Umbilical cord blood transplantation without in vivo T-cell depletion for children with MHC class II deficiency

• Published in: Journal of allergy and clinical immunology Vol. 141; no. 6; pp. 2279 - 2282.e2
• Main Authors: Elfeky, Reem, Furtado-Silva, Juliana M., Chiesa, Robert, Rao, Kanchan, Lucchini, Giovanna, Amrolia, Persis, Worth, Austen, Gaspar, Bobby, Qasim, Waseem, Veys, Paul
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.06.2018; Elsevier Limited
• Subjects: Adenoviruses; Anemia; Antigens; Child, Preschool; Children; Cord blood; Cord Blood Stem Cell Transplantation - methods; Cytomegalovirus; Female; Histocompatibility Antigens Class II; Humans; Immunoglobulins; Immunologic Deficiency Syndromes - therapy; Infant; Lymphocyte Depletion - methods; Lymphocytes; Lymphocytes T; Major histocompatibility complex; Male; Patients; Steroids; Tetanus; Transplantation; Umbilical cord; Viral infections
• ISSN: 0091-6749; 1097-6825; 1097-6825
• DOI: 10.1016/j.jaci.2017.10.051

To the Editor: Without hematopoietic stem cell transplant (HSCT), patients with MHC class II generally accumulate morbidities and have poor long-term survival.1 After transplant, immunity usually improves, although some patients continue to require immunoglobulin replacement therapy, and MHC class II antigen presentation in epithelial tissue may still be defective. Previous reports of cord transplants for MHC class II deficiency described in vivo T-cell depletion in 4 of 5 transplants.2,3,5-7 Disseminated viral infections and severe bacterial sepsis were noted, and 1 patient died of chemotherapy-related toxicity. In murine experiments, it has been shown that donor-derived thymocytes expressing MHC class II can facilitate CD4+ T-cell selection in the thymus.8 A major consequence of using T-replete donations is an increased risk of GvHD and this was problematic in 2 of our subjects, and may be a particular concern in the context of preexisting enteropathy and poor host immunity against viruses. [...]pharmacokinetic studies of rabbit anti-thymocyte globulin may allow dose-targeted host T-cell depletion while preserving donor- derived T-cell engraftment, which appears critical in treating MHC class II deficiency.