TY - CHAP M1 - Book, Section TI - Hematopoietic Stem Cell Transplantation for Primary Immune Deficiencies A1 - Notarangelo, Luigi D. A2 - Kline, Mark W. PY - 2018 T2 - Rudolph's Pediatrics, 23e AB - Primary immune deficiencies (PIDs) comprise more than 250 distinct disorders that affect the immune system’s development, function, and/or homeostasis. Inability to control infections represents a major challenge in several forms of PID, leading to increased incidences of mortality early in life. Furthermore, immune dysregulation, defined as disorders of immune homeostasis presenting with autoimmunity or inflammatory disease, is increasingly being recognized as an important manifestation of various forms of PID and may also affect quality and duration of life. In the majority of cases, PIDs result from genetic defects that are intrinsic to the hematopoietic system. When associated with a poor prognosis, such forms of PID can be treated and cured with allogeneic hematopoietic stem cell transplantation (HCT). However, despite continuous advances in donor selection, development of less toxic chemotherapy preparatory regimens, supportive care, and prevention of transplant-related complications, mortality and late effects continue to affect the outcomes of patients with PIDs treated with HCT. In this chapter, we will review the current status of HCT for PIDs and discuss possible developments in the field. SN - PB - McGraw-Hill Education CY - New York, NY Y2 - 2024/03/29 UR - accesspediatrics.mhmedical.com/content.aspx?aid=1182931675 ER -