Oral Low-Dose Chemotherapy in Pediatric Oncology: A Realworld Study on Feasibility and Outpatient Adherence

Background: In low- and middle-income countries (LMICs), pediatric oncology care is often limited by the high cost, toxicity, and infrastructure demands of conventional chemotherapy. Oral low-dose chemotherapy (OLDC) offers a potential solution by enabling continuous, home-based treatment with reduced resource dependence. This study evaluates the real-world feasibility, adherence, and outpatient tolerability of OLDC in children with relapsed or refractory malignancies. Methods: This prospective observational study included 133 pediatric patients aged 1–18 years with advanced solid or hematologic cancers, treated at the Departments of Medical Oncology and Pharmacology, Medical College Kolkata. All participants received a standardized oral low-dose chemotherapy protocol comprising rotating cycles of cyclophosphamide, methotrexate, etoposide, and 6-mercaptopurine. Patients were followed monthly for six months to assess treatment completion, adherence, interruptions, and outpatient feasibility. Results: The OLDC regimen was well tolerated and highly feasible in an outpatient setting. Over 92.5% of patients received treatment entirely on an outpatient basis, and 89.4% completed at least three full chemotherapy cycles. Adherence was strong, with only 9.8% of patients experiencing temporary dose interruptions, primarily due to mild, reversible toxicities. No patient required hospitalization solely for drug-related adverse events. Family-reported compliance and physician-recorded follow-up rates were consistently high, suggesting the acceptability and practicality of home-based oral therapy in a resource-constrained environment. Conclusion: This study demonstrates that oral low-dose chemotherapy is both feasible and well tolerated in pediatric patients with advanced cancers, with high adherence and minimal disruption in outpatient settings. These findings support broader adoption of OLDC as a viable therapeutic strategy in LMICs where access to intensive inpatient treatment is limited.