Alina Sadaf

and 3 more

Background Pediatric rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children. In low- and middle-income countries (LMIC) such as Pakistan, problems with delayed diagnosis, decreased access to care, treatment abandonment and lack of multidisciplinary care may lead to poor outcomes. Methods A retrospective chart review was conducted from January 1, 2017, to January 1, 2022 to identify patients with low- and intermediate-risk RMS at a tertiary cancer center in Pakistan. Curative treatment was given per the standard arms of the Children’s Oncology Group (COG) studies ARST0331 and ARST1431. Results Forty-eight patients were eligible for survival analysis. The most common tumor region was parameningeal (31.3%). The 3-year overall survival (OS) was 100% for the 14 low-risk patients and 76.5% for the 34 intermediate-risk patients. The worst performing tumor regions were “other sites”. The 3-year event-free survival (EFS) for radiation alone (55%) as a local control modality was worse than for surgery alone (66.7%) or a combination of surgery and radiation (66.7%). One-third of the patient cohort had severe malnutrition (< 5th percentile by weight) which was associated with lower 3-year OS of 75% vs 85.7%. Eight patients (17%) died (3 due to sepsis, 5 due to disease progression), 11 (23%) abandoned treatment and 15 (31%) had progressive disease. Conclusions There was a preponderance of intermediate-risk RMS in this cohort, with 3-year OS approaching that of high-income countries but with a lower PFS and EFS. Sepsis-related deaths, treatment abandonment, and malnutrition remain significant challenges.