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Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.

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Title: Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.
Authors: Cuvelier GDE; Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada., Logan BR; Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI., Prockop SE; Stem Cell Transplant Service, Dana Farber Cancer Institute/Boston Children's Hospital, Boston, MA., Buckley RH; Duke University Medical Center, Durham, NC., Kuo CY; Division of Allergy, Immunology, Rheumatology, Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, CA., Griffith LM; Division of Allergy, Immunology and Transplantation, National Institutes of Allergy, National Institutes of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD., Liu X; Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI., Yip A; University of California San Francisco Benioff Children's Hospital, San Francisco, CA., Hershfield MS; Duke University Medical Center, Durham, NC., Ayoub PG; Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, CA., Moore TB; Department of Pediatric Hematology-Oncology, Mattel Children's Hospital, University of California, Los Angeles, CA., Dorsey MJ; University of California San Francisco Benioff Children's Hospital, San Francisco, CA., O'Reilly RJ; Stem Cell Transplantation and Cellular Therapy, MSK Kids, Memorial Sloan-Kettering Cancer Center, New York, NY., Kapoor N; Division of Hematology, Oncology and Blood and Marrow Transplant, Children's Hospital, Los Angeles, CA., Pai SY; Immune Deficiency Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, Bethesda, MD., Kapadia M; Boston Children's Hospital, Dana-Farber Cancer Institute, Boston, MA., Ebens CL; Division of Pediatric Blood and Marrow Transplant and Cellular Therapy, MHealth Fairview Masonic Children's Hospital, Minneapolis, MN., Forbes Satter LR; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX., Burroughs LM; Fred Hutchinson Cancer Research Center, University of Washington, Department of Pediatrics and Seattle Children's Hospital, Seattle, WA., Petrovic A; Fred Hutchinson Cancer Research Center, University of Washington, Department of Pediatrics and Seattle Children's Hospital, Seattle, WA., Chellapandian D; Center for Cell and Gene Therapy for Non-Malignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, FL., Heimall J; Division of Allergy and Immunology, Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA., Shyr DC; Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Lucile Packard Children's Hospital, Stanford School of Medicine, Palo Alto, CA., Rayes A; Primary Children's Hospital, University of Utah, Salt Lake City, UT., Bednarski JJ; Washington University, St. Louis Children's Hospital, St. Louis, MO., Chandra S; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH., Chandrakasan S; Bone Marrow Transplantation and Immune Deficiency, Children's Hospital of Atlanta, Atlanta, GA., Gillio AP; Children's Cancer Institute, Hackensack University Medical Center, Hackensack, NJ., Madden L; Methodist Children's Hospital of South Texas, San Antonio, TX., Quigg TC; Pediatric Blood and Marrow Transplant and Cellular Therapy Program, Helen DeVos Children's Hospital, Michigan State University College of Human Medicine, Grand Rapids, MI., Caywood EH; Nemours Children's Health, Thomas Jefferson University, Wilmington, DE., Dávila Saldaña BJ; Division of Blood and Marrow Transplantation, Children's National Hospital, Washington, DC., DeSantes K; Division of Pediatric Hematology-Oncology & Bone Marrow Transplant, University of Wisconsin, American Family Children's Hospital, Madison, WI., Eissa H; Division of Pediatric Hematology-Oncology-BMT, Aurora, CO., Goldman FD; Division of Pediatric Hematology and Oncology and Bone Marrow Transplant, University of Alabama at Birmingham, Birmingham, AL., Rozmus J; British Columbia Children's Hospital, Vancouver, BC, Canada., Shah AJ; Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Lucile Packard Children's Hospital, Stanford School of Medicine, Palo Alto, CA., Vander Lugt MT; Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, MI., Thakar MS; Fred Hutchinson Cancer Research Center, University of Washington, Department of Pediatrics and Seattle Children's Hospital, Seattle, WA., Parrott RE; Duke University Medical Center, Durham, NC., Martinez C; Hematology/Oncology/BMT, Texas Children's Hospital, Baylor College of Medicine, Houston, TX., Leiding JW; Division of Allergy and Immunology, Johns Hopkins University, St Petersburg, FL., Torgerson TR; Allen Institute for Immunology, Seattle, WA., Pulsipher MA; Division of Pediatric Hematology and Oncology, Intermountain Primary Children's Hospital, Huntsman Cancer Institute at the University of Utah Spencer Fox Eccles School of Medicine, Salt Lake City, UT., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD; and., Cowan MJ; University of California San Francisco Benioff Children's Hospital, San Francisco, CA., Dvorak CC; University of California San Francisco Benioff Children's Hospital, San Francisco, CA., Haddad E; Department of Pediatrics, Centre Hospitalier Universitaire (CHU) Sainte-Justine, University of Montreal, Montreal, QC, Canada., Puck JM; University of California San Francisco Benioff Children's Hospital, San Francisco, CA., Kohn DB; Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, CA.
Source: Blood [Blood] 2022 Aug 18; Vol. 140 (7), pp. 685-705.
Publication Type: Clinical Trial; Journal Article
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.
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  Data: <searchLink fieldCode="AU" term="%22Cuvelier+GDE%22">Cuvelier GDE</searchLink>; Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada.<br /><searchLink fieldCode="AU" term="%22Logan+BR%22">Logan BR</searchLink>; Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI.<br /><searchLink fieldCode="AU" term="%22Prockop+SE%22">Prockop SE</searchLink>; Stem Cell Transplant Service, Dana Farber Cancer Institute/Boston Children's Hospital, Boston, MA.<br /><searchLink fieldCode="AU" term="%22Buckley+RH%22">Buckley RH</searchLink>; Duke University Medical Center, Durham, NC.<br /><searchLink fieldCode="AU" term="%22Kuo+CY%22">Kuo CY</searchLink>; Division of Allergy, Immunology, Rheumatology, Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, CA.<br /><searchLink fieldCode="AU" term="%22Griffith+LM%22">Griffith LM</searchLink>; Division of Allergy, Immunology and Transplantation, National Institutes of Allergy, National Institutes of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Liu+X%22">Liu X</searchLink>; Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI.<br /><searchLink fieldCode="AU" term="%22Yip+A%22">Yip A</searchLink>; University of California San Francisco Benioff Children's Hospital, San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Hershfield+MS%22">Hershfield MS</searchLink>; Duke University Medical Center, Durham, NC.<br /><searchLink fieldCode="AU" term="%22Ayoub+PG%22">Ayoub PG</searchLink>; Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, CA.<br /><searchLink fieldCode="AU" term="%22Moore+TB%22">Moore TB</searchLink>; Department of Pediatric Hematology-Oncology, Mattel Children's Hospital, University of California, Los Angeles, CA.<br /><searchLink fieldCode="AU" term="%22Dorsey+MJ%22">Dorsey MJ</searchLink>; University of California San Francisco Benioff Children's Hospital, San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22O'Reilly+RJ%22">O'Reilly RJ</searchLink>; Stem Cell Transplantation and Cellular Therapy, MSK Kids, Memorial Sloan-Kettering Cancer Center, New York, NY.<br /><searchLink fieldCode="AU" term="%22Kapoor+N%22">Kapoor N</searchLink>; 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Fred Hutchinson Cancer Research Center, University of Washington, Department of Pediatrics and Seattle Children's Hospital, Seattle, WA.<br /><searchLink fieldCode="AU" term="%22Chellapandian+D%22">Chellapandian D</searchLink>; Center for Cell and Gene Therapy for Non-Malignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, FL.<br /><searchLink fieldCode="AU" term="%22Heimall+J%22">Heimall J</searchLink>; Division of Allergy and Immunology, Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA.<br /><searchLink fieldCode="AU" term="%22Shyr+DC%22">Shyr DC</searchLink>; Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Lucile Packard Children's Hospital, Stanford School of Medicine, Palo Alto, CA.<br /><searchLink fieldCode="AU" term="%22Rayes+A%22">Rayes A</searchLink>; Primary Children's Hospital, University of Utah, Salt Lake City, UT.<br /><searchLink fieldCode="AU" term="%22Bednarski+JJ%22">Bednarski JJ</searchLink>; Washington University, St. Louis Children's Hospital, St. Louis, MO.<br /><searchLink fieldCode="AU" term="%22Chandra+S%22">Chandra S</searchLink>; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.<br /><searchLink fieldCode="AU" term="%22Chandrakasan+S%22">Chandrakasan S</searchLink>; Bone Marrow Transplantation and Immune Deficiency, Children's Hospital of Atlanta, Atlanta, GA.<br /><searchLink fieldCode="AU" term="%22Gillio+AP%22">Gillio AP</searchLink>; Children's Cancer Institute, Hackensack University Medical Center, Hackensack, NJ.<br /><searchLink fieldCode="AU" term="%22Madden+L%22">Madden L</searchLink>; Methodist Children's Hospital of South Texas, San Antonio, TX.<br /><searchLink fieldCode="AU" term="%22Quigg+TC%22">Quigg TC</searchLink>; Pediatric Blood and Marrow Transplant and Cellular Therapy Program, Helen DeVos Children's Hospital, Michigan State University College of Human Medicine, Grand Rapids, MI.<br /><searchLink fieldCode="AU" term="%22Caywood+EH%22">Caywood EH</searchLink>; Nemours Children's Health, Thomas Jefferson University, Wilmington, DE.<br /><searchLink fieldCode="AU" term="%22Dávila+Saldaña+BJ%22">Dávila Saldaña BJ</searchLink>; Division of Blood and Marrow Transplantation, Children's National Hospital, Washington, DC.<br /><searchLink fieldCode="AU" term="%22DeSantes+K%22">DeSantes K</searchLink>; Division of Pediatric Hematology-Oncology & Bone Marrow Transplant, University of Wisconsin, American Family Children's Hospital, Madison, WI.<br /><searchLink fieldCode="AU" term="%22Eissa+H%22">Eissa H</searchLink>; Division of Pediatric Hematology-Oncology-BMT, Aurora, CO.<br /><searchLink fieldCode="AU" term="%22Goldman+FD%22">Goldman FD</searchLink>; Division of Pediatric Hematology and Oncology and Bone Marrow Transplant, University of Alabama at Birmingham, Birmingham, AL.<br /><searchLink fieldCode="AU" term="%22Rozmus+J%22">Rozmus J</searchLink>; British Columbia Children's Hospital, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Shah+AJ%22">Shah AJ</searchLink>; Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Lucile Packard Children's Hospital, Stanford School of Medicine, Palo Alto, CA.<br /><searchLink fieldCode="AU" term="%22Vander+Lugt+MT%22">Vander Lugt MT</searchLink>; Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, MI.<br /><searchLink fieldCode="AU" term="%22Thakar+MS%22">Thakar MS</searchLink>; Fred Hutchinson Cancer Research Center, University of Washington, Department of Pediatrics and Seattle Children's Hospital, Seattle, WA.<br /><searchLink fieldCode="AU" term="%22Parrott+RE%22">Parrott RE</searchLink>; Duke University Medical Center, Durham, NC.<br /><searchLink fieldCode="AU" term="%22Martinez+C%22">Martinez C</searchLink>; Hematology/Oncology/BMT, Texas Children's Hospital, Baylor College of Medicine, Houston, TX.<br /><searchLink fieldCode="AU" term="%22Leiding+JW%22">Leiding JW</searchLink>; Division of Allergy and Immunology, Johns Hopkins University, St Petersburg, FL.<br /><searchLink fieldCode="AU" term="%22Torgerson+TR%22">Torgerson TR</searchLink>; Allen Institute for Immunology, Seattle, WA.<br /><searchLink fieldCode="AU" term="%22Pulsipher+MA%22">Pulsipher MA</searchLink>; Division of Pediatric Hematology and Oncology, Intermountain Primary Children's Hospital, Huntsman Cancer Institute at the University of Utah Spencer Fox Eccles School of Medicine, Salt Lake City, UT.<br /><searchLink fieldCode="AU" term="%22Notarangelo+LD%22">Notarangelo LD</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD; and.<br /><searchLink fieldCode="AU" term="%22Cowan+MJ%22">Cowan MJ</searchLink>; University of California San Francisco Benioff Children's Hospital, San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Dvorak+CC%22">Dvorak CC</searchLink>; University of California San Francisco Benioff Children's Hospital, San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Haddad+E%22">Haddad E</searchLink>; Department of Pediatrics, Centre Hospitalier Universitaire (CHU) Sainte-Justine, University of Montreal, Montreal, QC, Canada.<br /><searchLink fieldCode="AU" term="%22Puck+JM%22">Puck JM</searchLink>; University of California San Francisco Benioff Children's Hospital, San Francisco, CA.<br /><searchLink fieldCode="AU" term="%22Kohn+DB%22">Kohn DB</searchLink>; Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, CA.
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