Moving the needle forward for low- and intermediate-risk pediatric
rhabdomyosarcoma in LMIC: outcomes from a cancer center in Pakistan
Abstract
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.