Approach to Treating Solid Tumors in Children
All children with newly suspected or confirmed solid tumors must be immediately referred to a specialized pediatric cancer center for diagnosis and treatment—never initiate biopsy or surgery at a non-specialized hospital, as this frequently results in incorrect diagnosis and need for repeat procedures. 1, 2
Immediate Referral and Diagnostic Pathway
Refer urgently to a pediatric cancer center where the diagnostic biopsy should ideally be performed, as solid tumors in children are rare and incorrect histologic diagnoses occur frequently at non-specialized hospitals. 1, 2
The pediatric cancer center has necessary facilities for immunochemistry and molecular techniques required for accurate diagnosis, with mandatory pathology review by a pediatric oncology pathologist experienced in pediatric solid tumors. 1, 2
Look specifically for genetic alterations and biologic markers (such as NTRK fusions, BRAF V600E mutations, N-MYC amplification in neuroblastoma, 1p/16q deletions in Wilms tumor) as these determine risk stratification and treatment intensity. 1, 3
Multidisciplinary Team Structure
Treatment must be coordinated by a board-certified pediatric hematologist/oncologist who serves as the central coordinator, supported by: 1, 2
- Pediatric surgical oncologist (general surgery, urology, neurosurgery, or orthopedics depending on tumor location) 1
- Pediatric oncology pathologist 1, 2
- Pediatric radiation oncologist with specific training in pediatric oncology 1
- Specialized pediatric oncology nurses, social workers, pharmacists, nutritionists, and psychologists 1, 2
- Regular multidisciplinary tumor board review, which changes clinical management in 64% of cases where interpretation alterations occur 1, 4
Treatment Algorithm by Modality
Surgical Management
Timing of surgery depends on tumor type and resectability—inappropriate upfront resection leads to unnecessary morbidity, incomplete resection, and delays in systemic chemotherapy critical for metastatic disease. 5
For resectable tumors at diagnosis: Perform surgery by pediatric surgical oncologist at the cancer center, with tissue obtained for comprehensive molecular profiling. 1, 2
For unresectable or large symptomatic tumors: Initiate neoadjuvant chemotherapy first, then reassess for delayed surgical resection after tumor shrinkage. 1, 5
Minimally invasive procedures (laparoscopy, thoracoscopy) should only be performed by pediatric surgeons trained in these techniques. 1
Chemotherapy Approach
Risk-based chemotherapy protocols individualize treatment intensity based on tumor biology, stage, age, and molecular markers: 3
Low-risk tumors: Reduced-intensity chemotherapy to minimize long-term morbidity while maintaining survival 3
Intermediate-risk tumors: Standard multi-agent chemotherapy regimens 3
High-risk tumors: Intensive multi-agent chemotherapy, often with stem cell rescue 3
Specific FDA-approved chemotherapy agents for pediatric solid tumors include: 6
- Dactinomycin (actinomycin D) for Wilms tumor, rhabdomyosarcoma, and Ewing sarcoma as part of multi-phase combination chemotherapy 6
Targeted Therapeutics
For tumors with specific molecular alterations, targeted therapy should be prioritized: 1, 7
NTRK fusion-positive tumors: Larotrectinib (93% objective response rate in pediatric patients) or entrectinib (58% objective response rate), or repotrectinib for patients ≥12 years with progressive disease 1
BRAF V600E-mutated high-grade gliomas: Vemurafenib or dabrafenib plus trametinib (combined BRAF/MEK inhibition shows superior tumor reduction and disease stabilization) 1
Other commonly used targeted agents: Imatinib, bevacizumab, and rapamycin have shown mostly little to no severe adverse events with at least partial responses in subsets of patients 7
Radiation Therapy
Proton beam therapy (PBT) should be prioritized for pediatric solid tumors when radiation is indicated, as it provides improved dose distribution and decreased incidence of second cancers compared to conventional photon radiation. 1
Radiation oncologists with specific training and interest in pediatric oncology must deliver treatment, with careful attention to growth plate sparing and neurocognitive preservation. 1
Clinical Trial Enrollment
Enrollment in Children's Oncology Group (COG) clinical trials must be prioritized to provide access to state-of-the-art treatment protocols specifically designed for pediatric malignancies. 1, 2
- Membership or affiliation with COG provides access to cutting-edge protocols and ensures standardized data collection for ongoing research. 1
Ongoing Care Coordination
All treatment must be prescribed and initiated at the pediatric cancer center, with selected aspects of maintenance care potentially continued with the primary care pediatrician under continuing oversight. 1, 2
The primary care pediatrician maintains an important supportive role requiring excellent regular communication between oncologist and pediatrician. 1
Establish a formal program for long-term multidisciplinary follow-up at the original treatment center to monitor for late effects of therapy. 1
Critical Pitfalls to Avoid
Never perform initial biopsy or surgery at a non-specialized hospital—this results in incorrect diagnosis in a substantial proportion of cases, inadequate tissue for molecular studies, and need for repeat procedures that delay definitive treatment. 1, 2
Never delay referral to a pediatric cancer center—early detection, accurate diagnosis, and expeditious initiation of appropriate treatment are critical for achieving the 80% cure rate possible with modern multidisciplinary approaches. 1, 2, 8
Never initiate chemotherapy without comprehensive molecular profiling—risk stratification based on genetic alterations fundamentally determines treatment intensity and may identify targetable mutations. 1, 3
Avoid aggressive upfront resection of unresectable tumors—neoadjuvant chemotherapy often converts unresectable tumors to resectable, and premature surgery causes unnecessary morbidity and delays systemic therapy. 5
Recurrent or Progressive Disease
For recurrent/progressive disease with median overall survival <6 months, prioritize clinical trials of novel targeted therapeutics or immunotherapies over conventional salvage chemotherapy. 1
Palliative care consultation should be integrated early for symptom management and quality of life optimization, not reserved for end-of-life care. 1
Expected Outcomes
Comprehensive multidisciplinary treatment at pediatric cancer centers achieves superior outcomes, with approximately 80% of children with acute lymphoblastic leukemia, non-Hodgkin lymphoma, brain tumors, rhabdomyosarcoma, Wilms tumor, and Ewing sarcoma successfully treated. 1, 2, 8
Overall survival for children with malignant solid tumors has improved from 56% to 75% over the past 25 years through multidisciplinary cooperative studies using combined surgery, chemotherapy, and radiation. 3