“No Regrets:” The Pediatric Experience with Chimeric Antigen Receptor (CAR) T Cell Therapy

Outcomes 1. Utilizing a case-based approach, participants will describe symptoms faced by families of children treated with CAR T cell therapy, with application to future supportive care.2. Utilizing a case-based approach, participants will describe decision making considerations faced by families of children treated with CAR T cell therapy, with application to future supportive care. Key Message Fatigue, pain, and nausea are the most prevalent symptoms reported by patients. Even among those without disease response, families would choose this treatment again because of the favorable symptom profile. Introduction/Context Chimeric Antigen Receptor (CAR) T cell therapy, a type of immunotherapy, provides promising outcomes in relapsed B Acute Lymphoblastic Leukemia (ALL), however has high rates of toxicity and relatively poor long-term survival.1 Perceptions of treatment-related symptoms are based on clinical trial toxicity reports;2 the patient perspective remains understudied. Objectives Utilizing a case-based approach, participants will describe decision making and symptoms faced by families of children treated with CAR T cell therapy, with application to future supportive care. Methods English or Spanish-speaking patients (ages 8-25 years) undergoing CAR T-cell therapy for any malignancy and their primary caregiver were recruited from Seattle Children's Hospital (SCH), St Jude Children's Research Hospital (SJ), and the Pediatric Oncology Branch of the National Cancer Institute (NCI). Both patient and caregiver completed semi-structured dyadic interviews 3-months post-treatment. We used directed content analysis for codebook development3 and thematic network analysis for inductive qualitative analysis.4 Results Twenty families completed interviews (13 patients, 15 parents). Patients were a median age 16.5 years, predominantly female (65%), white (75%), and diagnosed with ALL (75%). Organizing themes included “living with uncertainty,” “symptoms were… better than expected,” and “no regrets.” When families were asked to describe the “most challenging part of treatment,” most described “the unknown.” They also reported “the symptoms really weren't that bad,” even among patients hospitalized for severe toxicity events. Fatigue, pain, and nausea were the most reported symptoms. Importantly, none of the families would have chosen a different therapy, if given another opportunity. Conclusions Although physical symptoms were largely tolerable, recognizing supportive care opportunities remains imperative, particularly for symptom management and coping with an uncertain future.5 Keywords Managing Suffering and Distress / Disease specific management