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From Misdiagnosis to Precision Medicine: Improving Brain Tumor Care for Children in Low- and Middle-Income Countries

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Original medical illustration for: From Misdiagnosis to Precision Medicine: Improving Brain Tumor Care for Children in Low- and Middle-Income Countries

Table of Contents

Key Points

  • In high-income countries, more than 90% of children with pLGG survive at least 10 years; in certain molecular subtypes, 5-year survival reached 100% in recent trials.
  • In low- and middle-income countries, misdiagnosis rates for pediatric central nervous system tumors range from 18% to 32%, and many hospitals lack molecular tests.
  • A survey of 22 centers in Francophone Africa found 45% lacked immunohistochemistry and 86% had no molecular diagnostic tools.
  • Oral MEK and BRAF inhibitors offer fewer hospital visits, no permanent central venous access, lower infection risk, better school attendance, and avoidance of hair loss and severe myelosuppression.
  • International tumor boards altered or refined management in about 32% of complex cases; a three-phase roadmap proposes basic diagnostics, telepathology, then local targeted assays.

Background: Why Where a Child Lives Matters

In modern cancer care, a patient's geographic location—often described as their "postal code"—can influence survival more than their genetic makeup. This disparity is especially striking for pediatric low-grade gliomas (pLGG), the most common brain tumors in children.

In high-income countries (HICs), pLGG is generally considered a slow-growing, chronically manageable brain tumor. The 10-year overall survival (OS) rate in HICs consistently exceeds 90%. Modern treatments such as MEK inhibitors have further improved these outcomes. For example, recent long-term results from selumetinib trials show 5-year overall survival rates of 100% for patients whose tumors have KIAA1549-BRAF fusions or BRAF V600E mutations.

In low- and middle-income countries (LMICs), the picture is very different. These tumors remain a major cause of illness and death in children. This gap is not primarily due to biological differences in the tumors themselves. Instead, it stems from structural barriers that delay diagnosis and block access to treatment.

Global data show a direct relationship between how much a government spends on health care each year and how many children survive cancer. More than 90% of childhood cancer deaths occur in resource-limited settings, where health-care systems are often underfunded.

Diagnostic bottlenecks make the problem worse. Comparative needs assessments have found major shortages in histopathology (the laboratory study of diseased tissue). Major referral centers in LMICs may have fewer specialized neuropathologists (brain tissue specialists) than HICs. These centers also often lack essential immunohistochemistry (IHC). IHC is a technique that uses antibodies to detect specific proteins in tissue. These centers also often lack molecular diagnostic markers such as FISH probes. FISH probes are fluorescence in situ hybridization, a test that detects specific gene changes.

Even when a diagnosis is made, treatment is often blocked by financial barriers. One-third (32%) of conventional chemotherapy drugs considered essential by the World Health Organization (WHO) are only available in many LMICs through full out-of-pocket payment. This places families at high risk for catastrophic health spending.

The WHO Global Initiative for Childhood Cancer (GICC) aims for a 60% global survival rate by 2030. Addressing the unique diagnostic and treatment challenges of pLGG is critical to achieving that goal. The GICC CureAll framework provides a tool to organize strategies for reaching global equity.

How This Review Was Conducted

The authors performed a targeted literature review to synthesize evidence on the diagnostic and therapeutic landscape for pLGG. The methodology was designed to find peer-reviewed publications at the intersection of precision medicine, resource-constrained environments, and pLGG clinical management.

Researchers searched major electronic databases, specifically PubMed/MEDLINE and Scopus. The search used combinations of key terms including:

  • Pediatric low-grade glioma
  • Low- and middle-income countries
  • Diagnostic discrepancy
  • MAPK-pathway inhibitors
  • Global health equity

The selection process prioritized data on the structural and clinical hurdles unique to pediatric neuro-oncology in LMICs. Inclusion criteria specifically targeted:

  • Epidemiological evidence documenting baseline rates of diagnostic misclassification and discordance for pediatric central nervous system (CNS) tumors in limited-resource settings
  • Landmark clinical trials and real-world implementation cohorts evaluating the safety, feasibility, and efficacy of targeted MAPK inhibitors
  • Infrastructural assessments for establishing telepathology partnerships and regionalized molecular diagnostic hubs
  • Policy research examining pooled procurement, differential drug pricing, and the integration of pLGG protocols into national cancer plans

Studies not explicitly involving pediatric populations, CNS malignancies, or the socioeconomic and logistical realities of LMIC oncology were excluded.

The authors synthesized the extracted findings thematically. They then applied the WHO GICC CureAll framework to the landscape of pediatric neuro-oncology care in LMICs to identify practical next steps.

The Diagnostic Divide: Why Accurate Diagnosis Is So Hard

Pediatric neuro-oncology has shifted from classifying tumors purely by how they look under a microscope to a sophisticated molecular taxonomy (classification based on genetic and molecular features). The 2021 WHO Classification of CNS Tumors confirmed that molecular profiling—such as identifying BRAF fusions or H3 mutations—is necessary for accurate risk stratification.

However, for most LMICs, the diagnostic "gold standard" remains limited to basic Hematoxylin and Eosin (H&E) staining and limited immunohistochemistry. H&E staining is a basic tissue-staining method that shows cell structure but not molecular details.

This infrastructure gap is particularly severe in Francophone Africa. A recent survey of 22 centers in the region found that nearly half (45%) lack access to immunohistochemistry. An even larger proportion—86%—have no access to molecular diagnostic tools. This leaves patients in 19 of the 22 surveyed centers without the markers now considered essential for modern care.

This high degree of diagnostic uncertainty severely compromises clinical management. It frequently leads to misidentification of tumor grade or type, which drives inappropriate treatment decisions.

Institutional experiences from regional referral centers in the Middle East further illustrate this diagnostic fragility. Central nervous system tumors accounted for 71% of all significant diagnostic disagreements when compared to other pediatric solid tumors. These errors often involve misidentifying tumor grade or type, directly leading to inappropriate treatment escalation or suboptimal management.

In settings where neurosurgical capacity is already stretched thin, an incorrect diagnosis can lead to inappropriate surgery or the use of toxic, ineffective chemotherapy. Both outcomes further strain already limited health-care resources.

Research also suggests a significant "diagnosis bias" exists in low-resource settings. CNS tumors are likely underrepresented in official data because of limited access to advanced imaging such as MRI (magnetic resonance imaging). This pattern is mirrored on a continental scale in Africa. Underdeveloped tracking frameworks and severe diagnostic infrastructure deficits drop the recorded incidence of pLGG far below global baselines. This means patients are frequently missing from registries entirely. The actual number of patients who could benefit from these life-changing agents is expected to be much larger than what is currently reported.

The Therapeutic Paradox: A Single-Pathway Disease

Pediatric low-grade gliomas are fundamentally characterized as a "single-pathway disease." Constitutive activation (constant switching on) of the mitogen-activated protein kinase (MAPK) signaling cascade occurs in 90% to 100% of all cases.

This molecular landscape is predominantly driven by targetable BRAF alterations. Specifically:

  • The KIAA1549-BRAF fusion accounts for 60–70% of cases
  • The BRAF V600E mutation accounts for 15–20% of cases
  • Other drivers include NF1 inactivation, FGFR1 variants, and rare but highly actionable fusions involving NTRK, ALK, or ROS1 (each less than 5%)

Because nearly all pLGG tumors depend on this pathway, the emergence of MAPK inhibitors—specifically MEK and BRAF inhibitors—represents a transformative turning point for pediatric neuro-oncology. The clinical efficacy of these agents has been validated in landmark trials, including CDRB436G2201, PBTC-029B, and FIREFLY-1. These trials demonstrated sustained response rates in relapsed or refractory pLGG.

Historically, novel cancer therapies have been considered too complex for resource-constrained environments. However, targeted agents such as selumetinib and tovorafenib are well-suited for these settings because of their "infrastructure-light" delivery profile.

Traditional cytotoxic chemotherapy requires rigorous intravenous access, specialized infusion monitoring, and intensive supportive care for neutropenic complications (dangerously low white blood cell counts). In contrast, oral targeted therapies are typically managed in an outpatient setting.

This decentralized model potentially reduces the need for central venous access. It also minimizes the risk of line-associated complications such as bacteremia (blood infection) or febrile neutropenia, which might otherwise require inpatient management. The relatively manageable toxicity profile of MAPK inhibitors may also support toxicity mitigation, potentially reducing hospitalizations in regions where tertiary care centers are remote or overextended.

Real-world implementation data from LMICs support these decentralized benefits. Institutional cohorts from India and Jordan demonstrate that transitioning to oral MEK and BRAF inhibitors significantly reduces the frequency of hospital visits. It also increases school attendance and yields high treatment satisfaction. These benefits come from avoiding alopecia (hair loss) and severe myelosuppression (bone marrow suppression), despite manageable, expected skin toxicities such as acneiform rash and paronychia (nail bed infection).

Nevertheless, a substantial implementation gap persists. Despite their low-intensity administration requirements, these life-saving agents remain largely inaccessible. The barriers include:

  • Prohibitive costs
  • Lack of inclusion on the WHO Model List of Essential Medicines
  • A deficit in the molecular diagnostic infrastructure necessary to identify eligible patients

The Precision Diagnosis Paradox

The clinical promise of MAPK-pathway inhibitors in resource-constrained environments introduces a profound operational contradiction. The authors call this the "precision diagnosis paradox."

On one hand, oral targeted agents such as selumetinib or tovorafenib present a highly feasible, "infrastructure-light" therapeutic option for LMICs. These oral targeted therapies eliminate the intensive clinical dependencies of conventional cytotoxic chemotherapy. Those dependencies include permanent central venous access, strict inpatient supportive care, and high-frequency toxicity monitoring. These oral targeted therapies offer a highly decentralized, outpatient-manageable treatment model.

On the other hand, the clinical safety, regulatory approval, and therapeutic efficacy of these agents are strictly contingent on precise molecular stratification. Clinicians cannot safely or ethically prescribe allele-specific BRAF inhibitors (such as dabrafenib) or MEK inhibitors. Clinicians must first confirm the presence of specific driving alterations, such as the KIAA1549-BRAF fusion or the BRAF V600E mutation.

This requirement creates a systemic bottleneck. A low-intensity therapeutic model cannot be deployed because its initiation is gated by highly complex, centralized, and costly diagnostic technologies. These technologies remain inaccessible to 80% of children living in resource-limited countries.

To resolve this paradox and prevent the wholesale exclusion of LMIC patients from precision oncology, the global neuro-oncology community must reject binary diagnostic paradigms. These paradigms demand either comprehensive next-generation sequencing (NGS) or nothing. Instead, the authors champion pragmatic, tiered diagnostic pathways.

Rather than waiting for universal implementation of genomic sequencing or DNA methylation profiling, LMICs can use highly accessible, localized "bridging technologies" to guide targeted therapy safely. For example:

  • Immunohistochemistry (IHC) using the monoclonal antibody clone VE1 offers a highly sensitive, rapid, and cost-effective surrogate for detecting the BRAF V600E mutation. IHC relies on basic pathology infrastructure that is already operational or easily scalable in regional referral centers. Because of this, VE1 staining can immediately identify candidates for BRAF-targeted therapies without requiring molecular extraction.
  • For the more prevalent KIAA1549-BRAF fusion, clinical networks can implement low-cost, targeted molecular platforms such as reverse transcription-polymerase chain reaction (RT-PCR) or single-plex fluorescence in situ hybridization (FISH). These targeted assays bypass the immense bioinformatics and financial burdens of NGS while maintaining high diagnostic accuracy.

As regional molecular hubs and telepathology programs are progressively scaled under the WHO's CureAll framework, these intermediate, assay-specific bridging strategies represent the only realistic pathway. They align clinical capability with therapeutic opportunity, ensuring that patient selection remains both safe and equitable.

Bridging the Equity Gap: The CureAll Framework

To translate current scientific advancements into tangible clinical reality within LMICs, a comprehensive, multi-tiered strategic framework is required. The GICC uses the CureAll framework as its primary operational strategy to address global inequities in pediatric oncology. pLGG is one of six priority cancers in this framework.

The CureAll approach is structured around four strategic pillars:

  1. Centers of excellence and care networks
  2. Universal health coverage
  3. Regimens for management (standardized treatment protocols)
  4. Evaluation and monitoring

These pillars are supported by three core enablers—advocacy, leveraged financing, and linked governance. These enablers ensure that childhood cancer is integrated into national health agendas and remains sustainable.

This strategic alignment is increasingly reflected in grassroots efforts of several LMICs to design and implement standardized, resource-stratified clinical guidelines tailored to their health-care ecosystems. For instance, collaborative capacity-building initiatives in Pakistan have demonstrated how establishing dedicated subspecialty pediatric neuro-oncology services and unified regional management protocols can drastically optimize clinical pathways. This holds true even when operating under severe radiotherapy or neurosurgical limitations.

Similarly, cooperative regional networks across Africa have initiated baseline mapping studies. These studies systematically document pLGG outcomes and infrastructure gaps. They establish a data-driven foundation for the rollout of GICC-aligned national protocols.

These national and regional efforts are further supported by real-world clinical evidence from pioneering institutional protocols in India and Jordan. Together, they prove that the systematic adaptation of standardized regimens is not only feasible but represents an indispensable prerequisite for improving survival rates and achieving diagnostic and therapeutic equity.

Real-World Collaborative Initiatives in LMICs

Beyond MAPK inhibitors as a specific innovation in pLGG care, the persistent survival disparity in pediatric neuro-oncology is a failure of clinical implementation, resource distribution, and severe diagnostic latency (delay).

Reducing diagnostic volatility through formalized neuropathology twinning and telepathology programs is a primary requirement. By establishing collaborative partnerships between LMIC centers and HIC departments, real-time expert consultation and secondary reviews can be institutionalized.

Crucially, the expansion of bilateral twinning initiatives into multi-center regional teleconferences has demonstrated that expert second opinions can significantly optimize clinical management. These second opinions altered or refined treatment modalities in 32% of complex cases. This intervention is of paramount importance given that the majority of LMIC oncologists manage these cases without formal subspecialty training in neuro-oncology.

This virtual collaborative paradigm is further enhanced by large-scale, international tumor boards. These boards successfully bridge resource gaps, enhance diagnostic precision, and guide therapeutic decision-making. Notably, a substantial proportion of consultations in these global forums are directed toward pLGG (23.4%) and neurofibromatosis-associated tumors (32.7%).

Diagnostic standardization must also be prioritized through the procurement of "minimum essential" immunohistochemical panels. These panels should incorporate markers such as GFAP, Ki-67, BRAF V600E, and H3K27M to serve as the baseline diagnostic threshold.

To explicitly address early diagnostic delays before patients reach these centers, the authors recommend incorporating frontline diagnostic heuristics. These include the "LOW-OR-PAY" clinical mnemonic or public-professional awareness frameworks. Such frameworks are modeled after the UK's "HeadSmart" campaign, which successfully reduced the median total diagnostic interval from 14 weeks to 6.7 weeks. These approaches are vital to accelerating the path to neuroimaging.

To overcome resource fragmentation, the authors propose adopting a "Hub-and-Spoke" model. In this model, centralized regional molecular diagnostic hubs concentrate technical expertise. These hubs use economy of scale to reduce the per-test costs of high-complexity assays, including Polymerase Chain Reaction (PCR) and Next-Generation Sequencing (NGS).

Such structured networks and specialized care delivery models mirror the documented success of dedicated pediatric neuro-oncology services (DPNOS) in LMIC settings. This institutional paradigm shift is compellingly demonstrated by a landmark single-institution experience in Pakistan, where the formal establishment of a DPNOS dramatically optimized clinical metrics.

Empirical data indicate that transitioning from generalized oncology care to dedicated subspecialty teams can dramatically expand referral volumes. These teams are integrated with formalized cross-institutional networks and memorandums of understanding. These networks circumvent local infrastructure deficits such as radiotherapy or neurosurgical limitations. It also elevates the proportion of pediatric patients treated with curative intent and significantly improves patient overall survival rates.

The table below summarizes collaborative initiatives and clinical implementations for pLGG management in LMICs:

  • Pakistan: Establishment of Dedicated Pediatric Neuro-Oncology Services (DPNOS) and implementation of unified regional management protocols. Outcomes included expanded patient referral volumes, an elevated proportion of pediatric patients treated with curative intent, and significantly improved patient overall survival rates.
  • Africa: Multicenter baseline mapping studies across regional networks and diagnostic resource surveys across 22 centers in Francophone Africa. Outcomes included documented critical baseline disparities (45% of surveyed centers lacked IHC and 86% lacked molecular tools) and establishment of a data-driven baseline to guide the rollout of GICC-aligned national protocols.
  • India and Jordan: Real-world, institutional cohort applications of oral targeted MAPK-pathway inhibitors (MEK and BRAF inhibitors). Outcomes included significantly reduced hospital visit frequencies, minimized inpatient stays, elevated school attendance and treatment satisfaction, and avoidance of toxicities like alopecia and severe myelosuppression while maintaining manageable dermatological profiles.
  • Lebanon: Retrospective single-institution reviews of pediatric oncology pathology. Demonstrated that central nervous system tumors carry the highest rate of major diagnostic discrepancies.
  • Global/Regional Networks: Multi-center international pediatric neuro-oncology teleconferences and global tumor boards. Optimally altered or refined clinical management decisions in approximately 32% of complex cases.

A Three-Phase Roadmap for Implementation

The authors propose a three-phase approach to move from misdiagnosis toward precision medicine in LMIC settings.

Phase 1 (Foundation): Health systems establish basic diagnostic accuracy. This includes securing minimum essential immunohistochemistry panels and building pathology capacity.

Phase 2 (Connectivity): Teams formalize neuropathology twinning and telepathology programs to secure continuous expert second opinions.

Phase 3 (Precision Integration): Teams integrate low-cost, targeted molecular assays locally. These include RT-PCR or single-plex FISH for KIAA1549-BRAF fusions. At the same time, they use centralized regional hubs for high-complexity genomic sequencing and DNA methylation profiling.

The transition toward precision medicine also requires a robust strategy for sustainable pharmacotherapy access. The authors advocate for the use of pooled procurement mechanisms. This approach leverages regional economic blocs to aggregate demand and negotiate equitable pricing.

Clinical Implications for Patients and Families

This review has several important implications for families of children with pLGG living in LMICs.

First, getting an accurate diagnosis is the critical first step. Without molecular testing, doctors cannot know whether a child's tumor has a BRAF alteration that could be treated with targeted oral drugs. Families should ask their care team whether molecular testing—even a basic IHC test for BRAF V600E—has been performed.

Second, oral targeted therapies offer significant practical advantages over traditional intravenous chemotherapy. These advantages include:

  • Fewer hospital visits
  • No need for permanent central venous access
  • Lower risk of serious infections
  • Better school attendance
  • Avoidance of hair loss and severe bone marrow suppression

Third, telepathology and international tumor boards can provide expert second opinions that change treatment decisions in about one-third of complex cases. Families should ask whether their hospital participates in any telepathology or international tumor board network.

Fourth, the cost of targeted therapies remains a major barrier. These drugs are not yet on the WHO Model List of Essential Medicines, and many families must pay out of pocket. Advocacy for inclusion in national essential medicine lists and pooled procurement programs is critical.

Limitations of This Review

This is a review article, not a clinical trial. It synthesizes existing evidence rather than generating new primary data. The findings and recommendations are based on the published literature available at the time of writing.

The review does not provide original patient-level data. It also does not include a formal meta-analysis or systematic quality assessment of all included studies. The evidence base for some recommendations—particularly around implementation science and health economics—remains limited.

The authors note that the recorded incidence of pLGG in LMICs is likely underestimated because of limited access to MRI and underdeveloped tracking frameworks. This means the true burden of disease is unknown, and the number of patients who could benefit from targeted therapies is expected to be larger than currently reported.

Finally, the proposed strategies have not been tested in large-scale prospective trials in LMIC settings. Their feasibility and effectiveness will need to be evaluated as they are implemented.

Recommendations and Actionable Advice

Based on this review, the authors and the evidence support the following actions:

  1. For health systems: Establish minimum essential immunohistochemistry panels including GFAP, Ki-67, BRAF V600E, and H3K27M as the baseline diagnostic threshold.
  2. For clinical teams: Formalize neuropathology twinning and telepathology partnerships with HIC centers to secure expert second opinions.
  3. For policymakers: Integrate pLGG protocols into national cancer control plans under the GICC CureAll framework.
  4. For procurement agencies: Use pooled procurement mechanisms through regional economic blocs to negotiate equitable pricing for targeted therapies.
  5. For diagnostic networks: Adopt a "Hub-and-Spoke" model where centralized regional molecular hubs concentrate expertise and reduce per-test costs.
  6. For clinicians: Use pragmatic, tiered diagnostic pathways—starting with accessible bridging technologies like VE1 IHC and RT-PCR—rather than waiting for universal NGS availability.
  7. For advocates: Push for inclusion of targeted therapies on the WHO Model List of Essential Medicines and national essential medicine lists.
  8. For awareness campaigns: Implement public and professional awareness frameworks modeled after the UK's "HeadSmart" campaign to reduce diagnostic delays.

Together, these initiatives can reduce diagnostic errors, expand access to precision medicine, and move LMICs closer to equitable outcomes for children with pLGG.

Frequently Asked Questions

What is pediatric low-grade glioma (pLGG)?

Pediatric low-grade glioma is the most common type of brain tumor in children. It is generally slow-growing and chronically manageable. In high-income countries, more than 90% of children with pLGG survive at least 10 years. In certain molecular subtypes, newer targeted drugs have pushed 5-year survival to 100% in recent trial results.

Why does where a child lives affect pLGG survival?

In low- and middle-income countries, children with pLGG face structural barriers that delay diagnosis and block treatment. Misdiagnosis rates for pediatric central nervous system tumors in these settings range from 18% to 32%. Many hospitals lack basic molecular tests needed to identify children who could benefit from precision medicines. More than 90% of childhood cancer deaths occur in resource-limited settings.

What molecular tests are needed for pLGG, and why?

Molecular tests identify specific gene changes, such as the KIAA1549-BRAF fusion or BRAF V600E mutation, that make a tumor eligible for targeted oral drugs. Without them, doctors cannot know if a child could benefit. A survey of 22 centers in Francophone Africa found 45% lacked immunohistochemistry and 86% had no molecular diagnostic tools.

What are the practical advantages of oral targeted therapies for pLGG?

Oral targeted therapies such as MEK and BRAF inhibitors are taken as outpatient treatment. Compared with traditional intravenous chemotherapy, they mean fewer hospital visits, no need for permanent central venous access, lower risk of serious infections, better school attendance, and avoidance of hair loss and severe bone marrow suppression. Real-world data from India and Jordan support these benefits.

Can a second opinion change my child's pLGG treatment?

Yes. Multi-center international pediatric neuro-oncology teleconferences and global tumor boards optimally altered or refined clinical management decisions in approximately 32% of complex cases. Families should ask whether their hospital participates in any telepathology or international tumor board network, because expert review can change treatment decisions in about one-third of complex cases.

Why are targeted pLGG drugs often not available in low- and middle-income countries?

Barriers include prohibitive costs, lack of inclusion on the WHO Model List of Essential Medicines, and a deficit in molecular diagnostic infrastructure needed to identify eligible patients. One-third (32%) of conventional chemotherapy drugs considered essential by the WHO are available in many low- and middle-income countries only through full out-of-pocket payment, risking catastrophic health spending.

What is the three-phase roadmap for improving pLGG care?

Phase 1 (Foundation): establish basic diagnostic accuracy with minimum essential immunohistochemistry panels and pathology capacity. Phase 2 (Connectivity): formalize neuropathology twinning and telepathology programs for expert second opinions. Phase 3 (Precision Integration): integrate low-cost targeted molecular assays locally, such as RT-PCR or single-plex FISH for KIAA1549-BRAF fusions, while using centralized hubs for complex sequencing.

Source Information

Original article title: "From misdiagnosis to precision medicine: strengthening pediatric neuro-oncology care in LMICs toward equitable pLGG outcomes".

Authors: Baroni LV, George NB, Crotty EE, Bouffet E, Abdelbaki MS.

Author affiliations: Division of Oncology, Hospital J.P. Garrahan, Buenos Aires, Argentina; Division of Hematology, Oncology, Bone Marrow Transplant & Cellular Therapy, Department of Pediatrics, Seattle Children's Hospital, University of Washington, Seattle, WA, United States; Division of Hematology/Oncology, Hospital for Sick Children, Toronto, ON, Canada; Division of Pediatric Hematology, Oncology and Bone Marrow Transplant, St. Louis Children's Hospital, Washington University School of Medicine, St. Louis, MO, United States

Journal: Frontiers in Oncology, Volume 16, Article 1895650

Publication details: Received 30 May 2026; Revised 13 August 2026; Accepted 17 August 2026; Published 26 August 2026

DOI: 10.3389/fonc.2026.1895650

Article type: Review

This patient-friendly article is based on peer-reviewed research. It is intended for educational purposes and should not replace consultation with a qualified health-care provider. Families should discuss any treatment decisions with their child's medical team.