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
• ISSN: 1083-8791; 1523-6536
• DOI: 10.1016/j.bbmt.2016.12.619

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 are more commonly seen in those with poor long-term outcomes. • Use of pre-HCT conditioning is controversial in SCID, and further studies are needed to determine the best agents and doses to use in young infants, with particular attention to those with radiosensitive SCID who are more susceptible than others to experience poor growth and other adverse effects with use of alkylator-based chemotherapy. • B cell engraftment is associated with higher likelihood of B cell function in most forms of SCID.