Abstract
Background
Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy and requires prolonged maintenance chemotherapy. Methotrexate (MTX) is a core maintenance agent, typically given weekly for extended durations. In many settings, MTX is only available as tablets, creating administration and adherence challenges in young children who cannot swallow solid dosage forms. Extemporaneously compounded MTX oral suspensions are widely used to address this gap, but clinical data on their tolerability and safety in pediatric oncology remain limited.
Objective
To evaluate the tolerability and safety of an extemporaneously prepared oral methotrexate suspension in pediatric ALL patients during maintenance therapy.
Methods
A prospective observational study was conducted over eight months (February–September 2025) at a comprehensive cancer center in Jordan. Pediatric ALL patients receiving compounded MTX suspension (2.5 mg/mL) were included. Tolerability was assessed using the validated Methotrexate Intolerance Severity Score questionnaire covering gastrointestinal and behavioral symptoms across post-dose, anticipatory, and associative domains. Descriptive analyses were performed using Python.
Results
One hundred patients were included (mean age 4.8 years; 61% male). No patient had renal impairment. Laboratory parameters remained within acceptable ranges. Antiemetics were used in 11%, and 38% had prior MTX tablet exposure. Post-dose stomach pain, nausea, and vomiting occurred in 13%, 18%, and 9%, respectively. Moderate-to-severe intolerance (MISS ≥10) was identified in 9%.
Conclusion
Compounded oral MTX suspension demonstrated favorable tolerability and safety and represents an alternative for pediatric ALL maintenance therapy.
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