![]() Two hundred eighty-five (86%) of the patients were determined to be eligible, and 47 (14%) were not eligible. Eligibility for inclusion in the study and classification into disease groups were established by using set criteria and applied by an expert review group. The Primary Immune Deficiency Treatment Consortium attempted to develop a uniform set of criteria for diagnosing SCID and related disorders and has evaluated the results as part of a retrospective study of SCID in North America.Ĭlinical records from 2000 through 2009 at 27 centers in North America were collected on 332 children treated with hematopoietic stem cell transplantation (HCT), enzyme replacement therapy, or gene therapy for SCID and related disorders. The approach to the diagnosis of severe combined immunodeficiency disease (SCID) and related disorders varies among institutions and countries. 11 Department of Pediatrics and Immunology, Duke University Medical Center, Durham, NC.10 Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital Cancer Center, Houston, Tex.9 Division of Hematology and Oncology, Boston Children's Hospital, and Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Mass.8 Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md.7 Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.6 Departments of Microbiology, Immunology & Molecular Genetics and Pediatrics, University of California, Los Angeles, Calif.5 Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.4 Division of Immunology and the Manton Center for Orphan Disease Research, Children's Hospital Boston, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass.3 Division of Biostatistics, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wis.Electronic address: 2 Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplant, Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif. 1 Department of Pediatrics, Section of Immunology, Allergy, and Rheumatology, Baylor College of Medicine, Texas Children's Hospital, Houston, Tex.
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