Differences in the Symptom Experience and Quality of Life of Children and Adolescents Undergoing Hematopoietic Stem Cell Transplantation Compared to Chimeric Antigen Receptor T-Cell Therapy

Background Allogeneic hematopoietic stem cell transplantation (HSCT) and chimeric antigen receptor (CAR) T-cell therapy are therapeutic options for children with recurrent or refractory malignancies. High symptom burden and poor quality of life (QoL) can occur secondary to these therapies. The purpose of this study is to compare the symptom, QoL trajectories of children undergoing allogeneic HSCT to those receiving CAR T-cell therapy. Methods A longitudinal, repeated measures design was used for this multi-site study. English or Spanish-speaking children ages 2-18 years with planned allogenic HSCT or CAR T-cell therapy were eligible. Children completed the Memorial Symptom Assessment Scale (MSAS) and the PedsQL Cancer Module at 4 timepoints: pre-HSCT or CAR T-cell infusion, and day+30, +60, +90 post. Parents completed corresponding parent proxy measures for younger children. Linear mixed effects models were used to test for associations between treatment (CAR-T vs allogenic HSCT) and child self-report outcomes. Results To date, 99 children were enrolled across 4 sites: 77 children received allogeneic HSCT, 22 received CAR T-cell therapy. Child mean age was 8.3 years (SD=4.9), 55.4% were male. Child symptom burden and QoL improved from pre-cell infusion through day +90 (Table 1). Child QoL was lowest and symptom burden was highest pre-cell infusion for children in both treatment groups. Children in the CAR-T group experienced significantly lower symptom burden compared to children undergoing allogeneic HSCT (p Conclusion Children receiving allogeneic HSCT experience more frequent, severe and bothersome symptoms compared to children undergoing CAR T-cell therapy. Routine and comprehensive symptom assessment with multimodal interventions are needed to improve the symptom experience, particularly for children undergoing HSCT. Differences patient-reported outcomes, such as symptom burden, may be considered when discussing therapeutic options with patients and families.