Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Severe Combined Immunodeficiency Patients: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT

• Published in: Biology of blood and marrow transplantation Vol. 23; no. 3; pp. 379 - 387
• Main Authors: Heimall, Jennifer, Puck, Jennifer, Buckley, Rebecca, Fleisher, Thomas A, Gennery, Andrew R, Neven, Benedicte, Slatter, Mary, Haddad, Elie, Notarangelo, Luigi D, Baker, K. Scott, Dietz, Andrew C, Duncan, Christine, Pulsipher, Michael A, Cowan, Mort J
• Format: Journal Article Conference Proceeding
• Language: English
• Published: United States Elsevier Inc 01.03.2017
• Subjects: Adolescent; Adult; B-Lymphocytes; Child; Child, Preschool; Graft Survival - immunology; Hematology, Oncology and Palliative Medicine; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Humans; Infant; Late effects; Pediatric allogeneic bone marrow transplantation; Research - trends; Severe combined immunodeficiency; Severe Combined Immunodeficiency - therapy; T-Lymphocytes - pathology; Time Factors; Transplantation Conditioning - methods; Young Adult; Severe combined immunodeficiency; Pediatric allogeneic bone marrow transplantation; Late effects

Highlights • Survival after HCT for SCID is >70% and can be higher in those treated prior to the onset of infections. • MRD is the best source for HSC, but MMRD, MRD, and UCB have all been successfully used to treat SCID. • Autoimmunity and cGVHD are seen in a minority of patients overall, but ar...