliation><wicri:noCountry code="subField">Pa</wicri:noCountry></affiliation></author><author><name sortKey="Thrasher, Adrian" sort="Thrasher, Adrian" uniqKey="Thrasher A" first="Adrian" last="Thrasher">Adrian Thrasher</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region><settlement type="city">Londres</settlement></placeName><orgName type="university">University College de Londres</orgName></affiliation></author><author><name sortKey="Torgerson, Troy R" sort="Torgerson, Troy R" uniqKey="Torgerson T" first="Troy R" last="Torgerson">Troy R. Torgerson</name><affiliation><nlm:affiliation>Pediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name><affiliation wicri:level="3"><nlm:affiliation>Blood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Blood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Weinberg, Kenneth" sort="Weinberg, Kenneth" uniqKey="Weinberg K" first="Kenneth" last="Weinberg">Kenneth Weinberg</name><affiliation><nlm:affiliation>Pediatric Stem Cell Transplantation and Hematology/Oncology, Lucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Zuniga Pflucker, Juan Carlos" sort="Zuniga Pflucker, Juan Carlos" uniqKey="Zuniga Pflucker J" first="Juan Carlos" last="Zuniga-Pflucker">Juan Carlos Zuniga-Pflucker</name><affiliation wicri:level="4"><nlm:affiliation>Department of Immunology, University of Toronto, Toronto, Ontario, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Department of Immunology, University of Toronto, Toronto, Ontario</wicri:regionArea><orgName type="university">Université de Toronto</orgName><placeName><settlement type="city">Toronto</settlement><region type="state">Ontario</region></placeName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2014">2014</date><idno type="RBID">pubmed:24139498</idno><idno type="pmid">24139498</idno><idno type="doi">10.1016/j.jaci.2013.07.052</idno><idno type="wicri:Area/PubMed/Corpus">003D71</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">003D71</idno><idno type="wicri:Area/PubMed/Curation">003D44</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">003D44</idno><idno type="wicri:Area/PubMed/Checkpoint">003D44</idno><idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">003D44</idno><idno type="wicri:Area/Ncbi/Merge">000424</idno><idno type="wicri:Area/Ncbi/Curation">000424</idno><idno type="wicri:Area/Ncbi/Checkpoint">000424</idno><idno type="wicri:Area/Main/Merge">001A18</idno><idno type="wicri:Area/Main/Curation">001959</idno><idno type="wicri:Area/Main/Exploration">001959</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Primary Immune Deficiency Treatment Consortium (PIDTC) report.</title><author><name sortKey="Griffith, Linda M" sort="Griffith, Linda M" uniqKey="Griffith L" first="Linda M" last="Griffith">Linda M. Griffith</name><affiliation><nlm:affiliation>Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md. Electronic address: LGriffith@niaid.nih.gov.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Cowan, Morton J" sort="Cowan, Morton J" uniqKey="Cowan M" first="Morton J" last="Cowan">Morton J. Cowan</name><affiliation><nlm:affiliation>Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Notarangelo, Luigi D" sort="Notarangelo, Luigi D" uniqKey="Notarangelo L" first="Luigi D" last="Notarangelo">Luigi D. Notarangelo</name><affiliation><nlm:affiliation>Division of Immunology, the Manton Center for Orphan Disease Research, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass.</nlm:affiliation><wicri:noCountry code="subField">Mass</wicri:noCountry></affiliation></author><author><name sortKey="Kohn, Donald B" sort="Kohn, Donald B" uniqKey="Kohn D" first="Donald B" last="Kohn">Donald B. Kohn</name><affiliation><nlm:affiliation>Departments of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Puck, Jennifer M" sort="Puck, Jennifer M" uniqKey="Puck J" first="Jennifer M" last="Puck">Jennifer M. Puck</name><affiliation><nlm:affiliation>Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif; Institute for Human Genetics, University of California San Francisco, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Pai, Sung Yun" sort="Pai, Sung Yun" uniqKey="Pai S" first="Sung-Yun" last="Pai">Sung-Yun Pai</name><affiliation><nlm:affiliation>Pediatric Hematology/Oncology, Children's Hospital, Harvard Medical School, Boston, Mass.</nlm:affiliation><wicri:noCountry code="subField">Mass</wicri:noCountry></affiliation></author><author><name sortKey="Ballard, Barbara" sort="Ballard, Barbara" uniqKey="Ballard B" first="Barbara" last="Ballard">Barbara Ballard</name><affiliation><nlm:affiliation>Immune Deficiency Foundation, Towson, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Bauer, Sarah C" sort="Bauer, Sarah C" uniqKey="Bauer S" first="Sarah C" last="Bauer">Sarah C. Bauer</name><affiliation><nlm:affiliation>Developmental and Behavioral Pediatrics, Lurie Children's Hospital of Chicago, Northwestern Feinberg School of Medicine, Chicago, Ill.</nlm:affiliation><wicri:noCountry code="subField">Ill</wicri:noCountry></affiliation></author><author><name sortKey="Bleesing, Jack J H" sort="Bleesing, Jack J H" uniqKey="Bleesing J" first="Jack J H" last="Bleesing">Jack J H. Bleesing</name><affiliation wicri:level="2"><nlm:affiliation>Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Ohio</region></placeName><wicri:cityArea>Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati</wicri:cityArea></affiliation></author><author><name sortKey="Boyle, Marcia" sort="Boyle, Marcia" uniqKey="Boyle M" first="Marcia" last="Boyle">Marcia Boyle</name><affiliation><nlm:affiliation>Immune Deficiency Foundation, Towson, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Brower, Amy" sort="Brower, Amy" uniqKey="Brower A" first="Amy" last="Brower">Amy Brower</name><affiliation><nlm:affiliation>Newborn Screening Translational Research Network, American College of Medical Genetics and Genomics, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Buckley, Rebecca H" sort="Buckley, Rebecca H" uniqKey="Buckley R" first="Rebecca H" last="Buckley">Rebecca H. Buckley</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Allergy and Immunology, Duke University School of Medicine, Durham, NC.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Caroline du Nord</region></placeName><wicri:cityArea>Pediatric Allergy and Immunology, Duke University School of Medicine, Durham</wicri:cityArea></affiliation></author><author><name sortKey="Van Der Burg, Mirjam" sort="Van Der Burg, Mirjam" uniqKey="Van Der Burg M" first="Mirjam" last="Van Der Burg">Mirjam Van Der Burg</name><affiliation wicri:level="3"><nlm:affiliation>Molecular Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Molecular Immunology, Erasmus Medical Center, Rotterdam</wicri:regionArea><placeName><settlement type="city">Rotterdam</settlement><region nuts="2" type="province">Hollande-Méridionale</region></placeName></affiliation></author><author><name sortKey="Burroughs, Lauri M" sort="Burroughs, Lauri M" uniqKey="Burroughs L" first="Lauri M" last="Burroughs">Lauri M. Burroughs</name><affiliation><nlm:affiliation>Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="Candotti, Fabio" sort="Candotti, Fabio" uniqKey="Candotti F" first="Fabio" last="Candotti">Fabio Candotti</name><affiliation><nlm:affiliation>Genetics & Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Cant, Andrew J" sort="Cant, Andrew J" uniqKey="Cant A" first="Andrew J" last="Cant">Andrew J. Cant</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Immunology and Infectious Diseases and Pediatric Bone Marrow Transplant, Newcastle General Hospital, Newcastle upon Tyne, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Pediatric Immunology and Infectious Diseases and Pediatric Bone Marrow Transplant, Newcastle General Hospital, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Chatila, Talal" sort="Chatila, Talal" uniqKey="Chatila T" first="Talal" last="Chatila">Talal Chatila</name><affiliation><nlm:affiliation>Pediatric Allergy/Immunology, Children's Hospital, Harvard Medical School, Boston, Mass.</nlm:affiliation><wicri:noCountry code="subField">Mass</wicri:noCountry></affiliation></author><author><name sortKey="Cunningham Rundles, Charlotte" sort="Cunningham Rundles, Charlotte" uniqKey="Cunningham Rundles C" first="Charlotte" last="Cunningham-Rundles">Charlotte Cunningham-Rundles</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Clinical Immunology, Mount Sinai School of Medicine, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatric Clinical Immunology, Mount Sinai School of Medicine, New York</wicri:cityArea></affiliation></author><author><name sortKey="Dinauer, Mary C" sort="Dinauer, Mary C" uniqKey="Dinauer M" first="Mary C" last="Dinauer">Mary C. Dinauer</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Hematology/Oncology, Washington University School of Medicine, St Louis, Mo.</nlm:affiliation><orgName type="university">École de médecine (Université Washington de Saint-Louis)</orgName><country>États-Unis</country><placeName><settlement type="city">Saint-Louis (Missouri)</settlement><region type="state">Missouri (État)</region></placeName></affiliation></author><author><name sortKey="Dvorak, Christopher C" sort="Dvorak, Christopher C" uniqKey="Dvorak C" first="Christopher C" last="Dvorak">Christopher C. Dvorak</name><affiliation><nlm:affiliation>Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Filipovich, Alexandra H" sort="Filipovich, Alexandra H" uniqKey="Filipovich A" first="Alexandra H" last="Filipovich">Alexandra H. Filipovich</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Clinical Immunology, Division of Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Ohio</region></placeName><wicri:cityArea>Pediatric Clinical Immunology, Division of Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati</wicri:cityArea></affiliation></author><author><name sortKey="Fleisher, Thomas A" sort="Fleisher, Thomas A" uniqKey="Fleisher T" first="Thomas A" last="Fleisher">Thomas A. Fleisher</name><affiliation><nlm:affiliation>Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Bobby Gaspar, Hubert" sort="Bobby Gaspar, Hubert" uniqKey="Bobby Gaspar H" first="Hubert" last="Bobby Gaspar">Hubert Bobby Gaspar</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region><settlement type="city">Londres</settlement></placeName><orgName type="university">University College de Londres</orgName></affiliation></author><author><name sortKey="Gungor, Tayfun" sort="Gungor, Tayfun" uniqKey="Gungor T" first="Tayfun" last="Gungor">Tayfun Gungor</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Immunology and Blood and Marrow Transplantation, Children's Hospital Zurich, University of Zurich, Zurich, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Pediatric Immunology and Blood and Marrow Transplantation, Children's Hospital Zurich, University of Zurich, Zurich</wicri:regionArea><orgName type="university">Université de Zurich</orgName><placeName><settlement type="city">Zurich</settlement><region nuts="3" type="region">Canton de Zurich</region></placeName></affiliation></author><author><name sortKey="Haddad, Elie" sort="Haddad, Elie" uniqKey="Haddad E" first="Elie" last="Haddad">Elie Haddad</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Pediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec</wicri:regionArea><wicri:noRegion>Quebec</wicri:noRegion></affiliation></author><author><name sortKey="Hovermale, Emily" sort="Hovermale, Emily" uniqKey="Hovermale E" first="Emily" last="Hovermale">Emily Hovermale</name><affiliation><nlm:affiliation>Immune Deficiency Foundation, Towson, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Huang, Faith" sort="Huang, Faith" uniqKey="Huang F" first="Faith" last="Huang">Faith Huang</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Allergy/Immunology, Mount Sinai Medical Center, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatric Allergy/Immunology, Mount Sinai Medical Center, New York</wicri:cityArea></affiliation></author><author><name sortKey="Hurley, Alan" sort="Hurley, Alan" uniqKey="Hurley A" first="Alan" last="Hurley">Alan Hurley</name><affiliation><nlm:affiliation>Chronic Granulomatous Disease Association, San Marino, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Hurley, Mary" sort="Hurley, Mary" uniqKey="Hurley M" first="Mary" last="Hurley">Mary Hurley</name><affiliation><nlm:affiliation>Chronic Granulomatous Disease Association, San Marino, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Iyengar, Sumathi" sort="Iyengar, Sumathi" uniqKey="Iyengar S" first="Sumathi" last="Iyengar">Sumathi Iyengar</name><affiliation><nlm:affiliation>Wiskott-Aldrich Foundation, Smyrna, Ga.</nlm:affiliation><wicri:noCountry code="subField">Ga</wicri:noCountry></affiliation></author><author><name sortKey="Kang, Elizabeth M" sort="Kang, Elizabeth M" uniqKey="Kang E" first="Elizabeth M" last="Kang">Elizabeth M. Kang</name><affiliation><nlm:affiliation>Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Logan, Brent R" sort="Logan, Brent R" uniqKey="Logan B" first="Brent R" last="Logan">Brent R. Logan</name><affiliation><nlm:affiliation>Center for International Blood and Marrow Transplant Research and Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.</nlm:affiliation><wicri:noCountry code="subField">Wis</wicri:noCountry></affiliation></author><author><name sortKey="Long Boyle, Janel R" sort="Long Boyle, Janel R" uniqKey="Long Boyle J" first="Janel R" last="Long-Boyle">Janel R. Long-Boyle</name><affiliation><nlm:affiliation>Department of Clinical Pharmacy, School of Pharmacy, University of California, San Francisco, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Malech, Harry L" sort="Malech, Harry L" uniqKey="Malech H" first="Harry L" last="Malech">Harry L. Malech</name><affiliation><nlm:affiliation>Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.</nlm:affiliation><wicri:noCountry code="subField">Md</wicri:noCountry></affiliation></author><author><name sortKey="Mcghee, Sean A" sort="Mcghee, Sean A" uniqKey="Mcghee S" first="Sean A" last="Mcghee">Sean A. Mcghee</name><affiliation><nlm:affiliation>Pediatric Allergy/Immunology, Lucile Packard Children's Hospital, Stanford University Medical Center, Stanford, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Modell, Fred" sort="Modell, Fred" uniqKey="Modell F" first="Fred" last="Modell">Fred Modell</name><affiliation wicri:level="2"><nlm:affiliation>Jeffrey Modell Foundation, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Jeffrey Modell Foundation, New York</wicri:cityArea></affiliation></author><author><name sortKey="Modell, Vicki" sort="Modell, Vicki" uniqKey="Modell V" first="Vicki" last="Modell">Vicki Modell</name><affiliation wicri:level="2"><nlm:affiliation>Jeffrey Modell Foundation, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Jeffrey Modell Foundation, New York</wicri:cityArea></affiliation></author><author><name sortKey="Ochs, Hans D" sort="Ochs, Hans D" uniqKey="Ochs H" first="Hans D" last="Ochs">Hans D. Ochs</name><affiliation><nlm:affiliation>Center for Immunity and Immunotherapy, Seattle Children's Hospital Research Institute, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="O Reilly, Richard J" sort="O Reilly, Richard J" uniqKey="O Reilly R" first="Richard J" last="O'Reilly">Richard J. O'Reilly</name><affiliation wicri:level="2"><nlm:affiliation>Pediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York</wicri:cityArea></affiliation></author><author><name sortKey="Parkman, Robertson" sort="Parkman, Robertson" uniqKey="Parkman R" first="Robertson" last="Parkman">Robertson Parkman</name><affiliation><nlm:affiliation>Division of Research Immunology/B.M.T., Children's Hospital Los Angeles, Los Angeles, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Rawlings, David J" sort="Rawlings, David J" uniqKey="Rawlings D" first="David J" last="Rawlings">David J. Rawlings</name><affiliation><nlm:affiliation>Pediatric Immunology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="Routes, John M" sort="Routes, John M" uniqKey="Routes J" first="John M" last="Routes">John M. Routes</name><affiliation><nlm:affiliation>Pediatric Allergy and Clinical Immunology, Children's Hospital of Wisconsin, Medical College of Wisconsin, Milwaukee, Wis.</nlm:affiliation><wicri:noCountry code="subField">Wis</wicri:noCountry></affiliation></author><author><name sortKey="Shearer, William T" sort="Shearer, William T" uniqKey="Shearer W" first="William T" last="Shearer">William T. Shearer</name><affiliation><nlm:affiliation>Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex.</nlm:affiliation><wicri:noCountry code="subField">Tex</wicri:noCountry></affiliation></author><author><name sortKey="Small, Trudy N" sort="Small, Trudy N" uniqKey="Small T" first="Trudy N" last="Small">Trudy N. Small</name><affiliation wicri:level="2"><nlm:affiliation>Pediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Pediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York</wicri:cityArea></affiliation></author><author><name sortKey="Smith, Heather" sort="Smith, Heather" uniqKey="Smith H" first="Heather" last="Smith">Heather Smith</name><affiliation><nlm:affiliation>SCID Angels for Life Foundation, Lakeland, Fla.</nlm:affiliation><wicri:noCountry code="subField">Fla</wicri:noCountry></affiliation></author><author><name sortKey="Sullivan, Kathleen E" sort="Sullivan, Kathleen E" uniqKey="Sullivan K" first="Kathleen E" last="Sullivan">Kathleen E. Sullivan</name><affiliation><nlm:affiliation>Pediatric Immunology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa.</nlm:affiliation><wicri:noCountry code="subField">Pa</wicri:noCountry></affiliation></author><author><name sortKey="Szabolcs, Paul" sort="Szabolcs, Paul" uniqKey="Szabolcs P" first="Paul" last="Szabolcs">Paul Szabolcs</name><affiliation><nlm:affiliation>Bone Marrow Transplantation and Cellular Therapies, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pa.</nlm:affiliation><wicri:noCountry code="subField">Pa</wicri:noCountry></affiliation></author><author><name sortKey="Thrasher, Adrian" sort="Thrasher, Adrian" uniqKey="Thrasher A" first="Adrian" last="Thrasher">Adrian Thrasher</name><affiliation wicri:level="4"><nlm:affiliation>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region><settlement type="city">Londres</settlement></placeName><orgName type="university">University College de Londres</orgName></affiliation></author><author><name sortKey="Torgerson, Troy R" sort="Torgerson, Troy R" uniqKey="Torgerson T" first="Troy R" last="Torgerson">Troy R. Torgerson</name><affiliation><nlm:affiliation>Pediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.</nlm:affiliation><wicri:noCountry code="subField">Wash</wicri:noCountry></affiliation></author><author><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name><affiliation wicri:level="3"><nlm:affiliation>Blood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London, United Kingdom.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Blood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Weinberg, Kenneth" sort="Weinberg, Kenneth" uniqKey="Weinberg K" first="Kenneth" last="Weinberg">Kenneth Weinberg</name><affiliation><nlm:affiliation>Pediatric Stem Cell Transplantation and Hematology/Oncology, Lucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, Calif.</nlm:affiliation><wicri:noCountry code="subField">Calif</wicri:noCountry></affiliation></author><author><name sortKey="Zuniga Pflucker, Juan Carlos" sort="Zuniga Pflucker, Juan Carlos" uniqKey="Zuniga Pflucker J" first="Juan Carlos" last="Zuniga-Pflucker">Juan Carlos Zuniga-Pflucker</name><affiliation wicri:level="4"><nlm:affiliation>Department of Immunology, University of Toronto, Toronto, Ontario, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Department of Immunology, University of Toronto, Toronto, Ontario</wicri:regionArea><orgName type="university">Université de Toronto</orgName><placeName><settlement type="city">Toronto</settlement><region type="state">Ontario</region></placeName></affiliation></author></analytic><series><title level="j">The Journal of allergy and clinical immunology</title><idno type="eISSN">1097-6825</idno><imprint><date when="2014" type="published">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Hematopoietic Stem Cell Transplantation</term><term>Humans</term><term>Immunologic Deficiency Syndromes (diagnosis)</term><term>Immunologic Deficiency Syndromes (therapy)</term><term>Infant, Newborn</term><term>Neonatal Screening</term><term>Pilot Projects</term><term>Societies, Scientific</term></keywords><keywords scheme="KwdFr" xml:lang="fr"><term>Déficits immunitaires ()</term><term>Déficits immunitaires (diagnostic)</term><term>Dépistage néonatal</term><term>Humains</term><term>Nouveau-né</term><term>Projets pilotes</term><term>Sociétés savantes</term><term>Transplantation de cellules souches hématopoïétiques</term></keywords><keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Immunologic Deficiency Syndromes</term></keywords><keywords scheme="MESH" qualifier="diagnostic" xml:lang="fr"><term>Déficits immunitaires</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Immunologic Deficiency Syndromes</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Hematopoietic Stem Cell Transplantation</term><term>Humans</term><term>Infant, Newborn</term><term>Neonatal Screening</term><term>Pilot Projects</term><term>Societies, Scientific</term></keywords><keywords scheme="MESH" xml:lang="fr"><term>Déficits immunitaires</term><term>Dépistage néonatal</term><term>Humains</term><term>Nouveau-né</term><term>Projets pilotes</term><term>Sociétés savantes</term><term>Transplantation de cellules souches hématopoïétiques</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.</div></front></TEI><affiliations><list><country><li>Canada</li><li>Pays-Bas</li><li>Royaume-Uni</li><li>Suisse</li><li>États-Unis</li></country><region><li>Angleterre</li><li>Canton de Zurich</li><li>Caroline du Nord</li><li>Grand Londres</li><li>Hollande-Méridionale</li><li>Missouri (État)</li><li>Ohio</li><li>Ontario</li><li>État de New York</li></region><settlement><li>Londres</li><li>Rotterdam</li><li>Saint-Louis (Missouri)</li><li>Toronto</li><li>Zurich</li></settlement><orgName><li>University College de Londres</li><li>Université de Toronto</li><li>Université de Zurich</li><li>École de médecine (Université Washington de Saint-Louis)</li></orgName></list><tree><noCountry><name sortKey="Ballard, Barbara" sort="Ballard, Barbara" uniqKey="Ballard B" first="Barbara" last="Ballard">Barbara Ballard</name><name sortKey="Bauer, Sarah C" sort="Bauer, Sarah C" uniqKey="Bauer S" first="Sarah C" last="Bauer">Sarah C. Bauer</name><name sortKey="Boyle, Marcia" sort="Boyle, Marcia" uniqKey="Boyle M" first="Marcia" last="Boyle">Marcia Boyle</name><name sortKey="Brower, Amy" sort="Brower, Amy" uniqKey="Brower A" first="Amy" last="Brower">Amy Brower</name><name sortKey="Burroughs, Lauri M" sort="Burroughs, Lauri M" uniqKey="Burroughs L" first="Lauri M" last="Burroughs">Lauri M. Burroughs</name><name sortKey="Candotti, Fabio" sort="Candotti, Fabio" uniqKey="Candotti F" first="Fabio" last="Candotti">Fabio Candotti</name><name sortKey="Chatila, Talal" sort="Chatila, Talal" uniqKey="Chatila T" first="Talal" last="Chatila">Talal Chatila</name><name sortKey="Cowan, Morton J" sort="Cowan, Morton J" uniqKey="Cowan M" first="Morton J" last="Cowan">Morton J. Cowan</name><name sortKey="Dvorak, Christopher C" sort="Dvorak, Christopher C" uniqKey="Dvorak C" first="Christopher C" last="Dvorak">Christopher C. Dvorak</name><name sortKey="Fleisher, Thomas A" sort="Fleisher, Thomas A" uniqKey="Fleisher T" first="Thomas A" last="Fleisher">Thomas A. Fleisher</name><name sortKey="Griffith, Linda M" sort="Griffith, Linda M" uniqKey="Griffith L" first="Linda M" last="Griffith">Linda M. Griffith</name><name sortKey="Hovermale, Emily" sort="Hovermale, Emily" uniqKey="Hovermale E" first="Emily" last="Hovermale">Emily Hovermale</name><name sortKey="Hurley, Alan" sort="Hurley, Alan" uniqKey="Hurley A" first="Alan" last="Hurley">Alan Hurley</name><name sortKey="Hurley, Mary" sort="Hurley, Mary" uniqKey="Hurley M" first="Mary" last="Hurley">Mary Hurley</name><name sortKey="Iyengar, Sumathi" sort="Iyengar, Sumathi" uniqKey="Iyengar S" first="Sumathi" last="Iyengar">Sumathi Iyengar</name><name sortKey="Kang, Elizabeth M" sort="Kang, Elizabeth M" uniqKey="Kang E" first="Elizabeth M" last="Kang">Elizabeth M. Kang</name><name sortKey="Kohn, Donald B" sort="Kohn, Donald B" uniqKey="Kohn D" first="Donald B" last="Kohn">Donald B. Kohn</name><name sortKey="Logan, Brent R" sort="Logan, Brent R" uniqKey="Logan B" first="Brent R" last="Logan">Brent R. Logan</name><name sortKey="Long Boyle, Janel R" sort="Long Boyle, Janel R" uniqKey="Long Boyle J" first="Janel R" last="Long-Boyle">Janel R. Long-Boyle</name><name sortKey="Malech, Harry L" sort="Malech, Harry L" uniqKey="Malech H" first="Harry L" last="Malech">Harry L. Malech</name><name sortKey="Mcghee, Sean A" sort="Mcghee, Sean A" uniqKey="Mcghee S" first="Sean A" last="Mcghee">Sean A. Mcghee</name><name sortKey="Notarangelo, Luigi D" sort="Notarangelo, Luigi D" uniqKey="Notarangelo L" first="Luigi D" last="Notarangelo">Luigi D. Notarangelo</name><name sortKey="Ochs, Hans D" sort="Ochs, Hans D" uniqKey="Ochs H" first="Hans D" last="Ochs">Hans D. Ochs</name><name sortKey="Pai, Sung Yun" sort="Pai, Sung Yun" uniqKey="Pai S" first="Sung-Yun" last="Pai">Sung-Yun Pai</name><name sortKey="Parkman, Robertson" sort="Parkman, Robertson" uniqKey="Parkman R" first="Robertson" last="Parkman">Robertson Parkman</name><name sortKey="Puck, Jennifer M" sort="Puck, Jennifer M" uniqKey="Puck J" first="Jennifer M" last="Puck">Jennifer M. Puck</name><name sortKey="Rawlings, David J" sort="Rawlings, David J" uniqKey="Rawlings D" first="David J" last="Rawlings">David J. Rawlings</name><name sortKey="Routes, John M" sort="Routes, John M" uniqKey="Routes J" first="John M" last="Routes">John M. Routes</name><name sortKey="Shearer, William T" sort="Shearer, William T" uniqKey="Shearer W" first="William T" last="Shearer">William T. Shearer</name><name sortKey="Smith, Heather" sort="Smith, Heather" uniqKey="Smith H" first="Heather" last="Smith">Heather Smith</name><name sortKey="Sullivan, Kathleen E" sort="Sullivan, Kathleen E" uniqKey="Sullivan K" first="Kathleen E" last="Sullivan">Kathleen E. Sullivan</name><name sortKey="Szabolcs, Paul" sort="Szabolcs, Paul" uniqKey="Szabolcs P" first="Paul" last="Szabolcs">Paul Szabolcs</name><name sortKey="Torgerson, Troy R" sort="Torgerson, Troy R" uniqKey="Torgerson T" first="Troy R" last="Torgerson">Troy R. Torgerson</name><name sortKey="Weinberg, Kenneth" sort="Weinberg, Kenneth" uniqKey="Weinberg K" first="Kenneth" last="Weinberg">Kenneth Weinberg</name></noCountry><country name="États-Unis"><region name="Ohio"><name sortKey="Bleesing, Jack J H" sort="Bleesing, Jack J H" uniqKey="Bleesing J" first="Jack J H" last="Bleesing">Jack J H. Bleesing</name></region><name sortKey="Buckley, Rebecca H" sort="Buckley, Rebecca H" uniqKey="Buckley R" first="Rebecca H" last="Buckley">Rebecca H. Buckley</name><name sortKey="Cunningham Rundles, Charlotte" sort="Cunningham Rundles, Charlotte" uniqKey="Cunningham Rundles C" first="Charlotte" last="Cunningham-Rundles">Charlotte Cunningham-Rundles</name><name sortKey="Dinauer, Mary C" sort="Dinauer, Mary C" uniqKey="Dinauer M" first="Mary C" last="Dinauer">Mary C. Dinauer</name><name sortKey="Filipovich, Alexandra H" sort="Filipovich, Alexandra H" uniqKey="Filipovich A" first="Alexandra H" last="Filipovich">Alexandra H. Filipovich</name><name sortKey="Huang, Faith" sort="Huang, Faith" uniqKey="Huang F" first="Faith" last="Huang">Faith Huang</name><name sortKey="Modell, Fred" sort="Modell, Fred" uniqKey="Modell F" first="Fred" last="Modell">Fred Modell</name><name sortKey="Modell, Vicki" sort="Modell, Vicki" uniqKey="Modell V" first="Vicki" last="Modell">Vicki Modell</name><name sortKey="O Reilly, Richard J" sort="O Reilly, Richard J" uniqKey="O Reilly R" first="Richard J" last="O'Reilly">Richard J. O'Reilly</name><name sortKey="Small, Trudy N" sort="Small, Trudy N" uniqKey="Small T" first="Trudy N" last="Small">Trudy N. Small</name></country><country name="Pays-Bas"><region name="Hollande-Méridionale"><name sortKey="Van Der Burg, Mirjam" sort="Van Der Burg, Mirjam" uniqKey="Van Der Burg M" first="Mirjam" last="Van Der Burg">Mirjam Van Der Burg</name></region></country><country name="Royaume-Uni"><noRegion><name sortKey="Cant, Andrew J" sort="Cant, Andrew J" uniqKey="Cant A" first="Andrew J" last="Cant">Andrew J. Cant</name></noRegion><name sortKey="Bobby Gaspar, Hubert" sort="Bobby Gaspar, Hubert" uniqKey="Bobby Gaspar H" first="Hubert" last="Bobby Gaspar">Hubert Bobby Gaspar</name><name sortKey="Thrasher, Adrian" sort="Thrasher, Adrian" uniqKey="Thrasher A" first="Adrian" last="Thrasher">Adrian Thrasher</name><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name></country><country name="Suisse"><region name="Canton de Zurich"><name sortKey="Gungor, Tayfun" sort="Gungor, Tayfun" uniqKey="Gungor T" first="Tayfun" last="Gungor">Tayfun Gungor</name></region></country><country name="Canada"><noRegion><name sortKey="Haddad, Elie" sort="Haddad, Elie" uniqKey="Haddad E" first="Elie" last="Haddad">Elie Haddad</name></noRegion><name sortKey="Zuniga Pflucker, Juan Carlos" sort="Zuniga Pflucker, Juan Carlos" uniqKey="Zuniga Pflucker J" first="Juan Carlos" last="Zuniga-Pflucker">Juan Carlos Zuniga-Pflucker</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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