Conquering Infant and Young Children Medulloblastoma in Resource-Limited Settings: Challenges, Opportunities, and Treatment Outcomes.
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BACKGROUND: Infant and young children medulloblastoma poses significant treatment challenges due to high recurrence rates and the severe neurodevelopmental risks associated with craniospinal irradiation (CSI). Outcome data from low-middle-income countries (LMICs) remain scarce. METHODS: A retrospective chart review was conducted for children (age <5 years) diagnosed with medulloblastoma at King Hussein Cancer Center, Jordan, between 2010 and 2023. RESULTS: Twenty-seven patients were identified. The median age was 2 years (range, 0.5-4.3 years) with 78% males, and over half (52%) presented with metastatic disease. Twelve patients (44%) had ≤ 1.5 cm2 residual tumor. Seventeen patients (63%) required ventriculoperitoneal shunt placement. Seven (26%) developed postoperative complications. Ten tumors were of classic histology, eight desmoplastic, six anaplastic, and three MBEN. Sixteen tumors (59%) were molecularly subgrouped: SHH (fourteen), Group-3 (two). Patients received various treatment protocols: HeadStart 1&2-like protocols (seventeen), SKK-92 (eight), and BBSFOP (two). Although 21 patients were planned for autologous stem cell transplantation (ASCT), only 13 received it (three received SKK-92 due to financial constraints, two prematurely stopped therapy, two developed early tumor progression, and one shifted to BBSFOP protocol due to chemotherapy toxicity). At a median follow-up of 7.4 years (range, 2.5-16 years), 17 patients (63%) are alive. Twelve patients (44%) are alive in CR1 without radiotherapy (five of whom also avoided ASCT), three in CR2 after CSI-36 Gy, one in CR3 after ASCT then CSI-23.4 Gy, and one patient lost to follow-up after salvage CSI-36 Gy. Two patients developed secondary tumors: one died from high-grade glioma and one developed thyroid carcinoma. Two survivors needed cataract surgery, and four had hearing loss. Ten survivors need hormonal supplements. CONCLUSIONS: Some infants and young children with medulloblastoma can achieve durable cure without ACST or radiotherapy, even in LMICs. While these therapies remain critical salvage options, lifelong comprehensive support is imperative to optimize survival and quality of life.