Life after HSCT: Survivorship and long-term issues

Hematopoietic stem cell transplant (HSCT) is a curative therapeutic option for not just hematologic malignancies but also many nonmalignant conditions such as beta thalassemia, sickle cell disease, metabolic disorders, and certain primary immunodeficiencies. Continued advances in the HSCT techniques, expansion of indications for HSCT, and tremendous progress in supportive care strategies and management of HSCT-related complications have collectively resulted in an expanding population of survivors of HSCT. Progress and expansion of alternative donor hematopoietic stem cell (HSC) sources (e.g., umbilical cord blood, haploidentical donors) are resulting in a continually increasing amount of HSCTs performed in children per year. These survivors are at risk of developing treatment-related late effects, and two-thirds of the HSCT survivors will develop at least one chronic health condition. The direct impact of these long-term effects on the morbidity and mortality of the HSCT survivors makes risk-based and exposure-related screening for these late effects a critical part of their care. This chapter reviews the most common secondary neoplasms seen in survivors of HSCT during childhood and adolescence. This chapter will also discuss the HSCT-associated long-term complications affecting the cardiac, pulmonary, endocrine, musculoskeletal, renal, and neurocognitive systems. Screening recommendations are also included.