Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation

• Published in: Journal of allergy and clinical immunology Vol. 139; no. 4; pp. 1282 - 1292
• Main Authors: de la Morena, M. Teresa, Leonard, David, Torgerson, Troy R., Cabral-Marques, Otavio, Slatter, Mary, Aghamohammadi, Asghar, Chandra, Sharat, Murguia-Favela, Luis, Bonilla, Francisco A., Kanariou, Maria, Damrongwatanasuk, Rongras, Kuo, Caroline Y., Dvorak, Christopher C., Meyts, Isabelle, Chen, Karin, Kobrynski, Lisa, Kapoor, Neena, Richter, Darko, DiGiovanni, Daniela, Dhalla, Fatima, Farmaki, Evangelia, Speckmann, Carsten, Español, Teresa, Shcherbina, Anna, Hanson, Imelda Celine, Litzman, Jiri, Routes, John M., Wong, Melanie, Fuleihan, Ramsay, Seneviratne, Suranjith L., Small, Trudy N., Janda, Ales, Bezrodnik, Liliana, Seger, Reinhard, Raccio, Andrea Gomez, Edgar, J. David M., Chou, Janet, Abbott, Jordan K., van Montfrans, Joris, González-Granado, Luis Ignacio, Bunin, Nancy, Kutukculer, Necil, Gray, Paul, Seminario, Gisela, Pasic, Srdjan, Aquino, Victor, Wysocki, Christian, Abolhassani, Hassan, Dorsey, Morna, Cunningham-Rundles, Charlotte, Knutsen, Alan P., Sleasman, John, Costa Carvalho, Beatriz Tavares, Condino-Neto, Antonio, Grunebaum, Eyal, Chapel, Helen, Ochs, Hans D., Filipovich, Alexandra, Cowan, Mort, Gennery, Andrew, Cant, Andrew, Notarangelo, Luigi D., Roifman, Chaim M.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.04.2017; Elsevier Limited
• Subjects: Adolescent; Adult; Agammaglobulinemia; Age; Allergy and Immunology; Antibody response; Aplasia; Bacillus Calmette-Guerin vaccine; Bacterial infections; BCG; Bile; Bile ducts; Blood cells; Bone marrow; Cancer; CD40 ligand; Central nervous system; Child; Child, Preschool; Children; Cholangitis; Cohort Studies; Collaboration; Computer programs; defects in class-switch recombination; Disease; Female; Follow-Up Studies; hematopoietic cell transplantation; Hematopoietic Stem Cell Transplantation - mortality; Humans; Hyper-IgM Immunodeficiency Syndrome - mortality; Hyper-IgM Immunodeficiency Syndrome - therapy; Hypersensitivity; Immunoglobulin G; Immunoglobulin M; Immunoglobulins; Infant; Infections; Kaplan-Meier Estimate; Karnofsky/Lansky scores; Ligands; Liver diseases; long-term outcomes; Lymphocytes B; Male; Malignancy; Medical prognosis; Middle Aged; Mortality; Mutation; Nervous system; Neurodegeneration; Neutropenia; Physicians; Pneumonia; Primary immunodeficiencies; primary immunodeficiency; Proportional Hazards Models; Retrospective Studies; Stem cell transplantation; Stem cells; Studies; Time; Transplants & implants; Well being; Wiskott-Aldrich syndrome; X-linked hyper-IgM syndrome; Young Adult; United States > US; XHIGM; defects in class-switch recombination; GVHD; PID; X-linked hyper-IgM syndrome; CD40LG; hematopoietic cell transplantation; CD40 ligand; primary immunodeficiency; HCT; REDCap; long-term outcomes; Karnofsky/Lansky scores; Graft-versus-host disease; Research Electronic Data Capture; Primary immunodeficiency disease

X-linked hyper-IgM syndrome (XHIGM) is a primary immunodeficiency with high morbidity and mortality compared with those seen in healthy subjects. Hematopoietic cell transplantation (HCT) has been considered a curative therapy, but the procedure has inherent complications and might not be available f...