{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Freeman AF"],"funding":["National Institute of Arthritis and Musculoskeletal and Skin Diseases","National Institute of Allergy and Infectious Diseases","Intramural NIH HHS","Clinical Center","National Institute of Diabetes and Digestive and Kidney Diseases","U.S. Department of Health and Human Services","National Cancer Institute","National Institutes of Health","Center for Cancer Research"],"pagination":["176-187"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11700784"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["155(1)"],"pubmed_abstract":["<h4>Background</h4>DOCK8 deficiency is a primary immunodeficiency in which allogeneic hematopoietic cell transplantation (HCT) represents the only known cure. We tested the ability of a busulfan-based regimen to achieve reliable engraftment and high levels of donor chimerism with acceptable toxicity in a prospective clinical trial in DOCK8 deficiency.<h4>Objectives</h4>To both evaluate the ability of HCT to reverse the clinical phenotype and to correct the immunologic abnormalities by 1 year post HCT.<h4>Methods</h4>We conducted a prospective HCT trial for recipients with DOCK8 deficiency. Subjects were recruited from October 5, 2010, to December 30, 2022. Donor sources included fully matched related and unrelated donors and haploidentical donors. The reduced toxicity, myeloablative condit"],"journal":["The Journal of allergy and clinical immunology"],"pubmed_title":["Hematopoietic cell transplantation for DOCK8 deficiency: Results from a prospective clinical trial."],"pmcid":["PMC11700784"],"funding_grant_id":["ZID BC012026","1ZIAAI001193","ZIA AI001193","ZIA BC011823","Z99 CA999999"],"pubmed_authors":["Jing H","Kayaoglu B","Pai SY","Gonzalez CE","Young FD","Shah NN","Heller T","Cuellar-Rodriguez J","Flannelly E","Gea-Banacloche J","Little L","Mo G","Steinberg SM","Hickstein DD","Freeman AF","Cole K","Hughes TE","Piette N","Kong HH","Holland SM","Hammoud DA","Yates B","Su HC"],"additional_accession":[]},"is_claimable":false,"name":"Hematopoietic cell transplantation for DOCK8 deficiency: Results from a prospective clinical trial.","description":"<h4>Background</h4>DOCK8 deficiency is a primary immunodeficiency in which allogeneic hematopoietic cell transplantation (HCT) represents the only known cure. We tested the ability of a busulfan-based regimen to achieve reliable engraftment and high levels of donor chimerism with acceptable toxicity in a prospective clinical trial in DOCK8 deficiency.<h4>Objectives</h4>To both evaluate the ability of HCT to reverse the clinical phenotype and to correct the immunologic abnormalities by 1 year post HCT.<h4>Methods</h4>We conducted a prospective HCT trial for recipients with DOCK8 deficiency. Subjects were recruited from October 5, 2010, to December 30, 2022. Donor sources included fully matched related and unrelated donors and haploidentical donors. The reduced toxicity, myeloablative condit","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Jan","modification":"2026-06-06T09:01:05.595Z","creation":"2026-05-28T03:11:20.985Z"},"accession":"S-EPMC11700784","cross_references":{"pubmed":["39233015"],"doi":["10.1016/j.jaci.2024.08.021"]}}