Affiliation:
1. Division of Pediatric General and Thoracic Surgery Cincinnati Children's Hospital Medical Center University of Cincinnati Cincinnati Ohio USA
2. Department of Biostatistics College of Public Health and Health Professions and College of Medicine University of Florida Gainesville Florida USA
3. Department of Surgery Howard University Washington District of Columbia USA
4. Department of Radiation Oncology Memorial Sloan Kettering Cancer Center New York New York USA
5. Department of Radiation Oncology Massachusetts General Hospital/Harvard Medical School Boston Massachusetts USA
6. Department of Pediatrics Texas Children's Cancer and Hematology Centers Baylor College of Medicine Houston Texas USA
7. Division of Pediatric Surgery University of Kentucky Medical College Lexington Kentucky USA
Abstract
AbstractPurposeTo describe clinical features, risk factors, and outcomes of patients with perineal and perianal rhabdomyosarcoma.MethodsThe records of 51 patients (38 perineal and 13 perianal) enrolled on Children's Oncology Group clinical trials between 1997 and 2012 were reviewed.ResultsAt presentation, 53% were female, 65% were older than 10 years of age, 76% were alveolar histology, 76% were more than 5 cm, 84% were invasive, 65% were regional node positive by imaging, 49% were metastatic, only 16% were grossly resected upfront, and 25% of patients had a delayed excision. At a median follow‐up of 6.13 years, estimated 5‐year event‐free survival (EFS) was 38% [22.17%–53.38%], and overall survival (OS) was 42% [26.66%–58.21%]. The rates of local, regional, and distant failure were 15.6%, 13.7%, 43.1%, respectively; all failures ultimately died. By univariate analysis, only age more than 10 years negatively impacted 5‐year EFS (p = .023) and OS (p = .09), and IRS Group also impacted OS (p = .043). In Cox proportional hazards model, neither of these variables were significant after adjusting for other factors.ConclusionPatients with perineal and perianal rhabdomyosarcoma have a poor overall prognosis, probably related to poor patient and disease characteristics at presentation.
Funder
St. Baldrick's Foundation