Vincristine, irinotecan, and temozolomide in children and adolescents with relapsed rhabdomyosarcoma
Relapsed rhabdomyosarcoma remains a disease with dismal prognosis in need of new treatment options. The addition of temozolomide to vincristine and irinotecan (VIT) in relapsed Ewing sarcoma and in two small studies of patients with relapsed RMS showed promise with improved response rates.
This multicenter retrospective study describes the progression-free survival of 19 patients from 5 tertiary care centers with relapsed RMS who received VIT at first or subsequent relapse. It is the largest series to date of children and adolescents treated with VIT for relapsed RMS.
VIT was used as first, second, third, or fourth line of therapy. Of 19 patients with median age 8 years (range 2-17), 4 received VIT as adjuvant therapy post surgery for local control and were not included in the response analysis. Of the 15 evaluable patients, none had complete or partial response, 5 had stable disease, and 11 had progressive disease. At median follow-up of 8 months, 2 patients were alive without disease, 3 were alive with disease, and 14 patients died or progressive disease. PFS at 3 months was 23%.
Small sample size, limited by quality of information documented/collected due to retrospective analysis, no central radiology/pathology review, interpatient variability in dosing of chemo, no toxicity data recorded. Physicians chose this regimen rather than randomly assigning patients to it so there may be selection bias.