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Immunol."},{"key":"10.1016\/j.ymthe.2017.12.022_bib68","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.bbmt.2008.11.022","article-title":"Association between busulfan exposure and outcome in children receiving intravenous busulfan before hematologic stem cell transplantation","volume":"15","author":"Bartelink","year":"2009","journal-title":"Biol. Blood Marrow Transplant."},{"key":"10.1016\/j.ymthe.2017.12.022_bib69","first-page":"1","article-title":"Guidelines for prevention and treatment of opportunistic infections in HIV-infected adults and adolescents: recommendations from CDC, the National Institutes of Health, and the HIV Medicine Association of the Infectious Diseases Society of America","volume":"58","author":"Kaplan","year":"2009","journal-title":"MMWR Recomm. Rep."},{"key":"10.1016\/j.ymthe.2017.12.022_bib70","unstructured":"European Society for Blood and Marrow Transplantation (EBMT), European Society\u00a0for Immunodeficiencies (ESID) (2017). Guidelines for haematopoietic stem\u00a0cell transplantation for primary immunodeficiencies. https:\/\/esid.org\/layout\/set\/print\/Working-Parties\/Inborn-Errors-Working-Party-IEWP\/Resources\/UPDATED!-EBMT-ESID-GUIDELINES-FOR-HAEMATOPOIETIC-STEM-CELL-TRANSPLANTATION-FOR-PI."},{"key":"10.1016\/j.ymthe.2017.12.022_bib71","unstructured":"National Cancer Institute (2009). Common Terminology Criteria for Adverse Events (CTCAE) v 4.0. National Institutes of Health."},{"key":"10.1016\/j.ymthe.2017.12.022_bib72","doi-asserted-by":"crossref","first-page":"1417","DOI":"10.1016\/j.jaci.2011.04.014","article-title":"Purine metabolism, immune reconstitution, and abdominal adipose tumor after gene therapy for adenosine deaminase deficiency","volume":"127","author":"Grunebaum","year":"2011","journal-title":"J.\u00a0Allergy Clin. 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