Effects of Palliative Oral Etoposide on Survival and Treatment Burden in Patients With Relapsed/Refractory Pediatric Malignancies

Oral etoposide has been given as palliative treatment for pediatric patients with relapsed/refractory malignancies. There is limited literature regarding the efficacy of oral etoposide for relapsed/refractory pediatric malignancies, and even less on its treatment burden for patients and families. We conducted a single-institution retrospective review evaluating patients with relapsed/refractory pediatric malignancies treated with single-agent oral etoposide over an 11-year period. Clinical efficacy and treatment burden measures were collected. Seventy six patients received at least one dose of oral etoposide. Median event-free survival was 47 days (95% CI: 25-68 d). Median overall survival was 113 days (95% CI: 73-149 d). Forty eight patients (63%) had imaging performed; 20 (41%) had stable disease (SD) lasting 11 to 1286 days (median 134 d) as their best response. There was a reduction in the number of clinic visits ( P =0.02), hospitalizations ( P =0.02), and hospital length of stay ( P =0.01) compared with the most recent prior cancer treatment. Our experience suggests that palliative oral etoposide benefits pediatric patients with relapsed/refractory malignancies based on imaging documenting SD lasting a median of 3 months in a significant proportion of patients, and a reduction in treatment burden compared with their most recent prior cancer treatment.