Pilocytic astrocytoma
ICD-10 C71.9 · ICD-11 2A00.0Y&XH29Q5

Pilocytic Astrocytoma in Children Under 18: What to Do When Initial Chemotherapy Has Not Controlled the Tumor

For children under 18 with pilocytic astrocytoma that cannot be completely resected, chemotherapy is typically the first systemic approach. When that chemotherapy fails to achieve sustained radiographic tumor response, a defined next-line protocol applies.

Previous line — failure condition

The prior chemotherapy approach — which may include carboplatin and vincristine in combination, weekly vinblastine, or the TPCV regimen, typically over 12–18 months — was intended to achieve radiographic tumor response without disease progression. When that goal is not reached, escalation to the next treatment line is indicated.

Next-line approach (partial overview)

This protocol involves targeted therapy directed at specific molecular alterations in the tumor. The choice of agent depends on the molecular profile of the disease. The complete selection criteria, sequencing, and agent-specific details are available in the full protocol.

Treatment goals

Radiographic tumor response and freedom from disease progression at 1 year.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1093/neuonc/noac188

The management of pediatric LGG that cannot be completely resected has evolved considerably over the recent decades.

MEK inhibitors and BRAF inhibitors have been successfully used in pediatric LGG patients.

A phase II trial of the MEK inhibitor selumetinib has shown high response rates in patients with recurrent NF1-related LGG and patients with recurrent LGG harboring the KIAA1549:BRAF fusion or BRAF V600E mutation.

A phase I/II trial of the BRAF inhibitor dabrafenib, that enrolled 32 patients with recurrent, refractory, or progressive disease after ≥1 standard therapy, demonstrated meaningful activity with a 1-year PFS of 85%.

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